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biotecMAX™ 2022 Insight
Lynparza (olaparib)
Lynparza approved in the EU as adjuvant treatment for patients with germline BRCA-mutated HER2-negative high-risk early breast cancer
PUBLISHED4 August 2022
4 August 2022 07:00 BST
First and only approved medicine targeting
BRCA mutations in early breast cancer
AstraZeneca and MSD’s Lynparza (olaparib) has been approved in the European Union (EU) as monotherapy or in combination with endocrine therapy for the adjuvant treatment of adult patients with germline BRCA1/2 mutations (gBRCAm), who have human epidermal growth factor receptor 2 (HER2)-negative high-risk early breast cancer previously treated with neoadjuvant or adjuvant chemotherapy.
This approval by the European Commission was based on results from the OlympiA Phase III trial published in The New England Journal of Medicine in June 2021 and follows the recommendation for approval in the EU by the Committee for Medicinal Products for Human Use of Lynparza in this setting.1 In the trial, Lynparza demonstrated a statistically significant and clinically meaningful improvement in invasive disease-free survival (iDFS), reducing the risk of invasive breast cancer recurrences, new cancers, or death by 42% versus placebo (based on a hazard ratio [HR] of 0.58; 99.5% confidence interval [CI] 0.41-0.82; p<0.0001).
Lynparza also demonstrated a statistically significant and clinically meaningful improvement in overall survival (OS), reducing the risk of death by 32% versus placebo (based on a HR of 0.68; 98.5% CI 0.47-0.97; p=0.009). The safety and tolerability profile of Lynparza in this trial was in line with that observed in prior clinical trials.
Breast cancer is the most diagnosed cancer worldwide with an estimated 2.3 million patients diagnosed in 2020.2 Approximately 90% of all breast cancer patients worldwide are diagnosed with early breast cancer and BRCA mutations are found in approximately 10% of HER2-negative patients in Europe.3-5
Professor Andrew Tutt, Global Chair of the OlympiA Phase III trial and Professor of Oncology at The Institute of Cancer Research, London and King’s College London, said: “Today’s approval marks a new era of care in Europe for patients with an inherited form of breast cancer. For patients with high-risk early-stage breast cancer, including those with germline BRCA mutations, recurrence rates remain unacceptably high, with more than one in four of these patients seeing their cancer return following surgery and systemic treatment. Olaparib is the first PARP inhibitor to demonstrate improved overall survival for high-risk early-stage breast cancer patients with germline BRCA mutations and I am hopeful it will become a new standard of care.”
Dave Fredrickson, Executive Vice President, Oncology Business Unit, AstraZeneca, said:
“With this approval, Lynparza is now the first and only PARP inhibitor available for patients with germline BRCA-mutated HER2-negative early breast cancer in Europe. We can now bring the benefits of Lynparza to this earlier setting to help reduce the risk of life-threatening recurrence.”
Dr Eliav Barr, Senior Vice President, Head of Global Clinical Development and Chief Medical Officer, MSD Research Laboratories, said: “Today’s approval offers patients with germline BRCA-mutated HER2-negative early-stage breast cancer a new, much-needed treatment option. Lynparza as adjuvant treatment can significantly reduce the risk of disease recurrence and death, reinforcing the importance of conducting germline BRCA testing as soon as possible after diagnosis.”
In March 2022, Lynparza was approved in the US for the treatment of gBRCAm, HER2-negative high-risk early breast cancer. Lynparza is also approved in the US, EU, Japan, and many other countries for the treatment of patients with gBRCAm, HER2-negative, metastatic breast cancer previously treated with chemotherapy based on results from the OlympiAD Phase III trial. In the EU, this indication also includes patients with locally advanced breast cancer.
OlympiA
OlympiA is a Phase III, double-blind, parallel group, placebo-controlled, multicentre trial testing the efficacy and safety of Lynparza tablets versus placebo as adjuvant treatment in patients with gBRCAm high-risk HER2-negative early breast cancer, who have completed definitive local treatment and neoadjuvant or adjuvant chemotherapy.12
The primary endpoint of the trial was iDFS defined as time from randomisation to date of first locoregional or distant recurrence or new cancer or death from any cause.1
The OlympiA Phase III trial is led by the Breast International Group in partnership with the Frontier Science & Technology Research Foundation, NRG Oncology, the US National Cancer Institute, AstraZeneca and MSD. The trial is sponsored by NRG Oncology in the US and by AstraZeneca outside the US.
BRCA
BRCA1 and BRCA2 are human genes that produce proteins responsible for repairing damaged DNA and play an important role maintaining the genetic stability of cells.9 When either of these genes is mutated or altered such that its protein product either is not made or does not function correctly, DNA damage may not be repaired properly, and cells become unstable. As a result, cells are more likely to develop additional alterations that can lead to cancer. Cancers with BRCA mutations are more likely to be sensitive to PARP inhibitors including Lynparza.13-16
Lynparza
Lynparza (olaparib) is a first-in-class PARP inhibitor and the first targeted treatment to block DNA damage response (DDR) in cells/tumours harbouring a deficiency in homologous recombination repair (HRR), such as those with mutations in BRCA1 and/or BRCA2, or those where deficiency is induced by other agents (such as new hormonal agents – NHAs).
Inhibition of PARP proteins with Lynparza leads to the trapping of PARP bound to DNA single-strand breaks, stalling of replication forks, their collapse and the generation of DNA double-strand breaks and cancer cell death.
Lynparza is currently approved in a number of countries across PARP-dependent tumour types with defects and dependencies in the DDR pathway including maintenance treatment of platinum-sensitive relapsed ovarian cancer and as both monotherapy and in combination with bevacizumab for the 1st-line maintenance treatment of BRCA-mutated (BRCAm) and homologous recombination repair deficient (HRD)-positive advanced ovarian cancer, respectively; for gBRCAm, HER2-negative metastatic breast cancer (in the EU and Japan this includes locally advanced breast cancer); for gBRCAm, HER2-negative high-risk early breast cancer (in the EU and US); for gBRCAm metastatic pancreatic cancer; and HRR gene-mutated metastatic castration-resistant prostate cancer (BRCAm only in the EU and Japan).
Lynparza, which is being jointly developed and commercialised by AstraZeneca and MSD, is the foundation of AstraZeneca's industry-leading portfolio of potential new medicines targeting DDR mechanisms in cancer cells.
The AstraZeneca and MSD strategic oncology collaboration
In July 2017, AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US, known as MSD outside the US and Canada, announced a global strategic oncology collaboration to co-develop and co-commercialise Lynparza, the world’s first PARP inhibitor, and Koselugo (selumetinib), a mitogen-activated protein kinase (MEK) inhibitor, for multiple cancer types.
Working together, the companies will develop Lynparza and Koselugo in combination with other potential new medicines and as monotherapies. The companies will develop Lynparza and Koselugo in combination with their respective PD-L1 and PD-1 medicines independently.
AstraZeneca
AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialisation of prescription medicines in Oncology, Rare Diseases, and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca.
The AstraZeneca and MSD strategic oncology collaboration
In July 2017, AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US, known as MSD outside the US and Canada, announced a global strategic oncology collaboration to co-develop and co-commercialise Lynparza, the world’s first PARP inhibitor, and Koselugo (selumetinib), a mitogen-activated protein kinase (MEK) inhibitor, for multiple cancer types.
Working together, the companies will develop Lynparza and Koselugo in combination with other potential new medicines and as monotherapies. The companies will develop Lynparza and Koselugo in combination with their respective PD-L1 and PD-1 medicines independently.
AstraZeneca/Merck's Lynparza gets approval in EU for expanded use in breast cancer subtype
Aug. 04, 2022 5:41 AM ET
By: Ravikash, SA News Editor
- AstraZeneca (NASDAQ:AZN) and Merck's (NYSE:MRK) medicine Lynparza was approved for expanded use in treating certain patients with breast cancer in the EU.
- https://seekingalpha.com/news/3866864-astrazenecamercks-lynparza-gets-approval-in-eu-for-expanded-use-in-breast-cancer-subtype
- https://seekingalpha.com/symbol/MRK
- https://seekingalpha.com/symbol/AZN
- https://www.lynparza.com/
MYFEMBREE® (relugolix, estradiol, and norethindrone acetate)
Myovant Sciences and Pfizer Receive U.S. FDA Approval of MYFEMBREE®, a Once-Daily Treatment for the Management of Moderate to Severe Pain Associated With Endometriosis
Friday, August 05, 2022 - 09:15pm
• Data from the Phase 3 SPIRIT program showed MYFEMBREE reduced menstrual pain and non-menstrual pelvic pain in premenopausal women with endometriosis, and a loss of mean bone mineral density of less than 1% from baseline through one year of treatment
• Myovant and Pfizer will continue to jointly commercialize MYFEMBREE, with product available immediately
• Myovant to host conference call and webcast on Monday, August 8, 2022, at 8:30 a.m. Eastern Time / 5:30 a.m. Pacific Time
BASEL, Switzerland and NEW YORK, [August 5] (GLOBE NEWSWIRE) -- Myovant Sciences (NYSE: MYOV) and Pfizer Inc. (NYSE: PFE) today announced that the U.S. Food and Drug Administration (FDA) has approved MYFEMBREE® (relugolix 40 mg, estradiol 1 mg, and norethindrone acetate 0.5 mg) as a one-pill, once-a-day therapy for the management of moderate to severe pain associated with endometriosis in pre-menopausal women, with a treatment duration of up to 24 months. The approval is supported by one-year efficacy and safety data, including 24-week data from the Phase 3 SPIRIT 1 and SPIRIT 2 trials, which were published in The Lancet, and the first 28 weeks of an open-label extension study for eligible women who completed either SPIRIT 1 or SPIRIT 2. MYFEMBREE also is approved for heavy menstrual bleeding associated with uterine fibroids in pre-menopausal women. Myovant and Pfizer will continue to jointly commercialize MYFEMBREE in the U.S. and product is available immediately.
“Endometriosis is a painful, chronic disease with limited therapies to manage symptoms,” said Juan Camilo Arjona Ferreira, M.D., Chief Medical Officer of Myovant Sciences, Inc. “The new MYFEMBREE indication helps advance our mission to redefine care for women by helping address a disease with high unmet need, giving women and physicians a new meaningful treatment option to manage moderate to severe pain associated with endometriosis.”
“This approval is an important milestone reflecting Pfizer and Myovant’s commitment to women’s health in areas of significant unmet need,” said James Rusnak, M.D., Ph.D., Senior Vice President, Chief Development Officer, Internal Medicine and Hospital, Global Product Development at Pfizer. “We look forward to making MYFEMBREE available to women with endometriosis and broadening their options in managing this complex disorder.”
MYFEMBREE offers an effective, once-daily treatment option for the management of moderate to severe pain associated with endometriosis, with a treatment duration of up to 24 months. Endometriosis is a serious chronic condition that requires long-term interventions. Optimization of medical therapies is the recommended treatment paradigm. 1,2,3 MYFEMBREE introduces an option for up to two years of pharmacological management of moderate to severe pain associated with endometriosis in pre-menopausal women.
“The data from the SPIRIT studies showed the clinical benefit that relugolix combination therapy can have on moderate to severe pain associated with endometriosis and how it can impact patients,” said Linda Giudice, M.D., Ph.D., Distinguished Professor at the University of California, San Francisco (UCSF), and Chair, SPIRIT Program Steering Committee. “This newly approved option for patients with pain from endometriosis offers the convenience of one pill taken once daily with a mean change in bone mineral density of <1% that did not appear to worsen at 12 months of treatment; however, monitoring is recommended.”
This approval is supported by one-year data from the Phase 3 SPIRIT program, which included two 24-week multi-national clinical studies (SPIRIT 1 and SPIRIT 2) in more than 1,200 women with pain associated with endometriosis, as well as the first 28 weeks of an open-label extension study to assess its longer-term use. Overall, these studies showed MYFEMBREE reduced menstrual pain and non-menstrual pelvic pain with a loss of mean bone mineral density of less than 1% from baseline through one year of treatment.4
SPIRIT 1 and 2 each met their co-primary endpoints with 75% of women in the MYFEMBREE group in both studies achieving a clinically meaningful reduction in dysmenorrhea compared with 27% and 30% of women in the placebo groups at Week 24, respectively (both p <0.0001). For non-menstrual pelvic pain, treatment with MYFEMBREE demonstrated a clinically meaningful reduction in pain in 59% and 66% of women, compared with 40% and 43% of women in the placebo groups (p < 0.0001). Adverse reactions occurring in at least 3% of women treated with MYFEMBREE and greater than placebo were: headache, vasomotor symptoms, mood disorders, abnormal uterine bleeding, nausea, toothache, back pain, decreased sexual desire and arousal, arthralgia, fatigue, and dizziness.
The open-label extension study for eligible women who completed either SPIRIT 1 or SPIRIT 2 showed mean bone mineral density loss of less than 1% from baseline through one year of treatment; some patients (19.7%) had losses >3%. Annual bone density measurement is recommended while treating women for endometriosis.
MYFEMBREE is available immediately to patients with moderate to severe pain associated with endometriosis with a prescription from their healthcare provider. Myovant and Pfizer also are committed to supporting women in the U.S. who are prescribed MYFEMBREE throughout their treatment journeys. The MYFEMBREE Support Program provides access support services, including insurance benefits checks, prior authorization support, co-pay support for commercially insured patients, and patient assistance for qualifying uninsured patients. Program terms and conditions apply. For more information and additional resources, please contact 833-MYFEMBREE (833-693-3627), 8 a.m. – 8 p.m. Eastern Time, Monday – Friday.
About MYFEMBREE®
MYFEMBREE (relugolix, estradiol, and norethindrone acetate) is a once-daily oral treatment approved by the U.S. Food and Drug Administration for the management of moderate to severe pain associated with endometriosis, with a treatment duration of up to 24 months. It is also currently available in the U.S. for the management of heavy menstrual bleeding associated with uterine fibroids in premenopausal women, with a treatment duration of up to 24 months. MYFEMBREE contains relugolix, which reduces the amount of estrogen (and other hormones) produced by ovaries, estradiol (an estrogen) which may reduce the risk of bone loss, and norethindrone acetate (a progestin) which is necessary when women with a uterus (womb) take estrogen.
For full prescribing information including Boxed Warning and patient information, please click here.
Indications and Usage
MYFEMBREE is indicated in premenopausal women for the management of:
• Heavy menstrual bleeding associated with uterine leiomyomas (fibroids)
• Moderate to severe pain associated with endometriosis
Limitations of Use: Use of MYFEMBREE should be limited to 24 months due to the risk of continued bone loss which may not be reversible.
About Myovant Sciences
Myovant Sciences aspires to redefine care for women and for men through purpose-driven science, empowering medicines, and transformative advocacy. Founded in 2016, Myovant has executed five successful Phase 3 clinical trials across oncology and women’s health leading to three regulatory approvals by the U.S. Food and Drug Administration (FDA) for men with advanced prostate cancer, women with heavy menstrual bleeding associated with uterine fibroids, and pre-menopausal women with moderate to severe pain associated with endometriosis, respectively. Myovant also has received regulatory approvals by the European Commission (EC) and the United Kingdom Medicines and Healthcare Products Regulatory Agency (MHRA) for women with symptomatic uterine fibroids and for men with advanced hormone-sensitive prostate cancer. Myovant has a supplemental New Drug Application under review with the FDA for updates to the United States Prescribing Information (USPI) based on safety and efficacy data from the Phase 3 LIBERTY randomized withdrawal study (RWS) of MYFEMBREE in premenopausal women with heavy menstrual bleeding due to uterine fibroids for up to two years. Myovant also is conducting a Phase 3 study to evaluate the prevention of pregnancy in women with uterine fibroids or endometriosis. Myovant also is developing MVT-602, an investigational oligopeptide kisspeptin-1 receptor agonist, which has completed a Phase 2a study for female infertility as part of assisted reproduction. Sumitovant Biopharma, Ltd., a wholly owned subsidiary of Sumitomo Pharma Co., Ltd., is Myovant’s majority shareholder. For more information, please visit www.myovant.com. Follow @Myovant on Twitter and LinkedIn.
Myovant, Pfizer win approval of Myfembree for pain associated with endometriosis
Aug. 06, 2022 6:48 AM ET
By: Jonathan Block, SA News Editor2 Comments
- The U.S. FDA has approved Myfembree from Myovant Sciences (NYSE:MYOV) and Pfizer (NYSE:PFE) for pain associated with endometriosis in pre-menopausal women.
- https://seekingalpha.com/news/3868520-myovant-pfizer-win-approval-of-myfembree-for-pain-associated-with-endometriosis
- https://seekingalpha.com/symbol/MYOV
- https://seekingalpha.com/symbol/PFE
- https://www.myfembree.com/
- https://www.pfizer.com/
ENHERTU® (trastuzumab deruxtecan; fam-trastuzumab deruxtecan-nxki in the U.S. only)
ENHERTU® Granted Priority Review in the U.S. for Patients with HER2 Low Metastatic Breast Cancer • Based on DESTINY-Breast04 results which showed Daiichi Sankyo and AstraZeneca’s ENHERTU is the first HER2 directed therapy to demonstrate a survival benefit in this population • Application being evaluated under FDA Real-Time Oncology Review and Project Orbis Tokyo and Basking Ridge, NJ – (July 25, 2022) – Daiichi Sankyo (TSE: 4568) and AstraZeneca (LSE/STO/Nasdaq: AZN) have received notification of acceptance by the U.S. Food and Drug Administration (FDA) of the supplemental Biologics License Application (sBLA) of ENHERTU® (famtrastuzumab deruxtecan-nxki) for the treatment of adult patients with unresectable or metastatic HER2 low (immunohistochemistry (IHC) 1+ or IHC 2+/in-situ hybridization (ISH)-negative) breast cancer who have received a prior therapy in the metastatic setting. The application has been granted Priority Review. ENHERTU is a specifically engineered HER2 directed antibody drug conjugate (ADC) being jointly developed and commercialized by Daiichi Sankyo and AstraZeneca. The FDA grants Priority Review to applications for medicines that, if approved, would offer significant improvements over available options by demonstrating safety or efficacy improvements, preventing serious conditions or enhancing patient compliance. The Prescription Drug User Fee Act date (PDUFA), the FDA action date for their regulatory decision, is during the fourth quarter of the 2022 calendar year. The Priority Review follows receipt of Breakthrough Therapy Designation, granted by the FDA in April 2022 for ENHERTU in metastatic HER2 low breast cancer. The sBLA is being reviewed under the Real-Time Oncology Review (RTOR) program and Project Orbis, two initiatives of the FDA which are designed to bring safe and effective cancer treatments to patients as early as possible. RTOR allows the FDA to review components of an application before submission of the complete application. Project Orbis provides a framework for concurrent submission and review of oncology medicines among participating international partners. “The results seen in the DESTINY-Breast04 trial represent a significant advance and reinforce the potential for ENHERTU to become a new standard of care for patients with previously treated HER2 low metastatic breast cancer,” said Ken Takeshita, MD, Global Head, R&D, Daiichi Sankyo. “The prioritization of this 2 application by the FDA and inclusion in both the Real-Time Oncology Review and Project Orbis initiatives support the importance of these data, and we look forward to working with the FDA to potentially bring ENHERTU to patients with HER2 low metastatic breast cancer as quickly as possible.” “The data from DESTINY-Breast04 represent the first time a HER2 targeted therapy has shown a survival benefit in patients with HER2 low metastatic breast cancer,” said Susan Galbraith, MBBChir, PhD, Executive Vice President, Oncology R&D, AstraZeneca. “For more than two decades, only patients with HER2 positive breast cancer have been able to benefit from HER2 targeted therapies. If approved, ENHERTU will redefine how we classify and treat metastatic breast cancer, enabling patients whose tumors have lower levels of HER2 expression the opportunity to benefit from a HER2 directed therapy.” The sBLA is based on data from the DESTINY-Breast04 phase 3 trial recently presented at the presidential plenary session of the American Society of Clinical Oncology (#ASCO22) Annual Meeting and simultaneously published in The New England Journal of Medicine. In DESTINY-Breast04, ENHERTU demonstrated superior and clinically meaningful efficacy in progression-free survival (PFS) and overall survival (OS) in previously treated patients with HER2 low unresectable and/or metastatic breast cancer with hormone receptor (HR) positive or HR negative disease versus standard of care physician’s choice of chemotherapy. The safety profile of ENHERTU was consistent with previous clinical trials with no new safety concerns identified. Interstitial lung disease (ILD) or pneumonitis rates were consistent with those observed in other late-line HER2 positive breast cancer trials of ENHERTU, as determined by an independent adjudication committee. About DESTINY-Breast04 DESTINY-Breast04 is a global, randomized, open-label, pivotal phase 3 trial evaluating the efficacy and safety of ENHERTU (5.4 mg/kg) versus physician’s choice of chemotherapy (capecitabine, eribulin, gemcitabine, paclitaxel or nab-paclitaxel) in patients with HR positive or HR negative, HER2 low unresectable and/or metastatic breast cancer previously treated with one or two prior lines of chemotherapy. Patients were randomized 2:1 to receive either ENHERTU or chemotherapy. The primary endpoint of DESTINY-Breast04 is PFS in patients with HR positive disease based on blinded independent central review (BICR). Key secondary endpoints include PFS based on BICR in all randomized patients (HR positive and HR negative disease), OS in patients with HR positive disease and OS in all randomized patients (HR positive and HR negative disease). Other secondary endpoints include PFS based on investigator assessment, objective response rate based on BICR and on investigator assessment, duration of response based on BICR and safety. DESTINY-Breast04 enrolled 557 patients at multiple sites in Asia, Europe and North America. For more information about the trial, visit ClinicalTrials.gov.
About ENHERTU ENHERTU® (trastuzumab deruxtecan; fam-trastuzumab deruxtecan-nxki in the U.S. only) is a HER2 directed ADC. Designed using Daiichi Sankyo’s proprietary DXd ADC technology, ENHERTU is the lead ADC in the oncology portfolio of Daiichi Sankyo and the most advanced program in AstraZeneca’s ADC scientific platform. ENHERTU consists of a HER2 monoclonal antibody attached to a topoisomerase I inhibitor payload, an exatecan derivative, via a stable tetrapeptide-based cleavable linker. ENHERTU (5.4 mg/kg) is approved in more than 30 countries for the treatment of adult patients with unresectable or metastatic HER2 positive breast cancer who have received a prior anti-HER2-based regimen either in the metastatic setting, or in the neoadjuvant or adjuvant setting and have developed disease recurrence during or within six months of completing therapy, based on results from the DESTINY-Breast03 4 trial. ENHERTU also is approved in several countries for the treatment of adult patients with unresectable or metastatic HER2 positive breast cancer who have received two or more prior anti-HER2-based regimens based on the results from the DESTINY-Breast01 trial. ENHERTU (6.4 mg/kg) is approved in several countries for the treatment of adult patients with locally advanced or metastatic HER2 positive gastric or gastroesophageal junction (GEJ) adenocarcinoma who have received a prior trastuzumab-based regimen based on the results from the DESTINY-Gastric01 trial. ENHERTU is approved in the U.S. with Boxed WARNINGS for Interstitial Lung Disease and Embryo-Fetal Toxicity. For more information, please see the accompanying full Prescribing Information, including Boxed WARNINGS, and Medication Guide. About the ENHERTU Clinical Development Program A comprehensive global development program is underway evaluating the efficacy and safety of ENHERTU monotherapy across multiple HER2 targetable cancers including breast, gastric, lung and colorectal cancers. Trials in combination with other anticancer treatments, such as immunotherapy, also are underway. Regulatory applications for ENHERTU are currently under review in China, Japan and several other countries for the treatment of adult patients with HER2 positive unresectable or metastatic breast cancer who have received a prior anti-HER2-based regimen based on the results from the DESTINY-Breast03 trial. ENHERTU is under review in Europe and Japan for the treatment of adult patients with unresectable or metastatic HER2 low (IHC 1+ or IHC 2+/ISH-negative) breast cancer who have received a prior systemic therapy in the metastatic setting or developed disease recurrence during or within six months of completing adjuvant chemotherapy, based on the results from the DESTINY-Breast04 trial. Patients with HR positive breast cancer must additionally have received or be ineligible for endocrine therapy. ENHERTU also is currently under review in the U.S. for the treatment of adult patients with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have a HER2 (ERBB2) mutation and who have received a prior systemic therapy based on the results from the DESTINY-Lung01 trial, and in Europe for the treatment of adult patients with locally advanced or metastatic HER2 positive gastric or GEJ adenocarcinoma who have received a prior anti-HER2-based regimen based on the DESTINY-Gastric01 and DESTINY-Gastric02 trials. 5 About the Daiichi Sankyo and AstraZeneca Collaboration Daiichi Sankyo Company, Limited (referred to as Daiichi Sankyo) and AstraZeneca entered into a global collaboration to jointly develop and commercialize ENHERTU in March 2019 and datopotamab deruxtecan (Dato-DXd) in July 2020, except in Japan where Daiichi Sankyo maintains exclusive rights for each ADC. Daiichi Sankyo is responsible for the manufacturing and supply of ENHERTU and datopotamab deruxtecan.
Indications ENHERTU is a HER2-directed antibody and topoisomerase inhibitor conjugate indicated for the treatment of adult patients with: • Unresectable or metastatic HER2-positive breast cancer who have received a prior anti-HER2-based regimen either: – In the metastatic setting, or – In the neoadjuvant or adjuvant setting and have developed disease recurrence during or within six months of completing therapy • Locally advanced or metastatic HER2-positive gastric or gastroesophageal junction adenocarcinoma who have received a prior trastuzumab-based regimen
Please see accompanying full Prescribing Information, including Boxed WARNINGS, and Medication Guide. About Daiichi Sankyo Daiichi Sankyo is dedicated to creating new modalities and innovative medicines by leveraging our world-class science and technology for our purpose “to contribute to the enrichment of quality of life around the world.” In addition to our current portfolio of medicines for cancer and cardiovascular disease, Daiichi Sankyo is primarily focused on developing novel therapies for people with cancer as well as other diseases with high unmet medical needs. With more than 100 years of scientific expertise and a presence in more than 20 countries, Daiichi Sankyo and its 16,000 employees around the world draw upon a rich legacy of innovation to realize our 2030 Vision to become an “Innovative Global Healthcare Company Contributing to the Sustainable Development of Society.” For more information, please visit: www.daiichisankyo.com.
AstraZeneca, Daiichi Sankyo win FDA label expansion for Enhertu in HER2-low breast cancer
Aug. 05, 2022 12:16 PM ET
AstraZeneca PLC (AZN)DSKYF, DSNKY
By: Dulan Lokuwithana, SA News Editor1 Comment
- The U.S. Food and Drug Administration (FDA) on Friday approved the cancer medication Enhertu, developed by AstraZeneca (NASDAQ:AZN) and its Japanese partner Daiichi Sankyo (OTCPK:DSKYF) (OTCPK:DSNKY), for adult patients with HER2-low breast cancer.
- https://seekingalpha.com/news/3868282-astrazeneca-daiichi-sankyo-win-fda-label-expansion-for-enhertu
- https://seekingalpha.com/symbol/AZN
- https://www.astrazeneca.com/
- https://seekingalpha.com/symbol/DSKYF
- https://seekingalpha.com/symbol/DSNKY
- https://www.enhertu.com/en
Veklury® (Remdesivir)
July 22, 2022
CHMP Adopts Positive Opinion Recommending Veklury® (Remdesivir) Receive Full Marketing Authorization for the Treatment of Patients With COVID-19
-- If Granted by the European Commission, Veklury will Become the Only Direct-Acting Antiviral with Full Marketing Authorization in the EU --
FOSTER CITY, Calif.--(BUSINESS WIRE)-- Gilead Sciences, Inc. (Nasdaq: GILD) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Commission (EC) adopted a positive opinion on the fulfillment of the last specific obligation and recommended the granting of Marketing Authorization (MA) for Veklury® (remdesivir) that is no longer subject to specific obligations. Veklury was initially granted a conditional marketing authorization in July 2020 for the treatment of COVID-19 in adults and adolescents (from 12 years of age and weighing at least 40 kilograms) with pneumonia requiring supplemental oxygen (low- or high-flow oxygen or other non-invasive ventilation at the start of treatment). In December of 2021, the conditional authorization was expanded to include adults who do not require supplemental oxygen and are at increased risk of developing severe COVID-19. The EC will review the CHMP recommendation and, pending adoption, Veklury will be fully authorized for these patients with COVID-19.
“We welcome the Committee’s positive opinion recommending a full marketing authorization for Veklury. Veklury continues to demonstrate durable activity against SARS-CoV-2 as it evolves, and it is the most used antiviral in hospitalized patients,” said Merdad Parsey, MD, PhD, Chief Medical Officer of Gilead. “More than two years into this pandemic, it’s critical to continue to secure access to effective treatments. We are proud of the role Veklury plays as the COVID-19 antiviral standard of care for hospitalized patients, and we remain committed to make it available to all patients that can benefit from it. We look forward to the EC’s decision.”
This positive opinion is based upon the fulfilment of the last specific obligation for Veklury, which included the review of virology data inclusive of in vitro data showing Veklury retains activity against variants of concern, including Alpha, Beta, Gamma, Delta and Omicron (BA.1 and BA.2). In addition, an assessment of the current risk-benefit of Veklury, which considered the efficacy and safety data accumulated since the initial granting of the conditional marketing authorization, was reviewed by the CHMP to support the change to a full marketing authorization.
In the European Economic Area (EEA), Veklury is the only antiviral indicated for both the treatment of COVID-19 in adult and adolescent patients with pneumonia requiring supplemental oxygen (low- or high-flow oxygen or other non-invasive ventilation) and adults who do not require supplemental oxygen and are at increased risk of developing severe COVID-19.
About Veklury
Veklury (remdesivir) is a nucleotide analog invented by Gilead, building on more than a decade of the company’s antiviral research. Veklury is a foundation for the treatment of hospitalized patients with COVID-19 and is a recommended treatment for reducing disease progression in non-hospitalized patients at high risk of disease progression. Veklury has an established safety profile and minimal drug interactions in diverse populations. It can help reduce disease progression across a spectrum of disease severity and enable patients to recover faster, freeing up limited hospital resources and saving healthcare systems money.
Veklury directly inhibits viral replication inside of the cell by targeting the SARS-CoV-2 viral RNA polymerase. Based on sequence analyses, Veklury should remain active against Omicron BA.4 and BA.5, as there are no new substitutions in the polymerase of BA.4 and BA.5. This suggests that Veklury will continue to be active against known Omicron variants. Gilead continuously evaluates the activity of Veklury against new SARS-CoV-2 variants of concern as they emerge around the world.
Veklury is approved in 50 countries worldwide. To date, Veklury and generic remdesivir have been made available to more than 11 million patients around the world, including more than 7 million people in 127 middle- and low-income countries through Gilead’s voluntary licensing program. These licenses currently remain royalty-free, reflecting Gilead’s existing commitment to enabling broad patient access to remdesivir.
U.S. Indication for Veklury
Veklury® (remdesivir 100 mg for injection) is indicated for the treatment of COVID-19 in adults and pediatric patients (≥28 days old and weighing ≥3 kg) with positive results of SARS-CoV-2 viral testing, who are:
- Hospitalized, or
- Not hospitalized and have mild-to-moderate COVID-19 and are at high risk for progression to severe COVID-19, including hospitalization or death.
For more information, please see the U.S. full Prescribing Information available at www.gilead.com.
About Gilead Sciences
Gilead Sciences, Inc. is a biopharmaceutical company that has pursued and achieved breakthroughs in medicine for more than three decades, with the goal of creating a healthier world for all people. The company is committed to advancing innovative medicines to prevent and treat life-threatening diseases, including HIV, viral hepatitis and cancer. Gilead operates in more than 35 countries worldwide, with headquarters in Foster City, California.
U.S. full Prescribing Information for Veklury is available at www.gilead.com.
Veklury, Gilead and the Gilead logo are registered trademarks of Gilead Sciences, Inc., or its related companies.
For more information about Gilead, please visit the company’s website at www.gilead.com, follow Gilead on Twitter (@Gilead Sciences) or call Gilead Public Affairs at 1-800-GILEAD-5 or 1-650-574-3000.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220721005784/en/
Source: Gilead Sciences, Inc.
Gilead COVID-19 drug Veklury gets EMA panel nod for full approval in EU
Jul. 22, 2022 7:43 AM ET
By: Ravikash, SA News Editor1 Comment
- A committee of the European Medicines Agency (EMA) recommended granting full marketing authorization to Gilead Sciences (NASDAQ:GILD) Veklury (remdesivir) to treat patients with COVID-19.
- https://seekingalpha.com/news/3859553-gilead-covid-19-drug-veklury-gets-ema-panel-nod-for-full-approval-in-eu
- https://seekingalpha.com/symbol/GILD
- https://www.veklury.com/
- https://www.gilead.com/
STELARA® (ustekinumab)
STELARA® (ustekinumab) Approved by the U.S. Food and Drug Administration to Treat Pediatric Patients with Active Psoriatic ArthritisAs the first and only biologic targeting both cytokines interleukin (IL)-12 and IL-23, STELARA provides a new therapeutic option for children six years of age and older living with active psoriatic arthritis
Active psoriatic arthritis in pediatric patients is a rare disease affecting five to eight percent of children and adolescents with chronic inflammatory arthritis*1-6
HORSHAM, PENNSYLVANIA, August 1, 2022 – The Janssen Pharmaceutical Companies of Johnson & Johnson today announced that the U.S. Food and Drug Administration (FDA) has approved STELARA® (ustekinumab) for the treatment of pediatric patients six years of age and older with active psoriatic arthritis (PsA). This rare disease that resembles adult PsA affects five to eight percent of children and adolescents with chronic inflammatory arthritis.*1-7 Two of the four indications for STELARA now include pediatric patients, further expanding its treatment profile since the first approval in 2009 for adults living with moderate to severe plaque psoriasis (PsO).
STELARA is a fully human monoclonal antibody that selectively inhibits both interleukin (IL)-12 and IL-23, two cytokines thought to play an important role in tempering the overactive inflammatory response in several autoimmune diseases. STELARA is administered as a subcutaneous injection dosed four times per year after two starter doses for the treatment of pediatric patients six years of age and older with active PsA.
“We know active pediatric psoriatic arthritis is a challenging inflammatory disease given its rarity and that symptoms, such as swollen joints and skin lesions, can vary significantly in presentation and severity,” said Terence Rooney, M.D., Ph.D., Vice President, Rheumatology and Maternal Fetal Disease Area, Janssen Research & Development, LLC. “With this pediatric approval of STELARA, we’re pleased to help address the unmet needs of these young patients and provide physicians with a much-needed treatment option that has an established track record of safety and efficacy.”
The FDA’s approval was based on pharmacokinetic (PK) data and extrapolation of the established efficacy and existing safety profile of STELARA in multiple Phase 3 studies in adult and pediatric patients with moderate to severe plaque PsO (PSTELLAR, CADMUS, and CADMUS Jr) and adult patients with active PsA (PSUMMIT I and II). With the limited availability of pediatric PsA patients for inclusion in clinical trials, researchers utilized an extrapolation approach based on previous PK, efficacy and safety observations from a closely adjacent population of pediatric patients with moderate to severe plaque PsO who also had active PsA, as well as adult patients with moderate to severe plaque PsO or active PsA. An analysis of the data demonstrated that PK exposure of STELARA in these pediatric PsO patients with active PsA was consistent with that of Phase 3 clinical trials of STELARA in pediatric PsO patients without active PsA, as well as with adult patients with moderate to severe plaque PsO or adult patients with active PsA, while data on common efficacy endpoints were similar in these pediatric PsO patients with active PsA.
“The approval of STELARA for use in children six years of age and older with active psoriatic arthritis, which follows the 2020 approval for moderate to severe plaque psoriasis in this population, is complemented by more than 12 years of clinical trial and real-world evidence across all approved indications demonstrating the safety and efficacy of this biologic therapy,” said Jennifer Davidson, DO, Vice President of Immunology Medical Affairs, Janssen Scientific Affairs, LLC. “As a global leader in immunology, Janssen is dedicated to reducing the burden of chronic autoimmune diseases, and this additional approval for STELARA builds on our legacy of bringing important treatment options to younger patients.”
Access to STELARA®
Janssen is actively working toward greater patient accessibility through improved commercial first-line formulary coverage, as well as patient-specific support services specifically for patients to start and stay on STELARA® treatment after a prescribing decision has been made.
STELARA withMe offers a comprehensive support program that helps patients get started on STELARA and stay on track. STELARA withMe provides information on insurance coverage, potential out-of-pocket costs, and treatment support, and identifies options that may help make treatment more affordable, including the STELARA withMe Savings Program for commercially insured patients who are eligible.
About Active Psoriatic Arthritis in Pediatric Patients
Active psoriatic arthritis (PsA) in pediatric patients, a rare disease that resembles adult PsA, affects five to eight percent of children and adolescents with chronic inflammatory arthritis.*1-7 Symptoms of active pediatric PsA can vary significantly in presentation and severity from patient to patient, but often include joint inflammation and skin lesions.8 PsA can be a challenging disease to treat – especially in younger populations – reinforcing the need for additional treatment options.
About PSTELLAR
PSTELLAR, a Phase 3b, randomized, double-blind, active-controlled, multicenter study assessed the effect of extending maintenance dosing intervals beyond 12 weeks on the clinical efficacy and safety of STELARA in patients with moderate to severe plaque psoriasis. Adults with moderate to severe plaque psoriasis received STELARA at weeks 0, 4 and 16 during open-label treatment. Patients achieving a week-28 Physician's Global Assessment (PGA) score of cleared/minimal (PGA = 0/1) were randomized 1:4 to group 1 [approved every 12 weeks (q12 wk) maintenance] or group 2 (q12-24 wk; response-based dosing determined by time to loss of PGA = 0/1). Key endpoints included the number of visits with PGA = 0/1 (primary end point) and ≥ 75 percent improvement in Psoriasis Area and Severity Index (PASI 75) between weeks 88 and 112, and PGA/PASI responses between weeks 28 and 112.
About CADMUS
CADMUS, a Phase 3, randomized, double-blind, placebo-controlled, parallel, multicenter trial, evaluated the efficacy and safety of STELARA in adolescent patients 12 to 17 years of age with moderate to severe plaque psoriasis. Patients (N=110) had been diagnosed with psoriasis more than six months prior to first study agent administration and had a Psoriasis Area Severity Index (PASI) score greater than or equal to 12, a Physician's Global Assessment (PGA) score greater than or equal to 3 and body surface area (BSA) involvement of at least 10 percent. In addition, patients were inadequately controlled with topical therapy or were candidates for systemic/phototherapy.
A Phase 3 study, CADMUS Jr, evaluated the efficacy and safety of STELARA in the treatment of pediatric patients 6 to 11 years of age living with moderate to severe plaque psoriasis.
About PSUMMIT
Two Phase 3 multicenter, randomized, double-blind, placebo-controlled trials of STELARA in adult patients with active psoriatic arthritis, PSUMMIT I and PSUMMIT II, evaluated the efficacy and safety of subcutaneously administered STELARA 45 mg or 90 mg at weeks 0, 4 and then every 12 weeks. The trials included adult patients diagnosed with active psoriatic arthritis who had at least five tender and five swollen joints and C-reactive protein (CRP) levels of at least 0.3 mg/dL despite previous treatment with conventional therapy. PSUMMIT II also included adult patients with previous exposure to tumor necrosis factor (TNF) inhibitors. The primary endpoints for both studies were the proportion of patients demonstrating at least a 20 percent improvement in arthritis signs and symptoms [American College of Rheumatology (ACR) 20] at week 24. Secondary endpoints at week 24 included in the submissions were: improvements in Health Assessment Questionnaire Disability Index (HAQ-DI) scores, a 50 or 70 percent improvement in arthritis signs and symptoms (ACR 50 or ACR 70), and at least a 75 percent improvement in psoriatic skin lesions as measured by the Psoriasis Area Severity Index (PASI 75) in patients with at least three percent body surface area involvement at baseline.
About STELARA® (ustekinumab)9
STELARA® (ustekinumab), a human interleukin (IL)-12 and IL-23 antagonist, is approved in the United States for the treatment of: 1) adults and children six years and older with moderate to severe plaque psoriasis who are candidates for phototherapy or systemic therapy; 2) adults and children six years and older with active psoriatic arthritis; 3) adult patients (18 years and older) with moderately to severely active Crohn’s disease; 4) adult patients (18 years and older) with moderately to severely active ulcerative colitis.
The Janssen Pharmaceutical Companies of Johnson & Johnson maintain exclusive worldwide marketing rights to STELARA.
* When other known causes of arthritis have been excluded.3,5,10
Please click to read the full Prescribing Information and Medication Guide for STELARA® and discuss any questions you have with your doctor.
About the Janssen Pharmaceutical Companies of Johnson & Johnson
At Janssen, we’re creating a future where disease is a thing of the past. We’re the Pharmaceutical Companies of Johnson & Johnson, working tirelessly to make that future a reality for patients everywhere by fighting sickness with science, improving access with ingenuity, and healing hopelessness with heart. We focus on areas of medicine where we can make the biggest difference: Cardiovascular, Metabolism, & Retina; Immunology; Infectious Diseases & Vaccines; Neuroscience; Oncology; and Pulmonary Hypertension.
Learn more at www.janssen.com. Follow us at www.twitter.com/JanssenGlobal and www.twitter.com/JanssenUS.
Janssen Research & Development, LLC, Janssen Biotech, Inc. and Janssen Scientific Affairs, LLC are part of the Janssen Pharmaceutical Companies of Johnson & Johnson.
Johnson & Johnson unit’s arthritis injection now indicated for children in U.S.
Aug. 01, 2022 1:12 PM ET
By: Dulan Lokuwithana, SA News Editor
- The Janssen unit of Johnson & Johnson (NYSE:JNJ) announced on Monday that the FDA approved a label expansion for its STELARA subcutaneous injection allowing its use in children aged six years of age and older with active psoriatic arthritis ((PsA)).
- https://seekingalpha.com/news/3863900-johnson-johnson-units-arthritis-injection-now-indicated-for-children-in-us
- https://seekingalpha.com/symbol/JNJ
- https://www.stelarainfo.com/
- https://www.janssen.com/
JUVÉDERM® VOLUX™ XC
August 3, 2022
FDA Approves JUVÉDERM® VOLUX™ XC for Improvement of Jawline Definition
JUVÉDERM® VOLUX™ XC IS THE FIRST AND ONLY HYALURONIC ACID (HA) FILLER TO RECEIVE U.S. FDA APPROVAL FOR IMPROVING JAWLINE DEFINITION
IRVINE, Calif., Aug. 3, 2022 /PRNewswire/ -- Today, Allergan Aesthetics, an AbbVie company (NYSE: ABBV), announced the U.S. FDA approval of JUVÉDERM® VOLUX™ XC for the improvement of jawline definition in adults over the age of 21 with moderate to severe loss of jawline definition. 1
"The approval of JUVÉDERM® VOLUX™ XC represents the largest leap in innovation for our U.S. HA portfolio since the introduction of JUVÉDERM® VOLUMA® XC, 2" said Carrie Strom, President, Global Allergan Aesthetics and Senior Vice President, AbbVie. "JUVÉDERM® VOLUX™ XC complements our existing product line to provide even more structure, cohesivity and lift capacity to create an improved jawline that appears more defined in real life and on camera. JUVÉDERM® VOLUX™ XC is what our providers have been asking for to deliver the jaw-dropping results their patients are seeking. 1"
As the category leader, the JUVÉDERM® Collection of Fillers offers the broadest portfolio of specifically designed treatment options, and this latest approval of JUVÉDERM® VOLUX™ XC marks the sixth product offering in the lineup alongside JUVÉDERM® VOLUMA® XC, JUVÉDERM® VOLLURE® XC, JUVÉDERM® Ultra Plus XC, JUVÉDERM® Ultra XC, and JUVÉDERM® VOLBELLA® XC.1-3,5-7
In the pivotal clinical study, JUVÉDERM® VOLUX™ XC was found to effectively improve jawline definition (69.9%, 102/146) at six months. Participants reported satisfaction using the Satisfaction with Lower Face and Jawline module of the FACE-Q questionnaire, most treatment group participants (82.3%, 116/141) reported satisfaction with the appearance of their lower face and jawline through 12 months following treatment with JUVÉDERM® VOLUX™ XC. 4 Additionally, 81.5% (119/146) of participants at six months were satisfied with how sculpted (well-defined) their jawline looked compared to 12.2% (19/156) at baseline. At six months, 70.5% (103/146) of participants were satisfied with how smooth their lower face looked (i.e., no jowls or folds of fatty skin) compared to 7.7% (12/156) at baseline and 73.1% (106/145) of participants at six months were satisfied with how nice their lower face looked compared to 9.0% (14/156) at baseline. 4
"Requests for treatment in the lower facial region transcend age, gender, race, and ethnicity in my practice. As people age, many factors can contribute to how the lower face changes, such as genetics and soft tissue loss," says Dr. Jeremy Green, a fellowship-trained board-certified Miami cosmetic dermatologist and pivotal clinical trial investigator. "This can cause reduced definition around the jawline area that may impact and change the shape of the face and lead to the appearance of jowls. Clinical trial participants reported high satisfaction with the results of their treatment. In fact, at six months post-treatment, 89.7% (131/146) of treatment group participants were willing to recommend the treatment to a friend, with the majority continuing to recommend treatment at 12 months (87.2%, 123/141). 4 I am excited to now be able to offer this treatment option to all of my patients seeking an improved jawline."
Commonly reported side effects in the clinical study included tenderness, lumps/bumps, pain, swelling, firmness, bruising, redness, itching, and discoloration at the injection sites, as reported in participants' 30-day daily diaries. These side effects are consistent with HA filler injection and were usually mild (causing little discomfort and no effect on daily activities) or moderate (causing some discomfort and effect on daily activities) in severity. Most of these side effects went away on their own within two weeks. Participants also reported similar side effects after maintenance injection. 1
Allergan Aesthetics is dedicated to training health care providers on safe and effective use of all its products. Allergan Medical Institute will be providing an in-depth product training program for JUVÉDERM® VOLUX™ XC, which will include facial anatomy, considerations for safe injection in this area, appropriate patient selection, and aseptic technique. Training will begin in fall of 2022. JUVÉDERM® VOLUX™ XC will be more broadly available to consumers in early 2023.
For more information on the JUVÉDERM® Collection of Fillers, visit Juvederm.com and follow @JUVÉDERM on Instagram.
Consumers and new patients who receive an aesthetic treatment with a product from the JUVÉDERM® Collection of Fillers can also enroll in Allē, Allergan Aesthetics' loyalty rewards program. Consumers can enroll to unlock access to curated content, exclusive offers, and personalized rewards that can be used for savings on the Allergan Aesthetics portfolio of products and redeemed at a participating provider's office, subject to applicable program terms and conditions. Allē is the first and only loyalty program in the aesthetics market to also offer consumers the ability to earn points on over 40 non-Allergan Aesthetics treatments and brands. To learn more about Allē, visit Alle.com.
About AbbVie
AbbVie's mission is to discover and deliver innovative medicines that solve serious health issues today and address the medical challenges of tomorrow. We strive to have a remarkable impact on people's lives across several key therapeutic areas: immunology, oncology, neuroscience, eye care, virology, women's health and gastroenterology, in addition to products and services across its Allergan Aesthetics portfolio. For more information about AbbVie, please visit us at www.abbvie.com. Follow @abbvie on Twitter, Facebook, Instagram, YouTube and LinkedIn.
JUVÉDERM® Injectable Gel Fillers Important Information
APPROVED USES
JUVÉDERM® VOLUX™ XC injectable gel is for deep injection to improve moderate to severe loss of jawline definition in adults over the age of 21.
JUVÉDERM® VOLUMA® XC injectable gel is for deep injection in the cheek area to correct age-related volume loss and for augmentation of the chin region to improve the chin profile in adults over 21.
JUVÉDERM® VOLLURE® XC, JUVÉDERM® Ultra Plus XC, and JUVÉDERM® Ultra XC injectable gels are for injection into the facial tissue for the correction of moderate to severe facial wrinkles and folds, such as nasolabial folds. JUVÉDERM® VOLLURE® XC injectable gel is for adults over 21.
JUVÉDERM® Ultra XC injectable gel is also for injection into the lips and perioral area for lip augmentation in adults over 21.
JUVÉDERM® VOLBELLA® XC injectable gel is indicated for injection into the lips for lip augmentation and correction of perioral lines, and for injection into the undereye hollows to improve the appearance of undereye hollows in adults over the age of 21.
AbbVie gets FDA approval for JUVÉDERM to improve jawline definition
Aug. 03, 2022 9:31 AM ET
By: Ravikash, SA News Editor3 Comments
- The U.S. Food and Drug Administration (FDA) approved AbbVie's (NYSE:ABBV) JUVÉDERM Volux XC for improving jawline definition in adults over the age of 21 with moderate to severe loss of jawline definition.
- https://seekingalpha.com/news/3865740-abbvie-gets-fda-approval-for-juvderm-to-improve-jawline-definition
- https://seekingalpha.com/symbol/ABBV
- https://www.juvederm.com/
PADCEV® (enfortumab vedotin-ejfv) with KEYTRUDA® (pembrolizumab)
Seagen and Astellas Announce Positive Topline Results For PADCEV® (enfortumab vedotin-ejfv) with KEYTRUDA® (pembrolizumab) as First-Line Treatment for Advanced Urothelial Cancer
07/26/2022
– Companies plan to discuss results with regulatory authorities –
BOTHELL, Wash. & TOKYO--(BUSINESS WIRE)-- Seagen Inc. (Nasdaq:SGEN) and Astellas Pharma Inc. (TSE:4503, President and CEO: Kenji Yasukawa, Ph.D., “Astellas”) today announced positive topline results from the phase 1b/2 EV-103 clinical trial (also known as KEYNOTE-869) Cohort K evaluating PADCEV® (enfortumab vedotin-ejfv) in combination with Merck’s anti-PD-1 therapy KEYTRUDA® (pembrolizumab) as first-line treatment in patients with unresectable locally advanced or metastatic urothelial cancer (la/mUC) who are ineligible to receive cisplatin-based chemotherapy. Merck is known as MSD outside the United States and Canada.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20220726005377/en/
In patients treated with enfortumab vedotin and pembrolizumab, results demonstrated a 64.5% confirmed objective response rate (ORR) (95% CI: 52.7 to 75.1) per blinded independent central review (BICR), the primary endpoint of Cohort K. The median duration of response (DOR) per BICR was not reached. The most frequently reported treatment-emergent adverse events Grade 3 or greater that occurred in more than 5% of patients were rash maculo-papular, anemia, lipase increased, urinary tract infection, hyperglycemia, fatigue, neutropenia, hematuria, diarrhea, acute kidney injury, hyponatremia, chronic kidney disease, weight decreased, syncope, hypophosphatemia, pneumonitis, sepsis, and alanine aminotransferase increased. Overall, the results are generally consistent with previously reported efficacy and safety results of the EV-103 dose-escalation cohort and expansion Cohort A.1 Additional Cohort K results will be reported at an upcoming scientific congress.
EV-103 Cohort K is a randomized cohort investigating enfortumab vedotin alone or in combination with pembrolizumab as first-line treatment in patients with unresectable la/mUC who are ineligible to receive cisplatin-based chemotherapy. Secondary endpoints include ORR per investigator assessment; DOR, disease control rate and progression-free survival per BICR and investigator assessment; overall survival; and assessment of safety.
Please see Important Safety Information at the end of this press release, including BOXED WARNING for enfortumab vedotin.
“We are encouraged by the positive topline results of Cohort K for the combination of enfortumab vedotin and pembrolizumab in first-line locally advanced or metastatic urothelial cancer, and we look forward to sharing results at an upcoming medical meeting,” said Roger Dansey, M.D., interim CEO and Chief Medical Officer, Seagen.
“Approximately half of patients with advanced urothelial carcinoma are ineligible for cisplatin-based chemotherapy,” said Ahsan Arozullah, M.D., M.P.H., Senior Vice President and Head of Development Therapeutic Areas, Astellas. “We intend to discuss Cohort K results with regulatory authorities as we seek to develop a new first-line treatment combination for these patients.”
Seagen, Astellas and Merck are investigating enfortumab vedotin plus pembrolizumab as part of an extensive collaboration, which also includes three Phase 3 studies: the EV-302/KEYNOTE-A39 trial intended to confirm these results, as well as EV-304/KEYNOTE-B15 and EV-303/KEYNOTE-905 in muscle-invasive bladder cancer.
In February 2020, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation for enfortumab vedotin in combination with pembrolizumab for patients with unresectable la/mUC who are ineligible to receive cisplatin-based chemotherapy in the first-line setting. The designation is based on results from the dose-escalation cohort and expansion Cohort A of the phase 1b/2 trial, EV-103 (NCT03288545), evaluating patients with la/mUC who are ineligible to receive cisplatin-based chemotherapy treated in the first-line setting with enfortumab vedotin in combination with pembrolizumab.
About the EV-103 Trial (Cohort K)
The EV-103 trial (NCT03288545) is an ongoing, multi-cohort, open-label, multicenter phase 1b/2 trial of enfortumab vedotin alone or in combination with pembrolizumab and/or chemotherapy in first- or second-line settings in patients with locally advanced or metastatic urothelial cancer (la/mUC) and in patients with muscle-invasive bladder cancer.
Cohort K of the EV-103 trial is investigating enfortumab vedotin alone (n=73) or in combination with pembrolizumab (n=76) in adult patients with unresectable la/mUC who are ineligible for cisplatin-based chemotherapy and have received no prior treatment for la/mUC. The enfortumab vedotin monotherapy study arm is intended to characterize the activity of enfortumab vedotin alone in this patient population. Key outcome measures of EV-103 Cohort K are objective response rate (ORR) per blinded independent central review (BICR) using RECIST 1.1 and assessment of safety.
About PADCEV
PADCEV (enfortumab vedotin-ejfv) is a first-in-class antibody-drug conjugate (ADC) that is directed against Nectin-4, a protein located on the surface of cells and highly expressed in bladder cancer.5 Nonclinical data suggest the anticancer activity of PADCEV is due to its binding to Nectin-4 expressing cells followed by the internalization and release of the anti-tumor agent monomethyl auristatin E (MMAE) into the cell, which result in the cell not reproducing (cell cycle arrest) and in programmed cell death (apoptosis).6
Indication
PADCEV® is indicated for the treatment of adult patients with locally advanced or metastatic urothelial cancer (mUC) who:
- have previously received a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor and platinum-containing chemotherapy, or
- are ineligible for cisplatin-containing chemotherapy and have previously received one or more prior lines of therapy.5
- https://www.padcev.com/
About Seagen
Seagen Inc. is a global biotechnology company that discovers, develops and commercializes transformative cancer medicines to make a meaningful difference in people’s lives. Seagen is headquartered in the Seattle, Washington area, and has locations in California, Canada, Switzerland and the European Union. For more information on the company’s marketed products and robust pipeline, visit www.seagen.com and follow @SeagenGlobal on Twitter.
About Astellas
Astellas Pharma Inc. is a pharmaceutical company conducting business in more than 70 countries around the world. We are promoting the Focus Area Approach that is designed to identify opportunities for the continuous creation of new drugs to address diseases with high unmet medical needs by focusing on Biology and Modality. Furthermore, we are also looking beyond our foundational Rx focus to create Rx+® healthcare solutions that combine our expertise and knowledge with cutting-edge technology in different fields of external partners. Through these efforts, Astellas stands on the forefront of healthcare change to turn innovative science into value for patients. For more information, please visit our website at https://www.astellas.com/en.
About the Seagen, Astellas and Merck Collaboration
Seagen and Astellas entered a clinical collaboration agreement with Merck to evaluate the combination of Seagen’s and Astellas’ PADCEV® (enfortumab vedotin-ejfv) and Merck’s KEYTRUDA® (pembrolizumab) in patients with previously untreated metastatic urothelial cancer. KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220726005377/en/
Source: Seagen Inc.
Astellas/Seagen, Merck drug combo shows promise in bladder cancer subtype in trial
Jul. 26, 2022 5:14 AM ET
Seagen Inc. (SGEN), ALPMY, MRK, ALPMF
By: Ravikash, SA News Editor1 Comment
Astellas Pharma (OTCPK:ALPMY) (OTCPK:ALPMF) and Seagen (NASDAQ:SGEN) said Cohort K of a phase 1b/2 trial of their drug Padcev in combination with Merck's (NYSE:MRK) Keytruda showed response in certain patients with a type of bladder cancer.
https://seekingalpha.com/symbol/ALPMY
https://seekingalpha.com/symbol/ALPMF
https://seekingalpha.com/symbol/SGEN
https://seekingalpha.com/symbol/MRK
Upadacitinib (RINVOQ®)
July 27, 2022
AbbVie Submits Regulatory Applications to FDA and EMA for Upadacitinib (RINVOQ®) in Crohn's Disease
- Submissions are supported by three Phase 3 clinical trials demonstrating upadacitinib achieved the co-primary endpoints of clinical remission and endoscopic response as induction and maintenance treatment1-4
- Safety results were generally consistent with the known safety profile of upadacitinib, with no new safety risks observed1-8
- Crohn's disease is a chronic, systemic disease that manifests as inflammation within the gastrointestinal tract, causing persistent diarrhea and abdominal pain9,10
NORTH CHICAGO, Ill., July 27, 2022 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced that it has submitted applications for a new indication to the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) for upadacitinib (RINVOQ®, 45 mg [induction dose] and 15 mg and 30 mg [maintenance dose]) for the treatment of adult patients with moderately to severely active Crohn's disease.4,11
"Crohn's disease can be debilitating and have a significant impact on a person's daily life," said Neil Gallagher, M.D., Ph.D., vice president, development, chief medical officer, AbbVie. "Those patients who are still suffering fuel our continued commitment to innovation in care for patients with IBD, and we look forward to potentially introducing a new treatment option for this disruptive condition."
The applications to the FDA and EMA are supported by data from three Phase 3 clinical trials, including two induction studies (U-EXCEED & U-EXCEL) and one maintenance study (U-ENDURE).1-4 Across all three studies, significantly more patients treated with upadacitinib achieved the co-primary endpoints of clinical remission and endoscopic response, with clinical remission measured by the Crohn's Disease Activity Index (CDAI) or by the patient-reported symptoms of stool frequency/abdominal pain (SF/AP).1-4 Additionally, more patients receiving upadacitinib 45 mg once daily at week 12 in the induction studies or 15 mg and 30 mg once daily at 52 weeks in the maintenance study achieved the secondary endpoint of corticosteroid-free clinical remission per CDAI and per SF/AP compared to placebo among patients taking corticosteroids at baseline.1-4
The safety results of upadacitinib in U-EXCEED, U-EXCEL and U-ENDURE were generally consistent with the known safety profile of upadacitinib, with no new safety risks observed.
About the U-EXCEED and U-EXCEL Induction and U-ENDURE Maintenance Studies1,2,3
The three Phase 3 studies are multicenter, randomized, double-blind, placebo-controlled studies to evaluate the efficacy and safety of upadacitinib 45 mg once daily as induction therapy, and upadacitinib 15 mg and 30 mg once daily as maintenance therapy in adults with moderately to severely active Crohn's disease. Topline results from U-EXCEED and U-EXCEL induction studies were announced in December 2021 and February 2022, respectively, and topline results from the U-ENDURE maintenance study were announced in May 2022. These studies include assessments of efficacy, safety and tolerability of upadacitinib. More information on these trials can be found at www.clinicaltrials.gov (NCT03345836, NCT03345849, NCT03345823).
About upadacitinib (RINVOQ®)
Discovered and developed by AbbVie scientists, RINVOQ is a JAK inhibitor that is being studied in several immune-mediated inflammatory diseases.1–8 Based on enzymatic and cellular assays, RINVOQ demonstrated greater inhibitory potency for JAK-1 vs. JAK-2, JAK-3 and TYK-2. The relevance of inhibition of specific JAK enzymes to therapeutic effectiveness and safety is not currently known.12
Phase 3 trials of RINVOQ in rheumatoid arthritis, atopic dermatitis, psoriatic arthritis, axial spondyloarthritis, ulcerative colitis, giant cell arteritis and Takayasu arteritis are ongoing.4–8,11,13-18 The use of upadacitinib in Crohn's disease is not approved and its safety and efficacy have not been evaluated by regulatory authorities.
RINVOQ® (upadacitinib) U.S. Use and Important Safety Information12
RINVOQ is a prescription medicine used to treat:
- Adults with moderate to severe rheumatoid arthritis when 1 or more medicines called tumor necrosis factor (TNF) blockers have been used, and did not work well or could not be tolerated.
- Adults with active psoriatic arthritis when 1 or more medicines called TNF blockers have been used, and did not work well or could not be tolerated.
- Adults with moderate to severe ulcerative colitis when 1 or more medicines called TNF blockers have been used, and did not work well or could not be tolerated.
- Adults with active ankylosing spondylitis when 1 or more medicines called TNF blockers have been used, and did not work well or could not be tolerated.
It is not known if RINVOQ is safe and effective in children with juvenile idiopathic arthritis, psoriatic arthritis, ulcerative colitis, or ankylosing spondylitis.
- Adults and children 12 years of age and older with moderate to severe eczema (atopic dermatitis) that did not respond to previous treatment and their eczema is not well controlled with other pills or injections, including biologic medicines, or the use of other pills or injections is not recommended.
RINVOQ is safe and effective in children 12 years of age and older weighing at least 88 pounds (40 kg) with atopic dermatitis.
It is not known if RINVOQ is safe and effective in children under 12 years of age with atopic dermatitis.
About AbbVie
AbbVie's mission is to discover and deliver innovative medicines that solve serious health issues today and address the medical challenges of tomorrow. We strive to have a remarkable impact on people's lives across several key therapeutic areas: immunology, oncology, neuroscience, eye care, virology, women's health and gastroenterology, in addition to products and services across its Allergan Aesthetics portfolio. For more information about AbbVie, please visit us at www.abbvie.com. Follow @abbvie on Twitter, Facebook, LinkedIn or Instagram.
SOURCE AbbVie
AbbVie asks FDA, EMA to approve Rinvoq for Crohn's disease
Jul. 28, 2022 11:58 AM ET
By: Jonathan Block, SA News Editor
- AbbVie (NYSE:ABBV) has submitted regulatory applications to the U.S. FDA and the European Medicines Agency seeking approval of Rinvoq (upadacitinib) for Crohn's disease.
- https://seekingalpha.com/news/3862507-abbvie-asks-fda-ema-to-approve-rinvoq-for-crohns-disease
- https://seekingalpha.com/symbol/ABBV
ABBV Dividend History
https://www.nasdaq.com/market-activity/stocks/abbv/dividend-history
https://seekingalpha.com/symbol/ABBV
Jul 26, 2022
NORTH CHICAGO, Ill., July 26, 2022 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced the European Commission (EC) approved RINVOQ® (upadacitinib 45 mg [induction dose] and 15 mg and 30 mg [maintenance doses]) for the treatment of adult patients with moderately to severely active ulcerative colitis who have had an inadequate response, lost response or were intolerant to either conventional therapy or a biologic agent.*
Jul 29, 2022
NORTH CHICAGO, Ill., July 29, 2022 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced that the European Commission (EC) has approved RINVOQ® (upadacitinib 15 mg, once daily), an oral therapy, for the treatment of active non-radiographic axial spondyloarthritis (nr-axSpA) in adult patients with objective signs of inflammation, as indicated by elevated C-reactive protein (CRP) and/or magnetic resonance imaging (MRI), who have responded inadequately to nonsteroidal anti-inflammatory drugs (NSAIDs).*1
NULIBRY® (fosdenopterin)
BridgeBio Pharma and Sentynl Therapeutics Receive Positive CHMP Opinion for NULIBRY® (fosdenopterin) for the Treatment of MoCD Type A
– CHMP recommendation for approval of NULIBRY in the European Union (EU) for the treatment of patients with molybdenum cofactor deficiency (MoCD) Type A is based on the efficacy and safety data collected to date compared to data from a natural history study
– Under an accelerated assessment pathway, a decision by the European Commission (EC), which authorizes marketing approval in the European Union (EU), is expected on the NULIBRY application later this year
– If approved by the EC, NULIBRY would be the first and only approved therapy in the EU to treat patients with MoCD type A, an ultra-rare, life-threatening genetic disorder that often progresses rapidly in infants with a median overall survival age of about four years
– NULIBRY was BridgeBio’s first FDA-approved therapeutic; Sentynl acquired global rights to NULIBRY in March 2022
Palo Alto and Solana Beach, CA – July 25, 2022 — BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio), a commercial-stage biopharmaceutical company that focuses on genetic diseases and cancers, and Sentynl Therapeutics, Inc. (Sentynl), a U.S.-based biopharmaceutical company focused on bringing innovative therapies to patients living with rare diseases owned by Zydus Lifesciences Ltd. (formerly known as Cadila Healthcare Ltd.), today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended that the European Commission authorize marketing under exceptional circumstances for NULIBRY® (fosdenopterin) for Injection as the first therapy for the treatment of patients with molybdenum cofactor deficiency (MoCD) Type A. MoCD Type A is an ultra-rare and progressive condition, known to impact less than 150 patients globally with a median survival of four years.
NULIBRY is a first-in-class cPMP substrate replacement therapy that was approved by the U.S. Food and Drug Administration (FDA) in 2021 to reduce the risk of mortality in patients with MoCD Type A. If approved by the European Commission, NULIBRY would be the first and only approved therapy in the EU for MoCD Type A.
In March 2022, Sentynl acquired the global rights to NULIBRY and is responsible for the ongoing development and commercialization of NULIBRY in the United States and developing, manufacturing, and commercializing fosdenopterin globally. Sentynl and BridgeBio share development responsibilities through the approval of the marketing authorization application under accelerated assessment with the EMA and through approval of NULIBRY’s regulatory submission with the Israeli Ministry of Health.
“Our work on NULIBRY and MoCD Type A epitomizes BridgeBio’s belief that no disease is too rare to address. With this positive CHMP opinion, we are closer to delivering a treatment option to all children across the globe who suffer with MoCD Type A,” said BridgeBio founder and CEO Neil Kumar, Ph.D.
The positive CHMP opinion is supported by data from three clinical trials that demonstrated efficacy of NULIBRY for the treatment of patients with MoCD Type A compared to data from a natural history study. These studies showed that NULIBRY reduced the risk of death by 86% and increased the probability of survival to 86% at three years compared to 52% in the untreated, genotype-matched, historical control group in the natural history study.
“We are thrilled by the CHMP’s recommendation in favor of NULIBRY and hope that patients living with MoCD Type A in Europe and around the world can access this therapy,” said Matt Heck, CEO of Sentynl. “The CHMP positive opinion marks important progress not only for the program but also for the MoCD Type A patients outside of the U.S. who are seeking ways to treat their life-threatening and progressive disease.”
Based on the CHMP recommendation, a decision by the EC, which authorizes marketing applications in the EU, is expected on the NULIBRY application later this year. The recommendation for marketing authorization under exceptional circumstances is granted to medicines where the applicant is unable to provide comprehensive data under normal conditions of use because the disease being treated is so rare.
In April 2022, BridgeBio received New Drug Application (NDA) Approval in Principle from the Israeli Ministry of Health and the application is currently undergoing the final review processes.
About NULIBRY® (Fosdenopterin) for Injection
NULIBRY®(Fosdenopterin) for Injection is a substrate replacement therapy that provides an exogenous source of cPMP, which is converted to molybdopterin. Molybdopterin is then converted to molybdenum cofactor, which is needed for the activation of molybdenum-dependent enzymes, including sulfite oxidase, an enzyme that reduces levels of neurotoxic sulfites. It is the first and only FDA-approved therapy indicated to reduce the risk of mortality in patients with MoCD Type A, and clinical trials have demonstrated that patients treated with NULIBRY or rcPMP had an improvement in overall survival compared to the untreated, genotype-matched, historical control group.
About Sentynl Therapeutics
Sentynl Therapeutics is a U.S.-based biopharmaceutical company focused on bringing innovative therapies to patients living with rare diseases. The company was acquired by the Zydus Group in 2017. Sentynl’s experienced management team has previously built multiple successful pharmaceutical companies. With a focus on commercialization, Sentynl looks to source effective and highly differentiated products across a broad spectrum of therapeutic areas to address unmet needs. Sentynl is committed to the highest ethical standards and compliance with all applicable laws, regulations, and industry guidelines. For more information, visit www.sentynl.com.
About Zydus
The Zydus Group, with an overarching purpose of empowering people with freedom to live healthier and more fulfilled lives, is an innovative, global pharmaceutical company that discovers, develops, manufactures, and markets a broad range of healthcare therapies. The group employs over 23000 people worldwide and is driven by its mission to unlock new possibilities in life-sciences through quality healthcare solutions that impact lives. The group aspires to become a global life-sciences company transforming lives through pathbreaking discoveries. For more information, visit https://www.zyduslife.com/zyduslife/.
About BridgeBio Pharma, Inc.
BridgeBio Pharma, Inc. (BridgeBio) is a commercial-stage biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBio’s pipeline of development programs ranges from early science to advanced clinical trials. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn and Twitter.
BridgeBio, Sentynl's Nulibry get EMA panel nod for approval in EU
Jul. 25, 2022 8:37 AM ET
By: Ravikash, SA News Editor
- BridgeBio Pharma (NASDAQ:BBIO) and Sentynl Therapeutics' said a committee of the European Medicines Agency (EMA) recommended approval under exceptional circumstances of their therapy Nulibry to treat patients with molybdenum cofactor deficiency (MoCD) Type A.
- https://seekingalpha.com/pr/18876756-bridgebio-pharma-and-sentynl-therapeutics-receive-positive-chmp-opinion-for-nulibry?source=content_type%3Areact%7Csection%3Amain_content%7Cbutton%3Abody_link%7Cfirst_level_url%3Anews
- https://seekingalpha.com/symbol/BBIO
ENHERTU® (fam-trastuzumab deruxtecan-nxki)
ENHERTU® Granted Priority Review in the U.S. for Patients with HER2 Low Metastatic Breast Cancer • Based on DESTINY-Breast04 results which showed Daiichi Sankyo and AstraZeneca’s ENHERTU is the first HER2 directed therapy to demonstrate a survival benefit in this population • Application being evaluated under FDA Real-Time Oncology Review and Project Orbis Tokyo and Basking Ridge, NJ – (July 25, 2022) – Daiichi Sankyo (TSE: 4568) and AstraZeneca (LSE/STO/Nasdaq: AZN) have received notification of acceptance by the U.S. Food and Drug Administration (FDA) of the supplemental Biologics License Application (sBLA) of ENHERTU® (famtrastuzumab deruxtecan-nxki) for the treatment of adult patients with unresectable or metastatic HER2 low (immunohistochemistry (IHC) 1+ or IHC 2+/in-situ hybridization (ISH)-negative) breast cancer who have received a prior therapy in the metastatic setting. The application has been granted Priority Review. ENHERTU is a specifically engineered HER2 directed antibody drug conjugate (ADC) being jointly developed and commercialized by Daiichi Sankyo and AstraZeneca. The FDA grants Priority Review to applications for medicines that, if approved, would offer significant improvements over available options by demonstrating safety or efficacy improvements, preventing serious conditions or enhancing patient compliance. The Prescription Drug User Fee Act date (PDUFA), the FDA action date for their regulatory decision, is during the fourth quarter of the 2022 calendar year. The Priority Review follows receipt of Breakthrough Therapy Designation, granted by the FDA in April 2022 for ENHERTU in metastatic HER2 low breast cancer. The sBLA is being reviewed under the Real-Time Oncology Review (RTOR) program and Project Orbis, two initiatives of the FDA which are designed to bring safe and effective cancer treatments to patients as early as possible. RTOR allows the FDA to review components of an application before submission of the complete application. Project Orbis provides a framework for concurrent submission and review of oncology medicines among participating international partners. “The results seen in the DESTINY-Breast04 trial represent a significant advance and reinforce the potential for ENHERTU to become a new standard of care for patients with previously treated HER2 low metastatic breast cancer,” said Ken Takeshita, MD, Global Head, R&D, Daiichi Sankyo. “The prioritization of this 2 application by the FDA and inclusion in both the Real-Time Oncology Review and Project Orbis initiatives support the importance of these data, and we look forward to working with the FDA to potentially bring ENHERTU to patients with HER2 low metastatic breast cancer as quickly as possible.” “The data from DESTINY-Breast04 represent the first time a HER2 targeted therapy has shown a survival benefit in patients with HER2 low metastatic breast cancer,” said Susan Galbraith, MBBChir, PhD, Executive Vice President, Oncology R&D, AstraZeneca. “For more than two decades, only patients with HER2 positive breast cancer have been able to benefit from HER2 targeted therapies. If approved, ENHERTU will redefine how we classify and treat metastatic breast cancer, enabling patients whose tumors have lower levels of HER2 expression the opportunity to benefit from a HER2 directed therapy.” The sBLA is based on data from the DESTINY-Breast04 phase 3 trial recently presented at the presidential plenary session of the American Society of Clinical Oncology (#ASCO22) Annual Meeting and simultaneously published in The New England Journal of Medicine. In DESTINY-Breast04, ENHERTU demonstrated superior and clinically meaningful efficacy in progression-free survival (PFS) and overall survival (OS) in previously treated patients with HER2 low unresectable and/or metastatic breast cancer with hormone receptor (HR) positive or HR negative disease versus standard of care physician’s choice of chemotherapy. The safety profile of ENHERTU was consistent with previous clinical trials with no new safety concerns identified. Interstitial lung disease (ILD) or pneumonitis rates were consistent with those observed in other late-line HER2 positive breast cancer trials of ENHERTU, as determined by an independent adjudication committee. About DESTINY-Breast04 DESTINY-Breast04 is a global, randomized, open-label, pivotal phase 3 trial evaluating the efficacy and safety of ENHERTU (5.4 mg/kg) versus physician’s choice of chemotherapy (capecitabine, eribulin, gemcitabine, paclitaxel or nab-paclitaxel) in patients with HR positive or HR negative, HER2 low unresectable and/or metastatic breast cancer previously treated with one or two prior lines of chemotherapy. Patients were randomized 2:1 to receive either ENHERTU or chemotherapy. The primary endpoint of DESTINY-Breast04 is PFS in patients with HR positive disease based on blinded independent central review (BICR). Key secondary endpoints include PFS based on BICR in all randomized patients (HR positive and HR negative disease), OS in patients with HR positive disease and OS in all randomized patients (HR positive and HR negative disease). Other secondary endpoints include PFS based on investigator assessment, objective response rate based on BICR and on investigator assessment, duration of response based on BICR and safety. DESTINY-Breast04 enrolled 557 patients at multiple sites in Asia, Europe and North America. For more information about the trial, visit ClinicalTrials.gov.
About ENHERTU ENHERTU® (trastuzumab deruxtecan; fam-trastuzumab deruxtecan-nxki in the U.S. only) is a HER2 directed ADC. Designed using Daiichi Sankyo’s proprietary DXd ADC technology, ENHERTU is the lead ADC in the oncology portfolio of Daiichi Sankyo and the most advanced program in AstraZeneca’s ADC scientific platform. ENHERTU consists of a HER2 monoclonal antibody attached to a topoisomerase I inhibitor payload, an exatecan derivative, via a stable tetrapeptide-based cleavable linker. ENHERTU (5.4 mg/kg) is approved in more than 30 countries for the treatment of adult patients with unresectable or metastatic HER2 positive breast cancer who have received a prior anti-HER2-based regimen either in the metastatic setting, or in the neoadjuvant or adjuvant setting and have developed disease recurrence during or within six months of completing therapy, based on results from the DESTINY-Breast03 4 trial. ENHERTU also is approved in several countries for the treatment of adult patients with unresectable or metastatic HER2 positive breast cancer who have received two or more prior anti-HER2-based regimens based on the results from the DESTINY-Breast01 trial. ENHERTU (6.4 mg/kg) is approved in several countries for the treatment of adult patients with locally advanced or metastatic HER2 positive gastric or gastroesophageal junction (GEJ) adenocarcinoma who have received a prior trastuzumab-based regimen based on the results from the DESTINY-Gastric01 trial. ENHERTU is approved in the U.S. with Boxed WARNINGS for Interstitial Lung Disease and Embryo-Fetal Toxicity. For more information, please see the accompanying full Prescribing Information, including Boxed WARNINGS, and Medication Guide. About the ENHERTU Clinical Development Program A comprehensive global development program is underway evaluating the efficacy and safety of ENHERTU monotherapy across multiple HER2 targetable cancers including breast, gastric, lung and colorectal cancers. Trials in combination with other anticancer treatments, such as immunotherapy, also are underway. Regulatory applications for ENHERTU are currently under review in China, Japan and several other countries for the treatment of adult patients with HER2 positive unresectable or metastatic breast cancer who have received a prior anti-HER2-based regimen based on the results from the DESTINY-Breast03 trial. ENHERTU is under review in Europe and Japan for the treatment of adult patients with unresectable or metastatic HER2 low (IHC 1+ or IHC 2+/ISH-negative) breast cancer who have received a prior systemic therapy in the metastatic setting or developed disease recurrence during or within six months of completing adjuvant chemotherapy, based on the results from the DESTINY-Breast04 trial. Patients with HR positive breast cancer must additionally have received or be ineligible for endocrine therapy. ENHERTU also is currently under review in the U.S. for the treatment of adult patients with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have a HER2 (ERBB2) mutation and who have received a prior systemic therapy based on the results from the DESTINY-Lung01 trial, and in Europe for the treatment of adult patients with locally advanced or metastatic HER2 positive gastric or GEJ adenocarcinoma who have received a prior anti-HER2-based regimen based on the DESTINY-Gastric01 and DESTINY-Gastric02 trials. 5 About the Daiichi Sankyo and AstraZeneca Collaboration Daiichi Sankyo Company, Limited (referred to as Daiichi Sankyo) and AstraZeneca entered into a global collaboration to jointly develop and commercialize ENHERTU in March 2019 and datopotamab deruxtecan (Dato-DXd) in July 2020, except in Japan where Daiichi Sankyo maintains exclusive rights for each ADC. Daiichi Sankyo is responsible for the manufacturing and supply of ENHERTU and datopotamab deruxtecan. Important Safety Information for ENHERTU Indications ENHERTU is a HER2-directed antibody and topoisomerase inhibitor conjugate indicated for the treatment of adult patients with: • Unresectable or metastatic HER2-positive breast cancer who have received a prior anti-HER2-based regimen either: – In the metastatic setting, or – In the neoadjuvant or adjuvant setting and have developed disease recurrence during or within six months of completing therapy • Locally advanced or metastatic HER2-positive gastric or gastroesophageal junction adenocarcinoma who have received a prior trastuzumab-based regimen
Please see accompanying full Prescribing Information, including Boxed WARNINGS, and Medication Guide. About Daiichi Sankyo Daiichi Sankyo is dedicated to creating new modalities and innovative medicines by leveraging our world-class science and technology for our purpose “to contribute to the enrichment of quality of life around the world.” In addition to our current portfolio of medicines for cancer and cardiovascular disease, Daiichi Sankyo is primarily focused on developing novel therapies for people with cancer as well as other diseases with high unmet medical needs. With more than 100 years of scientific expertise and a presence in more than 20 countries, Daiichi Sankyo and its 16,000 employees around the world draw upon a rich legacy of innovation to realize our 2030 Vision to become an “Innovative Global Healthcare Company Contributing to the Sustainable Development of Society.” For more information, please visit: www.daiichisankyo.com.
AstraZeneca, Daiichi Sankyo's Enhertu gets FDA priority review for breast cancer subtype
Jul. 25, 2022 5:10 AM ET
AstraZeneca PLC (AZN), DSKYF, DSNKY
By: Ravikash, SA News Editor
- The U.S. Food and Drug Administration (FDA) granted priority review to AstraZeneca (NASDAQ:AZN) and Daiichi Sankyo's (OTCPK:DSKYF) (OTCPK:DSNKY) Enhertu to treat adult patients with unresectable or metastatic HER2-low (immunohistochemistry (IHC) 1+ or IHC 2+/in-situ hybridization (ISH)-negative) breast cancer who have received a prior therapy in the metastatic setting.
- https://seekingalpha.com/news/3859868-astrazeneca-daiichi-sankyos-enhertu-gets-fda-priority-review-for-breast-cancer-subtype
- https://seekingalpha.com/symbol/AZN
- https://seekingalpha.com/symbol/DSKYF
- https://seekingalpha.com/symbol/DSNKY
- https://www.astrazeneca.com/
- https://www.enhertu.com/en
TECVAYLI® (teclistamab)
Janssen Receives Positive CHMP Opinion for Novel Bispecific Antibody TECVAYLI® (teclistamab) for the Treatment of Patients with Relapsed and Refractory Multiple Myeloma (RRMM)
Jul 22, 2022Belgium
Teclistamab is the first T-cell redirecting bispecific antibody to receive a positive CHMP opinion for adults with RRMM and highlights Janssen’s commitment to innovation in multiple myeloma
The opinion is based on the MajesTEC-1 study where teclistamab induced durable responses that deepened over time in patients with heavily pretreated RRMM[1]
BEERSE, Belgium, 22 July 2022 – The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended conditional marketing authorisation (CMA) for TECVAYLI® (teclistamab) as monotherapy for adult patients with relapsed and refractory multiple myeloma (RRMM), who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody and have demonstrated disease progression on the last therapy. Teclistamab is an off-the-shelf, T-cell redirecting bispecific antibody. It targets both B-cell maturation antigen (BCMA), a marker found on multiple myeloma cells, and CD3, on T-cells.1
CMA is the approval of a medicine that addresses unmet medical needs of patients based on less comprehensive data than normally required, where the benefit of immediate availability of the medicine outweighs the risk, and the applicant is able to provide comprehensive clinical data in the future.[2] While newer treatment options have nearly doubled survival outcomes for patients living with multiple myeloma over the past few decades, it remains an incurable disease.[3] Nearly all patients will relapse and require subsequent therapy.[4] Generally, efficacy outcomes decrease with each line of therapy, and patients face poor prognoses.[5]
In December 2021, the EMA granted accelerated assessment for teclistamab. Accelerated assessment reduces the timeframe for the CHMP to review a marketing authorisation application (MAA) and is granted when a medicinal product is of major interest for public health and therapeutic innovation.[6]
“We endeavour to deliver our robust multiple myeloma pipeline of diverse mechanisms and targets with the aim of improving outcomes for patients,” said Peter Lebowitz, M.D., Ph.D., Global Therapeutic Area Head, Oncology, Janssen Research & Development, LLC. “Teclistamab is testament to this approach. If adopted by the European Commission, the approval could be the first worldwide for teclistamab, as the first T-cell redirecting bispecific antibody for the treatment of patients with relapsed and refractory multiple myeloma.”
This CHMP recommendation is based on positive results from the multicohort, open-label, Phase 1/2 MajesTEC-1 study (NCT03145181 and NCT04557098), evaluating the safety and efficacy of teclistamab in adults with RRMM.[7],[8]
The latest findings from the study were recently presented at the American Society of Clinical Oncology (ASCO) 2022 Annual Meeting and published in The New England Journal of Medicine.1 Teclistamab resulted in deep and durable responses in patients with triple-class exposed multiple myeloma (n=165). With a median follow-up of approximately 14 months (14.1), the overall response rate was 63 percent (95 percent confidence interval [CI]: 55.2–70.4), with 39.4 percent having a complete response (CR) or better.1 Almost half (46 percent) of patients who achieved a CR or better were minimal residual disease (MRD) negative (10-5).1
Adverse events (AEs) were consistent with this patient population and toxicities consistent with T-cell redirection were mostly Grade 1/2.1 The most common AEs were cytokine release syndrome (72.1 percent; 0.6 percent Grade 3, no Grade 4) and neutropenia (70.9 percent; 64.2 percent Grade 3 or 4).1 Infections were frequent (76.4 percent; 44.8 percent Grade 3 or 4).1 The overall incidence of neurotoxic events was low (24 patients; 14.5 percent) and five patients (three percent) had immune effector cell-associated neurotoxicity syndrome.1 There were five treatment-related deaths, and dose reductions and discontinuations due to AEs were infrequent.1
“Our ambition to eliminate multiple myeloma is stronger today than ever before. We aim to reach this goal by investing in cutting-edge innovations that address individual patient needs and offer healthcare professionals options they have not had before,” said Edmond Chan MBChB M.D. (Res), Senior Director EMEA Therapeutic Area Lead Haematology, Janssen-Cilag Limited. “Today’s recommendation from the CHMP marks exciting progress in this journey, and we look forward to working with health authorities to make teclistamab available to patients across the region, as soon as possible.”
#ENDS#
About Teclistamab
Teclistamab is an investigational, fully humanised, T-cell redirecting, IgG4 bispecific antibody targeting both BCMA and CD3, on T-cells.1 BCMA is expressed at high levels on multiple myeloma cells.[9],[10],[11] Teclistamab redirects CD3-positive T-cells to BCMA-expressing myeloma cells to induce killing of tumour cells.[12]
Teclistamab is currently being evaluated in several monotherapy and combination studies.[13],[14],[15],[16],[17] In January 2021 and June 2021, teclistamab received a PRIority MEdicines (PRIME) designation by the EMA and Breakthrough Therapy Designation (BTD) by the U.S. FDA, respectively. PRIME offers enhanced interaction and early dialogue to optimise drug development plans and speed up evaluation of cutting-edge, scientific advances that target a high unmet medical need.[18] The FDA grants BTD to expedite the development and regulatory review of an investigational medicine that is intended to treat a serious or life-threatening condition based on preliminary clinical evidence that demonstrates the drug may have substantial improvement in at least one clinically significant endpoint over available therapy.[19]
In December 2021, Janssen Research & Development, LLC submitted a Biologics License Application (BLA) to the FDA seeking approval of teclistamab for the treatment of patients with RRMM; the MAA was submitted to the EMA for teclistamab approval in January 2022.
About the Janssen Pharmaceutical Companies of Johnson & Johnson
At Janssen, we’re creating a future where disease is a thing of the past. We’re the Pharmaceutical Companies of Johnson & Johnson, working tirelessly to make that future a reality for patients everywhere by fighting sickness with science, improving access with ingenuity, and healing hopelessness with heart. We focus on areas of medicine where we can make the biggest difference: Cardiovascular, Metabolism & Retina; Immunology; Infectious Diseases & Vaccines; Neuroscience; Oncology; and Pulmonary Hypertension.
Learn more at www.janssen.com/emea. Follow us at www.twitter.com/janssenEMEA for our latest news. Janssen Pharmaceutica NV, Janssen-Cilag Limited and Janssen Research & Development, LLC are part of the Janssen Pharmaceutical Companies of Johnson & Johnson.
J&J gets EMA panel nod for conditional approval of Tecvayli for blood cancer subtype
Jul. 22, 2022 8:26 AM ET
By: Ravikash, SA News Editor
- A committee of the European Medicines Agency (EMA) recommended granting conditional marketing authorization to Johnson & Johnson's (NYSE:JNJ) Tecvayli (teclistamab) to treat certain patients with multiple myeloma (MM).
- https://seekingalpha.com/news/3859583-jj-gets-ema-panel-nod-for-conditional-approval-of-tecvayli-for-blood-cancer-subtype
- https://seekingalpha.com/symbol/JNJ
TECARTUS® (brexucabtagene autoleucel)
Kite’s CAR T-cell Therapy Tecartus® Receives Positive CHMP Opinion in Relapsed or Refractory Acute Lymphoblastic Leukemia (r/r ALL)
– Tecartus ® (Brexucabtagene Autoleucel) First and Only CAR T in Europe to Receive Positive CHMP Opinion to Treat Adults 26+ with r/r ALL –
– If Approved, it will Address a Significant Unmet Need for a Patient Population with Limited Treatment Options –
SANTA MONICA, Calif.--(BUSINESS WIRE)-- Kite, a Gilead Company (Nasdaq: GILD), today announces that the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion for Tecartus® (brexucabtagene autoleucel) for the treatment of adult patients 26 years of age and above with relapsed or refractory (r/r) B-cell precursor acute lymphoblastic leukemia (ALL). If approved, Tecartus will be the first and only Chimeric Antigen Receptor (CAR) T-cell therapy for this population of patients who have limited treatment options. Half of adults with ALL will relapse, and median overall survival (OS) for this group is only approximately eight months with current standard-of-care treatments.
“Kite’s goal is clear: to bring the hope of survival to more patients with cancer around the world through cell therapy,” said Christi Shaw, CEO, Kite. “Today’s CHMP positive opinion in adult ALL brings us a step closer to delivering on the promise that cell therapies have to transform the way cancer is treated.”
Following this positive opinion, the European Commission will now review the CHMP opinion; the final decision on the Marketing Authorization is expected in the coming months.
“Adults with relapsed or refractory ALL often undergo multiple treatments including chemotherapy, targeted therapy and stem cell transplant, creating a significant burden on a patient’s quality of life,” said Max S. Topp, MD, professor and head of Hematology, University Hospital of Wuerzburg, Germany. “If approved, patients in Europe will have a meaningful advancement in treatment. Tecartus has demonstrated durable responses, suggesting the potential for long-term remission and a new approach to care.”
Results from the ZUMA-3 international multicenter, single-arm, open-label, registrational Phase 1/2 study of adult patients (≥18 years old) with relapsed or refractory ALL, demonstrated that 71% of the evaluable patients (n=55) achieved complete remission (CR) or CR with incomplete hematological recovery (CRi) with a median follow-up of 26.8 months. In an extended data set of all patients dosed with the pivotal dose (n=78) the median overall survival for all patients was more than two years (25.4 months) and almost four years (47 months) for responders (patients who achieved CR or CRi). Among efficacy-evaluable patients, median duration of remission (DOR) was 18.6 months. Among the patients treated with Tecartus at the target dose (n=100), Grade 3 or higher cytokine release syndrome (CRS) and neurologic events occurred in 25% and 32% of patients, respectively, and were generally well-managed.
About ZUMA-3
ZUMA-3 is an ongoing international multicenter (US, Canada, EU), single arm, open label, registrational Phase 1/2 study of Tecartus in adult patients (≥18 years old) with ALL whose disease is refractory to or has relapsed following standard systemic therapy or hematopoietic stem cell transplantation. The primary endpoint is the rate of overall complete remission or complete remission with incomplete hematological recovery by central assessment. Duration of remission and relapse-free survival, overall survival, minimal residual disease (MRD) negativity rate, and allo-SCT rate were assessed as secondary endpoints.
About Tecartus
Please see full FDA Prescribing Information, including BOXED WARNING and Medication Guide.
Tecartus is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of:
- Adult patients with relapsed or refractory mantle cell lymphoma (MCL).
This indication is approved under accelerated approval based on overall response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
- Adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL).
- https://www.tecartus.com/
About Kite
Kite, a Gilead Company, is a global biopharmaceutical company based in Santa Monica, California, with manufacturing operations in North America and Europe. Kite’s singular focus is cell therapy to treat and potentially cure cancer. As the cell therapy leader, Kite has more approved CAR T indications to help more patients than any other company. For more information on Kite, please visit www.kitepharma.com. Follow Kite on social media on Twitter (@KitePharma) and LinkedIn.
About Gilead Sciences
Gilead Sciences, Inc. is a biopharmaceutical company that has pursued and achieved breakthroughs in medicine for more than three decades, with the goal of creating a healthier world for all people. The company is committed to advancing innovative medicines to prevent and treat life-threatening diseases, including HIV, viral hepatitis and cancer. Gilead operates in more than 35 countries worldwide, with headquarters in Foster City, California.
U.S. Prescribing Information for Tecartus including BOXED WARNING, is available at www.kitepharma.com and www.gilead.com .
Kite, the Kite logo, Tecartus and GILEAD are trademarks of Gilead Sciences, Inc. or its related companies .
View source version on businesswire.com: https://www.businesswire.com/news/home/20220722005258/en/
Source: Gilead Sciences, Inc.
July 22, 2022
Gilead's Tecartus gets EMA panel backing for expanded use in blood cancer subtype
Jul. 22, 2022 8:05 AM ET
By: Ravikash, SA News Editor3 Comments
- A committee of the European Medicines Agency (EMA) recommended the expanded approval of Gilead Sciences' (NASDAQ:GILD) CAR T cell therapy Tecartus to treat adult patients 26 years of age and above with relapsed or refractory B-cell precursor acute lymphoblastic leukaemia (ALL).
- https://seekingalpha.com/news/3859570-gileads-tecartus-gets-ema-panel-backing-for-expanded-use-in-blood-cancer-subtype
- https://seekingalpha.com/symbol/GILD
- https://www.tecartus.com/
biotecMAX™ 2022 Insight
Opdualag (nivolumab and relatlimab)
Bristol Myers Squibb Receives Positive CHMP Opinion Recommending Approval for LAG-3-Blocking Antibody Combination Opdualag (nivolumab and relatlimab) for Treatment of Patients with Unresectable or Metastatic Melanoma
07/22/2022CATEGORY:
Approval recommended for first-line treatment of advanced melanoma patients with tumor cell PD-L1 expression < 1%
Recommendation based on results from the Phase 2/3 RELATIVITY-047 trial, in which the fixed-dose combination of the PD-1 inhibitor nivolumab and novel LAG-3-blocking antibody relatlimab more than doubled median progression-free survival compared to nivolumab monotherapy
If approved, it would be the first LAG-3 blocking antibody combination in Europe
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended approval of the fixed-dose combination of nivolumab and relatlimab for the first-line treatment of advanced (unresectable or metastatic) melanoma in adults and adolescents 12 years of age and older with tumor cell PD-L1 expression < 1%. The European Commission (EC), which has the authority to approve medicines for the European Union (EU), will now review the CHMP opinion.
“We are very proud of the role we have played in progressing the treatment of advanced melanoma over the years. As part of our mission to deliver new medicines for patients, we have continued to develop new dual immunotherapy combinations,” said Paul Basciano, development lead, relatlimab, Bristol Myers Squibb. “This positive CHMP opinion marks the first step toward the potential approval of the first LAG-3 blocking antibody combination – and the third distinct checkpoint inhibitor for BMS – for advanced melanoma patients in the EU.”
The positive opinion is based upon efficacy and safety results from the Phase 2/3 RELATIVITY-047 trial. The trial showed that treatment with the fixed-dose combination of nivolumab and relatlimab more than doubled the median progression-free survival (PFS), including in patients with tumor cell PD-L1 expression < 1%, when compared to nivolumab monotherapy – an established standard of care. The proposed indication for the EU is based upon an exploratory analysis of the data in patients with tumor cell PD-L1 expression < 1%. No new safety events were identified with the combination when compared to nivolumab monotherapy.
On March 18, 2022, the U.S. Food and Drug Administration (FDA) approved the fixed-dose combination of nivolumab and relatlimab as Opdualag™ (nivolumab and relatlimab-rmbw) for the treatment of adult and pediatric patients 12 years of age or older with unresectable or metastatic melanoma. Please see important safety information from the U.S. prescribing information below.
Bristol Myers Squibb thanks the patients and investigators involved in the RELATIVITY-047 trial.
About RELATIVITY-047
RELATIVITY-047 is a global, randomized, double-blind Phase 2/3 study evaluating the fixed-dose combination of nivolumab and relatlimab versus nivolumab alone in patients with previously untreated metastatic or unresectable melanoma. Patients were enrolled regardless of tumor cell PD-L1 expression. The trial excluded patients with active autoimmune disease, medical conditions requiring systemic treatment with moderate or high dose corticosteroids or immunosuppressive medications, uveal melanoma, and active or untreated brain or leptomeningeal metastases. The primary endpoint of the trial is progression-free survival (PFS) determined by Blinded Independent Central Review (BICR) using Response Evaluation Criteria in Solid Tumors (RECIST v1.1) in the all-comer population. The secondary endpoints are overall survival (OS) and objective response rate (ORR) in the all-comer population. A total of 714 patients were randomized 1:1 to receive a fixed-dose combination of nivolumab (480 mg) and relatlimab (160 mg) or nivolumab (480 mg) by intravenous infusion every four weeks until disease progression, unacceptable toxicity or withdrawal of consent.
About LAG-3
Lymphocyte-activation gene 3 (LAG-3) is a cell-surface molecule expressed on effector T cells and regulatory T cells (Tregs) and functions to control T-cell response, activation and growth. Preclinical studies indicate that inhibition of LAG-3 may restore effector function of exhausted T cells and potentially promote an anti-tumor response. Early research demonstrates that targeting LAG-3 in combination with other potentially complementary immune checkpoints may be a key strategy to more effectively potentiate anti-tumor immune activity.
Bristol Myers Squibb is evaluating relatlimab, its LAG-3-blocking antibody, in clinical trials in combination with other agents in a variety of tumor types.
OPDUALAG U.S. INDICATION
Opdualag™ (nivolumab and relatlimab-rmbw) is indicated for the treatment of adult and pediatric patients 12 years of age or older with unresectable or metastatic melanoma.
About Bristol Myers Squibb
Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.
Bristol-Myers new immunotherapy Opdualag gets EMA panel nod to treat skin cancer
Jul. 22, 2022 7:23 AM ET Bristol-Myers Squibb Company (BMY)
By: Ravikash, SA News Editor1 Comment
- A committee of the European Medicines Agency (EMA) recommended approval of Bristol-Myers Squibb's (NYSE:BMY) Opdualag to treat a type of skin cancer.
- https://seekingalpha.com/news/3859540-bristol-myers-new-immunotherapy-opdualag-gets-ema-panel-nod-to-treat-skin-cancer
- https://seekingalpha.com/symbol/BMY
- https://www.opdualag.com/
Mounjaro & Retsevmo
Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 18-21 July 2022
22/07/2022
11 new medicines recommended for approval
EMA’s human medicines committee (CHMP) recommended 11 medicines for approval at its July 2022 meeting.
The CHMP recommended granting a marketing authorisation for Amvuttra* (vutrisiran) for the treatment of adults with hereditary transthyretin-mediated amyloidosis, a rare life-threatening disease that damages multiple nerves across the body.
The committee adopted a positive opinion for Celdoxome pegylated liposomal (doxorubicin hydrochloride) for the treatment of metastatic breast cancer, advanced ovarian cancer, progressive multiple myeloma and Kaposi's sarcoma, a type of cancer that affects people with AIDS.
Illuzyce (lutetium (177lu) chloride), a radiopharmaceutical precursor, received a positive opinion from the CHMP. Illuzyce is not intended for direct use in patients and must be used only for the radiolabelling of carrier medicines that have been specifically developed and authorised for radiolabelling with lutetium chloride.
The CHMP recommended granting a marketing authorisation for Lupkynis (voclosporin) for the treatment of lupus nephritis, an inflammation of the kidney caused by lupus. Lupus is an auto immune disease in which the body's immune system attacks healthy tissues.
The committee adopted a positive opinion for Mounjaro (tirzepatide) for the treatment of adults with type 2 diabetes mellitus. Around 30 million people suffer from diabetes in the European Union (EU).
The CHMP gave a positive opinion under exceptional circumstances for Nulibry* (fosdenopterin) for the treatment of molybdenum cofactor deficiency type A. This is an ultra-rare condition that appears shortly after birth and leads to brain injury and death.
Opdualag (relatlimab / nivolumab), intended for the treatment of melanoma, a type of skin cancer, that has spread to other parts of the body and cannot be removed by surgery, received a positive opinion from the CHMP.
The committee recommended granting a conditional marketing authorisation for Tecvayli* (teclistamab) for the treatment of adults with relapsed and refractory multiple myeloma, who have received at least three prior therapies. Multiple myeloma is a rare cancer of the bone marrow that affects plasma cells, a type of white blood cell that produces antibodies. Tecvayli was supported through EMA's PRIority MEdicines (PRIME) scheme, which provides early and enhanced scientific and regulatory support for medicines that have a particular potential to address patients' unmet medical needs. The CHMP reviewed the application for marketing authorisation under an accelerated timetable to enable faster patient access to this medicine. See more information in the news announcement in the grid below.
The CHMP gave a positive opinion for Tezspire (Tezepelumab), intended as an add-on treatment in adult and adolescent patients with severe asthma. Asthma is a chronic condition that affects around 6% of the EU population.
EMA panel recommends approval of Lilly drug Mounjaro, extension of indication for Retsevmo
Jul. 22, 2022 9:13 AM ET
By: Anuron Mitra, SA News Editor
- The European Union drug regulator's human medicines committee has recommended granting a marketing authorization to Eli Lilly's (NYSE:LLY) Mounjaro for the treatment of type 2 diabetes, according to the highlights of their latest meeting released on Friday.
- https://seekingalpha.com/news/3859615-ema-panel-recommends-approval-of-lilly-drug-mounjaro-extension-of-indication-for-retsevmo
- https://seekingalpha.com/symbol/LLY
- https://www.mounjaro.com/
Tezspire (tezepelumab)
Tezspire
tezepelumab
Table of contents
Opinion
On 21 July 2022, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Tezspire, intended as add-on treatment in adult and adolescent patients with severe asthma. The applicant for this medicinal product is AstraZeneca AB.
Tezspire will be available as a 210 mg solution for injection. The active substance of Tezspire is tezepelumab, a human monoclonal antibody (IgG2 lambda) directed against thymic stromal lymphopoietin (TSLP) (ATC code: R03DX11). Blocking TSLP with tezepelumab reduces a broad spectrum of biomarkers and cytokines associated with airway inflammation, but the mechanism of action of tezepelumab in asthma has not been definitively established.
The benefits of Tezspire are its ability to reduce the exacerbation rate and improve lung function in patients with severe asthma. The most common side effects are pharyngitis, rash, arthralgia and injection site reactions.
The full indication is:
Tezspire is indicated as an add-on maintenance treatment in adults and adolescents 12 years and older with severe asthma who are inadequately controlled despite high dose inhaled corticosteroids plus another medicinal product for maintenance treatment.
Tezspire should be prescribed by physicians experienced in the treatment of severe asthma.
Detailed recommendations for the use of this product will be described in the summary of product characteristics (SmPC), which will be published in the European public assessment report (EPAR) and made available in all official European Union languages after the marketing authorisation has been granted by the European Commission.
EMA panel backs approval of Amgen/AstraZeneca's asthma drug Tezspire; Ultomiris for expanded use
Jul. 22, 2022 9:41 AM ET
By: Ravikash, SA News Editor
- A committee of the European Medicines Agency (EMA) recommended the approval of Amgen (NASDAQ:AMGN) and AstraZeneca's (NASDAQ:AZN) Tezspire as an add-on maintenance therapy for patients 12 years and older with severe asthma who are inadequately controlled despite high dose inhaled corticosteroids plus another medicinal product for maintenance treatment.
- https://seekingalpha.com/news/3859634-ema-panel-backs-approval-of-amgenastrazenecas-asthma-drug-tezspire-ultomiris-for-expanded-use
- https://seekingalpha.com/symbol/AMGN
- https://seekingalpha.com/symbol/AZN
- https://www.tezspire.com/
NEXPOVIO® (selinexor)
Karyopharm and Menarini Group Receive Full Marketing Authorisation from the European Commission for NEXPOVIO® (selinexor) for the Treatment of Patients with Multiple Myeloma After at Least One Prior Therapy
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– Based on Results from Phase 3 BOSTON Study, Marketing Authorisation Expands Multiple Myeloma Indication –
– Approval Follows Positive Opinion by European Committee for Medicinal Products for Human Use (CHMP) in May 2022 –
NEWTON, Mass. and FLORENCE, Italy, July 21, 2022 /PRNewswire/ -- Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, and the Menarini Group ("Menarini"), a privately-held, leading international pharmaceutical company, today announced that the European Commission (EC) has granted Marketing Authorisation for NEXPOVIO® (selinexor), a first-in-class, oral exportin 1 (XPO1) inhibitor, in combination with once-weekly bortezomib (Velcade®) and low-dose dexamethasone (SVd) for the treatment of adults with multiple myeloma who have received at least one prior therapy. With this approval for the extension of NEXPOVIO®'s indication in the European Union (EU), the conditional marketing authorisation is now converted to a full approval. The marketing authorisation, which marks the second indication for NEXPOVIO®, is valid in all 27 member states of the EU as well as Iceland, Liechtenstein, Norway, and Northern Ireland. Stemline Therapeutics B.V., a wholly owned subsidiary of the Menarini Group, will be responsible for all commercialization activities in Europe.
The approval follows a positive opinion granted in May 2022 by the CHMP based on results from the Phase 3 BOSTON study that demonstrated once-weekly SVd resulted in a statistically significant reduction in the risk of disease progression or death compared to standard twice-weekly bortezomib plus dexamethasone (Vd) regimen. The results from the BOSTON study were published in The Lancet (Grosicki, et al.) in November 2020.
"The European Commission's approval of an expanded use of NEXPOVIO® provides another option for patients with multiple myeloma who have relapsed, or become resistant to current treatment regimens," said Richard Paulson, President and Chief Executive Officer of Karyopharm. "Our decision to pursue approval for this patient population is indicative of our commitment to expand access to selinexor across the globe and we look forward to working closely with Menarini who will commercialize NEXPOVIO® in Europe."
"The approval of NEXPOVIO® marks an important step forward for patients in Europe where nearly 51,000 new cases of multiple myeloma are diagnosed each year and therapeutic options are limited," said Elcin Barker Ergun, Chief Executive Officer of Menarini. "We are committed to offering patients and physicians a valuable new treatment option and are working hard to make NEXPOVIO® available in different European countries as quickly as possible."
About the BOSTON study
The Marketing Authorisation is based upon the Phase 3 BOSTON (Bortezomib, Selinexor and Dexamethasone) study, which was a multi-center, randomized study (NCT03110562) that evaluated 402 adult patients with relapsed or refractory multiple myeloma who had received one to three prior lines of therapy. The study was designed to compare the efficacy, safety and certain health-related quality of life parameters of once-weekly SVd versus twice-weekly Vd. The primary endpoint of the study was progression-free survival and key secondary endpoints included overall response rate, rate of peripheral neuropathy, and others. To learn more about this study, please refer to Karyopharm and Menarini's press release on the positive CHMP opinion issued on May 20, 2022.
About NEXPOVIO® (selinexor)
NEXPOVIO®, which is marketed as XPOVIO® in the U.S., has been approved in the following oncology indications by the European Commission: (i) in combination with dexamethasone for the treatment of multiple myeloma in adult patients who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, two immunomodulatory agents and an anti-CD38 monoclonal antibody, and who have demonstrated disease progression on the last therapy; and (ii) in combination with bortezomib and dexamethasone for the treatment of adults with multiple myeloma who have received at least one prior therapy.
The expanded NEXPOVIO® indication now allows adult patients with multiple myeloma to be treated in earlier lines of therapy. The indication for NEXPOVIO® is valid in the EU Member States as well as Iceland, Liechtenstein, Norway, and Northern Ireland. NEXPOVIO® is also approved in the UK under a Conditional Marketing Authorisation. The extension of indication in combination with bortezomib and dexamethasone for the treatment of adults with multiple myeloma who have received at least one prior therapy is currently under review by Medicines and Healthcare Products Regulatory Agency.
NEXPOVIO® is a first-in-class, oral exportin 1 (XPO1) inhibitor. NEXPOVIO® functions by selectively binding to and inhibiting the nuclear export protein exportin 1 (XPO1, also called CRM1). NEXPOVIO® blocks the nuclear export of tumor suppressor, growth regulatory and anti-inflammatory proteins, leading to accumulation of these proteins in the nucleus and enhancing their anti-cancer activity in the cell. The forced nuclear retention of these proteins can counteract a multitude of the oncogenic pathways that, unchecked, allow cancer cells with severe DNA damage to continue to grow and divide in an unrestrained fashion.
Please see NEXPOVIO® Summary of Product Characteristics and European Public Assessment Report at https://ec.europa.eu/health/documents/community-register/html/h1537.htm
About Karyopharm Therapeutics
Karyopharm Therapeutics Inc. (Nasdaq: KPTI) is a commercial-stage pharmaceutical company pioneering novel cancer therapies. Since its founding, Karyopharm has been the industry leader in oral Selective Inhibitor of Nuclear Export (SINE) compound technology, which was developed to address a fundamental mechanism of oncogenesis: nuclear export dysregulation. Karyopharm's lead SINE compound and first-in-class, oral exportin 1 (XPO1) inhibitor, XPOVIO® (selinexor), is approved in the U.S. and marketed by the Company in three oncology indications and has received regulatory approvals in various indications in a growing number of ex-U.S. territories and countries, including Europe and the United Kingdom (as NEXPOVIO®), China, Singapore, Canada, Israel, South Korea, and Australia. Karyopharm has a focused pipeline targeting multiple high unmet need cancer indications, including in multiple myeloma, endometrial cancer, myelodysplastic syndromes and myelofibrosis. For more information about our people, science and pipeline, please visit www.karyopharm.com, and follow us on Twitter at @Karyopharm and LinkedIn.
About Menarini Group
The Menarini Group is a leading international pharmaceutical and diagnostics company, with a turnover of over $4 billion and over 17,000 employees. Menarini is focused on therapeutic areas with high unmet needs with products for oncology, cardiology, pneumology, gastroenterology, infectious diseases, diabetology, inflammation, and analgesia. With 18 production sites and 9 Research and Development centers, Menarini's products are available in 140 countries worldwide. For further information, please visit www.menarini.com.
Karyopharm wins full EU approval for multiple myeloma therapy
Jul. 21, 2022 7:19 AM ET
Karyopharm Therapeutics Inc. (KPTI)
By: Dulan Lokuwithana, SA News Editor1 Comment
- Commercial-stage biotech Karyopharm Therapeutics (NASDAQ:KPTI) and Italian pharma company Menarini Group announced on Thursday that the European Commission granted marketing authorization for a multiple myeloma therapy containing the oral exportin 1 inhibitor Nexpovio.
- https://seekingalpha.com/news/3858977-kpti-stock-in-focus-with-full-eu-approval-for-multiple-myeloma-therapy
- https://seekingalpha.com/symbol/KPTI
- https://www.xpoviopro.com/
- https://www.karyopharm.com/
Toripalimab
Coherus and Junshi Biosciences Announce FDA Acceptance of Resubmission of BLA for Toripalimab for the Treatment of Nasopharyngeal Carcinoma
- FDA has set a target action date of December 23, 2022 for the toripalimab BLA -
- Toripalimab will be the first and only immuno-oncology agent for NPC in U.S., if approved -
REDWOOD CITY, Calif., and SHANGHAI, China, July 06, 2022 (GLOBE NEWSWIRE) -- Coherus BioSciences, Inc. (“Coherus”, Nasdaq: CHRS) and Shanghai Junshi Biosciences Co., Ltd. (“Junshi Biosciences”, HKEX: 1877; SSE: 688180) announced today that the United States Food and Drug Administration ("FDA") has accepted for review the Biologics License Application (“BLA”) resubmission for toripalimab in combination with gemcitabine and cisplatin as first-line treatment for patients with advanced recurrent or metastatic nasopharyngeal carcinoma (“NPC”) and for toripalimab monotherapy for the second-line or later treatment of recurrent or metastatic NPC after platinum-containing chemotherapy.
The FDA has set a Prescription Drug User Fee Act ("PDUFA") action date for December 23, 2022. The Agency earlier communicated that the review timeline for the BLA resubmission would be six months, as onsite inspections in China would be required. Travel restrictions related to the COVID-19 pandemic previously hindered the FDA’s ability to complete required inspections. Coherus plans to launch toripalimab in the United States in the first quarter of 2023, if approved.
“Toripalimab would address a critical unmet medical need for patients with nasopharyngeal carcinoma, an aggressive cancer for which there are currently no FDA-approved immunotherapy treatments. We collaborated closely with our partner, Junshi Biosciences, to complete the quality process changes requested by the FDA and facilitate the rapid resubmission of the toripalimab BLA,” said Dr. Theresa LaVallee, Chief Development Officer of Coherus.
“Although the COVID-19 pandemic has created tremendous challenges for everyone, our dedication to bring better treatment options to patients around the world remains steadfast,” said Dr. Patricia Keegan, Chief Medical Officer of Junshi Biosciences. “Through our concerted efforts with our partner, Coherus, we have made continual progress towards obtaining toripalimab’s first marketing authorization outside of China. Over the next several months, we will work closely with the FDA to facilitate the review of this novel drug.”
“For Coherus, the toripalimab resubmission is one of several key development and commercialization milestones we are sharply focusing on over the next twelve months, and we are pleased with the Company’s execution and progress on all of them,” said Denny Lanfear, CEO of Coherus. “We now look forward to the August 2, 2022 target action date for our BLA for CIMERLI™, our Lucentis® biosimilar, followed by product launch which we are confident will be very successful. The toripalimab December 2022 PDUFA date follows directly, and the projected toripalimab launch in Q1 2023 will formally mark our entry into immuno-oncology, where Coherus will be one of just a handful of companies with a proprietary PD-1 as a foundation stone to build its oncology franchise upon. Lastly, twelve months from now, in July 2023, we expect to begin marketing our Humira® biosimilar, YUSIMRY®, which was approved by the FDA in December 2021. Preparations for that commercial launch are going very well. Biosimilar market execution is a demonstrated Coherus competency, and we believe that our commercialization strategy provides a robust framework against which we can successfully execute to meet our market expectations and share projections.”
Following approval of toripalimab for NPC, Coherus’ strategy in the US includes evaluating toripalimab’s ability to deliver substantial clinical benefit in significant indications, in combination with other cancer drugs and immunotherapies, through co-development agreements.
About Toripalimab in NPC
NPC is a type of aggressive cancer that starts in the nasopharynx, the upper part of the throat behind the nose and near the base of skull. Due to the location of the primary tumor, surgery is rarely an option, and patients with localized disease are treated primarily with radiation and chemotherapy. In the United States, there are presently no immunotherapies approved for the treatment of NPC.
The toripalimab BLA is supported by the results from JUPITER-02, a randomized, double blind, placebo-controlled, international multi-center Phase 3 clinical trial, as well as POLARIS-02, a multi-center, open-label, pivotal Phase 2 clinical study. The JUPITER-02 results were first presented in June 2021 in a plenary session of the American Society of Clinical Oncology (“ASCO”) annual meeting (#LBA2) and subsequently published in detail as the cover article of the September 2021 issue of Nature Medicine. The POLARIS-02 results were published online in January 2021 in the Journal of Clinical Oncology.
The FDA has granted Breakthrough Therapy designation (“BTD”) for toripalimab in combination with chemotherapy (gemcitabine and cisplatin) for the 1st line treatment of recurrent, locally advanced or primary metastatic non-keratinizing NPC and for toripalimab monotherapy for patients with recurrent or metastatic non-keratinizing NPC with disease progression on or after platinum-containing chemotherapy. Additionally, the FDA has granted Orphan Drug designation for toripalimab for NPC.
In China, the National Medical Products Administration (“NMPA”) in 2021 approved toripalimab for two NPC indications.
About Toripalimab
Toripalimab is an anti-PD-1 monoclonal antibody developed for its ability to block PD-1 interactions with its ligands, PD-L1 and PD-L2, and for enhanced receptor internalization (endocytosis function). Blocking PD-1 interactions with PD-L1 and PD-L2 promotes the immune system’s ability to attack and kill tumor cells. In China, toripalimab was the first domestic anti-PD-1 monoclonal antibody approved for marketing (approved in China as TUOYI®). Currently, there are five approved indications for toripalimab in China.
About Junshi Biosciences
Founded in December 2012, Junshi Biosciences (HKEX: 1877; SSE: 688180) is an innovation-driven biopharmaceutical company dedicated to the discovery, development, and commercialization of innovative therapeutics. The company has established a diversified R&D pipeline comprising over 50 drug candidates, with five therapeutic focus areas covering cancer, autoimmune, metabolic, neurological, and infectious diseases. Junshi Biosciences was the first Chinese pharmaceutical company that obtained marketing approval for anti-PD-1 monoclonal antibody in China. Its first-in-human anti-BTLA monoclonal antibody for the treatment of various cancers is the first in the world to be approved for clinical trials by the FDA and NMPA and has since entered Phase Ib/II trials in both China and the US. Its anti-PCSK9 monoclonal antibody was the first in China to be approved for clinical trials by the NMPA.
In the face of the COVID-19 pandemic, Junshi Biosciences responded swiftly and strongly, joining forces with Chinese and international scientific research institutions and enterprises to develop an arsenal of drug candidates to combat COVID-19, taking the initiative to shoulder the social responsibility of Chinese pharmaceutical companies by prioritizing and accelerating COVID-19 R&D. Among the many drug candidates is JS016 (etesevimab), China’s first neutralizing fully human monoclonal antibody against SARS-CoV-2 and the result of the combined efforts of Junshi Biosciences, the Institute of Microbiology of the Chinese Academy of Science and Lilly. JS016 administered with bamlanivimab has been granted Emergency Use Authorizations (EUA) in over 15 countries and regions worldwide. Meanwhile, VV116, a new oral nucleoside analog anti-SARS-CoV-2 drug designed to hinder virus replication, is in global Phase III clinical trials. The JS016 and VV116 programs are a part of the company’s continuous innovation for disease control and prevention of the global pandemic.
Junshi Biosciences has more than 2,800 employees in the United States (San Francisco and Maryland) and China (Shanghai, Suzhou, Beijing and Guangzhou). For more information, please visit: http://junshipharma.com.
About Coherus BioSciences
Coherus is a commercial stage biopharmaceutical company building a leading immuno-oncology franchise funded with cash generated by its FDA-approved products. In 2021, Coherus in-licensed toripalimab, an anti-PD-1 antibody, in the United States and Canada. The resubmission of the BLA for toripalimab for the treatment of NPC was accepted by the FDA in July 2022. Toripalimab is also being evaluated in pivotal clinical trials for the treatment of rare and highly prevalent cancers. Coherus markets UDENYCA® (pegfilgrastim-cbqv), a biosimilar of Neulasta® in the United States, and expects to launch the FDA-approved Humira® biosimilar YUSIMRY™ (adalimumab-aqvh) in the United States in 2023. The FDA is currently reviewing the biologics license application for CIMERLI™, a biosimilar of Lucentis® (ranibizumab injection), with a target action date of August 2022.
Source: Coherus BioSciences, Inc.
Coherus/Junshi's toripalimab gets orphan drug tag in EU for head/neck cancer subtype
Jul. 21, 2022 7:21 AM ET
Shanghai Junshi Biosciences Co., Ltd. (SHJBF), CHRS
By: Ravikash, SA News Editor
- The European Commission granted orphan medicinal product designation to Coherus BioSciences (NASDAQ:CHRS) and Shanghai Junshi Biosciences' (OTCPK:SHJBF) medicine toripalimab to treat nasopharyngeal carcinoma (NPC).
- https://seekingalpha.com/news/3858979-coherusjunshis-toripalimab-gets-orphan-drug-tag-in-eu-for-headneck-cancer-subtype
- https://seekingalpha.com/symbol/CHRS
- https://seekingalpha.com/symbol/SHJBF
- https://www.coherus.com/
- https://www.hkex.com.hk/Market-Data/Securities-Prices/Equities/Equities-Quote?sym=1877&sc_lang=en
Opzelura™ (Ruxolitinib) Cream
Incyte Announces U.S. FDA Approval Of Opzelura™ (Ruxolitinib) Cream For The Treatment Of Vitiligo
July 18, 2022 at 9:08 PM EDTPDF Version
- Opzelura is the first and only FDA-approved product for repigmentation in nonsegmental vitiligo
- Phase 3 data supporting the approval show treatment with Opzelura resulted in improvements in facial and total body repigmentation
- Fifty-two week data demonstrate continued improvements in repigmentation with longer duration of treatment
- Investor conference call and webcast scheduled for July 19, 2022, at 8:00 a.m. EDT
WILMINGTON, Del.--(BUSINESS WIRE)--Jul. 18, 2022--Incyte (Nasdaq:INCY) today announced that the U.S. Food and Drug Administration (FDA) has approved Opzelura™ (ruxolitinib) cream 1.5% for the topical treatment of nonsegmental vitiligo in adult and pediatric patients 12 years of age and older. Opzelura is the first and only FDA-approved treatment for repigmentation in patients with vitiligo, and the only topical formulation of a Janus kinase (JAK) inhibitor approved in the United States. Vitiligo is a chronic autoimmune disease characterized by depigmentation of skin.
“With the approval of Opzelura in nonsegmental vitiligo, Incyte has once again delivered a treatment to patients with high unmet medical need who previously had no approved therapies,” said Hervé Hoppenot, Chief Executive Officer,Incyte. “We are proud of Incyte’s scientists and development teams that have made this milestone possible, and we're pleased that eligible vitiligo patients now have a choice to address repigmentation.”
In patients with non-segmental vitiligo, Opzelura is approved for continuous topical use twice daily to affected areas of up to 10% body surface area. Satisfactory patient response may require treatment with Opzelura for more than 24 weeks.
The FDA approval was based on data from the pivotal Phase 3 TRuE-V clinical trial program (TRuE-V1 and TRuE-V2), evaluating the safety and efficacy of Opzelura versus vehicle in more than 600 people with nonsegmental vitiligo, age 12 and older. In the studies, treatment with Opzelura resulted in significant improvements in VASI scores, which represent improvements in facial and total body repigmentation at Week 24 (primary analysis) compared to vehicle (non-medicated cream) and in an open-label extension at Week 52.
Results at Week 24, which were consistent across both studies, showed that approximately 30% of patients treated with Opzelura achieved ≥75% improvement from baseline in the facial Vitiligo Area Scoring Index (F-VASI75), the primary endpoint, compared to approximately 8% and 13% of patients treated with vehicle in TRuE-V1 and TRuE-V2, respectively. At Week 52, approximately 50% of Opzelura-treated patients achieved F-VASI75.
Additionally, at Week 24, more than 15% of patients treated with Opzelura achieved ≥90% improvement from baseline in F-VASI (F-VASI90), compared to approximately 2% of patients treated with vehicle. At Week 52, the percentage of Opzelura-treated patients who achieved F-VASI90 doubled to approximately 30%.
In the vehicle controlled period of the Phase 3 studies, the most common adverse reactions (incidence ≥ 1%) are application site acne, application site pruritus, nasopharyngitis, headache, urinary tract infection, application site erythema, and pyrexia1. The labeling for Opzelura includes a Boxed Warning for serious infections, mortality, malignancy, major adverse cardiovascular events and thrombosis. See additional Important Safety Information below.
Week 52 data from the Phase 3 TRuE-V studies were featured in an oral presentation at the late-breaking abstract session at the American Academy of Dermatology (AAD) Annual 2022 Meeting.
Vitiligo is a chronic autoimmune disease characterized by depigmentation of skin that results from the loss of pigment-producing cells known as melanocytes. Over-activity of the JAK signaling pathway is believed to drive inflammation involved in the pathogenesis and progression of vitiligo. In the United States, more than 1.5 million people are diagnosed with vitiligo2. The overall prevalence of the condition is estimated to be approximately 2-3 million3, with the majority of patients (approximately 85%) suffering from nonsegmental vitiligo4. Vitiligo can occur at any age, although many patients with vitiligo will experience initial symptoms before the age of 305.
“Vitiligo is an immune-mediated disease that can be unpredictable, making it particularly difficult to treat,” said David Rosmarin, M.D., Vice Chair of Research and Education, Department of Dermatology at Tufts Medical Center. “There have been no FDA-approved therapies available to date and the approval of Opzelura therefore marks a significant milestone. I welcome a medical treatment that helps my patients with nonsegmental vitiligo who are interested in potentially reversing the depigmentation caused by their disease.”
In September 2021, Opzelura was approved by the FDA for the topical short-term and non-continuous chronic treatment of mild to moderate atopic dermatitis (AD) in non-immunocompromised patients 12 years of age and older whose disease is not adequately controlled with topical prescription therapies, or when those therapies are not advisable.
Incyte is committed to supporting patients and removing barriers to access medicines. Eligible patients in the United States who are prescribed Opzelura have access to IncyteCARES (Connecting to Access, Reimbursement, Education and Support), a program offering patient support, including financial assistance and ongoing education and resources to eligible patients. For more information about IncyteCARES, please visit www.incytecares.com or call 1-800-583-6964, Monday through Friday, from 8 a.m. to 8 p.m. EDT.
About TRuE-V
The TRuE-V clinical trial program includes two Phase 3 studies, TRuE-V1 (NCT04052425) and TRuE-V2 (NCT04057573), evaluating the safety and efficacy of ruxolitinib cream in patients with vitiligo. Each study enrolled approximately 300 patients (age ≥12 years) who have been diagnosed with nonsegmental vitiligo.
About Opzelura™ (ruxolitinib) Cream 1.5%
Opzelura, a novel cream formulation of Incyte’s selective JAK1/JAK2 inhibitor ruxolitinib, is the first and only topical JAK inhibitor approved for use in the United States, indicated for the topical treatment of nonsegmental vitiligo in adult and pediatric patients 12 years of age and older and the topical short-term and non-continuous chronic treatment of mild to moderate atopic dermatitis (AD) in non-immunocompromised patients 12 years of age and older whose disease is not adequately controlled with topical prescription therapies, or when those therapies are not advisable. Use of Opzelura in combination with therapeutic biologics, other JAK inhibitors, or potent immunosuppressants, such as azathioprine or cyclosporine, is not recommended.
In October 2021, Incyte announced the validation of the European Marketing Authorization Application (MAA) for ruxolitinib cream as a potential treatment for adolescents and adults (age >12 years) with nonsegmental vitiligo with facial involvement.
Incyte has worldwide rights for the development and commercialization of ruxolitinib cream, marketed in the United States as Opzelura.
Opzelura is a trademark of Incyte.
Please see the Full Prescribing Information, including Boxed Warning, and Medication Guide for OPZELURA.
INDICATIONS AND USAGE
OPZELURA is a prescription medicine used on the skin (topical) for:
- short-term and non-continuous chronic treatment of mild to moderate eczema (atopic dermatitis) in non-immunocompromised adults and children 12 years of age and older whose disease is not well controlled with topical prescription therapies or when those therapies are not recommended
- the treatment of a type of vitiligo called nonsegmental vitiligo in adults and children 12 years of age and older
The use of OPZELURA along with therapeutic biologics, other JAK inhibitors, or strong immunosuppressants such as azathioprine or cyclosporine is not recommended.
It is not known if OPZELURA is safe and effective in children less than 12 years of age with atopic dermatitis or nonsegmental vitiligo.
About Incyte
Incyte is a Wilmington, Delaware-based, global biopharmaceutical company focused on finding solutions for serious unmet medical needs through the discovery, development and commercialization of proprietary therapeutics. For additional information on Incyte, please visit Incyte.com and follow @Incyte.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220718005819/en/
Source: Incyte
Incyte's Opzelura for vitiligo wins FDA approval following extended review
Jul. 19, 2022 6:10 AM ET Incyte Corporation (INCY)
By: Ravikash, SA News Editor
- The U.S. Food and Drug Administration (FDA) approved Incyte (NASDAQ:INCY) Opzelura cream to treat non-segmental vitiligo in patients 12 years of age and older.
- https://seekingalpha.com/news/3857907-incytes-opzelura-for-vitiligo-wins-fda-approval
- https://seekingalpha.com/symbol/INCY
- https://www.opzelura.com/
- https://www.incyte.com/
ENHERTU® (fam-trastuzumab deruxtecan-nxki) for injection
Enhertu approved in the EU for patients with HER2-positive metastatic breast cancer treated with one or more prior
anti-HER2-based regimens
PUBLISHED19 July 2022 19 July 2022 07:00 BST
Approval broadens indication for AstraZeneca and Daiichi Sankyo’s Enhertu across Europe to earlier use in HER2-positive metastatic breast cancer
Based on ground-breaking DESTINY-Breast03 results in which Enhertu demonstrated a 72% reduction in the risk of disease progression or death vs. trastuzumab emtansine
(T-DM1)
AstraZeneca and Daiichi Sankyo’s Enhertu (trastuzumab deruxtecan) has been approved in the European Union (EU) as a monotherapy for the treatment of adult patients with unresectable or metastatic HER2-positive breast cancer who have received one or more prior anti-HER2-based regimens.
Enhertu is a specifically engineered HER2-directed antibody drug conjugate (ADC) being jointly developed and commercialised by AstraZeneca and Daiichi Sankyo.
The approval by the European Commission (EC) follows the positive opinion of the Committee for Medicinal Products for Human Use and is based on results from the DESTINY-Breast03 Phase III trial, which were published in The New England Journal of Medicine.1 In the trial, Enhertu reduced the risk of disease progression or death by 72% versus trastuzumab emtansine (T-DM1) (hazard ratio [HR] 0.28; 95% confidence interval [CI] 0.22-0.37; p<0.000001) in patients with HER2-positive unresectable and/or metastatic breast cancer previously treated with trastuzumab and a taxane.
In Europe, more than 530,000 patients are diagnosed with breast cancer each year.2 Approximately one in five patients with breast cancer are considered HER2-positive.3 Despite initial treatment with trastuzumab, pertuzumab and a taxane, patients with HER2-positive metastatic breast cancer will often experience disease progression.4,5
Javier Cortés, MD, PhD, Head, International Breast Cancer Center (IBCC), Barcelona, Spain, said: “This approval is an important milestone for patients and clinicians in Europe, since previously treated patients with HER2-positive metastatic breast cancer typically experience disease progression in less than a year with historical standard of care treatment. In the DESTINY-Breast03 trial, the time to progression was extended well beyond a year for patients receiving Enhertu, illustrating the potential for this medicine to set a new benchmark in the treatment of HER2-positive metastatic breast cancer.”
Dave Fredrickson, Executive Vice President, Oncology Business Unit, AstraZeneca, said: “With this approval, patients across Europe with HER2-positive metastatic breast cancer will have the opportunity to be treated with Enhertu even earlier in the treatment of their disease, improving their chance for better outcomes beyond what we can already offer patients treated in later-line settings. Today’s news is a further step in achieving our vision to continuously bring the transformative potential of Enhertu to patients as early as possible in their treatment to improve cancer outcomes.”
Ken Keller, Global Head of Oncology Business, and President and CEO, Daiichi Sankyo, Inc., said: “We believe there is a significant need to transform outcomes for patients with HER2-positive metastatic breast cancer in Europe. In DESTINY-Breast03, treatment with Enhertu demonstrated superior progression-free survival and a doubling of the response rate compared to another HER2-directed ADC. With this approval we are now able to offer patients with HER2-positive metastatic breast cancer another option earlier in their treatment.”
Additional results from the DESTINY-Breast03 Phase III trial showed that in the secondary endpoint of overall survival (OS), there was a strong trend towards improved OS with Enhertu (HR 0.55; 95% CI 0.36-0.86), however this analysis is not yet mature and further follow-up is ongoing. Nearly all patients (96.1%) treated with Enhertu were alive at nine months compared to 91.3% of patients treated with T-DM1. Confirmed objective response rate (ORR) was more than doubled in the Enhertu arm versus the T-DM1 arm (79.7% vs. 34.2%).
The safety of Enhertu has been evaluated in a pooled analysis of 573 patients across multiple tumour types who had received at least one dose of Enhertu (5.4 mg/kg) in clinical trials. The most common adverse reactions were nausea (77.0%), fatigue (57.2%), vomiting (46.8%), alopecia (38.0%) and neutropenia (34.6%). Cases of interstitial lung disease (ILD) or pneumonitis were reported in 12.0% of patients. Most ILD cases were Grade 1 (2.6%) and Grade 2 (7.3%). Grade 3 cases occurred in 0.7% of patients, no Grade 4 cases occurred, and Grade 5 cases occurred in 1.4% of patients.
Based on the results of DESTINY-Breast03, the European Society for Medical Oncology Clinical Practice Guidelines were updated in October 2021 to recommend Enhertu for use as the preferred second-line therapy for patients with HER2-positive metastatic breast cancer following progression with a taxane and trastuzumab.6
As part of this approval, the EC has also extended the market protection period for Enhertu in this setting by one extra year based on the significant clinical benefit compared to existing approved therapies.
DESTINY-Breast03
DESTINY-Breast03 is a global, head-to-head, randomised, open-label, registrational Phase III trial evaluating the efficacy and safety of Enhertu (5.4mg/kg) versus T-DM1 in patients with HER2-positive unresectable and/or metastatic breast cancer previously treated with trastuzumab and a taxane.
The primary efficacy endpoint of DESTINY-Breast03 is PFS based on blinded independent central review. OS is a key secondary efficacy outcome measure. Secondary efficacy endpoints include ORR, duration of response and PFS based on investigator assessment.
DESTINY-Breast03 enrolled 524 patients at multiple sites in Asia, Europe, North America, Oceania and South America. Results from DESTINY-Breast03 have been published in The New England Journal of Medicine.1 For more information about the trial, visit ClinicalTrials.gov.
Enhertu
Enhertu is a HER2-directed ADC. Designed using Daiichi Sankyo’s proprietary DXd ADC technology, Enhertu is the lead ADC in the oncology portfolio of Daiichi Sankyo and the most advanced programme in AstraZeneca’s ADC scientific platform. Enhertu consists of a HER2 monoclonal antibody attached to a topoisomerase I inhibitor payload, an exatecan derivative, via a stable tetrapeptide-based cleavable linker.
Enhertu (5.4mg/kg) is approved in more than 30 countries for the treatment of adult patients with unresectable or metastatic HER2-positive breast cancer who have received a (or one or more) prior anti-HER2-based regimen either in the metastatic setting, or in the neoadjuvant or adjuvant setting and have developed disease recurrence during or within six months of completing therapy, based on the results from the DESTINY-Breast03 trial.
Enhertu (5.4mg/kg) is also approved in several countries for the treatment of adult patients with unresectable or metastatic HER2-positive breast cancer who have received two or more prior anti-HER2-based regimens based on the results from the DESTINY-Breast01 trial.
Enhertu (6.4mg/kg) is approved in several countries for the treatment of adult patients with locally advanced or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma who have received a prior trastuzumab-based regimen based on the results from the DESTINY-Gastric01 trial.
Enhertu development programme
A comprehensive development programme is underway globally, evaluating the efficacy and safety of Enhertu monotherapy across multiple HER2-targetable cancers, including breast, gastric, lung and colorectal cancers. Trials in combination with other anticancer treatments, such as immunotherapy, are also underway.
Regulatory applications for Enhertu are currently under review in China, Japan and several other countries for the treatment of adult patients with unresectable or metastatic HER2-positive breast cancer who have received a prior anti-HER2-based regimen based on the results from the DESTINY-Breast03 trial.
Enhertu is under review in Europe for the treatment of adult patients with unresectable or metastatic HER2-low (immunohistochemistry (IHC) 1+ or IHC 2+/ in-situ hybridisation (ISH)-negative) breast cancer who have received a prior systemic therapy in the metastatic setting or developed disease recurrence during or within six months of completing adjuvant chemotherapy, based on the results from the DESTINY-Breast04 trial. Patients with hormone receptor (HR) positive breast cancer must additionally have received or be ineligible for endocrine therapy.
Enhertu is also currently under review in the US for the treatment of adult patients with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumours have a HER2 (ERBB2) mutation and who have received a prior systemic therapy based on the results of the DESTINY-Lung01 trial, and in Europe for the treatment of adult patients with locally advanced or metastatic HER2-positive gastric or GEJ adenocarcinoma who have received a prior anti-HER2-based regimen based on the DESTINY-Gastric01 and DESTINY-Gastric02 trials.
Enhertu was granted Breakthrough Therapy Designation in the US for the treatment of adult patients with unresectable or metastatic HER2-low (IHC 1+ or IHC 2+/ISH-negative) breast cancer who have received a prior systemic therapy in the metastatic setting or developed disease recurrence during or within six months of completing adjuvant chemotherapy, based on the results of the DESTINY-Breast04 trial. Patients with HR-positive breast cancer should additionally have received or be ineligible for endocrine therapy.
Daiichi Sankyo collaboration
Daiichi Sankyo Company, Limited (TSE:4568) [referred to as Daiichi Sankyo] and AstraZeneca entered into a global collaboration to jointly develop and commercialise Enhertu (a HER2-directed ADC) in March 2019, and datopotamab deruxtecan (DS-1062; a TROP2-directed ADC) in July 2020, except in Japan where Daiichi Sankyo maintains exclusive rights. Daiichi Sankyo is responsible for manufacturing and supply of Enhertu and datopotamab deruxtecan.
AstraZeneca in breast cancer
Driven by a growing understanding of breast cancer biology, AstraZeneca is starting to challenge, and redefine, the current clinical paradigm for how breast cancer is classified and treated to deliver even more effective treatments to patients in need – with the bold ambition to one day eliminate breast cancer as a cause of death.
AstraZeneca has a comprehensive portfolio of approved and promising compounds in development that leverage different mechanisms of action to address the biologically diverse breast cancer tumour environment.
AstraZeneca aims to continue to transform outcomes for HR-positive breast cancer with foundational medicines Faslodex (fulvestrant) and Zoladex (goserelin) and the next-generation oral selective oestrogen receptor degrader (SERD) and potential new medicine camizestrant.
PARP inhibitor Lynparza (olaparib) is a targeted treatment option that has been studied in HER2-negative early and metastatic breast cancer patients with an inherited BRCA mutation. AstraZeneca with MSD (Merck & Co., Inc. in the US and Canada) continue to research Lynparza in metastatic breast cancer patients with an inherited BRCA mutation and are exploring new opportunities to treat these patients earlier in their disease.
Building on the initial approvals of Enhertu, a HER2-directed ADC, in previously treated HER2-positive metastatic breast cancer, AstraZeneca and Daiichi Sankyo are exploring its potential in earlier lines of treatment and in new breast cancer settings.
To bring much needed treatment options to patients with triple-negative breast cancer, an aggressive form of breast cancer, AstraZeneca is testing immunotherapy Imfinzi (durvalumab) in combination with other oncology medicines, including Lynparza and Enhertu, evaluating the potential of AKT kinase inhibitor, capivasertib, in combination with chemotherapy, and collaborating with Daiichi Sankyo to explore the potential of TROP2-directed ADC, datopotamab deruxtecan.
Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca.
AstraZeneca, Daiichi Sankyo Enhertu gets EU approval for earlier use in breast cancer subtype
Jul. 19, 2022 5:28 AM ET
AstraZeneca PLC (AZN), DSKYF, DSNKY
By: Ravikash, SA News Editor
- The European Commission (EC) approved AstraZeneca (NASDAQ:AZN) and Daiichi Sankyo's (OTCPK:DSKYF) (OTCPK:DSNKY) Enhertu to treat certain patients with breast cancer.
- https://seekingalpha.com/news/3857895-astrazeneca-daiichi-sankyo-enhertu-gets-eu-approval-for-earlier-use-in-breast-cancer-subtype
- https://seekingalpha.com/symbol/AZN
- https://seekingalpha.com/symbol/DSKYF
- https://www.enhertu.com/en
QULIPTA (atogepant)
July 18, 2022
AbbVie Submits Marketing Authorization Application to EMA for Atogepant for the Preventive Treatment of Migraine
- The submission is based on two pivotal Phase 3 studies evaluating atogepant in adult patients with episodic and chronic migraine
- If approved, atogepant would be the first daily oral calcitonin gene-related peptide (CGRP) receptor antagonist (gepant) for the prophylaxis of migraine in Europe
- AbbVie would become the only company with a portfolio of medicines to offer two treatments for those with chronic migraine, one oral and one injectable
NORTH CHICAGO, Ill., July 18, 2022 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced it has submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) for atogepant for the prophylaxis of migraine in adult patients who have at least four migraine days per month. The application is supported by the pivotal Phase 3 ADVANCE and PROGRESS studies evaluating the safety, efficacy, and tolerability of atogepant in adult patients with episodic migraine and chronic migraine, respectively.1,2
Migraine is a complex neurological disease and one of the leading causes of disability worldwide.3 It is highly prevalent, affecting more than 1 billion people worldwide,3 including an estimated 11.4 percent of the population in Europe.4 If approved, atogepant would be the first daily oral CGRP receptor antagonist for the prophylaxis of migraine for adult patients in Europe.
"Far too many people around the world are impacted from the debilitating challenges of migraine, which places a significant social and work-life burden for patients and care partners," said Michael Gold, M.D., therapeutic area head, neuroscience development, AbbVie. "At AbbVie, we are committed to advancing science to provide patients impacted by migraine with effective treatment options. If approved, atogepant will provide a prophylactic treatment option for adult migraine patients suffering for more than four days a month."
The pivotal, Phase 3, multicenter, randomized, double-blind, placebo-controlled, parallel-group ADVANCE trial evaluated the efficacy, safety, and tolerability of once daily (QD) oral atogepant for the prophylaxis of episodic migraine. The study met its primary endpoint of a statistically significant reduction in mean monthly migraine days across the 12-week treatment period compared to placebo. This was found across all active treatment arms of atogepant – 10 mg, 30 mg, and 60 mg QD doses. The adult patients enrolled met the International Classification of Headache Disorders (ICHD) criteria for a diagnosis of migraine with or without aura. The study also found that a greater proportion of atogepant-treated participants achieved at least a 50% reduction in mean monthly migraine days for all doses compared to placebo and met other key secondary endpoints.
The pivotal, Phase 3, global, randomized, double-blind, placebo-controlled, parallel-group PROGRESS study, evaluating the safety, efficacy, and tolerability of oral atogepant in adult patients for the prophylaxis of chronic migraine, met its primary endpoint of statistically significant reduction from baseline in mean monthly migraine days compared to placebo across the 12-week treatment period. The trial also demonstrated that treatment with atogepant 60 mg once daily (QD) and 30 mg daily (BID), resulted in statistically significant improvements in all secondary endpoints. This includes a key secondary endpoint that measured the proportion of patients that achieved at least a 50 percent reduction in mean monthly migraine days across the 12-week treatment period.
In both, the Phase 3 PROGRESS and Phase 3 ADVANCE studies, all doses were well tolerated, and the overall safety profiles were consistent with safety findings observed in previous studies for the prophylaxis of episodic migraine and chronic migraine populations. The most common adverse events were constipation and nausea.
The atogepant MAA will be reviewed by the Committee for Medicinal Products for Human Use, which will issue an opinion that will be valid for all member states of the European Union, as well as Iceland, Lichtenstein, Northern Ireland and Norway.
About Atogepant
Atogepant is an orally administered, CGRP receptor antagonist (gepant) specifically developed for the prophylaxis treatment of migraine. CGRP and its receptors are expressed in regions of the nervous system associated with migraine pathophysiology. Studies have shown that CGRP levels are elevated during migraine attacks and selective CGRP receptor antagonists confer clinical benefit in migraine.
About the Phase 3 ADVANCE Clinical Trial1
The pivotal Phase 3, multicenter, randomized, double-blind, placebo-controlled, parallel-group trial was designed to evaluate the efficacy, safety, and tolerability of oral atogepant for the prevention of migraine in those with 4 to 14 migraine days per month. A total of 910 patients were randomized to one of four treatment groups evaluating 10 mg, 30 mg, or 60 mg of atogepant once daily, or placebo. Efficacy analyses were based on the modified intent-to-treat (mITT) population of 873 patients.
The primary endpoint was change from baseline in mean monthly migraine days across the 12-week treatment period. All atogepant dose groups met the primary endpoint and demonstrated statistically significantly greater decreases in mean monthly migraine days compared to placebo. Patients treated in the 10 mg/30 mg/60 mg atogepant arms experienced a decrease of 3.69/3.86/4.2 days, respectively, all compared to patients in the placebo arm, who experienced a decrease of 2.48 days (all dose groups vs. placebo, p=<.0001).
A key secondary endpoint measured the proportion of patients that achieved at least a 50% reduction in mean monthly migraine days across the 12-week treatment period. The trial demonstrated that 55.6%/58.7%/60.8% of patients in the 10 mg/30 mg/60 mg atogepant arms, respectively, achieved at least a 50% reduction, compared to 29.0% of patients in the placebo arm (all dose groups vs. placebo, p=<.0001).
Additional secondary endpoints measured across the 12-week treatment period included change from baseline in mean monthly headache days, mean monthly acute-medication use days, and mean monthly performance of daily activities and physical impairment domain scores of the Activity Impairment in Migraine-Diary (AIM-D), and change from baseline in the Migraine-Specific Quality of Life Questionnaire (MSQ) Role Function-Restrictive domain score at week 12. The trial demonstrated that treatment with 30 mg and 60 mg doses resulted in statistically significant improvements in all secondary endpoints, while treatment with the 10 mg dose resulted in statistically significant improvements in four out of the six secondary endpoints.
No new safety risks were observed compared to the safety profile observed in the previous trial evaluating atogepant. Serious adverse events occurred in 0.9% of patients treated in the atogepant 10 mg arm and 0.9% of patients in the placebo arm. No patients in the atogepant 30 mg or 60 mg treatment arms experienced a serious adverse event. The most common adverse events reported with a frequency ≥ 5% in at least one atogepant treatment arm, and greater than placebo, were constipation (7.7%, 7.0% and 6.9% in the 10 mg/30 mg/60 mg atogepant arms, respectively vs. 0.5% for placebo), nausea (5.0%, 4.4% and 6.1% in the 10 mg/30 mg/60 mg atogepant arms, respectively vs. 1.8% for placebo), and upper respiratory tract infection (4.1%, 5.7% and 3.9% in the 10 mg/30 mg/60 mg atogepant arms, respectively vs. 4.5% for placebo). The majority of cases of constipation, nausea and upper respiratory tract infection were mild or moderate in severity and did not lead to discontinuation. There were no hepatic safety issues identified in this trial.
About the Phase 3 PROGRESS Clinical Trial2
The Phase 3 PROGRESS clinical trial evaluated the safety, tolerability and efficacy of oral atogepant for the prophylaxis treatment of chronic migraine. The patient population for the study included patients with a diagnosis of chronic migraine for at least one year, and ≥ to 15 headache days with eight migraine days in the 28 days prior to randomization. The primary endpoint measured the reduction from baseline in mean monthly migraine days compared to placebo, for both doses, including 60 mg once daily (QD) and 30 mg twice daily (BID), across a 12-week treatment period. The overall safety profile of the Phase 3 PROGRESS study was consistent with safety findings observed in previous studies in an episodic migraine population.
Key secondary endpoints for all regions included: Change from baseline in mean monthly headache days across the 12-week of treatment period (baseline is defined as the number of migraine days during the last 28 days prior to the randomization date); Change from baseline in mean monthly acute medication use days across the 12-week treatment period (baseline is defined as the number of migraine days during the last 28 days prior to the randomization date); Proportion of participants with at least a 50% reduction in mean monthly migraine days across the 12-week treatment period; and change from baseline in MSQ v2.1 Role Function-Restrictive domain score at Week 12. The MSQ v2.1 is a questionnaire designed to measure health-related quality of life impairments attributed to migraine in the past four weeks. It is divided into three domains, assessing how a patient's daily, social, and work activities are limited by migraine; how migraine prevents these activities; and assesses the emotional function related with migraine.
For a full listing of secondary endpoints across all regions, please go to www.clinicaltrials.gov (NCT03855137).
About AbbVie
AbbVie's mission is to discover and deliver innovative medicines that solve serious health issues today and address the medical challenges of tomorrow. We strive to have a remarkable impact on people's lives across several key therapeutic areas: immunology, oncology, neuroscience, eye care, virology, women's health and gastroenterology, in addition to products and services across its Allergan Aesthetics portfolio. For more information about AbbVie, please visit us at www.abbvie.com.
Follow @AbbVie on Twitter, Facebook, Instagram, YouTube, and LinkedIn
SOURCE AbbVie
AbbVie files for EU approval of atogepant to prevent migraine
Jul. 18, 2022 6:13 AM ET AbbVie Inc. (ABBV)
By: Ravikash, SA News Editor
- AbbVie (NYSE:ABBV) submitted an application to the European Medicines Agency (EMA) seeking approval of atogepant to prevent migraine in adult patients who have at least four migraine days per month.
- https://seekingalpha.com/news/3857427-abbvie-files-for-eu-approval-of-atogepant-to-prevent-migraine
- https://seekingalpha.com/symbol/ABBV
- https://clinicaltrials.gov/ct2/show/NCT03777059
- https://www.qulipta.com/
Hemlibra® (emicizumab)
New data from phase III HAVEN 6 study reinforce favourable safety and efficacy profile of Roche’s Hemlibra in people with moderate or mild haemophilia A July 11, 2022
- Hemlibra continues to demonstrate clinically meaningful bleed control, with 66.7% of participants with moderate or mild haemophilia A experiencing zero treated bleeds at 55.6 weeks median follow-up [1]
- New data also reinforce Hemlibra’s favourable safety profile, with no new safety signals observed [1]
- There is limited information and treatment guidance on moderate and mild haemophilia A, which can lead to delayed or missed diagnoses of bleeding episodes [2]
- Hemlibra is approved to treat people of all ages with haemophilia A with factor VIII inhibitors in more than 110 countries and for people of all ages without factor VIII inhibitors in more than 95 countries
Basel, 11 July 2022 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced results from the primary analysis of the phase III HAVEN 6 study, which show that Hemlibra® (emicizumab) continued to demonstrate a favourable safety profile and effective bleed control in people with moderate or mild haemophilia A, without factor VIII inhibitors.[1] The data will be presented at the 30th International Society on Thrombosis and Haemostasis (ISTH) Annual Congress, on 11 July 2022, in London, United Kingdom, and are planned to support a submission to the European Medicines Agency to update the label for Hemlibra to include non-severe haemophilia A patients.
“We are proud that Hemlibra continues to redefine the standard of care for more people living with haemophilia A,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. “The data presented at ISTH this year underscore Roche’s commitment to addressing gaps in care for haemophilia A, thereby ensuring that broader populations can potentially benefit from Hemlibra.”
In addition to HAVEN 6, data from the CHESS II (Cost of Haemophilia across Europe: a Socioeconomic Survey-II) and CHESS PAEDs studies will also be presented at ISTH 2022. These data show most adults with moderate or mild haemophilia A and more than half of children with moderate haemophilia A may not receive preventative treatments. This can result in worsened clinical burden, as more than 30% of adults and approximately 40% of children with moderate haemophilia A who were not taking preventative treatment in the study experienced at least three bleeds a year.[3]
HAVEN 6 is a phase III, multicentre, open-label, single-arm study evaluating the safety, efficacy, pharmacokinetics and pharmacodynamics of Hemlibra in people with moderate or mild haemophilia A without factor VIII inhibitors. The primary analysis included data from 72 participants (69 men and three women) who warranted prophylaxis; 21 had mild haemophilia A without factor VIII inhibitors and 51 had moderate haemophilia A without factor VIII inhibitors at a median follow-up of 55.6 weeks. At baseline, 37 participants were receiving factor VIII prophylactic treatment and 24 had target joints.[1]
The data show that Hemlibra maintained low treated bleed rates across the study period, with 66.7% of participants experiencing no bleeds that required treatment, 81.9% experiencing no spontaneous bleeds that required treatment, and 88.9% experiencing no joint bleeds that required treatment.[1] Model-based annualised bleed rates (ABR) remained low throughout the evaluation period at 0.9 (95% CI: 0.55-1.52).
The results also show that Hemlibra’s safety profile was consistent with findings across various subpopulations of people with haemophilia A, from previous HAVEN and STASEY studies, with no new safety signals observed. The most common adverse event (AE) related to treatment occurring in 10% or more people in the HAVEN 6 study was local injection site reactions (ISRs) (16.7%). Fifteen people (20.8%) reported a Hemlibra-related AE, of which the majority were local ISRs. One participant experienced a grade one thromboembolic event unrelated to Hemlibra. There were no deaths or cases of thrombotic microangiopathy, reinforcing Hemlibra’s favourable safety profile.[1]
Hemlibra is approved to treat people with haemophilia A with factor VIII inhibitors in more than 110 countries worldwide and for people without factor VIII inhibitors in more than 95 countries worldwide, including the US and Japan for all severities of haemophilia A, and the EU for only severe haemophilia A. It has been studied in one of the largest clinical trial programmes in people with haemophilia A with and without factor VIII inhibitors, including eight phase III studies.
About Hemlibra® (emicizumab)
Hemlibra is a bispecific factor IXa- and factor X-directed antibody. It is designed to bring together factor IXa and factor X, proteins involved in the natural coagulation cascade, and restore the blood clotting process for people with haemophilia A. Hemlibra is a prophylactic (preventative) treatment that can be administered by an injection of a ready-to-use solution under the skin (subcutaneously) once-weekly, every two weeks, or every four weeks (after an initial once-weekly dose for the first four weeks). Hemlibra was created by Chugai Pharmaceutical Co., Ltd. and is being co-developed globally by Chugai, Roche and Genentech. It is marketed in the United States by Genentech as Hemlibra (emicizumab-kxwh), with kxwh as the suffix designated in accordance with Nonproprietary Naming of Biological Products Guidance for Industry issued by the U.S. Food and Drug Administration.
https://www.hemlibra.com/patient.html
About Roche
Founded in 1896 in Basel, Switzerland, as one of the first industrial manufacturers of branded medicines, Roche has grown into the world’s largest biotechnology company and the global leader in in-vitro diagnostics. The company pursues scientific excellence to discover and develop medicines and diagnostics for improving and saving the lives of people around the world. We are a pioneer in personalised healthcare and want to further transform how healthcare is delivered to have an even greater impact. To provide the best care for each person we partner with many stakeholders and combine our strengths in Diagnostics and Pharma with data insights from the clinical practice.
In recognizing our endeavor to pursue a long-term perspective in all we do, Roche has been named one of the most sustainable companies in the pharmaceuticals industry by the Dow Jones Sustainability Indices for the thirteenth consecutive year. This distinction also reflects our efforts to improve access to healthcare together with local partners in every country we work.
Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan.
For more information, please visit www.roche.com.
All trademarks used or mentioned in this release are protected by law.
Roche's Hemlibra helps control bleeding in mild/moderate hemophilia A in trial
Jul. 12, 2022 4:51 AM ET
Roche Holding AG (RHHBY), RHHBF
By: Ravikash, SA News Editor
- Roche (OTCQX:RHHBY) (OTCQX:RHHBF) on July 11 said that a primary analysis of a phase 3 trial showed that Hemlibra continued to show safety and effective bleed control in people with moderate or mild hemophilia A without factor VIII inhibitors.
- https://seekingalpha.com/news/3855911-roches-hemlibra-helps-control-bleeding-in-mildmoderate-hemophilia-a-over-4-years-in-trial
- https://seekingalpha.com/symbol/RHHBY
- https://seekingalpha.com/symbol/RHHBF
- https://www.hemlibra.com/patient.html
Imfinzi (durvalumab) plus chemotherapy
Imfinzi plus chemotherapy significantly improved pathologic complete response in AEGEAN Phase III trial in resectable non-small cell lung cancer
PUBLISHED30 June 2022
30 June 2022 07:00 BST
Trial will continue to assess additional primary endpoint of event-free survival
Positive high-level results from a planned interim analysis of the AEGEAN Phase III trial showed treatment with AstraZeneca’s Imfinzi (durvalumab) in combination with neoadjuvant chemotherapy before surgery demonstrated a statistically significant and meaningful improvement in pathologic complete response (pCR) compared to neoadjuvant chemotherapy alone for patients with resectable non-small cell lung cancer (NSCLC).
A statistically significant improvement in major pathologic response (MPR) was also observed. The trial will continue as planned to assess the additional primary endpoint of event-free survival (EFS) to which the Company, investigators and participants remain blinded.
The safety and tolerability of adding Imfinzi to neoadjuvant chemotherapy was consistent with the known profile for this combination and did not decrease the number of patients able to undergo successful surgery versus chemotherapy alone.
Up to 30% of all patients globally with NSCLC are diagnosed early enough to have surgery with curative intent.1-3 However, only around 56-65% of patients with Stage II disease will survive for five-years. This decreases to 24-41% for patients with Stage III disease.4
Susan Galbraith, Executive Vice President, Oncology R&D, said: ‘‘Treating resectable lung cancer early provides the best chance for a cure, yet lung cancer will still recur within five years for the majority of patients despite chemotherapy and successful surgery. Engaging the immune response with Imfinzi both before and after surgery is an exciting new strategy, and we hope these early findings from AEGEAN will lead to improved survival for lung cancer patients in this potentially curative setting.”
These pCR data will be shared with global health authorities and presented at a forthcoming medical meeting when EFS results are available.
AstraZeneca has several ongoing registrational trials focused on testing Imfinzi in earlier stages of lung cancer, including in resectable NSCLC (ADJUVANT BR.31) and unresectable NSCLC (PACIFIC-2, 4, 5, 8 and 9), and in limited-stage small cell lung cancer (SCLC) (ADRIATIC).
Imfinzi is approved in the curative-intent setting of unresectable Stage III NSCLC in patients whose disease has not progressed after chemoradiotherapy in the US, Japan, China, across the EU and many other countries, and is the global standard of care in this setting based on the PACIFIC Phase III trial. Imfinzi is also approved in the US, EU, Japan, China and many other countries around the world for the treatment of extensive-stage SCLC based on the CASPIAN Phase III trial.
AEGEAN
AEGEAN is a randomised, double-blind, multi-centre, global Phase III trial evaluating Imfinzi as perioperative treatment for patients with resectable Stage IIA-IIIB (tumours greater than or equal to 4cm or node positive) NSCLC with no EGFR or ALK genomic tumour aberrations, irrespective of PD-L1 expression. Perioperative therapy includes treatment before and after surgery, also known as neoadjuvant/adjuvant therapy. In the trial, 802 patients were randomised to receive a 1500mg fixed dose of Imfinzi every three weeks plus chemotherapy or placebo plus chemotherapy for four cycles prior to surgery, followed by Imfinzi or placebo every four weeks (for up to 12 cycles) after surgery.
In the AEGEAN trial, the primary endpoints are pCR, defined as no viable tumour following neoadjuvant therapy, and EFS, defined as the time from randomisation to an event like tumour recurrence or progression. At this interim analysis EFS was not assessed. Key secondary endpoints are MPR, defined as residual viable tumour of less than or equal to ten percent following neoadjuvant therapy, disease-free survival, overall survival, safety and quality of life. The trial is being conducted across 264 centres in more than 25 countries including in the US, Canada, Europe, South America and Asia.
Imfinzi
Imfinzi (durvalumab) is a human monoclonal antibody that binds to the PD-L1 protein and blocks the interaction of PD-L1 with the PD-1 and CD80 proteins, countering the tumour’s immune-evading tactics and releasing the inhibition of immune responses.
As well as global approvals in lung cancer, Imfinzi is approved for previously treated patients with advanced bladder cancer in several countries.
Since the first approval in May 2017, more than 100,000 patients have been treated with Imfinzi.
As part of a broad development programme, Imfinzi is being tested as a single treatment and in combinations with other anti-cancer treatments for patients with SCLC, NSCLC, bladder cancer, several GI cancers, ovarian cancer, endometrial cancer and other solid tumours.
In the past year, Imfinzi combinations have resulted in positive Phase III trials in multiple additional cancer settings including; unresectable advanced liver cancer (HIMALAYA), biliary tract cancer (TOPAZ-1) and metastatic NSCLC (POSEIDON) and the data are under review with global health authorities.
AstraZeneca
AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialisation of prescription medicines in Oncology, Rare Diseases, and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca.
AstraZeneca Imfinzi/chemo combo shows efficacy in lung cancer patients before surgery in trial
Jun. 30, 2022 5:51 AM ET
By: Ravikash, SA News Editor
AstraZeneca (NASDAQ:AZN) said interim data from a phase 3 trial of Imfinzi/chemo combo before surgery showed improvement in absence of detectable disease in patients with resectable non-small cell lung cancer (NSCLC).
https://seekingalpha.com/symbol/AZN
GAVRETO® (pralsetinib)
CStone Announces the NDA Approval of GAVRETO® (pralsetinib) for the Treatment of RET Fusion-Positive treatment-naïve (first-line) and pretreated Non-Small Cell Lung Cancer (NSCLC) in Hong Kong, China
Times：2022.07.15 Author：CStone
- GAVRETO is the first highly selective rearranged during transfection (RET) inhibitor approved in Hong Kong, China for the treatment of RET fusion-positive metastatic non-small cell lung cancer
- GAVRETO is CStone’s second therapy approved in Hong Kong, China
- GAVRETO is CStone’s ninth NDA approval in the Greater China region
Suzhou, China, July 15, 2022 — CStone Pharmaceuticals (“CStone”, HKEX: 2616), a leading biopharmaceutical company focused on research, development, and commercialization of innovative immuno-oncology therapies and precision medicines, announced today that the new drug application (NDA) for GAVRETO® (pralsetinib) has been approved in Hong Kong, China for the treatment of adult patients with rearranged during transfection (RET) fusion-positive metastatic non-small cell lung cancer (NSCLC).
GAVRETO is a potent and selective RET inhibitor discovered by CStone’s partner Blueprint Medicines. CStone has an exclusive collaboration and license agreement with Blueprint Medicines for the development and commercialization of GAVRETO in Greater China, which encompasses Mainland China, Hong Kong, Macau and Taiwan.
Dr. Frank Jiang, Chief Executive Officer of CStone, said, “We are very glad about the NDA approval of GAVRETO in Hong Kong, China, which came only four months after its NDA acceptance. This came on the heels of the NDA approval of our first-in-class precision therapy AYVAKIT® (avapritinib) in this city. GAVRETO has already been approved in Mainland China, and we are very excited to bring forward this innovative therapy to more patients in the Greater China region. CStone is committed to providing high-quality innovative medicines for patients worldwide. Moving forward, we will continue our efforts to accelerate the development of innovative drugs to fulfill the unmet medical needs of more cancer patients.”
The NDA approval of GAVRETO in Hong Kong, China is based on results from the global phase 1/2 ARROW study. This trial is designed to evaluate the safety, tolerability, and efficacy of GAVRETO in patients with RET-fusion positive NSCLC, RET-mutant medullary thyroid cancer (MTC), and other advanced solid tumors with RET fusions. Results from the ARROW trial in global patients with advanced RET fusion-positive NSCLC were presented at the American Society of Clinical Oncology (ASCO) Annual Meeting in June 2021. As of a data cutoff date of November 6, 2020, GAVRETO showed durable clinical benefits in patients with RET fusion-positive NSCLC who had measurable disease at baseline and received a starting dose of 400 mg once daily.
- In 68 treatment-naïve patients, the overall response rate (ORR) was 79 percent (95% CI: 68%, 88%). The complete response (CR) rate was 6 percent, 10 percent of patients had complete regression of target tumors, and 74 percent of patients had a partial response (PR). The median duration of response (DOR) was not reached (95% CI: 9.0 months, not reached).
- In 126 patients who previously received platinum-based chemotherapy, the ORR was 62 percent (95% CI: 53%, 70%). The CR rate was 4 percent, 12 percent of patients had complete regression of target tumors, and 58 percent of patients had a PR. The median DOR was 22.3 months (95% CI: 15.1 months, not reached).
- As of the data cutoff date, a total of 471 patients were enrolled across tumor types. The most common treatment-related adverse events (AEs) reported by investigators were neutropenia, increased aspartate aminotransferase, anemia, decreased white blood cell count, increased alanine aminotransferase, hypertension, constipation and asthenia.
About GAVRETO®(pralsetinib)
GAVRETO is a once-daily oral targeted therapy, approved by the National Medical Products Administration (NMPA) of China for the treatment of adults with locally advanced or metastatic rearranged during transfection (RET) fusion-positive NSCLC after platinum-based chemotherapy, and for the treatment of adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant MTC who requires systemic therapy, and adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who requires systemic therapy and radioactive iodine-refractory (if radioactive iodine treatment is appropriate). GAVRETO has been approved in Hong Kong, China for the treatment of adult patients with RET fusion-positive metastatic NSCLC.
GAVRETO is approved by the U.S. Food and Drug Administration (FDA) for the treatment of three indications: adult patients with metastatic RET fusion-positive NSCLC as detected by an FDA approved test, adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant MTC, and adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate). These indications are approved under accelerated approval based on ORR and DOR. Continued approval for these indications may be contingent upon verification and description of clinical benefit in confirmatory trials.
The European Commission (EC) has granted conditional marketing authorization for GAVRETO as a monotherapy for the treatment of adult patients with RET fusion-positive advanced NSCLC not previously treated with a RET inhibitor.
GAVRETO is not approved for the treatment of any other indication in China, the U.S. or Europe.
GAVRETO is designed to selectively and potently target oncogenic RET alterations, including secondary RET mutations predicted to drive resistance to treatment. In preclinical studies, pralsetinib inhibited RET at lower concentrations than other pharmacologically relevant kinases, including VEGFR2, FGFR2, and JAK2.
Blueprint Medicines and Roche are co-developing GAVRETO globally (excluding Greater China) for the treatment of patients with RET-altered NSCLC, thyroid cancer, and other solid tumors. Blueprint Medicines and Genentech, a member of the Roche Group, are co-commercializing GAVRETO in the U.S., and Roche has exclusive commercialization rights for GAVRETO outside of the U.S. (excluding Greater China).
About CStone
CStone (HKEX: 2616) is a biopharmaceutical company focused on researching, developing, and commercializing innovative immuno-oncology and precision medicines to address the unmet medical needs of cancer patients in China and worldwide. Established in 2015, CStone has assembled a world-class management team with extensive experience in innovative drug development, clinical research, and commercialization. The company has built an oncology-focused pipeline of 15 drug candidates with a strategic emphasis on immuno-oncology combination therapies. Currently, CStone has received nine NDA approvals for four drugs. Multiple late-stage drug candidates are now under pivotal clinical trials or registration. CStone's vision is to become globally recognized as a world-renowned biopharmaceutical company by bringing innovative oncology therapies to cancer patients worldwide.
For more information about CStone, please visit: www.cstonepharma.com.
Blueprint Medicines, AYVAKIT, GAVRETO and associated logos are trademarks of Blueprint Medicines Corporation.
Disclaimer: only for communication and scientific use by medical and health professionals.
Roche, Blueprint, CStone's Gavreto gets approval in Hong Kong for lung cancer subtype
Jul. 15, 2022 5:56 AM ET
CStone Pharmaceuticals (CSPHF), RHHBY, RHHBF, BPMC
By: Ravikash, SA News Editor
- Blueprint Medicines (NASDAQ:BPMC), CStone Pharmaceuticals (OTCPK:CSPHF) and Roche's (OTCQX:RHHBY) (OTCQX:RHHBF) Gavreto was approved in Hong Kong for certain patients with non-small cell lung cancer (NSCLC).
- https://seekingalpha.com/news/3857100-roche-blueprint-cstones-gavreto-gets-approval-in-hong-kong-for-lung-cancer-subtype
- https://seekingalpha.com/symbol/BPMC
- https://seekingalpha.com/symbol/RHHBY
- https://seekingalpha.com/symbol/CSPHF
- https://www.gavreto.com/
VAZKEPA® (ICOSAPENT ETHYL)
NICE ISSUES FINAL GUIDANCE FOR REIMBURSEMENT MAKING VAZKEPA® (ICOSAPENT ETHYL) AVAILABLE ACROSS THE NHS IN ENGLAND & WALES
July 13, 2022 at 8:00 AM EDTPDF Version
-- Newly published final guidance by the National Institute for Health and Care Excellence (NICE) in the UK confirms its prior draft recommendation for the use of VAZKEPA® (icosapent ethyl) in England and Wales to reduce the risk of cardiovascular (CV) events in adult statin-treated patients at high CV risk who have elevated triglycerides (≥150 mg/dL [≥ 1.7 mmol/L]), controlled LDL-C between 1.04 mmol/L - 2.60 mmol/L and established cardiovascular disease (eCVD).1,2--
DUBLIN, Ireland and BRIDGEWATER, N.J., July 13, 2022 (GLOBE NEWSWIRE) -- Amarin Corporation plc (NASDAQ: AMRN) today announced that NICE has issued its final guidance recommending VAZKEPA® (icosapent ethyl) for reimbursement and use across the National Health Service (NHS) in England and Wales to help reduce the risk of major CV events in high-risk statin-treated patients with eCVD, at a price of £144.21 per 120 soft capsules (i.e. 30 day supply; the equivalent of approximately 171 EUR or 172 USD*).
This announcement marks a major milestone for Amarin globally and in the UK, as following final guidance, all local NHS formularies in England and Wales will need to make VAZKEPA available within 90 and 60 days, respectively. Today’s final guidance also further supports the successful execution of Amarin’s European growth strategy, and the Company’s efforts to unlock the multi-billion-dollar revenue opportunities for the product outside of the U.S.**
Karim Mikhail, president and chief executive officer of Amarin said, “Receiving this final guidance from NICE is a significant moment, as it is another important step in our international expansion. Our teams in Europe are working incredibly hard to ensure a successful launch of VAZKEPA, so we can help transform the lives of CV patients across the region and move closer to realizing our bold vision of reaching the day when heart disease is no longer a leading cause of death.”
The publication of the final guidance supports the growing recognition of VAZKEPA’s clinical benefits. It is the last step in the NICE Health Technology Appraisal (HTA) process, used to assess the clinical benefits and cost-effectiveness of medicines and treatments in England to ensure the NHS uses its resources fairly and cost-effectively. Based on the collaborative relationship between the Welsh Government and the All-Wales Medicines Strategy Group (AWMSG), the final NICE guidance will also be implemented across the NHS in Wales, in line with the devolved powers of the Welsh Assembly.
Commenting on today’s news, Laurent Abuaf, senior vice president and president, Amarin Europe said, “We have a once in a generation opportunity to transform the lives of cardiovascular patients across Europe, and today’s announcement regarding NICE’s final guidance will help us realize that mission in one of our key markets. Following the successful completion of the HTA assessment in the UK, and the positive reimbursement guidance, our local teams in every country in Europe will be inspired by how the UK will be prioritizing access to local health economies. Our teams in the UK will of course work tirelessly to make this medicine available across the whole territory in the coming months.”
Off the back of this success, Amarin continues to drive forward reimbursement discussions in other major European markets and remains on track to receive pricing decisions in up to eight countries with plans to launch VAZKEPA in up to six European countries this year.
*Based on exchange rate of EUR and USD as of the date of this release.
** U.S. Dollar
About Amarin®
Amarin is an innovative pharmaceutical company leading a new paradigm in cardiovascular disease management. From our scientific research foundation to our focus on clinical trials, and now our commercial expansion, we are evolving and growing rapidly. Amarin has offices in Bridgewater, New Jersey in the United States, Dublin in Ireland, Zug in Switzerland, and other countries in Europe as well as commercial partners and suppliers around the world. We are committed to rethinking cardiovascular risk through the advancement of scientific understanding of the impact on society of significant residual risk that exists beyond traditional therapies, such as statins for cholesterol management.
About VAZKEPA® (icosapent ethyl) Capsules
VAZKEPA capsules are the first prescription treatment comprised solely of the active ingredient, icosapent ethyl, a highly purified form of eicosapentaenoic acid. Since launch, icosapent ethyl has been prescribed more than 18 million times globally. In addition to the United States, icosapent ethyl is approved and sold in Canada, Lebanon and the United Arab Emirates under the brand name VASCEPA. In March 2021, marketing authorization was granted to icosapent ethyl in the European Union under the brand name VAZKEPA to reduce the risk of cardiovascular events in adult statin-treated patients at high cardiovascular risk with elevated triglycerides (≥ 150 mg/dL [≥ 1.7 mmol/L]) and established cardiovascular disease or diabetes and at least one other cardiovascular risk factor3. In April 2021 marketing authorization for VAZKEPA (icosapent ethyl) was granted in Great Britain (applying to England, Scotland and Wales). VAZKEPA (icosapent ethyl) is currently approved and sold in Europe in Germany, Sweden, Denmark and the UK.
EU Product Information
VAZKEPA® SOFT CAPSULES
This medicine is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions.
Indication: Vazkepa is indicated to reduce the risk of cardiovascular events in adult statin-treated patients at high cardiovascular risk with elevated triglycerides (≥150 mg/dL; ≥ 1.7 mmol/L) and either: established cardiovascular disease, or diabetes and at least one other cardiovascular risk factor.
Further information about the Summary of Product Characteristics (SmPC) for VAZKEPA® in Europe, can be found here.
Globally, prescribing information varies; please refer to the individual country product label for complete information.
Amarin's Vazkepa medicine gets final guidance from UK body for reimbursement and use
Jul. 13, 2022 9:46 AM ETAmarin Corporation plc (AMRN)By: Anuron Mitra, SA News Editor1 Comment
- Amarin (NASDAQ:AMRN) on Wednesday said UK's National Institute for Health and Care Excellence (NICE) had issued its final guidance recommending the company's Vazkepa medicine for reimbursement and use across the national health service in England and Wales.
- https://seekingalpha.com/news/3856411-amarins-vazkepa-medicine-gets-final-guidance-from-uk-body-for-reimbursement-and-use
- https://seekingalpha.com/symbol/AMRN
- https://www.vascepa.com/
- https://amarincorp.com/
biotecMAX™ Feb/Mar/APR/May/June 2022 Insight
OPDIVO® (nivolumab)
Health Canada Approves OPDIVO® (nivolumab) as Monotherapy for the Adjuvant treatment of Adults with Urothelial Carcinoma (UC) at High Risk of Recurrence after Undergoing Radical Resection of UC
Jun. 28, 2022 11:25 AM ETBristol-Myers Squibb Company (BMY)
First adjuvant Immunotherapy for patients at high risk of disease recurrence
MONTREAL, June 28, 2022 /CNW/ - Today, Bristol Myers Squibb Canada (BMS) announced Health Canada has issued a Notice of Compliance with Conditions (NOC/c) for OPDIVO®, as a monotherapy for the adjuvant treatment of adult patients with urothelial carcinoma (UC) who are at high risk of recurrence after undergoing radical resection of UC.i OPDIVO® is the first immuno-oncology treatment to bring benefit in the adjuvant setting of UC and represents a potential new standard of care for patients at high risk of disease recurrence.ii Unlike traditional cancer therapies that target the tumour directly, immuno-oncology activates the body's own immune system to help recognize and attack cancer cells.iii
"For years, patients with muscle-invasive urothelial carcinoma (MIUC) have lived with the unfortunate reality that, despite being diagnosed early enough to have their cancer removed, more than fifty percent face disease recurrence, with few safe and effective treatment options available to improve outcomes in these patients," said Dr. Wassim Kassouf, Professor, Department of Surgery (Urologic Oncology), McGill University. "The approval of OPDIVO® is particularly important because clinicians now have an immunotherapy option to offer certain patients after surgery, that may reduce the risk of disease recurrence. This approval has the potential to significantly impact the way we treat MIUC in Canada."
Clinical Data for Approval
The Health Canada NOC/c was based on CheckMate-274 which is a Phase 3, randomized, double-blind, placebo-controlled, multi-center trial evaluating OPDIVO® as an adjuvant treatment in patients who had undergone radical resection of urothelial carcinoma (UC) originating in the bladder or upper urinary tract and were at high risk of recurrence.
Treatment was provided up to one year, or until disease recurrence, toxicity, or withdrawal of consent occurred. The two primary endpoints of the trial were disease-free survival (DFS) among all the patients (intention-to-treat population) and among patients with a tumor programmed death ligand 1 (PD-L1) expression level of 1% or more. The key secondary endpoints include non-urothelial tract recurrence-free survival (NUTRFS), disease-specific survival (DSS) and overall survival (OS).i The trial met its primary endpoint of disease-free survival demonstrating that adjuvant OPDIVO® offers patients a chance to delay or potentially prevent disease recurrence in all randomized patients (intention-to-treat population) and those with a tumor PD-L1 expression level of 1% or more. A positive association was observed between tumor PD-L1 expression and the magnitude of the treatment benefit. An improvement in overall survival has not yet been established.
About Bristol Myers Squibb Canada Co.
Bristol Myers Squibb Canada Co. is an indirect wholly-owned subsidiary of Bristol Myers Squibb Company, a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. Bristol Myers Squibb Canada Co. employs close to 300 people across the country. For more information, please visit https://www.bms.com/ca/en.
About Bristol Myers Squibb
Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.
https://seekingalpha.com/symbol/BMY
Tislelizumab (BGB-A317)
BeiGene Announces Late-Breaking Data at ESMO GI Showing Overall Survival Benefit for Tislelizumab Plus Chemotherapy in First-Line Advanced or Metastatic Esophageal Squamous Cell Carcinoma
Jun 30, 2022 4:00 AM
- PD-1 inhibitor tislelizumab plus chemotherapy demonstrated a statistically significant and clinically meaningful survival benefit, extending survival by more than six months compared to chemotherapy alone
- Incidence of most common treatment-related adverse events similar for both arms of the study, with no new safety signal identified for tislelizumab
CAMBRIDGE, Mass. & BASEL, Switzerland & BEIJING--(BUSINESS WIRE)-- BeiGene (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global, science-driven biotechnology company focused on developing innovative and affordable medicines to improve treatment outcomes and access for patients worldwide, today announced new data from RATIONALE 306, a global Phase 3 trial evaluating tislelizumab plus chemotherapy in adult patients with advanced or metastatic esophageal squamous cell carcinoma (ESCC) without prior systemic treatment for advanced disease. Study results presented today as a late-breaking oral presentation at the 2022 European Society for Medical Oncology (ESMO) World Congress on Gastrointestinal Cancer (Abstract #LBA-1) showed a statistically significant and clinically meaningful improvement in overall survival (OS) for patients receiving tislelizumab in combination with chemotherapy with a median OS of 17.2 months [95% CI: 15.8,20.1] versus 10.6 months [95% CI: 9.3,12.1] for those receiving chemotherapy plus placebo. The combination of tislelizumab with chemotherapy reduced the risk of death by 34% (HR=0.66 [95% CI: 0.54,0.80, p<0.0001]) compared to chemotherapy plus placebo.
“We are encouraged by the consistent, clinically meaningful benefit seen with tislelizumab and chemotherapy in key endpoints measuring efficacy and durability of response and across pre-specified subgroups in this 1L ESCC treatment setting,” said Mark Lanasa, M.D., Chief Medical Officer, Solid Tumors at BeiGene. “We sincerely appreciate the patients with ESCC from across the world who chose to participate in this study as we search for treatment options for this challenging condition.”
The OS benefit for tislelizumab plus chemotherapy was observed regardless of baseline PD-L1 expression. The median OS for patients with PD-L1 score ≥10% was 16.6 months [(95% CI: 15.3,24.4] in the tislelizumab plus chemotherapy group versus 10.0 months [95% CI: 8.6,13.0] for patients receiving chemotherapy plus placebo (HR=0.62; 95% CI, 0.44,0.86, p=0.0020). Analysis of patients with a PD-L1 score <10% showed a median OS of 16.7 months [95% CI: 13.0,20.1] for tislelizumab plus chemotherapy versus 10.4 months [95% CI: 9.1,13.0]; (HR=0.72 [95% CI: 0.55,0.94]) for chemotherapy plus placebo. This survival benefit was consistent across all other pre-specified subgroups, including race, region, and choice of chemotherapy.
“ESCC represents the majority of esophageal cancer worldwide, but unfortunately chemotherapy by itself provides a median survival in the range of only a year, so the survival benefit seen when tislelizumab was added to chemotherapy in our study is compelling” said Harry Yoon, MD, Associate Professor of Oncology and Chair of the Gastroesophageal Cancer Disease Group at Mayo Clinic in Rochester, Minnesota. “Additionally, it is encouraging to see a familiar safety and tolerability profile for the combination consistent with those established for chemotherapy in the community.”
Progression-free survival was significantly improved for the tislelizumab plus chemotherapy (7.3 months) group compared to chemotherapy alone (5.6 months) (HR=0.62 [95% CI: 0.52,0.75, p<0.0001]), Additional benefit in overall response (ORR) was seen with tislelizumab and chemotherapy compared to chemotherapy [ORR 63.5% vs 42.4%; p<0.0001) and the median duration of response was 7.1 months [95% CI: 6.1,8.1] for tislelizumab plus chemotherapy versus 5.7 months [95% CI: 4.4,7.1] chemotherapy alone.
The incidence of treatment-related adverse events (TRAEs) was similar in both arms; the most commonly reported TRAEs (≥ 20%) were anemia, decreased neutrophil count, decreased white blood cell count, decreased appetite, nausea and peripheral sensory neuropathy.
Tislelizumab is currently under review by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for advanced or metastatic ESCC after prior chemotherapy. The EMA is also reviewing tislelizumab for advanced or metastatic non-small cell lung cancer (NSCLC) after prior chemotherapy, and in combination with chemotherapy for previously untreated advanced or metastatic NSCLC. In January 2021, BeiGene announced a collaboration with Novartis to accelerate the clinical development and marketing of tislelizumab in North America, Europe, and Japan.
Tislelizumab is approved by the China National Medical Products Administration (NMPA) as a treatment for nine indications, including a recent approval for use in patients with locally advanced or metastatic ESCC who have disease progression or are intolerant to first-line standard chemotherapy. Tislelizumab is not approved for use outside of China.
About RATIONALE 306
RATIONALE 306 (NCT03783442) is a randomized, placebo-controlled, double-blind, global Phase 3 study to evaluate the efficacy and safety of tislelizumab in combination with chemotherapy as a first-line treatment in patients with advanced or metastatic ESCC. The primary endpoint of the trial is overall survival (OS). Secondary endpoints include progression free survival, overall response rate, duration of response per RECIST v1.1, and OS in patients with PD-L1 score ≥10%, as well as health-related quality of life measures and safety.
The trial enrolled 649 patients at research centers across Asia-Pacific, Europe, and North America. Patients were randomized 1:1 to receive either tislelizumab plus chemotherapy or placebo plus chemotherapy.
About Tislelizumab
Tislelizumab is a humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to Fc-gamma (Fcγ) receptors on macrophages, helping to aid the body’s immune cells to detect and fight tumors. In pre-clinical studies, binding to Fcγ receptors on macrophages has been shown to compromise the anti-tumor activity of PD-1 antibodies through activation of antibody-dependent macrophage-mediated killing of T effector cells.
Tislelizumab is the first drug from BeiGene’s immuno-oncology biologics program and is being developed internationally as a monotherapy and in combination with other therapies for the treatment of a broad array of both solid tumor and hematologic cancers.
BeiGene has initiated or completed more than 20 potentially registration-enabling clinical trials in 35 countries and regions, including 17 Phase 3 trials and four pivotal Phase 2 trials. More information on the clinical trial program for tislelizumab can be found at: https://www.beigene.com/en-us/science-and-product-portfolio/pipeli
About BeiGene
BeiGene is a global, science-driven biotechnology company focused on developing innovative and affordable medicines to improve treatment outcomes and access for patients worldwide. With a broad portfolio of more than 40 clinical candidates, we are expediting development of our diverse pipeline of novel therapeutics through our own capabilities and collaborations. We are committed to radically improving access to medicines for two billion more people by 2030. BeiGene has a growing global team of over 8,000 colleagues across five continents. To learn more about BeiGene, please visit www.beigene.com and follow us on Twitter at @BeiGeneGlobal.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220630005285/en/
Source: BeiGene
Novartis, BeiGene tislelizumab/chemo improves survival of esophageal cancer patients in trial
Jun. 30, 2022 6:54 AM ET
By: Ravikash, SA News Editor
BeiGene (NASDAQ:BGNE) and Novartis (NYSE:NVS) said tislelizumab plus chemotherapy helped improve overall (OS) survival in certain patients with esophageal cancer in a late-stage study.
https://seekingalpha.com/symbol/NVS
https://seekingalpha.com/symbol/BGNE
https://www.beigene.com/our-science-and-medicines/tislelizumab/
EYLEA® (AFLIBERCEPT) INJECTION
June 29, 2022 at 7:30 AM EDT
EYLEA® (AFLIBERCEPT) INJECTION SBLA FOR EVERY 16-WEEK DOSING REGIMEN IN PATIENTS WITH DIABETIC RETINOPATHY ACCEPTED FOR FDA REVIEW
If approved, extended regimen would provide a longer treatment interval and additional dosing flexibility, alongside approved every 4- and 8-week dosing regimens
TARRYTOWN, N.Y., June 29, 2022 /PRNewswire/ -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced the U.S. Food and Drug Administration (FDA) has accepted for review the EYLEA® (aflibercept) Injection supplemental Biologics License Application (sBLA) for an every 16-week 2 mg dosing regimen (after initial monthly doses) in patients with diabetic retinopathy (DR). The target action date for the FDA decision is February 28, 2023.
DR is the leading cause of blindness among working-age American adults, affecting more than 8 million people in the U.S. alone. In 2019, EYLEA was approved for the treatment of all stages of DR with a dosing regimen of every 4 or 8 weeks after five initial monthly doses. If approved, the 16-week dosing regimen could offer certain patients a potentially longer treatment interval and doctors with greater flexibility to individualize treatment.
The sBLA is supported by data from the Phase 3 PANORAMA trial investigating every 8- and 16-week EYLEA dosing regimens, versus sham, in patients with severe non-proliferative diabetic retinopathy (NPDR) without diabetic macular edema (DME). The submission was further supported by data from the NIH-sponsored Protocol W trial investigating an EYLEA every 16-week dosing regimen in patients with moderate to severe NPDR without center-involved DME versus sham.
At 1 year, PANORAMA met its primary endpoint of proportion of patients with ≥2-step improvement in Diabetic Retinopathy Severity Scale (DRSS) score. At 2 years, in both the PANORAMA and Protocol W trials, a greater proportion of patients receiving EYLEA every 16-weeks experienced a ≥2-step improvement in DRSS score, along with greater reductions in the risk of developing vision-threatening complications, versus sham. The rates of serious ocular adverse events and intraocular inflammation in patients treated with EYLEA every 16 weeks were similar across both studies.
In addition to DR with a 4- or 8-week dosing regimen, EYLEA is approved for the treatment of neovascular (Wet) age-related macular degeneration, macular edema following retinal vein occlusion and DME. The potential use of the 16-week dosing regimen for EYLEA in DR has not been fully evaluated by any regulatory authority.
About EYLEA
EYLEA is a VEGF inhibitor formulated as an injection for the eye. It is designed to block the growth of new blood vessels and decrease the ability of fluid to pass through blood vessels (vascular permeability) in the eye by blocking VEGF-A and placental growth factor (PLGF), two growth factors involved in angiogenesis. The EYLEA safety and efficacy profile is supported by a robust body of research that includes eight pivotal Phase 3 trials, 10 years of real-world experience and more than 50 million EYLEA injections globally.
IMPORTANT EYLEA INDICATIONS AND SAFETY INFORMATION
EYLEA® (aflibercept) Injection 2 mg (0.05 mL) is a prescription medicine approved for the treatment of patients with Wet Age-related Macular Degeneration (AMD), Macular Edema following Retinal Vein Occlusion (RVO), Diabetic Macular Edema (DME), and Diabetic Retinopathy (DR).
- EYLEA® (aflibercept) Injection is a prescription medicine administered by injection into the eye. You should not use EYLEA if you have an infection in or around the eye, eye pain or redness, or known allergies to any of the ingredients in EYLEA, including aflibercept.
- Injections into the eye with EYLEA can result in an infection in the eye and retinal detachment (separation of retina from back of the eye) can occur. Inflammation in the eye has been reported with the use of EYLEA.
- In some patients, injections with EYLEA may cause a temporary increase in eye pressure within 1 hour of the injection. Sustained increases in eye pressure have been reported with repeated injections, and your doctor may monitor this after each injection.
- There is a potential but rare risk of serious and sometimes fatal side effects, related to blood clots, leading to heart attack or stroke in patients receiving EYLEA.
- The most common side effects reported in patients receiving EYLEA were increased redness in the eye, eye pain, cataract, vitreous (gel-like substance) detachment, vitreous floaters, moving spots in the field of vision, and increased pressure in the eye.
- You may experience temporary visual changes after an EYLEA injection and associated eye exams; do not drive or use machinery until your vision recovers sufficiently.
- Contact your doctor right away if you think you might be experiencing any side effects, including eye pain or redness, light sensitivity, or blurring of vision, after an injection.
- For additional safety information, please talk to your doctor and see the full Prescribing Information for EYLEA.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
Please see the full Prescribing Information for EYLEA.
For more information, please visit www.Regeneron.com or follow @Regeneron on Twitter.
View original content:https://www.prnewswire.com/news-releases/eylea-aflibercept-injection-sbla-for-every-16-week-dosing-regimen-in-patients-with-diabetic-retinopathy-accepted-for-fda-review-301577424.html
SOURCE Regeneron Pharmaceuticals, Inc.
FDA accepts Regeneron's application for Eylea extended regimen to treat diabetic blindness
Jun. 29, 2022 10:17 AM ET
Regeneron Pharmaceuticals, Inc. (REGN)
By: Anuron Mitra, SA News Editor
- Regeneron Pharmaceuticals (NASDAQ:REGN) on Wednesday said the U.S. FDA had accepted its application requesting for an extended regimen for its Eylea injection to treat patients with diabetic retinopathy (DR).
- https://seekingalpha.com/news/3852874-fda-accepts-regenerons-application-for-eylea-extended-regimen-to-treat-diabetic-blindness
- https://seekingalpha.com/symbol/REGN
- https://www.regeneron.com/
- https://eylea.us/
YESCARTA® (axicabtagene ciloleucel)
June 28, 2022
Kite’s CAR T-cell Therapy Yescarta® Granted European Marketing Authorization for the Treatment of Relapsed or Refractory Follicular Lymphoma
– Pivotal ZUMA-5 Study Demonstrates Overall Response Rate of 91% and a Complete Response rate of 77% in Patients Who Received Yescarta After Three or More Lines of Therapy –
– Kite’s Third Approved Cell Therapy Indication in Europe –
SANTA MONICA, Calif.--(BUSINESS WIRE)-- Kite, a Gilead Company (Nasdaq: GILD), today announces that the European Commission (EC) has approved its CAR T-cell therapy Yescarta® (axicabtagene ciloleucel) for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after three or more lines of systemic therapy. Yescarta has maintained orphan medicinal product designation in this indication.
“Patients with advanced relapsed or refractory follicular lymphoma have a high need for new treatment options,” said Christi Shaw, CEO, Kite. “This is the third approved indication for a Kite cell therapy in Europe, and we are pleased to enable more patients with different lymphomas greater access to this treatment innovation.”
Follicular lymphoma is a form of non-Hodgkin lymphoma in which tumors grow slowly but can become more aggressive over time. FL is the second most common type of lymphoma globally and accounts for approximately 22% of all lymphomas diagnosed worldwide.In Europe, approximately 27,000 new cases are diagnosed each year.
“Follicular lymphoma that has relapsed multiple times is a difficult-to-treat disease with an especially poor prognosis as only 20% of patients are still alive at five years after their second relapse,” said Ibrahim Yakoub-Agha, MD, PhD, Head of the Hematopoietic Cell Transplantation and Cellular Therapy Unit, Lille University Hospital. “Ninety-one percent of patients in the ZUMA-5 study responded to axicabtagene ciloleucel after three or more prior lines of therapy, and more than half of these were still in response two years later. This sign of durable remission is critical for patients who need options that can deliver long-term benefit.”
“Follicular lymphoma is often misunderstood as easy to treat or non life-threatening, even when it has reached a significantly advanced stage,” said Nicola Mendelsohn, Founder and Chair of the Follicular Lymphoma Foundation (FLF). “For patients with later-line relapsed or refractory disease, it is often very aggressive. Axicabtagene ciloleucel represents an important advance for a patient population in Europe with limited treatment options.”
The approval is supported by data from the pivotal, single-arm Phase 2 ZUMA-5 international study in patients with relapsed or refractory FL who had received at least two prior lines of systemic therapy, including the combination of an anti-CD20 monoclonal antibody and an alkylating agent. Among patients who had received three or more lines of prior therapy (n=75), the overall response rate (ORR) was 91%, and the complete response (CR) rate was 77% at the 24-month analysis. The median duration of response (DoR) was 38.6 months, and the proportion of responders still in response at Month 24 was 56%.
Among all evaluable patients within ZUMA-5 (n=119), safety observations were consistent with the known safety profile for Yescarta. Grade ≥3 cytokine release syndrome (CRS) occurred in 6% of patients and neurologic events occurred 16% of patients. Most CRS cases (99%) of any grade resolved by the time of data cut-off and 60% of neurologic events were resolved within three weeks. The most significant and frequently occurring adverse events were CRS (77%), infections (59%) and encephalopathy (47%). For full details on the Special Warnings and Precautions for Use and Adverse Reactions (including appropriate management), please refer to the EU Summary of Product Characteristics (SmPC).
Additional data were shared separately during an oral presentation at the 2021 American Society of Hematology Meeting.
About ZUMA-5
ZUMA-5 is an ongoing, single-arm, open-label, international, multicentre trial evaluating 122 patients (≥18 years old) with relapsed or refractory follicular lymphoma (FL), who received at least two prior lines of systemic therapy, including the combination of an anti-CD20 monoclonal antibody and an alkylating agent. The primary endpoint was ORR, and secondary endpoints included CR rate, ORR and CR in patients who had received three or more lines of prior therapy, DoR, overall survival, progression-free survival and incidence of adverse events.
About Yescarta
Please see full US Prescribing Information, including BOXED WARNING and Medication Guide.
YESCARTA is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of:
- Adult patients with large B-cell lymphoma that is refractory to first-line chemoimmunotherapy or that relapses within 12 months of first-line chemoimmunotherapy.
- Adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma, high-grade B-cell lymphoma, and DLBCL arising from follicular lymphoma.
Limitations of Use: YESCARTA is not indicated for the treatment of patients with primary central nervous system lymphoma. - Adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy. This indication is approved under accelerated approval based on the response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trial(s).
- https://www.yescarta.com/
About Kite
Kite, a Gilead Company, is a global biopharmaceutical company based in Santa Monica, California, with manufacturing operations in North America and Europe. Kite’s singular focus is cell therapy to treat and potentially cure cancer. As the cell therapy leader, Kite has more approved CAR T indications to help more patients than any other company. For more information on Kite, please visit www.kitepharma.com. Follow Kite on social media on Twitter (@KitePharma) and LinkedIn.
About Gilead Sciences
Gilead Sciences, Inc. is a biopharmaceutical company that has pursued and achieved breakthroughs in medicine for more than three decades, with the goal of creating a healthier world for all people. The company is committed to advancing innovative medicines to prevent and treat life-threatening diseases, including HIV, viral hepatitis and cancer. Gilead operates in more than 35 countries worldwide, with headquarters in Foster City, California.
U.S. Prescribing Information for Yescarta including BOXED WARNING, is available at www.kitepharma.com and www.gilead.com .
Kite, the Kite logo, Yescarta, and GILEAD are trademarks of Gilead Sciences, Inc. or its related companies.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220628005521/en/
Source: Gilead Sciences, Inc.
Gilead's Yescarta gets approval in EU for expanded use in blood cancer subtype
Jun. 28, 2022 6:43 AM ET
By: Ravikash, SA News Editor1 Comment
- The European Commission (EC) approved the expanded use of Gilead's (NASDAQ:GILD) CAR T-cell therapy Yescarta to treat adult patients with relapsed or refractory follicular lymphoma (FL) after three or more lines of systemic therapy.
- https://seekingalpha.com/news/3852319-gileads-yescarta-gets-approval-in-eu-for-expanded-use-in-blood-cancer-subtype
- https://seekingalpha.com/symbol/GILD
- https://www.yescarta.com/
Talquetamab
Janssen Announces U.S. FDA Breakthrough Therapy Designation Granted for Talquetamab for the Treatment of Relapsed or Refractory Multiple MyelomaNovel GPRC5DxCD3 Bispecific Antibody Receives Breakthrough Therapy Designation Based Upon Results from the Phase 1/2 MonumenTAL-1 Study
June 29, 2022 (RARITAN, N.J.) – The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for talquetamab for the treatment of adult patients with relapsed or refractory multiple myeloma, who have previously received at least 4 prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 antibody. Talquetamab is an investigational, off-the-shelf, T-cell redirecting bispecific antibody targeting both GPRC5D, a novel drug target, on multiple myeloma cells and CD3 on T-cells. This distinction for talquetamab follows a PRIME (PRIority MEdicines) designation from the European Medicines Agency (EMA) on January 29, 2021, and an Orphan Drug Designation (ODD) from the FDA on May 3, 2021. Today’s milestone marks the 12th BTD received by Janssen in oncology and the third such designation for the company’s portfolio of bispecific antibodies.
“This Breakthrough Therapy Designation marks an important step in the continued development of talquetamab, a first-in-class bispecific antibody T-cell engager using GPRC5D, a novel target for the treatment of patients with relapsed or refractory multiple myeloma,” said Sen Zhuang, M.D., Ph.D., Vice President, Clinical Research and Development, Janssen Research & Development, LLC. “Despite the therapies available for patients with relapsed or refractory multiple myeloma, new targets and treatments are needed because of the heterogeneity of the disease, which can impact a patient’s response to treatment. We are resolute in our commitment to advance science and develop new therapies and regimens for patients with the goal of delivering the best possible outcomes while driving toward cures.”
The Breakthrough Therapy Designation is supported by data from the Phase 1/2, first-in-human dose-escalation MonumenTAL-1 study of talquetamab (Phase 1: NCT03399799; Phase 2: NCT04634552) for the treatment of heavily pretreated patients with relapsed or refractory multiple myeloma.[1]
Data from the MonumenTAL-1 study were featured during the 2022 European Hematology Association (EHA) Annual Congress as an oral presentation (Abstract #S182)[2] and were presented at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting (Abstract #8015).[3]
The FDA grants BTD to expedite the development and regulatory review of an investigational medicine that is intended to treat a serious or life-threatening condition and is based on preliminary clinical evidence that demonstrates the drug may have substantial improvement in at least one clinically significant endpoint over available therapy.[4]
About Talquetamab
Talquetamab is a potential first-in-class, investigational T-cell redirecting bispecific antibody targeting both GPRC5D, a novel multiple myeloma target that does not shed over time, and CD3, a component of the T-cell receptor.[1] CD3 is involved in activating T-cells, and GPRC5D is highly expressed on multiple myeloma cells.[5],[6] Results from preclinical studies in mouse models demonstrate that talquetamab induces T-cell-mediated killing of GPRC5D-expressing multiple myeloma cells through the recruitment and activation of CD3-positive T-cells and inhibits tumor formation and growth.[7]
Talquetamab is currently being evaluated in a Phase 1/2 clinical study for the treatment of relapsed or refractory multiple myeloma (NCT03399799) and is also being explored in combination studies (NCT04586426).
About the Janssen Pharmaceutical Companies of Johnson & Johnson
At Janssen, we’re creating a future where disease is a thing of the past. We’re the Pharmaceutical Companies of Johnson & Johnson, working tirelessly to make that future a reality for patients everywhere by fighting sickness with science, improving access with ingenuity, and healing hopelessness with heart. We focus on areas of medicine in which we can make the biggest difference: Cardiovascular, Metabolism, & Retina; Immunology; Infectious Diseases & Vaccines; Neuroscience; Oncology; and Pulmonary Hypertension.
Learn more at www.janssen.com. Follow us at @JanssenGlobal. Janssen Research & Development, LLC is part of the Janssen Pharmaceutical Companies of Johnson & Johnson.
J&J multiple myeloma candidate for talquetamab granted Breakthrough Therapy Designation
Jun. 29, 2022 9:55 AM ET
By: Jonathan Block, SA News Editor
- The U.S. FDA has granted Breakthrough Therapy Designation to Johnson & Johnson's (NYSE:JNJ) talquetamab for multiple myeloma.
- Talquetamab is being developed by J&J's (JNJ) Janssen division for relapsed/refractory multiple myeloma in those who have failed on four or more prior therapies. It is a T-cell redirecting bispecific antibody targeting GPRC5D on multiple myeloma cells and CD3 on T-cells.
- https://seekingalpha.com/news/3852832-johnson-johnson-janssen-multiple-myeloma-candidate-for-talquetamab-granted-breakthrough-therapy-designation
- https://seekingalpha.com/symbol/JNJ
Zeposia (ozanimod)
Bristol Myers Squibb Presents New Data Showing Effect of Early Zeposia (ozanimod) Treatment in Improving and Preserving Cognitive Function in People With Relapsing Multiple Sclerosis
06/24/2022CATEGORY:
Results showed improved or preserved cognitive function in a majority of people regardless of baseline values, with the greatest effect observed in almost 80% of people with high thalamic volume (45.5% improved and 34.1% preserved) at Month 48 of the DAYBREAK open-label extension trial
Zeposia was well tolerated, with more than 80% of people staying on therapy through 48 months
New analyses to be presented at the 8th European Academy of Neurology Congress in Vienna, Austria
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE:BMY) today announced new post-hoc analyses from the Zeposia (ozanimod) Phase 3 DAYBREAK open-label extension (OLE) and Phase 3 SUNBEAM trials, showing early Zeposia use demonstrated cognitive benefits in people with relapsing multiple sclerosis (MS), with the greatest effect seen in people with high thalamic volume (TV), supporting an association between preserved brain volume (BV) and improved long-term cognitive outcomes. These data (Presentation #EPO-127) are being presented at the European Academy of Neurology (EAN) Congress taking place in Vienna, Austria, from June 25-28.
“Multiple sclerosis can lead to significant, irreversible brain volume loss and decreased cognition if not treated quickly upon diagnosis. These new analyses show the potential of early treatment with Zeposia to help stabilize and even improve cognition in people with multiple sclerosis with high brain volume, which is important for doctors and people with multiple sclerosis,” said John DeLuca, PhD, senior vice president for research and training, Kessler Foundation, and professor, Department of Physical Medicine & Rehabilitation and of Neurology, Rutgers New Jersey Medical School.
In these new exploratory analyses, Zeposia treatment showed improved or preserved cognitive function in a majority of patients, with the greatest improvement seen when used early in the disease when TV remains high, supporting a positive association between preserved BV and long-term cognitive performance. Zeposia was well tolerated with more than 80% of people who started the Phase 3 SUNBEAM trial (N=399 at baseline) remaining on continuous therapy through 48 months of the Phase 3 DAYBREAK OLE study (N=326).
Findings from the new research showed that people with high versus low BV, particularly TV, had higher cognitive performance, as assessed by the symbol digit modalities test (SDMT) score, at baseline. This trend remained stable or improved over 4-5 years of Zeposia treatment, leading to improved or preserved cognitive function in almost 80% of people with high TV (SDMT improved: 45.1%; SDMT preserved: 34.4%) and approximately 66% of people with low BV (SDMT improved: 35.6%; SDMT preserved: 30.7%) at Month 48 of the Phase 3 DAYBREAK OLE study.
“At Bristol Myers Squibb, we’re committed to pathbreaking science in multiple immune-mediated diseases with the goal of alleviating the symptoms and disease progression experienced by individuals suffering from these illnesses and, ultimately, elevating the standard of care,” said Jonathan Sadeh, MD, MSc, senior vice president of Immunology and Fibrosis Development, Bristol Myers Squibb. “We’re excited by the potential effect of Zeposia in protecting cognitive function when used early in treatment before brain volume is lost and what it can mean for individuals with relapsing multiple sclerosis.”
Bristol Myers Squibb thanks the patients and investigators involved in the Phase 3 DAYBREAK OLE and Phase 3 SUNBEAM clinical trials.
About DAYBREAK
DAYBREAK is a Phase 3, multicenter, long-term open-label extension (OLE), randomized, double-blind, double-dummy, active-controlled, parallel group study to evaluate the safety and efficacy of Zeposia (ozanimod) administered orally to patients with relapsing forms of multiple sclerosis (MS).
Eligible patients from the RADIANCE, SUNBEAM and RPC01-1001 trials diagnosed with relapsing forms of MS are enrolled to receive treatment until the end of the DAYBREAK trial or until the development program is discontinued. Patients in the trial are receiving Zeposia 0.92 mg (equivalent to ozanimod HCl 1 mg). In total, 2,639 participants completed the parent clinical trials, and this interim analysis (data cutoff February 2021) includes a total of 2,494 participants with mean (range) Zeposia exposure of 46.8 (0.03-62.7) months in the OLE study.
About SUNBEAM
SUNBEAM was a pivotal, Phase 3, multicenter, randomized, double-blind, double-dummy, active-controlled trial evaluating the efficacy, safety and tolerability of two doses of oral Zeposia (0.92 mg and 0.46 mg, equivalent to 1 mg and 0.5 mg ozanimod HCl, respectively) against weekly intramuscular AVONEX® (interferon beta-1a) for at least a 12-month treatment period. The study included 1,346 people living with relapsing forms of multiple sclerosis (RMS) across 152 sites in 20 countries.
The primary endpoint of the trial was annualized relapse rates during the treatment period. The secondary MRI endpoints included the number of new or enlarging hyperintense T2-weighted brain MRI lesions over 12 months, number of gadolinium-enhanced brain MRI lesions at Month 12 and percent change from baseline in whole brain volume at Month 12. Cortical grey and thalamic volume changes were also prospectively assessed versus active comparator.
An analysis of the time to onset of three-month confirmed disability progression was prespecified using pooled data from both the SUNBEAM and RADIANCE Part B Phase 3 trials.
About Zeposia (ozanimod)
Zeposia (ozanimod) is an oral, sphingosine 1-phosphate (S1P) receptor modulator that binds with high affinity to S1P receptors 1 and 5. Zeposia blocks the capacity of lymphocytes to egress from lymph nodes, reducing the number of lymphocytes in peripheral blood. The mechanism by which Zeposia exerts therapeutic effects in multiple sclerosis (MS) is unknown but may involve the reduction of lymphocyte migration into the central nervous system.
The European Commission approved Zeposia for the treatment of adult patients with relapsing remitting multiple sclerosis (RRMS) with active disease as defined by clinical or imaging features in May 2020 and for the treatment of adults with moderately to severely active ulcerative colitis (UC) who have had an inadequate response, lost response, or were intolerant to either conventional therapy or a biologic agent in November 2021. The U.S. Food and Drug Administration (FDA) approved Zeposia for the treatment of adults with relapsing forms of MS in March 2020 and for adults with moderately to severely active UC on May 27, 2021.
U.S. FDA-APPROVED INDICATIONS FOR ZEPOSIA
ZEPOSIA (ozanimod) is indicated for the treatment of:
1. Relapsing forms of multiple sclerosis (MS), to include clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease, in adults.
2. Moderately to severely active ulcerative colitis (UC) in adults.
For additional safety information, please see the full Prescribing Information and Medication Guide.
Source: Bristol Myers Squibb
BMY Dividend History
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Kesimpta® (ofatumumab)
New Novartis extension phase data show nearly 80% of RMS patients treated with Kesimpta® (ofatumumab) had no evidence of disease activity (NEDA-3)
Jun 27, 2022
- New data from the Phase 3 ASCLEPIOS I/II trials and ALITHIOS open-label extension show that after four years nearly 8 out of 10 of people with relapsing multiple sclerosis (RMS) treated continuously with Kesimpta® (ofatumumab) had no evidence of disease activity (NEDA-3) compared with 5 out of 10 of those who switched to Kesimpta at a later date after initial teriflunomide treatment1
- Earlier initiation with Kesimpta resulted in a more than three-fold increased likelihood of maintaining NEDA-3 throughout the study, highlighting the importance of earlier initiation with a high efficacy therapy 1
- An additional analysis presented from ASCLEPIOS I/II shows that treatment with Kesimpta was associated with significant improvements in cognitive processing speed (CPS) versus those treated with teriflunomide; these improvements were more pronounced in the subgroup of patients recently diagnosed with RMS2
Basel, June 27, 2022 — Novartis today announced new data from the Phase 3 ASCLEPIOS I/II trials and the ALITHIOS open-label extension showing continuous treatment with Kesimpta® (ofatumumab) significantly increased the odds of achieving no evidence of disease activity (NEDA-3) versus switching from teriflunomide1. These data were presented at the European Academy of Neurology (EAN) Annual Meeting being held in Vienna, Austria and virtually on June 25–28, 2022.
These data show that after four years of treatment, 78.8% of those who continuously received Kesimpta achieved NEDA-3 (defined as having no MS relapses, no disability worsening and no MRI activity) versus only 51.8% of those who switched from teriflunomide to Kesimpta in the extension phase (odds ratio: 3.89; p<0.001)1. These data build on the previously presented efficacy data from ASCLEPIOS I/II and ALITHIOS showing sustained differences in cumulative relapses, MRI lesion activity and the risk of disability worsening between those who were continuously treated with Kesimpta versus those who switched at a later date1.
“Early initiation of high-efficacy therapies for the treatment of relapsing multiple sclerosis has been shown to improve long-term outcomes versus escalating from lower efficacy therapies,” said Professor Ludwig Kappos, University Hospital Basel. “NEDA-3 is an important endpoint for physicians to consider when deciding to initiate high efficacy therapy, with this latest data from ALITHIOS we can clearly see the benefit of starting Kesimpta early versus switching to it later from teriflunomide.”
About Kesimpta® (ofatumumab)
Kesimpta is a targeted, precisely dosed and delivered B-cell therapy that provides the flexibility of self-administration for adults with relapsing forms of multiple sclerosis (RMS). It is an anti-CD20 monoclonal antibody (mAb) self-administered by a once-monthly injection, delivered subcutaneously7,8. Initial doses of Kesimpta are at Weeks 0, 1 and 2, with the first injection performed under the guidance of a healthcare professional. As shown in preclinical studies, Kesimpta is thought to work by binding to a distinct epitope on the CD20 molecule inducing potent B-cell lysis and depletion9. The selective mechanism of action and subcutaneous administration of Kesimpta allows precise delivery to the lymph nodes, where B-cell depletion in MS is needed, and preclinical studies have shown that it may preserve the B-cells in the spleen10. Once-monthly dosing of Kesimpta differs from other anti-CD20 therapies as it allows faster repletion of B-cells, offering more flexibility in MS management6. Ofatumumab was originally developed by Genmab and licensed to GlaxoSmithKline. Novartis obtained rights for ofatumumab from GlaxoSmithKline in all indications, including RMS, in December 201511.
Ofatumumab has been approved for the treatment of relapsing forms of multiple sclerosis in the United States, European Union, United Kingdom, Canada, China, Switzerland, Singapore, Australia, Japan, Argentina, United Arab Emirates, Albania, and India etc.
About Novartis
Novartis is reimagining medicine to improve and extend people’s lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world’s top companies investing in research and development. Novartis products reach nearly 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 108,000 people of more than 140 nationalities work at Novartis around the world. Find out more at https://www.novartis.com.
https://seekingalpha.com/symbol/NVS
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Cosentyx® (secukinumab)
Cosentyx® (secukinumab) receives expanded approvals in EU for use in childhood arthritic conditions
Jun 27, 2022
- Approvals based on data from the JUNIPERA trial, showing that Cosentyx® (secukinumab) reduced the risk of flare and disease activity compared to placebo over 2 years in pediatric patients1 with enthesitis-related arthritis (ERA) and psoriatic arthritis (PsA)
- Safety in these pediatric populations was consistent with the known safety profile across approved adult and pediatric indications1,2
- Since its initial approval in 2015, Cosentyx has a proven sustained efficacy profile across several systemic inflammatory conditions and has been used to treat more than 700,000 patients worldwide1–11
Basel, June 27, 2022 — Novartis today announced the European Commission (EC) has approved Cosentyx® (secukinumab), used alone or in combination with methotrexate, in the juvenile idiopathic arthritis (JIA) categories of enthesitis-related arthritis (ERA) and juvenile psoriatic arthritis (JPsA) in patients 6 years and older whose disease has responded inadequately to, or who cannot tolerate, conventional therapy1.
“The approval of Cosentyx is very positive news for children affected by JPsA and ERA across Europe. We are now able to offer a new therapeutic target, which was not on the market for this disease in children and also offers a lower frequency of administration. Cosentyx adds to the body of other approved treatments that may provide children and adolescent patients, with the opportunity to participate in all daily activities, and even sports,” said Ivan Foeldvari, M.D., Hamburg Centre for Pediatric Rheumatology, Germany.
ERA and JPsA are two forms of juvenile idiopathic arthritis (JIA) and are progressive, debilitating autoimmune diseases12–14. ERA is characterized by joint swelling and pain where tendons and ligaments attach to bone and may present with lower back pain or tenderness at the palpation of the hips13,14. JPsA is characterized by joint swelling and skin psoriasis and may present with nail changes, inflammation of fingers and/or toes or psoriatic skin changes in a first-degree relative. If left untreated, these diseases can lead to high levels of pain and disability12–15.
“Cosentyx could now provide a treatment option for eligible patients who continue to struggle with the painful symptoms which negatively impact their quality of life, such as inflammation of the joints, swollen fingers and toes,” said Todd Fox, Global Head of Medical Affairs Immunology at Novartis. “This approval represents an important step in our ambition to expand Cosentyx to 10 indications for children and adults living with rheumatic and dermatologic diseases.”
The approval is based on data from the Phase III JUNIPERA trial, a 2-year, three-part, double-blind, placebo-controlled, randomized withdrawal trial showing significantly longer time to flare in Cosentyx versus placebo in pediatric patients with ERA and JPsA16. Safety in this pediatric population was consistent with the known safety profile of Cosentyx across approved adult and pediatric indications1,2.
Novartis is working closely with regulatory agencies to ensure that eligible European patients can start benefitting from Cosentyx as quickly as possible. In July 2020, Cosentyx received European Medicines Agency approval as a first-line systemic treatment for pediatric psoriasis in patients aged 6–18 years old and recently received approval in the US and China1,17. In 2021, Cosentyx was also approved in Japan for pediatric psoriasis18. Cosentyx was also approved in the US in December 2021 and earlier this year in Brazil to treat ERA in patients 4 years or older and JPsA in patients aged 2 years and older19.
About the JUNIPERA trial
The EC approval is based on data from the Phase III JUNIPERA trial, a 2-year, three-part, double-blind, placebo-controlled, randomized withdrawal trial that enrolled 86 children and adolescents aged 2–18 years old with a confirmed diagnosis of ERA or JPsA according to the modified International League of Associations for Rheumatology classification criteria16. The primary endpoint of the trial was time to flare in the treatment period 2 (Week 12 to Week 104)16. The trial met its primary endpoint and demonstrated a statistically significant longer time to disease flare in treatment period 2 for ERA and JPsA with secukinumab versus placebo. The risk of flare was reduced by 72% for patients on secukinumab compared with patients on placebo in treatment period 2 (hazard ratio=0.28, 95% confidence interval: 0.13 to 0.63; P<0.001). A total of 21 patients in the placebo group experienced a flare (11 JPsA and 10 ERA) compared with 10 patients in the secukinumab group (4 JPsA and 6 ERA) during the placebo-controlled treatment period 2 of the trial1,10. Safety in this pediatric population was consistent with the known safety profile of Cosentyx for the treatment of adult and pediatric plaque psoriasis, PsA and axial spondyloarthritis2.
About Cosentyx
Cosentyx is the first and only fully human biologic that directly inhibits interleukin-17A, an important cytokine involved in the inflammation of psoriatic arthritis (PsA), moderate to severe plaque psoriasis, ankylosing spondylitis (AS) and non-radiographic axial spondyloarthritis (nr-axSpA)1,20. Cosentyx is a proven medicine and has been studied clinically for more than 14 years. The medicine is backed by robust evidence, including 5 years of clinical data in adults supporting long-term safety and efficacy in adult patients with moderate to severe plaque psoriasis, PsA, AS and nr-axSpA3–5,7–9,21. These data strengthen the position of Cosentyx as a treatment option in these conditions, and are supported by more than 700,000 patients treated worldwide since launch in 20151,11,22.
About Novartis
Novartis is reimagining medicine to improve and extend people’s lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world’s top companies investing in research and development. Novartis products reach nearly 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 108,000 people of more than 140 nationalities work at Novartis around the world. Find out more at https://www.novartis.com.
Novartis Cosentyx gets approval in EU for expanded use in certain kids with arthritis
Jun. 27, 2022 5:19 AM ET
By: Ravikash, SA News Editor
- The European Commission (EC) approved the expanded use of Novartis' (NYSE:NVS) psoriasis/arthritis drug Cosentyx.
- https://seekingalpha.com/news/3851932-novartis-cosentyx-gets-approval-in-eu-for-expanded-use-in-certain-kids-with-arthritis
- https://seekingalpha.com/symbol/NVS
- https://www.novartis.com/
- https://www.cosentyx.com/
Atogepant (QULIPTA™)
June 21, 2022
AbbVie Submits Supplemental New Drug Application to U.S. FDA for Atogepant (QULIPTA™) to Support Label Expansion for the Preventive Treatment of Migraine
- Submission is based on pivotal Phase 3 PROGRESS chronic migraine study evaluating atogepant (QULIPTATM) in adult patients that met primary endpoint of statistically significant reduction from baseline in mean monthly migraine days compared to placebo
- If approved, atogepant (QULIPTA) would be the first gepant (oral calcitonin gene-related peptide [CGRP] receptor antagonist) with a broad preventive treatment of migraine indication that expands treatment to patients with chronic migraine
- Label expansion would make AbbVie the only company to offer two preventive treatments for those with chronic migraine, atogepant (QULIPTA) and onabotulinumtoxinA (BOTOX®)
NORTH CHICAGO, Ill., June 21, 2022 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced that it has submitted a supplemental New Drug Application (sNDA) for atogepant (QULIPTATM) to the U.S. Food and Drug Administration (FDA) to support the preventive treatment of chronic migraine in adults. If approved, atogepant (QULIPTA) would be the first gepant (oral calcitonin gene-related peptide [CGRP] receptor antagonist) approved for the broad indication of the preventive treatment of migraine, including episodic and chronic. The sNDA submission includes data from the pivotal Phase 3 PROGRESS trial in patients with chronic migraine, which supplements the existing data in episodic migraine. People living with chronic migraine experience headaches for 15 or more days per month, which, on at least eight of those days per month, have the features of migraine.1
"Having one oral medication to treat both episodic and chronic migraine would be an important advancement for health care providers and patients," said Michael Gold, M.D., therapeutic area head, neuroscience development, AbbVie. "This sNDA approval would also diversify AbbVie's migraine portfolio and make it the only company to offer two approved preventive treatments for those living with chronic migraine. No two migraine patients are alike, so having multiple treatment options with unique mechanisms of action is critical."
The pivotal Phase 3 PROGRESS trial met its primary endpoint of statistically significant reduction from baseline in mean monthly migraine days compared to placebo across the 12-week treatment period in adults with chronic migraine. The trial also demonstrated that treatment with atogepant 60 mg once daily (QD) and 30 mg daily (BID) resulted in statistically significant improvements in all six secondary endpoints. This includes a key secondary endpoint that measured the proportion of patients that achieved at least a 50 percent reduction in mean monthly migraine days across the 12-week treatment period.
The overall safety profile of the Phase 3 PROGRESS study was consistent with safety findings observed in previous studies in an episodic migraine population. The most common adverse events were constipation and nausea.
Atogepant is marketed as QULIPTATM in the United States and is FDA-approved to treat adults with episodic migraine. Use of atogepant for the preventive treatment of chronic migraine in the United States is not approved, and its safety and efficacy have not been evaluated by regulatory authorities.
About the Phase 3 PROGRESS Clinical Trial
The Phase 3 PROGRESS clinical trial evaluated the safety, tolerability and efficacy of oral atogepant for the preventive treatment of chronic migraine.2 The patient population for the study included patients with a diagnosis of chronic migraine for at least one year, and greater than or equal to 15 headache days with greater than or equal to eight migraine days in the 28 days prior to randomization.2 The primary endpoint measured the reduction from baseline in mean monthly migraine days compared to placebo, for both doses, including 60 mg once daily (QD) and 30 mg twice daily (BID), across a 12-week treatment period.2
Key secondary endpoints for all regions included: Change from baseline in mean monthly headache days across the 12-week of treatment period (baseline is defined as the number of migraine days during the last 28 days prior to the randomization date); Change from baseline in mean monthly acute medication use days across the 12-week treatment period (baseline is defined as the number of migraine days during the last 28 days prior to the randomization date); Proportion of participants with at least a 50% reduction in mean monthly migraine days across the 12-week treatment period; and change from baseline in MSQ v2.1 Role Function-Restrictive domain score at Week 12. The MSQ v2.1 is a questionnaire designed to measure health-related quality of life impairments attributed to migraine in the past four weeks. It is divided into three domains, assessing how a patient's daily, social, and work activities are limited by migraine; how migraine prevents these activities; and assesses the emotional function related with migraine.
For a full listing of secondary endpoints across all regions, please go to www.clinicaltrials.gov (NCT03855137).
About QULIPTA™ (atogepant)
QULIPTA™ was approved by the U.S. Food and Drug Administration (FDA) for the preventive treatment of episodic migraine in adults in September 2021. It is available in the United States as the first and only gepant (oral calcitonin gene-related peptide [CGRP] receptor antagonist) developed specifically for the preventive treatment of migraine and is now under review by the FDA to expand the episodic indication to also treat patients with chronic migraine. CGRP and its receptors are expressed in regions of the nervous system associated with migraine pathophysiology, and studies have shown that CGRP levels are elevated during migraine attacks. QULIPTA blocks CGRP through a once-daily dose and is available in three strengths – 10 mg, 30 mg and 60 mg.
Please see full Prescribing Information.
Globally, prescribing information varies; refer to the individual country product label for complete information.
About AbbVie in Migraine
At AbbVie, we are committed to empowering people living with migraine disease. We advance science that enables health care providers to care for people impacted across the spectrum of migraine. Through education and partnerships with the migraine community, we strive to help those with migraine navigate barriers to care, access effective treatments and reduce the impact of migraine on their lives.
About AbbVie in Neuroscience
At AbbVie, our commitment to preserve the personhood of those living with neurological and psychiatric disorders is unwavering. Every challenge in this uncharted territory makes us more determined and drives us harder to discover and deliver solutions for patients, care partners and clinicians. AbbVie's Neuroscience portfolio consists of approved therapies in neurological and psychiatric disorders, including bipolar I disorder, cervical dystonia, major depressive disorder, migraine, Parkinson's disease, post-stroke spasticity, schizophrenia and others along with a robust pipeline.
We have a strong investment in neuroscience research, with our Foundational Neuroscience Center in Cambridge, Massachusetts, and our Neuroscience Discovery site in Ludwigshafen, Germany, where our research and resilience in these challenging therapeutic areas is yielding a deeper understanding of the pathophysiology of neurological and psychiatric disorders, and identifying targets for potential disease-modifying therapeutics aimed at making a difference in people's lives.
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Lenacapavir
June 24, 2022
Investigational Lenacapavir Receives Positive CHMP Opinion for People With Multi-Drug Resistant HIV
– Recommendation is Based on Week 26 Data from the CAPELLA Trial Showing Twice-Yearly Lenacapavir Achieved High Rates of Virologic Suppression in Heavily Treatment-Experienced People with HIV –
– If Authorized, Lenacapavir Could Offer a New, Every Six-Month Treatment Option for People with Limited Treatment Choices –
FOSTER CITY, Calif.--(BUSINESS WIRE)-- Gilead Sciences, Inc. (Nasdaq: GILD) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion for investigational lenacapavir for the treatment of HIV-1 infection, in combination with other antiretroviral(s), in adults with multi-drug resistant HIV-1 infection for whom it is otherwise not possible to construct a suppressive anti-viral regimen.
The CHMP positive opinion is a scientific recommendation to the European Commission (EC) to grant marketing authorization in Europe and will be reviewed by the EC, which has the authority to authorize medicines in the 27 Member States of the European Union, as well as Norway, Iceland and Liechtenstein. The final European Commission decision is expected later this year.
“Treatment options are extremely limited for people living with HIV whose virus is no longer effectively controlled by their current regimen. We are encouraged by this CHMP positive opinion for lenacapavir, as it is an important step toward a potential new treatment option for individuals with multi-drug resistant HIV,” said Jared Baeten, MD, PhD, Vice President, HIV Clinical Development, Gilead Sciences. “We look forward to the final decision by the European Commission and the potential for lenacapavir to help fill a critical unmet need for persons living with HIV with complex prior treatment histories.”
The positive opinion is supported by data from the Phase 2/3 CAPELLA trial, a double-blinded, placebo-controlled global multicenter study designed to evaluate the antiviral activity of lenacapavir administered every six months as a subcutaneous injection, in combination with other antiretroviral(s), in heavily treatment-experienced people with multi-drug resistant HIV-1 infection. In this patient population of high unmet medical need, 81% (n=29/36) of participants receiving lenacapavir in addition to an optimized background regimen achieved an undetectable viral load (<50 copies/mL) at Week 26. Additionally, CAPELLA participants achieved a mean increase in CD4 count of 81 cells/µL. The New England Journal of Medicine published the primary outcome results of the CAPELLA trial in its May 11, 2022 issue - Capsid Inhibition with Lenacapavir in Multidrug-Resistant HIV-1 Infection. Through Week 26, lenacapavir was generally well tolerated, with no serious adverse events related to lenacapavir as determined by the study investigator. The most common adverse events observed in the trial were injection-site reactions.
Lenacapavir is an investigational compound and is not approved by any regulatory authority for any use and its safety and efficacy are not established. There is no cure for HIV or AIDS.
About Lenacapavir
Lenacapavir is Gilead’s potential first-in-class, investigational long-acting HIV-1 capsid inhibitor in development for the treatment of HIV-1 infection. The safety, efficacy and dosing of Gilead’s investigational, long-acting HIV-1 capsid inhibitor lenacapavir are being evaluated in multiple ongoing clinical studies. Lenacapavir's multi-stage mechanism of action is distinguishable from currently approved classes of antiviral agents and is designed to provide a new avenue for the development of long-acting therapy options for people with or at risk for HIV-1. While most antivirals act on just one stage of viral replication, lenacapavir is designed to inhibit HIV-1 at multiple stages of its lifecycle and has no known cross resistance to other existing drug classes. If authorized, lenacapavir would be the only HIV-1 treatment option administered twice yearly.
About CAPELLA (NCT04150068)
CAPELLA is a Phase 2/3, double-blinded, placebo-controlled global multicenter study designed to evaluate the antiviral activity of lenacapavir administered every six months as a subcutaneous injection in heavily treatment-experienced people with multi-drug resistant HIV-1 infection. CAPELLA includes men and women with HIV-1 and is being conducted at research centers in North America, Europe and Asia.
In CAPELLA, 36 participants with multi-class HIV-1 drug resistance and a detectable viral load while on a failing regimen were randomly allocated to receive oral lenacapavir or placebo in a 2:1 ratio for 14 days, in addition to continuing their failing regimen (functional monotherapy). An additional 36 participants were enrolled in a separate treatment cohort. Both cohorts are part of the ongoing maintenance period of the study evaluating the safety and efficacy of subcutaneous lenacapavir administered every six months in combination with an optimized background regimen. The primary endpoint was the proportion of participants randomly allocated to receive lenacapavir or placebo for 14 days, in addition to continuing their failing regimen, achieving ≥0.5 log10 copies/mL reduction from baseline in HIV-1 RNA at the end of the functional monotherapy period.
Following the 14-day functional monotherapy period, participants randomly allocated to receive lenacapavir or placebo, in addition to continuing their failing regimen, started open-label lenacapavir and an optimized background regimen, while those enrolled in a separate treatment cohort received open-label lenacapavir and an optimized background regimen on Day 1. This ongoing maintenance period of the study is evaluating the additional trial endpoints of safety and efficacy of subcutaneous lenacapavir administered every six months in combination with an optimized background regimen.
For further information, please see https://clinicaltrials.gov/ct2/show/NCT04150068.
GILEAD and the GILEAD logo are trademarks of Gilead Sciences, Inc. All other trademarks are the property of their respective owners.
For more information about Gilead, please visit the company’s website at www.gilead.com , follow Gilead on Twitter ( @Gilead Sciences ) or call Gilead Public Affairs at 1-800-GILEAD-5 or 1-650-574-3000.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220623005107/en/
Source: Gilead Sciences, Inc.
Gilead receives positive European Medicine Agency opinion for HIV treatment
Jun. 24, 2022 8:55 AM ET
By: Dania Nadeem, SA News Editor1 Comment
- Gilead Sciences (NASDAQ:GILD) said on Friday it had received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) for its investigational therapy, lenacapavir, for the treatment of HIV-1 infection, in combination with other antiretrovirals.
- https://seekingalpha.com/news/3851507-lilly-receives-positive-european-medicine-agency-opinion-for-hiv-treatment
- https://seekingalpha.com/symbol/GILD
- https://www.gilead.com/
ROCTAVIAN™ (valoctocogene roxaparvovec)
Jun 24, 2022
European Commission Approval Expected Q3 2022
1st Gene Therapy for Treatment of Hemophilia A Recommended for Approval in Europe
More than 20,000 Adults with Severe Hemophilia A in BioMarin Territories Across Europe, the Middle East and Africa
Conference Call Scheduled for Friday, June 24 at 11 a.m. ET
SAN RAFAEL, Calif., June 24, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending conditional marketing authorization (CMA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. A final approval decision, typically consistent with the CHMP recommendation, is expected from the European Commission in Q3 2022.
The one-time infusion is planned to be marketed under the brand name ROCTAVIAN™ (valoctocogene roxaparvovec), for the treatment of severe hemophilia A (congenital factor VIII deficiency) in adult patients without a history of factor VIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5 (AAV5). Roctavian is the first gene therapy to be recommended for approval in Europe for hemophilia A.
It is estimated that more than 20,000 adults across Europe, Middle East, and Africa are affected by severe hemophilia A. BioMarin anticipates additional patient access through named patient sales based on an EMA approval in countries in the Middle East and Africa and expects additional market registrations to be facilitated by an anticipated EMA license.
"Today's positive CHMP opinion for Roctavian addresses the unmet medical needs in severe hemophilia A by providing a treatment option that has been shown in clinical studies can maintain effective levels of endogenously produced coagulation Factor VIII over multiple years with a single intravenous administration. Currently available treatment options require long-term, chronic use with a high degree of compliance to a prescribed schedule to be effective," said Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin. "We are grateful to the patients, investigators and community who have been an integral part in reaching this important milestone that brings us one step closer to delivering on the promise and ingenuity of gene therapy. We are proud of this scientific accomplishment and committed to the ongoing study of Roctavian."
"The positive CHMP opinion offers hope for a new treatment option for people with severe hemophilia A, who have been bound to lifelong treatment and still experience serious health complications, such as breakthrough bleeding, pain, and joint damage, as well as having to constantly consider their condition in all aspects of their lives," said Professor Johannes Oldenburg, Director of the Institute of Experimental Haematology and Transfusion Medicine and the Haemophilia Centre at the University Clinic in Bonn, Germany. "The robust data set from the clinical trial program underscores the potential impact of gene therapy for patients, including a substantial and sustained reduction in bleeding that would have previously required Factor VIII infusions."
People with hemophilia A have a mutation or irregularity in the gene responsible for producing Factor VIII (FVIII), a protein necessary for blood clotting. The standard of care for patients with severe hemophilia A is chronic lifelong injectable therapy to maintain enough clotting factor in the bloodstream to prevent bleeds. Investigational valoctocogene roxaparvovec gene therapy works by delivering a functional gene that is designed to enable the body to produce FVIII on its own with the goal of reducing the need for ongoing prophylaxis.
The CHMP based its positive opinion on the totality of data from the valoctocogene roxaparvovec clinical development program, the most extensively studied gene therapy for hemophilia A, including two-year outcomes from the global GENEr8-1 Phase 3 study, supported by five and four years of follow-up from the 6e13 vg/kg and 4e13 vg/kg dose cohorts respectively, in the ongoing Phase 1/2 dose escalation study. BioMarin has committed to continue working with the broader community to monitor the long-term effects of treatment.
The CHMP is a scientific committee composed of representatives from the 27-member states of the EU, and Iceland, Norway, and Liechtenstein. The committee reviews medical product applications on its scientific and clinical merit and provides advice to the European Commission (EC), which has the authority to approve medicines for the EU.
A conditional marketing authorization (CMA) recognizes that benefit to public health of the immediate availability on the market outweighs the uncertainties inherent to the fact that the science is still new, as is the case with any gene therapy, and the fact that additional data are still required. Once a CMA has been granted by EMA, BioMarin will provide further data from ongoing studies within defined timelines to confirm that the benefits continue to outweigh the risks, building on what already constitutes the largest clinical data package for gene therapy in hemophilia A. Conversion to a standard marketing authorization will be contingent on the provision of additional data from currently ongoing Roctavian clinical studies, including longer-term follow up of patients enrolled in the pivotal trial GENEr8-1, as well as a study of corticosteroids for which enrollment is now complete. The final summary of product characteristics will be available when the product is approved by the European Commission.
Robust Clinical Program
BioMarin has multiple clinical studies underway in its comprehensive gene therapy program for the treatment of hemophilia A. In addition to the global Phase 3 study GENEr8-1 and the ongoing Phase 1/2 dose escalation study, the Company is also conducting a Phase 3B, single arm, open-label study to evaluate the efficacy and safety of valoctocogene roxaparvovec at a dose of 6e13 vg/kg with prophylactic corticosteroids in people with hemophilia A (Study 270-303). In addition, the Company is running a Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with hemophilia A with pre-existing AAV5 antibodies (Study 270-203), as well as another Phase 1/2 Study with the 6e13 vg/kg dose of valoctocoge
About BioMarin
BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening rare diseases and medical conditions. The Company selects product candidates for diseases and conditions that represent a significant unmet medical need, have well-understood biology and provide an opportunity to be first-to-market or offer a significant benefit over existing products. The Company's portfolio consists of eight commercial products and multiple clinical and preclinical product candidates for the treatment of various diseases. For additional information, please visit www.biomarin.com.
BioMarin® is a registered trademark and ROCTAVIAN™ is a trademark of BioMarin Pharmaceutical Inc.
SOURCE BioMarin Pharmaceutical Inc.
BioMarin hemophilia gene therapy gets EMA panel backing for conditional approval in EU
Jun. 24, 2022 9:00 AM ET
BioMarin Pharmaceutical Inc. (BMRN)
By: Ravikash, SA News Editor1 Comment
- A committee of the European Medicines Agency (EMA) recommended conditional marketing authorization of BioMarin Pharmaceutical's (NASDAQ:BMRN) gene therapy Roctavian to treat severe hemophilia A.
- https://seekingalpha.com/news/3851509-biomarin-hemophilia-gene-therapy-gets-ema-panel-backing-for-conditional-approval-in-eu
- https://seekingalpha.com/symbol/BMRN
- https://www.biomarin.com/our-treatments/pipeline/valoctocogene-roxaparvovec-for-severe-hemophilia-a/
RINVOQ® (upadacitinib) extended-release tablets
Rinvoq upadacitinib
Opinion
On 23 June 2022, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending a change to the terms of the marketing authorisation for the medicinal product Rinvoq.1 The marketing authorisation holder for this medicinal product is AbbVie Deutschland GmbH & Co. KG.
The CHMP adopted a new indication as follows:
Rinvoq is indicated for the treatment of active non-radiographic axial spondyloarthritis in adult patients with objective signs of inflammation as indicated by elevated C-reactive protein (CRP) and/or magnetic resonance imaging (MRI), who have responded inadequately to nonsteroidal anti-inflammatory drugs (NSAIDs).
For information, the full indications for Rinvoq will therefore be as follows:2
Rheumatoid arthritis
RINVOQ is indicated for the treatment of moderate to severe active rheumatoid arthritis in adult patients who have responded inadequately to, or who are intolerant to one or more disease-modifying anti-rheumatic drugs (DMARDs). RINVOQ may be used as monotherapy or in combination with methotrexate.
Psoriatic arthritis
RINVOQ is indicated for the treatment of active psoriatic arthritis in adult patients who have responded inadequately to, or who are intolerant to one or more DMARDs. RINVOQ may be used as monotherapy or in combination with methotrexate.
Axial spondyloarthritis
Non-radiographic axial spondyloarthritis (nr-axSpA)
RINVOQ is indicated for the treatment of active non-radiographic axial spondyloarthritis in adult patients with objective signs of inflammation as indicated by elevated C-reactive protein (CRP) and/or magnetic resonance imaging (MRI), who have responded inadequately to nonsteroidal anti-inflammatory drugs (NSAIDs).
Ankylosing spondylitis (AS, radiographic axial spondyloarthritis)
RINVOQ is indicated for the treatment of active ankylosing spondylitis in adult patients who have responded inadequately to conventional therapy.
EMA advisors recommend indication extension for AbbVie's Rinvoq
Jun. 24, 2022 8:19 AM ET
By: Jonathan Block, SA News Editor
- An advisory panel to the European Medicines Agency (EMA) has recommended that AbbVie's (NYSE:ABBV) Rinvoq (upadacitinib) receive an additional indication for non-radiographic axial spondyloarthritis.
- https://seekingalpha.com/news/3851486-ema-advisors-recommend-indication-extension-for-abbvie-rinvoq
- https://seekingalpha.com/symbol/ABBV
- https://www.abbvie.com/
- https://www.rinvoq.com/
biotecMAX™ Feb/Mar/APR/May/June 2022 Insight
ENHERTU® (fam-trastuzumab deruxtecan-nxki) for injection
Trastuzumab Deruxtecan Type II Variation Application Validated by EMA for Patients with HER2 Low Metastatic Breast Cancer with HR Positive and HR Negative Disease • Application based on DESTINY-Breast04 results that showed Daiichi Sankyo and AstraZeneca’s trastuzumab deruxtecan demonstrated superior progression-free and overall survival versus chemotherapy
Tokyo and Munich – (June 22, 2022) – Daiichi Sankyo (TSE: 4568) today announced that the European Medicines Agency (EMA) has validated the Type II Variation application for trastuzumab deruxtecan as monotherapy for the treatment of adult patients with unresectable or metastatic HER2 low (immunohistochemistry (IHC) 1+ or IHC 2+/in-situ hybridization (ISH)-negative) breast cancer who have received a prior systemic therapy in the metastatic setting or developed disease recurrence during or within six months of completing adjuvant chemotherapy. Patients with hormone receptor (HR) positive breast cancer must additionally have received or be ineligible for endocrine therapy. Trastuzumab deruxtecan is a specifically engineered HER2 directed antibody drug conjugate (ADC) being jointly developed and commercialized by Daiichi Sankyo and AstraZeneca (LSE/STO/Nasdaq: AZN). Validation confirms that the application is complete and commences the scientific review process by the EMA’s Committee for Medicinal Products for Human Use (CHMP). This application is based on data from the DESTINY-Breast04 phase 3 trial recently presented at the plenary session of the American Society of Clinical Oncology (#ASCO22) Annual Meeting and simultaneously published in The New England Journal of Medicine. “Trastuzumab deruxtecan is the first HER2 directed therapy to demonstrate a survival benefit in patients with HER2 low metastatic breast cancer. We now have the potential to redefine how we classify and treat approximately half of all metastatic breast cancers,” said Gilles Gallant, BPharm, PhD, FOPQ, Senior Vice President, Global Head, Oncology Clinical Development, Oncology R&D, Daiichi Sankyo. “In addition to the ongoing review of two other applications for the treatment of patients with HER2 positive metastatic breast cancer or gastric cancer in Europe, we are pleased to have received this third validation for HER2 low metastatic breast cancer with the goal of bringing trastuzumab deruxtecan to as many eligible patients with HER2 targetable cancers as possible.” 2 In DESTINY-Breast04, trastuzumab deruxtecan demonstrated superior and clinically meaningful efficacy in progression-free survival (PFS) and overall survival (OS) in previously treated patients with HER2 low unresectable and/or metastatic breast cancer with HR positive or HR negative disease versus standard of care physician’s choice of chemotherapy. The safety profile of trastuzumab deruxtecan was consistent with previous clinical trials with no new safety concerns identified. Interstitial lung disease (ILD) or pneumonitis rates were consistent with that observed in late-line HER2 positive breast cancer trials of trastuzumab deruxtecan with a lower rate of grade 5 ILD observed, as determined by an independent adjudication committee. About DESTINY-Breast04 DESTINY-Breast04 is a global, randomized, open-label, pivotal phase 3 trial evaluating the efficacy and safety of trastuzumab deruxtecan (5.4 mg/kg) versus physician’s choice of chemotherapy (capecitabine, eribulin, gemcitabine, paclitaxel or nab-paclitaxel) in patients with HR positive or HR negative, unresectable and/or metastatic breast cancer with low HER2 expression previously treated with one or two prior lines of chemotherapy. Patients were randomized 2:1 to receive either trastuzumab deruxtecan or chemotherapy. The primary endpoint of DESTINY-Breast04 is PFS in patients with HR positive disease based on blinded independent central review (BICR). Key secondary endpoints include PFS based on BICR in all randomized patients (HR positive and HR negative disease), OS in patients with HR positive disease and OS in all randomized patients (HR positive and HR negative disease). Other secondary endpoints include PFS based on investigator assessment, objective response rate based on BICR and on investigator assessment, duration of response based on BICR and safety. DESTINY-Breast04 enrolled 557 patients at multiple sites in Asia, Europe and North America. For more information about the trial, visit ClinicalTrials.gov.
About Trastuzumab Deruxtecan Trastuzumab deruxtecan (fam-trastuzumab deruxtecan-nxki in the U.S. only) is a HER2 directed ADC. Designed using Daiichi Sankyo’s proprietary DXd ADC technology, trastuzumab deruxtecan is the lead ADC in the oncology portfolio of Daiichi Sankyo and the most advanced program in AstraZeneca’s ADC scientific platform. Trastuzumab deruxtecan consists of a HER2 monoclonal antibody attached to a topoisomerase I inhibitor payload, an exatecan derivative, via a stable tetrapeptide-based cleavable linker. Trastuzumab deruxtecan (5.4 mg/kg) is approved in Canada, Israel and the U.S. for the treatment of adult patients with unresectable or metastatic HER2 positive breast cancer who have received a prior anti-HER2- based regimen either in the metastatic setting, or in the neoadjuvant or adjuvant setting and have developed disease recurrence during or within six months of completing therapy, based on results from the DESTINYBreast03 trial. Trastuzumab deruxtecan also is approved in approximately 40 countries for the treatment of adult patients with unresectable or metastatic HER2 positive breast cancer who have received two or more prior anti-HER2-based regimens based on the results from the DESTINY-Breast01 trial. Trastuzumab deruxtecan (6.4 mg/kg) is approved in several countries for the treatment of adult patients with locally advanced or metastatic HER2 positive gastric or gastroesophageal junction (GEJ) adenocarcinoma who have received a prior trastuzumab-based regimen based on the results from the DESTINY-Gastric01 trial.
About the Daiichi Sankyo and AstraZeneca Collaboration Daiichi Sankyo Company, Limited (referred to as Daiichi Sankyo) and AstraZeneca entered into a global collaboration to jointly develop and commercialize trastuzumab deruxtecan in March 2019 and datopotamab deruxtecan (Dato-DXd) in July 2020, except in Japan where Daiichi Sankyo maintains exclusive rights for each ADC. Daiichi Sankyo is responsible for the manufacturing and supply of trastuzumab deruxtecan and datopotamab deruxtecan.
For more information, please visit: www.daiichisankyo.com.
AstraZeneca, Daiichi Sankyo Enhertu gets EMA review for breast cancer subtype
Jun. 22, 2022 7:08 AM ET
AstraZeneca PLC (AZN), DSNKY, DSKYF
By: Ravikash, SA News Editor1 Comment
The European Medicines Agency (EMA) validated an application for AstraZeneca (NASDAQ:AZN) and Daiichi Sankyo's (OTCPK:DSKYF) (OTCPK:DSNKY) Enhertu to treat certain patients with breast cancer.
https://seekingalpha.com/symbol/DSKYF
https://seekingalpha.com/symbol/DSNKY
https://seekingalpha.com/symbol/AZN
TAFINLAR® (dabrafenib) capsules and MEKINIST® (trametinib) tablets
Novartis Tafinlar + Mekinist receives FDA approval for first tumor-agnostic indication for BRAF V600E solid tumors
Jun 23, 2022
- Tafinlar + Mekinist, the worldwide targeted therapy leader in BRAF/MEK-inhibition, is the first and only therapy to be approved with a tumor-agnostic indication for adult and pediatric patients with solid tumors that have a BRAF V600E mutation1,2
- Approval supported by results from Phase II ROAR and NCI-MATCH studies demonstrating overall response rates up to 80% in patients with BRAF V600E solid tumors1,2
- BRAF mutations drive tumor growth across more than 20 tumor types, including thyroid, brain and gynecologic cancers3,4
Basel, June 23, 2022 — Novartis today announced the US Food and Drug Administration (FDA) granted accelerated approval for Tafinlar® (dabrafenib) + Mekinist® (trametinib) for the treatment of adult and pediatric patients 6 years of age and older with unresectable or metastatic solid tumors with BRAF V600E mutation who have progressed following prior treatment and have no satisfactory alternative treatment options1,2. In accordance with the Accelerated Approval Program, continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). Tafinlar + Mekinist is the first and only BRAF/MEK inhibitor to be approved with a tumor-agnostic indication for solid tumors carrying the BRAF V600E mutation, which drives tumor growth in more than 20 different tumor types, and it is the only BRAF/MEK inhibitor approved for use in pediatric patients1,2.
“The combination of dabrafenib and trametinib demonstrated meaningful efficacy in multiple BRAF-positive tumor types, including in some patients with rare cancers who have no other treatment options available,” said principal investigator Dr. Vivek Subbiah, M.D., associate professor of Investigational Cancer Therapeutics and center medical director of the Clinical Center for Targeted Therapy, Division of Cancer Medicine, at The University of Texas MD Anderson Cancer Center in Houston, Texas. “Physicians should consider a BRAF test as a routine diagnostic step that could enable a new option for treating patients with many solid tumors.”
The FDA approval was based on clinical efficacy and safety demonstrated in three clinical trials. In the Phase II ROAR (Rare Oncology Agnostic Research) basket study and the NCI-MATCH Subprotocol H study, Tafinlar + Mekinist resulted in overall response rates of up to 80% in patients with BRAF V600E solid tumors, including high- and low-grade glioma, biliary tract cancer and certain gynecological and gastrointestinal cancers. An additional study (Study X2101) demonstrated the clinical benefit and acceptable safety profile of Tafinlar + Mekinist in pediatric patients1,2.
“Tackling cancer is complex, which is why it is so important that we continue to follow the science as we pursue meaningful advances and new approaches to treating cancer,” said Reshema Kemps-Polanco, Head, Novartis Oncology US. “We are grateful to the patients, and to the multitude of individuals and teams working together to make this latest approval possible as we strive to do more for more people living with cancer.”
The safety profile of Tafinlar + Mekinist observed in these studies was consistent with the known safety profile in other approved indications.
BRAF mutations have been identified as drivers of cancer growth across a wide range of solid tumors, including in rare cancer types that can be challenging to study in Phase III trials and often have limited treatment options3,4. BRAF V600E is the most common type of BRAF mutation, accounting for up to 90% of BRAF-mutant cancers3.
Full prescribing information for Tafinlar + Mekinist can be found at https://www.novartis.us/sites/www.novartis.us/files/tafinlar.pdf and https://www.novartis.us/sites/www.novartis.us/files/mekinist.pdf.
About Tafinlar + Mekinist
The combination of Tafinlar + Mekinist, the worldwide targeted therapy leader in BRAF/MEK-inhibition research and patients reached, may help to slow tumor growth by blocking signals associated with the BRAF and MEK kinases that are implicated in the growth of various types of cancer1-5. Tafinlar + Mekinist has been studied in more than 6,000 BRAF-positive patients in more than 20 ongoing and completed trials, including in pediatric patients 1 year of age and older, and has been prescribed to more than 200,000 patients worldwide5.
Tafinlar + Mekinist is also approved for use in BRAF V600 mutation-positive unresectable or metastatic melanoma, as an adjuvant treatment for BRAF V600 mutation-positive melanoma after surgery, in BRAF V600 mutation-positive metastatic non-small cell lung cancer, and in BRAF V600 mutation-positive anaplastic thyroid cancer1,2. Tafinlar + Mekinist is not indicated for treatment of patients with colorectal cancer or for treatment of patients with wild-type BRAF solid tumors.
https://www.us.tafinlarmekinist.com/
Find out more at https://www.novartis.com.
Novartis Tafinlar/Mekinist combo gets FDA nod for solid tumors with gene mutation
Jun. 23, 2022 4:42 AM ET
By: Ravikash, SA News Editor
The U.S. Food and Drug Administration (FDA) granted accelerated approval to Novartis' (NYSE:NVS) Tafinlar plus Mekinist to treat patients 6 years of age and older with unresectable or metastatic solid tumors with BRAF V600E mutation who have progressed after prior therapy and have no satisfactory alternative treatment options.
- https://seekingalpha.com/news/3851060-novartis-tafinlarmekinist-combo-gets-fda-nod-for-solid-tumors-with-gene-mutation
- https://seekingalpha.com/symbol/NVS
- https://www.us.tafinlarmekinist.com/
- https://www.novartis.com/
TYVYT® (sintilimab injection)
Innovent and Lilly Jointly Announce the Approval of TYVYT® (sintilimab injection) by China NMPA in Combination with Chemotherapy as First-Line Treatment for Esophageal Squamous Cell Carcinoma
Published on: Jun 20th, 2022
SAN FRANCISCO, U.S., INDIANAPOLIS, U.S., and SUZHOU, China, June 20, 2022 — Innovent Biologics, Inc. (“Innovent”) (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high-quality medicines for the treatment of cancer, autoimmune, metabolic, ophthalmology and other major diseases, and Eli Lilly and Company (“Lilly”, NYSE: LLY) today announced that the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) has approved the supplemental New Drug Application (sNDA) for TYVYT® (sintilimab injection) in combination with cisplatin plus paclitaxel or cisplatin plus 5-fluorouracil chemotherapy for the first-line treatment of unresectable, locally advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC).
This is the fifth NMPA-approved indication of TYVYT®. In China, TYVYT® was approved for: the treatment of relapsed or refractory classical Hodgkin's lymphoma in December 2018; the first-line treatment of non-squamous non-small cell lung cancer (NSCLC) in February 2021; and the first-line treatment of squamous NSCLC as well as the first-line treatment of hepatocellular carcinoma in June 2021.
The new approval was based on the interim analysis of ORIENT-15, a global randomized, double-blind, multi-center Phase 3 clinical trial – which evaluated sintilimab in combination with chemotherapy compared to placebo in combination with chemotherapy as first-line therapy for ESCC. Based on the interim analysis conducted by the Independent Data Monitoring Committee (IDMC), sintilimab in combination with chemotherapy demonstrated a statistically significant improvement in the primary endpoint of overall survival (OS) compared to placebo in combination with chemotherapy regardless of PD-L1 expression status, meeting the pre-defined superior efficacy criteria. Safety profile was consistent with that observed in previously reported studies of sintilimab without new or unexpected safety signals. The results of ORIENT-15 were published in British Medical Journal on April 19, 2022 .
Prof. Shen Lin, Principal Investigator of ORIENT-15 Study, Peking University Cancer Hospital and Institute, stated," Esophageal cancer is one of the most common cancers in China ranking fifth in cancer prevalence and the fourth in mortality cases, with squamous cell carcinoma as most predominant histologic type . In the past, median OS was approximately 10 months for chemotherapy as the first-line standard of care . The results of ORIENT-15 demonstrated that sintilimab plus chemotherapy as the first-line treatment for ESCC significantly improved overall survival (OS) and progression-free survival (PFS) compared to placebo plus chemotherapy, with median OS of 16.7 months（vs. 12.5 months, HR=0.63） and median PFS of 7.2 months（vs. 5.7months,HR=0.56） for sintilimab plus chemotherapy. In addition, the results showed the general applicability of sintilimab with two different chemotherapy regimens1. The approval of sintilimab in combination with chemotherapy as a first-line treatment for ESCC is exciting news and will provide an effective and affordable treatment option for patients living with ESCC in China.”
Dr. Yongjun Liu, President of Innovent, stated,” TYVYT® (sintilimab injection) is the only innovative PD-1 inhibitor with positive Phase 3 studies results as a first-line treatment for five major types of cancer, including the squamous/non-squamous non-small cell lung cancer, liver cancer, gastric cancer and now esophageal cancer. We are encouraged by the results of the ORIENT-15 study, a global multi-center phase 3 trial demonstrating sintilimab as a high quality treatment option with great clinical value for people living with esophageal cancer. Innovent is committed to our mission of developing high-quality biopharmaceuticals that are affordable and contribute to the ‘Healthy China 2030’ Plan for cancer prevention and treatment.”
Dr. Hui Zhou, Senior Vice President of Innovent, stated, “There is a huge unmet clinical need for the first-line treatment of advanced or metastatic ESCC. The results of ORIENT-15 demonstrated that sintilimab can bring significant clinical benefit to the treatment of ESCC. Today, the NMPA of China approval marks another important milestone for sintilimab, and we believe the positive study results will soon translate into superior clinical benefits for ESCC patients. We believe the approval of this new indication will further strengthen the leadership position of TYVYT® (sintilimab injection) and bring hopes to more Chinese cancer patients in broader market.”
Mr. Julio Gay-Ger, President and General Manager of Lilly China, stated, “From Hodgkin’s lymphoma, lung cancer, liver cancer, and now to esophageal squamous cell carcinoma (ESCC), we are excited to see another indication of TYVYT® (sintilimab injection) approved in China in a short of time, bringing new options to Chinese esophageal cancer patients. With our commitment to oncology, Lilly strives to bring high-quality and affordable innovative drugs to Chinese cancer patients through both independent R&D and local partnerships. TYVYT® (sintilimab injection) sets a great example for our partnership with Innovent, and the new approval will further benefit more Chinese cancer patients.”
Dr. Li Wang, Senior Vice President of Lilly China and Head of Lilly China Drug Development and Medical Affairs Center, stated, “The approval of TYVYT® (sintilimab injection) for the first-line indication of esophageal squamous cell carcinoma (ESCC) demonstrated the clinical value of combined immunotherapy in this field. The number of new cases and deaths of esophageal cancer in China accounts for more than half of the world's total2. The ORIENT-15 study, starting from the Chinese ESCC population while having a global perspective, achieved promising results of benefiting the entire population, bringing new options and new hope for the treatment of ESCC patients1.”
About the ORIENT-15 Study
ORIENT-15 is a global randomized, double-blind, multicenter Phase 3 clinical study evaluating sintilimab in combination with chemotherapy (cisplatin plus paclitaxel or 5-fluorouracil [5-FU]), compared to placebo in combination with chemotherapy, for the first-line treatment of unresectable locally advanced, recurrent or metastatic esophageal squamous cell carcinoma (ClinicalTrials.gov, NCT03748134). At the time of interim analysis, a total of 659 eligible patients (of the planned 676 estimated participants) were enrolled and randomly assigned into the experimental group or control group in a 1:1 ratio. The primary endpoints were overall survival (OS) in all randomized patients and OS in PD-L1 positive (defined as CPS ≥10) patients1.
Based on the interim analysis conducted by the Independent Data Monitoring Committee (IDMC), sintilimab in combination with chemotherapy demonstrated a statistically significant improvement in the primary endpoint of overall survival (OS) compared to placebo in combination with chemotherapy, regardless of PD-L1 expression status, meeting the pre-defined superior efficacy criteria. Safety analyses revealed no new safety signals. The results of ORIENT-15 were published in British Medical Journal on April 19, 20221.
About Esophageal Squamous Cell Carcinoma (ESCC)
Esophageal cancer (EC) is one of the most common malignant tumors worldwide that begins in the inner layer (mucosa) of the esophagus, which connects the throat to the stomach. Based on GLOBOCAN 2020 estimates, approximately 600,000 new cases of esophageal cancer are diagnosed and approximately 540,000 deaths result from the disease worldwide each year . Esophageal cancer is the seventh most commonly diagnosed cancer and the sixth leading cause of death from cancer worldwide4. More than half of new and fatal cases of esophageal cancer in the world occur in China2. In China, it is estimated there were approximately 320,000 new cases of esophageal cancer diagnosed and approximately 300,000 deaths resulting from the disease in 20202. Esophageal cancer is the fifth most commonly diagnosed cancer and the fourth leading cause of death from cancer in China, where it has a five-year survival rate of only 30%2.
The two main types of esophageal cancer are squamous cell carcinoma (SCC) and adenocarcinoma. In China, SCC is the predominant histologic type, accounting for more than 90% of all esophageal cancer . In the past, first-line standard systemic therapy was chemotherapy based on platinum drugs for unresectable locally advanced, recurrent or metastatic ESCC, which calls for more effective first-line treatment options. Several PD-1 inhibitors have been approved as first-line treatment in combination with chemotherapy ， .
About Sintilimab
Sintilimab, marketed as TYVYT® (sintilimab injection) in China, is a PD-1 immunoglobulin G4 monoclonal antibodyjointly developed by Innovent and Eli Lilly and Company. Sintilimab is a type of immunoglobulin G4 monoclonal antibody, which binds to PD-1 molecules on the surface of T-cells, blocks the PD-1 / PD-Ligand 1 (PD-L1) pathway, and reactivates T-cells to kill cancer cells . Innovent is currently conducting more than 20 clinical studies of sintilimab to evaluate its safety and efficacy in a wide variety of cancer indications, including more than 10 registrational or pivotal clinical trials.
In China, sintilimab has been approved and included in the National Reimbursement Drug List (NRDL) for four indications, and recently approved for one additional indication including:
• The treatment of relapsed or refractory classic Hodgkin’s lymphoma after two lines or later of systemic chemotherapy;
• In combination with pemetrexed and platinum chemotherapy, for the first-line treatment of non-squamous non-small cell lung cancer lacking EGFR or ALK driver mutations;
• In combination with gemcitabine and platinum chemotherapy, for the first-line treatment of squamous non-small cell lung cancer;
• In combination with BYVASDA® (bevacizumab biosimilar injection) for the first-line treatment of unresectable or advanced hepatocellular carcinoma;
• In combination with cisplatin plus paclitaxel or cisplatin plus 5-fluorouracil for the first-line treatment of esophageal squamous cell carcinoma.
Additionally, Innovent currently has two regulatory submissions under review in the China’s NMPA for sintilimab:
• In combination with chemotherapy for the first-line treatment of unresectable, locally advanced, recurrent or metastatic gastric or gastroesophageal junction adenocarcinoma;
• In combination with bevacizumab biosimilar and chemotherapy for EGFR-mutated non-squamous NSCLC following EGFR-TKI treatment.
Additionally, two clinical studies of sintilimab have met their primary endpoints:
• Phase 2 study as second-line treatment of esophageal squamous cell carcinoma;
• Phase 3 study as second-line treatment for squamous NSCLC with disease progression following platinum-based chemotherap
For more information, please visit: www.innoventbio.com. and www.linkedin.com/company/innovent-biologics/.
To learn more about Lilly, please visit us at www.lilly.com and http://newsroom.lilly.com/social-channels.
Lilly/Innovent Tyvyt gets approval in China for expanded use in esophagus cancer
Jun. 21, 2022 6:35 AM ET
Eli Lilly and Company (LLY), IVBIY
By: Ravikash, SA News Editor
- China's National Medical Products Administration approved the expanded use of Innovent Biologics (OTCPK:IVBIY) and Eli Lilly's (NYSE:LLY) Tyvyt, in combination with certain chemotherapies, to treat esophagus cancer.
- https://seekingalpha.com/news/3849948-lillyinnovent-tyvyt-gets-approval-in-china-for-expanded-use-in-esophagus-cancer
- https://seekingalpha.com/symbol/IVBIY
- https://seekingalpha.com/symbol/LLY
KEYTRUDA® (pembrolizumab)
FDA Accepts Application for Merck’s KEYTRUDA® (pembrolizumab) as Adjuvant Therapy for Stage IB (≥4 centimeters)-IIIA Non-Small Cell Lung Cancer Following Complete Surgical Resection
June 13, 2022 6:45 am ET
Acceptance based on results from the Phase 3 KEYNOTE-091 trial, the seventh positive pivotal study evaluating a KEYTRUDA-based regimen in earlier stages of cancer
RAHWAY, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced the U.S. Food and Drug Administration (FDA) has accepted for review a new supplemental Biologics License Application (sBLA) seeking approval for KEYTRUDA for the adjuvant treatment of patients with stage IB (≥4 centimeters), II or IIIA non-small cell lung cancer (NSCLC) following complete surgical resection. The sBLA is based on data from the pivotal Phase 3 KEYNOTE-091 trial, also known as EORTC-1416-LCG/ETOP-8-15 – PEARLS, conducted in collaboration with the European Organisation for Research and Treatment of Cancer (EORTC) and the European Thoracic Oncology Platform (ETOP). The FDA has set a Prescription Drug User Fee Act (PDUFA), or target action, date of January 29, 2023, however, further data may be provided during the review process that may delay this date.
The study has dual primary endpoints of disease-free survival (DFS) regardless of PD-L1 expression and DFS in patients whose tumors express PD-L1 (tumor proportion score [TPS] ≥50%). At the interim analysis of this study, adjuvant treatment with KEYTRUDA demonstrated a significant improvement in DFS for patients regardless of PD-L1 expression compared to placebo. Disease-free survival in patients whose tumors express PD-L1 (TPS ≥50%) did not reach statistical significance per the pre-specified statistical plan. The trial will continue to analyze DFS in patients whose tumors express high levels of PD-L1 (TPS ≥50%) as well as other secondary endpoints. The safety profile of KEYTRUDA in this study was consistent with that observed in previously reported studies. Results were recently presented at a European Society for Medical Oncology (ESMO) Virtual Plenary.
“KEYTRUDA is foundational in the treatment of metastatic non-small cell lung cancer. The acceptance of our application demonstrates the progress we are making in earlier lines and earlier stages of certain cancers across our oncology portfolio,” said Dr. Eliav Barr, senior vice president, head of global clinical development and chief medical officer, Merck Research Laboratories. “If approved, KEYTRUDA would be the first adjuvant immunotherapy-based option in the U.S. for patients with stage IB (≥4 centimeters) to IIIA non-small cell lung cancer following surgical resection regardless of PD-L1 expression.”
In addition to KEYNOTE-091, six other pivotal trials evaluating a KEYTRUDA-based regimen in patients with earlier stages of cancer met their primary endpoint(s). These trials included: KEYNOTE-716 in stage IIB and IIC melanoma; KEYNOTE-054 in stage III melanoma; KEYNOTE-564 in renal cell carcinoma; KEYNOTE-522 in triple-negative breast cancer; KEYNOTE-629 in cutaneous squamous cell carcinoma; and KEYNOTE-057 in Bacillus Calmette-Guerin-unresponsive, high-risk, non-muscle invasive bladder cancer.
Merck has an extensive clinical development program in lung cancer and is advancing multiple registration-enabling studies, with research directed at earlier stages of disease and novel combinations. Key studies in earlier stages of NSCLC include KEYNOTE-091, KEYNOTE-671, KEYNOTE-867, KEYLYNK-012 and KEYVIBE-006.
About KEYNOTE-091
KEYNOTE-091, also known as EORTC-1416-LCG/ETOP-8-15 – PEARLS, is a randomized, Phase 3 trial (ClinicalTrials.gov, NCT02504372) sponsored by Merck and conducted in collaboration with EORTC and ETOP evaluating KEYTRUDA compared to placebo for the adjuvant treatment of patients with stage IB (≥4 centimeters) to IIIA NSCLC following surgical resection (lobectomy or pneumonectomy) and with adjuvant chemotherapy when indicated. The dual primary endpoints are DFS in the overall population and in patients whose tumors express PD-L1 (TPS ≥50%). Disease-free survival is calculated as the time from randomization to the date of disease recurrence, occurrence of second primary lung cancer, occurrence of second malignancy or death from any cause, whichever occurs first.
The study randomized 1,177 patients (1:1) to receive either KEYTRUDA (200 mg intravenously [IV] every three weeks [Q3W] for one year or maximum 18 doses; n=590); or placebo (IV Q3W for one year or maximum 18 doses; n=587). The median number of doses was 17 for KEYTRUDA and 18 for placebo.
About KEYTRUDA® (pembrolizumab) injection, 100 mg
KEYTRUDA is an anti-programmed death receptor-1 (PD-1) therapy that works by increasing the ability of the body’s immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.
Merck has the industry’s largest immuno-oncology clinical research program. There are currently more than 1,700 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.
Selected KEYTRUDA® (pembrolizumab) Indications in the U.S.
Non-Small Cell Lung Cancer
KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) ≥1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is:
- stage III where patients are not candidates for surgical resection or definitive chemoradiation, or
- metastatic.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS ≥1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.
See additional selected indications for KEYTRUDA in the U.S. after the Selected Important Safety Information
For more information, visit www.merck.com and connect with us on Twitter, Facebook, Instagram, YouTube and LinkedIn.
Merck's Keytruda to get FDA review as adjuvant non-small cell lung cancer treatment
Jun. 13, 2022 7:58 AM ET Merck & Co., Inc. (MRK)FULC
By: Jonathan Block, SA News Editor
- The FDA will review Merck's (NYSE:MRK) supplemental Biologics License Application for Keytruda (pembrolizumab) as an adjuvant treatment for stage IB, II or IIIA non-small cell lung cancer following complete surgical resection.
- https://seekingalpha.com/news/3847929-merck-keytruda-to-get-fda-review-as-adjuvant-non-small-cell-lung-cancer-treatment
- https://seekingalpha.com/symbol/MRK
- https://www.keytruda.com/
- https://www.merck.com/
Hepcludex® (Bulevirtide)
June 23, 2022
Treatment With Hepcludex® (Bulevirtide) Meets Primary Endpoint and Achieves Significant Response in Chronic Hepatitis Delta Virus at 48 Weeks
-- Hepcludex Achieves Significant Viral Declines at Week 48, Reinforcing the Clinical Utility of Sustained Treatment for Hepatitis Delta Virus (HDV) --
FOSTER CITY, Calif.--(BUSINESS WIRE)-- Gilead Sciences, Inc. (Nasdaq: GILD) today announced Week 48 results from the Pivotal Phase 3 clinical trial evaluating the first-in-class entry inhibitor Hepcludex® (bulevirtide) for the treatment of chronic hepatitis delta virus (HDV) infection. Findings from the study underscore the efficacy and safety of bulevirtide for the treatment of chronic HDV and are being presented today in the International Liver Congress™ (ILC) 2022 Official Press Program. In addition, Week 48 data demonstrating the positive impact of bulevirtide on patient-reported outcomes (PROs) in people living with chronic HDV will also be presented. Together, these data reinforce the clinical utility of bulevirtide as monotherapy for the treatment of chronic HDV. There are currently no other approved treatment options for HDV and people living with HDV typically have a poor prognosis.
“As the most severe form of viral hepatitis, HDV presents a significant disease burden with high healthcare-related costs and until recently, no approved treatment options,” said Heiner Wedemeyer, MD, Director, Clinic for Gastroenterology, Hepatology and Endocrinology at Hannover Medical School, and principal investigator of the study. “These results presented at ILC 2022 not only highlight the important clinical role that bulevirtide has to play as a safe and effective treatment option for chronic HDV, but critically also demonstrate that with prolonged treatment, we can achieve higher response rates so we can better manage this rare, life-threatening disease in more people.”
At Week 48, study participants treated with bulevirtide monotherapy at 2 mg or 10 mg once daily achieved a significantly greater combined virological and biochemical response (45% and 48%, respectively) when compared to participants who had not received antiviral treatment at this stage of the study (2%). Combined response was defined as undetectable HDV RNA or a decrease by 2 log10 IU/mL from baseline and ALT normalization. Similarly, when the Week 48 data is considered alongside the integrated Week 24 analyses of the ongoing Phase 2 studies (MYR202 and MYR203) and the interim Week 24 Phase 3 MYR301 data presented at ILC 2021, combined response rates of bulevirtide increased from Week 24 to Week 48, highlighting an improved response of bulevirtide with prolonged treatment.
The safety profile of bulevirtide at Week 48 is consistent with prior reports, with no participants having an adverse event (AE) leading to discontinuation of bulevirtide and no serious AEs attributed to bulevirtide treatment. The safety profile at both Week 24 and again at Week 48 reassert the safety and tolerability profile of bulevirtide, which is an important factor for people living with chronic HDV.
“The consequences of living with a serious and chronic condition like HDV can have a profound impact on the individual’s quality of life, affecting the physical and mental well-being of people living with HDV,” said Anu Osinusi, Vice President, Clinical Research for Hepatitis, Respiratory and Emerging Viruses at Gilead. “What we now see is that treatment with bulevirtide not only improves clinical measures associated with viral control but can significantly improve, and maintain, a range of quality-of-life markers in people living with HDV, ultimately improving the overall management of the condition.”
In an oral presentation, Gilead will also share an exploratory analysis of the Week 48 data from the Phase 3 MYR301 study, evaluating the impact of bulevirtide (2 mg once daily) on PROs in people living with chronic HDV. Participants treated with bulevirtide 2 mg (n=49) showed significant improvements from baseline at 48 weeks in almost all assessed health-related quality-of-life domains of the Hepatitis Quality of Life questionnaire. Participants in the control group (n=51) remained largely unchanged, apart from significant improvements in health distress and hepatitis-specific health distress. Of note, participants receiving bulevirtide 2 mg reported significant improvements in performance of daily activities related to hepatitis, emotional impact of hepatitis and improvement in work compared with controls.
Bulevirtide was granted Conditional Marketing Authorization by the European Commission and is an investigational agent in the U.S. and outside of the European Economic Area. In these regions, health authorities have not established the safety and efficacy of bulevirtide. A Biologics License Application (BLA) was submitted in Q4 2021 to the U.S. Food and Drug Administration (FDA) for bulevirtide for injection (2 mg) to treat adults with HDV and compensated liver disease. This Phase 3 data is included in the filing of bulevirtide to the FDA. Bulevirtide has been granted Breakthrough Therapy and Orphan Drug designations by the FDA. In 2020 bulevirtide 2 mg was granted Conditional Marketing Authorization by the European Commission and PRIority MEdicines (PRIME) scheme eligibility by the European Medicines Agency as the first approved treatment in Europe for adults with chronic HDV and compensated liver disease.
About MYR301
MYR301 is an ongoing Phase 3 clinical trial evaluating the long-term efficacy and safety of bulevirtide in 150 people living with chronic HDV randomly allocated to treatment with bulevirtide 2 mg once daily (n=49), 10 mg once daily (n=50) or no antiviral treatment (delayed treatment, n=51). Primary efficacy and safety data will be assessed at Week 48. After Week 48, participants in the delayed treatment group of the study will be switched to bulevirtide 10 mg once daily for an additional 96 weeks. The total duration of treatment across all groups in the study is 144 weeks. The primary endpoint, combined response, is defined as an undetectable HDV RNA (<LoD (Limit of Detection)) or ≥2log10 IU/ml decline from baseline and ALT normalization at Week 48. Secondary endpoints at Week 48 include undetectable HDV RNA (key secondary endpoint), ALT normalization, and a change from baseline in liver stiffness measured by transient elastography.
About Gilead Sciences
Gilead Sciences, Inc. is a biopharmaceutical company that has pursued and achieved breakthroughs in medicine for more than three decades, with the goal of creating a healthier world for all people. The company is committed to advancing innovative medicines to prevent and treat life-threatening diseases, including HIV, viral hepatitis and cancer. Gilead operates in more than 35 countries worldwide, with headquarters in Foster City, California.
Hepcludex, Gilead and the Gilead logo are registered trademarks of Gilead Sciences, Inc., or its related companies.
For more information about Gilead, please visit the company’s website at www.gilead.com, follow Gilead on Twitter (@Gilead Sciences) or call Gilead Public Affairs at 1-800-GILEAD-5 or 1-650-574-3000.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220623005008/en/
Source: Gilead Sciences, Inc.
Gilead's Hepcludex achieves primary endpoint in late-stage trial for hepatitis D
Jun. 23, 2022 9:14 AM ET
By: Jonathan Block, SA News Editor1 Comment
Gilead Sciences' (NASDAQ:GILD) Hepcludex (bulevirtide) achieved its primary endpoint at 48 weeks in a phase 3 trial for chronic hepatitis D.
- https://seekingalpha.com/news/3851191-gilead-hepcludex-achieves-primary-endpoint-in-late-stage-trial-for-hepatitis-d
- https://seekingalpha.com/symbol/GILD
- https://www.gilead.com/
VAXNEUVANCE™ (Pneumococcal 15-valent Conjugate Vaccine)
U.S. FDA Approves Merck’s VAXNEUVANCE™ (Pneumococcal 15-valent Conjugate Vaccine) for the Prevention of Invasive Pneumococcal Disease in Infants and Children
June 22, 2022 6:45 am ET
Clinical data supporting approval demonstrated non-inferior immune responses for all serotypes shared with PCV13 following a four-dose series and superior immune responses for important disease-causing shared serotype 3 and unique serotypes 22F and 33F compared to PCV13
With this expanded indication, VAXNEUVANCE is the first pneumococcal conjugate vaccine approved in almost a decade to help protect pediatric populations against invasive pneumococcal disease
RAHWAY, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, announced today that the U.S. Food and Drug Administration (FDA) has approved an expanded indication for VAXNEUVANCE™(Pneumococcal 15-valent Conjugate Vaccine) (pronounced VAKS-noo-vans) to include children 6 weeks through 17 years of age. VAXNEUVANCE is now indicated for active immunization for the prevention of invasive disease caused by Streptococcus pneumoniae serotypes 1, 3, 4, 5, 6A, 6B, 7F, 9V, 14, 18C, 19A, 19F, 22F, 23F and 33F in individuals 6 weeks of age and older. The approval follows the FDA’s Priority Review of Merck’s supplemental application. VAXNEUVANCE is contraindicated for individuals with a severe allergic reaction (e.g., anaphylaxis) to any component of VAXNEUVANCE or to diphtheria toxoid; see additional Select Safety Information below.
The U.S. Centers for Disease Control and Prevention’s (CDC) Advisory Committee on Immunization Practices (ACIP) is expected to meet today to discuss and make recommendations on the use of VAXNEUVANCE in pediatric populations.
“Despite decreases in incidence of invasive pneumococcal disease in children, certain key serotypes continue to cause serious illness that can lead to death in children under the age of 5, with serotypes 3, 22F and 33F responsible for more than a quarter of all invasive pneumococcal disease cases in this population,” said Dr. Steven Shapiro, chairman, department of pediatrics, Jefferson Abington Hospital, and investigator for the PNEU-PED trial. “With the robust clinical data supporting VAXNEUVANCE and this FDA approval, VAXNEUVANCE will be an important new option to help advance protection for children.”
Invasive pneumococcal disease (IPD) is an infection caused by the bacterium Streptococcus pneumoniae, or pneumococcus. While there are approximately 100 different types of S. pneumoniae, called serotypes, a smaller number of serotypes are responsible for IPD in children. Serotypes 3, 22F and 33F are three of the top five serotypes causing childhood cases of IPD. IPD can lead to hospitalization or death. Some examples of IPD are bacteremia (an infection in the blood) and meningitis (an infection of the coverings of the brain and spinal cord), which can also result in long-term neurological complications. Children under the age of 2 are particularly vulnerable to IPD.
The FDA’s approval was based on data from seven randomized, double-blind clinical studies assessing safety, tolerability and immunogenicity of VAXNEUVANCE in infants, children and adolescents (see “Clinical Data Supporting FDA Approval” below for additional details). Clinical data from the pivotal study showed that immune responses elicited by VAXNEUVANCE following a four-dose pediatric series were non-inferior to the currently available 13-valent pneumococcal conjugate vaccine (PCV13) for the 13 shared serotypes based on serotype-specific immunoglobulin G (IgG) geometric mean concentrations (GMCs).
In a secondary analysis, immune responses for VAXNEUVANCE following a four-dose pediatric series were superior to PCV13 for shared serotype 3 and the two serotypes unique to VAXNEUVANCE, 22F and 33F. Randomized controlled trials assessing the clinical efficacy of VAXNEUVANCE compared to PCV13 have not been conducted.
Data from the clinical program also support the use of VAXNEUVANCE concomitantly with other commonly administered routine pediatric vaccines, and in a variety of clinical settings, such as interchangeable use following initiation of an infant vaccination schedule with PCV13 or in a catch-up setting for older children who are either pneumococcal vaccine-naïve or who previously received an incomplete series of another PCV. Additionally, data support the use of VAXNEUVANCE in special populations, such as in preterm infants and children living with HIV infection or sickle cell disease.
“Our goal with VAXNEUVANCE is to expand coverage of key invasive disease-causing serotypes and provide a strong immune response to serotypes that pose substantial risk to infants and children,” said Dr. Eliav Barr, senior vice president, head of global clinical development and chief medical officer, Merck Research Laboratories. “With this approval, we bring forward our first pediatric pneumococcal conjugate vaccine – and the first pediatric pneumococcal conjugate vaccine to be approved in almost a decade – building on our commitment to preventing invasive pneumococcal disease and on our legacy in pediatric vaccine development. We thank the investigators and the families of our clinical trial participants for participating in the research studies and the role they played in this milestone.”
About VAXNEUVANCE (Pneumococcal 15-valent Conjugate Vaccine)
VAXNEUVANCE, Merck’s 15-valent pneumococcal conjugate vaccine, consists of purified capsular polysaccharides from S.pneumoniae serotypes 1, 3, 4, 5, 6A, 6B, 7F, 9V, 14, 18C, 19A, 19F, 22F, 23F and 33F individually conjugated to CRM197 carrier protein. VAXNEUVANCE is indicated for active immunization of individuals 6 weeks of age and older for the prevention of invasive disease caused by the S. pneumoniae serotypes contained in the vaccine. The FDA initially approved VAXNEUVANCE in July 2021. The FDA previously granted VAXNEUVANCE Breakthrough Therapy designation and Priority Review for the pediatric indication.
Clinical data supporting FDA approval of VAXNEUVANCE in children 6 weeks through 17 years of age
VAXNEUVANCE was approved for use in infants and children based on data from seven randomized, double-blind clinical studies designed to evaluate its safety, tolerability and immunogenicity. These clinical studies included:
- Children receiving a four-dose series. The pivotal Phase 3, multicenter, randomized, double-blind, active comparator-controlled study evaluated the safety, tolerability and immunogenicity of a four-dose series of VAXNEUVANCE in healthy infants (n=1720) (V114-029/PNEU-PED [NCT03893448]). In the study, participants were randomized one-to-one to receive a four-dose series of VAXNEUVANCE or PCV13 at 2, 4, 6, and 12-15 months of age.
The assessed immune responses included serotype specific IgG response rates against capsular polysaccharides of S. pneumoniae at 30 days post-dose 3 (PD3) and IgG geometric mean concentrations (GMCs) at 30 days PD3 and post-dose 4 (PD4). Additionally, antibody responses to other routine licensed pediatric vaccines were evaluated when administered concomitantly with VAXNEUVANCE or PCV13.
VAXNEUVANCE elicited immune responses for all 15 serotypes contained in the vaccine. Based on serotype-specific IgG GMCs, at 30 days PD4, VAXNEUVANCE was non-inferior to PCV13 for all 13 shared serotypes and the two serotypes unique to VAXNEUVANCE, 22F and 33F. Serotypes 22F and 33F were compared with serotype 4, which had the lowest IgG GMC of all shared serotypes in PCV13, excluding serotype 3. Results of the secondary analysis showed superior immune responses for VAXNEUVANCE in comparison to PCV13 for serotypes 3, 22F and 33F.
- PCV interchangeability. A Phase 3, randomized, double-blind, active comparator-controlled, descriptive study evaluated the interchangeability of VAXNEUVANCE and PCV13 with respect to safety, tolerability and immunogenicity in healthy infants (n=900) (V114-027/PNEU-DIRECTION [NCT03620162]). The study demonstrated generally comparable immune responses for participants completing the vaccination series with VAXNEUVANCE compared to those who completed series with PCV13, for the 13 shared serotypes. The safety profile observed when VAXNEUVANCE was used to complete a four-dose pneumococcal conjugate vaccine series initiated with PCV13 was similar to the safety profile following a complete four-dose regimen of either VAXNEUVANCE or PCV13.
- Use as part of a catch-up series. A Phase 3, randomized, double-blind, active comparator-controlled, descriptive study evaluated the safety, tolerability and immunogenicity of catch-up vaccination regimens of VAXNEUVANCE in healthy infants, children and adolescents 7 months to 17 years of age (n=606) (V114-024/PNEU-PLAN [NCT03885934]). The study demonstrated generally comparable immune responses after receipt of the last dose for the 13 serotypes targeted by VAXNEUVANCE and PCV13, and higher immune responses for the two serotypes unique to VAXNEUVANCE, 22F and 33F.
- https://www.merckvaccines.com/vaxneuvance-adult/
For more information, visit www.merck.com and connect with us on Twitter, Facebook, Instagram, YouTube and LinkedIn.
Please see Prescribing Information for VAXNEUVANCE (Pneumococcal 15-valent Conjugate Vaccine) at https://www.merck.com/product/usa/pi_circulars/v/vaxneuvance/vaxneuvance_pi.pdfand Patient Information/Medication Guide for VAXNEUVANCE at https://www.merck.com/product/usa/pi_circulars/v/vaxneuvance/vaxneuvance_ppi.pdf.
Source: Merck & Co., Inc.
Merck wins FDA approval for pneumococcal vaccine to include children
Jun. 22, 2022 7:02 AM ET
By: Dulan Lokuwithana, SA News Editor
- Merck (NYSE:MRK) announced on Wednesday that the FDA approved an expanded indication for Vaxneuvance, a 15-valent pneumococcal conjugate vaccine for the prevention of invasive pneumococcal disease in infants and children aged 6 weeks – 17 years.
- https://seekingalpha.com/news/3850646-merck-wins-fda-approval-for-pneumococcal-vaccine-to-include-children
- https://seekingalpha.com/symbol/MRK
VENCLYXTO®/VENCLEXTA®
June 10, 2022
New Data Demonstrates AbbVie's VENCLYXTO®/VENCLEXTA® Combination Sustained Progression-Free Survival in Chronic Lymphocytic Leukemia Patients After Four Years Off Treatment
- Five-year follow-up analysis of the Phase 3 CLL14 trial (median follow-up of 65.4 months) continues to demonstrate longer progression-free survival (PFS) after fixed-duration venetoclax plus obinutuzumab compared to fixed-duration chlorambucil plus obinutuzumab in previously untreated patients with chronic lymphocytic leukemia and coexisting conditions
- PFS was significantly superior for venetoclax plus obinutuzumab compared to chlorambucil plus obinutuzumab (median not reached [NR] vs. 36.4 months, p<0.0001)
- A majority of patients treated with venetoclax and obinutuzumab remain without relapse four years after completing the venetoclax-based combination treatment
NORTH CHICAGO, Ill., June 10, 2022 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced five-year follow-up results from the Phase 3 CLL14 trial, finding that over 60 percent of patients with previously untreated chronic lymphocytic leukemia (CLL) who had received one-year fixed-duration combination treatment of VENCLYXTO®/VENCLEXTA® (venetoclax) plus obinutuzumab (GAZYVA®) continued to show longer progression-free survival (PFS) and higher rates of undetectable minimal residual disease (MRD) after four years off treatment.1 The findings were presented at the 2022 European Hematology Association (EHA) Annual Congress (Abstract #S148)."Long-term data from the CLL14 trial show that the one-year fixed-duration combination regimen of venetoclax and obinutuzumab offers patients the possibility of four years of CLL treatment-free response without disease progression," said Mohamed Zaki, M.D., Ph.D., vice president and global head of oncology clinical development, AbbVie. "Since its approval, this chemotherapy-free combination option has helped transform the therapeutic landscape for CLL."Data shows that after more than five years of median follow-up (65.4 months), PFS remained significantly superior among patients treated with the VENCLYXTO/VENCLEXTA and obinutuzumab combination compared to the chlorambucil and obinutuzumab chemotherapy regimen (n=432; median NR vs 36.4 months; hazard ratio [HR] 0.35 [95% CI 0.26-0.46], p<0.0001). The therapies were administered for a fixed-duration of 12 months for VENCLYXTO/VENCLEXTA in combination with six cycles of obinutuzumab. At five years after randomization, the estimated PFS rate after one-year fixed-duration treatment was 62.6 percent for the VENCLYXTO/VENCLEXTA-based combination compared to 27.0 percent for the chlorambucil combination 1 The improvement in PFS was maintained across all risk groups, including patients with TP53 mutation/deletion and unmutated IGHV status.1Among the secondary endpoints, patients were assessed for MRD in peripheral blood and/or bone marrow, using next generation sequencing. Undetectable MRD (uMRD) was defined as less than one CLL cell being identified per 10,000 lymphocytes sampled. Four years after treatment completion, 18.1 percent of patients treated with the VENCLYXTO/VENCLEXTA-based combination still had uMRD compared to 1.9 percent of patients in the chlorambucil combination study arm.1The estimated overall survival (OS) rate was 81.9 percent in the VENCLYXTO/VENCLEXTA-based combination and 77.0 percent in the chlorambucil combination group (HR 0.72 [0.48-1.09], p=0.12) at five years after randomization.1No new safety signals were observed in the five-year follow-up analysis. The most frequently occurring serious adverse reactions (>=2%) in patients receiving the VENCLYXTO/VENCLEXTA-based combination were pneumonia, sepsis, febrile neutropenia, and tumor lysis syndrome.1"Four years following treatment completion, we are pleased to report that approximately three out of five patients who received the fixed-duration combination treatment with venetoclax have remained progression free," said Othman Al-Sawaf, M.D., investigator in the CLL14 study, hematologist-oncologist at the University Hospital Cologne in Germany, and study physician at the German CLL Study Group. "Additionally, it is notable that the population of patients who received chlorambucil combination was observed to have slightly more than twice the rate of progression events, compared to the patients who received the venetoclax combination."VENCLYXTO/VENCLEXTA is being developed by AbbVie and Roche. It is jointly commercialized by AbbVie and Genentech, a member of the Roche Group, in the U.S. and by AbbVie outside of the U.S
.About the CLL14 Phase 3 Trial3,4,5
The prospective, multicenter, open-label, randomized Phase 3 CLL14 trial, which was conducted in close collaboration with the German CLL Study Group (DCLLSG), evaluated the efficacy and safety of a combined regimen of VENCLYXTO/VENCLEXTA and obinutuzumab (n=216) versus obinutuzumab and chlorambucil (n=216) in previously untreated patients with CLL and coexisting medical conditions (total Cumulative Illness Rating Scale [CIRS] score >6 or creatinine clearance <70 mL/min). The therapies were administered for a fixed-duration of 12 months for VENCLYXTO/VENCLEXTA in combination with six cycles of obinutuzumab. The trial enrolled 432 patients, all of whom were previously untreated, according to the International Workshop on Chronic Lymphocytic Leukemia (iwCLL) criteria. Efficacy was based on PFS, as assessed by an independent review committee.Key secondary endpoints were rates of MRD in peripheral blood and bone marrow, overall and complete response rates, MRD in complete response in peripheral blood and bone marrow, and overall survival.In patients with CLL receiving venetoclax combination therapy with obinutuzumab, serious adverse reactions (ARs) were most often due to febrile neutropenia and pneumonia (5 percent each). The most common ARs (≥20 percent) of any grade were neutropenia (60 percent), diarrhea (28 percent), and fatigue (21 percent). Fatal ARs that occurred in the absence of disease progression and with onset within 28 days of the last study treatment were reported in 2 percent (4/212) of patients, most often from infection.
About VENCLYXTO® (venetoclax)
VENCLYXTO® (venetoclax) is a first-in-class medicine that selectively binds and inhibits the B-cell lymphoma-2 (BCL-2) protein. In some blood cancers, BCL-2 prevents cancer cells from undergoing their natural death or self-destruction process, called apoptosis. VENCLYXTO targets the BCL-2 protein and works to help restore the process of apoptosis.VENCLYXTO is being developed by AbbVie and Roche. It is jointly commercialized by AbbVie and Genentech, a member of the Roche Group, in the U.S. and by AbbVie outside of the U.S. Together, the companies are committed to BCL-2 research and to studying venetoclax in clinical trials across several blood and other cancers. Venetoclax is approved in more than 80 countries, including the U.S.
Indication and Important VENCLYXTO (venetoclax) EU Safety Information4IndicationsVenclyxto in combination with obinutuzumab is indicated for the treatment of adult patients with previously untreated chronic lymphocytic leukaemia (CLL).Venclyxto in combination with rituximab is indicated for the treatment of adult patients with CLL who have received at least one prior therapy.Venclyxto monotherapy is indicated for the treatment of CLL:
- In the presence of 17p deletion or TP53 mutation in adult patients who are unsuitable for or have failed a B-cell receptor pathway inhibitor, or
- In the absence of 17p deletion or TP53 mutation in adult patients who have failed both chemoimmunotherapy and a B-cell receptor pathway inhibitor.
Venclyxto in combination with a hypomethylating agent is indicated for the treatment of adult patients with newly diagnosed acute myeloid leukaemia (AML) who are ineligible for intensive chemotherapy.
Contraindications
Hypersensitivity to the active substance or to any of the excipients is contraindicated. Concomitant use of strong CYP3A inhibitors at initiation and during the dose-titration phase due to increased risk for tumour lysis syndrome (TLS). Concomitant use of preparations containing St. John's wort as Venclyxto efficacy may be reduced
For more information about AbbVie, please visit us at www.abbvie.com. Follow @abbvie on Twitter, Facebook, Instagram, YouTube and LinkedIn.
AbbVie presents 5-year follow-up data for its CLL combination treatment developed with Roche
Jun. 10, 2022 9:50 AM ETAbbVie Inc. (ABBV)RHHBY, RHHBF
By: Anuron Mitra, SA News Editor1 Comment
- AbbVie (NYSE:ABBV) on Friday announced five-year follow-up results from its phase 3 CLL14 trial in patients with chronic lymphocytic leukemia (CLL), a type of cancer in which the bone marrow makes too many lymphocytes, which is a type of white blood cell.
- https://seekingalpha.com/news/3847680-abbvie-presents-5-year-follow-up-data-for-its-cll-combination-treatment-developed-with-roche
- https://seekingalpha.com/symbol/ABBV
- https://seekingalpha.com/symbol/RHHBY
- https://www.venclexta.com/
- http://www.abbvie.com/oncology
- https://www.abbvie.com/
ObsEva Announces European Commission Marketing Authorization for Yselty® (linzagolix), an Oral GnRH Antagonist, for the Treatment of Uterine Fibroids
June 17, 2022
-Yselty® (linzagolix) is the first and only approved GnRH antagonist to provide flexible dosing options with and without hormonal add-back therapy-
-Theramex to commercialize Yselty®; ObsEva to receive royalties on commercial sales, as well as development, commercial, and sales-based milestone payments–
-In the United States, the New Drug Application (NDA) for linzagolix is currently under review by the Food and Drug Administration (FDA), with a Prescription Drug User Fee Act (PDUFA) target action date of September 13, 2022-
Ad hoc announcement pursuant to Art. 53 LR of the SIX Swiss Exchange
GENEVA, Switzerland – June 17, 2022 – ObsEva SA (NASDAQ: OBSV; SIX: OBSN), a biopharmaceutical company developing and commercializing novel therapies for women’s health, today announced that the European Commission (EC) has granted marketing authorization for Yselty® (linzagolix), an oral GnRH antagonist, for the management of moderate to severe symptoms of uterine fibroids (UF) in adult women (over 18 years of age) of reproductive age.
The EC decision follows confirmation of a positive opinion from the Committee for Medicinal Products for Human Use of the European Medicines Agency in April 2022, and is valid in all 27 European Union Member States, as well as Iceland, Norway, and Liechtenstein.
Yselty® is the only approved oral GnRH antagonist to offer flexible dosing options, with and without additional hormonal therapy, for women suffering from UF. For women with UF for whom hormonal add-back therapy (ABT, estradiol 1 mg and norethisterone acetate 0.5 mg) is appropriate, Yselty® offers a potentially best-in-class efficacy rate 1,2,3 and favourable tolerability profile. For women with UF who cannot or do not want to take hormones, Yselty® is the first and only approved oral GnRH antagonist with a non-hormonal dosing option. Yselty® is approved in the EU at the following doses:
- 100 mg or 200 mg once daily with hormonal ABT, with no limitation in treatment duration
- 100 mg once daily for women in whom ABT is not recommended or who prefer to avoid hormonal therapy, with no limitation in treatment duration
- 200 mg once daily for short-term use (< 6 months) in clinical situations when reduction of uterine and fibroid volume is desired
The approval is based on positive data from the Company’s two Phase 3 PRIMROSE trials. The pooled week 24 data from these studies support a potentially best-in-class profile, with a responder rate of 84.5% in women receiving linzagolix 200 mg with hormonal ABT, and 56.5% in women receiving linzagolix 100 mg without ABT.
“As the first and only approved GnRH antagonist to provide flexible dosing options with and without hormonal add-back therapy, Yselty® has the potential to transform the treatment paradigm and significantly advance medical options for women in the EU with uterine fibroids,” said Dr. Brandi Howard, Chief Clinical Officer of ObsEva. “We are pleased to be the first to provide women and doctors with a non-hormonal dosing option for the millions of women who either have contraindications to or a personal preference to avoid the use of hormonal add-back therapy, while also providing dosing options for women for whom hormonal add-back therapy is appropriate.”
Brian O’Callaghan, CEO of ObsEva, commented, “Our first approval marks a major milestone for ObsEva and further validates our work to address one of the most challenging unmet needs facing women. As we transition to a commercial stage company, our agreement with Theramex provides a strong foundation to realize the potential for linzagolix across key markets, and we look forward to commercial launch in Europe.”
In February 2022, ObsEva entered into a strategic licensing agreement with Theramex, a global leader in women’s health, to support the commercialization and market introduction of linzagolix across international markets outside of the U.S., Canada, and Asia. Under the terms of the agreement, ObsEva is entitled to receive royalties of a mid-thirties percentage on commercial sales, which includes the cost of goods sold to Theramex. Furthermore, the agreement contains up to EUR72.75 million in upfront and milestone payments, including up to EUR13.75 million in development and commercial milestones and up to EUR54 million in sales-based milestones.
The NDA for linzagolix in the U.S. is currently under review by the FDA, with a PDUFA target action date of September 13, 2022.
About Yselty® (linzagolix)
Linzagolix is a novel, once daily, oral GnRH receptor antagonist with a potentially best-in-class profile 1,2,3. Linzagolix was developed to offer flexible dosing options with and without hormonal add-back therapy to women suffering from uterine fibroids, and is approved in the EU. For women with uterine fibroids for whom hormonal add-back therapy is appropriate, linzagolix has the potential to offer a best-in-class efficacy rate and favourable tolerability profile. For women with uterine fibroids who cannot or do not want to take hormones, linzagolix is the first and only approved oral GnRH antagonist with a non-hormonal dosing option in the EU. ObsEva licensed linzagolix from Kissei in late 2015 and retains worldwide commercial rights, excluding Asia, for the product. Linzagolix is currently under review by the FDA, with a PDUFA target action date of September 13, 2022.
About ObsEva
ObsEva is a biopharmaceutical company developing and commercializing novel therapies to improve women’s reproductive health and pregnancy. The Company’s first, just recently approved drug is Yselty® (linzagolix), a once daily, oral GnRH receptor antagonist that was developed to offer flexible dosing options to women suffering from uterine fibroids, and is approved in the EU. Through strategic in-licensing and disciplined drug development, ObsEva has established a late-stage clinical pipeline with development programs focused on new therapies for the treatment of uterine fibroids, endometriosis, and preterm labor. ObsEva is listed on the Nasdaq Global Select Market and is traded under the ticker symbol “OBSV” and on the SIX Swiss Exchange where it is traded under the ticker symbol “OBSN”. For more information, please visit www.ObsEva.com
About Theramex
Theramex is a leading global speciality pharmaceutical company dedicated to women and their health. Theramex supports women at every stage of their lives by providing a broad portfolio of innovative and established brands covering contraception, fertility, menopause and osteoporosis. Theramex’s commitment is to listen to and understand its patients, serve their needs and offer healthcare solutions to help improve their lives. Theramex’s vision is to be a lifetime partner for women and the healthcare professionals who treat them by providing patient-focused and effective solutions that care for and support women through every stage of life.
About Kissei
Linzagolix has been discovered by Central Research Laboratories of Kissei Pharmaceutical Co., Ltd. Kissei is a Japanese pharmaceutical company based on the management philosophy “contributing to society through high-quality, innovative pharmaceutical products” and “serving society through our employees.” As a strong R&D-oriented corporation, it concentrates on providing innovative pharmaceuticals to patients worldwide in the focus fields of urology, nephrology/dialysis, gynecology and rare/intractable diseases.
ObsEva granted European nod for uterine fibroids therapy
Jun. 17, 2022 7:51 AM ET ObsEva SA (OBSV)
By: Dulan Lokuwithana, SA News Editor
- Women’s health company ObsEva (NASDAQ:OBSV) announced on Friday that the European Commission (EC) granted marketing authorization for Yselty as a treatment for symptoms related to uterine fibroids (UF) in adult women of reproductive age.
- https://seekingalpha.com/news/3849531-obseva-granted-european-nod-for-uterine-fibroids-therapy
- https://seekingalpha.com/symbol/OBSV
- https://www.obseva.com/linzagolix/
SKYRIZI® (risankizumab-rzaa)
June 17, 2022
SKYRIZI® (risankizumab-rzaa) Receives FDA Approval as the First and Only Specific Interleukin-23 (IL-23) to Treat Moderately to Severely Active Crohn's Disease in Adults
- Third approved indication for SKYRIZI (risankizumab-rzaa) is supported by safety and efficacy data from two induction and one maintenance clinical trials evaluating SKYRIZI in moderately to severely active Crohn's disease, ADVANCE, MOTIVATE and FORTIFY1-4
- As early as week 4 in the induction studies, clinical response and clinical remission were achieved by significantly more subjects treated with SKYRIZI versus placebo, as were co-primary endpoints of endoscopic response and clinical remission at week 12 and week 521-4
- Crohn's disease is chronic, systemic and progressive; most patients experience unpredictable symptoms that have a significant impact on daily life5-8
NORTH CHICAGO, Ill., June 17, 2022 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced that the U.S. Food and Drug Administration (FDA) has approved SKYRIZI® (risankizumab-rzaa) as the first and only specific interleukin-23 (IL-23) inhibitor for the treatment of adults with moderately to severely active Crohn's disease (CD).4 In two induction and one maintenance clinical trials, SKYRIZI demonstrated significant improvements in endoscopic response (defined as a decrease of greater than 50% from the baseline Simple Endoscopic Score in CD [SES-CD] or for patients with isolated ileal disease and SES-CD of 4, at least a 2-point reduction from baseline) and clinical remission (defined as a Crohn's Disease Activity Index [CDAI] of less than 150), compared to placebo, as both an induction and maintenance therapy.4
Experience the interactive Multimedia News Release here: https://www.multivu.com/players/English/8978352-abbvie-fda-crohns-disease/
"We are proud to offer the first new treatment option in six years for moderately to severely active CD, which may provide patients with a meaningful level of endoscopic improvement," said Thomas Hudson, M.D., senior vice president, research and development, chief scientific officer, AbbVie. "With more than 30 ongoing or planned trials in inflammatory bowel disease, AbbVie is committed to advancing the standards of care for patients by exploring and investing in research for those living with immune-mediated, gastroenterological conditions."
The dosing regimen for SKYRIZI for the treatment of CD is 600 mg administered by intravenous infusion over at least one hour at week 0, week 4, and week 8, followed by 360 mg self-administered by subcutaneous injection (SC) with an on-body injector (OBI) at week 12, and every 8 weeks thereafter.4 A 180 mg self-administered SC maintenance dose option remains under review by the FDA.
Endoscopic and Clinical Outcomes1-4
The co-primary endpoints of the clinical trials were endoscopic response and clinical remission. In the 12-week induction studies, ADVANCE and MOTIVATE, a significantly greater proportion of patients treated with SKYRIZI achieved endoscopic response and clinical remission compared to placebo. As early as week 4, clinical response (defined as a 100-point reduction in CDAI) and clinical remission were achieved in a significantly greater proportion of patients receiving SKYRIZI as compared to placebo.
In the 52-week maintenance trial, FORTIFY, a significantly greater proportion of patients achieved the co-primary endpoints of endoscopic response and clinical remission as compared with the placebo group (risankizumab induction responders) after one year.
"In both the induction and maintenance clinical trials, a significantly greater number of adult patients saw few or no symptoms and a meaningful reduction of visible signs of intestinal inflammation, compared to placebo," said Marla Dubinsky, M.D., chief, division of pediatric gastroenterology for the Mount Sinai Health System, co-director of the Susan and Leonard Feinstein IBD Center at Mount Sinai.* "This approval provides healthcare professionals with a greatly needed additional option for treating the disruptive symptoms of Crohn's disease."
About the ADVANCE and MOTIVATE Studies1-2
The ADVANCE and MOTIVATE studies are Phase 3, multicenter, randomized, double-blind, placebo-controlled induction studies designed to evaluate the efficacy and safety of two doses of risankizumab, 600 mg and 1200 mg, in adults with moderately to severely active Crohn's disease, compared to placebo. Both studies included different sets of primary and secondary endpoints for outside U.S. (OUS) protocol and U.S. protocol. The primary endpoints were achievement of clinical remission (per PRO-2 for the OUS protocol, which was measured by daily stool frequency and abdominal pain score, and per CDAI for the U.S. protocol, which was measured by a CDAI score less than 150) and endoscopic response (for both protocols) at week 12. Endoscopic response is defined as a decrease of greater than 50% from the baseline SES-CD or for patients with isolated ileal disease and SES-CD of 4, at least a 2-point reduction from baseline, as scored by a central reviewer.
The ADVANCE study included a mixed population of patients who had responded inadequately or were intolerant to conventional and/or biologic therapy. The MOTIVATE study evaluated patients who had responded inadequately or were intolerant to biologic therapy. Topline results of the studies were shared in January 2021. More information can be found on www.clinicaltrials.gov (ADVANCE: NCT03105128; MOTIVATE: NCT03104413).
About the FORTIFY Study3
The FORTIFY study is a Phase 3, multicenter, randomized, double-blind, control group, 52-week maintenance study designed to evaluate the efficacy and safety of risankizumab 180 mg and 360 mg as maintenance therapy versus withdrawal in patients who responded to risankizumab induction treatment in the ADVANCE and MOTIVATE studies. This study included different sets of primary and secondary endpoints for the OUS analysis plan and U.S. analysis plan due to regulatory requirements in the different regions. The co-primary endpoints were achievement of endoscopic response and clinical remission at week 52. Endoscopic response is defined as a decrease of greater than 50% from the baseline SES-CD or for patients with isolated ileal disease and SES-CD of 4, at least a 2-point reduction from baseline, as scored by a central reviewer. Clinical remission is defined by SF/AP, which was measured by daily stool frequency and abdominal pain score, in the OUS analysis plan and defined by CDAI, which was measured by a CDAI score less than 150, in the U.S. analysis plan.
Topline results were announced in June 2021. An open label extension of FORTIFY will continue to assess the long-term safety of risankizumab in subjects who completed participation in FORTIFY. More information can be found on www.clinicaltrials.gov (NCT03105102).
*Dr. Dubinsky is a paid consultant and advisor for AbbVie.
About SKYRIZI® (risankizumab-rzaa)
SKYRIZI is an interleukin-23 (IL-23) inhibitor that selectively blocks IL-23 by binding to its p19 subunit.9,10 IL-23, a cytokine involved in inflammatory processes, is thought to be linked to a number of chronic immune-mediated diseases, including Crohn's disease.10 The approved dose to treat adults with moderately to severely active Crohn's disease is 600 mg administered by intravenous infusion over at least one hour at week 0, week 4, and week 8, followed by 360 mg administered by subcutaneous injection at week 12, and every 8 weeks thereafter.4 SKYRIZI is also approved in the U.S. to treat moderate to severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy, as well as to treat active psoriatic arthritis in adults, and the recommended dosage is 150 mg administered by subcutaneous injection at week 0, week 4, and every 12 weeks thereafter.4 Phase 3 trials of SKYRIZI in psoriasis, Crohn's disease, ulcerative colitis and psoriatic arthritis are ongoing.11,12
SKYRIZI U.S. Uses and Important Safety Information4
SKYRIZI is a prescription medicine used to treat adults with:
- moderate to severe plaque psoriasis who may benefit from taking injections or pills (systemic therapy) or treatment using ultraviolet or UV light (phototherapy).
- active psoriatic arthritis (PsA).
- moderately to severely active Crohn's disease.
For more information on AbbVie in gastroenterology, visit https://www.abbvie.com/our-science/therapeutic-focus-areas/immunology/immunology-focus-areas/gastroenterology.html.
For more information about AbbVie, please visit us at www.abbvie.com. Follow @abbvie on Twitter, Facebook, LinkedIn or Instagram
SOURCE AbbVie
AbbVie wins FDA approval for Skyrizi in Crohn's disease
Jun. 17, 2022 8:22 AM ET
By: Dulan Lokuwithana, SA News Editor4 Comments
- AbbVie (NYSE:ABBV) announced on Friday that the U.S. Food and Drug Administration (FDA) approved its interleukin-23 (IL-23) inhibitor Skyrizi as a treatment for adults with moderately to severely active Crohn's disease.
- https://seekingalpha.com/news/3849551-abbvie-wins-fda-approval-for-skyrizi-in-crohns-disease
- https://seekingalpha.com/symbol/ABBV
- https://www.skyrizi.com/
- https://www.abbvie.com/
AMVUTTRA™ (vutrisiran)
Alnylam Announces FDA Approval of AMVUTTRA™ (vutrisiran), an RNAi Therapeutic for the Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in Adults
Jun 13, 2022
– First and Only FDA-approved Treatment Demonstrating Reversal in Neuropathy Impairment with Subcutaneous Administration Once Every Three Months –
– AMVUTTRA Met Primary and All Secondary Endpoints, with Significant Improvement in Polyneuropathy, Quality of Life and Gait Speed Relative to External Placebo –
– Company Expects to Launch in Early July, with Value-Based Agreements to Accelerate Access –
– Alnylam to Host Conference Call Tomorrow, Tuesday, June 14, 2022 at 8:00 a.m. ET –
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jun. 13, 2022-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) approved AMVUTTRA™ (vutrisiran), an RNAi therapeutic administered via subcutaneous injection once every three months (quarterly) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. hATTR amyloidosis is a rare, inherited, rapidly progressive, and fatal disease with debilitating polyneuropathy manifestations, for which there are few treatment options. The FDA approval is based on positive 9-month results from the HELIOS-A Phase 3 study, where AMVUTTRA significantly improved the signs and symptoms of polyneuropathy, with more than 50 percent of patients experiencing halting or reversal of their disease manifestations.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20220603005487/en/
AMVUTTRA™ (vutrisiran) product logo (Photo: Business Wire)
“Twenty years ago, Alnylam was founded with the bold vision for RNA interference to make a meaningful impact on the lives of people around the world in need of new approaches to address serious diseases with significant unmet medical needs, such as hATTR amyloidosis. Today, AMVUTTRA has the potential to change the standard of care for people living with the polyneuropathy of this devastating disease,” said Yvonne Greenstreet, MBChB, Chief Executive Officer of Alnylam Pharmaceuticals. “We are so thankful to the patients, families and investigators involved in making AMVUTTRA a reality for the hATTR amyloidosis community. As the fifth RNAi therapeutic developed by Alnylam to receive regulatory approval in less than four years, we believe AMVUTTRA represents an important milestone that brings us one step closer to achieving our P5x25 goals aimed at Alnylam’s transition to a leading biotech company.”
The FDA approval of AMVUTTRA is based on positive 9-month results from HELIOS-A, a global, randomized, open-label, multicenter, Phase 3 study that evaluated the efficacy and safety of AMVUTTRA across a diverse group of patients with hATTR amyloidosis with polyneuropathy. 164 patients with hATTR amyloidosis were randomized 3:1 to receive either 25 mg of vutrisiran (N=122) via subcutaneous injection once every three months or 0.3 mg/kg of patisiran (N=42) via intravenous infusion once every three weeks (reference group) for 18 months. The efficacy of AMVUTTRA was assessed by comparing the AMVUTTRA group in HELIOS-A with the placebo group (n=77) from the landmark APOLLO Phase 3 study of patisiran, a randomized controlled study in a comparable patient population.
AMVUTTRA met the primary endpoint of the study, the change from baseline in the modified Neuropathy Impairment Score + 7 (mNIS+7) at 9 months. Treatment with AMVUTTRA (N=114) resulted in a 2.2 point mean decrease (improvement) in mNIS+7 from baseline as compared to a 14.8 point mean increase (worsening) reported for the external placebo group (N=67), resulting in a 17.0 point mean difference relative to placebo (p<0.0001); by 9 months, 50 percent of patients treated with AMVUTTRA experienced improvement in neuropathy impairment relative to baseline.
AMVUTTRA also met all secondary endpoints in the study at 9 months, with significant improvement in the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) score and timed 10-meter walk test (10-MWT), and improvements were observed in exploratory endpoints, including change from baseline in modified body mass index (mBMI), all relative to external placebo. Efficacy results at 18 months were consistent with 9-month data, with AMVUTTRA achieving statistically significant improvements compared to external placebo for all secondary endpoints including mNIS+7, Norfolk QoL-DN, 10-MWT and mBMI, and non-inferiority in serum TTR reduction relative to the within-study patisiran reference group.
AMVUTTRA demonstrated an encouraging safety and tolerability profile with 9 months of dosing and there were no drug-related discontinuations or deaths. The most commonly reported adverse events (AEs) in AMVUTTRA-treated patients included arthralgia (11 percent), dyspnea (7 percent) and vitamin A decreased (7 percent). Injection site reactions (ISRs) were reported in 5 patients (4 percent) and were all mild and transient.
“The FDA approval of AMVUTTRA is very encouraging for the hATTR amyloidosis community, who need additional therapies to address the polyneuropathy of this progressive, life-threatening, multisystem disease,” said Michael Polydefkis, M.D., MHS, Professor, Johns Hopkins Neurology and HELIOS-A Study Investigator. “AMVUTTRA is a new therapeutic option that has demonstrated the potential to halt or reverse polyneuropathy progression in patients with an acceptable safety profile, along with an infrequent, subcutaneous dosing regimen that may also help to improve the disease management experience for patients.”
“Today we celebrate the FDA’s approval of vutrisiran, a welcomed treatment option for hATTR amyloidosis patients experiencing the challenges of the polyneuropathy of the disease,” said Isabelle Lousada, Founder and CEO, Amyloidosis Research Consortium. “With this approval, Alnylam has expanded treatment options that may support improvements in quality of life, providing hope for patients and families in the amyloidosis community.”
Alnylam has a strong and proven track record to ensure those who may benefit from RNAi therapeutics will have access to them, as outlined in the Company’s latest Patient Access Philosophy report. Consistent with our Patient Access Philosophy, AMVUTTRA is priced in line with the value delivered. Our existing innovative value-based agreement (VBA) framework is anticipated to help accelerate access to this important therapy for patients. AMVUTTRA is expected to be available for shipment to healthcare providers in the U.S. in early July.
Alnylam offers a patient support services program, Alnylam Assist™, for people in the U.S. prescribed AMVUTTRA and their families to receive help accessing this new therapy. Alnylam Assist includes Case Managers, a team dedicated to helping assist patients with verification of insurance benefits and financial assistance for those who qualify. Patient Education Liaisons are also available to answer patients’ questions about their disease and treatment. Physicians and patients can learn more about Alnylam’s patient support services program by visiting AlnylamAssist.com or calling 1-833-256-2748.
Vutrisiran is under review by the European Medicines Agency (EMA), the Brazilian Health Regulatory Agency (ANVISA) and the Japanese Pharmaceuticals and Medical Devices Agency (PMDA). Vutrisiran was previously granted Orphan Drug Designation in the U.S. and the European Union (EU) for the treatment of ATTR amyloidosis and in Japan for transthyretin type familial amyloidosis with polyneuropathy. A biannual 50mg dosing regimen is under evaluation within the ongoing randomized treatment extension (RTE) period in the HELIOS-A trial. Vutrisiran is also being evaluated in the HELIOS-B Phase 3 study for the treatment of patients with ATTR amyloidosis with cardiomyopathy, including both hATTR and wild-type ATTR (wtATTR) amyloidosis.
Visit AMVUTTRA.com for more information, including full Prescribing Information.
For additional information about AMVUTTRA, please see the full Prescribing Information.
About Alnylam Pharmaceuticals
Alnylam (Nasdaq: ALNY) has led the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines. Since its founding 20 years ago, Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam’s commercial RNAi therapeutic products are ONPATTRO® (patisiran), GIVLAARI® (givosiran), OXLUMO® (lumasiran), AMVUTTRA™ (vutrisiran) and Leqvio® (inclisiran) being developed and commercialized by Alnylam’s partner, Novartis. Alnylam has a deep pipeline of investigational medicines, including five product candidates that are in late-stage development. Alnylam is executing on its “Alnylam P5x25” strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA. For more information about our people, science and pipeline, www.alnylam.com and engage with us on Twitter at @Alnylam, on LinkedIn, or on Instagram.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220603005487/en/
Alnylam Pharmaceuticals, Inc.
Source: Alnylam Pharmaceuticals, Inc.
Alnylam RNAi therapy for rare protein disorder gets FDA approval
Jun. 14, 2022 5:56 AM ET
Alnylam Pharmaceuticals, Inc. (ALNY)
By: Ravikash, SA News Editor
- The U.S. Food and Drug Administration (FDA) approved Alnylam Pharmaceuticals' (NASDAQ:ALNY) RNAi therapy Amvuttra (vutrisiran) to treat polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults.
- https://seekingalpha.com/news/3848299-alnylam-rnai-therapy-for-rare-protein-disorder-gets-fda-approval
- https://seekingalpha.com/symbol/ALNY
- www.alnylam.com
- AMVUTTRA.com
UPLIZNA® (inebilizumab)
Horizon Therapeutics plc Submits Regulatory Filing for UPLIZNA® (inebilizumab) in Brazil
06/15/22PDF Version
DUBLIN--(BUSINESS WIRE)--Jun. 15, 2022-- Horizon Therapeutics plc (Nasdaq: HZNP) today announced that it has submitted a regulatory filing to the Brazil National Health Surveillance Agency (ANVISA) for UPLIZNA for the treatment of adult patients with anti-aquaporin-4 immunoglobulin G seropositive (AQP4-IgG+) neuromyelitis optica spectrum disorder (NMOSD).
“This regulatory submission is an important milestone as we continue to expand our commitment to NMOSD patients around the world,” said Vikram Karnani, executive vice president and president, international operations, Horizon. “NMOSD is a devastating disease with unpredictable attacks, which can result in potential loss of vision and motor function. We are hopeful that we can bring a potential new treatment option to the estimated ten thousand people living with NMOSD in Brazil.”
In the N-MOmentum Phase 3 clinical trial, the largest NMOSD trial to date, UPLIZNA demonstrated a significant reduction in the risk of an NMOSD attack with only two infusions per year, following the initial two loading doses. Additionally, 89% of patients in the AQP4-IgG+ group remained attack-free during the six-month period post-treatment and 83% of patients on treatment remained attack-free for at least four years.1,2
UPLIZNA was approved by the U.S. Food and Drug Administration (FDA) in June 2020, by the Japanese Ministry of Health, Labor and Welfare in March 2021 and by the European Commission (EC) in April 2022. Mitsubishi Tanabe Pharma Corporation has the rights to develop and commercialize UPLIZNA in Japan, Thailand, South Korea, Indonesia, Vietnam, Malaysia, the Philippines, Singapore and Taiwan. Hansoh Pharmaceutical Group Company Limited, another strategic partner to Horizon, has also recently received manufacturing and marketing approval from the National Medical Products Administration of the People’s Republic of China for UPLIZNA.
About UPLIZNA (inebilizumab-cdon)
INDICATION
UPLIZNA is indicated for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive.
About Horizon
Horizon is a global biotechnology company focused on the discovery, development and commercialization of medicines that address critical needs for people impacted by rare, autoimmune and severe inflammatory diseases. Our pipeline is purposeful: We apply scientific expertise and courage to bring clinically meaningful therapies to patients. We believe science and compassion must work together to transform lives. For more information on how we go to incredible lengths to impact lives, visit www.horizontherapeutics.com and follow us on Twitter, LinkedIn, Instagram and Facebook.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220615005234/en/
Source: Horizon Therapeutics plc
Horizon Therapeutics submits regulatory filing for approval of Uplizna in Brazil
Jun. 15, 2022 10:01 AM ET
Horizon Therapeutics Public Limited Company (HZNP)
By: Anuron Mitra, SA News Editor
- Horizon Therapeutics (NASDAQ:HZNP) on Wednesday said it had submitted a filing to Brazilian health regulator Anvisa for approval of its Uplizna medicine for the treatment of anti-aquaporin-4 immunoglobulin G seropositive neuromyelitis optica spectrum disorder (NMOSD).
- https://seekingalpha.com/news/3848856-horizon-therapeutics-submits-regulatory-filing-for-approval-of-uplizna-in-brazil
- https://seekingalpha.com/symbol/HZNP
- https://www.horizontherapeutics.com/
- https://www.uplizna.com/
For Monjuvi® (Tafasitamab-Cxix) In Combination With TTI-622
Pfizer, MorphoSys And Incyte Enter Into Clinical Trial Collaboration For Monjuvi® (Tafasitamab-Cxix) In Combination With TTI-622, A Fusion Protein Directed Against CD47
June 13, 2022 at 4:01 PM EDTPDF Version
NEW YORK & BOSTON & WILMINGTON, Del.--(BUSINESS WIRE)--Pfizer Inc. (NASDAQ:PFE), MorphoSys U.S. Inc., a fully owned subsidiary of MorphoSys AG (FSE: MOR; NASDAQ:MOR), and Incyte (NASDAQ:INCY) today announced a clinical trial collaboration and supply agreement to investigate the immunotherapeutic combination of Pfizer’s TTI-622, a novel SIRPα-Fc fusion protein, and Monjuvi® (tafasitamab-cxix) plus lenalidomide in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) who are not eligible for autologous stem cell transplantation (ASCT).
“TTI-622 blocks the signal-regulatory protein (SIRP)α–CD47 axis, which is a key checkpoint expected to become an important backbone immunotherapy across multiple tumors, especially hematological cancers,” said Chris Boshoff, M.D., Ph.D., Chief Development Officer, Oncology, Pfizer Global Product Development. “The early results for TTI-622 in late-line advanced lymphoid malignancies reflect the potential for class-leading monotherapy activity, and preclinical evidence with a diverse set of therapeutic agents provide a strong rationale for testing combination therapies. We are pleased to collaborate with MorphoSys and Incyte, generating additional evidence on the potential of TTI-622 to improve outcomes for patients with DLBCL.”
‟Monjuvi in combination with lenalidomide is an important treatment option for patients with relapsed or refractory diffuse large B-cell lymphoma, and its mechanism of action, efficacy and safety profile make it an attractive combination partner,” said Malte Peters, M.D., MorphoSys Chief Research and Development Officer. “We believe that the addition of novel immunotherapies, such as the investigational anti-CD47 blocking agent TTI-622, to the backbone of Monjuvi plus lenalidomide have the potential to provide new meaningful combination treatment options for patients with relapsed or refractory diffuse large B-cell lymphoma.”
‟This collaboration has the potential to advance patient care in an area where there continues to be significant unmet medical need,” said Lance Leopold, M.D., Group Vice President, Clinical Development Hematology and Oncology at Incyte. “We are proud to support this research effort to evaluate the potential of a new chemotherapy-free combination for these patients.”
Pfizer’s TTI-622 is currently in Phase 1b/2 development across several indications, with a focus on hematological malignancies. CD47 is an innate immune checkpoint that binds SIRPα and delivers a "don’t eat me" signal to suppress macrophage phagocytosis. Overexpression of CD47 in solid and hematological malignancies, including in DLBCL, is associated with poor prognosis.
Monjuvi (marketed ex-U.S. as Minjuvi®), a CD19-directed immunotherapy, in combination with lenalidomide is a treatment for adult patients with relapsed or refractory DLBCL not otherwise specified, and who are not eligible for ASCT. In this indication, accelerated or conditional approvals were granted by the U.S. Food and Drug Administration, the European Medicines Agency and other regulatory authorities. Monjuvi is being co-commercialized by MorphoSys and Incyte in the United States. Incyte has exclusive commercialization rights outside the United States.
Preclinical data by Morphosys have shown a strong synergy of Monjuvi and anti-CD47 antibodies in in vitro and in vivo lymphoma models, providing scientific rationale for investigating this combination in clinical trials. This preclinical data was presented at the 62nd American Society of Hematology (ASH) Annual Meeting & Exposition in 2020.
Under the terms of the agreement, Pfizer will initiate a multicenter, international Phase 1b/2 study of TTI-622 with Monjuvi and lenalidomide for patients with relapsed or refractory DLBCL who are not eligible for ASCT. MorphoSys and Incyte will provide Monjuvi for the study, which will be sponsored and funded by Pfizer and is planned to be conducted in North America, Europe and Asia-Pacific.
The collaboration is effective immediately upon the execution of the agreement.
About Tafasitamab
Tafasitamab is a humanized Fc-modified CD19 targeting immunotherapy. In 2010, MorphoSys licensed exclusive worldwide rights to develop and commercialize tafasitamab from Xencor, Inc. Tafasitamab incorporates an XmAb® engineered Fc domain, which mediates B-cell lysis through apoptosis and immune effector mechanism including Antibody-Dependent Cell-Mediated Cytotoxicity (ADCC) and Antibody-Dependent Cellular Phagocytosis (ADCP).
In the United States, Monjuvi® (tafasitamab-cxix) is approved by the U.S. Food and Drug Administration in combination with lenalidomide for the treatment of adult patients with relapsed or refractory DLBCL not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT). This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
In Europe, Minjuvi® (tafasitamab) received conditional marketing authorization in combination with lenalidomide, followed by Minjuvi monotherapy, for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) who are not eligible for autologous stem cell transplant (ASCT).
Tafasitamab is being clinically investigated as a therapeutic option in B-cell malignancies in several ongoing combination trials.
Monjuvi® and Minjuvi® are registered trademarks of MorphoSys AG. Tafasitamab is co-marketed by Incyte and MorphoSys under the brand name MONJUVI® in the U.S., and marketed by Incyte under the brand name Minjuvi® in Europe, the UK and Canada.
XmAb® is a registered trademark of Xencor, Inc.
MONJUVI (tafasitamab-cxix)
About MorphoSys
At MorphoSys, we are driven by our mission: More life for people with cancer. As a global commercial-stage biopharmaceutical company, we use groundbreaking science and technologies to discover, develop, and deliver innovative cancer medicines to patients. MorphoSys is headquartered in Planegg, Germany, and has its U.S. operations anchored in Boston, Massachusetts. To learn more, visit us at www.morphosys.com and follow us on Twitter and LinkedIn .
About Incyte
Incyte is a Wilmington, Delaware-based, global biopharmaceutical company focused on finding solutions for serious unmet medical needs through the discovery, development and commercialization of proprietary therapeutics. For additional information on Incyte, please visit Incyte.com and follow @Incyte .
About Pfizer Oncology
At Pfizer Oncology, we are committed to advancing medicines wherever we believe we can make a meaningful difference in the lives of people living with cancer. Today, we have an industry-leading portfolio of 24 approved innovative cancer medicines and biosimilars across more than 30 indications, including breast, genitourinary, colorectal, blood and lung cancers, as well as melanoma.
Pfizer, MorphoSys, Incyte enter trial collaboration for Monjuvi/TTI-622 combination for lymphoma
Jun. 13, 2022 4:25 PM ET
Pfizer Inc. (PFE), MPSYF, INCY
By: Anuron Mitra, SA News Editor
- Pfizer (NYSE:PFE), a U.S. unit of MorphoSys (OTCPK:MPSYF) and Incyte (NASDAQ:INCY) on Monday entered into a clinical trial collaboration to investigate a combination of PFE's TTI-622 protein with MorphoSys and Incyte's Monjuvi to treat diffuse large B-cell lymphoma (DLBCL) in patients who cannot get a stem cell transplant.
- https://seekingalpha.com/news/3848192-pfizer-morphosys-incyte-enter-trial-collaboration-for-monjuvitti-622-combination-for-lymphoma
- https://seekingalpha.com/symbol/PFE
- https://seekingalpha.com/symbol/INCY
- https://seekingalpha.com/symbol/MPSYF
- https://www.monjuvi.com/
biotecMAX™ Feb/Mar/APR/May/June 2022 Insight
TECENTRIQ® (atezolizumab) injection
European Commission approves Roche’s Tecentriq as adjuvant treatment for a subset of people with early-stage non-small cell lung cancer June 9, 2022
- In the Phase III IMpower010 trial, adjuvant Tecentriq reduced the risk of disease recurrence or death by 57% in people with PD-L1 high resected Stage II-III NSCLC, compared with best supportive care
- Tecentriq is now the first and only cancer immunotherapy available for certain people with early-stage NSCLC in Europe
- Today’s approval marks Tecentriq’s sixth lung cancer indication in Europe
Basel, 09 June 2022 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the European Commission has approved Tecentriq® (atezolizumab) as an adjuvant treatment, following complete resection and platinum-based chemotherapy, for adults with non-small cell lung cancer (NSCLC) with a high risk of recurrence* whose tumours express PD-L1≥50% and who do not have EGFR mutant or ALK-positive NSCLC.
“Today’s approval represents an important advance, as Tecentriq becomes the first cancer immunotherapy approved in Europe for the treatment of certain types of early-stage NSCLC,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. “Since approximately half of all people with early NSCLC develop recurrence after surgery, which in some cases is no longer curable, treating this cancer at an earlier stage offers the best chance to prevent recurrence.”
This approval is based on results from an interim analysis of the Phase III IMpower010 study. The results showed treatment with Tecentriq, following complete resection and platinum-based chemotherapy, reduced the risk of disease recurrence or death (DFS) by 57% (hazard ratio [HR]=0.43, 95% CI: 0.26-0.71)** in people with resected Stage II-IIIA NSCLC (UICC/AJCC 7th edition) whose tumours express PD-L1≥50%, who do not have EGFR mutant or ALK-positive NSCLC, compared with best supportive care (BSC).1 A DFS benefit was consistently seen across most subgroups including histology or stage of disease with adjuvant Tecentriq, compared with BSC. Overall survival (OS) data for patients with PD-L1 high resected Stage II-III NSCLC, and who do not have EGFR mutant or ALK-positive disease are immature and were not formally tested at the DFS interim analysis, however, a trend towards OS improvement with Tecentriq was seen, with a stratified HR of 0.39 (95% CI: 0.18-0.82).2
Follow-up will continue with planned analyses of more mature OS data later this year. Safety data for Tecentriq were consistent with its known safety profile and no new safety signals were identified.1
“Today’s approval now offers patients in Europe, whose tumours express high levels of PD-L1, the opportunity to reduce their risk of disease recurrence following surgery and chemotherapy,” said Professor Enriqueta Felip, Head of the Thoracic Cancer Unit at Vall d’Hebron Institute of Oncology, Barcelona, Spain. “This milestone reinforces the need for biomarker testing at diagnosis for all people with NSCLC, irrespective of disease stage, to ensure they receive optimal treatment.”
To date, Tecentriq has been approved in 19 countries, including the US and China, as adjuvant treatment, following complete resection and chemotherapy, for adults with Stage II-IIIA NSCLC (UICC/AJCC 7th edition) whose tumours express PD-L1≥1%. In three countries, including Canada and the UK, Tecentriq has been approved as adjuvant, treatment following complete resection and chemotherapy, for adult patients with Stage II-IIIA NSCLC (UICC/AJCC 7th edition) whose tumours have PD-L1 expression on ≥50% of tumour cells.
Tecentriq has shown clinically meaningful benefit in various types of lung cancer, with six currently approved indications in countries around the world. It was the first approved cancer immunotherapy for the first-line treatment of adults with extensive-stage small cell lung cancer (SCLC) in combination with carboplatin and etoposide (chemotherapy). Tecentriq also has four approved indications in advanced or metastatic NSCLC as either a single agent or in combination with targeted therapies and/or chemotherapies. Tecentriq is available in three dosing options, providing the flexibility to choose administration every two, three or four weeks.
Roche has an extensive development programme for Tecentriq including multiple ongoing and planned Phase III studies across lung, genitourinary, skin, breast, gastrointestinal, gynaecological, and head and neck cancers. This includes studies evaluating Tecentriq both alone and in combination with other medicines, as well as studies in metastatic, adjuvant and neoadjuvant settings across various tumour types.
About the IMpower010 study
IMpower010 is a Phase III, global, multicentre, open-label, randomised study evaluating the efficacy and safety of Tecentriq compared with BSC, in participants with Stage IB-IIIA NSCLC (UICC/AJCC 7th edition), following surgical resection and up to 4 cycles of adjuvant cisplatin-based chemotherapy. The study randomised 1,005 people with a ratio of 1:1 to receive either Tecentriq (up to 16 cycles) or BSC. The primary endpoint is investigator-determined DFS in the PD-L1-positive Stage II-IIIA, all randomised Stage II-IIIA and intention-to-treat (ITT) Stage IB-IIIA populations. Key secondary endpoints include overall survival in the overall study population, ITT Stage IB-IIIA NSCLC.
About Tecentriq
Tecentriq is a cancer immunotherapy approved for some of the most aggressive and difficult-to-treat forms of cancer. Tecentriq was the first cancer immunotherapy approved for the treatment of a certain type of early-stage non-small cell lung cancer (NSCLC), small cell lung cancer (SCLC) and hepatocellular carcinoma (HCC). Tecentriq is also approved in countries around the world, either alone or in combination with targeted therapies and/or chemotherapies, for various forms of metastatic NSCLC, certain types of metastatic urothelial cancer, PD-L1-positive metastatic triple-negative breast cancer and BRAF V600 mutation-positive advanced melanoma.
Tecentriq is a monoclonal antibody designed to bind with a protein called programmed death ligand-1 (PD-L1), which is expressed on tumour cells and tumour-infiltrating immune cells, blocking its interactions with both PD-1 and B7.1 receptors. By inhibiting PD-L1, Tecentriq may enable the activation of T-cells. Tecentriq is a cancer immunotherapy that has the potential to be used as a foundational combination partner with other immunotherapies, targeted medicines and various chemotherapies across a broad range of cancers. In addition to intravenous infusion, the formulation of Tecentriq is also being investigated as a subcutaneous injection to help address the growing burden of cancer treatment for patients and healthcare systems.
https://www.tecentriq-hcp.com/
To learn more about Roche’s scientific-led approach to cancer immunotherapy, please follow this link: http://www.roche.com/research_and_development/what_we_are_working_on/oncology/cancer-immunotherapy.htm
For more information, please visit www.roche.com.
Roche Tecentriq gets approval in EU for early-stage lung cancer subtype
Jun. 09, 2022 5:28 AM ET Roche Holding AG (RHHBY), RHHBF
By: Ravikash, SA News Editor
- The European Commission (EC) approved Roche's (OTCQX:RHHBY) (OTCQX:RHHBF) Tecentriq as an adjuvant treatment, following complete resection and platinum-based chemotherapy, for adults with non-small cell lung cancer (NSCLC) who are at are at high risk of disease recurrence and whose tumors express PD-L1≥50% and who do not have EGFR mutant or ALK-positive NSCLC.
- https://seekingalpha.com/news/3847064-roche-tecentriq-gets-approval-in-eu-for-early-stage-lung-cancer-subtype
- https://seekingalpha.com/symbol/RHHBY
- https://www.tecentriq-hcp.com/
Tislelizumab
China NMPA Approves Tislelizumab for Recurrent or Metastatic Nasopharyngeal Cancer
Jun 10, 2022 7:00 AM
Tislelizumab is now approved in nine indications in China
CAMBRIDGE, Mass. & BASEL, Switzerland & BEIJING--(BUSINESS WIRE)-- BeiGene (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global, science-driven biotechnology company focused on developing innovative and affordable medicines to improve treatment outcomes and access for patients worldwide, today announced that the China National Medical Products Administration (NMPA) has approved BeiGene’s anti-PD-1 antibody, tislelizumab, in combination with chemotherapy as a first-line treatment for patients with recurrent or metastatic nasopharyngeal cancer (NPC).
“NPC is one of the most common head and neck cancers in China and many parts of Asia. Treatment options have been limited, with chemotherapy primarily provided for front-line care. On behalf of these patients, today’s approval of tislelizumab, a potentially differentiated checkpoint inhibitor, for patients with recurrent or metastatic NPC could provide new hope,” commented Mark Lanasa, M.D., Ph.D., Chief Medical Officer, Solid Tumors at BeiGene. “We look forward to bringing this important immunotherapy to the underserved patient community in China.”
“With nine approved indications in China, our 3,100+ science-based commercial team is working to make tislelizumab more broadly available to those who may benefit from this important immunotherapy,” commented Xiaobin Wu, Ph.D., President, Chief Operating Officer, and General Manager of China, at BeiGene. “Today’s approval is a great step for patients in China with NPC.”
“In the pivotal Phase 3 RATIONALE-309 trial, comparing two arms of patients receiving either tislelizumab in combination with standard chemotherapy, or a placebo with standard chemotherapy, we observed statistical and clinically meaningful improvement in progression-free survival in the tislelizumab arm as assessed by both independent review committee and clinical investigators, and a positive trend in overall survival. These results were consistent with the updated survival data with a follow up time of 15 months, and tislelizumab was generally well tolerated,” said Li Zhang, M.D., professor at the Collaborative Innovation Center for Cancer Medicine, State Key Laboratory of Oncology in South China and Sun Yat-sen University Cancer, and the principal investigator of the trial. “The NMPA’s approval of tislelizumab in NPC is welcoming news to these many patients with the disease.”
This approval was supported by clinical results from the randomized, double-blind, Phase 3 clinical trial RATIONALE 309 (NCT03924986) to evaluate the efficacy and safety of tislelizumab combined with gemcitabine and cisplatin versus placebo combined with gemcitabine and cisplatin as a first-line treatment for patients with recurrent or metastatic NPC.
As announced in May 2021, RATIONALE 309 met the primary endpoint of PFS at the planned interim analysis. Updated efficacy analyses at a median follow-up of 15.5 months, tislelizumab in combination with chemotherapy continued to demonstrate a clinically significant progression-free survival (PFS) benefit over chemotherapy and placebo for patients with RM-NPC. Meanwhile, the tislelizumab arm continued to demonstrate a positive trend in overall survival (OS) and improvement in time to disease progression or death after next-line therapy (PFS2). The safety profile of the tislelizumab and chemotherapy combination was generally manageable and consistent with safety profiles of each treatment agent. These data were presented at an ASCO Virtual Plenary session in April and at the ASCO Annual Meeting in June 2022.
About Nasopharyngeal Cancer (NPC)
Nasopharyngeal cancer (NPC) is a malignant, squamous cell carcinoma which arises from the epithelial cells of the nasopharynx, most commonly originating in the pharyngeal recess (the fossa of Rosenmüller).i There were an estimated 62,555 new cases of NPC in China in 2020, accounting for 46.8 percent of the worldwide incidence.ii Despite the heavy public health burden of NPC in southern China and other endemic areas, relatively little is known about the etiology and prevention of NPC.iii The major risk factors for NPC are genetic predisposition, Epstein-Barr virus (EBV) infection, and consumption of salt-preserved food.iv The median overall survival rate is about 20 months in advanced NPC;v with progressively worsening prognoses falling to a three-year survival of 7-40% reported in patients with recurrent or metastatic NPC, indicating a high medical unmet need with more effective treatment urgently needed.vi,vii,viii
About RATIONALE-309
RATIONALE-309 is a multicenter, randomized, double-blind, placebo-controlled Phase 3 clinical trial (NCT03924986) designed to evaluate the efficacy and safety of tislelizumab combined with gemcitabine and cisplatin (Arm A) versus placebo combined with gemcitabine and cisplatin (Arm B) as a first-line treatment for patients with RM-NPC.
The primary endpoint of the trial is progression-free survival (PFS) in the intent-to-treat (ITT) population as assessed by an independent review committee (IRC) per RECIST v1.1 criteria; secondary endpoints include IRC-assessed overall response rate (ORR), IRC-assessed duration of response (DoR), overall survival (OS), investigator-assessed PFS, time to second objective disease progression (PFS2), and safety.
A total of 263 patients were enrolled in the trial, with 131 and 132 randomized to Arm A and Arm B, respectively, with balanced baseline characteristics between both arms. Interim results from the trial were presented in December at the European Society for Medical Oncology Immuno-Oncology (ESMO I-O) Congress. Those data showed that at a media follow-up time of 10 months, tislelizumab demonstrated a statistically significant improvement in terms of extending progression-free survival (PFS) as well as clinically meaningful benefit on other survival endpoints compared with chemotherapy and placebo and a generally manageable safety profile.
About Tislelizumab
Tislelizumab is an anti-programmed death receptor-1 (PD-1) inhibitor designed to help aid the body’s immune cells to detect and fight tumors. Tislelizumab, a humanized monoclonal antibody, is specifically designed to minimize binding to FcγR on macrophages. In pre-clinical studies, binding to FcγR on macrophages has been shown to compromise the anti-tumor activity of PD-1 antibodies through activation of antibody-dependent macrophage-mediated killing of T effector cells.
Tislelizumab is the first drug from BeiGene’s immuno-oncology biologics program and is being developed internationally as a monotherapy and in combination with other therapies for the treatment of a broad array of both solid tumor and hematologic cancers. BeiGene has initiated or completed more than 20 potentially registration-enabling clinical trials in 35 countries and regions, including 17 Phase 3 trials and four pivotal Phase 2 trials.
Tislelizumab is approved by the China National Medical Products Administration (NMPA) as a treatment for nine indications, including multiple approvals in non-small cell lung cancer (NSCLC). Tislelizumab has been submitted for regulatory review as a potential treatment for unresectable recurrent locally advanced or metastatic esophageal squamous cell carcinoma (ESCC) after prior systemic therapy in the U.S., and in NSCLC and second-line ESCC in Europe. In January 2021, BeiGene partnered with Novartis to accelerate the clinical development and marketing of tislelizumab in North America, Europe and Japan.
BeiGene Oncology
BeiGene is committed to advancing best- and first-in-class clinical candidates internally or with like-minded partners to develop impactful and affordable medicines for patients across the globe. We have a growing R&D and medical affairs team of approximately 2,900 colleagues dedicated to advancing more than 100 clinical trials that have involved more than 16,000 subjects. Our expansive portfolio is directed predominantly by our internal colleagues supporting clinical trials in more than 45 countries and regions. Hematology-oncology and solid tumor targeted therapies and immuno-oncology are key focus areas for the Company, with both mono- and combination therapies prioritized in our research and development. BeiGene currently has three approved medicines discovered and developed in our own labs: BTK inhibitor BRUKINSA in the United States, China, the EU and U.K., Canada, Australia and additional international markets; and the non-FC-gamma receptor binding anti-PD-1 antibody tislelizumab as well as the PARP inhibitor pamiparib in China.
BeiGene also partners with innovative companies who share our goal of developing therapies to address global health needs. We commercialize a range of oncology medicines in China licensed from Amgen, Bristol Myers Squibb, EUSA Pharma and Bio-Thera. We also plan to address greater areas of unmet need globally through our other collaborations including with Mirati Therapeutics, Seagen, and Zymeworks.
In January 2021 BeiGene and Novartis announced a collaboration granting Novartis rights to co-develop, manufacture, and commercialize BeiGene’s anti-PD1 antibody tislelizumab in North America, Europe, and Japan. Building upon this productive collaboration, including a biologics license application (BLA) under FDA review, BeiGene and Novartis announced an option, collaboration and license agreement in December 2021 for BeiGene’s TIGIT inhibitor, ociperlimab, that is in Phase 3 development. Novartis and BeiGene also entered into a strategic commercial agreement through which BeiGene is promoting five approved Novartis Oncology products across designated regions of China.
About BeiGene
BeiGene is a global, science-driven biotechnology company focused on developing innovative and affordable medicines to improve treatment outcomes and access for patients worldwide. With a broad portfolio of more than 40 clinical candidates, we are expediting development of our diverse pipeline of novel therapeutics through our own capabilities and collaborations. We are committed to radically improving access to medicines for two billion more people by 2030. BeiGene has a growing global team of over 8,000 colleagues across five continents. To learn more about BeiGene, please visit www.beigene.com and follow us on Twitter at @BeiGeneGlobal.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220610005109/en/
Source: BeiGene
BeiGene stock rises as tislelizumab gets approval in China for head/neck cancer subtype
Jun. 10, 2022 7:36 AM ETBeiGene, Ltd. (BGNE), NVS
By: Ravikash, SA News Editor1 Comment
- China's National Medical Products Administration (NMPA) approved BeiGene (NASDAQ:BGNE) and Novartis' (NYSE:NVS) tislelizumab in combination with chemotherapy as a first-line treatment for patients with recurrent or metastatic nasopharyngeal cancer (NPC)
- https://seekingalpha.com/news/3847610-beigene-stock-rises-as-tislelizumab-gets-approval-in-china-for-headneck-cancer-subtype
- https://seekingalpha.com/symbol/BGNE
- https://seekingalpha.com/symbol/NVS
- https://www.beigene.com/our-science-and-medicines/tislelizumab/
Navitoclax is an investigational, oral BCL-XL/BCL-2 inhibitor.
June 10, 2022
AbbVie Presents Investigational Navitoclax Preliminary Data in JAK Inhibitor Naïve Myelofibrosis Patients
- Data is supportive of early intervention in myelofibrosis to achieve improved clinical outcomes in spleen volume reduction (SVR), symptom score, bone marrow fibrosis (BMF), and anemia
- Results are from an exploratory analysis of navitoclax plus ruxolitinib from Cohort 3 of the Phase 2 REFINE study in JAK inhibitor naïve myelofibrosis patients
NORTH CHICAGO, Ill., June 10, 2022 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced new data from Cohort 3 of its Phase 2 REFINE study of investigational navitoclax in combination with ruxolitinib in JAK inhibitor naïve patients with myelofibrosis (MF), a rare and difficult to treat blood cancer. These preliminary findings show spleen volume and symptomatic improvement in this cohort. These data are consistent with previously observed data from relapsed/refractory patients in Cohort 1a1 and will be shared in an oral presentation at the 2022 European Hematology Association (EHA) Annual Congress (Abstract #S197).2
REFINE is a Phase 2 non-randomized open-label multi-cohort study evaluating the safety and efficacy of navitoclax alone or in combination with ruxolitinib in MF.
"Current treatment options for myelofibrosis are limited and targeted toward controlling disease symptoms," said Mohamed Zaki, M.D., Ph.D., vice president and global head of oncology clinical development, AbbVie. "Together with pre-clinical findings, early results of this study demonstrating anti-fibrosis activity of navitoclax in combination with ruxolitinib are promising. Specifically, the data findings regarding reduction in spleen volume, symptoms and bone marrow fibrosis help support the further exploration of disease modification in myelofibrosis."
The results presented at EHA 2022 were from a preliminary analysis of 32 JAK inhibitor naïve MF patients from Cohort 3 of the Phase 2 REFINE trial (NCT03222609).2 The primary endpoint was spleen volume reduction of ≥35 percent (SVR35) from baseline at week 24.2 Key secondary endpoints include ≥50 percent reduction in total symptom score (TSS50) at week 24, anemia response and BMF reduction.2
In the results, SVR35 was achieved by 63 percent of evaluable patients at week 24 (20/32) and by 78 percent at any time on treatment (25/32).2 At week 24, 41 percent (11/27) of evaluable patients with measurable baseline symptoms reached TSS50; notably, 67 percent of patients (18/27) met this endpoint at any time during the study.2 In this cohort, 35 percent of evaluable patients, with available fibrosis grade at baseline and during the study, (9/26) achieved reduction in BMF by ≥1 grade at any time during the study with three patients experiencing ≥2 grade reductions in BMF.2 Additionally, 40 percent of patients evaluable for anemia response (6/15) experienced improvement in anemia, a common clinical feature of MF.2
Preliminary safety analysis identified no new safety signals. Thirty-one (97 percent) patients reported one or more adverse event (AE).1 The most common Grade ≥3 AEs were thrombocytopenia (47 percent), anemia (34 percent), and neutropenia (25 percent).1 Seven patients (22 percent) reported experiencing serious AEs.1 Three patients (9 percent) experienced an AE leading to navitoclax discontinuation and three patients (9 percent) reported an AE leading to ruxolitinib discontinuation.2
"These data reinforce the importance of early intervention in myelofibrosis and the potential to achieve improved clinical outcomes," said Francesco Passamonti, Full Professor of Hematology, University of Insubria and Chief, Hematology, Varee Hospital. "These preliminary results show good responses to combination therapy with navitoclax that may continue to improve over time."
About Navitoclax
Navitoclax is an investigational, oral BCL-XL/BCL-2 inhibitor. The BCL-2 family of proteins are known regulators of the apoptosis pathway.3
Navitoclax is not approved by the U.S. Food and Drug Administration (FDA) or any Health Authority worldwide at this time. Its safety and efficacy are under evaluation as part of ongoing Phase 2 and registrational Phase 3 studies.
AbbVie has an extensive late-stage clinical trial program for investigational navitoclax that is currently enrolling. For more information about enrolling in a clinical trial, please visit us here.
About the REFINE Study
REFINE is a multi-cohort, Phase 2, randomized, open-label, multicenter study evaluating the tolerability and efficacy of navitoclax alone or when added to ruxolitinib in patients with myelofibrosis (MF).4 The primary outcome measure is the percentage of patients who achieve Spleen Volume Reduction of greater than or equal to 35 percent (SVR35) from baseline to Week 24. Secondary outcomes measures include percentage of participants achieving 50 percent reduction in Total Symptom Score from baseline to Week 24 and change in grade of bone marrow fibrosis assessed according to the European Consensus Grading System.
Data presented at EHA 2022 include preliminary safety and efficacy results from Cohort 3 of REFINE (n=32). Patients in Cohort 3 had primary or secondary MF with splenomegaly and had not received JAK-2 therapy or BET inhibitors prior to enrollment. Data presented at EHA 2022 are representative of data from Cohort 3 of the REFINE study as of February 7, 2022.
Data included in the official EHA 2022 Abstract Book are representative of data from Cohort 3 of the REFINE study as of October 4, 2021.
More information can be found on https://www.clinicaltrials.gov/ (NCT03222609).
About Myelofibrosis
Myelofibrosis (MF) is a rare, difficult-to-treat blood cancer that results in excessive scar tissue formation (fibrosis) in the bone marrow. Patients living with MF experience symptoms such as an enlarged spleen, fatigue, weakness, and severe anemia, that are often debilitating and greatly impact quality of life. MF also carries a risk of transformation to more aggressive disease such as acute myeloid leukemia.4
About AbbVie in Oncology
At AbbVie, we are committed to transforming standards of care for multiple blood cancers while advancing a dynamic pipeline of investigational therapies across a range of cancer types. Our dedicated and experienced team joins forces with innovative partners to accelerate the delivery of potentially breakthrough medicines. We are evaluating more than 20 investigational medicines in over 300 clinical trials across some of the world's most widespread and debilitating cancers. As we work to have a remarkable impact on people's lives, we are committed to exploring solutions to help patients obtain access to our cancer medicines. For more information, please visit http://www.abbvie.com/oncology.
About AbbVie
AbbVie's mission is to discover and deliver innovative medicines that solve serious health issues today and address the medical challenges of tomorrow. We strive to have a remarkable impact on people's lives across several key therapeutic areas: immunology, oncology, neuroscience, eye care, virology, women's health and gastroenterology, in addition to products and services across its Allergan Aesthetics portfolio. For more information about AbbVie, please visit us at www.abbvie.com. Follow @abbvie on Twitter, Facebook, Instagram, YouTube and LinkedIn.
AbbVie navitoclax, Jakafi combo shows promise in trial to treat rare bone marrow cancer
Jun. 10, 2022 7:13 AM ETAbbVie Inc. (ABBV)INCY
By: Ravikash, SA News Editor
AbbVie (NYSE:ABBV) said new data from a mid-stage trial suggest that eraly intervention in patients with myelofibrosis (MF) with navitoclax in combination with Incyte's (INCY) Jakafi led to improved outcomes.
https://seekingalpha.com/symbol/INCY
https://seekingalpha.com/symbol/ABBV
OMICRON-CONTAINING BIVALENT BOOSTER CANDIDATE MRNA-1273.214
MODERNA ANNOUNCES OMICRON-CONTAINING BIVALENT BOOSTER CANDIDATE MRNA-1273.214 DEMONSTRATES SUPERIOR ANTIBODY RESPONSE AGAINST OMICRON
JUNE, 08, 2022 DOWNLOAD(OPENS IN NEW WINDOW)
Data Show Significantly Higher Geometric Mean Titer Ratios, Meeting Prespecified Endpoints for Superiority Against Omicron Variant
mRNA-1273.214 Exhibited an 8-Fold Boost in Neutralizing Geometric Mean Titers Against Omicron Among Baseline Seronegative Participants
Safety and Tolerability Profile for mRNA-1273.214 is Consistent with Prior Booster Dose of mRNA-1273
Regulatory Submission Planned for Coming Weeks to Enable Use of mRNA-1273.214 For Fall Booster
Conference Call to be Held Today At 8:00 AM ET
CAMBRIDGE, MA / ACCESSWIRE / June 8, 2022 / Moderna, Inc., (NASDAQ:MRNA) a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, today announced new clinical data on its Omicron-containing bivalent COVID booster candidate, mRNA-1273.214, containing mRNA-1273 (Spikevax) and a vaccine candidate targeting the Omicron variant of concern. A 50 µg booster dose of mRNA-1273.214 met all pre-specified endpoints including superior neutralizing antibody response (geometric mean ratio) against the Omicron variant one month after administration when compared to the original mRNA-1273 vaccine.The booster dose of mRNA-1273.214 was generally well-tolerated, with side effects comparable to a booster dose of mRNA-1273 at the 50 µg dose level.
"We are thrilled to share the preliminary data analysis on mRNA-1273.214, which is the second demonstration of superiority of our bivalent booster platform against variants of concern and represents an innovation in the fight against COVID," said Stéphane Bancel, Chief Executive Officer of Moderna. "Looking at these data alongside the durability we saw with our first bivalent booster candidate, mRNA-1273.211, we anticipate more durable protection against variants of concern with mRNA-1273.214, making it our lead candidate for a Fall 2022 booster. We are submitting our preliminary data and analysis to regulators with the hope that the Omicron-containing bivalent booster will be available in the late summer. Taken together, our bivalent booster candidates demonstrate the power of Moderna's mRNA platform to develop vaccines that meet immediate, global public health threats."
mRNA-1273.214 met all primary endpoints in the Phase 2/3 trial including neutralizing antibody response against Omicron when compared to a 50 µg booster dose of mRNA-1273 in baseline seronegative participants. Pre-specified criteria for superiority as measured by neutralizing geometric mean titer ratio (GMR) with the lower bound of the confidence interval >1 was met. The GMR and corresponding 97.5% confidence interval was 1.75 (1.49, 2.04). A booster dose of mRNA-1273.214 increased neutralizing geometric mean titers (GMT) against Omicron approximately 8-fold above baseline levels. Primary endpoints of non-inferiority against ancestral SARS-CoV-2 were also met, with GMR against ancestral SAR-COV-2 (D614G) of 1.22 (1.08-1.37).
Among seronegative participants one month after administration, the neutralizing GMT against ancestral SARS-CoV-2 for mRNA-1273.214 was 5977 (CI: 5322, 6713) , compared to GMT for mRNA-1273 of 5649 (CI: 5057, 6311). The GMT against Omicron for mRNA-1273.214 was 2372 (CI: 2071, 2718), compared to GMT for mRNA-1273 of 1473 (CI: 1271, 1708).
Binding antibody titers (MSD) were also significantly higher (nominal alpha of 0.05) against all other variants of concern (Alpha, Beta, Gamma, Delta, Omicron) for mRNA-1273.214 when compared to mRNA-1273.
The mRNA-1273.214 50 μg booster dose was well-tolerated in the 437 study participants. The safety and reactogenicity profile of the mRNA-1273.214 50 μg booster dose was similar to that of mRNA-1273 50 μg dose when these vaccines were administered as a second booster dose.
In February 2021, Moderna announced its strategy to update booster candidates to address the ongoing evolution of the SARS-CoV-2 virus, including monovalent and bivalent candidates targeting multiple variants of concern. The Company's primary focus has been on the bivalent booster approach, which are boosters that address two viral strains simultaneously.
Results from the Company's Beta-containing bivalent booster candidate, mRNA-1273.211, announced in April 2022, demonstrated superiority against Beta, Delta and Omicron variants of concern one month after administration, with continued superiority that was durable against Beta and Omicron variants of concern six months after administration. Given the significantly higher antibody titers induced by mRNA-1273.214 compared to mRNA-1273, Moderna anticipates that antibody titers induced by mRNA-1273.214 will be more durable over time against Omicron as compared to mRNA-1273. Moderna will report data from Day 91 after vaccination later in the summer.
Moderna is planning to submit the interim analysis and data to regulators for review in the coming weeks.
To learn more, visit www.modernatx.com.
SOURCE: Moderna, Inc.
View source version on accesswire.com:
https://www.accesswire.com/704276/Moderna-Announces-Omicron-Containing-Bivalent-Booster-Candidate-mRNA-1273214-Demonstrates-Superior-Antibody-Response-Against-Omicron
Moderna says booster targeting Omicron outperformed current vaccine
Jun. 08, 2022 7:28 AM ET Moderna, Inc. (MRNA)
By: Dulan Lokuwithana, SA News Editor4 Comments
- Moderna (NASDAQ:MRNA) announced on Wednesday that its Omicron-containing COVID booster candidate demonstrated a superior neutralizing antibody response against the new variant compared to its original vaccine one month after administration.
- https://seekingalpha.com/news/3846618-moderna-says-booster-shot-targeting-omicron-outperformed-current-shot
- https://seekingalpha.com/symbol/MRNA
- https://investors.modernatx.com/overview/default.aspx
Priorix
06 June 2022
GSK announces US FDA approval of Priorix for the prevention of measles, mumps and rubella in individuals 12 months of age and older
Issued: London UK
- Priorix becomes an additional source of measles, mumps and rubella vaccine for US patients
GSK plc (LSE/NYSE: GSK) today announced that the US Food and Drug Administration (FDA) has approved Priorix (Measles, Mumps and Rubella Vaccine, Live) for active immunisation for the prevention of measles, mumps and rubella (MMR) in individuals 12 months of age and older.
Priorix is currently licenced in more than 100 countries worldwide, including all European countries, Canada, Australia and New Zealand, with more than 800 million doses distributed to date.
“We’re proud to make Priorix available in the US for the first time, adding a choice for providers to help protect patients against these highly-contagious diseases and to further strengthen offerings in our paediatric vaccine portfolio,” said Judy Stewart, Senior Vice President and Head of US Vaccines, GSK.
Measles, mumps and rubella are acute and highly-contagious viral diseases responsible for considerable morbidity and mortality throughout the world.[1][2] In recent years, measles outbreaks have occurred in the US and globally, with more than 400,000 cases confirmed in 2019, reversing decades of progress toward measles elimination in many countries.[3]
According to a recent US Centers for Disease Control and Prevention (CDC) report, vaccine ordering in the past two years through the CDC’s Vaccines For Children programme, the federal programme through which about half of the children in the country are immunised, dropped more than 10%, indicating that fewer vaccinations in children were occurring.[4] The report also noted 400,000 fewer children entered kindergarten in the 2020-2021 school year than expected nationally, meaning those children may not be up to date on childhood immunisations like their MMR vaccination.[4]
“Outbreaks of measles in recent years demonstrate how quickly diseases can return without widespread immunisation. Missed vaccinations during the pandemic makes children even more vulnerable to vaccine-preventable diseases like measles,” said Temi Folaranmi, MD, Vice President and Vaccines Therapeutic Area Head, US Medical Affairs, GSK. “Making Priorix available to patients in the US will ensure health care professionals have more than one option for this critical vaccine as they work to catch their patients up on recommended vaccinations.”
The safety of Priorix was evaluated in six clinical studies, in which a total of 12,151 participants (6,391 in the US) received at least one dose of Priorix: 8,780 children (4,148 in the US) 12 through 15 months of age; 2,917 children (1,950 in the US) 4 through 6 years of age; and 454 adults and children (293 in the US) 7 years of age and older. The most commonly reported adverse reactions were pain, redness, swelling, loss of appetite, irritability, drowsiness and fever. The efficacy of Priorix was demonstrated based on immunogenicity data versus the comparator vaccine.
Priorix will provide US healthcare professionals with another MMR vaccine choice. Priorix may be administered as a first dose, followed by a second dose of Priorix. Priorix may also be administered as a second dose to individuals who have previously received the first dose of another MMR-containing vaccine.
The CDC recommends people get a MMR vaccine to protect against measles, mumps and rubella. Children should get two doses of MMR vaccine, starting with the first dose at 12 through 15 months of age, and the second dose at 4 through 6 years of age. Teens and adults should also be up to date on their MMR vaccination.[5]
Priorix is scheduled to be on the agenda for the June CDC Advisory Committee on Immunization Practices (ACIP) meeting for consideration of formal inclusion into the vaccine schedule and recommendations.
The US Prescribing Information is available at: https://gskpro.com/content/dam/global/hcpportal/en_US/Prescribing_Information/Priorix/pdf/PRIORIX.PDF
For further information please visit
GSK's measles vaccine Priorix wins FDA approval
Jun. 06, 2022 4:33 AM ET
By: Ravikash, SA News Editor
- The U.S. Food and Drug Administration (FDA) approved GSK's (NYSE:GSK) vaccine Priorix for preventing measles, mumps and rubella (MMR) in individuals one year old and above.
- https://seekingalpha.com/news/3845794-gsks-measles-vaccine-priorix-wins-fda-approval
- https://seekingalpha.com/symbol/GSK
- https://gskpro.com/content/dam/global/hcpportal/en_MU/PI/Priorix-GDS15.pdf
Evusheld, formerly known as AZD7442 ( tixagevimab & cilgavimab)
Evusheld significantly prevented COVID-19 disease progression or death in TACKLE Phase III treatment trial
PUBLISHED8 June 2022
8 June 2022 07:00 BST
Results published in The Lancet Respiratory Medicine support benefits of Evusheld in the outpatient treatment of mild-to-moderate COVID-19
Detailed results from the TACKLE Phase III outpatient treatment trial showed AstraZeneca’s Evusheld (tixagevimab and cilgavimab, formerly AZD7442) provided clinically and statistically significant protection against progression to severe COVID-19 or death from any cause compared to placebo, with treatment with Evusheld earlier in the disease course leading to more favourable outcomes.1
The data have been published in The Lancet Respiratory Medicine.
TACKLE was conducted in non-hospitalised adults with mild-to-moderate COVID-19 who were symptomatic for seven days or less. In the trial, 90% of participants were at high risk of progression to severe COVID-19 due to co-morbidities or age.1
Hugh Montgomery, Professor of Intensive Care Medicine at University College London, UK and TACKLE principal investigator, said: “Despite the success of vaccines, many individuals such as older adults, individuals with co-morbidities and those who are immunocompromised, remain at risk for poor outcomes from severe COVID-19. Additional options are needed to prevent disease progression and reduce the burden on healthcare systems, especially with the continued emergence of new variants. The TACKLE results show that one intramuscular dose of Evusheld can prevent these individuals from progressing to severe COVID-19, with earlier treatment leading to even better results.”
Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, AstraZeneca, said: “These results published in The Lancet Respiratory Medicine add to the growing evidence supporting the use of Evusheld to help patients who most need additional protection against COVID-19. We are discussing the TACKLE data with regulatory authorities and continue to progress submissions in both treatment and prophylaxis indications to help combat COVID-19 on all fronts.”
In TACKLE, a single 600mg intramuscular (IM) dose of Evusheld significantly reduced the relative risk of progressing to severe COVID-19 or death (from any cause) by 50% (95% confidence interval [CI] 15, 71; p=0.010) through day 29 compared to placebo in non-hospitalised patients with mild-to-moderate COVID-19 who were symptomatic for seven days or less, the trial’s primary endpoint.
In pre-specified analyses of participants who received treatment within three days of symptom onset, Evusheld reduced the risk of developing severe COVID-19 or death (from any cause) by 88% compared to placebo (95% CI 9, 98), and the risk reduction was 67% (95% CI 31, 84) when participants received Evusheld within five days of symptom onset.1
Evusheld also reduced the risk of respiratory failure, a secondary endpoint, by 72% (95% CI 0.3, 92; nominal p=0·036), with three Evusheld participants (0.7%) versus 11 placebo participants (3%) requiring measures such as mechanical ventilation or extracorporeal membrane oxygenation1.
Evusheld was generally well-tolerated in the trial. Adverse events (AEs) occurred more frequently in the placebo group than the Evusheld group, 36% and 29%, respectively. The most common AE was COVID-19 pneumonia, occurring in 49 participants (11%) in the placebo group and 26 participants (6%) in the Evusheld group. Serious AEs occurred in 12% of participants in the placebo group and 7% in the Evusheld group.1 There were six COVID-19-reported deaths in the placebo group and three in the Evusheld group.1
TACKLE
TACKLE is a Phase III, randomised, double-blind, placebo-controlled, multi-centre trial assessing the safety and efficacy of a single 600mg IM dose of Evusheld compared to placebo for the outpatient treatment of mild-to-moderate COVID-19. The trial was conducted in 95 sites in the US, Latin America, Europe and Japan. 903 participants were randomised (1:1) to receive either Evusheld (n = 452) or saline placebo (n = 451), administered in two separate, sequential IM injections.
Participants were adults 18 years-old and over who had mild-to-moderate COVID-19, were symptomatic for seven days or less and were not hospitalised. Participants had a documented laboratory-confirmed SARS-CoV-2 infection, as determined by a molecular test (antigen or nucleic acid) from any respiratory tract specimen (e.g. oropharyngeal, nasopharyngeal, or nasal swab or saliva) collected no more than three days prior to day 1. Participants were not vaccinated against COVID-19 at the time of screening.
The primary efficacy endpoint was the composite of either severe COVID-19 or death from any cause through day 29. Subjects will continue to be followed for 15 months.
Approximately 13% of participants were 65 years and over. In addition, 90% had baseline co-morbidities and other characteristics that put them at high risk of progression to severe COVID-19, including cancer, diabetes, obesity, chronic lung disease or asthma, cardiovascular disease or immunosuppression. Approximately 62% were White/Caucasian, 4% Black/African American, 6% Asian and 24% American Indian or Alaskan Native. Approximately 52% of participants were Hispanic/Latino.
AstraZeneca previously announced positive high-level results from the TACKLE Phase III trial in the treatment of mild-to-moderate COVID-19.
Evusheld
Evusheld, formerly known as AZD7442, is a combination of two long-acting antibodies - tixagevimab (AZD8895) and cilgavimab (AZD1061) - derived from B-cells donated by individuals previously infected with the SARS-CoV-2 virus. Discovered by Vanderbilt University Medical Center and licensed to AstraZeneca in June 2020, the human monoclonal antibodies bind to distinct sites on the SARS-CoV-2 spike protein2 and were optimised by AstraZeneca with half-life extension and reduced Fc receptor and complement C1q binding.3 The half-life extension more than triples the durability of its action compared to conventional antibodies;4-6 data from the Phase III PROVENT trial show protection lasting at least six months.7 The reduced Fc receptor binding aims to minimise the risk of antibody-dependent enhancement of disease - a phenomenon in which virus-specific antibodies promote, rather than inhibit, infection and/or disease.8
There is a growing body of evidence from multiple independent in vitro and in vivo (animal model) studies supporting the potential of Evusheld to protect against Omicron SARS-CoV-2 subvariants and all tested variants of concern to date.9-13 In particular, data from Washington University School of Medicine demonstrated Evusheld retained neutralising activity against the highly transmissible BA.2 subvariant, which is currently the dominant strain globally.9,14 This study also showed that Evusheld reduced viral burden and limited inflammation in the lungs (in vivo) for Omicron BA.1, BA.1.1 and BA.2.9 A new preclinical study from the University of Oxford showed that Evusheld also retains neutralisation activity against the emerging Omicron BA.4 and BA.5 variants.13
Evusheld has marketing authorisation in the European Union and was granted conditional marketing authorisation by the Medicines and Healthcare products Regulatory Agency (MHRA) in Great Britain for pre-exposure prophylaxis of COVID-19. Evusheld is authorised for emergency use for pre-exposure prophylaxis of COVID-19 in the US. Evusheld is also authorised for use and being supplied in several other countries around the world. Regulatory filings are progressing in both prevention and treatment around the world.
Evusheld is being developed with support from the US government, including federal funds from the Department of Health and Human Services; Office of the Assistant Secretary for Preparedness and Response; Biomedical Advanced Research and Development Authority in partnership with the Department of Defense; Joint Program Executive Office for Chemical, Biological, Radiological and Nuclear Defense, under Contract No. W911QY-21-9-0001.
Under the terms of the licensing agreement with Vanderbilt, AstraZeneca will pay single-digit royalties on future net sales.
Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca.
AstraZeneca Evusheld 'significantly' cuts risk of severe COVID, death in phase 3 trial
Jun. 08, 2022 5:19 AM ET
By: Ravikash, SA News Editor
AstraZeneca (NASDAQ:AZN) said detailed results from a late stage outpatient treatment trial showed Evusheld provided statistically significant protection against progression to severe COVID-19 or death from any cause, compared to placebo, and outcomes were more favorable if the treatment was started early.
https://seekingalpha.com/symbol/AZN
https://www.evusheld.com/en/patient
BALVERSA® (erdafitinib)
Janssen Presents Initial Results from the Phase 2 RAGNAR Study of BALVERSA (erdafitinib) in Patients with Advanced Solid Tumors with FGFR Alterationss
Jun 07, 2022United States
Data from RAGNAR, the largest tumor-agnostic study reported for a targeted therapy and the first to evaluate FGFR-driven malignancies, featured in oral presentation at the
2022 ASCO Annual Meeting
June 7, 2022 (CHICAGO) – The Janssen Pharmaceutical Companies of Johnson & Johnson today announced initial results from the pivotal Phase 2 RAGNAR study evaluating the investigational use of BALVERSA® (erdafitinib), a fibroblast growth factor receptor (FGFR) kinase inhibitor, in patients with advanced solid tumors with prespecified FGFR alterations. At a planned interim analysis (IA), responses were observed across a variety of FGFR-driven solid tumors for patients who had exhausted standard treatment options prior to being treated with BALVERSA®.[1] These results will be featured in an oral presentation (Abstract #3007) today at the 2022 American Society of Clinical Oncology Annual Meeting.
RAGNAR (NCT04083976) is a Phase 2 clinical study designed to evaluate the efficacy and safety of BALVERSA® in patients with advanced or metastatic solid tumors and prespecified FGFR gene alterations, regardless of tumor location or histology (tumor-agnostic). The IA was based on 178 patients with 32 distinct solid tumor histologies.1 Patients in the study were prospectively identified by local molecular testing or central next-generation sequencing (NGS); the most common tumor types were cholangiocarcinoma (bile duct cancer) (n=31), high-grade glioma (tumor of the brain or spinal cord) (n=29), breast cancer (n=14), pancreatic cancer (n=13) and squamous non-small cell lung cancer (n=11).1 The study also included tumors that occur less frequently in the real world such as salivary gland and parathyroid carcinomas (rare endocrine malignancies), as well as tumors of unknown primary origin.1 Study participants were heavily pretreated, with 74.7 percent (n=133) having received two or more prior lines of therapy.1
The primary endpoint of the RAGNAR study is the overall response rate (ORR) as assessed by an independent review committee (IRC). At the IA data cutoff, IRC assessed an ORR of 29.2 percent (95 percent confidence interval [CI], 22.7-36.5) and a disease control rate (DCR) of 72.5 percent (95 percent CI, 65.3-78.9) for the overall tumor-agnostic patient population.1 Investigators observed responses in 14 distinct tumor types. This included responses in hard-to-treat malignancies such as salivary gland cancer (100 percent ORR; treated n=5, responders n=5), pancreatic cancer (31 percent ORR; treated n=13, responders n=4) and glioblastoma (21 percent ORR; treated n=29, responders=6).1 Investigators also observed an overall 7.1-month median duration of response (DOR) (95 percent CI, 5.5-9.3). At the data cutoff, 51.1 percent (n=24) of patients who had responded to treatment continued to show a response.1 The primary analysis for all patients treated in this RAGNAR cohort, known as the broad panel cohort, is anticipated later this year.
The safety profile of BALVERSA® observed in RAGNAR was consistent with the known safety profile of BALVERSA® in metastatic urothelial carcinoma (mUC). Across tumor types, 44.9 percent of patients experienced adverse events of grade three or above.1 Adverse events were manageable with supportive care and treatment interruptions or reductions, when necessary.1 The discontinuation rate due to drug-related adverse events was 7.3 percent.1
“Diagnostic advances in the identification of FGFR gene alterations have opened the door to targeted, tumor-agnostic treatment approaches for patients,” said Yohann Loriot, M.D., Ph.D., Institut Gustave Roussy and University of Paris-Saclay, and principal study investigator.‡ “Results from the RAGNAR study show that, through the targeted inhibition of FGFR receptors, we may be able to tailor treatment for patients with advanced FGFR-driven cancers, regardless of tumor location or histology.”
In 2019, BALVERSA® was granted accelerated approval by the U.S. Food and Drug Administration (FDA) as a targeted therapy for adult patients with locally advanced or mUC with susceptible FGFR2 or FGFR3 alterations and who have progressed during or following at least one line of prior platinum-containing chemotherapy, including within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy.[2]
“Janssen is committed to advancing precision medicine approaches for the treatment of patients with biomarker-driven cancers, an area of clear unmet need,” said Kiran Patel, M.D., Vice President, Clinical Development, Solid Tumors, Janssen Research & Development, LLC. “RAGNAR, Janssen’s first tumor-agnostic study, demonstrates our commitment to understand the biology of disease, identify new treatment pathways and improve patient outcomes. We look forward to progressing the development of BALVERSA for these patients and sharing additional updates on this program in the future.”
About the RAGNAR Study
RAGNAR (NCT04083976) is a Phase 2 clinical trial evaluating the safety and efficacy of BALVERSA® in patients with advanced solid tumors, regardless of cancer type or tumor location (tumor-agnostic), driven by FGFR1–4 alterations. Patients in the trial have progressed on or after at least one line of systemic therapy and have no alternative standard treatment options. Following screening by local molecular testing or central NGS, patients are enrolled in four separate cohorts: a broad panel cohort of patients with pathogenic FGFR mutations or gene fusions (tumor histologies evaluated include but are not limited to cholangiocarcinoma [bile duct cancer], high- and low-grade glioma [a tumor type occurring in the brain or spinal cord], breast, pancreatic, squamous and non-squamous non-small cell lung cancer, colorectal, endometrial, esophageal, salivary gland, ovarian, duodenal [cancer occurring in the first part of the small intestine], thyroid and cancer of unknown primary origin); an exploratory cohort of patients with other FGFR mutations; a cholangiocarcinoma expansion cohort; and a pediatric cohort of patients ages 6 to 17 with FGFR alterations.1
The primary endpoint of RAGNAR is IRC-assessed ORR. Key secondary endpoints include investigator-assessed ORR, DOR, DCR, clinical benefit rate, progression free survival, overall survival and incidence and severity of adverse events.
About BALVERSA®
BALVERSA® (erdafitinib) is a once-daily, oral FGFR kinase inhibitor that is approved by the U.S. FDA for the treatment of adults with locally advanced or mUC that has susceptible FGFR3 or FGFR2 genetic alterations and has progressed during or following at least one line of platinum-containing chemotherapy, including within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy. Patients are selected for therapy based on an FDA-approved companion diagnostic for BALVERSA®. Information on FDA-approved tests for the detection of FGFR genetic alterations in urothelial cancer is available at: http://www.fda.gov/CompanionDiagnostics. This indication is approved under accelerated approval based on tumor response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.2,[6]
In addition to RAGNAR, BALVERSA® is being studied in clinical trials including the Phase 3 THOR (NCT03390504) study evaluating BALVERSA® versus standard of care, consisting of chemotherapy (docetaxel or vinflunine) or anti-PD-1 agent pembrolizumab, in participants with advanced urothelial cancer and selected FGFR aberrations with disease progression following one or two prior lines of therapy; and the randomized Phase 2 THOR-2 (NCT04172675) study examining BALVERSA® versus investigator choice of intravesical chemotherapy in participants who received Bacillus Calmette-Guérin and recurred with high risk non-muscle-invasive bladder cancer.[7],[8]
In 2008, Janssen Pharmaceutica NV entered into an exclusive worldwide license and collaboration agreement with Astex Pharmaceuticals to develop and commercialize BALVERSA®.
For more information, visit www.BALVERSA.com.
Please see the full Prescribing Information for BALVERSA®.
About the Janssen Pharmaceutical Companies of Johnson & Johnson
At Janssen, we’re creating a future where disease is a thing of the past. We’re the Pharmaceutical Companies of Johnson & Johnson, working tirelessly to make that future a reality for patients everywhere by fighting sickness with science, improving access with ingenuity, and healing hopelessness with heart. We focus on areas of medicine where we can make the biggest difference: Cardiovascular, Metabolism, & Retina; Immunology; Infectious Diseases & Vaccines; Neuroscience; Oncology; and Pulmonary Hypertension.
Learn more at www.janssen.com. Follow us at @JanssenGlobal and @JanssenUS. Janssen Research & Development, LLC, Janssen Biotech, Inc. and Janssen Pharmaceutica NV are part of the Janssen Pharmaceutical Companies of Johnson & Johnson.
Johnson & Johnson posts interim data for cancer drug to indicate 29% overall response
Jun. 07, 2022 12:24 PM ET
By: Dulan Lokuwithana, SA News Editor1 Comment
Announcing mid-stage data for its oral FGFR kinase inhibitor, Balversa, the Janssen Pharmaceutical Companies of Johnson & Johnson (NYSE:JNJ), said on Tuesday that 29.2% of patients with FGFR-driven solid tumors responded to the treatment.
https://seekingalpha.com/symbol/JNJ
Viaskin™ Peanut 250 µg
Montrouge, France, June 7, 2022 DBV Technologies Announces Positive Topline Results from Phase 3 EPITOPE Trial of Viaskin Peanut in PeanutAllergic Toddlers • 67.0% of subjects treated with Viaskin Peanut 250 µg met response criteria at 12 months, compared with 33.5% of subjects in the placebo arm • Pivotal trial met primary endpoint: lower bound of the 95% confidence interval (CI) of the difference between treatment arms was 22.4%, exceeding the prespecified threshold of 15% • Safety results were generally consistent with safety profile of Viaskin Peanut 250 μg observed in children with peanut allergy ages 4 years and older in prior clinical trials • High treatment compliance rate observed over trial duration, with low rate of discontinuation due to adverse events • DBV will hold a conference call today at 5:00 p.m. ET to discuss the results DBV Technologies (Euronext: DBV – ISIN: FR0010417345 – Nasdaq Stock Market: DBVT), a clinical-stage biopharmaceutical company, today announced that its pivotal Phase 3 trial EPITOPE (EPIT in TOddlers with PEanut Allergy), assessing the safety and efficacy of Viaskin™ Peanut 250 µg for the treatment of peanut-allergic toddlers ages 1 to 3 years, met its primary endpoint. Viaskin Peanut demonstrated a statistically significant treatment effect(p<0.001), with 67.0% of subjects in the Viaskin Peanut arm meeting the treatment responder criteria after 12 months, as compared to 33.5% of subjects in the placebo arm (difference in response rates = 33.4%; 95% CI = 22.4% - 44.5%). DBV intends to further analyze the data from EPITOPE and explore regulatory pathways for Viaskin Peanut in children ages 1 to 3 years, given the high unmet need and absence of approved treatments for this vulnerable population. Separately, DBV continues productive dialogue with the U.S. Food and Drug Administration (FDA) on the protocol design of VITESSE, a pivotal Phase 3 trial of the modified Viaskin Peanut patch in peanut-allergic children ages 4 years and older. “Most peanut-allergic children are diagnosed between 1 to 3 years of age; however, there are currently no FDA-approved therapies for this age group. Furthermore, there is growing evidence on the benefits of treatment from a younger age,” said Dr. Hugh Sampson, Kurt Hirschhorn Professor of Pediatrics at the Icahn School of Medicine at Mount Sinai, Director Emeritus of the Jaffe Food Allergy Institute and Chairman of the DBV Scientific Advisory Board. “We believe these positive findings support the potential clinical benefit of Viaskin Peanut in this important, and underserved, population.” These results represent the second and final part of the EPITOPE trial, which enrolled 362 subjects ages 1to 3 years, of which 244 and 118 were in the active and placebo arms, respectively. Enrollment was balanced for age and baseline disease characteristics between the active and placebo treatment arms. The median subject baseline eliciting dose (ED) was 100 mg in each treatment arm. A double-blind, placebo-controlled food challenge (DBPCFC) was administered at baseline and month 12 to determine a subject’s ED at each timepoint. A treatment responder was defined as either a subject with a baseline ED ≤10 mg who reached an ED ≥300 mg of peanut protein at month 12, or a subject with a baseline ED >10 mg who reached an ED ≥1,000 mg of peanut protein at month 12. In an additional pre-specified efficacy analysis, treatment response was defined as achieving an ED ≥1,000 mg of peanut protein regardless of baseline ED. Using this response criterion, Viaskin Peanut demonstrated a statistically significant treatment effect (p<0.001), with 64.2% of subjects in the Viaskin Peanut arm meeting this treatment responder criterion after 12 months as compared to 29.6% of subjects in the placebo arm (difference in response rates = 34.7%; 95% CI = 23.6% - 45.7%). The response rate observed in the placebo arm appears consistent with the approximately 22% natural resolution rate in this patient population. 1 The EPITOPE safety results were generally consistent with the safety profile of Viaskin Peanut 250 μg observed in children with peanut allergy ages 4 years and older in prior clinical trials. No imbalance in the overall adverse event (AE) rate was observed in the trial between the active and placebo arms. Overall, 21 subjects (8.6%) in the Viaskin Peanut armand 3 subjects (2.5%) in the placebo arm experienced a serious adverse event (SAE). Only 1 of the SAEs (0.4%), which was mild periorbital edema (swelling around the eye) in the Viaskin Peanut arm, was deemed related to treatment. The most commonly reported adverse events were skin reactions localized to the administration site, the majority of which were mild to moderate in nature. Fifty-five subjects (22.5%) in the Viaskin Peanut arm experienced an application site reaction that was assessed as severe by an investigator compared with 10 subjects (8.5%) in the placebo arm. Based on investigators’ reported observations from examinations of the skin at each study visit, using the skin grading systems defined in the protocol, the severity of administration site skin reactions following patch application decreased throughout the course of the 12-month treatment period. Four (1.6%) subjects in the Viaskin Peanut arm experienced an anaphylactic reaction determined to be related to, or possibly related to, treatment. Among these anaphylactic reactions, 3 resolved with a single dose of epinephrine and 1 resolved without epinephrine. All anaphylactic reactions were mild to moderate in severity and were characterized mainly by skin and respiratory symptoms. Eight subjects (3.3%) in the Viaskin Peanut arm discontinued due to adverse events. In the 12-month treatment period, the trial completion rate was 84.8% and was balanced between the Viaskin Peanut and placebo arms. Mean subject compliance to daily patch treatment was above 95% in both the active and placebo arms. “We are thrilled by the topline results of EPITOPE, our second Phase 3 clinical trial to evaluate the safety and efficacy of Viaskin Peanut,” said Dr. Pharis Mohideen, Chief Medical Officer of DBV Technologies. “We are grateful to the toddlers and their parents, caregivers and allergists who are contributing to a brighter future by having participated in this first-of-its kind trial.” Following the completion of EPITOPE, all eligible subjects had the option to rollover into EPOPEX, a long-term, open-label extension study of Viaskin Peanut. There are currently 304 subjects (88% of all eligible subjects) enrolled in EPOPEX. DBV plans to present full EPITOPE trial results at future medical congresses as well as submit them for publication in a peer-reviewed journal.
About EPITOPE EPITOPE (NCT03211247) enrolled 413 subjects (51 in Part A and 362 in Part B) in approximately 50 centers across North America (Canada and the United States), Europe and Australia. The EPITOPE trial is a two-part trial: Part A was designed to assess the safety of Viaskin Peanut 100 µg and 250 µg and to determine the highest safe dose, and Part B wasdesigned to assess the efficacy and safety of the selected dose. Based on the results of Part A, the 250 µg dose was selected for Part B. In Part B, subjects were randomized 2:1 to receive Viaskin Peanut 250 µg or placebo. The primary endpoint wasbased on a responder analysis after 12 months of treatment with the selected dose of Viaskin Peanut. As a secondary efficacy endpoint, cumulative reactive dose (CRD) was also evaluated in EPITOPE to establish the total quantity of peanut protein that triggers subject reactions at month 12 of active treatment versus placebo. Serological markers were also measured at baseline, 3, 6 and 12 months in order to characterize the immunological changes in subjects. Following the completion of EPITOPE, all eligible subjects had the option to rollover into EPOPEX, a long-term, open-label extension study of Viaskin Peanut 250 µg. Now that the EPITOPE study results are publicly available, subjects enrolled in the EPOPEX study will be unblinded to their respective treatment group in EPITOPE. About DBV Technologies DBV Technologies is developing Viaskin™, an investigational proprietary technology platform with broad potential applications in immunotherapy. Viaskin is based on epicutaneous immunotherapy, or EPIT™, DBV Technologies’ method of delivering biologically active compounds to the immune system through intact skin. With this new class of non-invasive product candidates, the Company is dedicated to safely transforming the care of food allergic patients. DBV Technologies’ food allergies programs include ongoing clinical trials of Viaskin Peanut. DBV Technologies has global headquarters in Montrouge, France, and North American operations in Basking Ridge, NJ. The Company’s ordinary shares are traded on segment B of Euronext Paris (Ticker: DBV, ISIN code: FR0010417345) and the Company’s ADSs (each representing one-half of one ordinary share) are traded on the Nasdaq Global Select Market (Ticker: DBVT).
DBV Technologies' phase 3 trial for peanut allergy treatment in toddlers meets main goal
Jun. 07, 2022 5:45 PM ET
By: Anuron Mitra, SA News Editor2 Comments
- U.S.-listed shares of DBV Technologies (NASDAQ:DBVT) soared nearly 40% to $2.14 in Tuesday aftermarket trading, after the company said its phase 3 trial assessing its Viaskin Peanut for the treatment of peanut allergies in toddlers aged 1 to 3 years met its main goal.
- https://seekingalpha.com/news/3846509-dbv-technologies-phase-3-trial-for-peanut-allergy-treatment-in-toddlers-meets-main-goal
- https://seekingalpha.com/symbol/DBVT
- https://www.dbv-technologies.com/pipeline/viaskin-peanut/#:~:text=VIPES%20(Viaskin%E2%84%A2%20Peanut's%20Efficacy,to%2055%20years%20of%20age.
Galinpepimut-S Combined with Opdivo
SELLAS Life Sciences Reports Encouraging Updated Clinical Data Indicating Increased Survival from Ongoing Phase 1 Mesothelioma Study of Galinpepimut-S Combined with Opdivo
06/08/22 Download this Press Release PDF Format (opens in new window) (PDF)Median Overall Survival of 45.7 Weeks for Patients Treated With Combination Therapy for at Least One Month; Median Overall Survival in Relapsed/Refractory Patients Treated with Standard of Care is Approximately 28 Weeks
NEW YORK, June 08, 2022 (GLOBE NEWSWIRE) -- SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) ("SELLAS" or the “Company”), a late-stage clinical biopharmaceutical company focused on developing novel therapies for a broad range of cancer indications, today announced encouraging updated clinical data from a Phase 1 investigator-sponsored clinical trial of its lead clinical candidate, galinpepimut-S (GPS), combined with the checkpoint inhibitor nivolumab (Opdivo®) in patients with malignant pleural mesothelioma (MPM) who were either refractory to or relapsed after at least one line of the standard of care therapy.
Data from eight patients enrolled in the study have been analyzed, with final data in the clinical trial expected by the end of 2022. Of the eight patients, seven received at least three doses of GPS, the last of which was given in combination with nivolumab. All enrolled patients have received and progressed with, or were refractory to, frontline pemetrexed-based chemotherapy.
The study details are as follows:
- Of the eight evaluable patients, six were male and two were female, with the median age of 66. 75 percent of the patients entered the study as Stage III or IV patients, with 50 percent of patients entering as Stage IV. Initial tumor stages were II (two patients), III and IIIB (two patients) and IV (four patients).
- All patients had the MPM epithelioid and/or sarcomatoid variant, a tumor which is universally expressing Wilms Tumor 1 (WT1), one of the most widely expressed cancer antigens, ranked by the National Cancer Institute as the top priority among cancer antigens for immunotherapy.
- Median overall survival (OS) calculated as the time from the cessation of the most recent previous therapy until confirmed death or most recent data update for patients who are still alive (50 percent of patients) was 40.9 weeks (9.4 months) for all eight patients and 45.7 weeks (10.5 months) in patients who received the combination therapy (seven out of eight patients). The median progression-free survival (PFS) was 11.1 weeks for all eight patients and 11.9 weeks in patients who received the combination therapy.
- The safety profile of the GPS-nivolumab combination was similar to that seen with nivolumab alone, with the addition of only low-grade, temporary local reactions at the GPS injection site, consistent with previously performed clinical studies with GPS. No Grade 3/4 toxicities were observed for GPS and there were no dose-limiting toxicities.
“This updated data is very encouraging, as it not only confirms our data reported in June 2021, but now reflects an increased survival benefit even though almost all additionally enrolled patients had Grade III and IV malignant mesothelioma,” said Angelos Stergiou, M.D., Sc.D. h.c., President and CEO, SELLAS. “This increase in survival appears to be consistent with long term immunity-mediated antitumor effect with this immunotherapy combination and it reinforces the data we unveiled earlier this year from the Phase 1/2 clinical trial of GPS in combination with another checkpoint inhibitor, pembrolizumab, in relapsed and refractory ovarian cancer patients, in which GPS showed a superior disease control rate compared to that seen with checkpoint inhibitors alone.”
“Of additional importance is the fact that both trials addressed patients with bulky active disease, the setting in which other cancer vaccines have historically had very little effect. We believe that the results of both studies demonstrate the potential effectiveness of GPS as a combination therapy,” concluded Dr. Stergiou.
About SELLAS Life Sciences Group, Inc.
SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) is a late-stage clinical biopharmaceutical company focused on the development of novel therapeutics for a broad range of cancer indications. SELLAS’ lead product candidate, GPS, is licensed from Memorial Sloan Kettering Cancer Center and targets the WT1 protein, which is present in an array of tumor types. GPS has potential as a monotherapy or in combination with other therapies to address a broad spectrum of hematologic malignancies and solid tumor indications. The Company is also developing GFH009, a small molecule, highly selective CDK9 inhibitor, which is licensed from GenFleet Therapeutics (Shanghai), Inc., for all therapeutic and diagnostic uses in the world outside of Greater China.
For more information on SELLAS, please visit www.sellaslifesciences.com.
Opdivo® is a registered trademark of Bristol Myers Squibb, and is not a trademark of SELLAS. The manufacturer of this brand is not affiliated with and does not endorse SELLAS or its products.
Source: SELLAS Life Sciences Group, Inc.
Sellas' GPS, Opdivo combo shows promise in early stage study in type of lung cancer
Jun. 08, 2022 9:48 AM ET
SELLAS Life Sciences Group, Inc. (SLS), BMY
By: Ravikash, SA News Editor
Sellas Life Sciences (NASDAQ:SLS) said updated data from a phase 1 trial of its drug galinpepimut-S (GPS) in combination with Bristol-Myers Squibb's (NYSE:BMY) Opdivo suggested increased survival in patients with malignant pleural mesothelioma (MPM) who either relapsed or were resistant to treatment after at least one line of standard of care chemotherapy.
https://seekingalpha.com/symbol/BMY
https://seekingalpha.com/symbol/SLS
https://www.sellaslifesciences.com/galinpepimut-s-gps-therapy/
DUPIXENT® (DUPILUMAB)
FDA APPROVES DUPIXENT® (DUPILUMAB) AS FIRST BIOLOGIC MEDICINE FOR CHILDREN AGED 6 MONTHS TO 5 YEARS WITH MODERATE-TO-SEVERE ATOPIC DERMATITIS
Children treated with Dupixent and topical corticosteroids (TCS) achieved clearer skin, experienced significantly improved overall disease severity and significantly reduced itch compared to TCS alone at week 16 in a Phase 3 trial
Long-term safety data from a 52-week open-label extension trial in this age group reinforce the well-established safety profile of Dupixent observed across all other approved age groups
Dupixent is the first and only biologic medicine approved to treat moderate-to-severe atopic dermatitis from infancy through adulthood
TARRYTOWN, N.Y. and PARIS, June 7, 2022 /PRNewswire/ -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today announced that the U.S. Food and Drug Administration (FDA) has approved Dupixent® (dupilumab) for children aged 6 months to 5 years with moderate-to-severe atopic dermatitis whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable. Regulatory filings for this age group are underway by the European Medicines Agency and regulatory authorities in additional countries.
"Moderate-to-severe atopic dermatitis in babies and young children is more than just a rash – the intense itch can make them scratch uncontrollably throughout the day and night and cause their skin to crack and bleed," said Julie Block, President and Chief Executive Officer at National Eczema Association. "Caregivers do their best to manage skincare routines multiple times a day, but for many, topical treatments are not enough. We're pleased to see how scientific innovation and research continues to address unmet needs for the atopic dermatitis community, and we're hopeful for the positive impact Dupixent can have for these children and their families."
Atopic dermatitis is a chronic type 2 inflammatory skin disease. Eighty-five to ninety percent of patients first develop symptoms before 5 years of age, which can often continue through adulthood. Symptoms include intense, persistent itch and skin lesions that cover much of the body, resulting in skin dryness, cracking, pain, redness or darkening, and crusting and oozing. In the U.S., more than 75,000 children aged 5 years and younger have uncontrolled moderate-to-severe disease and are most in need of new treatment options. Moderate-to-severe atopic dermatitis may also significantly impact the quality of life of a young child and their caregivers.
"Young children with moderate-to-severe atopic dermatitis are a significantly underserved population of patients, who spend vulnerable years of their lives suffering through the relentless and far-reaching effects of this chronic disease," said George D. Yancopoulos, M.D., Ph.D., President and Chief Scientific Officer at Regeneron, and a principal inventor of Dupixent. "Dupixent has changed the atopic dermatitis treatment paradigm – significantly clearing skin and reducing itch – by targeting an underlying cause of this disease without broadly suppressing the immune system. Today's approval brings the proven efficacy and, importantly, well-established safety profile of Dupixent to these young children, making it the first of its kind to be approved for any U.S. patient aged six months or older living with this debilitating disease."
"Until today, treatment options in the U.S. for infants and children under the age of 6 suffering from moderate-to-severe atopic dermatitis have been limited to topical steroids – which may be associated with significant safety risks when used long-term. This has left patients and their caregivers in desperate need of medicines that can better address the chronic, long-term nature of the disease," Naimish Patel, M.D, Senior Vice President, Head of Global Development, Immunology and Inflammation at Sanofi. "These young people, and their families, often struggle to cope with the significant impact itch can have on them. This approval means that Dupixent, with its well-established safety and efficacy profile, is now available to some of the youngest people living with this disease."
The FDA evaluated Dupixent under Priority Review, which is reserved for medicines that represent potentially significant improvements in efficacy or safety in treating serious conditions. The approval is based on data that include a Phase 3 trial evaluating Dupixent every four weeks (200 mg or 300 mg, based on body weight) plus low-potency topical corticosteroids (TCS) or TCS alone. The trial met the primary and all secondary endpoints. At 16 weeks, patients who received Dupixent with TCS experienced the following, compared to TCS alone (placebo):
- 28% achieved clear or almost-clear skin compared to 4% with placebo, the primary endpoint.
- 53% achieved 75% or greater improvement in overall disease severity from baseline compared to 11% with placebo, the co-primary endpoint outside of the U.S.
- 48% achieved clinically meaningful reduction in itch compared to 9% with placebo.
The safety profile of Dupixent observed through 16 weeks in children aged 6 months to 5 years was similar to the safety profile in patients 6 years and older with atopic dermatitis. The long-term safety profile of Dupixent in children aged 6 months to 5 years through 52 weeks was also similar to the safety profile observed in the pivotal trial and consistent with what was observed in older patients with atopic dermatitis. Hand-foot-and-mouth disease and skin papilloma were, respectively, reported in 5% and 2% of Dupixent patients aged 6 months to 5 years, and none of these cases led to treatment discontinuation.
About the Dupixent Trial
The Phase 3 randomized, double-blind, placebo-controlled trial evaluated the efficacy and safety of Dupixent added to standard-of-care low-potency TCS compared to low-potency TCS alone (placebo) in 162 children aged 6 months to 5 years with uncontrolled moderate-to-severe atopic dermatitis.
The primary endpoints assessed the proportion of patients achieving an Investigator's Global Assessment (IGA) score of 0 (clear) or 1 (almost clear) and 75% improvement in Eczema Area and Severity Index (EASI-75) at week 16. Additional outcome measures included itch reduction, which was assessed using a caregiver-reported 0 to 10 Numerical Rating Scale, with a clinically meaningful improvement defined as ≥4-point improvement at week 16.
Children who completed the trials were eligible to enroll in an open-label extension trial to assess the safety and efficacy of long-term treatment with Dupixent in this age group.
About Dupixent
Dupixent is administered as an injection under the skin (subcutaneous injection) at different injection sites. In patients aged 6 months to 5 years, Dupixent is administered with a pre-filled syringe every four weeks based on weight (200 mg for children ≥5 to <15 kg and 300 mg for children ≥15 to <30 kg). Dupixent is intended for use under the guidance of a healthcare professional and can be given in a clinic or at home by self-administration after training by a healthcare professional. In children younger than 12 years of age, Dupixent should be administered by a caregiver if given at home. Dupixent does not require initial lab testing or ongoing lab monitoring.
Regeneron and Sanofi are committed to helping patients in the U.S. who are prescribed Dupixent gain access to the medicine and receive the support they may need with the DUPIXENT MyWay® program. For more information, please call 1-844-DUPIXENT (1-844-387-4936) or visit www.DUPIXENT.com.
Dupixent is approved for use in certain patients with atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyposis (CRSwNP) or eosinophilic esophagitis in different age populations in a number of countries around the world. Dupixent is currently approved across these indications in the U.S. and for one or more of these indications in the European Union and Japan and more than 60 countries. More than 400,000 patients have been treated globally.
Dupixent, which was invented using Regeneron's proprietary VelocImmune® technology, is a fully human monoclonal antibody that inhibits the signaling of the interleukin-4 (IL-4) and interleukin-13 (IL-13) pathways and is not an immunosuppressant. The Dupixent development program has shown significant clinical benefit and a decrease in type 2 inflammation in Phase 3 trials, establishing that IL-4 and IL-13 are key and central drivers of the type 2 inflammation that plays a major role in multiple related and often co-morbid diseases. These diseases include approved indications for Dupixent such as asthma, atopic derma
Dupilumab Development Program
Dupilumab is being jointly developed by Regeneron and Sanofi under a global collaboration agreement. To date, dupilumab has been studied across more than 60 clinical trials involving more than 10,000 patients with various chronic diseases driven in part by type 2 inflammation.
In addition to the currently approved indications, Regeneron and Sanofi are studying dupilumab in a broad range of diseases driven by type 2 inflammation or other allergic processes in Phase 3 trials, including prurigo nodularis, pediatric eosinophilic esophagitis, hand and foot atopic dermatitis, chronic inducible urticaria-cold, chronic spontaneous urticaria, chronic pruritis of unknown origin, chronic obstructive pulmonary disease with evidence of type 2 inflammation, chronic rhinosinusitis without nasal polyposis, allergic fungal rhinosinusitis, allergic bronchopulmonary aspergillosis and bullous pemphigoid. These potential uses of dupilumab are currently under clinical investigation, and the safety and efficacy in these conditions have not been fully evaluated by any regulatory authority.
U.S. Indications
DUPIXENT is a prescription medicine used:
- to treat adults and children 6 months of age and older with moderate-to-severe atopic dermatitis (eczema) that is not well controlled with prescription therapies used on the skin (topical), or who cannot use topical therapies. DUPIXENT can be used with or without topical corticosteroids. It is not known if DUPIXENT is safe and effective in children with atopic dermatitis under 6 months of age.
- with other asthma medicines for the maintenance treatment of moderate-to-severe eosinophilic or oral steroid dependent asthma in adults and children 6 years of age and older whose asthma is not controlled with their current asthma medicines. DUPIXENT helps prevent severe asthma attacks (exacerbations) and can improve your breathing. DUPIXENT may also help reduce the amount of oral corticosteroids you need while preventing severe asthma attacks and improving your breathing. DUPIXENT is not used to treat sudden breathing problems. It is not known if DUPIXENT is safe and effective in children with asthma under 6 years of age.
- with other medicines for the maintenance treatment of chronic rhinosinusitis with nasal polyposis (CRSwNP) in adults whose disease is not controlled. It is not known if DUPIXENT is safe and effective in children with chronic rhinosinusitis with nasal polyposis under 18 years of age.
- to treat adults and children 12 years of age and older, who weigh at least 88 pounds (40 kg), with eosinophilic esophagitis (EoE). It is not known if DUPIXENT is safe and effective in children with eosinophilic esophagitis under 12 years of age and who weigh at least 88 pounds (40 kg).
Please see accompanying full Prescribing Information including Patient Information.
For more information, please visit www.Regeneron.com or follow @Regeneron on Twitter.
Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY
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SOURCE Regeneron Pharmaceuticals
FDA approves Regeneron, Sanofi's Dupixent in babies, young children for eczema
Jun. 07, 2022 5:04 PM ET
Regeneron Pharmaceuticals, Inc. (REGN), SNY
By: Anuron Mitra, SA News Editor
- Regeneron Pharmaceuticals (NASDAQ:REGN) and Sanofi (NASDAQ:SNY) on Tuesday said the U.S. FDA had approved their medicine Dupixent for children aged 6 months to 5 years with inflammation of the skin.
- https://seekingalpha.com/news/3846498-fda-approves-regeneron-sanofis-dupixent-in-babies-young-children-for-eczema
- https://seekingalpha.com/symbol/SNY
- https://seekingalpha.com/symbol/REGN
- https://www.dupixent.com/
- https://www.regeneron.com/
ORLADEYO® (berotralstat)
BioCryst Announces Approval of ORLADEYO® (berotralstat) by Swissmedic
RESEARCH TRIANGLE PARK, N.C., June 07, 2022 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that Swissmedic has granted marketing authorization for oral, once-daily ORLADEYO® (berotralstat) for the routine prevention of recurrent hereditary angioedema (HAE) attacks in patients 12 years and older in Switzerland.
“ORLADEYO offers people with HAE in Switzerland and their physicians the first orally administered non-steroidal therapy for preventing HAE attacks and provides the community with another vitally important treatment option,” said Henrik Balle Boysen, executive vice president and chief operating officer of HAE International, a global non-profit network of patient associations dedicated to improving the lives of people with HAE.
“We have made significant progress in making ORLADEYO available to many patients in Europe since we received European Commission approval of our oral, once-daily prophylactic therapy last year,” said Waldemar Heiduk, chief executive officer of BioCryst Pharma Deutschland GmbH. “With today’s announcement, we look forward to launching ORLADEYO in Switzerland soon, pending finalization of our reimbursement plans.”
ORLADEYO was safe and well tolerated in clinical trials. The most frequently reported adverse reactions in patients receiving ORLADEYO compared with placebo were gastrointestinal reactions. These reactions generally occurred early after initiation of treatment with ORLADEYO, became less frequent with time and typically self-resolved.
About ORLADEYO® (berotralstat)
ORLADEYO® (berotralstat) is the first and only oral therapy designed specifically to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years and older. One capsule of ORLADEYO per day works to prevent HAE attacks by decreasing the activity of plasma kallikrein.
U.S. Indication and Important Safety Information
INDICATION
ORLADEYO® (berotralstat) is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older.
Please see full Prescribing Information.
About BioCryst Pharmaceuticals
BioCryst Pharmaceuticals discovers novel, oral, small-molecule medicines that treat rare diseases in which significant unmet medical needs exist and an enzyme plays a key role in the biological pathway of the disease. Oral, once-daily ORLADEYO® (berotralstat) is approved in the United States and multiple global markets. BioCryst has several ongoing development programs including BCX9930, an oral Factor D inhibitor for the treatment of complement-mediated diseases, BCX9250, an ALK-2 inhibitor for the treatment of fibrodysplasia ossificans progressiva, and galidesivir, a potential treatment for Marburg virus disease and yellow fever. RAPIVAB® (peramivir injection) is approved in the U.S. and multiple global markets, with post-marketing commitments ongoing. For more information, please visit the company’s website at www.biocryst.com.
BioCryst Orladeyo gets approval in Switzerland to prevent hereditary angioedema attacks
Jun. 07, 2022 9:40 AM ET
BioCryst Pharmaceuticals, Inc. (BCRX)
By: Ravikash, SA News Editor1 Comment
- Swissmedic granted marketing authorization to BioCryst Pharmaceuticals' (NASDAQ:BCRX) once-daily oral drug Orladeyo (berotralstat) for the routine prevention of recurrent hereditary angioedema (HAE) attacks in patients 12 years and older.
- https://seekingalpha.com/news/3846295-biocryst-orladeyo-gets-approval-in-switzerland-to-prevent-hereditary-angioedema-attacks
- https://seekingalpha.com/symbol/BCRX
- https://orladeyo.com/
- https://ir.biocryst.com/
NVX-CoV2373.
FDA Advisory Committee Recommends Emergency Use Authorization of Novavax COVID-19 Vaccine for People Aged 18 Years and OlderJun 7, 2022
- Novavax COVID-19 vaccine receives positive vote from U.S. Food and Drug Administration Vaccines and Related Biological Products Advisory Committee
- If Emergency Use Authorization is granted by the FDA, the Novavax COVID-19 vaccine would become the first protein-based COVID-19 vaccine available in the U.S.
GAITHERSBURG, Md., June 7, 2022 /PRNewswire/ -- Novavax, Inc. (Nasdaq: NVAX), a biotechnology company dedicated to developing and commercializing next-generation vaccines for serious infectious diseases, today announced the U.S. Food and Drug Administration (FDA) Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted 21 to 0, with one abstention, to recommend that the FDA grant Emergency Use Authorization (EUA) for the Novavax COVID-19 vaccine (NVX-CoV2373) for individuals aged 18 years and over.
"The advisory committee's positive recommendation acknowledges the strength of our data and the importance of a protein-based COVID-19 vaccine developed using an innovative approach to traditional vaccine technology," said Stanley C. Erck, President and Chief Executive Officer, Novavax. "In today's VRBPAC meeting, we heard the overwhelming support for our vaccine from physicians, healthcare organizations, and consumers who are eagerly anticipating a protein-based vaccine option. Consistent with submissions to regulatory authorities worldwide, we have already submitted an amendment with updated manufacturing information for the EUA to the FDA for review. We look forward to collaborating with the FDA as it makes its final decision."
The VRBPAC considered data from the pivotal Phase 3 clinical trial, PREVENT-19, which enrolled approximately 30,000 participants aged 18 years and older in the U.S. and Mexico and was published in the New England Journal of Medicine. In the trial, the Novavax COVID-19 vaccine demonstrated 90.4% efficacy (95% confidence interval [CI], 82.9 to 94.6; P<0.001) with a reassuring safety profile. Serious and severe adverse events were low in number and balanced between vaccine and placebo groups. The most common adverse reactions observed during the trial (frequency category of very common ≥1/10) were headache, nausea or vomiting, myalgia, arthralgia, injection site tenderness/pain, fatigue, and malaise. The data showed that overall the rate of myocarditis was balanced between the vaccine and placebo arms (0.007% and 0.005%) and in the post-crossover portions of Novavax trials the observed cases were all within the expected rate.
The FDA considers the recommendations of VRBPAC when making decisions on EUA.
The Novavax COVID-19 vaccine has received authorization for use in individuals aged 18 and over from more than 40 countries in addition to Emergency Use Listing from the World Health Organization.
Authorization in the U.S.
The Novavax COVID-19 vaccine (NVX-CoV2373) has not yet been authorized for use in the U.S.
About NVX-CoV2373
NVX-CoV2373 is a protein-based vaccine engineered from the genetic sequence of the first strain of SARS-CoV-2, the virus that causes COVID-19 disease. The vaccine was created using Novavax' recombinant nanoparticle technology to generate antigen derived from the coronavirus spike (S) protein and is formulated with Novavax' patented saponin-based Matrix-M™ adjuvant to enhance the immune response and stimulate high levels of neutralizing antibodies. NVX-CoV2373 contains purified protein antigen and can neither replicate, nor can it cause COVID-19.
The Novavax COVID-19 vaccine is packaged as a ready-to-use liquid formulation in a vial containing ten doses. The vaccination regimen calls for two 0.5 ml doses (5 mcg antigen and 50 mcg Matrix-M adjuvant) given intramuscularly 21 days apart. The vaccine is stored at 2°- 8° Celsius, enabling the use of existing vaccine supply and cold chain channels. Use of the vaccine should be in accordance with official recommendations.
Novavax has established partnerships for the manufacture, commercialization and distribution of NVX-CoV2373 worldwide. Existing authorizations leverage Novavax' manufacturing partnership with Serum Institute of India, the world's largest vaccine manufacturer by volume. They will later be supplemented with data from additional manufacturing sites throughout Novavax' global supply chain.
About the NVX-CoV2373 Phase 3 Trials
NVX-CoV2373 continues being evaluated in two pivotal Phase 3 trials.
PREVENT-19 (the PRE-fusion protein subunit Vaccine Efficacy Novavax Trial | COVID-19) is a 2:1 randomized, placebo-controlled, observer-blinded trial to evaluate the efficacy, safety and immunogenicity of NVX-CoV2373 with Matrix-M adjuvant in 29,960 participants 18 years of age and over in 119 locations in the U.S. and Mexico. The primary endpoint for PREVENT-19 was the first occurrence of PCR-confirmed symptomatic (mild, moderate or severe) COVID-19 with onset at least seven days after the second dose in serologically negative (to SARS-CoV-2) adult participants at baseline. The statistical success criterion included a lower bound of 95% CI >30%. A secondary endpoint was the prevention of PCR-confirmed, symptomatic moderate or severe COVID-19. Both endpoints were assessed at least seven days after the second study vaccination in volunteers who had not been previously infected with SARS-CoV-2. In the trial, NVX-CoV2373 achieved 90.4% efficacy overall. It was generally well-tolerated and elicited a robust antibody response after the second dose in both studies. Full results of the trial were published in the New England Journal of Medicine (NEJM).
The pediatric expansion of PREVENT-19 is a 2:1 randomized, placebo-controlled, observer-blinded trial to evaluate the safety, effectiveness, and efficacy of NVX-CoV2373 with Matrix-M adjuvant in 2,247 adolescent participants 12 to 17 years of age in 73 locations in the United States, compared with placebo. In the pediatric trial, NVX-CoV2373 achieved its primary effectiveness endpoint (non-inferiority of the neutralizing antibody response compared to young adult participants 18 through 25 years of age from PREVENT-19) and demonstrated 80% efficacy overall at a time when the Delta variant of concern was the predominant circulating strain in the U.S. Additionally, immune responses were about two-to-three-fold higher in adolescents than in adults against all variants studied.
PREVENT-19 is being conducted with support from the U.S. government, including the Department of Defense, the Biomedical Advanced Research and Development Authority (BARDA), part of the Office of the Assistant Secretary for Preparedness and Response at the U.S. Department of Health and Human Services (HHS), and the National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health at HHS. BARDA is providing up to $1.75 billion under a Department of Defense agreement (# MCDC2011-001).
Additionally, a trial conducted in the U.K. with 14,039 participants aged 18 years and over was designed as a randomized, placebo-controlled, observer-blinded study and achieved overall efficacy of 89.7%. The primary endpoint was based on the first occurrence of PCR-confirmed symptomatic (mild, moderate or severe) COVID-19 with onset at least seven days after the second study vaccination in serologically negative (to SARS-CoV-2) adult participants at baseline. Full results of the trial were published in NEJM.
About Matrix-M™ Adjuvant
Novavax' patented saponin-based Matrix-M adjuvant has demonstrated a potent and well-tolerated effect by stimulating the entry of antigen-presenting cells into the injection site and enhancing antigen presentation in local lymph nodes, boosting immune response.
For more information, visit www.novavax.com and connect with us on LinkedIn.
*NanoFlu identifies a recombinant hemagglutinin (HA) protein nanoparticle influenza vaccine candidate produced by Novavax. This investigational candidate was evaluated during a controlled phase 3 trial conducted during the 2019-2020 influenza season.
SOURCE Novavax, Inc.
Novavax COVID vaccine recommended for approval by FDA advisory panel
Jun. 07, 2022 3:29 PM ET
Novavax, Inc. (NVAX)BNTX, PFE, MRNA
By: Jonathan Block, SA News Editor67 Comments
- A panel of FDA advisors has voted to recommend approval of Novavax's COVID-19 vaccine.
- The vote was 21 in favor with one abstention.
- Trading in Novavax (NASDAQ:NVAX) was halted today in advance of the meeting.
- https://seekingalpha.com/news/3846434-novavax-covid-vaccine-recommended-for-approval-by-fda-advisory-panel
- https://seekingalpha.com/symbol/NVAX
- https://www.novavax.com/
biotecMAX™ Feb/Mar/APR/May/June 2022 Insight
Trodelvy® (sacituzumab govitecan-hziy)
June 06, 2022
Final Data From Phase 3 ASCENT Study Demonstrates Trodelvy Extends Overall Survival Over Chemotherapy in Second-Line Metastatic TNBC
– Trodelvy Also Reduced the Risk of Disease Progression or Death by 59% Compared to Physicians’ Choice of Chemotherapy –
CHICAGO--(BUSINESS WIRE)-- Gilead Sciences, Inc. (Nasdaq: GILD) today presented final data from the Phase 3 ASCENT study of Trodelvy® (sacituzumab govitecan-hziy) in patients with relapsed or refractory metastatic triple-negative breast cancer (TNBC) who received two or more prior systemic therapies, at least one of them for metastatic disease. In a follow-up analysis from the final database lock, Trodelvy improved median progression-free survival versus physicians’ choice of chemotherapy (4.8 vs. 1.7 months; HR: 0.41; p<0.0001) and extended median overall survival (OS) by almost five months (11.8 vs. 6.9 months; HR: 0.51; p<0.0001) in the intent-to-treat population. The two-year OS rate was 20.5% (95% CI: 15.4-26.1) in the Trodelvy arm, compared with 5.5% (95% CI: 2.8-9.4) with physicians’ choice of chemotherapy. Trodelvy also showed clinically meaningful improvements in health-related quality of life (HRQoL) compared to chemotherapy. The results, which were consistent with the final analysis previously published in The New England Journal of Medicine, were presented at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting (Abstract #1071).
“These final data from the Phase 3 ASCENT study confirm the survival and quality of life benefit seen with sacituzumab govitecan over traditional chemotherapy in patients with pre-treated metastatic triple-negative breast cancer,” said Aditya Bardia, MD, MPH, Director of Breast Cancer Research Program, Mass General Cancer Center and Associate Professor of Medicine at Harvard Medical School, and global principal investigator of the ASCENT study. “Until now, there was a longstanding gap in effective treatment options which had a severe impact on quality of life and contributed to poor outcomes for these patients.”
Trodelvy demonstrated higher clinically meaningful improvements in all five primary HRQoL domains compared to chemotherapy, which was consistent with previous reports. Metastatic TNBC is often associated with a significant decrease in quality of life, where patients may undergo many rounds of intensive chemotherapy, and assessing impact of symptom burden is especially important in this setting. Changes from baseline for Trodelvy vs. chemotherapy were -5.8 vs. -9.4 in global health status, -4.6 vs. -13.5 in physical functioning, -8.4 vs. -18.8 in role functioning, 5.1 vs. 14.0 in fatigue, and 2.8 vs. 6.8 in pain.
“Trodelvy is the cornerstone of our solid tumor portfolio and the first and only antibody-drug conjugate to demonstrate a statistically significant improvement in overall survival and quality of life versus single-agent chemotherapy in second-line metastatic TNBC,” said Bill Grossman, MD, PhD, Senior Vice President, Therapeutic Area Head, Gilead Oncology. “These final data from ASCENT reinforce Trodelvy as a new standard-of-care option in this setting.”
The safety profile of Trodelvy was consistent with prior reports. Key Grade ≥3 treatment-related adverse reactions for Trodelvy compared to chemotherapy were diarrhea (11% vs. <1%), neutropenia (52% vs. 33%), anemia (8% vs. 5%), and febrile neutropenia (6% vs. 2%). Treatment discontinuations due to adverse events were ≤3% in both arms. The Trodelvy U.S. Prescribing Information has a Boxed Warning for severe or life-threatening neutropenia and severe diarrhea; see below for Important Safety Information.
About the ASCENT Study
The ASCENT study is a global, open-label, randomized Phase 3 study that enrolled more than 500 patients across 230 study locations. The study evaluated the efficacy and safety of Trodelvy compared with a single-agent chemotherapy of the physician’s choice in patients with unresectable, locally advanced or metastatic TNBC who had received at least two prior systemic treatments. Patients were randomized to receive either Trodelvy or a chemotherapy chosen by the patients’ treating physicians. The primary endpoint was PFS (as determined by blinded independent central review) in patients without brain metastases. Secondary endpoints included: PFS for full study population or intention-to-treat (ITT) population, overall survival in both the ITT population and in the subgroup without brain metastasis, independently determined objective response rate, duration of response, time to onset of response according to Response Evaluation Criteria in Solid Tumors (RECIST 1.1), quality of life and safety. More information about ASCENT is available at http://clinicaltrials.gov/show/NCT02574455.
About Trodelvy
Trodelvy® (sacituzumab govitecan-hziy) is a first-in-class Trop-2 directed antibody-drug conjugate. Trop-2 is a cell surface antigen highly expressed in multiple tumor types, including in more than 90% of breast and bladder cancers. Trodelvy is intentionally designed with a proprietary hydrolyzable linker attached to SN-38, a topoisomerase I inhibitor payload. This unique combination delivers potent activity to both Trop-2 expressing cells and the microenvironment.
Trodelvy is approved in more than 35 countries, with multiple additional regulatory reviews underway worldwide, for the treatment of adult patients with unresectable locally advanced or metastatic triple-negative breast cancer (TNBC) who have received two or more prior systemic therapies, at least one of them for metastatic disease. Trodelvy is also approved in the U.S. under the accelerated approval pathway for the treatment of adult patients with locally advanced or metastatic urothelial cancer (UC) who have previously received a platinum-containing chemotherapy and either programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor.
Trodelvy is also being developed for potential investigational use in other TNBC and metastatic UC populations, as well as a range of tumor types where Trop-2 is highly expressed, including hormone receptor-positive/human epidermal growth factor receptor 2-negative (HR+/HER2-) metastatic breast cancer, metastatic non-small cell lung cancer (NSCLC), metastatic small cell lung cancer (SCLC), head and neck cancer, and endometrial cancer.
U.S. Indications for Trodelvy
In the United States, Trodelvy is indicated for the treatment of:
- Adult patients with unresectable locally advanced or metastatic TNBC who have received two or more prior systemic therapies, at least one of them for metastatic disease.
- Adult patients with locally advanced or metastatic UC who have previously received a platinum-containing chemotherapy and either programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
- https://www.trodelvy.com/
Please see full Prescribing Information
U.S. Prescribing Information for Trodelvy including BOXED WARNING, is available at www.gilead.com .
Trodelvy, Gilead and the Gilead logo are trademarks of Gilead Sciences, Inc., or its related companies.
For more information about Gilead, please visit the company’s website at www.gilead.com , follow Gilead on Twitter (@GileadSciences) or call Gilead Public Affairs at 1-800-GILEAD-5 or 1-650-574-3000.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220605005087/en/
Source: Gilead Sciences, Inc.
Gilead breast cancer drug cuts disease progression and improves survival by 59%
Jun. 06, 2022 9:30 AM ET
By: Dulan Lokuwithana, SA News Editor5 Comments
Announcing final data from a Phase 3 study, Gilead (NASDAQ:GILD) said on Monday that its antibody-drug conjugate, Trodelvy reduced the risk of disease progression and improved survival by as much as 59% in metastatic triple-negative breast cancer (TNBC) versus chemotherapy.
https://seekingalpha.com/symbol/GILD
LAGEVRIO™ (molnupiravir)
Merck and Ridgeback Announce New Data For Investigational LAGEVRIO™ (molnupiravir) From Phase 3 MOVe-OUT Study
June 7, 2022 6:45 am ET
Based on Prespecified Exploratory Endpoints, a Lower Proportion of Participants Treated With LAGEVRIO Had an Acute Care Visit Compared to Those Who Received Placebo
Based on a Post Hoc Analysis, Fewer Required Respiratory Interventions (Including Invasive Mechanical Ventilation)
RAHWAY, N.J. & MIAMI--(BUSINESS WIRE)-- Merck, known as MSD outside the United States and Canada, and Ridgeback Biotherapeutics today announced the Annals of Internal Medicine has published additional data from the Phase 3 MOVe-OUT trial evaluating LAGEVRIO™ (molnupiravir), an investigational oral antiviral medicine, in non-hospitalized adults with mild to moderate COVID-19 who were at high risk for progressing to severe disease.
Analyses of pre-specified exploratory endpoints indicate that a lower proportion of LAGEVRIO-treated participants in the modified intent-to-treat (MITT) population had an acute care visit or a COVID-19-related acute care visit versus placebo-treated participants in the MITT population: 7.2% of participants who received LAGEVRIO reported an acute care visit through Day 29, versus 10.6% of placebo participants, with a relative risk reduction [RRR] of 32.1% [CI, 4.4% to 51.7%]; 6.6% of participants who received LAGEVRIO reported a COVID-19-related acute care visit, versus 10.0% of placebo participants, with a RRR of 33.8% [CI, 5.6% to 53.6%]. The MITT population included all participants who were randomly assigned, received at least one dose of study drug, and were not hospitalized before the first dose of study drug. Based on a post hoc analysis, fewer LAGEVRIO-treated participants in the MITT population required respiratory interventions (including conventional oxygen therapy, a high-flow heated and humidified device, noninvasive mechanical ventilation, or invasive mechanical ventilation) versus placebo-treated participants, with a RRR of 34.3% [95% CI, 4.3% to 54.9%] for all respiratory interventions. Based on additional post hoc analyses, participants in the safety population who received LAGEVRIO showed earlier and larger reductions in mean C-reactive protein (CRP) values, and earlier and larger improvements in mean change from baseline oxygen saturation (SpO2) values, compared with participants who received placebo. The safety population consisted of all participants who had undergone randomization and had received at least one dose of LAGEVRIO.
Post hoc analyses also suggest that among the subgroup of participants who were hospitalized after randomization in MOVe-OUT, the median time to hospital discharge was nine days [CI, 7 to 12 days] for participants who received LAGEVRIO, versus 12 days [CI, 9 to 14 days] in the placebo group. Consistent with the full MITT population data, post hoc analyses also suggest that fewer LAGEVRIO-treated participants who were hospitalized after randomization required respiratory interventions versus placebo-treated participants, with a RRR of 21.3% [95% CI, 0.2% to 38.0%] for all respiratory interventions.
“The analyses add to our understanding of the clinical profile of LAGEVRIO and help to reinforce the importance of LAGEVRIO as part of the response to the COVID-19 pandemic,” said Dr. Dean Y. Li, president, Merck Research Laboratories.
“The primary data from MOVe-OUT demonstrated a significant reduction in the risk for progression to severe COVID-19, including hospitalization and death, when compared to placebo among non-hospitalized, at-risk patients. In light of the continued burden of COVID-19, we are encouraged by these new data,” said Wendy Holman, chief executive officer, Ridgeback Biotherapeutics. “We look forward to continuing to study LAGEVRIO with the goal of helping high-risk patients and overburdened healthcare systems globally continue to combat the COVID-19 pandemic.”
In addition to the MOVe-OUT trial, LAGEVRIO is being evaluated for post-exposure prophylaxis in MOVe-AHEAD, a global, multicenter, randomized, double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of LAGEVRIO in preventing the spread of COVID-19 within households.
About the MOVe-OUT Study
The Phase 3 MOVe-OUT clinical trial (NCT04575597) evaluated LAGEVRIO (molnupiravir) 800 mg twice-daily in non-hospitalized, unvaccinated adult patients with laboratory-confirmed mild to moderate COVID-19, symptom onset within five days of study randomization, and at least one risk factor associated with poor disease outcomes (e.g. heart disease, diabetes). The primary efficacy objective of MOVe-OUT was to evaluate the efficacy of LAGEVRIO 800 mg twice-daily for five days compared to placebo as assessed by the percentage of participants who were hospitalized and/or died through Day 29. These findings were published in the New England Journal of Medicine.
In analyses from all randomized patients (n=1433) in the MITT population, LAGEVRIO reduced the risk of hospitalization or death: 9.7% (68/699) of patients in the placebo group were hospitalized or died through Day 29 compared to 6.8% (48/709) of patients who received LAGEVRIO, for an absolute risk reduction of 3.0% (95% confidence interval [CI]: 0.1, 5.9). Nine deaths were reported in the placebo group, and one in the LAGEVRIO group.
The determination of primary efficacy was based on a planned interim analysis of 762 participants. At the interim analysis, treatment with LAGEVRIO significantly reduced the risk for hospitalizations and death through Day 29 following randomization: 14.1% (53/377) of patients in the placebo group were hospitalized or died, compared to 7.3% (28/385) of patients who received LAGEVRIO. The absolute risk reduction between the LAGEVRIO and the placebo arm was 6.8 percentage points (95% CI: 2.4, 11.3; p=0.0024).
The safety of LAGEVRIO was evaluated based on an analysis of MOVe-OUT in which 1,411 non-hospitalized subjects with COVID-19 were randomized and treated with LAGEVRIO (N=710) or placebo (N=701) for up to 5 days. Adverse events were those reported while subjects were on study intervention or within 14 days of study intervention completion/discontinuation. The most common adverse reactions for LAGEVRIO (incidence ≥1%) were diarrhea (2% for LAGEVRIO, 2% for placebo), nausea (1% for LAGEVRIO, 1% for placebo) and dizziness (1% for LAGEVRIO, 1% for placebo). Discontinuation of study intervention due to an adverse event (AE) occurred in 1% of subjects receiving LAGEVRIO and 3% of subjects receiving placebo. Serious AEs occurred in 7% of subjects receiving LAGEVRIO and 10% receiving placebo; most serious AEs were COVID-19 related.
About Merck’s Global Efforts to Accelerate Access to LAGEVRIO (molnupiravir) Following Regulatory Authorizations or Approvals
Global access has been a priority for Merck and Ridgeback since the inception of their LAGEVRIO collaboration. The companies are committed to providing timely access to LAGEVRIO globally through our comprehensive supply and access approach, which includes:
Supply: As of May 31, 2022, Merck has supplied more than 8 million courses of treatment to governments in more than 30 markets worldwide.
Voluntary licenses: As part of its commitment to widespread global access, Merck granted voluntary licenses (VLs) to generics manufacturers and to the Medicines Patent Pool to make generic molnupiravir available in more than 100 low- and middle-income countries following approvals or emergency authorization by local regulatory agencies. Through our VL agreements with generics manufacturers, more than 3 million courses of generic molnupiravir have been delivered to approximately 15 markets through March 2022.
Authorized Use of LAGEVRIO in the U.S.
The U.S. Food and Drug Administration (FDA) has issued an EUA for the emergency use of the unapproved product LAGEVRIO, a nucleoside analogue that inhibits SARS-CoV-2 replication by viral mutagenesis, for the treatment of mild to moderate coronavirus disease 2019 (COVID-19) in adults with positive results of direct SARS-CoV-2 viral testing, and who are at high risk for progression to severe COVID-19, including hospitalization or death, and for whom alternative COVID-19 treatment options approved or authorized by FDA are not accessible or clinically appropriate. LAGEVRIO is not FDA-approved for any use including for use for the treatment of COVID-19.
The emergency use of LAGEVRIO is only authorized for the duration of the declaration that circumstances exist justifying the authorization of the emergency use of drugs and biological products during the COVID-19 pandemic under Section 564(b)(1) of the Federal Food, Drug, and Cosmetic Act, 21 U.S.C. § 360bbb-3(b)(1) unless the declaration is terminated or authorization revoked sooner.
LAGEVRIO is not authorized for use in patients less than 18 years of age or for initiation of treatment in patients hospitalized due to COVID-19. Benefit of treatment with LAGEVRIO has not been observed in subjects when treatment was initiated after hospitalization due to COVID-19. LAGEVRIO is not authorized for use for longer than five consecutive days. LAGEVRIO is not authorized for pre-exposure or post-exposure prophylaxis for prevention of COVID-19. LAGEVRIO may only be prescribed for an individual patient by physicians, advanced practice registered nurses, and physician assistants that are licensed or authorized under state law to prescribe drugs in the therapeutic class to which LAGEVRIO belongs (i.e., anti-infectives).
About LAGEVRIO (molnupiravir)
LAGEVRIO(molnupiravir) (MK-4482) is an investigational, orally administered nucleoside analog that inhibits the replication of SARS-CoV-2, the causative agent of COVID-19.
Merck and Ridgeback’s “orange COVID-19 pill” is a Swedish Orange opaque capsule with the Merck corporate logo and “82” printed in white ink, available in certain markets as LAGEVRIO.
Results from the Phase 3 MOVe-OUT study demonstrated the efficacy benefit of LAGEVRIO treatment was generally consistent across patients infected with SARS-CoV-2 variants of concern, Delta, Gamma and Mu. Preclinical data has shown that LAGEVRIO has antiviral activity against the variant, Omicron (B1.1.529). LAGEVRIO has yet to be evaluated against Omicron in clinical studies.
Molnupiravir was invented at Emory University. Drug Innovation Ventures at Emory (DRIVE), LLC, which was formed by Emory to develop early-stage drug candidates for viral diseases of global concern, advanced molnupiravir through IND submission. Emory/DRIVE received some research funding from the U.S. Department of Defense and the U.S. National Institutes of Health. LAGEVRIO is being developed by Merck in collaboration with Ridgeback Biotherapeutics. Ridgeback received an upfront payment from Merck and also is eligible to receive contingent payments dependent upon the achievement of certain developmental and regulatory approval milestones. Any profits from the collaboration will be split between the partners equally. Since licensed by Ridgeback, all funds used for the development of LAGEVRIO have been provided by Merck and Ridgeback.
LAGEVRIO was evaluated in MOVe-OUT, a global Phase 3, randomized, placebo-controlled, double-blind, multi-site study of non-hospitalized adult patients with symptomatic, laboratory-confirmed mild to moderate COVID-19 and at least one risk factor associated with poor disease outcomes. The Phase 3 portion of the MOVe-OUT trial was conducted globally in more than 170 sites in locations including Argentina, Brazil, Canada, Chile, Colombia, Egypt, France, Germany, Guatemala, Israel, Italy, Mexico, Philippines, Poland, Russia, South Africa, Spain, Sweden, Taiwan, Ukraine, the United Kingdom and the United States. For further information about the MOVe-OUT trial, please visit clinicaltrials.gov. Molnupiravir is also being evaluated for post-exposure prophylaxis in MOVe-AHEAD, a global, multicenter, randomized, double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of molnupiravir in preventing the spread of COVID-19 within households. For more information, please visit http://merckcovidresearch.com.
Please visit the Merck media library for molnupiravir images and b-roll.
About Ridgeback Biotherapeutics
Headquartered in Miami, Florida, Ridgeback Biotherapeutics LP is a biotechnology company focused on emerging infectious diseases. Ridgeback markets EbangaTM for the treatment of Ebola and has a late-stage development pipeline which includes molnupiravir for the treatment of COVID-19. The team at Ridgeback is dedicated to developing life-saving and life-changing solutions for patients and diseases that need champions as well as providing global access to these medicines. In line with Ridgeback’s mission for equitable global access, all Ridgeback services and treatment for Ebola patients in Africa are delivered free of charge.
https://www.molnupiravir-us.com/patients/
For more information, visit www.merck.com and connect with us on Twitter, Facebook, Instagram, YouTube and LinkedIn.
Source: Merck & Co., Inc.
Merck, Ridgeback say COVID-19 pill cut risk of hospitalizations by 34%
Jun. 07, 2022 7:24 AM ET
By: Dulan Lokuwithana, SA News Editor
Updating data from the Phase 3 MOVe-OUT trial, Merck (NYSE:MRK) and Ridgeback Biotherapeutics announced on Tuesday that the COVID-19 pill the companies jointly developed cut the risk of COVID-related acute care visits by nearly 34%.
https://seekingalpha.com/news/3846184-merck-ridgeback-say-covid-19-pill-cut-risk-of-hospitalizations
https://seekingalpha.com/symbol/MRK
https://www.molnupiravir-us.com/patients/
ORLADEYO® (berotralstat)
BioCryst Announces Health Canada has Authorized ORLADEYO® (berotralstat), the Only Oral Treatment for the Prevention of Hereditary Angioedema Attacks
RESEARCH TRIANGLE PARK, N.C., June 06, 2022 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that Health Canada has approved oral, once-daily ORLADEYO® (berotralstat) for the routine prevention of recurrent hereditary angioedema (HAE) attacks in patients 12 years and older.
“Today is a momentous day for the Canadian HAE community,” said Jacquie Badiou, president of HAE Canada. “HAE carries with it a severe burden on patients and their families, much of which stems from the unpredictable attacks they experience even when actively managing their disease. We have seen important treatment advancements in recent years, and I believe the first oral preventive therapy for HAE will be a welcome option for many Canadians who are living with this rare condition.”
“I believe this oral, once-daily prophylactic therapy will be a meaningful treatment option for patients who need a prophylactic therapy to help manage their HAE by reducing the frequency of attacks,” said Stephen Betschel, HBSc, MD, FRCPC, FAAAAI, Chair of the Canadian Hereditary Angioedema Network.
“We appreciate Health Canada’s thorough and timely review of ORLADEYO, and with this authorization we are one step closer to bringing this important treatment option to Canadians living with HAE,” said Jared Rhines, general manager of BioCryst Canada. “We look forward to working with stakeholders across the country to ensure these Canadians can receive timely and appropriate access to ORLADEYO. Further, this authorization follows approvals in the US, Europe and other markets, highlighting our ongoing commitment to individuals living with rare diseases regardless of geography.”
ORLADEYO was safe and well tolerated in clinical trials. The most frequently reported adverse reactions in patients receiving ORLADEYO compared with placebo were gastrointestinal reactions. These reactions generally occurred early after initiation of treatment with ORLADEYO, became less frequent with time and typically self-resolved.
About ORLADEYO® (berotralstat)
ORLADEYO® (berotralstat) is the first and only oral therapy designed specifically to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years and older. One capsule of ORLADEYO per day works to prevent HAE attacks by decreasing the activity of plasma kallikrein.
U.S. Indication and Important Safety Information
INDICATION
ORLADEYO® (berotralstat) is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older.
Please see full Prescribing Information.
About BioCryst Pharmaceuticals
BioCryst Pharmaceuticals discovers novel, oral, small-molecule medicines that treat rare diseases in which significant unmet medical needs exist and an enzyme plays a key role in the biological pathway of the disease. Oral, once-daily ORLADEYO® (berotralstat) is approved in the United States and multiple global markets. BioCryst has several ongoing development programs including BCX9930, an oral Factor D inhibitor for the treatment of complement-mediated diseases, BCX9250, an ALK-2 inhibitor for the treatment of fibrodysplasia ossificans progressiva, and galidesivir, a potential treatment for Marburg virus disease and yellow fever. RAPIVAB® (peramivir injection) is approved in the U.S. and multiple global markets, with post-marketing commitments ongoing. For more information, please visit the company’s website at www.biocryst.com.
BioCryst Orladeyo gets approval in Canada to prevent hereditary angioedema attacks
Jun. 06, 2022 8:31 AM ET
BioCryst Pharmaceuticals, Inc. (BCRX)
By: Ravikash, SA News Editor
- Health Canada approved BioCryst Pharmaceuticals' (NASDAQ:BCRX) once-daily oral drug Orladeyo (berotralstat) for the routine prevention of recurrent hereditary angioedema (HAE) attacks in patients 12 years and older.
- https://seekingalpha.com/news/3845876-biocryst-orladeyo-gets-approval-in-canada-to-prevent-hereditary-angioedema-attacks
- https://seekingalpha.com/symbol/BCRX
- https://orladeyo.com/
- https://ir.biocryst.com/
Opdivo (nivolumab) Plus Yervoy (ipilimumab) with Two Cycles of Chemotherapy
Three-Year Data from Phase 3 CheckMate -9LA Trial Demonstrate Long-Term, Durable Survival Outcomes of Opdivo (nivolumab) Plus Yervoy (ipilimumab) with Two Cycles of Chemotherapy for Patients with Metastatic Non-Small Cell Lung Cancer
06/06/2022CATEGORY:
Dual immunotherapy-based combination resulted in clinical benefits across key subgroups of mNSCLC patients with high unmet need, including those with PD-L1 expression <1%
Late-breaking data to be presented during the 2022 American Society of Clinical Oncology Annual Meeting
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced three-year follow-up results from the Phase 3 CheckMate -9LA trial demonstrating durable survival benefits with Opdivo (nivolumab) plus Yervoy (ipilimumab) with two cycles of chemotherapy compared to four cycles of chemotherapy in previously untreated patients with metastatic non-small cell lung cancer (mNSCLC). With a minimum follow-up of three years (36.1 months), the dual immunotherapy-based combination continued to show sustained improvement in overall survival (OS), the trial’s primary endpoint, with 27% of patients treated with Opdivo plus Yervoy with two cycles of chemotherapy alive compared to 19% of patients treated with chemotherapy alone at three years (Hazard Ratio [HR] 0.74; 95% Confidence Interval [CI]: 0.62 to 0.87).
The long-term, durable clinical benefit of Opdivo plus Yervoy with two cycles of chemotherapy was observed at three years across patient populations that typically have a poor prognosis, including patients with PD-L1 expression <1% and squamous histology.
- PD-L1 <1%: Among patients with tumor PD-L1 expression <1%, the OS rate was 25% for those treated with the dual immunotherapy-based combination vs. 15% for chemotherapy alone.
- Squamous histology: Among those with squamous histology, the OS rate was 24% for patients who received Opdivo plus Yervoy with chemotherapy, compared to 11% for those who received chemotherapy alone.
- In an exploratory analysis, a positive trend for OS benefit was also observed with Opdivo plus Yervoy with chemotherapy among patients with certain tumor mutations, such as STK11.
No new safety signals were observed with Opdivo plus Yervoy with two cycles of chemotherapy with extended follow-up in the CheckMate -9LA trial. These data will be featured in a late-breaking poster presentation (Abstract #LBA9026) at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting on June 6, 2022, from 9:00 a.m. to 12:00 p.m. EDT.
“While immunotherapy treatments have greatly improved outcomes for people with metastatic NSCLC, many patients, particularly those with low PD-L1 expression, are unfortunately not achieving durable, long-term survival,” said Luis G. Paz-Ares, M.D., Ph.D., CheckMate -9LA study investigator and chair of the medical oncology department, Hospital Universitario 12 De Octubre in Madrid, Spain. “The three-year data from CheckMate -9LA demonstrate the ongoing durability with early disease control following treatment with the combination of nivolumab plus ipilimumab, with a short course of chemotherapy. Importantly, we saw long-term benefit among patients with high unmet needs, such as those with PD-L1 expression <1% who tend to have worse outcomes and fewer treatment options.”
“Our research in thoracic cancers has led to different treatment choices that offer patients the best chance for long-term survival,” said Abderrahim Oukessou, M.D., vice president, thoracic cancers development lead, Bristol Myers Squibb. “The sustained improvement in overall survival outcomes demonstrated by Opdivo plus Yervoy plus two cycles of chemotherapy in non-small cell lung cancer builds on the established evidence for Opdivo plus Yervoy to change survival outcomes across a broad range of advanced cancers. With this, in addition to recent progress in earlier-stage disease, we’re seeing the full potential of Opdivo-based regimens to transform the lives of patients with thoracic cancers.”
Opdivo plus Yervoy-based combinations have shown significant improvements in OS in six Phase 3 clinical trials in five tumors to date: metastatic NSCLC, metastatic melanoma, advanced renal cell carcinoma, malignant pleural mesothelioma and esophageal squamous cell carcinoma.
About CheckMate -9LA
CheckMate -9LA is an open-label, global multi-center, randomized Phase 3 trial evaluating Opdivo (360 mg Q3W) plus Yervoy (1 mg/kg Q6W) combined with chemotherapy (two cycles) compared to chemotherapy alone (up to four cycles followed by optional pemetrexed maintenance therapy if eligible) as a first-line treatment in patients with metastatic non-small cell lung cancer (NSCLC) regardless of PD-L1 expression and histology. Patients in the experimental arm (n=361) were treated with immunotherapy for up to two years or until disease progression or unacceptable toxicity. Patients in the control arm (n=358) were treated with up to four cycles of chemotherapy and optional pemetrexed maintenance (if eligible) until disease progression or unacceptable toxicity. The primary endpoint of the trial was overall survival (OS) in the intent-to-treat (ITT) population. Secondary hierarchical endpoints included progression-free survival (PFS) and overall response rate (ORR), and the study also evaluated efficacy measures according to biomarkers.
About Opdivo
Opdivo is a programmed death-1 (PD-1) immune checkpoint inhibitor that is designed to uniquely harness the body’s own immune system to help restore anti-tumor immune response. By harnessing the body’s own immune system to fight cancer, Opdivo has become an important treatment option across multiple cancers.
Opdivo’s leading global development program is based on Bristol Myers Squibb’s scientific expertise in the field of Immuno-Oncology, and includes a broad range of clinical trials across all phases, including Phase 3, in a variety of tumor types. To date, the Opdivo clinical development program has treated more than 35,000 patients. The Opdivo trials have contributed to gaining a deeper understanding of the potential role of biomarkers in patient care, particularly regarding how patients may benefit from Opdivo across the continuum of PD-L1 expression.
In July 2014, Opdivo was the first PD-1 immune checkpoint inhibitor to receive regulatory approval anywhere in the world. Opdivo is currently approved in more than 65 countries, including the United States, the European Union, Japan and China. In October 2015, the Company’s Opdivo and Yervoy combination regimen was the first Immuno-Oncology combination to receive regulatory approval for the treatment of metastatic melanoma and is currently approved in more than 50 countries, including the United States and the European Union.
About Yervoy
Yervoy is a recombinant, human monoclonal antibody that binds to the cytotoxic T-lymphocyte-associated antigen-4 (CTLA-4). CTLA-4 is a negative regulator of T-cell activity. Yervoy binds to CTLA-4 and blocks the interaction of CTLA-4 with its ligands, CD80/CD86. Blockade of CTLA-4 has been shown to augment T-cell activation and proliferation, including the activation and proliferation of tumor infiltrating T-effector cells. Inhibition of CTLA-4 signaling can also reduce T-regulatory cell function, which may contribute to a general increase in T-cell responsiveness, including the anti-tumor immune response. On March 25, 2011, the U.S. Food and Drug Administration (FDA) approved Yervoy 3 mg/kg monotherapy for patients with unresectable or metastatic melanoma. Yervoy is approved for unresectable or metastatic melanoma in more than 50 countries. There is a broad, ongoing development program in place for Yervoy spanning multiple tumor types.
INDICATIONS
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of adult patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of adult patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of adult patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection.
OPDIVO® (nivolumab), in combination with platinum-doublet chemotherapy, is indicated as neoadjuvant treatment of adult patients with resectable (tumors ≥4 cm or node positive) non-small cell lung cancer (NSCLC).
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express PD-L1 (≥1%) as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab) and 2 cycles of platinum-doublet chemotherapy, is indicated for the first-line treatment of adult patients with metastatic or recurrent non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving OPDIVO.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with unresectable malignant pleural mesothelioma (MPM).
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with intermediate or poor risk advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab), in combination with cabozantinib, is indicated for the first-line treatment of adult patients with advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy.
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with classical Hodgkin lymphoma (cHL) that has relapsed or progressed after autologous hematopoietic stem cell transplantation (HSCT) and brentuximab vedotin or after 3 or more lines of systemic therapy that includes autologous HSCT. This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN) with disease progression on or after platinum-based therapy.
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
OPDIVO® (nivolumab), as a single agent, is indicated for the adjuvant treatment of adult patients with urothelial carcinoma (UC) who are at high risk of recurrence after undergoing radical resection of UC.
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of adult and pediatric (12 years and older) patients with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of adult patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) after prior fluoropyrimidine- and platinum-based chemotherapy.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of completely resected esophageal or gastroesophageal junction cancer with residual pathologic disease in adult patients who have received neoadjuvant chemoradiotherapy (CRT).
OPDIVO® (nivolumab), in combination with fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of adult patients with unresectable advanced or metastatic esophageal squamous cell carcinoma (ESCC).
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with unresectable advanced or metastatic esophageal squamous cell carcinoma (ESCC).
OPDIVO® (nivolumab), in combination with fluoropyrimidine- and platinum- containing chemotherapy, is indicated for the treatment of adult patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma.
Please see U.S. Full Prescribing Information for OPDIVO and YERVOY.
https://www.opdivo.com/about-opdivo/how-the-combination-works-combinationtherapy
For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.
Source: Bristol Myers Squibb
Bristol Myers Opdivo/Yervoy combo shows survival benefit for lung cancer patients in 3-year data
Jun. 06, 2022 7:49 AM ET
Bristol-Myers Squibb Company (BMY)
By: Ravikash, SA News Editor
Bristol Myers Squibb (NYSE:BMY) said 3-year follow-up data from a late stage study showed durable survival benefits with Opdivo plus Yervoy with two cycles of chemotherapy compared to four cycles of chemotherapy in previously untreated patients with metastatic non-small cell lung cancer (mNSCLC).
https://seekingalpha.com/symbol/BMY
https://www.opdivo.com/about-opdivo/how-the-combination-works-combinationtherapy
JARDIANCE® (empagliflozin tablets), for oral use
Jardiance® decreased relative risk of hospitalization for heart failure by 50% versus DPP-4 inhibitors and by 30% versus GLP-1 receptor agonists in adults with type 2 diabetes in real-world evidence study
June 5, 2022Download PDF
- Jardiance showed a reduction in relative risk of all-cause mortality by 40% compared with DPP-4 inhibitors in the subset of people with Medicare coverage
- Data from these final analyses of EMPRISE complements findings from the landmark EMPA-REG OUTCOME® trial
RIDGEFIELD, Conn. and INDIANAPOLIS, June 5, 2022 /PRNewswire/ -- Two analyses of the final U.S. data from the EMPagliflozin compaRative effectIveness and SafEty (EMPRISE) real-world study show that Jardiance® (empagliflozin) was associated with a reduction in risk of hospitalization for heart failure compared with two other classes of glucose-lowering therapies in adults with type 2 diabetes in routine care. Relative risk reductions were 50% versus dipeptidyl peptidase-4 (DPP-4) inhibitors and 30% versus glucagon-like peptide 1 (GLP-1) receptor agonists. The results were presented on behalf of Boehringer Ingelheim and Eli Lilly and Company (NYSE: LLY) at the American Diabetes Association Scientific Sessions 2022 in New Orleans.
"With more than 29 million people in the U.S. diagnosed with type 2 diabetes, up to 22% of whom may also have heart failure, it is critical that healthcare professionals caring for this population have treatments that demonstrate cardiovascular effectiveness in routine care," said Elisabetta Patorno, M.D., Dr.P.H., Division of Pharmacoepidemiology and Pharmacoeconomics, Brigham and Women's Hospital and associate professor of medicine, Harvard Medical School, and study co-investigator. "These five-year results from EMPRISE, showing empagliflozin was associated with a decreased risk of hospitalization for heart failure and for death, are encouraging data for adults with type 2 diabetes and their care team."
Compared with DPP-4 inhibitors, Jardiance was also associated with a 40% reduction in relative risk of all-cause mortality in people who had Medicare. In the overall EMPRISE population, Jardiance was associated with a 12% reduction in the risk of the composite outcome of myocardial infarction or stroke compared with DPP-4 inhibitors.
Compared with GLP-1 receptor agonists, Jardiance was associated with similar risks of heart attack, stroke and all-cause mortality. All results for Jardiance compared with GLP-1 receptor agonists, and with liraglutide (a GLP-1 receptor agonist) specifically, were consistent for people with and without cardiovascular disease.
Results from the EMPRISE real-world study, which assessed the first five years of use of Jardiance in the U.S., complement previously reported data from the landmark EMPA-REG OUTCOME® trial, in which Jardiance showed a 35% relative risk reduction in hospitalization for heart failure compared with placebo in adults with type 2 diabetes and established cardiovascular disease. EMPA-REG OUTCOME also showed a 38% relative risk reduction in cardiovascular death with Jardiance versus placebo.
"People with type 2 diabetes are four times as likely to be hospitalized for heart failure, and repeated hospitalizations lead to worse outcomes, making treatment options that improve clinical outcomes crucial," said Mohamed Eid, M.D., M.P.H., M.H.A., vice president, Clinical Development & Medical Affairs, Cardio-Metabolism & Respiratory Medicine, Boehringer Ingelheim Pharmaceuticals, Inc. "These findings suggest that treatment with Jardiance can help reduce heart failure hospitalizations compared to other classes of diabetes therapies, with a well-understood safety and tolerability profile."
The EMPRISE findings confirmed the well-established safety profile of Jardiance. Compared with DPP-4 inhibitors, Jardiance was associated with a reduction in relative risk of acute kidney injury. There was an increase in relative risk of hospitalization for diabetic ketoacidosis, which is consistent with Jardiance's known safety information. Risks for lower-limb amputations, fractures and renal and bladder cancers were similar.
"We are pleased to present the final data from EMPRISE in the U.S., which encompasses information from nearly 500,000 adults in a real-world care setting," said Leonard Glass, M.D., F.A.C.E., vice president of Diabetes Global Medical Affairs, Lilly. "As we strive to help fill unmet treatment needs, this study reinforces our longstanding commitment to people living with cardio-metabolic conditions. Building upon our robust clinical trial program, EMPRISE highlights the potential of Jardiance to improve health outcomes in routine clinical practice."
About EMPRISE
EMPRISE was initiated in 2016 to complement the EMPA-REG OUTCOME trial results by providing data on the comparative effectiveness and safety of Jardiance in routine clinical care versus DPP-4 inhibitors or GLP-1 receptor agonists.
The final analyses of EMPRISE U.S. data assessed the first five years of Jardiance use in nearly 500,000 adults with type 2 diabetes, with and without cardiovascular disease, in the U.S. between 2014 and 2019: the first included more than 230,000 adults who initiated either Jardiance or a DPP-4 inhibitor (115,116 adults in each treatment arm); the second included more than 260,000 adults who initiated either Jardiance or a GLP-1 receptor agonist (130,408 adults in each treatment arm).
Additional EMPRISE studies including Asia and Europe will provide insights from different regions of the world with an international perspective on the use of Jardiance in routine clinical care.
EMPRISE U.S. was initiated and led by academic researchers from the Division of Pharmacoepidemiology at Brigham and Women's Hospital and Harvard Medical School, Boston. The study is part of an academic collaboration between Brigham and Women's Hospital and Boehringer Ingelheim.
Prioritizing Cardio-Renal-Metabolic Care
Through research and educational initiatives, Boehringer Ingelheim and Lilly are driven to redefine care for people with cardio-renal-metabolic conditions, a group of interconnected disorders that affect more than one billion people worldwide and are a leading cause of death.
The cardiovascular, renal (kidney) and metabolic systems are closely intertwined and share many of the same disease-related pathways. Dysfunction in one system may accelerate the onset of dysfunction in others, resulting in the progression of comorbid diseases such as type 2 diabetes, heart failure and chronic kidney disease. Conversely, improving the health of one system can lead to positive effects across the others and can help reduce the risk for further complications.
Understanding their interconnected nature, we are working to advance treatments for people with cardio-renal-metabolic conditions. It is only through a holistic approach to care that we can truly transform outcomes and restore the harmony among these critical systems.
What is JARDIANCE?
JARDIANCE is a prescription medicine used to:
- reduce the risk of cardiovascular death and hospitalization for heart failure in adults with heart failure, when the heart cannot pump enough blood to the rest of your body
- reduce the risk of cardiovascular death in adults with type 2 diabetes who also have known cardiovascular disease
- lower blood sugar along with diet and exercise in adults with type 2 diabetes
JARDIANCE is not for people with type 1 diabetes. It may increase their risk of diabetic ketoacidosis (increased ketones in the blood or urine).
JARDIANCE is not for use to lower blood sugar in adults with type 2 diabetes who have severe kidney problems, because it may not work.
IMPORTANT SAFETY INFORMATION
Do not take JARDIANCE if you are allergic to empagliflozin or any of the ingredients in JARDIANCE.
Do not take JARDIANCE if you are on dialysis.
For more information, please see Prescribing Information and Medication Guide.
Boehringer Ingelheim and Eli Lilly and Company
In January 2011, Boehringer Ingelheim and Eli Lilly and Company announced an Alliance that centers on compounds representing several of the largest diabetes treatment classes. Depending on geographies, the companies either co-promote or separately promote the respective molecules each contributing to the Alliance. The Alliance leverages the strengths of two of the world's leading pharmaceutical companies to focus on patient needs. By joining forces, the companies demonstrate their commitment, not only to the care of people with diabetes, but also to investigating the potential to address areas of unmet medical need.
To learn more, visit lilly.com and lilly.com/newsroom or follow us on Facebook, Instagram, Twitter and LinkedIn.
Boehringer Ingelheim is committed to improving lives and strengthening our communities. Please visit www.boehringer-ingelheim.us/csr to learn more about Corporate Social Responsibility initiatives.
For more information, please visit www.boehringer-ingelheim.us, or follow us on Twitter @BoehringerUS.
Jardiance® and EMPA-REG OUTCOME® are registered trademarks of Boehringer Ingelheim.
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SOURCE Eli Lilly and Company
Lilly/Boehringer Jardiance cuts risk of heart failure hospitalization for diabetes patients in 5-year data
Jun. 06, 2022 6:45 AM ET
By: Ravikash, SA News Editor
Eli Lilly (NYSE:LLY) and Boehringer Ingelheim said real-world study data showed that Jardiance helped reduce the risk of hospitalization for heart failure compared with two other classes of glucose-lowering therapies in adults with type 2 diabetes in routine care.
https://seekingalpha.com/symbol/LLY
https://docs.boehringer-ingelheim.com/Prescribing%20Information/PIs/Jardiance/jardiance.pdf
CARVYKTI™ (ciltacabtagene autoleucel)
Longer-term Data from CARTITUDE-1 Study Demonstrate Continued Deep and Durable Responses to CARVYKTI™ (ciltacabtagene autoleucel) in Heavily Pretreated Patients with Relapsed or Refractory Multiple Myeloma
Jun 04, 2022United States
At nearly 28 months of median follow-up, median progression-free survival
and overall survival were not yet reached
Data presented at the 2022 ASCO Annual Meeting and published in
the Journal of Clinical Oncology
CHICAGO, June 4, 2022 -- The Janssen Pharmaceutical Companies of Johnson & Johnson announced today updated results from the Phase 1b/2 CARTITUDE-1 study evaluating the efficacy and safety of CARVYKTI™ (ciltacabtagene autoleucel; cilta-cel), a B-cell maturation antigen (BCMA)-directed chimeric antigen receptor T-cell (CAR-T) therapy. The study included patients with relapsed or refractory multiple myeloma (RRMM) who had received >3 lines of therapy including a proteasome inhibitor (PI), an anti-CD38 monoclonal antibody and an immunomodulatory agent (IMiD) or were double refractory to an IMiD and PI and who had received a PI, an IMiD and an anti-CD38 as part of previous therapy. A median overall survival (OS) of 9.3 months has been reported in refractory patients who were triple-class exposed.1 These data, featured as a poster presentation at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting (Abstract #8028), were simultaneously published in the Journal of Clinical Oncology.
The poster results showed that at a median follow-up of 28 months, in 97 patients treated with CARVYKTI™, the overall response rate (ORR) remained consistent at 98 percent (95 percent Confidence Interval [CI], 92.7 to 99.7), with 83 percent (95 percent CI, 73.4 to 89.4) of patients treated with CARVYKTI™ achieving a stringent complete response (sCR).2 The responses were durable, and median OS and progression free survival (PFS) were not reached. PFS and OS rates at 28 months follow-up were 55 percent (95 percent CI, 44.0 to 64.6) and 70 percent (95 percent CI, 60.1 to 78.6), respectively.2
Sixty-one patients had samples evaluable for minimal residual disease (MRD) status, 92 percent of whom achieved MRD negativity at the 10-5 threshold, which was sustained for ≥6 months in 68 percent (34/50 with sufficient follow-up) and ≥12 months in 55 percent (24/44 with sufficient follow-up).2 In those same patients, two-year PFS rates were 73 percent (95 percent CI, 52.1 to 85.9) and 79 percent (95 percent CI, 51.5 to 91.8), respectively, and two-year OS rates were 94 percent (95 percent CI, 76.1 to 98.3) and 91 percent (95 percent CI, 67.7 to 97.6), respectively.2 Both the two-year PFS and OS rates were favorable compared to the overall study population.2
"The latest results from the CARTITUDE-1 study further reinforce the potential of cilta-cel as an important treatment option for patients with a high unmet need who otherwise face a poor prognosis," said Saad Z. Usmani, M.D., M.B.A., F.A.C.P., Chief of the Myeloma Service at Memorial Sloan Kettering Cancer Center and study investigator.‡ "The response rates observed in the two-year follow-up, with median PFS not reached and the majority of patients achieving MRD negativity, demonstrate that cilta-cel provides the potential for long-term disease control and survival in heavily pretreated patients with relapsed or refractory multiple myeloma."
The study included patients (n=97) who received a median of six prior treatment regimens (range, 3-18).2 All patients were triple-class [IMiD, PI and anti-CD38 antibody] exposed, while 42 percent of patients were penta-drug refractory and 99 percent of patients were refractory to the last line of therapy.2
No new safety signals were observed with longer follow-up.2 The most common hematologic adverse events (AEs) observed were neutropenia (96 percent); anemia (81 percent); thrombocytopenia (79 percent); leukopenia (62 percent); and lymphopenia (54 percent).3 Since the primary 12-month publication, no new events or changes in incidence rate, time to onset, or duration of cytokine release syndrome (CRS) occurred, and one new case of treatment-related Parkinsonism, or movement and neurocognitive treatment (MNT) emergent adverse event occurred.
CARVYKTI™ Results in Earlier Lines of Treatment
Findings from Cohort B (n=19) of the Phase 2 CARTITUDE-2 (NCT04133636) study, evaluating the safety and efficacy of cilta-cel in patients with RRMM who received one prior line of therapy including a PI and IMiD and had disease progression within 12 months of treatment with autologous stem cell transplant (ASCT) or within 12 months of the start of antimyeloma therapy for patients who have not had ASCT, showed patients treated with cilta-cel experienced early and deep responses at a median follow-up of 13-months.4 In 19 patients treated in this cohort, the ORR was 100 percent (95 percent CI, 82.4 to 100), with 90 percent (95 percent CI, 66.9 to 98.7) of patients achieving a CR or better and 95 percent (95 percent CI, 74.0 to 99.9) of patients achieving a very good partial response (VGPR) or better.4 Median time to first response was 1 month (range, 0.9-9.7).4 The 12-month PFS rate was 90 percent.4 The overall safety profile, including incidence of CRS and most common hematologic AEs, was consistent with observations in the CARTITUDE-1 study.4 These data were presented for the first time at ASCO (Abstract #8029) and will be featured as an oral presentation at the European Hematology Association (EHA) 2022 Congress (Abstract #S185).4,5
Updated results from Cohort A (n=20) of the CARTITUDE-2 study evaluating cilta–cel safety and efficacy in multiple myeloma patients who are lenalidomide refractory with 1–3 prior lines of treatment were also presented as a poster presentation at ASCO 2022 (Abstract #8020).6
"We are pleased to see the clinical benefit of CARVYKTI as demonstrated in these results from CARTITUDE-1 that show deep and durable responses were maintained over time," said Sen Zhuang M.D., Ph.D., Vice President, Clinical Research and Development, Janssen Research & Development, LLC. "As part of our dedication to advance the science of multiple myeloma, we remain committed to further investigating the potential of CARVYKTI in earlier lines of treatment, including in the CARTITUDE-2 study as part of the CARVYKTI clinical development program."
CARVYKTI™ received approval by the U.S. FDA in February 2022 for the treatment of adults with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody. CARVYKTI™ is not currently approved in any other treatment setting.
On May 25, 2022, CARVYKTI™ was granted conditional marketing authorization by the European Medicines Agency (EMA) for the treatment of adults with RRMM who have received at least three prior therapies, including a PI, an IMiD and an anti-CD38 antibody, and have demonstrated disease progression on the last therapy.
About CARVYKTI™ (ciltacabtagene autoleucel)
CARVYKTI™ is a BCMA-directed, genetically modified autologous T-cell immunotherapy, which involves reprogramming a patient's own T cells with a transgene encoding a chimeric antigen receptor (CAR) that identifies and eliminates cells that express BCMA. BCMA is primarily expressed on the surface of malignant multiple myeloma B-lineage cells, as well as late-stage B cells and plasma cells. The CARVYKTI™ CAR protein features two BCMA-targeting single domain antibodies designed to confer high avidity against human BCMA. Upon binding to BCMA-expressing cells, the CAR promotes T-cell activation, expansion, and elimination of target cells.7
In December 2017, Janssen Biotech, Inc. entered into an exclusive worldwide license and collaboration agreement with Legend Biotech USA, Inc. to develop and commercialize CARVYKTI™.
For more information, visit www.CARVYKTI.com.
About the CARTITUDE-1 Study
CARTITUDE-1 (NCT03548207) is an ongoing Phase 1b/2, open-label, multi-center study evaluating ciltacabtagene autoleucel for the treatment of patients with relapsed or refractory multiple myeloma, who previously received a proteasome inhibitor (PI), an immunomodulatory agent (IMiD) and an anti-CD38 monoclonal antibody, and who had disease progression on or after the last regimen. Patients in the study had received a median of six prior treatment regimens (range, 3-18). Of the 97 patients enrolled in the trial, 99 percent were refractory to the last line of treatment and 88 percent were triple-class refractory, meaning their cancer did not respond to, or had progressed on, an IMiD, a PI and an anti-CD38 monoclonal antibody.2
About the CARTITUDE-2 Study
CARTITUDE-2 (NCT04133636) is an ongoing, multi-cohort Phase 2 study evaluating the safety and efficacy of cilta-cel in patients with multiple myeloma. Cohort B evaluates patients who received one line of prior therapy including a PI and an IMiD, and had disease progression per IMWG criteria within 12 months after treatment with autologous stem cell transplantation (ASCT) or from the start of anti-myeloma therapy for participants who have not had an ASCT. Cohort A evaluates patients who had progressive multiple myeloma after 1–3 prior lines of therapy including a PI and an IMiD, were lenalidomide refractory, and had no prior exposure to BCMA-targeting agents.
CARVYKTI™ Important Safety Information
INDICATIONS AND USAGE
CARVYKTI™ (ciltacabtagene autoleucel) is a B-cell maturation antigen (BCMA)-directed genetically modified autologous T cell immunotherapy indicated for the treatment of adult patients with relapsed or refractory multiple myeloma, after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.
Further information is available at www.CARVYKTIrems.com or 1-844-672-0067.
Please read full Prescribing Information including Boxed Warning for CARVYKTI™.
Learn more at www.janssen.com. Follow us at@JanssenGlobal and @JanssenUS. Janssen Research & Development, LLC, Janssen Biotech, Inc. and Janssen Pharmaceutica NV are part of the Janssen Pharmaceutical Companies of Johnson & Johnson.
J&J's Carvykti demonstrates durable response in heavily pretreated multiple myeloma
Jun. 06, 2022 11:27 AM ET
By: Jonathan Block, SA News Editor
- Johnson & Johnson's (NYSE:JNJ) Janssen division said that heavily pretreated patients with multiple myeloma treated with Carvykti (ciltacabtagene autoleucel; cilta-cel) achieved a durable response a me
- https://seekingalpha.com/news/3845973-johnson-johnson-carvykti-demonstrates-durable-response-in-heavily-pretreated-multiple-myelomadian of 28 months follow-up.
- https://seekingalpha.com/symbol/JNJ
- https://www.carvykti.com/
Tafinlar® (dabrafenib) + Mekinist® (trametinib)
Novartis Tafinlar® (dabrafenib) + Mekinist® (trametinib) demonstrates unprecedented efficacy in pediatric patients with BRAF V600 low-grade gliomas in Phase II/III study
Jun 06, 2022
- Treatment with targeted therapies Tafinlar + Mekinist resulted in 47% ORR versus chemotherapy (11%) and reduced risk of progression or death by 69%, showing significant efficacy improvement in patients ages 1 to 17 years old with BRAF V600 low-grade gliomas (LGG) requiring first systemic treatment1
- If approved, Tafinlar + Mekinist may offer a potential new standard-of-care for pediatric patients with this brain cancer who are in need of a more efficacious and convenient option that can be administered orally and may also improve quality of life1
- Results add to body of evidence demonstrating efficacy and safety of Tafinlar + Mekinist across multiple BRAF V600 solid tumors
- Data highlighted as part of ASCO official press briefing and will form basis of upcoming regulatory submissions
Basel, June 6, 2022 — Novartis today announced Tafinlar® (dabrafenib) + Mekinist® (trametinib) significantly improved efficacy in patients ages 1 to 17 years old with BRAF V600 pediatric low-grade glioma (pLGG) requiring first systemic treatment compared to chemotherapy, the current standard-of-care for these patients1. In this study, patients randomized to receive Tafinlar + Mekinist experienced a statistically significant overall response rate (ORR) of 47% (CI: 35-59%) compared to 11% (CI: 3-25%, p<0.001) for those randomized to receive chemotherapy. A new liquid formulation of Tafinlar + Mekinist that can be easier to administer than chemotherapy was used in this trial. The data will be highlighted today as part of an official press briefing and oral presentation at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting (Abstract #LBA2002).
“These results show dabrafenib and trametinib demonstrate an improvement over chemotherapy for children and adolescents with BRAF V600 low-grade gliomas,” said Eric Bouffet, MD, FRCPC, Senior Associate Scientist Emeritus at The Hospital for Sick Children (SickKids) in Toronto, Canada. “This work highlights the importance of testing for mutations like BRAF in patients with low-grade gliomas.”
LGG is the most common pediatric brain cancer and BRAF V600 mutations are present in 15-20% of pLGGs2,5. Currently, standard chemotherapy is associated with poor outcomes and a high burden of care6.
Additional results from the Phase II/III trial showed at a median follow-up of 18.9 months, median progression-free survival (PFS) was 20.1 months with Tafinlar + Mekinist (CI: 12.8 months-not estimable) compared to 7.4 months with chemotherapy (CI: 3.6-11.8 months, hazard ratio=0.31 [CI: 0.17-0.55] [p<0.001])1. Additionally, tumors shrank or remained stable in 86% of patients in the Tafinlar + Mekinist arm (n=73; CI: 76-93%) compared to 46% of patients in the chemotherapy arm (n=37; CI: 30-63%). After one year of follow-up, nearly all patients on Tafinlar + Mekinist (89%) had a reduction in tumor size compared with baseline versus 70% of patients in the chemotherapy arm. Results from a quality-of-life analysis favored Tafinlar + Mekinist compared to chemotherapy at all time points1.
“These young patients and their families experience a heavy burden of care as BRAF V600 low-grade glioma poses a risk of neurological impairment and current standard-of-care treatment is intravenous and associated with frequent trips to the cancer clinic or hospital,” said Jeff Legos, Executive Vice President, Global Head of Oncology & Hematology Development at Novartis. “Tafinlar + Mekinist has shown unprecedented efficacy, and we will work with health authorities to bring these children the possibility of a more effective and easier to administer liquid oral treatment option as quickly as possible.”
The safety profile of Tafinlar + Mekinist was generally consistent with established safety observed in previous studies. Patients in the Tafinlar + Mekinist arm had fewer grade 3 or higher adverse events (AEs; 47% vs 94%) and fewer discontinuations due to AEs (4% vs 18%) than patients in the chemotherapy arm evaluated for safety (n=33)1. The most frequent AEs in the Tafinlar + Mekinist arm were pyrexia, headache and vomiting1.
In a separate single-arm cohort of this study evaluating pediatric patients with relapsed or refractory BRAF V600 high-grade gliomas (HGG), treatment with Tafinlar + Mekinist showed an independently assessed ORR of 56.1% [CI: 39.7%-71.5%] and generally consistent safety results4. These data were presented in a poster discussion at the 2022 ASCO Annual Meeting (Abstract #2009).
Tafinlar + Mekinist was granted Breakthrough Therapy designation by the US Food and Drug Administration for the treatment of pediatric patients one year of age and older with LGG with a BRAF V600E mutation who require systemic therapy.
About the TADPOLE trial
This global Phase II/III, multicenter, open-label trial is evaluating patients aged 1 to 17 with BRAF V600 LGG (n=110) or relapsed or refractory HGG (n=41)1. Tafinlar + Mekinist is administered orally either as new liquid formulations, or as capsules and tablets, respectively. The primary endpoint in both cohorts is overall response rate. Secondary outcome measures include duration of response, progression-free survival, overall survival and other endpoints1.
About Tafinlar + Mekinist
The combination of Tafinlar + Mekinist, the worldwide targeted therapy leader in BRAF/MEK-inhibition research and patients reached, may help to slow tumor growth by blocking signals associated with the BRAF and MEK kinases, which are implicated in the growth of various types of cancer11-15. Tafinlar + Mekinist has been studied in more than 6,000 BRAF-positive patients in more than 20 ongoing and completed trials and has been prescribed to more than 200,000 patients worldwide15.
Tafinlar + Mekinist is currently not approved for pediatric patients with LGG or HGG. These results will form the basis of regulatory discussions with global health authorities.
https://www.us.tafinlarmekinist.com/
About Novartis
Novartis is reimagining medicine to improve and extend people’s lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world’s top companies investing in research and development. Novartis products reach nearly 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 108,000 people of more than 140 nationalities work at Novartis around the world. Find out more at https://www.novartis.com.
Novartis says drug combination outperformed chemo in children with brain cancer
Jun. 06, 2022 11:30 AM ET
By: Dulan Lokuwithana, SA News Editor
Disclosing data from a global Phase 2/3 trial, Novartis (NYSE:NVS) (OTCPK:NVSEF) announced on Monday that its drug combination, Tafinlar and Mekinist, reduced the risk of disease progression or death for a type of childhood brain cancer by more than two thirds compared to chemotherapy.
https://seekingalpha.com/symbol/NVS
https://www.us.tafinlarmekinist.com/
YESCARTA® (axicabtagene ciloleucel)
June 03, 2022
Yescarta® CAR T-cell Therapy Demonstrates Consistent Survival Outcomes and Safety in Real-World Setting Regardless of Race and Ethnicity
-- Largest Analysis Examining Use of CAR T-cell Therapy Across Race and Ethnicity --
-- Black or African American Patients Experienced Longer Time from Diagnosis to Infusion Compared to White Patients, Possibly Impacting Overall and Complete Response Rates; More Study Warranted --
-- Clinical Trials of Yescarta in the U.S. and Real-World Use from CIBMTR Registry Show Consistent Participation of Approximately 5% Black or African American Patients --
SANTA MONICA, Calif.--(BUSINESS WIRE)-- Kite, a Gilead Company (Nasdaq: GILD), today announced findings from a safety and efficacy retrospective analysis by race and ethnicity from the ongoing post-authorization study of Yescarta® (axicabtagene ciloleucel) in adult patients with relapsed or refractory large B-cell lymphoma (LBCL). In the largest real-world analysis of its kind evaluating data from the CIBMTR® (Center for International Blood and Marrow Transplant Research®), overall outcomes including overall survival (OS) and progression-free survival (PFS) rates were consistent with Yescarta in the real-world setting, regardless of race and ethnicity. The findings were presented today in an oral session during the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting (Abstract #7571).
The incidence of diffuse LBCL in the U.S. is 4.8 per 100,000 per year in non-Hispanic Black or African Americans and 7.1 per 100,000 per year in non-Hispanic Whites. Clinical trials of Yescarta in the U.S. have enrolled an average of 6% Black or African American patients, consistent with the roughly 5% of patients in the real-world CIBMTR registry.* Further research is ongoing to investigate whether or not there is under-representation by race and ethnicity in both clinical trials and the real-world usage of CAR T-cell therapy.
“The investigation of CAR T-cell therapy outcomes by race and ethnicity is important to the continued understanding of the impact of these innovative therapies, and an area in which there is a significant deficit in clinical trials and real-world studies published to date,” said Frederick L. Locke, MD, lead author and Co-Leader of the Immuno-Oncology Program at Moffitt Cancer Center, Tampa, Florida. “The results of this analysis are encouraging in that axi-cel was safe and effective regardless of race or ethnicity, and also warrant further investigation to understand the lower rate of response among Black or African American patients and the potential role of factors such as higher disease burden, disease biology and, importantly, differential access to care.”
A total of 1,389 adult patients with LBCL treated with Yescarta in the commercial setting in the U.S. from October 2017 to August 2020 were included in the analysis. Race and ethnicity (Hispanic or Latino vs. not Hispanic or Latino) were self-reported and included: White (81%); Black or African American (5%); Asian (6%); American Indian or Alaska Native <1%; Native Hawaiian or other Pacific Islander <1%; More than one race <1%; Race not reported (7%). Eleven percent of patients evaluated self-identified as Hispanic or Latino.
About Yescarta
Please see full Prescribing Information, including BOXED WARNING and Medication Guide.
YESCARTA is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of:
- Adult patients with large B-cell lymphoma that is refractory to first-line chemoimmunotherapy or that relapses within 12 months of first-line chemoimmunotherapy.
- Adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma, high-grade B-cell lymphoma, and DLBCL arising from follicular lymphoma.
Limitations of Use: YESCARTA is not indicated for the treatment of patients with primary central nervous system lymphoma. - Adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy. This indication is approved under accelerated approval based on the response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trial(s).
- https://www.yescarta.com/
About Kite
Kite, a Gilead Company, is a global biopharmaceutical company based in Santa Monica, California, with manufacturing operations in North America and Europe. Kite’s singular focus is cell therapy to treat and potentially cure cancer. As the cell therapy leader, Kite has more approved CAR T indications to help more patients than any other company. For more information on Kite, please visit www.kitepharma.com.
About Gilead Sciences
Gilead Sciences, Inc. is a biopharmaceutical company that has pursued and achieved breakthroughs in medicine for more than three decades, with the goal of creating a healthier world for all people. The company is committed to advancing innovative medicines to prevent and treat life-threatening diseases, including HIV, viral hepatitis, and cancer. Gilead operates in more than 35 countries worldwide, with headquarters in Foster City, California.
U.S. Prescribing Information for Yescarta including BOXED WARNING, is available at www.kitepharma.com and www.gilead.com.
Kite, the Kite logo, Yescarta, Tecartus, XLP and GILEAD are trademarks of Gilead Sciences, Inc. or its related companies.
For more information on Kite, please visit the company’s website at www.kitepharma.com. Follow Kite on social media on Twitter (@KitePharma) and LinkedIn.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220603005447/en/
Source: Gilead Sciences, Inc.
Gilead's Yescarta shows consistent real-world survival outcomes regardless of race
Jun. 03, 2022 4:24 PM ET
By: Anuron Mitra, SA News Editor
Gilead Sciences' (NASDAQ:GILD) Kite on Friday announced findings from an analysis of race and ethnicity from its ongoing post-authorization study of Yescarta in adults with large B-cell lymphoma (LBCL), a type of cancer in white blood cells called lymphocytes.
https://seekingalpha.com/symbol/GILD
https://www.gilead.com/-/media/files/pdfs/medicines/oncology/yescarta/yescarta-pi.pdf
June 04, 2022
Sub-analyses of Landmark ZUMA-7 Trial Reinforce Yescarta® CAR T-cell Therapy Superiority Over Standard of Care (SOC) as Initial Treatment for Patients With Relapsed or Refractory Large B-cell Lymphoma (LBCL)
-- Patients Aged 65+ Had Over Three-fold Improvement in Two-year Event-Free Survival (EFS) Rate, and Over Eight-fold Greater Median EFS and Clinically Meaningful Improvements in Quality of Life (QoL) with Yescarta vs SOC --
-- Separate Exploratory Analysis Found Yescarta Benefit vs SOC Was Consistent Across Subgroups, Including in Patients with High Tumor Burden and Elevated Lactate Dehydrogenase (LDH) --
SANTA MONICA, Calif.--(BUSINESS WIRE)-- Kite, a Gilead Company (Nasdaq: GILD),
Teclistamab
Updated Data for Janssen’s Bispecific Teclistamab Suggest Continued Deep and Durable Responses in the Treatment of Patients with Relapsed or Refractory Multiple Myeloma
New teclistamab data presented at the 2022 ASCO Annual Meeting report longer follow-up from Phase 1/2 MajesTEC-1 study evaluating the BCMAxCD3 bispecific antibody, including progression-free survival and subgroup analyses
Data from MajesTEC-1 study published in The New England Journal of Medicine
CHICAGO, ILLINOIS, June 5, 2022 – The Janssen Pharmaceutical Companies of Johnson & Johnson announced updated efficacy and safety results from the teclistamab Phase 1/2 MajesTEC-1 study. Teclistamab is an investigational, off-the-shelf, T-cell redirecting bispecific antibody targeting B-cell maturation antigen (BCMA), which is being studied in patients with relapsed or refractory multiple myeloma (RRMM) who have received three or more prior lines of therapy.1 The data were featured as part of an oral session during the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting. Additional poster presentations featured data on teclistamab as a monotherapy, as well as in combination with DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj). Applications seeking approval of teclistamab are currently under health authority review in the U.S. and Europe.
The multicohort, open-label, Phase 1/2 MajesTEC-1 study is investigating the safety and efficacy of teclistamab in patients with RRMM who received at least three prior lines of therapy. As of March 2022, 165 patients were treated with teclistamab at the recommended subcutaneous (SC) Phase 2 dose (RP2D) of 1.5 mg/kg preceded by step-up doses of 0.06 and 0.3 mg/kg across both Phase 1 (NCT03145181) and Phase 2 (NCT04557098) of the study.
Longer Follow-up from MajesTEC-1 Study in Patients with Triple Class Exposed Multiple Myeloma (Abstract #8007)
At a median follow-up of 14.1 months (0.26–24.4), an overall response rate (ORR) of 63 percent (95 percent Confidence Interval [CI], range, 55.2–70.4) was observed in patients with triple class exposed multiple myeloma, with a complete response (CR) or better achieved in 39.4 percent of patients.1 Study participants had three or more prior lines of therapy, with a median of five prior lines, including a prior proteasome inhibitor, immunomodulatory drug and anti-CD38 antibody.1 The majority of patients were triple-class refractory and/or refractory to their last line of treatment.1 Although response duration data are not mature, the median duration of response at this time is 18.4 months and has not been reached in patients who achieved a CR or better (95 percent CI, 14.9 not estimable).1 This suggests responses to teclistamab were durable and deepened over time.1 The medium progression-free survival (PFS) was 11.3 months (95 percent CI, 8.8–17.1).1 Adverse events (AEs) were low-grade for the most part and manageable with no new safety signals seen.1
These results from the MajesTEC-1 study were also simultaneously published online in The New England Journal of Medicine.2
“The MajesTEC-1 study update suggests patients with relapsed or refractory multiple myeloma receiving teclistamab achieved a deep response that was also durable,” said Ajay K. Nooka, M.D., MPH, FACP, Associate Professor of Hematology and Medical Oncology at Emory School of Medicine and principal study investigator.‡ “These longer-term data, notably the overall response rate and progression-free survival, are encouraging in this heavily pretreated patient population.”
No new safety signals were observed with longer follow-up.1 In 14.1 month follow-up data presented at ASCO 2022, the most common grade 3/4 hematologic AEs were neutropenia (64.2 percent); anemia (37 percent); lymphopenia (32.7 percent) and thrombocytopenia (21.2 percent). Infections occurred in 76.4 percent of patients (44.8 percent grade 3/4).1 The most common nonhematologic AE was cytokine release syndrome (CRS), all of which were grade 1/2 except for 1 transient grade 3 CRS (72.1 percent all grade).1 The median time to CRS onset was two days (range, 1–6) and median duration was two days (range, 1–9).1 There were five treatment-related deaths, and dose reductions and discontinuations due to AEs were infrequent.1
First Results from Cohort C of the MajesTEC-1 Study of Teclistamab in Patients with RRMM with Prior Exposure to BCMA Targeted Treatment (Abstract #8013)
Initial results were also presented from Cohort C of the MajesTEC-1 study evaluating teclistamab in the treatment of patients with RRMM who had previously been exposed to an anti-BCMA treatment.3 These patients had received a median of six prior lines of therapy, most (85 percent) were triple-class refractory and 35 percent were penta-drug refractory.3 The use of teclistamab following prior treatment with chimeric antigen receptor T cell (CAR-T) therapy and/or an antibody drug conjugate (ADC) (e.g., belantamab mafodotin) targeting BCMA resulted in a promising response rate in patients with heavily pretreated RRMM.3 At a median follow-up of 12.5 months (0.7-14.4), the ORR was 52.5 percent (95 percent CI, 36.1–68.5) among 40 patients who received teclistamab in Cohort C.3 Responses to teclistamab occurred early and deepened over time, with comparable response rates in patients previously treated with an ADC and/or CAR-T.3
A tolerable side-effect profile was observed in patients previously treated with anti-BCMA treatment, with no dose reductions or discontinuations due to AEs.3 The safety profile for Cohort C was comparable with that observed in BCMA treatment-naive patients, with no new safety signals.3 In 12.5 month follow-up data, 26 patients (65 percent; 30 percent grade 3/4) had infections.3 The most common AEs (n=40) were CRS (65 percent any grade), with a median time to CRS onset and duration of two days (range, 2-6) and two days (range, 1-4) respectively.3 Cytopenias (grade 3/4) were noted as follows; neutropenia (62.5 percent); thrombocytopenia (30 percent); anemia (35 percent); and lymphopenia (42.5 percent).3
Initial Patient-Reported Health-Related Quality of Life (HRQoL) Outcomes in Patients with RRMM Treated with Teclistamab (Abstract #8033)
Initial results from an analysis of patient-reported health-related quality of life (HRQoL) outcomes following treatment with teclistamab were also shared in a poster session.4 The study analyzed patient-reported assessments of quality of life metrics among patients in the MajesTEC-1 trial who had received their first treatment dose by March 18, 2021.4 The metrics analyzed include function (physical, role, emotional, cognitive, social); symptoms (fatigue, nausea/vomiting, pain, appetite loss, constipation, diarrhea); and generic health (mobility, self-care, usual activities, pain/discomfort, anxiety/depression).4 Over 80 percent of the 110 patients included in the patient-reported outcomes (PRO) analysis noted meaningful improvement (percentages of patients with clinically meaningful change from baseline (EORTC QLQ-C30 scales: ≥10 points)) in at least one of the symptom scales.4 Reduction in pain scores occurred as early as cycle two.4 At the moment, no meaningful improvement was observed in the scales for physical functioning and fatigue.4 These initial PRO results complement recent clinical data and support teclistamab as a potential off-the-shelf, T-cell redirecting therapy for patients with RRMM.4
As of September 7, 2021, median duration of treatment was 5.7 months and median follow-up was 7.8 months.4 Global health status scores significantly improved from baseline (95 percent CIs for least squares mean change did not cross 0) at cycles four, six, and eight; emotional functioning significantly improved at all time points.4 PRO assessments included European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 item (EORTC QLQ-C30).4 PROs were assessed on day one of each treatment cycle (28 days per cycle).4 Additional follow-up is needed to assess the full benefit of meaningful improvement in functional outcomes.4
About Teclistamab
Teclistamab is an investigational, fully humanized IgG4, T-cell redirecting, bispecific antibody targeting both BCMA (B-cell maturation antigen) and CD3, the T-cell receptor. BCMA is expressed at high levels on multiple myeloma cells.9,10,11,12,13 Teclistamab redirects CD3-positive T-cells to BCMA-expressing myeloma cells to induce killing of tumor cells.8
Teclistamab is currently being evaluated in several monotherapy and combination studies. In 2020, the European Commission and the U.S. Food and Drug Administration (FDA) each granted teclistamab Orphan Drug Designation for the treatment of multiple myeloma. In January 2021 and June 2021, teclistamab received a PRIority MEdicines (PRIME) designation by the European Medicines Agency (EMA) and Breakthrough Therapy Designation (BTD) by the U.S. FDA, respectively. PRIME offers enhanced interaction and early dialogue to optimize drug development plans and speed up evaluation of cutting-edge, scientific advances that target a high unmet medical need.14 The U.S. FDA grants BTD to expedite the development and regulatory review of an investigational medicine that is intended to treat a serious or life-threatening condition and is based on preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy.15 In December 2021, Janssen submitted a Biologics License Application (BLA) to the FDA seeking approval of teclistamab for the treatment of patients with relapsed or refractory multiple myeloma; a marketing authorization application (MAA) was submitted to the EMA for teclistamab approval in January 2022.
Learn more at www.janssen.com. Follow us at www.twitter.com/JanssenGlobal. Janssen Research & Development, LLC is one of the Janssen Pharmaceutical Companies of Johnson & Johnson.
JNJ Dividend History
https://www.nasdaq.com/market-activity/stocks/jnj/dividend-history
https://seekingalpha.com/symbol/JNJ
ENHERTU® (fam-trastuzumab deruxtecan-nxki) for injection
Enhertu reduced the risk of disease progression or death by 50% vs. chemotherapy in patients with HER2-low metastatic breast cancer with HR-positive and HR-negative disease
PUBLISHED5 June 2022
5 June 2022 13:00 BST
AstraZeneca and Daiichi Sankyo’s Enhertu also improved median overall survival by more than 6 months vs. chemotherapy in all patients evaluated in DESTINY-Breast04
Enhertu met the primary endpoint of progression-free survival in patients with HR-positive disease, reducing the risk of disease progression or death by 49% vs. chemotherapy
Enhertu is the first HER2-directed therapy to demonstrate a survival benefit in this population, potentially redefining treatment for approximately half of all patients with breast cancer
Detailed positive results from the pivotal DESTINY-Breast04 Phase III trial showed that Enhertu (trastuzumab deruxtecan) demonstrated superior and clinically meaningful progression-free survival (PFS) and overall survival (OS) in previously treated patients with HER2-low (immunohistochemistry (IHC) 1+ or IHC 2+/in-situ hybridisation (ISH)-negative) unresectable and/or metastatic breast cancer with hormone receptor (HR) positive or HR-negative disease versus standard of care physician’s choice of chemotherapy. Results will be presented during the Plenary Session today at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting, and have been simultaneously published in The New England Journal of Medicine.
Enhertu is a specifically engineered HER2-directed antibody drug conjugate (ADC) being jointly developed and commercialised by AstraZeneca and Daiichi Sankyo.
In the primary endpoint analysis for DESTINY-Breast04, Enhertu demonstrated a 49% reduction in the risk of disease progression or death versus physician’s choice of chemotherapy in patients with HER2-low metastatic breast cancer with HR-positive disease (PFS hazard ratio [HR] 0.51; 95% confidence interval [CI]: 0.40-0.64; p<0.001). A median PFS of 10.1 months was seen in patients treated with Enhertu compared to 5.4 months with chemotherapy, as assessed by blinded independent central review (BICR).
Results also showed a 36% reduction in the risk of death with Enhertu compared to chemotherapy in patients with HR-positive disease (OS HR 0.64; 95% CI: 0.48-0.86; p=0.003) with a median OS of 23.9 months with Enhertu versus 17.5 months with chemotherapy, meeting a key secondary endpoint of the trial.
Additionally, data showed consistent efficacy for Enhertu in the overall trial population of patients with HER2-low metastatic breast cancer with HR-positive or HR-negative disease and across levels of HER2 expression (IHC 1+ and IHC 2+/ISH-). In the key secondary endpoint analysis of PFS by BICR in all patients, a similar 50% reduction in the risk of disease progression or death was observed between Enhertu and chemotherapy (PFS HR 0.50; 95% CI: 0.40-0.63; p<0.001). Results also showed a 36% reduction in the risk of death with Enhertu compared to chemotherapy (OS HR 0.64; 95% CI: 0.49-0.84; p=0.001) with a median OS of 23.4 months for Enhertu versus 16.8 months with chemotherapy.
Shanu Modi, MD, Medical Oncologist, Memorial Sloan Kettering Cancer Center, US and Principal Investigator for the trial, said: “The results of DESTINY-Breast04 show for the first time that a HER2-directed therapy can provide a survival benefit to patients with low HER2 expression, indicating we must reconsider the way we categorise patients with metastatic breast cancer. The efficacy seen with Enhertu also reinforces the potential to establish a new standard of care for more than half of all patients with breast cancer currently categorised as having HER2-negative disease, but who actually have tumours with low HER2 expression.”
Susan Galbraith, Executive Vice President, Oncology R&D, AstraZeneca said: “Today’s results represent a pivotal moment demonstrating the potential for Enhertu to redefine the treatment of HER2-targetable cancers. DESTINY-Breast04 validates targeting the lower end of the spectrum of HER2 expression, since Enhertu reduced the risk of disease progression or death across all types of patients in the trial by half, and reduced the risk of death by over a third. We must now evolve the way we classify and treat metastatic breast cancer to ensure these patients are effectively diagnosed and treated.”
Ken Takeshita, Global Head, R&D, Daiichi Sankyo said: “As innovative research organisations, extending the survival for patients is one of our primary goals as we seek to identify potentially new treatment options for patients with metastatic breast cancer. These potentially practice-changing data show that DESTINY-Breast04 takes us one step closer to achieving this goal, as Enhertu is the first HER2-directed medicine to demonstrate a survival benefit in patients with HER2-low metastatic breast cancer. We are honoured by the recognition these important findings are receiving at one of the world’s most prominent oncology meetings as well as in one of the leading medical journals.”
DESTINY-Breast04
DESTINY-Breast04 is a global, randomised, open-label, registrational Phase III trial evaluating the efficacy and safety of Enhertu (5.4mg/kg) versus physician’s choice of chemotherapy (capecitabine, eribulin, gemcitabine, paclitaxel or nab-paclitaxel) in patients with HR-positive or HR-negative HER2-low unresectable and/or metastatic breast cancer previously treated with one or two prior lines of chemotherapy. Patients were randomised 2:1 to receive either Enhertu or chemotherapy.
The primary endpoint of DESTINY-Breast04 is PFS in patients with HR-positive disease based on blinded independent central review (BICR). Key secondary endpoints include PFS based on BICR in all randomised patients (HR-positive and HR-negative disease), OS in patients with HR-positive disease and OS in all randomised patients (HR-positive and HR-negative disease). Other secondary endpoints include PFS based on investigator assessment, objective response rate based on BICR and on investigator assessment, duration of response based on BICR and safety.
DESTINY-Breast04 enrolled approximately 557 patients at multiple sites in Asia, Europe and North America. For more information about the trial, visit ClinicalTrials.gov.
Enhertu
Enhertu is a HER2-directed ADC. Designed using Daiichi Sankyo’s proprietary DXd ADC technology, Enhertu is the lead ADC in the oncology portfolio of Daiichi Sankyo and the most advanced programme in AstraZeneca’s ADC scientific platform. Enhertu consists of a HER2 monoclonal antibody attached to a topoisomerase I inhibitor payload, an exatecan derivative, via a stable tetrapeptide-based cleavable linker.
Enhertu (5.4mg/kg) is approved in the US and Israel for the treatment of adult patients with unresectable or metastatic HER2-positive breast cancer who have received a prior anti-HER2-based regimen either in the metastatic setting, or in the neoadjuvant or adjuvant setting and have developed disease recurrence during or within six months of completing therapy, based on results from the DESTINY-Breast03 trial.
Enhertu (5.4mg/kg) is also approved in approximately 40 countries for the treatment of adult patients with unresectable or metastatic HER2-positive breast cancer who have received two or more prior anti-HER2-based regimens based on the results from the DESTINY-Breast01 trial.
Enhertu (6.4mg/kg) is approved in several countries for the treatment of adult patients with locally advanced or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma who have received a prior trastuzumab-based regimen based on the results from the DESTINY-Gastric01 trial.
https://www.enhertu.com/en/breast
Enhertu development programme
A comprehensive development programme is underway globally, evaluating the efficacy and safety of Enhertu monotherapy across multiple HER2-targetable cancers, including breast, gastric, lung and colorectal cancers. Trials in combination with other anticancer treatments, such as immunotherapy, are also underway.
Regulatory applications for Enhertu are currently under review in China, Europe, Japan and several other countries for the treatment of adult patients with unresectable or metastatic HER2-positive breast cancer who have received a prior anti-HER2 based regimen based on the results from the DESTINY-Breast03 trial.
Enhertu was granted Breakthrough Therapy Designation in the US for the treatment of adult patients with unresectable or metastatic HER2-low (IHC 1+ or IHC 2+/ISH-negative) breast cancer who have received a prior systemic therapy in the metastatic setting or developed disease recurrence during or within six months of completing adjuvant chemotherapy, based on the results of the DESTINY-Breast04 trial. Patients with hormone receptor (HR) positive breast cancer should additionally have received or be ineligible for endocrine therapy.
Enhertu is also currently under review in the US for the treatment of adult patients with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumours have a HER2 (ERBB2) mutation and who have received a prior systemic therapy, based on the DESTINY-Lung01 trial, and in Europe for the treatment of adult patients with locally advanced or metastatic HER2-positive gastric or GEJ adenocarcinoma who have received a prior anti-HER2-based regimen based on the DESTINY-Gastric01 and DESTINY-Gastric02 trials.
Daiichi Sankyo collaboration
Daiichi Sankyo Company, Limited (TSE: 4568) [referred to as Daiichi Sankyo] and AstraZeneca entered into a global collaboration to jointly develop and commercialise Enhertu (a HER2-directed ADC) in March 2019, and datopotamab deruxtecan (DS-1062; a TROP2-directed ADC) in July 2020, except in Japan where Daiichi Sankyo maintains exclusive rights. Daiichi Sankyo is responsible for the manufacturing and supply of Enhertu and datopotamab deruxtecan.
AstraZeneca Enhertu data could expand use in breast cancer
Jun. 05, 2022 11:46 AM ETAZN
By: Jonathan Block, SA News Editor
- New data on AstraZeneca's (AZN) Enhertu (trastuzumab) could position the oncologic for expanded use in breast cancer.
- Results from the phase 3 DESTINY-Breast04 study released Sunday showed that patients treated with Enhertu saw a 49% reduction in the risk of disease progression or death versus other chemotherapy in patients with HER2-low metastatic breast cancer with HR-positive disease.
- https://seekingalpha.com/news/3845740-astrazeneca-enhertu-data-could-expand-use-in-breast-cancer
- https://seekingalpha.com/symbol/AZN
- https://www.enhertu.com/en/breast
- https://www.astrazeneca.com/
Trodelvy® (sacituzumab govitecan-hziy)
June 04, 2022
Trodelvy® Improved Progression-Free Survival by 34% in Heavily Pre-Treated HR+/HER2- Metastatic Breast Cancer Patients
– At One Year, 3X as Many Patients Were Progression-Free Compared to Physicians’ Choice of Chemotherapy in Phase 3 TROPiCS-02 Study –
– At The First Interim Analysis, Overall Survival Demonstrated a Positive Trend, Patients Will Continue to be Followed –
– Analysis of Patient-Reported Outcomes Shows Trodelvy Also Improved Quality of Life over Physicians’ Choice of Chemotherapy –
CHICAGO--(BUSINESS WIRE)-- Gilead Sciences, Inc. (Nasdaq: GILD) today announced positive results from the primary analysis of the Phase 3 TROPiCS-02 study of Trodelvy® (sacituzumab govitecan-hziy) versus physicians’ choice of chemotherapy (TPC) in heavily pre-treated HR+/HER2- metastatic breast cancer patients who received prior endocrine therapy, CDK4/6 inhibitors and two to four lines of chemotherapy. The study met its primary endpoint of progression-free survival (PFS) with a statistically significant and clinically meaningful 34% reduction in the risk of disease progression or death (median PFS 5.5 vs. 4 months; HR: 0.66; 95% CI: 0.53-0.83; P<0.0003). The first interim analysis of the key secondary endpoint of overall survival (OS) demonstrated a trend in improvement. These data are immature, and patients will be followed for subsequent OS analysis. These findings will be featured in both a press briefing and an oral session (Abstract #LBA1001) on Saturday, June 4, during the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20220603005437/en/
The study demonstrated that at the one-year mark, three times as many patients were progression-free when treated with Trodelvy compared to those who received TPC (21% versus 7%). Improvements in PFS with Trodelvy were also consistent across key patient subgroups, including patients who had previously received three or more chemotherapy regimens for metastatic disease (HR: 0.70; CI: 0.52-0.95), patients with visceral metastasis (HR: 0.66; CI: 0.53-0.83), and the elderly (≥65 years of age; HR: 0.59; CI: 0.38-0.93).
“For patients with HR+/HER2- metastatic breast cancer, resistance to endocrine therapy is inevitable in almost all cases. The standard of care is then limited to sequential single agent chemotherapy, with declining response rates, disease control and quality of life,” said Dr. Hope Rugo, Professor of Medicine and Director, Breast Oncology and Clinical Trials Education at the University of California San Francisco Comprehensive Cancer Center, U.S. “In TROPiCS-02, we enrolled heavily pre-treated patients with metastatic breast cancer who had disease progression following multiple lines of chemotherapy. To observe a clinically meaningful reduction in the risk of disease progression or death in these patients with limited treatment options is remarkable. Sacituzumab govitecan-hziy will be an important potential future treatment option for these patients.”
A prespecified quality of life (QoL) analysis, one of the secondary endpoints using the EORTC QLQ-C30 instrument, also favored Trodelvy over TPC demonstrating meaningful benefit. In the evaluable population, improvements in global health status and fatigue with Trodelvy (n=234) compared with those who received TPC (n=207) were also observed.
“With Trodelvy, our bold ambition is that it will help transform care for people living with cancer, including in pre-treated HR+/HER2- metastatic breast cancer, where more options are needed,” said Bill Grossman, MD, PhD, Senior Vice President, Therapeutic Area Head, Gilead Oncology. “We look forward to continuing discussions with regulatory agencies to further understand how Trodelvy can impact this patient population with a high unmet need.”
The safety profile for Trodelvy was consistent with prior studies, with no new safety concerns identified in this patient population. The most frequent Grade ≥3 treatment-related adverse reactions for Trodelvy compared to TPC were neutropenia (51% versus 38%), diarrhea (9% versus 1%), leukopenia (9% versus 5%), anemia (6% versus 3%), fatigue (6% versus 2%) and febrile neutropenia (5% versus 4%).
Trodelvy has not been approved by any regulatory agency for the treatment of HR+/HER2- metastatic breast cancer. Its safety and efficacy have not been established for this indication. Trodelvy has a Boxed Warning for severe or life-threatening neutropenia and severe diarrhea; please see below for additional Important Safety Information.
About the TROPiCS-02 Study
The TROPiCS-02 study is a global, multicenter, open-label, Phase 3 study, randomized 1:1 to evaluate Trodelvy versus physicians’ choice of chemotherapy (eribulin, capecitabine, gemcitabine, or vinorelbine) in 543 patients with HR+/HER2- metastatic breast cancer who were previously treated with endocrine therapy, CDK4/6 inhibitors and two to four lines of chemotherapy for metastatic disease. The primary endpoint is progression-free survival per Response Evaluation Criteria in Solid Tumors (RECIST 1.1) as assessed by blinded independent central review (BICR) for participants treated with Trodelvy compared to those treated with chemotherapy. Secondary endpoints include overall survival, overall response rate, clinical benefit rate and duration of response, as well as assessment of safety and tolerability and quality of life measures. In the study, HER2 negativity was defined per American Society of Clinical Oncology (ASCO) and the College of American Pathologists (CAP) criteria as immunohistochemistry (IHC) score of 0, IHC 1+ or IHC 2+ with a negative in-situ hybridization (ISH) test. More information about TROPiCS-02 is available at https://clinicaltrials.gov/ct2/show/NCT03901339.
About Trodelvy
Trodelvy® (sacituzumab govitecan-hziy) is a first-in-class Trop-2 directed antibody-drug conjugate. Trop-2 is a cell surface antigen highly expressed in multiple tumor types, including in more than 90% of breast and bladder cancers. Trodelvy is intentionally designed with a proprietary hydrolyzable linker attached to SN-38, a topoisomerase I inhibitor payload. This unique combination delivers potent activity to both Trop-2 expressing cells and the microenvironment.
Trodelvy is approved in more than 35 countries, with multiple additional regulatory reviews underway worldwide, for the treatment of adult patients with unresectable locally advanced or metastatic triple-negative breast cancer (TNBC) who have received two or more prior systemic therapies, at least one of them for metastatic disease. Trodelvy is also approved in the U.S. under the accelerated approval pathway for the treatment of adult patients with locally advanced or metastatic urothelial cancer (UC) who have previously received a platinum-containing chemotherapy and either programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor.
Trodelvy is also being developed for potential investigational use in other TNBC and metastatic UC populations, as well as a range of tumor types where Trop-2 is highly expressed, including hormone receptor-positive/human epidermal growth factor receptor 2-negative (HR+/HER2-) metastatic breast cancer, metastatic non-small cell lung cancer (NSCLC), metastatic small cell lung cancer (SCLC), head and neck cancer, and endometrial cancer.
U.S. Indication for Trodelvy
In the United States, Trodelvy is indicated for the treatment of:
- Adult patients with unresectable locally advanced or metastatic TNBC who have received two or more prior systemic therapies, at least one of them for metastatic disease.
- Adult patients with locally advanced or metastatic UC who have previously received a platinum-containing chemotherapy and either programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
- https://www.trodelvy.com/
U.S. Prescribing Information for Trodelvy including BOXED WARNING, is available at www.gilead.com.
Trodelvy, Gilead and the Gilead logo are trademarks of Gilead Sciences, Inc., or its related companies.
For more information about Gilead, please visit the company’s website at www.gilead.com, follow Gilead on Twitter (@GileadSciences) or call Gilead Public Affairs at 1-800-GILEAD-5 or 1-650-574-3000.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220603005437/en/
Source: Gilead Sciences, Inc.
Gilead drug cuts disease progression in commonest type of breast cancer
Jun. 04, 2022 11:10 AM ET
By: Dulan Lokuwithana, SA News Editor16 Comments
Announcing data from a Phase 3 study, Gilead (NASDAQ:GILD) said on Saturday that its antibody-drug conjugate Trodelvy delayed the disease progression by 1.5 months in a group of patients with heavily pre-treated HR+/HER2- metastatic breast cancer compared to chemotherapy.
The 543-patient trial was designed to evaluate the effect of Trodelvy in HR+/HER2- metastatic breast cancer, the most common type of breast cancer. The patients were previously treated with endocrine therapy and up to four lines of chemotherapy.
https://seekingalpha.com/symbol/GILD
IMBRUVICA® (ibrutinib)
Phase 3 SHINE Results Show IMBRUVICA® (ibrutinib)-Based Combination Regimen Significantly Reduced the Risk of Disease Progression or Death in Older Patients with Newly Diagnosed Mantle Cell Lymphoma
Jun 03, 2022United States
Primary results from the first frontline Phase 3 study of a Bruton’s tyrosine kinase inhibitor in MCL to be presented as late-breaking data at the 2022 ASCO Annual Meeting and also published in The New England Journal of Medicine
June 3, 2022 (CHICAGO) – The Janssen Pharmaceutical Companies of Johnson & Johnson today announced primary results from the Phase 3 SHINE study (Abstract #7502), which demonstrated that the combination of once-daily oral IMBRUVICA® (ibrutinib) plus bendamustine-rituximab (BR) and rituximab maintenance significantly reduced the risk of disease progression or death by 25 percent compared to patients who received placebo plus BR and rituximab maintenance in patients aged 65 years or older with newly diagnosed mantle cell lymphoma (MCL).[i] This study is one of the largest clinical trials ever conducted in first-line MCL and the first for a Bruton’s tyrosine kinase inhibitor (BTKi).1 The data are being presented in an oral session and featured in a press briefing during the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting, and were published in The New England Journal of Medicine today. The data will also be presented as an oral presentation at the 2022 European Hematology Association (EHA) Annual Congress.
MCL is a type of aggressive, rare non-Hodgkin lymphoma (NHL) that is incurable and difficult to treat.[i] It commonly affects people over the age of 65, who typically cannot tolerate intensive chemoimmunotherapy and stem cell transplantation, resulting in poor clinical outcomes and contributing to the need to develop additional treatment options for these patients.2
“There is an urgent need to improve outcomes for older patients with MCL,” said Michael L. Wang, M.D., Professor, Department of Lymphoma & Myeloma, The University of Texas MD Anderson Cancer Center and principal study investigator.‡ “Given the median progression-free survival of 6.7 years, the ibrutinib combination demonstrated the potential to be a first-line treatment in this population.”
The Phase 3 SHINE (MCL3002) study (NCT01776840) – sponsored by Janssen Biotech, Inc., in collaboration with Pharmacyclics LLC, an AbbVie Company – enrolled 523 patients aged 65 years or older with newly diagnosed MCL.1 All participants were randomly assigned to receive IMBRUVICA® (560 mg orally daily until progression or unacceptable toxicities) or placebo plus BR for a maximum of six 28-day cycles; participants with a complete response (CR) or partial response (PR) continued to receive maintenance therapy with rituximab every second cycle for a maximum of 12 additional doses.1IMBRUVICA® or placebo was administered daily until progressive disease or unacceptable toxicity.1
The SHINE study met its primary endpoint of progression-free survival (PFS). Key findings from the Phase 3 SHINE study include:
- With a median follow-up of 84.7 months, the IMBRUVICA® plus BR and rituximab maintenance combination showed a statistically significant and clinically meaningful 2.3-year improvement in median PFS (6.7 years) vs. BR (4.4 years).1 This is a 50 percent improvement as compared to patients treated with BR and rituximab maintenance (stratified hazard ratio [HR]: 0.75, 95 percent confidence interval [CI], 0.59-0.96; p = 0.011).1
- Key secondary endpoints included: CR, time-to-next treatment (TTNT), overall survival (OS), and overall response rate (ORR).1
- A CR was achieved in 171 patients (65.5 percent) in the IMBRUVICA® plus BR arm and 151 patients (57.6 percent) in the placebo arm (p = 0.057).1 The rates of objective response were similar between the two arms (IMBRUVICA® plus BR: 89.7 percent; placebo: 88.5 percent).1
- Median TTNT was not reached in the IMBRUVICA® plus BR arm, the median TTNT was 92 months in the placebo plus BR arm (p < 0.001).1
- OS was similar between treatment arms and median OS was not reached in either treatment arm (p = 0.06).1,3
"More than eight years since its first FDA approval, IMBRUVICA has treated over 250,000 patients globally, fundamentally changing the treatment paradigm for complex B-cell malignancies," said Craig Tendler, M.D., Vice President, Late Development and Global Medical Affairs, Janssen Research & Development, LLC. "The Phase 3 SHINE study reinforces our continued commitment to the development of IMBRUVICA to provide meaningful differences and change outcomes for patients with B-cell malignancies where high unmet medical needs still remain."
The safety profile of the IMBRUVICA® plus BR regimen was consistent with known safety profiles of IMBRUVICA® as well as BR.1 Across all treated patients, the most common Grade 3/4 Adverse Events (AEs) ≥5 percent were neutropenia (IMBRUVICA® plus BR: 47.1 percent; BR: 48.1 percent), pneumonia (IMBRUVICA® plus BR: 20.1 percent; BR:14.2 percent), anemia (IMBRUVICA® plus BR: 15.4 percent; BR: 8.8 percent), thrombocytopenia (IMBRUVICA® plus BR: 12.7 percent; BR: 13.1 percent), rash (IMBRUVICA® plus BR: 12 percent; BR: 1.9 percent), and diarrhea (IMBRUVICA® plus BR: 6.9 percent; BR: 3.8 percent).1 Treatment-emergent AEs of clinical interest with BTKis included atrial fibrillation (AF) which was reported in 13.9 percent of patients in the IMBRUVICA® plus BR arm and 6.5 percent in the placebo arm; hypertension in 13.5 percent and 11.2 percent; major bleeding in 5.8 percent and 4.2 percent; any bleeding 42.9 percent and 21.5 percent; and arthralgia in 17.4 percent and 16.9 percent, respectively.1
IMBRUVICA® is currently approved globally for the treatment of adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy.4 Within the U.S., this indication is approved under accelerated approval based on overall response rate (ORR). Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
About IMBRUVICA®
IMBRUVICA® (ibrutinib) is a once-daily oral medication that is jointly developed and commercialized by Janssen Biotech, Inc. and Pharmacyclics LLC, an AbbVie company. IMBRUVICA® blocks the Bruton's tyrosine kinase (BTK) protein, which is needed by normal and abnormal B cells, including specific cancer cells, to multiply and spread. By blocking BTK, IMBRUVICA® may help move abnormal B cells out of their nourishing environments and inhibits their proliferation.5,6,7
IMBRUVICA® is approved in more than 100 countries for at least one indication and has been used to treat more than 250,000 patients worldwide. There are more than 50 company-sponsored clinical trials, including 18 Phase 3 studies, with over 11 years evaluating the efficacy and safety of IMBRUVICA®.
IMBRUVICA® was first approved by the U.S. Food and Drug Administration (FDA) in November 2013, and today is indicated for adult patients in six disease areas, including five hematologic cancers. These include indications to treat adults with CLL/SLL with or without 17p deletion (del17p), and adults with Waldenström's macroglobulinemia (WM), and adult patients with previously treated mantle cell lymphoma (MCL)*, as well as to treat adult patients with previously treated marginal zone lymphoma (MZL) who require systemic therapy and have received at least one prior anti-CD20-based therapy†, and adult patients with previously treated chronic graft-versus-host disease (cGVHD) after failure of one or more lines of systemic therapy.4
†Accelerated approval was granted for MCL and MZL based on overall response rate. Continued approval for MCL and MZL may be contingent upon verification and description of clinical benefit in confirmatory trials.
The National Comprehensive Cancer Network® (NCCN®), recommends ibrutinib (IMBRUVICA®) as a preferred regimen for the initial treatment of CLL/SLL and has Category 1 treatment status for treatment-naïve patients without deletion 17p/TP53 mutation and as a preferred treatment for treatment-naïve patients with deletion 17p/TP53 mutation. The NCCN Guidelines also recommend IMBRUVICA®, with or without rituximab, as a preferred regimen for the treatment of relapsed/refractory MCL, as a Category 1 preferred regimen for both untreated and previously treated WM patients, and as a preferred regimen for relapsed/refractory MZL.7
For more information, visit www.IMBRUVICA.com.
Please click here to see the full Prescribing Information.
Learn more at www.janssen.com. Follow us at @JanssenGlobal and @JanssenUS. Janssen Research & Development, LLC and Janssen Biotech, Inc. are part of the Janssen Pharmaceutical Companies of Johnson & Johnson.
J&J/AbbVie's Imbruvica 'significantly' cuts risk of disease progression in rare lymphoma, in trial
Jun. 03, 2022 11:19 AM ET
By: Ravikash, SA News Editor
Johnson & Johnson (NYSE:JNJ) and AbbVie's (NYSE:ABBV) Imbruvica plus standard therapy helped reduce the risk of disease progression or death by 25%, compared to placebo plus standard treatment, in older patients with mantle cell lymphoma (MCL) in a late-stage study.
https://seekingalpha.com/symbol/JNJ
https://seekingalpha.com/symbol/ABBV
biotecMAX™ Feb/Mar/APR/May 2022 Insight
Orencia (abatacept)
Bristol Myers Squibb Announces Topline Results Showing Treatment with Orencia (abatacept) Improved Survival in People Hospitalized with COVID-19
06/02/2022CATEGORY:
The Phase 3 ACTIV-1 Immune Modulators study was sponsored by the National Institutes of Health as part of the ACTIV initiative
Orencia was one of two immune modulators that improved survival for people hospitalized with COVID-19
Safety profile of Orencia remained consistent, with no new safety signals reported
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced topline results from the Phase 3 Accelerating COVID-19 Therapeutic Interventions and Vaccines (ACTIV-1) Immune Modulators clinical trial, sponsored by the National Institutes of Health (NIH). The study evaluated the safety and efficacy of a single dose of immune modulators, including Orencia (abatacept) IV (10 mg/kg) versus placebo when given with standard of care to determine if modulating the immune system’s response could speed recovery and reduce death in adults hospitalized with moderate to severe COVID-19.
Treatment with Orencia versus placebo displayed a strong but not statistically significant improvement in the primary endpoint of time to recovery as measured by day of hospital discharge. Analyses of the secondary endpoints, which included mortality and clinical status, demonstrated Orencia reduced participants’ risk of death and improved their clinical status at 28 days after entering the study when compared with placebo. The risk of death was lower for participants who received Orencia at 11%, versus 15% for those who received placebo, and the odds of dying were 37.4% lower. The relative improvement in mortality was similar in both moderately and severely ill participants. People in the Orencia group had 34.2% better odds of clinical improvement than those in the placebo group. The safety profile of Orencia remained consistent, with no new safety signals reported in the study.
“With the continued need across the globe for treatment options to address the threat of COVID-19, we are proud of our involvement in the ACTIV-1 Immune Modulators clinical trial and our scientific research related to the virus. The devastating resurgences associated with circulating and emerging COVID variants underscore the need for additional therapeutic options for those who are hospitalized with COVID-19,” said Samit Hirawat, MD, chief medical officer, Bristol Myers Squibb. “We are pleased with the data demonstrating the risk of death was lower for participants who received Orencia and look forward to continued collaboration with the NIH to assess the data and potentially bring this treatment option to those in need.”
The full report on these data will be published in a peer-reviewed scientific journal. Given the positive findings from the topline data, Bristol Myers Squibb plans to discuss these data and potential next steps with the U.S. Food and Drug Administration.
About ORENCIA
ORENCIA® is a selective costimulation modulator that disrupts the continuous cycle of T-cell activation.
U.S. Indications/Usage and Important Safety Information for ORENCIA® (abatacept)
Indications and Usage
Adult Rheumatoid Arthritis: ORENCIA® (abatacept) is indicated for the treatment of adult patients with moderately to severely active rheumatoid arthritis (RA).
Polyarticular Juvenile Idiopathic Arthritis: ORENCIA is indicated for the treatment of patients 2 years of age and older with moderately to severely active polyarticular juvenile idiopathic arthritis (pJIA).
Adult Psoriatic Arthritis: ORENCIA is indicated for the treatment of adult patients with active psoriatic arthritis (PsA).
Prophylaxis for Acute Graft versus Host Disease: ORENCIA is indicated for the prophylaxis of acute graft versus host disease (aGVHD), in combination with a calcineurin inhibitor and methotrexate, in adults and pediatric patients 2 years of age or older undergoing hematopoietic stem cell transplantation (HSCT) from a matched or 1 allele-mismatched unrelated-donor.
Please click here for Full Prescribing Information.
For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.
Source: Bristol Myers Squibb
Bristol Myers discloses topline results for arthritis drug in COVID-19
Jun. 02, 2022 11:54 AM ET
Bristol-Myers Squibb Company (BMY)
By: Dulan Lokuwithana, SA News Editor1 Comment
- Bristol Myers Squibb (NYSE:BMY) said on Thursday that a Phase 3 trial for its arthritis therapy Orencia did not reach the primary endpoint with statistical significance in adults hospitalized with moderate to severe COVID-19.
- https://seekingalpha.com/news/3845077-bmy-stock-lower-after-topline-results-for-arthritis-drug-in-covid-19
- https://seekingalpha.com/symbol/BMY
- https://www.bms.com/
- https://www.orencia.com/
Tofersen (formerly IONIS-SOD1Rx), also known as BIIB067
New 12-Month Tofersen Data Presented at ENCALS Meeting Show Clinically Meaningful Benefit in People With SOD1-ALS
JUNE 3, 2022 • INVESTOR RELATIONS
- 12-month data show that earlier initiation of tofersen slowed decline across measures of clinical and respiratory function, strength, and quality of life
- Tofersen also led to robust and sustained reductions in neurofilament, a marker of neurodegeneration
- SOD1-ALS is a rare, progressive and fatal genetic form of the disease, leading to the loss of everyday functions and affecting approximately 2% of people with ALS
CAMBRIDGE, Mass., June 03, 2022 (GLOBE NEWSWIRE) -- Biogen Inc. (NASDAQ: BIIB) today announced new 12-month data for tofersen, an investigational antisense drug for people with superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). The data show that earlier initiation of tofersen compared to delayed initiation (six months later in the open-label extension [OLE] study) slowed declines in clinical function, respiratory function, muscle strength, and quality of life. At the time of the analysis, because the majority of participants survived without permanent ventilation (PV), the median time to death or PV could not be estimated. However, early survival data suggest a lower risk of death or PV with earlier initiation of tofersen. These results are based on new integrated data from the pivotal Phase 3 VALOR study and its OLE study.
The data were presented today at the European Network to Cure ALS (ENCALS) meeting in Edinburgh, Scotland between 9-10:25 a.m. BST. An archived version of the presentation will be available on the Investors section of Biogen’s website at investors.biogen.com.
Clinical Results
As previously reported in October 2021, VALOR, a six-month Phase 3 randomized study, did not meet the primary endpoint of change from baseline to week 28 in the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R). However, trends of reduced disease progression across multiple secondary and exploratory endpoints were observed. The new 12-month data further build on the results previously observed in the initial readout.
“The initial six-month and now 12-month results show that tofersen had an impact on important measures critical to people with SOD1-ALS,” said Timothy Miller, M.D., Ph.D., principal investigator of VALOR and ALS Center co-Director at Washington University School of Medicine, St. Louis. “These new 12-month data showed tofersen consistently slowed disease progression across endpoints and, if approved, may meaningfully change the lives of people living with SOD1-ALS.”
The 12-month data compare early initiation of tofersen (at the start of VALOR) to delayed initiation of tofersen (six months later, in the OLE). Over 12 months in the overall study population, results favored earlier start tofersen on measures of:
- Clinical function as measured by ALSFRS-R (difference of 3.5 points; 95% confidence interval [CI]: 0.4, 6.7)
- Respiratory function as measured by slow vital capacity (difference of 9.2 percent-predicted; 95% CI: 1.7, 16.6)
- Muscle strength as measured by the handheld dynamometry megascore (difference of 0.28; 95% CI: 0.05, 0.52)
- Quality of life as measured by the 5-item amyotrophic lateral sclerosis assessment questionnaire (ALSAQ-5) (difference of 10.3 points; 95% CI: -17.3, -3.2)
At the time of the analysis, because the majority of participants survived without PV, the median time to death or PV and median time to death, could not be estimated. However, early survival data suggest a lower risk of death or PV (Hazard ratio [HR] 0.36; 95% CI: 0.137, 0.941) and death (HR 0.27; 95% CI: 0.084, 0.890) with earlier initiation of tofersen.
Biomarker Results
The latest 12-month results show that reductions in total SOD1 protein (a marker of target engagement) and neurofilament (a marker of axonal injury and neurodegeneration) were sustained over time.
“In ALS, people with more rapidly progressing disease have higher neurofilament levels, most likely because their neurons and axons are degenerating more quickly,” said Merit Cudkowicz, M.D., co-principal investigator of the VALOR trial and co-founder of the Northeast ALS Consortium, Director of the Healey & AMG Center for ALS and Chair of Neurology at Massachusetts General Hospital and the Julieanne Dorn Professor of Neurology at Harvard Medical School. “Tofersen lowered neurofilament levels by approximately 40-50 percent. The combination of these biomarker results and the clinical outcomes data provide additional evidence of tofersen's potential to effectively slow the relentless progression of SOD1-ALS.”
Tofersen reduced total CSF SOD1 protein and plasma neurofilament levels in both early- and delayed-start groups as follows:
- 33 percent and 21 percent reduction in SOD1 protein, the intended target for tofersen, respectively
- 51 percent and 41 percent reduction in plasma neurofilament, a marker of neuron injury, respectively
Safety Results
The most common adverse events (AEs) in participants receiving tofersen in VALOR and the OLE study were headache, procedural pain, fall, back pain and pain in extremity. Most AEs in both VALOR and the OLE were mild to moderate in severity. Serious AEs were reported in 36.5 percent of participants who received tofersen in VALOR and/or the OLE and 17.3 percent of participants discontinued treatment due to an AE. Serious neurologic events including myelitis, radiculitis, aseptic meningitis, and papilledema, were reported in 6.7 percent of participants receiving tofersen in VALOR and its OLE. There were 14 deaths reported in tofersen-treated participants in VALOR and the OLE, all of which were determined not to be related to tofersen.
About VALOR and the OLE
VALOR was a 28-week Phase 3, randomized, double-blind, placebo-controlled study to evaluate the effects of tofersen 100 mg in 108 adults with ALS associated with a SOD1 mutation. In total, 108 participants were randomized in VALOR (n=72 to tofersen 100 mg and n=36 to placebo). Of these participants, 95 enrolled in the ongoing OLE. At the time of the analysis all participants had an opportunity for at least 12 months of follow-up, with a median exposure to tofersen of approximately 20 months (range: 1 – 34 months).
To account for disease heterogeneity, the planned clinical analyses adjusted for neurofilament levels as a marker of the disease progression rate at baseline. Neurofilaments are proteins that increase in blood and cerebrospinal fluid when neurons or their axons are damaged. Neurofilaments have been shown to be a prognostic marker of disease progression and survival in ALS.
“For more than a decade Biogen has pursued new medicines for ALS. These additional data further reinforce our belief in tofersen and we will continue to follow the science to change the course of this cruel and deadly disease,” said Toby Ferguson, M.D., Ph.D., Vice President and Head of the Neuromuscular Development Unit at Biogen. “Biogen is engaging with FDA and regulators around the world, the medical community and patient advocacy groups and will provide updates on next steps when appropriate.”
About Tofersen
Tofersen is an antisense drug being evaluated for the potential treatment of SOD1-ALS. Tofersen binds to SOD1 mRNA, allowing for its degradation by RNase-H in an effort to reduce synthesis of SOD1 protein production. In addition to the ongoing open label extension of VALOR, tofersen is being studied in the Phase 3 ATLAS study designed to evaluate whether tofersen can delay clinical onset when initiated in presymptomatic individuals with a SOD1 genetic mutation and biomarker evidence of disease activity. Biogen licensed tofersen from Ionis Pharmaceuticals, Inc. under a collaborative development and license agreement.
We routinely post information that may be important to investors on our website at www.biogen.com. Follow us on social media - Twitter, LinkedIn, Facebook, YouTube.
Biogen/Ionis tofersen slows decline in certain functions in ALS patients in new data
Jun. 03, 2022 6:26 AM ET
By: Ravikash, SA News Editor
Biogen (NASDAQ:BIIB) said that 12-month data for tofersen showed that earlier initiation of the drug slowed decline in clinical, and respiratory function, muscle strength, and quality of life in patients with superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS).
Tofersen was licensed by Biogen from Ionis (NASDAQ:IONS) in 2018. The drug had failed to meet the main goal of a phase 3 trial, called VALOR, data from which is part of this 12-month data.
https://seekingalpha.com/symbol/IONS
https://seekingalpha.com/symbol/BIIB
REMICADE® (infliximab)
Thursday, June 2, 2022
Immune modulator drugs improved survival for people hospitalized with COVID-19
Colorized scanning electron micrograph of an apoptotic cell (green) infected with SARS-COV-2 virus particles (blue), isolated from a patient sample.NIAID
A large randomized, placebo-controlled clinical trial led by the National Institutes of Health shows that treating adults hospitalized with COVID-19 with infliximab or abatacept – drugs widely used to treat certain autoimmune diseases – did not significantly shorten time to recovery but did substantially improve clinical status and reduce deaths.
Some COVID-19 patients experience an immune response in which the immune system unleashes excessive amounts of proteins that trigger inflammation that can lead to acute respiratory distress syndrome, multiple organ failure and other life-threatening complications. As part of the Accelerating COVID-19 Therapeutic Interventions and Vaccines (ACTIV) public-private initiative, NIH launched the ACTIV-1 Immune Modulators clinical trial to determine if certain drugs that help minimize the effects of an overactive immune response could speed recovery and reduce deaths in adults hospitalized with moderate to severe COVID-19. The ACTIV-1 master protocol included three sub-studies; each one tested an immune modulator drug as compared to a placebo. This approach allowed for coordinated and efficient evaluation of multiple investigational agents simultaneously.
The topline results showed:
- Compared to placebo, participants receiving infliximab (Remicade) displayed a strong but not statistically significant improvement in the primary endpoint of time to recovery as measured by day of discharge from hospital. Substantial improvements for both key secondary endpoints of mortality and clinical status at 28 days were observed. The 518 participants receiving infliximab had a death rate of 10.0%, compared to 14.5% for the 519 participants receiving placebo, resulting in 40.5% lower adjusted odds of dying. The relative improvement in mortality was similar in both moderately and severely ill participants. People in the infliximab group had 43.8% better odds of clinical improvement than those in the placebo group. Infliximab, which was given as a single dose, was developed and is marketed by Janssen Biotech, Inc., one of the Janssen Pharmaceutical Companies of Johnson & Johnson.
Johnson & Johnson arthritis drug leads to clinical improvement in hospitalized COVID-19
Jun. 02, 2022 12:25 PM ET
By: Dulan Lokuwithana, SA News Editor1 Comment
- The National Institutes of Health (NIH) announced Thursday that infliximab (Remicade), an arthritis drug marketed by Johnson & Johnson (NYSE:JNJ), led to a clinical improvement in hospitalized COVID-19 patients, but without statistical significance
- https://seekingalpha.com/news/3845099-johnson-johnson-arthritis-drug-leads-to-clinical-improvement-in-hospitalized-covid-19
- https://seekingalpha.com/symbol/JNJ
- https://www.jnj.com/
- https://www.remicade.com/
OLPASIRAN
AMGEN ANNOUNCES POSITIVE TOPLINE PHASE 2 DATA FOR INVESTIGATIONAL OLPASIRAN IN ADULTS WITH ELEVATED LIPOPROTEIN(a)
Study Showed Significant, Sustained Reduction in Lipoprotein(a) During Treatment Period
Lipoprotein(a) Reduction was Consistent With Phase 1 Results
THOUSAND OAKS, Calif., May 31, 2022 /PRNewswire/ -- Amgen (NASDAQ:AMGN) today announced positive topline data from the Phase 2 OCEAN(a)-DOSE clinical study, evaluating olpasiran (formerly AMG 890) in 281 adult patients with Lipoprotein(a), or Lp(a), levels over 150 nmol/L and evidence of atherosclerotic cardiovascular disease (ASCVD). Olpasiran is a small interfering RNA designed to lower the body's production of apolipoprotein(a), a key component of Lp(a) that has been associated with an increased risk of cardiovascular events.
In the double-blind placebo-controlled treatment period, olpasiran was administered up to 225 mg subcutaneously every 12 weeks to patients with a median baseline Lp(a) of approximately 260 nmol/L. These data demonstrated a significant reduction from baseline in Lp(a) of up to or greater than 90 percent at week 36 (primary endpoint) and week 48 (end of treatment period) for the majority of doses. No new safety concerns were identified during this treatment period.
"Lp(a) has remained an elusive target since it was first discovered almost 60 years ago because diet and exercise have minimal influence on Lp(a) levels as do currently available medicines, leaving patients with limited options," said David M. Reese, M.D., executive vice president of Research and Development at Amgen. "We are very enthusiastic about these results and look forward to advancing olpasiran as a potential treatment for patients with elevated Lp(a)."
Data from the Phase 2 study will be presented at a future medical congress and submitted for publication.
About Lp(a)
Lp(a) is genetically determined1,2 and reported to be an independent risk factor for cardiovascular disease (CVD). Although an agreed upon threshold for elevated Lp(a) is not firmly established, approximately 20% of adults have Lp(a) >125 nmol/L (or approximately 50 mg/dL).3 Evidence has emerged from pathophysiological, epidemiologic, and genetic studies on the potential role of elevated Lp(a) in contributing to myocardial infarction, stroke, and peripheral arterial disease .2
About OCEAN(a)
The OCEAN(a) (Olpasiran Trials of Cardiovascular Events And LipoproteiN(a) Reduction) clinical program for Amgen's investigational olpasiran is designed to treat patients with atherosclerotic cardiovascular disease (ASCVD) and elevated Lp(a) levels to reduce the risk of cardiovascular events.
The OCEAN(a)-DOSE trial is a multicenter, randomized, double-blind, placebo-controlled dosefinding Phase 2 study in 281 subjects with ASCVD and Lp(a) >150 nmol/L. Patients were randomly assigned to one of four active subcutaneous doses of olpasiran (10 mg Q12 weeks, 75 mg Q12 weeks, 225 mg Q12 weeks or 225 mg Q24 weeks) or matched placebo. The primary endpoint is percent change from baseline in Lp(a) at 36 weeks. A secondary endpoint is percent change from baseline in Lp(a) at 48 weeks.
For more information, visit www.amgen.com and follow us on www.twitter.com/amgen.
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SOURCE Amgen
Amgen's olpasiran helps reduce cholesterol level in patients with heart condition in trial
May 31, 2022 12:42 PM ET
By: Ravikash, SA News Editor5 Comments
Amgen's (NASDAQ:AMGN) olpasiran helped reduce a type of bad cholesterol in a phase 2 trial.
https://seekingalpha.com/symbol/AMGN
https://clinicaltrials.gov/ct2/show/NCT04270760
PAXLOVID™ (nirmatrelvir and ritonavir)
June 2, 20222:26 PM EDT Healthcare & Pharmaceuticals
Pfizer's Paxlovid reduces COVID risk in seniors regardless of vaccine status -study
JERUSALEM, June 2 (Reuters) - Pfizer Inc's (PFE.N) antiviral treatment Paxlovid reduces COVID-19 hospitalization and death rates in vaccinated and unvaccinated patients 65 years and older, according to a new study in Israel conducted during the rise of the Omicron variant of the coronavirus.
The treatment, however, was not found to prevent severe illness among younger adults, according to research from Clalit Health Services, Israel's largest healthcare provider.
Use of Pfizer's pills, authorized to treat newly infected, at-risk people in order to prevent severe illness, has soared in the United States along with a spike infections. Biden administration officials have pushed for wide use of Paxlovid, which the government purchased and provides free.
Pfizer's clinical trial tested Paxlovid in unvaccinated people who had risk factors for serious disease and found that the two-drug treatment cut the risk of hospitalization and death by 90%. That was during the Delta wave of the virus.
There has been some concern, amid anecdotal reports of bounceback COVID symptoms after patients get relief from taking Paxlovid, that the treatment might not be as effective in vaccinated patients.
The Israeli study, which was published without peer review as a preprint by online platform Research Square, included data from nearly 110,000 participants between Jan. 9 to March 10, when Omicron was the country's dominant coronavirus variant.
Senior citizens who had no prior immunity - meaning they were neither vaccinated nor recovered from a previous COVID case - saw an 86% drop in hospitalizations with Paxlovid. Those who had prior immunity also benefited, but at a lower rate of 60%.
In total, 0.6% of those 65 and older treated with Paxlovid - or 14 of 2,504 - had to be hospitalized. Those who did not receive Paxlovid were hospitalized about three times more often, or 762 out of 40,315.
Pfizer antiviral found to cut COVID risk in seniors irrespective of vaccination
Jun. 02, 2022 2:48 PM ET
By: Dulan Lokuwithana, SA News Editor
A new study has indicated that Pfizer’s (NYSE:PFE) COVID-19 pill Paxlovid could reduce hospitalization and death rates in COVID-19 patients aged 65 years and older regardless of their vaccination status during a period the Omicron variant was dominant.
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VABYSMO™ FARICIMAB-SVOA
Health Canada authorizes VABYSMO® (faricimab injection) for the treatment of Neovascular (Wet) Age-related Macular Degeneration (AMD) and Diabetic Macular Edema (DME)
Jun. 01, 2022 7:00 AM ET
- Neovascular (wet) age-related macular degeneration (AMD) and diabetic macular edema (DME) are two leading causes of vision loss in Canada1
- VABYSMO® (faricimab injection) is the first bispecific antibody that acts by neutralizing both VEGF-A and Ang-2 proteins, a dual mode of action (MoA) 2, 3, 4, 5
MISSISSAUGA, ON, June 1, 2022 /CNW/ - Hoffmann-La Roche Limited (Roche Canada) is pleased to announce today that Health Canada authorized VABYSMO® (faricimab injection) for the treatment of neovascular (wet) age-related macular degeneration (AMD) and diabetic macular edema (DME),6 two of the leading causes of vision loss among Canadians.1
VABYSMO is the first treatment for wet AMD and DME in Canada that acts by targeting both VEGF-A and Ang-2, two key drivers of vascular instability that have been associated with vision-threatening retinal conditions.2,3,4,5 This unique dual MoA is made possible by Roche's antibody engineering expertise.
"Faricimab, a new drug to treat wet AMD and DME, shows from the pivotal clinical trials an increase in treatment durability that may lessen the burden for patients and doctors while best maintaining our patient's vision," said Dr. David T. Wong, Ophthalmologist-in-Chief and Associate Professor, Unity Health at St. Michael's Hospital.
In wet AMD and DME, VABYSMO may improve vision outcomes with fewer injections into the eye7 – potentially helping to reduce the treatment burden for patients, caregivers, and their families.8
"The approval of VABYSMO is welcomed by Canadians living with wet AMD and DME," said Doug Earle, President and CEO, Fighting Blindness Canada. "It's critical to have treatment options and strategies that can reduce the burden of frequent injections for those living with a blinding eye disease. Frequent appointments can be challenging for both patients and caregivers, particularly for those in rural areas or with limited mobility – and access to treatment is critical to addressing vision loss."
Positive results from four Phase III studies support the potential of VABYSMO to offer an effective new treatment option for people living with DME and wet AMD, while maintaining a similar safety profile to the current standard of care.9
About the Health Canada Approval
In wet AMD, the Phase III TENAYA and LUCERNE studies show VABYSMO – when given at intervals of up to four months based on assessments of pre-specified visual and anatomic criteria at Weeks 20 and 24 as well as treating physician clinical assessment – consistently offers visual acuity gains non-inferior to the current standard of care, aflibercept. The primary endpoint of these studies is the average change in best-corrected visual acuity (BCVA) score from baseline. The average vision gains from baseline at one year in the VABYSMO arms were +5.8 and +6.6 letters, respectively, compared to +5.1 and +6.6 letters in the aflibercept arms.10
In DME, the Phase III YOSEMITE and RHINE studies showed that VABYSMO met its primary endpoint with dosing intervals of up to every four months consistently shown to offer visual acuity gains that were non-inferior to aflibercept given every two months. According to these findings, the average vision gains from baseline at one year in YOSEMITE were +11.6 and +10.7 eye chart letters in the VABYSMO treat-and-extend and two-month arms, respectively (with +10.9 letters in the aflibercept arm); in RHINE, the average gains from baseline at one year were +10.8 and +11.8 letters in the VABYSMO treat-and-extend and two-month arms, respectively (with +10.3 letters in the aflibercept arm).11
About VABYSMO® (faricimab injection)
VABYSMO is a humanized bispecific immunoglobulin G1 (IgG1) antibody that acts through inhibition of both Ang-2 and vascular endothelial growth factor A (VEGF-A). By inhibiting VEGF-A, faricimab suppresses endothelial cell proliferation, neovascularization and vascular permeability. By inhibiting Ang-2, faricimab is thought to increase vascular stability and desensitize blood vessels to the effects of VEGF-A. Ang-2 levels are increased in some patients with wet AMD and DME.21
For more information, please visit www.RocheCanada.com or follow us on Twitter @RocheCanada.
SOURCE Roche Canada
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Upadacitinib (RINVOQ®)
June 1, 2022
AbbVie Presents Positive Results from Phase 3 SELECT-AXIS 2 Trials of Upadacitinib (RINVOQ®) in Patients with Axial Spondyloarthritis at EULAR 2022
- Results from the SELECT-AXIS 2 non-radiographic axial spondyloarthritis (nr-axSpA) study demonstrated significantly greater improvements in signs and symptoms, pain, function, disease activity, health-related quality of life, and MRI-detected SI joint inflammation with upadacitinib (RINVOQ®) compared to placebo at week 141
- Results from the SELECT-AXIS 2 ankylosing spondylitis (AS) bDMARD-IR study demonstrated significantly greater improvements in signs and symptoms, pain, function, disease activity, health-related quality of life, and MRI-detected spine inflammation with upadacitinib compared to placebo at week 142
- Safety data were consistent with the known safety profile of upadacitinib, with no new risks observed1,2,3,4,5
NORTH CHICAGO, Ill., June 1, 2022 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced the presentation of full results from two studies from the Phase 3 SELECT-AXIS 2 program evaluating upadacitinib (RINVOQ®), an oral therapy, in adult patients with active non-radiographic axial spondyloarthritis (nr-axSpA) and patients with treatment-refractory active ankylosing spondylitis (AS) with an inadequate response (IR) to biologic disease-modifying antirheumatic drugs (bDMARDs). Both studies met the primary endpoint of Assessment of SpondyloArthritis international Society 40 percent response criteria (ASAS40) at week 14 versus placebo.1,2
In the SELECT-AXIS 2 nr-axSpA study, significantly more nr-axSpA patients achieved the primary endpoint of ASAS40 at week 14 with upadacitinib versus placebo (45 percent versus 23 percent; p<0.0001).1 Statistical significance was also achieved in the first 12 of 14 multiplicity-controlled secondary endpoints compared to placebo at week 14 including change from baseline in patient's assessment of total back pain, Bath Ankylosing Spondylitis Functional Index (BASFI), Ankylosing Spondylitis Disease Activity Score (ASDAS) Low Disease Activity, Ankylosing Spondylitis Quality of Life (ASQoL), and MRI Spondyloarthritis Research Consortium of Canada (SPARCC) score for SI joints.1
"The new data observed from SELECT-AXIS 2 reinforce the potential of upadacitinib for patients across the spectrum of axial spondyloarthritis disease," said Neil Gallagher, M.D., Ph.D., vice president, development, chief medical officer, AbbVie. "At AbbVie, the needs of patients drive us to continue to innovate new ways to change the treatment landscape. We are encouraged by these positive data, which we hope will lead to the availability of a new treatment option for patients with nr-axSpA."
These findings, for which AbbVie disclosed topline results in 2021, were submitted as part of the regulatory applications to the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) to treat adults with active nr-axSpA with objective signs of inflammation who have responded inadequately to nonsteroidal anti-inflammatory drugs (NSAIDs).
"Non-radiographic axial spondyloarthritis is a progressive and disabling inflammatory disease, with limited treatment options. It often affects young adults, causing spinal inflammation that leads to back pain and stiffness and can significantly decrease quality of life," said Filip Van den Bosch, M.D., SELECT-AXIS 2 investigator and professor in the Department of Rheumatology at the University Hospital of Ghent University.* "These data suggest the potential of upadacitinib to help counter inflammation, relieve pain and improve function, helping patients living with nr-axSpA take control of their disease."
In the SELECT-AXIS 2 AS bDMARD-IR study, significantly more patients achieved the primary endpoint of ASAS40 at week 14 with upadacitinib versus placebo (45 percent versus 18 percent; p<0.0001).2 Statistical significance was also achieved in all multiplicity-controlled secondary endpoints compared to placebo at week 14, including change from baseline in patient's assessment of total back pain, BASFI, ASDAS low disease activity, ASQoL, and MRI SPARCC score for spine.2
No new safety risks were identified with upadacitinib when compared with its known safety profile.1,2,3,4,5 Through week 14 in the nr-axSpA study, the proportion of patients who experienced an adverse event (AE) was similar between both treatment groups (upadacitinib at 48 percent and placebo at 46 percent).1 Serious AEs were reported in four patients on upadacitinib and in two patients on placebo.1 Through week 14 in the AS bDMARD-IR study, the proportion of patients who experienced an adverse event was also similar (upadacitinib at 41 percent and placebo at 37 percent).2 In both the nr-axSpA study and the AS bDMARD-IR study, there were no reports of malignancy, major adverse cardiovascular events, venous thromboembolic events or death in patients receiving upadacitinib.1,2
The full results for both studies will be presented at the EULAR 2022 Congress. The SELECT-AXIS 2 nr-axSpA study results will be presented in an oral presentation on 1 June, 4:35 – 4:45pm (CEST) (OP0016) and the SELECT-AXIS 2 AS bDMARD-IR study results will be presented in a poster tour on 4 June, 11:37 – 11:45am (CEST) (POS0306).
Use of upadacitinib in nr-axSpA is not approved in the U.S. or EU, and its efficacy and safety are currently under review by the respective regulatory authorities.
About the SELECT-AXIS 2 program
SELECT-AXIS 2 (NCT04169373) was conducted under a master protocol and includes two separate studies (SELECT-AXIS 2 AS bDMARD-IR study, or Study 1, and SELECT-AXIS 2 nr-axSpA study, or Study 2). AbbVie previously announced topline data showing that upadacitinib 15 mg met the primary endpoint vs placebo at week 14 in Study 1 and Study 2.
More information on the SELECT-AXIS 2 program is available at www.clinicaltrials.gov (NCT04169373).
Study 1: SELECT-AXIS 2 bDMARD-IR AS study1
A randomized, double-blind, placebo-controlled, Phase 3 trial, which evaluated the efficacy and safety of upadacitinib compared with placebo, in 420 patients with a clinical diagnosis of AS who fulfilled the modified New York criteria, had BASDAI score ≥4 and total back pain score ≥4 (based on a numerical scale of 0-10), and had an inadequate response to bDMARD therapy.
Study 2: SELECT-AXIS 2 nr-axSpA study2
A randomized, double-blind, placebo-controlled, Phase 3 trial which evaluated the efficacy and safety of upadacitinib compared with placebo, in 314 patients with a clinical diagnosis of nr-axSpA. Patients enrolled in the study had active signs of inflammation consistent with axSpA on MRI of the sacroiliac (SI) joints, and/or high sensitivity C-reactive protein (hs-CRP) >upper limit of normal (2.87 mg/L) at screening, and who had BASDAI score ≥4 and a total back pain score ≥4 (based on a numerical scale of 0-10).
*Dr. Van den Bosch is a consultant and advisor for AbbVie.
About Axial Spondyloarthritis (axSpA)
Axial spondyloarthritis is a chronic inflammatory disease that affects the spine, causing back pain, limited mobility, and structural damage.6 It consists of two subsets that have been clinically defined as radiographic axial SpA (ankylosing spondylitis) and non-radiographic axial spondyloarthritis (nr-axSpA).6 In ankylosing spondylitis, patients have definitive structural damage of the sacroiliac joints visible on x-rays.6 Non-radiographic axial spondyloarthritis is clinically defined by the absence of definitive x-ray evidence of structural damage to the sacroiliac (SI) joint by plain x-ray.6
About RINVOQ® (upadacitinib)5
Discovered and developed by AbbVie scientists, RINVOQ is a selective JAK inhibitor that is being studied in several immune-mediated inflammatory diseases. In human cellular assays, RINVOQ preferentially inhibits signaling by JAK1 or JAK1/3 with functional selectivity over cytokine receptors that signal via pairs of JAK2.5
In the EU, RINVOQ is approved for the treatment of adults with moderate to severe active rheumatoid arthritis who have responded inadequately to, or who are intolerant to one or more disease-modifying anti-rheumatic drugs; for the treatment of active psoriatic arthritis (PsA) in adult patients who have responded inadequately to, or who are intolerant to one or more DMARDs; for the treatment of active ankylosing spondylitis (AS) in adult patients who have responded inadequately to conventional therapy; and for adults (15 mg and 30 mg) and adolescents (15 mg) with moderate to severe atopic dermatitis.
Phase 3 trials of RINVOQ in atopic dermatitis, axial spondyloarthritis, Crohn's disease, ulcerative colitis, giant cell arteritis and Takayasu arteritis are ongoing.7,8,9,10,11,12 Use of RINVOQ in nr-axSpA is not approved and remains under review by regulatory authorities.
EU Indications and Important Safety Information about RINVOQ® (upadacitinib)5
Indications
Rheumatoid arthritis
RINVOQ is indicated for the treatment of moderate to severe active rheumatoid arthritis in adult patients who have responded inadequately to, or who are intolerant to one or more disease-modifying anti-rheumatic drugs (DMARDs). RINVOQ may be used as monotherapy or in combination with methotrexate.
Psoriatic arthritis
RINVOQ is indicated for the treatment of active psoriatic arthritis in adult patients who have responded inadequately to, or who are intolerant to one or more DMARDs. RINVOQ may be used as monotherapy or in combination with methotrexate.
Ankylosing spondylitis
RINVOQ is indicated for the treatment of active ankylosing spondylitis in adult patients who have responded inadequately to conventional therapy.
Atopic dermatitis
RINVOQ is indicated for the treatment of moderate to severe atopic dermatitis in adults and adolescents 12 years and older who are candidates for systemic therapy.
For more information on AbbVie in rheumatology, visit https://www.abbvie.com/our-science/therapeutic-focus-areas/immunology/immunology-focus-areas/rheumatology.html.
SOURCE AbbVie
ABBV Dividend History
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AbbVie JAK inhibitor meets main goal in late-stage trials for arthritic disorders
Jun. 01, 2022 11:35 AM ET
By: Dulan Lokuwithana, SA News Editor
- AbbVie (NYSE:ABBV) said on Wednesday that its oral JAK inhibitor upadacitinib (Rinvoq) reached the primary endpoint versus placebo in a late-stage program targeted at certain adult patients with axial spondyloarthritis and active ankylosing spondylitis.
- https://seekingalpha.com/news/3844461-abbvie-rinvoq-meets-main-goal-in-late-stage-trials-for-arthritic-disorders
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TAZVERIK® (tazemetostat)
HUTCHMED Announces TAZVERIK® Approved to be Used in Hainan Pilot Zone in China
Hong Kong, Shanghai & Florham Park, NJ — Wednesday, June 1, 2022: HUTCHMED (China) Limited (“HUTCHMED”) (Nasdaq/AIM:HCM; HKEX:13) today announces that TAZVERIK® (tazemetostat) has been approved by the Health Commission and Medical Products Administration of Hainan Province to be used in the Hainan Boao Lecheng International Medical Tourism Pilot Zone (“Hainan Pilot Zone”), under the Clinically Urgently Needed Imported Drugs scheme, for the treatment of certain patients with epithelioid sarcoma (“ES”) and follicular lymphoma (“FL”) consistent with the label as approved by the U.S. Food and Drug Administration (“FDA”). Launched in 2013 and located in China, the Hainan Pilot Zone is a destination for international medical tourism and global hub for scientific innovation, welcoming 83,900 medical tourists in 2020, according to official data.
TAZVERIK® is a methyltransferase inhibitor of EZH2[1] developed by Epizyme, Inc. (“Epizyme”). It is approved by the FDA for the treatment of certain patients with ES and certain patients with FL under FDA accelerated approval granted in January and June 2020, respectively.
Dr Weiguo Su, Chief Executive Officer and Chief Scientific Officer of HUTCHMED, said: “The approval of TAZVERIK® in the Hainan Pilot Zone allows patients to gain early access to this first-in-class EZH2 inhibitor in China, as part of our commitment to bringing innovative medicines to people in need. In addition to its use in the Hainan Pilot Zone, we also plan to initiate further registration-enabling studies in China under the terms of our agreement with Epizyme to facilitate wider and easier patient access.”
In August 2021, HUTCHMED entered into a strategic collaboration with Epizyme to research, develop, manufacture and commercialize TAZVERIK® in China, Hong Kong, Macau and Taiwan.
About FL and ES
Follicular lymphoma (FL) is a subtype of non-Hodgkin’s lymphoma (“NHL”). FL accounts for approximately 17% of NHL. In 2020, there were an estimated 16,000 and 13,000 new cases of FL in China and the U.S., respectively. [2],[3],[4]
Epithelioid sarcoma (ES) is a rare, slow-growing type of soft tissue cancer. Radical tumor resection is the primary treatment for patients with ES. However, ES is known for its high propensity for locoregional recurrence and distant metastases. The survival of patients with ES is often unsatisfactory with very limited treatment options.[5]
About TAZVERIK® (tazemetostat)
TAZVERIK® is a methyltransferase inhibitor indicated in the United States for the treatment of:
- Adults and pediatric patients aged 16 years and older with metastatic or locally advanced epithelioid sarcoma not eligible for complete resection.
- Adult patients with relapsed or refractory follicular lymphoma whose tumors are positive for an EZH2 mutation as detected by an FDA-approved test and who have received at least two prior systemic therapies.
- Adult patients with relapsed or refractory follicular lymphoma who have no satisfactory alternative treatment options.
These indications are approved under accelerated approval by the U.S. FDA based on overall response rate and duration of response. Continued approval for these indications may be contingent upon verification and description of clinical benefit in confirmatory trials.
View the U.S. Full Prescribing Information here: www.tazverik.com
About Tazemetostat Clinical Development in China
HUTCHMED and Epizyme are developing tazemetostat in various hematological and solid tumors in Greater China, with HUTCHMED leading the China portion of Epizyme’s SYMPHONY-1 study. HUTCHMED and Epizyme also intend to conduct additional global studies jointly.
SYMPHONY-1 (EZH-302) is an international, multicenter, randomized, double-blind, active-controlled, 3-stage, biomarker-enriched, confirmatory Phase 1b/3 study, which is designed to evaluate the safety and efficacy of tazemetostat in combination with R² in patients with relapsed or refractory FL after at least one prior line of therapy(clinicaltrials.gov identifier: NCT04224493).
We intend to initiate a bridging study in FL to support registration of tazemetostat in China, as well as several combination studies of tazemetostat with HUTCHMED assets.
For more information, please visit: www.hutch-med.com or follow us on LinkedIn.
Hutchmed, Epizyme cancer drug Tazverik gets China's Hainan province health regulator nod
Jun. 01, 2022 6:41 AM ET
HUTCHMED (China) Limited (HCM), EPZM
By: Ravikash, SA News Editor
- Hutchmed (China) Epizyme's (NASDAQ:EPZM) cancer drug Tazverik (tazemetostat) was approved by the Health Commission and Medical Products Administration of Hainan Province.
- https://seekingalpha.com/news/3844268-hutchmed-epizyme-cancer-drug-tazverik-gets-chinas-hainan-province-health-regulator-nod
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- https://www.epizyme.com/medicines/
- https://www.tazverik.com/
Zuranolone (SAGE-217/BIIB125)
June 1, 2022
Sage Therapeutics and Biogen Announce that the Phase 3 SKYLARK Study of Zuranolone in Postpartum Depression Met its Primary and All Key Secondary Endpoints
Zuranolone 50 mg demonstrated a statistically significant and clinically meaningful improvement in depressive symptoms at Day 15, the primary endpoint, and at Days 3, 28, and 45, key secondary endpoints
Zuranolone 50 mg was generally well-tolerated and demonstrated a safety profile consistent with prior studies
Postpartum depression is one of the most common medical complications during and after pregnancy impacting approximately 1 in 8 women annually in the U.S.
Sage Therapeutics to host conference call today at 8:00 am ET
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jun. 1, 2022-- Sage Therapeutics, Inc. (Nasdaq: SAGE) and Biogen Inc. (Nasdaq: BIIB) today announced that the Phase 3 SKYLARK Study of zuranolone, an investigational oral drug being evaluated in women with postpartum depression (PPD), met its primary and all key secondary endpoints. Women treated with zuranolone 50 mg (n=98) demonstrated a statistically significant and clinically meaningful improvement in depressive symptoms at Day 15, the primary endpoint, compared to placebo (n=97) as measured by a change from baseline (CFB) in the 17-item Hamilton Rating Scale for Depression (HAMD-17) total score. The least-squares (LS) mean (SE) CFB in HAMD-17 total score at Day 15 for women who received zuranolone 50 mg was -15.6 (0.82) compared with -11.6 (0.82) for women who received placebo (LS mean difference -4.0 points; p=0.0007).
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“Reducing suffering from postpartum depression as rapidly and effectively as possible to restore maternal mental health is of the utmost importance for moms and their babies,” said Dr. Kristina Deligiannidis, principal investigator of the SKYLARK Study and Associate Professor, the Feinstein Institutes for Medical Research in Manhasset, New York. “These encouraging results are another important step in efforts to develop a novel treatment option for patients who suffer from this prevalent condition.”
The study met all key secondary endpoints with rapid and statistically significant improvement in depressive symptoms as early as Day 3 for participants treated with zuranolone 50 mg compared to placebo, which was sustained at all measured timepoints through Day 45 as measured by CFB in HAMD-17 total score.
About the SKYLARK Study
The SKYLARK Study (217-PPD-301) was a Phase 3, randomized, double-blind, placebo-controlled trial assessing the efficacy and safety of zuranolone 50 mg compared to placebo in adult women with severe PPD. The 200 patients enrolled in the study were randomized to receive zuranolone 50 mg or a placebo once nightly for 14 days. People in the study were then followed for an additional four weeks. The primary endpoint was the change from baseline in the 17-item Hamilton Rating Scale for Depression (HAMD-17) total score at Day 15. The companies anticipate further data disclosures at upcoming meetings and publications. For more information about this trial, please visit clinicaltrials.gov.
About Zuranolone
Zuranolone (SAGE-217/BIIB125) is a once-daily, two-week, investigational drug in development for the treatment of major depressive disorder (MDD) and postpartum depression (PPD). Zuranolone is an investigational oral neuroactive steroid (NAS) GABA-A receptor positive allosteric modulator (PAM). The GABA system is the major inhibitory signaling pathway of the brain and central nervous system and contributes to regulating brain function. Zuranolone has been granted Fast Track and Breakthrough Therapy Designation for MDD and Fast Track Designation for PPD by the U.S. Food & Drug Administration.
Zuranolone is being evaluated in the LANDSCAPE and NEST clinical development programs. The two development programs include multiple studies examining use of zuranolone in several thousand people with a variety of dosing, clinical endpoints, and treatment paradigms. The LANDSCAPE program includes five studies of zuranolone in people with MDD (MDD-201B, MOUNTAIN, SHORELINE, WATERFALL, and CORAL Studies). The NEST program includes two placebo-controlled studies of zuranolone in women with PPD (ROBIN and SKYLARK Studies). Additionally, Shionogi completed a Phase 2 study of zuranolone in Japan in people with MDD.
For more information, please visit www.sagerx.com.
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Source: Sage Therapeutics, Inc.
Biogen/Sage's zuranolone helps reduce postpartum depression symptoms, meets main goal of trial
Jun. 01, 2022 7:23 AM ET
By: Ravikash, SA News Editor
Biogen (NASDAQ:BIIB) and Sage Therapeutics's (NASDAQ:SAGE) oral drug zuranolone helped reduce symptoms of postpartum depression (PPD), meeting the main goal of a late-stage study.
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TREMFYA® (guselkumab)
New Data Show Patients Treated with First-in-Class TREMFYA® (guselkumab) Achieve Durable Efficacy Across Joint and Axial Symptoms of Active Psoriatic Arthritis Through Two Years
Adult patients with active psoriatic arthritis experienced persistent multi-domain efficacy and a safety profile consistent with that seen in plaque psoriasis
Further analyses show TREMFYA provided sustained improvements across measures of health-related quality of life
SPRING HOUSE, PENNSYLVANIA, June 1, 2022 – The Janssen Pharmaceutical Companies of Johnson & Johnson today announced new data from Phase 3 studies demonstrating patients treated with TREMFYA® (guselkumab) achieved consistent, long-term efficacy through two years across the domains of active psoriatic arthritis (PsA) – including joint, skin, enthesitis,a dactylitis,b spinal pain and disease severityc endpoints – irrespective of baseline characteristics.1 Further analyses showed TREMFYA also provided patients with sustained improvements in measures of health-related quality of life (HRQoL), including fatigue, pain and work productivity.2-5 These new data from the DISCOVER-1, DISCOVER-2, and COSMOS studies are among 38 abstracts Janssen is presenting during the 2022 Annual European Congress for Rheumatology (EULAR) meeting taking place virtually and in-person in Copenhagen on June 1-4, 2022. TREMFYA is the first and only fully human selective interleukin (IL)-23 inhibitor therapy approved in the U.S. for adults with moderate to severe plaque psoriasis (PsO) and adults with active psoriatic arthritis.6
“Psoriatic arthritis is a complex disease, with a range of joint, skin, and axial symptoms. Patients need long-lasting therapies that can provide efficacy across these varied challenges,” said presenting study author Philip Mease, M.D., Swedish Medical Center/Providence St. Joseph Health and University of Washington in Seattle, Washington.d “These new data reinforce previous research showing the durable efficacy of TREMFYA and demonstrate its effect on health-related quality of life, which is important for patients facing the debilitating effects of psoriatic arthritis in their everyday lives.”
The data presented at EULAR show:
Durable Efficacy Across Joint and Axial Symptoms
- TREMFYA-treated patients in DISCOVER-2 achieved consistent, long-term efficacy across domains of active PsA (joint, skin, enthesitis, dactylitis, spinal pain and disease severity endpoints) irrespective of baseline characteristics (POS0072).1
Further analyses of data from DISCOVER-2 show TREMFYA provided continued improvements across the key domains of active PsA recommended by the Group for Research and Assessment of Psoriasis and Psoriatic Arthritis (GRAPPAe; POS1017).7 In addition, increasing proportions of TREMFYA-treated patients with active PsA met minimal disease activity (MDA)f criteria through week 100 (POS1067).8 At week 100, 40 percent of patients treated with TREMFYA every eight weeks had achieved MDA, while 59 percent achieved a Disease Activity Index for Psoriatic Arthritis (DAPSA)g score of ≤14, indicating low disease activity, and 24 percent achieved DAPSA ≤4, indicating remission.7
- Data from the COSMOS study show complete resolution of dactylitis was achieved in ≥80% of patients who continued to receive TREMFYA at week 48 (AB0898).9 COSMOS investigated patients who had demonstrated inadequate response to tumor necrosis factor inhibition (TNFi-IR), who tend to have more difficult-to-treat manifestations of active PsA.9
- Substantial proportions of TREMFYA-treated patients in DISCOVER-2 also maintained resolution of dactylitis and enthesitis through two years (POS1028).10 Among those with the condition at baseline, resolution rates of dactylitis and enthesitis were observed at 64 and 54 percent, respectively, at week 24 among patients treated every 8 weeks (q8w) with TREMFYA.10 These rates increased through week 52 (78 percent and 61 percent, respectively) and were maintained at week 100 (83 percent and 70 percent, respectively, among q8w).10
- Patients with imaging-confirmed sacroiliitish who received TREMFYA maintained improvements in symptoms of axial involvement through two years (POS1037).11
Low Rates of Radiographic Progression
- For TREMFYA-treated patients in DISCOVER-2, mean changes in radiographic scores indicated low rates of radiographic progression through two years, showing the impact of reducing the progression of structural damage caused by active PsA (POS1035).12,i Radiographic progression is a measure of the structural damage caused by active PsA over time.13
- A further analysis showed earlier clinical response to TREMFYA predicts low rates of radiographic progression through two years in biologic-naïve active PsA patients (POS1031).14
About DISCOVER-1 (NCT03162796)26
DISCOVER-1 was a randomized, double-blind, multicenter Phase 3 study evaluating the efficacy and safety of TREMFYA administered by subcutaneous (SC) injection in participants with active PsA, including those previously treated with one or two TNF inhibitors. DISCOVER-1 evaluated 381 participants who were treated and followed through approximately one year. The primary endpoint was response of ACR20 at week 24 and primary endpoint data were previously presented at scientific congresses and published in The Lancet.27 In addition to ACR20, multiple other clinical outcomes were assessed, including ACR50/70, resolution of soft tissue inflammation, enthesitis and dactylitis, improvement in physical function, skin clearance (IGA), and general health outcomes (36-Item Short-Form Health Survey [SF-36] Physical Component Summary [PCS] and Mental Component Summary [MCS]).
The study consisted of a screening phase of up to six weeks, a blinded treatment phase of 52 weeks that included a placebo-controlled period from week 0 to week 24 and a blinded active treatment period from week 24 to week 52. It also included a safety follow-up phase through week 60 (i.e., approximately 12 weeks from the last administration of study agent at week 48). Efficacy, safety, pharmacokinetic, immunogenicity and biomarker evaluations were performed in the study on a defined schedule.
About DISCOVER-2 (NCT03158285)28
DISCOVER-2 is a randomized, double-blind, multicenter Phase 3 study evaluating the efficacy and safety of TREMFYA administered by SC injection in biologic-naïve patients with active PsA. DISCOVER-2 evaluated 739 participants who were treated and followed through approximately two years. The primary endpoint was response of ACR20 at week 24 and primary endpoint data were previously presented at scientific congresses and published in The Lancet.29 In addition to ACR20, multiple other clinical outcomes were assessed, including ACR50/70; resolution of soft tissue inflammation, enthesitis and dactylitis; improvement in physical function; skin clearance (IGA); and general health outcomes (SF-36 PCS and MCS). DISCOVER-2 also assessed changes in structural damage as a key secondary endpoint (PsA- modified vdH-S score).
The study consisted of a screening phase of up to six weeks, a blinded treatment phase of approximately 100 weeks that included a placebo-controlled period from week 0 to week 24 and a blinded active treatment period from week 24 to week 100. It also included a safety follow-up phase through week 112 (i.e., approximately 12 weeks after the last administration of study agent at week 100). Clinical efficacy, radiographic efficacy, health economics, safety, pharmacokinetics, immunogenicity, biomarker, and pharmacogenomics evaluations were performed in the study on a defined schedule.
About COSMOS (NCT03796858)30
COSMOS was a Phase 3b, multicenter, randomized, double-blind, placebo- controlled study to evaluate the safety and efficacy of TREMFYA in 285 patients with active PsA and IR to TNFi therapy. The primary endpoint was ACR20 response at week 24. Participants were randomized (2:1) to receive TREMFYA 100 mg at weeks 0, 4 and q8w thereafter, or placebo. The study included two periods: a 24-week double-blind, placebo-controlled period for the primary analysis of the efficacy and safety of TREMFYA compared with placebo and a 32-week active-treatment and safety follow-up period for additional analysis of the efficacy and safety of TREMFYA. Through week 48, non-responder imputation (NRI) rules were used for missing data (after the application of treatment failure rules [TFR]). Safety was monitored throughout the study to week 56.
About TREMFYA® (guselkumab)6
Developed by Janssen, TREMFYA is the first approved fully human monoclonal antibody that selectively binds to the p19 subunit of IL-23 and inhibits its interaction with the IL-23 receptor. IL-23 is an important driver of the pathogenesis of inflammatory diseases such as moderate to severe plaque PsO and active PsA. TREMFYA is approved in the U.S., Canada, Japan, and a number of other countries worldwide for the treatment of adults with moderate to severe plaque PsO who are candidates for injections or pills (systemic therapy) or phototherapy (treatment using ultraviolet light), and for the treatment of adult patients with active PsA. It is also approved in the EU for the treatment of moderate to severe plaque PsO in adults who are candidates for systemic therapy and for the treatment of active PsA in adult patients who have had an inadequate response or who have been intolerant to a prior disease-modifying antirheumatic drug therapy. TREMFYA is being investigated in Phase 3 clinical trials in both adults with moderately to severely active Crohn’s disease (NCT03466411) and adults with moderately to severely active ulcerative colitis (NCT04033445).
The Janssen Pharmaceutical Companies of Johnson & Johnson maintain exclusive worldwide marketing rights to TREMFYA®.
Infections. TREMFYA® may lower the ability of your immune system to fight infections and may increase your risk of infections. Your healthcare provider should check you for infections and tuberculosis (TB) before starting treatment with TREMFYA® and may treat you for TB before you begin treatment with TREMFYA® if you have a history of TB or have active TB. Your healthcare provider should watch you closely for signs and symptoms of TB during and after treatment with TREMFYA®.
Tell your healthcare provider right away if you have an infection or have symptoms of an infection, including:
- fever, sweats, or chills
- muscle aches
- weight loss
- cough
- warm, red, or painful skin or sores on your body different from your psoriasis
- diarrhea or stomach pain
- shortness of breath
- blood in your phlegm (mucus)
- burning when you urinate or urinating more often than normal
Please read the full Prescribing Information, including Medication Guide for TREMFYA®, and discuss any questions that you have with your doctor.
Learn more at www.janssen.com. Follow us at www.twitter.com/JanssenGlobal.
Johnson & Johnson's Tremfya demonstrates long-term benefit in psoriatric arthritis
Jun. 01, 2022 9:51 AM ET
By: Jonathan Block, SA News Editor
- Johnson & Johnson's (NYSE:JNJ) Janssen unit said that new data from a phase 3 study found that treatment with Tremfya (guselkumab) led to improvement in many symptoms of psoriatric arthritis over two years.
- https://seekingalpha.com/news/3844409-johnson-johnson-janssen-tremfya-demonstrates-long-term-benefit-in-psoriatric-arthritis
- https://seekingalpha.com/symbol/JNJ
- https://www.tremfya.com/
- https://www.jnj.com/
efanesoctocog alfa (BIVV001)
FDA grants efanesoctocog alfa Breakthrough Therapy designation for haemophilia A
1 Jun, 2022 07:00
Efanesoctocog alfa is the first factor VIII therapy to be awarded Breakthrough Therapy designation by the FDA
Designation is based on XTEND-1 phase 3 study data demonstrating a clinically meaningful prevention of bleeds and superiority in prevention of bleeding episodes compared to prior prophylaxis factor treatment
Efanesoctocog alfa is a novel and investigational factor VIII therapy designed to provide normal to near-normal factor activity levels for the majority of the week in a once-weekly prophylactic treatment regimen
Swedish Orphan Biovitrum AB (publ) (Sobi®) (STO:SOBI) and Sanofi (EURONEXT:SAN and NASDAQ:SNY) today announced that the United States Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to efanesoctocog alfa (BIVV001) for the treatment of people with haemophilia A, a rare and life-threatening bleeding disorder, based on data from the pivotal XTEND-1 phase 3 study. Sanofi and Sobi collaborate on the development and commercialisation of efanesoctocog alfa.
Breakthrough Therapy designation is designed to expedite the development and review of drugs in the US that target serious or life-threatening conditions. Drugs qualifying for this designation must show preliminary clinical evidence that the drug may demonstrate a substantial improvement on clinically significant endpoints over available therapies.
"The Breakthrough Therapy designation highlights efanesoctocog alfa's potential to transform treatment for people with haemophilia A by providing higher protection for longer duration," said John Reed, MD, PhD, Global Head of Research and Development at Sanofi. "This potential new class of factor VIII therapy represents how we are boldly advancing science to address unmet needs for the haemophilia community. We are excited to work with regulatory authorities during the filing and review of this innovative therapy."
"This designation supports the innovation of efanesoctocog alfa and acknowledges its potential to fulfil an unmet medical need for people living with haemophilia A," said Anders Ullman, MD, PhD, Head of Research & Development and Chief Medical Officer at Sobi. "We are committed to transforming lives for people living with rare diseases, and this is a testament to the medical innovation that science can bring."
Topline results from the pivotal XTEND-1 phase 3 study demonstrated that efanesoctocog alfa met the primary endpoint, showing a clinically meaningful prevention of bleeds in people with severe haemophilia A over a 52-week period. Importantly, the key secondary endpoint was also met, demonstrating that efanesoctocog alfa was superior to prior prophylactic factor VIII replacement therapy in preventing bleeding events based on an intra-patient comparison. Efanesoctocog alfa was well-tolerated, and inhibitor development to factor VIII was not detected. The most common treatment-emergent adverse events (>5 per cent of participants overall) were headache, arthralgia, fall, and back pain.
Data from the XTEND-1 phase 3 study are expected to be shared at an upcoming medical meeting and will serve as the basis for regulatory submission to the FDA by mid-year 2022. The FDA granted efanesoctocog alfa Orphan Drug designation in August 2017 and Fast Track designation in February 2021. The European Commission also granted efanesoctocog alfa Orphan Drug designation in June 2019. Regulatory submission in the EU will follow availability of data from the ongoing XTEND-Kids paediatric study, expected in 2023.
Haemophilia A occurs in about one in 5,000 male births annually, and more rarely in females. It is a lifelong condition in which the ability of a person's blood to clot is impaired due to a coagulation factor deficiency. People with haemophilia can experience bleeding episodes that can cause pain, irreversible joint damage, and life-threatening haemorrhages. Unmet medical needs remain for people with haemophilia to strengthen protection, reduce treatment burden and improve quality of life.
About efanesoctocog alfa (BIVV001)
Efanesoctocog alfa is a novel and investigational recombinant factor VIII (FVIII) therapy that is designed to extend protection from bleeds with once-weekly prophylactic dosing for people with haemophilia A. It builds on the innovative Fc fusion technology by adding a region of von Willebrand factor and XTEN® polypeptides to extend its time in circulation. It is the first investigational FVIII therapy that has been shown to break through the von Willebrand factor ceiling, which imposes a half-life limitation on current FVIII therapies. Efanesoctocog alfa is currently under clinical investigation and its safety and efficacy have not been evaluated by any regulatory authority.
About the Sobi and Sanofi collaboration
Sobi and Sanofi collaborate on the development and commercialisation of Alprolix® and Elocta®/Eloctate®. The companies also collaborate on the development and commercialisation of efanesoctocog alfa, an investigational factor VIII therapy with the potential to provide high sustained factor activity levels with once-weekly dosing for people with haemophilia A. Sobi has final development and commercialisation rights in the Sobi territory (essentially Europe, North Africa, Russia and most Middle Eastern markets). Sanofi has final development and commercialisation rights in North America and all other regions in the world excluding the Sobi territory.
Sobi®
Sobi is a specialised international biopharmaceutical company transforming the lives of people with rare diseases. Providing sustainable access to innovative medicines in the areas of haematology, immunology, and specialty care, Sobi has approximately 1,600 employees across Europe, North America, the Middle East, and Asia. In 2021, revenue amounted to SEK 15.5 billion. Sobi's share (STO: SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com, LinkedIn and YouTube.
Sanofi, Sobi's hemophilia A therapy gets FDA breakthrough therapy status
Jun. 01, 2022 5:15 AM ET Sanofi (SNY)
By: Ravikash, SA News Editor1 Comment
- The U.S. Food and Drug Administration (FDA) granted breakthrough therapy designation to Sanofi (NASDAQ:SNY) and Swedish Orphan Biovitrum's (Sobi) efanesoctocog alfa (BIVV001) to treat people with hemophilia A.
- https://seekingalpha.com/news/3844247-sanofi-sobis-hemophilia-a-therapy-gets-fda-breakthrough-therapy-status
- https://seekingalpha.com/symbol/SNY
- https://www.sobi.com/en/haematology
OMBLASTYS® (omburtamab)
- Y-mAbs Announces FDA Acceptance of Biologics License Application for OMBLASTYS® (omburtamab) for the Treatment of Neuroblastoma for Priority Review
Y-mAbs Announces FDA Acceptance of Biologics License Application for OMBLASTYS® (omburtamab) for the Treatment of Neuroblastoma for Priority Review
May 31, 2022PDF Version
NEW YORK, May 31, 2022 (GLOBE NEWSWIRE) -- Y-mAbs Therapeutics, Inc. (the “Company” or “Y-mAbs”) (Nasdaq: YMAB) a commercial-stage biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, today announced that the Biologics License Application (“BLA”) for OMBLASTYS® (omburtamab) for the treatment of pediatric patients with CNS/leptomeningeal metastasis from neuroblastoma has been accepted for priority review by the U.S. Food and Drug Administration (“FDA”). The FDA set an action date of November 30, 2022, under the Prescription Drug User Fee Act (“PDUFA”). The Agency also indicated in the BLA filing communication letter that it is planning to hold an advisory committee meeting in October 2022 to discuss the application.
“We believe that the FDA’s acceptance of our OMBLASTYS® BLA for priority review is a significant achievement for Y-mAbs and a crucial step forward as we anticipate that OMBLASTYS, if approved by the FDA, can address a significant unmet medical need for children with CNS/leptomeningeal metastasis from high-risk neuroblastoma, where no standard therapy currently exists, potentially adding a second rare disease product to our commercial portfolio and without any significant further investment in our existing commercial infrastructure. Further, OMBLASTYS® has a Rare Pediatric Disease Designation which, if approved, will provide Y-mAbs with a Priority Review Voucher, our second PRV, which would further strengthen our financial position as we would seek to monetize the OMBLASTYS® PRV,” stated Thomas Gad, Founder, President and Interim CEO. “We look forward to working with the Agency to bring OMBLASTYS® to the appropriate patients. We are excited to move forward and plan for a seamless commercial launch of OMBLASTYS®, if approved by the FDA.”
Researchers at Memorial Sloan Kettering Cancer Center (“MSK”) developed omburtamab, which is exclusively licensed by MSK to Y-mAbs. As a result of this licensing arrangement, MSK has institutional financial interests in the compound and in Y-mAbs.
DANYELZA®, OMBLASTYS® and Y-mAbs® are registered trademarks of Y-mAbs Therapeutics, Inc.
Source: Y-mAbs Therapeutics, Inc
Y-mAbs Omblastys gets FDA priority review to treat children with nerve cell cancer
May 31, 2022 9:46 AM ET
Y-mAbs Therapeutics, Inc. (YMAB)
By: Ravikash, SA News Editor
- The U.S. Food and Drug Administration granted priority review to Y-mAbs Therapeutics's (NASDAQ:YMAB) application seeking approval of Omblastys (omburtamab) to treat children with CNS/leptomeningeal metastasis from neuroblastoma.
- https://seekingalpha.com/news/3843965-y-mabs-omblastys-gets-fda-priority-review-to-treat-children-with-nerve-cell-cancer
- https://seekingalpha.com/symbol/YMAB
- https://ymabs.com/research-development/pipeline/
- https://ymabs.com/
biotecMAX™ Feb/Mar/APR/May 2022 Insight
Opdivo® (nivolumab)
U.S. Food and Drug Administration Approves Two Opdivo® (nivolumab)-Based Regimens as First-Line Treatments for Unresectable Advanced or Metastatic Esophageal Squamous Cell Carcinoma
05/27/2022CATEGORY:
Opdivo in combination with chemotherapy and Opdivo plus Yervoy® (ipilimumab) approved based on a Phase 3 trial showing improved overall survival versus chemotherapy alone1,2
Opdivo-based treatments are now approved for five indications in upper gastroesophageal cancers1
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) has approved both Opdivo® (nivolumab) (injection for intravenous use) in combination with fluoropyrimidine- and platinum-containing chemotherapy and Opdivo® plus Yervoy® (ipilimumab) as a first-line treatment for adult patients with unresectable advanced or metastatic esophageal squamous cell carcinoma (ESCC) regardless of PD-L1 status. The approvals are based on the Phase 3 CheckMate -648 trial, which evaluated Opdivo in combination with chemotherapy (n=321) and Opdivo plus Yervoy (n=325) each compared to chemotherapy alone (n=324), and was the largest Phase 3 trial of an immunotherapy in first-line ESCC.1
In the trial, Opdivo in combination with chemotherapy demonstrated superior overall survival (OS) compared to chemotherapy alone, both in all randomized patients, a secondary endpoint, which was hierarchically tested (Hazard Ratio [HR] 0.74, 95% Confidence Interval [CI]: 0.61 to 0.90, P=0.0021) and in patients whose tumors express PD-L1 (≥1%), a primary endpoint (HR 0.54, 95% CI: 0.41 to 0.71, P<0.0001).1,2 In all randomized patients the median OS (mOS) was 13.2 months (95% CI: 11.1 to 15.7) with Opdivo in combination with chemotherapy versus 10.7 months (95% CI: 9.4 to 11.9) with chemotherapy alone.1 In patients whose tumors express PD-L1 (≥1%) the mOS was 15.4 months (95% CI: 11.9 to 19.5) for Opdivo in combination with chemotherapy versus 9.1 months (95% CI: 7.7 to 10) with chemotherapy alone.1 The median progression-free survival (PFS) in all randomized patients, which was a hierarchically tested secondary endpoint, was 5.8 months (95% CI: 5.6 to 7.0) for Opdivo in combination with chemotherapy and 5.6 months (95% CI: 4.3 to 5.9) for chemotherapy alone (HR= 0.81; 95% CI: 0.67 to 0.99, P=not significant). Per pre-specified analysis, PFS did not meet statistical significance.1 The median PFS in patients whose tumors express PD-L1 (≥1%), which was a co-primary endpoint, was 6.9 months (95% CI: 5.7 to 8.3) for Opdivo in combination with chemotherapy and 4.4 months (95% CI: 2.9 to 5.8) for chemotherapy alone (HR 0.65; 95% CI: 0.49 to 0.86, P=0.0023).1
Opdivo plus Yervoy also improved OS compared to chemotherapy in all-randomized patients, a secondary endpoint, which was hierarchically tested (HR 0.78, 95% CI: 0.65 to 0.95, P=0.0110) and patients whose tumors express PD-L1 (≥1%), a primary endpoint (HR 0.64, 95% CI: 0.49 to 0.84, P=0.0010).1,2 The mOS was 12.8 months (95% CI: 11.3 to 15.5) with Opdivo plus Yervoy versus 10.7 months (95% CI: 9.4 to 11.9) with chemotherapy alone in all randomized patients and 13.7 months (95% CI: 11.2 to 17.0) with Opdivo plus Yervoy versus 9.1 months (95% CI: 7.7 to 10) with chemotherapy alone in patients whose tumors express PD-L1 (≥1%).1 The median PFS in patients whose tumors express PD-L1 (≥1%), which was a co-primary endpoint, was 4.0 months (95% CI: 2.4 to 4.9) for Opdivo plus Yervoy and 4.4 months (95% CI: 2.9 to 5.8) for chemotherapy alone (HR 1.02; 95% CI: 0.78 to 1.34, P=not significant). Per pre-specified analysis, PFS did not meet statistical significance.1,2 Median PFS in the PD-L1 (≥1%) population was not statistically significant and therefore it was not hierarchically tested in the all comers population.
Opdivo alone and Opdivo plus Yervoy are associated with the following Warnings and Precautions: severe and fatal immune-mediated adverse reactions including pneumonitis, colitis, hepatitis and hepatotoxicity, endocrinopathies, nephritis and renal dysfunction, dermatologic adverse reactions, other immune-mediated adverse reactions; infusion-related reactions; complications of allogeneic hematopoietic stem cell transplantation (HSCT); embryo-fetal toxicity; and increased mortality in patients with multiple myeloma when Opdivo is added to a thalidomide analogue and dexamethasone, which is not recommended outside of controlled clinical trials.1 Please see the Important Safety Information section below.
“Today brings welcome news for many advanced or metastatic esophageal squamous cell carcinoma patients and oncologists,” said Jaffer A. Ajani, M.D., CheckMate -648 co-first author and lead U.S. investigator, and professor of Gastrointestinal Medical Oncology at The University of Texas MD Anderson Cancer Center. “Unresectable advanced or metastatic esophageal squamous cell carcinoma is a challenging disease, and there’s a need for additional treatment options that may extend survival in the first-line setting.3,4 In the CheckMate -648 trial, two nivolumab-based combinations showed a survival benefit compared to chemotherapy alone, offering new treatment options regardless of PD-L1 status.”1
This application was reviewed under the FDA’s Real-Time Oncology Review (RTOR) pilot program, which aims to ensure that safe and effective treatments are available to patients as early as possible.5
“At Bristol Myers Squibb, we recognize the need that exists for many patients facing upper gastroesophageal cancers, including advanced or metastatic esophageal squamous cell carcinoma, and we are focused on our goal to bring forward new treatment options with proven survival benefits regardless of PD-L1 status and histology,” said Adam Lenkowsky, senior vice president and general manager, U.S., Cardiovascular, Immunology, Oncology, Bristol Myers Squibb.6 “Today’s approvals bring two first-line immunotherapy-based treatment options at once, Opdivo in combination with chemotherapy and Opdivo plus Yervoy as the first dual immunotherapy option, to newly diagnosed patients with unresectable advanced or metastatic esophageal squamous cell carcinoma, further building on the role of Opdivo-based regimens in upper gastroesophageal cancers.”1
About CheckMate -648
CheckMate -648 is a randomized Phase 3 study evaluating Opdivo plus Yervoy or Opdivo in combination with chemotherapy (fluorouracil and cisplatin) against chemotherapy (fluorouracil plus cisplatin) alone in adult patients with previously untreated unresectable advanced, recurrent, or metastatic esophageal squamous cell carcinoma.1,2 The primary endpoints of the trial are overall survival (OS) and progression-free survival (PFS) determined by blinded independent central review (BICR) in patients whose tumors express PD-L1 (≥1%) for both Opdivo-based combinations versus chemotherapy.2 Secondary endpoints of the trial, including OS and PFS as determined by BICR in the all randomized population, were tested hierarchically only if corresponding primary endpoints were significant.1,2
In the Opdivo plus Yervoy arm, patients received treatment with Opdivo 3 mg/kg every 2 weeks and Yervoy 1 mg/kg every 6 weeks up to 2 years or until disease progression or unacceptable toxicity.1,2 In the Opdivo in combination with chemotherapy arm, patients received treatment with Opdivo 240 mg on Day 1 and Day 15, fluorouracil 800 mg/m²/day on Day 1 through Day 5 for five days, and cisplatin 80 mg/m² on Day 1 of a four-week cycle.1,2 Patients were treated with Opdivo until disease progression, unacceptable toxicity, or up to 2 years.1,2 In patients who received Opdivo in combination with chemotherapy and in whom either fluorouracil and/or cisplatin were discontinued, other components of the treatment regimen were allowed to be continued. 2 Patients who discontinued combination therapy because of an adverse reaction attributed to ipilimumab were permitted to continue Opdivo as a single agent.2
Select Safety Profile from CheckMate -648 Study
Opdivo and/or chemotherapy were discontinued in 39% of patients and were delayed in 71% of patients for an adverse reaction.1 Serious adverse reactions occurred in 62% of patients receiving Opdivo in combination with chemotherapy.1 The most frequent (≥2%) serious adverse reactions in patients receiving Opdivo in combination with chemotherapy were pneumonia (11%), dysphagia (7%), esophageal stenosis (2.9%), acute kidney injury (2.9%), and pyrexia (2.3%).1 Fatal adverse reactions occurred in 5 (1.6%) patients treated with Opdivo in combination with chemotherapy; these included pneumonitis, pneumatosis intestinalis, pneumonia, and acute kidney injury.1 The most common (≥20%) adverse reactions in patients treated with Opdivo in combination with chemotherapy were nausea (65%), decreased appetite (51%), fatigue (47%), constipation (44%), stomatitis (44%), diarrhea (29%), and vomiting (23%).1
Opdivo and/or Yervoy were discontinued in 23% of patients and were delayed in 46% of patients for an adverse reaction.1 Serious adverse reactions occurred in 69% of patients receiving Opdivo plus Yervoy.1 The most frequent (≥2%) serious adverse reactions in patients receiving Opdivo plus Yervoy were pneumonia (10%), pyrexia (4.3%), pneumonitis (4%), aspiration pneumonia (3.7%), dysphagia (3.7%), hepatic function abnormal (2.8%), decreased appetite (2.8%), adrenal insufficiency (2.5%), and dehydration (2.5%).1 Fatal adverse reactions occurred in 5 (1.6%) patients treated with Opdivo plus Yervoy; these included pneumonitis, interstitial lung disease, pulmonary embolism, and acute respiratory distress syndrome.1 The most common (≥20%) adverse reactions in patients treated with Opdivo plus Yervoy were rash (31%), fatigue (28%), pyrexia (23%), nausea (22%), diarrhea (22%), and constipation (20%).1
INDICATIONS
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) after prior fluoropyrimidine- and platinum-based chemotherapy.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of completely resected esophageal or gastroesophageal junction cancer with residual pathologic disease in adult patients who have received neoadjuvant chemoradiotherapy (CRT).
OPDIVO® (nivolumab), in combination with fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of adult patients with unresectable advanced or metastatic esophageal squamous cell carcinoma (ESCC).
OPDIVO® (nivolumab), in combination with ipilimumab, is indicated for the first-line treatment of adult patients with unresectable advanced or metastatic esophageal squamous cell carcinoma (ESCC).
OPDIVO® (nivolumab), in combination with fluoropyrimidine- and platinum- containing chemotherapy, is indicated for the treatment of adult patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma.
Please see US Full Prescribing Information for OPDIVO and YERVOY.
For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.
Source: Bristol Myers Squibb
BMY Dividend History
https://www.nasdaq.com/market-activity/stocks/bmy/dividend-history
https://seekingalpha.com/symbol/BMY
Evrysdi® (risdiplam)
FDA approves Roche’s Evrysdi for use in babies under two months with spinal muscular atrophy (SMA)May 31, 2022
- Approval based on interim RAINBOWFISH data which show pre-symptomatic babies treated with Evrysdi for at least one year were able to sit, stand and walk
- Prescribing information updated with FIREFISH data showing the majority of symptomatic babies treated with Evrysdi for at least two years could sit for at least five seconds
- Evrysdi has proven efficacy in babies, children and adults with more than 5,000 patients treated to date
Basel, 31 May 2022 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the U.S. Food and Drug Administration (FDA) has approved a label extension for Evrysdi® (risdiplam) to include babies under two months old with spinal muscular atrophy (SMA). The approval is based on interim efficacy and safety data from the RAINBOWFISH study in newborns, which showed that the majority of pre-symptomatic babies treated with Evrysdi achieved key milestones such as sitting and standing with half walking after 12 months of treatment. Evrysdi is now approved in the US to treat SMA in children and adults of all ages.
Of the babies with 2 or 3 copies of the SMN2 gene (n=6), 100% were able to sit after one year of treatment with Evrysdi, 67% could stand and 50% of infants could walk independently. All infants were alive at 12 months without permanent ventilation.
“The approval of Evrysdi for pre-symptomatic babies is particularly important, as early treatment of SMA, before symptoms start to arise, can help babies to achieve motor milestones,” said Richard Finkel, M.D., RAINBOWFISH Principal Investigator and Director of the Experimental Neuroscience Program at St. Jude Children’s Research Hospital. “With the inclusion of SMA in newborn screening programmes, this approval provides the opportunity to start treating at home with Evrysdi soon after the diagnosis is confirmed.”
As part of the label extension, the Evrysdi prescribing information has also been updated to include recent two-year pooled data from Parts 1 and 2 of the FIREFISH study, which demonstrate long-term efficacy and safety in symptomatic infants with Type 1 SMA. The study enrolled babies aged 1-7 months and after two years of treatment with Evrysdi at the recommended dose (n=58), 60% of infants were able to sit without support* for five seconds, 40% for 30 seconds and 28% of infants were able to stand.**
Without treatment, infants do not achieve these milestones in the natural history of the disease. There were no treatment-related adverse events leading to withdrawal. The most common adverse reactions were upper respiratory tract infection (including nasopharyngitis, rhinitis), lower respiratory tract infection (including pneumonia, bronchitis), constipation, vomiting, and cough.
“The priority review and subsequent approval of Evrysdi for babies under two months of age speaks to the urgent ongoing need for additional treatment options for babies with SMA,” said Levi Garraway, M.D., Ph. D., Roche’s Chief Medical Officer and Head of Global Product Development. “Because of its efficacy in multiple settings, Evrysdi is now available for people with SMA from pre-symptomatic newborns to older adults. We are proud of this achievement, which has the potential to make a real difference to those living with SMA and their caregivers.”
Evrysdi is approved in 81 countries and the dossier is under review in a further 27 countries. More than 5,000 patients have now been treated worldwide with Evrysdi in clinical trials, compassionate use or real-world settings. Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.
About Evrysdi® (risdiplam)
Evrysdi is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency. Evrysdi is administered daily at home in liquid form by mouth or by feeding tube.
Evrysdi is designed to treat SMA by increasing and sustaining the production of the survival motor neuron (SMN) protein in the central nervous system (CNS) and peripheral tissues. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and movement.
Evrysdi was granted PRIME designation by the European Medicines Agency (EMA) in 2018 and Orphan Drug Designation by the U.S Food and Drug Administration in 2017. In 2021, Evrysdi was awarded Drug Discovery of the Year by the British Pharmacological Society as well as the Society for Medicines Research award for Drug Discovery. Evrysdi is currently approved in 81 countries and the dossier is under review in a further 27 countries.
Evrysdi is currently being evaluated in five multicentre trials in people with SMA:
- FIREFISH (NCT02913482) – an open-label, two-part pivotal clinical trial in infants with Type 1 SMA. Part 1 was a dose-escalation study in 21 infants with the primary objective of assessing the safety profile of risdiplam in infants and determining the dose for Part 2. Part 2 is a pivotal, single-arm study of risdiplam in 41 infants with Type 1 SMA treated for 2 years, followed by an open-label extension. Enrolment for Part 2 was completed in November 2018. The primary objective of Part 2 was to assess efficacy as measured by the proportion of infants sitting without support after 12 months of treatment, as assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development – Third Edition (BSID-III) (defined as sitting without support for 5 seconds). The study met its primary endpoint.
- SUNFISH (NCT02908685) – SUNFISH is a two-part, double-blind, placebo-controlled pivotal study in people aged 2-25 years with Types 2 or 3 SMA. Part 1 (n=51) determined the dose for the confirmatory Part 2. Part 2 (n=180) evaluated motor function using the total score of Motor Function Measure 32 (MFM-32) at 12 months. MFM-32 is a validated scale used to evaluate fine and gross motor function in people with neurological disorders, including SMA. The study met its primary endpoint.
- JEWELFISH (NCT03032172) – an open-label exploratory trial designed to assess the safety, tolerability, pharmacokinetics and pharmacodynamics in people with SMA aged 6 months to 60 years who previously received other investigational or approved SMA therapies prior to receiving Evrysdi. The study has completed recruitment (n=174).
- RAINBOWFISH (NCT03779334) – an open-label, single-arm, multicentre study, investigating the efficacy, safety, pharmacokinetics and pharmacodynamics of risdiplam in babies (~n=25), from birth to six weeks of age (at first dose) with genetically diagnosed SMA who are not yet presenting with symptoms. The study is fully enrolled.
- MANATEE (NCT05115110) – a global phase 2/3 clinical study to evaluate the safety and efficacy of GYM329 (RO7204239), an anti-myostatin molecule targeting muscle growth, in combination with risdiplam for the treatment of SMA in patients 2-10 years of age. The FDA Office of Orphan Products Development granted GYM329 Orphan Drug Designation for the treatment of patients with SMA in December 2021. The study is commencing recruitment in Q1 2022.
- https://www.evrysdi.com/
For more information, please visit www.roche.com.
Roche/PTC's SMA therapy Evrysdi gets FDA nod for use in infants below 2 months
May 31, 2022 5:30 AM ET
Roche Holding AG (RHHBY), RHHBFPTCT
By: Ravikash, SA News Editor1 Comment
- The U.S. Food and Drug Administration (FDA) approved the expanded use of Roche's (OTCQX:RHHBY) (OTCQX:RHHBF) Evrysdi in babies under two months old with spinal muscular atrophy (SMA).
- https://seekingalpha.com/news/3843787-roches-sma-therapy-evrysdi-gets-fda-nod-for-use-in-infants-below-2-months
- https://seekingalpha.com/symbol/RHHBY
- https://seekingalpha.com/symbol/PTCT
- https://www.evrysdi.com/
- https://ir.ptcbio.com/news-releases/news-release-details/fda-approves-label-extension-evrysdir-infants-spinal-muscular
- https://www.ptcbio.com/
Glofitamab
Thursday, May 26, 2022
New Pivotal Data Demonstrate Clinical Benefit of Genentech’s Glofitamab, a Potential First-in-Class Bispecific Antibody for People with Aggressive Lymphoma
Data to be presented for the first time at ASCO and EHA 2022 show glofitamab induces high and durable complete response rates in people with heavily pre-treated diffuse large B-cell lymphoma
Glofitamab has the potential to offer a readily available, fixed-duration CD20xCD3 bispecific antibody treatment approach for people with aggressive lymphoma
Glofitamab is part of Genentech’s industry-leading CD20xCD3 bispecific antibody development program, which aims to address the diverse needs and preferences of people with blood cancers
South San Francisco, CA -- May 26, 2022 --
Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that new pivotal data on its investigational CD20xCD3 T-cell engaging bispecific antibody, glofitamab, will be presented for the first time at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting from June 3-7 and the European Hematology Association (EHA) 2022 Congress from June 9-12. Data from the Phase II NP30179 expansion study demonstrated that, after a median follow-up of more than 12 months, fixed-duration glofitamab (given for a fixed amount of time, and not taken until disease progression) induces durable complete responses (CRs) in patients with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) who had received a median of three prior therapies.
“These data bring us one step closer towards our goal of finding solutions for people with heavily pre-treated diffuse large B-cell lymphoma, which often relapses and becomes more aggressive each time it returns,” said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. “The potential of glofitamab as a new fixed-duration, readily available treatment could be instrumental to improving outcomes for people with this difficult-to-treat cancer who otherwise have limited options.”
The pivotal Phase II NP30179 expansion study included patients with heavily pre-treated and highly refractory DLBCL, with 58.3% of patients refractory to their initial therapy and about one-third (33.1%) having received prior CAR T-cell therapy. After a median follow-up of 12.6 months, 39.4% of patients (n=61/155) achieved a CR (primary efficacy endpoint) and half of them (51.6%; n=80/155) achieved an overall response (the percentage of patients with a partial or complete response; secondary efficacy endpoint), as assessed by an independent review committee. The majority (77.6%) of complete responses were durable and ongoing at 12 months and the median duration of complete response had not yet been reached (not evaluable [16.8 months, not evaluable]). Cytokine release syndrome (CRS) was the most common adverse event occurring in 63.0% of patients. CRS events were predictable, generally low grade (mainly Grade 1 [47.4%] or 2 [11.7%]), occurred at initial doses, and only one patient discontinued glofitamab due to CRS. Incidence of Grade 3+ CRS was low (3.9%), with no Grade 5 events.
“I’m encouraged by these data as they signify new hope for these patients who otherwise have limited effective treatment options and have faced disappointment from not responding to multiple rounds of treatments,” said Associate Professor Michael Dickinson, Hematologist and Lead of the Aggressive Lymphoma Disease Group within Clinical Haematology at Peter MacCallum Cancer Centre and Royal Melbourne Hospital, Australia. “These glofitamab data suggest that patients may be able to achieve durable responses with a set course of treatment that they don’t have to take continuously until disease progression.”
Data from the NP30179 study have been submitted for approval to the European Medicines Agency (EMA), and submissions to additional health authorities worldwide, including to the U.S. Food and Drug Administration (FDA), are planned this year. Glofitamab is being investigated in several clinical trials and explored in earlier lines of lymphoma treatment.
Genentech is committed to improving standards of care to enhance the treatment experience and outcomes for people with blood cancers and the scientific data we are sharing at ASCO and EHA from our portfolio propels us further towards this goal. Genentech is investigating its CD20xCD3 T-cell engaging bispecific antibodies glofitamab and mosunetuzumab further as subcutaneous formulations and in additional Phase III studies that will expand the understanding of their impact in earlier lines of treatment, with the aim of providing people with different types of lymphomas with robust and durable treatment outcomes. Additionally, the European Commission (EC) recently granted approval of Polivy® (polatuzumab vedotin) in combination with Rituxan® (rituximab) plus cyclophosphamide, doxorubicin, and prednisone (R-CHP) for the treatment of adult patients with previously untreated diffuse large B-cell lymphoma (DLBCL), and the EMA’s Committee for Medicinal Products for Human Use recommended mosunetuzumab for approval for patients with R/R follicular lymphoma, who have received at least two prior systemic therapies.
About Glofitamab
Glofitamab is an investigational CD20xCD3 T-cell engaging bispecific antibody designed to target CD20 on the surface of B cells and CD3 on the surface of T cells. Glofitamab is based on a novel structural format called ‘2:1’. It is engineered to have two ‘Fab’ regions which bind to CD20, and one ‘Fab’ region which binds to CD3. This dual targeting activates and redirects a patient’s existing T cells to engage and eliminate target B cells by releasing cytotoxic proteins into the B cells. A robust clinical development program for glofitamab is ongoing, investigating the molecule as a monotherapy and in combination with other medicines, for the treatment of people with B-cell non-Hodgkin’s lymphomas, including diffuse large B-cell lymphoma and mantle cell lymphoma, and other blood cancers.
About the NP30179 Study
The NP30179 study [NCT03075696] is a Phase I/II, multicenter, open-label, dose-escalation and expansion study evaluating the safety, efficacy and pharmacokinetics of glofitamab in people with relapsed or refractory diffuse large B-cell lymphoma. Outcome measures include complete response rate by independent review committee (primary endpoint), overall response rate, duration of response, progression-free survival, safety and tolerability (secondary endpoints).
About Polivy® (polatuzumab vedotin-piiq)
Polivy is a first-in-class anti-CD79b antibody-drug conjugate (ADC). The CD79b protein is expressed specifically in the majority of B cells, an immune cell impacted in some types of non-Hodgkin’s lymphoma (NHL), making it a promising target for the development of new therapies. Polivy binds to CD79b and destroys these B cells through the delivery of an anti-cancer agent, which is thought to minimize the effects on normal cells. Polivy is being developed by Genentech using Seagen ADC technology and is currently being investigated for the treatment of several types of NHL.
Polivy U.S. Indication
Polivy is a prescription medicine used with other medicines, bendamustine and a rituximab product, to treat diffuse large B-cell lymphoma in adults who have progressed after at least two prior therapies.
The accelerated approval of Polivy is based on a type of response rate. There are ongoing studies to confirm the clinical benefit of Polivy.
For additional information about the company, please visit http://www.gene.com.
Genetech's glofitamab demonstrates durable complete response in large B-cell lymphoma
May 27, 2022 12:50 PM ET
By: Jonathan Block, SA News Editor
- A phase 2 study found that Genetech's bispecific antibody glofitamab led to high and durable complete response ("CR") rates in heavily pre-treated diffuse large B-cell lymphoma patients.
- https://seekingalpha.com/news/3843538-genetech-glofitamab-demonstrates-durable-complete-response-in-large-b-cell-lymphoma
- https://seekingalpha.com/symbol/RHHBY
- https://www.gene.com/
ADCETRIS® (brentuximab vedotin) plus chemotherapy
Takeda and Seagen to Highlight ADCETRIS® Combination Data Showing Statistically Significant Improvement in Overall Survival (OS) for Patients with Advanced Hodgkin Lymphoma
05/26/2022
– Randomized Phase 3 Clinical Trial of ADCETRIS Combination Met Key Secondary OS Endpoint, Demonstrating a 41% Reduction in Risk of Death vs. Standard of Care in Patients With Advanced Hodgkin Lymphoma –
CAMBRIDGE, Mass., OSAKA, Japan, and BOTHELL, Wash.- May 26, 2022—Takeda Pharmaceutical Company Limited (TSE:4502) and Seagen Inc., (NASDAQ:SGEN) today announced that overall survival (OS) data from the Phase 3 ECHELON-1 clinical trial of an ADCETRIS® (brentuximab vedotin) plus chemotherapy combination will be presented in an oral session at the 59th American Society of Clinical Oncology (ASCO) Annual Meeting on Friday, June 3, 2022, 1:00-4:00 PM CT, and at the 27th European Hematology Association (EHA) Annual Meeting on Friday, June 10, 2022, 11:30 – 12:45 CEST.
“The longer-term follow-up data from the ECHELON-1 trial have significant clinical importance, as this trial represents one of only two frontline randomized studies in advanced stage Hodgkin lymphoma that shows an overall survival advantage for the experimental arm,” said Stephen Ansell, M.D., Ph.D., Mayo Clinic, and ECHELON-1 study investigator. “These results clearly show that the addition of brentuximab vedotin to chemotherapy improves the long-term outcome of patients and the combination should be considered a standard of care.”
Data from the ECHELON-1 trial demonstrated a statistically significant improvement in OS in adult patients with previously untreated Stage III or IV classical Hodgkin lymphoma treated with ADCETRIS plus doxorubicin, vinblastine and dacarbazine (A+AVD) vs. doxorubicin, bleomycin, vinblastine, and dacarbazine (ABVD). With approximately six years median follow up (73 months), patients receiving A+AVD had a 41 percent reduction in the risk of death (hazard ratio [HR] 0.59; 95% confidence interval [CI]: 0.396 to 0.879), with an estimated OS rate (95% CI) of 93.9% (91.6, 95.5) at 6 years. The safety profile of ADCETRIS was consistent with previous studies, and no new safety signals were observed. Please see Important Safety Information, including a SPECIAL/BOXED WARNING for progressive multifocal leukoencephalopathy (PML), for ADCETRIS below.
“Patients with advanced-stage Hodgkin lymphoma have not benefitted from an improvement in overall survival outcomes for far too long,” said Chris Arendt, Ph.D., Head of Oncology Cell Therapy and Therapeutic Area Unit, Research and Development, at Takeda. “We are extremely proud of the results of the ECHELON-1 trial, as these findings represent a transformative improvement in care that can profoundly impact the lives of patients with advanced-stage disease. We look forward to sharing the data with regulators around the world.”
“These data unequivocally demonstrate the ability of the ADCETRIS combination regimen to improve upon a current standard of care, ABVD, for people with Hodgkin lymphoma by delivering an unsurpassed overall survival benefit,” said Roger Dansey, M.D., Interim CEO and Chief Medical Officer, Seagen. “We continue to evaluate the potential of ADCETRIS in different patient populations and in combination with other approved and investigational medicines.”
ADCETRIS is indicated for the treatment of adult patients with previously untreated Stage III or IV classical Hodgkin lymphoma in combination with AVD in the United States and for the treatment of adult patients with previously untreated CD30-positive Stage IV Hodgkin lymphoma in combination with AVD in Europe.
First-line brentuximab vedotin plus chemotherapy to improve overall survival in patients with stage III/IV classical Hodgkin lymphoma: An updated analysis of ECHELON-1. (Hematologic Malignancies—Lymphoma and Chronic Lymphocytic Leukemia on Friday, June 3, 2022, 1:00 PM-4:00 PM CT at McCormick Place, Hall A8)
Key findings, which will be presented by Dr. Ansell, include:
- The trial achieved its key secondary endpoint with the combination of A+AVD, resulting in a statistically significant improvement in OS versus the control arm of ABVD as assessed by an Independent Review Facility (IRF) (HR 0.59; p-value=0.009). This corresponds to a 41 percent reduction in the risk of death.
- At a median follow up of 73 months, 39 and 64 OS events occurred in the A+AVD and ABVD arms, respectively.
- Estimated six-year OS rates (95% CI) were 93.9% (91.6, 95.5) with A+AVD vs. 89.4% (86.6, 91.7) with ABVD.
- Subgroup analyses supported a consistent benefit for A+AVD vs. ABVD.
- The six-year PFS estimate (95% CI) was 82.3% (79.1, 85.0) with A+AVD vs. 74.5% (70.8, 77.7) with ABVD.
- A+AVD resulted in a manageable safety profile consistent with prior reports.
- Treatment-emergent peripheral neuropathy continued to resolve or improve in both arms, with 86% (379/443) and 87% (249/286) of patients in the A+AVD and ABVD arms, respectively, either completely resolving (72% vs. 79%) or improving (14% vs. 8%) by last follow up.
- Fewer patients reported second malignancies in the A+AVD vs. ABVD arm (23 vs. 32).
- No new safety signals were identified.
About the ECHELON-1 Trial
The ECHELON-1 trial, which compared the use of ADCETRIS in combination with AVD to ABVD in 1,334 patients with previously untreated Stage III or IV classical Hodgkin lymphoma, had a primary endpoint of modified progression-free survival (PFS) per independent review facility (IRF). A key secondary endpoint was OS, which was an event-driven, pre-specified, alpha-controlled analysis in the intention-to-treat population.
About ADCETRIS® (brentuximab vedotin)
ADCETRIS is an antibody-drug conjugate (ADC) comprising an anti-CD30 monoclonal antibody attached by a protease-cleavable linker to a microtubule disrupting agent, monomethyl auristatin E (MMAE), utilizing Seagen’s proprietary technology. The ADC employs a linker system that is designed to be stable in the bloodstream but to release MMAE upon internalization into CD30-positive tumor cells.
ADCETRIS injection for intravenous infusion has received FDA approval for six indications in adult patients with: (1) previously untreated systemic anaplastic large cell lymphoma (sALCL) or other CD30-expressing peripheral T-cell lymphomas (PTCL), including angioimmunoblastic T-cell lymphoma and PTCL not otherwise specified, in combination with cyclophosphamide, doxorubicin, and prednisone, (2) previously untreated Stage III or IV classical Hodgkin lymphoma (cHL), in combination with doxorubicin, vinblastine, and dacarbazine, (3) cHL at high risk of relapse or progression as post-autologous hematopoietic stem cell transplantation (auto-HSCT) consolidation, (4) cHL after failure of auto-HSCT or failure of at least two prior multi-agent chemotherapy regimens in patients who are not auto-HSCT candidates, (5) sALCL after failure of at least one prior multi-agent chemotherapy regimen, and (6) primary cutaneous anaplastic large cell lymphoma (pcALCL) or CD30-expressing mycosis fungoides (MF) who have received prior systemic therapy.
Health Canada granted ADCETRIS approval with conditions for relapsed or refractory Hodgkin lymphoma and sALCL in 2013, and non-conditional approval for post-autologous stem cell transplantation (ASCT) consolidation treatment of Hodgkin lymphoma patients at increased risk of relapse or progression in 2017, adults with pcALCL or CD30-expressing MF who have had prior systemic therapy in 2018, for previously untreated Stage IV Hodgkin lymphoma in combination with doxorubicin, vinblastine, and dacarbazine in 2019, and for previously untreated adult patients with sALCL, peripheral T-cell lymphoma-not otherwise specified (PTCL-NOS) or angioimmunoblastic T-cell lymphoma (AITL), whose tumors express CD30, in combination with cyclophosphamide, doxorubicin, prednisone in 2019.
ADCETRIS received conditional marketing authorization from the European Commission in October 2012. The approved indications in Europe are: (1) for the treatment of adult patients with previously untreated CD30-positive Stage IV Hodgkin lymphoma in combination with doxorubicin, vinblastine and dacarbazine (AVD), (2) for the treatment of adult patients with CD30-positive Hodgkin lymphoma at increased risk of relapse or progression following ASCT, (3) for the treatment of adult patients with relapsed or refractory CD30-positive Hodgkin lymphoma following ASCT, or following at least two prior therapies when ASCT or multi-agent chemotherapy is not a treatment option, (4) for the treatment of adult patients with relapsed or refractory sALCL, and (5) for the treatment of adult patients with CD30-positive cutaneous T-cell lymphoma (CTCL) after at least one prior systemic therapy.
ADCETRIS has received marketing authorization by regulatory authorities in more than 70 countries for relapsed or refractory Hodgkin lymphoma and sALCL. See Important Safety Information below.
ADCETRIS is being evaluated broadly in more than 70 clinical trials, including a Phase 3 study in first-line Hodgkin lymphoma (ECHELON-1) and another Phase 3 study in first-line CD30-positive peripheral T-cell lymphomas (ECHELON-2), as well as trials in many additional types of CD30-positive malignancies.
Seagen and Takeda are jointly developing ADCETRIS. Under the terms of the collaboration agreement, Seagen has U.S. and Canadian commercialization rights and Takeda has rights to commercialize ADCETRIS in the rest of the world. Seagen and Takeda are funding joint development costs for ADCETRIS on a 50:50 basis, except in Japan where Takeda is solely responsible for development costs.
ADCETRIS (brentuximab vedotin) Important Safety Information (European Union)
Please refer to Summary of Product Characteristics (SmPC) before prescribing.
CONTRAINDICATIONS
ADCETRIS is contraindicated for patients with hypersensitivity to brentuximab vedotin and its excipients. In addition, combined use of ADCETRIS with bleomycin causes pulmonary toxicity.
Please see the full Prescribing Information, including BOXED WARNING, for ADCETRIS here.
For more information on our marketed products and robust pipeline, visit www.seagen.com and follow @SeagenGlobal on Twitter.
For more information, visit www.takedaoncology.com.
For more information, visit https://www.takeda.com.
ihttps://www.cancer.org/cancer/hodgkin-lymphoma.html
Seagen, Takeda's Adcetris meets secondary endpoint in Hodgkin lymphoma trial
May 26, 2022 6:02 PM ET
Takeda Pharmaceutical Company Limited (TAK), SGEN
By: Jonathan Block, SA News Editor
- Seagen (NASDAQ:SGEN) and Takeda Pharmaceutical's (NYSE:TAK) Adcetris (brentuximab vedotin) plus chemotherapy met its secondary endpoint of improvement in overall survival in an advanced Hodgkin lymphoma phase 3 trial.
- https://seekingalpha.com/news/3843327-seagen-takeda-adcetris-meets-secondary-endpoint-in-hodgkin-lymphoma-trial
- https://seekingalpha.com/symbol/TAK
- https://seekingalpha.com/symbol/SGEN
- https://www.adcetris.com/
- https://www.seagen.com/
XPOVIO® (selinexor)
Karyopharm to Present New Selinexor Data at the 2022 American Society of Clinical Oncology Annual Meeting
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– Encouraging Initial Data Observed in Phase 1/2 Study of Selinexor in Combination with Ruxolitinib in Treatment-Naïve Myelofibrosis, Including Favorable Tolerability with No Dose Limiting Toxicities and 75% of Evaluable Patients Demonstrating ≥35% Spleen Volume Reduction (SVR 35) at Week 12 –
– FDA Grants Orphan Drug Designation for Selinexor for the Treatment of Myelofibrosis –
– Exploratory Subgroup Analyses from SIENDO Trial in Patients with Endometrial Cancer Treated with Selinexor as Maintenance Therapy Identified p53 Wild-type as a Potentially Important Predictor of Efficacy, with 10-month PFS Improvement over Placebo; No Benefit Was Seen in Patients with p53 Mutant Tumors –
NEWTON, Mass., May 26, 2022 /PRNewswire/ -- Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, today announced promising initial data from a Phase 1/2 study evaluating selinexor in combination with ruxolitinib in patients with treatment-naïve myelofibrosis and subgroup analyses and molecular classification data from the SIENDO study evaluating selinexor in endometrial cancer. These data, and four additional abstracts, will be presented at the 2022 American Society of Clinical Oncology Annual Meeting (ASCO 2022), being held in Chicago from June 3-7, 2022.
Results from Phase 1/2 Study Evaluating Selinexor in Combination with Ruxolitinib in Patients with Treatment-Naïve Myelofibrosis
The initial data to be presented at ASCO 2022 were based on the Phase 1 portion of the Phase 1/2 study evaluating the safety and preliminary efficacy of once-weekly selinexor in combination with standard dose ruxolitinib in patients with treatment-naïve myelofibrosis. As of May 1, 2022, 15 patients had been dosed with one of two dose levels of selinexor, 40 mg (n=3) and 60 mg (n=12), in combination with ruxolitinib 15/20 mg BID.
Seventy-five percent of evaluable patients (6 out of 8) demonstrated ≥35% reduction in spleen volume (SVR35) at week 12. Five out of 10 transfusion independent patients who had at least eight weeks of treatment maintained stable hemoglobin (± 2g/dL) or improved hemoglobin level (>2g/dL increase) at last follow up. In addition, all of the evaluable 7 patients, who had been at least 12 weeks on treatment and had complete data, experienced rapid reductions in their symptom scores with 3 of 7 patients having ≥50% reduction (TSS50) at week 12.
There were two patients who discontinued therapy in the trial: One patient discontinued after 5 months of therapy due to unrelated AEs (dizziness, atrial fibrillation, and pulmonary hypertension) and another patient discontinued after 8 weeks of therapy due to progression to AML.
The combination of selinexor and ruxolitinib was generally well-tolerated and manageable. No dose-limiting toxicities were reported at either dose level of selinexor, and the most common adverse event (AE) was nausea (40%), the majority of which were grade 1/2. Both the 40 mg and 60 mg dose levels were generally well tolerated, with the most common reported Grade 3-4 treatment-emergent AEs being thrombocytopenia (27%), anemia (20%), neutropenia (20%) and atrial fibrillation (20%). Hematologic adverse events were reversible with dose interruptions and reductions.
The trial is currently enrolling patients in the Phase 1b dose expansion part of the study.
"Despite tremendous improvements in the lives of patients with myelofibrosis with the introduction of the JAK inhibitors, there remains a significant unmet need. We are excited to develop a novel combination that may further improve outcomes for these patients," said Reshma Rangwala, MD, PhD, Chief Medical Officer of Karyopharm. "Following the promising initial results of selinexor in relapsed and refractory myelofibrosis, we are very excited with these preliminary data in treatment-naïve myelofibrosis patients in combination with ruxolitinib, with encouraging results seen across multiple measures including spleen volume reductions, improvements in symptom scores and management of anemia. We look forward to sharing these promising initial data with the broader medical and scientific community at ASCO 2022."
SIENDO Study Subgroup Analysis
A preliminary analysis of exploratory subgroups of the SIENDO study assessed four distinct molecular subtypes in endometrial cancer using The Cancer Genome Atlas (TCGA), one of the accepted gynecologic oncology algorithms that is used to calculate prognostic risk scores. This analysis indicated that patients with p53 wild-type endometrial cancer treated with selinexor showed a median progression-free survival of 13.7 months compared to 3.7 months for patients on placebo.
"These data suggest that selinexor may provide meaningful benefit to patients with p53 wild-type endometrial cancer and reinforce the need to further evaluate selinexor's potential in a registration-enabling Phase 3 study, that we are planning to initiate in the second half of this year," added Dr. Rangwala.
About the SIENDO Study
The Phase 3 SIENDO study (ENGOT-EN5/GOG-3055) is a multicenter, blinded, placebo-controlled, randomized study evaluating the efficacy and safety of selinexor as a maintenance therapy following chemotherapy in patients with advanced or recurrent endometrial cancer. The study enrolled 263 patients with primary stage IV or recurrent disease who had a partial or complete response after at least 12 weeks of standard taxane-platinum combination chemotherapy. Patients were randomized 2:1 to receive either maintenance therapy of 80mg of selinexor taken once weekly, or placebo, until disease progression. The primary endpoint of the study was statistically significant improvement of progression-free survival compared to placebo. The goal of the study was to demonstrate a hazard ratio of 0.6 or better. In partnership with Karyopharm, the study was initiated by the European Network for Gynaecological Oncological Trial (ENGOT) group. In the U.S., the collaboration includes the GOG Foundation, Inc. (GOG-F).
About XPOVIO® (selinexor)
XPOVIO is a first-in-class, oral exportin 1 (XPO1) inhibitor and the first of Karyopharm's Selective Inhibitor of Nuclear Export (SINE) compounds to be approved for the treatment of cancer. XPOVIO functions by selectively binding to and inhibiting the nuclear export protein XPO1. XPOVIO is approved in the U.S. and marketed by Karyopharm in multiple oncology indications, including: (i) in combination with Velcade® (bortezomib) and dexamethasone (XVd) in patients with multiple myeloma after at least one prior therapy; (ii) in combination with dexamethasone in patients with heavily pre-treated multiple myeloma; and (iii) in patients with diffuse large B-cell lymphoma (DLBCL), including DLBCL arising from follicular lymphoma, after at least two lines of systemic therapy. XPOVIO (also known as NEXPOVIO® in certain countries) has received regulatory approvals in a growing number of ex-U.S. territories and countries, including Europe, the United Kingdom, China, South Korea, Singapore and Israel, and is marketed in those areas by Karyopharm's global partners. Selinexor is also being investigated in several other mid- and late-stage clinical trials across multiple high unmet need cancer indications, including multiple myeloma, endometrial cancer and myelofibrosis. For more information about Karyopharm's products or clinical trials, please contact the Medical Information department at:
Tel: +1 (888) 209-9326
Email: medicalinformation@karyopharm.com
XPOVIO® (selinexor) is a prescription medicine approved:
- In combination with bortezomib and dexamethasone for the treatment of adult patients with multiple myeloma who have received at least one prior therapy (XVd).
- In combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti–CD38 monoclonal antibody (Xd).
- For the treatment of adult patients with relapsed or refractory diffuse large B–cell lymphoma (DLBCL), not otherwise specified, including DLBCL arising from follicular lymphoma, after at least 2 lines of systemic therapy. This indication is approved under accelerated approval based on response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).
- https://www.xpovio.com/
For additional product information, including full prescribing information, please visit www.XPOVIO.com.
For more information about our people, science and pipeline, please visit www.karyopharm.com, and follow us on Twitter at @Karyopharm and LinkedIn.
XPOVIO® and NEXPOVIO® are registered trademarks of Karyopharm Therapeutics Inc. Any other trademarks referred to in this release are the property of their respective owners.
SOURCE Karyopharm Therapeutics Inc.
Karyopharm wins FDA’s Orphan Drug status for leading asset in myelofibrosis
May 26, 2022 1:04 PM ET
Karyopharm Therapeutics Inc. (KPTI)
By: Dulan Lokuwithana, SA News Editor
- The U.S. Food and Drug Administration (FDA) on Thursday granted its Orphan Drug Designation for Karyopharm Therapeutics (NASDAQ:KPTI) for the company’s leading product, selinexor in myelofibrosis.
- https://seekingalpha.com/news/3843203-kpti-stock-gains-as-company-wins-fdas-orphan-drug-status-for-cancer-drug
- https://seekingalpha.com/symbol/KPTI
- https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=880222
- https://www.xpovio.com/
- https://www.karyopharm.com/
Breyanzi (lisocabtagene maraleucel)
Data from Phase 2 PILOT Study of Bristol Myers Squibb’s CAR T cell Therapy Breyanzi Show Substantial Durable Responses in Patients with Refractory or Relapsed Large B-cell Lymphoma After First-Line Therapy
05/26/2022CATEGORY:
First disclosure of results from primary analysis of Phase 2 PILOT study shows Breyanzi delivered complete responses in more than half of patients with refractory or relapsed large B-cell lymphoma after first-line therapy who were not deemed candidates for stem cell transplant
PILOT patient-reported outcomes analysis showed treatment with Breyanzi improved health-related quality of life measures for patients
Breyanzi is the only CAR T cell therapy that has been evaluated in two distinct trials in the second-line setting for large B-cell lymphoma, underscoring its value, if approved, as an important treatment option after failure of first-line therapy
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced results from the primary analysis of PILOT, a multicenter, Phase 2 study evaluating Breyanzi (lisocabtagene maraleucel) in adults with refractory or relapsed large B-cell lymphoma (LBCL) after first-line therapy who were not deemed candidates for high-dose chemotherapy and hematopoietic stem cell transplant (HSCT). The PILOT study is the only company-sponsored trial to evaluate a CAR T cell therapy as a second-line treatment for patients with relapsed or refractory LBCL who are not considered candidates for stem cell transplant. The data will be presented in a poster presentation at the American Society of Clinical Oncology (ASCO) Annual Meeting on Saturday, June 4 from 9:00 AM – 12:00 PM EDT (ABSTRACT 7062).
The PILOT study enrolled a broad patient population of adults with refractory or relapsed LBCL after first-line treatment who were not considered candidates for transplant based on age, performance status and/or organ function and comorbidities, and regardless of time to relapse following first-line treatment. With a median follow-up of 12.3 months, the majority of patients treated with Breyanzi (n=61) saw a reduction in disease, with 80% of patients responding to treatment (overall response rate; 95% CI: 68.2 – 89.4) and 54% of patients achieving a complete response (CR; 95% CI:40.8-66.9). Responses with Breyanzi were durable, with a median duration of response of 12.1 months (95% CI: 6.2-NR) at 15.5 months median follow-up. In patients who achieved a CR, median duration of response was 21.7 months (95% CI: 12.7-NR). Median progression-free survival with Breyanzi was 9.0 months (95% CI: 4.2-NR), and median overall survival has not been reached (95% CI: 17.3-NR). In the PILOT study, patients were treated with Breyanzi and monitoredin both the inpatient and outpatient setting.
“For patients with large B-cell lymphoma that is refractory to or relapses after first-line therapy, stem cell transplant has been the only potentially curative treatment option, but the reality is many patients are not candidates for stem cell transplant, leaving limited treatment options,” said Leo I. Gordon, M.D., study investigator, Professor in Medicine, Northwestern University Feinberg School of Medicine and the Robert H. Lurie Comprehensive Cancer Center, Chicago, Illinois. “The results from the PILOT study, including the patient-reported outcomes, show that treatment with liso-cel as a second-line therapy offers durable responses with improved quality of life for patients who historically have had poor prognosis.”
LBCL, the most common type of non-Hodgkin lymphoma, is an aggressive blood cancer and approximately 40% of patients will have disease that is refractory to or relapses after first-line treatment. High-dose chemotherapy followed by autologous stem cell transplant has been the mainstay of care in the second-line setting; however, less than half of patients with primary refractory or relapsed disease are considered candidates for a stem cell transplant. For these patients, there are limited treatments that provide long-term disease control and palliative care is often the only option. If left untreated, patients with relapsed or refractory LBCL have a life expectancy of just three to four months.
“At Bristol Myers Squibb, we strive for cure by advancing innovative therapies that may provide long-term clinical benefit for some of the most challenging cancers with the hope of creating new standards of care that not only improve outcomes but also the patient experience,” said Anne Kerber, senior vice president, Cell Therapy Development, Bristol Myers Squibb. “With Breyanzi, we have boldly designed a broad clinical trial program in relapsed or refractory LBCL, including patients who are not intended for stem cell transplant after failure of first-line therapy. These results from the PILOT study continue to demonstrate the practice-changing potential of Breyanzi in this setting, delivering on the promise of CAR T cell therapy for more patients.”
In the PILOT study, Breyanzi showed a manageable safety profile with no new safety signals and low rates of severe cytokine release syndrome (CRS) or neurologic events, and no Grade 4/5 CRS or neurologic events reported. Any grade CRS occurred in 38% of patients, with Grade 3 CRS reported in one patient (2%). Any grade neurologic events were seen in 31% of patients with Grade 3 neurologic events reported in three patients (5%).
In a separate analysis of patient-reported outcomes (PRO) from the PILOT study, patients who received Breyanzi and were evaluable for the PRO analysis (n=56) showed significant improvements in fatigue and pain. Improvements in overall lymphoma symptoms were clinically meaningful following treatment with Breyanzi, and in an individual patient-level analysis, 70% of patients reported meaningful improvements in quality of life based on FACT-LymS scores at month 6. Results from the analysis will be presented in a poster presentation on Monday, June 6 from 2:15 PM – 5:15 PM EDT (Abstract 6567).
A supplemental Biologics License Application for Breyanzi for the treatment of relapsed or refractory LBCL after failure of first-line therapy is currently under Priority Review with the U.S. Food and Drug Administration (FDA), with an assigned Prescription Drug User Fee Act (PDUFA) goal date of June 24, 2022.
Breyanzi, a differentiated CD-19 directed CAR T cell therapy, is currently approved by the FDA for the treatment of adult patients with relapsed or refractory LBCL after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified (including DLBCL arising from indolent lymphoma), high-grade B-cell lymphoma, primary mediastinal large B-cell lymphoma, and follicular lymphoma grade 3B. Breyanzi is not indicated for the treatment of patients with primary central nervous system lymphoma.
About Breyanzi
Breyanzi is a CD-19 directed chimeric antigen receptor (CAR) T cell therapy, administered as a defined composition to reduce variability of the CD8 and CD4 component dose. Breyanzi has a 4-1BB costimulatory domain which enhances the expansion and persistence of the CAR T cells. Breyanzi was previously approved by the U.S. Food and Drug Administration for the treatment of adult patients with relapsed or refractory LBCL after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified (including DLBCL arising from indolent lymphoma), high-grade B-cell lymphoma, primary mediastinal large B-cell lymphoma, and follicular lymphoma grade 3B. Breyanzi is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the BREYANZI REMS.
Breyanzi is also approved in the European Union, Switzerland, Japan and Canada for relapsed and refractory LBCL after two or more lines of systemic therapy. Bristol Myers Squibb’s clinical development program for Breyanzi includes clinical studies in earlier lines of treatment for patients with relapsed or refractory LBCL and other types of lymphomas and leukemia. For more information, visit clinicaltrials.gov.
Breyanzi is not approved in any region for the second-line treatment of LBCL.
Please see full Prescribing Information, including Boxed WARNINGS and Medication Guide.
Learn more about the science behind cell therapy and ongoing research at Bristol Myers Squibb here.
For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.
Source: Bristol Myers Squibb
BMY Dividend History
https://www.nasdaq.com/market-activity/stocks/bmy/dividend-history
https://seekingalpha.com/symbol/BMY
CARVYKTI® (ciltacabtagene autoleucel)
CARVYKTI® (ciltacabtagene autoleucel) Granted Conditional Approval by the European Commission for the Treatment of Patients with Relapsed and Refractory Multiple Myeloma • CARVYKTI® is Legend Biotech’s first European Commission-approved product • The approval is based on the pivotal phase 1b/2 CARTITUDE-1 study, which demonstrated an overall response rate (ORR) of 98 percent in patients with relapsed or refractory multiple myeloma following a one-time treatment with ciltacabtagene autoleucel
SOMERSET, N.J.— (BUSINESS WIRE)—May 26, 2022—Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global biotechnology company developing, manufacturing and commercializing novel therapies to treat life-threatening diseases, today announced that the European Commission (EC) has granted conditional marketing authorization of CARVYKTI® (ciltacabtagene autoleucel; cilta-cel) for the treatment of adults with relapsed and refractory multiple myeloma (RRMM) who have received at least three prior therapies, including a proteasome inhibitor (PI), an immunomodulatory agent (IMiD) and an anti-CD38 antibody, and have demonstrated disease progression on the last therapy. Legend Biotech entered into an exclusive worldwide license and collaboration agreement with Janssen Biotech, Inc. (Janssen) to develop and commercialize cilta-cel in December 2017. CARVYKTI® is a chimeric antigen receptor T-cell (CAR-T) therapy featuring two B-cell maturation antigen (BCMA)-targeting single domain antibodies.1 CAR-T therapy is specifically developed for each individual patient, and it is administered as a single infusion. 1,2 “The approval of CARVYKTI® by the European Commission marks Legend’s first approval in the region and is a significant milestone as we advance our commitment to bring innovative cell therapies to patients with the highest unmet need,” said Ying Huang, Ph.D., Chief Executive Officer of Legend Biotech. “The CARTITUDE-1 data showed that CARVYKTI® provides the potential for long, treatment-free intervals for heavily pre-treated patients, and is a valuable treatment option for patients with multiple myeloma. We look forward to working with Janssen to bring this new option to patients across Europe.” This approval was supported by the pivotal CARTITUDE-1 study, including patients who had received a median of six prior treatment regimens (range, 3-18), and had previously received an IMiD, PI and anti-CD38 monoclonal antibody. 3 Findings showed that at a median duration of 18 months follow-up (range, 1.5-30.5), a one-time treatment with cilta-cel resulted in deep and durable responses, with 98 percent (95 percent confidence interval [CI], 92.7-99.7) of patients with RRMM responding to therapy (98 percent overall response rate [ORR] (N=97)). 3,4 Notably, 80 percent of the patients achieved stringent complete response (sCR), a measure in which a physician is unable to observe any signs or symptoms of disease via imaging or other tests after treatment. 3 The safety of cilta-cel was evaluated in 179 adult patients across two open-label clinical trials (MMY2001 and MMY2003). The most common adverse reactions (≥20 percent) were neutropenia (91 percent), cytokine release syndrome (CRS) (88 percent), pyrexia (88 percent), thrombocytopenia (73 percent), anemia (72 percent), leukopenia (54 percent), lymphopenia (45 percent), musculoskeletal pain (43 percent), hypotension (41 percent), fatigue (40 percent), transaminase elevation (37 percent), upper respiratory tract infection (32 percent), diarrhea (28 percent), hypocalcemia (27 percent), hypophosphatemia (26 percent), nausea (26 percent), headache (25 percent), cough (25 percent), tachycardia (23 percent), chills (23 percent), encephalopathy (22 percent), decreased appetite (22 percent), oedema (22 percent), and hypokalemia (20 percent).4 “There continues to be an unmet need for patients with relapsed or refractory multiple myeloma who have exhausted existing treatment options. Data from the CARTITUDE-1 trial have shown that cilta-cel provides deep and durable responses for heavily pre-treated patients. Today’s approval by the European Commission reinforces the potential of this new treatment option for multiple myeloma patients in Europe,” said Hermann Einsele, Full Professor of Internal Medicine and Director of the Medizinische Klinik und Poliklinik II of the Julius Maximilian University, Würzburg, Germany.* As a highly personalized medicine, where a patient’s own T-cells are reprogrammed to target and kill cancer cells, administration of CAR-T therapy requires extensive training, preparation, and certification to ensure the highest quality product and experience for patients.2 Through a phased approach, Legend Biotech’s strategic partner, Janssen, will work diligently to activate a limited network of certified treatment centers and will aim to increase availability of cilta-cel across Europe, in an effort to provide oncologists and patients with treatment in a reliable manner. This EC Marketing Authorization follows the approval of CARVYKTI® by the U.S. Food and Drug Administration (FDA) on February 28, 2022. *Hermann Einsele, M.D., FRCP has not been paid for contributing to this press release. ### About CARVYKTI® (ciltacabtagene autoleucel; cilta-cel) CARVYKTI® is a BCMA-directed, genetically modified autologous T-cell immunotherapy, which involves reprogramming a patient’s own T-cells with a transgene encoding a chimeric antigen receptor (CAR) that identifies and eliminates cells that express BCMA.4 BCMA is primarily expressed on the surface of malignant multiple myeloma B-lineage cells, as well as late-stage B-cells and plasma cells.4 The CARVYKTI® CAR protein features two BCMAtargeting single domain antibodies designed to confer high avidity against human BCMA.4 Upon binding to BCMA-expressing cells, the CAR promotes T-cell activation, expansion, and elimination of target cells.4 In December 2017, Legend Biotech Corporation entered into an exclusive worldwide license and collaboration agreement with Janssen Biotech, Inc. (Janssen) to develop and commercialize cilta-cel.5 In April 2021, Legend announced the submission of a Marketing Authorisation Application to the European Medicines Agency seeking approval of cilta-cel for the treatment of patients with relapsed or refractory multiple myeloma. In addition to U.S. Breakthrough Therapy Designation granted in December 2019, cilta-cel also received Breakthrough Therapy Designation in China in August 2020. In February 2019, cilta-cel received Orphan Drug Designation from the U.S. FDA from the European Commission in February 2020, and from the Pharmaceuticals and Medicinal Devices Agency (PMDA) in Japan in June 2020. In May 2022, the Committee for Orphan Medicinal Products recommended by consensus that the orphan designation for cilta-cel be maintained, on the basis of clinical data demonstrating improved and sustained complete response rates following treatment.6 Cilta-cel was approved the U.S. Food and Drug Administration (FDA) in February 2022.5 About CARTITUDE-1 CARTITUDE-1 (NCT03548207) 7 is an ongoing Phase 1b/2, open-label, single arm, multicenter trial evaluating cilta-cel for the treatment of adult patients with relapsed or refractory multiple myeloma, who previously received at least three prior lines of therapy including a proteasome inhibitor (PI), an immunomodulatory agent (IMiD) and an anti-CD38 monoclonal antibody. Of the 97 patients enrolled in the trial, 99 percent were refractory to the last line of treatment and 88 percent were triple-class refractory, meaning their cancer did not respond, or no longer responds, to an IMiD, a PI and an anti-CD38 monoclonal antibody.4
Learn more at www.legendbiotech.com and follow us on Twitter and LinkedIn.
Source: Legend Biotech
J&J, Legend Biotech's CARVYKTI gets conditional approval in EU to treat multiple myeloma
May 26, 2022 4:47 AM ET
Legend Biotech Corporation (LEGN), JNJ
By: Ravikash, SA News Editor
- The European Commission (EC) granted conditional marketing authorization to Johnson & Johnson (NYSE:JNJ) and Legend Biotech's (NASDAQ:LEGN) CAR-T therapy CARVYKTI to treat certain adults with mhttps://seekingalpha.com/news/3842943-jj-legend-biotechs-carvykti-gets-approval-in-eu-to-treat-multiple-myelomaultiple myeloma (MM).
- https://seekingalpha.com/symbol/LEGN
- https://seekingalpha.com/symbol/JNJ
- https://www.carvykti.com/
- https://legendbiotech.com/
Ervogastat/Clesacostat
Pfizer Granted FDA Fast Track Designation for Ervogastat/Clesacostat Combination for the Treatment of Non-Alcoholic Steatohepatitis (NASH)
Thursday, May 26, 2022 - 06:45am
NEW YORK--(BUSINESS WIRE)-- Pfizer Inc. (NYSE: PFE) today announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Pfizer’s investigational combination therapy for the treatment of non-alcoholic steatohepatitis (NASH) with liver fibrosis: ervogastat (PF-06865571, a diacylglycerol O-acyltransferase 2 inhibitor, or DGAT2i) and clesacostat (PF-05221304, an acetyl-CoA carboxylase inhibitor, or ACCi). Fast Track is a process designed to facilitate the development and expedite the review of new drugs and vaccines intended to treat or prevent serious conditions and address unmet medical need.
The FDA’s decision is informed by the results of Pfizer’s nonclinical studies and a Phase 2a clinical study of ervogastat/clesacostat, which showed that treatment with ervogastat/clesacostat reduced liver fat with a favorable safety and tolerability profile. These data were recently published in Nature Medicine.
“Receiving Fast Track designation from the FDA reinforces Pfizer’s belief in ervogastat/clesacostat as a potential treatment for NASH, a serious, progressive liver disease with no currently approved therapies,” said James Rusnak, M.D., Ph.D., Senior Vice President and Chief Development Officer, Internal Medicine and Hospital, Pfizer. “We are proud to be advancing this investigational combination as part of our goal to develop innovative medicines to address some of the world’s most widespread health challenges that affect millions of people—including diseases like NASH.”
Pfizer is currently studying ervogastat/clesacostat in an ongoing Phase 2 clinical trial evaluating the impact of treatment on resolution of NASH or improvement in liver fibrosis (NCT04321031), expected to complete in 2024. The results of this study, which also includes arms investigating ervogastat as monotherapy, will inform a potential Phase 3 development program.
About Ervogastat/Clesacostat
Diacylglycerol O-acyltransferase 2 (DGAT2) and acetyl-CoA carboxylase (ACC) are two key enzymes that regulate lipid metabolism. Inhibitors of ACC and DGAT2 have demonstrated the ability to lower liver fat in patients with non-alcoholic fatty liver disease (NAFLD).8 Pfizer believes that ervogastat/clesacostat, its investigational DGAT2i/ACCi combination therapy, has the potential to deliver direct improvements in inflammation and fibrosis.
In addition, to learn more, please visit us on www.Pfizer.com and follow us on Twitter at @Pfizer and @Pfizer News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220526005017/en/
Source: Pfizer Inc.
Pfizer's ervogastat/clesacostat combo gets FDA fast track status for liver disease
May 26, 2022 7:30 AM ET
By: Ravikash, SA News Editor1 Comment
- The U.S. Food and Drug Administration (FDA) granted fast track designation to Pfizer's (NYSE:PFE) combo therapy ervogastat and clesacostat to treat non-alcoholic steatohepatitis (NASH) with liver fibrosis.
- https://seekingalpha.com/news/3843014-pfizers-ervogastatclesacostat-combo-gets-fda-fast-track-status-for-liver-diseasebrosis.
- https://seekingalpha.com/symbol/PFE
- https://www.pfizer.com/
Polivy® (polatuzumab vedotin)
Roche’s Polivy combination approved by European Commission for people with previously untreated diffuse large B-cell lymphoma May 25, 2022
- First new treatment option in more than 20 years to show a clinically meaningful improvement in progression-free survival is approved for people with previously untreated diffuse large B-cell lymphoma (DLBCL)
- Approval is based on pivotal data from the phase III POLARIX study, where Polivy plus R-CHP significantly improved progression-free survival with comparable safety versus the standard of care, R-CHOP
- First-line treatment with Polivy plus R-CHP has the potential to reduce the burden on patients and healthcare systems, associated with disease progression¹
Basel, 25 May 2022 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the European Commission (EC) has granted approval of Polivy® (polatuzumab vedotin) in combination with MabThera®(rituximab) plus cyclophosphamide, doxorubicin, and prednisone (R-CHP) for the treatment of adult patients with previously untreated diffuse large B-cell lymphoma (DLBCL).
DLBCL is an aggressive blood cancer and the most common form of non-Hodgkin lymphoma.² Each year in Europe, it is estimated that 40,000 people are diagnosed with DLBCL.3 While many patients are responsive to initial treatment, four out of ten are not cured with the current standard of care, and the majority of people who require subsequent lines of therapy have poor outcomes.⁴˒⁵ Most DLBCL relapses occur within 24 months of starting treatment and patients who remain progression-free 24 months following initiation of first-line therapy have favourable survival outcomes.⁴
“After more than 20 years with very limited treatment advances, the approval of Polivy plus R-CHP marks a new era for people battling this aggressive disease,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. “We are delighted that the European Commission has approved this Polivy regimen and believe it has the potential to make a significant impact on the lives of people with DLBCL.”
“The approval of Polivy plus R-CHP in the first-line setting is great news for people in the EU diagnosed with this aggressive lymphoma, giving them a greater opportunity for positive outcomes,” said Professor Hervé Tilly, POLARIX Principal Investigator and Professor of Haematology at the University of Rouen. “Treatment with this regimen has been shown to reduce the chance of relapse and therefore need for subsequent treatments, limiting the burden of DLBCL.”
Today’s EC approval was based on results from the phase III POLARIX study (GO39942), the first trial to show a clinically meaningful improvement in progression free survival (PFS), compared to standard of care rituximab plus cyclophosphamide, doxorubicin, vincristine and prednisone (R-CHOP), in people with previously untreated DLBCL. All patients were followed for at least 24 months and at a median follow-up of 28.2 months, results of the study showed a 27% reduction in the risk of disease worsening or death with Polivy plus R-CHP compared to R-CHOP in first-line DLBCL (hazard ratio [HR] 0.73; 95% confidence interval [CI]: 0.57–0.95; P<0.0177).⁶ The safety profile was comparable for Polivy plus R-CHP versus R-CHOP. The most frequently-reported (≥ 30%) adverse events with Polivy plus R-CHP were peripheral neuropathy (52.9%), nausea (41.6%), neutropenia (38.4%), and diarrhoea (30.8%).⁶ Results were presented for the first time in December 2021 at the 63rd American Society of Hematology Annual Meeting & Exposition and simultaneously published in the New England Journal of Medicine. The POLARIX study is being conducted in collaboration with The Lymphoma Study Association (LYSA) and The Lymphoma Academic Research Organisation (LYSARC).
In addition to today’s approval, the EC also converted Polivy's initial conditional marketing authorisation in the EU for the treatment of adult patients with relapsed or refractory DLBCL, who are not candidates for a haematopoietic stem cell transplant, to a full approval.
Roche continues to explore areas where Polivy has the potential to deliver additional benefit, including ongoing studies investigating combinations of Polivy with CD20xCD3 T-cell engaging bispecific antibodies in previously treated/untreated DLBCL.
About Polivy® (polatuzumab vedotin)
Polivy is a first-in-class anti-CD79b antibody-drug conjugate (ADC). The CD79b protein is expressed specifically in the majority of B-cells, an immune cell impacted in some types of non-Hodgkin lymphoma (NHL), making it a promising target for the development of new therapies.⁷˒⁸ Polivy binds to cancer cells such as CD79b and destroys these B-cells through the delivery of an anti-cancer agent, which is thought to minimise the effects on normal cells.⁹˒¹⁰ Polivy is being developed by Roche using Seagen ADC technology and is currently being investigated for the treatment of several types of NHL. Polivy is currently marketed in the EU for the treatment of relapsed or refractory diffuse large B-cell lymphoma.
About the POLARIX study
POLARIX [NCT03274492] is an international phase III, randomised, double-blind, placebo-controlled study evaluating the efficacy, safety and pharmacokinetics of Polivy® (polatuzumab vedotin) plus MabThera® (rituximab), cyclophosphamide, doxorubicin, and prednisone (R-CHP) versus rituximab, cyclophosphamide, doxorubicin, vincristine and prednisone (R-CHOP) in people with previously untreated diffuse large B-cell lymphoma. Eight-hundred and seventy-nine patients were randomised 1:1 to receive either Polivy plus R-CHP plus a vincristine placebo for six cycles, followed by rituximab for two cycles; or R-CHOP plus a Polivy placebo for six cycles, followed by two cycles of rituximab. The primary outcome measure is progression-free survival (PFS) as assessed by the investigator using the Lugano Response Criteria for malignant lymphoma. PFS is a clinically meaningful disease-related outcome for patients with previously untreated DLBCL as it represents the goal of first-line therapy: decreasing the risk of disease worsening.
For more information, please visit www.roche.com.
Roche wins EC approval of Polivy as first-line treatment for large B-cell lymphoma
May 25, 2022 12:30 PM ET
By: Jonathan Block, SA News Editor
- The European Commission approved Roche's (OTCQX:RHHBY) Polivy (polatuzumab vedotin) in combination with rituximab and other cancer drugs for diffuse large B-cell lymphoma.
- https://seekingalpha.com/news/3842713-roche-wins-ec-approval-of-polivy-as-first-line-treatment-for-diffuse-large-b-cell-lymphoma
- https://seekingalpha.com/symbol/RHHBY
- https://www.polivy.com/content/polivy/en_us/patient.html
- https://www.roche.com/
KEYTRUDA® (pembrolizumab) Plus Chemotherapy
European Commission Approves KEYTRUDA® (pembrolizumab) Plus Chemotherapy as Neoadjuvant Treatment, Then Continued as Adjuvant Monotherapy After Surgery for Locally Advanced or Early-Stage Triple-Negative Breast Cancer at High Risk of Recurrence
May 24, 2022 6:45 am ET
Approval based on event-free survival benefit demonstrated in Phase 3 KEYNOTE-522 trial
This KEYTRUDA combination is the first immunotherapy option approved in the EU for high-risk early-stage TNBC
Decision marks fifth approval for KEYTRUDA in a breast or gynecologic cancer in the EU in less than one year
RAHWAY, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced that the European Commission has approved KEYTRUDA, Merck’s anti-PD-1 therapy, in combination with chemotherapy as neoadjuvant treatment, and then continued as monotherapy as adjuvant treatment after surgery for adults with locally advanced or early-stage triple-negative breast cancer (TNBC) at high risk of recurrence.
The approval is based on results from the pivotal Phase 3 KEYNOTE-522 trial, in which KEYTRUDA in combination with chemotherapy before surgery and continued as a single agent after surgery prolonged event-free survival (EFS), reducing the risk of EFS events or death by 37% (HR=0.63 [95% CI, 0.48-0.82]; p=0.00031) compared to neoadjuvant chemotherapy alone in this patient population. Median follow-up time for all patients was 37.8 months (range, 2.7-48).
KEYNOTE-522 was the first large, randomized Phase 3 study to report a statistically significant and clinically meaningful EFS result among patients with stage II and III TNBC. With this decision, this KEYTRUDA combination becomes the first immunotherapy option approved for patients in the European Union (EU) in this setting.
“Triple-negative breast cancer has a high risk of recurrence within the first five years, so it’s meaningful for patients to have access to new therapies that can reduce the risk of disease progression,” said Dr. Peter Schmid, lead, Centre for Experimental Cancer Medicine, Barts Cancer Institute in London, England. “The approval of this KEYTRUDA regimen marks a turning point for patients with high-risk early-stage TNBC, as they now have an immunotherapy option in early stages of the disease that has demonstrated significant improvements in pathological complete response and event-free survival compared to neoadjuvant chemotherapy.”
The safety of KEYTRUDA plus chemotherapy has been evaluated in 3,123 patients across tumor types. The incidence of Grade 3-5 adverse reactions in patients with TNBC was 80% for KEYTRUDA plus chemotherapy and 77% for chemotherapy.
“KEYTRUDA was first approved in Europe to address an unmet need in certain patients with metastatic TNBC, and today’s approval extends the use of KEYTRUDA to more patients facing this difficult-to-treat cancer – this time in earlier stages of TNBC,” said Dr. Gursel Aktan, vice president, global clinical development, Merck Research Laboratories. “We are very proud that today’s approval marks the fifth indication for KEYTRUDA in Europe for patients with breast or a gynecological cancer, an important area where patients need continued research and innovation.”
This approval allows marketing of this KEYTRUDA regimen in all 27 EU member states plus Iceland, Lichtenstein, Norway and Northern Ireland. This is the second indication for KEYTRUDA in breast cancer in Europe. In October 2021, KEYTRUDA plus chemotherapy was approved for the first-line treatment of certain patients with locally recurrent unresectable or metastatic TNBC.
Merck is committed to delivering meaningful advances in gynecologic and breast cancers to patients around the world through its extensive clinical development program across its oncology portfolio of investigational and approved medicines. Within just the last year, KEYTRUDA has been approved in Europe for five new indications across breast, cervical and endometrial cancers as monotherapy and in novel combinations.
About KEYNOTE-522
The approval was based on data from KEYNOTE-522 (ClinicalTrials.gov, NCT03036488), a randomized, double-blind Phase 3 trial. The dual primary endpoints were pathological complete response rate, defined as pathological stage ypT0/Tis ypN0 at the time of definitive surgery, and EFS, defined as the time from randomization to the time of first occurrence of either disease progression that precluded definitive surgery, a local/distant recurrence, a second primary cancer or death from any cause. A key secondary endpoint was overall survival. The study enrolled 1,174 patients who were randomized 2:1 to receive either:
- The KEYTRUDA regimen: KEYTRUDA plus chemotherapy (paclitaxel and carboplatin), followed by KEYTRUDA plus chemotherapy (cyclophosphamide and either doxorubicin or epirubicin) as neoadjuvant therapy prior to surgery, followed by KEYTRUDA monotherapy as adjuvant therapy post-surgery (n=784) or;
- The chemotherapy-placebo regimen: Placebo plus chemotherapy (paclitaxel and carboplatin), followed by placebo plus chemotherapy (cyclophosphamide and either doxorubicin or epirubicin) as neoadjuvant therapy prior to surgery, followed by placebo monotherapy as adjuvant therapy post-surgery (n=390).
About KEYTRUDA® (pembrolizumab) injection, 100 mg
KEYTRUDA is an anti-programmed death receptor-1 (PD-1) therapy that works by increasing the ability of the body’s immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.
Merck has the industry’s largest immuno-oncology clinical research program. There are currently more than 1,700 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.
Selected KEYTRUDA® (pembrolizumab) Indications in the U.S.
Melanoma
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.
KEYTRUDA is indicated for the adjuvant treatment of adult and pediatric (12 years and older) patients with stage IIB, IIC, or III melanoma following complete resection.
Non-Small Cell Lung Cancer
KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) ≥1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is:
- stage III where patients are not candidates for surgical resection or definitive chemoradiation, or
- metastatic.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS ≥1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.
Head and Neck Squamous Cell Cancer
KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [Combined Positive Score (CPS) ≥1] as determined by an FDA-approved test.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.
Classical Hodgkin Lymphoma
KEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).
KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.
Primary Mediastinal Large B-Cell Lymphoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy.
KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.
Urothelial Carcinoma
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC):
- who are not eligible for any platinum-containing chemotherapy, or
- who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
Non-muscle Invasive Bladder Cancer
KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.
Microsatellite Instability-High or Mismatch Repair Deficient Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options.
This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.
Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).
Gastric Cancer
KEYTRUDA, in combination with trastuzumab, fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of patients with locally advanced unresectable or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma.
This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Esophageal Cancer
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic esophageal or gastroesophageal junction (GEJ) (tumors with epicenter 1 to 5 centimeters above the GEJ) carcinoma that is not amenable to surgical resection or definitive chemoradiation either:
- in combination with platinum- and fluoropyrimidine-based chemotherapy, or
- as a single agent after one or more prior lines of systemic therapy for patients with tumors of squamous cell histology that express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
Cervical Cancer
KEYTRUDA, in combination with chemotherapy, with or without bevacizumab, is indicated for the treatment of patients with persistent, recurrent, or metastatic cervical cancer whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test.
Hepatocellular Carcinoma
KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Merkel Cell Carcinoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Renal Cell Carcinoma
KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of adult patients with advanced renal cell carcinoma (RCC).
KEYTRUDA is indicated for the adjuvant treatment of patients with RCC at intermediate-high or high risk of recurrence following nephrectomy, or following nephrectomy and resection of metastatic lesions.
Endometrial Carcinoma
KEYTRUDA, as a single agent, is indicated for the treatment of patients with advanced endometrial carcinoma that is MSI-H or dMMR, as determined by an FDA-approved test, who have disease progression following prior systemic therapy in any setting and are not candidates for curative surgery or radiation.
Tumor Mutational Burden-High Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [≥10 mutations/megabase] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.
Cutaneous Squamous Cell Carcinoma
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) or locally advanced cSCC that is not curable by surgery or radiation.
Triple-Negative Breast Cancer
KEYTRUDA is indicated for the treatment of patients with high-risk early-stage triple-negative breast cancer (TNBC) in combination with chemotherapy as neoadjuvant treatment, and then continued as a single agent as adjuvant treatment after surgery.
KEYTRUDA, in combination with chemotherapy, is indicated for the treatment of patients with locally recurrent unresectable or metastatic TNBC whose tumors express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
For more information, visit www.merck.com and connect with us on Twitter, Facebook, Instagram, YouTube and LinkedIn.
Please see Prescribing Information for KEYTRUDA (pembrolizumab) at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_pi.pdf and Medication Guide for KEYTRUDA at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_mg.pdf .
Source: Merck & Co., Inc.
Merck's blockbuster drug Keytruda gets approval in EU for expanded use in breast cancer subtype
May 24, 2022 7:07 AM ET
By: Ravikash, SA News Editor
- The European Commission approved Merck's (NYSE:MRK) blockbuster drug Keytruda to treat certain patients with triple-negative breast cancer (TNBC).
- https://seekingalpha.com/news/3842105-mercks-blockbuster-drug-keytruda-gets-approval-in-eu-for-expanded-use-in-breast-cancer-subtype
- https://seekingalpha.com/symbol/MRK
- https://www.keytruda.com/
- https://www.merck.com/
Protein Oral Delivery (POD™)
Oramed Granted U.S. Combination Therapy Patent for Oral GLP-1 & Insulin for the Treatment of Diabetes
NEW YORK, May 24, 2022 /PRNewswire/ — Oramed Pharmaceuticals Inc. (Nasdaq: ORMP) (TASE: ORMP) (www.oramed.com), a clinical-stage pharmaceutical company focused on the development of oral drug delivery platforms, today announced that the United States Patent and Trademark Office has granted the Company a patent titled “Methods and Compositions for Treating Diabetes.” The patent addresses methods and compositions for treating diabetes mellitus through oral pharmaceutical compositions comprising insulin in combination with Glucagon-like Peptide 1 (GLP-1).
About Oramed Pharmaceuticals
Oramed Pharmaceuticals (Nasdaq/TASE: ORMP) is a platform technology pioneer in the field of oral delivery solutions for drugs currently delivered via injection. Established in 2006, with offices in the United States and Israel, Oramed has developed a novel Protein Oral Delivery (POD™) technology. Oramed is seeking to transform the treatment of diabetes through its proprietary lead candidate, ORMD-0801, which is being evaluated in two pivotal Phase 3 studies and has the potential to be the first commercial oral insulin capsule for the treatment of diabetes. In addition, Oramed is developing an oral GLP-1 (Glucagon-like peptide-1) analog capsule (ORMD-0901).
For more information, please visit www.oramed.com.
View original content:https://www.prnewswire.com/news-releases/oramed-granted-us-combination-therapy-patent-for-oral-glp-1–insulin-for-the-treatment-of-diabetes-301553700.html
SOURCE Oramed Pharmaceuticals Inc.
Oramed granted U.S. combination therapy patent for oral insulin
May 24, 2022 9:37 AM ET
Oramed Pharmaceuticals Inc. (ORMP)
By: Dania Nadeem, SA News Editor
- Oramed Pharma (NASDAQ:ORMP) said on Tuesday that the United States Patent and Trademark Office had granted a patent for methods and compositions for treating diabetes.
- https://seekingalpha.com/news/3842224-oramed-granted-us-combination-therapy-patent-for-oral-insulin
- https://seekingalpha.com/symbol/ORMP
Etrasimod
Pfizer Presents ELEVATE Pivotal Findings Demonstrating Etrasimod’s Potentially Best-in-Class Profile in Ulcerative Colitis
Tuesday, May 24, 2022 - 06:45am
- Analyst call will be held to discuss data today at 4:00 PM Eastern Time
NEW YORK--(BUSINESS WIRE)-- Pfizer Inc. (NYSE: PFE) today announced detailed results from two pivotal studies that make up the ELEVATE UC Phase 3 registrational program evaluating etrasimod, a once-daily, oral, selective sphingosine 1-phosphate (S1P) receptor modulator for the treatment of moderately-to-severely active ulcerative colitis (UC). These data were presented as a late-breaker presentation (abstract number 968a) at Digestive Disease Week (DDW) 2022.
Both Phase 3, multi-center, randomized, placebo-controlled trials achieved all primary and key secondary endpoints, with etrasimod demonstrating a safety profile consistent with previous studies. In the 52-week ELEVATE UC 52 study, clinical remission was 27.0% for patients receiving etrasimod compared to 7.4% for patients receiving placebo at week 12 (19.8% differential, P=˂.001) and was 32.1% compared to 6.7% at week 52 (25.4% differential, P=˂.001). In the 12-week ELEVATE UC 12 study, clinical remission was achieved among 24.8% of patients receiving etrasimod compared to 15.2% of patients receiving placebo (9.7% differential, P=.0264).
UC is a chronic and often debilitating inflammatory bowel disease1 that affects an estimated 3.8 million people in North America and Europe.2 Symptoms of UC can include chronic diarrhea with blood and mucus, abdominal pain and urgency.3,4 UC can have a significant effect on work, family and social activities.4
“Etrasimod could offer a differentiated clinical profile for people living with moderately-to-severely active ulcerative colitis considering the clear benefit it has shown over 52 weeks in a treat-through trial design, its mechanism of action, and its unique pharmacologic properties,” said Michael Corbo, Chief Development Officer, Inflammation & Immunology, Pfizer Global Product Development. “Patients often need multiple options to help manage their disease and there is a significant need for new therapies. In the ELEVATE clinical program, etrasimod has shown an encouraging balance of efficacy and safety that we believe could have a meaningful impact for patients and physicians, if approved.”
The 52-week ELEVATE UC 52 trial utilized a treat-through design which closely mimics real-world clinical practice. Statistically significant improvements were attained in all key secondary endpoints in ELEVATE UC 52. These included endoscopic improvement, symptomatic remission, and mucosal healing at weeks 12 and 52, and corticosteroid-free remission and sustained clinical remission at week 52. All key secondary endpoints were also met at week 12 in ELEVATE UC 12. These included endoscopic improvement, symptomatic remission, and mucosal healing.
Treatment-emergent adverse events (AEs), including serious AEs, were similar between treatment groups in both trials. The most common treatment-emergent AEs in 3% or more of etrasimod-treated patients and greater than placebo up to week 52 in either trial were headache, worsening of UC, COVID-19 infection, dizziness, pyrexia, arthralgia, abdominal pain and nausea. There were no reports of bradycardia or atrioventricular block as serious AEs.
The data from ELEVATE UC 52 & UC 12 are expected to form the basis for planned future regulatory filings, which will be initiated later this year. Additional information about the studies can be found at www.clinicaltrials.gov under the identifiers NCT03945188, NCT03996369, and NCT03950232.
Etrasimod was developed by Arena Pharmaceuticals, which was recently acquired by Pfizer.
Pfizer Conference Call
Pfizer Inc. invites Pfizer investors and the general public to view and listen to a webcast of a live conference call with investment analysts at 4 p.m. ET on May 24.
To view and listen to the webcast visit Pfizer’s website at www.pfizer.com/investors or directly at https://pfizer.rev.vbrick.com/#/events/333186b6-252b-4c60-be45-947638abd93f. Information on accessing and pre-registering for the webcast will be available at www.pfizer.com/investors beginning today. Participants are advised to pre-register in advance of the conference call.
You can listen to the conference call by dialing either (833) 708-1779 in the United States or Canada or (602) 585-9859 outside of the United States and Canada. The password is “9991403.” Please join the call five minutes prior to the start time to avoid operator hold times.
The transcript and webcast replay of the call will be made available on Pfizer’s website at www.pfizer.com/investors within 24 hours after the end of the live conference call and will be accessible for at least 90 days.
About Etrasimod
Etrasimod is an oral, once-a-day, selective sphingosine 1-phosphate (S1P) receptor modulator designed for optimized pharmacology and engagement of S1P receptors 1, 4, and 5. It is being investigated for a range of immuno-inflammatory diseases, including ulcerative colitis, Crohn’s disease, atopic dermatitis, eosinophilic esophagitis, and alopecia areata.
About ELEVATE UC 52 and ELEVATE UC 12
ELEVATE UC 52 and ELEVATE UC 12 are pivotal trials that are part of the ELEVATE UC Phase 3 registrational program.
ELEVATE UC 52 is a randomized, double-blind, placebo-controlled trial that utilized a treat-through design. The primary objective of this trial was to assess the safety and efficacy of etrasimod 2 mg once-daily on clinical remission after both 12 and 52 weeks. All patients that dropped out of the study across either treatment arm over the 52-week study was counted as a non-responder. The primary endpoint is based on the 3-domain, modified Mayo score (MMS). Key secondary measures included endoscopic improvement, symptomatic remission, and mucosal healing at weeks 12 and 52, and corticosteroid free remission and sustained clinical remission at week 52.
ELEVATE UC 12 is a randomized, double-blind, placebo-controlled trial to assess the efficacy and safety of etrasimod 2 mg once-daily in subjects with moderately-to-severely active UC. The primary objective of this trial was to assess the safety and efficacy of etrasimod on clinical remission at 12 weeks assessed by the FDA-required, 3-domain, modified Mayo score. Key secondary measures included endoscopic improvement, symptomatic remission, and mucosal healing.
62.6% of etrasimod-treated patients in both trials and 61.8% and 62.9% of placebo-treated patients in ELEVATE UC 52 and ELEVATE UC 12, respectively, were naïve to biologic or JAK inhibitor therapy.
In addition, to learn more, please visit us on www.pfizer.com and follow us on Twitter at @Pfizer and @Pfizer_News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220524005178/en/
Media Contact:
+1 (212) 733-1226
PfizerMediaRelations@Pfizer.com
Source: Pfizer Inc.
Pfizer presents additional late-stage results supporting etrasimod for ulcerative colitis
May 24, 2022 10:02 AM ET
By: Jonathan Block, SA News Editor2 Comments
- More results from a phase 3 trial of Pfizer's (NYSE:PFE) ulcerative colitis candidate etrasimod confirmed that all primary endpoints were met.
- https://seekingalpha.com/news/3842213-pfizer-presents-additional-late-stage-data-supporting-etrasimod-for-ulcerative-colitis
- https://seekingalpha.com/symbol/PFE
biotecMAX™ Feb/Mar/APR/May 2022 Insight
TUKYSA® (tucatinib) in Combination With Trastuzumab
Seagen Announces Positive Topline Results of Pivotal Phase 2 Clinical Trial of TUKYSA® (tucatinib) in Combination With Trastuzumab in HER2-Positive Metastatic Colorectal Cancer
05/23/2022
- Data Accepted for Presentation at ESMO World Congress on Gastrointestinal Cancer -
BOTHELL, Wash.--(BUSINESS WIRE)-- Seagen Inc. (Nasdaq:SGEN) today announced positive topline results from the pivotal phase 2 MOUNTAINEER clinical trial investigating TUKYSA® (tucatinib) in combination with trastuzumab in patients with previously treated HER2-positive metastatic colorectal cancer (mCRC). Data from this trial will form the basis of a planned supplemental New Drug Application to the U.S. Food and Drug Administration (FDA) under the FDA’s Accelerated Approval Program. Merck, known as MSD outside the U.S. and Canada, is commercializing TUKYSA in regions outside of the U.S., Canada and Europe and plans to discuss these results with health authorities as it continues to accelerate the filing of TUKYSA in its territory.
Results showed a 38.1% confirmed objective response rate (cORR) [95% Confidence Interval (CI): 27.7, 49.3] per blinded independent central review (BICR). The median duration of response (DoR) per BICR was 12.4 months [95% CI: 8.5, 20.5]. The combination of tucatinib and trastuzumab was generally well-tolerated, and the most common (greater than or equal to 20%) treatment-emergent adverse events were diarrhea, fatigue, nausea and infusion-related reaction, which were primarily low-grade.
Please see Important Safety Information at the end of this press release for further safety information regarding tucatinib.
“People with HER2-positive previously treated metastatic colorectal cancer have a significant unmet need for new therapies. We are excited by the potential for this tucatinib combination to help patients based on the excellent anti-tumor activity with durable responses and a tolerable safety profile,” said Roger Dansey, M.D., interim CEO and Chief Medical Officer, Seagen. “Based on the strength of these data, we are planning to engage in regulatory discussions with the FDA with the intent to submit a supplemental New Drug Application for TUKYSA.”
Full data from the MOUNTAINEER trial will be presented by John H. Strickler, M.D., Duke University Medical Center, at the European Society for Medical Oncology (ESMO) World Congress on Gastrointestinal Cancer in Barcelona, Spain from June 29 through July 2, 2022.
About MOUNTAINEER
MOUNTAINEER is a U.S. and European multicenter, open-label, phase 2 clinical trial of tucatinib in combination with trastuzumab or as a single agent in 117 patients with HER2-positive metastatic or unresectable colorectal cancer following previous standard-of-care therapies. The primary endpoint of the trial is confirmed objective response rate by RECIST (Response Evaluation Criteria in Solid Tumors) version 1.1 criteria per blinded independent central review in patients receiving the combination of tucatinib and trastuzumab. Duration of response, progression-free survival, overall survival and safety and tolerability of the combination regimen are secondary objectives.
About TUKYSA (tucatinib)
TUKYSA is an oral medicine that is a tyrosine kinase inhibitor of the HER2 protein. In vitro (in lab studies), TUKYSA inhibited phosphorylation of HER2 and HER3, resulting in inhibition of downstream MAPK and AKT signaling and cell growth (proliferation), and showed anti-tumor activity in HER2-expressing tumor cells. In vivo (in living organisms), TUKYSA inhibited the growth of HER2-expressing tumors. The combination of TUKYSA and the anti-HER2 antibody trastuzumab showed increased anti-tumor activity in vitro and in vivo compared to either medicine alone.
TUKYSA is approved in 36 countries. It was approved by the U.S. FDA in April 2020 and by the European Medicines Agency and the UK Medicines and Healthcare Products Regulatory Agency in February 2021. Merck, known as MSD outside the U.S. and Canada, has exclusive rights to commercialize TUKYSA in Asia, the Middle East and Latin America and other regions outside of the U.S., Canada and Europe.
U.S. Indication and Important Safety Information
TUKYSA is indicated in combination with trastuzumab and capecitabine for treatment of adult patients with advanced unresectable or metastatic HER2-positive breast cancer, including patients with brain metastases, who have received one or more prior anti-HER2-based regimens in the metastatic setting
For more information on the company’s marketed products and robust pipeline, visit www.seagen.com and follow @SeagenGlobal on Twitter.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220523005352/en/
Source: Seagen Inc.
Seagen's Tukysa shows efficacy for colorectal cancer in pivotal trial
May 23, 2022 9:09 AM ETSeagen Inc. (SGEN)
By: Jonathan Block, SA News Editor1 Comment
- Topline data from a phase 2 trial of Seagen's (NASDAQ:SGEN) Tukysa (tucatinib) showed that the oncologic demonstrated a good objective response rate and duration of response in patients with HER-2 positive colorectal cancer.
- https://seekingalpha.com/news/3841697-seagen-tukysa-shows-efficacy-for-colorectal-cancer-in-pivotal-trial
- https://seekingalpha.com/symbol/SGEN
- https://www.tukysa.com/
- https://www.seagen.com/
OXLUMO® (lumasiran)
Alnylam Presents New Results from the ILLUMINATE-C Phase 3 Study of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1
May 24, 2022
– Lumasiran Demonstrated Evidence of Improvements in Exploratory Endpoints of Systemic Oxalosis, Including Echocardiographic Structure and Function, Nephrocalcinosis, and Kidney Stone Events –
– Expands Upon Previously Reported Reductions in Plasma Oxalate in PH1 Patients with Severe Renal Impairment –
CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 24, 2022-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced new positive results from the six-month primary analysis period of the ILLUMINATE-C Phase 3 open-label study of lumasiran, an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – that is being investigated for the treatment of patients of all ages with advanced primary hyperoxaluria type 1 (PH1). As previously reported, treatment with lumasiran resulted in substantial reductions in plasma oxalate (POx) at six months. Results from new exploratory analyses provide evidence that lumasiran treatment resulted in improvements in cardiac measures, nephrocalcinosis and kidney stone events. In addition, some patients treated with lumasiran experienced improvements in their most burdensome symptoms (as assessed by the investigator), including fatigue, nausea, and bone pain, with no patients reporting worsening of those symptoms. These data were presented at the European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) International Congress being held both in Paris, France, and as a virtual event on May 19-22.
“Patients with compromised renal function due to advanced PH1 tend to have elevated levels of plasma oxalate, which can lead to systemic spread of oxalate to organs beyond the kidneys, including the heart,” said Professor Jaap Groothoff, M.D., Ph.D., Head of the Department of Pediatric Nephrology, Emma Children’s Hospital, Amsterdam UMC, University of Amsterdam, The Netherlands. “The ILLUMINATE-C study of lumasiran shows impressive plasma oxalate lowering in patients with severely impaired kidney function or who are on dialysis. These data, along with previous findings from ILLUMINATE-A and ILLUMINATE-B Phase 3 studies, provide evidence supporting the potential of lumasiran to benefit not only these patients, but those across the full spectrum of PH1 severity.”
“We are thrilled to be presenting preliminary data on clinical outcomes from our ILLUMINATE-C study. We believe the reductions in plasma oxalate seen with lumasiran in this study, along with this newly reported evidence of improvements in exploratory endpoints of cardiac measures, kidney stone events and nephrocalcinosis, are encouraging and signal the potential to establish lumasiran as a therapeutic option across the spectrum of PH1 disease severity,” said Pushkal Garg, M.D., Chief Medical Officer at Alnylam. “We look forward to further analyzing these endpoints over a longer period of time in the ongoing extension period of up to 54 months to continue to assess the impact of treatment with lumasiran on long-term clinical outcomes, including the manifestations of systemic oxalosis.”
Results
ILLUMINATE-C enrolled a total of 21 patients, including six who were not on dialysis at study start (Cohort A) and 15 who were on hemodialysis (Cohort B). As previously presented at the American Society of Nephrology (ASN) Kidney Week Meeting in 2021, in Cohorts A and B, respectively, treatment with lumasiran led to 33 percent and 42 percent mean reductions in POx from baseline to Month 6. Additionally, treatment with lumasiran led to 35 µmol/L (Cohort A) and 48 µmol/L (Cohort B) mean absolute reductions in POx from baseline to Month 6.
In new exploratory analyses, left ventricular ejection fraction (LVEF) and global longitudinal strain (GLS) were measured to assess the impact of lumasiran on the cardiac manifestations of systemic oxalosis. Among patients with abnormal LVEFi at baseline across Cohort A (N=1) and Cohort B (N=4), the patient in Cohort A and two of four patients in Cohort B experienced a ≥ 5 percent improvement following six months of treatment with lumasiran. Among patients with abnormal GLSii at baseline across Cohort A (N=1) and Cohort B (N=3), all four patients showed a ≥2 percent improvement at Month 6.
In patients with medullary nephrocalcinosis at baseline in Cohort A (N=5), two patients remained stable, three improved (two unilateral and one bilateral improvement) and none worsened following six months of treatment with lumasiran. In Cohort A, one patient did not have nephrocalcinosis at baseline and had bilateral worsening at six months. In patients with nephrocalcinosis at baseline in Cohort B (N=2), both improved (one unilateral and one bilateral improvement) following six months of treatment with lumasiran.
In Cohort A, the rate of kidney stone events per person-year decreased from 3.2 (95% CI: 2, 5.2) in the 12 months prior to consent to 1.5 (95% CI: 0.6, 3.9) during the first six months of treatment with lumasiran. As expected, given patients in Cohort B are on hemodialysis, the rate of kidney stone events was low: 0.07 (95% CI: 0.01, 0.71) in the 12 months prior to consent and 0.0 (95% CI: 0.0, 0.53) during the first six months of treatment with lumasiran. For patients in both cohorts, the most burdensome symptoms at baseline (as assessed by the investigator), including fatigue, nausea/decreased appetite, bone pain and decreased mobility, improved or remained stable following six months of treatment with lumasiran; none of the symptoms worsened.
The most common adverse event related to lumasiran was injection-site reaction (ISR) reported in 5 of 21 patients (24 percent). All ISRs were mild and transient, and the most common symptoms included erythema, discoloration, and hematoma.
A separate analysis presented at ERA-EDTA demonstrated that the recommended weight-based dosing regimens of lumasiran showed equivalent efficacy in patients of all ages and degrees of kidney function, including patients on hemodialysis.
To view all results presented by Alnylam at ERA-EDTA please visit www.alnylam.com/capella.
OXLUMO® (lumasiran) INDICATION AND IMPORTANT SAFETY INFORMATION
Indication
OXLUMO is indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.
For additional information about OXLUMO, please see the full U.S. Prescribing Information.
About Lumasiran
Lumasiran is a subcutaneously administered RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) in development for the treatment of advanced primary hyperoxaluria type 1 (PH1). HAO1 encodes glycolate oxidase (GO). Thus, by silencing HAO1 and depleting the GO enzyme, lumasiran inhibits production of oxalate – the metabolite that directly contributes to the pathophysiology of PH1. Lumasiran utilizes Alnylam's Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate technology, which enables subcutaneous dosing with increased potency and durability and a wide therapeutic index. Lumasiran has received regulatory approvals from the U.S. Food and Drug Administration (FDA) for the treatment of PH1 to lower urinary oxalate levels in pediatric and adult patients and from the European Medicines Agency (EMA) for the treatment of PH1 in all age groups under the brand name OXLUMO®.
About ILLUMINATE-C Phase 3 Study
ILLUMINATE-C (NCT04152200) is a single arm, open-label, multinational Phase 3 study evaluating the safety and efficacy of lumasiran in PH1 patients of all ages with severe renal impairment (eGFR ≤ 45 mL/min/1.73m2 or elevated serum creatinine for patients <12 months of age). The study is being conducted at 13 study sites across 10 countries around the world. Cohort A enrolled six patients with advanced PH1 who do not yet require dialysis, and Cohort B enrolled 15 patients who are hemodialysis-dependent. The dosing regimen is based on weight with three monthly starting doses followed by ongoing monthly or quarterly doses. The primary efficacy endpoint for Cohort A was the percent change in plasma oxalate from baseline to month six, and the primary endpoint for Cohort B was the percent change in pre-dialysis plasma oxalate from baseline to month six. Key secondary endpoints are designed to evaluate additional measures of plasma oxalate and changes in urinary oxalate. Kidney function, frequency and mode of dialysis, frequency of kidney stone events, and measures of systemic oxalosis, including clinical manifestations, will also be evaluated in the extension period of the study.
For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on Twitter at @Alnylam, on LinkedIn, or on Instagram.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220524005288/en/
Alnylam Pharmaceuticals, Inc.
Source: Alnylam Pharmaceuticals, Inc.
Alnylam kidney disorder therapy Oxlumo improves cardiac measures in new data from phase 3 trial
May 24, 2022 8:20 AM ET
Alnylam Pharmaceuticals, Inc. (ALNY)
By: Ravikash, SA News Editor
Alnylam Pharmaceuticals (NASDAQ:ALNY) said new analysis from a late-stage study of its drug Oxlumo (lumasiran) showed that the drug helped improve cardiac measures, calcium deposit levels and kidney stone events in patients with advanced primary hyperoxaluria type 1 (PH1).
https://seekingalpha.com/symbol/ALNY
Mirikizumab
Fifty Percent of Patients with Ulcerative Colitis Treated with Mirikizumab Achieved Clinical Remission at One Year in Lilly's Pivotal Phase 3 Study
May 24, 2022Download PDF
Nearly all mirikizumab-treated patients who achieved clinical remission at one year were not taking steroids
INDIANAPOLIS, May 24, 2022 /PRNewswire/ -- In Eli Lilly and Company's (NYSE:LLY) pivotal, Phase 3 LUCENT-2 study, patients with ulcerative colitis (UC) who responded to mirikizumab at 12 weeks achieved and maintained statistically superior and clinically meaningful improvements at one year compared to placebo across the primary endpoint of clinical remission and all key secondary endpoints, including bowel urgency severity, using a novel, patient-reported outcome measure. Late-breaking results are being presented today at Digestive Disease Week (DDW). If approved, mirikizumab would become the first and only anti-IL23p19 treatment for people with UC.
"Ulcerative colitis can significantly impact a patient's quality of life, including fecal incontinence due to bowel movement urgency that may even result in individuals wearing diapers. Patients often spend years trying different treatments, including steroids and TNF inhibitors, hoping to achieve remission, reduce inflammation and get relief from painful, disruptive and sometimes embarrassing symptoms," said Marla C. Dubinsky, M.D., Professor of Pediatrics and Medicine, Co-director of the Susan and Leonard Feinstein IBD Clinical Center, Chief of the Division of Pediatric Gastroenterology and Nutrition at the Icahn School of Medicine at Mount Sinai. "I'm encouraged by what the LUCENT-2 findings could mean for patients with UC, as one-half of mirikizumab patients achieved clinical remission and 98 percent of those patients were also not taking steroids. Additionally, 40 percent of mirikizumab patients achieved resolution or near resolution of their distressing symptom of bowel urgency."
Mirikizumab was superior to placebo on clinical, symptomatic, endoscopic and histologic endpoints regardless of previous failure to TNF inhibitors, tofacitinib or other biologics. Among patients who had responded to 12-week induction treatment with mirikizumab, one-half of patients receiving mirikizumab maintenance treatment (49.9%, n=182/365) achieved clinical remission at one year compared to one-fourth of patients on placebo (25.1%, n=45/179, p<0.001). Nearly two-thirds of patients receiving mirikizumab who achieved clinical remission at 12 weeks maintained clinical remission at one year (63.6%, n=91/143) compared to one-third of patients on placebo (36.9%, n=24/65, p<0.001). Nearly all patients receiving mirikizumab who achieved clinical remission at one year were not taking corticosteroids for at least three months prior to the end of maintenance treatment (97.8%, n=178/182).
A patient-centric, 11-point scale developed by Lilly was used to assess changes in bowel urgency severity. Among patients who achieved clinical response in the 12-week induction study and who had a baseline urgency severity of 3 or greater, more than two in five patients on mirikizumab (42.9%, n=144/336) achieved resolution or near resolution of bowel urgency severity at one year compared to one in four on placebo (25%, n=43/172, p<0.001). Among patients who achieved clinical response in the 12-week induction study, patients receiving mirikizumab had a statistically significant average reduction in bowel urgency severity of 3.80 (3.53 to 4.07) at one year, compared to 2.74 (2.35 to 3.14) points for patients on placebo (p<0.001).
Patients receiving mirikizumab in the LUCENT-2 study reported a lower frequency of serious adverse events compared to placebo (mirikizumab: 3.3%, n=13/389; placebo: 7.8%, n=15/192) and were less likely to discontinue the study due to adverse events (mirikizumab: 1.5%, n=6/389; placebo: 8.3%, n=16/192). The overall safety profile was consistent with previous mirikizumab studies in UC and consistent with that of other anti-IL23p19 antibodies in other therapeutic areas.
"We're thrilled to share patients with UC receiving mirikizumab achieved long-term clinical remission, improvement in hard-to-treat symptoms like bowel urgency, and remission of acute inflammation in the colon," said Lotus Mallbris, M.D., Ph.D., vice president of global immunology development and medical affairs at Lilly. "These results are particularly meaningful for patients whose TNF inhibitors, tofacitinib or other biologic therapies have failed them. Lilly leads the way in studying patient-centric outcomes like bowel urgency. We look forward to regulatory decisions next year."
In the first quarter of 2022, Lilly submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) and a Marketing Authorization Application (MAA) in the European Union for approval of mirikizumab in UC. Regulatory decisions in the U.S., E.U. and other countries around the world are expected in 2023.
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About Mirikizumab
Mirikizumab is a humanized IgG4 monoclonal antibody that binds to the p19 subunit of interleukin 23. Mirikizumab is being studied for the treatment of immune-mediated diseases, including ulcerative colitis and Crohn's disease.
About The LUCENT-2 Study
LUCENT-2 (NCT03524092) is a multicenter, randomized, double-blind, placebo-controlled, Phase 3 maintenance study in patients with moderately-to-severely active ulcerative colitis who have previously failed conventional and/or biologic therapies and/or JAK inhibitors and required additional treatment to manage their disease, and who have also completed the 12-week induction study (LUCENT-1). For LUCENT-2, the primary analysis was based on patients who had responded to mirikizumab induction treatment in LUCENT-1 and were re-randomized to receive mirikizumab subcutaneously or placebo for an additional 40 weeks.
Patients who responded to mirikizumab in the 12-week induction study (LUCENT-1) and randomized into LUCENT-2 were on average 42.7 years old and had lived with UC for approximately 6.8 years since first experiencing symptoms at an average age of 36.1 years. Approximately 58.5% (n=318/544) of patients were male. Three out of five patients (62.7%, n=341/544) had severe intestinal mucosal inflammation, as measured by a Mayo endoscopic subscore of 3. 35.3% (n=192/544) had previously failed one or more biologics or tofacitinib, and 37.3% (n=203/544) had baseline corticosteroid use.
Clinical remission, the primary endpoint of the LUCENT-2 clinical trial, is achieved when inflammation of the colon is controlled or resolved, leading to normalization or near-normalization of symptoms such as stool frequency and bleeding, and is defined by a stool frequency (SF) score = 0, or 1 with a ≥1-point decrease from baseline, a rectal bleeding (RB) score = 0, and endoscopy score (ES) = 0 or 1, excluding friability, which is the propensity for tissue that covers the inside of the colon (also known as colonic mucosa) to be damaged or bleed because of contact with an endoscope or biopsy instrument.
Clinical response is measured by the decrease in the modified Mayo score of ≥2 points and ≥30% decrease from baseline (BL) and decrease of ≥1 point in the RB subscore from baseline or a RB score of 0 or 1.
Rather than using a binary scale to measure the presence or absence of bowel urgency, Lilly developed the bowel urgency numeric rating scale (NRS), a patient-centric 11-point scale (0 – 10) to assess change in bowel urgency severity from baseline in order to better understand UC patients' experiences living with bowel urgency. Using this measure, patients receiving mirikizumab achieved clinically meaningful reduction in bowel urgency severity at one year compared to placebo.
About the LUCENT Clinical Trial Program
The LUCENT Phase 3 clinical development program for mirikizumab began in 2018 and includes LUCENT-1, LUCENT-2 and LUCENT-3. LUCENT-2 is a multicenter, randomized, double-blind, placebo-controlled maintenance study of mirikizumab in patients who have completed the 12-week induction study (LUCENT-1). Patients in LUCENT-1 who achieved clinical response with mirikizumab induction therapy were re-randomized to receive mirikizumab or placebo subcutaneously for an additional 40 weeks in the LUCENT-2 study. LUCENT-3 (NCT03519945) is an open label extension study for eligible patients who have participated in mirikizumab UC trials. Additional data from the Phase 3 LUCENT program, the first Phase 3 study of an anti-IL23p19 antibody in UC, will be disclosed at upcoming congresses and in publications in 2022.
To learn more, visit Lilly.com and Lilly.com/newsroom or follow us on Facebook, Instagram, Twitter and LinkedIn.
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SOURCE Eli Lilly and Company
Lilly mirikizumab helps reduce ulcerative colitis symptoms in 50% patients at 1 year in trial
May 24, 2022 5:38 AM ET
By: Ravikash, SA News Editor2 Comments
Eli Lilly (NYSE:LLY) said ~50% of patients with ulcerative colitis (UC) treated with mirikizumab achieved clinical remission at one year, compared to placebo in a late-stage study.
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EVKEEZA® (EVINACUMAB)
EVKEEZA® (EVINACUMAB) PHASE 3 TRIAL DEMONSTRATES 48% LDL-C REDUCTION IN CHILDREN WITH ULTRA-RARE FORM OF HIGH CHOLESTEROL
TARRYTOWN, N.Y., May 21, 2022 /PRNewswire/ --
Children already on other lipid-lowering therapies entered the trial with dangerously high LDL-C (264 mg/dL on average), and 79% saw their LDL-C reduced by at least half at 24 weeks
FDA submission planned by end of 2022
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced positive results from a Phase 3 trial evaluating Evkeeza® (evinacumab) in children aged 5 to 11 with homozygous familial hypercholesterolemia (HoFH). The trial met its primary endpoint, showing children who added investigational Evkeeza to other lipid-lowering therapies reduced their low-density lipoprotein-cholesterol (LDL-C) by 48% at week 24 on average. Detailed results were presented today at the 5th European Atherosclerosis Society Pediatric Familial Hypercholesterolemia symposium and will form the basis of a regulatory submission to the U.S. Food and Drug Administration (FDA) later this year.
"Children living with HoFH have an incredibly rare and severe disease that causes dangerously high LDL-C levels. On current treatment options alone, many patients don't reach their treatment goals, leaving them with an uncertain future," said M. Doortje Reijman, M.D., Research Associate in Pediatric Metabolic Diseases and Nephrology at the Amsterdam University Medical Center, and a trial investigator. "Evinacumab has already demonstrated significant LDL-C reductions in adolescents and adults with HoFH. This latest Phase 3 trial illustrates the potential of this medicine to be a breakthrough HoFH therapy for children as young as 5-years old, helping them control their LDL-C early in the course of their disease."
Despite treatment with other lipid-lowering therapies, children (n=14) entered the trial with an average LDL-C level of 264 mg/dL, more than twice the target (<130 mg/dL) for pediatric patients with HoFH. After 24 weeks of Evkeeza treatment (15 mg/kg every 4 weeks delivered intravenously [IV]), the Phase 3 trial met its primary endpoint with additional results showing:
- 79% of patients reduced their LDL-C by at least half
- An absolute 132 mg/dL reduction in LDL-C from baseline, on average
- Reductions in levels of all lipid endpoint parameters assessed, which were generally observed within the first 8 weeks of treatment. These lipid parameters were apolipoprotein B, non-high-density lipoprotein cholesterol, lipoprotein(a) and total cholesterol.
Evkeeza was generally well-tolerated with all patients completing the trial. The most common adverse events (AEs) were throat pain (oropharyngeal pain, 21%) as well as upper abdominal pain, diarrhea, headache and nasopharyngitis (all 14%). There were 2 severe AEs (aortic stenosis and tonsilitis), both of which were considered unrelated to treatment.
Evkeeza is the first ANGPTL3-targeted (angiopoietin-like 3-targeted) therapy approved by the FDA (as evinacumab-dgnb) and European Commission as an adjunct therapy for certain patients aged 12 years and older with HoFH.
The potential use of Evkeeza in HoFH patients aged 5 to 11 years is currently under clinical development, and its safety and efficacy have not been fully evaluated by any regulatory authority.
About the trial
The Phase 3 data are the Part B portion of a three-part, single-arm, open-label trial evaluating Evkeeza in pediatric patients with HoFH aged 5 to 11 years. In Part B, 14 patients were enrolled with an average age of 9 years. Among them, 86% were on statins, 93% were on ezetimibe, 50% were on LDL apheresis and 14% were on lomitapide.
During the 24-week treatment period, patients received Evkeeza 15 mg/kg every four weeks via IV alongside their lipid-lowering treatment regimen. The primary endpoint was change in LDL-C at week 24. Secondary endpoints included the effect of Evkeeza on other lipid parameters, efficacy by mutation status, safety and tolerability, immunogenicity and pharmacokinetics (PK).
Part A was a Phase 1b trial designed to assess the PK, safety and tolerability of Evkeeza. Patients who completed Part A or B were allowed to continue treatment in Part C, an ongoing Phase 3 extension trial. Parts A, B and C were not designed to evaluate the effect of Evkeeza on cardiovascular events.
About Evkeeza® (evinacumab)
Evkeeza was invented using Regeneron's VelocImmune® technology and is a fully human monoclonal antibody that binds to and blocks the function of ANGPTL3, a protein that inhibits lipoprotein lipase (LPL) and endothelial lipase (EL) and regulates circulating lipids, including LDL-C.
Regeneron scientists discovered the angiopoietin gene family more than two decades ago (see publications from 1996, 1997 and 1999). Human genetics research published in New England Journal of Medicine in 2017 by scientists from the Regeneron Genetics Center® found that patients whose ANGPTL3 gene did not function properly (called a "loss-of function mutation") have significantly lower levels of key blood lipids, including LDL-C, and that this is associated with a significantly lower risk of coronary artery disease.
The generic name for Evkeeza in its approved U.S. indications is evinacumab-dgnb, with dgnb the suffix designated in accordance with Nonproprietary Naming of Biological Products Guidance for Industry issued by the U.S. FDA. The safety and effectiveness of Evkeeza have not been established in patients with other causes of hypercholesterolemia, including those with heterozygous familial hypercholesterolemia (HeFH). The effect of Evkeeza on cardiovascular morbidity and mortality has not been determined.
Regeneron is responsible for the development and distribution of Evkeeza in the U.S. and is collaborating with Ultragenyx to clinically develop, commercialize and distribute Evkeeza outside of the U.S.
https://www.evkeeza.com/s/#isi-10
Please see full Prescribing Information, including Patient Information.
For additional information about the company, please visit www.regeneron.com or follow @Regeneron on Twitter.
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SOURCE Regeneron Pharmaceuticals, Inc.
Regeneron, Ultragenyx's Evkeeza helps reduce bad cholesterol in kids 5 to 11 with rare disorder in study
May 23, 2022 5:55 AM ET Regeneron Pharmaceuticals, Inc. (REGN)RARE
By: Ravikash, SA News Editor1 Comment
Regeneron Pharmaceuticals (NASDAQ:REGN) said a phase 3 trial of Evkeeza (evinacumab) met its main goal in children aged 5 to 11 with homozygous familial hypercholesterolemia (HoFH).
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Vaxzevria (ChAdOx1-S [Recombinant], formerly AZD1222)
Vaxzevria approved in the EU as third dose booster against COVID-19
PUBLISHED23 May 2022
23 May 2022 07:05 BST
Approval follows CHMP recommendation for use in patients previously vaccinated with Vaxzevria or an EU-approved mRNA COVID-19 vaccine
AstraZeneca's COVID-19 vaccine, Vaxzevria (ChAdOx1-S [Recombinant]), has been granted approval in the European Union (EU) by the European Medicine Agency (EMA) as a third dose booster in adults.
Healthcare professionals can now use Vaxzevria as a third dose booster in patients previously given a primary vaccine schedule of either Vaxzevria or an EU-approved mRNA COVID-19 vaccine.
The authorisation is based on a review by the Committee for Medicinal Products for Human Use (CHMP) of the substantial body of evidence demonstrating an increased immune response after a third dose booster with Vaxzevria following a primary vaccine schedule of either Vaxzevria or an mRNA COVID-19 vaccine.1-5
Although more than 65% of the global population has received at least one dose of a COVID-19 vaccine6, there remains a significant challenge to ensure people receive both their primary vaccine schedule and third dose booster, and healthcare professionals now have greater flexibility in their choice of vaccine.
Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, AstraZeneca said: “Today’s marketing authorisation for AstraZeneca’s COVID-19 vaccine as a third dose booster is an important step towards our goal of providing continued protection against COVID-19 for all populations. Ensuring a longer duration of immune protection is essential to the long-term management of COVID-19 globally, and boosters can address the waning of protection over time that has been seen with all primary vaccine schedules to date.”
There is a substantial body of evidence supporting Vaxzevria as a third dose booster following all primary vaccination schedules tested to date including Vaxzevria, mRNA vaccines, and CoronaVac.1,7-12
Vaxzevria is already authorised as a homologous booster (patients previously given a primary vaccine schedule of Vaxevria) in the UK, and several countries in Asia and Latin America. It has also been authorised as a heterologous booster (patients previously given a primary vaccine schedule of either a viral vector vaccine other than Vaxzevria or an inactivated vaccine or an mRNA COVID-19 vaccine) in a number of non-EU countries.
Vaxzevria is estimated to have helped prevent 50 million COVID-19 cases, five million hospitalisations, and saved more than one million lives worldwide, based on model outcomes assessing COVID-19 worldwide.13
Notes
Vaxzevria (ChAdOx1-S [Recombinant], formerly AZD1222)
AstraZeneca COVID-19 vaccine was invented by the University of Oxford. It uses a replication-deficient chimpanzee viral vector based on a weakened version of a common cold virus (adenovirus) that causes infections in chimpanzees and contains the genetic material of the SARS-CoV-2 virus spike protein. After vaccination, the surface spike protein is produced, priming the immune system to attack the SARS-CoV-2 virus if it later infects the body.
Vaxzevria is a ‘viral vector’ vaccine, which means a version of a virus that cannot cause disease is used as part of the vaccine, leaving the body knowing how to fight it if it is exposed to the real virus later. This vaccine technology has been used by scientists over the past 40 years to fight other infectious diseases such as the flu, Ebola, and HIV.14
The vaccine has been granted a conditional marketing authorisation or emergency use in more than 125 countries. It also has Emergency Use Listing from the World Health Organization, which accelerates the pathway to access in up to 144 countries through the COVAX Facility.
Under a sub-license agreement with AstraZeneca, the vaccine is manufactured and supplied by the Serum Institute of India under the name COVISHIELD.
Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca.
AstraZeneca's COVID Vaxzevria gets approval in EU as booster dose
May 23, 2022 4:39 AM ET
By: Ravikash, SA News Editor
- The European Medicine Agency granted approval to AstraZeneca's (NASDAQ:AZN) COVID-19 vaccine Vaxzevria for use as a third dose booster shot in adults in the EU.
- https://seekingalpha.com/news/3841585-astrazenecas-covid-vaxzevria-gets-approval-in-eu-as-booster-dose
- https://seekingalpha.com/symbol/AZN
COMIRNATY® (COVID-19 Vaccine, mRNA)
Pfizer-BioNTech COVID-19 Vaccine Demonstrates Strong Immune Response, High Efficacy and Favorable Safety in Children 6 Months to Under 5 Years of Age Following Third Dose
Monday, May 23, 2022 - 06:45am
- Based on topline data, three doses of the Pfizer-BioNTech COVID-19 Vaccine met all immunobridging criteria required for Emergency Use Authorization
- The third 3-µg dose was well tolerated among 1,678 children under 5 years of age with a safety profile similar to placebo
- Vaccine efficacy of 80.3% was observed in descriptive analysis of three doses during a time when Omicron was the predominant variant
- The 3-µg dose level, which is one-tenth the dose for adults, was selected for children under 5 years of age based on safety, tolerability and immunogenicity
NEW YORK & MAINZ, Germany--(BUSINESS WIRE)-- Pfizer Inc. (NYSE: PFE) and BioNTech SE (Nasdaq: BNTX) today announced topline safety, immunogenicity and vaccine efficacy data from a Phase 2/3 trial evaluating a third 3-µg dose of the Pfizer-BioNTech COVID-19 Vaccine in children 6 months to under 5 years of age. Following a third dose in this age group, the vaccine was found to elicit a strong immune response, with a favorable safety profile similar to placebo.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20220522005063/en/
Vaccine efficacy, a secondary endpoint in this trial, was 80.3% in children 6 months to under 5 years of age. This descriptive analysis was based on 10 symptomatic COVID-19 cases identified from seven days after the third dose and accrued as of April 29, 2022. The trial protocol specifies a formal analysis will be performed when at least 21 cases have accrued from seven days after the third dose. Final vaccine efficacy data will be shared once available.
“Our COVID-19 vaccine has been studied in thousands of children and adolescents, and we are pleased that our formulation for the youngest children, which we carefully selected to be one-tenth of the dose strength for adults, was well tolerated and produced a strong immune response,” said Albert Bourla, Chairman and Chief Executive Officer, Pfizer. “These topline safety, immunogenicity and efficacy data are encouraging, and we look forward to soon completing our submissions to regulators globally with the hope of making this vaccine available to younger children as quickly as possible, subject to regulatory authorization.”
“The study suggests that a low 3-ug dose of our vaccine, carefully selected based on tolerability data, provides young children with a high level of protection against the recent COVID-19 strains,” said Prof. Ugur Sahin, M.D., CEO and co-founder of BioNTech. “We are preparing the relevant documents and expect completing the submission process to the FDA this week, with submissions to EMA and other regulatory agencies to follow within the coming weeks.”
In the Phase 2/3 trial, 1,678 children received a third dose of the 3-µg formulation at least two months after the second dose at a time when Omicron was the predominant variant. The immunogenicity analysis of geometric mean titer (GMT) ratio and seroresponse rate was conducted on a subset of study participants one month following the third dose in children 6 months to under 5 years of age, compared to the second dose in the 16- to 25-year-old population. Non-inferiority was met for both the 6- to 24-month-old population and the 2- to under 5-year-old population for both co-primary endpoints. Three 3-µg doses of the Pfizer-BioNTech COVID-19 Vaccine was well-tolerated in this age group, and no new safety signals were identified. The majority of adverse events were mild or moderate.
Studies in adults, adolescents, and children over 5 years of age continue to indicate that three doses of the Pfizer-BioNTech COVID-19 Vaccine enhances protection compared to two doses. The safety, immunogenicity and vaccine efficacy data for three doses of the vaccine in children under 5 years of age are consistent with the data seen in adults, suggesting that a third dose will provide similar benefit in children.
In February 2022, the companies initiated a rolling submission for Emergency Use Authorization (EUA) of their COVID-19 vaccine in children 6 months to under 5 years of age, following a request by the U.S. Food and Drug Administration (FDA). At that time, a two-dose series was determined to be well-tolerated in this age group. Pfizer and BioNTech plan to submit these new safety, immunogenicity, and vaccine efficacy data on three doses to the rolling U.S. EUA application this week, with submissions to regulators worldwide to follow.
The Pfizer-BioNTech COVID-19 Vaccine, which is based on BioNTech’s proprietary mRNA technology, was developed by both BioNTech and Pfizer. BioNTech is the Marketing Authorization Holder in the United States, the European Union, the United Kingdom, Canada and the holder of emergency use authorizations or equivalents in the United States (jointly with Pfizer) and other countries. Submissions to pursue regulatory approvals in those countries where emergency use authorizations or equivalent were initially granted are planned.
About the Phase 1/2/3 Trial in Children
The Phase 1/2/3 trial has enrolled more than 10,000 children ages 6 months to under 12 years of age in the United States, Finland, Poland, and Spain from more than 90 clinical trial sites. The trial evaluated the safety, tolerability, and immunogenicity of three doses of the Pfizer-BioNTech COVID-19 Vaccine in three age groups: ages 5 to under 12 years; ages 2 to under 5 years; and ages 6 months to under 2 years. Based on the Phase 1 dose-escalation portion of the trial, children ages 5 to under 12 years received a two-dose schedule of 10 µg each while children under age 5 received a lower 3 µg dose for each injection in the Phase 2/3 study. The trial enrolled children with or without prior evidence of SARS-CoV-2 infection.
U.S. Indication & Authorized Use
Pfizer-BioNTech COVID-19 Vaccine is FDA authorized under Emergency Use Authorization (EUA) for active immunization to prevent coronavirus disease 2019 (COVID-19) caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) in individuals 5 years of age and older.
Pfizer-BioNTech COVID-19 Vaccine is FDA authorized to provide:
COMIRNATY®INDICATION
COMIRNATY® (COVID-19 Vaccine, mRNA) is a vaccine approved for active immunization to prevent coronavirus disease 2019 (COVID-19) caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) in individuals 16 years of age and older.
- COMIRNATY® is administered as a 2-dose primary series
COMIRNATY® AUTHORIZED USES
COMIRNATY® (COVID-19 Vaccine, mRNA) is FDA authorized under Emergency Use Authorization (EUA) to provide:
Primary Series
- a 2-dose primary series to individuals 12 through 15 years of age
- a third primary series dose to individuals 12 years of age and older with certain kinds of immunocompromise
Booster Dose
- a first booster dose to individuals 12 years of age and older who have completed a primary series with Pfizer-BioNTech COVID-19 Vaccine or COMIRNATY®
- a first booster dose to individuals 18 years of age and older who have completed primary vaccination with another authorized or approved COVID-19 vaccine. The booster schedule is based on the labeling information of the vaccine used for the primary series
- a second booster dose to individuals 50 years of age and older who have received a first booster dose of any authorized or approved COVID-19 vaccine
- a second booster dose to individuals 12 years of age and older with certain kinds of immunocompromise and who have received a first booster dose of any authorized or approved COVID-19 vaccine
For more information, please visit www.BioNTech.de. www.pfizer.com.
Pfizer-BioNTech share three-dose data for COVID-19 vaccine in children under five
May 23, 2022 7:35 AM ET Pfizer Inc. (PFE), BNTX
By: Dulan Lokuwithana, SA News Editor1 Comment
Announcing topline data from a Phase 2/3 trial for their COVID-19 vaccine in children aged 6 months to under 5 years, Pfizer (NYSE:PFE) and BioNTech (NASDAQ:BNTX) said on Monday that a third dose of the vaccine at 3-µg offered about 80% efficacy when the Omicron variant was dominant.
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https://seekingalpha.com/symbol/BNTX
https://www.pfizer.com/products/product-detail/comirnaty
Cariprazine (VRAYLAR®)
May 23, 2022
AbbVie Presents Positive Data from Phase 3 Study of Cariprazine (VRAYLAR®) for the Adjunctive Treatment of Major Depressive Disorder at 2022 APA Annual Meeting
- In Study 3111-301-001, cariprazine (VRAYLAR®; 1.5mg/day) achieved the primary endpoint of statistically significant improvement on the Montgomery-Åsberg Depression Rating Scale (MADRS) total score in patients with major depressive disorder (MDD) with an inadequate response to ongoing antidepressant therapy
- Safety profile was consistent with that of previous studies across indications in the treatment of adults with depressive episodes associated with bipolar I disorder, the acute treatment of manic or mixed episodes associated with bipolar I disorder and schizophrenia
- AbbVie's supplemental New Drug Application (sNDA) for cariprazine, which includes Study 3111-301-001, is currently under review by the U.S. Food and Drug Administration (FDA) for expanded use in the adjunctive treatment of MDD with a decision expected by year-end
NORTH CHICAGO, Ill., May 23, 2022 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced it will present positive data from a Phase 3 trial of cariprazine (VRAYLAR®; 1.5mg/day), Study 3111-301-001, for the adjunctive treatment of major depressive disorder (MDD) in patients with an inadequate response to ongoing antidepressant therapy. The study met its primary endpoint of statistically significant improvement using the Montgomery-Åsberg Depression Rating Scale (MADRS) total score in patients compared with placebo. These results will be presented (Poster Number: P7-037) on Tuesday, May 24, at the American Psychiatric Association (APA) Annual Meeting in New Orleans.
AbbVie's supplemental New Drug Application (sNDA) for cariprazine was supported by two positive registration-enabling studies, of which one was Study 3111-301-001. The sNDA is currently under review by the U.S. Food and Drug Administration (FDA) for expanded use in the adjunctive treatment of MDD with a decision expected by year-end.
"We are pleased to present these data demonstrating cariprazine's potential to have an impact on patients as an adjunctive treatment of major depressive disorder," said Michael Gold, M.D., therapeutic area head, neuroscience development, AbbVie. "The results from this study, along with previously reported data, served as the basis of our recent sNDA submission to the FDA and further underpin AbbVie's commitment to advancing innovative psychiatric therapies for patients in need."
The Phase 3 study showed a statistically significant change from baseline to week six in the MADRS total score for patients treated with cariprazine at 1.5 mg/day compared with placebo (p-value = 0.0050). Patients treated with cariprazine at 3.0 mg/day demonstrated improvement in MADRS total score at week six compared to placebo but did not meet statistical significance (p-value=0.0727).
In this study, the safety data was consistent with the established safety profile of cariprazine across indications. There were no deaths in the trial, with serious adverse events (SAE) occurring in both treatment (two SAEs) and placebo (two SAEs) groups. The most common adverse events (AE) in the treatment group were akathisia and nausea (≥5%).
"The Phase 3 trial results, along with previous study results, indicate that cariprazine may have the potential to help patients with MDD for whom antidepressant therapy alone is not sufficient," said Gary Sachs, MD, associate clinical professor of psychiatry at Massachusetts General Hospital and lead author of the study. "Nearly half of patients with MDD do not experience satisfactory results from their current treatment regimen, so ongoing research to bring more options to patients is critical."
Cariprazine is marketed as VRAYLAR® in the United States and is FDA-approved to treat adults with depressive, acute manic and mixed episodes associated with bipolar I disorder, as well as schizophrenia. Cariprazine is being co-developed by AbbVie and Gedeon Richter Plc. More than 8,000 patients worldwide have been treated with cariprazine across more than 20 clinical trials evaluating the efficacy and safety of cariprazine for a broad range of psychiatric disorders.
About Study 3111-301-001
Study 3111-301-001 is a randomized, double-blind, placebo-controlled, multicenter trial with 751 participants conducted in United States, Bulgaria, Estonia, Germany, Hungary, Ukraine and the United Kingdom. Following a screening period of up to 14 days, patients with an inadequate clinical response to their antidepressant monotherapy (ADT) were randomized into three treatment groups (1:1:1). The first group received cariprazine 1.5 mg/day + ADT, the second group received cariprazine 3.0 mg/day + ADT, and the third group received placebo + ADT. For six weeks, the medication was given once daily in addition to the ongoing ADT treatment.
The primary endpoint was determined using the MADRS total score, a 10-item, clinician-rated scale that evaluates the patient's depressive symptomatology during the past week. Patients are rated on items assessing feelings of sadness, lassitude, pessimism, inner tension, suicidality, reduced sleep or appetite, difficulty in concentration, and lack of interest. Each item is scored on a 7-point scale with a score of 0 reflecting no symptoms and a score of 6 reflecting symptoms of maximum severity.4
More information can be found on www.clinicaltrials.gov (NCT03738215).
About VRAYLAR® (cariprazine)
VRAYLAR is an oral, once-daily atypical antipsychotic approved for the treatment of depressive episodes associated with bipolar I disorder (bipolar depression) in adults (1.5 or 3 mg/day) and for the acute treatment of adults with manic or mixed episodes associated with bipolar I disorder (3 to 6 mg/day). VRAYLAR is also approved for the treatment of schizophrenia in adults (1.5 to 6 mg/day). Use of VRAYLAR in adjunctive treatment of major depressive disorder is not approved and its safety and efficacy have not been evaluated by regulatory authorities.
While the mechanism of action of VRAYLAR is unknown, the efficacy of VRAYLAR could be mediated through a combination of partial agonist activity at central dopamine D₂ and serotonin 5-HT1A receptors and antagonist activity at serotonin 5-HT2A receptors. Pharmacodynamic studies with VRAYLAR have shown that it may act as a partial agonist with high binding affinity at dopamine D3, dopamine D2, and serotonin 5-HT1A receptors. VRAYLAR demonstrated up to ~8-fold greater in vitro affinity for dopamine D3 vs D2 receptors. VRAYLAR also acts as an antagonist at serotonin 5-HT2B and 5-HT2A receptors with high and moderate binding affinity, respectively as well as it binds to the histamine H1 receptors. VRAYLAR shows lower binding affinity to the serotonin 5-HT2C and α1A- adrenergic receptors and has no appreciable affinity for cholinergic muscarinic receptors.5 The clinical significance of these in vitro data is unknown.
VRAYLAR is being developed jointly by AbbVie and Gedeon Richter Plc, with AbbVie responsible for commercialization in the U.S., Canada, Japan, Taiwan and certain Latin American countries (including Argentina, Bolivia, Brazil, Chile, Colombia, Ecuador, Mexico, Peru and Venezuela).
VRAYLAR® (cariprazine) Uses and Important Safety Information
VRAYLAR is a prescription medicine used in adults:
- to treat schizophrenia
- for short-term (acute) treatment of manic or mixed episodes that happen with bipolar I disorder
- to treat depressive episodes that happen with bipolar I disorder (bipolar depression)
For more information about AbbVie, please visit us at www.abbvie.com. Follow @abbvie on Twitter, Facebook, Instagram, YouTube and LinkedIn.
SOURCE AbbVie
https://seekingalpha.com/symbol/ABBV
ABBV Dividend History
https://www.nasdaq.com/market-activity/stocks/abbv/dividend-history
Upadacitinib (RINVOQ®)
May 23, 2022
CHMP Recommends European Commission Approval of Upadacitinib (RINVOQ®) for the Treatment of Adults with Moderate to Severe Ulcerative Colitis
- CHMP positive opinion is based on results from three Phase 3 studies: two for induction and one for maintenance1-3
- Ulcerative colitis is a chronic, immune-mediated inflammatory bowel disease that can lead to substantial burden and often disability among patients4-6
- If approved by the European Commission (EC), this would be upadacitinib's fifth therapeutic indication in the EU
- The EC decision is anticipated in the third quarter of 2022
NORTH CHICAGO, Ill., May 23, 2022 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending the approval of upadacitinib (RINVOQ®, 45 mg [induction dose] and 15 mg and 30 mg [maintenance dose]) for the treatment of adult patients with moderately to severely active ulcerative colitis (UC) who have had an inadequate response, lost response or were intolerant to either conventional therapy or a biologic agent.*
UC is the chronic inflammation of the large intestine, usually beginning in the rectum and lower colon, but may also spread continuously to involve the entire colon, which could lead to a significant burden and disability for patients.5 Living with UC impacts all aspects of a patient's life, largely due to unpredictable symptoms such as bowel urgency, abdominal pain, rectal bleeding and bowel incontinence.5
"As leaders in the advancement of care for people with inflammatory bowel disease, we are committed to continued research and development of treatment options that go beyond managing symptoms and include endoscopic and histologic outcomes," said Neil Gallagher, M.D., Ph.D., vice president, development, chief medical officer, AbbVie. "With today's CHMP recommendation of upadacitinib in ulcerative colitis, we've taken a giant step forward toward helping more patients achieve their treatment goals."
AbbVie's application for the approval of upadacitinib in UC is supported by data from two induction studies, U-ACHIEVE induction and U-ACCOMPLISH, and one maintenance study, U-ACHIEVE maintenance.1-3 Across all three Phase 3 studies, significantly more patients treated with upadacitinib achieved the primary endpoint of clinical remission† and all secondary endpoints. This includes statistically significant improvements in these endpoints compared to placebo with 45 mg once daily for the induction studies, and with both 15 mg and 30 mg once-daily doses for the maintenance study.1-3 Additionally, safety results of upadacitinib in UC were consistent with the known safety profile of upadacitinib in rheumatoid arthritis, with no new important safety risks observed.1-3, 7-10
"Patients with ulcerative colitis often face numerous complications and risk relapse. Achieving critical endpoints like clinical remission and mucosal healing can make a significant difference in symptom management and health related quality of life," said Silvio Danese, M.D., Ph.D., director of Gastroenterology and Endoscopy at San Raffaele Hospital and professor of gastroenterology at University Vita-Salute San Raffaele, Milan, Italy. "Upadacitinib could be a promising option for adult patients who continue to have moderately to severely active disease despite treatment with conventional or biologic therapies and I look forward to the European Commission's final decision on its use in ulcerative colitis."
About the U-ACHIEVE Induction, U-ACCOMPLISH and U-ACHIEVE Maintenance Studies1-3,11-13
The three Phase 3 studies are multicenter, randomized, double-blind, placebo-controlled studies to evaluate the efficacy and safety of upadacitinib 45 mg once daily as induction therapy, and upadacitinib 15 mg and 30 mg once daily as maintenance therapy in subjects with moderate to severe ulcerative colitis. Topline results of the U-ACHIEVE induction study were announced in December 2020, topline results of the second induction study, U-ACCOMPLISH, were announced in February 2021, and topline results of the U-ACHIEVE maintenance study were announced in June 2021. More information can be found on http://www.clinicaltrials.gov (NCT03006068, NCT03653026, NCT02819635).
About upadacitinib (RINVOQ®)
Discovered and developed by AbbVie scientists, RINVOQ is a selective and reversible JAK inhibitor that is being studied in several immune-mediated inflammatory diseases.15-20 In human cellular assays, upadacitinib preferentially inhibits signalling by JAK1 or JAK1/3 with functional selectivity over cytokine receptors that signal via pairs of JAK2.14
Phase 3 trials of RINVOQ in rheumatoid arthritis, atopic dermatitis, psoriatic arthritis, axial spondyloarthritis, Crohn's disease, ulcerative colitis, giant cell arteritis and Takayasu arteritis are ongoing.13,15-20 The use of upadacitinib in ulcerative colitis is not approved and its safety and efficacy have not been evaluated by regulatory authorities outside of the U.S. and Puerto Rico.
EU Indications and Important Safety Information about RINVOQ® (upadacitinib)14
Rheumatoid arthritis
RINVOQ is indicated for the treatment of moderate to severe active rheumatoid arthritis in adult patients who have responded inadequately to, or who are intolerant to one or more disease-modifying anti-rheumatic drugs (DMARDs). RINVOQ may be used as monotherapy or in combination with methotrexate.
Psoriatic arthritis
RINVOQ is indicated for the treatment of active psoriatic arthritis in adult patients who have responded inadequately to, or who are intolerant to one or more DMARDs. RINVOQ may be used as monotherapy or in combination with methotrexate.
Ankylosing spondylitis
RINVOQ is indicated for the treatment of active ankylosing spondylitis in adult patients who have responded inadequately to conventional therapy.
Atopic dermatitis
RINVOQ is indicated for the treatment of moderate to severe atopic dermatitis in adults and adolescents 12 years and older who are candidates for systemic therapy.
See RINVOQ full summary of product characteristics (SmPC) at www.ema.europa.eu.
SOURCE AbbVie
AbbVie's Rinvoq gets EMA panel nod for expanded use in ulcerative colitis
May 23, 2022 6:20 AM ET AbbVie Inc. (ABBV)
By: Ravikash, SA News Editor
- A committee of the European Medicines Agency (EMA) recommended the expanded use approval of AbbVie's (NYSE:ABBV) Rinvoq (upadacitinib) to treat adult patients with moderately to severely active ulcerative colitis (UC) who have had an inadequate response, lost response or were intolerant to either conventional therapy or a biologic agent.
- https://seekingalpha.com/news/3841602-abbvies-rinvoq-gets-ema-panel-nod-for-expanded-use-in-ulcerative-colitis
- https://seekingalpha.com/symbol/ABBV
- https://www.rinvoq.com/
Cosentyx® (secukinumab)
Novartis Cosentyx® (secukinumab) receives positive CHMP opinion for expanded use in childhood arthritic conditions
May 20, 2022
- Positive opinion could expand the role of Cosentyx®(secukinumab) in reducing flare risk in pediatric enthesitis-related arthritis (ERA) and psoriatic arthritis (PsA) patients in the EU
- Safety in these pediatric populations was consistent with the known safety profile across approved adult and pediatric indications
- Since its initial approval, Cosentyx has a proven sustained efficacy profile across several systemic inflammatory conditions and has treated more than 700,000 patients worldwide 1-11
Basel, May 20, 2022 — Novartis today announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion for Cosentyx® (secukinumab), used alone or in combination with methotrexate, in the juvenile idiopathic arthritis (JIA) categories of enthesitis-related arthritis (ERA) and juvenile psoriatic arthritis (JPsA) in patients 6 years and older whose disease has responded inadequately to, or who cannot tolerate, conventional therapy8.
“JIA is the most common childhood rheumatic disease, affecting millions of children worldwide. Unfortunately, due to the limitations of current or available treatment options, some patients continue to experience symptoms, with an impact on their life. If approved in Europe, Cosentyx could offer a much-needed, additional effective treatment for the underserved ERA and JPsA patient populations,” said Ivan Foeldvari, M.D., Hamburg Centre for Pediatric Rheumatology, Germany.
ERA and JPsA are progressive, debilitating autoimmune diseases12-14. ERA is characterized by joint swelling and pain where tendons and ligaments attach to bone and may present with lower back pain or tenderness at the palpation of the hips13-15. JPsA is characterized by joint swelling and skin psoriasis and may present with nail changes, inflammation of fingers and/or toes or psoriatic skin changes in a first-degree relative. If left untreated, they can lead to high levels of pain and disability12-15.
“The positive CHMP opinion reinforces that children and adults living with immunologic rheumatic and dermatological diseases, and the physicians who treat them, may feel confident in the management of these diseases with Cosentyx,” said Todd Fox, Global Head of Medical Affairs Immunology at Novartis. “We’re committed to bringing innovative treatments to young people living with rheumatic diseases across the world. With recent approvals in the US, Japan and Brazil, we are one step closer in our ambition to expand Cosentyx to 10 indications in areas of high unmet need.”
The positive CHMP opinion is based on data from the Phase III JUNIPERA study, a 2-year, three-part, double-blind, placebo-controlled, randomized withdrawal trial showing significantly longer time to flare in Cosentyx vs placebo (P<.001) in pediatric ERA and JPsA patients16. Safety in this pediatric population was consistent with the known safety profile of Cosentyx across approved adult and pediatric indications9.
This CHMP recommendation supports Novartis ongoing commitment to the pediatric community across a range of inflammatory conditions. In July 2020, Cosentyx received EMA approval as a first-line systemic treatment for pediatric psoriasis in patients aged 6-18 years old and recently received approval in the US and China8, 17. In 2021, Cosentyx was also approved in Japan to treat both PsA and psoriasis in pediatric patients aged 6 years or older, as well as those with generalized pustular psoriasis18. Earlier this year, Cosentyx was also approved in Brazil to treat ERA in patients 4 years or older and JPsA in patients aged 2 years and older19.
About the JUNIPERA trial
The positive CHMP opinion is based on data from the Phase III JUNIPERA study, a 2-year, three-part, double-blind, placebo-controlled, randomized withdrawal trial that enrolled 86 children and adolescents aged 2–18 years old with a confirmed diagnosis of ERA or JPsA according to a modified International League of Associations for Rheumatology classification criteria16. The primary endpoint of the study was time to flare in the treatment period 2 (Week 12 to Week 104)16. The study met its primary endpoint and demonstrated a statistically significant longer time to disease flare in treatment period 2 for ERA and JPsA with secukinumab versus placebo. The risk of flare was reduced by 72% for patients on secukinumab compared with patients on placebo in treatment period 2 (Hazard ratio=0.28, 95% CI: 0.13 to 0.63, p<0.001). A total of 21 patients in the placebo group experienced a flare event (11 JPsA and 10 ERA) compared with 10 patients in the secukinumab group (4 JPsA and 6 ERA) during the placebo-controlled treatment period 2 of the study8, 10. Safety in this pediatric population was consistent with the known safety profile of Cosentyx for the treatment of adult and pediatric plaque psoriasis, PsA and axial spondyloarthritis9.
About Cosentyx
Cosentyx is the first and only fully human biologic that directly inhibits interleukin-17A, an important cytokine involved in the inflammation of psoriatic arthritis (PsA), moderate to severe plaque psoriasis, ankylosing spondylitis (AS) and non-radiographic axial spondyloarthritis (nr-axSpA)8, 20. Cosentyx is a proven medicine and has been studied clinically for more than 14 years. The medicine is backed by robust evidence, including 5 years of clinical data in adults supporting long-term safety and efficacy across moderate to severe plaque psoriasis, PsA and AS1-3, 5-7, 21. These data strengthen the position of Cosentyx as a treatment across AS, nr-axSpA, PsA and moderate to severe plaque psoriasis, supported by more than 700,000 patients treated worldwide since launch in 20158, 11, 22.
Find out more at https://www.novartis.com.
Novartis' Cosentyx gets EMA panel nod for expanded use in certain types of arthritis
May 20, 2022 9:37 AM ET Novartis AG (NVS)
By: Ravikash, SA News Editor
- A committee of the European Medicines Agency (EMA) recommended the approval for expanded use of Novartis' (NYSE:NVS) drug Cosentyx.
- https://seekingalpha.com/news/3841365-novartis-cosentyx-gets-ema-panel-nod-for-expanded-use-in-certain-types-of-arthritis
- https://seekingalpha.com/symbol/NVS
- https://www.cosentyx.com/
- https://www.novartis.com/
Vidaza (azacitidine injection)
Bristol-Myers Squibb's azacitidine granted additional indication for juvenile leukemia
May 20, 2022 4:54 PM ET
Bristol-Myers Squibb Company (BMY)
By: Jonathan Block, SA News Editor
- The U.S. FDA has approved Bristol-Myers Squibb (NYSE:BMY) subsidiary Celgene's Vidaza (azacitidine injection) for pediatric patients with newly diagnosed juvenile myelomonocytic leukemia.
- https://seekingalpha.com/news/3841501-bristol-myers-squibbs-azacitidine-granted-additional-indication-for-juvenile-leukemia
- https://seekingalpha.com/symbol/BMY
- https://www.bms.com/
- https://packageinserts.bms.com/pi/pi_vidaza.pdf
XELJANZ- tofacitinib tablet, film coated
Xeljanz
Opinion
On 19 May 2022, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending a change to the terms of the marketing authorisation for Xeljanz prolonged-release tablets.1 The marketing authorisation holder for this medicinal product is Pfizer Europe MA EEIG.
The CHMP adopted a new indication for the treatment of ankylosing spondylitis. The full indications for Xeljanz prolonged-release tablets will therefore be as follows:2
Rheumatoid arthritis
Tofacitinib in combination with methotrexate (MTX) is indicated for the treatment of moderate to severe active rheumatoid arthritis (RA) in adult patients who have responded inadequately to, or who are intolerant to one or more disease-modifying antirheumatic drugs (DMARDs) (see section 5.1). Tofacitinib can be given as monotherapy in case of intolerance to MTX or when treatment with MTX is inappropriate (see sections 4.4 and 4.5).
Psoriatic arthritis
Tofacitinib in combination with MTX is indicated for the treatment of active psoriatic arthritis (PsA) in adult patients who have had an inadequate response or who have been intolerant to a prior disease modifying antirheumatic drug (DMARD) therapy (see section 5.1).
Ankylosing spondylitis
Tofacitinib is indicated for the treatment of adult patients with active ankylosing spondylitis (AS) who have responded inadequately to conventional therapy.
Detailed recommendations for the use of this product will be described in the updated summary of product characteristics (SmPC), which will be published in the revised European public assessment report (EPAR), and will be available in all official European Union languages after a decision on this change to the marketing authorisation has been granted by the European Commission.
EMA panel recommends Pfizer's Xeljanz for ankylosing spondylitis
May 20, 2022 9:59 AM ET
By: Jonathan Block, SA News Editor
- An advisory panel to the European Medicines Agency (EMA) is recommending approval of Pfizer's (NYSE:PFE) JAK inhibitor Xeljanz (tofacitinib) for ankylosing spondylitis.
- https://seekingalpha.com/symbol/PFE
- https://www.xeljanz.com/
LUPKYNIS™
Aurinia Presents Results from the Two-Year AURORA 2 Continuation Study at the 2022 European Renal Association (ERA) Congress
DOWNLOAD AS PDF MAY 20, 2022
Use of LUPKYNIS was safe and well tolerated in patients for up to three years of treatment, with no new safety signals
In AURORA 2, long-term treatment with LUPKYNIS led to a clinically relevant preservation of kidney function in LN patients
A pooled analysis from the AURA-LV and AURORA 1 studies, also presented at ERA, showed early treatment response to LUPKYNIS with reductions in proteinuria across lupus nephritis biopsy classes
VICTORIA, British Columbia--(BUSINESS WIRE)-- Aurinia Pharmaceuticals Inc. (NASDAQ:AUPH) (Aurinia or the Company), a biopharmaceutical company committed to delivering therapeutics that change the course of autoimmune disease, today presented for the first time the results of the AURORA 2 continuation study evaluating the long-term safety and tolerability of LUPKYNIS™ (voclosporin) for the treatment of adults with active lupus nephritis (LN), a serious complication in patients with systemic lupus erythematosus (SLE). The results were presented during an oral session at the 59th European Renal Association (ERA) Congress, held in Paris and virtually May 19-22, 2022.
The AURORA 2 study assessed long-term safety and tolerability of voclosporin compared to placebo (both taken in combination with mycophenolate mofetil (MMF) and low-dose oral steroids) in patients with LN receiving treatment for an additional 24 months following completion of one year on treatment in the AURORA 1 study. The primary endpoint was safety and included assessments of adverse events, deaths, and hematological assessments. Secondary endpoints include renal response, renal flare, renal outcomes, and changes in urine protein to creatinine ratio (UPCR) and estimated glomerular filtration rate (eGFR).
“Lupus nephritis is a severe complication of lupus that will occur in up to half of patients diagnosed with SLE,” said Y.K. Onno Teng, M.D., Ph.D., Leiden University Medical Center, Leiden, The Netherlands, presenting author of the AURORA 2 ERA presentation and principal study investigator. “These results demonstrated that patients on long-term voclosporin therapy continue to experience clinically relevant benefits, including significant preservation of kidney function long-term, with no unexpected, new safety signals.”
Voclosporin was well tolerated during the study period with a similar safety profile to control and no unexpected safety signals. Specific findings included:
- Overall rates of serious adverse events were similar in both the control (28.0%) and voclosporin (26.0%) arms and there was no increase in infectious events.
- Generally adverse events were low and decreased over time on treatment.
- Significant and meaningful reductions in proteinuria achieved in AURORA 1 were maintained.
- In AURORA 2, there was a significant difference in eGFR slope in favor of voclosporin (-0.2 mL/min/1.73 m2) compared to control arm (-5.4 mL/min/1.73 m2).
- There was a small, expected, and early decrease in mean eGFR in the first four weeks of treatment in AURORA 1, after which eGFR remained stable through the end of AURORA 2.
- The mean UPCR was lower in the voclosporin-treated groups at all time points during the three years.
- There were four deaths in the active control group during AURORA 2, while none in the voclosporin-treated group.
“When treating patients with lupus nephritis, the ultimate goal is to preserve kidney function,'' said Neil Solomons, M.D., Chief Medical Officer at Aurinia. “We’re thrilled to build out the body of data that reinforces voclosporin as a safe and effective treatment option for lupus nephritis patients over the long term.”
A post-hoc analysis of pooled data from the AURA-LV and AURORA 1 studies was also presented during an oral session at the ERA Congress and examined the impact of voclosporin on the time to proteinuria reduction by biopsy class. The analysis reviewed pure class III, class IV, and class V biopsy groups and showed patients treated with voclosporin in addition to MMF and low dose steroids achieved earlier reductions in proteinuria across biopsy classes. These results support the efficacy results observed in the AURA-LV and AURORA 1 trials respectively, indicating a faster time to response when voclosporin is added to MMF and low-dose steroid treatment.
“The breadth of the AURORA clinical program provides important insights demonstrating the efficacy of voclosporin for the treatment of lupus nephritis across varying patient types," said Anca Askanase, M.D., M.P.H., Columbia University Medical Center and presenting author of the AURA-LV and AURORA 1 pooled analysis. "This analysis strengthens the clinical evidence supporting voclosporin as an important option that physicians can use in addition to the current standard of care treatment regimen to rapidly control lupus nephritis and protect kidney function.”
AURORA 2 Study Design
AURORA 2 (NCT03597464) is a Phase 3 randomized, double-blind, placebo-controlled clinical trial to assess the long-term safety and tolerability of voclosporin, in addition to MMF/steroids. Patients who completed 12 months of treatment in the Phase 3 AURORA 1 study were eligible to enroll in the AURORA 2 continuation study with the same randomized treatment of voclosporin at 23.7 mg twice daily or placebo, in combination with MMF at 1 g twice daily with low-dose oral steroids, for up to an additional 24 months. A total of 216 LN patients continued into AURORA 2, with 116 patients in the voclosporin-treated group and 100 patients in the active control group.
About AURORA 1
The AURORA 1 study was a 52-week study designed to evaluate the efficacy and safety of LUPKYNIS (23.7 mg twice daily) when added to background therapy of MMF and corticosteroids tapered to a low dose, compared to background therapy alone in an ethnically and racially diverse patient population with active LN. Three hundred fifty-seven patients with a diagnosis of SLE and LN according to the American College of Rheumatology criteria and a kidney biopsy within two years that showed Class III, IV and/or V LN were enrolled. AURORA 1 met its primary endpoint, achieving statistically superior complete renal response rates of 41% in the LUPKYNIS group versus 23% in the control group (odds ratio [OR] 2.65, 95% confidence interval [CI] 1.64-4.27; p < 0.0001). LUPKYNIS also achieved statistical significance in all pre-specified hierarchical secondary endpoints, including reduced time to 50% reduction from baseline in UPCR and time to UPCR <0.5 mg/mg compared to control. The primary endpoint was complete renal response at 52 weeks defined as urine protein creatinine ratio (UPCR) ≤0.5 mg/mg, with stable renal function (defined as estimated glomerular filtration rate [eGFR] ≥60 mL/min/1.73 m2 or no confirmed decrease from baseline in eGFR of >20%), no administration of rescue medication, and no more than 10 mg prednisone equivalent per day for three or more consecutive days or for seven or more days during Weeks 44 through 52. LUPKYNIS was well tolerated with no unexpected safety signals. Serious adverse events (SAEs) were reported in 21% of those treated with LUPKYNIS and in 21% of those in the control group. Results from the completed AURORA 1 study (NCT03021499) were published in May 2021 in The Lancet.
About AURA-LV
The AURA–LV study (Aurinia Urinary protein Reduction in Active Lupus with Voclosporin) was a 48-week study comparing the efficacy of two doses of voclosporin added to current standard of care of MMF against standard of care with placebo in achieving CR in patients with active LN. All arms also received low doses of steroids as background therapy. 265 patients were enrolled at centers in 20 countries worldwide. On entry to the study, patients were required to have a diagnosis of LN according to established diagnostic criteria (American College of Rheumatology) and clinical and biopsy features indicative of highly active nephritis. The 24-week primary and secondary endpoints were released in Q3 2016 where the primary and all secondary endpoints were met. Complete remission (CR) is a composite endpoint that includes: confirmed UPCR of ≤0.5 mg/mg; normal, stable renal function (≥60 mL/min/1.73m2 or no confirmed decrease from baseline in eGFR of ≥20%); presence of sustained, low dose steroids (≤10mg prednisone from week 16-24); and no administration of rescue medications. Partial remission (PR) in the trial is measured by a ≥50% reduction in UPCR with no concomitant use of rescue medication.
About LUPKYNIS
LUPKYNIS™ is the first FDA-approved oral medicine for the treatment of adult patients with active LN. LUPKYNIS is a novel, structurally modified calcineurin inhibitor (CNI) with a dual mechanism of action, acting as an immunosuppressant through inhibition of T-cell activation and cytokine production and promoting podocyte stability in the kidney. The recommended starting dose of LUPKYNIS is three capsules twice daily with no requirement for serum drug monitoring. Dose modifications can be made based on Aurinia’s proprietary personalized eGFR-based dosing protocol. Boxed Warning, warnings and precautions for LUPKYNIS are consistent with those of other CNI-immunosuppressive treatments.
INDICATIONS
LUPKYNIS is indicated in combination with a background immunosuppressive therapy regimen for the treatment of adult patients with active LN. Limitations of Use: Safety and efficacy of LUPKYNIS have not been established in combination with cyclophosphamide. Use of LUPKYNIS is not recommended in this situation.
Please see Prescribing Information, including Boxed Warning, and Medication Guide for LUPKYNIS
View source version on businesswire.com: https://www.businesswire.com/news/home/20220520005341/en/
Source: Aurinia Pharmaceuticals Inc.
Released May 20, 2022
Aurinia Pharmaceuticals touts long-term benefit of Lupkynis for lupus
May 20, 2022 12:32 PM ET
Aurinia Pharmaceuticals Inc. (AUPH)
By: Jonathan Block, SA News Editor8 Comments
- Aurinia Pharmaceuticals (NASDAQ:AUPH) said that data from a study showed that its lupus nephritis treatment Lupkynis (voclosporin) led to preservation of kidney function over three years of treatment.
- https://seekingalpha.com/news/3841424-aurinia-pharmaceuticals-touts-long-term-benefit-of-lupkynis-for-lupus
- https://seekingalpha.com/symbol/AUPH
- https://www.lupkynis.com/
- https://www.auriniapharma.com/
biotecMAX™ Feb/Mar/APR/May 2022 Insight
OLUMIANT® (baricitinib)
CHMP Recommends Approval of Lilly and Incyte's OLUMIANT® (baricitinib) as the First and Only Centrally-Authorized Treatment for Adults with Severe Alopecia Areata (AA)
May 20, 2022Download PDF
INDIANAPOLIS, May 20, 2022 /PRNewswire/ -- Eli Lilly and Company (NYSE: LLY) and Incyte (NASDAQ:INCY) announced today that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion for OLUMIANT® (baricitinib) for the treatment of adults with severe alopecia areata (AA).
This opinion marks the first step toward European regulatory approval of OLUMIANT for patients with severe AA, and it is now referred to the European Commission for final action. If approved, OLUMIANT would be the first centrally-authorized oral treatment and first JAK inhibitor for patients with severe AA in the European Union. The European Commission's decision is expected in the next one to two months.
"Alopecia areata is an often-misunderstood autoimmune disease that can lead to unpredictable hair loss, ranging from bald patches to complete loss of all hair. The disease carries significant psychosocial burden and can impact patients of any race, ethnicity, or age, with many experiencing alopecia in their early to mid-20s," said Bianca Maria Piraccini, M.D., Ph.D., professor and head of the Dermatology Unit at the University of Bologna. "As there has never been a centrally-authorized therapy for alopecia areata, I'm delighted about Lilly's potential to provide this oral medicine with statistically significant and clinically meaningful Phase 3 clinical trial results for adults with severe alopecia areata across Europe."
The positive opinion was based on Lilly's Phase 3 BRAVE-AA1 and BRAVE-AA2 trials evaluating the efficacy and safety of OLUMIANT in 1,200 patients with severe AA, the largest Phase 3 clinical trial program with completed primary endpoints. Severe AA was defined as having a Severity of Alopecia Tool (SALT) score ≥50 (≥50% scalp hair loss). The primary endpoint was the proportion of patients achieving SALT ≤20 (i.e., 80% or more scalp hair coverage) at Week 36. Across both studies, 1 out of 3 patients treated with OLUMIANT 4-mg achieved 80% or more scalp hair coverage (BRAVE-AA1=35.2% [n=99]; BRAVE-AA2=32.5% [n=76]), compared to 1 out of 20 patients (5.3%, n=10) and 1 out of 50 patients (2.6%, n=4) taking placebo in BRAVE-AA1 and BRAVE-AA2, respectively (p≤0.001 for all comparisons to placebo).
Achievement of full regrowth or regrowth with minimal gaps in eyebrow and eyelash hair was also seen at 36 weeks with OLUMIANT 4-mg for 1 in 3 patients who at baseline had significant gaps or no notable eyebrows or eyelashes, as compared to patients taking placebo (BRAVE-AA1: 4-mg dose: eyebrow=31.4% [n=59]; eyelash=33.5% [n=56]; placebo: eyebrow=3.2% [n=4]; eyelash=3.1% [n=3]; BRAVE-AA2: 4-mg dose: eyebrow=34.8% [n=56]; eyelash=34.3% [n=48]; placebo: eyebrow=4.5% [n=5]; eyelash=5.6% [n=5]; p≤0.001 for all comparisons to placebo). Eyebrow and eyelash hair loss was evaluated using the Clinician-Reported Outcome (ClinRO) Measure for Eyebrow Hair Loss™ and ClinRO Measure for Eyelash Hair Loss™ – novel, clinically-validated tools developed by Lilly.
The Phase 3 BRAVE-AA clinical program also evaluated the safety profile of OLUMIANT, and no new safety signals were observed. Few patients discontinued treatment due to adverse events (2.6% or less across both studies), and the majority of treatment-emergent adverse events were mild or moderate in severity.
"We're proud of today's CHMP opinion as it reflects our commitment to immunological diseases with high unmet need," said Patrik Jonsson, Lilly senior vice president, president of Lilly Immunology and Lilly USA, and chief customer officer. "This is a significant step for OLUMIANT on the path to becoming the first and only centrally-authorized medicine in Europe for adults with severe alopecia areata. We eagerly anticipate additional regulatory decisions around the world this year."
In February 2022, the U.S. Food and Drug Administration (FDA) granted priority review for OLUMIANT in adults with severe AA. Lilly expects additional regulatory decisions in the U.S. and Japan in 2022.
About OLUMIANT®
OLUMIANT, a once-daily, oral JAK inhibitor, was discovered by Incyte and licensed to Lilly. It is approved in the U.S. and more than 75 countries as a treatment for adults with moderate to severe rheumatoid arthritis and is approved in more than 50 countries, including the European Union and Japan, for the treatment of adult patients with moderate to severe atopic dermatitis who are candidates for systemic therapy. FDA approval was granted for OLUMIANT for the treatment of certain hospitalized adult patients with COVD-19 in May 2022. Marketing authorization for OLUMIANT in COVID-19 has been granted in six other countries including Japan and Switzerland. Baricitinib is authorized for emergency use in eight countries. Over 400,000 patients have been treated with OLUMIANT for approved indications, in addition to nearly one million patients with COVID-19 worldwide. The U.S. FDA-approved labeling for OLUMIANT includes a Boxed Warning for Serious Infections, Mortality, Malignancy, Major Adverse Cardiovascular Events, and Thrombosis. See the full Prescribing Information here. OLUMIANT is being investigated in alopecia areata (AA), juvenile idiopathic arthritis (JIA) and in pediatric patients with atopic dermatitis (AD).
In December 2009, Lilly and Incyte announced an exclusive worldwide license and collaboration agreement for the development and commercialization of OLUMIANT and certain follow-on compounds for patients with inflammatory and autoimmune diseases.
Indications and Usage for OLUMIANT (baricitinib) tablets (in the United States)
OLUMIANT is indicated for the treatment of adult patients with moderately to severely active rheumatoid arthritis who have had an inadequate response to one or more tumor necrosis factor (TNF) antagonist therapies. Limitations of Use: Not recommended for use in combination with other JAK inhibitors, biologic disease-modifying antirheumatic drugs (DMARDs), or with potent immunosuppressants such as azathioprine and cyclosporine.
OLUMIANT is indicated for the treatment of coronavirus disease 2019 (COVID-19) in hospitalized adults requiring supplemental oxygen, non-invasive or invasive mechanical ventilation, or extracorporeal membrane oxygenation (ECMO).
Please click to access full Prescribing Information, including Boxed Warning about Serious Infections, Mortality, Malignancy, Major Adverse Cardiovascular Events, and Thrombosis, and Medication Guide.
To learn more, visit Lilly.com and Lilly.com/newsroom or follow us on Facebook, Instagram, Twitter and LinkedIn.
About Incyte
Incyte is a Wilmington, Delaware-based, global biopharmaceutical company focused on finding solutions for serious unmet medical needs through the discovery, development and commercialization of proprietary therapeutics. For additional information on Incyte, please visit Incyte.com and follow @Incyte.
OLUMIANT® is a registered trademark owned or licensed by Eli Lilly and Company, its subsidiaries, or affiliates.
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SOURCE Eli Lilly and Company; Incyte
Lilly, Incyte's Olumiant get EMA panel nod for hair loss disorder alopecia areata
May 20, 2022 9:56 AM ET Incyte Corporation (INCY), LLY
By: Ravikash, SA News Editor
- A committee of the European Medicines Agency (EMA) recommended the approval of Eli Lilly (NYSE:LLY) and Incyte's (NASDAQ:INCY) Olumiant to treat adults with severe alopecia areata (AA).
- https://seekingalpha.com/news/3841375-lilly-incytes-olumiant-get-ema-panel-nod-for-hair-loss-disorder-alopecia-areata
- https://www.olumiant.com/
- https://seekingalpha.com/symbol/LLY
- https://seekingalpha.com/symbol/INCY
Sarclisa® (isatuximab)
PRESS RELEASES May 15 2022
Press Release: Sarclisa® (isatuximab) combination provides unprecedented median progression free survival in patients with relapsed multiple myeloma receiving a proteasome inhibitor therapy
Sarclisa® (isatuximab) combination provides unprecedented median progression free survival in patients with relapsed multiple myeloma receiving a proteasome inhibitor therapy
- Latest results of the Phase 3 IKEMA trial demonstrate the longest median progression free survival (mPFS) on a proteasome inhibitor backbone in patients who relapsed after a prior therapy, including lenalidomide
- The median progression free survival, increased from 19.2 months to 35.7 months when Sarclisa was added to carfilzomib and dexamethasone
- Further analysis, following U.S. Food and Drug Administration recommendations on censoring rules, showed mPFS increased from 20.8 to 41.7 months when Sarclisa was added to carfilzomib and dexamethasone
PARIS, May 15, 2022. Latest results from the Phase 3 IKEMA clinical trial evaluating Sarclisa® (isatuximab) in combination with carfilzomib and dexamethasone (Kd) demonstrated a median progression free survival (mPFS) of 35.7 months (Hazard Ratio HR 0.58; 95% Confidence Interval CI: 25.8 to 44.0; n=179), compared to 19.2 months in patients treated with Kd alone (95% CI: 15.8 to 25.1; n=123), as evaluated by an Independent Review Committee. These results, presented at the Controversies in Multiple Myeloma World Congress, represent the longest mPFS among studies investigating a proteasome inhibitor backbone in the second-line setting for the treatment of relapsed multiple myeloma (MM). These data will also be presented at the European Society for Medical Oncology on May 19.
Philippe Moreau, MD
Head of the Department of Hematology, University Hospital of Nantes, France
“The increase in progression free survival, observed consistently across all subgroups, when adding Sarclisa to carfilzomib and dexamethasone is remarkable in patients with relapsed multiple myeloma in a proteasome inhibitor combination. Relapse is common in multiple myeloma, creating the need for differentiated second-line treatments that provide patients a longer period of time without disease progression. This updated analysis reinforces the potential for Sarclisa to become a new standard of care for patients with relapsed multiple myeloma.”
A PFS analysis following the U.S. Food and Drug Administration recommendations on censoring rules, as applied in the approved U.S. prescribing information, showed an mPFS of 41.7 months for Sarclisa added to Kd (Sarclisa combination therapy) compared to 20.8 months in patients treated with Kd alone (HR 0.59; 95% CI: 27.1 to Not Calculable NC).
Time to next treatment for patients treated with Sarclisa combination therapy was 44.9 months (HR 0.55; 95% CI: 31.6 to NC) versus those treated with Kd alone at 25 months (95% CI: 17.9 to 31.3). Time to next treatment measured the interval from the date of randomization1 to the date of commencement of the next line of therapy, thereby allowing for measurement of the period of therapeutic benefit.2
Peter C. Adamson, MD
Global Head of Oncology Clinical Development and Pediatric Innovation at Sanofi
“To observe progression free survival of more than three years in patients with relapsed multiple myeloma when Sarclisa was added to a proteasome inhibitor backbone of therapy is unprecedented and reinforces our confidence in Sarclisa as a potential best in class anti-CD38 antibody.”
The safety and tolerability of Sarclisa observed in this analysis were consistent with the safety profile of Sarclisa in other clinical trials, with no new safety signals observed. For the Sarclisa combination therapy and Kd groups, the most common adverse events were infusion related reaction (45.8%, 3.3%), diarrhea (39.5%, 32%), hypertension (37.9%, 35.2%), upper respiratory tract infection (37.3%, 27%), fatigue (31.6%, 20.5%), dyspnoea (30.5%, 22.1%), pneumonia (27.1%, 21.3%), back pain (25.4%, 21.3%), insomnia (25.4%, 24.6%), and bronchitis (24.3%, 12.3%). Treatment exposure in the Sarclisa combination therapy arm was 30 weeks longer than in the control arm. Treatment emergent adverse events (TEAEs) of ≥ Grade 3 were reported in 83.6% of patients treated with Sarclisa combination therapy and in 73% of those treated with Kd alone. Serious TEAEs were higher in the Sarclisa combination therapy arm versus Kd alone (70.1% versus 59.8%). No difference was observed after exposure adjustment.”
These results will be discussed with regulatory authorities at a future date.
About the IKEMA trial
The randomized, multi-center, open label Phase 3 IKEMA clinical trial enrolled 302 patients with relapsed MM across 69 centers spanning 16 countries. All study participants had received one to three prior anti-myeloma therapies. During the trial, Sarclisa was administered through an intravenous infusion at a dose of 10mg/kg once weekly for four weeks, then every other week for 28-day cycles in combination with carfilzomib twice weekly at the 20/56mg/m2 dose and dexamethasone at the standard dose for the duration of treatment. The primary endpoint of IKEMA was progression free survival. Secondary endpoints included overall response rate, the rate of complete response or better, the rate of very good partial response or better, rate of minimal residual disease-negativity, overall survival and safety.3
About Sarclisa
Sarclisa is a monoclonal antibody that targets a specific epitope on the CD38 receptor on multiple myeloma (MM) cells. It is designed to work through multiple mechanisms of action including programmed tumor cell death (apoptosis) and immunomodulatory activity. CD38 is highly and uniformly expressed on the surface of MM cells, making it a potential target for antibody-based therapeutics such as Sarclisa.
Based on the Phase 3 ICARIA-MM study, Sarclisa is approved in a number of countries, including the U.S. and EU, in combination with pomalidomide and dexamethasone for the treatment of patients with relapsed refractory MM (RRMM) who have received ≥2 prior therapies, including lenalidomide and a proteasome inhibitor. Based on the Phase 3 IKEMA study, Sarclisa is also approved in multiple countries in combination with carfilzomib and dexamethasone, including in the U.S. for the treatment of patients with RRMM who have received 1–3 prior lines of therapy and in the European Union for patients with MM who have received at least 1 prior therapy. In the U.S., the generic name for Sarclisa is isatuximab-irfc, with irfc as the suffix designated in accordance with Nonproprietary Naming of Biological Products Guidance for Industry issued by the U.S. Food and Drug Administration (FDA).
Sarclisa continues to be evaluated in multiple ongoing Phase 3 clinical trials in combination with current standard treatments across the MM treatment continuum. It is also under investigation for the treatment of other hematologic malignancies and solid tumors. The safety and efficacy of these additional uses have not been reviewed by any regulatory authority worldwide.
For more information on Sarclisa clinical trials, please visit www.clinicaltrials.gov.
Sanofi's Sarclisa combo reduces risk of multiple myeloma getting worse by ~3 years in study
May 16, 2022 6:12 AM ET Sanofi (SNY)
By: Ravikash, SA News Editor
Sanofi (NASDAQ:SNY) said new data from a late stage study of its multiple myeloma therapy Sarclisa, in combination with other drugs, showed reducing the risk of disease getting worse by a median of nearly three years.
https://seekingalpha.com/symbol/SNY
Verquvo™ (vericiguat)
Thursday - May 19, 2022
Not intended for U.S. and UK Media
Verquvo™ (vericiguat) approved in China to treat patients with chronic heart failure and reduced ejection fraction
Studied in adults with symptomatic chronic heart failure and reduced ejection fraction who are stabilized after a recent decompensation event with IV therapy / Approved in China to reduce the risk of heart failure (HF) hospitalization or the need for intravenous (IV) diuretics in emergency care / Approximately 13.7 million people in China live with heart failure(1)
Berlin, Germany, May 19, 2022 – Bayer announced today that China’s National Medical Products Administration (NMPA) has approved vericiguat under the brand name Verquvo™. Verquvo (2.5 mg, 5 mg, and 10 mg), a soluble guanylate cyclase (sGC) stimulator, is indicated in China to reduce the risk of heart failure (HF) hospitalization or requiring intravenous (IV) diuretics in emergency, in adults with symptomatic chronic HF and reduced ejection fraction (less than 45%) who are stabilized after a recent decompensation event with IV therapy. It works differently to existing heart failure treatments, providing a specific approach to managing chronic heart failure after a recent decompensation event with IV therapy, also known as a worsening heart failure event.1,2,3
“The approval of Verquvo is an important milestone for heart failure patients across China, providing a new option to help break the cycle of worsening heart failure events. Such events can lead to a downward spiral for many patients, resulting in repeated hospitalization,” said Dr. Michael Devoy, Head of Medical Affairs & Pharmacovigilance of Bayer AG’s Pharmaceuticals Division and Bayer Chief Medical Officer. “With each hospital visit, the risk of death increases along with the emotional toll carried by patients and their families. For this reason, Bayer is proud to provide clinicians with access to Verquvo, to help improve outcomes and help alleviate the burden faced by patients living with chronic heart failure in China.”
Current therapies block the harmful effects of the natural neurohormonal systems that are activated by the myocardial and vascular dysfunction present in heart failure. Verquvo works through a different mode of action.2,4 It specifically restores the deficient NO-sGC-cGMP pathway, which plays a critical role in the progression of heart failure and aggravating its symptoms.4 Verquvo was studied and approved on top of standard-of-care.
Verquvo has been approved in the U.S, the EU, Japan, and many other countries worldwide. Multiple other submissions for marketing authorizations are ongoing worldwide.
Verquvo is being jointly developed with MSD (a tradename of Merck & Co., Inc., Kenilworth, NJ, USA).
About Verquvo™ (vericiguat)
Vericiguat 2.5 mg, 5 mg, and 10 mg is an oral once daily stimulator of soluble guanylate cyclase (sGC), an important enzyme in the nitric oxide (NO) signaling pathway.5 When NO binds to sGC, the enzyme catalyzes the synthesis of intracellular cyclic guanosine monophosphate (cGMP), a second messenger that plays a role in the regulation of vascular tone, cardiac contractility, and cardiac remodeling.5 Heart failure is associated with impaired synthesis of NO and decreased activity of sGC, which may contribute to myocardial and vascular dysfunction.5 By directly stimulating sGC, independently of and synergistically with NO, vericiguat augments levels of intracellular cGMP, leading to smooth muscle relaxation and vasodilation.4 In the EU Verquvo is indicated for symptomatic chronic heart failure in adult patients with reduced ejection fraction who are stabilized after a recent decompensation event requiring intravenous (IV) therapy.
About the Worldwide Collaboration between Bayer and MSD
Since October 2014, Bayer and MSD have pursued a worldwide collaboration in the field of sGC modulators. The collaboration brings together two leading companies that have stated their intent to fully evaluate this therapeutic class in areas of unmet medical need. The vericiguat program is being co-developed by Bayer and MSD. MSD has the commercial rights to vericiguat in the U.S. and Bayer has the exclusive commercial rights in the rest of world. The companies share equally the costs of the development of vericiguat.
R&D expenses before special items amounted to 5.3 billion euros. For more information, go to www.bayer.com.
Bayer, Merck's Verquvo gets approval in China to treat certain patients with heart failure
May 19, 2022 5:08 AM ET
Bayer Aktiengesellschaft (BAYRY), BAYZFMRK
By: Ravikash, SA News Editor
- China's National Medical Products Administration approved Bayer (OTCPK:BAYRY) (OTCPK:BAYZF) and Merck's (MRK) Verquvo to treat certain patients with heart failure (HF).
- https://seekingalpha.com/news/3840751-bayer-mercks-verquvo-gets-approval-in-china-to-treat-certain-patients-with-heart-failure
- https://seekingalpha.com/symbol/BAYRY
- https://seekingalpha.com/symbol/BAYZF
- https://seekingalpha.com/symbol/MRK
- https://www.verquvo-us.com/
- https://www.merck.com/
KEYTRUDA® (pembrolizumab)
Merck Receives Positive EU CHMP Opinion for KEYTRUDA® (pembrolizumab) as Adjuvant Treatment for Adult and Adolescent (≥12 Years of Age) Patients With Stage IIB or IIC Melanoma Following Complete Resection
May 20, 2022 8:15 am ET
Opinion granted based on positive results from the Phase 3 KEYNOTE-716 trial
If approved, KEYTRUDA would be the first anti-PD-1 immunotherapy treatment option for patients 12 years and older in the EU across stage IIB, IIC and III melanoma following complete resection
RAHWAY, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending approval of KEYTRUDA, Merck’s anti-PD-1 therapy, as monotherapy for the adjuvant treatment of adults and adolescents aged 12 years and older with stage IIB or IIC melanoma and who have undergone complete resection. Additionally, the CHMP recommended expanding the indications for KEYTRUDA in advanced (unresectable or metastatic) melanoma and stage III melanoma (as adjuvant treatment following complete resection) to include adolescent patients aged 12 years and older.
“Based on the results of the KEYNOTE-716 trial, KEYTRUDA has shown a significant improvement in recurrence-free survival and distant metastasis-free survival for these patients with resected stage IIB or IIC melanoma,” said Dr. Scot Ebbinghaus, vice president, global clinical development, Merck Research Laboratories. “The CHMP’s positive recommendation brings us one step closer to providing patients 12 years and older in the European Union with a new option for resected stage IIB or IIC melanoma that can reduce the risk of their cancer returning.”
The positive opinion was granted based on results from the Phase 3 KEYNOTE-716 trial, which demonstrated a statistically significant improvement in recurrence-free survival with KEYTRUDA compared to placebo (HR=0.65 [95% CI, 0.46-0.92]; p=0. 0.00658) in patients 12 years and older with stage IIB and IIC melanoma following complete resection. Earlier this year, Merck reported that KEYNOTE-716 also met its key secondary endpoint of distant metastasis-free survival. These results will be featured in a late-breaking oral presentation at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting on June 5 (Abstract #LBA9500).
The CHMP’s recommendation will now be reviewed by the European Commission for marketing authorization in the European Union, and a final decision is expected in the second or third quarter of 2022.
About Merck’s research in melanoma
Melanoma, the most serious form of skin cancer, is characterized by the uncontrolled growth of pigment-producing cells. The rates of melanoma have been rising over the past few decades, with nearly 325,000 new cases of melanoma diagnosed worldwide in 2020, and melanoma is the leading cause of skin cancer deaths, with more than 57,000 deaths from the disease worldwide in 2020. In Europe, it is estimated there were more than 150,000 new cases of melanoma diagnosed and more than 26,000 deaths from the disease in 2020.
The recurrence rates for resected melanoma are estimated to be 32-46% for patients with stage IIB and IIC disease and 39-74% for patients with stage III disease. The five-year survival rates are estimated to be 87% for stage IIB, 82% for stage IIC, 93% for stage IIIA, 83% for stage IIIB, 69% for stage IIIC and 32% for stage IIID.
Merck is committed to delivering meaningful advances for patients with melanoma with KEYTRUDA and to continuing research in skin cancers through a broad clinical development program across investigational and approved medicines. KEYTRUDA has been established as an important treatment option for the adjuvant treatment of adult patients with resected stage III melanoma and is approved in over 90 countries based on the results from EORTC1325/KEYNOTE-054. KEYTRUDA is also approved worldwide for the treatment of patients with unresectable or metastatic melanoma.
About KEYTRUDA® (pembrolizumab) injection, 100 mg
KEYTRUDA is an anti-programmed death receptor-1 (PD-1) therapy that works by increasing the ability of the body’s immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.
Merck has the industry’s largest immuno-oncology clinical research program. There are currently more than 1,700 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.
Selected KEYTRUDA® (pembrolizumab) Indications in the U.S.
Melanoma
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.
KEYTRUDA is indicated for the adjuvant treatment of adult and pediatric (12 years and older) patients with stage IIB, IIC, or III melanoma following complete resection.
See additional selected KEYTRUDA indications in the U.S. after the Selected Important Safety Information.
Selected Important Safety Information for KEYTRUDA
Severe and Fatal Immune-Mediated Adverse Reactions
KEYTRUDA is a monoclonal antibody that belongs to a class of drugs that bind to either the PD-1 or the PD-L1, blocking the PD-1/PD-L1 pathway, thereby removing inhibition of the immune response, potentially breaking peripheral tolerance and inducing immune-mediated adverse reactions. Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue, can affect more than one body system simultaneously, and can occur at any time after starting treatment or after discontinuation of treatment. Important immune-mediated adverse reactions listed here may not include all possible severe and fatal immune-mediated adverse reactions.
Monitor patients closely for symptoms and signs that may be clinical manifestations of underlying immune-mediated adverse reactions. Early identification and management are essential to ensure safe use of anti–PD-1/PD-L1 treatments. Evaluate liver enzymes, creatinine, and thyroid function at baseline and periodically during treatment. For patients with TNBC treated with KEYTRUDA in the neoadjuvant setting, monitor blood cortisol at baseline, prior to surgery, and as clinically indicated. In cases of suspected immune-mediated adverse reactions, initiate appropriate workup to exclude alternative etiologies, including infection. Institute medical management promptly, including specialty consultation as appropriate.
Withhold or permanently discontinue KEYTRUDA depending on severity of the immune-mediated adverse reaction. In general, if KEYTRUDA requires interruption or discontinuation, administer systemic corticosteroid therapy (1 to 2 mg/kg/day prednisone or equivalent) until improvement to Grade 1 or less. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Consider administration of other systemic immunosuppressants in patients whose adverse reactions are not controlled with corticosteroid therapy.
Immune-Mediated Pneumonitis
KEYTRUDA can cause immune-mediated pneumonitis. The incidence is higher in patients who have received prior thoracic radiation. Immune-mediated pneumonitis occurred in 3.4% (94/2799) of patients receiving KEYTRUDA, including fatal (0.1%), Grade 4 (0.3%), Grade 3 (0.9%), and Grade 2 (1.3%) reactions. Systemic corticosteroids were required in 67% (63/94) of patients. Pneumonitis led to permanent discontinuation of KEYTRUDA in 1.3% (36) and withholding in 0.9% (26) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, 23% had recurrence. Pneumonitis resolved in 59% of the 94 patients.
Pneumonitis occurred in 8% (31/389) of adult patients with cHL receiving KEYTRUDA as a single agent, including Grades 3-4 in 2.3% of patients. Patients received high-dose corticosteroids for a median duration of 10 days (range: 2 days to 53 months). Pneumonitis rates were similar in patients with and without prior thoracic radiation. Pneumonitis led to discontinuation of KEYTRUDA in 5.4% (21) of patients. Of the patients who developed pneumonitis, 42% interrupted KEYTRUDA, 68% discontinued KEYTRUDA, and 77% had resolution.
Immune-Mediated Colitis
KEYTRUDA can cause immune-mediated colitis, which may present with diarrhea. Cytomegalovirus infection/reactivation has been reported in patients with corticosteroid-refractory immune-mediated colitis. In cases of corticosteroid-refractory colitis, consider repeating infectious workup to exclude alternative etiologies. Immune-mediated colitis occurred in 1.7% (48/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (1.1%), and Grade 2 (0.4%) reactions. Systemic corticosteroids were required in 69% (33/48); additional immunosuppressant therapy was required in 4.2% of patients. Colitis led to permanent discontinuation of KEYTRUDA in 0.5% (15) and withholding in 0.5% (13) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, 23% had recurrence. Colitis resolved in 85% of the 48 patients.
Hepatotoxicity and Immune-Mediated Hepatitis
KEYTRUDA as a Single Agent
KEYTRUDA can cause immune-mediated hepatitis. Immune-mediated hepatitis occurred in 0.7% (19/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.4%), and Grade 2 (0.1%) reactions. Systemic corticosteroids were required in 68% (13/19) of patients; additional immunosuppressant therapy was required in 11% of patients. Hepatitis led to permanent discontinuation of KEYTRUDA in 0.2% (6) and withholding in 0.3% (9) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, none had recurrence. Hepatitis resolved in 79% of the 19 patients.
KEYTRUDA With Axitinib
KEYTRUDA in combination with axitinib can cause hepatic toxicity. Monitor liver enzymes before initiation of and periodically throughout treatment. Consider monitoring more frequently as compared to when the drugs are administered as single agents. For elevated liver enzymes, interrupt KEYTRUDA and axitinib, and consider administering corticosteroids as needed. With the combination of KEYTRUDA and axitinib, Grades 3 and 4 increased alanine aminotransferase (ALT) (20%) and increased aspartate aminotransferase (AST) (13%) were seen at a higher frequency compared to KEYTRUDA alone. Fifty-nine percent of the patients with increased ALT received systemic corticosteroids. In patients with ALT ≥3 times upper limit of normal (ULN) (Grades 2-4, n=116), ALT resolved to Grades 0-1 in 94%. Among the 92 patients who were rechallenged with either KEYTRUDA (n=3) or axitinib (n=34) administered as a single agent or with both (n=55), recurrence of ALT ≥3 times ULN was observed in 1 patient receiving KEYTRUDA, 16 patients receiving axitinib, and 24 patients receiving both. All patients with a recurrence of ALT ≥3 ULN subsequently recovered from the event.
Immune-Mediated Endocrinopathies
Adrenal Insufficiency
KEYTRUDA can cause primary or secondary adrenal insufficiency. For Grade 2 or higher, initiate symptomatic treatment, including hormone replacement as clinically indicated. Withhold KEYTRUDA depending on severity. Adrenal insufficiency occurred in 0.8% (22/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.3%), and Grade 2 (0.3%) reactions. Systemic corticosteroids were required in 77% (17/22) of patients; of these, the majority remained on systemic corticosteroids. Adrenal insufficiency led to permanent discontinuation of KEYTRUDA in <0.1% (1) and withholding in 0.3% (8) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement.
Hypophysitis
KEYTRUDA can cause immune-mediated hypophysitis. Hypophysitis can present with acute symptoms associated with mass effect such as headache, photophobia, or visual field defects. Hypophysitis can cause hypopituitarism. Initiate hormone replacement as indicated. Withhold or permanently discontinue KEYTRUDA depending on severity. Hypophysitis occurred in 0.6% (17/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.3%), and Grade 2 (0.2%) reactions. Systemic corticosteroids were required in 94% (16/17) of patients; of these, the majority remained on systemic corticosteroids. Hypophysitis led to permanent discontinuation of KEYTRUDA in 0.1% (4) and withholding in 0.3% (7) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement.
Thyroid Disorders
KEYTRUDA can cause immune-mediated thyroid disorders. Thyroiditis can present with or without endocrinopathy. Hypothyroidism can follow hyperthyroidism. Initiate hormone replacement for hypothyroidism or institute medical management of hyperthyroidism as clinically indicated. Withhold or permanently discontinue KEYTRUDA depending on severity. Thyroiditis occurred in 0.6% (16/2799) of patients receiving KEYTRUDA, including Grade 2 (0.3%). None discontinued, but KEYTRUDA was withheld in <0.1% (1) of patients.
Hyperthyroidism occurred in 3.4% (96/2799) of patients receiving KEYTRUDA, including Grade 3 (0.1%) and Grade 2 (0.8%). It led to permanent discontinuation of KEYTRUDA in <0.1% (2) and withholding in 0.3% (7) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement. Hypothyroidism occurred in 8% (237/2799) of patients receiving KEYTRUDA, including Grade 3 (0.1%) and Grade 2 (6.2%). It led to permanent discontinuation of KEYTRUDA in <0.1% (1) and withholding in 0.5% (14) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement. The majority of patients with hypothyroidism required long-term thyroid hormone replacement. The incidence of new or worsening hypothyroidism was higher in 1185 patients with HNSCC, occurring in 16% of patients receiving KEYTRUDA as a single agent or in combination with platinum and FU, including Grade 3 (0.3%) hypothyroidism. The incidence of new or worsening hypothyroidism was higher in 389 adult patients with cHL (17%) receiving KEYTRUDA as a single agent, including Grade 1 (6.2%) and Grade 2 (10.8%) hypothyroidism.
Type 1 Diabetes Mellitus (DM), Which Can Present With Diabetic Ketoacidosis
Monitor patients for hyperglycemia or other signs and symptoms of diabetes. Initiate treatment with insulin as clinically indicated. Withhold KEYTRUDA depending on severity. Type 1 DM occurred in 0.2% (6/2799) of patients receiving KEYTRUDA. It led to permanent discontinuation in <0.1% (1) and withholding of KEYTRUDA in <0.1% (1) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement.
Immune-Mediated Nephritis With Renal Dysfunction
KEYTRUDA can cause immune-mediated nephritis. Immune-mediated nephritis occurred in 0.3% (9/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.1%), and Grade 2 (0.1%) reactions. Systemic corticosteroids were required in 89% (8/9) of patients. Nephritis led to permanent discontinuation of KEYTRUDA in 0.1% (3) and withholding in 0.1% (3) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, none had recurrence. Nephritis resolved in 56% of the 9 patients.
Immune-Mediated Dermatologic Adverse Reactions
KEYTRUDA can cause immune-mediated rash or dermatitis. Exfoliative dermatitis, including Stevens-Johnson syndrome, drug rash with eosinophilia and systemic symptoms, and toxic epidermal necrolysis, has occurred with anti–PD-1/PD-L1 treatments. Topical emollients and/or topical corticosteroids may be adequate to treat mild to moderate nonexfoliative rashes. Withhold or permanently discontinue KEYTRUDA depending on severity. Immune-mediated dermatologic adverse reactions occurred in 1.4% (38/2799) of patients receiving KEYTRUDA, including Grade 3 (1%) and Grade 2 (0.1%) reactions. Systemic corticosteroids were required in 40% (15/38) of patients. These reactions led to permanent discontinuation in 0.1% (2) and withholding of KEYTRUDA in 0.6% (16) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, 6% had recurrence. The reactions resolved in 79% of the 38 patients.
Other Immune-Mediated Adverse Reactions
The following clinically significant immune-mediated adverse reactions occurred at an incidence of <1% (unless otherwise noted) in patients who received KEYTRUDA or were reported with the use of other anti–PD-1/PD-L1 treatments. Severe or fatal cases have been reported for some of these adverse reactions. Cardiac/Vascular: Myocarditis, pericarditis, vasculitis; Nervous System: Meningitis, encephalitis, myelitis and demyelination, myasthenic syndrome/myasthenia gravis (including exacerbation), Guillain-Barré syndrome, nerve paresis, autoimmune neuropathy; Ocular: Uveitis, iritis and other ocular inflammatory toxicities can occur. Some cases can be associated with retinal detachment. Various grades of visual impairment, including blindness, can occur. If uveitis occurs in combination with other immune-mediated adverse reactions, consider a Vogt-Koyanagi-Harada-like syndrome, as this may require treatment with systemic steroids to reduce the risk of permanent vision loss; Gastrointestinal: Pancreatitis, to include increases in serum amylase and lipase levels, gastritis, duodenitis; Musculoskeletal and Connective Tissue: Myositis/polymyositis, rhabdomyolysis (and associated sequelae, including renal failure), arthritis (1.5%), polymyalgia rheumatica; Endocrine: Hypoparathyroidism; Hematologic/Immune: Hemolytic anemia, aplastic anemia, hemophagocytic lymphohistiocytosis, systemic inflammatory response syndrome, histiocytic necrotizing lymphadenitis (Kikuchi lymphadenitis), sarcoidosis, immune thrombocytopenic purpura, solid organ transplant rejection.
For more information, visit www.merck.com and connect with us on Twitter, Facebook, Instagram, YouTube and LinkedIn.
Please see Prescribing Information for KEYTRUDA (pembrolizumab) at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_pi.pdf and Medication Guide for KEYTRUDA at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_mg.pdf.
Source: Merck & Co., Inc.
Merck's blockbuster Keytruda gets EMA panel nod for expanded use in skin cancer
May 20, 2022 9:20 AM ET
By: Ravikash, SA News Editor
A committee of the European Medicines Agency (EMA) recommended the approval for expanded use of Merck's (NYSE:MRK) blockbuster drug Keytruda in certain patients with a type of skin cancer called melanoma.
https://seekingalpha.com/symbol/MRK
OXLUMO™ (lumasiran)
Alnylam Announces Health Canada Authorization of OXLUMO™ (lumasiran), the First and Only Treatment for Primary Hyperoxaluria Type 1 to Lower Urinary Oxalate Levels in Paediatric and Adult Patients
May 18, 2022 8:00 AM ET
Alnylam Pharmaceuticals, Inc. (ALNY)
Authorization Based on two Pivotal Phase III trials, ILLUMINATE-A and ILLUMINATE-B; Showing Significant Reduction in Urinary Oxalate which Drives the Progression of PH1 Disease
TORONTO, May 18, 2022 /CNW/ - Alnylam Pharmaceuticals, Inc. (ALNY) (Nasdaq: ALNY) is pleased to announce that Health Canada has issued a Notice of Compliance (NOC) authorizing OXLUMO™ (lumasiran) injection for subcutaneous use for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in paediatric and adult patients.1
PH1 is an ultra-rare and debilitating genetic disease of the liver characterized by oxalate overproduction.2 Oxalate is an end-product of metabolism and high levels of it are toxic because it cannot be broken down by the human body. Oxalate overproduction results in the deposition of calcium oxalate crystals in the kidneys and urinary tract and can lead to the formation of painful and recurrent kidney stones, nephrocalcinosis (renal deposition of calcium oxalate crystals), progression to ESKD (kidney failure), and systemic organ dysfunction.3
There are several types of primary hyperoxaluria (PH), however, PH1 is the most common and the most severe form accounting for 70 to 80 per cent of all PH cases.4 PH1 affects approximately four individuals per million with some regions, such as the Middle East and North Africa having a higher genetic prevalence.5 Symptom onset ranges from early infancy to sixty years of age with the median age being four to six years.6 The remainder of affected cases present in adulthood with 20 to 50 per cent presenting late stages of chronic kidney disease when diagnosed.7
Until today, there were no authorized pharmaceutical therapies for PH1. The only curative treatment is a liver transplant and if the patient has already progressed to kidney failure, then a dual liver/kidney transplant is required. Unfortunately, liver transplantation is associated with high morbidity and mortality, and life-long immunosuppression, leaving patients with limited options.
"PH1 is a challenging condition to diagnose and treat due to significant variations in the age of onset, rate of disease progression, and associated clinical manifestations," says Dr. Elizabeth Harvey, Pediatric Nephrologist, The Hospital for Sick Children (SickKids), Associate Professor Pediatrics, University of Toronto and member of Alnylam Canada's Scientific Advisory Board. "The approval of this therapy presents patients with a novel treatment option that may potentially reduce the oxalate burden responsible for causing their disease."
OXLUMO™ was granted NOC based on the results of a randomized, double-blind, placebo-controlled clinical study in patients six years and older with PH1 (ILLUMINATE-A) and in a single-arm clinical study in patients less than 6 years of age with PH1 (ILLUMINATE-B).8 The ILLUMINATE-A study showed that OXLUMO™ met its primary endpoint, evidenced by a 53 per cent mean reduction in urinary oxalate, and a 65 per cent mean reduction in urinary oxalate relative to baseline.9 In ILLUMINATE-B, OXLUMO™ demonstrated a 72 per cent mean reduction in spot urinary oxalate:creatinine ratio from baseline to month six (averaged from months three to six), meeting its primary endpoint.10
"This milestone is exciting, as it represents the third RNAi treatment to be brought to Canada in 3 years to treat rare and ultra-rare diseases," says Colleen Coxson, Country General Manager, Canada, Alnylam Pharmaceuticals. "Unfortunately for many patients, it can often take years to diagnose a rare disease, and just as long to find a treatment. Bringing Oxlumo to Canadian patients with PH1 further cements Alnylam Canada's commitment to the rare disease community."
OXLUMO™ is a double-stranded siRNA that reduces the levels of the enzyme glycolate oxidase (GO) responsible for supporting the production of oxalate by targeting the hydroxy acid oxidase 1 (HAO1) messenger ribonucleic acid (mRNA) in major cells of the liver known as hepatocytes through RNA interference. As a result, decreased GO enzyme levels reduce the amount of urinary and plasma oxalate levels, the underlying cause of disease manifestations in patients with PH1.11
"Previously there have been no authorized treatment options for PH1 in Canada, so this is a potentially life-changing milestone for people diagnosed with this debilitating disease – many of whom are infants and children. OXLUMO™ represents an exciting new frontier for the treatment of patients with this serious genetic disease," says Dr. Kristopher Garlick, Country Medical Director, Canada, Alnylam Pharmaceuticals.
About OXLUMO™ (lumasiran)
OXLUMOTM is a double-stranded iRNA therapeutic that reduces the levels of the enzyme glycolate oxidase (GO) by targeting the hydroxyacid oxidase 1 (HAO1) messenger ribonucleic acid (mRNA) in hepatocytes through RNA interference. Decreased GO enzyme levels reduce the amount of available glyoxylate, a substrate for oxalate production. This results in reduction of urinary and plasma oxalate levels, the underlying cause of disease manifestations in patients with PH1. As the GO enzyme is upstream of the alanine:glyoxylate aminotransferase (AGT) enzyme, the deficiency of which causes PH1, the mechanism of action of lumasiran is independent of the underlying AGXT gene mutation encoding AGT.12 In the ILLUMINATE-A study, OXLUMOTM was shown to significantly reduce levels of urinary oxalate relative to placebo, with the majority of patients reaching normal or near-normal levels. Injection site reactions (ISRs) were the most common drug-related adverse reaction.13 In the ILLUMINATE-B study, OXLUMOTM demonstrated an efficacy and safety profile consistent to that observed in ILLUMINATE-A.14 OXLUMOTM is administered via subcutaneous injection once monthly for three months, then once quarterly thereafter at a dose based on actual body weight. For patients who weigh less than 10 kg, ongoing dosing remains monthly. OXLUMOTM should be administered by a healthcare professional.15
SOURCE Alnylam Pharmaceuticals, Inc.
Alnylam's Oxlumo gets Health Canada nod for rare kidney disorder
May 18, 2022 8:30 AM ET
Alnylam Pharmaceuticals, Inc. (ALNY)
By: Ravikash, SA News Editor
- Alnylam Pharmaceuticals (NASDAQ:ALNY) said Health Canada issued a notice of compliance (NOC) authorizing its Oxlumo (lumasiran) injection for subcutaneous use as a therapy for primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in children and adult patients.
- https://seekingalpha.com/news/3840385-alnylams-oxlumo-gets-health-canada-nod-for-rare-kidney-disorder
- https://seekingalpha.com/symbol/ALNY
- https://www.alnylam.com/
- https://www.oxlumo.com/
PT027, a novel fixed-dose combination of albuterol and budesonide
PT027, a novel fixed-dose combination of albuterol and budesonide, used as an as-needed rescue medicine, significantly reduced the risk of a severe exacerbation compared to albuterol by 27% in patients with asthma
PUBLISHED15 May 2022
15 May 2022 17:30 BST
First time an albuterol/budesonide fixed-dose combination rescue medication has been shown to reduce severe exacerbations
MANDALA Phase III trial results published in the New England Journal of Medicine and presented at ATS 2022 International Conference
Full results from the positive MANDALA Phase III trial showed that PT027 (albuterol/budesonide) at two different strengths of budesonide, an inhaled corticosteroid (ICS), used as an as-needed rescue medicine, demonstrated a statistically significant reduction in the risk of a severe exacerbation versus albuterol rescue in patients with moderate to severe asthma.1,2
PT027 is a potential first-in-class inhaled, fixed-dose combination rescue medication containing albuterol, a short-acting beta2-agonist (SABA), and budesonide in the US. It is being developed by AstraZeneca and Avillion.
Globally, more than 176 million asthma attacks are experienced each year.3
Compared with albuterol rescue, PT027 at the 180mcg albuterol/160mcg budesonide dose reduced the risk of a severe exacerbation by 27% (p<0.001) in adults and adolescents.1,2 In the trial, patients were randomised to receive PT027 or albuterol rescue, on top of their usually prescribed maintenance ICS, with or without additional controller medicines.1,2
In secondary endpoints, PT027 (180mcg albuterol/160mcg budesonide) demonstrated a 33% reduction in mean annualised total systemic corticosteroid exposure (p=0.002) and a 24% reduction in annualised severe exacerbation rate (p=0.008).1,2 A numerically higher odds of patients experiencing an improvement in symptom control and quality of life was also observed after 24 weeks of treatment with PT027 compared to albuterol rescue.1,2
Adverse events (AEs) were similar across the treatment groups in the trial and consistent with the known safety profiles of the individual components, with the most common AEs including nasopharyngitis and headache.1
Bradley E. Chipps, Past President of the American College of Allergy, Asthma & Immunology and Medical Director of Capital Allergy & Respiratory Disease Center in Sacramento, US, said:
“The MANDALA Phase III trial results demonstrated that PT027, a novel fixed-dose combination of albuterol/budesonide used as-needed, provided additional anti-inflammatory treatment in response to patient symptoms, which led to a reduced risk of severe exacerbations compared with albuterol alone. These data further strengthen the growing body of evidence around the value of as-needed anti-inflammatory treatment in asthma and support PT027’s potential to transform the current rescue treatment approach.”
Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, AstraZeneca, said: “Asthma is an inflammatory, variable disease and patients are at risk of experiencing a severe exacerbation regardless of disease severity and adherence to treatment. The results from these Phase III trials support the clinical benefit of PT027, an albuterol/budesonide rescue inhaler, which has the potential to be a first-in-class treatment approach that can prevent asthma attacks over and above their current maintenance therapies.”
In the MANDALA trial, PT027 at a lower budesonide dose (180mcg albuterol/80mcg budesonide), also demonstrated a statistically significant reduction of 17% in the risk of severe exacerbation versus albuterol rescue (p=0.041), when used as an as-needed rescue medicine in adults, adolescents, and children aged 4–11 years.1,2
The results were published in the New England Journal of Medicine and will be presented at the American Thoracic Society (ATS) 2022 International Conference.1,2,4
Also being presented at the ATS International Conference this week are the positive DENALI Phase III trial results. In this trial, PT027 demonstrated a statistically significant improvement in lung function measured by forced expiratory volume in one second (FEV1), compared to the individual components albuterol and budesonide, and compared to placebo in patients with mild to moderate asthma aged 12 years or older. Onset of action and duration of effect were similar for PT027 and albuterol. The safety and tolerability of PT027 in DENALI was consistent with the known profiles of the components.5
MANDALA
MANDALA1,19 was a Phase III, randomised, double-blind, multicentre, parallel-group, event-driven trial evaluating the efficacy and safety of PT027 compared to albuterol on the time to first severe asthma exacerbation in 3,132 adults, adolescents, and children (aged 4–11 years) with moderate to severe asthma taking ICS alone or in combination with a range of asthma maintenance therapies, including long-acting beta2-agonists (LABA), leukotriene receptor antagonists (LTRA), long-acting muscarinic antagonists (LAMA) or theophylline. The trial comprised a two-to-four-week screening period, at least a 24-week treatment period and a two-week post-treatment follow-up period.
Patients were randomly assigned to one of the following three treatment groups in a 1:1:1 ratio: PT027 180/160mcg (excluding children aged 4–11 years), PT027 180/80mcg or albuterol 180mcg, taken as an as-needed rescue medicine. PT027 and the albuterol comparator were delivered in a pressurised metered-dose inhaler (pMDI) using AstraZeneca’s Aerosphere delivery technology. The primary efficacy endpoint was the time to first severe asthma exacerbation during the treatment period. Secondary endpoints included severe exacerbation rate (annualised), total systemic corticosteroid exposure (annualised), asthma control and health-related quality of life.
DENALI
DENALI4,20,21 was a Phase III, randomised, double-blind, placebo-controlled, multicentre, parallel-group trial evaluating the efficacy and safety of PT027 compared to its components albuterol and budesonide on improvement in lung function in 1,001 adults, adolescents, and children aged 4–11 years with mild to moderate asthma previously treated either with SABA as-needed alone or in addition to regular low-dose ICS maintenance therapy. The trial comprised a two-to-four-week screening period, a 12-week treatment period and a two-week post-treatment follow-up period.
Patients were randomly assigned to one of the following five treatment groups in a 1:1:1:1:1 ratio: PT027 180/160mcg four times daily (excluding children aged 4–11 years), PT027 180/80mcg four times daily, albuterol 180mcg four times daily, budesonide 160mcg four times daily (excluding children aged 4–11 years) and placebo four times daily. PT027, the albuterol and budesonide comparators and placebo were delivered in a pMDI using AstraZeneca’s Aerosphere delivery technology. The dual primary efficacy endpoints were change from baseline in FEV1 area under the curve 0-6 hours over 12 weeks of PT027 compared to budesonide to assess the effect of albuterol and change from baseline in trough FEV1 at Week 12 of PT027 compared to albuterol to assess the effect of budesonide. Secondary endpoints included the time to onset and duration of response on day one, number of patients who achieved a clinically meaningful improvement in asthma control from baseline at Week 12 and trough FEV1 at Week 1.
PT027
PT027 is a potential first-in-class SABA/ICS rescue treatment for asthma in the US, to be taken as needed. It is an inhaled, fixed-dose combination rescue medication containing albuterol (also known as salbutamol), a SABA, and budesonide, a corticosteroid, and is being developed in a pMDI using AstraZeneca’s Aerosphere delivery technology.
AstraZeneca and Avillion collaboration
In March 2018, AstraZeneca and Avillion signed an agreement to advance PT027 through a global clinical development programme for the treatment of asthma. Under the terms of the agreement, Avillion became the trial sponsor responsible for executing and funding the multicentre, global clinical trial programme for PT027 through NDA filing to a regulatory decision in the US. Following the successful approval of PT027, AstraZeneca has the option, upon certain financial payments, to commercialise the medicine in the US.
Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca.
AstraZeneca, Avillion's asthma inhaler therapy PT027 reduces exacerbations in phase 3 trial
May 16, 2022 6:45 AM ET
By: Ravikash, SA News Editor
AstraZeneca (NASDAQ:AZN) said full results from a late stage study of its inhaled asthma therapy PT027 showed that the rescue medication helped reduce severe exacerbations by 27%.
https://seekingalpha.com/symbol/AZN
OLUMIANT® (baricitinib)
FDA Approves Lilly and Incyte's OLUMIANT® (baricitinib) for the Treatment of Certain Hospitalized Patients with COVID-19
May 11, 2022Download PDF
OLUMIANT is the first and only JAK inhibitor FDA-approved for the treatment of COVID-19 in certain hospitalized adults requiring various degrees of oxygen support
INDIANAPOLIS, May 11, 2022 /PRNewswire/ -- Eli Lilly and Company (NYSE: LLY) and Incyte (NASDAQ: INCY) announced today the U.S. Food and Drug Administration (FDA) has approved OLUMIANT® (baricitinib) for the treatment of COVID-19 in hospitalized adults requiring supplemental oxygen, non-invasive or invasive mechanical ventilation, or extracorporeal membrane oxygenation (ECMO) with a recommended dose of 4-mg once daily for 14 days or until hospital discharge, whichever comes first.
"More than two years into the pandemic, COVID-19 is still hospitalizing many people and burdening our healthcare system. I'm grateful to have OLUMIANT as a treatment option for those who require various degrees of respiratory support, from supplemental oxygen to mechanical ventilation or ECMO," said Andre Kalil, M.D., M.P.H., Professor of Medicine at the University of Nebraska Medical Center and principal investigator of the Adaptive COVID-19 Treatment Trial 2 (ACTT-2) sponsored by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH). "I'm encouraged by the FDA's full approval of OLUMIANT for the treatment of these patients based on results from the rigorous, placebo-controlled, double-blind, randomized trials. While there are therapies currently available, there is still an urgent need for more options to help improve outcomes for patients hospitalized due to COVID-19."
The FDA's approval is supported by results from two randomized, double-blind, placebo-controlled Phase 3 studies (ACTT-2 and COV-BARRIER, including the COV-BARRIER OS 7 addendum study), announced previously. No new safety signals potentially related to the use of OLUMIANT were identified in the studies.
"Nearly one million people with COVID-19 have been treated with OLUMIANT (baricitinib) in approximately 15 countries worldwide," said Patrik Jonsson, Lilly senior vice president, president of Lilly Immunology and Lilly USA, and chief customer officer. "Today's full approval reflects both our confidence in OLUMIANT's role in treating these hospitalized patients and Lilly's tireless efforts to support the medical community and patients in the ongoing fight against COVID-19."
Baricitinib has been available in the U.S. under Emergency Use Authorization (EUA) since November 2020. An EUA will remain in place for hospitalized pediatric patients 2 to less than 18 years old who require various degrees of oxygen support. The emergency authorization is not an approval and is temporary for the duration where circumstances justify the authorization. For additional information about the authorized use, please see the FDA Letter of Authorization, Fact Sheet for Healthcare Providers and Fact Sheet for Patients, Parents and Caregivers.
Lilly has submitted applications for regulatory approval or authorization to multiple regulatory agencies around the world and anticipates further regulatory decisions to follow.
The U.S. FDA-approved labeling for OLUMIANT carries a boxed warning for risk of serious infections, mortality, malignancy, major adverse cardiovascular events (MACE) and thrombosis. Patients treated with OLUMIANT are at an increased risk of serious bacterial, fungal, viral and opportunistic infections leading to hospitalization or death, including tuberculosis. Higher rates of all-cause mortality and MACE have been observed with another JAK inhibitor versus tumor necrosis factor (TNF) blockers. Malignancies and thrombosis have occurred in patients treated with OLUMIANT and higher rates of each have been observed with another JAK inhibitor versus TNF blockers. Consider the risks and benefits of treatment prior to initiating or continuing therapy with OLUMIANT. Please see additional Important Safety Information below.
OLUMIANT is a once-daily, oral JAK inhibitor discovered by Incyte and licensed to Lilly. To learn more about OLUMIANT, please visit www.OLUMIANT.com. OLUMIANT is available in the U.S. as 1-mg and 2-mg tablets through Lilly's authorized specialty distributors.
About ACTT-2 (COVID I) Study
ACTT-2 was a randomized, double-blind, placebo-controlled clinical trial of certain hospitalized adults with confirmed SARS-CoV-2 infection that compared treatment with OLUMIANT and remdesivir (combination group; n=515) to treatment with placebo and remdesivir (placebo group; n=518). Patients treated with the combination received OLUMIANT 4-mg once daily (orally) for 14 days or until hospital discharge, whichever was first, and remdesivir 200-mg on Day 1 and 100-mg once-daily (via intravenous infusion) on subsequent days for a total treatment duration of 10 days or until hospital discharge, whichever was first.
The primary endpoint, for the intent to treat population, was time to recovery within 29 days after randomization. Recovery was defined as being discharged from the hospital without limitations on activities, being discharged from the hospital with limitations on activities and/or requiring home oxygen, or hospitalized but not requiring supplemental oxygen and no longer requiring medical care. The key secondary endpoint was clinical status on Day 15, as assessed on an 8-point ordinal scale.
About COV-BARRIER (COVID II) Study
COV-BARRIER was a randomized, double-blind, placebo-controlled clinical trial of certain hospitalized adults with confirmed SARS-CoV-2 infection that compared treatment with OLUMIANT 4-mg once daily (n=764) with placebo (n=761). OLUMIANT was administered for 14 days or until hospital discharge, whichever came first. Patients could remain on background standard of care, as defined per local guidelines.
Patients requiring invasive mechanical ventilation or ECMO at baseline were enrolled in an exploratory addendum study of COV-BARRIER. These patients were not included in the main COV-BARRIER study population and were analyzed separately.
The primary endpoint was the proportion of patients who died or progressed to non-invasive ventilation/high-flow oxygen or invasive mechanical ventilation within the first 28 days of the study. Patients who required non-invasive ventilation/high-flow oxygen at baseline needed to worsen by at least 1 point on an 8-point ordinal scale to progress. A key secondary endpoint was all-cause mortality by Day 28.
About COV-BARRIER OS 7 Addendum Study
The COV-BARRIER OS 7 addendum study was an exploratory, randomized, double-blind, placebo-controlled substudy of COV-BARRIER in certain hospitalized adults with confirmed SARS-CoV-2 infection requiring invasive mechanical ventilation or ECMO at baseline. This substudy compared treatment with OLUMIANT 4-mg once daily + standard of care (SOC) (n=51) with placebo + SOC (n=50). All patients received SOC in keeping with local clinical practice for COVID-19 management. OLUMIANT was administered for 14 days or until hospital discharge, whichever occurred first. All endpoints in this substudy are considered exploratory, including the prespecified endpoint of all-cause mortality by Day 28.
Indication and Usage for OLUMIANT (baricitinib) tablets (in the United States)
OLUMIANT is indicated for the treatment of coronavirus disease 2019 (COVID-19) in hospitalized adults requiring supplemental oxygen, non-invasive or invasive mechanical ventilation, or extracorporeal membrane oxygenation (ECMO).
Please click to access full Prescribing Information, including Boxed Warning about Serious Infections, Mortality, Malignancy, Major Adverse Cardiovascular Events, and Thrombosis, and Medication Guide.
BA HCP ISI COV 11MAY2022
About OLUMIANT®
OLUMIANT, a once-daily, oral JAK inhibitor, was discovered by Incyte and licensed to Lilly. It is approved in the U.S. and more than 75 countries as a treatment for adults with moderate to severe rheumatoid arthritis. Marketing authorization for the treatment of hospitalized patients with COVID-19 and approval has been granted for OLUMIANT in multiple countries. To date, nearly one million patients worldwide with COVID-19 have been treated with OLUMIANT (baricitinib). The U.S. FDA-approved labeling for OLUMIANT includes a Boxed Warning for Serious Infections, Mortality, Malignancy, Major Adverse Cardiovascular Events, and Thrombosis. See the full Prescribing Information here.
In December 2009, Lilly and Incyte announced an exclusive worldwide license and collaboration agreement for the development and commercialization of OLUMIANT and certain follow-on compounds for patients with inflammatory and autoimmune diseases.
To learn more, visit Lilly.com and Lilly.com/newsroom or follow us on Facebook, Instagram, Twitter and LinkedIn. P-LLY
For additional information on Incyte, please visit Incyte.com and follow @Incyte.
OLUMIANT® is a registered trademark owned or licensed by Eli Lilly and Company, its subsidiaries, or affiliates.
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SOURCE Eli Lilly and Company
Lilly, Incyte get FDA approval of Olumiant to treat certain hospitalized COVID-19 patients
May 11, 2022 7:16 AM ET
Eli Lilly and Company (LLY), INCY
By: Ravikash, SA News Editor
The U.S. Food and Drug Administration (FDA) approved Eli Lilly (NYSE:LLY) and Incyte's (NASDAQ:INCY) JAK inhibitor medicine Olumiant (baricitinib) to treat certain patients with COVID-19.
https://seekingalpha.com/symbol/INCY
https://seekingalpha.com/symbol/LLY
Upadacitinib (RINVOQ®)
May 11, 2022
Upadacitinib (RINVOQ®) Achieved Clinical Remission and Endoscopic Response at One Year in Phase 3 Maintenance Study in Patients with Crohn's Disease
- In patients with moderate to severe Crohn's disease who achieved clinical response to upadacitinib induction treatment, a significantly greater proportion treated with either 15 mg or 30 mg of upadacitinib achieved clinical remission,a,b endoscopic responsec and endoscopic remissiond at week 52 compared to placebo
- The safety results in this study were generally consistent with the known profile of upadacitinib, with no new safety risks observed
- Upadacitinib, a JAK inhibitor discovered and developed by AbbVie, is being studied as an oral therapy for moderate to severe Crohn's disease and several other immune-mediated diseases
NORTH CHICAGO, Ill., May 11, 2022 /PRNewswire/ -- AbbVie today announced positive topline results from U-ENDURE, its Phase 3 maintenance study evaluating upadacitinib in adult patients with moderate to severe Crohn's disease who had an inadequate response or were intolerant to a conventional or biologic therapy. The results showed that more patients treated with either dose of upadacitinib (15 mg or 30 mg once daily) achieved the co-primary endpoints of endoscopic response and clinical remission, as well as the secondary endpoint of endoscopic remission, at one year (week 52) compared to placebo.1 Use of upadacitinib in Crohn's disease has not been evaluated by health authorities. Results from the U-ENDURE maintenance study, in addition to results from the U-EXCEED and U-EXCEL induction studies, will be included in future regulatory submissions.
In the U-ENDURE maintenance study, patients from U-EXCEED and U-EXCEL who responded to 12 weeks of upadacitinib 45 mg oral induction treatment were re-randomized to receive upadacitinib 15 mg, upadacitinib 30 mg, or placebo. Clinical remission was defined by the Crohn's Disease Activity Index (CDAI) or by stool frequency and abdominal pain score (SF/AP).
A significantly higher proportion of patients who received upadacitinib 15 mg or 30 mg achieved clinical remission per the CDAI at week 52: 37 and 48 percent, respectively, versus 15 percent in the placebo group (p<0.0001).1 Results also showed that 36 and 46 percent of patients who received upadacitinib 15 mg and 30 mg, respectively, achieved clinical remission at week 52 per SF/AP compared to 14 percent in the placebo group (p<0.0001).1 At week 52, 28 and 40 percent of patients who received upadacitinib 15 mg and 30 mg, respectively, achieved endoscopic response compared to 7 percent of patients who received placebo (p<0.0001).1 In addition, 19 and 29 percent of patients who received upadacitinib 15 mg and 30 mg achieved endoscopic remission, respectively, compared to 5 percent of patients in the placebo group (p<0.0001).1 A significantly higher proportion of patients who received upadacitinib 15 mg or 30 mg achieved corticosteroid-free clinical remission per CDAI and per SF/AP compared to placebo at week 52 among patients taking corticosteroids at baseline.1
"We are deeply committed to supporting Crohn's disease patients who continue to live with challenging symptoms that impact their daily lives," said Thomas Hudson, M.D., senior vice president, research and development, chief scientific officer, AbbVie. "These results represent important progress as we work to bring new treatment options to patients with inflammatory bowel disease."
"Symptomatic relief as well as healing of the intestinal mucosa in Crohn's disease are important long-term treatment targets which may be associated with slowing disease progression and better quality of life for patients," said Julian Panes, Emeritus Professor of Medicine and the Chief of the IBD Unit at Hospital Clínic de Barcelona and lead study investigator. "These results are encouraging and would be particularly important for patients who have not found relief with other conventional or biologic treatment options."
About U-ENDURE
The U-ENDURE study is a Phase 3, multicenter, randomized, double-blind, placebo-controlled maintenance and long-term study designed to evaluate the efficacy and safety of upadacitinib 15 mg and 30 mg in adults with moderate to severe Crohn's disease. U-ENDURE enrolled patients who responded to 12 weeks of induction treatment from the U-EXCEED and U-EXCEL studies. In addition to the double-blind, placebo-controlled component, the U-ENDURE study also included patients from the induction studies who either responded to placebo or to extended treatment (an additional 12 weeks with 30 mg upadacitinib). During the study, patients who lost response were eligible to receive 30 mg upadacitinib as rescue therapy.
The study included slightly different sets of primary and secondary endpoints for the U.S. Food and Drug Administration (FDA) and the EU European Medicines Agency (EMA). The primary endpoints were achievement of clinical remission (per CDAI for the U.S. FDA, and per SF/AP for the EU EMA, which was measured by average daily very soft or liquid stool frequency and abdominal pain score) and endoscopic response (per SES-CD) at week 52. More information can be found on www.clinicaltrials.gov (NCT03345823).
About the Upadacitinib Phase 3 Crohn's Disease Program4,5,6
The global upadacitinib Phase 3 program evaluates more than 1,000 patients with moderate to severe Crohn's disease across two induction studies and a maintenance study. These studies include assessments of efficacy, safety and tolerability of upadacitinib. More information on these trials can be found at www.clinicaltrials.gov (NCT03345836, NCT03345849, NCT03345823).
About Upadacitinib (RINVOQ®)
Discovered and developed by AbbVie scientists, RINVOQ is a JAK inhibitor that is being studied in several immune-mediated inflammatory diseases.7-14 Based on enzymatic and cellular assays, RINVOQ demonstrated greater inhibitory potency for JAK1 vs JAK2, JAK3, and TYK2.14 The relevance of inhibition of specific JAK enzymes to therapeutic effectiveness is not currently known.
Phase 3 trials of RINVOQ in rheumatoid arthritis, psoriatic arthritis, axial spondyloarthritis, atopic dermatitis, ulcerative colitis, giant cell arteritis, Takayasu arteritis and vitiligo are ongoing.7-14 The use of upadacitinib in Crohn's disease is not approved and its safety and efficacy have not been evaluated by regulatory authorities.
RINVOQ® (upadacitinib) U.S. Use and Important Safety Information14
RINVOQ is a prescription medicine used to treat:
- Adults with moderate to severe rheumatoid arthritis when 1 or more tumor necrosis factor (TNF) blockers have been used, and did not work well or could not be tolerated.
- Adults with active psoriatic arthritis when 1 or more tumor necrosis factor (TNF) blockers have been used, and did not work well or could not be tolerated.
- Adults with moderate to severe ulcerative colitis when 1 or more medicines called TNF blockers have been used, and did not work well or could not be tolerated.
- Adults with active ankylosing spondylitis when 1 or more medicines called TNF blockers have been used, and did not work well or could not be tolerated.
It is not known if RINVOQ is safe and effective in children with juvenile idiopathic arthritis, psoriatic arthritis, ulcerative colitis, or ankylosing spondylitis.
- Adults and children 12 years of age and older with moderate to severe eczema (atopic dermatitis) that did not respond to previous treatment and whose eczema is not well controlled with other pills or injections, including biologic medicines, or when the use of other pills or injections is not recommended.
RINVOQ is safe and effective in children 12 years of age and older weighing at least 88 pounds (40 kg) with atopic dermatitis.
It is not known if RINVOQ is safe and effective in children under 12 years of age with atopic dermatitis.
For more information about AbbVie, please visit us at www.abbvie.com. Follow @abbvie on Twitter, Facebook, LinkedIn or Instagram.
AbbVie's Rinvoq demonstrates remission at one year in Crohn's patients
May 11, 2022 9:58 AM ET
By: Jonathan Block, SA News Editor2 Comments
- Topline data from a phase 3 trial showed that more Crohn's disease patients treated with AbbVie's (NYSE:ABBV) Rinvoq (upadacitinib) achieved clinical and endoscopic remission after one year compared to those on placebo.
- https://seekingalpha.com/news/3836825-abbvie-rinvoq-demonstrates-remission-at-one-year-in-crohns-patients
- https://seekingalpha.com/symbol/ABBV
- https://www.rinvoq.com/
- https://www.abbvie.com/
Onyx Frontier™ drug-eluting stent
MAY 13, 2022
Medtronic receives FDA approval for latest generation drug-eluting coronary stent system
The Onyx Frontier™ drug-eluting stent offers an innovative delivery system and builds upon the acute performance and clinical data from the Resolute Onyx™ drug-eluting stent
DUBLIN, May 13, 2022 /PRNewswire(opens new window)/ -- Medtronic plc (NYSE:MDT), a global leader in healthcare technology, today announced it received U.S. Food and Drug Administration (FDA) approval for the Onyx Frontier™ drug-eluting stent (DES). As the latest evolution in the Resolute DES family, Onyx Frontier DES leverages the same best-in-class stent platform as Resolute Onyx™ DES, with an enhanced delivery system1 designed to improve deliverability and increase acute performance2 in even the most challenging of cases.1
The Onyx Frontier DES is used for the treatment of patients with coronary artery disease (CAD), which is caused by plaque buildup on the inside of the coronary arteries. These plaque deposits can narrow or clog the inside of the arteries, which decreases the supply of blood and oxygen to the heart. CAD is the leading cause of death for both men and women in the United States.3 To help to restore blood flow, a physician may use a stent (a flexible metal scaffolding) that is delivered during a minimally invasive procedure to prop open the artery. A drug-eluting stent is the most common type of stent used to treat a blockage of the heart arteries.4
"The new Onyx Frontier DES, with its enhanced deliverability, will continue to help interventional cardiologists treat complex coronary cases and larger ranges of vessel sizes more efficiently," said Azeem Latib, M.D., section head of interventional cardiology and medical director of structural heart interventions at Montefiore Medical Center in New York City. "Delivering safe and effective outcomes to our patients is our number one priority. It's important that physicians have access to tools like the Onyx Frontier DES that can allow them to efficiently achieve those outcomes."
Meaningful design changes, including increased catheter flexibility, an innovative dual-layer balloon technology and a lower crossing profile led to a 16% improvement in deliverability with Onyx Frontier vs. the previous generation Resolute Onyx DES.2 In addition to the delivery system enhancements, Onyx Frontier offers a broad size matrix to treat more patients and is the only 2.0 mm DES available in the United States (similar to Resolute Onyx). Further, Onyx Frontier continues to provide 4.50-5.00 mm sizes that can be expanded to 6.00 mm - specifically designed to support extra-large vessels. Onyx Frontier shares the same clinical indications as Resolute Onyx, including the most recent approval for patients that are at high risk of bleeding who may benefit from a dual antiplatelet therapy (DAPT) duration as short as one month.2
"The Onyx Frontier DES FDA approval is a very important milestone for Medtronic's Coronary business and demonstrates our commitment to interventional cardiologists by providing best-in-class products," said Jason Weidman, senior vice president and president of the Coronary & Renal Denervation business, which is part of the Cardiovascular Portfolio at Medtronic. "The Onyx Frontier launch also correlates directly to Medtronic's commitment to engineering. The team built upon the design and clinical successes of the Resolute Onyx DES and has continued to evolve proven DES technology to further address the needs of physicians. We look forward to continuing the pursuit of innovation each day."
The Onyx Frontier DES is now approved in the United States and is pending CE (Conformité Européene) Mark.
For more information on Medtronic (NYSE:MDT), visit www.Medtronic.com(opens new window) and follow @Medtronic(opens new window) on Twitter and LinkedIn(opens new window).
SOURCE Medtronic plc
Medtronic gets FDA approval for Onyx Frontier drug-eluting stent for coronary artery disease
May 13, 2022 8:31 AM ET
By: Ravikash, SA News Editor
- Medtronic (NYSE:MDT) said it received U.S. Food and Drug Administration (FDA) approval for its Onyx Frontier drug-eluting stent (DES).
- https://seekingalpha.com/news/3838563-medtronic-gets-fda-approval-for-onyx-frontier-drug-eluting-stent-for-coronary-artery-disease
- https://seekingalpha.com/symbol/MDT
- https://www.medtronic.com/us-en/index.html
Mounjaro™ (tirzepatide) injection
FDA approves Lilly's Mounjaro™ (tirzepatide) injection, the first and only GIP and GLP-1 receptor agonist for the treatment of adults with type 2 diabetes
May 13, 2022Download PDF
Mounjaro delivered superior A1C reductions versus all comparators in phase 3 SURPASS clinical trials
While not indicated for weight loss, Mounjaro led to significantly greater weight reductions versus comparators in a key secondary endpoint
Mounjaro represents the first new class of diabetes medicines introduced in nearly a decade and is expected to be available in the U.S. in the coming weeks
INDIANAPOLIS, May 13, 2022 /PRNewswire/ -- The U.S. Food and Drug Administration (FDA) approved Mounjaro™ (tirzepatide) injection, Eli Lilly and Company's (NYSE: LLY) new once-weekly GIP (glucose-dependent insulinotropic polypeptide) and GLP-1 (glucagon-like peptide-1) receptor agonist indicated as an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes. Mounjaro has not been studied in patients with a history of pancreatitis and is not indicated for use in patients with type 1 diabetes mellitus.
As the first and only FDA-approved GIP and GLP-1 receptor agonist, Mounjaro is a single molecule that activates the body's receptors for GIP and GLP-1, which are natural incretin hormones.1
"Mounjaro delivered superior and consistent A1C reductions against all of the comparators throughout the SURPASS program, which was designed to assess Mounjaro's efficacy and safety in a broad range of adults with type 2 diabetes who could be treated in clinical practice. The approval of Mounjaro is an exciting step forward for people living with type 2 diabetes given the results seen in these clinical trials," said Juan Pablo Frías, M.D., Medical Director, National Research Institute and Investigator in the SURPASS program.
Mounjaro will be available in six doses (2.5 mg, 5 mg, 7.5 mg, 10 mg, 12.5 mg, 15 mg) and will come in Lilly's well-established auto-injector pen with a pre-attached, hidden needle that patients do not need to handle or see.
The approval was based on results from the phase 3 SURPASS program, which included active comparators of injectable semaglutide 1 mg, insulin glargine and insulin degludec. Efficacy was evaluated for Mounjaro 5 mg, 10 mg and 15 mg used alone or in combination with commonly prescribed diabetes medications, including metformin, SGLT2 inhibitors, sulfonylureas and insulin glargine. Participants in the SURPASS program achieved average A1C reductions between 1.8% and 2.1% for Mounjaro 5 mg and between 1.7% and 2.4% for both Mounjaro 10 mg and Mounjaro 15 mg. While not indicated for weight loss, mean change in body weight was a key secondary endpoint in all SURPASS studies. Participants treated with Mounjaro lost between 12 lb. (5 mg) and 25 lb. (15 mg) on average.1
Side effects reported in at least 5% of patients treated with Mounjaro include nausea, diarrhea, decreased appetite, vomiting, constipation, indigestion (dyspepsia), and stomach (abdominal) pain. The labeling for Mounjaro contains a Boxed Warning regarding thyroid C-cell tumors. Mounjaro is contraindicated in patients with a personal or family history of medullary thyroid carcinoma or in patients with Multiple Endocrine Neoplasia syndrome type 2.1
"Lilly has a nearly 100-year heritage of advancing care for people living with diabetes – never settling for current outcomes. We're not satisfied knowing that half of the more than 30 million Americans living with type 2 diabetes are not reaching their target blood glucose levels," said Mike Mason, president, Lilly Diabetes. "We are thrilled to introduce Mounjaro, which represents the first new class of type 2 diabetes medication introduced in almost a decade and embodies our mission to bring innovative new therapies to the diabetes community."
Mounjaro is expected to be available in the United States in the coming weeks. Lilly is committed to helping people access the medicines they are prescribed and will work with insurers, health systems and providers to help enable patient access to Mounjaro. Lilly plans to offer a Mounjaro savings card for people who qualify. Patients or healthcare professionals with questions about Mounjaro can visit www.Mounjaro.com or call The Lilly Answers Center at 1-800-LillyRx (1-800-545-5979).
Tirzepatide is also under regulatory review for the treatment of type 2 diabetes in Europe, Japan and several additional markets. A multimedia gallery is available on Lilly.com.
About the SURPASS clinical trial program
The SURPASS phase 3 global clinical development program for tirzepatide began in late 2018 and included five global registration trials and two regional trials in Japan. These studies ranged from 40 to 52 weeks and evaluated the efficacy and safety of Mounjaro 5 mg, 10 mg and 15 mg as a monotherapy and as an add-on to various standard-of-care medications for type 2 diabetes. The active comparators in the studies were injectable semaglutide 1 mg, insulin glargine and insulin degludec. Collectively, the five global registration trials consistently demonstrated A1C reductions for participants taking Mounjaro across multiple stages of their type 2 diabetes journeys, from an average around five to 13 years of having diabetes.2-8
- SURPASS-1 (NCT03954834) was a 40-week study comparing the efficacy and safety of Mounjaro 5 mg (N=121), 10 mg (N=121) and 15 mg (N=120) as monotherapy to placebo (N=113) in adults with type 2 diabetes inadequately controlled with diet and exercise alone. From a baseline A1C of 7.9%, Mounjaro reduced participants' A1C by a mean of 1.8%* (5 mg) and 1.7%* (10 mg and 15 mg) compared to 0.1% for placebo. In a key secondary endpoint, from a baseline weight of 189 lb., Mounjaro reduced participants' weight by a mean of 14 lb.* (5 mg), 15 lb.* (10 mg) and 17 lb.* (15 mg) compared to 2 lb. for placebo.2,3
- SURPASS-2 (NCT03987919) was a 40-week study comparing the efficacy and safety of Mounjaro 5 mg (N=470), 10 mg (N=469) and 15 mg (N=469) to injectable semaglutide 1 mg (N=468) in adults with type 2 diabetes inadequately controlled with ≥1500 mg/day metformin alone. From a baseline A1C of 8.3%, Mounjaro reduced participants' A1C by a mean of 2.0%ꝉ (5 mg), 2.2%* (10 mg) and 2.3%* (15 mg) compared to 1.9% for semaglutide. In a key secondary endpoint, from a baseline weight of 207 lb., Mounjaro reduced participants' weight by a mean of 17 lb.ꝉ (5 mg), 21 lb.* (10 mg) and 25 lb.* (15 mg) compared to 13 lb. for semaglutide.4,5
- SURPASS-3 (NCT03882970) was a 52-week study comparing the efficacy of Mounjaro 5 mg (N=358), 10 mg (N=360) and 15 mg (N=358) to titrated insulin degludec (N=359) in adults with type 2 diabetes treated with metformin with or without an SGLT-2 inhibitor. From a baseline A1C of 8.2%, Mounjaro reduced participants' A1C by a mean of 1.9%* (5 mg), 2.0%* (10 mg) and 2.1%* (15 mg) compared to 1.3% for insulin degludec. From a baseline weight of 208 lb., Mounjaro reduced participants' weight by a mean of 15 lb.* (5 mg), 21 lb.* (10 mg) and 25 lb.* (15 mg) compared to an increase of 4 lb. for insulin degludec.6
- SURPASS-4 (NCT03730662) was a 104-week study comparing the efficacy and safety of Mounjaro 5 mg (N=328), 10 mg (N=326) and 15 mg (N=337) to insulin glargine (N=998) in adults with type 2 diabetes inadequately controlled with at least one and up to three oral antihyperglycemic medications (metformin, sulfonylureas or SGLT-2 inhibitors), who have increased cardiovascular (CV) risk. The primary endpoint was measured at 52 weeks. From a baseline A1C of 8.5%, Mounjaro reduced participants' A1C by a mean of 2.1%* (5 mg), 2.3%* (10 mg) and 2.4%* (15 mg) compared to 1.4% for insulin glargine. From a baseline weight of 199 lb., Mounjaro reduced weight by a mean of 14 lb.* (5 mg), 20 lb.* (10 mg) and 23 lb.* (15 mg) compared to an increase of 4 lb. for insulin glargine.7
- SURPASS-5 (NCT04039503) was a 40-week study comparing the efficacy and safety of Mounjaro 5 mg (N=116), 10 mg (N=118) and 15 mg (N=118) to placebo (N=119) in adults with inadequately controlled type 2 diabetes already being treated with insulin glargine, with or without metformin. From a baseline A1C of 8.3%, Mounjaro reduced A1C by a mean of 2.1%* (5 mg), 2.4%* (10 mg) and 2.3%* (15 mg) compared to 0.9% for placebo. From a baseline weight of 210 lb., Mounjaro reduced participants' weight by a mean of 12 lb.* (5 mg), 17 lb.* (10 mg) and 19 lb.* (15 mg) compared to an increase of 4 lb. for placebo.8
*p<0.001 for superiority vs. placebo or active comparator, adjusted for multiplicity
ꝉp<0.05 for superiority vs. semaglutide 1 mg, adjusted for multiplicity
About Mounjaro™ (tirzepatide) injection1
Mounjaro™ (tirzepatide) injection is FDA-approved as an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes mellitus. As the first and only FDA-approved GIP and GLP-1 receptor agonist, Mounjaro is a single molecule that activates the body's receptors for GIP (glucose-dependent insulinotropic polypeptide) and GLP-1 (glucagon-like peptide-1). Mounjaro will be available in six doses (2.5 mg, 5 mg, 7.5 mg, 10 mg, 12.5 mg, 15 mg) and will come in Lilly's well-established auto-injector pen with a pre-attached, hidden needle that patients do not need to handle or see.
Learn more
For more information, call 1-800-LillyRx (1-800-545-5979) or go to www.mounjaro.com.
This information does not take the place of talking with your healthcare provider. Be sure to talk to your healthcare provider about Mounjaro and how to take it. Your healthcare provider is the best person to help you decide if Mounjaro is right for you.
MounjaroTM and its delivery device base are trademarks owned or licensed by Eli Lilly and Company, its subsidiaries, or affiliates.
Please click to access full Prescribing Information and Medication Guide.
To learn more, visit Lilly.com and Lilly.com/newsroom or follow us on Facebook, Instagram, Twitter and LinkedIn. P-LLY
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SOURCE Eli Lilly and Company
Eli Lilly's dual-receptor diabetes drug tirzepatide granted FDA approval
May 13, 2022 3:44 PM ET
Eli Lilly and Company (LLY)NVO
By: Jonathan Block, SA News Editor2 Comments
- The U.S. FDA has approved Eli Lilly's (NYSE:LLY) Mounjaro (tirzepatide), the first-ever diabetes medication that activates glucagon-like peptide-1 (GLP-1) and glucose-dependent insulinotropic polypeptide (GIP) receptors.
- https://seekingalpha.com/news/3838811-eli-lilly-dual-receptor-diabetes-drug-tirzepatide-mounjaro-granted-fda-approval
- https://www.fda.gov/news-events/press-announcements/fda-approves-novel-dual-targeted-treatment-type-2-diabetes
- https://seekingalpha.com/symbol/LLY
- https://www.lilly.com/
Nirsevimab
PRESS RELEASESMay 11 2022
Press Release: New nirsevimab data analyses reinforce efficacy against RSV
New nirsevimab data analyses reinforce efficacy against RSV
- A prespecified pooled analysis of Phase 3 and Phase 2b data demonstrated an efficacy of 79.5% against medically attended lower respiratory tract infections (LRTI), including hospitalizations, caused by respiratory syncytial virus (RSV)1
- Nirsevimab is the first investigational immunization designed to protect all infants across the RSV season with a single dose
- Two analyses are being presented at the European Society for Paediatric Infectious Diseases meeting1,2
Paris, May 11, 2022. Results from a prespecified pooled analysis of the pivotal Phase 3 MELODY and Phase 2b nirsevimab trials demonstrated an efficacy (relative risk reduction versus placebo) of 79.5% (95% CI 65.9 to 87.7; P<0.0001) against medically attended LRTI, such as bronchiolitis or pneumonia, caused by RSV in infants born at term or preterm entering their first RSV season.1
In a separate pooled post-hoc analysis of the trials, blood samples taken from infants dosed with nirsevimab exhibited RSV neutralizing antibodies that were approximately 50-fold higher than baseline at Day 151 post-dose. RSV neutralizing antibody levels remained greater than 19-fold higher than placebo recipients with no known RSV infection through Day 361, suggesting protection may extend beyond Day 151.2
The safety profile across the nirsevimab and placebo groups, as reported in previous trials, remains similar.3-6 These findings contribute to the growing body of evidence suggesting that nirsevimab can protect all infants through their first RSV season with a single dose.1-7
Eric Simões, MD
Clinical Professor, Pediatrics-Infectious Diseases, UC Denver School of Medicine
“RSV remains the most common cause of LRTI in infants and results in seasonal epidemics globally each year. These new analyses strengthen nirsevimab’s potential to protect all infants across the RSV season with a single dose, which may lead to a paradigm shift in RSV prevention.”
Jean-François Toussaint
Global Head of Research and Development Vaccines, Sanofi
“These new analyses are very consistent with and confirm the strong results observed in all Phase 2 and Phase 3 studies that evaluated nirsevimab in diverse pediatric populations. We take pride in the progress made to develop a potential solution to address this long unmet need for all infants.”
Mene Pangalos
Executive Vice President, BioPharmaceuticals R&D, AstraZeneca
“Each year, RSV causes seasonal epidemics of LRTIs in infants. These analyses add to nirsevimab’s compelling body of evidence as the first potential single-dose preventative immunization for all infants against RSV, addressing a clear unmet need in the RSV preventative landscape.”
The data are being presented at the 40th Annual Meeting of the European Society for Paediatric Infectious Diseases (ESPID) from May 9-13 in Athens, Greece.
Nirsevimab is being developed by Sanofi and AstraZeneca.
About nirsevimab
Nirsevimab is an investigational long-acting antibody designed to protect all infants from birth entering their first RSV season with a single dose. Due to its extended half-life technology, nirsevimab is being developed as a single dose for protection of all infants through their first RSV season.5,6,8
Nirsevimab is an immunization designed to provide direct RSV protection to all infants via an antibody to help prevent LRTI caused by RSV. Monoclonal antibodies do not require the activation of the immune system to help offer rapid and direct protection against disease.9
In March 2017, Sanofi and AstraZeneca announced an agreement to develop and commercialize nirsevimab. Under the terms of the agreement, AstraZeneca leads all development and manufacturing activities and Sanofi will lead commercialization activities and record revenues. Under the terms of the global agreement, Sanofi made an upfront payment of €120m, has paid a development milestone of €30m and will pay up to a further €465m upon achievement of certain development and sales-related milestones. The two companies share all costs and profits. Revenue from the agreement is reported as Collaboration Revenue in the Company’s financial statements.
Nirsevimab has been granted regulatory designations to facilitate expedited development by several regulatory agencies around the world. These include Breakthrough Therapy Designation by The China Center for Drug Evaluation under the National Medical Products Administration; Breakthrough Therapy Designation from the US Food and Drug Administration; access granted to the European Medicines Agency (EMA) PRIority MEdicines scheme; Promising Innovative Medicine designation by the UK Medicines and Healthcare products Regulatory Agency; and named “a medicine for prioritized development” under the Project for Drug Selection to Promote New Drug Development in Pediatrics by the Japan Agency for Medical Research and Development (AMED). The safety and efficacy of nirsevimab is currently being evaluated under an accelerated assessment procedure by the EMA. Nirsevimab has not been approved by any regulatory authority.
About the pivotal nirsevimab clinical trials
The Phase 2b trial was a randomized, placebo-controlled trial designed to measure the efficacy of nirsevimab against medically attended LRTI through 150 days post-dose. Healthy preterm infants of 29–35 weeks’ gestation were randomized (2:1) to receive a single 50mg intramuscular injection of nirsevimab or placebo. Between November 2016 and December 2017, 1,453 infants were randomized (nirsevimab, n=969; placebo, n=484) at the RSV season start. Research was conducted in both hemispheres, at 164 sites in 23 countries.6 Data was published in the New England Journal of Medicine (NEJM) in July 2020. The dosing regimen was optimized based on further exploration of this data. The subsquent Phase 3 study MELODY applied the optimized dosing regimen.1,5
The Phase 3 MELODY trial was a randomized, placebo-controlled trial conducted across 21 countries designed to determine efficacy of nirsevimab against medically attended LRTI due to RSV confirmed by reverse transcriptase polymerase chain reaction testing through 150 days after dosing, versus placebo, in healthy late preterm and term infants (35 weeks gestational age or greater) entering their first RSV season.5 Infants were randomized (2:1) to receive a single 50mg (in infants weighing <5kg) or 100mg (in infants weighing ≥5kg) intramuscular injection of nirsevimab or placebo. Between July 2019 and March 2020, 1,490 infants were randomized to either nirsevimab or placebo at the RSV season start.3 Data was published on the primary analysis in NEJM in March 2022.
The prespecified pooled analyses of the Phase 3 and the Phase 2b trials looked at infants receiving the optimized dosing regimen (infants <5 kg at dosing and receiving the 50 mg dose from Phase 2b and the infants from Phase 3), and demonstrated an efficacy of 79.5% (95% CI 65.9 to 87.7, P<0.0001) against medically attended LRTI and 77.3% (95% CI 50.3, 89.7, P<0.001) against RSV LRTI hospitalizations. The analysis was based on 2,350 infants of which 1,564 infants were randomized to receive nirsevimab and 786 infants were randomized to receive placebo.1
The results of MELODY, Phase 2/3 MEDLEY and the Phase 2b trials demonstrate that nirsevimab provides protection against RSV in all infants entering their first RSV season with a single dose.1-5 This all-infant population includes preterm, healthy late preterm and term infants, as well as infants with specific conditions.
These trials form the basis of regulatory submissions that began in 2022.
Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY
AstraZeneca/Sanofi's single-dose nirsevimab reduces RSV infection in infants by ~80% shows data
May 11, 2022 5:35 AM ET
By: Ravikash, SA News Editor1 Comment
AstraZeneca (NASDAQ:AZN) Sanofi's (NASDAQ:SNY) said a pooled analysis from two studies showed 79.5% efficacy of their single dose antibody nirsevimab against lower respiratory tract infections (LRTI), caused by respiratory syncytial virus (RSV), in infants.
https://seekingalpha.com/symbol/SNY
https://seekingalpha.com/symbol/AZN
lecanemab (BAN2401)
EISAI COMPLETES ROLLING SUBMISSION TO THE U.S. FDA FOR BIOLOGICS LICENSE APPLICATION OF LECANEMAB FOR EARLY ALZHEIMER’S DISEASE UNDER THE ACCELERATED APPROVAL PATHWAY
MAY 9, 2022 • INVESTOR RELATIONS
TOKYO and CAMBRIDGE, Eisai Co., Ltd. (Headquarters: Tokyo, CEO: Haruo Naito, "Eisai") and Biogen Inc. (Nasdaq: BIIB, Corporate headquarters: Cambridge, Massachusetts, CEO: Michel Vounatsos, "Biogen") announced today that Eisai has completed the rolling submission to the U.S. Food and Drug Administration (FDA) of a Biologics License Application (BLA) under the accelerated approval pathway for the investigational anti-amyloid beta (Aβ) protofibril antibody lecanemab (BAN2401) for the treatment of mild cognitive impairment (MCI) due to Alzheimer’s disease (AD) and mild AD (collectively known as early AD) with confirmed presence of amyloid pathology in the brain. As part of the completed rolling submission, Eisai has requested Priority Review. If the FDA accepts the BLA, the Prescription Drug User Fee Act (PDUFA) action date (target date for completion of examination) will be set. While Eisai is currently submitting lecanemab under the accelerated approval pathway, the lecanemab Phase 3 confirmatory Clarity AD clinical trial conducted with 1,795 patients will report out in the Fall of 2022. The FDA has agreed that the results of Clarity AD, when completed, can serve as the confirmatory study to verify the clinical benefit of lecanemab. Dependent upon the results of the Clarity AD clinical trial, Eisai may submit for full approval of lecanemab to the FDA during fiscal year 2022.
The BLA submission for lecanemab is based on clinical, biomarker and safety data from the proof-of-concept Phase 2b (Study 201 Core) in 856 people with early AD with confirmed presence of amyloid pathology, biomarker and safety data from the Study 201 OLE (open-label extension study, 180 subjects), and blinded safety data from the confirmatory Clarity AD Phase 3 study (1,795 subjects). The large number of participants across these studies provides the FDA with extensive safety data. Study 201 explored the impact of treatment with lecanemab on reducing amyloid plaque and clinical decline. At 18 months of treatment, 10 mg/kg biweekly lecanemab reduced brain amyloid by a mean of 0.306 SUVr units (from a baseline mean of 1.37), and over 80% of subjects became amyloid negative by visual read. Furthermore, the extent of reduction in amyloid was correlated with slower clinical decline on ADCOMS (Alzheimer’s Disease Composite Score), CDR-SB (Clinical Dementia Rating-Sum-of-Boxes), and ADAS-cog (Alzheimer Disease Assessment Scale-Cognitive Subscale) at the treatment group and patient level. In the Core study, the overall rate of amyloid-related imaging abnormalities-edema/effusion (ARIA-E), an adverse event associated with anti-amyloid beta antibodies therapies was 9.9% (16/161) of patients treated with lecanemab 10 mg/kg biweekly compared with 0.8% (2/245) of placebo patients. The results from Study 201 were published in a peer-reviewed journal Alzheimer’s Research and Therapy in April 2021.
“We would like to thank the people living with early AD and the healthcare professionals who participated in the lecanemab 201 study for their cooperation allowing completion of this BLA to the U.S. FDA. Alzheimer’s disease is a progressive and devastating disease with few treatment options,” said Haruo Naito, Chief Executive Officer at Eisai Co., Ltd. “Eisai employees have spent time with people living with Alzheimer’s disease and their families to truly understand their feelings and challenges and have been working to create new treatments for many years. Our comprehensive medicine creation approach along the Alzheimer’s disease continuum reflects Eisai’s long-term commitment to providing innovative treatments to the people living with AD, their families and healthcare professionals who urgently need new treatment options.”
“With Alzheimer’s disease, patients and their loved ones don’t have the luxury of time. There is an enormous unmet need in this space, and we continue to make progress in advancing additional treatment options for people living with this devastating disease,” said Michel Vounatsos, Chief Executive Officer at Biogen. “Anti-amyloid antibodies are a new wave of important medicines, which could provide patients and their physicians more options in addressing this complex disease.”
Lecanemab was granted Breakthrough Therapy and Fast Track designations by the FDA in June and December 2021, respectively. In March 2022, Eisai initiated submission of application data to the Pharmaceuticals and Medical Devices Agency (PMDA) under the prior assessment consultation system in Japan with the aim of obtaining early approval for lecanemab, and aims to file for the manufacturing and marketing approval based on the results of Clarity AD during Eisai’s fiscal year 2022.
Eisai serves as the lead of lecanemab development and regulatory submissions globally with both Eisai and Biogen co-commercializing and co-promoting the product and Eisai having final decision-making authority.
1. About Lecanemab (BAN2401)
Lecanemab is an investigational humanized monoclonal antibody for Alzheimer’s disease (AD) that is the result of a strategic research alliance between Eisai and BioArctic. Lecanemab selectively binds to neutralize and eliminate soluble, toxic amyloid-beta (Aβ) aggregates (protofibrils) that are thought to contribute to the neurodegenerative process in AD. As such, lecanemab may have the potential to have an effect on disease pathology and to slow down the progression of the disease. Currently, lecanemab is being developed as the only anti- Aβ antibody that can be used for the treatment of early AD without the need for titration. With regard to the results from pre-specified analysis at 18 months of treatment, Study 201 demonstrated reduction of brain Aβ accumulation (P<0.0001) and slowing of disease progression measured by ADCOMS* (P<0.05) in early AD patients. The study did not achieve its primary outcome measure** at 12 months of treatment. The Study 201 open-label extension was initiated after completion of the Core period and a Gap period off treatment of 9-59 months (average of 24 months, n=180 from core study enrolled) to evaluate safety and efficacy, and is underway.
Currently, lecanemab is being studied in a confirmatory Phase 3 clinical study in symptomatic early AD (Clarity-AD), following the outcome of the Phase 2 clinical study (Study 201). Since July 2020 the Phase 3 clinical study (AHEAD 3-45) for individuals with preclinical AD, meaning they are clinically normal and have intermediate or elevated levels of amyloid in their brains, is ongoing. AHEAD 3-45 is conducted as a public-private partnership between the Alzheimer’s Clinical Trial Consortium that provides the infrastructure for academic clinical trials in AD and related dementias in the U.S, funded by the National Institute on Aging, part of the National Institutes of Health, Eisai and Biogen. Since January 2022, the Tau NexGen clinical study for Dominantly Inherited Alzheimer’s disease (DIAD), that is conducted by Dominantly Inherited Alzheimer Network Trials Unit (DIAN-TU), led by Washington University School of Medicine in St. Louis, is ongoing. Furthermore, Eisai has initiated a lecanemab subcutaneous dosing Phase 1 study. Eisai obtained the global rights to study, develop, manufacture and market lecanemab for the treatment of AD pursuant to an agreement concluded with BioArctic in December 2007.
* Developed by Eisai, ADCOMS (AD Composite Score) combines items from the ADAS-Cog (Alzheimer’s Disease Assessment Scale-cognitive subscale), CDR (Clinical Dementia Rating) and the MMSE (Mini-Mental State Examination) scales to enable a sensitive detection of changes in clinical functions of early AD symptoms and changes in memory. The ADCOMS scale ranges from a score of 0.00 to 1.97, with higher score indicating greater impairment.
** An 80% or higher estimated probability of demonstrating 25% or greater slowing in clinical decline at 12 months treatment measured by ADCOMS from baseline compared to placebo.
2. About the Collaboration between Eisai and Biogen for Alzheimer’s Disease
Eisai and Biogen are collaborating on the joint development and commercialization of AD treatments. Eisai serves as the lead in the co-development of lecanemab.
3. About the Collaboration between Eisai and BioArctic for Alzheimer’s Disease
Since 2005, BioArctic has had a long-term collaboration with Eisai regarding the development and commercialization of drugs for the treatment of AD. The commercialization agreement on the lecanemab antibody was signed in December 2007, and the development and commercialization agreement on the antibody lecanemab back-up for AD, which was signed in May 2015. Eisai is responsible for the clinical development, application for market approval and commercialization of the products for AD. BioArctic has no development costs for lecanemab in AD.
4. About Eisai Co., Ltd.
Eisai Co., Ltd. is a leading global pharmaceutical company headquartered in Japan. Eisai’s corporate philosophy is based on the human health care (hhc) concept, which is to give first thought to patients and their families, and to increase the benefits that health care provides to them. With a global network of R&D facilities, manufacturing sites and marketing subsidiaries, we strive to realize our hhc philosophy by delivering innovative products to target diseases with high unmet medical needs, with a particular focus in our strategic areas of Neurology and Oncology.
Leveraging the experience gained from the development and marketing of a treatment for Alzheimer’s disease, Eisai aims to establish the “Eisai Dementia Platform.” Through this platform, Eisai plans to deliver novel benefits to those living with dementia and their families through constructing a “Dementia Ecosystem,” by collaborating with partners such as medical organizations, diagnostic development companies, research organizations, and bio-ventures in addition to private insurance agencies, finance industries, fitness clubs, automobile makers, retailers, and care facilities. For more information about Eisai Co., Ltd., please visit https://www.eisai.com.
5. About Biogen
As pioneers in neuroscience, Biogen discovers, develops, and delivers worldwide innovative therapies for people living with serious neurological diseases as well as related therapeutic adjacencies. One of the world’s first global biotechnology companies, Biogen was founded in 1978 by Charles Weissmann, Heinz Schaller, Sir Kenneth Murray, and Nobel Prize winners Walter Gilbert and Phillip Sharp. Today, Biogen has a leading portfolio of medicines to treat multiple sclerosis, has introduced the first approved treatment for spinal muscular atrophy, and is providing the first and only approved treatment to address a defining pathology of Alzheimer’s disease. Biogen is also commercializing biosimilars and focusing on advancing the industry’s most diversified pipeline in neuroscience that will transform the standard of care for patients in several areas of high unmet need.
In 2020, Biogen launched a bold 20-year, $250 million initiative to address the deeply interrelated issues of climate, health, and equity. Healthy Climate, Healthy Lives™ aims to eliminate fossil fuels across the company’s operations, build collaborations with renowned institutions to advance the science to improve human health outcomes, and support underserved communities.
The company routinely posts information that may be important to investors on its website at www.biogen.com. To learn more, please visit www.biogen.com and follow Biogen on social media – Twitter, LinkedIn, Facebook, YouTube.
Biogen, Eisai finish filing with FDA for Alzheimer’s therapy lecanemab, seek priority review
May 10, 2022 6:11 AM ET Eisai Co., Ltd. (ESALY), ESALF, BIIB
By: Ravikash, SA News Editor
Biogen (NASDAQ:BIIB) and Eisai (OTCPK:ESALY) (OTCPK:ESALF) completed a rolling submission to the U.S. Food and Drug Administration (FDA) of their medicine lecanemab to treat Alzheimer’s disease (AD).
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ENHERTU® (fam-trastuzumab deruxtecan-nxki)
Enhertu approved in the US for patients with HER2-positive metastatic breast cancer treated with a prior anti-HER2-based regimen
PUBLISHED5 May 2022
5 May 2022 07:10 BST
Approval broadens indication for AstraZeneca and Daiichi Sankyo’s Enhertu to earlier use in metastatic breast cancer
Based on ground-breaking DESTINY-Breast03 results showing Enhertu reduced the risk of disease progression or death by 72% versus trastuzumab emtansine (T-DM1)
AstraZeneca and Daiichi Sankyo’s Enhertu (trastuzumab deruxtecan) has been approved in the US for the treatment of adult patients with unresectable or metastatic HER2-positive breast cancer who have received a prior anti-HER2-based regimen either in the metastatic setting, or in the neoadjuvant or adjuvant setting and have developed disease recurrence during or within six months of completing therapy.
Enhertu is a specifically engineered HER2-directed antibody drug conjugate (ADC) being jointly developed and commercialised by AstraZeneca and Daiichi Sankyo.
The approval by the Food and Drug Administration (FDA) was based on positive results from the DESTINY-Breast03 Phase III trial that showed Enhertu reduced the risk of disease progression or death by 72% versus trastuzumab emtansine (T-DM1) (hazard ratio [HR] 0.28; 95% confidence interval [CI]: 0.22-0.37; p<0.0001) in patients with HER2-positive unresectable and/or metastatic breast cancer previously treated with trastuzumab and a taxane.
The approval was granted under the FDA’s Real-Time Oncology Review (RTOR) programme and converts the accelerated approval of Enhertu in later line HER2-positive metastatic breast cancer to standard approval, broadening Enhertu’s breast cancer indication in the US to earlier lines of use in patients with HER2-positive metastatic breast cancer.
Erika Hamilton, MD, Director of the Breast Cancer and Gynecological Cancer Research Program for Sarah Cannon Research Institute, Nashville, Tennessee, US, said: “Enhertu has demonstrated significant progression-free survival in the earlier metastatic setting, potentially establishing it as a new standard of care in previously treated patients with HER2-positive metastatic breast cancer. Today’s approval is an important milestone for the clinical community as we will now be able to offer Enhertu to these patients earlier in their treatment.”
Catherine Ormerod, Executive Vice President, Strategy and Mission, Living Beyond Breast Cancer, said: “This is an important day for the breast cancer community. With this approval, Enhertu now provides a new treatment option for patients with HER2-positive metastatic breast cancer which can be used earlier in treatment to potentially delay progression of disease.”
Dave Fredrickson, Executive Vice President, Oncology Business Unit, AstraZeneca, said: “Enhertu is already established in the later-line treatment of patients with HER2-positive metastatic breast cancer, and we are thrilled that with this approval, patients in the US will now be able to access the transformative potential of Enhertu earlier in their treatment. We look forward to bringing this important, potentially paradigm-shifting medicine to even more patients across the globe in an earlier setting as quickly as possible.”
Ken Keller, Global Head, Oncology Business and President and CEO, Daiichi Sankyo, Inc, said: “Today’s FDA approval, which converts the accelerated approval of Enhertu to regular approval, highlights the importance of the FDA’s accelerated pathway that allows for earlier approval of medicines to treat serious medical conditions such as breast cancer. Data from DESTINY-Breast03 not only confirmed the results of DESTINY-Breast01, but also demonstrated the superiority of Enhertu in prolonging progression-free survival compared to T-DM1 in an earlier setting of HER2-positive metastatic breast cancer.”
The DESTINY-Breast03 Phase III trial results were recently published online in The New England Journal of Medicine.1 In the trial, the safety profile of Enhertu was consistent with previous clinical trials, with no new safety concerns identified and no Grade 4 or 5 treatment-related interstitial lung disease events.
Based on the DESTINY-Breast03 data, fam-trastuzumab deruxtecan-nxki (Enhertu) recently was added to the NCCN Clinical Practical Guidelines in Oncology (NCCN Guidelines®) as the Category 1 preferred regimen as second-line therapy for recurrent unresectable (local or regional) or Stage IV HER2-positive disease.2
The US regulatory submission was reviewed under Project Orbis, which provides a framework for concurrent submission and review of oncology medicines among participating international partners. Five national health authorities collaborated with the FDA on this review, including the Australian Therapeutic Goods Administration, the Brazilian Health Regulatory Agency (ANVISA), Health Canada, Israel’s Ministry of Health Pharmaceutical Administration and Switzerland’s Swissmedic.
This approval follows the recent Priority Review and Breakthrough Therapy Designation of Enhertu in the US in this earlier setting.
Regulatory applications for Enhertu are currently under review in Europe, Japan and several other countries for the treatment of adult patients with unresectable or metastatic HER2-positive breast cancer who have received a prior anti-HER2-based regimen based on the results from the DESTINY-Breast03 trial.
DESTINY-Breast03
DESTINY-Breast03 is a global, head-to-head, randomised, open-label, registrational Phase III trial evaluating the efficacy and safety of Enhertu (5.4mg/kg) versus T-DM1 in patients with HER2-positive unresectable and/or metastatic breast cancer previously treated with trastuzumab and a taxane.
The primary efficacy endpoint of DESTINY-Breast03 is progression-free survival (PFS) based on blinded independent central review. Secondary efficacy endpoints include overall survival, objective response rate (ORR), duration of response, PFS based on investigator assessment and safety.
DESTINY-Breast03 enrolled approximately 500 patients at multiple sites in Asia, Europe, North America, Oceania and South America. For more information about the trial, visit ClinicalTrials.gov.
Enhertu
Enhertu is a HER2-directed ADC. Designed using Daiichi Sankyo’s proprietary DXd ADC technology, Enhertu is the lead ADC in the oncology portfolio of Daiichi Sankyo and the most advanced programme in AstraZeneca’s ADC scientific platform. Enhertu consists of a HER2 monoclonal antibody attached to a topoisomerase I inhibitor payload, an exatecan derivative, via a stable tetrapeptide-based cleavable linker.
Enhertu (5.4mg/kg) is approved in the US for the treatment of adult patients with unresectable or metastatic HER2-positive breast cancer who have received a prior anti-HER2-based regimen either in the metastatic setting, or in the neoadjuvant or adjuvant setting and have developed disease recurrence during or within six months of completing therapy, based on results from the DESTINY-Breast03 trial.
Enhertu (5.4mg/kg) is also approved in approximately 40 countries for the treatment of adult patients with unresectable or metastatic HER2-positive breast cancer who have received two or more prior anti-HER2-based regimens based on the results from the DESTINY-Breast01 trial.
Enhertu (6.4mg/kg) is approved in several countries for the treatment of adult patients with locally advanced or metastatic HER2-positive gastric or gastroesophageal junction adenocarcinoma who have received a prior trastuzumab-based regimen based on the results from the DESTINY-Gastric01 trial.
Daiichi Sankyo collaboration
Daiichi Sankyo Company, Limited (TSE:4568) [referred to as Daiichi Sankyo] and AstraZeneca entered into a global collaboration to jointly develop and commercialise Enhertu (a HER2-directed ADC) in March 2019, and datopotamab deruxtecan (DS-1062; a TROP2-directed ADC) in July 2020, except in Japan where Daiichi Sankyo maintains exclusive rights. Daiichi Sankyo is responsible for manufacturing and supply of Enhertu and datopotamab deruxtecan.
Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca.
AstraZeneca, Daiichi Sankyo's Enhertu wins FDA approval to treat breast cancer subtype
May 05, 2022 4:48 AM ET AstraZeneca PLC (AZN), DSNKYDSKYF
By: Ravikash, SA News Editor1 Comment
The U.S. Food and Drug Administration (FDA) approved AstraZeneca (NASDAQ:AZN) and Daiichi Sankyo's (OTCPK:DSKYF) (OTCPK:DSNKY) Enhertu to treat certain patients with breast cancer.
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Jakavi® (ruxolitinib) Jakafi
Novartis receives European Commission approval for Jakavi® to be the first post-steroid treatment for acute and chronic graft-versus-host disease
May 05, 2022
- Jakavi is the first JAK1/2 inhibitor available for patients in Europe who previously had no approved therapies for the treatment of steroid-refractory graft-versus-host disease (GvHD)1,2
- In clinical trials, Jakavi demonstrated superiority versus best available therapy in patients with steroid-refractory/dependent acute and chronic GvHD, with overall response rates of 62% vs. 39%, and 50% vs. 26% respectively2,3
- GvHD is a common and potentially life-threatening complication that arises in approximately half of patients following allogeneic stem cell transplants4
Basel, May 5, 2022 — Novartis today announced the European Commission (EC) has approved Jakavi (ruxolitinib) for the treatment of patients aged 12 years and older with acute or chronic GvHD who have inadequate response to corticosteroids or other systemic therapies.
“Today, 30-60% of patients with GvHD do not respond to first-line steroid treatment, underscoring the need for new approaches to ensure long-term treatment goals are met,” said Dr. Robert Zeiser, University Hospital Freiburg, Department of Haematology, Oncology and Stem Cell Transplantation, Freiburg, Germany. “The approval of Jakavi offers healthcare providers and patients with GvHD who remain dependent on or refractory to steroids a new way to manage this debilitating and life-threatening condition.”
The approval of Jakavi follows the positive opinion granted in March by the Committee for Medicinal Products for Human Use of the European Medicines Agency, based on the Phase III REACH2 and REACH3 trials in which Jakavi demonstrated superiority in overall response rate (ORR) compared to best available therapy (BAT). Results of REACH2 showed 62% ORR with Jakavi at Day 28, compared to 39% for BAT; and REACH3 demonstrated a significantly improved ORR at week 24 (50% vs. 26%) with a higher best ORR (76% vs. 60%) vs. BAT, among steroid-refractory/dependent chronic GvHD patients2,3.
“Five out of ten patients who receive allogeneic stem cell transplants experience the serious and sometimes fatal symptoms of graft-versus-host disease,” says Marie-France Tschudin, Novartis President of Innovative Medicines International and Chief Commercial Officer. “Jakavi, with this new indication in GvHD, will help to redefine treatment for patients who do not respond to first-line care.”
GvHD occurs when donor cells see the recipient's healthy cells as foreign and attack them. Symptoms of GvHD can appear in the skin, gastrointestinal tract, liver, mouth, eyes, genitals, lungs and joints. Approximately 50% of allogeneic stem cell transplant recipients will develop either acute or chronic GvHD. Both acute and chronic GvHD can be fatal and until now both have lacked an established standard of care for patients who do not adequately respond to first-line steroid treatment1,4-9. Currently, there are no other approved therapies for the treatment of GvHD after steroid failures1,2.
About Jakavi® (ruxolitinib)
Jakavi® (ruxolitinib) is an oral inhibitor of the JAK 1 and JAK 2 tyrosine kinases. Jakavi is approved by the European Commission for the treatment of adult patients with polycythemia vera (PV) who are resistant to or intolerant of hydroxyurea and for the treatment of disease- related splenomegaly or symptoms in adult patients with primary myelofibrosis (MF) (also known as chronic idiopathic MF), post-polycythemia vera MF or post-essential thrombocythemia MF, and also for patients aged 12 years and older with acute or chronic GvHD who have inadequate response to corticosteroids or other systemic therapies. Jakavi is approved in over 100 countries for patients with MF, including EU countries, Switzerland, Canada, Japan and in more than 85 countries for patients with PV, including EU countries, Switzerland, Japan and Canada. The exact indication for Jakavi varies by country. Additional worldwide regulatory filings are underway in MF and PV.
Novartis licensed ruxolitinib from Incyte for development and commercialization outside the United States. Ruxolitinib is marketed in the United States by Incyte as Jakafi® for adults with PV who have had an inadequate response to or are intolerant of hydroxyurea, for adults with intermediate or high-risk MF, for adult and pediatric patients 12 years and older with steroid-refractory acute GvHD, and adult and pediatric patients 12 years and older with chronic GvHD after failure of one or two lines of corticosteroids or other systemic therapy.
Jakavi is a registered trademark of Novartis AG in countries outside the United States. Jakafi is a registered trademark of Incyte. The safety and efficacy profile of Jakavi has not yet been established outside of its approved indications.
Find out more at https://www.novartis.com.
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Kymriah® (tisagenlecleucel) suspension
Novartis Kymriah® receives EC approval as first CAR-T cell therapy for adults with relapsed or refractory follicular lymphoma
May 04, 2022
- Kymriah offers patients in Europe with advanced follicular lymphoma a potentially definitive, single infusion CAR-T cell therapy with a remarkable safety profile
- Approval based on the Phase II ELARA trial showing Kymriah demonstrated high response rates in heavily pretreated patients, including an 86% overall response rate and a 69% complete response1
- Follicular lymphoma is an incurable malignancy in which patients often relapse despite treatment with several lines of therapy2,3
- Approval for relapsed or refractory follicular lymphoma is the third indication for Kymriah in the EU
Basel, May 4, 2022 — Novartis announced today that the European Commission (EC) has approved Kymriah® (tisagenlecleucel), a CAR-T cell therapy, for the treatment of adult patients with relapsed or refractory (r/r) follicular lymphoma (FL) after two or more lines of systemic therapy. The approval follows a positive opinion in March by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) and is applicable to all 27 European Union member states plus Iceland, Norway and Liechtenstein. This approval marks the third indication for Kymriah and makes it the first CAR-T cell therapy approved in the EU for these patients, which include those with r/r FL grade 1, 2 and 3A1.
“When follicular lymphoma fails to respond to treatment or comes back, it is typically more aggressive and difficult to treat; patients often end up cycling through multiple lines of therapy with decreasing benefit,” said Catherine Thieblemont, MD, PhD, Professor of Hematology in the Paris VII- University, France and Head of the Hemato-Oncology Unit of St-Louis Hospital in Paris. “The approval of Kymriah in Europe brings patients closer to a potentially definitive therapy, providing us hope for improved outcomes.”
The approval is based on the global Phase II ELARA trial showing that 86% of patients who were treated with Kymriah had a response, including 69% who had a complete response (CR)1. Prolonged durable response to treatment was demonstrated with an estimated 87% of patients who achieved a CR still in response at or more than nine months after initial response1. In the trial, 94 infused patients were evaluated for efficacy with a median follow-up of approximately 21 months1.
Among 97 patients evaluable for safety, the safety profile of Kymriah was remarkable1. Cytokine release syndrome (CRS) was reported in 50% of patients after Kymriah infusion, and no Grade 3 or 4 events were reported, as defined by the Lee scale1. Neurological adverse reactions occurred in 9% of patients (1% were Grade 3 or 4) within eight weeks after Kymriah infusion1. Severe infections (Grade 3 or 4) occurred in 16% of patients1.
“With this approval, we are pleased to be able to offer this transformative therapy to more people across the globe living with this advanced blood cancer,” said Marie-France Tschudin, President, Innovative Medicines International & Chief Commercial Officer, Novartis. “With long-lasting responses and a safety profile that allows for flexible administration, we are striving to rewrite cancer survival and alleviate the burden of this disease for patients and the healthcare system.”
In addition to r/r FL, Kymriah is approved for the treatment of pediatric and young adult patients up to and including 25 years of age with B cell acute lymphoblastic leukemia (ALL) that is refractory, in relapse post-transplant or in second or later relapse, and adult patients with r/r diffuse large B cell lymphoma (DLBCL) after two or more lines of systemic therapy1.
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Novartis CAR-T cell therapy Kymriah gets approval in EU for blood cancer subtype
May 05, 2022 5:54 AM ET Novartis AG (NVS)
By: Ravikash, SA News Editor
- The European Commission (EC) approved Novartis' (NYSE:NVS) CAR-T cell therapy Kymriah to treat adult patients with relapsed or refractory (r/r) follicular lymphoma (FL) after two or more lines of systemic therapy.
- https://seekingalpha.com/news/3833059-novartis-car-t-cell-therapy-kymriah-gets-approval-in-eu-for-blood-cancer-subtype
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- https://www.us.kymriah.com/
- https://www.novartis.us/sites/www.novartis.us/files/kymriah.pdf
Farxiga (dapagliflozin)
Farxiga met primary endpoint in DELIVER Phase III trial, reducing risk of cardiovascular death or worsening heart failure in patients with preserved ejection fraction
PUBLISHED5 May 2022
5 May 2022 07:05 BST
Results from the DELIVER and DAPA-HF Phase III trials demonstrate Farxiga’s efficacy in heart failure regardless of ejection fraction
High-level results from the DELIVER Phase III trial showed AstraZeneca’s Farxiga (dapagliflozin) reached a statistically significant and clinically meaningful reduction in the primary composite endpoint of cardiovascular (CV) death or worsening heart failure (HF). The trial was conducted in patients with HF with mildly reduced or preserved ejection fraction (defined as left ventricular ejection fraction [LVEF] greater than 40%).
HF is a chronic, long-term condition that worsens over time1. It affects nearly 64 million people globally2 and is associated with substantial morbidity and mortality3. There are several main categories of HF related to ejection fraction (EF), a measurement of the percentage of blood leaving the heart each time it contracts including: HF with reduced EF (HFrEF) (LVEF less than or equal to 40%), HF with mildly reduced EF (HFmrEF) (LVEF 41-49%) and preserved EF (HFpEF) (LVEF greater than or equal to 50%)4. Approximately half of all HF patients have mildly reduced or preserved EF with few therapeutic options available4,5. Farxiga already has approved indications relating to the treatment of type-2 diabetes (T2D), HFrEF and chronic kidney disease (CKD).
Dr. Scott Solomon, Professor of Medicine at Harvard Medical School and Brigham and Women’s Hospital and Principal Investigator of the DELIVER Phase III trial, said: “We are delighted to have met the primary endpoint in this patient population which has few treatment options. DELIVER is the largest and broadest trial to date in heart failure with mildly reduced or preserved ejection fraction. The results of DELIVER extend the benefit of dapagliflozin to the full spectrum of patients with heart failure.”
Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, AstraZeneca, said: "Today’s groundbreaking results coupled with those from the DAPA-HF trial show that Farxiga is effective in treating heart failure regardless of ejection fraction. These data build upon our previous studies demonstrating cardiorenal protection across patients with either diabetes, chronic kidney disease or heart failure.”
The safety and tolerability profile of Farxiga in the DELIVER Phase III trial were consistent with the well-established safety profile of the medicine.
The full DELIVER Phase III trial results will be submitted for presentation at a forthcoming medical meeting and regulatory submissions will be made in the coming months.
DELIVER
DELIVER was an international, randomised, double-blind, parallel-group, placebo-controlled, event-driven Phase III trial designed to evaluate the efficacy of Farxiga, compared with placebo, in the treatment of HF patients with LVEF greater than 40% with or without T2D. Farxiga was given once daily in addition to background therapy (regional standard of care for all comorbidities, including diabetes and hypertension, with the exception of concomitant use of a sodium-glucose cotransporter 2 [SGLT2] inhibitor)13. DELIVER is the largest clinical trial to date in HF patients with EF above 40%, with 6,263 randomised patients13,14.
The primary endpoint was the time to first occurrence of CV death, hospitalisation for HF (hHF) or an urgent HF visit. The secondary endpoint includes the total number of HF events (hHF or urgent HF visit) and CV death, change from baseline in the total symptom score of the Kansas City Cardiomyopathy Questionnaire at eight months, time to the occurrence of CV death and time to the occurrence of death from any cause13.
Farxiga
Farxiga (dapagliflozin) is a first-in-class, oral, once-daily SGLT2 inhibitor. Research has shown Farxiga’s efficacy in preventing and delaying cardiorenal disease, while also protecting the organs – important findings given the underlying links between the heart, kidneys and pancreas15-17. Damage to one of these organs can cause the other organs to fail, contributing to leading causes of death worldwide, including T2D, HF and CKD2,18-20.
In the US, Farxiga is approved as an adjunct to diet and exercise to improve glycaemic control in adults with T2D and in T2D to reduce the risk of hHF or CV death when added to standard of care based on the findings of the DECLARE-TIMI 58 Phase III CV outcomes trial17. Farxiga is also approved for the treatment of HFrEF and the treatment of CKD based on the findings of the DAPA-HF and DAPA-CKD Phase III trials. In the European Union, Forxiga is indicated as both monotherapy (when metformin is appropriate) and as part of combination therapy for the treatment of insufficiently controlled T2D, with the additional benefits of weight loss and blood-pressure reduction, as an adjunct to diet and exercise in adults with T2D. Forxiga is also approved for the treatment of symptomatic chronic HFrEF in adults with and without T2D and for the treatment of CKD in adults with and without T2D.
DapaCare is a robust programme of clinical trials to evaluate the potential CV, renal and organ protection benefits of Farxiga. It includes more than 35 completed and ongoing Phase IIb/III trials in more than 35,000 patients, as well as more than 2.5 million patient-years’ experience. Farxiga is currently being tested in the DAPA-MI Phase III trial - a first of its kind, indication-seeking registry-based randomised controlled trial in patients without T2D following an acute myocardial infarction (MI) or heart attack21.
Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca.
AstraZeneca Farxiga cuts risk of death/heart failure in certain patients in late-stage study
May 05, 2022 7:57 AM ET
By: Ravikash, SA News Editor
AstraZeneca (NASDAQ:AZN) said data from a late-stage study showed that its drug Farxiga helped reduce the risk of death or heart failure (HF) in certain patients.
https://seekingalpha.com/symbol/AZN
RINVOQ® (upadacitinib)
April 29, 2022
RINVOQ® (upadacitinib) Approved by U.S. FDA as an Oral Treatment for Adults with Active Ankylosing Spondylitis
- Across the two pivotal trials, RINVOQ delivered rapid and meaningful disease control with nearly half of ankylosing spondylitis (AS) patients achieving ASAS40 (51% and 44.5% with RINVOQ versus 26% and 18.2% with placebo) at week 14 compared to placebo1
- RINVOQ demonstrated significant improvement in signs and symptoms of AS at week 141-4
- FDA approval in AS marks the fifth indication for RINVOQ in chronic immune-mediated diseases1
NORTH CHICAGO, Ill., April 29, 2022 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced that the U.S. Food and Drug Administration (FDA) has approved RINVOQ® (upadacitinib; 15 mg, once daily) for the treatment of adults with active ankylosing spondylitis (AS) who have had an inadequate response or intolerance to one or more tumor necrosis factor (TNF) blockers.1
"Ankylosing spondylitis is a debilitating disease that often affects younger adults and, over time, can result in lasting structural damage that can take an emotional toll on a patient's life," said Thomas Hudson, M.D., senior vice president, research and development, chief scientific officer, AbbVie. "This latest approval demonstrates another important step forward in our mission to advance the standards of care in rheumatic diseases."
The FDA approval in AS is supported by efficacy and safety data from the Phase 3 SELECT-AXIS 2 clinical trial (Study 1) evaluating RINVOQ in patients who had an inadequate response or intolerance to one or two biologic disease-modifying anti-rheumatic drugs (bDMARDs) and the Phase 2/3 SELECT-AXIS 1 clinical trial evaluating RINVOQ in patients who were naïve to bDMARDs and had an inadequate response or intolerance to at least two nonsteroidal anti-inflammatory drugs (NSAIDs).1-3
"Many patients with ankylosing spondylitis do not achieve disease control with current biologic therapies and additional treatments are needed to help relieve the signs and symptoms of this disease," said Atul Deodhar, M.D., professor of medicine and medical director of the Rheumatology Clinics for the Division of Arthritis and Rheumatic Diseases at Oregon Health & Science University, and investigator of the SELECT-AXIS 1 trial. "With today's FDA approval, patients who do not respond to a TNF inhibitor have an additional oral treatment option, in partnership with their rheumatologist, to help take control of this disease."
In both SELECT-AXIS 1 and SELECT-AXIS 2 clinical trials, a significantly greater proportion of patients receiving RINVOQ 15 mg achieved an ASAS40* response, the primary endpoint, (51% and 44.5%, respectively) compared to those receiving placebo (26% and 18.2%, respectively) at week 14. Clinical responses were observed as early as week four in SELECT-AXIS 2 for ASAS40.1,3
"Currently, there are limited treatment options for people living with ankylosing spondylitis, particularly when painful symptoms persist despite being on a TNF blocker treatment," said Cassie Shafer, chief executive officer, Spondylitis Association of America (SAA). "The approval of a new medicine is welcome news to our community of patients, offering the potential to help more people find meaningful relief from the symptoms of AS and to help reach their treatment goals."
AS is a chronic inflammatory musculoskeletal disease primarily affecting the spine and characterized by debilitating symptoms of inflammatory back pain, stiffness and restricted mobility. An estimated one out of every 200 adults in the U.S., or approximately 1.1 million people, is affected by AS.5
Additional study results include the following:
Improvement in AS Signs & Symptoms at Week 141-3
Treatment with RINVOQ 15 mg resulted in improvements in the signs and symptoms of AS, including total back pain, as well as improvements in physical function (Bath Ankylosing Spondylitis Functional Index (BASFI)) and disease activity (Patient Global Assessment of Disease Activity) versus placebo at week 14.
In SELECT-AXIS 2, patients receiving RINVOQ 15 mg at week 14 experienced:
- A significantly greater mean decrease from baseline in Total Back Pain (-3.1 change from baseline) compared to those receiving placebo (-1.5).6
- A significantly greater improvement in physical function (-2.3 change from baseline) as assessed by mean change from baseline in BASFI compared to patients on placebo (-1.09).6
*ASAS40 is a composite index that measures disease activity.2 To achieve an ASAS40 response, a patient's disease activity must have improved by at least 40%, as well as improved by two units in at least three of four disease areas assessed, and the remaining area must not have gotten worse, including back pain, patient global assessment of disease activity, physical functional and morning stiffness.2
About SELECT-AXIS 1 and SELECT-AXIS 2 trial programs2,3
SELECT-AXIS 1 is a Phase 2/3, multicenter, randomized, double-blind, parallel-group, placebo-controlled study designed to evaluate the safety and efficacy of RINVOQ in adult patients with active ankylosing spondylitis (AS) who are bDMARD-naïve and had inadequate response to at least two NSAIDs or intolerance to/contraindication for NSAIDs. Period 2 is an open-label extension period to evaluate the long-term safety, tolerability and efficacy of RINVOQ in subjects who completed Period 1. More information on this trial can be found at www.clinicaltrials.gov (NCT03178487).
SELECT-AXIS 2 was conducted as a master study protocol that contains two standalone studies with randomization, data collection, analysis and reporting conducted independently. The Phase 3, randomized, placebo-controlled, double-blind studies are evaluating the efficacy and safety of RINVOQ compared with placebo on reduction of signs and symptoms in adult participants with active axial spondyloarthritis (axSpA), including bDMARD-IR AS (Study 1) and non-radiographic axial spondyloarthritis (nr-axSpA) (Study 2). More information on this trial can be found at https://www.clinicaltrials.gov/ (NCT04169373).
In both clinical trials, the primary endpoint was the percentage of subjects achieving an ASAS40 response after 14 weeks of treatment with RINVOQ versus placebo.
About RINVOQ® (upadacitinib)
Discovered and developed by AbbVie scientists, RINVOQ is a selective JAK inhibitor that is being studied in several immune-mediated inflammatory diseases. Based on enzymatic and cellular assays, RINVOQ demonstrated greater inhibitory potency for JAK-1 vs JAK-2, JAK-3, and TYK-2.1 The relevance of inhibition of specific JAK enzymes to therapeutic effectiveness and safety is not currently known.
In the U.S., RINVOQ 15 mg is approved for adults with moderately to severely active rheumatoid arthritis who have had an inadequate response or intolerance to one or more TNF blockers; adults with active psoriatic arthritis who have had an inadequate response or intolerance to one or more TNF blockers; and adults with active ankylosing spondylitis (AS) who have had an inadequate response or intolerance to one or more tumor necrosis factor (TNF) blockers.1 RINVOQ 45 mg is approved for use in adult patients with moderately to severely active ulcerative colitis who have had an inadequate response or intolerance to one or more TNF blockers as an induction therapy once daily for 8 weeks. The recommended dose of RINVOQ for maintenance treatment is 15 mg once daily. A dosage of 30 mg once daily may be considered for patients with refractory, severe or extensive disease. Discontinue RINVOQ if an adequate response is not achieved with the 30 mg dose. Use the lowest effective dosage needed to maintain response. RINVOQ 15 mg once daily can also be initiated in adults and children 12 years of age and older weighing at least 40 kg with refractory, moderate to severe atopic dermatitis whose disease is not adequately controlled with other system drug products, including biologics or when use of those therapies is inadvisable. In these children and adults less than 65 years of age who do not achieve an adequate response, the dose may be increased to 30 mg once daily.
Phase 3 trials of RINVOQ in rheumatoid arthritis, atopic dermatitis, psoriatic arthritis, axial spondyloarthritis, Crohn's disease, ulcerative colitis, giant cell arteritis and Takayasu arteritis are ongoing.7-13
RINVOQ® (upadacitinib) U.S. Use and Important Safety Information1
RINVOQ is a prescription medicine used to treat:
- Adults with moderate to severe rheumatoid arthritis when 1 or more tumor necrosis factor (TNF) blockers have been used, and did not work well or could not be tolerated.
- Adults with active psoriatic arthritis when 1 or more tumor necrosis factor (TNF) blockers have been used, and did not work well or could not be tolerated.
- Adults with moderate to severe ulcerative colitis when 1 or more medicines called TNF blockers have been used, and did not work well or could not be tolerated.
- Adults with active ankylosing spondylitis when 1 or more medicines called TNF blockers have been used, and did not work well or could not be tolerated.
It is not known if RINVOQ is safe and effective in children with juvenile idiopathic arthritis, psoriatic arthritis, ulcerative colitis, or ankylosing spondylitis.
- Adults and children 12 years of age and older with moderate to severe eczema (atopic dermatitis) that did not respond to previous treatment and whose eczema is not well controlled with other pills or injections, including biologic medicines, or when the use of other pills or injections is not recommended.
RINVOQ is safe and effective in children 12 years of age and older weighing at least 88 pounds (40 kg) with atopic dermatitis.
It is not known if RINVOQ is safe and effective in children under 12 years of age with atopic dermatitis.
For more information about AbbVie, please visit us at www.abbvie.com. Follow @abbvie on Twitter, Facebook, LinkedIn or Instagram.
SOURCE AbbVie
AbbVie announces FDA approval of Rinvoq for new arthritic indication
Apr. 29, 2022 3:09 PM ET
By: Dulan Lokuwithana, SA News Editor3 Comments
- AbbVie (NYSE:ABBV) announced on Friday that the U.S. Food and Drug Administration (FDA) approved its JAK inhibitor Rinvoq (upadacitinib) for the treatment of adults with active ankylosing spondylitis (AS), a rare form of arthritis that leads to pain and stiffness in the spine.
- https://seekingalpha.com/news/3829620-abbv-stock-slips-despite-fda-approval-of-rinvoq-for-new-arthritic-indication
- https://seekingalpha.com/symbol/ABBV
- https://www.abbvie.com/
- https://www.rinvoq.com/
BLINCYTO® (Blinatumomab)
BeiGene Announces the Approval in China of BLINCYTO® (Blinatumomab) for Injection for Pediatric Patients with Relapsed or Refractory B-Cell Precursor Acute Lymphoblastic Leukemia (ALL)
May 04, 2022 5:53 AM
CAMBRIDGE, Mass. & BASEL, Switzerland & BEIJING--(BUSINESS WIRE)-- BeiGene (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global, science-driven biotechnology company focused on developing innovative and affordable medicines to improve treatment outcomes and access for patients worldwide, today announced that the China National Medical Products Administration (NMPA) has granted conditional approval of BLINCYTO® (blinatumomab) for injection for the treatment of pediatric patients with relapsed or refractory (R/R) CD19-positive B-cell precursor acute lymphoblastic leukemia (ALL). The NMPA granted conditional approval for adult patients in this indication in December 2020.
Developed by Amgen and licensed to BeiGene in China under a strategic collaboration commenced in 2020, this is the second approval for BLINCYTO in China. The pediatric Supplemental Biologic License Application (sBLA) was submitted by BeiGene.
“This approval of BLINCYTO provides us with an opportunity to offer pediatric patients in China with relapsed or refractory B-cell precursor ALL the first approved biospecific immunotherapy treatment option for their disease,” commented Xiaobin Wu, Ph.D., President, Chief Operating Officer, and General Manager of China, at BeiGene. “We are proud to be able to offer BLINCYTO to help these young patients as they fight this disease. Our commercial organization of more than 3,100 people in China is excited to add this BLINCYTO indication to our portfolio, which includes 16 approved cancer treatments.”
BLINCYTO for injection for the treatment of adult patients with R/R CD19-positive B-cell precursor ALL was approved conditionally based on ex-China data and interim analysis results of the Phase 3 clinical trial of adult patients in China (NCT03476239). This conditional approval in pediatric patients with the above indication was granted based on ex-China research data and Chinese adult data. The full approval in this indication will depend on the results of a post-marketing study in China.
About BLINCYTO® (blinatumomab)
BLINCYTO is a BiTE® (bispecific T-cell engager) immuno-oncology therapy that targets CD19 surface antigens on B cells. BiTE molecules fight cancer by helping the body's immune system detect and target malignant cells by engaging T cells (a type of white blood cell capable of killing other cells perceived as threats) to cancer cells. By bringing T cells near cancer cells, the T cells can inject toxins and trigger cancer cell death (apoptosis). BiTE immuno-oncology therapies are currently being investigated for their potential to treat a wide variety of cancers.
BLINCYTO was granted breakthrough therapy and priority review designations by the U.S. Food and Drug Administration and is approved in the U.S. for the treatment of:
- relapsed or refractory CD-19 positive B-cell precursor ALL in adults and children.
- CD-19 positive B-cell precursor ALL in first or second complete remission with minimal residual disease (MRD) greater than or equal to 0.1% in adults and children. This indication is approved under accelerated approval based on MRD response rate and hematological relapse-free survival. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
In the European Union (EU), BLINCYTO is indicated as monotherapy for the treatment of:
- adults with Philadelphia chromosome negative CD19 positive relapsed or refractory B-precursor acute lymphoblastic leukemia (ALL). Patients with Philadelphia chromosome positive B-precursor ALL should have failed treatment with at least 2 tyrosine kinase inhibitors (TKIs) and have no alternative treatment options.
- adults with Philadelphia chromosome negative CD19 positive B-precursor ALL in first or second complete remission with minimal residual disease (MRD) greater than or equal to 0.1%.
- pediatric patients aged 1 year or older with Philadelphia chromosome negative CD19 positive B-precursor ALL which is refractory or in relapse after receiving at least two prior therapies or in relapse after receiving prior allogeneic hematopoietic stem cell transplantation
- pediatric patients aged 1 year or older with high-risk first relapsed Philadelphia chromosome negative CD19 positive B-precursor ALL as part of the consolidation therapy.
In China, BLINCYTO is indicated for the treatment of adult patients with relapsed or refractory B-cell precursor ALL.
To learn more about BeiGene, please visit www.beigene.com and follow us on Twitter at @BeiGeneGlobal.
BLINCYTO® and BiTE® are registered trademarks of Amgen Inc.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220504005602/en/
Source: BeiGene
Amgen, BeiGene's Blincyto gets conditional approval in China to treat blood cancer subtype in children
May 04, 2022 6:30 AM ET
By: Ravikash, SA News Editor
- China's National Medical Products Administration (NMPA) granted conditional approval to BeiGene (NASDAQ:BGNE) and Amgen's (NASDAQ:AMGN) Blincyto injection to treat children with relapsed or refractory (R/R) CD19-positive B-cell precursor acute lymphoblastic leukemia (ALL).
- https://seekingalpha.com/news/3831802-amgen-beigenes-blincyto-gets-conditional-approval-in-china-to-treat-blood-cancer-subtype-in-children
- https://seekingalpha.com/symbol/BGNE
- https://seekingalpha.com/symbol/AMGN
- https://www.beigene.com/
- https://www.amgen.com/
- https://www.blincyto.com/
Imfinzi (durvalumab) plus chemotherapy
Imfinzi plus chemotherapy granted Priority Review in the US for patients with locally advanced or metastatic biliary tract cancer based on TOPAZ-1 Phase III trial
PUBLISHED4 May 2022
4 May 2022 07:00 BST
First Phase III trial in this setting to show improved overall survival with an immunotherapy added to chemotherapy over standard-of-care chemotherapy alone
AstraZeneca’s supplemental Biologics License Application (sBLA) for Imfinzi (durvalumab), in combination with standard-of-care chemotherapy, has been accepted and granted Priority Review in the US for patients with locally advanced or metastatic biliary tract cancer (BTC).
The Food and Drug Administration (FDA) grants Priority Review to applications for medicines that, if approved, would offer significant improvements over available options by demonstrating safety or efficacy improvements, preventing serious conditions, or enhancing patient compliance.1 The Prescription Drug User Fee Act date, the FDA action date for their regulatory decision, is during the third quarter of 2022.
BTC is a group of rare and aggressive cancers that occur in the bile ducts and gallbladder. 2,3 Approximately 23,000 people in the US are diagnosed with BTC each year.2 These patients have a poor prognosis, with approximately 5% to 15% of patients with BTC surviving five years.4
Susan Galbraith, Executive Vice President, Oncology R&D, AstraZeneca, said: “People with advanced biliary tract cancer have faced poor outcomes and limited treatment options for too long, and today’s news for the TOPAZ-1 trial underscores the urgency to deliver new, effective therapies in this setting. We are working closely with the FDA to bring the first immunotherapy-based option to patients with this devastating cancer and potentially set a new standard of care with Imfinzi plus chemotherapy.”
The sBLA was based on results from an interim analysis of the TOPAZ-1 Phase III trial presented during the 2022 American Society of Clinical Oncology Gastrointestinal Cancers (ASCO GI) Symposium. The data showed Imfinzi plus chemotherapy (gemcitabine plus cisplatin) reduced the risk of death by 20% versus chemotherapy alone (based on a hazard ratio [HR] of 0.80; 95% confidence interval [CI], 0.66-0.97; 2-sided p=0.021). An estimated one in four (25%) patients treated with Imfinzi plus chemotherapy were alive at two years compared to one in 10 (10%) treated with chemotherapy alone.
Results also showed a statistically significant 25% reduction in the risk of disease progression or death with Imfinzi plus chemotherapy (HR, 0.75; 95% CI, 0.64-0.89; 2-sided p=0.001). The Imfinzi combination was generally well tolerated and did not increase the discontinuation rate due to adverse events compared to chemotherapy alone.
In December 2020, Imfinzi was granted Orphan Drug Designation in the US for the treatment of BTC.
TOPAZ-1
TOPAZ-1 is a randomised, double-blind, placebo controlled, multicentre, global Phase III trial of Imfinzi in combination with chemotherapy (gemcitabine plus cisplatin) versus placebo in combination with chemotherapy as a 1st-line treatment in 685 patients with unresectable advanced or metastatic BTC including intrahepatic and extrahepatic cholangiocarcinoma, and gallbladder cancer. Patients with ampullary carcinoma were excluded.
The primary endpoint is overall survival and key secondary endpoints included progression-free survival, objective response rate and safety. The trial was conducted in 105 centres across 17 countries including in the US, Europe, South America and several countries in Asia including South Korea, Thailand, Japan and China.
Imfinzi
Imfinzi (durvalumab) is a human monoclonal antibody that binds to the PD-L1 protein and blocks the interaction of PD-L1 with the PD-1 and CD80 proteins, countering the tumour’s immune-evading tactics and releasing the inhibition of immune responses.
Imfinzi is the only approved immunotherapy in the curative-intent setting of unresectable, Stage III non-small cell lung cancer (NSCLC) in patients whose disease has not progressed after chemoradiotherapy, and is the global standard of care in this setting based on the PACIFIC Phase III trial.
Imfinzi is also approved in the US, EU, Japan, China and many other countries around the world for the treatment of extensive-stage small cell lung cancer (ES-SCLC) based on the CASPIAN Phase III trial. In 2021, updated results from the CASPIAN trial showed Imfinzi plus chemotherapy tripled patient survival at three years versus chemotherapy alone.
Imfinzi is also approved for previously treated patients with advanced bladder cancer in several countries.
Since the first approval in May 2017, more than 100,000 patients have been treated with Imfinzi.
As part of a broad development programme, Imfinzi is being tested as a single treatment and in combinations with other anti-cancer treatments for patients with small cell lung cancer (SCLC), NSCLC, bladder cancer, several GI cancers, ovarian cancer, endometrial cancer, and other solid tumours.
In the past year, Imfinzi combinations have demonstrated clinical benefit in multiple additional cancer settings with positive Phase III trials in unresectable advanced liver cancer (HIMALAYA) and metastatic NSCLC (POSEIDON).
Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca.
AstraZeneca's Imfinzi gets FDA priority review for biliary tract cancer
May 05, 2022 6:08 AM ET AstraZeneca PLC (AZN) By: Ravikash, SA News Editor
- The U.S. Food and Drug Administration granted priority review to AstraZeneca's (NASDAQ:AZN) Imfinzi, in combination with standard-of-care chemotherapy, to treat patients with locally advanced or metastatic biliary tract cancer (BCT).
- https://seekingalpha.com/news/3833069-astrazenecas-imfinzi-gets-fda-priority-review-for-biliary-tract-cancer
- https://seekingalpha.com/symbol/AZN
- https://www.astrazeneca.com
- https://www.imfinzi.com/
Zuranolone (SAGE-217/BIIB125)
May 2, 2022
Sage Therapeutics and Biogen Initiate Rolling Submission of New Drug Application (NDA) to U.S. Food and Drug Administration for Zuranolone for the Potential Treatment of Major Depressive Disorder (MDD)
The companies expect to complete submission of the NDA for treatment of MDD in the second half of 2022; associated filing for postpartum depression anticipated in the first half of 2023
CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 2, 2022-- Sage Therapeutics, Inc. (Nasdaq: SAGE) and Biogen Inc. (Nasdaq: BIIB) initiated a rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for zuranolone in the treatment of major depressive disorder (MDD). Zuranolone is an investigational two-week, once-daily oral drug being developed for MDD and postpartum depression (PPD). The companies have submitted the nonclinical module of the NDA to the FDA and plan to submit the remaining components for the MDD filing in the second half of 2022.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20220429005707/en/
Data from the completed studies of zuranolone in the LANDSCAPE and NEST clinical development programs, including data from the ongoing open-label SHORELINE Study in MDD, as well as data from the completed clinical pharmacology studies, will comprise the full submission package. The rolling submission process allows completed sections of an NDA to be submitted to the FDA for review on an ongoing basis.
“There are millions of people living with depression and the initiation of the rolling NDA submission brings us one step closer to our goal of offering zuranolone as a potential new treatment option,” said Barry Greene, Chief Executive Officer at Sage. “We believe the results from the LANDSCAPE and NEST programs, in which zuranolone demonstrated rapid and sustained effects and a well-tolerated safety profile in clinical trials, support zuranolone as a potential novel treatment option for MDD, if approved. We look forward to providing an update when the rolling submission for zuranolone in MDD is complete, which we expect to occur in the second half of this year.”
“Zuranolone has the potential to help address a significant unmet medical need in depression as an innovative option in a therapeutic area where little has changed in the past 30 years,” said Priya Singhal, M.D., M.P.H., Head of Global Safety and Regulatory Sciences and Interim Head of R&D at Biogen. “We are committed to advancing the science and developing new approaches to treating mental health, a major public health challenge that was exacerbated by the COVID-19 pandemic.”
Zuranolone was granted Fast Track Designation by the FDA in 2017 in MDD and Breakthrough Therapy Designation in 2018. Sage and Biogen plan to submit an associated NDA filing for PPD in the first half of 2023.
About Zuranolone
Zuranolone (SAGE-217/BIIB125) is a once-daily, two-week, investigational drug in development for the treatment of major depressive disorder (MDD) and postpartum depression (PPD). Zuranolone is an investigational oral neuroactive steroid (NAS) GABA-A receptor positive allosteric modulator (PAM). The GABA system is the major inhibitory signaling pathway of the brain and central nervous system and contributes to regulating brain function. Zuranolone has been granted Fast Track and Breakthrough Therapy Designation for MDD and Fast Track Designation for PPD by the U.S. Food & Drug Administration.
Zuranolone is being evaluated in the LANDSCAPE and NEST clinical development programs. The two development programs include multiple studies examining use of zuranolone in several thousand people with a variety of dosing, clinical endpoints, and treatment paradigms. The LANDSCAPE program includes five studies of zuranolone in people with MDD (MDD-201B, MOUNTAIN, SHORELINE, WATERFALL, and CORAL Studies). The NEST program includes two placebo-controlled studies of zuranolone in people with PPD (ROBIN and SKYLARK Studies). Additionally, Shionogi completed a Phase 2 study of zuranolone in Japan in people with MDD.
For more information, please visit www.sagerx.com.
We routinely post information that may be important to investors on our website at www.biogen.com. Follow us on social media - Twitter, LinkedIn, Facebook, YouTube.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220429005707/en/
Source: Sage Therapeutics, Inc.
Sage, Biogen begin rolling submission with FDA for zuranolone to treat depression
May 02, 2022 7:18 AM ET
Sage Therapeutics, Inc. (SAGE), BIIB
By: Ravikash, SA News Editor
- Sage Therapeutics (NASDAQ:SAGE) and Biogen (NASDAQ:BIIB) began a rolling submission of a new drug application (NDA) with the U.S. Food and Drug Administration (FDA) for approval of zuranolone to treat major depressive disorder (MDD).
- https://seekingalpha.com/news/3829885-sage-biogen-begin-rolling-submission-with-fda-for-zuranolone-to-treat-depression
- https://seekingalpha.com/symbol/SAGE
- https://seekingalpha.com/symbol/BIIB
- https://www.sagerx.com/
- https://www.sagerx.com/programs-research/pipeline/
VBI Vaccines Announces European Commission Marketing Authorisation for PreHevbri™, a 3-Antigen Adult Hepatitis B Vaccine
May 2, 2022 | Hepatitis B, Press Releases
- PreHevbri™ is the only approved 3-antigen hepatitis B vaccine for adults in the EU and EEA
- Approval follows the positive opinion granted by European Committee for Medicinal Products for Human Use (CHMP) in February 2022
- Regulatory review in the United Kingdom is ongoing as part of the European Commission Decision Reliance Procedure
VBI Vaccines Inc. (Nasdaq: VBIV) (VBI), a biopharmaceutical company driven by immunology in the pursuit of powerful prevention and treatment of disease, today announced that the European Commission (EC) has granted Marketing Authorisation for PreHevbri™ [Hepatitis B vaccine (recombinant, adsorbed)] for active immunisation against infection caused by all known subtypes of the hepatitis B virus (HBV) in adults. It can also be expected that hepatitis D will be prevented by immunisation with PreHevbri as hepatitis D (caused by the delta agent) does not occur in the absence of hepatitis B infection. The use of PreHevbri should be in accordance with official recommendations. PreHevbri contains the full antigenic composition of the hepatitis B virus surface antigen, including the S, pre-S2, and pre-S1 HBV surface antigens, and is the only approved 3-antigen HBV vaccine for adults in the European Union (EU) and in the European Economic Area (EEA) countries – Iceland, Liechtenstein, and Norway.
“We are excited to be able to announce the approval of PreHevbri in Europe, marking our second major approval for this differentiated HBV vaccine in five months, following the U.S. FDA approval at the end of November last year,” said Jeff Baxter, VBI’s President and CEO. “Hepatitis B is a highly infectious, under-reported, persistent public health problem in Europe and we believe PreHevbri has the potential to be a meaningful new tool for healthcare providers as they endeavor to fight it. We are committed to being part of this fight and are working hard to make PreHevbri available in different European countries as quickly as possible.”
The European Commission’s centralized marketing authorisation is valid in all EU Member States as well as in the European Economic Area (EEA) countries (Iceland, Liechtenstein, and Norway). VBI expects to make PreHevbri available in certain European countries beginning at the end of 2022.
The approval follows a positive opinion granted in February 2022 by the EMA’s Committee for Medicinal Products for Human Use (CHMP), which was based on the positive results from two pivotal, randomized, double-blind, controlled Phase 3 clinical studies, PROTECT and CONSTANT. Data from these studies were published, respectively, in The Lancet Infectious Diseases in May 2021 and The Journal of the American Medical Association Network Open in October 2021. Both studies compared PreHevbri to Engerix-B, a single-antigen HBV vaccine. Results from the PROTECT study showed that PreHevbri elicited higher rates of seroprotection in all subjects age 18+ (91.4% vs. 76.5%), including in adults age 45+ (89.4% vs. 73.1%). The integrated safety analysis of both studies demonstrated good tolerability with no unexpected reactogenicity. The most common adverse events in all age groups were injection site pain and tenderness, myalgia, and fatigue, all which generally resolved without intervention in 1-2 days.
VBI continues to support the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) review as part of the EC Decision Reliance Procedure (ECDRP), which was initiated upon receipt of the positive CHMP opinion in February.
About Hepatitis B
Hepatitis B is one of the world’s most significant infectious disease threats with more than 290 million people infected globally. HBV infection is the leading cause of liver disease and, with current treatments, it is very difficult to cure, with many patients going on to develop liver cancers. An estimated 900,000 people die each year from complications of chronic HBV such as liver decompensation, cirrhosis, and hepatocellular carcinoma.
About PreHevbri™
VBI’s hepatitis B vaccine is the only 3-antigen hepatitis B vaccine, comprised of the three hepatitis B surface antigens of the hepatitis B virus – S, pre-S1, and pre-S2. It is approved for use in the European Union/European Economic Area, the United States, and Israel. The brand names for this vaccine are : PreHevbri™ (EU/EEA), PreHevbrio™ (US), and Sci-B-Vac® (Israel).
Full European Summary of Product Characteristics for PreHevbri are available from the EMA website at www.ema.europa.eu.
Please visit www.PreHevbrio.com for U.S. Important Safety Information for PreHevbrio™ [Hepatitis B Vaccine (Recombinant)], or please see U.S. Full Prescribing Information.
VBI is headquartered in Cambridge, Massachusetts, with research operations in Ottawa, Canada, and a research and manufacturing site in Rehovot, Israel. For more information, visit www.vbivaccines.com.
VBI Vaccines stock rises as hepatitis B shot PreHevbri gets approval in EU
May 02, 2022 8:58 AM ET
By: Ravikash, SA News Editor
- VBI Vaccines (NASDAQ:VBIV) said the European Commission (EC) granted marketing authorization to its vaccine PreHevbri for active immunization against infection caused by all known subtypes of the hepatitis B virus (HBV) in adults.
- https://seekingalpha.com/news/3829970-vbi-vaccines-stock-rises-as-hepatitis-b-shot-prehevbri-gets-approval-in-eu
- https://seekingalpha.com/symbol/VBIV
- https://www.prehevbrio.com/
- https://www.vbivaccines.com/
KEYTRUDA® (pembrolizumab)
European Commission Approves Merck’s KEYTRUDA® (pembrolizumab) for Patients With Microsatellite Instability-High (MSI-H) or Deficient Mismatch Repair (dMMR) Tumors in Five Different Types of Cancer
April 29, 2022 6:45 am ET
KEYTRUDA is the First Immunotherapy to be Approved for Patients with MSI-H/dMMR Biomarkers in Five Different Types of Cancer in Europe
KENILWORTH, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced that the European Commission has approved KEYTRUDA, Merck’s anti-PD-1 therapy, as monotherapy for the treatment of microsatellite instability-high (MSI-H) or deficient mismatch repair (dMMR) tumors in adults with: unresectable or metastatic colorectal cancer after previous fluoropyrimidine-based combination therapy; advanced or recurrent endometrial carcinoma, who have disease progression on or following prior treatment with a platinum-containing therapy in any setting and who are not candidates for curative surgery or radiation; unresectable or metastatic gastric, small intestine or biliary cancer, who have disease progression on or following at least one prior therapy. This is the second approval for KEYTRUDA in Europe based on the MSI-H/dMMR biomarker. KEYTRUDA is also approved for the first-line treatment of metastatic MSI-H or dMMR colorectal cancer in adults.
“Our company has a strong track record of applying precision medicine, through biomarkers like MSI-H and dMMR, to help identify patients most likely to respond to KEYTRUDA based on the genetic makeup of their individual cancer,” said Dr. Scot Ebbinghaus, vice president, global clinical development, Merck Research Laboratories. “For patients with MSI-H/dMMR colorectal cancer, KEYTRUDA monotherapy was approved in Europe as a first-line option in January 2021. Building on that approval, we are pleased that KEYTRUDA is now approved for the treatment of additional MSI-H/dMMR tumors, in certain second- or later-line patients with colorectal, endometrial, gastric, small intestine or biliary cancer.”
“In the two studies supporting this approval, KEYTRUDA monotherapy showed strong objective response rates and durability of response in patients with five different types of MSI-H/dMMR cancers,” said Dr. Aurélien Marabelle, Immuno-Oncologist at Gustave Roussy Cancer Center and Professor of Clinical Immunology at the University of Paris Saclay. “The EU approval of KEYTRUDA is an important milestone for patients living with these MSI-H/dMMR cancers who have had few treatment options and face worse outcomes when diagnosed at an advanced stage.”
This approval allows marketing of KEYTRUDA monotherapy in all 27 European Union (EU) member states plus Iceland, Lichtenstein, Norway and Northern Ireland.
Data Supporting the European Approval
The approval was based on data from KEYNOTE-164 (NCT02460198) and KEYNOTE-158 (NCT02628067), multicenter, non-randomized, open-label Phase 2 trials evaluating KEYTRUDA in patients with advanced MSI-H or dMMR solid tumors. The KEYNOTE-164 trial enrolled 124 patients with unresectable or metastatic MSI-H or dMMR colorectal cancer that progressed following prior fluoropyrimidine-based therapy in combination with irinotecan and/or oxaliplatin. The KEYNOTE-158 trial enrolled 355 patients with unresectable or metastatic MSI-H or dMMR solid tumors, including endometrial, gastric, small intestine or biliary cancer. Microsatellite instability or MMR tumor status was determined by prospectively using polymerase chain reaction or immunohistochemistry, respectively. Patients received KEYTRUDA 200 mg administered intravenously every three weeks until unacceptable toxicity or disease progression. Clinically stable patients with initial evidence of disease progression were permitted to remain on treatment until disease progression was confirmed. Patients without disease progression were treated for up to 24 months (up to 35 cycles). The primary efficacy outcome measure for the trials was objective response rate (ORR) as assessed by blinded independent central review using RECIST v1.1. The secondary efficacy outcome measures for the trials included duration of response (DOR), progression-free survival and overall survival.
Efficacy results from the KEYNOTE-164 and KEYNOTE-158 trials are summarized below. For patients with:
- Colorectal cancer (n=124), the ORR was 34% (95% CI, 25.6-42.9), including a complete response (CR) rate of 10% and a partial response (PR) rate of 24%, at a median follow-up time of 37.3 months (range, 0.1 to 65.2). Median DOR was not reached (range, 4.4 to 58.5+ months), and of responding patients, 92% had responses lasting at least three years.
- Endometrial cancer (n=83), the ORR was 51% (95% CI, 39.4-61.8), including a CR rate of 16% and a PR rate of 35%, at a median follow-up time of 21.9 months (range, 1.5 to 64.0). Median DOR was not reached (range, 2.9 to 60.4+ months), and of responding patients, 85% had responses lasting at least one year, and 60% had responses lasting at least three years.
- Gastric cancer (n=51), the ORR was 37% (95% CI, 24.1-51.9), including a CR rate of 14% and a PR rate of 24%, at a median follow-up time of 13.9 months (range, 1.1 to 66.9). Median DOR was not reached (range, 6.2 to 63.0+ months), and of responding patients, 90% had responses lasting at least one year, and 81% had responses lasting at least three years.
- Small intestine cancer (n=27), the ORR was 56% (95% CI, 35.3-74.5), including a CR rate of 15% and a PR rate of 41%, at a median follow-up time of 29.1 months (range, 4.2 to 67.7). Median DOR was not reached (range, 3.7+ to 57.3+ months), and of responding patients, 93% had responses lasting at least one year, and 73% had responses lasting at least three years.
- Biliary cancer (n=22), the ORR was 41% (95% CI, 20.7-63.6), including a CR rate of 14% and a PR rate of 27%, at a median follow-up time of 19.4 months (range, 1.1 to 60.8). Median DOR was 30.6 months (range, 6.2 to 46.0+), and of responding patients, 89% had responses lasting at least one year, and 42% had responses lasting at least three years.
The safety of KEYTRUDA as monotherapy has been evaluated in 7,148 patients with advanced melanoma, resected stage III melanoma (adjuvant therapy), non-small cell lung cancer, classical Hodgkin lymphoma, urothelial carcinoma, head and neck squamous cell carcinoma, colorectal cancer, endometrial, gastric, small intestine, biliary, pancreatic cancer or adjuvant therapy of renal cell carcinoma across four doses (2 mg/kg bodyweight [bw] every three weeks, 200 mg every three weeks or 10 mg/kg bw every two or three weeks) in clinical studies. In this patient population, the most frequent adverse reactions with KEYTRUDA were fatigue (31%), diarrhea (22%) and nausea (21%). The majority of adverse reactions reported for KEYTRUDA monotherapy were of Grades 1 or 2 severity. The most serious adverse reactions were immune-related adverse reactions and severe infusion-related reactions. The incidences of immune-related adverse reactions were 36.1% for all Grades and 8.9% for Grades 3-5 for KEYTRUDA monotherapy in the adjuvant setting (n=1,480) and 24.2% for all Grades and 6.4% for Grades 3-5 for KEYTRUDA monotherapy in the metastatic setting (n=5,375). No new immune-related adverse reactions were identified in the adjuvant setting.
About Microsatellite Instability-High (MSI-H) and Deficient Mismatch Repair (dMMR)
Microsatellite instability (MSI) and deficient mismatch repair (dMMR) are biomarkers that have been identified in many different types of cancer and that can be hereditary or random. MSI is a change that occurs in the DNA of certain cells, such as cancer cells, in which the number of repeated DNA bases in a microsatellite (which is a short, repeated sequence of DNA) is different from what it was when the microsatellite was inherited. dMMR describes cells that have mutations in certain genes involved in correcting mistakes made when DNA is copied into a cell when dividing. High levels of MSI (MSI-H) and dMMR can occur when a cell is unable to repair mistakes during that division process.
About KEYTRUDA® (pembrolizumab) Injection, 100 mg
KEYTRUDA is an anti-programmed death receptor-1 (PD-1) therapy that works by increasing the ability of the body’s immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.
Merck has the industry’s largest immuno-oncology clinical research program. There are currently more than 1,700 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.
Selected KEYTRUDA® (pembrolizumab) Indications in the U.S.
Melanoma
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.
KEYTRUDA is indicated for the adjuvant treatment of adult and pediatric (12 years and older) patients with stage IIB, IIC, or III melanoma following complete resection.
Non-Small Cell Lung Cancer
KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) ≥1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is:
- stage III where patients are not candidates for surgical resection or definitive chemoradiation, or
- metastatic.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS ≥1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.
Head and Neck Squamous Cell Cancer
KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [Combined Positive Score (CPS) ≥1] as determined by an FDA-approved test.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.
Classical Hodgkin Lymphoma
KEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).
KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.
Primary Mediastinal Large B-Cell Lymphoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy.
KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.
Urothelial Carcinoma
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC):
- who are not eligible for any platinum-containing chemotherapy, or
- who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
Non-muscle Invasive Bladder Cancer
KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.
Microsatellite Instability-High or Mismatch Repair Deficient Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options.
This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.
Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).
Gastric Cancer
KEYTRUDA, in combination with trastuzumab, fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of patients with locally advanced unresectable or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma.
This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Esophageal Cancer
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic esophageal or gastroesophageal junction (GEJ) (tumors with epicenter 1 to 5 centimeters above the GEJ) carcinoma that is not amenable to surgical resection or definitive chemoradiation either:
- in combination with platinum- and fluoropyrimidine-based chemotherapy, or
- as a single agent after one or more prior lines of systemic therapy for patients with tumors of squamous cell histology that express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
Cervical Cancer
KEYTRUDA, in combination with chemotherapy, with or without bevacizumab, is indicated for the treatment of patients with persistent, recurrent, or metastatic cervical cancer whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test.
Hepatocellular Carcinoma
KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Merkel Cell Carcinoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Renal Cell Carcinoma
KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of adult patients with advanced renal cell carcinoma (RCC).
KEYTRUDA is indicated for the adjuvant treatment of patients with RCC at intermediate-high or high risk of recurrence following nephrectomy, or following nephrectomy and resection of metastatic lesions.
Endometrial Carcinoma
KEYTRUDA, as a single agent, is indicated for the treatment of patients with advanced endometrial carcinoma that is MSI-H or dMMR, as determined by an FDA-approved test, who have disease progression following prior systemic therapy in any setting and are not candidates for curative surgery or radiation.
Tumor Mutational Burden-High Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [≥10 mutations/megabase] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.
Cutaneous Squamous Cell Carcinoma
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) or locally advanced cSCC that is not curable by surgery or radiation.
Triple-Negative Breast Cancer
KEYTRUDA is indicated for the treatment of patients with high-risk early-stage triple-negative breast cancer (TNBC) in combination with chemotherapy as neoadjuvant treatment, and then continued as a single agent as adjuvant treatment after surgery.
KEYTRUDA, in combination with chemotherapy, is indicated for the treatment of patients with locally recurrent unresectable or metastatic TNBC whose tumors express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
For more information, visit www.merck.com and connect with us on Twitter, Facebook, Instagram, YouTube and LinkedIn.
Please see Prescribing Information for KEYTRUDA (pembrolizumab) at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_pi.pdf and Medication Guide for KEYTRUDA at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_mg.pdf .
Source: Merck & Co., Inc.
Merck wins European approval to market Keytruda for more cancer indications
Apr. 29, 2022 7:09 AM ET
By: Dulan Lokuwithana, SA News Editor
- Merck (NYSE:MRK) announced on Friday that the European Commission approved its anti-PD-1 therapy, Keytruda for a few more indications in cancer.
- https://seekingalpha.com/news/3829276-merck-wins-european-approval-to-market-keytruda-for-more-cancer-indications
- https://seekingalpha.com/symbol/MRK
- https://www.keytruda.com/
- https://www.merck.com/
Evrysdi® (risdiplam)
Thursday, Apr 28, 2022
New Three-Year Data for Genentech’s Evrysdi (risdiplam) Show Long-Term Improvements in Survival and Motor Milestones in Babies With Type 1 Spinal Muscular Atrophy (SMA)
91% of infants treated with Evrysdi in the FIREFISH study were still alive at three years
Infants treated with Evrysdi maintained or continued to improve in measures of motor function, including their ability to sit without support for 5 and 30 seconds
Evrysdi has proven efficacy in infants and adults, with more than 5,000 patients treated to date
South San Francisco, CA -- April 28, 2022 --
Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced new three-year data from the FIREFISH study, including one-year data from the open label extension, reinforcing the long-term efficacy and safety of Evrysdi® (risdiplam) in infants with symptomatic Type 1 spinal muscular atrophy (SMA). The data showed an estimated 91% of infants (n=58) treated with Evrysdi were alive after three years of treatment. The Evrysdi-treated infants continued to improve or maintain motor functions, including the ability to swallow, sit without support, stand with support and walk while holding on, between two and three years of treatment. Without treatment, children with Type 1 SMA are never able to sit without support. The study also showed overall continued reductions in serious adverse events (SAEs) and hospitalizations over time.
The FIREFISH study evaluated the efficacy and safety of Evrysdi in infants aged 1-7 months at the time of enrollment with Type 1 SMA. The study was in two parts, with Part 1 being the dose-finding period and Part 2 evaluating the efficacy and safety at the dose selected in Part 1. The pooled population includes participants treated with Evrysdi at the approved dose for a minimum of three years. These long-term data will be presented at the 14th European Paediatric Neurology Society (EPNS) Congress, April 28 – May 2, 2022.
“These long-term results in babies treated with Evrysdi are very encouraging, with the vast majority improving or maintaining motor functions after three years. Without treatment, they would typically not survive beyond two years of age,” said Levi Garraway, M.D., Ph.D., Genentech’s chief medical officer and head of Global Product Development. “Support for the compelling efficacy of Evrysdi continues to grow for a broad range of people, including infants with one of the most severe forms of SMA.”
Infants treated with Evrysdi maintained or continued to improve in their ability to sit without support between 24-36 months. Among the infants with an available assessment (n=48) treated with Evrysdi, 32 infants maintained and 4 gained the ability to sit without support for at least 5 seconds since month 24, as assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III). In addition, 20 infants maintained and 15 gained the ability to sit without support for at least 30 seconds. No infant who gained the ability to sit without support lost this ability after three years of treatment. The majority of infants treated with Evrysdi maintained the ability to feed orally and swallow up to month 36.
Most of the infants treated with Evrysdi continued to improve or maintain measures of the Hammersmith Infant Neurological Examination 2 (HINE-2) between 24-36 months, including being able to hold their heads upright (36 maintained, 3 gained and none lost the ability since month 24), pivot while sitting (15 maintained, 11 gained and none lost the ability), stand with support (6 maintained, 5 gained and 1 lost the ability) and walk while holding on (1 maintained, 2 gained and none lost the ability).
The most common adverse events (AEs) were pyrexia (60%), upper respiratory tract infection (57%), pneumonia (43%), constipation (26%), nasopharyngitis (24%), diarrhea (21%), rhinitis (19%), vomiting (19%) and cough (17%). The most common SAEs were pneumonia (36%), respiratory distress (10%), viral pneumonia (9%), acute respiratory failure (5%) and respiratory failure (5%). The rate of AEs, including pneumonia, continued to decrease over time. The rate of SAEs similarly decreased, with a reduction of approximately 50% after each 12-month treatment period and a 78% reduction between the first and third year of treatment. All AEs and SAEs reported were reflective of the underlying disease and there were no treatment-related AEs leading to withdrawal or treatment discontinuation. The rate of hospitalizations decreased from 1.24 hospitalizations per patient year over 12 months to 0.70 hospitalizations over 36 months. No additional deaths have occurred since the primary analysis of FIREFISH, up to the data cut-off of this analysis (November 23, 2021).
Genentech leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.
About Evrysdi® (risdiplam)
Evrysdi is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency. Evrysdi is administered daily at home in liquid form by mouth or by feeding tube.
Evrysdi is designed to treat SMA by increasing and sustaining the production of SMN protein in the central nervous system (CNS) and peripheral tissues as demonstrated in animal models. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and movement.
Evrysdi was granted PRIME designation by the European Medicines Agency (EMA) in 2018 and Orphan Drug Designation by the U.S. Food and Drug Administration in 2017. In 2021 Evrysdi was awarded Drug Discovery of the Year by the British Pharmacological Society as well as the Society for Medicines Research award for Drug Discovery. Evrysdi is currently approved in 76 countries and the dossier is under review in a further 29 countries.
Evrysdi is currently being evaluated in five multicenter trials in people with SMA:
- FIREFISH (NCT02913482) – an open-label, two-part pivotal clinical trial in infants with Type 1 SMA. Part 1 was a dose-escalation study in 21 infants with the primary objective of assessing the safety profile of Evrysdi in infants and determining the dose for Part 2. Part 2 is a pivotal, single-arm study of Evrysdi in 41 infants with Type 1 SMA treated for two years, followed by an open-label extension. Enrollment for Part 2 was completed in November 2018. The primary objective of Part 2 was to assess efficacy as measured by the proportion of infants sitting without support after 12 months of treatment, as assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development – Third Edition (BSID-III) (defined as sitting without support for 5 seconds). The study met its primary endpoint.
- SUNFISH (NCT02908685) – SUNFISH is a two-part, double-blind, placebo-controlled pivotal study in people aged 2-25 years with Types 2 or 3 SMA. Part 1 (n=51) determined the dose for the confirmatory Part 2. Part 2 (n=180) evaluated motor function using the total score of Motor Function Measure 32 (MFM-32) at 12 months. MFM-32 is a validated scale used to evaluate fine and gross motor function in people with neurological disorders, including SMA. The study met its primary endpoint.
- JEWELFISH (NCT03032172) – an open-label exploratory trial designed to assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) in people with SMA aged 6 months to 60 years who received other investigational or approved SMA therapies for at least 90 days prior to receiving Evrysdi. The study has completed recruitment (n=174).
- RAINBOWFISH (NCT03779334) – an open-label, single-arm, multicenter study, investigating the efficacy, safety, PK and PD of Evrysdi in babies (~n=25), from birth to six weeks of age (at first dose) with genetically diagnosed SMA who are not yet presenting with symptoms. The study is fully enrolled.
- MANATEE (NCT05115110) – a global phase 2/3 clinical study to evaluate the safety and efficacy of GYM329 (RO7204239), an anti-myostatin molecule targeting muscle growth, in combination with Evrysdi for the treatment of SMA in patients 2-10 years of age. The FDA Office of Orphan Products Development granted GYM329 Orphan Drug Designation for the treatment of patients with SMA in December 2021. The study is commencing recruitment in Q2 2022.
What is Evrysdi?
Evrysdi is a prescription medicine used to treat spinal muscular atrophy (SMA) in adults and children 2 months of age and older.
It is not known if Evrysdi is safe and effective in children under 2 months of age.
- https://www.evrysdi.com/
- Please see the full Prescribing Information for additional Important Safety Information.
For additional information about the company, please visit http://www.gene.com.
Genentech's Evrysdi shows long-term improvements in spinal muscular atrophy patients
Apr. 29, 2022 9:59 AM ET
By: Jonathan Block, SA News Editor
- Evrysdi (risdiplam), a treatment for spinal muscular atrophy from Roche (OTCQX:RHHBY) unit Genentech, demonstrated improvements in survival and motor functions after three years.
- https://seekingalpha.com/news/3829435-genentech-roche-evrysdi-shows-long-term-improvements-in-spinal-muscular-atrophy-patients
- https://seekingalpha.com/symbol/RHHBY
- https://www.evrysdi.com/
- https://www.gene.com/
tirzepatide
Lilly's tirzepatide delivered up to 22.5% weight loss in adults with obesity or overweight in SURMOUNT-1
April 28, 2022Download PDF
Participants taking tirzepatide lost up to 52 lb. (24 kg) in this 72-week phase 3 study
63% of participants taking tirzepatide 15 mg achieved at least 20% body weight reductions as a key secondary endpoint
INDIANAPOLIS, April 28, 2022 /PRNewswire/ -- Tirzepatide (5 mg, 10 mg, 15 mg) achieved superior weight loss compared to placebo at 72 weeks of treatment in topline results from Eli Lilly and Company's (NYSE: LLY) SURMOUNT-1 clinical trial, with participants losing up to 22.5% (52 lb. or 24 kg) of their body weight for the efficacy estimandi. This study enrolled 2,539 participants and was the first phase 3 global registration trial evaluating the efficacy and safety of tirzepatide in adults with obesity, or overweight with at least one comorbidity, who do not have diabetes. Tirzepatide met both co-primary endpoints of superior mean percent change in body weight from baseline and greater percentage of participants achieving body weight reductions of at least 5% compared to placebo for both estimandsii. The study also achieved all key secondary endpoints at 72 weeks.
For the efficacy estimand, participants taking tirzepatide achieved average weight reductions of 16.0% (35 lb. or 16 kg on 5 mg), 21.4% (49 lb. or 22 kg on 10 mg) and 22.5% (52 lb. or 24 kg on 15 mg), compared to placebo (2.4%, 5 lb. or 2 kg). Additionally, 89% (5 mg) and 96% (10 mg and 15 mg) of people taking tirzepatide achieved at least 5% body weight reductions compared to 28% of those taking placebo.
In a key secondary endpoint, 55% (10 mg) and 63% (15 mg) of people taking tirzepatide achieved at least 20% body weight reductions compared to 1.3% of those taking placebo. In an additional secondary endpoint not controlled for type 1 error, 32% of participants taking tirzepatide 5 mg achieved at least 20% body weight reductions. The mean baseline body weight of participants was 231 lb. (105 kg).
"Obesity is a chronic disease that often does not receive the same standard of care as other conditions, despite its impact on physical, psychological and metabolic health, which can include increased risk of hypertension, heart disease, cancer and decreased survival," said Louis J. Aronne, MD, FACP, DABOM, director of the Comprehensive Weight Control Center and the Sanford I. Weill Professor of Metabolic Research at Weill Cornell Medicine, obesity expert at NewYork-Presbyterian/Weill Cornell Medical Center and Investigator of SURMOUNT-1. "Tirzepatide delivered impressive body weight reductions in SURMOUNT-1, which could represent an important step forward for helping the patient and physician partnership treat this complex disease."
For the treatment-regimen estimandiii, results showed:
- Average body weight reductions: 15.0% (5 mg), 19.5% (10 mg), 20.9% (15 mg), 3.1% (placebo)
- Percentage of participants achieving body weight reductions of ≥5%: 85% (5 mg), 89% (10 mg), 91% (15 mg), 35% (placebo)
- Percentage of participants achieving body weight reductions of ≥20%: 30% (5 mg, not controlled for type 1 error), 50% (10 mg), 57% (15 mg), 3.1% (placebo)
The overall safety and tolerability profile of tirzepatide was similar to other incretin-based therapies approved for the treatment of obesity. The most commonly reported adverse events were gastrointestinal-related and generally mild to moderate in severity, usually occurring during the dose escalation period. For those treated with tirzepatide (5 mg, 10 mg and 15 mg, respectively), nausea (24.6%, 33.3%, 31.0%), diarrhea (18.7%, 21.2%, 23.0%), vomiting (8.3%, 10.7%, 12.2%) and constipation (16.8%, 17.1%, 11.7%) were more frequently experienced compared to placebo (9.5% [nausea], 7.3% [diarrhea], 1.7% [vomiting], 5.8% [constipation]).
Treatment discontinuation rates due to adverse events were 4.3% (5 mg), 7.1% (10 mg), 6.2% (15 mg) and 2.6% (placebo). The overall treatment discontinuation rates were 14.3% (5 mg), 16.4% (10 mg), 15.1% (15 mg) and 26.4% (placebo).
Participants who had pre-diabetes at study commencement will remain enrolled in SURMOUNT-1 for an additional 104 weeks of treatment following the initial 72-week completion date to evaluate the impact on body weight and the potential differences in progression to type 2 diabetes at three years of treatment with tirzepatide compared to placebo.
"Tirzepatide is the first investigational medicine to deliver more than 20 percent weight loss on average in a phase 3 study, reinforcing our confidence in its potential to help people living with obesity," said Jeff Emmick, MD, Ph.D., vice president, product development, Lilly. "Obesity is a chronic disease that requires effective treatment options, and Lilly is working relentlessly to support people with obesity and modernize how this disease is approached. We're proud to research and develop potentially innovative treatments like tirzepatide, which helped nearly two thirds of participants on the highest dose reduce their body weight by at least 20 percent in SURMOUNT-1."
Tirzepatide is a novel investigational once-weekly GIP (glucose-dependent insulinotropic polypeptide) receptor and GLP-1 (glucagon-like peptide-1) receptor agonist, representing a new class of medicines being studied for the treatment of obesity. Tirzepatide is a single peptide that activates the body's receptors for GIP and GLP-1, two natural incretin hormones. Obesity is a chronic, progressive disease caused by disruptions in the mechanisms that control body weight, often leading to an increase in food intake and/or a decrease in energy expenditure. These disruptions are multifactorial and can be related to genetic, developmental, behavioral, environmental and social factors. To learn more, visit Lilly.com/obesity.
Lilly will continue to evaluate the SURMOUNT-1 results, which will be presented at an upcoming medical meeting and submitted to a peer-reviewed journal. Additional studies are ongoing for tirzepatide as a potential treatment for obesity or overweight.
About tirzepatide
Tirzepatide is a once-weekly GIP (glucose-dependent insulinotropic polypeptide) receptor and GLP-1 (glucagon-like peptide-1) receptor agonist that integrates the actions of both incretins into a single novel molecule. GIP is a hormone that may complement the effects of GLP-1 receptor agonists. In preclinical models, GIP has been shown to decrease food intake and increase energy expenditure therefore resulting in weight reductions, and when combined with GLP-1 receptor agonism, may result in greater effects on markers of metabolic dysregulation such as body weight, glucose and lipids. Tirzepatide is in phase 3 development for adults with obesity or overweight with weight-related comorbidity and is currently under regulatory review as a treatment for adults with type 2 diabetes. It is also being studied as a potential treatment for non-alcoholic steatohepatitis (NASH) and heart failure with preserved ejection fraction (HFpEF). Studies of tirzepatide in obstructive sleep apnea (OSA) and in morbidity/mortality in obesity are planned as well.
About SURMOUNT-1 and the SURMOUNT clinical trial program
SURMOUNT-1 (NCT04184622) is a multi-center, randomized, double-blind, parallel, placebo-controlled trial comparing the efficacy and safety of tirzepatide 5 mg, 10 mg and 15 mg to placebo as an adjunct to a reduced-calorie diet and increased physical activity in adults without type 2 diabetes who have obesity, or overweight with at least one of the following comorbidities: hypertension, dyslipidemia, obstructive sleep apnea or cardiovascular disease. The trial randomized 2,539 participants across the U.S., Argentina, Brazil, China, India, Japan, Mexico, Russia and Taiwan in a 1:1:1:1 ratio to receive either tirzepatide 5 mg, 10 mg or 15 mg or placebo. The co-primary objectives of the study were to demonstrate that tirzepatide 10 mg and/or 15 mg is superior in percentage of body weight reductions from baseline and percentage of participants achieving ≥5% body weight reduction at 72 weeks compared to placebo. Participants who had pre-diabetes at study commencement will remain enrolled in SURMOUNT-1 for an additional 104 weeks of treatment following the initial 72-week completion date to evaluate the impact on body weight and potential differences in progression to type 2 diabetes at three years of treatment with tirzepatide compared to placebo.
All participants in the tirzepatide treatment arms started the study at a dose of tirzepatide 2.5 mg once-weekly and then increased the dose in a step-wise approach at four-week intervals to their final randomized maintenance dose of 5 mg (via a 2.5 mg step), 10 mg (via steps at 2.5 mg, 5 mg and 7.5 mg) or 15 mg (via steps at 2.5 mg, 5 mg, 7.5 mg, 10 mg and 12.5 mg).
The SURMOUNT phase 3 global clinical development program for tirzepatide began in late 2019 and has enrolled more than 5,000 people with obesity or overweight across six clinical trials, four of which are global studies. Results from SURMOUNT-2, -3, and -4 are anticipated in 2023.
To learn more, visit Lilly.com and Lilly.com/newsroom or follow us on Facebook, Instagram, Twitter and LinkedIn. P-LLY
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SOURCE Eli Lilly and Company
Lilly's tirzepatide shows weight loss benefit in adults without diabetes
Apr. 28, 2022 9:28 AM ET Eli Lilly and Company (LLY)NVO
By: Jonathan Block, SA News Editor4 Comments
- Eli Lilly's (NYSE:LLY) tirzepatide has demonstrated a significant weight loss benefit in individuals without diabetes in a late-stage trial.
- https://seekingalpha.com/news/3828625-eli-lilly-tirzepatide-shows-weight-loss-benefit-in-adults-without-diabetes
- https://seekingalpha.com/symbol/LLY
- https://www.lilly.com/
biotecMAX™ Feb/Mar/APR 2022 Insight
ENHERTU® (fam-trastuzumab deruxtecan-nxki)
Enhertu granted Breakthrough Therapy Designation in the US for patients with HER2-low metastatic breast cancer
PUBLISHED27 April 2022
27 April 2022 07:00 BST
Based on DESTINY-Breast04 results where AstraZeneca and Daiichi Sankyo’s Enhertu demonstrated a significant improvement in both progression-free survival and overall survival
Enhertu has now been granted five Breakthrough Therapy Designations, including three in breast cancer and one in both lung and gastric cancers
AstraZeneca and Daiichi Sankyo’s Enhertu (trastuzumab deruxtecan) has been granted Breakthrough Therapy Designation (BTD) in the US for the treatment of adult patients with unresectable or metastatic HER2-low (IHC 1+ or IHC 2+/ISH-negative) breast cancer who have received a prior systemic therapy in the metastatic setting or developed disease recurrence during or within six months of completing adjuvant chemotherapy. Patients with hormone receptor (HR) positive breast cancer should additionally have received or be ineligible for endocrine therapy.
Enhertu is a specifically engineered HER2-directed antibody drug conjugate (ADC) being jointly developed and commercialised by AstraZeneca and Daiichi Sankyo.
The Food and Drug Administration’s (FDA) BTD is designed to accelerate the development and regulatory review of potential new medicines that are intended to treat a serious condition and address a significant unmet medical need. The new medicine needs to have shown encouraging preliminary clinical results that demonstrate substantial improvement on a clinically significant endpoint over available medicines.
Up to half of all patients with breast cancer have tumours with a HER2 immunohistochemistry (IHC) score of 1+, or 2+ in combination with a negative in-situ hybridisation (ISH) test, a level of HER2 expression not currently eligible for HER2-targeted therapy.1-4 Low HER2 expression occurs in both HR-positive and HR-negative disease.5
HER2 testing is routinely used to determine appropriate treatment options for patients with metastatic breast cancer. Targeting the lower range of expression in the HER2 spectrum may offer another approach to delay disease progression and extend survival in patients with metastatic breast cancer.6 Currently chemotherapy remains the only treatment option for patients with HR-positive tumours following progression on endocrine (hormone) therapy.7 Few targeted options are available for those who are HR-negative.8
Susan Galbraith, Executive Vice President, Oncology R&D, AstraZeneca said: “Today’s news is a significant validation of the potential we see for the historic DESTINY-Breast04 trial to enable a paradigm shift in how breast cancer is classified by targeting the full spectrum of HER2 expression. Enhertu continues to show transformative potential, and this milestone represents an important advance for patients with HER2-low metastatic breast cancer who are in urgent need of new treatment options and better outcomes.”
Ken Takeshita, Global Head, R&D, Daiichi Sankyo said: “Historically, only patients with HER2-positive metastatic breast cancer were shown to benefit from HER2-directed therapy. DESTINY-Breast04, in which Enhertu showed a clinically meaningful survival benefit in patients with HER2-low metastatic breast cancer, is the first trial to demonstrate that selecting patients for treatment based on low expression of HER2 has the potential to change the diagnostic and treatment paradigms for these patients. This Breakthrough Therapy Designation acknowledges the potential of Enhertu to fulfil an unmet medical need and we look forward to working closely with the FDA to bring the first HER2-directed therapy to patients with metastatic breast cancer whose tumours have lower levels of HER2 expression.”
The FDA granted the BTD based on data from the pivotal DESTINY-Breast04 Phase III trial which reported positive high-level results in February 2022. In the trial, Enhertu demonstrated a statistically significant and clinically meaningful improvement in both progression-free survival (PFS) and overall survival (OS) in patients with HER2-low unresectable and/or metastatic breast cancer in all randomised patients with HR-positive and HR-negative disease versus physician’s choice of chemotherapy, which is the current standard of care. The safety profile of Enhertu was consistent with previous clinical trials with no new safety concerns identified. The data will be presented at an upcoming medical meeting.
This is the third BTD for Enhertu in breast cancer. Enhertu previously received BTD’s for the treatment of second-line HER2-positive metastatic breast cancer in 2021 and later-line HER2-positive metastatic breast cancer in 2017. Two additional BTD’s for Enhertu were granted in 2020 for HER2-mutant metastatic non-small cell lung cancer (NSCLC) and HER2-positive metastatic gastric cancer.
DESTINY-Breast04
DESTINY-Breast04 is a global, randomised, open-label, registrational Phase III trial evaluating the efficacy and safety of Enhertu (5.4mg/kg) versus physician’s choice of chemotherapy (capecitabine, eribulin, gemcitabine, paclitaxel or nab-paclitaxel) in patients with HR-positive (n=480) or HR-negative (n=60) HER2-low unresectable and/or metastatic breast cancer previously treated with one or two prior lines of chemotherapy. Patients were randomised 2:1 to receive either Enhertu or chemotherapy.
The primary endpoint of DESTINY-Breast04 is PFS in patients with HR-positive disease based on blinded independent central review (BICR). Key secondary endpoints include PFS based on BICR in all randomised patients (HR-positive and HR-negative disease), OS in patients with HR-positive disease and OS in all randomised patients (HR-positive and HR-negative disease). Other secondary endpoints include PFS based on BICR and investigator assessment, duration of response based on BICR and safety.
DESTINY-Breast04 enrolled approximately 540 patients at multiple sites in Asia, Europe and North America. For more information about the trial, visit ClinicalTrials.gov.
Enhertu
Enhertu is a HER2-directed ADC. Designed using Daiichi Sankyo’s proprietary DXd ADC technology, Enhertu is the lead ADC in the oncology portfolio of Daiichi Sankyo and the most advanced programme in AstraZeneca’s ADC scientific platform. Enhertu consists of a HER2 monoclonal antibody attached to a topoisomerase I inhibitor payload, an exatecan derivative, via a stable tetrapeptide-based cleavable linker.
Enhertu (5.4mg/kg) is approved in more than 40 countries for the treatment of adult patients with unresectable or metastatic HER2-positive breast cancer who have received two or more prior anti-HER2-based regimens based on the results from the DESTINY-Breast01 trial.
Enhertu (6.4mg/kg) is approved in several countries for the treatment of adult patients with locally advanced or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma who have received a prior trastuzumab-based regimen based on the results from the DESTINY-Gastric01 trial.
Daiichi Sankyo collaboration
Daiichi Sankyo Company, Limited (TSE: 4568) [referred to as Daiichi Sankyo] and AstraZeneca entered into a global collaboration to jointly develop and commercialise Enhertu (a HER2-directed ADC) in March 2019, and datopotamab deruxtecan (DS-1062; a TROP2-directed ADC) in July 2020, except in Japan where Daiichi Sankyo maintains exclusive rights. Daiichi Sankyo is responsible for manufacturing and supply of Enhertu and datopotamab deruxtecan.
Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca.
AstraZeneca, Daiichi Sankyo's Enhertu gets FDA breakthrough therapy tag for breast cancer subtype
Apr. 27, 2022 6:20 AM ET Daiichi Sankyo Company, Limited (DSNKY), AZN DSKYF By: Ravikash, SA News Editor1 Comment
- The Food and Drug Administration (FDA) granted breakthrough therapy designation to AstraZeneca (NASDAQ:AZN) and Daiichi Sankyo's (OTCPK:DSKYF) (OTCPK:DSNKY) Enhertu to treat certain patients with breast cancer.
- https://seekingalpha.com/news/3827402-astrazeneca-daiichi-sankyos-enhertu-gets-fda-breakthrough-therapy-tag-for-breast-cancer-subtype
- https://seekingalpha.com/symbol/DSKYF
- https://seekingalpha.com/symbol/DSNKY
- https://seekingalpha.com/symbol/AZN
- https://www.enhertu.com/en/
- https://www.astrazeneca.com/
BRUKINSA (zanubrutinib)
BeiGene Announces BRUKINSA (zanubrutinib) Approval in Uruguay in Mantle Cell Lymphoma, Marginal Zone Lymphoma, and Waldenström’s Macroglobulinemia
Apr 28, 2022 7:00 AM
BRUKINSA is now available in four countries in Latin America, following previous approvals in Brazil, Chile, and Ecuador
Under an exclusive distribution agreement, Adium will commercialize BRUKINSA in Latin America
MONTEVIDEO, Uruguay & CAMBRIDGE, Mass & BEIJING--(BUSINESS WIRE)-- BeiGene (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global, science-driven biotechnology company focused on developing innovative and affordable medicines to improve treatment outcomes and access for patients worldwide, today announced that the BTK inhibitor BRUKINSA (zanubrutinib) has been approved in Uruguay for the treatment of adult patients with previously treated mantle cell lymphoma (MCL), relapsed or refractory marginal zone lymphoma (MZL), and Waldenström’s macroglobulinemia (WM). BeiGene and Adium entered into an exclusive distribution agreement for Adium to commercialize BRUKINSA in Latin America.
“Tolerability of treatments for B-cell malignancies is an important consideration with BTK inhibition, and BRUKINSA was designed with that in mind to maximize BTK occupancy and minimize off-target binding. Today, we have a new treatment option for patients with MCL, MZL and WM in Uruguay, providing hope for improved treatment outcomes,” said Dr. Karina Cicinelli, Corporate Medical Director at Adium.
“We are proud of the progress BeiGene has made in Latin America over the past year, with this approval in three indications in Uruguay following recent launches in Brazil, Chile and Ecuador. With Adium’s established commercial presence in Latin America, we hope patients with MCL, MZL, and WM will soon have access to this important treatment option,” commented Eduardo Molinari, Senior Director of New Market Development in Latin America at BeiGene.
About BRUKINSA
BRUKINSA is a small molecule inhibitor of Bruton’s tyrosine kinase (BTK) discovered by BeiGene scientists that is currently being evaluated globally in a broad clinical program as a monotherapy and in combination with other therapies to treat various B-cell malignancies. Because new BTK is continuously synthesized, BRUKINSA was specifically designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity. With differentiated pharmacokinetics compared to other approved BTK inhibitors, BRUKINSA has been demonstrated to inhibit the proliferation of malignant B cells within a number of disease relevant tissues.
BRUKINSA has previously been approved for three indications in the United States: for the treatment of mantle cell lymphoma (MCL) in adult patients who have received at least one prior therapy (Nov. 2019)*; for the treatment of adult patients with Waldenström’s macroglobulinemia (WM) (Aug. 2021); and for the treatment of adult patients with relapsed or refractory marginal zone lymphoma (MZL) who have received at least one anti-CD20-based regimen (Sept. 2021)*.
BRUKINSA is supported by a broad clinical program which includes more than 3,900 subjects in 35 trials across 28 markets. To date, BRUKINSA has received more than 20 approvals covering more than 40 countries and regions, including the United States, China, the EU and Great Britain, Canada, Australia and additional international markets. Currently, more than 40 additional regulatory submissions are in review around the world.
* This indication was approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
To learn more about BeiGene, please visit www.beigene.com and follow us on Twitter at @BeiGeneGlobal.
Please see full U.S. Prescribing Information at www.beigene.com/PDF/BRUKINSAUSPI.pdf and Patient Information at www.beigene.com/PDF/BRUKINSAUSPPI.pdf.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220427006162/en/
Source: BeiGene
BeiGene Brukinsa gets approval in Uruguay to treat certain blood cancers
Apr. 28, 2022 8:51 AM ET
By: Ravikash, SA News Editor
- BeiGene (NASDAQ:BGNE) said its medicine Brukinsa was approved in Uruguay to treat adult patients with previously treated mantle cell lymphoma (MCL), relapsed or refractory marginal zone lymphoma (MZL), and Waldenström's macroglobulinemia (WM).
- https://seekingalpha.com/news/3828582-beigene-brukinsa-gets-approval-in-uruguay-to-treat-certain-blood-cancers
- https://seekingalpha.com/symbol/BGNE
- https://www.brukinsa.com/
- https://www.beigene.com/
Niraparib and ZYTIGA® (abiraterone acetate)
Janssen Submits Marketing Authorisation Application to EMA Seeking Approval of Niraparib and Abiraterone Acetate Dual Action Tablet Plus Prednisone for the Treatment of Patients with HRR Gene-Mutated Metastatic Castration Resistant Prostate Cancer
The submission to the European Medicines Agency is based on results from the Phase 3 MAGNITUDE study evaluating niraparib in combination with abiraterone acetate plus prednisonefor the treatment of patients with mCRPC who are positive for HRR gene alterations.1
April 28, 2022 08:52 AM Eastern Daylight Time
BEERSE, Belgium--(BUSINESS WIRE)--The Janssen Pharmaceutical Companies of Johnson & Johnson today announced the submission of a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) seeking approval of niraparib in combination with abiraterone acetate, in the form of a dual action tablet (DAT)* plus prednisolone, for the treatment of patients with prostate cancer who have progressed to metastatic castration-resistant prostate cancer (mCRPC) and are positive for homologous recombination repair (HRR)+ gene alterations. When approved by the European Commission, niraparib in combination with AAP will be the first dual action tablet formulation in the European Union specifically targeting HRR gene alterations in mCRPC.
The combination of niraparib, a PARP (poly adenosine diphosphate-ribose polymerase) inhibitor, and abiraterone acetate, a CYP17 inhibitor, targets two oncogenic drivers in patients with mCRPC, AR-axis and HRR gene alterations. The DAT formulation is also intended to be more convenient for patients, and thus aims to improve treatment compliance. Prostate cancer is one of the most common cancers in Europe with approximately 473,000 patients diagnosed in 2020.2 Up to approximately 30% of patients with mCRPC have HRR gene alterations which are associated with a worse prognosis compared to patients without HRR gene alterations.1
“People with prostate cancer harbouring BRCA alterations face a more aggressive form of disease with worse outcomes and faster progression, sadly leading to a shorter life expectancy,” commented Professor Gerhardt Attard=, Primary Study Investigator and Clinician Scientist and Team Leader at University College London Cancer Institute. “This submission is an important step towards improving the outcomes for people with metastatic prostate cancer harbouring BRCA alterations using a targeted therapy that significantly delays the time to their cancer progressing.”
The EU MAA is supported by data from the MAGNITUDE study (NCT03748641), a Phase 3, randomised, double-blind, placebo-controlled, multicentre study evaluating the safety and efficacy of niraparib combined with abiraterone acetate plus prednisone (AAP) in patients with mCRPC. The study showed that at the final analysis for radiographic progression-free survival (rPFS), the treatment combination of niraparib and AAP demonstrated a statistically significant improvement in patients with HRR gene alterations as compared to placebo and AAP.1 First results from the study were presented at the American Society of Clinical Oncology – Genitourinary Cancers Symposium (ASCO GU 2022) Annual Meeting (Abstract #12). The study continues to collect data on the secondary endpoints, which include time-to-initiation of cytotoxic chemotherapy, time to symptomatic progression and overall survival.1
“The data supporting this submission demonstrate the benefit of niraparib in combination with AAP in patients with specific gene alterations and reinforce the importance of biomarker testing in helping to provide an individualised treatment for these patients,” said Kiran Patel, M.D., Vice President, Clinical Development, Solid Tumors, Janssen Research & Development, LLC. “We are committed to advancing targeted therapeutic options for patients with prostate cancer as we build upon our deep understanding of the disease, with a focus on improving outcomes for patients.”
“The submission of niraparib in combination with AAP to the European Medicines Agency marks an important milestone in addressing specific genetic alterations in prostate cancer,” said Mathai Mammen, M.D., Ph.D., Executive Vice President, Pharmaceuticals, R&D, Johnson & Johnson. “We are determined to transform this complex disease through innovation, science and ingenuity.”
###
About Niraparib
Niraparib is an orally administered, selective poly-ADP ribose polymerase (PARP) inhibitor, that is currently being studied by Janssen for the treatment of patients with prostate cancer.1 Additional ongoing studies include the Phase 3 AMPLITUDE study evaluating the combination of niraparib and AAP in a biomarker-selected patient population with metastatic hormone-sensitive prostate cancer (mHSPC).3
In April 2016, Janssen Biotech, Inc. entered a worldwide (except Japan) collaboration and license agreement with TESARO, Inc. (acquired by GSK in 2018), for exclusive rights to niraparib in prostate cancer. In the European Union, niraparib is indicated for the maintenance treatment of adult patients with advanced epithelial ovarian, fallopian tube or primary peritoneal cancer who are in a complete or partial response to first-line platinum-based chemotherapy; for the maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy (Zejula SmPC 2021). Niraparib is currently marketed by GSK as ZEJULA®.4
About abiraterone acetate
Abiraterone acetate is an orally-administered androgen biosynthesis inhibitor. In the European Union, abiraterone acetate is indicated with prednisone or prednisolone for the treatment of newly diagnosed high risk mHSPC in adult men in combination with ADT; the treatment of mCRPC in adult men who are asymptomatic or mildly symptomatic after failure of ADT in whom chemotherapy is not yet clinically indicated; and the treatment of mCRPC in adult men whose disease has progressed on or after a docetaxel based chemotherapy regimen (ZYTIGA SmPC 2020).5
Abiraterone acetate is currently marketed by Janssen Janssen-Cilag International NV as ZYTIGA®.5
About MAGNITUDE
MAGNITUDE (NCT03748641) is a Phase 3 randomised, double-blind, placebo-controlled, multicentre clinical study evaluating the safety and efficacy of the combination of niraparib and AAP for patients with mCRPC, with or without certain HRR gene alterations. The study includes two cohorts in which patients were randomised to receive either niraparib and AAP or placebo and AAP cohorts: one cohort of patients with predefined HRR gene alterations (including ATM, BRCA1, BRCA2, BRIP1, CDK12, CHEK2, FANCA, HDAC2, PALB2 alterations) and one cohort of patients without HRR gene alterations. In a third, open-label cohort, all patients received the dual action tablet formulation of niraparib and AAP.1
The primary endpoint of the MAGNITUDE trial is rPFS. Secondary endpoints include time-to-initiation of cytotoxic chemotherapy, time to symptomatic progression and overall survival.1
Learn more at www.janssen.com/emea/. Follow us at www.twitter.com/JanssenEMEA for our latest news. Janssen Research & Development, LLC; Janssen-Cilag, S.A.; and Janssen Biotech, Inc. are part of the Janssen Pharmaceutical Companies of Johnson & Johnson.
J&J seeks approval of Zytiga/Zejula combination for prostate cancer to EMA
Apr. 28, 2022 10:13 AM ET
By: Jonathan Block, SA News Editor
- Johnson and Johnson's (NYSE:JNJ) Janssen unit has submitted a marketing authorization application to the European Medicines Agency (EMA) seeking approval of a combination of Zytiga (abiraterone acetate) and Zejula (niraparib) for prostate cancer.
- https://seekingalpha.com/news/3828664-johnson-johnson-janssen-seeks-approval-of-zytiga-zejula-combination-for-prostate-cancer-to-ema
- https://seekingalpha.com/symbol/JNJ
- https://www.zytiga.com/
- https://zejula.com/
- https://www.janssen.com/
- https://www.jnj.com/
Tremelimumab in combination with Imfinzi (durvalumab)
Tremelimumab accepted under Priority Review in the US for patients with unresectable liver cancer in combination with Imfinzi
PUBLISHED25 April 2022
25 April 2022 07:00 BST
STRIDE regimen of a single priming dose of tremelimumab added to Imfinzi is the first dual immune checkpoint blockade regimen to improve overall survival in a Phase III trial in this setting
AstraZeneca’s Biologics License Application (BLA) for tremelimumab has been accepted for Priority Review in the US, supporting the indication of a single priming dose of the anti-CTLA4 antibody added to Imfinzi (durvalumab) for the treatment of patients with unresectable hepatocellular carcinoma (HCC). A supplemental BLA (sBLA) has also been submitted for Imfinzi in this indication. This novel dose and schedule of the combination is called the STRIDE regimen (Single Tremelimumab Regular Interval Durvalumab).
The Prescription Drug User Fee Act date, the Food and Drug Administration (FDA) action date for their regulatory decision, is during the fourth quarter of 2022 following the use of a priority review voucher.
Liver cancer, of which HCC is the most common type, is the third-leading cause of cancer death and the sixth most commonly diagnosed cancer worldwide.1,2 Approximately 26,000 people in the US present with advanced, unresectable HCC each year.3
Susan Galbraith, Executive Vice President, Oncology R&D, AstraZeneca, said: “The HIMALAYA Phase III trial showed an unprecedented three-year overall survival in this setting with a single priming dose of tremelimumab added to Imfinzi, highlighting the potential for this regimen to improve longer-term survival outcomes. Patients with advanced liver cancer are in great need of new treatment options, and we are working closely with the FDA to bring this novel approach to patients in the US as soon as possible.”
The BLA for tremelimumab and sBLA for Imfinzi are based on final results from the HIMALAYA Phase III trial presented at the 2022 American Society of Clinical Oncology Gastrointestinal Cancers Symposium.
In this trial, patients treated with the STRIDE regimen experienced a 22% reduction in the risk of death versus sorafenib (based on a hazard ratio [HR] of 0.78, 96.02% confidence interval [CI] 0.65-0.93; p=0.0035).4 Nearly one in three (31%) patients were still alive at three years versus one in five (20%) for sorafenib.4
The safety profiles of the STRIDE regimen and for Imfinzi alone were consistent with the known profiles of each medicine, and no new safety signals were identified.
Imfinzi and tremelimumab were granted Orphan Drug Designation in the US for the treatment of HCC in January 2020.
As part of its extensive clinical development programme in gastrointestinal (GI) cancers, AstraZeneca is further assessing Imfinzi across multiple liver cancer settings, including locoregional HCC (EMERALD-1, EMERALD-3) and adjuvant HCC
HIMALAYA
HIMALAYA was a randomised, open-label, multicentre, global Phase III trial of Imfinzi monotherapy and the STRIDE regimen, comprising a single priming dose of tremelimumab 300mg added to Imfinzi 1500mg followed by Imfinzi every four weeks versus sorafenib, a standard-of-care multi-kinase inhibitor.
The trial included a total of 1,324 patients with unresectable, advanced HCC who had not been treated with prior systemic therapy and were not eligible for locoregional therapy (treatment localised to the liver and surrounding tissue).
The trial was conducted in 181 centres across 16 countries, including in the US, Canada, Europe, South America and Asia. The primary endpoint is overall survival (OS) for STRIDE versus sorafenib and key secondary endpoints included OS for Imfinzi versus sorafenib, objective response rate and progression-free survival (PFS) for STRIDE and for Imfinzi alone.
Imfinzi
Imfinzi (durvalumab) is a human monoclonal antibody that binds to the PD-L1 protein and blocks the interaction of PD-L1 with the PD-1 and CD80 proteins, countering the tumour’s immune-evading tactics and releasing the inhibition of immune responses.
Imfinzi is the only approved immunotherapy in the curative-intent setting of unresectable, Stage III non-small cell lung cancer (NSCLC) in patients whose disease has not progressed after chemoradiotherapy, and is the global standard of care in this setting based on the PACIFIC Phase III trial.
Imfinzi is also approved in the US, EU, Japan, China and many other countries around the world for the treatment of extensive-stage small cell lung cancer (ES-SCLC) based on the CASPIAN Phase III trial. In 2021, updated results from the CASPIAN trial showed Imfinzi plus chemotherapy tripled patient survival at three years versus chemotherapy alone.
In the past year, Imfinzi has demonstrated clinical benefit in multiple additional cancer settings with positive Phase III trials in advanced biliary tract cancer (TOPAZ-1), unresectable advanced liver cancer (HIMALAYA) and metastatic NSCLC (POSEIDON).
As part of a broad development programme, Imfinzi is being tested as a single treatment and in combinations with other anti-cancer treatments for patients with small cell lung cancer, NSCLC, bladder cancer, several gastrointestinal cancers, ovarian cancer, endometrial cancer, and other solid tumours.
Tremelimumab
Tremelimumab is a human monoclonal antibody and potential new medicine that targets the activity of cytotoxic T-lymphocyte-associated protein 4 (CTLA-4). Tremelimumab blocks the activity of CTLA-4, contributing to T-cell activation, priming the immune response to cancer and fostering cancer cell death.
Beyond HIMALAYA, tremelimumab is being tested in combination with Imfinzi across multiple tumour types including locoregional HCC (EMERALD-3), SCLC (ADRIATIC) and bladder cancer (VOLGA and NILE).
Tremelimumab is also under review by global regulatory authorities in combination with Imfinzi and chemotherapy in 1st-line metastatic NSCLC based on the results of the POSEIDON Phase III trial, which showed the addition of a short course of tremelimumab to Imfinzi plus chemotherapy improved both overall and progression-free survival compared to chemotherapy alone.
Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca.
AstraZeneca gets FDA priority review for new dosing regimen of tremelimumab/Imfinzi in liver cancer
Apr. 25, 2022 5:42 AM ET AstraZeneca PLC (AZN)BAYZF, BAYRY
By: Ravikash, SA News Editor1 Comment
The Food and Drug Administration (FDA) granted priority review to AstraZeneca's (NASDAQ:AZN) application for a single priming dose of tremelimumab added to Imfinzi (durvalumab) to treat patients with unresectable hepatocellular carcinoma, a type of liver cancer.
https://seekingalpha.com/symbol/AZN
Ultomiris (ravulizumab-cwvz)
28 April 2022 07:00 BST
First and only long-acting C5 complement inhibitor to demonstrate clinical improvement in patients with generalised myasthenia gravis
Ultomiris showed early effect and lasting improvement in activities of daily living and has potential to reduce treatment burden with dosing every 8 weeks
Ultomiris (ravulizumab-cwvz) has been approved in the US for the treatment of adult patients with generalised myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive, which represents 80% of people living with the disease.1-5
The approval by the Food and Drug Administration (FDA) was based on positive results from the CHAMPION-MG Phase III trial, in which Ultomiris was superior to placebo in the primary endpoint of change from baseline in the Myasthenia Gravis-Activities of Daily Living Profile (MG-ADL) total score at Week 26, a patient-reported scale that assesses patients’ abilities to perform daily activities.1
This FDA action marks the first and only approval for a long-acting C5 complement inhibitor for the treatment of gMG.
gMG is a rare, debilitating, chronic, autoimmune neuromuscular disease that leads to a loss of muscle function and severe weakness.6 The diagnosed prevalence of gMG in the US is estimated at approximately 90,000.7
Professor James F. Howard, Jr, MD, Department of Neurology at The University of North Carolina School of Medicine and lead primary investigator in the CHAMPION-MG trial said: “Despite recent advances, managing gMG is complex. Earlier intervention can preserve function and quality of life. This approval offers patients, including those with milder symptoms, a long-acting C5 inhibitor with early onset and reliable efficacy.”
Samantha Masterson, Chief Executive Officer, Myasthenia Gravis Foundation of America (MGFA), said: “gMG takes a physical and emotional toll on those living with the disease. We are grateful for continued innovation and research into new treatment and dosing options to meet the needs of more patients and reduce the treatment burden. With the approval of Ultomiris, we’re excited that MG patients now have another option to consider as part of their personalised treatment strategies that may offer more convenience and improve muscle weakness.”
Marc Dunoyer, Chief Executive Officer, Alexion, said: “Since bringing forward the first complement inhibitor, we’ve continued to listen to the community and focused innovation on the needs of gMG patients. We’re proud to deliver on this commitment with today’s approval. Ultomiris, the only long-acting C5 inhibitor, will benefit a broader range of patients, including those with milder symptoms. As presented at the 2022 American Academy of Neurology Annual Meeting, Ultomiris has demonstrated clinical benefit through 60 weeks, with treatment every eight weeks, compared to Soliris every two weeks.”
In the trial, the safety profile of Ultomiris was comparable to placebo and consistent with that observed in Phase III trials of Ultomiris in paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS). The most common adverse reactions in patients receiving Ultomiris were upper respiratory tract infection and diarrhoea.1
Results from the CHAMPION-MG trial were recently published online in NEJM Evidence and presented at the 2022 American Academy of Neurology Annual Meeting in April.
Regulatory submissions for Ultomiris for the treatment of gMG are currently under review with multiple health authorities, including in the European Union (EU) and Japan.
CHAMPION-MG
The global Phase III randomised, double-blind, placebo-controlled, multicentre 26-week trial evaluated the safety and efficacy of Ultomiris in adults with gMG. The trial enrolled 175 patients across North America, Europe, Asia-Pacific and Japan. Participants were required to have a confirmed myasthenia gravis diagnosis at least six months prior to the screening visit with a positive serologic test for anti-AChR antibodies, MG-ADL total score of at least 6 at trial entry and Myasthenia Gravis Foundation of America Clinical Classification Class II to IV at screening. Patients could stay on stable standard of care medicines, with a few exceptions, for the duration of the randomised control period.13
Patients were randomised 1:1 to receive Ultomiris or placebo for a total of 26 weeks. Patients received a single weight-based loading dose on Day 1, followed by regular weight-based maintenance dosing beginning on Day 15, every eight weeks. The primary endpoint of change from baseline in the MG-ADL total score at Week 26 was assessed along with multiple secondary endpoints evaluating improvement in disease-related and quality-of-life measures.
Patients who completed the randomised control period were eligible to continue into an open-label extension period evaluating the safety and efficacy of Ultomiris, which is ongoing.
Ultomiris
Ultomiris (ravulizumab-cwvz), the first and only long-acting C5 complement inhibitor, offers immediate, complete and sustained complement inhibition. The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. When activated in an uncontrolled manner, the complement cascade over-responds, leading the body to attack its own healthy cells. Ultomiris is administered intravenously every eight weeks in adult patients, following a loading dose.
Ultomiris is approved in the US for the treatment of certain adults with gMG.
Ultomiris is also approved in the US, EU and Japan for the treatment of certain adults and children with PNH.
Additionally, Ultomiris is approved in the US, EU and Japan for certain adults and children with aHUS to inhibit complement-mediated thrombotic microangiopathy.
As part of a broad development programme, Ultomiris is being assessed for the treatment of additional haematology and neurology indications.
Alexion
Alexion, AstraZeneca Rare Disease, is the group within AstraZeneca focused on rare diseases, created following the 2021 acquisition of Alexion Pharmaceuticals, Inc. As a leader in rare diseases for nearly 30 years, Alexion is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialisation of life-changing medicines. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on haematology, nephrology, neurology, metabolic disorders, cardiology and ophthalmology. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries.
Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca.
AstraZeneca's Ultomiris wins FDA approval for rare neuromuscular disorder
Apr. 28, 2022 6:59 AM ET
By: Ravikash, SA News Editor
The U.S. Food and Drug Administration approved AstraZeneca's (NASDAQ:AZN) Ultomiris to treat adult patients with generalised myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive.
- https://seekingalpha.com/news/3828408-astrazenecas-ultomiris-wins-fda-approval-for-rare-neuromuscular-disorder
- https://seekingalpha.com/symbol/AZN
- https://ultomiris.com/
- https://www.astrazeneca.com/
REPATHA® (EVOLOCUMAB)
AMGEN ANNOUNCES RESULTS FROM TWO OPEN LABEL EXTENSION STUDIES OF REPATHA® (EVOLOCUMAB)
Studies Showed Sustained Reduction in LDL-C With no new Safety Findings
The Combined Studies Evaluated Safety and Tolerability of Repatha in More Than 6,600 Patients for Over Five Years After Completing the Phase 3 FOURIER Trial
Repatha is the Longest Studied PCSK9i
THOUSAND OAKS, Calif., April 27, 2022 /PRNewswire/ -- Amgen (NASDAQ:AMGN) today announced top-line results from two Repatha® (evolocumab) open label extension (OLE) studies to the Phase 3 FOURIER cardiovascular outcomes trial. The studies were designed to assess the long-term safety and tolerability of Repatha over five years in adults with clinically evident atherosclerotic cardiovascular disease.
The FOURIER-OLE (Further cardiovascular OUtcomes Research with PCSK9 Inhibition in Subjects with Elevated – Risk-Open Label Extension) studies were composed of study 20130295 (NCT02867813) with 5,035 patients enrolled in Eastern Europe and the United States and study 20160250 (NCT03080935) with 1,600 patients enrolled in Western Europe. Both studies showed that Repatha, administered at 140 mg every two weeks or 420 mg monthly, was safe and well-tolerated. In the OLE studies, patients received Repatha for approximately 5 years, with some patients receiving Repatha for up to 8 1/2 years in aggregate across the FOURIER and OLE studies. No new long-term safety findings were observed.
In addition, medically significant and sustained reduction in low-density lipoprotein cholesterol (LDL-C) levels were observed, with more than 85 percent of patients achieving an LDL-C level of <40 mg/dL during the OLE period.
"The combined results from these studies reinforce the well-established safety profile of Repatha with long-term use in lowering LDL-C," said David M. Reese, M.D., executive vice president of Research and Development at Amgen. "As the PCSK9i leader, with more than one million patients worldwide who have received Repatha, we are extremely encouraged by the sustained benefit of this effective medicine for patients with cardiovascular disease who still struggle to get their LDL-C level below recommended goals."
Other study measures included exploratory analyses of non-high-density lipoprotein cholesterol, apolipoprotein B, total cholesterol, Lipoprotein(a), triglycerides, high-density lipoprotein cholesterol, very-low-density lipoprotein cholesterol, and apolipoprotein A-1 levels, as well as cardiovascular events of interest.
Detailed study results will be shared with regulatory authorities and submitted for presentation at an upcoming medical congress later this year. Prolonged LDL-C reduction with Repatha is also being studied in the ongoing VESALIUS-CV (NCT03872401) outcomes trial.
Repatha® Cardiovascular Open-Label Extension (FOURIER-OLE) Study Design
FOURIER (20110118) was a randomized placebo-controlled study of evolocumab, in patients with clinically evident atherosclerotic CVD on stable effective statin therapy. FOURIER-OLE (Further cardiovascular OUtcomes Research with PCSK9 Inhibition in Subjects with Elevated – Risk-Open Label Extension) were multicenter, open-label extension (OLE) studies designed to assess the extended long-term safety of evolocumab in subjects who completed the FOURIER study (20110118). The FOURIER-OLE is composed of studies 20130295 and 20160250, which enrolled 5,035 and 1,600 subjects who completed FOURIER study (20110118) to receive open-label evolocumab and were followed up for a median of 5 and 4.6 years, respectively.
PROFICIO Program
FOURIER is part of Amgen's PROFICIO (Program to Reduce LDL-C and cardiovascular Outcomes Following Inhibition of PCSK9 In different pOpulations) program of clinical studies investigating the impact of Repatha® on LDL-C and CVD across multiple populations at high CV risk, including those managed by statins, statin-intolerant patients, those with genetic disorders and patients with atherosclerosis. To date, the PROFICIO program consists of 36 trials including more than 38,000 patients worldwide.
For more information, visit www.amgen.com and follow us on www.twitter.com/amgen.
About Repatha® (evolocumab)
Repatha® is a human monoclonal antibody that inhibits proprotein convertase subtilisin/kexin type 9 (PCSK9). Repatha® binds to PCSK9 and inhibits circulating PCSK9 from binding to the low-density lipoprotein (LDL) receptor (LDLR), preventing PCSK9-mediated LDLR degradation and permitting LDLR to recycle back to the liver cell surface. By inhibiting the binding of PCSK9 to LDLR, Repatha® increases the number of LDLRs available to clear LDL from the blood, thereby lowering LDL-C levels.
Repatha® is approved in more than 75 countries, including the U.S., Japan, Canada and in all 28 countries that are members of the European Union. Applications in other countries are pending.
INDICATIONS
Repatha® is indicated:
- In adults with established cardiovascular disease to reduce the risk of myocardial infarction, stroke, and coronary revascularization
- As an adjunct to diet, alone or in combination with other low-density lipoprotein cholesterol (LDLC)-lowering therapies, in adults with primary hyperlipidemia, including heterozygous familial hypercholesterolemia (HeFH), to reduce LDL-C
- As an adjunct to diet and other LDL-C-lowering therapies in pediatric patients aged 10 years and older with HeFH, to reduce LDL-C
- As an adjunct to other LDL-C-lowering therapies in adults and pediatric patients aged 10 years and older with homozygous familial hypercholesterolemia (HoFH), to reduce LDL-C
The safety and effectiveness of Repatha® have not been established in pediatric patients with HeFH or HoFH who are younger than 10 years old or in pediatric patients with other types of hyperlipidemia.
Immunogenicity: Repatha® is a human monoclonal antibody. As with all therapeutic proteins, there is potential for immunogenicity with Repatha®.Please contact Amgen Medinfo at 800-77-AMGEN (800-772-6436) or 844-REPATHA (844-737-2842) regarding Repatha® availability or find more information, including full Prescribing Information, at www.amgen.com and www.Repatha.com.
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SOURCE Amgen
Amgen's Repatha sustains reduction in bad cholesterol as per long term data from 2 studies
Apr. 27, 2022 9:48 AM ET
By: Ravikash, SA News Editor2 Comments
Amgen (NASDAQ:AMGN) said data from two studies showed that its drug Repatha sustained reduction in 'bad' cholesterol.
https://seekingalpha.com/symbol/AMGN
Veklury® (Remdesivir)
April 24, 2022
Several New Studies Presented at ECCMIC 2022 Confirm Veklury® (Remdesivir) Activity in Treating COVID-19
-- Real-World Evidence from More than 850,000 Hospitalized Patients Provides Clinical Insights on the Use of Veklury (Remdesivir) as Stand of Care COVID-19 Treatment --
-- New Post-hoc Analysis of Non-Hospitalized COVID-19 Patients in the Phase 3 PINETREE Study Demonstrates Veklury Treatment Initiated Within 5 Days of Symptoms Reduced Risk for Hospitalization by 90% --
FOSTER CITY, Calif.--(BUSINESS WIRE)-- Gilead Sciences, Inc. (Nasdaq: GILD) today announced findings from two studies, which provide further insights on the use of Veklury® (remdesivir) for the treatment of hospitalized and non-hospitalized patients with COVID-19. The first study is a retrospective observational analysis of the real-world treatment data from the Premier Healthcare Database consisting of 853,219 patients hospitalized with COVID-19 across the United States. This analysis found that more than 50% of hospitalized COVID-19 patients received Veklury, predominantly in combination with other therapies. An oral presentation of this real-world data analysis will be given on April 25 at 11 a.m. Western European Summer Time in Hall P at the European Congress of Clinical Microbiology & Infectious Diseases (ECCMID 2022). A separate study, which is a new post-hoc analysis of data from the Phase 3 PINETREE study, demonstrated that use of Veklury within five days of symptom onset or between 5 to 7 days of symptom onset reduced hospitalizations in patients at high risk for severe COVID-19 disease. This post-hoc analysis was presented as a poster (#L0447) at ECCMID.
The observational analysis of real-world data also found that as the pandemic progressed, initiation of Veklury within two days of hospitalization increased from 41% to 91% between May 2020 and December 2021. During this period, as new variants arose and disease severity fluctuated, median hospital length of stay (LOS) decreased from seven to six days with the greatest benefit in invasive mechanical ventilation/ ECMO patients (15 to 11 days). While ICU use decreased from 34% to 27%, with the greatest benefit in high-flow oxygen/non-invasive ventilation (66% to 52%), overall ICU LOS remained the same. Overall mortality rates remained stable at 16%, with the greatest decline over time in patients on low-flow supplemental oxygen (15% to 12%). These results confirm Veklury’s position as a foundational treatment for hospitalized patients with COVID-19 and signify the need to treat patients early before they become more severely ill with COVID-19.
“In a pandemic, real world analysis has a particularly important role in helping us understand how treatment choices are evolving over time as we work to discover the most effective treatment options for patients. We've long understood that antivirals work optimally for respiratory viruses when they are given as early as possible, without delay. This data confirms that antiviral treatment is now initiated sooner in hospitalizations and that throughout the pandemic, remdesivir has remained the foundation for hospitalized patients with COVID-19,” said Robert L. Gottlieb, MD, PhD, Baylor University Medical Center and Baylor Scott & White Research Institute. “Additionally, as healthcare providers' experience and confidence in COVID-19 therapeutic options has increased, so too has the use of therapeutic combinations, reflecting the incremental incorporation of evidence-based therapies.”
The new post-hoc analysis from a Phase 3 double-blind, placebo-controlled trial (PINETREE) demonstrating that a three-day course of Veklury treatment significantly reduced the risk of hospitalization was also presented at ECCMID. The analysis assessed the variability of treatment effect with Veklury by time of symptom onset and number of baseline risk factors. The study concluded that Veklury reduced hospitalizations in patients at high risk for severe COVID-19 disease when initiated anytime within a 7-day window from symptom onset. As expected with antiviral therapy, the benefit was modestly greater the sooner Veklury was administered. Patients treated with Veklury within five days of symptom onset had a 90% reduced risk for hospitalization. Additionally, patients who received Veklury after five 5 days of symptom onset experienced an 81% reduction in risk of hospitalization. This new analysis builds on the previously presented primary endpoint analysis, in which Veklury demonstrated a statistically significant 87% reduction in risk for the composite primary endpoint of COVID-19 related hospitalization or all-cause death by Day 28 (0.7% [2/279]) compared with placebo (5.3% [15/283]) p=0.008); no deaths occurred in either arm of the study through the primary endpoint.
“The data presented at ECCMID, not only underscore Veklury as the antiviral standard of care for COVID-19 treatment in hospitalized patients, but they also further emphasize that patients can benefit from Veklury when it’s given up to seven days after the onset of symptoms and that the benefit is greater the sooner it is administered,” said Frank Duff, Senior Vice President, Virology Therapeutic Head, Gilead Sciences. “As the pandemic has progressed and the utility of other treatments against new variants has shifted, we are proud that Veklury has remained the foundation for patients hospitalized with COVID-19.”
Gilead presented two additional studies from the company’s COVID-19 clinical and real-world evidence programs at the conference.
- Data from the CARAVAN study evaluated safety, pharmacokinetic, virologic, and clinical outcomes of Veklury treatment in pediatric patients who were 28 days of age and older. The interim analysis of Veklury in pediatric patients hospitalized with COVID-19 with ages ranging from 28 days to less than 18 years demonstrated that Veklury was generally well tolerated, with a high proportion of participants showing clinical improvement and recovery. Overall, no new safety findings for Veklury were noted. In the study, 75% and 85% showed clinical improvement (≥2 point increase on the ordinal scale) at Day 10 and last assessment, respectively, while 60% and 83% were discharged by Day 10 and Day 30, respectively. Overall, 38 patients (72%) experienced AEs, with 11 patients (21%) experiencing serious adverse events (SAEs) that were determined not to be study-drug related, including 3 participant deaths which were consistent with the patients’ underlying medical condition prior to study entry or with COVID-19 disease during hospitalization.
- A real-world evidence analysis evaluated data from 2,310 patients hospitalized with COVID-19 who had previously undergone kidney transplantation. The analysis found that in this patient population, overall mortality was comparable to the general population hospitalized with COVID-19, but markedly increased for those with diminished renal function, comorbidities, and higher oxygen requirements upon admission. Insights from this study help inform clinical decision-making in the context of management of kidney transplant patients and other solid-organ transplant recipients, alike.
About Veklury
Veklury (remdesivir) is a nucleotide analog invented by Gilead, building on more than a decade of the company’s antiviral research. Veklury is a foundation for the treatment of hospitalized patients with COVID-19 and is a recommended treatment for reducing disease progression in non-hospitalized patients at high risk of disease progression. Veklury has an established safety profile and minimal drug interactions in diverse populations. At this time, more than half of patients hospitalized with COVID-19 in the United States are treated with Veklury. It can help reduce disease progression across a spectrum of disease severity and enable patients to recover faster, freeing up limited hospital resources and saving healthcare systems money.
Veklury was approved by the FDA in October 2020, for adults and pediatric patients 12 years of age and older and weighing at least 40 kg for the treatment of COVID-19 requiring hospitalization. In January 2022, the FDA approved a supplemental NDA to expand the indication to non-hospitalized adult and adolescent patients who are at high risk of progression to severe COVID-19, including hospitalization or death. This allows for Veklury to be administered in qualified outpatient settings that can administer daily intravenous (IV) infusions over three consecutive days. Veklury also has an Emergency Use Authorization (EUA) for non-hospitalized pediatric patients weighing at least 3.5 kg who are younger than 12 years of age or weighing less than 40 kg who are at high risk of disease progression, in addition to those with COVID-19 requiring hospitalization. Veklury is contraindicated in patients who are allergic to Veklury or any of its components; please see below for additional Important Safety Information for Veklury.
Veklury continues to demonstrate durable activity against SARS-CoV2 as it evolves. Veklury is a nucleotide analog that directly inhibits viral replication inside of the cell by targeting the SARS-CoV-2 viral RNA polymerase. In vitro laboratory testing in multiple independent studies shows that Veklury continues to demonstrate durable activity against SARS-CoV2 as it evolves, including the Omicron variant and its subvariants BA.1 and BA.2. As new SARS-CoV-2 variants of concern emerge around the world, Gilead continuously evaluates the effectiveness of Veklury against viral variants.
Veklury is approved or authorized for temporary use in approximately 50 countries worldwide. To date, Veklury and generic remdesivir have been made available to more than 11 million patients around the world, including more than 7 million people in 127 middle- and low-income countries through Gilead’s voluntary licensing program. These licenses currently remain royalty-free, reflecting Gilead’s existing commitment to enabling broad patient access to remdesivir.
U.S. Indication for Veklury
Veklury® (remdesivir 100 mg for injection) is indicated for the treatment of COVID-19 in adults and pediatric patients (12 years of age and older and weighing at least 40 kg) with positive results of direct SARS-CoV-2 viral testing, who are:
- Hospitalized, or
- Not hospitalized and have mild-to-moderate COVID-19 and are at high risk for progression to severe COVID-19, including hospitalization or death.
Veklury should only be administered in settings in which health care providers have immediate access to medications to treat a severe infusion or hypersensitivity reaction, such as anaphylaxis, and the ability to activate the emergency medical system (EMS), as necessary. Veklury must be administered by intravenous infusion. Veklury is contraindicated in patients who are allergic to Veklury or any of its components. For more information, please see the U.S. full Prescribing Information available at www.gilead.com.
U.S. full Prescribing Information for Veklury is available at www.gilead.com.
Veklury, Gilead and the Gilead logo are registered trademarks of Gilead Sciences, Inc., or its related companies.
For more information about Gilead, please visit the company’s website at www.gilead.com , follow Gilead on Twitter (@Gilead Sciences) or call Gilead Public Affairs at 1-800-GILEAD-5 or 1-650-574-3000.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220424005046/en/
Source: Gilead Sciences, Inc.
Gilead data shows COVID drug Veklury shows benefit if given early, cuts hospitalization risk
Apr. 25, 2022 6:27 AM ET Gilead Sciences, Inc. (GILD)
By: Ravikash, SA News Editor2 Comments
Gilead Sciences (NASDAQ:GILD) reported data showing activity of its COVID-19 therapy Veklury (remdesivir) based on real-world results from over 850K patients, and a post-hoc analysis of a phase 3 trial.
https://seekingalpha.com/symbol/GILD
April 25, 2022
Veklury® (Remdesivir) is First and Only Approved Treatment for Pediatric Patients Under 12 Years of Age with COVID-19
-- Approval is Supported by Phase 2/3 Data Demonstrating the Safety and Tolerability Profile and Clinical Improvement Outcome in Hospitalized Pediatric Patients Treated with Veklury --
FOSTER CITY, Calif.--(BUSINESS WIRE)-- Gilead Sciences, Inc. (Nasdaq: GILD) today announced that the U.S. Food and Drug Administration (FDA) has approved a supplemental new drug application (sNDA) for Veklury® (remdesivir) for the treatment of pediatric patients who are older than 28 days, weighing at least 3 kg, and are either hospitalized with COVID-19 or have mild-to-moderate COVID-19 and are considered high risk for progression to severe COVID-19, including hospitalization or death. This approval follows the recent sNDA approval for Veklury for the treatment of non-hospitalized adult and adolescent patients who are at high risk of progression to severe COVID-19.
https://seekingalpha.com/symbol/GILD
VRAYLAR® (cariprazine)
Health Canada Approves VRAYLAR® (cariprazine) for the Treatment of Bipolar l Disorder and Schizophrenia in Adults
Apr. 27, 2022 7:00 AM ET AbbVie Inc. (ABBV)
VRAYLAR® is a new atypical antipsychotic medication with partial agonist activity at central dopamine D3 receptors in addition to targeted activity at D2 and serotonin 5-HT1A and 5-HT2A receptors. 1
MONTREAL, April 27, 2022 /CNW Telbec/ - AbbVie (NYSE: ABBV), a global, research and development-based biopharmaceutical company announced today that Health Canada has approved VRAYLAR® (cariprazine) as monotherapy for the acute management of manic, mixed, and depressive episodes associated with bipolar l disorder in adults, as well as the treatment of schizophrenia in adults.1
Bipolar I disorder is a mental illness characterized by extreme mood swings that can impact an individuals' ability to think, behave and function.2 It can consist of three states which includes manic, depressive, and mixed episodes.3 While the causes of bipolar I disorder are unknown, biological factors can play a role and can be difficult to diagnose due to the wide range of symptoms.3
About VRAYLAR® (cariprazine) 1
VRAYLAR® is an oral, once daily atypical antipsychotic approved as monotherapy for the acute management of manic, mixed, and depressive episodes associated with bipolar l disorder in adults, as well as the treatment of schizophrenia in adults.
The mechanism of action of cariprazine in schizophrenia and bipolar I disorder is unknown. However, the therapeutic effect of cariprazine may be mediated through a combination of partial agonist activity at central dopamine D3, D2 and serotonin 5-HT1A receptors and antagonist activity at 5-HT2A receptors. Cariprazine forms two major metabolites, desmethyl cariprazine (DCAR) and didesmethyl cariprazine (DDCAR), that have in vitro receptor binding and functional activity profiles similar to the parent drug.
VRAYLAR® is contraindicated in patients who are hypersensitive to cariprazine or to any ingredient in the formulation, including any non-medicinal ingredient, or component of the container. VRAYLAR® is also contraindicated with concomitant use with strong and moderate CYP3A4 inhibitors / inducers. Due to the slow elimination of cariprazine and its metabolites, treatment with strong and moderate CYP3A4 inhibitors must be initiated at least 2 weeks after VRAYLAR discontinuation.
VRAYLAR is being developed jointly by AbbVie and Gedeon Richter Plc, with AbbVie responsible for commercialization in the U.S., Canada, Japan, Taiwan and certain Latin American countries (including Argentina, Bolivia, Brazil, Chile, Colombia, Ecuador, Mexico, Peru and Venezuela).
Please consult the VRAYLAR® Product Monograph at www.allergan.ca.
For more information about AbbVie, please visit us at www.abbvie.ca. Follow AbbVie Canada on Twitter, on Instagram or find us on LinkedIn.
SOURCE AbbVie Canada
https://seekingalpha.com/symbol/ABBV
VYDURA® (Rimegepant)
Pfizer and Biohaven’s VYDURA® (Rimegepant) Granted First Ever Marketing Authorization by European Commission for Both Acute Treatment of Migraine and Prophylaxis of Episodic Migraine
Wednesday, April 27, 2022 - 06:45am
NEW YORK & NEW HAVEN, Conn.--(BUSINESS WIRE)-- Pfizer Inc. (NYSE: PFE) and Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) today announced that the European Commission (EC) has granted marketing authorization for VYDURA® (rimegepant), a calcitonin gene-related peptide (CGRP) receptor antagonist for both the acute treatment of migraine with or without aura, and prophylaxis of episodic migraine in adults who have at least four migraine attacks per month. VYDURA®, an orally disintegrating tablet, is the first medicine approved for both acute and prophylactic treatment of migraine in the European Union (EU). Migraine is a leading cause of disability worldwide with approximately one in ten people living with the condition in Europe alone. Globally, migraine disproportionately affects women by three to four times compared to men.
“There is a significant unmet need for people in the European Union living with the pain and disability caused by frequent migraines,” said Nick Lagunowich, Global President, Pfizer Internal Medicine. “The comprehensive clinical program has established VYDURA’s efficacy and safety as both an acute and preventive treatment of migraine. Studies in acute migraine demonstrated a rapid and long-lasting relief of migraine headache and other symptoms with a single dose, while the prevention study found a significant reduction in migraine attacks with every other day dosing. We have great confidence in the positive impact VYDURA could have on people living with this debilitating condition in the EU.”
Results from the Phase 3 study published in Lancet demonstrated that a single dose of rimegepant provided superior pain reduction and associated symptoms of migraine at two hours compared to placebo. The prevention study, also published in Lancet, demonstrated that rimegepant taken every other day provided superior reduction in the number of days per month with migraine in Weeks 9 –12 of the 12-week treatment period compared to placebo, that was maintained with continued dosing during the 12-month open-label extension period.
“Today’s approval marks a huge step forward for patients in Europe who are living with migraine. Migraine is often overlooked and undertreated, resulting in substantial disability with suboptimal care for patients,” commented Professor Peter Goadsby, Director of the National Institute for Health and Care Research (NIHR) Clinical Research Facility and Professor of Neurology at King’s College London. “VYDURA’s promising efficacy and favorable benefit-risk profile spark hope for people in need of new migraine treatment options. This approval has the potential to advance the standard of care for migraine in the EU and I am hopeful it will improve the quality of life for many people living with the burden of this prevalent neurological disease.”
The Marketing Authorization follows the recommendation for approval by the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) in February. The EC approval will be valid for all 27 EU member states as well as Iceland, Liechtenstein, and Norway and local reimbursement approval will follow. Assessment of the marketing authorization application by the Medicines & Healthcare products Regulatory Agency (MHRA) is underway and approval is expected to shortly follow in the United Kingdom.
About VYDURA® (rimegepant)
VYDURA® targets a key component of migraine by reversibly blocking CGRP receptors. CGRP is increased during a migraine attack, dilates blood vessels and is involved in nociceptor signaling. CGRP receptor antagonists work by reversibly blocking CGRP receptors, thereby inhibiting the biologic activity of the endogenous CGRP neuropeptide.
The Marketing Authorization for VYDURA® was based, in part, on the review of the results from three Phase 3 studies for acute treatment, a long-term, open-label safety study in acute treatment of migraine and a Phase 3 study with a 1-year open-label extension in the preventive treatment of migraine. VYDURA® is taken orally as needed, up to once daily, to stop migraine attacks or taken every other day to help prevent migraine attacks.
The most frequent adverse event in clinical trials with VYDURA® was nausea, occurring in 3% of patients compared to 1% with placebo, while hypersensitivity reactions including rash occurred in less than 1% of patients. Less than 2% of patients discontinued from VYDURA® due to adverse events. VYDURA® does not have addiction potential and was not associated with medication overuse headache or rebound headache in clinical trials, although overuse of any type of medicinal products for headache can make them worse.
VYDURA® is commercialized as Nurtec® and Nurtec® ODT outside Europe. It is commercialized in the U.S. for the acute treatment of migraine and for the preventive treatment of episodic migraine in adults, and ex-U.S. is approved for the acute treatment of migraine in Kuwait and the United Arab Emirates, and for acute treatment of migraine and preventive treatment of episodic migraine in Israel.
Earlier this year, Pfizer and Biohaven entered into an agreement for the commercialization of VYDURA®. Under the terms of the agreement, Pfizer has commercialization rights to rimegepant in markets outside the U.S. Biohaven continues to lead research and development globally and retains the U.S. market.
https://www.biohavenpharma.com/science-pipeline/cgrp/rimegepant
More information about Biohaven is available at www.biohavenpharma.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220427005213/en/
Source: Pfizer Inc.
Pfizer, Biohaven's Vydura gets approval in EU to prevent/treat migraine
Apr. 27, 2022 7:05 AM ET
Biohaven Pharmaceutical Holding Company Ltd. (BHVN), PFE
By: Ravikash, SA News Editor
- The European Commission (EC) granted marketing authorization to Biohaven Pharmaceutical (NYSE:BHVN) and Pfizer's (NYSE:PFE) Vydura (rimegepant) for the acute treatment of migraine with or without aura, and to prevent episodic migraine in adults who have at least four migraine attacks per month.
- https://seekingalpha.com/news/3827439-pfizer-biohavens-vydura-gets-approval-in-eu-to-preventtreat-migraine
- https://seekingalpha.com/symbol/BHVN
- https://www.biohavenpharma.com/science-pipeline/cgrp/rimegepant
- https://seekingalpha.com/symbol/PFE
- https://www.biohavenpharma.com/science-pipeline/cgrp/rimegepant
- https://www.nurtec.com/
RETEVMO® (selpercatinib)
Retsevmo Shareselpercatinib
On 22 April 2022, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending a change to the terms of the marketing authorisation for the medicinal product Retsevmo. The marketing authorisation holder for this medicinal product is Eli Lilly Nederland B.V.
The CHMP adopted an extension to an existing indication as follows:1
Retsevmo as monotherapy is indicated for the treatment of adults with advanced RET fusion positive non-small cell lung cancer (NSCLC) not previously treated with a RET inhibitorwho require systemic therapy following prior treatment with immunotherapy and/or platinum-based chemotherapy.
For information, the full indication for Retsevmo will therefore be as follows:
Retsevmo as monotherapy is indicated for the treatment of adults with:
- advanced RET fusion‑positive non‑small cell lung cancer (NSCLC) not previously treated with a RETinhibitor
- advanced RET fusion‑positive thyroid cancer who require systemic therapy following prior treatment with sorafenib and/or lenvatinib
Retsevmo as monotherapy is indicated for the treatment of adults and adolescents 12 years and older with advanced RET‑mutant medullary thyroid cancer (MTC) who require systemic therapy following prior treatment with cabozantinib and/or vandetanib.
Detailed recommendations for the use of this product will be described in the updated summary of product characteristics (SmPC), which will be published in the revised European public assessment report (EPAR), and will be available in all official European Union languages after a decision on this change to the marketing authorisation has been granted by the European Commission.
https://www.retevmo.com/what-is-ret#testing-for-ret
EMA panel recommends extending indication for Lilly's cancer therapy
Apr. 22, 2022 12:32 PM ET Eli Lilly and Company (LLY)
By: Dania Nadeem, SA News Editor
- The European Union drug regulator's Committee for Medicinal Products for Human Use has recommended extending the indication for Eli Lilly's (NYSE:LLY) Retsevmo to treat lung cancer.
- https://seekingalpha.com/news/3825852-ema-panel-recommends-extending-indication-for-lillys-cancer-therapy
- https://seekingalpha.com/symbol/LLY
- https://www.retevmo.com/what-is-ret#testing-for-ret
- https://www.lilly.com/
EDIT-301: Transfusion-dependent beta thalassemia (TDT)
Editas Medicine Receives FDA Rare Pediatric Disease Designation For EDIT-301 For The Treatment Of Beta Thalassemia
April 26, 2022 at 9:00 AM EDTDownload PDF
CAMBRIDGE, Mass., April 26, 2022 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced that the U.S. Food and Drug Administration (FDA) granted Rare Pediatric Disease designation to EDIT-301, an investigational, gene-edited medicine for the treatment of beta thalassemia. The FDA previously granted Rare Pediatric Disease designation to EDIT-301 for the treatment of sickle cell disease.
“Beta thalassemia is a devastating disease that leads to severe anemia, organ failure, and premature death. Receiving Rare Pediatric Disease designation for EDIT-301 for beta thalassemia highlights the dire need for new treatment options,” said James C. Mullen, Chairman, President, and Chief Executive Officer, Editas Medicine. “EDIT-301 is a potentially transformative medicine for patients living with beta thalassemia, and we look forward to dosing the first patient in our clinical trial this year.”
The FDA defines a rare pediatric disease as a serious or life-threatening disease in which the disease manifestations primarily affect individuals aged from birth to 18 years. Pediatric diseases recognized as “rare” affect fewer than 200,000 people in the United States.
Under the FDA’s Rare Pediatric Disease Designation and Voucher Programs, if Editas receives marketing approval for EDIT-301 for beta thalassemia, the Company may be eligible to receive a Priority Review Voucher (PRV) from the FDA that can be redeemed to receive priority review of a subsequent marketing application for a different product, or the PRV may be sold or transferred.
EDIT-301 is being investigated in a clinical study in patients with severe SCD (RUBY trial, NCT04853576). Editas expects to initiate a Phase 1/2 study of EDIT-301 in patients with transfusion-dependent beta thalassemia in 2022.
About EDIT-301
EDIT-301 is an experimental cell therapy medicine under investigation for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). EDIT-301 consists of patient-derived CD34+ hematopoietic stem and progenitor cells edited at the gamma globin gene (HBG1 and HBG2) promoters, where naturally occurring fetal hemoglobin (HbF) inducing mutations reside, by a highly specific and efficient proprietary engineered AsCas12a nuclease. Red blood cells derived from EDIT-301 CD34+ cells demonstrate a sustained increase in fetal hemoglobin production, which has the potential to provide a one-time, durable treatment benefit for people living with severe sickle cell disease and transfusion-dependent beta thalassemia.
For the latest information and scientific presentations, please visit www.editasmedicine.com.
Editas gets FDA rare pediatric disease status for gene-edited drug EDIT-301 for beta thalassemia
Apr. 26, 2022 9:46 AM ET
By: Ravikash, SA News Editor
- The U.S. Food and Drug Administration (FDA) granted rare pediatric disease designation to Editas Medicine's (NASDAQ:EDIT) gene-edited medicine EDIT-301 to treat beta thalassemia, an inherited blood disorder.
- https://seekingalpha.com/news/3826797-editas-gets-fda-rare-pediatric-disease-status-for-gene-edited-drug-edit-301-for-beta-thalassemia
- https://seekingalpha.com/symbol/EDIT
- https://www.editasmedicine.com/gene-editing-pipeline/
VLA15 – Valneva’s Lyme disease vaccine candidate
Valneva and Pfizer Report Positive Phase 2 Pediatric Data for Lyme Disease Vaccine Candidate
April 26, 2022
- Strong immunogenicity profile observed in study participants aged 5-17 years one month after the primary vaccination series
- Safety profile observed in pediatric participants similar to previously reported data in adult participants
- Pediatric population to be included in planned Phase 3 trial – expected to start in Q3 2022, subject to regulatory approval
Saint-Herblain (France) and New York, April 26, 2022 – Valneva SE (Nasdaq: VALN; Euronext Paris: VLA), a specialty vaccine company, and Pfizer Inc. (NYSE: PFE) today reported positive Phase 2 pediatric data for their Lyme disease vaccine candidate, VLA15. Based on these new results, Valneva and Pfizer plan to proceed with inclusion of pediatric participants in their planned Phase 3 trial. The trial will evaluate VLA15 in adults and pediatric subjects 5 years of age and above and is expected to be initiated in the third quarter of 2022, subject to regulatory approval.
The Phase 2 trial, VLA15-221, is the first clinical study with VLA15 which enrolled a pediatric population (5-17 years old). It compared the immunogenicity and safety of VLA15 after administration of two (at months 0 and 6) or three (at months 0, 2 and 6) primary series doses in groups aged 5-11, 12-17 and 18-65 years. In pediatric participants (5-17 years old) who received VLA15 in either the two-dose schedule (N=93) or three-dose schedule (N=97), VLA15 was found to be more immunogenic than in adults with both vaccination schedules tested. These data build on the strong immunogenicity profile already reported for adult participants (18-65 years old) in February 2022[1]. Like in adults, the immunogenicity and safety data support a three-dose primary vaccination schedule in pediatric participants in the Phase 3 study.
The safety and tolerability profile observed in the 5- to 17-year age group was similar to the previously reported profile in adult participants. No vaccine-related serious adverse events (SAEs) were observed.
Valneva and Pfizer plan to submit these data for publication and presentation at a future scientific congress.
Juan Carlos Jaramillo M.D., Chief Medical Officer of Valneva, said, “Lyme disease affects all age groups, but with their affinity for being active outdoors, the pediatric population is at the greatest risk of Lyme disease. These first pediatric results are therefore extremely important and support the inclusion of pediatric participants in our planned Phase 3 trial. In partnership with Pfizer, we are excited to further investigate our VLA15 vaccine candidate, which will hopefully help protect both adults and children against Lyme disease.”
Kathrin U. Jansen, Ph.D., Senior Vice President and Head of Vaccine Research & Development at Pfizer, said: “The medical need for vaccination against Lyme disease is steadily increasing as the geographic footprint of the disease widens. These positive pediatric data mark an important step forward in the ongoing development of VLA15, and we are excited to continue working with Valneva to potentially help protect both adults and children from Lyme disease.”
About VLA15
VLA15 is the only Lyme disease vaccine candidate currently in clinical development. This investigational multivalent protein subunit vaccine uses an established mechanism of action for a Lyme disease vaccine that targets the outer surface protein A (OspA) of Borrelia burgdorferi, the bacteria that cause Lyme disease. OspA is one of the most dominant surface proteins expressed by the bacteria when present in a tick. Blocking OspA inhibits the bacterium’s ability to leave the tick and infect humans. The vaccine covers the six most common OspA serotypes expressed by Borrelia burgdorferi sensu lato species that are prevalent in North America and Europe. VLA15 has demonstrated a strong immunogenicity and safety profile in pre-clinical and clinical studies so far. The program was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) in July 20172. Valneva and Pfizer entered into a collaboration agreement in April 2020 to co-develop VLA153.
About Clinical Study VLA15-221
VLA15-221 is a randomized, observer-blind, placebo-controlled Phase 2 study. It is the first clinical study with VLA15 which enrolled a pediatric population (5-17 years old).
585 healthy participants received VLA15 at two different immunization schedules (month 0-2-6 [N=190] or month 0-6 [N=187]) or three doses of placebo (month 0-2-6 [N=208]). Vaccine recipients received VLA15 at a dose of 180 µg, which was selected based on data generated in the two previous Phase 2 studies. The main safety and immunogenicity readout was performed one month after the primary vaccination series. A subset of participants will receive a booster dose of VLA15 or placebo at month 18 (booster phase) and will be followed for three additional years to monitor antibody persistence.
VLA15 is tested as an alum-adjuvanted formulation and administered intramuscularly. The study is being conducted at U.S. sites located in areas where Lyme disease is endemic and has enrolled both volunteers with a cleared past infection with Borrelia burgdorferi as well as Borrelia burgdorferi-naïve volunteers.
About Valneva SE
Valneva is a specialty vaccine company focused on the development and commercialization of prophylactic vaccines for infectious diseases with significant unmet medical need. The Company takes a highly specialized and targeted approach to vaccine development and then applies its deep understanding of vaccine science to develop prophylactic vaccines addressing these diseases. Valneva has leveraged its expertise and capabilities both to successfully commercialize two vaccines and to rapidly advance a broad range of vaccine candidates into and through the clinic, including candidates against Lyme disease, the chikungunya virus and COVID-19.
In addition, to learn more, please visit us on www.Pfizer.com and follow us on Twitter at @Pfizer and @Pfizer News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.
Pfizer, Valneva Lyme disease vaccine shows response in kids over 5 in phase 2 trial
Apr. 26, 2022 5:57 AM ET
By: Ravikash, SA News Editor
Pfizer (NYSE:PFE) Valneva (NASDAQ:VALN) said they plan to include children above 5 years of age in a planned phase 3 trial of their Lyme disease vaccine VLA15 after positive pediatric data was reported from a phase 2 trial, which included children aged 5 years to 17 years, among others.
https://seekingalpha.com/symbol/VALN
https://seekingalpha.com/symbol/PFE
biotecMAX™ Feb/Mar/APR 2022 Insight
ENHERTU® (fam-trastuzumab deruxtecan-nxki)
Enhertu granted Priority Review in the US for patients with previously treated HER2-mutant metastatic non-small cell lung cancer
PUBLISHED19 April 2022 19 April 2022 07:00 BST
Based on pivotal DESTINY-Lung01 results showing AstraZeneca and Daiichi Sankyo’s Enhertu demonstrated a 54.9% tumour response rate
If approved, Enhertu to provide patients with a much-needed targeted therapy option
AstraZeneca and Daiichi Sankyo have received notification of acceptance of the supplemental Biologics License Application (sBLA) of Enhertu (trastuzumab deruxtecan) for the treatment of adult patients in the US with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumours have a HER2 (ERBB2) mutation and who have received a prior systemic therapy. The application has also been granted Priority Review.
Enhertu is a HER2-directed antibody drug conjugate (ADC) being jointly developed by AstraZeneca and Daiichi Sankyo.
The Food and Drug Administration (FDA) grants Priority Review to applications for medicines that, if approved, would offer significant improvements over available options by demonstrating safety or efficacy improvements, preventing serious conditions, or enhancing patient compliance.1 The Prescription Drug User Fee Act (PDUFA) date, the FDA action date for their regulatory decision, is during the third quarter of 2022. The Priority Review follows Breakthrough Therapy Designation granted by the FDA for Enhertu in this cancer type in May 2020.
Lung cancer is the second most common form of cancer globally, with more than two million new cases diagnosed in 2020.2 For patients with metastatic NSCLC, prognosis is particularly poor, as only approximately 8% will live beyond five years after diagnosis.3 There are currently no HER2-directed therapies approved specifically for the treatment of HER2-mutant NSCLC,4 which occurs in approximately 2-4% of patients with non-squamous NSCLC.4,5
Susan Galbraith, Executive Vice President, Oncology R&D, AstraZeneca, said: “The DESTINY-Lung01 trial confirmed the HER2 mutation as an actionable biomarker in non-small cell lung cancer. If approved, Enhertu has the potential to become a new standard treatment in this patient population, offering a much-needed option for patients with HER2-mutant metastatic non-small cell lung cancer who currently have no targeted treatment options.”
Ken Takeshita, MD, Global Head, R&D, Daiichi Sankyo, said: “The results of DESTINY-Lung01 showed that Enhertu is the first HER2-directed therapy to demonstrate a strong and robust tumour response in more than half of patients with previously treated HER2-mutant metastatic non-small cell lung cancer. Seeking approval in the US for a third tumour type in three years further demonstrates the significant potential of Enhertu in treating multiple HER2-targetable cancers.”
The sBLA is based on data from the registrational DESTINY-Lung01 Phase II trial published in The New England Journal of Medicine, and is supported by the Phase I trial (DS8201-A-J101) published in Cancer Discovery.
Primary results from previously-treated patients with HER2-mutations (cohort 2) of DESTINY-Lung01 demonstrated a confirmed objective response rate (ORR) of 54.9% (95% confidence interval [CI]: 44.2-65.4) in patients treated with Enhertu (6.4mg/kg) as assessed by independent central review (ICR). One (1.1%) complete response (CR) and 49 (53.8%) partial responses (PR) were observed.
A confirmed disease control rate (DCR) of 92.3% was seen with a reduction in tumour size observed in most patients. After a median follow-up of 13.1 months, the median duration of response (DoR) for Enhertu was 9.3 months. The median progression-free survival (PFS) was 8.2 months and the median overall survival (OS) was 17.8 months.
The safety profile of the most common adverse events with Enhertu in DESTINY-Lung01 was consistent with previous clinical trials with no new safety concerns identified.
Enhertu is being further assessed in a comprehensive clinical development programme evaluating efficacy and safety across multiple HER2-targetable cancers, including breast, gastric, lung and colorectal cancers.
DESTINY-Lung01
DESTINY-Lung01 is a global Phase II, open-label, two-cohort trial evaluating the safety and efficacy of Enhertu in patients with HER2-mutant (6.4mg/kg) or HER2-overexpressing (defined as IHC3+ or IHC2+) [6.4mg/kg and 5.4mg/kg] unresectable and/or metastatic non-squamous NSCLC who had progressed after one or more systemic therapies. The primary endpoint is confirmed ORR by ICR. Key secondary endpoints include DoR, DCR, PFS, OS and safety. DESTINY-Lung01 enrolled approximately 180 patients at multiple sites, including Asia, Europe and North America. For more information about the trial, visit ClinicalTrials.gov.
Enhertu
Enhertu is a HER2-directed ADC. Designed using Daiichi Sankyo’s proprietary DXd ADC technology, Enhertu is the lead ADC in the oncology portfolio of Daiichi Sankyo and the most advanced programme in AstraZeneca’s ADC scientific platform. Enhertu consists of a HER2 monoclonal antibody attached to a topoisomerase I inhibitor payload, an exatecan derivative, via a stable tetrapeptide-based cleavable linker.
Enhertu (5.4mg/kg) is approved in more than 40 countries for the treatment of adult patients with unresectable or metastatic HER2-positive breast cancer who have received two or more prior anti-HER2-based regimens based on the results from the DESTINY-Breast01 trial.
Enhertu (6.4mg/kg) is approved in several countries for the treatment of adult patients with locally advanced or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma who have received a prior trastuzumab-based regimen based on the results from the DESTINY-Gastric01 trial.
Daiichi Sankyo collaboration
Daiichi Sankyo Company, Limited (TSE: 4568) [referred to as Daiichi Sankyo] and AstraZeneca entered into a global collaboration to jointly develop and commercialise Enhertu (a HER2-directed ADC) in March 2019, and datopotamab deruxtecan (DS-1062; a TROP2-directed ADC) in July 2020, except in Japan where Daiichi Sankyo maintains exclusive rights. Daiichi Sankyo is responsible for the manufacturing and supply of Enhertu and datopotamab deruxtecan.
Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca.
AstraZeneca, Daiichi Sankyo's Enhertu gets FDA priority review in lung cancer subtype
Apr. 19, 2022 4:36 AM ET AstraZeneca PLC (AZN), DSNKYDSKYF
By: Ravikash, SA News Editor
- The U.S. Food and Drug Administration (FDA) granted priority review to AstraZeneca (NASDAQ:AZN) and Daiichi Sankyo's (OTCPK:DSKYF) (OTCPK:DSNKY) Enhertu to treat adult patients with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have a HER2 (ERBB2) mutation and who have received a prior systemic therapy.
- https://seekingalpha.com/news/3824153-astrazeneca-daiichi-sankyos-enhertu-gets-fda-priority-review-in-lung-cancer-subtype
- https://seekingalpha.com/symbol/DSNKY
- https://seekingalpha.com/symbol/DSKYF
- https://seekingalpha.com/symbol/AZN
- https://www.enhertu.com/en/
- https://www.astrazeneca.com/
TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor)
Health Canada Grants Marketing Authorization for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in Children With Cystic Fibrosis Ages 6 Through 11 years With At Least One F508del Mutation
-Approximately 500 Canadians ages 6-11 are now eligible for TRIKAFTA®-
-Vertex has submitted this indication to CADTH & INESSS for Health Technology Assessments-
BOSTON--(BUSINESS WIRE)--Apr. 20, 2022--
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Health Canada has granted Marketing Authorization for the expanded use of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include children with cystic fibrosis (CF) ages 6 through 11 years who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. With this announcement, approximately 500 Canadians with CF ages 6-11 are now eligible for TRIKAFTA®. As a result of this approval, an additional dosage strength of TRIKAFTA® tablets is now available (elexacaftor 50 mg/tezacaftor 25 mg/ivacaftor 37.5 mg and ivacaftor 75 mg).
“We are delighted that TRIKAFTA is now available for these young patients in Canada. It provides a new treatment option for those with CF ages 6-11 with at least one F508del mutation and a first-in-class treatment option for the approximately 500 6-11-year-olds who are newly eligible for a medicine that treats the underlying cause of their disease,” said Reshma Kewalramani, M.D., Chief Executive Officer and President at Vertex. “This important milestone brings us one step closer to our ultimate goal of developing treatments for all patients living with CF. We will now work closely with all provinces and territories to secure access for eligible patients as quickly as possible.”
Vertex completed a 24-week Phase 3 open-label, multicenter study which enrolled 66 children ages 6 through 11 years old with CF who have either two copies of the F508del mutation or one copy of the F508del mutation and one minimal function mutation to evaluate the safety, pharmacokinetics and efficacy of TRIKAFTA®. The regimen was generally well tolerated, and safety data were consistent with those observed in previous studies in patients ages 12 years and older.
“As a trial investigator, I have seen firsthand the demonstrated efficacy of TRIKAFTA in people ages 6-11 living with cystic fibrosis,” said Larry C. Lands, M.D., Ph.D., Director, Pediatric Respiratory Medicine, Pediatric Cystic Fibrosis Clinic, and Pediatric Pulmonary Function Laboratory, Montreal Children’s Hospital, McGill University Health Center, and Professor, Department of Pediatrics, Faculty of Medicine, McGill University. “This is an exciting next step that will allow eligible patients to begin treatment earlier.”
Vertex has also submitted this indication to both the Canadian Agency for Drugs and Technologies in Health (CADTH) and the Institut national d'excellence en santé et en services sociaux (INESSS) in Québec for Health Technology Assessments.
About TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor)
In people with certain types of mutations in the CFTR gene, the CFTR protein is not processed or folded normally within the cell, and this can prevent the CFTR protein from reaching the cell surface and functioning properly. TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is an oral medicine designed to increase the quantity and function of the CFTR protein at the cell surface. Elexacaftor and tezacaftor work together to increase the amount of mature protein at the cell surface by binding to different sites on the CFTR protein. Ivacaftor, which is known as a CFTR potentiator, is designed to facilitate the ability of CFTR proteins to transport salt and water across the cell membrane. The combined actions of elexacaftor, tezacaftor and ivacaftor help hydrate and clear mucus from the airways. TRIKAFTA® is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients ages 6 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220413006045/en/
Source: Vertex Pharmaceuticals Incorporated
Canada approves expanded use of Vertex's cystic fibrosis treatment Trikafta for kids aged 6-11
Apr. 20, 2022 6:02 PM ET
Vertex Pharmaceuticals Incorporated (VRTX)
By: Anuron Mitra, SA News Editor
- Vertex Pharmaceuticals (NASDAQ:VRTX) on Wednesday said Canada approved the expanded use of the company's cystic fibrosis treatment Trikafta to include children aged 6-11 years.
- https://seekingalpha.com/news/3825062-canada-approves-expanded-use-of-vertexs-cystic-fibrosis-treatment-trikafta-for-kids-aged-6-11
- https://seekingalpha.com/symbol/VRTX
- https://www.trikafta.com/
Tislelizumab
China NMPA Approves Tislelizumab for Patients with Second-Line Esophageal Squamous Cell Carcinoma
Apr 15, 2022 6:00 AM
Tislelizumab is now approved for eight indications in China
CAMBRIDGE, Mass. & BASEL, Switzerland & BEIJING--(BUSINESS WIRE)-- BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global, science-driven biotechnology company focused on developing innovative and affordable medicines to improve treatment outcomes and access for patients worldwide, today announced that the China National Medical Products Administration (NMPA) has granted approval to BeiGene’s anti-PD-1 antibody, tislelizumab, as a treatment for patients with locally advanced or metastatic esophageal squamous cell carcinoma (ESCC) who have disease progression or are intolerant to first-line standard chemotherapy.
“As a second-line treatment for patients with ESCC, this differentiated checkpoint inhibitor demonstrated significant improvements in overall survival and was generally well-tolerated in our Phase 3 trial of tislelizumab,” commented Mark Lanasa, M.D., Ph.D., Senior Vice President, Chief Medical Officer, Solid Tumors, at BeiGene. “Tislelizumab regulatory submissions in this indication submitted by Novartis are under review by the U.S. FDA and the European Medicines Agency, highlighting our commitment to advancing its progress on behalf of the many patients around the world with ESCC and other forms of cancer.”
“With eight approved indications in China, our science-based commercial team of more than 3,100+ professionals is working to make tislelizumab more broadly available to those in China who may benefit from this important immunotherapy,” commented Xiaobin Wu, Ph.D., President, Chief Operating Officer, and General Manager of China, at BeiGene. “Today’s approval is a great step for patients in China with ESCC.”
“The NMPA’s approval of tislelizumab is welcome news to patients with previously treated ESCC, for whom we are pleased to now be able to provide this new treatment option,” said Lin Shen, Vice President of Clinical Oncology, Beijing Cancer Hospital, and the principal investigator of the trial. “The global Phase 3 clinical trial of tislelizumab demonstrated positive safety and efficacy outcomes as a second-line treatment for patients with ESCC, one of the most common malignant tumors in the digestive tract.”
This approval was supported by clinical results from a randomized, open-label, multi-center, global Phase 3 clinical trial, RATIONALE 302 (NCT03430843), to evaluate the efficacy and safety of tislelizumab as a second-line treatment for patients with locally advanced or metastatic ESCC compared to chemotherapy. The primary endpoint of this trial is overall survival (OS) in the intent-to-treat (ITT) population; a key secondary endpoint is OS in patients with high PD-L1 expression (defined as visually-estimated combined positive score [vCPS] ≥10%); and other secondary endpoints include progression-free survival (PFS), objective response rate (ORR), duration of response (DoR), and safety. A total of 512 patients were enrolled in the trial in 11 countries and regions in Asia, Europe, and North America, randomized 1:1 to either the tislelizumab arm or chemotherapy arm (investigator’s choice of paclitaxel, docetaxel, or irinotecan). Results of this trial were presented at the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting.
Tislelizumab is also under regulatory review in the U.S. and the European Union, submitted by Novartis in their licensed territories, as a second-line treatment for patients with locally advanced or metastatic ESCC.
About Tislelizumab
Tislelizumab is an anti-programmed death receptor-1 (PD-1) inhibitor designed to help aid the body’s immune cells to detect and fight tumors. Tislelizumab, a humanized monoclonal antibody, is specifically designed to minimize binding to FcγR on macrophages. In pre-clinical studies, binding to FcγR on macrophages has been shown to compromise the anti-tumor activity of PD-1 antibodies through activation of antibody-dependent macrophage-mediated killing of T effector cells.
Tislelizumab is the first drug from BeiGene’s immuno-oncology biologics program and is being developed internationally as a monotherapy and in combination with other therapies for the treatment of a broad array of both solid tumor and hematologic cancers. BeiGene has initiated or completed more than 20 potentially registration-enabling clinical trials in 35 countries and regions, including 17 Phase 3 trials and four pivotal Phase 2 trials.
Tislelizumab is approved by the China National Medical Products Administration (NMPA) as a treatment for eight indications, including multiple approvals in non-small cell lung cancer (NSCLC). Tislelizumab has been submitted for regulatory review in one additional indication in China and as a potential treatment for unresectable recurrent locally advanced or metastatic ESCC after prior systemic therapy in the U.S., and in NSCLC and ESCC in Europe. In January 2021, BeiGene announced a collaboration with Novartis to accelerate the clinical development and marketing of tislelizumab in North America, Europe and Japan.
Tislelizumab is not approved for use outside of China.
https://www.beigene.com/en-us/science-and-product-portfolio/pipeline/tislelizumab
To learn more about BeiGene, please visit www.beigene.com and follow us on Twitter at @BeiGeneGlobal.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220415005045/en/
Source: BeiGene
BeiGene, Novartis' tislelizumab gets approval in China for esophagus cancer
Apr. 18, 2022 6:52 AM ET BeiGene, Ltd. (BGNE)NVS
By: Ravikash, SA News Editor
- BeiGene (NASDAQ:BGNE) and Novartis' (NVS) tislelizumab was approved by China's National Medical Products Administration (NMPA) to treat patients with locally advanced or metastatic esophageal squamous cell carcinoma (ESCC) who have disease progression or are intolerant to first-line standard chemotherapy.
- https://seekingalpha.com/news/3823759-beigene-novartis-tislelizumab-gets-approval-in-china-for-esophagus-cancer
- https://seekingalpha.com/symbol/NVS
- https://seekingalpha.com/symbol/BGNE
Opdivo (nivolumab) with Chemotherapy
Neoadjuvant Opdivo (nivolumab) with Chemotherapy Significantly Improves Event-Free Survival in Patients with Resectable Non-Small Cell Lung Cancer in Phase 3 CheckMate -816 Trial
04/11/2022CATEGORY:
When administered before surgery, three cycles of Opdivo in combination with chemotherapy reduced the risk of disease recurrence, progression or death by 37% and showed an encouraging early trend in overall survival
CheckMate -816 is the first positive Phase 3 trial with an immunotherapy-based combination in the neoadjuvant setting of non-small cell lung cancer and fourth Opdivo trial to demonstrate benefit in earlier stages of cancer
Data featured as an oral presentation during a Clinical Trials Plenary Session at the American Association for Cancer Research Annual Meeting 2022 and simultaneously published in The New England Journal of Medicine
Based on the CheckMate -816 data, Opdivo in combination with chemotherapy received U.S. Food and Drug Administration approval for the neoadjuvant treatment of certain patients with resectable non-small cell lung cancer
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced results from the Phase 3 CheckMate -816 trial, which showed that neoadjuvant treatment with three cycles of Opdivo (nivolumab) in combination with chemotherapy significantly improved event-free survival (EFS), a primary endpoint, compared to chemotherapy alone in patients with resectable non-small cell lung cancer (NSCLC). With a minimum follow-up of 21.0 months, Opdivo with chemotherapy reduced the risk of disease recurrence, progression or death by 37% (Hazard Ratio [HR] 0.63; 97.38% Confidence Interval [CI]: 0.43 to 0.91; p=0.0052) across randomized patients when administered before surgery. In patients receiving the combination, median EFS was 31.6 months, compared to 20.8 months for patients treated with chemotherapy alone.
Additionally, while the data are still immature and the analysis did not reach statistical significance, favorable early overall survival (OS) results were observed with Opdivo in combination with chemotherapy (HR 0.57; 99.67% CI: 0.30 to 1.07). At two years, 83% of patients treated with neoadjuvant Opdivo and chemotherapy were alive, compared to 71% with chemotherapy alone. OS will continue to be followed for upcoming analyses.
CheckMate -816 represents the first Phase 3 study with an immunotherapy-based combination to show a significant improvement in EFS, as well as in the other primary endpoint of pathologic complete response (pCR), in the neoadjuvant setting of NSCLC. The EFS data are being presented for the first time during the Neoadjuvant and Perioperative Immunotherapy Clinical Trials Plenary Session (Abstract #CT012) at the American Association for Cancer Research (AACR) Annual Meeting 2022 on Monday, April 11, 2022, from 10:15 a.m. to 12:15 p.m. CT and simultaneously published in The New England Journal of Medicine.
“While resectable non-small cell lung cancer may be curable in some cases, patients face a high probability of recurrence after surgery, so we need effective systemic treatment options to interrupt this trajectory,” said Nicolas Girard, M.D., Ph.D., CheckMate -816 investigator and professor and head of the Thorax Institute Curie-Montsouris. “The results from CheckMate -816 represent the first demonstration of clear and significant benefits with neoadjuvant immunotherapy-based treatment over chemotherapy alone for these patients, initially seen with increased pathologic complete response and now with improved event-free survival and a positive trend in overall survival. As we work toward the ultimate goal of curing these patients, these data suggest the potential for better long-term outcomes with nivolumab in combination with chemotherapy.”
In the study, the safety profile of the neoadjuvant Opdivo-chemotherapy combination was consistent with previous reports, and no new safety signals were observed at the time of the EFS analysis. Rates of Grade 3-4 treatment-related adverse events were similar with the Opdivo-chemotherapycombination versus chemotherapy alone (34% vs. 37%), as were all causality surgery-related Grade 3-4 adverse events (11% with the combination vs. 15% with chemotherapy). With Opdivo in combination with chemotherapy, 83% of patients went on to receive surgery, compared to 75% with chemotherapy.
“Surgery is still the cornerstone of cure for patients with non-small cell lung cancer,” said Jonathan Spicer M.D., Ph.D., CheckMate -816 investigator; associate professor of surgery, McGill University; and attending surgeon, division of thoracic and upper gastrointestinal surgery, Montreal General Hospital, McGill University Health Centre. “The fact that neoadjuvant nivolumab with chemotherapy enabled shorter, less invasive and less extensive operations without increasing complications or adverse events is of tremendous importance to thoracic surgeons and their patients. These findings, combined with the improved survival outcomes, have the potential to completely change the way surgeons and oncologists collaborate in treating patients with resectable non-small cell lung cancer.”
“Immunotherapy has ushered in a new era of treatment in metastatic cancers, changing survival expectations for patients with lung cancer and many other tumor types. More recently, our understanding of the biology of the immune system and cancer has led us to explore the role of immunotherapy in the neoadjuvant, adjuvant and peri-operative settings,” said Abderrahim Oukessou, M.D., vice president, thoracic cancers development lead, Bristol Myers Squibb. “The data from CheckMate -816, including the positive early overall survival results, reinforce the importance of researching immunotherapy in earlier stages of disease, and we look forward to continuing to see this science translate into tangible benefits for patients and their families.”
Based on the EFS and pCR results from CheckMate -816,the U.S. Food and Drug Administration approved Opdivo in combination with platinum-doublet chemotherapy every three weeks for three cycles for adult patients with resectable (tumors ≥4 cm or node positive) NSCLC in the neoadjuvant setting in March 2022, and further applications are under review with health authorities globally.
In non-metastatic NSCLC, Bristol Myers Squibb and collaborators are exploring the use of immunotherapy in the neoadjuvant, adjuvant and peri-operative settings, as well as in association with chemoradiation. The scientific rationale for using immunotherapy in the neoadjuvant setting is twofold: the presence of a tumor during immunotherapy treatment may enable a stronger immune response, potentially making the treatment more effective against a primary tumor, while offering an early opportunity to target covert micro-metastasis. To date, Opdivo-based treatments have shown improved efficacy in the neoadjuvant or adjuvant treatment of four tumor types: lung cancer, bladder cancer, esophageal/gastroesophageal junction cancer and melanoma.
Bristol Myers Squibb thanks the patients and investigators involved in the CheckMate -816 clinical trial.
About CheckMate -816
CheckMate -816 is a Phase 3 randomized, open label, multi-center trial evaluating Opdivo in combination with chemotherapy compared to chemotherapy alone as neoadjuvant treatment in patients with resectable stage IB to IIIA non-small cell lung cancer (per the 7th edition American Joint Committee on Cancer/Union for International Cancer Control staging criteria), regardless of PD-L1 expression. For the primary analysis, 358 patients were randomized to receive either Opdivo 360 mg with histology-based platinum doublet chemotherapy every three weeks for three cycles, or platinum doublet chemotherapy every three weeks for three cycles, followed by surgery. The primary endpoints of the trial are event-free survival and pathologic complete response. Secondary endpoints include overall survival, major pathologic response, and time to death or distant metastases.
About Opdivo
Opdivo is a programmed death-1 (PD-1) immune checkpoint inhibitor that is designed to uniquely harness the body’s own immune system to help restore anti-tumor immune response. By harnessing the body’s own immune system to fight cancer, Opdivo has become an important treatment option across multiple cancers.
Opdivo’s leading global development program is based on Bristol Myers Squibb’s scientific expertise in the field of Immuno-Oncology, and includes a broad range of clinical trials across all phases, including Phase 3, in a variety of tumor types. To date, the Opdivo clinical development program has treated more than 35,000 patients. The Opdivo trials have contributed to gaining a deeper understanding of the potential role of biomarkers in patient care, particularly regarding how patients may benefit from Opdivo across the continuum of PD-L1 expression.
In July 2014, Opdivo was the first PD-1 immune checkpoint inhibitor to receive regulatory approval anywhere in the world. Opdivo is currently approved in more than 65 countries, including the United States, the European Union, Japan and China. In October 2015, the Company’s Opdivo and Yervoy combination regimen was the first Immuno-Oncology to receive regulatory approval for the treatment of metastatic melanoma and is currently approved in more than 50 countries, including the United States and the European Union.
INDICATIONS
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of adult patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of adult patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of adult patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection.
OPDIVO® (nivolumab), in combination with platinum-doublet chemotherapy, is indicated as neoadjuvant treatment of adult patients with resectable (tumors ≥4 cm or node positive) non-small cell lung cancer (NSCLC).
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express PD-L1 (≥1%) as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab) and 2 cycles of platinum-doublet chemotherapy, is indicated for the first-line treatment of adult patients with metastatic or recurrent non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving OPDIVO.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with unresectable malignant pleural mesothelioma (MPM).
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with intermediate or poor risk advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab), in combination with cabozantinib, is indicated for the first-line treatment of adult patients with advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy.
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with classical Hodgkin lymphoma (cHL) that has relapsed or progressed after autologous hematopoietic stem cell transplantation (HSCT) and brentuximab vedotin or after 3 or more lines of systemic therapy that includes autologous HSCT. This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN) with disease progression on or after platinum-based therapy.
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
OPDIVO® (nivolumab), as a single agent, is indicated for the adjuvant treatment of adult patients with urothelial carcinoma (UC) who are at high risk of recurrence after undergoing radical resection of UC.
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of adult and pediatric (12 years and older) patients with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of adult patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) after prior fluoropyrimidine- and platinum-based chemotherapy.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of completely resected esophageal or gastroesophageal junction cancer with residual pathologic disease in adult patients who have received neoadjuvant chemoradiotherapy (CRT).
OPDIVO® (nivolumab), in combination with fluoropyrimidine- and platinum- containing chemotherapy, is indicated for the treatment of adult patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma.
Please see US Full Prescribing Information for OPDIVO and YERVOY.
Source: Bristol Myers Squibb
Bristol Myers highlights potential of Opdivo when used before surgery in lung cancer
Apr. 11, 2022 12:34 PM ET
Bristol-Myers Squibb Company (BMY)
By: Dulan Lokuwithana, SA News Editor
Announcing Phase 3 data from its CheckMate -816 trial, Bristol Myers Squibb (NYSE:BMY) announced on Monday that its immune checkpoint inhibitor, Opdivo, “significantly improved” event-free survival (EFS) in lung cancer when administered with chemotherapy before surgery.
https://seekingalpha.com/symbol/BMY
V116, the Company’s Investigational 21-Valent Pneumococcal Conjugate Vaccine
Merck Announces U.S. FDA has Granted Breakthrough Therapy Designation for V116, the Company’s Investigational 21-Valent Pneumococcal Conjugate Vaccine, for the Prevention of Invasive Pneumococcal Disease and Pneumococcal Pneumonia in Adults
April 14, 2022 6:45 am ET
V116 Designed to Target Serotypes that Account for 85% of all Invasive Pneumococcal Disease in Individuals Aged 65 and Over in the United States as of 20191
Phase 3 Clinical Trials for V116 to be Initiated in 2022
KENILWORTH, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, announced today that V116, the company’s investigational 21-valent pneumococcal conjugate vaccine, has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for the prevention of invasive pneumococcal disease (IPD) and pneumococcal pneumonia caused by Streptococcus pneumoniae serotypes 3, 6A/C, 7F, 8, 9N, 10A, 11A, 12F, 15A, 15B/C, 16F, 17F, 19A, 20, 22F, 23A, 23B, 24F, 31, 33F, 35B in adults 18 years of age and older. Phase 3 clinical trials for V116 are to be initiated later this year.
The FDA’s decision was informed by data from a two-part randomized, comparator-controlled, double-blind Phase 1/2 study, V116-001 (NCT04168190), that assessed the safety, tolerability, and immunogenicity of a single dose of V116 in pneumococcal vaccine-naïve adults 18-49 years of age (Phase 1) and 50 years of age and older (Phase 2). Full results from the V116-001 study are planned for presentation at the upcoming International Symposium on Pneumococci and Pneumococcal Diseases (ISPPD) in June.
“V116 is specifically designed to address strains of disease-causing pneumococcal bacteria that are most prevalent in adults, reflecting our population-specific approach to developing pneumococcal conjugate vaccines. V116 targets serotypes that account for 85% of all invasive pneumococcal disease in individuals aged 65 and over in the United States as of 20191 and it includes 8 serotypes not covered by currently licensed vaccines,” said Dr. Eliav Barr, senior vice president, head of global clinical development and chief medical officer, Merck Research Laboratories. “We look forward to discussing the ongoing development of this investigational vaccine, including the approach for Phase 3 studies, with the FDA and other regulatory agencies.”
The Breakthrough Therapy Designation is an FDA program designed to expedite the development and review of products intended for serious or life-threatening conditions. To qualify for this designation, preliminary clinical evidence must indicate that the product may demonstrate substantial improvement over currently available options on at least one clinically significant endpoint. The benefits of this Breakthrough Therapy Designation include more intensive guidance from the FDA on an efficient development program, access to a scientific liaison to help accelerate review time and potential eligibility for Priority Review if relevant criteria are met.
For more information, visit www.merck.com and connect with us on Twitter, Facebook, Instagram, YouTube and LinkedIn.
Source: Merck & Co., Inc.
Merck's pneumococcal vaccine V116 gets FDA breakthrough therapy designation
Apr. 14, 2022 7:05 AM ET
By: Ravikash, SA News Editor2 Comments
The U.S. Food and Drug Administration (FDA) granted breakthrough therapy designation to Merck's (NYSE:MRK) pneumococcal vaccine V116.
https://seekingalpha.com/symbol/MRK
Lebrikizumab Combined with Topical Corticosteroids
Lilly's Lebrikizumab Combined with Topical Corticosteroids Showed Significant Improvements in Disease Severity for Atopic Dermatitis
April 11, 2022Download PDF
Lebrikizumab significantly improved several areas of great importance to patients with atopic dermatitis, including
skin and itch, in pivotal combination trial that met all primary and key secondary endpoints
INDIANAPOLIS, April 11, 2022 /PRNewswire/ -- At 16 weeks, 70 percent of patients with moderate-to-severe atopic dermatitis (AD) receiving lebrikizumab combined with standard-of-care topical corticosteroids (TCS) achieved at least 75 percent improvement in overall disease severity (EASI-75*) in the ADhere trial, Eli Lilly and Company (NYSE: LLY) announced today at the 4th Annual Revolutionizing Atopic Dermatitis (RAD) Conference. Lebrikizumab, an investigational IL-13 inhibitor, also showed improvements in itch, sleep interference, and quality of life when combined with TCS, compared to placebo plus TCS.
"Today's ADhere data, together with results from the ADvocate monotherapy studies, demonstrate the potential for lebrikizumab to reduce disease burden and provide relief for people with uncontrolled atopic dermatitis when used either alone or combined with topicals," said Eric Simpson, M.D., M.C.R., Professor of Dermatology and Director of Clinical Research at Oregon Health & Science University in Portland, and principal investigator of ADhere. "Lebrikizumab specifically targets the IL-13 pathway, which plays the central role in this chronic inflammatory disease. These results strengthen our understanding of lebrikizumab in atopic dermatitis and help establish it as a possible new treatment option."
Lebrikizumab is a novel, monoclonal antibody (mAb) that binds to the interleukin 13 (IL-13) protein with high affinity to specifically prevent the formation of IL-13Rα1/IL-4Rα (Type 2 receptor) which blocks downstream signaling through the IL-13 pathway.1-5 IL-13 plays the central role in Type 2 inflammation in AD.6,7 In AD, IL-13 underlies the signs and symptoms including skin barrier dysfunction, itch, infection and hard, thickened areas of skin.8
Among patients taking lebrikizumab plus TCS, 41 percent achieved clear or almost clear skin (IGA) at 16 weeks compared to 22 percent of patients taking placebo plus TCS. At 16 weeks, 70 percent of patients taking lebrikizumab plus TCS achieved an EASI-75 response compared to 42 percent taking placebo plus TCS. Differences between patients receiving lebrikizumab in combination with TCS and placebo with TCS were observed as early as four weeks for EASI-75.
Patients treated with lebrikizumab plus TCS also achieved statistically significant improvements across key secondary endpoints including skin clearance and itching, interference of itch on sleep, and quality of life measures, compared to placebo with TCS. Clinically meaningful differences were observed as early as four weeks for itch, interference of itch on sleep, and quality of life measures.
Safety results were consistent with prior lebrikizumab studies in AD. Patients taking lebrikizumab plus TCS, compared to placebo plus TCS, reported a higher frequency of adverse events (lebrikizumab plus TCS: 43%, placebo plus TCS: 35%). Most adverse events were mild or moderate in severity and nonserious and did not lead to treatment discontinuation. The most common adverse events for those on lebrikizumab were conjunctivitis (5%) and headache (5%).
"Lilly is working to empower people with skin-related diseases, such as atopic dermatitis, to live their lives to the fullest potential," said Lotus Mallbris, M.D., Ph.D., vice president of global immunology development and medical affairs at Lilly. "We recognize the critical need for more options for people whose disease cannot be controlled with topicals. We look forward to seeing full results from our broader Phase 3 program and advancing lebrikizumab worldwide."
Lilly recently announced 16-week data from the ongoing ADvocate studies, and an encore presentation of results was presented at RAD 2022. Additionally, longer term data from the ADvocate studies will be disclosed in coming months.
"These results are a further step in our commitment to deliver innovative therapies that make a meaningful difference to patients. We look forward to announcing exciting new milestones in the months to come," commented Karl Ziegelbauer, Ph.D., Almirall S.A.'s Chief Scientific Officer.
Lilly has exclusive rights for development and commercialization of lebrikizumab in the United States and the rest of the world outside Europe. Almirall has licensed the rights to develop and commercialize lebrikizumab for the treatment of dermatology indications, including AD, in Europe.
*EASI=Eczema Area and Severity Index, EASI-75=75 percent reduction in EASI from baseline to Week 16
About ADhere and the Phase 3 Program
ADhere is a 16-week randomized, double-blind, placebo-controlled, parallel-group, global, Phase 3 study to evaluate the efficacy and safety of lebrikizumab in combination with TCS initiated in 211 adult and adolescent patients (aged 12 to less than 18 years of age and weighing at least 40 kg) with moderate-to-severe AD. In the study, patients' AD symptoms were inadequately controlled by TCS with or without topical calcineurin inhibitors (TCI). The study was designed to be more reflective of clinical practice and patients were provided with mid-potency TCS (triamcinolone acetonide 0.1% cream), and low-potency TCS (hydrocortisone 1% cream, for use on sensitive skin areas) which could be tapered, stopped or resumed at the patient's discretion.
The primary endpoints were measured by an Investigator Global Assessment (IGA) score of clear (0) or almost clear (1) skin with a reduction from baseline and at least 75 percent change in baseline in the Eczema Area and Severity Index (EASI-75) score at 16 weeks. EASI measures extent and severity of the disease. Key secondary endpoints were measured by EASI, the Pruritus Numeric Rating Scale, Sleep-Loss due to Pruritus and the Dermatology Life Quality Index.
The U.S. Food and Drug Administration (FDA) granted lebrikizumab Fast Track designation in AD in December 2019. The lebrikizumab Phase 3 program consists of five key global studies including two monotherapy studies (ADvocate 1 and 2), a combination study (ADhere), as well as long-term extension (ADjoin) and adolescent open label (ADore) studies.
About Lebrikizumab
Lebrikizumab is a novel, investigational, monoclonal antibody designed to bind IL-13 with high affinity to specifically prevent the formation of the IL-13Rα1/IL-4Rα heterodimer complex and subsequent signaling, thereby inhibiting the biological effects of IL-13 in a targeted and efficient fashion. IL-13 is the central pathogenic mediator of AD, promoting type 2 inflammation that drives skin barrier dysfunction, itch, skin thickening and infection.6-8
To learn more, visit Lilly.com and Lilly.com/newsroom or follow us on Facebook, Instagram, Twitter and LinkedIn.
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SOURCE Eli Lilly and Company
Lilly atopic dermatitis candidate lebrikizumab shows improvement in disease severity
Apr. 11, 2022 12:45 PM ET
Eli Lilly and Company (LLY)TLPPF
By: Jonathan Block, SA News Editor
- A late-stage trial of Eli Lilly's (NYSE:LLY) lebrikizumab for atopic dermatitis showed significant improvement in measures of disease severity when combined with topical corticosteroids.
- https://seekingalpha.com/news/3822353-lilly-atopic-dermatitis-candidate-lebrikizumab-shows-improvement-in-disease-severity
- https://seekingalpha.com/symbol/LLY
- https://www.lilly.com/
DUPIXENT® (DUPILUMAB)
April 7, 2022 at 12:59 AM EDT Back
DUPIXENT® (DUPILUMAB) APPROVED BY EUROPEAN COMMISSION FOR CHILDREN AGED 6 TO 11 YEARS WITH SEVERE ASTHMA WITH TYPE 2 INFLAMMATION
Approval based on Phase 3 data showing Dupixent significantly reduced severe asthma attacks and also improved lung function and health-related quality of life for children
Data reinforce well-established safety profile of Dupixent
TARRYTOWN, N.Y. and PARIS, April 7, 2022 /PRNewswire/ -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today announced that the European Commission (EC) has expanded the marketing authorization for Dupixent® (dupilumab) in the European Union. Dupixent is now also approved in children aged 6 to 11 years as an add-on maintenance treatment for severe asthma with type 2 inflammation characterized by raised blood eosinophils and/or raised fractional exhaled nitric oxide (FeNO), who are inadequately controlled with medium to high dose inhaled corticosteroids (ICS) plus another medicinal product for maintenance treatment.
"Today's approval in Europe recognizes the benefits of Dupixent in helping children living with the profound effects of severe asthma, including unpredictable asthma attacks, routine disruption to daily activities and the use of systemic steroids that can impede children's growth," said George D. Yancopoulos, M.D., Ph.D., President and Chief Scientific Officer at Regeneron. "Dupixent is the only treatment available that specifically blocks two key drivers of type 2 inflammation, IL-4 and IL-13, which our trials show plays a major role in childhood asthma, as well as in related conditions such as chronic rhinosinusitis with nasal polyposis and the often co-morbid condition, atopic dermatitis. In clinical trials, Dupixent significantly reduced asthma attacks, helped children breathe better and improved their health-related quality of life. We also remain committed to investigating Dupixent in other conditions where type 2 inflammation may significantly impact patients' lives, including eosinophilic esophagitis, prurigo nodularis and chronic spontaneous urticaria."
Asthma is one of the most common chronic diseases in children. Up to 85% of children with asthma may have type 2 inflammation and are more likely to have higher disease burden. Despite treatment with current standard-of-care ICS and bronchodilators, these children may continue to experience serious symptoms such as coughing, wheezing and difficulty breathing. Severe asthma may impact children's developing airways and cause potentially life-threatening exacerbations. Children with severe asthma also may require the use of multiple courses of systemic corticosteroids that carry significant risks. Uncontrolled severe asthma can interfere with day-to-day activities, like sleeping, attending school and playing sports.
Dupixent, which was invented using Regeneron's proprietary VelocImmune® technology, is a fully human monoclonal antibody that inhibits the signaling of the interleukin-4 (IL-4) and interleukin-13 (IL-13) pathways and is not an immunosuppressant. By demonstrating significant clinical benefit together with a decrease in type 2 inflammation following IL-4 and IL-13 blockade with Dupixent, the Dupixent Phase 3 clinical program has established that IL-4 and IL-13 are key drivers of the type 2 inflammation that plays a major role in multiple related and often co-morbid diseases for which Dupixent is approved including asthma, atopic dermatitis and chronic rhinosinusitis with nasal polyposis (CRSwNP), as well as investigational diseases such as eosinophilic esophagitis and prurigo nodularis, which have been studied in Phase 3 trials.
"We are excited to bring the well-established safety and efficacy profile of Dupixent to even younger patients living with uncontrolled severe asthma in Europe. In addition to greatly reducing severe asthma attacks and improving lung function, patients in our clinical trials also reduced their oral corticosteroid use. This is particularly meaningful as these are medicines that can carry significant safety risks if used long term," said Naimish Patel, M.D. Head of Global Development, Immunology and Inflammation at Sanofi. "This approval underscores our continued commitment to bringing Dupixent to as many patients as possible suffering from the negative effects of severe asthma with the hope of improving their quality of life."
The EC decision is based on pivotal data from the Phase 3 VOYAGE trial evaluating the efficacy and safety of Dupixent combined with standard-of-care asthma therapy in 408 children with uncontrolled moderate-to-severe asthma.
Two pre-specified populations with evidence of type 2 inflammation were evaluated for the primary analysis: 1) patients with baseline blood eosinophils (EOS) ≥300 cells/μl (n=259) and 2) patients with either baseline FeNO ≥20 parts per billion (ppb) or baseline blood EOS ≥150 cells/μl (n=350). Patients who added Dupixent to standard-of-care in these two groups, respectively, experienced:
- Substantially reduced rates of severe asthma attacks, with a 65% and 59% average reduction over one year compared to placebo (0.24 and 0.31 events per year for Dupixent vs. 0.67 and 0.75 for placebo, respectively).
- Improved lung function observed as early as two weeks and sustained for up to 52 weeks, measured by percent predicted FEV1 (FEV1pp).
- At 12 weeks, patients taking Dupixent improved their lung function by 5.32 and 5.21 percentage points compared to placebo, respectively.
- Improved asthma control, with 81% and 79% of patients reporting a clinically meaningful improvement at 24 weeks, based on disease symptoms and impact compared to 64% and 69% of placebo patients, respectively.
- Improved health-related quality of life, with 73% and 73% of patients reporting a clinically meaningful improvement at 24 weeks, compared to 63% and 65% of placebo patients, respectively.
- Reduced systemic corticosteroid use by an average of 66% and 59% over one year compared to placebo (0.27 and 0.35 courses per year for Dupixent vs. 0.81 and 0.86 for placebo, respectively).
The safety results from the trial were generally consistent with the known safety profile of Dupixent in patients aged 12 years and older with uncontrolled moderate-to-severe asthma. The overall rates of adverse events were 83% for Dupixent and 80% for placebo. Adverse events that were more commonly observed with Dupixent compared to placebo included injection site reactions (18% Dupixent, 13% placebo), viral upper respiratory tract infections (12% Dupixent, 10% placebo) and eosinophilia (7% Dupixent, 1% placebo). Helminth infections were also more commonly observed with Dupixent in patients aged 6 to 11 years and were reported in 2% of Dupixent patients and 0% of placebo patients.
About the LIBERTY ASTHMA VOYAGE Trial
The Phase 3 randomized, double-blind, placebo-controlled trial evaluated the efficacy and safety of Dupixent (100 mg or 200 mg every two weeks, based on weight tier) combined with standard-of-care asthma therapy in 408 children aged 6 to 11 years with uncontrolled moderate-to-severe asthma. More than 90% of children in the trial had at least one concurrent atopic medical condition such as allergic rhinitis and atopic dermatitis.
The primary endpoint was the annualized rate of severe asthma exacerbations over one year, and the key secondary endpoint was the change from baseline in percentage of predicted pre-bronchodilator FEV1 (FEV1pp) at week 12. The FEV1pp seeks to evaluate a patient's change in lung function compared to their predicted lung function based on age, height, sex and ethnicity to account for children's growing lung capacity at different stages of development. Additional secondary endpoints included responder rates for asthma control as measured by a ≥0.5 improvement on the Asthma Control Questionnaire-7 Interviewer Administered (ACQ-7-IA; 7-point scale) and health-related quality of life as measured by a ≥0.5 improvement on the Pediatric Asthma Quality of Life Questionnaire with Standardized Activities-Interviewer Administered (PAQLQ(S)-IA; 7-point scale).
About Dupixent
Dupixent is also approved in Europe, U.S., Japan and other countries around the world for use in certain patients with asthma, specific patients with moderate-to-severe atopic dermatitis as well as CRSwNP in different age populations. Dupixent is also approved in one or more of these indications in more than 60 countries around the world, and more than 400,000 patients have been treated globally.
Dupixent is an injection under the skin (subcutaneous injection) at different injection sites. In the EU for pediatric patients aged 6 to 11 years, Dupixent dosing is based on weight tier (100 mg every two weeks or 300 mg every four weeks for children ≥15 to <30 kg, 200 mg every two weeks or 300 mg every four weeks for children ≥30 to <60 kg and 200 mg every two weeks for children ≥60 kg) and is supplied as a pre-filled syringe. It is also available as a pre-filled pen for adolescents (12 to 17 years) and adults at 200 and 300 mg doses. Dupixent is intended for use under the guidance of a healthcare professional and can be given in a clinic or at home by self-administration after training by a healthcare professional. In children younger than 12 years of age, Dupixent should be administered by a caregiver if given at home.
U.S. Indications
DUPIXENT is a prescription medicine used:
- to treat adults and children 6 years of age and older with moderate-to-severe atopic dermatitis (eczema) that is not well controlled with prescription therapies used on the skin (topical), or who cannot use topical therapies. DUPIXENT can be used with or without topical corticosteroids. It is not known if DUPIXENT is safe and effective in children with atopic dermatitis under 6 years of age.
- with other asthma medicines for the maintenance treatment of moderate-to-severe eosinophilic or oral steroid dependent asthma in adults and children 6 years of age and older whose asthma is not controlled with their current asthma medicines. DUPIXENT helps prevent severe asthma attacks (exacerbations) and can improve your breathing. DUPIXENT may also help reduce the amount of oral corticosteroids you need while preventing severe asthma attacks and improving your breathing. DUPIXENT is not used to treat sudden breathing problems. It is not known if DUPIXENT is safe and effective in children with asthma under 6 years of age.
- with other medicines for the maintenance treatment of chronic rhinosinusitis with nasal polyposis (CRSwNP) in adults whose disease is not controlled. It is not known if DUPIXENT is safe and effective in children with chronic rhinosinusitis with nasal polyposis under 18 years of age.
Please see accompanying full Prescribing Information including Patient Information.
For additional information about the company, please visit www.regeneron.com or follow @Regeneron on Twitter.
View original content:https://www.prnewswire.com/news-releases/dupixent-dupilumab-approved-by-european-commission-for-children-aged-6-to-11-years-with-severe-asthma-with-type-2-inflammation-301519649.html
SOURCE Regeneron Pharmaceuticals, Inc.
Regeneron, Sanofi win EU approval for Dupixent in younger children with asthma
Apr. 07, 2022 9:27 AM ET
Regeneron Pharmaceuticals, Inc. (REGN), SNY
By: Dulan Lokuwithana, SA News Editor
- The European Union (EU) has expanded the approval of Dupixent, allowing its use as an add-on maintenance treatment for severe asthma in certain patients aged 6 to 11 years, the developers, Regeneron (NASDAQ:REGN) and Sanofi (NASDAQ:SNY) announced on Thursday.
- https://seekingalpha.com/news/3821670-regeneron-sanofi-win-eu-nod-for-dupixent-in-younger-children-with-asthma
- https://seekingalpha.com/symbol/SNY
- https://seekingalpha.com/symbol/REGN
- https://www.regeneron.com/
- https://www.dupixent.com/
BAVENCIO® (avelumab)
NICE reverses stance on Merck’s Bavencio for bladder cancer
Phil Taylor April 7, 2022
Last year, NICE said that Merck KGaA/Pfizer’s Bavencio was too expensive for routine in NHS use as a first-line maintenance treatment for bladder cancer, even though the drug is the only cancer immunotherapy approved for this use.
Now, the cost-effectiveness agency has relented – with the help of an additional discount from the company – clearing the way for Bavencio (avelumab) to be used in around 800 eligible people with locally advanced or metastatic urothelial carcinoma, the most common form of bladder cancer, who are progression-free following platinum-based chemotherapy.
The change in stance also followed “a successful appeal by stakeholders” that included further evidence from the pharma company of Bavencio’s efficacy in this setting, said NICE, which rejected that use in draft guidance issued in May 2021 – both for routine use and via the Cancer Drugs Fund (CDF) – saying it was not a cost-effective use of NHS resources.
From today, the PD-L1 inhibitor can be prescribed to eligible patients in England, Wales and Northern Ireland, according to Pfizer. Bavencio was approved for NHS use as a first-line maintenance therapy for bladder cancer in Scotland last August, creating unequal access to the drug across the UK.
“Clinical trial evidence showed that avelumab increases both how long people live and the length of time before their disease gets worse compared with best supportive care alone,” said NICE in a statement.
Pfizer-Merck KGaA's Bavencio gets UK's NICE backing for use in bladder cancer
Apr. 08, 2022 4:48 AM ET
Pfizer Inc. (PFE), MKGAF, MKKGY
By: Ravikash, SA News Editor
U.K.'s National Institute for Health and Care Excellence (NICE) recommended the use of Pfizer (NYSE:PFE) and Merck KGaA's (OTCPK:MKGAF) (OTCPK:MKKGY) Bavencio for certain patients with bladder cancer, following a rejection last year.
- https://seekingalpha.com/news/3821939-pfizer-merck-kgaas-bavencio-gets-uks-nice-backing-for-use-in-bladder-cancer
- https://www.bavencio.com/hcp
- https://seekingalpha.com/symbol/MKKGY
- https://seekingalpha.com/symbol/PFE
NATIONAL INSTITUTE FOR HEALTH AND CARE EXCELLENCE Final appraisal document Avelumab for maintenance treatment of locally advanced or metastatic urothelial cancer after platinum-based chemotherapy 1 Recommendations
https://www.nice.org.uk/guidance/gid-ta10624/documents/final-appraisal-determination-document-2
BAVENCIO® (avelumab)
Indications
Metastatic Merkel cell carcinoma (MCC)
- BAVENCIO® (avelumab) is indicated for the treatment of adults and pediatric patients 12 years and older with metastatic MCC. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials
First-line maintenance treatment of urothelial carcinoma (UC)
- BAVENCIO is indicated for the maintenance treatment of patients with locally advanced or metastatic UC that has not progressed with first-line platinum-containing chemotherapy
Previously treated urothelial carcinoma (UC)
- BAVENCIO is indicated for the treatment of patients with locally advanced or metastatic UC who have disease progression during or following platinum-containing chemotherapy, or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy
Please see full Prescribing Information and Medication Guide.
Tislelizumab
Novartis announces anti-PD-1 tislelizumab accepted by EMA for regulatory review in esophageal and lung cancers
Apr 06, 2022
- European Medicines Agency validated MAAs for tislelizumab in multiple non-small cell lung cancer and esophageal squamous cell carcinoma indications
- Submissions based on data from Phase III RATIONALE 302, 303, 304 and 307 trials, in which tislelizumab monotherapy and in combination with chemotherapy demonstrated significant clinical benefits versus chemotherapy in multiple cancer types and lines of therapy
- EMA filings follow US FDA filing acceptance for tislelizumab in esophageal cancer
- Novartis is evaluating tislelizumab in 14 pivotal clinical trials in a broad array of solid tumors, with more than 8,800 patients enrolled to date in 35 countries
Basel, April 6, 2022 — Novartis today announced that the European Medicines Agency (EMA) validated Marketing Authorization Applications (MAAs) for the immune checkpoint inhibitor tislelizumab for adults with:
- Locally advanced or metastatic, squamous or non-squamous non-small cell lung cancer (NSCLC) as first-line treatment in combination with chemotherapy,
- Locally advanced or metastatic NSCLC as monotherapy after prior chemotherapy, and
- Unresectable, recurrent, locally advanced or metastatic esophageal squamous cell carcinoma (ESCC) as monotherapy after prior chemotherapy.
Tislelizumab is a humanized IgG4 anti-PD-1 monoclonal antibody being developed both as a monotherapy and in combination with other therapies.1
"This is an important step toward expanding treatment options for cancer patients in Europe, and builds on the US FDA filing acceptance for tislelizumab in esophageal cancer,” said Jeff Legos, Executive Vice President, Global Head of Oncology & Hematology Development. “We look forward to working with the EMA to make tislelizumab available to people with these aggressive cancers, while continuing to expand our development program to investigate the potential of novel, synergistic combinations.”
The MAAs include data from the pivotal Phase III RATIONALE 302 trial, in which tislelizumab demonstrated a significant improvement in overall survival versus chemotherapy as treatment for people with ESCC who had received prior chemotherapy. The submissions also include data from the pivotal Phase III RATIONALE 303 trial, which showed tislelizumab significantly improved overall survival versus chemotherapy in people with NSCLC after treatment with chemotherapy, and from RATIONALE 304 and 307, which showed tislelizumab plus chemotherapy significantly improved progression-free survival versus chemotherapy in people with untreated squamous and non-squamous NSCLC.
Lung cancer is one of the most common cancers worldwide, accounting for more than 2 million new cases diagnosed each year.2 More people die of lung cancer every year than any other cancer.3 ESCC is the most common type of esophageal cancer globally, with an estimated 604,000 new cases and 544,000 deaths from esophageal cancer internationally in 2020.3
About RATIONALE Trials
RATIONALE 302 (NCT03430843) is a multi-regional, open-label, randomized Phase III study of tislelizumab versus chemotherapy in patients with advanced unresectable/metastatic esophageal squamous cell carcinoma who had received prior systemic therapy. Approximately 513 patients were randomized 1:1 to receive tislelizumab or investigator chosen chemotherapy. The primary endpoint is overall survival (OS); the key secondary endpoint was OS in the PD-L1 positive population. Other secondary endpoints include progression-free survival (PFS), objective response rate (ORR), duration of response (DoR), health-related quality of life measures and safety.
RATIONALE 303 (NCT03358875) is a multi-regional, open-label, multicenter, randomized Phase III study of tislelizumab versus chemotherapy in patients with locally advanced or metastatic NSCLC who have progressed on a prior platinum-containing regimen. Approximately 805 patients were randomized 1:1 to receive tislelizumab or chemotherapy. The co-primary endpoints are OS in all patients and OS in PD-L1 positive patients. Secondary endpoints include PFS, ORR, DoR, health-related quality of life measures and safety.
RATIONALE 304 (NCT03663205) is an open-label, multicenter, randomized Phase III study of tislelizumab plus chemotherapy versus chemotherapy alone in patients with untreated advanced non-squamous NSCLC. Approximately 334 patients were randomized 1:1 to receive either tislelizumab plus chemotherapy or chemotherapy. The primary endpoint is PFS. Secondary endpoints include OS, ORR, DoR, health-related quality of life measures and safety.
RATIONALE 307 (NCT03594747) is an open-label, multicenter, randomized Phase III study of tislelizumab plus chemotherapy versus chemotherapy in patients with untreated advanced squamous NSCLC. Approximately 360 patients were randomized 1:1:1 to receive tislelizumab plus paclitaxel, tislelizumab plus nab-paclitaxel, or chemotherapy alone. The primary endpoint is PFS. Secondary endpoints include OS, ORR, DoR, health-related quality of life measures and safety.
About Tislelizumab
Novartis is evaluating tislelizumab, a uniquely designed anti-PD-1 monoclonal antibody, in a global clinical development program consisting of 14 pivotal clinical trials across a broad array of solid tumors, with more than 8,800 patients enrolled to date in 35 countries. Novartis four distinct therapeutic platforms (immunotherapy, radioligand therapy, cell and gene therapy, targeted therapy) offer a unique opportunity to study tislelizumab in differentiated, potentially synergistic combinations across our pipeline and portfolio of market compounds.
Novartis has the rights to develop, manufacture and commercialize tislelizumab in North America, Europe and Japan through a collaboration and license agreement with BeiGene.
Find out more at https://www.novartis.com.
Novartis, BeiGene's tislelizumab gets EMA review in esophageal, lung cancers
Apr. 06, 2022 5:35 AM ET BeiGene, Ltd. (BGNE), NVS By: Ravikash, SA News Editor1 Comment
The European Medicines Agency accepted to review Novartis (NYSE:NVS) and BeiGene's (NASDAQ:BGNE) tislelizumab for patients with advanced or metastatic esophageal squamous cell carcinoma (ESCC) after prior systemic chemotherapy.
https://seekingalpha.com/symbol/BGNE
https://seekingalpha.com/symbol/NVS
https://www.beigene.com/en-us/science-and-product-portfolio/pipeline/tislelizumab
Impella 5.5 with SmartAssist
First Patient in Japan Treated with Impella 5.5 with SmartAssist
5,000 Patients Treated Globally
DANVERS, Mass.--(BUSINESS WIRE)-- The versatility and innovation of Abiomed’s (NASDAQ: ABMD) Impella 5.5 with SmartAssist, a minimally-invasive forward flow heart pump, is leading to continued adoption of the game-changing technology by surgeons around the world. Today, Abiomed announces the first patient in Japan has been treated successfully with Impella 5.5 with SmartAssist. Additionally, the number of patients treated globally has surpassed 5,000.
Surgeons at Osaka Police Hospital view a fluoroscopy image showing Impella 5.5 with SmartAssist pumping in the patient’s heart during the first procedure in Japan. (Photo: Business Wire)
The first Impella 5.5 with SmartAssist procedure in Japan took place at Osaka Police Hospital when an 82-year-old man was treated for cardiogenic shock. “Our facility is proud to be the first in Japan to treat patients using Impella 5.5 with SmartAssist,” said Yoshiki Sawa, MD, PhD, director at Osaka Police Hospital. “We are excited to utilize this innovative technology and look forward to providing a minimally invasive surgical option for our acute heart failure patients.”
The milestone 5,000th procedure was performed by Tamer Attia, MD, at Emory University Hospital in Atlanta on a 41-year-old woman with cardiomyopathy. “Impella 5.5 with SmartAssist has completely changed how we are able to support our cardiogenic shock and cardiomyopathy patients,” said Mani Daneshmand, MD, chief of the section of thoracic transplant and mechanical circulatory support surgery at Emory University Hospital. “Given the safety and efficacy of Impella 5.5 with SmartAssist, Dr. Attia and his team are able to treat patients who otherwise would not have had other options for heart recovery.”
Impella 5.5 with SmartAssist is:
- Minimally invasive, eliminating the need for a sternotomy or coring of the left ventricle;
- Designed for heart surgeons, implanted via the axillary artery or the anterior aorta;
- Forward flow, to provide the patient with coronary flow and renal perfusion;
- Fully unloading, to reduce the heart’s oxygen demand and work; and
- Equipped with SmartAssist, designed to provide weaning algorithms to optimize survival and native heart recovery.
Additionally, more than 90% of patients are securely monitored in the cloud via Impella Connect, giving providers access to Impella status data 24/7.
Impella 5.5 with SmartAssist, which was granted the highest level of approval for safety and efficacy by the U.S. Food and Drug Administration (FDA) in 2019, is now being used in more than 350 centers across the U.S. for indications that include AMI cardiogenic shock, cardiomyopathy and post-cardiotomy cardiogenic shock. Historically, the cardiogenic shock survival rate has been approximately 50%. Published in Innovations, a study of 200 patients treated with Impella 5.5 with SmartAssist at 42 medical centers in the United States demonstrates a 74% survival to explant, with 58% of those patients achieving native heart recovery.
For additional information, please visit: www.abiomed.com.
ABOUT IMPELLA HEART PUMPS
The Impella 2.5® and Impella CP® devices are US FDA approved to treat certain advanced heart failure patients undergoing elective and urgent percutaneous coronary interventions (PCI), such as stenting or balloon angioplasty, to reopen blocked coronary arteries.
Impella 2.5, Impella CP, Impella CP with SmartAssist®, Impella 5.0®, Impella LD®, and Impella 5.5® with SmartAssist® are US FDA approved to treat heart attack or cardiomyopathy patients in cardiogenic shock and have the unique ability to enable native heart recovery, allowing patients to return home with their own heart.
https://www.heartrecovery.com/products-and-services/impella/impella-55-with-smartassist
Abiomed Impella 5.5 with SmartAssist used for 1st time in Japan
Apr. 06, 2022 9:14 AM ET
By: Ravikash, SA News Editor
- Abiomed (NASDAQ:ABMD) said the first patient in Japan was treated successfully with Impella 5.5 with SmartAssist.
- https://seekingalpha.com/news/3821126-abiomed-impella-55-with-smartassist-used-for-1st-time-in-japan
- https://seekingalpha.com/symbol/ABMD
- https://www.heartrecovery.com/products-and-services/impella/impella-55-with-smartassist
Ultomiris (ravulizumab-cwvz)
Ultomiris demonstrated sustained improvements in functional activities and quality of life in adults with generalised myasthenia gravis through 60 weeks
PUBLISHED6 April 2022
6 April 2022 07:00 BST
Analysis of Phase III CHAMPION-MG trial open-label extension adds to growing body of safety and efficacy data for Ultomiris in generalised myasthenia gravis
Patients who transitioned from placebo to Ultomiris showed rapid and sustained response, reinforcing clinical benefit of C5 inhibition
New, prolonged follow-up results from the Phase III CHAMPION-MG trial open-label extension (OLE) showed that Ultomiris (ravulizumab-cwvz) demonstrated long-term efficacy in adults with anti-acetylcholine receptor (AChR) antibody-positive generalised myasthenia gravis (gMG), with improvements in activities of daily living, muscle strength and quality of life, sustained through 60 weeks.1 Ultomiris was also well tolerated throughout this analysis.
Results from the trial were presented on 5 April at the 2022 American Academy of Neurology (AAN) Annual Meeting.
gMG is a rare, debilitating, chronic, autoimmune neuromuscular disease that leads to a loss of muscle function and severe weakness.2 Symptoms of gMG may include disabling fatigue, slurred speech, difficulty swallowing and eating, double or blurred vision, immobility requiring assistance, shortness of breath, and episodes of respiratory failure.3-5
Professor James F. Howard, Jr, MD, Department of Neurology at The University of North Carolina School of Medicine and lead primary investigator in the CHAMPION-MG trial said: “gMG is a complex, devastating disease, disrupting many aspects of daily living, and helping patients improve muscle strength and function should be essential to any treatment plan. These results reinforce that C5 inhibition with predictable dosing is an important treatment option which provides sustained improvement of functional activities.”
Gianluca Pirozzi, MD, PhD, Senior Vice President, Head of Development and Safety, Alexion, said: “Alexion has pioneered the research of complement inhibition as a treatment approach for rare diseases, and we are continuing to innovate to benefit as many patients as possible. These data are encouraging because they suggest Ultomiris has the potential to help a broader range of gMG patients, including those with milder symptoms, regain control of their lives and experience sustained clinical benefit through 60 weeks. We are deeply grateful for continued input and collaboration from the gMG community.”
Upon completion of the randomised control period (RCP) of the CHAMPION-MG trial, 99.4% of participants (n=161) entered the OLE, during which all patients received Ultomiris. At the time of data cut off, 113 patients had reached 60 weeks. Efficacy analysis included all patients who received ≥1 dose of Ultomiris in the OLE.1
Ultomiris demonstrated statistically significant improvements from baseline (defined as initiation of Ultomiris therapy) in measures of functional activity, muscle strength and quality of life at 60 weeks of the OLE, including Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score (-4.0 [95% CI -4.8, -3.1], p<0.0001). Additionally, patients transitioning from placebo (n=83) showed rapid response at a similar magnitude and time course as those who received Ultomiris during the RCP.1
CHAMPION-MG
The global Phase III randomised, double-blind, placebo-controlled, multicentre 26-week trial evaluated the safety and efficacy of Ultomiris in adults with gMG who were not previously treated with a complement inhibitor medicine. The trial enrolled 175 patients across North America, Europe, Asia-Pacific and Japan. Participants were required to have a confirmed myasthenia gravis diagnosis at least six months prior to the screening visit with a positive serologic test for anti-AChR antibodies, MG-ADL total score of at least 6 at trial entry and Myasthenia Gravis Foundation of America Clinical Classification Class II to IV at screening. There was no requirement for prior treatment failure, and patients could stay on stable standard of care medicines, with a few exceptions, for the duration of the randomised control period.10
Patients were randomised 1:1 to receive Ultomiris or placebo for a total of 26 weeks. Patients received a single weight-based loading dose on Day 1, followed by regular weight-based maintenance dosing beginning on Day 15, every eight weeks. The primary endpoint of change from baseline in the MG-ADL total score at Week 26 was assessed along with multiple secondary endpoints evaluating improvement in disease-related and quality-of-life measures.
Patients who completed the randomised control period were eligible to continue into an open-label extension period evaluating the safety and efficacy of Ultomiris, which is ongoing.
Ultomiris
Ultomiris (ravulizumab-cwvz), the first and only long-acting C5 complement inhibitor, offers immediate, complete and sustained complement inhibition. The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. When activated in an uncontrolled manner, the complement cascade over-responds, leading the body to attack its own healthy cells. Ultomiris is administered intravenously every eight weeks in adult patients, following a loading dose.
Ultomiris is approved in the US, EU and Japan for the treatment of certain adults and children with paroxysmal nocturnal haemoglobinuria (PNH) based on the ALXN1210-PNH-302 and ALXN1210-PNH-304 Phase III trials.
Additionally, Ultomiris is approved in the US, EU and Japan for certain adults and children with atypical haemolytic uraemic syndrome (aHUS) based on the ALXN1210-aHUS-311 and ALXN1210-aHUS-312 Phase III trials.
As part of a broad development programme, Ultomiris is being assessed for the treatment of additional haematology and neurology indications.
Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca.
Alexion
Alexion, AstraZeneca Rare Disease, is the group within AstraZeneca focused on rare diseases, created following the 2021 acquisition of Alexion Pharmaceuticals, Inc. As a leader in rare diseases for nearly 30 years, Alexion is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialisation of life-changing medicines. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on haematology, nephrology, neurology, metabolic disorders, cardiology and ophthalmology. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries.
AstraZeneca's Ultomiris sustains function improvement over 1 year in muscle disorder patients
Apr. 06, 2022 6:40 AM ET
By: Ravikash, SA News Editor
- AstraZeneca (NASDAQ:AZN) said Ultomiris helped improve activities of daily living, muscle strength and quality of life through 60 weeks in patients with generalised myasthenia gravis (gMG).
- https://seekingalpha.com/news/3821032-astrazenecas-ultomiris-sustains-function-improvement-over-1-year-in-muscle-disorder-patients
- https://seekingalpha.com/symbol/AZN
- https://ultomiris.com/
Pemazyre® (pemigatinib)
Innovent Announces the Approval of Pemazyre® (pemigatinib) by the NMPA for the Treatment of Adults with Locally Advanced or Metastatic Cholangiocarcinoma with A FGFR2 Fusion or Rearrangement As Confirmed By A Validated Diagnostic Test That Have Progressed
Published on: Apr 6th, 2022
SAN FRANCISCO, U.S. and SUZHOU, China, April 06, 2022 — Innovent Biologics, Inc. (Innovent) (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high-quality medicines for the treatment of cancer, metabolic, autoimmune and other major diseases announced that the National Medical Products Administration (NMPA) has approved Pemazyre® (pemigatinib) for the treatment of adults with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement as confirmed by a validated diagnostic test that have progressed after at least one prior line of systemic therapy.
Pemazyre®, discovered by Incyte and licensed to Innovent for development and commercialization in Mainland China, Hong Kong, Macau and Taiwan markets, is the first selective tyrosine kinase inhibitor approved for the treatment of cholangiocarcinoma, a type of biliary tract cancer, in China, representing a new milestone following its approval in Hong Kong market in January 2022, and in the Taiwan market in June 2021.
The approval in China was based on two clinical studies. One is the FIGHT-202 study, which is a Phase 2, multi-center, open-label, single-arm study (NCT02924376) evaluating the safety and efficacy of pemigatinib in adult (age ≥18 years) patients with previously treated, locally advanced or metastatic cholangiocarcinoma with documented FGFR2 fusion or rearrangement. The other study was a bridging study (CIBI375A201, NCT04256980) conducted in China evaluating the safety and efficacy of pemigatinib in Chinese cholangiocarcinoma patients. The primary end point was overall response rate (ORR) evaluated by an independent radiological review committee (IRRC) per RECIST V1.1.
In the FIGHT-202 study[1], as data cut of date (April 7th,2020), a total of 108 subjects with FGFR2 fusion/rearrangement were enrolled and orally received pemigatinib 13.5mg per day（Q3W 2 weeks on/1 week off）, the IRRC-confirmed ORR was 37.0% (95% CI: 27.94%, 46.86%), including 4 complete responses(CR). The median duration of response (DOR) was 8.08 months with responses lasting ≥ 6 months in 26 of the 40 (66.0%) responding patients and ≥ 12 months in 15 (37.5%) patients. In study CIBI375A201, as of data cut-off date (January 29th, 2021), among 30 efficacy evaluable Chinese subjects enrolled, the IRRC-confirmed ORR was 50%（95% CI: 31.3%,68.7%）. The overall safety profiles of FIGHT-202 and the study CIBI375A201 are similar and the majority of the adverse events were grade 1 or 2 per CTCAE V5.0. Pemigatinib was generally well tolerated in Chinese patients with cholangiocarcinoma.
Professor Jian Zhou in Zhong Shan Hospital Fudan University stated that: “Cholangiocarcinoma is the second most common malignancy originated in the liver with a high incidence rate in Asia. The disease is usually not diagnosed until it has already developed into an advanced unresectable and/or metastatic stage. There are limited treatment options currently, which call for innovative drugs.”
“The approval of Pemazyre® by the NMPA, following the approval in Hong Kong and Taiwan market earlier in Greater China market, represents that Innovent has further broadened our product market coverage. At the same time, the approval of Pemazyre® also provided a new treatment option for cholangiocarcinoma patients in China.” Dr. Yongjun Liu, President of Innovent, stated: “Data from previous clinical trials of Pemazyre® in participants with advanced cholangiocarcinoma with FGFR2 fusion that have progressed after at least one prior line of systemic therapy has shown satisfactory safety results and also revealed encouraging efficacy signals. The approval further enhanced our confidence and interests in conducting in-depth clinical development of pemigatinib in more potential indications, including the ongoing global clinical trial (including China) evaluating pemigatinib as a first-line therapy for cholangiocarcinoma with FGFR2 fusion.”
About Advanced Cholangiocarcinoma and FGFR2 Rearrangement
Cholangiocarcinoma is a malignant tumour originated from biliary epithelium cells and it is categorized as intrahepatic or extrahepatic based on anatomical location of origin. The incidence of cholangiocarcinoma has been increasing progressively over the past decade. Surgery is the first line treatment for patients with resectable disease. However, most cholangiocarcinomas have been in advanced and/or metastatic status at diagnosis and lost the chance for surgical resection. The treatment options for patients who relapse after surgery or have advanced / metastatic disease are limited and the recommended therapy method is systemic chemotherapy with gemcitabine plus cisplatin, which has a medium overall survival of less than a year.
Aberrant signaling through FGFR resulting from gene amplification or mutation, chromosomal translocation, and ligand-dependent activation of the receptors has been demonstrated in multiple types of human cancers. Fibroblast growth factor receptor signaling contributes to the development of malignancies by promoting tumor cell proliferation, survival, migration, and angiogenesis. Results from early clinical studies of selective FGFR inhibitors, including Pemazyre, have shown a tolerable safety profile for the class and preliminary signs of clinical benefit in participants with FGF/FGFR alterations.
About Pemazyre® (pemigatinib)
In April 2020, the U.S. Food and Drug Administration (FDA) approved Incyte’s Pemazyre® (pemigatinib), a selective, oral inhibitor of FGFR isoforms 1, 2 and 3, for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement as detected by an FDA-approved test. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
In Japan, Pemazyre® is approved for the treatment of patients with unresectable biliary tract cancer with a FGFR2 fusion gene, worsening after cancer chemotherapy. In Europe, Pemazyre® is approved for the treatment of adults with locally advanced or metastatic cholangiocarcinoma with a FGFR2 fusion or rearrangement that have progressed after at least one prior line of systemic therapy. Pemazyre® is marketed by Incyte in the United States, Europe and Japan.
In December 2018, Innovent and Incyte entered into a strategic collaboration for three clinical-stage product candidates discovered and developed by Incyte, including pemigatinib (FGFR1/2/3 inhibitor). Under the terms of the agreement, Innovent has received the rights to develop and commercialize the three assets in Mainland China, Hong Kong, Macau and Taiwan.
In March 2020, Innovent announced that the first patient was dosed in the pivotal registrational trial evaluating pemigatinib in patients with advanced cholangiocarcinoma in China.
In June 2021, Taiwan Food and Drug Administration (TFDA) approved Pemazyre® for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a FGFR2 fusion or rearrangement.
In January 2022, Hong Kong Department of Health (DH) approved Pemazyre® for the treatment of adults with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that have progressed after at least one prior line of systemic therapy.
In April 2022, the National Medical Products Administration (NMPA) of China approved Pemazyre® for the treatment of adults with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2（FGFR2） fusion or rearrangement as confirmed by a validated diagnostic test that have progressed after at least one prior line of systemic therapy.
Pemazyre® is a trademark of Incyte Corporation.
For more information, please visit: www.innoventbio.com. and www.linkedin.com/company/innovent-biologics/.
Incyte, Innovent's Pemazyre for bile duct cancer gets approval in China
Apr. 06, 2022 6:03 AM ET Innovent Biologics, Inc. (IVBIY), INCY By: Ravikash, SA News Editor
- Incyte (NASDAQ:INCY) and Innovent Biologics' (OTCPK:IVBIY) Pemazyre (pemigatinib) was approved in China for treating adults with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement as confirmed by a validated diagnostic test that have progressed after at least one prior line of systemic therapy.
- https://seekingalpha.com/news/3821022-incyte-innovents-pemazyre-for-bile-duct-cancer-gets-approval-in-china
- https://seekingalpha.com/symbol/IVBIY
- https://seekingalpha.com/symbol/INCY
- https://www.pemazyre.com/
INNOVENT BIOLOGICS, INC. (1801)
https://www.hkex.com.hk/Market-Data/Securities-Prices/Equities/Equities-Quote?sym=1801&sc_lang=en
biotecMAX™ Feb/Mar 2022 Insight
Opdivo (nivolumab)
Bristol Myers Squibb Receives European Commission Approval for Opdivo (nivolumab) as Adjuvant Treatment for Patients with Radically Resected, High-Risk Muscle-Invasive Urothelial Carcinoma with Tumor Cell PD-L1 Expression ≥1%
04/05/2022
CATEGORY: Corporate/Financial News
Approval based on Phase 3 CheckMate -274 trial results showing that adjuvant treatment with Opdivo significantly reduced patients’ risk of disease recurrence or death compared to placebo
Opdivo is now the first and only adjuvant immunotherapy option approved in this setting in the European Union
Decision marks the third approval for Opdivo in earlier stages of cancer in the European Union, following melanoma and esophageal/gastroesophageal junction cancer
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced that the European Commission (EC) has approved Opdivo (nivolumab) for the adjuvant treatment of adults with muscle-invasive urothelial carcinoma with tumor cell PD-L1 expression ≥1% who are at a high risk of recurrence after undergoing radical resection.With this EC decision, Opdivo becomes the first adjuvant immunotherapy option approved for patients in the European Union (EU) in this setting.
In the Phase 3 CheckMate -274 trial, Opdivo demonstrated a statistically significant and clinically meaningful improvement in disease-free survival (DFS) compared to placebo in both all-randomized patients and in patients whose tumor cells express PD-L1 ≥1%. The EC decision is based on the results in patients with tumor cell PD-L1 expression ≥1%, which showed a 47% reduction in the risk of disease recurrence or death with Opdivo vs. placebo (Hazard Ratio [HR] 0.53; 95% Confidence Interval [CI]: 0.38 to 0.75; p=0.0005), with median DFS not reached with Opdivo compared to 8.41 months with placebo. Opdivo was generally well tolerated, with a safety profile that was consistent with previously reported Opdivo studies in patients with solid tumors.
“For years, patients with muscle-invasive urothelial carcinoma have lived with the unfortunate reality that, despite being diagnosed early enough to have their cancer removed, around half will face disease recurrence, with few safe and effective treatment options available to help prevent this cycle,” said Fred Witjes, M.D., professor of oncological urology, Radboud Institute for Molecular Life Sciences. “With the approval of nivolumab, clinicians will now have an immunotherapy treatment option to offer certain patients after surgery that, in the CheckMate -274 clinical trial, significantly reduced the risk of disease recurrence or death. This approval has the potential to transform the way we treat muscle-invasive urothelial carcinoma for appropriate patients in the European Union.”
The EC approval allows for the use of Opdivo for the adjuvant treatment of adults with radically resected, high-risk muscle-invasive urothelial carcinoma whose tumor cells express PD-L1 ≥1%inthe 27 member states of the EU, as well as Iceland, Liechtenstein and Norway. In addition to the EU, Opdivo has received approvals based on the CheckMate -274 trial in seven countries, including indications regardless of PD-L1 expression levels in the United States and Japan, and further applications are under review with health authorities globally. Results from CheckMate -274 were first presented at the American Society of Clinical Oncology (ASCO) Genitourinary Symposium in February 2021 and published in the New England Journal of Medicine in June 2021.
“We are driven by the goal of advancing new treatment options that can help change the outlook for patients with cancer, whether they are diagnosed at an earlier stage or with metastatic disease,” said Dana Walker, M.D., M.S.C.E., vice president, development program lead, genitourinary cancers, Bristol Myers Squibb. “This approval for Opdivo gives us the opportunity to introduce a new post-surgery standard of care for certain patients with urothelial cancer, building on our legacy as the first company to bring immune checkpoint inhibitors to patients in the European Union in the adjuvant settings of melanoma and esophageal cancers. We look forward to working with European stakeholders to make Opdivo available to eligible patients as soon as possible.”
“With the second highest incidence of bladder cancer globally, Europe has had a pressing need for new treatment options for patients after surgery, who are managing the physical and mental impacts of surgery combined with the fear of their cancer returning,” said Alex Filicevas, executive director, World Bladder Cancer Patient Coalition. “We are pleased to see the approval of a new adjuvant treatment that has the potential to help reduce the risk of recurrence for patients. Additionally, undergoing surgical removal of the bladder for muscle-invasive bladder cancer is life-altering, making it all the more important that published data show nivolumab maintained quality of life compared to placebo. This positive decision may have the potential to give people with muscle-invasive urothelial carcinoma more good quality time with their loved ones.”
Opdivo has now been approved for the adjuvant treatment of three different carcinoma types in the EU: urothelial carcinoma, melanoma and esophageal/gastroesophageal junction cancer. In addition, the company has a broad development program in earlier stages of cancer that currently spans eight different tumor types across the neoadjuvant, adjuvant and peri-operative settings.
About CheckMate -274
CheckMate -274 is a Phase 3 randomized, double-blind, multi-center study evaluating Opdivo compared to placebo in patients with muscle-invasive urothelial carcinoma at a high risk of recurrence after radical resection. A total of 709 patients were randomized 1:1 to receive Opdivo 240 mg or placebo every two weeks for up to one year. The primary endpoints of the trial are disease-free survival (DFS) in all randomized patients (i.e., the intention-to-treat population) and in the subset of patients whose tumor cells express PD-L1 ≥1%. Key secondary endpoints include overall survival (OS), non-urothelial tract recurrence-free survival (NUTRFS) and disease-specific survival (DSS).
About Opdivo
Opdivo is a programmed death-1 (PD-1) immune checkpoint inhibitor that is designed to uniquely harness the body’s own immune system to help restore anti-tumor immune response. By harnessing the body’s own immune system to fight cancer, Opdivo has become an important treatment option across multiple cancers.
Opdivo’s leading global development program is based on Bristol Myers Squibb’s scientific expertise in the field of Immuno-Oncology, and includes a broad range of clinical trials across all phases, including Phase 3, in a variety of tumor types. To date, the Opdivo clinical development program has treated more than 35,000 patients. The Opdivo trials have contributed to gaining a deeper understanding of the potential role of biomarkers in patient care, particularly regarding how patients may benefit from Opdivo across the continuum of PD-L1 expression.
In July 2014, Opdivo was the first PD-1 immune checkpoint inhibitor to receive regulatory approval anywhere in the world. Opdivo is currently approved in more than 65 countries, including the United States, the European Union, Japan and China. In October 2015, the Company’s Opdivo and Yervoy combination regimen was the first Immuno-Oncology to receive regulatory approval for the treatment of metastatic melanoma and is currently approved in more than 50 countries, including the United States and the European Union.
U.S. FDA-Approved Indications
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of adult patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of adult patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of adult patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection.
OPDIVO® (nivolumab), in combination with platinum-doublet chemotherapy, is indicated as neoadjuvant treatment of adult patients with resectable (tumors ≥4 cm or node positive) non-small cell lung cancer (NSCLC).
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express PD-L1 (≥1%) as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab) and 2 cycles of platinum-doublet chemotherapy, is indicated for the first-line treatment of adult patients with metastatic or recurrent non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving OPDIVO.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with unresectable malignant pleural mesothelioma (MPM).
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with intermediate or poor risk advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab), in combination with cabozantinib, is indicated for the first-line treatment of adult patients with advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy.
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with classical Hodgkin lymphoma (cHL) that has relapsed or progressed after autologous hematopoietic stem cell transplantation (HSCT) and brentuximab vedotin or after 3 or more lines of systemic therapy that includes autologous HSCT. This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN) with disease progression on or after platinum-based therapy.
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
OPDIVO® (nivolumab), as a single agent, is indicated for the adjuvant treatment of adult patients with urothelial carcinoma (UC) who are at high risk of recurrence after undergoing radical resection of UC.
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of adult and pediatric (12 years and older) patients with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of adult patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) after prior fluoropyrimidine- and platinum-based chemotherapy.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of completely resected esophageal or gastroesophageal junction cancer with residual pathologic disease in adult patients who have received neoadjuvant chemoradiotherapy (CRT).
OPDIVO® (nivolumab), in combination with fluoropyrimidine- and platinum- containing chemotherapy, is indicated for the treatment of adult patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma.
For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.
Bristol Myers Squibb Receives European Commission Approval for Opdivo (nivolumab) with Chemotherapy as First-Line Treatment for Patients with...
04/05/2022CATEGORY:
Opdivo with chemotherapy demonstrated statistically significant and clinically meaningful improvement in overall survival compared to chemotherapy alone in this patient population; approval based on Phase 3 results from the CheckMate -648 trial
Opdivo plus Yervoy also received EC approval for the same indication
PRINCETON, N.J.--(BUSINESS WIRE)--
Bristol Myers Squibb Receives European Commission Approval for Opdivo (nivolumab) with Chemotherapy as First-Line Treatment for Patients with Unresectable Advanced, Recurrent or Metastatic Esophageal Squamous Cell Carcinoma with Tumor Cell PD-L1 Expression ≥ 1%
Bristol Myers Squibb (NYSE: BMY) today announced that the European Commission (EC) has approved Opdivo (nivolumab) in combination with fluoropyrimidine- and platinum-based chemotherapy for the first-line treatment of adult patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) with tumor cell PD-L1 expression ≥ 1%. The EC’s decision is based on results from the Phase 3 CheckMate -648 trial, in which Opdivo with chemotherapy demonstrated a statistically significant and clinically meaningful overall survival (OS) benefit compared to chemotherapy alone at the pre-specified interim analysis. The safety profile of Opdivo with chemotherapy was consistent with previously reported studies. Results from CheckMate -648 were presented at the American Society of Clinical Oncology (ASCO) Annual Meeting in June 2021.
“This approval is an important advancement for patients in the EU, especially given the highly aggressive nature of advanced ESCC,” said Ian M. Waxman, M.D., development lead, gastrointestinal cancers, Bristol Myers Squibb. “Opdivo with chemotherapy is now one of two newly approved Opdivo-based combinations to show superior overall survival benefit compared to chemotherapy alone, offering higher hopes for patients with unresectable advanced, recurrent or metastatic ESCC with tumor cell PD-L1 expression ≥ 1%. We are eager to introduce this new treatment option to patients in the European Union and potentially improve their survival outcomes.”
The EC has also approved Opdivo in combination with Yervoy (ipilimumab) for the first-line treatment of adult patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) with tumor cell PD-L1 expression ≥ 1%.
The EC approval allows for the use of Opdivo with fluoropyrimidine- and platinum-based combination chemotherapy for the first-line treatment of adults with unresectable advanced, recurrent or metastatic ESCC with tumor cell PD-L1 expression ≥ 1% in the 27 member states of the European Union, as well as Iceland, Liechtenstein and Norway.
CheckMate -648 Efficacy and Safety Results
Results from CheckMate -648 include:
- OS in participants with tumor cell PD-L1 expression ≥ 1%. (primary endpoint): Median OS was 15.44 months (95% Confidence Interval [CI]: 11.93, 19.52) for nivolumab with chemotherapy vs. 9.07 months (95% CI: 7.69, 9.95) for chemotherapy (Hazard Ratio [HR] = 0.54; 95% CI: 0.37, 0.80; p= <0.0001).
- PFS in participants with tumor cell PD-L1 expression ≥ 1%. (primary endpoint): Median PFS was 6.93 months (95% CI: 5.68, 8.34) for nivolumab with chemotherapy vs. 4.44 months (95% CI: 2.89, 5.82) for chemotherapy (HR = 0.65; 95% CI: 0.46, 0.92; p=0.0023)
- ORR (secondary endpoint): The ORR was 53.2% (95% CI: 45.1, 61.1) for nivolumab with chemotherapy vs. 19.7% (95% CI: 13.8, 26.8) for chemotherapy.
- Safety (based on pooled data across tumor types): Incidence of Grade 3-5 adverse reactions was 76% for nivolumab with chemotherapy and 62% for chemotherapy alone, with 1.4% fatal adverse reactions attributed to nivolumab in combination with chemotherapy including pneumonia, febrile neutropenia, thrombosis, pneumonitis, diarrhea, and renal failure. Median duration of therapy was 6.44 months (95% CI: 5.95, 6.80) for nivolumab with chemotherapy and 4.34 months (95% CI: 4.04, 4.70) for chemotherapy.
About CheckMate -648
CheckMate -648 is a randomized Phase 3 study evaluating Opdivo plus Yervoy (N=325) or Opdivo with fluorouracil and cisplatin (N=321) against fluorouracil plus cisplatin alone (N=324) in patients with previously untreated, unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma.
The primary endpoints of the trial are overall survival (OS) and progression-free survival (PFS) by blinded independent central review (BICR) in patients with tumor cell PD-L1 expression ≥1% for both Opdivo-based combinations versus chemotherapy. Secondary endpoints of the trial include OS and PFS by BICR in the all-randomized population.
In the Opdivo with chemotherapy arm, patients received treatment with Opdivo 240 mg on Day 1 and Day 15, fluorouracil 800 mg/m²/day on Day 1 through Day 5 (for 5 days), and cisplatin 80 mg/m² on Day 1 of four-week cycle. Patients received Opdivo for up to 24 months or until disease progression, unacceptable toxicity or withdrawal of consent, and chemotherapy until disease progression, unacceptable toxicity or withdrawal of consent.
In the Opdivo plus Yervoy arm, patients received treatment with Opdivo 3 mg/kg every 2 weeks and Yervoy 1 mg/kg every 6 weeks up to 24 months or until disease progression, unacceptable toxicity or withdrawal of consent.
Source: Bristol Myers Squibb
Bristol Myers's Opdivo gets approval for 2 cancer types in EU
Apr. 05, 2022 7:16 AM ET Bristol-Myers Squibb Company (BMY) By: Ravikash, SA News Editor2 Comments
The European Commission (EC) approved Bristol Myers Squibb's (NYSE:BMY) drug Opdivo for a type of bladder cancer and a form of esophageal cancer.
The EC approved Opdivo (nivolumab) for the adjuvant treatment of adults with muscle-invasive urothelial carcinoma with tumor cell PD-L1 expression ≥1% who are at a high risk of recurrence after undergoing radical resection.
https://seekingalpha.com/news/3820670-bristol-myerss-opdivo-gets-approval-for-2-cancer-types-in-eu
https://seekingalpha.com/symbol/BMY
Breyanzi (lisocabtagene maraleucel)
Bristol Myers Squibb Receives European Commission Approval for CAR T Cell Therapy Breyanzi (lisocabtagene maraleucel) for Certain Forms of Relapsed or Refractory Large B-cell Lymphoma
04/05/2022
CATEGORY:
Breyanzi represents a differentiated CAR T cell therapy with demonstrated rapid and durable complete responses and a manageable safety profile
Approval of Breyanzi isbased on results from TRANSCEND WORLD, and TRANSCEND NHL 001, the largest pivotal trial of patients with relapsed or refractory large B-cell lymphoma after at least two prior therapies,including those with a broad range of histologies and high-risk disease
Approval marks Bristol Myers Squibb’s second CAR T cell therapy approved in the EU, underscoring commitment to bringing the transformative potential of CAR T cell therapies to more patients
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced that the European Commission (EC) has granted Marketing Authorization for Breyanzi (lisocabtagene maraleucel; liso-cel), a CD19-directed chimeric antigen receptor (CAR) T cell immunotherapy, for the treatment of adult patients with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL), primary mediastinal large B-cell lymphoma (PMBCL), and follicular lymphoma grade 3B (FL3B) after two or more lines of systemic therapy. The Marketing Authorization approves Breyanzi for use in all European Union (EU) member states.*
Breyanzi is delivered asapersonalized treatment via a single infusion. Treatment with Breyanzi has demonstrated sustained complete responses in a high proportion of patients with R/R large B-cell lymphoma (LBCL) and a manageable and differentiated safety profile.
“Advancing cell therapies is a significant part of our commitment to deliver innovative and potentially curative treatments in order to transform the lives of people living with cancer,” said Samit Hirawat, M.D., chief medical officer, Bristol Myers Squibb. “Breyanzi addresses an ongoing unmet need for patients in Europe battling relapsed or refractory large B-cell lymphoma who have few treatment options that provide long-term remission. The EC approval of Breyanzi is a significant step towards bringing the novel and personalized science of CAR T cell therapies to more patients around the world.”
DLBCL is an aggressive blood disease accounting for one out of every three non-Hodgkin lymphoma (NHL) cases diagnosed, making it the most common form of NHL.1 More than two-thirds of patients with DLBCL will not respond to or will relapse following second-line treatment and, historically, response rates for these patients are low with complete response rates ranging from 2% to 15%.2 Despite recent treatment advancements, new options offering long-term clinical benefits are still needed.
“In DLBCL, the goal of treatment is to provide patients with durable remission. However, for patients whose disease relapses or doesn’t respond to initial therapy, there are limited treatment options that provide long-term disease control,” said Ulrich Jäger, M.D., hematologist at the Medical University of Vienna / Vienna General Hospital and investigator for TRANSCEND WORLD. “Liso-cel is an exciting new and differentiated option for patients in Europe with relapsed or refractory large B-cell lymphoma, offering those with a historically poor prognosis a potentially curative treatment option, and results from TRANSCEND NHL 001 and TRANSCEND WORLD reinforce liso-cel as a valuable treatment for a broad range of patients with DLBCL who have received at least two prior therapies.”
The Marketing Authorization is based on results from the TRANSCEND NHL 001 study evaluating Breyanzi in adult patients with R/R DLBCL, PMBCL and FL3B, including those with a broad range of histologies and high-risk disease. In 216 patients treated with Breyanzi and evaluable for efficacy, 73% of patients achieved a response (95% CI: 67%-78.5%), including 53% who had minimal or no detectable lymphoma remaining following treatment (complete response [CR]; 95% CI: 47%-60%). Median duration of response was 20.2 months in all responders (95% CI: 8 – NR), and for patients who achieved a CR, median duration of response was 26.1 months (95% CI: 23 – NR).
The safety of Breyanzi is based on pooled data from 314 patients with R/R LBCL treated with Breyanzi within a dose range of 44 to 120 x 106 CAR+ viable T cells across four studies (TRANSCEND NHL 001, TRANSCEND WORLD, PLATFORM and OUTREACH). Any grade cytokine release syndrome (CRS) occurred in 39% of patients, 3% of whom experienced Grade 3 or 4 CRS. The median time to onset was five days (range: 1 to 14 days) and the median duration was five days (range: 1 to 17 days). Neurologic toxicities (NT) occurred in 26% of patients receiving Breyanzi, including Grade 3 or 4 in 10% of patients. The median time to onset of the first NT event was nine days (range: 1 to 66 days); 99% of all NT occurred within the first eight weeks following Breyanzi infusion. The median duration of NT was 10 days (range: 1 to 84 days).
The most common Grade >3 adverse reactions were neutropenia, anemia, thrombocytopenia, leukopenia, infection with an unspecified pathogen and febrile neutropenia. For full details on the Special Warnings and Precautions for Use and Adverse Reactions (including appropriate management), please refer to the EU Summary of Product Characteristics (SmPC).
Bristol Myers Squibb offers various programs and resources to address the needs of patients and caregivers and help support access to therapies, including Breyanzi.
*Centralized Marketing Authorization does not include approval in Great Britain (England, Scotland and Wales).
About TRANSCEND NHL 001
TRANSCEND NHL 001 is an open-label, multicenter, pivotal Phase 1 study conducted in the U.S. to determine the safety, antitumor activity and pharmacokinetics of Breyanzi in patients with R/R LBCL, including DLBCL, high-grade B-cell lymphoma (HGL), PMBCL and FL3B. The primary outcome measures included treatment-related adverse events, dose-limiting toxicities and objective response rate. Key secondary outcome measures included complete response rate, duration of response, progression-free survival and overall survival. The TRANSCEND program is a broad clinical program evaluating Breyanzi in multiple hematologic indications and treatment lines.
About Breyanzi
Breyanzi is a CD19-directed chimeric antigen receptor (CAR) T cell therapy with a defined composition and 4-1BB costimulatory domain. Breyanzi is administered as a defined composition to reduce variability of the CD8 and CD4 component dose. The 4-1BB signaling domain enhances the expansion and persistence of the CAR T cells. In the EU, Breyanzi is administered via a single infusion containing 100 × 106 CAR+ viable T cells (consisting of a target 1:1 ratio of CD8+ and CD4+ cell components) within a range of 44 to 120 × 106 CAR+ viable T cells.
Breyanzi is approved by the U.S. Food and Drug Administration for the treatment of adult patients with relapsed or refractory (R/R) large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B cell lymphoma (DLBCL) not otherwise specified (including DLBCL arising from indolent lymphoma), high-grade B-cell lymphoma, primary mediastinal large B-cell lymphoma, and follicular lymphoma grade 3B. Breyanzi is also approved in Japan for the treatment of patients with third-line plus R/R LBCL and follicular lymphoma.
Full European Summary of Product Characteristics for Breyanzi is available from the EMA website at www.ema.europa.eu.
Further information is available at www.BreyanziREMS.com, or contact Bristol Myers Squibb at 1-888-423-5436.
Learn more about the science behind cell therapy and ongoing research at Bristol Myers Squibb here.
For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.
Source: Bristol Myers Squibb
Bristol Myers' CAR T cell therapy Breyanzi gets approval in EU to treat certain blood cancers
Apr. 05, 2022 11:55 AM ET
Bristol-Myers Squibb Company (BMY)
By: Ravikash, SA News Editor1 Comment
- The European Commission (EC) approved Bristol Myers Squibb's (NYSE:BMY) Breyanzi to treat adult patients with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL), primary mediastinal large B-cell lymphoma (PMBCL), and follicular lymphoma grade 3B (FL3B) after two or more lines of systemic therapy.
- https://seekingalpha.com/news/3820839-bristol-myers-car-t-cell-therapy-breyanzi-gets-approval-in-eu-to-treat-certain-blood-cancers
- https://seekingalpha.com/symbol/BMY
- https://www.bms.com/
- https://www.breyanzi.com/
RYBREVANT® (amivantamab)
Janssen Announces Health Canada Approval of RYBREVANT® (amivantamab), the First and Only Targeted Treatment for Patients with Non-Small Cell Lung Cancer with EGFR Exon 20 Insertion MutationsTargeting both cancer-driving and resistance mechanism pathways, RYBREVANT® offers a unique treatment approach for an underserved patient population
Toronto, ON, April 4, 2022 – The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that Health Canada has issued a Notice of Compliance with Conditions (NOC/c) approving RYBREVANT® (amivantamab), a fully-human, bispecific antibody, for the treatment of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with activating epidermal-growth factor receptor (EGFR) Exon 20 insertion mutations whose disease has progressed on or after platinum-based chemotherapy.[i]
Health Canada NOC/c is granted to promising new therapies for patients diagnosed with serious, life-threatening or severely debilitating diseases, conditions for which no drug is currently marketed in Canada, or for which a significant increase in efficacy or significant decrease in risk is demonstrated in relation to existing drugs marketed in Canada.[ii] This conditional approval is pending the results of trials to verify its clinical benefits.[i]
Lung cancer is the leading cause of cancer death among men and women in Canada, accounting for almost 25 per cent of all cancer deaths.[iii] More people die from lung cancer in Canada than breast, colorectal and prostate cancers combined.[iv]
An estimated 15 per cent of Canadians with non-squamous NSCLC have an activating EGFR mutation.[v] The frequency of EGFR mutations is even greater in patients of Asian descent (~39 per cent) and in Asia-Pacific countries (~47 per cent).[vi],[vii] Those with the third most prevalent variant, EGFR Exon 20 insertion mutation, tend to have a worse prognosis and shorter survival rates compared with individuals with more common EGFR mutations.[viii],[ix],[x] In fact, patients newly diagnosed with metastatic NSCLC with EGFR Exon 20 insertion mutations have a real-world median overall survival (OS) of 16.2 months, about nine months less than those with the more common EGFR Exon 19 deletions/L858R mutations (25.5 months).[xi]
“This approval marks a significant development for people living with this rare type of lung cancer who, until now, have had no approved treatment options to target their disease,” said Shem Singh, Executive Director, Lung Cancer Canada. “This specialized treatment option offers patients and their families new hope in managing the disease as they continue along their cancer journey.”
“Lung cancer is a complex disease and many patients have been left with sub-optimal treatment options. Thankfully, years of research have granted us a deeper understanding of lung cancer’s genetic alterations, giving rise to more sensitive testing and new targeted treatments like amivantamab,” says Natasha Leighl, MD, MMSc, FRCPC, FASCO, Lung Site Lead, Medical Oncology, Princess Margaret Cancer Centre, and principal study investigator**. “This approval means new hope and effective targeted treatment for a group of patients with lung cancer that previously had no options other than chemotherapy.”
“With previous treatment options there has been a high unmet need and gap in treating NSCLC attributed to Exon 20 insertion mutations. Amivantamab represents an exciting new frontier for the treatment of patients with this rare and complex genetic alteration,” Normand Blais, MD, MS, FRCPC, Head at the CHUM (University of Montreal Health Centre/Centre Hospitalier de l’Universite de Montreal), Integrated Cancer Centre (CICC) – Thoracic Oncology Program**. “Here in Canada, we’re fortunate to have tests such as Next Generation Sequencing (NGS) that are sensitive enough to identify these mutations. These capabilities, paired with a treatment with a distinct mechanism of action, brings an important and much-needed treatment option to target the disease and to improve patient outcomes.”
RYBREVANT® was issued an NOC/c based on the results of the CHRYSALIS study, a multi-center, open-label, multi-cohort clinical evaluation of the safety and efficacy of RYBREVANT® in patients whose disease has progressed on or after platinum-based chemotherapy.[i],[xii] The single-arm trial examined disease response based on overall response rate (ORR) and duration of response (DOR).[i]
“This milestone reflects progress and determination in our mission to develop and deliver transformational therapies to improve the lives of people diagnosed with this rare type of lung cancer,” said Kiran Patel, M.D., Vice President, Clinical Development, Solid Tumors, Janssen Research & Development, LLC. “The approval of RYBREVANT® strengthens our commitment to change the trajectory of lung cancer, the world’s leading cause of cancer mortality.”
About RYBREVANT®
RYBREVANT® is a fully-human bispecific antibody directed against EGFR and Mesenchymal-epithelial transition factor (MET) receptors.[i] It binds extracellularly, or to the outside of the cell, slowing or inhibiting tumour growth and leading to tumour cell death.[i] RYBREVANT® is indicated for the treatment of adult patients with locally advanced or metastatic NSCLC with activating EGFR Exon 20 insertion mutations whose disease has progressed on or after platinum-based chemotherapy.[i] A validated test is required to identify EGFR Exon 20 insertions mutation-positive status prior to treatment.[i]
About the CHRYSALIS Study
EDI1001 (CHRYSALIS) is a multi-center, open-label, multi-cohort study conducted to assess the safety and efficacy of RYBREVANT® (amivantamab) in 81 patients with locally advanced or metastatic NSCLC who had EGFR Exon 20 insertion mutations as determined by previous local standard of care testing, whose disease had progressed on or after platinum-based chemotherapy, and who had median efficacy follow-up of 9.7 months.[i] RYBREVANT® was administered intravenously at 1,050 mg for patients < 80 kg or 1400 mg for patients ≥ 80 kg once weekly for four weeks, then every two weeks starting at week 5 until disease progression or unacceptable toxicity.[i] Disease response using ORR, per Response Evaluation Criteria in Solid Tumors Version 1.1* (RECIST v1.1) as evaluated by Blinded Independent Central Review (BICR), was the primary endpoint.[i] Duration of response (DOR) by BICR was assessed as an additional measure of efficacy. In the prior-platinum chemotherapy treated cohort (n=81), the confirmed ORR, as assessed by BICR, was 40 per cent (95 per cent CI, 29 – 51), with 3.7 per cent having complete responses (CR) and 35.8 per cent achieving partial responses (PR).[i] The study also demonstrated a median DOR of 11.1 months (95 per cent CI, 6.9-not estimable) with 63 per cent of patients achieving a DOR of 6 months or longer.
In a safety population of 129 patients, adverse reactions (AR) resulting in permanent discontinuation of RYBREVANT® in greater than or equal to one per cent of patients were pneumonia, infusion-related reactions (IRRs), pneumonitis and pleural effusion.[i] Dose reductions of RYBREVANT® due to an AR occurred in 15 per cent of patients.[i] Adverse reactions requiring dose reductions in greater than or equal to two per cent of patients included dermatitis acneiform, and paronychia.[i] The most common ARs (greater than or equal to 20 per cent) in patients who received RYBREVANT® were dermatitis acneiform, rash, infusion-related reactions (IRR), nausea, paronychia, fatigue, hypoalbuminemia, constipation, stomatitis, and peripheral edema.[i]
Serious ARs occurred in 30 per cent of patients who received RYBREVANT®.[i] Serious adverse reactions in greater than or equal to two per cent of patients included pulmonary embolism, pneumonitis, dyspnea, back pain, and muscular weakness.[i]
*RECIST (version 1.1) refers to Response Evaluation Criteria in Solid Tumours, which is a standard way to measure how well solid tumours respond to treatment and is based on whether tumours shrink, stay the same or get bigger.[viii]
Johnson & Johnson's Rybrevant gets conditional approval in Canada for lung cancer subtype
Apr. 04, 2022 8:23 AM ET
By: Ravikash, SA News Editor
- Johnson & Johnson (NYSE:JNJ) said Health Canada granted conditional approval to Rybrevant (amivantamab) to treat adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with activating epidermal-growth factor receptor (EGFR) Exon 20 insertion mutations whose disease has progressed on or after platinum-based chemotherapy.
- https://seekingalpha.com/news/3820309-johnson-johnsons-rybrevant-gets-conditional-approval-in-canada-for-lung-cancer-subtype
- https://seekingalpha.com/symbol/JNJ
- https://www.rybrevant.com/
- https://www.jnj.com/
DUPIXENT® (DUPILUMAB)
FDA ACCEPTS DUPIXENT® (DUPILUMAB) FOR PRIORITY REVIEW IN PATIENTS AGED 12 YEARS AND OLDER WITH EOSINOPHILIC ESOPHAGITIS
If approved, Dupixent would be the first medicine available in the U.S. indicated to treat eosinophilic esophagitis
There are approximately 160,000 patients in the U.S. living with eosinophilic esophagitis who are currently treated, of whom approximately 48,000 have failed multiple treatments
TARRYTOWN, N.Y. and PARIS, April 4, 2022 /PRNewswire/ -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today announced the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the supplemental Biologics License Application (sBLA) for Dupixent® (dupilumab) 300 mg weekly to treat adult and pediatric patients aged 12 years and older with eosinophilic esophagitis (EoE), a chronic and progressive type 2 inflammatory disease that damages the esophagus and impairs the ability to swallow. The target action date for the FDA decision on this investigational use is August 3, 2022.
The sBLA is supported by data from two Phase 3 trials evaluating the efficacy and safety of Dupixent 300 mg weekly in patients aged 12 years and older with EoE (Part A and Part B), and data from an active long-term extension trial. Dupixent 300 mg weekly significantly improved the signs and symptoms of EoE at 24 weeks compared to placebo, including the ability to swallow and reduction in eosinophil count in the esophagus. The safety results of these trials were generally consistent with the known safety profile of Dupixent in its approved indications. The most common adverse event observed with Dupixent, in Part A and Part B, was injection site reactions.
In September 2020, the U.S. FDA granted Breakthrough Therapy designation to Dupixent for the treatment of patients aged 12 years and older with EoE. Dupixent was also granted Orphan Drug designation for the potential treatment of EoE in 2017. Priority review is granted to therapies that have the potential to provide significant improvements in the treatment, diagnosis or prevention of serious conditions. Regulatory filings around the world are also planned in 2022. The potential use of Dupixent in EoE is currently under clinical development, and the safety and efficacy have not been fully evaluated by any regulatory authority.
About Eosinophilic Esophagitis (EoE)
EoE is a chronic, progressive type 2 inflammatory disease that damages the esophagus and prevents it from working properly. For people with EoE, swallowing the smallest amount of food can be a painful and worrisome choking experience. Those with EoE live with anxiety and frustration from having a constantly evolving list of foods to avoid. This disease can also cause narrowing of the esophagus and dilation (physical expansion) of the esophagus may be needed, which is often painful. In severe cases, a feeding tube is the only option to ensure proper caloric intake and adequate nutrition. People with EoE may have poor quality of life and are more likely to experience depression than people without EoE. There are approximately 160,000 patients in the U.S. living with EoE who are currently treated, of whom approximately 48,000 have failed multiple treatments.
About Dupixent
Dupixent, which was invented using Regeneron's proprietary VelocImmune® technology, is a fully human monoclonal antibody that inhibits the signaling of the interleukin-4 (IL-4) and interleukin-13 (IL-13) pathways and is not an immunosuppressant. By demonstrating significant clinical benefit together with a decrease in type 2 inflammation following IL-4 and IL-13 blockade with Dupixent, the Dupixent Phase 3 clinical program has established that IL-4 and IL-13 are key drivers of the type 2 inflammation that plays a major role in multiple related and often co-morbid diseases for which Dupixent is approved including atopic dermatitis, asthma and chronic rhinosinusitis with nasal polyposis (CRSwNP), as well as investigational diseases such as EoE and prurigo nodularis, which have been studied in Phase 3 trials.
In the U.S., Dupixent is approved in patients aged 6 years and older with uncontrolled moderate-to-severe atopic dermatitis; as an add-on maintenance treatment of patients aged 6 years and older with moderate-to-severe asthma characterized by an eosinophilic phenotype or with oral corticosteroid-dependent asthma; and for use with other medicines for the maintenance treatment of chronic rhinosinusitis with nasal polyposis (CRSwNP) in adults whose disease is not controlled.
Dupixent is also approved in Europe, Japan and other countries around the world for use in specific patients with moderate-to-severe atopic dermatitis and certain patients with asthma or CRSwNP in different age populations. Dupixent is approved in one or more of these indications in more than 60 countries around the world, and more than 400,000 patients have been treated globally.
U.S. Indications
DUPIXENT is a prescription medicine used:
- to treat adults and children 6 years of age and older with moderate-to-severe atopic dermatitis (eczema) that is not well controlled with prescription therapies used on the skin (topical), or who cannot use topical therapies. DUPIXENT can be used with or without topical corticosteroids. It is not known if DUPIXENT is safe and effective in children with atopic dermatitis under 6 years of age.
- with other asthma medicines for the maintenance treatment of moderate-to-severe eosinophilic or oral steroid dependent asthma in adults and children 6 years of age and older whose asthma is not controlled with their current asthma medicines. DUPIXENT helps prevent severe asthma attacks (exacerbations) and can improve your breathing. DUPIXENT may also help reduce the amount of oral corticosteroids you need while preventing severe asthma attacks and improving your breathing. DUPIXENT is not used to treat sudden breathing problems. It is not known if DUPIXENT is safe and effective in children with asthma under 6 years of age.
- with other medicines for the maintenance treatment of chronic rhinosinusitis with nasal polyposis (CRSwNP) in adults whose disease is not controlled. It is not known if DUPIXENT is safe and effective in children with chronic rhinosinusitis with nasal polyposis under 18 years of age.
Please see accompanying full Prescribing Information including Patient Information.
For additional information about the company, please visit www.regeneron.com or follow @Regeneron on Twitter.
Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY.
View original content:https://www.prnewswire.com/news-releases/fda-accepts-dupixent-dupilumab-for-priority-review-in-patients-aged-12-years-and-older-with-eosinophilic-esophagitis-301516398.html
SOURCE Regeneron Pharmaceuticals, Inc.
Sanofi, Regeneron's Dupixent gets FDA priority review for eosinophilic esophagitis
Apr. 04, 2022 6:01 AM ET
By: Ravikash, SA News Editor
- Regeneron Pharmaceuticals (NASDAQ:REGN) and Sanofi (NASDAQ:SNY) said the U.S. Food and Drug Administration (FDA) granted priority review to Dupixent to treat patients aged 12 years and older with eosinophilic esophagitis (EoE).
- https://seekingalpha.com/news/3820235-sanofi-regenerons-dupixent-gets-fda-priority-review-for-eosinophilic-esophagitis
- https://seekingalpha.com/symbol/SNY
- https://seekingalpha.com/symbol/REGN
- https://www.dupixent.com/
- https://www.regeneron.com/
EDSIVO™ (celiprolol)
Acer Therapeutics’ EDSIVO™ (celiprolol) Granted FDA Breakthrough Therapy Designation for Vascular Ehlers-Danlos Syndrome
Discussions ongoing with FDA through special protocol assessment (SPA) seeking agreement on planned pivotal Phase 3 DiSCOVER trial with initiation planned by end of Q2 2022
NEWTON, MA – April 4, 2022 – Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs, today announced the U.S. Food and Drug Administration (FDA) has granted celiprolol Breakthrough Therapy designation (BTD) in the U.S. for the treatment of patients with COL3A1-positive vascular Ehlers-Danlos syndrome (vEDS).
BTD is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on at least one clinically significant endpoint(s) over available therapy.1
“With no currently approved treatments for vEDS anywhere in the world, this designation by FDA marks an important step forward in support of our goal to provide treatment options like EDSIVO™ to rare disease patients, who are often overlooked or underserved,” said Adrian Quartel, MD, Chief Medical Officer of Acer. “We look forward to continuing our discussions with FDA, through the SPA process, to seek agreement on the protocol design of the proposed pivotal Phase 3 DiSCOVER trial that we plan to initiate by the end of Q2 2022 once agreement is reached.”
EDSIVO™ (celiprolol) Regulatory Path Forward
In December 2021, Acer submitted to FDA a protocol for its proposed prospective pivotal trial, along with an IND which received FDA clearance in January 2022. Acer is working with FDA to reach agreement on a special protocol assessment (SPA), a process in which sponsors may ask to meet with FDA to reach agreement on the design and size of certain clinical trials, clinical studies, or animal studies.2 Based on initial statistical power calculations, the Company anticipates that the trial would plan to enroll approximately 150 COL3A1-positive vEDS patients, all in the U.S. The duration of the clinical trial is currently estimated to be approximately 3.5 years to completion, once fully enrolled.
If agreement on the design of the protocol is reached with FDA through the SPA, initiation of the EDSIVO™ Decentralized Study of Celiprolol on vEDS-related Event Reduction (DiSCOVER) pivotal clinical trial is expected by the end of Q2 2022, subject to additional capital required to conduct and complete the trial beyond mid-2022. There can be no assurance that the resulting data from the trial would be adequate to support approval of an NDA, or that the NDA will be approved, although having a SPA agreement in place would indicate concurrence by FDA with the adequacy and acceptability of specific critical elements of overall protocol design (e.g., study conduct, entry criteria, dose selection, endpoints, and planned statistical and other analyses) for a study intended to support a future marketing application. EDSIVO™ is an investigational drug and is not currently FDA approved for any indication. More information is available at www.discoverceliprolol.com.
About EDSIVO™ (celiprolol)
Acer is developing EDSIVO™, a new chemical entity (NCE), for the treatment of COL3A1-positive vEDS patients. Celiprolol received FDA Orphan Drug Designation for the treatment of vEDS in 2015. The EDSIVO™ NDA was originally submitted based on data obtained from the BBEST trial4 and accepted for filing in October 2018 with priority review. Following FDA review, Acer received a Complete Response Letter (CRL) in June 2019 stating that it will be necessary to conduct an adequate and well-controlled trial to determine whether EDSIVO™ reduces the risk of clinical events in patients with vEDS. Acer subsequently appealed the FDA decision and while FDA denied the appeal, it described possible paths forward for Acer to explore. In a Type B meeting in May 2021, the Company discussed with FDA the conduct of an U.S.-based prospective, randomized, double-blind, placebo-controlled, decentralized clinical trial in patients with COL3A1-positive vEDS, and sought FDA’s opinion on various proposed design features of the study. The official Type B meeting minutes outlined: the acceptability of a decentralized (virtual) clinical trial design and use of an independent centralized adjudication committee; acceptability of a primary endpoint based on clinical events associated with disease outcome; agreement with modest safety data collection (based on the known safety profile of the drug4,5,6,7); and a statistical plan that considers the rare disease classification of vEDS.
https://www.acertx.com/rare-disease-research/edsivo-celiprolol-for-veds/
For more information, visit www.acertx.com.
Acer gains on FDA’s Breakthrough status for Edsivo in genetic disorder
Apr. 04, 2022 9:12 AM ET
By: Dulan Lokuwithana, SA News Editor
- The clinical-stage pharma company, Acer Therapeutics (NASDAQ:ACER), has added ~27% in the pre-market Monday after announcing that the U.S. Food and Drug Administration (FDA) granted the Breakthrough Therapy designation for experimental therapy, celiprolol.
- https://seekingalpha.com/news/3820360-acer-stock-on-fdas-breakthrough-status-for-edsivo-in-genetic-disorder
- https://seekingalpha.com/symbol/ACER
- https://www.acertx.com/rare-disease-research/edsivo-celiprolol-for-veds/
Actemra®/RoActemra® (tocilizumab)
U.S. FDA grants priority review to Roche’s Actemra/RoActemra for the treatment of COVID-19 in hospitalised adultsApril 4, 2022
- If approved, Actemra/RoActemra would be the first U.S. FDA-approved immunomodulator for the treatment of COVID-19 in hospitalised patients
- Since the beginning of the pandemic, more than one million people hospitalised with COVID-19 have been treated with Actemra/RoActemra worldwide1
- Actemra/RoActemra is approved for the treatment of COVID-19 in many territories including the European Union
- Roche has established a comprehensive access approach to improve availability of Actemra/RoActemra around the world
Basel, 04 April 2022 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s supplemental Biologics License Application (sBLA) and has granted Priority Review for Actemra®/RoActemra® (tocilizumab) intravenous for the treatment of COVID-19 in hospitalised adults who are receiving systemic corticosteroids and require supplemental oxygen, non-invasive or invasive mechanical ventilation, or extracorporeal membrane oxygenation. A decision on U.S. FDA approval is expected in the second half of this year.
“The high rate of unvaccinated people will continue to put a strain on hospitals and healthcare systems around the world, furthering the need for effective treatments for patients hospitalised with COVID-19,” said Levi Garraway, M.D., Ph.D., Chief Medical Officer and Head of Global Product Development, Roche. “More than one million people with severe or critical COVID-19 have already been treated with Actemra/RoActemra worldwide, demonstrating the important role of this medicine in the fight against the pandemic.”
The sBLA submission is based on results from four randomised, controlled studies that evaluated Actemra/RoActemra for the treatment of COVID-19 in more than 5,500 hospitalised patients. Altogether, the results of these four studies (EMPACTA, COVACTA, REMDACTA, and RECOVERY) suggest that Actemra/RoActemra may improve outcomes in patients receiving corticosteroids and requiring supplemental oxygen or breathing support.2-5
In June 2021, Actemra/RoActemra received Emergency Use Authorization from the U.S. FDA and is currently approved for use in 16 countries around the world for defined patients hospitalised with severe or critical COVID-19.6,7 In February 2022, the World Health Organization (WHO) prequalified Actemra/RoActemra for patients with severe or critical COVID-19, supporting access to care in low- and middle-income countries.8
In addition to working with health authorities, Roche has established a comprehensive access approach to improve availability of its COVID-19 medicines around the world including:
- Providing Actemra/RoActemra at cost to WHO and partners of the Access to COVID-19 Tools Accelerator (ACT-A) Initiative, building on the significant portion of Actemra/RoActemra supply that Roche has provided to upper-middle- and lower-middle-income countries since the beginning of the pandemic.7
- Implementing an international differentiated pricing strategy specifically designed to address needs during this pandemic and improve affordability.7
Following the emergence of the SARS-CoV-2 variant of concern, Omicron (B.1.1.529), in December 2021 WHO reported that interleukin-6 receptor blockers, such as Actemra/RoActemra, are expected to still be effective for managing patients with severe COVID-19.9
Roche stands together with society, governments, healthcare providers and all those working towards the common goal of overcoming the COVID-19 pandemic.
About Actemra®/RoActemra® (tocilizumab) in COVID-19
Actemra/RoActemra is approved for use in multiple territories including the European Union, Japan, Bolivia, Chile, Guatemala, Ecuador, Honduras, Hong Kong, Myanmar, Peru, Philippines, the United Kingdom and Ukraine, provisionally approved in Australia, and authorised for emergency use in Ghana, Korea and the United States for defined patients hospitalised with severe or critical COVID-19. It has also been recommended and prequalified by the World Health Organization.
About the Actemra®/RoActemra® (tocilizumab) COVID-19 Clinical Trial Programme
Roche’s clinical trial programme evaluated the safety and efficacy of Actemra/RoActemra in hospitalised patients with COVID-19. Actemra/RoActemra is not U.S. Food and Drug Administration (FDA)-approved for this use and there is limited information known about the safety or effectiveness of using Actemra/RoActemra to treat people in the hospital with COVID-19. COVACTA and EMPACTA were the first two global phase III, multicentre, randomised, placebo-controlled studies of Actemra/RoActemra in patients hospitalised with COVID-19 associated pneumonia. COVACTA was conducted in collaboration with the Biomedical Advanced Research and Development Authority (BARDA), part of the Office of the Assistant Secretary for Preparedness and Response at the United States Department of Health and Human Services (HHS). EMPACTA aimed to address research questions about the safety and efficacy of Actemra/RoActemra in underserved populations by emphasising enrollment from minority patients often underrepresented in clinical trials. Both studies were published in the New England Journal of Medicine. Roche also partnered with Gilead Sciences, Inc., on REMDACTA, a phase III, randomised, double-blind, multicentre study to evaluate the safety and efficacy of Actemra/RoActemra plus Veklury® (remdesivir), versus placebo plus Veklury, in hospitalised patients with severe COVID-19 associated pneumonia.
About the U.S. Food and Drug Administration (FDA) Emergency Use Authorization (EUA) for Actemra®/RoActemra® (tocilizumab)
Actemra/RoActemra has not been approved by the U.S. FDA in this setting, but the FDA has made Actemra/RoActemra available under an emergency access mechanism called an EUA as a treatment for certain patients with COVID-19. There is limited information known about the safety or effectiveness of using Actemra/RoActemra to treat people in the hospital with COVID-19. The EUA is supported by a Secretary of Health and Human Services (HHS) declaration that circumstances exist to justify the emergency use of drugs and biological products during the COVID-19 pandemic. The authorisation is temporary and does not replace the formal review and approval process, which is being pursued. Actemra/RoActemra is authorised under the EUA only for the duration of the declaration that circumstances exist justifying the authorisation of the emergency use of Actemra/RoActemra under Section 564(b)(1) of the Act, 21 U.S.C. § 360bbb-3(b)(1), unless the authorisation is terminated or revoked sooner.
About Actemra®/RoActemra® (tocilizumab)
Actemra/RoActemra was the first humanised interleukin-6 (IL-6) receptor antagonist approved for the treatment of adult patients with moderately to severely active rheumatoid arthritis (RA) who have used one or more disease-modifying antirheumatic drugs (DMARDs), such as methotrexate (MTX), that did not provide enough relief. The extensive Actemra/RoActemra RA intravenous (IV) clinical development programme included five phase III clinical studies and enrolled more than 4,000 people with RA in 41 countries. The Actemra/RoActemra RA subcutaneous clinical development programme included two phase III clinical studies and enrolled more than 1,800 people with RA in 33 countries. Actemra/RoActemra subcutaneous injection is also approved for the treatment of adult patients with giant cell arteritis (GCA), for the treatment of patients two years of age and older with active polyarticular juvenile idiopathic arthritis (PJIA) or active systemic juvenile idiopathic arthritis (SJIA), and for slowing the rate of decline in pulmonary function in adult patients with systemic sclerosis-associated interstitial lung disease (SSc-ILD). In addition, Actemra/RoActemra is also approved in the IV formulation for patients two years of age and older with active PJIA, SJIA, GCA, or CAR T cell-induced cytokine release syndrome (CRS). Actemra/RoActemra is not approved for subcutaneous use in people with CRS. It is not known if Actemra/RoActemra is safe and effective in children with PJIA, SJIA or CRS under two years of age or in children with conditions other than PJIA, SJIA or CRS.
For more information on how Roche is responding to the global COVID-19 pandemic, please visit our COVID-19 response page.
For more information, please visit www.roche.com.
Roche's Actemra gets FDA priority review to treat COVID-19
Apr. 04, 2022 4:31 AM ET
Roche Holding AG (RHHBF), RHHBY
By: Ravikash, SA News Editor
- Roche (OTCQX:RHHBF) (OTCQX:RHHBY) said the U.S. Food and Drug Administration (FDA) granted priority review to Actemra/RoActemra to treat certain patients with COVID-19.
- https://seekingalpha.com/news/3820213-roches-actemra-gets-fda-priority-review-to-treat-covid-19
- https://seekingalpha.com/symbol/RHHBF
- https://www.actemra.com/
- https://www.roche.com/
Rubraca® (Rucaparib)
Clovis Oncology’s Rubraca® (Rucaparib) Significantly Improves Progression-Free Survival In First-Line Maintenance Treatment In Women With Ovarian Cancer Regardless Of Their Biomarker Status In Phase 3 ATHENA-MONO Trial
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- ATHENA study evaluating Rubraca monotherapy versus placebo (ATHENA-MONO) successfully achieved the primary endpoint of improved PFS in both populations in the primary efficacy analyses: HRD-positive and all patients randomized (ITT)
- Median PFS of 20.2 months for Rubraca vs 9.2 months for placebo in the ITT population
- The exploratory PFS endpoints were also achieved in both HRD-negative and BRCA mutant subgroups of patients
- Safety of Rubraca observed in ATHENA-MONO was consistent with both the current US and European labels
- ATHENA-MONO results will serve as the basis of a supplemental NDA for US label expansion to be submitted during Q2 2022; European submission to follow during Q3 2022
- These data, including additional analyses, have been submitted for presentation at the American Society of Clinical Oncology Annual Meeting in June 2022
BOULDER, Colo.--(BUSINESS WIRE)-- Clovis Oncology, Inc. (NASDAQ: CLVS) today announced positive top-line data from the monotherapy arm of the ATHENA (GOG 3020/ENGOT-ov45) trial (ATHENA-MONO) demonstrating that Rubraca as maintenance treatment successfully achieved the primary endpoint of significantly improved investigator-assessed progression-free survival (PFS) compared with placebo. Benefit was observed in both primary efficacy analyses of newly-diagnosed patients with advanced ovarian cancer following successful treatment with platinum-based chemotherapy: those who had homologous recombination deficiency (HRD-positive)1, including deleterious BRCA mutations, as well as all patients randomized in the trial (overall intent-to-treat population (ITT)). Benefit in PFS was also seen in the exploratory subgroups of patients with HRD-negative2 and BRCA mutant (BRCAm) tumors. The safety of Rubraca observed in the ATHENA-MONO study was consistent with both the US and European labels.
Based on these results, the Company plans to submit a supplemental New Drug Application (sNDA) to the US FDA during the second quarter of 2022 followed by a Type II Variation to the EMA during the third quarter of 2022 for a first-line maintenance treatment indication for women with advanced ovarian cancer regardless of biomarker status who have responded to first-line platinum-based chemotherapy.
“The results from the ATHENA-MONO study of Rubraca in first-line maintenance treatment ovarian cancer exceeded our expectations in terms of significant improvement in PFS versus placebo in each of the primary efficacy populations, including the all-comers or intent-to-treat population,” said Patrick J. Mahaffy, President and CEO of Clovis Oncology. “We believe that the positive results from ATHENA-MONO demonstrate that Rubraca will provide an important new treatment option for women with advanced ovarian cancer in the first-line maintenance setting, and we look forward to submitting these data to the regulatory authorities in the US and Europe during Q2 and Q3 2022, respectively. Most importantly, I would like to thank the patients, physicians, and our colleagues whose commitment to this trial made these results possible, which now offer the potential to make a difference in the lives of many women with advanced ovarian cancer. We would also like to thank GOG and ENGOT for their partnership in conducting this large and very important trial.”
“While PARP inhibitors have shown efficacy as first-line maintenance treatment for patients with advanced ovarian cancer, questions still remain about the patient population that may benefit from their use. The results of ATHENA-MONO address many of these unanswered questions and expands the opportunity for rucaparib in all patients regardless of biomarker status,” said Bradley J. Monk, MD, FACOG, FACS, at GOG Foundation, HonorHealth Research Institute, University of Arizona College of Medicine, Creighton University School of Medicine, Phoenix, AZ and global primary investigator of the ATHENA trial.
“I believe the significant improvement in PFS demonstrated in the ATHENA-MONO trial underscores the importance of first-line maintenance therapy and the benefit that rucaparib can provide to women with advanced ovarian cancer irrespective of HRD status,” said Rebecca S. Kristeleit, MD, PhD, of Guy’s and St Thomas’ NHS Foundation Trust in London and lead ENGOT/NCRI National Cancer Research Institute (https://www.ncri.org.uk/) investigator of the ATHENA trial. “Ovarian cancer remains a leading cause of cancer-related death among women, which highlights the continued need for new treatment options and strategies for women with newly-diagnosed disease. The ATHENA-MONO study demonstrates the role of rucaparib monotherapy in the first-line maintenance treatment setting for advanced ovarian cancer.”
ATHENA is a double-blind, placebo-controlled, Phase 3 trial of rucaparib in first-line ovarian cancer maintenance treatment. It has two parts which are statistically independent. The top-line results reported today are from the ATHENA-MONO part (rucaparib vs placebo) with results from the ATHENA-COMBO part (rucaparib+nivolumab vs rucaparib) now expected in Q1 2023 based on a slower than expected event count.
ATHENA-MONO enrolled 538 women with high-grade ovarian, fallopian tube, or primary peritoneal cancer. The primary efficacy analysis evaluated two prospectively defined molecular sub-groups in a step-down manner: 1) HRD-positive (inclusive of BRCAm tumors), and 2) all patients randomized (ITT) in ATHENA-MONO.
Following is a summary of the primary efficacy analyses by investigator review, the primary analysis of ATHENA-MONO.
Significant Improvement in PFS in the HRD-positive Patient Population
By investigator review, the rucaparib arm (n=185) successfully achieved statistical significance over the placebo arm (n=49) for the primary endpoint of PFS with a hazard ratio of 0.47 (95% CI: 0.31-0.72). The median PFS for the HRD-positive patient population treated with rucaparib was 28.7 months vs 11.3 months among those who received placebo (p=0.0004).
Significant Improvement in PFS in All Patients Studied (ITT or all comers)
Rucaparib also showed statistical significance in all 538 patients randomized in the ATHENA-MONO comparison. By investigator review, the rucaparib arm (n=427) successfully achieved statistical significance over the placebo arm (n=111) for the primary endpoint of PFS with a hazard ratio of 0.52 (95% CI: 0.40-0.68). The median PFS for all patients enrolled in ATHENA-MONO and treated with rucaparib was 20.2 months vs 9.2 months among those who received placebo (p<0.0001).
Treatment Benefit in PFS Endpoint for Exploratory HRD-negative Subgroup
By investigator review, the PFS endpoint in the exploratory subgroup of HRD-negative demonstrated a hazard ratio of 0.65 (95% CI: 0.45-0.95). The median PFS for these patients treated with rucaparib (n=189) was 12.1 months vs. 9.1 months for those who received placebo (n=49) (p=0.0284).
Treatment Benefit in PFS Endpoint for Exploratory BRCAm Subgroup
By investigator review, the PFS endpoint in the exploratory subgroup of BRCAm demonstrated a hazard ratio of 0.40 (95% CI: 0.21-0.75). The median PFS for these patients treated with rucaparib (n=91) was Not Reached vs 14.7 months for those who received placebo (n=24) (p=0.0041).
Results were consistent for the germline BRCA (n=68) and somatic BRCA (n=33) and unknown (n=14) populations.
Summary of ATHENA-MONO Safety
The safety of Rubraca observed in ATHENA-MONO was consistent with both the current US and European labels. The most common (≥5%) treatment-emergent grade 3/4 adverse events (TEAEs) among all patients treated with rucaparib in the monotherapy portion of the ATHENA study were anemia/decreased hemoglobin (28.7%), neutropenia (14.6%), ALT/AST increase (10.6%), and thrombocytopenia (7.1%). The discontinuation rate for TEAEs was 11.8% for rucaparib-treated patients and 5.5% for the placebo arm. The rate of treatment-emergent myelodysplastic syndrome (MDS)/acute myeloid leukemia (AML) in the rucaparib arm was 0.2%, and no patients on the placebo arm experienced treatment-emergent MDS/AML.
Clovis Oncology plans to provide an expanded description of the ATHENA-MONO results in a scientific session at a medical meeting later this year; these data have been submitted for presentation at the American Society of Clinical Oncology Annual Meeting in June 2022.
Rubraca is not currently approved in the first-line ovarian cancer maintenance setting. Clovis intends to provide these data to US and European regulatory authorities and is on track to submit filings during the second and third quarters of 2022, respectively, in those geographies.
About the ATHENA Clinical Trial
ATHENA (GOG 3020/ENGOT-ov45) (NCT03522246) is an international, randomized, double-blind, phase III trial consisting of two separate and fully independently powered study comparisons evaluating Rubraca monotherapy (ATHENA-MONO) and Rubraca in combination with nivolumab (ATHENA-COMBO) as maintenance treatment for patients with newly diagnosed advanced epithelial ovarian, fallopian tube, or primary peritoneal cancer. ATHENA enrolled approximately 1000 patients across 24 countries, all women with newly diagnosed ovarian cancer who responded to their first-line chemotherapy. The trial completed accrual in 2020 and was conducted in association with the Gynecologic Oncology Group (GOG) in the US and the European Network of Gynaecological Oncological Trial groups (ENGOT) in Europe. GOG and ENGOT are the two largest cooperative groups in the US and Europe dedicated to the treatment of gynecological cancers.
ATHENA-MONO is evaluating the benefit of Rubraca monotherapy versus placebo in 538 women in this patient population. The primary efficacy analysis evaluated two prospectively defined molecular sub-groups in a step-down manner: 1) HRD-positive (inclusive of BRCA mutant) tumors, and 2) the intent-to-treat population, or all patients treated in ATHENA-MONO.
The ATHENA-COMBO portion of the trial, anticipated to readout in Q1 2023, is evaluating the magnitude of benefit of adding Opdivo (nivolumab) to Rubraca monotherapy in the ovarian cancer first-line maintenance treatment setting. ATHENA-COMBO is anticipated to be the first Phase 3 dataset to readout evaluating the combination of a PARP inhibitor and an immune checkpoint inhibitor as maintenance treatment following completion and response to front-line chemotherapy.
About Rubraca (rucaparib)
Rucaparib is an oral, small molecule inhibitor of PARP1, PARP2 and PARP3 being developed in multiple tumor types, including ovarian and metastatic castration-resistant prostate cancers, as monotherapy, and in combination with other anti-cancer agents. Exploratory studies in other tumor types are also underway.
Rubraca is an unlicensed medical product outside of the US and Europe.
Rubraca Ovarian Cancer US FDA Approved Indications
Rubraca is indicated for the maintenance treatment of adult women with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy.
Rubraca is indicated for the treatment of adult women with a deleterious BRCA mutation (germline and/or somatic)-associated epithelial ovarian, fallopian tube, or primary peritoneal cancer who have been treated with two or more chemotherapies. Select patients for therapy based on an FDA-approved companion diagnostic for Rubraca.
Clovis Oncology is headquartered in Boulder, Colorado, with additional office locations in the US and Europe. Please visit www.clovisoncology.com for more information.
Source: Clovis Oncology, Inc.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220331005384/en/
Clovis Oncology extends two-day rally after trial win for ovarian cancer therapy
Apr. 04, 2022 3:59 PM ET
By: Dulan Lokuwithana, SA News Editor
- The commercial-stage biotech Clovis Oncology (CLVS +9.0%) continues to trade higher on Monday extending the recent rally to third consecutive session following the positive late-stage results announced by the company last week for Rubraca in ovarian cancer.
- The announcement that the monotherapy arm of the Phase 3 ATHENA trial met the primary endpoint for Rubraca as maintenance treatment propelled beaten-down Clovis (NASDAQ:CLVS) shares last week.
- https://seekingalpha.com/news/3820519-clovis-oncology-extends-two-day-rally-after-trial-win-for-ovarian-cancer-therapy
- https://seekingalpha.com/symbol/CLVS
- https://www.rubraca.com/
Mavacamten
Mavacamten Demonstrated Significant Reduction in Need for Septal Reduction Therapy in Symptomatic Obstructive HCM Patients in Phase 3 VALOR Trial
04/02/2022CATEGORY:
Mavacamten is a first-in-class, investigational cardiac myosin inhibitor being developed by Bristol Myers Squibb
Study met all primary and secondary endpoints and patients receiving mavacamten demonstrated improvement in key cardiac measures after 16 weeks of treatment
VALOR-HCM study presented as late-breaking clinical trial at the American College of Cardiology’s 71st Annual Scientific Session
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced results from the Phase 3 VALOR-HCM study, which showed the addition of mavacamten, an investigational, first-in-class cardiac myosin inhibitor, significantly reduced the need for septal reduction therapy (SRT) in patients with severely symptomatic obstructive hypertrophic cardiomyopathy (obstructive HCM) who had been appropriate for SRT per the 2011 American College of Cardiology/American Heart Association (ACC/AHA) Guidelines at baseline. Study participants were on maximally tolerated background regimens when they entered the trial and remained on them through the duration of the study. These data were presented today as a late-breaking clinical trial at the American College of Cardiology’s 71st Annual Scientific Session.
At 16 weeks the primary and all secondary endpoints were met. Of patients treated with mavacamten, 82% had not proceeded with SRT and no longer met the criteria for SRT according to the 2011 ACC/AHA Guidelines compared to 23% of patients receiving placebo. Patients in the mavacamten arm also demonstrated reduction in left ventricular outflow tract (LVOT) gradients, improvement in New York Heart Association (NYHA) Classification, improvement in quality-of-life measures and improvement in cardiac biomarkers at a high degree of statistical significance compared to the placebo arm. No new safety signals were observed.
“VALOR-HCM builds upon findings of the Phase 3 EXPLORER-HCM trial and shows mavacamten to be an effective potential treatment option for those with severe symptomatic obstructive HCM who meet guideline criteria for SRT,” said Milind Desai, M.D., MBA, director of HCM center and director of clinical operations, Heart, Vascular & Thoracic Institute, Cleveland Clinic. “The data presented today are clinically meaningful and have demonstrated the potential to impact parameters leading to SRT eligibility.”
Key findings at Week 16 included:
- The composite percentage of patients who proceeded with SRT before or after Week 16 and those who remained eligible for SRT was significantly lower in those taking mavacamten than those receiving placebo (17.9% [10/56] vs 76.8% [43/56]; P<0.0001).
- Post-exercise LVOT peak gradient significantly decreased in patients treated with mavacamten vs placebo (mean values at Week 16 = 42.0 mmHg ± 30.0 mmHg vs 83.2 mmHg ± 36.4 mmHg due to reductions from baseline with mavacamten of -39.1 mmHg ± 36.5 mmHg vs -1.8 mmHg ± 28.8 mmHg with placebo).
- The proportion of patients who improved ≥1 NYHA Class was significantly greater with mavacamten vs placebo (62.5% [35/56] vs 21.4% [12/56]; P<0.0001).
- On the patient-reported 23-item Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-23 CSS), average scores of symptom frequency, symptom burden and physical limitation significantly improved in patients treated with mavacamten vs placebo (change from baseline: +10.4 ± 16.1 vs +1.9 ± 12.0; P<0.0001).
- Improvement in biomarkers of cardiac wall stress and myocardial injury with mavacamten treatment over placebo showed reduction in N-terminal pro brain natriuretic peptide (NT-proBNP) that was 67% greater and reduction in cardiac troponin I that was 47% greater (p<0.0001).
- Safety data show no subjects permanently discontinuing therapy due to left ventricular ejection fraction (LVEF) ≤30% and no subjects experiencing serious adverse events of congestive heart failure, syncope or sudden cardiac death. Two subjects transiently experienced LVEF <50% and resumed treatment on a lower dose after a short interruption.
“These results validate the promising potential of mavacamten as an important treatment option for symptomatic oHCM patients,” said Marie-Laure Papi, vice president and mavacamten development lead, Bristol Myers Squibb.
In the Phase 3 study, patients with symptomatic obstructive HCM (NYHA Class III-IV or Class II with exertional syncope or near syncope) who met the 2011 ACC/AHA Guideline criteria and were referred for SRT were randomized 1:1 to mavacamten (n=56) or placebo (n=56) for 16 weeks. Study participants remained consistent on their maximally tolerated baseline standard of care regimens, which included ß-blockers, calcium channel blockers and/or disopyramide administered as monotherapy or in combination. Echocardiograms were conducted to evaluate LVOT gradient and LVEF at baseline and during drug titration to guide dosing and assess safety at Weeks 4, 8 and 12. Change from baseline in SRT eligibility, post-exercise LVOT peak gradient, NYHA Class, KCCQ-23 CSS and biomarkers (NT-proBNP and cardiac troponin I) were analyzed at Week 16.
Eligibility for SRT was determined based on NYHA Class III or Class IV and LVOT gradient ≥ 50 mmHg at rest or with exertion from Valsalva or exercise, or NYHA Class II with exertional symptoms of syncope or near syncope and elevated gradients. NYHA Classification ranges from I to IV, with Class I showing no symptoms and Class IV exhibiting symptoms at rest.1 The KCCQ-23 CCS is the average score of patient-reported clinical symptoms, including the frequency and burden of lower extremity swelling, fatigue and dyspnea as well as physical limitations, including, but not limited to, dressing, showering, walking and yardwork.2 Scores are based on a scale of 0 (worst) to 100 (best), with a change of 5 points considered clinically important and ≥10 to 20 points considered a moderate-to-large improvement.3,4
About the Phase 3 VALOR-HCM Trial
VALOR-HCM (NCT04349072) is a randomized, double-blind, placebo-controlled, multicenter Phase 3 study of patients with symptomatic obstructive HCM (NYHA Class III-IV or NYHA Class II with exertional syncope or near syncope) who meet guideline criteria for septal reduction therapy (SRT) and have been referred for an invasive procedure. The study enrolled 112 patients randomized on a 1:1 basis to receive mavacamten or placebo. VALOR-HCM includes three treatment periods over 128 weeks: a 16-week placebo-controlled period, a 16-week active treatment period where all patients will receive mavacamten and a 96-week long-term extension period where all patients will continue to receive mavacamten.
The primary endpoint of VALOR-HCM is a composite of the number of patients who decide to proceed with SRT prior to or at Week 16 and the number of patients who remain SRT-guideline eligible (LVOT gradient of ≥50mmHg and NYHA Class III-IV or Class II with syncope) at Week 16 in the mavacamten group compared with the placebo group. Key secondary endpoints include impact on exercise gradient LVOT, NYHA Class and Kansas City Cardiomyopathy Questionnaire (KCCQ) and cardiac biomarkers (NT-proBNP and cardiac troponin I) at Week 16.
About Mavacamten
Mavacamten is a first-in-class, oral, allosteric modulator of cardiac myosin being investigated for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (obstructive HCM) which is a progressive disease that thickens the heart walls and makes it harder for the heart to expand normally and fill with blood. It is a selective cardiac myosin inhibitor that targets the underlying pathophysiology of obstructive HCM.
Mavacamten has been shown to reduce cardiac muscle contractility by inhibiting excessive myosin-actin cross-bridge formation that results in hypercontractility, left ventricular hypertrophy and reduced compliance. Based on data from the EXPLORER-HCM study, the company has a PDUFA date in the U.S. of April 28, 2022.
In clinical and preclinical studies, mavacamten has consistently reduced biomarkers of cardiac wall stress, lessened excessive cardiac contractility, increased diastolic compliance and lessened left ventricular outflow tract (LVOT) gradients. Mavacamten is an investigational therapy and is not approved for use in any country.
About Bristol Myers Squibb
Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.
Source: Bristol Myers Squibb
Bristol Myers posts long-term data for heart-disease drug in late-stage trial
Apr. 04, 2022 7:06 AM ET
Bristol-Myers Squibb Company (BMY)LIAN
By: Dulan Lokuwithana, SA News Editor
Bristol Myers Squibb (NYSE:BMY) shared new interim data from a late-stage study to indicate that its oral cardiac myosin inhibitor, mavacamten led to sustained improvements in clinically meaningful cardiovascular outcomes in patients with symptomatic obstructive hypertrophic cardiomyopathy.
https://seekingalpha.com/symbol/BMY
Ondexxya (andexanet alfa)
Ondexxya approved in Japan for reversal of acute major bleeds in patients on Factor Xa inhibitors
PUBLISHED29 March 2022
29 March 2022 07:00 BST
Ondexxya is the first approved reversal agent specifically for Factor Xa inhibitors, providing a major advance in the treatment of patients hospitalised with life-threatening bleeding
Ondexxya (andexanet alfa) has been approved in Japan for patients treated with the Factor Xa (FXa) inhibitors apixaban, rivaroxaban or edoxaban, when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding.
The approval by the Japanese Ministry of Health, Labour and Welfare was based on positive results from the ANNEXA-4 Phase III clinical trial showing Ondexxya rapidly and markedly reversed anti-FXa activity in patients with acute major bleeding.
Ondexxya is the first approved medicine in Japan to specifically reverse the anticoagulant effect of FXa inhibitors apixaban, rivaroxaban or edoxaban in patients experiencing a life-threatening or uncontrolled bleed. Japan is also the first country to provide full regulatory approval of Ondexxya for use with all three of the FXa inhibitors currently available. FXa inhibitors are increasingly used for the prevention and treatment of thrombotic events, including deep vein thrombosis and pulmonary embolism, or in patients at high risk of a stroke due to an irregular heart rate (atrial fibrillation). While they prevent unwanted clots from forming, they can also increase the risk of major bleeding, which can be life-threatening.1,2
Masahiro Yasaka, MD, PhD, National Hospital Organisation Kyushu Medical Centre, Fukuoka, Japan, said: “FXa inhibitors are essential medicines for people prone to developing blood clots, but they can also present a risk of uncontrolled bleeding and related complications, which can be fatal if left untreated. Ondexxya’s rapid reversal of the anticoagulating effects of apixaban, rivaroxaban and edoxaban effectively reduces the bleeding and is a major advancement in patient care.”
Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, AstraZeneca, said: “With the approval of Ondexxya in Japan, we are working to make this important medicine available as quickly as possible for the small proportion of patients with life-threatening or uncontrolled bleeding who are on FXa inhibitors and who have not previously had an approved reversal agent treatment option.”
Ondexxya received approval by the US Food and Drug Administration under the accelerated approval pathway in May 2018 and conditional approval by the European Commission in April 2019 for adults treated with FXa inhibitors apixaban and rivaroxaban. In the US, Ondexxya is marketed under the trade name Andexxa [coagulation factor Xa (recombinant), inactivated-zhzo].
ANNEXA-4
The approval of Ondexxya is supported by data from the ANNEXA-4 Phase III trial, which evaluated the haemostatic efficacy and safety of Ondexxya in patients receiving a FXa inhibitor who were experiencing an acute major bleed.6 In the trial, Ondexxya markedly reversed anti-FXa activity within minutes, with 80% of patients having excellent or good haemostatic efficacy sustained at 12 hours following administration. During the trial, 10.4% of patients experienced at least one thrombotic event, the majority of which occurred in patients who delayed or did not restart anticoagulation during the follow-up period.6 Consistent with previous trial results in patients who are at increased risk of thrombosis, 15.7% of patients died during the trial.6
Ondexxya
Ondexxya (andexanet alfa) is a recombinant protein specifically designed to bind to FXa inhibitors and rapidly reverse their anticoagulant effect. Ondexxya is a modified form of the human FXa molecule, an enzyme that helps blood clot. Ondexxya works by acting as a decoy for oral and injectable FXa inhibitors, which target and bind to FXa, allowing them to exert their anticoagulant effect. When Ondexxya is given through an intravenous infusion to a patient with FXa inhibitor-related bleeding, it binds with high affinity to the FXa inhibitor, prevents it from inhibiting the activity of FXa and reverses the anticoagulant effects of the inhibitor.
Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca.
AstraZeneca's Ondexxya gets approval in Japan to reverse effect of anti blood clot drugs
Mar. 29, 2022 5:11 AM ET AstraZeneca PLC (AZN) By: Ravikash, SA News Editor
The Japanese Ministry of Health, Labour and Welfare approved AstraZeneca's (NASDAQ:AZN) Ondexxya for patients treated with the Factor Xa (FXa) inhibitors apixaban, rivaroxaban or edoxaban, when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding.
https://seekingalpha.com/symbol/AZN
https://www.astrazeneca.com/our-therapy-areas/rare-disease.html
Yescarta® (axicabtagene ciloleucel )
April 01, 2022
Yescarta® Receives U.S. FDA Approval as First CAR T-cell Therapy for Initial Treatment of Relapsed or Refractory Large B-cell Lymphoma (LBCL)
-- First LBCL Treatment to Improve Upon Standard of Care in Nearly 30 Years --
-- Landmark ZUMA-7 Study Demonstrated Patients on Yescarta Were 2.5 Times More Likely to Be Alive at Two Years Without Cancer Progression or Need for Additional Cancer Treatment --
-- Yescarta is First CAR T-cell Therapy to Receive NCCN Treatment Guideline Category 1 Recommendation --
SANTA MONICA, Calif.--(BUSINESS WIRE)-- Kite, a Gilead Company (Nasdaq: GILD), today announced the U.S. Food and Drug Administration (FDA) has approved Yescarta® (axicabtagene ciloleucel) CAR T-cell therapy for adult patients with large B-cell lymphoma that is refractory to first-line chemoimmunotherapy or that relapses within 12 months of first-line chemoimmunotherapy. Yescarta demonstrated a clinically meaningful and statistically significant improvement in event-free survival (EFS; hazard ratio 0.398; P< 0.0001) over the current standard of care (SOC) that has been in place for decades. EFS was determined by blinded central review and defined as the time from randomization to the earliest date of disease progression, commencement of new lymphoma therapy, or death from any cause. Additionally, 2.5 times more patients receiving Yescarta (40.5%) were alive at two years without disease progression or need for additional cancer treatment, after their one-time infusion of Yescarta vs. SOC (16.3%), and the median EFS was four-fold greater (8.3 months vs. 2.0 months) with Yescarta vs. SOC. Yescarta is also being reviewed by global regulatory authorities for additional indications inclusive of the ZUMA-7 patient population. ZUMA-7 is considered a landmark trial for being the first and largest trial of its kind, with the longest follow-up.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20220401005519/en/
Earlier this month, the National Comprehensive Cancer Network (NCCN) updated its Clinical Practice Guidelines in Oncology for B-cell Lymphomas to include Yescarta for “Relapsed disease <12 mo or Primary Refractory disease” under Diffuse Large B-cell Lymphoma (DLBCL) as a Category 1 recommendation. Yescarta is the first CAR T-cell therapy to receive a NCCN Category 1 recommendation. NCCN defines Category 1 as recommendations based upon high-level evidence with uniform NCCN consensus that the intervention is appropriate.
About ZUMA-7 Study
The FDA approval of Yescarta CAR T-cell therapy for adult patients with large B-cell lymphoma (LBCL) that is refractory to first-line chemoimmunotherapy or that relapses within 12 months of first-line chemoimmunotherapy is based on results from the ZUMA-7 study. Patients had not yet received treatment for relapsed or refractory lymphoma and were potential candidates for autologous stem cell transplant (ASCT). Results were presented in a Plenary session at the American Society of Hematology’s (ASH) Annual Meeting & Exposition in December 2021 and simultaneously published in the NewEngland Journal of Medicine (NEJM).
ZUMA-7 is a randomized, open-label, global, multicenter, Phase 3 study evaluating the safety and efficacy of Yescarta versus current standard of care (SOC) for second-line therapy (platinum-based salvage combination chemoimmunotherapy regimen followed by high-dose therapy [HDT] and ASCT in those who respond to salvage chemotherapy) in adult patients with relapsed or refractory LBCL within 12 months of first-line therapy. In the study, 359 patients in 77 centers around the world were randomized (1:1) to receive a single infusion of Yescarta or current SOC second-line therapy. The primary endpoint is event-free survival (EFS) as determined by blinded central review and defined as the time from randomization to the earliest date of disease progression per Lugano Classification, commencement of new lymphoma therapy, or death from any cause. Key secondary endpoints include objective response rate (ORR) and overall survival (OS). Additional secondary endpoints include patient reported outcomes (PROs) and safety.
Yescarta demonstrated a 2.5-fold increase in patients who were alive at two years and did not experience cancer progression or require the need for additional cancer treatment (40.5% vs. 16.3%) and a four-fold greater median EFS (8.3 mo. vs. 2.0 mo.) compared to SOC (hazard ratio 0.398; 95% CI: 0.308-0.514, P<0.0001). In addition to being the largest and longest study of its kind, ZUMA-7 study participants on the Yescarta arm did not receive additional bridging chemotherapy that could have potentially confounded results.
Nearly three times as many patients randomized to Yescarta ultimately received the definitive CAR T-cell therapy treatment (94%) versus those randomized to SOC (35%) who received on-protocol HDT+ASCT. More patients responded to Yescarta (ORR: 83% vs. 50%, odds ratio: 5.31 [95% CI: 3.1-8.9; P<0.0001) and achieved a complete response (CR) with Yescarta (CR rate: 65% vs. 32%) than with SOC. At a pre-specified interim analysis at the time of the primary EFS analysis, OS has not met the criteria for statistical significance, but favored Yescarta.
Fifty-five percent of patients in the SOC arm subsequently received CD19-directed CAR T-cell therapy off study.
In the study, Yescarta had a safety profile that was consistent with previous studies. Among the 168 Yescarta-treated patients evaluable for safety, Grade ≥3 cytokine release syndrome (CRS) and neurologic events were observed in 7% and 25% of patients, respectively. In the SOC arm, 83% of patients had high grade events, mostly cytopenias (low blood counts).
The Yescarta U.S. Prescribing Information has a BOXED WARNING for the risks of CRS and neurologic toxicities, and Yescarta is approved with a Risk Evaluation and Mitigation Strategy (REMS) due to these risks; see below for Important Safety Information.
About LBCL
Globally, LBCL is the most common type of non-Hodgkin lymphoma (NHL). In the United States, more than 18,000 people are diagnosed with LBCL each year. About 30-40% of patients with LBCL will need second-line treatment, as their cancer will either relapse (return) or become refractory (not respond) to initial treatment.
About Yescarta
Please see full Prescribing Information, including BOXED WARNING and Medication Guide.
YESCARTA is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of:
- Adult patients with large B-cell lymphoma that is refractory to first-line chemoimmunotherapy or that relapses within 12 months of first-line chemoimmunotherapy.
- Adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma, high grade B-cell lymphoma, and DLBCL arising from follicular lymphoma.
Limitations of Use: YESCARTA is not indicated for the treatment of patients with primary central nervous system lymphoma.
- Adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy. This indication is approved under accelerated approval based on response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).
- https://www.yescarta.com/
Further information is available at www.YescartaTecartusREMS.com or 1-844-454-KITE (5483).
For more information on Kite, please visit www.kitepharma.com.
U.S. Prescribing Information for Yescarta including BOXED WARNING, is available at www.kitepharma.com and www.gilead.com.
Kite, the Kite logo, Yescarta, Tecartus, XLP and GILEAD are trademarks of Gilead Sciences, Inc. or its related companies.
For more information on Kite, please visit the company’s website at www.kitepharma.com. Follow Kite on social media on Twitter (@KitePharma) and LinkedIn.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220401005519/en/
Source: Gilead Sciences, Inc.
Gilead granted FDA label expansion for Yescarta in B-cell lymphoma
Apr. 01, 2022 6:30 PM ET Gilead Sciences, Inc. (GILD)
By: Dulan Lokuwithana, SA News Editor7 Comments
- The U.S. Food and Drug Administration (FDA) has approved the CAR T-cell therapy Yescarta as an initial treatment for adult patients with relapsed or refractory large B-cell lymphoma (LBCL), Kite, a unit of Gilead (NASDAQ:GILD) announced on Friday.
- https://seekingalpha.com/news/3820134-gilead-granted-fda-label-expansion-for-yescarta-in-b-cell-lymphoma
- https://seekingalpha.com/symbol/GILD
- https://www.gilead.com/
Etrasimod
Pfizer Announces Positive Top-line Results from Yearlong Phase 3 Trial of Etrasimod in Ulcerative Colitis, Underscoring Best-in-Class Potential
Tuesday, March 29, 2022 - 06:45am
ELEVATE UC 52 met the co-primary endpoints of clinical remission at both weeks 12 and 52 and all key secondary endpoints
- Etrasimod demonstrated a safety profile consistent with previous studies
NEW YORK--(BUSINESS WIRE)--
Pfizer Inc. (NYSE: PFE) today announced positive top-line results from a second Phase 3 study of etrasimod, an investigational, oral, once-a-day, selective sphingosine 1-phosphate (S1P) receptor modulator in development for the treatment of moderately to severely active ulcerative colitis (UC). The positive 12- and 52-week results from ELEVATE UC 52 follow the recent announcement of positive 12-week findings from the ELEVATE UC 12 trial on March 23.
In this 52-week study, also known as ELEVATE UC 52, etrasimod patients achieved statistically significant improvements in the co-primary endpoints of clinical remission at weeks 12 and 52 when compared to placebo. Statistically significant improvements were attained in all key secondary endpoints at both 12 and 52 weeks. Etrasimod demonstrated a safety profile consistent with previous studies, including the Phase 2 OASIS trial.
The global Phase 3 multi-center, randomized, double-blind, placebo-controlled study enrolled 433 UC patients who had previously failed or were intolerant to at least one conventional, biologic, or Janus kinase (JAK) inhibitor therapy. Participants received etrasimod 2 mg or placebo once-daily. ELEVATE UC 52 utilized a treat-through design in which patients were eligible to continue with their randomized treatment independent of whether they reached the objective criteria of clinical response at week 12.
“For patients suffering with moderate to severe ulcerative colitis, these most recent data further demonstrate the substantial potential benefits of this medicine and clearly confirm its ability to achieve significant induction of remission at 12 weeks and now clinical remission at week 52. These data underscore etrasimod’s potential, if approved, as a best-in-class therapy,” said Michael Corbo, Chief Development Officer, Inflammation & Immunology, Pfizer Global Product Development. “Etrasimod can potentially provide a new, once-daily, oral option with a rapid onset of action and without first dose titration. Further, we believe the treat-through design of the ELEVATE UC 52 study more accurately reflects a real-world treatment approach than the re-randomization design often used in UC clinical trials.”
Full results from the studies will be submitted for future scientific publication and presentation. These data, along with results from ELEVATE UC 12 and the long-term extension from these two trials (ELEVATE UC OLE), are expected to form the basis for planned future regulatory filings. Pfizer expects to initiate regulatory filings later this year. Additional information about the studies can be found at www.clinicaltrials.gov under the identifiers NCT03945188, NCT03996369, and NCT03950232.
Etrasimod was developed by Arena Pharmaceuticals, which was recently acquired by Pfizer.
About Etrasimod
Etrasimod is an oral, once-a-day, selective sphingosine 1-phosphate (S1P) receptor modulator designed for optimized pharmacology and engagement of S1P receptors 1, 4, and 5. It is being investigated for a range of immuno-inflammatory diseases including ulcerative colitis, Crohn’s Disease, atopic dermatitis, eosinophilic esophagitis, and alopecia areata.
In a Phase 2, randomized, placebo-controlled, dose-ranging study (OASIS) in moderate to severe UC patients, most patients who achieved clinical response, clinical remission, or endoscopic improvement at week 12 experienced sustained or improved effects up to week 46, with etrasimod 2 mg in the open-label extension. Etrasimod also demonstrated a favorable benefit/risk profile, consistent with safety findings reported in the double-blind portion of OASIS.
About ELEVATE UC 52
ELEVATE UC 52 is one of two pivotal trials that are part of the ELEVATE UC global Phase 3 registrational program. ELEVATE UC 52 is a 2:1 randomized, double-blind, placebo-controlled trial to assess the efficacy and safety of etrasimod 2 mg once-daily in participants with moderately-to-severely active UC. This is a one-year trial evaluating clinical remission at 12 weeks, or induction, and at 52 weeks. ELEVATE UC 52 utilized a treat-through design in which patients were eligible to continue with etrasimod independent of whether they reached clinical response at week 12.
The primary objective of this trial is to assess the safety and efficacy of etrasimod on clinical remission after both 12 and 52 weeks. The primary endpoint is based on the 3-domain, modified Mayo score. Key secondary measures include the efficacy of etrasimod, symptomatic remission, endoscopic improvement, corticosteroid-free remission, and mucosal healing in these participants at time points up to 52 weeks of treatment.
In addition, to learn more, please visit us on www.pfizer.com and follow us on Twitter at @Pfizer and @Pfizer_News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220329005198/en/
Source: Pfizer Inc.
Pfizer etrasimod helps improve remission in ulcerative colitis in 2nd phase 3 trial
Mar. 30, 2022 5:20 AM ET
By: Ravikash, SA News Editor
Pfizer reported results from a second phase 3 trial of etrasimod to treat patients with moderately to severely active ulcerative colitis (UC), an inflammatory bowel disease.
https://seekingalpha.com/symbol/PFE
Retevmo® (selpercatinib)
Lilly Presents Updated Data on Retevmo® (selpercatinib) in Advanced RET Fusion-Positive Non-Small-Cell Lung Cancer (NSCLC) at the 2022 European Lung Cancer Congress
April 1, 2022Download PDF
INDIANAPOLIS, April 1, 2022 /PRNewswire/ -- Eli Lilly and Company (NYSE: LLY) today announced updated data from the Phase 1/2 LIBRETTO-001 trial of Retevmo® (selpercatinib 40 mg & 80 mg capsules) in patients with RET fusion-positive non-small cell lung cancer (NSCLC). Retevmo (marketed as Retsevmo® outside of the U.S.) is a selective and potent RET kinase inhibitor that is approved in multiple countries including the United States for treatment of adult patients with metastatic rearranged during transfection (RET) fusion-positive NSCLC, and the treatment of adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) who require systemic therapy, or advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate). These data were presented at the European Lung Cancer (ELCC) 2022 (poster 27p).
"The LIBRETTO trial provides the largest set of clinical data for a RET inhibitor and these results continue to demonstrate evidence of meaningful clinical outcomes for patients with metastatic RET fusion-positive NSCLC treated with Retevmo, including those with difficult-to-treat brain metastases," said David Hyman, M.D. chief medical officer, oncology at Lilly. "We are continuing to build on the robust body of evidence supporting Retevmo, including through an ongoing randomized Phase 3 confirmatory study, with a planned readout in 2023."
The updated analysis utilized a June 15, 2021, data cut-off and included 355 patients who were eligible for efficacy analysis, 247 of which were previously treated with at least one line of platinum chemotherapy and 69 of which were treatment-naïve. Patients who were previously treated with at least one line of platinum chemotherapy received a median of two prior treatment regimens (range: 1-15), with 58% having received anti-PD-1 or anti-PD-L1 therapy. Responses are based on independent review committee (IRC) assessment.
Among 247 patients previously treated with platinum chemotherapy, the confirmed objective response rate (ORR) was 61.1% (95% CI: 54.7-67.2%) and among 69 treatment-naïve patients, the confirmed ORR was 84.1% (95% CI: 73.3-91.8%). Twenty-six patients had measurable central nervous system (CNS) metastases at baseline and treatment with Retevmo resulted in a CNS ORR of 84.6%, with 22 patients having a confirmed best response of complete response or partial response.
At a median follow-up of approximately two years in both the treatment-naïve and platinum-chemotherapy pretreated populations, median duration of response (DoR) is estimated at 20.2 (55.2% censoring rate; 20.3 months median duration of follow-up) and 28.6 (60.9% censoring rate; 21.2 months median duration of follow-up) months, respectively and median progression free survival (PFS) is estimated at 22.0 (53.6% censoring rate; 21.9 months median duration of follow-up) and 24.9 (55.9% censoring rate; 24.7 months median duration of follow-up) months, respectively. Of the 26 patients with measurable CNS disease, Retevmo treatment resulted in a median intracranial PFS of 19.4 months. These median estimates remain immature.
Safety among patients in this cohort was consistent with the known safety profile of Retevmo. In the safety population (all NSCLC patients that received at least one dose of Retevmo, N=356), the most common adverse events (AEs in ≥25% of patients) were dry mouth, diarrhea, hypertension, increased ALT/AST, peripheral edema, constipation, rash, headache, and fatigue. Thirty-four patients discontinued due to an adverse event (10%), eleven (3%) of which were deemed related to Retevmo.
A global, randomized, Phase 3 trial is currently recruiting and will compare treatment with Retevmo to the current standard of care in the first-line treatment of advanced or metastatic RET fusion-positive NSCLC.
Retevmo was the first RET inhibitor to receive Accelerated Approval from the U.S. Food and Drug Administration (FDA) in May 2020 and was the first approved by the European Commission in February 2021. Retevmo was approved under the FDA's Accelerated Approval regulations based on the LIBRETTO-001 Phase 1/2 trial's endpoints of objective response rate and duration of response. Continued approval for these indications may be contingent upon verification and description of clinical benefit in confirmatory trials.
About LIBRETTO-001
The Phase 1/2 LIBRETTO-001 trial is the largest clinical trial of patients with RET-driven cancers treated with a RET inhibitor. The trial, which spans 16 countries and 89 sites, included a dose escalation phase (Phase 1) and a dose expansion phase (Phase 2). The primary objective was to determine ORR by independent review committee (IRC) and key secondary objectives included DoR, CNS ORR & DOR, safety and PFS.
LIBRETTO-001 continues to enroll patients with RET-altered tumors beyond lung cancer.
About Retevmo® (selpercatinib)
Retevmo (selpercatinib, formerly known as LOXO-292) (pronounced ret- tév-mo) is a selective and potent RET kinase inhibitor. Retevmo may affect both tumor cells and healthy cells, which can result in side effects. RET-driver alterations are predominantly mutually exclusive from other oncogenic drivers. Retevmo is an U.S. FDA-approved oral prescription medicine, 120 mg or 160 mg dependent on weight (<50 kg or ≥50 kg, respectively), taken twice daily until disease progression or unacceptable toxicity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials.
Please see full Prescribing Information for Retevmo.
To learn more, visit Lilly.com and Lilly.com/newsroom or follow us on Facebook, Instagram, Twitter and LinkedIn. P-LLY
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SOURCE Eli Lilly and Company
Lilly reports new data from phase 1/2 trial of Retevmo in advanced lung cancer
Apr. 01, 2022 8:01 AM ET Eli Lilly and Company (LLY) By: Ravikash, SA News Editor
Eli Lilly (NYSE:LLY) reported updated data from a phase 1/2 trial of Retevmo (selpercatinib 40 mg & 80 mg capsules) in patients with RET fusion-positive non-small cell lung cancer (NSCLC).
https://seekingalpha.com/symbol/LLY
biotecMAX™ Feb/Mar 2022 Insight
Evusheld, formerly known as AZD7442,
Evusheld long-acting antibody combination approved in the EU for pre-exposure prophylaxis (prevention) of COVID-19 in a broad population
PUBLISHED28 March 2022
28 March 2022 07:00 BST
Evusheld significantly reduced the risk of developing symptomatic COVID-19 in PROVENT Phase III trial, with protection lasting at least six months
Evusheld retains neutralising activity against the Omicron BA.2 subvariant, now the dominant strain in Europe
AstraZeneca's Evusheld (tixagevimab co-packaged with cilgavimab), a long-acting antibody combination, has been granted marketing authorisation in the European Union (EU) for the pre-exposure prophylaxis (prevention) of COVID-19 in a broad population of adults and adolescents aged 12 years and older weighing at least 40 kg.
The approval by the European Commission was based on results from the Evusheld clinical development programme, including data from the PROVENT Phase III pre-exposure prophylaxis trial which showed a 77% reduction in the risk of developing symptomatic COVID-19 compared to placebo at the primary analysis and an 83% reduction at a six-month median analysis, with protection from the virus lasting at least six months.1-3 Evusheld was generally well-tolerated in the trial.1-3
Christoph D. Spinner, MD, Consulting Physician Infectious Diseases and Pandemic Officer at the University Hospital Rechts der Isar and adjunct teaching professor at the Technical University of Munich, Munich, Germany, said: “Increasing COVID-19 cases, driven by the highly-transmissible BA.2 subvariant, and withdrawal of several pandemic public health measures make it important to protect vulnerable populations, such as the immunocompromised, from SARS-CoV-2 infection. The authorisation of Evusheld for a broad population will allow health authorities in the EU to identify the populations who are most at-risk and need additional protection.”
Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, AstraZeneca, said: “The EU approval represents an important milestone in our efforts to help prevent COVID-19, and we will continue to work with governments across Europe to make Evusheld available as quickly as possible. Evusheld has the potential to provide long-lasting protection against COVID-19 for a broad population of individuals, including those who aren’t adequately protected by a COVID-19 vaccine, as well as those at increased risk of exposure.”
The recommended dose of Evusheld in Europe is 150mg of tixagevimab and 150mg of cilgavimab, administered as two separate sequential intramuscular (IM) injections.
There is a growing body of evidence from multiple independent in vitro and in vivo (animal model) studies supporting the potential of Evusheld to protect against the BA.1, BA.1.1 and BA.2 Omicron SARS-CoV-2 subvariants in circulation around the world.4-6 New data from Washington University School of Medicine demonstrated Evusheld retained potent neutralising activity against the emerging and highly transmissible BA.2 subvariant, which is the dominant strain in many European countries and currently accounts for over 60% of COVID-19 infections in Europe.6,7 This study also showed Evusheld reduced viral burden and limited inflammation in the lungs (in vivo) across all Omicron variants.6
Evusheld is authorised for emergency use for pre-exposure prophylaxis of COVID-19 in the US and has been granted conditional marketing authorisation by the Medicines and Healthcare products Regulatory Agency (MHRA) in Great Britain for pre-exposure prophylaxis of COVID-19. Additionally, there are a number of countries across Europe that have agreements in place to provide Evusheld.
People who are not adequately protected by a COVID-19 vaccine may particularly benefit from pre-exposure prophylaxis with Evusheld.8-12 This population includes about three million people in the EU who are immunocompromised such as people with cancer or transplant patients or anyone taking immunosuppressive medicines.13 People at increased risk of exposure to the SARS-CoV-2 virus could also benefit from protection with Evusheld.14
Evusheld is the only long-acting antibody combination with positive Phase III data in the prevention and treatment of COVID-19.2,15 AstraZeneca is progressing with filings around the globe for potential emergency use authorisation or marketing approval of Evusheld in both COVID-19 prophylaxis and treatment.
Evusheld
Evusheld, formerly known as AZD7442, is a combination of two long-acting antibodies - tixagevimab (AZD8895) and cilgavimab (AZD1061) - derived from B-cells donated by convalescent patients after SARS-CoV-2 infection. Discovered by Vanderbilt University Medical Center and licensed to AstraZeneca in June 2020, the human monoclonal antibodies bind to distinct sites on the SARS-CoV-2 spike protein16 and were optimised by AstraZeneca with half-life extension and reduction of Fc effector function. The half-life extension more than triples the durability of its action compared to conventional antibodies;17-19 data from the PROVENT Phase III trial show protection lasting at least six months.1,3 The reduced Fc effector function aims to minimise the risk of antibody-dependent enhancement of disease - a phenomenon in which virus-specific antibodies promote, rather than inhibit, infection and/or disease.20
Evusheld has been granted marketing authorisation in the European Union and Great Britain for pre-exposure prophylaxis (prevention) of COVID-19. In the United States, Evusheld is authorised for emergency use for pre-exposure prophylaxis of COVID-19 in people with moderate to severe immune compromise due to a medical condition or immunosuppressive medications and who may not mount an adequate immune response to COVID-19 vaccination, as well as those individuals for whom COVID-19 vaccination is not recommended due to a history of severe adverse reaction to a COVID-19 vaccine. This population includes people with blood cancers or other cancers being treated with chemotherapy, and those taking medications after an organ transplant or who are taking immunosuppressive drugs for conditions including multiple sclerosis and rheumatoid arthritis.1 It is also authorised for use and being supplied in several other countries around the world.
The primary data supporting the Evusheld authorisations are from the ongoing PROVENT Phase III pre-exposure prevention trial, which showed a statistically significant reduction in the risk of developing symptomatic COVID-19 compared to placebo, with protection from the virus continuing for at least six months (77% at primary analysis [8/3441 (0.2%) Evusheld arm, 17/1731 (1.0%) placebo arm]; 83% at median six month analysis [11/3441 (0.3%) Evusheld arm, 31/1731 (1.8%) placebo arm]).1-3 Follow-up is ongoing to establish the full duration of protection provided by Evusheld.
In October 2021, AstraZeneca announced positive high-level results from the TACKLE Phase III outpatient treatment trial in which a 600mg IM dose of Evusheld was generally well-tolerated. AstraZeneca is discussing the TACKLE mild-to-moderate COVID-19 treatment data with health authorities.
Evusheld was generally well-tolerated in the trials.
Evusheld is being developed with support from the US government, including federal funds from the Department of Health and Human Services; Office of the Assistant Secretary for Preparedness and Response; Biomedical Advanced Research and Development Authority in partnership with the Department of Defense; Joint Program Executive Office for Chemical, Biological, Radiological and Nuclear Defense, under Contract No. W911QY-21-9-0001.
Under the terms of the licensing agreement with Vanderbilt, AstraZeneca will pay single-digit royalties on future net sales
Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca.
AstraZeneca's Evusheld gets approval in EU to prevent COVID-19
Mar. 28, 2022 5:28 AM ET
By: Ravikash, SA News Editor
- The European Commission approved AstraZeneca's (NASDAQ:AZN) antibody cocktail Evusheld for preventing COVID-19 in people aged 12 years and older weighing at least 40 kg.
- The approval follows the recommendation made by a panel of the European Medicines Agency last week.
- https://seekingalpha.com/news/3817851-astrazenecas-evusheld-gets-approval-in-eu-to-prevent-covid-19
- https://seekingalpha.com/symbol/AZN
READY-TO-USE EPHEDRINE INJECTION VIALS
NEVAKAR INJECTABLES ANNOUNCES THE LAUNCH OF READY-TO-USE EPHEDRINE INJECTION VIALS
- Published on: March 27, 2022
- Press Releases
Bridgewater, NJ, USA, March 28, 2022 – Nevakar Injectables Inc., a biopharmaceutical company developing multiple sterile injectable products for use in the critical and ambulatory care settings, announced today the launch of its ready-to-use Ephedrine Sulfate Injection (“Ephedrine”), a formulation pre-diluted to a 5mg/mL concentration and supplied in a 10mL vial. The product will be marketed and sold in the U.S. by the Par Sterile Products (“Par”) business of Endo International plc (“Endo”) (NASDAQ: ENDP). Ephedrine is a widely used injectable vasopressor agent indicated for the treatment of clinically important hypotension occurring during surgery.
“Ephedrine serves a very important role and is used by anesthesiologists, nurse anesthetists, and other health care professionals to treat hypotension during surgery,” stated Sriram Ramanathan, MS, MBA, Chief Executive Officer of Nevakar Injectables. “Our convenient and ready-to-use Ephedrine vial fills a critical need in the marketplace by reducing the potential for dilution errors and simplifying the drug preparation process. Working together with our partners at Par, we are looking forward to additional regulatory filings, FDA approvals, and commercial launches associated with the products that Endo licensed from Nevakar in 2018,” commented Mr. Ramanathan.
“We’re pleased to mark a milestone in our partnership with Nevakar with this launch—a first step in fulfilling our joint promise to serve our hospital customers and their patients through a portfolio of enhanced injectable products,” said Scott Sims, Senior Vice President and General Manager, Sterile Products at Endo.
About Nevakar Injectables, Inc.
Nevakar Injectables, Inc. is a wholly owned subsidiary of Nevakar, Inc., a fully integrated privately held, late-stage biopharmaceutical company with an extensive portfolio of products in the ophthalmic and injectable areas. Nevakar Injectables is developing a broad portfolio of
injectable products for use in the hospital and ambulatory care settings. The Company has active programs in critical patient care, acute pain management, long acting injectables, and hospital injectables. For additional information please visit www.nevakarinjectables.com.
About Endo
Endo (NASDAQ: ENDP) is a specialty pharmaceutical company committed to helping everyone we serve live their best life through the delivery of quality, life-enhancing therapies. Our decades of proven success come from passionate team members around the globe collaborating to bring the best treatments forward. Together, we boldly transform insights into treatments benefiting those who need them, when they need them. Learn more at www.endo.com or connect with us on LinkedIn.
About Par Pharmaceutical
Par Pharmaceutical develops, manufactures and markets safe, innovative and cost-effective generic pharmaceutical and branded injectable products that help improve patient quality of life. Par, among the top leaders in the U.S. generics industry, possesses an expanding portfolio that includes sterile injectables, alternative dosage forms and other differentiated products. Par Pharmaceutical is an Endo company. Learn more at www.parpharm.com.
Nevakar launches ready-to-use Ephedrine injection for low blood pressure
Mar. 28, 2022 8:32 AM ET
By: Ravikash, SA News Editor
- Privately held Nevakar launched ready-to-use Ephedrine Sulfate injection, a formulation pre-diluted to a 5mg/mL concentration and supplied in a 10mL vial.
- The product will be marketed and sold in the U.S. by the Par Sterile Products business of Endo International (NASDAQ:ENDP).
- https://seekingalpha.com/news/3817945-nevakar-launches-ready-to-use-ephedrine-injection-for-low-blood-pressure
- https://seekingalpha.com/symbol/ENDP
- https://www.endo.com/
MARCH 28, 2022
Endo Begins Shipment of Premixed Ephedrine Sulfate Injection in Ready-to-Use Vials
(PRNewsfoto/Endo International plc) NEWS PROVIDED BY Endo International plc Mar 28, 2022, 07:30 ET
Xenpozyme® (olipudase alfa)
PRESS RELEASESMarch 28 2022
Press Release: Xenpozyme® (olipudase alfa) approved in Japan, first and only approved therapy indicated to treat acid sphingomyelinase deficiency
Xenpozyme® (olipudase alfa) approved in Japan, first and only approved therapy indicated to treat acid sphingomyelinase deficiency
- Xenpozyme represents first Sanofi therapy to be approved under the SAKIGAKE ‘fast-track’ designation
- Approval based on positive results from two separate clinical trials in children and adults demonstrating improvement in lung function (as measured by DLco) and reduction of spleen and liver volumes
Paris, March 28, 2022. The Japanese Ministry of Health, Labor, and Welfare (MHLW) has granted marketing authorization for Xenpozyme® (olipudase alfa) for the treatment of adult and pediatric patients with non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD), a rare, progressive, and potentially life-threatening genetic disease. Xenpozyme is currently the only approved treatment for ASMD and represents Sanofi’s first therapy to be approved under the SAKIGAKE (or “pioneer”) designation, which is the Japanese government’s regulatory fast-track pathway to promote research and development of innovative new medical products addressing urgent unmet medical needs.
Xenpozyme is a recombinant human acid sphingomyelinase enzyme developed to replace deficient or defective acid sphingomyelinase (ASM), an enzyme that allows for the breakdown of the lipid sphingomyelin. Accumulation of sphingomyelin in cells can cause harm to the lungs, spleen, and liver, as well as other organs, potentially leading to early death.
The approval of Xenpozyme in Japan is based on positive results from the ASCEND and ASCEND-Peds clinical trials, showing that Xenpozyme provided improvement in lung function (as measured by diffusing capacity of the lung for carbon monoxide, or DLco) and reduction of spleen and liver volumes, with a well-tolerated safety profile in adults and children with ASMD. These data were presented at the American Society of Human Genetics (ASHG) 2020 Virtual Meeting.
Xenpozyme has been evaluated in children and adults to treat non-CNS manifestations of ASMD type A/B and ASMD type B. Xenpozyme has not been studied in patients with ASMD type A.
Historically known as Niemann-Pick disease types A, A/B, and B, ASMD is a genetically-based, progressive, and potentially life-threatening disease. ASMD represents a spectrum of disease, with two types that may represent opposite ends of a continuum referred to as ASMD type A and ASMD type B. ASMD type A/B is an intermediate form that includes varying degrees of CNS involvement. Until now, no approved therapies for ASMD have been available anywhere in the world.
Outside of Japan, olipudase alfa is being evaluated by regulatory authorities around the world. A Biologics License Application (BLA) for olipudase alfa was accepted for Priority Review by the U.S. Food and Drug Administration (FDA), with a decision expected early Q3 2022. The European Medicines Agency (EMA) has awarded olipudase alfa the PRIority MEdicines (PRIME) designation, and a decision is anticipated in the second half of 2022.
Sanofi's Xenpozyme gets approval in Japan for rare genetic disease
Mar. 28, 2022 6:23 AM ET
By: Ravikash, SA News Editor
- The Japanese Ministry of Health, Labor, and Welfare (MHLW) approved Sanofi's (NASDAQ:SNY) Xenpozyme (olipudase alfa) to treat adult and pediatric patients with non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD).
- https://seekingalpha.com/news/3817863-sanofis-xenpozyme-gets-approval-in-japan-for-rare-genetic-disease
- https://seekingalpha.com/symbol/SNY
- https://www.sanofi.com/en
- https://www.sanofi.com/en/science-and-innovation/research-and-development/rd-pipeline
DUPIXENT® (DUPILUMAB)
LATE-BREAKING PHASE 3 DATA AT AAD 2022 SHOW DUPIXENT® (DUPILUMAB) SIGNIFICANTLY IMPROVED SIGNS AND SYMPTOMS OF PRURIGO NODULARIS
TARRYTOWN, N.Y. and PARIS, March 26, 2022 /PRNewswire/ --
Dupixent significantly reduced itch at 12 weeks, and at 24 weeks nearly three times as many Dupixent patients experienced clinically meaningful reductions in itch and skin lesions
There are currently no approved systemic treatments for prurigo nodularis; regulatory filings for Dupixent in prurigo nodularis are planned in the first half of 2022
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi announced that detailed positive results from the Phase 3 PRIME2 trial evaluating the efficacy and safety of Dupixent® (dupilumab) were presented today in a late-breaking session at the American Academy of Dermatology (AAD) 2022 Annual Meeting. The companies previously announced topline results from PRIME2 and a second trial, called PRIME, investigating the use of Dupixent in adults with uncontrolled prurigo nodularis. In both trials, Dupixent significantly reduced itch and skin lesions compared to placebo. In total, 21 scientific abstracts evaluating the safety and efficacy of Dupixent in patients with atopic dermatitis in different age groups, as well as investigational indications of prurigo nodularis and chronic spontaneous urticaria will be presented at the congress.
"Prurigo nodularis is a relentless and often misunderstood itchy skin disease that leaves many patients with uncontrolled symptoms such as unbearable itch and painful skin lesions, along with a significantly impaired quality of life that should not be underestimated," said Gil Yosipovitch, M.D., Professor of Dermatology at the Miller School of Medicine at University of Miami, Director of the Miami Itch Center and principal investigator of the trial. "These positive results are the first time a Phase 3 trial has demonstrated that targeting key drivers of type 2 inflammation, IL-4 and IL-13, with dupilumab significantly improved itch and skin lesions in this highly burdensome disease."
The randomized, placebo-controlled PRIME2 trial met its primary and all key secondary endpoints, with data presented at AAD 2022 showing:
- 37% of Dupixent patients experienced a clinically meaningful reduction in itch from baseline compared to 22% of placebo patients (p=0.0216) at week 12, the primary endpoint.
- Nearly three times as many Dupixent patients experienced a clinically meaningful reduction in itch from baseline at week 24: 58% of Dupixent patients compared to 20% of placebo patients (p<0.0001).
- Nearly three times as many Dupixent patients achieved clear or almost clear skin at week 24: 45% of Dupixent patients compared to 16% of placebo patients (p<0.0001).
The safety results of the trial were generally consistent with the known safety profile of Dupixent in its approved dermatology indications. For the 24-week treatment period, overall rates of adverse events were generally similar between Dupixent and placebo groups (57% Dupixent, 51% placebo). Adverse events that were more commonly (≥5%) observed with Dupixent were herpes viral infections (7% Dupixent, 0% placebo). A lower rate of skin infections was observed with Dupixent (5% Dupixent, 9% placebo). Additionally, 3% of Dupixent patients and 30% of placebo patients discontinued prior to week 24.
Results from the confirmatory PRIME trial will be presented at an upcoming medical congress. Data from both trials will form the basis of regulatory submissions around the world for Dupixent in prurigo nodularis, which are planned to begin in the first half of 2022.
The potential use of Dupixent in prurigo nodularis is currently under clinical development, and the safety and efficacy have not been fully evaluated by any regulatory authority.
About the Trial
PRIME2, part of the LIBERTY-PN PRIME clinical program, is a randomized, Phase 3, double-blind, placebo-controlled trial that evaluated the efficacy and safety of Dupixent in 160 adults with prurigo nodularis inadequately controlled with topical prescription therapies or with whom those therapies were not advisable. During the 24-week treatment period, patients received Dupixent or placebo every two weeks with or without topical treatments (low- or medium-dose topical corticosteroids or topical calcineurin inhibitors were continued if patients were using these treatments at randomization).
The primary endpoint evaluated the proportion of patients with clinically meaningful improvement in itch at week 12 (measured by a ≥4-point reduction in Worst-Itch Numeric Rating Scale [WI-NRS] of 0-10). Key secondary endpoints included the proportion of patients with clinically meaningful improvement in itch at week 24 and the proportion of patients with clear or almost clear skin at week 24 (measured by a score of 0 or 1 on the Investigator's Global Assessment PN-Stage [IGA PN-S] 0-4 scale).
About Dupixent
Dupixent, which was invented using Regeneron's proprietary VelocImmune® technology, is a fully human monoclonal antibody that inhibits the signaling of the interleukin-4 (IL-4) and interleukin-13 (IL-13) pathways and is not an immunosuppressant. IL-4 and IL-13 are key and central drivers of the type 2 inflammation that plays a major role in atopic dermatitis, asthma and chronic rhinosinusitis with nasal polyposis (CRSwNP).
Dupixent is currently approved in the U.S., Europe, Japan and other countries around the world for use in specific patients with moderate-to-severe atopic dermatitis, as well as certain patients with asthma or CRSwNP in different age populations. Dupixent is also approved in one or more of these indications in more than 60 countries around the world and more than 400,000 patients have been treated globally.
U.S. Indications
DUPIXENT is a prescription medicine used:
- to treat adults and children 6 years of age and older with moderate-to-severe atopic dermatitis (eczema) that is not well controlled with prescription therapies used on the skin (topical), or who cannot use topical therapies. DUPIXENT can be used with or without topical corticosteroids. It is not known if DUPIXENT is safe and effective in children with atopic dermatitis under 6 years of age.
- with other asthma medicines for the maintenance treatment of moderate-to-severe eosinophilic or oral steroid dependent asthma in adults and children 6 years of age and older whose asthma is not controlled with their current asthma medicines. DUPIXENT helps prevent severe asthma attacks (exacerbations) and can improve your breathing. DUPIXENT may also help reduce the amount of oral corticosteroids you need while preventing severe asthma attacks and improving your breathing. DUPIXENT is not used to treat sudden breathing problems. It is not known if DUPIXENT is safe and effective in children with asthma under 6 years of age.
- with other medicines for the maintenance treatment of chronic rhinosinusitis with nasal polyposis (CRSwNP) in adults whose disease is not controlled. It is not known if DUPIXENT is safe and effective in children with chronic rhinosinusitis with nasal polyposis under 18 years of age.
- https://www.dupixent.com/
Please see accompanying full Prescribing Information including Patient Information.
For additional information about the company, please visit www.regeneron.com
View original content:https://www.prnewswire.com/news-releases/late-breaking-phase-3-data-at-aad-2022-show-dupixent-dupilumab-significantly-improved-signs-and-symptoms-of-prurigo-nodularis-301511154.html
SOURCE Regeneron Pharmaceuticals, Inc.
https://seekingalpha.com/symbol/REGN
https://seekingalpha.com/symbol/SNY
Jyseleca® (Filgotinib 200mg and 100mg tablets)
Jyseleca® approved in Japan for ulcerative colitis
01-Gilead Japan Jyseleca UC approval_250322_FINAL
Approval of additional indication based on Phase 2b/3 SELECTION study in patients with moderate-to-severe ulcerative colitis
Mechelen, Belgium; 28 March 2022, 08.01 CET; Galapagos NV (Euronext & NASDAQ: GLPG) reports that Gilead Sciences K.K. (Tokyo, Japan), Eisai Co., Ltd. (Tokyo, Japan) and EA Pharma Co., Ltd. (Tokyo, Japan) today announced the approval by the Japanese Ministry of Health, Labour and Welfare (MHLW), of a second indication for Jyseleca (filgotinib), a once-daily, oral, JAK1 preferential inhibitor, for the treatment of patients with moderate-to-severe active ulcerative colitis (UC).
The approval of this second indication for Jyseleca in Japan is based on data from the randomized, double-blind, placebo-controlled phase 2b/3 SELECTION study. This study evaluated the efficacy and safety of Jyseleca for induction and maintenance of remission in patients with moderately to severely active ulcerative colitis who had an inadequate response with, lost response to, or were intolerant to either conventional therapy or a biologic agent. The study showed that Jyseleca 200mg was well-tolerated and efficacious as induction and maintenance therapy with no new safety findings reported. Jyseleca was approved in Japan in September 2020 for the treatment of rheumatoid arthritis (RA), including the prevention of structural joint damage, in patients who had inadequate response to conventional therapies.
Gilead Japan will hold the marketing authorization of Jyseleca in Japan and will be responsible for product supply. Eisai will be responsible for product distribution, and together with EA Pharma, its subsidiary focused on gastrointestinal diseases, will jointly commercialize the medicine to make it available to physicians and patients across the country.
About filgotinib
Filgotinib is approved and marketed as Jyseleca (200mg and 100mg tablets) in the European Union, Great Britain, and Japan for the treatment of adults with moderate to severe active rheumatoid arthritis (RA) who have responded inadequately or are intolerant to one or more disease modifying anti-rheumatic drugs (DMARDs). Filgotinib may be used as monotherapy or in combination with methotrexate (MTX). Filgotinib is also approved and marketed as Jyseleca (200mg tablets) in the European Union, Great Britain and Japan for the treatment of adult patients with moderately to severely active UC who have had an inadequate response with, lost response to, or were intolerant to either conventional therapy or a biologic agent. The European Summary of Product Characteristics for filgotinib, which includes contraindications and special warnings and precautions, is available at www.ema.europa.eu. The Great Britain Summary of Product Characteristics for filgotinib can be found at www.medicines.org.uk/emc and the Northern Ireland Summary of Product Characteristics for filgotinib can be found at www.emcmedicines.com/en-GB/northernireland. The interview form from the Japanese Ministry of Health, Labour and Welfare is available at www.info.pmda.go.jp. A global Phase 3 program with filgotinib is ongoing in Crohn’s Disease. More information about clinical trials can be accessed at https://www.clinicaltrials.gov.
Jyseleca® is a trademark of Galapagos NV and Gilead Sciences, Inc. or its related companies.
https://www.jyseleca.eu/en/landing.html
About the filgotinib collaboration
Gilead and Galapagos NV are collaborative partners in the global development and commercialization of filgotinib. Galapagos is responsible for the commercialization of filgotinib in Europe, while Gilead remains responsible for filgotinib outside of Europe, including in Japan, where filgotinib is co-marketed with Eisai.
More information at www.glpg.com.
Gilead, Galapagos' Jyseleca gets approval in Japan for expanded use in ulcerative colitis
Mar. 28, 2022 6:41 AM ET
Galapagos NV (GLPG), GILDESALF, ESALY
By: Ravikash, SA News Editor
- The Japanese Ministry of Health, Labour and Welfare (MHLW) approved Gilead Sciences (NASDAQ:GILD), Galapagos (NASDAQ:GLPG) and Eisai's (OTCPK:ESALF) drug Jyseleca (filgotinib) to treat patients with moderate-to-severe active ulcerative colitis (UC), an inflammatory bowel disease.
- https://seekingalpha.com/news/3817867-gilead-galapagos-jyseleca-gets-approval-in-japan-for-expanded-use-in-ulcerative-colitis
- https://seekingalpha.com/symbol/GLPG
- https://seekingalpha.com/symbol/ESALF
- https://seekingalpha.com/symbol/GILD
- https://www.jyseleca.eu/en/landing.html
AYVAKYT® (avapritinib)
Blueprint Medicines' AYVAKYT® (avapritinib) Receives European Commission Approval for the Treatment of Adults with Advanced Systemic Mastocytosis
March 25, 2022 at 10:15 AM EDT
-- AYVAKYT is the first approved therapy in the European Union designed to selectively target the KIT D816V mutation, the primary driver of disease --
-- Advanced Systemic Mastocytosis (AdvSM) is a debilitating disease characterized by damage across multiple organ systems, reduced overall survival and poor quality of life --
-- Initial commercial launch is planned for Germany immediately following the EC approval --
CAMBRIDGE, Mass., March 25, 2022 /PRNewswire/ -- Blueprint Medicines Corporation (NASDAQ: BPMC) today announced that the European Commission (EC) has expanded the current indication for AYVAKYT® (avapritinib) to include monotherapy for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematological neoplasm (SM-AHN) or mast cell leukemia (MCL), after at least one systemic therapy.
"Today we are incredibly proud to bring an innovative new treatment option to individuals who have been impacted by advanced systemic mastocytosis," said Georg Pirmin Meyer, M.D., Senior Vice President, International at Blueprint Medicines. "We believe that AYVAKYT has the potential to shift the treatment paradigm to a precision therapy approach in advanced forms this disease, and we look forward to working closely with national reimbursement bodies across Europe to bring AYVAKYT to patients."
In Europe, Blueprint Medicines plans to initiate its first commercial launch in Germany following the EC approval, and the timing of AYVAKYT commercial availability will vary for other countries based on local reimbursement and access pathways. AYVAKYT will be available in 25 mg, 50 mg, 100mg and 200mg dose strengths, and the recommended starting dose in advanced SM is 200 mg once daily.
The EC decision follows the positive opinion by the Committee for Medicinal Products for Human Use (CHMP) and is based on results from the Phase 1 EXPLORER trial and Phase 2 PATHFINDER trial, in which AYVAKYT showed durable clinical efficacy in advanced SM patients across all disease subtypes after at least one systemic therapy and a generally well-tolerated safety profile.
About AYVAKYT® (avapritinib)
AYVAKYT® (avapritinib) is a kinase inhibitor approved by the European Commission for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumors (GIST) harboring the PDGFRA D842V mutation and for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematological neoplasm (SM-AHN) or mast cell leukemia (MCL), after at least one systemic therapy. Under the brand name AYVAKIT, the medicine is approved in the U.S. for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations, and for the treatment of adults with Advanced SM, including aggressive SM (ASM), SM-AHN and mast cell leukemia (MCL).1
It is also approved under the brand name AYVAKIT in Mainland China for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations, and in Hong Kong and Taiwan for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA D842V mutation.2-4
AYVAKYT/AYVAKIT is not approved for the treatment of any other indication in the European Union, UK, U.S., or Greater China, or for any indication in any other jurisdiction by any other health authority.
Blueprint Medicines is developing AYVAKYT/AYVAKIT globally for the treatment of advanced and non-advanced SM. The European Commission granted orphan medicinal product designation for AYVAKYT for the treatment of GIST and mastocytosis. The U.S. Food and Drug Administration (FDA) granted breakthrough therapy designation to AYVAKIT for the treatment of moderate to severe indolent SM.
To learn about ongoing or planned clinical trials, contact Blueprint Medicines at medinfoeurope@blueprintmedicines.com and +31 85 064 4001. Additional information is available at blueprintclinicaltrials.com and clinicaltrials.gov.
Please click here to see the Summary of Product Characteristics for AYVAKYT.
For more information, visit www.BlueprintMedicines.com and follow us on Twitter (@BlueprintMeds) and LinkedIn.
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SOURCE Blueprint Medicines Corporation
Blueprint Medicines' Ayvakyt gets approval in EU to treat systemic mastocytosis
Mar. 25, 2022 10:40 AM ET
Blueprint Medicines Corporation (BPMC)
By: Ravikash, SA News Editor
- The European Commission approved the expanded use of Blueprint Medicines' (NASDAQ:BPMC) Ayvakyt (avapritinib) as a monotherapy for adult patients with aggressive systemic mastocytosis, systemic mastocytosis with an associated hematological neoplasm or mast cell leukemia, after at least one systemic therapy.
- https://seekingalpha.com/news/3817509-blueprint-medicines-ayvakyt-gets-approval-in-eu-to-treat-systemic-mastocytosis
- https://seekingalpha.com/symbol/BPMC
- https://ayvakit.com/
- https://www.blueprintmedicines.com/
CABOMETYX® (cabozantinib)
Exelixis’ Partner Ipsen Receives Positive CHMP Opinion for CABOMETYX® (cabozantinib) for Patients with Previously Treated Radioactive Iodine-Refractory Differentiated Thyroid CancerPDF Version– CHMP recommendation follows September 2021 U.S. FDA approval of CABOMETYX in this setting –ALAMEDA, Calif.--(BUSINESS WIRE)--Mar. 25, 2022-- Exelixis, Inc. (Nasdaq: EXEL) today announced that its partner Ipsen received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMA), for CABOMETYX® (cabozantinib) as a monotherapy for the treatment of adult patients with locally advanced or metastatic differentiated thyroid carcinoma (DTC), refractory or not eligible to radioactive iodine (RAI) who have progressed during or after prior systemic therapy. The European Commission, which has the authority to approve medicines for the European Union, will now review the CHMP recommendation, and a final decision on the application is expected in the coming months.“The CHMP’s recommendation is an important milestone for our partner Ipsen in their efforts to bring CABOMETYX to patients in Europe with radioactive iodine-refractory differentiated thyroid cancer that has progressed following prior systemic therapy,” said Vicki L. Goodman, M.D., Executive Vice President, Product Development & Medical Affairs, and Chief Medical Officer at Exelixis. “These patients have a poor prognosis, and there are currently no standard treatment options approved in Europe following progression on anti-VEGFR therapy. We look forward to hearing the European Commission’s decision in the coming months, with the hope that a new therapy will soon be available to these patients and their caregivers.”The CHMP recommendation is based on results from COSMIC-311, the phase 3 pivotal trial that demonstrated significant improvement in progression-free survival (PFS) with CABOMETYX versus placebo in patients with RAI-refractory DTC who progressed after up to two prior VEGFR-targeted therapies. COSMIC-311 was the basis for the U.S. Food and Drug Administration (FDA) approval of CABOMETYX in September 2021 for the treatment of adult and pediatric patients 12 years of age and older with locally advanced or metastatic DTC that has progressed following VEGFR-targeted therapy and who are RAI-refractory or ineligible. CABOMETYX is currently approved in the European Union as a monotherapy for the treatment of advanced renal cell carcinoma (RCC) in adults who have received prior VEGF-targeted therapy, for previously untreated intermediate- or poor-risk advanced RCC and for hepatocellular carcinoma (HCC) in adults who have been previously treated with sorafenib. CABOMETYX in combination with OPDIVO® (nivolumab) is approved as a first-line treatment for advanced RCC.
About COSMIC-311COSMIC-311 was a multicenter, randomized, double-blind, placebo-controlled phase 3 pivotal trial that enrolled 258 patients at 164 sites globally. Patients were randomized in a 2:1 ratio to receive either CABOMETYX 60 mg or placebo once daily. The primary endpoints were PFS and objective response rate. Exelixis is sponsoring COSMIC-311, and Ipsen is co-funding the trial. More information about this trial is available at ClinicalTrials.gov.
About CABOMETYX® (cabozantinib)In the U.S., CABOMETYX tablets are approved for the treatment of patients with advanced RCC; for the treatment of patients with HCC who have been previously treated with sorafenib; for patients with advanced RCC as a first-line treatment in combination with nivolumab; and for adult and pediatric patients 12 years of age and older with locally advanced or metastatic DTC that has progressed following prior VEGFR-targeted therapy and who are RAI-refractory or ineligible. CABOMETYX tablets have also received regulatory approvals in the European Union and additional countries and regions worldwide. In 2016, Exelixis granted Ipsen exclusive rights for the commercialization and further clinical development of cabozantinib outside of the U.S. and Japan. Under the terms of the license agreement with Ipsen, Exelixis is eligible to receive development, regulatory and sales milestone payments from Ipsen and further receive royalties on net sales of cabozantinib by Ipsen outside of the U.S. and Japan. In 2017, Exelixis granted exclusive rights to Takeda Pharmaceutical Company Limited for the commercialization and further clinical development of cabozantinib for all future indications in Japan. Exelixis holds the exclusive rights to develop and commercialize cabozantinib in the U.S. https://www.cabometyx.com/
Please see accompanying full Prescribing Information https://www.cabometyx.com/downloads/CABOMETYXUSPI.pdf.
For more information about Exelixis, please visit www.exelixis.com, follow @ExelixisInc on Twitter or like Exelixis, Inc. on Facebook.
Exelixis, the Exelixis logo, CABOMETYX and COMETRIQ are registered U.S. trademarks of Exelixis. COTELLIC is a registered trademark of Genentech, Inc. MINNEBRO is a registered trademark of Daiichi Sankyo Company, Limited. OPDIVO® is a registered trademark of Bristol-Myers Squibb Company.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220323005988/en/ Source: Exelixis, Inc.
Ipsen, Exelixis' Cabometyx gets EMA panel nod for expanded use in thyroid cancer
Mar. 25, 2022 9:30 AM ET Ipsen S.A. (IPSEY), IPSEF, EXEL By: Ravikash, SA News Editor2 Comments
- A committee of the European Medicines Agency (EMA) recommended the approval of expanded use of Ipsen (OTCPK:IPSEF) (OTCPK:IPSEY) and Exelixis' (NASDAQ:EXEL) Cabometyx as a standalone therapy for certain patients with differentiated thyroid cancer (DTC).
- https://seekingalpha.com/news/3817472-ipsen-exelixis-cabometyx-gets-ema-panel-nod-for-expanded-use-thyroid-cancer
- https://seekingalpha.com/symbol/IPSEY
- https://seekingalpha.com/symbol/IPSEF
- https://seekingalpha.com/symbol/EXEL
- https://www.cabometyx.com/
MARCH 25, 2022
Ipsen receives positive CHMP opinion for Cabometyx® in radioactive iodine-refractory differentiated thyroid cancer
- Recommendation based on data from the COSMIC-311 Phase III trial, in which Cabometyx® (cabozantinib) demonstrated a 78% reduction in risk of disease progression or death versus placebo1
PARIS, FRANCE, 25 March 2022 – Ipsen (Euronext: IPN; ADR: IPSEY)https://www.ipsen.com/press-releases/ipsen-receives-positive-chmp-opinion-for-cabometyx-in-radioactive-iodine-refractory-differentiated-thyroid-cancer/
CARVYKTI™ (ciltacabtagene autoleucel)
CARVYKTI® (ciltacabtagene autoleucel) Receives Positive CHMP Opinion for the Treatment of Patients with Relapsed and Refractory Multiple Myeloma Ciltacabtagene autoleucel, if approved by the European Commission (EC), will be Legend Biotech’s first EC-approved product SOMERSET, N.J.— (BUSINESS WIRE)—March 25, 2022—Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global biotechnology company developing, manufacturing and commercializing novel therapies to treat life-threatening diseases, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommended Janssen Pharmaceutica NV’s marketing authorization of CARVYKTI® (ciltacabtagene autoleucel; cilta-cel) for the treatment of adults with relapsed and refractory multiple myeloma who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody and have demonstrated disease progression on the last therapy. Cilta-cel is a chimeric antigen receptor T-cell (CAR-T) therapy featuring two B-cell maturation antigen (BCMA)-targeting single domain antibodies.1 CAR-T therapy is a highly personalized technology where a person’s own T-cells are engineered to target and kill cancer cells in a single infusion.2 Data from the ongoing pivotal CARTITUDE-1 study supported the positive CHMP opinion. Two-year follow-up results were presented at the American Society of Hematology (ASH) 2021 Annual Meeting (Abstract #549). 1 “The positive CHMP opinion reinforces the potential of cilta-cel for patients with multiple myeloma around the world,” said Ying Huang, Ph.D., Chief Executive Officer and Chief Financial Officer of Legend Biotech. “We look forward to the EMA to the potential of European Commission approval in the future and continued progress in the development of cilta-cel.” Multiple myeloma is an incurable blood cancer affecting a type of white blood cell called plasma cells, which are found in the bone marrow. 3 The majority of patients relapse after undergoing initial treatment and face poor prognoses after treatment with three major drug classes, including an immunomodulatory agent, a proteasome inhibitor and anti-CD38 monoclonal antibody. 4,5,6 This CHMP Opinion follows the approval of cilta-cel by the U.S. Food and Drug Administration (FDA) on February 28, 2022. About Ciltacabtagene autoleucel (cilta-cel) Cilta-cel is a BCMA-directed, genetically modified autologous T-cell immunotherapy, which involves reprogramming a patient’s own T-cells with a transgene encoding a chimeric antigen receptor (CAR) that identifies and eliminates cells that express BCMA. BCMA is primarily expressed on the surface of malignant multiple myeloma B-lineage cells, as well as late-stage B-cells and plasma cells. The cilta-cel CAR protein features two BCMA-targeting single domain antibodies designed to confer high avidity against human BCMA. Upon binding to BCMA-expressing cells, the CAR promotes T-cell activation, expansion, and elimination of target cells. In December 2017, Legend Biotech Corporation entered into an exclusive worldwide license and collaboration agreement with Janssen Biotech, Inc. to develop and commercialize ciltacel. In April 2021, Legend announced the submission of a Marketing Authorisation Application to the European Medicines Agency seeking approval of cilta-cel for the treatment of patients with relapsed and/or refractory multiple myeloma. In addition to U.S. Breakthrough Therapy Designation granted in December 2019, cilta-cel received a Breakthrough Therapy Designation in China in August 2020. Cilta-cel also received Orphan Drug Designation from the U.S. FDA in February 2019, and from the European Commission in February 2020.
Learn more at www.legendbiotech.com
Source: Legend Biotech
J&J, Legend Biotech's Carvykti for multiple myeloma gets EMA panel nod for EU approval
Mar. 25, 2022 8:12 AM ET
Legend Biotech Corporation (LEGN), JNJ
By: Ravikash, SA News Editor
- A committee of the European Medicines Agency (EMA) recommended the approval of Johnson & Johnson (NYSE:JNJ) and Legend Biotech's (NASDAQ:LEGN) CAR-T therapy Carvykti to treat adults with relapsed and refractory multiple myeloma, who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody and have shown disease progression on the last therapy.
- https://seekingalpha.com/news/3817421-jj-legend-biotechs-carvykti-for-multiple-myeloma-gets-ema-panel-nod-for-eu-approval
- https://seekingalpha.com/symbol/LEGN
- https://seekingalpha.com/symbol/JNJ
- https://www.carvykti.com/
- https://legendbiotech.com/
Ruxolitinib (Jakavi®)
Incyte Announces Positive CHMP Opinion for Ruxolitinib (Jakavi®) for the Treatment of Acute and Chronic Graft-Versus-Host Disease
March 25, 2022 DownloadPDF Format (opens in new window)
- The positive opinion from the CHMP is based on data from the Phase 3 REACH2 and REACH3 trials, which demonstrated that ruxolitinib (Jakavi®) improved response rates and failure-free survival compared to best available therapy, which provides a promising opportunity for patients living with graft-versus-host disease (GvHD)1,2
- Graft-versus-host disease (GVHD) is a life-threatening complication of stem cell transplants, with no established standard of care in Europe for patients who do not adequately respond to first-line steroid treatment3,4
- Nearly half of patients experience acute or chronic GVHD following allogeneic transplants 3,4
WILMINGTON, Del.--(BUSINESS WIRE)-- Incyte (Nasdaq:INCY) today announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending approval of ruxolitinib (Jakavi®) for the treatment of patients aged 12 years and older with acute graft-versus-host disease or chronic graft-versus-host disease (GVHD) and who have inadequate response to corticosteroids or other systemic therapies. If approved, ruxolitinib will be the first JAK1/2 inhibitor available for patients with GVHD in Europe.4 Ruxolitinib is marketed as Jakavi by Novartis in Europe and as Jakafi® by Incyte in the U.S.
“We are pleased that the CHMP has recommended approval of ruxolitinib for the treatment of acute or chronic GVHD in Europe,” said Peter Langmuir, M.D., Group Vice President, Oncology Targeted Therapies, Incyte. “With this positive opinion, patients living with these life-threatening complications who do not respond to first-line steroids therapies are one step closer to having a new potential standard of care.”
The CHMP positive opinion was based on data from the Phase 3 REACH2 and REACH3 clinical studies, in which ruxolitinib demonstrated superiority versus best available therapy (BAT) in patients with steroid-refractory and steroid-dependent acute and chronic GVHD, respectively.
Results from the REACH2 trial showed an overall response rate (ORR) at Day 28 was superior in the ruxolitinib arm at 62.3% vs. 39.4% in the BAT arm (odds ratio [OR], 2.64; p<0.001) in patients with steroid refractory/dependent acute GVHD; and in those patients who maintained response at Day 56, the ORR in the Jakavi arm was 40% vs. 22% in the BAT arm (p<0.001). In REACH3, treatment with ruxolitinib led to significant improvements in ORR compared to BAT (49.7% vs. 25.6%; OR, 2.99; P<0.0001) in patients with steroid refractory/dependent chronic GVHD at week 24, the primary endpoint of the study, regardless of the individual organs involved at baseline. Also, best overall response (BOR) rate at any time up to week 24 was achieved in 76.4% of patients in the ruxolitinib arm compared to 60.4% in the BAT arm (OR, 2.17; 95% CI, 1.34-3.52). Results from the two studies were published in the April 22, 2020 (REACH2), and July 15, 2021 (REACH3) issues of The New England Journal of Medicine.1,2
GVHD is a condition that can occur after an allogeneic stem cell transplant (the transfer of stem cells from a donor) where the donated cells initiate an immune response and attack the transplant recipient’s organs, leading to significant morbidity and mortality. There are two major forms of GVHD: acute, which generally occurs within 100 days of transplant, and chronic, which generally occurs more than 100 days after transplant.2 GVHD can affect multiple organ systems including the skin, gastrointestinal (digestive) tract and liver.
“For many hematologic diseases allogeneic transplant is the only treatment with the potential to be curative; however, half will go on to develop acute or chronic GVHD,” said Dr. Robert Zeiser, University Hospital Freiburg, Department of Haematology, Oncology and Stem Cell Transplantation, Freiburg, Germany. “It is encouraging that we may soon have a new standard of care for patients with this often debilitating condition who do not adequately respond to first-line corticosteroids.”
The CHMP opinion to recommend the use of ruxolitinib in acute and chronic GVHD is now being reviewed by the European Commission, which has the authority to grant marketing authorization for medicinal products in the European Union. The EC will review the CHMP recommendations and is expected to make a final decision within approximately 2 months.
In 2019, Jakafi® (ruxolitinib) was approved by the U.S. Food and Drug Administration (FDA) for the treatment of steroid-refractory acute GVHD in adult and pediatric patients 12 years and older, based on the positive results of the Phase 2 REACH1 trial.5 Additionally, in 2021, Jakafi was approved by the FDA for treatment of chronic GVHD after failure of one or two lines of systemic therapy in adult and pediatric patients 12 years and older, based on the positive results of the Phase 3 REACH3 trial.5
About Jakafi® (ruxolitinib)
Jakafi® (ruxolitinib) is a JAK1/JAK2 inhibitor approved by the U.S. FDA for treatment of polycythemia vera (PV) in adults who have had an inadequate response to or are intolerant of hydroxyurea; intermediate or high-risk myelofibrosis (MF), including primary MF, post-polycythemia vera MF and post-essential thrombocythemia MF in adults; steroid-refractory acute GVHD in adult and pediatric patients 12 years and older; and chronic GVHD after failure of one or two lines of systemic therapy in adult and pediatric patients 12 years and older.5
Jakafi is marketed by Incyte in the United States and by Novartis as Jakavi® (ruxolitinib) outside the United States. Jakafi is a registered trademark of Incyte Corporation. Jakavi is a registered trademark of Novartis AG in countries outside the United States.
Please see the Full Prescribing Information, which includes a more complete discussion of the risks associated with Jakafi.
For additional information on Incyte, please visit Incyte.com and follow @Incyte.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220325005243/en/
Source: Incyte
Novartis, Incyte Jakavi gets EMA panel nod for expanded use in graft-versus-host disease
Mar. 25, 2022 9:00 AM ET
Incyte Corporation (INCY), NVS
By: Ravikash, SA News Editor
- A committee of the European Medicines Agency (EMA) recommended the approval for expanded use of Incyte (NASDAQ:INCY) and Novartis' Jakavi (ruxolitinib) to treat patients aged 12 years and older with acute graft-versus-host disease or chronic graft-versus-host disease (GVHD) who have inadequate response to corticosteroids or other systemic therapies.
- https://seekingalpha.com/news/3817449-novartis-incyte-jakavi-gets-ema-panel-nod-for-expanded-use-in-graft-versus-host-disease
- https://seekingalpha.com/symbol/INCY
- https://seekingalpha.com/symbol/NVS
- https://www.jakavi.com/
- https://www.incyte.com/
KEYTRUDA® (pembrolizumab)
Merck Receives Positive EU CHMP Opinion for KEYTRUDA® (pembrolizumab) for Patients With Microsatellite Instability-High (MSI-H) or Mismatch Repair Deficient (dMMR) Tumors in Five Different Types of Cancer
March 25, 2022 9:00 am ET
Recommendation Supports Use of KEYTRUDA for Certain Patients With Unresectable or Metastatic MSI-H/dMMR Colorectal, Gastric, Small Intestine or Biliary Cancer, as Well as Advanced or Recurrent MSI-H/dMMR Endometrial Carcinoma
KENILWORTH, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending approval of KEYTRUDA, Merck’s anti-PD-1 therapy, as monotherapy for the treatment of the following microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) tumors in adults with: unresectable or metastatic colorectal cancer after previous fluoropyrimidine-based combination therapy; advanced or recurrent endometrial carcinoma who have disease progression on or following prior treatment with a platinum-containing therapy in any setting and who are not candidates for curative surgery or radiation; unresectable or metastatic gastric, small intestine or biliary cancer who have disease progression on or following at least one prior therapy.
“We are committed to advancing the use of biomarkers to identify patients most likely to respond to KEYTRUDA,” said Dr. Scot Ebbinghaus, vice president, global clinical development, Merck Research Laboratories. “This positive CHMP opinion reinforces the predictive value of MSI-H/dMMR across many different cancer types and the importance of biomarker testing. KEYTRUDA has already become an important first-line treatment option for certain patients in Europe with MSI-H or dMMR colorectal cancer, and we are pleased the CHMP has recommended KEYTRUDA as a monotherapy for additional patients with MSI-H or dMMR tumors.”
The CHMP’s recommendation was based on results from the Phase 2 KEYNOTE-158 trial as well as results from the Phase 2 KEYNOTE-164 trial, both of which also supported the U.S. Food and Drug Administration’s accelerated approval of KEYTRUDA as the first cancer treatment approved based on a biomarker in MSI-H or dMMR solid tumors, regardless of tumor type.
The CHMP’s recommendation will now be reviewed by the European Commission for marketing authorization in the European Union, and a final decision is expected in the second quarter of 2022.
About Microsatellite Instability-High (MSI-H) and Deficient Mismatch Repair (dMMR)
Microsatellite instability (MSI) and deficient mismatch repair (dMMR) are biomarkers that have been identified in many different types of cancer and that can be hereditary or random. MSI is a change that occurs in the DNA of certain cells, such as cancer cells, in which the number of repeated DNA bases in a microsatellite (which is a short, repeated sequence of DNA) is different from what it was when the microsatellite was inherited. dMMR describes cells that have mutations in certain genes involved in correcting mistakes made when DNA is copied into a cell when dividing. High levels of MSI (MSI-H) and dMMR can occur when a cell is unable to repair mistakes during that division process.
About KEYTRUDA® (pembrolizumab) Injection, 100 mg
KEYTRUDA is an anti-programmed death receptor-1 (PD-1) therapy that works by increasing the ability of the body’s immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.
Merck has the industry’s largest immuno-oncology clinical research program. There are currently more than 1,700 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.
Selected KEYTRUDA® (pembrolizumab) Indications in the U.S.
Melanoma
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.
KEYTRUDA is indicated for the adjuvant treatment of adult and pediatric (12 years and older) patients with stage IIB, IIC, or III melanoma following complete resection.
Non-Small Cell Lung Cancer
KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) ≥1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is:
- stage III where patients are not candidates for surgical resection or definitive chemoradiation, or
- metastatic.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS ≥1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.
Head and Neck Squamous Cell Cancer
KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [Combined Positive Score (CPS) ≥1] as determined by an FDA-approved test.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.
Classical Hodgkin Lymphoma
KEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).
KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.
Primary Mediastinal Large B-Cell Lymphoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy.
KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.
Urothelial Carcinoma
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC):
- who are not eligible for any platinum-containing chemotherapy, or
- who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
Non-muscle Invasive Bladder Cancer
KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.
Microsatellite Instability-High or Mismatch Repair Deficient Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options.
This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.
Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).
Gastric Cancer
KEYTRUDA, in combination with trastuzumab, fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of patients with locally advanced unresectable or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma.
This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Esophageal Cancer
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic esophageal or gastroesophageal junction (GEJ) (tumors with epicenter 1 to 5 centimeters above the GEJ) carcinoma that is not amenable to surgical resection or definitive chemoradiation either:
- in combination with platinum- and fluoropyrimidine-based chemotherapy, or
- as a single agent after one or more prior lines of systemic therapy for patients with tumors of squamous cell histology that express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
Cervical Cancer
KEYTRUDA, in combination with chemotherapy, with or without bevacizumab, is indicated for the treatment of patients with persistent, recurrent, or metastatic cervical cancer whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test.
Hepatocellular Carcinoma
KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Merkel Cell Carcinoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Renal Cell Carcinoma
KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of adult patients with advanced renal cell carcinoma (RCC).
KEYTRUDA is indicated for the adjuvant treatment of patients with RCC at intermediate-high or high risk of recurrence following nephrectomy, or following nephrectomy and resection of metastatic lesions.
Endometrial Carcinoma
KEYTRUDA, as a single agent, is indicated for the treatment of patients with advanced endometrial carcinoma that is MSI-H or dMMR, as determined by an FDA-approved test, who have disease progression following prior systemic therapy in any setting and are not candidates for curative surgery or radiation.
Tumor Mutational Burden-High Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [≥10 mutations/megabase] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.
Cutaneous Squamous Cell Carcinoma
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) or locally advanced cSCC that is not curable by surgery or radiation.
Triple-Negative Breast Cancer
KEYTRUDA is indicated for the treatment of patients with high-risk early-stage triple-negative breast cancer (TNBC) in combination with chemotherapy as neoadjuvant treatment, and then continued as a single agent as adjuvant treatment after surgery.
KEYTRUDA, in combination with chemotherapy, is indicated for the treatment of patients with locally recurrent unresectable or metastatic TNBC whose tumors express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
For more information about our oncology clinical trials, visit www.merck.com/clinicaltrials.
For more information, visit www.merck.com and connect with us on Twitter, Facebook, Instagram, YouTube and LinkedIn.
Please see Prescribing Information for KEYTRUDA (pembrolizumab) at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_pi.pdf and Medication Guide for KEYTRUDA at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_mg.pdf .
Source: Merck & Co., Inc.
Merck's Keytruda gets EMA panel nod for expanded use in certain tumors in 5 cancers
Mar. 25, 2022 9:49 AM ET Merck & Co., Inc. (MRK) By: Ravikash, SA News Editor
- A committee of the European Medicines Agency (EMA) recommended the approval of expanded use of Merck's (NYSE:MRK) blockbuster drug Keytruda as a monotherapy for microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) tumors in five different types of cancers.
- https://seekingalpha.com/news/3817481-mercks-keytruda-gets-ema-panel-nod-for-expanded-use-in-certain-tumors-in-5-cancers
- https://seekingalpha.com/symbol/MRK
- https://www.keytruda.com/
- https://www.merck.com/
Respiratory Syncytial Virus Vaccine
Pfizer Granted FDA Breakthrough Therapy Designation for Respiratory Syncytial Virus Vaccine Candidate for the Prevention of RSV in Older Adults
Thursday, March 24, 2022 - 06:45am
NEW YORK--(BUSINESS WIRE)-- Pfizer Inc. (NYSE:PFE) today announced that its respiratory syncytial virus (RSV) vaccine candidate, PF-06928316 or RSVpreF, received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for the prevention of lower respiratory tract disease caused by RSV in individuals 60 years of age or older.
The FDA decision is primarily informed by the positive results of a proof-of-concept, Phase 2a study evaluating the safety, immunogenicity, and efficacy of a single dose of 120µg RSVpreF in a human viral challenge model in healthy adults 18 to 50 years of age.
“Today’s decision is a significant step forward in our efforts to help protect vulnerable populations, particularly older adults, against certain potentially serious respiratory illnesses, including RSV,” said Kathrin U. Jansen, Ph.D., Senior Vice President and Head of Vaccine Research & Development at Pfizer Inc. “The clinical and economic burden of RSV represents a critical need, and we look forward to our ongoing dialogue with the FDA to accelerate the development of our RSV vaccine candidate.”
In September 2021, Pfizer announced the initiation of RENOIR (RSV vaccine Efficacy study iNOlder adults Immunized against RSV disease), a Phase 3 clinical trial (NCT05035212) evaluating the efficacy, immunogenicity, and safety of a single dose of RSVpreF, in adults ages 60 years or older. This study remains ongoing.
The FDA’s Breakthrough Therapy Designation is designed to expedite the development and review of drugs and vaccines that are intended to treat or prevent serious conditions and preliminary clinical evidence indicates that the drug or vaccine may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).1
About RSVpreF
Pfizer’s investigational RSV vaccine candidate builds on foundational basic science discoveries including those made at the National Institutes of Health (NIH), which detailed the crystal structure of prefusion F, a key form of the viral fusion protein (F) that RSV uses to attack human cells. The NIH research showed that antibodies specific to the prefusion form were highly effective at blocking virus infection, suggesting a prefusion F-based vaccine may confer optimal protection against RSV. After this important discovery, Pfizer tested numerous versions of the viral protein, and identified those that elicited a strong anti-viral immune response in pre-clinical evaluation. The vaccine candidate is composed of two preF proteins selected to optimize protection against RSV A and B.
Earlier this month, Pfizer announced RSVpreF received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for the prevention of RSV-associated lower respiratory tract disease in infants from birth up to six months of age by active immunization of pregnant women. The FDA designation was informed by the results of the Phase 2b proof-of-concept study of RSVpreF (NCT04032093), which evaluated the safety, tolerability and immunogenicity of RSVpreF in vaccinated pregnant women ages 18 through 49 and their infants. This followed the FDA’s November 2018 decision to grant Fast Track status to RSVpreF.
In June 2020, Pfizer announced the initiation of a multicenter, international Phase 3 clinical trial (NCT04424316) evaluating the efficacy and safety of RSVpreF when administered to pregnant women to help protect their babies from RSV after birth. This study remains ongoing.
In addition, to learn more, please visit us on www.Pfizer.com and follow us on Twitter at @Pfizer and @Pfizer News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.
Pfizer granted FDA’s Breakthrough Therapy Designation for RSV vaccine candidate
Mar. 24, 2022 7:16 AM ETPfizer Inc. (PFE)By: Dulan Lokuwithana, SA News Editor
- Pfizer (NYSE:PFE) announced on Thursday that the U.S. Food and Drug Administration (FDA) awarded the agency’s Breakthrough Therapy Designation for PF-06928316 (RSVpreF), a vaccine candidate targeted at respiratory syncytial virus (RSV).
- https://seekingalpha.com/news/3816833-pfizer-granted-fdas-breakthrough-therapy-designation-for-rsv-vaccine-candidate
- https://seekingalpha.com/symbol/PFE
- https://www.pfizer.com/health-wellness/disease-conditions/respiratory-syncytial-virus-rsv
Etrasimod
Pfizer Announces Positive Top-Line Results for Phase 3 Trial of Etrasimod in Ulcerative Colitis Patients
Wednesday, March 23, 2022 - 06:45am
- ELEVATE 12 study met primary and key secondary endpoints of improving clinical remission at week 12 -
- Safety profile consistent with previous Phase 2 studies -
NEW YORK--(BUSINESS WIRE)-- Pfizer Inc. (NYSE: PFE) today announced positive topline results from a Phase 3 study of etrasimod, an investigational, oral, once-a-day, selective sphingosine 1-phosphate (S1P) receptor modulator in development for the treatment of moderately to severely active ulcerative colitis (UC). In the study, etrasimod patients achieved statistically significant improvements in the primary endpoint of clinical remission at week 12 as compared with placebo. Statistically significant improvements were achieved in all key secondary endpoints in the trial as well. The safety profile was consistent with previous Phase 2 studies.
“These positive results demonstrate that etrasimod, if approved, could be a potential breakthrough option for patients with ulcerative colitis who aren’t able to experience improvement on current therapies,” said Michael Corbo, Chief Development Officer, Inflammation & Immunology, Pfizer Global Product Development. “This outcome represents a continuation of our effort to develop new therapeutic approaches to treat immuno-inflammatory diseases and address the need for oral therapies for UC patients with inadequate response, loss of response, or intolerance to conventional or advanced therapies.”
The global phase 3 multi-center, randomized, double-blind, placebo-controlled study, also known as ELEVATE 12, enrolled 354 UC patients who had previously failed or were intolerant to at least one conventional, biologic, or JAK therapy. Participants received etrasimod 2mg once daily.
Full results from the study will be submitted for future scientific publication and presentation. These data along with results from ELEVATE 52 are expected to form the basis for planned future regulatory filings. Results from the ELEVATE 52 study will be available by the end of Q1. Additional information about the studies can be found at www.clinicaltrials.gov under the identifiers NCT03996369 and NCT03945188.
Etrasimod was developed by Arena Pharmaceuticals, which was recently acquired by Pfizer.
About Etrasimod
Etrasimod is an oral, once-a-day, selective sphingosine 1-phosphate (S1P) receptor modulator being investigated for a range of immuno-inflammatory diseases including ulcerative colitis, Crohn’s Disease, atopic dermatitis, eosinophilic esophagitis, and alopecia areata.
In a Phase 2, randomized, placebo-controlled, dose-ranging study (OASIS) in moderate to severe UC patients, most patients who achieved clinical response, clinical remission, or endoscopic improvement at week 12 experienced sustained or improved effects up to week 46, with etrasimod 2 mg in the open-label extension. Etrasimod also demonstrated a favorable benefit/risk profile, consistent with safety findings reported in the double-blind portion of OASIS.
In addition, to learn more, please visit us on www.pfizer.com and follow us on Twitter at @Pfizer and @Pfizer_News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220323005370/en/
Source: Pfizer Inc.
Pfizer's etrasimod helps improve remission in ulcerative colitis patients in phase 3 trial
Mar. 23, 2022 7:08 AM ET Pfizer Inc. (PFE) By: Ravikash, SA News Editor1 Comment
- Pfizer (NYSE:PFE) said a late stage study of its medicine etrasimod met the main goal of improving clinical remission at week 12 in patients with ulcerative colitis (UC).
- https://seekingalpha.com/news/3816341-pfizers-etrasimod-improving-remission-in-ulcerative-colitis-patients-in-phase-3-trial
- https://seekingalpha.com/symbol/PFE
- https://www.pfizer.com/
biotecMAX™ Feb/Mar 2022 Insight
KEYTRUDA® (pembrolizumab)
Merck’s KEYTRUDA® (pembrolizumab) Significantly Improved Disease-Free Survival (DFS) Versus Placebo as Adjuvant Therapy in Patients With Stage IB-IIIA Non-Small Cell Lung Cancer (NSCLC) Regardless of PD-L1 Expression
March 17, 2022 2:15 pm ET
First Phase 3 Study To Demonstrate Statistically Significant Improvement in DFS in the Adjuvant Setting for Patients With Stage IB-IIIA NSCLC Regardless of PD-L1 Expression
KENILWORTH, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, the European Organisation for Research and Treatment of Cancer (EORTC) and the European Thoracic Oncology Platform (ETOP) today announced results from the pivotal Phase 3 KEYNOTE-091 trial, also known as EORTC-1416-LCG/ETOP-8-15 – PEARLS. The study found that adjuvant treatment with KEYTRUDA significantly improved disease-free survival (DFS), one of the dual primary endpoints, reducing the risk of disease recurrence or death by 24% compared to placebo (hazard ratio [HR]=0.76 [95% CI, 0.63-0.91]; p=0.0014) in patients with stage IB (≥4 centimeters) to IIIA non-small cell lung cancer (NSCLC) following surgical resection regardless of PD-L1 expression. Median DFS was 53.6 months for KEYTRUDA versus 42.0 months for placebo, an improvement of nearly one year. These data are being presented today during a European Society for Medical Oncology (ESMO) Virtual Plenary and will be shared with regulatory authorities worldwide.
“These are the first positive results for KEYTRUDA in the adjuvant setting for non-small cell lung cancer, and represent the sixth positive pivotal study evaluating a KEYTRUDA-based regimen in earlier stages of cancer,” said Dr. Roy Baynes, senior vice president and head of global clinical development, chief medical officer, Merck Research Laboratories. “KEYTRUDA has become foundational in the treatment of metastatic non-small cell lung cancer, and we are pleased to present these data showing the potential of KEYTRUDA to help more patients with lung cancer in earlier stages of disease. We thank the patients, their caregivers and investigators for participating in this study.”
As previously announced, there was also an improvement in DFS for patients whose tumors express PD-L1 (tumor proportion score [TPS] ≥50%) treated with KEYTRUDA compared to placebo, the other dual primary endpoint; these results did not reach statistical significance per the pre-specified statistical plan (HR=0.82 [95% CI, 0.57-1.18]; p=0.14). Among these patients, median DFS was not reached in either arm. Additionally, a favorable trend in overall survival (OS), a key secondary endpoint, was observed for KEYTRUDA versus placebo regardless of PD-L1 expression (HR=0.87 [95% CI, 0.67-1.15]; p=0.17); these OS data are not mature and did not reach statistical significance at the time of this interim analysis. The trial will continue to evaluate DFS in patients whose tumors express high levels of PD-L1 (TPS ≥50%) and OS. The safety profile of KEYTRUDA in this study was consistent with that observed in previously reported studies.
“Lung cancer is most treatable at earlier stages, and adding treatment after surgery may help reduce the risk of recurrence,” said Professor Mary O'Brien, consultant medical oncologist and head of the Lung Unit at The Royal Marsden NHS Foundation Trust and professor of practice (medical oncology) at Imperial College London, as well as co-principal investigator. “We are encouraged by these new Phase 3 data, as they represent the first time adjuvant immunotherapy has demonstrated a statistically significant and clinically meaningful improvement in disease-free survival for patients with stage IB-IIIA non-small cell lung cancer.”
“While significant advancements have been made in the treatment of metastatic non-small cell lung cancer, there remains an unmet need for patients with earlier stages of this disease, as up to 43% of them will experience disease recurrence following surgery,” said Dr. Luis Paz-Ares, chair of the medical oncology department, Hospital Universitario Doce de Octubre, Madrid, Spain and co-principal investigator. “The positive disease-free survival data observed in this study with the use of KEYTRUDA in the adjuvant setting has the potential to have important implications for how we treat patients with stage IB-IIIA non-small cell lung cancer.”
In addition to KEYNOTE-091, five other pivotal trials evaluating a KEYTRUDA-based regimen in patients with earlier stages of cancer met their primary endpoint(s). These trials included: KEYNOTE-716 in stage IIB and IIC melanoma; KEYNOTE-054 in stage III melanoma; KEYNOTE-564 in renal cell carcinoma; KEYNOTE-522 in triple-negative breast cancer; and KEYNOTE-057 in Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, non-muscle invasive bladder cancer.
Merck has an extensive clinical development program in lung cancer and is advancing multiple registration-enabling studies, with research directed at earlier stages of disease and novel combinations. Key studies in earlier stages of NSCLC include KEYNOTE-091, KEYNOTE-671, KEYNOTE-867 and KEYLYNK-012.
Study Design and Additional Data From KEYNOTE-091
KEYNOTE-091, also known as EORTC-1416-LCG/ETOP-8-15 – PEARLS, is a randomized, Phase 3 trial (ClinicalTrials.gov, NCT02504372) sponsored by Merck and conducted in collaboration with EORTC and ETOP evaluating KEYTRUDA compared to placebo for the adjuvant treatment of patients with stage IB (≥4 centimeters) to IIIA NSCLC following surgical resection (lobectomy or pneumonectomy) and with adjuvant chemotherapy when indicated. The dual primary endpoints are DFS in the overall population and in patients whose tumors express PD-L1 (TPS ≥50%). Disease-free survival is calculated as the time from randomization to the date of disease recurrence, occurrence of second primary lung cancer, occurrence of second malignancy, or death from any cause, whichever occurs first. The secondary endpoints include OS and lung cancer-specific survival (the time from randomization to date of death due to lung cancer specifically). The study randomized 1,177 patients (1:1) to receive either KEYTRUDA (200 mg intravenously [IV] every three weeks [Q3W] for one year or maximum 18 doses; n=590); or placebo (IV Q3W for one year or maximum 18 doses; n=587). The median number of doses was 17 for KEYTRUDA and 18 for placebo. As of data cut-off for this interim analysis (September 20, 2021), median time from randomization to data cut-off was 35.6 months (range, 16.5-68.0 months).
Grade ≥3 adverse events occurred in 34.1% of patients receiving KEYTRUDA and 25.8% of patients receiving placebo. Adverse events resulting in discontinuation of any treatment occurred in 19.8% of patients receiving KEYTRUDA and 5.9% of patients receiving placebo; there were four treatment-related deaths in the KEYTRUDA arm and no treatment-related deaths in the placebo arm.
About KEYTRUDA® (pembrolizumab) Injection, 100 mg
KEYTRUDA is an anti-programmed death receptor-1 (PD-1) therapy that works by increasing the ability of the body’s immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.
Merck has the industry’s largest immuno-oncology clinical research program. There are currently more than 1,700 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.
Selected Indications for KEYTRUDA® (pembrolizumab) in the U.S.
Non-Small Cell Lung Cancer
KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) ≥1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is:
- stage III where patients are not candidates for surgical resection or definitive chemoradiation, or
- metastatic.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS ≥1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.
See additional selected indications for KEYTRUDA in the U.S. after the Selected Important Safety Information
Additional Indications for KEYTRUDA in the U.S.
Melanoma
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.
KEYTRUDA is indicated for the adjuvant treatment of adult and pediatric (12 years and older) patients with stage IIB, IIC, or III melanoma following complete resection.
Head and Neck Squamous Cell Cancer
KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS) ≥1] as determined by an FDA-approved test.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.
Classical Hodgkin Lymphoma
KEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).
KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.
Primary Mediastinal Large B-Cell Lymphoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy.
KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.
Urothelial Carcinoma
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC):
- who are not eligible for any platinum-containing chemotherapy, or
- who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
Non-muscle Invasive Bladder Cancer
KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.
Microsatellite Instability-High or Mismatch Repair Deficient Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options.
This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.
Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).
Gastric Cancer
KEYTRUDA, in combination with trastuzumab, fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of patients with locally advanced unresectable or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma.
This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Esophageal Cancer
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic esophageal or gastroesophageal (GEJ) (tumors with epicenter 1 to 5 centimeters above the GEJ) carcinoma that is not amenable to surgical resection or definitive chemoradiation either:
- in combination with platinum- and fluoropyrimidine-based chemotherapy, or
- as a single agent after one or more prior lines of systemic therapy for patients with tumors of squamous cell histology that express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
Cervical Cancer
KEYTRUDA, in combination with chemotherapy, with or without bevacizumab, is indicated for the treatment of patients with persistent, recurrent, or metastatic cervical cancer whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test.
Hepatocellular Carcinoma
KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Merkel Cell Carcinoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Renal Cell Carcinoma
KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of adult patients with advanced renal cell carcinoma (RCC).
KEYTRUDA is indicated for the adjuvant treatment of patients with RCC at intermediate-high or high risk of recurrence following nephrectomy, or following nephrectomy and resection of metastatic lesions.
Tumor Mutational Burden-High Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [≥10 mutations/megabase] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.
Cutaneous Squamous Cell Carcinoma
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) or locally advanced cSCC that is not curable by surgery or radiation.
Triple-Negative Breast Cancer
KEYTRUDA is indicated for the treatment of patients with high-risk early-stage triple-negative breast cancer (TNBC) in combination with chemotherapy as neoadjuvant treatment, and then continued as a single agent as adjuvant treatment after surgery.
KEYTRUDA, in combination with chemotherapy, is indicated for the treatment of patients with locally recurrent unresectable or metastatic TNBC whose tumors express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
For more information, visit www.merck.com and connect with us on Twitter, Facebook, Instagram, YouTube and LinkedIn
Please see Prescribing Information for KEYTRUDA (pembrolizumab) at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_pi.pdf and Medication Guide for KEYTRUDA at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_mg.pdf .
Source: Merck & Co., Inc.
Merck Keytruda's add on use in early- stage lung cancer cuts death risk by 24% in study
Mar. 18, 2022 4:55 AM ET Merck & Co., Inc. (MRK)
By: Ravikash, SA News Editor
Merck (NYSE:MRK) said a phase 3 trial showed that postsurgical use of Keytruda significantly improved disease-free survival (DFS), reducing the risk of disease recurrence or death by 24% compared to placebo in patients with stage IB-IIIA non-small cell lung cancer (NSCLC) regardless of PD-L1 expression.
https://seekingalpha.com/symbol/MRK
Merck announces FDA approval of Keytruda for second indication in endometrial cancer
Mar. 21, 2022 4:45 PM ET Merck & Co., Inc. (MRK) By: Dulan Lokuwithana, SA News Editor
https://seekingalpha.com/symbol/MRK
ZEJULA® (Niraparib)
Mar 20, 2022
Zai Lab Presents Positive Results from Phase 3 PRIME Study of ZEJULA® (Niraparib) at Society of Gynecologic Oncology Meeting
- PRIME study demonstrates that niraparib treatment had a statistically significant and clinically meaningful improvement in progression-free survival in the overall study population regardless of biomarker status when compared to placebo
- The primary endpoint of the trial was met, with median progression-free survival of 24.8 months for niraparib patients versus 8.3 months for patients taking placebo
- Treatment was tolerable in the population studied and showed a safety profile consistent with previous trials
SHANGHAI and SAN FRANCISCO and CAMBRIDGE, Mass., March 20, 2022 (GLOBE NEWSWIRE) -- Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688), a patient-focused, innovative, commercial-stage, global biopharmaceutical company, today presented data from the Phase 3 PRIME study of ZEJULA (niraparib) as maintenance therapy at the Society of Gynecologic Oncology annual meeting. ZEJULA demonstrated a statistically significant and clinically meaningful improvement in progression-free survival (PFS) with a tolerable safety profile in Chinese patients with newly diagnosed advanced epithelial ovarian, fallopian tube, or primary peritoneal cancer (collectively termed as ovarian cancer) following a response to platinum-based chemotherapy, regardless of biomarker status.
In the PRIME study, median PFS was significantly longer for patients treated with niraparib compared to placebo: 24.8 months versus 8.3 months, hazard ratio (HR), 0.45; p<0.001. Other pre-specified efficacy results included:
- gBRCAmut patients: mPFS, not reached vs. 10.8 months; HR and 95% CI: 0.40 (0.23, 0.68);
- Non-gBRCAmut patients: mPFS, 19.3 months vs. 8.3 months; HR and 95% CI: 0.48 (0.34, 0.67);
- Overall survival data are still immature (percentage of death in niraparib and placebo groups are 14.5% vs. 21.7%); there is a trend in favor of niraparib at the data cut-off.
The PRIMA study previously conducted by Zai Lab’s partner GlaxoSmithKline plc (GSK) demonstrated that niraparib conferred a PFS benefit to patients with advanced ovarian cancer after a response to first-line platinum-based chemotherapy compared with placebo, regardless of biomarker status. An individualized starting dosing (ISD) based on baseline bodyweight and platelet count to personalize treatment of niraparib was used in approximately 35% of patients in PRIMA. The starting dose was individualized at 200 mg except for those patients with a baseline body weight ≥77kg and a platelet count ≥150K/μL, in which case the starting dose was 300 mg.
The current PRIME study was designed to prospectively assess the efficacy and safety of niraparib with this ISD as maintenance therapy in patients with newly diagnosed advanced ovarian cancer after a response to first-line platinum-based chemotherapy, regardless of biomarker status and postoperative residual disease status.
In PRIME, the safety profile of niraparib was improved with the ISD prospectively applied to all patients. Based on the prospective ISD with niraparib, less than 7% of patients discontinued treatment due to adverse events, the lowest rate of any PARPi Phase 3 first-line maintenance ovarian cancer trial. Compared with previous fixed starting dose, the ISD reduced the incidence of hematological treatment-emergent adverse events (TEAEs). Grade ≥3 hematological TEAEs of neutrophil count decrease, anemia, and platelet count decrease in patients treated with niraparib versus placebo were 17.3% vs. 1.6%, 18.0% vs. 1.6%, and 14.1% vs. 0.8%, respectively.
“The PRIME data continue to support niraparib monotherapy as the standard of care after first-line platinum-based chemotherapy regardless of biomarker status,” said Alan Sandler, M.D., President and Head of Global Development, Oncology, Zai Lab. “More specifically, ZEJULA is the first and only PARP inhibitor approved globally, including in China, as monotherapy for all-comer patients in the first-line maintenance treatment settings.”
“I believe the data of the PRIME study will have a significant impact on the clinical practice in the first-line treatment of ovarian cancer in China and beyond, as the individualized starting dose regimen has demonstrated an improved efficacy and safety profile,” said Dr. Lingying Wu, Director of the Department of Gynecologic Oncology, National Cancer Center / National Clinical Research Center for Cancer / Cancer Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College. “In addition, the PRIME study is the only study conducted in China that has demonstrated that a PARP inhibitor significantly improved PFS when given as first-line monotherapy maintenance treatment in all Chinese patients with newly diagnosed advanced ovarian cancer, regardless of biomarker status and postoperative residual disease status.”
In September 2020, the China National Medical Products Administration (NMPA) approved ZEJULA’s supplemental New Drug Application as a maintenance treatment of adult patients with advanced ovarian cancer who are in a complete or partial response to platinum-based chemotherapy. ZEJULA was also approved by the Hong Kong Department of Health as a maintenance treatment for adult patients with high-grade serous epithelial ovarian cancer who are in a complete response or partial response to platinum-based chemotherapy.
In December 2021, Zai Lab announced that the National Reimbursement Drug List (NRDL) released by China’s National Healthcare Security Administration (NHSA) was updated to include ZEJULA (niraparib) as a first-line maintenance treatment of adult patients with advanced ovarian cancer following a response to platinum-based chemotherapy, regardless of biomarker status.
About PRIME Study
The fully powered Phase 3 PRIME study was evaluated in 384 advanced ovarian cancer patients who were in a complete or partial response to platinum-based chemotherapy and who were randomized 2:1 to receive ZEJULA or placebo as maintenance therapy. The study evaluated the efficacy of ZEJULA as a maintenance treatment, with the primary endpoint of PFS as assessed by blinded independent central review. The starting dose was individualized at 200 mg except for those patients with a baseline body weight ≥77kg and a platelet count ≥150K/μL, in which case the starting dose was 300 mg.
About ZEJULA (niraparib)
ZEJULA (niraparib) is an oral, once-daily poly (ADP-ribose) polymerase (PARP) inhibitor indicated as monotherapy for the maintenance treatment of adult patients with advanced and recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in response (complete or partial) to first- and second-line platinum-based chemotherapy.
In addition to the PRIME study, Zai Lab has completed several studies in Chinese patients with ovarian cancer:
- In September 2020, Zai Lab announced that ZEJULA demonstrated a significant PFS benefit with an improved safety profile in the company’s Phase 3 NORA study of ZEJULA as maintenance therapy for Chinese patients with platinum-sensitive, recurrent ovarian cancer, regardless of biomarker status.
- A Phase 1 pharmacokinetic study of ZEJULA was conducted in Chinese patients with ovarian cancer.
Zai Lab has a collaboration and license agreement with GSK for the development and commercialization of ZEJULA (independently manufactured by Zai Lab) in mainland China, Hong Kong, and Macau.
For additional information about the Company, please visit www.zailaboratory.com or follow us at www.twitter.com/ZaiLab_Global.
Source: Zai Lab Limited
Zai Lab posts late-stage data for niraparib as maintenance therapy in ovarian cancer
Mar. 21, 2022 7:13 AM ET
By: Dulan Lokuwithana, SA News Editor1 Comment
- On Monday, the Chinese biotech, Zai Lab (NASDAQ:ZLAB), announced the results from its Phase 3 PRIME study for ZEJULA (niraparib) as maintenance therapy in a group of patients with ovarian cancer.
- https://seekingalpha.com/news/3815261-zai-lab-posts-late-stage-data-for-niraparib-in-ovarian-cancer
- https://seekingalpha.com/symbol/ZLAB
- https://www.zailaboratory.com/
- https://zejula.com/
- https://seekingalpha.com/symbol/GSK
Opdualag™ (nivolumab and relatlimab-rmbw)
U.S. Food and Drug Administration Approves First LAG-3-Blocking Antibody Combination, Opdualag™ (nivolumab and relatlimab-rmbw), as Treatment for Patients with Unresectable or Metastatic Melanoma
03/18/2022CATEGORY:
Opdualag is a first-in-class, fixed-dose dual immunotherapy combination treatment of the PD-1 inhibitor nivolumab and novel LAG-3-blocking antibody relatlimab1
In RELATIVITY-047, Opdualag more than doubled median progression-free survival compared to nivolumab monotherapy, an established standard of care1,2
Relatlimab is the third immune checkpoint inhibitor from Bristol Myers Squibb, adding to the Company’s growing and differentiated oncology portfolio
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced that OpdualagTM (nivolumab and relatlimab-rmbw), a new, first-in-class, fixed-dose combination of nivolumab and relatlimab, administered as a single intravenous infusion, was approved by the U.S. Food and Drug Administration (FDA) for the treatment of adult and pediatric patients 12 years of age or older with unresectable or metastatic melanoma.1 The approval is based on the Phase 2/3 RELATIVITY-047 trial, which compared Opdualag (n=355) to nivolumab alone (n=359).1,2
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20220304005561/en/
Opdualag Logo, Bristol Myers Squibb
The trial met its primary endpoint, progression-free survival (PFS), and Opdualag more than doubled the median PFS when compared to nivolumab monotherapy, 10.1 months (95% Confidence Interval [CI]: 6.4 to 15.7) versus 4.6 months (95% CI: 3.4 to 5.6); (Hazard Ratio [HR] 0.75; 95% CI: 0.62 to 0.92, P=0.0055).1 The Opdualag safety profile was similar to that previously reported for nivolumab.1,2 No new safety events were identified with the combination when compared to nivolumab monotherapy.1,2 Grade 3/4 drug-related adverse events were 18.9% in the Opdualag arm compared to 9.7% in the nivolumab arm.2 Drug-related adverse events leading to discontinuation were 14.6% in the Opdualag arm compared to 6.7% in the nivolumab arm.2
“Since the approval of the first immune checkpoint inhibitor more than 10 years ago, we’ve seen immunotherapy, alone and in combination, revolutionize the treatment of patients with advanced melanoma,” said F. Stephen Hodi, M.D., director of the Melanoma Center and the Center for Immuno-Oncology at Dana-Farber Cancer Institute.3 “Today’s approval is particularly significant, as it introduces an entirely new combination of two immunotherapies that may act together to help improve anti-tumor response by targeting two different immune checkpoints — LAG-3 and PD-1.”1,2
Opdualag is associated with the following Warnings & Precautions: severe and fatal immune-mediated adverse reactions (IMARs) including pneumonitis, colitis, hepatitis, endocrinopathies, nephritis with renal dysfunction, dermatologic adverse reactions, myocarditis and other immune-mediated adverse reactions; infusion-related reactions; complications of allogeneic hematopoietic stem cell transplantation (HSCT); and embryo-fetal toxicity.1 Please see Important Safety Information below.
“While we have made great progress in the treatment of advanced melanoma over the past decade, we are committed to expanding dual immunotherapy treatment options for these patients,” said Samit Hirawat, chief medical officer, global drug development, Bristol Myers Squibb.3 “Inhibiting LAG-3 with relatlimab, in a fixed-dose combination with nivolumab, represents a new treatment approach that builds on our legacy of bringing innovative immunotherapy options to patients. The approval of a new medicine that includes our third distinct checkpoint inhibitor marks an important step forward in giving patients more options beyond monotherapy treatment.”
Lymphocyte activation gene-3 (LAG-3) and programmed death-1 (PD-1) are two distinct inhibitory immune checkpoints that are often co-expressed on tumor-infiltrating lymphocytes, thus contributing to tumor-mediated T-cell exhaustion.2 The combination of nivolumab (anti-PD-1) and relatlimab (anti-LAG-3) results in increased T-cell activation compared to the activity of either antibody alone.1 Relatlimab (in combination with nivolumab) is the first LAG-3-blocking antibody to demonstrate a benefit in a Phase 3 study.1 It is the third checkpoint inhibitor (along with anti-PD-1 and anti-CTLA-4) for Bristol Myers Squibb.
“Today’s approval is exciting news and offers new hope to the melanoma community. The availability of this treatment combination may enable patients to potentially benefit from a new, first-in-class dual immunotherapy,” said Michael Kaplan, president and CEO, Melanoma Research Alliance.
The FDA-approved dosing for adult patients and pediatric patients 12 years of age or older who weigh at least 40 kg is 480 mg nivolumab and 160 mg relatlimab administered intravenously every four weeks.1 The recommended dosage for pediatric patients 12 years of age or older who weigh less than 40 kg, and pediatric patients younger than 12 years of age, has not been established.1
This application was approved under the FDA’s Real-Time Oncology Review (RTOR) pilot program, which aims to ensure that safe and effective treatments are available to patients as early as possible.4 The review was also conducted under the FDA’s Project Orbis initiative, which enabled concurrent review by the health authorities in Australia, Brazil and Switzerland, where the application remains under review.
About RELATIVITY-047
RELATIVITY-047 is a global, randomized, double-blind Phase 2/3 study evaluating the fixed-dose combination of nivolumab and relatlimab versus nivolumab alone in patients with previously untreated metastatic or unresectable melanoma.1,2 The trial excluded patients with active autoimmune disease, medical conditions requiring systemic treatment with moderate or high dose corticosteroids or immunosuppressive medications, uveal melanoma, and active or untreated brain or leptomeningeal metastases.1 The primary endpoint of the trial is progression-free survival (PFS) determined by Blinded Independent Central Review (BICR) using Response Evaluation Criteria in Solid Tumors (RECIST v1.1).1 The secondary endpoints are overall survival (OS) and objective response rate (ORR).1 A total of 714 patients were randomized 1:1 to receive a fixed-dose combination of nivolumab (480 mg) and relatlimab (160 mg) or nivolumab (480 mg) by intravenous infusion every four weeks until disease progression or unacceptable toxicity.1
OPDUALAG INDICATION
Opdualag TM (nivolumab and relatlimab-rmbw) is indicated for the treatment of adult and pediatric patients 12 years of age or older with unresectable or metastatic melanoma.
Please see US Full Prescribing Information for OPDIVO and YERVOY.
More information about our access and reimbursement support can be obtained by calling BMS Access Supportat 1-800-861-0048 or by visiting www.bmsaccesssupport.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220304005561/en/
Source: Bristol Myers Squibb
Bristol gains approval of first-in-class immunotherapy for melanoma Opdualag
Mar. 19, 2022 1:10 PM ET
Bristol-Myers Squibb Company (BMY)
By: Jonathan Block, SA News Editor11 Comments
- The U.S. FDA has approved Bristol-Myers Squibb's (NYSE:BMY) Opdualag, a combination of Opdivo (nivolumab) and the novel agent relatlimab, for unresectable or metastatic melanoma.
- https://seekingalpha.com/news/3815159-bristol-gains-approval-of-first-in-class-immunotherapy-for-melanoma-opdualag
- https://seekingalpha.com/symbol/BMY
- https://www.businesswire.com/news/home/20220304005561/en/
RINVOQ® (upadacitinib)
March 16, 2022
RINVOQ® (upadacitinib) Receives FDA Approval for the Treatment of Adults with Moderately to Severely Active Ulcerative Colitis
- In clinical trials, RINVOQ (upadacitinib) achieved the primary endpoints of clinical remission (per modified Mayo Score [mMS]) at weeks 8 and 521-4
- A greater proportion of RINVOQ-treated patients achieved clinical response (per partial mMS [pmMS]) as early as week 2 and steroid-free clinical remission at one year, as well as key endoscopic and histologic improvement endpoints, at weeks 8 and 524
- First approved in 2019, RINVOQ is a JAK inhibitor approved for four indications across gastroenterology, dermatology and rheumatology4
NORTH CHICAGO, Ill., March 16, 2022 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced that the U.S. Food and Drug Administration (FDA) has approved RINVOQ® (upadacitinib) for the treatment of adults with moderately to severely active ulcerative colitis (UC) who have had an inadequate response or intolerance to one or more tumor necrosis factor (TNF) blockers. This FDA approval is the first indication for RINVOQ in gastroenterology and is supported by efficacy and safety data from three Phase 3 randomized, double-blind, placebo-controlled clinical studies.
Experience the interactive Multimedia News Release here: https://www.multivu.com/players/English/8978351-abbvie-fda-ulcerative-colitis/
"There remains an unmet need for patients with moderately to severely active UC, who suffer from debilitating symptoms that are often unpredictable and burdensome," said Thomas Hudson, MD, senior vice president of research and development, chief scientific officer, AbbVie. "With the approval of RINVOQ as a new treatment option, AbbVie continues our leadership in advancing research that can help impact the lives of people living with ulcerative colitis."
The two induction studies (U-ACHIEVE and U-ACCOMPLISH) utilized RINVOQ 45 mg once daily for 8 weeks, and then 15 mg or 30 mg once daily for the maintenance study (U-ACHIEVE maintenance) through 52 weeks.1-4 Across all clinical trials, significantly more patients treated with RINVOQ achieved clinical remission at weeks 8 and 52, the primary endpoint based on the mMS: stool frequency subscore (SFS) ≤ 1 and not greater than Baseline, rectal bleeding subscore (RBS) = 0, endoscopy subscore (ES) of ≤ 1 without friability, compared to placebo. In addition, the studies met all ranked secondary endpoints, including endoscopic improvement and histologic-endoscopic mucosal improvement (HEMI), as well as corticosteroid-free clinical remission in the maintenance study. All primary and ranked secondary endpoints achieved p-values of <0.001 versus placebo.1-3
"Ulcerative colitis patients live with unpredictable symptoms such as increased stool frequency and bleeding, which can make daily activities difficult," said Maria T. Abreu, M.D., Professor of Medicine, Professor of Microbiology and Immunology, University of Miami Miller School of Medicine and Director, Crohn's & Colitis Center, University of Miami Health System.* "In clinical trials, RINVOQ showed its ability to rapidly control symptoms in just eight weeks for many patients and sustained responses at one year. I believe these types of improvements can make a positive difference for my patients."
Clinical Response and Durable Remission1-4
- During the U-ACHIEVE and U-ACCOMPLISH induction trials at week 8, 26 percent and 33 percent of patients treated with RINVOQ 45 mg achieved clinical remission, the primary endpoint based on mMS, compared to 5 percent and 4 percent of patients who received placebo.
- Onset of response occurred as early as Week 2, with a greater proportion of patients receiving RINVOQ 45 mg once daily achieving clinical response, defined as a decrease of ≥1 point and ≥30 percent from Baseline and a decrease in RBS of ≥1 or an absolute RBS ≤1 per the pmMS, compared to placebo.
- During the maintenance trial, 42 percent and 52 percent of patients treated with RINVOQ 15 mg or 30 mg, respectively, achieved clinical remission at week 52, the primary endpoint, compared to 12 percent of patients who received placebo.
- Additionally, 57 percent and 68 percent of patients receiving RINVOQ 15 mg or 30 mg, respectively, achieved corticosteroid free remission, defined as clinical remission (per mMS) and corticosteroid free for ≥90 days immediately preceding Week 52 among patients who achieved clinical remission at the end of the induction treatment, compared to 22 percent of patients on placebo.
Endoscopic Improvement and Mucosal Healing1-4
- Endoscopic improvement is defined as a ES of ≤1 without friability and was achieved in U-ACHIEVE and U-ACCOMPLISH.
- In the induction studies at week 8, endoscopic improvement was observed in 36 percent and 44 percent of patients treated with RINVOQ 45 mg in U-ACHIEVE and U-ACCOMPLISH, respectively, compared to 7 percent and 8 percent, respectively, of patients on placebo.
- In the maintenance study at week 52, endoscopic improvement was seen in 49 percent and 62 percent of patients with RINVOQ 15 mg and 30 mg, respectively, compared to 14 percent of patients on placebo.
- Mucosal healing was achieved in U-ACHIEVE and U-ACCOMPLISH and is defined by HEMI, which combines an ES of ≤1 without friability with a Geboes score of ≤ 3.1 (indicating neutrophil infiltration in <5 percent of crypts, no crypt destruction, and no erosions, ulcerations or granulation tissue); the relationship between HEMI and disease progression and/or long-term outcomes has not been established.
- Mucosal healing was observed in 30 percent and 37 percent of patients treated with RINVOQ 45 mg in U-ACHIEVE and U-ACCOMPLISH, respectively, at week 8, compared to 7 percent and 6 percent of patients, respectively, who received placebo.
- At Week 52, mucosal healing was seen in 35 percent and 50 percent of patients treated with RINVOQ 15 mg and 30 mg, respectively, compared to 12 percent patients who received placebo.
About RINVOQ® (upadacitinib)
Discovered and developed by AbbVie scientists, RINVOQ is a selective JAK inhibitor that is being studied in several immune-mediated inflammatory diseases.4,10 Based on enzymatic and cellular assays, RINVOQ demonstrated greater inhibitory potency for JAK-1 vs JAK-2, JAK-3, and TYK-2.4 The relevance of inhibition of specific JAK enzymes to therapeutic effectiveness and safety is not currently known.
In the U.S., RINVOQ 45 mg is approved for use in adult patients with moderately to severely active ulcerative colitis who have had an inadequate response or intolerance to one or more TNF blockers as an induction therapy once daily for 8 weeks. The recommended dose of RINVOQ for maintenance treatment is 15 mg once daily. A dosage of 30 mg once daily may be considered for patients with refractory, severe or extensive disease. Discontinue RINVOQ if an adequate response is not achieved with the 30 mg dose. Use the lowest effective dosage needed to maintain response. RINVOQ 15 mg once daily can be initiated in adults and children 12 years of age and older weighing at least 40 kg with refractory, moderate to severe atopic dermatitis whose disease is not adequately controlled with other system drug products, including biologics or when use of those therapies is inadvisable. In these children and adults less than 65 years of age who do not achieve an adequate response, the dose may be increased to 30 mg once daily. RINVOQ 15 mg is also approved in the U.S. for adults with moderately to severely active rheumatoid arthritis who have had an inadequate response or intolerance to one or more TNF blockers as well as adults with active psoriatic arthritis who have had an inadequate response or intolerance to one or more TNF blockers.4 In the EU, RINVOQ 15 mg is approved for the treatment of adults with moderate to severe active rheumatoid arthritis, adults with active psoriatic arthritis and adults with active ankylosing spondylitis. RINVOQ is also approved in the EU for adults (15 mg and 30 mg) and adolescents (15 mg) with moderate to severe atopic dermatitis.4
Phase 3 trials of RINVOQ in rheumatoid arthritis, atopic dermatitis, psoriatic arthritis, axial spondyloarthritis, Crohn's disease, giant cell arteritis and Takayasu arteritis are ongoing.11-17
RINVOQ (upadacitinib) U.S. Use and Important Safety Information4
RINVOQ is a prescription medicine used:
- To treat adults with moderate to severe rheumatoid arthritis when 1 or more medicines called tumor necrosis factor (TNF) blockers have been used, and did not work well or could not be tolerated.
- To treat adults with active psoriatic arthritis when 1 or more medicines called TNF blockers have been used, and did not work well or could not be tolerated.
- To treat adults with moderately to severely active ulcerative colitis when 1 or more medicines called TNF blockers have been used, and did not work well or could not be tolerated.
It is not known if RINVOQ is safe and effective in children with juvenile idiopathic arthritis, psoriatic arthritis, or with ulcerative colitis.
- To treat adults and children 12 years of age and older with moderate to severe eczema (atopic dermatitis) that did not respond to previous treatment and their eczema is not well controlled with other pills or injections, including biologic medicines, or when the use of other pills or injections is not recommended.
RINVOQ is safe and effective in children 12 years of age and older weighing at least 88 pounds (40 kg) with atopic dermatitis.
It is not known if RINVOQ is safe and effective in children under 12 years of age with atopic dermatitis.
For more information on AbbVie in gastroenterology, visit https://www.abbvie.com/our-science/therapeutic-focus-areas/immunology/immunology-focus-areas/gastroenterology.html.
For more information about AbbVie, please visit us at www.abbvie.com.
Follow @abbvie on Twitter, Facebook, LinkedIn or Instagram.
SOURCE AbbVie
AbbVie Rinvoq gets US FDA approval to treat ulcerative colitis
Mar. 17, 2022 5:52 AM ET AbbVie Inc. (ABBV)
By: Ravikash, SA News Editor4 Comments
- The U.S. Food and Drug Administration (FDA) approved AbbVie' (NYSE:ABBV) medicine Rinvoq (upadacitinib) to treat adults with moderately to severely active ulcerative colitis (UC) who have had an inadequate response or intolerance to one or more tumor necrosis factor (TNF) blockers.
- https://seekingalpha.com/news/3814446-abbvie-rinvoq-gets-us-fda-approval-to-treat-ulcerative-colitis
- https://seekingalpha.com/symbol/ABBV
- https://www.rinvoq.com/
- https://www.abbvie.com/
SPIKEVAX (mRNA-1273)
HEALTH CANADA AUTHORIZES MODERNA'S COVID-19 VACCINE IN CHILDREN (6-11 YEARS)
Announcement follows recent authorization of the Company's mRNA COVID-19 vaccine in Australia and the European Union for active immunization to prevent COVID-19 caused by SARS-CoV-2 in children aged 6-11 years.
CAMBRIDGE, MA / ACCESSWIRE / March 17, 2022 /
Moderna, Inc. (NASDAQ:MRNA), a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, today announced that Health Canada has approved the use of Moderna's mRNA COVID-19 vaccine, SPIKEVAX™, in a two-dose series of 50 µg per dose for active immunization to prevent COVID-19 caused by SARS-CoV-2 in children aged 6 to11 years. The announcement follows the recent authorization of the Company's COVID-19 vaccine in the same age group in both Australia and the European Union.
"Health Canada was the first regulator to fully approve our COVID-19 vaccine, and we are pleased they have taken this important step to expand this authorization to children aged 6 to11 years," said Stéphane Bancel, Chief Executive Officer of Moderna. "I want to thank the Government of Canada for their partnership and the trust this decision shows in the effectiveness and safety of our vaccine in this important patient population."
Moderna's vaccine was investigated in the ongoing Phase 2/3 "KidCOVE" study, a randomized, observer-blind, placebo-controlled expansion study to evaluate the safety, tolerability, reactogenicity, and effectiveness of SPIKEVAX (mRNA-1273) given to healthy children 28 days apart. Data submitted to Health Canada from over 4,000 children demonstrated that vaccination of children aged 6 to11 years with a 50 μg mRNA-1273 primary series is associated with non-inferior anti-SARS-CoV-2 neutralizing antibody responses when compared to that in individuals 18 to 25 years old from the Phase 3 COVE study. Positive direct efficacy of two 50 μg doses of mRNA-1273 was also demonstrated, and vaccination was generally well tolerated.
The study is being conducted in collaboration with the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH) and the Biomedical Advanced Research and Development Authority (BARDA), part of the Office of the Assistant Secretary for Preparedness and Response at the U.S. Department of Health and Human Services (HHS). Over 400 children in Canada were randomized into the study and the ClinicalTrials.gov identifier is NCT04796896.
Recently, the Therapeutic Goods Administration in Australia and the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) authorized Moderna's mRNA COVID-19 vaccine for active immunization to prevent COVID-19 caused by SARS-CoV-2 in children aged 6 to 11 years.
Authorized Use
Health Canada has approved the use of Moderna's mRNA COVID-19 vaccine, SPIKEVAX (elasomeran mRNA vaccine), for active immunization to prevent COVID-19 caused by SARS-CoV-2 in individuals 6 years of age and older.
To learn more, visit www.modernatx.com.
SOURCE: Moderna, Inc.
View source version on accesswire.com:
https://www.accesswire.com/693551/Health-Canada-Authorizes-Modernas-COVID-19-Vaccine-in-Children-6-11-Years
mRNA-1273/Spikevax®
https://www.modernatx.com/mrna-1273spikevax%C2%AE
Moderna COVID-19 shot cleared in Canada for children aged 6 to 11 years
Mar. 17, 2022 11:14 AM ET
By: Dulan Lokuwithana, SA News Editor7 Comments
- Moderna (NASDAQ:MRNA) announced on Thursday that Health Canada approved its COVID-19 vaccine, branded as Spikevax, for immunization against COVID-19 in children aged 6 to11 years.
- https://seekingalpha.com/news/3814647-moderna-covid-19-shot-cleared-in-canada-for-children-aged-6-to-11-years
- https://seekingalpha.com/symbol/MRNA
- https://www.modernatx.com/mrna-1273spikevax%C2%AE
- https://www.modernatx.com/
Evusheld▼ (tixagevimab co-packaged with cilgavimab)
AstraZeneca’s antibody combination, Evusheld▼ (tixagevimab co-packaged with cilgavimab) authorised for use in Great Britain for pre-exposure prophylaxis (prevention) of COVID-19
PUBLISHED17 March 2022
17 March 2022, London, UK: 07:00 GMT
Evidence shows efficacy and protection (estimated for at least six months) after one dose in high-risk populations*
AstraZeneca's Evusheld▼ (tixagevimab co-packaged with cilgavimab) is authorised by the Medicines and Healthcare products Regulatory Agency (MHRA) and is the first antibody combination for pre-exposure prophylaxis (PrEP) against COVID-19 licensed in Great Britain. The use of this medicine is for adults who are not currently infected with (or know exposure to) the COVID-19 virus and are unlikely to mount an adequate response to COVID-19 vaccination – including those for whom vaccination is not recommended.1
Tom Keith Roach, President, AstraZeneca UK, said: “Evusheld fills an urgent gap in the UK’s fight against COVID-19, providing protection for people for whom vaccination may not be effective and who are often amongst the most clinically vulnerable in society. We hope to see this critical medicine made available to UK patients as quickly as possible, in line with other countries.”
Hugh Montgomery, Professor of Intensive Care Medicine at University College London, said: “This announcement is really good news. Sensible public health actions with vaccination are the mainstay of protection for most individuals in the UK. However, for a considerable number in society with existing health problems, protection against the virus through vaccination is limited. Availability of this antibody medicine now offers an increased likelihood of pre-exposure protection, and all from a single, effective immunisation that can last for months."
Tixagevimab co-packaged with cilgavimab, formerly known as AZD7442, is a combination of two monoclonal antibodies given as separate sequential intramuscular (IM) injections.1
About 500,000 people in the UK are immunocompromised and may benefit from this medicine for pre-exposure prophylaxis of COVID-19.2 Nearly 40% of people with immunocompromised or immunosuppressed conditions mount a low or undetectable immune response after vaccination and approximately 11% fail to generate any antibodies.3 This includes people with blood cancers, those taking immunosuppressive drugs after an organ transplant or for conditions including multiple sclerosis and rheumatoid arthritis.3
The primary data from the ongoing PROVENT Phase III trial (which met its primary endpoint) showed a statistically significant reduction in the risk of developing symptomatic COVID-19 with AZD7442 compared to placebo. The trial has shown protection from the virus continuing for at least six months. The antibody was tolerated and follow-up is needed to establish the full duration of protection.1
Based on the primary analysis of 5,172 participants (AZD7442 n = 3,441 and saline placebo n = 1,731), the antibody combination demonstrated a 77% relative risk reduction (RRR) in incidence of symptomatic COVID-19 [95% Confidence Interval (CI) 46-90; p <0.001; 8/3,441 (0.2%) AZD7442 and 17/1,731 (1.0%) placebo], and 0.8% Absolute Risk Reduction (ARR) compared to placebo, with median follow-up time post-administration of 83 days.1,4
In a subsequent analysis, the antibody combination demonstrated an 83% RRR in the incidence of symptomatic COVID-19 [95% CI: 66-91; 11/3,441 (0.3%) AZD7442 and 31/1,731 (1.8%) placebo] and 1.5% ARR compared to placebo, after a median 6.5-month follow-up.1
No hospitalisation or deaths were seen in the treatment arm. In the placebo arm, there were five cases of severe COVID-19 and two COVID-19 related deaths. Adverse events were reported in 35% (1,221/3,461) of participants receiving AZD7442 and 34% (593/1,736) receiving placebo, with the vast majority being mild to moderate in nature. The most frequently reported adverse reaction in the pooled analysis was injection site reaction.4,5
*Available data indicate that tixagevimab and cilgavimab may be effective for pre-exposure prophylaxis for six months based on the variants prevalent during the study. The trial is ongoing and more data is being generated to establish an exact duration of prophylactic protection. It is not known how pseudovirus or authentic SARS-CoV-2 neutralisation susceptibility data correlate with clinical outcome however data across multiple independent studies (pseudovirus and authentic ‘live’ virus) show that the anitbody combination retained neutralising activity against Omicron.6,7,8
For more information, please visit www.astrazeneca.co.uk and follow us on Twitter @AstraZenecaUK.
AstraZeneca's Evusheld gets approval in UK to prevent COVID-19
Mar. 17, 2022 5:10 AM ET
By: Ravikash, SA News Editor1 Comment
- U.K.'s Medicines and Healthcare products Regulatory Agency (MHRA) approved AstraZeneca's (NASDAQ:AZN) antibody cocktail Evusheld for preventing COVID-19.
- https://seekingalpha.com/news/3814440-astrazenecas-evusheld-gets-approval-in-uk-to-prevent-covid-19
- https://seekingalpha.com/symbol/AZN
- https://www.gov.uk/government/news/evusheld-approved-to-prevent-covid-19-in-people-whose-immune-response-is-poor
Lynparza (olaparib)
Lynparza approved in the US as adjuvant treatment for patients with germline BRCA-mutated HER2-negative high-risk early breast cancer
PUBLISHED11 March 2022
11 March 2022 21:00 GMT
First and only approved medicine targeting BRCA mutations in early breast cancer
New data show Lynparza demonstrated overall survival benefit in early breast cancer
AstraZeneca and MSD’s Lynparza (olaparib) has been approved in the US for the adjuvant treatment of patients with germline BRCA-mutated (gBRCAm) HER2-negative high-risk early breast cancer who have already been treated with chemotherapy either before or after surgery.
The approval by the US Food and Drug Administration (FDA) was based on results from the OlympiA Phase III trial presented during the 2021 American Society of Clinical Oncology Annual Meeting and published in The New England Journal of Medicine.1
In the trial, Lynparza demonstrated a statistically significant and clinically meaningful improvement in invasive disease-free survival (iDFS), reducing the risk of invasive breast cancer recurrences, second cancers or death, by 42% versus placebo (based on a hazard ratio [HR] of 0.58; 95% confidence interval [CI] 0.46-0.74; p<0.0001).
New updated results from the OlympiA trial also showed Lynparza demonstrated a statistically significant and clinically meaningful improvement in the key secondary endpoint of overall survival (OS), reducing the risk of death by 32% versus placebo (based on a HR of 0.68; 95% CI 0.50-0.91; p=0.0091). The safety and tolerability profile of Lynparza in this trial was in line with that observed in prior clinical trials. The OS data will be presented at an upcoming European Society for Medical Oncology virtual plenary on 16 March 2022.
Breast cancer is the most diagnosed cancer worldwide with an estimated 2.3 million patients diagnosed in 2020.2 Almost 91% of all breast cancer patients in the US are diagnosed at an early stage of disease and BRCA mutations are found in approximately 5-10% of patients.3,4
Professor Andrew Tutt, Global Chair of the OlympiA Phase III trial and Professor of Oncology at The Institute of Cancer Research, London and King’s College London, said: “Today’s approval of olaparib is great news for patients with a specific inherited form of breast cancer. Most breast cancers are identified in the early stages and many patients will do very well, but for those with higher risk disease at diagnosis, the risk of cancer returning can be unacceptably high and new treatment options are needed. OlympiA has shown that identifying a BRCA1/2 mutation in women with high risk disease opens the additional option of eligibility for olaparib treatment, which reduces the risk of recurrence and improves survival for these breast cancer patients.”
Dave Fredrickson, Executive Vice President, Oncology Business Unit, AstraZeneca, said: “This important approval gives early-stage breast cancer patients in the US with a germline BRCA mutation a new targeted therapy option in the adjuvant setting starting today. Lynparza reduces the risk of disease recurrence in these high-risk patients and now new data confirm it also significantly extends patients’ lives versus placebo. These data underline the importance of germline BRCA testing as soon as possible after diagnosis to identify patients that may be eligible for Lynparza.”
Roy Baynes, Senior Vice President and Head of Global Clinical Development, Chief Medical Officer, MSD Research Laboratories, said: “For patients with germline BRCA-mutated, HER2-negative high-risk early breast cancer, who often present with more aggressive disease, today’s approval is an important step forward. Compared to placebo, Lynparza as adjuvant treatment offers these patients the potential to live longer without their cancer recurring. We thank the patients, caregivers and healthcare providers for their participation in the OlympiA trial.”
Lynparza is now indicated for the adjuvant treatment of adult patients with deleterious or suspected deleterious gBRCAm HER2-negative high-risk early breast cancer who have been treated with neoadjuvant or adjuvant chemotherapy. Patients are to be selected for treatment based on an FDA-approved companion diagnostic test for Lynparza.
Lynparza is approved in the US, EU, Japan and several other countries for the treatment of patients with gBRCAm, HER2-negative, metastatic breast cancer previously treated with chemotherapy based on results from the OlympiAD Phase III trial. In the EU, this indication also includes patients with locally advanced breast cancer.
Lynparza
Lynparza (olaparib) is a first-in-class PARP inhibitor and the first targeted treatment to block DNA damage response (DDR) in cells/tumours harbouring a deficiency in homologous recombination repair (HRR), such as those with mutations in BRCA1 and/or BRCA2, or those where deficiency is induced by other agents (such as new hormonal agents – NHAs).
Inhibition of PARP proteins with Lynparza leads to the trapping of PARP bound to DNA single-strand breaks, stalling of replication forks, their collapse and the generation of DNA double-strand breaks and cancer cell death.
Lynparza is currently approved in a number of countries across PARP-dependent tumour types with defects and dependencies in the DDR pathway including maintenance treatment of platinum-sensitive relapsed ovarian cancer and as both monotherapy and in combination with bevacizumab for the 1st-line maintenance treatment of BRCA-mutated (BRCAm) and homologous recombination repair deficient (HRD)-positive advanced ovarian cancer, respectively; for germline BRCAm, HER2-negative metastatic breast cancer (in the EU this includes locally advanced breast cancer); for germline BRCAm metastatic pancreatic cancer; and HRR gene-mutated metastatic castration-resistant prostate cancer (BRCAm only in the EU and Japan).
Lynparza, which is being jointly developed and commercialised by AstraZeneca and MSD, is the foundation of AstraZeneca's industry-leading portfolio of potential new medicines targeting DDR mechanisms in cancer cells.
The AstraZeneca and MSD strategic oncology collaboration
In July 2017, AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US, known as MSD outside the US and Canada, announced a global strategic oncology collaboration to co-develop and co-commercialise Lynparza, the world’s first PARP inhibitor, and Koselugo (selumetinib), a mitogen-activated protein kinase (MEK) inhibitor, for multiple cancer types.
Working together, the companies will develop Lynparza and Koselugo in combination with other potential new medicines and as monotherapies. The companies will develop Lynparza and Koselugo in combination with their respective PD-L1 and PD-1 medicines independently.
Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca.
AstraZeneca and Merck's Lynparza gets FDA nod for early breast cancer
Mar. 11, 2022 6:42 PM ET Merck & Co., Inc. (MRK), AZN By: Jonathan Block, SA News Editor
- The U.S. FDA has granted an additional indication for AstraZeneca (NASDAQ:AZN) and Merck's (NYSE:MRK) Lynparza (olaparib) as an adjuvant treatment for a type of early-stage breast cancer.
- https://seekingalpha.com/news/3812883-astrazeneca-and-merck-lynparza-gets-fda-nod-for-early-breast-cancer
- https://seekingalpha.com/symbol/AZN
- https://seekingalpha.com/symbol/MRK
- https://www.lynparza.com/
Lynparza reduced risk of death by 32% in the adjuvant treatment of patients with germline BRCA-mutated high-risk early breast cancer PUBLISHED 16 March 2022 16 March 2022 17:30 GMT First PARP inhibitor to demonstrate overall survival benefit in early breast cancer
AstraZeneca and Merck's Lynparza shows survival benefit in early breast cancer Mar. 16, 2022 5:00 PM ET
Merck & Co., Inc. (MRK), AZN
By: Jonathan Block, SA News Editor
https://seekingalpha.com/symbol/MRK
https://seekingalpha.com/symbol/AZN
Dexcom G7
Dexcom G7 Receives CE Mark – Next-Generation Continuous Glucose Monitoring System to Revolutionize Diabetes Management
- The best-selling real-time CGM in the world1 is now more powerful and easier to use with a small, all-in-one wearable and completely redesigned mobile app
- New system has the fastest sensor warmup on the market,* with no fingersticks† or scanning required
- Dexcom G7 builds on the trusted performance of Dexcom CGM, which is clinically proven to lower A1C, reduce hyper- and hypoglycemia, and increase time in range2,3
EDINBURGH, Scotland & SAN DIEGO--(BUSINESS WIRE)--Mar. 14, 2022-- DexCom, Inc. (NASDAQ:DXCM), the global leader in real-time continuous glucose monitoring for people with diabetes, announced today it has secured CE Mark (Conformité Européenne) for the Dexcom G7 Continuous Glucose Monitoring (CGM) System for people with diabetes in Europe age two years and older, including pregnant women.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20220309005998/en/
With Dexcom G7, real-time glucose readings are sent automatically to a compatible display device. (Photo: Business Wire)
Dexcom G7 offers a better way to help users gain greater control of their diabetes, so that they can manage it more confidently each day. Its low-profile, all-in-one wearable warms up faster than any other CGM on the market,* sending real-time glucose readings automatically to a compatible smart device‡ or receiver, no fingersticks required.† Dexcom G7 also offers a suite of customizable alerts that can warn of high or low glucose levels and help users spend more time in range.§,2 Industry-leading remote monitoring and reporting capabilities also allow users to stay connected with their loved ones and care teams anytime, anywhere.||
“Today marks an incredible milestone for our company and for people with diabetes in Europe,” said Kevin Sayer, chairman, president and CEO of Dexcom. “This all-new platform offers an incredibly powerful CGM that is simple to use, providing our users with insightful glucose data on one screen that helps them spend less time managing diabetes and more time doing the things they love. Dexcom G7 takes everything people love about G6 and makes it even better.”
New features with Dexcom G7:
- 60 percent smaller, all-in-one, discreet wearable, developed in partnership with Verily
- 30-minute sensor warm up, fastest of any CGM on the market*
- 12-hour grace period to replace finished sensors for a more seamless transition between sessions
- Redesigned and simplified mobile app with Dexcom Clarity integration#,**
- Improved alert settings for enhanced discretion
- Redesigned optional receiver that is smaller, with a more vibrant, easier to read display
- Direct to Apple Watch (anticipated future software release)
Visit Dexcom.com and get started with Dexcom G6 today to experience the benefits of CGM now. To learn more about Dexcom G7 and for additional information about when it will be available in Europe, visit Dexcom.com/G7.
Dexcom submitted a comprehensive 510(k) pre-market notification to the U.S. Food and Drug Administration for regulatory review of the Dexcom G7 CGM System in accordance with the iCGM Special Controls in Q4 2021.††
About DexCom, Inc.
Dexcom, Inc. empowers people to take control of diabetes through innovative continuous glucose monitoring (CGM) systems. Headquartered in San Diego, California, and with operations across Europe, Dexcom has emerged as a leader of diabetes care technology. By listening to the needs of users, caregivers, and providers, Dexcom simplifies and improves diabetes management around the world. For more information about Dexcom CGM, visit www.dexcom.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220309005998/en/
Source: DexCom, Inc.
Dexcom glucose monitoring system for managing diabetes gets CE Mark in Europe
Mar. 14, 2022 4:52 AM ET
By: Ravikash, SA News Editor
- DexCom (NASDAQ:DXCM) said it secured CE Mark (Conformité Européenne) for the Dexcom G7 Continuous Glucose Monitoring system for people with diabetes in Europe age two years and older, including pregnant women.
- https://seekingalpha.com/news/3812960-dexcom-glucose-monitoring-system-for-managing-diabetes-gets-ce-mark-in-europe
- https://seekingalpha.com/symbol/DXCM
- https://www.dexcom.com/
Somatuline® Autogel®/Somatuline® Depot (lanreotide)
MARCH 8, 2022
New patient-reported data demonstrated high satisfaction levels and fewer patients reporting injection-site pain with Somatuline® Autogel®/Somatuline® Depot (lanreotide)
- New data presented at ENETS 2022 showed patients treated with Somatuline® Autogel®/Somatline® Depot (lanreotide) pre-filled syringe reported less frequent injection-site pain lasting more than two days than those treated with octreotide long-acting release syringe
- People living with gastroenteropancreatic neuroendocrine tumors enrolled on Somatuline® Autogel® patient support programs reported feeling in control and satisfied when administering at-home injections
PARIS, France, 8 March 2022 – Ipsen (Euronext: IPN; ADR: IPSEY) published today new data from seven abstracts to be presented at the hybrid-setting 19th Annual European Neuroendocrine Tumor Society (ENETS) Conference, 10-11 March 2022, in Barcelona, Spain. Presentations include data from the PRESTO 2 and HomeLAN surveys which demonstrated patient-reported benefits when administering Somatuline® Autogel®/Somatuline® Depot (lanreotide). These include fewer patients reporting experiencing injection-site pain and high levels of injection experience satisfaction when participating in patient support programs (PSP), respectively.
Data from PRESTO 2, an e-survey which evaluated injection site pain in people living with gastroenteropancreatic neuroendocrine tumors (GEP-NETs) or acromegaly (n=219 and n=85), demonstrated that significantly fewer patients receiving Somatuline Autogel/Somatuline Depot pre-filled syringe had reported injection-site pain lasting more than two days after their last dose compared with the octreotide long acting release syringe (OCT) (6.0% vs 22.8% [primary endpoint]; odds ratio, adjusted for disease group and occurrence of injection-site reaction: 0.13 [95% confidence interval (CI) 0.06, 0.30]; p<0.0001).1 Furthermore, compared with OCT, fewer patients treated with Somatuline Autogel/Somatuline Depot, reported interference with daily life as a result of injection-site pain (41% vs 60%).1
About PRESTO 21
PRESTO 2 is an international patient survey to evaluate impact of injection and delivery system on local pain when administrating somatostatin analogue (SSA) therapy. An e-survey of adults with NETs or acromegaly from Canada, the USA, UK and Ireland, who had received more than three months’ of treatment with LAN or OCT (n=304), was used to investigate the proportion of patients with injection-site pain lasting more than two days after last injection (primary end point) as well as interference with daily life as a result of injection-site pain (among secondary end points).
About HomeLAN2
HomeLAN is a non-interventional, cross-sectional survey of adults with NETs, enrolled in PSPs in Belgium, Greece, Spain and The Netherlands for six or more months and receiving LAN at home. The target sample size was 120, based on the number of eligible patients and a 15% response rate. Endpoints included patient satisfaction with the most recent LAN injection (primary) and reasons for choosing their mode of administration (among secondary endpoints).
About Somatuline® Autogel®/Somatuline® Depot (lanreotide)7
Somatuline® Autogel®/Somatuline® Depot is made of the active substance lanreotide and is a long-acting somatostatin analogue that inhibits the secretion of growth hormone and certain hormones secreted by the digestive system. The licensed indications of Somatuline® Autogel® are7:
- The treatment of individuals with acromegaly when the circulating levels of Growth Hormone and/or Insulin-like Growth Factor-1 remain abnormal after surgery and/or radiotherapy, or in patients who otherwise require medical treatment.
- The treatment of grade 1 and a subset of grade 2 (Ki-67 index up to 10%) gastroenteropancreatic neuroendocrine tumors (GEP-NETs) of midgut, pancreatic or unknown origin where hindgut sites of origin have been excluded, in adult patients with unresectable locally advanced or metastatic disease.
- The treatment of symptoms associated with neuroendocrine (particularly carcinoid) tumors. The recommended starting dose is one injection of Somatuline Autogel 60 to 120 mg administered every 28 days.
The decision regarding independent administration of Somatuline Autogel/Somatuline Depot is only applicable to countries where this option is approved in the Product Information/Summary of Product Characteristics and for patients on stable dose upon HCP decision and after appropriate training by HCP.
The detailed recommendations for the use of Somatuline® Autogel® are described in the Summary of Product Characteristics (SmPC) and US Prescribing Information (PI).
For more information, visit ipsen.com.
Ipsen's data shows patient benefit when using Somatuline
Mar. 08, 2022 7:09 AM ET Ipsen S.A. (IPSEY), IPSEF
By: Ravikash, SA News Editor
- Ipsen (OTCPK:IPSEF) (OTCPK:IPSEY) reported data from PRESTO 2 and HomeLAN surveys which showed patient-reported benefits when administering Somatuline Autogel/Somatuline Depot (lanreotide).
- https://seekingalpha.com/news/3810532-ipsens-data-shows-patient-benefit-when-using-somatuline
- https://seekingalpha.com/symbol/IPSEF
- https://seekingalpha.com/symbol/IPSEY
- https://www.somatulinedepot.com/
- https://www.ipsen.com/our-science/
- https://www.ipsen.com/
Jardiance® (empagliflozin)
Jardiance® (empagliflozin) becomes the first and only approved treatment in Europe for adults with symptomatic chronic heart failure regardless of ejection fraction
Ingelheim, Monday, 03/07/2022 - 08:00
- The breakthrough approval expands the existing indication of Jardiance® to include adults with heart failure with preserved ejection fraction (HFpEF), an underserved patient population that previously had no approved therapies in Europe
- European marketing authorization follows the U.S. Food and Drug Administration (FDA)’s approval received on 24 February 2022
Ingelheim, Germany, 07 March 2022 – The European Commission (EC) has granted marketing authorization for Jardiance® (empagliflozin) as a treatment for adults with symptomatic chronic heart failure, Boehringer Ingelheim and Eli Lilly and Company have announced.1 The landmark approval makes Jardiance® the first and only approved treatment for all adults with symptomatic chronic heart failure, which includes patients across the full spectrum of LVEF, including both heart failure with reduced and preserved ejection fraction (HFrEF and HFpEF).
About the EMPEROR heart failure clinical trials7,8
The EMPEROR (EMPagliflozin outcomE tRial in patients with chrOnic heaRt failure) chronic heart failure trials were two Phase III, randomized, double-blind trials that investigated once-daily empagliflozin compared to placebo in adults with chronic HFrEF or HFpEF, with or without diabetes:
- EMPEROR-Reduced [NCT03057977] investigated the safety and efficacy of empagliflozin in patients with chronic HFrEF.
o Primary endpoint: time to first event of adjudicated cardiovascular death or adjudicated hospitalization for heart failure
o Number of patients: 3,730
o Completion: 2020
- EMPEROR-Preserved [NCT03057951] investigated the safety and efficacy of empagliflozin in patients with chronic HFpEF.
o Primary endpoint: time to first event of adjudicated cardiovascular death or adjudicated hospitalization for heart failure
o Number of patients: 5,988
o Completion: 2021
About the EMPOWER program
The Alliance has developed the EMPOWER program to explore the impact of empagliflozin on major clinical cardiovascular and renal outcomes in a spectrum of cardio-renal-metabolic conditions. Cardio-renal-metabolic conditions are the leading cause of mortality worldwide and account for up to 20 million deaths annually.9 Through the EMPOWER program, Boehringer Ingelheim and Lilly are working to advance knowledge of these interconnected systems and create care which offers integrated, multi-organ benefits. Comprised of nine clinical trials and two real-world evidence studies, EMPOWER reinforces the long-term commitment of the Alliance to improve outcomes for people living with cardio-renal-metabolic conditions. With more than 400,000 adults enrolled worldwide in studies, it is one of the broadest and most comprehensive clinical programs for an SGLT2 inhibitor to date.
About empagliflozin
Empagliflozin (marketed as Jardiance®) is an oral, once-daily, highly selective sodium-glucose cotransporter 2 (SGLT2) inhibitor and the first type 2 diabetes medicine to include cardiovascular death risk reduction data in its label in several countries.18,19
Boehringer Ingelheim and Eli Lilly and Company
In January 2011, Boehringer Ingelheim and Eli Lilly and Company announced an Alliance that centers on compounds representing several of the largest diabetes treatment classes. Depending on geographies, the companies either co-promote or separately promote the respective molecules each contributing to the Alliance. The Alliance leverages the strengths of two of the world’s leading pharmaceutical companies to focus on patient needs. By joining forces, the companies demonstrate their commitment, not only to the care of people with diabetes, but also to investigating the potential to address areas of unmet medical need. Clinical trials have been initiated to evaluate the impact of empagliflozin on people living with heart failure or chronic kidney disease.
About Boehringer Ingelheim
Boehringer Ingelheim is working on breakthrough therapies that improve the lives of humans and animals. As a leading research-driven biopharmaceutical company, the company creates value through innovation in areas of high unmet medical need. Founded in 1885 and family-owned ever since, Boehringer Ingelheim takes a long-term perspective. Around 52,000 employees serve more than 130 markets in the three business areas, Human Pharma, Animal Health, and Biopharmaceutical Contract Manufacturing. Learn more at www.boehringer-ingelheim.com.
About Eli Lilly and Company
Lilly is a global health care leader that unites caring with discovery to create medicines that make life better for people around the world. We were founded more than a century ago by a man committed to creating high-quality medicines that meet real needs, and today we remain true to that mission in all our work. Across the globe, Lilly employees work to discover and bring life-changing medicines to those who need them, improve the understanding and management of disease, and give back to communities through philanthropy and volunteerism. To learn more about Lilly, please visit us at lilly.com and lilly.com/newsroom.
Lilly, Boehringer Ingelheim's Jardiance gets EU approval for expanded use in heart failure
Mar. 08, 2022 5:42 AM ET Eli Lilly and Company (LLY)
By: Ravikash, SA News Editor
- The European Commission (EC) approved the expanded use of Eli Lilly (NYSE:LLY) and Boehringer Ingelheim's Jardiance (empagliflozin) to treat adults with symptomatic chronic heart failure.
- https://seekingalpha.com/news/3810496-lilly-boehringer-ingelheims-jardiance-gets-eu-approval-for-expanded-use-in-heart-failure
- https://seekingalpha.com/symbol/LLY
- https://www.jardiance.com/
- https://www.lilly.com/
Atogepant (QULIPTA™)
March 10, 2022
AbbVie Announces Positive Phase 3 Atogepant (QULIPTA™) Data for the Preventive Treatment of Chronic Migraine
- Pivotal Phase 3 study evaluating atogepant in adult patients with chronic migraine meets primary endpoint of statistically significant reduction from baseline in mean monthly migraine days compared to placebo across the 12-week treatment period¹
- Study also demonstrates statistically significant improvements in all secondary endpoints¹
- Overall safety profile is consistent with safety findings observed in previous studies with an episodic migraine population¹
- Data from this study will support a submission to expand the use of atogepant to include preventive treatment of chronic migraine in the United States and additional submissions globally
- These results strengthen AbbVie's commitment to advancing its portfolio of medicines to help more people living with migraine
NORTH CHICAGO, Ill., March 10, 2022 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced that the Phase 3 PROGRESS trial evaluating atogepant (QULIPTA™ in the United States), an oral calcitonin gene-related peptide (CGRP) receptor antagonist (gepant) for the preventive treatment of chronic migraine in adults, met its primary endpoint of statistically significant reduction from baseline in mean monthly migraine days compared to placebo, for both the 60 mg once daily (QD) and 30 mg twice daily (BID) doses, across the 12-week treatment period. The study also demonstrated that treatment with atogepant 60 mg QD and 30 mg BID resulted in statistically significant improvements in all secondary endpoints after adjustment for multiple comparisons.1
This Phase 3, global, randomized, double-blind, placebo-controlled, parallel-group study evaluated the efficacy, safety, and tolerability of oral atogepant for the preventive treatment of chronic migraine, which is a debilitating neurological disease where patients experience headache occurring on 15 or more days per month for more than three months, which on at least eight days per month has features of migraine headache.2,3 A total of 778 patients with at least a one-year history of chronic migraine were randomized into one of three treatment groups to receive 60 mg QD of atogepant, 30 mg BID of atogepant, or placebo.2
Efficacy was analyzed using two slightly different definitions of the patient population based on regulatory agency feedback in the United States and European Union. The United States-focused, modified intent-to-treat (mITT) population included 755 patients with evaluable headache eDiary data collected during the double-blinded treatment period. The European Union-focused off-treatment hypothetical estimand (OTHE) population included 760 patients with evaluable headache eDiary data collected during the double-blind treatment period and the follow-up period.
Across the 12 weeks, based on the mITT population, patients in the atogepant 60 mg QD and 30 mg BID treatment arms of the study, experienced a decrease of 6.88 and 7.46 monthly migraine days, respectively, compared to patients in the placebo arm, who experienced a decrease of 5.05 monthly migraine days (60 mg QD vs. placebo, p=0.0009; 30 mg BID vs. placebo, p<0.0001, adjusted for multiple comparisons). Based on the OTHE population, across the 12 weeks, patients in the 60 mg QD and 30 mg BID of atogepant treatment arms of the study, experienced a decrease of 6.75 and 7.33 monthly migraine days, respectively, compared to patients in the placebo arm, who experienced a decrease of 5.09 monthly migraine days (60 mg QD vs. placebo, p=0.0024; 30 mg BID vs. placebo, p=0.0001, adjusted for multiple comparisons).1
The study demonstrated that treatment with atogepant 60 mg QD and 30 mg BID resulted in statistically significant improvements in all secondary endpoints for both efficacy analysis populations.
A key secondary endpoint measured the proportion of patients that achieved at least a 50% reduction in mean monthly migraine days across the 12-week treatment period. Based on the mITT population, the trial demonstrated that 41.0%/42.7% of patients in the 60 mg QD and 30 mg BID atogepant arms, respectively, achieved at least a 50% reduction, compared to 26.0% of patients in the placebo arm (all dose groups vs. placebo, p≤0.0009, adjusted for multiple comparisons). Based on the OTHE population, the trial demonstrated that 40.1%/42.1% of patients in the 60 mg QD and 30 mg BID atogepant arms, respectively, achieved at least a 50% reduction, compared to 26.5% of patients in the placebo arm (all dose groups vs. placebo, p≤0.0024, adjusted for multiple comparisons). 1
The overall safety profile of the Phase 3 PROGRESS study was consistent with safety findings observed in previous studies in an episodic migraine population. The most common adverse events reported with a frequency ≥ 5% in at least one atogepant treatment arm, and greater than placebo, were constipation (10.0% for atogepant 60 mg QD, 10.9% for atogepant 30 mg BID, and 3.1% for placebo), and nausea (9.6% for atogepant 60 mg QD, 7.8% for atogepant 30 mg BID, and 3.5% for placebo). Most of the events of constipation and nausea were mild or moderate in severity. Most cases of constipation and nausea did not lead to discontinuation. There were no hepatic safety issues identified. Serious adverse events occurred in 2.7% of patients with atogepant 60 mg QD and 1.6 % of patients treated with atogepant 30 mg BID, compared to 1.2% of patients with placebo. None of these treatment-emergent adverse events were assessed as treatment-related by the investigator.1
"AbbVie has nearly 12 years of experience in treating chronic migraine, a debilitating disease. We know that no two migraine patients are alike, so it is important for health care providers to have a variety of treatment options," said Michael Severino, M.D., vice chairman and president, AbbVie. "These data and pending regulatory submissions solidify our commitment to our leading migraine portfolio to help the more than one billion people worldwide living with the migraine. We look forward to taking the next steps to potentially expand the use of atogepant in the United States to include the preventive treatment of chronic migraine in adults, and to working with regulatory agencies globally on additional submissions."
These data build on the Phase 3 ADVANCE study results, which evaluated atogepant for the preventive treatment of episodic migraine.4 The primary endpoint of the Phase 3 ADVANCE study was a statistically significant reduction in mean monthly migraine days across the 12-week treatment period compared to placebo.4
Based on the results of phase 3 PROGRESS trial in chronic migraine, AbbVie intends to submit a supplemental New Drug Application (sNDA) with the U.S. Food and Drug Administration for the expanded use of atogepant to include the preventive treatment of chronic migraine. Additionally, study results from the Phase 3 PROGRESS trial, along with the Phase 3 ADVANCE trial data, in episodic migraine, will form the basis for future regulatory submissions globally. Use of atogepant for the preventive treatment of chronic migraine in the United States is not approved and its safety and efficacy have not been evaluated by regulatory authorities. Use of atogepant for the preventive treatment of episodic migraine and chronic migraine outside of the United States is not approved and its safety and efficacy have not been evaluated by regulatory authorities.
Full results from the Phase 3 PROGRESS trial will be presented at future medical meetings and more information about the trial can be found at www.clinicaltrials.gov (NCT03855137).
About the Phase 3 PROGRESS Clinical Trial
The Phase 3 PROGRESS clinical trial evaluated the safety, tolerability and efficacy of oral atogepant for the preventive treatment of chronic migraine.2 The patient population for the study included patients with a diagnosis of chronic migraine for at least one year, and greater than or equal to 15 headache days with greater than or equal to eight migraine days in the 28 days prior to randomization.2 The primary endpoint measured the reduction from baseline in mean monthly migraine days compared to placebo, for both doses, including 60 mg once daily (QD) and 30 mg twice daily (BID), across a 12-week treatment period.2
Key secondary endpoints for all regions included: Change from baseline in mean monthly headache days across the 12-week of treatment period (baseline is defined as the number of migraine days during the last 28 days prior to the randomization date); Change from baseline in mean monthly acute medication use days across the 12-week treatment period (baseline is defined as the number of migraine days during the last 28 days prior to the randomization date); Proportion of participants with at least a 50% reduction in mean monthly migraine days across the 12-week treatment period; and change from baseline in MSQ v2.1 Role Function-Restrictive domain score at Week 12. The MSQ v2.1 is a questionnaire designed to measure health-related quality of life impairments attributed to migraine in the past four weeks. It is divided into three domains, assessing how a patient's daily, social, and work activities are limited by migraine; how migraine prevents these activities; and assesses the emotional function related with migraine.
For a full listing of secondary endpoints across all regions, please go to www.clinicaltrials.gov (NCT03855137).
About QULIPTA™ (atogepant)
QULIPTA™, which was approved by the U.S. Food and Drug Administration (FDA) in September 2021, is available in the United States as the first and only oral calcitonin gene-related peptide (CGRP) receptor antagonist (gepant) in the country specifically developed for the preventive treatment of episodic migraine.5 CGRP and its receptors are expressed in regions of the nervous system associated with migraine pathophysiology, and studies have shown that CGRP levels are elevated during migraine attacks.6 QULIPTA blocks CGRP receptors through a once-daily dose and is available in three strengths – 10 mg, 30 mg and 60 mg.5
Please see full Prescribing Information and Patient Information.
For more information about AbbVie, please visit us at www.abbvie.com.
SOURCE AbbVie
AbbVie to seek label expansion of migraine therapy after main goal met in late-stage study
Mar. 10, 2022 9:05 AM ET AbbVie Inc. (ABBV) By: Dulan Lokuwithana, SA News Editor
AbbVie (NYSE:ABBV) said on Thursday that its Phase 3 trial for migraine therapy atogepant met the primary endpoint among adult patients suffering from the chronic form of the disease.
Atogepant, branded as QULIPTA in the U.S., is an FDA-approved therapy for the preventive treatment of episodic migraine.
https://seekingalpha.com/symbol/ABBV
Tislelizumab (BGB-A317)
China NMPA Approves Tislelizumab for Patients with Microsatellite Instability-High or Mismatch Repair-Deficient Solid Tumors
Mar 11, 2022 7:00 AM
Tislelizumab is now approved in seven indications in China
CAMBRIDGE, Mass. & BEIJING & BASEL, Switzerland--(BUSINESS WIRE)-- BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global, science-driven biotechnology company focused on developing innovative and affordable medicines to improve treatment outcomes and access for patients worldwide, today announced that the China National Medical Products Administration (NMPA) has granted conditional approval to BeiGene’s anti-PD-1 antibody, tislelizumab, for the treatment of adult patients with advanced unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors, including:
- Patients with advanced colorectal cancer (CRC) who had been treated with fluoropyrimidine, oxaliplatin and irinotecan; and
- Other advanced solid tumors who develop disease progression after prior treatment and have no satisfactory alternative treatment options.
“Results from the clinical trial of tislelizumab in patients with MSI-H and dMMR solid tumors demonstrated that tislelizumab’s treatment effect was consistent and durable across tumor types and endpoints. We are proud of this approval in China as it underscores our ongoing commitment to pursuing the full potential of tislelizumab and expanding its access where there is unmet medical need,” commented Mark Lanasa, M.D., Ph.D., Senior Vice President, Chief Medical Officer, Solid Tumors, at BeiGene.
“With seven approved indications in China, our 3,100+ science-based commercial team is working to make tislelizumab more broadly available to those who may benefit from this important immunotherapy,” commented Xiaobin Wu, Ph.D., President, Chief Operating Officer, and General Manager of China, at BeiGene. “Today’s approval is a great step for patients in China with MSI-H and dMMR solid tumors.”
“In the pivotal Phase 2 trial, we observed consistent responses across tumor types with tislelizumab and it was generally well tolerated,” said Lin Shen, Ph.D., Vice President at the Beijing Cancer Hospital, and the principal investigator of the trial. “The NMPA’s approval of tislelizumab is welcoming news to patients with MSI-H and dMMR solid tumors, which are particularly prevalent among the many patients with cancers of the gastrointestinal tract. We are pleased to have a tissue-agnostic treatment approach with tislelizumab now available to those patients in need.”
This approval was supported by clinical results from a single-arm, multi-center, open-label, pivotal Phase 2 clinical trial (NCT03736889) to evaluate efficacy and safety of tislelizumab as monotherapy in patients with previously treated locally advanced unresectable or metastatic MSI-H or dMMR solid tumors, with an enrollment of 80 patients in China. Patients received tislelizumab 200 mg intravenously every three weeks until disease progression, unacceptable toxicity, or withdrawal. The primary endpoint of this trial is objective response rate (ORR) as assessed by independent review committee (IRC) per RECIST v1.1; secondary endpoints include time to response (TTR), duration of response (DoR), disease control rate (DCR), and progression-free survival (PFS) as assessed by investigator and IRC, overall survival (OS), and safety and tolerability. Results of this study were presented at the 2021 American Society of Clinical Oncology Annual Meeting.
About Microsatellite Instability-High or Mismatch Repair Deficient Solid Tumors
Microsatellite instability-high (MSI-H) cancer cells have a greater than normal number of genetic markers called microsatellites, which are short, repeated sequences of DNA. Cancer cells that have large numbers of microsatellites may have defects in the ability to correct mistakes (also known as mismatch repair deficiency, or dMMR) that occur when DNA is copied in the cell. MSI-H and dMMR tumors are found most often in colorectal cancer, other types of gastrointestinal cancer and endometrial cancer, although they may also be found in cancers of the breast, prostate, bladder and thyroid.i
About Tislelizumab
Tislelizumab (BGB-A317) is a humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to FcγR on macrophages. In pre-clinical studies, binding to FcγR on macrophages has been shown to compromise the anti-tumor activity of PD-1 antibodies through activation of antibody-dependent macrophage-mediated killing of T effector cells. Tislelizumab is the first drug from BeiGene’s immuno-oncology biologics program and is being developed internationally as a monotherapy and in combination with other therapies for the treatment of a broad array of both solid tumor and hematologic cancers.
The China National Medical Products Administration (NMPA) has approved tislelizumab in seven indications, including full approval for first-line treatment of patients with advanced squamous non-small cell lung cancer (NSCLC) in combination with chemotherapy, for first-line treatment of patients with advanced non-squamous NSCLC in combination with chemotherapy, and for second- or third-line treatment of patients with locally advanced or metastatic NSCLC who progressed on prior platinum-based chemotherapy. The NMPA has also granted conditional approval for the treatment of patients with classical Hodgkin’s lymphoma (cHL) who received at least two prior therapies, for the treatment of patients with locally advanced or metastatic urothelial carcinoma (UC) with PD-L1 high expression whose disease progressed during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy, for the treatment of patients with hepatocellular carcinoma (HCC) who have received at least one systemic therapy, and for the treatment of patients with advanced unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors. Full approval for these indications is contingent upon results from ongoing randomized, controlled confirmatory clinical trials or other confirmatory trials approved by the health authority.
In addition, two supplemental Biologics License Applications for tislelizumab are under review by the Center for Drug Evaluation (CDE) of the NMPA, including for the treatment of patients with locally advanced or metastatic esophageal squamous cell carcinoma (ESCC) who have disease progression following or are intolerant to first-line standard chemotherapy, and for first-line treatment of patients with recurrent or metastatic nasopharyngeal cancer (NPC).
In the U.S., a Biologics License Application for tislelizumab as a treatment for patients with unresectable recurrent locally advanced or metastatic ESCC after prior systemic therapy is currently under review by the U.S. Food and Drug Administration with a PDUFA target action date of July 12, 2022.
BeiGene has initiated or completed 17 potentially registration-enabling clinical trials in China and globally, including 13 Phase 3 trials and four pivotal Phase 2 trials.
In January 2021, BeiGene and Novartis entered into a collaboration and license agreement granting Novartis rights to develop, manufacture, and commercialize tislelizumab in North America, Europe, and Japan.
Tislelizumab is not approved for use outside of China.
About the Tislelizumab Clinical Program
Clinical trials of tislelizumab include:
- Phase 3 trial comparing tislelizumab with docetaxel in the second- or third-line setting in patients with NSCLC (NCT03358875);
- Phase 3 trial comparing tislelizumab to salvage chemotherapy in patients with relapsed or refractory classical Hodgkin Lymphoma (cHL; NCT04486391);
- Phase 3 trial in patients with locally advanced or metastatic urothelial carcinoma (NCT03967977);
- Phase 3 trial of tislelizumab in combination with chemotherapy versus chemotherapy as first-line treatment for patients with advanced squamous NSCLC (NCT03594747);
- Phase 3 trial of tislelizumab in combination with chemotherapy versus chemotherapy as first-line treatment for patients with advanced non-squamous NSCLC (NCT03663205);
- Phase 3 trial of tislelizumab in combination with platinum-based doublet chemotherapy as neoadjuvant treatment for patients with NSCLC (NCT04379635);
- Phase 3 trial of tislelizumab combined with platinum and etoposide versus placebo combined with platinum and etoposide in patients with extensive-stage small cell lung cancer (NCT04005716);
- Phase 3 trial comparing tislelizumab with sorafenib as first-line treatment for patients with hepatocellular carcinoma (HCC; NCT03412773);
- Phase 2 trial in patients with previously treated unresectable HCC (NCT03419897);
- Phase 2 trial in patients with locally advanced or metastatic urothelial bladder cancer (NCT04004221);
- Phase 3 trial comparing tislelizumab with chemotherapy as second-line treatment for patients with advanced esophageal squamous cell carcinoma (ESCC; NCT03430843);
- Phase 3 trial of tislelizumab in combination with chemotherapy as first-line treatment for patients with ESCC (NCT03783442);
- Phase 3 trial of tislelizumab versus placebo in combination with chemoradiotherapy in patients with localized ESCC (NCT03957590);
- Phase 3 trial of tislelizumab combined with chemotherapy versus placebo combined with chemotherapy as first-line treatment for patients with gastric cancer (NCT03777657);
- Phase 2 trial of tislelizumab in patients with relapsed or refractory cHL (NCT03209973);
- Phase 2 trial in patients with MSI-H/dMMR solid tumors (NCT03736889); and
- Phase 3 trial of tislelizumab combined with chemotherapy versus placebo combined with chemotherapy as first-line treatment in patients with nasopharyngeal cancer (NCT03924986).
To learn more about BeiGene, please visit www.beigene.com and follow us on Twitter at @BeiGeneGlobal.
i National Cancer Institute. Available at https://www.cancer.gov/publications/dictionaries/cancer-terms/def/mismatch-repair-deficiency. Accessed June 2021.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220310006055/en/
Source: BeiGene, Ltd.
BeiGene's tislelizumab gets approval in China for expanded use in solid tumors
Mar. 11, 2022 7:41 AM ET BeiGene, Ltd. (BGNE)
By: Ravikash, SA News Editor
- BeiGene (NASDAQ:BGNE) said the China National Medical Products Administration (NMPA) granted conditional approval to tislelizumab to treat adult patients with advanced unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors.
- https://seekingalpha.com/news/3812650-beigenes-tislelizumab
- https://seekingalpha.com/symbol/BGNE
- https://www.beigene.com/en-us/science-and-product-portfolio/pipeline/tislelizumab
- https://www.beigene.com/
biotecMAX™ Feb/MAR 2022 Insight
KEYTRUDA® (pembrolizumab)
Merck’s KEYTRUDA® (pembrolizumab) Demonstrated Significant Improvement in Distant Metastasis-Free Survival (DMFS) Compared to Placebo as Adjuvant Therapy for Patients With Resected Stage IIB and IIC Melanoma in Phase 3 KEYNOTE-716 Trial
March 7, 2022 6:45 am ET
New Results Build on Previously Reported Significant Recurrence-Free Survival (RFS) Benefit Seen in These Patients
KENILWORTH, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced that the Phase 3 KEYNOTE-716 trial investigating KEYTRUDA, Merck’s anti-PD-1 therapy, as adjuvant treatment for patients with resected stage IIB and IIC melanoma met its key secondary endpoint of distant metastasis-free survival (DMFS) at a pre-specified interim analysis. In the trial, adjuvant treatment with KEYTRUDA demonstrated a statistically significant improvement in the endpoint of DMFS compared to placebo in these patients. No new safety signals were observed.
At this interim analysis, treatment with KEYTRUDA also continued to show an improvement in recurrence-free survival (RFS) compared to placebo. As previously reported, KEYNOTE-716 met the primary endpoint of RFS at the first interim analysis (HR=0.65 [95% CI, 0.46-0.92]; p=0.00658), which supported U.S. Food and Drug Administration approval for this indication in December 2021. Full results from this analysis of KEYNOTE-716 will be presented at an upcoming medical meeting.
“Patients with melanoma that has spread to distant sites have a significantly worse prognosis and the goal of adjuvant therapy is to delay disease recurrence, especially distant metastases,” said Dr. Scot Ebbinghaus, vice president, clinical research, Merck Research Laboratories. “In KEYNOTE-716, adjuvant treatment with KEYTRUDA first showed a significant improvement in recurrence-free survival and has now demonstrated a significant improvement in the time until the first diagnosis of a distant metastasis compared to placebo. The distant metastasis-free survival data from KEYNOTE-716 reinforce the evidence for KEYTRUDA as adjuvant therapy in stage IIB and IIC melanoma.”
To date, KEYTRUDA has shown significant improvements in RFS and DMFS compared to placebo across resected stage IIB and stage IIC (KEYNOTE-716) and stage III melanoma (EORTC1325/KEYNOTE-054: RFS HR=0.57 [95% CI, 0.46, 0.70] p<0.001; DMFS HR=0.60 [95% CI, 0.49-0.73]; p<0.001).
In the U.S., KEYTRUDA is approved for the adjuvant treatment of adult and pediatric (12 years and older) patients with stage IIB, IIC or III melanoma following complete resection. Additional global regulatory submissions based on KEYNOTE-716 are ongoing.
About KEYNOTE-716
KEYNOTE-716 (ClinicalTrials.gov, NCT03553836) is a multicenter, randomized, double-blind, placebo-controlled Phase 3 trial that enrolled 976 patients (12 years and older) with resected stage IIB or IIC melanoma. The primary endpoint of the trial is RFS, and secondary endpoints include DMFS and overall survival (OS). Following complete surgical resection, patients were randomized to KEYTRUDA 200 mg or the pediatric (≥12 years old) dose of KEYTRUDA 2 mg/kg intravenously (up to a maximum of 200 mg) every three weeks or placebo for up to one year until disease recurrence or unacceptable toxicity.
About EORTC1325/KEYNOTE-054
EORTC1325/KEYNOTE-054 (ClinicalTrials.gov, NCT02362594) is a multicenter, randomized, double-blind, placebo-controlled Phase 3 trial sponsored by Merck and conducted in collaboration with the European Organisation for Research and Treatment of Cancer that enrolled 1,019 patients with completely resected stage IIIA (>1 mm lymph node metastasis), IIIB, or IIIC melanoma who were randomized 1:1 to receive KEYTRUDA 200 mg every three weeks or placebo for up to one year or until disease recurrence or unacceptable toxicity. The co-primary endpoints are RFS for all patients and RFS in patients whose tumors expressed PD-L1. Secondary endpoints include DMFS and OS in all patients and in patients whose tumors expressed PD-L1.
About KEYTRUDA® (pembrolizumab) Injection, 100 mg
KEYTRUDA is an anti-programmed death receptor-1 (PD-1) therapy that works by increasing the ability of the body’s immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.
Merck has the industry’s largest immuno-oncology clinical research program. There are currently more than 1,700 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.
Selected KEYTRUDA® (pembrolizumab) Indications in the U.S.
Melanoma
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.
KEYTRUDA is indicated for the adjuvant treatment of adult and pediatric (12 years and older) patients with stage IIB, IIC, or III melanoma following complete resection.
See additional selected KEYTRUDA indications in the U.S. after the Selected Important Safety Information
Additional Selected KEYTRUDA
Indications in the U.S.
Non-Small Cell Lung Cancer
KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) ≥1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is:
- stage III where patients are not candidates for surgical resection or definitive chemoradiation, or
- metastatic.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS ≥1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.
Head and Neck Squamous Cell Cancer
KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS) ≥1] as determined by an FDA-approved test.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.
Classical Hodgkin Lymphoma
KEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).
KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.
Primary Mediastinal Large B-Cell Lymphoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy.
KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.
Urothelial Carcinoma
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC):
- who are not eligible for any platinum-containing chemotherapy, or
- who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
Non-muscle Invasive Bladder Cancer
KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.
Microsatellite Instability-High or Mismatch Repair Deficient Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options.
This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.
Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).
Gastric Cancer
KEYTRUDA, in combination with trastuzumab, fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of patients with locally advanced unresectable or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma.
This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Esophageal Cancer
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic esophageal or gastroesophageal (GEJ) (tumors with epicenter 1 to 5 centimeters above the GEJ) carcinoma that is not amenable to surgical resection or definitive chemoradiation either:
- in combination with platinum- and fluoropyrimidine-based chemotherapy, or
- as a single agent after one or more prior lines of systemic therapy for patients with tumors of squamous cell histology that express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
Cervical Cancer
KEYTRUDA, in combination with chemotherapy, with or without bevacizumab, is indicated for the treatment of patients with persistent, recurrent, or metastatic cervical cancer whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test.
Hepatocellular Carcinoma
KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Merkel Cell Carcinoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Renal Cell Carcinoma
KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of adult patients with advanced renal cell carcinoma (RCC).
KEYTRUDA is indicated for the adjuvant treatment of patients with RCC at intermediate-high or high risk of recurrence following nephrectomy, or following nephrectomy and resection of metastatic lesions.
Tumor Mutational Burden-High Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [≥10 mutations/megabase] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.
Cutaneous Squamous Cell Carcinoma
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) or locally advanced cSCC that is not curable by surgery or radiation.
Triple-Negative Breast Cancer
KEYTRUDA is indicated for the treatment of patients with high-risk early-stage triple-negative breast cancer (TNBC) in combination with chemotherapy as neoadjuvant treatment, and then continued as a single agent as adjuvant treatment after surgery.
KEYTRUDA, in combination with chemotherapy, is indicated for the treatment of patients with locally recurrent unresectable or metastatic TNBC whose tumors express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
For more information about our oncology clinical trials, visit www.merck.com/clinicaltrials.
For more information, visit www.merck.com and connect with us on Twitter, Facebook, Instagram, YouTube and LinkedIn
Please see Prescribing Information for KEYTRUDA (pembrolizumab) at
http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_pi.pdf and
Medication Guide for KEYTRUDA at
http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_mg.pdf .
Source: Merck & Co., Inc.
Merck says late-stage melanoma trial for Keytruda met main secondary goal
Mar. 07, 2022 7:02 AM ET
By: Dulan Lokuwithana, SA News Editor
- Updating results from its KEYNOTE-716 trial, Merck (NYSE:MRK) said on Monday that its blockbuster anti-PD-1 therapy, Keytruda met the key secondary endpoint of the trial among patients with resected stage IIB and IIC melanoma.
- https://seekingalpha.com/news/3810089-merck-says-late-stage-melanoma-trial-for-keytruda-met-main-secondary-goal
- https://seekingalpha.com/symbol/MRK
- https://www.keytruda.com/
Trodelvy® (sacituzumab govitecan-hziy)
March 07, 2022
Phase 3 TROPiCS-02 Study Met the Primary Endpoint of Progression-Free Survival in Late-Line HR+/HER2- Metastatic Breast Cancer
– Study Will Continue to Follow Patients for Overall Survival, a Key Secondary Endpoint –
FOSTER CITY, Calif.--(BUSINESS WIRE)-- Gilead Sciences, Inc. (Nasdaq: GILD) today announced results from the Phase 3 TROPiCS-02 study evaluating Trodelvy® (sacituzumab govitecan-hziy) in patients with HR+/HER2- metastatic breast cancer who received prior endocrine therapy, CDK4/6 inhibitors and two to four lines of chemotherapy. The study met its primary endpoint with a statistically significant improvement in progression-free survival (PFS) versus physician’s choice of chemotherapy. The trial targeted a 30% reduction in the risk of disease progression or death. The primary endpoint results were consistent with those observed in the Phase 1/2 IMMU-132-01 study in a subset of HR+/HER2- metastatic breast cancer patients.1 The first interim analysis of the key secondary endpoint of overall survival in the TROPiCS-02 study demonstrated a trend in improvement for overall survival. Patients will be followed for a subsequent overall survival analysis. The safety profile for Trodelvy was consistent with prior studies, and no new safety concerns emerged in this patient population.
“Trodelvy demonstrated consistent activity in this difficult-to-treat patient population,” said Merdad Parsey, MD, PhD, Chief Medical Officer, Gilead Sciences. “We are evaluating the data and will explore potential pathways with regulatory authorities to bring Trodelvy to this group of patients. As we work to expand the patient benefit of Trodelvy beyond its current indications for second-line metastatic triple-negative breast cancer and accelerated approval in second-line metastatic bladder cancer, we are pursuing studies across multiple tumor types and earlier lines of therapy.”
“HR+/HER2- breast cancer accounts for approximately 70% of all breast cancer cases. Patients with advanced breast cancer may eventually develop endocrine resistance, then resistance to a limited set of sequential chemotherapy options,” said Hope Rugo, MD, Professor of Medicine and Director, Breast Oncology and Clinical Trials Education at the University of California San Francisco Comprehensive Cancer Center. “These data show the potential for Trodelvy to address an important unmet need for patients with HR+/HER2- metastatic breast cancer who have been heavily pretreated.”
Detailed results from TROPiCS-02 will be presented at an upcoming medical conference.
Gilead has filed a current report on Form 8-K that includes this press release and some FAQs related to the information in this release. The information in this press release should be read in conjunction with the FAQs.
Trodelvy has not been approved by any regulatory agency for the treatment of HR+/HER2- metastatic breast cancer. Its safety and efficacy have not been established for this indication. Trodelvy has a Boxed Warning for severe or life-threatening neutropenia and severe diarrhea; please see below for additional Important Safety Information.
About the TROPiCS-02 Study
The TROPiCS-02 study is a global, multicenter, open-label, Phase 3 study, randomized 1:1 to evaluate Trodelvy versus physician’s choice of chemotherapy (eribulin, capecitabine, gemcitabine, or vinorelbine) in 543 patients with HR+/HER2- metastatic breast cancer who were previously treated with endocrine therapy, CDK4/6 inhibitors and two to four lines of chemotherapy. The primary endpoint is progression-free survival per Response Evaluation Criteria in Solid Tumors (RECIST 1.1) as assessed by blinded independent central review for participants treated with Trodelvy compared to those treated with chemotherapy. The trial targets a 30% reduction in the risk of disease progression or death and is powered to detect a statistically significant difference of at least 0.9 months in median PFS. Secondary endpoints include overall survival, duration of response, clinical benefit rate and overall response rate as well as assessment of safety and tolerability and quality of life measures. More information about TROPiCS-02 is available at https://clinicaltrials.gov/ct2/show/NCT03901339.
About Trodelvy
Trodelvy® (sacituzumab govitecan-hziy) is a first-in-class Trop-2 directed antibody-drug conjugate. Trop-2 is a cell surface antigen highly expressed in multiple tumor types, including in more than 90% of breast and bladder cancers. Trodelvy is intentionally designed with a proprietary hydrolyzable linker attached to SN-38, a topoisomerase I inhibitor payload. This unique combination delivers potent activity to both Trop-2 expressing cells and the microenvironment.
Trodelvy is approved in more than 35 countries, with multiple additional regulatory reviews underway worldwide, for the treatment of adult patients with unresectable locally advanced or metastatic triple-negative breast cancer (TNBC) who have received two or more prior systemic therapies, at least one of them for metastatic disease. Trodelvy is also approved in the U.S. under the accelerated approval pathway for the treatment of adult patients with locally advanced or metastatic urothelial cancer (UC) who have previously received a platinum-containing chemotherapy and either programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor.
Trodelvy is also being developed for potential investigational use in other TNBC and metastatic UC populations, as well as a range of tumor types where Trop-2 is highly expressed, including hormone receptor-positive/human epidermal growth factor receptor 2-negative (HR+/HER2-) metastatic breast cancer, metastatic non-small cell lung cancer (NSCLC), metastatic small cell lung cancer (SCLC), head and neck cancer, and endometrial cancer.
U.S. Indication for Trodelvy
In the United States, Trodelvy is indicated for the treatment of:
- Adult patients with unresectable locally advanced or metastatic TNBC who have received two or more prior systemic therapies, at least one of them for metastatic disease.
- Adult patients with locally advanced or metastatic UC who have previously received a platinum-containing chemotherapy and either programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
Please see full Prescribing Information, including BOXED WARNING.
U.S. Prescribing Information for Trodelvy including BOXED WARNING, is available at www.gilead.com.
Trodelvy, Gilead and the Gilead logo are trademarks of Gilead Sciences, Inc., or its related companies.
For more information about Gilead, please visit the company’s website at www.gilead.com, follow Gilead on Twitter
View source version on businesswire.com: https://www.businesswire.com/news/home/20220306005060/en/
Source: Gilead Sciences, Inc.
Gilead's Trodelvy meets primary endpoint in HR+/HER2- metastatic breast cancer trial
Mar. 07, 2022 9:01 AM ETPFS, GILD
By: Jonathan Block, SA News Editor4 Comments
- Gilead Sciences' (NASDAQ:GILD) Trodelvy (sacituzumab) met its primary endpoint in a late-stage trial in patients with HR+/HER2- metastatic breast cancer.
- https://seekingalpha.com/news/3810179-gilead-trodelvy-meets-primary-endpoint-in-hr-her2-metastatic-breast-cancer-trial
- https://seekingalpha.com/symbol/GILD
- https://www.trodelvy.com/
LECANEMAB
EISAI INITIATES SUBMISSION OF APPLICATION DATA UNDER THE PRIOR ASSESSMENT CONSULTATION SYSTEM IN JAPAN WITH THE AIM OF OBTAINING EARLY APPROVAL FOR INVESTIGATIONAL ANTI-AMYLOID BETA PROTOFIBRIL ANTIBODY LECANEMAB
MARCH 4, 2022 • INVESTOR RELATIONS
TOKYO and CAMBRIDGE, Eisai Co., Ltd. (Headquarters: Tokyo, CEO: Haruo Naito, "Eisai") and Biogen Inc. (Nasdaq: BIIB, Corporate headquarters: Cambridge, Massachusetts, CEO: Michel Vounatsos, "Biogen") announced today that Eisai has initiated a submission to the Pharmaceuticals and Medical Devices Agency (PMDA) of application data under the prior assessment consultation system in Japan for the investigational anti-amyloid beta (Aβ) protofibril antibody lecanemab (BAN2401). The lecanemab Clarity AD Phase 3 clinical study for mild cognitive impairment (MCI) due to Alzheimer’s disease (AD) and mild AD (collectively known as early AD) is ongoing.
The PMDA’s process, known as “prior assessment consultation”, is conducted at the development stage before the new drug application submission, which is based on available quality, non-clinical and clinical data. By identifying and resolving any potential issues prior to submission, the aim is to shorten application review time.
Based on discussions with the Ministry of Health, Labour and Welfare (MHLW) and PMDA, Eisai and PMDA have agreed to utilize this system with the aim of shortening the review period from formal application to final determination, and has submitted the non-clinical lecanemab data. The additional data of the application package will be submitted hereafter. Eisai plans to obtain the primary endpoint data from Clarity AD study in the fall of 2022, and based on the results of the study, aims to file for the manufacturing and marketing approval in Japan during Eisai’s fiscal year 2022.
In September 2021, Eisai initiated a rolling submission to the U.S. Food and Drug Administration (FDA) of a Biologics License Application (BLA) for lecanemab, an investigational agent under the Accelerated Approval pathway for the treatment of early AD with confirmed amyloid pathology, and expects to complete this rolling submission in the beginning of Eisai’s fiscal year 2022. Based on the results of Clarity AD study as the confirmatory study, Eisai plans to submit for full approval of lecanemab to the U.S. FDA during fiscal year 2022. Eisai and Biogen are committed to providing innovative treatments to persons living with early AD, their families and healthcare professionals who are waiting for new treatment options, as early as possible.
- About the Collaboration between Eisai and BioArctic for Alzheimer’s Disease
Eisai and Biogen are collaborating on the joint development and commercialization of AD treatments. Eisai serves as the lead in the co-development of lecanemab. - About the Collaboration between Eisai and BioArctic for Alzheimer’s Disease
Since 2005, BioArctic has had a long-term collaboration with Eisai regarding the development and commercialization of drugs for the treatment of AD. The commercialization agreement on the lecanemab antibody was signed in December 2007, and the development and commercialization agreement on the antibody lecanemab back-up for AD, which was signed in May 2015. Eisai is responsible for the clinical development, application for market approval and commercialization of the products for AD. BioArctic has no development costs for lecanemab in AD.
For more information about Eisai Co., Ltd., please visit https://www.eisai.com.
The company routinely posts information that may be important to investors on its website at www.biogen.com. To learn more, please visit www.biogen.com and follow Biogen on social media – Twitter, LinkedIn, Facebook, YouTube.
Biogen, Eisai begin filing application data for Alzheimer's drug lecanemab in Japan
Mar. 04, 2022 6:18 AM ET
By: Ravikash, SA News Editor
Naeblys/iStock via Getty Images
Biogen (NASDAQ:BIIB) and Eisai (OTCPK:ESALY) began submission of application data in Japan aimed at getting an early approval of their Alzheimer's disease (AD) therapy lecanemab.
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https://www.alzforum.org/therapeutics/lecanemab
Opdivo® (nivolumab) with Chemotherapy
U.S. Food and Drug Administration Approves Opdivo® (nivolumab) with Chemotherapy as Neoadjuvant Treatment for Certain Adult Patients with Resectable Non-Small Cell Lung Cancer
03/04/2022CATEGORY:
Approval marks the first-and-only immunotherapy-based treatment for use before surgery for non-small cell lung cancer1
In the Phase 3 CheckMate -816 trial, Opdivo plus platinum-doublet chemotherapy significantly improved event-free survival and pathologic complete response compared to platinum-doublet chemotherapy alone1
Opdivo-based combinations now approved in both metastatic and earlier stages of non-small cell lung cancer
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) approved Opdivo®(nivolumab) 360 mg (injection for intravenous use) in combination with platinum-doublet chemotherapy every three weeks for three cycles for adult patients with resectable (tumors ≥4 cm or node positive) non-small cell lung cancer (NSCLC) in the neoadjuvant setting.1Opdivo plus chemotherapy is approved regardless of PD-L1 status.1 The approval is based on the CheckMate -816 trial, the first positive Phase 3 trial of an immunotherapy-based combination used before surgery for resectable NSCLC. The primary endpoints included event-free survival (EFS) and pathologic complete response (pCR), which were evaluated using independent blinded review, and an additional efficacy outcome measure was overall survival (OS).1 The study compared Opdivo plus platinum-doublet chemotherapy (n=179) to platinum-doublet chemotherapy alone (n=179).1
In the trial, when given before surgery, Opdivoplus chemotherapy showed a statistically significant improvement in EFS with a 37% reduction in the risk of progression, recurrence or death (Hazard Ratio [HR] 0.63; 95% Confidence Interval [CI]: 0.45 to 0.87; P=0.0052) compared to chemotherapy alone.1Opdivo plus chemotherapy showed a median EFS of 31.6 months (95% CI: 30.2 to Not Reached [NR]) compared to 20.8 months for patients treated with chemotherapy alone (95% CI: 14.0 to 26.7).1Additionally, 24% of patients treated with Opdivo plus chemotherapy achieved pCR (95% CI: 18.0 to 31.0), compared to 2.2% of patients treated with chemotherapy alone (95% CI: 0.6 to 5.6; estimated treatment difference 21.6; 95% CI: 15.1 to 28.2; P<0.0001).1 A prespecified interim analysis for OS resulted in a HR of 0.57 (95% CI: 0.38 to 0.87), which did not cross the boundary for statistical significance.1
“Given the rates of disease recurrence in patients with resectable NSCLC, additional treatment options are needed that can be given before surgery to help improve the chance of successful surgical treatment and support the goal of reducing the risk of cancer returning,” said Mark Awad, MD, PhD, CheckMate -816 study investigator and clinical director of the Lowe Center for Thoracic Oncology at Dana-Farber Cancer Institute.2,3 “The approval of nivolumab with platinum-doublet chemotherapy marks a turning point in how we treat resectable NSCLC and it enables us to use immunotherapy and chemotherapy as neoadjuvant treatment for patients before surgery. Today’s announcement reinforces the need to increase the rates of NSCLC screening and early detection, and for patients to discuss treatment options with their providers.”1
Opdivo is associated with the following Warnings & Precautions: severe and fatal immune-mediated adverse reactions including pneumonitis, colitis, hepatitis and hepatotoxicity, endocrinopathies, dermatologic adverse reactions, nephritis with renal dysfunction, other immune-mediated adverse reactions; infusion-related reactions; complications of allogeneic hematopoietic stem cell transplantation (HSCT); embryo-fetal toxicity; and increased mortality in patients with multiple myeloma when Opdivo is added to a thalidomide analogue and dexamethasone, which is not recommended outside of controlled clinical trials.1 Please see Important Safety Information below.
“At Bristol Myers Squibb, we are leading innovative science in the use of immunotherapy in earlier stages of cancer and are committed to bringing these options to patients,” said Adam Lenkowsky, senior vice president and general manager, U.S. Cardiovascular, Immunology and Oncology at Bristol Myers Squibb. “Today’s approval builds on that commitment and expands the role of Opdivo-based treatment in NSCLC, the most common form of lung cancer, so patients may benefit earlier in the course of their disease.”1,4
This application was approved under the FDA’s Real-Time Oncology Review (RTOR) pilot program, which aims to ensure that safe and effective treatments are available to patients as early as possible.5 The review was also conducted under the FDA’s Project Orbis initiative, which enabled concurrent review by the health authorities in Australia, Canada and the United Kingdom, where the application remains under review. The EFS data from the Phase 3 CheckMate -816 trial will be presented at the American Association for Cancer Research Annual Meeting 2022 in April.
About CheckMate -816
CheckMate -816 is a randomized, open label trial evaluating Opdivo plus platinum-doublet chemotherapy compared to chemotherapy alone as neoadjuvant treatment in adult patients with resectable non-small cell lung cancer, regardless of PD-L1 expression.1 The trial included patients with histologically confirmed Stage IB (≥4 cm), II or IIIA NSCLC (per the 7th edition American Joint Committee on Cancer/Union for International Cancer Control [AJCC/UICC] staging criteria), ECOG performance status 0 or 1, and measurable disease (per RECIST version 1.1).1 Patients with unresectable or metastatic NSCLC, known EGFR mutations or ALK translocations, Grade 2 or greater peripheral neuropathy, active autoimmune disease, or medical conditions requiring systemic immunosuppression were excluded from the study.1 For the primary analysis, 358 patients were randomized to receive either Opdivo 360 mg plus histology-based platinum doublet chemotherapy on the same day every three weeks for up to three cycles, or platinum doublet chemotherapy every three weeks for up to three cycles, followed by surgery.1
The primary endpoints of the trial were EFS determined by Blinded Independent Central Review (BICR) and pCR determined by Blinded Independent Pathology Review (BIPR).1 EFS is defined as the length of time from randomization to any of the following events: any progression of disease precluding surgery, progression, or recurrence of disease after surgery, or death due to any cause.1 In addition, pCR was defined as 0% residual viable tumor cells in both primary tumor and sampled lymph nodes as assessed by BIPR.1 Additional efficacy outcome measures included OS.1
INDICATIONS
OPDIVO® (nivolumab), in combination with platinum-doublet chemotherapy, is indicated as neoadjuvant treatment of adult patients with resectable (tumors ≥4 cm or node positive) non-small cell lung cancer (NSCLC).
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of adult patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express PD-L1 (≥1%) as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab) and 2 cycles of platinum-doublet chemotherapy, is indicated for the first-line treatment of adult patients with metastatic or recurrent non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab), as a single agent, is indicated for the adjuvant treatment of adult patients with urothelial carcinoma (UC) who are at high risk of recurrence after undergoing radical resection of UC.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of completely resected esophageal or gastroesophageal junction cancer with residual pathologic disease in adult patients who have received neoadjuvant chemoradiotherapy (CRT).
IMPORTANT SAFETY INFORMATION
Please see US Full Prescribing Information for OPDIVO and YERVOY.
For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram. Celgene and Juno Therapeutics are wholly owned subsidiaries of Bristol-Myers Squibb Company. In certain countries outside the U.S., due to local laws, Celgene and Juno Therapeutics are referred to as, Celgene, a Bristol Myers Squibb company and Juno Therapeutics, a Bristol Myers Squibb company.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220301006264/en/
Source: Bristol Myers Squibb
Bristol Myers Opdivo combo gets FDA nod for expanded use in lung cancer
Mar. 07, 2022 4:27 AM ET Bristol-Myers Squibb Company (BMY) By: Ravikash, SA News Editor1 Comment
- The U.S. Food and Drug Administration (FDA) approved Bristol Myers Squibb's (NYSE:BMY) Opdivo (nivolumab) in combination with platinum-doublet chemotherapy for adult patients with resectable non-small cell lung cancer (NSCLC) in the neoadjuvant setting (use before surgery).
- https://seekingalpha.com/news/3810049-bristol-myers-opdivo-combo-gets-fda-nod-for-expanded-use-in-lung-cancer
- https://seekingalpha.com/symbol/BMY
- https://www.bms.com/
- https://www.opdivo.com/
Olumiant (baricitinib)
HEALTHCARE & PHARMAMARCH 3, 202210:13 AMUPDATED A By Reuters Staff
Arthritis drug shown to reduce risk of COVID death in large UK trial
(Reuters) - Eli Lilly and Incyte’s arthritis drug baricitinib helped reduce the risk of death in hospitalised COVID-19 patients by 13% regardless of which other coronavirus treatment they were given, according to a large British study.
Over 8,000 patients were administered baricitinib in addition to usual care, at random, or usual care alone, as part of the so-called RECOVERY trial, scientists from the University of Oxford said on Thursday.
Results showed 546 patients in the usual care group died within 28 days but only 513 patients in the baricitinib group died where they were also given a corticosteroid like dexamethasone, tocilizumab or remdesivir.
“This result confirms and extends earlier findings, providing greater certainty that baricitinib is beneficial and new data to guide the treatment of COVID-19 patients with a combination of drugs to dampen the immune response,” said Peter Horby, Oxford professor and joint chief investigator.
The findings are consistent with the U.S. drugmakers’ own research from a smaller trial last August and comes after a World Health Organization panel had earlier this year recommended baricitinib for patients with severe COVID-19 in combination with corticosteroids.
Baricitinib belongs to a class of drugs called Janus Kinase (JAK) inhibitors which work by blocking actions of enzymes that play a role in the immune system processes and lead to inflammation, often seen in severe COVID-19 as lung damage.
U.S. authorities have approved the emergency use of baricitinib, sold under the brand name Olumiant, with or without taking Gilead’s antiviral remdesivir, while European regulators are reviewing the treatment for approval.
In the RECOVERY trial, baricitinib also increased the chances of patients being discharged alive within 28 days and reduced the risk of their condition worsening, scientists said.
Scientists behind the RECOVERY trial had shown dexamethasone saved the lives of COVID-19 patients, in what was called a “major breakthrough” in the pandemic, and also found tocilizumab worked against coronavirus.
Reporting by Pushkala Aripaka in Bengaluru; Editing by Krishna Chandra Eluri
Eli Lilly and Co (LLY.N) and partner Incyte Corp (INCY.O)
Lilly/Incyte's baricitinib reduces risk of death in COVID patients in UK study- Reuters
Mar. 04, 2022 4:48 AM ET
Eli Lilly and Company (LLY), INCY
By: Ravikash, SA News Editor
A U.K. study showed that Eli Lilly (NYSE:LLY) Incyte's (NASDAQ:INCY) Olumiant (baricitinib) helped reduce the risk of death in patients with COVID-19 patients who were hospitalized by 13% regardless of any other COVID therapy they were given, Reuters reported.
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LUMYKRAS® (sotorasib)
Hundreds of patients to benefit from revolutionary lung cancer drug on the NHS
3 March 2022
A revolutionary targeted drug for lung cancer will be made available to anyone who is eligible, thanks to a new drug deal, the head of the NHS announced today.
Sotorasib targets a genetic mutation, dubbed the ‘death star’, by medics and scientists, and has been proven during trials to prevent lung cancer from growing for seven months.
NHS patients in England were the first in Europe to benefit from the drug in September thanks to an early access agreement made with the manufacturer.
Around 100 patients have already received the treatment.
Announcing the new deal, Amanda Pritchard, NHS chief executive, said it meant even more patients could now benefit, with 600 people eligible for the ‘cutting edge’ drug every year.
Around one in eight lung cancer patients will have this lethal ‘death star’ mutation of the KRAS gene, so called because of its spherical appearance and impenetrable nature.
This first-of-its-kind treatment has taken more than four decades to develop and is the latest deal struck by the NHS as part of its Long Term Plan commitment to secure access to more innovative therapies.
The drug, which can be taken at home making it more convenient for patients, could also represent a major breakthrough in treatments for some of the world’s other deadliest cancers, including pancreatic and colorectal cancers.
It is also expected to offer a better and longer life than standard chemotherapy could, while also producing fewer side effects, boosting patients’ quality of life.
Amgen inks deal with UK's NHS for lung cancer drug Lumykras
Mar. 04, 2022 5:07 AM ET Amgen Inc. (AMGN)
By: Ravikash, SA News Editor
- Amgen (NASDAQ:AMGN) signed an agreement with U.K.'s National Health Service for its lung cancer drug Lumykras (sotorasib).
- https://seekingalpha.com/news/3809482-amgen-inks-deal-with-uks-nhs-for-lung-cancer-drug-lumykras
- https://seekingalpha.com/symbol/AMGN
- https://www.amgen.com/
IMBRUVICA® (ibrutinib)
February 28, 2022
AbbVie Seeks New Indication for IMBRUVICA® (ibrutinib) in Pediatric Patients with Chronic Graft Versus Host Disease (cGVHD)
- cGVHD is a life-threatening and rare condition that currently does not have an approved treatment for children under 12 and can occur in patients after receiving peripheral blood or bone marrow stem cell transplantation - If approved, IMBRUVICA would offer pediatric and adolescent patients the first BTKi treatment option with an oral suspension formulation to help manage their cGVHD after failure of one or more lines of systemic therapy - The sNDA and NDA submissions were primarily based on results of three years of data from the Phase 1/2 iMAGINE clinical trial - IMBRUVICA was the first treatment approved in the U.S. in 2017 for adult patients with cGVHD after failure of one or more lines of systemic therapyNORTH CHICAGO, Ill., Feb. 28, 2022 /PRNewswire/ -- AbbVie (NYSE: ABBV), today announced the submission of a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for IMBRUVICA® (ibrutinib) for the treatment of pediatric and adolescent patients one year and older with chronic graft versus host disease (cGVHD) after failure of one or more lines of systemic therapy. A New Drug Application (NDA) was also submitted for an oral suspension formulation of IMBRUVICA to provide an alternative administration option for pediatric patients. If approved, this represents AbbVie's first pediatric indication for IMBRUVICA.cGVHD is a life-threatening complication for about 14,000 patients each year after receiving a donor stem cell or bone marrow transplantation.1,2 Nearly half of these transplant patients develop cGVHD, and there are currently no FDA-approved treatment options for children under 12.3 The applications seek to update the IMBRUVICA U.S. Prescribing Information primarily based on the analysis of three years of data from the Phase 1/2 iMAGINE clinical trial, including use of a new oral suspension formulation of the treatment."We are committed to this work with IMBRUVICA in the hopes of providing the first FDA-approved BTKi treatment option for younger patients with cGVHD, including a new oral suspension formulation," said James Dean, M.D., Ph.D., IMBRUVICA Global Development Lead and Executive Medical Director at AbbVie. "For young children, the availability of a liquid oral suspension versus an oral capsule or tablet can be significant to enable them to take the recommended dose and address challenges swallowing capsules or tablets."The iMAGINE clinical trial enrolled 59 patients 1-19 years of age with relapsed/refractory (R/R) or new-onset moderate/severe cGVHD. The primary endpoints of the study were pharmacokinetics (PK) and safety; secondary endpoints included overall response rate. Results showed an overall response rate of 78 percent with IMBRUVICA and that PK data was consistent with adult dosing of IMBRUVICA. After 20 weeks, sustained response rates were observed in 70 percent and 58 percent of treatment-naive and R/R responders, respectively. Safety was consistent with the established profile for IMBRUVICA, with observed adverse events (AEs) consistent with those observed in adult patients with moderate to severe cGVHD. The most common Grade ≥3 AEs (≥ 5% of subjects) overall were pyrexia (8.5%), neutropenia (6.8%), stomatitis (6.8%), hypoxia (6.8%), osteonecrosis (6.8%), alanine aminotransferase increased (5.1%), hypokalemia (5.1%), and pneumothorax (5.1%)."It is important to empower patients and their families with evidence-based knowledge and I am encouraged by the results from the iMAGINE clinical trial of IMBRUVICA," said Dr. Paul A. Carpenter, attending physician at Seattle Children's Hospital and study principal investigator. "Results show that PK and safety were consistent with the known profile of IMBRUVICA and that of cGVHD. Efficacy results, including sustained response rates, were also encouraging."In 2017, IMBRUVICA was first approved as a single-agent therapy for adult patients with cGVHD who have experienced failure of prior systemic therapy, becoming the first FDA-approved treatment for adults with cGVHD. IMBRUVICA could be the first FDA-approved BTKi treatment option for pediatric and adolescent patients with cGVHD.
About cGVHDcGVHD occurs when donated peripheral blood or bone marrow stem cells view the recipient's body as foreign and the donated cells launch an immune attack on the body.4 cGVHD impacts major organs, with the skin, eyes, mouth and liver being most common.5 About 35 percent of the estimated 8,000 patients who undergo life-saving allogeneic Hematopoietic Stem Cell Transplant (HSCT) per year develop cGVHD that requires systemic treatment.4 Additionally, cGVHD is the most common cause of morbidity after allogeneic transplant.6 Steroids are the current standard treatment for pediatric cGVHD.7
About the iMAGINE StudyiMAGINE (PCYC-1146-IM) is an interventional single group treatment Phase 1/2 study, which enrolled 59 pediatric patients with chronic graft versus host disease (cGVHD). In the two-part study, Part A patients ages one to <12 years with moderate or severe cGVHD after failure of one or more lines of systemic therapy received IMBRUVICA oral suspension or capsule starting at 120mg/m2 once daily. To determine the Recommended Pediatric Equivalent Dose (RPED), dosage was escalated to 240mg/m2 after 14 days if no Grade 3 or higher related toxicity was observed. In Part B, patients 12 to <22 years of age with moderate or severe cGVHD who were newly diagnosed or had failed one or more lines of systemic therapy were given 420mg IMBRUVICA orally once daily. Once the RPED was determined in Part A, patients 1 to <12 years of age with new-onset or relapsed/refractory cGVHD could be enrolled in Part B. Primary endpoints included pharmacokinetics (PK) and safety, and secondary endpoints included overall response rate (ORR; CR/PR) per 2014 NIH criteria, overall survival, duration of response (DOR), and patient reported outcomes.
About IMBRUVICAIMBRUVICA® (ibrutinib) is a once-daily oral medication that is jointly developed and commercialized by Pharmacyclics LLC, an AbbVie Company and Janssen Biotech, Inc. IMBRUVICA® blocks the Bruton's tyrosine kinase (BTK) protein, which is needed by normal and abnormal B cells, to multiply and spread. By blocking BTK, IMBRUVICA may help move abnormal B cells out of their nourishing environments in the lymph nodes, bone marrow, and other organs.8,9,10IMBRUVICA® is approved in more than 100 countries and has been used to treat more than 250,000 patients worldwide. There are more than 50 company-sponsored clinical trials, including 18 ongoing or completed Phase 3 studies, over 11 years evaluating the efficacy and safety of IMBRUVICA®.IMBRUVICA® was first approved by the U.S. Food and Drug Administration (FDA) in November 2013, and today is indicated for adult patients in six disease areas, including five hematologic cancers. These include adults with CLL/small lymphocytic lymphoma (SLL) with or without 17p deletion (del17p) and adults with Waldenström's macroglobulinemia (WM), as well as adult patients with previously treated mantle cell lymphoma (MCL)*, adult patients with previously treated marginal zone lymphoma (MZL) who require systemic therapy and have received at least one prior anti-CD20-based therapy*, as well as adult patients with previously treated chronic graft-versus-host disease (cGVHD) after failure of one or more lines of systemic therapy.11*Accelerated approval was granted for MCL and MZL based on overall response rate. Continued approval for MCL and MZL may be contingent upon verification and description of clinical benefit in confirmatory trials.Since 2019, the National Comprehensive Cancer Network® (NCCN®), recommends ibrutinib (IMBRUVICA®) as a preferred regimen for first-line treatment of CLL/SLL, with Category 1 status for previously untreated patients without del17p. Additionally, IMBRUVICA® is a preferred treatment regimen for previously untreated patients with del17p. Since January 2020, the NCCN Guidelines recommend IMBRUVICA® as a category 2A preferred regimen for the treatment of relapsed/refractory MCL. Since September 2020, the NCCN Guidelines recommend IMBRUVICA® with or without rituximab as a Category 1 preferred regimen for both untreated and previously treated WM patients.12For more information, visit www.IMBRUVICA.com.
UsesWhat is IMBRUVICA® (ibrutinib)?IMBRUVICA® (ibrutinib) is a prescription medicine used to treat adults with:
- Mantle cell lymphoma (MCL) who have received at least one prior treatment
- Chronic lymphocytic leukemia (CLL)/Small lymphocytic lymphoma (SLL)
- Chronic lymphocytic leukemia (CLL)/Small lymphocytic lymphoma (SLL) with 17p deletion
- Waldenström's macroglobulinemia (WM)
- Marginal zone lymphoma (MZL) who require a medicine by mouth or injection (systemic therapy) and have received a certain type of prior treatment
- Chronic graft versus host disease (cGVHD) after failure of 1 or more lines of systemic therapy
It is not known if IMBRUVICA® is safe and effective in children.
Please see the full Important Product Information.About AbbVie in OncologyAt AbbVie, we are committed to transforming standards of care for multiple blood cancers while advancing a dynamic pipeline of investigational therapies across a range of cancer types. Our dedicated and experienced team joins forces with innovative partners to accelerate the delivery of potentially breakthrough medicines. We are evaluating more than 20 investigational medicines in over 300 clinical trials across some of the world's most widespread and debilitating cancers. As we work to have a remarkable impact on people's lives, we are committed to exploring solutions to help patients obtain access to our cancer medicines. For more information, please visit http://www.abbvie.com/oncology.
For more information about AbbVie, please visit us at www.abbvie.com. Follow @abbvie on Twitter, Facebook, Instagram, YouTube and LinkedIn.
AbbVie submits sNDA for Imbruvica in pediatric indication
Feb. 28, 2022 9:29 AM ET AbbVie Inc. (ABBV) By: Jonathan Block, SA News Editor2 Comments
- AbbVie (NYSE:ABBV) has submitted a supplemental New Drug Application (sNDA) for Imbruvica (ibrutinib) to the FDA as a second-line therapy for chronic graft versus host disease (cGVHD) for children one year and older.
- https://seekingalpha.com/news/3806885-abbvie-submits-snda-for-imbruvica-in-pediatric-indication
- https://seekingalpha.com/symbol/ABBV
- https://www.imbruvica.com/
- https://www.abbvie.com/
BRUKINSA® (zanubrutinib)
BeiGene Announces Health Canada Approval for BRUKINSA® (zanubrutinib) in Relapsed or Refractory Marginal Zone Lymphoma
Mar 03, 2022 7:00 AM
Follows U.S. approval for relapsed or refractory marginal zone lymphoma in 2021
Third approved indication for BRUKINSA in Canada, following mantle cell lymphoma (MCL) and Waldenström’s macroglobulinemia (WM)
MISSISSAUGA, Ontario & CAMBRIDGE, Mass. & BEIJING & BASEL, Switzerland--(BUSINESS WIRE)-- BeiGene (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global, science-driven biotechnology company focused on developing innovative and affordable medicines, today announced that BRUKINSA® (zanubrutinib) has been approved by Health Canada for the treatment of adult patients with marginal zone lymphoma (MZL) who have received at least one prior anti-CD20-based therapy.
“We are pleased that BRUKINSA is now approved in its third indication in Canada, R/R MZL, and this terrific milestone was made possible by the participating patients and investigators. This approval further supports our belief that BRUKINSA is a potentially best-in-class BTK inhibitor, with the MAGNOLIA trial results in R/R MZL providing additional evidence that the selective design of BRUKINSA can be translated into improved treatment outcomes,” said Jane Huang, M.D., Chief Medical Officer of Hematology at BeiGene. “We will continue to work with physicians and their patients as part of our broad global clinical program for BRUKINSA, which includes 35 trials and more than 3,900 subjects across 28 markets.”
“BRUKINSA is a highly selective next-generation BTK inhibitor designed to improve tolerability by minimizing off-target binding. In clinical trials, BRUKINSA achieved a high overall response rate among relapsed/refractory MZL patients and was generally well-tolerated. With this approval, Canadian patients with R/R MZL will have the option to receive BRUKINSA monotherapy as a treatment and a new hope for improved treatment outcomes,” said Dr. Anthea Peters, a hematologist at the Cross Cancer Institute in Edmonton, Alberta.
“The approval of this medicine for the treatment of R/R MZL represents a new treatment option and is welcome news to Canadians living with this rare type of lymphoma,” commented Antonella Rizza, Chief Executive Officer of Lymphoma Canada.
“We are delighted that BRUKINSA is now approved for patients with R/R MZL. Since our first approval 11 months ago, we have been expanding our organization in Canada and working to deliver on our commitment to create access to BRUKINSA for patients living with MCL, WM, and now MZL,” added Peter Brenders, General Manager of Canada at BeiGene.
The Health Canada marketing approval for BRUKINSA in R/R MZL is based on efficacy results from two open-label, multicenter, single arm clinical trials. In the MAGNOLIA trial (NCT03846427) (n=68), which included previously treated patients with MZL who had received at least one prior anti-CD20-based therapy, 38% of patients had extranodal MZL, 38% had nodal MZL, 18% had splenic MZL and 6% patients had unknown subtype. In BGB-3111-AU-003 (NCT02343120) (n=20), which included patients with previously treated MZL, 45% had extranodal MZL, 25% had nodal MZL and 30% had splenic MZL. BRUKINSA Total Daily Dose was 320 mg daily, given as 160 mg twice daily or 320 mg once daily.
As assessed by Independent Review Committee per 2014 Lugano Classification, BRUKINSA achieved an overall response rate (ORR) of 68% (95% CI; 56, 79) in the MAGNOLIA trial and 80% (95% CI; 56, 94) in BGB-311-AU-003. In the MAGNOLIA trial, the median response time was 2.8 months (range: 1.7 to 11.1 months).
Serious treatment-emergent adverse events were reported in 35 (40%) patients. The most common serious adverse events (≥ 2% of patients) were pyrexia (8%), pneumonia (7%), influenza (2%), anemia (2%), diarrhea (2%), atrial fibrillation and flutter (2%) and fall (2%).
Of the 88 patients with MZL treated with BRUKINSA, five (6%) patients discontinued treatment due to adverse event. The adverse events leading to treatment discontinuation included two cases of pneumonia (due to COVID-19 pneumonia), one case each of pyrexia, myocardial infarction, and diarrhea. Two (2%) patients had a dose reduction due to adverse events. Death due to adverse events within 30 days of last dose occurred in three (3%) patients. The adverse events leading to death were: COVID-19 pneumonia in two patients (2%) and myocardial infarction in one patient (1%).
The recommended dose of BRUKINSA is either 160 mg twice daily or 320 mg once daily, taken orally with or without food. The dose may be adjusted for adverse reactions and reduced for patients with severe hepatic impairment and certain drug interactions.
About BRUKINSA
BRUKINSA is a small molecule inhibitor of Bruton’s tyrosine kinase (BTK) discovered by BeiGene scientists that is currently being evaluated globally in a broad clinical program as a monotherapy and in combination with other therapies to treat various B-cell malignancies. Because new BTK is continuously synthesized, BRUKINSA was specifically designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity. With differentiated pharmacokinetics compared to other approved BTK inhibitors, BRUKINSA has been demonstrated to inhibit the proliferation of malignant B cells within a number of disease relevant tissues.
To date, BRUKINSA has received more than 20 approvals covering more than 40 countries and regions, including the United States, China, the EU and Great Britain, Canada, Australia, and additional international markets. Currently, more than 40 additional regulatory submissions are in review around the world.
To learn more about BeiGene, please visit www.beigene.ca and follow us on Twitter at @BeiGeneGlobal.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220303005298/en/
Source: BeiGene
BeiGene Brukinsa gets approval in Canada for blood cancer subtype
Mar. 03, 2022 7:38 AM ET
By: Ravikash, SA News Editor
- BeiGene (NASDAQ:BGNE) medicine Brukinsa was approved by Health Canada to treat adult patients with marginal zone lymphoma (MZL) who have received at least one prior anti-CD20-based therapy.
- https://seekingalpha.com/news/3809039-beigene-brukinsa-gets-approval-in-canada-for-blood-cancer-subtype
- https://seekingalpha.com/symbol/BGNE
- https://www.brukinsa.com/
- https://www.beigene.com/
Respiratory Syncytial Virus (RSV) Vaccine Candidate
Pfizer Granted FDA Breakthrough Therapy Designation for Respiratory Syncytial Virus (RSV) Vaccine Candidate for the Prevention of RSV in Infants from Birth up to Six Months of Age by Active Immunization of Pregnant Women
Wednesday, March 02, 2022 - 06:45am
NEW YORK--(BUSINESS WIRE)-- Pfizer Inc. (NYSE:PFE) today announced that its respiratory syncytial virus (RSV) vaccine candidate, PF-06482077 or RSVpreF, received Breakthrough Therapy Designation from the US Food and Drug Administration (FDA) for prevention of RSV-associated lower respiratory tract illness in infants from birth up to six months of age by active immunization of pregnant women.
The FDA decision is informed by the results of the Phase 2b proof-of-concept study of RSVpreF (NCT04032093), a global, double-blinded, placebo-controlled study that assessed the safety and immunogenicity of RSVpreF in healthy pregnant women ages 18 through 49 years old, who were vaccinated between 28- and 36-weeks gestation, and their infants. Pfizer will publish outcomes from this clinical trial at a future date.
“Today’s decision is a pivotal next step in our path towards potential regulatory approval for our maternal RSV vaccine candidate and is an important milestone in our efforts to help address the detrimental impact RSV disease has on infants,” said Kathrin U. Jansen, Ph.D., Senior Vice President and Head of Vaccine Research & Development at Pfizer Inc. “If approved by the FDA, this maternal immunization has the potential to be the first vaccine candidate to help protect infants in their vulnerable first months of life from disease caused by this highly-contagious virus. We look forward to our ongoing dialogue with the FDA to accelerate the development of our maternal RSV vaccine candidate.”
The FDA’s Breakthrough Therapy Designation is designed to expedite the development and review of drugs and vaccines that are intended to treat or prevent serious conditions and preliminary clinical evidence indicates that the drug or vaccine may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).1 This decision follows the FDA’s November 2018 decision to grant Fast Track status to RSVpreF. Fast Track status is a process designed to facilitate the development and expedite the review of new drugs and vaccines intended to treat or prevent serious conditions and address a medical need.2
About RSVpreF
Pfizer’s investigational RSV vaccine candidate builds on foundational basic science discoveries including those made at the National Institutes of Health (NIH), which detailed the crystal structure of a key form of the viral fusion protein (F) that RSV uses to attack human cells, prefusion F. The NIH research showed that antibodies specific to the prefusion form were highly effective at blocking virus infection, suggesting a prefusion F-based vaccine may confer optimal protection against RSV. After this important discovery, Pfizer tested numerous versions of the viral protein, and identified those that elicited a strong anti-viral immune response in pre-clinical evaluation. The vaccine candidate is composed of two preF proteins selected to optimize protection against RSV A and B and is currently being evaluated in ongoing late-stage human trials.
In April 2020, positive top-line results were achieved for a Phase 2b proof-of-concept study of RSVpreF, which evaluated the safety, tolerability and immunogenicity of RSVpreF in vaccinated pregnant women ages 18 through 49 and their infants. Pfizer will publish outcomes from this clinical trial at a future date.
In June 2020, Pfizer announced the initiation of a multicenter, international Phase 3 clinical trial (NCT04424316) evaluating the efficacy, immunogenicity and safety of RSVpreF when administered to pregnant women to help protect their babies from the disease after they are born. This study remains ongoing, but Pfizer expects the trial to readout in the first half of 2022 (1H22) and will seek to present and publish the results of MATISSE (MATernal Immunization Study for Safety and Efficacy) at a later date.
In September 2021, Pfizer announced the initiation of RENOIR (RSV vaccine Efficacy study iNOlder adults Immunized against RSV disease), a Phase 3 clinical trial (NCT05035212) evaluating the efficacy, immunogenicity and safety of a single dose of RSVpreF, in adults ages 60 years or older. Top-line data for the trial are expected in 1H22, and Pfizer will seek to present and publish the results at a later date. The initiation of this trial followed the company’s July 2021 update on a Phase 2a study evaluating the safety, immunogenicity and efficacy of RSVpreF in a virus challenge model in healthy adults 18 to 50 years of age. The results of the study supported proceeding to Phase 3.
In addition, to learn more, please visit us on www.Pfizer.com and follow us on Twitter at @Pfizer and @Pfizer News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220302005162/en/
Source: Pfizer Inc.
Pfizer's RSV vaccine for pregnant women gets FDA breakthrough therapy status
Mar. 02, 2022 7:15 AM ET Pfizer Inc. (PFE)GSK
By: Ravikash, SA News Editor
- The U.S. Food and Drug Administration (FDA) granted breakthrough therapy designation to Pfizer (NYSE:PFE) respiratory syncytial virus (RSV) vaccine candidate PF-06482077 or RSVpreF, for preventing RSV-linked lower respiratory tract illness in infants from birth up to six months of age by active immunization of pregnant women.
- https://seekingalpha.com/news/3808337-pfizers-rsv-vaccine-for-pregnant-women-gets-fda-breakthrough-therapy-status
- https://seekingalpha.com/symbol/PFE
Opdivo (nivolumab) plus Yervoy (ipilimumab)
Bristol Myers Squibb Receives Positive CHMP Opinion Recommending Approval for Opdivo (nivolumab) plus Yervoy (ipilimumab) for First-Line Treatment of Patients with Unresectable Advanced, Recurrent or Metastatic Esophageal Squamous Cell Carcinoma...
02/25/2022CATEGORY:
Recommendation based on results from the Phase 3 CheckMate -648 trial, in which the immunotherapy combination demonstrated significantly improved overall survival compared to fluoropyrimidine- and platinum-containing chemotherapy
Opdivo plus Yervoy is the first immunotherapy combination to show a superior survival benefit versus chemotherapy in this setting
PRINCETON, N.J.--(BUSINESS WIRE)--
Bristol Myers Squibb (NYSE:BMY):
Bristol Myers Squibb Receives Positive CHMP Opinion Recommending Approval for Opdivo (nivolumab) plus Yervoy (ipilimumab) for First-Line Treatment of Patients with Unresectable Advanced, Recurrent or Metastatic Esophageal Squamous Cell Carcinoma with Tumor Cell PD-L1 Expression ≥ 1%
Bristol Myers Squibb (NYSE: BMY) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended approval of Opdivo (nivolumab) plus Yervoy (ipilimumab) for the first-line treatment of adults with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) with tumor cell PD-L1 expression ≥ 1%. The European Commission (EC), which has the authority to approve medicines for the European Union (EU), will now review the CHMP opinion.
“Currently, the median overall survival for advanced ESCC patients is around ten months with standard chemotherapy,” said Ian M. Waxman, M.D., development lead, gastrointestinal cancers, Bristol Myers Squibb. “The CHMP’s positive recommendation for Opdivo plus Yervoy as a first-line treatment option for patients with unresectable advanced, recurrent or metastatic ESCC with tumor cell PD-L1 expression ≥1% marks an important step forward for patients in the EU awaiting new therapeutic options for their disease.”
The positive opinion is based on results from the Phase 3 CheckMate -648 trial, which showed that treatment with Opdivo plus Yervoy demonstrated a statistically significant and clinically meaningful overall survival (OS) benefit compared to fluoropyrimidine- and platinum-containing chemotherapy at the pre-specified interim analysis in patients with unresectable advanced, recurrent or metastatic ESCC with tumor cell PD-L1 expression ≥1% (median, 13.7 months vs 9.1 months, HR = 0.64; 98.6% CI: 0.46 to 0.90; p-value = 0.001). The safety profile of Opdivo plus Yervoy was consistent with previously reported studies. Results from CheckMate -648 were presented at the American Society of Clinical Oncology (ASCO) Annual Meeting in June 2021.
Bristol Myers Squibb thanks the patients and investigators involved in the CheckMate -648 trial.
About CheckMate -648
CheckMate -648 is a randomized Phase 3 study evaluating Opdivo plus Yervoy (N=325) or Opdivo plus fluorouracil and cisplatin (N=321) against fluorouracil plus cisplatin alone (N=324) in patients with previously untreated, unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma.
The primary endpoints of the trial are overall survival (OS) and progression-free survival (PFS) by blinded independent central review (BICR) in patients with tumor cell PD-L1 expression ≥1% for both Opdivo-based combinations versus chemotherapy. Secondary endpoints of the trial include OS and PFS by BICR in the all-randomized population.
In the Opdivo plus chemotherapy arm, patients received treatment with Opdivo 240 mg on Day 1 and Day 15, fluorouracil 800 mg/m²/day on Day 1 through Day 5 (for 5 days), and cisplatin 80 mg/m² on Day 1 of four-week cycle. Patients received Opdivo for up to 24 months or until disease progression, unacceptable toxicity or withdrawal of consent, and chemotherapy until disease progression, unacceptable toxicity or withdrawal of consent.
In the Opdivo plus Yervoy arm, patients received treatment with Opdivo 3 mg/kg every 2 weeks and Yervoy 1 mg/kg every 6 weeks up to 24 months or until disease progression, unacceptable toxicity or withdrawal of consent.
About Opdivo
Opdivo is a programmed death-1 (PD-1) immune checkpoint inhibitor that is designed to uniquely harness the body’s own immune system to help restore anti-tumor immune response. By harnessing the body’s own immune system to fight cancer, Opdivo has become an important treatment option across multiple cancers.
Opdivo’s leading global development program is based on Bristol Myers Squibb’s scientific expertise in the field of Immuno-Oncology and includes a broad range of clinical trials across all phases, including Phase 3, in a variety of tumor types. To date, the Opdivo clinical development program has treated more than 35,000 patients. The Opdivo trials have contributed to gaining a deeper understanding of the potential role of biomarkers in patient care, particularly regarding how patients may benefit from Opdivo across the continuum of PD-L1 expression.
In July 2014, Opdivo was the first PD-1 immune checkpoint inhibitor to receive regulatory approval anywhere in the world. Opdivo is currently approved in more than 65 countries, including the United States, the European Union, Japan and China. In October 2015, the Company’s Opdivo and Yervoy combination regimen was the first Immuno-Oncology combination to receive regulatory approval for the treatment of metastatic melanoma and is currently approved in more than 50 countries, including the United States and the European Union.
INDICATIONS
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express PD-L1 (≥1%) as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab) and 2 cycles of platinum-doublet chemotherapy, is indicated for the first-line treatment of adult patients with metastatic or recurrent non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab) is indicated for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving OPDIVO.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with unresectable malignant pleural mesothelioma (MPM).
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of patients with intermediate or poor risk advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab), in combination with cabozantinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab) is indicated for the treatment of patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy.
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with classical Hodgkin lymphoma (cHL) that has relapsed or progressed after autologous hematopoietic stem cell transplantation (HSCT) and brentuximab vedotin or after 3 or more lines of systemic therapy that includes autologous HSCT. This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab) is indicated for the treatment of patients with recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN) with disease progression on or after platinum-based therapy.
OPDIVO® (nivolumab) is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
OPDIVO® (nivolumab), as a single agent, is indicated for the adjuvant treatment of patients with urothelial carcinoma (UC) who are at high risk of recurrence after undergoing radical resection of UC.
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of adult and pediatric (12 years and older) patients with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response.
Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
OPDIVO® (nivolumab) is indicated for the treatment of patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) after prior fluoropyrimidine- and platinum-based chemotherapy.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of completely resected esophageal or gastroesophageal junction cancer with residual pathologic disease in patients who have received neoadjuvant chemoradiotherapy (CRT).
OPDIVO® (nivolumab), in combination with fluoropyrimidine- and platinum- containing chemotherapy, is indicated for the treatment of patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma.
For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220224006058/en/
Bristol Myers Squibb
Source: Bristol Myers Squibb
Bristol Myers Opdivo gets EMA panel nod for use as adjuvant therapy in urothelial cancer
Feb. 25, 2022 7:44 AM ET Bristol-Myers Squibb Company (BMY) By: Ravikash, SA News Editor
- Bristol Myers Squibb (NYSE:BMY) said a committee of the European Medicines Agency (EMA) recommended approval of Opdivo (nivolumab) for the adjuvant treatment of adults with muscle-invasive urothelial carcinoma with tumor cell PD-L1 expression ≥1% who are at a high risk of recurrence after undergoing surgery.
- https://seekingalpha.com/news/3805853-bristol-myers-opdivo-gets-ema-panel-nod-for-use-as-adjuvant-therapy-in-urothelial-cancer
- https://seekingalpha.com/symbol/BMY
- https://www.opdivo.com/about-opdivo/how-the-combination-works-combinationtherapy#:~:text=(advanced%20melanoma).-,OPDIVO%C2%AE%20(nivolumab)%20is%20a%20prescription%20medicine%20used%20in%20combination,than%2018%20years%20of%20age.
02/25/2022
02/25/2022
02/25/2022
CARVYKTI™ (ciltacabtagene autoleucel)
U.S. FDA Approves CARVYKTI™ (ciltacabtagene autoleucel), Janssen’s First Cell Therapy, a BCMA-Directed CAR-T Immunotherapy for the Treatment of Patients with Relapsed or Refractory Multiple MyelomaIn the pivotal clinical study, 98 percent of patients with relapsed or refractory multiple myeloma responded to a one-time treatment with ciltacabtagene autoleucel and 78 percent of patients who responded experienced a stringent complete response
HORSHAM, Pa., February 28, 2022 – The Janssen Pharmaceutical Companies of Johnson & Johnson announced today the U.S. Food and Drug Administration (FDA) has approved CARVYKTI™ (ciltacabtagene autoleucel; cilta-cel) for the treatment of adults with relapsed or refractory multiple myeloma (RRMM) after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.1 The approval is based on data from the pivotal CARTITUDE-1 study, which included patients who had received a median of six prior treatment regimens (range, 3-18), and had previously received a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 monoclonal antibody.1 In December 2017, Janssen entered into an exclusive worldwide license and collaboration agreement with Legend Biotech USA, Inc. to develop and commercialize ciltacabtagene autoleucel.
CARVYKTI™ is a chimeric antigen receptor T-cell (CAR-T) therapy featuring two B-cell maturation antigen (BCMA)-targeting single domain antibodies.1 In the pivotal CARTITUDE-1 study, one-time treatment with ciltacabtagene autoleucel resulted in deep and durable responses, with 98 percent (95 percent Confidence Interval [CI], 92.7-99.7) of patients with RRMM responding to therapy (98 percent overall response rate [ORR] (n=97).1 Notably, 78 percent (95 percent CI, 68.8-86.1) of the patients achieving this level of response (n=76) experienced a stringent complete response (sCR), a measure in which a physician is unable to observe any signs or symptoms of disease via imaging or other tests after treatment.1 At a median of 18 months follow-up, median duration of response (DOR) was 21.8 months.1
CARVYKTI™ is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the CARVYKTI™ REMS Program.1 The Safety Information for CARVYKTI™ includes a Boxed Warning regarding Cytokine Release Syndrome (CRS), Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS), Parkinsonism and Guillain-Barré syndrome, hemophagocytic lymphohistiocytosis/macrophage activation syndrome (HLH/MAS), and prolonged and/or recurrent cytopenias.1 Warnings and Precautions include prolonged and recurrent cytopenias, infections, hypogammaglobulinemia, hypersensitivity reactions, secondary malignancies, and effects on ability to drive and use machines.1 The most common adverse reactions (≥20 percent) are pyrexia, CRS, hypogammaglobulinemia, hypotension, musculoskeletal pain, fatigue, infections-pathogens unspecified, cough, chills, diarrhea, nausea, encephalopathy, decreased appetite, upper respiratory tract infection, headache, tachycardia, dizziness, dyspnea, edema, viral infections, coagulopathy, constipation, and vomiting.1
“We are committed to harnessing our science, deep disease understanding and capabilities to bring forward cell therapies like CARVYKTI as we continue to focus on our ultimate goal of delivering a cure for multiple myeloma,” said Peter Lebowitz, M.D., Ph.D., Global Therapeutic Area Head, Oncology, Janssen Research & Development, LLC. “We extend our sincere gratitude to the patients, their families and the teams of researchers and study centers who have participated in the clinical study of CARVYKTI and enabled today’s approval.”
About CARVYKTI™ (ciltacabtagene autoleucel)
CARVYKTI™ is a BCMA-directed, genetically modified autologous T-cell immunotherapy, which involves reprogramming a patient’s own T-cells with a transgene encoding a chimeric antigen receptor (CAR) that identifies and eliminates cells that express the B-cell maturation antigen (BCMA). BCMA is primarily expressed on the surface of malignant multiple myeloma B-lineage cells, as well as late-stage B-cells and plasma cells. The CARVYKTI™ CAR protein features two BCMA-targeting single domain antibodies designed to confer high avidity against human BCMA. Upon binding to BCMA-expressing cells, the CAR promotes T-cell activation, expansion, and elimination of target cells.1
In December 2017, Janssen Biotech, Inc. entered into an exclusive worldwide license and collaboration agreement with Legend Biotech USA, Inc. to develop and commercialize ciltacabtagene autoleucel.
In April 2021, Janssen announced the submission of a Marketing Authorisation Application to the European Medicines Agency seeking approval of CARVYKTI™ for the treatment of patients with relapsed and/or refractory multiple myeloma. In addition to a U.S. Breakthrough Therapy Designation granted in December 2019, ciltacabtagene autoleucel received a Breakthrough Therapy Designation in China in August 2020. Janssen also received an Orphan Drug Designation for CARVYKTI™ from the U.S. FDA in February 2019, and from the European Commission in February 2020.
About the CARTITUDE-1 Study
CARTITUDE-1 (NCT03548207) is an ongoing Phase 1b/2, open-label, multi-center study evaluating ciltacabtagene autoleucel for the treatment of patients with relapsed or refractory multiple myeloma, who previously received a proteasome inhibitor (PI), an immunomodulatory agent (IMiD) and an anti-CD38 monoclonal antibody, and who had disease progression on or after the last regimen. All patients in the study had received a median of six prior treatment regimens (range, 3-18). Of the 97 patients enrolled in the trial, 99 percent were refractory to the last line of treatment and 88 percent were triple-class refractory, meaning their cancer did not respond, or no longer responds, to an IMiD, a PI and an anti-CD38 monoclonal antibody.1
INDICATIONS AND USAGE
CARVYKTI™ (ciltacabtagene autoleucel) is a B-cell maturation antigen (BCMA)- directed genetically modified autologous T cell immunotherapy indicated for the treatment of adult patients with relapsed or refractory multiple myeloma, after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.
Further information is available at www.CARVYKTIrems.com or 1-844-672-0067.
Please read full Prescribing Information including Boxed Warning for CARVYKTI™.
Learn more at www.janssen.com. Follow us at www.twitter.com/JanssenUS and www.twitter.com/JanssenGlobal. Janssen Research & Development, LLC and Janssen Biotech, Inc. are part of the Janssen Pharmaceutical Companies of Johnson & Johnson.
J&J, Legend Biotech's Carvykti for multiple myeloma gets US FDA approval
Mar. 01, 2022 5:25 AM ET
Legend Biotech Corporation (LEGN), JNJ
By: Ravikash, SA News Editor1 Comment
The U.S. Food and Drug Administration (FDA) approved Johnson & Johnson (NYSE:JNJ) and Legend Biotech's (NASDAQ:LEGN) CAR-T therapy Carvykti to treat adults with relapsed or refractory multiple myeloma (RRMM) after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.
The approval of Carvykti (ciltacabtagene autoleucel; cilta-cel) is based on positive data from the CARTITUDE-1 study.
https://seekingalpha.com/symbol/LEGN
https://seekingalpha.com/symbol/JNJ
https://legendbiotech.com/pipeline#pipeline
Nurtec ODT (rimegepant)
BIOHAVEN AND PFIZER RECEIVE POSITIVE CHMP OPINION FOR MIGRAINE TREATMENT
02/25/2022
NEW HAVEN, Conn. and NEW YORK, Feb. 25, 2022 /PRNewswire/ -- Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) and Pfizer Inc. (NYSE: PFE) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion for rimegepant, a calcitonin gene-related peptide (CGRP) receptor antagonist, recommending the 75 mg dose of rimegepant (available as an orally dissolving tablet) for marketing authorization for both the acute treatment of migraine with or without aura in adults and the preventive treatment of episodic migraine in adults who have at least four migraine attacks per month.
The CHMP's positive opinion will now be reviewed by the European Commission (EC). The decision on whether to approve rimegepant, whose European Union (EU) trade name will be VYDURA™, will be made by the EC and would be valid in all 27 EU member states as well as in Iceland, Lichtenstein, and Norway. If approved, rimegepant will be the first oral CGRP receptor antagonist in the EU, and the only migraine medication approved for both acute and preventive treatment.
"This expression of confidence in rimegepant brings us closer to our goal of helping patients suffering from this debilitating neurological disease find appropriate treatment," said Nick Lagunowich, Global President, Pfizer Internal Medicine. "Pfizer is proud to have a strong footprint in Europe, which will help bring this important potential new treatment option to millions of adults in Europe living with migraine."
The CHMP positive opinion was based on the review of the results from three Phase 3 studies and a long-term, open-label safety study in acute treatment of migraine, and a Phase 3 study with a 1-year open-label extension in the preventive treatment of migraine. In these studies, rimegepant was safe and well-tolerated with rates of adverse events similar to that of placebo.
"The recommendation for rimegepant marks an important milestone for the migraine community," said Vlad Coric, M.D., Chief Executive Officer and Chairman of the Board of Biohaven. "Together with Pfizer, we are dedicated to helping patients and hope to provide rimegepant to patients in Europe soon, and eventually those worldwide, who are living with this debilitating disease, many of whom do not have satisfactory treatment options today."
Earlier this year, Pfizer and Biohaven entered into an agreement for the commercialization of rimegepant. Under the terms of the agreement, Pfizer has commercialization rights to rimegepant in markets outside of the U.S. Biohaven continues to lead research and development globally and retains the U.S. market.
About Rimegepant
Rimegepant targets a key component of migraine by reversibly blocking CGRP receptors, thereby inhibiting the biologic cascade that results in a migraine attack. A single, quick-dissolving tablet of 75 mg rimegepant provides fast pain relief, significant pain reduction and return to normal function, and has a lasting effect of up to 48 hours in many patients. Rimegepant is taken orally as needed, up to 18 doses/month to stop migraine attacks or taken every other day to help prevent migraine attacks and reduce the number of monthly migraine days. Rimegepant does not have addiction potential and was not associated with medication overuse headache or rebound headache in clinical trials.
Rimegepant is commercialized as Nurtec® ODT in the U.S. approved for the acute and preventive treatment of migraine in adults, and ex-U.S. is approved for the acute treatment of migraine in Kuwait and the United Arab Emirates, and for the acute and preventive treatment of migraine in Israel.
In addition, to learn more, please visit us on www.Pfizer.com and follow us on Twitter at @Pfizer and @Pfizer News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.
More information about Biohaven is available at www.biohavenpharma.com.
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SOURCE Biohaven Pharmaceutical Holding Company Ltd.
Pfizer, Biohaven's rimegepant gets EMA panel nod for migraine treatment
Feb. 25, 2022 8:40 AM ET
Biohaven Pharmaceutical Holding Company Ltd. (BHVN), PFE
By: Ravikash, SA News Editor1 Comment
Biohaven Pharmaceutical (NYSE:BHVN) and Pfizer (NYSE:PFE) said a committee of the European Medicines Agency (EMA) recommended the approval of 75 mg dose of rimegepant for the acute treatment of migraine with or without aura in adults and the preventive treatment of episodic migraine in adults who have at least four migraine attacks per month.
- https://seekingalpha.com/news/3805895-pfizer-biohaven-rimegepant-gets-ema-panel-nod-for-for-migraine-treatment
- https://seekingalpha.com/symbol/BHVN
- https://seekingalpha.com/symbol/PFE
- https://www.biohavenpharma.com/
biotecMAX™ Feb 2022 Insight
KEYTRUDA® (pembrolizumab) Plus LENVIMA® (lenvatinib)
KEYTRUDA® (pembrolizumab) Plus LENVIMA® (lenvatinib) Approved in Japan for Radically Unresectable or Metastatic Renal Cell Carcinoma
February 25, 2022 6:45 am ETResults From CLEAR/KEYNOTE-581 Showed KEYTRUDA Plus LENVIMA Significantly Reduced the Risk of Disease Progression or Death by 61%, With a Median Progression-Free Survival of Nearly Two Years Versus Nine Months for SunitinibKEYTRUDA Plus LENVIMA Now Approved in Japan for Two Types of CancerKENILWORTH, N.J. & TOKYO--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, and Eisai today announced that the Japanese Ministry of Health, Labour and Welfare (MHLW) has approved the combination of KEYTRUDA, Merck’s anti-PD-1 therapy, plus LENVIMA, the orally available multiple receptor tyrosine kinase inhibitor discovered by Eisai, for radically unresectable or metastatic renal cell carcinoma (RCC). KEYTRUDA plus LENVIMA is also approved in the U.S. and Europe for the first-line treatment of adult patients with advanced RCC. This marks the second approval of this combination in Japan; in December 2021, KEYTRUDA plus LENVIMA was approved for unresectable, advanced or recurrent endometrial carcinoma that progressed after chemotherapy.The approval is based on results from the pivotal Phase 3 CLEAR (Study 307)/KEYNOTE-581 trial, in which KEYTRUDA plus LENVIMA demonstrated statistically significant improvements versus sunitinib in the primary efficacy outcome of progression-free survival (PFS). Results showed KEYTRUDA plus LENVIMA (n=355) reduced the risk of disease progression or death by 61% (HR=0.39 [95% CI, 0.32-0.49]; p<0.0001), with a median PFS of 23.9 months versus 9.2 months for sunitinib (n=357).“Nearly one in three cases of renal cell carcinoma are diagnosed at an advanced stage, and patients are in need of new treatment options that may improve survival outcomes,” said Dr. Gregory Lubiniecki, Vice President, Oncology Clinical Research, Merck Research Laboratories. “In the CLEAR/KEYNOTE-581 trial, KEYTRUDA plus LENVIMA reduced the risk of disease progression or death by 61% versus sunitinib, a current standard of care. We are encouraged that patients with certain types of advanced renal cell carcinoma may have the opportunity to benefit from this combination.”“Today’s milestone for KEYTRUDA plus LENVIMA as a treatment for radically unresectable or metastatic renal cell carcinoma is particularly exciting as it marks the second approval for the combination in Japan,” said Terushige Iike, President of Eisai Japan, Senior Vice President, Eisai. “We are thrilled to be able to provide Japanese patients with a new treatment option, illustrating our shared commitment with Merck to develop therapies with the aim of addressing the unmet needs of those living with difficult-to-treat cancers. We would like to thank the patients, families and healthcare providers who made this approval possible.”The Japanese package inserts for KEYTRUDA and LENVIMA note that in the CLEAR/KEYNOTE-581 trial, adverse reactions were observed in 341 (96.9%) of 352 patients (including 42 of 42 Japanese patients) in the safety analysis set. The most common adverse reactions observed in 20% or more of patients included diarrhea in 192 patients (54.5%), hypertension in 184 patients (52.3%), hypothyroidism in 150 patients (42.6%), decreased appetite in 123 patients (34.9%), fatigue in 113 patients (32.1%), stomatitis in 113 patients (32.1%), palmar-plantar erythrodysesthesia syndrome in 99 patients (28.1%), proteinuria in 97 patients (27.6%), nausea in 94 patients (26.7%), dysphonia in 87 patients (24.7%), rash in 77 patients (21.9%), and asthenia in 71 patients (20.2%).Renal cell carcinoma is the most common type of kidney cancer worldwide; about nine out of 10 kidney cancer diagnoses are RCC. In Japan, there were more than 25,000 new cases of kidney cancer diagnosed and more than 8,000 deaths from the disease in 2020. In the U.S., approximately 30% of patients with RCC will have metastatic disease at diagnosis. Survival is highly dependent on the stage at diagnosis, and with a five-year survival rate in the U.S. of 14% for patients diagnosed with metastatic disease, the prognosis for these patients is poor.Merck and Eisai continue to study the KEYTRUDA plus LENVIMA combination across several types of cancer with more than 20 clinical trials.
About CLEAR/KEYNOTE-581 TrialThe approval is based on data from CLEAR (Study 307)/KEYNOTE-581 (ClinicalTrials.gov, NCT02811861), a Phase 3, multicenter, open-label, randomized trial conducted in 1,069 patients with advanced RCC in the first-line setting. The primary efficacy outcome was PFS as assessed by blinded independent central review (BICR) according to RECIST v1.1. Key secondary efficacy outcome measures were overall survival and objective response rate.Patients were randomized 1:1:1 to receive KEYTRUDA (200 mg intravenously every three weeks for up to 24 months) plus LENVIMA (20 mg orally once daily), or LENVIMA (18 mg orally once daily) plus everolimus (5 mg orally once daily), or sunitinib (50 mg orally once daily for four weeks on treatment, followed by two weeks off treatment). Treatment continued until unacceptable toxicity or disease progression as determined by investigator and confirmed by BICR using RECIST v1.1. If disease progression was observed by imaging evaluation, clinically stable patients with no symptoms indicating disease progression were allowed to continue on study treatment until disease progression was observed in a subsequent imaging evaluation.
About KEYTRUDA® (pembrolizumab) Injection, 100 mg
KEYTRUDA is an anti-programmed death receptor-1 (PD-1) therapy that works by increasing the ability of the body’s immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.Merck has the industry’s largest immuno-oncology clinical research program. There are currently more than 1,700 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.
Selected KEYTRUDA® (pembrolizumab) Indications in the U.S.MelanomaKEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.KEYTRUDA is indicated for the adjuvant treatment of adult and pediatric (12 years and older) patients with stage IIB, IIC, or III melanoma following complete resection.Non-Small Cell Lung CancerKEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) ≥1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is:
- stage III where patients are not candidates for surgical resection or definitive chemoradiation, or
- metastatic.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS ≥1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.Head and Neck Squamous Cell CancerKEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS ≥1)] as determined by an FDA-approved test.KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.Classical Hodgkin LymphomaKEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.Primary Mediastinal Large B-Cell LymphomaKEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.Urothelial CarcinomaKEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC):
- who are not eligible for any platinum-containing chemotherapy, or
- who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
Non-muscle Invasive Bladder CancerKEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.Microsatellite Instability-High or Mismatch Repair Deficient CancerKEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options.This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.Microsatellite Instability-High or Mismatch Repair Deficient Colorectal CancerKEYTRUDA is indicated for the treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).Gastric CancerKEYTRUDA, in combination with trastuzumab, fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of patients with locally advanced unresectable or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma.This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.Esophageal CancerKEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic esophageal or GEJ (tumors with epicenter 1 to 5 centimeters above the GEJ) carcinoma that is not amenable to surgical resection or definitive chemoradiation either:
- in combination with platinum- and fluoropyrimidine-based chemotherapy, or
- as a single agent after one or more prior lines of systemic therapy for patients with tumors of squamous cell histology that express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
Cervical CancerKEYTRUDA, in combination with chemotherapy, with or without bevacizumab, is indicated for the treatment of patients with persistent, recurrent, or metastatic cervical cancer whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test.KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test.Hepatocellular CarcinomaKEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.Merkel Cell CarcinomaKEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.Renal Cell CarcinomaKEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of adult patients with advanced renal cell carcinoma (RCC).KEYTRUDA, in combination with LENVIMA, is indicated for the first-line treatment of adult patients with advanced RCC.KEYTRUDA is indicated for the adjuvant treatment of patients with renal cell carcinoma (RCC) at intermediate-high or high risk of recurrence following nephrectomy, or following nephrectomy and resection of metastatic lesions.Endometrial CarcinomaKEYTRUDA, in combination with LENVIMA, is indicated for the treatment of patients with advanced endometrial carcinoma that is not MSI-H or dMMR, who have disease progression following prior systemic therapy in any setting and are not candidates for curative surgery or radiation.Tumor Mutational Burden-High CancerKEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [≥10 mutations/megabase] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.Cutaneous Squamous Cell CarcinomaKEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) or locally advanced cSCC that is not curable by surgery or radiation.Triple-Negative Breast CancerKEYTRUDA is indicated for the treatment of patients with high-risk early-stage triple-negative breast cancer (TNBC) in combination with chemotherapy as neoadjuvant treatment, and then continued as a single agent as adjuvant treatment after surgery.KEYTRUDA, in combination with chemotherapy, is indicated for the treatment of patients with locally recurrent unresectable or metastatic TNBC whose tumors express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
Please see Prescribing Information for KEYTRUDA (pembrolizumab) at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_pi.pdf and Medication Guide for KEYTRUDA at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_mg.pdf.About LENVIMA® (lenvatinib); available as 10 mg and 4 mg capsulesLENVIMA, discovered and developed by Eisai, is a multiple receptor tyrosine kinase inhibitor that inhibits the kinase activities of vascular endothelial growth factor (VEGF) receptors VEGFR1 (FLT1), VEGFR2 (KDR), and VEGFR3 (FLT4). LENVIMA inhibits other kinases that have been implicated in pathogenic angiogenesis, tumor growth, and cancer progression in addition to their normal cellular functions, including fibroblast growth factor (FGF) receptors FGFR1-4, the platelet derived growth factor receptor alpha (PDGFRα), KIT, and RET. The combination of LENVIMA and everolimus showed increased anti-angiogenic and anti-tumor activity as demonstrated by decreased human endothelial cell proliferation, tube formation, and VEGF signaling in vitro and tumor volume in mouse xenograft models of human renal cell cancer greater than each drug alone. In syngeneic mouse tumor models, the combination of lenvatinib with an anti-PD-1 monoclonal antibody decreased tumor-associated macrophages, increased activated cytotoxic T cells, and demonstrated greater antitumor activity compared to either treatment alone.
LENVIMA® (lenvatinib) Indications in the U.S.
- For the treatment of patients with locally recurrent or metastatic, progressive, radioactive iodine-refractory differentiated thyroid cancer (DTC)
- In combination with KEYTRUDA, for the first-line treatment of adult patients with advanced renal cell carcinoma (RCC)
- In combination with everolimus, for the treatment of adult patients with advanced renal cell carcinoma (RCC) following one prior anti-angiogenic therapy
- For the first-line treatment of patients with unresectable hepatocellular carcinoma (HCC)
- In combination with KEYTRUDA, for the treatment of patients with advanced endometrial carcinoma (EC) that is not microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR), who have disease progression following prior systemic therapy in any setting and are not candidates for curative surgery or radiation.
Please see Prescribing Information for LENVIMA (lenvatinib) at http://www.lenvima.com/pdfs/prescribing-information.pdf .About the Merck and Eisai Strategic CollaborationIn March 2018, Eisai and Merck, known as MSD outside the United States and Canada, through an affiliate, entered into a strategic collaboration for the worldwide co-development and co-commercialization of LENVIMA. Under the agreement, the companies will jointly develop, manufacture and commercialize LENVIMA, both as monotherapy and in combination with Merck’s anti-PD-1 therapy KEYTRUDA.In addition to ongoing clinical studies evaluating the KEYTRUDA plus LENVIMA combination across several different tumor types, the companies have jointly initiated new clinical studies through the LEAP (LEnvatinib And Pembrolizumab) clinical program and are evaluating the combination in more than 10 different tumor types across more than 20 clinical trials.
For more information, visit www.merck.com and connect with us on Twitter, Facebook, Instagram, YouTube and LinkedIn.
For more information about Eisai, please visit www.eisai.com (for global headquarters: Eisai Co., Ltd.), us.eisai.com (for U.S. headquarters: Eisai Inc.) or www.eisai.eu (for Europe, Middle East, Africa, Russia, Australia, and New Zealand headquarters: Eisai Europe Ltd.), and connect with us on Twitter (U.S. and global) and LinkedIn (for U.S. and EMEA).
Merck-Eisai's Keytruda/Lenvima combo gets approval in Japan for kidney cancer
Feb. 25, 2022 7:13 AM ETMerck & Co., Inc. (MRK), ESALY, ESALFBy: Ravikash, SA News Editor
- Merck (NYSE:MRK) and Eisai's (OTCPK:ESALF) (OTCPK:ESALY) drug combination of Keytruda and Lenvima was approved in Japan for radically unresectable or metastatic renal cell carcinoma (RCC), a type of kidney cancer.
- https://seekingalpha.com/news/3805827-merck-eisais-keytrudalenvima-combo-gets-approval-in-japan-for-kidney-cancer
- https://seekingalpha.com/symbol/ESALY
- https://seekingalpha.com/symbol/MRK
- https://www.keytrudalenvima.com/advanced-endometrial-cancer/
SPIKEVAX (elasomeran mRNA vaccine)
EMA COMMITTEE FOR MEDICINAL PRODUCTS FOR HUMAN USE (CHMP) ADOPTS POSITIVE OPINION RECOMMENDING AUTHORIZATION FOR THE USE OF THE MODERNA COVID-19 VACCINE IN CHILDREN (6-11 YEARS) IN THE EUROPEAN UNION
FEBRUARY 24, 2022 DOWNLOAD(OPENS IN NEW WINDOW)
Announcement follows CHMP's previous decision to adopt a positive opinion recommending marketing authorization for Moderna's COVID-19 vaccine to include adolescents 12 years of age and older.
CAMBRIDGE, MA / ACCESSWIRE / February 24, 2022 / Moderna, Inc. (NASDAQ:MRNA), a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, today announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending a variation to the conditional marketing authorization (CMA) to include a 50 µg two-dose series of Spikevax, the Company's vaccine against COVID-19, in children ages 6-11 years. Following the CHMP's positive opinion, the European Commission will make an authorization decision on the use of Spikevax in children ages 6-11 years.
"The CHMP recommendation to authorize the use of our COVID-19 vaccine in children ages 6-11 years in Europe is an important milestone. It highlights the effectiveness and safety of our vaccine in this age group and helps to keep our children safe and able to experience a normal school and family life," said Stéphane Bancel, Chief Executive Officer of Moderna. "We are grateful to the CHMP for their thorough review of our submission and look forward to an authorization decision from the European Commission."
Moderna's vaccine was investigated in the ongoing Phase 2/3 "KidCOVE" study, a randomized, observer-blind, placebo-controlled expansion study to evaluate the safety, tolerability, reactogenicity, and effectiveness of Spikevax (mRNA-1273) given to healthy children 28 days apart. Data submitted to the CHMP from over 4,000 children demonstrated that vaccination of children ages 6-11 years with a 50 μg mRNA-1273 primary series is associated with non-inferior anti-SARS-CoV-2 neutralizing antibody responses when compared to that in individuals 18-25 years old from the Phase 3 COVE study. Positive direct efficacy of two 50 μg doses of mRNA-1273 was also demonstrated and vaccination was generally well tolerated.
The study is being conducted in collaboration with the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH) and the Biomedical Advanced Research and Development Authority (BARDA), part of the Office of the Assistant Secretary for Preparedness and Response at the U.S. Department of Health and Human Services (HHS). The ClinicalTrials.gov identifier is NCT04796896.
The EMA has also recommended updates to the summary of product characteristics (SmPC) for the use of Spikevax in the European Union. The booster dose of 50 µg indicated for individuals 18 years of age and older, is now recommended at least three months following the second dose. This timing has been shortened from the previously approved timeframe of six months. The changes also include the possibility to administer a heterologous (mix-and-match) booster dose, such as Spikevax, following completion of primary vaccination with another authorized COVID-19 vaccine.
Moderna recently announced that the Therapeutic Goods Administration in Australia also granted provisional registration for the use of the Company's mRNA COVID-19 vaccine for active immunization to prevent COVID-19 caused by SARS-CoV-2 in children ages 6-11 years.
Authorized Use
SPIKEVAX (elasomeran mRNA vaccine) has been granted Conditional Marketing Authorization by the European Commission, based upon the recommendation of the European Medicines Agency and is indicated for active immunization to prevent coronavirus disease 2019 (COVID-19) caused by SARS-CoV-2 in individuals six years of age and older. A booster dose may be given at least three months after the second dose for people aged 18 years and older.
To learn more, visit www.modernatx.com.
SOURCE: Moderna, Inc.
View source version on accesswire.com:
https://www.accesswire.com/690252/EMA-Committee-for-Medicinal-Products-for-Human-Use-CHMP-Adopts-Positive-Opinion-Recommending-Authorization-for-The-Use-of-The-Moderna-Covid-19-Vaccine-in-Children-6-11-Years-In-the-European-Union
Moderna COVID vaccine recommended by EMA for children 6-11 years old
Feb. 24, 2022 11:41 AM ET Moderna, Inc. (MRNA) By: Jonathan Block, SA News Editor
- An advisory panel of the European Medicines Agency ("EMA") has recommended that Moderna's (MRNA +11.5%) COVID-19 vaccine be authorized for children 6 to 11 years old.
- https://seekingalpha.com/news/3805278-moderna-covid-vaccine-recommended-by-ema-for-children-6-11-years-old
- https://seekingalpha.com/symbol/MRNA
- https://www.modernatx.com/
- https://www.ema.europa.eu/en/medicines/human/EPAR/spikevax
BRUKINSA (zanubrutinib)
BeiGene Announces U.S. FDA Acceptance of Supplemental New Drug Application for BRUKINSA (zanubrutinib) in Chronic Lymphocytic Leukemia
Feb 22, 2022 7:00 AM
The filing is supported by two global Phase 3 trials of BRUKINSA in chronic lymphocytic leukemia covering both treatment-naïve and relapsed or refractory patient populations
The Prescription Drug User Fee Act target action date is October 22, 2022
CAMBRIDGE, Mass. and BEIJING--(BUSINESS WIRE)-- BeiGene (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global, science-driven biotechnology company focused on developing innovative and affordable medicines to improve treatment outcomes and access for patients worldwide, today announced that the U.S. Food and Drug Administration (FDA) has accepted a supplemental new drug application (sNDA) for BRUKINSA® (zanubrutinib) for the treatment of adult patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). CLL is the most common form of adult leukemia. The Prescription Drug User Fee Act (PDUFA) target action date is October 22, 2022.
“We are pleased with the FDA’s acceptance of BRUKINSA’s filing in CLL. This is an important milestone in BRUKINSA’s global registration program. With superiority in investigator-assessed ORR over ibrutinib in ALPINE for relapsed or refractory patients and in PFS over chemoimmunotherapy in the SEQUOIA study for treatment-naïve patients, BRUKINSA has demonstrated its potential to improve treatment outcomes for CLL patients,” commented Jane Huang, M.D., Chief Medical Officer of Hematology at BeiGene. “We look forward to furthering our discussions with the FDA on this filing and the potential to bring this important treatment option to the CLL community in the U.S.”
The sNDA filing in CLL/SLL includes data from two pivotal randomized Phase 3 studies and eight supportive studies in B cell malignancies. The two global Phase 3 trials of BRUKINSA in CLL/SLL are: ALPINE (NCT03734016) comparing BRUKINSA to ibrutinib in relapsed or refractory (R/R) patients and SEQUOIA (NCT03336333) comparing BRUKINSA to bendamustine and rituximab in treatment-naïve (TN) patients. Additionally, the SEQUOIA study enrolled patients with deletion 17p in a non-randomized arm evaluating BRUKINSA monotherapy in this high risk population. ALPINE and SEQUOIA enrolled patients from a total of 17 countries, including the United States, China, Australia, New Zealand and multiple countries in Europe. Results from the ALPINE trial and the SEQUOIA trial were reported at the 26th European Hematology Association (EHA2021) Virtual Congress in June 2021 and at the 63rd American Society for Hematology (ASH) Annual Meeting in December 2021, respectively.
“While previously approved BTK inhibitors have been transformational for some patients with CLL, there continues to be unmet need as not all patients achieve a favorable clinical response while others are unable to tolerate currently approved BTKi therapies,” said Jennifer R. Brown, MD, PhD, Director of the CLL Center of the Division of Hematologic Malignancies at Dana-Farber Cancer Institute, and a principal investigator in the two studies. “As demonstrated in both the ALPINE AND SEQUOIA studies, BRUKINSA was generally well-tolerated in CLL patients with low rates of atrial fibrillation and showed strong efficacy compared to ibrutinib and chemoimmunotherapy, respectively.”
About BRUKINSA
BRUKINSA is a small molecule inhibitor of Bruton’s tyrosine kinase (BTK) discovered by BeiGene scientists that is currently being evaluated globally in a broad clinical program as a monotherapy and in combination with other therapies to treat various B-cell malignancies. Because new BTK is continuously synthesized, BRUKINSA was specifically designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity. With differentiated pharmacokinetics compared to other approved BTK inhibitors, BRUKINSA has been demonstrated to inhibit the proliferation of malignant B cells within a number of disease relevant tissues.
BRUKINSA has previously been approved for three indications in the United States: for the treatment of mantle cell lymphoma (MCL) in adult patients who have received at least one prior therapy (Nov. 2019)*; for the treatment of adult patients with Waldenström’s macroglobulinemia (WM) (Aug. 2021); and for the treatment of adult patients with relapsed or refractory marginal zone lymphoma (MZL) who have received at least one anti-CD20-based regimen (Sept. 2021)*.
BRUKINSA is supported by a broad clinical program which includes more than 3,900 subjects in 35 trials across 28 markets. To date, BRUKINSA has received more than 20 approvals covering more than 40 countries and regions, including the United States, China, the EU and Great Britain, Canada, Australia and additional international markets. Currently, more than 40 additional regulatory submissions are in review around the world.
* This indication was approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
Please see full U.S. Prescribing Information at www.beigene.com/PDF/BRUKINSAUSPI.pdf and Patient Information at www.beigene.com/PDF/BRUKINSAUSPPI.pdf.
To learn more about BeiGene, please visit www.beigene.com and follow us on Twitter at @BeiGeneGlobal.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220222005574/en/
Source: BeiGene
BeiGene's Brukinsa gets FDA review for expanded use in blood cancer subtype
Feb. 22, 2022 9:20 AM ET BeiGene, Ltd. (BGNE)
By: Ravikash, SA News Editor
- The U.S. Food and Drug Administration (FDA) accepted BeiGene's (NASDAQ:BGNE) application seeking approval of Brukinsa (zanubrutinib) to treat adult patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma.
- https://seekingalpha.com/news/3802479-beigenes-brukinsa-gets-fda-review-for-expanded-use-in-blood-cancer-subtype
- https://seekingalpha.com/symbol/BGNE
- https://www.brukinsa.com/
- https://www.beigene.com/
Jardiance (empagliflozin)
FDA Approves Treatment for Wider Range of Patients with Heart Failure
For Immediate Release: February 24, 2022
Today, the U.S. Food and Drug Administration approved Jardiance (empagliflozin) to reduce the risk of cardiovascular death and hospitalization for heart failure in adults.
Jardiance was originally approved by the FDA in 2014 as a supplement to diet and exercise to improve glucose control in adults with type 2 diabetes. Jardiance is also approved to reduce the risk of cardiovascular death in adults with type 2 diabetes and established cardiovascular disease, and to reduce the risk of death and hospitalization in patients with heart failure and low ejection fraction
See the prescribing information for additional information on risks associated with Jardiance.
Jardiance received Priority Review designation for this indication.
The FDA granted the approval of Jardiance to Boehringer Ingelheim.
Related Information
Lilly-Boehringer Ingelheim's Jardiance granted expanded indication in heart failure
Feb. 24, 2022 3:24 PM ET
By: Jonathan Block, SA News Editor
- The U.S. FDA has granted an additional indiction to Eli Lilly (LLY +1.7%) and Boehringer Ingelheim's Jardiance (empagliflozin) to reduce the risk of cardiac death and hospitalization for heart failure patients.
- https://seekingalpha.com/news/3805360-lilly-boehringer-ingelheims-jardiance-granted-expanded-indication-in-heart-failure
- https://seekingalpha.com/symbol/LLY
- https://www.jardiance.com/
ILUVIEN® (fluocinolone acetonide intravitreal implant) 0.19 mg
February 23, 2022 8:00 AM
Phase 4 PALADIN Study with ILUVIEN® Published in Peer-Reviewed Journal Ophthalmology
- Best corrected visual acuity and central subfield thickness were significantly improved at 36 months
- ILUVIEN treatment resulted in a 70.5% reduction in treatment burden
- Results confirm that use of prior corticosteroid per the U.S. label mitigates the risk of IOP lowering surgery
ATLANTA, Feb. 23, 2022 (GLOBE NEWSWIRE) -- Alimera Sciences, Inc. (Nasdaq: ALIM) (“Alimera”), a global pharmaceutical company whose mission is to be invaluable to patients, physicians and partners concerned with retinal health and maintaining better vision longer, announced today that the 3-year results of the PALADIN study have been published in the peer-reviewed journal Ophthalmology .
These study results show that patients with diabetic macular edema (DME) who received a single dose of the sustained release implant ILUVIEN (fluocinolone acetonide intravitreal implant) 0.19 mg demonstrated statistically significant improvements in best corrected visual acuity, central subfield thickness, and treatment burden at 36 months. Treatment frequency for patients in the PALADIN Study was reduced by 70.5% from a median of 3.4 treatments per year in the 36 months preceding the injection of ILUVIEN to a median of 1.0 treatments per year in the 36 months following the ILUVIEN injection. Further, 2.97% of eyes required IOP lowering surgery, only half of which were due to steroid induced ocular hypertension following the administration of ILUVIEN in accordance with the U.S. label. This compares favorably to the 4.8% in the pivotal FAME Studies. For full publication click Here .
A post hoc analysis of 36-month data from PALADIN found that patients with ≤6 treatments for DME before ILUVIEN administration experienced significantly higher visual acuity gains from baseline compared with patients who had >6 treatments before receiving ILUVIEN (5.70 letters, P=.0048 vs 1.78 letters, P = .8337). These findings suggest that use of ILUVIEN earlier in a treatment algorithm for DME could lead to more desirable visual acuity outcomes.
“We are very pleased to see confirmation from the PALADIN study that the side effect risk of intraocular pressure can be effectively mitigated when ILUVIEN is used in accordance with our U.S. FDA label,” said Rick Eiswirth, Alimera’s President and CEO. “Additionally, we are excited that the PALADIN Study provides another data set demonstrating ILUVIEN’s ability to improve visual acuity with fewer treatment interventions and further supports the hypothesis of our NEW DAY Study.”
The PALADIN study was a phase 4, open-label, prospective, observational study conducted over 36 months at 41 sites across the United States. The study was designed and developed to confirm the benefit of using a prior course of corticosteroid as indicated in the ILUVIEN U.S. label, to mitigate the risk uncontrolled IOP elevation. Researchers enrolled 202 eyes in 159 patients with DME who had previously received corticosteroid treatment without a clinically significant rise in IOP. All eyes were treated with ILUVIEN and patients were followed for up to 36 months.
“The 3-year results of the PALADIN study demonstrate ILUVIEN’s ability to safely and effectively improve visual acuity and retinal anatomy while also reducing the recurrence of edema as evidenced by the decreased treatment burden in patients with DME,” said Michael Singer, MD, Clinical Professor of Ophthalmology, University of Texas Health Science Center, San Antonio, and Director of Clinical Research Medical Center Ophthalmology, San Antonio, Texas. “This is especially important for the diabetic population. Many diabetics are actively working and would otherwise be subject to a high treatment burden of injections that are currently required with anti-vegf therapy alone These findings further support that earlier use of ILUVIEN in a treatment algorithm for DME could lead to more desirable anatomical and functional outcomes.”
Primary outcomes included changes in IOP and interventions to manage IOP elevations; secondary outcomes examined visual, anatomic, and treatment burden–related outcomes. Among the findings at 36 months:
- Mean best corrected visual acuity increased by 3.61 letters ( P = .0222) from baseline.
- Mean central subfield thickness decreased by 60.69 µm ( P < .0001) from baseline.
- Patients received median 1.0 treatment per year after receiving ILUVIEN. In the 36-month period immediately preceding ILUVIEN administration, patients received median 3.4 treatments per year. Thus, ILUVIEN treatment resulted in a 70.5% reduction in treatment burden.
- Surgery to address elevated IOP occurred in 2.97% of eyes; only 1.49% of patients received surgery due to steroid related IOP elevation, with the remaining cases of IOP elevation chiefly attributed to neovascular glaucoma.
- IOP response ≤25 mm Hg after the steroid challenge predicted a similar outcome after ILUVIEN treatment at the final visit in 96.9% of eyes.
- Mean IOP remained stable throughout the study, and IOP increases of >30 mm Hg occurred in only 10.9% of eyes. IOP increases that occurred were considered manageable with standard treatments, according to the study authors.
Full study details are available here .
Visit www.dmeandme.com
About ILUVIEN
The Company’s sole product is ILUVIEN (fluocinolone acetonide intravitreal implant) 0.19 mg sustained release intravitreal implant, injected into the back of the eye. With its CONTINUOUS MICRODOSING™ technology, ILUVIEN is designed to release sub-microgram levels of fluocinolone acetonide, a corticosteroid, for 36 months, to reduce the recurrence of disease and number of treatments required, enabling patients to maintain vision longer with fewer injections. ILUVIEN® (fluocinolone acetonide intravitreal implant) 0.19 mg is indicated in the U.S. for the treatment of diabetic macular edema (DME) in patients who have been previously treated with a course of corticosteroids and did not have a clinically significant rise in intraocular pressure. For safety information please visit https://iluvien.com/#isi and scroll down the page.
About Alimera Sciences, Inc.
Alimera Sciences a global pharmaceutical company whose mission is to be invaluable to patients, physicians and partners concerned with retinal health and maintaining better vision longer. For more information, please visit www.alimerasciences.com .
Alimera's 3-year data for ILUVIEN shows benefit in diabetic macular edema
Feb. 23, 2022 9:48 AM ET
By: Ravikash, SA News Editor
- Alimera Sciences (NASDAQ:ALIM) reported three-year results of a study called PALADIN evaluating the ILUVIEN implant in patients with diabetic macular edema (DME), an eye complication in people with diabetes.
- https://seekingalpha.com/news/3804150-alimeras-3-year-data-for-iluvien-shows-vision-benefit-in-diabetic-macular-edema
- https://seekingalpha.com/symbol/ALIM
- https://iluvien.com/
- https://alimerasciences.com/
Enhertu (trastuzumab deruxtecan)
21 February 2022 07:00 GMT
First HER2-low metastatic breast cancer Phase III results for AstraZeneca and Daiichi Sankyo’s Enhertu offer potential to redefine how the disease is classified and treated
Positive high-level results from the pivotal DESTINY-Breast04 Phase III trial showed Enhertu (trastuzumab deruxtecan) demonstrated a statistically significant and clinically meaningful improvement in both progression-free survival (PFS) and overall survival (OS) in patients with HER2-low unresectable and/or metastatic breast cancer regardless of hormone receptor (HR) status versus physician’s choice of chemotherapy.
Enhertu is a HER2-directed antibody drug conjugate (ADC) being jointly developed by AstraZeneca and Daiichi Sankyo.
All patients in the trial received a HER2 test, and the results were centrally confirmed. HER2-low status was defined as an immunohistochemistry (IHC) score of 1+ or IHC 2+ with a negative in-situ hybridisation (ISH) score.
Up to 55% of all patients with breast cancer have tumours with a HER2 IHC score of 1+, or 2+ in combination with a negative ISH test, a level of HER2 expression not currently eligible for HER2-targeted therapy.1,2 HER2-low expression occurs in both HR-positive and HR-negative disease.3
HER2 testing is well established to determine an appropriate treatment strategy in metastatic breast cancer. Targeting the lower range of HER2 expression may offer another approach to delay disease progression and extend survival in patients with metastatic breast cancer.4 Currently, chemotherapy remains the only treatment option both for patients with HR-positive tumours following progression on endocrine (hormone) therapy, and for those who are HR-negative.5
DESTINY-Breast04 met its primary endpoint, where Enhertu demonstrated superior PFS in previously treated patients with HR-positive HER2-low metastatic breast cancer compared to the standard-of-care chemotherapy. The trial met the key secondary endpoint of PFS in patients with HER2-low metastatic breast cancer regardless of HR status (HR-positive or HR-negative). The trial also met the key secondary endpoints of OS in patients with HR-positive disease and in patients regardless of HR status at interim analysis.
The safety profile of Enhertu was consistent with previous clinical trials, with no new safety concerns identified. Overall interstitial lung disease (ILD) rates were consistent with that observed in late-line HER2-positive breast cancer trials of Enhertu, with a lower rate of Grade 5 ILD observed as determined by an independent adjudication committee.
Susan Galbraith, Executive Vice President, Oncology R&D, AstraZeneca said: “Today’s historic news from DESTINY-Breast04 could reshape how breast cancer is classified and treated. A HER2-directed therapy has never-before shown a benefit in patients with HER2-low metastatic breast cancer. These results for Enhertu are a huge step forward and could potentially expand our ability to target the full spectrum of HER2 expression, validating the need to change the way we categorise and treat breast cancer.”
Ken Takeshita, Global Head, R&D, Daiichi Sankyo said: “Enhertu continues to redefine the treatment of HER2-targetable cancers. DESTINY-Breast04 is the first ever Phase III trial of a HER2-directed therapy in patients with HER2-low metastatic breast cancer to show statistically significant and clinically meaningful benefit in progression-free and overall survival compared to standard treatment. We look forward to sharing the detailed findings of DESTINY-Breast04 with the medical community and initiating discussions with regulatory agencies globally with the goal of bringing Enhertu to patients with metastatic breast cancer previously considered to be HER2-negative.”
The data will be presented at a forthcoming medical meeting and shared with global health authorities.
Enhertu (5.4mg/kg) is approved in more than 40 countries for the treatment of adult patients with unresectable or metastatic HER2-positive breast cancer who have received two or more prior anti-HER2-based regimens based on the results from the DESTINY-Breast01 trial.
Enhertu is being further assessed in a comprehensive clinical development programme evaluating efficacy and safety across multiple HER2-targetable cancers, including breast, gastric, lung and colorectal cancers.
DESTINY-Breast04
DESTINY-Breast04 is a global, randomised, open-label, registrational Phase III trial evaluating the efficacy and safety of Enhertu (5.4 mg/kg) versus physician’s choice of chemotherapy (capecitabine, eribulin, gemcitabine, paclitaxel or nab-paclitaxel) in patients with HR-positive (n=480) or HR-negative (n=60) HER2-low unresectable and/or metastatic breast cancer previously treated with one or two prior lines of chemotherapy. Patients were randomised 2:1 to receive either Enhertu or chemotherapy.
The primary endpoint of DESTINY-Breast04 is PFS in patients with HR-positive disease based on blinded independent central review (BICR). Key secondary endpoints include PFS based on BICR in all randomised patients (regardless of HR status), OS in patients with HR-positive disease and OS in all randomised patients (regardless of HR status). Other secondary endpoints include PFS based on BICR and investigator assessment, duration of response based on BICR and safety.
DESTINY-Breast04 enrolled approximately 540 patients at multiple sites in Asia, Europe and North America. For more information about the trial, visit ClinicalTrials.gov.
Enhertu
Enhertu is a HER2-directed ADC. Designed using Daiichi Sankyo’s proprietary DXd ADC technology, Enhertu is the lead ADC in the oncology portfolio of Daiichi Sankyo and the most advanced programme in AstraZeneca’s ADC scientific platform. Enhertu consists of a HER2 monoclonal antibody attached to a topoisomerase I inhibitor payload, an exatecan derivative, via a stable tetrapeptide-based cleavable linker.
Enhertu (5.4mg/kg) is approved in more than 40 countries for the treatment of adult patients with unresectable or metastatic HER2-positive breast cancer who have received two or more prior anti-HER2-based regimens based on the results from the DESTINY-Breast01 trial.
Enhertu (6.4mg/kg) is approved in several countries for the treatment of adult patients with locally advanced or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma who have received a prior trastuzumab-based regimen based on the results from the DESTINY-Gastric01 trial.
Daiichi Sankyo collaboration
Daiichi Sankyo Company, Limited (referred to as Daiichi Sankyo) and AstraZeneca entered into a global collaboration to jointly develop and commercialise Enhertu (a HER2-directed ADC) in March 2019, and datopotamab deruxtecan (DS-1062; a TROP2-directed ADC) in July 2020, except in Japan where Daiichi Sankyo maintains exclusive rights. Daiichi Sankyo is responsible for manufacturing and supply of Enhertu and datopotamab deruxtecan.
Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca.
AstraZeneca gains on positive late-stage data for breast cancer therapy
Feb. 21, 2022 9:52 AM ET
AstraZeneca PLC (AZN), DSKYF, DSNKY
By: Dulan Lokuwithana, SA News Editor1 Comment
AstraZeneca (NASDAQ:AZN) shares outperformed the broader market in London on Monday in reaction to positive late-stage data for Enhertu in a group of breast cancer patients with low levels of a protein called HER2.
The Anglo-Swedish drugmaker has partnered with Daiichi Sankyo (OTCPK:DSKYF) (OTCPK:DSNKY) to design Enhertu as an antibody-drug conjugate (ADC) targeted at HER2, a tyrosine kinase receptor expressed on multiple types of cancer.
https://seekingalpha.com/news/3802228-azn-stock-gains-on-positive-data-for-breast-cancer-therapy
https://seekingalpha.com/symbol/AZN
https://seekingalpha.com/symbol/DSKYF
https://seekingalpha.com/symbol/DSNKY
VRAYLAR® (cariprazine)
February 22, 2022
AbbVie Submits Supplemental New Drug Application to U.S. FDA for cariprazine (VRAYLAR®) for the Adjunctive Treatment of Major Depressive Disorder
- Submission is based on clinical trial results that include findings showing clinically and statistically significant improvement in the Montgomery-Åsberg Depression Rating Scale (MADRS) total score in patients with major depressive disorder (MDD) treated with cariprazine (VRAYLAR®) and an antidepressant
- If approved, this milestone will be the fourth indication for cariprazine (VRAYLAR®) joining approvals for the treatment of adults with schizophrenia, the acute treatment of manic or mixed episodes associated with bipolar I disorder and the treatment of depressive episodes associated with bipolar I disorder
NORTH CHICAGO, Ill., Feb. 22, 2022 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced that it has submitted a supplemental New Drug Application (sNDA) for cariprazine (VRAYLAR®) to the U.S. Food and Drug Administration (FDA) for the adjunctive treatment of major depressive disorder (MDD) in patients who are receiving ongoing antidepressant therapy. The submission is supported by results from previously announced clinical trials.
A Phase 3 Study 3111-301-001 showed a clinically and statistically significant change from baseline to week six in the Montgomery-Åsberg Depression Rating Scale (MADRS) total score for patients treated with cariprazine at 1.5 mg/day compared with placebo. A second registration-enabling study, RGH-MD-75, showed a clinically and statistically significant change from baseline to week eight in the MADRS total score for patients treated with cariprazine at 2-4.5 mg/day compared with placebo. In both of these studies, safety data were consistent with the established safety profile of cariprazine across indications, with no new safety events identified. Also supporting the submission is study RGH-MD-76 that examined the long-term safety and tolerability of cariprazine over 26 weeks.
"Many people living with major depressive disorder struggle to find a treatment that reduces their depressive symptoms, with many taking years to find the right treatment. Cariprazine, when added to ongoing antidepressant treatment in patients with major depressive disorder, demonstrated that it can reduce depressive symptoms," said Michael Severino, M.D., vice chairman and president, AbbVie. "We look forward to working closely with the FDA during the review of our submission to bring a potential new adjunctive therapy to patients with major depressive disorder who are taking an antidepressant and seeking additional relief. This submission demonstrates our strong commitment to addressing additional gaps in the care of people affected by psychiatric disorders."
Cariprazine is marketed as VRAYLAR® in the United States and is FDA-approved to treat adults with depressive, acute manic and mixed episodes associated with bipolar I disorder, as well as schizophrenia. Cariprazine is being co-developed by AbbVie and Gedeon Richter Plc. More than 8,000 patients worldwide have been treated with cariprazine across more than 20 clinical trials evaluating the efficacy and safety of cariprazine for a broad range of psychiatric disorders.
About Study 3111-301-001
Study 3111-301-001 is a randomized, double-blind, placebo-controlled, multicenter trial with 759 participants conducted in United States, Bulgaria, Estonia, Germany, Hungary, Ukraine and the United Kingdom. Following a screening period of up to 14 days, patients with an inadequate clinical response to their antidepressant monotherapy (ADT) were randomized into three treatment groups (1:1:1). The first group received cariprazine 1.5 mg/day + ADT, the second group received cariprazine 3.0 mg/day + ADT, and the third group received placebo + ADT. For six weeks, the medication was given once daily in addition to the ongoing ADT treatment. Preliminary study findings were announced on October 29th, 2021 and will be presented at a future medical meeting.
About Study RGH-MD-75
Study RGH-MD-75 is a randomized, double-blind, placebo-controlled, flexible-dose, outpatient, multicenter trial with 808 participants, conducted in United States, Estonia, Finland, Slovakia, Ukraine and Sweden. After 7-14 days of screening and washout of prohibited medications, eligible patients entered an 8-week, double-blind treatment period in which they continued antidepressant treatment and were randomized (1:1:1) to adjunctive cariprazine 1-2 mg/day, cariprazine 2-4.5 mg/day, or placebo. Data from Study RGH-MD-75 were published in the Journal of Clinical Psychiatry.4
About Study RGH-MD-76
Study RGH-MD-76 is a long-term, multi-center, open label, flexible-dose safety and tolerability study with 347 participants, conducted in the United States. The study had one treatment group that received caripriazine 1.5-4.5 mg/d + ADT for 26 weeks. Patients entering from the 8-week lead-in study continued ADT at their lead-in study dose; new patients continued their protocol-allowed ADT. On day 1, cariprazine was initiated at 0.5 mg/day; the dosage was increased by 0.5 mg/day until the target dose of 3.0 mg/day was received on days 6 and 7. Dosages could be decreased to 1.5 mg/day for tolerability reasons at any time beginning at week 1 or increased to 4.5 mg/day for inadequate response between weeks 2 and 10. Data from Study RGH-MD-76 were published in International Clinical Psychopharmacology.5
More information can be found on www.clinicaltrials.gov (NCT03738215, NCT01469377, NCT01838876).
About VRAYLAR® (cariprazine)
VRAYLAR is an oral, once-daily atypical antipsychotic approved for the acute treatment of adults with manic or mixed episodes associated with bipolar I disorder (3 to 6 mg/day) and for the treatment of depressive episodes associated with bipolar I disorder (bipolar depression) in adults (1.5 or 3 mg/day). VRAYLAR is also approved for the treatment of schizophrenia in adults (1.5 to 6 mg/day). Use of VRAYLAR in adjunctive treatment of major depressive disorder is not approved and its safety and efficacy have not been evaluated by regulatory authorities.
While the mechanism of action of VRAYLAR is unknown, the efficacy of VRAYLAR could be mediated through a combination of partial agonist activity at central dopamine D₂ and serotonin 5-HT1A receptors and antagonist activity at serotonin 5-HT2A receptors. Pharmacodynamic studies with VRAYLAR have shown that it may act as a partial agonist with high binding affinity at dopamine D3, dopamine D2, and serotonin 5-HT1A receptors. VRAYLAR demonstrated up to ~8-fold greater in vitro affinity for dopamine D3 vs D2 receptors. VRAYLAR also acts as an antagonist at serotonin 5-HT2B and 5-HT2A receptors with high and moderate binding affinity, respectively as well as it binds to the histamine H1 receptors. VRAYLAR shows lower binding affinity to the serotonin 5-HT2C and α1A- adrenergic receptors and has no appreciable affinity for cholinergic muscarinic receptors.6 The clinical significance of these in vitro data is unknown.
VRAYLAR is being developed jointly by AbbVie and Gedeon Richter Plc, with AbbVie responsible for commercialization in the U.S., Canada, Japan, Taiwan and certain Latin American countries (including Argentina, Bolivia, Brazil, Chile, Colombia, Ecuador, Mexico, Peru and Venezuela).
VRAYLAR® (cariprazine) Uses and Important Safety Information
VRAYLAR is a prescription medicine used in adults:
- to treat schizophrenia
- for short-term (acute) treatment of manic or mixed episodes that happen with bipolar I disorder
- to treat depressive episodes that happen with bipolar I disorder (bipolar depression)
- https://www.vraylar.com/
For more information about AbbVie, please visit us at www.abbvie.com. Follow @abbvie on Twitter, Facebook, Instagram, YouTube and LinkedIn.
SOURCE AbbVie
AbbVie seeks FDA approval for fourth indication of cariprazine
Feb. 22, 2022 8:50 AM ET
By: Dulan Lokuwithana, SA News Editor
- AbbVie (NYSE:ABBV) announced Tuesday it submitted a supplemental New Drug Application (sNDA) for the antipsychotic medication cariprazine to the U.S. Food and Drug Administration (FDA) seeking its approval for major depressive disorder (MDD).
- https://seekingalpha.com/news/3802446-abbvie-stock-in-focus-on-fda-filing-for-anti-depressant-drug
- https://seekingalpha.com/symbol/ABBV
- https://www.vraylar.com/
- https://www.abbvie.com/
COVISHIELD (STI-9167)
Sorrento Announces COVISHIELD (STI-9167) Antibody Strongly Neutralizes BA.2 Omicron Sublineage Virus as Well as BA.1 and BA.1+R346k Omicron Viruses
February 22, 2022 at 9:00 AM ESTDownload PDF
- STI-9167 neutralizing antibody (nAb) maintains high neutralization potency against the recently emerging Omicron sublineage BA.2 as was previously demonstrated for the Omicron sublineage BA.1 viruses, including Omicron BA.1 (+R346K mutation).
- An IND application for COVISHIELD™ IN, formulated for intranasal (IN) administration for clinical use in the context of early symptomatic and asymptomatic infections, was submitted to the FDA on February 1, 2022.
SAN DIEGO, Feb. 22, 2022 (GLOBE NEWSWIRE) -- Sorrento Therapeutics, Inc. (Nasdaq: SRNE, "Sorrento") today announced that additional preclinical results demonstrate broad spectrum COVISHIELD (STI-9167) neutralizing activity against Omicron BA.1, Omicron BA.1+R346K, and the increasingly prevalent sublineage, Omicron BA.2. The Omicron BA.2 encodes the core spike mutations that serve to generally define the Omicron lineage, however, it carries additional spike mutations that were not identified within the BA.1 Omicron sublineage. The BA.2 Omicron virus is reported to have 50% more enhanced transmissibility as compared to the BA.1 Omicron viruses and may cause potentially similar disease severity as the Delta virus. Using VSV pseudotyped viruses, STI-9167 neutralization potency was benchmarked against antibody reagents based on EUA-approved SARS-CoV-2 nAbs. STI-9167 neutralization activity was the topmost performing nAb among the comparative nAb treatments, similar to the recently EUA-approved LY-1404 nAb but superior to all other EUA-approved nAbs in neutralizing Omicron BA.1, BA.1+R346K and BA.2 viruses.
About COVISHIELD and COVISHIELD IN
Initially isolated as a SARS-CoV-2 (WA-1 strain) neutralizing antibody candidate following vaccination of transgenic mice, the STI-9167 antibody was optimized to maximize protein stability and minimize interactions with host Fc gamma receptors. COVISHIELD (STI-9167) is an intravenous (IV) formulation and COVISHIELD IN (STI-9199) is a proprietary IN formulation of COVISHIELD. Using established master cell banks, GMP drug products for COVISHIELD and COVISHIELD IN have been generated at Sorrento in preparation for anticipated Phase 1 through pivotal Phase 2/3 human clinical trials. Technology transfer of methods and GMP processes in support of commercial-scale GMP manufacturing is currently underway.
About Clinical Development Plans for COVISHIELD (STI-9167) and COVISHIELD IN (STI-9199)
Sorrento has demonstrated the protective effects of SARS-CoV-2 nAbs administered by either IV or IN routes in preclinical COVID-19 animal models and the safety of other SARS-CoV-2 nAbs administered by IV and IN routes to human subjects (e.g., COVI-AMG™ and COVIDROPS™). Development of COVISHIELD and COVISHIELD IN formulations will proceed along independent paths. Current clinical study plans for COVISHIELD IN, outlined in an IND application received for review on February 1, 2022 and pending feedback from regulatory agencies, call for evaluation of safety following antibody administration at single doses in healthy normal adults or asymptomatic Omicron infected patients, followed by large Phase 2/3 clinical trials globally for newly infected COVID-19 patients. The IND application in support of similar Phase 1 trials of COVISHIELD is scheduled for submission in the first half of March 2022.
About Sorrento Intranasal nAbs for the Treatment of COVID-19
In Phase 1 and Phase 2 clinical studies of COVIDROPS (STI-2099), the IN formulation of COVI-AMG (STI-2020), a SARS CoV-2 nAb with demonstrated activity in vitro and in vivo against multiple VOCs including Alpha, Delta, Lambda, and Mu, IN dose levels up to 60 mg have been well tolerated. COVID-19 patients with newly diagnosed SARS-CoV-2 infections were treated with STI-2099 in randomized, double-blind, placebo-controlled Phase 2 trials in the US and UK. The primary efficacy endpoint in Phase 2 studies is viral load reduction in nasopharyngeal (NP) swab material by qRT-PCR.
In the US Phase 2 study, outpatient adults received a single instillation of 10 mg or 20 mg or 40 mg COVIDROPS or placebo with a randomization of 1:1:1:1. The US Phase 2 study recently concluded (n=96 individuals treated) and data is pending. In the UK Phase 2 study, outpatient adults received a single instillation of 10 mg or 20 mg COVIDROPS or placebo in their homes with a randomization of 2:2:1. The study recently reached the 50% enrollment threshold (n=179 individuals treated) and the interim analysis data (including VOC sequencing) is pending.
For more information visit www.sorrentotherapeutics.com
Source: Sorrento Therapeutics, Inc.
Sorrento' COVISHIELD shows neutralizing activity vs Omicron subvariants
Feb. 22, 2022 10:37 AM ETSorrento Therapeutics, Inc. (SRNE)
By: Ravikash, SA News Editor2 Comments
- Sorrento Therapeutics (SRNE +0.5%) said additional preclinical results showed broad spectrum COVISHIELD (STI-9167) neutralizing activity against Omicron BA.1, Omicron BA.1+R346K, and the increasingly prevalent sublineage, Omicron BA.2.
- https://seekingalpha.com/news/3802558-sorrento-covishield-shows-neutralizing-activity-vs-omicron-subvariants
- https://seekingalpha.com/symbol/SRNE
- https://sorrentotherapeutics.com/research/covid-19/covishield/
Zeposia (ozanimod)
Bristol Myers Squibb Presents Interim Results from Long-Term Study Reinforcing Maintenance of Response and Safety Profile of Zeposia (ozanimod) in Patients with Moderately to Severely Active Ulcerative Colitis
02/17/2022CATEGORY:
The percentage of patients achieving clinical remission, clinical response, endoscopic improvement and corticosteroid-free remission was maintained through Week 142
Zeposia is the first and only oral sphingosine 1-phosphate (S1P) receptor modulator approved to treat patients with ulcerative colitis
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced interim results from the True North open-label extension study evaluating the long-term efficacy and safety profile of Zeposia (ozanimod) in patients with moderately to severely active ulcerative colitis (UC). Findings show that the percentage of patients achieving clinical remission, clinical response, endoscopic improvement and corticosteroid-free remission was maintained through Week 142. No new safety signals emerged in the study. These data (Presentation #DOP44) will be presented at the 17th Congress of the European Crohn's and Colitis Organisation (ECCO), taking place February 16-19, 2022.
“For clinicians treating patients with this serious, chronic disease, results from the True North extension study provide an understanding of long-term therapeutic outcomes and help to identify an appropriate treatment approach for their patients with ulcerative colitis,” said Silvio Danese, M.D., Director, Gastroenterology and Endoscopy, IRCCS, San Raffaele Hospital and University Vita-Salute San Raffaele in Milan. “These findings demonstrate important and clinically meaningful responses across multiple key endpoints and build upon our current knowledge of the efficacy and safety profile of Zeposia.”
In the True North extension study, data from an interim analysis of patients (n=823) who had previously participated in the Phase 3 True North Zeposia clinical trial were examined.At Weeks 46, 94 and 142, 45% (203/452), 51% (109/213) and 45% (39/87) of participants, respectively, were in clinical remission, and 80% (352/441), 84% (176/209) and 86% (73/85) achieved clinical response, respectively. The efficacy of Zeposia in those who entered the long-term study as responders on Day 1 was higher compared to the total population, with 70% (107/152) and 69% (42/61) achieving clinical remission at Weeks 46 and 94, respectively, and 95% (145/152) and 98% (60/61) achieving clinical response at Weeks 46 and 94, respectively. At the time of this analysis, of the 823 patients from the Phase 3 True North trial who entered the open-label extension study, 64% subsequently completed Week 46, 34% completed Week 94, and 14% completed Week 142.The most common reason for discontinuation was lack of efficacy (21%). No new safety signals were seen with longer-term Zeposia use in the 1,158 patients within the pooled population, including patients from the Phase 2 Touchstone study and the Phase 3 True North study.
Additional Bristol Myers Squibb-sponsored abstracts presented at the ECCO 2022 Congress can be accessed online here.
“These data reinforce the growing body of evidence demonstrating the long-term efficacy and safety of Zeposia and showcase its role as an important therapeutic option before biologic and Janus kinase inhibitor treatments in patients with moderately to severely active ulcerative colitis,” said Jonathan Sadeh, M.D., MSc., senior vice president of Immunology and Fibrosis Development, Bristol Myers Squibb. “With our heritage in transformational science for immune-mediated diseases – and our commitment to inflammatory bowel disease research – we are seeking to find solutions that have the goal of redefining treatment outcomes and elevating standards of care for the gastroenterology community.”
Visit this page on BMS.com for more information on Bristol Myers Squibb’s scientific approach and resources on gastrointestinal immune-mediated diseases.
About True North
True North is a Phase 3, multicenter, randomized, double-blind, placebo-controlled clinical trial assessing the efficacy and safety of Zeposia 0.92 mg in patients with moderately to severely active ulcerative colitis (UC) who had an inadequate response or were intolerant to any of the following: oral aminosalicylates, corticosteroids, immunomodulators or a biologic. Patients were to be receiving treatment with oral aminosalicylates and/or corticosteroids prior to and during the induction period. A total of 30% of patients had previously failed or were intolerant to tumor necrosis factor (TNF) blockers. Of these patients, 63% received at least two biologics including TNF blockers. At study entry, mean age was 42 years, 60% were male and mean disease duration was 7 years; patient characteristics were well balanced across treatment groups. In the 10-week induction study (UC Study 1), a total of 645 patients were randomized 2:1 to receive Zeposia (n=429) or placebo (n=216), of whom 94% and 89%, respectively, completed the induction study. No new safety signals were observed in the induction phase.
In maintenance study (UC Study 2) a total of 457 patients who received Zeposia in either UC Study 1 or in an open-label arm and achieved clinical response at Week 10 were re-randomized 1:1 and were treated with either Zeposia 0.92 mg (n=230) or placebo (n=227) for 42 weeks, for a total of 52 weeks of treatment. Concomitant aminosalicylates were required to remain stable through Week 52. Patients on concomitant corticosteroids were to taper their dose upon entering the maintenance study. Of these, 80% and 54.6% of patients who received Zeposia and placebo, respectively, completed the study. In the maintenance phase, the overall safety profile was consistent with the known safety profile for Zeposia and patients with moderate to severe UC. More information about the True North trial can be found on www.clinicaltrials.gov, NCT02435992.
For the True North open-label extension (OLE) study, eligible patients who completed the maintenance UC Study 2 were rolled into the OLE study, which is ongoing and designed to assess the longer-term profile of Zeposia for the treatment of moderately to severely active UC. Patients were also eligible to enter the OLE study if they completed UC Study 1 and did not achieve a clinical response or if they experienced a disease relapse during UC Study 2. Among patients who entered the trial, clinical remission, clinical response, endoscopic improvement and symptomatic remission were generally maintained through Week 142. No new safety concerns were identified in this study extension in patients with ulcerative colitis. More information about the open-label extension trial can be found on www.clinicaltrials.gov, NCT02531126.
Bristol Myers Squibb thanks the patients and investigators involved in the True North clinical trial.
About Zeposia (ozanimod)
Zeposia (ozanimod) is an oral, sphingosine 1-phosphate (S1P) receptor modulator that binds with high affinity to S1P receptors 1 and 5. Zeposia reduces the capacity of lymphocytes to migrate from lymphoid tissue, reducing the number of circulating lymphocytes in peripheral blood. The mechanism by which Zeposia exerts therapeutic effects in ulcerative colitis (UC) is unknown but may involve the reduction of lymphocyte migration into the intestines.
Bristol Myers Squibb is continuing to evaluate Zeposia in the ongoing True North open-label extension trial, designed to assess the longer-term profile of Zeposia for the treatment of moderately to severely active UC. The company is also investigating Zeposia for the treatment of moderately to severely active Crohn’s disease in the ongoing Phase 3 YELLOWSTONE clinical trial program.
The European Commission approved Zeposia for the treatment of adult patients with relapsing remitting multiple sclerosis (RRMS) with active disease as defined by clinical or imaging features in May 2020 and for the treatment of adults with moderately to severely active UC who have had an inadequate response, lost response, or were intolerant to either conventional therapy or a biologic agent in November 2021. The U.S. Food and Drug Administration (FDA) approved Zeposia for the treatment of adults with relapsing forms of multiple sclerosis (RMS) in March 2020and adults with moderately to severely active UC on May 27, 2021.
U.S. FDA APPROVED INDICATIONS
ZEPOSIA (ozanimod) is indicated for the treatment of:
1. Relapsing forms of multiple sclerosis (MS), to include clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease, in adults.
2. Moderately to severely active ulcerative colitis (UC) in adults.
For additional safety information, please see the full
Prescribing InformationandMedication Guide.
For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220215005932/en/
Source: Bristol Myers Squibb
https://seekingalpha.com/symbol/BMY
BMY Dividend History
https://www.nasdaq.com/market-activity/stocks/bmy/dividend-history
BAVENCIO® (avelumab)
Long-Term Follow-Up Data Reinforce Continued Overall Survival Benefit of BAVENCIO® (avelumab) First-Line Maintenance Treatment in Patients with Advanced Urothelial Carcinoma
Not intended for UK-based media
- 38-month median follow-up data from the Phase III JAVELIN Bladder 100 trial demonstrated prolonged median OS of 23.8 months with BAVENCIO plus best supportive care (BSC) in the first-line maintenance setting versus a median OS of 15.0 months with BSC alone
- BAVENCIO continues to be the first and only immunotherapy to improve survival in the first-line setting in locally advanced or metastatic UC in a Phase III study
Rockland, MA, February 18, 2022– EMD Serono, the healthcare business of Merck KGaA, Darmstadt, Germany, in the US and Canada, today announced the results of an exploratory analysis from the Phase III JAVELIN Bladder 100 trial with 19 additional months of follow-up data from the initial primary analysis. This analysis reinforced the original results and showed that BAVENCIO® (avelumab) plus best supportive care (BSC) in the first-line maintenance setting prolonged median overall survival (OS) by 8.8 months versus BSC alone for patients with locally advanced or metastatic urothelial carcinoma (UC) whose tumors had not progressed on a platinum-based chemotherapy. These results were presented at the 2022 American Society of Clinical Oncology’s annual Genitourinary Cancers Symposium taking place February 17-19, 2022.
“Since the introduction of the first-line maintenance regimen of BAVENCIO plus best supportive care, it has been recommended with the highest level of evidence in the NCCN, ESMO, EAU and JUA guidelines, and BAVENCIO first-line maintenance has become a standard of care in the locally advanced and metastatic UC treatment setting. The results from this analysis further reinforce the benefit of first-line maintenance therapy, and of BAVENCIO as the only immunotherapy in the maintenance setting shown to improve survival in this disease,” said Thomas Powles, MBBS, MRCP, MD, Professor of Genitourinary Oncology, Lead for Solid Tumor Research at Barts Cancer Institute, Queen Mary University of London, and Director of Barts Cancer Centre, London, UK.
At 38 months median follow-up, patients who received first-line maintenance BAVENCIO plus BSC showed consistent overall survival benefit over patients on BSC alone.
- Median OS was 23.8 months (95% CI, 19.9 to 28.8) vs 15.0 months (95% CI, 13.5 to 18.2) (HR 0.76; 95% CI, 0.631 to 0.915).1
- 43.7% of patients (95% CI, 38.2% to 49.0%) in the BAVENCIO arm were alive at 30 months vs 33.5% (95% CI, 28.4% to 38.7%) of patients who received BSC alone.1
In the population of patients with PD-L1+ tumors (n=358):
- Median OS was 30.9 months (95% CI, 24.0-39.8) vs 18.5 months (95% CI, 14.1-24.2) (HR 0.69; 95% CI, 0.521 to 0.901).1
- More than half (51.3%; 95% CI, 43.7% to 58.4%) of patients who received BAVENCIO were alive at 30 months vs 38.5% (95% CI, 30.9% to 46.1%) in the BSC arm.1
Median overall survival was measured from the time of randomization, after completion of four to six cycles of platinum-based chemotherapy.
The safety profile for BAVENCIO was consistent with the overall JAVELIN monotherapy clinical development program, with no new safety signals. Patients continued treatment until disease progression, unacceptable toxicity or any other criteria for withdrawal occurred. In the primary population of all randomized patients, 19.5% of patients received ≥2 years of treatment, with 10.2% of patients discontinuing due to treatment-related adverse event with onset after ≥12 months of treatment.
“Locally advanced and metastatic urothelial carcinoma has a low five-year survival rate and high recurrence rate, requiring additional medicines to maintain the benefits of chemotherapy and improve survival rates. The continued improvement in survival in the JAVELIN Bladder 100 trial further supports the use of BAVENCIO as maintenance therapy in patients whose disease has not progressed on first-line platinum-containing chemotherapy and reinforces our commitment to further evaluation of BAVENCIO in advanced bladder cancer,” said Victoria Zazulina, MD, Head of Development Unit Oncology, Merck KGaA, Darmstadt, Germany.
About JAVELIN Bladder 100
JAVELIN Bladder 100 (NCT02603432) is a Phase III, multicenter, multinational, randomized, open-label, parallel-arm study investigating first-line maintenance treatment with BAVENCIO plus BSC versus BSC alone in patients with locally advanced or metastatic UC. The primary endpoint was OS in the two primary populations of all patients and patients with PD-L1+ tumors defined by the Ventana SP263 assay. Secondary endpoints included progression-free survival, anti-tumor activity, safety, pharmacokinetics, immunogenicity, predictive biomarkers and patient-reported outcomes in the co-primary populations. All primary and secondary endpoints are measured from the time of randomization.
About BAVENCIO® (avelumab)
BAVENCIO is a human anti-programmed death ligand-1 (PD-L1) antibody. BAVENCIO has been shown in preclinical models to engage both the adaptive and innate immune functions. By blocking the interaction of PD-L1 with PD-1 receptors, BAVENCIO has been shown to release the suppression of the T cell-mediated antitumor immune response in preclinical models.7-9 In November 2014, Merck KGaA, Darmstadt, Germany and Pfizer announced a strategic alliance to co-develop and co-commercialize BAVENCIO.
BAVENCIO Approved Indications
BAVENCIO® (avelumab) is indicated in the US for the maintenance treatment of patients with locally advanced or metastatic urothelial carcinoma (UC) that has not progressed with first-line platinum-containing chemotherapy. BAVENCIO is also indicated for the treatment of patients with locally advanced or metastatic UC who have disease progression during or following platinum-containing chemotherapy, or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy
BAVENCIO in combination with axitinib is indicated in the US for the first-line treatment of patients with advanced renal cell carcinoma (RCC).
In the US, the FDA granted accelerated approval for BAVENCIO for the treatment of adults and pediatric patients 12 years and older with metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials.
BAVENCIO is currently approved for at least one indication for patients in more than 50 countries.
Please see full US Prescribing Information and Medication Guide available at http://www.BAVENCIO.com
About EMD Serono, Inc.
EMD Serono - the healthcare business of Merck KGaA, Darmstadt, Germany in the U.S. and Canada - aspires to create, improve and prolong life for people living with difficult-to-treat conditions like infertility, multiple sclerosis and cancer. The business is imagining the future of healthcare by working to translate the discovery of molecules into potentially meaningful outcomes for people with serious unmet medical needs. EMD Serono’s global roots go back more than 350 years with Merck KGaA, Darmstadt, Germany. Today, the business has approximately 1,500 employees around the country with commercial, clinical and research operations in Massachusetts. www.emdserono.com.
About Merck KGaA, Darmstadt, Germany
Merck KGaA, Darmstadt, Germany, a leading science and technology company, operates across healthcare, life science and electronics. Around 58,000 employees work to make a positive difference to millions of people’s lives every day by creating more joyful and sustainable ways to live. From advancing gene editing technologies and discovering unique ways to treat the most challenging diseases to enabling the intelligence of devices – the company is everywhere. In 2020, Merck KGaA, Darmstadt, Germany, generated sales of € 17.5 billion in 66 countries.
Merck KGaA Bavencio shows survival benefit in bladder cancer in long term data
Feb. 18, 2022 9:41 AM ET
MERCK Kommanditgesellschaft auf Aktien (MKKGY)
By: Ravikash, SA News Editor
- Merck KGaA (OTCPK:MKKGY) said long term data showed overall survival benefit of Bavencio (avelumab)as first-line maintenance treatment in patients with advanced urothelial carcinoma, a type of bladder cancer.
- https://seekingalpha.com/news/3801959-merck-kgaa-bavencio-shows-survival-benefit-in-bladder-cancer-in-long-term-data
- https://seekingalpha.com/symbol/MKKGY
- https://www.bavencio.com/hcp
- https://www.emdgroup.com/en
TREMFYA® (guselkumab)
New Phase 2b Data Show the Majority of Adults with Moderately to Severely Active Ulcerative Colitis Achieved Clinical Response with TREMFYA® (guselkumab) at 12 Weeks
Feb 18, 2022United States
Data from the Phase 2b QUASAR Induction Study 1 showed approximately 60 percent of patients achieved the primary endpoint of clinical response, and approximately 30 percent of patients showed endoscopic improvement with TREMFYA treatment compared with placebo
Results also showed a greater proportion of TREMFYA-treated patients achieved clinical remission and other major secondary endpoints at week 12 compared with placebo
SPRING HOUSE, PENNSYLVANIA, February 18, 2022 – The Janssen Pharmaceutical Companies of Johnson & Johnson today announced results from the Phase 2b QUASAR Induction Study 1. Results showed a significantly greater proportion of adults with moderately to severely active ulcerative colitis (UC) who previously had an inadequate response or intolerance to conventional therapies and/or selected advanced therapies and were treated with TREMFYA® (guselkumab) achieved clinical responsea at week 12, the study’s primary endpoint (intravenous [IV] TREMFYA 200 mg: 61.4 percent [62/101] and IV TREMFYA 400 mg: 60.7 percent [65/107]) compared with placebo (27.6 percent [29/105]).1 Safety data at week 12 were consistent with the safety profile for TREMFYA in approved indications.1,2 TREMFYA is not currently approved for the treatment of adults with UC in the U.S.2
These new efficacy and safety data are the first reported on the investigational use of TREMFYA for moderately to severely active UC in an analysis of 313 patients enrolled in the QUASAR clinical program.3 These findings were presented today as an oral presentation (OP23) at the 17th Congress of the European Crohn’s and Colitis Organisation (ECCO) taking place virtually from February 16-19.1
“Despite available treatment options, there are patients with moderately to severely active ulcerative colitis who are still in need of additional therapeutic approaches due to inadequate response or intolerance to their current treatment,” said presenting study author Axel Dignass, M.D., Ph.D., Head of the Department of Medicine and Professor of Medicine and Gastroenterology at the Agaplesion Markus Hospital, Goethe University in Frankfurt, Germany.b “Results from the QUASAR study show both IV induction doses of TREMFYA achieved clinical responses in patients with moderately to severely active ulcerative colitis at rates greater than placebo.”
In the study, the primary endpoint of the clinical response is defined as a decrease from induction baseline in the modified Mayo score by ≥30 percent and ≥2 points, with either a ≥1 point decrease from baseline in the rectal bleeding subscore or a rectal bleeding subscore of 0 or 1.1,c Major secondary endpoints in the QUASAR study include clinical remission,d symptomatic remission,e endoscopic improvement,f histo-endoscopic mucosal improvementg and endoscopic normalizationh at week 12.1,c
Results from the five secondary endpoints showed:1,c
- Clinical remission ratesd were achieved in 25.7 and 25.2 percent of patients in the 200 and 400 mg TREMFYA groups, respectively, compared with 9.5 percent in the placebo group.
- Symptomatic remission ratese were achieved in 50.5 and 47.7 percent of patients in the respective 200 and 400 mg TREMFYA groups compared with 20.0 percent in the placebo group.
- Endoscopic improvement ratesf were achieved in 30.7 and 30.8 percent of patients in the respective 200 and 400 mg TREMFYA groups compared with 12.4 percent in the placebo group.
- Histo-endoscopic mucosal improvement ratesg were achieved in 19.8 and 27.1 percent of patients in the respective 200 and 400 mg TREMFYA groups compared with 8.6 percent in the placebo group.
- Endoscopic normalization ratesh were achieved in 17.8 and 14 percent of patients in the respective 200 and 400 mg TREMFYA groups compared with 6.7 percent in the placebo group.
Safety findings for both TREMFYA dose groups were consistent with the known safety profile for TREMFYA in approved indications.1,2 The proportions of patients reporting adverse events (AEs), serious AEs, and AEs leading to discontinuation in both TREMFYA dose groups were not greater compared with placebo.1 No serious infections, cases of malignancy or death were reported for TREMFYA.1
“Data from the Phase 2b QUASAR study provide the initial evidence in the development of TREMFYA as a potential treatment for adult patients with moderately to severely active ulcerative colitis,” said Jan Wehkamp, M.D., Ph.D., Vice President, Gastroenterology Disease Area Leader, Janssen Research & Development, LLC. “We look forward to advancing this important research in the ongoing QUASAR Phase 3 induction and maintenance studies as we continue in our commitment to develop therapeutic options for patients with debilitating diseases like ulcerative colitis.”
Phase 3 clinical trials evaluating TREMFYA for the treatment of adults with moderately to severely active UC are ongoing and enrolling participants. Learn more through the Janssen Global Trial Finder.
Editor’s Notes:
- Clinical response is defined as a decrease from induction baseline in the modified Mayo score by ≥30 percent and ≥2 points, with either a ≥1 point decrease from baseline in the rectal bleeding subscore or a rectal bleeding subscore of 0 or 1.1 Modified Mayo score is a three-component (stool frequency, rectal bleeding, and endoscopy subscores) Mayo score without the physician’s global assessment.4
- Professor Dignass is a paid consultant for Janssen. He has not been compensated for any media work.
- Please see the ‘About QUASAR Induction Study 1’ section below for further details regarding the study design.
- Clinical remission is defined as a stool frequency subscore of 0 or 1, a rectal bleeding score of 0, and an endoscopy subscore of 0 or 1 with no friability present on the endoscopy, where the stool frequency subscore has not increased from induction baseline.1
- Symptomatic remission is defined as a stool frequency subscore of 0 or 1 and a rectal bleeding subscore of 0, where the stool frequency subscore has not increased from induction baseline.1
- Endoscopic improvement is defined as an endoscopy subscore of 0 or 1 with no friability present on the endoscopy.1
- Histo-endoscopic mucosal improvement is defined as achieving a combination of histologic improvement (neutrophil infiltration in <5 percent of crypts, no crypt destruction, and no erosions, ulcerations or granulation tissue according to the Geboes grading system) and endoscopic improvement.1
- Endoscopic normalization is defined as an endoscopy subscore of 0.1
About QUASAR Induction Study 1 (NCT04033445; EudraCT 2018-004002-25 )3,5
QUASAR Induction Study 1 is a 12-week, double-blind, randomized, placebo-controlled, multicenter Phase 2b induction dose-ranging study evaluating the efficacy and safety of TREMFYA in adults with moderately to severely active UC with inadequate response/intolerance to conventional therapies (thiopurines or corticosteroids) and/or advanced therapies (TNFα antagonists, vedolizumab, or tofacitinib).
Participants had to have a baseline modified Mayo score of 5 to 9 (inclusive), with a Mayo rectal bleeding subscore ≥1 and a Mayo endoscopy subscore ≥2 obtained during central review of video endoscopy.
Participants were randomized equally into three groups receiving treatment at weeks 0, 4 and 8 with either TREMFYA IV dosed at 200 or 400 mg, or matched placebo.1
About TREMFYA® (guselkumab)2
Developed by Janssen, TREMFYA is the first approved fully human monoclonal antibody that selectively binds to the p19 subunit of interleukin (IL)-23 and inhibits its interaction with the IL-23 receptor. TREMFYA is approved in the U.S., Canada, Japan, and a number of other countries worldwide for the treatment of adults with moderate to severe plaque psoriasis (PsO) who may benefit from taking injections or pills (systemic therapy) or phototherapy (treatment using ultraviolet light), and for the treatment of adult patients with active psoriatic arthritis (PsA). It is also approved in the European Union for the treatment of moderate to severe plaque PsO in adults who are candidates for systemic therapy, and alone or in combination with methotrexate (MTX) for the treatment of active PsA in adult patients who have had an inadequate response or who have been intolerant to a prior disease-modifying antirheumatic drug therapy.
The Janssen Pharmaceutical Companies of Johnson & Johnson maintain exclusive worldwide marketing rights to TREMFYA®.
Please read the full Prescribing Information, including Medication Guide for TREMFYA®, and discuss any questions that you have with your doctor.
Learn more at www.janssen.com. Follow us at www.twitter.com/JanssenGlobal.
Johnson & Johnson’s Janssen unit posts 48-week data for Tremfya in Crohn’s disease
Feb. 18, 2022 11:19 AM ET
By: Dulan Lokuwithana, SA News Editor
The Janssen Pharmaceutical, a subsidiary of Johnson & Johnson (NYSE:JNJ), said that the company’s experimental therapy for Crohn’s disease (CD), Tremfya led to clinical remission in the majority of adults with moderate to severe form of the disease in a Phase 2 clinical trial at week 48.
https://seekingalpha.com/symbol/JNJ
Freezor™ and Freezor™ Xtra Cardiac Cryoablation Focal Catheters
FEB 18, 2022
Medtronic receives FDA expanded approval for cardiac cryoablation catheters for pediatric treatment of a common heart rhythm condition
Family of focal ablation catheters are the first and only to treat specific type of supraventricular tachycardia (SVT)
DUBLIN, Feb. 18, 2022 /PRNewswire(opens new window)/ -- Medtronic plc (NYSE:MDT), a global leader in healthcare technology, today announced that the Freezor™ and Freezor™ Xtra Cardiac Cryoablation Focal Catheters are the first and only ablation catheters approved by the U.S. Food and Drug Administration (FDA) to treat the growing prevalence of pediatric Atrioventricular Nodal Reentrant Tachycardia (AVNRT).
AVNRT is the most common form of supraventricular tachycardia (SVT), and is a life-threatening abnormal heart rhythm, with 89,000 cases each year and growing. Nearly 35% of AVNRT cases occur in pediatrics, or, children under the age of 18. Due to an abnormal circuit within the heart's conduction system, AVNRT causes a very rapid heart rhythm, and if left untreated, can affect the heart's ability to pump normally, leading to palpitations, lightheadedness, and syncope.
Catheter ablation is a first-line therapy for treatment of AVNRT. The Freezor and Freezor Xtra Catheters are flexible, single-use devices used to freeze cardiac tissue and block unnecessary electrical signals within the heart. The Freezor family enables safe and effective focal cryoablation therapy and has treated more than 140,000 patients across 67 countries. Cryoablation can reduce the risk of permanent AV block, a complication of AVNRT procedures performed with radiofrequency (RF) ablations that results in the partial or complete interruption of the heart's electrical signals, which dangerously disrupts heart rhythm.
"There are very few devices approved to treat medically complex pediatric cardiology patients today," said Bryan C. Cannon, M.D., professor of pediatrics and past president of the Pediatric & Congenital Electrophysiology Society (PACES), the largest organization in the world for pediatric rhythm specialty doctors. "With an FDA indication expansion, the Freezor and Freezor Xtra cardiac cryoablation catheters allow even the youngest of cardiology patients access to a safe, life-enhancing technology that will help advance cardiac care for AVNRT."
The indication expansion approval is supported by results from ICY-AVNRT and multiple pediatric randomized, multi-center studies that demonstrated the safety and effectiveness of the treatment of AVNRT using the Freezor and Freezor Xtra cardiac cryoablation catheters. ICY-AVNRT data reported acute procedural success of 95% with no reports of permanent pacemaker due to complete AV block.1 The larger body of evidence, including a total of sixteen studies, also observed high efficacy rates and low adverse events.2-17
The Freezor cardiac cryoablation catheter was first commercially available in the U.S. for adult use of AVNRT in 2003, followed by the Freezor Xtra cardiac cryoablation catheter in 2016. The Freezor family of cryoablation catheters(opens new window) also includes the Freezor MAX cardiac cryoablation catheter, which is approved for use in conjunction with the Arctic Front™ Advance cryoballoon for the treatment of atrial fibrillation (AF).
"We're proud of our work with PACES and FDA in this first-of-its-kind, multi-stakeholder initiative to address a critical patient population," said Rebecca Seidel, president of the Cardiac Ablation Solutions business, which is part of the Cardiovascular Portfolio at Medtronic. "The shared commitment to collaborate and grow this therapy's unique position to treat AVNRT patients demonstrates our confidence in the proven safety and efficacy of our cryoablation technology."
Medtronic has pioneered cryoablation technology, with an industry-leading and extensive body of evidence, including proven safety and efficacy in treating AF and AVNRT. To date, more than one million patients have been treated with Medtronic cryoablation therapy worldwide.
In collaboration with leading clinicians, researchers, and scientists worldwide, Medtronic offers the broadest range of innovative medical technology for the interventional and surgical treatment of cardiovascular disease and cardiac arrhythmias. The company strives to offer products and services of the highest quality that deliver clinical and economic value to healthcare consumers and providers around the world.
For more information on Medtronic (NYSE:MDT), visit www.Medtronic.com(opens new window) and follow @Medtronic(opens new window) on Twitter and LinkedIn(opens new window).
SOURCE Medtronic plc
Medtronic announces FDA approval of Freezor cardiac catheters for use in children
Feb. 18, 2022 12:16 PM ET Medtronic plc (MDT)
By: Dulan Lokuwithana, SA News Editor
- Medtronic (NYSE:MDT) announced on Friday that the U.S. Food and Drug Administration (FDA) approved Freezor and Freezor Xtra Cardiac Cryoablation Catheters for pediatric use in a common heart condition called Atrioventricular Nodal Reentrant Tachycardia (AVNRT).
- https://seekingalpha.com/news/3802015-medtronic-wins-fda-approval-for-freezor-cardiac-catheters-in-children
- https://seekingalpha.com/symbol/MDT
- https://www.medtronic.com/us-en/healthcare-professionals/products/cardiac-rhythm/ablation-atrial-fibrillation/freezor-max-cardiac-cryoablation-catheter.html
- https://www.medtronic.com/us-en/index.html
biotecMAX™ Jan/Feb 2022 Insight
Opdivo® (nivolumab) in Combination with CABOMETYX® (cabozantinib)
Opdivo® (nivolumab) in Combination with CABOMETYX® (cabozantinib) Demonstrates Continued Survival Benefits with Over Two Years of Follow-Up in the CheckMate -9ER Trial in First-Line Advanced Renal Cell Carcinoma
Opdivo® (nivolumab) in Combination with CABOMETYX® (cabozantinib) Demonstrates Continued Survival Benefits with Over Two Years of Follow-Up in the CheckMate -9ER Trial in First-Line Advanced Renal Cell CarcinomaPDF Version
- Updated results to be presented at ASCO GU 2022 show sustained efficacy benefits with Opdivo in combination with CABOMETYX compared to sunitinib
- Patients treated with Opdivo and CABOMETYX continue to report improvements in health-related quality of life in a separate analysis
PRINCETON, N.J. & ALAMEDA, Calif.--(BUSINESS WIRE)--Today Bristol Myers Squibb (NYSE: BMY) and Exelixis, Inc. (NASDAQ: EXEL) announced two-year (25.4 months minimum; 32.9 months median) follow-up results from analyses of the Phase 3 CheckMate -9ER trial, demonstrating sustained survival and response rate benefits, as well as health-related quality of life (HRQoL) improvements, with the combination of Opdivo® (nivolumab) and CABOMETYX® (cabozantinib) versus sunitinib in the first-line treatment of advanced renal cell carcinoma (RCC). These updated results will be featured in two poster presentations at the American Society of Clinical Oncology (ASCO) 2022 Genitourinary Cancers Symposium from February 17-19, 2022.
“The new data from CheckMate -9ER evaluating nivolumab and cabozantinib are significant for patients with first-line advanced renal cell carcinoma, as they provide further evidence of efficacy benefits as well as favorable patient-reported quality of life outcomes with this combination,” said Toni Choueiri, M.D., Director of the Lank Center for Genitourinary Oncology at Dana-Farber Cancer Institute and Jerome and Nancy Kohlberg Professor of Medicine at Harvard Medical School. “As clinicians, we are constantly looking for therapies that can help more patients control their disease without reporting a detriment in their quality of life.”
Abstract #350: Final overall survival analysis and organ-specific target lesion assessments with 2-year follow-up in CheckMate 9ER: nivolumab plus cabozantinib vs sunitinib for patients with advanced renal cell carcinoma (Powles, et. al.)
With median follow-up of 32.9 months (25.4 months minimum), Opdivo in combination with CABOMETYX (n=323) continued to show superior overall survival (OS), progression-free survival (PFS), objective response rates (ORR), duration of response (DoR) and complete response (CR) rates compared to sunitinib (n=328). No new safety signals emerged with extended follow-up. In the full study population:
- OS: At the final OS analysis, Opdivo in combination with CABOMETYX continued to show meaningful improvements in median OS (37.7 months vs. 34.3 months) and demonstrated a 30% reduction in the risk of death (Hazard Ratio [HR] 0.70; 95% Confidence Interval [CI]: 0.55 to 0.90) compared to sunitinib.
- PFS: PFS benefits were maintained, with the combination continuing to double median PFS vs. sunitinib (16.6 months vs. 8.3 months, respectively; HR 0.56; 95% CI: 0.46 to 0.68).
- ORR and DoR: ORR benefits were sustained, with nearly twice as many patients responding to Opdivo in combination with CABOMETYX vs. sunitinib (55.7% vs. 28.4%). Responses were also more durable with the combination, with a median DoR of 23.1 months, compared to 15.1 months with sunitinib.
- CR: CR rates more than doubled among patients treated with the combination, with 12.4% having a CR vs. 5.2% of those treated with sunitinib.
- Safety: 97.2% of patients treated with Opdivo plus CABOMETYX (n=320) experienced a treatment-related adverse event (TRAE) of any grade, compared to 93.1% of patients treated with sunitinib (n=320); 65.0% vs. 54.1% had a grade ≥3 TRAE, respectively.
Additionally, in an exploratory analysis of depth of response in target lesions by organ site, a higher percentage of patients experienced any tumor shrinkage benefits with Opdivo in combination with CABOMETYX vs. sunitinib across lung (90.5% vs. 76.0%), lymph node (88.4% vs. 72.6%), kidney (89.0% vs. 71.6%), liver (72.7% vs. 53.8%) and bone (85.2% vs. 65.0%) target lesions.
Abstract #323: Health-related quality of life (HRQoL) in previously untreated patients with advanced renal cell carcinoma (aRCC): CheckMate 9ER updated results. (Cella, et. al.)
In a separate analysis with 32.9 months median follow-up from the CheckMate -9ER trial, patients continued to report clinically meaningful HRQoL benefits with Opdivo in combination with CABOMETYX compared to sunitinib. HRQoL scores were improved or maintained over time amongst patients treated with the combination, while reductions in scores were observed with sunitinib. Additionally, those who received Opdivo in combination with CABOMETYX were 48% less likely to be notably bothered by treatment side effects than patients in the sunitinib arm. These exploratory outcomes were measured using Functional Assessment of Cancer Therapy Kidney Symptom Index-19 (FKSI-19), a quality of life tool specific to kidney cancer, and EQ-5D-3L instruments.
“The results from these analyses of CheckMate -9ER provide additional clinical evidence supporting Opdivo in combination with CABOMETYX as an important first-line treatment for patients with advanced renal cell carcinoma who may benefit from an immunotherapy plus tyrosine kinase inhibitor regimen,” said Dana Walker, M.D., M.S.C.E., vice president, development program lead, genitourinary cancers, Bristol Myers Squibb. “These results exemplify the collaborative nature of our approach to research and development and demonstrate how we’re working across the healthcare landscape to explore how our therapies may work with potentially complementary mechanisms of action to help more patients achieve better and longer-lasting outcomes.”
“We are pleased that these additional findings from CheckMate -9ER showing continued superior efficacy and improved quality of life with longer follow-up are being presented at ASCO GU, as they further indicate the value of CABOMETYX in combination with Opdivo as a first-line option for patients with advanced renal cell carcinoma,” said Vicki L. Goodman, M.D., Executive Vice President, Product Development and Medical Affairs, and Chief Medical Officer, Exelixis. “The positive data from CheckMate -9ER, which are a culmination of a robust collaborative effort with Bristol Myers Squibb, reinforce our commitment to advancing additional CABOMETYX-based regimens in our continuing journey to identify treatments for people with difficult-to-treat cancers.”
Bristol Myers Squibb and Exelixis thank the patients and investigators involved in the CheckMate -9ER clinical trial.
About CheckMate -9ER
CheckMate -9ER is an open-label, randomized, multi-national Phase 3 trial evaluating patients with previously untreated advanced or metastatic renal cell carcinoma (RCC). A total of 651 patients (23% favorable risk, 58% intermediate risk, 20% poor risk; 25% PD-L1≥1%) were randomized to receive Opdivo plus CABOMETYX (n=323) vs. sunitinib (n=328). The primary endpoint is progression-free survival (PFS). Secondary endpoints include overall survival (OS) and objective response rate (ORR). The primary efficacy analysis is comparing the doublet combination vs. sunitinib in all randomized patients. The trial is sponsored by Bristol Myers Squibb and Ono Pharmaceutical Co and co-funded by Exelixis, Inc., Ipsen and Takeda Pharmaceutical Company Limited.
About OPDIVO®
Opdivo is a programmed death-1 (PD-1) immune checkpoint inhibitor that is designed to uniquely harness the body’s own immune system to help restore anti-tumor immune response. By harnessing the body’s own immune system to fight cancer, Opdivo has become an important treatment option across multiple cancers.
Opdivo’s leading global development program is based on Bristol Myers Squibb’s scientific expertise in the field of Immuno-Oncology and includes a broad range of clinical trials across all phases, including Phase 3, in a variety of tumor types. To date, the Opdivo clinical development program has treated more than 35,000 patients. The Opdivo trials have contributed to gaining a deeper understanding of the potential role of biomarkers in patient care, particularly regarding how patients may benefit from Opdivo across the continuum of PD-L1 expression.
In July 2014, Opdivo was the first PD-1 immune checkpoint inhibitor to receive regulatory approval anywhere in the world. Opdivo is currently approved in more than 65 countries, including the United States, the European Union, Japan and China. In October 2015, the Company’s Opdivo and Yervoy combination regimen was the first Immuno-Oncology combination to receive regulatory approval for the treatment of metastatic melanoma and is currently approved in more than 50 countries, including the United States and the European Union.
OPDIVO INDICATIONS
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express PD-L1 (≥1%) as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab) and 2 cycles of platinum-doublet chemotherapy, is indicated for the first-line treatment of adult patients with metastatic or recurrent non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab) is indicated for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving OPDIVO.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with unresectable malignant pleural mesothelioma (MPM).
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of patients with intermediate or poor risk advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab), in combination with cabozantinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab) is indicated for the treatment of patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy.
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with classical Hodgkin lymphoma (cHL) that has relapsed or progressed after autologous hematopoietic stem cell transplantation (HSCT) and brentuximab vedotin or after 3 or more lines of systemic therapy that includes autologous HSCT. This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab) is indicated for the treatment of patients with recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN) with disease progression on or after platinum-based therapy.
OPDIVO® (nivolumab) is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
OPDIVO® (nivolumab), as a single agent, is indicated for the adjuvant treatment of patients with urothelial carcinoma (UC) who are at high risk of recurrence after undergoing radical resection of UC.
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of adult and pediatric (12 years and older) patients with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
OPDIVO® (nivolumab) is indicated for the treatment of patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) after prior fluoropyrimidine- and platinum-based chemotherapy.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of completely resected esophageal or gastroesophageal junction cancer with residual pathologic disease in patients who have received neoadjuvant chemoradiotherapy (CRT).
OPDIVO® (nivolumab), in combination with fluoropyrimidine- and platinum- containing chemotherapy, is indicated for the treatment of patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma.
About CABOMETYX® (cabozantinib)
In the U.S., CABOMETYX tablets are approved for the treatment of patients with advanced RCC; for the treatment of patients with hepatocellular carcinoma who have been previously treated with sorafenib; for patients with advanced RCC as a first-line treatment in combination with nivolumab; and for adult and pediatric patients 12 years of age and older with locally advanced or metastatic differentiated thyroid cancer that has progressed following prior VEGFR-targeted therapy and who are radioactive iodine-refractory or ineligible. CABOMETYX tablets have also received regulatory approvals in the European Union and additional countries and regions worldwide. In 2016, Exelixis granted Ipsen exclusive rights for the commercialization and further clinical development of cabozantinib outside of the U.S. and Japan. In 2017, Exelixis granted exclusive rights to Takeda Pharmaceutical Company Limited for the commercialization and further clinical development of cabozantinib for all future indications in Japan. Exelixis holds the exclusive rights to develop and commercialize cabozantinib in the U.S.
https://www.cabometyx.com/kidney-cancer/cabometyx-opdivo
For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.
For more information about Exelixis, please visit www.exelixis.com, follow @ExelixisInc on Twitter or like Exelixis, Inc. on Facebook.
Exelixis, the Exelixis logo, CABOMETYX and COMETRIQ are registered U.S. trademarks of Exelixis.
COTELLIC is a registered trademark of Genentech, Inc.
MINNEBRO is a registered trademark of Daiichi Sankyo Company, Limited.
Exelixis/Bristol-Myers' drug combo shows sustained survival benefits in kidney cancer
Feb. 15, 2022 6:36 AM ET
Ipsen S.A. (IPSEY), IPSEFEXEL, BMY, TAK
By: Ravikash, SA News Editor3 Comments
Ipsen (OTCPK:IPSEF) said two-year follow-up data from a phase 3 CheckMate -9ER trial, showed sustained survival benefits, and health related quality of life (HRQoL) improvements with the combination of Exelixis (NASDAQ:EXEL) Cabometyx (cabozantinib) and Bristol-Myers Squibb's (NYSE:BMY) Opdivo (nivolumab) against sunitinib as first-line treatment of advanced renal cell carcinoma (aRCC).
https://seekingalpha.com/symbol/EXEL
https://seekingalpha.com/symbol/BMY
https://www.cabometyx.com/kidney-cancer/cabometyx-opdivo
Breyanzi (lisocabtagene maraleucel)
U.S. Food and Drug Administration (FDA) Accepts for Priority Review Bristol Myers Squibb’s Supplemental Biologics License Application for Breyanzi (lisocabtagene maraleucel) as a Second-Line Therapy for Relapsed or Refractory Large B-cell Lymphoma
02/17/2022CATEGORY:
Application based on the pivotal Phase 3 TRANSFORM study in which Breyanzi showed significant and clinically meaningful improvements over salvage chemotherapy followed by high-dose chemotherapy plus autologous stem cell transplant, which has been the standard of care for more than 20 years
U.S. FDA has assigned a target action date of June 24, 2022
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental Biologics License Application (sBLA) for Breyanzi (lisocabtagene maraleucel), a CD19-directed chimeric antigen receptor (CAR) T cell therapy, to expand its current indication to include earlier use of Breyanzi for the treatment of adults with relapsed or refractory large B-cell lymphoma (LBCL) after failure of first-line therapy. The FDA has granted the application Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) goal date of June 24, 2022.
“Breyanzi as a differentiated CD19-directed CAR T cell therapyhas already proven to be an important treatment option for patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy and nowhas the potential to be a new standard of care for patients after failure of first-line therapy, offering significantly improved outcomes beyond the current mainstay of care,” said Anne Kerber, senior vice president, Cell Therapy Development, Bristol Myers Squibb. “This acceptance from the FDA brings us one step closer to delivering a practice-changing treatment for primary refractory or relapsed large B-cell lymphoma, making Breyanzi available to more patients in need, and underscores the advancements we’re making in cell therapy research to transform the lives of patients with difficult-to-treat blood cancers, including lymphoma.”
The sBLA is based on results from the Phase 3 TRANSFORM trial, a global, randomized, multicenter study evaluating Breyanzi as a second-line treatment in adults with relapsed or refractory LBCL compared to the standard of care consisting of salvage chemotherapy followed by high-dose chemotherapy plus autologous hematopoietic stem cell transplant. Results showed Breyanzi provided highly statistically significant and clinically meaningful improvements in event-free survival, complete responses and progression-free survival, and a positive trend in overall survival in patients with LBCL whose disease was primary refractory or relapsed within 12 months after first-line therapy compared to standard of care. Results were presented at the American Society of Hematology (ASH) Annual Meeting and Exposition in December 2021.
About TRANSFORM
TRANSFORM (NCT03575351) is a pivotal, global, randomized, multicenter Phase 3 trial evaluating Breyanzi compared to current standard of care regimens in adults with large B-cell lymphoma that is primary refractory or relapsed within 12 months after first-line therapy and who are intended for stem cell transplant. Patients were randomized to receive Breyanzi or standard of care salvage chemotherapy, including rituximab plus dexamethasone, high-dose cytarabine, and cisplatin (R-DHAP), rituximab plus ifosfamide, carboplatin and etoposide (R-ICE), or rituximab plus gemcitabine, dexamethasone and cisplatin (R-GDP) per the investigators’ choice before proceeding to high-dose chemotherapy (HDCT) and hematopoietic stem cell transplant (HSCT). The primary endpoint of the study is event-free survival, defined as time from randomization to death from any cause, progressive disease, failure to achieve complete response or partial response, or start of new antineoplastic therapy due to efficacy concerns, whichever occurs first. Key secondary endpoints include complete response rate, progression-free survival, and overall survival. Overall response rate and duration of response are additional secondary endpoints.
About Breyanzi
Breyanzi is a CD-19 directed chimeric antigen receptor (CAR) T cell therapy with a defined and purified composition and 4-1BB costimulatory domain. Breyanzi is administered as a defined composition to reduce variability of the CD8 and CD4 component dose. The 4-1BB signaling domain enhances the expansion and persistence of the CAR T cells. Breyanzi is approved by the U.S. Food and Drug Administration for the treatment of adult patients with relapsed or refractory LBCL after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified (including DLBCL arising from indolent lymphoma), high-grade B-cell lymphoma, primary mediastinal large B-cell lymphoma, and follicular lymphoma grade 3B. Breyanzi is not indicated for the treatment of patients with primary central nervous system lymphoma. The U.S. Prescribing Information for Breyanzi has a BOXED WARNING for the risks of cytokine release syndrome (CRS) and neurologic toxicities (NT). Breyanzi is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the BREYANZI REMS.
Breyanzi is also approved in Japan for relapsed and refractory LBCL after two or more lines of systemic therapy, and Marketing Authorization Applications for Breyanzi for this indicationare currently under review in the European Union, Switzerland and Canada. Bristol Myers Squibb’s clinical development program for Breyanzi includes clinical studies in earlier lines of treatment for patients with relapsed or refractory LBCL and other types of lymphoma. For more information, visit clinicaltrials.gov.
Further information is available at www.BreyanziREMS.com, or contact Bristol Myers Squibb at 1-888-423-5436.
Please see full Prescribing Information, including Boxed WARNINGS and Medication Guide.
For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220215006166/en/
Source: Bristol Myers Squibb
Bristol Myers' Breyanzi gets FDA priority review for expanded use in blood cancer subtype
Feb. 18, 2022 5:08 AM ET
Bristol-Myers Squibb Company (BMY)
By: Ravikash, SA News Editor4 Comments
The U.S. Food and Drug Administration (FDA) granted priority review for expanded use of Bristol Myers Squibb's (NYSE:BMY) Breyanzi as a second line treatment of adults with relapsed or refractory large B-cell lymphoma (LBCL) after failure of first-line therapy.
- https://seekingalpha.com/news/3801835-bristol-myers-breyanzi-gets-fda-priority-review-for-expanded-use-in-blood-cancer-subtype
- https://seekingalpha.com/symbol/BMY
- https://www.bms.com/
- https://www.breyanzi.com/
Repotrectinib
Feb 17, 2022
Zai Lab Announces Breakthrough Therapy Designation Granted for Repotrectinib in China
PDF Version--- Potential best-in-class therapy for ROS1-positive metastatic NSCLC and advanced solid tumors
SHANGHAI and SAN FRANCISCO and CAMBRIDGE, Mass., Feb. 17, 2022 (GLOBE NEWSWIRE) -- Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688), a patient-focused, innovative, commercial-stage, global biopharmaceutical company, today announced that the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) granted Breakthrough Therapy Designation for investigational repotrectinib for the treatment of patients with ROS1-positive metastatic non-small-cell lung cancer (NSCLC) who have not been treated with a ROS1 tyrosine kinase inhibitor (TKI). The breakthrough therapy designation for repotrectinib was supported by the initial data from both global and Chinese TKI-naïve ROS1-positive NSCLC patients enrolled in the Phase 1/2 TRIDENT-1 study.
“In granting Breakthrough Therapy Designation, we are pleased to see that the CDE recognizes the promise of repotrectinib,” said Alan Sandler, M.D., President and Head of Global Development, Oncology, at Zai Lab. “We believe repotrectinib has the potential to be the best-in-class treatment for patients with ROS1-positive NSCLC, including patients who are either TKI-naïve or TKI-pretreated. We look forward to working with regulatory authorities in China to bring this important medicine to patients in need as soon as possible.”
The Breakthrough Therapy Designation review policy is designed to facilitate the development and expeditious review of novel medicines that are intended for the prevention or treatment of serious, life-threatening diseases or diseases that severely impact the quality of life for which there is no existing treatment, or where sufficient evidence indicates advantages of the novel drug over currently available treatment options. Drugs granted the Breakthrough Therapy Designation receive priority communications and guidance from the CDE to promote and expedite the drug development progress.
Lung cancer is both the most commonly diagnosed cancer type and the leading cause of cancer death in China. The incidence of lung cancer in China in 2020 was 815,563 cases, with 714,699 deaths.1 NSCLC accounts for approximately 85% of lung cancer, and about 70% of NSCLC is locally advanced or metastatic at initial diagnosis. In China, ROS1 rearrangements occur in 2-3% of patients with advanced NSCLC.
1Globocan 2020.
About Repotrectinib
Repotrectinib is a next-generation kinase inhibitor targeting the ROS1 and NTRK oncogenic drivers of NSCLC and advanced solid tumors. Tumors with mutations to their ROS1 and NTRK genes have a higher likelihood of developing resistance to existing targeted therapies. In many cases, these mutations prevent existing medicine from targeting and binding to the tumor as effectively as tumors that do not carry the mutations. Repotrectinib is designed to be smaller and less bulky than existing targeted therapies and may circumvent some of the resistance mechanisms found in tumors with ROS1 and NTRK mutations. Zai Lab and Turning Point Therapeutics are studying repotrectinib in TRIDENT-1, a registrational Phase 1/2 study in adults, and CARE, a Phase 1/2 study in pediatric patients. The compound has shown antitumor activity and durable responses among kinase inhibitor treatment-naïve and pre-treated patients. Zai Lab is enrolling patients in the registrational Phase 2 portion of TRIDENT-1 in Greater China, while Turning Point Therapeutics is enrolling patients in other regions of the world.
The U.S. Food and Drug Administration has granted repotrectinib breakthrough therapy designation both for the indication described above as well as for the treatment of patients with advanced solid tumors that have an NTRK gene fusion who have progressed following treatment with one or two prior TRK tyrosine kinase inhibitors, with or without prior chemotherapy, and have no satisfactory alternative treatments. Additionally, repotrectinib was previously granted four Fast-Track designations in ROS1-positive advanced NSCLC patients who are ROS1 TKI naïve; ROS1-positive advanced NSCLC patients who have been previously treated with one prior line of platinum-based chemotherapy and one prior ROS1 TKI; ROS1-positive advanced NSCLC patients pretreated with one prior ROS1 TKI without prior platinum-based chemotherapy; and NTRK-positive patients with advanced solid tumors who have progressed following treatment with at least one prior line of chemotherapy and one or two prior TRK TKIs and have no satisfactory alternative treatments. Repotrectinib was also granted an Orphan Drug designation in 2017.
Zai Lab has an exclusive license to develop and commercialize repotrectinib in Greater China.
For additional information about the company, please visit www.zailaboratory.com or follow us at www.twitter.com/ZaiLab_Global.
Source: Zai Lab Limited
Zai Lab's repotrectinib for lung cancer subtype gets breakthrough therapy tag in China
Feb. 17, 2022 8:25 AM ET Zai Lab Limited (ZLAB)
By: Ravikash, SA News Editor
- Zai Lab (NASDAQ:ZLAB) said the Center for Drug Evaluation of China's National Medical Products Administration granted breakthrough therapy designation to repotrectinib for treating patients with ROS1-positive metastatic non-small-cell lung cancer (NSCLC) who have not been treated with a ROS1 tyrosine kinase inhibitor (TKI).
- https://seekingalpha.com/news/3801393-zai-labs-repotrectinib-for-lung-cancer-subtype-gets-breakthrough-therapy-tag-in-china
- https://seekingalpha.com/symbol/ZLAB
- https://www.zailaboratory.com/
- https://www.tptherapeutics.com/corporate/pipeline/repotrectinib
BRUKINSA (zanubrutinib)
BeiGene Announces Approval for BRUKINSA (zanubrutinib) by Swissmedic for Treatment of Adult Patients with Waldenström’s Macroglobulinemia
Feb 17, 2022 5:00 AM
With this approval, BRUKINSA is now approved in 44 markets as BeiGene continues to advance its global registration, including the EU, US, and Great Britain
BASEL, Switzerland & CAMBRIDGE, Mass. & BEIJING--(BUSINESS WIRE)-- BeiGene (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global, science-driven biotechnology company focused on developing innovative and affordable medicines, today announced that BeiGene’s BTK inhibitor BRUKINSA (zanubrutinib) received approval from Swissmedic for the treatment of adult patients with Waldenström’s macroglobulinemia (WM) who have received at least one prior line of therapy, or for treatment-naïve patients who are not suited for standard chemo-immunotherapy. BRUKINSA had previously been granted orphan drug status.
“The authorization of BRUKINSA will bring a new option and an innovative medicine that has potential to offer deep and durable response for eligible patients with WM in Switzerland," said Pr. Davide Rossi, Deputy Head of the Division of Hematology of the Oncology Institute of Southern Switzerland IOSI. “BRUKINSA is a next-generation BTK inhibitor which has also provided meaningful improvements in tolerability for some patients with WM compared to ibrutinib, as treatment discontinuation remains a concern.”
Reto Kessler, Country Manager, Switzerland at BeiGene added, “This approval is a significant development for people living with WM in Switzerland and for BeiGene’s expansion in Europe. Our teams are committed to collaborating with the Federal Office of Public Health and healthcare professionals to ensure access to BRUKINSA for patients in Switzerland.”
The Marketing Authorization Application (MAA) is supported by data from the global Phase 3 ASPEN clinical trial, a Phase 3 randomized, open-label, multicenter trial (NCT03053440) that evaluated BRUKINSA compared to ibrutinib in patients with relapsed/refractory (R/R) or treatment-naïve (TN) WM who harbor a MYD88 mutation (MYD88MUT). In the ASPEN trial, BRUKINSA demonstrated a numerically higher very good partial response (VGPR) rate and a favorable safety profile over ibrutinib, although the primary endpoint of statistical superiority related to deep response (VGPR or better) was not met. As assessed by independent review committee (IRC) per adaptation of the response criteria updated at the Sixth International Workshop on Waldenström’s Macroglobulinemia (IWWM), the combined complete response (CR) + VGPR rate in the overall intention-to-treat (ITT) population was 29% with BRUKINSA (95% CI: 20, 40), compared to 19% with ibrutinib (95% CI: 12, 30).
In the ASPEN trial, of the 101 patients with WM randomized and treated with BRUKINSA, four percent of patients discontinued due to adverse events, including cardiomegaly, neutropenia, plasma cell myeloma, and subdural hemorrhage. Adverse events leading to dose reduction occurred in 14% of patients, with the most common being neutropenia (3%) and diarrhea (2%).
The recommended dose of BRUKINSA is either 160 mg twice daily or 320 mg once daily, taken orally with or without food. The dose may be adjusted for adverse reactions and reduced for patients with severe hepatic impairment and certain drug interactions.
About Waldenström’s Macroglobulinemia
WM is a rare B-cell lymphoma that occurs in less than two percent of patients with non-Hodgkin lymphomas.2 The disease usually affects older adults and is primarily found in bone marrow, although lymph nodes and the spleen may be involved.1 Throughout Europe, the estimated incidence rate of WM is approximately seven for every one million men and four for every one million women.2
About BRUKINSA
BRUKINSA is a small molecule inhibitor of Bruton’s tyrosine kinase (BTK) discovered by BeiGene scientists that is currently being evaluated globally in a broad clinical program as a monotherapy and in combination with other therapies to treat various B-cell malignancies. Because new BTK is continuously synthesized, BRUKINSA was specifically designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity. With differentiated pharmacokinetics compared to other approved BTK inhibitors, BRUKINSA has been demonstrated to inhibit the proliferation of malignant B cells within a number of disease relevant tissues.
BRUKINSA is supported by a broad clinical program which includes more than 3,900 subjects in 35 trials across 28 markets. To date, BRUKINSA has received more than 20 approvals covering more than 40 countries and regions, including the U.S., European Union, China, Australia, Great Britain and Switzerland. Currently, more than 40 additional regulatory submissions are in review around the world.
To learn more about BeiGene, please visit www.beigene.com and follow us on Twitter at @BeiGeneGlobal.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220217005344/en/
Source: BeiGene
BeiGene's Brukinsa for blood cancer subtype gets approval in Switzerland
Feb. 17, 2022 5:55 AM ET BeiGene, Ltd. (BGNE)
By: Ravikash, SA News Editor
BeiGene's (NASDAQ:BGNE) Brukinsa (zanubrutinib) was approved by Swissmedic to treat adult patients with Waldenström’s macroglobulinemia (WM) who have received at least one prior line of therapy or for patients who have not received treatment and are not suited for standard chemo-immunotherapy.
- https://seekingalpha.com/news/3801258-beigenes-brukinsa-for-blood-cancer-subtype-gets-approval-in-switzerland
- https://seekingalpha.com/symbol/BGNE
- https://www.brukinsa.com/
- https://www.beigene.com/
Lenacapavir
February 16, 2022
New Clinical Data Support the Sustained Efficacy of Long-acting Lenacapavir, Gilead’s Investigational HIV-1 Capsid Inhibitor
– One-Year Data From the CAPELLA and CALIBRATE Trials Show Lenacapavir Leads to High Rates of Virologic Suppression in Heavily Treatment-Experienced People Living with Multi-Drug Resistant HIV and Treatment-Naïve People Living with HIV –
FOSTER CITY, Calif.--(BUSINESS WIRE)-- Gilead Sciences, Inc. (Nasdaq: GILD) today announced new one-year results from the ongoing Phase 2/3 CAPELLA trial evaluating lenacapavir, the company’s investigational, long-acting HIV-1 capsid inhibitor, in heavily treatment-experienced people living with multi-drug resistant HIV. The findings demonstrated that lenacapavir, administered subcutaneously every six months in combination with other antiretrovirals, achieved high rates of virologic suppression and clinically meaningful increases in CD4 counts in people living with HIV whose virus was no longer effectively responding to their current therapy. In this patient population with high unmet medical need, 83% (n=30/36) of participants receiving lenacapavir in combination with an optimized background regimen achieved an undetectable viral load (<50 copies/mL) at Week 52. The data were presented at the 29th Conference on Retroviruses and Opportunistic Infections (virtual CROI 2022).
“I am really encouraged by the results presented today showing that the positive outcomes achieved with lenacapavir can be sustained at one year of treatment, which is a remarkable achievement for this group of people living with HIV who have limited treatment options and are at a greater risk of progressing to AIDS,” said Onyema Ogbuagu, MD, FACP, Director of HIV Clinical Trials program at Yale School of Medicine. “The potential of a long-acting antiretroviral treatment option that may achieve and maintain an undetectable viral load and that is administered only twice a year would be a true advancement that could potentially transform how providers care for certain patients with the virus.”
Lenacapavir is Gilead’s potential first-in-class, investigational long-acting HIV-1 capsid inhibitor in development for the treatment and prevention of HIV-1 infection. Lenacapavir's multi-stage mechanism of action is distinguishable from currently approved classes of antiviral agents and could provide a new avenue for the development of long-acting therapy options for people living with or at risk for HIV-1. While most antiretroviral agents act on just one stage of viral replication, lenacapavir inhibits HIV-1 at multiple stages of its lifecycle and has no known cross resistance to other existing drug classes. If approved, lenacapavir would be the only HIV-1 treatment option administered twice yearly.
“Continued scientific innovation is essential to helping end the global HIV epidemic. Gilead is committed to driving advances in HIV treatment with the goal of offering long-acting options that address the differentiated needs and preferences of a diverse range of individuals and communities impacted by this disease,” said Jared Baeten, MD, PhD, Vice President, HIV Clinical Development, Gilead Sciences. “These latest results provide further evidence of the potential for lenacapavir, as a breakthrough innovation, to fulfill the needs of heavily treatment-experienced people living with multi-drug resistant HIV, irrespective of their prior treatment history.”
In addition to high rates of viral suppression, participants in CAPELLA achieved a mean increase in CD4 count of 83 cells/µL. Data previously presented at virtual CROI 2021, showed that the CAPELLA trial achieved its primary endpoint by demonstrating that a significantly higher proportion of participants randomly allocated to receive lenacapavir (n=24) achieved a clinically meaningful viral load reduction of at least 0.5 log10 copies/mL from baseline compared with those randomly allocated to receive placebo (n=12) during the 14-day functional monotherapy period (88% vs. 17%, p<0.0001). Those who received lenacapavir achieved a statistically significant greater mean decrease in viral load than those who received placebo during the functional monotherapy period (-1.93 log10 copies/mL vs. -0.29 log10 copies/mL, p<0.0001).
Lenacapavir was generally well tolerated in CAPELLA, with one adverse event (AE) leading to study drug discontinuation at Week 52 and no serious adverse events related to lenacapavir. The most common adverse events observed to date in the CAPELLA study were injection site reactions (63%), which were mostly mild or moderate in severity. The most common adverse events, excluding injection site reactions, were nausea and diarrhea (13% each) and COVID-19 (11%).
Gilead presented additional lenacapavir clinical data from the Phase 2 CALIBRATE trial, an ongoing, open-label, active-controlled trial in treatment-naïve people with HIV-1 infection. The trial showed lenacapavir, given subcutaneously in combination with oral daily emtricitabine/tenofovir alafenamide (F/TAF) in the first 6 months, followed by combination with either oral daily tenofovir alafenamide (TAF) or bictegravir (BIC), or given orally in combination with emtricitabine/tenofovir alafenamide (F/TAF), achieved high rates of viral suppression by Week 54. Specifically, in the subcutaneous lenacapavir + TAF arm, 90% achieved an undetectable viral load (<50 copies/mL). In the subcutaneous lenacapavir + BIC arm, 85% achieved an undetectable viral load. In the oral lenacapavir + F/TAF arm, 85% achieved an undetectable viral load. Lenacapavir was generally well tolerated, with no study drug-related serious AEs. The most common AEs observed were injection site reactions (ISRs), which were generally mild or moderate in severity. The most frequent non-ISR AEs were headache and nausea (13% each) and COVID-19 (10%).
These results support the ongoing evaluation and further development of lenacapavir in combination with other long-acting partner agents for the treatment of HIV-1 infection and support Gilead’s long-acting oral and injectable development program.
Lenacapavir is an investigational compound and is not approved by any regulatory authority for any use and its safety and efficacy are not established. There is no cure for HIV or AIDS.
About CAPELLA (NCT04150068)
CAPELLA is a Phase 2/3, double-blinded, placebo-controlled global multicenter study designed to evaluate the antiviral activity of Gilead’s investigational, long-acting HIV-1 capsid inhibitor lenacapavir administered every six months as a subcutaneous injection in heavily treatment-experienced people with multi-drug resistant HIV-1 infection. CAPELLA includes men and women living with HIV-1 and is being conducted at research centers in North America, Europe and Asia.
In CAPELLA, 36 participants with multi-class HIV-1 drug resistance and a detectable viral load while on a failing regimen were randomly allocated to receive oral lenacapavir or placebo in a 2:1 ratio for 14 days, in addition to continuing their failing regimen (functional monotherapy). An additional 36 participants were enrolled in a separate treatment cohort. Both cohorts are part of the ongoing maintenance period of the study evaluating the safety and efficacy of subcutaneous lenacapavir administered every six months in combination with an optimized background regimen. The primary endpoint was the proportion of participants randomly allocated to receive lenacapavir or placebo for 14 days, in addition to continuing their failing regimen, achieving ≥0.5 log10 copies/mL reduction from baseline in HIV-1 RNA at the end of the functional monotherapy period.
Following the 14-day functional monotherapy period, participants randomly allocated to receive lenacapavir or placebo, in addition to continuing their failing regimen, started open-label lenacapavir and an optimized background regimen, while those enrolled in a separate treatment cohort received open-label lenacapavir and an optimized background regimen on Day 1. This ongoing maintenance period of the study is evaluating the additional trial endpoints of safety and efficacy of subcutaneous lenacapavir administered every six months in combination with an optimized background regimen.
For further information, please see https://clinicaltrials.gov/ct2/show/NCT04150068.
About CALIBRATE (NCT04143594)
CALIBRATE is an ongoing, phase 2, open-label, active-controlled study in treatment-naïve people with HIV-1 infection designed to evaluate the efficacy and safety profile of lenacapavir-containing regimens. CALIBRATE includes men and women living with HIV-1 and is being conducted at research centers in North America, Puerto Rico and the Dominican Republic.
In CALIBRATE, 182 participants were randomly allocated (2:2:2:1) into one of the four treatment groups. The first and second groups received subcutaneous lenacapavir every 26 weeks following an oral lead-in period together with oral daily F/TAF; at Week 28, those achieving HIV-1 RNA viral load <50 copies/mL switched their F/TAF to oral daily TAF or BIC, while continuing lenacapavir. The third group received oral daily lenacapavir with F/TAF. The fourth group received oral daily B/F/TAF. The primary endpoint of the study is the proportion of participants achieving a viral load of <50 c/mL at Week 54.
For further information, please see https://clinicaltrials.gov/ct2/show/NCT04143594.
GILEAD and the GILEAD logo are trademarks of Gilead Sciences, Inc. All other trademarks are the property of their respective owners.
For more information about Gilead, please visit the company’s website at www.gilead.com , follow Gilead on Twitter (@Gilead Sciences) or call Gilead Public Affairs at 1-800-GILEAD-5 or 1-650-574-3000.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220215006286/en/
Source: Gilead Sciences, Inc.
Gilead's lenacapavir demonstrates long-term efficacy against HIV
Feb. 16, 2022 2:13 PM ET Gilead Sciences, Inc. (GILD) By: Jonathan Block, SA News Editor2 Comments
- Data from a phase 3 study on Gilead Sciences' (GILD +0.8%) lenacapavir given subcutaneously every six months in combination with other antiretrovirals led to high rates of virologic suppression over one year for treatment of HIV.
- https://seekingalpha.com/news/3800932-gilead-sciences-lenacapavir-demonstrates-long-term-efficacy-against-hiv
- https://seekingalpha.com/symbol/GILD
- https://www.gilead.com/
Saphnelo (anifrolumab)
Saphnelo approved in the EU for the treatment of moderate to severe systemic lupus erythematosus
PUBLISHED16 February 2022
16 February 2022 07:00 GMT
Saphnelo is a first-in-class type I interferon receptor antibody and the only new medicine in over a decade for patients with systemic lupus erythematosus
AstraZeneca’s Saphnelo (anifrolumab) has been approved in the European Union as an add-on therapy for the treatment of adult patients with moderate to severe, active autoantibody-positive systemic lupus erythematosus (SLE), despite receiving standard therapy.
Saphnelo is the first biologic for SLE approved in Europe with an indication that is not restricted to patients with a high degree of disease activity. SLE is a serious and complex autoimmune condition that can affect any organ, and patients often experience inadequate disease control, long-term organ damage and poor health-related quality of life.1-3 There are approximately 250,000 people with SLE in Europe, and most are women who are diagnosed between the ages of 15 and 45.4
The approval by the European Commission was based on results from the Saphnelo clinical development programme, including the TULIP Phase III trials and the MUSE Phase II trial.5-7 Across clinical trials, more patients treated with Saphnelo experienced a reduction in overall disease activity across organ systems and achieved sustained reduction in oral corticosteroid (OCS) use compared to placebo.5-7 Minimising OCS use while reducing disease activity is an important treatment goal in SLE to reduce the risk of organ damage.8,9 The approval follows the recommendation by the Committee for Medicinal Products for Human Use of the European Medicines Agency in December 2021.
Ronald van Vollenhoven, Chair of Rheumatology and Director of the Amsterdam Rheumatology Center in Amsterdam, the Netherlands said, “In Europe, there have been limited treatment options for patients living with systemic lupus erythematosus and many patients face poor outcomes. Anifrolumab targets the type I interferon pathway, which is known to play a central role in lupus pathophysiology. Today’s approval is an important step forward in treating this disease.”
Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, AstraZeneca, said: “Saphnelo is the first new medicine for systemic lupus erythematosus to gain approval in Europe in over a decade and is the only biologic not restricted to patients with a high degree of disease activity. Saphnelo has demonstrated clinically meaningful benefits and we look forward to bringing it to patients as quickly as possible.”
The most frequent adverse reactions that occurred in patients who received Saphnelo in the controlled clinical trials included upper respiratory tract infection, bronchitis, infusion-related reactions and herpes zoster.5-7
Saphnelo was recently approved in the US, Japan and Canada for the treatment of SLE, and regulatory reviews are ongoing in additional countries. The Phase III trial in SLE using subcutaneous delivery has been initiated and additional Phase III trials are planned for lupus nephritis, cutaneous lupus erythematosus and myositis. 10,11
TULIP-1, TULIP-2 and MUSE
All three trials for Saphnelo (TULIP-1, TULIP-2 and MUSE) were randomised, double-blinded, placebo-controlled trials in patients with moderate to severe SLE who were receiving standard therapy.5-7 Standard therapy included at least one of the following: OCS, antimalarials and immunosuppressants (methotrexate, azathioprine or mycophenolate mofetil).5-7
The pivotal TULIP (Treatment of Uncontrolled Lupus via the Interferon Pathway) Phase III programme included two trials, TULIP-1 and TULIP-2, that evaluated the efficacy and safety of Saphnelo versus placebo.5,6 TULIP-2 demonstrated superiority across multiple efficacy endpoints versus placebo with both arms receiving standard therapy.5 In the trial, 362 eligible patients were randomised (1:1) and received a fixed-dose intravenous infusion of 300mg Saphnelo or placebo every four weeks.5 TULIP-2 assessed the effect of Saphnelo in reducing disease activity as measured by the BILAG-Based Composite Lupus Assessment (BICLA) scale.5 In TULIP-1, 457 eligible patients were randomised (1:2:2) and received a fixed-dose intravenous infusion of 150mg Saphnelo, 300mg Saphnelo or placebo every four weeks, in addition to standard therapy.6 The trial did not meet its primary endpoint based on the SLE Responder Index 4 (SRI4) composite measure.6
The MUSE Phase II trial evaluated the efficacy and safety of two doses of Saphnelo versus placebo.7 In MUSE, 305 adults were randomised and received a fixed-dose intravenous infusion of 300mg Saphnelo, 1,000mg Saphnelo or placebo every four weeks, in addition to standard therapy, for 48 weeks.7 The trial showed improvement versus placebo across multiple efficacy endpoints with both arms receiving standard therapy.7
Results from the TULIP-2 Phase III trial were published in The New England Journal of Medicine in January 2020, results from the TULIP-1 Phase III trial were published in The Lancet Rheumatology in December 2019 and results from the MUSE Phase II trial were published in Arthritis & Rheumatology in November 2016.
In SLE, along with the pivotal TULIP Phase III programme, Saphnelo continues to be evaluated in a long-term extension Phase III trial15 and a Phase III trial assessing subcutaneous delivery.10 In addition, AstraZeneca is exploring the potential of Saphnelo in a variety of diseases in which type I interferon (IFN) plays a key role, including lupus nephritis, cutaneous lupus erythematosus and myositis.11
Saphnelo
Saphnelo (anifrolumab) is a fully human monoclonal antibody that binds to subunit 1 of the type I IFN receptor, blocking the activity of type I IFN.7 Type I IFNs, such as IFN-alpha, IFN-beta and IFN-kappa, are cytokines involved in regulating the inflammatory pathways implicated in SLE.16-21 The majority of adults with SLE have increased type I IFN signalling, which is associated with increased disease activity and severity.16,22
Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca.
AstraZeneca's Saphnelo gets approval in EU for systemic lupus erythematosus
Feb. 16, 2022 5:18 AM ET
By: Ravikash, SA News Editor
AstraZeneca's (NASDAQ:AZN) Saphnelo was approved in the EU as an add-on therapy to treat adult patients with moderate to severe, active autoantibody-positive systemic lupus erythematosus (SLE), despite receiving standard therapy.
https://seekingalpha.com/symbol/AZN
Zuranolone 50 mg Co-initiated with Standard of Care Antidepressant
February 16, 2022
Sage Therapeutics and Biogen Announce the Phase 3 CORAL Study Met its Primary and Key Secondary Endpoints - Comparing Zuranolone 50 mg Co-initiated with Standard of Care Antidepressant vs. Standard of Care Co-initiated with Placebo in People with MDD
At the Day 3 primary endpoint, zuranolone 50 mg co-initiated with a standard of care antidepressant showed a statistically significant reduction in depressive symptoms
Key secondary endpoint demonstrates zuranolone co-initiated with an antidepressant was statistically significant in reducing depressive symptoms compared to an antidepressant co-initiated with placebo over the 2-week treatment period
Zuranolone 50 mg co-initiated with a standard of care antidepressant was generally well-tolerated with most TEAEs reported as mild or moderate and no new safety signals identified
Sage Therapeutics to host conference call today at 8:00 a.m. ET
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Feb. 16, 2022-- Sage Therapeutics, Inc. (Nasdaq: SAGE), and Biogen Inc. (Nasdaq: BIIB) today announced the CORAL Study in people with major depressive disorder (MDD) met the trial objectives, demonstrating a rapid and statistically significant reduction in depressive symptoms at Day 3 and over the 2-week treatment period, achieving the primary and key secondary endpoints. This significance was demonstrated at the first measured time point, Day 3, with zuranolone 50 mg co-initiated with an open-label standard of care antidepressant (ADT) as assessed by change from baseline in the 17-item Hamilton Rating Scale for Depression (HAMD-17). The CORAL Study also met its key secondary endpoint, with zuranolone co-initiated with a standard of care ADT demonstrating a statistically significant improvement in depressive symptoms compared to ADT co-initiated with placebo, over the 2-week treatment period. Zuranolone was generally well-tolerated, and no new safety signals attributable to zuranolone were identified. In meeting its pre-defined objectives, the CORAL Study supports the potential of zuranolone, when co-initiated with standard of care, to accelerate the benefit of depression treatment compared to treatment with ADTs alone.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20220215006261/en/
The CORAL Study was an active comparator trial comparing the combination of zuranolone 50 mg co-initiated with an active standard of care ADT to standard of care ADT co-initiated with placebo in people with MDD blinded to receipt of zuranolone or placebo. The trial demonstrated a mean change from baseline in HAMD-17 total score of -8.9 ± 0.39 (n=210) at Day 3 for people in the zuranolone co-initiated with ADT arm compared with -7.0 ± 0.38 (n=215) mean change from baseline for people in the ADT co-initiated with placebo arm. The key secondary endpoint measured the treatment effect over the 2-week treatment period at all scheduled visits (measured using equal weighted means for Days 3, 8, 12 and 15 of the study). The mean change over the treatment period for people who received zuranolone co-initiated with an ADT was -11.7 ±0.40 (n=210) compared with -10.1 ±0.39 (n=215) for people who received ADT co-initiated with placebo. Other secondary endpoints demonstrated a statistically significant reduction in HAMD-17 score in the zuranolone co-initiated with ADT arm compared to the ADT arm at Days 8 and 12, while Day 15 demonstrated numerical superiority and Day 42 showed equivalence.
Based on consistent findings suggesting a benefit of zuranolone in people with MDD with elevated anxiety across the LANDSCAPE program, the CORAL Study prospectively examined this population. In this CORAL Study subgroup (n=218 of 425 people (51.3%) with HAM-A total score ≥20 at baseline) zuranolone co-initiated with an ADT was nominally statistically significant to ADT with placebo in reducing depressive symptoms as measured by the primary endpoint (-9.3 compared to -6.0; HAMD-17 total score change from baseline) and key secondary endpoint (-11.7 compared to -9.4; HAMD-17 total score change from baseline) demonstrating the potential to address the unmet need for this population, which has been historically less responsive to chronically administered ADTs.
CORAL Study Summary Results
The CORAL Study was a Phase 3, randomized, double-blind, placebo-controlled trial, which enrolled 440 people with MDD (n=220 per treatment arm). People in the study received zuranolone 50 mg co-initiated with an open-label standard of care ADT or open-label standard of care ADT co-initiated with placebo once nightly for 14 days. Results for the primary and key secondary efficacy endpoints during the treatment period are outlined in the following table and all favor zuranolone. The mean (SD) baseline HAMD-17 score at entry into the study was 26.8 (2.5) in the zuranolone co-initiated with ADT arm and 26.6 (2.6) in the ADT co-initiated with placebo arm. 180 (84.9%) people in the study who received zuranolone co-initiated with ADT, and 177 (81.2%) people who received ADT co-initiated with placebo, completed the study.
CORAL Study Summary Results
The CORAL Study was a Phase 3, randomized, double-blind, placebo-controlled trial, which enrolled 440 people with MDD (n=220 per treatment arm). People in the study received zuranolone 50 mg co-initiated with an open-label standard of care ADT or open-label standard of care ADT co-initiated with placebo once nightly for 14 days. Results for the primary and key secondary efficacy endpoints during the treatment period are outlined in the following table and all favor zuranolone.
- The mean (SD) baseline HAMD-17 score at entry into the study was 26.8 (2.5) in the zuranolone co-initiated with ADT arm and 26.6 (2.6) in the ADT co-initiated with placebo arm.
- 180 (84.9%) people in the study who received zuranolone co-initiated with ADT, and 177 (81.2%) people who received ADT co-initiated with placebo, completed the study.
About the CORAL Study
The CORAL Study (217-MDD-305) was a Phase 3, randomized, double-blind, placebo-controlled trial assessing the efficacy and safety of zuranolone 50 mg co-initiated with an open-label standard of care antidepressant (ADT) compared to a standard of care ADT co-initiated with placebo in adults with MDD. In the study, 440 people were enrolled. People in the study were randomized to receive zuranolone 50 mg with a standard of care ADT or a standard of care ADT co-initiated with placebo once nightly for two weeks. People in the study were then followed for four weeks during which they continued their ADT. The study included five ADTs across different SSRI and SNRIs (sertraline, escitalopram, citalopram, duloxetine, desvenlafaxine) that represent the most commonly used ADTs. The choice of ADT was made by the clinician. The primary endpoint was the change in baseline 17-item Hamilton Rating Scale for Depression (HAMD-17) total score at Day 3.
About Major Depressive Disorder (MDD)
Major depressive disorder (MDD) is a common but serious mood disorder in which people experience depressive symptoms that impair their social, occupational, educational, or other important functioning, such as a depressed mood or loss of interest or pleasure in daily activities, consistently for at least a two-week period. It is estimated that more than 19 million adults in the U.S. and more than 260 million people worldwide suffer from depression. While antidepressants are widely used to treat MDD, large-scale studies have demonstrated the need for additional therapies with a differentiated profile.
About Zuranolone
Zuranolone (SAGE-217/BIIB125) is a once-daily, two-week, investigational drug in development for the treatment of major depressive disorder (MDD) and postpartum depression (PPD). Zuranolone is an investigational oral neuroactive steroid (NAS) GABA-A receptor positive allosteric modulator (PAM). The GABA system is the major inhibitory signaling pathway of the brain and central nervous system and contributes to regulating brain function.
Zuranolone is being evaluated in the LANDSCAPE and NEST clinical trial programs. The two development programs include multiple studies examining use of zuranolone in several thousand people with a variety of dosing, clinical endpoints, and treatment paradigms. The LANDSCAPE program includes five studies of zuranolone in people with MDD (MDD-201B, MOUNTAIN, SHORELINE, WATERFALL, and CORAL Studies). The NEST program includes two placebo-controlled studies of zuranolone in people with PPD (ROBIN and SKYLARK Studies). Additionally, Shionogi recently completed a Phase 2 study of zuranolone in Japan in people with MDD.
For more information, please visit. www.sagerx.com.
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View source version on businesswire.com: https://www.businesswire.com/news/home/20220215006261/en/
Source: Sage Therapeutics, Inc.
Sage, Biogen announce late-stage trial for depression therapy met key goals
Feb. 16, 2022 7:52 AM ET
Sage Therapeutics, Inc. (SAGE), BIIB
By: Dulan Lokuwithana, SA News Editor
Sage (NASDAQ:SAGE) and Biogen (NASDAQ:BIIB) announced that their Phase 3 CORAL study for zuranolone met primary and key secondary endpoints in adults with major depressive disorder (MDD). However, Sage (SAGE) shares are trading ~6% lower in the pre-market on Wednesday.
https://seekingalpha.com/news/3800651-sage-stock-lower-after-late-stage-data-for-depression-therapy
https://seekingalpha.com/symbol/SAGE
https://seekingalpha.com/symbol/BIIB
20-Valent Pneumococcal Conjugate Vaccine
European Medicines Agency Approves Pfizer’s 20-Valent Pneumococcal Conjugate Vaccine Against Invasive Pneumococcal Disease and Pneumonia in Adults
Tuesday, February 15, 2022 - 10:02am
APEXXNAR® [pneumococcal polysaccharide conjugate vaccine (20-valent, adsorbed)] is the first pneumococcal conjugate vaccine to help protect adults ages 18 years and older against 20 serotypes responsible for the majority of invasive disease and pneumococcal pneumonia
NEW YORK--(BUSINESS WIRE)-- Pfizer Inc. (NYSE:PFE) today announced that the European Medicines Agency (EMA) has approved the company’s 20-valent pneumococcal conjugate vaccine (PCV20), which will be marketed in the European Union (EU) under the brand name APEXXNAR. The vaccine is approved for active immunization for the prevention of invasive disease and pneumonia caused by Streptococcuspneumoniae in individuals 18 years of age and older.
“The EMA’s authorization of APEXXNAR for adults continues Pfizer’s ongoing commitment to help prevent certain potentially-serious infectious respiratory diseases, including invasive pneumococcal disease and pneumonia,” said Nanette Cocero, Ph.D., Global President of Pfizer Vaccines. “APEXXNAR helps protect against the 20 serotypes in the vaccine, and today’s approval offers adults -- through a single dose -- the broadest serotype protection of any available pneumococcal conjugate vaccine in Europe.”
Today’s authorization follows the recent positive opinion from the EMA's Committee for Medicinal Products for Human Use (CHMP) announced on December 17, 2021. The authorization is valid in all 27 EU member states plus Iceland, Lichtenstein, and Norway. The EMA had previously accepted review of Pfizer’s Marketing Authorization Application (MAA) for the 20-valent pneumococcal conjugate vaccine candidate in February 2021.
The EMA authorization for APEXXNAR is based on evidence from a clinical program in adults, including Phase 1 and 2 trials, and three Phase 3 trials (NCT03760146, NCT03828617, and NCT03835975) describing the safety and evaluating the immunogenicity of the vaccine. More than 6,000 adult subjects 18 years and older participated in the three Phase 3 trials, including adults 65 years of age and older. The populations included adults with stable chronic medical conditions, pneumococcal vaccine-naïve adults, and adults with a history of prior pneumococcal vaccination.
About PCV20
PCV20 – known as APEXXNAR in the EU and PREVNAR 20TM in the US -- is Pfizer’s next-generation pneumococcal conjugate vaccine that includes capsular polysaccharide conjugates for the 13 serotypes (1, 3, 4, 5, 6A, 6B, 7F, 9V, 14, 18C, 19A, 19F and 23F) already included in PREVENAR 13® (Pneumococcal 13-valent Conjugate Vaccine [Diphtheria CRM197 Protein]), known as PREVNAR 13® in the U.S. The vaccine also contains capsular polysaccharide conjugates for seven additional serotypes (8, 10A, 11A, 12F, 15B, 22F and 33F) that cause invasive pneumococcal disease (IPD),1,2,3,4,5 and have been associated with high case-fatality rates,6,7,8,9 antibiotic resistance,10,11,12 and/or meningitis.13,14 APEXXNAR contains the broadest serotype coverage of any available conjugate vaccine and helps protect against the 20 Streptococcus pneumoniae serotypes in the vaccine.
On June 8, 2021, Pfizer announced the U.S. Food and Drug Administration (FDA) approved PREVNAR 20, which is the U.S trade name for PCV20, for the prevention of invasive disease and pneumonia in adults age 18 years or older. Pfizer has recently submitted to the FDA a supplemental Biologics License Application to include data in the PREVNAR 20 prescribing information for adults age 18 years or older regarding coadministration of PREVNAR 20 with a seasonal inactivated influenza vaccine.
Pivotal Phase 3 studies of the 20-valent pneumococcal conjugate vaccine candidate in infants are expected to read out in the second half of 2022 and, if positive, form the basis of potential regulatory submissions to the FDA and EMA later this year.
U.S. INDICATIONS FOR PREVNAR 20™
- PREVNAR 20™ is a vaccine indicated for active immunization for the prevention of pneumonia and invasive disease caused by Streptococcus pneumoniae serotypes 1, 3, 4, 5, 6A, 6B, 7F, 8, 9V, 10A, 11A, 12F, 14, 15B, 18C, 19A, 19F, 22F, 23F, and 33F in adults 18 years of age and older
- This indication for the prevention of pneumonia caused by S. pneumoniae serotypes 8, 10A, 11A, 12F, 15B, 22F, and 33F is approved under accelerated approval based on immune responses as measured by opsonophagocytic activity (OPA) assay. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
Please see full prescribing information for PREVNAR 20™.
In addition, to learn more, please visit us on www.Pfizer.com and follow us on Twitter at @Pfizer and @Pfizer News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.
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Source: Pfizer Inc.
Pfizer wins European approval for next-gen pneumococcal vaccine
Feb. 15, 2022 11:41 AM ET Pfizer Inc. (PFE)MRK
By: Dulan Lokuwithana, SA News Editor1 Comment
Pfizer (PFE -0.5%) announced that the European Medicines Agency (EMA) approved its 20-valent pneumococcal conjugate vaccine (PCV20) allowing its marketing across 27 EU countries and several other countries in the region.
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Mavacamten
Bristol Myers Squibb Announces Positive Topline Results from Phase 3 VALOR-HCM Trial, Evaluating Mavacamten in Patients with Obstructive Hypertrophic Cardiomyopathy Who are Eligible for Septal Reduction Therapy
02/16/2022CATEGORY:
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced that VALOR-HCM, the Phase 3 randomized, double-blind, placebo-controlled study evaluating mavacamten in adults with symptomatic obstructive hypertrophic cardiomyopathy (obstructive HCM) who are eligible for septal reduction therapy (SRT), met its primary endpoint at Week 16. The safety of mavacamten was consistent with previous studies.
“We are encouraged by the findings from this important study, which add to the growing body of clinical evidence that supports the promise of mavacamten for patients living with obstructive HCM,” said Roland Chen, M.D., Senior Vice President, Cardiovascular Development at Bristol Myers Squibb. “We look forward to sharing the results from VALOR-HCM at the American College of Cardiology 71st Annual Scientific Session & Expo taking place in April.”
The company plans to share these data with regulatory authorities. Bristol Myers Squibb thanks the patients and investigators participating in the VALOR-HCM clinical trial.
About the Phase 3 VALOR-HCM Trial
VALOR-HCM (NCT04349072) is a randomized, double-blind, placebo-controlled, multicenter Phase 3 study of patients with symptomatic, obstructive HCM (NYHA class III-IV) who meet guideline criteria for septal reduction therapy (SRT) and have been referred for an invasive procedure. The study enrolled over 100 patients randomized on a 1:1 basis to receive mavacamten or placebo. VALOR-HCM includes three treatment periods over 128 weeks: a 16-week placebo-controlled period, a 16-week active treatment period where all patients will receive mavacamten and a 96-week long-term extension period where all patients will continue to receive mavacamten.
The primary endpoint of VALOR-HCM is a composite of the number of patients who decide to proceed with SRT prior to or at Week 16 and the number of patients who remain SRT-guideline eligible (LVOT gradient of ≥50mmHg and NYHA Class III-IV) at Week 16 in the mavacamten group compared with the placebo group. Key secondary endpoints include impact on exercise gradient LVOT, NYHA Class and Kansas City Cardiomyopathy Questionnaire (KCCQ) and biomarkers at Week 16.
About Mavacamten
Mavacamten is a first-in-class, oral, allosteric modulator of cardiac myosin being investigated for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (obstructive HCM) which is a progressive disease that thickens the heart walls and makes it harder for the heart of expand normally and fill with blood. It is a selective cardiac myosin inhibitor that targets the underlying pathophysiology of obstructive HCM.
Mavacamten has been shown to reduce cardiac muscle contractility by inhibiting excessive myosin-actin cross-bridge formation that results in hypercontractility, left ventricular hypertrophy and reduced compliance. Based on data from the EXPLORER-HCM study, the company has a PDUFA date in the U.S. of April 28, 2022.
In clinical and preclinical studies, mavacamten has consistently reduced biomarkers of cardiac wall stress, lessened excessive cardiac contractility, increased diastolic compliance and lessened left ventricular outflow tract (LVOT) gradients. Mavacamten is an investigational therapy and is not approved for use in any country.
For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.
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Source: Bristol Myers Squibb
Bristol Myers mavacamten for heart disease meets main goal in late-stage study
Feb. 16, 2022 7:59 AM ET Bristol-Myers Squibb Company (BMY) By: Ravikash, SA News Editor
- Bristol Myers Squibb's (NYSE:BMY) phase 3 trial evaluating mavacamten to treat a type of heart condition met its main goal.
- https://seekingalpha.com/news/3800653-bristol-myers-mavacamten-for-heart-disease-meets-main-goal-in-late-stage-study
- https://seekingalpha.com/symbol/BMY
- https://www.bms.com/
NGENLA™ (somatrogon)
Pfizer and OPKO’s Once-Weekly NGENLA™ (somatrogon) Injection Receives Marketing Authorization in European Union for Treatment of Pediatric Growth Hormone Deficiency
Tuesday, February 15, 2022 - 11:08am
NEW YORK & MIAMI--(BUSINESS WIRE)-- Pfizer Inc. (NYSE: PFE) and OPKO Health, Inc. (NASDAQ: OPK) announced today that the European Commission has granted marketing authorization for the next-generation long-acting recombinant human growth hormone NGENLA™ (somatrogon), a once-weekly injection to treat children and adolescents from 3 years of age with growth disturbance due to insufficient secretion of growth hormone. NGENLA provides pediatric patients, their caregivers and healthcare providers with a new treatment option for growth hormone deficiency (GHD) that reduces the frequency of required injections from once daily to once weekly.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20220214005834/en/
“Growth hormone deficiency takes a substantial toll on children living with this rare disease and their families, and for many years, daily injections have been the standard of care, adding to the challenges they face,” said Reda Guiha, Regional President for International Developed Markets, Pfizer Rare Disease. “With NGENLA, we are proud to continue to advance the care of children in Europe who are impacted by growth hormone deficiency with a new, longer-acting option that significantly reduces treatment burden from once-daily to once-weekly injections.”
GHD is a rare disease characterized by the inadequate secretion of the growth hormone, somatropin, from the pituitary gland and affects one in approximately 4,000 to 10,000 children worldwide. The disease can be caused by genetic mutations or acquired after birth. Without treatment, affected children will have persistent growth attenuation and a very short height in adulthood, and puberty may be delayed.Children may also experience other problems with physical health and mental well-being.
“By helping to minimize disease management burden, we believe NGENLA has the potential to improve quality of life for children impacted by growth hormone deficiency and their families, as well as increase treatment adherence, which can improve outcomes,” said Phillip Frost, M.D., Chairman and Chief Executive Officer, OPKO Health. “We are pleased that the marketing authorization in the European Union will enable more children with growth hormone deficiency to benefit from once-weekly treatment.”
The European Union (EU) marketing authorization of NGENLA was supported by results from a global, randomized, open-label, active-controlled Phase 3 study which evaluated the safety and efficacy of once-weekly NGENLA compared to once-daily GENOTROPIN® (somatropin). The study met its primary endpoint of NGENLA non-inferiority compared to GENOTROPIN, as measured by annual height velocity at 12 months. NGENLA was generally well tolerated in the study and had a safety profile comparable to GENOTROPIN.
The marketing authorization of NGENLA is valid in all EU Member States as well as Iceland, Norway and Liechtenstein.
About the NGENLATM (somatrogon) Injection Studies
The safety and efficacy of NGENLA™ (somatrogon) was demonstrated in a randomized, open-label, active-controlled Phase 3 study conducted in over 20 countries. In this study, 224 prepubertal, treatment-naïve children with growth hormone deficiency (GHD) were randomized 1:1 to receive NGENLA once-weekly or GENOTROPIN® (somatropin) once-daily. The study met its primary endpoint of NGENLA non-inferiority compared to GENOTROPIN, as measured by annual height velocity at 12 months. NGENLA was generally well tolerated in the study and had a safety profile comparable to GENOTROPIN. Participants had the opportunity to enroll in a global, open-label, multicenter, long-term extension study in which they were able to either continue receiving or switch to NGENLA. Approximately 95% of the patients switched into the open-label extension study and received NGENLA treatment.
An additional Phase 3 study (C0311002) assessed the perception of the treatment burden of once-weekly NGENLA versus once-daily GENOTROPIN among children three to <18 years of age with GHD and their caregivers. This randomized, multicenter, open-label, crossover study included 87 participants who were on stable growth hormone therapy: 43 were randomized to Sequence 1 (12 weeks of treatment with GENOTROPIN followed by 12 weeks of treatment with NGENLA) and 44 were randomized to Sequence 2 (12 weeks of treatment with NGENLA followed by 12 weeks of treatment with GENOTROPIN). The study demonstrated that treatment with NGENLA improved the mean overall Life Interference total score after 12 weeks of treatment compared to GENOTROPIN.
About NGENLATM (somatrogon) Injection
NGENLA™ (somatrogon) is a synthetic growth hormone that works by replacing the lack of growth hormone in the body. NGENLA is taken by injection just below the skin. Compared to the growth hormone GENOTROPIN® (somatropin), its action in the body lasts longer, enabling weekly injections instead of daily. NGENLA is approved for the treatment of pediatric growth hormone deficiency (GHD) in Canada, Australia and Japan, and the EU marketing authorization is valid in all EU Member States as well as Iceland, Norway and Liechtenstein.
In 2014, Pfizer and OPKO entered into a worldwide agreement for the development and commercialization of NGENLA for the treatment of GHD. Under the agreement, OPKO is responsible for conducting the clinical program and Pfizer is responsible for registering and commercializing NGENLA for GHD.
About GENOTROPIN® (somatropin)
GENOTROPIN® (somatropin) is a man-made, prescription treatment option. The indications GENOTROPIN is approved for vary by market. GENOTROPIN is approved for growth failure due to growth hormone deficiency (GHD) and adult GHD, Prader-Willi Syndrome, Idiopathic Short Stature, Turner Syndrome, Small for Gestational Age (with no catch-up growth) and Chronic Renal Insufficiency. GENOTROPIN is taken by injection just below the skin and is available in a wide range of devices to fit a range of individual dosing needs. GENOTROPIN is just like the natural growth hormone that our bodies make and has an established safety profile.
In addition, to learn more, please visit us on www.Pfizer.com and follow us on Twitter at @Pfizer and @Pfizer News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.
For more information, visit http://www.OPKO.com.
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Pfizer
Source: Pfizer Inc.
Pfizer, OPKO pediatric growth hormone deficiency injection somatrogon gets EU nod
Feb. 15, 2022 1:19 PM ET Pfizer Inc. (PFE), OPK
By: Jonathan Block, SA News Editor
- The European Commission has approved Ngenla (somatrogon), a long-acting recombinant human growth hormone to treat pediatric growth hormone deficiency, from Pfizer (PFE -0.8%) and OPKO Health (OPK +5.6%).
- https://seekingalpha.com/news/3800269-pfizer-opko-pediatric-growth-hormone-deficiency-somatrogon-injection-gets-eu-nod
- https://seekingalpha.com/symbol/OPK
- https://seekingalpha.com/symbol/PFE
- https://www.opko.com/what-we-do/our-research/somatrogon
NURTEC ODT (rimegepant)
BIOHAVEN AND PFIZER ANNOUNCE POSITIVE TOPLINE RESULTS OF PIVOTAL TRIAL OF RIMEGEPANT FOR THE ACUTE TREATMENT OF MIGRAINE IN CHINA AND SOUTH KOREA
02/14/2022
NEW HAVEN, Conn. and NEW YORK, Feb. 14, 2022 /PRNewswire/ -- Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) and Pfizer Inc. (NYSE: PFE), today announced positive top-line results from an Asia-Pacific, Phase 3 clinical trial of rimegepant in 1,431 adults for the acute treatment of migraine. Led by BioShin Limited, a subsidiary of Biohaven in China and South Korea, the randomized, regional, multi-center study met the co-primary endpoints evaluating the efficacy and safety of the orally dissolving tablet (ODT) formulation of rimegepant, an oral calcitonin gene-related peptide (CGRP) receptor antagonist.
This is the fourth positive Phase 3 study of rimegepant for the acute treatment of migraine and the first to be conducted in Asia Pacific. The study met its co-primary endpoints of freedom from pain (p<0.0001) and freedom from most bothersome migraine–associated symptom (MBS) including either nausea, phonophobia or photophobia (p<0.0001) at 2-hours following a single oral dose of rimegepant. In the study, a single oral dose of rimegepant 75 mg provided significant relief of migraine symptoms and return to normal function at 2 hours and delivered sustained efficacy that lasted up to 48 hours for many patients. Rimegepant showed a favorable safety and tolerability profile among study participants that was consistent with prior clinical trial results in the United States. Detailed data from the study will be presented at future medical meetings to help inform ongoing and future research.
Under the terms of the collaboration agreement between Biohaven and Pfizer, Pfizer has commercialization rights to rimegepant in markets outside of the U.S. Biohaven continues to lead research and development globally and retains the U.S. market. Rimegepant is commercialized as Nurtec® ODT in the U.S. and is the only oral CGRP receptor antagonist approved for both the acute and preventive treatment of migraine in adults. An application for the approval of rimegepant is currently under review by the European Medicines Agency with a decision expected in the first half of 2022. Rimegepant is approved for the acute treatment of migraine in Kuwait and the United Arab Emirates, and for the acute and preventive treatment of migraine in Israel.
Vlad Coric, M.D., Chief Executive Officer and Chairman of the Board of Biohaven commented, "These top-line trial results clearly show the consistent clinical profile of rimegepant to relieve migraine symptoms and return patients to normal function. Through our partnership with Pfizer, we are committed to rapidly expanding the availability of rimegepant to patients around the world, particularly in Asia Pacific where migraine is a common disease and a leading cause of disability."
"It is very exciting to see the completion and positive results of the first Phase 3 study of rimegepant in Asia Pacific," said Nick Lagunowich, Global President, Pfizer Internal Medicine. "With millions of patients in the region impacted by this debilitating neurological disease, these results provide hope for a potentially new effective acute treatment for patients in need. We are moving as quickly as possible in our effort to get this potential treatment into the hands of patients, and we look forward to working with regulatory agencies around the world to do so."
Professor Shengyuan Yu, Principal Investigator of the study and Director of the Department of Neurology, Chinese PLA General Hospital, said, "We need new, effective and safe treatment options to help improve the lives of our migraine patients in Asia and are encouraged by the positive results of this study."
Donnie McGrath, M.D., Executive Chairman of Biohaven's wholly-owned subsidiary in China, BioShin, added, "The results from this study demonstrate the effectiveness of rimegepant and highlight the potential impact for patients in Asia Pacific, if approved. I'm so proud of the BioShin R&D team who executed this study."
About Rimegepant
Rimegepant targets a key component of migraine by reversibly blocking CGRP receptors, thereby inhibiting the biologic cascade that results in a migraine attack. Rimegepant was approved by the U.S. Food and Drug Administration (FDA) under the trade name Nurtec ODT for the acute treatment of migraine in February 2020 and for the preventive treatment of episodic migraine in May 2021. A single dose of 75 mg Nurtec ODT provides fast pain relief, significant pain reduction and return to normal function, and has a lasting effect of up to 48 hours in many patients. Nurtec ODT is taken orally as needed, up to 18 doses/month to stop migraine attacks or taken every other day to help prevent migraine attacks and reduce the number of monthly migraine days. Nurtec ODT does not have addiction potential and is not associated with medication overuse headache or rebound headache.
About NURTEC ODT
NURTEC ODT (rimegepant) is the first and only calcitonin gene-related peptide (CGRP) receptor antagonist available in a quick-dissolve ODT formulation that is approved by the U.S. Food and Drug Administration (FDA) for the acute treatment of migraine with or without aura and the preventive treatment of episodic migraine in adults. The activity of the neuropeptide CGRP is thought to play a causal role in migraine pathophysiology. NURTEC ODT is a CGRP receptor antagonist that works by reversibly blocking CGRP receptors, thereby inhibiting the biologic activity of the CGRP neuropeptide. For more information about NURTEC ODT, visit www.nurtec.com.
Indication
NURTEC ODT orally disintegrating tablets is a prescription medicine that is used to treat migraine in adults. It is for the acute treatment of migraine attacks with or without aura and the preventive treatment of episodic migraine. It is not known if NURTEC ODT is safe and effective in children.
See full Prescribing Information and Patient Information.
More information about Biohaven is available at www.biohavenpharma.com.
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SOURCE Biohaven Pharmaceutical Holding Company Ltd.
Pfizer/ Biohaven announce late-stage migraine trial met main goals in Asia Pacific
Feb. 14, 2022 9:20 AM ET Pfizer Inc. (PFE), BHVN By: Dulan Lokuwithana, SA News Editor2 Comments
Pfizer (NYSE:PFE) and Biohaven Pharmaceutical (NYSE:BHVN) announced on Monday that rimegepant known as NURTEC ODT in the U.S. met co-primary endpoints in a late-stage trial for the first time in Asia Pacific.
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EYLEA® (aflibercept) Injection
February 11, 2022 at 7:50 AM EST Back
REGENERON PRESENTS ENCOURAGING PHASE 2 RESULTS FOR HIGH-DOSE AFLIBERCEPT 8 MG IN WET AGE-RELATED MACULAR DEGENERATION AT ANGIOGENESIS MEETING
TARRYTOWN, N.Y., Feb. 11, 2022 /PRNewswire/ --
Trial met primary safety endpoint and no new safety signals seen through week 44
Results favored aflibercept 8 mg in visual acuity, drying and other anatomical measures through week 44
Phase 3 results in wet age-related macular degeneration and diabetic macular edema expected in the second half of 2022
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced results from its Phase 2 proof-of-concept trial evaluating an investigational 8 mg high dose of aflibercept compared to the currently-approved 2 mg dose of EYLEA® (aflibercept) Injection in patients with wet age-related macular degeneration (wet AMD). The results will be presented at the Angiogenesis (Angiogenesis, Exudation, and Degeneration) 2022 annual meeting on Saturday, February 12.
As previously announced, more patients treated with aflibercept 8 mg had no retinal fluid at week 16, when the primary efficacy endpoint was assessed. At this timepoint, 43% (n=23/53) had no fluid in the macula compared to 26% for EYLEA (n=14/53) (p=0.0667); and 51% (n=27) had no fluid in the center subfield compared to 34% for EYLEA (n=18) (p=0.0770). At week 16, patients in both treatment groups had received three initial doses (administered at weeks 0, 4 and 8), after which dosing was extended to every 12 weeks. In new results presented for the first time, aflibercept 8 mg continued to show numeric improvements in anatomical and vision outcomes compared to EYLEA through 44 weeks.
"These Phase 2 data in wet AMD demonstrate the exciting potential for aflibercept 8 mg to maintain dryness and improve vision compared to the standard-of-care EYLEA," said Dr. David Brown, Director of Research at Retina Consultants of Texas. "This is the first time we have seen a promising trend towards sustained improved vision over EYLEA in wet AMD. I look forward to seeing the results of the Phase 3 program investigating extended dosing of aflibercept 8 mg."
Eyes treated with aflibercept 8 mg were more likely to be dry in the center subfield on optical coherence tomography (OCT) compared to EYLEA at every timepoint measured throughout the trial after the initial monthly dosing period. At week 44 when the trial ended, key anatomical and vision changes included:
- 40% (n=21/53) of patients treated with aflibercept 8 mg did not have fluid in the center subfield compared to 28% (n=15/53) of patients treated with EYLEA (nominal p=0.2185).
- Twice as many patients treated with aflibercept 8 mg (32%, n=17/53) had no macular fluid compared to patients treated with EYLEA (15%, n=8/53) (nominal p=0.0395), as measured by spectral domain OCT. Measuring macular fluid provides an evaluation of a larger area of the retina compared to the center subfield and may provide a better understanding of the anatomical effects of treatment in wet AMD.
- 7.9 average letter improvement from baseline in the aflibercept 8 mg group, compared to 5.1 letters in the EYLEA group, as measured by Early Treatment Diabetic Retinopathy Study (ETDRS) letters (nominal p=0.1957).
- Nearly half (47%) of aflibercept 8 mg patients achieved at least a 10-letter gain (2 lines on a vision test) and more than a quarter (28%) achieved more than 15 letters (3 lines on a vision test), compared to 35% and 18% for patients treated with EYLEA, respectively.
Through 44 weeks, adverse events (AEs) in the study eye occurred in 38% (n=20/53) of both aflibercept 8 mg and EYLEA patients. There were no serious AEs of intraocular inflammation (including occlusive retinal vasculitis), and no anti-platelet trialists' collaboration (APTC)-defined arterial thromboembolic events. The most common ocular AEs that occurred more frequently in the aflibercept 8 mg group were vitreous detachment (4 aflibercept 8 mg, 2 EYLEA), conjunctival hemorrhage (3 aflibercept 8 mg, 2 EYLEA) and retinal tear (2 aflibercept 8 mg, 0 EYLEA). There was one patient death in the aflibercept 8 mg unrelated to treatment.
"After more than two decades of following the science in retinal diseases, we are very proud of our legacy helping millions of patients to retain or improve their vision with EYLEA, which has set a very high bar for any new treatment," said George D. Yancopoulos, M.D., Ph.D., President and Chief Scientific Officer at Regeneron. "The results of this Phase 2 trial for aflibercept 8 mg are promising and we look forward to seeing the results from the Phase 3 program, which we hope will show that aflibercept 8 mg can deliver clinical outcomes that, at a minimum, will be comparable to standard-of-care EYLEA, but allow for extended dosing regimens."
Wet AMD is the leading cause of vision loss among people 50 years and older in the U.S. Existing anti-VEGF treatments including EYLEA have helped change the course of disease for millions of patients worldwide, and efforts to develop new medicines are focused on further enhancing clinical effectiveness while extending the time between treatment doses.
Aflibercept 8 mg is being jointly developed by Regeneron and Bayer. This new, concentrated high-dose aflibercept formulation enables a greater amount of medicine to be administered with each treatment, and could potentially extend the time between doses while retaining the efficacy and safety profile seen with EYLEA. Aflibercept 8 mg is currently under clinical development and its safety and efficacy have not been fully evaluated by any regulatory authority.
About the Phase 2 Trial
The Phase 2 randomized, single-masked CANDELA trial (NCT04126317) enrolled 106 treatment-naïve patients with wet AMD. The trial was designed to investigate the safety, efficacy and tolerability of aflibercept 8 mg compared to the currently-approved 2 mg dose of EYLEA. Patients were randomized into two groups, with one group receiving aflibercept 8 mg (n=53) and the other group receiving EYLEA (n=53). Patients in both groups received three initial intravitreal injections (weeks 0, 4 and 8), before the primary endpoint was assessed at week 16, after which dosing was extended to every 12 weeks, or more frequently if required due to persistent or worsening disease. Efficacy was assessed via the presence of retinal fluid in the center subfield on optical coherence tomography at this timepoint.
Trial participants were at least 50 years of age (mean: 77 years), mean baseline retinal thickness was 502.1 microns, and the best corrected visual acuity (BCVA) ETDRS letter score was between 24 to 78 in the study eye (mean: 59 letters).
About the Phase 3 Clinical Program
There are two ongoing pivotal trials to investigate the efficacy and safety of aflibercept 8 mg versus EYLEA. In diabetic macular edema (DME), Regeneron is sponsoring the Phase 2/3 multi-center, randomized, double-masked PHOTON trial (NCT04429503). In wet AMD, Bayer is sponsoring the Phase 3 multi-center, randomized, double-masked PULSAR trial (NCT04423718) in treatment-naïve patients. Across both trials, patients are randomized into one of three treatment groups, testing aflibercept 8 mg with dosing regimens at either 12- or 16-week intervals or EYLEA with an 8-week dosing regimen.
IMPORTANT EYLEA SAFETY INFORMATION AND INDICATIONS
INDICATIONS
EYLEA® (aflibercept) Injection 2 mg (0.05 mL) is indicated for the treatment of patients with Neovascular (Wet) Age-related Macular Degeneration (AMD), Macular Edema following Retinal Vein Occlusion (RVO), Diabetic Macular Edema (DME), and Diabetic Retinopathy (DR).
CONTRAINDICATIONS
- EYLEA is contraindicated in patients with ocular or periocular infections, active intraocular inflammation, or known hypersensitivity to aflibercept or to any of the excipients in EYLEA.
- For more information, please see full Prescribing Information.
- https://eylea.us/
For additional information about the company, please visit www.regeneron.com or follow @Regeneron on Twitter.
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SOURCE Regeneron Pharmaceuticals, Inc.
Regeneron gains on new long-term Phase 2 data for Eylea in wet AMD
Feb. 11, 2022 3:38 PM ET Regeneron Pharmaceuticals, Inc. (REGN)RHHBY
By: Dulan Lokuwithana, SA News Editor5 Comments
Regeneron (REGN +3.6%) has recorded the biggest intraday gain in more than three months after reporting 44-week data from a Phase 2 trial for the company’s blockbuster therapy Eylea (aflibercept) at 8 mg dose in wet age-related macular degeneration (wet AMD).
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