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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.
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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/
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