Moorestown, New Jersey, United States
biotecMAX™ April/May/June/July/Aug/Sept 2021 Insight
KEYTRUDA® (pembrolizumab) in Combination With Chemotherapy
Merck Receives Positive EU CHMP Opinion for KEYTRUDA® (pembrolizumab) in Combination With Chemotherapy for Certain Patients With Locally Recurrent Unresectable or Metastatic Triple-Negative Breast Cancer Whose Tumors Express PD-L1 (CPS ≥10)
September 17, 2021 7:15 am ET
Recommendation Based on Data From Phase 3 KEYNOTE-355 Trial
KENILWORTH, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended approval of KEYTRUDA, Merck’s anti-PD-1 therapy, in combination with chemotherapy for the treatment of locally recurrent unresectable or metastatic triple-negative breast cancer (TNBC) in adults whose tumors express PD-L1 (Combined Positive Score [CPS] ≥10) and who have not received prior chemotherapy for metastatic disease.
The positive opinion is based on progression-free survival (PFS) and overall survival (OS) results from the Phase 3 KEYNOTE-355 trial, which showed that treatment with KEYTRUDA in combination with chemotherapy (nab-paclitaxel, paclitaxel or gemcitabine/carboplatin), as compared to chemotherapy alone, significantly improved PFS and OS in these patients. Overall survival data from KEYNOTE-355 will be presented at the European Society for Medical Oncology (ESMO) Congress 2021 on Sept. 19. The CHMP’s recommendation will now be reviewed by the European Commission for marketing authorization in the European Union.
“Triple-negative breast cancer grows and spreads faster than other types of breast cancer and consequently has a worse prognosis,” said Dr. Vicki Goodman, vice president, clinical research, Merck Research Laboratories. “This positive CHMP opinion is an important step forward in bringing a new immunotherapy treatment option with KEYTRUDA to appropriate patients in Europe with metastatic triple-negative breast cancer. Importantly, this treatment regimen can be used in combination with different chemotherapy agents. We look forward to the European Commission’s decision in the coming months.”
Merck is rapidly advancing a broad portfolio in gynecologic and breast cancers through an extensive clinical development program for KEYTRUDA and several other investigational and approved medicines across these areas.
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 1,600 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.
Selected KEYTRUDA® (pembrolizumab) Indications in the U.S.
Melanoma
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.
KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.
Non-Small Cell Lung Cancer
KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) ≥1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is:
- stage III where patients are not candidates for surgical resection or definitive chemoradiation, or
- metastatic.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS ≥1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.
Head and Neck Squamous Cell Cancer
KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS ≥1)] as determined by an FDA-approved test.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.
Classical Hodgkin Lymphoma
KEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).
KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.
Primary Mediastinal Large B-Cell Lymphoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.
Urothelial Carcinoma
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC):
- who are not eligible for any platinum-containing chemotherapy, or
- who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.
Microsatellite Instability-High or Mismatch Repair Deficient Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options.
This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.
Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).
Gastric Cancer
KEYTRUDA, in combination with trastuzumab, fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of patients with locally advanced unresectable or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma.
This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Esophageal Cancer
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic esophageal or GEJ (tumors with epicenter 1 to 5 centimeters above the GEJ) carcinoma that is not amenable to surgical resection or definitive chemoradiation either:
- in combination with platinum- and fluoropyrimidine-based chemotherapy, or
- as a single agent after one or more prior lines of systemic therapy for patients with tumors of squamous cell histology that express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
Cervical Cancer
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Hepatocellular Carcinoma
KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Merkel Cell Carcinoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Renal Cell Carcinoma
KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of adult patients with advanced renal cell carcinoma.
Tumor Mutational Burden-High Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [≥10 mutations/megabase] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.
Cutaneous Squamous Cell Carcinoma
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) or locally advanced cSCC that is not curable by surgery or radiation.
Triple-Negative Breast Cancer
KEYTRUDA is indicated for the treatment of patients with high-risk early-stage triple-negative breast cancer (TNBC) in combination with chemotherapy as neoadjuvant treatment, and then continued as a single agent as adjuvant treatment after surgery.
KEYTRUDA, in combination with chemotherapy, is indicated for the treatment of patients with locally recurrent unresectable or metastatic TNBC whose tumors express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
For more information, visit www.merck.com
Please see Prescribing Information for KEYTRUDA (pembrolizumab) at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_pi.pdf and Medication Guide for KEYTRUDA at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_mg.pdf.
Merck wins positive opinion in EU for KEYTRUDA plus chemotherapy for breast cancer
Sep. 17, 2021 7:35 AM ETMerck & Co., Inc. (MRK)By: Dulan Lokuwithana, SA News Editor3 Comments
- Merck (NYSE:MRK) announced that European Medicines Agency (EMA) recommended its anti-PD-1 therapy KEYTRUDA in combination with chemotherapy for a certain group of patients with locally recurrent unresectable or metastatic triple-negative breast cancer (TNBC).
- https://seekingalpha.com/news/3740757-merck-wins-positive-opinion-in-eu-for-keytruda-plus-chemotherapy-for-breast-cancer
- https://seekingalpha.com/symbol/MRK
- https://www.keytruda.com/
GAVRETO® (pralsetinib)
Roche receives positive CHMP opinion for Gavreto® (pralsetinib) for the treatment of adults with RET fusion-positive advanced non-small cell lung cancer
- Gavreto showed robust and durable clinical responses in people with NSCLC with RET fusions
- If approved, Gavreto will be the first and only targeted treatment approved by the EMA that includes first-line treatment of people with RET fusion-positive advanced NSCLC
Basel, 17 September 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of Gavreto® (pralsetinib) as a monotherapy for the treatment of adult patients with rearranged during transfection (RET) fusion-positive advanced non-small cell lung cancer (NSCLC) not previously treated with a RET inhibitor.
“This positive CHMP opinion for Gavreto represents another important step towards our goal of providing effective therapeutics that target genomic drivers of disease for as many cancer patients as possible," said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. “Advances in personalised medicine also underscore the importance of tumour genomic profiling to identify patients who may benefit from targeted therapies.”
RET alterations are key disease drivers in many cancer types, including NSCLC and multiple types of thyroid cancer. RET fusion-positive NSCLC affects approximately 37,500 people worldwide each year and the disease often affects those who least expect it;[1,2,3] RET fusion-positive NSCLC is often identified in younger people with a minimal to no history of smoking.[3] These cancers also typically represent a high unmet need, due to limitations associated with standard therapies.[4,5,6] Biomarker testing for these fusions is the most effective way to identify people with advanced NSCLC who are eligible for treatment with Gavreto. Gavreto is a highly selective, potent, and CNS-penetrant RET inhibitor and, together with Alecensa® (alectinib) and Rozlytrek® (entrectinib), is part of Roche’s portfolio of targeted treatments for NSCLC. Together, they offer personalised treatment options for almost one in ten people with advanced NSCLC.[7]
The CHMP recommendation is based on the results of the phase I/II ARROW study, in which Gavreto demonstrated rapid, potent, and durable clinical activity in patients with advanced RET fusion-positive NSCLC.[8] A final decision regarding the approval of Gavreto is expected from the European Commission in the coming months.
Gavreto has also shown activity across multiple solid tumour types, reflecting tumour-agnostic potential.[9] In September 2020, the U.S. Food and Drug Administration (FDA) approved Gavreto for the treatment of adults with metastatic RET fusion-positive NSCLC, and in December 2020 it was approved for the treatment of adult and paediatric patients 12 years of age and older with advanced RET-altered thyroid cancers. Gavreto has since been approved in Canada, mainland China and Switzerland. In the European Union, the MAA for Gavreto for the treatment of adults with RET fusion-positive NSCLC is ongoing, and a submission for RET-altered thyroid cancers is planned. Regulatory submissions for these indications are underway in multiple countries worldwide.
Blueprint Medicines and Roche are co-developing Gavreto globally, with the exception of certain territories in Asia, including China.* Blueprint Medicines and Genentech, a wholly owned member of the Roche Group, are commercialising Gavreto in the US and Roche has exclusive commercialisation rights for Gavreto outside of the US, with the exception of certain territories in Asia, including China.*
*CStone Pharmaceuticals retains all rights to the development and commercialisation of Gavreto in these territories under its existing collaboration with Blueprint Medicines.
About the ARROW study[10]
ARROW is an ongoing phase I/II, open-label, first-in-human study designed to evaluate the safety, tolerability and efficacy of Gavreto, administered orally in people with rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC), RET-mutant medullary thyroid cancer, RET fusion-positive thyroid cancer and other RET-altered solid tumours. ARROW is being conducted at multiple sites across the United States, Europe and Asia.
An update from the ARROW study was presented at the American Society of Clinical Oncology (ASCO) 2021 Virtual Scientific Programme, 4-8 June.[11] In 126 patients with RET fusion-positive NSCLC who previously received platinum-based chemotherapy, Gavreto demonstrated an overall response rate (ORR) of 62% (95% CI: 53%, 70%), a clinical benefit rate (CBR) of 74% (95% CI: 65%, 81%), and a disease control rate (DCR) of 91% (95% CI: 85%, 96%). Median progression-free survival (PFS) was 16.5 months (95% CI: 10.5 months, 24.1 months). In 68 treatment-naïve patients, the confirmed ORR was 79% (95% CI: 68%, 88%), the CBR was 82% (95% CI: 71%, 91%), and the DCR was 93% (95% CI: 84%, 98%). Median PFS was 13.0 months (95% CI: 9.1 months, not reached (NR)). In 25 treatment-naïve patients who were enrolled after eligibility criteria were revised, to allow candidates for platinum-based therapy, the confirmed ORR was 88% (95% CI: 69%, 98%), the CBR was 88% (95% CI: 69%, 98%), and the DCR was 96% (95% CI: 80%, 100%). Median PFS was not reached. In addition, Gavreto was well-tolerated; of the 471 ARROW trial patients across RET-altered tumour types, the most common (≥ 25%) treatment-related adverse events were neutropenia, increased liver enzymes (aspartate aminotransferase [AST] and alanine aminotransferase [ALT]), anaemia, white blood cell count decrease, high blood pressure (hypertension) and lack of energy (asthenia).
About Gavreto® (pralsetinib)
Gavreto is a once-daily, oral targeted treatment designed to selectively target rearranged during transfection (RET) alterations, including fusions and mutations, regardless of the tissue of origin. Preclinical data have shown that Gavreto inhibits primary RET fusions and mutations that cause cancer in subsets of patients, as well as secondary RET mutations predicted to drive resistance to treatment. Blueprint Medicines and Roche are co-developing Gavreto for the treatment of people with various types of RET-altered cancers.
The Roche Group, headquartered in Basel, Switzerland, is active in over 100 countries and in 2020 employed more than 100,000 people worldwide. In 2020, Roche invested CHF 12.2 billion in R&D and posted sales of CHF 58.3 billion. 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.
Blueprint Medicines, Gavreto and associated logos are trademarks of Blueprint Medicines Corporation.
Roche receives positive CHMP opinion for Gavreto for non-small cell lung cancer
Sep. 17, 2021 8:00 AM ET Roche Holding AG (RHHBY)
By: Jonathan M Block, SA News Editor
- The European Medicine Agency's Committee for Medicinal Products for Human Use ("CHMP") has issued a positive opinion on Roche's (OTCQX:RHHBY) Gavreto (pralsetinib) as a monotherapy for non-small cell lung cancer ("NSCLC").
- https://seekingalpha.com/news/3740769-roche-receives-positive-chmp-opinion-for-gavreto-for-non-small-cell-lung-cancer
- https://seekingalpha.com/symbol/RHHBY
- https://www.roche.com/
- https://www.gavreto.com/index.html
- https://seekingalpha.com/symbol/BPMC
- https://www.blueprintmedicines.com/
- https://www.blueprintmedicines.com/medicines/
PIQRAY® (alpelisib)
Novartis announces findings from a real-world study of alpelisib demonstrating clinical benefit in people with PIK3CA-Related Overgrowth Spectrum (PROS)
Sep 17, 2021
- PROS is a spectrum of rare disorders caused by PIK3CA mutations and is characterized by atypical, visible overgrowths and anomalies in blood vessels, the lymphatic system and other tissues
- At 24 weeks, 38% of patients achieved ≥20% reduction in the volume of the PROS lesions assessed in the primary endpoint analysis; no patients experienced disease progression or death
- Alpelisib is the first potential treatment to specifically address the root cause of PROS conditions
- EPIK-P1 study findings presented at ESMO Virtual Congress 2021 support FDA submission
Basel, September 17, 2021— Novartis today announced important findings from a real-world study evaluating the safety and efficacy of alpelisib for people living with PIK3CA-Related Overgrowth Spectrum (PROS) who received treatment daily for at least 24 weeks. Results from EPIK-P1 showed alpelisib effectively reduced volume of clinically significant PROS-related lesions and improved signs and symptoms in pediatric and adult patients. Results were presented at the European Society of Medical Oncology (ESMO) Virtual Congress 2021 [LBA23].
“There are few options available to manage PROS conditions, and they are mainly focused on addressing worsening symptoms. It is devastating for patients to be without treatments that address the underlying cause of PROS,” said Guillaume Canaud, MD, PhD, Necker-Enfants Malades Hospital – AP-HP, the Paris Descartes University, Inserm (INEM Institute Necker Enfants Malades – Centre for Molecular Medicine). “The EPIK-P1 findings show robust clinical benefit for adult and pediatric patients and a potential new path forward for those impacted by PROS conditions.”
In EPIK-P1, alpelisib reduced target lesion volume and improved PROS-related symptoms and manifestations. The primary endpoint analysis conducted at week 24 in patients with complete cases (n=32) showed 38% of patients achieving a response to treatment which was defined as 20% or greater reduction in the sum of PROS target lesion volume. Nearly three in four patients (74%) showed some reduction in target lesion volume, with a mean reduction of 13.7%, and no patients experienced disease progression at time of primary analysis.
About EPIK-P1
EPIK-P1 is a global, site-based retrospective non-interventional medical chart review of pediatric and adult male and female patients aged 2 years or older with PIK3CA-Related Overgrowth Spectrum (PROS) who received alpelisib via a compassionate use program. Primary endpoint is proportion of patients with response at 24 weeks, defined as achieving at least 20% reduction from index date in the sum of measurable target lesion volume via central imaging. Data were obtained from medical charts of 57 patients (39 pediatric, 18 adult) at seven sites in five countries. There were 32 complete cases, meaning there were complete scans for comparison at baseline and date of analysis, and one patient discontinued prior to week 24 due to lack of clinical efficacy.
Alpelisib is not approved by any regulatory authority for the treatment of PROS conditions.
Find out more at https://www.novartis.com.
Novartis' alpelisib shows robust clinical benefit in people with PROS conditions
Sep. 17, 2021 8:31 AM ETNovartis AG (NVS)By: Mamta Mayani, SA News Editor
- Novartis (NYSE:NVS) announces findings from a real-world study evaluating the safety and efficacy of alpelisib in people with PIK3CA-Related Overgrowth Spectrum (PROS) who received treatment daily for at least 24 weeks.
- https://seekingalpha.com/news/3740790-novartis-alpelisib-shows-robust-clinical-benefit-in-people-with-pros-conditions
- https://seekingalpha.com/symbol/NVS
- https://www.hcp.novartis.com/products/piqray/metastatic-breast-cancer/
- https://www.novartis.com/
VUMERITY® (diroximel fumarate)
CHMP Recommends VUMERITY® (diroximel fumarate) for Approval in the European Union as a Treatment for Relapsing-Remitting Multiple Sclerosis
SEPTEMBER 17, 2021 • INVESTOR RELATIONS
- VUMERITY is a next-generation oral fumarate with a well-characterized efficacy and safety profile
- Data from the Phase 3 EVOLVE-MS-2 study have demonstrated that treatment with VUMERITY results in low discontinuation rates due to its gastrointestinal (GI) tolerability profile
- Upon approval, VUMERITY will offer a new oral option for MS patients as they consider treatment initiation in the context of the COVID-19 environment
CAMBRIDGE, Mass., Sept. 17, 2021 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) today announced that the Committee for Medicinal Products for Human Use (CHMP), part of the European Medicines Agency (EMA), issued a positive opinion and has recommended granting marketing authorization for VUMERITY® (diroximel fumarate) in the European Union (EU). VUMERITY is a next-generation oral fumarate for the treatment of adults with relapsing-remitting multiple sclerosis (RRMS). An estimated 2.8 million people live with MS across the globe, with some European countries demonstrating the highest prevalence of MS in the world.1
“With MS, finding the right treatment option is as much about managing the clinical aspects of the disease as it is about how treatment fits into a person’s life,” said Simon Faissner, M.D., PhD, Assistant Professor at the Department of Neurology, Ruhr-University Bochum. “Today’s CHMP opinion is a crucial step forward in providing an oral therapeutic option that is easy to integrate into a patient’s daily life, which helps with ongoing care management.”
The CHMP’s positive opinion will now be referred to the European Commission (EC), which grants marketing authorizations for medicines in Europe.
The positive CHMP opinion was based on data from pharmacokinetic bridging studies comparing VUMERITY and TECFIDERA® (dimethyl fumarate) to establish bioequivalent exposure of monomethyl fumarate, the active metabolite, and relied in part on the well-established long-term safety and efficacy profile of TECFIDERA. The CHMP also assessed findings from EVOLVE-MS-2, a large, randomized, double-blind, five-week, multi-center Phase 3 study to evaluate the gastrointestinal (GI) tolerability of VUMERITY compared to TECFIDERA in patients with RRMS. In EVOLVE-MS-2, the rate of overall treatment discontinuation was lower in participants treated with VUMERITY compared to those treated with TECFIDERA (1.6% compared to 6%, respectively). The difference in the discontinuation rates due to GI tolerability was 0.8% for VUMERITY compared to 4.8% for TECFIDERA.
VUMERITY was first approved by the U.S. Food and Drug Administration in October 2019 and is currently the number one prescribed oral MS therapy in the country. Since its launch in the U.S., real-world data have reinforced the positive GI tolerability profile of VUMERITY and confirmed that the experience demonstrated in clinical trials is consistent with clinical practice.2 Biogen continues to file regulatory submissions in other countries.
About VUMERITY® (diroximel fumarate)
VUMERITY is an oral fumarate with a distinct chemical structure from TECFIDERA® (dimethyl fumarate), approved in the U.S. for the treatment of relapsing forms of multiple sclerosis in adults, to include clinically isolated syndrome, relapsing-remitting disease and active secondary progressive disease. Once in the body, VUMERITY rapidly converts to monomethyl fumarate, the same active metabolite of dimethyl fumarate providing similar efficacy and safety profiles.
VUMERITY is contraindicated in patients with known hypersensitivity to diroximel fumarate, dimethyl fumarate or to any of the excipients of VUMERITY; and in patients taking dimethyl fumarate. Serious side effects for VUMERITY are based on data from dimethyl fumarate (which has the same active metabolite as VUMERITY) and include anaphylaxis and angioedema, progressive multifocal leukoencephalopathy, which is a rare opportunistic viral infection of the brain that has been associated with death or severe disability, a decrease in mean lymphocyte counts during the first year of treatment, herpes zoster and other serious infections, liver injury and flushing. The most common adverse events, obtained using data from dimethyl fumarate (which has the same active metabolite as VUMERITY), were flushing, abdominal pain, diarrhea and nausea.
Please click here for Important Safety Information and full Prescribing Information, including Patient Information for VUMERITY in the U.S.
About TECFIDERA® (dimethyl fumarate)
TECFIDERA, a treatment for relapsing forms of multiple sclerosis (MS) in adults, is the most prescribed oral medication for relapsing MS in the world and has been shown to reduce the rate of MS relapses, slow the progression of disability and impact the number of MS brain lesions, while demonstrating a well-characterized safety profile in people with relapsing forms of MS. TECFIDERA is approved in 69 countries, and more than 500,000 patients have been treated with it, representing more than 1,000,000 patient-years of exposure across clinical trial use and patients prescribed TECFIDERA. Of these, 6,335 patients (14,241 patient-years) were from clinical trials.3
TECFIDERA is contraindicated in patients with a known hypersensitivity to dimethyl fumarate or any of the excipients of TECFIDERA. Serious side effects include anaphylaxis and angioedema, and cases of progressive multifocal leukoencephalopathy, a rare opportunistic viral infection of the brain which has been associated with death or severe disability, have been seen with TECFIDERA patients in the setting of lymphopenia. Other serious side effects include a decrease in mean lymphocyte counts during the first year of treatment, herpes zoster and other serious infections, liver injury and flushing. In clinical trials, the most common adverse events associated with TECFIDERA were flushing, abdominal pain, diarrhea and nausea.
For information on TECFIDERA prescribing information in the EU, please visit: https://www.ema.europa.eu/en/medicines/human/EPAR/tecfidera. Please click here for Important Safety Information and full Prescribing Information, including Patient Information for TECFIDERA in the U.S., or visit your respective country’s product website.
https://www.biogenoptions.com/en_us/home/treatment-overview/tecfidera.html
We routinely post information that may be important to investors on our website at www.biogen.com.
Biogen gets CHMP positive recommendation for multiple sclerosis treatment Vumerity
Sep. 17, 2021 9:28 AM ETBiogen Inc. (BIIB)By: Aakash Babu, SA News Editor1 Comment
- Biogen (NASDAQ:BIIB) announces that the Committee for Medicinal Products for Human Use (CHMP) of the EMA has issued a positive opinion for the approval of Vumerity (diroximel fumarate) in the EU.
- https://seekingalpha.com/news/3740837-biogen-gets-chmp-positive-recommendation-for-multiple-sclerosis-treatment-vumerity
- https://seekingalpha.com/symbol/BIIB
- https://www.vumerity.com/
Nucala (mepolizumab)
17 September 2021
GSK receives CHMP positive opinions recommending approval of Nucala (mepolizumab) in three additional eosinophil-driven diseases
For media and investors only
Issued: 17 September 2021, London UK
- If approved in Europe, mepolizumab would be the only treatment indicated for use in four eosinophil-driven diseases
- CHMP positive opinions advance efforts to provide the first targeted treatment for eosinophilic granulomatosis with polyangiitis (EGPA) and the first anti-IL-5 biologic treatment for patients with hypereosinophilic syndrome (HES) or chronic rhinosinusitis with nasal polyps (CRSwNP)
GlaxoSmithKline (GSK) plc today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued positive opinions recommending Nucala (mepolizumab), a monoclonal antibody that targets interleukin-5 (IL-5), for use in three eosinophil-driven diseases; hypereosinophilic syndrome (HES), eosinophilic granulomatosis with polyangiitis (EGPA) and chronic rhinosinusitis with nasal polyps (CRSwNP). The CHMP opinion is one of the final steps in the marketing authorisation procedure prior to approval decision by the European Commission.
The three positive opinions are based on data from pivotal studies investigating the role of targeted IL-5 inhibition with mepolizumab in these eosinophil-driven diseases. Eosinophil-driven diseases are inflammatory conditions associated with elevated levels of eosinophils, a type of white blood cell.
Mepolizumab is already approved for use in Europe as an add-on treatment for patients with severe eosinophilic asthma. Epidemiological, clinical, and pathophysiological studies strongly suggest that CRSwNP and asthma are closely linked and often coexist. Additionally, patients with EGPA frequently have severe asthma. This overlap across eosinophil-driven diseases underscores the importance of understanding the complex role of eosinophils in disease.
Mepolizumab has been studied in over 4,000 patients in a total of 41 clinical trials evaluating the role mepolizumab may play in targeting the underlying cause of inflammation and reducing eosinophils through IL-5 inhibition. Through ongoing research, GSK is committed to improving the lives of those living with disease associated with uncontrolled eosinophilic inflammation, continuously innovating in order to address the unmet needs in this broad patient group.
About mepolizumab
First approved in 2015 for severe eosinophilic asthma (SEA), mepolizumab is the first-in-class monoclonal antibody that targets IL-5. It is believed to work by preventing IL-5 from binding to its receptor on the surface of eosinophils, reducing blood eosinophils and maintaining them within normal levels. A normal level of blood eosinophils being less than 500 eosinophils/microlitre. The mechanism of action for mepolizumab has not been definitively established.
Mepolizumab has been developed for the treatment of diseases that are driven by inflammation caused by eosinophils. It has been studied in over 4,000 patients in 41 clinical trials across a number of eosinophilic indications and has been approved under the brand name Nucala in the US, Europe and in over 25 other markets, as an add-on maintenance treatment for patients with SEA. Mepolizumab is approved in 17 markets, including Europe and the US, for paediatric use in SEA from ages 6-17 years, with approval in an additional 7 markets for use in patients with SEA aged 12-17 years. The first approval for mepolizumab in CRSwNP was granted by the FDA in July 2021. In a total of 13 markets including the US, Japan and Canada it is approved for use in adult patients with EGPA. Mepolizumab was approved for use in HES in the US in September 2020, followed by Brazil in February 2021, Argentina in May 2021 and Canada in September 2021. Mepolizumab is currently being investigated in COPD. It is not currently approved for use in COPD anywhere in the world.
For further information please visit www.gsk.com/about-us.
European advisory group backs GlaxoSmithKline's Nucala in three eosinophil-driven diseases
Sep. 17, 2021 8:56 AM ETGlaxoSmithKline plc (GSK)
By: Mamta Mayani, SA News Editor
GlaxoSmithKline (NYSE:GSK) announces that the EMA's Committee for Medicinal Products for Human Use (CHMP) has issued positive opinions recommending Nucala (mepolizumab) for use in three eosinophil-driven diseases; hypereosinophilic syndrome (HES), eosinophilic granulomatosis with polyangiitis (EGPA) and chronic rhinosinusitis with nasal polyps (CRSwNP).
- https://seekingalpha.com/news/3740811-european-advisory-group-backs-glaxosmithklines-nucala-in-three-eosinophil-driven-diseases
- https://seekingalpha.com/symbol/GSK
- https://www.nucala.com/
JYSELECA® (FILGOTINIB)
GALAPAGOS ANNOUNCES POSITIVE CHMP OPINION FOR JYSELECA® (FILGOTINIB) FOR THE TREATMENT OF ADULTS WITH MODERATELY TO SEVERELY ACTIVE ULCERATIVE COLITIS
Mechelen, Belgium; 17 September 2021; 13.15 CET; Galapagos NV (Euronext & Nasdaq: GLPG) announced today that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion for Jyseleca® (filgotinib), a once-daily, oral, JAK1 preferential inhibitor for the treatment of adult patients with moderately to severely active ulcerative colitis (UC) who have had an inadequate response with, lost response to, or were intolerant to either conventional therapy or a biologic agent. Following this positive opinion, a final decision from the European Commission is expected later this year.
The CHMP positive opinion is based on data from the pivotal Phase 2b/3 SELECTION program, which evaluated filgotinib as an induction and maintenance therapy in adult patients with moderately to severely active UC who have failed conventional therapy or biologics. SELECTION comprised two placebo-controlled induction studies, one in biologic-naive patients and the other in biologic-experienced patients, followed by a 47-week maintenance study for those who responded to filgotinib after 10 weeks. Responders to placebo continued on blinded placebo during the maintenance phase. The trial was recently published in The Lancet1.
Dr Walid Abi-Saab, Chief Medical Officer at Galapagos, said: “Ulcerative colitis can have significant and profound effects on the people who suffer with the condition. Persistent inflammation and uncontrolled disease mean patients may experience debilitating relapses, may need increasing doses of steroids and in some instances may require surgery, which impacts them not only physically, but also psychologically. Today’s decision brings us one step closer to providing a new treatment option for people living with this chronic disease.”
The CHMP positive opinion will now be reviewed by the European Commission and a decision is expected before year end 2021. This positive opinion follows the previous approval of filgotinib for the treatment of patients with moderate to severe active rheumatoid arthritis. The use of filgotinib for UC is investigational and is not approved anywhere globally.
About filgotinib
Filgotinib is approved and marketed as Jyseleca (200mg and 100mg tablets) in the European Union, Great Britain, and Japan for the treatment of adults with moderate to severe active rheumatoid arthritis (RA) who have responded inadequately or are intolerant to one or more disease modifying anti-rheumatic drugs (DMARDs). Filgotinib may be used as monotherapy or in combination with methotrexate (MTX). The European Summary of Product Characteristics for filgotinib, which includes contraindications and special warnings and precautions, is available at www.ema.europa.eu. The interview form from the Japanese Ministry of Health, Labour and Welfare is available at www.info.pmda.go.jp. The individual Great Britain and Northern Ireland Summary of Product Characteristics can be found at www.medicines.org.uk/emc and www.emcmedicines.com/en-GB/northernireland, respectively. Applications have been submitted to the European Medicines Agency (EMA), the UK’s Medicines and Healthcare products Regulatory Agency (MHRA), and Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) for the treatment of adults with moderately to severely active ulcerative colitis (UC) who have had an inadequate response with, lost response to, or were intolerant to either conventional therapy or a biologic agent and are currently under review. Filgotinib is not approved in any other countries.
Jyseleca® is a trademark of Galapagos NV and Gilead Sciences, Inc. or its related companies.
About the filgotinib collaboration
Gilead and Galapagos NV are collaborative partners in the global development and commercialization of filgotinib. Galapagos will be responsible for the commercialization of filgotinib in Europe (transition anticipated to be completed by end of 2021), while Gilead will remain responsible for filgotinib outside of Europe, including in Japan, where filgotinib is co-marketed with Eisai. Filgotinib in UC has been filed in Europe, Great-Britain and Japan, and a global Phase 3 program is ongoing in Crohn’s Disease. More information about clinical trials can be accessed at https://www.clinicaltrials.gov.
More information at www.glpg.com.
Galapagos' filgotinib wins positive CHMP opinion for treatment of ulcerative colitis
Sep. 17, 2021 8:41 AM ET
By: Mamta Mayani, SA News Editor
- Galapagos (NASDAQ:GLPG) announces that the EMA's Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion for Jyseleca (filgotinib), a once-daily, oral, JAK1 preferential inhibitor for the treatment of adult patients with moderately to severely active ulcerative colitis (UC).
- https://seekingalpha.com/news/3740800-galapagos-filgotinib-wins-positive-chmp-opinion-for-treatment-of-ulcerative-colitis
- https://seekingalpha.com/symbol/GLPG
- https://seekingalpha.com/symbol/GILD
- https://www.glpg.com/
IMFINZI® (durvalumab) oleclumab, an anti-CD73 monoclonal antibody, or monalizumab, an anti-NKG2A monoclonal antibody, in combination with IMFINZI® (durvalumab)
Imfinzi combined with novel immunotherapies improved clinical outcomes for patients with unresectable, Stage III non-small cell lung cancer
PUBLISHED17 September 2021
17 September 2021 12:30 BST
COAST Phase II trial showed oleclumab or monalizumab in combination with Imfinzi significantly delayed disease progression and increased objective response rate
First effectiveness data from PACIFIC-R reinforced long-term benefit of Imfinzi in the real-world setting
Results from the large, randomised COAST Phase II trial showed oleclumab, an anti-CD73 monoclonal antibody, or monalizumab, an anti-NKG2A monoclonal antibody, in combination with Imfinzi (durvalumab) improved progression-free survival (PFS) and objective response rate (ORR) compared to Imfinzi alone in patients with unresectable, Stage III non-small cell lung cancer (NSCLC) who had not progressed after concurrent chemoradiation therapy (CRT).
After a median follow-up of 11.5 months, the results of an interim analysis showed Imfinzi in combination with oleclumab reduced the risk of disease progression or death by 56% (hazard ratio [HR] of 0.44; 95% confidence interval [CI] 0.26-0.75), and in combination with monalizumab by 35% (HR of 0.65; 95% CI 0.49-0.85), when compared to Imfinzi alone in Stage III NSCLC patients following CRT. The 10-month PFS rate was 64.8% for the durvalumab plus oleclumab combination and 72.7% for durvalumab plus monalizumab, versus 39.2% with durvalumab alone.
The results, presented during the European Society for Medical Oncology (ESMO) Congress 2021 today, also showed an increase in the primary endpoint of confirmed ORR for Imfinzi plus oleclumab over Imfinzi alone (30% vs. 18%) and for Imfinzi plus monalizumab over Imfinzi alone (36% vs. 18%).
One in four patients with NSCLC are diagnosed at Stage III, where the majority of tumours are unresectable (cannot be removed with surgery).1,2 Imfinzi after CRT is the global standard of care for patients in this setting, based on the PACIFIC Phase III trial.3-5
PACIFIC-real world observational study (PACIFIC-R) demonstrated benefit of Imfinzi in real-world setting at the ESMO Congress 2021
Data from a planned analysis of real-world PFS (rwPFS) from the PACIFIC-R observational study was also presented during ESMO, showing the first effectiveness data from over a thousand patients with unresectable, Stage III NSCLC who were treated with Imfinzi in the real-world setting as part of AstraZeneca’s global PACIFIC Early Access Programme. The analysis showed a median rwPFS of 21.7 months in the real-world setting.
In comparison, a median PFS of 16.9 months was observed among patients treated with Imfinzi in the randomised, double-blinded, placebo-controlled PACIFIC Phase III trial. These results demonstrate the long-term effectiveness of Imfinzi in this real-world patient population and reinforce the PACIFIC regimen as the established standard of care today following platinum-based CRT.
Imfinzi is approved in the curative-intent setting of unresectable, Stage III NSCLC after CRT in the US, Japan, China, across the EU and in many other countries with more than 80,000 patients treated with Imfinzi in this setting since its first approval in February 2018. Imfinzi is also approved for the 1st-line treatment of extensive-stage small cell lung cancer (ES-SCLC) in combination with etoposide and either carboplatin or cisplatin in more than 55 countries, including the US, Japan, China and across the EU, based on the CASPIAN Phase III trial.
AstraZeneca has several ongoing registrational trials focused on evaluating Imfinzi in earlier stages of lung cancer, including in potentially curative settings (PACIFIC-2, 4 and 5, MERMAID-1 and 2, AEGEAN, ADJUVANT BR.31, and ADRIATIC Phase III trials). The Company is also testing novel combinations with Imfinzi in the Phase II NeoCOAST trial in the neoadjuvant early-stage setting.
Imfinzi
Imfinzi is a human monoclonal antibody that binds to PD-L1 and blocks the interaction of PD-L1 with PD-1 and CD80, countering the tumour’s immune-evading tactics and releasing the inhibition of immune responses.
In addition to approvals in unresectable, Stage III NSCLC and ES-SCLC, Imfinzi is also approved for previously treated patients with advanced bladder cancer in several countries.
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 NSCLC, SCLC, bladder cancer, hepatocellular carcinoma, biliary tract cancer (a form of liver cancer), oesophageal cancer, gastric and gastroesophageal cancer, cervical cancer, ovarian cancer, endometrial cancer, and other solid tumours.
Oleclumab
Oleclumab is a potentially first-in-class, anti-CD73 monoclonal antibody that selectively binds to and inhibits the activity of CD73. CD73 is a cell surface enzyme which is overexpressed in the tumour microenvironment and promotes tumour growth by limiting anti-tumour immunity via the adenosine receptor pathway.
Preclinical studies have demonstrated that CD73 blockade improved anti-tumour activity in combination with radiotherapy, chemotherapy and immunotherapy. Oleclumab is also being examined in various Phase II trials for solid tumour malignancies.
Monalizumab
Monalizumab is a potentially first-in-class, anti-NKG2A antibody. NKG2A is a checkpoint receptor expressed on tumour-infiltrating cytotoxic T-cells and natural killer cells that inhibits their anti-cancer functions.
Monalizumab is also being studied in the ongoing INTERLINK-1 Phase III trial, evaluating monalizumab in combination with cetuximab for the treatment of patients with previously-treated recurrent or metastatic head and neck squamous cell carcinoma.
Monalizumab is being developed in collaboration with Innate Pharma. AstraZeneca obtained full oncology rights to monalizumab in October 2018 through a co-development and commercialisation agreement initiated in 2015.
Please visit astrazeneca.com
AstraZeneca's Imfinzi combo improves clinical outcomes in lung cancer patients
Sep. 17, 2021 8:17 AM ET AstraZeneca PLC (AZN)Innate Pharma S.A. (IPHA) By: Mamta Mayani, SA News Editor
- AstraZeneca (NASDAQ:AZN) announces that Imfinzi (durvalumab) in combination with oleclumab or monalizumab in Phase II COAST trial improved progression-free survival (PFS) and objective response rate (ORR) compared to Imfinzi alone in patients with unresectable, Stage III non-small cell lung cancer (NSCLC) who had not progressed after concurrent chemoradiation therapy (CRT).
- https://seekingalpha.com/news/3740782-astrazenecas-imfinzi-combo-improved-clinical-outcomes-in-lung-cancer-patients
- https://seekingalpha.com/symbol/AZN
- https://www.imfinzi.com/
- https://seekingalpha.com/symbol/IPHA
MONALIZUMAB DATA FROM COAST TRIAL PRESENTED AT ESMO CONGRESS 2021
Fri, 09/17/2021 - 13:45
Innate Pharma SA (Euronext Paris: IPH; Nasdaq: IPHA) (“Innate” or the “Company”) today announced that AstraZeneca (LSE/STO/Nasdaq: AZN) presented results from the randomized COAST Phase 2 trial during the European Society for Medical Oncology (ESMO) Congress 2021 on September 17, 2021.
In particular, the results of the interim analysis showed monalizumab in combination with durvalumab improved progression-free survival (PFS) and objective response rate (ORR) compared to durvalumab alone in patients with unresectable, Stage III non-small cell lung cancer (NSCLC) who had not progressed after concurrent chemoradiation therapy (CRT). Monalizumab, Innate’s lead partnered asset, is a potentially first-in-class immune checkpoint inhibitor targeting NKG2A receptors expressed on tumor infiltrating cytotoxic CD8+ T cells and NK cells.
To read more about the Phase 2 COAST results, please see AstraZeneca’s press release here.
https://www.innate-pharma.com/products/monalizumab
https://www.innate-pharma.com/products/lacutamab
https://www.innate-pharma.com/
LUMAKRAS™ (Sotorasib) Combined With Vectibix® (Panitumumab)
LUMAKRAS™ (Sotorasib) Combined With Vectibix® (Panitumumab) Showed Encouraging Efficacy And Safety In Patients With KRAS G12C-Mutated Colorectal Cancer
New Data at ESMO 2021 Support Initiation of Phase 3 Trial of LUMAKRAS Plus Vectibix in Patients With 3L+ Colorectal Cancer
Amgen is Leading the Largest and Most Comprehensive Development Program in Patients with the KRAS G12C Mutation
THOUSAND OAKS, Calif., Sept. 16, 2021 /PRNewswire/ -- Amgen (NASDAQ: AMGN) today announced the first combination study results from the Phase 1b/2 CodeBreaK 101 study, the most comprehensive global clinical development program in patients with KRAS G12C-mutated advanced colorectal cancer (CRC). These new data show that combining LUMAKRAS™ (sotorasib) with Vectibix® (panitumumab), Amgen's monoclonal antibody epidermal growth factor receptor (EGFR) inhibitor, demonstrated encouraging efficacy and safety. Overall, the objective response rate (ORR) was 27% (confirmed and unconfirmed) among 26 patients in the efficacy analysis set (which included 5 patients who had progressed with prior sotorasib monotherapy). The disease control rate (DCR) was 81%. ORR and DCR were secondary endpoints. In the expansion cohort of sotorasib-naïve patients with refractory CRC (n=18), 33% of patients experienced a response (confirmed and unconfirmed). These data are being featured during the European Society of Medical Oncology 2021 (ESMO21) Virtual Congress.
Advancing Tarlatamab (formerly AMG 757) and AMG 404 in Small Cell Lung Cancer
In addition to the LUMAKRAS combination research, a presentation will detail the design of an ongoing study of half-life extended (HLE) bispecific T cell engager (BiTE®) molecule tarlatamab with anti-PD-1 antibody AMG 404 in patients with small cell lung cancer. The multicenter, open-label, Phase 1b study will evaluate the safety and tolerability of the combination and determine dosing as primary objectives, as well as examine preliminary antitumor activity and pharmacokinetics as secondary objectives.
About LUMAKRASTM (sotorasib)
Amgen took on one of the toughest challenges of the last 40 years in cancer research by developing LUMAKRAS, a KRASG12C inhibitor.1 LUMAKRAS has demonstrated a positive benefit-risk profile with rapid, deep and durable anticancer activity in patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring the KRAS G12C mutation with a once daily oral formulation.2
In May 2021, LUMAKRAS was the first KRASG12C inhibitor to receive regulatory approval anywhere in the world with its approval in the U.S., under accelerated approval. LUMAKRAS is also approved in the United Arab Emirates, and in Great Britain and Canada under Project Orbis.
Amgen is progressing the largest and broadest global KRASG12C inhibitor development program with unparalleled speed and exploring more than 10 sotorasib combination regimens, including triplets, with clinical trial sites spanning five continents. To date, LUMAKRAS has treated almost 3,000 patients around the world through the clinical development program and commercial use.
In the U.S., LUMAKRAS was reviewed by the FDA under its Real-Time Oncology Review (RTOR), a pilot program that aims to explore a more efficient review process that ensures safe and effective treatments are made available to patients as early as possible. Amgen is participating in the FDA's Project Orbis initiative and through the initiative, has Marketing Authorization Applications (MAAs) for sotorasib in review in Australia and Brazil. Additionally, Amgen has submitted an MAA in the European Union, Japan, Switzerland, South Korea, Singapore, Israel, Turkey, Taiwan, Colombia, Thailand, Mexico and Hong Kong.
LUMAKRAS is also being studied in multiple other solid tumors.1
About CodeBreaK
The CodeBreaK clinical development program for Amgen's drug sotorasib is designed to treat patients with an advanced solid tumor with the KRAS G12C mutation and address the longstanding unmet medical need for these cancers. As the most advanced KRASG12C inhibitor clinical development program, CodeBreaK has enrolled more than 800 patients across 13 tumor types since its inception.
CodeBreaK 100, the Phase 1 and 2, first-in-human, open-label multicenter study, enrolled patients with KRAS G12C-mutant solid tumors. Eligible patients must have received a prior line of systemic anticancer therapy, consistent with their tumor type and stage of disease. The primary endpoint for the Phase 2 study was centrally assessed objective response rate. The Phase 2 trial in NSCLC enrolled 126 patients, 124 of whom had centrally evaluable lesions by RECIST at baseline. The Phase 2 trial in colorectal cancer (CRC) is fully enrolled and results have been submitted for publication.
A global Phase 3 randomized active-controlled study comparing sotorasib to docetaxel in patients with KRAS G12C-mutated NSCLC (CodeBreaK 200) has completed enrollment. Amgen also has several Phase 1b studies investigating sotorasib monotherapy and sotorasib combination therapy across various advanced solid tumors (CodeBreaK 101) open for enrollment. A Phase 2 randomized study will evaluate sotorasib in patients with stage IV KRAS G12C-mutated NSCLC in need of first-line treatment (CodeBreaK 201).
For information, please visit www.hcp.codebreaktrials.com.
LUMAKRASTM (sotorasib) U.S. Indication
LUMAKRASTM is indicated for the treatment of adult patients with KRAS G12C-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC), as determined by an FDA-approved test, who have received at least one prior systemic therapy.
This indication is approved under accelerated approval based on overall response rate (ORR) and duration of response (DOR). Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
Please see LUMAKRASTM full Prescribing Information.
About Vectibix® (panitumumab)
Vectibix is the first fully human monoclonal anti-EGFR antibody approved by the FDA for the treatment of mCRC. Vectibix was approved in the U.S. in September 2006 as a monotherapy for the treatment of patients with EGFR-expressing mCRC after disease progression after prior treatment with fluoropyrimidine-, oxaliplatin-, and irinotecan-containing chemotherapy.
In May 2014, the FDA approved Vectibix for use in combination with FOLFOX, as first-line treatment in patients with wild-type KRAS (exon 2) mCRC. With this approval, Vectibix became the first-and-only biologic therapy indicated for use with FOLFOX, one of the most commonly used chemotherapy regimens, in the first-line treatment of mCRC for patients with wild-type KRAS mCRC.
In June 2017, the FDA approved a refined indication for Vectibix for use in in patients with wild-type RAS (defined as wild-type in both KRAS and NRAS as determined by an FDA-approved test for this use) mCRC.
INDICATION AND LIMITATION OF USE
Vectibix® is indicated for the treatment of patients with wild-type RAS (defined as wild-type in both KRAS and NRAS as determined by an FDA-approved test for this use) metastatic colorectal cancer (mCRC): as first-line therapy in combination with FOLFOX, and as monotherapy following disease progression after prior treatment with fluoropyrimidine-, oxaliplatin-, and irinotecan-containing chemotherapy.
Limitation of Use: Vectibix® is not indicated for the treatment of patients with RAS mutant mCRC or for whom RAS mutation status is unknown.
To see the Vectibix® Prescribing Information, including Boxed Warning visit www.vectibix.com.
For more information, visit www.amgen.com
View original content to download multimedia:https://www.prnewswire.com/news-releases/lumakras-sotorasib-combined-with-vectibix-panitumumab-showed-encouraging-efficacy-and-safety-in-patients-with-kras-g12c-mutated-colorectal-cancer-301378188.html
SOURCE Amgen
Amgen's Lumakras + Vectibix shows encouraging efficacy and safety in colorectal cancer
Sep. 16, 2021 5:11 AM ETAmgen Inc. (AMGN)By: Mamta Mayani, SA News Editor
- Amgen (NASDAQ:AMGN) announces the first combination study results from Phase 1b/2 CodeBreaK 101 study in patients with KRAS G12C-mutated advanced colorectal cancer (CRC).
- https://seekingalpha.com/news/3740278-amgens-lumakras-vectibix-shows-encouraging-efficacy-and-safety-in-colorectal-cancer
- https://seekingalpha.com/symbol/AMGN
- https://www.vectibix.com/
- https://www.lumakras.com/
Bemarituzumab (FPA144)
Zai Lab Announces Breakthrough Therapy Designation Granted for Bemarituzumab (FPA144) in China
September 14, 2021 at 7:30 AM EDTPDF VersionPotential first-in-class therapy for a subset of gastric and gastroesophageal (GEJ) cancers that overexpress fibroblast growth factor receptor 2 (FGFR2b)
SHANGHAI and SAN FRANCISCO, Sept. 14, 2021 (GLOBE NEWSWIRE) -- Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688), an innovative commercial-stage biopharmaceutical company, today announced that the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) granted Breakthrough Therapy Designation for investigational bemarituzumab (FPA144), for first-line treatment for patients with FGFR2b overexpressing and human epidermal growth factor receptor (HER2)-negative metastatic and locally advanced gastric and GEJ cancers in combination with modified FOLFOX6 (fluoropyrimidine, leucovorin, and oxaliplatin).
“In granting Breakthrough Therapy Designation, we are pleased to see that the CDE recognizes the promise of bemarituzumab. In combination with chemotherapy, bemarituzumab demonstrated clinically meaningful outcomes in key endpoints for patients with advanced gastric or GEJ cancer as a frontline therapy,” said Alan Sandler, M.D., president and head of global development, oncology, at Zai Lab. “We look forward to working with regulatory authorities in China as we advance bemarituzumab into global, registrational studies.”
The designation is supported by results from the Phase 2 FIGHT study, which evaluated bemarituzumab plus chemotherapy (modified FOLFOX6) versus chemotherapy alone in patients with FGFR2b overexpression, HER2-negative frontline advanced gastric or GEJ cancer. All three efficacy endpoints in the FIGHT trial – PFS, OS and ORR – achieved pre-specified statistical significance in the bemarituzumab arm compared to the placebo arm. Additional analysis showed a positive correlation between benefit and the prevalence of FGFR2b overexpression tumor cells, affirming both the importance of the FGFR2b target and the activity of bemarituzumab against this target. The Breakthrough Therapy Designation was granted based upon this subset of patients, based on IHC testing, showing at least 10% of tumor cells overexpressing FGFR2b.
More than one million new gastric cancer cases are diagnosed annually, and approximately half of all gastric cancer cases occur in China1. Nearly 88% of patients with advanced gastric and GEJ cancers are HER2-negative, and approximately 30% of these patients present with FGFR2b overexpression.
The Breakthrough Therapy Designation review policy is designed to promote the research and creation of drugs with apparent clinical advantages, which are intended for the prevention or treatment of serious life-threatening diseases or diseases which severely impact the quality of life for which there is no existing treatment or where sufficient evidence indicates advantages of the novel drug over currently available treatment options. Drugs that have been granted the Breakthrough Therapy Designation are prioritized by the CDE in communications, and in receiving guidance to promote the drug development progress.
Source: (1) Globocan 2020.
About Bemarituzumab
Bemarituzumab (anti-FGFR2b), also called FPA144, is a potential first-in-class investigational targeted antibody that is designed to block fibroblast growth factors (FGFs) from binding and activating FGFR2b, inhibiting several downstream pro-tumor signaling pathways and potentially slowing cancer progression. Bemarituzumab is being developed in gastric and GEJ cancers as a targeted therapy for tumors that overexpress FGFR2b.
Zai Lab has an exclusive license to develop and commercialize bemarituzumab in Greater China. Zai Lab collaborated with Five Prime (later acquired by Amgen) on the Phase 2 FIGHT trial in Greater China.
Initiation of the registrational program of bemarituzumab by Amgen is planned for the fourth quarter of 2021. Planning is underway for bemarituzumab clinical studies in other solid tumors, including squamous NSCLC.
Bemarituzumab (FGFR2b)
Overview
Bemarituzumab is a humanized monoclonal antibody specific to the human fibroblast growth factor receptor 2b (FGFR2b) in clinical development as a targeted therapy for tumors that overexpress FGFR2b, including gastric and gastroesophageal junction cancers. We have an exclusive license for bemarituzumab in mainland China, Hong Kong (China), Macau (China) and Taiwan region from Five Prime Therapeutics, Inc. (Five Prime). We are collaborating with Five Prime to conduct the randomized, controlled Phase III clinical trial FIGHT to evaluate bemarituzumab in combination with modified FOLFOX6 chemotherapy as a front-line therapy for the treatment of gastric and gastro-esophageal junction cancers. In a Phase I trial, bemarituzumab demonstrated monotherapy activity in heavily pre-treated patients with FGFR2b-positive gastric cancer and did not cause some of the toxicities that had been seen with less selective small molecule FGFR2 inhibitors. FGFR2b is a splice variant of a receptor for some members of the fibroblast growth factor (FGF) family. An estimated 10% of patients with gastric cancer have tumors that overexpress FGFR2b or have FGFR2 gene amplification, which is associated with poor prognosis.
http://www.zailaboratory.com/pipeline/info.aspx?lcid=5#d
For additional information about the company, please visit www.zailaboratory.com
http://www.zailaboratory.com/pipeline/list.aspx
Zai Lab nabs Breakthrough Therapy tag in China for GIT cancer therapy
Sep. 14, 2021 8:56 AM ET Zai Lab Limited (ZLAB) By: Dulan Lokuwithana, SA News Editor
- Zai Lab (NASDAQ:ZLAB) has been granted the Breakthrough Therapy Designation in China for experimental therapy bemarituzumab (FPA144) and modified FOLFOX6 combo as a first-line treatment for patients with certain forms of gastric and GEJ cancers.
- https://seekingalpha.com/news/3739480-zai-lab-nabs-breakthrough-therapy-tag-in-china-for-git-cancer-therapy
- https://seekingalpha.com/symbol/ZLAB
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab)
Five-Year Data from CheckMate -214 Show Opdivo (nivolumab) Plus Yervoy (ipilimumab) Demonstrates Longest Median Overall Survival Currently Reported in Phase 3 Trial of Patients with Previously Untreated Advanced or Metastatic Renal Cell Carcinoma
09/16/2021CATEGORY:
Nearly half of patients treated with the dual immunotherapy combination were alive at five years from the start of therapy in CheckMate -214 trial
Opdivo plus Yervoy continued to show durable responses, with the median duration of response not reached among all randomized patients after five years of follow-up
Data to be presented at the European Society for Medical Oncology Virtual Congress 2021
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced that Opdivo (nivolumab) plus Yervoy (ipilimumab) continued to demonstrate durable, long-term survival in the Phase 3 CheckMate -214 trial, with a five-year survival rate of 48% in patients with previously untreated advanced or metastatic renal cell carcinoma (RCC). After a median follow-up of 67.7 months, Opdivo plus Yervoy maintained superior overall survival (OS) and response benefits versus sunitinib in both patients with intermediate- and poor-risk prognostic factors, the primary endpoint population, and across all randomized patients.
In intermediate- and poor-risk patients (n=847), Opdivo plus Yervoy (n=425) maintained treatment effects over five years, with ongoing improvements in OS and overall response rate (ORR), two of the trial’s primary endpoints, as well as in supportive endpoints:
- OS: The median OS was 47.0 months for intermediate- and poor-risk patients treated withOpdivo plus Yervoyversus 26.6 months with sunitinib (Hazard Ratio [HR] 0.68; 95% Confidence Interval [CI]: 0.58 to 0.81), and five-year survival rates were 43% and 31%, respectively.
- ORR: ORR benefits were maintained with Opdivoplus Yervoy compared to sunitinib (42% vs. 27%). In addition, more patients treated with the combinationachieved complete responses (11% vs. 2%).
- Duration of response (DOR): For patients treated with Opdivoplus Yervoy, median DOR was not reached, compared to 19.7 months with sunitinib.
An analysis of all randomized patients, or the intent-to-treat (ITT) population (n=1,096), similarly showed long-term benefits with Opdivo plus Yervoy (n=550):
- OS: Opdivoplus Yervoydemonstrated a median OS of 55.7 months in the ITT population, representing the longest survival outcome reported to date in a Phase 3 trial in advanced RCC, compared to 38.4 months with sunitinib (HR 0.72; 95% CI: 0.62 to 0.85). Five-year OS rates were 48% for patients treated with the dual immunotherapy combination and 37% for those receiving sunitinib.
- ORR: Opdivoplus Yervoy continued to show a higher ORR (39% vs. 32%) and complete response rate than sunitinib (12% vs. 3%). Also, more patients treated with the combinationachieved complete responses and subsequently did not progress (9.6% vs. 2.4%).
- DOR: Median DOR was not reached with Opdivoplus Yervoy and was 24.8 months with sunitinib.
Opdivo plus Yervoy-based combinations have shown significant improvements in OS in six Phase 3 clinical trials in five tumors to date: advanced renal cell carcinoma, non-small cell lung cancer, metastatic melanoma, malignant pleural mesothelioma and esophageal squamous cell carcinoma.
About CheckMate -214
CheckMate -214 is a Phase 3, randomized, open-label study evaluating the combination of Opdivo plus Yervoy versus sunitinib in patients with previously untreated advanced or metastatic renal cell carcinoma (RCC). Patients in the combination group (n=550) received Opdivo 3 mg/kg plus Yervoy 1 mg/kg every three weeks for four doses followed by Opdivo 3 mg/kg every two weeks. Patients in the comparator group (n=546) received sunitinib 50 mg once daily for four weeks, followed by two weeks off before continuation of treatment. Patients were treated until progression or unacceptable toxic effects for up to two years. The primary endpoints of the trial are overall survival (OS), progression-free survival (PFS) and objective response rate (ORR) in an intermediate- to poor-risk patient population (approximately 75% of patients). ORR was assessed by independent radiology review committee (IRRC).
About Opdivo
Opdivo is a programmed death-1 (PD-1) immune checkpoint inhibitor that is designed to uniquely harness the body’s own immune system to help restore anti-tumor immune response. By harnessing the body’s own immune system to fight cancer, Opdivo has become an important treatment option across multiple cancers.
Opdivo’s leading global development program is based on Bristol Myers Squibb’s scientific expertise in the field of Immuno-Oncology, and includes a broad range of clinical trials across all phases, including Phase 3, in a variety of tumor types. To date, the Opdivo clinical development program has treated more than 35,000 patients. The Opdivo trials have contributed to gaining a deeper understanding of the potential role of biomarkers in patient care, particularly regarding how patients may benefit from Opdivo across the continuum of PD-L1 expression.
In July 2014, Opdivo was the first PD-1 immune checkpoint inhibitor to receive regulatory approval anywhere in the world. Opdivo is currently approved in more than 65 countries, including the United States, the European Union, Japan and China. In October 2015, the Company’s Opdivo and Yervoy combination regimen was the first Immuno-Oncology to receive regulatory approval for the treatment of metastatic melanoma and is currently approved in more than 50 countries, including the United States and the European Union.
About Yervoy
Yervoy is a recombinant, human monoclonal antibody that binds to the cytotoxic T-lymphocyte-associated antigen-4 (CTLA-4). CTLA-4 is a negative regulator of T-cell activity. Yervoy binds to CTLA-4 and blocks the interaction of CTLA-4 with its ligands, CD80/CD86. Blockade of CTLA-4 has been shown to augment T-cell activation and proliferation, including the activation and proliferation of tumor infiltrating T-effector cells. Inhibition of CTLA-4 signaling can also reduce T-regulatory cell function, which may contribute to a general increase in T-cell responsiveness, including the anti-tumor immune response. On March 25, 2011, the U.S. Food and Drug Administration (FDA) approved Yervoy 3 mg/kg monotherapy for patients with unresectable or metastatic melanoma. Yervoy is approved for unresectable or metastatic melanoma in more than 50 countries. There is a broad, ongoing development program in place for Yervoy spanning multiple tumor types.
INDICATIONS
OPDIVO® (nivolumab) is indicated for the treatment of patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express PD-L1 (≥1%) as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab) and 2 cycles of platinum-doublet chemotherapy, is indicated for the first-line treatment of adult patients with metastatic or recurrent non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab) is indicated for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving OPDIVO.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with unresectable malignant pleural mesothelioma (MPM).
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of patients with intermediate or poor risk advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab), in combination with cabozantinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab) is indicated for the treatment of patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy.
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with classical Hodgkin lymphoma (cHL) that has relapsed or progressed after autologous hematopoietic stem cell transplantation (HSCT) and brentuximab vedotin or after 3 or more lines of systemic therapy that includes autologous HSCT. This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab) is indicated for the treatment of patients with recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN) with disease progression on or after platinum-based therapy.
OPDIVO® (nivolumab) is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
OPDIVO® (nivolumab), as a single agent, is indicated for the adjuvant treatment of patients with urothelial carcinoma (UC) who are at high risk of recurrence after undergoing radical resection of UC.
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of adult and pediatric (12 years and older) patients with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection.
OPDIVO® (nivolumab) is indicated for the treatment of patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) after prior fluoropyrimidine- and platinum-based chemotherapy.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of completely resected esophageal or gastroesophageal junction cancer with residual pathologic disease in patients who have received neoadjuvant chemoradiotherapy (CRT).
OPDIVO® (nivolumab), in combination with fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the treatment of patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma.
Please see US Full Prescribing Information for OPDIVO and YERVOY.
For more information about Bristol Myers Squibb, visit us at BMS.com
Bristol Myers' Opdivo + Yervoy shows longest overall survival in late-stage kidney cancer study
Sep. 16, 2021 8:18 AM ETBristol-Myers Squibb Company (BMY)By: Mamta Mayani, SA News Editor4 Comments
- Bristol Myers Squibb (NYSE:BMY) announces that Opdivo (nivolumab) plus Yervoy (ipilimumab) continued to demonstrate durable, long-term survival in Phase 3 CheckMate -214 trial, with a five-year survival rate of 48% in patients with previously untreated advanced or metastatic renal cell carcinoma (RCC).
- https://seekingalpha.com/news/3740391-bristol-myers-opdivo-yervoy-shows-longest-median-overall-survival-in-late-stage-kidney-cancer-study
- https://seekingalpha.com/symbol/BMY
- https://www.bms.com/
- https://www.opdivo.com/about-opdivo/how-the-combination-works-combinationtherapy
BRUKINSA® (Zanubrutinib)
U.S. FDA Grants BRUKINSA® (Zanubrutinib) Accelerated Approval in Relapsed or Refractory Marginal Zone Lymphoma
Sep 15, 2021 7:00 AM
This marks the third FDA approval for BRUKINSA and first approval in marginal zone lymphoma
Twenty percent of patients achieved complete remission with single-agent BRUKINSA
BRUKINSA was generally well-tolerated, consistent with its known safety profile
CAMBRIDGE, Mass. & BEIJING--(BUSINESS WIRE)-- BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a global biotechnology company focused on developing and commercializing innovative medicines worldwide, today announced that BRUKINSA® (zanubrutinib) has received accelerated approval from the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with relapsed or refractory (R/R) marginal zone lymphoma (MZL) who have received at least one anti-CD20-based regimen.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210915005310/en/
(Photo: Business Wire)
This accelerated approval is based on overall response rate (ORR). Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
“We are excited about the FDA’s approval for BRUKINSA in patients with previously treated marginal zone lymphoma, a significant milestone that was made possible by the diligent BeiGene team, the dedicated investigators, and the participating patients and their families. The MAGNOLIA trial results provided additional evidence that the selective design of BRUKINSA can be translated to improved treatment outcomes for these patients,” said Jane Huang, M.D., Chief Medical Officer, Hematology at BeiGene. “The ongoing evaluation of BRUKINSA in its broad global clinical program will enable us to further understand this potentially best-in-class BTK inhibitor and its impact on patients. Since the initial FDA approval in November 2019, BRUKINSA has been granted 12 approvals in four indications globally. We will continue to execute on our mission to improve access to innovative and quality treatments for cancer patients worldwide.”
“BTK plays a critical role in B-cell receptor signaling, a driver in the development of marginal zone lymphoma. In the MAGNOLIA trial, BRUKINSA demonstrated impressive overall response and complete remission rates, with responses observed in all MZL subtypes. In addition, this next-generation BTK inhibitor was well-tolerated in these patients, with low rate of discontinuation due to adverse reactions. We are optimistic that BRUKINSA will bring clinically meaningful benefit to patients with relapsed or refractory marginal zone lymphoma,” said Stephen Opat, FRACP, FRCPA, MBBS, Director of Clinical Hematology at Monash Health, Head of Department of Hematology at Monash University, and lead principal investigator of the MAGNOLIA trial.
“The approval of BRUKINSA offers patients with relapsed and refractory marginal zone lymphoma a new treatment option and new hope for improving patient outcomes,” commented Meghan Gutierrez, Chief Executive Officer of the Lymphoma Research Foundation.
The FDA approval of BRUKINSA is based on efficacy results from two single-arm clinical trials, with ORR as assessed by independent review committee (IRC) per 2014 Lugano Classification as the primary endpoint.
About BRUKINSA
BRUKINSA is a small molecule inhibitor of Bruton’s tyrosine kinase (BTK) discovered by BeiGene scientists that is currently being evaluated globally in a broad clinical program as a monotherapy and in combination with other therapies to treat various B-cell malignancies. Because new BTK is continuously synthesized, BRUKINSA was specifically designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity. With differentiated pharmacokinetics compared to other approved BTK inhibitors, BRUKINSA has been demonstrated to inhibit the proliferation of malignant B cells within a number of disease relevant tissues.
BRUKINSA is approved in the following indications and regions:
- For the treatment of mantle cell lymphoma (MCL) in adult patients who have received at least one prior therapy (United States, November 2019)*;
- For the treatment of MCL in adult patients who have received at least one prior therapy (China, June 2020)**;
- For the treatment of chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) in adult patients who have received at least one prior therapy (China, June 2020)**;
- For the treatment of relapsed or refractory MCL (United Arab Emirates, February 2021);
- For the treatment of Waldenström’s macroglobulinemia (WM) in adult patients (Canada, March 2021);
- Registered and reimbursed for the treatment of MCL in patients who have received at least one prior therapy (Israel, April 2021);
- For the treatment of adult patients with WM who have received at least one prior therapy (China, June 2021)**;
- For the treatment of MCL in adult patients who have received at least one prior therapy (Canada, July 2021);
- For the treatment of MCL in adult patients who have received at least one prior therapy (Chile, July 2021);
- For the treatment of adult patients with MCL who have received at least one previous therapy (Brazil, August 2021);
- For the treatment of adult patients with WM (United States, August 2021); and
- For the treatment of adult patients with marginal zone lymphoma (MZL) who have received at least one anti-CD20-based regimen (United States, September 2021)*.
To date, more than 30 marketing authorization applications in multiple indications have been submitted in the United States, China, the European Union, and more than 20 other countries or regions.
* This indication was approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
** This indication was approved under conditional approval. Complete approval for this indication may be contingent upon results from ongoing randomized, controlled confirmatory clinical trials.
Please see full U.S. Prescribing Information at www.beigene.com/PDF/BRUKINSAUSPI.pdf and Patient Information at www.beigene.com/PDF/BRUKINSAUSPPI.pdf.
To learn more about BeiGene, please visit www.beigene.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20210915005310/en/
Source: BeiGene, Ltd.
FDA grants BeiGene's Brukinsa accelerated approval in marginal zone lymphoma
Sep. 15, 2021 7:44 AM ET BeiGene, Ltd. (BGNE)
By: Mamta Mayani, SA News Editor
- BeiGene (NASDAQ:BGNE) announces that Brukinsa (zanubrutinib) has received accelerated approval from the FDA for the treatment of adult patients with relapsed or refractory (R/R) marginal zone lymphoma (MZL) who have received at least one anti-CD20-based regimen.
- https://seekingalpha.com/news/3739857-fda-grants-beigenes-brukinsa-accelerated-approval-in-marginal-zone-lymphoma
- https://seekingalpha.com/symbol/BGNE
- https://www.beigene.com/
Tislelizumab (BGB-A317)
BeiGene Announces U.S. FDA Acceptance of Biologics License Application for Tislelizumab in Esophageal Squamous Cell Carcinoma
Sep 13, 2021 12:00 AM
This marks the first ex-China regulatory filing for tislelizumab, following approval in five indications in China
The accepted BLA, filed in collaboration with Novartis, is supported by the positive global Phase 3 RATIONALE 302 trial in patients with previously treated, advanced or metastatic ESCC and safety data from tislelizumab’s broad clinical program
With its second internally developed medicine filed outside China, BeiGene furthers its commitment to expanding access to innovative treatments for cancer patients worldwide
CAMBRIDGE, Mass. & BEIJING--(BUSINESS WIRE)-- BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a global, science-driven biotechnology company focused on developing innovative and affordable medicines to improve treatment outcomes and access for patients worldwide, today announced that the U.S. Food and Drug Administration (FDA) accepted for review a Biologics License Application (BLA) for its anti-PD-1 antibody tislelizumab as a treatment for patients with unresectable recurrent locally advanced or metastatic esophageal squamous cell carcinoma (ESCC) after prior systemic therapy. The Prescription Drug User Fee Act (PDUFA) target action date is July 12, 2022.
“Our uniquely designed anti-PD-1 antibody tislelizumab has been shown to significantly improve survival compared to chemotherapy for people with a variety of solid tumors and hematologic malignancies. We previously shared the compelling results at ASCO 2021 with tislelizumab significantly prolonging survival and demonstrating a favorable safety profile over chemotherapy in patients with locally advanced or metastatic ESCC, a devastating disease with an average five-year survival rate of just five percent. This BLA acceptance brings us closer to potentially providing tislelizumab as a treatment for these patients in the United States,” said Yong (Ben) Ben, M.D., Chief Medical Officer, Immuno-Oncology at BeiGene. “Tislelizumab is already approved in five indications in China and has the potential to become a preferred immunotherapy option there. We look forward to continued collaboration with Novartis to work to bring access to tislelizumab to patients around the world.”
The BLA submission is based on results from RATIONALE 302, a randomized, open-label, multicenter global Phase 3 trial (NCT03430843) designed to evaluate the efficacy and safety of tislelizumab when compared to investigator’s choice chemotherapy as a second-line treatment for patients with advanced or metastatic ESCC. Results of this trial were presented at the 2021 American Society of Clinical Oncology Annual Meeting (ASCO 2021). The submission also included safety data on 1,972 patients who received tislelizumab as a monotherapy from seven clinical trials.
In addition to the United States, tislelizumab is also under regulatory review in China as a treatment for patients with locally advanced or metastatic ESCC who have disease progression following or are intolerant to first-line standard chemotherapy.
About Tislelizumab
Tislelizumab (BGB-A317) is a humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to FcγR on macrophages. In pre-clinical studies, binding to FcγR on macrophages has been shown to compromise the anti-tumor activity of PD-1 antibodies through activation of antibody-dependent macrophage-mediated killing of T effector cells. Tislelizumab is the first drug from BeiGene’s immuno-oncology biologics program and is being developed internationally as a monotherapy and in combination with other therapies for the treatment of a broad array of both solid tumor and hematologic cancers.
The China National Medical Products Administration (NMPA) has approved tislelizumab in five indications, including full approval for first-line treatment of patients with advanced squamous non-small cell lung cancer (NSCLC) in combination with chemotherapy and for first-line treatment of patients with advanced non-squamous NSCLC in combination with chemotherapy. NMPA also granted conditional approval for the treatment of patients with classical Hodgkin’s lymphoma (cHL) who received at least two prior therapies, for the treatment of patients with locally advanced or metastatic urothelial carcinoma (UC) with PD-L1 high expression whose disease progressed during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy, and for the treatment of patients with hepatocellular carcinoma (HCC) who have received at least one systemic therapy. Full approval for these indications is contingent upon results from ongoing randomized, controlled confirmatory clinical trials.
In addition, three supplemental Biologics License Applications for tislelizumab are under review by the Center for Drug Evaluation (CDE) of the NMPA, including as second- or third-line treatment of patients with locally advanced or metastatic NSCLC who progressed on prior platinum-based chemotherapy, for patients with previously treated, locally advanced unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair-deficient (dMMR) solid tumors and for the treatment of patients with locally advanced or metastatic esophageal squamous cell carcinoma (ESCC) who have disease progression following or are intolerant to first-line standard chemotherapy.
In the U.S., a Biologics License Application for tislelizumab as a treatment for patients with unresectable recurrent locally advanced or metastatic ESCC after prior systemic therapy is currently under review by the U.S. Food and Drug Administration with a PDUFA target action date of July 12, 2022.
BeiGene has initiated or completed 17 potentially registration-enabling clinical trials in China and globally, including 13 Phase 3 trials and four pivotal Phase 2 trials.
In January 2021, BeiGene and Novartis entered into a collaboration and license agreement granting Novartis rights to develop, manufacture, and commercialize tislelizumab in North America, Europe, and Japan.
Tislelizumab is not approved for use outside of China.
About the Tislelizumab Clinical Program
Clinical trials of tislelizumab include:
- Phase 3 trial comparing tislelizumab with docetaxel in the second- or third-line setting in patients with NSCLC (NCT03358875);
- Phase 3 trial comparing tislelizumab to salvage chemotherapy in patients with relapsed or refractory classical Hodgkin Lymphoma (cHL; NCT04486391);
- Phase 3 trial in patients with locally advanced or metastatic urothelial carcinoma (NCT03967977);
- Phase 3 trial of tislelizumab in combination with chemotherapy versus chemotherapy as first-line treatment for patients with advanced squamous NSCLC (NCT03594747);
- Phase 3 trial of tislelizumab in combination with chemotherapy versus chemotherapy as first-line treatment for patients with advanced non-squamous NSCLC (NCT03663205);
- Phase 3 trial of tislelizumab in combination with platinum-based doublet chemotherapy as neoadjuvant treatment for patients with NSCLC (NCT04379635);
- Phase 3 trial of tislelizumab combined with platinum and etoposide versus placebo combined with platinum and etoposide in patients with extensive-stage small cell lung cancer (NCT04005716);
- Phase 3 trial comparing tislelizumab with sorafenib as first-line treatment for patients with hepatocellular carcinoma (HCC; NCT03412773);
- Phase 2 trial in patients with previously treated unresectable HCC (NCT03419897);
- Phase 2 trial in patients with locally advanced or metastatic urothelial bladder cancer (NCT04004221);
- Phase 3 trial comparing tislelizumab with chemotherapy as second-line treatment for patients with advanced esophageal squamous cell carcinoma (ESCC; NCT03430843);
- Phase 3 trial of tislelizumab in combination with chemotherapy as first-line treatment for patients with ESCC (NCT03783442);
- Phase 3 trial of tislelizumab versus placebo in combination with chemoradiotherapy in patients with localized ESCC (NCT03957590);
- Phase 3 trial of tislelizumab combined with chemotherapy versus placebo combined with chemotherapy as first-line treatment for patients with gastric cancer (NCT03777657);
- Phase 2 trial of tislelizumab in patients with relapsed or refractory cHL (NCT03209973);
- Phase 2 trial in patients with MSI-H/dMMR solid tumors (NCT03736889); and
- Phase 3 trial of tislelizumab combined with chemotherapy versus placebo combined with chemotherapy as first-line treatment in patients with nasopharyngeal cancer (NCT03924986).
To learn more about BeiGene, please visit www.beigene.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20210912005031/en/
Source: BeiGene, Ltd.
FDA accepts BLA from BeiGene/Novartis for tislelizumab for esophageal cancer
Sep. 14, 2021 12:10 PM ET BeiGene, Ltd. (BGNE), NVS By: Jonathan M Block, SA News Editor
- The FDA has accepted a BLA filing from BeiGene (BGNE +0.5%) for its anti-PD-1 immune checkpoint inhibitor tislelizumab for the treatment of unresectable recurrent locally advanced or metastatic esophageal squamous cell carcinoma as a second-line therapy.
- https://seekingalpha.com/news/3739572-fda-accepts-bla-from-beigene-novartis-for-tislelizumab-for-esophageal-cancer
- https://seekingalpha.com/symbol/BGNE
- https://seekingalpha.com/symbol/NVS
biotecMAX™ April/May/June/July/Aug 2021 Insight
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab)
Opdivo (nivolumab) Plus Yervoy (ipilimumab) Demonstrates Durable Overall Survival at Three YearsCompared to Chemotherapy in First-Line Unresectable Malignant Pleural Mesothelioma in Phase 3 CheckMate -743 Trial
09/13/2021CATEGORY:
CheckMate -743 remains the only Phase 3 trial to show a survival improvement – now sustained over three years – with first-line immunotherapy treatment in patients with unresectable malignant pleural mesotheliomaData to be featured in a Proffered Paper session during the 2021 European Society for Medical Oncology Virtual CongressMesothelioma is the fourth tumor type in which Opdivo plus Yervoy has demonstrated durable, superior survival with three years or more of follow-upPRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced three-year data from the CheckMate -743 trial that demonstrated a durable survival benefit with first-line treatment with Opdivo (nivolumab) plus Yervoy (ipilimumab) compared to platinum-based standard-of-care chemotherapy in patients with unresectable malignant pleural mesothelioma (MPM), regardless of histology.With a minimum follow-up of three years (35.5 months):
- Among patients treated with Opdivo plus Yervoy, 23% were alive at three years, compared to 15% of patients treated with chemotherapy.
- Treatment with the dual immunotherapy combination continued to show a reduction in the risk of death (Hazard Ratio [HR] 0.73; 95% Confidence Interval [CI]: 0.61 to 0.87), and improvement in median overall survival (OS), the trial’s primary endpoint, vs. chemotherapy (18.1 months vs. 14.1 months, respectively).
The safety profile for Opdivo plus Yervoy remained consistent with previously reported data in first-line MPM, with no new safety signals identified. These data will be presented on September 17, 2021 at 13:40 CEST/7:40 a.m. EDT (Abstract #LBA65) during the Proffered Paper session at the 2021 European Society for Medical Oncology (ESMO) Virtual Congress.“For patients with malignant pleural mesothelioma, the prognosis is generally poor, with a five-year survival rate of approximately 10%,” said Solange Peters, M.D., Ph.D., Medical Oncology Service, Chair, Thoracic Oncology, Lausanne University Hospital, Lausanne, Switzerland. “In this aggressive cancer that historically has had limited treatment options, we’ve now not only seen the potential for patients to live longer with nivolumab plus ipilimumab, but that this benefit is sustained at three years compared to treatment with chemotherapy. These results give us further proof of the durability of the outcomes achieved with this combination.”At three years of follow-up and approximately one year of being off treatment per protocol, more patients who responded to Opdivo plus Yervoy remained in response than those treated with chemotherapy, and duration of response (DOR) was longer with the dual immunotherapy combination, regardless of histology:
- 28% of patients who responded to Opdivo plus Yervoy remained in response at three years compared to no patients (0%) in the chemotherapy arm.
- Those treated with the dual immunotherapy combinationhad a median DOR of 11.6 months, compared to 6.7 months with chemotherapy.
- Objective response rate (ORR) in patients treated with the combination was comparable to chemotherapy (39.6% vs. 44.0%, respectively).
“The results from the CheckMate -743 trial have changed the way physicians treat malignant pleural mesothelioma, a disease that had no new systemic treatment options for nearly 15 years before the approval of Opdivo plus Yervoy,” said Abderrahim Oukessou, M.D., vice president, thoracic cancers development lead, Bristol Myers Squibb. “We continue to see more evidence for the sustained survival benefits of dual immunotherapy across several tumors, including durable overall survival at four years in non-small cell lung cancer. Now, the combination has demonstrated long-term improvements in overall survival in mesothelioma, another thoracic cancer, extending the lives of patients with a devastating disease.”Opdivo plus Yervoy has received approval for use in first-line unresectable MPM from 14 health authorities around the world, including in the U.S., European Union, Japan and China. Additional regulatory applications are under review globally.Opdivo plus Yervoy-based combinations have shown significant improvements in OS in six Phase 3 clinical trials in five tumors to date: malignant pleural mesothelioma, non-small cell lung cancer, metastatic melanoma, advanced renal cell carcinoma and esophageal squamous cell carcinoma.
About CheckMate -743CheckMate -743 is an open-label, multi-center, randomized Phase 3 trial evaluating Opdivo plus Yervoy compared to chemotherapy (pemetrexed and cisplatin or carboplatin) in patients with previously untreated unresectable malignant pleural mesothelioma (n=605). In the trial, 303 patients were randomized to receive Opdivo at 3 mg/kg every two weeks and Yervoy at 1 mg/kg every six weeks; 302 patients were randomized to receive cisplatin 75 mg/m2 or carboplatin AUC 5 plus pemetrexed 500 mg/m2 in 21-day cycles for six cycles. Treatment in both arms continued until disease progression or unacceptable toxicity or, in the Opdivo plus Yervoy arm, up to 24 months. The primary endpoint of the trial was overall survival (OS) in all randomized patients. Additional efficacy outcome measures included progression-free survival (PFS), objective response rate (ORR) and duration of response (DOR), as assessed by blinded independent central review (BICR) utilizing modified RECIST criteria. Exploratory endpoints included safety, pharmacokinetics, immunogenicity and patient reported outcomes.
About OpdivoOpdivo is a programmed death-1 (PD-1) immune checkpoint inhibitor that is designed to uniquely harness the body’s own immune system to help restore anti-tumor immune response. By harnessing the body’s own immune system to fight cancer, Opdivo has become an important treatment option across multiple cancers.Opdivo’s leading global development program is based on Bristol Myers Squibb’s scientific expertise in the field of Immuno-Oncology, and includes a broad range of clinical trials across all phases, including Phase 3, in a variety of tumor types. To date, the Opdivo clinical development program has treated more than 35,000 patients. The Opdivo trials have contributed to gaining a deeper understanding of the potential role of biomarkers in patient care, particularly regarding how patients may benefit from Opdivo across the continuum of PD-L1 expression.In July 2014, Opdivo was the first PD-1 immune checkpoint inhibitor to receive regulatory approval anywhere in the world. Opdivo is currently approved in more than 65 countries, including the United States, the European Union, Japan and China. In October 2015, the Company’s Opdivo and Yervoy combination regimen was the first Immuno-Oncology to receive regulatory approval for the treatment of metastatic melanoma and is currently approved in more than 50 countries, including the United States and the European Union.
About YervoyYervoy is a recombinant, human monoclonal antibody that binds to the cytotoxic T-lymphocyte-associated antigen-4 (CTLA-4). CTLA-4 is a negative regulator of T-cell activity. Yervoy binds to CTLA-4 and blocks the interaction of CTLA-4 with its ligands, CD80/CD86. Blockade of CTLA-4 has been shown to augment T-cell activation and proliferation, including the activation and proliferation of tumor infiltrating T-effector cells. Inhibition of CTLA-4 signaling can also reduce T-regulatory cell function, which may contribute to a general increase in T-cell responsiveness, including the anti-tumor immune response. On March 25, 2011, the U.S. Food and Drug Administration (FDA) approved Yervoy 3 mg/kg monotherapy for patients with unresectable or metastatic melanoma. Yervoy is approved for unresectable or metastatic melanoma in more than 50 countries. There is a broad, ongoing development program in place for Yervoy spanning multiple tumor types.
INDICATIONSOPDIVO® (nivolumab) is indicated for the treatment of patients with unresectable or metastatic melanoma.OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with unresectable or metastatic melanoma.OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express PD-L1 (≥1%) as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations.OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab) and 2 cycles of platinum-doublet chemotherapy, is indicated for the first-line treatment of adult patients with metastatic or recurrent non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.OPDIVO® (nivolumab) is indicated for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving OPDIVO.OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with unresectable malignant pleural mesothelioma (MPM).OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of patients with intermediate or poor risk advanced renal cell carcinoma (RCC).OPDIVO® (nivolumab), in combination with cabozantinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).OPDIVO® (nivolumab) is indicated for the treatment of patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy.OPDIVO® (nivolumab) is indicated for the treatment of adult patients with classical Hodgkin lymphoma (cHL) that has relapsed or progressed after autologous hematopoietic stem cell transplantation (HSCT) and brentuximab vedotin or after 3 or more lines of systemic therapy that includes autologous HSCT. This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.OPDIVO® (nivolumab) is indicated for the treatment of patients with recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN) with disease progression on or after platinum-based therapy.OPDIVO® (nivolumab) is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.OPDIVO® (nivolumab), as a single agent, is indicated for the adjuvant treatment of patients with urothelial carcinoma (UC) who are at high risk of recurrence after undergoing radical resection of UC.OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of adult and pediatric (12 years and older) patients with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.OPDIVO® (nivolumab) is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection.OPDIVO® (nivolumab) is indicated for the treatment of patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) after prior fluoropyrimidine- and platinum-based chemotherapy.OPDIVO® (nivolumab) is indicated for the adjuvant treatment of completely resected esophageal or gastroesophageal junction cancer with residual pathologic disease in patients who have received neoadjuvant chemoradiotherapy (CRT).OPDIVO® (nivolumab), in combination with fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the treatment of patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma.
Please see US Full Prescribing Information for OPDIVO and YERVOY.
For more information about Bristol Myers Squibb, visit us at BMS.com
Opdivo/Yervoy combo shows long-term survival benefit as first-line mesothelioma treatment
Sep. 13, 2021 11:56 AM ET Bristol-Myers Squibb Company (BMY) By: Jonathan M Block, SA News Editor4 Comments
- A combination of Bristol-Myers Squibb's (BMY +0.1%) Opdivo (nivolumab) and Yervoy (ipilimumab) demonstrated a superior survival benefit over at least three years compared to chemotherapy as a first-line therapy in unresectable malignant pleural mesothelioma.
- https://seekingalpha.com/news/3739158-opdivoyervoy-combo-shows-long-term-survival-benefit-as-first-line-mesothelioma-treatment
- https://www.opdivo.com/about-opdivo/how-the-combination-works-combinationtherapy
- https://www.bms.com/
- https://seekingalpha.com/symbol/BMY
Zabdeno® (Ad26.ZEBOV) and Mvabea® (MVA-BN-Filo)
Johnson & Johnson Ebola Vaccine Regimen Demonstrated Robust and Durable Immune Response in Adults and Children in Data Published in The Lancet Infectious Diseases
Sep 13, 2021United States
Data show the vaccine regimen induced neutralizing antibody responses in nearly all participating adults and children 21 days after the second dose
Adults receiving booster shots two years after initial vaccination regimen showed strong immune responses
The data support the potential prophylactic use of the Johnson & Johnson Ebola vaccine regimen to protect adults and children
NEW BRUNSWICK, N.J., September 13, 2021 – Data from two papers published in The Lancet Infectious Diseases demonstrated that the Johnson & Johnson (the Company) Ebola vaccine regimen, Zabdeno® (Ad26.ZEBOV) and Mvabea® (MVA-BN-Filo), generated robust humoral (antibody) immune responses in adults and children (ages 1-17) with the immune responses persisting in adults for at least two years. The data also showed that booster vaccination with Ad26.ZEBOV, administered to adults two years after the initial vaccination, induced a strong anamnestic (immune) response within seven days. These findings support the potential prophylactic use of the vaccine regimen, which was developed by the Janssen Pharmaceutical Companies of Johnson & Johnson (Janssen) in collaboration with Bavarian Nordic A/S, and was granted Marketing Authorisation by the European Commission in July 2020 and Prequalification from the World Health Organization (WHO) in April 2021.
The data is from the Phase 3 EBOVAC-Salone clinical study and showed that the vaccine regimen was well-tolerated and induced antibody responses to the Zaire ebolavirus species 21 days after the second dose in 98 percent of all participants. There were no safety signals of concern.
“These peer-reviewed data support the prophylactic use of the Johnson & Johnson Ebola vaccine regimen to protect people at risk of Ebola, which is essential to our vision of preventing Ebola outbreaks before they can begin,” said Paul Stoffels, M.D., Vice Chairman of the Executive Committee and Chief Scientific Officer of Johnson & Johnson. “Recent and ongoing outbreaks in Africa underscore that the threat of Ebola is not going away, which is why we collaborated to develop a vaccine regimen capable of inducing long-term immunity against Ebola and are now working to ensure that it is accessible to people in need.”
The EBOVAC-Salone study was conducted in Sierra Leone and is the first to assess the safety and tolerability of the Johnson & Johnson Ebola vaccine regimen in adults in a region affected by the 2014-2016 West African Ebola outbreak, which was the worst on record. It is also the first study evaluating the Johnson & Johnson Ebola vaccine regimen in a randomized, double-blind, controlled trial in a pediatric population.
Phase 3 Study Design (NCT02509494)
This Phase 3 study was designed to gather information on the safety and immunogenicity of the two-dose, heterologous (containing different vaccine components administered at different timepoints) Johnson & Johnson Ebola vaccine regimen. In this regimen, Ad26.ZEBOV was administered intramuscularly as the first dose vaccination followed 56 days later by MVA-BN-Filo as the second dose vaccination.
The study was divided into two stages. In stage one, 43 adults aged 18 years or older were vaccinated to gain information about the safety and immunogenicity of the two-dose vaccine regimen. In stage two, 400 adults and 576 children or adolescents (including 192 in each of the three age cohorts of 1-3, 4-11 and 12-17 years of age) were vaccinated. Consenting adults participating in stage one of the study were administered a booster dose of A26.ZEBOV two years after the first dose.
The study was conducted at three clinics in Kambia District, Sierra Leone. Long term follow-up of the study participants is underway.
Johnson & Johnson’s Ebola vaccine regimen originates from a collaborative research program with the NIH and received direct funding and preclinical services from the National Institute of Allergy and Infectious Diseases, part of NIH, under Contract Number HHSN272200800056C. Further funding for the Ebola vaccine regimen has been provided in part with federal funds from the Office of the Assistant Secretary for Preparedness and Response, BARDA under Contract Numbers HHSO100201700013C and HHSO100201500008C.
Learn more at www.JNJ.com/Ebola.
Learn more at www.jnj.com.
Learn more at www.jnj.com.
Learn more at www.janssen.com.
Johnson & Johnson Ebola vaccine yields strong immune response, peer reviewed data show
Sep. 14, 2021 6:39 AM ET Johnson & Johnson (JNJ)
By: Dulan Lokuwithana, SA News Editor2 Comments
- The Ebola vaccine, developed by Johnson & Johnson (NYSE:JNJ) in partnership with Bavarian Nordic A/S, generated a strong immune reaction in adults and children and the response last at least two years, according to two papers published in the medical journal The Lancet Infectious Diseases.
- In Phase 3 EBOVAC-Salone trial, antibody responses to the Zaire ebolavirus species were seen 21 days after the second dose in 98% of the participants who received the Zabdeno (Ad26.ZEBOV) and Mvabea (MVA-BN-Filo) vaccine regimen, J&J (JNJ) said.
- https://seekingalpha.com/news/3739398-johnson-johnson-ebola-vaccine-yields-strong-immune-response-peer-reviewed-data-show
- https://seekingalpha.com/symbol/JNJ
- https://www.jnj.com/
- https://www.janssen.com/
- https://www.jnj.com/ebola
Zanidatamab
Zymeworks Announces Abstract For Zanidatamab In First-Line HER2-Expressing Gastroesophageal Cancers (GEA) At The European Society For Medical Oncology (ESMO) Annual Congress
09/12/2021 at 6:49 PM EDTPDF Version
- Abstract data demonstrate encouraging antitumor activity in first-line GEA
- Full ESMO presentation available September 16 at 8:30 am CEST, 2:30 am ET
- Conference call and webcast with principal investigator, Dr. Geoffrey Ku, on September 16 at 7:30 am ET
VANCOUVER, British Columbia--(BUSINESS WIRE)--Sep. 12, 2021-- Zymeworks Inc. (NYSE: ZYME), a clinical-stage biopharmaceutical company developing multifunctional biotherapeutics, today announced the publication of an abstract highlighting new clinical data for zanidatamab, a HER2-targeted bispecific antibody, in first-line HER2-expressing GEA. An updated and expanded data set will be presented at the ESMO Annual Congress taking place virtually on September 16-21, 2021.
Abstract highlights from March 18, 2021 data cut:
- Thirty patients had been treated with zanidatamab in combination with standard of care chemotherapy (either mFOLFOX6, CAPOX, or FP), and 14 patients remained on treatment.
- The confirmed objective response rate was 68.2% and the disease control rate was 90.9% in 22 HER2-positive response-evaluable patients.
- Treatment related adverse events were generally consistent with previous reports of zanidatamab and/or the chemotherapy regimens, with the majority reported as Grade 1 or 2 in severity.
“The initial data from the abstract highlight an encouraging objective response rate for zanidatamab combined with standard of care chemotherapy in patients with metastatic HER2-positive GEA,” said Neil Josephson, M.D., Zymeworks’ Interim Chief Medical Officer. “We’re looking forward to presenting at the Congress the full updated data, which further support zanidatamab’s potential as the new foundational HER2-targeted therapy.”
ESMO Presentation
The abstract is available on the ESMO conference website. The presentation will be available on Thursday, September 16 at 8:30 am CEST, 2:30 am ET, to conference registrants on the ESMO conference website as well as to the general public on the Zymeworks website at https://ir.zymeworks.com/events-and-presentations.
Title: Phase (Ph) 2 Study of Zanidatamab + Chemotherapy (chemo) in First Line (1L) HER2-expressing Gastroesophageal Adenocarcinoma (GEA)
Lead Author: Geoffrey Ku, M.D., Memorial Sloan Kettering Cancer Center, New York, NY, US
Abstract: 3678
E-poster: 1380P
About Zanidatamab
Zanidatamab is a bispecific antibody, based on Zymeworks’ Azymetric™ platform, that can simultaneously bind two non-overlapping epitopes of HER2, known as biparatopic binding. This unique design results in multiple mechanisms of action including dual HER2 signal blockade, increased binding and removal of HER2 protein from the cell surface, and potent effector function leading to encouraging antitumor activity in patients. Zymeworks is developing zanidatamab in multiple Phase 1, Phase 2, and pivotal clinical trials globally as a targeted treatment option for patients with solid tumors that express HER2. The FDA has granted Breakthrough Therapy designation for zanidatamab in patients with previously treated HER2 gene-amplified biliary tract cancer (BTC), and two Fast Track designations to zanidatamab, one as a single agent for refractory BTC and one in combination with standard of care chemotherapy for first-line gastroesophageal adenocarcinoma (GEA). These designations mean zanidatamab is eligible for Accelerated Approval, Priority Review and Rolling Review, as well as intensive FDA guidance on an efficient drug development program. Zanidatamab has also received Orphan Drug designations for the treatment of biliary tract, gastric and ovarian cancers from the FDA, as well as Orphan Drug designation for the treatment of biliary tract and gastric cancer from the European Medicines Agency.
For additional information about Zymeworks, visit www.zymeworks.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20210912005060/en/
Source: Zymeworks Inc.
Zymeworks' zanidatamab shows encouraging antitumor activity in esophageal cancer
Sep. 12, 2021 11:52 PM ET Zymeworks Inc. (ZYME) By: Mamta Mayani, SA News Editor1 Comment
- Zymeworks (NYSE:ZYME) announces the publication of an abstract highlighting new clinical data for zanidatamab, a HER2-targeted bispecific antibody, in first-line HER2-expressing Gastroesophageal Adenocarcinoma (GEA).
- https://seekingalpha.com/news/3738906-zymeworks-zanidatamab-shows-encouraging-antitumor-activity-in-esophageal-cancer
- https://seekingalpha.com/symbol/ZYME
- https://www.zymeworks.com/pipeline
Jardiance® (empagliflozin)
FDA grants Jardiance® Breakthrough Therapy designation for heart failure with preserved ejection fraction
September 9, 2021Download PDF- Designation is based on results from the EMPEROR-Preserved phase III trial, which established Jardiance as the first therapy to show statistically significant improvement in heart failure outcomes in adults with heart failure with preserved ejection fraction
RIDGEFIELD, Conn. and INDIANAPOLIS, Sept. 9, 2021 /PRNewswire/ -- The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation for Jardiance® (empagliflozin) as an investigational treatment for adults with heart failure with preserved ejection fraction (HFpEF), Boehringer Ingelheim and Eli Lilly and Company (NYSE: LLY) announced today.The decision is based on results from the landmark EMPEROR-Preserved phase III trial, in which Jardiance demonstrated a 21% relative risk reduction for the composite primary endpoint of cardiovascular death or hospitalization for heart failure in adults with heart failure with preserved ejection fraction compared with placebo. The benefit was independent of ejection fraction or diabetes status. Results from EMPEROR-Preserved were presented at the European Society of Cardiology Congress 2021 on August 27 and published in The New England Journal of Medicine.HFpEF accounts for approximately half of the more than 6 million heart failure cases in the U.S. No currently approved treatments have been clinically proven to significantly improve outcomes specifically for people with HFpEF."This Breakthrough Therapy designation underscores the potential of Jardiance to help fill a critical need for a clinically proven treatment for people with this highly prevalent, difficult-to-treat condition," said Mohamed Eid, M.D., M.P.H., M.H.A., vice president, Clinical Development & Medical Affairs, Cardio-Metabolism & Respiratory Medicine, Boehringer Ingelheim Pharmaceuticals, Inc. "Following the recent FDA approval of Jardiance for heart failure with reduced ejection fraction, this is another important milestone supporting the potential of Jardiance as the first therapy clinically proven to improve outcomes across the full heart failure spectrum."According to the FDA, Breakthrough Therapy designation is designed to expedite the development and review of therapies that are intended to treat a serious condition where preliminary clinical evidence indicates potentially substantial improvement over available therapies on a clinically significant endpoint. "Given the scarcity of treatment options for this debilitating condition, the benefits demonstrated in the EMPEROR-Preserved trial constituted a major clinical breakthrough," said Jeff Emmick, M.D., Ph.D., vice president, Product Development, Lilly. "Together with our Boehringer Ingelheim partners, we look forward to working closely with the FDA through this accelerated process to potentially bring Jardiance to adults with heart failure with preserved ejection fraction as soon as possible."The FDA previously granted Fast Track designation for the development of Jardiance to reduce the risk of cardiovascular death and hospitalization for heart failure. This designation is for the EMPEROR program, which consists of the EMPEROR-Reduced and EMPEROR-Preserved trials. The EMPEROR-Reduced results formed the basis of the recent FDA approval for heart failure with reduced ejection fraction. Jardiance is not indicated for the treatment of HFpEF.
About EMPEROR-PreservedEMPEROR-Preserved (NCT03057951) was a phase III international, randomized, double–blind trial that enrolled 5,988 adults with and without type 2 diabetes. All participants had heart failure (New York Heart Association [NYHA] functional class II, III or IV) and preserved ejection fraction (left ventricular ejection fraction > 40%).Participants were randomized to once-daily Jardiance 10 mg (n=2997) or placebo (n=2991), on top of treatment with guideline-directed heart failure therapy. Median follow-up time was 26.2 months. The composite primary endpoint was defined as time to first event of cardiovascular death or hospitalization for heart failure.
What is JARDIANCE? (www.jardiance.com)JARDIANCE is a prescription medicine used to:
- lower blood sugar along with diet and exercise in adults with type 2 diabetes
- reduce the risk of cardiovascular death in adults with type 2 diabetes who also have known cardiovascular disease
- reduce the risk of cardiovascular death and hospitalization for heart failure (when the heart is weak and cannot pump enough blood to the rest of your body) in adults with heart failure
JARDIANCE is not for people with type 1 diabetes. It may increase their risk of diabetic ketoacidosis (increased ketones in the blood or urine).JARDIANCE is not for use to lower blood sugar in adults with type 2 diabetes who have severe kidney problems, because it may not work.
Boehringer Ingelheim and Eli Lilly and CompanyIn January 2011, Boehringer Ingelheim and Eli Lilly and Company announced an alliance that centers on compounds representing several of the largest diabetes treatment classes. Depending on geographies, the companies either co-promote or separately promote the respective molecules each contributing to the alliance. The alliance leverages the strengths of two of the world's leading pharmaceutical companies to focus on patient needs. By joining forces, the companies demonstrate their commitment, not only to the care of people with diabetes, but also to investigating the potential to address areas of unmet medical need. Clinical trials have been initiated to evaluate the impact of Jardiance on people living with heart failure or chronic kidney disease.
For more information, please see Prescribing Information and Medication Guide.
For more information, please visit www.boehringer-ingelheim.us,
To learn more about Lilly, please visit us at lilly.com
View original content to download multimedia:https://www.prnewswire.com/news-releases/fda-grants-jardiance-breakthrough-therapy-designation-for-heart-failure-with-preserved-ejection-fraction-301372022.htmlSOURCE Eli Lilly and Company
Eli Lilly's Jardiance wins FDA's Breakthrough Therapy status for heart failure indication
Sep. 09, 2021 8:35 AM ETEli Lilly and Company (LLY)By: Aakash Babu, SA News Editor
- The U.S. FDA has granted Eli Lilly (NYSE:LLY) and Boehringer Ingelheim's Jardiance Breakthrough Therapy designation as an investigational treatment for adults with heart failure with preserved ejection fraction ((HFpEF)).
- https://seekingalpha.com/news/3738291-eli-lillys-jardiance-wins-fdas-breakthrough-therapy-status-for-heart-failure-indication
- https://seekingalpha.com/symbol/LLY
- https://www.lilly.com/
Crysvita™ (Burosumab Injection)
Ultragenyx Canada Announces Health Canada Approval of a Second Indication for Crysvita™ (Burosumab Injection) for the Treatment of Tumour Induced Osteomalacia in Adults
Sep. 07, 2021 8:00 AM ETUltragenyx Pharmaceutical Inc. (RARE)
OTTAWA, Sept. 07, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, today announced that Crysvita™ (Burosumab Injection) has been approved by Health Canada for the treatment of Tumour Induced Osteomalacia (TIO) in adults. Crysvita is also approved for the treatment of X-Linked Hypophosphatemia.
TIO is a rare disease that is characterized by the development of tumours that cause weakened and softened bones. The tumours associated with TIO release a protein known as fibroblast growth factor 23 (FGF23) that lowers phosphate levels.
INDICATION FOR TUMOUR INDUCED OSTEOMALACIA (TIO)
CRYSVITA (Burosumab Injection) is indicated for the treatment of fibroblast growth factor 23 (FGF23)–related hypophosphatemia in tumour-induced osteomalacia (TIO) associated with tumours that cannot be curatively resected or localized in adult patients.
For more information on Ultragenyx, please visit the company's website at: www.ultragenyx.com.
https://seekingalpha.com/symbol/RARE
Tezepelumab
New Tezepelumab Data Show 86% Reduction In Exacerbations In Patients With Severe Asthma And Comorbid Nasal Polyps
Improvements Also Demonstrated in Lung Function and Nasal Polyp Symptoms
Exploratory Analysis From NAVIGATOR Phase 3 Trial Presented at European Respiratory Society (ERS) International Congress 2021
NEWS PROVIDED BY
Sep 05, 2021, 07:15 ET
SHARE THIS ARTICLE
THOUSAND OAKS, Calif., Sept. 5, 2021 /PRNewswire/ -- Amgen (NASDAQ:AMGN) today announced new data from the pivotal NAVIGATOR Phase 3 trial demonstrating that tezepelumab reduced exacerbations and improved lung function and nasal symptoms in patients with severe, uncontrolled asthma and comorbid nasal polyps.1 Tezepelumab is a potential first-in-class treatment that acts at the top of the inflammatory cascade by targeting thymic stromal lymphopoietin (TSLP), an epithelial cytokine, and has the potential to treat a broad population of patients with severe asthma.2,3 Tezepelumab is being developed by Amgen in collaboration with AstraZeneca.
The pre-specified exploratory analysis evaluated the effect of tezepelumab in NAVIGATOR patients with or without reported nasal polyps (NP+ or NP−) in the past two years. The analysis showed tezepelumab achieved an 86% reduction in the annualized asthma exacerbation rate (AAER) in NP+ patients (95% CI: 70, 93) and 52% (95% CI: 42, 61) in NP− patients over 52 weeks, compared to placebo when added to standard of care (SoC).1
Tezepelumab improved lung function at week 52 in both groups of patients with an increase in pre-bronchodilator forced expiratory volume in one second (FEV1) of 0.20 L (95% CI: 0.02, 0.37) and 0.13 L (95% CI: 0.08, 0.18) versus placebo in NP+ and NP− patients, respectively.1 Tezepelumab also achieved a clinically relevant improvement in nasal polyp symptoms at week 52, as measured by the Sinonasal Outcome Test (SNOT-22), reducing the SNOT-22 score in NP+ patients by 9.6 points (95% CI: 0.9, 18.2) versus placebo.1,4 The adjusted mean score reductions from baseline for tezepelumab and placebo were 20.10 points (SE: 3.07) and 10.55 points (SE: 2.94). Baseline mean (sd) SNOT-22 score was 49.4 (21.5) and 47.8 (19.0) for tezepelumab and placebo, respectively.1
"This new analysis from NAVIGATOR is exciting for the up to one in five severe asthma patients who have comorbid nasal polyps," said Professor Andrew Menzies-Gow, director of the Lung Division, Royal Brompton Hospital, London, UK, the principal investigator of the NAVIGATOR trial. "The analysis shows tezepelumab's ability to reduce exacerbations, improve lung function and reduce the symptoms of nasal polyps in this comorbid population who are typically more prone to asthma attacks, have an increased likelihood of airway obstruction, and may have a worse quality of life."
Results from the NAVIGATOR Phase 3 trial were published in the New England Journal of Medicine in May 2021.5 A Phase 3 clinical trial, WAYPOINT, has been initiated to explore the efficacy and safety of tezepelumab in adults with severe, chronic rhinosinusitis with nasal polyps.6
About the NAVIGATOR and the PATHFINDER Clinical Trial Program
Building on the Phase 2b PATHWAY trial, the Phase 3 PATHFINDER program included two trials, NAVIGATOR5,23 and SOURCE.24,25 The program includes additional planned mechanistic and long-term safety trials.26 In addition, a Phase 3 clinical trial, WAYPOINT, has been initiated to explore the efficacy and safety of tezepelumab in adults with severe, chronic rhinosinusitis with nasal polyps.6
NAVIGATOR is a Phase 3, randomized, double-blinded, placebo-controlled trial in 1,061 adults (18–80 years old) and adolescents (12–17 years old) with severe, uncontrolled asthma, who were receiving treatment with medium- or high-dose inhaled corticosteroids (ICS) plus at least one additional controller medication with or without OCS. Of the 1,061 randomized patients, 1,059 received either tezepelumab 210 mg (n=528) or placebo (n=531). In total for this pre-specified exploratory analysis, 83 patients had NP in the past 2 years (tezepelumab 210 mg, n=42; placebo, n=41) and 976 did not (tezepelumab 210 mg, n=486; placebo, n=490). NAVIGATOR met the primary endpoint with tezepelumab added to SoC demonstrating a statistically significant and clinically meaningful reduction in the AAER over 52 weeks in the overall patient population, compared to placebo added to SoC. The trial also met the primary endpoint in the subgroup of patients with baseline eosinophil counts less than 300 cells per microliter, with tezepelumab demonstrating a statistically significant and clinically meaningful reduction in AAER in that patient population. Similar reductions in AAER were observed in the subgroup of patients with baseline eosinophil counts less than 150 cells per microliter.26,27
About Tezepelumab
Tezepelumab is being developed by AstraZeneca in collaboration with Amgen (see AstraZeneca and Amgen collaboration below) as an investigational, potential first-in-class human monoclonal antibody that works on the primary source of inflammation: the airway epithelium, which is the first point of contact for viruses, allergens, pollutants and other environmental insults. Specifically, tezepelumab targets and blocks thymic stromal lymphopoietin (TSLP), a key epithelial cytokine that sits at the top of multiple inflammatory cascades and initiates an overreactive immune response to allergic, eosinophilic and other types of airway inflammation associated with severe asthma.2,29
TSLP is released in response to multiple triggers associated with asthma exacerbations, including allergens, viruses and other airborne particles.2,29 Expression of TSLP is increased in the airways of patients with asthma and has been correlated with disease severity.2,3 Blocking TSLP may prevent the release of pro-inflammatory cytokines by immune cells, resulting in the prevention of asthma exacerbations and improved asthma control.2,3 By working at the top of the cascade, tezepelumab helps stop inflammation at the source and has the potential to treat a broad population of severe asthma patients.2,3
About the Amgen and AstraZeneca Collaboration
In 2020, Amgen and AstraZeneca updated the 2012 collaboration agreement for tezepelumab. Both companies will continue to share costs and profits equally after payment by AstraZeneca of a mid-single-digit royalty to Amgen. AstraZeneca continues to lead development and Amgen continues to lead manufacturing. All aspects of the collaboration are under the oversight of joint governing bodies. Under the amended agreement in North America, Amgen and AstraZeneca will jointly commercialize tezepelumab; Amgen will record sales in the U.S. and AstraZeneca will record sales in Canada. Outside the U.S., Amgen will record sales as collaboration revenue.
For more information, visit www.amgen.com
Amgen/AstraZeneca's tezepelumab reduces exacerbations in severe asthma patients
Sep. 07, 2021 5:10 AM ET Amgen Inc. (AMGN), AZN
By: Mamta Mayani, SA News Editor
- Amgen (NASDAQ:AMGN) announces new data from Phase 3 NAVIGATOR trial demonstrating that tezepelumab reduced exacerbations and improved lung function and nasal symptoms in patients with severe, uncontrolled asthma and comorbid nasal polyps. Tezepelumab is being developed in collaboration with AstraZeneca (NASDAQ:AZN).
- https://seekingalpha.com/news/3737395-amgenastrazenecas-tezepelumab-reduces-exacerbations-in-severe-asthma-patients
- https://seekingalpha.com/symbol/AMGN
- https://www.amgen.com/
- https://seekingalpha.com/symbol/AZN
- https://www.astrazeneca.com/
KEYTRUDA® (pembrolizumab)
Merck’s KEYTRUDA® (pembrolizumab) Approved in China in Combination With Chemotherapy for First-Line Treatment of Patients With Locally Advanced Unresectable or Metastatic Esophageal or Gastroesophageal Junction (GEJ) Carcinoma
September 7, 2021 6:45 am ET
First Anti-PD-1 Regimen Approved in China for First-Line Treatment of Advanced Esophageal or GEJ Cancer, Regardless of Histology or PD-L1 Expression
KEYTRUDA Is Now Approved for Eight Indications Across Five Different Types of Cancer in ChinaKENILWORTH, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced that KEYTRUDA, Merck’s anti-PD-1 therapy, has been approved by the National Medical Products Administration (NMPA) in China in combination with platinum- and fluoropyrimidine-based chemotherapy for the first-line treatment of patients with locally advanced unresectable or metastatic carcinoma of the esophagus or gastroesophageal junction (GEJ). This new indication was granted approval based on overall survival (OS) findings from the pivotal Phase 3 KEYNOTE-590 trial. KEYTRUDA is now approved for eight indications across five different types of cancer in China.“In China, esophageal and gastroesophageal junction cancers are leading causes of death, and there have been few treatment advances for patients over the past several decades,” said Dr. Scot Ebbinghaus, vice president, clinical research, Merck Research Laboratories. “With this approval of KEYTRUDA plus chemotherapy – the first for an anti-PD-1 regimen in the first-line setting – we can now provide patients with an immunotherapy treatment option earlier in the course of treatment that has been shown to significantly improve survival.”In the global KEYNOTE-590 study, KEYTRUDA in combination with chemotherapy (5-fluorouracil [5-FU] plus cisplatin) demonstrated statistically significant improvements in OS and progression-free survival (PFS) compared with 5-FU and cisplatin alone. KEYTRUDA plus 5-FU and cisplatin reduced the risk of death by 27% (HR=0.73 [95% CI, 0.62-0.86]; p<0.0001). The median OS was 12.4 months (95% CI, 10.5-14.0) for patients treated with KEYTRUDA plus 5-FU and cisplatin and 9.8 months (95% CI, 8.8-10.8) for patients treated with 5-FU and cisplatin alone. KEYTRUDA plus 5-FU and cisplatin reduced the risk of disease progression or death by 35% (HR=0.65 [95% CI, 0.55-0.76]; p<0.0001). The median PFS was 6.3 months (95% CI, 6.2-6.9) for patients treated with KEYTRUDA plus 5-FU and cisplatin and 5.8 months (95% CI, 5.0-6.0) for patients treated with 5-FU and cisplatin alone in all randomized patients. The Chinese package insert notes that the safety of KEYTRUDA plus chemotherapy was investigated in 51 Chinese patients with esophageal carcinoma in KEYNOTE-590, and the safety profile of the Chinese patients was generally consistent with the known safety profiles of KEYTRUDA monotherapy and chemotherapy.“Compared to the rest of the world, China has a disproportionate number of patients who are diagnosed with and who die from esophageal cancer, the fifth most commonly diagnosed cancer and the fourth leading cause of cancer-related death in the country,” said Prof. Shen Lin, vice president, Clinical Oncology, Beijing Cancer Hospital and Peking University, and deputy director, Beijing Institute for Cancer Research. “This approval for KEYTRUDA provides an important new option to patients with advanced esophageal carcinoma in China who currently have limited available treatment options in the first-line setting.”
About KEYNOTE-590KEYNOTE-590 (ClinicalTrials.gov, NCT03189719) is a multicenter, randomized, double-blind, placebo-controlled trial that enrolled 749 patients with locally advanced unresectable or metastatic esophageal or GEJ carcinoma (Siewert type I) who were not candidates for surgical resection or definitive chemoradiation. Patients were randomized (1:1) to receive either KEYTRUDA (200 mg on Day 1 every three weeks) or placebo (on Day 1 every three weeks) in combination with cisplatin (80 mg/m2 on Day 1 every three weeks for up to six cycles) plus 5-FU (800 mg/m2 per day on Days 1 to 5 every three weeks, or per local standard for 5-FU administration, for up to 24 months); all study medications were administered via intravenous infusion.The primary endpoints were OS and PFS, as assessed by the investigator according to RECIST v1.1, in squamous cell histology, PD-L1 expression (CPS ≥10) and in all patients. The secondary endpoints include objective response rate and duration of response, as assessed by the investigator according to RECIST v1.1.
About KEYTRUDA® (pembrolizumab) Injection, 100 mgKEYTRUDA 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 1,500 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.
Selected KEYTRUDA® (pembrolizumab) Indications in the U.S.MelanomaKEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.Non-Small Cell Lung CancerKEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) ≥1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is:
- stage III where patients are not candidates for surgical resection or definitive chemoradiation, or
- metastatic.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS ≥1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.Head and Neck Squamous Cell CancerKEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS ≥1)] as determined by an FDA-approved test.KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.Classical Hodgkin LymphomaKEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.Primary Mediastinal Large B-Cell LymphomaKEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.Urothelial CarcinomaKEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC):
- who are not eligible for any platinum-containing chemotherapy, or
- who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.Microsatellite Instability-High or Mismatch Repair Deficient CancerKEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options.This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.Microsatellite Instability-High or Mismatch Repair Deficient Colorectal CancerKEYTRUDA is indicated for the treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).Gastric CancerKEYTRUDA, in combination with trastuzumab, fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of patients with locally advanced unresectable or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.Esophageal CancerKEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic esophageal or GEJ (tumors with epicenter 1 to 5 centimeters above the GEJ) carcinoma that is not amenable to surgical resection or definitive chemoradiation either:
- in combination with platinum- and fluoropyrimidine-based chemotherapy, or
- as a single agent after one or more prior lines of systemic therapy for patients with tumors of squamous cell histology that express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
Cervical CancerKEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.Hepatocellular CarcinomaKEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.Merkel Cell CarcinomaKEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.Renal Cell CarcinomaKEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of adult patients with advanced renal cell carcinoma.Tumor Mutational Burden-High CancerKEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [≥10 mutations/megabase] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.Cutaneous Squamous Cell CarcinomaKEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) or locally advanced cSCC that is not curable by surgery or radiation.Triple-Negative Breast CancerKEYTRUDA is indicated for the treatment of patients with high-risk early-stage triple-negative breast cancer (TNBC) in combination with chemotherapy as neoadjuvant treatment, and then continued as a single agent as adjuvant treatment after surgery.KEYTRUDA, in combination with chemotherapy, is indicated for the treatment of patients with locally recurrent unresectable or metastatic TNBC whose tumors express PD-L1 (CPS ≥10) as determined by an FDA-approved test.https://www.keytruda.com/
For more information, visit www.merck.com
Please see Prescribing Information for KEYTRUDA (pembrolizumab) at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_pi.pdf and Medication Guide for KEYTRUDA at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_mg.pdf.View source version on businesswire.com: https://www.businesswire.com/news/home/20210907005361/en/ Source: Merck & Co., Inc.
Merck’s Keytruda combo OK'd in China for first-line treatment of esophageal cancer
Sep. 07, 2021 7:14 AM ETMerck & Co., Inc. (MRK)By: Mamta Mayani, SA News Editor
- Merck's (NYSE:MRK) Keytruda has been approved by the National Medical Products Administration in China in combination with chemotherapy for the first-line treatment of patients with locally advanced unresectable or metastatic carcinoma of the esophagus or gastroesophageal junction (GEJ).
- https://seekingalpha.com/news/3737429-mercks-keytruda-combo-okd-in-china-for-first-line-treatment-of-esophageal-cancer
- https://seekingalpha.com/symbol/MRK
- https://www.keytruda.com/
- https://www.merck.com/
ULTOMIRIS® (ravulizumab)
Ultomiris approved in the EU for children and adolescents with paroxysmal nocturnal haemoglobinuria
PUBLISHED3 September 2021
3 September 2021 07:00 BST
Approval based on interim results from Phase III trial demonstrating complete terminal complement inhibition with reduced dosing frequency compared to Soliris
Ultomiris (ravulizumab) has been approved in the European Union for expanded use to include children (with a body weight of 10 kg or above) and adolescents with paroxysmal nocturnal haemoglobinuria (PNH), an ultra-rare and severe blood disorder characterised by the destruction of red blood cells that can cause thrombosis (blood clots) and result in organ damage and potentially premature death.1-4
The approval by the European Commission was based on interim results from the Phase III clinical trial in children and adolescents with PNH that demonstrated the safety and efficacy of Ultomiris in these patients.
Ultomiris has an established safety and efficacy profile, offering reduced dosing frequency compared to Soliris (eculizumab), which is the current standard of care in the EU for the treatment of children and adolescents with PNH. Ultomiris is administered every four or eight weeks (depending on body weight), following a loading dose. The approval follows the recommendation of the Committee for Medicinal Products for Human Use of the European Medicines Agency in July 2021.
Austin Kulasekararaj, MD, King’s College Hospital, London, UK, said: “PNH is a devastating disease, and Ultomiris provides an advancement for paediatric patients in the EU with an established safety and efficacy profile. By requiring fewer infusions each year than Soliris, Ultomiris may reduce the need for these young patients to miss school to receive treatment.”
Marc Dunoyer, Chief Executive Officer, Alexion, said: “This approval of Ultomiris reflects our ongoing commitment to delivering new treatments that can make a meaningful difference in patients’ lives. Ultomiris has become the standard of care for the treatment of adults with PNH and we will make it available to this younger patient population as soon as possible.”
Interim results from the Phase III trial demonstrated Ultomiris was effective in achieving complete C5 complement inhibition through 26 weeks for the treatment of patients up to 18 years of age with PNH. Additionally, Ultomiris had no reported treatment-related severe adverse events, and no patients discontinued treatment during the primary evaluation period or experienced breakthrough haemolysis, which can lead to disabling or potentially fatal blood clots.1 The efficacy and safety profile of Ultomiris in children and adolescents is consistent with the profile of Ultomiris in clinical trials involving adults with PNH and is representative of the broad PNH patient population seen in the real-world clinical setting.5,6
Ultomiris was first approved in the EU in 2019 for the treatment of adults with PNH and is also approved in the EU for the treatment of adults and children with atypical haemolytic uraemic syndrome (aHUS). In June 2021, the US Food and Drug Administration approved the expanded use of Ultomiris to include children (one month of age and older) and adolescents with PNH, the first and only treatment for this age group in the US.
Ultomiris
Ultomiris (ravulizumab), the first and only long-acting C5 complement inhibitor, offers immediate, complete, and sustained complement inhibition. The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. When activated in an uncontrolled manner, the complement cascade over-responds, leading the body to attack its own healthy cells. Ultomiris is administered intravenously every eight weeks or, for paediatric patients less than 20 kg, every four weeks, following a loading dose. Ultomiris is approved in the US for the treatment of adults and children (one month of age and older) with PNH; in the EU for adults, as well as for children (with a body weight of 10 kg or above) and adolescents with PNH who experience haemolysis with clinical symptom(s) indicative of high disease activity, as well as for individuals who are clinically stable after having been treated with Soliris for at least the past six months; and in Japan as a treatment for adults with PNH. It is also approved in the US for aHUS to inhibit complement-mediated thrombotic microangiopathy in adult and paediatric (one month of age and older) patients, in the EU for the treatment of adults and children with a body weight of at least 10 kg with aHUS, as well as in Japan for adults and children with aHUS.
Please visit astrazeneca.com
AstraZeneca's Ultomiris OK'd in EU for children with rare blood disorder
Sep. 03, 2021 6:07 AM ET AstraZeneca PLC (AZN) By: Mamta Mayani, SA News Editor
- AstraZeneca (NASDAQ:AZN) announces that Ultomiris (ravulizumab) has been approved in the European Union (EU) for expanded use to include children and adolescents with paroxysmal nocturnal haemoglobinuria (PNH).
- https://seekingalpha.com/news/3737086-astrazenecas-ultomiris-okd-in-eu-for-children-with-rare-blood-disorder
- https://seekingalpha.com/symbol/AZN
- https://www.ultomiris.com/
- https://www.astrazeneca.com/
BRUKINSA® (Zanubrutinib)
U.S. FDA Grants BRUKINSA® (Zanubrutinib) Approval in Waldenström’s Macroglobulinemia
Sep 01, 2021 5:33 PM
This marks the second FDA approval for BRUKINSA and its third approval in Waldenström’s macroglobulinemia globally
The approval is based on Phase 3 ASPEN trial comparing BRUKINSA against ibrutinib
CAMBRIDGE, Mass. & BEIJING--(BUSINESS WIRE)-- BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a global biotechnology company focused on developing and commercializing innovative medicines worldwide, today announced that BRUKINSA® (zanubrutinib) has received approval from the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with Waldenström’s macroglobulinemia (WM).
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210901006049/en/
“We are delighted by today’s FDA approval for BRUKINSA in its second indication, offering a new treatment option with demonstrated efficacy and safety benefits for patients with Waldenström’s macroglobulinemia. As shown in the ASPEN trial, BRUKINSA can improve treatment outcomes for these patients and potentially make a positive impact on their lives,” commented Jane Huang, M.D., Chief Medical Officer, Hematology at BeiGene.
The FDA’s approval of BRUKINSA in WM is primarily based on efficacy results from the multicenter, open-label Phase 3 ASPEN trial (NCT03053440) comparing BRUKINSA to ibrutinib in patients with WM. A total of 201 patients with a MYD88 mutation (MYD88MUT) were enrolled in the randomized Cohort 1.
The primary efficacy endpoint of the ASPEN trial was very good partial response (VGPR) rate in the overall intention-to-treat (ITT) population as assessed by independent review committee (IRC). Based on the modified Sixth International Workshop on Waldenström’s Macroglobulinemia (IWWM-6) response criteria (Treon 2015), the VGPR rate was 28% with BRUKINSA, compared to 19% with ibrutinib; based on the IWWM-6 response criteria (Owen et al 2013), the VGPR rate was 16% with BRUKINSA, compared to 7% with ibrutinib.
In the FDA-approved label, the major efficacy outcome is defined as response rate of partial response (PR) or better as assessed by IRC. Based on either IWWM-6 response criteria, the response rate was 78% with BRUKINSA (95% CI: 68, 85), compared to 78% with ibrutinib (95% CI: 68, 86), and the event-free duration of response (DoR) at 12 months was 94% with BRUKINSA (95% CI: 86, 98), compared to 88% with ibrutinib (95% CI: 77, 94).
The most common (≥20%) adverse reactions based on the pooled safety population of 779 patients were decreased neutrophil count, upper respiratory tract infection, decreased platelet count, rash, hemorrhage, musculoskeletal pain, decreased hemoglobin, bruising, diarrhea, pneumonia, and cough.
The recommended dose of BRUKINSA is either 160 mg twice daily or 320 mg once daily, taken orally with or without food. The dose may be adjusted for adverse reactions and reduced for patients with severe hepatic impairment and certain drug interactions.
About BRUKINSA
BRUKINSA is a small molecule inhibitor of Bruton’s tyrosine kinase (BTK) discovered by BeiGene scientists that is currently being evaluated globally in a broad clinical program as a monotherapy and in combination with other therapies to treat various B-cell malignancies. Because new BTK is continuously synthesized, BRUKINSA was specifically designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity. With differentiated pharmacokinetics compared to other approved BTK inhibitors, BRUKINSA has been demonstrated to inhibit the proliferation of malignant B cells within a number of disease relevant tissues.
BRUKINSA is approved in the following indications and regions:
- For the treatment of mantle cell lymphoma (MCL) in adult patients who have received at least one prior therapy (United States, November 2019)*;
- For the treatment of MCL in adult patients who have received at least one prior therapy (China, June 2020)**;
- For the treatment of chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) in adult patients who have received at least one prior therapy (China, June 2020)**;
- For the treatment of relapsed or refractory MCL (United Arab Emirates, February 2021);
- For the treatment of Waldenström’s macroglobulinemia (WM) in adult patients (Canada, March 2021);
- Registered and reimbursed for the treatment of MCL in patients who have received at least one prior therapy (Israel, April 2021);
- For the treatment of adult patients with WM who have received at least one prior therapy (China, June 2021)**;
- For the treatment of MCL in adult patients who have received at least one prior therapy (Canada, July 2021);
- For the treatment of adult patients with MCL who have received at least one previous therapy (Brazil, August 2021); and
- For the treatment of adult patients with WM (United States, August 2021).
To-date, more than 30 marketing authorization applications in multiple indications have been submitted in the United States, China, the European Union, and more than 20 other countries or regions.
* This indication was approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
** This indication was approved under conditional approval. Complete approval for this indication may be contingent upon results from ongoing randomized, controlled confirmatory clinical trials.
Please see full U.S. Prescribing Information at https://www.beigene.com/PDF/BRUKINSAUSPI.pdf and Patient Information at https://www.beigene.com/PDF/BRUKINSAUSPPI.pdf.
To learn more about BeiGene, please visit www.beigene.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20210901006049/en/
Source: BeiGene, Ltd.
FDA approves BeiGene's Brukinsa in rare blood cancer
Sep. 02, 2021 4:14 AM ET BeiGene, Ltd. (BGNE) By: Mamta Mayani, SA News Editor
- BeiGene (NASDAQ:BGNE) announces that BRUKINSA (zanubrutinib) has received FDA approval for the treatment of adult patients with Waldenström’s macroglobulinemia (WM).
- https://seekingalpha.com/news/3736525-fda-approves-beigenes-brukinsa-in-rare-blood-cancer
- https://seekingalpha.com/symbol/BGNE
- https://www.brukinsa.com/
- https://www.beigene.com/
INVEGA HAFYERA™ (6-month paliperidone palmitate)
Janssen Announces U.S. FDA Approval of INVEGA HAFYERA™
(6-month paliperidone palmitate), First and Only Twice-Yearly Treatment for Adults with Schizophrenia
INVEGA HAFYERA™ offers patients the fewest doses per year for a life less defined by schizophrenia medication
Phase 3 non-inferiority study results showed over 92% of participants were relapse-free at 12 months
Approval is backed by nearly two decades of proven efficacy and safety of Janssen’s long-acting injectable portfolio of schizophrenia medicines
TITUSVILLE, N.J., Sept. 1, 2021 – The Janssen Pharmaceutical Companies of Johnson & Johnson today announced the U.S. Food and Drug Administration (FDA) has approved long-acting atypical antipsychotic INVEGA HAFYERA™ (6-month paliperidone palmitate), the first-and-only twice-yearly injectable for the treatment of schizophrenia in adults. Before transitioning to INVEGA HAFYERA™, patients must be adequately treated with INVEGA SUSTENNA® (1-month paliperidone palmitate) for at least four months, or INVEGA TRINZA® (3-month paliperidone palmitate) for at least one 3-month injection cycle.1
The FDA approval of INVEGA HAFYERA™ is based on the results of a 12-month, randomized, double-blind, non-inferiority Phase 3 global study that enrolled 702 adults (ages 18-70) living with schizophrenia from 20 countries. The results showed non-inferiority of INVEGA HAFYERA™ compared to INVEGA TRINZA® on the primary endpoint of time to first relapse at the end of the 12-month period. Results found that 92.5 percent of patients treated with INVEGA HAFYERA™ and 95 percent treated with INVEGA TRINZA® were relapse-free at 12 months.1 Relapse was defined as psychiatric hospitalization, increase in Positive and Negative Syndrome Scale [PANSS] total score, increase in individual PANSS item scores, self-injury, violent behavior, or suicidal/homicidal ideation.
The safety profile observed in the trial was consistent with previous studies of INVEGA SUSTENNA® and INVEGA TRINZA® with no new safety signals emerging.1 The most common adverse reactions (≥5%) in the INVEGA HAFYERA™ clinical trial were upper respiratory tract infection (12%), injection site reaction (11%), weight increase (9%), headache (7%), and parkinsonism (5%).1
Click-to-Tweet: #BREAKINGNEWS the @US_FDA approved new long-acting injectable treatment with fewer doses a year for adult patients living with #schizophrenia. Click here to learn more: https://bit.ly/3gDSI3j
“Before I found the right treatment plan for me, my symptoms often got in the way of things that I loved to do,” said Patrick, an adult living with schizophrenia and a participant in the clinical trial. “But since my doctor introduced me to Janssen’s long-acting injectable options and my symptoms are controlled, I have the clarity to focus on the present, but also the stability to plan for my future.”
Click-to-Tweet: Patrick, an adult living with schizophrenia, talks about his treatment journey and how a long-acting injectable has helped him stay on track with his goals: https://bit.ly/3gDSI3j
Schizophrenia is a complex and chronic brain disorder in which the symptoms and potential for relapse (or recurrence of symptoms) can impact many aspects of a person’s daily life. On average, an adult with schizophrenia experiences nine relapses in less than six years, often due to missed doses of medication.2 Adults living with schizophrenia and their loved ones face ongoing functional, emotional, and financial burdens. In addition, patients who experience more relapses may have more hospitalizations, which can lead to higher medical costs for patients, hospital systems, and payers.
“For too long, we’ve accepted relapse as a normal part of living with schizophrenia, while research continues to demonstrate that stronger medication adherence means better patient outcomes,” said Gustavo Alva*, M.D., DFAPA, Medical Director at ATP Clinical Research and 6-month paliperidone palmitate clinical trial investigator. “The Phase 3 trial results provide compelling evidence that 6-month paliperidone palmitate offers longer-term symptom control with the fewest doses per year, which may support greater patient adherence.”
Recently, the National Council for Mental Wellbeing and the American Psychiatric Association updated their schizophrenia treatment guidance and guidelines to expand the recommended use of long-acting injectables for appropriate adult patients living with schizophrenia.3,4,5
INVEGA HAFYERA™ is a long-acting injectable treatment that is administered by a healthcare provider in the upper buttocks area every six months. INVEGA HAFYERA™ dissolves slowly into the bloodstream after injection, resulting in continuous treatment and symptom control over six months.1
“Long-acting injectable treatments offer a number of advantages compared to oral medication for schizophrenia, including relief from needing to remember to take medication daily, lower discontinuation rates, and sustained treatment over longer periods,” said Bill Martin, Ph.D., Global Therapeutic Area Head, Neuroscience, Janssen Research & Development, LLC. “Today’s approval enables us to rethink how we manage this chronic disease by offering patients and caregivers the potential for a life less defined by schizophrenia medication.”
Please click here to read the full Prescribing Information, including Boxed WARNING, for INVEGA HAFYERA™.
INDICATION
INVEGA HAFYERA™ is a prescription medicine given by injection by a healthcare provider 1 time every 6 months and used for the treatment of schizophrenia in adults who have been adequately treated with either:
- A 1-time-each-month paliperidone palmitate extended-release injectable suspension for at least 4 months
- A 1-time-every-3-months paliperidone palmitate extended-release injectable suspension for at least 3 months
Learn more at www.janssen.com.
Johnson & Johnson wins FDA approval for long-acting schizophrenia med
Sep. 01, 2021 8:45 AM ET Johnson & Johnson (JNJ) By: Dulan Lokuwithana, SA News Editor1 Comment
- The Janssen Pharmaceutical Companies, a unit of Johnson & Johnson (NYSE:JNJ) announced that the FDA approved its long-acting atypical antipsychotic INVEGA HAFYERA for adults with schizophrenia.
- INVEGA HAFYERA (6-month paliperidone palmitate) is the first and only twice-yearly treatment available for schizophrenia, according to the company.
- https://seekingalpha.com/news/3736021-johnson-johnson-wins-fda-approval-for-long-acting-schizophrenia-med
- https://seekingalpha.com/symbol/JNJ
VOXZOGO® (vosoritide)
European Commission Approves BioMarin's VOXZOGO® (vosoritide) for the Treatment of Children with Achondroplasia from Age 2 Until Growth Plates Close
Achondroplasia is the Most Common Cause of DwarfismFirst Medicine Approved to Treat Children with Achondroplasia in EuropeAug 27, 2021
SAN RAFAEL, Calif., Aug. 27, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the European Commission (EC) has granted marketing authorization for VOXZOGO® (vosoritide), a once daily injection to treat achondroplasia in children from the age of 2 until growth plates are closed, which occurs after puberty when children reach final adult height. Voxzogo is the first medicine to be approved to treat children with achondroplasia in Europe. Voxzogo, a modified C-type natriuretic peptide (CNP), directly targets the underlying pathophysiology of achondroplasia by down regulating fibroblast growth factor receptor 3 (FGFR3) signaling and consequently promoting endochondral bone formation.
"Today represents an important milestone for the European achondroplasia community. For the first time medical professionals in Europe can offer a meaningful targeted therapeutic treatment option for children and families affected by achondroplasia," said Klaus Mohnike, Professor of Paediatrics at Magdeburg University Hospital in Germany and investigator for the Voxzogo clinical program. "Achondroplasia is a serious, progressive, and lifelong condition, which can cause multi-system complications that in some cases require surgical intervention. This regulatory approval is based on improved height gain, one important determinant of day-to-day function for people with achondroplasia, and is a first step to understand the potential benefits of Voxzogo beyond height over the long term."
It is estimated that over 11,000 children across Europe, Middle East, and Africa are affected by achondroplasia and could be eligible for treatment with Voxzogo. Approximately a third of this population are in countries authorized under the EMA license. Also, the French National Agency for Medicines and Health Products Safety (ANSM) granted an Autorisation Temporaire d'Utilisation de cohorte (ATU cohort), or Temporary Authorization for Use to allow access of Voxzogo to begin immediately under an authorized process. An ATU allows access to drugs not yet approved in France, when provided for rare diseases with no alternative options, and when the benefit/risk is presumed positive. The list price in France under the ATU process is 712€ per vial and constitutes flat vial pricing across the spectrum of ages and weights translating to an estimated annual per patient cost of approximately 260,00€ or $300,000, assuming 100% compliance and excluding any discounts. We expect the initial German list price to be consistent with the French ATU price. The Company expects that these list prices will be subject, in one to two years, to material discounts after reimbursement negotiations in key markets such as Germany, France and Italy.
The EC based its decision on the totality of data from the Voxzogo clinical development program including the outcomes from the randomized, double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of Voxzogo. The Phase 3 Study was further supported by the ongoing long-term safety and efficacy from the Phase 2 dose-finding study, which showed that growth rates have been sustained above participants' baseline rates and above the expected annualized growth velocity for untreated children with achondroplasia throughout the five-year observation period for which data are currently available. No acceleration of bone age was observed, suggesting that Voxzogo is not reducing the total duration of growth. The data package included results from an ongoing Phase 2 randomized double-blind study in infants and young children, including extensive pharmacokinetic and biomarker data, as well as preliminary growth data from participants in the 2 to 5-year age cohort. Data in sentinel study participants showed a positive effect on growth following two years of Voxzogo treatment in subjects aged 2 to 5 years. In addition, the data package included data from the Phase 3 extension study and extensive natural history data.
The U.S. New Drug Application (NDA) for Voxzogo is under review by the FDA with a Prescription Drug User Fee Act (PDUFA) target action date of November 20, 2021. The Company successfully closed out the in-person FDA pre-approval inspection of its manufacturing facilities for Voxzogo earlier this year.
Regulatory Status
In January 2021, the Company received notice from the FDA that the NDA for Voxzogo had been granted Priority Review Designation based on the serious pediatric indication it addresses, and the lack of treatment options currently available. Consistent with FDA's policy on changes to review classification for an ongoing application review, the PDUFA action date is not affected by this designation. If approved, the Voxzogo NDA may qualify for a Priority Review Voucher (PRV). A PRV confers priority review to a subsequent drug application that would not otherwise qualify for that designation. The rare pediatric disease review voucher program is designed to encourage development of new drugs and biologics for the prevention or treatment of rare pediatric diseases.
Voxzogo received orphan drug designation from the FDA and EMA for the treatment of children with achondroplasia. The Orphan Drug Designation program is intended to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions.
For additional information, please visit www.biomarin.com
European Commission approves Biomarin's achondroplasia treatment Voxzogo
Aug. 27, 2021 9:15 AM ET BioMarin Pharmaceutical Inc. (BMRN)
By: Aakash Babu, SA News Editor4 Comments
- The European Commission (EC) has granted marketing authorization for BioMarin Pharmaceutical's (NASDAQ:BMRN) Voxzogo (vosoritide) to treat certain children with achondroplasia, a common form of skeletal dysplasia leading to disproportionate short stature.
- https://seekingalpha.com/news/3734858-european-commission-approves-biomarins-achondroplasia-treatment-voxzogo
- https://seekingalpha.com/symbol/BMRN
- https://www.biomarin.com/our-treatments/pipeline/vosoritide-for-achondroplasia/
- https://www.biomarin.com/
FLUMIST® QUADRIVALENT (Influenza Vaccine Live, Intranasal)
FLUMIST® QUADRIVALENT (Influenza Vaccine Live, Intranasal) Vaccine Now Available in the US for Annual Flu Season
Needle-free vaccine option can help reduce influenza infections
The Centers for Disease Control and Prevention (CDC) recommends influenza vaccines as the primary protection against the flu virus to reduce the burden on the US healthcare system
August 31, 2021 07:00 AM Eastern Daylight Time
WILMINGTON, Del.--(BUSINESS WIRE)--AstraZeneca today announced that FLUMIST® QUADRIVALENT (Influenza Vaccine Live, Intranasal) doses are now available in the US for the 2021-2022 influenza season. FLUMIST QUADRIVALENT, the only Food and Drug Administration (FDA)-approved nasal-spray flu vaccine, is indicated for people ages 2 through 49. The bulk of AstraZeneca’s supply for the 2021-2022 flu season is expected to be available by mid-September.
In line with the 2021-2022 recommendations from the CDC’s Advisory Committee on Immunization Practices (ACIP) and the American Academy of Pediatrics (AAP), FLUMIST QUADRIVALENT continues to be an option for flu vaccination in the US this season. Vaccines may not prevent influenza in everyone who gets vaccinated.
The CDC continues to stress the importance of receiving a yearly flu vaccine, citing vaccination as the most effective protection against seasonal flu infection. Additionally, they reinforce that flu vaccination during the 2021-2022 season will help protect the public against preventable illness, as well as help alleviate burden on the healthcare system.
“As the world continues to navigate an unpredictable public health landscape with coinciding flu and COVID-19 viruses, AstraZeneca remains committed to following the science and putting people and their communities first by helping ensure families have access to a needle-free vaccination option,” said Mina Makar, Senior Vice President, US Respiratory & Immunology at AstraZeneca. “Improving patient and public health is at the heart of what we do, and we are proud to be doing our part in fighting the flu again this season by working with our partners to provide FLUMIST QUADRIVALENT to those eligible this year.”
Since FLUMIST QUADRIVALENT is the only FDA-approved flu vaccine to use a needle-free nasal spray administrative technique, it may be preferred by eligible patients. AstraZeneca’s live attenuated influenza vaccine (LAIV) (either as trivalent or quadrivalent formulations) has been approved by the FDA since 2003 and is covered by most health insurance plans. The most common side effects of FLUMIST QUADRIVALENT are runny or stuffy nose, sore throat, and fever over 100°F.
Please note, all available influenza vaccines are manufactured differently and different preparations have different indications as licensed by the FDA. Those interested in receiving a flu vaccine are encouraged to speak with a healthcare provider to determine if FLUMIST QUADRIVALENT is right for them and can use an online vaccine locator tool to find where it is available in their local area.
INDICATION
FLUMIST QUADRIVALENT is a vaccine indicated for active immunization of persons 2-49 years of age for the prevention of influenza disease caused by influenza A subtype viruses and type B viruses contained in the vaccine. FLUMIST QUADRIVALENT is for intranasal administration only.
Please see full Prescribing Information for FLUMIST QUADRIVALENT, including Patient Information.
For more information, please visit www.astrazeneca-us.com
AstraZeneca FluMist vaccine now available in the U.S.
Aug. 31, 2021 9:17 AM ET AstraZeneca PLC (AZN) By: Jonathan M Block, SA News Editor
- The FluMist Quadrivalent intranasal influenza vaccine from AstraZeneca (NASDAQ:AZN) is now available in the U.S. for the 2021-2022 flu season.
- The vaccine is approved from ages 2-49.
- https://seekingalpha.com/news/3735639-astrazeneca-flumist-vaccine-now-available-in-the-us
- https://seekingalpha.com/symbol/AZN
- https://www.astrazeneca.com/
- https://www.flumistquadrivalent.com/
biotecMAX™ April/May/June/July/Aug 2021 Insight
Repatha® (evolocumab)
New Data At ESC Congress 2021 Shows Repatha® (evolocumab) Improves Features Of Plaque Stability In Patients With Acute Coronary Syndrome (ACS)
New Positive Phase 3 Data for Repatha in ACS Patients who Initiated Treatment in the Acute Setting
Repatha in Addition to Optimized Statin Therapy Improves Features of Plaque Morphology Twice as Well as Statins Alone
Results May Provide Mechanistic Insight for Cardiovascular Event Reduction as Seen in Atherosclerotic Cardiovascular Disease Patients in the FOURIER Study
THOUSAND OAKS, Calif., Aug. 27, 2021 /PRNewswire/ -- Amgen (NASDAQ:AMGN) today announced positive data from the HUYGENS Phase 3 study showing that Repatha® (evolocumab) in addition to optimized statin therapy, in comparison with optimized statin therapy alone, significantly improved features of plaque stability in patients with coronary artery disease (CAD). These data are being presented during an oral presentation at ESC Congress 2021, organized by the European Society of Cardiology, Aug. 27-30.
The HUYGENS study met its primary endpoint, with Repatha in addition to optimized statin therapy increasing fibrous cap thickness by 42.7 um in comparison with an increase of 21.5 um (75% increase versus 39%) on optimized statin therapy alone (p=0.01), as measured by optical coherence tomography (OCT). Thus, the addition of Repatha improved this feature twice as well as statins alone. Repatha also improved all of the study's secondary endpoints, including decreasing the maximum lipid arc by -57.5° versus -31.4° (p=0.01), as measured by OCT.
"The majority of acute coronary syndrome events are caused by plaque rupture, and those who have had a heart attack are especially vulnerable to additional episodes of plaque rupture, demonstrating the importance of maintaining the thickness of the fibrous cap to help stabilize plaques," said Stephen J. Nicholls, M.D., Ph.D., professor of Cardiology and director, Monash University Victorian Heart Institute, Melbourne, Australia and first author of the HUYGENS study. "These encouraging results reaffirm the potential of Repatha and highlight the benefits of Repatha in ACS patients who initiated treatment early."
Results from the randomized, double-blind 52-week study in ACS patients on optimized statin therapy demonstrate that Repatha treatment, initiated within a week after the ACS event, reduced LDL-C from 140 to 28 mg/dL (-80%) versus reductions from 142 to 87 mg/dL (-39%) with statin optimization alone. No new safety risks were identified. The most common treatment-emergent adverse events (>3%) were angina pectoris, myalgia, hypertension, diarrhea, fatigue and cough.
"Amgen continues to build a body of evidence to support the clinical profile of Repatha and demonstrate its benefit in patients at elevated risk of suffering another heart attack or stroke," said David M. Reese, M.D., executive vice president of Research and Development at Amgen. "This study builds on the findings from the GLAGOV study and provides evidence that low LDL-C levels can change characteristics of coronary plaque, which may explain the biology of cardiovascular event reduction we saw in the FOURIER study."
While HUYGENS did not evaluate cardiovascular outcomes, the results build on the growing body of evidence already supporting the clinical profile of Repatha. The HUYGENS study results add relevant insights to the science of plaque biology and contribute to our understanding of the important benefits of initiating Repatha after a heart attack. Fifty clinical trials, conducted with over 47,000 patients randomized to Repatha or placebo, have demonstrated the clinical benefits of Repatha, which include reduction in myocardial infarction and stroke, rapid (within four weeks) and dramatic LDL-C lowering over the long term (median 2.2 years), and consistent safety over a five-year treatment period generally consistent with the FOURIER study.7
About the Data
Previous studies include GLAGOV which showed Repatha, when added to optimal statin therapy, reduced plaque burden by decreasing plaque atheroma volume in patients with CAD.8 This was the first study to demonstrate that lowering LDL-C levels through PCSK9 inhibition reduces atherosclerotic plaque burden.
HUYGENS demonstrated that Repatha in addition to optimized statin therapy, in comparison with optimized statin therapy alone, significantly improved a key feature of plaque stability in patients with CAD by increasing the fibrous cap thickness. HUYGENS may offer mechanistic insight for the CV event reduction seen in the FOURIER outcomes study.9
For more information, visit www.amgen.com
About Repatha® (evolocumab)
Repatha is a human monoclonal antibody that inhibits proprotein convertase subtilisin/kexin type 9 (PCSK9). Repatha binds to PCSK9 and inhibits circulating PCSK9 from binding to the low-density lipoprotein (LDL) receptor (LDLR), preventing PCSK9-mediated LDLR degradation and permitting LDLR to recycle back to the liver cell surface. By inhibiting the binding of PCSK9 to LDLR, Repatha increases the number of LDLRs available to clear LDL from the blood, thereby lowering LDL-C levels.
Repatha is approved in 76 countries, including the U.S., Japan, China and in all 27 countries that are members of the European Union. Applications in other countries are pending.
Please see full Prescribing Information.
View original content to download multimedia:https://www.prnewswire.com/news-releases/new-data-at-esc-congress-2021-shows-repatha-evolocumab-improves-features-of-plaque-stability-in-patients-with-acute-coronary-syndrome-acs-301364057.html
SOURCE Amgen
Amgen's Repatha improves features of plaque stability in acute coronary syndrome patients
Aug. 27, 2021 4:59 AM ET Amgen Inc. (AMGN) By: Mamta Mayani, SA News Editor
- Amgen (NASDAQ:AMGN) announces positive data from the HUYGENS Phase 3 study showing that Repatha (evolocumab) in addition to optimized statin therapy, in comparison with optimized statin therapy alone, significantly improved features of plaque stability in patients with coronary artery disease (CAD).
- https://seekingalpha.com/news/3734734-amgens-repatha-improves-features-of-plaque-stability-in-acute-coronary-syndrome-patients
- https://seekingalpha.com/symbol/AMGN
- https://www.repatha.com/
- https://www.amgen.com/
TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor)
PDF Version Aug 26, 2021
Vertex Announces Publication in The New England Journal of Medicine of Phase 3 Results for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in People With Cystic Fibrosis
BOSTON--(BUSINESS WIRE)--Aug. 26, 2021-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced publication in The New England Journal of Medicine (NEJM) of results from a Phase 3 study of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in people with cystic fibrosis (CF) ages 12 years and older who have one copy of the F508del mutation and one gating (F/G) or residual function (F/RF) mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The manuscript includes data on primary and key secondary endpoints, which were previously reported and showed statistically significant and clinically meaningful improvements in lung function and sweat chloride, when compared to active control (either ivacaftor or tezacaftor/ivacaftor), as well as more detailed efficacy and safety data, including subgroup efficacy analyses.
“This study is the third of three Phase 3 clinical trials in the TRIKAFTA program in the 12 years and older age group. Consistent with the prior outcomes, these results show clinically meaningful improvements in pulmonary function, sweat chloride and Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain scores," said Carmen Bozic, M.D., Executive Vice President and Chief Medical Officer, Vertex. “These results are especially notable given that all patients were treated with a CFTR modulator prior to initiating TRIKAFTA.”
“The outcomes within this study, in particular those from the subgroup efficacy analysis by F/G and F/RF, are remarkable because they demonstrate additional benefit on top of standard of care and build further confidence for clinicians to treat people with CF who may have these mutations,” said Steven Rowe, M.D., Director, Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham.
Study 445-104
The data published today are from a global Phase 3, randomized, double-blind, parallel-group study. All patients had a 4-week run-in period of either ivacaftor or tezacaftor/ivacaftor. Following the run-in, 258 patients were randomized to receive TRIKAFTA® or to remain on their prior regimen of ivacaftor or tezacaftor/ivacaftor for 8 weeks. Baseline was measured at the end of the run-in period, prior to the start of the 8-week treatment period. TRIKAFTA® improved the percent predicted forced expiratory volume in 1 second (ppFEV1) by 3.7 percentage points (95% CI, 2.8 to 4.6; P<0.001) from baseline and by 3.5 percentage points (95% CI, 2.2 to 4.7; P<0.001) vs. active control and improved sweat chloride concentration by ‑22.3 mmol/liter (95% CI, ‑24.5 to ‑20.2; P<0.001) from baseline and by ‑23.1 mmol/liter (95% CI, ‑26.1 to ‑20.1; P<0.001) vs. active control. The change in the CFQ-R respiratory domain score was +10.3 points from baseline (95% CI, 8.0 to 12.7) and +8.7 points vs. active control (95% CI, 5.3 to 12.1). Subgroup analyses of patients with F/G and F/RF genotypes are also included in the manuscript. Safety data were consistent with those observed in previous Phase 3 studies with TRIKAFTA®.
INDICATION AND IMPORTANT SAFETY INFORMATION FOR TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) TABLETS
What is TRIKAFTA?
TRIKAFTA is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or another mutation that is responsive to treatment with TRIKAFTA. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if TRIKAFTA is safe and effective in children under 6 years of age.
For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20210826005091/en/
Source: Vertex Pharmaceuticals Incorporated
https://seekingalpha.com/symbol/VRTX
FINTEPLA® (Fenfluramine)
Zogenix Receives Orphan Drug Designation for FINTEPLA® (Fenfluramine) in Japan
08.26.21PDF Version
- Zogenix plans to submit a new drug application (J-NDA) for FINTEPLA in Japan for the treatment of seizures associated with Dravet syndrome by year end.
- Dravet syndrome is a rare, life-long form of epilepsy marked by severe seizures, significant developmental delays, frequent hospitalizations and medical emergencies, and increased risk of premature death.
- Seizures associated with Dravet syndrome are highly resistant to existing anti-epileptic medicines, pointing to a major need for new treatment options.
EMERYVILLE, Calif., Aug. 26, 2021 (GLOBE NEWSWIRE) -- Zogenix (NASDAQ: ZGNX), a global biopharmaceutical company developing rare disease therapies, today announced that the Japanese Ministry of Health, Labour & Welfare (MHLW) has granted Orphan Drug Designation to FINTEPLA® (fenfluramine) oral solution, which Zogenix is developing in Japan as a potential treatment for seizures associated with Dravet syndrome.
Dravet syndrome is a rare infant- and childhood-onset epilepsy marked by frequent and severe treatment-resistant seizures, associated hospitalizations and medical emergencies, significant developmental and motor impairments, and an increased risk of sudden premature death.
The MHLW accreditation of Orphan Drug Designation follows the Japanese Pharmaceutical Affairs & Food Sanitation Council’s (PAFSC) First Committee on Drugs agreement on July 28 to grant orphan drug designation for FINTEPLA for Dravet syndrome. The PAFSC is organized under the MHLW and consists of several expert committees from various fields, who serve as the decision-making body for drug approval (J-NDA, s-NDA) as well as Orphan Drug designation. In Japan, Orphan Drug Designation may be granted to drug candidates designed to treat diseases with fewer than 50,000 patients or diseases that are designated as intractable and the need for improved medical care is high.
Zogenix is on track to submit a J-NDA for FINTEPLA for the treatment of seizures associated with Dravet syndrome later this year. If approved, the product will be made available in Japan through an exclusive distribution agreement with Nippon Shinyaku, Co., Ltd., a leading Japanese pharmaceutical product developer and distributor.
“The orphan drug designation of FINTEPLA in Japan is an important milestone in our mission to meet the unmet needs of severe, rare epilepsy patients around the world,” said Stephen J. Farr, Ph.D., President and Chief Executive Officer of Zogenix. “We are proud to have worked with patients, physicians, and regulators in Japan to achieve this milestone and reinforce our commitment to working with the MHLW to bring this much-needed potential treatment option to patients and families living with Dravet syndrome in Japan.”
The incidence of Dravet syndrome in Japan is estimated to be 1 in 20,000-40,000. There are an estimated 3,000 Dravet syndrome patients in Japan based on the Ministry of Health, Labour and Welfare (MHLW) Patient Survey.
About FINTEPLA®
FINTEPLA is a prescription medicine used to treat the seizures associated with Dravet syndrome in patients 2 years of age and older. FINTEPLA possesses dual activities to inhibit seizures: as a serotonergic agent, acting as a potent 5-HT releaser with agonist activity at 5-HT1D, 2A, and 2C receptors, and as a positive modulator of Sigma1R. Fenfluramine is approved in the United States and Europe and is in development in Japan for the treatment of seizures associated with Dravet syndrome.
Across multiple clinical studies, FINTEPLA demonstrated significant and sustained reduction of convulsive seizures associated with Dravet syndrome. In two pivotal Phase 3 trials, the reduction in convulsive seizure frequency per 28 days was statistically significantly greater for all dose groups of FINTEPLA compared to placebo. Please see important FINTEPLA prescribing and safety information at www.fintepla.com.
Zogenix gets orphan drug status for FINTEPLA in Japan
Aug. 26, 2021 8:25 AM ETZogenix, Inc. (ZGNX)By: Aakash Babu, SA News Editor3 Comments
- Zogenix (NASDAQ:ZGNX) announces that the Japanese Ministry of Health, Labour & Welfare (MHLW) has granted Orphan Drug Designation to FINTEPLA (fenfluramine) oral solution, which is being developed in Japan as a potential treatment for seizures associated with Dravet syndrome.
- https://seekingalpha.com/news/3734418-zogenix-gets-orphan-drug-status-for-fintepla-in-japan
- https://seekingalpha.com/symbol/ZGNX
- https://www.finteplahcp.com/
- https://www.zogenix.com/
Opdivo® (nivolumab)
U.S. Food and Drug Administration Approves Opdivo® (nivolumab) for the Adjuvant Treatment of Patients with High-Risk Urothelial Carcinoma
08/20/2021CATEGORY:
In CheckMate -274, Opdivo nearly doubled median disease-free survival compared to placebo in the intent-to-treat population1
Opdivo is now approved in earlier stages of disease for three types of cancer, including the first and only PD-1 inhibitor approved for urothelial carcinoma in the adjuvant setting
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced that Opdivo®(nivolumab) 240 mg every two weeks or 480 mg every four weeks (injection for intravenous use) was approved by the U.S. Food and Drug Administration (FDA) for the adjuvant treatment of patients with urothelial carcinoma (UC) who are at high risk of recurrence after undergoing radical resection, regardless of prior neoadjuvant chemotherapy, nodal involvement or PD-L1 status.1 The approval is based on the Phase 3 CheckMate -274 trial, which compared Opdivo 240 mg (n=353) to placebo (n=356).1 This application was approved under the FDA’s Real-Time Oncology Review (RTOR) pilot program, which aims to ensure that safe and effective treatments are available to patients as early as possible.2
In the trial, among patients who received Opdivo, median disease-free survival (DFS) was nearly twice as long as in those who received placebo (20.8 months [95% Confidence Interval (CI): 16.5 to 27.6] versus 10.8 months [95% CI: 8.3 to 13.9]).1Opdivo reduced the risk of disease recurrence or death by 30% compared to placebo (Hazard Ratio [HR] 0.70, 95% CI: 0.57 to 0.86; P=0.0008).1 Among patients whose tumors express PD-L1 ≥1%, median DFS was not reached (95% CI: 21.2 to NE; n=140) for those who received Opdivo versus 8.4 months (95% CI: 5.6 to 21.2; n=142) for placebo; Opdivo reduced the risk of disease recurrence or death by 45% (HR 0.55, 95% CI: 0.39 to 0.77; P=0.0005).1
“This approval is a major milestone for patients who have undergone major surgery to remove the bladder or parts of the urinary tract and are in need of additional treatment approaches that can help reduce the risk of their UC returning,” said Matthew D. Galsky,* M.D., a CheckMate -274 primary investigator and Professor of Medicine, Director of Genitourinary Medical Oncology, Co-Director of the Center of Excellence for Bladder Cancer, and Associate Director for Translational Research at The Tisch Cancer Institute and the Icahn School of Medicine at Mount Sinai.3 “Nivolumabprovides a new FDA-approved treatment shown to reduce the risk of disease recurrence or death based on the safety and efficacy findings from CheckMate -274, and has the potential to become a new standard of care option in this setting.”1
About CheckMate -274
CheckMate -274 is a 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.1 The UC pathologic staging criteria that defines high risk patients was ypT2-ypT4a or ypN+ for patients who received neoadjuvant cisplatin chemotherapy or pT3-pT4a or pN+ for patients who did not receive neoadjuvant cisplatin and who also either were ineligible for or refused adjuvant cisplatin chemotherapy.1
Patients were randomized (n=353 and n=356 to the Opdivo and placebo arms, respectively) to receive Opdivo 240 mg or placebo by intravenous infusion over 30 minutes every two weeks until recurrence or unacceptable toxicity for a maximum treatment duration of one year.1 Eligible patients were randomized in a 1:1 ratio to Opdivo or placebo and were stratified by pathologic nodal status (N+ vs. N0/x with <10 nodes removed vs. N0 with ≥10 nodes removed), tumor cells expressing PD-L1 (≥1% vs. <1%/indeterminate as determined by the central lab using the PD L1 IHC 28-8 pharmDx assay), and use of neoadjuvant cisplatin (yes vs. no).1 The major efficacy outcome measures were investigator-assessed DFS in all randomized patients and in patients with tumors expressing PD-L1 ≥1%.1 DFS was defined as time to first recurrence (local urothelial tract, local non-urothelial tract, or distant metastasis), or death.1 Additional efficacy outcome measures included overall survival.1 The FDA-approved dosing for Opdivo is 240 mg every two weeks (30-minute intravenous infusion) or 480 mg every four weeks (30-minute intravenous infusion) until disease recurrence or unacceptable toxicity for up to one year.1
INDICATIONS
OPDIVO® (nivolumab), as a single agent, is indicated for the adjuvant treatment of patients with urothelial carcinoma (UC) who are at high risk of recurrence after undergoing radical resection of UC.
OPDIVO® (nivolumab) is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of completely resected esophageal or gastroesophageal junction cancer with residual pathologic disease in patients who have received neoadjuvant chemoradiotherapy (CRT).
For more information about Bristol Myers Squibb, visit us at BMS.com
Bristol Myers wins FDA approval for Opdivo as adjuvant therapy for bladder cancer
Aug. 20, 2021 12:09 PM ET Bristol-Myers Squibb Company (BMY) By: Dulan Lokuwithana, SA News Editor
- Bristol Myers Squibb (BMY +1.0%) announced that the FDA approved Opdivo (nivolumab) for the adjuvant treatment of patients with urothelial carcinoma (UC) who have a high risk of cancer return after surgical resection.
- https://seekingalpha.com/news/3732759-bristol-myers-wins-fda-approval-for-opdivo-as-adjuvant-therapy-for-bladder-cancer
- https://seekingalpha.com/symbol/BMY
- https://www.bms.com/
RINVOQ® (upadacitinib)
August 24, 2021
European Commission Approves RINVOQ® (upadacitinib) as First JAK Inhibitor in the European Union for the Treatment of Both Adults and Adolescents with Moderate to Severe Atopic Dermatitis
- Approval supported by data from one of the largest registrational Phase 3 programs in atopic dermatitis evaluating RINVOQ (upadacitinib) monotherapy or with topical corticosteroids[1]
- RINVOQ met all primary and secondary endpoints, demonstrating rapid and significant improvement in skin clearance and itch reduction compared to placebo at week 16 and earlier time points (p<0.001)[1]
- Results at week 16 continued to be maintained through week 52[1]
- RINVOQ has a safety profile supported by clinical programs evaluating more than 10,500 patients* across approved indications, and on-market experience in rheumatoid arthritis, psoriatic arthritis and ankylosing spondylitis[2-9]
- Milestone marks the fourth EC-approved indication for RINVOQ[1]
NORTH CHICAGO, Ill., Aug. 24, 2021 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced the European Commission (EC) approved RINVOQ® (upadacitinib), an oral, selective and reversible JAK inhibitor, for the treatment of moderate to severe atopic dermatitis in adults and adolescents 12 years and older who are candidates for systemic therapy.1 The recommended dose of RINVOQ for atopic dermatitis in adults is 15 mg or 30 mg once daily based on individual patient presentation, and 15 mg once daily for adolescents (12-17 years of age) and adults 65 years and older.1 RINVOQ can be used with or without topical corticosteroids (TCS).1
"This is a significant milestone for AbbVie in our pursuit to transform care in atopic dermatitis," said Michael Severino, M.D., vice chairman and president, AbbVie. "We are excited to provide an additional treatment option in Europe to help alleviate the burden of unrelenting itch and rash that many of these patients struggle with in daily life, despite available treatment options."
The EC approval is supported by data from one of the largest registrational Phase 3 programs in atopic dermatitis with more than 2,500 adults and adolescents with moderate to severe disease.1 These studies evaluated the efficacy and safety of RINVOQ monotherapy (Measure Up 1 [MU1] and Measure Up 2 [MU2]) and with topical corticosteroids (AD Up [AU]) compared to placebo.1 In all three studies, the co-primary endpoints were at least a 75 percent improvement in the Eczema Area and Severity Index (EASI 75) and validated Investigator's Global Assessment for Atopic Dermatitis (vIGA-AD) score of 0/1 (clear or almost clear) at week 16.1
Highlights From the Global Phase 3 Atopic Dermatitis Clinical Trial Program1
Across the Phase 3 studies, all primary and secondary endpoints were met with 15 mg and 30 mg doses of RINVOQ compared to placebo. Highlights include:
- Significantly more patients achieved EASI 75 at week 16 in the RINVOQ 15 mg group (MU1: 70%; MU2: 60%; AU: 65%) and the RINVOQ 30 mg group (MU1: 80%; MU2: 73%; AU: 77%), compared to placebo (MU1: 16%; MU2: 13%; AU: 26%).
- Significantly more patients achieved vIGA-AD 0/1 at week 16 in the RINVOQ 15 mg group (MU1: 48%; MU2: 39%; 40: 31%) and the RINVOQ 30 mg group (MU1: 62%; MU2: 52%; AU: 59%) compared to placebo (MU1: 8%; MU2: 5%; AU: 11%).
- Significantly more patients achieved clinically meaningful itch reduction (improvement in Worst Pruritus NRS ≥4) in the RINVOQ 15 mg group (MU1: 52%; MU2: 42%; AU: 52%) and the RINVOQ 30 mg group (MU1: 60%; MU2: 60%; AU: 64%) compared to placebo (MU1: 12%; MU2: 9%; AU: 15%) at week 16.
- Clinically meaningful itch reduction (improvement in Worst Pruritus NRS ≥4) and skin clearance (EASI 75) were observed as early as week 1 and week 2, respectively, in patients treated with either dose of RINVOQ compared to those treated with placebo.
- Results at week 16 continued to be maintained through week 52 in patients treated with either dose of RINVOQ.
The most commonly reported adverse reactions (≥5% of patients) with RINVOQ 15 mg or 30 mg were upper respiratory tract infection (25.4%), acne (15.1%), herpes simplex (8.4%), headache (6.3%) and increased blood creatine phosphokinase (CPK; 5.5%).1 The most common serious adverse reactions were serious infections (<1.0%).1
The Marketing Authorization means that RINVOQ is approved in all member states of the European Union, as well as Iceland, Liechtenstein, Norway and Northern Ireland. RINVOQ is already approved for the treatment of moderate to severe atopic dermatitis in Russia, Saudi Arabia, United Arab Emirates, New Zealand and Chile, and is currently under review in the U.S. by the Food and Drug Administration (FDA).
*10,500 patients includes all patients across all arms (active treatment and placebo) in 8 Phase 3 trials in rheumatoid arthritis, 2 in psoriatic arthritis, 1 in ankylosing spondylitis and 5 in atopic dermatitis.2-9 This includes 344 adolescent patients (aged 12 to 17 years) in the Phase 3 Measure Up 1, Measure Up 2 and, AD Up studies in atopic dermatitis.1,2,5 Of the total number of patients included in these trials, 6,280 were randomized to receive RINVOQ at either dose.2-9
About RINVOQ® (upadacitinib)
Discovered and developed by AbbVie scientists, RINVOQ is a selective and reversible JAK inhibitor that is being studied in several immune-mediated inflammatory diseases.1,10-20 In human cellular assays, RINVOQ preferentially inhibits signaling by JAK1 or JAK1/3 with functional selectivity over cytokine receptors that signal via pairs of JAK2.1 RINVOQ 15 mg is also approved by the U.S. Food and Drug Administration (FDA) for adults with moderately to severely active rheumatoid arthritis, and by the European Commission for adults with moderate to severe active rheumatoid arthritis, adults with active psoriatic arthritis (PsA) and adults with active ankylosing spondylitis (AS). Phase 3 trials of RINVOQ in rheumatoid arthritis, atopic dermatitis, psoriatic arthritis, axial spondyloarthritis, Crohn's disease, ulcerative colitis, giant cell arteritis and Takayasu arteritis are ongoing.12-20
Please see the RINVOQ full SmPC for complete prescribing information at http://www.EMA.europa.eu.
European Commission approves AbbVie's atopic dermatitis treatment RINVOQ
Aug. 24, 2021 8:12 AM ETAbbVie Inc. (ABBV)
By: Aakash Babu, SA News Editor9 Comments
- European Commission (EC) has approved AbbVie's (NYSE:ABBV) RINVOQ for the treatment of moderate to severe atopic dermatitis in adults and adolescents 12 years and older who are candidates for systemic therapy.
- https://seekingalpha.com/news/3733438-european-commission-approves-abbvies-atopic-dermatitis-treatment-rinvoq
- https://seekingalpha.com/symbol/ABBV
- https://www.rinvoq.com/
EYLEA® (aflibercept) Injection
August 24, 2021 at 7:00 AM EDT Back
REGENERON ANNOUNCES ENCOURAGING TOPLINE PHASE 2 DATA OF HIGH-DOSE AFLIBERCEPT IN WET AGE-RELATED MACULAR DEGENERATION
TARRYTOWN, N.Y., Aug. 24, 2021 /PRNewswire/ --
Phase 3 trials in wet AMD and diabetic macular edema fully recruited, with results expected in the second half of 2022
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that an ongoing Phase 2 proof-of-concept trial evaluating an investigational 8 mg dose of aflibercept met its primary safety endpoint, with no new safety signals observed compared to the currently-approved 2 mg dose of EYLEA® (aflibercept) Injection in patients with wet age-related macular degeneration (wet AMD). In this small trial involving 106 patients, a higher proportion of patients in the aflibercept 8 mg group had no retinal fluid (43.4%, n=23/53) compared to patients treated with EYLEA 2 mg (26.4%, n=14/53) (p=0.067) at week 16, the primary efficacy endpoint. At this timepoint patients had received three initial doses (administered at weeks 0, 4 and 8), after which dosing was extended.
Aflibercept 8 mg is currently being evaluated in two large Phase 3 trials in wet AMD and diabetic macular edema (DME), which are expected to report results in the second half of 2022. The trials will assess the safety and efficacy of aflibercept 8 mg for up to two years, with visual acuity as the primary efficacy endpoint at 48 weeks, measured by the Early Treatment Diabetic Retinopathy Study (ETDRS) Best Corrected Visual Acuity (BCVA). Both trials will assess aflibercept 8 mg compared to EYLEA 2 mg, testing dosing intervals of every 12 weeks and every 16 weeks.
"We are cautiously optimistic that these early data suggest that a higher dose of aflibercept may potentially benefit patients with wet AMD, and we look forward to Phase 3 data next year, which will be crucial to understand its overall efficacy and safety," said George D. Yancopoulos, M.D., Ph.D., President and Chief Scientific Officer of Regeneron. "Having worked for nearly two decades in retinal disease, we know that large, robust data sets are required to fully understand whether a medicine can achieve three critical things: improved visual and anatomic outcomes, convenient dosing, and a safety profile that is consistent with EYLEA."
During the initial 16 weeks of the Phase 2 trial, adverse events (AEs) in the study eye occurred in 17.0% (9 of 53) of aflibercept 8 mg patients and 22.6% (12 of 53) of EYLEA 2 mg patients. Serious ocular AEs occurred in two patients overall, one in the aflibercept 8 mg group (retinal tear) and one in the EYLEA 2 mg group (visual acuity reduced). There were no AEs of intraocular inflammation (including occlusive retinal vasculitis), anti-platelet trialists' collaboration (APTC)-defined arterial thromboembolic events or deaths in either patient group.
Wet AMD is the leading cause of vision loss among people 50 years and older in the U.S. Existing anti-VEGF treatments including EYLEA have helped change the course of disease for millions of patients worldwide, and efforts to develop new medicines are focused on further enhancing clinical effectiveness while extending the time between treatment doses. This new, concentrated high-dose aflibercept formulation enables a greater amount of medicine to be administered with each treatment, potentially extending the time between doses while retaining the efficacy and safety profile seen with EYLEA 2 mg.
Aflibercept 8 mg is being jointly developed by Regeneron and Bayer.
About the Phase 2 Trial
The Phase 2 randomized, single-masked trial (NCT04126317) enrolled 106 treatment-naïve patients with wet AMD. The trial was designed to investigate the safety, efficacy and tolerability of high-dose aflibercept (8 mg) compared to the existing approved dose of EYLEA (2 mg). Patients were randomized into two groups, with one group receiving aflibercept 8 mg (n=53) and the other group receiving EYLEA 2 mg (n=53). Patients in both groups received three initial injections (weeks 0, 4 and 8), before the primary endpoint was assessed at week 16, after which dosing was extended to every 12 weeks, or more frequently if required due to persistent or worsening disease. Efficacy was assessed via the presence of retinal fluid in the center subfield on optical coherence tomography (OCT) at this timepoint. The trial will continue through week 44.
Trial participants were at least 50 years of age (mean: 77 years), baseline retinal thickness was 502.1 microns, and the BCVA ETDRS letter score was between 24 to 78 in the study eye (mean: 59 letters).
About the Phase 3 Clinical Program
There are two ongoing pivotal trials to investigate the efficacy and safety of aflibercept 8 mg versus EYLEA 2 mg. In DME, Regeneron is sponsoring the Phase 2/3 multi-center, randomized, double-masked PHOTON trial (NCT04429503). In wet AMD, Bayer is sponsoring the Phase 3 multi-center, randomized, double-masked PULSAR trial (NCT04423718) in treatment naïve patients. Across both trials, patients are randomized into one of three treatment groups, testing aflibercept 8 mg with dosing regimens at either 12- or 16-week intervals or EYLEA 2 mg with an 8-week dosing regimen.
INDICATIONS
EYLEA® (aflibercept) Injection 2 mg (0.05 mL) is indicated for the treatment of patients with Neovascular (Wet) Age-related Macular Degeneration (AMD), Macular Edema following Retinal Vein Occlusion (RVO), Diabetic Macular Edema (DME), and Diabetic Retinopathy (DR).
For additional information about the company, please visit www.regeneron.com
View original content:https://www.prnewswire.com/news-releases/regeneron-announces-encouraging-topline-phase-2-data-of-high-dose-aflibercept-in-wet-age-related-macular-degeneration-301361118.html
SOURCE Regeneron Pharmaceuticals, Inc.
Regeneron's aflibercept met primary safety endpoint in mid-stage wet AMD study
Aug. 24, 2021 9:15 AM ET
Regeneron Pharmaceuticals, Inc. (REGN)Bayer Aktiengesellschaft (BAYRY)Bayer Aktiengesellschaft (BAYZF)
By: Mamta Mayani, SA News Editor
- Regeneron Pharmaceuticals (NASDAQ:REGN) announces that an ongoing Phase 2 trial evaluating an 8 mg dose of aflibercept met its primary safety endpoint, with no new safety signals observed compared to the currently-approved 2 mg dose of EYLEA (aflibercept) Injection in patients with wet age-related macular dhttps://seekingalpha.com/news/3733500-regenerons-aflibercept-met-primary-safety-endpoint-in-mid-stage-wet-amd-studyegeneration (wet AMD).
- https://seekingalpha.com/news/3733500-regenerons-aflibercept-met-primary-safety-endpoint-in-mid-stage-wet-amd-study
- https://seekingalpha.com/symbol/REGN
- https://www.regeneron.com/
- https://eylea.us/
CABOMETYX® (cabozantinib) in Combination with OPDIVO® (nivolumab)
Press Release
Exelixis Announces Partner Takeda and Ono Receive Approval in Japan for CABOMETYX® (cabozantinib) in Combination with OPDIVO® (nivolumab) for the Treatment of Unresectable or Metastatic Renal Cell CarcinomaPDF Version
– Approval based on the phase 3 CheckMate -9ER pivotal trial, which showed CABOMETYX in combination with OPDIVO improved overall survival and doubled median progression-free survival and objective response rate versus sunitinib –
ALAMEDA, Calif.--(BUSINESS WIRE)--Aug. 25, 2021-- Exelixis, Inc. (Nasdaq: EXEL) today announced Takeda Pharmaceutical Company Limited (Takeda), its partner responsible for the clinical development and commercialization of CABOMETYX® (cabozantinib) in Japan, and Ono Pharmaceutical Co., Ltd. (Ono) received approval from the Japanese Ministry of Health, Labor and Welfare to manufacture and market CABOMETYX in combination with OPDIVO® (nivolumab) as a treatment for unresectable or metastatic renal cell carcinoma (RCC).
“We’re excited our partner Takeda, along with Ono, will be able to bring CABOMETYX in combination with OPDIVO to patients with advanced kidney cancer in Japan following regulatory approvals as a first-line treatment in the U.S. and EU earlier this year,” said Michael M. Morrissey, Ph.D., Exelixis’ President and Chief Executive Officer. “With approximately 25,000 new cases of kidney cancer diagnosed in Japan annually, we’re pleased that this important new treatment option will now be available to Japanese patients in need of new therapies.”
The approval is based on CheckMate -9ER, a phase 3 pivotal trial evaluating CABOMETYX in combination with OPDIVO in previously untreated patients with advanced or metastatic RCC compared with sunitinib. In CheckMate -9ER, CABOMETYX in combination with OPDIVO demonstrated superior overall survival (OS) and doubled median progression-free survival (PFS) and objective response rate (ORR) versus sunitinib, with a favorable safety profile.
Per the terms of Exelixis and Takeda’s collaboration and license agreement, Exelixis is eligible to receive a milestone payment of $20 million from Takeda upon the first commercial sale of CABOMETYX in combination with OPDIVO for the treatment of RCC. Exelixis continues to be eligible to receive additional development, regulatory and first-sale milestones for potential future cabozantinib indications and is also eligible for sales revenue milestones and royalties on net sales of cabozantinib in Japan.
Takeda previously received approvals to manufacture and market CABOMETYX in Japan as a treatment for patients with curatively unresectable or metastatic RCC and for patients with unresectable hepatocellular carcinoma (HCC) that has progressed after prior systemic therapy.
About CheckMate -9ER
CheckMate -9ER is an open-label, randomized (1:1), multi-national phase 3 trial evaluating patients with previously untreated advanced or metastatic RCC with a clear cell component. A total of 651 patients (22% favorable risk, 58% intermediate risk, 20% poor risk; 25% PD-L1 ≥1%) were randomized to CABOMETYX at a dose of 40 mg once-daily and OPDIVO (n = 323) versus sunitinib (n = 328). The primary endpoint is PFS; secondary endpoints include OS and ORR. The primary efficacy analysis compares the doublet combination regimen of CABOMETYX and OPDIVO versus sunitinib in all randomized patients. The trial is sponsored by Bristol Myers Squibb and Ono Pharmaceutical Co. and co-funded by Exelixis, Ipsen and Takeda Pharmaceutical Company Limited.
About RCC
The American Cancer Society’s 2021 statistics cite kidney cancer as among the top ten most commonly diagnosed forms of cancer among both men and women in the U.S.1 Clear cell RCC is the most common form of kidney cancer in adults.2 If detected in its early stages, the five-year survival rate for RCC is high; for patients with advanced or late-stage metastatic RCC, however, the five-year survival rate is only 13%.1 Approximately 32,000 patients in the U.S. and 71,000 worldwide will require systemic treatment for advanced kidney cancer in 2021.3
About 70% of RCC cases are known as “clear cell” carcinomas, based on histology.4 The majority of clear cell RCC tumors have below-normal levels of a protein called von Hippel-Lindau, which leads to higher levels of MET, AXL and VEGF.5,6 These proteins promote tumor angiogenesis (blood vessel growth), growth, invasiveness and metastasis.7,8,9,10 MET and AXL may provide escape pathways that drive resistance to VEGF receptor inhibitors.6,7
About CABOMETYX® (cabozantinib)
In the U.S., CABOMETYX tablets are approved for the treatment of patients with advanced RCC; for the treatment of patients with HCC who have been previously treated with sorafenib; and for patients with advanced RCC as a first-line treatment in combination with OPDIVO. CABOMETYX tablets have also received regulatory approvals in the European Union and additional countries and regions worldwide. In 2016, Exelixis granted Ipsen exclusive rights for the commercialization and further clinical development of cabozantinib outside of the U.S. and Japan. In 2017, Exelixis granted exclusive rights to Takeda Pharmaceutical Company Limited for the commercialization and further clinical development of cabozantinib for all future indications in Japan. Exelixis holds the exclusive rights to develop and commercialize cabozantinib in the U.S.
For more information about Exelixis, please visit www.exelixis.com
Exelixis, the Exelixis logo, CABOMETYX, COMETRIQ and COTELLIC are registered U.S. trademarks of Exelixis. MINNEBRO is a registered trademark of Daiichi Sankyo Company, Limited.
OPDIVO® is a registered trademark of Bristol-Myers Squibb Company.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210825005760/en/ Source: Exelixis, Inc.
Exelixis and Takeda win Japanese approval for CABOMETYX in renal cell carcinoma
Aug. 25, 2021 4:39 PM ET Exelixis, Inc. (EXEL), TAK By: Dulan Lokuwithana, SA News Editor3 Comments
- Exelixis (NASDAQ:EXEL) and Takeda (NYSE:TAK) announced that the two companies along with their partner Ono Pharmaceutical received the regulatory approval for CABOMETYX (cabozantinib) in combination with OPDIVO (nivolumab) as a treatment of unresectable or metastatic renal cell carcinoma (RCC).
- https://seekingalpha.com/news/3734223-exelixis-and-takeda-win-japanese-approval-for-cabometyx-in-renal-cell-carcinoma
- https://seekingalpha.com/symbol/EXEL
- https://seekingalpha.com/symbol/TAK
- https://www.exelixis.com/
- https://www.cabometyx.com/
- https://www.opdivo.com/advanced-kidney-cancer/opdivo-cabometyx-combination
Tislelizumab (BGB-A317)
BeiGene Announces Acceptance of a Supplemental Biologics License Application in China for Tislelizumab in Nasopharyngeal Cancer
Aug 22, 2021 7:00 PM
CAMBRIDGE, Mass. & BEIJING--(BUSINESS WIRE)-- BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a global biotechnology company focused on developing and commercializing innovative medicines worldwide, today announced that the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) has accepted a supplemental Biologics License Application (sBLA) for anti-PD-1 antibody tislelizumab in combination with chemotherapy as a first-line treatment for patients with recurrent or metastatic nasopharyngeal cancer (NPC).
“Treatment options for NPC, one of the most common head and neck cancers in China and many parts of Asia, are limited, with chemotherapy continuing to dominate front-line care. Supported by the positive RATIONALE 309 trial, the NMPA acceptance of this sBLA, which is the ninth for tislelizumab in China, represents an incredible milestone in its development history and serves as a validation of this potentially differentiated checkpoint inhibitor,” commented Yong (Ben) Ben, M.D., Chief Medical Officer, Immuno-Oncology at BeiGene. “We look forward to bringing this important immunotherapy to the underserved NPC patient community in China.”
The sBLA is supported by clinical results from a randomized, double-blind, Phase 3 clinical trial RATIONALE 309 (NCT03924986) to evaluate the efficacy and safety of tislelizumab combined with gemcitabine and cisplatin versus placebo combined with gemcitabine and cisplatin as a first-line treatment for patients with recurrent or metastatic NPC. The primary endpoint of this trial is progression-free survival (PFS) as assessed by independent review committee (IRC) in the intention-to-treat (ITT) population; secondary endpoints include overall survival (OS), IRC-assessed overall response rate (ORR) and duration of response (DoR), and investigator-assessed PFS. A total of 263 Asian patients were enrolled and randomized 1:1 to either the tislelizumab plus chemotherapy arm or the placebo plus chemotherapy arm.
As announced in May 2021, RATIONALE 309 met the primary endpoint of PFS at the planned interim analysis. The safety profile of tislelizumab was consistent with its known risks, with no new safety signals identified with the addition of chemotherapy. BeiGene expects to present results from the RATIONALE 309 trial at an upcoming medical conference.
About Tislelizumab
Tislelizumab (BGB-A317) is a humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to FcγR on macrophages. In pre-clinical studies, binding to FcγR on macrophages has been shown to compromise the anti-tumor activity of PD-1 antibodies through activation of antibody-dependent macrophage-mediated killing of T effector cells. Tislelizumab is the first drug from BeiGene’s immuno-oncology biologics program and is being developed internationally as a monotherapy and in combination with other therapies for the treatment of a broad array of both solid tumor and hematologic cancers.
The China National Medical Products Administration (NMPA) has granted tislelizumab approval in five indications, including full approval for first-line treatment of patients with advanced squamous non-small cell lung cancer (NSCLC) in combination with chemotherapy and for first-line treatment of patients with advanced non-squamous NSCLC in combination with chemotherapy; and conditional approval for the treatment of patients with classical Hodgkin’s lymphoma (cHL) who received at least two prior therapies, for the treatment of patients with locally advanced or metastatic urothelial carcinoma (UC) with PD-L1 high expression whose disease progressed during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy, and for the treatment of patients with hepatocellular carcinoma (HCC) who have received at least one systemic therapy. Full approval for these indications is contingent upon results from ongoing randomized, controlled confirmatory clinical trials.
In addition, four supplemental Biologics License Applications for tislelizumab have been accepted by the Center for Drug Evaluation (CDE) of the NMPA and are under review for second- or third-line treatment of patients with locally advanced or metastatic NSCLC who progressed on prior platinum-based chemotherapy, for patients with previously treated, locally advanced unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair-deficient (dMMR) solid tumors, for the treatment of patients with locally advanced or metastatic esophageal squamous cell carcinoma (ESCC) who have disease progression following or are intolerant to first-line standard chemotherapy, and for first-line treatment of patients with recurrent or metastatic nasopharyngeal cancer (NPC).
BeiGene has initiated or completed 17 potentially registration-enabling clinical trials in China and globally, including 13 Phase 3 trials and four pivotal Phase 2 trials.
In January 2021, BeiGene and Novartis entered into a collaboration and license agreement granting Novartis rights to develop, manufacture, and commercialize tislelizumab in North America, Europe, and Japan.
Tislelizumab is not approved for use outside of China.
To learn more about BeiGene, please visit www.beigene.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20210822005013/en/
Source: BeiGene, Ltd.
BeiGene's tislelizumab sBLA accepted in China for nasopharyngeal cancer
Aug. 22, 2021 11:46 PM ET
By: Mamta Mayani, SA News Editor
- BeiGene (NASDAQ:BGNE) announces that the Center for Drug Evaluation of China has accepted a supplemental Biologics License Application (sBLA) for anti-PD-1 antibody tislelizumab in combination with chemotherapy as a first-line treatment for patients with recurrent or metastatic nasopharyngeal cancer (NPC).
- https://seekingalpha.com/news/3732892-beigenes-tislelizumab-sbla-accepted-in-china-for-nasopharyngeal-cancer
- https://seekingalpha.com/symbol/BGNE
- https://www.beigene.com/en-us
Orencia (abatacept)
U.S. Food and Drug Administration Accepts for Priority Review Bristol Myers Squibb’s Application for Orencia (abatacept) for the Prevention of Acute Graft Versus Host Disease (aGvHD)
08/23/2021CATEGORY:
U.S. Food and Drug Administration assigned an action date of December 23, 2021
The sBLA is supported by the Phase 2 ABA2 Trial evaluating Orencia in adults and children to prevent aGvHD
If approved, Orencia would become the first therapy for the prevention of aGvHD
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE:BMY) today announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental Biologics License Application (sBLA) for Orencia (abatacept) for the prevention of moderate to severe acute graft versus host disease (aGvHD) in patients 6 years of age and older receiving unrelated donor hematopoietic stem cell transplantation (HSCT). The FDA granted the application Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) goal date of December 23, 2021.
“While stem cell transplants are an effective treatment for aggressive leukemias and other hematological malignancies, patients who receive stem cell transplants from unrelated and human leukocyte antigens (HLA)-mismatched donors are at high risk for developing aGvHD,” said study lead investigator Leslie Kean, M.D., PhD, Director of the Pediatric Stem Cell Transplantation Program, Boston Children's Hospital/Dana-Farber Cancer Institute. “There is a tremendous need to expand the stem cell donor pool by lowering the risk of aGvHD in both adults and children receiving unrelated donor stem cell transplants.”
Stem cell transplants include infusion of donor T-cells, a type of white blood cell that recognizes and destroys foreign invaders in the recipient’s body, including cancer cells. GvHD occurs when the donor T-cells also recognize the patient’s healthy cells as foreign and start attacking healthy tissues and organs. To initiate this attack, T-cells require activation through a signaling process called co-stimulation. Between 30 and 70 percent of transplant recipients develop aGvHD, depending on donor type, transplant technique, and other features. Orencia, a therapy currently approved to treat various arthritic conditions, binds to and inhibits protein targets involved in co-stimulation, thus inhibiting T-cell activation.
“For patients who receive unrelated donor stem cell transplants, in particular for racial and ethnic minority patient populations, there is a heightened risk of developing aGvHD, a potentially life-threatening medical complication for which there are no approved preventive therapies,” said Mary Beth Harler, M.D., head of Immunology and Fibrosis Development, Bristol Myers Squibb. “We look forward to working with the FDA to bring Orencia to this new patient population and employ pathbreaking science in an effort to address unmet needs of underserved patients.”
The sBLA submitted to the FDA is based on results from the Phase 2 ABA2 trial and a registry trial based on real world evidence. The ABA2 trial assessed the impact of Orencia on the prevention of severe aGvHD, when added to a standard GvHD prophylactic regimen administered to patients with hematologic malignancies receiving a stem cell transplant from an unrelated, HLA-matched or mismatched donor. A mismatch in HLA increases the risk of GvHD. Results from ABA2 showed that treatment with Orencia resulted in a significant reduction in severe aGvHD and associated morbidity without an increase in disease relapse. The findings of the real-world analysis were consistent with those of ABA2.
About ABA2
The ABA2 study was a multicenter, Phase 2 investigator sponsored trial conducted by Dr. Leslie Kean of Boston Children’s Hospital/Dana Farber Cancer Institute. ABA2 had two cohorts: a single arm cohort for patients receiving transplants from mismatched unrelated donors (MMUD) (“7/8” cohort), and a randomized, double blind, placebo-controlled cohort for patients receiving transplants from 8/8 matched unrelated donors (MUD) (“8/8” cohort). All subjects received a calcineurin inhibitor (CNI), with dosing starting on day -2 and continuing through at least Day 100 as tolerated, and methotrexate (MTX) on days one, three, six and 11 (transplant day is Day 0). Orencia-treated subjects received 10 mg/kg Orencia on days -1, 5, 14 and 28.
In the ABA2 clinical trial, addition of Orencia to SOC aGvHD prophylaxis of MTX+CNI resulted in a significantly higher aGvHD-free survival (GFS) rate compared to registry controls in the single-arm 7/8 HLA-matched cohort, and numerically higher severe GFS rate in the double-blind, placebo-controlled 8/8 HLA-matched cohort at 180 days post-transplant.
About ORENCIA ®
ORENCIA® is an immunomodulator that disrupts the continuous cycle of T-cell activation.
U.S. Indications/Usage and Important Safety Information for ORENCIA® (abatacept)
Indications and Usage
Adult Rheumatoid Arthritis: ORENCIA® (abatacept) is indicated for the treatment of adult patients with moderately to severely active rheumatoid arthritis (RA).
Polyarticular Juvenile Idiopathic Arthritis: ORENCIA® (abatacept) is indicated for the treatment of patients 2 years of age and older with moderately to severely active polyarticular juvenile idiopathic arthritis (pJIA).
Adult Psoriatic Arthritis: ORENCIA® (abatacept) is indicated for the treatment of adult patients with active psoriatic arthritis (PsA).
Limitations of Use: The concomitant use of ORENCIA with other potent immunosuppressants [e.g., biologic disease-modifying antirheumatic drugs (bDMARDS), Janus kinase (JAK) inhibitors] is not recommended.
For more information about Bristol Myers Squibb, visit us at BMS.com
FDA grants accelerated review to Bristol Myers' Orencia application for prevention of aGvHD
Aug. 23, 2021 7:19 AM ET Bristol-Myers Squibb Company (BMY)
By: Mamta Mayani, SA News Editor1 Comment
- Under priority review, the FDA has accepted Bristol Myers Squibb's (NYSE:BMY) supplemental Biologics License Application (sBLA) for Orencia (abatacept) for the prevention of moderate to severe acute graft versus host disease (aGvHD) in patients 6 years of age and older receiving unrelated donor hematopoietic stem cell transplantation (HSCT).
- https://seekingalpha.com/symbol/BMY
- https://www.bms.com/
- https://www.orencia.com/
Jardiance® (empagliflozin)
US FDA approves Jardiance® (empagliflozin) to treat adults living with heart failure with reduced ejection fraction
August 18, 2021Download PDF- New treatment options are critical, as approximately half of all people with heart failure die within five years of diagnosis
- Heart failure accounts for more than one million hospitalizations a year in the U.S.
RIDGEFIELD, Conn. and INDIANAPOLIS, Aug. 18, 2021 /PRNewswire/ -- Jardiance® (empagliflozin) 10 mg has been approved by the U.S. Food and Drug Administration to reduce the risk of cardiovascular death plus hospitalization for heart failure in adults with heart failure with reduced ejection fraction (HFrEF), Boehringer Ingelheim and Eli Lilly and Company (NYSE: LLY) announced today.
About EMPEROR-Reduced
EMPEROR–Reduced (NCT03057977) was a phase III international, randomized, double–blind trial that enrolled 3,730 adults with and without type 2 diabetes. All participants had heart failure (New York Heart Association [NYHA] functional class II, III or IV) for at least 3 months and a left ventricular ejection fraction of 40% or less at their most recent assessment. At randomization, 75% of patients were NYHA class II, 24% were class III and 0.5% were class IV.
Participants were randomized to once-daily Jardiance 10 mg (n=1,863) or placebo (n=1,867), on top of treatment with guideline-directed heart failure therapy. Median follow-up time was 16 months. The composite primary endpoint was defined as time to first event of cardiovascular death or hospitalization for heart failure.
What is JARDIANCE? (www.jardiance.com)
JARDIANCE is a prescription medicine used to:
- lower blood sugar along with diet and exercise in adults with type 2 diabetes
- reduce the risk of cardiovascular death in adults with type 2 diabetes who also have known cardiovascular disease
- reduce the risk of cardiovascular death and hospitalization for heart failure (when the heart is weak and cannot pump enough blood to the rest of your body) in adults with heart failure
JARDIANCE is not for people with type 1 diabetes. It may increase their risk of diabetic ketoacidosis (increased ketones in the blood or urine).
JARDIANCE is not for use to lower blood sugar in adults with type 2 diabetes who have severe kidney problems, because it may not work.
For more information, please see Prescribing Information and Medication Guide.
Boehringer Ingelheim and Eli Lilly and Company
In January 2011, Boehringer Ingelheim and Eli Lilly and Company announced an alliance that centers on compounds representing several of the largest diabetes treatment classes. Depending on geographies, the companies either co-promote or separately promote the respective molecules each contributing to the alliance. The alliance leverages the strengths of two of the world's leading pharmaceutical companies to focus on patient needs. By joining forces, the companies demonstrate their commitment, not only to the care of people with diabetes, but also to investigating the potential to address areas of unmet medical need. Clinical trials have been initiated to evaluate the impact of Jardiance on people living with heart failure or chronic kidney disease.
For more information, please visit www.boehringer-ingelheim.us,
For the latest updates, visit http://www.lillydiabetes.com/
To learn more about Lilly, please visit us at lilly.com
View original content to download multimedia:https://www.prnewswire.com/news-releases/us-fda-approves-jardiance-empagliflozin-to-treat-adults-living-with-heart-failure-with-reduced-ejection-fraction-301358260.html
SOURCE Eli Lilly and Company
FDA approves Lilly's Jardiance to treat certain patients with heart failure
Aug. 18, 2021 2:21 PM ET Eli Lilly and Company (LLY) By: Aakash Babu, SA News Editor1 Comment
- Eli Lilly (LLY -0.5%) and development partner Boehringer Ingelheim announce that the U.S. FDA has approved Jardiance (empagliflozin) 10 mg to reduce the risk of cardiovascular death plus hospitalization for heart failure in adults with heart failure with reduced ejection fraction ((HFrEF)).
- https://seekingalpha.com/news/3731988-fda-approves-lillys-jardiance-to-treat-certain-patients-with-heart-failure
- https://seekingalpha.com/symbol/LLY
- https://www.lilly.com/
- https://www.jardiance.com/
Landmark trial demonstrates Jardiance® (empagliflozin) is the first therapy to show statistically significant improvement in heart failure outcomes in adults with preserved ejection fraction
August 27, 2021Download PDF- In this clinical first for adults with heart failure with preserved ejection fraction, Jardiance demonstrated an impressive 21% relative risk reduction in the composite primary endpoint of cardiovascular death or hospitalization for heart failure
- The benefit in the primary endpoint was independent of ejection fraction or diabetes status
- Jardiance also reduced the relative risk of first and recurrent hospitalizations for heart failure by 27% and significantly slowed kidney function decline
- Results from the EMPEROR-Preserved phase III trial were presented today at the European Society of Cardiology Congress 2021 and published in The New England Journal of Medicine
RIDGEFIELD, Conn. and INDIANAPOLIS, Aug. 27, 2021 /PRNewswire/ -- Full results from the landmark EMPEROR-Preserved phase III trial demonstrated that Jardiance® (empagliflozin) showed an impressive 21% relative risk reduction for the composite primary endpoint of cardiovascular death or hospitalization for heart failure in adults with heart failure with preserved ejection fraction (HFpEF) compared with placebo. The benefit was independent of ejection fraction or diabetes status, establishing Jardiance as the first and only treatment to significantly improve outcomes for the full spectrum of heart failure patients. The results were presented today at the European Society of Cardiology (ESC) Congress 2021 and published in The New England Journal of Medicine, Boehringer Ingelheim and Eli Lilly and Company (NYSE: LLY) announced.
Key secondary endpoint analyses from the trial showed that Jardiance also reduced the relative risk of first and recurrent hospitalizations for heart failure by 27% and significantly slowed kidney function decline.
EVRENZO™ (roxadustat)
Astellas Receives European Commission Approval for First-in-Class EVRENZO™ (roxadustat) for Adult Patients with Symptomatic Anemia of Chronic Kidney Disease
Roxadustat is the first orally administered hypoxia-inducible factor (HIF) prolyl hydroxylase (PH) inhibitor available for adult patients with anemia associated with chronic kidney disease in Europe
TOKYO, Aug. 19, 2021 /PRNewswire/ -- Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., "Astellas") and FibroGen, Inc. (Nasdaq: FGEN, CEO: Enrique Conterno, "FibroGen") today announced that the European Commission (EC) has approved EVRENZO™ (roxadustat) for the treatment of adult patients with symptomatic anemia associated with chronic kidney disease (CKD).
"We are very pleased EVRENZO has been approved as the first oral HIF-PH inhibitor to treat adult patients with symptomatic anemia associated with CKD in the European Union," said Steven Benner, M.D., M.H.S., President of Development, Astellas. "Today's approval provides patients, regardless of dialysis status, with a first-in-class treatment option to address the multifaceted nature of this condition. We look forward to making roxadustat available to adult patients with anemia of CKD in countries across the European Union."
Roxadustat is the first orally administered HIF-PH inhibitor available in the European Union. Roxadustat increases hemoglobin (Hb) levels through a different mechanism of action compared to injectable erythropoiesis-stimulating agents (ESAs) which are typically co-administered with intravenous iron. As a HIF-PH inhibitor, roxadustat activates the body's natural response to reduced oxygen levels in the blood. This response involves the regulation of multiple, coordinated processes that allow management of anemia with a reduced use of intravenous iron.
"HIF-PH inhibitors represent a major advance in the treatment of anemia of CKD," said Mark Eisner, M.D., M.P.H., Chief Medical Officer, FibroGen. "Roxadustat provides a novel breakthrough for patients who suffer from this condition."
This approval follows the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) positive opinion to authorize roxadustat in June6 based on results from a comprehensive pivotal Phase 3 program comprising of eight multicenter and randomized studies, which involved 9,600 patients worldwide.7-12 The results of this program showed roxadustat was efficacious in achieving and maintaining target Hb levels (10-12g/dL) in patients with symptomatic anemia of CKD regardless of dialysis status and irrespective of prior ESA treatment.7-11 The safety profile observed in the roxadustat development program is reflective of the CKD populations studied and comparable to ESAs.7-12
https://www.fibrogen.com/roxadustat
For more information, please visit our website at https://www.astellas.com/en.
For more information, please visit www.fibrogen.com.
FibroGen rises as EU approval of Roxadustat prompts Raymond James upgrade
Aug. 20, 2021 8:06 AM ET FibroGen, Inc. (FGEN)Astellas Pharma Inc. (ALPMF)
By: Dulan Lokuwithana, SA News Editor
- Raymond James has upgraded FibroGen (NASDAQ:FGEN) to market perform from underperform after the company joined Astellas Pharma (OTCPK:ALPMF) on Thursday to announce the European approval of EVRENZO (roxadustat) for adults with anemia linked to chronic kidney disease (CKD).
- https://seekingalpha.com/news/3732643-fibrogen-rises-as-eu-approval-of-roxadustat-prompts-raymond-james-upgrade
- https://seekingalpha.com/symbol/FGEN
- https://seekingalpha.com/symbol/ALPMF
- https://www.astellas.com/en/
Abecma (Idecabtagene Vicleucel)
Bristol Myers Squibb Receives European Commission Approval for Abecma (Idecabtagene Vicleucel), the First Anti-BCMA CAR T Cell Therapy for Relapsed and Refractory Multiple Myeloma
08/19/2021CATEGORY:
Abecma represents the only cell therapy approved for multiple myeloma
Approval of Abecma is based on the pivotal KarMMa trial of patients worldwide, including five European countries, which demonstrated rapid, deep and durable responses with a well-understood and predictable safety profile
Abecma expands upon Bristol Myers Squibb’s leadership in cell therapy research and multiple myeloma, offering an innovative option to patients in need
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced that the European Commission (EC) has granted Conditional Marketing Authorization for Abecma (idecabtagene vicleucel; ide-cel), a first-in-class B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy, for the treatment of adult patients with relapsed and refractory multiple myeloma, who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody and have demonstrated disease progression on the last therapy.
Abecma is the first and only CAR T cell therapy approved that is directed to recognize and bind to BCMA, a protein that is nearly universally expressed on cancer cells in multiple myeloma, leading to the death of BCMA-expressing cells.1Abecma is delivered via a single infusion with a target dose of 420 x 106 CAR-positive viable T cells within a range of 260 to 500 x 106 CAR-positive viable T cells. Abecma is approved for use in all European Union (EU) member states.*
“The EC approval of Abecma is an important milestone for the treatment of multiple myeloma, and moves us closer to offering a first-in-class, personalized therapy to patients in Europe battling this incurable disease after exhausting prior treatment options with the three standards of care,” said Samit Hirawat, M.D., chief medical officer, Bristol Myers Squibb. “With this third regulatory approval for Abecma worldwide, we are proud to be advancing the science of cell therapy and continuing to bring this first anti-BCMA CAR T cell therapy to patients in need.”
Abecma Clinical Trial Results
The efficacy of Abecma is based on results from the pivotal KarMMa study in which 128 patients with relapsed and refractory multiple myeloma who had received at least three prior therapies including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody and were refractory to the last treatment regimen were treated with Abecma.7
In the study, the overall response rate (ORR) was 73% (95% CI: 66-81), and 33% of patients achieved a complete response (CR; 95% CI: 25-41). Onset of response was rapid with a median time to response of one month. In addition, responses were durable, with a median duration of response of 10.6 months (95% CI: 8.0 – 11.4), and 23 months (95% CI: 11.4 – 23.3) for those who achieved a CR.7
In a pooled safety analysis of 184 patients treated with Abecma in the KarMMa and CRB-401 studies, cytokine release syndrome (CRS) occurred in 81% of patients, with Grade >3 CRS, using the Lee grading system, occurring in 5.4% of patients. There was one case of fatal (Grade 5) CRS reported. The median time to onset of CRS was one day (range: 1-17 days) and the median duration of CRS was five days (range: 1-63 days). Any grade neurotoxicity (NT) of the 128 patients receiving Abecma in the KarMMa study occurred in 18% of patients, including Grade 3 events in 3.1% of patients, with no Grade 4 or 5 events occurring. The median time to onset of NT was two days (range: 1-10 days) and the median duration was three days (range: 1-26 days).7
The most common (>20%) adverse reactions in the pooled safety analysis included neutropenia, CRS, anaemia, thrombocytopenia, infections - pathogen unspecified, leucopenia, fatigue, diarrhoea, hypokalaemia, hypophosphataemia, nausea, lymphopenia, pyrexia, cough, hypocalcaemia, infections - viral, headache, hypomagnesaemia, upper respiratory tract infection, arthralgia, and oedema peripheral. The most common Grade 3 or 4 adverse reactions were neutropenia (88.6%), anaemia (58.2%), thrombocytopenia (53.5%), leucopenia (45.1%), lymphopenia (30.4%), infections - pathogen unspecified (17.9%), hypophosphataemia (17.4%), febrile neutropenia (14.7%), hypocalcaemia (7.1%), infections - viral (7.1%), pneumonia (6.0%), CRS (5.4%), hypertension (5.4%) and hyponatraemia (5.4%).7
Please see full Prescribing Information, including Boxed WARNINGS and Medication Guide, and Summary of Product Characteristics for ABECMA.
About Abecma
Abecma is the first-in-class B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy, first approved in the U.S. in March 2021 for the treatment of adult patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody. Abecma is also approved in Canada for relapsed and refractory multiple myeloma. Abecma recognizes and binds to BCMA on the surface of multiple myeloma cells leading to CAR T cell proliferation, cytokine secretion, and subsequent cytolytic killing of BCMA-expressing cells.
Abecma is being jointly developed and commercialized in the U.S. as part of a Co-Development, Co-Promotion, and Profit Share Agreement with Bristol Myers Squibb and bluebird bio. Bristol Myers Squibb will assume sole responsibility for Abecma drug product manufacturing and commercialization outside of the U.S.
For more information about Bristol Myers Squibb, visit us at BMS.com
EC approves Bristol Myers Squibb's Abecma to treat certain patients with multiple myeloma
Aug. 19, 2021 9:50 AM ETBristol-Myers Squibb Company (BMY)By: Aakash Babu, SA News Editor
- The European Commission (EC) has granted conditional marketing authorization to Bristol Myers Squibb's (BMY +0.1%) Abecma for the treatment of certain patients with relapsed and refractory multiple myeloma.
- https://seekingalpha.com/news/3732327-ec-approves-bristol-myers-squibbs-abecma-to-treat-certain-patients-with-multiple-myeloma
- https://seekingalpha.com/symbol/BMY
- https://seekingalpha.com/symbol/BLUE
biotecMAX™ April/May/June/July/Aug 2021 Insight
Opdivo (nivolumab) + Yervoy (ipilimumab) and Opdivo + Chemotherapy
EMA Validates Bristol Myers Squibb’s Applications for Opdivo (nivolumab) + Yervoy (ipilimumab) and Opdivo + Chemotherapy as First-Line Treatments for Unresectable Advanced, Recurrent or Metastatic Esophageal Squamous Cell Carcinoma
08/17/2021CATEGORY:
Applications based on positive results from the Phase 3 CheckMate -648 trial, in which both Opdivo-based combinations demonstrated a significant survival benefit over chemotherapy alone
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced that the European Medicines Agency (EMA) has validated its Type II Variation Marketing Authorization Applications (MAA) for both Opdivo (nivolumab) in combination with Yervoy (ipilimumab) and Opdivo in combination with fluoropyrimidine- and platinum-containing chemotherapy as first-line treatments for adult patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC). Validation of these applications confirm that the submissions are complete and begins the EMA’s centralized review process.
“Outcomes for patients with advanced esophageal squamous cell carcinoma treated with chemotherapy alone remain poor, and there is a clear need for additional options beyond this long-standing standard of care,” said Ian M. Waxman, M.D., development lead, gastrointestinal cancers, Bristol Myers Squibb. “The validation of our applications moves us a step closer to potentially bringing these two Opdivo-based regimens to patients in the EU who may benefit.”
The applications are based on results from the pivotal Phase 3 CheckMate -648 trial, in which both Opdivo-based treatment combinations — Opdivo plus Yervoy and Opdivo plus chemotherapy — demonstrated a statistically significant and clinically meaningful overall survival (OS) benefit compared to chemotherapy at the pre-specified interim analysis in patients with unresectable advanced or metastatic esophageal squamous cell carcinoma (ESCC) with tumor cell PD-L1 expression ≥1%, as well as in the all-randomized population. Opdivo plus Yervoy is the first dual immunotherapy combination to demonstrate a superior survival benefit versus chemotherapy in this setting. The safety profiles of Opdivo and chemotherapy and of Opdivo plus Yervoy were consistent with the known safety profiles of the individual components.
Results from CheckMate -648 were presented in an oral session during the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting and were selected for the official ASCO press program.
Bristol Myers Squibb thanks the patients and investigators involved in the CheckMate -648 trial.
About CheckMate -648
CheckMate -648 is a randomized Phase 3 study evaluating Opdivo plus Yervoy or Opdivo plus fluorouracil and cisplatin against fluorouracil plus cisplatin alone in patients with unresectable advanced or metastatic esophageal squamous cell carcinoma. The primary endpoints of the trial are overall survival (OS) and progression-free survival (PFS) by blinded independent central review (BICR) in patients whose tumors express PD-L1 ≥1% for both Opdivo-based combinations versus chemotherapy. Secondary endpoints of the trial include OS and PFS by BICR in the all-randomized population.
In the Opdivo plus Yervoy arm, patients received treatment with Opdivo 3 mg/kg every 2 weeks and Yervoy 1 mg/kg every 6 weeks up to 24 months or until disease progression or unacceptable toxicity. In the Opdivo plus chemotherapy arm, patients received treatment with Opdivo 240 mg on Day 1 and Day 15, fluorouracil 800 mg/m²/day on Day 1 through Day 5 (for 5 days), and cisplatin 80 mg/m² on Day 1 of four-week cycle. Patients received Opdivo for up to 24 months or until disease progression or unacceptable toxicity, and chemotherapy until disease progression or unacceptable toxicity.
About Opdivo
Opdivo is a programmed death-1 (PD-1) immune checkpoint inhibitor that is designed to uniquely harness the body’s own immune system to help restore anti-tumor immune response. By harnessing the body’s own immune system to fight cancer, Opdivo has become an important treatment option across multiple cancers.
Opdivo’s leading global development program is based on Bristol Myers Squibb’s scientific expertise in the field of Immuno-Oncology and includes a broad range of clinical trials across all phases, including Phase 3, in a variety of tumor types. To date, the Opdivo clinical development program has treated more than 35,000 patients. The Opdivo trials have contributed to gaining a deeper understanding of the potential role of biomarkers in patient care, particularly regarding how patients may benefit from Opdivo across the continuum of PD-L1 expression.
In July 2014, Opdivo was the first PD-1 immune checkpoint inhibitor to receive regulatory approval anywhere in the world. Opdivo is currently approved in more than 65 countries, including the United States, the European Union, Japan and China. In October 2015, the Company’s Opdivo and Yervoy combination regimen was the first Immuno-Oncology combination to receive regulatory approval for the treatment of metastatic melanoma and is currently approved in more than 50 countries, including the United States and the European Union.
About Yervoy
Yervoy is a recombinant, human monoclonal antibody that binds to the cytotoxic T-lymphocyte-associated antigen-4 (CTLA-4). CTLA-4 is a negative regulator of T-cell activity. Yervoy binds to CTLA-4 and blocks the interaction of CTLA-4 with its ligands, CD80/CD86. Blockade of CTLA-4 has been shown to augment T-cell activation and proliferation, including the activation and proliferation of tumor infiltrating T-effector cells. Inhibition of CTLA-4 signaling can also reduce T-regulatory cell function, which may contribute to a general increase in T-cell responsiveness, including the anti-tumor immune response. On March 25, 2011, the U.S. Food and Drug Administration (FDA) approved Yervoy 3 mg/kg monotherapy for patients with unresectable or metastatic melanoma. Yervoy is approved for unresectable or metastatic melanoma in more than 50 countries. There is a broad, ongoing development program in place for Yervoy spanning multiple tumor types.
INDICATIONS
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express PD-L1 (≥1%) as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab) and 2 cycles of platinum-doublet chemotherapy, is indicated for the first-line treatment of adult patients with metastatic or recurrent non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab) is indicated for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving OPDIVO.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with unresectable malignant pleural mesothelioma (MPM).
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of patients with intermediate or poor risk advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab), in combination with cabozantinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab) is indicated for the treatment of patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy.
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with classical Hodgkin lymphoma (cHL) that has relapsed or progressed after autologous hematopoietic stem cell transplantation (HSCT) and brentuximab vedotin or after 3 or more lines of systemic therapy that includes autologous HSCT. This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab) is indicated for the treatment of patients with recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN) with disease progression on or after platinum-based therapy.
OPDIVO® (nivolumab) is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of adult and pediatric (12 years and older) patients with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection.
OPDIVO® (nivolumab) is indicated for the treatment of patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) after prior fluoropyrimidine- and platinum-based chemotherapy.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of completely resected esophageal or gastroesophageal junction cancer with residual pathologic disease in patients who have received neoadjuvant chemoradiotherapy (CRT).
OPDIVO® (nivolumab), in combination with fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the treatment of patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma.
For more information about Bristol Myers Squibb, visit us at BMS.com
EMA validates Bristol Myers' Opdivo combo applications in esophageal cancer
Aug. 17, 2021 7:07 AM ET Bristol-Myers Squibb Company (BMY) By: Mamta Mayani, SA News Editor
- Bristol Myers Squibb (NYSE:BMY) announces that the EMA has validated its Type II Variation Marketing Authorization Applications (MAA) for both Opdivo (nivolumab) in combination with Yervoy (ipilimumab) and Opdivo + fluoropyrimidine- and platinum-containing chemotherapy as first-line treatments for adult patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC).
- https://seekingalpha.com/news/3731281-ema-validates-bristol-myers-opdivo-combo-applications-in-esophageal-cancer
- https://seekingalpha.com/symbol/BMY
- https://www.bms.com/
VENTANA MMR RxDx Panel
Roche receives FDA approval for first companion diagnostic to identify dMMR solid tumour patients eligible for anti-PD-1 immunotherapy
- Cancer is the second leading cause of death worldwide, with nearly 10 million deaths annually.1, 2 In the U.S., approximately 1.9 million new cancer cases are expected to be diagnosed in 2021.1
- Based on cancer biomarkers, the first-of-its-kind VENTANA MMR RxDx Panel helps determine which solid tumour patients may benefit from GSK immunotherapy.
- Roche/GSK collaboration represents an important step towards a personalised healthcare strategy for certain solid tumour patients.
Basel, 18 August 2021 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced U.S. Food and Drug Administration (FDA) approval of the VENTANA MMR RxDx Panel, advancing the company's commitment to personalised healthcare through tests that determine which patients are most likely to benefit from specific and targeted therapies. The VENTANA MMR RxDx Panel is the first companion diagnostic test to aid in identifying patients whose solid tumours are deficient in DNA mismatch repair (MMR), who may be eligible for JEMPERLI (dostarlimab-gxly) monotherapy, an anti-PD-1 immunotherapy from GSK. The test evaluates a panel of MMR proteins in tumours to provide important treatment information to clinicians.
MMR is a naturally occurring mechanism that scans our DNA, correcting errors that cause disease. When MMR is deficient (dMMR), cells mutate, which can lead to cancer. While MMR deficiency is most common in endometrial cancer, other high prevalence dMMR tumour types include gastric, colorectal, small intestine, cervical and neuroendocrine cancers. In the U.S., prevalence of dMMR across patients with solid tumours has been estimated at 14 percent.3 PD-1 inhibitors can be effective treatment in cancers with MMR deficiency.
FDA approval of the VENTANA MMR RxDx Panel provides clinicians with access to a fully automated panel of MMR biomarkers tested by immunohistochemistry (IHC), enabling impactful treatment decisions for patients. JEMPERLI was approved by the FDA on 17 August 2021 for the treatment of adult patients with dMMR recurrent or advanced solid tumours, as determined by an FDA-approved test, that have progressed on or following prior treatment and who have no satisfactory alternative treatment options. This indication received accelerated approval based on tumour response rate and durability of response. Continued approval for this indication may depend on verification and description of clinical benefit in a confirmatory trial(s).
The VENTANA MMR RxDx Panel and JEMPERLI were earlier approved by the FDA for use in endometrial cancer in April 2021.
Read more about Roche innovation in MMR biomarker testing.
About the VENTANA MMR RxDx Panel
The VENTANA MMR RxDx Panel is a label expansion of Roche’s current on-market VENTANA MMR IHC Panel. The VENTANA MMR RxDx Panel is intended for the assessment of expression of MMR proteins in formalin-fixed, paraffin-embedded (FFPE) tumour tissue stained with OptiView DAB IHC Detection Kit and ancillary reagents in the panel for VENTANA anti-MLH1 (M1), VENTANA anti-MSH2 (G219-1129) and VENTANA anti-MSH6 (SP93) and OptiView DAB IHC Detection Kit with the OptiView Amplification Kit and ancillary reagents for VENTANA anti-PMS2 (A16-4) on a BenchMark ULTRA instrument. DNA mismatch repair (MMR) proteins have been clinically proven to be predictive biomarkers for PD-1 targeted therapy; specifically, a loss of expression of one or more MMR proteins might predict an increased likelihood of response to such therapy.4,5,6 PD-1 inhibitors can be effective in cancers with MMR deficiency.4,6 MMR is a conserved molecular mechanism that functions to correct the improper base substitutions that spontaneously occur during DNA replication. Defects in the MMR machinery have been attributed to mutations in the MMR proteins.
Roche announces FDA approval of companion diagnostic for dMMR solid tumors
Aug. 18, 2021 12:04 PM ET Roche Holding AG (RHHBY)AnaptysBio, Inc. (ANAB)GlaxoSmithKline plc (GSK)
By: Dulan Lokuwithana, SA News Editor
- Roche (OTCQX:RHHBY +0.2%) said that the FDA approved VENTANA MMR RxDx Panel, a companion diagnostic to help identify patients with solid tumors that are deficient in DNA mismatch repair (MMR) who may be eligible for anti-PD-1 immunotherapy.
- https://seekingalpha.com/news/3731933-roche-announces-fda-approval-of-companion-diagnostic-for-dmmr-solid-tumors
- https://seekingalpha.com/symbol/RHHBY
- https://www.roche.com/
- https://diagnostics.roche.com/global/en/products/tests/ventana-mmr-rxdx-panel.html
Impella 2.5® and Impella CP® devices
FDA Grants Breakthrough Device Designation to Impella ECP, the World’s Smallest Heart Pump
Play Video
This animation shows Impella ECP, the world's smallest heart pump. Impella ECP is placed percutaneously into the heart's left ventricle, expands, and supports the heart’s pumping function, providing flow greater than 3.5 L/min.
August 18, 2021 08:02 AM Eastern Daylight Time
DANVERS, Mass.--(BUSINESS WIRE)--The United States Food and Drug Administration (FDA) has granted breakthrough device designation to Abiomed’s (NASDAQ: ABMD) Impella ECP expandable percutaneous heart pump. The designation means the FDA will prioritize Impella ECP’s regulatory review processes including design iterations, clinical study protocols and pre-market approval (PMA) application.
Impella ECP is the smallest heart pump in the world and the first to be compatible with small bore access and closure techniques. It measures 9 French (3 millimeters) in diameter upon insertion and removal from the body. While in the heart, it expands to support the heart’s pumping function, providing flow greater than 3.5 L/min.
The FDA granted breakthrough device designation in part based on positive clinical data from the first 21 Impella ECP patients treated as part of an FDA regulated early feasibility study. In granting the designation, the FDA determined Impella ECP meets the FDA’s stringent requirements for a breakthrough device.
“This is yet another validation from the FDA of the clinical benefits of Impella technology and an affirmation of the innovative nature of Impella ECP which, due to its smaller vascular access size, has the potential to provide even safer procedures and be available to more patients who need hemodynamic support for coronary revascularization,” said Chuck Simonton, MD, Abiomed’s chief medical officer.
In the United States, an estimated 440,000 patients are indicated and yet undertreated for high-risk PCI. Impella ECP’s size may enable more physicians to provide critical hemodynamic support to coronary artery disease patients who need it.
The first Impella ECP patient in the world is Robert Matthews, an 80-year-old retired auto worker from Detroit, a father of four, and grandfather of eleven. For more than 20 years, Robert lived with heart disease and endured multiple procedures. In 2020, he was referred to Amir Kaki, MD, an interventional cardiologist and director of mechanical circulatory support at Ascension St. John Hospital in Detroit. Dr. Kaki discovered multiple blockages and poor heart function and identified Robert as an appropriate candidate for a Protected PCI with Impella. Robert became the first patient in the world treated with Impella ECP when Dr. Kaki inserted the heart pump prior to opening blockages and placing stents.
Two days later, Robert returned home, and his family and friends immediately noticed his renewed energy. Today, Robert is grateful for the cutting-edge technology that restored his quality of life.
Caution: Impella ECP is an investigational device, limited by federal law to investigational use only.
ABOUT IMPELLA HEART PUMPS
The Impella 2.5® and Impella CP® devices are U.S. FDA approved to treat certain advanced heart failure patients undergoing elective and urgent percutaneous coronary interventions (PCI), such as stenting or balloon angioplasty, to reopen blocked coronary arteries.
The Impella 2.5, Impella CP, Impella CP with SmartAssist®, Impella 5.0®, Impella LD®, and Impella 5.5® with SmartAssist® are U.S. FDA approved to treat heart attack or cardiomyopathy patients in cardiogenic shock, and have the unique ability to enable native heart recovery, allowing patients to return home with their own heart.
For additional information, please visit: www.abiomed.com.
Impella®
The World's Smallest Heart Pump
https://www.abiomed.com/products-and-services/impella
FDA grants breakthrough device status to Abiomed's Impella ECP heart pump
Aug. 18, 2021 8:41 AM ETAbiomed, Inc. (ABMD)
By: Aakash Babu, SA News Editor2 Comments
- The U.S. FDA has granted breakthrough device designation to Abiomed's (NASDAQ:ABMD) Impella ECP expandable percutaneous heart pump.
- https://seekingalpha.com/news/3731803-fda-grants-breakthrough-device-status-to-abiomeds-impella-ecp-heart-pump
- https://seekingalpha.com/symbol/ABMD
- https://www.heartrecovery.com/products-and-services/impella/impella-25
- https://www.abiomed.com/
KEYTRUDA® (pembrolizumab) Plus LENVIMA® (lenvatinib)
FDA Approves KEYTRUDA® (pembrolizumab) Plus LENVIMA® (lenvatinib) Combination for First-Line Treatment of Adult Patients With Advanced Renal Cell Carcinoma (RCC)
August 11, 2021 6:30 pm ET
KEYTRUDA Plus LENVIMA Is Now Approved for Two Types of Cancer, Including Advanced RCC
Based on Phase 3 CLEAR/KEYNOTE-581 Trial, KEYTRUDA Plus LENVIMA Significantly Reduced Risk of Disease Progression or Death by 61% Versus Sunitinib
KENILWORTH, N.J. & WOODCLIFF LAKE, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, and Eisai today announced that the U.S. Food and Drug Administration (FDA) has approved the combination of KEYTRUDA, Merck’s anti-PD-1 therapy, plus LENVIMA, the orally available multiple receptor tyrosine kinase inhibitor discovered by Eisai, for the first-line treatment of adult patients with advanced renal cell carcinoma (RCC). The approval is based on results from the pivotal Phase 3 CLEAR (Study 307)/KEYNOTE-581 trial, in which KEYTRUDA plus LENVIMA demonstrated statistically significant improvements versus sunitinib in the efficacy outcome measures of progression-free survival (PFS), overall survival (OS) and confirmed objective response rate (ORR).
For PFS, KEYTRUDA plus LENVIMA reduced the risk of disease progression or death by 61% (HR=0.39 [95% CI: 0.32-0.49]; p<0.0001) with a median PFS of 23.9 months versus 9.2 months for sunitinib. For OS, KEYTRUDA plus LENVIMA reduced the risk of death by 34% (HR=0.66 [95% CI: 0.49-0.88]; p=0.0049) versus sunitinib. Additionally, the confirmed ORR was 71% (95% CI: 66-76) (n=252) for patients who received KEYTRUDA plus LENVIMA versus 36% with sunitinib (95% CI: 31-41) (n=129). KEYTRUDA plus LENVIMA achieved a complete response (CR) rate of 16% and partial response (PR) rate of 55% versus a CR rate of 4% and a PR rate of 32% for those who received sunitinib.
Data Supporting the Approval
The approvalwas based on data from the CLEAR (Study 307)/KEYNOTE-581 trial (ClinicalTrials.gov, NCT02811861), a Phase 3, multicenter, open-label, randomized trial conducted in 1,069 patients with advanced RCC in the first-line setting. Patients were enrolled regardless of PD-L1 tumor expression status. Patients with active autoimmune disease or a medical condition that required immunosuppression were ineligible. Randomization was stratified by geographic region (North America and Western Europe vs. “Rest of the World”) and Memorial Sloan Kettering Cancer Center (MSKCC) prognostic groups (favorable vs. intermediate vs. poor risk).
Patients were randomized (1:1:1) to one of the following treatment arms:
- LENVIMA (20 mg orally once daily) in combination with KEYTRUDA (200 mg intravenously [IV] every three weeks for up to 24 months); or
- LENVIMA (18 mg orally once daily) in combination with everolimus (5 mg orally once daily); or
- Sunitinib (50 mg orally once daily for four weeks on treatment, followed by two weeks off treatment).
Treatment continued until unacceptable toxicity or disease progression. Administration of KEYTRUDA plus LENVIMA was permitted beyond Response Evaluation Criteria in Solid Tumors (RECIST)-defined disease progression if the patient was clinically stable and considered by the investigator to be deriving clinical benefit. KEYTRUDA was continued for a maximum of 24 months; however, treatment with LENVIMA could be continued beyond 24 months. Assessment of tumor status was performed at baseline and then every eight weeks.
The study population characteristics were: median age of 62 years (range: 29 to 88 years), 42% age 65 or older; 75% male; 74% White, 21% Asian, 1% Black, and 2% other races; 18% and 82% of patients had a baseline Karnofsky Performance Status (KPS) of 70 to 80 and 90 to 100, respectively; patient distribution by MSKCC risk categories was 27% favorable, 64% intermediate, and 9% poor. Common sites of metastases in patients were lung (68%), lymph node (45%), and bone (25%).
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 1,500 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.
Selected KEYTRUDA® (pembrolizumab) Indications in the U.S.
Melanoma
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.
KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.
Non-Small Cell Lung Cancer
KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) ≥1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is
- stage III where patients are not candidates for surgical resection or definitive chemoradiation, or
- metastatic.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS ≥1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.
Head and Neck Squamous Cell Cancer
KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS) ≥1)] as determined by an FDA-approved test.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.
Classical Hodgkin Lymphoma
KEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).
KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.
Primary Mediastinal Large B-Cell Lymphoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.
Urothelial Carcinoma
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 (CPS ≥10), as determined by an FDA-approved test, or in patients who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
KEYTRUDA is indicated for the treatment of patients with locally advanced or mUC who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.
Microsatellite Instability-High or Mismatch Repair Deficient Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options.
This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.
Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).
Gastric Cancer
KEYTRUDA, in combination with trastuzumab, fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of patients with locally advanced unresectable or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Esophageal Cancer
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic esophageal or GEJ (tumors with epicenter 1 to 5 centimeters above the GEJ) carcinoma that is not amenable to surgical resection or definitive chemoradiation either:
- in combination with platinum- and fluoropyrimidine-based chemotherapy, or
- as a single agent after one or more prior lines of systemic therapy for patients with tumors of squamous cell histology that express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
Cervical Cancer
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Hepatocellular Carcinoma
KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Merkel Cell Carcinoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Renal Cell Carcinoma
KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of adult patients with advanced renal cell carcinoma (RCC).
KEYTRUDA, in combination with LENVIMA, is indicated for the first-line treatment of adult patients with advanced RCC.
Endometrial Carcinoma
KEYTRUDA, in combination with LENVIMA, is indicated for the treatment of patients with advanced endometrial carcinoma that is not MSI-H or dMMR, who have disease progression following prior systemic therapy in any settings and are not candidates for curative surgery or radiation.
Tumor Mutational Burden-High Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [≥10 mutations/megabase] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.
Cutaneous Squamous Cell Carcinoma
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) or locally advanced cSCC that is not curable by surgery or radiation.
Triple-Negative Breast Cancer
KEYTRUDA is indicated for the treatment of patients with high-risk early-stage TNBC in combination with chemotherapy as neoadjuvant treatment, and then continued as a single agent as adjuvant treatment after surgery.
KEYTRUDA, in combination with chemotherapy, is indicated for the treatment of patients with locally recurrent unresectable or metastatic TNBC whose tumors express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
Please see Prescribing Information for KEYTRUDA (pembrolizumab) at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_pi.pdf and Medication Guide for KEYTRUDA at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_mg.pdf.
About LENVIMA® (lenvatinib); available as 10 mg and 4 mg capsules
LENVIMA® (lenvatinib) is a kinase inhibitor that is indicated:
- For the treatment of patients with locally recurrent or metastatic, progressive, radioactive iodine-refractory differentiated thyroid cancer (DTC)
- In combination with KEYTRUDA, for the first-line treatment of adult patients with advanced renal cell carcinoma (RCC)
- In combination with everolimus, for the treatment of adult patients with advanced renal cell carcinoma (RCC) following one prior anti-angiogenic therapy
- For the first-line treatment of patients with unresectable hepatocellular carcinoma (HCC)
- In combination with KEYTRUDA, for the treatment of patients with advanced endometrial carcinoma (EC) that is not microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR), who have disease progression following prior systemic therapy in any setting and are not candidates for curative surgery or radiation.
LENVIMA, discovered and developed by Eisai, is a kinase inhibitor that inhibits the kinase activities of vascular endothelial growth factor (VEGF) receptors VEGFR1 (FLT1), VEGFR2 (KDR), and VEGFR3 (FLT4). LENVIMA inhibits other kinases that have been implicated in pathogenic angiogenesis, tumor growth, and cancer progression in addition to their normal cellular functions, including fibroblast growth factor (FGF) receptors FGFR1-4, the platelet derived growth factor receptor alpha (PDGFRα), KIT, and RET. The combination of LENVIMA and everolimus showed increased anti-angiogenic and anti-tumor activity as demonstrated by decreased human endothelial cell proliferation, tube formation, and VEGF signaling in vitro and tumor volume in mouse xenograft models of human renal cell cancer greater than each drug alone. In syngeneic mouse tumor models, lenvatinib decreased tumor-associated macrophages, increased activated cytotoxic T cells, and demonstrated greater antitumor activity in combination with an anti-PD-1 monoclonal antibody compared to either treatment alone.
For more information, visit www.merck.com
For more information about Eisai, please visit www.eisai.com (for global headquarters: Eisai Co., Ltd.), us.eisai.com (for U.S. headquarters: Eisai, Inc.) or www.eisai.eu (for Europe, Middle East, Africa, Russia, Australia, and New Zealand headquarters: Eisai Europe Ltd.), and connect with us on Twitter (U.S. and global) and LinkedIn (for U.S. and EMEA).
View source version on businesswire.com: https://www.businesswire.com/news/home/20210811005902/en/
Source: Merck & Co., Inc.
FDA Approves LENVIMA® (lenvatinib) Plus KEYTRUDA® (pembrolizumab) Combination for First-Line Treatment of Adult Patients With Advanced Renal Cell Carcinoma (RCC)LENVIMA Plus KEYTRUDA Is Now Approved for Two Types of Cancer, Including Advanced RCC
Based on Phase 3 CLEAR/KEYNOTE-581 Trial, LENVIMA Plus KEYTRUDA Significantly Reduced Risk of Disease Progression or Death by 61% Versus Sunitinib
- August 12, 2021
TOKYO and KENILWORTH, N.J., Aug. 12, 2021 – Eisai (Headquarters: Tokyo, CEO: Haruo Naito) and Merck & Co., Inc., Kenilworth, N.J., U.S.A. (known as MSD outside the United States and Canada)
https://www.eisai.com/news/2021/news202169.html
FDA approves Merck's Keytruda/Lenvima combo for renal cell carcinoma
Aug. 12, 2021 2:12 AM ET Merck & Co., Inc. (MRK) By: Mamta Mayani, SA News Editor2 Comments
- Merck (NYSE:MRK) and Eisai announce that the FDA has approved the combination of Keytruda plus Lenvima, the orally available multiple receptor tyrosine kinase inhibitor discovered by Eisai, for the first-line treatment of adult patients with advanced renal cell carcinoma (RCC).
- https://www.merck.com/news/fda-approves-keytruda-pembrolizumab-plus-lenvima-lenvatinib-combination-for-first-line-treatment-of-adult-patients-with-advanced-renal-cell-carcinoma-rcc/
- https://seekingalpha.com/symbol/MRK
- https://www.eisai.com/index.html
QARZIBA® (Dinutuximab Beta)
BeiGene and EUSA Pharma Announce China NMPA Approval of QARZIBA® (Dinutuximab Beta) for Patients with High-Risk Neuroblastoma
Aug 17, 2021 7:00 AM
CAMBRIDGE, Mass. & BEIJING & HEMEL HEMPSTEAD, England--(BUSINESS WIRE)-- BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160) and EUSA Pharma (UK), Ltd. today announced that the China National Medical Products Administration (NMPA) has granted QARZIBA® (dinutuximab beta) conditional approval for the treatment of high-risk neuroblastoma in patients aged 12 months and above who have previously received induction chemotherapy and achieved at least a partial response, followed by myeloablative therapy and stem cell transplantation, as well as patients with a history of relapsed or refractory (R/R) neuroblastoma with or without residual disease. Dinutuximab beta is a targeted immunotherapy approved by the European Medicines Agency (EMA).i
“Dinutuximab beta represents an important biologic therapy for pediatric patients in China, having been listed in the first batch of New Drugs in Urgent Clinical Need Marketed Overseas by the NMPA,” commented Xiaobin Wu, Ph.D., President, Chief Operating Officer, and General Manager of China at BeiGene. “For these young patients fighting neuroblastoma in China, we are proud to bring the first approved treatment.”
“We are delighted that the benefit of dinutuximab beta has been recognized in China. This approval represents an important milestone in our mission and collaboration with BeiGene of bringing innovative cancer and rare disease therapies to patients,” said Carsten Thiel, Ph.D., Chief Executive Officer of EUSA Pharma.
The approval of dinutuximab beta in China for the treatment of patients with high-risk neuroblastoma was supported by clinical results available from key trials conducted by SIOPEN (The International Society of Paediatric Oncology Europe Neuroblastoma Group) in collaboration with APEIRON Biologics and EUSA Pharma. These randomized controlled trials evaluated the efficacy of dinutuximab beta by comparing the administration of dinutuximab beta with and without interleukin-2 (IL-2) in the first-line treatment of patients with high-risk neuroblastoma and in two single-arm studies in the R/R setting. In the SIOPEN trial (HR-NBL1), the five-year event-free survival (EFS) rate in patients treated with dinutuximab beta was 57% vs. 42% of historical controls (p<0.01) and the five-year overall survival (OS) rate was 64% vs. 50% (p≤0.0001).ii The safety of dinutuximab beta has been evaluated in 514 patients. The most common adverse reactions were pyrexia and pain that occurred despite analgesic treatment. Other frequent adverse reactions were hypersensitivity, vomiting, diarrhea, capillary leak syndrome, and hypotension.
About QARZIBA® (dinutuximab beta)
QARZIBA® is a monoclonal antibody that is specifically directed against the carbohydrate moiety of disialoganglioside 2 (GD2), which is overexpressed on neuroblastoma cells. Dinutuximab beta was approved by the European Commission in 2017 (See EMA Summary of Product Characteristics (SmPC)) and is indicated for the treatment of high-risk neuroblastoma in patients aged 12 months and above, who have previously received induction chemotherapy and achieved at least a partial response, followed by myeloablative therapy and stem cell transplantation, as well as patients with a history of relapsed or refractory neuroblastoma, with or without residual disease. Prior to the treatment of relapsed neuroblastoma, any actively progressing disease should be stabilized by other suitable measures.
For more information please visit www.eusapharma.com.
To learn more about BeiGene, please visit www.beigene.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20210817005501/en/
Source: BeiGene, Ltd.
BeiGene's Qarziba OK'd in China for patients with high-risk neuroblastoma
Aug. 17, 2021 7:31 AM ET BeiGene, Ltd. (BGNE)
By: Mamta Mayani, SA News Editor
- BeiGene (NASDAQ:BGNE) and EUSA Pharma (UK) announce that the China National Medical Products Administration (NMPA) has granted QARZIBA (dinutuximab beta) conditional approval for the treatment of high-risk neuroblastoma in patients aged 12 months and above.
- Dinutuximab beta is a targeted immunotherapy approved by the EMA.
- https://seekingalpha.com/news/3731303-beigenes-qarziba-okd-in-china-for-phttps://seekingalpha.com/symbol/BGNEatients-with-high-risk-neuroblastoma
- https://eusapharma.com/research-areas
Lyumjev® Rapid-Acting Insulin for Use with Omnipod® Products
Aug 16, 2021
Insulet Announces FDA Clearance of Lyumjev® Rapid-Acting Insulin for Use with Omnipod® Products in the United States
ACTON, Mass.--(BUSINESS WIRE)--Aug. 16, 2021-- Insulet Corporation (NASDAQ: PODD) (Insulet or the Company), the leader in tubeless insulin pump technology with its Omnipod® brand of products, today announced FDA clearance for use of Eli Lilly and Company’s (NYSE: LLY) Lyumjev® (insulin lispro-aabc injection) 100 units/mL with Insulet’s Omnipod® Insulin Management System and Omnipod DASH® Insulin Management System. Insulet is the only insulin pump manufacturer in the United States to obtain this indication, providing more flexibility for people with diabetes.
Lyumjev, approved by the FDA in June 2020, is a formulation of insulin lispro developed by Eli Lilly and Company to speed the absorption of insulin into the blood stream. As a rapid-acting insulin, Lyumjev helps control glucose levels after meals in adults with diabetes, so it more closely matches a body’s typical insulin response to a meal.
The FDA has approved an expanded label for the rapid-acting insulin to include administration through continuous subcutaneous insulin infusion (CSII) with an insulin pump, providing more options for people with diabetes to manage their glucose levels and reduce post-meal elevations. Omnipod is the only insulin pump cleared for use with Lyumjev in the United States.
“Rapid-acting insulin is a popular option for people who want added flexibility in their meal-time diabetes regimen,” said Dr. Trang Ly MBBS, FRACP, PhD, Insulet Senior Vice President and Medical Director. “We are thrilled to provide additional choice to the diabetes community, making their lives with diabetes even simpler.”
Insulet collaborated with Lilly to add Lyumjev to the label of the Omnipod Insulin Management System and the Omnipod DASH Insulin Management System.
Please see Lyumjev Full Prescribing Information including Patient Prescribing Information. For additional questions, please call the Lilly Answer Center at 1-800-545-5979.
For more information, please visit insulet.com and omnipod.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210816005133/en/
Source: Insulet Corporation
FDA clears Insulet's Omnipod insulin management systems for use with Eli Lilly's Lyumjev
Aug. 16, 2021 7:39 AM ET Eli Lilly and Company (LLY), PODD By: Aakash Babu, SA News Editor
- The U.S. FDA has cleared the use of Insulet's (NASDAQ:PODD) Omnipod and Omnipod Dash insulin management systems with Eli Lilly's Lyumjev (insulin lispro-aabc injection) 100 units/mL in certain patients with type 1 and type 2 diabetes.
- https://seekingalpha.com/symbol/PODD
- https://seekingalpha.com/symbol/LLY
- https://www.lyumjev.com/
- https://www.insulet.com/
- https://www.omnipod.com/
KEYTRUDA® (pembrolizumab)
FDA Accepts Application for Merck’s KEYTRUDA® (pembrolizumab) as Single Agent for Certain Patients With MSI-H/dMMR Advanced Endometrial Carcinoma
August 10, 2021 6:45 am ET
Data Supporting Application to Be Presented at ESMO Congress 2021
KENILWORTH, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review a new supplemental Biologics License Application (sBLA) seeking approval for KEYTRUDA, Merck’s anti-PD-1 therapy, as a single agent for the treatment of patients with advanced endometrial carcinoma that is microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR), who have disease progression following prior systemic therapy in any setting and are not candidates for curative surgery or radiation. The application is based on overall response data from Cohorts D and K of the KEYNOTE-158 trial, which will be presented for the first time at the European Society for Medical Oncology (ESMO) Congress 2021 (Abstract #795P). The FDA has set a Prescription Drug User Fee Act (PDUFA), or target action, date of March 28, 2022.
“The FDA’s acceptance of our application adds to our momentum of advancing new treatment options to address the most challenging cancers facing women,” said Dr. Scot Ebbinghaus, vice president, clinical research, Merck Research Laboratories. “KEYTRUDA monotherapy is already playing a role for the treatment of certain patients with advanced endometrial carcinoma through the tumor-agnostic MSI-H indication which received accelerated approval four years ago. We look forward to sharing the latest results from KEYNOTE-158, including updated data for KEYTRUDA in certain types of MSI-H/dMMR advanced endometrial carcinoma, at the ESMO Congress in September.”
KEYTRUDA is approved for the treatment of adult and pediatric patients with unresectable or metastatic MSI-H or dMMR solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established. This accelerated approval was based on efficacy data from patients enrolled in one of five open-label, multi-cohort trials, including KEYNOTE-158.
KEYTRUDA was also granted accelerated approval in September 2019, and received full approval in July 2021, in combination with LENVIMA®(lenvatinib) for the treatment of patients with advanced endometrial carcinoma that is not MSI-H or dMMR, who have disease progression following prior systemic therapy in any setting and are not candidates for curative surgery or radiation.
Merck is rapidly advancing a broad portfolio in gynecologic and breast cancers with an extensive clinical development program for KEYTRUDA and several other investigational and approved medicines across these areas.
About KEYNOTE-158
KEYNOTE-158 (ClinicalTrials.gov, NCT02628067) is an ongoing global, open-label, non-randomized, multi-cohort, multi-center, Phase 2 study evaluating KEYTRUDA in patients with multiple types of advanced solid tumors – including endometrial carcinoma – that have progressed on standard of care therapy. Cohort K enrolled 79 patients with MSI-H endometrial carcinoma and Cohort D enrolled 11 patients with MSI-H endometrial carcinoma for a total of 90 pooled patients who received KEYTRUDA as monotherapy (200 mg fixed dose every three weeks). The primary endpoint was overall response rate as evaluated by independent central review using RECIST v1.1. Secondary endpoints are progression-free survival, overall survival, duration of response, and safety.
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 1,500 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.
Selected KEYTRUDA® (pembrolizumab) Indications in the U.S.
Melanoma
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.
KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.
Non-Small Cell Lung Cancer
KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) ≥1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is:
- stage III where patients are not candidates for surgical resection or definitive chemoradiation, or
- metastatic.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS ≥1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.
Head and Neck Squamous Cell Cancer
KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS ≥1)] as determined by an FDA-approved test.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.
Classical Hodgkin Lymphoma
KEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).
KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.
Primary Mediastinal Large B-Cell Lymphoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.
Urothelial Carcinoma
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 (CPS ≥10), as determined by an FDA-approved test, or in patients who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
KEYTRUDA is indicated for the treatment of patients with locally advanced or mUC who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.
Microsatellite Instability-High or Mismatch Repair Deficient Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options.
This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.
Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).
Gastric Cancer
KEYTRUDA, in combination with trastuzumab, fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of patients with locally advanced unresectable or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Esophageal Cancer
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic esophageal or GEJ (tumors with epicenter 1 to 5 centimeters above the GEJ) carcinoma that is not amenable to surgical resection or definitive chemoradiation either:
- in combination with platinum- and fluoropyrimidine-based chemotherapy, or
- as a single agent after one or more prior lines of systemic therapy for patients with tumors of squamous cell histology that express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
Cervical Cancer
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Hepatocellular Carcinoma
KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Merkel Cell Carcinoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Renal Cell Carcinoma
KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma.
Endometrial Carcinoma
KEYTRUDA, in combination with LENVIMA, is indicated for the treatment of patients with advanced endometrial carcinoma that is not MSI-H or dMMR, who have disease progression following prior systemic therapy in any setting and are not candidates for curative surgery or radiation.
Tumor Mutational Burden-High Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [≥10 mutations/megabase] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.
Cutaneous Squamous Cell Carcinoma
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) or locally advanced cSCC that is not curable by surgery or radiation.
Triple-Negative Breast Cancer
KEYTRUDA is indicated for the treatment of patients with high-risk early-stage triple-negative breast cancer (TNBC) in combination with chemotherapy as neoadjuvant treatment, and then continued as a single agent as adjuvant treatment after surgery.
KEYTRUDA, in combination with chemotherapy, is indicated for the treatment of patients with locally recurrent unresectable or metastatic TNBC whose tumors express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
Please see Prescribing Information for KEYTRUDA (pembrolizumab) at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_pi.pdf and Medication Guide for KEYTRUDA at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_mg.pdf.
About LENVIMA® (lenvatinib) Capsules
LENVIMA® (lenvatinib) is a kinase inhibitor that is indicated:
- For the treatment of patients with locally recurrent or metastatic, progressive, radioactive iodine-refractory differentiated thyroid cancer (DTC)
- In combination with everolimus for the treatment of patients with advanced renal cell carcinoma (RCC) following one prior anti-angiogenic therapy
- For the first-line treatment of patients with unresectable hepatocellular carcinoma (HCC)
- In combination with KEYTRUDA, for the treatment of patients with advanced endometrial carcinoma that is not microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR), who have disease progression following prior systemic therapy in any setting and are not candidates for curative surgery or radiation.
LENVIMA, discovered and developed by Eisai, is a kinase inhibitor that inhibits the kinase activities of vascular endothelial growth factor (VEGF) receptors VEGFR1 (FLT1), VEGFR2 (KDR), and VEGFR3 (FLT4). LENVIMA inhibits other kinases that have been implicated in pathogenic angiogenesis, tumor growth, and cancer progression in addition to their normal cellular functions, including fibroblast growth factor (FGF) receptors FGFR1-4, the platelet derived growth factor receptor alpha (PDGFRα), KIT, and RET. In syngeneic mouse tumor models, lenvatinib decreased tumor-associated macrophages, increased activated cytotoxic T cells, and demonstrated greater antitumor activity in combination with an anti-PD-1 monoclonal antibody compared to either treatment alone.
LENVIMA (lenvatinib) is available as 10 mg and 4 mg capsules.
Please see Prescribing Information for LENVIMA (lenvatinib) at http://www.lenvima.com/pdfs/prescribing-information.pdf.
For more information, visit www.merck.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20210810005544/en/
Source: Merck & Co., Inc.
FDA accepts Merck's Keytruda application in endometrial cancer
Aug. 10, 2021 7:03 AM ET Merck & Co., Inc. (MRK) By: Mamta Mayani, SA News Editor1 Comment
- Merck (NYSE:MRK) announces that the FDA has accepted for review a new supplemental Biologics License Application (sBLA) seeking approval for Keytruda for the treatment of patients with advanced endometrial carcinoma that is microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR), who have disease progression following prior systemic therapy in any setting and are not candidates for curative surgery or radiation.
- https://seekingalpha.com/news/3728041-fda-accepts-mercks-keytruda-application-in-endometrial-cancer
- https://seekingalpha.com/symbol/MRK
- https://www.keytruda.com/
- https://www.lenvima.com/
Tyvyt® (Sintilimab Injection) in Combination with Chemotherapy
Innovent Announces Sintilimab in Combination with Chemotherapy Meets the Primary Endpoint of Overall Survival in the Phase 3 ORIENT-16 Study for the First-Line Treatment of Advanced or Metastatic Gastric or Gastroesophageal Junction Adenocarcinoma
Aug. 15, 2021 8:00 PM ET Innovent Biologics, Inc. (IVBIY)
SAN FRANCISCO and SUZHOU, China, Aug. 15, 2021 /PRNewswire/ -- Innovent Biologics, Inc. (IVBIY) ("Innovent") (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high quality medicines for the treatment of oncology, metabolic, autoimmune and other major diseases, today announced that the sintilimab Phase 3 ORIENT-16 study met its predefined primary endpoint of overall survival (OS). ORIENT-16 is a randomized, double-blind, multicenter Phase 3 clinical trial evaluating sintilimab in combination with chemotherapy (oxaliplatin and capecitabine) compared to chemotherapy alone in the first-line treatment of patients with unresectable, locally advanced, recurrent or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma.
In an interim analysis, sintilimab in combination with chemotherapy demonstrated a statistically significant improvement in the primary endpoint of OS compared to placebo plus chemotherapy, in both the intention-to-treat (ITT) group and PD-L1 positive group. The safety profile of sintilimab in this study was consistent with that observed in previously reported studies of sintilimab, and no additional safety signals were identified for the combination of sintilimab and chemotherapy. These results will be presented at an upcoming medical meeting.
About the ORIENT-16 Study
ORIENT-16 is a randomized, double-blind, multicenter Phase 3 clinical study evaluating sintilimab in combination with chemotherapy (oxaliplatin and capecitabine), compared to placebo in combination with chemotherapy, for the first-line treatment of unresectable, locally advanced, recurrent or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma (ClinicalTrials.gov, NCT03745170). The primary endpoint was overall survival, in all randomized and in PD-L1 positive patients.
About Sintilimab
Sintilimab, marketed as TYVYT® (sintilimab injection) in China, is an innovative PD-1 inhibitor with global quality standards jointly developed by Innovent and Eli Lilly and Company. Sintilimab is an 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 for four indications, 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 nonsquamous non-small cell lung cancer
- 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 hepatocellular carcinoma
Additionally, Innovent currently has a regulatory submission under review in China for sintilimab for the second-line treatment of squamous non-small cell lung cancer.
Innovent also has three clinical studies of sintilimab that have met their primary endpoints:
- In combination with cisplatin plus paclitaxel or cisplatin plus 5-fluorouracil for the first-line treatment of esophageal squamous cell carcinoma
- In combination with oxaliplatin and capecitabine for the first-line treatment of unresectable, locally advanced, recurrent or metastatic gastric or gastroesophageal junction adenocarcinoma.
- The second-line treatment of esophageal squamous cell carcinoma
In May 2021, the U.S. FDA accepted for review the Biologics License Application (BLA) for sintilimab in combination with pemetrexed and platinum chemotherapy for the first-line treatment of nonsquamous non-small cell lung cancer.
Sintilimab was included in China's National Reimbursement Drug List (NRDL) in 2019 as the first PD-1 inhibitor and the only PD-1 included in the list in that year.
About Tyvyt® (Sintilimab Injection)
Tyvyt® (sintilimab injection), an innovative drug jointly developed in China by Innovent and Lilly, has been granted marketing approval by the NMPA for relapsed or refractory classic Hodgkin's lymphoma after at least second-line system chemotherapy, and is included in the 2019 Guidelines of Chinese Society of Clinical Oncology for Lymphoid Malignancies. Tyvyt is the only PD-1 inhibitor with global quality that has been included in the new Catalogue of the National Reimbursement Drug List (NRDL) in November 2019.
Tyvyt 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 for sintilimab injection to evaluate its safety and efficacy in a wide variety of cancer indications, including eight registration or pivotal clinical trials.
Tyvyt (sintilimab injection) is not an approved product in the United States. ALIMTA® (pemetrexed for injection) is not approved for use in combination with Tyvyt in the United States.
Sintilimab is not an approved product in the United States.
For more information, please visit http://cn.innoventbio.com/en/#/
SOURCE Innovent Biologics
https://seekingalpha.com/symbol/IVBIY
https://www.hkex.com.hk/Market-Data/Securities-Prices/Equities/Equities-Quote?sym=1801&sc_lang=en
belzutifan
FDA approves belzutifan for cancers associated with von Hippel-Lindau disease
On August 13, 2021, the Food and Drug Administration approved belzutifan (Welireg, Merck), a hypoxia-inducible factor inhibitor for adult patients with von Hippel-Lindau (VHL) disease who require therapy for associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumors (pNET), not requiring immediate surgery.
Belzutifan was investigated in the ongoing Study 004 (NCT03401788), an open-label clinical trial in 61 patients with VHL-associated RCC (VHL-RCC) diagnosed based on a VHL germline alteration and with at least one measurable solid tumor localized to the kidney. Enrolled patients had other VHL-associated tumors, including CNS hemangioblastomas and pNET. Patients received belzutifan 120 mg once daily until disease progression or unacceptable toxicity.
The primary efficacy endpoint was overall response rate (ORR) measured by radiology assessment, as assessed by an independent review committee using RECIST v1.1. Additional efficacy endpoints included duration of response (DoR), and time- to- response (TTR). An ORR of 49% (95% CI:36, 62) was reported in patients with VHL-associated RCC. All patients with VHL-RCC with a response were followed for a minimum of 18 months from the start of treatment. The median DoR was not reached; 56% of responders had DoR ≥ 12 months and a median TTR of 8 months. In patients with other VHL-associated non-RCC tumors, 24 patients with measurable CNS hemangioblastomas had an ORR of 63% and 12 patients with measurable pNET had an ORR of 83%. Median DoR was not reached, with 73% and 50% of patients having response durations ≥ 12 months for CNS hemangioblastomas and pNET, respectively.
The recommended belzutifan dosage is 120 mg administered orally once daily with or without food.
View full prescribing information for Welireg.
This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with the Australian Therapeutic Goods Administration (TGA), Health Canada, and the Medicines and Healthcare products Regulatory Agency (MHRA) of the United Kingdom. The application reviews are ongoing at the other regulatory agencies.
This review used the Real-Time Oncology Review (RTOR) pilot program, which streamlined data submission prior to the filing of the entire clinical application, as well as the Assessment Aid and the Product Quality Assessment Aid, voluntary submissions from the applicant to facilitate the FDA’s assessment. The FDA approved this application approximately 1 month ahead of the FDA goal date.
This application was granted priority review for this indication. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics.
Merck wins FDA approval for belzutifan to treat cancers linked to rare genetic disease
Aug. 13, 2021 12:36 PM ET
By: Dulan Lokuwithana, SA News Editor
- Merck (MRK +0.8%) has received FDA approval for belzutifan as a treatment of certain cancers associated with the rare genetic disorder, von Hippel-Lindau (VHL) disease.
- https://seekingalpha.com/news/3730396-merck-wins-fda-approval-for-belzutifan-to-treat-cancers-linked-to-rare-genetic-disease
- https://seekingalpha.com/symbol/MRK
- https://www.merck.com/?s=belzutifan
KEYTRUDA® (pembrolizumab)
FDA Grants Priority Review to Merck’s Supplemental Biologics License Application for KEYTRUDA® (pembrolizumab) as Adjuvant Therapy in Certain Patients With Renal Cell Carcinoma (RCC) Following Surgery
August 10, 2021 6:42 am ET
Application Based on KEYNOTE-564, a Phase 3 Study Evaluating Adjuvant Immunotherapy in RCC
KENILWORTH, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced that the U.S. Food and Drug Administration (FDA) has accepted and granted priority review for a new supplemental Biologics License Application (sBLA) for KEYTRUDA, Merck’s anti-PD-1 therapy, for the adjuvant treatment of patients with renal cell carcinoma (RCC) at intermediate-high or high risk of recurrence following nephrectomy (surgical removal of a kidney), or following nephrectomy and resection of metastatic lesions. This sBLA is based on data from the pivotal Phase 3 KEYNOTE-564 trial, in which KEYTRUDA demonstrated a statistically significant and clinically meaningful improvement in disease-free survival (DFS) compared to placebo. These data were presented at the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting. The FDA has set a Prescription Drug User Fee Act (PDUFA), or target action, date of December 10, 2021.
“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. Scot Ebbinghaus, vice president, clinical research, Merck Research Laboratories. “We look forward to working with the FDA towards the goal of bringing the first adjuvant immunotherapy option to appropriate patients with renal cell carcinoma in the U.S.”
KEYTRUDA is currently approved in the U.S., Europe and Japan in combination with axitinib for the first-line treatment of patients with advanced RCC. Merck is continuing to study KEYTRUDA, in combination or as monotherapy, as well as other investigational products across multiple settings and stages of RCC, including adjuvant and advanced or metastatic disease. The broad clinical development program exploring KEYTRUDA in RCC includes over 20 clinical studies and more than 4,000 patients worldwide.
About KEYNOTE-564
KEYNOTE-564 is a randomized, double-blind, Phase 3 trial (ClinicalTrials.gov, NCT03142334) evaluating KEYTRUDA monotherapy for the adjuvant treatment of patients with RCC who have undergone nephrectomy and who have intermediate-high risk, high risk, or M1 no evidence of disease (M1 NED) RCC with clear cell component. The study enrolled 994 patients who were randomized to receive either KEYTRUDA (200 mg intravenously [IV] on Day 1 of each three-week cycle for up to 17 cycles) or placebo (saline solution IV on Day 1 of each three-week cycle for up to 17 cycles). The primary endpoint is DFS, and the secondary endpoints include overall survival and safety.
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 1,500 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.
Selected KEYTRUDA® (pembrolizumab) Indications in the U.S.
Melanoma
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.
KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.
Non-Small Cell Lung Cancer
KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) ≥1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is:
- stage III where patients are not candidates for surgical resection or definitive chemoradiation, or
- metastatic.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS ≥1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.
Head and Neck Squamous Cell Cancer
KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS ≥1)] as determined by an FDA-approved test.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.
Classical Hodgkin Lymphoma
KEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).
KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.
Primary Mediastinal Large B-Cell Lymphoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.
Urothelial Carcinoma
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 (CPS ≥10), as determined by an FDA-approved test, or in patients who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
KEYTRUDA is indicated for the treatment of patients with locally advanced or mUC who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.
Microsatellite Instability-High or Mismatch Repair Deficient Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options.
This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.
Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).
Gastric Cancer
KEYTRUDA, in combination with trastuzumab, fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of patients with locally advanced unresectable or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Esophageal Cancer
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic esophageal or GEJ (tumors with epicenter 1 to 5 centimeters above the GEJ) carcinoma that is not amenable to surgical resection or definitive chemoradiation either:
- in combination with platinum- and fluoropyrimidine-based chemotherapy, or
- as a single agent after one or more prior lines of systemic therapy for patients with tumors of squamous cell histology that express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
Cervical Cancer
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Hepatocellular Carcinoma
KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Merkel Cell Carcinoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Renal Cell Carcinoma
KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma.
Tumor Mutational Burden-High Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [≥10 mutations/megabase] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.
Cutaneous Squamous Cell Carcinoma
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) or locally advanced cSCC that is not curable by surgery or radiation.
Triple-Negative Breast Cancer
KEYTRUDA is indicated for the treatment of patients with high-risk early-stage triple-negative breast cancer (TNBC) in combination with chemotherapy as neoadjuvant treatment, and then continued as a single agent as adjuvant treatment after surgery.
KEYTRUDA, in combination with chemotherapy, is indicated for the treatment of patients with locally recurrent unresectable or metastatic TNBC whose tumors express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
For more information about our oncology clinical trials, visit www.merck.com/clinicaltrials.
For more information, visit www.merck.com
Please see Prescribing Information for KEYTRUDA (pembrolizumab) at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_pi.pdf and Medication Guide for KEYTRUDA at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_mg.pdf.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210810005542/en/
Source: Merck & Co., Inc.
FDA grants accelerated review to Merck’s Keytruda application in kidney cancer
Aug. 10, 2021 6:58 AM ET Merck & Co., Inc. (MRK) By: Mamta Mayani, SA News Editor1 Comment
- Under priority review, the FDA has accepted Merck's (NYSE:MRK) new supplemental Biologics License Application (sBLA) for Keytruda, for the adjuvant treatment of patients with renal cell carcinoma (RCC) at intermediate-high or high risk of recurrence following nephrectomy (surgical removal of a kidney), or following nephrectomy and resection of metastatic lesions.
- https://seekingalpha.com/news/3728032-fda-grants-accelerated-review-to-mercks-keytruda-application-in-kidney-cancer
- https://seekingalpha.com/symbol/MRK
- https://www.keytruda.com/
- https://www.merck.com/
Polivy® (polatuzumab vedotin-piiq)
Sunday, Aug 8, 2021
Phase III Study Shows Genentech's Polivy Plus R-CHP is the First Regimen in 20 Years to Significantly Improve Outcomes in Previously Untreated Aggressive Form of Lymphoma Compared to Standard of Care
Pivotal Phase III POLARIX trial comparing Polivy in combination with chemotherapy regimen R-CHP versus the standard of care R-CHOP in treatment of first-line diffuse large B-cell lymphoma (DLBCL) met its primary endpoint of investigator assessed progression-free survival
Prolonging survival without disease advancement could be transformative for newly diagnosed DLBCL patients, as currently 40% of patients relapse after disease progression
Data will be submitted to health authorities globally as soon as possible and presented at an upcoming medical meeting
South San Francisco, CA -- August 8, 2021 --
Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the pivotal Phase III POLARIX trial investigating Polivy ® (polatuzumab vedotin) in combination with Rituxan® (rituximab) plus cyclophosphamide, doxorubicin and prednisone (R-CHP) versus Rituxan plus cyclophosphamide, doxorubicin, vincristine and prednisone (R-CHOP) met its primary endpoint by demonstrating significantly improved and clinically meaningful progression-free survival in people with previously untreated diffuse large B-cell lymphoma (DLBCL). Safety outcomes were consistent with those seen in previous trials.
“Since 40% of people with DLBCL relapse after initial therapy, achieving meaningful treatment effects in the front-line setting has the potential to be transformative,” said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. “This Polivy regimen is the first in two decades to improve progression-free survival in DLBCL compared to the standard of care, and we look forward to sharing these results with health authorities to bring this important potential new treatment option to patients as soon as possible.”
Today’s POLARIX results will be presented at an upcoming medical meeting and submitted to health authorities as part of Genentech’s commitment to transforming the treatment of DLBCL by providing options tailored to patient and healthcare professional needs. Genentech would like to thank all investigators, academic partners and people with DLBCL who participated in the study.
About the POLARIX study
POLARIX [NCT03274492] is an international Phase III, randomized, double-blind, placebo-controlled study evaluating the efficacy, safety and pharmacokinetics of Polivy ® (polatuzumab vedotin) plus Rituxan® (rituximab), cyclophosphamide, doxorubicin and prednisone (R-CHP) versus Rituxan, cyclophosphamide, doxorubicin, vincristine and prednisone (R-CHOP) in people with previously untreated diffuse large B-cell lymphoma (DLBCL). Eight-hundred and seventy-nine patients were randomized 1:1 to receive either Polivy plus R-CHP plus a vincristine placebo for six cycles, followed by Rituxan for two cycles; or R-CHOP plus a Polivy placebo for six cycles, followed by two cycles of Rituxan. The primary outcome measure is progression-free survival as assessed by the investigator using the Lugano Response Criteria for malignant lymphoma. POLARIX is being conducted in collaboration with The Lymphoma Study Association (LYSA) and The Lymphoma Academic Research Organisation (LYSARC).
About Polivy® (polatuzumab vedotin-piiq)
Polivy is a first-in-class anti-CD79b antibody-drug conjugate (ADC). The CD79b protein is expressed specifically in the majority of B cells, an immune cell impacted in some types of non-Hodgkin’s lymphoma (NHL), making it a promising target for the development of new therapies. Polivy binds to CD79b and destroys these B cells through the delivery of an anti-cancer agent, which is thought to minimize the effects on normal cells. Polivy is being developed by Genentech using Seagen ADC technology and is currently being investigated for the treatment of several types of NHL.
Polivy U.S. Indication
Polivy is a prescription medicine used with other medicines, bendamustine and a rituximab product, to treat diffuse large B-cell lymphoma in adults who have progressed after at least two prior therapies.
The accelerated approval of Polivy is based on a type of response rate. There are ongoing studies to confirm the clinical benefit of Polivy.
Please visit http://www.Polivy.com for the full Prescribing Information for additional Important Safety Information.
For more information visit http://www.gene.com/hematology.
For additional information about the company, please visit http://www.gene.com.
Genentech's Polivy meets primary endpoint as first-line treatment for B-cell lymphoma
Aug. 09, 2021 7:54 AM ET Roche Holding AG (RHHBY) By: Jonathan M Block, SA News Editor
- Genetech's (OTCQX:RHHBY) Polivy (polatuzumab vedotin) met its primary endpoint -- progression-free survival -- in a pivotal trial comparing it to standard of care for people with previously untreated diffuse large B-cell lymphoma ("DLBCL").
- https://seekingalpha.com/news/3727389-genentech-roche-polivy-meets-primary-endpoint-as-first-line-treatment-for-b-cell-lymphoma
- https://seekingalpha.com/symbol/RHHBY
- https://www.gene.com/
SKYRIZI® (risankizumab-rzaa)
August 9, 2021
SKYRIZI® (risankizumab-rzaa) Now Available in the U.S. as a Single 150 mg Injection for Adults with Moderate to Severe Plaque Psoriasis
- SKYRIZI is now the only four-dose-a-year biologic for psoriasis with options to administer via a single-dose pen or prefilled syringe
NORTH CHICAGO, Ill., Aug. 9, 2021 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced that SKYRIZI® (risankizumab-rzaa), an interleukin-23 (IL-23) inhibitor, is now available in the U.S. as a single-dose 150 mg injection for the treatment of adults with moderate to severe plaque psoriasis. Previously two 75 mg injections per dose, SKYRIZI 150 mg is now administered with one injection per dose – via either a prefilled pen or syringe – every 12 weeks following two starter doses.1
The new SKYRIZI 150 mg pen incorporates wide grip handles, audible cues to help guide the administration process and an indicator to signal when administration is complete.
"We know many people living with psoriasis are looking for flexibility in how they manage their disease," said Patrick Horber, President, U.S. Immunology, AbbVie. "That's why we're proud to provide an updated SKYRIZI treatment experience that allows for fewer injections of the same SKYRIZI that patients and providers have come to know and trust."
The U.S. Food and Drug Administration (FDA) approved SKYRIZI 150 mg in April based on data from three clinical trials showing the single-dose SKYRIZI 150 mg injection was bioequivalent, working the same as two injections of SKYRIZI 75 mg per dose with a consistent efficacy and safety profile.
"Helping my psoriasis patients find an ideal treatment option for them requires not only assessment of the plaques on their skin, but consideration of a treatment experience that fits their lifestyle," said Lindsay Ackerman, M.D., board-certified dermatologist and founder of Medical Dermatology Specialists in Phoenix, AZ. "The single-dose injection options for SKYRIZI 150 mg allow for a maintenance treatment plan of only four injections per year and are a welcome addition to the psoriasis treatment mix."
SKYRIZI is part of a collaboration with Boehringer Ingelheim, with AbbVie leading development and commercialization of SKYRIZI globally.
About SKYRIZI® (risankizumab-rzaa) in the United States1
SKYRIZI is indicated for the treatment of moderate to severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy.
For more information about AbbVie, please visit us at www.abbvie.com.
AbbVie's Skyrizi now available in U.S. as a single 150 mg injection for plaque psoriasis
Aug. 09, 2021 9:01 AM ETAbbVie Inc. (ABBV)By: Mamta Mayani, SA News Editor
- AbbVie (NYSE:ABBV) announces that SKYRIZI (risankizumab-rzaa), an interleukin-23 (IL-23) inhibitor, is now available in the U.S. as a single-dose 150 mg injection for the treatment of adults with moderate to severe plaque psoriasis.
- https://seekingalpha.com/news/3727469-abbvies-skyrizi-now-available-in-us-as-a-single-150-mg-injection-for-plaque-psoriasis
- https://seekingalpha.com/symbol/ABBV
- https://www.skyrizi.com/
- https://www.abbvie.com/
biotecMAX™ April/May/June/July 2021 Insight
LIBTAYO® (CEMIPLIMAB-RWLC) COMBINED WITH CHEMOTHERAPY
August 5, 2021 at 6:00 AM EDT Back
PHASE 3 TRIAL OF LIBTAYO® (CEMIPLIMAB-RWLC) COMBINED WITH CHEMOTHERAPY STOPPED EARLY DUE TO SIGNIFICANT IMPROVEMENT IN OVERALL SURVIVAL IN PATIENTS WITH FIRST-LINE ADVANCED NON-SMALL CELL LUNG CANCER
TARRYTOWN, N.Y. and PARIS, Aug. 5, 2021 /PRNewswire/ --
Libtayo combined with chemotherapy increased median overall survival from 13 to 22 months, leading to a 29% reduction in the risk of death
Trial enrolled patients with locally advanced and metastatic disease with squamous or non-squamous histology, across all PD-L1 expression levels
Libtayo has now demonstrated improved overall survival as a monotherapy or in combination with chemotherapy in first-line advanced non-small cell lung cancer
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today announced the Phase 3 trial of PD-1 inhibitor Libtayo in combination with platinum-doublet chemotherapy was stopped early after meeting its overall survival (OS) primary endpoint in patients with advanced non-small cell lung cancer (NSCLC). Adding Libtayo to chemotherapy significantly improved OS, compared to chemotherapy alone, in the trial that enrolled patients with metastatic or locally advanced disease and tumors with either squamous or non-squamous histology and across all PD-L1 expression levels. These data are planned to form the basis of regulatory submissions in the U.S. and European Union.
"Libtayo in combination with chemotherapy increased median overall survival to 22 months in patients with advanced non-small cell lung cancer, compared to 13 months with chemotherapy alone," said Miranda Gogishvili, M.D., an oncologist at the High Technology Medical Center, University Clinic, in Tbilisi, Georgia and a trial investigator. "Notably, the Phase 3 trial enrolled patients with a variety of challenging-to-treat disease characteristics, as well as those with locally advanced disease. These data add to the growing body of evidence supporting Libtayo in advanced non-small cell lung cancer, which also include the pivotal results for Libtayo monotherapy in cases of high PD-L1 expression."
The decision to stop the trial early was based on a recommendation by the Independent Data Monitoring Committee (IDMC) during a protocol-specified interim analysis. In this top-line initial analysis of 466 patients, combining Libtayo with chemotherapy reduced the risk of death by 29% compared to chemotherapy alone (hazard ratio: 0.71; 95% confidence interval [CI]: 0.53-0.93; p=0.014). Median OS was 22 months (95% CI: 16 months to not evaluable) for Libtayo and chemotherapy, and 13 months (95% CI: 12 to 16 months) for chemotherapy alone. No new Libtayo safety signals were identified in the IDMC analysis, and additional detailed efficacy and safety data will be presented at an upcoming medical meeting.
About the Phase 3 Trial
The randomized, multicenter Phase 3 trial, called EMPOWER-Lung 3, investigated a first-line combination treatment of Libtayo and platinum-doublet chemotherapy, compared to platinum-doublet chemotherapy alone, in squamous or non-squamous advanced NSCLC irrespective of PD-L1 expression. Specifically, the trial included 466 patients who tested negative for ALK, EGFR and ROS1 mutations and had either previously untreated metastatic NSCLC (stage IV) or locally advanced NSCLC (stage IIIB/C) and were not candidates for definitive chemoradiation.
Patients were randomized 2:1 to receive either Libtayo 350 mg (n=312) or placebo (n=154) administered intravenously every three weeks for 108 weeks, plus platinum-doublet chemotherapy administered every three weeks for four cycles. The co-primary endpoints were OS and progression-free survival, and key secondary endpoints included objective response rate and best overall response.
Among trial patients, 30% (n=139) had tumors with <1% PD-L1 expression, 38% (n=175) had tumors with 1% to 49% PD-L1 expression, and 33% (n=152) had tumors with ≥50% PD-L1 expression.
About Libtayo
Libtayo is a fully human monoclonal antibody targeting the immune checkpoint receptor PD-1 on T-cells. By binding to PD-1, Libtayo has been shown to block cancer cells from using the PD-1 pathway to suppress T-cell activation.
The generic name for Libtayo in its approved U.S. indications is cemiplimab-rwlc, with rwlc as the suffix designated in accordance with Nonproprietary Naming of Biological Products Guidance for Industry issued by the U.S. FDA. Libtayo is being jointly developed by Regeneron and Sanofi under a global collaboration agreement.
The extensive clinical program for Libtayo is focused on difficult-to-treat cancers. Libtayo is currently being investigated in advanced cervical cancer, as well as in trials combining Libtayo with either conventional or novel therapeutic approaches for other solid tumors and blood cancers. These potential uses are investigational, and their safety and efficacy have not been evaluated by any regulatory authority.
What is Libtayo?
Libtayo is a prescription medicine used to treat people with a type of skin cancer called cutaneous squamous cell carcinoma (CSCC) that has spread or cannot be cured by surgery or radiation.
Libtayo is a prescription medicine used to treat people with a type of skin cancer called basal cell carcinoma that cannot be removed by surgery (locally advanced BCC) and have received treatment with an HHI, or cannot receive treatment with a HHI.
Libtayo is a prescription medicine used to treat people with a type of skin cancer called basal cell carcinoma that has spread (metastatic BCC) and have received treatment with a hedgehog pathway inhibitor (HHI), or cannot receive treatment with a HHI. This use is approved based on how many patients responded to treatment and how long they responded. Studies are ongoing to provide additional information about clinical benefit.
Libtayo is a prescription medicine used to treat people with a type of lung cancer called non-small cell lung cancer (NSCLC). Libtayo may be used as your first treatment when your lung cancer has not spread outside your chest (locally advanced lung cancer) and you cannot have surgery or chemotherapy with radiation, or your lung cancer has spread to other areas of your body (metastatic lung cancer), and your tumor tests positive for high "PD-L1" and your tumor does not have an abnormal "EGFR"," ALK "or "ROS1" gene.
It is not known if Libtayo is safe and effective in children.
Please see accompanying full Prescribing Information, including Medication Guide.
For additional information about the company, please visit www.regeneron.com
SOURCE Regeneron Pharmaceuticals, Inc.
https://seekingalpha.com/symbol/REGN
CABOMETYX® (cabozantinib)
Exelixis Announces U.S. FDA Accepts for Priority Review the Supplemental New Drug Application for CABOMETYX® (cabozantinib) for Patients with Previously Treated Radioactive Iodine-Refractory Differentiated Thyroid CancerPDF Version
– U.S. Food and Drug Administration assigned a Prescription Drug User Fee Act action date of December 4, 2021 –
– The supplemental New Drug Application is based on the phase 3 COSMIC-311 pivotal trial, which demonstrated significant improvement in progression-free survival with CABOMETYX versus placebo –
ALAMEDA, Calif.--(BUSINESS WIRE)--Aug. 5, 2021-- Exelixis, Inc. (NASDAQ: EXEL) today announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s supplemental New Drug Application (sNDA) for CABOMETYX® (cabozantinib) as a treatment for patients 12 years and older with differentiated thyroid cancer (DTC) who have progressed following prior therapy and are radioactive iodine-refractory (if radioactive iodine is appropriate). The FDA granted Priority Review designation and assigned a Prescription Drug User Fee Act (PDUFA) target action date of December 4, 2021.
“The FDA’s acceptance of our sNDA with Priority Review is an important step toward our goal of bringing CABOMETYX to patients with previously treated radioactive iodine-refractory differentiated thyroid cancer,” said Michael M. Morrissey, Ph.D., Exelixis’ President and Chief Executive Officer. “Considering the lack of a standard of care in the treatment of this cancer following anti-VEGFR therapy, the progression-free survival benefit demonstrated in the phase 3 COSMIC-311 pivotal trial means CABOMETYX, if approved, could become an important new treatment for these patients.”
The sNDA is based on the results of COSMIC-311, a phase 3 pivotal trial evaluating CABOMETYX versus placebo in patients with radioactive iodine-refractory DTC who progressed after up to two prior vascular endothelial growth factor receptor (VEGFR)-targeted therapies. At a planned interim analysis, CABOMETYX met one of the trial’s primary endpoints, demonstrating a significant improvement in progression-free survival versus placebo. In February 2021, the FDA granted Breakthrough Therapy Designation to CABOMETYX as a potential treatment for patients with DTC that has progressed following prior therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate) based on these results. Detailed study findings were presented at the 2021 American Society of Clinical Oncology Annual Meeting and were published by The Lancet Oncology in July 2021.
About COSMIC-311
COSMIC-311 is a multicenter, randomized, double-blind, placebo-controlled phase 3 pivotal trial that aimed to enroll approximately 300 patients at 150 sites globally. Patients were randomized in a 2:1 ratio to receive either cabozantinib 60 mg or placebo once daily. The primary endpoints are progression-free survival and objective response rate. More information about this trial is available at ClinicalTrials.gov.
About CABOMETYX® (cabozantinib)
In the U.S., CABOMETYX tablets are approved for the treatment of patients with advanced RCC; for the treatment of patients with HCC who have been previously treated with sorafenib; and for patients with advanced RCC as a first-line treatment in combination with nivolumab. CABOMETYX tablets have also received regulatory approvals in the European Union and additional countries and regions worldwide. In 2016, Exelixis granted Ipsen exclusive rights for the commercialization and further clinical development of cabozantinib outside of the United States and Japan. In 2017, Exelixis granted exclusive rights to Takeda Pharmaceutical Company Limited for the commercialization and further clinical development of cabozantinib for all future indications in Japan. Exelixis holds the exclusive rights to develop and commercialize cabozantinib in the United States.
CABOMETYX is not indicated for radioactive iodine-refractory differentiated thyroid cancer.
Please see accompanying full Prescribing Information https://www.cabometyx.com/downloads/CABOMETYXUSPI.pdf.
For more information about Exelixis, please visit www.exelixis.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20210805005256/en/
Source: Exelixis, Inc.
FDA grants priority review to Exelixis' Cabometyx in thyroid cancer
Aug. 05, 2021 8:44 AM ET Exelixis, Inc. (EXEL)Exelixis, Inc. (EXEL) By: Mamta Mayani, SA News Editor
- The FDA has accepted Exelixis' (NASDAQ:EXEL) supplemental New Drug Application (sNDA) for CABOMETYX (cabozantinib) as a treatment for patients 12 years and older with differentiated thyroid cancer (DTC) who have progressed following prior therapy and are radioactive iodine-refractory.
- https://seekingalpha.com/news/3726092-fda-grants-priority-review-to-exelixis-cabometyx-in-thyroid-cancer
- https://seekingalpha.com/symbol/EXEL
- https://www.cabometyx.com/
- https://www.exelixis.com/
Olumiant® baricitinib
Lilly and Incyte's baricitinib reduced deaths among patients with COVID-19 receiving invasive mechanical ventilation
August 3, 2021Download PDF- New data from Phase 3 COV-BARRIER sub-study indicates one death prevented for every six baricitinib-treated patients on mechanical ventilation or ECMO compared to placebo
- Data showed 46% risk reduction in mortality by Day 28 and 44% risk reduction in mortality by Day 60
INDIANAPOLIS, Aug. 3, 2021 /PRNewswire/ -- Eli Lilly and Company (NYSE: LLY) and Incyte (NASDAQ:INCY) announced today results from an additional cohort of 101 adult patients from the COV-BARRIER trial. In this sub-study, patients with COVID-19 on mechanical ventilation or extracorporeal membrane oxygenation (ECMO) who received baricitinib plus standard of care were 46 percent less likely to die by Day 28 compared to patients who received placebo plus standard of care (nominal p-value=0.0296; hazard ratio [HR] [95% CI] = 0.54 [0.31, 0.96]; analysis not adjusted for multiplicity). The cumulative proportion of patients who died by Day 28 was 39.2 percent (n/N: 20/51) in the baricitinib arm versus 58 percent in the placebo arm (n/N: 29/50). Similar mortality benefit was observed by Day 60 (HR [96% CI] = 0.56 [0.33, 0.97]) with a cumulative proportion of death of 45.1 percent (n/N: 23/51) for baricitinib compared to 62.0 percent for placebo (n/N: 31/50). These findings are consistent with the reduction in mortality observed in the overall COV-BARRIER patient population.
"As additional data from COV-BARRIER become available, it is increasingly evident that treatment with baricitinib may help prevent death in some of the most critically ill COVID-19 patients and that baricitinib represents an important treatment option for this vulnerable group of patients in this constantly evolving pandemic," said E. Wesley Ely, M.D., M.P.H., professor of medicine and co-director of the Critical Illness, Brain Dysfunction, and Survivorship (CIBS) Center at Vanderbilt University Medical Center and co-principal investigator of COV-BARRIER.
By Day 28, the frequency of adverse events, serious adverse events and serious infections were similar in the baricitinib group (88%, 50% and 44%, respectively) compared to placebo (95.9%, 71.4% and 53.1%, respectively). Venous thromboembolic events were reported in 6 percent of patients treated with baricitinib and 6.1 percent of patients treated with placebo. No new safety signals were identified.
On July 28, 2021, the U.S. Food and Drug Administration (FDA) broadened the Emergency Use Authorization (EUA) for baricitinib to allow for treatment with or without remdesivir. The EUA provides for the use of baricitinib for treatment of COVID-19 in hospitalized adults and pediatric patients two years of age or older requiring supplemental oxygen, non-invasive or invasive mechanical ventilation or ECMO. For more information about the authorized use of baricitinib in COVID-19 and mandatory requirements of the EUA, please see the FDA Letter of Authorization, Fact Sheet for Healthcare Providers and Fact Sheet for Patients, Parents and Caregivers (English) (Spanish).
Baricitinib is an oral JAK inhibitor discovered by Incyte and licensed to Lilly.
Authorized Use Under the EUA and Important Safety Information for baricitinib (in the United States) for COVID-19
Baricitinib is authorized for use under an Emergency Use Authorization (EUA) for treatment of COVID-19 in hospitalized adults and pediatric patients 2 years of age or older requiring supplemental oxygen, non-invasive or invasive mechanical ventilation, or extracorporeal membrane oxygenation (ECMO).
Baricitinib has not been approved for the treatment of COVID-19, but has been authorized for emergency use by the FDA. Baricitinib is authorized under an EUA only for the duration of the declaration that circumstances exist justifying the authorization of the EUA of baricitinib under Section 564(b)(1) of the Act, 21 U.S.C. § 360bbb-3(b)(1), unless the declaration is terminated or authorization revoked sooner.
For more information about the authorized use of baricitinib in COVID-19 and mandatory requirements of the EUA, please see the FDA Letter of Authorization, Fact Sheet for Healthcare Providers and Fact Sheet for Patients, Parents and Caregivers (English) (Spanish).
Please see Fact Sheet for Healthcare Providers and Fact Sheet for Patients, Parents and Caregivers (English) or Fact Sheet for Patients, Parents and Caregivers (Spanish).
BC HCP EUA ISI 28JUL2021
About OLUMIANT ® (baricitinib)
OLUMIANT, a once-daily, oral JAK inhibitor was discovered by Incyte and licensed to Lilly. It is approved in the U.S. and more than 75 countries as a treatment for adults with moderate to severe rheumatoid arthritis. It is also approved for the treatment of certain hospitalized patients with COVID-19 in Japan. The U.S. FDA-approved labeling for OLUMIANT includes a Boxed Warning for Serious Infections, Malignancy, and Thrombosis. See the full Prescribing Information here.
In December 2009, Lilly and Incyte announced an exclusive worldwide license and collaboration agreement for the development and commercialization of baricitinib and certain follow-on compounds for patients with inflammatory and autoimmune diseases.
About COV-BARRIER Sub-Study
COV-BARRIER sub-study was a randomized, double-blind, placebo-controlled trial, initiated in December 2020, to assess the efficacy and safety of baricitinib versus placebo when added to standard of care which included corticosteroids (86% of the patients) in patients hospitalized with COVID-19 requiring invasive mechanical ventilation or ECMO at baseline. A total of 101 patients were randomized to baricitinib or placebo with 51 receiving baricitinib and 50 receiving placebo. The pre-specified endpoints include 28-days mortality (and 60-day) and the number of ventilator-free days. All analyses are exploratory and no multiplicity adjustment was applied.
COV-BARRIER was a global, randomized, double-blind, placebo-controlled study of hospitalized patients comparing baricitinib 4 mg once daily plus standard of care versus placebo plus standard of care. Patients could remain on background standard of care, as defined per local guidelines, including antimalarials, antivirals, corticosteroids, and/or azithromycin. The most frequently used therapies were corticosteroids (79% of patients, mostly dexamethasone) and remdesivir (19% of patients). While the composite primary endpoint of COV-BARRIER, which was defined as a difference in the estimated proportion of participants progressing to non-invasive ventilation including high flow oxygen or invasive mechanical ventilation (including ECMO) or death by Day 28, did not meet statistical significance, baricitinib-treated patients (27.8%) were less likely than those receiving standard of care (30.5%) to progress to ventilation or death (odds ratio [OR]: 0.85; 95% CI: 0.67, 1.08; p=0.180). A pre-specified key secondary endpoint showed baricitinib, in addition to standard of care, meaningfully reduced the risk of death by 39 percent by Day 28 when compared to standard of care alone (n/N: 62/764 [8.1%] baricitinib, 101/761 [13.3%] placebo; [estimated difference in Day 28 probability of mortality = -4.9% (95% CI: -8.0%, -1.9%); hazard ratio [HR] = 0.56 (95% CI: 0.41, 0.77)]. No new safety signals were identified. The study findings from COV-BARRIER have been submitted to a peer-reviewed journal for future print publication.
To learn more about Lilly, please visit us at lilly.com
For additional information on Incyte, please visit Incyte.com
View original content to download multimedia:https://www.prnewswire.com/news-releases/lilly-and-incytes-baricitinib-reduced-deaths-among-patients-with-covid-19-receiving-invasive-mechanical-ventilation-301346651.html
SOURCE Eli Lilly and Company
Lilly, Incyte's baricitinib reduces deaths among COVID-19 patients on mechanical ventilation
Aug. 03, 2021 6:55 AM ET Eli Lilly and Company (LLY), INCY Eli Lilly and Company (LLY)Incyte Corporation (INCY) By: Mamta Mayani, SA News Editor
- Eli Lilly (NYSE:LLY) and Incyte (NASDAQ:INCY) announce results from an additional cohort of 101 adult patients from the COV-BARRIER trial.
- https://seekingalpha.com/news/3723668-lilly-incytes-baricitinib-reduces-deaths-among-covid-19-patients-on-mechanical-ventilation
- https://seekingalpha.com/symbol/LLY
- https://seekingalpha.com/symbol/INCY
- https://www.olumiant.com/
- https://investor.lilly.com/
KEYTRUDA® (pembrolizumab)
Merck’s KEYTRUDA® (pembrolizumab) Significantly Prolonged Recurrence-Free Survival (RFS) Compared to Placebo as Adjuvant Therapy for Patients With Stage II Resected High-Risk Melanoma in Phase 3 KEYNOTE-716 Trial
August 5, 2021 6:45 am ET
US FDA Grants Priority Review to Merck’s Application for KEYTRUDA Based on These Data
KENILWORTH, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced that the Phase 3 KEYNOTE-716 trial investigating KEYTRUDA, Merck’s anti-PD-1 therapy, met its primary endpoint of recurrence-free survival (RFS) for the adjuvant treatment of patients with surgically resected high-risk stage II melanoma. At an interim analysis, treatment with KEYTRUDA as a single agent showed a statistically significant and clinically meaningful improvement in RFS compared with placebo as adjuvant therapy for these patients. No new safety signals were observed. These results will be presented at an upcoming medical meeting. Based on these data, the U.S. Food and Drug Administration (FDA) has accepted a new supplemental Biologics License Application (sBLA) for KEYTRUDA for the adjuvant treatment of adult and pediatric (12 years and older) patients with stage IIB or IIC melanoma following complete resection. The FDA granted the application Priority Review and assigned a Prescription Drug User Fee Act (PDUFA), or target action, date of December 4, 2021.
“KEYNOTE-716 is the first Phase 3 study to evaluate adjuvant therapy solely for stage IIB and IIC melanoma – an area with high unmet need,” said Dr. Jason Luke, director, Cancer Immunotherapeutics Center at UPMC Hillman Cancer Center. “By moving immunotherapy with KEYTRUDA to earlier stages of melanoma, we have the opportunity to reduce the risk of recurrence for high-risk stage II patients compared to observation alone following complete resection.”
Merck has an expansive program investigating KEYTRUDA in early disease states, with approximately 20 registrational studies ongoing. KEYNOTE-716 adds to previous results seen in earlier stages of disease in four tumor types (melanoma, renal cell carcinoma, triple-negative breast cancer and non-muscle invasive bladder cancer).
About KEYNOTE-716
KEYNOTE-716 is a randomized, two-part, Phase 3 trial (ClinicalTrials.gov, NCT03553836) evaluating KEYTRUDA for the adjuvant treatment of patients with completely resected high-risk stage II melanoma. The study enrolled 954 patients ages 12 years and older. The primary endpoint is RFS, and the secondary endpoints include distant metastasis-free survival, overall survival, safety and quality of life.
In Part 1 of the study, which was double-blind, adult patients were randomized to receive KEYTRUDA (200 mg intravenously [IV]) or placebo (saline by IV) every three weeks for up to 17 cycles (approximately one year); pediatric patients were randomized to receive KEYTRUDA (2 mg/kg [200 mg maximum] by IV) or placebo every three weeks for up to 17 cycles (approximately one year).
In Part 2 of the study, which was open-label, eligible adult and pediatric patients received up to 35 additional cycles (approximately two years) of KEYTRUDA. Eligibility for Part 2 included patients who recurred after receiving placebo or completed 17 cycles of KEYTRUDA (approximately one year); patients on KEYTRUDA must not have experienced disease recurrence within six months of completing treatment.
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 1,500 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.
Selected KEYTRUDA® (pembrolizumab) Indications in the U.S.
Melanoma
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.
KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.
Non-Small Cell Lung Cancer
KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) ≥1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is:
- stage III where patients are not candidates for surgical resection or definitive chemoradiation, or
- metastatic.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS ≥1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.
Head and Neck Squamous Cell Cancer
KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS ≥1)] as determined by an FDA-approved test.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.
Classical Hodgkin Lymphoma
KEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).
KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.
Primary Mediastinal Large B-Cell Lymphoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.
Urothelial Carcinoma
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 (CPS ≥10), as determined by an FDA-approved test, or in patients who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
KEYTRUDA is indicated for the treatment of patients with locally advanced or mUC who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.
Microsatellite Instability-High or Mismatch Repair Deficient Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options.
This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.
Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).
Gastric Cancer
KEYTRUDA, in combination with trastuzumab, fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of patients with locally advanced unresectable or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Esophageal Cancer
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic esophageal or GEJ (tumors with epicenter 1 to 5 centimeters above the GEJ) carcinoma that is not amenable to surgical resection or definitive chemoradiation either:
- in combination with platinum- and fluoropyrimidine-based chemotherapy, or
- as a single agent after one or more prior lines of systemic therapy for patients with tumors of squamous cell histology that express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
Cervical Cancer
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Hepatocellular Carcinoma
KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Merkel Cell Carcinoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Renal Cell Carcinoma
KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma.
Tumor Mutational Burden-High Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [≥10 mutations/megabase] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.
Cutaneous Squamous Cell Carcinoma
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) or locally advanced cSCC that is not curable by surgery or radiation.
Triple-Negative Breast Cancer
KEYTRUDA is indicated for the treatment of patients with high-risk early-stage TNBC in combination with chemotherapy as neoadjuvant treatment, and then continued as a single agent as adjuvant treatment after surgery.
KEYTRUDA, in combination with chemotherapy, is indicated for the treatment of patients with locally recurrent unresectable or metastatic TNBC whose tumors express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
For more information, visit www.merck.com
Please see Prescribing Information for KEYTRUDA (pembrolizumab) at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_pi.pdf and Medication Guide for KEYTRUDA at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_mg.pdf.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210805005406/en/
Source: Merck & Co., Inc.
Merck’s Keytruda prolonged recurrence-free survival in late-stage melanoma study
Aug. 05, 2021 8:32 AM ETMerck & Co., Inc. (MRK)Merck & Co., Inc. (MRK)By: Mamta Mayani, SA News Editor3 Comments
- Merck (NYSE:MRK) announces that the Phase 3 KEYNOTE-716 trial investigating Keytruda, met its primary endpoint of recurrence-free survival (RFS) for the adjuvant treatment of patients with surgically resected high-risk stage II melanoma.
- https://seekingalpha.com/news/3726071-mercks-keytruda-prolonged-recurrence-free-survival-in-late-stage-melanoma-study
- https://seekingalpha.com/symbol/MRK
- https://www.merck.com/
- https://www.keytruda.com/
XPOVIO® (selinexor)
Antengene Announces the Approval of First-in-Class Oral XPO1 Inhibitor Selinexor in South Korea for the Treatment of Relapsed or Refractory Multiple Myeloma and Diffuse Large B-Cell Lymphoma
Aug 02, 2021View PDF
Seoul, South Korea, July XX, 2021 – Antengene Corporation Limited (“Antengene”, SEHK: 6996.HK), a leading innovative biopharmaceutical company dedicated to discovering, developing and commercializing global first-in-class and/or best-in-class therapeutics in hematology and oncology, today announced that through a priority review process, the South Korean Ministry of Food and Drug Safety (MFDS) has approved the company’s New Drug Application (NDA) for the Orphan Drug-designated first-in-class oral inhibitor of XPO1, selinexor (XPOVIO®), in combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma (rrMM) who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody (penta-refractory); and as a monotherapy for the treatment of adult patients with relapsed/refractory diffuse large B-cell lymphoma (rrDLBCL) who have received at least two prior lines of treatment.
About XPOVIO® (selinexor)
XPOVIO is a first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) compound. XPOVIO functions by selectively binding to and inhibiting the nuclear export protein exportin 1 (XPO1, also called CRM1). XPOVIO 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.
Antengene's selinexor approved in South Korea for multiple myeloma and diffuse large b-cell lymphoma
Aug. 02, 2021 2:01 AM ETKaryopharm Therapeutics Inc. (KPTI)Karyopharm Therapeutics Inc. (KPTI)By: Mamta Mayani, SA News Editor
- Karyopharm Therapeutics' (NASDAQ:KPTI) partner Antengene announces that through a priority review process, the South Korean Ministry of Food and Drug Safety has approved its New Drug Application (NDA) for the Orphan Drug-designated oral inhibitor of XPO1, selinexor (XPOVIO), in combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma (rrMM) who have received at least four prior therapies; and as a monotherapy for the treatment of adult patients with relapsed/refractory diffuse large B-cell lymphoma (rrDLBCL) who have received at least two prior lines of treatment.
- https://seekingalpha.com/news/3722774-antengenes-selinexor-approved-in-south-korea-for-multiple-myeloma-and-diffuse-large-b-cell-lymphoma
- https://seekingalpha.com/symbol/KPTI
- https://www.karyopharm.com/pipeline/oral-selinexor/
- https://www.xpovio.com/
- https://www.karyopharm.com/
BRUKINSA® (Zanubrutinib)
BeiGene Announces Positive Topline Results from Phase 3 SEQUOIA Trial Comparing BRUKINSA® (Zanubrutinib) to Bendamustine Plus Rituximab in Patients with Treatment-Naïve Chronic Lymphocytic Leukemia
Jul 29, 2021 8:00 PM
Trial met the primary endpoint at interim analysis, with BRUKINSA significantly prolonging progression-free survival compared to chemoimmunotherapy and safety and tolerability consistent with its known profile
SEQUOIA is the second positive global Phase 3 trial of BRUKINSA in chronic lymphocytic leukemia, following ALPINE in relapsed or refractory setting
CAMBRIDGE, Mass. & BEIJING, China--(BUSINESS WIRE)-- BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a global, science-driven biotechnology company, today announced positive topline results from an interim analysis of the Phase 3 SEQUOIA trial comparing BRUKINSA® (zanubrutinib) to bendamustine and rituximab (B+R) in patients with treatment-naïve (TN) chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) whose tumor did not exhibit the deletion of chromosome 17p13.1 (del[17p]).
With a median follow-up of 25.8 months, the SEQUOIA trial met the primary endpoint of progression-free survival (PFS) as assessed by independent review committee (IRC), as BRUKINSA achieved a highly statistically significant improvement in PFS compared to B+R.
In addition, the trial demonstrated a statistically significant improvement in PFS per investigator assessment, a secondary endpoint. BRUKINSA was also generally well-tolerated, consistent with its known safety profile.
“The combined clinical evidence from SEQUOIA, ALPINE1, the 205 trial2, and the AU-003 trial3 validates our confidence in BRUKINSA as a regimen which can offer improvements in treatment outcomes for hundreds of thousands of patients living with CLL,” said Jane Huang, M.D., Chief Medical Officer, Hematology at BeiGene. “We are pleased to see that at the interim analysis of the SEQUOIA trial, BRUKINSA significantly prolonged progression-free survival for treatment-naïve CLL patients, and that the demonstrated safety profile was consistent with what we have observed in its global development program with more than 2,300 patients treated with BRUKINSA to date.”
1. Results from the interim analysis of ALPINE with a median follow-up time of 15.3 months were reported at the 2021 European Hematology Association (EHA2021) Congress in June 2021. Available at EHA Open Access Library.
2. Long-term results from BGB-3111-205 with a median follow-up time of 34 months were reported at the EHA2021 Congress in June 2021. Available at EHA Open Access Library.
3. Long-term results from BGB-3111-AU-003 in relapsed or refractory CLL with a median follow-up time of 39.4 months were shared at the BeiGene Investor Conference Call in June 2021. Available ir.beigene.com.
About SEQUOIA
SEQUOIA is a randomized, multicenter, global Phase 3 trial (NCT03336333) designed to evaluate the efficacy and safety of BRUKINSA compared to B+R in patients with TN CLL or SLL. The trial consists of three cohorts:
- Cohort 1 (n=479): randomized 1:1 to receive BRUKINSA (n=241) or B+R (n=238) until disease progression or unacceptable toxicity, in patients not harboring del(17p); data from this group comprise the primary endpoint;
- Cohort 2 (n=110): patients with del(17p) receiving BRUKINSA as a monotherapy; and
- Cohort 3 (enrollment ongoing): patients with del(17p) or pathogenic TP53 variant receiving BRUKINSA in combination with venetoclax.
Patients with del(17p) were not randomized to B+R, as they experience poor clinical outcomes and poor response to chemoimmunotherapy. The primary endpoint of the trial is IRC-assessed PFS. Secondary endpoints include investigator-assessed PFS, IRC- and investigator-assessed overall response rate (ORR), overall survival (OS), PFS and ORR in patients with del(17p), and safety.
Cohort 2, representing high-risk patients treated with BRUKINSA monotherapy, was previously presented at the American Society for Hematology (ASH) Annual Meeting in December 2020. This cohort of patients with del(17p) achieved significant efficacy with an 18-month PFS of 90.6%, as assessed by investigator.
BeiGene plans to consult with global regulatory authorities on next steps and present these data at an upcoming major medical conference.
About BRUKINSA
BRUKINSA is a small molecule inhibitor of Bruton’s tyrosine kinase (BTK) discovered by BeiGene scientists that is currently being evaluated globally in a broad clinical program as a monotherapy and in combination with other therapies to treat various B-cell malignancies. Because new BTK is continuously synthesized, BRUKINSA was specifically designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity. With differentiated pharmacokinetics compared to other approved BTK inhibitors, BRUKINSA has been demonstrated to inhibit the proliferation of malignant B cells within a number of disease relevant tissues.
BRUKINSA is approved in the following indications and regions:
- For the treatment of mantle cell lymphoma (MCL) in adult patients who have received at least one prior therapy (United States, November 2019)*;
- For the treatment of MCL in adult patients who have received at least one prior therapy (China, June 2020)**;
- For the treatment of chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) in adult patients who have received at least one prior therapy (China, June 2020)**;
- For the treatment of relapsed or refractory MCL (United Arab Emirates, February 2021);
- For the treatment of Waldenström’s macroglobulinemia (WM) in adult patients (Canada, March 2021);
- Registered and reimbursed for the treatment of MCL in patients who have received at least one prior therapy (Israel, April 2021);
- For the treatment of adult patients with WM who have received at least one prior therapy (China, June 2021)**; and
- For the treatment of MCL in adult patients who have received at least one prior therapy (Canada, July 2021).
To date, more than 30 marketing authorization applications in multiple indications have been submitted covering the United States, the European Union, and more than 20 other countries or regions.
* This indication was approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
** This indication was approved under conditional approval. Complete approval for this indication may be contingent upon results from ongoing randomized, controlled confirmatory clinical trials.
To learn more about BeiGene, please visit www.beigene.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20210729006225/en/
Source: BeiGene, Ltd.
BeiGene reports positive results from Phase 3 Brukinsa study in blood cancer
Jul. 30, 2021 4:20 AM ET
BeiGene, Ltd. (BGNE)BeiGene, Ltd. (BGNE)
By: Mamta Mayani, SA News Editor
- BeiGene (NASDAQ:BGNE) announces positive topline results from an interim analysis of Phase 3 SEQUOIA trial comparing BRUKINSA (zanubrutinib) to bendamustine and rituximab (B+R) in patients with treatment-naïve (TN) chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) whose tumor did not exhibit the deletion of chromosome 17p13.1 (del[17p]).
- https://seekingalpha.com/news/3722142-beigene-reports-positive-results-from-phase-3-brukinsa-study-in-blood-cancer
- https://seekingalpha.com/symbol/BGNE
- https://www.brukinsa.com/
- https://www.beigene.com/
Tecentriq®(atezolizumab)
Monday, Aug 2, 2021
FDA Grants Priority Review to Genentech’s Tecentriq as Adjuvant Treatment for Certain People With Early Non-Small Cell Lung Cancer
Application is being reviewed under FDA's Real-Time Oncology Review
pilot program
Based on results of the Phase III IMpower010 study presented at ASCO that showed adjuvant Tecentriq improved disease-free survival by more than one-third in PD-L1-positive early-stage lung cancer, compared with best supportive care
Tecentriq is the first and only cancer immunotherapy to demonstrate positive Phase III results in the adjuvant lung cancer setting
South San Francisco, CA -- August 2, 2021 --
Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s supplemental Biologics License Application (sBLA) and granted Priority Review for Tecentriq® (atezolizumab) as adjuvant treatment following surgery and platinum-based chemotherapy for people with non-small cell lung cancer (NSCLC) whose tumors express PD-L1≥1%, as determined by an FDA-approved test. The FDA is reviewing the application under the Real-Time Oncology Review pilot program, which aims to explore a more efficient review process to ensure safe and effective treatments are available to patients as early as possible. The FDA is expected to make a decision on approval by December 1, 2021.
This application is based on disease-free survival (DFS) results from an interim analysis of the Phase III IMpower010 study, the first and only Phase III study of a cancer immunotherapy to demonstrate positive results in the adjuvant lung cancer setting. The study showed that treatment with Tecentriq following surgery and platinum-based chemotherapy reduced the risk of disease recurrence or death (DFS) by 34% (hazard ratio [HR]=0.66, 95% CI: 0.50-0.88) in people with Stage II-IIIA NSCLC whose tumors express PD-L1≥1%, compared with best supportive care (BSC). In this population, median DFS was not yet reached for Tecentriq compared with 35.3 months for BSC. Follow-up on the IMpower010 trial will continue with planned analyses of DFS in the overall intent-to-treat (ITT) population, including Stage IB patients, which at the time of analysis did not cross the threshold, and overall survival (OS) data, which were immature at the time of interim analysis. Safety data for Tecentriq were consistent with its known safety profile and no new safety signals were identified. Results from the IMpower010 trial were presented at the 2021 ASCO Annual Meeting.
About the IMpower010 study
IMpower010 is a Phase III, global, multicenter, open-label, randomized study evaluating the efficacy and safety of Tecentriq compared with BSC, in participants with Stage IB-IIIA NSCLC (UICC 7th edition), following surgical resection and up to 4 cycles of adjuvant cisplatin-based chemotherapy. The study randomized 1,005 people with a ratio of 1:1 to receive either Tecentriq (up to 16 cycles) or BSC. The primary endpoint is investigator-determined DFS in the PD-L1-positive Stage II-IIIA, all randomized Stage II-IIIA and ITT Stage IB-IIIA populations. Key secondary endpoints include OS in the overall study population, ITT Stage IB-IIIA NSCLC.
About Tecentriq®(atezolizumab)
Tecentriq is a monoclonal antibody designed to bind with a protein called PD-L1. Tecentriq is designed to bind to PD-L1 expressed on tumor cells and tumor-infiltrating immune cells, blocking its interactions with both PD-1 and B7.1 receptors. By inhibiting PD-L1, Tecentriq may enable the re-activation of T cells. Tecentriq may also affect normal cells.
Tecentriq U.S. Indications
Tecentriq is a prescription medicine used to treat adults with:
A type of lung cancer called non-small cell lung cancer (NSCLC).
- Tecentriq may be used alone as their first treatment when their lung cancer:
- has spread or grown, and
- their cancer tests positive for “high PD-L1”, and
- their tumor does not have an abnormal “EGFR” or “ALK” gene
- Tecentriq may be used with the medicines bevacizumab, paclitaxel, and carboplatin as their first treatment when their lung cancer:
- has spread or grown, and
- is a type called “non-squamous NSCLC,” and
- their tumor does not have an abnormal “EGFR” or “ALK” gene
- Tecentriq may be used with the medicines paclitaxel protein-bound and carboplatin as their first treatment when their lung cancer:
- has spread or grown, and
- is a type called “non-squamous NSCLC,” and
- their tumor does not have an abnormal “EGFR” or “ALK” gene
- Tecentriq may also be used when their lung cancer:
- has spread or grown, and
- they have tried chemotherapy that contains platinum, and it did not work or is no longer working
- if their tumor has an abnormal “EGFR” or “ALK” gene, they should have also tried an FDA-approved therapy for tumors with these abnormal genes, and it did not work or is no longer working
A type of lung cancer called small cell lung cancer (SCLC).
- Tecentriq may be used with the chemotherapy medicines carboplatin and etoposide as their first treatment when their lung cancer:
- is a type called “extensive-stage small cell lung cancer,” which means that it has spread or grown
It is not known if Tecentriq is safe and effective in children.
Please see http://www.Tecentriq.com for full Prescribing Information and additional Important Safety Information.
For more information visit http://www.gene.com/cancer-immunotherapy.
For additional information about the company, please visit http://www.gene.com.
FDA grants priority review to Genentech’s Tecentriq in lung cancer
Aug. 03, 2021 5:11 AM ETRoche Holding AG (RHHBY)Roche Holding AG (RHHBY)By: Mamta Mayani, SA News Editor
- The FDA has accepted Roche (OTCQX:RHHBY) unit, Genentech's supplemental Biologics License Application (sBLA) and granted Priority Review for Tecentriq (atezolizumab) as adjuvant treatment following surgery and platinum-based chemotherapy for people with non-small cell lung cancer (NSCLC) whose tumors express PD-L1≥1%, as determined by an FDA-approved test.
- https://seekingalpha.com/news/3723599-fda-grants-priority-review-to-genentechs-tecentriq-in-lung-cancer
- https://seekingalpha.com/symbol/RHHBY
- https://www.tecentriq.com/
- https://www.gene.com/
UPTRAVI® (selexipag)
UPTRAVI® (selexipag) Receives FDA Approval for Intravenous Use in Adult Patients with Pulmonary Arterial Hypertension (PAH)
Jul 30, 2021United States
New formulation allows for uninterrupted treatment for PAH patients temporarily unable to take oral therapy
TITUSVILLE, N.J. – July 30, 2021 – The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that the U.S. Food and Drug Administration (FDA) has approved UPTRAVI® (selexipag) injection for intravenous (IV) use for the treatment of pulmonary arterial hypertension (PAH, WHO Group I) in adult patients with WHO functional class (FC) II–III, who are temporarily unable to take oral therapy. UPTRAVI® IV is a therapeutic option that will allow patients to avoid short-term treatment interruptions and stay on UPTRAVI® therapy, as uninterrupted treatment is considered key for individuals with PAH. UPTRAVI® tablets were first approved by the FDA in 2015 to delay disease progression and reduce the risk of hospitalization for PAH.1
“Given the progressive nature of this disease, maintaining treatment is important to help control PAH. However, there are times where patients may be unable to take oral medications. For patients on UPTRAVI, bridging short-term temporary interruptions of UPTRAVI tablets with UPTRAVI IV may maintain the treatment effect and avoid the need to change therapy or re-titrate UPTRAVI tablets after re-initiation,” said Kelly Chin, M.D.*, UPTRAVI® IV study senior author and Associate Professor of Internal Medicine and Director of the Pulmonary Hypertension Program at The University of Texas Southwestern Medical Center.
The FDA approval of the New Drug Application (NDA) for UPTRAVI® is based upon the findings from a prospective, multi-center, open-label single sequence cross-over Phase 3 study designed to assess the safety, tolerability and pharmacokinetics of temporarily switching between UPTRAVI® tablets and UPTRAVI® IV. The results of the study were published earlier this year in Respiratory Research and examined switching from a stable dose of UPTRAVI® tablets to a corresponding dose of UPTRAVI® IV and back to UPTRAVI® tablets.2
“Today marks an important day for patients who rely on UPTRAVI, as this new intravenous formulation meets a current unmet need for these patients. As part of our commitment to investing in research and understanding the science around the potential of UPTRAVI, we’re inspired by this approval and are proud to be paving the way to advance treatment options and care for patients with PAH,” said Neil Davie, PhD**, Global Therapeutic Area Head, Pulmonary Hypertension, Janssen.
The UPTRAVI® IV study enrolled 20 patients who received all UPTRAVI® doses (either tablets or IV). The study found that the switch between UPTRAVI® tablets and UPTRAVI® IV was well tolerated with no unexpected safety findings. Adverse events (AEs) that resulted from UPTRAVI® IV were similar to those associated with UPTRAVI® tablets, with the exception of infusion site reactions reported in two patients (both of which were considered mild-to-moderate in intensity and neither led to study and/or treatment discontinuation). The prostacyclin-associated AEs included headache, diarrhea, nausea, vomiting, pain in jaw, myalgia, pain in extremity, flushing, and arthralgia.2
About UPTRAVI® (selexipag)
Selexipag, a selective prostacyclin IP receptor agonist, is a compound discovered by Nippon Shinyaku and licensed to Actelion Pharmaceuticals Ltd outside Japan. It is licensed for the oral treatment of PAH in more than 60 countries.
About the UPTRAVI® IV Study
The UPTRAVI® IV study (NCT03187678) was a prospective, multi-center, open-label single-sequence cross–over, Phase 3 study designed to assess the safety, tolerability and pharmacokinetics of temporarily switching between oral UPTRAVI® and UPTRAVI® IV in 20 patients. The study examined switching from a stable dose of UPTRAVI® tablets to a corresponding dose of UPTRAVI® IV and back to UPTRAVI® tablets. The treatment and observation phase was divided into three periods. In Period 1, patients received their stable oral dose of UPTRAVI® twice daily (morning and evening of Day 1). In Period 2, patients received three infusions of corresponding UPTRAVI® IV doses (morning and evening of Day 2, and morning of Day 3). In Period 3, patients resumed their stable oral UPTRAVI® dose twice daily in the evening of Day 3 for 9 days, which was continued through the safety follow-up. Patients were hospitalized during Periods 1 and 2.2
INDICATION AND IMPORTANT SAFETY INFORMATION
What is UPTRAVI®?
UPTRAVI® (selexipag) is a prescription medicine used to treat pulmonary arterial hypertension (PAH, WHO Group 1), which is high blood pressure in the arteries of your lungs.
UPTRAVI® can help delay (slow down) the progression of your disease and lower your risk of being hospitalized for PAH.
It is not known if UPTRAVI® is safe and effective in children.
Please see full Prescribing Information and Patient Product Information.
Learn more at www.janssen.com. Follow us at www.twitter.com/JanssenGlobal and www.twitter.com/JanssenUS. Actelion Pharmaceuticals US, Inc. and Actelion Pharmaceuticals Ltd are Janssen Pharmaceutical Companies of Johnson & Johnson.
FDA OKs Janssen Pharma's Uptravi for intravenous use in adults with PAH
Jul. 30, 2021 8:43 AM ETJohnson & Johnson (JNJ)Johnson & Johnson (JNJ)By: Mamta Mayani, SA News Editor
- Johnson & Johnson (NYSE:JNJ) company Janssen Pharmaceutical announces that the FDA has approved UPTRAVI (selexipag) injection for intravenous (IV) use for the treatment of pulmonary arterial hypertension (PAH) in adult patients with WHO functional class II–III, who are temporarily unable to take oral therapy.
- https://seekingalpha.com/news/3722343-fda-oks-janssen-pharmas-uptravi-for-intravenous-use-in-adults-with-pah
- https://seekingalpha.com/symbol/JNJ
- https://www.janssen.com/
- https://www.uptravi.com/
Nucala (mepolizumab)
29 July 2021
GSK announces FDA approval for Nucala (mepolizumab) for use in adults with chronic rhinosinusitis with nasal polyps
For media and investors only
Issued: 29 July 2021, London UK
- First IL-5 therapy approved as an add-on treatment in the US for adults with chronic rhinosinusitis with nasal polyps to target eosinophilic inflammation
- Fourth indication for mepolizumab in the US for eosinophil-driven diseases
GlaxoSmithKline plc (GSK) today announced that the US Food and Drug Administration (FDA) has approved Nucala (mepolizumab), a monoclonal antibody that targets interleukin-5 (IL-5), as a treatment for patients with chronic rhinosinusitis with nasal polyps (CRSwNP). This new indication for mepolizumab is for the add-on maintenance treatment of CRSwNP in adult patients 18 years of age and older with inadequate response to nasal corticosteroids.
CRSwNP accounts for 2-4% of the US population, affecting more than 5 million people. CRSwNP is one of a variety of diseases arising from inflammation in different tissues associated with elevated levels of a type of white blood cell called eosinophils. It is often characterised by raised eosinophil levels, in which soft tissue growth, known as nasal polyps, develop in the sinuses and nasal cavity. CRSwNP can cause chronic symptoms such as nasal obstruction, loss of smell, facial pressure and nasal discharge.
Mepolizumab is the first anti-IL-5 biologic to be approved for adult patients with CRSwNP in the US.
Mepolizumab is also approved for use in three other eosinophilic driven diseases, the first indication being for patients with severe eosinophilic asthma aged six years and older. Additionally, mepolizumab was the first biologic therapy indicated for adults with eosinophilic granulomatosis with polyangiitis (EGPA) and also the first biologic to be approved for patients aged 12 years and older with hypereosinophilic syndrome (HES).
With 41 clinical trials, mepolizumab has been studied in over 4,000 patients. GSK is committed to improving the lives of those living with disease associated with uncontrolled eosinophilic inflammation, continuously innovating in order to address the unmet needs in this broad patient group.
Nucala is indicated in the US:
- As an add-on maintenance treatment of adult and pediatric patients aged 6 years and older with severe asthma and with an eosinophilic phenotype. Nucala is not indicated for the relief of acute bronchospasm or status asthmaticus.
- For the treatment of adult patients with EGPA.
- For the treatment of adult and paediatric patients aged 12 years and older with HES for ≥6 months without an identifiable non-hematologic secondary cause.
- As an add-on maintenance treatment of CRSwNP in adult patients 18 years of age and older with inadequate response to nasal corticosteroids.
About mepolizumab
First approved in 2015 for severe eosinophilic asthma (SEA), mepolizumab is the first-in-class monoclonal antibody that targets IL-5. It is believed to work by preventing IL-5 from binding to its receptor on the surface of eosinophils, reducing blood eosinophils and maintaining them within normal levels. A normal level of blood eosinophils being less than 500 eosinophils/microliter. The mechanism of action for mepolizumab has not been definitively established.
Mepolizumab has been developed for the treatment of diseases that are driven by inflammation caused by eosinophils. It has been studied in over 4,000 patients in 41 clinical trials across a number of eosinophilic indications and has been approved under the brand name Nucala in the US, Europe and in over 20 other markets, as an add-on maintenance treatment for patients with SEA. It is approved for paediatric use in SEA from ages six to 17 in Europe, the US and several other markets. In the US, Japan, Canada and a number of other markets, it is approved for use in adult patients with EGPA. Mepolizumab was approved for use in HES in the US in September 2020, followed by Brazil in February 2021 and Argentina in May 2021. Mepolizumab is currently being investigated in COPD. It is not currently approved for use in COPD anywhere in the world.
For further information please visit www.gsk.com/about-us.
GlaxoSmithKline announces FDA approval of Nucala for new eosinophilic-driven indication
Jul. 29, 2021 3:04 PM ET
GlaxoSmithKline plc (GSK)GlaxoSmithKline plc (GSK)
By: Dulan Lokuwithana, SA News Editor1 Comment
- GlaxoSmithKline (GSK +1.7%) announced the FDA approval of Nucala (mepolizumab) for the treatment of chronic rhinosinusitis with nasal polyps (CRSwNP), a disease characterized by a rise in white blood cells called eosinophils.
- https://seekingalpha.com/news/3721784-glaxosmithkline-announces-fda-approval-of-nucala-for-new-e
- https://seekingalpha.com/symbol/GSKosinophilic-driven-indication
- https://www.gsk.com/en-gb/
- https://www.nucala.com/
Sotrovimab is an investigational SARS-CoV-2 monoclonal antibody
28 July 2021
GSK and Vir Biotechnology announce Joint Procurement Agreement with European Commission for COVID-19 treatment, sotrovimab
For media and investors only
Issued: London UK and San Francisco, US
GlaxoSmithKline plc and Vir Biotechnology, Inc. today announced they have signed a Joint Procurement Agreement with the European Commission to supply up to 220,000 doses of sotrovimab, an
investigational single dose SARS-CoV-2 monoclonal antibody for the treatment of adults and adolescents (aged 12 years and over and weighing at least 40 kg) with COVID-19 who do not require oxygen supplementation and who are at risk of progressing to severe COVID-19. The Joint Procurement Agreement enables participating European Union (EU) Member States to quickly purchase sotrovimab, following local emergency authorisation or authorisation at the EU level, to treat high-risk patients with COVID-19 who may benefit from early treatment with sotrovimab.
This action follows the positive scientific opinion issued by the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP), under Article 5(3) of Regulation 726/2004, which can be considered by the national authorities in EU Member States when taking evidence-based decisions on the early use of the medicine prior to marketing authorisation. Sotrovimab is included in the European Commission’s portfolio of promising candidate therapies as part of its COVID-19 Therapeutics Strategy. In addition, the documentation to support the forthcoming marketing authorisation application for sotrovimab is under rolling regulatory review with the EMA. In June, the companies announced confirmatory full results for the Phase 3 COMET-ICE trial, which resulted in a 79% reduction (adjusted relative risk reduction) (p<0.001) in hospitalisations for more than 24 hours or death due to any cause by Day 29 compared to placebo, meeting the primary endpoint of the trial.
GSK and Vir have secured supply agreements with multiple governments around the world and will continue those efforts as the pandemic continues to evolve. In May 2021, sotrovimab was granted Emergency Use Authorisation (EUA) by the U.S. Food and Drug Administration (FDA) for the treatment of mild-to moderate COVID-19 in high risk patients. GSK and Vir have announced plans to submit a Biologics License Application (BLA) to the U.S. FDA in the second half of 2021. Sotrovimab has also been authorised for emergency use in Bahrain, Kuwait, Qatar, Singapore and United Arab Emirates.
GSK and Vir are committed to ongoing evaluation of sotrovimab as the COVID-19 landscape continues to evolve at different rates across the globe and new variants of concern and interest emerge. Updated in vitro data, published in bioRxiv, demonstrate that sotrovimab retains activity against currently circulating variants of concern and interest of the SARS-CoV-2 virus including Alpha (B.1.1.7), Beta (B.1.351), Delta (B.1.617.2), Epsilon (B.1.427/B.1.429), Gamma (P.1), Iota (B.1.526), Kappa (B.1.617.1), and Lambda (C.37), as well as new variants from Bristol (B.1.1.7+E484K) and Cameroon (B.1.619), which encodes both N440K and E484K mutations that may lead to reduced activity for other neutralising monoclonal antibodies against the SARS-CoV-2 virus. GSK and Vir are continuing to evaluate the ability of sotrovimab to maintain activity against new and emerging variants through in vitro studies. The clinical impact of these in vitro variant data is not yet known.
About the COMET-ICE Study
The multi-centre, double-blind, placebo-controlled, Phase 3 COMET-ICE trial investigated intravenous
(IV) infusion of sotrovimab in adults with mild-to-moderate COVID-19 at high risk of progression to severe disease. In March 2021, an Independent Data Monitoring Committee recommended that the COMET-ICE trial be stopped for enrolment due to evidence of profound efficacy and is continuing to follow trial participants for 24 weeks. Interim data results have been shared with regulatory authorities and formed the basis of the positive scientific opinion reached by the EMA’s CHMP, under Article 5(3) of Regulation 726/2004.
This ongoing trial evaluated the safety and efficacy of a single IV infusion of sotrovimab (500 mg) or placebo in non-hospitalised participants globally. The primary efficacy endpoint was the proportion of patients who have progression of COVID-19 as defined by the need for hospitalisation for greater than 24 hours for acute management of any illness or death from any cause.
The final COMET-ICE trial results in the full study population of 1,057 participants demonstrated a 79% reduction (adjusted relative risk reduction) (p<0.001) in hospitalisation for more than 24 hours or death due to any cause by Day 29 compared to placebo, meeting the primary endpoint of the trial. The number of patients who were hospitalised for >24 hours for acute management of any illness or death from any cause at Day 29 was six patients in the sotrovimab arm (1%), versus 30 patients in the placebo arm (6%). In the sotrovimab arm, it is possible that half of those patients who were hospitalised were for reasons other than progression of COVID-19 (e.g., small bowel obstruction, lung cancer and diabetic foot ulcer); this was not the case for patients in the placebo arm.
In the safety analysis, 1,037 participants were followed through at least 29 days. The most common adverse events observed in the sotrovimab treatment group in COMET-ICE were rash (1%) and diarrhoea (2%), all of which were Grade 1 (mild) or Grade 2 (moderate). No other treatment-emergent adverse events were reported at a higher rate with sotrovimab compared to placebo.
About the Sotrovimab Clinical Development Programme
In addition to the COMET-ICE trial, the full COMET clinical development programme for sotrovimab includes:
- COMET-PEAK, a pharmacokinetic trial in outpatients with mild-to-moderate COVID-19 investigating intramuscular (IM) administration of sotrovimab, is near completion and initial data are expected in the second half of 2021.
- COMET-TAIL has been initiated. This is a Phase 3 trial evaluating the role of IM-administered sotrovimab for the early treatment of mild-to-moderate COVID-19 in high-risk non-hospitalised adult and paediatric patients (12 years of age and older). Data are anticipated in the first half of 2022.
The companies also plan to investigate the use of sotrovimab in uninfected immunocompromised adults to determine whether sotrovimab can prevent symptomatic COVID-19 infection.
About Sotrovimab
Sotrovimab is an investigational SARS-CoV-2 monoclonal antibody. Preclinical data suggest it has the potential to both block viral entry into healthy cells and clear infected cells. The antibody binds to an epitope on SARS-CoV-2 that is shared with SARS-CoV-1 (the virus that causes SARS), indicating that the epitope is highly conserved, which may make it more difficult for resistance to develop. Sotrovimab, which incorporates Xencor’s Xtend™ technology, also has been designed to achieve high concentration in the lungs to ensure optimal penetration into airway tissues affected by SARS-CoV-2 and to have an extended half-life.
Important Information about Sotrovimab in Europe
For more information on the EMA positive scientific opinion, please review the EU Conditions of Use.
All side effects have been mild or moderate. Healthcare professionals should look out for side effects and take appropriate action.
Sotrovimab in the United States
Healthcare providers in the U.S. should review the Fact Sheets for information on the authorized use of sotrovimab and mandatory requirements of the EUA.
Sotrovimab has been authorized by the U.S. FDA for the emergency use described below. Sotrovimab is not FDA-approved for this use.
Sotrovimab is authorized only for the duration of the declaration that circumstances exist justifying the emergency use of sotrovimab under section 564(b)(1) of the Act, 21 U.S.C. § 360bbb-3(b)(1), unless the authorization is terminated or revoked sooner.
Authorized Use
The U.S. Food and Drug Administration (FDA) has issued an Emergency Use Authorization (EUA) to permit the emergency use of the unapproved product sotrovimab for the treatment of mild-to-moderate coronavirus disease 2019 (COVID-19) in adults and pediatric patients (12 years of age and older weighing at least 40 kg) with positive results of direct SARS-CoV-2 viral testing, and who are at high risk for progression to severe COVID-19, including hospitalization or death.
About the Vir and GSK Collaboration
In April 2020, Vir and GSK entered into a collaboration to research and develop solutions for coronaviruses, including SARS-CoV-2, the virus that causes COVID-19. The collaboration uses Vir’s proprietary monoclonal antibody platform technology to accelerate existing and identify new anti-viral antibodies that could be used as therapeutic or preventive options to help address the current COVID-19 pandemic and future outbreaks. The companies will leverage GSK’s expertise in functional genomics and combine their capabilities in CRISPR screening and artificial intelligence to identify anti-coronavirus compounds that target cellular host genes. They will also apply their combined expertise to research SARS-CoV-2 and other coronavirus vaccines.
GSK is a science-led global healthcare company. For further information please visit www.gsk.com/aboutus.
GSK, Vir Bio ink joint procurement agreement with EC for COVID-19 treatment, sotrovimab
Jul. 28, 2021 6:51 AM ET GlaxoSmithKline plc (GSK), VIR GlaxoSmithKline plc (GSK)Vir Biotechnology, Inc. (VIR)
By: Mamta Mayani, SA News Editor
- GlaxoSmithKline (NYSE:GSK) and Vir Biotechnology (NASDAQ:VIR) have signed a Joint Procurement Agreement with the European Commission (EC) to supply up to 220,000 doses of sotrovimab, an investigational single dose SARS-CoV-2 monoclonal antibody for the treatment of adults and adolescents with COVID-19 who do not require oxygen supplementation and at risk of progressing to severe COVID-19.
- https://seekingalpha.com/news/3720289-gsk-vir-bio-ink-joint-procurement-agreement-with-ec-for-covid-19-treatment-sotrovimab
- https://seekingalpha.com/symbol/GSK
- https://seekingalpha.com/symbol/VIR
OPDIVO® (nivolumab)
Bristol Myers Squibb Receives European Commission Approval for Opdivo (nivolumab) as Adjuvant Treatment for Esophageal or Gastroesophageal Junction Cancer Patients with Residual Pathologic Disease Following Chemoradiotherapy
07/30/2021CATEGORY:
Approval is based on Phase 3 results from the CheckMate -577 trial
Opdivo is now the first and only adjuvant therapy approved in this setting in the European Union
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced that the European Commission (EC) has approved Opdivo (nivolumab) for the adjuvant treatment of adult patients with esophageal or gastroesophageal junction (GEJ) cancer who have residual pathologic disease following prior neoadjuvant chemoradiotherapy (CRT). The EC’s decision is based on results from the Phase 3 CheckMate -577 trial, which demonstrated that treatment with Opdivo following neoadjuvant CRT and complete surgical resection doubled the primary endpoint of disease-free survival (DFS) compared to placebo in the all-randomized population. The safety profile of Opdivo was consistent with previously reported studies.
Results from CheckMate -577 were presented at the European Society for Medical Oncology (ESMO) Virtual Congress in September 2020 and at the American Society of Clinical Oncology (ASCO) Annual Meeting in June 2021.
“We have demonstrated that the use of immunotherapy in earlier stages of cancer has the potential to prevent recurrence for certain patients,” said Ian M. Waxman, M.D., development lead, gastrointestinal cancers, Bristol Myers Squibb. “BMS was the first company to bring checkpoint inhibitors into the adjuvant setting for the treatment of patients with melanoma, and we are pleased to be the first to bring adjuvant therapy to patients in the EU with esophageal or gastroesophageal junction cancers who continue to face a high unmet need.”
The EC decision allows for the use of Opdivo for the adjuvant treatment of adult patients with esophageal or GEJ cancer who have residual pathologic disease following prior neoadjuvant CRT in the 27 member states of the European Union, as well as Iceland, Liechtenstein, and Norway. Opdivo also received approval from the U.S. Food and Drug Administration (FDA) in May 2021 for the adjuvant treatment of completely resected esophageal or GEJ cancer with residual pathologic disease in patients who have received CRT.
About CheckMate -577
CheckMate -577 is a Phase 3 randomized, multi-center, double-blind study evaluating Opdivo as an adjuvant therapy in patients with resected esophageal or gastroesophageal junction (GEJ) cancer who have received neoadjuvant chemoradiotherapy (CRT) and have not achieved a pathological complete response. The primary endpoint of the trial is disease-free survival (DFS) and the secondary endpoint is overall survival (OS). Following neoadjuvant CRT and complete tumor surgical resection (also known as trimodality therapy), a total of 794 patients were randomized to receive placebo (n=262) or Opdivo (n=532) 240 mg by intravenous infusion every two weeks for 16 weeks followed by placebo or Opdivo 480 mg every four weeks until disease recurrence, unacceptable toxicity or withdrawal of consent, with a maximum of one-year total treatment duration. Follow-up for OS is ongoing.
About Opdivo
Opdivo is a programmed death-1 (PD-1) immune checkpoint inhibitor that is designed to uniquely harness the body’s own immune system to help restore anti-tumor immune response. By harnessing the body’s own immune system to fight cancer, Opdivo has become an important treatment option across multiple cancers.
Opdivo’s leading global development program is based on Bristol Myers Squibb’s scientific expertise in the field of Immuno-Oncology, and includes a broad range of clinical trials across all phases, including Phase 3, in a variety of tumor types. To date, the Opdivo clinical development program has treated more than 35,000 patients. The Opdivo trials have contributed to gaining a deeper understanding of the potential role of biomarkers in patient care, particularly regarding how patients may benefit from Opdivo across the continuum of PD-L1 expression.
In July 2014, Opdivo was the first PD-1 immune checkpoint inhibitor to receive regulatory approval anywhere in the world. Opdivo is currently approved in more than 65 countries, including the United States, the European Union, Japan and China. In October 2015, the Company’s Opdivo and Yervoy combination regimen was the first Immuno-Oncology combination to receive regulatory approval for the treatment of metastatic melanoma and is currently approved in more than 50 countries, including the United States and the European Union.
INDICATIONS
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express PD-L1 (≥1%) as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab) and 2 cycles of platinum-doublet chemotherapy, is indicated for the first-line treatment of adult patients with metastatic or recurrent non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab) is indicated for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving OPDIVO.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with unresectable malignant pleural mesothelioma (MPM).
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of patients with intermediate or poor risk advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab), in combination with cabozantinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab) is indicated for the treatment of patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy.
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with classical Hodgkin lymphoma (cHL) that has relapsed or progressed after autologous hematopoietic stem cell transplantation (HSCT) and brentuximab vedotin or after 3 or more lines of systemic therapy that includes autologous HSCT. This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab) is indicated for the treatment of patients with recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN) with disease progression on or after platinum-based therapy.
OPDIVO® (nivolumab) is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of adult and pediatric (12 years and older) patients with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection.
OPDIVO® (nivolumab) is indicated for the treatment of patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) after prior fluoropyrimidine- and platinum-based chemotherapy.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of completely resected esophageal or gastroesophageal junction cancer with residual pathologic disease in patients who have received neoadjuvant chemoradiotherapy (CRT).
OPDIVO® (nivolumab), in combination with fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the treatment of patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma.
Please see US Full Prescribing Information for OPDIVO and YERVOY
For more information about Bristol Myers Squibb, visit us at BMS.com
Bristol Myers Squibb wins European approval for Opdivo in two GIT cancers
Jul. 30, 2021 7:31 AM ET
Bristol-Myers Squibb Company (BMY)Bristol-Myers Squibb Company (BMY)
By: Dulan Lokuwithana, SA News Editor3 Comments
- Bristol Myers Squibb (BMY -0.8%) says European Commission (EC) has approved Opdivo (nivolumab) for two cancer types in the gastrointestinal tract (GIT).
- https://seekingalpha.com/news/3722247-bristol-myers-squibb-wins-european-approval-for-opdivo-in-two-git-cancers
- https://seekingalpha.com/symbol/BMY
- https://www.bms.com/
KEYTRUDA® (pembrolizumab)
FDA Approves KEYTRUDA® (pembrolizumab) for Treatment of Patients With High-Risk Early-Stage Triple-Negative Breast Cancer in Combination With Chemotherapy as Neoadjuvant Treatment, Then Continued as Single Agent as Adjuvant Treatment After Surgery
July 27, 2021 6:30 am ET
This KEYTRUDA Combination Is the First Immunotherapy Regimen Approved for High-Risk Early-Stage Triple-Negative Breast Cancer (TNBC)
KEYTRUDA Is Now Approved in the US for 30 Indications
KENILWORTH, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced that the U.S. Food and Drug Administration (FDA) has approved KEYTRUDA, Merck’s anti-PD-1 therapy, for the treatment of patients with high-risk early-stage triple-negative breast cancer (TNBC) in combination with chemotherapy as neoadjuvant treatment and then continued as a single agent as adjuvant treatment after surgery, based on the Phase 3 KEYNOTE-522 trial. TNBC is an aggressive type of breast cancer with an increased risk for disease recurrence. KEYNOTE-522 showed that KEYTRUDA in combination with chemotherapy (carboplatin and paclitaxel, followed by doxorubicin or epirubicin and cyclophosphamide) before surgery and continued as a single agent after surgery significantly prolonged event-free survival (EFS) versus the same neoadjuvant chemotherapy regimens alone in patients with previously untreated stage II or stage III TNBC – there was a 37% reduction in the risk of disease progression that precluded definitive surgery, a local/distant recurrence, a second primary cancer, or death from any cause (HR=0.63 [95% CI, 0.48-0.82]; p=0.00031). With this approval, KEYTRUDA is now approved in the U.S. for 30 indications.
“Even when TNBC is diagnosed early, 30-40% of patients will suffer cancer recurrence after standard neoadjuvant chemotherapy and surgery,” said Dr. Joyce O’Shaughnessy, chair of Breast Cancer Research, Baylor University Medical Center, Texas Oncology, U.S. Oncology, Dallas, Texas. “Therefore, there is a high unmet need for new treatment options. Today’s approval is very welcome news and has the potential to change the treatment paradigm by now including an immunotherapy as part of the regimen for patients with high-risk early-stage TNBC.”
Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue and can affect more than one body system simultaneously. Immune-mediated adverse reactions can occur at any time during or after treatment with KEYTRUDA, including pneumonitis, colitis, hepatitis, endocrinopathies, nephritis, dermatologic reactions, solid organ transplant rejection, and complications of allogeneic hematopoietic stem cell transplantation. Important immune-mediated adverse reactions listed here may not include all possible severe and fatal immune-mediated adverse reactions. Early identification and management of immune-mediated adverse reactions are essential to ensure safe use of KEYTRUDA. Based on the severity of the adverse reaction, KEYTRUDA should be withheld or permanently discontinued and corticosteroids administered if appropriate. KEYTRUDA can also cause severe or life-threatening infusion-related reactions. Based on its mechanism of action, KEYTRUDA can cause fetal harm when administered to a pregnant woman. For more information, see “Selected Important Safety Information” below.
“Triple-negative is a difficult-to-treat type of breast cancer that unfortunately is more common in the U.S. in younger women and in Black women,” said Dr. Vicki Goodman, vice president, clinical research, Merck Research Laboratories. “We are proud to offer a new treatment option for patients faced with this challenging cancer. This neoadjuvant and adjuvant combination with KEYTRUDA is the first immunotherapy regimen to be approved in high-risk early-stage TNBC, marking a meaningful milestone for the breast cancer community.”
Data Supporting the Approval
The approval was based on data from KEYNOTE-522 (ClinicalTrials.gov, NCT03036488), a randomized, multicenter, double-blind, placebo-controlled trial conducted in 1,174 patients with newly diagnosed previously untreated high-risk early-stage TNBC (tumor size >1 centimeter [cm] but ≤2 cm in diameter with nodal involvement or tumor size >2 cm in diameter regardless of nodal involvement). Patients were enrolled regardless of tumor programmed death ligand 1 (PD‑L1) expression. Patients with active autoimmune disease that required systemic therapy within two years of treatment or a medical condition that required immunosuppression were ineligible. Randomization was stratified by nodal status (positive vs. negative), tumor size (T1/T2 vs. T3/T4) and choice of carboplatin (dosed every three weeks vs. weekly). Patients were randomized (2:1) to one of the following two treatment arms; all study mediations were administered intravenously:
- Arm 1:
- Four cycles of preoperative KEYTRUDA 200 mg every three weeks on Day 1 of Cycles 1-4 of treatment regimen in combination with carboplatin Area Under Curve (AUC) 5 mg/mL/min every three weeks on Day 1 of Cycles 1-4 of treatment regimen or AUC 1.5 mg/mL/min every week on Days 1, 8, and 15 of Cycles 1-4 of treatment regimen plus paclitaxel 80 mg/m2 every week on Days 1, 8, and 15 of Cycles 1-4 of treatment regimen
- Followed by four additional cycles of preoperative KEYTRUDA 200 mg every three weeks on Day 1 of Cycles 5-8 of treatment regimen in combination with either doxorubicin 60 mg/m2or epirubicin 90 mg/m2 every three weeks on Day 1 of Cycles 5-8 of treatment regimen plus cyclophosphamide 600 mg/m2 every three weeks on Day 1 of Cycles 5-8 of treatment regimen
- Following surgery, nine cycles of KEYTRUDA 200 mg every three weeks were administered.
- Arm 2:
- Four cycles of preoperative placebo every three weeks on Day 1 of Cycles 1-4 of treatment regimen in combination with carboplatin AUC 5 mg/mL/min every three weeks on Day 1 of Cycles 1-4 of treatment regimen or AUC 1.5 mg/mL/min every week on Days 1, 8, and 15 of Cycles 1-4 of treatment regimen plus paclitaxel 80 mg/m2 every week on Days 1, 8, and 15 of Cycles 1-4 of treatment regimen
- Followed by four cycles of preoperative placebo every three weeks on Day 1 of Cycles 5-8 of treatment regimen in combination with either doxorubicin 60 mg/m2or epirubicin 90 mg/m2 every three weeks on Day 1 of Cycles 5-8 of treatment regimen plus cyclophosphamide 600 mg/m2 every three weeks on Day 1 of Cycles 5-8 of treatment regimen
- Following surgery, nine cycles of placebo every three weeks were administered.
The main efficacy outcomes were pathological complete response (pCR) rate and EFS. Pathological complete response rate was defined as absence of invasive cancer in the breast and lymph nodes (ypT0/Tis ypN0) and was assessed by the blinded local pathologist at the time of definitive surgery. Event-free survival was defined as the time from randomization to the first occurrence of any of the following events: progression of disease that precludes definitive surgery, local or distant recurrence, secondary primary malignancy, or death due to any cause. An additional efficacy outcome was overall survival (OS).
The study population characteristics were: median age of 49 years (range, 22 to 80), 11% age 65 or older; 99.9% female; 64% White, 20% Asian, 4.5% Black, and 1.8% American Indian or Alaska Native; 87% Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) of 0, and 13% ECOG PS of 1; 56% were pre-menopausal, and 44% were post-menopausal; 7% were primary Tumor 1 (T1), 68% T2, 19% T3, and 7% T4; 49% were nodal involvement 0 (N0), 40% N1, 11% N2, and 0.2% N3; 75% of patients were overall stage II, and 25% were stage III.
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 1,500 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.
Selected KEYTRUDA® (pembrolizumab) Indications in the U.S.
Melanoma
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.
KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.
Non-Small Cell Lung Cancer
KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) ≥1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is:
- stage III where patients are not candidates for surgical resection or definitive chemoradiation, or
- metastatic.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS ≥1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.
Head and Neck Squamous Cell Cancer
KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS ≥1)] as determined by an FDA-approved test.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.
Classical Hodgkin Lymphoma
KEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).
KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.
Primary Mediastinal Large B-Cell Lymphoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.
Urothelial Carcinoma
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 (CPS ≥10), as determined by an FDA-approved test, or in patients who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
KEYTRUDA is indicated for the treatment of patients with locally advanced or mUC who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.
Microsatellite Instability-High or Mismatch Repair Deficient Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options.
This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.
Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).
Gastric Cancer
KEYTRUDA, in combination with trastuzumab, fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of patients with locally advanced unresectable or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Esophageal Cancer
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic esophageal or GEJ (tumors with epicenter 1 to 5 centimeters above the GEJ) carcinoma that is not amenable to surgical resection or definitive chemoradiation either:
- in combination with platinum- and fluoropyrimidine-based chemotherapy, or
- as a single agent after one or more prior lines of systemic therapy for patients with tumors of squamous cell histology that express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
Cervical Cancer
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Hepatocellular Carcinoma
KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Merkel Cell Carcinoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Renal Cell Carcinoma
KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma.
Tumor Mutational Burden-High Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [≥10 mutations/megabase] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.
Cutaneous Squamous Cell Carcinoma
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) or locally advanced cSCC that is not curable by surgery or radiation.
Triple-Negative Breast Cancer
KEYTRUDA is indicated for the treatment of patients with high-risk early-stage TNBC in combination with chemotherapy as neoadjuvant treatment, and then continued as a single agent as adjuvant treatment after surgery.
KEYTRUDA, in combination with chemotherapy, is indicated for the treatment of patients with locally recurrent unresectable or metastatic TNBC whose tumors express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
More information is available by calling 855-257-3932 or visiting www.merckaccessprogram-keytruda.com.
About Merck’s Patient Support Program for KEYTRUDA
Merck is committed to helping provide patients and their caregivers support throughout their treatment with KEYTRUDA. The KEY+YOU Patient Support Program provides a range of resources and support. For further information and to sign up, eligible patients may call 85-KEYTRUDA (855-398-7832) or visit www.keytruda.com.
For more information, visit www.merck.com
Please see Prescribing Information for KEYTRUDA (pembrolizumab) at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_pi.pdf and Medication Guide for KEYTRUDA at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_mg.pdf.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210727005394/en/
Source: Merck & Co., Inc.
FDA OKs Merck's Keytruda/Chemotherapy combo in triple-negative breast cancer
Jul. 27, 2021 6:48 AM ET Merck & Co., Inc. (MRK) Merck & Co., Inc. (MRK) By: Mamta Mayani, SA News Editor
- Merck (NYSE:MRK) announces that the FDA has approved KEYTRUDA, an anti-PD-1 therapy, for the treatment of patients with high-risk early-stage triple-negative breast cancer (TNBC) in combination with chemotherapy as neoadjuvant treatment and then continued as a single agent as adjuvant treatment after surgery, based on the Phase 3 KEYNOTE-522 trial.
- https://seekingalpha.com/news/3719482-fda-oks-mercks-keytrudachemotherapy-combo-in-triple-negative-breast-cancer
- https://seekingalpha.com/symbol/MRK
Merck Announces Phase 3 KEYNOTE-355 Trial Met Primary Endpoint of Overall Survival (OS) in Patients with Metastatic Triple-Negative Breast Cancer Whose Tumors Expressed PD-L1 (CPS ≥10)
July 27, 2021 6:45 am ET
KEYTRUDA Is the First Anti-PD-1 Therapy in Combination With Chemotherapy to Show Statistically Significant OS for This Patient Population
KENILWORTH, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced positive overall survival (OS) results from the pivotal Phase 3 KEYNOTE-355 trial evaluating KEYTRUDA, Merck’s anti-PD-1 therapy, in combination with chemotherapy for the treatment of patients with metastatic triple-negative breast cancer (mTNBC). Findings from the final analysis show first-line treatment with KEYTRUDA in combination with chemotherapy (nab-paclitaxel, paclitaxel or gemcitabine/carboplatin) demonstrated a statistically significant and clinically meaningful improvement in OS compared with chemotherapy alone in patients with mTNBC whose tumors expressed PD-L1 (Combined Positive Score [CPS] ≥10). No new safety signals were identified. These OS results will be presented at an upcoming medical meeting and submitted to regulatory authorities.
biotecMAX™ April/May/June/July 2021 Insight
Venetoclax (VENCLEXTA®)
July 21, 2021
Venetoclax (VENCLEXTA®) Granted US FDA Breakthrough Therapy Designation (BTD) in Higher Risk Myelodysplastic Syndrome (MDS)
- This is the sixth BTD granted for venetoclax as AbbVie continues to show ongoing commitment across blood cancers including tough to treat myeloid malignancies
NORTH CHICAGO, Ill., July 21, 2021 /PRNewswire/ -- AbbVie (NYSE: ABBV) announced today that the U.S. Food and Drug Administration (FDA) granted a Breakthrough Therapy Designation (BTD) to venetoclax (VENCLEXTA®) in combination with azacitidine for the potential treatment of adult patients with previously untreated intermediate-, high- and very high-risk myelodysplastic syndromes (MDS) based on revised International Prognostic Scoring System (IPSS-R). A BTD is intended to expedite the development and review of medications to treat a serious medical condition and is granted when preliminary clinical evidence indicates the investigational therapy may demonstrate substantial improvement over existing therapies.1 This marks the sixth BTD granted to venetoclax.
MDS are a group of diverse bone marrow disorders in which the bone marrow does not produce enough healthy blood cells.2 Patients living with MDS may experience symptoms such as infection, anemia, spontaneous bleeding, and easy bruising. Roughly 10,000 patients in the US are diagnosed with MDS each year3 and around 30 percent of those patients will progress to acute myeloid leukemia (AML). Although MDS can occur at any age, it is most commonly found in patients aged 60 and older.4
"MDS is a devastating diagnosis - not only does it have the potential to greatly impact patients' quality of life, but 30 percent of patients will also progress to AML," said Jalaja Potluri, executive medical director, oncology, AbbVie. "This Breakthrough Therapy Designation underscores the need for more treatment options for these patients and the utility of venetoclax to potentially treat different forms of blood cancer."
This designation is supported by data from the Phase 1b M15-531 study. In addition to the Phase Ib M15-531 study, venetoclax is being investigated in combination with azacitidine for the treatment of MDS in the Phase Ib M15-522 study in patients with relapsed or refractory disease, and the Phase 3 randomized VERONA study in patients with newly diagnosed higher-risk MDS.
VENCLEXTA is being developed by AbbVie and Roche. It is jointly commercialized by AbbVie and Genentech, a member of the Roche Group, in the U.S. and by AbbVie outside of the U.S.
About VENCLEXTA® (venetoclax)
VENCLEXTA®/VENCLYXTO® (venetoclax) is a first-in-class medicine that selectively binds and inhibits the B-cell lymphoma-2 (BCL-2) protein. In some blood cancers, BCL-2 prevents cancer cells from undergoing their natural death or self-destruction process, called apoptosis. VENCLXEXTA/VENCLYXTO targets the BCL-2 protein and works to help restore the process of apoptosis.
VENCLEXTA/VENCLYXTO is being developed by AbbVie and Roche. It is jointly commercialized by AbbVie and Genentech, a member of the Roche Group, in the U.S. and by AbbVie outside of the U.S. Together, the companies are committed to BCL-2 research and to studying venetoclax in clinical trials across several blood cancers. Venetoclax is approved in more than 80 countries, including the U.S.
Uses of VENCLEXTA® (venetoclax) in US
VENCLEXTA is a prescription medicine used:
- to treat adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).
- in combination with azacitidine, or decitabine, or low-dose cytarabine to treat adults with newly diagnosed acute myeloid leukemia (AML) who:
- are 75 years of age or older, or
- have other medical conditions that prevent the use of standard chemotherapy.
It is not known if VENCLEXTA is safe and effective in children.
The full U.S. prescribing information, including Medication Guide, for VENCLEXTA® can be found here.
For more information about AbbVie, please visit us at www.abbvie.com.
Abbvie's Venclexta granted Breakthrough Therapy designation for myelodysplastic syndromes
Jul. 21, 2021 8:58 AM ETRoche Holding AG (RHHBY), ABBVBy: Jonathan M Block, SA News Editor2 Comments
- The FDA has granted Breakthrough Therapy designation to Abbvie's (NYSE:ABBV) Venclexta (venetoclax) in combination with azacitidine as a potential treatment for myelodysplastic syndromes ("MDS").
- https://seekingalpha.com/news/3717358-abbvie-venclexta-granted-breakthrough-therapy-designation-for-myelodysplastic-syndromes-roche
- https://seekingalpha.com/symbol/ABBV
- https://seekingalpha.com/symbol/RHHBY
- The first-in-class medicine is being jointly developed for MDS by Abbvie and Roche (OTCQX:RHHBY).
- https://www.venclexta.com/
Shingrix (Zoster Vaccine Recombinant, Adjuvanted)
26 July 2021
Shingrix approved in the US for prevention of shingles in immunocompromised adults
For media and investors only
Issued: Philadelphia, London
GlaxoSmithKline plc today announced that the US Food and Drug Administration (FDA) has approved Shingrix (Zoster Vaccine Recombinant, Adjuvanted) for the prevention of shingles (herpes zoster) in adults aged 18 years and older who are or who will be at increased risk of shingles due to immunodeficiency or immunosuppression caused by known disease or therapy. Immunocompromised individuals are at greater risk of shingles and associated complications than immunocompetent individuals.
Shingrix, a non-live, recombinant sub-unit adjuvanted vaccine, given intramuscularly in two doses, was initially approved by FDA in 2017 for the prevention of shingles in adults 50 years of age or older. Shingrix is not indicated for prevention of primary varicella infection (chickenpox). The approval for this new population expands the number of people who can be protected against shingles by Shingrix.
This approval for a new population was based on clinical studies examining the safety and efficacy of Shingrix in adults (=18 years of age) who had undergone an autologous hematopoietic stem cell transplant (auHSCT) and those undergoing treatment for hematological malignancies (post-hoc analysis). Further safety and immunogenicity data were generated in adults who were, or were anticipated to be, immunodeficient or immunosuppressed due to known disease or therapy, including patients with HIV, solid tumors, and renal transplants.[1],[2],[3],[4],[5],[6]
“In addition to this new patient population, there are more than 100 million adults 50 years and older in the US already recommended to receive Shingrix,” said Breuer. “We know many of these individuals missed recommended vaccines during the pandemic and we hope this can be a reminder to them to catch up on all their immunizations, including Shingrix.”
According to recently published report from Avalere Health and supported by GSK, more than 17 million doses of recommended vaccines, including Shingrix, were missed by adults during the pandemic.
Shingrix is the first shingles vaccine indicated for use in those who are at increased risk of the disease due to being immunodeficient or immunosuppressed due to disease or therapy. It combines a non-live antigen, to trigger a targeted immune response, with a specifically designed adjuvant system to generate a Varicella Zoster Virus (VZV)-specific immune response. For immunocompetent adults, Shingrix is intended to be administered in two doses, 2 to 6 months apart.
About Shingrix
Shingrix is a non-live, recombinant subunit vaccine approved in the United States, Canada, EU, UK, China, Japan, Hong Kong, Australia, New Zealand, and Singapore to help prevent shingles (herpes zoster) in people aged 50 years or older. It combines an antigen, glycoprotein E, and an adjuvant system, AS01B, intended to generate a Varicella Zoster Virus (VZV)-specific immune response immune response that can help overcome the decline in immunity as people age.
Shingrix was previously approved by the European Commission (EC) and in the UK for prevention of shingles and post-herpetic neuralgia (PHN) in adults 18 years of age or older at increased risk of shingles and granted marketing authorization on August 25, 2020.
The updated US Prescribing Information will be available soon at www.gskpro.com.
For further information please visit www.gsk.com/about-us.
https://www.shingrix.com/index.html
FDA approves GlaxoSmithKline's Shingrix to treat certain patients with shingles
Jul. 26, 2021 9:55 AM ETGlaxoSmithKline plc (GSK)GlaxoSmithKline plc (GSK)By: Aakash Babu, SA News Editor
syahrir maulana/iStock via Getty Images
- GlaxoSmithKline (NYSE:GSK) announces that the U.S. FDA has approved Shingrix for the prevention of shingles in certain adult patients at increased risk of the condition.
- https://seekingalpha.com/symbol/GSK
- https://seekingalpha.com/news/3719039-fda-approves-glaxosmithklines-shingrix-to-treat-certain-patients-with-shingles
- https://www.shingrix.com/index.html
- https://www.gsk.com/en-gb/
BRUKINSA® (Zanubrutinib)
BeiGene Announces Approval in Canada of BRUKINSA® (Zanubrutinib) for the Treatment of Patients with Mantle Cell Lymphoma
Jul 26, 2021 7:00 AM
Second approval for BTK Inhibitor BRUKINSA in Canada
CAMBRIDGE, Mass. & BEIJING--(BUSINESS WIRE)-- BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a global, science-driven biotechnology company focused on developing innovative and affordable medicines to improve treatment outcomes and access for patients worldwide, today announced that BRUKINSA® (zanubrutinib) has been approved by Health Canada for the treatment of mantle cell lymphoma (MCL) in adult patients who have received at least one prior therapy. This is the second approval for BRUKINSA in Canada, following its initial approval in March 2021 for adult patients with Waldenström’s macroglobulinemia (WM).
“BRUKINSA was specifically designed by BeiGene scientists to provide deep and durable responses for patients with hematologic malignancies, while also reducing the frequency of certain off-target side effects seen with first-generation BTK inhibitors. Today’s approval in Canada for patients with MCL follows its approval for patients with WM earlier in the year, where Canada was the first country to grant approval for BRUKINSA in patients with WM,” said Jane Huang, M.D., Chief Medical Officer, Hematology, BeiGene. “We are excited to continue working with patients and physicians in Canada, as well as in other markets, as part of our broad clinical development program for BRUKINSA investigating eight indications in over 25 clinical trials, with more than 3,100 patients participating.”
“For many patients treated with previously approved BTK inhibitors for MCL, adequate responses are not achievable, or they can be forced to discontinue treatment early due to side effects. Today, we have a new option for our adult patients in Canada who have received one prior systemic or targeted therapy and are living with MCL, an aggressive blood cancer that is often diagnosed at a more advanced stage,” said John Kuruvilla, M.D., FRCPC, Associate Professor of Medicine at the University of Toronto and Clinical Investigator at the Princess Margaret Cancer Centre in Toronto.
“The approval of BRUKINSA as a second line therapy is positive news for patients undergoing treatment for mantle cell lymphoma,” said Antonella Rizza, Chief Executive Officer at Lymphoma Canada. “Expanded treatment options have the potential to transform the patient experience and provide hope to people living with a mantle cell diagnosis.”
The Health Canada approval for BRUKINSA in MCL is based on efficacy results from two single-arm clinical trials. Across both trials, as assessed by independent review committee (IRC) per 2014 Lugano Classification BRUKINSA achieved an overall response rate (ORR) of 84%, defined as the combined rate of complete responses (CRs) and partial responses (PRs).
About BRUKINSA® (zanubrutinib)
BRUKINSA is a small molecule inhibitor of Bruton’s tyrosine kinase (BTK) discovered by BeiGene scientists that is currently being evaluated globally in a broad clinical program as a monotherapy and in combination with other therapies to treat various B-cell malignancies. Because new BTK is continuously synthesized, BRUKINSA was specifically designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity. With differentiated pharmacokinetics compared to other approved BTK inhibitors, BRUKINSA has been demonstrated to inhibit the proliferation of malignant B cells within a number of disease relevant tissues.
To learn more about BeiGene, please visit www.beigene.ca
View source version on businesswire.com: https://www.businesswire.com/news/home/20210726005208/en/
Source: BeiGene, Ltd.
Health Canada approves BeiGene's Brukinsa for mantle cell lymphoma
Jul. 26, 2021 7:35 AM ETBeiGene, Ltd. (BGNE)BeiGene, Ltd. (BGNE)By: Mamta Mayani, SA News Editor
- BeiGene's (NASDAQ:BGNE) BRUKINSA (zanubrutinib) has been approved by Health Canada for the treatment of mantle cell lymphoma (MCL) in adult patients who have received at least one prior therapy.
- https://seekingalpha.com/news/3718917-health-canada-approves-beigenes-brukinsa-for-mantle-cell-lymphoma
- https://seekingalpha.com/symbol/BGNE
- https://www.brukinsa.com/
- https://www.beigene.com/
Ultomiris (ravulizumab)
Ultomiris recommended for approval in the EU by CHMP for children and adolescents with paroxysmal nocturnal haemoglobinuria
PUBLISHED26 July 2021
26 July 2021 07:00 BST
Opinion based on results from Ultomiris Phase III trial that showed an established efficacy and safety profile with reduced treatment burden for children with PNH and their families
Alexion’s Ultomiris (ravulizumab) has been recommended for marketing authorisation in the European Union (EU) for expanded use to include children and adolescents with paroxysmal nocturnal haemoglobinuria (PNH).
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency based its positive opinion on interim results from the Phase III clinical trial in children and adolescents with PNH, which were recently presented during the European Hematology Association 2021 Virtual Congress.
This trial demonstrated that Ultomiris was effective in achieving complete C5 complement inhibition through 26 weeks for the treatment of children and adolescents up to 18 years of age with PNH. Additionally, Ultomiris had no reported treatment-related severe adverse events, and no patients discontinued treatment during the primary evaluation period or experienced breakthrough haemolysis, which can lead to disabling or potentially fatal blood clots.1
The efficacy and safety of Ultomiris in children and adolescents is consistent with the established profile of Ultomiris in clinical trials involving adults with PNH and is representative of the broad PNH patient population seen in the real-world clinical setting.2,3
The CHMP recommended the expanded use of Ultomiris to include children (with a body weight of 10 kg or above) and adolescents with PNH who experience haemolysis with clinical symptom(s) indicative of high disease activity, as well as for individuals who are clinically stable after having been treated with Soliris for at least the past six months.
Ultomiris was first approved in the EU in 2019 for the treatment of adults with PNH and is also approved in the EU for the treatment of adults and children with atypical haemolytic uraemic syndrome (aHUS). In June 2021, the US Food and Drug Administration approved the expanded use of Ultomiris to include children (one month of age and older) and adolescents with PNH, the first and only treatment for this age group in the US.
Ultomiris
Ultomiris (ravulizumab), the first and only long-acting C5 complement inhibitor, offers immediate, complete, and sustained complement inhibition. The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. When activated in an uncontrolled manner, the complement cascade over-responds, leading the body to attack its own healthy cells. Ultomiris is administered intravenously every eight weeks or, for paediatric patients less than 20 kg, every four weeks, following a loading dose. Ultomiris is approved in the US for the treatment of adults and children (one month of age and older) with PNH, as well as in the EU and Japan as a treatment for adults with PNH. It is also approved in the US for aHUS to inhibit complement-mediated thrombotic microangiopathy in adult and paediatric (one month of age and older) patients, in the EU for the treatment of adults and children with a body weight of at least 10 kg with aHUS, as well as in Japan for adults and children with aHUS.
Please visit astrazeneca.com
EMA advisory committee backs Alexion’s Ultomiris for blood disorder in children
Jul. 26, 2021 4:45 AM ET
AstraZeneca PLC (AZN), ALXNAlexion Pharmaceuticals, Inc. (ALXN)AstraZeneca PLC (AZN)
By: Mamta Mayani, SA News Editor
AstraZeneca (NASDAQ:AZN) announces that Alexion’s (NASDAQ:ALXN) Ultomiris (ravulizumab) has been recommended for marketing authorization in the European Union (EU) for expanded use to include children and adolescents with paroxysmal nocturnal haemoglobinuria (PNH).
https://seekingalpha.com/symbol/AZN
https://seekingalpha.com/symbol/ALXN
BYDUREON BCise (exenatide extended-release)
BYDUREON BCise (exenatide extended-release) Approved in the US for the Treatment of Type 2 Diabetes in Pediatric Patients Ages 10 Years and Older
BYDUREON BCise (exenatide extended-release) is the first once-weekly GLP-1 RA treatment option for pediatric patients with type 2 diabetes in the US
July 23, 2021 07:00 AM Eastern Daylight Time
WILMINGTON, Del.--(BUSINESS WIRE)--AstraZeneca’s BYDUREON BCise (exenatide extended-release), once-weekly injectable suspension has been approved in the US for the treatment of type 2 diabetes (T2D); to improve glycemic control in pediatric patients (10 to 17 years) as an adjunct to diet and exercise.
The approval by the US Food and Drug Administration (FDA) is the first regulatory approval for a once-weekly glucagon-like peptide-1 receptor agonist (GLP-1 RA) in this population, supported by the positive results of the BCB114 Phase III trial in youth with T2D between 10 and <18 years of age; which showed on top of standard of care exenatide extended-release significantly improved glycemic control compared to placebo in pediatrics.
This is the first completed trial of a once-weekly GLP-1 RA in a pediatric population with T2D. The approval is an important development in diabetes care for this specific group of patients as the only non-insulin options for adolescents are metformin and liraglutide.
BYDUREON BCise (exenatide extended-release) was first approved in the US in October 2017 as a once-weekly single-dose autoinjector device for adults with T2D whose blood sugar remains uncontrolled on one or more oral medicines in addition to diet and exercise, to improve glycemic control. It was also approved for use in the EU in August 2018.
BCB114 was a 24-week, randomized, double-blind, placebo-controlled Phase III trial with a 28-week open-label extension. Pediatric patients aged 10 to 17 years (N=82) with T2D treated with diet and exercise alone or in combination with a stable dose of oral antidiabetic agents and/or insulin were randomized to receive exenatide extended-release 2 mg or placebo. The primary efficacy endpoint of the Phase III trial was change in glycated hemoglobin A1c (HbA1c) from baseline to week 24. Results demonstrated that patients administered exenatide extended-release achieved a significantly greater mean change in HbA1c from baseline compared to placebo (-0.25%, n=58, baseline A1C 8.13% vs +0.45%, n=24, baseline A1C 8.28%, respectively; p<0.05).
Overall, the adverse reactions observed in this pediatric population were consistent with that observed in the adult population. Safety and effectiveness of exenatide extended-release have not been established in pediatric patients less than 10 years of age.
INDICATION AND LIMITATIONS OF USE
BYDUREON BCise is indicated as an adjunct to diet and exercise to improve glycemic control in adults and pediatric patients aged 10 years and older with type 2 diabetes mellitus
- Not recommended as first-line therapy for patients inadequately controlled on diet and exercise
- Should not be used to treat type 1 diabetes
- Do not coadminister with other exenatide-containing products
- Not studied in patients with a history of pancreatitis. Consider other antidiabetic therapies in patients with a history of pancreatitis
Please click here for Medication Guide, and click here for Full Prescribing Information for BYDUREON BCise.
BCB114 Phase III trial
BCB114 was a Phase III, double-blind, placebo-controlled, randomized, multi-center, parallel trial assessing the safety and efficacy of exenatide extended-release compared to placebo in youth with T2D between 10 and <18 years of age; which showed on top of standard of care exenatide extended-release significantly improved glycemic control compared to placebo in pediatrics. It is the first completed trial of a once-weekly GLP-1 RA in pediatrics with T2D. The trial included 82 children or adolescents treated with diet and exercise alone or in combination with an oral antidiabetic agent (metformin and/or sulfonylurea [SU]) and/or insulin. As clinical studies have demonstrated that once-weekly exenatide extended-release improved glycemic control in adults with T2D, the BCB114 trial was designed to evaluate the effects of exenatide extended-release on glycemic control in pediatrics with T2D.
For more information, please visit www.astrazeneca-us.com
FDA OKs AstraZeneca's BYDUREON BCise in children with type 2 diabetes
Jul. 23, 2021 7:18 AM ETAstraZeneca PLC (AZN)AstraZeneca PLC (AZN)By: Mamta Mayani, SA News Editor
- AstraZeneca’s (NASDAQ:AZN) BYDUREON BCise (exenatide extended-release), once-weekly injectable suspension has been approved in U.S. for the treatment of type 2 diabetes (T2D); to improve glycemic control in pediatric patients (10 to 17 years) as an adjunct to diet anhttps://seekingalpha.com/news/3718492-fda-oks-astrazenecas-bydureon-bcise-in-children-with-type-2-diabetesd exercise.
- https://seekingalpha.com/symbol/AZN
- https://www.bydureon.com/bydureon-bcise.html
SKYSONA™ (elivaldogene autotemcel, Lenti-D™)
bluebird bio Receives EC Approval for SKYSONA™ (elivaldogene autotemcel, Lenti-D™) Gene Therapy for Patients Less Than 18 Years of Age With Early Cerebral Adrenoleukodystrophy (CALD) Without Matched Sibling Donor
SKYSONA is the first and only gene therapy approved in the EU to treat early CALD
CALD is a rare neurodegenerative disease that can lead to progressive, irreversible loss of neurologic function, and death
One-time treatment with SKYSONA has been shown to have a durable effect in improving survival outcomes and preserving neurologic function across pivotal and long-term studies, with the longest follow-up of nearly seven years (82.7 months)
SKYSONA uses the patient’s own blood stem cells and there have been no reports of graft-versus-host disease (GVHD), graft failure or rejection, or transplant-related mortality (TRM) (n=51) across clinical studies to-date
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jul. 21, 2021-- bluebird bio, Inc. (Nasdaq: BLUE) today announced that the European Commission (EC) has granted marketing authorization of SKYSONA™ (elivaldogene autotemcel, Lenti-D™), a one-time gene therapy for the treatment of early cerebral adrenoleukodystrophy (CALD) in patients less than 18 years of age with an ABCD1 genetic mutation, and for whom a human leukocyte antigen (HLA)-matched sibling hematopoietic (blood) stem cell (HSC) donor is not available. SKYSONA is the first one-time gene therapy approved in the European Union (EU) to treat CALD, a rare neurodegenerative disease that occurs in childhood and can rapidly lead to progressive, irreversible loss of neurologic function, and death.
“SKYSONA is the first and only one-time gene therapy approved in the EU for patients with CALD, a devastating neurodegenerative disease, and we are immensely grateful to all who have brought us to this milestone, from the patients and their loved ones, to all study investigators, regulators, the ALD community and of course, the extended bluebird family,” said Andrew Obenshain, president, severe genetic diseases, bluebird bio. “bluebird bio was founded with the mission of developing a therapy to recode CALD on the genetic level, and today’s announcement represents over twenty years of research and development that has laid the groundwork for future gene therapies to be possible.”
SKYSONA is a one-time gene therapy custom-designed to treat the underlying cause of the neurologic condition CALD. SKYSONA uses ex vivo transduction with the Lenti-D lentiviral vector (LVV) to add functional copies of the ABCD1 gene into a patient’s own HSCs. The addition of the functional ABCD1 gene allows patients to produce the ALD protein (ALDP), which is thought to facilitate the breakdown of VLCFAs. The expression of ALDP and effect of SKYSONA is expected to be life-long. The goal of treatment with SKYSONA is to stop the progression of CALD and, consequently, preserve as much neurological function as possible, including the preservation of motor function and communication ability. Importantly, with SKYSONA, there is no need for donor HSCs from another person.
Previously, the only therapeutic option available to CALD patients was transplantation of stem cells from a donor, called allogeneic hematopoietic stem cell transplant (allo-HSCT), which is associated with severe potential complications and mortality that increase in patients without a matched sibling donor (MSD). It is estimated that more than 80% of patients diagnosed with CALD do not have an MSD.
SKYSONA was reviewed as part of the European Medicines Agency’s Priority Medicines scheme (PRIME) and was previously granted Orphan Medicinal Product status. The marketing authorization is valid in all 27 member states of the EU, as well as Norway, Liechtenstein, and Iceland.
Data Supporting Clinical Profile of SKYSONA From Clinical Development Program
ALD-102 and ALD-104
The marketing authorization of SKYSONA is supported by efficacy and safety data from the Phase 2/3 Starbeam study (ALD-102). Additionally, the Phase 3 ALD-104 study (N=19; as of October 2020) is ongoing. All patients who completed ALD-102, as well as those who will complete ALD-104, will be asked to participate in a long-term follow-up study (LTF-304).
The primary efficacy endpoint of the pivotal ALD-102 study was Major Functional Disabilities (MFD)-free survival, measuring the proportion of patients who did not have any of the six MFDs, were alive, did not receive a second allo-HSCT or rescue cell administration, and had not withdrawn or been lost to follow-up at Month 24. In ALD-102, 32 patients have been treated with SKYSONA and, as of October 2020, 30 of 32 patients were evaluable for follow-up at Month 24. As of the data cutoff date, 90% (27/30) of the patients met the Month 24 MFD-free survival endpoint. As previously reported, two patients withdrew from the study at investigator discretion, and one experienced rapid disease progression early in the study, resulting in MFDs and subsequent death.
In ALD-102, 26 of 28 evaluable patients maintained a neurologic function score (NFS) less than or equal to 1 through Month 24, and 24 of those patients had no change in their NFS, which showed maintenance of neurological function in the majority of patients. All patients who completed ALD-102 enrolled for long-term follow-up in the LTF-304 study.
SKYSONA showed a durable effect on MFD-free survival, with most patients (26/27, 96.3%) that enrolled in LTF-304 remaining alive and maintaining their MFD-free status through their last follow-up on study. The median duration of follow-up was 3.2 years (38.6 months; min.: 13.4; max.: 82.7) and 14 patients reached at least their Year 5 follow-up visit. One patient enrolled in LTF-304 but refused further follow-up later.
The treatment regimen, comprising stem cell mobilization and collection, conditioning, and SKYSONA infusion, had a safety/tolerability profile primarily reflective of the known effects of mobilization/apheresis and conditioning.
ALD-103
The efficacy and safety of allo-HSCT in patients with CALD was observed in a contemporaneous comparator study ALD-103 (N=59), which assessed safety and efficacy outcomes of this therapeutic option in boys 17 years of age or younger with CALD. Of the 59 patients, 27 patients matched the ALD-102 population based on disease characteristics. This population was further divided into those who received an allo-HSCT from an MSD (N=10; ALD-103 Efficacy Population with MSD) and those who received an allo-HSCT from an alternative donor source, i.e., not an MSD (N=17; ALD-103 Efficacy Population without MSD). Proportion of MFD-free survival at Month 24 was analyzed in ALD-102 (90% [95% CI: 73.5, 97.9]; 27/30 evaluable patients) and compared to 17 patients treated with allo-HSCT without an MSD in ALD-103 (66.7% [95% CI: 29.9, 92.5]; 6/9 evaluable patients).
The proportion of evaluable patients who experienced either acute (≥ Grade II) or chronic GVHD in ALD-102 vs. ALD-103 by Month 24, was 0 vs. 52%. No patients experienced TRM, a secondary endpoint, at 100 days or 365 days after transplant in ALD-102 and ALD-104. In contrast, 2/59 (3.4%) patients experienced TRM at 100 days and 8/59 (13.6%) patients experienced TRM at 365 days after transplant in the ALD-103 Safety Population.
About SKYSONA (elivaldogene autotemcel, formerly Lenti-D™ gene therapy)
The U.S. Food and Drug Administration (FDA) granted SKYSONA Orphan Drug status, Rare Pediatric Disease designation and Breakthrough Therapy designation for the treatment of CALD. bluebird bio is currently on track to submit the Biologics License Application (BLA) in the U.S. by mid-2021.
In the EU, SKYSONA is approved for the treatment of early CALD in patients less than 18 years of age with an ABCD1 genetic mutation, and for whom an HLA-matched sibling HSC donor is not available. A marketing authorization application for SKYSONA is currently under review by the Medicines and Healthcare products Regulatory Agency (MHRA) in Great Britain.
The Phase 3 ALD-104 study, designed to assess the efficacy and safety of SKYSONA after myeloablative conditioning using busulfan and fludarabine in patients with CALD, is approaching enrollment completion; enrollment in Europe is closed.
The Phase 2/3 Starbeam study (ALD-102) is complete. For more information about our studies, visit: www.bluebirdbio.com/our-science/clinical-trials or clinicaltrials.gov.
Additionally, bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-304) for patients who have been treated with SKYSONA for CALD and completed two years of follow-up in bluebird bio-sponsored studies. Patients treated with SKYSONA in Europe are expected to enroll in the REG-502 Stargazer registry.
For more information, visit bluebirdbio.com.
Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.
SKYSONA and bluebird bio are trademarks of bluebird bio, Inc.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210721005331/en/
Source: bluebird bio, Inc.
bluebird nabs EC approval for gene therapy Skysona for rare neurodegenerative disease
Jul. 21, 2021 8:08 AM ET bluebird bio, Inc. (BLUE)
By: Jonathan M Block, SA News Editor2 Comments
- The European Commission has granted marketing authorization for bluebird bio's (NASDAQ:BLUE) Skysona (elivaldogene autotemcel, Lenti-D), a gene therapy for early cerebral adrenoleukodystrophy in those below the age of 18.
- https://seekingalpha.com/news/3717313-bluebird-nabs-ec-approval-for-gene-therapy-skysona-for-rare-neurodegenerative-disease
- https://seekingalpha.com/symbol/BLUE
- https://www.bluebirdbio.com/our-science/pipeline
- https://www.bluebirdbio.com/
https://www.nasdaq.com/market-activity/stocks/blue/historical
REGEN-COV™ (casirivimab and imdevimab)
JAPAN BECOMES FIRST COUNTRY TO APPROVE REGENERON ANTIBODY COCKTAIL (CASIRIVIMAB AND IMDEVIMAB) FOR THE TREATMENT OF MILD TO MODERATE COVID-19
TARRYTOWN, N.Y., July 20, 2021 /PRNewswire/ -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that Japan's Ministry of Health, Labour and Welfare (MHLW) has approved Regeneron's casirivimab and imdevimab antibody cocktail to treat patients with mild to moderate COVID-19. This marks the first time the antibody cocktail, known as REGEN-COVTM in the U.S. and Ronapreve™ in other countries, has received a full approval to treat COVID-19. Emergency or temporary pandemic use authorizations are currently in place in more than 20 countries, including in the U.S., European Union, India, Switzerland and Canada.
"After a record-speed discovery and development program, we are pleased that our COVID-19 antibody cocktail continues to reach even more people around the globe," said George D. Yancopoulos, M.D., Ph.D., President and Chief Scientific Officer of Regeneron. "Unfortunately, this virus continues to spread despite increasing rates of vaccination, and there will be an important continued need for treatments that remain active against the variants of concern."
In Japan, the antibody cocktail was granted a Special Approval Pathway under article 14-3 of the Pharmaceuticals and Medical Devices Act. The approval was based on results from a Phase 3 trial in high-risk non-hospitalized patients, which showed the antibody cocktail reduced the risk of hospitalization or death by 70%, as well as results from a Phase 1 trial that examined the safety, tolerability and pharmacokinetics in Japanese people.
Regeneron invented REGEN-COV and is collaborating with Roche to increase global supply of the antibody cocktail, with Roche primarily responsible for development and distribution outside the U.S. In December 2020, Chugai obtained development and exclusive commercialization rights in Japan from Roche, and is working with the Japanese government to ensure an appropriate and timely supply of the antibody cocktail.
The development and manufacturing of REGEN-COV have been funded in part with federal funds from the Biomedical Advanced Research and Development Authority (BARDA), part of the U.S. Department of Health and Human Services' Office of the Assistant Secretary for Preparedness and Response, under OT number: HHSO100201700020C.
About the REGEN-COV Antibody Cocktail
REGEN-COV (casirivimab and imdevimab) is a cocktail of two monoclonal antibodies that was designed specifically to block infectivity of SARS-CoV-2, the virus that causes COVID-19, using Regeneron's proprietary VelocImmune® and VelociSuite® technologies. The two potent, virus-neutralizing antibodies that form the cocktail bind non-competitively to the critical receptor binding domain of the virus's spike protein, which diminishes the ability of mutant viruses to escape treatment and protects against spike variants that have arisen in the human population, as detailed in Science.
Over the past few months Regeneron has announced results from multiple Phase 3 trials demonstrating the ability of REGEN-COV to reduce the burden of COVID-19, from prevention through to hospitalization. This includes trials assessing the ability of REGEN-COV to treat outpatients already infected with SARS-COV-2 (including symptomatic outpatients and recently infected asymptomatic patients) and in certain hospitalized patients, including the RECOVERY trial.
Multiple analyses, including a recent publication in Cell, have shown that REGEN-COV retains potency against the main variants of concern circulating within the U.S.; consequently, REGEN-COV remains available for use in all 50 states. REGEN-COV retains potency against variants including Delta (B.1.162.2; first identified in India), Gamma (P.1; first identified in Brazil), Beta (B.1.351; first identified in South Africa).
REGEN-COV is available throughout the U.S. – information on availability in your area is available from the Department of Health and Human Services and the National Infusion Center Association. REGEN-COV has not been approved by the U.S. Food and Drug Administration (FDA), but is currently authorized in the U.S. under an Emergency Use Authorization (EUA) to treat mild-to-moderate COVID-19 in adults and pediatric patients (12 years of age and older weighing ≥40 kg) with positive results of direct SARS-CoV-2 viral testing, and who are at high risk for progression to severe COVID-19, including hospitalization or death. This use is authorized only for the duration of the declaration that circumstances exist justifying the authorization of the emergency use under section 564(b)(1) of the Act, 21 U.S.C. § 360bbb-3(b)(1), unless the authorization is terminated or revoked sooner. REGEN-COV is not authorized for use in patients who are hospitalized due to COVID-19 or require oxygen therapy, or for people currently using chronic oxygen therapy because of an underlying comorbidity who require an increase in baseline oxygen flow rate due to COVID-19.
In the U.S., REGEN-COV can be administered by intravenous infusion (as short as 20 minutes) or by subcutaneous injection (4 injections), which is an alternative when intravenous infusion is not feasible and would lead to a delay in treatment. It is now authorized as a co-formulated single vial, or in individual vials to be administered together.
Regeneron and Roche share a commitment to making the antibody cocktail available to COVID-19 patients around the globe and will support access in low- and lower-middle-income countries through drug donations to be made in partnership with public health organizations.
AUTHORIZED USE AND IMPORTANT SAFETY INFORMATION
REGEN-COV, (casirivimab and imdevimab) co-formulated product and REGEN-COV (casirivimab and imdevimab) supplied as individual vials to be administered together, is authorized for the treatment of mild to moderate coronavirus disease 2019 (COVID-19) in adults and pediatric patients (12 years of age and older weighing at least 40 kg) with positive results of direct SARS-CoV-2 viral testing, and who are at high risk for progression to severe COVID-19, including hospitalization or death. [see Limitations of Authorized Use]
- REGEN-COV has not been approved, but has been authorized for emergency use by FDA
- This use is authorized only for the duration of the declaration that circumstances exist justifying the authorization of the emergency use under section 564(b)(1) of the Act, 21 U.S.C. § 360bbb-3(b)(1), unless the authorization is terminated or revoked sooner
- Healthcare providers should review the Fact Sheet for Healthcare Providers for information on the authorized use of REGEN-COV and mandatory requirements of the EUA and must comply with the requirements of the EUA. The FDA Letter of Authorization is available for reference, as well as the Dear Healthcare Provider Letter and Patient Fact Sheet
Limitations of Authorized Use
- REGEN-COV (casirivimab and imdevimab) is not authorized for use in patients:
- who are hospitalized due to COVID-19, OR
- who require oxygen therapy due to COVID-19, OR
- who require an increase in baseline oxygen flow rate due to COVID-19 in those on chronic oxygen therapy due to underlying non-COVID-19 related comorbidity
- Benefit of treatment with REGEN-COV has not been observed in patients hospitalized due to COVID-19. Monoclonal antibodies, such as REGEN-COV, may be associated with worse clinical outcomes when administered to hospitalized patients with COVID-19 requiring high-flow oxygen or mechanical ventilation
For additional information about the company, please visit www.regeneron.com
View original content:https://www.prnewswire.com/news-releases/japan-becomes-first-country-to-approve-regeneron-antibody-cocktail-casirivimab-and-imdevimab-for-the-treatment-of-mild-to-moderate-covid-19-301336962.html
SOURCE Regeneron Pharmaceuticals, Inc.
Japan approves Regeneron/Roche's antibody cocktail REGEN-COV in COVID-19
Jul. 20, 2021 6:20 AM ETRegeneron Pharmaceuticals, Inc. (REGN)Roche Holding AG (RHHBY)Regeneron Pharmaceuticals, Inc. (REGN)By: Mamta Mayani, SA News Editor
- Regeneron Pharmaceuticals (NASDAQ:REGN) announces that Japan's Ministry of Health, Labour and Welfare has approved its casirivimab and imdevimab antibody cocktail to treat patients with mild to moderate COVID-19.
- This marks the first time the antibody cocktail, known as REGEN-COV in U.S. and Ronapreve in other countries, has received a full approval for COVID-19.
- https://seekingalpha.com/news/3716701-japan-approves-regeneronroches-antibody-cocktail-regen-cov-in-covid-19
- https://seekingalpha.com/symbol/REGN
- https://seekingalpha.com/symbol/RHHBY
- https://www.regeneron.com/medicines/casirivimab-imdevimab
- https://www.regeneron.com/
RYEQO® (relugolix 40 mg, estradiol 1.0 mg, and norethindrone acetate 0.5 mg)
Myovant Sciences Announces European Commission Approval for RYEQO® for the Treatment of Women With Uterine Fibroids
July 20, 2021 at 2:00 AM EDTPDF Version
- RYEQO is the first and only once-daily long-term treatment for uterine fibroids in Europe
- Indication has no limitation for duration of use, as supported by safety and efficacy data from the Phase 3 LIBERTY program
- Gedeon Richter to commercialize RYEQO, starting in the second half of 2021; Myovant to receive a regulatory milestone payment and is eligible to receive tiered royalties on net sales as well as sales milestone payments
- Marketing authorization application for RYEQO for endometriosis-associated pain on track for submission this calendar year
BASEL, Switzerland, July 20, 2021 (GLOBE NEWSWIRE) -- Myovant Sciences (NYSE: MYOV), a healthcare company focused on redefining care for women and for men, today announced the European Commission (EC) has approved the marketing authorization application for RYEQO® (relugolix 40 mg, estradiol 1.0 mg, and norethindrone acetate 0.5 mg) for the treatment of moderate to severe symptoms of uterine fibroids in adult women of reproductive age, with no limitation for duration of use. The EC decision is valid in all 27 member states of the European Union, as well as Iceland, Norway, and Liechtenstein.
The approval is based on safety and efficacy data from the Phase 3 LIBERTY program, which consisted of two replicate, 24-week, multinational clinical studies (LIBERTY 1 and LIBERTY 2), a one-year extension study, and supportive bone mineral density data from a randomized withdrawal study. Results from the LIBERTY 1 and LIBERTY 2 studies were published in the New England Journal of Medicine in February 2021.
In March 2020, Myovant and Gedeon Richter entered into an exclusive license agreement for Gedeon Richter to commercialize relugolix combination tablet for uterine fibroids and endometriosis in Europe, the Commonwealth of Independent States including Russia, Latin America, Australia, and New Zealand. Under the agreement, Myovant received an upfront payment of $40 million and is eligible to receive up to $40 million in regulatory milestones and $107.5 million in sales milestones for a total of $147.5 million, and tiered royalties on net sales following regulatory approval. Gedeon Richter will be responsible for local clinical development, manufacturing, and all commercialization for its territories. Myovant and Gedeon Richter will also continue to collaborate on the marketing authorization application for endometriosis, which is expected to be submitted in the second half of calendar year 2021.
About RYEQO®
RYEQO (relugolix 40 mg, estradiol 1.0 mg, and norethindrone acetate 0.5 mg) is approved for the treatment of moderate to severe symptoms of uterine fibroids in adult women of reproductive age. RYEQO 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 more information, please visit our website at www.myovant.com.
RELUGOLIX
COMBINATION
TABLET
Relugolix Combination Tablet* consists of relugolix 40 mg, estradiol 1.0 mg, and norethindrone acetate 0.5 mg. Relugolix Combination Tablet is under investigation for the treatment of endometriosis and for the prevention of pregnancy.
*tablet is the intended commercial presentation based on pharmaco-equivalence data with relugolix combination therapy (co-administration of relugolix 40 mg with estradiol 1.0 mg and norethindrone acetate 0.5 mg)
https://www.myovant.com/our-science/relugolix-combination-tablet/
EC approves Myovant Sciences' Ryeqo marketing application for uterine fibroids
Jul. 20, 2021 5:59 AM ETMyovant Sciences Ltd. (MYOV)Myovant Sciences Ltd. (MYOV)By: Mamta Mayani, SA News Editor2 Comments
- Myovant Sciences (NYSE:MYOV) announces that the European Commission (EC) has approved the marketing authorization application (MAA) for Ryeqo (relugolix 40 mg, estradiol 1.0 mg, and norethindrone acetate 0.5 mg) for the treatment of moderate to severe symptoms of uterine fibroids in adult women of reproductive age, with no limitation for duration of use.
- The approval is based on safety and efficacy data from the Phase 3 LIBERTY program.
- https://seekingalpha.com/news/3716692-ec-approves-myovant-sciences-ryeqo-marketing-application-for-uterine-fibroids
- https://seekingalpha.com/symbol/MYOV
- https://www.myovant.com/
Valoctocogene Roxaparvovec
BioMarin Announces Oral Presentation of Positive One-Year Results from Phase 3 Pivotal Trial with Valoctocogene Roxaparvovec Gene Therapy in Adults with Severe Hemophilia A at International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress
Largest Gene Therapy Study in Hemophilia A Demonstrates Superiority in Key Clinical Efficacy Endpoints Compared to Baseline Factor VIII Prophylactic TherapySignificantly Reduced Mean Annualized Bleeding Rate (ABR) by 84% (p-value <0.001) and mean annualized Factor VIII Utilization Rate by 99% (p-value <0.001) Compared to Baseline Factor VIII Prophylactic TherapyJul 19, 2021
SAN RAFAEL, Calif., July 19, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced new data for valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A, in its positive pivotal study, GENEr8-1, during an oral presentation at the International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress. The pivotal study demonstrated superiority to Factor VIII prophylaxis in key clinical efficacy endpoints. With 134 participants, this is the largest global Phase 3 study to date for gene therapy in hemophilia. All participants in the study received a single dose of valoctocogene roxaparvovec and completed a year or more of follow-up. Top-line one-year results from this study were previously communicated in January 2021.
New data presented at ISTH include more details on annualized bleeding rate (ABR) in all study participants and annualized Factor VIII utilization rate, in terms of international units per kilogram per year (IU/kg/year) of replacement Factor VIII. Over 90 percent (N=134) of all participants in the GENEr8-1 study had an annualized bleed rate (ABR) of zero or a lower bleed rate than baseline after week 4 after treatment with valoctocogene roxaparvovec.
Study participants also experienced a clinically meaningful increase in endogenous Factor VIII expression. At the end of the first year post-infusion with valoctocogene roxaparvovec, participants in the modified intent-to-treat (mITT) population (N=132) had a significant increase in mean endogenous Factor VIII expression level from an imputed baseline of 1 IU/dL to 42.9 IU/dL (median 23.9) (p-value <0.001) as measured by the chromogenic substrate (CS) assay, supporting the marked clinical benefits observed with abrogation of bleeding episodes and Factor VIII utilization and infusion rates. In a subset of the mITT population that had been dosed at least two years prior to the data cut date (N=17), Factor VIII expression declined from a mean of 42.2 (median 23.9) IU/dL at the end of year one to 24.4 (median 14.7) IU/dL at the end of year two with continued hemostatic efficacy.
"The demonstrated bleed control at 52 weeks and beyond in this pivotal study supports our thesis that gene therapy can play an important role in the treatment of severe hemophilia A and potentially creates the possibility for a new treatment paradigm," said Margareth C. Ozelo, MD, PhD, Director, INCT do Sangue Hemocentro UNICAMP, University of Campinas and Lead Principal Investigator of the GENEr8-1 Study. "It is encouraging to see meaningful endogenous Factor VIII expression and decreases in bleeding and Factor VIII infusions for people in this study. These pivotal results contribute to the growing body of data to increase understanding of the safety and efficacy of gene therapy treatment over time."
"From the start of our valoctocogene roxaparvovec program, our goal remains to advance treatment options for people with severe hemophilia A in light of the unmet need in bleed control. Current prophylactic therapies for hemophilia A cannot maintain Factor VIII levels for sustained periods, leading to the need for frequent, regular infusions or injections while still having a risk of ongoing, unpredictable bleeds and unavoidable, irreversible joint damage even with standard of care treatment," said Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin. "These data build upon the foundation for a potential transformative treatment option that addresses the root cause of severe hemophilia A. Later at ISTH, we look forward to sharing five years of clinical data from the ongoing Phase 1/2 study with the longest duration of clinical experience, which complements this pivotal Phase 3 study, the largest study of a gene therapy in hemophilia A."
GENEr8-1 Study Description
The global Phase 3 GENEr8-1 study evaluates superiority of valoctocogene roxaparvovec at the 6e13 vg/kg dose compared to FVIII prophylactic therapy. All study participants had severe hemophilia A at baseline, defined as less than or equal to 1 IU/dL of Factor VIII activity. The study included 134 total participants, all of whom had a minimum of 12 months of follow-up at the time of the data cut. The first 22 participants were directly enrolled into the Phase 3 study, 17 of whom were HIV-negative and dosed at least 2 years prior to the data cut date (referred to as the subset). The remaining 112 participants (rollover population) completed at least six months in a separate non-interventional study to prospectively assess bleeding episodes, Factor VIII use, and health-related quality of life while receiving Factor VIII prophylaxis prior to rolling over to receive a single infusion of valoctocogene roxaparvovec in the GENEr8-1 study.
Regulatory Status
The European Medicines Agency (EMA) validated BioMarin's resubmission of a Marketing Authorization Application (MAA) on July 15, 2021. In May 2021, the EMA granted the Company's request for accelerated assessment. Accelerated assessment potentially reduces the time frame for the EMA Committee for Medicinal Products for Human Use (CHMP) and Committee for Advanced Therapies (CAT) to review a MAA for an Advanced Therapy Medicinal Product (ATMP), although an application initially designated for accelerated assessment can revert to the standard procedure during the review for a variety of reasons. The decision to grant accelerated assessment has no impact on the eventual CHMP and CAT opinion on whether a marketing authorization should be granted. A CHMP and CAT opinion is anticipated in the first half of 2022.
The MAA submission includes safety and efficacy data from the 134 subjects enrolled in the Phase 3 GENEr8-1 study, all of whom have been followed for at least one year after treatment with valoctocogene roxaparvovec, as well as four and three 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.
In the United States, BioMarin intends to submit two-year follow-up safety and efficacy data on all study participants from the Phase 3 GENEr8-1 study to support the benefit/risk assessment of valoctocogene roxaparvovec, as previously requested by the Food and Drug Administration (FDA). BioMarin is targeting a Biologics License Application (BLA) resubmission in the second quarter of 2022, assuming favorable study results, followed by an expected six-month review by the FDA.
The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to valoctocogene roxaparvovec in March 2021. RMAT is an expedited program intended to facilitate development and review of regenerative medicine therapies, such as valoctocogene roxaparvovec, that are intended to address an unmet medical need in patients with serious conditions. The RMAT designation is complementary to Breakthrough Therapy Designation, which the Company received in 2017.
In addition to the RMAT Designation and Breakthrough Therapy Designation, BioMarin's valoctocogene roxaparvovec also has received orphan drug designation from the FDA and EMA for the treatment of severe hemophilia A. The Orphan Drug Designation program is intended to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions.
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 actively enrolling participants in 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. The Company is also 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, as well as another Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with hemophilia A with active or prior FVIII inhibitors.
For additional information, please visit www.biomarin.com
Valoctocogene roxaparvovec is an investigational AAV5 gene therapy under regulatory review for the treatment of severe hemophilia A.
Hemophilia A is a genetic disease caused by the deficiency of clotting factor VIII. It is the most common type of hemophilia and occurs much more frequently in males; incidence is estimated at 1 in 4,000-5,000 male births.
https://www.biomarin.com/our-treatments/pipeline/valoctocogene-roxaparvovec-for-severe-hemophilia-a/
BioMarin phase 3 data demonstrates superiority of gene therapy for hemophilia A
Jul. 19, 2021 12:38 PM ET
BioMarin Pharmaceutical Inc. (BMRN)BioMarin Pharmaceutical Inc. (BMRN)
By: Jonathan M Block, SA News Editor
- New data demonstrates that BioMarin Pharmaceutical's (BMRN -0.9%) gene therapy valoctocogene roxaparvovec, was superior to Factor VIII prophylaxis in a phase 3 trial based on efficacy endpoints.
- https://seekingalpha.com/symbol/BMRN
- https://www.biomarin.com/
Imfinzi (durvalumab)
Imfinzi approved in China for the treatment of extensive-stage small cell lung cancer
PUBLISHED19 July 2021
19 July 2021 07:00 BST
Only PD-1/PD-L1 immunotherapy to demonstrate both a significant survival benefit and improved response rate in combination with a choice of chemotherapies
AstraZeneca’s Imfinzi (durvalumab) has been approved in China for the 1st-line treatment of adult patients with extensive-stage small cell lung cancer (ES-SCLC), in combination with standard of care platinum chemotherapy (etoposide plus a choice of either carboplatin or cisplatin).
The approval by China’s National Medical Products Administration was based on positive results from the CASPIAN Phase III trial. The trial showed that Imfinzi plus chemotherapy demonstrated a statistically significant and clinically meaningful improvement in overall survival (OS) versus chemotherapy alone. In addition, results from the China cohort of patients were consistent with the global results.
Small cell lung cancer (SCLC) is a highly aggressive, fast-growing form of lung cancer that typically recurs and progresses rapidly despite initial response to chemotherapy.1,2 Prognosis is particularly poor, as only 7% of all SCLC patients and 3% of those with extensive-stage disease will be alive five years after diagnosis.3
Dave Fredrickson, Executive Vice President, Oncology Business Unit, said: “Today’s approval of Imfinzi plus chemotherapy brings an important global standard of care to patients with extensive-stage small cell lung cancer in China, who have had few treatment options and a dire prognosis. Physicians can now offer these patients a well-tolerated immunotherapy regimen with sustained overall survival and prolonged treatment response, as well as convenient dosing. This is also the first time physicians have had the choice to combine immunotherapy with cisplatin, a preferred chemotherapy in this setting in China.”
The CASPIAN Phase III trial met the primary endpoint of OS for Imfinzi plus chemotherapy in June 2019, reducing the risk of death by 27% versus chemotherapy alone (based on a hazard ratio [HR] of 0.73; 95% confidence interval [CI] 0.59-0.91; p=0.0047), with a median OS of 13.0 months versus 10.3 months for chemotherapy alone. These results were published in The Lancet in October 2019.4 Results also showed an increased confirmed objective response rate for Imfinzi plus chemotherapy (68% versus 58% for chemotherapy alone). Imfinzi added to chemotherapy delayed the time for disease symptoms to worsen.4
An updated analysis showed sustained efficacy for Imfinzi plus chemotherapy after a median follow up of more than two years (OS HR of 0.75; 95% CI 0.62-0.91; nominal p=0.0032), with median OS of 12.9 months versus 10.5 months for chemotherapy alone. An estimated 22.2% of patients treated with Imfinzi plus chemotherapy were alive at 24 months versus 14.4% for chemotherapy alone. The safety and tolerability for Imfinzi and chemotherapy were consistent with the known safety profiles of these medicines. No patients tested positive for treatment-emergent anti-drug antibodies to Imfinzi.
Safety and efficacy results in the China cohort of patients were consistent with results in the overall global trial population. Detailed results from this cohort will be presented at a forthcoming medical meeting.
The CASPIAN Phase III trial used a fixed dose of Imfinzi (1500mg) administered every three weeks for four cycles while in combination with chemotherapy and then every four weeks as a monotherapy until disease progression. Based on the trial results, Imfinzi, in combination with etoposide and either carboplatin or cisplatin, is approved in the 1st-line setting of ES-SCLC in more than 55 countries, including the US, Japan and across the EU.
Imfinzi is also being tested following concurrent chemoradiation therapy in patients with limited-stage SCLC in the ADRIATIC Phase III trial as part of a broad development programme. In addition, Imfinzi is also approved to treat non-small cell lung cancer (NSCLC) in the curative-intent setting of unresectable, Stage III disease after chemoradiation therapy in the US, Japan, China, across the EU and in many other countries, based on results from the PACIFIC Phase III trial.
CASPIAN
CASPIAN was a randomised, open-label, multi-centre, global Phase III trial in the 1st-line treatment of 805 patients with ES-SCLC. The trial compared Imfinzi in combination with etoposide and either carboplatin or cisplatin platinum chemotherapy, or Imfinzi and chemotherapy with the addition of a second immunotherapy, tremelimumab, versus chemotherapy alone. In the two experimental arms, patients were treated with four cycles of chemotherapy. In comparison, the control arm allowed up to six cycles of chemotherapy and optional prophylactic cranial irradiation.
The trial was conducted in more than 200 centres across 23 countries, including the US, Europe, South America, Asia and the Middle East. The primary endpoint was OS in each of the two experimental arms. In June 2019, AstraZeneca announced the CASPIAN Phase III trial had met one primary endpoint of demonstrating OS for Imfinzi plus chemotherapy at a planned interim analysis. In March 2020, however, it was announced that the second experimental arm with tremelimumab did not meet its primary endpoint of OS.
Imfinzi
Imfinzi (durvalumab) is a human monoclonal antibody that binds to PD-L1 and blocks the interaction of PD-L1 with PD-1 and CD80, countering the tumour’s immune-evading tactics and releasing the inhibition of immune responses.
In addition to approvals in ES-SCLC and unresectable, Stage III NSCLC, 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 NSCLC, SCLC, bladder cancer, hepatocellular carcinoma, biliary tract cancer (a form of liver cancer), oesophageal
China approves AstraZeneca's Imfinzi for small cell lung cancer
Jul. 19, 2021 7:46 AM ET
By: Jonathan M Block, SA News Editor
- China's National Medical Products Administration has approved AstraZeneca's (AZN -0.3%) Imfinzi (durvalumab) as a first-0line treatment for extensive-stage small cell lung cancer.
- The approval was based on data from the phase 3 CASPIAN trial that showed Imfinzi plus chemotherapy bested chemotherapy alone in terms of overall survival.
- https://seekingalpha.com/news/3716205-china-approves-astrazenecas-imfinzi-for-small-cell-lung-cancer
- https://seekingalpha.com/symbol/AZN
- https://www.imfinzi.com/
- https://www.astrazeneca.com/
VAXNEUVANCE™ (Pneumococcal 15-valent Conjugate Vaccine)
Merck Announces U.S. FDA Approval of VAXNEUVANCE™ (Pneumococcal 15-valent Conjugate Vaccine) for the Prevention of Invasive Pneumococcal Disease in Adults 18 Years and Older Caused by 15 Serotypes
July 16, 2021 5:15 pm ET
Clinical Data Supporting Approval Demonstrated Non-Inferior Immune Responses for the Serotypes Shared with PCV13 (1, 3, 4, 5, 6A, 6B, 7F, 9V, 14, 18C, 19A, 19F and 23F)
VAXNEUVANCE Elicited Superior Immune Responses for Serotypes 3, 22F and 33F Compared to PCV13, Which Are Major Causes of Disease
KENILWORTH, N.J.--(BUSINESS WIRE)-- (NYSE: MRK), known as MSD outside the United States and Canada, today announced the U.S. Food and Drug Administration (FDA) approved VAXNEUVANCE™ (Pneumococcal 15-valent Conjugate Vaccine) (pronounced VAKS-noo-vans) for active immunization for the prevention of invasive disease caused by Streptococcus pneumoniae serotypes 1, 3, 4, 5, 6A, 6B, 7F, 9V, 14, 18C, 19A, 19F, 22F, 23F and 33F in adults 18 years of age and older. The approval follows the FDA’s Priority Review of Merck’s application. VAXNEUVANCE is contraindicated for individuals with a history of severe allergic reaction (e.g., anaphylaxis) to any component of VAXNEUVANCE or to diphtheria toxoid; see additional Select Safety Information below.
The U.S. Centers for Disease Control and Prevention’s (CDC) Advisory Committee on Immunization Practices (ACIP) is expected to meet in October to discuss and make recommendations on the use of VAXNEUVANCE in adults.
VAXNEUVANCE was approved based on data from seven randomized, double-blind clinical studies assessing safety, tolerability, and immunogenicity in adults (see “Clinical Data Supporting FDA Approval” below for additional details). Clinical data showed that immune responses elicited by VAXNEUVANCE were non-inferior to the currently available 13-valent pneumococcal conjugate vaccine (PCV13) for the 13 shared serotypes, as assessed by opsonophagocytic activity (OPA) Geometric Mean Titers (GMTs).
Additionally, immune responses for VAXNEUVANCE were superior to PCV13 for shared serotype 3 and for the two serotypes unique to VAXNEUVANCE, 22F and 33F. In the pivotal Phase 3 PNEU-AGE (V114-019) study, superiority for VAXNEUVANCE relative to PCV13 was based on statistically significantly greater OPA GMT ratios for serotypes 22F [GMT Ratio 32.52 (95% Confidence Interval (CI) 25.87, 40.88)] and 33F [GMT Ratio 7.19 (95% CI 6.13, 8.43)], as well as for the key secondary objective assessing serotype 3 [GMT Ratio 1.62 (95% CI 1.40, 1.87)]. Randomized controlled trials assessing the clinical efficacy of VAXNEUVANCE compared to PCV13 have not been conducted.
“Some adults, including older adults or those with certain chronic medical conditions or immunocompromising conditions, are at increased risk for pneumococcal disease and its serious, sometimes life-threatening complications,” said Dr. Jose Cardona, Indago Research and Health Center, coordinating investigator for the PNEU-AGE trial. “The FDA’s approval of VAXNEUVANCE is based on robust Phase 2 and 3 studies assessing immune responses in a broad range of adult populations and provides an important new option in protection from invasive pneumococcal disease.”
About VAXNEUVANCE
VAXNEUVANCE, Merck’s approved 15-valent pneumococcal conjugate vaccine, consists of purified capsular polysaccharides from S. pneumoniae serotypes 1, 3, 4, 5, 6A, 6B, 7F, 9V, 14, 18C, 19A, 19F, 22F, 23F and 33F individually conjugated to CRM197 carrier protein. VAXNEUVANCE is indicated for active immunization for the prevention of invasive disease caused by the S. pneumoniae serotypes contained in the vaccine. VAXNEUVANCE previously received Breakthrough Therapy designation from the FDA for the prevention of IPD in adults 18 years of age and older. In January 2021, it received Priority Review designation.
Merck is involved in litigation challenging the validity of several Pfizer Inc. patents that relate to pneumococcal vaccine technology in the United States and several foreign jurisdictions.
For more information, visit www.merck.com
Please see Prescribing Information for VAXNEUVANCE (pneumococcal 15-valent conjugate vaccine) at https://www.merck.com/product/usa/pi_circulars/v/vaxneuvance/vaxneuvance_pi.pdf.
and Patient Information at https://www.merck.com/product/usa/pi_circulars/v/vaxneuvance/vaxneuvance_ppi.pdf.
Brands mentioned are trademarks of their respective owners.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210716005480/en/
Source: Merck & Co., Inc.
Merck wins FDA approval for new pneumonia vaccine
Jul. 17, 2021 10:09 PM ET Pfizer Inc. (PFE), MRK Pfizer Inc. (PFE)Merck & Co., Inc. (MRK) By: Dulan Lokuwithana, SA News Editor7 Comments
- The FDA has approved a new version of a Pneumococcal vaccine developed by Merck (NYSE:MRK) for the prevention of invasive disease caused by 15 strains of Streptococcus pneumoniae, the company announced on Friday.
- VAXNEUVANCE (Pneumococcal 15-valent Conjugate Vaccine) is indicated for adults 18 years of age and older.
- https://seekingalpha.com/news/3716142-merck-wins-fda-approval-for-new-pneumonia-vaccine
- https://seekingalpha.com/symbol/MRK
- https://www.merck.com/
https://www.reuters.com/companies/mrk
Cabenuva (cabotegravir and rilpivirine
17 July 2021
ViiV Healthcare study shows new long-acting HIV regimen Cabenuva (cabotegravir/rilpivirine) can be successfully implemented in broad range of US healthcare practices, even during COVID-19
For media and investors only
Issued: London, UK
- Healthcare teams from a wide variety of US HIV clinics reported that optimal implementation of long-acting cabotegravir and rilpivirine dosed monthly was achieved within 1 – 3 months, and implementation was acceptable, appropriate and feasible
- Majority of patients (74%) in the study reported few barriers to monthly injection appointments even during COVID-19
ViiV Healthcare, the global specialist HIV company majority owned by GlaxoSmithKline plc (“GSK”), with Pfizer Inc. and Shionogi Limited as shareholders, today presented positive findings from the CUSTOMIZE (Cabotegravir plus Rilpivirine long acting in the US To Optimize and Measure Implementation and Experience) trial. The study, which included people living with HIV and healthcare teams and overlapped with the COVID-19 pandemic, demonstrated that Cabenuva (a co-pack with two injectable medicines including ViiV Healthcare’s cabotegravir and Janssen’s rilpivirine) can be successfully implemented across a range of healthcare settings in the US.[i] The 12-month findings were presented at the International AIDS Society Conference 2021 (IAS 2021) being held virtually 18-21 July.
CUSTOMIZE was initiated in 2019 to identify successful methods of integrating the long-acting regimen of cabotegravir and rilpivirine (dosed monthly) for the treatment of HIV-1 after product availability into clinical practices in the US, in a variety of clinic types.[i],[ii] This study included a variety of clinic types from private practices, university clinics and federally qualified health centres, to integrated health care systems.[i] Regardless of clinic types, the majority of healthcare staff (96%, n=22/23) either agreed, or completely agreed, that the long-acting regimen was feasible to implement in their clinic, and most (78%, n=18/23) felt that optimal implementation was achieved within 1-3 months, with only minor adjustments to clinic logistics required.[i]
About CUSTOMIZE (NCT04001803)[i],[ii],[vii]
CUSTOMIZE is a single-arm, multicentre, one-year evaluation of the effect of an implementation strategy on the degree of acceptability, appropriateness, feasibility, fidelity and sustainability of clinic practices to deliver the monthly, long-acting regimen of cabotegravir and rilpivirine to appropriate people living with HIV. The study, which involved 115 people living with HIV and 24 healthcare providers, evaluated both qualitative and quantitative measures across a range of clinic types, including university hospitals as well as private and public clinics, with varied geographic and demographic representation.
A suite of educational items, training aids, treatment and resource planning tools, appointment reminders and patient-directed support items were made available as part of this study. Staff study participants from each site (physician/primary care practitioner, nurse/medication administration personnel, administrator/clinic manager) took part in the study through participation in surveys and interviews. Sustainment of implementation strategies was assessed via surveys and semi-structured interviews of staff study participants as well as patient study participants. The primary endpoint was change from baseline to the injection site visit at Month 12 in site survey responses for acceptability, appropriateness and feasibility.
About Cabenuva (cabotegravir and rilpivirine)[viii]
Cabenuva is indicated as a complete regimen for the treatment of HIV-1 infection in adults who are virologically suppressed (HIV-1 RNA less than 50 copies per milliliter [mL]) on a stable regimen, with no history of treatment failure, and with no known or suspected resistance to either cabotegravir or rilpivirine. Cabenuva is administered as two intramuscular injections (cabotegravir and rilpivirine) in the buttocks during the same visit at a specialist clinic by a healthcare professional.
The complete regimen combines the integrase strand transfer inhibitor (INSTI) cabotegravir, developed by ViiV Healthcare, with rilpivirine, a non-nucleoside reverse transcriptase inhibitor (NNRTI) developed by Janssen Sciences Ireland UC, one of the Janssen Pharmaceutical Companies of Johnson & Johnson.
INSTIs, like cabotegravir, inhibit HIV replication by preventing the viral DNA from integrating into the genetic material of human immune cells (T-cells). This step is essential in the HIV replication cycle and is also responsible for establishing chronic infection. Rilpivirine is an NNRTI that works by interfering with an enzyme called reverse transcriptase, which in turn stops the virus from multiplying.
Trademarks are owned by or licensed to the ViiV Healthcare group of companies.
Please see full Prescribing Information.
For more information on the company, its management, portfolio, pipeline and commitment, please visit www.viivhealthcare.com.
For further information please visit www.gsk.com/about-us.
https://seekingalpha.com/symbol/GSK
https://seekingalpha.com/symbol/PFE
https://viivhealthcare.com/en-us/
biotecMAX™ April/May/June/July 2021 Insight
KEYTRUDA® (pembrolizumab) Plus Chemotherapy
KEYTRUDA® (pembrolizumab) Plus Chemotherapy Before Surgery and Continued as a Single Agent After Surgery Showed Statistically Significant Event-Free Survival (EFS) Result Versus Neoadjuvant Chemotherapy Alone in High-Risk Early-Stage TNBC
July 15, 2021 1:30 pm ET
KEYNOTE-522 Is the First Phase 3 Study With an Immunotherapy to Show Positive EFS Results in High-Risk Early-Stage Triple-Negative Breast Cancer
Merck Has Submitted These Data From KEYNOTE-522 to the US FDA for Review
KENILWORTH, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced positive event-free survival (EFS) data from the pivotal neoadjuvant/adjuvant Phase 3 study KEYNOTE-522. The trial investigated neoadjuvant KEYTRUDA, Merck’s anti-PD-1 therapy, plus chemotherapy followed by adjuvant KEYTRUDA as monotherapy (the KEYTRUDA regimen) compared with neoadjuvant chemotherapy followed by adjuvant placebo (the chemotherapy-placebo regimen) in patients with high-risk early-stage triple-negative breast cancer (TNBC). This is the first time an anti-PD-1/L1 therapy has demonstrated a statistically significant EFS result as combined neoadjuvant and adjuvant therapy for these patients. These results are being presented today during a European Society for Medical Oncology (ESMO) Virtual Plenary.
After a median follow-up of 39 months, the KEYTRUDA regimen reduced the risk of EFS events by 37% (HR=0.63 [95% CI, 0.48-0.82]; p=0.00031) versus the chemotherapy-placebo regimen – a statistically significant and clinically meaningful EFS result. EFS was defined as the time from randomization to the first occurrence of either disease progression that precluded definitive surgery, a local/distant recurrence, a second primary cancer, or death from any cause. As previously announced, KEYNOTE-522 met the dual primary endpoint of pathological complete response (pCR) at the first interim analysis. The trial is continuing to allow for additional follow-up of overall survival (OS), a key secondary endpoint. At this fourth interim analysis, although these data have not crossed the boundary for statistical significance, there was a 28% reduction in the risk of death with the KEYTRUDA regimen versus the chemotherapy-placebo regimen (HR=0.72 [95% CI, 0.51-1.02]; p=0.03214). The safety profile of the KEYTRUDA regimen was consistent with the known profiles of each regimen, and no new safety concerns were identified.
Study Design and Additional Data From KEYNOTE-522
KEYNOTE-522 is a Phase 3, randomized, double-blind trial (ClinicalTrials.gov, NCT03036488). The dual primary endpoints are pCR, defined as pathological stage ypT0/Tis ypN0 at the time of definitive surgery, and EFS, defined as the time from randomization to the first occurrence of either disease progression that precluded definitive surgery, a local/distant recurrence, a second primary cancer, or death from any cause in all patients randomized. Secondary endpoints include pCR rate using alternative definitions, OS in all patients randomized, pCR rate according to all definitions, EFS and OS in patients whose tumors express PD-L1 (CPS ≥1), safety and health-related quality of life assessments. The study enrolled 1,174 patients who were randomized 2:1 to receive either:
- The KEYTRUDA regimen: KEYTRUDA (every three weeks) plus paclitaxel (weekly) and carboplatin (weekly or every three weeks) for four cycles, followed by KEYTRUDA plus cyclophosphamide and either doxorubicin or epirubicin (every three weeks) for four cycles as neoadjuvant therapy prior to surgery, followed by nine cycles of KEYTRUDA (every three weeks) as adjuvant therapy post-surgery (n=784)
- The chemotherapy-placebo regimen: Placebo (every three weeks) plus paclitaxel (weekly) and carboplatin (weekly or every three weeks) for four cycles, followed by placebo plus cyclophosphamide and either doxorubicin or epirubicin (every three weeks) for four cycles as neoadjuvant therapy prior to surgery, followed by nine cycles of placebo (every three weeks) as adjuvant therapy post-surgery (n=390)
As previously announced, KEYNOTE-522 met the success criterion for the dual primary endpoint of pCR at the first interim analysis; pCR was observed in 64.8% of patients treated with KEYTRUDA plus chemotherapy (n=401), an increase of 13.6% (p=0.00055) from 51.2% in patients treated with placebo plus chemotherapy (n=201). At the fourth interim analysis, KEYNOTE-522 met the success criterion for the dual primary endpoint of EFS. The study is continuing to allow for additional follow-up of OS.
At three years, 84.5% of patients treated with the KEYTRUDA regimen were alive and did not experience an EFS event compared to 76.8% of patients treated with the chemotherapy-placebo regimen.
In pre-specified exploratory subgroup analyses of EFS, the EFS benefit seen with the KEYTRUDA regimen was independent of PD-L1 expression. In the PD-L1-positive subgroup (n=973), defined as CPS ≥1, treatment with the KEYTRUDA regimen reduced the risk of EFS events by 33% (HR=0.67 [95% CI, 0.49-0.92]) versus the chemotherapy-placebo regimen. In the PD-L1-negative subgroup (n=197), defined as CPS <1, treatment with the KEYTRUDA regimen reduced the risk of EFS events by 52% (HR=0.48 [95% CI, 0.28-0.85]) versus the chemotherapy-placebo regimen.
In a pre-specified but non-randomized exploratory analysis of EFS by pCR outcome, the reduction in EFS events with the KEYTRUDA regimen was observed independent of pCR outcome at definitive surgery.
Treatment-related adverse events (TRAEs) were examined in the neoadjuvant phase, the adjuvant phase and the combined phases. TRAEs in the neoadjuvant phase have been previously reported. At the time of this data cutoff, no patients were still receiving protocol treatment. For the combined neoadjuvant and adjuvant phases, TRAEs occurred in 98.9% of patients receiving the KEYTRUDA regimen (n=783) and 99.7% of patients receiving the chemotherapy-placebo regimen (n=389); Grade 3-5 TRAEs occurred in 77.1% versus 73.3%, respectively. TRAEs led to death in 0.5% of patients receiving the KEYTRUDA regimen (n=4) and 0.3% of patients receiving the chemotherapy-placebo regimen (n=1). No new safety concerns were identified. In the adjuvant phase, TRAEs occurred in 53.7% of patients receiving adjuvant KEYTRUDA (n=588) and 48.6% of patients receiving adjuvant placebo (n=331), including 6.3% and 2.7%, respectively, who had at least one Grade ≥3 event.
Immune-mediated adverse events (AEs) and infusion reactions of any grade in the combined neoadjuvant and adjuvant phases occurred in 43.6% of patients receiving the KEYTRUDA regimen and 21.9% of patients receiving the chemotherapy-placebo regimen. The most common of these events (occurring in ≥10% of patients) were infusion reactions (18.0%) and hypothyroidism (15.1%) in patients receiving the KEYTRUDA regimen and infusion reactions (11.6%) in patients receiving the chemotherapy-placebo regimen. Immune-mediated AEs led to death in 0.3% of patients receiving the KEYTRUDA regimen (n=2) and no patients receiving the chemotherapy-placebo regimen. In the adjuvant phase, immune-mediated AEs and infusion reactions occurred in 10.2% of patients receiving adjuvant KEYTRUDA and 6.0% of patients receiving adjuvant placebo, including 2.9% and 0.3%, respectively, who had at least one Grade ≥3 event.
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 1,500 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.
Selected KEYTRUDA® (pembrolizumab) Indications in the U.S.
Melanoma
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.
KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.
Non-Small Cell Lung Cancer
KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) ≥1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is:
- stage III where patients are not candidates for surgical resection or definitive chemoradiation, or
- metastatic.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS ≥1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.
Head and Neck Squamous Cell Cancer
KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.
Classical Hodgkin Lymphoma
KEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).
KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.
Primary Mediastinal Large B-Cell Lymphoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.
Urothelial Carcinoma
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 (CPS ≥10) as determined by an FDA-approved test, or in patients who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
KEYTRUDA is indicated for the treatment of patients with locally advanced or mUC who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.
Microsatellite Instability-High or Mismatch Repair Deficient Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options.
This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.
Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).
Gastric Cancer
KEYTRUDA, in combination with trastuzumab, fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of patients with locally advanced unresectable or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Esophageal Cancer
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic esophageal or GEJ (tumors with epicenter 1 to 5 centimeters above the GEJ) carcinoma that is not amenable to surgical resection or definitive chemoradiation either:
- in combination with platinum- and fluoropyrimidine-based chemotherapy, or
- as a single agent after one or more prior lines of systemic therapy for patients with tumors of squamous cell histology that express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
Cervical Cancer
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Hepatocellular Carcinoma
KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Merkel Cell Carcinoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Renal Cell Carcinoma
KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma.
Tumor Mutational Burden-High Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [≥10 mutations/megabase] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.
Cutaneous Squamous Cell Carcinoma
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) or locally advanced cSCC that is not curable by surgery or radiation.
Triple-Negative Breast Cancer
KEYTRUDA, in combination with chemotherapy, is indicated for the treatment of patients with locally recurrent unresectable or metastatic triple-negative breast cancer (TNBC) whose tumors express PD-L1 (CPS ≥10) as determined by an FDA-approved test. This indication is approved under accelerated approval based on progression-free survival. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Merck's Keytruda demonstrates event-free survival in high-risk, early-stage breast cancer
Jul. 15, 2021 2:07 PM ET
Merck & Co., Inc. (MRK) By: Jonathan M Block, SA News Editor
- Merck's (MRK +0.2%) anti-PD-1 therapy Keytruda (pembrolizumab) demonstrated statistically significant event-free survival ("EFS") compared to a chemotherapy-placebo regimen in a phase 3 study for high-risk, early-stage triple-negative breast cancer.
- Merck's Keytruda demonstrates event-free survival in high-risk, early-stage breast cancer | Seeking Alpha
- Merck & Co., Inc. (MRK) Stock Price Today, Quote & News | Seeking Alpha
- https://www.merck.com/
- https://www.keytruda.com/
selinexor (XPOVIO®)
Antengene Submits New Drug Application for Selinexor in Taiwan for the Treatment of Three Indications in Hematologic Malignancies
Jul 14, 2021View PDF
-In July 2019, selinexor (XPOVIO®) was approved by the U.S. FDA and became the first and only XPO1 inhibitor indicated for the treatment of hematologic malignancies.
-Selinexor has been approved in the U.S., Israel, UK, and EU countries.
Shanghai and Hong Kong, PRC, June 14, 2021 – Antengene Corporation Limited (“Antengene”, SEHK: 6996.HK), a leading innovative biopharmaceutical company dedicated to discovering, developing and commercializing global first-in-class and/or best-in-class therapeutics in hematology and oncology, today announced that it has submitted a New Drug Application (NDA) to the Taiwan Food and Drug Administration (TFDA) for selinexor, a first-in-class XPO1 inhibitor, for three indications: in combination with bortezomib and dexamethasone (XVd), or in combination with dexamethasone (Xd) for the treatment of patients with relapsed and/or refractory multiple myeloma (RRMM); and as monotherapy in adult patients with relapsed and/or refractory diffuse large B-cell lymphoma (rrDLBCL), including DLBCL arising from follicular lymphoma, who have received at least two lines of systemic therapy.
Antengene has submitted New Drug Applications (NDAs) for selinexor in multiple Asia Pacific markets including China, Australia, South Korea, and Singapore, and was granted Priority Review status by China’s National Medical Products Administration (NMPA) and Orphan Drug Designation by the Ministry of Food and Drug Safety of South Korea (MFDS). This NDA submitted in Taiwan marks another milestone in Antengene’s expansion in the APAC markets and the company’s effort to address the unmet clinical needs of patients with hematologic malignancies.
“Selinexor is an anti-tumor drug with a highly novel mechanism of action. It has been approved by the FDA for three indications in two tumor types (MM and DLBCL) in less than two years, demonstrating its broad anti-tumor effects,” said Kevin Lynch, Chief Medical Officer of Antengene. “Selinexor treatment in combination with dexamethasone still has 25.3% objective response rate (ORR) in patients who have failed five established therapies (penta-refractory) myeloma; and for patients with multiple myeloma who received at least one prior line of treatment, the median progression-free survival (PFS) is 13.9 months, which is significantly higher than that of the control group receiving bortezomib-based standard of care. In the subgroup analysis, patients who are 65 years or older, with renal insufficiency or high-risk cytogenetics can still achieve significant benefits from the XVd regimen. These are patients who are otherwise very difficult to treat. Finally, selinexor monotherapy can enable patients with rrDLBCL to obtain deep and durable responses with a median duration of response of 23.0 months for patients with complete response. We are therefore very optimistic about the potential efficacy benefits of selinexor combination regimens in DLBCL.”
Antengene and Karyopharm Therapeutics Inc. (NASDAQ: KPTI) have entered into an exclusive collaboration and license agreement for the development and commercialization of selinexor and other two XPO1 inhibitors, and a PAK4/NAMPT inhibitor in 17 APAC markets including Mainland China.
About Selinexor (XPOVIO®)
Selinexor, a first-in-class and only-in-class oral selective inhibitor of nuclear export (SINE) compound discovered and developed by Karyopharm, is currently being developed by Antengene, which has the exclusive development and commercial rights in certain Asia-Pacific markets, including Greater China, South Korea, Australia, New Zealand and the ASEAN countries.
In July 2019, the US Food and Drug Administration (FDA) approved selinexor in combination with low-dose dexamethasone for the treatment of RRMM and in June 2020 approved selinexor as a single-agent for the treatment of rrDLBCL. In December 2020, selinexor also received FDA approval as a combination treatment (XVd) for MM after at least one prior therapy. In February 2021, selinexor was approved by the Israeli Ministry of Health for the treatment of patients with RRMM or rrDLBCL and in March 2021, the European Commission (EC) has granted conditional marketing authorization for selinexor (NEXPOVIO) for the treatment of adult patients with RRMM.
Selinexor is so far the first and only oral SINE compound approved by the FDA and is the first drug approved for the treatment of both MM and DLBCL. Selinexor is also being evaluated in several other mid-and later-phase clinical trials across multiple solid tumor indications, including liposarcoma and endometrial cancer. In November 2020, at the Connective Tissue Oncology Society 2020 Annual Meeting (CTOS 2020), Antengene’s partner, Karyopharm, presented positive results from the Phase III randomized, double blind, placebo controlled, cross-over SEAL trial evaluating single agent, oral selinexor versus matching placebo in patients with liposarcoma. Karyopharm also announced that the ongoing Phase III SIENDO trial of selinexor in patients with endometrial cancer passed the planned interim futility analysis and the Data and Safety Monitoring Board (DSMB) recommended the trial should proceed as planned without any modifications. Top-line SIENDO trial results are expected in the second half of 2021.
Antengene is currently conducting five late-stage clinical trials of selinexor in China for the treatment of MM, DLBCL, non-small cell lung cancer, and peripheral T and NK/T-cell lymphoma.
Antengene submits selinexor application in Taiwan in hematologic malignancies
Jul. 14, 2021 6:11 AM ETKaryopharm Therapeutics Inc. (KPTI)By: Mamta Mayani, SA News Editor
- Karyopharm Therapeutics' (NASDAQ:KPTI) partner Antengene has submitted a New Drug Application (NDA) to the Taiwan Food and Drug Administration for selinexor, a XPO1 inhibitor, for three indications:
- https://seekingalpha.com/news/3714989-antengene-submits-selinexor-application-in-taiwan-in-hematologic-malignancies
- https://seekingalpha.com/symbol/KPTI
- https://www.xpoviopro.com/
- https://www.antengene.com/
Valoctocogene Roxaparvovec
European Medicines Agency Validates BioMarin's Marketing Authorization Application for Valoctocogene Roxaparvovec to Treat Severe Hemophilia A
Potential 1st Gene Therapy in Europe for Treatment of Hemophilia ACHMP Opinion Anticipated in 1H 2022Jul 15, 2021
SAN RAFAEL, Calif., July 15, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the European Medicines Agency (EMA) validated the Company's Marketing Authorization Application (MAA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. With today's validation the MAA review can now commence. A CHMP opinion is anticipated in the first half of 2022.
The submission includes safety and efficacy data from the 134 subjects enrolled in the Phase 3 GENEr8-1 study, all of whom have been followed for at least one year after treatment with valoctocogene roxaparvovec, as well as four and three 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.
Regulatory Status
BioMarin resubmitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) on June 25, 2021. In the United States, BioMarin intends to submit two-year follow-up safety and efficacy data on all study participants from the Phase 3 GENEr8-1 study to support the benefit/risk assessment of valoctocogene roxaparvovec, as previously requested by the Food and Drug Administration (FDA). BioMarin is targeting a Biologics License Application (BLA) resubmission in the second quarter of 2022, assuming favorable study results, followed by an expected six-month review by the FDA.
The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to valoctocogene roxaparvovec in March 2021. RMAT is an expedited program intended to facilitate development and review of regenerative medicine therapies, such as valoctocogene roxaparvovec, that are intended to address an unmet medical need in patients with serious conditions. The RMAT designation is complementary to Breakthrough Therapy Designation, which the Company received in 2017.
In addition to the RMAT Designation and Breakthrough Therapy Designation, BioMarin's valoctocogene roxaparvovec also has received orphan drug designation from the FDA and EMA for the treatment of severe hemophilia A. The Orphan Drug Designation program is intended to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions.
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 actively enrolling participants in 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. The Company is also 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, as well as another Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with hemophilia A with active or prior FVIII inhibitors.
For additional information, please visit www.biomarin.com.
EMA accepts BioMarin's valoctocogene roxaparvovec hemophilia MAA for review
Jul. 15, 2021 8:55 AM ET
BioMarin Pharmaceutical Inc. (BMRN) By: Aakash Babu, SA News Editor
- BioMarin Pharmaceutical (NASDAQ:BMRN) announces that the EMA has accepted to review the company's Marketing Authorization Application (MAA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A.
- https://seekingalpha.com/news/3715491-ema-accepts-biomarins-valoctocogene-roxaparvovec-hemophilia-maa-for-review
- https://seekingalpha.com/symbol/BMRN
- https://www.biomarin.com/our-treatments/pipeline/valoctocogene-roxaparvovec-for-severe-hemophilia-a/
- https://www.biomarin.com/
KYPROLIS® (Carfilzomib)
BeiGene Announces the Approval in China of KYPROLIS® (Carfilzomib) for Injection for Adult Patients with Relapsed or Refractory Multiple Myeloma
July 9, 2021 at 12:00 AM EDT
BEIJING & CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jul. 9, 2021-- BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a global biotechnology company focused on developing and commercializing innovative medicines worldwide, today announced that the China National Medical Products Administration (NMPA) has conditionally approved KYPROLIS® (carfilzomib) for injection in combination with dexamethasone for the treatment of adult patients with relapsed or refractory (R/R) multiple myeloma who have received at least two prior therapies, including a proteasome inhibitor and an immunomodulatory agent. KYPROLIS is licensed to BeiGene in China under a strategic collaboration with Amgen. This is the first approval for KYPROLIS in China.
The conditional approval of KYPROLIS was based on results from the Phase 3 trial (NCT03029234) in China evaluating the efficacy and safety of KYPROLIS plus dexamethasone in 123 patients with R/R multiple myeloma. Results of the trial showed that the efficacy in Chinese patients who had received a median of four prior lines of therapy were generally consistent with that seen in the global studies. The overall response rate, the primary endpoint, was 35.8% (95% CI: 27.3, 44.9). The median progression-free survival (PFS) assessed by Independent Review Committee (IRC) was 5.6 months (95% CI: 4.6, 6.5). The safety profile observed for Chinese patients in this study was consistent with that observed in the global studies evaluating KYPROLIS in R/R multiple myeloma. In global studies, the most common adverse reactions in the combination therapy trials were anemia, diarrhea, fatigue, hypertension, pyrexia, upper respiratory tract infection, thrombocytopenia, cough, dyspnea, and insomnia. No new safety risks were identified based on analyses of adverse events in Chinese patients. Full approval is subject to results of a confirmatory trial.
About KYPROLIS® (carfilzomib) for injection
Proteasomes play an important role in cell function and growth by breaking down proteins that are damaged or no longer needed. KYPROLIS has been shown to block proteasomes, leading to an excessive build-up of proteins within cells. In some cells, KYPROLIS can cause cell death, especially in myeloma cells because they are more likely to contain a higher amount of abnormal proteins.
Since its first approval in 2012, approximately 150,000 patients worldwide have received KYPROLIS. KYPROLIS is approved in the U.S. for the following:
- for the treatment of adult patients with relapsed or refractory multiple myeloma who have received one to three lines of therapy in combination with
- Lenalidomide and dexamethasone; or
- Dexamethasone; or
- Daratumumab and dexamethasone.
- as a single agent for the treatment of adult patients with relapsed or refractory multiple myeloma who have received one or more lines of therapy.
KYPROLIS is also approved in countries or regions including Algeria, Argentina, Australia, Bahrain, Belarus, Brazil, Canada, Chile, Colombia, Ecuador, Egypt, European Union, Hong Kong, India, Israel, Japan, Jordan, Kazakhstan, Kuwait, Lebanon, Macao, Malaysia, Mexico, Morocco, New Zealand, Oman, Peru, Philippines, Qatar, Russia, Saudi Arabia, Serbia, Singapore, S. Africa, S. Korea, Switzerland, Taiwan, Thailand, Turkey and United Arab Emirates.
U.S. INDICATIONS
- KYPROLIS® (carfilzomib) is indicated in combination with dexamethasone or with lenalidomide plus dexamethasone or with daratumumab and dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma who have received one to three lines of therapy.
- KYPROLIS® is indicated as a single agent for the treatment of adult patients with relapsed or refractory multiple myeloma who have received one or more lines of therapy.
To learn more about BeiGene, please visit www.beigene.com
KYPROLIS® is a registered trademark of Amgen Inc.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210708005989/en/
Source: BeiGene, Ltd.
BeiGene's Kyprolis OK'd in China for multiple myeloma
Jul. 09, 2021 12:10 AM ET BeiGene, Ltd. (BGNE) By: Mamta Mayani, SA News Editor
- BeiGene (NASDAQ:BGNE) announces that the China National Medical Products Administration has conditionally approved Kyprolis (carfilzomib) for injection in combination with dexamethasone for the treatment of adults with relapsed or refractory (R/R) multiple myeloma who have received at least two prior therapies.
- Kyprolis is licensed to BeiGene in China under a strategic collaboration with Amgen (NASDAQ:AMGN).
- https://seekingalpha.com/news/3713872-beigenes-kyprolis-okd-in-china-for-multiple-myeloma
- https://seekingalpha.com/symbol/BGNE
- https://seekingalpha.com/symbol/AMGN
- https://www.kyprolis.com/
- https://www.beigene.com/science-and-product-portfolio/pipeline
DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) in Combination with Pomalidomide and Dexamethasone
Janssen Announces U.S. FDA Approval of DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) in Combination with Pomalidomide and Dexamethasone for Patients with Multiple Myeloma After First or Subsequent Relapse
DARZALEX FASPRO® is now the first and only subcutaneous anti-CD38 monoclonal antibody approved in combination with pomalidomide and dexamethasone
NEWS PROVIDED BY
The Janssen Pharmaceutical Companies of Johnson & Johnson
Jul 12, 2021, 08:00 ET
SHARE THIS ARTICLE
HORSHAM, Pa., July 12, 2021 /PRNewswire/ -- The Janssen Pharmaceutical Companies of Johnson & Johnson announced today the U.S. Food and Drug Administration (FDA) approval of DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) in combination with pomalidomide and dexamethasone (Pd) for the treatment of adult patients with multiple myeloma who have received at least one prior line of therapy, including lenalidomide and a proteasome inhibitor. The approval follows the regulatory submission to the FDA in November 2020 and marks the sixth indication for DARZALEX FASPRO® in the treatment of multiple myeloma. Findings from the Phase 3 APOLLO study were presented at the 2020 American Society of Hematology (ASH) Annual Meeting and were recently published in The Lancet Oncology.
"Clinical studies including APOLLO have continued to show the ability of daratumumab-based combination treatment regimens to significantly reduce the risk of progression in patients with multiple myeloma," said Meletios A. Dimopoulos, M.D.*, Professor and Chairman of the Department of Clinical Therapeutics at the National and Kapodistrian University of Athens School of Medicine, Athens, Greece, and principal investigator. "With this approval, we are now able to combine pomalidomide and dexamethasone with a daratumumab subcutaneous option that can be administered in minutes rather than the hours needed for intravenous administration."
About the APOLLO Study1
APOLLO (NCT01960348) is an ongoing multicenter, Phase 3, randomized, open-label study comparing DARZALEX FASPRO®/ daratumumab SC in combination with pomalidomide and low-dose dexamethasone with pomalidomide and low-dose dexamethasone alone in patients with relapsed or refractory multiple myeloma who have received at least one prior treatment regimen, have received both lenalidomide and a proteasome inhibitor, and have demonstrated disease progression. The study enrolled 304 participants. The primary endpoint is PFS. Secondary endpoints include rates of overall response (ORR), very good partial response (VGPR) or better, complete response (CR) or better, and duration of response.
About DARZALEX FASPRO®
In August 2012, Janssen Biotech, Inc. and Genmab A/S entered into a worldwide agreement, which granted Janssen an exclusive license to develop, manufacture and commercialize daratumumab. DARZALEX FASPRO® is the only CD38-directed antibody approved to be given subcutaneously to treat patients with multiple myeloma and now AL amyloidosis. DARZALEX FASPRO® is co-formulated with recombinant human hyaluronidase PH20 (rHuPH20), Halozyme's ENHANZE® drug delivery technology.
DARZALEX FASPRO® is indicated for the treatment of adult patients with multiple myeloma:
- in combination with bortezomib, melphalan and prednisone in newly diagnosed patients who are ineligible for autologous stem cell transplant
- in combination with lenalidomide and dexamethasone in newly diagnosed patients who are ineligible for autologous stem cell transplant and in patients with relapsed or refractory multiple myeloma who have received at least one prior therapy
- in combination with bortezomib, thalidomide, and dexamethasone in newly diagnosed patients who are eligible for autologous stem cell transplant
- in combination with bortezomib and dexamethasone in patients who have received at least one prior therapy
- in combination with pomalidomide and dexamethasone in patients who have received at least one prior line of therapy including lenalidomide and a proteosome inhibitor
- as monotherapy, in patients who have received at least three prior lines of therapy including a proteasome inhibitor (PI) and an immunomodulatory agent or who are double-refractory to a PI and an immunomodulatory agent
DARZALEX FASPRO® in combination with bortezomib, cyclophosphamide, and dexamethasone is indicated for the treatment of adult patients with newly diagnosed light chain (AL) amyloidosis. This indication is approved under accelerated approval based on response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
Limitations of Use
DARZALEX FASPRO® is not indicated and is not recommended for the treatment of patients with light chain (AL) amyloidosis who have NYHA Class IIIB or Class IV cardiac disease or Mayo Stage IIIB outside of controlled clinical trials.
Full prescribing information for DARZALEX FASPRO® is available here.
Please see full Prescribing Information for DARZALEX FASPRO®.
SOURCE The Janssen Pharmaceutical Companies of Johnson & Johnson
FDA approves Janssen's Darzalex Faspro combo in multiple myeloma
Jul. 12, 2021 8:24 AM ETJohnson & Johnson (JNJ)By: Mamta Mayani, SA News Editor
- The Janssen Pharmaceutical Companies of Johnson & Johnson (NYSE:JNJ) announces FDA approval of Darzalex Faspro (daratumumab and hyaluronidase-fihj) in combination with pomalidomide and dexamethasone (Pd) for the treatment of adult patients with multiple myeloma who have received at least one prior line of therapy, including lenalidomide and a proteasome inhibitor.
- https://seekingalpha.com/news/3714230-fda-approves-janssens-darzalex-faspro-combo-in-multiple-myeloma
- https://seekingalpha.com/symbol/JNJ
- https://www.darzalex.com/
- https://www.jnj.com/
- https://www.janssen.com/
Filgotinib
Galapagos announces new data supporting rapid symptom improvement and sustained steroid-free remission with filgotinib in patients with Ulcerative Colitis
New post-hoc analyses of data from SELECTION Phase 3 program presented at European Crohn’s and Colitis Organisation (ECCO) virtual congress
Mechelen, Belgium; 10 July 2021, 11.10 CET; Galapagos NV (Euronext & NASDAQ: GLPG) today announced new post-hoc analyses from the Phase 3 SELECTION program, supporting the activity and tolerability of filgotinib, a once-daily, oral JAK1 preferential inhibitor, under investigation for the treatment of patients with moderately to severely active ulcerative colitis (UC). These data were presented at the European Crohn’s and Colitis Organisation (ECCO) 16th annual congress.
A post-hoc analysis of the induction study data from SELECTION showed significant improvements in patient-reported outcomes (PROs) of stool frequency (SF) and of rectal bleeding (RB), that were observed as early as the first week of therapy in patients on 200mg of filgotinib daily versus placebo in patients with moderately to severely active UC. These findings were observed in both biologic-naïve and biologic-experienced patients. More patients receiving filgotinib 200mg versus placebo achieved a composite score of RB=0 and SF≤1 as early as day 9 in Induction study A (biologic-naïve; filgotinib 200mg 18.8%, placebo 9.5%, p<0.05) and as early as day 7 in Induction study B (biologic-experienced; filgotinib 200mg 10.7%; placebo 4.2%, p<0.05).1
About the SELECTION Phase 3 Trial
The SELECTION Phase 3 trial is a multi-center, randomized, double-blind, placebo-controlled trial to assess the safety and efficacy of the preferential JAK1 inhibitor filgotinib in adult patients with moderately to severely active UC. The SELECTION trial comprises two induction trials and a maintenance trial. The Induction Study A enrolled biologic-naïve patients, and the Induction Study B enrolled biologic-experienced patients.
Across both induction studies, 1348 patients with moderately to severely active UC were randomized to receive either filgotinib 200mg, filgotinib 100mg or placebo in a 2:2:1 ratio. Moderately to severely active UC was defined as a centrally read endoscopy score ≥ 2, a rectal bleeding score ≥ 1, a stool frequency score ≥ 1 and Physician Global Assessment (PGA) of ≥ 2 based on the Mayo Clinic Score (MCS). 644 patients with clinical remission or response at Week 10 of induction were subsequently re-randomized to the induction dose of filgotinib or placebo in a 2:1 ratio and treated through Week 58.
The primary objectives of SELECTION were to evaluate the efficacy of filgotinib compared with placebo in establishing clinical remission as determined by the Mayo endoscopic subscore of 0 or 1, rectal bleeding subscore of 0, and ≥ 1-point decrease in stool frequency from baseline to achieve a subscore of 0 or 1 at Week 10 in the induction studies and Week 58 in the maintenance study. Eligible patients who were enrolled in the SELECTION trial were enrolled in the ongoing SELECTION long-term extension trial to evaluate the long-term safety of filgotinib in patients with moderately to severely active UC. A majority of patients included in the trials had a MCS of 9 or higher at baseline, and 43% of biologic experienced patients had insufficient response to a TNF antagonist and vedolizumab as well.
For SELECTION study information visit: https://clinicaltrials.gov/ct2/show/NCT02914522
About filgotinib
Filgotinib is approved and marketed as Jyseleca (200mg and 100mg tablets) in the European Union, Great Britain, and Japan for the treatment of adults with moderate to severe active rheumatoid arthritis (RA) who have responded inadequately or are intolerant to one or more disease modifying anti-rheumatic drugs (DMARDs). Filgotinib may be used as monotherapy or in combination with methotrexate (MTX). The European Summary of Product Characteristics for filgotinib, which includes contraindications and special warnings and precautions, is available at www.ema.europa.eu. The interview form from the Japanese Ministry of Health, Labour and Welfare is available at www.info.pmda.go.jp. The Great Britain and Northern Ireland Summary of Product Characteristics is available at www.medicines.org.uk/emc. Applications have been submitted to the European Medicines Agency (EMA), the UK’s Medicines and Healthcare products Regulatory Agency (MHRA), and Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) for the treatment of adults with moderately to severely active ulcerative colitis who have had an inadequate response with, lost response to, or were intolerant to either conventional therapy or a biologic agent and are currently under review. Filgotinib is not approved in any other countries.
About the filgotinib collaboration
Gilead and Galapagos NV are collaborative partners in the global development and commercialization of filgotinib. Galapagos will be responsible for the commercialization of filgotinib in Europe (transition anticipated to be completed by end of 2021), while Gilead will remain responsible for filgotinib outside of Europe, including in Japan, where filgotinib is co-marketed with Eisai. Filgotinib in UC has been filed in Europe, the UK and Japan, and a global Phase 3 program is ongoing in Crohn’s Disease. More information about clinical trials can be accessed at https://www.clinicaltrials.gov
More information at www.glpg.com.
Galapagos' filgotinib shows rapid response, sustained steroid-free remission in ulcerative colitis
Jul. 12, 2021 4:30 AM ETGalapagos NV (GLPG)By: Mamta Mayani, SA News Editor2 Comments
- Galapagos (NASDAQ:GLPG) announces new post-hoc analyses from the Phase 3 SELECTION program, supporting the activity and tolerability of filgotinib for the treatment of patients with moderately to severely active ulcerative colitis (UC).
- https://seekingalpha.com/news/3714161-galapagos-filgotinib-shows-rapid-response-sustained-steroid-free-remission-in-ulcerative-colitis
- https://seekingalpha.com/symbol/GLPG
- https://seekingalpha.com/symbol/GILD
Jardiance® (empagliflozin)
Breakthrough results for Jardiance® (empagliflozin) confirm EMPEROR-Preserved as first and only successful trial for heart failure with preserved ejection fraction
July 6, 2021Download PDF- The EMPEROR-Preserved phase III trial met its primary endpoint and demonstrated significant risk reduction with Jardiance for the composite of cardiovascular death or hospitalization for heart failure in adults with heart failure with preserved ejection fraction
- Heart failure with preserved ejection fraction has been classified as "the single largest unmet need in cardiovascular medicine"¹ based on prevalence, poor outcomes and absence of clinically proven therapies to date
- With approval, Jardiance would become the first and only clinically proven therapy to improve outcomes for the full spectrum of heart failure patients regardless of ejection fraction
RIDGEFIELD, Conn. and INDIANAPOLIS, July 6, 2021 /PRNewswire/ -- The EMPEROR-Preserved phase III trial met its primary endpoint, establishing Jardiance® (empagliflozin) as the first and only therapy to significantly reduce the risk of the composite of cardiovascular death or hospitalization for heart failure in adults, with or without diabetes, who live with heart failure with preserved ejection fraction (HFpEF). Boehringer Ingelheim and Eli Lilly and Company (NYSE: LLY) announced the topline results today. When added to the EMPEROR-Reduced trial results, these findings demonstrate Jardiance's efficacy in all forms of heart failure regardless of ejection fraction. The safety profile was generally consistent with the known safety profile of Jardiance.
The EMPEROR-Reduced results formed the basis of the recent approval of a new indication for Jardiance for the treatment of adults with HFrEF by the European Commission. In the U.S., Jardiance is not approved for the treatment of heart failure. A supplemental New Drug Application (sNDA) for Jardiance to reduce the risk of cardiovascular death or hospitalization for heart failure in adults with HFrEF has been submitted to the U.S. Food and Drug Administration (FDA), with a decision expected later this year. Research is ongoing regarding Jardiance's effects on hospitalization for heart failure and mortality in post-myocardial infarction (heart attack) patients with high risk of heart failure. Jardiance is also currently being investigated in chronic kidney disease.
About the EMPEROR Heart Failure Studies
The EMPEROR (EMPagliflozin outcomE tRial in patients with chrOnic heaRt failure) heart failure studies are two phase III, randomized, double-blind trials investigating once-daily Jardiance compared with placebo in adults with heart failure with preserved or reduced ejection fraction*, both with and without diabetes, who are receiving current standard of care:
- EMPEROR-Reduced [NCT03057977] investigated the safety and efficacy of Jardiance in patients with chronic heart failure with reduced ejection fraction (HFrEF).
- Primary endpoint: time to first event of adjudicated cardiovascular death or adjudicated hospitalization for heart failure
- Number of patients: 3,730
- Completion: 2020
- EMPEROR-Preserved [NCT03057951] investigated the safety and efficacy of Jardiance in patients with chronic heart failure with preserved ejection fraction (HFpEF).
- Primary endpoint: time to first event of adjudicated cardiovascular death or adjudicated hospitalization for heart failure [Time Frame: up to 38 months]
- Number of patients: 5,988
- Completion: 2021
*Ejection fraction is a measurement of the percentage of blood the left ventricle pumps out with each contraction. When the heart relaxes, the ventricle refills with blood.
- HFrEF occurs when the heart muscle does not contract effectively, and less blood is pumped out to the body compared with a normally functioning heart.
- HFpEF occurs when the heart muscle contracts normally but the ventricle does not fill with enough blood, so less blood can enter the heart compared with a normally functioning heart.
About the EMPOWER program
The Alliance has developed the EMPOWER program to explore the impact of Jardiance on major clinical cardiovascular and renal outcomes in a spectrum of cardio-renal-metabolic conditions. Cardio-renal-metabolic conditions are the leading cause of mortality worldwide and account for up to 20 million deaths annually. Through the EMPOWER program, Boehringer Ingelheim and Lilly are working to advance knowledge of these interconnected systems and create care which offers integrated, multi-organ benefits. Comprised of nine clinical trials and two real-world evidence studies, EMPOWER reinforces the long-term commitment of the Alliance to improve outcomes for people living with cardio-renal-metabolic conditions. With more than 400,000 adults enrolled worldwide in clinical trials, it is one of the broadest and most comprehensive clinical programs for an SGLT2 inhibitor to date.
The development program encompasses:
- EMPEROR-Reduced, in adults with chronic heart failure with reduced ejection fraction to reduce the risk of cardiovascular death or hospitalization due to heart failure
- EMPEROR-Preserved, in adults with chronic heart failure with preserved ejection fraction to reduce the risk of cardiovascular death or hospitalization due to heart failure
- EMPULSE, in adults hospitalized for acute heart failure and stabilized to improve clinical and patient reported outcomes
- EMPACT-MI, to evaluate all-cause mortality and hospitalization for heart failure in adults with and without type 2 diabetes who have had an acute myocardial infarction, with the aim to prevent heart failure and improve outcomes
- EMPA-KIDNEY, in adults with established chronic kidney disease to reduce the progression of kidney disease and the occurrence of cardiovascular death
- EMPERIAL-Reduced, in adults with chronic heart failure with reduced ejection fraction to evaluate functional ability and patient-reported outcomes
- EMPERIAL-Preserved, in adults with chronic heart failure with preserved ejection fraction to evaluate functional ability and patient-reported outcomes
- EMPA-REG OUTCOME®, in adults with type 2 diabetes and established cardiovascular disease to reduce the risk of major adverse cardiovascular events, including cardiovascular death
- EMPRISE, two non-interventional studies (U.S. and EU-Asia) of the effectiveness, safety, healthcare utilization and cost of care of empagliflozin in routine clinical practice in adults with type 2 diabetes across the cardiovascular risk continuum
What is JARDIANCE? (www.jardiance.com)
JARDIANCE is a prescription medicine used along with diet and exercise to lower blood sugar in adults with type 2 diabetes.
JARDIANCE is also used to reduce the risk of cardiovascular death in adults with type 2 diabetes who have known cardiovascular disease.
JARDIANCE is not for people with type 1 diabetes. It may increase their risk of diabetic ketoacidosis (increased ketones in the blood or urine).
JARDIANCE is not for use to lower blood sugar in adults with type 2 diabetes who have severe kidney problems, because it may not work.
For more information, please see Prescribing Information and Medication Guide.
For more information, please visit www.boehringer-ingelheim.us,
For the latest updates, visit http://www.lillydiabetes.com/
Jardiance® and EMPA-REG OUTCOME® are registered trademarks of Boehringer Ingelheim.
View original content to download multimedia:https://www.prnewswire.com/news-releases/breakthrough-results-for-jardiance-empagliflozin-confirm-emperor-preserved-as-first-and-only-successful-trial-for-heart-failure-with-preserved-ejection-fraction-301325881.html
SOURCE Eli Lilly and Company
Lilly's Jardiance successful in late-stage EMPEROR-Preserved trial for cardiovascular diseaase
Jul. 06, 2021 7:20 AM ETEli Lilly and Company (LLY)By: Mamta Mayani, SA News Editor
- Eli Lilly (NYSE:LLY) and Boehringer Ingelheim announce that the EMPEROR-Preserved phase III trial met its primary endpoint, establishing Jardiance (empagliflozin) as the first and only therapy to significantly reduce the risk of the composite of cardiovascular death or hospitalization for heart failure in adults, with or without diabetes, who live with heart failure with preserved ejection fraction (HFpEF).
- https://seekingalpha.com/news/3712811-lillys-jardiance-successful-in-late-stage-emperor-preserved-trial-for-cardiovascular-diseaase
- https://seekingalpha.com/symbol/LLY
- https://www.lilly.com/
- https://www.jardiance.com/
Tislelizumab (BGB-A317)
BeiGene Announces Acceptance of a Supplemental Biologics License Application in China for Tislelizumab in Esophageal Squamous Cell Carcinoma (ESCC)
July 7, 2021 at 8:00 PM EDT
CAMBRIDGE, Mass. & BEIJING--(BUSINESS WIRE)--Jul. 7, 2021--
BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a global biotechnology company focused on developing and commercializing innovative medicines worldwide, today announced that the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) has accepted a supplemental Biologics License Application (sBLA) for anti-PD1 antibody tislelizumab for the treatment of patients with locally advanced or metastatic esophageal squamous cell carcinoma (ESCC) who have disease progression following or are intolerant to first-line standard chemotherapy.
“We are excited to submit the eighth marketing application for tislelizumab, of which five have been approved in China. As the first global Phase 3 ESCC clinical trial, the differences in clinical practice in various countries and regions have been fully taken into account, which indicates the result is fully globally representative.” commented Yong (Ben) Ben, M.D., Chief Medical Officer, Immuno-Oncology at BeiGene. “The results demonstrated improved efficacy of tislelizumab monotherapy over chemotherapy in second-line treatment for patients with locally advanced or metastatic ESCC and the potential to benefit patients with improved overall survival (OS) compared to chemotherapy. We look forward to further communication with CDE and hope this medicine will benefit Chinese patients with locally advanced or metastatic ESCC soon.”
About Tislelizumab
Tislelizumab (BGB-A317) is a humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to FcγR on macrophages. In pre-clinical studies, binding to FcγR on macrophages has been shown to compromise the anti-tumor activity of PD-1 antibodies through activation of antibody-dependent macrophage-mediated killing of T effector cells. Tislelizumab is the first drug from BeiGene’s immuno-oncology biologics program and is being developed internationally as a monotherapy and in combination with other therapies for the treatment of a broad array of both solid tumor and hematologic cancers.
The China National Medical Products Administration (NMPA) has granted tislelizumab approval in five indications, including full approval for first-line treatment of patients with advanced squamous non-small cell lung cancer (NSCLC) in combination with chemotherapy and for first-line treatment of patients with advanced non-squamous NSCLC in combination with chemotherapy; and conditional approval for the treatment of patients with classical Hodgkin’s lymphoma (cHL) who received at least two prior therapies, for the treatment of patients with locally advanced or metastatic urothelial carcinoma (UC) with PD-L1 high expression whose disease progressed during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy, and for the treatment of patients with hepatocellular carcinoma (HCC) who have received at least one systemic therapy. Full approval for these indications is contingent upon results from ongoing randomized, controlled confirmatory clinical trials.
In addition, three supplemental Biologics License Applications for tislelizumab have been accepted by the Center for Drug Evaluation (CDE) of the NMPA and are under review for second- or third-line treatment of patients with locally advanced or metastatic NSCLC who progressed on prior platinum-based chemotherapy, for patients with previously treated, locally advanced unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair-deficient (dMMR) solid tumors and for the treatment of patients with locally advanced or metastatic esophageal squamous cell carcinoma (ESCC) who have disease progression following or are intolerant to first-line standard chemotherapy.
BeiGene has initiated or completed 17 potentially registration-enabling clinical trials in China and globally, including 13 Phase 3 trials and four pivotal Phase 2 trials.
In January 2021, BeiGene and Novartis entered into a collaboration and license agreement granting Novartis rights to develop, manufacture, and commercialize tislelizumab in North America, Europe, and Japan.
Tislelizumab is not approved for use outside of China.
About the Tislelizumab Clinical Program
Clinical trials of tislelizumab include:
- Phase 3 trial comparing tislelizumab with docetaxel in the second- or third-line setting in patients with NSCLC (NCT03358875);
- Phase 3 trial comparing tislelizumab to salvage chemotherapy in patients with relapsed or refractory classical Hodgkin Lymphoma (cHL; NCT04486391);
- Phase 3 trial in patients with locally advanced or metastatic urothelial carcinoma (NCT03967977);
- Phase 3 trial of tislelizumab in combination with chemotherapy versus chemotherapy as first-line treatment for patients with advanced squamous NSCLC (NCT03594747);
- Phase 3 trial of tislelizumab in combination with chemotherapy versus chemotherapy as first-line treatment for patients with advanced non-squamous NSCLC (NCT03663205);
- Phase 3 trial of tislelizumab in combination with platinum-based doublet chemotherapy as neoadjuvant treatment for patients with NSCLC (NCT04379635);
- Phase 3 trial of tislelizumab combined with platinum and etoposide versus placebo combined with platinum and etoposide in patients with extensive-stage small cell lung cancer (NCT04005716);
- Phase 3 trial comparing tislelizumab with sorafenib as first-line treatment for patients with hepatocellular carcinoma (HCC; NCT03412773);
- Phase 2 trial in patients with previously treated unresectable HCC (NCT03419897);
- Phase 2 trial in patients with locally advanced or metastatic urothelial bladder cancer (NCT04004221);
- Phase 3 trial comparing tislelizumab with chemotherapy as second-line treatment for patients with advanced esophageal squamous cell carcinoma (ESCC; NCT03430843);
- Phase 3 trial of tislelizumab in combination with chemotherapy as first-line treatment for patients with ESCC (NCT03783442);
- Phase 3 trial of tislelizumab versus placebo in combination with chemoradiotherapy in patients with localized ESCC (NCT03957590);
- Phase 3 trial of tislelizumab combined with chemotherapy versus placebo combined with chemotherapy as first-line treatment for patients with gastric cancer (NCT03777657);
- Phase 2 trial of tislelizumab in patients with relapsed or refractory cHL (NCT03209973);
- Phase 2 trial in patients with MSI-H/dMMR solid tumors (NCT03736889); and
- Phase 3 trial of tislelizumab combined with chemotherapy versus placebo combined with chemotherapy as first-line treatment in patients with nasopharyngeal cancer (NCT03924986).
To learn more about BeiGene, please visit www.beigene.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20210707005829/en/
Source: BeiGene, Ltd.
BeiGene's tislelizumab application accepted in China for esophageal cancer
Jul. 08, 2021 3:11 AM ET BeiGene, Ltd. (BGNE)
By: Mamta Mayani, SA News Editor
- The China National Medical Products Administration has accepted BeiGene's (NASDAQ:BGNE) supplemental Biologics License Application (sBLA) for anti-PD1 antibody tislelizumab for the treatment of locally advanced or metastatic esophageal squamous cell carcinoma (ESCC) who have disease progression following or are intolerant to first-line standard chemotherapy.
- https://seekingalpha.com/news/3713507-beigenes-tislelizumab-application-accepted-in-china-for-esophageal-cancer
- https://seekingalpha.com/symbol/BGNE
- https://www.beigene.com/science-and-product-portfolio/pipeline/tislelizumab
Tezepelumab
Tezepelumab Granted Priority Review By U.S. FDA
Tezepelumab is the First and Only Biologic to Consistently and Significantly Reduce Asthma Exacerbations in a Broad Population of Patients Across Phase 2 and 3 Clinical Trials
THOUSAND OAKS, Calif., July 7, 2021 /PRNewswire/ -- Amgen (NASDAQ:AMGN) today announced that the U.S. Food and Drug Administration (FDA) has accepted a Biologics License Application (BLA) and granted Priority Review for tezepelumab in the treatment of asthma. Tezepelumab is being developed by Amgen in collaboration with AstraZeneca.
The FDA grants Priority Review to applications for medicines that offer significant advantages over available options by demonstrating safety or efficacy improvements, preventing serious conditions or enhancing patient compliance.1 The Prescription Drug User Fee Act goal date for a decision by the FDA is during the first quarter of 2022.
Despite recent advances in severe asthma, many patients may not qualify for or respond well to current biologic medicines.2-5 Patients with severe, uncontrolled asthma experience frequent exacerbations, significant limitations on lung function and a reduced quality of life.2,6,7
Results from the NAVIGATOR Phase 3 trial were published in the New England Journal of Medicine in May 2021.
Tezepelumab was granted an FDA Breakthrough Therapy Designation for patients with severe asthma without an eosinophilic phenotype in September 2018.
About the NAVIGATOR and the PATHFINDER Clinical Trial Program
Building on the Phase 2b PATHWAY trial, the Phase 3 PATHFINDER program included two trials, NAVIGATOR8,18 and SOURCE.19,20 The program includes additional planned mechanistic and long-term safety trials.21
NAVIGATOR is a Phase 3, randomized, double-blinded, placebo-controlled trial in 1,061 adults (18–80 years old) and adolescents (12–17 years old) with severe, uncontrolled asthma, who were receiving treatment with medium- or high-dose inhaled corticosteroids (ICS) plus at least one additional controller medication with or without OCS. NAVIGATOR met the primary endpoint with tezepelumab added to standard of care (SoC) demonstrating a statistically significant and clinically meaningful reduction in the annualized asthma exacerbation rate (AAER) over 52 weeks in the overall patient population, compared to placebo added to SoC. The trial also met the primary endpoint in the subgroup of patients with baseline eosinophil counts less than 300 cells per microliter, with tezepelumab demonstrating a statistically significant and clinically meaningful reduction in AAER in that patient population. Similar reductions in AAER were observed in the subgroup of patients with baseline eosinophil counts less than 150 cells pstoper microliter.21,22
NAVIGATOR is the first Phase 3 trial to show benefit in severe asthma irrespective of eosinophils by targeting the thymic stromal lymphopoietin (TSLP). The U.S. Food and Drug Administration Breakthrough Therapy Designation was granted to tezepelumab in September 2018 for patients with severe asthma, without an eosinophilic phenotype.
SOURCE is a Phase 3 multicenter, randomized, double-blinded, parallel-group, placebo-controlled trial for 48 weeks in adult patients with severe asthma who require continuous treatment with ICS plus long-acting beta2-agonists (LABA), and chronic treatment with maintenance OCS therapy. In the trial, patients were randomized to receive tezepelumab 210 mg every four weeks or placebo as add-on therapy, with patients maintained on their currently prescribed ICS plus LABA, with or without other asthma controller therapy.
Patients who participated in the NAVIGATOR and SOURCE trials were eligible to continue in DESTINATION, a Phase 3 extension trial assessing long-term safety and efficacy.23
About Tezepelumab
Tezepelumab is being developed by AstraZeneca in collaboration with Amgen (see AstraZeneca and Amgen collaboration below) as an investigational, potential first-in-class human monoclonal antibody that works on the primary source of inflammation: the airway epithelium, which is the first point of contact for viruses, allergens, pollutants and other environmental insults. Specifically, tezepelumab targets and blocks thymic stromal lymphopoietin (TSLP), a key epithelial cytokine that sits at the top of multiple inflammatory cascades and initiates an overreactive immune response to allergic, eosinophilic and other types of airway inflammation associated with severe asthma.24,25
TSLP is released in response to multiple triggers associated with asthma exacerbations, including allergens, viruses and other airborne particles.24,25 Expression of TSLP is increased in the airways of patients with asthma and has been correlated with disease severity.24,26 Blocking TSLP may prevent the release of pro-inflammatory cytokines by immune cells, resulting in the prevention of asthma exacerbations and improved asthma control.24,26 By working at the top of the cascade, tezepelumab helps stop inflammation at the source and has the potential to treat a broad population of severe asthma patients.24,26
About the Amgen and AstraZeneca Collaboration
In 2020, Amgen and AstraZeneca updated the 2012 collaboration agreement for tezepelumab. Both companies will continue to share costs and profits equally after payment by AstraZeneca of a mid-single-digit royalty to Amgen. AstraZeneca continues to lead development and Amgen continues to lead manufacturing. All aspects of the collaboration are under the oversight of joint governing bodies. Under the amended agreement in North America, Amgen and AstraZeneca will jointly commercialize tezepelumab; Amgen will record sales in the U.S. and AstraZeneca will record sales in Canada. Outside the U.S., Amgen will record sales as collaboration revenue.
For more information, visit www.amgen.com
View original content to download multimedia:https://www.prnewswire.com/news-releases/tezepelumab-granted-priority-review-by-us-fda-301327578.html
SOURCE Amgen
FDA grants Priority Review to Amgen's tezepelumab application in asthma
Jul. 08, 2021 6:08 AM ET Amgen Inc. (AMGN)
By: Mamta Mayani, SA News Editor1 Comment
- Amgen (NASDAQ:AMGN) announces that the FDA has accepted a Biologics License Application (BLA) and granted Priority Review for tezepelumab in the treatment of asthma.
- Tezepelumab is being developed by Amgen in collaboration with AstraZeneca.
- https://seekingalpha.com/news/3713522-fda-grants-priority-review-to-amgens-tezepelumab-application-in-asthma
- https://seekingalpha.com/symbol/AMGN
- https://seekingalpha.com/symbol/AZN
- https://www.amgenpipeline.com/
KEYTRUDA® (pembrolizumab)
FDA Approves Expanded Indication for Merck’s KEYTRUDA® (pembrolizumab) in Locally Advanced Cutaneous Squamous Cell Carcinoma (cSCC)
July 6, 2021 6:45 am ET
KEYTRUDA Is Now Approved for the Treatment of Patients With Recurrent or Metastatic or Locally Advanced cSCC That Is Not Curable by Surgery or Radiation
KENILWORTH, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced that the U.S. Food and Drug Administration (FDA) has approved an expanded label for KEYTRUDA, Merck’s anti-PD-1 therapy, as monotherapy for the treatment of patients with locally advanced cutaneous squamous cell carcinoma (cSCC) that is not curable by surgery or radiation. This approval is based on data from the second interim analysis of the Phase 2 KEYNOTE-629 trial, in which KEYTRUDA demonstrated an objective response rate (ORR) of 50% (95% CI, 36-64) (n=54), including a complete response rate of 17% and a partial response rate of 33% in the cohort of patients with locally advanced disease. Among the 27 responding patients, 81% had a duration of response (DOR) of six months or longer, and 37% had a DOR of 12 months or longer. In June 2020, KEYTRUDA was granted its first indication in cSCC, as monotherapy for the treatment of patients with recurrent or metastatic disease that is not curable by surgery or radiation.
“This approval is great news for these patients and further demonstrates Merck’s commitment to the skin cancer community. KEYTRUDA has shown meaningful efficacy in patients with locally advanced or recurrent or metastatic cutaneous squamous cell carcinoma that cannot be cured by surgery or radiation,” said Dr. Vicki Goodman, vice president, clinical research, Merck Research Laboratories. “This expanded indication reinforces the role of KEYTRUDA in this cancer type, which is the second most common form of non-melanoma skin cancer.”
Data Supporting the Approval
The approval was based on data from KEYNOTE-629 (ClinicalTrials.gov, NCT03284424), a multicenter, multi-cohort, non-randomized, open-label trial that enrolled patients with recurrent or metastatic cSCC or locally advanced cSCC. The trial excluded patients with autoimmune disease or a medical condition that required immunosuppression.
Patients received KEYTRUDA 200 mg intravenously every three weeks until documented disease progression, unacceptable toxicity or a maximum of 24 months. Patients with initial radiographic disease progression could receive additional doses of KEYTRUDA during confirmation of progression unless disease progression was symptomatic, rapidly progressive, required urgent intervention, or occurred with a decline in performance status.
Assessment of tumor status was performed every six weeks during the first year and every nine weeks during the second year. The major efficacy outcome measures were ORR and DOR as assessed by blinded independent central review (BICR) according to RECIST v1.1, modified to follow a maximum of 10 target lesions and a maximum of five target lesions per organ.
Among the 54 patients with locally advanced cSCC treated, the study population characteristics were: median age of 76 years (range, 35 to 95), 80% age 65 or older; 72% male; 83% white, 13% race unknown; 41% Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0 and 59% ECOG PS of 1. Twenty-two percent received one or more prior lines of therapy; 63% received prior radiation therapy.
The ORR was 50% (95% CI, 36-64), including a complete response rate of 17% and a partial response rate of 33%, for patients treated with KEYTRUDA. After a median follow-up of 13.4 months, the median DOR had not yet been reached (range, 1.0+ to 17.2+ months). Among the 27 responding patients, 81% had a DOR of six months or longer, and 37% had a DOR of 12 months or longer.
Among the 159 patients with advanced cSCC (recurrent or metastatic or locally advanced disease) enrolled in KEYNOTE-629, the median duration of exposure to KEYTRUDA was 6.9 months (range, 1 day to 28.9 months). Adverse reactions occurring in patients with recurrent or metastatic cSCC or locally advanced cSCC were similar to those occurring in 2,799 patients with melanoma or non-small cell lung cancer treated with KEYTRUDA as a single agent. Laboratory abnormalities (Grades 3-4) that occurred at a higher incidence included lymphopenia (10%) and decreased sodium (10%).
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 1,500 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.
Selected KEYTRUDA® (pembrolizumab) Indications in the U.S.
Melanoma
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.
KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.
Non-Small Cell Lung Cancer
KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) ≥1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is stage III where patients are not candidates for surgical resection or definitive chemoradiation, or metastatic.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS ≥1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.
Head and Neck Squamous Cell Cancer
KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.
Classical Hodgkin Lymphoma
KEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).
KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.
Primary Mediastinal Large B-Cell Lymphoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.
Urothelial Carcinoma
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 (CPS ≥10), as determined by an FDA-approved test, or in patients who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
KEYTRUDA is indicated for the treatment of patients with locally advanced or mUC who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.
Microsatellite Instability-High or Mismatch Repair Deficient Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR)solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options
This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.
Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).
Gastric Cancer
KEYTRUDA, in combination with trastuzumab, fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of patients with locally advanced unresectable or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent locally advanced or metastatic gastric or GEJ adenocarcinoma whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test, with disease progression on or after two or more prior lines of therapy including fluoropyrimidine- and platinum-containing chemotherapy and if appropriate, HER2/neu-targeted therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Esophageal Cancer
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic esophageal or GEJ (tumors with epicenter 1 to 5 centimeters above the GEJ) carcinoma that is not amenable to surgical resection or definitive chemoradiation either:
- in combination with platinum- and fluoropyrimidine-based chemotherapy, or
- as a single agent after one or more prior lines of systemic therapy for patients with tumors of squamous cell histology that express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
Cervical Cancer
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Hepatocellular Carcinoma
KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Merkel Cell Carcinoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Renal Cell Carcinoma
KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma.
Tumor Mutational Burden-High Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [≥10 mutations/megabase] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.
Cutaneous Squamous Cell Carcinoma
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) or locally advanced cSCC that is not curable by surgery or radiation.
Triple-Negative Breast Cancer
KEYTRUDA, in combination with chemotherapy, is indicated for the treatment of patients with locally recurrent unresectable or metastatic triple-negative breast cancer (TNBC) whose tumors express PD-L1 (CPS ≥10) as determined by an FDA-approved test. This indication is approved under accelerated approval based on progression-free survival. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
More information is available by calling 855-257-3932 or visiting www.merckaccessprogram-keytruda.com.
For more information about our oncology clinical trials, visit www.merck.com/clinicaltrials.
For more information, visit www.merck.com
Please see Prescribing Information for KEYTRUDA (pembrolizumab) at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_pi.pdf and Medication Guide for KEYTRUDA at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_mg.pdf.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210706005062/en/
Source: Merck & Co., Inc.
FDA approves expanded indication for Merck’s Keytruda in skin cancer
Jul. 06, 2021 7:06 AM ETMerck & Co., Inc. (MRK)By: Mamta Mayani, SA News Editor5 Comments
- Merck (NYSE:MRK) announces that the FDA has approved an expanded label for Keytruda as monotherapy for the treatment of patients with locally advanced cutaneous squamous cell carcinoma (cSCC) that is not curable by surgery or radiation.
- https://seekingalpha.com/news/3712804-fda-approves-expanded-indication-for-mercks-keytruda-in-skin-cancer
- https://seekingalpha.com/symbol/MRK
- https://www.merck.com/
- https://www.keytruda.com/
Vyxeos (daunorubicin and cytarabine liposome for injection)
Health Canada Approves Vyxeos®, the First Chemotherapy Advance in over 40 years for Adults with High-risk Acute Myeloid Leukemia (AML)
July 07, 2021Download PDF
DUBLIN, July 7, 2021 /CNW/ - Jazz Pharmaceuticals plc (NASDAQ: JAZZ) today announced the Health Canada approval and availability of Vyxeos® (daunorubicin and cytarabine liposome for injection) for the treatment of adults with newly diagnosed therapy-related AML (t-AML) or AML with myelodysplasia-related changes (AML-MRC).1
About Vyxeos
Vyxeos (daunorubicin and cytarabine liposome for injection) is a chemotherapy treatment option specifically indicated for the treatment of adults with newly diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC).1
Vyxeos is administered by intravenous infusion over a period of 90 minutes.1
Vyxeos liposomes accumulate and persist in the bone marrow where they are taken up intact by leukemia cells to a greater extent than by non-leukemic bone marrow cells based on in-vivo animal studies.7
It is the first product to be developed with Jazz Pharmaceuticals' CombiPlex® technology platform, which uniquely enables the design, rapid evaluation and delivery of various combinations of therapies.8
For more information, please visit www.jazzpharmaceuticals.com
Health Canada approves Jazz Pharmaceuticals' Vyxeos to treat certain patients with leukemia
Jul. 07, 2021 8:25 AM ETJazz Pharmaceuticals plc (JAZZ)By: Aakash Babu, SA News Editor
- Health Canada has approved Jazz Pharmaceuticals' (NASDAQ:JAZZ) Vyxeos for the treatment of certain adult patients with acute myeloid leukemia (AML).
- https://seekingalpha.com/news/3713234-health-canada-approves-jazz-pharmaceuticals-vyxeos-to-treat-certain-patients-with-leukemia
- https://seekingalpha.com/symbol/JAZZ
- https://www.jazzpharma.com/
- https://vyxeos.com/
XIENCE™ stent
ABBOTT'S XIENCE STENT RECEIVES FDA APPROVAL FOR SHORTEST BLOOD THINNER COURSE FOR HIGH BLEEDING RISK PATIENTS- New labeling approval for Abbott's XIENCE™ stent builds on a legacy of safety and allows for one-month (as short as 28 days) dual anti-platelet therapy (DAPT) for patients at high bleeding risk (HBR)- The new XIENCE Skypoint™ stent received FDA approval in the U.S. and CE Mark approval in Europe- XIENCE stents have 10+ years of clinical data across 120 clinical trials, more than 125,000 patients and 15 million stent¹ implants
ABBOTT PARK, Ill., June 30, 2021 /PRNewswire/ -- Abbott (NYSE: ABT) today announced its XIENCE family of stents has received U.S. Food and Drug Administration (FDA) approval for one-month (as short as 28 days) DAPT labeling for high bleeding risk (HBR) patients in the U.S. In addition, XIENCE stents recently received CE Mark approval for DAPT as short as 28 days – giving XIENCE stents the shortest DAPT indication in the world.
In addition to the HBR indication, Abbott has also received FDA approval and European CE Mark approval for its next-generation XIENCE Skypoint stent. XIENCE Skypoint is easier to place and allows physicians to treat larger blood vessels through improved stent expansion that can open clogged vessels more effectively.
"The new FDA approval for DAPT for the XIENCE family of stents provides interventional cardiologists confidence they are delivering the best care to patients with high bleeding risk. A short DAPT duration minimizes risks for high bleeding risk patients and allows them to return to daily life sooner and with more assurance," says Roxana Mehran, M.D., professor of medicine and director of Interventional Cardiovascular Research and Clinical Trials at the Zena and Michael A. Wiener Cardiovascular Institute at Icahn School of Medicine at Mount Sinai and the global principal investigator for Abbott's Short DAPT program (XIENCE 28 and XIENCE 90).
Stents are a critical therapy option to open blood vessels that have been clogged by calcium or fatty deposits. Following stent implantation, DAPT is routinely prescribed to help prevent clotting (thrombosis), which can block a blood vessel and potentially result in heart attack or death. However, DAPT can also increase the risk of bleeding complications in some patients.2 In fact, of all patients undergoing stenting procedures, approximately one in five are considered at high risk for bleeding.3
Patients who receive stents are typically on DAPT regimens (aspirin and P2Y12 inhibitors) for six to 12 months to prevent blood clots from blocking the stented vessel. However, HBR patients can experience side effects such as bleeding during prolonged courses of DAPT. With the shortest approved DAPT labeling, Abbott's XIENCE stents can help physicians treat their HBR patients more effectively. Abbott's XIENCE 28 and XIENCE 904 studies show that DAPT can be safely discontinued early as short as 28 days with no increased risk in patient adverse events, solidifying the industry-leading safety profile of the XIENCE family of stents.
XIENCE SKYPOINT: INNOVATION FOR OPTIMAL PATIENT CARE
XIENCE Skypoint, the latest in a line of trusted XIENCE stents, is now approved for use in HBR patients with one-month DAPT labeling – as short as 28 days – in the U.S. and in Europe. Supported by 10 years of clinical data, including 120 clinical trials studying 125,000 patients and the implantation of 15 million stents, XIENCE drug-eluting stents (DES) have been proven safe and effective, consistently delivering successful patient outcomes.
XIENCE is supported by the largest body of DAPT patient evidence, with over 24,000 patients analyzed.5 Abbott continues to focus on further improving the class-leading XIENCE stent platform through innovation, supported by clinical data from the most complex patient populations, including HBR, with the aim of driving optimal care for patients suffering from coronary heart disease.
Important Safety Information: XIENCE Drug-Eluting Stents | Abbott (cardiovascular.abbott)
Connect with us at www.abbott.com
Abbott Xient stents receive FDA approval for shorter blood thinning indication
Jun. 30, 2021 10:10 AM ETAbbott Laboratories (ABT)By: Jonathan M Block, SA News Editor1 Comment
- The FDA has granted Abbott Laboratories (ABT -1.3%) approval for one-month dual antiplatelet therapy (DAPT) labeling for its Xient family of stents in high bleeding risk patients.
- The stents received CE Mark approval for the same indication in Europe as well.
- https://seekingalpha.com/symbol/ABT
- https://www.xiencestent.com/us/
biotecMAX™ April/May/June 2021 Insight
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab)
Bristol Myers Squibb Receives European Commission Approval for Opdivo (nivolumab) Plus Yervoy (ipilimumab) for the Treatment of Mismatch Repair Deficient or Microsatellite Instability–High Metastatic Colorectal Cancer After Prior Chemotherapy
06/29/2021CATEGORY:
Approval based on Phase 2 CheckMate -142 trial results showing nearly two-thirds of patients responded to the Opdivo plus Yervoy combination with durable responses observed
Opdivo plus Yervoy is the first dual immunotherapy regimen approved in the European Union for colorectal cancer
Opdivo plus Yervoy-based combinations now indicated in the European Union for five different advanced cancer types: mesothelioma, non-small cell lung cancer, melanoma, renal cell carcinoma and colorectal cancer
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) announced that the European Commission (EC) has approved Opdivo (nivolumab) plus Yervoy (ipilimumab) for the treatment of adult patients with mismatch repair deficient (dMMR) or microsatellite instability-high (MSI-H) metastatic colorectal cancer (mCRC) after prior fluoropyrimidine-based combination chemotherapy. The EC’s decision is based on results from the Phase 2 CheckMate -142 trial in which Opdivo plus Yervoy demonstrated a clinically meaningful improvement in objective response rate (ORR) in patients with MSI-H/dMMR mCRC who received prior treatment with fluoropyridine, oxaliplatin and irinotecan. The safety profile for Opdivo plus Yervoy was consistent with previous studies of the combination in other tumor types.
“Metastatic colorectal cancer is an aggressive disease with a poor prognosis, leaving patients with a critical need for additional treatment options beyond standard chemotherapy,” said Ian M. Waxman, M.D., development lead, gastrointestinal cancers, Bristol Myers Squibb. “With this approval, patients in the EU with mismatch repair deficient or microsatellite instability-high metastatic colorectal cancer will now have the first dual immunotherapy treatment available to them, and we look forward to working with stakeholders to advance this rational combination.”
The combination of Opdivo plus Yervoy is the first approved dual immunotherapy treatment option for any GI tumor in the European Union (EU). This combination is also approved in the EU for non-small cell lung cancer and renal cell carcinoma. The Marketing Authorization approves use of Opdivo plus Yervoy in all EU member states, as well as Norway, Iceland and Liechtenstein.
Opdivo plus Yervoy received approval from the U.S. Food and Drug Administration (FDA) in July 2018 for the treatment of adult and pediatric patients 12 years and older with MSI-H or dMMR mCRC that has progressed following treatment with fluoropyrimidine, oxaliplatin and irinotecan. Opdivo plus Yervoy was also approved in Japan by the Ministry of Health, Labour and Welfare (MHLW) in September 2020 for the treatment of MSI-H unresectable advanced or recurrent colorectal cancer progressing after cancer chemotherapy.
CheckMate -142 Efficacy and Safety Results
Results from the CheckMate -142 trial at minimum follow-up of 46.9 months include:
- ORR: In the study, 64.7% (95% Confidence Interval: 55.4 to 73.2) of patients responded to treatment with Opdivo plus Yervoy, with 12.6% achieving a complete response.
- DOR: Median duration of response was not reached in the Opdivoplus Yervoyarm (1.4, 58.0+ months).
- Safety: The most frequent adverse reactions, occurring in 10% or more of patients treated with Opdivo plus Yervoy, were fatigue (58%), diarrhea (41%), musculoskeletal pain (39%), rash (38%), pruritus (35%), nausea (30%), cough (29%), pyrexia (29%), abdominal pain (22%), arthralgia (22%), decreased appetite (22%), upper respiratory tract infection (21%), vomiting (21%), headache (19%), dyspnoea (19%), hypothyroidism (18%), constipation (18%), oedema (including peripheral oedema) (16%), dizziness (14%), hyperthyroidism (12%), dry skin (11%), hypertension (10%). The majority of adverse reactions were mild to moderate (Grade 1 or 2).
About CheckMate -142
CheckMate -142 included a multicenter, non-randomized, open-label cohort investigating Opdivo plus Yervoy in patients with mismatch repair deficient (dMMR) or microsatellite instability–high (MSI-H) metastatic colorectal cancer (mCRC) whose disease had progressed during or after prior treatment with a fluoropyrimidine, oxaliplatin- or irinotecan-based chemotherapy.
In this combination cohort, patients received Opdivo 3 mg/kg with Yervoy 1 mg/kg every three weeks for four doses, followed by Opdivo 3 mg/kg as a single agent every two weeks until disease progression, death or unacceptable toxicity. Efficacy outcome measures included objective response rate (ORR) as assessed by blinded independent central review using Response Evaluation Criteria in Solid Tumors (RECIST v1.1) and duration of response (DOR).
About Opdivo
Opdivo is a programmed death-1 (PD-1) immune checkpoint inhibitor that is designed to uniquely harness the body’s own immune system to help restore anti-tumor immune response. By harnessing the body’s own immune system to fight cancer, Opdivo has become an important treatment option across multiple cancers.
Opdivo’s leading global development program is based on Bristol Myers Squibb’s scientific expertise in the field of Immuno-Oncology, and includes a broad range of clinical trials across all phases, including Phase 3, in a variety of tumor types. To date, the Opdivo clinical development program has treated more than 35,000 patients. The Opdivo trials have contributed to gaining a deeper understanding of the potential role of biomarkers in patient care, particularly regarding how patients may benefit from Opdivo across the continuum of PD-L1 expression.
In July 2014, Opdivo was the first PD-1 immune checkpoint inhibitor to receive regulatory approval anywhere in the world. Opdivo is currently approved in more than 65 countries, including the United States, the European Union, Japan and China. In October 2015, the Company’s Opdivo and Yervoy combination regimen was the first Immuno-Oncology combination to receive regulatory approval for the treatment of metastatic melanoma and is currently approved in more than 50 countries, including the United States and the European Union.
INDICATIONS
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express PD-L1 (≥1%) as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab) and 2 cycles of platinum-doublet chemotherapy, is indicated for the first-line treatment of adult patients with metastatic or recurrent non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab) is indicated for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving OPDIVO.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with unresectable malignant pleural mesothelioma (MPM).
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of patients with intermediate or poor risk advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab), in combination with cabozantinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab) is indicated for the treatment of patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy.
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with classical Hodgkin lymphoma (cHL) that has relapsed or progressed after autologous hematopoietic stem cell transplantation (HSCT) and brentuximab vedotin or after 3 or more lines of systemic therapy that includes autologous HSCT. This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab) is indicated for the treatment of patients with recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN) with disease progression on or after platinum-based therapy.
OPDIVO® (nivolumab) is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of adult and pediatric (12 years and older) patients with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab) is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection.
OPDIVO® (nivolumab) is indicated for the treatment of patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) after prior fluoropyrimidine- and platinum-based chemotherapy.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of completely resected esophageal or gastroesophageal junction cancer with residual pathologic disease in patients who have received neoadjuvant chemoradiotherapy (CRT).
OPDIVO® (nivolumab), in combination with fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the treatment of patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma.
https://www.opdivo.com/about-opdivo/how-the-combination-works-combinationtherapy
Please see US Full Prescribing Information for OPDIVO and YERVOY.
For more information about Bristol Myers Squibb, visit us at BMS.com
Bristol Myers' Opdivo + Yervoy combo OK'd in Europe for colorectal cancer
Jun. 29, 2021 7:19 AM ETBristol-Myers Squibb Company (BMY)By: Mamta Mayani, SA News Editor1 Comment
- Bristol Myers Squibb (NYSE:BMY) announces that the European Commission (EC) has approved Opdivo (nivolumab) plus Yervoy (ipilimumab) for the treatment of adult patients with metastatic colorectal cancer (mCRC) after prior fluoropyrimidine-based combination chemotherapy.
- https://seekingalpha.com/news/3710849-bristol-myers-opdivo-yervoy-combo-okd-in-europe-for-colorectal-cancer
- https://seekingalpha.com/symbol/BMY
- https://www.opdivo.com/about-opdivo/how-the-combination-works-combinationtherapy
- https://www.bms.com/
Impella RP
FDA Grants Highest Level of Approval to the Next Generation of Impella RP to Treat Right Heart Failure
DANVERS, Mass.--(BUSINESS WIRE)--Jun. 29, 2021-- Abiomed’s (NASDAQ: ABMD) newest right heart pump, the Impella RP with SmartAssist, has received U.S. Food and Drug Administration (FDA) pre-market approval (PMA) as safe and effective to treat acute right heart failure for up to 14 days.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210629005367/en/
Impella RP with SmartAssist Receives FDA Pre-Market Approval (Photo: Business Wire)
Impella RP with SmartAssist is the first single-access temporary percutaneous ventricular support device with dual-sensor technology. Impella RP with SmartAssist is an innovative advancement of Impella RP, which was granted a PMA by the FDA in 2017 and has treated thousands of patients globally with right heart failure or decompensation following left ventricular assist device implantation, myocardial infarction, heart transplant, or open-heart surgery. Additionally in June 2020, the FDA issued an emergency use authorization (EUA) for Impella RP as a treatment for patients suffering from COVID-19-related right heart failure or decompensation, including pulmonary embolism (PE).
“Impella RP with SmartAssist further improves an incredibly valuable tool to treat right heart dysfunction,” said Robert Salazar, MD, interventional cardiologist, Memorial Hermann Health System Northeast Hospital and Kingwood Medical Center. “The addition of SmartAssist technology to Impella RP is an important advancement to help physicians achieve even better patient outcomes with an improved design and intuitive metrics.”
Impella RP with SmartAssist is:
- The first dual-sensor technology heart pump, providing real-time guidance and trends to help with pump management and weaning
- Designed for a simplified setup and insertion
- Enhanced with Impella Connect to enable remote monitoring from any internet-connected device through a secure, HIPAA-compliant website
SmartAssist technology is also available on Impella CP and Impella 5.5 for management of left heart failure and early identification of right heart failure.
Early identification and treatment of patients requiring right heart support is critical because, as demonstrated in Lala et al., 37% of acute myocardial infarction cardiogenic shock (AMICS) patients exhibit right heart dysfunction, which results in a significantly increased risk of mortality. When right heart failure is present, the use of Impella RP within 48 hours of cardiogenic shock onset leads to 73% survival, compared to 14% survival when implanted after 48 hours, according to a FDA PMA post-approval study presented at TCT Connect 2020.
“Early detection of right heart failure and early action is key to improving patient survival rates,” said Nishant Patel, MD, cardiothoracic surgeon at Palm Beach Gardens Medical Center. “Data demonstrates that Impella RP dramatically improves survival rates for critically ill patients in need of right heart hemodynamic support. I look forward to improving outcomes further with this new generation of Impella RP.”
The FDA indication for use of Impella RP with SmartAssist is as follows:
The Impella RP with SmartAssist System is indicated for providing temporary right ventricular support for up to 14 days in patients with a body surface area ≥1.5 m2, who develop acute right heart failure or decompensation following left ventricular assist device implantation, myocardial infarction, heart transplant, or open-heart surgery.
Impella RP with SmartAssist will be introduced in the United States through a controlled rollout at hospitals that follow cardiogenic shock best practice protocols.
ABOUT IMPELLA HEART PUMPS
The Impella RP® and the Impella RP® with SmartAssist® System are U.S. FDA approved to provide temporary right ventricular support for up to 14 days in patients with a body surface area ≥1.5 m², who develop acute right heart failure or decompensation following left ventricular assist device implantation, myocardial infarction, heart transplant, or open-heart surgery.
The Impella RP is also authorized for emergency use by healthcare providers (HCPs) in the hospital setting for providing temporary right ventricular support for up to 14 days in critical care patients with a body surface area ≥1.5 m², for the treatment of acute right heart failure or decompensation caused by complications related to coronavirus disease 2019 (COVID‐19), including pulmonary embolism (PE). The Impella RP has not been cleared or approved for the treatment of acute right heart failure or decompensation caused by complications related to COVID-19. The Impella RP has been authorized for the above emergency use by FDA under an EUA and has been authorized only for the duration of the declaration that circumstances exist justifying the authorization of the emergency use of medical devices under section 564(b)(1) of the Act, 21 U.S.C. § 360bbb-3(b)(1), unless the authorization is terminated or revoked sooner.
For additional information, please visit: www.abiomed.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210629005367/en/
Source: Abiomed, Inc.
Abiomed wins FDA approval for Impella heart pump
Jun. 29, 2021 8:36 AM ET Abiomed, Inc. (ABMD)
By: Dulan Lokuwithana, SA News Editor2 Comments
- Abiomed (NASDAQ:ABMD) announced that the FDA issued the pre-market approval (PMA) for the right heart pump, the Impella RP with SmartAssist as a potential treatment of acute right heart failure for up to 14 days.
- https://seekingalpha.com/symbol/ABMD
- https://www.heartrecovery.com/products-and-services/impella/impella-rp
- https://www.abiomed.com/
Upadacitinib (RINVOQ®)
June 29, 2021
Upadacitinib (RINVOQ®) Met the Primary and All Secondary Endpoints in the 52-Week Phase 3 Maintenance Study in Ulcerative Colitis Patients
- Significantly more upadacitinib-treated (15 mg or 30 mg, once daily) patients achieved the primary endpoint of clinical remission (per Adapted Mayo Score) compared to patients on placebo at one-year (week 52) (p<0.001)[1]
- All secondary endpoints were met, including the achievement of endoscopic improvement, HEMI and corticosteroid-free clinical remission at one-year (p<0.001)[1]
- Safety results were consistent with the previous Phase 3 induction studies and the known safety profile of upadacitinib, with no new safety risks observed[1-6]
- Upadacitinib, a selective and reversible JAK inhibitor discovered and developed by AbbVie, is being studied as an oral therapy for moderate to severe ulcerative colitis and several other immune-mediated inflammatory diseases[1,7-14]
NORTH CHICAGO, Ill., June 29, 2021 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced that upadacitinib (15 mg or 30 mg, once daily) met the primary endpoint of clinical remission (per Adapted Mayo Score) and all secondary endpoints at one-year (week 52) in the Phase 3 ulcerative colitis maintenance study.1 Significantly more upadacitinib-treated patients achieved clinical remission at week 52 compared to placebo (15 mg: 42 percent and 30 mg: 52 percent versus placebo: 12 percent; p<0.001).1
"Ulcerative colitis is a disease with unpredictable symptoms and frequent flares, which can make daily life challenging," said Michael Severino, M.D., vice chairman and president, AbbVie. "We are encouraged by these results that demonstrate upadacitinib's potential as a treatment option for patients with moderate to severe ulcerative colitis."
In this study, adults with moderate to severe ulcerative colitis who achieved a clinical response (per partial Adapted Mayo Score) following an 8-week study period of once-daily upadacitinib (45 mg) induction treatment were re-randomized to receive upadacitinib 15 mg, upadacitinib 30 mg or placebo for an additional 52 weeks.1
All secondary endpoints were met, including the achievement of endoscopic improvement, histologic-endoscopic mucosal improvement (HEMI) and corticosteroid-free clinical remission at week 52.1 49 percent of patients treated with upadacitinib 15 mg and 62 percent of patients treated with upadacitinib 30 mg achieved endoscopic improvement at 52 weeks versus 14 percent of patients in the placebo group (p<0.001).1 In addition, 35 percent of patients on upadacitinib 15 mg and 49 percent of patients on upadacitinib 30 mg achieved HEMI compared to 12 percent of patients in the placebo group (p<0.001).1 Of patients who were in remission at the completion of the 8-week induction studies, corticosteroid-free remission was achieved in 57 percent of patients in the upadacitinib 15 mg group and 68 percent of patients in the upadacitinib 30 mg group compared to 22 percent of patients in the placebo group (p<0.001).1
About the Phase 3 Maintenance Study1,9
The Phase 3 maintenance study is an ongoing, Phase 3 multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of upadacitinib in patients with moderate to severe ulcerative colitis. Results from the induction studies, U-ACHIEVE and U-ACCOMPLISH, were announced in December 2020 and February 2021, respectively. The objective of this maintenance study is to evaluate the efficacy and safety of upadacitinib 15 mg and 30 mg, once daily, as a maintenance therapy compared to the placebo group.
The primary endpoint was achievement of clinical remission (per Adapted Mayo Score) at week 52. Secondary endpoints included achievement of endoscopic improvement, HEMI and corticosteroid-free clinical remission at one-year. More information can be found on www.clinicaltrials.gov (NCT02819635).
About the Upadacitinib Ulcerative Colitis Program9,20,21
The global upadacitinib ulcerative colitis program evaluates more than 1,300 patients with moderately to severely active ulcerative colitis across three pivotal studies. These studies include assessments of efficacy and safety of upadacitinib. Key measures of efficacy include clinical remission (per Adapted Mayo Score), clinical response (per Adapted Mayo Score), endoscopic improvement and endoscopic response. More information on these trials can be found at www.clinicaltrials.gov (NCT02819635, NCT03653026, NCT03006068).
About Upadacitinib (RINVOQ®)
Discovered and developed by AbbVie scientists, RINVOQ is a selective and reversible JAK inhibitor that is being studied in several immune-mediated inflammatory diseases.7-14 In human cellular assays, RINVOQ preferentially inhibits signaling by JAK1 or JAK1/3 with functional selectivity over cytokine receptors that signal via pairs of JAK2.7 In August 2019, RINVOQ received U.S. FDA approval for adult patients with moderately to severely active rheumatoid arthritis who have had an inadequate response or intolerance to methotrexate. RINVOQ is approved by the European Commission for the treatment of adult patients with moderate to severe active rheumatoid arthritis who have responded inadequately to, or who are intolerant to one or more disease-modifying anti-rheumatic drugs (DMARDs); for the treatment of active psoriatic arthritis (PsA) in adult patients who have responded inadequately to, or who are intolerant to one or more DMARDs; and for the treatment of active ankylosing spondylitis (AS) in adult patients who have responded inadequately to conventional therapy. The approved dose for RINVOQ is 15 mg. Phase 3 trials of RINVOQ in atopic dermatitis, axial spondyloarthritis, Crohn's disease, ulcerative colitis, giant cell arteritis and Takayasu arteritis are ongoing.9-14 Use of RINVOQ in ulcerative colitis is not approved and its safety and efficacy have not been evaluated by regulatory authorities.
For more information about AbbVie, please visit us at www.abbvie.com
AbbVie's upadacitinib successful in late-stage ulcerative colitis maintenance study
Jun. 29, 2021 8:21 AM ETAbbVie Inc. (ABBV)By: Mamta Mayani, SA News Editor6 Comments
- AbbVie (NYSE:ABBV) announces that upadacitinib (Rinvoq) (15 mg or 30 mg, once daily) met the primary and all secondary endpoints in Phase 3 ulcerative colitis maintenance study.
- https://seekingalpha.com/news/3710897-abbvies-upadacitinib-successful-in-late-stage-ulcerative-colitis-maintenance-study
- https://seekingalpha.com/symbol/ABBV
- https://www.abbvie.com/
- https://www.rinvoq.com/
KEYTRUDA® (pembrolizumab) Plus Chemotherapy
European Commission Approves Merck’s KEYTRUDA® (pembrolizumab) Plus Chemotherapy for Certain Patients With Esophageal Cancer or HER2-Negative Gastroesophageal Junction (GEJ) Adenocarcinoma
June 29, 2021 6:45 am ET
KEYTRUDA is Now Approved in Combination With Platinum- and Fluoropyrimidine-Based Chemotherapy As First-Line Treatment for Certain Patients With Locally Advanced Unresectable or Metastatic Esophageal Carcinoma or HER2-Negative GEJ Adenocarcinoma Whose Tumors Express PD-L1 (CPS ≥10)
KEYTRUDA is First Anti-PD-1 Therapy Approved in Europe in Combination With Chemotherapy for First-Line Treatment of Advanced Esophageal or GEJ Cancer, Regardless of Histology
KENILWORTH, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced that the European Commission (EC) has approved KEYTRUDA, Merck’s anti-PD-1 therapy, in combination with platinum- and fluoropyrimidine-based chemotherapy for the first-line treatment of patients with locally advanced unresectable or metastatic carcinoma of the esophagus or human epidermal growth factor receptor 2 (HER2)-negative gastroesophageal junction (GEJ) adenocarcinoma in adults whose tumors express PD-L1 (Combined Positive Score [CPS] ≥10).
This approval is based on results from the Phase 3 KEYNOTE-590 trial, in which KEYTRUDA plus 5-fluorouracil (5-FU) and cisplatin demonstrated statistically significant improvements in overall survival (OS) and progression-free survival (PFS) compared with 5-FU and cisplatin alone in all pre-specified study populations. KEYTRUDA plus 5-FU and cisplatin reduced the risk of death by 27% (HR=0.73 [95% CI, 0.62-0.86]; p<0.0001) and reduced the risk of disease progression or death by 35% (HR=0.65 [95% CI, 0.55-0.76]; p<0.0001) versus 5-FU and cisplatin alone. In a pre-specified analysis of patients whose tumors expressed PD-L1 (CPS ≥10) (n=383/749), KEYTRUDA plus 5-FU and cisplatin reduced the risk of death by 38% (HR=0.62 [95% CI, 0.49-0.78]; p<0.0001) and reduced the risk of disease progression or death by 49% (HR=0.51 [95% CI, 0.41-0.65]; p<0.0001) versus 5-FU and cisplatin alone. For patients treated with KEYTRUDA plus 5-FU and cisplatin, the objective response rate (ORR) was 51.1% (95% CI, 43.7-58.5), with a complete response (CR) rate of 5.9% and a partial response (PR) rate of 45.2%, versus 26.9% (95% CI, 20.8-33.7), with a CR rate of 2.5% and a PR rate of 24.4%, for patients treated with 5-FU and cisplatin alone (p<0.0001).
“KEYTRUDA plus chemotherapy is the first anti-PD1 therapy approved in Europe in this first-line setting, allowing these patients to be treated with immunotherapy earlier in the course of their treatment,” said Dr. Scot Ebbinghaus, vice president, clinical research, Merck Research Laboratories.
This approval allows marketing of the KEYTRUDA combination in all 27 European Union member states plus Iceland, Lichtenstein, Norway and Northern Ireland.
Merck is continuing to study KEYTRUDA across multiple settings and stages of gastrointestinal cancer through its broad clinical program, including studies in esophageal, gastric, hepatobiliary, pancreatic, colorectal and anal cancers.
Data Supporting the European Approval
The approval was based on data from KEYNOTE-590 (NCT03189719), a multicenter, randomized, double-blind placebo-controlled trial that enrolled 749 patients with locally advanced unresectable or metastatic esophageal or GEJ carcinoma (Siewert Type I) who were not candidates for surgical resection or definitive chemoradiation. Patients were randomized (1:1) to receive either KEYTRUDA (200 mg on Day 1 every three weeks) or placebo (on Day 1 every three weeks) in combination with cisplatin (80 mg/m2 on Day 1 every three weeks for up to six cycles) plus 5-FU (800 mg/m2 per day on Days 1 to 5 every three weeks, or per local standard for 5-FU administration, for up to 24 months); all study medications were administered via intravenous infusion.
Randomization was stratified by tumor histology (squamous cell carcinoma vs. adenocarcinoma), geographic region (Asia vs. ex-Asia), and Eastern Cooperative Oncology Group (ECOG) performance status (PS) (0 vs. 1).
Treatment with KEYTRUDA or chemotherapy continued until unacceptable toxicity or disease progression or a maximum of 24 months. Patients could be treated with KEYTRUDA for up to 24 months in the absence of disease progression. Patients randomized to KEYTRUDA were permitted to continue beyond the first RECIST v1.1-defined disease progression if clinically stable until the first radiographic evidence of disease progression was confirmed at least four weeks later with repeat imaging. Assessment of tumor status was performed every nine weeks.
The primary efficacy outcome measures were OS and PFS, as assessed by the investigator according to RECIST v1.1 in squamous cell histology, PD-L1 expression (CPS ≥10), and in all patients. Secondary efficacy outcome measures were ORR and duration of response (DOR), as assessed by the investigator according to RECIST v1.1.
In a pre-specified analysis of KEYNOTE-590 in patients whose tumors expressed PD-L1 (CPS ≥10) (n=383), performed at a median follow-up time of 13.5 months (range, 0.5 to 32.7), KEYTRUDA plus 5-FU and cisplatin showed a median OS of 13.5 months (95% CI, 11.1-15.6) versus 9.4 months (95% CI, 8.0-10.7) for patients treated with 5-FU and cisplatin alone. Additionally, KEYTRUDA plus 5-FU and cisplatin showed a median PFS of 7.5 months (95% CI, 6.2-8.2) versus 5.5 months (95% CI, 4.3-6.0) for patients treated with 5-FU and cisplatin alone. Median DOR was 10.4 months (range, 1.9 to 28.9+) with KEYTRUDA plus 5-FU and cisplatin versus 5.6 months (range, 1.5+ to 25+) with 5-FU and cisplatin alone.
The safety of KEYTRUDA in combination with chemotherapy has been evaluated in 1,437 patients with non-small cell lung cancer (NSCLC) or head and neck squamous cell carcinoma (HNSCC) or esophageal carcinoma receiving 200 mg, 2 mg/kg bodyweight (bw) or 10 mg/kg bw KEYTRUDA every 3 weeks, in clinical studies. In this patient population, the most frequent adverse reactions were nausea (55%), anemia (51%), fatigue (39%), constipation (37%), diarrhea (33%), neutropenia (29%) and vomiting (28%). Incidences of Grades 3-5 adverse reactions in patients with NSCLC were 67% for KEYTRUDA combination therapy and 66% for chemotherapy alone; in patients with HNSCC were 85% for KEYTRUDA combination therapy and 84% for chemotherapy plus cetuximab; and in patients with esophageal carcinoma were 86% for KEYTRUDA combination therapy and 83% for chemotherapy alone.
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 1,500 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.
Selected KEYTRUDA® (pembrolizumab) Indications in the U.S.
Melanoma
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.
KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.
Non-Small Cell Lung Cancer
KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic NSCLC, with no EGFR or ALK genomic tumor aberrations.
KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) ≥1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is stage III where patients are not candidates for surgical resection or definitive chemoradiation, or metastatic.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS ≥1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.
Head and Neck Squamous Cell Cancer
KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, HNSCC.
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.
Classical Hodgkin Lymphoma
KEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).
KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.
Primary Mediastinal Large B-Cell Lymphoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.
Urothelial Carcinoma
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 (CPS ≥10), as determined by an FDA-approved test, or in patients who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
KEYTRUDA is indicated for the treatment of patients with locally advanced or mUC who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.
Microsatellite Instability-High or Mismatch Repair Deficient Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR)
- solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options, or
- colorectal cancer that has progressed following treatment with fluoropyrimidine, oxaliplatin, and irinotecan.
This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.
Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer
KEYTRUDA is indicated for the first-line treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).
Gastric Cancer
KEYTRUDA, in combination with trastuzumab, fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of patients with locally advanced unresectable or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent locally advanced or metastatic gastric or GEJ adenocarcinoma whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test, with disease progression on or after two or more prior lines of therapy including fluoropyrimidine- and platinum-containing chemotherapy and if appropriate, HER2/neu-targeted therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Esophageal Cancer
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic esophageal or GEJ (tumors with epicenter 1 to 5 centimeters above the GEJ) carcinoma that is not amenable to surgical resection or definitive chemoradiation either:
- in combination with platinum- and fluoropyrimidine-based chemotherapy, or
- as a single agent after one or more prior lines of systemic therapy for patients with tumors of squamous cell histology that express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
Cervical Cancer
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Hepatocellular Carcinoma
KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Merkel Cell Carcinoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Renal Cell Carcinoma
KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma.
Tumor Mutational Burden-High Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [≥10 mutations/megabase] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.
Cutaneous Squamous Cell Carcinoma
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) that is not curable by surgery or radiation.
Triple-Negative Breast Cancer
KEYTRUDA, in combination with chemotherapy, is indicated for the treatment of patients with locally recurrent unresectable or metastatic triple-negative breast cancer (TNBC) whose tumors express PD-L1 (CPS ≥10) as determined by an FDA-approved test. This indication is approved under accelerated approval based on progression-free survival. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Merck's Keytruda + chemo combo OK'd in Europe for esophageal/gastric cancer
Jun. 29, 2021 7:12 AM ET Merck & Co., Inc. (MRK) By: Mamta Mayani, SA News Editor4 Comments
- The European Commission (EC) has approved Merck's (NYSE:MRK) Keytruda in combination with platinum- and fluoropyrimidine-based chemotherapy for the first-line treatment of patients with esophageal cancer or HER2-negative gastroesophageal junction (GEJ) adenocarcinoma in adults whose tumors express PD-L1.
- https://seekingalpha.com/news/3710846-mercks-keytruda-chemo-combo-okd-in-europe-for-esophagealgastric-cancer
- https://seekingalpha.com/symbol/MRK
- https://www.keytruda.com/
- https://www.merck.com/
Soliqua® (insulin glargine 100 Units/mL and lixisenatide, iGlarLixi)
June 28 2021
New Soliqua® data shows improved blood sugar control without weight gain versus premixed insulin
New Soliqua®* data shows improved blood sugar control without weight gain versus premixed insulin
- Study also shows more people on Soliqua had improved blood sugar control without weight gain and without low blood sugar events (hypoglycemia) in first head-to-head comparison with premixed insulin
JUNE 28, 2021
A new study of Soliqua (insulin glargine 100 Units/mL and lixisenatide, iGlarLixi) met its two primary endpoints and all key secondary endpoints in a head-to-head comparison against premixed insulin (biphasic insulin aspart 30, BIAsp 30) in adults living with type 2 diabetes, the most common form of diabetes, uncontrolled on insulin and one or two oral anti-diabetic medicines. The findings were presented today at the American Diabetes Association (ADA) 81st Scientific Sessions1 and simultaneously published in Diabetes Care.2
The study met both primary endpoints with Soliqua demonstrating noninferiority of blood sugar (HbA1c) reduction and superiority on body weight change from baseline compared to premixed insulin. The study also met its key secondary endpoints with Soliqua achieving a greater proportion of people reaching a HbA1c target of <7% without weight gain, a greater proportion of people reaching a HbA1c target of <7% without weight gain and without hypoglycemia, and superiority in reduction of HbA1c compared with those using premixed insulin.
About the SoliMix study
The SoliMix study was a 26-week, randomized controlled trial of 887 adults living with type 2 diabetes who were uncontrolled on insulin plus metformin with or without a sodium-glucose cotransporter-2 inhibitor (SGLT-2i). The study compared the efficacy and safety of Soliqua compared to a commonly used premixed insulin (BIAsp 30). Participants were randomized to switch from their prior insulin to either Soliqua once daily or premixed insulin twice daily, with starting doses determined and adjusted weekly. Any metformin or SGLT-2i treatment was maintained through the study period.
The study met its two primary endpoints and all three of its key secondary endpoints.
- TRIM-D results demonstrated greater improvements for participants using Soliqua compared to those using premixed insulin, across domains including compliance, diabetes management, and psychological health, among others.3
- GTEE findings showed that nearly twice as many patients reported complete control of their diabetes with Soliqua compared to premixed insulin (27.5% vs 15%). Similarly, nearly twice as many physicians also reported treatment effectiveness (i.e., complete control of diabetes) with Soliqua compared to premixed insulin (29.9% vs 15.3%).3
Safety findings were in line with the established profiles of both treatments. The most commonly reported adverse events in the study were hypoglycemia (31.2% Soliqua, 42.4% premixed insulin), nausea (7.7% Soliqua, 0% premixed insulin), headache (2.5% Soliqua, 0.5% premixed insulin), dizziness (1.4% Soliqua, 0.5% premixed insulin), and vomiting (1.1% Soliqua, 0.2% premixed insulin).
* Soliqua® is an injectable prescription medicine that contains two diabetes medicines, insulin glargine and lixisenatide. Soliqua® is marketed in the EU as Suliqua®, where it is indicated in combination with metformin for the treatment of adults with type 2 diabetes mellitus to improve glycemic control when this has not been provided by metformin alone or metformin combined with another oral glucose lowering medicinal product or with basal insulin. It is marketed in the U.S. as Soliqua® 100/33, where it is indicated as an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes mellitus. It is marketed as Soliqua® in other geographies where it is approved.
https://www.soliqua100-33.com/
ENSPRYNG® (satralizumab)
Roche’s ENSPRYNG approved by European Commission as first and only at-home subcutaneous treatment for neuromyelitis optica spectrum disorder (NMOSD)
- ENSPRYNG is the first and only treatment approved for both adults and adolescents in the EU with AQP4-IgG seropositive NMOSD
- ENSPRYNG can be used as a monotherapy or in combination with immunosuppressive therapy to reduce relapses and prevent permanent disability
- In Phase III studies, ENSPRYNG significantly reduced the number and severity of relapses in people with AQP4-IgG seropositive NMOSD
Basel, 28 June 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the European Commission (EC) has approved ENSPRYNG® (satralizumab) for the treatment of adults and adolescents from 12 years of age living with anti-aquaporin-4 antibody (AQP4-IgG) seropositive neuromyelitis optica spectrum disorder (NMOSD), as a monotherapy or in combination with immunosuppressive therapy (IST). ENSPRYNG is the first and only NMOSD treatment that is administered subcutaneously every four weeks, allowing home-dosing after appropriate training.
ENSPRYNG is the first and only approved medicine for NMOSD in the EU designed to bind to and block the interleukin-6 (IL-6) receptor, a central driver of the inflammation associated with NMOSD. The treatment was designed by Chugai, a member of the Roche Group, using novel recycling antibody technology. When compared to conventional antibodies, ENSPRYNG’s recycling antibody technology enables the medicine to remain in the bloodstream for a longer period of time and bind repeatedly to its target (the IL-6 receptor) – maximally sustaining IL-6 suppression in a chronic disease like NMOSD and enabling subcutaneous dosing every four weeks.
Roche is working closely with reimbursement and health technology assessment bodies in EU member states to provide access to ENSPRYNG for people who may benefit from this treatment option as soon as possible.
About SAkuraStar and SAkuraSky in NMOSD
ENSPRYNG has been investigated in two pivotal Phase III studies in neuromyelitis optica spectrum disorder (NMOSD), with the primary endpoint of both studies being time to first protocol-defined relapse (PDR) adjudicated by an independent review committee in the double-blind period.
The Phase III SAkuraStar study evaluated the efficacy and safety of ENSPRYNG monotherapy administered to adults with NMOSD. In the anti-aquaporin-4 antibody (AQP4-IgG) seropositive subgroup, 83% treated with ENSPRYNG remained relapse free at 48 weeks, compared with 55% of those treated with placebo. At 96 weeks, 77% of those treated with ENSPRYNG remained relapse free, compared with 41% with placebo.
The Phase III SAkuraSky study evaluated the efficacy and safety of ENSPRYNG in combination with baseline immunosuppressive therapy in adults and adolescents with NMOSD. Overall, 92% of AQP4-IgG seropositive participants receiving ENSPRYNG in combination with IST remained relapse free at 48 and 96 weeks, compared with 60% and 53% with placebo, respectively.
ENSPRYNG showed a favourable safety and tolerability profile in the Phase III studies. The most common adverse reactions observed in the safety population were: headache, arthralgia, white blood cell count decrease, hyperlipidaemia and injection-related reactions.
About ENSPRYNG® (satralizumab)
ENSPRYNG, which was designed by Chugai, a member of the Roche Group, is a humanised monoclonal antibody that targets interleukin-6 (IL-6) receptor activity. The cytokine IL-6 is believed to be a key driver in NMOSD disease processes, triggering the inflammation cascade and leading to damage and disability. ENSPRYNG was designed using novel recycling antibody technology. When compared to conventional antibodies, ENSPRYNG’s recycling antibody technology enables the medicine to remain in the bloodstream for a longer period of time and bind repeatedly to its target (the IL-6 receptor) - maximally sustaining IL-6 suppression in a chronic disease like NMOSD and enabling subcutaneous dosing every four weeks.
Positive Phase III results for ENSPRYNG, as both monotherapy and in combination with baseline immunosuppressive therapy, suggest that IL-6 inhibition is an effective therapeutic approach for NMOSD. The Phase III clinical development programme for ENSPRYNG included two studies: SAkuraStar and SAkuraSky.
ENSPRYNG is currently approved in 54 countries, including the United States, Canada, Japan, China and EMA territory countries.
ENSPRYNG has been designated as an orphan drug in the U.S., Europe and Japan. In addition, it was granted Breakthrough Therapy Designation for the treatment of NMOSD by the FDA in December 2018, which is given to treatments that may demonstrate substantial improvement over other available options.
Roche’s Enspryng OK'd in Europe as first at-home treatment for neuromyelitis optica spectrum disorder
Jun. 28, 2021 3:06 AM ETRoche Holding AG (RHHBY), CHGCYBy: Mamta Mayani, SA News Editor
- Roche (OTCQX:RHHBY) has been granted Marketing Authorization from the European Commission for Enspryng (satralizumab), created by Chugai Pharmaceutical (OTCPK:CHGCF), as the first subcutaneous treatment option for adults and adolescents from 12 years of age living with anti-aquaporin-4 antibody (AQP4-IgG) seropositive neuromyelitis optica spectrum disorder (NMOSD), as a monotherapy or in combination with immunosuppressive therapy.
- https://seekingalpha.com/news/3710335-roches-enspryng-okd-in-europe-as-first-at-home-treatment-for-neuromyelitis-optica-spectrum-disorder
- https://seekingalpha.com/symbol/RHHBY
- https://seekingalpha.com/symbol/CHGCF
- https://www.enspryng.com/
Opdivo (nivolumab)
Bristol Myers Squibb Receives Positive CHMP Opinion for Opdivo (nivolumab) as Adjuvant Treatment for Esophageal or Gastroesophageal Junction Cancer Patients with Residual Pathologic Disease Following Chemoradiotherapy
06/25/2021CATEGORY:
Recommendation based on positive results from the Phase 3 CheckMate -577 trial in which Opdivo doubled disease-free survival compared to placebo in the all-randomized population
If approved, Opdivo could be the first and only adjuvant therapeutic option in the European Union that has shown a significant improvement in disease-free survival for patients in this setting
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 Opdivo (nivolumab) for the adjuvant treatment of adult patients with esophageal or gastroesophageal junction (GEJ) cancer who have residual pathologic disease following prior neoadjuvant chemoradiotherapy (CRT). The European Commission (EC), which has the authority to approve medicines for the European Union (EU), will now review the CHMP recommendation.
“For many patients with localized esophageal or gastroesophageal junction cancer, the risk of recurrence is high, even after neoadjuvant chemoradiotherapy and surgery. This leaves patients in need of additional treatment options,” said Ian M. Waxman, M.D., development lead, gastrointestinal cancers, Bristol Myers Squibb. “We believe that the use of immunotherapy in earlier stages of cancer is important because of its potential to prevent recurrence. The CHMP’s positive recommendation for Opdivo as an adjuvant treatment for esophageal or gastroesophageal junction cancer represents a step forward for people living with these cancers as we see the science translate into outcomes.”
The positive opinion is based on results from the Phase 3 CheckMate -577 trial which showed that treatment with Opdivo following neoadjuvant CRT and complete surgical resection doubled the primary endpoint of disease-free survival (DFS) compared to placebo in the all-randomized population. The safety profile of Opdivo was consistent with previously reported studies. Results from CheckMate -577 were presented at the 2020 European Society for Medical Oncology (ESMO) Virtual Congress in September 2020 and at the American Society of Clinical Oncology (ASCO) Annual Meeting in June 2021.
Opdivo is approved in the United States for the adjuvant treatment of completely resected esophageal or GEJ cancer with residual pathologic disease in patients who have received neoadjuvant CRT.
Bristol Myers Squibb thanks the patients and investigators involved in the CheckMate -577 trial.
About CheckMate -577
CheckMate -577 is a Phase 3 randomized, multi-center, double-blind study evaluating Opdivo as an adjuvant therapy in patients with resected esophageal or gastroesophageal junction (GEJ) cancer who have received neoadjuvant chemoradiotherapy (CRT) and have not achieved a pathological complete response. The primary endpoint of the trial is disease-free survival (DFS) and the secondary endpoint is overall survival (OS). Following neoadjuvant CRT and complete tumor surgical resection (also known as trimodality therapy), a total of 794 patients were randomized to receive placebo (n=262) or Opdivo (n=532) 240 mg by intravenous infusion every two weeks for 16 weeks followed by placebo or Opdivo 480 mg every four weeks until disease recurrence, unacceptable toxicity or withdrawal of consent, with a maximum of one-year total treatment duration. Follow up for OS is ongoing.
About Opdivo
Opdivo is a programmed death-1 (PD-1) immune checkpoint inhibitor that is designed to uniquely harness the body’s own immune system to help restore anti-tumor immune response. By harnessing the body’s own immune system to fight cancer, Opdivo has become an important treatment option across multiple cancers.
Opdivo’s leading global development program is based on Bristol Myers Squibb’s scientific expertise in the field of Immuno-Oncology and includes a broad range of clinical trials across all phases, including Phase 3, in a variety of tumor types. To date, the Opdivo clinical development program has treated more than 35,000 patients. The Opdivo trials have contributed to gaining a deeper understanding of the potential role of biomarkers in patient care, particularly regarding how patients may benefit from Opdivo across the continuum of PD-L1 expression.
In July 2014, Opdivo was the first PD-1 immune checkpoint inhibitor to receive regulatory approval anywhere in the world. Opdivo is currently approved in more than 65 countries, including the United States, the European Union, Japan and China. In October 2015, the Company’s Opdivo and Yervoy combination regimen was the first Immuno-Oncology combination to receive regulatory approval for the treatment of metastatic melanoma and is currently approved in more than 50 countries, including the United States and the European Union.
INDICATIONS
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express PD-L1 (≥1%) as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab) and 2 cycles of platinum-doublet chemotherapy, is indicated for the first-line treatment of adult patients with metastatic or recurrent non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab) is indicated for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving OPDIVO.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with unresectable malignant pleural mesothelioma (MPM).
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of patients with intermediate or poor risk advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab), in combination with cabozantinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab) is indicated for the treatment of patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy.
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with classical Hodgkin lymphoma (cHL) that has relapsed or progressed after autologous hematopoietic stem cell transplantation (HSCT) and brentuximab vedotin or after 3 or more lines of systemic therapy that includes autologous HSCT. This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab) is indicated for the treatment of patients with recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN) with disease progression on or after platinum-based therapy.
OPDIVO® (nivolumab) is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of adult and pediatric (12 years and older) patients with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab) is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection.
OPDIVO® (nivolumab) is indicated for the treatment of patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) after prior fluoropyrimidine- and platinum-based chemotherapy.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of completely resected esophageal or gastroesophageal junction cancer with residual pathologic disease in patients who have received neoadjuvant chemoradiotherapy (CRT).
OPDIVO® (nivolumab), in combination with fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the treatment of patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma.
Please see US Full Prescribing Information for OPDIVO and YERVOY
For more information about Bristol Myers Squibb, visit us at BMS.com
Bristol Myers wins positive CHMP opinion for Opdivo in esophageal cancer
Jun. 25, 2021 7:40 AM ET Bristol-Myers Squibb Company (BMY) By: Mamta Mayani, SA News Editor
- Bristol Myers Squibb (NYSE:BMY) reports that EMA's Committee for Medicinal Products for Human Use (CHMP) has recommended approval of Opdivo (nivolumab) for the adjuvant treatment of adult patients with esophageal or gastroesophageal junction (GEJ) cancer who have residual pathologic disease following prior neoadjuvant chemoradiotherapy (CRT).
- https://seekingalpha.com/news/3710065-bristol-myers-wins-positive-chmp-opinion-for-opdivo-in-esophageal-cancer
- https://seekingalpha.com/symbol/BMY
- https://www.bms.com/
- https://www.opdivo.com/
Tirzepatide
Lilly's SURPASS-1 results published in The Lancet show tirzepatide's superior A1C and body weight reductions versus placebo in adults with type 2 diabetes
June 26, 2021Download PDFData presented at ADA's 81st Scientific Sessions® show the highest dose of tirzepatide (15 mg) reduced A1C by 2.07 percent and weight by 9.5 kg (20.9 lb., 11.0 percent)
Majority of study population was treatment-naïve; lowest dose of tirzepatide (5 mg) led to A1C and body weight reductions of 1.87 percent and 7.0 kg (15.4 lb., 7.9 percent)
INDIANAPOLIS, June 26, 2021 /PRNewswire/ -- Tirzepatide led to superior A1C and body weight reductions from baseline across all three doses in adults with type 2 diabetes after 40 weeks of treatment in Eli Lilly and Company's (NYSE: LLY) SURPASS-1 monotherapy clinical trial evaluating the efficacy and safety of tirzepatide compared to placebo. Detailed SURPASS-1 results were presented today in an oral presentation during the American Diabetes Association's® (ADA) 81st Scientific Sessions®, were simultaneously published in The Lancet and will be featured during an ADA-sponsored symposium on Tuesday, June 29.
Study participants in SURPASS-1, 54.2 percent of whom were treatment-naïve, had a relatively short mean duration of diabetes of 4.7 years, a baseline A1C of 7.9 percent and a baseline weight of 85.9 kg. For the efficacy estimandi, tirzepatide reduced A1C by up to 2.07 percent and body weight by up to 9.5 kg (20.9 lb., 11.0 percent) compared to placebo (+0.04 A1C change and body weight change of -0.7 kg [1.5 lb., 0.9 percent]). Up to 52 percent of participants achieved an A1C less than 5.7 percent – the level seen in people without diabetes. Tirzepatide also led to improvements in the change in fasting serum glucose from baseline. In an additional secondary endpoint, tirzepatide led to improvements in the change in two-hour post-meal glucose values from baseline from self-monitored blood glucose data.1,2
The overall safety profile of tirzepatide was similar to the well-established glucagon-like peptide-1 (GLP-1) receptor agonist class, with gastrointestinal side effects being the most commonly reported adverse events. Treatment discontinuation rates due to adverse events were less than 7 percent in each tirzepatide treatment arm.1
Tirzepatide is a novel investigational once-weekly dual glucose-dependent insulinotropic polypeptide (GIP) and GLP-1 receptor agonist that integrates the actions of the GIP and GLP-1 incretins into a single molecule, representing a new class of medicines being studied for the treatment of type 2 diabetes.
For both estimandsii, all three tirzepatide doses reached statistical significance in A1C and body weight reductions from baseline and in the percentage of participants who achieved an A1C of less than 7 percent (the American Diabetes Association's recommended target for people with diabetes) or less than 5.7 percent.1
At 40 weeks, tirzepatide led to a significant decrease in fasting serum glucose (FSG) compared to placebo. In an additional secondary endpoint, the mean two-hour post-meal glucose values for tirzepatide across all three doses were under 140 mg/dL (considered normal values in individuals without diabetes).2
Specifically, the efficacy estimand results showed:
- A1C change: -1.87% (5 mg), -1.89% (10 mg), -2.07% (15 mg), +0.04% (placebo)
- Weight reduction: -7.0 kg (-7.9%, 5 mg), -7.8 kg (-9.3%, 10 mg), -9.5 kg (-11.0%, 15 mg), -0.7 kg (-0.9%, placebo)
- Percent of participants achieving A1C <7%: 87% (5 mg), 92% (10 mg), 88% (15 mg), 20% (placebo)
- Percent of participants achieving A1C <5.7%: 34% (5 mg), 31% (10 mg), 52% (15 mg), 1% (placebo)
- Change in FSG: -43.6 mg/dL (5 mg), -45.9 mg/dL (10 mg), -49.3 mg/dL (15 mg), +12.9 mg/dL (placebo)
The treatment-regimen estimandiii results showed:
- A1C reduction: -1.75% (5 mg), -1.71% (10 mg), -1.69% (15 mg), -0.09% (placebo)
- Weight reduction: -6.3 kg (5 mg), -7.0 kg (10 mg), -7.8 kg (15 mg), -1.0 kg (placebo)
- Percent of participants achieving A1C <7%: 82% (5 mg), 85% (10 mg), 78% (15 mg), 23% (placebo)
- Percent of participants achieving A1C <5.7%: 31% (5 mg), 27% (10 mg), 38% (15 mg), 1% (placebo)
- Change in FSG: -39.6 mg/dL (5 mg), -39.8 mg/dL (10 mg), -38.6 mg/dL (15 mg), +3.7 mg/dL (placebo)
No events of severe hypoglycemia or hypoglycemia less than 54 mg/dL were observed in the tirzepatide treatment arms.1,2
About tirzepatide
Tirzepatide is a once-weekly dual glucose-dependent insulinotropic polypeptide (GIP) and glucagon-like peptide-1 (GLP-1) receptor agonist that integrates the actions of both incretins into a single novel molecule. GIP is a hormone that may complement the effects of GLP-1. In preclinical models, GIP has been shown to decrease food intake and increase energy expenditure therefore resulting in weight reductions, and when combined with a GLP-1 receptor agonist, may result in greater effects on glucose and body weight. Tirzepatide is in phase 3 development for blood glucose management in adults with type 2 diabetes and for chronic weight management. It is also being studied as a potential treatment for non-alcoholic steatohepatitis (NASH) and heart failure with preserved ejection fraction (HFpEF).
About SURPASS-1 and the SURPASS clinical trial program
SURPASS-1 (NCT03954834) is a 40-week, multi-center, randomized, double-blind, parallel, placebo-controlled trial comparing the efficacy and safety of tirzepatide 5 mg, 10 mg and 15 mg as monotherapy to placebo in adults with type 2 diabetes inadequately controlled with diet and exercise alone. The trial randomized 478 study participants across the U.S., Mexico, India and Japan in 1:1:1:1 ratio to receive either tirzepatide 5 mg, 10 mg or 15 mg or placebo. The objective of the study was to demonstrate that tirzepatide (5 mg, 10 mg or 15 mg) is superior in A1C reduction from baseline after 40 weeks in people with type 2 diabetes naïve to injectable therapy who haven't used any oral antidiabetic medicines within three months compared to placebo. Study participants had a mean A1C between 7 percent and 9.5 percent and a BMI greater than or equal to 23 kg/m2. All participants in the tirzepatide treatment arms started the study at a dose of tirzepatide 2.5 mg once-weekly and then increased the dose in a step-wise approach at four-week intervals to their final randomized maintenance dose of 5 mg (via a 2.5 mg step), 10 mg (via steps at 2.5 mg, 5 mg and 7.5 mg) or 15 mg (via steps at 2.5 mg, 5 mg, 7.5 mg, 10 mg and 12.5 mg).
The SURPASS phase 3 global clinical development program for tirzepatide has enrolled more than 19,000 people with type 2 diabetes across 10 clinical trials, five of which are global registration studies. The program began in late 2018, and all five global registration trials have been completed.
For the latest updates, visit http://www.lillydiabetes.com/
To learn more about Lilly, please visit us at lilly.com
View original content to download multimedia:http://www.prnewswire.com/news-releases/lillys-surpass-1-results-published-in-the-lancet-show-tirzepatides-superior-a1c-and-body-weight-reductions-versus-placebo-in-adults-with-type-2-diabetes-301320385.html
SOURCE Eli Lilly and Company
Lilly's tirzepatide shows superior A1C and body weight reductions in late-stage diabetes study
Jun. 28, 2021 3:39 AM ETEli Lilly and Company (LLY)By: Mamta Mayani, SA News Editor
- Eli Lilly (NYSE:LLY) announces results from Phase 3 SURPASS-1 monotherapy clinical trial evaluating the efficacy and safety of tirzepatide compared to placebo in adults with type 2 diabetes after 40 weeks.
- https://seekingalpha.com/news/3710338-lillys-tirzepatide-shows-superior-a1c-and-body-weight-reductions-in-late-stage-type-2-diabetes-study
- https://seekingalpha.com/symbol/LLY
Actemra® (tocilizumab)
Thursday, Jun 24, 2021
Genentech’s Actemra Receives FDA Emergency Use Authorization for the Treatment of COVID-19 In Hospitalized Adults and Children
The authorization enables emergency use of Actemra for the treatment of COVID-19 in hospitalized adult and pediatric patients
South San Francisco, CA -- June 24, 2021 --
Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has issued an Emergency Use Authorization (EUA) for intravenous Actemra® (tocilizumab) for the treatment of COVID-19 in hospitalized adults and pediatric patients (2 years of age and older) who are receiving systemic corticosteroids and require supplemental oxygen, non-invasive or invasive mechanical ventilation, or extracorporeal membrane oxygenation (ECMO). The EUA is based on results from four randomized, controlled studies that evaluated Actemra for the treatment of COVID-19 in more than 5,500 hospitalized patients. The results of these studies suggest that Actemra may improve outcomes in patients receiving corticosteroids and requiring supplemental oxygen or breathing support.
The four randomized, controlled studies included in the EUA submission investigated the safety and efficacy of Actemra in more than 5,500 hospitalized patients with COVID-19. The RECOVERY Actemra study was led by researchers in the United Kingdom and included more than 4,000 hospitalized COVID-19 patients. Genentech-sponsored global trials included the placebo-controlled EMPACTA, COVACTA and REMDACTA studies. There have been no new safety signals identified for Actemra in any of these studies. The most common adverse reactions seen (incidence ≥ 3%) are constipation, anxiety, diarrhea, insomnia, hypertension and nausea.
The FDA Letter of Authorization and Fact Sheets for patients and health care professionals are available for download with the latest information on this EUA. For more information on how Genentech is responding to the global COVID-19 pandemic, please visit our COVID-19 response page.
About the Emergency Use Authorization (EUA) for Actemra
Actemra has not been approved by the U.S. FDA in this setting, but the FDA has made Actemra available under an emergency access mechanism called an EUA as a treatment for certain patients with COVID-19. There is limited information known about the safety or effectiveness of using Actemra to treat people in the hospital with COVID-19. The EUA is supported by a Secretary of Health and Human Services (HHS) declaration that circumstances exist to justify the emergency use of drugs and biological products during the COVID-19 pandemic. The authorization is temporary and does not replace the formal review and approval process. Actemra is authorized under the EUA only for the duration of the declaration that circumstances exist justifying the authorization of the emergency use of Actemra under Section 564(b)(1) of the Act, 21 U.S.C. § 360bbb-3(b)(1), unless the authorization is terminated or revoked sooner. Genentech has existing distribution channels established to ship Actemra to hospitals across the United States.
About the Actemra COVID-19 Clinical Trial Program
Genentech’s clinical trial program evaluated the safety and efficacy of Actemra in hospitalized patients with COVID-19. Actemra is not FDA-approved for this use and there is limited information known about the safety or effectiveness of using Actemra to treat people in the hospital with COVID-19. COVACTA and EMPACTA were the first two global Phase III, multicenter, randomized, placebo-controlled studies of Actemra in patients hospitalized with COVID-19 associated pneumonia. COVACTA was conducted in collaboration with the Biomedical Advanced Research and Development Authority (BARDA), part of the Office of the Assistant Secretary for Preparedness and Response at the United States Department of Health and Human Services (HHS). EMPACTA aimed to address research questions about the safety and efficacy of Actemra in underserved populations by emphasizing enrollment from minority patients often underrepresented in clinical trials. Both studies were published in the New England Journal of Medicine. Genentech also partnered with Gilead Sciences, Inc., on REMDACTA, a Phase III, randomized, double-blind, multicenter study to evaluate the safety and efficacy of Actemra plus Veklury® (remdesivir), versus placebo plus Veklury, in hospitalized patients with severe COVID-19 associated pneumonia.
About Actemra
Actemra was the first humanized interleukin-6 (IL-6) receptor antagonist approved for the treatment of adult patients with moderately to severely active rheumatoid arthritis (RA) who have used one or more disease-modifying antirheumatic drugs (DMARDs), such as methotrexate (MTX), that did not provide enough relief. The extensive Actemra RA IV clinical development program included five Phase III clinical studies and enrolled more than 4,000 people with RA in 41 countries. The Actemra RA subcutaneous clinical development program included two Phase III clinical studies and enrolled more than 1,800 people with RA in 33 countries. Actemra subcutaneous injection is also approved for the treatment of adult patients with giant cell arteritis (GCA), for the treatment of patients two years of age and older with active polyarticular juvenile idiopathic arthritis (PJIA) or active systemic juvenile idiopathic arthritis (SJIA), and for slowing the rate of decline in pulmonary function in adult patients with systemic sclerosis-associated interstitial lung disease (SSc-ILD). In addition, Actemra is also approved in the IV formulation for patients two years of age and older with active PJIA, SJIA or CAR T cell-induced cytokine release syndrome (CRS). Actemra is not approved for subcutaneous use in people with CRS. It is not known if Actemra is safe and effective in children with PJIA, SJIA or CRS under two years of age or in children with conditions other than PJIA, SJIA or CRS.
Actemra is intended for use under the guidance of a healthcare practitioner.
IMPORTANT INFORMATION ABOUT ACTEMRA FOR COVID-19
AUTHORIZED USE
ACTEMRA is authorized for emergency use under an Emergency Use Authorization (EUA) for the treatment of COVID-19 in hospitalized adults and pediatric patients (2 years of age and older) who are receiving corticosteroids and who require supplemental oxygen, or a machine that helps with their breathing (ventilator) or a machine that adds oxygen to the blood outside the body (extracorporeal membrane oxygenation or ECMO).
ACTEMRA is not FDA-approved for this use. There is limited information known about the safety or effectiveness of using ACTEMRA to treat people in the hospital with COVID-19. The safety and effectiveness of ACTEMRA have not been studied in pediatric patients hospitalized with COVID-19.
The EUA for ACTEMRA is in effect for the duration of the COVID-19 declaration justifying emergency use of this product, unless terminated or revoked (after which the products may no longer be used under the EUA).
Please visit http://www.actemra.com for the full Prescribing Information, including Boxed Warning and Medication Guide, for additional Important Safety Information or call 1-800-ACTEMRA (228-3672).
For additional information about the company, please visit http://www.gene.com.
Roche's Actemra wins FDA emergency use nod for hospitalized COVID-19 patients
Jun. 25, 2021 12:35 AM ET Roche Holding AG (RHHBY) By: Mamta Mayani, SA News Editor1 Comment
- Roche (OTCQX:RHHBY) unit Genentech announces that the FDA has issued an Emergency Use Authorization (EUA) for intravenous Actemra (tocilizumab) for the treatment of COVID-19 in hospitalized adults and pediatric patients (2 years of age and older) who are receiving systemic corticosteroids and require supplemental oxygen, mechanical ventilation, or extracorporeal membrane oxygenation (ECMO).
- https://seekingalpha.com/news/3710013-roches-actemra-wins-fda-emergency-use-nod-for-hospitalized-covid-19-patients
- https://seekingalpha.com/symbol/RHHBY
VOXZOGO™ (vosoritide)
BioMarin Receives Positive CHMP Opinion in Europe for Vosoritide for the Treatment of Children with Achondroplasia from Age 2 Until Growth Plates Close
European Commission Decision expected Q3 2021Temporary Authorization for Use (ATU) Granted in France to Allow Access and Reimbursement of Vosoritide to Begin ImmediatelyVosoritide is Potentially the First Medicine to be Approved to Treat Children with Achondroplasia in EuropeEstimated Over 11,000 Children Across Europe, Middle East, and Africa Affected by Achondroplasia and Eligible for Treatment if ApprovedFDA Review of Vosoritide NDA Ongoing, PDUFA Target Action Date Nov. 20, 2021Jun 25, 2021
SAN RAFAEL, Calif., June 25, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending marketing authorization for vosoritide, a once daily injection analog of C-type Natriuretic Peptide (CNP) to treat achondroplasia in children from the age of 2 until growth plates are closed, which occurs after puberty when children reach final adult height. Achondroplasia is the most common form of disproportionate short stature in humans. A final approval decision, typically consistent with the CHMP recommendation, is expected from the European Commission in Q3 2021. Vosoritide is potentially the first medicine to be approved to treat children with achondroplasia in Europe and would be marketed under the brand name VOXZOGO™ (vosoritide).
The CHMP based its opinion on the totality of data from the vosoritide clinical development program including the outcomes from the randomized, double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of vosoritide. The Phase 3 Study was further supported by the ongoing long-term safety and efficacy from the Phase 2 dose-finding study, which showed that growth rates have been sustained above participants' baseline rates and above the expected annualized growth velocity for untreated children with achondroplasia throughout the five year observation period for which data are currently available. No acceleration of bone age was observed, suggesting that vosoritide is not reducing the total duration of the growth period. The data package included results from an ongoing Phase 2 randomized double-blind study in infants and young children, including extensive pharmacokinetic and biomarker data, as well as preliminary growth data from participants in the 2 to 5 year age cohort. Data in sentinel study participants showed a positive effect on growth following two years of vosoritide treatment in subjects aged 2 to 5 years. In addition, the data package included the Phase 3 extension study and extensive natural history data.
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 their scientific and clinical merit and provides advice to the European Commission (EC), which has the authority to approve medicines for the EU.
Regulatory Status
The U.S. New Drug Application (NDA) for vosoritide is under review by the FDA with a Prescription Drug User Fee Act (PDUFA) target action date of November 20, 2021. The Company successfully closed out the in-person FDA pre-approval inspection of its manufacturing facilities for vosoritide earlier this year.
In January 2021, the Company received notice from the FDA that the NDA for vosoritide had been granted Priority Review Designation based on the serious pediatric indication it addresses, and the lack of treatment options currently available. Consistent with FDA's policy on changes to review classification for an ongoing application review, the PDUFA action date is not affected by this designation. If approved, the vosoritide NDA may qualify for a Priority Review Voucher (PRV). A PRV confers priority review to a subsequent drug application that would not otherwise qualify for that designation. The rare pediatric disease review voucher program is designed to encourage development of new drugs and biologics for the prevention or treatment of rare pediatric diseases.
Vosoritide received orphan drug designation from the FDA and EMA for the treatment of children with achondroplasia. The Orphan Drug Designation program is intended to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions.
Description of Phase 3 Study and Extension
The global Phase 3 study was a randomized, double-blind, placebo-controlled study of vosoritide in 121 children with achondroplasia aged 5 to 14 for 52 weeks. (The enrollment age criteria are 5 to 18 per the study protocol). Vosoritide is being tested in children whose growth plates are still open. This is approximately 25% of people with achondroplasia. Children in this study completed a minimum six-month baseline study to determine their respective baseline growth velocity prior to entering the Phase 3 study. The primary endpoint of the study is the change in growth velocity from baseline over one year in children treated with vosoritide compared to placebo. A wide range of secondary and exploratory endpoints include anthropometric measures such as height Z-score, body and limb proportionality and joint geometry; biochemical, biomarker and radiological assessments of bone growth and health; and evaluations of health-related quality of life (HRQoL), developmental status, and functional independence. These additional endpoints address the overall impact vosoritide has on achondroplasia and will continue to be evaluated in an ongoing open-label extension study where all subjects receive active treatment.
The ongoing open-label long-term extension study to the completed pivotal, double-blind, placebo-controlled study of vosoritide in children with achondroplasia has a total of 119 children enrolled in the extension study after completion of the pre-treatment observation period and one year of treatment in the pivotal phase 3 study, and all are receiving open-label treatment with vosoritide 15 mcg/kg daily.
Description of Phase 2 Dose Finding Study
The primary objectives of the open-label, sequential cohort, dose-finding study were to evaluate the safety and tolerability of daily subcutaneous vosoritide and to determine the dose to carry forward to Phase 3. Secondary objectives were to evaluate the effects of vosoritide on change from pre-treatment baseline in annualized growth velocity (cm/year), height Z-scores, and body segment proportionality, the vosoritide pharmacokinetic (PK) profile, and biomarkers of vosoritide activity, and endochondral ossification. The study is followed by the ongoing open label extension study to evaluate long-term safety and efficacy of vosoritide.
Description of Phase 2 Study in Infants and Toddlers
The fully enrolled Phase 2 study is an ongoing randomized, placebo-controlled study of vosoritide in approximately 70 infants and young children with achondroplasia ages zero to less than 60 months for 52 weeks. The study is followed by a subsequent open-label extension. Children in this study will have completed a minimum six-month baseline study to determine their respective baseline growth prior to entering the Phase 2 study. The primary objectives of the study are to evaluate safety, tolerability, and the effect of vosoritide on height Z-scores, which is the number of standard deviations in relation to the mean height of age-matched, average stature children. The study also evaluates the PK profile of vosoritide and effects on specific biomarkers of chondrocyte activity. The company also plans to augment the height Z-score data with assessments including proportionality, functionality, quality of life, sleep apnea, and foramen magnum dimension, as well as the advent of major illnesses and surgeries.
For additional information, please visit www.biomarin.com.
BioMarin gets positive CHMP opinion for vosoritide marketing authorization
Jun. 25, 2021 7:38 AM ET
BioMarin Pharmaceutical Inc. (BMRN)
By: Aakash Babu, SA News Editor1 Comment
- BioMarin Pharmaceutical (NASDAQ:BMRN) announce that the EMA's Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending marketing authorization for vosoritide to treat achondroplasia in children.
- https://seekingalpha.com/news/3710064-biomarin-gets-positive-chmp-opinion-for-vosoritide-marketing-authorization
- https://seekingalpha.com/symbol/BMRN
- https://www.biomarin.com/our-treatments/pipeline/vosoritide-for-achondroplasia/
Monjuvi®(tafasitamab-cxix)
Incyte and MorphoSys Announce Positive CHMP Opinion for Tafasitamab in Combination with Lenalidomide for the Treatment of Adults with Relapsed or Refractory Diffuse Large B-Cell Lymphoma
June 25, 2021 DownloadPDF Format (opens in new window)
- If approved, tafasitamab in combination with lenalidomide would represent an important new therapeutic option for eligible DLBCL patients in the European Union
- The positive opinion from the CHMP is based on data from the Phase 2 L-MIND study evaluating tafasitamab in combination with lenalidomide as a treatment for patients with relapsed or refractory DLBCL
WILMINGTON, Del. & PLANEGG, Germany & MUNICH--(BUSINESS WIRE)-- Incyte (Nasdaq:INCY) and MorphoSys AG (FSE:MOR; NASDAQ:MOR) today announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending the conditional marketing authorization of tafasitamab in combination with lenalidomide, followed by tafasitamab monotherapy, for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) who are not eligible for autologous stem cell transplantation (ASCT).
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210625005231/en/
“The CHMP’s positive opinion of tafasitamab is a pivotal step towards addressing an urgent unmet medical need for the 30-40% of patients with relapsed or refractory DLBCL who do not respond to initial therapy or relapse thereafter,” said Steven Stein, M.D., Chief Medical Officer, Incyte. “Following the U.S. FDA’s approval of tafasitamabin July 2020, we eagerly await the European Commission’s decision as we look forward to bringing this new therapy to eligible patients in Europe as soon as possible.”
“Tafasitamab in combination with lenalidomide represents an important new targeted treatment option for patients with relapsed or refractory DLBCL,” said Dr. Malte Peters, Chief Research & Development Officer, MorphoSys. “Patients with relapsed or refractory DLBCL have limited treatment options and often face a poor prognosis. There is an urgent need for effective therapies and if approved, this combination could provide patients in Europe with an important new therapeutic option.”
The CHMP opinion to recommend the use of tafasitamab is now being reviewed by the European Commission, which has the authority to grant marketing authorization for medicinal products in the European Union (EU). If approved, tafasitamab would be commercialized in the EU under the brand name Minjuvi®.
Incyte and MorphoSys share global development rights to tafasitamab and Incyte has exclusive commercialization rights to tafasitamab outside the United States. Incyte and MorphoSys co-commercialize tafasitamab under the brand name Monjuvi® in the United States.
About L-MIND
The L-MIND trial is a single arm, open-label Phase 2 study (NCT02399085) investigating the combination of tafasitamab and lenalidomide in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) who have had at least one, but no more than three prior lines of therapy, including an anti-CD20 targeting therapy (e.g., rituximab), who are not eligible for high-dose chemotherapy (HCD) or autologous stem cell transplant (ASCT). The study’s primary endpoint is overall response rate (ORR). Secondary outcome measures include duration of response (DoR), progression-free survival (PFS) and overall survival (OS). The study reached its primary completion in May 2019.
For more information about L-MIND, visit https://clinicaltrials.gov/ct2/show/NCT02399085.
About Tafasitamab
Tafasitamab is a humanized Fc-modified cytolytic CD19 targeting monoclonal antibody. In 2010, MorphoSys licensed exclusive worldwide rights to develop and commercialize tafasitamab from Xencor, Inc. Tafasitamab incorporates an XmAb® engineered Fc domain, which mediates B-cell lysis through apoptosis and immune effector mechanism including Antibody-Dependent Cell-Mediated Cytotoxicity (ADCC) and Antibody-Dependent Cellular Phagocytosis (ADCP).
Monjuvi®(tafasitamab-cxix) is approved by the U.S. Food and Drug Administration in combination with lenalidomide for the treatment of adult patients with relapsed or refractory DLBCL not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT). This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
Tafasitamab is being clinically investigated as a therapeutic option in B-cell malignancies in several ongoing combination trials.
Minjuvi® and Monjuvi® are registered trademarks of MorphoSys AG. Tafasitamab is marketed under the brand name Monjuvi® in the US. If approved in the EU, tafasitamab will be marketed under the brand name Minjuvi ®.
XmAb® is a registered trademark of Xencor, Inc.
For additional information on Incyte, please visit Incyte.com
More information at www.morphosys.com or www.morphosys-us.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210625005231/en/
Source: Incyte
Incyte/Morphosys' tafasitamab gets CHMP opinion to treat certain patients with lymphoma
Jun. 25, 2021 7:53 AM ET
Incyte Corporation (INCY), MOR By: Aakash Babu, SA News Editor
- Incyte (NASDAQ:INCY) and MorphoSys AG (NASDAQ:MOR) announces that the EMA's Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending the conditional marketing authorization of tafasitamab for the treatment of certain patients with lymphoma.
- https://seekingalpha.com/news/3710073-incytemorphosys-tafasitamab-gets-chmp-opinion-to-treat-certain-patients-with-lymphoma
- https://seekingalpha.com/symbol/MOR
- https://seekingalpha.com/symbol/INCY
- https://www.monjuvi.com/
Abecma (idecabtagene vicleucel)
Bristol Myers Squibb Receives Positive CHMP Opinion for Anti-BCMA CAR T Cell Therapy Abecma (idecabtagene vicleucel) for Relapsed and Refractory Multiple Myeloma
06/25/2021CATEGORY:
Recommendation for approval based on results from pivotal KarMMa study
Abecma is the first CAR T cell therapy for adults with relapsed and refractory multiple myeloma to receive positive CHMP opinion and builds on the company’s innovative and leading portfolio across the disease
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended granting Conditional Marketing Authorization for Abecma (idecabtagene vicleucel; ide-cel), the company’s B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy, for the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody and have demonstrated disease progression on the last therapy. The CHMP recommendation will now be reviewed by the European Commission (EC), which has the authority to approve medicines for the European Union (EU).
The CHMP adopted a positive opinion based on results from the pivotal Phase 2 KarMMa study evaluating the efficacy and safety of Abecma in 128 patients with heavily pre-treated and highly refractory multiple myeloma.
“As the first CAR T cell therapy for relapsed and refractory multiple myeloma to receive a positive CHMP opinion, Abecma represents a potential new treatment approach for patients in Europe battling this incurable blood cancer,” said Noah Berkowitz, M.D., Ph.D., senior vice president, Cellular Therapy Development, Bristol Myers Squibb. “We look forward to the European Commission’s decision as we build on our innovative multiple myeloma and cell therapy research to offer novel and personalized treatment options for patients in need.”
The EC is expected to deliver its final decision within 67 days of receipt of the CHMP opinion. The decision will be applicable to all European Union member states and Iceland, Norway and Liechtenstein. The EMA previously granted Abecma access to the PRIority MEdicines (PRIME) scheme for the treatment of relapsed and refractory multiple myeloma.
About KarMMa
KarMMa (NCT03361748) is a pivotal, open-label, single-arm, multicenter, multinational, Phase 2 study evaluating the efficacy and safety of ide-cel in adults with relapsed and refractory multiple myeloma in North America and Europe. The primary endpoint of the study is overall response rate as assessed by an independent review committee (IRC) according to the International Myeloma Working Group (IMWG) criteria. Complete response rate is a key secondary endpoint. Other secondary endpoints include time to response, duration of response, progression-free survival, overall survival, minimal residual disease evaluated by Next-Generation Sequencing (NGS) assay and safety. The study enrolled 140 patients, of whom 128 received ide-cel across the target dose levels of 150-450 x 106 CAR+ T cells after receiving lymphodepleting chemotherapy. All enrolled patients had received at least three prior treatment regimens, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody, and were refractory to their last regimen, defined as progression during or within 60 days of their last therapy.
About Abecma
Abecma is the first-in-class B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy, approved in the U.S. for the treatment of adult patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody. Abecma recognizes and binds to BCMA on the surface of multiple myeloma cells leading to CAR T cell proliferation, cytokine secretion, and subsequent cytolytic killing of BCMA-expressing cells.
Abecma is being jointly developed and commercialized in the U.S. as part of a Co-Development, Co-Promotion, and Profit Share Agreement with Bristol Myers Squibb and bluebird bio. Bristol Myers Squibb will assume sole responsibility for Abecma drug product manufacturing and commercialization outside of the U.S.
U.S. FDA-Approved Indication
ABECMA (idecabtagene vicleucel) is a B-cell maturation antigen (BCMA)-directed genetically modified autologous T cell immunotherapy indicated for the treatment of adult patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody.
Please see full Prescribing Information, including Boxed WARNINGS and Medication Guide.
Learn more about the science behind cell therapy and ongoing research at Bristol Myers Squibb here.
For more information about Bristol Myers Squibb, visit us at BMS.com
Celgene and Juno Therapeutics are wholly owned subsidiaries of Bristol-Myers Squibb Company. In certain countries outside the U.S., due to local laws, Celgene and Juno Therapeutics are referred to as Celgene, a Bristol Myers Squibb company and Juno Therapeutics, a Bristol Myers Squibb company.
ABECMA®, Cell Therapy 360®, and the related logos are trademarks of Celgene Corporation, a Bristol-Myers Squibb Company.
© 2021 Bristol-Myers Squibb Company. All rights reserved. US-IDE-20-0020 03/2021
Bristol Myers wins positive CHMP opinion for Abecma in multiple myeloma
Jun. 25, 2021 7:33 AM ET
Bristol-Myers Squibb Company (BMY)
By: Mamta Mayani, SA News Editor1 Comment
- Bristol Myers Squibb (NYSE:BMY) announces that the EMA's Committee for Medicinal Products for Human Use (CHMP) has recommended granting Conditional Marketing Authorization for Abecma (idecabtagene vicleucel; ide-cel), the company’s BCMA-directed chimeric antigen receptor (CAR) T cell immunotherapy, for the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least three prior therapies.
- https://seekingalpha.com/news/3710061-bristol-myers-wins-positive-chmp-opinion-for-abecma-in-multiple-myeloma
- https://seekingalpha.com/symbol/BMY
- https://seekingalpha.com/symbol/BLUE
- https://www.bluebirdbio.com/our-products
biotecMAX™ April/May/June 2021 Insight
LIBTAYO® (CEMIPLIMAB)
LIBTAYO® (CEMIPLIMAB) APPROVED BY THE EUROPEAN COMMISSION AS THE FIRST IMMUNOTHERAPY INDICATED FOR PATIENTS WITH ADVANCED BASAL CELL CARCINOMA
TARRYTOWN, N.Y. and PARIS, June 25, 2021 /PRNewswire/ --
Approval based on data from the largest trial to date in patients with advanced basal cell carcinoma previously treated with a hedgehog pathway inhibitor
Libtayo now approved by the European Commission for three advanced cancers
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today announced that the European Commission (EC) has approved the PD-1 inhibitor Libtayo® (cemiplimab) to treat adults with locally advanced or metastatic basal cell carcinoma (BCC) who have progressed on or are intolerant to a hedgehog pathway inhibitor (HHI).
BCC is the most common type of skin cancer worldwide, representing up to 80% of non-melanoma skin cancers, and incidence is increasing across many European countries. While the large majority of BCCs are caught early and easily cured with surgery and/or radiation, a small proportion of cases can develop into advanced BCC and penetrate deeper into surrounding tissues (locally advanced) or spread to other parts of the body (metastatic), becoming more difficult to treat.
"Libtayo is the first immunotherapy to show a clinical benefit in patients with advanced BCC after HHI therapy in a pivotal trial, and with this first-in-class approval has the potential to transform treatment for patients in Europe whose cancer has progressed despite HHI treatment," said Israel Lowy, M.D., Ph.D., Senior Vice President, Translational and Clinical Sciences, Oncology at Regeneron. "We look forward to continuing to investigate this medicine in additional settings, with the goal of helping more patients with difficult-to-treat cancers around the world."
Libtayo is now approved for three advanced cancers in the European Union, following the EC's concurrent approval of Libtayo for the first-line treatment of adult patients with advanced non-small cell lung cancer (NSCLC) whose tumor cells have ≥50% PD-L1 expression and no EGFR, ALK or ROS1 aberrations. In 2019, Libtayo was approved by the EC as the first treatment for adults with metastatic or locally advanced cutaneous squamous cell carcinoma (CSCC) who are not candidates for curative surgery or curative radiation. Across all of its approved indications, Libtayo had a generally consistent safety profile. Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue during or after treatment with Libtayo.
The EC approval in BCC is based on data from the largest prospective clinical trial (n=119) in patients with advanced BCC previously treated with an HHI to date. Libtayo-treated patients with locally advanced BCC experienced an objective response rate (ORR) of 32% (95% confidence interval [CI]: 22-43) (25% partial response, 7% complete response) by independent central review. Libtayo-treated patients with metastatic BCC demonstrated an ORR of 29% (95% CI: 15-46) (26% partial response, 3% complete response) by investigator assessment. In addition, approximately 90% of patients across both groups had a duration of response (DOR) of 6 months or longer per Kaplan Meier estimates, and the median DOR has not been reached for either group. Median duration of follow-up was 16 months for locally advanced BCC and 9 months for metastatic BCC.
About the Phase 2 Trial in Advanced BCC
The EC approval was based on data from an ongoing open-label, multi-center, non-randomized Phase 2 trial of patients with unresectable locally advanced BCC or metastatic BCC (nodal or distant). Patients in both cohorts had either progressed on HHI therapy, had not had an objective response after nine months on HHI therapy, or were intolerant of prior HHI therapy. The primary efficacy endpoint was confirmed ORR, and a key secondary endpoint was DOR, assessed by independent central review.
About Libtayo
Libtayo is a fully human monoclonal antibody targeting the immune checkpoint receptor PD-1 on T-cells. By binding to PD-1, Libtayo has been shown to block cancer cells from using the PD-1 pathway to suppress T-cell activation.
The recommended dose of Libtayo is 350 mg administered as an intravenous infusion over 30 minutes every three weeks, until disease progression or unacceptable toxicity. Libtayo is available as a single-dose 350 mg vial. No PD-L1 or tumor mutational burden (TMB) testing is required before starting treatment with Libtayo for advanced BCC.
The extensive clinical program for Libtayo is focused on difficult-to-treat cancers. Current clinical development programs include Libtayo in combination with chemotherapy for advanced NSCLC irrespective of PD-L1 expression and Libtayo monotherapy for advanced cervical cancer. Libtayo is also being investigated in combination with either conventional or novel therapeutic approaches for other solid tumors and blood cancers. These potential uses are investigational, and their safety and efficacy have not been evaluated by any regulatory authority.
The generic name for Libtayo in its approved U.S. indications is cemiplimab-rwlc, with rwlc as the suffix designated in accordance with Nonproprietary Naming of Biological Products Guidance for Industry issued by the U.S. Food and Drug Administration (FDA). Libtayo is being jointly developed by Regeneron and Sanofi under a global collaboration agreement.
About Regeneron's VelocImmune® Technology
Regeneron's VelocImmune technology utilizes a proprietary genetically engineered mouse platform endowed with a genetically humanized immune system to produce optimized fully human antibodies. When Regeneron's President and Chief Scientific Officer George D. Yancopoulos was a graduate student with his mentor Frederick W. Alt in 1985, they were the first to envision making such a genetically humanized mouse, and Regeneron has spent decades inventing and developing VelocImmune and related VelociSuite® technologies. Dr. Yancopoulos and his team have used VelocImmune technology to create approximately a quarter of all original, FDA-approved fully human monoclonal antibodies currently available. This includes REGEN–COV™ (casirivimab and imdevimab), Dupixent® (dupilumab), Libtayo® (cemiplimab-rwlc), Praluent® (alirocumab), Kevzara® (sarilumab), Evkeeza® (evinacumab-dgnb) and Inmazeb™ (atoltivimab, maftivimab and odesivimab-ebgn).
What is Libtayo?
Libtayo is a prescription medicine used to treat people with a type of skin cancer called cutaneous squamous cell carcinoma (CSCC) that has spread or cannot be cured by surgery or radiation.
Libtayo is a prescription medicine used to treat people with a type of skin cancer called basal cell carcinoma that cannot be removed by surgery (locally advanced BCC) and have received treatment with an HHI, or cannot receive treatment with an HHI.
Libtayo is a prescription medicine used to treat people with a type of skin cancer called basal cell carcinoma that has spread (metastatic BCC) and have received treatment with a hedgehog pathway inhibitor (HHI), or cannot receive treatment with an HHI. This use is approved based on how many patients responded to treatment and how long they responded. Studies are ongoing to provide additional information about clinical benefit.
Libtayo is a prescription medicine used to treat people with a type of lung cancer called non-small cell lung cancer (NSCLC). Libtayo may be used as your first treatment when your lung cancer has not spread outside your chest (locally advanced lung cancer) and you cannot have surgery or chemotherapy with radiation, or your lung cancer has spread to other areas of your body (metastatic lung cancer), and your tumor tests positive for high "PD-L1" and your tumor does not have an abnormal "EGFR", "ALK "or "ROS1" gene.
It is not known if Libtayo is safe and effective in children.
Please see full Prescribing Information, including Medication Guide.
For additional information about the company, please visit www.regeneron.com
View original content:http://www.prnewswire.com/news-releases/libtayo-cemiplimab-approved-by-the-european-commission-as-the-first-immunotherapy-indicated-for-patients-with-advanced-basal-cell-carcinoma-301319988.html
SOURCE Regeneron Pharmaceuticals, Inc.
Regeneron/Sanofi's Libtayo OK'd in Europe for skin cancer
Jun. 25, 2021 1:38 AM ET Regeneron Pharmaceuticals, Inc. (REGN), SNY By: Mamta Mayani, SA News Editor
https://seekingalpha.com/symbol/REGN
https://seekingalpha.com/symbol/SNY
https://seekingalpha.com/news/3710015-regeneronsanofis-libtayo-okd-in-europe-for-skin-cancer
RINVOQ® (upadacitinib)
June 25, 2021
CHMP Recommends Approval of RINVOQ® (upadacitinib) for the Treatment of Atopic Dermatitis
- Positive opinion based on three global Phase 3 pivotal studies evaluating the safety and efficacy of RINVOQ (15 mg or 30 mg, once daily) used with or without topical corticosteroids in adults and adolescents with moderate to severe atopic dermatitis[1],[2]
- If approved, this will be the fourth indication for RINVOQ in the European Union[3]
- The European Commission decision is anticipated in the third quarter of 2021
NORTH CHICAGO, Ill., June 25, 2021 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending the approval of RINVOQ® (upadacitinib), an oral, selective and reversible JAK inhibitor, for the expanded use in adults (15 mg or 30 mg, once daily) and adolescents 12 years and older (15 mg, once daily) with moderate to severe atopic dermatitis who are candidates for systemic therapy. RINVOQ is being studied in several immune-mediated inflammatory diseases.4-10
The CHMP positive opinion was supported by data from the global Phase 3 program evaluating more than 2,500 patients with moderate to severe atopic dermatitis across three global pivotal studies: Measure Up 1, Measure Up 2 and AD Up.1,2 Across the three studies, both doses of RINVOQ met all primary and secondary endpoints, demonstrating rapid and significant improvement in skin clearance and reduction in itch compared to placebo at week 16 and other time points (p<0.001) in patients with moderate to severe atopic dermatitis.1,2 The most commonly reported adverse events in patients treated with RINVOQ were acne, nasopharyngitis and upper respiratory tract infections.1,2
"This milestone is an important step forward in our journey to improve care for people living with atopic dermatitis," said Michael Severino, M.D., vice chairman and president, AbbVie. "Despite available treatments, many people with moderate to severe forms of this disease continue to experience a relentless and burdensome cycle of skin and itch symptoms. We are encouraged that the CHMP has recognized RINVOQ's potential as an additional treatment option for these patients."
The CHMP positive opinion is a scientific recommendation for marketing authorization to the European Commission, which authorizes marketing approval in the European Union. The Marketing Authorization will be valid in all member states of the European Union, as well as Iceland, Liechtenstein, Norway and Northern Ireland. If approved, this will be the fourth indication for RINVOQ, and RINVOQ will be the first JAK inhibitor in the European Union to treat moderate to severe atopic dermatitis in both adults and adolescents 12 years and older.3
About the RINVOQ Atopic Dermatitis Global Phase 3 Study Program
The global Phase 3 program evaluated more than 2,500 patients worldwide across three global pivotal studies: Measure Up 1, Measure Up 2 and AD Up.1,2 The studies evaluated the efficacy and safety of RINVOQ (15 mg and 30 mg, once daily), with and without topical corticosteroids (TCS), in adults and adolescents with moderate to severe atopic dermatitis who were candidates for systemic therapy.1,2 The co-primary endpoints across all three studies were at least a 75 percent improvement in Eczema Area and Severity Index (EASI 75) and a validated Investigator's Global Assessment for Atopic Dermatitis (vIGA-AD) score 0/1 at week 16.1,2 Secondary endpoints included reduction of itch defined as ≥4 point improvement in Worst Pruritus Numerical Rating Scale (NRS) from baseline at week 16 and other timepoints, as well as EASI 90 and EASI 100 at week 16.1,2 More information on this program can be found at www.clinicaltrials.gov (NCT03569293, NCT03607422, NCT03568318).
About RINVOQ® (upadacitinib)
Discovered and developed by AbbVie scientists, RINVOQ is a selective and reversible JAK inhibitor that is being studied in several immune-mediated inflammatory diseases.1-10 In human cellular assays, RINVOQ preferentially inhibits signaling by JAK1 or JAK1/3 with functional selectivity over cytokine receptors that signal via pairs of JAK2.3 In August 2019, RINVOQ received U.S. FDA approval for adult patients with moderately to severely active rheumatoid arthritis who have had an inadequate response or intolerance to methotrexate. RINVOQ is approved by the European Commission for the treatment of adult patients with moderate to severe active rheumatoid arthritis who have responded inadequately to, or who are intolerant to one or more disease-modifying anti-rheumatic drugs (DMARDs); for the treatment of active psoriatic arthritis (PsA) in adult patients who have responded inadequately to, or who are intolerant to one or more DMARDs; and for the treatment of active ankylosing spondylitis (AS) in adult patients who have responded inadequately to conventional therapy. The approved dose for RINVOQ in these indications is 15 mg. Phase 3 trials of RINVOQ in axial spondyloarthritis, Crohn's disease, ulcerative colitis, giant cell arteritis and Takayasu arteritis are ongoing.4-10 Use of RINVOQ in atopic dermatitis is not approved and its safety and efficacy are under evaluation by regulatory authorities.
Please see the full SmPC for complete prescribing information at http://www.EMA.europa.eu.
EMA advisory group backs approval of AbbVie's RINVOQ in atopic dermatitis
Jun. 25, 2021 4:29 AM ET
AbbVie Inc. (ABBV) By: Mamta Mayani, SA News Editor3 Comments
- AbbVie (NYSE:ABBV) announces the EMA's Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending the approval of RINVOQ (upadacitinib), an oral, selective and reversible JAK inhibitor, for the expanded use in adults (15 mg or 30 mg, once daily) and adolescents 12 years and older (15 mg, once daily) with moderate to severe atopic dermatitis who are candidates for systemic therapy.
- https://seekingalpha.com/news/3710023-ema-advisory-group-backs-approval-of-abbvies-rinvoq-in-atopic-dermatitis
- https://www.rinvoq.com/
- https://seekingalpha.com/symbol/ABBV
Vutrisiran
Alnylam Announces U.S. Food and Drug Administration Acceptance of New Drug Application for Investigational Vutrisiran for the Treatment of the Polyneuropathy of Hereditary ATTR Amyloidosis
Jun 24, 2021
– PDUFA Date Set for April 14, 2022 –
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jun. 24, 2021-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the U.S. Food and Drug Administration (FDA) has accepted the Company’s New Drug Application (NDA) for vutrisiran, an investigational RNAi therapeutic for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. The FDA has set an action date of April 14, 2022 under the Prescription Drug User Fee Act (PDUFA), and the Agency has indicated that they are not currently planning an advisory committee meeting as part of the NDA review.
“We are excited that the FDA has accepted our NDA for vutrisiran, which was based on 9-month results from the HELIOS-A study. Today’s announcement marks another important milestone as we work to make vutrisiran available to hATTR amyloidosis patients with polyneuropathy,” said Rena Denoncourt, Vice President, TTR Franchise Lead. “If approved, once-quarterly, subcutaneously administered vutrisiran may represent a new treatment option that potentially reverses polyneuropathy manifestations of disease.”
Vutrisiran has been granted Orphan Drug Designation in the U.S. and the European Union (EU) for the treatment of ATTR amyloidosis. Vutrisiran has also been granted a Fast-Track designation in the U.S. for the treatment of the polyneuropathy of hATTR amyloidosis in adults. The Company plans to also submit regulatory filings in the EU, Brazil, and Japan in 2021 based on the HELIOS-A results.
About Vutrisiran
Vutrisiran is an investigational, subcutaneously administered RNAi therapeutic in development for the treatment of ATTR amyloidosis, which encompasses both hereditary (hATTR) and wild-type (wtATTR) amyloidosis. It is designed to target and silence specific messenger RNA, blocking the production of wild-type and variant transthyretin (TTR) protein before it is made. Quarterly administration of vutrisiran may help to reduce deposition and facilitate the clearance of TTR amyloid deposits in tissues and potentially restore function to these tissues. Vutrisiran utilizes Alnylam’s Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate delivery platform, designed for increased potency and high metabolic stability that may allow for infrequent subcutaneous injections. The safety and efficacy of vutrisiran have not been evaluated by the U.S. Food and Drug Administration, European Medicines Agency or any other health authority.
About the HELIOS-A Phase 3 Study
HELIOS-A (NCT03759379) is a Phase 3 global, randomized, open-label study to evaluate the efficacy and safety of vutrisiran. The study enrolled 164 patients with hATTR amyloidosis with polyneuropathy at 57 sites in 22 countries. Patients were randomized 3:1 to receive either 25mg of vutrisiran (N=122) via subcutaneous injection once every three months or 0.3 mg/kg of patisiran (N=42) via intravenous infusion once every three weeks (as a reference comparator) for 18 months. The efficacy results of vutrisiran in HELIOS-A are compared to the placebo group from the landmark APOLLO Phase 3 study, which evaluated the efficacy and safety of patisiran in a patient population similar to that studied in HELIOS-A. The primary endpoint is the change from baseline in mNIS+7 score at nine months. Secondary endpoints at 9 months are the change from baseline in the Norfolk QoL-DN score and the timed 10-MWT. Changes from baseline in NT-proBNP were evaluated as an exploratory endpoint at nine months. Additional secondary endpoints at 18 months will be evaluated in the HELIOS-A study, including change from baseline in mNIS+7, Norfolk QoL-DN, 10-MWT, modified body mass index (mBMI), Rasch-built Overall Disability Scale (R-ODS), and serum transthyretin (TTR) levels. Additional exploratory cardiac endpoint data at the 18-month time point will be evaluated, including NT-proBNP, echocardiographic measures and cardiac amyloid assessments with technetium scintigraphy imaging. Following the 18-month treatment period, all patients are eligible to receive vutrisiran for an additional 18 months as part of the randomized treatment extension where they will receive either 25mg vutrisiran once quarterly or 50mg vutrisiran once every six months.
For more information about our people, science and pipeline, please visit www.alnylam.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20210624005230/en/
Source: Alnylam Pharmaceuticals, Inc.
FDA accepts Alnylam's vutrisiran application for hereditary ATTR amyloidosis
Jun. 24, 2021 7:18 AM ETAlnylam Pharmaceuticals, Inc. (ALNY)By: Mamta Mayani, SA News Editor
- Alnylam Pharmaceuticals (NASDAQ:ALNY) announces that the FDA has accepted its New Drug Application (NDA) for vutrisiran, an investigational RNAi therapeutic for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults.
- https://seekingalpha.com/news/3709460-fda-accepts-alnylams-vutrisiran-application-for-hereditary-attr-amyloidosis
- https://seekingalpha.com/symbol/ALNY
- https://www.alnylam.com/
- https://www.alnylam.com/alnylam-rnai-pipeline/
LYNPARZA® (olaparib)
Lynparza approved in China for the treatment of BRCA-mutated metastatic castration-resistant prostate cancer
PUBLISHED24 June 2021
24 June 2021 07:00 BST
Only PARP inhibitor to improve overall survival versus new hormonal treatments in advanced prostate cancer
First PARP inhibitor approved in China in advanced prostate cancer
AstraZeneca and MSD's Lynparza (olaparib) has been granted conditional approval in China to treat adult patients with germline or somatic BRCA-mutated metastatic castration-resistant prostate cancer (mCRPC) who have progressed following treatment that included a new hormonal agent (abiraterone, enzalutamide).
In China, prostate cancer is the sixth most prevalent cancer in men, with approximately 115,000 new patients diagnosed each year and about 7% have germline BRCA mutations.1,2 Prostate cancer patients with these mutations are more likely to have poorer outcomes than those without the mutations.3 Around 70% of prostate cancer patients in China have advanced disease at the time of diagnosis, and for those with mCRPC, the median survival is less than two years.4,5
The approval by China's National Medical Products Administration was based on a subgroup analysis of the PROfound Phase III trial, which showed that Lynparza demonstrated a substantial improvement in radiographic progression-free survival (rPFS) and overall survival (OS) versus abiraterone or enzalutamide in men with BRCA1/2 mutations. Continued approval is contingent upon verification and description of clinical benefit in a planned bridging trial with Chinese patients.
The subgroup analysis from the PROfound Phase III trial showed Lynparza reduced the risk of disease progression or death by 78% (based on a hazard ratio [HR] of 0.22, 95% confidence interval [CI] 0.15-0.32; nominal p<0.0001) and improved rPFS to a median of 9.8 months versus 3.0 with abiraterone or enzalutamide in men with mCRPC with BRCA1/2 mutations. In addition, Lynparza reduced the risk of death by 37% (HR of 0.63, 95% CI 0.42-0.95) with median OS of 20.1 months versus 14.4 with abiraterone or enzalutamide.
The primary results and OS results from the PROfound Phase III trial were published in The New England Journal of Medicine.
Lynparza is approved in the US to treat men with homologous recombination repair gene-mutated (HRRm) mCRPC and in the EU, Japan and several other countries for BRCA-mutated mCRPC patients based on the PROfound Phase III trial. In addition, regulatory reviews are ongoing in other countries around the world.
AstraZeneca and MSD are testing Lynparza in additional trials in metastatic prostate cancer, including the ongoing PROpel Phase III trial of Lynparza as a 1st-line treatment for patients with mCRPC in combination with abiraterone versus abiraterone alone. Results are anticipated in the second half of 2021.
BRCA1 and BRCA2
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. 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 genetic alterations that can lead to cancer and confer sensitivity to PARP inhibitors, including Lynparza.9-12
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 HRR, such as mutations in BRCA1 and/or BRCA2. Inhibition of PARP 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 being tested in a range of PARP-dependent tumour types with defects and dependencies in the DDR pathway.
Lynparza is currently approved in several countries, including those in the EU, for the maintenance treatment of platinum-sensitive relapsed ovarian cancer. It is approved in the US, the EU, Japan, China, and several other countries as 1st-line maintenance treatment of BRCA-mutated (BRCAm) advanced ovarian cancer following response to platinum-based chemotherapy. It is also approved in the US, the EU, and Japan as 1st-line maintenance treatment with bevacizumab for patients with HRD-positive advanced ovarian cancer (BRCAm and/or genomic instability). In addition, Lynparza is approved in the US, Japan, and several other countries for germline BRCAm, HER2-negative, metastatic breast cancer, previously treated with chemotherapy; in the EU, this includes locally advanced breast cancer. It is also approved in the US, the EU, Japan, and several other countries to treat germline BRCAm metastatic pancreatic cancer. In addition, Lynparza is approved in the US for HRR gene-mutated mCRPC (BRCAm and other HRR gene mutations) and in the EU and Japan for BRCAm metastatic castration-resistant prostate cancer. Regulatory reviews are underway in several countries for ovarian, breast, pancreatic and prostate cancers.
Lynparza, which is being jointly developed and commercialised by AstraZeneca and MSD, has been used to treat over 40,000 patients worldwide. Lynparza has the broadest and most advanced clinical trial development programme of any PARP inhibitor. AstraZeneca and MSD are working together to understand how it may affect multiple PARP-dependent tumours as a monotherapy and in combination across multiple cancer types. Lynparza 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. Independently, the companies will develop Lynparza and Koselugo in combination with their respective PD-L1 and PD-1 medicines.
Please visit astrazeneca.com
AstraZeneca/Merck's Lynparza OK'd in China BRCA-mutated prostate cancer
Jun. 24, 2021 7:09 AM ETAstraZeneca PLC (AZN), MRKBy: Mamta Mayani, SA News Editor
- AstraZeneca (NASDAQ:AZN) and Merck (NYSE:MRK) announce that Lynparza has been granted conditional approval in China as monotherapy for the treatment of adults with germline or somatic BRCA-mutated metastatic castration-resistant prostate cancer (mCRPC) who have progressed following prior treatment that included a new hormonal agent (abiraterone, enzalutamide).
- https://seekingalpha.com/news/3709455-astrazenecamercks-lynparza-okd-in-china-brca-mutated-prostate-cancer
- https://seekingalpha.com/symbol/MRK
- https://seekingalpha.com/symbol/AZN
- https://www.lynparza.com/
- https://www.astrazeneca.com/
Aimovig® (Erenumab)
Amgen Announces Approval Of Aimovig® (Erenumab) In Japan For The Suppression Of Onset Of Migraine Attacks In Adults
Migraine is a Disabling Neurological Disease that Affects More Than 8.4 Million People in Japan[1],[2]
Aimovig is the First and Only Approved Treatment in Japan to Block the Calcitonin Gene-Related Peptide Receptor (CGRP-R) That Plays an Important Role in Migraine[3]
Aimovig Continues to be the Most Utilized Anti-CGRP Pathway Therapy, With More Than Half a Million Patients Prescribed Worldwide[4]
THOUSAND OAKS, Calif., June 23, 2021 /PRNewswire/ -- Amgen (NASDAQ:AMGN) today announced that the Japanese Ministry of Health, Labour and Welfare has granted marketing approval for Aimovig® (erenumab) for the suppression of onset of migraine attacks in adults. Aimovig is the first and only approved treatment in Japan to block the calcitonin gene-related peptide receptor (CGRP-R), which is believed to play a critical role in migraine.3 This is also the first independent submission and approval for Amgen K.K., a wholly owned affiliate of Amgen Inc. headquartered in Tokyo.
"Today's approval further strengthens Amgen's commitment to the migraine community, and we continue to look for ways to expand the availability of Aimovig to help more patients," said Murdo Gordon, executive vice president of Global Commercial Operations at Amgen. "We've seen how much Aimovig has already helped many people living with migraine around the world. Having this treatment approved in Japan will enable us to ultimately serve more patients and help them find the right treatment for this disabling, neurological disease."
Aimovig's approval in Japan is based on results from a Phase II study (20120309) evaluating the safety and efficacy of Aimovig in adult Japanese patients with episodic migraine, and a Phase III study (20170609) evaluating the efficacy and safety of Aimovig in adult Japanese patients with episodic and chronic migraine. In both studies, Aimovig significantly reduced monthly migraine days (MMD) from baseline over months 4, 5 and 6 of the double-blind treatment period (DBTP).5,6 The safety and tolerability of Aimovig was also consistent with previously available global data. The most commonly reported adverse reactions include constipation, injection site reactions and somnolence at an incidence of 1% or more.5,6
About the 20120309 Study
The 20120309 study is a Phase II, randomized, placebo-controlled study comprised of a six-month (24 weeks) DBTP that evaluated the safety and efficacy of erenumab for the prevention of episodic migraine in adult Japanese patients. Participants were randomized to receive subcutaneous administration of placebo or erenumab 28 mg, 70 mg or 140 mg once every four weeks for six months. The primary endpoint was the change from baseline in mean MMD over months 4, 5, and 6 of the DBTP.5
About the 20170609 Study
The 20170609 study is a Phase III, randomized, placebo-controlled study comprised of a six-month (24 weeks) DBTP that evaluated the safety and efficacy of erenumab for migraine prevention in adult Japanese patients with episodic or chronic migraine. There were 261 participants randomized with a 1:1 allocation to receive subcutaneous administration of placebo or erenumab 70 mg once every four weeks. The primary endpoint was the change from baseline in mean MMD over months 4, 5, and 6 of the DBTP. Secondary endpoints were the proportion of participants with at least a 50% reduction in MMD and the change from baseline in mean monthly acute migraine-specific medication treatment days over months 4, 5, and 6 of the DBTP.6
About Aimovig in the U.S.
Aimovig (erenumab-aooe) is the first FDA-approved migraine preventive treatment that targets the calcitonin gene-related peptide (CGRP) receptor, which is associated with migraine.3,14 Aimovig has been studied in several large, global, randomized, double-blind, placebo-controlled studies to assess its efficacy and safety in migraine prevention.15,16 Aimovig is self-administered once monthly via the easy-to-use SureClick® autoinjector, without a required loading dose.14,17 More than 3,000 patients participated in registrational trials of Aimovig across four placebo-controlled Phase 2 and Phase 3 clinical studies and their open-label extensions.15,16,18 - 20
Aimovig is also being evaluated through CATALYST, a comprehensive evidence generation program initiated by Amgen and Novartis that includes over 7,500 patients across ongoing clinical trials and a robust assessment of real-world evidence. Spanning over 39 countries globally, CATALYST clinical trials will explore the role of Aimovig in comparative studies, assessing impact on novel migraine outcomes, understanding predictive biomarkers and investigating Aimovig's use in additional study populations. To date, more than 450,000 patients in the United States and 500,000 patients worldwide have been prescribed Aimovig for the preventive treatment of migraine in adults.4,21
IMPORTANT SAFETY INFORMATION REGARDING AIMOVIG U.S. INDICATION
Aimovig® (erenumab-aooe) is indicated for the preventive treatment of migraine in adults.
Contraindication: Aimovig® is contraindicated in patients with serious hypersensitivity to erenumab-aooe or to any of the excipients. Reactions have included anaphylaxis and angioedema.
Please see Aimovig® full Prescribing Information.
For more information, visit www.amgen.com
View original content to download multimedia:http://www.prnewswire.com/news-releases/amgen-announces-approval-of-aimovig-erenumab-in-japan-for-the-suppression-of-onset-of-migraine-attacks-in-adults-301318079.html
SOURCE Amgen
Amgen's Aimovig OK'd in Japan for migraine attacks in adults
Jun. 23, 2021 4:54 AM ET Amgen Inc. (AMGN) By: Mamta Mayani, SA News Editor
- Amgen (NASDAQ:AMGN) announces that the Japanese Ministry of Health, Labour and Welfare has granted marketing approval for Aimovig (erenumab) for the suppression of onset of migraine attacks in adults.
- https://seekingalpha.com/news/3708996-amgens-aimovig-okd-in-japan-for-migraine-attacks-in-adults
- https://seekingalpha.com/symbol/AMGN
- https://www.amgen.com/
- https://seekingalpha.com/symbol/NVS
- https://www.aimovig.com/
XARELTO® (rivaroxaban)
Janssen Submits New Drug Application to U.S. FDA for XARELTO® (rivaroxaban) to Help Prevent and Treat Blood Clots in Pediatric PatientsApplication seeks two pediatric indications, including an age-appropriate new weight-based oral suspension formulation to help minimize dosing errors
If approved, XARELTO® will be the first and only oral Factor Xa inhibitor indicated in the U.S. for use in appropriate pediatric patients
RARITAN, NJ, June 23, 2021 – The Janssen Pharmaceutical Companies of Johnson & Johnson announced today it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the use of XARELTO® (rivaroxaban) in pediatric patients. The NDA seeks two pediatric indications: treatment of venous thromboembolism (VTE, or blood clots) and reduction in the risk of recurrent VTE in patients aged birth to less than 18 years of age after at least five days of initial parenteral anticoagulant treatment; and thromboprophylaxis (prevention of blood clots) in patients aged 2 years and older with congenital heart disease who have undergone the Fontan procedure. If approved, XARELTO® will be the first and only oral Factor Xa inhibitor indicated in the U.S. for use in pediatric patients.
CLICK TO TWEET: @JanssenUS announces NDA submission to @US_FDA for two new potential pediatric indications for the treatment and prevention of #bloodclots. Learn more: https://ctt.ac/dS3qz+
Current guidelines are limited and recommend treating pediatric patients with or at risk for reoccurring blood clots with standard anticoagulant therapy, which often requires painful injections, dietary restrictions and regular laboratory monitoring. There are currently no FDA-approved anticoagulation therapies for pediatric patients with congenital heart disease who have undergone the Fontan procedure, a surgical procedure that redirects blood flow from the lower body to the lungs. The limited guidance for managing these patients leaves physicians to extrapolate adult data to infer pediatric dosing and then regularly monitor their patients.
About EINSTEIN-Jr
EINSTEIN-Jr is a randomized, multicenter, active-controlled, open-label Phase 3 study that evaluated the use of XARELTO® in 500 children, aged birth to 17 years, with previously diagnosed acute VTE who had started parenteral anticoagulation therapy. Participants were enrolled from November 2014 to September 2018, from 107 sites in 28 countries, and were assigned in a 2:1 ratio to receive either an open-label, body weight-adjusted dose of XARELTO® (tablets or new oral suspension) (n=335) to approximate a 20 mg adult dose or standard anticoagulation therapy (n=165). EINSTEIN-Jr is the largest pediatric study completed to date for the treatment of VTE. It is part of the comprehensive EINSTEIN program, which also included four pivotal Phase 3 studies in adult populations: EINSTEIN-DVT, EINSTEIN-PE, EINSTEIN-EXT and EINSTEIN CHOICE.
About UNIVERSE
UNIVERSE is a randomized, multicenter, open-label, active controlled, two-part, Phase 3 study that examined the use of a novel, oral suspension XARELTO® formulation in children 2-8 years old with single ventricle physiology who had the Fontan procedure within four months before enrollment. From November 2016 to June 2019, a total of 112 participants were enrolled across 36 sites in 10 countries.
UNIVERSE was conducted in two parts. Part A evaluated the single- and multiple-dose PK and PD properties of XARELTO® while Part B evaluated the comparative safety and efficacy of XARELTO® versus aspirin when used for thromboprophylaxis for 12 months.
About EXPLORER
The EXPLORER clinical research program is unmatched by any oral anticoagulant in the Factor Xa inhibitor class in its size, scope and ambition. A collaborative effort between Janssen and Bayer, EXPLORER seeks to generate important clinical evidence on the safety and efficacy of XARELTO® and its potential role in addressing critical unmet medical needs. By the time of its completion, more than 275,000 patients will have participated in the EXPLORER clinical development program, other completed and ongoing clinical trials, investigative registries, and non-interventional studies. Since its inception, the program has built upon extensive evidence reinforcing the benefits of XARELTO® for the millions of patients to whom it has been prescribed since its launch in 2011.
WHAT IS XARELTO® (rivaroxaban)?
XARELTO® is a prescription medicine used to:
- reduce the risk of stroke and blood clots in people who have a medical condition called atrial fibrillation that is not caused by a heart valve problem. With atrial fibrillation, part of the heart does not beat the way it should. This can lead to the formation of blood clots, which can travel to the brain, causing a stroke, or to other parts of the body
- treat blood clots in the veins of your legs (deep vein thrombosis or DVT) or lungs (pulmonary embolism or PE)
- reduce the risk of blood clots happening again in people who continue to be at risk for DVT or PE after receiving treatment for blood clots for at least 6 months
- help prevent a blood clot in the legs and lungs of people who have just had hip or knee replacement surgery
- help prevent blood clots in certain people hospitalized for an acute illness and after discharge, who are at risk of getting blood clots because of the loss of or decreased ability to move around (mobility) and other risks for getting blood clots, and who do not have a high risk of bleeding
XARELTO® is used with low dose aspirin to:
- reduce the risk of serious heart problems, heart attack and stroke in people with coronary artery disease (a condition where the blood supply to the heart is reduced or blocked) or peripheral artery disease (a condition where the blood flow to the legs is reduced)
It is not known if XARELTO® is safe and effective in children.
Please read full Prescribing Information, including Boxed Warnings, and Medication Guide for XARELTO®.
Please visit www.janssen.com/cardiovascular-and-metabolism.
Learn more at www.janssen.com.
J&J Janssen division submits Xarelto NDA for blood clots in children
Jun. 23, 2021 11:13 AM ET Johnson & Johnson (JNJ)
By: Jonathan M Block, SA News Editor
- The Janssen division of Johnson and Johnson (NYSE:JNJ) has submitted an NDA to the FDA for its blood thinner Xarelto (rivaroxaban) for venous thromboembolism, also known as blood clots, in children.
- https://seekingalpha.com/news/3709231-johnson-johnson-janssen-division-submits-xarelto-nda-for-blood-clots-in-children
- https://seekingalpha.com/symbol/JNJ
- https://www.xarelto-us.com/
Veklury® (Remdesivir)
June 21, 2021
Gilead’s Veklury® (Remdesivir) Associated With a Reduction in Mortality Rate in Hospitalized Patients With COVID-19 Across Three Analyses of Large Retrospective Real-World Data Sets
– Real-World Evidence from Nearly 100,000 Hospitalized Patients Provides Clinical Insights on the Use of Veklury for the Treatment of COVID-19 –FOSTER CITY, Calif.--(BUSINESS WIRE)-- Gilead Sciences, Inc. (Nasdaq: GILD) today announced positive data from three retrospective studies of the real-world treatment of patients hospitalized with COVID-19, adding to the body of mortality and hospital discharge data for patients treated with Veklury® (remdesivir). Presented at the World Microbe Forum (WMF) this week, all three of the real-world analyses observed that, in the overall patient populations, patients who received Veklury treatment had significantly lower risk for mortality compared with matched controls. A reduction in mortality was observed across a spectrum of baseline oxygen requirements. The results were consistently observed at different timeframes over the course of the pandemic and across geographies. Two of the studies also observed that patients who received Veklury had a significantly increased likelihood of discharge from the hospital by Day 28.https://www.vekluryhcp.com/
In the United States, Veklury is indicated for adults and pediatric patients (12 years of age and older and weighing at least 40 kg) for the treatment of COVID-19 requiring hospitalization. Veklury is contraindicated in patients who are allergic to Veklury or any of its components; please see below for additional Important Safety Information for Veklury.
Aetion and HealthVerity Analysis (iPoster #WMF21-2970)This retrospective, real-world comparative analysis of U.S.-based claims data from HealthVerity, performed in collaboration with Aetion, assessed mortality and the likelihood of discharge in hospitalized COVID-19 patients who were treated with Veklury (n=24,856) versus matched controls (n=24,856) between May 1, 2020 and May 3, 2021. Controls were matched 1:1 to patients treated with Veklury using risk set sampling on date of admission, number of days from admission to start of Veklury, age, gender, baseline oxygen support requirement and corticosteroid use. A 1:1 propensity score matching was applied to establish comparable groups based on baseline clinical and demographic characteristics, comorbidities and concomitant medications. The primary endpoint was time to death.This analysis found that in the overall population, patients receiving Veklury had a statistically significant 23% lower mortality risk compared with controls (HR:0.77, 95% CI:0.73 to 0.81), regardless of baseline oxygen requirement. Overall, a significantly greater likelihood of discharge by Day 28 was observed in patients completing a full five-day course of Veklury compared with controls (HR:1.19, 95% CI:1.14 to 1.25); this result was most pronounced in patients with lower oxygen requirements at baseline.
Premier Analysis (iPoster #WMF21-2507)This retrospective, real-world comparative analysis of data from the Premier Healthcare Database assessed mortality in hospitalized patients who were treated with Veklury (n=28,855) versus matched patients who were not treated with Veklury (n=16,687) between August and November 2020. The analysis included adult hospitalized patients who were treated with Veklury within the first two days of hospitalization with those not treated with Veklury during their hospitalization. Patients were matched on baseline level of oxygenation, hospital, within a two-month hospital admission period, and all stayed in the hospital for a minimum of three days after initiating treatment. The primary endpoint was time to death.In this analysis, patients overall who were treated with Veklury had a significantly lower risk of mortality both at Day 14 (HR:0.76, 95% CI:0.70 to 0.83, p<0.0001) and at Day 28 (HR:0.89, 95% CI:0.82 to 0.96, p=0.003) compared with patients who did not receive Veklury. Patients who were treated with Veklury and received either no oxygen (HR:0.69, 95%, CI:0.57 to 0.83, p<0.001), low-flow oxygen (HR:0.68, 95% CI:0.60 to 0.77, p<0.0001) or invasive mechanical ventilation/ECMO (HR:0.70, 95% CI:0.58 to 0.84, p=0.0001) at baseline had a significantly lower risk of 14-day mortality. A significant reduction in mortality was also noted at 28 days for these same groups of patients, no oxygen (HR:0.80, 95%, CI:0.68 to 0.94, p=0.007), low-flow oxygen (HR:0.77, 95% CI:0.68 to 0.86, p<0.0001) or invasive mechanical ventilation/ECMO (HR:0.81, 95% CI:0.69 to 0.94, p=0.007). Patients on high-flow oxygen at baseline who received Veklury also had significantly lower 14-day mortality (HR:0.81, 95% CI:0.70 to 0.93, p=0.0043); at 28 days, the difference in mortality in patients receiving high-flow oxygen at baseline was not statistically significant (HR:0.97, 95% CI:0.84 to 1.11, p=0.646).
SIMPLE-Severe Analysis (iPoster #WMF21-2969)The SIMPLE-Severe study was a randomized, open-label, multicenter, Phase 3 study in hospitalized adult patients with severe COVID-19 (oxygen saturation of ≥94% on room air, or receiving supplemental oxygen and radiological evidence of pneumonia); these results have been previously presented. As the primary objective of the study was to evaluate five-day and 10-day dosing durations of Veklury, the initial phase of the study did not include a standard of care comparator arm. The retrospective real-world analysis presented at WMF compared mortality outcomes of hospitalized patients with COVID-19 who received Veklury in the open-label extension phase of the SIMPLE-Severe study (n=1,974) versus propensity-score weighted patients from a real-world retrospective longitudinal cohort study of hospitalized patients with COVID-19 who were not treated with Veklury (n=1,426). Propensity score weighting was used to ensure consistent baseline demographics, region, clinical characteristics, concomitant medications and comorbidities. The primary endpoint was time to all-cause death.This analysis found that in the overall population, treatment with Veklury was associated with a statistically significant 54% lower mortality risk at 28 days versus those not treated with Veklury, regardless of a patient’s baseline oxygen requirements (HR:0.46, 95% CI:0.39 to 0.54, p<0.001). Patients who completed a full 10-day course of Veklury had a significantly shorter time to discharge within 28 days compared with those who did not receive Veklury (HR:1.64, 95% CI:1.43 to 1.87, p<0.001); the result for time to discharge was not significant for patients receiving invasive mechanical ventilation or ECMO at baseline (HR:0.92, 95% CI:0.62 to 1.36, p=0.68).
ACTT-1The global, randomized, double-blind, placebo-controlled, Phase 3 clinical trial ACTT-1 (NTC04280705) sponsored by the National Institute of Allergy and Infectious Diseases (NIAID) evaluated the efficacy and safety of a 10-day treatment course of Veklury versus placebo in 1,063 hospitalized adult patients with mild, moderate or severe COVID-19 who also were receiving treatment with standard of care. The primary outcome measure was time to recovery within 29 days after randomization; overall mortality was a prespecified secondary endpoint. Study results and additional mortality data from a post-hoc analysis were published in the New England Journal of Medicine on October 8, 2020. These results have been previously presented.
U.S. Important Safety Information for Veklury
Contraindication
- Veklury is contraindicated in patients with a history of clinically significant hypersensitivity reactions to Veklury or any of its components.
U.S. Indication for Veklury
Veklury® (remdesivir 100 mg for injection) is indicated for adults and pediatric patients (12 years of age and older and weighing at least 40 kg) for the treatment of COVID-19 requiring hospitalization. Veklury should only be administered in a hospital or in a healthcare setting capable of providing acute care comparable to inpatient hospital care. https://www.gilead.com/remdesivir
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/20210621005210/en/ Source: Gilead Sciences, Inc.
Gilead highlights positive data from remdesivir COVID-19 retrospective studies
Jun. 21, 2021 8:24 AM ET Gilead Sciences, Inc. (GILD) By: Aakash Babu, SA News Editor
- Gilead Sciences (NASDAQ:GILD) announces positive data from three retrospective studies of the real-world treatment of patients hospitalized with COVID-19 treated with Veklury (remdesivir).
- https://seekingalpha.com/news/3708038-gilead-highlights-positive-data-from-remdesivir-covid-19-retrospective-studies
- https://seekingalpha.com/symbol/GILD
Tislelizumab (BGB-A317)
China NMPA Approves Tislelizumab in Non-Small Cell Lung Cancer and Hepatocellular Carcinoma
June 23, 2021 at 12:00 AM EDT
Tislelizumab is approved for the first-line treatment of advanced non-squamous non-small cell lung cancer following the previously approved squamous histology
Tislelizumab receives its first approval in liver cancer in previously treated hepatocellular carcinoma
CAMBRIDGE, Mass. & BEIJING--(BUSINESS WIRE)--Jun. 23, 2021-- BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a global biotechnology company focused on developing and commercializing innovative medicines worldwide, today announced that the China National Medical Products Administration (NMPA) has granted its anti-PD-1 antibody tislelizumab approval for the first-line treatment of patients with advanced non-squamous non-small cell lung cancer (NSCLC) and conditional approval for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with at least one systemic therapy.
“With today’s approvals, tislelizumab is now available in China in five indications covering lung, liver, bladder, and lymphoma, and is becoming an important immunotherapy in the world’s most populous country,” commented Xiaobin Wu, Ph.D., President, Chief Operating Officer, and General Manager of China at BeiGene. “This remarkable achievement, which began approximately 18 months ago with tislelizumab’s initial approval, was made possible through BeiGene’s integrated global clinical development approach. We hope to make tislelizumab available broadly in China through our science-based commercial team and globally through our collaboration with Novartis, in furtherance of our goal of expanding access to innovative, quality cancer treatments for more people worldwide.”
About Tislelizumab
Tislelizumab (BGB-A317) is a humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to FcγR on macrophages. In pre-clinical studies, binding to FcγR on macrophages has been shown to compromise the anti-tumor activity of PD-1 antibodies through activation of antibody-dependent macrophage-mediated killing of T effector cells. Tislelizumab is the first drug from BeiGene’s immuno-oncology biologics program and is being developed internationally as a monotherapy and in combination with other therapies for the treatment of a broad array of both solid tumor and hematologic cancers.
The China National Medical Products Administration (NMPA) has granted tislelizumab in five indications, including full approval for first-line treatment of patients with advanced squamous non-small cell lung cancer (NSCLC) in combination with chemotherapy and for first-line treatment of patients with advanced non-squamous NSCLC in combination with chemotherapy; and conditional approval for the treatment of patients with classical Hodgkin’s lymphoma (cHL) who received at least two prior therapies, for the treatment of patients with locally advanced or metastatic urothelial carcinoma (UC) with PD-L1 high expression whose disease progressed during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy, and for the treatment of patients with hepatocellular carcinoma (HCC) who have received at least one systemic therapy. Full approval for these indications is contingent upon results from ongoing randomized, controlled confirmatory clinical trials.
In addition, two supplemental Biologics License Applications for tislelizumab have been accepted by the Center for Drug Evaluation (CDE) of the NMPA and are under review for second- or third-line treatment of patients with locally advanced or metastatic NSCLC who progressed on prior platinum-based chemotherapy and for patients with previously treated, locally advanced unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair-deficient (dMMR) solid tumors.
BeiGene has initiated or completed 17 potentially registration-enabling clinical trials in China and globally, including 13 Phase 3 trials and four pivotal Phase 2 trials.
In January 2021, BeiGene and Novartis entered into a collaboration and license agreement granting Novartis rights to develop, manufacture, and commercialize tislelizumab in North America, Europe, and Japan.
Tislelizumab is not approved for use outside of China.
About the Tislelizumab Clinical Program
Clinical trials of tislelizumab include:
- Phase 3 trial comparing tislelizumab with docetaxel in the second- or third-line setting in patients with NSCLC (NCT03358875);
- Phase 3 trial comparing tislelizumab to salvage chemotherapy in patients with relapsed or refractory classical Hodgkin Lymphoma (cHL; NCT04486391);
- Phase 3 trial in patients with locally advanced or metastatic urothelial carcinoma (NCT03967977);
- Phase 3 trial of tislelizumab in combination with chemotherapy versus chemotherapy as first-line treatment for patients with advanced squamous NSCLC (NCT03594747);
- Phase 3 trial of tislelizumab in combination with chemotherapy versus chemotherapy as first-line treatment for patients with advanced non-squamous NSCLC (NCT03663205);
- Phase 3 trial of tislelizumab in combination with platinum-based doublet chemotherapy as neoadjuvant treatment for patients with NSCLC (NCT04379635);
- Phase 3 trial of tislelizumab combined with platinum and etoposide versus placebo combined with platinum and etoposide in patients with extensive-stage small cell lung cancer (NCT04005716);
- Phase 3 trial comparing tislelizumab with sorafenib as first-line treatment for patients with hepatocellular carcinoma (HCC; NCT03412773);
- Phase 2 trial in patients with previously treated unresectable HCC (NCT03419897);
- Phase 2 trial in patients with locally advanced or metastatic urothelial bladder cancer (NCT04004221);
- Phase 3 trial comparing tislelizumab with chemotherapy as second-line treatment for patients with advanced esophageal squamous cell carcinoma (ESCC; NCT03430843);
- Phase 3 trial of tislelizumab in combination with chemotherapy as first-line treatment for patients with ESCC (NCT03783442);
- Phase 3 trial of tislelizumab versus placebo in combination with chemoradiotherapy in patients with localized ESCC (NCT03957590);
- Phase 3 trial of tislelizumab combined with chemotherapy versus placebo combined with chemotherapy as first-line treatment for patients with gastric cancer (NCT03777657);
- Phase 2 trial of tislelizumab in patients with relapsed or refractory cHL (NCT03209973);
- Phase 2 trial in patients with MSI-H/dMMR solid tumors (NCT03736889); and
- Phase 3 trial of tislelizumab combined with chemotherapy versus placebo combined with chemotherapy as first-line treatment in patients with nasopharyngeal cancer (NCT03924986).
To learn more about BeiGene, please visit www.beigene.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20210622006142/en/
Source: BeiGene, Ltd.
BeiGene's tislelizumab OK'd in China in lung and liver cancer
Jun. 23, 2021 12:22 AM ET
By: Mamta Mayani, SA News Editor
- The China National Medical Products Administration (NMPA) has approved BeiGene's (NASDAQ:BGNE) anti-PD-1 antibody tislelizumab for the first-line treatment of patients with advanced non-squamous non-small cell lung cancer (NSCLC) and granted conditional approval for the treatment of patients with hepatocellular carcinoma (HCC) previously treated with at least one systemic therapy.
- https://seekingalpha.com/news/3708979-beigenes-tislelizumab-okd-in-china-in-lung-and-liver-cancer
- https://seekingalpha.com/symbol/BGNE
- https://www.beigene.com/en-us/science-and-product-portfolio/pipeline/tislelizumab
BRUKINSA® (Zanubrutinib)
BeiGene Announces China NMPA Approval of BRUKINSA® (Zanubrutinib) for the Treatment of Patients with Relapsed or Refractory Waldenström’s Macroglobulinemia
June 18, 2021 at 4:30 PM EDT
Third approval for BRUKINSA in China and second approval in WM worldwide
CAMBRIDGE, Mass. & BEIJING--(BUSINESS WIRE)--Jun. 18, 2021-- BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a global biotechnology company focused on developing and commercializing innovative medicines worldwide, today announced that BRUKINSA® (zanubrutinib) has received conditional approval from the China National Medical Products Administration (NMPA) for the treatment of adult patients with Waldenström’s macroglobulinemia (WM) who have received at least one prior therapy. The supplemental new drug application was previously granted priority review by the Center for Drug Evaluation (CDE) of the NMPA in October 2020.
“With NMPA approval for BRUKINSA, our next generation BTK inhibitor, we are proud to be able to offer patients, their families, and physicians a new option for treating WM, an incurable disease that can cause significant morbidities,” said Xiaobin Wu, Ph.D., General Manager of China and President of BeiGene. “This marks BRUKINSA’s third approval for the treatment of B-cell malignancies in China, and we believe it may serve an important role in addressing unmet needs for patients with blood cancers around the world.”
BRUKINSA was specifically designed by BeiGene scientists to limit off-target effects seen with first-generation BTK inhibitors and we have built a broad clinical development program to assess clinical benefit, including the head-to-head ASPEN trial,” said Jane Huang, M.D., Chief Medical Officer, Hematology, BeiGene. “We are grateful to all of the patients and clinicians who participated in the trial and hope to increase access for additional people impacted by WM and other hematologic malignancies as we advance global registration for BRUKINSA.”
The conditional approval granted by the NMPA was based on findings from a single-arm pivotal Phase 2 trial (NCT03332173) in China evaluating the safety and efficacy of BRUKINSA in patients with WM who have received at least one prior therapy. With a median study follow-up time of 14.9 months, the primary endpoint of major response rate (MRR) as assessed by independent review committee (IRC) was 72.1% (95% CI: 56.3, 84.7); MRR is defined as the combined rate of complete responses, very good partial responses, and partial responses. The adverse reaction profile was generally consistent with previous findings.
The recommended total daily dose of BRUKINSA for WM in China is 320mg.
About BRUKINSA® (zanubrutinib)
BRUKINSA is a small molecule inhibitor of Bruton’s tyrosine kinase (BTK) discovered by BeiGene scientists that is currently being evaluated globally in a broad clinical program as a monotherapy and in combination with other therapies to treat various B-cell malignancies. Because new BTK is continuously synthesized, BRUKINSA was specifically designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity. With differentiated pharmacokinetics compared to other approved BTK inhibitors, BRUKINSA has been demonstrated to inhibit the proliferation of malignant B cells within a number of disease relevant tissues.
BRUKINSA is approved in the following indications and regions:
- For the treatment of mantle cell lymphoma (MCL) in adult patients who have received at least one prior therapy (United States, November 2019)*;
- For the treatment of MCL in adult patients who have received at least one prior therapy (China, June 2020)**;
- For the treatment of chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) in adult patients who have received at least one prior therapy (China, June 2020)**;
- For the treatment of adult patients with Waldenström’s macroglobulinemia (WM) who have received at least one prior therapy (China, June 2021)**;
- For the treatment of relapsed or refractory MCL (United Arab Emirates, February 2021); and
- For the treatment of WM in adult patients (Canada, March 2021).
To-date, more than 30 marketing authorization applications in multiple indications have been submitted outside of the United States and China, covering countries in the European Union and more than 20 other countries.
* This indication was approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
** This indication was approved under conditional approval. Complete approval for this indication may be contingent upon results from ongoing randomized, controlled confirmatory clinical trials.
INDICATION
BRUKINSA is a kinase inhibitor indicated for the treatment of adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy.
This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
Please see full U.S. Prescribing Information at www.beigene.com/PDF/BRUKINSAUSPI.pdf and Patient Information at www.beigene.com/PDF/BRUKINSAUSPPI.pdf.
To learn more about BeiGene, please visit www.beigene.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20210618005318/en/
Source: BeiGene
BeiGene's BRUKINSA gets China NMPA conditional approval for rare form of lymphoma
Jun. 18, 2021 4:40 PM ETBeiGene, Ltd. (BGNE)By: Aakash Babu, SA News Editor1 Comment
- BeiGene (NASDAQ:BGNE) announces that BRUKINSA (zanubrutinib) has received conditional approval from the China National Medical Products Administration (NMPA) for the treatment of adult patients with Waldenström’s macroglobulinemia (WM) who have received at least one prior therapy.
- https://seekingalpha.com/news/3707834-beigenes-brukinsa-gets-china-nmpa-conditional-approval-for-rare-form-of-lymphoma
- https://seekingalpha.com/symbol/BGNE
- https://www.beigene.com/?loc=us
- https://www.brukinsa.com/
savolitinib
Oncology / Immunology, RNS Announcements | 22 Jun 2021
HUTCHMED announces savolitinib approved in China for patients with lung cancer with MET exon 14 skipping alterations
– First selective MET inhibitor approval in China in this setting –
– First regulatory approval for the oral, potent and selective MET tyrosine kinase inhibitor –
Hong Kong, Shanghai & Florham Park, NJ —Tuesday, June 22, 2021: HUTCHMED (China) Limited (“HUTCHMED”) (Nasdaq/AIM: HCM) today announces that AstraZeneca PLC (“AstraZeneca”) and HUTCHMED’s savolitinib has been granted conditional approval in China for the treatment of patients with non-small cell lung cancer (“NSCLC”) with MET exon 14 skipping alterations who have progressed following prior systemic therapy or are unable to receive chemotherapy. This approval follows a priority review designation by China’s National Medical Products Administration (“NMPA”) and marks the first regulatory approval globally for this oral, potent and selective MET tyrosine kinase inhibitor (“TKI”).
Approximately 2-3% of newly diagnosed NSCLC patients have MET exon 14 skipping alterations, a specific genetic mutation.
The approval by the NMPA was based on positive results from a Phase II trial conducted in China in patients with NSCLC with this mutation, including patients with the more aggressive pulmonary sarcomatoid carcinoma subtype. Savolitinib demonstrated effective anti-tumor activity based on an independent review of objective response rate (“ORR”) and disease control rate (“DCR”). The approval is conditional upon successful completion of a confirmatory study in this patient population.
About savolitinib
Savolitinib is an oral, potent and selective MET TKI that has demonstrated clinical activity in advanced solid tumors. It blocks atypical activation of the MET receptor tyrosine kinase pathway that occurs because of mutations (such as exon 14 skipping alterations).
Savolitinib is currently under development for multiple tumor types, including lung, kidney and gastric cancers, as a single treatment and in combination with other medicines.
HUTCHMED and AstraZeneca collaboration
In 2011, HUTCHMED and AstraZeneca entered a global licensing agreement with respect to the development and commercialization of savolitinib. HUTCHMED is responsible for the manufacturing and supply of savolitinib, and AstraZeneca is responsible for its commercialization in China and worldwide.
For more information, please visit: www.hutch-med.com
Please visit astrazeneca.com
AstraZeneca, HUTCHMED's savolitinib gets conditional approval in China
Jun. 22, 2021 1:25 PM ETHUTCHMED (China) Limited (HCM)By: Aakash Babu, SA News Editor
- AstraZeneca (NASDAQ:AZN) and HUTCHMED's (HCM +0.7%) savolitinib has been granted conditional approval in China for the treatment of certain patients with non-small cell lung cancer (“NSCLC”).
- https://seekingalpha.com/news/3708803-astrazeneca-hutchmeds-savolitinib-gets-conditional-approval-in-china
- https://seekingalpha.com/symbol/HCM
- https://seekingalpha.com/symbol/AZN
- https://www.astrazeneca.com/
Sotrovimab (previously VIR-7831)
GSK and Vir Biotechnology Announce Continuing Progress of the COMET Clinical Development Program for Sotrovimab
06/21/21 at 7:06 AM EDTPDF Version
– Analysis of final Day 29 data from COMET-ICE confirms sotrovimab significantly reduces
hospitalization and risk of death in adults with mild-to-moderate COVID-19 who are at high risk
of progression to severe disease –
– U.S. National Institutes of Health (NIH) COVID-19 Treatment Guidelines
updated to recommend use of sotrovimab –
– Further research initiated to evaluate intramuscular administration of sotrovimab for the
early treatment of mild-to-moderate COVID-19 in high-risk patients in a Phase 3 study –
LONDON and SAN FRANCISCO, June 21, 2021 (GLOBE NEWSWIRE) -- GlaxoSmithKline plc (LSE/NYSE: GSK) and Vir Biotechnology, Inc. (Nasdaq: VIR) today announced final, confirmatory results from the Phase 3 COMET-ICE (COVID-19 Monoclonal antibody Efficacy Trial – Intent to Care Early) trial demonstrating that sotrovimab, an investigational SARS-CoV-2 monoclonal antibody, significantly reduced the risk of hospitalization or death among high-risk adult outpatients with mild-to-moderate COVID-19. Additionally, the U.S. National Institutes of Health (NIH) updated its COVID-19 treatment guidelines to recommend sotrovimab for non-hospitalized patients with mild-to-moderate COVID-19 who are at high risk of clinical progression and noted that sotrovimab appears to retain activity against current variants of concern and interest.
The primary efficacy analysis of all 1,057 patients in the COMET-ICE trial demonstrated a 79% reduction (adjusted relative risk reduction) (p<0.001) in hospitalization for more than 24 hours or death due to any cause, by Day 29 compared to placebo, meeting the primary endpoint of the trial.
Updated NIH Guidelines Recommend Sotrovimab
The NIH recently updated its guidelines regarding the emergency use authorizations of anti-SARS-CoV-2 monoclonal antibodies for the treatment of COVID-19 in the U.S. to recommend the use of sotrovimab for non-hospitalized patients with mild-to-moderate COVID-19 who are at high risk of clinical progression. The guidelines note that the target binding site of sotrovimab is in a region of the virus that does not overlap with the binding site location of key mutations in current variants of concern and interest. These guidelines were based upon an interim analysis of 583 patients in the COMET-ICE trial, which was stopped early in March 2020 by an independent data monitoring committee because interim results demonstrated evidence of sotrovimab’s clinical efficacy. The interim study results demonstrated an 85% (p=0.002) reduction in hospitalization for more than 24 hours or death in those receiving sotrovimab compared to placebo, the primary endpoint of the trial.
These data have informed global regulatory reviews to date, including the positive scientific opinion issued by the European Medicines Agency’s (EMA) Committee for Human Medicinal Products (CHMP) under Article 5(3) of Regulation 726/2004, as well as the Emergency Use Authorization (EUA) granted by the U.S. Food and Drug Administration (FDA).
The companies are actively working with government agencies around the world to make sotrovimab available to patients in need of treatment.
- GSK and Vir plan to submit a Biologics License Application (BLA) to the U.S. FDA in the second half of 2021.
- The EMA has started a rolling review of data on sotrovimab that will continue until enough evidence is available to support the filing of a formal marketing authorization application.
- The companies’ strategic manufacturing network is enabling the manufacture of approximately two million doses to support emergency supply in the first year following U.S. Emergency Use Authorization, with approximately 450,000 doses on hand.
Continued Progress with the COMET Clinical Development Program
The companies are also pleased to announce continued progress with the robust COMET clinical development program, which aims to provide clinical evidence from several studies over the course of the next year.
- COMET-PEAK, a pharmacokinetic study in outpatients with mild-to-moderate COVID-19 investigating intramuscular (IM) administration of sotrovimab, is near completion and initial data is expected in second half of 2021.
- COMET-TAIL has been initiated. This is a Phase 3 study evaluating the role of IM-administered sotrovimab for the early treatment of mild-to-moderate COVID-19 in high-risk non-hospitalized adult and pediatric patients (12 years of age and older). Data are anticipated in the first half of 2022.
- A prophylaxis study is planned in uninfected immunocompromised adults to determine whether IM-administered sotrovimab can prevent symptomatic COVID-19 infection.
GSK and Vir are committed to ongoing evaluation of sotrovimab as the COVID-19 landscape continues to evolve at different rates across the globe and new variants of concern and interest emerge. Data from in vitro studies, published in bioRxiv, have demonstrated that sotrovimab maintains activity against circulating variants of concern and interest, including the Gamma (P.1), Epsilon (B.1.427/B.1.429), Delta (B.1.617.2), Iota (B.1.526), Beta (B.1.351) and Alpha (B.1.1.7) variants. GSK and Vir are continuing to evaluate the ability of sotrovimab to maintain activity against new and emerging variants through in vitro studies. The clinical impact of this in vitro variants data is not yet known.
About Sotrovimab (previously VIR-7831)
Sotrovimab is an investigational SARS-CoV-2 monoclonal antibody. Preclinical data suggest it has the potential to both block viral entry into healthy cells and clear infected cells. The antibody binds to an epitope on SARS-CoV-2 that is shared with SARS-CoV-1 (the virus that causes SARS), indicating that the epitope is highly conserved, which may make it more difficult for resistance to develop. Sotrovimab, which incorporates Xencor’s Xtend™ technology, also has been designed to achieve high concentration in the lungs to ensure optimal penetration into airway tissues affected by SARS-CoV-2 and to have an extended half-life.
The following is a summary of information for sotrovimab. Healthcare providers in the U.S. should review the Fact Sheets for information on the authorized use of sotrovimab and mandatory requirements of the EUA. Please see the FDA Letter of Authorization, Fact Sheet for Healthcare Providers, and Fact Sheet for Patients, Parents, and Caregivers. For more information on the EMA positive scientific opinion, please review the EU Conditions of Use.
Important Information about Sotrovimab
Sotrovimab has been authorized by the FDA for the emergency use described below. Sotrovimab is not FDA-approved for this use.
Sotrovimab is authorized only for the duration of the declaration that circumstances exist justifying the authorization of the emergency use of sotrovimab under section 564(b)(1) of the Act, 21 U.S.C. § 360bbb-3(b)(1), unless the authorization is terminated or revoked sooner.
Authorized Use
The U.S. Food and Drug Administration (FDA) has issued an Emergency Use Authorization (EUA) to permit the emergency use of the unapproved product sotrovimab for the treatment of mild-to-moderate coronavirus disease 2019 (COVID-19) in adults and pediatric patients (12 years of age and older weighing at least 40 kg) with positive results of direct SARS-CoV-2 viral testing, and who are at high risk for progression to severe COVID-19, including hospitalization or death.
About the Vir and GSK Collaboration
In April 2020, Vir and GSK entered into a collaboration to research and develop solutions for coronaviruses, including SARS-CoV-2, the virus that causes COVID-19. The collaboration uses Vir’s proprietary monoclonal antibody platform technology to accelerate existing and identify new anti-viral antibodies that could be used as therapeutic or preventive options to help address the current COVID-19 pandemic and future outbreaks. The companies will leverage GSK’s expertise in functional genomics and combine their capabilities in CRISPR screening and artificial intelligence to identify anti-coronavirus compounds that target cellular host genes. They will also apply their combined expertise to research SARS-CoV-2 and other coronavirus vaccines.
For further information please visit www.gsk.com/about-us.
For more information, please visit www.vir.bio.
GSK, Vir Bio confirm sotrovimab reduces risk of hospitalization/death due to COVID-19
Jun. 21, 2021 7:38 AM ETGlaxoSmithKline plc (GSK), VIRBy: Mamta Mayani, SA News Editor
- GlaxoSmithKline (NYSE:GSK) and Vir Biotechnology (NASDAQ:VIR) announce final, confirmatory results from Phase 3 COMET-ICE trial demonstrating that sotrovimab, an investigational SARS-CoV-2 monoclonal antibody, significantly reduced the risk of hospitalization or death among high-risk adult outpatients with mild-to-moderate COVID-19.
- https://seekingalpha.com/news/3708007-gsk-vir-bio-confirm-sotrovimab-reduces-risk-of-hospitalizationdeath-due-to-covid-
- https://seekingalpha.com/symbol/VIR
- https://seekingalpha.com/symbol/GSK
Aubagio® (teriflunomide)
June 18 2021
European Commission approves Aubagio® (teriflunomide) as the first oral MS therapy for first-line treatment of children and adolescents living with relapsing-remitting multiple sclerosis
European Commission approves Aubagio® (teriflunomide) as the first oral MS therapy for first-line treatment of children and adolescents living with relapsing-remitting multiple sclerosis
PARIS – June 18, 2021 - The European Commission (EC) has approved Aubagio® (teriflunomide) for the treatment of pediatric patients 10 to 17 years of age with relapsing-remitting multiple sclerosis (RRMS). The EC approval is based on data from the Phase 3 TERIKIDS study. The approval confirms Aubagio as the first oral multiple sclerosis (MS) therapy for first-line treatment of children and adolescents with MS in the European Union.
Aubagio Efficacy and Safety in Pediatric Patients
The Phase 3 TERIKIDS study is a multicenter, randomized, double-blind, placebo-controlled, parallel-group trial that enrolled 166 pediatric patients with relapsing-remitting forms of MS across 22 countries worldwide. The study consisted of a screening period (up to four weeks), followed by a double-blind treatment period (up to 96 weeks after randomization). An open-label TERIKIDS Phase 3 trial extension is ongoing. The primary endpoint was time to first confirmed clinical relapse, with prespecified sensitivity analysis including time to high magnetic resonance imaging (MRI) activity as relapse equivalent. Additionally, patients who completed the double-blind period, or had high MRI activity, were eligible to continue into the open-label extension.
The primary efficacy results and safety and tolerability data from the double-blind core study period (up to 96 weeks after randomization) were initially presented at the 2020 EAN Virtual Congress.
In the study, 109 and 57 patients were randomized to teriflunomide and placebo, respectively.
The primary endpoint was not statistically significant with numerically a lower risk (-34%) of clinical relapse for teriflunomide vs placebo (median time: 75.3 vs 39.1 weeks; HR 95% CI 0.66 0.39, 1.1 P=0.29). Switches from double-blind to open-label treatment due to high MRI activity were more frequent than anticipated. Switches were more frequent and earlier in the placebo group vs teriflunomide (26% and 14%, respectively). This decreased study power for the primary endpoint.
In the pre-specified sensitivity analysis of the composite endpoint of time to first clinical relapse or high MRI activity meeting study criteria to switch to open label, teriflunomide significantly reduced the time to clinical relapse or switch due to high MRI activity by 43% relative to placebo (median time: 72.1 vs 37.0 weeks; HR 95% CI 0.57 0.37, 0.87 P=0.04).
Key secondary endpoints showed teriflunomide significantly reduced the number of T1 gadolinium (Gd) -enhancing lesions per MRI scan (relative reduction 75%; P<0.0001) as well as the number of new and enlarging T2 lesions per MRI scan (relative reduction 55%, P=0.0006).
In the study, teriflunomide was well tolerated and had a manageable safety profile in the pediatric population. The overall incidences of adverse events (AEs) and serious adverse events (SAEs) were similar in the teriflunomide group and the placebo group (88.1% vs 82.5%, and 11.0% vs 10.5%), respectively. There were no deaths in the study. AEs reported more frequently in the teriflunomide group than the placebo group (with a difference of ≥ 5%) included nasopharyngitis, upper respiratory tract infection, alopecia, paresthesia, abdominal pain, and increased blood creatine phosphokinase (≥ 3 times the upper limit of normal). Cases of pancreatitis were reported in 1.8% (2/109) of the teriflunomide-treated patients compared to none in the placebo group, in the double-blind phase. In pediatric patients treated with teriflunomide in the open-label phase of the study, two additional cases of pancreatitis and one case of serious acute pancreatitis (with pseudo-papilloma), were reported.
For more information on the TERIKIDS Phase 3 clinical trial visit www.clinicaltrials.gov.
About Aubagio® (teriflunomide)
Aubagio is approved in more than 80 countries to treat certain patients with relapsing-remitting multiple sclerosis, with additional marketing applications under review by regulatory authorities globally. Aubagio is supported by one of the largest clinical programs of any MS therapy, with more than 5,000 trial participants in 36 countries, as well as a Phase 4 study program with more than 3,600 patients currently enrolled. There is over 16 years of combined clinical and real-world experience with Aubagio. More than 110,000 patients are currently being treated with Aubagio commercially worldwide.
European commission approves Sanofi Genzyme's Aubagio for pediatric multiple sclerosis
Jun. 18, 2021 10:14 AM ET Sanofi (SNY) By: Aakash Babu, SA News Editor
- The European Commission (EC) has approved Sanofi (SNY -1.8%) company Genzyme's Aubagio (teriflunomide) for the treatment of pediatric patients 10 to 17 years of age with relapsing-remitting multiple sclerosis (RRMS).
- https://seekingalpha.com/news/3707690-european-commission-approves-sanofi-genzymes-aubagio-in-multiple-sclerosis-indication
- https://seekingalpha.com/symbol/SNY
biotecMAX™ April/May/June 2021 Insight
MORAb-202 Antibody Drug Conjugate
Eisai and Bristol Myers Squibb Enter Into Global Strategic Collaboration for Eisai’s MORAb-202 Antibody Drug Conjugate
06/17/2021CATEGORY:
TOKYO & NEW YORK--(BUSINESS WIRE)-- Eisai Co., Ltd. and Bristol-Myers Squibb Company (NYSE: BMY) announced today that the companies have entered into an exclusive global strategic collaboration agreement for the co-development and co-commercialization of MORAb-202, an antibody drug conjugate (ADC). MORAb-202 is Eisai’s first ADC and combines Eisai’s in house developed anti-folate receptor alpha (FRα) antibody, and Eisai’s anticancer agent eribulin, using an enzyme cleavable linker. It is a potential best-in-class FRα ADC with a favorable pharmacology profile and demonstrated single agent activity in patients with advanced solid tumors. Eisai is currently investigating MORAb-202 in FRα-positive solid tumors (inclusive of endometrial, ovarian, lung and breast cancers) in two studies: a Phase 1 clinical study in Japan and a Phase 1/2 clinical study in the United States. The companies are planning to move into the registrational stage of development for this asset as early as next year.
Under the agreement, Eisai and Bristol Myers Squibb will jointly develop and commercialize MORAb-202 in the following collaboration territories: Japan; China; countries in the Asia-Pacific region*; the United States; Canada; Europe, including the European Union and the United Kingdom; and Russia. Bristol Myers Squibb will be solely responsible for developing and commercializing the drug in regions outside of the collaboration territories. Eisai will remain responsible for the manufacturing and supply of MORAb-202 globally.
“MORAb-202 combines Eisai’s in-house discovered antibody and payload using the company's advanced chemistry capabilities.” said Haruo Naito, Chief Executive Officer at Eisai. “It is characterized by its payload of eribulin, which is a product of our modern synthetic organic chemistry that has already made contributions to patients with breast cancer and soft tissue sarcoma. Our collaboration with Bristol Myers Squibb will accelerate the development of MORAb-202 with the goal of bringing a potentially impactful treatment option to patients globally.”
“This global collaboration with Eisai is an important strategic fit for Bristol Myers Squibb as it extends our leading position in oncology with a differentiated asset that complements our broad solid tumor portfolio and leverages our deep internal development expertise.” said Giovanni Caforio, M.D., board chair and chief executive officer, Bristol Myers Squibb. “We look forward to collaborating with Eisai as we work to bring this potential treatment option to patients in need as soon as possible.”
For more information about Eisai, please visit www.eisai.com (for global), us.eisai.com (for U.S.) or www.eisai.eu (for Europe, Middle East, Africa)
For more information about Bristol Myers Squibb, visit us at BMS.com
About MORAb-202
MORAb-202 is Eisai’s first antibody drug conjugate (ADC) that is composed of Eisai’s in-house developed anticancer agent farletuzumab, a humanized IgG1 monoclonal antibody that binds to the folate receptor alpha (FRα), and Eisai’s in-house developed anticancer agent eribulin, using an enzyme cleavable linker. Eisai is currently conducting a Phase 1 clinical study in Japan and a Phase 1/2 clinical study in the United States, respectively, for MORAb-202 targeting FRα-positive solid tumors. After MORAb-202 enters the target FRα-positive cancer cells, the linker is enzymatically cleaved, releasing eribulin from the antibody leading to its antitumor activity. Furthermore, in non-clinical studies, MORAb-202 demonstrated a bystander effect, with antitumor activity on the FRα-negative cancer cells surrounding the FRα-positive cancer cells.
The payload eribulin (product name: Halaven) is the first in the halichondrin class of microtubule dynamics inhibitors with a novel mechanism of action. Structurally eribulin is a simplified and synthetically produced version of halichondrin B, a natural product isolated from the marine sponge Halichondria okadai, and functions by inhibiting the growth phase of microtubule dynamics which prevents cell division. Eribulin is currently approved for use in the treatment of breast cancer in over 75 countries worldwide, including Japan, the United States, Europe, China, and other countries in Asia. Furthermore, eribulin is approved for use in the treatment of liposarcoma (soft tissue sarcoma in Japan) in over 75 countries worldwide, including Japan, the United States, as well as countries in Europe and Asia.
Bristol Myers to pay more than $2 billion to Eisai in a deal for experimental cancer therapy
Jun. 18, 2021 7:17 AM ET Eisai Co., Ltd. (ESALF), BMY By: Dulan Lokuwithana, SA News Editor3 Comments
Bristol-Myers Squibb (NYSE:BMY) and Eisai (OTCPK:ESALF) have announced a global collaboration to jointly develop and commercialize MORAb-202, an antibody-drug conjugate (ADC) currently undergoing studies in Japan and the U.S. for a range of cancers.
https://seekingalpha.com/symbol/ESALF
https://seekingalpha.com/symbol/BMY
Onureg® (azacitidine tablets)
Bristol Myers Squibb Receives European Commission Approval for Onureg® (azacitidine tablets) as Frontline Oral Maintenance Therapy for Adults with Acute Myeloid Leukemia
Onureg is the first and only once-daily, frontline oral maintenance therapy in the European Union (EU) for patients with a broad range of acute myeloid leukemia (AML) subtypes
In the pivotal QUAZAR® AML-001 study, Onureg significantly improved overall survival and relapse-free survival in patients with AML
June 18, 2021 09:34 AM Eastern Daylight Time
PRINCETON, N.J.--(BUSINESS WIRE)--Bristol Myers Squibb (NYSE: BMY) today announced that the European Commission (EC) has granted full Marketing Authorization for Onureg® (azacitidine tablets) as a maintenance therapy in adult patients with acute myeloid leukemia (AML) who achieved complete remission (CR) or complete remission with incomplete blood count recovery (CRi) following induction therapy with or without consolidation treatment and who are not candidates for, including those who choose not to proceed to, hematopoietic stem cell transplantation (HSCT). Onureg is the first and only once-daily, frontline oral maintenance therapy to demonstrate significant overall survival and show a relapse-free survival benefit in patients with a broad range of AML subtypes.
About QUAZAR® AML-001
QUAZAR® AML-001, is a Phase 3, international, randomized, double-blind study. Eligible patients were ages 55 years or older, had newly diagnosed AML, intermediate or poor cytogenetics, had achieved first CR or CRi following intensive induction chemotherapy with or without consolidation treatment (per investigator preference prior to study entry) within four months (+/- 7 days) before randomization, and were not candidates for HSCT at the time of screening. The study enrolled 472 patients, randomized 1:1 to receive either Onureg 300 mg (N=238) or placebo (N=234) orally, once daily, for 14 days of a 28-day cycle, plus best supportive care and results were published in the New England Journal of Medicine in December 2020.*3,4
Median OS, the primary endpoint, from time of randomization was greater than two years (24.7 months; 95% CI: 18.7 to 30.5) in the Onureg arm compared to 14.8 months for placebo (HR: 0.69, 95% CI: 0.55 to 0.86; p=0.0009). The median duration of treatment was 12 cycles (1 to 82) for Onureg and 6 cycles with placebo (1 to 76). Median relapse-free survival was also significantly longer with Onureg than with placebo (10.2 months and 4.8 months, respectively; p<0.001). Overall health-related quality of life was preserved during Onureg treatment.4
The most common adverse events in both treatment arms were Grade 1 or 2 gastrointestinal events. Common Grade 3 or 4 adverse events were neutropenia (41% of patients treated with Onureg and 24% of patients with placebo) and thrombocytopenia (22% and 21%, respectively).4
*Individual results associated with regulatory authorities may differ from publication.
About Onureg®
Onureg, the first and only frontline maintenance therapy approved in the European Union for patients with a broad range of AML subtypes, is a once-daily oral hypomethylating agent that incorporates into DNA and RNA. The main mechanism of action is thought to be hypomethylation of DNA, as well as direct cytotoxicity to abnormal hematopoietic cells in the bone marrow. Hypomethylation may restore normal function to genes that are critical for cell differentiation and proliferation.9,10 Onureg is approved in the U.S. for continued treatment of adult patients with acute myeloid leukemia who achieved first complete remission (CR) or complete remission with incomplete blood count recovery (CRi) following intensive induction chemotherapy and are not able to complete intensive curative therapy. Onureg is also approved in Canada as maintenance therapy for adult patients with acute myeloid leukemia (AML) who achieved complete remission (CR) or complete remission with incomplete blood count recovery (CRi) following induction therapy with or without consolidation treatment, and who are not eligible for hematopoietic stem cell transplantation (HSCT).
U.S. IMPORTANT SAFETY INFORMATION
CONTRAINDICATIONS
- ONUREG is contraindicated in patients with known severe hypersensitivity to azacitidine or its components.
Please see full Prescribing Information and Summary of Product Characteristics for ONUREG.
For more information about Bristol Myers Squibb, visit us at BMS.com
Bristol Myers Squibb's Onureg gets EC approval to treat certain leukemia patients
Jun. 18, 2021 9:47 AM ETBristol-Myers Squibb Company (BMY)By: Aakash Babu, SA News Editor4 Comments
- Bristol Myers Squibb (BMY -1.2%) announces that the European Commission (EC) has granted full Marketing Authorization for Onureg (azacitidine tablets) as a maintenance therapy in certain adult patients with acute myeloid leukemia (AML).
- https://seekingalpha.com/news/3707683-bristol-myers-squibbs-onureg-gets-ec-approval-to-treat-certain-leukemia-patients
- https://seekingalpha.com/symbol/BMY
KITE AND SHORELINE BIOSCIENCES ENTER INTO STRATEGIC PARTNERSHIP TO DEVELOP NOVEL ALLOGENEIC CELL THERAPIES
- Posted on June 17, 2021
- In Featured, Press Releases
— Partnership Follows Kite’s Investment in Shoreline’s Recent Series A Financing —
Santa Monica, Calif., and San Diego, June 17, 2021 – Kite, a Gilead Company (Nasdaq: GILD), and Shoreline Biosciences, Inc. (Shoreline), a biotechnology company developing intelligently designed allogeneic off-the-shelf, standardized and targeted natural killer (NK) and Macrophage cellular immunotherapies derived from induced pluripotent stem cells (iPSC) for cancer and other serious diseases, today announced a strategic partnership to develop novel cell therapies across a variety of cancer targets.
The collaboration will leverage Shoreline’s deep expertise in iPSC differentiation and genetic reprogramming in combination with Kite’s extensive cell therapy development, commercialization and manufacturing expertise to develop novel allogeneic candidates for a range of hematologic malignancies. The collaboration will focus initially on chimeric antigen receptor (CAR) NK targets, with Kite having an option to expand the collaboration to include an iPSC CAR Macrophage program for an undisclosed target to be selected post deal execution. This agreement follows Kite’s investment in Shoreline’s recent Series A financing.
For more information, please visit https://shorelinebio.com/
For more information on Kite, please visit www.kitepharma.com.
Kite, the Kite logo and GILEAD are trademarks of Gilead Sciences, Inc. or its related companies.
For more information on Kite, please visit the company’s website at www.kitepharma.com or call Gilead Public Affairs at 1-800-GILEAD-5 or 1-650-574-3000.
Gilead's Kite taps Shoreline Biosciences in strategic partnership for as much as $2.3B
Jun. 17, 2021 9:10 AM ET
By: Aakash Babu, SA News Editor
- Gilead (NASDAQ:GILD) company Kite and Shoreline Biosciences announce a strategic partnership to develop novel cell therapies across a variety of cancer targets.
- https://seekingalpha.com/news/3707273-gileads-kite-taps-shoreline-biosciences-in-strategic-partnership-for-as-much-as-23b
- https://seekingalpha.com/symbol/GILD
- https://www.kitepharma.com/
Forging New Breakthroughs in Lifesaving Immunotherapies
https://shorelinebio.com/our-science/#NK-Cells
REGEN-COV™ (CASIRIVIMAB AND IMDEVIMAB)
REGEN-COV™ (CASIRIVIMAB AND IMDEVIMAB) PHASE 3 RECOVERY TRIAL MEETS PRIMARY OUTCOME, IMPROVING SURVIVAL IN HOSPITALIZED COVID-19 PATIENTS LACKING AN IMMUNE RESPONSE TO SARS-COV-2
TARRYTOWN, N.Y., June 16, 2021 /PRNewswire/ --
UK RECOVERY investigators found REGEN-COV reduced risk of death by 20% in patients hospitalized with COVID-19 who had not mounted their own immune response (primary outcome for the primary analysis population)
First trial to demonstrate that any antibody treatment improved survival in patients hospitalized with COVID-19
Regeneron will share new data with regulatory authorities immediately and request that the U.S. EUA be expanded to include appropriate hospitalized patients
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today welcomed positive results from the largest trial assessing any monoclonal antibody treatment in patients hospitalized with severe COVID-19. The UK RECOVERY trial found that adding investigational REGEN-COV™ to usual care reduced the risk of death by 20% in patients who had not mounted a natural antibody response on their own against SARS-CoV-2, compared to usual care on its own.
"These results are very exciting. The hope was that by giving a combination of antibodies targeting the SARS-CoV-2 virus we would be able to reduce the worst manifestations of COVID-19," said Sir Peter Horby, Professor of Emerging Infectious Diseases in the Nuffield Department of Medicine, University of Oxford, and Joint Chief Investigator for the RECOVERY trial. "There was, however, great uncertainty about the value of antiviral therapies in late-stage COVID-19 disease. It is wonderful to learn that even in advanced COVID-19 disease, targeting the virus can reduce mortality in patients who have failed to mount an antibody response of their own."
RECOVERY is the first trial large enough to definitively determine whether REGEN-COV reduces mortality in patients hospitalized with severe COVID-19. Previous Phase 3 trials in non-hospitalized COVID-19 patients have shown that REGEN-COV reduced viral levels, shortened the time to resolution of symptoms and significantly reduced the risk of hospitalization or death. In a Phase 1/2 trial in hospitalized patients, REGEN-COV also rapidly reduced viral levels, with preliminary evidence suggesting that it lowered the risk of death or receiving mechanical ventilation, with the benefit driven by patients who entered the trial without having mounted a natural antibody response of their own (seronegative); and in the absence of REGEN-COV treatment, seronegative patients had higher mortality rates than patients who had already mounted their own immune response (seropositive).
Based on the above Phase 1/2 data, the independently-run RECOVERY trial prospectively focused on seronegative patients. Similar to the prior trial, patients in RECOVERY who received usual care alone had double the mortality rate at day 28 if they were seronegative (30%) compared to seropositive (15%); approximately one-third of hospitalized patients were seronegative (n=3,153), one-half were seropositive (n=5,272) and one-sixth had unknown serostatus (n=1,360). The mean age of patients for this comparison was 62 years, and more than 90% received corticosteroids across all groups.
The primary outcome of RECOVERY showed that adding REGEN-COV 8,000 mg to usual care reduced all-cause mortality by 20% in seronegative patients (primary analysis population), compared to usual care alone (24% of patients in the REGEN-COV group died versus 30% in the usual care group by day 28; rate ratio [RR]: 0.80; 95% confidence interval [CI]: 0.70-0.91; p=0.001). When combining the larger seropositive group (as well as those with unknown status) with the seronegative patients, there was no longer a significant effect on 28-day mortality (overall 20% of patients in the REGEN-COV group died, versus 21% in the usual care group; RR: 0.96; 95% CI: 0.86-1.03; p=0.17).
"Definitive Phase 3 trials have now demonstrated that REGEN-COV can alter the course of COVID-19 infection from prevention, to very early infection, all the way through to when patients are on a ventilator in the hospital," said George D. Yancopoulos, M.D., Ph.D., President and Chief Scientific Officer at Regeneron. "We are incredibly grateful to the RECOVERY team, participating investigators and patients for conducting this in-depth analysis, and hope that the results mean that even more patients may soon be able to benefit from this life-saving medicine. We intend to rapidly discuss these results with regulatory authorities, including in the U.S. where we will ask for our EUA to be expanded to include appropriate hospitalized patients."
Among seronegative patients in the RECOVERY trial, the median duration of hospital stay was 4 days shorter in the REGEN-COV group (13 days versus 17 days), and the proportion of patients discharged alive by day 28 was greater (64% versus 58%; RR: 1.19; 95% CI: 1.08-1.30). Among the seronegative patients not on invasive mechanical ventilation at baseline, the risk of progressing to the composite endpoint of invasive mechanical ventilation or death was lower among the REGEN-COV group than the usual care group (30% versus 37%; RR: 0.83; 95% CI: 0.75-0.92). No such benefits were seen in the overall trial population (combining patients with negative, positive, or unknown serostatus).
"The RECOVERY trial has shown that in patients who had not made their own antibodies against SARS-CoV-2, treating them with REGEN-COV antibodies dramatically reduced their risk of dying or being on a ventilator, and also shortened how many days they remained in the hospital," said David Weinreich, M.D., Executive Vice President, Global Clinical Development at Regeneron. "The trial was conducted at a time when most patients had not been fully vaccinated. These results provide hope to patients who have a poor immune response to either the vaccine or natural infection, as well as those who are exposed to variants for whom their existing antibodies might be sub-optimal."
Regeneron is collaborating with Roche to increase global supply of REGEN-COV. Regeneron is responsible for development and distribution of the treatment in the U.S., and Roche is primarily responsible for development and distribution outside the U.S. The companies share a commitment to making the antibody cocktail available to COVID-19 patients around the globe and will support access in low- and lower-middle-income countries through drug donations to be made in partnership with public health organizations.
The development and manufacturing of REGEN-COV have been funded in part with federal funds from the Biomedical Advanced Research and Development Authority (BARDA), part of the U.S. Department of Health and Human Services' Office of the Assistant Secretary for Preparedness and Response, under OT number: HHSO100201700020C.
About the Trial
The RECOVERY trial (Randomised Evaluation of COVid-19 thERapY) was conducted by the registered clinical trials units in the Nuffield Department of Population Health in partnership with the Nuffield Department of Medicine. The trial was supported by a grant to the University of Oxford from UK Research and Innovation/National Institute for Health Research (NIHR) and by core funding provided by NIHR Oxford Biomedical Research Centre, Wellcome, the Bill and Melinda Gates Foundation, the Department for International Development, Health Data Research UK, the Medical Research Council Population Health Research Unit and NIHR Clinical Trials Unit Support Funding.
Between September 18, 2020 and May 22, 2021, 9,785 patients hospitalized with COVID-19 were randomly allocated to receive usual care plus REGEN-COV (8,000 mg by intravenous infusion) or usual care alone as part of the RECOVERY trial. Usual care was determined by individual facilities and clinicians, and could include steroids and remdesivir. Follow-up is complete for 99% of participants.
The trial involved many thousands of doctors, nurses, pharmacists, and research administrators at 176 hospitals across the whole of the UK, supported by staff at the NIHR Clinical Research Network, NHS DigiTrials, Public Health England, Department of Health & Social Care, the Intensive Care National Audit & Research Centre, Public Health Scotland, the Secure Anonymised Information Linkage at University of Swansea, and the NHS in England, Scotland, Wales and Northern Ireland.
About the REGEN-COV Antibody Cocktail
REGEN-COV (casirivimab and imdevimab) is a cocktail of two monoclonal antibodies (also known as REGN10933 and REGN10987) that was designed specifically to block infectivity of SARS-CoV-2, the virus that causes COVID-19, using Regeneron's proprietary VelocImmune® and VelociSuite® technologies. The two potent, virus-neutralizing antibodies that form the cocktail bind non-competitively to the critical receptor binding domain of the virus's spike protein, which diminishes the ability of mutant viruses to escape treatment and protects against spike variants that have arisen in the human population, as detailed in Science.
Under an EUA, REGEN-COV is available throughout the U.S. – information on availability in your area is available from the Department of Health and Human Services and the National Infusion Center Association. REGEN-COV can be administered by intravenous infusion (as short as 20 minutes) or by subcutaneous injection (four injections), which is an alternative when intravenous infusion is not feasible and would lead to a delay in treatment. It is now authorized as a co-formulated single vial, or in individual vials to be administered together.
In the U.S., REGEN-COV is not authorized for use in patients who are hospitalized due to COVID-19 or require oxygen therapy, or for people currently using chronic oxygen therapy because of an underlying comorbidity who require an increase in baseline oxygen flow rate due to COVID-19.
AUTHORIZED USE AND IMPORTANT SAFETY INFORMATION
REGEN-COV, (casirivimab and imdevimab) co-formulated product and REGEN-COV (casirivimab and imdevimab) supplied as individual vials to be administered together, is authorized for the treatment of mild to moderate coronavirus disease 2019 (COVID-19) in adults and pediatric patients (12 years of age and older weighing at least 40 kg) with positive results of direct SARS-CoV-2 viral testing, and who are at high risk for progression to severe COVID-19, including hospitalization or death. [see Limitations of Authorized Use]
- REGEN-COV has not been approved, but has been authorized for emergency use by FDA
- This use is authorized only for the duration of the declaration that circumstances exist justifying the authorization of the emergency use under section 564(b)(1) of the Act, 21 U.S.C. § 360bbb-3(b)(1), unless the authorization is terminated or revoked sooner
- Healthcare providers should review the Fact Sheet for Healthcare Providers for information on the authorized use of REGEN-COV and mandatory requirements of the EUA and must comply with the requirements of the EUA. The FDA Letter of Authorization is available for reference, as well as the Dear Healthcare Provider Letter and Patient Fact Sheet
For additional information about the company, please visit www.regeneron.com
Trial results for a new Regeneron treatment have buoyed hopes that doctors could soon have a new way to tackle severe hospital cases.
Regeneron's REGEN-COV reduces death by 20% in hospitalized COVID-19 patients
Jun. 16, 2021 1:33 AM ET Regeneron Pharmaceuticals, Inc. (REGN) By: Mamta Mayani, SA News Editor3 Comments
Regeneron Pharmaceuticals (NASDAQ:REGN) announces positive results the from the Phase 3 RECOVERY trial assessing REGEN-COV (casirivimab and imdevimab) in patients hospitalized with severe COVID-19.
- https://seekingalpha.com/news/3706734-regenerons-regen-cov-reduces-death-by-20-in-hospitalized-covid-19-patients
- https://seekingalpha.com/symbol/REGN
SOURCE Regeneron Pharmaceuticals, Inc.
NVX-CoV2373
Novavax COVID-19 Vaccine Demonstrates 90% Overall Efficacy and 100% Protection Against Moderate and Severe Disease in PREVENT-19 Phase 3 Trial
Jun 14, 2021 at 6:00 AM EDTDownload PDF
- 93% efficacy against predominantly circulating Variants of Concern and Variants of Interest
- 91% efficacy in high-risk populations
- 100% efficacy against variants "not considered Variants of Concern/Interest"
- All COVID-19 hospitalizations/death occurred in the placebo group
- Company to host investor conference call today at 8:30 am ET
GAITHERSBURG, Md., June 14, 2021 /PRNewswire/ -- Novavax, Inc. (Nasdaq: NVAX), today announced that NVX-CoV2373, its recombinant nanoparticle protein-based COVID-19 vaccine, demonstrated 100% protection against moderate and severe disease, 90.4% efficacy overall, and met the primary endpoint in its PREVENT-19 pivotal Phase 3 trial. The study enrolled 29,960 participants across 119 sites in the U.S. and Mexico to evaluate efficacy, safety and immunogenicity, with an emphasis on recruiting a representative population of communities and demographic groups most impacted by the disease.
"Today, Novavax is one step closer to addressing the critical and persistent global public health need for additional COVID-19 vaccines. These clinical results reinforce that NVX-CoV2373 is extremely effective and offers complete protection against both moderate and severe COVID-19 infection," said Stanley C. Erck, President and Chief Executive Officer, Novavax. "Novavax continues to work with a sense of urgency to complete our regulatory submissions and deliver this vaccine, built on a well understood and proven platform, to a world that is still in great need of vaccines."
"PREVENT-19 confirms that NVX-CoV2373 offers a reassuring tolerability and safety profile," said Gregory M. Glenn, M.D., President of Research and Development, Novavax. "These data show consistent, high levels of efficacy and reaffirm the ability of the vaccine to prevent COVID-19 amid ongoing genetic evolution of the virus. Our vaccine will be a critical part of the solution to COVID-19 and we are grateful to the study participants and trial staff who made this study possible, as well as our supporters, including the U.S. Government."
Click here to view multimedia content, including B-roll, an illustrated fact sheet and other resources that accompany this press release.
Results: Consistent, high efficacy among circulating variants
In the placebo-controlled, observer-blinded study randomized 2:1, NVX-CoV2373 demonstrated overall efficacy of 90.4% (95% CI: 82.9, 94.6), achieving its primary endpoint. Seventy-seven cases were observed: 63 in the placebo group and 14 in the vaccine group. All cases observed in the vaccine group were mild as defined by the trial protocol. Ten moderate cases and four severe cases were observed, all in the placebo group, yielding a vaccine efficacy of 100% (95% CI: 87.0, 100) against moderate or severe disease.
Efficacy endpoints were accrued from January 25 through April 30, 2021 — a time when the Alpha (B.1.1.7) variant, first identified in the U.K., became the predominant strain in the U.S. Other strains, including Variants of Interest (VoI) and Variants of Concern (VoC), were also on the rise during the PREVENT-19 endpoint accrual window. Click here for CDC definitions of variants.
Sequence data are available for 54 of the 77 cases. PREVENT-19 met its key secondary endpoint, demonstrating 100% efficacy (95% CI: 80.8, 100) against variants not considered VoC/VoI. Of the sequenced cases, 35 (65%) were VoC, 9 (17%) were VoI, and 10 (19%) were other variants. Against VoC/VoI, which represented 82% of the cases, vaccine efficacy was 93.2% (95% CI: 83.9, 97.1), achieving a key exploratory endpoint of the study. Thirty-eight of the VoC/VoI cases were in the placebo group and 6 were in the vaccine group.
NVX-CoV2373 also showed success among "high-risk" populations (defined as over age 65, under age 65 with certain comorbidities or having life circumstances with frequent COVID-19 exposure): vaccine efficacy was 91.0% (95% CI: 83.6, 95.0), with 62 COVID-19 cases in the placebo group and 13 COVID-19 cases in the vaccine group.
About PREVENT-19
PREVENT-19 (the PRE-fusion protein subunit Vaccine Efficacy Novavax Trial | COVID-19) is a 2:1 randomized, placebo-controlled, observer-blinded study to evaluate the efficacy, safety and immunogenicity of NVX-CoV2373 with Matrix-M™ adjuvant in 29,960 participants 18 years of age and older in 119 locations in the United States and Mexico, compared with placebo.
PREVENT-19 is being conducted with support from the U.S. government, including the Department of Defense, the Biomedical Advanced Research and Development Authority (BARDA), part of the Office of the Assistant Secretary for Preparedness and Response at the U.S. Department of Health and Human Services (HHS), and the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH) at HHS. BARDA is providing up to $1.75 billion under a Department of Defense agreement.
About NVX-CoV2373
NVX-CoV2373 is a protein-based vaccine candidate engineered from the genetic sequence of the first strain of SARS-CoV-2, the virus that causes COVID-19 disease. NVX-CoV2373 was created using Novavax' recombinant nanoparticle technology to generate antigen derived from the coronavirus spike (S) protein and is formulated with Novavax' patented saponin-based Matrix-M™ adjuvant to enhance the immune response and stimulate high levels of neutralizing antibodies. NVX-CoV2373 contains purified protein antigen and can neither replicate, nor can it cause COVID-19. In preclinical studies, NVX-CoV2373 induced antibodies that blocked the binding of spike protein to cellular receptors and provided protection from infection and disease. It was generally well-tolerated and elicited robust antibody response in Phase 1/2 clinical testing.
NVX-CoV2373 is being evaluated in two pivotal Phase 3 trials: a trial in the U.K. that demonstrated efficacy of 96.4% against the original virus strain, 86.3% against the B.1.1.7 (Alpha) variant and 89.7% overall; and the PREVENT-19 trial in the U.S. and Mexico that began in December 2020. It is also being tested in two ongoing Phase 2 studies that began in August 2020: A Phase 2b trial in South Africa that demonstrated 55% efficacy overall in HIV-negative participants and 48.6% efficacy against the B.1.351 (Beta) variant, and a Phase 1/2 study in the U.S. and Australia.
NVX-CoV2373 is stored and stable at 2°- 8°C, allowing the use of existing vaccine supply chain channels for its distribution. It is packaged in a ready-to-use liquid formulation in 10-dose vials.
For more information, visit www.novavax.com
View original content to download multimedia:http://www.prnewswire.com/news-releases/novavax-covid-19-vaccine-demonstrates-90-overall-efficacy-and-100-protection-against-moderate-and-severe-disease-in-prevent-19-phase-3-trial-301311413.html
SOURCE Novavax, Inc.
Novavax stock up 9% as COVID-19 vaccine shows 90% overall efficacy
Jun. 14, 2021 7:01 AM ET Novavax, Inc. (NVAX) By: Mamta Mayani, SA News Editor6 Comments
- Novavax (NASDAQ:NVAX) soars 9% premarket in reaction to the announcement that NVX-CoV2373, its recombinant protein-based COVID-19 vaccine met the primary endpoint in PREVENT-19 Phase 3 trial.
- https://seekingalpha.com/news/3705886-novavax-soars-9-as-covid-19-vaccine-shows-90-overall-efficacy-plans-regulatory-filing-in-q3
- https://seekingalpha.com/symbol/NVAX
XELJANZ® (tofacitinib)
DATA PUBLISHED IN NEW ENGLAND JOURNAL OF MEDICINE SHOWS PFIZER’S TOFACITINIB MEETS PRIMARY ENDPOINT IN BRAZILIAN STUDY IN PATIENTS HOSPITALIZED WITH COVID-19 PNEUMONIA
Wednesday, June 16, 2021 - 05:19pm
- Trial demonstrates cumulative incidence of death or respiratory failure through day 28 was 18.1% (26 of 144) with tofacitinib compared to 29.0% (42 of 145) with placebo, in hospitalized patients with COVID-19 pneumonia
- Multi-center, randomized, double-blind, placebo-controlled trial conducted across 15 sites in Brazil
- Tofacitinib is not approved or authorized for the treatment of COVID-19 patients. Tofacitinib should not be used in patients with an active serious infection
NEW YORK--(BUSINESS WIRE)-- Pfizer Inc. (NYSE: PFE) and The Academic Research Organization (ARO) from the Hospital Israelita Albert Einstein today announced that the New England Journal of Medicine has published positive findings from the STOP-COVID study (NCT04469114) evaluating the efficacy and safety of oral Janus kinase (JAK) inhibitor tofacitinib in 289 hospitalized adult patients with COVID-19 pneumonia who were not on ventilation.
The trial was a research collaboration between Pfizer and the ARO from the Hospital Israelita Albert Einstein in Sao Paulo, Brazil, which was the trial coordinating center. It is important to note that tofacitinib has not been approved or authorized for use by any regulatory authority worldwide for the treatment of COVID-19 and tofacitinib should not be used in patients with an active serious infection.
The trial demonstrated a lower cumulative incidence of death or respiratory failure through day 28 – the primary outcome of the study – with tofacitinib (18.1%) compared to placebo (29.0%) (risk ratio 0.63; 95% confidence interval [CI], 0.41 to 0.97; p=0.04). Death from any cause through day 28 occurred in 2.8% of patients in the tofacitinib group and in 5.5% in the placebo group (hazard ratio 0.49; 95% CI, 0.15 to 1.63).
Serious adverse events occurred in 20 patients (14.1%) in the tofacitinib group and 17 (12.0%) in the placebo group. Among protocol-specified adverse events of special interest, deep vein thrombosis, acute myocardial infarction, ventricular tachycardia, and myocarditis occurred in one patient each in the tofacitinib group; hemorrhagic stroke and cardiogenic shock occurred in one patient each in the placebo group. The incidence of serious infection was 3.5% in the tofacitinib group and 4.2% in the placebo group.
For additional information about the study (NCT04469114), please visit https://www.clinicaltrials.gov.
About XELJANZ® (tofacitinib)
XELJANZ® (tofacitinib) isapproved in the U.S. in four indications: adults with moderately to severely active rheumatoid arthritis (RA) after methotrexate failure, adults with active psoriatic arthritis (PsA) after disease modifying antirheumatic drug (DMARD) failure, adults with moderately to severely active ulcerative colitis (UC) after tumor necrosis factor inhibitor (TNFi) failure and patients 2 years of age or older with active polyarticular course juvenile idiopathic arthritis (pcJIA). See Limitations of Use below.
XELJANZ has been studied in more than 50 clinical trials worldwide, including more than 20 trials in RA patients, and prescribed to over 300,000 adult patients (the majority of whom were RA patients) worldwide since 2012.i,ii,iii As the developer of tofacitinib, Pfizer is committed to advancing the science of JAK inhibition and enhancing understanding of tofacitinib through robust clinical development programs in the treatment of immune-mediated inflammatory conditions.
Pfizer recently communicated an increased rate of major adverse cardiovascular events (MACE) and malignancies (excluding non-melanoma skin cancer (NMSC)) for XELJANZ relative to anti-TNF therapy in RA patients who were 50 years of age or older and had at least one additional cardiovascular (CV) risk factor. Pfizer is continuing to work with the U.S. Food and Drug Administration (FDA), European Medicines Agency (EMA), and other regulatory agencies to review the full results and analysis.
INDICATIONS
Rheumatoid Arthritis
- XELJANZ/XELJANZ XR (tofacitinib) is indicated for the treatment of adult patients with moderately to severely active rheumatoid arthritis who have had an inadequate response or intolerance to methotrexate.
- Limitations of Use: Use of XELJANZ/XELJANZ XR in combination with biologic DMARDs or with potent immunosuppressants such as azathioprine and cyclosporine is not recommended.
Psoriatic Arthritis
- XELJANZ/XELJANZ XR (tofacitinib) is indicated for the treatment of adult patients with active psoriatic arthritis who have had an inadequate response or intolerance to methotrexate or other disease-modifying antirheumatic drugs (DMARDs).
- Limitations of Use: Use of XELJANZ/XELJANZ XR in combination with biologic DMARDs or with potent immunosuppressants such as azathioprine and cyclosporine is not recommended.
Ulcerative Colitis
- XELJANZ is indicated for the treatment of adult patients with moderately to severely active ulcerative colitis (UC), who have had an inadequate response or who are intolerant to TNF blockers.
- Limitations of Use: Use of XELJANZ in combination with biological therapies for UC or with potent immunosuppressants such as azathioprine and cyclosporine is not recommended.
Please see full Prescribing Information, including BOXED WARNING for XELJANZ/XELJANZ XR available at: www.xeljanzpi.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210616005998/en/
to learn more, please visit us on www.pfizer.com
Pfizer’s tofacitinib meets primary endpoint in Brazilian COVID-19 study
Jun. 17, 2021 3:00 AM ETPfizer Inc. (PFE)By: Mamta Mayani, SA News Editor
- Pfizer (NYSE:PFE) announces that the New England Journal of Medicine (NEJM) has published positive findings from the STOP-COVID study evaluating the efficacy and safety of oral JAK inhibitor Xeljanz (tofacitinib) in 289 hospitalized adult patients with COVID-19 pneumonia.
- https://seekingalpha.com/news/3707144-pfizers-tofacitinib-meets-primary-endpoint-in-brazilian-covid-19-study
- https://seekingalpha.com/symbol/PFE
- https://www.pfizer.com/
- https://www.xeljanz.com/
Verzenio® (abemaciclib)
Lilly announces new clinical data from Verzenio and oral SERD programs at the American Society of Clinical Oncology annual meeting
Jun. 14, 2021 4:15 PM ETCanada NewswireEli Lilly and Company (LLY)
- New exploratory analysis of a pre-specified subgroup of patients with HR+, HER2- high risk early breast cancer who received neoadjuvant chemotherapy in the monarchE trial showed Verzenio plus endocrine therapy resulted in a 6.6% absolute difference in invasive disease-free survival versus endocrine therapy alone
- First results from investigational oral selective estrogen receptor degrader (SERD) LY3484356 demonstrate pharmacokinetics, safety, and efficacy consistent with preclinical design
- Two Phase 3 trials to be initiated in 2021: Verzenio eMonarcHER trial in HR+, HER2+ high risk early breast cancer and oral SERD EMBER-3 trial in ER+, HER2- advanced breast cancer
TORONTO, June 14, 2021 /CNW/ - Eli Lilly and Company
Phase 3 EMBER-3 Trial of LY3484356
Lilly is preparing to initiate a randomized, open-label, Phase 3 study of LY3484356 in patients with ER+, HER2- locally advanced or metastatic breast cancer previously treated with endocrine therapy. Patients will be randomized to receive LY3484356 monotherapy or investigator's choice of monotherapy endocrine therapy (fulvestrant or exemestane). The trial, EMBER-3, is expected to begin enrollment in the third quarter of 2021.
Please refer to Lilly's press release from May 19, 2021 for a full list of presentations at the meeting.
About the monarchE Trial
monarchE is a Phase 3, multicenter, randomized, open-label trial that enrolled 5,637 patients with HR+, HER2-, node-positive, high risk early breast cancer. Patients were randomized 1:1 to Verzenio (150 mg twice daily) plus standard adjuvant ET or standard adjuvant ET alone. Patients were treated for two years (treatment period) or until meeting criteria for discontinuation. Patients in both arms will receive 5-10 years of ET as clinically indicated (2 years on study followed by a further 3-8 years in long-term follow-up). The primary objective is invasive disease-free survival (IDFS) defined according to the Standard Definitions for Efficacy Endpoints (STEEP) criteria. In adjuvant breast cancer trials, this includes the length of time before any cancer comes back, a new cancer develops or death. Secondary objectives include distant relapse-free survival, overall survival, safety, pharmacokinetics and health outcomes.
High risk was specifically defined as women (any menopausal status) and men with resected HR+, HER2- invasive early breast cancer with either ≥4 pathologically positive axillary lymph nodes (ALNs) or 1 to 3 positive ALNs and at least one of the following high-risk features: primary invasive tumor size ≥5 cm, histological grade 3 tumor, or central Ki-67 index ≥20%. If applicable, patients must have also completed adjuvant chemotherapy and radiotherapy prior to enrolling and have recovered from all acute side effects.
About Verzenio®
Verzenio (abemaciclib) is an inhibitor of cyclin-dependent kinases (CDK)4 & 6, which are activated by binding to D-cyclins. In estrogen receptor-positive (ER+) breast cancer cell lines, cyclin D1 and CDK4 & 6 promote phosphorylation of the retinoblastoma protein (Rb), cell cycle progression, and cell proliferation.
In vitro, continuous exposure to Verzenio inhibited Rb phosphorylation and blocked progression from G1 to S phase of the cell cycle, resulting in senescence and apoptosis (cell death). Preclinically, Verzenio dosed daily without interruption resulted in reduction of tumor size. Inhibiting CDK4 & 6 in healthy cells can result in side effects, some of which may be serious. Clinical evidence also suggests that Verzenio crosses the blood-brain barrier. In patients with advanced cancer, including breast cancer, concentrations of Verzenio and its active metabolites (M2 and M20) in cerebrospinal fluid are comparable to unbound plasma concentrations.
Verzenio is Lilly's first solid oral dosage form to be made using a faster, more efficient process known as continuous manufacturing. Continuous manufacturing is a new and advanced type of manufacturing within the pharmaceutical industry, and Lilly is one of the first companies to use this technology.
Indication
In Canada, Verzenio® (abemaciclib) is indicated for the treatment of hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced or metastatic breast cancer:
- in combination with an aromatase inhibitor in postmenopausal women as initial endocrine based therapy.
- in combination with fulvestrant in women with disease progression following endocrine therapy. Pre- or perimenopausal women must also be treated with a gonadotropin-releasing hormone (GnRH) agonist.
- as a single agent in women with disease progression following endocrine therapy and at least 2 prior chemotherapy regimens. At least one chemotherapy regimen should have been administered in the metastatic setting, and at least one should have contained a taxane.
https://seekingalpha.com/symbol/LLY
APL-1202 in combination with tislelizumab
The U.S. FDA Approved IND Application to Investigate Combination of Asieris' APL-1202 and BeiGene's Tislelizumab as Neoadjuvant Therapy for MIBC Patients
NEWS PROVIDED BY
Jun 14, 2021, 20:46 ET
SHANGHAI, June 14, 2021 /PRNewswire/ -- Asieris Pharmaceuticals (Asieris) today announced that the U.S. Food and Drug Administration (FDA) has approved the Investigational New Drug (IND) application of oral APL-1202 in combination with BeiGene's tislelizumab as neoadjuvant therapy in patients with muscle invasive bladder cancer (MIBC). Asieris will accelerate the initiation of the clinical trial enrollment in the U.S. and also file a Clinical Trial Application (CTA) to the National Medical Products Administration (NMPA) of China in the near future.
This is an open-label, multi-center Phase I/II clinical study with the following objectives: to evaluate the safety in MIBC patients; to determine the RP2D (recommended Phase 2 dose), and to assess efficacy as neoadjuvant therapy for MIBC.
APL-1202 is an orally available reversible MetAP2 Inhibitor with anti-angiogenic, anti-tumor activities and can also modulate tumor immune microenvironment. It is currently in Phase III/pivotal clinical trials in China, either as single agent as first-line treatment for patients with intermediate-risk non-muscle invasive bladder cancer (NMIBC), or in combination with a chemotherapy as second-line treatment in patients with intermediate and high-risk chemo-refractory NMIBC. Tislelizumab is a humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to FcγR on macrophages. The China National Medical Products Administration (NMPA) has granted tislelizumab approval in three indications, including full approval for first-line treatment of patients with advanced squamous non-small cell lung cancer (NSCLC) in combination with chemotherapy; and conditional approval for the treatment of patients with classical Hodgkin's lymphoma (cHL) who received at least two prior therapies and for the treatment of patients with locally advanced or metastatic urothelial carcinoma (UC) with PD-L1 high expression whose disease progressed during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy. Full approval for these indications is contingent upon results from ongoing randomized, controlled confirmatory clinical trials.
Product Pipeline
https://asieris.com/pipeline/product-pipeline/
FDA approves BeiGene's application for tislelizumab combo in bladder cancer
Jun. 15, 2021 2:44 AM ET
By: Mamta Mayani, SA News Editor1 Comment
- Asieris Pharmaceuticals (Asieris) announces that the FDA has approved the IND application of oral APL-1202 in combination with BeiGene's (NASDAQ:BGNE) tislelizumab as neoadjuvant therapy in patients with muscle invasive bladder cancer (MIBC).
- https://seekingalpha.com/news/3706263-fda-approves-beigenes-application-for-tislelizumab-combo-in-bladder-cancer
- https://seekingalpha.com/symbol/BGNE
- https://asieris.com/
DARZALEX® (daratumumab)
Janssen Announces Results from Phase 3 MAIA Study Showing Significant Overall Survival Benefits for Treatment with DARZALEX® (daratumumab) in Patients with Newly Diagnosed Multiple Myeloma Who are Transplant Ineligible
Jun 12, 2021United States
After nearly five years of follow-up, median progression-free survival was not reached, and a significant overall survival benefit was observed; data will be presented as a late-breaking abstract at the European Hematology Association (EHA) Virtual Congress
RARITAN, N.J., June 12, 2021 -- The Janssen Pharmaceutical Companies of Johnson & Johnson announced overall survival (OS) results from the Phase 3 MAIA (NCT02252172) study showing the addition of DARZALEX® (daratumumab) to lenalidomide and dexamethasone (D-Rd) resulted in a statistically significant survival benefit over lenalidomide and dexamethasone (Rd) alone in patients with newly diagnosed multiple myeloma (NDMM) who were ineligible for autologous stem cell transplant (ASCT) and were treated to progression.1 These data were featured in the European Hematology Association (EHA) 2021 Virtual Press Briefing and will be presented as a late-breaking abstract during the EHA Virtual Congress (Abstract #LB1901).
The prespecified interim analysis for OS found that after a median follow-up of nearly five years (56.2 months), a 32 percent reduction in the risk of death was observed in the D-Rd treatment arm vs. Rd arm.1 Median OS was not reached in either arm [hazard ratio (HR): 0.68, 95 percent confidence interval (CI), 0.53-0.86; p=0.0013].1 Median progression-free survival (PFS) was not reached after nearly five years and the PFS benefit observed with D-Rd was maintained, with a 47 percent reduction in risk of disease progression or death [HR: 0.53; 95 percent CI, 0.43-0.66; p<0.0001].1 These data are expected to form the basis of future regulatory submissions.
About the MAIA Trial
The randomized, open-label, multicenter Phase 3 study included 737 newly diagnosed patients with multiple myeloma ineligible for high-dose chemotherapy and ASCT, aged 45-90 years (median age of 73).1 Patients were randomized to receive either DARZALEX®-Rd (D-Rd) or Rd alone in 28-day cycles. In the D-Rd arm, patients received DARZALEX® 16 milligrams per kilogram (mg/kg) IV weekly for cycles 1 – 2, every two weeks for cycles 3 – 6 and every 4 weeks for cycle 7 and thereafter.1 Patients in the D-Rd and Rd treatment arms received 25 mg of lenalidomide on days 1 – 21 of each 28-day cycle, and dexamethasone at 40 mg once a week for each cycle. Patients in both treatment arms continued until disease progression or unacceptable toxicity.1
Earlier results from the MAIA study supported the U.S. Food and Drug Administration (FDA) approval of DARZALEX® in combination with Rd, marking the first approval of a CD-38 monoclonal antibody for patients with transplant ineligible NDMM. These data were also published in The New England Journal of Medicine in 2019.
About DARZALEX®
Janssen is committed to exploring the potential of DARZALEX® (daratumumab) for patients with multiple myeloma across the spectrum of the disease. DARZALEX® has been approved in eight indications, three of which are in the frontline setting, including newly diagnosed patients who are transplant eligible and ineligible.2
DARZALEX® has become a backbone therapy in the treatment of multiple myeloma, having been used in the treatment of more than 190,000 patients worldwide and more than 68,000 patients in the U.S. alone since its U.S. FDA approval in 2015.3 DARZALEX® is the first CD38-directed antibody approved globally to treat multiple myeloma.2
CD38 is a surface protein that is present in high numbers on multiple myeloma cells, regardless of the stage of disease.4 DARZALEX® binds to CD38 and inhibits tumor cell growth causing myeloma cell death.4 DARZALEX® may also have an effect on normal cells.4 Data across eight Phase 3 clinical trials, in both the frontline and relapsed settings, have shown that DARZALEX®-based regimens resulted in significant improvement in progression-free survival and/or overall survival.5,6,7,8,9,10,11,12
DARZALEX® INDICATIONS
DARZALEX® (daratumumab) is indicated for the treatment of adult patients with multiple myeloma:
In combination with lenalidomide and dexamethasone in newly diagnosed patients who are ineligible for autologous stem cell transplant and in patients with relapsed or refractory multiple myeloma who have received at least one prior therapy
In combination with bortezomib, melphalan, and prednisone in newly diagnosed patients who are ineligible for autologous stem cell transplant
In combination with bortezomib, thalidomide, and dexamethasone in newly diagnosed patients who are eligible for autologous stem cell transplant
In combination with bortezomib and dexamethasone in patients who have received at least one prior therapy
In combination with carfilzomib and dexamethasone in patients with relapsed or refractory multiple myeloma who have received one to three prior lines of therapy
In combination with pomalidomide and dexamethasone in patients who have received at least two prior therapies including lenalidomide and a proteasome inhibitor
As monotherapy in patients who have received at least three prior lines of therapy including a proteasome inhibitor (PI) and an immunomodulatory agent or who are double-refractory to a PI and an immunomodulatory agent
Please click here to see the full Prescribing Information.
Learn more at www.janssen.com.
JNJ's DARZALEX combo regime shows significant overall survival benefits in multiple myeloma
Jun. 14, 2021 4:55 AM ETJohnson & Johnson (JNJ)By: Mamta Mayani, SA News Editor1 Comment
- The Janssen Pharmaceutical Companies of Johnson & Johnson (NYSE:JNJ) announces overall survival (OS) results from the Phase 3 MAIA study showing the addition of DARZALEX (daratumumab) to lenalidomide and dexamethasone (D-Rd) resulted in a statistically significant survival benefit over lenalidomide and dexamethasone (Rd) alone in patients with newly diagnosed multiple myeloma who were ineligible for autologous stem cell transplant and were treated to progression.
- https://seekingalpha.com/news/3705858-jnjs-darzalex-combo-regime-shows-significant-overall-survival-benefits-in-multiple-myeloma
- https://seekingalpha.com/symbol/JNJ
- https://www.darzalex.com/
IMBRUVICA® (ibrutinib) Plus VENCLEXTA®/VENCLYXTO® (venetoclax)
June 12, 2021
IMBRUVICA® (ibrutinib) Plus VENCLEXTA®/VENCLYXTO® (venetoclax) Combination Shows Superior Progression-Free Survival Compared to Chlorambucil Plus Obinutuzumab in First-line Chronic Lymphocytic Leukemia (CLL) Phase 3 GLOW Study
- All-oral, once-daily, chemotherapy-free, fixed-duration ibrutinib plus venetoclax (I+V) combination met its primary endpoint of progression-free survival (PFS) as assessed by an independent review committee (IRC); I+V reduced the risk of disease progression or death by 78% compared to chlorambucil plus obinutuzumab (C+O)
- The safety profile of I+V was generally consistent with the safety profile of the single agents, and tolerability profiles were consistent with CLL treatment in the enrolled patient population
- Data will be presented during the late-breaking abstract session at the European Hematology Association (EHA) 2021 Virtual Congress (Abstract #LB1902)
NORTH CHICAGO, Ill., June 12, 2021 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced new data from the Phase 3 GLOW study comparing the efficacy and safety of the combination of IMBRUVICA® (ibrutinib) plus VENCLEXTA®/VENCLYXTO® (venetoclax) (I+V) versus chlorambucil plus obinutuzumab (C+O) for first-line treatment in patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) who had active disease requiring treatment per the International Workshop on CLL (iwCLL) criteria. The study met its primary endpoint of superior progression-free survival (PFS) as assessed by an independent review committee (IRC) with a HR 0.216 (95% CI, 0.131-0.357; p < 0.0001), demonstrating a reduction in the risk of disease progression or death for I+V of approximately 78% compared to C+O. I+V is the first all-oral, once-daily, chemotherapy-free, fixed-duration investigational combination. Results of the study will be presented at the European Hematology Association (EHA) 2021 Virtual Congress (Abstract #LB1902) during late-breaking abstract session on June 12 from 4:00-5:30 p.m. CEST.
The PFS benefit with I+V was consistent across pre-specified subgroups, including patients 65 years and older and those with comorbidities (CIRS >6). The median PFS for C+O was 21 months while the median PFS for I+V had not been reached at the time of analysis. The safety profile of I+V was generally consistent with the safety profile of the single agents and tolerability profiles were consistent with CLL treatment in the enrolled patient population.
"With CLL being one of the most common types of blood cancer, the expansion of research into additional treatment options for patients is an important clinical undertaking," said Arnon Kater, M.D., Ph.D., deputy head of hematology, University of Amsterdam Faculty of Medicine. "The progression-free survival findings of ibrutinib and venetoclax in the GLOW study are promising and show the potential to become an additional treatment option for people living with CLL."
About the GLOW Study
The GLOW study is a randomized, open label Phase 3 trial comparing progression-free survival in patients treated with either I+V or C+O as assessed by an Independent Review Committee. It enrolled patients (pts) aged ≥65 years or 18-64 years with cumulative illness rating scale score >6 or creatinine clearance <70 mL/min who had active disease requiring treatment per the International Workshop on CLL (iwCLL) criteria. Patients with del(17p) or known TP53 mutations were excluded. There were 211 patients randomly assigned in a 1:1 ratio to receive either I+V (106) and or C+O (105) and the median age was 71 years. Patients assigned to I+V received treatment for 15 cycles (1 cycle is 28 days), starting with three cycles of ibrutinib monotherapy lead-in followed by the combination of I+V for 12 cycles. Patients assigned to C+O were treated for six cycles.
About IMBRUVICA® (Ibrutinib)
IMBRUVICA® (ibrutinib) is a once-daily, first-in-class BTK inhibitor that is administered orally, and is jointly developed and commercialized by Pharmacyclics, LLC, an AbbVie Company, and Janssen Biotech, Inc. (Janssen). The BTK protein sends important signals that tell B cells to mature and produce antibodies. BTK signaling is needed by specific cancer cells to multiply and spread.4,5 By blocking BTK, IMBRUVICA may help move abnormal B cells out of their nourishing environments in the lymph nodes, bone marrow, and other organs.6
Since its launch in 2013, IMBRUVICA has received 11 FDA approvals across six disease areas: chronic lymphocytic leukemia (CLL) with or without 17p deletion (del17p); small lymphocytic lymphoma (SLL) with or without del17p; Waldenström macroglobulinemia; previously-treated patients with mantle cell lymphoma (MCL)*; previously-treated patients with marginal zone lymphoma (MZL) who require systemic therapy and have received at least one prior anti-CD20-based therapy* – and previously-treated patients with chronic graft-versus-host disease (cGVHD) after failure of one or more lines of systemic therapy.7
IMBRUVICA is now approved in more than 100 countries and has been used to treat more than 230,000 patients worldwide across its approved indications. IMBRUVICA is the only FDA-approved medicine in WM and cGVHD. IMBRUVICA has been granted four Breakthrough Therapy Designations from the U.S. FDA. This designation is intended to expedite the development and review of a potential new drug for serious or life-threatening diseases. IMBRUVICA was one of the first medicines to receive FDA approval via the Breakthrough Therapy Designation pathway.
Since 2019, the National Comprehensive Cancer Network® (NCCN®), a not-for-profit alliance of 28 leading cancer centers devoted to patient care, research, and education, recommends ibrutinib (IMBRUVICA®) as a preferred regimen for the initial treatment of CLL/SLL and has Category 1 treatment status for treatment-naïve patients without deletion 17p. Since January 2020, the NCCN Guidelines® have categorized IMBRUVICA with or without rituximab as a preferred regimen for the treatment of relapsed/refractory MCL. As of September 2020, the NCCN guidelines were updated to reflect IMBRUVICA with or without rituximab as the only Category 1 preferred regimen for both untreated and previously treated WM patients.
IMBRUVICA is being studied alone and in combination with other treatments in several blood and solid tumor cancers and other serious illnesses. IMBRUVICA is the most comprehensively studied BTK inhibitor, with more than 150 ongoing clinical trials. There are approximately 30 ongoing company-sponsored trials, 14 of which are in Phase 3, and more than 100 investigator-sponsored trials and external collaborations that are active around the world. For more information, visit www.IMBRUVICA.com
*Accelerated approval was granted for the MCL and MZL indications based on overall response rate. Continued approval for MCL and MZL may be contingent upon verification and description of clinical benefit in confirmatory trials.
About VENCLEXTA®/VENCLYXTO® (venetoclax)
VENCLEXTA®/VENCLYXTO® (venetoclax) is a first-in-class medicine that selectively binds and inhibits the B-cell lymphoma-2 (BCL-2) protein. In some blood cancers, BCL-2 prevents cancer cells from undergoing their natural death or self-destruction process, called apoptosis. VENCLXEXTA/VENCLYXTO targets the BCL-2 protein and works to help restore the process of apoptosis.
VENCLEXTA/VENCLYXTO is being developed by AbbVie and Roche. It is jointly commercialized by AbbVie and Genentech, a member of the Roche Group, in the U.S. and by AbbVie outside of the U.S. Together, the companies are committed to BCL-2 research and to studying venetoclax in clinical trials across several blood cancers. Venetoclax is approved in more than 80 countries, including the U.S.
Please click here for full Prescribing Information.
Uses of VENCLEXTA® (venetoclax) in US
VENCLEXTA is a prescription medicine used:
- to treat adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).
- in combination with azacitidine, or decitabine, or low-dose cytarabine to treat adults with newly diagnosed acute myeloid leukemia (AML) who:
- are 75 years of age or older, or
- have other medical conditions that prevent the use of standard chemotherapy.
It is not known if VENCLEXTA is safe and effective in children.
The full U.S. prescribing information, including Medication Guide, for VENCLEXTA® can be found here.
Indications and Important VENCLYXTO® (venetoclax) EU Safety Information9
Indication
Venclyxto in combination with obinutuzumab is indicated for the treatment of adult patients with previously untreated chronic lymphocytic leukaemia (CLL).
Venclyxto in combination with rituximab is indicated for the treatment of adult patients with CLL who have received at least one prior therapy.
Venclyxto monotherapy is indicated for the treatment of CLL:
- In the presence of 17p deletion or TP53 mutation in adult patients who are unsuitable for or have failed a B-cell receptor pathway inhibitor, or
- In the absence of 17p deletion or TP53 mutation in adult patients who have failed both chemoimmunotherapy and a B-cell receptor pathway inhibitor.
Venclyxto in combination with a hypomethylating agent is indicated for the treatment of adult patients with newly diagnosed acute myeloid leukaemia (AML) who are ineligible for intensive chemotherapy.
For more information, please visit http://www.abbvie.com/oncology.
For more information about AbbVie, please visit us at www.abbvie.com.
https://seekingalpha.com/symbol/ABBV
https://seekingalpha.com/symbol/JNJ
https://seekingalpha.com/symbol/RHHBY
JNJ/AbbVie's Imbruvica + Venclexta show promising progression-free survival in late-stage blood cancer study
Jun. 14, 2021 4:26 AM ETAbbVie Inc. (ABBV), JNJBy: Mamta Mayani, SA News Editor2 Comments
- AbbVie (NYSE:ABBV) announces new data from the Phase 3 GLOW study comparing the efficacy and safety of the combination of Johnson & Johnson (NYSE:JNJ) company Janssen Biotech's Imbruvica (ibrutinib) plus Venclexta/Venclyxto (venetoclax) (I+V) vs. chlorambucil plus obinutuzumab (C+O) for first-line treatment in patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).
- https://seekingalpha.com/news/3705857-jnjabbvies-imbruvicavenclexta-show-promising-progression-free-survival-in-blood-cancer
COVIDROPS™ (Intranasal COVI-AMG Neutralizing Antibody – STI 2099)
Sorrento Receives Authorization From the UK Regulatory Agency to Conduct a Phase 2 Clinical Trial for COVI-DROPS in an Outpatient Setting
June 11, 2021 at 4:11 PM EDTDownload PDF
- Large Phase 2 efficacy trial cleared to start in the United Kingdom in newly diagnosed SARS-CoV-2 infected patients.
- COVI-DROPS is administered by intranasal drops and the antibody is active against the original SARS-CoV-2 virus, as well as the UK/Alpha and India/Delta variants, currently prevalent in the UK and US.
SAN DIEGO, June 11, 2021 (GLOBE NEWSWIRE) -- Sorrento Therapeutics, Inc. (Nasdaq: SRNE, "Sorrento") today announced that the Medicines and Healthcare products Regulatory Agency (MHRA), the United Kingdom’s regulatory agency, has cleared Sorrento’s COVI-DROPS product candidate for a Phase 2 efficacy trial. The application was submitted as a rolling application and the MHRA cleared the study in less than a month from Sorrento’s first submission to the MHRA. The application was supported by the safety data from a healthy subject study completed in the US, which showed a safety profile comparable to placebo with doses up to 60 mg. In this study, there were no serious adverse effects or dose limiting toxicities and all adverse effects were mild in severity. The maximum tolerated dose was not reached.
The Phase 2 efficacy trial is a large double-blind clinical trial enrolling 350 outpatients with COVID-19 who are asymptomatic or have mild symptoms in a 2:2:1 randomization with patients receiving 10mg, 20mg or placebo (details can be found on www.ClinicalTrials.gov using the identifier NCT04900428). This trial will complement the Phase 2 trial currently being started in the US and a separate trial to be started in Mexico.
COVI-DROPS is administered as an intranasal instillation in each nares to recently infected patients and utilizes the same neutralizing antibody drug substance as COVI-AMG, the intravenous formulation. The antibody is active against the original SARS-CoV-2 virus as well as the most prevalent viral variants of concern (VoCs) currently infecting the UK and the US. These variants include the UK/Alpha and the India/Delta variants of concern.
The results of this Phase 2 trial in the UK will be combined with the results of the US and Mexico Phase 2 trials and should the results of these studies demonstrate that COVI-DROPS is both safe and effective against SARS-CoV-2, Sorrento will apply for Emergency Use Authorization in the US, India, UK, Mexico and European Union as well as other territories.
For more information visit www.sorrentotherapeutics.com.
COVIDROPS™ (Intranasal COVI-AMG Neutralizing Antibody – STI 2099)
In addition to the intravenous formulation of COVI-AMG™ neutralizing antibody, Sorrento is developing COVIDROPS which will be the intranasal formulation of COVI-AMG™.
https://sorrentotherapeutics.com/research/covid-19/covidrops/
Sorrento rises on U.K. regulatory clearance of mid-stage trial for COVID-19 therapy
Jun. 11, 2021 5:13 PM ET Sorrento Therapeutics, Inc. (SRNE)
By: Dulan Lokuwithana, SA News Editor3 Comments
- Sorrento Therapeutics (NASDAQ:SRNE) has risen ~3.5% in extended trading after announcing that the company received regulatory clearance in the U.K. to start a Phase 2 efficacy trial for its COVI-DROPS product candidate.
- https://seekingalpha.com/news/3705814-sorrento-rises-on-uk-regulatory-clearance-of-mid-stage-trial-for-covid-19-therapy
- https://seekingalpha.com/symbol/SRNE
- https://sorrentotherapeutics.com/
- https://sorrentotherapeutics.com/research/covid-19/covidrops/
Yescarta® (axicabtagene ciloleucel)
June 12, 2021
Longer-term Data for Kite’s Yescarta® in Relapsed or Refractory Follicular Lymphoma Demonstrate Substantial Survival Improvement Over Current Therapies in Comparative Analysis
-- 94% of Patients Achieved a Response at 18 Months in Pivotal ZUMA-5 Trial --
-- Yescarta Reduced Risk of Death by 58% Compared to Current Therapies in Weighted Analysis of ZUMA-5 Compared to SCHOLAR-5 External Control Cohort --
-- Late-breaking Data Presented at European Hematology Association (EHA) 2021 --
SANTA MONICA, Calif.--(BUSINESS WIRE)-- Kite, a Gilead Company (Nasdaq: GILD), today announced follow-up results from the pivotal ZUMA-5 trial of Yescarta® (axicabtagene ciloleucel) – the first and only CAR T-cell therapy approved in patients with relapsed or refractory indolent follicular lymphoma (FL). At a minimum follow-up of 18 months, 94% of patients had achieved a response, and secondary endpoints of median progression-free survival (PFS) and overall survival (OS) were not yet reached. In a weighted analysis comparing ZUMA-5 patients with a minimum of 18 months follow-up with those observed in SCHOLAR-5, an external control cohort, Yescarta demonstrated superior OS and PFS over currently available treatments. These data were presented today as a part of a late-breaking session at the 26th Annual Meeting of the European Hematology Association (EHA2021) taking place virtually this year from June 9-17 (Abstract #LB1904).
Yescarta received accelerated approval from the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with relapsed or refractory follicular lymphoma after two or more lines of systemic therapy in March 2021. The Yescarta U.S. Prescribing Information has a BOXED WARNING for the risks of CRS and neurologic toxicities, and Yescarta is approved with a risk evaluation and mitigation strategy (REMS) due to these risks; see below for Important Safety Information.
About ZUMA-5
ZUMA-5 is an ongoing, single-arm, open-label, multicenter trial evaluating 146 patients (≥18 years old) with relapsed or refractory iNHL including FL, who received at least two prior lines of systemic therapy, including the combination of an anti-CD20 monoclonal antibody and an alkylating agent. Efficacy was established on the basis of objective response rate (ORR) and duration of response (DoR) as assessed by an independent review committee per the 2014 Lugano Classification.
About SCHOLAR-5
SCHOLAR-5 is an international, multicenter, retrospective external control cohort of patients with relapsed or refractory FL. Data were sourced from seven institutions across five countries from patients who initiated a third-line or higher therapy after July 2014. In these later lines of treatment, therapeutic regimens are highly heterogenous and without any clear standard of care. Data from the pivotal idelalisib DELTA trial were also included. ZUMA-5 eligibility criteria were applied to the cohort, with patients excluded or censored upon transformation. Single agent anti-CD20 antibody was not counted as line of therapy for eligibility, similar to ZUMA-5 eligibility requirements.
About Yescarta
Yescarta is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of:
- Adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma, high grade B-cell lymphoma, and DLBCL arising from follicular lymphoma.
Limitations of Use: Yescarta is not indicated for the treatment of patients with primary central nervous system lymphoma. - Adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy. This indication is approved under accelerated approval based on response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).
- https://www.yescarta.com/
For more information on Kite, please visit www.kitepharma.com.
U.S. Prescribing Information for Yescarta including BOXED WARNING, is available at www.kitepharma.com and www.gilead.com.
Kite, the Kite logo, Yescarta, Tecartus, XLP and GILEAD are trademarks of Gilead Sciences, Inc. or its related companies.
For more information on Kite, please visit the company’s website at www.kitepharma.com or call Gilead Public Affairs at 1-800-GILEAD-5 or 1-650-574-3000. Follow Kite on social media on Twitter (@KitePharma) and LinkedIn.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210612005024/en/
Gilead company, Kite’s Yescarta shows survival improvement in follicular lymphoma
Jun. 14, 2021 12:45 AM ET
By: Mamta Mayani, SA News Editor
- Kite, a Gilead (NASDAQ:GILD) company announces follow-up results from ZUMA-5 trial of Yescarta (axicabtagene ciloleucel) – the CAR T-cell therapy approved in patients with relapsed or refractory indolent follicular lymphoma (FL).
- https://seekingalpha.com/news/3705850-gilead-company-kites-yescarta-shows-survival-improvement-in-follicular-lymphoma
- https://seekingalpha.com/symbol/GILD
- https://www.gilead.com/
biotecMAX™ April/May/June 2021 Insight
VENCLYXTO®/VENCLEXTA®(venetoclax)
June 11, 2021
New Data Shows AbbVie's VENCLYXTO®/VENCLEXTA® Fixed Duration Combination Demonstrates Sustained Progression-Free Survival in Chronic Lymphocytic Leukemia Patients after Three Years off Treatment
- Four-year follow-up analysis of Phase 3 CLL14 trial (median follow-up 52.4 months), continues to demonstrate longer progression-free survival (PFS) after fixed-duration venetoclax plus obinutuzumab compared to fixed duration chlorambucil plus obinutuzumab in previously untreated patients with chronic lymphocytic leukemia (CLL)
- A majority of patients treated with venetoclax and obinutuzumab remain without relapse three years after completing the venetoclax-based combination treatment
NORTH CHICAGO, Ill., June 11, 2021 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced results from a four-year follow-up analysis of the Phase 3 CLL14 trial, which showed that previously untreated patients with chronic lymphocytic leukemia (CLL) with coexisting conditions who were treated with VENCLYXTO®/VENCLEXTA® (venetoclax) plus obinutuzumab continued to show longer progression-free survival (PFS) and higher rates of undetectable minimal residual disease (MRD) compared to patients receiving a standard of care chemoimmunotherapy regimen of obinutuzumab and chlorambucil. The data were presented for the first time today during the virtual 26th European Hematology Association (EHA) Annual Congress (abstract #S146).
"The CLL14 trial results observed after four years of follow-up with treatment of venetoclax plus obinutuzumab show that these patients can experience long-lasting responses without disease progression, years after stopping treatment," said Mohamed Zaki, M.D., Ph.D., vice president and head, global oncology development, AbbVie.
After a median follow-up of more than four years (52.4 months), patients treated with the VENCLYXTO/VENCLEXTA and obinutuzumab combination (n=216) continued to demonstrate longer PFS compared to patients on treatment with obinutuzumab and chlorambucil, the risk of disease progression or death was reduced by 67 percent (n=216; median not reached compared to 36.4 months; hazard ratio [HR] 0.33 [95% CI 0.25-0.45]); results are descriptive. At four years after randomization, the PFS rate for patients treated with the VENCLYXTO/VENCLEXTA-based combination was 74 percent compared to 35.4 percent for patients treated with obinutuzumab and chlorambucil. The improvement in PFS was observed across all clinical and biological risk groups, including patients with TP53 mutation, 17p deletion and unmutated IGHV status.1
VENCLYXTO/VENCLEXTA is being developed by AbbVie and Roche. It is jointly commercialized by AbbVie and Genentech, a member of the Roche Group, in the U.S. and by AbbVie outside of the U.S.
About the CLL14 Phase 3 Trial3,7,8
The prospective, multicenter, open-label, randomized Phase 3 CLL14 trial, which was conducted in close collaboration with the German CLL Study Group (DCLLSG), evaluated the efficacy and safety of a combined regimen of VENCLYXTO/VENCLEXTA and obinutuzumab (n=216) versus obinutuzumab and chlorambucil (n=216) in previously untreated patients with CLL and coexisting medical conditions (total Cumulative Illness Rating Scale [CIRS] score >6 or creatinine clearance <70 mL/min). The therapies were administered for a fixed duration of 12 months for VENCLYXTO/VENCLEXTA in combination with six cycles of obinutuzumab. The trial enrolled 432 patients, all of whom were previously untreated according to the International Workshop on Chronic Lymphocytic Leukemia (iwCLL) criteria. Efficacy was based on PFS as assessed by an independent review committee.
Key secondary endpoints were rates of MRD in peripheral blood and bone marrow, overall and complete response rates, MRD in complete response in peripheral blood and bone marrow, and overall survival.
The four-year, follow-up analysis showed an OS rate of 85.4% with Ven-Obi versus 83.1% with chlorambucil in combination with obinutuzumab (Obi-Clb; HR 0.85, 95% CI [0.54-1.35]; P=0.49).
In patients with CLL receiving combination therapy with obinutuzumab, serious adverse reactions (ARs) were most often due to febrile neutropenia and pneumonia (5 percent each). The most common ARs (≥20 percent) of any grade were neutropenia (60 percent), diarrhea (28 percent), and fatigue (21 percent). Fatal ARs that occurred in the absence of disease progression and with onset within 28 days of the last study treatment were reported in 2 percent (4/212) of patients, most often from infection.
About VENCLYXTO® (venetoclax)
VENCLYXTO® (venetoclax) is a first-in-class medicine that selectively binds and inhibits the B-cell lymphoma-2 (BCL-2) protein. In some blood cancers, BCL-2 prevents cancer cells from undergoing their natural death or self-destruction process, called apoptosis. VENCLYXTO targets the BCL-2 protein and works to help restore the process of apoptosis.
VENCLYXTO is being developed by AbbVie and Roche. It is jointly commercialized by AbbVie and Genentech, a member of the Roche Group, in the U.S. and by AbbVie outside of the U.S. Together, the companies are committed to BCL-2 research and to studying venetoclax in clinical trials across several blood and other cancers. Venetoclax is approved in more than 80 countries, including the U.S.
Indication and Important VENCLYXTO (venetoclax) EU Safety Information3
Indications
Venclyxto in combination with obinutuzumab is indicated for the treatment of adult patients with previously untreated chronic lymphocytic leukaemia (CLL).
Venclyxto in combination with rituximab is indicated for the treatment of adult patients with CLL who have received at least one prior therapy.
Venclyxto monotherapy is indicated for the treatment of CLL:
- In the presence of 17p deletion or TP53 mutation in adult patients who are unsuitable for or have failed a B-cell receptor pathway inhibitor, or
- In the absence of 17p deletion or TP53 mutation in adult patients who have failed both chemoimmunotherapy and a B-cell receptor pathway inhibitor.
Venclyxto in combination with a hypomethylating agent is indicated for the treatment of adult patients with newly diagnosed acute myeloid leukaemia (AML) who are ineligible for intensive chemotherapy.
Contraindications
Hypersensitivity to the active substance or to any of the excipients is contraindicated. Concomitant use of strong CYP3A inhibitors at initiation and during the dose-titration phase due to increased risk for tumour lysis syndrome (TLS). Concomitant use of preparations containing St. John's wort as Venclyxto efficacy may be reduced.
For more information, please visit http://www.abbvie.com/oncology.
For more information about AbbVie, please visit us at www.abbvie.com.
AbbVie, Roche's Venclexta combo shows sustained benefit in blood cancers
Jun. 11, 2021 3:19 AM ET AbbVie Inc. (ABBV), RHHBY By: Mamta Mayani, SA News Editor3 Comments
- Roche (OTCQX:RHHBY) unit Genentech announces the latest data from Phase III studies of Venclexta (venetoclax), developed in collaboration with AbbVie (NYSE:ABBV) in chronic lymphocytic leukemia (CLL) and acute myeloid leukemia (AML).
- https://seekingalpha.com/news/3705524-abbvie-roches-venclexta-combo-shows-sustained-benefit-in-blood-cancers
- https://seekingalpha.com/symbol/RHHBY
- https://seekingalpha.com/symbol/ABBV
- https://www.abbvie.com/
- https://www.venclexta.com/
Breyanzi (lisocabtagene maraleucel)
Bristol Myers Squibb Announces Positive Topline Results from Phase 3 TRANSFORM Trial Evaluating Breyanzi (lisocabtagene maraleucel) Versus Chemotherapy Followed by Stem Cell Transplant in Second-line Relapsed or Refractory Large B-cell Lymphoma
06/10/2021CATEGORY:
Study met primary and key secondary endpoints, demonstrating a highly statistically significant improvement in event-free survival, complete response rate and progression-free survival compared to standard of care
Breyanzi safety results consistent with data from pivotal TRANSCEND NHL 001 trial
Results represent the first time a therapy has demonstrated benefit compared to high-dose chemotherapy and stem cell transplant in relapsed or refractory large B-cell lymphoma, and support the potential of Breyanzi in earlier lines of therapy in this patient population
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced positive topline results from TRANSFORM, a global, randomized, multicenter Phase 3 study evaluating Breyanzi (lisocabtagene maraleucel) as a second-line treatment in adults with relapsed or refractory large B-cell lymphoma (LBCL) compared to salvage therapy followed by high-dose chemotherapy and hematopoietic stem cell transplant, which is currently considered a gold standard treatment for these patients. Results of a pre-specified interim analysis conducted by an independent review committee showed the study met its primary endpoint of demonstrating a clinically meaningful and highly statistically significant improvement in event-free survival, as well as key secondary endpoints of complete response rate and progression-free survival compared to standard of care. Overall survival data were immature at the time of this interim analysis. Safety results were consistent with the known safety profile of Breyanzi for the treatment of LBCL in the third-line setting, and no new safety concerns were identified in this second-line setting.
“We ambitiously designed the TRANSFORM trial to evaluate Breyanzi’s potential in the second-line setting for patients with relapsed or refractory large B-cell lymphoma against the standard of care regimen of high-dose chemotherapy and autologous stem cell transplant,” said Noah Berkowitz, M.D., Ph.D., senior vice president, Hematology and Cell Therapy Development, Bristol Myers Squibb. “These positive interim results build on our commitment to bring CAR T cell therapies into earlier lines and highlight the potential of Breyanzi to transform the treatment paradigm for this difficult-to-treat disease, possibly supplanting the need for patients to undergo current aggressive treatment regimens.”
About TRANSFORM
TRANSFORM (NCT03575351) is a pivotal, global, randomized, multicenter Phase 3 trial evaluating Breyanzi compared to current standard of care regimens, including high-dose chemotherapy (HDCT) and hematopoietic stem cell transplant (HSCT), in adults with large B-cell lymphoma that is primary refractory or relapsed within 12 months of initial therapy and who are eligible for stem cell transplant. Patients were randomized to receive Breyanzi or standard of care salvage therapy, including rituximab plus dexamethasone, high-dose cytarabine, and cisplatin (R-DHAP), rituximab plus ifosfamide, carboplatin and etoposide (R-ICE), or rituximab plus gemcitabine, dexamethasone and cisplatin (R-GDP) per the investigators’ choice before proceeding to HDCT and HSCT. The primary endpoint of the study is event-free survival, defined as time from randomization to death from any cause, progressive disease, failure to achieve complete response or partial response by nine weeks post-randomization, or start of new antineoplastic therapy due to efficacy concerns, whichever occurs first. Complete response rate is a key secondary endpoint. Other efficacy endpoints include progression-free survival, overall survival, overall response rate and duration of response. All enrolled patients have large B-cell lymphoma and were relapsed or refractory within 12 months from CD20 antibody and anthracycline containing first-line therapy.
About Breyanzi
Breyanzi is a CD-19 directed chimeric antigen receptor (CAR) T cell therapy with a defined composition and 4-1BB costimulatory domain. Breyanzi is administered as a defined composition to reduce variability of the CD8 and CD4 component dose. The 4-1BB signaling domain enhances the expansion and persistence of the CAR T cells. Breyanzi is approved by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with relapsed or refractory LBCL after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified (including DLBCL arising from indolent lymphoma), high-grade B-cell lymphoma, primary mediastinal large B-cell lymphoma, and follicular lymphoma grade 3B.
Breyanzi is also approved in Japan for relapsed and refractory LBCL, and Marketing Authorization Applications for Breyanzi are currently under review in the European Union, Switzerland and Canada. Bristol Myers Squibb’s clinical development program for Breyanzi includes clinical studies in earlier lines of treatment for patients with relapsed or refractory LBCL and other types of lymphoma. For more information, visit clinicaltrials.gov.
U.S. FDA-Approved Indication for Breyanzi
BREYANZI is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of adult patients with relapsed or refractory (R/R) large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified (including DLBCL arising from indolent lymphoma), high-grade B-cell lymphoma, primary mediastinal large B-cell lymphoma, and follicular lymphoma grade 3B.
Limitations of Use: BREYANZI is not indicated for the treatment of patients with primary central nervous system lymphoma.
Please see full Prescribing Information, including Boxed WARNINGS and Medication Guide.
For more information about Bristol Myers Squibb, visit us at BMS.com
Bristol Myers announces positive topline data for Breyanzi in B-cell Lymphoma as second-line therapy
Jun. 10, 2021 7:21 AM ETBristol-Myers Squibb Company (BMY)By: Dulan Lokuwithana, SA News Editor
- Bristol Myers Squibb (NYSE:BMY) says that its chimeric antigen receptor (CAR) T cell therapy Breyanzi (lisocabtagene maraleucel) reached the primary endpoint with statistical and clinical significance in a Phase 3 trial as second-line treatment for patients with large B-cell lymphoma (LBCL). https://seekingalpha.com/news/3704917-bristol-myers-announces-positive-topline-data-for-breyanzi-in-b-cell-lymphoma-as-second-line-therapy
- https://seekingalpha.com/symbol/BMY
- https://www.bms.com/
Reblozyl® (luspatercept-aamt)
Bristol Myers Squibb and Acceleron Present First Results from Phase 2 BEYOND Study of Reblozyl® (luspatercept-aamt) in Adults with Non-Transfusion Dependent(NTD)Beta Thalassemia
06/11/2021CATEGORY:
Results showed treatment with Reblozyl plus best supportive care improved anemia in 77% of patients compared to placebo
Reblozyl was generally well tolerated and improvements in hemoglobin correlated with improved patient-reported outcomes over a continuous 12-week interval
In the study, 89.6% of patients treated with Reblozyl remained transfusion free vs. 67.3% of patients in the placebo arm at weeks 1-24
Results featured in Presidential Symposium of European Hematology Association’s Virtual Congress as one of top six abstracts submitted
PRINCETON, N.J. & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) and Acceleron Pharma Inc. (NASDAQ: XLRN) today announced the first data from the Phase 2 BEYOND study evaluating Reblozyl®(luspatercept-aamt), a first-in-class erythroid maturation agent, plus best supportive care in adult patients with non-transfusion dependent (NTD) beta thalassemia, were presented at the European Hematology Association (EHA) 2021 Virtual Congress as part of its Presidential Symposium (Abstract #S101). Results demonstrated that 77.7% of patients treated with Reblozyl achieved a hemoglobin increase (≥1.0 gram/deciliter) compared to 0% of patients in the placebo arm. Changes in patient-reported outcomes also correlated with increases in hemoglobin. NTD beta thalassemia is a term used to describe patients who do not require lifelong regular red blood cell (RBC) transfusions for survival, although they may require occasional or even frequent transfusions, usually for defined periods of time.
“Patients with non-transfusion dependent beta thalassemia experience chronic anemia and iron overload, which may lead to a range of clinical complications, and treatment options are greatly needed,” said Ali Taher, M.D., Ph.D., FRCP, of American University of Beirut and BEYOND study investigator. “Results from the BEYOND study show the clinical potential of luspatercept to sustain the elevation of hemoglobin levels in a majority of patients regardless of their baseline hemoglobin status, and improvements were noted in quality of life outcomes in adults with non-transfusion dependent beta thalassemia.”
Reblozyl is the first and only erythroid maturation agent approved in the European Union, United States and Canada to address anemia-associated beta thalassemia and lower-risk myelodysplastic syndromes, representing an important class of therapy for eligible patients.1,2,3
BEYOND Study Results
BEYOND is a Phase 2, randomized, double-blind, placebo-controlled multi-center study to determine the efficacy and safety of Reblozyl versus placebo in adults with non-transfusion dependent (NTD) beta thalassemia. Eligible patients were ≥18 years with beta thalassemia or hemoglobin (Hb) E beta thalassemia and received ≤5 red blood cell (RBC) units in the 24 weeks prior to randomization, with mean baseline Hb ≤10.0 gram/deciliter (g/dL).4
In the study, 145 patients were randomized 2:1 to receive Reblozyl, 1 milligram/kilogram (titration up to 1.25 mg/kg) or placebo subcutaneously every 3 weeks for ≥48 weeks. Patients in both arms continued to receive best supportive care, including RBC transfusions as indicated and iron chelation therapy. The primary endpoint was achievement of ≥1.0 g/dL mean Hb increase from baseline over a continuous 12-week interval from weeks 13-24 in the absence of RBC transfusions. Secondary endpoints included proportion of patients who remained transfusion free over weeks 1-24, who achieved mean Hb increase of ≥1.5 g/dL from baseline to weeks 13-24, and mean change in NTD beta thalassemia patient-reported outcome tiredness and weakness (NTDT-PRO T/W) domain scores (higher scores reflect worse quality of life; QoL).4
Over a continuous 12-week interval from weeks 13-24 in the absence of RBC transfusions, 74 of 96 (77.1%) patients in the Reblozyl treatment arm achieved the study’s primary endpoint, ≥1.0 g/dL mean Hb increase from baseline, versus 0 of 49 (0%) patients in the placebo arm (P<0.0001). The primary endpoint was achieved by 40 of 55 (72.7%) patients in the Reblozyl arm with mean baseline Hb of <8.5 g/dL versus 0 (0%) of patients in the placebo arm (P<0.0001), and 34 of 41 patients (82.9%) with mean baseline Hb of ≥8.5 g/dL versus 0 patients (0%) in the placebo arm (P<0.0001). In a key secondary endpoint of the study, during weeks 13-24, 50 of 96 patients (52.1%) in the Reblozyl arm achieved mean Hb increase of ≥1.5 g/dL compared to baseline versus 0 patients (0%) in the placebo arm (P<0.0001). 89.6% of patients in the Reblozyl arm remained transfusion free at weeks 1-24 versus 67.3% of patients in the placebo arm (P=0.0013). Improvements in patient-reported QoL outcomes (tiredness and weakness) were also observed to correlate with Hb increases.
The most common treatment-emergent adverse events of any grade occurring in ≥5% of patients were bone pain (36.5% Reblozyl versus 6.1% placebo), headache (30.2% versus 20.4%), and arthralgia (29.2% versus 14.3%). No malignancies or thromboembolic events were reported in patients treated with Reblozyl.
About Reblozyl®
Reblozyl (luspatercept-aamt), a first-in-class erythroid maturation agent, promotes late-stage red blood cell maturation in animal models. Bristol Myers Squibb and Acceleron are jointly developing Reblozyl as part of a global collaboration. Reblozyl is currently approved in the U.S. for the treatment of:
- anemia in adult patients with beta thalassemia who require regular red blood cell transfusions, and
- anemia failing an erythropoiesis stimulating agent and requiring 2 or more red blood cell units over 8 weeks in adult patients with very low- to intermediate-risk myelodysplastic syndromes with ring sideroblasts (MDS-RS) or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T).
Reblozyl is not indicated for use as a substitute for red blood cell transfusions in patients who require immediate correction of anemia.
Please see full Prescribing Information and Summary of Product Characteristics for REBLOZYL
For more information about Bristol Myers Squibb, visit us at BMS.com
For more information, please visit https://acceleronpharma.com.
Bristol Myers, Acceleron's Reblozyl shows encouraging action in adults with beta thalassemia
Jun. 11, 2021 7:01 AM ET
Bristol-Myers Squibb Company (BMY), XLRN
By: Mamta Mayani, SA News Editor1 Comment
- Bristol Myers Squibb (NYSE:BMY) and Acceleron Pharma (NASDAQ:XLRN) announce the first data from Phase 2 BEYOND study evaluating Reblozyl (luspatercept-aamt) plus best supportive care in adult patients with non-transfusion dependent (NTD) beta thhttps://seekingalpha.com/news/3705553-bristol-myers-accelerons-reblozyl-shows-encouraging-action-in-adults-with-beta-thalassemiaalassemia.
- https://seekingalpha.com/symbol/XLRN
- https://seekingalpha.com/symbol/BMY
- https://www.bms.com/
ADUHELM™ (aducanumab-avwa)
ADUHELM (aducanumab-avwa)
FDA approves Biogen’s Alzheimer’s drug, the first new therapy for the disease in nearly two decades
PUBLISHED MON, JUN 7 202111:03 AM EDTUPDATED 2 MIN AGOBerkeley Lovelace Jr.@BERKELEYJR
The Food and Drug Administration on Monday approved Biogen Alzheimer’s drug aducanumab, making it the first drug cleared by U.S. regulators to slow cognitive decline in people living with Alzheimer’s and the first new medicine for the disease in nearly two decades.
The FDA’s decision was highly anticipated. The drug, which is marketed under the name Aduhelm, is also expected to generate billions of dollars in revenue for the company.
“We are well-aware of the attention surrounding this approval,” Dr. Patrizia Cavazzoni, director of the FDA’s Center for Drug Evaluation and Research, said in a press release. “We understand that Aduhelm has garnered the attention of the press, the Alzheimer’s patient community, our elected officials, and other interested stakeholders.”
“With a treatment for a serious, life-threatening disease in the balance, it makes sense that so many people were following the outcome of this review,” Cavazzoni added.
https://www.cnbc.com/quotes/BIIB
Biogen’s Alzheimer’s Drug Approved in Treatment Landmark
From the Archive: Biogen Alzheimer’s Drug Puts FDA’s Judgment in Harsh Spotlight
Earlier: Biogen's New Alzheimer's Drug Faces Hurdles Reaching Patients, Even if It's Approved
June 7, 202111:08 AM EDT
U.S. FDA approves Biogen Alzheimer's drug
Biogen Alzheimer's drug aducanumab wins FDA approval (updated)
Jun. 07, 2021 11:02 AM ETAVXL, BIIB...By: Jonathan M Block, SA News Editor35 Comments
- The FDA has approved Biogen (NASDAQ:BIIB) and Eisai's (OTCPK:ESALF) aducanumab, the first new Alzheimer's disease drug since 2003 and the first that aims to slow disease progression.
- https://seekingalpha.com/news/3703736-biogen-alzheimers-drug-aducanumab-wins-fda-approval
- https://seekingalpha.com/symbol/BIIB
- https://www.biogen.com/en_us/pipeline.html#Aducanumab
- https://www.biogen.com/en_us/home.html
FDA grants accelerated approval for ADUHELM™ as the first and only Alzheimer’s disease treatment to address a defining pathology of the disease
JUNE 7, 2021 • INVESTOR RELATIONS
The accumulation of amyloid beta plaques in the brain is a defining pathology of Alzheimer’s disease
In clinical trials, ADUHELM reduced amyloid beta plaques by 59 to 71 percent at 18 months of treatment
CAMBRIDGE, Mass. and TOKYO, June 07, 2021 (GLOBE NEWSWIRE) -- Biogen (Nasdaq: BIIB) and Eisai, Co., Ltd. (Tokyo, Japan) today announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for ADUHELM™ (aducanumab-avwa) as the first and only Alzheimer’s disease treatment to address a defining pathology of the disease by reducing amyloid beta plaques in the brain.
The accelerated approval has been granted based on data from clinical trials demonstrating the effect of ADUHELM on reducing amyloid beta plaques, a biomarker that is reasonably likely to predict clinical benefit, in this case a reduction in clinical decline. Continued approval for ADUHELM’s indication as a treatment for Alzheimer’s disease may be contingent upon verification of clinical benefit in confirmatory trial(s).
About ADUHELM (aducanumab-avwa)
ADUHELM (aducanumab-avwa), a human monoclonal antibody, is the first and only Alzheimer’s disease treatment to address a defining pathology of the disease by reducing amyloid beta plaques in the brain. ADUHELM is indicated for the treatment of Alzheimer’s disease. This indication is granted under accelerated approval based on reduction in amyloid beta plaques in patients treated with ADUHELM. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s).
Biogen licensed ADUHELM from Neurimmune in 2007 under a collaborative development and license agreement. Since October 2017, Biogen and Eisai have collaborated on the development and commercialization of ADUHELM globally.
Epclusa® (sofosbuvir/velpatasvir)
June 10, 2021
U.S. Food and Drug Administration Approves New Formulation of Epclusa®, Expanding Pediatric Indication to Treat Children Ages 3 and Older With Chronic Hepatitis C
– New Oral Pellet Formulation Enables Treatment in Early Childhood with Pangenotypic, Panfibrotic Regimen –
FOSTER CITY, Calif.--(BUSINESS WIRE)-- Gilead Sciences, Inc. (Nasdaq: GILD) announced today that the U.S. Food and Drug Administration (FDA) has approved an expansion of the pediatric indication of Epclusa® (sofosbuvir/velpatasvir) for the treatment of chronic hepatitis C virus (HCV) to now include children as young as 3 years of age, regardless of HCV genotype or liver disease severity. The FDA approved a New Drug Application (NDA) for two strengths of an oral pellet formulation of Epclusa (sofosbuvir 200 mg/velpatasvir 50 mg and sofosbuvir 150 mg/velpatasvir 37.5 mg) developed for use by younger children who cannot swallow tablets. The recommended dosage of Epclusa in children ages 3 years and older is based on weight.
Treatment with Epclusa for 12 weeks was approved in patients without cirrhosis or with compensated cirrhosis (Child-Pugh A), and in combination with ribavirin (RBV) for patients with decompensated cirrhosis (Child-Pugh B or C). Sofosbuvir/velpatasvir is the only protease inhibitor-free, pangenotypic HCV regimen approved for patients as young as 3 years of age.
In the United States, as of 2018 there were approximately 35,300 to 60,500 children living with HCV and incidence has been on the rise. Mother-to-child transmission, the most common cause of HCV infection in children, increased 161% from 2009 to 2017, with intravenous drug use representing the primary driver of HCV infection among women of childbearing age.
The approval of Epclusa for children as young as 3 years of age is based on data from a Phase 2, open-label clinical trial that enrolled 41 children 3 years to less than 6 years of age to be treated with Epclusa for 12 weeks. At 12 weeks after treatment completion, Epclusa achieved a sustained virologic response (SVR12) or cure rate of 83% (34/41) among all patients, 88% (28/32) in children with HCV genotype 1, 50% (3/6) in children with HCV genotype 2, and 100% in children with HCV genotype 3 (2/2) and HCV genotype 4 (1/1). Of the seven patients who did not achieve cure, all discontinued treatment within one to 20 days of starting treatment.
The safety profile of Epclusa in children 3 to less than 6 years of age treated was generally consistent with that observed in clinical trials in adults. Vomiting and product use issue (spitting up the drug) were reported in 15% and 10% of subjects, respectively; these adverse reactions were mild (Grade 1 or 2) and led to treatment discontinuation in 5 (12%) subjects.
INDICATION
EPCLUSA is indicated for the treatment of adult and pediatric patients 3 years of age and older with chronic hepatitis C virus genotype 1, 2, 3, 4, 5, or 6 infection without cirrhosis or with compensated cirrhosis and in combination with ribavirin for those with decompensated cirrhosis.
U.S. Prescribing Information for Epclusa, including BOXED WARNING, is available at www.gilead.com.
Epclusa is a registered trademark 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/20210610005958/en/
FDA approves new formulation Gilead's Epclusa for children ages 3 and older with HCV
Jun. 11, 2021 2:12 AM ETGilead Sciences, Inc. (GILD)By: Mamta Mayani, SA News Editor1 Comment
- The FDA has approved an expanded use of Gilead Sciences' (NASDAQ:GILD) pediatric indication of Epclusa (sofosbuvir/velpatasvir) for chronic hepatitis C virus (HCV) to now include children as young as 3 years of age, regardless of HCV genotype or liver disease severity.
- https://seekingalpha.com/news/3705521-fda-approves-new-formulation-gileads-epclusa-for-children-ages-3-and-older-with-hcv
- https://seekingalpha.com/symbol/GILD
HyBryte™
Soligenix Receives Pediatric Investigational Plan Waiver for HyBryte™ in CTCL from the European Medicines Agency
PRINCETON, N.J., June 10, 2021 /PRNewswire/ -- Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, announced today that it has received a Pediatric Investigation Plan (PIP) waiver from the European Medicines Agency (EMA) for HyBryte™ (SGX301 or hypericin), which has recently and successfully concluded a Phase 3, pivotal clinical study for the treatment of early stage cutaneous T-cell lymphoma (CTCL).
As part of the regulatory process for the registration of new medicines with the EMA, pharmaceutical companies are required to provide a PIP outlining the Company's strategy for investigation of the new medicinal products in the pediatric population. In some instances, a waiver negating the need for a PIP for certain conditions may be granted by the EMA when development of a medicine for use in children is not feasible or appropriate, as is the case for HyBryte™ in CTCL which is extremely rare in children.
"This achievement is an important regulatory milestone as we move forward with marketing applications worldwide," stated Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. "The PIP waiver allows us to work towards advancing a marketing authorization application (MAA) with EMA in a more cost-effective manner since we will not need to expend significant time and resource to conduct a pediatric clinical study in the European Union. With the support of key patient advocacy organizations, such as the Cutaneous Lymphoma Foundation, and key opinion leaders, we are moving towards marketing approval and commercialization of HyBryte™, with the initial focus on the U.S. market followed by a MAA submission in Europe shortly thereafter."
About HyBryte™
HyBryte™ (SGX301) is a novel, first-in-class, photodynamic therapy utilizing safe, visible light for activation. The active ingredient in HyBryte™ is synthetic hypericin, a potent photosensitizer that is topically applied to skin lesions that is taken up by the malignant T-cells, and then activated by visible light 16 to 24 hours later which triggers apoptosis of the cell. The use of visible light in the red-yellow spectrum has the advantage of penetrating more deeply into the skin (much more so than ultraviolet light) and therefore potentially treating deeper skin disease and thicker plaques and lesions. This treatment approach avoids the risk of secondary malignancies (including melanoma) inherent with the frequently employed DNA-damaging drugs and other phototherapy that are dependent on ultraviolet exposure. Combined with photoactivation, hypericin has demonstrated significant anti-proliferative effects on activated normal human lymphoid cells and inhibited growth of malignant T-cells isolated from CTCL patients. In a published Phase 2 clinical study in CTCL, patients experienced a statistically significant (p=0.04) improvement with topical hypericin treatment whereas the placebo was ineffective. HyBryte™ has received orphan drug and fast track designations from the U.S. Food and Drug Administration (FDA), as well as orphan designation from the European Medicines Agency (EMA) and Promising Innovative Medicine (PIM) and "Innovation Passport" under the Innovative Licensing and Access Pathway (ILAP) from the Medicines and Healthcare Products Regulatory Agency (MHRA) of the United Kingdom.
The Phase 3 FLASH (Fluorescent Light Activated Synthetic Hypericin) trial enrolled a total of 169 patients (166 evaluable) with Stage IA, IB or IIA CTCL. The trial consisted of three treatment cycles. Treatments were administered twice weekly for the first 6 weeks and treatment response was determined at the end of the 8th week of each cycle. In the first double-blind treatment cycle, 116 patients received HyBryte™ treatment (0.25% synthetic hypericin) and 50 received placebo treatment of their index lesions. A total of 16% of the patients receiving HyBryte™ achieved at least a 50% reduction in their lesions (graded using a standard measurement of dermatologic lesions, the CAILS score) compared to only 4% of patients in the placebo group at 8 weeks (p=0.04) during the first treatment cycle (primary endpoint). HyBryte™ treatment in the first cycle was safe and well tolerated.
In the second open-label treatment cycle (Cycle 2), all patients received HyBryte™ treatment of their index lesions. Evaluation of 155 patients in this cycle (110 receiving 12 weeks of HyBryte™ treatment and 45 receiving 6 weeks of placebo treatment followed by 6 weeks of HyBryte™ treatment), demonstrated that the response rate among the 12-week treatment group was 40% (p<0.0001 vs the placebo treatment rate in Cycle 1). Comparison of the 12-week and 6-week treatment groups also revealed a statistically significant improvement (p<0.0001) between the two groups, indicating that continued treatment results in better outcomes. HyBryte™ continued to be safe and well tolerated. Additional analyses also indicated that HyBryte™ is equally effective in treating both plaque (response 42%, p<0.0001 relative to placebo treatment in Cycle 1) and patch (response 37%, p=0.0009 relative to placebo treatment in Cycle 1) lesions of CTCL, a particularly relevant finding given the historical difficulty in treating plaque lesions in particular.
The third (optional) treatment cycle (Cycle 3) was focused on safety and all patients could elect to receive HyBryte™ treatment of all their CTCL lesions. Of note, 66% of patients elected to continue with this optional compassionate use / safety cycle of the study. Of the subset of patients that received HyBryte™ throughout all 3 cycles of treatment, 49% of them demonstrated a treatment response (p<0.0001 vs patients receiving placebo in Cycle 1). Moreover, in a subset of patients evaluated in this cycle, it was demonstrated that HyBryte™ is not systemically available, consistent with the general safety of this topical product observed to date. At the end of Cycle 3, HyBryte™ continued to be well tolerated despite extended and increased use of the product to treat multiple lesions. Follow-up visits were completed in Q4 2020, and the clinical study report to support the NDA is in the process of being finalized.
Overall safety of HyBryte™ is a critical attribute of this treatment and was monitored throughout the three treatment cycles (Cycles 1, 2 and 3) and the 6-month follow-up period. Its mechanism of action is not associated with DNA damage, making it a safer alternative than currently available therapies, all of which are associated with significant and sometimes fatal, side effects. Predominantly these include the risk of melanoma and other malignancies, as well as the risk of significant skin damage and premature skin aging. Currently available treatments are only approved in the context of previous treatment failure with other modalities and there is no approved front-line therapy available. Within this landscape, treatment of CTCL is strongly motivated by the safety risk of each product. HyBryte™ potentially represents the safest available efficacious treatment for CTCL. With no systemic absorption, a compound that is not mutagenic and a light source that is not carcinogenic, there is no evidence to date of any potential safety issues.
The Phase 3 CTCL clinical study was partially funded by the National Cancer Institute via a Phase II SBIR grant (#1R44CA210848-01A1) awarded to Soligenix, Inc.
For further information regarding Soligenix, Inc., please visit the Company's website at https://www.soligenix.com.
Soligenix soars 13% after securing PIP waiver for HyBryte in blood cancer in Europe
Jun. 10, 2021 8:00 AM ETSoligenix, Inc. (SNGX)By: Mamta Mayani, SA News Editor
- Soligenix (NASDAQ:SNGX) jumps 13% premarket after receiving a Pediatric Investigation Plan (PIP) waiver from the EMA for HyBryte (SGX301 or hypericin), which has recently and successfully concluded a Phase 3 study for the treatment of early stage cutaneous T-cell lymphoma (CTCL).
- https://seekingalpha.com/news/3704944-soligenix-soars-13-after-securing-pip-waiver-for-hybryte-in-blood-cancer-in-europe
- https://seekingalpha.com/symbol/SNGX
- https://www.soligenix.com/
KEYTRUDA® (pembrolizumab) Plus LENVIMA® (lenvatinib)
New Data on KEYTRUDA® (pembrolizumab) Plus LENVIMA® (lenvatinib) Versus Sunitinib in First-Line Treatment for Patients With Advanced Renal Cell Carcinoma From Pivotal Phase 3 CLEAR/KEYNOTE-581 Trial Presented at 2021 ASCO Annual Meeting
June 7, 2021 6:45 am ET
Results From New Analysis Evaluating Health-Related Quality of Life (HRQoL) Based on Patient-Reported Outcomes Using Three HRQoL Scales
KENILWORTH, N.J., & WOODCLIFF LAKE, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, and Eisai Inc. today announced new investigational data from the pivotal Phase 3 CLEAR (Study 307)/KEYNOTE-581 trial, which evaluated the combinations of KEYTRUDA, Merck’s anti-PD-1 therapy, plus LENVIMA, the orally available multiple receptor tyrosine kinase inhibitor discovered by Eisai, and LENVIMA plus everolimus versus sunitinib for the first-line treatment of patients with advanced renal cell carcinoma (RCC). Results from a new analysis evaluating health-related quality of life (HRQoL) based on patient-reported outcomes are being presented during an oral abstract session at the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting (Abstract #4502). Data from CLEAR/KEYTNOTE-581 were originally presented at the 2021 Genitourinary Cancers Symposium (ASCO GU) and published in the New England Journal of Medicine, and data from this trial are currently under review with the U.S. Food and Drug Administration (FDA).
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210607005121/en/
“This new analysis expands our understanding of the results we’ve seen from the CLEAR/KEYNOTE-581 trial in the treatment of patients with advanced renal cell carcinoma,” said Dr. Robert Motzer, Medical Oncologist, Kidney Cancer Section Head, Genitourinary Oncology Service, Memorial Sloan Kettering Cancer Center. “The additional data showed an improvement of specific health-related quality of life measures for patients who received KEYTRUDA plus LENVIMA compared with sunitinib, supporting the importance of this combination as a potential new first-line treatment option for patients.”
Data From Health‐Related Quality of Life (HRQoL) Analysis From CLEAR/KEYNOTE-581
In an analysis of a secondary endpoint of HRQoL scores in the CLEAR/KEYNOTE-581 trial, KEYTRUDA plus LENVIMA and LENVIMA plus everolimus were evaluated to determine the impact on HRQoL compared to sunitinib in patients with advanced RCC. This was assessed based on patient-reported outcomes using three HRQoL and symptom measures: Functional Assessment of Cancer Therapy Kidney Symptom Index – Disease-Related Symptoms (FKSI-DRS), European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire for Patients With Cancer – Core 30 (EORTC QLQ-C30) and European Quality of Life Five-Dimensions – 3-Level System (EuroQoL EQ-5D-3L). Unless otherwise noted, HRQoL analyses were based on data from randomized patients who received at least one dose of study treatment. No adjustments for multiple testing or estimation were used; p-values (two-sided) and confidence intervals (CI) are nominal and descriptive. Longitudinal change from baseline was assessed by mixed model analysis. Least squares mean differences (LSMD) and 95% CI were calculated from baseline. Time to deterioration (based on changes in HRQoL and disease-related symptom scores ≥ meaningful thresholds) was assessed using time to first deterioration (TTD), which is the number of weeks between randomization and the first deterioration event, and time until definitive deterioration (TUDD), which is the number of weeks between randomization and the earliest deterioration event with no subsequent recovery above the deterioration threshold or no subsequent HRQoL assessment data. All times to deterioration were calculated and compared using the Kaplan-Meier method, stratified log-rank tests and Cox models.
KEYTRUDA plus LENVIMA demonstrated similar changes from baseline at mean follow-up (Week 46) on 14 out of 18 HRQoL and disease-related symptom scores and better HRQoL and disease-related symptom scores for the following measures (LSMD [95% CI]): physical functioning (3.01 [0.48, 5.54]), fatigue (-2.80 [-5.52, -0.08]), dyspnea (-2.79 [-5.33, -0.25]) and constipation (-2.19 [-4.19, -0.18]), as measured by the QLQ-C30, versus sunitinib. LENVIMA plus everolimus demonstrated similar changes from baseline at mean follow-up (Week 46) on 14 out of 18 HRQoL and disease-related symptom scores and worse HRQoL and disease-related symptom scores in the following measures (LSMD [95% CI]): Global Health Score/QoL (-2.81 [-5.08, -0.54]), pain (2.80 [0.11, 5.49]), appetite loss (4.23 [1.34, 7.13]) and diarrhea (5.26 [2.61, 7.91]) compared to sunitinib.
KEYTRUDA plus LENVIMA demonstrated a similar TTD in 14 out of 18 HRQoL and disease-related symptom scores, and a delay in TTD for physical functioning, dyspnea, appetite loss, and EQ-5D visual analog scale compared to sunitinib. KEYTRUDA plus LENVIMA demonstrated a delay in TUDD in 16 out of 18 HRQoL and disease-related symptom scores and a similar TUDD for cognitive functioning and financial difficulties compared to sunitinib.
Dr. Motzer has provided consulting and advisory services for Merck and Eisai.
About CLEAR (Study 307)/KEYNOTE-581
The CLEAR/KEYNOTE-581 trial is a multicenter, randomized, open-label, Phase 3 trial (ClinicalTrials.gov, NCT02811861) evaluating LENVIMA in combination with KEYTRUDA or in combination with everolimus versus sunitinib for the first-line treatment of patients with advanced RCC. The primary endpoint is progression-free survival, as assessed by independent review per RECIST v1.1. Secondary endpoints include overall survival, objective response rate, HRQoL and safety. A total of 1,069 patients were randomized (1:1:1) to receive:
- LENVIMA (20 mg orally once daily) in combination with KEYTRUDA (200 mg intravenously [IV] every three weeks for up to 24 months); or
- LENVIMA (18 mg orally once daily) in combination with everolimus (5 mg orally once daily); or
- Sunitinib (50 mg orally once daily for four weeks on treatment, followed by two weeks off treatment).
Treatment continued until unacceptable toxicity or disease progression as determined by the investigator and confirmed by independent radiologic review committee using RECIST v1.1. Administration of KEYTRUDA plus LENVIMA was permitted beyond RECIST-defined disease progression if the patient was clinically stable and considered by the investigator to be deriving clinical benefit. KEYTRUDA was continued for a maximum of 24 months; however, treatment with LENVIMA could be continued beyond 24 months. Assessment of tumor status was performed at baseline and then every eight weeks.
About KEYTRUDA® (pembrolizumab) Injection, 100 mg
KEYTRUDA is an anti-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 1,400 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.
Selected KEYTRUDA® (pembrolizumab) Indications in the U.S.
Melanoma
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.
KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.
Non-Small Cell Lung Cancer
KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) ≥1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is stage III where patients are not candidates for surgical resection or definitive chemoradiation, or metastatic.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS ≥1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.
Head and Neck Squamous Cell Cancer
KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS) ≥1] as determined by an FDA-approved test.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.
Classical Hodgkin Lymphoma
KEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).
KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.
Primary Mediastinal Large B-Cell Lymphoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.
Urothelial Carcinoma
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 (CPS ≥10), as determined by an FDA-approved test, or in patients who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.
Microsatellite Instability-High or Mismatch Repair Deficient Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR)
- solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options, or
- colorectal cancer that has progressed following treatment with fluoropyrimidine, oxaliplatin, and irinotecan.
This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.
Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer
KEYTRUDA is indicated for the first-line treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).
Gastric Cancer
KEYTRUDA, in combination with trastuzumab, and fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of patients with locally advanced unresectable or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent locally advanced or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test, with disease progression on or after two or more prior lines of therapy including fluoropyrimidine- and platinum-containing chemotherapy and if appropriate, HER2/neu-targeted therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Esophageal Cancer
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic esophageal or gastroesophageal junction (GEJ) (tumors with epicenter 1 to 5 centimeters above the GEJ) carcinoma that is not amenable to surgical resection or definitive chemoradiation either:
- in combination with platinum- and fluoropyrimidine-based chemotherapy, or
- as a single agent after one or more prior lines of systemic therapy for patients with tumors of squamous cell histology that express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
Cervical Cancer
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Hepatocellular Carcinoma
KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Merkel Cell Carcinoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Renal Cell Carcinoma
KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).
Endometrial Carcinoma
KEYTRUDA, in combination with LENVIMA, is indicated for the treatment of patients with advanced endometrial carcinoma that is not MSI-H or dMMR, who have disease progression following prior systemic therapy and are not candidates for curative surgery or radiation. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trial.
Tumor Mutational Burden-High
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [≥10 mutations/megabase] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.
Cutaneous Squamous Cell Carcinoma
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) that is not curable by surgery or radiation.
Triple-Negative Breast Cancer
KEYTRUDA, in combination with chemotherapy, is indicated for the treatment of patients with locally recurrent unresectable or metastatic triple-negative breast cancer (TNBC) whose tumors express PD-L1 (CPS ≥10) as determined by an FDA-approved test. This indication is approved under accelerated approval based on progression-free survival. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Please see Prescribing Information for KEYTRUDA (pembrolizumab) at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_pi.pdf and Medication Guide for KEYTRUDA at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_mg.pdf.
About LENVIMA® (lenvatinib) Capsules
LENVIMA® (lenvatinib) is a kinase inhibitor that is indicated:
- For the treatment of patients with locally recurrent or metastatic, progressive, radioactive iodine-refractory differentiated thyroid cancer (DTC)
- In combination with everolimus, for the treatment of patients with advanced renal cell carcinoma (RCC) following one prior anti-angiogenic therapy
- For the first-line treatment of patients with unresectable hepatocellular carcinoma (HCC)
- In combination with KEYTRUDA, for the treatment of patients with advanced endometrial carcinoma (EC) that is not microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR), who have disease progression following prior systemic therapy, and are not candidates for curative surgery or radiation. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trial
LENVIMA, discovered and developed by Eisai, is a kinase inhibitor that inhibits the kinase activities of vascular endothelial growth factor (VEGF) receptors VEGFR1 (FLT1), VEGFR2 (KDR), and VEGFR3 (FLT4). LENVIMA inhibits other kinases that have been implicated in pathogenic angiogenesis, tumor growth, and cancer progression in addition to their normal cellular functions, including fibroblast growth factor (FGF) receptors FGFR1-4, platelet derived growth factor receptor alpha (PDGFRα), KIT, and RET. Lenvatinib also exhibited antiproliferative activity in hepatocellular carcinoma cell lines dependent on activated FGFR signaling with a concurrent inhibition of FGD-receptor substrate 2 alpha (FRS2α) phosphorylation. The combination of lenvatinib and everolimus showed increased anti-angiogenic and anti-tumor activity as demonstrated by decreases in human endothelial cell proliferation, tube formation, and VEGF signaling in vitro and decreases in tumor volume in mouse xenograft models of human renal cell cancer greater than those with either drug alone. In syngeneic mouse tumor models, lenvatinib decreased tumor-associated macrophages, increased activated cytotoxic T cells, and demonstrated greater antitumor activity in combination with an anti-PD-1 monoclonal antibody compared to either treatment alone.
Please see Prescribing Information for LENVIMA (lenvatinib) at http://www.lenvima.com/pdfs/prescribing-information.pdf.
About the Merck and Eisai Strategic Collaboration
In March 2018, Eisai and Merck, known as MSD outside the United States and Canada, through an affiliate, entered into a strategic collaboration for the worldwide co-development and co-commercialization of LENVIMA. Under the agreement, the companies will jointly develop, manufacture and commercialize LENVIMA, both as monotherapy and in combination with Merck’s anti-PD-1 therapy KEYTRUDA.
In addition to ongoing clinical studies evaluating the KEYTRUDA plus LENVIMA combination across several different tumor types, the companies have jointly initiated new clinical studies through the LEAP (LEnvatinib And Pembrolizumab) clinical program and are evaluating the combination in 14 different tumor types (endometrial carcinoma, hepatocellular carcinoma, melanoma, non-small cell lung cancer, renal cell carcinoma, squamous cell carcinoma of the head and neck, urothelial cancer, biliary tract cancer, colorectal cancer, gastric cancer, glioblastoma, ovarian cancer, pancreatic cancer and triple-negative breast cancer) across more than 20 clinical trials.
For more information about our oncology clinical trials, visit www.merck.com/clinicaltrials.
For more information, visit www.merck.com
For more information about Eisai, please visit www.eisai.com (for global), us.eisai.com (for U.S.) or www.eisai.eu (for Europe, Middle East, Africa),
Merck, Eisai report new Keytruda/Lenvima data in kidney cancer at ASCO21
Jun. 07, 2021 7:38 AM ETMerck & Co., Inc. (MRK), ESALFBy: Mamta Mayani, SA News Editor
- Merck (NYSE:MRK) and Eisai (OTCPK:ESALF) announce new investigational data from the Phase 3 CLEAR (Study 307)/KEYNOTE-581 trial, evaluating the combinations of Keytruda, plus Eisai's Lenvima, and Lenvima plus everolimus vs. sunitinib for the first-line treatment of patients with advanced renal cell carcinoma (RCC).
- https://seekingalpha.com/news/3703597-merck-eisai-report-new-keytrudalenvima-data-in-kidney-cancer-at-asco21
- https://seekingalpha.com/symbol/ESALF
- https://seekingalpha.com/symbol/MRK
What is LENVIMA?
LENVIMA is a prescription medicine that is used to treat certain kinds of cancer.
- LENVIMA is used by itself to treat differentiated thyroid cancer (DTC), a type of thyroid cancer that can no longer be treated with radioactive iodine and is progressing
- LENVIMA is used along with another medicine called everolimus to treat advanced renal cell carcinoma (RCC), a type of kidney cancer, after one course of treatment with another anti-cancer medicine
- LENVIMA is used by itself as the first treatment for a type of liver cancer called hepatocellular carcinoma (HCC) when it cannot be removed by surgery
It is not known if LENVIMA is safe and effective in children.
SPINRAZA® (nusinersen)
New Data at Cure SMA 2021 Highlight the Long-Term Efficacy of SPINRAZA® (nusinersen) and Biogen’s Commitment to Innovation in SMA Therapy
JUNE 10, 2021 • INVESTOR RELATIONS
- New data analysis suggests an investigational higher dose of SPINRAZA may lead to clinically meaningful improvements in motor function
- A NURTURE study analysis shows 92 percent of children who initiated SPINRAZA treatment as pre-symptomatic infants maintained the ability to swallow after approximately 4 years
- Among children and teens with later-onset SMA in the SHINE study, long-term treatment with SPINRAZA improved walking distance and reduced fatigue
CAMBRIDGE, Mass., June 10, 2021 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) today announced new research supporting the continued development of an investigational higher dose of SPINRAZA® (nusinersen) and additional data reinforcing the strength of SPINRAZA’s clinical profile in improving the lives of individuals with spinal muscular atrophy (SMA) over the long term. These data are being presented at the virtual Cure SMA Research & Clinical Care Meeting taking place June 9-11, 2021.
“Intervention with SPINRAZA can meaningfully impact the trajectory of SMA, and we remain relentless in our aim of improving outcomes for people with SMA. We continue to better understand and explore SPINRAZA’s potential with our new and ongoing global clinical studies,” said Alfred Sandrock, Jr., M.D., Ph.D., Head of Research and Development at Biogen. “The data we are presenting at Cure SMA 2021 demonstrate the long-term benefits with SPINRAZA as individuals age. Additionally, a new analysis provides further support for the potential for a higher dose of SPINRAZA to offer even greater improvements in motor function for SMA patients.”
As previously reported,1 safety data from Part A (n=6; 28 mg) support continued development of a higher dose of SPINRAZA and an updated analysis presented at the meeting include data collected up to 10 months. Enrollment in the pivotal Part B of DEVOTE is ongoing and will evaluate a higher-dose regimen (two loading doses of 50 mg 14 days apart followed by 28 mg maintenance doses every four months) compared to the currently approved dosing regimen for SPINRAZA.2 More information about DEVOTE is available at ClinicalTrials.gov (NCT04089566).
New Findings Support Continued Development of an Investigational Higher Dose of SPINRAZA
An analysis of data from the Phase 2 CS3A and Phase 3 ENDEAR studies in children with infantile-onset SMA used pharmacokinetic (PK)/pharmacodynamic (PD) modelling to predict the potential efficacy of an investigational higher dose regimen of SPINRAZA. This analysis suggests that a higher dose of SPINRAZA may lead to a clinically meaningful increase in the CHOP INTEND (Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders) score beyond that already observed with the 12 mg approved dose. These findings further reinforce the scientific rationale for the evaluation of a higher dose of SPINRAZA in the ongoing DEVOTE study.
DEVOTE is a Phase 2/3 study evaluating the safety, tolerability and potential for even greater efficacy of SPINRAZA when administered at a higher dose than currently approved. It is a three-part study that includes an open-label safety evaluation cohort (Part A), a pivotal, double-blind, randomized, active-controlled treatment cohort (Part B) and an open-label cohort of patients transitioning from the approved 12 mg dosing regimen of SPINRAZA to the higher dose regimen (Part C).
Data Reinforce Long-term Efficacy of SPINRAZA in a Broad Range of People with SMA
An analysis of data from the NURTURE study (n=25) shows 92 percent of patients who initiated SPINRAZA treatment as pre-symptomatic infants maintained the ability to swallow after a median of 3.8 years. This is in contrast with the natural history of SMA where impaired swallowing is expected for people with 2 or 3 SMN2 copies3 and can lead to an increased risk of aspiration pneumonia, choking and failure to thrive. In this analysis, NURTURE study participants were consistently rated by their caregiver as, on average, never to rarely experiencing difficulty for the majority of measures related to general feeding, drinking liquids and eating solid foods. Additionally, all participants with 3 SMN2 copies and 73 percent (11 of 15) of participants with 2 SMN2 copies were reported by their caregiver as being fed exclusively by mouth.
In addition, post-hoc data from the open-label CS2-CS12 and SHINE extension studies indicate children and teens with later-onset SMA (n=14) showed improvement in walking distance over five years of SPINRAZA treatment and stabilization in fatigue.
In all analyses presented at the meeting, the safety profile of SPINRAZA was consistent with previously reported findings.
Featured SPINRAZA Data Presentations Include:
- Scientific Rationale for a Higher Dose of Nusinersen
- Part A Results From the Ongoing DEVOTE Study to Explore a Higher Dose of Nusinersen in SMA
- Preserved Swallowing Function in Infants Who Initiated Nusinersen Treatment in the Presymptomatic Stage of SMA: Results From the NURTURE Study
- Nusinersen in Later-onset Spinal Muscular Atrophy: Walking Distance and Fatigue in CS2/12 and SHINE Participants
About SPINRAZA® (nusinersen)
SPINRAZA is approved to treat infants, children and adults with spinal muscular atrophy (SMA) and is available in more than 50 countries. As a foundation of care in SMA, more than 11,000 individuals have been treated with SPINRAZA worldwide.4
SPINRAZA is an antisense oligonucleotide (ASO) that targets the root cause of SMA by continuously increasing the amount of full-length survival motor neuron (SMN) protein produced in the body.5 It is administered directly into the central nervous system, where motor neurons reside, to deliver treatment where the disease starts.5
SPINRAZA has demonstrated sustained efficacy across ages and SMA types with a well-established safety profile based on data in patients treated up to 7 years, combined with unsurpassed real-world experience.6 The SPINRAZA clinical development program encompasses 10 clinical studies, which have included more than 300 individuals across a broad spectrum of patient populations,6 including two randomized controlled studies (ENDEAR and CHERISH). The ongoing SHINE and NURTURE open-label extension studies are evaluating the long-term impact of SPINRAZA. The most common adverse events observed in clinical studies were respiratory infection, fever, constipation, headache, vomiting and back pain. Laboratory tests can monitor for renal toxicity and coagulation abnormalities, including acute severe low platelet counts, which have been observed after administration of some ASOs.
Biogen licensed the global rights to develop, manufacture and commercialize SPINRAZA from Ionis Pharmaceuticals, Inc. (Nasdaq: IONS), the leader in antisense therapeutics. Please click here for Important Safety Information and full Prescribing Information for SPINRAZA in the U.S., or visit your respective country’s product website.
We routinely post information that may be important to investors on our website at www.biogen.com.
Data suggests higher dose of Biogen Spinraza may better improve motor function
Jun. 10, 2021 8:23 AM ET
By: Jonathan M Block, SA News Editor1 Comment
- A new data analysis suggests that a higher dose of Biogen's (NASDAQ:BIIB) Spinraza (nusinersen) can lead to even better improvement in motor function that the currently approved dose.
- https://seekingalpha.com/news/3704962-data-suggests-higher-dose-of-biogen-spinraza-may-better-improve-motor-function
- https://seekingalpha.com/symbol/BIIB
- https://www.biogen.com/
- https://www.spinraza.com/
TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor
BackPDF VersionJun 9, 2021
Vertex Announces U.S. FDA Approval for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in Children With Cystic Fibrosis Ages 6 through 11 With Certain Mutations
- With this approval approximately 1,500 children with one minimal function mutation and one F508del mutation have a medicine to treat the underlying cause of their disease for the first time -
BOSTON--(BUSINESS WIRE)--Jun. 9, 2021-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) approved expanded use of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include children with cystic fibrosis (CF) ages 6 through 11 years who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that is responsive to TRIKAFTA based on in vitro data. TRIKAFTA was previously approved by the FDA for use in people with cystic fibrosis 12 years and older with at least one copy of the F508del mutation or one copy of a mutation that is responsive in vitro. An additional dosage strength of TRIKAFTA tablets is now available (elexacaftor 50 mg/tezacaftor 25 mg/ivacaftor 75 mg and ivacaftor 75 mg) in connection with this approval.
“Today’s approval is a critical milestone in our efforts to deliver medicines that help treat the underlying cause of this devastating disease as early in life as possible,” said Reshma Kewalramani, M.D., Chief Executive Officer and President, Vertex. “We can now reach approximately 1,500 newly eligible children in the U.S., and we continue to pursue approval for this expanded indication in other countries.”
Vertex completed a 24-week Phase 3 open-label, multicenter study which enrolled 66 children ages 6 through 11 years old with cystic fibrosis (CF) who have either two copies of the F508del mutation or one copy of the F508del mutation and one minimal function mutation to evaluate the safety, pharmacokinetics and efficacy of TRIKAFTA. The regimen was generally well tolerated, and safety data were similar with those observed in previous studies of patients ages 12 years and older. The full data from this study were recently published in American Journal of Respiratory and Critical Care Medicine.
TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor
“Clinical experience with TRIKAFTA in patients 12 and older over the past 20 months has demonstrated this medicine has a meaningful and unprecedented clinical benefit for patients. I look forward to now being able to treat younger patients with this breakthrough medicine, including those who have not presented major signals of disease progression,” said Terri Laguna M.D., M.S.C.S., Associate Director of the Cystic Fibrosis Center and Division Head, Pulmonary and Sleep Medicine, Ann & Robert H. Lurie Children’s Hospital of Chicago. “In addition to bringing TRIKAFTA to a younger patient population, patients not previously eligible for any CFTR modulator will now be able to access a treatment that targets the underlying cause of their disease.”
TRIKAFTA® is already approved for the treatment of patients with CF ages 12 years and older with certain mutations in the U.S. Switzerland, Australia and Israel, as well as in the EU and the U.K. as KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with KALYDECO® (ivacaftor). Vertex has submitted applications for use of TRIKAFTA/KAFTRIO in children ages 6 through 11 years to the European Medicines Agency (EMA) and the Medicines & Healthcare products Regulatory Agency (MHRA) and plans to file for this expanded use in Switzerland, Australia and Israel this year.
For more information on TRIKAFTA, patient assistance programs or to find additional eligibility details, visit TRIKAFTA.com, VertexGPS.com or vertextreatments.com.
INDICATION AND IMPORTANT SAFETY INFORMATION FOR TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) TABLETS
What is TRIKAFTA?
TRIKAFTA is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or another mutation that is responsive to treatment with TRIKAFTA. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if TRIKAFTA is safe and effective in children under 6 years of age.
These are not all the possible side effects of TRIKAFTA. Please click the product link to see the full Prescribing Information for TRIKAFTA.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210609005466/en/
Vertex Pharmaceuticals Incorporated
FDA OKs expanded use of Vertex's Trikafta in children with cystic fibrosis
Jun. 09, 2021 9:47 AM ET
Vertex Pharmaceuticals Incorporated (VRTX)
By: Mamta Mayani, SA News Editor3 Comments
- The FDA has approved expanded use of Vertex Pharmaceuticals' (VRTX +1.4%) Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include children with cystic fibrosis (CF) ages 6 through 11 years with certain mutations.
- https://seekingalpha.com/news/3704593-fda-oks-expanded-use-of-vertexs-trikafta-in-children-with-cystic-fibrosis
- https://seekingalpha.com/symbol/VRTX
- https://www.vrtx.com/
- https://www.trikafta.com/
LUMAKRAS™ (sotorasib)
Results From Phase 2 CodeBreaK 100 Show LUMAKRAS™ (sotorasib) Is The First And Only KRAS G12C Inhibitor With Overall Survival Data
LUMAKRAS Shows Median Overall Survival of 12.5 Months in Patients With Previously Treated Non-Small Cell Lung Cancer
Data Confirms Rapid, Deep, and Durable Responses With Median Duration of Response of 11.1 Months
Data Presented at ASCO 2021 and Simultaneously Published in New England Journal of Medicine
THOUSAND OAKS, Calif., June 4, 2021 /PRNewswire/ -- Amgen (NASDAQ: AMGN) today presented data on overall survival, a secondary endpoint, from the Phase 2 results of the CodeBreaK 100 clinical study for LUMAKRASTM (sotorasib) in previously treated patients with non-small cell lung cancer (NSCLC) during the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting. These data were also simultaneously published in the New England Journal of Medicine (NEJM). The publication includes mature overall survival and duration of response data, and results from subgroup and exploratory biomarker analyses.
LUMAKRAS shows a median overall survival (OS) of 12.5 months among 124 evaluable patients, the majority of which were previously treated with both platinum-based chemotherapy and immunotherapy (81%) (data cutoff of March 15, 2021). The results confirmed an objective response rate (ORR) of 37.1%, duration of response (DoR) of 11.1 months and disease control rate (DCR) of 80.6%, with an additional patient achieving complete response (bringing the total to four complete responses and 42 partial responses) compared to previously reported results. The data published in NEJM are updated from results presented at the International Association for the Study of Lung Cancer (IASLC) 2020 World Conference on Lung Cancer (WCLC) held in January 2021 and are based on a longer follow-up time of 15.3 months.
"Sotorasib is the first KRASG12C inhibitor to show an overall survival benefit, and the data represent a major step forward for patients with KRAS G12C-mutated non-small cell lung cancer where standard of care options are suboptimal," said lead author Ferdinandos Skoulidis, M.D., Ph.D., assistant professor of Thoracic/Head and Neck Medical Oncology at The University of Texas MD Anderson Cancer Center. "KRAS has been one of the most challenging therapeutic targets in cancer research, and these practice-changing results give hope to patients with the KRAS G12C mutation who previously had no targeted treatment options."
LUMAKRAS received approval from the U.S. FDA on May 28, 2021, for the treatment of adult patients with KRAS G12C-mutated locally advanced or metastatic NSCLC, as determined by an FDA-approved test, who have received at least one prior systemic therapy. LUMAKRAS has received accelerated approval based on ORR and DoR. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
Skoulidis reports research support from Amgen Inc.
About LUMAKRASTM (sotorasib)
Amgen has taken on one of the toughest challenges of the last 40 years in cancer research by developing LUMAKRAS, a KRASG12C inhibitor.1 LUMAKRAS was the first KRASG12C inhibitor to enter the clinic and is being studied in the largest clinical program exploring more than 10 combinations with global investigator sites spanning five continents.
LUMAKRAS has demonstrated a positive benefit-risk profile with rapid, deep and durable anticancer activity in patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring the KRAS G12C mutation with a once daily oral formulation. As part of the evaluation for this accelerated approval, FDA is requiring a post-marketing trial to investigate whether a lower dose will have a similar clinical effect.
LUMAKRAS is also being studied in multiple other solid tumors.1
LUMAKRAS was granted Breakthrough Therapy designation in the U.S. and China. In the U.S., LUMAKRAS was reviewed by the FDA under its Real-Time Oncology Review (RTOR), a pilot program that aims to explore a more efficient review process that ensures safe and effective treatments are made available to patients as early as possible. Amgen is participating in the FDA's Project Orbis initiative and through the initiative, has submitted Marketing Authorization Applications (MAAs) for sotorasib in Australia, Brazil, Canada and the United Kingdom. Additionally, Amgen has submitted an MAA in the EU and New Drug Applications in Japan (J-NDA), Switzerland, South Korea and United Arab Emirates.
LUMAKRASTM (sotorasib) U.S. Indication
LUMAKRASTM is indicated for the treatment of adult patients with KRAS G12C-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC), as determined by an FDA-approved test, who have received at least one prior systemic therapy.
This indication is approved under accelerated approval based on overall response rate (ORR) and duration of response (DOR). Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
LUMAKRAS™ (sotorasib) Important Safety Information
Please see LUMAKRASTM full Prescribing Information.
For information, please visit www.hcp.codebreaktrials.com.
For more information, follow us on www.twitter.com/amgenoncology.
For more information, visit www.amgen.com
View original content to download multimedia:http://www.prnewswire.com/news-releases/results-from-phase-2-codebreak-100-show-lumakras-sotorasib-is-the-first-and-only-kras-g12c-inhibitor-with-overall-survival-data-301306186.html
SOURCE Amgen
Data supports overall response, durable survival for Amgen's Lumakras in lung cancer
Jun. 04, 2021 5:01 PM ET Amgen Inc. (AMGN) By: Jonathan M Block, SA News Editor6 Comments
- In 124 patients with non-small cell lung cancer ("NSCLC"), Amgen's (NASDAQ:AMGN) KRAS inhibitor Lumakras (sotorasib) demonstrated 12.4 months of median survival and a duration of response of 11.1 months, according to new phase 2 results.
- https://seekingalpha.com/news/3703480-data-supports-overall-response-durable-survival-for-amgen-miratis-lumakras-in-lung-cancer
- https://seekingalpha.com/symbol/AMGN
- https://www.amgen.com/
RINVOQ® (upadacitinib)
Health Canada Approves AbbVie's RINVOQ® (upadacitinib) for the Treatment of Adults with Active Psoriatic Arthritis
Jun. 07, 2021 7:01 AM ETCanada Newswire
- Approval supported by efficacy and safety data of two pivotal Phase 3 studies in which RINVOQ demonstrated improved joint outcomes, physical function and skin symptoms, with a greater proportion of patients achieving minimal disease activity versus placebo* 1,2
- Significantly more patients taking RINVOQ achieved an ACR20 response than patients receiving placebo 1,2
MONTREAL, June 7, 2021 /CNW/ - AbbVie (ABBV), a research-based global biopharmaceutical company, today announced that Health Canada has approved RINVOQ® (upadacitinib, 15 mg), an oral, once-daily selective and reversible JAK inhibitor for the treatment of adults with active psoriatic arthritis (PsA) who have had an inadequate response or intolerance to methotrexate or other Disease-Modifying Anti-Rheumatic Drugs (DMARDs).3
For more information about AbbVie, please visit us at www.abbvie.ca.
About SELECT-PsA 1 1,3
SELECT-PsA 1 is a Phase 3, multicenter, randomized, double-blind, parallel-group, active and placebo-controlled study designed to evaluate the safety and efficacy of upadacitinib compared to placebo and adalimumab in adult patients with active psoriatic arthritis who have a history of inadequate response to at least one non-biologic DMARD.
Top-line results from SELECT-PsA 1 were previously announced in February 2020. More information on this trial can be found at www.clinicaltrials.gov (NCT03104400).
About SELECT-PsA 2 2,3
SELECT-PsA 2 is a Phase 3, multicenter, randomized, double-blind, parallel-group, placebo-controlled study designed to evaluate the safety and efficacy of RINVOQ in adult patients with active psoriatic arthritis who have a history of inadequate response to at least one biologic DMARD.
Top-line results from SELECT-PsA 2 were previously announced in October 2019. More information on this trial can be found at www.clinicaltrials.gov (NCT03104374).
About RINVOQ® (upadacitinib)
RINVOQ is a 15 mg, once-daily oral medication in an extended-release tablet. It is a Janus kinase (JAK) inhibitor that interferes with the JAK-STAT signaling pathway, which is thought to play a role in inflammatory response.
RINVOQ is indicated for the treatment of adults with moderately to severely active rheumatoid arthritis who have had an inadequate response or intolerance to methotrexate, as well as for adults with active psoriatic arthritis who have had an inadequate response or intolerance to methotrexate or other DMARDs. In RA, RINVOQ may be used as a monotherapy or in combination with methotrexate or other nonbiologic DMARDs. In PsA, RINVOQ may be used as a monotherapy or in combination with methotrexate.
For important safety information, please consult the RINVOQ Product Monograph at www.abbvie.ca.
SOURCE AbbVie Canada
https://seekingalpha.com/symbol/ABBV
tislelizumab
Novartis investigational checkpoint inhibitor tislelizumab met primary endpoint of overall survival in pivotal Phase III trial of esophageal cancer after systemic therapy
Jun 04, 2021
- Tislelizumab demonstrated a 30% reduction in the risk of death and extended median overall survival by 2.3 months compared to chemotherapy in advanced or metastatic esophageal squamous cell carcinoma after prior systemic therapy1
- Additional Phase II data presented at ASCO showed tislelizumab demonstrated durable anti-tumor activity in patients with microsatellite instability-high, or mismatch-repair-deficient, solid tumors2
Basel, June 4, 2021 — Novartis announced today results from the pivotal Phase III RATIONALE 302 trial showing the investigational anti-PD-1 immune checkpoint inhibitor tislelizumab improved overall survival (OS) versus chemotherapy (median 8.6 months vs. 6.3 months, p=0.0001).1 The study evaluated tislelizumab in patients with unresectable recurrent locally advanced or metastatic esophageal squamous cell carcinoma (ESCC) who had received prior systemic therapy. Results were presented at the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting.
Results from RATIONALE 302 in ESCC showed tislelizumab extended median OS by 2.3 months compared to chemotherapy with a 30% reduction in the risk of death (HR=0.70, 95% CI: 0.57-0.85, p=0.0001).1 In PD-L1 positive patients, tislelizumab extended median OS by 3.5 months with a 46% reduction in the risk of death (HR=0.54, 95% CI: 0.36-0.79, p=0.0006).1
RATIONALE 302 is a randomized, global Phase III study assessing tislelizumab versus chemotherapy in patients with advanced unresectable/metastatic ESCC after prior systemic therapy. The primary endpoint is OS in the intent-to-treat (ITT) population. The key secondary endpoint is OS in PD-L1 positive patients (vCPS ≥10%). Additional secondary endpoints included PFS, ORR, DoR and safety endpoints.1
Data on tislelizumab in MSI-H cancers presented
The RATIONALE 209 study reported that tislelizumab showed durable anti-tumor activity in patients with previously treated, locally advanced, unresectable or metastatic microsatellite instability-high (MSI-H) and mismatch repair deficient (dMMR) cancers, which are known to be more responsive to immune checkpoint modulation. Treatment with tislelizumab demonstrated an ORR of 45.9% among all tumor types, including four complete responses (CR) and 30 partial responses (PR). No disease progression was reported in the 34 responders (CR + PR), with a 12-month DoR rate of 100%).2
Five percent of patients treated with tislelizumab discontinued treatment due to TRAEs, and no new safety signals were identified. Grade ≥3 TRAEs occurred in 42.5% of patients.2
MSI-H cancer cells have a defect in the ability to correct mistakes that occur when DNA is copied, leading to mutations that contribute to cancerous growth. Many types of cancer may have a high level of microsatellite instability, but it is seen most often in CRC, gastric cancer and endometrial cancer.6
RATIONALE 209 is a single-arm, open-label Phase II study evaluating the efficacy and safety of tislelizumab monotherapy in adult patients with previously treated, locally advanced, unresectable or metastatic histologically confirmed MSI-H/dMMR solid tumors. Radiological imaging was performed at nine weeks, then every six weeks for the first year of therapy and every 12 weeks thereafter. The primary endpoint was IRC-assessed ORR. Secondary endpoints included time to response, DoR, disease control rate, PFS, OS and safety.2
Visit https://www.hcp.novartis.com/virtual-congress/a-2021/ for the latest information from Novartis, including our commitment to the Oncology community, and access to our ASCO21 Virtual Scientific Program data presentations (for registered participants).
About tislelizumab
Tislelizumab was specifically engineered to minimize binding to macrophage Fcγ receptors, a potential mechanism of anti–PD-1 resistance.7 Tislelizumab is an important component of Novartis’s immuno-oncology strategy – one of four bold approaches to reimagining cancer and transforming patients’ lives.
In an agreement finalized earlier this year, BeiGene granted Novartis rights to develop, manufacture, and commercialize tislelizumab in North America, Europe, and Japan through a collaboration and license agreement.
Find out more at https://www.novartis.com.
https://www.beigene.com/en-us/science-and-product-portfolio/pipeline/tislelizumab
Novartis' tislelizumab improves overall survival, shows anti-tumor activity in esophageal cancer patients
Jun. 07, 2021 6:32 AM ETNovartis AG (NVS)By: Mamta Mayani, SA News Editor
- Novartis (NYSE:NVS) announces results from Phase III RATIONALE 302 trial showing the investigational anti-PD-1 immune checkpoint inhibitor tislelizumab improved overall survival (OS) vs. chemotherapy (median 8.6 months vs. 6.3 months, p=0.0001)
- https://seekingalpha.com/news/3703561-novartis-tislelizumab-improves-overall-survival-shows-anti-tumor-activity-in-esophageal-cancer-patients
- https://seekingalpha.com/symbol/NVS
- https://seekingalpha.com/symbol/BGNE
biotecMAX™ April/May/June 2021 Insight
Imfinzi (durvalumab)
Imfinzi demonstrated unprecedented survival in unresectable, Stage III lung cancer with 43% of patients surviving five years
PUBLISHED4 June 2021
4 June 2021 07:10 BST
Long-term data from PACIFIC Phase III trial at ASCO showed 33% of patients remained progression-free at five years
Longest-ever survival reported in a Phase III immunotherapy trial in this setting
Updated results from the positive PACIFIC Phase III trial showed AstraZeneca’s Imfinzi (durvalumab) demonstrated a sustained, clinically meaningful overall survival (OS) and progression-free survival (PFS) benefit at five years in patients with unresectable, Stage III non-small cell lung cancer (NSCLC) who had not progressed following concurrent chemoradiation therapy (CRT).
Lung cancer is the leading cause of cancer death accounting for about one-fifth of all cancer deaths, and 80-85% of patients with lung cancer have NSCLC.1-3 One in four patients with NSCLC are diagnosed at Stage III, where the majority of tumours are unresectable (cannot be removed with surgery).4,5 The approval of Imfinzi in this setting based on the results of this trial was the first new treatment to be available to these patients in decades.2,6,7
Results from the updated post-hoc analyses showed an estimated five-year OS rate of 42.9% for patients treated with Imfinzi versus 33.4% for those on placebo after CRT. Median OS was 47.5 months for Imfinzi versus 29.1 for placebo. Following a maximum treatment course of one year, an estimated 33.1% of patients treated with Imfinzi had not progressed five years after enrolment versus 19% for placebo. These results build on the primary PFS and OS analyses published in The New England Journal of Medicine in 2017 and 2018, which demonstrated a sustained, significant benefit with Imfinzi for these primary endpoints.8,9
PACIFIC
The PACIFIC trial was a Phase III, randomised, double-blinded, placebo-controlled, multi-centre trial of Imfinzi as treatment in ‘all-comer’ patients (regardless of PD-L1 status) with unresectable, Stage III NSCLC whose disease had not progressed following concurrent platinum-based CRT.
The trial was conducted at 235 centres across 26 countries involving 713 patients. The primary endpoints of the trial were PFS and OS, and secondary endpoints included landmark PFS and OS, objective response rate and duration of response.
Imfinzi
Imfinzi (durvalumab) is a human monoclonal antibody that binds to PD-L1 and blocks the interaction of PD-L1 with PD-1 and CD80, countering the tumour's immune-evading tactics and releasing the inhibition of immune responses.
In addition to approvals in the unresectable, Stage III NSCLC setting, Imfinzi is approved for the treatment of extensive-stage small cell lung cancer (ES-SCLC) based on the CASPIAN Phase III trial in the EU, US, Japan and many other countries around the world. Imfinzi is also approved for previously treated patients with advanced bladder cancer in several countries.
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 NSCLC, SCLC, bladder cancer, hepatocellular carcinoma, biliary tract cancer (a form of liver cancer), oesophageal cancer, gastric and gastroesophageal cancer, cervical cancer, ovarian cancer, endometrial cancer and other solid tumours.
Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca.
AstraZeneca's Imfinzi shows long-term survival benefit in stage III lung cancer patients
Jun. 04, 2021 7:18 AM ETAstraZeneca PLC (AZN) By: Mamta Mayani, SA News Editor
- AstraZeneca’s (NASDAQ:AZN) Imfinzi (durvalumab) demonstrates a sustained, clinically meaningful overall survival (OS) and progression-free survival (PFS) benefit at five years in Phase III PACIFIC trial in patients with unresectable Stage III non-small cell lung cancer (NSCLC) who had not progressed following concurrent chemoradiation therapy (CRT).
- https://seekingalpha.com/news/3703234-astrazenecas-imfinzi-shows-long-term-survival-benefit-in-stage-iii-lung-cancer-patients
- https://seekingalpha.com/symbol/AZN
- https://www.astrazeneca.com/
- https://www.imfinzi.com/
Wegovy™ (semaglutide) injection 2.4 mg
Novo Nordisk receives FDA approval for Wegovy™ to treat adults with obesity based on unprecedented efficacy for a prescription medicine in clinical trials
03:46
4 June 2021
PLAINSBORO, N.J., June 4, 2021 /PRNewswire/ -- Novo Nordisk today announced that the US Food and Drug Administration (FDA) has approved the new drug application (NDA) for Wegovy™ (semaglutide) injection 2.4 mg, the first-and-only prescription weight-loss medication with once-weekly dosing. Wegovy™ is used with a reduced calorie meal plan and increased physical activity for adults with obesity (BMI ≥30) or overweight (excess weight) (BMI ≥27) who also have weight related medical problems to help them lose weight and keep the weight off.1
Experience the interactive Multichannel News Release here: https://www.multivu.com/players/English/8888551-novo-nordisk-semaglutide-2-4-fda-approval/
Approximately 42% of adults in the US live with obesity, a chronic disease which is recognized by the Centers for Disease Control and Prevention (CDC) as an epidemic.2,3 Obesity is a gateway disease associated with at least 60 other serious health conditions and decreased life expectancy.4,5
The FDA approval of Wegovy™ was based on the results of the STEP (Semaglutide Treatment Effect in People with Obesity) clinical trial program, which included four phase 3a clinical trials involving approximately 4,500 patients. The trials demonstrated that patients taking Wegovy™ with a reduced calorie meal plan and increased physical activity achieved a significant reduction in body weight compared with placebo (reduced calorie meal plan and increased physical activity only). In the STEP 1 trial, results also showed that patients taking Wegovy™ achieved an average weight loss of 14.9% of body weight at 68-weeks vs. 2.4% for placebo. In addition, 83.5% of patients achieved 5% or more body weight reduction in the Wegovy™ arm vs. 31.1% for placebo.1
Novo Nordisk expects to launch Wegovy™ later this month. For further information, please visit www.Wegovy.com.
Wegovy™ Indication and Important Safety Information
What is Wegovy™?
Wegovy™ (semaglutide) injection 2.4 mg is an injectable prescription medicine used for adults with obesity (BMI ≥30) or overweight (excess weight) (BMI ≥27) who also have weight-related medical problems to help them lose weight and keep the weight off.
- Wegovy™ should be used with a reduced calorie meal plan and increased physical activity.
- Wegovy™ contains semaglutide and should not be used with other semaglutide-containing products or other GLP-1 receptor agonist medicines.
- It is not known if Wegovy™ is safe and effective when taken with other prescription, over-the- counter, or herbal weight loss products.
- It is not known if Wegovy™ can be used safely in people with a history of pancreatitis.
- It is not known if Wegovy™ is safe and effective for use in children under 18 years of age.
About Wegovy™
Wegovy™ is a GLP-1 receptor agonist.1 It is currently under review in the European Union and other countries.11
For more information, visit novonordisk.us
FDA approves Novo Nordisk obesity drug Wegovy
Jun. 04, 2021 3:31 PM ETNovo Nordisk A/S (NVO)By: Jonathan M Block, SA News Editor6 Comments
- The FDA has granted approval to Novo Nordisk's (NYSE:NVO) Wegovy (semaglutide) for chronic weight management.
- https://seekingalpha.com/news/3703446-fda-approves-novo-nordisk-obesity-drug-wegovy
- https://seekingalpha.com/symbol/NVO
- https://www.novonordisk-us.com/partnering-and-innovation/our-scientific-approach.html
Puma Biotechnology Presents Data Comparing Findings from the Phase II CONTROL Trial with the Neratinib Arm of the Phase III ExteNET Trial at the ASCO 2021 Annual Meeting
Dose escalation of neratinib lowers the frequency of severe diarrhea and improves overall tolerability in patients with HER2-positive early stage breast cancer
LOS ANGELES, Calif., June 4, 2021 - Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, presented results at the virtual 2021 ASCO Annual Meeting comparing the diarrhea mitigation strategies investigated in the Phase II CONTROL trial with the neratinib treatment arm of the ExteNET trial where diarrhea prophylaxis was not required. The presentation, entitled “Dose escalation for mitigating diarrhea: Ranked tolerability assessment of antidiarrheal regimens in patients receiving neratinib for early-stage breast cancer,” is included in the Breast Cancer—Local/Regional/Adjuvant Poster Session (#536).
The CONTROL trial is an international, open-label, Phase II study investigating the use of antidiarrheal prophylaxis or dose escalation to improve the tolerability of neratinib-associated diarrhea. The primary endpoint of the trial is the incidence of grade 3 diarrhea. Patients ≥18 years of age with stage I–IIIc HER2-positive breast cancer received neratinib (240 mg/day orally for 1 year) together with one of the regimens investigated: loperamide alone, in combination with budesonide or colestipol, or neratinib dose escalation (DE): 120 mg/day on days 1–7, 160 mg/day on days 8–14, then 240 mg/day thereafter + loperamide PRN.
In the analysis presented at ASCO 2021, five CONTROL cohorts that had completed follow up were evaluated for 13 endpoints related to tolerability. The DE cohort ranked the best among the CONTROL cohorts and was then compared with the neratinib arm of the ExteNET trial, which included patients ≥18 years of age with stage I–III HER2-positive breast cancer receiving neratinib 240 mg/day or matching placebo for one year, without mandated anti-diarrheal treatment. ExteNET was a multicenter, randomized, double-blind, Phase III trial (NCT00878709) of 2,840 HER2-positive early stage breast cancer patients who received neratinib after neoadjuvant and/or adjuvant therapy with chemotherapy and a trastuzumab-based regimen.
Comparison of the CONTROL DE cohort with the ExteNET neratinib arm demonstrated that grade 3 diarrhea was substantially lower in CONTROL DE compared to ExteNET (13.3% vs. 39.9%). Neratinib DE also resulted in fewer total days of grade 3 diarrhea compared to ExteNET (2.5 days vs 5 days). Dose escalation also led to fewer discontinuations due to diarrhea in the first three months of treatment compared to ExteNET (3.3% vs 14.5%). Additionally, the average duration of treatment with neratinib was much longer in the DE cohort versus ExteNET. Overall, the findings of this analysis suggest that escalating the dose of neratinib in the first 2 weeks of treatment may help patients stay on neratinib longer, allowing them the opportunity to complete the recommended 1-year of treatment.
Further information about Puma Biotechnology may be found at www.pumabiotechnology.com.
NERLYNX® (neratinib) tablets, for oral use
Please see Full Prescribing Information for additional safety information.
NERLYNX® (neratinib) tablets, for oral use
INDICATIONS AND USAGE: NERLYNX is a kinase inhibitor indicated:
- As a single agent, for the extended adjuvant treatment of adult patients with early-stage HER2-positive breast cancer, to follow adjuvant trastuzumab-based therapy.
- In combination with capecitabine, for the treatment of adult patients with advanced or metastatic HER2-positive breast cancer, who have received two or more prior anti-HER2 based regimens in the metastatic setting.
CONTRAINDICATIONS: None
Puma Bio reports data from mid-stage study of neratinib in EGFR exon 18-mutant NSCLC patients
Jun. 04, 2021 9:26 AM ETPuma Biotechnology, Inc. (PBYI)By: Mamta Mayani, SA News Editor
- Puma Biotechnology (NASDAQ:PBYI) presents interim results from the Phase II SUMMIT basket trial, assessing the efficacy of neratinib in patients with EGFR exon 18-mutant non-small cell lung cancer (NSCLC), including patients with central nervous system (CNS) involvement, at the virtual 2021 ASCO Annual Meeting.
- https://seekingalpha.com/news/3703319-puma-bio-reports-data-from-mid-stage-study-of-neratinib-in-egfr-exon-18-mutant-nsclc-patients
- https://seekingalpha.com/symbol/PBYI
- https://www.pumabiotechnology.com/index.html
- https://nerlynx.com/
IMBRUVICA® (ibrutinib)
June 4, 2021
Results from IMBRUVICA® (ibrutinib) RESONATE-2 Study Provide Up to Seven Years of Progression-Free and Overall Survival Data in First-Line Chronic Lymphocytic Leukemia (CLL)
- With up to seven years of data, progression-free and overall survival benefits continue to be observed with first-line single-agent IMBRUVICA treatment in CLL patients including selected patients with or without high-risk genomic features
- Safety data from RESONATE-2 showed most rates of any grade adverse event generally decreased or remained stable over time with no new safety signals reported
- RESONATE-2 represents the longest Phase 3 survival data ever reported for any BTK inhibitor in CLL
NORTH CHICAGO, Ill., June 4, 2021 /PRNewswire/ -- AbbVie (NYSE: ABBV), today announced extended long-term data from the Phase 3 RESONATE-2 study (PCYC-1115/1116) evaluating single-agent IMBRUVICA (ibrutinib) versus chlorambucil with up to seven years of follow-up in patients with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL).1 These data will be presented on June 4th during the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting (Abstract #7523). Additionally, new data will be presented during the European Hematology Association (EHA) Virtual Congress from June 9-17, including findings from the informCLL™ real-world prospective observational registry assessing how real-world treatment patterns align with National Comprehensive Cancer Network (NCCN®)-recommended regimens for CLL/SLL.
"These data add to the overall body of evidence that patients treated with ibrutinib can achieve extended progression-free survival and overall survival when used as a first-line therapy," said Paul M. Barr, M.D., lead study investigator of the Phase 3 RESONATE-2 trial, and Professor of Medicine at the Wilmot Cancer Institute, University of Rochester. "The results add to the extensive clinical evidence supporting the use of single-agent ibrutinib for long-term disease control."
The RESONATE-2 study evaluated 269 patients 65 years or older with previously untreated CLL/SLL, without 17p deletion, who received continuous single-agent IMBRUVICA until progression or chlorambucil up to 12 cycles.1 With up to seven years of follow-up, progression-free survival (PFS) benefit with single-agent IMBRUVICA was sustained (Hazard Ratio [HR] 0.160 [95 percent Confidence Interval (CI): 0.111–0.230]).1 At 6.5 years of follow-up, median PFS in the IMBRUVICA treatment arm was not reached: the PFS rate for patients treated with single-agent IMBRUVICA was 61 percent compared with only nine percent in patients treated with chlorambucil.1 Additionally, at 6.5 years, the IMBRUVICA treatment arm showed an overall survival (OS) rate of 78 percent; OS was not captured for the chlorambucil treatment arm for patients with disease progression after a median of five years of follow-up.1 In this latest follow-up, the overall response rate (ORR) was 92 percent.1
The pivotal Phase 3 RESONATE-2 study served as the basis for the FDA approval of IMBRUVICA as a single-agent in first-line treatment for CLL/SLL in 2016, following initial approval for relapsed/refractory (R/R) patients in 2014 based on the RESONATE study.2
(Abstract EP635) Real-World Application of NCCN Clinical Practice Guidelines (NCCN Guidelines®) for CLL/SLL from the informCLL Prospective Observational Registry
Results from the informCLL™ real-world registry assessing treatment alignment with NCCN Guidelines® will be presented as a poster during the EHA Virtual Congress on June 11th.
The informCLL™ registry enrolled 1,462 patients, of whom 58 percent were previously untreated and 42 percent were relapsed or refractory. Community-based practices enrolled 93 percent of the patients.3 The median age was 71 years, and the median Charlson Comorbidity Index (CCI) was one with 16 percent having a CCI >3. Single-agent ibrutinib was the most common treatment across line of therapy at enrollment. For this analysis, NCCN Guidelines® for CLL/SLL V.2.2017 were used given 74 percent enrollment in 2017.3
The latest analysis showed that a third of high-risk patients with 17p deletion/TP53 mutation, who typically have poor outcomes after chemoimmunotherapy (CIT), did not receive NCCN®-recommended regimens.3 Most patients without 17p deletion/TP53 mutation received recommended therapy across age/comorbidity groups.3 Despite guideline recommendations for prognostic testing, the majority of patients in the registry were not tested for both 17p deletion/TP53 mutation and therefore may have received suboptimal treatment with CIT.3 These results underscore the importance of prognostic marker testing and appropriate selection of therapies based on NCCN Guidelines® recommendations, especially in the community setting.3
About IMBRUVICA®
IMBRUVICA (ibrutinib) is a once-daily, first-in-class BTK inhibitor that is administered orally, and is jointly developed and commercialized by Pharmacyclics, LLC, an AbbVie Company, and Janssen Biotech, Inc. (Janssen). The BTK protein sends important signals that tell B cells to mature and produce antibodies. BTK signaling is needed by specific cancer cells to multiply and spread.4,5 By blocking BTK, IMBRUVICA may help move abnormal B cells out of their nourishing environments in the lymph nodes, bone marrow, and other organs.6
Since its launch in 2013, IMBRUVICA® has received 11 FDA approvals across six disease areas: chronic lymphocytic leukemia (CLL) with or without 17p deletion (del17p); small lymphocytic lymphoma (SLL) with or without del17p; Waldenström macroglobulinemia; previously-treated patients with mantle cell lymphoma (MCL)*; previously-treated patients with marginal zone lymphoma (MZL) who require systemic therapy and have received at least one prior anti-CD20-based therapy* – and previously-treated patients with chronic graft-versus-host disease (cGVHD) after failure of one or more lines of systemic therapy.7
IMBRUVICA® is now approved in 101 countries and has been used to treat more than 230,000 patients worldwide across its approved indications. IMBRUVICA® is the only FDA-approved medicine in WM and cGVHD. IMBRUVICA® has been granted four Breakthrough Therapy Designations from the U.S. FDA. This designation is intended to expedite the development and review of a potential new drug for serious or life-threatening diseases. IMBRUVICA® was one of the first medicines to receive FDA approval via the Breakthrough Therapy Designation pathway.
Since 2019, the National Comprehensive Cancer Network® (NCCN®), a not-for-profit alliance of 28 leading cancer centers devoted to patient care, research, and education, recommends ibrutinib (IMBRUVICA®) as a preferred regimen for the initial treatment of CLL/SLL and has Category 1 treatment status for treatment-naïve patients without deletion 17p. In January 2020, the NCCN Guidelines® were updated to elevate IMBRUVICA® with or without rituximab from other recommended regimens to a preferred regimen for the treatment of relapsed/refractory MCL, regardless of duration of response to prior chemoimmunotherapy. As of September 2020, the NCCN guidelines were updated to reflect IMBRUVICA® with or without rituximab as the only Category 1 preferred regimen for both untreated and previously treated WM patients.
IMBRUVICA® is being studied alone and in combination with other treatments in several blood and solid tumor cancers and other serious illnesses. IMBRUVICA® is the most comprehensively studied BTK inhibitor, with more than 150 ongoing clinical trials. There are approximately 30 ongoing company-sponsored trials, 14 of which are in Phase 3, and more than 100 investigator-sponsored trials and external collaborations that are active around the world. For more information, visit www.IMBRUVICA.com
*Accelerated approval was granted for the MCL and MZL indications based on overall response rate. Continued approval for MCL and MZL may be contingent upon verification and description of clinical benefit in confirmatory trials.
Please click here for full Prescribing Information.
IMBRUVICA is a registered trademark of Pharmacyclics LLC.
SOURCE AbbVie Inc.
Long-term data shows survival benefit of J&J/Abbvie Imbruvica in leukemia
Jun. 04, 2021 9:29 AM ET Johnson & Johnson (JNJ), ABBV By: Jonathan M Block, SA News Editor4 Comments
- New long-term phase 3 data shows that Johnson & Johnson (NYSE:JNJ) and Abbvie's (NYSE:ABBV) Imbruvica (ibrutinib) leads to up to seven years of progression-free survival ("PFS") in patients with chronic lymphocytic leukemia ("CLL")/small lymphocytic lymphoma ("SLL").
- https://seekingalpha.com/news/3703307-long-term-data-shows-survival-benefit-of-johnson-johnson-abbvie-ihttps://seekingalpha.com/symbol/JNJmbruvica-in-leukemia
- https://seekingalpha.com/symbol/JNJ
- https://seekingalpha.com/symbol/ABBV
- https://www.imbruvica.com/
Lifileucel in Combination with Pembrolizumab
Iovance Biotherapeutics Announces Clinical Data for Lifileucel in Combination with Pembrolizumab in Advanced Melanoma at ASCO 2021 Annual MeetingPDF Version
86% Overall Response Rate (ORR) and 43% Complete Response Rate in Immune Checkpoint Inhibitor (ICI) Naïve Advanced Melanoma Patients in IOV-COM-202 Clinical Study
Initial 7 Patients Show 3 Complete Responses, 3 Partial Responses and 1 Best Response of Stable Disease
ASCO Update Conference Call and Webcast on Sunday, June 6 at 12 p.m. ET
SAN CARLOS, Calif., June 04, 2021 (GLOBE NEWSWIRE) -- Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a late-stage biotechnology company developing novel T cell-based cancer immunotherapies, today announced clinical data for lifileucel in combination with pembrolizumab in patients with advanced melanoma. The data are now available in an ePoster at the ASCO 2021 Annual Meeting.
Antonio Jimeno M.D., Ph.D., Professor of Medicine/Oncology and Otolaryngology at the University of Colorado School of Medicine stated, “Anti-PD-1 therapy has become standard of care in frontline melanoma, yet we are still looking for ways to help more patients respond and to improve upon the depth and durability of responses. The 86% Overall Response Rate (ORR) for lifileucel in combination with pembrolizumab is remarkable and suggests a potential additive effect for early-line treatment of patients with melanoma. I look forward to investigating this treatment approach in additional patients with melanoma as well as in other tumor types such as head and neck squamous cancer.”
Early data suggest the response rate of lifileucel plus pembrolizumab may be additive and confirm the potential feasibility and activity of this combination in patients with immune checkpoint inhibitor (ICI)-naïve advanced melanoma. Cohort 1A in the IOV-COM-202 study is evaluating lifileucel in combination with pembrolizumab in patients who are naïve to ICI, or anti-PD-1, therapy. Initial patients (n=7) enrolled in Cohort 1A had high tumor burden at baseline, and 71.4% had not received any prior systemic therapy.
Six of the seven patients had a confirmed objective response, representing an 86% ORR (2 complete responses (CR), 1 unconfirmed CR (uCR) who had not yet reached the confirmatory CR assessment, and 3 partial responses (PR)), with one best response of stable disease. Responses deepened over time and the CR/uCR rate was 43%. Poster data extraction was in April 2021 and the median follow up was 8.2 months. ORR was investigator-assessed per RECIST 1.1. In a subsequent data cut in May 2021, all ongoing responses continued.
The Cohort 1A results also demonstrated that lifileucel can be safely combined with pembrolizumab. The treatment-emergent adverse event (TEAE) profile was consistent with the underlying disease and known adverse event (AE) profiles of pembrolizumab, non-myeloablative lymphodepletion (NMA-LD) and IL-2. The median number of doses of IL-2 and pembrolizumab were six and 10, respectively.
For more information, please visit www.iovance.com.
Iovance Biotherapeutics posts positive data from lifileucel combo cancer trial
Jun. 04, 2021 9:20 AM ETIovance Biotherapeutics, Inc. (IOVA)By: Aakash Babu, SA News Editor6 Comments
- Iovance Biotherapeutics (NASDAQ:IOVA) announces positive clinical data for lifileucel in combination with pembrolizumab in patients with advanced melanoma.
- https://seekingalpha.com/news/3703314-iovance-biotherapeutics-posts-positive-data-from-lifileucel-combo-cancer-trial
- https://seekingalpha.com/symbol/IOVA
- https://www.iovance.com/
Polivy® (polatuzumab vedotin)
Friday, Jun 4, 2021
Genentech Presents Latest Advances With Immunotherapies in Non-Hodgkin’s Lymphoma
Data for investigational CD20xCD3 bispecific antibodies and new combination regimens with Polivy showed enhanced clinical benefits for people with non-Hodgkin’s lymphoma in early studies
South San Francisco, CA -- June 4, 2021 --
Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that new data on its investigational CD20xCD3 T-cell engaging bispecific antibodies, mosunetuzumab and glofitamab, and its first-in-class anti-CD79b antibody-drug conjugate, Polivy® (polatuzumab vedotin), in non-Hodgkin’s lymphoma (NHL) will be presented at the 2021 ASCO Annual Meeting from June 4-8, 2021.
“People with difficult-to-treat blood cancers such as non-Hodgkin’s lymphoma still need more options to help improve outcomes,” said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. “We are encouraged by promising data from our emerging T-cell engaging bispecific antibodies, mosunetuzumab and glofitamab, and the antibody-drug conjugate, Polivy, that demonstrate the potential of these novel immunotherapeutic approaches for various groups of patients.”
Polivy® (polatuzumab vedotin)
About Polivy® (polatuzumab vedotin-piiq)
Polivy is a first-in-class anti-CD79b antibody-drug conjugate (ADC). The CD79b protein is expressed specifically in the majority of B-cells, an immune cell impacted in some types of non-Hodgkin’s lymphoma (NHL), making it a promising target for the development of new therapies. Polivy binds to CD79b and destroys these B-cells through the delivery of an anti-cancer agent, which is thought to minimize the effects on normal cells. Polivy is being developed by Roche using Seagen ADC technology and is currently being investigated for the treatment of several types of NHL.
About the NP30179 study
The NP30179 study [NCT03075696] is a Phase I/Ib, multicenter, open-label, dose-escalation study, evaluating the efficacy, safety, tolerability and pharmacokinetics of glofitamab. In this study, glofitamab is assessed as a single-agent and in combination with Gazyva® (obinutuzumab), following pre-treatment with a one-time, fixed-dose of Gazyva, in people with relapsed or refractory (R/R) B-cell NHL. Outcome measures include overall response rate, complete response rate per Lugano 2014 criteria, maximum tolerated dose and tolerability.
About the GO40516 study
The GO40516 study [NCT03671018] is a Phase I/II, multicenter, open-label study, evaluating the efficacy, safety, tolerability and pharmacokinetics of mosunetuzumab in combination with Polivy in people with B-cell NHL. It consists of a dose finding portion followed by an expansion phase for second line or later (2L+) participants with R/R DLBCL and 2L+ R/R FL. Outcome measures include best overall response rate, maximum tolerated dose and tolerability.
About the GO29834 study
The GO29834 study [ NCT02600897] is a Phase Ib/II, multicenter, open-label study, evaluating the efficacy and safety of Polivy with Rituxan® (rituximab) and lenalidomide in R/R DLBCL. Outcome measures include complete response and tolerability.
Polivy U.S. Indication
Polivy is a prescription medicine used with other medicines, bendamustine and a rituximab product, to treat diffuse large B-cell lymphoma in adults who have progressed after at least two prior therapies.
The accelerated approval of Polivy is based on a type of response rate. There are ongoing studies to confirm the clinical benefit of Polivy.
Please visit http://www.Polivy.com for the full Prescribing Information for additional Important Safety Information.
For additional information about the company, please visit http://www.gene.com.
Genentech presents new data for a range of blood cancer therapies
Jun. 04, 2021 8:52 AM ET Roche Holding AG (RHHBY)
By: Dulan Lokuwithana, SA News Editor
- Genentech, a unit of Roche (OTCQX:RHHBY) announced new data for a range of experimental blood cancer therapies: mosunetuzumab alone, glofitamab in combination Polivy, and a triplet combination of Polivy with Rituxan and lenalidomide.
- https://seekingalpha.com/news/3703293-genentech-presents-new-data-for-a-range-of-blood-cancer-therapies
- https://seekingalpha.com/symbol/RHHBY
- https://www.gene.com/
- https://www.polivy.com/
Lynparza (olaparib)
Lynparza reduced the risk of cancer recurrence by 42% in the adjuvant treatment of patients with germline BRCA-mutated high-risk early breast cancer in OlympiA Phase III trial
PUBLISHED3 June 2021
3 June 2021 22:00 BST
First medicine targeting BRCA mutations to show clinical benefit in adjuvant setting
Results from the OlympiA Phase III trial showed AstraZeneca and MSD’s Lynparza (olaparib) demonstrated a statistically significant and clinically meaningful improvement in invasive disease-free survival (iDFS) versus placebo in the adjuvant treatment of patients with germline BRCA-mutated (gBRCAm) high-risk human epidermal growth factor receptor 2 (HER2)-negative early breast cancer.
The results will be presented during the plenary session of the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting on 6 June 2021 (abstract LBA#1) and were published today in The New England Journal of Medicine.
An estimated 2.3 million people were diagnosed with breast cancer worldwide in 2020 and BRCA mutations are found in approximately 5% of breast cancer patients.1,2
In the overall trial population of patients who had completed local treatment and standard neoadjuvant or adjuvant chemotherapy, results showed Lynparza reduced the risk of invasive breast cancer recurrences, second cancers or death by 42% (based on a hazard ratio [HR] of 0.58; 99.5% confidence interval [CI] 0.41-0.82; p<0.0001). At three years, 85.9% of patients treated with Lynparza remained alive and free of invasive breast cancer and second cancers versus 77.1% on placebo.
Lynparza also demonstrated a statistically significant and clinically meaningful improvement in the key secondary endpoint of distant disease-free survival (DDFS) in the overall trial population. Lynparza reduced the risk of distant disease recurrence or death by 43% (based on an HR of 0.57; 99.5% CI 0.39-0.83; p<0.0001). At the time of this initial data cut-off, fewer deaths had occurred in patients receiving Lynparza, but the difference in overall survival (OS) did not reach statistical significance. The trial will continue to assess OS as a secondary endpoint.
In February 2021, the Independent Data Monitoring Committee recommended for the OlympiA trial to move to early primary analysis and reporting. Based on the planned interim analysis, the IDMC concluded that the trial crossed the superiority boundary for its primary endpoint of iDFS and demonstrated a sustainable and clinically relevant treatment effect for Lynparza versus placebo.
OlympiA
OlympiA is a Phase III, double-blind, 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. The primary endpoint of the trial is iDFS defined as time from randomisation to date of first loco-regional or distant recurrence, new cancer or death from any cause. Key secondary endpoints include OS and DDFS, which is defined as time from randomisation until documented evidence of first distant recurrence of breast cancer or death without distant recurrence.3
BIG
The Breast International Group (BIG) is an international not-for-profit organisation for academic breast cancer research groups from around the world, based in Brussels, Belgium.
Founded by leading European opinion leaders in 1999, the organisation aims to address fragmentation in breast cancer research and now represents a network of over 50 like-minded research groups affiliated with specialised hospitals, research centres and leading experts across approximately 70 countries on six continents.
BIG’s research is supported in part by its philanthropy unit, known as BIG against breast cancer, which is used to interact with the general public and donors, and to raise funds for BIG’s purely academic breast cancer trials and research programmes.
FSTRF
Frontier Science & Technology Research Foundation (FSTRF) is a non-profit, research organisation which supports research networks, pharmaceutical companies and investigators to conduct scientifically meaningful high-quality clinical trials. The OlympiA trial involved research staff in the US and in the Affiliate office in Scotland.
FSTRF works with scientists and technicians in more than 800 laboratories, universities and medical centres around the world to provide a comprehensive range of research services throughout the clinical trial process including design, analysis and reporting.
Through its work, FSTRF aims to advance the application of statistical science and practice and data management techniques in science, healthcare and education.
BRCA1 and BRCA2
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. 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 genetic alterations that can lead to cancer and confer sensitivity to PARP inhibitors including Lynparza.8-11
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 mutations in BRCA1 and/or BRCA2. Inhibition of PARP 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 being tested in a range of PARP-dependent tumour types with defects and dependencies in the DDR pathway.
Lynparza is currently approved in a number of countries, including those in the EU, for the maintenance treatment of platinum-sensitive relapsed ovarian cancer. It is approved in the US, the EU, Japan, China, and several other countries as 1st-line maintenance treatment of BRCA-mutated advanced ovarian cancer following response to platinum-based chemotherapy. It is also approved in the US, EU and Japan as a 1st-line maintenance treatment with bevacizumab for patients with HRD-positive advanced ovarian cancer (BRCAm and/or genomic instability). Lynparza is approved in the US, Japan, and a number of other countries for germline BRCA-mutated, HER2-negative, metastatic breast cancer, previously treated with chemotherapy; in the EU, this includes locally advanced breast cancer. It is also approved in the US, the EU, Japan and several other countries for the treatment of germline BRCAm metastatic pancreatic cancer. Lynparza is approved in the US for HRR gene-mutated metastatic castration-resistant prostate cancer (BRCAm and other HRR gene mutations) and in the EU and Japan for BRCAm metastatic castration-resistant prostate cancer. Regulatory reviews are underway in several countries for ovarian, breast, pancreatic and prostate cancers.
Lynparza, which is being jointly developed and commercialised by AstraZeneca and MSD, has been used to treat over 40,000 patients worldwide. Lynparza has the broadest and most advanced clinical trial development programme of any PARP inhibitor, and AstraZeneca and MSD are working together to understand how it may affect multiple PARP-dependent tumours as a monotherapy and in combination across multiple cancer types. Lynparza 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. Independently, the companies will develop Lynparza and Koselugo in combination with their respective PD-L1 and PD-1 medicines.
Please visit astrazeneca.com
AstraZeneca shows clinical benefit of Lynparza in late-stage trial for breast cancer
Jun. 03, 2021 5:40 PM ET AstraZeneca PLC (AZN) By: Dulan Lokuwithana, SA News Editor
- AstraZeneca (NASDAQ:AZN) ADR’s have added ~1.4% in extended hours after the results of a Phase 3 trial for Lynparza (olaparib) indicated clinically and statistically significant improvement as adjuvant therapy in a subset of patients with early breast cancer.
- https://seekingalpha.com/news/3703153-astrazeneca-shows-clinical-benefit-of-lynparza-in-late-stage-trial-for-breast-cancer
- https://seekingalpha.com/symbol/AZN
- https://seekingalpha.com/symbol/MRK
- AstraZeneca and Merck (NYSE:MRK) collaborate in the development and commercialization of Lynparza.
- https://www.lynparza.com/
Filgotinib
Mechelen, Belgium; 4 June 2021; 07.01 CET; – Galapagos NV (Euronext & Nasdaq: GLPG) today announced that primary and secondary endpoint results from the phase 3 SELECTION induction and maintenance study (NCT02914522) were published in The Lancet (doi.org/10.1016/S0140-6736(21)00666-8). The study, sponsored by Gilead Sciences, Inc., was designed to assess the efficacy and safety of the once-daily, oral preferential JAK1 inhibitor, filgotinib, under investigation in patients with moderately to severely active ulcerative colitis (UC).
In addition to the primary and secondary endpoints, which have been reported at the United European Gastroenterology congress in October 2020 and can be found on the Galapagos website, www.glpg.com/press-releases, the publication also reports data from a post-hoc analysis by induction cohort of filgotinib 200mg versus placebo during the maintenance study on multiple efficacy endpoints. These include sustained clinical remission, 6-month corticosteroid free remission, Mayo Clinic Score (MCS), endoscopic and histologic remission, MCS response and endoscopic improvement. Across all these endpoints, numerically greater differences in favor of filgotinib 200mg compared to placebo were shown. This is reported independent of previous biologic treatment status (biologic-naïve and biologic-experienced) with an overall larger numerical effect among biologic-naïve patients1.
A further post-hoc analysis in the publication reports mucosal healing, a composite endpoint defined as endoscopic improvement (Mayo endoscopy score 0-1) and histological remission in the same patient. The proportion of patients achieving mucosal healing after 10 weeks of induction treatment with filgotinib 200mg was numerically greater compared with placebo (23.3% vs 10.9% in biologic-naïve and 9.9% vs 4.2% in biologic-experienced) and at week 58 in the overall study population (32.7% vs 10.2%)2.
About the SELECTION Phase 3 Trial
The SELECTION Phase 3 trial is a multi-center, randomized, double-blind, placebo-controlled trial to assess the safety and efficacy of the preferential JAK1 inhibitor filgotinib in adult patients with moderately to severely active UC. The SELECTION trial comprises two induction trials and a maintenance trial. The Induction Study A enrolled biologic-naïve patients, and the Induction Study B enrolled biologic-experienced patients.
The primary objectives of SELECTION were to evaluate the efficacy of filgotinib compared with placebo in establishing clinical remission as determined by the Mayo endoscopic subscore of 0 or 1, rectal bleeding subscore of 0, and ≥ 1-point decrease in stool frequency from baseline to achieve a subscore of 0 or 1 at Week 10 in the induction studies and Week 58 in the maintenance study. Eligible patients who were enrolled in the SELECTION trial were enrolled in the ongoing SELECTION long-term extension trial to evaluate the long-term safety of filgotinib in patients with moderately to severely active UC. A majority of patients included in the trials had a MCS score of 9 or higher at baseline, and 43% of biologic experienced patients had insufficient response to a TNF antagonist and vedolizumab as well.
Data on the primary endpoint showed that a greater proportion of biologic-naïve and biologic-experienced patients receiving filgotinib 200mg achieved clinical remission at Week 10 than those on placebo (26.1% versus 15.3% p=0.0157 and 11.5% versus 4.2% p=0.013 respectively). This clinical remission was maintained up to 58 weeks for those receiving filgotinib 200mg versus those receiving placebo (37.2% versus 11.2% p<0.001). The difference for filgotinib 100mg was not statistically significant in the induction study, however in the maintenance study at Week 58 a statistically significant difference was seen versus placebo (23.8% versus 13.5%, p=0.0420).
Overall, the incidence of adverse events (AEs), serious AEs and discontinuations due to AEs were similar in the filgotinib and placebo groups in both the induction and maintenance periods of the study, as reported above.
For SELECTION study information visit: https://clinicaltrials.gov/ct2/show/NCT02914522
For access to SELECTION in The Lancet publication visit; http://www.thelancet.com/journals/lancet/article/PIIS0140-6736(21)00666-8/fulltext
1 Feagan. B., et al: Filgotinib as induction and maintenance therapy for ulcerative colitis: the SELECTION trial. The Lancet https://doi.org/10.1016/S0140-6736(21)00666-8. Appendix page 29, Table S4
2 Feagan. B., et al: Filgotinib as induction and maintenance therapy for ulcerative colitis: the SELECTION trial. The Lancet https://doi.org/10.1016/S0140-6736(21)00666-8. Appendix page 15, Figure S5
About filgotinib
Filgotinib is approved and marketed as Jyseleca (200mg and 100mg tablets) in the European Union, Great Britain, and Japan for the treatment of adults with moderate to severe active rheumatoid arthritis (RA) who have responded inadequately or are intolerant to one or more disease modifying anti-rheumatic drugs (DMARDs). Filgotinib may be used as monotherapy or in combination with methotrexate (MTX). The European Summary of Product Characteristics for filgotinib, which includes contraindications and special warnings and precautions, is available at www.ema.europa.eu. The interview form from the Japanese Ministry of Health, Labour and Welfare is available at www.info.pmda.go.jp. The Great Britain Summary of Product Characteristics is available at www.medicines.org.uk/emc. Applications have been submitted to the European Medicines Agency (EMA), the UK’s Medicines and Healthcare products Regulatory Agency (MHRA), and Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) for the treatment of adults with moderately to severely active ulcerative colitis who have had an inadequate response with, lost response to, or were intolerant to either conventional therapy or a biologic agent and are currently under review. Filgotinib is not approved in any other countries.
About the filgotinib collaboration
Gilead and Galapagos NV are collaborative partners in the global development and commercialization of filgotinib. Galapagos will be responsible for the commercialization of filgotinib in Europe (transition anticipated to be completed by end of 2021), while Gilead will remain responsible for filgotinib outside of Europe, including in Japan, where filgotinib is co-marketed with Eisai. Filgotinib in UC has been filed in Europe and Japan a global Phase 3 program is ongoing in Crohn’s Disease. More information about clinical trials can be accessed at www.clinicaltrials.gov.
More information at www.glpg.com.
https://www.glpg.com/filgotinib
Galapagos, Gilead's Selection study on filgotinib in ulcerative colitis published in the Lancet
Jun. 04, 2021 6:18 AM ET Galapagos NV (GLPG)
By: Mamta Mayani, SA News Editor1 Comment
- Results from Galapagos' (NASDAQ:GLPG) Phase 3 SELECTION induction and maintenance study were published in The Lancet.
- The study, sponsored by Gilead Sciences (NASDAQ:GILD) was designed to assess the efficacy and safety of the once-daily, oral preferential JAK1 inhibitor, filgotinib, under investigation in patients with moderately to severely active ulcerative colitis (UC).
- In addition to the primary and secondary endpoints which we
- https://seekingalpha.com/news/3703214-galapagos-gileads-selection-study-on-filgotinib-in-ulcerative-colitis-published-in-the-lancet
- https://seekingalpha.com/symbol/GLPG
- https://seekingalpha.com/symbol/GILD
- https://www.glpg.com/
Trodelvy® (sacituzumab govitecan-hziy)
June 04, 2021
Trodelvy® Demonstrates Superior Outcomes to Standard of Care in Second-Line Treatment of Metastatic Triple-Negative Breast Cancer in Phase 3 ASCENT Study
– Trodelvy More than Doubled Overall Survival as Second-Line Treatment in New ASCENT Subgroup Analysis –
FOSTER CITY, Calif.--(BUSINESS WIRE)-- Gilead Sciences, Inc. (Nasdaq: GILD) today announced new data from the Phase 3 ASCENT study evaluating Trodelvy® (sacituzumab govitecan-hziy) in relapsed or refractory metastatic triple-negative breast cancer (TNBC). In this subgroup analysis of brain metastases-negative patients who received only one line of prior systemic therapy in the metastatic setting in addition to having disease recurrence or progression within 12 months of (neo)adjuvant chemotherapy, Trodelvy improved progression-free survival (PFS), with a 59% reduction in the risk of disease worsening or death (HR: 0.41; 95% CI: 0.22-0.76) and a median PFS of 5.7 months (n=33) versus 1.5 months with chemotherapy (n=32). Trodelvy also extended median overall survival to 10.9 months versus 4.9 months with chemotherapy (HR: 0.51; 95% CI: 0.28-0.91). The results were presented at the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting (Abstract #1080).
Additional results showed Trodelvy demonstrated a higher overall response rate compared with chemotherapy (30% versus 3%). Efficacy results from this subgroup were consistent with those observed in the overall ASCENT study population.
The safety profile of Trodelvy in this subgroup was consistent with prior reports. The most frequent Grade ≥3 treatment-related adverse reactions for Trodelvy compared to chemotherapy were neutropenia (61% versus 21%), leukopenia (9% versus 0%), diarrhea (6% versus 0%), anemia (3% versus 6%), and fatigue (3% versus 0%). One patient in this subgroup who received Trodelvy experienced febrile neutropenia. Adverse reactions leading to treatment discontinuation were low across both groups (6% in each). There were no treatment-related deaths with Trodelvy in this subgroup. The Trodelvy U.S. Prescribing Information has a BOXED WARNING for severe or life-threatening neutropenia and severe diarrhea; see below for Important Safety Information.
About the ASCENT Study
The Phase 3 ASCENT study, an open-label, active-controlled, randomized confirmatory trial, enrolled more than 500 patients with relapsed/refractory metastatic triple-negative breast cancer who had received two or more prior systemic therapies, including a taxane, at least one of them for metastatic disease. Patients were randomized to receive either Trodelvy or a chemotherapy chosen by the patients’ treating physicians. The primary efficacy outcome was PFS in patients without brain metastases at baseline, as measured by a blinded, independent, centralized review using RECIST v1.1 criteria. Additional efficacy measures included PFS for the full population, including all patients with and without brain metastases, and overall survival. More information about ASCENT is available at http://clinicaltrials.gov/show/NCT02574455.
About Trodelvy
Trodelvy (sacituzumab govitecan-hziy) is a first-in-class antibody and topoisomerase inhibitor conjugate directed to the Trop-2 receptor, a protein frequently expressed in multiple types of epithelial tumors, including metastatic triple-negative breast cancer and metastatic urothelial cancer, where high expression is associated with poor survival and relapse.
In the U.S., Trodelvy is indicated for the treatment of:
- Adult patients with unresectable locally advanced or metastatic triple-negative breast cancer who have received two or more prior systemic therapies, at least one of them for metastatic disease.
- Adult patients with locally advanced or metastatic urothelial cancer who have previously received a platinum-containing chemotherapy and either programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor.
Beyond the regulatory approvals of Trodelvy in the U.S., regulatory reviews for Trodelvy in metastatic triple-negative breast cancer are currently underway in the EU,U.K., Canada, Switzerland and Australia, as well as in Singapore through our partner Everest Medicines. Trodelvy is also being investigated as potential treatment for hormone receptor-positive/human epidermal growth factor receptor 2-negative (HR+/HER2-) metastatic breast cancer and metastatic non-small cell lung cancer. Additional evaluation across multiple solid tumors is also underway.
Please see full Prescribing Information, including BOXED WARNING.
U.S. Prescribing Information for Trodelvy including BOXED WARNING, is available at www.gilead.com.
Trodelvy, Gilead and the Gilead logo are trademarks of Gilead Sciences, Inc., or its related companies.
For more information about Gilead, please visit the company’s website at www.gilead.com, follow Gilead on Twitter (@GileadSciences) or call Gilead Public Affairs at 1-800-GILEAD-5 or 1-650-574-3000.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210604005059/en/
Source: Gilead Sciences, Inc.
https://seekingalpha.com/symbol/GILD
Gilead highlights potential of Trodelvy in breast cancer as second line therapy
Jun. 04, 2021 10:47 AM ET Gilead Sciences, Inc. (GILD)
By: Dulan Lokuwithana, SA News Editor
- Gilead Sciences (GILD +1.3%) says that its cancer therapy Trodelvy (sacituzumab govitecan-hziy) improved the outcomes of patients with relapsed or refractory metastatic triple-negative breast cancer (TNBC) in a second-line setting.
- https://seekingalpha.com/news/3703362-gilead-highlights-potential-of-trodelvy-in-breast-cancer-as-second-line-therapy
- https://seekingalpha.com/symbol/GILD
- https://trodelvy.com/
REGEN-COV (casirivimab and imdevimab)
FDA AUTHORIZES LOWER 1,200 MG INTRAVENOUS AND SUBCUTANEOUS DOSE OF REGEN-COV™ (CASIRIVIMAB AND IMDEVIMAB) ANTIBODY COCKTAIL TO TREAT PATIENTS WITH COVID-19
TARRYTOWN, N.Y., June 4, 2021 /PRNewswire/ --
EUA supported by pivotal Phase 3 data showing 1,200 mg dose reduced risk of hospitalization or death by 70%
Only antibody therapy currently available in all 50 states, including eight states with high rates of two variants of concern
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced the U.S. Food and Drug Administration (FDA) updated the Emergency Use Authorization (EUA) for REGEN-COV™, lowering the dose to 1,200 mg (600 mg casirivimab and 600 mg imdevimab), which is half the dose originally authorized. As part of the updated EUA, REGEN-COV should be administered by intravenous (IV) infusion; subcutaneous (SC) injections are an alternative when IV infusion is not feasible and would lead to a delay in treatment.
"Despite increased use of vaccines, thousands of patients are still becoming infected in the U.S. every day, with many at high risk of serious complications from COVID-19. Unfortunately, to date only a fraction of patients eligible for antibody treatments have received them, which we hope will change based on this updated FDA authorization. REGEN-COV is readily available and supplied free of charge by the U.S. government," said George D. Yancopoulos, M.D., Ph.D., President and Chief Scientific Officer at Regeneron. "REGEN-COV has also demonstrated potency against the main variants of concern to date in vitro and is the only antibody therapy currently available across the U.S., including in states where variants first identified in Brazil and South Africa are circulating at a higher rate."
REGEN-COV is authorized for use under an EUA to treat mild-to-moderate COVID-19 in adults and pediatric patients (12 years of age and older weighing ≥40 kg) with positive results of direct SARS-CoV-2 viral testing, and who are at high risk for progression to severe COVID-19, including hospitalization or death. The Fact Sheet updates remove the previously authorized 2,400 mg IV REGEN-COV dose.
The updated FDA authorization is based on data from several trials, including a recently presented Phase 3 trial which showed REGEN-COV reduced the risk of hospitalization or death by 70% in high-risk non-hospitalized patients, and that the treatment effect was consistent between the 1,200 mg and 2,400 mg doses. The SC administration was authorized based on the totality of scientific evidence, incorporating clinical, viral load reduction and pharmacokinetic data.
In addition, in vitro research has shown that REGEN-COV retains potency against the main variants of concern circulating within the U.S., including the P.1 variant (first identified in Brazil, now classified by the World Health Organization [WHO] as Gamma) and the B.1.351 variant (first identified in South Africa, now classified by the WHO as Beta). Consequently, REGEN-COV remains available for use in all 50 states. The combined frequency of the P.1 and B.1.351 variants now exceeds 10% of new COVID-19 diagnoses across eight states (Arizona, California, Florida, Illinois, Indiana, Massachusetts, Oregon and Washington), and the prevalence of these and other variants continues to be closely monitored.
REGEN-COV Supply Update
The U.S. government will purchase all REGEN-COV doses delivered by June 30, 2021 and may accept additional doses through September 30, 2021 at its discretion, up to a maximum amount of 1.25 million doses. Regeneron expects to deliver at least 1 million REGEN-COV doses to the U.S. government in the second quarter. The specific quantity of doses delivered will be impacted by the timeliness of manufacturing operations. Additionally, Regeneron expects its second quarter 2021 GAAP and non-GAAP effective tax rates to be similar and approximately 17% and the company's full year effective tax rate guidance remains unchanged.
About the REGEN-COV Antibody Cocktail
REGEN-COV (casirivimab and imdevimab) is a cocktail of two monoclonal antibodies (also known as REGN10933 and REGN10987) that was designed specifically to block infectivity of SARS-CoV-2, the virus that causes COVID-19, using Regeneron's proprietary VelocImmune® and VelociSuite® technologies. The two potent, virus-neutralizing antibodies that form the cocktail bind non-competitively to the critical receptor binding domain of the virus's spike protein, which diminishes the ability of mutant viruses to escape treatment and protects against spike variants that have arisen in the human population, as detailed in Science.
Under this EUA, REGEN-COV is available throughout the U.S. – information on availability in your area is available from the Department of Health and Human Services and the National Infusion Center Association. REGEN-COV can be administered by IV infusion (as short as 20 minutes) or by SC injection (four injections), which is an alternative when IV infusion is not feasible and would lead to a delay in treatment. It is now authorized as a co-formulated single vial, or in individual vials to be administered together.
In the U.S., REGEN-COV is not authorized for use in patients who are hospitalized due to COVID-19 or require oxygen therapy, or for people currently using chronic oxygen therapy because of an underlying comorbidity who require an increase in baseline oxygen flow rate due to COVID-19.
Regeneron is collaborating with Roche to increase global supply of REGEN-COV. Regeneron is responsible for development and distribution of the treatment in the U.S., and Roche is primarily responsible for development and distribution outside the U.S. The companies share a commitment to making the antibody cocktail available to COVID-19 patients around the globe and will support access in low- and lower-middle-income countries through drug donations to be made in partnership with public health organizations.
AUTHORIZED USE AND IMPORTANT SAFETY INFORMATION
REGEN-COV, (casirivimab and imdevimab) co-formulated product and REGEN-COV (casirivimab and imdevimab) supplied as individual vials to be administered together, is authorized for the treatment of mild to moderate coronavirus disease 2019 (COVID-19) in adults and pediatric patients (12 years of age and older weighing at least 40 kg) with positive results of direct SARS-CoV-2 viral testing, and who are at high risk for progression to severe COVID-19, including hospitalization or death. [see Limitations of Authorized Use]
- REGEN-COV has not been approved, but has been authorized for emergency use by FDA
- This use is authorized only for the duration of the declaration that circumstances exist justifying the authorization of the emergency use under section 564(b)(1) of the Act, 21 U.S.C. § 360bbb-3(b)(1), unless the authorization is terminated or revoked sooner
- Healthcare providers should review the Fact Sheet for Healthcare Providers for information on the authorized use of REGEN-COV and mandatory requirements of the EUA and must comply with the requirements of the EUA. The FDA Letter of Authorization is available for reference, as well as the Dear Healthcare Provider Letter and Patient Fact Sheet
Limitations of Authorized Use
- REGEN-COV (casirivimab and imdevimab) is not authorized for use in patients:
- who are hospitalized due to COVID-19, OR
- who require oxygen therapy due to COVID-19, OR
- who require an increase in baseline oxygen flow rate due to COVID-19 in those on chronic oxygen therapy due to underlying non-COVID-19 related comorbidity
- Benefit of treatment with REGEN-COV has not been observed in patients hospitalized due to COVID-19. Monoclonal antibodies, such as REGEN-COV, may be associated with worse clinical outcomes when administered to hospitalized patients with COVID-19 requiring high-flow oxygen or mechanical ventilation
For additional information about the company, please visit www.regeneron.com
SOURCE Regeneron Pharmaceuticals, Inc.
FDA updates REGEN-COV EUA to treat patients with COVID-19 at lower dose
Jun. 04, 2021 7:51 AM ET Regeneron Pharmaceuticals, Inc. (REGN)
By: Aakash Babu, SA News Editor3 Comments
- Regeneron Pharmaceuticals (NASDAQ:REGN) announces that the U.S. FDA has updated the Emergency Use Authorization (EUA) for COVID-19 treatment REGEN-COV, lowering the dose to 1,200 mg, which is half the dose originally authorized.
- https://seekingalpha.com/news/3703251-fda-updates-regen-cov-eua-to-treat-patients-with-covid-19-at-lower-dose
- https://seekingalpha.com/symbol/REGN
- https://www.regeneron.com/
- https://www.regencov.com/
KEYTRUDA® (pembrolizumab)
Merck’s KEYTRUDA® (pembrolizumab) Given After Surgery Reduced the Risk of Disease Recurrence or Death by 32% Versus Placebo as Adjuvant Therapy in Patients With Renal Cell Carcinoma (RCC)
June 3, 2021 5:00 pm ET
KEYNOTE-564 is the First Phase 3 Study to Show Positive Results for Adjuvant Immunotherapy in RCC
First-Time Disease-Free Survival Data to be Presented During Plenary Session at the 2021 ASCO Annual Meeting
KENILWORTH, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced first-time results from the pivotal Phase 3 KEYNOTE-564 trial evaluating KEYTRUDA, Merck’s anti-PD-1 therapy, for the potential adjuvant treatment of patients with renal cell carcinoma (RCC) at intermediate-high or high risk of recurrence following nephrectomy (surgical removal of a kidney) or following nephrectomy and resection of metastatic lesions. After a median follow-up of 24.1 months (14.9-41.5), KEYTRUDA demonstrated a statistically significant and clinically meaningful reduction in the risk of disease recurrence or death by 32% compared to placebo (HR=0.68 [95% CI, 0.53–0.87]; p=0.0010). Additionally, a favorable trend in overall survival (OS) was observed with a 46% reduction in the risk of death with KEYTRUDA as compared to placebo (HR=0.54 [95% CI, 0.30–0.96]; p=0.0164). As previously announced, the trial will continue to evaluate OS, a key secondary endpoint.
“With the results of KEYNOTE-564, pembrolizumab is the first immunotherapy to show a clinical benefit in the adjuvant setting in kidney cancer,” said Dr. Toni K. Choueiri, director of the Lank Center for Genitourinary Oncology, Dana-Farber Cancer Institute and professor of medicine at Harvard Medical School. “It took several decades to achieve this milestone. We hope to build on this important research and provide new treatment options to kidney cancer patients.”
Merck is continuing to study KEYTRUDA, in combination or as monotherapy, as well as other investigational products across multiple settings and stages of RCC including adjuvant and advanced or metastatic disease through our broad clinical development program, which includes over 20 clinical studies and more than 4,000 patients.
KEYTRUDA is currently approved in the U.S., Europe and Japan in combination with axitinib for the first-line treatment of patients with advanced RCC.
Study Design and Additional Data from KEYNOTE-564
KEYNOTE-564 is a randomized, double-blind, Phase 3 trial (ClinicalTrials.gov, NCT03142334) evaluating KEYTRUDAmonotherapy versus placebo for the adjuvant treatment of patients with RCC who have undergone nephrectomy and who have intermediate-high risk, high risk, or M1 no evidence of disease (M1 NED) RCC with clear cell component. The study enrolled 994 patients who were randomized to receive either KEYTRUDA (200 mg intravenously [IV] on Day 1 of each three-week cycle for up to 17 cycles) or placebo (saline solution IV on Day 1 of each three-week cycle for up to 17 cycles). The primary endpoint is disease-free survival (DFS), and the secondary endpoints include OS and safety.
As of data cutoff (Dec. 14, 2020), the median study follow-up was 24.1 months. Findings showed KEYTRUDA demonstrated a statistically significant improvement in DFS in patients with RCCfollowing nephrectomy or following nephrectomy and resection of metastatic lesions compared with placebo (HR=0.68 [95% CI, 0.53–0.87]; p=0.0010). Additionally, the two-year estimated DFS rate was 77.3% with KEYTRUDA versus 68.1% with placebo. Overall, the DFS benefit was consistent across subgroups. Median DFS was not achieved in either treatment arm based on event accrual.
Grade 3-5 treatment-related adverse events (TRAEs) occurred in 18.9% of patients in the KEYTRUDA arm and 1.2% of patients in the placebo arm. TRAEs resulting in discontinuation of any treatment occurred in 17.6% of patients in the KEYTRUDA arm and 0.6% of patients in the placebo arm. The most common TRAEs of any grade (occurring in ≥5% of patients) were fatigue (20.3%), pruritus (18.6%) and hypothyroidism (17.6%) in the KEYTRUDA arm and fatigue (14.3%), pruritus (11.5%) and diarrhea (10.3%) in the placebo arm. The most common immune-mediated adverse events of any grade (occurring in ≥3% of patients) were hypothyroidism (21.1%) and hyperthyroidism (11.9%) in the KEYTRUDA arm and hypothyroidism (3.6%) in the placebo arm. No treatment-related deaths occurred.
About KEYTRUDA® (pembrolizumab) Injection, 100 mg
KEYTRUDA is an anti-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 1,400 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.
Selected KEYTRUDA® (pembrolizumab) Indications in the U.S.
Melanoma
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.
KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.
Non-Small Cell Lung Cancer
KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) ≥1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is stage III where patients are not candidates for surgical resection or definitive chemoradiation, or metastatic.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS ≥1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.
Head and Neck Squamous Cell Cancer
KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS) ≥1] as determined by an FDA-approved test.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.
Classical Hodgkin Lymphoma
KEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).
KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.
Primary Mediastinal Large B-Cell Lymphoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.
Urothelial Carcinoma
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 (CPS ≥10), as determined by an FDA-approved test, or in patients who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.
Microsatellite Instability-High or Mismatch Repair Deficient Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR)
- solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options, or
- colorectal cancer that has progressed following treatment with fluoropyrimidine, oxaliplatin, and irinotecan.
This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.
Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer
KEYTRUDA is indicated for the first-line treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).
Gastric Carcinoma
KEYTRUDA, in combination with trastuzumab, and fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of patients with locally advanced unresectable or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent locally advanced or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test, with disease progression on or after two or more prior lines of therapy including fluoropyrimidine- and platinum-containing chemotherapy and if appropriate, HER2/neu-targeted therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Esophageal Carcinoma
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic esophageal or gastroesophageal junction (GEJ) (tumors with epicenter 1 to 5 centimeters above the GEJ) carcinoma that is not amenable to surgical resection or definitive chemoradiation either:
- in combination with platinum- and fluoropyrimidine-based chemotherapy, or
- as a single agent after one or more prior lines of systemic therapy for patients with tumors of squamous cell histology that express PD L1 (CPS ≥10) as determined by an FDA approved test.
Cervical Carcinoma
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Hepatocellular Carcinoma
KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Merkel Cell Carcinoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Renal Cell Carcinoma
KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).
Tumor Mutational Burden-High
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [≥10 mutations/megabase] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.
Cutaneous Squamous Cell Carcinoma
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) that is not curable by surgery or radiation.
Triple-Negative Breast Cancer
KEYTRUDA, in combination with chemotherapy, is indicated for the treatment of patients with locally recurrent unresectable or metastatic triple-negative breast cancer (TNBC) whose tumors express PD-L1 (CPS ≥10) as determined by an FDA-approved test. This indication is approved under accelerated approval based on progression-free survival. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
For more information, visit www.merck.com
Please see Prescribing Information for KEYTRUDA (pembrolizumab) at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_pi.pdf and Medication Guide for KEYTRUDA at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_mg.pdf.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210603006033/en/
Source: Merck & Co., Inc.
Merck Keytruda reduces kidney cancer return risk by 32% when given after surgery
Jun. 03, 2021 6:42 PM ET Merck & Co., Inc. (MRK) By: Jonathan M Block, SA News Editor7 Comments
- After surgery, Merck's (NYSE:MRK) Keytruda (pembrolizumab) can reduce the risk of renal cell carcinoma returning or death by nearly one-third, new data shows.
- https://seekingalpha.com/news/3703169-merck-keytruda-reduces-renal-cell-carcinoma-return-risk-by-32-when-given-after-surgery
- https://seekingalpha.com/symbol/MRK
- https://www.merck.com/
- https://www.keytruda.com/
Opdivo plus Chemotherapy and Opdivo plus Yervoy
Bristol Myers Squibb Presents Data from CheckMate -648 Showing Opdivo plus Chemotherapy and Opdivo plus Yervoy Significantly Improved Overall Survival Compared to Chemotherapy in Unresectable Advanced or Metastatic Esophageal Squamous Cell Carcinoma
06/03/2021CATEGORY:
Opdivo demonstrated significant overall survival benefit over chemotherapy alone in both PD-L1 positive and all-randomized populations in both treatment arms
CheckMate -648 marks the third global trial in which Opdivo demonstrated a significant benefit for patients with upper gastrointestinal cancers
Data to be featured in an oral presentation during the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting
Not intended for media in the UK & Ireland
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced results from the Phase 3 CheckMate -648 trial, in which two Opdivo-based treatment combinations — Opdivo (nivolumab)plus chemotherapy and Opdivo plus Yervoy (ipilimumab) —demonstrated a statistically significant and clinically meaningful overall survival (OS) benefit compared to chemotherapy at the pre-specified interim analysis in patients with unresectable advanced or metastatic esophageal squamous cell carcinoma (ESCC) with tumor cell PD-L1 expression ≥1%, as well as in the all-randomized population. Opdivo plus Yervoy is the first dual immunotherapy combination to demonstrate a superior survival benefit versus chemotherapy in this setting. The data will be presented in an oral session on Saturday, June 5, 2021 from 1:45 p.m. to 4:45 p.m. EDT and featured in the official press program during the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting.
For the combination of Opdivo plus chemotherapy, median OS was 15.4 months vs. 9.1 months for chemotherapy in patients whose tumors express PD-L1, a primary endpoint (hazard ratio [HR] 0.54, 99.5% CI: 0.37-0.80, p<0.0001), and 13.2 months vs. 10.7 months in the all-randomized patient population, a secondary endpoint (HR 0.74, 99.1% CI: 0.58-0.96, p=0.0021). A statistically significant progression-free survival (PFS) benefit was also observed with Opdivo plus chemotherapy in patients whose tumors express PD-L1, with a median PFS by blinded independent central review (BICR) of 6.9 months compared to 4.4 months with chemotherapy alone (HR 0.65, 98.5% CI: 0.46-0.92, p=0.0023).
For the combination of Opdivo plus Yervoy, median OS was 13.7 months vs. 9.1 months for chemotherapy in patients whose tumors express PD-L1, a primary endpoint (HR 0.64, 98.6% CI: 0.46-0.90, p=0.001), and 12.8 months vs. 10.7 months, respectively, in the all-randomized patient population, a secondary endpoint (HR 0.78, 98.2% CI: 0.62-0.98, p=0.011). Opdivo plus Yervoy did not meet its other primary endpoint of PFS by BICR in patients whose tumors express PD-L1 (4.0 months vs. 4.4 months; HR 1.02, 98.5% CI: 0.73-1.43, p=0.8958).
About CheckMate -648
CheckMate -648 is a randomized Phase 3 study evaluating Opdivo plus Yervoy or Opdivo plus fluorouracil and cisplatin against fluorouracil plus cisplatin alone in patients with unresectable advanced or metastatic esophageal squamous cell carcinoma.
The primary endpoints of the trial are overall survival (OS) and progression-free survival (PFS) by blinded independent central review (BICR) in patients whose tumors express PD-L1 ≥1% for both Opdivo-based combinations versus chemotherapy. Secondary endpoints of the trial include OS and PFS by BICR in the all-randomized population.
In the Opdivo plus chemotherapy arm (N=321), patients received treatment with Opdivo 240 mg on Day 1 and Day 15, fluorouracil 800 mg/m²/day on Day 1 through Day 5 (for 5 days), and cisplatin 80 mg/m² on Day 1 of four-week cycle. Patients received Opdivo for up to 24 months or until disease progression or unacceptable toxicity, and chemotherapy until disease progression or unacceptable toxicity.
In the Opdivo plus Yervoy arm (N=325), patients received treatment with Opdivo 3 mg/kg every 2 weeks and Yervoy 1 mg/kg every 6 weeks up to 24 months or until disease progression or unacceptable toxicity.
About Opdivo
Opdivo is a programmed death-1 (PD-1) immune checkpoint inhibitor that is designed to uniquely harness the body’s own immune system to help restore anti-tumor immune response. By harnessing the body’s own immune system to fight cancer, Opdivo has become an important treatment option across multiple cancers.
Opdivo’s leading global development program is based on Bristol Myers Squibb’s scientific expertise in the field of Immuno-Oncology, and includes a broad range of clinical trials across all phases, including Phase 3, in a variety of tumor types. To date, the Opdivo clinical development program has treated more than 35,000 patients. The Opdivo trials have contributed to gaining a deeper understanding of the potential role of biomarkers in patient care, particularly regarding how patients may benefit from Opdivo across the continuum of PD-L1 expression.
In July 2014, Opdivo was the first PD-1 immune checkpoint inhibitor to receive regulatory approval anywhere in the world. Opdivo is currently approved in more than 65 countries, including the United States, the European Union, Japan and China. In October 2015, the Company’s Opdivo and Yervoy combination regimen was the first Immuno-Oncology to receive regulatory approval for the treatment of metastatic melanoma and is currently approved in more than 50 countries, including the United States and the European Union.
About Yervoy
Yervoy is a recombinant, human monoclonal antibody that binds to the cytotoxic T-lymphocyte-associated antigen-4 (CTLA-4). CTLA-4 is a negative regulator of T-cell activity. Yervoy binds to CTLA-4 and blocks the interaction of CTLA-4 with its ligands, CD80/CD86. Blockade of CTLA-4 has been shown to augment T-cell activation and proliferation, including the activation and proliferation of tumor infiltrating T-effector cells. Inhibition of CTLA-4 signaling can also reduce T-regulatory cell function, which may contribute to a general increase in T-cell responsiveness, including the anti-tumor immune response. On March 25, 2011, the U.S. Food and Drug Administration (FDA) approved Yervoy 3 mg/kg monotherapy for patients with unresectable or metastatic melanoma. Yervoy is approved for unresectable or metastatic melanoma in more than 50 countries. There is a broad, ongoing development program in place for Yervoy spanning multiple tumor types.
INDICATIONS
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express PD-L1 (≥1%) as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab) and 2 cycles of platinum-doublet chemotherapy, is indicated for the first-line treatment of adult patients with metastatic or recurrent non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab) is indicated for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving OPDIVO.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with unresectable malignant pleural mesothelioma (MPM).
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of patients with intermediate or poor risk advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab), in combination with cabozantinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab) is indicated for the treatment of patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy.
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with classical Hodgkin lymphoma (cHL) that has relapsed or progressed after autologous hematopoietic stem cell transplantation (HSCT) and brentuximab vedotin or after 3 or more lines of systemic therapy that includes autologous HSCT. This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab) is indicated for the treatment of patients with recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN) with disease progression on or after platinum-based therapy.
OPDIVO® (nivolumab) is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of adult and pediatric (12 years and older) patients with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab) is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection.
OPDIVO® (nivolumab) is indicated for the treatment of patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) after prior fluoropyrimidine- and platinum-based chemotherapy.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of completely resected esophageal or gastroesophageal junction cancer with residual pathologic disease in patients who have received neoadjuvant chemoradiotherapy (CRT).
OPDIVO® (nivolumab), in combination with fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the treatment of patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma.
Please see US Full Prescribing Information for OPDIVO and YERVOY.
For more information about Bristol Myers Squibb, visit us at BMS.com
Bristol Opdivo + Yervoy combo improves survival in esopageal squamous cell carcinoma
Jun. 03, 2021 6:22 PM ET Bristol-Myers Squibb Company (BMY) By: Jonathan M Block, SA News Editor1 Comment
- Bristol-Myers Squibb's (NYSE:BMY) combination of Opdivo (nivolumab) + Yervoy (ipilimumab) demonstrated better overall survival ("OS") compared to chemotherapy in a phase 3 trial of patients with unresectable advanced or metastatic esophageal squamous cell carcinoma.
- https://seekingalpha.com/news/3703168-bristol-opdivo-yervoy-combo-improves-survival-in-esopageal-squamous-cell-carcinoma
- https://seekingalpha.com/symbol/BMY
- https://www.bms.com/
- https://www.opdivo.com/about-opdivo/how-the-combination-works-combinationtherapy
biotecMAX™ April/May/June 2021 Insight
Kisqali® (ribociclib)
Novartis Kisqali® reports longest median overall survival in postmenopausal HR+/HER2- metastatic breast cancer patients
Jun 02, 2021
- MONALEESA-3 median overall survival (OS) results of 53.7 months underscore that Kisqali offers more life to postmenopausal women with HR+/HER2- metastatic breast cancer (MBC) in addition to the OS benefit demonstrated for premenopausal women as shown in MONALEESA-71,2
- The relative risk reduction of death by 36% in the MONALEESA-3 first-line (1L) postmenopausal population highlights that Kisqali is the only CDK4/6i with proven OS for 1L in combination with fulvestrant1
- Time to chemotherapy was delayed to 4 years (48.1 months) in postmenopausal women taking Kisqali in combination with fulvestrant compared to 2.4 years (28.8 months) for women receiving fulvestrant only1
- MBC takes a life in the US approximately every 12 minutes, creating an urgent need for treatment proven to extend life while preserving quality of life3-6
Basel, June 2, 2021 — Novartis today announced updated median overall survival (OS) results for Kisqali® (ribociclib) in combination with fulvestrant in postmenopausal women with hormone receptor positive, human epidermal growth factor receptor-2 negative (HR+/HER2-) metastatic breast cancer. The exploratory analysis of OS after an additional 16.9 months of follow-up of the Phase III MONALEESA-3 trial evaluated Kisqali plus fulvestrant as first- or second-line treatment compared to fulvestrant alone in postmenopausal women with HR+/HER2- metastatic breast cancer1. The analysis found that with extended follow-up of more than four years, Kisqali in combination with fulvestrant continued to demonstrate a clinically relevant OS benefit of more than a year compared with fulvestrant alone1. These updated median OS data (Abstract #1001) will be presented in an oral presentation at the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting.
Visit https://www.hcp.novartis.com/virtual-congress/a-2021/ for the latest information from Novartis, including our commitment to the Oncology community, and access to our ASCO21 Virtual Scientific Program data presentations (for registered participants).
Kisqali® (ribociclib)
About Kisqali® (ribociclib)
Kisqali was initially approved by the US Food and Drug Administration (FDA) in March 2017 and by the European Commission (EC) in August 2017, as initial endocrine-based therapy for postmenopausal women with HR+/HER2- locally advanced or metastatic breast cancer in combination with an aromatase inhibitor based on findings from the pivotal MONALEESA-2 trial. Kisqali in combination with an aromatase inhibitor was approved for the treatment of pre-, peri- or postmenopausal women as initial endocrine-based therapy, and also indicated for use in combination with fulvestrant as both first- or second-line therapy in postmenopausal women by the FDA in July 2018 and by the EC in December 2018. Regulatory filings are underway with other health authorities worldwide.
Kisqali was developed by the Novartis Institutes for BioMedical Research (NIBR) under a research collaboration with Astex Pharmaceuticals.
https://www.us.kisqali.com/metastatic-breast-cancer/
Please see full Prescribing Information for Kisqali, available at www.Kisqali.com
Find out more at https://www.novartis.com.
Novartis' Kisqali shows long-term survival benefit in metastatic breast cancer
Jun. 02, 2021 10:06 AM ETNovartis AG (NVS)By: Jonathan M Block, SA News Editor
- Novartis' (NYSE:NVS) Kisqali (ribociclib) demonstrated long-term overall survival benefit in postmenopausal women with hormone receptor positive, human epidermal growth factor receptor-2 negative (HR+/HER2-) metastatic breast cancer.
- https://seekingalpha.com/news/3702336-novartis-kisqali-shows-long-term-survival-benefit-in-metastatic-breast-cancer
- https://seekingalpha.com/symbol/NVS
- https://www.us.kisqali.com/metastatic-breast-cancer/
- https://www.novartis.com/
ORLADEYO™ (berotralstat)
BioCryst Launches ORLADEYO™ (berotralstat) in Germany
RESEARCH TRIANGLE PARK, N.C., June 03, 2021 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that oral, once-daily ORLADEYO™ (berotralstat) is now available for patients with a prescription in Germany.
ORLADEYO was approved by the European Medicines Agency (EMA) on April 30, 2021 for the prevention of recurrent hereditary angioedema (HAE) attacks in HAE patients 12 years and older. The full European Summary of Product Characteristics (SMPC) for ORLADEYO is available on the EMA website at www.ema.europa.eu.
“We have an experienced team in place in Germany that is excited and honored to bring the first oral, once daily therapy to HAE patients in Europe,” said Charlie Gayer, chief commercial officer of BioCryst. “Patients and physicians across Europe have told us about the significant need for a targeted oral therapy for HAE, and Germany is the first of many upcoming European launches for ORLADEYO.”
About ORLADEYO™ (berotralstat)
ORLADEYO™ (berotralstat) is the first and only oral therapy designed specifically to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years and older. One capsule of ORLADEYO per day works to prevent HAE attacks by decreasing the activity of plasma kallikrein.
For more information, please visit the company’s website at www.biocryst.com.
https://www.biocryst.com/orladeyo/
BioCryst launches ORLADEYO in Germany
Jun. 03, 2021 12:35 PM ET BioCryst Pharmaceuticals, Inc. (BCRX)
By: Aakash Babu, SA News Editor2 Comments
- BioCryst Pharmaceuticals (BCRX +0.4%) announces that oral, once-daily ORLADEYO (berotralstat) is now available for patients with a prescription in Germany.
- ORLADEYO was approved by the European Medicines Agency (EMA) for the prevention of recurrent hereditary angioedema (HAE) attacks in HAE patients 12 years and older, in April.
- https://seekingalpha.com/news/3702989-biocryst-launches-orladeyo-in-germany
- https://www.biocryst.com/
- https://orladeyo.com/
XPOVIO® (selinexor)
Karyopharm Announces XPOVIO® (selinexor) Is Now Available in Additional Strength Tablets-- Three New Strength Tablets Approved by the FDA: 40 mg, 50 mg and 60 mg ---- New Tablet Strengths Provide Additional Patient Convenience and Dosing Flexibility --
NEWTON, Mass., June 3, 2021 /PRNewswire/ -- Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, today announced that three new strength tablets for XPOVIO, the Company's first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) medicine, are now commercially available. The availability of these additional strength tablets follows a U.S. Food & Drug Administration (FDA) approval on April 15, 2021. In addition to the original 20 mg strength tablets, XPOVIO is now available in 40 mg, 50 mg, and 60 mg strength tablets. XPOVIO tablets are available in seven different package sizes to help healthcare providers individualize the dosing and administration of XPOVIO based on patient needs. The additional dosage strength tablets may also increase patient compliance by simplifying their treatment regimen and reducing the pill burden experienced by some patients. With the introduction of these new tablet strengths and additional package sizes, there is no change to the efficacy and safety profile of XPOVIO.
About XPOVIO® (selinexor)
XPOVIO is a first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) compound. XPOVIO functions by selectively binding to and inhibiting the nuclear export protein exportin 1 (XPO1, also called CRM1). XPOVIO 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. Karyopharm received accelerated U.S. Food and Drug Administration (FDA) approval of XPOVIO in July 2019 in combination with dexamethasone for the treatment of adult patients with relapsed refractory multiple myeloma (RRMM) who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody. NEXPOVIO® (selinexor) has also been granted conditional marketing authorization for adult patients with heavily pretreated multiple myeloma by the European Commission. Karyopharm's supplemental New Drug Application (sNDA) requesting an expansion of its indication to include the treatment for patients with multiple myeloma after at least one prior therapy was approved by the FDA on December 18, 2020. In June 2020, Karyopharm received accelerated FDA approval of XPOVIO for its second indication in adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), not otherwise specified, including DLBCL arising from follicular lymphoma, after at least 2 lines of systemic therapy. Selinexor is also being evaluated in several other mid-and later-phase clinical trials across multiple cancer indications, including as a potential backbone therapy in combination with approved myeloma therapies (STOMP) and in endometrial cancer (SIENDO), among others. Additional Phase 1, Phase 2 and Phase 3 studies are ongoing or currently planned, including multiple studies in combination with approved therapies in a variety of tumor types to further inform Karyopharm's clinical development priorities for selinexor. Additional clinical trial information for selinexor is available at www.clinicaltrials.gov.
For more information about Karyopharm's products or clinical trials, please contact the Medical Information department at:
Tel: +1 (888) 209-9326
Email: medicalinformation@karyopharm.com
For additional product information, including full prescribing information, please visit www.XPOVIO.com.
For more information, please visit www.karyopharm.com.
Karyopharm's XPOVIO now available in additional strength tablets
Jun. 03, 2021 12:28 PM ETKaryopharm Therapeutics Inc. (KPTI)By: Aakash Babu, SA News Editor
- Karyopharm Therapeutics (KPTI -1.2%) announces that three new strength tablets for XPOVIO, the company's oral Selective Inhibitor of Nuclear Export (SINE) medicine, are now commercially available.
- https://seekingalpha.com/news/3702986-karyopharms-xpovio-now-available-in-additional-strength-tablets
- https://seekingalpha.com/symbol/KPTI
- https://www.karyopharm.com/
- https://www.karyopharm.com/pipeline/oral-selinexor/
177Lu-PSMA-617
Novartis 177Lu-PSMA-617 significantly improves overall survival and radiographic progression-free survival for men with metastatic castration-resistant prostate cancer in Phase III VISION study
Jun 03, 2021
- Men who received 177Lu-PSMA-617 plus best standard of care had a 38% reduction in risk of death (median OS benefit of 4 months) and a 60% reduction in the risk of radiographic disease progression or death (median rPFS benefit of 5 months) compared to best standard of care alone1
- Significant improvement demonstrated in all key secondary endpoints, including time to first symptomatic skeletal event, overall response rate and disease control rate1
- VISION study findings to be presented during 2021 ASCO plenary; regulatory submissions to US and EU Health Authorities on track for 2H21; two additional pivotal studies in earlier lines of treatment for metastatic prostate cancer to start 1H21, goal to move into earlier stages of disease
- Novartis commitment to leadership in radioligand therapy (RLT) further strengthened by recent partnerships and investments; more than 15 ongoing research and discovery programs to identify and accelerate next wave of RLTs for cancer
Basel, June 3, 2021 — Novartis today announced that results of the Phase III VISION study evaluating 177Lu-PSMA-617, a targeted radioligand therapy, plus best standard of care (SOC) demonstrated significant improvement in overall survival (OS) compared to SOC alone, in patients with progressive PSMA-positive metastatic castration-resistant prostate cancer (mCRPC)1. The difference in OS between study arms was statistically significant (one-sided p<0.001), with an estimated 38% reduction in risk of death in the 177Lu-PSMA-617 arm (n=551) compared to the best standard of care only arm (n=280) (hazard ratio: 0.62 with 95% confidence interval (CI): (0.52, 0.74))1. These results will be presented during the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting plenary session on June 6.
Patients receiving 177Lu-PSMA-617 also demonstrated a statistically significant (one-sided p<0.001) 60% risk reduction for radiographic progression-free survival or death (rPFS), compared to the best standard of care only arm (hazard ratio: 0.40 with 99.2% CI: (0.29 0.57))1. There was a higher rate of drug-related treatment emergent adverse events reported in the 177Lu-PSMA-617 treatment arm (85.3%) compared to standard of care alone (28.8%)1.
Across both arms of the study, rates of treatment discontinuation associated with treatment-emergent adverse events occurred as follows: In the 177Lu-PSMA-617 plus standard of care (SOC) arm, 11.9% of patients discontinued 177Lu-PSMA-617 and 8.5% discontinued SOC; while in the SOC alone arm 7.8% of patients discontinued treatment1.
Visit https://www.hcp.novartis.com/virtual-congress/a-2021/ for the latest information from Novartis, including our commitment to the Oncology community, and access to our ASCO21 Virtual Scientific Program data presentations (for registered participants).
177Lu-PSMA-617
About 177Lu-PSMA-617
177Lu-PSMA-617 is an investigational PSMA-targeted radioligand therapy for metastatic castration-resistant prostate cancer. It is a type of precision cancer treatment combining a targeting compound (ligand) with a therapeutic radioisotope (a radioactive particle)11-13. After administration into the bloodstream, 177Lu-PSMA-617 binds to prostate cancer cells that express PSMA14, a transmembrane protein, with high tumor-to-normal tissue uptake11,15,16. Once bound, emissions from the radioisotope damage tumor cells, disrupting their ability to replicate and/or triggering cell death17-19. The radiation from the radioisotope works over very short distances to limit damage to surrounding cells10,11,15.
About VISION
VISION is an international, prospective, randomized, open-label, multicenter, phase III study to assess the efficacy and safety of 177Lu-PSMA-617 (7.4 GBq administered by intravenous infusion every 6 weeks for a maximum of 6 cycles) plus investigator-chosen best standard of care in the investigational arm, versus best standard of care in the control arm20. Patients with PSMA PET-scan positive mCRPC, and progression after prior taxane and androgen receptor pathway inhibitors, were randomized in a 2:1 ratio in favor of the investigational arm20. The alternate primary endpoints were rPFS and OS20. The study enrolled 831 patients1.
Find out more at https://www.novartis.com.
Novartis' Lutathera demonstrates survival benefit in prostate cancer
Jun. 03, 2021 12:47 PM ET Novartis AG (NVS) By: Jonathan M Block, SA News Editor
- Novartis' (NYSE:NVS) peptide receptor radionuclide therapy Lutathera (177Lu-PSMA-617) significantly improved overall survival ("OS") and progression-free survival in a phase 3 study of prostate cancer patients.
- https://seekingalpha.com/news/3702992-novartis-lutathera-demonstrates-survival-benefit-in-prostate-cancer
- https://seekingalpha.com/symbol/NVS
What is LUTATHERA?
LUTATHERA is a prescription medicine used to treat adults with a type of cancer known as gastroenteropancreatic neuroendocrine tumors (GEP-NETs) that are positive for the hormone receptor somatostatin, including GEP-NETs in the foregut, midgut, and hindgut.
TYVYT® (sintilimab injection) in Combination with Gemcitabine and Platinum Chemotherapy
Innovent and Lilly Jointly Announce the China NMPA Approval of TYVYT® (sintilimab injection) in Combination with Gemcitabine and Platinum Chemotherapy as First-Line Therapy for People with Squamous Non-Small Cell Lung CancerPublished on: Jun 3rd, 2021Back
SAN FRANCISCO, U.S. and SUZHOU, China, June 3, 2021 — Innovent Biologics, Inc. (Innovent) (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high-quality medicines for the treatment of cancer, metabolic, autoimmune and other major diseases, and Eli Lilly and Company (NYSE: LLY) today jointly announced that the National Medical Products Administration (NMPA) of China has approved the supplemental New Drug Application (sNDA) for TYVYT® (sintilimab injection) in combination with gemcitabine and platinum chemotherapy as first-line therapy for people with unresectable locally advanced or metastatic squamous non-small cell lung cancer (sqNSCLC).
This is the third NMPA-approved indication of TYVYT® (sintilimab injection), following the approval in December 2018 for the treatment of relapsed or refractory classical Hodgkin's lymphoma, and the approval in February 2021 for the first-line treatment of nonsquamous non-small cell lung cancer (nsqNSCLC). This is also the first regulatory approval of a PD-1/PD-L1 immunotherapy in combination with gemcitabine and platinum chemotherapy for the first-line treatment for people with sqNSCLC.
This new approval was based on a randomized, double-blind, Phase 3 clinical trial (ORIENT-12) which evaluated TYVYT® (sintilimab injection) or placebo in combination with GEMZAR® (gemcitabine) and platinum chemotherapy as first-line therapy for people with unresectable locally advanced or metastatic sqNSCLC.
TYVYT® (sintilimab injection) in Combination with Gemcitabine and Platinum Chemotherapy
About ORIENT-12
ORIENT-12 （ClinicalTrials.gov, NCT03629925）is a randomized, double-blind, Phase 3 clinical trial evaluating TYVYT® (sintilimab injection) or placebo in combination with GEMZAR® (gemcitabine) and platinum chemotherapy (carboplatin or cisplatin) as a first-line treatment for advanced or metastatic squamous non-small cell lung cancer (sqNSCLC). A total of 357 participants were enrolled and randomized in a 1:1 ratio (179 participants in the TYVYT® (sintilimab injection) combination arm, 178 participants in the placebo combination arm).
ORIENT-12 demonstrated a statistically significant improvement in progression-free survival (PFS), the study’s primary endpoint, of TYVYT® (sintilimab injection) in combination with GEMZAR® and platinum chemotherapy compared with placebo in combination with GEMZAR® and platinum chemotherapy. The median PFS of the TYVYT® (sintilimab injection) combination arm was 5.5 months compared to 4.9 months on the placebo combination arm (HR=0.536, 95% CI: 0.422-0.681, P< 0.00001) as assessed by Independent Radiographic Review Committee (IRRC), and 6.7 months compared to 4.9 months respectively (HR=0.532, 95% CI: 0.419-0.674, P< 0.00001), as assessed by the study’s investigators. Overall survival (OS), a secondary endpoint, of the TYVYT® (sintilimab injection) combination arm showed a positive trend when compared to the placebo combination arm (HR=0.567, 95%CI: 0.353-0.909, P=0.01701), though the data were not yet mature at an interim analysis. The safety profile is consistent with previously reported TYVYT® (sintilimab injection) studies, and no new safety signals were identified.
About Sintilimab
Sintilimab, marketed as TYVYT® (sintilimab injection) in China, is an innovative PD-1 inhibitor with global quality standards jointly developed by Innovent and Lilly. 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 for three indications, 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 nonsquamous non-small cell lung cancer
- In combination with gemcitabine and platinum chemotherapy, for the first-line treatment of squamous non-small cell lung cancer
Additionally, Innovent currently has regulatory submissions under review in China for sintilimab:
- In combination with BYVASDA® (bevacizumab injection) for the first-line treatment of hepatocellular carcinoma
- The second-line treatment of squamous non-small cell lung cancer
In May 2021, the U.S. FDA accepted for review the Biologics License Application (BLA) for sintilimab in combination with pemetrexed and platinum chemotherapy for the first-line treatment of nonsquamous non-small cell lung cancer.
Sintilimab was included in China’s National Reimbursement Drug List (NRDL) in 2019 as the first PD-1 inhibitor and the only PD-1 included in the list in that year.
For more information, please visit: www.innoventbio.com.
To learn more about Lilly, please visit lilly.comand lilly.com/newsroom.
About Innovent Biologics’ strategic collaboration with Eli Lilly and Company
Innovent entered into a strategic collaboration with Lilly focused on biological medicine in March 2015 – a groundbreaking partnership between a Chinese pharmaceutical company and a multinational pharmaceutical company. Under the agreement, Innovent and Lilly are co-developing and commercializing oncology medicines, including TYVYT® (sintilimab injection), in China. In October 2015, the two companies announced the extension of their existing collaboration to include co-development of three additional oncology antibodies targeting oncology indications. In August 2019, Innovent further entered into a licensing agreement with Lilly to develop and commercialize a potentially global best-in-class diabetes medicine in China. Its collaboration with Lilly is an example of how Innovent has established a comprehensive level of cooperation between China’s innovative pharmaceuticals sector and the international pharmaceuticals sector in fields such as R&D, CMC, clinical development and commercialization. In August 2020，Lilly and Innovent announced a global expansion of their strategic alliance for sintilimab, whereby Lilly obtained an exclusive license for sintilimab for geographies outside of China and plans to pursue registration of sintilimab in the U.S. and other geographies outside of China.
Note:
[1] The PFS and OS data in China approved indication label for TYVYT® (sintilimab injection).
TYVYT® (sintilimab injection; Innovent), BYVASDA® (bevacizumab biosimilar injection; Innovent), SULINNO® (adalimumab biosimilar injection; Innovent), and HALPRYZA® (rituximab biosimilar injection; Innovent) are not approved products in the United States.
Lilly, Innovent Bio's Tyvyt combo OK'd in China for lung cancer
Jun. 03, 2021 5:13 AM ET Eli Lilly and Company (LLY), IVBIY
By: Mamta Mayani, SA News Editor1 Comment
- Eli Lilly (NYSE:LLY) and Innovent Biologics' (OTCPK:IVBIY) supplemental New Drug Application (sNDA) for TYVYT (sintilimab injection) in combination with gemcitabine and platinum chemotherapy has been approved in China as first-line therapy for people with unresectable locally advanced or metastatic squamous non-small cell lung cancer (sqNSCLC).
- https://seekingalpha.com/news/3702755-lilly-innovent-bios-tyvyt-combo-okd-in-china-for-lung-cancer
- https://seekingalpha.com/symbol/IVBIY
- https://www.innoventbio.com/InvestorsAndMedia/StockInformation
- https://www.hkex.com.hk/Market-Data/Securities-Prices/Equities/Equities-Quote?sym=1801&sc_lang=en
- https://seekingalpha.com/symbol/LLY
- https://www.lilly.com/
TUKYSA® (tucatinib)
Seagen Announces Long-Term Results from TUKYSA® (tucatinib) Pivotal Trial in Patients with HER2-Positive Breast Cancer During the Virtual Scientific Program of the 2021 ASCO Annual Meeting
06/03/2021
- Updated Analysis Shows Median Overall Survival for TUKYSA Arm Extended to Two Years, with Benefit Maintained Across All Prespecified Patient Subgroups in HER2CLIMB Trial -
- Results Further Support TUKYSA as Well-Tolerated Treatment Option That Improves Survival in Patients with Previously Treated Metastatic HER2-Positive Breast Cancer With and Without Brain Metastases -
BOTHELL, Wash.--(BUSINESS WIRE)-- Seagen Inc. (Nasdaq:SGEN) today announced that improvements in overall survival (OS) and progression-free survival (PFS) were maintained with long-term follow up from the pivotal HER2CLIMB trial evaluating the addition of TUKYSA® (tucatinib) to trastuzumab and capecitabine in patients with HER2-positive metastatic breast cancer (MBC) with and without brain metastases. Data from the pre-specified exploratory analysis will be presented (Abstract #1043) as part of the virtual scientific program of the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting.
The overall survival benefit seen with TUKYSA at the time of the primary analysis was maintained after an additional 15.6 months of follow-up (total of 29.6 months), demonstrating a 5.5-month improvement in median OS (24.7 months (95% CI: 21.6, 28.9) for the TUKYSA regimen vs. 19.2 months (95% CI: 16.4, 21.4) for capecitabine and trastuzumab alone). This benefit continued to be generally consistent across pre-specified patient subgroups.
“These results support the significant impact that a TUKYSA regimen can have for patients with HER2-positive metastatic breast cancer,” said Roger D. Dansey, M.D., Chief Medical Officer of Seagen. “The long-term data from the HER2CLIMB trial showed that the survival benefit was sustained with median overall survival longer than that observed at the primary analysis.”
Overall Survival (OS):
- Median OS in the TUKYSA arm was 24.7 months (95% CI: 21.6, 28.9) compared to median OS of 19.2 months (95% CI: 16.4, 21.4) in the control arm (HR=0.73 [95% CI: 0.59-0.90]; p=0.004).
Progression Free Survival (PFS):
- The median PFS in the TUKYSA arm was 7.6 months (95% CI: 6.9, 8.3), compared to median PFS of 4.9 months (95% CI: 4.1, 5.6) in the control arm (HR=00.57 [95% CI: 0.47-0.70]; p<0.00001).
About TUKYSA (tucatinib)
TUKYSA is an oral medicine that is a tyrosine kinase inhibitor of the HER2 protein. In vitro (in lab studies), TUKYSA inhibited phosphorylation of HER2 and HER3, resulting in inhibition of downstream MAPK and AKT signaling and cell growth (proliferation), and showed anti-tumor activity in HER2-expressing tumor cells. In vivo (in living organisms), TUKYSA inhibited the growth of HER2-expressing tumors. The combination of TUKYSA and the anti-HER2 antibody trastuzumab showed increased anti-tumor activity in vitro and in vivo compared to either medicine alone.
For more information on the company’s marketed products and robust pipeline, visit www.seagen.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20210603005326/en/
Source: Seagen Inc.
Seagen's Tuksya shows sustained survival benefit in HER2-positive breast cancer patients
Jun. 03, 2021 6:29 AM ET Seagen Inc. (SGEN) By: Mamta Mayani, SA News Editor
- Seagen (NASDAQ:SGEN) announces results from from HER2CLIMB trial evaluating the addition of Tuksya (tucatinib) to trastuzumab and capecitabine in patients with HER2-positive metastatic breast cancer (MBC) with and without brain metastases.
- https://seekingalpha.com/news/3702772-seagens-tuksya-shows-sustained-survival-benefit-in-her2-positive-breast-cancer-patients
- https://seekingalpha.com/symbol/SGEN
- https://www.seagen.com/
- https://www.seagen.com/medicines/tukysa
OLUMIANT® (baricitinib)
OLUMIANT® Improved Pain, Physical Function and Morning Joint Stiffness in Rheumatoid Arthritis in Phase 3 Post-Hoc Analyses
June 1, 2021Download PDF
INDIANAPOLIS, June 1, 2021 /PRNewswire/ -- Eli Lilly and Company (NYSE: LLY) and Incyte (NASDAQ: INCY) will present data from post-hoc analyses that suggested OLUMIANT® (baricitinib) 4 mg tablet reduced pain and duration of morning joint stiffness, and improved overall physical function at 12 weeks, among patients with moderate to severe rheumatoid arthritis (RA), compared to HUMIRA® (adalimumab) and placebo. These results are being presented at the virtual Annual European Congress of Rheumatology (EULAR), June 2-5, 2021.
In a post-hoc analysis of the Phase 3 RA-BEAM study, patients treated with OLUMIANT 4 mg saw greater improvements in pain relief and physical function, as well as reduced duration of morning joint stiffness, at 12 weeks compared to HUMIRA and placebo. These differences in pain relief were not influenced by disease activity during treatment. In this analysis, improvements in fatigue with OLUMIANT 4 mg were greater than with placebo and similar to HUMIRA after 12 weeks of treatment
About The Analysis
- Baricitinib Provides Greater Improvements in Patient-Reported Outcomes Across All Disease Activity Levels Compared to Placebo and Adalimumab in Rheumatoid Arthritis
- In a post-hoc analysis, 1,305 patients from the Phase 3 RA-BEAM study were randomized into one of three treatment groups: oral, once-daily, OLUMIANT 4 mg on background methotrexate, injectable every-other-week adalimumab 40 mg on background methotrexate and placebo on background methotrexate.
- Pain was evaluated using a 0-100 mm visual analog scale, with higher scores indicating more pain; physical function was assessed using the HAQ-DI, with lower scores indicating better physical function and, thus, less disability; duration of morning joint stiffness (minutes) was reported by the patient, and fatigue was measured using the FACIT-F scale, with higher scores indicating less fatigue. Disease activity was measured using the CDAI and categorized as remission (REM, ≤2.8), low disease activity (LDA, >2.8 to ≤10), moderate disease activity (MDA, >10 to ≤22), or high disease activity (HDA, >22).
- Linear regression was used to model the relationship between change in patient reported outcomes at Week 12 (response) and CDAI values at 12 weeks (primary explanatory variable) to evaluate the extent of improvement in patient-reported outcomes with OLUMIANT 4 mg relative to placebo and adalimumab across a spectrum of disease activity levels. Last observation carried forward was used to impute missing values.
Indication and Usage for OLUMIANT (baricitinib) tablets (in the United States) for RA patients
OLUMIANT® (baricitinib) 2 mg is indicated for the treatment of adult patients with moderately to severely active rheumatoid arthritis who have had an inadequate response to one or more tumor necrosis factor (TNF) antagonist therapies. Limitation of Use: Not recommended for use in combination with other JAK inhibitors, biologic disease-modifying antirheumatic drugs (DMARDs), or with potent immunosuppressants such as azathioprine and cyclosporine.
Please click to access full Prescribing Information, including Boxed Warning about Serious infections, Malignancies, and Thrombosis, and Medication Guide.
BA HCP ISI 09JUL2020
About OLUMIANT®
OLUMIANT, a once-daily, oral JAK inhibitor was discovered by Incyte and licensed to Lilly. It is approved in the U.S. and more than 75 countries as a treatment for adults with moderate to severe rheumatoid arthritis and is approved in more than 40 countries, including the European Union and Japan, for the treatment of adult patients with moderate to severe atopic dermatitis who are candidates for systemic therapy. OLUMIANT was recently approved in Japan for the treatment of pneumonia associated with COVID-19 in hospitalized adult patients. The U.S. FDA-approved labeling for Olumiant includes a Boxed Warning for Serious Infections, Malignancy, and Thrombosis. See the full Prescribing Information here. Baricitinib is also being investigated in alopecia areata (AA), juvenile idiopathic arthritis (JIA) and systematic lupus erythematosus (SLE).1
In December 2009, Lilly and Incyte announced an exclusive worldwide license and collaboration agreement for the development and commercialization of baricitinib and certain follow-on compounds for patients with inflammatory and autoimmune diseases.
To learn more about Lilly, please visit us at lilly.com and lilly.com/newsroom.
For additional information on Incyte, please visit Incyte.com and follow @Incyte.
OLUMIANT® is a registered trademark owned or licensed by Eli Lilly and Company, its subsidiaries, or affiliates.
View original content to download multimedia:http://www.prnewswire.com/news-releases/olumiant-improved-pain-physical-function-and-morning-joint-stiffness-in-rheumatoid-arthritis-in-phase-3-post-hoc-analyses-301303457.html
SOURCE Eli Lilly & Company
Lilly/Incyte's Olumiant reduces pain and duration of morning joint stiffness in rheumatoid arthritis
Jun. 02, 2021 2:13 AM ET Eli Lilly and Company (LLY), INCY
By: Mamta Mayani, SA News Editor
- Eli Lilly (NYSE:LLY) and Incyte (NASDAQ:INCY) reports data from post-hoc analyses of Olumiant (baricitinib) 4 mg tablet in patients with moderate to severe rheumatoid arthritis (RA).
- https://seekingalpha.com/news/3702166-lillyincytes-olumiant-reduces-pain-and-duration-of-morning-joint-stiffness-in-rheumatoid-arthritis
- https://seekingalpha.com/symbol/INCY
- https://seekingalpha.com/symbol/LLY
- https://www.lilly.com/
- https://www.olumiant.com/
BREXAFEMME® (ibrexafungerp tablets)
SCYNEXIS Announces FDA Approval of BREXAFEMME® (ibrexafungerp tablets) as the First and Only Oral Non-Azole Treatment for Vaginal Yeast Infections
Download as PDF June 02, 2021
- Approval of BREXAFEMME (ibrexafungerp) represents the first approved drug in a novel antifungal class in more than 20 years
- BREXAFEMME, a one-day oral treatment for vaginal yeast infection, is the first FDA-approved indication of the ibrexafungerp development pipeline
- U.S. commercial launch expected in the second half of 2021 in partnership with Amplity Health, marking the evolution of SCYNEXIS to a commercial-stage antifungal company
- Leveraging 14 years of patent protection, SCYNEXIS continues to advance its pipeline of ibrexafungerp indications to create a long-lasting antifungal franchise in both hospital and community settings
- SCYNEXIS to host a conference call today at 8:30 a.m. ET
JERSEY CITY, N.J., June 02, 2021 (GLOBE NEWSWIRE) -- SCYNEXIS, Inc. (NASDAQ: SCYX), a biotechnology company pioneering innovative medicines to overcome and prevent difficult-to-treat and drug-resistant fungal infections, today announced that the U.S. Food and Drug Administration (FDA) has approved BREXAFEMME® (ibrexafungerp tablets), for oral use in patients with vulvovaginal candidiasis (VVC), also known as vaginal yeast infection. BREXAFEMME, which represents the first approved drug in a novel antifungal class in more than 20 years, was approved based on positive results from two Phase 3 studies in which oral ibrexafungerp demonstrated efficacy and a favorable tolerability profile in women with VVC. SCYNEXIS has partnered with Amplity Health, a leading global contract commercialization organization, to support U.S. commercialization of BREXAFEMME, with commercial launch scheduled in the second half of this year.
“The FDA approval of BREXAFEMME is the culmination of years of work and a significant milestone for SCYNEXIS, marking our evolution to a commercial-stage antifungal company. We are pleased with the approved label, highlighting the unique attributes of BREXAFEMME, and thrilled to be able to offer a new treatment option to women with vaginal yeast infections,” said Marco Taglietti, M.D., President and Chief Executive Officer of SCYNEXIS. “We believe ibrexafungerp has significant commercial potential. This first approval in the U.S. is a major step towards building the ibrexafungerp antifungal franchise as we intend to leverage an extended 10-year period of regulatory exclusivity and 14 years of patent protection. We are in a strong financial position to execute on our commercial plans and are committed to advancing our hospital programs to maximize the broad potential of this novel therapeutic class to help patients with serious and often resistant fungal infections.”
The slide and audio webcast can be accessed by visiting the Investors section of the Company's website at http://ir.scynexis.com. A replay of the webcast will be available shortly after the conclusion of the call and will be archived on the Company's website for 30 days.
About BREXAFEMME® (ibrexafungerp tablets), for oral use
BREXAFEMME is the trade name for ibrexafungerp, a novel oral antifungal approved for the treatment of vulvovaginal candidiasis (VVC), also known as vaginal yeast infection. Its mechanism of action, glucan synthase inhibition, is fungicidal against Candida species, meaning it kills fungal cells. The New Drug Application (NDA) for BREXAFEMME was approved by the U.S. Food and Drug Administration (FDA) on June 1, 2021. The NDA was supported by positive results from two Phase 3, randomized, double-blind, placebo-controlled, multi-center studies (VANISH-303 and VANISH-306), in which oral ibrexafungerp demonstrated efficacy and a favorable tolerability profile in women with VVC. BREXAFEMME represents the first approved drug in a new antifungal class in over 20 years and is the first and only treatment for vaginal yeast infections which is both oral and non-azole. For more information, visit www.brexafemme.com.
Please click here for full Prescribing Information.
Indication
BREXAFEMME® is a triterpenoid antifungal indicated for the treatment of adult and postmenarchal pediatric females with vulvovaginal candidiasis (VVC).
Dosage and Administration
The recommended dosage of BREXAFEMME is 300 mg (two tablets of 150 mg) twice a day for one day, for a total treatment dosage of 600 mg. BREXAFEMME may be taken with or without food.
For more information, visit www.brexafemme.com. We are also continuing late-stage clinical development of ibrexafungerp for the prevention of recurrent VVC as well as the treatment of life-threatening invasive fungal infections in hospitalized patients. For more information, visit www.scynexis.com.
BREXAFEMME® (ibrexafungerp tablets), for oral use
A novel oral antifungal approved for the treatment of vulvovaginal candidiasis (VVC), also known as vaginal yeast infection
Source: Scynexis
Released June 2, 2021
https://www.globenewswire.com/NewsRoom/AttachmentNg/63045483-5b8f-475e-995a-6143e068df39.
FDA approves SCYNEXIS' BREXAFEMME vaginal yeast infections treatment
Jun. 02, 2021 7:37 AM ET SCYNEXIS, Inc. (SCYX)
By: Aakash Babu, SA News Editor12 Comments
- SCYNEXIS (NASDAQ:SCYX) announces that the U.S. FDA has approved BREXAFEMME (ibrexafungerp tablets), for oral use in patients with vulvovaginal candidiasis (VVC), also known as vaginal yeast infection.
- BREXAFEMME was approved based on positive results from two Phase 3 studies in which oral ibrexafungerp demonstrated efficacy and a favorable tolerability profile in women with VVC.
- https://seekingalpha.com/news/3702229-fda-approves-scynexis-brexafemme-vaginal-yeast-infections-treatment
- https://seekingalpha.com/symbol/SCYX
- https://www.scynexis.com/
- https://www.brexafemme.com/
Taltz® (ixekizumab)
Taltz® Showed Consistent, Long-Term Improvement in Key Signs and Symptoms of Axial Spondyloarthritis Through Two Years in Phase 3 Study
June 1, 2021Download PDF-- Most patients treated with Taltz did not show bone damage progression of radiographic axial spondyloarthritis up to two years in the long-term extension of two Phase 3 studies --
INDIANAPOLIS, June 1, 2021 /PRNewswire/ -- Eli Lilly and Company (NYSE: LLY) will present new data from Phase 3 studies that further demonstrated the long-term efficacy and safety profile of Taltz® (ixekizumab) among patients with axial spondyloarthritis (axSpA). These results are being presented at the virtual Annual European Congress of Rheumatology (EULAR), June 2-5, 2021.
AxSpA is recognized as a single disease entity, with two subtypes which are defined depending on the presence (radiographic axSpA, or r-axSpA) or absence (non-radiographic axSpA, or nr-axSpA) of defined structural damage of the sacroiliac joints on plain x-ray films as per the modified New York (mNY) criteria.
"Patients living with axial spondyloarthritis deal with a range of chronic, debilitating symptoms, including inflammatory back pain, and are in need of treatment options that can provide long-term efficacy," said Lotus Mallbris, M.D., Ph.D., vice president of immunology development at Lilly. "We are excited to present a range of new data at EULAR that demonstrate treatment with Taltz provides consistent, long-term efficacy on common signs and symptoms over time in axial spondyloarthritis."
Taltz Showed Sustained Long-Term Improvements in axSpA Through Two Years
In COAST-Y, Taltz showed consistent and sustained long-term improvements in signs and symptoms, functionality and quality of life in patients with r- and nr-axSpA. In this study, more than half of patients (56.7%) treated continuously with Taltz (80 mg every four weeks, n=157) through two years achieved Assessment of SpondyloArthritis international Society 40% response (ASAS40).
Among those treated continuously with Taltz every four weeks for two years:
- 43.9% of patients achieved low disease activity status, as measured by Ankylosing Spondylitis Disease Activity Score (ASDAS) <2.1. Mean change from baseline (3.9) in ASDAS score was -1.6.
- 19.7% achieved ASAS partial remission status.
- Mean change from baseline (6.6) in Bath Ankylosing Spondylitis Functional Index (BASFI) was -2.8.
- Mean change from baseline (33.9) in Medical Outcomes Survey Short Form 36 Physical Component Summary (SF-36 PCS) was 8.4.
The safety profile of Taltz was consistent with previously published safety data, and no new safety signals were observed after up to two years of treatment.
For methodology, see the "About the Analyses" section below. Additional results from the Phase 3 COAST-Y study were also recently published in the Annals of the Rheumatic Diseases.
About the Analyses
- Long-term Treatment with Ixekizumab in Patients with Axial Spondyloarthritis: 2-year Results from COAST-Y
- COAST-Y is the two-year extension of the COAST-V, COAST-W and COAST-X trials. Upon completion of the initial trials, 773 patients continued with the dose received at the end of the originating trial at Week 52, either with 80 mg Taltz every two weeks or four weeks. Patients who had been assigned to adalimumab or placebo were re-randomized to Taltz every two weeks or every four weeks at Week 16 in COAST-V and COAST-W. Patients who had received placebo for 52 weeks in COAST-X were switched to Taltz every four weeks in COAST-Y. For this analysis, only patients continuously treated with Taltz since the originating studies were included. All other patients were analyzed separately.
- Standardized efficacy measures were used. Missing data were handled by non-responder imputation for categorical data and modified baseline observation carried forward for continuous data. Safety data were analyzed for all patients who received ≥1 dose of Taltz.
- Evaluation of Spinal Radiographic Progression in Patients with Radiographic Axial Spondyloarthritis Receiving Ixekizumab Therapy over 2 Years
- These analyses included biologic-naïve patients with active r-axSpA (COAST-V) or patients with prior inadequate response or intolerance to one or two TNF inhibitors (COAST-W) who received 80 mg Taltz every two weeks or four weeks for two years (108 weeks, of which 56 weeks were the COAST-Y long-term extension study).
- Mean change from baseline of mSASSS (average score from two selected readers, blinded for time order) for patients treated with Taltz for two years with data at both baseline and year 2 is presented (n=230; 54% of total randomized patients). Of the 657 patients who entered COAST-V or -W, 527 patients re-consented to enter COAST-Y; however, 104 patients had either baseline or Year 2 mSASSS data missing. Of 423 patients with baseline and Year 2 mSASSS data, 230 (54%) were treated with Taltz for at least two years. Of these, 110 were biologic-naïve and 120 were TNFi-experienced.
INDICATIONS AND USAGE FOR TALTZ
Taltz is approved for the treatment of patients 6 years of age and older with moderate to severe plaque psoriasis who are candidates for systemic therapy or phototherapy and for the treatment of adults with active psoriatic arthritis, active ankylosing spondylitis, or active non-radiographic axial spondyloarthritis with objective signs of inflammation.
CONTRAINDICATIONS
Taltz is contraindicated in patients with a previous serious hypersensitivity reaction, such as anaphylaxis, to ixekizumab or to any of the excipients.
Please see full Prescribing Information and Medication Guide for Taltz. See Instructions for Use included with the device.
IX HCP ISI 07MAY2020
About Taltz®
Taltz is a monoclonal antibody that selectively binds with interleukin 17A (IL-17A) cytokine and inhibits its interaction with the IL-17 receptor. IL-17A is a naturally occurring cytokine that is involved in normal inflammatory and immune responses. Taltz inhibits the release of pro-inflammatory cytokines and chemokines.
To learn more about Lilly, please visit us at lilly.com and lilly.com/news.
View original content to download multimedia:http://www.prnewswire.com/news-releases/taltz-showed-consistent-long-term-improvement-in-key-signs-and-symptoms-of-axial-spondyloarthritis-through-two-years-in-phase-3-study-301303328.html
SOURCE Eli Lilly and Company
Lilly's Taltz shows consistent, long-term efficacy in axial spondyloarthritis in late-stage study
Jun. 02, 2021 12:47 AM ETEli Lilly and Company (LLY)By: Mamta Mayani, SA News Editor
- Eli Lilly (NYSE:LLY) reports new data from Phase 3 studies that further demonstrated the long-term efficacy and safety profile of Taltz (ixekizumab) among patients with axial spondyloarthritis (axSpA).
- https://seekingalpha.com/news/3702162-lillys-taltz-shows-consistent-long-term-efficacy-in-axial-spondyloarthritis-in-late-stage-study
- https://seekingalpha.com/symbol/LLY
- https://www.lilly.com/
- https://www.taltz.com/
Kymriah® (tisagenlecleucel)
Novartis Kymriah® pivotal trial demonstrates strong response rates and a remarkable safety profile in relapsed or refractory follicular lymphoma
Jun 02, 2021
- Primary analysis of ELARA trial demonstrated a 66% complete response rate and 86% overall response rate with one-time Kymriah infusion1
- Robust response observed in heavily pretreated patients in critical need of a potentially definitive treatment option1,2
- No patients in ELARA trial experienced grade 3/4 cytokine release syndrome, the most common side effect associated with CAR-T therapy1
- Global regulatory submissions based on the ELARA trial on track for later this year
Basel, June 2, 2021 — Novartis today announced robust data from the primary analysis of the pivotal Phase II ELARA trial of Kymriah® (tisagenlecleucel) in patients with relapsed or refractory (r/r) follicular lymphoma (FL)1. Data will be presented as an oral presentation during the 2021 Annual American Society of Clinical Oncology (ASCO) Virtual Scientific Meeting (Abstract #7508; oral presentation: Monday, June 7, 10:30 AM CDT).
In the primary ELARA analysis, 97 patients were infused and evaluated for safety, 94 patients were evaluable for efficacy with a median follow-up of 11 months. Importantly, no patients experienced grade 3/4 cytokine release syndrome (CRS), the most common side effect associated with CAR-T therapy. Grade 1 or 2 CRS, as defined by the Lee Scale, occurred in 49% of patients. Grade 1 or 2 neurological events (NEs) (per CTCAE v4.03) occurred in 9% of patients and one patient experienced grade 4 NEs and recovered. Sixty-five percent of patients experienced grade ≥3 adverse events within 8 weeks post-infusion, most commonly neutropenia (28%) and anemia (13%). Three patients died from progressive disease and no deaths were treatment related. Kymriah was administered in the outpatient setting for 18% of patients in the ELARA trial1,3.
Kymriah led to responses for the majority of patients treated, with 66% achieving a complete response (CR) (95% CI, 56-75). The overall response rate was 86% (95% CI, 78-92). Response rates were consistent across high-risk patient subgroups. The median duration of response (DOR) in all responders (95% CI, NE-NE), progression free survival (PFS) (95% CI, 12.1-NE), and overall survival (OS) (95% CI, NE-NE) were not reached. Estimated DOR in patients with CR and PFS rates at six months were 94% (95% CI, 82-98) and 76% (95% CI, 65-84), respectively. Efficacy findings include data from nearly twice as many patients as were reported at the interim analysis, including high-risk and heavily pretreated patients who continued to relapse or have refractory disease despite exposure to numerous prior lines of therapy. The median number of prior therapies was 4 (range, 2-13), 78% of patients were refractory to their last treatment (76% to ≥2 prior regimens) and 60% progressed within 2 years of initial anti-CD20-containing treatment1,3.
Visit https://www.hcp.novartis.com/virtual-congress/a-2021/ for the latest information from Novartis, including our commitment to the Oncology community, and access to our ASCO21 Virtual Scientific Program data presentations (for registered participants).
About the ELARA trial
ELARA is a Phase II, single-arm, multicenter, open-label trial investigating the efficacy and safety of Kymriah in adult patients with r/r FL. This international trial has enrolled patients from over 30 sites in 12 countries worldwide. The primary endpoint is CRR based on best response by central review (Lugano 2014 criteria). Patients evaluable for efficacy had measurable disease at infusion and more than six months of follow-up from infusion or discontinued early. After infusion, disease assessments were performed every three months. Secondary endpoints include overall response rate, duration of response, progression-free survival, overall survival and safety.
In Q2 2020, the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to Kymriah in r/r FL, based on preliminary results from the ELARA trial. RMAT designation is intended to expedite the development and review of Kymriah as a regenerative therapy for this underserved patient population. Kymriah also has Orphan Drug designation from the FDA for this disease.
About Kymriah
Kymriah is the first-ever FDA-approved CAR-T cell therapy, and the first-ever CAR-T to be approved in two distinct indications. It is a one-time treatment designed to empower patients’ immune systems to fight their cancer. Kymriah is currently approved for the treatment of r/r pediatric and young adult (up to and including 25 years of age) acute lymphoblastic leukemia (ALL), and r/r adult diffuse large B-cell lymphoma (DLBCL)9.
Find out more at https://www.novartis.com.
Novartis' Kymriah demonstrates 66% complete response rate in follicular lymphoma
Jun. 02, 2021 10:39 AM ET Novartis AG (NVS) By: Jonathan M Block, SA News Editor
- In a phase 2 trial, a single infusion of Novartis' (NYSE:NVS) Kymriah (tisagenlecleucel) led to a 66% complete response ("CR") rate in patients with relapsed/refractory follicular lymphoma.
- https://seekingalpha.com/news/3702349-novartis-kymriah-demonstrates-66-complete-response-rate-in-follicular-lymphoma
- https://seekingalpha.com/symbol/NVS
- https://www.novartis.com/
- https://www.us.kymriah.com/
Cosentyx® (secukinumab)
Novartis Cosentyx receives FDA approval for treatment of children and adolescents with moderate to severe plaque psoriasis
Jun 01, 2021
- Approval for moderate to severe pediatric patients six years and older is based on pivotal trial data showing Cosentyx demonstrated superior improvements of skin symptoms compared to placebo1
- The safety profile of Cosentyx in pediatric patients with plaque psoriasis was demonstrated in two Phase III trials and is consistent with the established adult psoriasis indication1
- Plaque psoriasis is a chronic, inflammatory disease that may impact up to 350,000 children worldwide, with onset most common during adolescence2,3
Basel, June 1, 2021 — Novartis, a leader in immuno-dermatology and rheumatology, today announced the U.S. Food and Drug Administration (FDA) has approved Cosentyx® (secukinumab) for the treatment of moderate to severe plaque psoriasis in pediatric patients six years and older who are candidates for systemic therapy or phototherapy1. This is the first approval for Cosentyx in a pediatric population in the US. The Cosentyx clinical profile is supported by five years of adult data showing long-lasting efficacy and a consistent safety profile across moderate to severe plaque psoriasis, psoriatic arthritis and ankylosing spondylitis4-10.
Cosentyx® (secukinumab)
The approved pediatric dosing for Cosentyx is 75 mg or 150 mg depending on the child’s weight at the time of dosing and is administered by subcutaneous injection every four weeks after an initial loading regimen1. After initial counseling and proper training in injection technique, Cosentyx can be administered by an adult caregiver outside of a healthcare provider’s office via a single-dose prefilled syringe or Sensoready® pen.
“The impact of psoriasis on children is much deeper than skin and can potentially lead to life course impairment. Today’s FDA approval further demonstrates our commitment to reimagine medicine for pediatric plaque psoriasis patients,” said Angelika Jahreis MD, PhD, Novartis Global Head Development Unit Immunology, Hepatology & Dermatology. “With more than 400,000 patients treated in over 100 countries worldwide, we continue to build on the established safety and efficacy profile of Cosentyx, with plans to expand to 10 indications over the next 10 years.”
This Cosentyx approval is based on two Phase III studies evaluating the use of Cosentyx in children aged 6 to <18 years with plaque psoriasis. The safety profile reported in these trials was consistent with the safety profile reported in adult plaque psoriasis trials. No new safety signals were observed1.
The first study, which evaluated efficacy and safety, was a 52-week (236 weeks total), randomized, double-blind, placebo- and active-controlled study which included 162 children six years of age and older with severe plaque psoriasis. The data showed Cosentyx reduced psoriasis severity at Week 12 compared with placebo as demonstrated by the following efficacy results by baseline weight strata for the approved doses (75mg for <50kg and 150mg for ≥50kg): Psoriasis Area Severity Index (PASI) 75 response (55% 75 mg vs 10% placebo (N=22 and N=20, respectively), 86% 150 mg vs 19% placebo (N=21 and N=21, respectively), 70% total Cosentyx vs 15% total placebo (N=43 and N=41, respectively) and Investigator’s Global Assessment modified 2011 (IGA) “clear” or “almost clear” skin response (32% 75 mg vs 5% placebo, 81% 150 mg vs 5% placebo, 56% total Cosentyx vs 5% total placebo), co-primary endpoints of the study1.
The second Phase III study, which assessed safety, was a randomized open-label, 208-week trial of 84 subjects six years of age and older with moderate to severe plaque psoriasis1.
“Living with psoriasis is challenging, and can be highly stressful for children and adolescents,” said Randy Beranek, President and CEO, National Psoriasis Foundation. “Having expanded treatment options for this patient population is a step in the right direction to help reduce the burden of plaque psoriasis.”
About Cosentyx® (secukinumab)
Cosentyx is the first and only fully human biologic that directly inhibits interleukin-17A (IL-17A), a cornerstone cytokine involved in the inflammation and development of moderate-to-severe plaque psoriasis, psoriatic arthritis (PsA), ankylosing spondylitis (AS) and non-radiographic axial spondyloarthritis (nr-axSpA)14,15,16. Cosentyx is the only biologic with proven efficacy in all six key manifestations of PsA15,17,18.
Cosentyx is backed by more than 14 years of clinical experience and long-term five-year clinical data across three indications of psoriasis, PsA and AS, as well as real-world evidence8,15,19. These data strengthen the unique position of Cosentyx as a rapid and long-lasting comprehensive treatment across axial spondyloarthritis, PsA and psoriatic disease, with more than 400,000 patients treated worldwide with Cosentyx since launch20.
Find out more at https://www.novartis.com.
Novartis granted FDA approval to expand Cosentyx use in children with plaque psoriasis
Jun. 01, 2021 5:04 PM ET Novartis AG (NVS)
By: Dulan Lokuwithana, SA News Editor
- Novartis (NYSE:NVS) announced the FDA approval for Cosentyx (secukinumab) for the treatment of a certain group of pediatric patients with moderate to severe plaque psoriasis.
- https://seekingalpha.com/news/3702099-novartis-granted-fda-approval-to-expand-cosentyx-use-in-children-with-plaque-psoriasis
- https://seekingalpha.com/symbol/NVS
Bria-IMT™
BriaCell Reports 12.0 Months Overall Survival Benefit in Advanced Breast Cancer; 100% Resolution of ‘Eye-Bulging’ Tumor
June 02, 2021
- 12.0 months average overall survival benefit, including 13.4 months in patients with 2+ HLA matches and 12.5 months in patients with Grade I/II tumors(1)
- Top Responder: 21.4 months survival plus 100% resolution of ‘eye-bulging’ orbital tumor
- Compares to 7.2-9.8 months survival in historical comparison treatment trials(2)
BERKELEY, Calif. and VANCOUVER, British Columbia, June 02, 2021 (GLOBE NEWSWIRE) -- BriaCell Therapeutics Corp. (Nasdaq: BCTX, BCTXW) (TSX-V:BCT) (“BriaCell” or the “Company”), a clinical-stage biotechnology company specializing in targeted immunotherapies for advanced breast cancer, today provides an update on the overall survival (OS) data on its previously disclosed advanced breast cancer patients. These women were treated with BriaCell’s lead candidate Bria-IMT™ as monotherapy and also in combination with checkpoint inhibitors, including pembrolizumab (KEYTRUDA®; manufactured by Merck & Co., Inc.) and Incyte’s retifanlimab (manufactured and provided under a corporate collaboration with Incyte Corporation).
HLA-Typing: Cells with HLA (human leukocyte antigen) molecules on their surface determine and trigger the body’s immune response. BriaCell’s immunotherapy treatment appears most effective when the patient’s HLA-type matches with Bria-IMT™, allowing BriaCell to potentially identify patients most likely to respond. HLA-typing is a simple and widely available test.
Highlighting BriaCell’s Top Responder:
Initially announced on Sep. 19, 2019 and subsequently on Jan. 13, 2020, BriaCell has continued to emphasize this patient’s positive response to BriaCell’s treatment. Prior to BriaCell’s treatment, the patient had received 12 regimens with 16 agents (incl. 13 chemotherapies), yet her condition worsened and included a gruesome orbital tumor that metastasized behind her left eye and caused the eye to bulge from its socket (proptosis). After just six months of BriaCell’s treatment, the orbital tumor had been completely eliminated, and she survived for over 21 months, a significant clinical benefit.
For additional information on BriaCell, please visit: https://briacell.com/.
BriaCell Therapeutics skyrockets 104% on robust overall survival benefit in advanced breast cancer patients
Jun. 02, 2021 8:22 AM ETBriacell Therapeutics (BCTX), BCTXWBy: Mamta Mayani, SA News Editor
- BriaCell Therapeutics (NASDAQ:BCTX) soars 104% premarket after providing an update on the overall survival (OS) data in advanced breast cancer patients.
- https://seekingalpha.com/news/3702258-briacell-therapeutics-skyrockets-104-on-robust-overall-survival-benefit-in-advanced-breast-cancer-patients
- https://seekingalpha.com/symbol/BCTX
- https://briacell.com/
PHASE I/IIA COMBINATION STUDY OF BRIA-IMT™ WITH INCMGA00012 AND EPACADOSTAT IN ADVANCED BREAST CANCER
FDA has approved the combination study of Bria-IMT™ with Incyte Corporation’s drugs, INCMGA00012 and epacadostat. The combination study is listed in ClinicalTrials.gov as NCT03328026.
Patients With advanced breast cancer will be eligible for combination therapy with INCMGA00012 and epacadostat if they have failed 2 or more prior lines of therapy.
biotecMAX™ April/May 2021 Insight
VAXELIS
Merck and Sanofi’s First and Only Six-in-One Pediatric Combination Vaccine Now Available in the United States
June 1, 2021 6:45 am ET
This Combination Vaccine May Simplify Execution and Reduce Shots Needed to Complete CDC’s Recommended Child and Adolescent Immunization Schedule
KENILWORTH, N.J. & BRIDGEWATER, N.J.--(BUSINESS WIRE)-- VAXELIS™ (Diphtheria and Tetanus Toxoids and Acellular Pertussis, Inactivated Poliovirus, Haemophilus b Conjugate and Hepatitis B Vaccine), developed as part of a U.S.-based partnership between Merck (NYSE: MRK), known as MSD outside the United States and Canada, and Sanofi Pasteur, the global vaccines business unit of Sanofi, is now available in the U.S. VAXELIS is the first and only hexavalent (six-in-one) combination vaccine available in the U.S. indicated for active immunization to help prevent diphtheria, tetanus, pertussis, poliomyelitis, hepatitis B, and invasive disease due to Haemophilus influenzae type b. VAXELIS is approved for use as a 3-dose series in children 6 weeks through 4 years of age (prior to the 5th birthday).
“Given the potential to reduce the number of shots by as many as three in the first six months of life, as compared to pentavalent vaccines plus hepatitis B or Haemophilus influenzae type b vaccines, VAXELIS represents an important option for healthcare professionals and parents,” said Joanne Monahan, senior vice president, U.S. Vaccines, Merck. “Merck and Sanofi have been in contact with the major insurers and the appropriate pricing publications to notify them of the availability of VAXELIS, and we look forward to working with payers and providers to deliver this novel hexavalent vaccine to age-appropriate children. This news should also help to remind providers and parents of the importance of continuing routine vaccinations and recommended well visits.”
On February 11, 2021, the U.S. Centers for Disease Control and Prevention’s Advisory Committee included VAXELIS as a combination vaccine option in the CDC’s Recommended Child and Adolescent Immunization Schedule. VAXELIS will be widely available in the U.S. through traditional public and private channels, including directly from Sanofi Pasteur via vaccineshoppe.com.
About Combination Vaccines
Combination vaccines take two or more vaccines that could be given individually and put them into a single shot. Combination vaccines have been a significant catalyst for change for the healthcare industry since their introduction more than 50 years ago. Public health authorities and leading professional associations generally recommend the use of combination vaccines over separate injections, when appropriate, including the American Academy of Pediatrics, The American Academy of Family Physicians, U.S. Centers for Disease Control and Prevention, The World Health Organization and others.
About VAXELIS
VAXELIS was developed as part of a U.S.-based partnership established in 1991 between Merck and Sanofi Pasteur and draws upon both companies' experience in the development, manufacturing and marketing of individual and combination vaccines. VAXELIS includes antigens for diphtheria, tetanus, pertussis (whooping cough), and poliomyelitis from Sanofi Pasteur and antigens for Haemophilus influenzae type b and hepatitis B from Merck.
VAXELIS is a vaccine indicated for active immunization to prevent diphtheria, tetanus, pertussis, poliomyelitis, hepatitis B, and invasive disease due to Haemophilus influenzae type b. VAXELIS is approved for use as a 3-dose series in children 6 weeks through 4 years of age (prior to the 5th birthday).
Please see Prescribing Information for VAXELIS™ (Diphtheria and Tetanus Toxoids and Acellular Pertussis, Inactivated Poliovirus, Haemophilus b Conjugate and Hepatitis B Vaccine) at https://www.merck.com/product/usa/pi_circulars/v/vaxelis/vaxelis_pi.pdf and Patient Information for VAXELIS™ at https://www.merck.com/product/usa/pi_circulars/v/vaxelis/vaxelis_ppi.pdf.
For more information, visit www.merck.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20210601005089/en/
Source: Merck & Co., Inc.
Merck, Sanofi’s six-in-one pediatric combination vaccine now available in U.S.
Jun. 01, 2021 8:51 AM ET Merck & Co., Inc. (MRK), SNY
By: Mamta Mayani, SA News Editor9 Comments
- Vaxelis developed as part of a U.S.-based partnership between Merck (NYSE:MRK) and Sanofi Pasteur, the global vaccines business unit of Sanofi (NASDAQ:SNY), is now available in the U.S.
- https://seekingalpha.com/news/3701643-merck-sanofis-six-in-one-pediatric-chttps://www.merck.com/ombination-vaccine-now-available-in-us
- https://seekingalpha.com/symbol/SNY
- https://seekingalpha.com/symbol/MRK
- https://www.merck.com/
Encaleret
BridgeBio Pharma Receives FDA Fast Track Designation For Encaleret For The Treatment Of Autosomal Dominant Hypocalcemia Type 1
PALO ALTO, CA – June 1, 2021 — BridgeBio Pharma, Inc. (Nasdaq: BBIO), a commercial-stage biopharmaceutical company founded to discover, create, test and deliver meaningful medicines for patients with genetic diseases and cancers with clear genetic drivers, today announced that the U.S. Food and Drug Administration (FDA) granted Fast Track designation for encaleret for the treatment of autosomal dominant hypocalcemia (ADH1). The FDA Fast Track designation program is designed to facilitate the development and to expedite the review of new therapies hoping to treat or prevent serious conditions and fill an unmet medical need. BridgeBio announced the Fast Track designation on World Hypoparathyroidism Awareness Day, an annual global awareness and education event designed to spotlight and support people living with ADH1 and other types of hypoparathyroidism.
“Balancing near-normal blood and avoiding excess urinary calcium is a daily struggle for patients with ADH1 as the range of symptoms produced by the highs and lows of the condition cannot adequately be addressed by current standard-of-care,” said Jonathan Fox, M.D., Ph.D., Chief Medical Officer of the cardio-renal affiliates at BridgeBio, including Calcilytix, which is focused on developing encaleret. “With respect to the mechanism of disease, which is driven by gain-of-function variants in CASR, encaleret, as an allosteric negative modulator of the receptor’s calcium sensing activity, has the potential to correct the disease mechanism at its source. It is encouraging to receive Fast Track designation from the FDA as it recognizes the seriousness of ADH1 and the potential for encaleret to address this profound unmet medical need.”
Promising early results from its ongoing Phase 2b proof-of-concept, open-label study of encaleret, orally administered, for patients with ADH1 were presented at the Endocrine Society’s 2021 Annual Meeting, which showed the normalization of blood calcium and urine calcium in six of six (100%) ADH1 participants evaluated over five days and demonstrated clinical proof-of-concept.2 BridgeBio plans to engage with regulatory health authorities to discuss the design of a Phase 3 registrational study in patients with ADH1. If the development program is successful, encaleret could be the first approved therapy indicated specifically for the treatment of ADH1.
For more information on the Phase 2b clinical trial currently recruiting participants with ADH1, please visit clinicaltrials.gov (Identifier: NCT04581629).
For more information visit bridgebio.com.
https://bridgebio.com/pipeline
BridgeBio Pharma's encaleret Fast Track's in U.S. for autosomal dominant hypocalcemia
Jun. 01, 2021 7:40 AM ETBridgeBio Pharma, Inc. (BBIO)
By: Mamta Mayani, SA News Editor
- The FDA has granted Fast Track designation for BridgeBio Pharma's (NASDAQ:BBIO) encaleret for the treatment of autosomal dominant hypocalcemia (ADH1).
- https://seekingalpha.com/news/3701588-bridgebio-pharmas-encaleret-fast-tracks-in-us-for-autosomal-dominant-hypocalcemia
- https://seekingalpha.com/symbol/BBIO
LUMAKRAS™ (Sotorasib)
FDA Approves LUMAKRAS™ (Sotorasib), The First And Only Targeted Treatment For Patients With KRAS G12C-Mutated Locally Advanced Or Metastatic Non-Small Cell Lung Cancer
KRAS G12C, a Driver Mutation Found in About 13% of Patients With Non-Squamous Non-Small Cell Lung Cancer, is Now Actionable(1)Once-Daily Dosing of LUMAKRAS Demonstrated Durable Responses and a Positive Benefit-Risk ProfileBiomarker Testing Critical to Identify KRAS G12C Mutation
THOUSAND OAKS, Calif., May 28, 2021 /PRNewswire/ -- Amgen (NASDAQ: AMGN) today announced that the U.S. Food and Drug Administration (FDA) has approved LUMAKRAS™ (sotorasib) for the treatment of adult patients with KRAS G12C-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC), as determined by an FDA-approved test, who have received at least one prior systemic therapy. LUMAKRAS has received accelerated approval based on overall response rate (ORR) and duration of response (DoR). Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
To view the multimedia assets associated with this release, please click: https://www.multivu.com/players/English/8812853-amgen-fda-approval-lumakras-sotorasib-targeted-kras-g12c-lung-cancer/
"The FDA approval of LUMAKRAS is a breakthrough moment for patients with KRAS G12C-mutated non-small cell lung cancer because there is now a targeted therapy for this common, but previously elusive, mutation," said David M. Reese, M.D., executive vice president of Research and Development at Amgen. "KRAS has challenged cancer researchers for more than 40 years with many deeming it as 'undruggable.' The LUMAKRAS development program was a race against cancer for Amgen's scientists and clinical trial investigators who together have now successfully delivered this new medicine to patients in less than three years—from first patient dosed to U.S. regulatory approval."
The FDA approval of LUMAKRAS is based on results from a subset of patients in CodeBreaK 100, the largest clinical trial conducted to date exclusively for patients with the KRAS G12C mutation. The trial demonstrated favorable efficacy and tolerability in 124 patients with KRAS G12C mutation-positive NSCLC who had disease progression after receiving an immunotherapy and/or chemotherapy. In the trial, 960 mg of LUMAKRAS administered orally once-daily demonstrated an ORR (a proportion of patients with ≥ 30% decrease in tumor) of 36% (95% CI: 28-45) with 81% (95% CI: 73-87) of patients achieving disease control (percentage of patients who have achieved complete response, partial response and stable disease for more than three months). The median DoR was 10 months. The most common adverse reactions (≥ 20%) were diarrhea, musculoskeletal pain, nausea, fatigue, hepatotoxicity and cough. Adverse reactions resulting in permanent discontinuation of LUMAKRAS occurred in 9% of patients.
"Sotorasib represents a major advancement in oncology and changes the treatment paradigm for patients with KRAS G12C-mutated non-small cell lung cancer," said Bob T. Li, M.D., Ph.D., MPH, principal investigator at Memorial Sloan Kettering Cancer Center. "Patients with non-small cell lung cancer who have progressed beyond first-line treatment face a poor prognosis and have limited treatment options available to them. Sotorasib delivers a new option for these patients, and it is the first KRAS-targeted therapy to be approved after nearly four decades of research."
About LUMAKRAS™ (sotorasib)
Amgen has taken on one of the toughest challenges of the last 40 years in cancer research by developing LUMAKRAS, a KRASG12C inhibitor.6 LUMAKRAS was the first KRASG12C inhibitor to enter the clinic and is being studied in the largest clinical program exploring 11 combinations with global investigator sites spanning five continents.
LUMAKRAS has demonstrated a positive benefit-risk profile with rapid, deep and durable anticancer activity in patients with locally advanced and metastatic non-small cell lung cancer (NSCLC) harboring the KRAS G12C mutation with a once daily oral formulation. As part of the evaluation for this accelerated approval, FDA is requiring a post-marketing trial to investigate whether a lower dose will have a similar clinical effect.
LUMAKRAS is also being studied in multiple other solid tumors.6
In the U.S., LUMAKRAS was reviewed by the FDA under its Real-Time Oncology Review (RTOR), a pilot program that aims to explore a more efficient review process that ensures safe and effective treatments are made available to patients as early as possible. Amgen submitted a Marketing Authorization Application (MAA) in the EU in December 2020 and New Drug Applications in Japan (J-NDA) and Switzerland in April 2021. Additionally, Amgen submitted MAAs for sotorasib in Australia, Brazil, Canada and the United Kingdom in January 2021 to participate in the FDA's Project Orbis initiative. Sotorasib was granted Breakthrough Therapy Designation in the U.S. and China.
LUMAKRAS™ (sotorasib) U.S. Indication
LUMAKRAS™ is indicated for the treatment of adult patients with KRAS G12C-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC), as determined by an FDA-approved test, who have received at least one prior systemic therapy.
This indication is approved under accelerated approval based on overall response rate (ORR) and duration of response (DOR). Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
Please see LUMAKRAS™ full Prescribing Information.
About CodeBreaK
The CodeBreaK clinical development program for Amgen's drug sotorasib is designed to treat patients with an advanced solid tumor with the KRAS G12C mutation and address the longstanding unmet medical need for these cancers. As the most advanced KRAS G12C clinical development program, CodeBreaK has enrolled more than 800 patients across 13 tumor types since its inception.
CodeBreaK 100, the Phase 1 and 2, first-in-human, open-label multicenter study, enrolled patients with KRAS G12C-mutant solid tumors. Eligible patients must have received a prior line of systemic anticancer therapy, consistent with their tumor type and stage of disease. The primary endpoint for the Phase 2 study was centrally assessed objective response rate. The Phase 2 trial in NSCLC enrolled 126 patients, 124 of whom had centrally evaluable lesions by RECIST at baseline. The Phase 2 trial in colorectal cancer (CRC) is fully enrolled and topline results are expected later in 2021.
A global Phase 3 randomized active-controlled study comparing sotorasib to docetaxel in patients with KRAS G12C-mutated NSCLC (CodeBreaK 200) has completed enrollment. Amgen also has several Phase 1b studies investigating sotorasib monotherapy and sotorasib combination therapy across various advanced solid tumors (CodeBreaK 101) open for enrollment.
For information, please visit www.hcp.codebreaktrials.com.
For more information, visit www.amgen.com
SOURCE Amgen
Amgen, Mirati's Lumakras can target 13% of the NSCLC market
May 28, 2021 3:00 PM ET Amgen Inc. (AMGN), MRTX Amgen Inc. (AMGN) Mirati Therapeutics, Inc. (MRTX)Eli Lilly and Company (LLY)
By: Jonathan M Block, SA News Editor1 Comment
- Amgen (NASDAQ:AMGN) and Mirati Therapeutics' (NASDAQ:MRTX) just approved KRAS inhibitor Lumakras (sotorasib) for non-small cell lung cancer ("NSCLC") could be used with as many as 13% of those patients.
- https://seekingalpha.com/news/3701338-amgen-mirati-lumakras-can-target-13-of-the-nsclc-market
- https://seekingalpha.com/symbol/MRTX
- https://seekingalpha.com/symbol/AMGN
- https://www.amgen.com/
ERLEADA® (apalutamide)
Long-Term ERLEADA® (apalutamide) Patient-Reported Outcomes Data in Metastatic Castration-Sensitive Prostate Cancer Demonstrate Maintenance of Health-Related Quality of Life for Patients
May 26, 2021United States
Results at ASCO from Phase 3 TITAN study final analysis confirm survival benefit achieved with the addition of ERLEADA® to androgen deprivation therapy without significant side effect burden
RARITAN, N.J., May 26, 2021 -- The Janssen Pharmaceutical Companies of Johnson & Johnson today announced patient-reported outcomes (PRO) data from the pre-specified final analysis of the Phase 3 TITAN study in patients with metastatic castration-sensitive prostate cancer (mCSPC). The TITAN study previously demonstrated statistically significant improvement in overall survival (OS) after a median follow-up of 44 months in patients receiving ERLEADA® plus androgen deprivation therapy (ADT).1 The new PRO data showed that the addition of ERLEADA® to ADT maintained patients' health-related quality of life (HRQoL) and did not worsen side effect burden, consistent with ADT alone.1 These data are being presented at the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting, June 4-8 (Abstract #5068).
No significant differences in quality of life were observed between patients who received ERLEADA® plus ADT and patients who received placebo plus ADT.1 Overall, patients in both groups reported being relatively asymptomatic with a good baseline HRQoL; the outcomes were assessed using the Brief Pain Inventory-Short Form (BPI-SF) and Functional Assessment of Cancer Therapy-Prostate (FACT-P) questionnaires.1 On the BPI pain severity scale of zero (no pain/interference in daily activities) to 10 (worst pain/interference), median patient scores were 1.1 in the ERLEADA® group and 1 in the placebo plus ADT group. On the FACT-P HRQoL scale (1-156, higher score = better quality of life), median patient scores were 113 in the ERLEADA® group and 113.3 in the placebo plus ADT group.1 ERLEADA® plus ADT was also shown to maintain physical, social and family, emotional, functional, and mental well-being beyond two years, as assessed by FACT-P.1 There were no significant differences between groups in median time to deterioration in any BPI or FACT-P scores, further demonstrating maintenance of quality of life with ERLEADA®.1
ERLEADA® has previously shown a statistically significant improvement in OS for both of its approved indications in prostate cancer, specifically mCSPC (TITAN study) and non-metastatic castration-resistant prostate cancer (SPARTAN study).2 The TITAN final analysis data, presented at the 2021 ASCO Genitourinary Cancers Symposium and recently published in the Journal of Clinical Oncology, reaffirmed that the addition of ERLEADA® to ADT continued to provide statistically significant OS benefit after 44 months in patients with mCSPC.3 ERLEADA® plus ADT reduced the risk of death by 35 percent compared with ADT alone (hazard ratio [HR]=0.65; 95 percent confidence interval [CI], 0.53-0.79; P<0.0001).3 SPARTAN final analysis data were presented at the 2020 ASCO Annual Meeting, showing that ERLEADA® plus ADT prolonged median OS by 14 months (73.9 vs. 59.9 months median OS for ERLEADA® and placebo groups, respectively) and reduced the risk of death by 22 percent (HR=0.78; 95 percent CI, 0.64-0.96; P=0.016).4
"Patient-reported outcomes provide meaningful input into treatment decisions by giving us insights into the way patients feel and function," said Mary Guckert, RN, MSN, Vice President, Development Leader, Prostate Cancer, Janssen Research & Development, LLC. "It is critically important to offer treatments, such as ERLEADA, that have demonstrated significant survival advantage and that maintain quality of life in patients with metastatic castration-sensitive prostate cancer."
About the TITAN Study3
TITAN (NCT02489318) is a Phase 3, randomized, placebo-controlled, double-blind study in patients with mCSPC. The study included 1,052 patients in 23 countries across 260 sites in North America, Latin America, South America, Europe, and Asia Pacific.6 Patients with mCSPC were randomized 1:1 and received either ERLEADA® (240 mg) plus ADT (n=524), or placebo plus ADT (n=527). The recruitment period for the study spanned from December 2015 to July 2017.2 The study included patients with mCSPC with both low- and high-volume disease, those who were newly diagnosed, and those who had received prior definitive local therapy or prior treatment with up to six cycles of docetaxel for mCSPC.3
An Independent Data-Monitoring Committee was commissioned by the sponsor to monitor safety and efficacy.6 Dual primary endpoints of the study were OS and radiographic progression free survival (rPFS). Secondary endpoints included time to cytotoxic chemotherapy, time to pain progression, time to chronic opioid use, and time to skeletal-related events.6 Exploratory endpoints included time to prostate specific antigen (PSA) progression, second progression free survival (PFS2) and time to symptomatic progression.6 For additional study information, visit ClinicalTrials.gov.
About ERLEADA® (apalutamide)
ERLEADA® is an androgen receptor inhibitor indicated for the treatment of patients with non-metastatic castration-resistant prostate cancer (nmCRPC) and for the treatment of patients with metastatic castration-sensitive prostate cancer (mCSPC).2 ERLEADA® received U.S. Food and Drug Administration (FDA) approval for nmCRPC on February 14, 2018 and was approved for mCSPC on September 17, 2019.2 To date, more than 25,000 patients worldwide have been treated with ERLEADA®. ERLEADA® is taken orally, once daily, with or without food.2 The NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Prostate Cancer include apalutamide (ERLEADA®) with continued androgen deprivation therapy**† as a Category 1 Preferred treatment option for patients with non-metastatic (M0) castration-resistant prostate cancer and a PSADT ≤10 months.7 The NCCN Clinical Practice Guidelines® also include apalutamide (ERLEADA®) with androgen deprivation**† as a Category 1 Preferred treatment option for patients with metastatic (M1) castration-naive prostate cancer.‡6 The American Urological Association (AUA) Guidelines for Castration-Resistant Prostate Cancer (CRPC) recommend clinicians offer apalutamide (ERLEADA®) with continued androgen deprivation therapy (ADT) as one of the treatment options for patients with nmCRPC at high risk for developing metastatic disease (Standard; Evidence Level Grade A)***.8 ERLEADA® is being further studied in two ongoing Phase 3 clinical trials.
For more information about ERLEADA®, visit www.ERLEADA.com.
Please see the full Prescribing Information for ERLEADA®.
Learn more at www.janssen.com.
https://seekingalpha.com/symbol/JNJ
TRUSELTIQ™ (infigratinib)
BridgeBio Pharma’s Affiliate QED Therapeutics And Partner Helsinn Group Announce FDA Approval Of TRUSELTIQ™ (Infigratinib) For Patients With Cholangiocarcinoma
Pivotal study demonstrated a clinically meaningful rate of tumor shrinkage (overall response rate) and duration of response in patients with previously-treated advanced cholangiocarcinoma (CCA) harboring an FGFR2 fusion or rearrangement
BridgeBio, through its affiliate QED (“BridgeBio”), and Helsinn will co-commercialize TRUSELTIQ in the U.S.
TRUSELTIQ is BridgeBio’s first FDA approved therapeutic in oncology and second approved therapeutic this year
PALO ALTO, CA and LUGANO, Switzerland, May 28, 2021– BridgeBio Pharma, Inc. (Nasdaq: BBIO), through its affiliate QED Therapeutics, Inc., and Helsinn Group today announced that the US Food and Drug Administration (FDA) has approved TRUSELTIQ™ (infigratinib) under the accelerated approval program for the treatment of patients with previously-treated locally advanced or metastatic cholangiocarcinoma (CCA) harboring an FGFR2 fusion or rearrangement. TRUSELTIQ is an orally administered, ATP-competitive, tyrosine kinase inhibitor of FGFR. In the pivotal trial of patients with advanced, unresectable CCA, an aggressive malignancy with poor prognosis, TRUSELTIQ led to cases of tumor shrinkage. CCA is known to affect approximately 20,000 people in the United States and European Union each year and has a median five-year survival rate of only 9%.1
Visit TRUSELTIQ.com for more information, including full Prescribing Information.
About TRUSELTIQ™ (infigratinib)
TRUSELTIQ (infigratinib) is an orally administered, ATP-competitive, tyrosine kinase inhibitor of FGFR, approved for the treatment of individuals with FGFR2 fusion-driven cholangiocarcinoma (bile duct cancer). TRUSELTIQ targets the fibroblast growth factor receptor (FGFR) protein, blocking downstream activity. In clinical studies, TRUSELTIQ demonstrated a clinically meaningful rate of tumor shrinkage (overall response rate) and duration of response. Visit TRUSELTIQ.com for more information. Infigratinib is not FDA approved for any other indication in the U.S. and is not approved for use by any other health authority. It is currently being evaluated in clinical studies for first-line cholangiocarcinoma and urothelial carcinoma (bladder cancer). For more information, visit QEDTx.com.
Clinical Studies1
The efficacy of TRUSELTIQ was based on a single-arm Phase 2 study which included 108 patients with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with an FGFR2 fusion or rearrangement. Ninety-nine percent of patients had metastatic (Stage IV) disease at the time of study entry.
These 108 adult patients with advanced/metastatic CCA received infigratinib 125 mg orally for 21 days of each 28-day cycle until unacceptable toxicity or disease progression. All patients received prophylaxis with the oral phosphate binder sevelamer. TRUSELTIQ achieved a 23% objective response rate (ORR) and a median duration of response (DOR) of 5.0 months.
U.S. Indication for TRUSELTIQ
TRUSELTIQ is indicated for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test.
Accelerated approval was granted based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s).
For more information, please visit
For more information, please visit QEDTx.com.
For more information visit bridgebio.com.
TRUSELTIQ is a trademark of QED Therapeutics. QED Therapeutics is a member of the BridgeBio family. ForgingBridges is a trademark of BridgeBio.
BridgeBio's affiliate QED Therapeutics says TRUSELTIQ cancer treatment approved by FDA
May 28, 2021 3:12 PM ET
BridgeBio Pharma, Inc. (BBIO)BridgeBio Pharma, Inc. (BBIO)
By: Aakash Babu, SA News Editor
- BridgeBio Pharma (BBIO +2.1%) through its affiliate QED Therapeutics and Helsinn Group announce that the U.S. FDA has approved TRUSELTIQ (infigratinib) under the accelerated approval program for the treatment of certain patients with cholangiocarcinoma, a cancer of the bile ducts of the liver.
https://seekingalpha.com/symbol/BBIO
Teplizumab (PRV-031)
FDA Advisory Committee Votes in Favor of the Benefits of Teplizumab Outweighing the Risks in Support of Approval to Delay Clinical Type 1 Diabetes (T1D)If approved, teplizumab will be the first disease-modifying therapy in T1DMay 27, 2021
RED BANK, N.J., May 27, 2021 /PRNewswire/ -- Provention Bio, Inc. (Nasdaq: PRVB), a biopharmaceutical company dedicated to intercepting and preventing immune-mediated disease, today announced that the Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) of the U.S. Food and Drug Administration (FDA) voted 10 yes and 7 no on the question, "Does the information provided in the background documents and presentations by the Applicant and FDA show that the benefits of teplizumab outweigh the risks in support of approval to delay clinical type 1 diabetes mellitus?".
The EMDAC based its recommendation on safety and efficacy data from the pivotal TN-10 Study in which a single 14-day course of teplizumab delayed insulin-dependent, clinical-stage disease by a median of at least two years in presymptomatic patients with Stage 2 type 1 diabetes (T1D) compared to placebo. Acknowledging the significant unmet medical need facing early-stage T1D patients, the Committee Members discussed the strengths and limitations of the clinical data and provided opinions on the proposed indication statement and potential post-marketing studies.
About Teplizumab (PRV-031):
Teplizumab is an investigational anti-CD3 monoclonal antibody (mAb) with a filed BLA under Priority Review by the FDA for the delay of clinical T1D in at-risk individuals. In the pivotal TN-10 Study, a single 14-day course of teplizumab delayed insulin-dependent, clinical-stage disease by a median of at least two years in presymptomatic patients with Stage 2 T1D compared to placebo. The observed adverse events are mechanism-based, transient, and predictable, including lymphopenia, transaminase elevations, rash, and cytokine release events. These results were published in the New England Journal of Medicine and simultaneously presented at the American Diabetes Association meeting in 2019. More than 800 patients have received teplizumab in multiple clinical studies involving more than 1,000 subjects. In previous studies of newly diagnosed patients, teplizumab consistently demonstrated the ability to preserve beta-cell function as shown by C-peptide, a measure of endogenous insulin production. It correspondingly reduced the need for exogenous insulin use. Teplizumab has been granted Breakthrough Therapy Designation by the FDA and PRIME designation by the European Medicines Administration. Provention is currently also evaluating teplizumab in patients with newly diagnosed insulin-dependent T1D (the Phase 3 PROTECT study).
Provention Bio, Inc. uses its website, www.proventionbio.com
FDA Advisory Committee supports Provention Bio's teplizumab in delaying diabetes
May 28, 2021 6:36 AM ETProvention Bio, Inc. (PRVB)Provention Bio, Inc. (PRVB)By: Mamta Mayani, SA News Editor1 Comment
- Provention Bio (NASDAQ:PRVB) announces that the FDA's advisory committee voted 10-7, confirming that the benefits of PRVB's drug candidate teplizumab in delaying clinical type 1 diabetes (T1D) outweigh the risks.
- https://seekingalpha.com/news/3701109-fda-advisory-committee-supports-provention-bios-teplizumab-in-delaying-diabetes
- https://seekingalpha.com/symbol/PRVB
- https://proventionbio.com/intercepting-autoimmune-disease
Zeposia® (ozanimod)
U.S. Food and Drug Administration Approves Bristol Myers Squibb’s Zeposia® (ozanimod), an Oral Treatment for Adults with Moderately to Severely Active Ulcerative Colitis
05/27/2021CATEGORY:
Zeposia, the first and only oral sphingosine 1-phosphate (S1P) receptor modulator approved for ulcerative colitis1, represents a new way of treating this chronic immune-mediated disease2
Zeposia demonstrated significant improvements across all primary and key secondary efficacy endpoints – including clinical remission, clinical response, endoscopic improvement and endoscopic-histologic mucosal improvement – versus placebo at Week 10 and Week 52 in True North Phase 3 study1
First approved gastrointestinal disease treatment for Bristol Myers Squibb’s growing immunology franchise and marks the second indication for Zeposia1
Bristol Myers Squibb is committed to making Zeposia accessible to appropriate patients who need it through the Zeposia 360 Support™ program
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE:BMY) today announced that the U.S. Food and Drug Administration (FDA) approved Zeposia® (ozanimod) 0.92 mg for the treatment of adults with moderately to severely active ulcerative colitis (UC), a chronic inflammatory bowel disease (IBD).1 Zeposia, an oral medication taken once daily, is the first and only sphingosine 1-phosphate (S1P) receptor modulator approved for patients with moderately to severely active UC. The mechanism by which Zeposia exerts therapeutic effects in UC is unknown but may involve the reduction of lymphocyte migration into the intestines. It is thought that by targeting S1P receptors on lymphocytes, a type of immune system cell, Zeposia reduces the number of lymphocytes in peripheral blood.1,3,4
Zeposia® (ozanimod)
The approval is based on data from True North, a pivotal Phase 3 trial evaluating Zeposia as an induction and maintenance therapy versus placebo in adult patients with moderately to severely active UC. During induction at Week 10 (Zeposia N=429 versus placebo N=216) the trial met its primary endpoint of clinical remissiona (18% versus 6%, p<0.0001) as well as key secondary endpoints, including clinical responseb (48% versus 26%, p<0.0001), endoscopic improvementc (27% versus 12%, p<0.0001) and endoscopic-histologic mucosal improvementd (13% versus 4%, p<0.001) for Zeposia versus placebo, respectively. During maintenance at Week 52 (Zeposia N=230 versus placebo N=227) the trial met its primary endpoint of clinical remissiona (37% versus 19%, p<0.0001) as well as key secondary endpoints, including clinical response (60% versus 41%, p<0.0001), endoscopic improvement (46% versus 26%, p<0.001), corticosteroid-free clinical remissione (32% versus 17%, p<0.001) and endoscopic-histologic mucosal improvement (30% versus 14%, p<0.001) for Zeposia versus placebo, respectively. Decreases in rectal bleeding and stool frequency subscores were observed as early as Week 2 (i.e.,1 week after completing the required 7-day dosage titration) in patients treated with Zeposia.
Zeposia is contraindicated in patients who in the last six months experienced myocardial infarction, unstable angina, stroke, transient ischemic attack (TIA), decompensated heart failure requiring hospitalization, or Class III/IV heart failure or have the presence of Mobitz type II second-degree or third degree atrioventricular (AV) block, sick sinus syndrome, or sino-atrial block, unless the patient has a functioning pacemaker; patients with severe untreated sleep apnea; and patients taking a monoamine oxidase (MAO) inhibitor. Zeposia is associated with the following Warnings and Precautions: increased risk of infections, bradyarrhythmia and atrioventricular conduction delays, liver injury, fetal risk, increased blood pressure, respiratory effects, macular edema, posterior reversible encephalopathy syndrome (PRES), unintended additive immunosuppressive effects from prior immunosuppressive or immune-modulating drugs, and severe increase in disability and immune system effects after stopping Zeposia. Please see Important Safety Information for additional details. The most common adverse reactions (incidence ≥ 4%) were liver test increased, upper respiratory infection, and headache.1
“In True North, Zeposia demonstrated efficacy for endpoints such as clinical remission, endoscopic and histological mucosal improvement and safety. All are very relevant considerations for patients with ulcerative colitis,” said Michael Chiorean, M.D., AGAF, FASGE, co-director of IBD Center, Swedish Medical Center, Seattle, Washington.6 “Zeposia has the potential to be an important new treatment option for adult patients with moderate to severe ulcerative colitis.”1
“Ulcerative colitis can be debilitating and unpredictable for the people living with this chronic inflammatory bowel disease,” said Michael Osso, President & CEO of the Crohn’s & Colitis Foundation.2,7,8 “The approval of this new oral treatment is welcome news for our community and provides hope to many patients who are looking for new options to achieve symptom relief and remission.”1
About True North
True North is a Phase 3, multicenter, randomized, double-blind, placebo-controlled clinical trial assessing the efficacy and safety of Zeposia® (ozanimod) 0.92 mg in patients with moderately to severely active ulcerative colitis who had an inadequate response or were intolerant to any of the following: oral aminosalicylates, corticosteroids, immunomodulators or a biologic. Patients were to be receiving treatment with oral aminosalicylates and/or corticosteroids prior to and during the induction period.1 A total of 30% of patients had previously failed or were intolerant to TNF blockers. Of these patients, 63% received at least two biologics including TNF blockers. At study entry, mean age was 42 years, 60% were male and mean disease duration was 7 years; patient characteristics were well-balanced across treatment groups.9 In the 10 week induction study (UC Study 1), a total of 645 patients were randomized 2:1 to receive Zeposia (N=429) or placebo (N=216), of whom 94% and 89%, respectively, completed the induction study.9
In maintenance (UC Study 2), a total of 457 patients who received Zeposia in either UC Study 1 or in an open-label arm and achieved clinical response at Week 10 were re-randomized 1:1 and were treated with either Zeposia 0.92 mg (N=230) or placebo (N=227) for 42 weeks, for a total of 52 weeks of treatment.1 Concomitant aminosalicylates were required to remain stable through Week 52. Patients on concomitant corticosteroids were to taper their dose upon entering the maintenance study.1 Of these, 80% and 54.6% of patients who received Zeposia and placebo, respectively, completed the study.10
All eligible patients were rolled into an open-label extension trial, which is ongoing and designed to assess the longer-term profile of Zeposia for the treatment of moderately to severely active ulcerative colitis.10 More information can be found on www.clinicaltrials.gov, NCT02435992.
About Zeposia® (ozanimod)
Zeposia® (ozanimod) is an oral, sphingosine 1-phosphate (S1P) receptor modulator that binds with high affinity to S1P receptors 1 and 5.1,4Zeposia reduces the capacity of lymphocytes to migrate from lymphoid tissue, reducing the number of circulating lymphocytes in peripheral blood.4 The mechanism by which Zeposia exerts therapeutic effects in ulcerative colitis is unknown but may involve the reduction of lymphocyte migration into the intestines.
Bristol Myers Squibb is continuing to evaluate Zeposia in an open-label extension trial, which is ongoing and designed to assess the longer-term profile of Zeposia for the treatment of moderately to severely active ulcerative colitis. The company is also investigating Zeposia for the treatment of moderately to severely active Crohn’s disease in the ongoing Phase 3 YELLOWSTONE clinical trial program.
Zeposia was approved by the U.S. Food and Drug Administration (FDA) for the treatment of adults with relapsing forms of multiple sclerosis (RMS) in March 2020. 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. The European Medicines Agency (EMA) validated Bristol Myers Squibb’s Marketing Authorization Application for Zeposia for the treatment of adults with moderately to severely active ulcerative colitis in December 2020. A regulatory decision from the EMA is expected in the second half of 2021.
INDICATIONS
ZEPOSIA® (ozanimod) is indicated for the treatment of:
1. Relapsing forms of multiple sclerosis (MS), to include clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease, in adults.
2. Moderately to severely active ulcerative colitis (UC) in adults.
For more information about Bristol Myers Squibb, visit us at BMS.com
Bristol Myers wins FDA approval for expanded use of Zeposia in ulcerative colitis
May 27, 2021 4:28 PM ET
Bristol-Myers Squibb Company (BMY)Bristol-Myers Squibb Company (BMY)
By: Dulan Lokuwithana, SA News Editor7 Comments
- Bristol Myers Squibb (NYSE:BMY) announced the approval for Zeposia (ozanimod) 0.92 mg for the treatment of adults with moderately to severely active ulcerative colitis (UC).
- https://seekingalpha.com/news/3700996-bristol-myers-wins-fda-approval-for-expanded-use-of-zeposia-in-ulcerative-colitis
- https://seekingalpha.com/symbol/BMY
- https://www.zeposia.com/
NURTEC® ODT (RIMEGEPANT)
FDA APPROVES BIOHAVEN'S NURTEC® ODT (RIMEGEPANT) FOR PREVENTION: NOW THE FIRST AND ONLY MIGRAINE MEDICATION FOR BOTH ACUTE AND PREVENTIVE TREATMENT
05/27/2021- NURTEC ODT 75 mg is the first oral CGRP antagonist approved to prevent migraine- NURTEC ODT 75 mg is now the only migraine medication approved to both treat acute migraine attacks and help prevent future migraine attacks- In a pivotal Phase 3 clinical trial, NURTEC rapidly and effectively prevented migraine, reducing migraine days by 30% after just 1 week of every other day treatment; by 3 months of treatment, approximately half of patients experienced at least a 50% reduction in moderate-to-severe migraine days- NURTEC ODT was previously approved for acute treatment in all eligible adult patients with migraine, and the new indication of preventive treatment in adult patients with less than 15 headaches days per month expands the potential preventive use of NURTEC ODT to cover approximately 95% of individuals with migraine
NEW HAVEN, Conn., May 27, 2021 /PRNewswire/ -- Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN), today announced that the U.S. Food and Drug Administration (FDA) has approved NURTEC® ODT (rimegepant 75 mg) for the preventive treatment of migraine. NURTEC® ODT is indicated for adult patients with episodic migraine, e.g. those who experience less than 15 headache days per month. The approved product label was also expanded to include the use of NURTEC ODT 75 mg up to 18 doses/month, allowing for both acute and preventive therapy in the same patient. This new approval makes NURTEC ODT the first oral CGRP antagonist approved for the preventive treatment of migraine, and the only migraine medication approved as a dual therapy for both the acute and preventive treatment. NURTEC ODT is approved for acute treatment in all eligible adult patients with migraine, regardless of the number of monthly migraine days. Since approximately 95% of all U.S. migraine patients experience less than 15 headache days per month, the new indication of preventive treatment significantly expands the market potential of NURTEC ODT and provides a new preventive treatment option for the vast majority of people living with migraine.
Biohaven Patient Access Commitment
NURTEC ODT is now available in pharmacies for the acute and preventive treatment of migraine. People with migraine should talk with their healthcare provider about using NURTEC ODT to treat and prevent migraine attacks.
Biohaven is committed to supporting the migraine community by eliminating barriers to medication access. The company plans to continue the $0 co-pay card in which eligible people will pay as little as $0. For eligibility requirements and to enroll in the patient support program, please call 1-833-4-NURTEC or visit www.NURTEC.com.
About NURTEC ODT
NURTEC® ODT (rimegepant) is the first and only calcitonin gene-related peptide (CGRP) receptor antagonist available in a quick-dissolve ODT formulation that is approved by the U.S. Food and Drug Administration (FDA) for the acute treatment of migraine with or without aura and the preventive treatment of episodic migraine in adults. The activity of the neuropeptide CGRP is thought to play a causal role in migraine pathophysiology. NURTEC ODT is a CGRP receptor antagonist that works by reversibly blocking CGRP receptors, thereby inhibiting the biologic activity of the CGRP neuropeptide. The recommended dose of NURTEC ODT is 75 mg, taken as needed, up to once daily to treat or every other day to help prevent migraine attacks. For more information about NURTEC ODT, visit www.NURTEC.com. The most common adverse reaction was nausea and abdominal pain/indigestion. Avoid concomitant administration of NURTEC ODT with strong inhibitors of CYP3A4, strong or moderate inducers of CYP3A or inhibitors of P-gp or BCRP. Avoid another dose of NURTEC ODT within 48 hours when it is administered with moderate inhibitors of CYP3A4.
Indication
NURTEC ODT orally disintegrating tablets is a prescription medicine that is used to treat migraine in adults. It is for the acute treatment of migraine attacks with or without aura and the preventive treatment of episodic migraine. It is not known if NURTEC ODT is safe and effective in children.
More information about Biohaven is available at www.biohavenpharma.com.
View original content to download multimedia:http://www.prnewswire.com/news-releases/fda-approves-biohavens-nurtec-odt-rimegepant-for-prevention-now-the-first-and-only-migraine-medication-for-both-acute-and-preventive-treatment-301301304.html
SOURCE Biohaven Pharmaceutical Holding Company Ltd.
FDA approves Biohaven's NURTEC ODT as preventative treatment for migraine
May 27, 2021 4:25 PM ET Biohaven Pharmaceutical Holding Company Ltd. (BHVN)Biohaven Pharmaceutical Holding Company Ltd. (BHVN)
By: Aakash Babu, SA News Editor2 Comments
- Biohaven Pharmaceutical Holding (NYSE:BHVN) announces that the U.S. FDA has approved NURTEC ODT (rimegepant 75 mg) for the preventive treatment of migraine.
- https://seekingalpha.com/news/3700993-fda-approves-biohavens-nurtec-odt-as-preventative-treatment-for-migraine
- https://seekingalpha.com/symbol/BHVN
- https://www.nurtec.com/
GSK and Vir Biotechnology Announce Sotrovimab (VIR-7831) Receives Emergency Use Authorization from the US FDA for Treatment of Mild-to-Moderate COVID-19 in High-Risk Adults and Pediatric Patients
05/26/21 at 6:57 PM EDTPDF Version
– Treatment with sotrovimab resulted in an 85% reduction in the risk of hospitalization or death in high-risk adult outpatients compared to placebo, based on interim results from Phase 3 COMET-ICE trial –
– In vitro data indicate sotrovimab maintains activity against all known variants of concern, including the variant from India –
– Sotrovimab will be available for appropriate patients diagnosed with COVID-19 in the U.S. in the coming weeks –
– Discussions with global regulators regarding authorizations in additional countries continue to advance –
LONDON and SAN FRANCISCO, May 26, 2021 (GLOBE NEWSWIRE) -- GlaxoSmithKline plc (LSE/NYSE: GSK) and Vir Biotechnology, Inc. (Nasdaq: VIR) today announced the U.S. Food and Drug Administration (FDA) granted an Emergency Use Authorization (EUA) for sotrovimab (previously VIR-7831), an investigational single-dose monoclonal antibody, for the treatment of mild-to-moderate COVID-19 in adults and pediatric patients (12 years of age and older weighing at least 40 kg) with positive results of direct SARS-CoV-2 viral testing, and who are at high risk for progression to severe COVID-19, including hospitalization or death.
Evidence of Sotrovimab’s Profound Clinical Efficacy
The EUA was granted to sotrovimab based on an interim analysis of efficacy and safety data from the Phase 3 COMET-ICE (COVID-19 Monoclonal antibody Efficacy Trial – Intent to Care Early) trial in high-risk adult outpatients, which was stopped early by an independent data monitoring committee in March 2021 due to evidence of profound clinical efficacy. As previously announced, interim study results demonstrated an 85% (p=0.002) reduction in hospitalization for more than 24 hours or death in those receiving sotrovimab compared to placebo, the primary endpoint of the trial. The most common adverse events observed in the sotrovimab treatment group in COMET-ICE were rash (2%) and diarrhea (1%), all of which were Grade 1 (mild) or Grade 2 (moderate). No other treatment-emergent adverse events were reported at a higher rate with sotrovimab compared to placebo. The EUA includes a warning for hypersensitivity including anaphylaxis and infusion-related reactions.
In Vitro Data Indicate Sotrovimab Maintains Activity Against All Known Variants of Concern
Sotrovimab targets a conserved epitope of the spike protein that is less likely to mutate over time. The EUA submission also included data from published in vitro studies, which demonstrated that sotrovimab maintains activity against all known circulating variants of concern, including the variants from Brazil (P.1), California (B.1.427/B.1.429), India (B.1.617), New York (B.1.526), South Africa (B.1.351) and the UK (B.1.1.7). GSK and Vir will continue to evaluate the ability of sotrovimab to maintain activity against new and emerging variants. The clinical impact of these in vitro variant data is not yet known. Data collection and analysis is still ongoing.
GSK and Vir’s Commitment to Patient Access to Sotrovimab
GSK and Vir are working to make sotrovimab available to U.S. patients in the coming weeks with the intent that all appropriate patients will have access to it, with little to no out-of-pocket costs. Patients and healthcare professionals can access more information about eligibility, availability and financial support at gskcovidcontactcenter.com or by calling 866-GSK-COVID (866-475-2684).
GSK and Vir are actively working with government agencies around the world to make sotrovimab available to patients in need of treatment.
- On May 21, 2021, the European Medicines Agency’s (EMA) Committee for Human Medicinal Products (CHMP) issued a positive scientific opinion following the referral of sotrovimab to the CHMP under Article 5(3) of Regulation 726/2004. The opinion relates to the use of sotrovimab for the treatment of adults and adolescents (aged 12 years and over and weighing at least 40 kg) with COVID-19 who do not require oxygen supplementation and who are at risk of progressing to severe COVID-19.
- The EMA has also started a rolling review of data on sotrovimab that will continue until enough evidence is available to support the filing of a formal marketing authorization application.
- In April, Health Canada initiated a review of sotrovimab under the expedited Interim Order application pathway for COVID-19 drugs.
- GSK and Vir are continuing discussions with other global regulators on the regulatory pathways available so that sotrovimab can be made available to patients with COVID-19 as soon as possible.
About the COMET-ICE Study Design
The multi-center, double-blind, placebo-controlled, Phase 3 COMET-ICE trial investigated intravenous (IV) infusion of sotrovimab in adults with mild or moderate COVID-19 at high risk of progression to severe disease.
This ongoing trial evaluated the safety and efficacy of a single IV infusion of sotrovimab (500 mg) or placebo in non-hospitalized participants globally. The safety of sotrovimab is primarily based on an interim analysis from 868 patients (430 patients in the treatment arm and 438 in the placebo arm) through day 15. Among those studied, 63% were Hispanic or Latino and 7% were Black or African American. According to the Centers for Disease Control and Prevention, these populations are approximately three times more likely to be hospitalized and approximately two times more likely to die of COVID-191. The primary efficacy endpoint was the proportion of patients who have progression of COVID-19 as defined by the need for hospitalization for greater than 24 hours for acute management of illness or death.
In March 2021, an Independent Data Monitoring Committee recommended that the COMET-ICE trial be stopped for enrollment due to evidence of profound efficacy.
About the Sotrovimab Clinical Development Program
In addition to the COMET-ICE trial, the full COMET clinical development program for sotrovimab includes:
- COMET-PEAK: An ongoing Phase 2 trial with two parts: to compare the safety and viral kinetics of 500 mg intramuscularly (IM) administered sotrovimab to 500 mg intravenously administered sotrovimab among low-risk adults with mild to moderate COVID-19 and to evaluate the similarity in pharmacokinetics between sotrovimab manufactured by different processes
- COMET-TAIL: A Phase 3 trial expected to begin in the second quarter of 2021 as an early treatment for COVID-19 in high-risk adults, to assess whether IM-administered sotrovimab can reduce hospitalization or death due to COVID-19
- COMET-STAR: A Phase 3 trial expected to begin in the second half of 2021 in uninfected adults at high risk to determine whether IM-administered sotrovimab can prevent symptomatic infection.
Sotrovimab was also evaluated in the outpatient setting in BLAZE-4, a Phase 2 trial sponsored by Eli Lilly and Company, designed to assess the safety and efficacy of bamlanivimab (LY-CoV555) alone and bamlanivimab with other neutralizing antibodies, including sotrovimab, versus placebo in low-risk adults with mild to moderate COVID-19. An interim analysis found that bamlanivimab (700 mg) co-administered with sotrovimab (500 mg) demonstrated a 70% relative reduction in patients with persistently high viral load at day 7 compared to placebo, meeting the primary endpoint.
Additionally, sotrovimab, along with VIR-7832 is being evaluated in the Phase 1b/2a National Health Service-supported AGILE trial in adults with mild to moderate COVID-19. VIR-7832 is the second monoclonal antibody from the Vir-GSK collaboration to be investigated as a potential COVID-19 treatment.
About Sotrovimab (previously VIR-7831)
Sotrovimab is an investigational SARS-CoV-2 monoclonal antibody. Preclinical data suggest it has the potential to both block viral entry into healthy cells and clear infected cells. The antibody binds to an epitope on SARS-CoV-2 that is shared with SARS-CoV-1 (the virus that causes SARS), indicating that the epitope is highly conserved, which may make it more difficult for resistance to develop. Sotrovimab, which incorporates Xencor’s Xtend™ technology, also has been designed to achieve high concentration in the lungs to ensure optimal penetration into airway tissues affected by SARS-CoV-2 and to have an extended half-life.
About VIR-7832 / GSK4182137
VIR-7832 is an investigational dual-action SARS-CoV-2 monoclonal antibody. Preclinical data suggest it has the potential to both block viral entry into healthy cells and an enhanced ability to clear infected cells. The antibody binds to an epitope on SARS-CoV-2 that is shared with SARS-CoV-1 (the virus that causes SARS), indicating that the epitope is highly conserved, which may make it more difficult for resistance to develop. VIR-7832, which incorporates Xencor’s Xtend and other Fc technologies, has been designed to achieve high concentration in the lungs to ensure optimal penetration into airway tissues affected by SARS-CoV-2 and to have an extended half-life. Importantly, VIR-7832 also has been engineered to potentially enhance virus-specific T cell function, which could help treat and/or prevent COVID-19 infection.
The following is a summary of information for sotrovimab. Healthcare providers should review the Fact Sheets for information on the authorized use of sotrovimab and mandatory requirements of the EUA. Please see the FDA Letter of Authorization, Fact Sheet for Healthcare Providers, and Fact Sheet for Patients, Parents, and Caregivers.
Important Information about Sotrovimab
Sotrovimab has been authorized by the FDA for the emergency use described below. Sotrovimab is not FDA-approved for this use.
Sotrovimab is authorized only for the duration of the declaration that circumstances exist justifying the authorization of the emergency use of sotrovimab under section 564(b)(1) of the Act, 21 U.S.C. § 360bbb-3(b)(1), unless the authorization is terminated or revoked sooner.
Authorized Use
The U.S. Food and Drug Administration (FDA) has issued an Emergency Use Authorization (EUA) to permit the emergency use of the unapproved product sotrovimab for the treatment of mild-to-moderate coronavirus disease 2019 (COVID-19) in adults and pediatric patients (12 years of age and older weighing at least 40 kg) with positive results of direct SARS-CoV-2 viral testing, and who are at high risk for progression to severe COVID-19, including hospitalization or death.
About the Vir and GSK Collaboration
In April 2020, Vir and GSK entered into a collaboration to research and develop solutions for coronaviruses, including SARS-CoV-2, the virus that causes COVID-19. The collaboration uses Vir’s proprietary monoclonal antibody platform technology to accelerate existing and identify new anti-viral antibodies that could be used as therapeutic or preventive options to help address the current COVID-19 pandemic and future outbreaks. The companies will leverage GSK’s expertise in functional genomics and combine their capabilities in CRISPR screening and artificial intelligence to identify anti-coronavirus compounds that target cellular host genes. They will also apply their combined expertise to research SARS-CoV-2 and other coronavirus vaccines.
For further information please visit www.gsk.com/about-us.
For more information, please visit www.vir.bio.
GSK/Vir COVID-19 biologic wins FDA Emergency Use Authorization; Vir up 9%
May 26, 2021 11:12 PM ETGILD, GSK...By: Jonathan M Block, SA News Editor5 Comments
- The FDA has granted Emergency Use Authorization ("EUA") to sotrovimab, a monoclonal antibody from GlaxoSmithKline (NYSE:GSK) and Vir Biotechnology (NASDAQ:VIR), for mild-to-moderate COVID-19.
- https://seekingalpha.com/news/3700603-gsk-vir-covid-19-biologic-wins-fda-emergency-use-authorization-vir-up-9
- https://seekingalpha.com/symbol/GSK
- https://seekingalpha.com/symbol/VIR
VENCLYXTO® (venetoclax) in Combination with a Hypomethylating Agent
May 25, 2021
AbbVie Receives European Commission Approval of VENCLYXTO® (venetoclax) in Combination with a Hypomethylating Agent for Patients with Newly Diagnosed Acute Myeloid Leukemia Who Are Ineligible for Intensive Chemotherapy
-- VENCLYXTO® (venetoclax) in combination with a hypomethylating agent is a new regimen approved by the European Commission (EC) for patients with newly diagnosed acute myeloid leukemia (AML) who are ineligible for intensive chemotherapy [1]
-- Approval is based on data from AbbVie's clinical trial program for VENCLYXTO, including the Phase 3 VIALE-A trial, which showed patients treated with VENCLYXTO in combination with azacitidine demonstrated improvements in overall survival versus patients treated with placebo in combination with azacitidine [2]
-- Approval is also based on results of the Phase 1b M14-358 trial which showed patients treated with VENCLYXTO in combination with azacitidine or decitabine achieved high remission rates [3]
NORTH CHICAGO, Ill., May 25, 2021 /PRNewswire/ -- AbbVie (NYSE: ABBV) announced today that the European Commission (EC) has approved VENCLYXTO® (venetoclax) in combination with a hypomethylating agent, azacitidine or decitabine, for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) who are ineligible for intensive chemotherapy.1 The approval is valid in all 27 member states of the EU, as well as Iceland, Liechtenstein, and Norway.
"VENCLYXTO has proven incremental overall survival in treating newly diagnosed AML in patients who are ineligible for intensive chemotherapy when treated with VENCLYXTO plus azacitidine compared to those treated with azacitidine alone," said Mohamed Zaki, M.D., Ph.D., vice president and head, global oncology development, AbbVie. "We look forward to bringing VENCLYXTO to more AML patients who can potentially benefit from this important new treatment option in EU countries."
This is the third extension of indications for VENCLYXTO, a first-in-class B-cell lymphoma-2 (BCL-2) inhibitor. BCL-2 is a protein that prevents cancer cells from undergoing apoptosis, the process that leads to the natural death or self-destruction of cancer cells.1
About the VENCLYXTO AML Clinical Trial Program
AbbVie's clinical trial program to evaluate VENCLYXTO combination with a hypomethylating agent in patients with newly diagnosed acute myeloid leukemia (AML) who were ineligible for intensive chemotherapy included two studies conducted around the world.
VIALE-A (M15-656) Phase 3 Trial2
The randomized, double-blind, placebo-controlled VIALE-A (M15-656) trial evaluated the efficacy and safety of VENCLYXTO in combination with azacitidine in patients with newly diagnosed AML who were ineligible for intensive chemotherapy. The study met its primary endpoints of statistically significant improvement of overall survival (OS) and composite complete remission (complete remission [CR]+complete remission with incomplete hematologic recovery [CRi]) (CR + CRi). Overall survival was 14.7 months for the VENCLYXTO plus azacitidine arm versus 9.6 months in the placebo plus azacitidine arm, and the composite complete remission rate was 66.4 percent versus 28.3 percent, respectively. The study also met secondary endpoints, with the VENCLYXTO plus azacitidine arm resulting in a CR rate of 36.7 percent vs. 17.9 percent in the placebo plus azacitidine arm. The safety profile of VENCLYXTO plus azacitidine was consistent with the known side-effect profiles of both agents, and adverse events (AEs) were consistent with expectations for an older AML population. The most frequently reported serious AEs in the VENCLYXTO plus azacitidine arm and placebo plus azacitidine arm were febrile neutropenia (in 30 percent and 10 percent), pneumonia (in 17 percent and 22 percent), sepsis (in 6 percent and 8 percent), and haemorrhage (in 9 percent and 6 percent), respectively.2
M14-358 Phase 1b Trial3
The non-randomized, open-label M14-358 trial evaluated VENCLYXTO in combination with azacitidine or decitabine in patients with newly diagnosed AML who were ineligible for intensive chemotherapy. Patients treated with VENCLYXTO in combination with decitabine achieved a CR+CRi rate of 74 percent and a 30-day mortality rate of 6.5 percent. The median follow-up was 40.4 months (range: 0.7 to 42.7 months) for venetoclax in combination with decitabine. The most frequently reported serious AEs (≥5%) in patients receiving VENCLYXTO in combination with decitabine were febrile neutropenia, pneumonia, bacteraemia and sepsis, with neutropenia reported in 35 percent (all grades) and 35 percent (grade 3 or 4) of patients in the venetoclax + decitabine arm. No events of laboratory or clinical TLS were reported with venetoclax in combination with decitabine.
About VENCLYXTO® (venetoclax)
VENCLYXTO® (venetoclax) is a first-in-class medicine that selectively binds and inhibits the B-cell lymphoma-2 (BCL-2) protein. In some blood cancers, BCL-2 prevents cancer cells from undergoing their natural death or self-destruction process, called apoptosis. VENCLYXTO targets the BCL-2 protein and works to help restore the process of apoptosis.
VENCLYXTO is also approved in combination with obinutuzumab for the treatment of adult patients with previously untreated chronic lymphocytic leukemia (CLL), in combination with rituximab for the treatment of adult patients with CLL who have received at least one prior therapy, and as a monotherapy for the treatment of CLL in the presence or absence of 17p deletion or TP53 mutation in adult patients who are unsuitable for or have failed a B-cell receptor pathway inhibitor.1
VENCLYXTO is being developed by AbbVie and Roche. It is jointly commercialized by AbbVie and Genentech, a member of the Roche Group, in the U.S. and by AbbVie outside of the U.S. Together, the companies are committed to BCL-2 research and to studying venetoclax in clinical trials across several blood. Venetoclax is approved in more than 80 countries, including the U.S.
Indications and Important Venclyxto (venetoclax) EU Safety Information1
Indications
Venclyxto in combination with obinutuzumab is indicated for the treatment of adult patients with previously untreated chronic lymphocytic leukaemia (CLL).
Venclyxto in combination with rituximab is indicated for the treatment of adult patients with CLL who have received at least one prior therapy.
Venclyxto monotherapy is indicated for the treatment of CLL:
- In the presence of 17p deletion or TP53 mutation in adult patients who are unsuitable for or have failed a B-cell receptor pathway inhibitor, or
- In the absence of 17p deletion or TP53 mutation in adult patients who have failed both chemoimmunotherapy and a B-cell receptor pathway inhibitor.
Venclyxto in combination with a hypomethylating agent is indicated for the treatment of adult patients with newly diagnosed acute myeloid leukaemia (AML) who are ineligible for intensive chemotherapy.
For more information, please visit http://www.abbvie.com/oncology.
For more information about AbbVie, please visit us at www.abbvie.com.
AbbVie's Venclyxto combo OK'd in Europe for treatment of acute myeloid leukemia
May 25, 2021 8:55 AM ET AbbVie Inc. (ABBV) AbbVie Inc. (ABBV) By: Mamta Mayani, SA News Editor
- The European Commission (EC) has approved AbbVie's (NYSE:ABBV) Venclyxto (venetoclax) in combination with a hypomethylating agent, azacitidine or decitabine, for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) who are ineligible for intensive chemotherapy.
- https://seekingalpha.com/news/3699828-abbvies-venclyxto-combo-okd-in-europe-for-treatment-of-acute-myeloid-leukemia
- https://seekingalpha.com/symbol/ABBV
- https://seekingalpha.com/symbol/RHHBY
- https://www.venclexta.com/
- https://www.medicines.org.uk/emc/product/10476/pil
- https://www.abbviepro.com/gb/public/public-listing.html
LY3819469
Dicerna Announces FDA Acceptance of Lilly’s Investigational New Drug (IND) Application for LY3819469
May 27, 2021 PDF Version
– Second GalXC™ RNAi Investigational New Drug (IND) Application Filed by Lilly Under Companies’ Global Research Collaboration and Licensing Agreement –
LEXINGTON, Mass.--(BUSINESS WIRE)--May 27, 2021-- Dicerna Pharmaceuticals, Inc. (Nasdaq: DRNA) (the “Company” or “Dicerna”), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, today announced the U.S. Food and Drug Administration (“FDA”) acceptance of the Investigational New Drug (“IND”) application filed by Eli Lilly and Company (“Lilly”) for LY3819469, the second clinical-stage candidate to emerge from Dicerna’s collaboration with Lilly. The IND milestone triggers a $10 million payment to Dicerna and enables Lilly to initiate a Phase 1 clinical trial of LY3819469, an investigational GalXC™ RNAi candidate targeting the LPA gene as a potential treatment of cardiometabolic diseases.
“This milestone marks the second IND generated through our productive collaboration with Lilly,” said Bob D. Brown, Ph.D., Chief Scientific Officer and Executive Vice President of R&D at Dicerna. “In addition to the two candidates now at clinical stage, there are so far, nine discovery research programs connected to this alliance, emphasizing Dicerna’s and Lilly’s shared commitment to bringing forward new RNAi-based therapies to treat a broad range of diseases.”
The IND filing for LY3819469 is the second development milestone achieved under a 2018 global licensing and research collaboration between Dicerna and Lilly focused on the discovery, development and commercialization of potential new therapies for cardiometabolic and neurodegenerative diseases and pain. This investigational cardiometabolic therapy and future therapies to emerge from the two companies’ collaboration leverage Dicerna's proprietary GalXC RNAi technology platform.
Under the agreement, Dicerna is eligible to receive up to $350 million in development and commercialization milestones for each GalXC hepatocyte target and $355 million for each non-hepatocyte target, as well as tiered royalties ranging from the mid-single-digits to low double-digits on potential product sales.
About RNAi and Dicerna’s GalXC™ RNAi Platform Technologies
Ribonucleic acid interference, or RNAi, provides a unique advantage to other disease inhibitor technologies, like small-molecule pharmaceuticals or monoclonal antibodies. Instead of targeting proteins after they have been produced and released, RNAi silences the genes themselves via the specific destruction of the messenger RNA (mRNA) made from the gene. Rather than seeking to inhibit a protein, the RNAi approach can prevent a disease-causing protein’s creation, directly impacting disease manifestation.
Dicerna’s proprietary GalXC™ RNAi platform aims to advance the development of next-generation RNAi-based therapies. Investigational therapeutics developed using our flagship GalXC technology utilize a proprietary N-acetyl-D-galactosamine (GalNAc)-mediated structure of double-stranded RNA molecules that are designed to bind specifically to receptors on liver cells, leading to selective hepatocyte internalization and access to the RNAi machinery within the cells. Dicerna is continuously innovating and exploring new applications of RNAi technology beyond GalNAc-mediated delivery to the liver, including alternative RNA structures and fully synthetic ligands that target other tissues and enable new therapeutic applications, referred to as GalXC-Plus™.
For more information, please visit www.dicerna.com.
GalXC™ and GalXC-Plus™ are trademarks of Dicerna Pharmaceuticals, Inc.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210527005186/en/
Source: Dicerna Pharmaceuticals, Inc.
FDA accepts Lilly and Dicerna's cardiometabolic diseases IND
May 27, 2021 7:37 AM ET
Dicerna Pharmaceuticals, Inc. (DRNA)Dicerna Pharmaceuticals, Inc. (DRNA)Eli Lilly and Company (LLY)
By: Aakash Babu, SA News Editor
- Dicerna Pharmaceuticals (NASDAQ:DRNA) announces that the U.S. FDA has accepted the Investigational New Drug (“IND”) application filed by Eli Lilly (NYSE:LLY) for LY3819469, which is a potential treatment of cardiometabolic diseases.
- https://seekingalpha.com/news/3700702-fda-accepts-lilly-and-dicernas-cardiometabolic-diseases-ind
- https://seekingalpha.com/symbol/DRNA
- https://seekingalpha.com/symbol/LLY
- https://dicerna.com/pipeline/
Teclistamab
Janssen's Updated Phase 1 Results for Teclistamab Suggest Deep, Durable Responses in Patients with Heavily Pretreated Multiple Myeloma
May 24, 2021United States
Subcutaneous administration of BCMAxCD3 T-cell redirecting bispecific antibody demonstrated clinical activity and a promising safety profile according to new data at ASCO
RARITAN, N.J., May 24, 2021 -- The Janssen Pharmaceutical Companies of Johnson & Johnson announced today longer follow-up results from the Phase 1 MajesTEC-1 study, the first-in-human dose-escalation study of teclistamab, an off-the-shelf T-cell redirecting bispecific antibody, in the treatment of patients with relapsed or refractory multiple myeloma (NCT03145181). With a median follow-up of more than six months, an overall response rate (ORR) of 65 percent was observed at the recommended subcutaneous (SC) Phase 2 dose (RP2D) in a cohort of heavily pretreated patients (n=40) who had received a median of five prior lines of therapy.1 These data will be featured during the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting as an oral presentation on Tuesday, June 8 (Abstract #8007).1
Study results show that responses were durable and deepened over time – 58 percent of patients receiving teclistamab achieved a very good partial response (VGPR) or better, and 40 percent achieved a complete response (CR) or better at the RP2D. The median time to first confirmed response was one month.1 After a median follow-up of 7.1 months (range, 3.0–12.2 months), median duration of response was not reached and 85 percent (22/26) of responders were alive and continuing treatment.1
There were no dose-limiting toxicities at the RP2D in part 1 of the study. Grade 1 neurotoxicity was reported in one patient treated at the RP2D.1 The most common adverse events at the RP2D were cytokine release syndrome (70 percent; all Grade 1/2) and neutropenia (65 percent; 40 percent Grade 3/4). The promising safety, efficacy, pharmacokinetics and pharmacodynamics confirm the selection of the 1500 ug/kg SC as the RP2D.1
Forty patients were treated at the RP2D, identified as 1500 µg/kg SC.1 Patients receiving the RP2D of teclistamab in this study had received a median of five prior lines of therapy (range 2–11);100 percent were triple-class (proteasome inhibitor [PI], immunomodulatory drug (IMiD), CD38 antibody) exposed; 65 percent (n=26) were penta-drug (2 PIs, 2 IMiDs, CD38 antibody) exposed; 83 percent (n=33) were triple-class refractory; 38 percent (n=15) were penta-drug refractory; 83 percent (n=33) were refractory to their last line of therapy.1 Patients with triple-class refractory and penta-drug refractory multiple myeloma often experience poor survival outcomes as treatment options are limited.1
About Teclistamab
Teclistamab is an investigational, T-cell redirecting bispecific antibody targeting both BCMA and CD3. BCMA, B-cell maturation antigen, is expressed at high levels on multiple myeloma cells.3,4,5,6,7 Teclistamab redirects CD3-positive T-cells to BCMA-expressing myeloma cells to induce killing of tumor cells.5,6 Results from preclinical studies demonstrate that teclistamab kills myeloma cell lines and bone marrow-derived myeloma cells from heavily pretreated patients.6
Teclistamab is currently being evaluated in a Phase 2 clinical study for the treatment of relapsed or refractory multiple myeloma (NCT04557098) and is also being explored in combination studies (NCT04586426, NCT04108195, NCT04722146). In 2020, the European Commission and the U.S. Food and Drug Administration each granted teclistamab orphan drug designation for the treatment of multiple myeloma.
Learn more at www.janssen.com.
https://clinicaltrials.gov/ct2/show/NCT04557098
biotecMAX™ April/May 2021 Insight
LIBTAYO® (CEMIPLIMAB)
LIBTAYO® (CEMIPLIMAB) RECEIVES POSITIVE CHMP OPINION FOR THE TREATMENT IN EUROPE OF TWO ADVANCED CANCERS
TARRYTOWN, N.Y. and PARIS, May 24, 2021 /PRNewswire/ --
Libtayo recommended for approval in the first-line treatment of certain patients with advanced non-small cell lung cancer whose tumors have ≥50% PD-L1 expression
Libtayo also recommended for approval in patients with advanced basal cell carcinoma who have progressed on or are intolerant to a hedgehog pathway inhibitor
CHMP has now issued three positive opinions for Libtayo in advanced cancers
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted positive opinions for Libtayo® (cemiplimab) as monotherapy in two advanced cancers.
The CHMP recommended the approval of Libtayo for the first-line treatment of adults with non-small cell lung cancer (NSCLC) expressing PD-L1 in ≥50% of tumor cells with no EGFR, ALK or ROS1 aberrations. Patients must have metastatic disease or locally advanced disease that is not a candidate for definitive chemoradiation. Libtayo was also recommended for approval in adults with locally advanced or metastatic basal cell carcinoma (BCC) who have progressed on or are intolerant to a hedgehog pathway inhibitor (HHI). The European Commission is expected to make a decision on both indications in the coming months.
The positive opinion for Libtayo in advanced NSCLC is based on results from a Phase 3 trial, which allowed for the enrollment of patients with disease characteristics frequently underrepresented in advanced NSCLC pivotal trials, including those with pre-treated and clinically stable brain metastases or locally advanced NSCLC and who were not candidates for definitive chemoradiation. Results from the pivotal trial were published in The Lancet in February 2021.
About the Phase 3 Trial in Advanced NSCLC
EMPOWER-Lung 1 was an open-label, randomized, multi-center Phase 3 trial designed to investigate Libtayo monotherapy compared to platinum-doublet chemotherapy as first-line treatment in patients with advanced NSCLC who tested positive for PD-L1 in ≥50% of tumor cells and had no EGFR, ALK or ROS1 aberrations. PD-L1 expression was confirmed using the Agilent Dako PD-L1 IHC 22C3 pharmDx kit. The primary endpoints were overall survival and progression-free survival, and secondary endpoints included objective response rate (ORR), duration of response (DOR) and quality of life. In 2020, the trial was stopped early due to significant improvement in overall survival.
The trial randomized 710 patients with either previously untreated metastatic NSCLC (stage IV) or locally advanced NSCLC (stage IIIB/C) who were not candidates for surgical resection or definitive chemoradiation or who had progressed after treatment with definitive chemoradiation. Among those enrolled, 12% had pre-treated and clinically stable brain metastases and 16% had locally advanced NSCLC that was not a candidate for definitive chemoradiation.
Patients whose disease progressed in the trial were able to change their therapy: those assigned to chemotherapy were allowed to crossover to Libtayo treatment, while those assigned to Libtayo monotherapy were allowed to continue Libtayo treatment and add four cycles of chemotherapy. There was a >70% crossover rate to Libtayo following disease progression on chemotherapy.
About the Pivotal Trial in Advanced BCC
EMPOWER-BCC 1 was an open-label, multi-center, non-randomized Phase 2 trial of patients with unresectable locally advanced or metastatic BCC (nodal or distant). Patients in both cohorts had either progressed on HHI therapy, had not had an objective response after nine months on HHI therapy, or were intolerant of prior HHI therapy. The primary efficacy endpoint was confirmed ORR and a key secondary endpoint was DOR, assessed by independent central review.
About Libtayo
Libtayo is a fully human monoclonal antibody targeting the immune checkpoint receptor PD-1 on T-cells. By binding to PD-1, Libtayo has been shown to block cancer cells from using the PD-1 pathway to suppress T-cell activation.
Libtayo is currently approved as the first systemic treatment in the U.S., EU and other countries for adults with metastatic CSCC or locally advanced CSCC who are not candidates for curative surgery or curative radiation. In the U.S., Libtayo is also approved as the first immunotherapy indicated for patients with advanced BCC previously treated with an HHI or for whom an HHI is not appropriate that is either locally advanced (full approval) or metastatic (accelerated approval), as well as for the first-line treatment of certain patients with advanced NSCLC with ≥50% PD-L1 expression and no EGFR, ALK or ROS1 aberrations.
The generic name for Libtayo in its approved U.S. indications is cemiplimab-rwlc, with rwlc as the suffix designated in accordance with Nonproprietary Naming of Biological Products Guidance for Industry issued by the U.S. Food and Drug Administration (FDA).
The extensive clinical program for Libtayo is focused on difficult-to-treat cancers. Current clinical development programs include Libtayo in combination with chemotherapy for advanced NSCLC irrespective of PD-L1 expression and Libtayo monotherapy for advanced cervical cancer. Libtayo is also being investigated in combination with either conventional or novel therapeutic approaches for other solid tumors and blood cancers. These potential uses are investigational, and their safety and efficacy have not been evaluated by any regulatory authority.
Libtayo is being jointly developed by Regeneron and Sanofi under a global collaboration agreement.
What is Libtayo?
Libtayo is a prescription medicine used to treat people with a type of skin cancer called cutaneous squamous cell carcinoma (CSCC) that has spread or cannot be cured by surgery or radiation.
Libtayo is a prescription medicine used to treat people with a type of skin cancer called basal cell carcinoma that cannot be removed by surgery (locally advanced BCC) and have received treatment with an HHI, or cannot receive treatment with an HHI.
Libtayo is a prescription medicine used to treat people with a type of skin cancer called basal cell carcinoma that has spread (metastatic BCC) and have received treatment with a hedgehog pathway inhibitor (HHI), or cannot receive treatment with an HHI. This use is approved based on how many patients responded to treatment and how long they responded. Studies are ongoing to provide additional information about clinical benefit.
Libtayo is a prescription medicine used to treat people with a type of lung cancer called non-small cell lung cancer (NSCLC). Libtayo may be used as your first treatment when your lung cancer has not spread outside your chest (locally advanced lung cancer) and you cannot have surgery or chemotherapy with radiation, or your lung cancer has spread to other areas of your body (metastatic lung cancer), and your tumor tests positive for high "PD-L1" and your tumor does not have an abnormal "EGFR", "ALK "or "ROS1" gene.
It is not known if Libtayo is safe and effective in children.
For additional information about the company, please visit www.regeneron.com
View original content:http://www.prnewswire.com/news-releases/libtayo-cemiplimab-receives-positive-chmp-opinion-for-the-treatment-in-europe-of-two-advanced-cancers-301297287.html
SOURCE Regeneron Pharmaceuticals, Inc.
EMA advisory group backs Regeneron, Sanofi's Libtayo in lung and skin cancer
May 24, 2021 1:38 AM ET Regeneron Pharmaceuticals, Inc. (REGN), SNY Regeneron Pharmaceuticals, Inc. (REGN)Sanofi (SNY)
By: Mamta Mayani, SA News Editor
- The EMA's Committee for Medicinal Products for Human Use (CHMP) has adopted positive opinions for Regeneron Pharmaceuticals (NASDAQ:REGN) and Sanofi's (NASDAQ:SNY) Libtayo (cemiplimab) as monotherapy in two advanced cancers.
- https://seekingalpha.com/news/3699313-ema-advisory-group-backs-regeneron-sanofis-libtayo-in-lung-and-skin-cancer
- https://seekingalpha.com/symbol/SNY
- https://seekingalpha.com/symbol/REGN
- https://www.libtayo.com/
Valoctocogene Roxaparvovec
European Medicines Agency Grants BioMarin's Request for Accelerated Assessment of Valoctocogene Roxaparvovec for Treatment of Severe Hemophilia A
MAA Resubmission on Track for June 2021May 24, 2021
SAN RAFAEL, Calif., May 24, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the European Medicines Agency (EMA) has granted its request for accelerated assessment of valoctocogene roxaparvovec, for adults with severe hemophilia A. Accelerated assessment reduces the time frame for the EMA Committee for Medicinal Products for Human Use (CHMP) and Committee for Advanced Therapies (CAT) to review a MAA for an Advanced Therapy Medicinal Product (ATMP). A CHMP opinion is expected in the first half of 2022.
"BioMarin is pleased that the EMA has granted accelerated assessment for the review of valoctocogene roxaparvovec, which acknowledges its therapeutic innovative potential, and the unmet medical need that exists" said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin. "We continue to work closely with the EMA to make valoctocogene roxaparvovec, the potential first gene therapy to treat hemophilia A, available as soon as possible."
BioMarin plans to submit a Marketing Authorization Application (MAA) for valoctocogene roxaparvovec for the treatment of severe hemophilia A in June 2021. The submission will include 52 weeks of data from the Phase 3 GENEr8-1 study, as well as four and three years of follow-up from the respective dose cohorts in the ongoing Phase 1/2 dose escalation study.
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 actively enrolling participants in 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. The Company is running a Phase 1/2 Study with the 6e13kg/vg dose of valoctocogene roxaparvovec in approximately 10 participants with pre-existing AAV5 antibodies, as well as another Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with hemophilia A with active or prior FVIII inhibitors.
For additional information, please visit www.biomarin.com.
EMA grants Biomarin's request for accelerated assessment of valoctocogene roxaparvovec in severe hemophilia A
May 24, 2021 8:48 AM ETBioMarin Pharmaceutical Inc. (BMRN)BioMarin Pharmaceutical Inc. (BMRN)By: Mamta Mayani, SA News Editor
- BioMarin Pharmaceutical (NASDAQ:BMRN) announces that the EMA has granted its request for accelerated assessment of valoctocogene roxaparvovec, for adults with severe hemophilia A.
- https://seekingalpha.com/news/3699410-ema-grants-biomarins-request-for-accelerated-assessment-of-valoctocogene-roxaparvovec-in-severe-hemophilia-a
- https://seekingalpha.com/symbol/BMRN
- https://www.biomarin.com/
STELARA® (ustekinumab)
anssen Presents Results of First Head-to-Head Study of Biologic Therapies in Patients with Moderate to Severe Crohn’s Disease
May 23, 2021United States
Late-breaker is one of 20 Janssen abstracts, 16 of which show the safety profile and efficacy of STELARA in treating Crohn’s disease and ulcerative colitis at
Digestive Disease Week (DDW) Virtual 2021
SPRING HOUSE, Pa., May 23, 2021 -- The Janssen Pharmaceutical Companies of Johnson & Johnson today announced efficacy and safety data for STELARA® (ustekinumab) in Crohn's disease (CD) and ulcerative colitis (UC),1-4 including data from the SEAVUE study, the first head-to-head study of biologic therapies in patients with CD, presented in a Clinical Science Late-Breaking Abstract Plenary session.1 SEAVUE data showed treatment with STELARA demonstrated high rates of clinical remission, corticosteroid-free remission, clinical response and endoscopic response through one year in biologic-naïve patients with moderately to severely active CD, although the primary endpoint of statistical superiority versus adalimumab was not demonstrated.1 These head-to-head data are one of 20 abstracts Janssen presented from the Company's gastroenterology pipeline and portfolio at DDW Virtual 2021, which took place May 21-23.1-4
STELARA vs. adalimumab efficacy and safety in biologic-naïve CD patients through one year (Presentation #775d):1 The SEAVUE study examined a total of 386 patients with moderately to severely active CD. Patients were randomized 1:1 to treatment with STELARA approximately 6 mg/kg intravenous (IV) at baseline, then 90 mg subcutaneous (SC) every eight weeks (q8w), or treatment with adalimumab 160/80 mg SC at baseline/week two, then 40 mg SC every two weeks per the U.S. Food and Drug Administration-approved regimens without dose modifications. Results did not show statistically significant differences:
About DDW
Digestive Disease Week® (DDW) is the largest international gathering of physicians, researchers and academics in the fields of gastroenterology, hepatology, endoscopy and gastrointestinal surgery. Jointly sponsored by the American Association for the Study of Liver Diseases (AASLD), the American Gastroenterological Association (AGA) Institute, the American Society for Gastrointestinal Endoscopy (ASGE) and the Society for Surgery of the Alimentary Tract (SSAT), DDW is a fully virtual meeting from May 21-23, 2021. The meeting showcases more than 2,000 abstracts and hundreds of lectures on the latest advances in GI research, medicine and technology. More information can be found at www.ddw.org.
About SEAVUE7
SEAVUE is a Phase 3b, multicenter, randomized, blinded, active-controlled study to compare the efficacy and safety of STELARA to adalimumab in the treatment of biologic-naïve patients with moderately to severely active Crohn's disease. The study consists of screening (within 1 to 5 weeks prior to week 0), treatment phase (weeks 0 to 52), and follow-up phase (up to week 76). Study assessments include Crohn's Disease Activity Index (CDAI), video ileocolonoscopy; CD-related healthcare utilization; patient-reported outcomes (PROs); laboratory evaluations; biomarkers; review of concomitant medications and adverse events (AEs); and evaluation of serum concentrations of study agent as well as development of antibodies to study agent. All participants were randomly assigned to receive either ustekinumab or adalimumab. No participants were treated with placebo only.
About IM-UNITI8
IM-UNITI, a Phase 3, multicenter, randomized, double-blind, placebo-controlled, parallel group study, evaluated the efficacy and safety of STELARA maintenance therapy in adult patients with moderate to severe Crohn's disease. Patients who had responded to a single IV dose of STELARA in the UNITI-1 or UNITI-2 induction studies were randomized equally to receive maintenance SC STELARA 90 mg q8w or q12w, or placebo. There were 1,281 patients enrolled in the maintenance study. In randomized patients who met loss of response criteria between weeks 8–32, a one-time dose adjustment to 90 mg q8w occurred. All patients completing week 44 were eligible to enter the long-term extension program, continuing their current regimen up to week 252.
About STELARA® (ustekinumab)16
STELARA® (ustekinumab) is a fully human monoclonal antibody and is the first biologic treatment to selectively inhibit the interleukin (IL)-12 and IL-23 pathways. STELARA 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) adult patients (18 years or older) with active psoriatic arthritis, used alone or in combination with methotrexate (MTX); 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®.
IMPORTANT SAFETY INFORMATION
STELARA® is a prescription medicine that affects your immune system. STELARA® can increase your chance of having serious side effects including:
https://www.stelarainfo.com/crohns-disease/
Please read the full Prescribing Information and Medication Guide for STELARA® and discuss any questions you have with your doctor.
Learn more at www.janssen.com.
STELARA® Prescribing Information. Available at: http://www.janssenlabels.com/package-insert/product-monograph/prescribing-information/STELARA-pi.pdf. Accessed April 22, 2021
Janssen presents Stelara data from late-stage Seavue study in Crohn's disease
May 24, 2021 12:51 AM ET
Johnson & Johnson (JNJ)Johnson & Johnson (JNJ)
By: Mamta Mayani, SA News Editor1 Comment
- The Janssen Pharmaceutical Companies of Johnson & Johnson (NYSE:JNJ) announces data from the Phase 3b SEAVUE study evaluating Stelara (ustekinumab) vs. adalimumab in Crohn's disease (CD).
- https://seekingalpha.com/news/3699311-janssen-presents-stelara-data-from-late-stage-seavue-study-in-crohns-disease
- https://seekingalpha.com/symbol/JNJ
Opdivo (nivolumab) plus Yervoy (ipilimumab)
Bristol Myers Squibb Receives Positive CHMP Opinion Recommending Opdivo (nivolumab) plus Yervoy (ipilimumab) for Treatment of Mismatch Repair Deficient or Microsatellite Instability–High Metastatic Colorectal Cancer After Prior Chemotherapy
05/21/2021CATEGORY:
Application based on positive results from the Phase 2 CheckMate -142 trial demonstrating a durable clinical benefit in the Opdivo plus Yervoy cohort
Recommendation from the CHMP represents the first positive opinion in the EU for a dual immunotherapy in colorectal cancer
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) recommended approval of Opdivo (nivolumab) in combination with Yervoy (ipilimumab) for the treatment of adult patients with mismatch repair deficient (dMMR) or microsatellite instability-high (MSI-H) metastatic colorectal cancer (mCRC) after prior fluoropyrimidine-based combination chemotherapy. The opinion was based on data from the Phase 2 CheckMate -142 trial. The European Commission (EC), which is authorized to approve medicines for the European Union (EU), will now review the CHMP recommendation.
“Metastatic colorectal cancers with mismatch repair deficient or microsatellite instability-high biomarkers can be difficult to treat, and patients who progress on or after first-line chemotherapy still face a great unmet need despite overall progress in the field,” said Ian M. Waxman, M.D., development lead, gastrointestinal cancers, Bristol Myers Squibb. "The CHMP’s positive opinion further supports our goal to advance rational combinations that target distinct but complementary immune pathways. We look forward to the EC’s decision later this year and are excited by the potential positive impact this novel combination could have for patients in need throughout the EU.”
Opdivo plus Yervoy received approval from the U.S. Food and Drug Administration (FDA) in July 2018 for the treatment of adult and pediatric patients 12 years and older with MSI-H or dMMR mCRC that has progressed following treatment with fluoropyrimidine, oxaliplatin and irinotecan. Opdivo plus Yervoy was also approved in Japan by the Pharmaceuticals and Medical Devices Agency (PMDA) in September 2020 for the treatment of MSI-H unresectable, advanced or recurrent colorectal cancer progressing after cancer chemotherapy.
About CheckMate -142
CheckMate -142 included a multicenter, non-randomized, open-label cohort investigating Opdivo plus Yervoy in patients with mismatch repair deficient (dMMR) or microsatellite instability–high (MSI-H) metastatic colorectal cancer (mCRC) whose disease had progressed during or after prior treatment with fluoropyrimidine, oxaliplatin and irinotecan.
In this combination cohort, patients received Opdivo 3 mg/kg with Yervoy 1 mg/kg every three weeks for four doses, followed by Opdivo 3 mg/kg as a single agent every two weeks until disease progression, death, or unacceptable toxicity. Efficacy outcome measures included objective response rate (ORR) as assessed by blinded independent central review using Response Evaluation Criteria in Solid Tumors (RECIST v1.1) and duration of response (DoR).
About Opdivo
Opdivo is a programmed death-1 (PD-1) immune checkpoint inhibitor that is designed to uniquely harness the body’s own immune system to help restore anti-tumor immune response. By harnessing the body’s own immune system to fight cancer, Opdivo has become an important treatment option across multiple cancers.
Opdivo’s leading global development program is based on Bristol Myers Squibb’s scientific expertise in the field of Immuno-Oncology, and includes a broad range of clinical trials across all phases, including Phase 3, in a variety of tumor types. To date, the Opdivo clinical development program has treated more than 35,000 patients. The Opdivo trials have contributed to gaining a deeper understanding of the potential role of biomarkers in patient care, particularly regarding how patients may benefit from Opdivo across the continuum of PD-L1 expression.
In July 2014, Opdivo was the first PD-1 immune checkpoint inhibitor to receive regulatory approval anywhere in the world. Opdivo is currently approved in more than 65 countries, including the United States, the European Union, Japan, and China. In October 2015, the Company’s Opdivo and Yervoy combination regimen was the first Immuno-Oncology combination to receive regulatory approval for the treatment of metastatic melanoma and is currently approved in more than 50 countries, including the United States and the European Union.
About Yervoy
Yervoy is a recombinant, human monoclonal antibody that binds to the cytotoxic T-lymphocyte-associated antigen-4 (CTLA-4). CTLA-4 is a negative regulator of T-cell activity. Yervoy binds to CTLA-4 and blocks the interaction of CTLA-4 with its ligands, CD80/CD86. Blockade of CTLA-4 has been shown to augment T-cell activation and proliferation, including the activation and proliferation of tumor infiltrating T-effector cells. Inhibition of CTLA-4 signaling can also reduce T-regulatory cell function, which may contribute to a general increase in T-cell responsiveness, including the anti-tumor immune response. On March 25, 2011, the U.S. Food and Drug Administration (FDA) approved Yervoy 3 mg/kg monotherapy for patients with unresectable or metastatic melanoma. Yervoy is approved for unresectable or metastatic melanoma in more than 50 countries. There is a broad, ongoing development program in place for Yervoy spanning multiple tumor types.
INDICATIONS
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express PD-L1 (≥1%) as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab) and 2 cycles of platinum-doublet chemotherapy, is indicated for the first-line treatment of adult patients with metastatic or recurrent non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab) is indicated for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving OPDIVO.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with unresectable malignant pleural mesothelioma (MPM).
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of patients with intermediate or poor risk advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab), in combination with cabozantinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab) is indicated for the treatment of patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy.
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with classical Hodgkin lymphoma (cHL) that has relapsed or progressed after autologous hematopoietic stem cell transplantation (HSCT) and brentuximab vedotin or after 3 or more lines of systemic therapy that includes autologous HSCT. This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab) is indicated for the treatment of patients with recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN) with disease progression on or after platinum-based therapy.
OPDIVO® (nivolumab) is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of adult and pediatric (12 years and older) patients with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab) is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection.
OPDIVO® (nivolumab) is indicated for the treatment of patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) after prior fluoropyrimidine- and platinum-based chemotherapy.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of completely resected esophageal or gastroesophageal junction cancer with residual pathologic disease in patients who have received neoadjuvant chemoradiotherapy (CRT).
OPDIVO® (nivolumab), in combination with fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the treatment of patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma.
Please see US Full Prescribing Information for OPDIVO and YERVOY.
For more information about Bristol Myers Squibb, visit us at BMS.com
Bristol Myers wins positive opinion in Europe for Opdivo and Yervoy combo for colorectal cancer
May 21, 2021 4:35 PM ET Bristol-Myers Squibb Company (BMY)Bristol-Myers Squibb Company (BMY) By: Dulan Lokuwithana, SA News Editor
- Bristol Myers Squibb (NYSE:BMY) announced that the European Medicines Agency (EMA) has recommended Opdivo (nivolumab) and Yervoy (ipilimumab) as a second-line treatment for a certain group of adult patients with metastatic colorectal cancer ((mCRC)).
- https://seekingalpha.com/news/3699260-bristol-myers-wins-positive-opinion-in-europe-for-opdivo-and-yervoy-combo-for-colorectal-cancer
- https://seekingalpha.com/symbol/BMY
- https://www.bms.com/
- https://www.opdivo.com/
SKYSONA (elivaldogene autotemcel, formerly Lenti-D™ gene therapy)
bluebird bio Receives Positive CHMP Opinion for SKYSONA™ (elivaldogene autotemcel, Lenti-D™) Gene Therapy for Patients Less Than 18 Years of Age with Early Cerebral Adrenoleukodystrophy (CALD)
SKYSONA is the first and only gene therapy recommended for approval for patients with CALD, a progressive, neurodegenerative disease
As of the data cutoff date, 90% of patients (27/30) treated with SKYSONA in the pivotal ALD-102 clinical study met the primary endpoint of major functional disability (MFD)-free survival at two years of follow-up
Data from the long-term follow-up study (LTF-304) suggest that SKYSONA continues to show a durable effect on MFD-free survival, with the longest follow-up of nearly seven years (82.7 months)
Among 51 patients treated with SKYSONA across clinical studies to date, there have been no reports of graft-versus-host disease (GVHD), graft failure or rejection, or transplant-related mortality (TRM)
CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 21, 2021-- bluebird bio, Inc. (Nasdaq: BLUE) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending marketing authorization for SKYSONA™ (elivaldogene autotemcel, Lenti-D™), a one-time gene therapy for the treatment of early cerebral adrenoleukodystrophy (CALD) in patients less than 18 years of age with an ABCD1 genetic mutation, and for whom a human leukocyte antigen (HLA)-matched sibling hematopoietic stem cell (HSC) donor is not available. If approved by the European Commission (EC), SKYSONA will be the first one-time gene therapy approved to treat CALD, a rare neurodegenerative disease that occurs in childhood and can lead to progressive, irreversible loss of neurological function and death.
The CHMP’s positive opinion will now be reviewed by the EC, which has the authority to grant marketing authorization for SKYSONA in the European Union (EU). A CHMP positive opinion is one of the final steps before the EC decides whether to authorize a new medicine. A final decision by the EC for SKYSONA is anticipated in mid-2021. SKYSONA is not approved for any indication in any geography.
Data Supporting Clinical Profile of SKYSONA
The positive CHMP opinion is supported by efficacy and safety data from the Phase 2/3 Starbeam study (ALD-102). All patients who completed ALD-102, plus those who will complete a second Phase 3 study (ALD-104), will be asked to participate in a long-term follow-up study (LTF-304).
The primary efficacy endpoint of the pivotal ALD-102 study was the proportion of patients who did not have any of the six MFDs, were alive, did not receive a second allo-HSCT or rescue cell administration and had not withdrawn or been lost to follow-up at Month 24. To date, 32 patients have been treated with SKYSONA in ALD-102, and 30/32 patients were evaluable for follow-up at Month 24. As of the last data cutoff date, 90% (27/30) of the patients met the Month 24 MFD-free survival endpoint. In addition, as previously reported, two patients withdrew from the study at investigator discretion, and one experienced rapid disease progression early on in the study, resulting in MFDs and subsequent death.
In ALD-102, 26/28 evaluable patients maintained a neurologic function score (NFS) less than or equal to 1 through Month 24, and 24 of those patients had no change in their NFS, which showed maintenance of neurological function in the majority of patients. All patients who completed ALD-102 enrolled for long-term follow-up in the LTF-304 study. The median duration of follow-up was 38.59 months (min.: 13.4; max.: 82.7).
The treatment regimen, comprising mobilization/apheresis, conditioning and SKYSONA infusion, had a safety/tolerability profile primarily reflective of the known effects of mobilization/apheresis and conditioning.
Adverse reactions attributed to SKYSONA observed in clinical trials include cystitis viral, pancytopenia and vomiting.
There have been no reports of GVHD, graft failure or rejection, TRM or replication competent lentivirus in the 51 patients treated with SKYSONA in clinical studies (ALD-102/LTF-304 and ALD-104). Additionally, there have been no reports of lentiviral vector-mediated insertional mutagenesis resulting in oncogenesis, including myelodysplasia, leukemia or lymphoma, associated with SKYSONA. Nevertheless, there is a theoretical risk of malignancy after treatment with SKYSONA. Clonal expansion resulting in clonal predominance without clinical evidence of malignancy has been detected in some patients treated with SKYSONA.
SKYSONA continues to be evaluated in the Phase 3 ALD-104 study and the long-term follow-up study LTF-304. If approved by the EC, patients treated with SKYSONA in Europe will be expected to enroll in the REG-502 Stargazer registry.
About SKYSONA (elivaldogene autotemcel, formerly Lenti-D™ gene therapy)
The U.S. Food and Drug Administration (FDA) granted SKYSONA Orphan Drug status, Rare Pediatric Disease designation and Breakthrough Therapy designation for the treatment of CALD. bluebird bio is currently on track to submit the Biologics License Application (BLA) in the U.S. by mid-2021.
SKYSONA is not approved for any indication in any geography.
The Phase 3 ALD-104 study, designed to assess the efficacy and safety of SKYSONA after myeloablative conditioning using busulfan and fludarabine in patients with CALD, is approaching enrollment completion; enrollment in Europe is expected to be closed at the end of May.
The Phase 2/3 Starbeam study (ALD-102) is complete. For more information about our studies, visit: www.bluebirdbio.com/our-science/clinical-trials or clinicaltrials.gov.
Additionally, bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-304) for patients who have been treated with SKYSONA for CALD and completed two years of follow-up in bluebird bio-sponsored studies. If approved by the EC, patients treated with SKYSONA in Europe will be expected to enroll in the REG-502 Stargazer registry.
For more information, visit bluebirdbio.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210521005242/en/
Source: bluebird bio, Inc.
EMA advisory group backs bluebird bio's Skysona in rare neurodegenerative disease
May 21, 2021 8:14 AM ETbluebird bio, Inc. (BLUE)bluebird bio, Inc. (BLUE)By: Mamta Mayani, SA News Editor7 Comments
- bluebird bio (NASDAQ:BLUE) announces that the Committee for Medicinal Products for Human Use (CHMP) of the EMA has adopted positive opinion recommending marketing authorization for Skysona (elivaldogene autotemcel, Lenti-D™), a one-time gene therapy for the treatment of early cerebral adrenoleukodystrophy (CALD) in patients less than 18 years of age.
- https://seekingalpha.com/news/3699059-ema-advisory-group-backs-bluebird-bios-skysona-in-rare-neurodegenerative-disease
- https://seekingalpha.com/symbol/BLUE
- https://www.bluebirdbio.com/our-science/pipeline
DARZALEX® (daratumumab) Subcutaneous Formulation Utilizing ENHANZE®
Halozyme Announces Janssen Receives Two Positive CHMP Opinions for DARZALEX® (daratumumab) Subcutaneous Formulation Utilizing ENHANZE®
Share This
05/21/2021- For Use of Subcutaneous Daratumumab in Combination with Cyclophosphamide, Bortezomib and Dexamethasone (D-VCd) in Newly Diagnosed Adult Patients with Systemic Light Chain (AL) Amyloidosis -
- For Use of Subcutaneous Daratumumab in Combination with Pomalidomide and Dexamethasone (D-Pd) in Adult Patients with Relapsed or Refractory Multiple Myeloma -
SAN DIEGO, May 21, 2021 /PRNewswire/ -- Halozyme Therapeutics, Inc. (NASDAQ: HALO) today announced that Janssen Pharmaceutica N.V. (Janssen) received two positive opinions from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommending to broaden the existing marketing authorization for the DARZALEX® (daratumumab) subcutaneous (SC) formulation, which uses ENHANZE® technology, in two new indications. One recommendation is for the use in combination with cyclophosphamide, bortezomib and dexamethasone (D-VCd), for the treatment of adult patients with newly diagnosed systemic light chain (AL) amyloidosis. The second recommendation is for the use of daratumumab SC in combination with pomalidomide and dexamethasone (D-Pd) for the treatment of adult patients with multiple myeloma who have received one prior therapy containing a proteasome inhibitor and lenalidomide and were lenalidomide-refractory, or who have received at least two prior therapies that included lenalidomide and a proteasome inhibitor and have demonstrated disease progression on or after the last therapy. These two positive opinions will next be reviewed by the European Commission (EC), which has the authority to grant final approval of the indications.
"Janssen's obtaining positive CHMP opinions is an important step forward in the EU and we are delighted subcutaneous DARZALEX® may soon be available in these important new indications," said Helen Torley, president and chief executive officer at Halozyme. "This would be the first approval in Europe for the treatment of AL amyloidosis and also introduces a new treatment option for certain patients with multiple myeloma."
DARZALEX® (daratumumab) Subcutaneous Formulation Utilizing ENHANZE®
"Janssen's obtaining positive CHMP opinions is an important step forward in the EU and we are delighted subcutaneous DARZALEX® may soon be available in these important new indications," said Helen Torley, president and chief executive officer at Halozyme. "This would be the first approval in Europe for the treatment of AL amyloidosis and also introduces a new treatment option for certain patients with multiple myeloma."
The Positive CHMP Opinion for the AL amyloidosis indication is supported by data from Janssen's Phase 3 ANDROMEDA study.1 Janssen reported that the study met the primary endpoint of percentage of patients with hematologic complete response.
The Positive CHMP Opinion for daratumumab SC in combination with Pd in the treatment of relapsed or refractory multiple myeloma is supported by data from the Phase 3 APOLLO study conducted in collaboration with the European Myeloma Network. 2 Janssen reported that the study met its primary endpoint of improved progression-free survival (PFS).
For more information related Phase 3 ANDROMEDA and APOLLO study findings, please view Janssen's press release
JNJ wins positive opinion in Europe for expanded use of DARZALEX
May 21, 2021 11:02 AM ET Johnson & Johnson (JNJ), HALOJohnson & Johnson (JNJ)Halozyme Therapeutics, Inc. (HALO)
By: Dulan Lokuwithana, SA News Editor
- Janssen Pharmaceutica, a unit of Johnson & Johnson (JNJ +0.6%) has received two positive opinions from the European Medicines Agency (EMA) to broaden the current marketing authorization for DARZALEX (daratumumab) subcutaneous formulation (SC).
- https://seekingalpha.com/news/3699162-jnj-wins-positive-opinion-in-europe-for-expanded-use-of-darzalex
- https://seekingalpha.com/symbol/JNJ
- Daratumumab subcutaneous SC uses ENHANZE technology developed by Halozyme Therapeutics (HALO -1.6%).
- https://seekingalpha.com/symbol/HALO
- https://www.halozyme.com/enhanze/overview/default.aspx
- https://www.halozyme.com/enhanze/pipeline/default.aspx
KEYTRUDA® (pembrolizumab) in Combination With Chemotherapy
Merck Receives Positive EU CHMP Opinion for KEYTRUDA® (pembrolizumab) in Combination With Chemotherapy as First-Line Treatment for Certain Patients With Esophageal Cancer or HER2-Negative Gastroesophageal Junction (GEJ) Adenocarcinoma
May 24, 2021 6:45 am ET
Opinion Supports Use of KEYTRUDA in Combination With Platinum- and Fluoropyrimidine-Based Chemotherapy in Patients Whose Tumors Express PD-L1 (CPS ≥10)
Recommendation Based on Significant Survival Benefit Demonstrated With KEYTRUDA Plus Chemotherapy Versus Chemotherapy in Phase 3 KEYNOTE-590 Trial
KENILWORTH, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending approval of KEYTRUDA, Merck’s anti-PD-1 therapy, in combination with platinum- and fluoropyrimidine-based chemotherapy for the first-line treatment of patients with locally advanced unresectable or metastatic carcinoma of the esophagus or human epidermal growth factor receptor 2 (HER2)-negative gastroesophageal junction (GEJ) adenocarcinoma in adults whose tumors express PD-L1 (Combined Positive Score [CPS] ≥10). The CHMP’s recommendation will now be reviewed by the European Commission for marketing authorization in the European Union, and a final decision is expected in the second quarter of 2021.
“Patients with metastatic esophageal cancer currently face five-year survival rates of just 5%,” said Dr. Scot Ebbinghaus, vice president, clinical research, Merck Research Laboratories. “There is a critical need for new treatment options in the first-line setting that can potentially extend their lives. Today’s positive opinion for KEYTRUDA is an important step forward for patients in Europe with certain types of gastrointestinal cancers.”
About KEYTRUDA® (pembrolizumab) Injection, 100 mg
KEYTRUDA is an anti-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 1,400 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.
Selected KEYTRUDA® (pembrolizumab) Indications in the U.S.
Melanoma
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.
KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.
Non-Small Cell Lung Cancer
KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) ≥1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is stage III where patients are not candidates for surgical resection or definitive chemoradiation, or metastatic.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS ≥1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.
Head and Neck Squamous Cell Cancer
KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS) ≥1] as determined by an FDA-approved test.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.
Classical Hodgkin Lymphoma
KEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).
KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.
Primary Mediastinal Large B-Cell Lymphoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.
Urothelial Carcinoma
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 (CPS ≥10), as determined by an FDA-approved test, or in patients who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.
Microsatellite Instability-High or Mismatch Repair Deficient Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR)
- solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options, or
- colorectal cancer that has progressed following treatment with fluoropyrimidine, oxaliplatin, and irinotecan.
This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.
Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer
KEYTRUDA is indicated for the first-line treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).
Gastric Carcinoma
KEYTRUDA, in combination with trastuzumab, fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of patients with locally advanced unresectable or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent locally advanced or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test, with disease progression on or after 2 or more prior lines of therapy including fluoropyrimidine- and platinum-containing chemotherapy and if appropriate, HER2/neu-targeted therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Esophageal Carcinoma
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic esophageal or gastroesophageal junction (GEJ) (tumors with epicenter 1 to 5 centimeters above the GEJ) carcinoma that is not amenable to surgical resection or definitive chemoradiation either:
- in combination with platinum- and fluoropyrimidine-based chemotherapy, or
- as a single agent after one or more prior lines of systemic therapy for patients with tumors of squamous cell histology that express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
Cervical Carcinoma
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Hepatocellular Carcinoma
KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Merkel Cell Carcinoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Renal Cell Carcinoma
KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).
Tumor Mutational Burden-High
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [≥10 mutations/megabase] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.
Cutaneous Squamous Cell Carcinoma
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) that is not curable by surgery or radiation.
Triple-Negative Breast Cancer
KEYTRUDA, in combination with chemotherapy, is indicated for the treatment of patients with locally recurrent unresectable or metastatic triple-negative breast cancer (TNBC) whose tumors express PD-L1 (CPS ≥10) as determined by an FDA-approved test. This indication is approved under accelerated approval based on progression-free survival. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Please see Prescribing Information for KEYTRUDA (pembrolizumab) at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_pi.pdf and Medication Guide for KEYTRUDA at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_mg.pdf.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210524005053/en/
Source: Merck & Co., Inc.
Merck's Keytruda + chemotherapy wins positive CHMP opinion for esophageal and gastric cancer
May 24, 2021 7:01 AM ET Merck & Co., Inc. (MRK)Merck & Co., Inc. (MRK)
By: Mamta Mayani, SA News Editor1 Comment
- The EMA's Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending approval of Merck's (NYSE:MRK) Keytruda in combination with platinum- and fluoropyrimidine-based chemotherapy for the first-line treatment of patients with locally advanced unresectable or metastatic carcinoma of the esophagus or human epidermal growth factor receptor 2 (HER2)-negative gastroesophageal junction (GEJ) adenocarcinoma in adults whose tumors express PD-L1 (Combined Positive Score [CPS] ≥10).
- https://seekingalpha.com/news/3699350-mercks-keytruda-chemotherapy-wins-positive-chmp-opinion-for-esophageal-and-gastric-cancer
- https://seekingalpha.com/symbol/MRK
- https://www.keytruda.com/
RYEQO® (Relugolix Combination Tablet)
Myovant Sciences Receives Positive CHMP Opinion for RYEQO® (Relugolix Combination Tablet) for the Treatment of Women With Uterine Fibroids
May 21, 2021 at 8:30 AM EDTPDF Version
- CHMP opinion recommending approval based on data from the Phase 3 LIBERTY program in women with uterine fibroids
- Gedeon Richter will commercialize RYEQO for uterine fibroids, if approved, in Europe
- Relugolix combination tablet for uterine fibroids is also under U.S. FDA review with a target action date of June 1, 2021
BASEL, Switzerland, May 21, 2021 (GLOBE NEWSWIRE) -- Myovant Sciences (NYSE: MYOV) today announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion recommending the approval of RYEQO® (relugolix 40 mg, estradiol 1.0 mg, and norethindrone acetate 0.5 mg) for the treatment of moderate to severe symptoms of uterine fibroids in adult women of reproductive age. The European Commission will review the CHMP recommendation, and a final decision on the Marketing Authorization Application is expected to be available in approximately two months. The decision will be applicable to all 27 European Union member states plus Iceland, Norway, and Liechtenstein.
“Over 25% of women of reproductive age develop uterine fibroids. This chronic disease can cause debilitating symptoms that have a significant impact on quality of life and require long-term treatment, yet there are currently limited treatment options in Europe and many women are faced with the decision to undergo surgery to alleviate symptoms,” said Roberta Venturella, M.D., Ph.D., Associate Professor, Magna Græcia University of Catanzaro and investigator in the LIBERTY program. “The CHMP’s positive opinion further validates RYEQO’s potential to effectively address heavy menstrual bleeding and pain associated with uterine fibroids and serve as an important new treatment option for patients and physicians.”
“This positive CHMP opinion represents an important step in advancing our mission to redefine care for women living with uterine fibroids,” said David Marek, Chief Executive Officer of Myovant Sciences, Inc. “We look forward to Gedeon Richter’s launch of RYEQO, if approved, as a new treatment option for uterine fibroids.”
Relugolix combination tablet for the treatment of uterine fibroids is also under review by the U.S. Food and Drug Administration, with a target action date of June 1, 2021.
About RYEQO®
RYEQO (relugolix 40 mg, estradiol 1.0 mg, and norethindrone acetate 0.5 mg) is being evaluated for the treatment of moderate to severe symptoms of uterine fibroids in adult women of reproductive age. RYEQO 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.
RYEQO is not approved for any indication in any geography.
For more information, please visit our website at www.myovant.com.
Myovant Sciences gets positive CHMP opinion for RYEQO uterine fibroids treatment
May 21, 2021 8:44 AM ETMyovant Sciences Ltd. (MYOV)Myovant Sciences Ltd. (MYOV)By: Aakash Babu, SA News Editor1 Comment
- Myovant Sciences (NYSE:MYOV) announces that the Committee for Medicinal Products for Human Use (CHMP) of the EMA has adopted a positive opinion for approval of RYEQO (relugolix 40 mg, estradiol 1.0 mg, and norethindrone acetate 0.5 mg) for the treatment of moderate to severe symptoms of uterine fibroids.
- https://seekingalpha.com/news/3699084-myovant-sciences-gets-positive-chmp-opinion-for-ryeqo-uterine-fibroids-treatment
- https://seekingalpha.com/symbol/MYOV
- https://www.orgovyx.com/
What is ORGOVYX?
ORGOVYX is a prescription medicine used in adults for the treatment of advanced prostate cancer.
It is not known if ORGOVYX is safe or effective in females or children.
Please see full Prescribing Information and Patient Product Information for ORGOVYX.
RYBREVANTTM (amivantamab-vmjw)
RYBREVANTTM (amivantamab-vmjw) Receives FDA Approval as the First Targeted Treatment for Patients with Non-Small Cell Lung Cancer with EGFR Exon 20 Insertion MutationsRYBREVANTTM is the first fully-human, bispecific antibody approved in lung cancer
Simultaneous FDA approval of a companion diagnostic aids in the identification of exon 20 insertion mutations
May 21, 2021 (HORSHAM, P.A.) – The Janssen Pharmaceutical Companies of Johnson & Johnson announced today the U.S. Food and Drug Administration (FDA) has granted the accelerated approval of RYBREVANTTM (amivantamab-vmjw) for the treatment of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations, as detected by an FDA-approved test, whose disease has progressed on or after platinum-based chemotherapy.[1] RYBREVANTTM is the first fully-human, bispecific antibody approved for the treatment of patients with NSCLC that targets EGFR exon 20 insertion mutations, which are the third most prevalent activating EGFR mutation.1,[2] Today’s approval follows the FDA’s decision to grant Breakthrough Therapy Designation (BTD) in March 2020 and to initiate a Priority Review of the Biologics License Application (BLA) in December 2020. This indication is approved under accelerated approval based on overall response rate and duration of response.1 Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.1
RYBREVANTTM is a fully-human bispecific antibody directed against EGFR and MET receptors.1 RYBREVANTTM binds extracellularly (outside of the cell) inhibiting tumor growth and leading to tumor cell death.1 Today’s accelerated FDA approval is based on positive results from the Phase 1 CHRYSALIS study, a multicenter, open-label, clinical study evaluating RYBREVANTTM as a monotherapy in patients enrolled in the prior platinum containing chemotherapy cohort.1,[10] Initial results from the CHRYSALIS EGFR exon 20 insertion mutation population, which supported the BTD, were presented at the American Society of Clinical Oncology 2020 Virtual Scientific Program, and updated results were presented at the IASLC WCLC 2020.
“The approval of RYBREVANT, along with the companion diagnostic test, addresses high unmet need in the treatment of people with genetically defined non-small cell lung cancer,” said Peter Lebowitz, M.D., Ph.D., Global Therapeutic Area Head, Oncology, Janssen Research & Development, LLC. “At Janssen, we are committed to the development of innovative therapies like RYBREVANT and believe that advancing medicines targeting specific pathways can bring the greatest benefits and improve outcomes for patients with tumor alterations such as EGFR and MET.”
The FDA simultaneously approved Guardant Health’s Guardant360® CDx liquid biopsy blood test as a companion diagnostic for use with RYBREVANTTM.
Next-generation sequencing tests offer an alternative to polymerase chain reaction (PCR)-based tests which fail to identify 50 percent or more of exon 20 insertion mutations.[11] Information on FDA-approved tests for the detection of EGFR genetic alterations in NSCLC is available at: http://www.fda.gov/CompanionDiagnostics.
“Today’s milestone reflects progress and determination in our mission to develop and deliver transformational therapies to improve the lives of people diagnosed with some of the most devastating and complex diseases of our time,” said Mathai Mammen, M.D., Ph.D., Global Head, Janssen Research & Development, Johnson & Johnson. “The approval of RYBREVANT, the first lung cancer treatment for Johnson & Johnson, strengthens our commitment to harness science, expertise and scale to dramatically alter the trajectory of lung cancer, and reduce the impact of the world’s leading cause of cancer mortality.”
About RYBREVANTTM
RYBREVANTTM is being studied in a comprehensive clinical development program for people with untreated advanced EGFR-mutated NSCLC, including the Phase 3 MARIPOSA (NCT04487080) trial studying RYBREVANTTM in combination with lazertinib**.[12] Another Phase 3 trial, PAPILLON (NCT04538664), is studying RYBREVANTTM in combination with carboplatin-pemetrexed for people with advanced or metastatic EGFR-mutated NSCLC with exon 20 insertion mutations.[13] RYBREVANTTM received U.S. FDA BTD in March 2020 and Priority Review Designation following the BLA announcement in December 2020.[14],[15] Janssen has filed regulatory submissions for RYBREVANTTM with health authorities in Europe and other markets.
**In 2018, Janssen Biotech, Inc. entered into a license and collaboration agreement with Yuhan Corporation for the development of lazertinib.
About the CHRYSALIS Study
CHRYSALIS (NCT02609776) is a Phase 1 open-label, multicenter, first-in-human study to evaluate the safety, pharmacokinetics and preliminary efficacy of RYBREVANTTM as a monotherapy and in combinations including with lazertinib, a novel third-generation EGFR TKI[16], in adults with advanced NSCLC.10 The study consists of two parts: RYBREVANTTM monotherapy and combination-dose escalations and RYBREVANTTM monotherapy and combination-dose expansions.10
Access to RYBREVANTTM (amivantamab-vmjw)
Janssen offers comprehensive access and support information and resources to assist patients in gaining access to RYBREVANTTM. Janssen will work closely with payers and providers to ensure RYBREVANTTM is broadly accessible and affordable for people with NSCLC with EGFR exon 20 insertion mutations. Our comprehensive patient support program, Janssen CarePath, helps patients get started on RYBREVANTTM and stay on track. Janssen CarePath helps verify insurance coverage for patients, provides reimbursement information, helps find financial assistance options for eligible patients and provides ongoing support to help patients start and stay on RYBREVANTTM. If patients have commercial or private health insurance and need help paying for RYBREVANT™, the Janssen CarePath Savings Program may be able to help. For more information, visit Rybrevant.JanssenCarePathSavings.com. If patients don’t have commercial or private health insurance, Janssen CarePath can provide information about other resources that may help with out-of-pocket medication costs. Learn more at JanssenCarePath.com/Rybrevant or call a Janssen CarePath Care Coordinator at 833-RYBREVANT (833-792-7382).
Please read full Prescribing Information for RYBREVANT™.
FDA approves J&J's Rybrevant as targeted therapy for NSCLC patient subset
May 21, 2021 2:37 PM ETJohnson & Johnson (JNJ), GHJohnson & Johnson (JNJ)Guardant Health, Inc. (GH)AstraZeneca PLC (AZN)By: Jonathan M Block, SA News Editor1 Comment
- The FDA has approved Johnson & Johnson's (NYSE:JNJ) Rybrevant (amivantamab-vmjw) as the first-ever treatment for patients with non small-cell lung cancer ("NSCLC") with a specific genetic mutation.
- https://seekingalpha.com/news/3699217-fda-approves-johnson-johnson-rybrevant-as-targeted-therapy-for-nsclc-patient-subset
- https://seekingalpha.com/symbol/JNJ
IMBRUVICA® (ibrutinib)
IMBRUVICA® (ibrutinib)-Based Combination Regimen as a Fixed-Duration, First-Line Treatment for Chronic Lymphocytic Leukemia Demonstrates High Rates of Disease Control
May 19, 2021United States
Janssen presents new data at ASCO showing efficacy of IMBRUVICA® plus venetoclax (CAPTIVATE study) in previously untreated patients with chronic lymphocytic leukemia, and up to seven-year follow-up results (RESONATE-2 study) on progression-free and overall survival benefits with single-agent IMBRUVICA®
May 19, 2021 (RARITAN, N.J.) – The Janssen Pharmaceutical Companies of Johnson & Johnson today announced new data from the fixed-duration cohort of the investigational Phase 2 CAPTIVATE study, showing that 95 percent of patients treated with combined IMBRUVICA® plus venetoclax were alive and progression-free at two years.[1] Deep remissions were seen across all subgroups, including patients with high-risk chronic lymphocytic leukemia (CLL).1 In addition, long-term data from the RESONATE-2 (PCYC-1115/1116) study will be presented, providing the longest follow-up Phase 3 data for any BTK inhibitor to date. These data reinforce the long-term survival benefits and well-established safety profile of single-agent IMBRUVICA® for patients with CLL.[2] The data will be presented during the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting, June 4-8.
First Data from the Fixed-Duration Cohort of the Phase 2 CAPTIVATE (PCYC-1142) Study of IMBRUVICA®-Based Combination Regimen in Previously Untreated CLL Patients (Abstract #7501)1
The CAPTIVATE study evaluated previously untreated CLL/small lymphocytic lymphoma (SLL) patients 70 years or younger, including patients with high-risk disease.1 In the fixed-duration cohort (N=159; median age, 60 years), all patients received three months of IMBRUVICA® lead-in therapy followed by 12 months of combination IMBRUVICA® plus venetoclax therapy and then stopped therapy regardless of minimal residual disease (MRD) status.1 More than 90 percent of patients completed 12 cycles of IMBRUVICA® plus venetoclax treatment.1
Findings from the uMRD-guided cohort of the CAPTIVATE study were presented at the 2020 American Society of Hematology (ASH) Annual Meeting. The Phase 3 GLOW study (NCT03462719) is also evaluating fixed-duration IMBRUVICA® plus venetoclax, with a comparison to chlorambucil plus obinutuzumab, for first-line treatment of elderly or younger unfit patients with CLL or SLL. These studies are part of a comprehensive development program exploring the potential of IMBRUVICA®-based fixed-duration therapy.
Long-Term Data from the Phase 3 RESONATE-2 Study Demonstrate Efficacy and Safety of Single-Agent IMBRUVICA® in Previously Untreated CLL Patients (Abstract #7523)2
The RESONATE-2 study evaluated 269 previously untreated patients with CLL ages 65 years or older, without del(17p), who were randomly assigned to receive continuous IMBRUVICA® or chlorambucil up to 12 cycles.2 With up to seven years of follow-up, PFS benefit with single-agent IMBRUVICA® was sustained (PFS Hazard Ratio [HR] 0.160 [95 percent CI, 0.111–0.230]).2 At 6.5 years, the median PFS with IMBRUVICA® has not been reached; 61 percent of patients treated with single-agent IMBRUVICA® were alive and progression-free compared with nine percent of patients treated with chlorambucil.2 The PFS benefit for patients treated with IMBRUVICA® was seen in all subgroups, including those with high-risk genomic features of TP53 mutation, unmutated IGHV or 11q deletion (HR 0.091 [95 percent CI, 0.054–0.152]).2 Additionally, 78 percent of patients in the IMBRUVICA® treatment arm were alive at 6.5 years.2 The CR rate with IMBRUVICA® treatment has increased over time to 34 percent.2 Nearly half of patients continue to receive IMBRUVICA® treatment with up to seven years of follow-up.2
About IMBRUVICA®
IMBRUVICA® (ibrutinib) is a once-daily oral medication that is jointly developed and commercialized by Janssen Biotech, Inc. and Pharmacyclics LLC, an AbbVie company. IMBRUVICA® blocks the Bruton's tyrosine kinase (BTK) protein, which is needed by specific cancer cells to survive.[3],[4] IMBRUVICA® helps to force cancerous B cells out of environments in which they thrive and multiply, like the lymph nodes, and prevents them from returning. This action of IMBRUVICA, along with other effects of blocking BTK, reduces the ability of cancerous B cells to survive.[5]
IMBRUVICA® is approved in more than 100 countries, and, to date, has been used to treat more than 230,000 patients worldwide. IMBRUVICA® is the only BTKi that has demonstrated overall survival benefits in three CLL clinical trials, with response durability persisting up to eight years,[6],[7],[8] and seven out of 10 patients alive and without disease progression after five years.7 IMBRUVICA® is the only BTKi that has been shown to mediate short- and long-term immune restoration.[9]
IMBRUVICA® was first approved by the U.S. Food and Drug Administration (FDA) in November 2013, and today is indicated in six disease areas, including five hematologic cancers – chronic lymphocytic leukemia (CLL) /small lymphocytic lymphoma (SLL) with or without 17p deletion (del17p), Waldenström’s macroglobulinemia (WM), patients with previously treated mantle cell lymphoma (MCL)*, patients with previously treated marginal zone lymphoma (MZL) who require systemic therapy and have received at least one prior anti-CD20-based therapy* – and patients with previously treated chronic graft-versus-host disease (cGVHD) after failure of one or more lines of systemic therapy.[10]
*Accelerated approval was granted for MCL and MZL based on overall response rate. Continued approval for MCL and MZL may be contingent upon verification and description of clinical benefit in confirmatory trials.
IMBRUVICA® is the most comprehensively studied BTKi, with more than 150 active clinical trials in several blood cancers and other serious diseases. For more information, visit www.IMBRUVICA.com.
Learn more at www.janssen.com. Follow us at @JanssenUS and @JanssenGlobal. Janssen Research & Development, LLC and Janssen Biotech, Inc. are part of the Janssen Pharmaceutical Companies of Johnson & Johnson.
JNJ highlights potential of IMBRUVICA as a first-line treatment for leukemia
May 20, 2021 1:51 PM ETJohnson & Johnson (JNJ), ABBVJohnson & Johnson (JNJ)AbbVie Inc. (ABBV)By: Dulan Lokuwithana, SA News Editor
- Johnson & Johnson (JNJ +0.7%) updated results from a fixed-duration cohort of the Phase 2 CAPTIVATE study for IMBRUVICA-based combination regimen in patients with chronic lymphocytic leukemia (CLL).
- Jointly developed and commercialized by Janssen Biotech of JNJ and AbbVie (ABBV +1.0%) IMBRUVICA is a blocker of Bruton's tyrosine kinase (BTK) proteins which are believed to be important for the survival of certain cancer cells.
- https://seekingalpha.com/news/3698811-jnj-highlights-potential-of-imbruvica-as-a-first-line-treatment-for-leukemia
- https://seekingalpha.com/symbol/JNJ
- https://seekingalpha.com/symbol/ABBV
Maribavir
U.S. Food & Drug Administration Grants Priority Review of Maribavir for the Treatment of Post-Transplant Recipients With Cytomegalovirus Infection in Those Resistant and/or Refractory to Prior Anti-CMV Treatment
May 21, 2021
− If Approved, Maribavir Will Be the First and Only Treatment Indicated for Post-Transplant Cytomegalovirus (CMV) Infection in Those That Are Refractory, With or Without Resistance (R/R)
− NDA based on Phase 3 Trial of Maribavir Which Met Its Primary Endpoint of Superiority Compared to Conventional Antiviral Therapies in Transplant Recipients with R/R CMV Infection1
− The U.S. Food & Drug Administration Granted Maribavir Breakthrough Therapy Designation as a Treatment for CMV Infection in Transplant Patients Resistant or Refractory to Prior Therapy
− Maribavir is Takeda’s Fourth New Molecular Entity Accepted for Regulatory Review in Six Months
OSAKA, Japan, May 21, 2021 – Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) (“Takeda”) today announced that the U.S. Food & Drug Administration (FDA) has accepted a New Drug Application (NDA) for maribavir for the treatment of CMV infection in those that are refractory with or without resistance (R/R), in solid organ transplant (SOT) or hematopoietic cell transplant (HCT) recipients.
This is an inflection year for Takeda’s pipeline with up to six regulatory submissions and four potential approvals anticipated by the end of fiscal year 2021. The maribavir NDA acceptance is Takeda’s fourth new molecular entity accepted for regulatory review in six months, following the FDA submissions of TAK-721 for the treatment of eosinophilic esophagitis, mobocertinib for the treatment of EGFR Exon20 insertion mutation positive metastatic non-small cell lung cancer, and the European Medicines Agency submission of the Company’s dengue vaccine candidate (TAK-003), which is being investigated for the prevention of dengue due to any dengue virus serotype in individuals ages four to 60.
About Maribavir
Maribavir, an orally bioavailable anti-CMV compound, is the only antiviral agent presently in Phase 3 development for the treatment of post-transplant patients with CMV in SOT or HCT. Maribavir is an investigational treatment that has not been approved for use by the U.S. Food and Drug Administration (FDA), European Medicines Agency (EMA) or any other regulatory authorities. Maribavir is the only CMV antiviral drug that targets and inhibits the UL97 protein kinase and its natural substrates.1,15–17
Maribavir has been granted Orphan Drug Designation by the European Commission as a treatment of CMV disease in patients with impaired cell mediated immunity and by the FDA for treatment of clinically significant CMV viremia and disease in at-risk patients. Orphan status is granted to certain investigational medicines intended for the treatment or prevention of a rare, life-threatening disease. The FDA has also granted maribavir Breakthrough Therapy Designation as a treatment for CMV infection and disease in transplant patients resistant or refractory to prior therapy. Breakthrough Therapy Designation expedites the development and review of investigational treatments for serious conditions with preliminary clinical evidence indicating that the drug may demonstrate substantial improvement over available therapy. These designations do not guarantee that the EMA or FDA will approve maribavir for the treatment of CMV infections in transplant patients, and the timing of any such approval is uncertain.
About Takeda’s SOLSTICE Trial
The TAK-620-303 (SOLSTICE) trial (NCT02931539) is a multicenter, randomized, open-label, active-controlled trial comparing treatment with either maribavir or investigator assigned treatment, IAT, (conventional antiviral therapy) in hematopoietic cell transplant and solid organ transplant recipients with CMV infection refractory, with or without resistance, to one or a combination of the conventional antiviral therapies: ganciclovir, valganciclovir, foscarnet or cidofovir. Patients underwent a 2-week screening period, followed by randomization 2:1 to maribavir (n=235) (400 mg) or IAT (n=117) for an 8-week treatment period, plus 12 weeks of follow-up.
The trial’s primary endpoint was defined as the proportion of patients who achieved confirmed CMV viremia clearance (plasma CMV DNA <137 IU/mL in two consecutive tests ≥5 days apart at central laboratory) compared to IAT at the end of Study Week 8. The key secondary endpoint was defined as achievement of CMV viremia clearance and symptom control at end of Study Week 8, maintained through Study Week 16.
For more information, visit https://www.takeda.com.
FDA accepts Takeda's maribavir application for CMV infection during organ transplant
May 21, 2021 5:07 AM ET
Takeda Pharmaceutical Company Limited (TAK)Takeda Pharmaceutical Company Limited (TAK)
By: Mamta Mayani, SA News Editor2 Comments
- The FDA has accepted Takeda Pharmaceutical's (NYSE:TAK) New Drug Application for maribavir for the treatment of CMV (cytomegalovirus) infection in solid organ transplant or hematopoietic cell transplant recipients.
- https://seekingalpha.com/news/3698999-fda-accepts-takedas-maribavir-application-for-cmv-infection-during-organ-transplant
- https://seekingalpha.com/symbol/TAK
- https://www.takeda.com/what-we-do/t-cira/portfolios/
Tislelizumab
BeiGene Announces Positive Topline Results from Phase 3 Trial of Tislelizumab in Combination with Chemotherapy as First-Line Treatment for Recurrent or Metastatic Nasopharyngeal Cancer
May 21, 2021 at 7:00 AM EDT
Tislelizumab combined with chemotherapy demonstrated a statistically significant improvement in progression-free survival at the interim analysis
Safety findings of tislelizumab were consistent with known risks
CAMBRIDGE, Mass. & BEIJING--(BUSINESS WIRE)--May 21, 2021-- BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a global biotechnology company focused on developing and commercializing innovative medicines worldwide, today announced that the Phase 3 RATIONALE 309 trial of its anti-PD-1 antibody tislelizumab combined with chemotherapy versus placebo combined with chemotherapy as a first-line treatment for patients with recurrent or metastatic nasopharyngeal cancer (NPC) met its primary endpoint of progression-free survival (PFS) at the interim analysis, as recommended by an independent data monitoring committee. In the trial results, tislelizumab in combination with chemotherapy demonstrated a statistically significant improvement in PFS in the intention-to-treat (ITT) population when compared to chemotherapy alone, as assessed by an independent review committee (IRC). The safety profile of tislelizumab was consistent with its known risks, with no new safety signals identified with the addition of chemotherapy.
RATIONALE 309 Trial of Tislelizumab with Chemotherapy Versus Placebo with Chemotherapy in Recurrent or Metastatic NPC
RATIONALE 309 is a randomized, double-blind, placebo-controlled Phase 3 clinical trial (NCT03924986) designed to evaluate the efficacy and safety of tislelizumab combined with gemcitabine and cisplatin versus placebo combined with gemcitabine and cisplatin as a first-line treatment for patients with recurrent or metastatic NPC. The trial’s primary endpoint is PFS as assessed by IRC in the ITT population. Key secondary endpoints include overall survival (OS), IRC-assessed objective response rate (ORR) and duration of response (DoR), and investigator-assessed PFS. A total of 263 Asian patients were enrolled and randomized 1:1 to either the tislelizumab plus chemotherapy arm or the placebo plus chemotherapy arm.
About Tislelizumab
Tislelizumab (BGB-A317) is a humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to FcγR on macrophages. In pre-clinical studies, binding to FcγR on macrophages has been shown to compromise the anti-tumor activity of PD-1 antibodies through activation of antibody-dependent macrophage-mediated killing of T effector cells. Tislelizumab is the first drug from BeiGene’s immuno-oncology biologics program and is being developed internationally as a monotherapy and in combination with other therapies for the treatment of a broad array of both solid tumor and hematologic cancers.
The China National Medical Products Administration (NMPA) has granted tislelizumab market authorization in four indications, including full approval for first-line treatment of patients with advanced squamous non-small cell lung cancer (NSCLC) in combination with chemotherapy; and conditional approval for the treatment of patients with classical Hodgkin’s lymphoma (cHL) who received at least two prior therapies and for the treatment of patients with locally advanced or metastatic urothelial carcinoma (UC) with PD-L1 high expression whose disease progressed during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy. Full approval for these indications is contingent upon results from ongoing randomized, controlled confirmatory clinical trials.
In addition, three supplemental Biologics License Applications for tislelizumab have been accepted by the Center for Drug Evaluation (CDE) of the NMPA and are under review for first-line treatment of patients with advanced non-squamous NSCLC in combination with chemotherapy, for second- or third-line treatment of patients with locally advanced or metastatic NSCLC who progressed on prior platinum-based chemotherapy, and for patients with previously treated unresectable hepatocellular carcinoma.
BeiGene has initiated or completed 17 potentially registration-enabling clinical trials in China and globally, including 13 Phase 3 trials and four pivotal Phase 2 trials.
In January 2021, BeiGene and Novartis entered into a collaboration and license agreement granting Novartis rights to develop, manufacture, and commercialize tislelizumab in North America, Europe, and Japan.
Tislelizumab is not approved for use outside of China.
To learn more about BeiGene, please visit www.beigene.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20210521005092/en/
Source: BeiGene, Ltd.
BeiGene's tislelizumab met primary endpoint in late-stage nasopharyngeal cancer study
May 21, 2021 7:06 AM ET
BeiGene, Ltd. (BGNE)BeiGene, Ltd. (BGNE
)By: Mamta Mayani, SA News Editor
- BeiGene's (NASDAQ:BGNE) Phase 3 RATIONALE 309 trial of anti-PD-1 antibody tislelizumab combined with chemotherapy vs. placebo combined with chemotherapy as a first-line treatment for patients with recurrent or metastatic nasopharyngeal cancer met its primary endpoint of progression-free survival (PFS).
- https://seekingalpha.com/news/3699023-beigenes-tislelizumab-met-primary-endpoint-in-late-stage-nasopharyngeal-cancer-study
- https://seekingalpha.com/symbol/BGNE
- https://www.beigene.com/science-and-product-portfolio/pipeline/tislelizumab
- https://www.beigene.com/
biotecMAX™ April/May 2021 Insight
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab)
Six-and-a-Half-Year Outcomes for Opdivo (nivolumab) in Combination with Yervoy (ipilimumab) Continue to Demonstrate Durable Long-Term Survival Benefits in Patients with Advanced Melanoma
05/19/2021
CATEGORY: Corporate/Financial News
Data evaluating Opdivo plus Yervoy represent the longest reported median overall survival from a Phase 3 advanced melanoma trial
49% of patients treated with Opdivo plus Yervoy were alive at 6.5 years and 77% of these patients remained treatment-free
Data to be featured in an oral presentation during the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced new six-and-a-half-year data from CheckMate -067, a randomized, double-blind, Phase 3 clinical trial, demonstrating durable improvement in survival with first-line Opdivo (nivolumab) plus Yervoy (ipilimumab) therapy and Opdivo monotherapy, versus Yervoy alone, in patients with advanced melanoma. With a minimum follow-up of 6.5 years, median overall survival (OS) was 72.1 months with Opdivo plus Yervoy (95% CI: 38.2-NR), the longest reported median OS in a Phase 3 advanced melanoma trial, 36.9 months with Opdivo (95% CI: 28.2-58.7) and 19.9 months with the Yervoy group (95% CI: 16.8-24.6). In addition, the Opdivo plus Yervoy combination demonstrated a 6.5-year progression-free survival (PFS) rate of 34% (median of 11.5 months) while PFS rates were 29% (median of 6.9 months) and 7% (median of 2.9 months) for Opdivo alone and Yervoy alone, respectively. Of the 49% of patients alive and in follow-up, 77% of patients who received the combination (112/145), 69% of Opdivo-treated patients (84/122) and 43% (27/63) of Yervoy-treated patients have been off treatment and never received subsequent systemic therapy.
Durable, sustained clinical benefit was also observed with Opdivo plus Yervoy or Opdivo alone across relevant subgroups, including in patients with BRAF mutation, wild-type tumors, and baseline liver metastases. Among patients with BRAF-mutant tumors, the rate of OS at 6.5 years was 57% in patients who received Opdivo plus Yervoy, 43% for Opdivo alone, and 25% for Yervoy alone. In patients with BRAF wild-type tumors, the rate of OS was 46% in patients who received Opdivo plus Yervoy, 42% for Opdivo alone and 22% for Yervoy alone. The rate of OS among patients with liver metastases was 38% for those who received Opdivo plus Yervoy, 31% for Opdivo alone, and 22% for Yervoy alone. Median duration of response (DoR) was not reached for those who received Opdivo plus Yervoy nor Opdivo,while the DoR for Yervoy-treated patientswas 19.2 months.
About CheckMate -067
CheckMate -067 is a Phase 3, double-blind, randomized trial that evaluated the combination of Opdivo plus Yervoy or Opdivo monotherapy versus Yervoy monotherapy in 945 patients with previously untreated advanced melanoma. Patients in the combination group (n=314) received Opdivo 1 mg/kg plus Yervoy 3 mg/kg every three weeks (Q3W) for four doses followed by Opdivo 3 mg/kg every two weeks (Q2W). Patients in the Opdivo monotherapy group (n=316) received Opdivo 3 mg/kg Q2W plus placebo. Patients in the Yervoy monotherapy group (n=315) received Yervoy 3 mg/kg Q3W for four doses plus placebo. Patients were treated until progression or unacceptable toxic effects. Overall survival (OS) and progression-free survival (PFS) were dual endpoints of the trial. Secondary endpoints included objective response rates (ORR), descriptive efficacy assessments and safety.
About Opdivo
Opdivo is a programmed death-1 (PD-1) immune checkpoint inhibitor that is designed to uniquely harness the body’s own immune system to help restore anti-tumor immune response. By harnessing the body’s own immune system to fight cancer, Opdivo has become an important treatment option across multiple cancers.
Opdivo’s leading global development program is based on Bristol Myers Squibb’s scientific expertise in the field of Immuno-Oncology and includes a broad range of clinical trials across all phases, including Phase 3, in a variety of tumor types. To date, the Opdivo clinical development program has treated more than 35,000 patients. The Opdivo trials have contributed to gaining a deeper understanding of the potential role of biomarkers in patient care, particularly regarding how patients may benefit from Opdivo across the continuum of PD-L1 expression.
In July 2014, Opdivo was the first PD-1 immune checkpoint inhibitor to receive regulatory approval anywhere in the world. Opdivo is currently approved in more than 65 countries, including the United States, the European Union, Japan and China. In October 2015, the Company’s Opdivo and Yervoy combination regimen was the first Immuno-Oncology combination to receive regulatory approval for the treatment of metastatic melanoma and is currently approved in more than 50 countries, including the United States and the European Union.
INDICATIONS
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express PD-L1 (≥1%) as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab) and 2 cycles of platinum-doublet chemotherapy, is indicated for the first-line treatment of adult patients with metastatic or recurrent non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab) is indicated for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving OPDIVO.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with unresectable malignant pleural mesothelioma (MPM).
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of patients with intermediate or poor risk advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab), in combination with cabozantinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab) is indicated for the treatment of patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy.
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with classical Hodgkin lymphoma (cHL) that has relapsed or progressed after autologous hematopoietic stem cell transplantation (HSCT) and brentuximab vedotin or after 3 or more lines of systemic therapy that includes autologous HSCT. This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab) is indicated for the treatment of patients with recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN) with disease progression on or after platinum-based therapy.
OPDIVO® (nivolumab) is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of adult and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab) is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection.
OPDIVO® (nivolumab) is indicated for the treatment of patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) after prior fluoropyrimidine- and platinum-based chemotherapy.
OPDIVO® (nivolumab), in combination with fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the treatment of patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma.
Please see US Full Prescribing Information for OPDIVO and YERVOY.
For more information about Bristol Myers Squibb, visit us at BMS.com
How do OPDIVO® (nivolumab) and YERVOY® (ipilimumab) team up to fight cancer differently than chemotherapy?
OPDIVO + YERVOY works by helping your immune system to fight cancer. It does this by combining 2 immunotherapy medications that work in different but complementary ways.
Together, OPDIVO and YERVOY can help your immune system launch a response against cancer that's greater than when either medication is used alone.
https://www.opdivo.com/about-opdivo/how-the-combination-works-combinationtherapy
https://seekingalpha.com/symbol/BMY
PNEU-DIRECTION (V114-027) and PNEU-PLAN (V114-024)
Merck Announces Positive Topline Results from PNEU-DIRECTION (V114-027) and PNEU-PLAN (V114-024) Phase 3 Pediatric Studies for V114, Merck’s Investigational 15-valent Pneumococcal Conjugate Vaccine
May 20, 2021 6:45 am ET
V114 Met Primary Immunogenicity and Safety Endpoints in Both Trials
KENILWORTH, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced V114, the company’s investigational 15-valent pneumococcal conjugate vaccine, met its primary immunogenicity and safety endpoints in two trials of the V114 Phase 3 pediatric clinical program. These data support the potential use of V114 in healthy infants who may have previously started a pneumococcal vaccination series with the currently available 13-valent pneumococcal conjugate vaccine (PCV13) (PNEU-DIRECTION), and in a catch-up setting for healthy children who were either pneumococcal vaccine-naïve or who previously received a full or partial regimen with lower valency pediatric pneumococcal conjugate vaccines (PCV) (PNEU-PLAN).
In the PNEU-DIRECTION (V114-027) interchangeability study in healthy infants 42-90 days of age, immune responses in those who received a four-dose series of PCV13, and those who received a mixed dose schedule of PCV13 followed by V114, were generally comparable for the 13 serotypes, or strains of pneumococcal disease, targeted by both vaccines. In the PNEU-PLAN (V114-024) catch-up study, immune responses were generally comparable to PCV13 for the 13 shared serotypes when V114 was used as a catch-up regimen in healthy children 7 months to 17 years of age who were either pneumococcal vaccine-naïve or who previously received a partial or full regimen of a licensed pediatric PCV. For serotypes 22F and 33F, two serotypes included in V114 but not PCV13, immunogenicity in PNEU-PLAN was higher in the V114 group than in the PCV13 group. In each study, V114 was generally well-tolerated, with a safety profile comparable to PCV13.
About PNEU-DIRECTION
PNEU-DIRECTION is a Phase 3, multi-center, randomized, double-blind study evaluating the interchangeability of V114 and PCV13 with respect to safety, tolerability, and immunogenicity in healthy infants 42-90 days of age (n=900).
Participants were randomized to one of five vaccination groups receiving a complete 4-dose schedule with PCV13, one of three mixed 4-dose schedules with PCV13 followed by V114, or a complete 4-dose schedule with V114. Immune responses for the 13 shared serotypes contained in V114 and PCV13 were measured at 30 days post-dose four (PD4) and compared between the groups receiving mixed PCV13/V114 schedules and the group receiving a complete PCV13 schedule. Immune responses were assessed based on anti-pneumococcal polysaccharide (PnPs) serotype-specific immunoglobulin G (IgG) geometric mean concentrations (GMCs). Results of the primary immunogenicity analyses demonstrated that the IgG GMCs for the 13 shared serotypes at 30 days PD4 were generally comparable between participants administered mixed dosing schedules with PCV13/V114 and participants administered a complete dosing schedule with PCV13.
Safety analyses were performed on the All-Participants-As-Treated (APaT) population, defined as all randomized participants who received at least one dose of study vaccination, and showed comparable safety profiles across the five vaccination groups.
About PNEU-PLAN
PNEU-PLAN is a Phase 3, multicenter, randomized, double-blind, active comparator-controlled, descriptive study to evaluate the safety, tolerability, and immunogenicity of catch-up vaccination regimens of V114 in healthy infants, children, and adolescents 7 months to 17 years of age (n=606).
Participants who were either pneumococcal vaccine-naïve or who previously received a partial or full regimen of licensed pediatric PCV were randomized to receive either V114 or PCV13. Participants received a three, two or one dose regimen of V114 depending on their age cohort at randomization. The three age cohorts were children 7 to 11 months of age (three dose regimen), 12 to 23 months of age (two dose regimen) and 2 to 17 years of age (one dose regimen). Immune responses for the 13 shared serotypes contained in V114 and PCV13 and the two serotypes unique to V114 were measured at 30 days post final vaccination. Immunogenicity comparisons were assessed based on anti-PNPs serotype specific IgG GMCs. Results of the primary immunogenicity analyses demonstrated that the IgG GMCs for the 13 shared serotypes 30 days post final vaccine were generally comparable between groups for each of the three cohorts, and higher in V114 groups for serotypes 22F and 33F, the two serotypes included in V114 but not PCV13.
Results of the safety analyses demonstrated that V114 was generally well-tolerated with a safety profile comparable to PCV13.
About V114
V114 is Merck’s investigational 15-valent pneumococcal conjugate vaccine in Phase 3 development for the prevention of pneumococcal disease in adults and children. V114 consists of pneumococcal polysaccharides from 15 serotypes conjugated to a CRM197 carrier protein and includes serotypes 22F and 33F, which are commonly associated with invasive pneumococcal disease worldwide and are not contained in the pneumococcal conjugate vaccine currently licensed for use in children and adults.
To learn more about Merck’s infectious diseases pipeline, visit www.merck.com.
For more information, visit www.merck.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20210520005299/en/
Merck's V114-027 met primary immunogenicity endpoints in late-stage pneumococcal disease studies
May 20, 2021 7:45 AM ET Merck & Co., Inc. (MRK)
By: Mamta Mayani, SA News Editor
- Merck's (NYSE:MRK) V114, an investigational 15-valent pneumococcal conjugate vaccine, met its primary immunogenicity and safety endpoints in two trials of the V114 Phase 3 pediatric clinical program.
- https://seekingalpha.com/news/3698586-mercks-v114-027-met-primary-immunogenicity-endpoints-in-late-stage-pneumococcal-disease-studies
- https://seekingalpha.com/symbol/MRK
- https://www.merck.com/
Tirzepatide
Lilly's tirzepatide achieves all primary and key secondary study outcomes against insulin glargine in adults with type 2 diabetes and increased cardiovascular risk in SURPASS-4 trial
May 20, 2021Download PDF
Highest dose of tirzepatide reduced A1C by 2.58 percent and body weight by 11.7 kg (25.8 lb., 13.0 percent)
SURPASS program has now met regulatory submission requirements for evaluating cardiovascular risk; Lilly intends to submit registration package to regulatory authorities by the end of 2021
INDIANAPOLIS, May 20, 2021 /PRNewswire/ -- Tirzepatide led to superior A1C and body weight reductions from baseline across all three doses in adults with type 2 diabetes who have increased cardiovascular (CV) risk compared to titrated insulin glargine in topline results from Eli Lilly and Company's (NYSE: LLY) SURPASS-4 clinical trial. For the efficacy estimandi, the highest dose of tirzepatide led to an A1C reduction of 2.58 percent and reduced body weight by 11.7 kg (25.8 lb., 13.0 percent) compared to results for those treated with insulin glargine (A1C reduction of 1.44 percent and weight gain of 1.9 kg [4.2 lb., 2.2 percent]) at 52 weeks.
The overall safety profile of tirzepatide was consistent with the glucagon-like peptide-1 (GLP-1) receptor agonist class in this patient population. Gastrointestinal side effects were the most commonly reported adverse events, usually occurring during the escalation period and then decreasing over time.
SURPASS-4 is the largest and longest trial of the program to date and is the fifth and final global registration study for tirzepatide in type 2 diabetes to be completed. The study completion was driven by the accrual of major adverse cardiovascular events (MACE) in order to meet the regulatory submission requirements for evaluating CV risk for type 2 diabetes therapies. The primary endpoint was measured at 52 weeks, and many participants continued treatment beyond 52 weeks, some up to two years.
Tirzepatide is a novel investigational once-weekly dual glucose-dependent insulinotropic polypeptide (GIP) and GLP-1 receptor agonist that integrates the actions of the GIP and GLP-1 incretins into a single molecule, representing a new class of medicines being studied for the treatment of type 2 diabetes.
"Tirzepatide delivered impressive results in this study, providing superior A1C reductions compared to insulin glargine – as well as the addition of significant weight loss – in people with type 2 diabetes who have increased cardiovascular risk," said John Doupis, M.D., Ph.D., Director, Diabetes Division and Clinical Research Center, Iatriko Paleou Falirou Medical Center, Athens, Greece and Senior Investigator for SURPASS-4. "Type 2 diabetes is a complex condition that requires personalized approaches to treatment, and results from SURPASS-4 demonstrate the potential of tirzepatide to be an important option to help reduce A1C and weight for people with type 2 diabetes on one or up to three oral medicines."
About tirzepatide
Tirzepatide is a once-weekly dual glucose-dependent insulinotropic polypeptide (GIP) and glucagon-like peptide-1 (GLP-1) receptor agonist that integrates the actions of both incretins into a single novel molecule. GIP is a hormone that may complement the effects of GLP-1 receptor agonists. In preclinical models, GIP has been shown to decrease food intake and increase energy expenditure therefore resulting in weight reductions, and when combined with a GLP-1 receptor agonist, may result in greater effects on glucose and body weight. Tirzepatide is in phase 3 development for blood glucose management in adults with type 2 diabetes and for chronic weight management. It is also being studied as potential treatments for non-alcoholic steatohepatitis (NASH) and heart failure with preserved ejection fraction (HFpEF).
About SURPASS-4 and the SURPASS clinical trial program
SURPASS-4 (NCT03730662) is a 52-week, randomized, parallel, open-label trial comparing the efficacy and safety of tirzepatide 5 mg, 10 mg and 15 mg to insulin glargine in adults with type 2 diabetes inadequately controlled with at least one and up to three oral antihyperglycemic medications (metformin, sulfonylureas or SGLT-2 inhibitors), who have increased cardiovascular (CV) risk. The trial randomized 2,002 study participants in a 1:1:1:3 ratio to receive either tirzepatide 5 mg, 10 mg or 15 mg or insulin glargine. Participants were located in the European Union, North America (Canada and the United States), Australia, Israel, Taiwan and Latin America (Brazil, Argentina and Mexico). The primary objective of the study was to demonstrate that tirzepatide (10 mg and/or 15 mg) is non-inferior to insulin glargine for change from baseline A1C at 52 weeks in people with type 2 diabetes and increased CV risk. The primary and key secondary endpoints were assessed at 52 weeks, with some participants continuing treatment for up to two years. Study participants had a mean A1C between 7.5 percent and 10.5 percent and a BMI greater than or equal to 25 kg/m2. All participants in the tirzepatide treatment arms started the study at a dose of tirzepatide 2.5 mg once-weekly and then increased the dose in a step-wise approach at four-week intervals to their final randomized maintenance dose of 5 mg (via a 2.5 mg step), 10 mg (via steps at 2.5 mg, 5 mg and 7.5 mg) or 15 mg (via steps at 2.5 mg, 5 mg, 7.5 mg, 10 mg and 12.5 mg). All participants in the titrated insulin glargine treatment arm started with a baseline dose of 10 units per day and titrated following a treat-to-target algorithm to reach a fasting blood glucose below 100 mg/dL.
The SURPASS phase 3 global clinical development program for tirzepatide has enrolled more than 13,000 people with type 2 diabetes across 10 clinical trials, five of which are global registration studies. The program began in late 2018, and all five global registration trials have been completed.
For the latest updates, visit http://www.lillydiabetes.com/ or follow us on Twitter: @LillyDiabetes and Facebook: LillyDiabetesUS.
To learn more about Lilly, please visit us at lilly.com and lilly.com/newsroom.
View original content to download multimedia:http://www.prnewswire.com/news-releases/lillys-tirzepatide-achieves-all-primary-and-key-secondary-study-outcomes-against-insulin-glargine-in-adults-with-type-2-diabetes-and-increased-cardiovascular-risk-in-surpass-4-trial-301295524.html
SOURCE Eli Lilly & Company
Lilly’s Tirzepatide reaches endpoints in open label study for patients with type 2 diabetes
May 20, 2021 7:37 AM ET Eli Lilly and Company (LLY)
By: Dulan Lokuwithana, SA News Editor
- Eli Lilly (NYSE:LLY) posted topline results from its SURPASS-4 clinical trial, an open-label global trial to evaluate its tirzepatide in people with type 2 diabetes.
- https://seekingalpha.com/news/3698580-lillys-tirzepatide-reaches-endpoints-in-open-label-study-for-patients-with-type-2-diabetes
- https://seekingalpha.com/symbol/LLY
- https://www.lilly.com/
- https://www.lillydiabetes.com/
Relatlimab & OPDIVO® (nivolumab)
Bristol Myers Squibb Announces LAG-3-Blocking Antibody Relatlimab and Nivolumab Fixed-Dose Combination Significantly Improves Progression-Free Survival vs. Opdivo (nivolumab) in Patients with Previously Untreated Metastatic or Unresectable Melanoma
05/19/2021CATEGORY:
First presentation of Phase 3 data from a trial evaluating a LAG-3-blocking antibody
Fixed-dose combination of relatlimab and nivolumab demonstrated statistically significant and clinically meaningful benefit over Opdivo monotherapy, an established standard of care
Data demonstrate inhibiting LAG-3 together with PD-1 helps improve outcomes for patients
Data to be featured in an oral presentation during the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced results from the Phase 2/3 RELATIVITY-047 trial, which showed that the fixed-dose combination of relatlimab, a LAG-3-blocking antibody, and nivolumab, administered as a single infusion, demonstrated a statistically significant and clinically meaningful progression-free survival (PFS) benefit compared to Opdivo (nivolumab) alone in patients with previously untreated metastatic or unresectable melanoma. This is the first regimen to demonstrate a statistical benefit over anti-PD-1 monotherapy in metastatic melanoma. Among patients treated with the combination, the median PFS (mPFS) was significantly longer at 10.12 months (95% Confidence Interval [CI]: 6.37-15.74) vs. 4.63 in those who received Opdivo (95% CI: 3.38–5.62); (Hazard Ratio [HR] 0.75; 95% CI: 0.62-0.92, p=0.0055). The PFS benefit of the fixed-dose combination was observed early, at the time of the first scan, and was consistent over time. In exploratory, descriptive analyses, the combination of relatlimab and nivolumab extended PFS regardless of pre-specified subgroups and stratification factors.
These findings (Abstract #9503), the first from a Phase 3 trial evaluating a LAG-3-blocking antibody, will be presented in an oral abstract session on Sunday, June 6, 2021, from 8:00 a.m. – 11:00 a.m. EDT during the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting and have been selected for the official ASCO press program.
About RELATIVITY-047 (CA224-047)
RELATIVITY-047 (CA224-047) is a global, randomized, double-blind Phase 2/3 study evaluating the fixed-dose combination of relatlimab and nivolumab in patients with previously untreated metastatic or unresectable melanoma versus Opdivo alone. The primary endpoint of the trial is progression-free survival (PFS) by Blinded Independent Central Review (BICR) and the secondary endpoints are overall survival (OS) and objective response rate (ORR). A total of 714 patients were randomized 1:1 to receive a fixed-dose combination of relatlimab 160 mg and nivolumab 480 mg or Opdivo 480 mg by intravenous infusion every four weeks until disease recurrence, unacceptable toxicity or withdrawal of consent. Follow-up for the secondary endpoints of OS and ORR is ongoing and the Company remains blinded.
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.
About Opdivo
Opdivo is a programmed death-1 (PD-1) immune checkpoint inhibitor that is designed to uniquely harness the body’s own immune system to help restore anti-tumor immune response. By harnessing the body’s own immune system to fight cancer, Opdivo has become an important treatment option across multiple cancers.
Opdivo’s leading global development program is based on Bristol Myers Squibb’s scientific expertise in the field of Immuno-Oncology and includes a broad range of clinical trials across all phases, including Phase 3, in a variety of tumor types. To date, the Opdivo clinical development program has treated more than 35,000 patients. The Opdivo trials have contributed to gaining a deeper understanding of the potential role of biomarkers in patient care, particularly regarding how patients may benefit from Opdivo across the continuum of PD-L1 expression.
In July 2014, Opdivo was the first PD-1 immune checkpoint inhibitor to receive regulatory approval anywhere in the world. Opdivo is currently approved in more than 65 countries, including the United States, the European Union, Japan and China. In October 2015, the Company’s Opdivo and Yervoy combination regimen was the first Immuno-Oncology combination to receive regulatory approval for the treatment of metastatic melanoma and is currently approved in more than 50 countries, including the United States and the European Union.
INDICATIONS
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express PD-L1 (≥1%) as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab) and 2 cycles of platinum-doublet chemotherapy, is indicated for the first-line treatment of adult patients with metastatic or recurrent non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab) is indicated for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving OPDIVO.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with unresectable malignant pleural mesothelioma (MPM).
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of patients with intermediate or poor risk advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab), in combination with cabozantinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab) is indicated for the treatment of patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy.
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with classical Hodgkin lymphoma (cHL) that has relapsed or progressed after autologous hematopoietic stem cell transplantation (HSCT) and brentuximab vedotin or after 3 or more lines of systemic therapy that includes autologous HSCT. This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab) is indicated for the treatment of patients with recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN) with disease progression on or after platinum-based therapy.
OPDIVO® (nivolumab) is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of adult and pediatric (12 years and older) patients with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab) is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection.
OPDIVO® (nivolumab) is indicated for the treatment of patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) after prior fluoropyrimidine- and platinum-based chemotherapy.
OPDIVO® (nivolumab), in combination with fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the treatment of patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma.
For more information about Bristol Myers Squibb, visit us at BMS.com
Bristol's Opdivo + relatlimab combo beats Opdivo alone in melanoma trial
May 19, 2021 5:45 PM ET Bristol-Myers Squibb Company (BMY) By: Jonathan M Block, SA News Editor6 Comments
- New phase 2/3 data has demonstrated that a combination of Bristol-Myers Squibb's (NYSE:BMY) Opdivo (nivolumab) plus the experimental LAG-3 blocking antibody relatlimab led to better progression-free survival in melanoma than Opdivo monotherapy.
- https://seekingalpha.com/news/3698472-bristol-opdivo-relatlimab-combo-beats-opdivo-alone-in-melanoma-trial
- https://seekingalpha.com/symbol/BMY
- https://www.bms.com/
TYVYT® (sintilimab injection) in Combination with Pemetrexed and Platinum Chemotherapy
U.S. FDA Accepts Regulatory Submission for Sintilimab in Combination with Pemetrexed and Platinum Chemotherapy for the First-Line Treatment of People with Nonsquamous Non-Small Cell Lung Cancer
NEWS PROVIDED BY
May 18, 2021, 00:09 ET
SAN FRANCISCO and SUZHOU, China, May 18, 2021 /PRNewswire/ -- Innovent Biologics, Inc. (HKEX: 01801) and Eli Lilly and Company (NYSE: LLY) today jointly announced that the U.S. Food and Drug Administration (FDA) accepted for review a Biologics License Application (BLA) for sintilimab injection in combination with pemetrexed and platinum chemotherapy for the first-line treatment of people with nonsquamous non-small cell lung cancer (NSCLC). This is the first U.S. regulatory submission of sintilimab, a PD-1 inhibitor being developed and commercialized under a global collaboration agreement between Innovent and Lilly.
"The acceptance of this application – the first for sintilimab in the U.S. and outside of China – is an important milestone in Innovent's global commercialization strategy and in our collaboration with Lilly," said Dr. Yongjun Liu, president of Innovent. "We look forward to working closely with the FDA to potentially bring this sintilimab-pemetrexed-platinum chemotherapy combination as a treatment option in the U.S., following the regimen's regulatory approval in China earlier this year."
About the ORIENT-11 Trial
ORIENT-11 is a randomized, double-blind, Phase 3 clinical trial assessing the efficacy and safety of sintilimab in combination with pemetrexed and platinum chemotherapy compared to placebo in combination with pemetrexed and platinum chemotherapy as a first-line treatment for patients with advanced or metastatic nonsquamous non-small cell lung cancer (NSCLC), with no sensitizing EGFR mutations or ALK rearrangements. The primary endpoint is progression-free survival (PFS) as assessed by Independent Radiographic Review Committee (IRRC) based on RECIST v1.1., and secondary endpoints include overall survival (OS) and safety profile.
A total of 397 patients were enrolled and randomized 2:1 to receive either sintilimab 200mg or placebo in combination with pemetrexed and platinum chemotherapy every three weeks for up to four cycles, followed by either sintilimab or placebo plus pemetrexed maintenance therapy. Patients received treatment until radiographic disease progression, unacceptable toxicity or any other conditions that required treatment discontinuation. Conditional crossover was permitted. The results of the ORIENT-11 study were published in 2020.1
About Sintilimab
Sintilimab, marketed as TYVYT® (sintilimab injection) in China, is an innovative PD-1 inhibitor with global quality standards jointly developed by Innovent and Lilly. 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 for:
- 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 nonsquamous non-small cell lung cancer
Additionally, Innovent currently has regulatory submissions under review in China for sintilimab:
- In combination with gemcitabine and platinum chemotherapy, for the first-line treatment of squamous non-small cell lung cancer
- In combination with BYVASDA® (bevacizumab injection) for the first-line treatment of hepatocellular carcinoma
- The second-line treatment of squamous non-small cell lung cancer
In May 2021, the U.S. FDA accepted for review the Biologics License Application (BLA) for sintilimab in combination with pemetrexed and platinum chemotherapy for the first-line treatment of nonsquamous non-small cell lung cancer.
Sintilimab was included in China's National Reimbursement Drug List (NRDL) in 2019 as the first PD-1 inhibitor and the only PD-1 included in the list in that year.
For more information, please visit: www.innoventbio.com.
To learn more about Lilly's commitment to people with cancer, please visit www.LillyOncology.com.
To learn more about Lilly, please visit www.lilly.com and lilly.com/newsroom.
About Innovent Biologics' Strategic Collaboration with Eli Lilly and Company
Innovent entered into a strategic collaboration with Lilly focused on biological medicine in March 2015 – a groundbreaking partnership between a Chinese pharmaceutical company and a multinational pharmaceutical company. Under the agreement, Innovent and Lilly are co-developing and commercializing oncology medicines, including sintilimab in China. In October 2015, the two companies announced the extension of their existing collaboration to include co-development of three additional oncology antibodies targeting oncology indications. In August 2019, Innovent further entered a licensing agreement with Lilly to develop and commercialize a potentially global best-in-class diabetes medicine in China. Its collaboration with Lilly indicates that Innovent has established a comprehensive level of cooperation between China's innovative pharmaceuticals sector and the international pharmaceuticals sector in fields such as R&D, CMC, clinical development and commercialization. In August 2020，Lilly and Innovent announced a global expansion of their strategic alliance for sintilimab, whereby Lilly obtained an exclusive license for sintilimab for geographies outside of China.
Note:
TYVYT® (sintilimab injection; Innovent), BYVASDA® (bevacizumab biosimilar injection; Innovent), SULINNO® (adalimumab biosimilar injection; Innovent), and HALPRYZA® (rituximab biosimilar injection; Innovent) are not approved products in the United States.
FDA accepts Lilly, Innovent's sintilimab + chemotherapy application in lung cancer
May 18, 2021 12:32 AM ET Eli Lilly and Company (LLY) By: Mamta Mayani, SA News Editor1 Comment
- Eli Lilly and Company (NYSE:LLY) and Innovent Biologics (OTCPK:IVBIY) announce that the FDA has accepted for review a Biologics License Application (BLA) for sintilimab injection in combination with pemetrexed and platinum chemotherapy for the first-line treatment of people with nonsquamous non-small cell lung cancer (NSCLC).
- https://seekingalpha.com/news/3697564-fda-accepts-lilly-innovents-sintilimab-chemotherapy-application-in-lung-cancer
- https://seekingalpha.com/symbol/IVBIY
- https://www.hkex.com.hk/Market-Data/Securities-Prices/Equities/Equities-Quote?sym=1801&sc_lang=en
- www.innoventbio.com
- https://seekingalpha.com/symbol/LLY
- https://www.lilly.com/
INNOVENT BIOLOGICS, INC. (1801)
https://www.hkex.com.hk/Market-Data/Securities-Prices/Equities/Equities-Quote?sym=1801&sc_lang=en
BRUKINSA® (Zanubrutinib)
BeiGene Announces U.S. FDA Acceptance and Priority Review of Supplemental New Drug Application for BRUKINSA® (Zanubrutinib) in Marginal Zone Lymphoma
May 19, 2021 at 7:00 AM EDT
CAMBRIDGE, Mass. & BEIJING--(BUSINESS WIRE)--May 19, 2021-- BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a global biotechnology company focused on developing and commercializing innovative medicines worldwide, today announced that the U.S. Food and Drug Administration (FDA) has accepted a supplemental new drug application (sNDA) for BRUKINSA® (zanubrutinib) for the treatment of adult patients with marginal zone lymphoma (MZL) who have received at least one prior anti-CD20-based therapy and granted priority review. The Prescription Drug User Fee Act (PDUFA) target action date is September 19, 2021.
“This is our first regulatory submission in MZL, a serious disease diagnosed in more than 2,000 patients every year in the U.S., with no clear standard of care. In clinical trials, BRUKINSA has demonstrated promising efficacy and tolerability in MZL and presents a potential new option for MZL patients,” said Jane Huang, M.D., Chief Medical Officer, Hematology at BeiGene. “We look forward to continuing our communications with the FDA in the coming months as we work on advancing the broad global development program for our potentially best-in-class BTK inhibitor.”
BRUKINSA® (Zanubrutinib)
About BRUKINSA
BRUKINSA is a small molecule inhibitor of Bruton’s tyrosine kinase (BTK) discovered by BeiGene scientists that is currently being evaluated globally in a broad clinical program as a monotherapy and in combination with other therapies to treat various B-cell malignancies. Because new BTK is continuously synthesized, BRUKINSA was specifically designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity. With differentiated pharmacokinetics compared to other approved BTK inhibitors, BRUKINSA has been demonstrated to inhibit the proliferation of malignant B cells within a number of disease relevant tissues.
BRUKINSA is approved in the following indications and regions:
- For the treatment of mantle cell lymphoma (MCL) in adult patients who have received at least one prior therapy (United States, November 2019)*;
- For the treatment of MCL in adult patients who have received at least one prior therapy (China, June 2020)**;
- For the treatment of chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) in adult patients who have received at least one prior therapy (China, June 2020)**;
- For the treatment of relapsed or refractory MCL (United Arab Emirates, February 2021); and
- For the treatment of Waldenström’s macroglobulinemia (WM) in adult patients (Canada, March 2021).
To-date, more than 30 marketing authorization applications in multiple indications have been submitted outside of the United States and China, covering countries in the European Union and more than 20 other countries.
* This indication was approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
** This indication was approved under conditional approval. Complete approval for this indication may be contingent upon results from ongoing randomized, controlled confirmatory clinical trials.
Please see full U.S. Prescribing Information at www.beigene.com/PDF/BRUKINSAUSPI.pdf and Patient Information at www.beigene.com/PDF/BRUKINSAUSPPI.pdf.
To learn more about BeiGene, please visit www.beigene.com
1. Lymphoma Research Foundation. Available at https://lymphoma.org/aboutlymphoma/nhl/mzl/ Accessed April 2021.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210519005166/en/
Source: BeiGene, Ltd.
BeiGene's Brukinsa nabs accelerated review in U.S. for marginal zone lymphoma
May 19, 2021 7:14 AM ET BeiGene, Ltd. (BGNE)
By: Mamta Mayani, SA News Editor
- Under priority review, the FDA has accepted BeiGene's (NASDAQ:BGNE) supplemental new drug application (sNDA) for Brukinsa (zanubrutinib) for the treatment of adult patients with marginal zone lymphoma (MZL) who have received at least one prior anti-CD20-based therapy and granted priority review.
- https://seekingalpha.com/news/3698123-beigenes-brukinsa-nabs-accelerated-review-in-us-for-marginal-zone-lymphoma
- https://seekingalpha.com/symbol/BGNE
- https://www.beigene.com/?loc=us
- https://www.brukinsa.com/
BEIGENE, LTD. (6160)
https://www.hkex.com.hk/Market-Data/Securities-Prices/Equities/Equities-Quote?sym=6160&sc_lang=en
KEYTRUDA® (pembrolizumab) In Combination With Chemotherapy Before Surgery
Merck Announces Phase 3 KEYNOTE-522 Trial Met Dual Primary Endpoint of Event-Free Survival (EFS) in Patients With High-Risk Early-Stage Triple-Negative Breast Cancer (TNBC)
May 13, 2021 6:45 am ET
In Pivotal Study, KEYTRUDA® (pembrolizumab) In Combination With Chemotherapy Before Surgery and Continuing as a Single Agent After Surgery Showed Statistically Significant Improvement in EFS Versus Pre-Operative Chemotherapy
KEYTRUDA Is the First Anti-PD-1 Therapy to Show a Statistically Significant Improvement in EFS as Neoadjuvant and Adjuvant Therapy for TNBC
KENILWORTH, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced positive results from the pivotal neoadjuvant/adjuvant Phase 3 KEYNOTE-522 trial investigating KEYTRUDA, Merck’s anti-PD-1 therapy, in combination with chemotherapy as pre-operative (neoadjuvant) treatment and then continuing as a single agent (adjuvant) treatment after surgery. KEYNOTE-522 met its dual primary endpoint of event-free survival (EFS) for the treatment of patients with high-risk early-stage triple-negative breast cancer (TNBC). Based on an interim analysis conducted by the independent Data Monitoring Committee (DMC), neoadjuvant KEYTRUDA plus chemotherapy followed by adjuvant KEYTRUDA as monotherapy showed a statistically significant and clinically meaningful improvement in EFS compared with neoadjuvant chemotherapy alone. As previously communicated, KEYNOTE-522 met its other dual primary endpoint of pathological complete response (pCR). The safety profile of KEYTRUDA in this trial was consistent with that observed in previously reported studies; no new safety signals were identified.
“KEYTRUDA is the first immunotherapy to show positive results for event-free survival in patients with high-risk early-stage TNBC, a particularly aggressive form of breast cancer,” said Dr. Roy Baynes, senior vice president and head of global clinical development, chief medical officer, Merck Research Laboratories. “The improvement in pathological complete response rates initially observed following pre-operative treatment was encouraging, and now that we are seeing the data mature after four years to include a statistically significant improvement in event-free survival, we look forward to working with the FDA and other global authorities to bring this new option to patients as quickly as possible. We are grateful to the study participants who are critical to our efforts to advance potential treatment options for patients with TNBC.”
About KEYNOTE-522
KEYNOTE-522 is a Phase 3, randomized, double-blind trial (ClinicalTrials.gov, NCT03036488), evaluating a regimen of neoadjuvant KEYTRUDA in combination with chemotherapy followed by adjuvant KEYTRUDA as monotherapy versus a regimen of neoadjuvant chemotherapy followed by adjuvant placebo. The dual primary endpoints are pCR and EFS. The secondary endpoints include pCR rate using alternative definitions (i.e., no invasive or noninvasive residual cancer in breast or nodes) at the time of definitive surgery, overall survival, EFS in patients whose tumors express PD-L1 (Combined Positive Score [CPS] ≥1), safety and patient-reported outcomes. The study enrolled 1,174 patients who were randomized 2:1 to receive either:
- KEYTRUDA (every three weeks) plus paclitaxel (weekly) and carboplatin (weekly or every three weeks) for four cycles, followed by KEYTRUDA plus cyclophosphamide and either doxorubicin or epirubicin (every three weeks) for four cycles as neoadjuvant therapy prior to surgery, followed by nine cycles of KEYTRUDA (every three weeks) as adjuvant therapy post-surgery or;
- Placebo (every three weeks) plus paclitaxel (weekly) and carboplatin (weekly or every three weeks) for four cycles, followed by placebo plus cyclophosphamide and either doxorubicin or epirubicin (every three weeks) for four cycles as neoadjuvant therapy prior to surgery, followed by nine cycles of placebo (every three weeks) as adjuvant therapy post-surgery.
About KEYTRUDA® (pembrolizumab) Injection, 100 mg
KEYTRUDA is an anti-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 1,400 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.
Selected KEYTRUDA® (pembrolizumab) Indications in the U.S.
Melanoma
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.
KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.
Non-Small Cell Lung Cancer
KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) ≥1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is stage III where patients are not candidates for surgical resection or definitive chemoradiation, or metastatic.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS ≥1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.
Head and Neck Squamous Cell Cancer
KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS) ≥1] as determined by an FDA-approved test.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.
Classical Hodgkin Lymphoma
KEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).
KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.
Primary Mediastinal Large B-Cell Lymphoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.
Urothelial Carcinoma
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 (CPS ≥10), as determined by an FDA-approved test, or in patients who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.
Microsatellite Instability-High or Mismatch Repair Deficient Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR)
- solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options, or
- colorectal cancer that has progressed following treatment with fluoropyrimidine, oxaliplatin, and irinotecan.
This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.
Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer
KEYTRUDA is indicated for the first-line treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).
Gastric Carcinoma
KEYTRUDA, in combination with trastuzumab, and fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of patients with locally advanced unresectable or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent locally advanced or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test, with disease progression on or after two or more prior lines of therapy including fluoropyrimidine- and platinum-containing chemotherapy and if appropriate, HER2/neu-targeted therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Esophageal Carcinoma
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic esophageal or gastroesophageal junction (GEJ) (tumors with epicenter 1 to 5 centimeters above the GEJ) carcinoma that is not amenable to surgical resection or definitive chemoradiation either:
- in combination with platinum- and fluoropyrimidine-based chemotherapy, or
- as a single agent after one or more prior lines of systemic therapy for patients with tumors of squamous cell histology that express PD L1 (CPS ≥10) as determined by an FDA approved test.
Cervical Carcinoma
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Hepatocellular Carcinoma
KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Merkel Cell Carcinoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Renal Cell Carcinoma
KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).
Tumor Mutational Burden-High
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [≥10 mutations/megabase] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.
Cutaneous Squamous Cell Carcinoma
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) that is not curable by surgery or radiation.
Triple-Negative Breast Cancer
KEYTRUDA, in combination with chemotherapy, is indicated for the treatment of patients with locally recurrent unresectable or metastatic triple-negative breast cancer (TNBC) whose tumors express PD-L1 (CPS ≥10) as determined by an FDA-approved test. This indication is approved under accelerated approval based on progression-free survival. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
For more information, visit www.merck.com
Please see Prescribing Information for KEYTRUDA (pembrolizumab) at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_pi.pdf and Medication Guide for KEYTRUDA at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_mg.pdf.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210513005321/en/
Merck's Keytruda meets dual primary endpoint in late-stage triple-negative breast cancer study
May 13, 2021 8:29 AM ET Merck & Co., Inc. (MRK) By: Mamta Mayani, SA News Editor1 Comment
- Merck (NYSE:MRK) announces positive results from Phase 3 KEYNOTE-522 trial investigating Keytruda in combination with chemotherapy as pre-operative (neoadjuvant) treatment and then continuing as a single agent (adjuvant) treatment after surgery.
- https://seekingalpha.com/news/3695880-mercks-keytruda-meets-dual-primary-endpoint-in-late-stage-triple-negative-breast-cancer-study
- https://seekingalpha.com/symbol/MRK
- https://www.merck.com/
- https://www.keytruda.com/
XARELTO® (rivaroxaban) Plus Aspirin
Late-Breaking Data at ACC.21 Show XARELTO® (rivaroxaban) Plus Aspirin Significantly Reduced Total Ischemic Events in Peripheral Artery Disease (PAD) Patients After Lower-Extremity Revascularization Phase 3 VOYAGER PAD is the first investigational study in 20 years to highlight the benefit of long-term treatment in these high-risk patients Data add to growing body of evidence on the role of dual pathway inhibition in targeting both thrombin generation and platelets RARITAN, N.J., May 16, 2021 – The Janssen Pharmaceutical Companies of Johnson & Johnson today presented new data from the Phase 3 VOYAGER PAD study which showed XARELTO® (rivaroxaban) (2.5 mg twice daily) in combination with aspirin (100 mg once daily) consistently reduced severe vascular events in patients with peripheral artery disease (PAD) after lower-extremity revascularization (LER) compared to aspirin alone regardless of whether it was the first, second, third, or subsequent event. The primary results of VOYAGER PAD showed that XARELTO® plus aspirin reduced first events by 15 percent among patients with PAD after LER. This analysis showed a very high burden of subsequent events and a consistent 14 percent reduction in both primary endpoint events and total vascular events over a median of 2.5 years. These data were presented as a late-breaking presentation during the virtual American College of Cardiology’s 70th Annual Scientific Session (ACC.21) and simultaneously published in the Journal of the American College of Cardiology.
Late-breaking data at #ACC21 offer insights on long-term prevention of future thrombotic events in patients with #PAD after lower extremity revascularization.
About VOYAGER PAD The Phase 3 VOYAGER PAD study included 6,564 patients from 542 sites across 34 countries worldwide. Patients were randomized in a 1:1 ratio and received either XARELTO® (2.5 mg twice daily) plus aspirin (100 mg once daily) (n=3,286) or aspirin alone (100 mg once daily) (n=3,278). Patients were stratified by revascularization procedure type (endovascular vs. surgical) and use of clopidogrel, which was administered at the treating physician’s discretion. Patients were followed for a median of 28 months. The primary efficacy endpoint was a composite of major adverse limb and cardiovascular (CV) events, including acute limb ischemia, major amputation for vascular causes, heart attack (myocardial infarction), ischemic stroke, or death from CV causes. Additional prespecified categories of vascular events included subsequent peripheral revascularizations of both index and contralateral leg and venous thromboembolic events. The principal safety endpoint was major bleeding according to the TIMI classification.
Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088. You may also report side effects to Janssen Pharmaceuticals, Inc., at 1-800-JANSSEN (1-800-526-7736). Please read full Prescribing Information, including Boxed Warnings, and Medication Guide for XARELTO®. Trademarks are those of their respective owners. Janssen and Bayer together are developing rivaroxaban. For more information about XARELTO®, visit www.xarelto.com.
Learn more at www.janssen.com
Late-Breaking Data at ACC.21 Show XARELTO® (rivaroxaban) Plus Aspirin Significantly Reduced Total Ischemic Events in Peripheral Artery Disease (PAD) Patients After Lower-Extremity Revascularization
May 16, 2021 8:01 AM ETPR Newswire
Data add to growing body of evidence on the role of dual pathway inhibition in targeting both thrombin generation and platelets
PR Newswire
RARITAN, N.J., May 16, 2021 /PRNewswire/ -- The Janssen Pharmaceutical Companies of Johnson & Johnson today presented new data from the Phase 3 VOYAGER PAD study which showed XARELTO® (rivaroxaban) (2.5 mg twice daily) in combination with aspirin (100 mg once daily) consistently reduced severe vascular events in patients with peripheral artery disease (PAD) after lower-extremity revascularization (LER) compared to aspirin alone regardless of whether it was the first, second, third, or subsequent event. The primary results of VOYAGER PAD showed that XARELTO® plus aspirin reduced first events by 15 percent among patients with PAD after LER. This analysis showed a very high burden of subsequent events and a consistent 14 percent reduction in both primary endpoint events and total vascular events over a median of 2.5 years. These data were presented as a late-breaking presentation during the virtual American College of Cardiology's 70th Annual Scientific Session (ACC.21) and simultaneously published in the Journal of the American College of Cardiology.
Janssen Pharma's Xarelto + aspirin significantly reduces total ischemic events in peripheral artery disease
May 17, 2021 12:35 AM ETJohnson & Johnson (JNJ)By: Mamta Mayani, SA News Editor1 Comment
- The Janssen Pharmaceutical Companies of Johnson & Johnson (NYSE:JNJ) announces new data from Phase 3 VOYAGER PAD study which showed Xarelto (rivaroxaban) (2.5 mg twice daily) in combination with aspirin (100 mg once daily) consistently reduced severe vascular events in patients with peripheral artery disease (PAD) after lower-extremity revascularization (LER) compared to aspirin alone.
- https://seekingalpha.com/news/3696894-janssen-pharmas-xarelto-aspirin-significantly-reduces-total-ischemic-events-in-peripheral-artery-disease
- https://seekingalpha.com/symbol/JNJ
- https://www.xarelto-us.com/
- https://www.janssen.com/us/
Mavacamten
Bristol Myers Squibb Presents Late-Breaking Phase 3 Data Demonstrating Health Status Benefits of Mavacamten in Patients with Obstructive Hypertrophic Cardiomyopathy at American College of Cardiology’s 70th Annual Scientific Session
05/15/2021
CATEGORY:
Mavacamten, a potential first-in-class cardiac myosin inhibitor for patients with symptomatic obstructive hypertrophic cardiomyopathy (oHCM), showed improvement in health status compared to placebo at 30 weeks in EXPLORER-HCM trial
Findings of new analysis published simultaneously in The Lancet
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced a new analysis of data from the Phase 3 EXPLORER-HCM study evaluating mavacamten, an investigational, first-in-class cardiac myosin inhibitor, in patients with obstructive hypertrophic cardiomyopathy (oHCM), which was presented at the American College of Cardiology’s 70th Annual Scientific Session (ACC.21), with simultaneous publication in The Lancet. At 30 weeks, the change in Kansas City Cardiomyopathy Questionnaire Overall Summary Score (KCCQ OSS) was greater in mavacamten patients than placebo, with similar benefits across all KCCQ subscales. Moreover, a greater proportion of mavacamten patients achieved a very large, clinically meaningful improvement (≥20 points) in the KCCQ OSS, compared to placebo, 36% [33/92] vs. 15% [13/88]. A change of at least 5 points is required to be considered clinically significant. These results were presented today as part of the Featured Clinical Research I Session (403-09) in the Hot Topics Channel from 12:15-1:30 p.m. EDT.
About the Phase 3 EXPLORER-HCM Mavacamten Hypertrophic Cardiomyopathy Trial
The EXPLORER-HCM Phase 3 trial enrolled a total of 251 patients with symptomatic (NYHA Class II or III), obstructive hypertrophic cardiomyopathy. All participants had measurable left ventricular outflow tract (LVOT) gradient (resting and/or provoked) ≥50 mmHg at baseline.
The primary endpoint for EXPLORER-HCM was a composite functional analysis designed to capture mavacamten’s effect on both symptoms and function. Secondary endpoints were changes from baseline to week 30 in postexercise LVOT gradient, pVO2, proportion of patients with at least one NYHA class improvement, and measures of patientreported outcomes. Additional endpoints included changes from baseline to Week 30 in echocardiographic indices, circulating biomarkers, cardiac rhythm patterns and accelerometry.
About Mavacamten
Mavacamten is a potential first-in-class, oral, allosteric modulator of cardiac myosin, under investigation for the treatment of conditions in which excessive cardiac contractility and impaired diastolic filling of the heart are the underlying cause. Mavacamten reduces cardiac muscle contractility by inhibiting excessive myosin-actin cross-bridge formation that results in hypercontractility, left ventricular hypertrophy and reduced compliance. In clinical and preclinical studies, mavacamten has consistently reduced biomarkers of cardiac wall stress, lessened excessive cardiac contractility and increased diastolic compliance.
For more information about Bristol Myers Squibb, visit us at BMS.com
Bristol Myers’ mavacamten improves health status in patients with obstructive hypertrophic cardiomyopathy
May 15, 2021 10:12 PM ET Bristol-Myers Squibb Company (BMY)
By: Dulan Lokuwithana, SA News Editor4 Comments
- Bristol Myers Squibb (NYSE:BMY) announced peer-reviewed late-stage data for its cardiac myosin inhibitor mavacamten indicating that it led to improvement in health status compared to placebo in patients with symptomatic obstructive hypertrophic cardiomyopathy ((oHCM)).
- https://seekingalpha.com/news/3696875-bristol-myers-mavacamten-improves-patients-with-obstructive-hypertrophic-cardiomyopathy
- https://seekingalpha.com/symbol/BMY
- https://www.bms.com/
DUPIXENT® (DUPILUMAB)
May 17, 2021 at 9:59 AM EDT Back
PIVOTAL DATA AT ATS 2021 SHOW DUPIXENT® (DUPILUMAB) SIGNIFICANTLY REDUCED ASTHMA ATTACKS AND IMPROVED LUNG FUNCTION IN CHILDREN
TARRYTOWN, N.Y. and PARIS, May 17, 2021 /PRNewswire/ --
Dupixent is the only biologic medicine to improve lung function in children aged 6 to 11 years with uncontrolled moderate-to-severe asthma in a randomized Phase 3 trial, with potential to be best-in-class treatment for these patients
FDA decision for children with moderate-to-severe asthma expected by October 21, 2021
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today announced that detailed results from a Phase 3 trial showed Dupixent® (dupilumab) significantly reduced severe asthma attacks, and within two weeks rapidly improved lung function in children aged 6 to 11 years with uncontrolled moderate-to-severe asthma, with evidence of type 2 inflammation. Dupixent also significantly improved overall asthma symptom control and reduced an airway biomarker of type 2 inflammation that plays a major role in asthma, called fractional exhaled nitric oxide (FeNO). These data are being presented at the 2021 American Thoracic Society International Conference (ATS 2021) and featured in the Breaking News: Clinical Trial Results in Pulmonary Medicine Scientific Symposium.
DUPIXENT® (DUPILUMAB)
"Children living with uncontrolled moderate-to-severe asthma experience serious and persistent symptoms that can impact many crucial aspects of their lives including school, sleep and exercise," said Leonard B. Bacharier, M.D., Professor of Pediatrics and Director of the Center for Pediatric Asthma Research, Monroe Carell Jr. Children's Hospital at Vanderbilt University Medical Center in Nashville, Tennessee and principal investigator of the trial. "The trial results show that dupilumab, when added to standard-of-care therapy, significantly reduced asthma attacks, rapidly improved lung function and improved asthma control, which is especially important to these children during a particularly formative time in their lives."
The Phase 3, randomized, double-blind, placebo-controlled VOYAGE trial evaluated the efficacy and safety of Dupixent (100 mg or 200 mg every two weeks, based on weight) combined with standard-of-care asthma therapy in 408 children with uncontrolled moderate-to-severe asthma. Two pre-specified populations with evidence of type 2 inflammation were evaluated for the primary analysis: 1) patients with baseline blood eosinophils (EOS) ≥300 cells/μl (n=259) and 2) patients with FeNO ≥20 parts per billion (ppb) or EOS ≥150 cells/μl (n=350).
Top line results from the trial, which met its primary and key secondary endpoints, were announced in October 2020. These data showed that patients who added Dupixent to standard-of-care in these two patient groups, respectively, experienced:
- Substantially reduced rate of severe asthma attacks, with a 65% (p<0.0001) and 59% (p<0.0001) average reduction over one year compared to placebo (0.24 and 0.31 events per year for Dupixent vs. 0.67 and 0.75 for placebo, respectively).
- Improved lung function observed as early as two weeks and sustained for up to 52 weeks, measured by percent predicted FEV1 (FEV1pp).
- At 12 weeks, patients taking Dupixent improved their lung function by 5.32 and 5.21 percentage points vs. placebo (p=0.0036 and p=0.0009, respectively).
- This measure seeks to evaluate a patient's change in lung function compared to their predicted lung function based on age, height, sex and ethnicity to account for children's growing lung capacity at different stages of development.
New VOYAGE Data Presented at ATS
Results presented for the first time at ATS in the primary patient populations showed patients taking Dupixent experienced:
- Significant improvement in asthma control at week 24 based on patient-reported disease symptoms and impact, measured on a 0-6 scale. On average, patients taking Dupixent improved their scores by 1.34 and 1.33 from baseline compared to 0.88 and 1.00 for placebo (average improvement of –0.46 and -0.33 for Dupixent vs. placebo, p<0.0001 and p=0.0001, respectively). The improvement from baseline in patients taking Dupixent was more than double the clinically meaningful threshold of 0.5 points on the Asthma Control Questionnaire 7-Interviewer Administered (ACQ-7-IA).
- Significant reduction in mean FeNO levels to below the threshold for type 2 inflammation, which is 20 parts per billion (ppb). Patients taking Dupixent had an average improvement in FeNO levels by -20.59 and -17.84 ppb vs. placebo from baseline to week 12 (p<0.0001 for both values).
The safety results from the trial were generally consistent with the known safety profile of Dupixent in patients aged 12 years and older with uncontrolled moderate-to-severe asthma. The overall rates of adverse events were 83% for Dupixent and 80% for placebo. Adverse events that were most commonly observed with Dupixent versus placebo included injection site reactions (18% Dupixent, 13% placebo), viral upper respiratory tract infections (12% Dupixent, 10% placebo) and eosinophilia (6% Dupixent, 1% placebo).
The safety and efficacy of Dupixent in this pediatric population have not been fully evaluated by any regulatory authority.
Dupixent is a fully human monoclonal antibody that inhibits the signaling of the interleukin-4 (IL-4) and interleukin-13 (IL-13) pathways and is not an immunosuppressant. It was invented using Regeneron's proprietary VelocImmune® technology. IL-4 and IL-13 are key and central drivers of the type 2 inflammation that plays a major role in asthma, chronic rhinosinusitis with nasal polyposis (CRSwNP), atopic dermatitis, and eosinophilic esophagitis.
About Dupixent
Dupixent is approved in the U.S. to treat patients aged 6 years and older with moderate-to-severe atopic dermatitis that is not well controlled with prescription therapies used on the skin (topical), or who cannot use topical therapies; for use with other asthma medicines for the maintenance treatment of moderate-to-severe eosinophilic or oral steroid dependent asthma in patients aged 12 years and older whose asthma is not controlled with their current asthma medicines; and for use with other medicines for the maintenance treatment of CRSwNP in adults whose disease is not controlled.
Outside of the U.S., Dupixent is approved for specific patients with moderate-to-severe atopic dermatitis and certain patients with asthma in a number of other countries around the world, including those in the EU and Japan. Dupixent is also approved in the EU and Japan to treat certain adults with severe CRSwNP. Across all approved indications globally, more than 260,000 patients have been treated with Dupixent.
About Regeneron's VelocImmune® Technology
Regeneron's VelocImmune technology utilizes a proprietary genetically engineered mouse platform endowed with a genetically humanized immune system to produce optimized fully human antibodies. When Regeneron's co-Founder, President and Chief Scientific Officer George D. Yancopoulos was a graduate student with his mentor Frederick W. Alt in 1985, they were the first to envision making such a genetically humanized mouse, and Regeneron has spent decades inventing and developing VelocImmune and related VelociSuite® technologies. Dr. Yancopoulos and his team have used VelocImmune technology to create multiple antibodies including Dupixent, Libtayo® (cemiplimab-rwlc), Praluent® (alirocumab), Kevzara® (sarilumab), Evkeeza® (evinacumab-dgnb) and InmazebTM (atoltivimab, maftivimab, and odesivimab-ebgn).
U.S. INDICATIONS
DUPIXENT is a prescription medicine used:
- to treat people aged 6 years and older with moderate-to-severe atopic dermatitis (eczema) that is not well controlled with prescription therapies used on the skin (topical), or who cannot use topical therapies. DUPIXENT can be used with or without topical corticosteroids. It is not known if DUPIXENT is safe and effective in children with atopic dermatitis under 6 years of age.
- with other asthma medicines for the maintenance treatment of moderate-to-severe eosinophilic or oral steroid dependent asthma in people aged 12 years and older whose asthma is not controlled with their current asthma medicines. DUPIXENT helps prevent severe asthma attacks (exacerbations) and can improve your breathing. DUPIXENT may also help reduce the amount of oral corticosteroids you need while preventing severe asthma attacks and improving your breathing. DUPIXENT is not used to treat sudden breathing problems. It is not known if DUPIXENT is safe and effective in children with asthma under 12 years of age.
- with other medicines for the maintenance treatment of chronic rhinosinusitis with nasal polyposis (CRSwNP) in adults whose disease is not controlled. It is not known if DUPIXENT is safe and effective in children with chronic rhinosinusitis with nasal polyposis under 18 years of age.
Please see accompanying full Prescribing Information including Patient Information.
For additional information about the company, please visit www.regeneron.com
View original content:http://www.prnewswire.com/news-releases/pivotal-data-at-ats-2021-show-dupixent-dupilumab-significantly-reduced-asthma-attacks-and-improved-lung-function-in-children-301292416.html
SOURCE Regeneron Pharmaceuticals, Inc.
Regeneron, Sanofi post promising data from late-stage Dupixent asthma trial
May 17, 2021 10:09 AM ET Regeneron Pharmaceuticals, Inc. (REGN) By: Aakash Babu, SA News Editor1 Comment
- Regeneron Pharmaceuticals (REGN +0.4%) and Sanofi (SNY +1.0%) announce detailed results from a Phase 3 trial in which Dupixent (dupilumab) significantly reduced severe asthma attacks, and improved lung function in children aged 6 to 11 years with uncontrolled moderate-to-severe asthma, with evidence of type 2 inflammation.
- https://seekingalpha.com/news/3697179-regeneron-sanofi-post-promising-data-from-late-stage-dupixent-asthma-trial
- https://seekingalpha.com/symbol/SNY
- https://seekingalpha.com/symbol/REGN
- https://www.regeneron.com/
Selinexor (XPOVIO®)
Antengene Announces IND Approval in China for a Global Phase III Trial of Selinexor in Advanced or Recurrent Endometrial Cancer
May 12, 2021View PDF
Shanghai and Hong Kong, PRC, May 13, 2021– Antengene Corporation Limited (“Antengene”, SEHK: 6996.HK), a leading innovative biopharmaceutical company dedicated to discovering, developing and commercializing global first-in-class and/or best-in-class therapeutics in hematology and oncology, today announced that China’s National Medical Products Administration (NMPA) has approved the Investigational New Drug (IND) application for a Phase III clinical trial designed to evaluate the safety and efficacy of selinexor (XPOVIO®) in the treatment of advanced or recurrent endometrial cancer (the SIENDO trial).
Selinexor (XPOVIO®) is an US Food and Drug Administration (FDA) approved oral selective inhibitor of nuclear export that has been included in five regimens recommended by the National Comprehensive Cancer Network (NCCN®) Guidelines and multiple regimens recommended by the Chinese Society of Clinical Oncology (CSCO) Diagnosis and Treatment Guidelines, for the treatment of multiple myeloma (MM) and diffuse large B-cell lymphoma (DLBCL). Selinexor inhibits XPO1, the only clinically proven nuclear export protein target. Based on its unique mechanism of action, selinexor can be combined with various other drugs in order to potentially enhance efficacy. The SIENDO trial is a global trial being conducted at over 80 centers across North America, Europe and Asia.
Selinexor (XPOVIO®)
About Selinexor (XPOVIO®)
Selinexor, a first-in-class and only-in-class oral selective inhibitor of nuclear export (SINE) compound discovered and developed by Karyopharm Therapeutics Inc. (NASDAQ: KPTI), is currently being developed by Antengene, which has the exclusive development and commercial rights in certain Asia-Pacific markets, including Greater China, South Korea, Australia, New Zealand and the ASEAN countries.
In July 2019, the US Food and Drug Administration (FDA) approved selinexor in combination with low-dose dexamethasone for the treatment of relapsed/refractory multiple myeloma (rrMM) and in June 2020 approved selinexor as a single-agent for the treatment of relapsed/refractory diffuse large B-cell lymphoma (rrDLBCL). In December 2020, selinexor also received FDA approval as a combination treatment for multiple myeloma after at least one prior therapy. In February 2021, selinexor was approved by the Israeli Ministry of Health for the treatment of patients with rrMM or rrDLBCL and in March 2021, the European Commission (EC) has granted conditional marketing authorization for selinexor (NEXPOVIO) for the treatment of adult patients with rrMM.
Selinexor is so far the first and only oral SINE compound approved by the FDA and is the first drug approved for the treatment of both MM and DLBCL. Selinexor is also being evaluated in several other mid-and later-phase clinical trials across multiple solid tumor indications, including liposarcoma and endometrial cancer. In November 2020, at the Connective Tissue Oncology Society 2020 Annual Meeting (CTOS 2020), Antengene’s partner, Karyopharm, presented positive results from the Phase III randomized, double blind, placebo controlled, cross-over SEAL trial evaluating single agent, oral selinexor versus matching placebo in patients with liposarcoma. Karyopharm also announced that the ongoing Phase III SIENDO trial of selinexor in patients with endometrial cancer passed the planned interim futility analysis and the Data and Safety Monitoring Board (DSMB) recommended the trial should proceed as planned without any modifications. Top-line SIENDO trial results are expected in the second half of 2021.
Antengene is currently conducting five late-stage clinical trials of selinexor for the treatment of MM, DLBCL, non-small cell lung cancer, and peripheral T and NK/T-cell lymphoma. Furthermore, Antengene has submitted New Drug Applications (NDAs) for selinexor in multiple Asia Pacific markets including China, Australia, South Korea, and Singapore, and was granted the Priority Review status by China’s NMPA and an Orphan Drug Designation by the Ministry of Food and Drug Safety of South Korea (MFDS).
Selinexor is a first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) compound. Selinexor functions by binding with, and inhibiting, the nuclear export protein, XPO1, leading to the accumulation of tumor suppressor proteins in the cell nucleus. This reinitiates and amplifies their tumor suppressor function and is believed to lead to the selective induction of apoptosis in cancer cells, while largely sparing normal cells.
Selinexor, also known as XPOVIO, has only been approved by the United States Food and Drug Administration (FDA) for the following indications:
• In combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody.
https://www.karyopharm.com/pipeline/oral-selinexor/
*XPOVIO® and NEXPOVIO® are registered trade mark of KaryopharmTherapeutics Inc..
https://www.karyopharm.com/pipeline/
Karyopharm/Antengene's selinexor application OK'd in China for endometrial cancer
May 13, 2021 1:40 AM ET Karyopharm Therapeutics Inc. (KPTI) By: Mamta Mayani, SA News Editor
- Antengene announces that China's National Medical Products Administration (NMPA) has approved the Investigational New Drug (IND) application for a Phase III trial to evaluate the safety and efficacy of selinexor (Xpovio) being co-developed by Karyopharm Therapeutics (NASDAQ:KPTI) in the treatment of advanced or recurrent endometrial cancer (the SIENDO trial).
- https://seekingalpha.com/news/3695693-karyopharmantengenes-selinexor-application-okd-in-china-for-endometrial-cancer
- https://seekingalpha.com/symbol/KPTI
- Antengene Corporation Limited (“Antengene”, SEHK: 6996.HK)
- https://www.hkex.com.hk/Market-Data/Securities-Prices/Equities/Equities-Quote?sym=6996&sc_lang=en
- ANTENGENE CORPORATION LTD. - B (6996)
- https://www.antengene.com/
REGEN-COV™ (CASIRIVIMAB WITH IMDEVIMAB)
May 17, 2021 at 10:01 AM EDT Back
PHASE 3 DATA PRESENTED AT ATS 2021 SHOW REGEN-COV™ (CASIRIVIMAB WITH IMDEVIMAB) REDUCED RISK OF HOSPITALIZATION OR DEATH BY 70% IN NON-HOSPITALIZED COVID-19 PATIENTS
TARRYTOWN, N.Y., May 17, 2021 /PRNewswire/ --
Patients treated with REGEN-COV had 4-day shorter duration of symptoms and significantly reduced viral load compared to placebo
Similar efficacy observed with both doses (1,200 mg and 2,400 mg); U.S. FDA is currently reviewing request to add lower 1,200 mg dose to EUA
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced the presentation of detailed results from the Phase 3 pivotal trial showing REGEN–COV™ (casirivimab with imdevimab) significantly reduced the risk of hospitalization or death, shortened symptom duration and reduced viral load in non-hospitalized patients (outpatients) with COVID-19. These data were presented at the 2021 American Thoracic Society International Conference (ATS 2021) in the Breaking News: Clinical Trial Results in Pulmonary Medicine Scientific Symposium, which features late-breaking information on leading clinical trials in pulmonary and critical care medicine.
REGEN-COV™ (CASIRIVIMAB WITH IMDEVIMAB)
"Despite increasing vaccination rates, many continue to be diagnosed with COVID-19 who could benefit from REGEN-COV due to underlying conditions like asthma or COPD that put them at higher risk for severe disease," said Julie Philley, M.D., Chair of Internal Medicine at the University of Texas Health Science Center and trial investigator. "The Phase 3 trial showed REGEN-COV helped patients avoid hospitalization or even death while speeding up recovery time. These results add to the growing body of clinical evidence, real-world experience and strong recommendations by the National Institutes of Health COVID-19 treatment guidelines that collectively underscore the urgent need to ensure all appropriate patients are treated."
The Phase 3, randomized, double-blind, placebo-controlled trial evaluated REGEN–COV in 4,567 high-risk outpatients with COVID-19. Two REGEN-COV doses were studied in the trial – the currently authorized 2,400 mg dose and a 1,200 mg dose under evaluation with the U.S. Food and Drug Administration (FDA). All patients evaluated for efficacy had at least one risk factor for severe COVID-19, such as chronic lung disease (including asthma), obesity, cardiovascular disease or being at least 50 years of age.
Positive topline results from the trial were announced in March 2021, showing REGEN–COV met its primary and all secondary endpoints, with similar efficacy observed for both doses. These data demonstrated patients treated with 1,200 mg or 2,400 mg REGEN–COV experienced:
- 70% (p=0.0024) and 71% (p<0.0001) reduced risk of hospitalization or death through day 29, compared to placebo.
- 4-day shorter time to symptom resolution, with a median of 10 days with both REGEN-COV groups compared to a median of 14 days with placebo.
- Reduced viral load by 0.71 log10 copies/mL and 0.86 log10 copies/mL in seven days, compared to placebo (p<0.0001 for both).
REGEN–COV continues to be assessed in the outpatient (symptomatic and asymptomatic infections), prevention and certain hospitalized COVID-19 patient settings. In April 2021, Regeneron announced positive initial results from a Phase 3 trial evaluating the ability of REGEN-COV to prevent COVID-19 infection among household contacts of SARS-CoV-2 infected individuals.
The development and manufacturing of REGEN–COV have been funded in part with federal funds from the Biomedical Advanced Research and Development Authority (BARDA), part of the U.S. Department of Health and Human Services, Office of the Assistant Secretary for Preparedness and Response, under OT number: HHSO100201700020C.
About the REGEN–COV Antibody Cocktail
REGEN–COV (casirivimab with imdevimab) is a cocktail of two monoclonal antibodies (also known as REGN10933 and REGN10987) that was designed specifically to block infectivity of SARS-CoV-2, the virus that causes COVID-19, using Regeneron's proprietary VelocImmune® and VelociSuite® technologies. The two potent, virus-neutralizing antibodies that form the cocktail bind non-competitively to the critical receptor binding domain of the virus's spike protein, which diminishes the ability of mutant viruses to escape treatment and protects against spike variants that have arisen in the human population, as detailed in Science.
Under an EUA issued by the FDA, REGEN–COV is currently available in the U.S. to treat mild-to-moderate COVID-19 in adults, as well as in pediatric patients at least 12 years of age and weighing at least 40 kg, who have received positive results of direct SARS-CoV-2 viral testing and are at high risk for progressing to severe COVID-19 and/or hospitalization. REGEN–COV has not been approved by FDA but has been authorized for emergency use. This use is authorized only for the duration of the declaration that circumstances exist justifying the authorization of the emergency use under section 564(b)(1) of the Act, 21 U.S.C. § 360bbb-3(b)(1), unless the authorization is terminated or revoked sooner.
The criteria for 'high-risk' patients were recently updated and are described in the Fact Sheet for Healthcare Providers. For additional information on medical conditions and factors associated with increased risk for progressing to severe COVID-19, see the CDC website. In the U.S., REGEN–COV is not authorized for use in patients who are hospitalized due to COVID-19 or require oxygen therapy, or for people currently using chronic oxygen therapy because of an underlying comorbidity who require an increase in baseline oxygen flow rate due to COVID-19.
Under this EUA, REGEN–COV is widely available throughout the U.S. – information on availability in your area is available from the Department of Health and Human Services and the National Infusion Center Association.
Regeneron is collaborating with Roche to increase global supply of REGEN–COV. Regeneron is responsible for development and distribution of the treatment in the U.S., and Roche is primarily responsible for development and distribution outside the U.S. The companies share a commitment to making the antibody cocktail available to COVID-19 patients around the globe and will support access in low- and lower-middle-income countries through drug donations to be made in partnership with public health organizations.
About Regeneron's VelocImmune Technology
Regeneron's VelocImmune technology utilizes a proprietary genetically engineered mouse platform endowed with a genetically humanized immune system to produce optimized fully human antibodies. When Regeneron's co-Founder, President and Chief Scientific Officer George D. Yancopoulos was a graduate student with his mentor Frederick W. Alt in 1985, they were the first to envision making such a genetically humanized mouse, and Regeneron has spent decades inventing and developing VelocImmune and related VelociSuite technologies. Dr. Yancopoulos and his team have used VelocImmune technology to create approximately a quarter of all original, FDA-approved fully human monoclonal antibodies currently available. This includes REGEN–COV™ (casirivimab with imdevimab), Dupixent® (dupilumab), Libtayo® (cemiplimab-rwlc), Praluent® (alirocumab), Kevzara® (sarilumab), Evkeeza® (evinacumab-dgnb) and Inmazeb™ (atoltivimab, maftivimab and odesivimab-ebgn).
Authorized Emergency Use
REGEN–COV (casirivimab with imdevimab to be administered together) is authorized for the treatment of mild to moderate coronavirus disease 2019 (COVID-19) in adults and pediatric patients (12 years of age and older weighing at least 40 kg) with positive results of direct SARS-CoV-2 viral testing, and who are at high risk for progressing to severe COVID-19 and/or hospitalization. [see Limitations of Authorized Use]
For additional information on medical conditions and factors associated with increased risk for progression to severe COVID, see the CDC website: https://www.cdc.gov/coronavirus/2019-ncov/need-extra-precautions/people-with-medical-conditions.html. Healthcare providers should consider the benefit-risk for an individual patient
For additional information about the company, please visit www.regeneron.com
SOURCE Regeneron Pharmaceuticals, Inc.
Regeneron is collaborating with Roche to increase global supply of REGEN–COV. Regeneron is responsible for development and distribution of the treatment in the U.S., and Roche is primarily responsible for development and distribution outside the U.S. The companies share a commitment to making the antibody cocktail available to COVID-19 patients around the globe and will support access in low- and lower-middle-income countries through drug donations to be made in partnership with public health organizations.
https://seekingalpha.com/symbol/REGN
biotecMAX™ April/May 2021 Insight
LIBTAYO® (CEMIPLIMAB)
May 12, 2021 at 1:30 PM EDT Back
POSITIVE PHASE 3 LIBTAYO® (CEMIPLIMAB) RESULTS IN ADVANCED CERVICAL CANCER PRESENTED AT ESMO VIRTUAL PLENARY
TARRYTOWN, N.Y. and PARIS, May 12, 2021 /PRNewswire/ --
Libtayo is the first immunotherapy to demonstrate an improvement in overall survival in advanced cervical cancer, as well as progression-free survival and objective response rate, compared to chemotherapy
Improvements in overall survival were seen in the overall population and both squamous cell carcinoma and adenocarcinoma subgroups
Additionally, the Phase 3 trial found significant differences in patient-reported outcomes favoring Libtayo over chemotherapy
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today announced the presentation of positive results from the Phase 3 trial investigating the PD-1 inhibitor Libtayo® (cemiplimab) in patients with recurrent or metastatic cervical cancer who had previously progressed on chemotherapy. The data were shared as part of a European Society for Medical Oncology (ESMO) Virtual Plenary and add to previously reported data showing an improvement in overall survival (OS) with Libtayo compared to chemotherapy. The data will form the basis of regulatory submissions in 2021.
"In this Phase 3 trial, Libtayo demonstrated a significant improvement in overall survival in women with advanced cervical cancer after progression on chemotherapy, reducing the risk of death by 31% compared to chemotherapy in the overall population," said Krishnansu S. Tewari, M.D., Professor and Director of the Division of Gynecologic Oncology at the University of California, Irvine and a trial investigator. "Improvements in progression-free survival and objective response rate were also demonstrated in the overall population compared to chemotherapy. Taken together, this landmark trial – which enrolled patients regardless of PD-L1 expression status – helps support the use of Libtayo as a potential new second-line treatment for women with advanced cervical cancer who face a poor prognosis and limited treatment options."
In the overall population, those treated with Libtayo (n=304) experienced significant improvements in OS, progression-free survival (PFS) and objective response rate (ORR), compared to chemotherapy (n=304), including a:
- 31% reduction in the risk of death (hazard ratio [HR]: 0.69; 95% confidence interval [CI]: 0.56-0.84; one-sided p=0.00011).
- 25% reduction in the risk of disease progression (HR: 0.75; 95% CI: 0.63-0.89; one-sided p=0.00048).
- 16% ORR (50 patients; 95% CI: 13-21%; one-sided p=0.00004), compared to 6% with chemotherapy (19 patients). Median duration of response was 16 months with Libtayo (95% CI: 12 months to not yet evaluable) and 7 months with chemotherapy (95% CI: 5-8 months), per Kaplan-Meier estimates.
In the trial, 78% of patients had advanced cervical cancer that was classified as squamous cell carcinoma (SCC). In this patient subgroup, significant improvements were also seen with Libtayo (n=239), compared to chemotherapy (n=238), including a:
- 27% reduction in the risk of death (HR: 0.73; 95% CI: 0.58-0.91; one-sided p=0.00306).
- 29% reduction in the risk of disease progression (HR: 0.71; 95% CI: 0.58-0.86; one-sided p=0.00026).
- 18% ORR (42 patients; 95% CI: 13-23%), compared to 7% with chemotherapy (16 patients; 95% CI: 4-11%).
While assessment of the adenocarcinoma was not a pre-specified endpoint, a post-hoc analysis demonstrated the following outcomes for Libtayo-treated patients (n=65) compared to chemotherapy (n=66), including a:
- 44% reduction in the risk of death (HR: 0.56; 95% CI: 0.36-0.85; nominal one-sided p<0.005).
- 9% reduction in the risk of disease progression (HR: 0.91; 95% CI: 0.62-1.34).
- 12% ORR (8 patients; 95% CI: 6-23%), compared to 5% with chemotherapy (3 patients; 95% CI: 1-13%).
About the Phase 3 Trial
The Phase 3, open-label, multi-center trial is the largest randomized clinical trial in advanced cervical cancer, and investigated Libtayo monotherapy versus an investigator's choice of chemotherapy in patients with recurrent or metastatic cervical cancer that has progressed on platinum-based chemotherapy. Patients were allowed to enroll regardless of PD-L1 expression status, with 78% of patients having SCC and 22% having adenocarcinoma or adenosquamous carcinoma. The trial included women from 14 countries: the U.S., Japan, Taiwan, South Korea, Canada, Russia, Poland, Spain, Brazil, Australia, the UK, Italy, Greece and Belgium.
Patients (median age: 51 years) were randomized to receive Libtayo monotherapy (350 mg every three weeks) or an investigator's choice of commonly used chemotherapy (pemetrexed, vinorelbine, topotecan, irinotecan or gemcitabine). The primary endpoint for the trial was OS, analyzed first among patients with SCC, then in the total population.
In March, the trial was stopped early based on the highly significant effect of Libtayo on OS among SCC patients and following a unanimous recommendation by the Independent Data Monitoring Committee.
About Libtayo
Libtayo is a fully human monoclonal antibody targeting the immune checkpoint receptor PD-1 on T-cells. By binding to PD-1, Libtayo has been shown to block cancer cells from using the PD-1 pathway to suppress T-cell activation.
In the U.S., Libtayo is approved for certain patients with advanced stages of cutaneous squamous cell carcinoma (CSCC), basal cell carcinoma (BCC) and non-small cell lung cancer (NSCLC) with ≥50% PD-L1 expression. Outside of the U.S., Libtayo is approved for certain patients with advanced CSCC in the European Union and six other countries, including Australia, Brazil, the UK and Canada.
The generic name for Libtayo in its approved U.S. indications is cemiplimab-rwlc, with rwlc as the suffix designated in accordance with Nonproprietary Naming of Biological Products Guidance for Industry issued by the U.S. Food and Drug Administration (FDA). Outside of the U.S., the generic name for Libtayo in its approved indication is cemiplimab.
What is Libtayo?
Libtayo is a prescription medicine used to treat people with a type of skin cancer called cutaneous squamous cell carcinoma (CSCC) that has spread or cannot be cured by surgery or radiation.
Libtayo is a prescription medicine used to treat people with a type of skin cancer called basal cell carcinoma that cannot be removed by surgery (locally advanced BCC) and have received treatment with an HHI, or cannot receive treatment with an HHI.
Libtayo is a prescription medicine used to treat people with a type of skin cancer called basal cell carcinoma that has spread (metastatic BCC) and have received treatment with a hedgehog pathway inhibitor (HHI), or cannot receive treatment with an HHI. This use is approved based on how many patients responded to treatment and how long they responded. Studies are ongoing to provide additional information about clinical benefit.
Libtayo is a prescription medicine used to treat people with a type of lung cancer called non-small cell lung cancer (NSCLC). Libtayo may be used as your first treatment when your lung cancer has not spread outside your chest (locally advanced lung cancer) and you cannot have surgery or chemotherapy with radiation, or your lung cancer has spread to other areas of your body (metastatic lung cancer), and your tumor tests positive for high "PD-L1" and your tumor does not have an abnormal "EGFR", "ALK "or "ROS1" gene.
It is not known if Libtayo is safe and effective in children.
What is the most important information I should know about Libtayo?
Libtayo is a medicine that may treat certain cancers by working with your immune system. Libtayo can cause your immune system to attack normal organs and tissues in any area of your body and can affect the way they work. These problems can sometimes become severe or life-threatening and can lead to death. You can have more than one of these problems at the same time. These problems may happen anytime during treatment or even after your treatment has ended.
Please see full Prescribing Information, including Medication Guide.
For additional information about the company, please visit www.regeneron.com
View original content:http://www.prnewswire.com/news-releases/positive-phase-3-libtayo-cemiplimab-results-in-advanced-cervical-cancer-presented-at-esmo-virtual-plenary-301290046.html
SOURCE Regeneron Pharmaceuticals, Inc.
Regeneron and Sanofi updates data from late-stage trial for Libtayo in cervical cancer
May 12, 2021 2:32 PM ETRegeneron Pharmaceuticals, Inc. (REGN)By: Dulan Lokuwithana, SA News Editor
- Regeneron Pharmaceuticals (REGN +1.6%) and Sanofi (SNY -0.4%) shared updated results from the Phase 3 trial evaluating PD-1 inhibitor Libtayo (cemiplimab) as second-line therapy in patients with recurrent or metastatic cervical cancer.
- https://seekingalpha.com/news/3695433-regeneron-and-sanofi-updates-data-from-late-stage-trial-for-libtayo-in-cervical-cancer
- https://seekingalpha.com/symbol/SNY
- https://seekingalpha.com/symbol/REGN
- https://www.regeneron.com/
(NanoFlu™) and COVID-19 vaccine candidate (NVX-CoV2373)
Novavax Announces Positive Preclinical Data for Combination Influenza and COVID-19 Vaccine Candidate
May 10, 2021 at 9:12 AM EDTDownload PDF
- Manuscript highlights development of robust responses to both influenza and COVID-19 and protection against the SARS-CoV-2 virus
- Data shared via preprint server for biology, bioRxiv, ahead of publication
GAITHERSBURG, Md., May 10, 2021 /PRNewswire/ -- Novavax, Inc. (Nasdaq: NVAX), a biotechnology company developing next-generation vaccines for serious infectious diseases, today announced data from a preclinical study of the company's combination quadrivalent seasonal flu vaccine (NanoFlu™) and COVID-19 vaccine candidate (NVX-CoV2373). The NanoFlu/NVX-CoV2373 combination vaccine demonstrated positive immune responses to both influenza and SARS-CoV-2. A pre-print of the manuscript is available at bioRxiv.org.
The manuscript, titled 'Combination Respiratory Vaccine Containing Recombinant SARS-CoV-2 Spike and Quadrivalent Seasonal Influenza Hemagglutinin Nanoparticles with Matrix-M™ Adjuvant,' studied a combination vaccine comprising a quadrivalent nanoparticle influenza vaccine formulated together with a recombinant SARS-CoV-2 spike protein vaccine and Matrix-M™ adjuvant. The combination vaccine elicited robust responses to both influenza A and B and protected against the SARS-CoV-2 virus. Clinical studies of the combination vaccine are expected to begin by the end of the year.
Immunogenicity Results
The preclinical study found that the combination NanoFlu/NVX-CoV2373 (qNIV/CoV2373) vaccine induced functional influenza and COVID antibodies in ferrets. Hemagglutination inhibition (HAI) and ACE2 receptor-inhibiting titers were comparable between immunization with the combination vaccine and with its respective component vaccines. Antibody titers were elevated two weeks after a single dose and increased even further two weeks following a second immunization.
Hamsters that received the combination NanoFlu/NVX-CoV2373 vaccine had elevated levels of SARS-CoV-2 anti-S IgG two weeks after the first immunization, which increased significantly after a second dose, with levels comparable to animals that received the NVX-CoV2373 vaccine alone. Human ACE2 receptor inhibiting antibody levels responded similarly. The immune responses to influenza A and B strains elicited by NanoFlu/NVX-CoV2373 were comparable to immunization with NanoFlu alone. Further, the combination vaccine induced antibodies against SARS-CoV-2 neutralizing epitopes, including at hidden or cryptic sites, that are common between USA-WA1 and the B.1.351 variant.
About NVX-CoV2373
NVX-CoV2373 is a protein-based vaccine candidate engineered from the genetic sequence of the first strain of SARS-CoV-2, the virus that causes COVID-19 disease. NVX-CoV2373 was created using Novavax' recombinant nanoparticle technology to generate antigen derived from the coronavirus spike (S) protein and is adjuvanted with Novavax' patented saponin-based Matrix-M™ to enhance the immune response and stimulate high levels of neutralizing antibodies. NVX-CoV2373 contains purified protein antigen and can neither replicate, nor can it cause COVID-19. In preclinical studies, NVX-CoV2373 induced antibodies that blocked the binding of spike protein to cellular receptors and provided protection from infection and disease. It was generally well-tolerated and elicited robust antibody response in Phase 1/2 clinical testing.
NVX-CoV2373 is being evaluated in two pivotal Phase 3 trials: a trial in the U.K. that demonstrated 100% protection against severe disease, efficacy of 96.4% against the original virus strain, 86.3% against the B.1.1.7/501Y.V1 variant and 89.7% overall; and the PREVENT-19 trial in the U.S. and Mexico that began in December 2020. It is also being tested in two ongoing Phase 2 studies that began in August 2020: A Phase 2b trial in South Africa that demonstrated 100% protection against severe disease and 48.6% efficacy against a newly emerging escape variant first described in South Africa, and a Phase 1/2 continuation in the U.S. and Australia.
NVX-CoV2373 is stored and stable at 2°- 8°C, allowing the use of existing vaccine supply chain channels for its distribution. It is packaged in a ready-to-use liquid formulation in 10-dose vials.
About Matrix-M™
Novavax' patented saponin-based Matrix-M™ adjuvant has demonstrated a potent and well-tolerated effect by stimulating the entry of antigen presenting cells into the injection site and enhancing antigen presentation in local lymph nodes, boosting immune response.
About NanoFlu™
NanoFlu™ is a recombinant hemagglutinin (HA) protein nanoparticle influenza vaccine produced by Novavax in its SF9 insect cell baculovirus system. NanoFlu uses HA amino acid protein sequences that are the same as the recommended wild-type circulating virus HA sequences. NanoFlu contains Novavax' patented saponin-based Matrix-M™ adjuvant.
For more information, visit www.novavax.com
View original content to download multimedia:http://www.prnewswire.com/news-releases/novavax-announces-positive-preclinical-data-for-combination-influenza-and-covid-19-vaccine-candidate-301287543.html
SOURCE Novavax, Inc.
Novavax reports positive preclinical data for influenza and COVID-19 vaccine combo
May 10, 2021 9:29 AM ET Novavax, Inc. (NVAX)
By: Mamta Mayani, SA News Editor
- Novavax (NASDAQ:NVAX) announces data from a preclinical study of its combination quadrivalent seasonal flu vaccine (NanoFlu) and COVID-19 vaccine candidate (NVX-CoV2373).
- https://seekingalpha.com/news/3693852-novavax-reports-positive-preclinical-data-for-influenza-and-covid-19-vaccine-combo
- https://seekingalpha.com/symbol/NVAX
- https://www.novavax.com/our-pipeline#nanoflu
COVID-19 Vaccine
PFIZER AND BIONTECH RECEIVE FIRST U.S. AUTHORIZATION FOR EMERGENCY USE OF COVID-19 VACCINE IN ADOLESCENTS
Monday, May 10, 2021 - 09:02pm
- In a Phase 3 trial, the vaccine was 100% effective and generally well tolerated in participants aged 12 to 15 years
- Data also submitted to European Medicines Agency (EMA) and other global regulators, with additional authorizations expected in coming weeks
NEW YORK & MAINZ, Germany--(BUSINESS WIRE)-- Pfizer Inc. (NYSE: PFE) and BioNTech SE (Nasdaq: BNTX) announced today that the U.S. Food and Drug Administration (FDA) has expanded the Emergency Use Authorization (EUA) for their COVID-19 vaccine to include individuals 12 to 15 years of age. This is the first COVID-19 vaccine authorized in the U.S. for use in this age group.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210510006021/en/
The Pfizer-BioNTech COVID-19 Vaccine has not been approved or licensed by the U.S. Food and Drug Administration (FDA), but has been authorized for emergency use by FDA under an Emergency Use Authorization (EUA) to prevent Coronavirus Disease 2019 (COVID-19) caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) for use in individuals 12 years of age and older. The emergency use of this product is only authorized for the duration of the declaration that circumstances exist justifying the authorization of emergency use of the medical product under Section 564 (b) (1) of the FD&C Act unless the declaration is terminated or authorization revoked sooner. Please see Emergency Use Authorization (EUA) Fact Sheet for Healthcare Providers Administering Vaccine (Vaccination Providers) and Full EUA Prescribing Information available at www.cvdvaccine-us.com.
AUTHORIZED USE IN THE U.S.:
The Pfizer-BioNTech COVID19 Vaccine is authorized for use under an Emergency Use Authorization (EUA) for active immunization to prevent coronavirus disease 2019 (COVID-19) caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) in individuals 12 years of age and older.
In addition, to learn more, please visit us on www.Pfizer.com
For more information, please visit www.BioNTech.de.
Before administration of Pfizer-BioNTech COVID-19 Vaccine, please see Emergency Use Authorization (EUA) Fact Sheet for Healthcare Providers Administering Vaccine (Vaccination Providers) including Full EUA Prescribing Information available at www.cvdvaccine-us.com
Pfizer/ BioNTech COVID-19 vaccine authorized for adolescents in the U.S.
May 10, 2021 9:54 PM ET Pfizer Inc. (PFE)
By: Dulan Lokuwithana, SA News Editor22 Comments
- The FDA has expanded the emergency use authorization of the COVID-19 vaccine developed by Pfizer (NYSE:PFE) and BioNTech (NASDAQ:BNTX) for use in adolescents aged 12 to 15 years of age.
- https://seekingalpha.com/news/3694355-pfizer-biontech-covid-19-vaccine-authorized-for-adolescents-in-the-us
- https://seekingalpha.com/symbol/BNTX
- https://seekingalpha.com/symbol/PFE
- https://www.pfizer.com/
05.10.2021 - Pfizer and BioNTech Receive First U.S. Authorization for Emergency Use of COVID-19 Vaccine in Adolescents
05.06.2021 - Pfizer and BioNTech to Provide COVID-19 Vaccine Doses for Olympic Athletes at the 2020 Tokyo Games
Extending availability to children is considered key to achieving herd immunity but could set off debates about vaccine mandates in schools.By Carolyn Y. Johnson
- Maker of latest experimental vaccine will not seek authorization until July at the earliest
- Tracking the coronavirus vaccine
KEYTRUDA® (pembrolizumab) Plus LENVIMA® (lenvatinib)
Merck and Eisai Receive Priority Review From FDA for KEYTRUDA® (pembrolizumab) Plus LENVIMA® (lenvatinib) Applications for Advanced Renal Cell Carcinoma and for Advanced Endometrial Carcinoma
May 6, 2021 6:30 am ET
Applications Based on Progression-Free Survival, Overall Survival, and Objective Response Rate Data From Respective Pivotal Phase 3 Trials
KENILWORTH, N.J. & WOODCLIFF LAKE, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, and Eisai Inc. today announced that the U.S. Food and Drug Administration (FDA) has accepted and granted priority review for applications seeking two new approvals for the combination of KEYTRUDA, Merck’s anti-PD-1 therapy, plus LENVIMA, the orally available multiple receptor tyrosine kinase inhibitor discovered by Eisai. The first set of applications (a supplemental Biologics License Application [sBLA] for KEYTRUDA and a supplemental New Drug Application [sNDA] for LENVIMA) are for the first-line treatment of patients with advanced renal cell carcinoma (RCC), based on progression-free survival (PFS), overall survival (OS) and objective response rate (ORR) data from the pivotal Phase 3 CLEAR study (KEYNOTE-581/Study 307). The second set of applications are for the treatment of patients with advanced endometrial carcinoma who have disease progression following prior systemic therapy in any setting and are not candidates for curative surgery or radiation, based on PFS, OS and ORR data from the pivotal Phase 3 KEYNOTE-775/Study 309 trial. These are the first applications to be submitted in the U.S. for this combination therapy based on Phase 3 clinical data. The FDA has set Prescription Drug User Fee Act (PDUFA) dates, or target action dates, of August 25 and 26, 2021, for the advanced RCC sNDA and sBLA applications, respectively, and September 3, 2021, for the advanced endometrial carcinoma applications.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210506005287/en/
About CLEAR Study (KEYNOTE-581/Study 307)
The CLEAR study (KEYNOTE-581/Study 307) is a multicenter, randomized, open-label, Phase 3 trial (ClinicalTrials.gov, NCT02811861) evaluating LENVIMA in combination with KEYTRUDA or in combination with everolimus versus sunitinib for the first-line treatment of patients with advanced RCC. The primary endpoint is PFS, as assessed by independent review per Response Evaluation Criteria in Solid Tumors (RECIST) v1.1. Key secondary endpoints include OS, ORR and safety. A total of 1,069 patients were randomized (1:1:1) to receive:
- LENVIMA (20 mg orally once daily) in combination with KEYTRUDA (200 mg intravenously [IV] every three weeks for up to 24 months; or
- LENVIMA (18 mg orally once daily) in combination with everolimus (5 mg orally once daily); or
- Sunitinib (50 mg orally once daily for four weeks on treatment, followed by two weeks off treatment).
About KEYNOTE-775/Study 309
KEYNOTE-775/Study 309 is a multicenter, randomized, open-label, Phase 3 trial (ClinicalTrials.gov, NCT03517449) evaluating KEYTRUDA in combination with LENVIMA in patients with advanced endometrial carcinoma who had been previously treated with at least one prior platinum-based chemotherapy regimen in any setting, including in the neoadjuvant and adjuvant settings. The dual primary endpoints are PFS, as assessed by blinded independent central review (BICR) per RECIST v1.1, and OS. Select secondary endpoints include ORR and duration of response (DOR), as assessed by BICR. A total of 827 patients were randomized (1:1) to receive:
- KEYTRUDA (200 mg IV every three weeks) in combination with LENVIMA (20 mg orally once daily); or
- Investigator’s choice of either doxorubicin (60 mg/m2 IV every three weeks) or paclitaxel (80 mg/m2 IV given weekly, three weeks of receiving weekly paclitaxel and one week of not receiving paclitaxel).
About KEYTRUDA® (pembrolizumab) Injection, 100 mg
KEYTRUDA is an anti-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 1,400 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.
Selected KEYTRUDA® (pembrolizumab) Indications in the U.S.
Melanoma
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.
KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.
Non-Small Cell Lung Cancer
KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) ≥1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is stage III where patients are not candidates for surgical resection or definitive chemoradiation, or metastatic.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS ≥1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.
Head and Neck Squamous Cell Cancer
KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS) ≥1] as determined by an FDA-approved test.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.
Classical Hodgkin Lymphoma
KEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).
KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.
Primary Mediastinal Large B-Cell Lymphoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.
Urothelial Carcinoma
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 (CPS ≥10), as determined by an FDA-approved test, or in patients who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.
Microsatellite Instability-High or Mismatch Repair Deficient Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR)
- solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options, or
- colorectal cancer that has progressed following treatment with fluoropyrimidine, oxaliplatin, and irinotecan.
This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.
Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer
KEYTRUDA is indicated for the first-line treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).
Gastric Carcinoma
KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test, with disease progression on or after two or more prior lines of therapy including fluoropyrimidine-and platinum-containing chemotherapy and if appropriate, HER2/neu-targeted therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Esophageal Carcinoma
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic esophageal or gastroesophageal junction (GEJ) (tumors with epicenter 1 to 5 centimeters above the GEJ) carcinoma that is not amenable to surgical resection or definitive chemoradiation either:
- in combination with platinum- and fluoropyrimidine-based chemotherapy, or
- as a single agent after one or more prior lines of systemic therapy for patients with tumors of squamous cell histology that express PD L1 (CPS ≥10) as determined by an FDA approved test.
Cervical Carcinoma
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Hepatocellular Carcinoma
KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Merkel Cell Carcinoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Renal Cell Carcinoma
KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).
Endometrial Carcinoma
KEYTRUDA, in combination with LENVIMA, is indicated for the treatment of patients with advanced endometrial carcinoma that is not MSI-H or dMMR, who have disease progression following prior systemic therapy and are not candidates for curative surgery or radiation. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trial.
Tumor Mutational Burden-High
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [≥10 mutations/megabase] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.
Cutaneous Squamous Cell Carcinoma
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) that is not curable by surgery or radiation.
Triple-Negative Breast Cancer
KEYTRUDA, in combination with chemotherapy, is indicated for the treatment of patients with locally recurrent unresectable or metastatic triple-negative breast cancer (TNBC) whose tumors express PD-L1 (CPS ≥10) as determined by an FDA-approved test. This indication is approved under accelerated approval based on progression-free survival. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Please see Prescribing Information for KEYTRUDA (pembrolizumab) at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_pi.pdf and Medication Guide for KEYTRUDA at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_mg.pdf.
About LENVIMA® (lenvatinib) Capsules
LENVIMA® (lenvatinib) is a kinase inhibitor that is indicated:
- For the treatment of patients with locally recurrent or metastatic, progressive, radioactive iodine-refractory differentiated thyroid cancer (RAI-refractory DTC)
- In combination with everolimus, for the treatment of patients with advanced renal cell carcinoma (RCC) following one prior anti-angiogenic therapy
- For the first-line treatment of patients with unresectable hepatocellular carcinoma (HCC)
- In combination with KEYTRUDA, for the treatment of patients with advanced endometrial carcinoma (EC) that is not microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR), who have disease progression following prior systemic therapy, and are not candidates for curative surgery or radiation. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trial
LENVIMA, discovered and developed by Eisai, is a kinase inhibitor that inhibits the kinase activities of vascular endothelial growth factor (VEGF) receptors VEGFR1 (FLT1), VEGFR2 (KDR), and VEGFR3 (FLT4). LENVIMA inhibits other kinases that have been implicated in pathogenic angiogenesis, tumor growth, and cancer progression in addition to their normal cellular functions, including fibroblast growth factor (FGF) receptors FGFR1-4, the platelet derived growth factor receptor alpha (PDGFRα), KIT, and RET. The combination of lenvatinib and everolimus showed increased anti-angiogenic and anti-tumor activity as demonstrated by decreased human endothelial cell proliferation, tube formation, and VEGF signaling in vitro and tumor volume in mouse xenograft models of human renal cell cancer greater than each drug alone. In syngeneic mouse tumor models, lenvatinib decreased tumor-associated macrophages, increased activated cytotoxic T cells, and demonstrated greater antitumor activity in combination with an anti-PD-1 monoclonal antibody compared to either treatment alone.
Please see Prescribing Information for LENVIMA (lenvatinib) at http://www.lenvima.com/pdfs/prescribing-information.pdf.
For more information about our oncology clinical trials, visit www.merck.com/clinicaltrials.
For more information, visit www.merck.com
For more information about Eisai, please visit www.eisai.com (for global), us.eisai.com (for U.S.) or www.eisai.eu (for Europe, Middle East, Africa)
FDA grants accelerated review to Merck, Eisai's Keytruda + Lenvima applications in kidney and endometrial cancer
May 06, 2021 9:07 AM ET Merck & Co., Inc. (MRK) By: Mamta Mayani, SA News Editor1 Comment
- Under priority review, the FDA has accepted Merck (NYSE:MRK) and Eisai's (OTCPK:ESALF) applications seeking two new approvals for the combination of Keytruda plus Lenvima, the orally available multiple receptor tyrosine kinase inhibitor discovered by Eisai.
- https://seekingalpha.com/news/3692263-fda-grants-accelerated-review-to-merck-eisais-keytruda-lenvima-applications-in-kidney-and-endometrial-cancer
- https://seekingalpha.com/symbol/ESALF
- https://seekingalpha.com/symbol/MRK
- https://www.keytruda.com/
- http://www.lenvima.com/
Vidutolimod (CMP-001) in Combination with Libtayo® (cemiplimab)
Checkmate Pharmaceuticals Announces Clinical Supply Agreement with Regeneron to Evaluate Vidutolimod (CMP-001) in Combination with Libtayo® (cemiplimab)
May 10, 2021 at 6:00 AM EDTDownload PDF
CAMBRIDGE, Mass., May 10, 2021 (GLOBE NEWSWIRE) -- Checkmate Pharmaceuticals, Inc. (NASDAQ: CMPI) (“Checkmate”), a clinical stage biopharmaceutical company focused on developing its proprietary technology to harness the power of the immune system to combat cancer, today announced the development program expansion of vidutolimod (CMP-001) into non-melanoma skin cancers in combination with Libtayo® (cemiplimab) under a clinical supply agreement with Regeneron Pharmaceuticals, Inc. (“Regeneron”). Vidutolimod is an advanced generation Toll-like receptor 9 (TLR9) agonist, delivered as a biologic virus-like particle utilizing a CpG-A oligodeoxynucleotide as a key component. Cemiplimab is a PD-1 blocking antibody being jointly developed by Regeneron and Sanofi.
Checkmate and Regeneron will collaborate on a multi-indication, Phase 2, proof-of-concept clinical trial of vidutolimod in combination with cemiplimab in the following patient cohorts: (a) PD-1 treatment-naïve subjects with cutaneous squamous cell carcinoma (CSCC), (b) subjects with cutaneous squamous cell carcinoma (CSCC) that is refractory to PD-1 blockade, and (c) subjects with Merkel cell carcinoma (MCC) that is refractory to PD-1 blockade. Checkmate will be the sponsor of the clinical trial, and Regeneron will supply cemiplimab.
Vidutolimod (CMP-001) in Combination with Libtayo® (cemiplimab)
“We’re pleased to collaborate with Regeneron as we expand evaluation of vidutolimod as a potent stimulator of innate immune activity to patients with life-threatening non-melanoma skin cancers such as CSCC and MCC,” said Barry Labinger, President and Chief Executive Officer of Checkmate. “We look forward to advancing vidutolimod in combination with Libtayo to further unlock the capabilities and impact of immuno-oncology therapeutics.”
Information regarding Checkmate Pharmaceuticals is available at www.checkmatepharma.com.
Checkmate Pharma teams up with Regeneron to evaluate vidutolimod + Libtayo in skin cancer
May 10, 2021 6:52 AM ETCheckmate Pharmaceuticals, Inc. (CMPI)
By: Mamta Mayani, SA News Editor1 Comment
- Checkmate Pharmaceuticals (NASDAQ:CMPI) announces the development program expansion of vidutolimod (CMP-001) into non-melanoma skin cancers in combination with Libtayo (cemiplimab) under a clinical supply agreement with Regeneron Pharmaceuticals (NASDAQ:REGN).
- https://seekingalpha.com/news/3693677-checkmate-pharma-teams-up-with-regeneron-to-evaluate-vidutolimod-libtayo-in-skin-cancer
- https://seekingalpha.com/symbol/CMPI
- https://seekingalpha.com/symbol/REGN
- https://checkmatepharma.com/
- https://www.libtayohcp.com/
Pamiparib (BGB-290)
China NMPA Approves PARP Inhibitor Pamiparib for Patients with Previously Treated Advanced Ovarian Cancer
May 7, 2021 at 12:00 AM EDT
Pamiparib becomes the first PARP inhibitor approved in both platinum-sensitive and platinum-resistant relapsed ovarian cancer in China
This marks the first approval of pamiparib and the third BeiGene-discovered medicine to receive regulatory approval
BEIJING & CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 7, 2021-- BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a global biotechnology company focused on developing and commercializing innovative medicines worldwide, today announced that its PARP inhibitor pamiparib has received conditional approval from the China National Medical Products Administration (NMPA) for the treatment of patients with germline BRCA (gBRCA) mutation-associated recurrent advanced ovarian, fallopian tube, or primary peritoneal cancer who have been treated with two or more lines of chemotherapy. The new drug application was previously granted priority review by the Center for Drug Evaluation (CDE) in July 2020. BeiGene is preparing to launch pamiparib this month.
About Pamiparib
Pamiparib is an inhibitor of PARP1 and PARP2 which has demonstrated pharmacological properties such as brain penetration and PARP-DNA complex trapping in preclinical models. Discovered by BeiGene scientists, pamiparib is currently in global clinical development as a monotherapy or in combination with other agents for a variety of solid tumor malignancies. To date, more than 1,200 patients have been enrolled in clinical trials of pamiparib.
In China, pamiparib received conditional approval for treatment of patients with germline BRCA (gBRCA) mutation-associated recurrent advanced ovarian, fallopian tube, or primary peritoneal cancer who have been treated with two or more lines of chemotherapy in May 2021. Full approval for this indication is contingent upon results from ongoing corroborative trials confirming the clinical benefit of pamiparib in this population.
About the Pamiparib Clinical Program
Clinical trials of pamiparib include:
- Phase 3 trial in China of pamiparib as maintenance versus placebo in patients with platinum-sensitive recurrent ovarian cancer (NCT03519230);
- Phase 2 trial of pamiparib in patients with metastatic castration-resistant prostate cancer with homologous recombination deficiency (NCT03712930);
- Phase 2 trial in China of pamiparib in patients with metastatic HER2-negative breast cancer with BRCA mutation (NCT03575065);
- Phase 2 trial of pamiparib in patients with advanced or inoperable gastric cancer (NCT03427814);
- Phase 1/2 trial in China of pamiparib in patients with advanced ovarian cancer, fallopian cancer, and primary peritoneal cancer or advanced triple negative breast cancer (NCT03333915);
- Phase 1b/2 trial of pamiparib in combination with radiation therapy and/or temozolomide in patients with first-line or recurrent/refractory glioblastoma (NCT03150862);
- Phase 1b trial of pamiparib in combination with temozolomide in patients with locally advanced or metastatic solid tumors (NCT03150810); and
- Phase 1b trial of pamiparib in combination with tislelizumab for a variety of solid tumor malignancies (NCT02660034).
To learn more about BeiGene, please visit www.beigene.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20210506006335/en/
BeiGene's pamiparib OK'd in China for ovarian cancer
May 07, 2021 6:35 AM ET BeiGene, Ltd. (BGNE)
By: Mamta Mayani, SA News Editor
- BeiGene (NASDAQ:BGNE) announces that its PARP inhibitor pamiparib has received conditional approval from the China National Medical Products Administration (NMPA) for the treatment of patients with germline BRCA (gBRCA) mutation-associated recurrent advanced ovarian, fallopian tube, or primary peritoneal cancer who have been treated with two or more lines of chemotherapy.
- https://seekingalpha.com/news/3693008-beigenes-pamiparib-okd-in-china-for-ovarian-cancer
- https://seekingalpha.com/symbol/BGNE
- https://www.beigene.com/?loc=us
Farxiga (dapagliflozin)
Farxiga approved in the US for the treatment of chronic kidney disease in patients at risk of progression with and without type-2 diabetes
PUBLISHED30 April 2021
30 April 2021 22:00 BST
Approval is the most significant advancement in the treatment
of chronic kidney disease in more than 20 years
In DAPA-CKD Phase III trial, Farxiga demonstrated unprecedented reduction in the risk of the composite of worsening of renal function, end-stage kidney disease and cardiovascular or renal death
AstraZeneca’s Farxiga (dapagliflozin), a sodium-glucose cotransporter 2 (SGLT2) inhibitor, has been approved in the US to reduce the risk of sustained estimated glomerular filtration rate (eGFR) decline, end-stage kidney disease (ESKD), cardiovascular (CV) death and hospitalisation for heart failure (hHF) in adults with chronic kidney disease (CKD) at risk of progression.
The approval by the Food and Drug Administration (FDA) was based on positive results from the DAPA-CKD Phase III trial. The decision follows the Priority Review designation granted by the FDA earlier this year.
CKD, a condition defined by decreased kidney function, is often associated with a heightened risk of heart disease or stroke, or the need for dialysis or kidney transplant.1-3 CKD is expected to become the fifth leading cause of mortality globally by 2040.4 Currently in the US, 37 million people are estimated to have CKD.1
DAPA-CKD
DAPA-CKD was an international, multi-centre, randomised, double-blinded Phase III trial in 4,304 patients designed to evaluate the efficacy of Farxiga 10mg, compared with placebo, in patients with CKD Stages 2-4 and elevated urinary albumin excretion, with and without T2D. Farxiga was given once daily in addition to standard of care. The primary composite endpoint was worsening of renal function or risk of death (defined as a composite of an eGFR decline ≥50%, onset of ESKD or death from CV or renal cause). The secondary endpoints included the time to first occurrence of the renal composite (sustained ≥50% eGFR decline, ESKD or renal death), the composite of CV death or hHF, and death from any cause. The trial was conducted in 21 countries.9 Detailed results from the trial were published in The New England Journal of Medicine.9
DECLARE-TIMI 58
DECLARE-TIMI 58 was an AstraZeneca-sponsored, randomised, double-blinded, multi-centre Phase III trial designed to evaluate the effect of Farxiga compared with placebo on CV outcomes in adults with T2D at risk of CV events, including patients with multiple CV risk factors or established CV disease, and also assessed key renal exploratory endpoints. The trial included more than 17,000 patients across 882 sites in 33 countries and was independently conducted in collaboration with academic investigators from the TIMI study group (Boston, US) and the Hadassah Hebrew University Medical Center (Jerusalem, Israel). Results from the trial were published in The Lancet.10
Farxiga
Farxiga (dapagliflozin) is a first-in-class, oral, once-daily SGLT2 inhibitor. The research for Farxiga is advancing from cardiorenal effects to prevention and organ protection as science continues to identify the underlying links between the heart, kidneys and pancreas. Damage to one of these organs can cause the other organs to fail - contributing to leading causes of death worldwide, including T2D, HF and CKD.
For nearly a decade Farxiga has been an effective monotherapy and part of combination therapy as an adjunct to diet and exercise to improve glycaemic control in adults with T2D. Following results from the landmark DECLARE-TIMI 58 Phase III CV outcomes trial, it is approved in adults with T2D to reduce the risk of hHF or CV death when added to standard of care.11 Farxiga is also the first SGLT2 inhibitor approved for the treatment of HFrEF in adults with and without T2D.
In August 2020, results from the DAPA-CKD Phase III trial demonstrated that Farxiga achieved unprecedented reduction in the composite risk of kidney failure and CV or renal death in patients with CKD with and without T2D versus placebo.9 It is now the first SGLT2 inhibitor shown to significantly improve overall survival in a renal outcomes trial for this patient population and provide organ protection.
DapaCare is a robust programme of clinical trials to evaluate the potential CV, renal and organ protection benefits of Farxiga. It includes more than 35 completed and ongoing Phase IIb/III trials in more than 35,000 patients, as well as more than 2.5 million patient-years’ experience. It is currently being assessed in patients with HF with preserved ejection fraction in the DELIVER Phase III trial. Farxiga is also being tested in patients without T2D following an acute myocardial infarction (MI) or heart attack in the DAPA-MI Phase III trial - a first of its kind, indication-seeking registry-based randomised controlled trial.
Please visit astrazeneca.com
AstraZeneca wins FDA approval for Farxiga in chronic kidney disease
Apr. 30, 2021 4:57 PM ET AstraZeneca PLC (AZN) By: Dulan Lokuwithana, SA News Editor1 Comment
- The FDA announced the approval of Farxiga (dapagliflozin) oral tablets for adults with chronic kidney disease who are at risk of disease progression.
- https://seekingalpha.com/news/3688945-astrazeneca-wins-fda-approval-for-farxiga-in-chronic-kidney-disease
- https://seekingalpha.com/symbol/AZN
- https://www.farxiga.com/
- https://www.astrazeneca.com/
Otezla® (apremilast)
FDA Accepts Amgen's Supplemental New Drug Application For Otezla® (apremilast) For Adults With Mild-To-Moderate Plaque Psoriasis
Potential to Be First and Only Approved Oral Therapy for the Mild-to-Moderate Patient Population
Acceptance Based on Data From Phase 3 ADVANCE Study, Demonstrating Clinically Meaningful Improvements Through Week 32
FDA Action Date Set for December 19, 2021
THOUSAND OAKS, Calif., May 5, 2021 /PRNewswire/ -- Amgen (NASDAQ:AMGN) today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the supplemental New Drug Application (sNDA) for Otezla® (apremilast) for the treatment of adults with mild-to-moderate plaque psoriasis who are candidates for phototherapy or systemic therapy. The FDA has set a Prescription Drug User Fee Act (PDUFA) action date of December 19, 2021.
"Otezla has been prescribed to hundreds of thousands of patients with moderate-to-severe plaque psoriasis. Based on the positive Phase 3 ADVANCE data, we believe it could play an important role in addressing the unmet need for adults affected by mild-to-moderate plaque psoriasis who have had challenges managing their disease with existing topical therapies alone," said David M. Reese, M.D., executive vice president of Research and Development at Amgen. "The FDA's acceptance of this sNDA for Otezla is a significant milestone toward achieving our goal of potentially providing the first and only oral treatment option for the underserved mild-to-moderate patient population."
About ADVANCE (PSOR-022)
ADVANCE (NCT03721172) is a Phase 3, multicenter, randomized, placebo-controlled, double-blind study evaluating the efficacy and safety of Otezla in patients with mild-to-moderate plaque psoriasis (defined as BSA involvement of 2% to 15%, Psoriasis Area and Severity Index (PASI) score of 2 to 15, sPGA score of 2 to 3). The study randomized 595 patients 1:1 to receive Otezla (n=297) 30 mg twice daily or placebo (n=298) for the first 16 weeks. All patients then received Otezla during an open-label extension phase through week 32.
The primary endpoint was the percentage of patients with sPGA response [defined as a sPGA score of clear (0) or almost clear (1) with at least a 2-point reduction from baseline] at week 16.
About Otezla® (apremilast)
OTEZLA® (apremilast) is an oral small-molecule inhibitor of phosphodiesterase 4 (PDE4) specific for cyclic adenosine monophosphate (cAMP). PDE4 inhibition results in increased intracellular cAMP levels, which is thought to indirectly modulate the production of inflammatory mediators. The specific mechanism(s) by which Otezla exerts its therapeutic action in patients is not well defined.
Otezla® (apremilast) U.S. INDICATIONS
Otezla® (apremilast) is indicated for the treatment of adult patients with moderate to severe plaque psoriasis who are candidates for phototherapy or systemic therapy.
Otezla is indicated for the treatment of adult patients with active psoriatic arthritis.
Otezla is indicated for the treatment of adult patients with oral ulcers associated with Behçet's Disease.
Otezla® (apremilast) U.S. IMPORTANT SAFETY INFORMATION
Contraindications
- Otezla® (apremilast) is contraindicated in patients with a known hypersensitivity to apremilast or to any of the excipients in the formulation
Please click here for Otezla® Full Prescribing Information.
For more information, visit www.amgen.com and follow us on www.twitter.com/amgen.
View original content to download multimedia:http://www.prnewswire.com/news-releases/fda-accepts-amgens-supplemental-new-drug-application-for-otezla-apremilast-for-adults-with-mild-to-moderate-plaque-psoriasis-301283931.html
SOURCE Amgen
FDA accepts Amgen's Otezla application in plaque psoriasis
May 05, 2021 9:24 AM ET Amgen Inc. (AMGN)
By: Mamta Mayani, SA News Editor
- Amgen (NASDAQ:AMGN) announces that the FDA has accepted for review the supplemental New Drug Application (sNDA) for Otezla (apremilast) for the treatment of adults with mild-to-moderate plaque psoriasis who are candidates for phttps://seekingalpha.com/news/3691096-fda-accepts-amgens-otezla-application-in-plaque-psoriasishototherapy or systemic therapy.
- https://seekingalpha.com/symbol/AMGN
- https://www.amgen.com/
- https://www.otezla.com/
ORLADEYO™ (berotralstat)
BioCryst Receives European Commission Approval of ORLADEYO™ (berotralstat), First Oral, Once-daily Therapy to Prevent Attacks in Hereditary Angioedema Patients
RESEARCH TRIANGLE PARK, N.C., April 30, 2021 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the European Commission (EC) has approved oral, once-daily ORLADEYO™ (berotralstat) for the prevention of recurrent hereditary angioedema (HAE) attacks in HAE patients 12 years and older.
“As the first targeted oral prophylactic therapy approved in Europe, ORLADEYO represents a major advance in treatment for HAE patients who have been waiting for a preventive therapy. Physicians will be delighted to discuss this new option with their patients,” said Emel Aygören-Pürsün, M.D., head of the HAE Center at the University Hospital in Frankfurt.
“ORLADEYO offers people with HAE in Europe and their physicians the first orally administered non-steroidal option for preventing HAE attacks and represents a vitally important and most welcome step in making more treatment options available,” said Henrik Balle Boysen, executive vice president and chief operating officer of HAE International, a global non-profit network of patient associations dedicated to improving the lives of people with HAE.
The EC approval of ORLADEYO is applicable to all European Union member states plus Iceland, Norway and Liechtenstein.
About ORLADEYO™ (berotralstat)
ORLADEYO™ (berotralstat) is the first and only oral therapy designed specifically to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years and older. One capsule of ORLADEYO per day works to prevent HAE attacks by decreasing the activity of plasma kallikrein.
The full European Summary of Product Characteristics (SMPC) for ORLADEYO will be available from the European Medicines Association website at www.ema.europa.eu.
For more information, please visit the company’s website at www.biocryst.com.
BioCryst gets European Commission approval for ORLADEYO
Apr. 30, 2021 9:29 AM ET BioCryst Pharmaceuticals, Inc. (BCRX)
By: Aakash Babu, SA News Editor1 Comment
- BioCryst Pharmaceuticals (NASDAQ:BCRX) announces that the European Commission (EC) has approved oral, once-daily ORLADEYO (berotralstat) for the prevention of recurrent hereditary angioedema (HAE) attacks in HAE patients 12 years and older.
- https://seekingalpha.com/news/3688681-biocryst-gets-european-commission-approval-of-orladeyo
- https://seekingalpha.com/symbol/BCRX
- https://orladeyo.com/
- https://ir.biocryst.com/
XTANDI™ (enzalutamide)
Astellas' XTANDI™ (enzalutamide) Approved by European Commission for Men with Metastatic Hormone-Sensitive Prostate CancerEnzalutamide is now the only oral therapy approved by the European Commission to treat three distinct types of advanced prostate cancer
TOKYO, May 4, 2021 /PRNewswire/ -- Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., "Astellas") announced today that the European Commission (EC) has approved an additional indication for the oral once-daily therapy XTANDI™ (enzalutamide) for adult men with metastatic hormone-sensitive prostate cancer (mHSPC, also known as metastatic castration-sensitive prostate cancer or mCSPC). Men diagnosed with mHSPC tend to have a poor prognosis, with a median survival of approximately 3-4 years,1 underscoring the need for new treatment options.
With this indication, enzalutamide is now the only oral treatment approved by the EC to treat three distinct types of advanced prostate cancer — non-metastatic and metastatic castration-resistant prostate cancer (CRPC) and mHSPC.2 The EC approval is based on results from the pivotal Phase 3 ARCHES trial which evaluated enzalutamide in men with mHSPC.3
XTANDI™ (enzalutamide)
Data from the ARCHES trial showed enzalutamide plus androgen deprivation therapy (ADT) significantly reduced the risk of radiographic progression or death by 61% versus placebo plus ADT in men with mHSPC (n=1,150; hazard ratio [HR]=0.39 [95% confidence interval (CI): 0.30-0.50]; P<0.0001).3
"Enzalutamide has been an established standard of care for men with advanced prostate cancer and has been prescribed to more than 610,000 patients worldwide since it was first approved in 2012," said Andrew Krivoshik, M.D., Ph.D., Senior Vice President and Global Therapeutic Area Head, Oncology Development, Astellas. "This new indication for enzalutamide provides men with mHSPC a much-needed, additional therapy option earlier in their treatment journey. We look forward to working with health authorities across Europe to ensure men with mHSPC have access to enzalutamide as soon as possible."
About XTANDI™ (enzalutamide)
Enzalutamide is an androgen receptor signaling inhibitor indicated in the EU for the treatment of adult men with:2
- Metastatic hormone-sensitive prostate cancer (mHSPC, also known as metastatic castration-sensitive prostate cancer or mCSPC) in combination with ADT.
- High-risk non-metastatic castration-resistant prostate cancer (CRPC).
- Metastatic CRPC who are asymptomatic or mildly symptomatic after failure of androgen deprivation therapy in whom chemotherapy is not yet clinically indicated. It is also indicated in adult men with metastatic CRPC whose disease has progressed on or after docetaxel therapy.
About ARCHES
The company-sponsored, Phase 3, randomized, double-blind, placebo-controlled, multinational ARCHES trial (NCT02677896) enrolled 1,150 patients with mHSPC at sites in the U.S., Canada, Europe, South America, and the Asia-Pacific region. Patients in the trial were randomized to receive enzalutamide 160 mg daily or placebo and continued on a luteinizing hormone-releasing hormone (LHRH) agonist or antagonist or had a history of bilateral orchiectomy. The primary endpoint of the trial was radiographic progression-free survival (rPFS) assessed by blinded independent central review. rPFS was defined as the time from randomization to radiographic disease progression at any time or death within 24 weeks after study drug discontinuation. Radiographic disease progression was defined by identification of two or more new bone lesions on a bone scan with confirmation (Prostate Cancer Working Group 2 criteria) and/or progression in soft tissue disease. Patients were stratified by volume of disease (low vs high) and prior docetaxel therapy for prostate cancer (no prior docetaxel, 1-5 cycles, or 6 prior cycles).3
For more information, please visit our website at https://www.astellas.com/en.
About the Pfizer/Astellas Collaboration
In October 2009, Medivation, Inc., which is now part of Pfizer (NYSE:PFE), and Astellas (TSE: 4503) entered into a global agreement to jointly develop and commercialize enzalutamide. The companies jointly commercialize enzalutamide in the United States and Astellas has responsibility for manufacturing and all additional regulatory filings globally, as well as commercializing enzalutamide outside the United States.
Astellas Pharma's Xtandi OK'd in Europe for prostate cancer
May 04, 2021 2:14 AM ET
By: Mamta Mayani, SA News Editor
- The European Commission (EC) has approved an additional indication for Astellas Pharma's (OTCPK:ALPMF) oral once-daily therapy Xtandi (enzalutamide) for adult men with metastatic hormone-sensitive prostate cancer (mHSPC, also known as metastatic castration-sensitive prostate cancer or mCSPC).
- https://seekingalpha.com/news/3689888-astellas-pharmas-xtandi-okd-in-europe-for-prostate-cancer
- https://seekingalpha.com/symbol/ALPMF
- https://www.astellas.com/us/
- https://seekingalpha.com/symbol/PFE
- https://www.xtandi.com/
BRUKINSA® (Zanubrutinib)
BRUKINSA® (Zanubrutinib) Demonstrates Superior Objective Response Rate by Investigator Assessment and Reduced Rates of Atrial Fibrillation or Flutter at Interim Analysis in Head-to-Head Trial Against Ibrutinib in Chronic Lymphocytic Leukemia
April 28, 2021 at 7:00 AM EDT
CAMBRIDGE, Mass. & BEIJING--(BUSINESS WIRE)--Apr. 28, 2021-- BeiGene (NASDAQ: BGNE; HKEX: 06160) announced positive results from a planned interim analysis of the Phase 3 ALPINE trial comparing BRUKINSA® (zanubrutinib) against ibrutinib in adults with relapsed or refractory (R/R) chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).
BRUKINSA met the primary endpoint of the trial, demonstrating non-inferiority in objective response rate (ORR) by both investigator and independent review committee (IRC) assessments (p < 0.0001). The trial also demonstrated superior ORR with a statistically significant improvement in ORR for BRUKINSA vs. ibrutinib (p = 0.0006) by investigator assessment, as well as a numerically higher ORR but not statistically significant improvement by IRC (p = 0.0121 compared to the two-sided stringent statistical boundary of p < 0.0099 set for the interim analysis). The interim analysis from this fully-enrolled, ongoing trial is based on 415 of 652 patients followed for a minimum of 12 months.
BRUKINSA® (Zanubrutinib)
What is BRUKINSA?
BRUKINSA is a prescription medicine used to treat adults with mantle cell lymphoma (MCL) who have received at least one prior treatment for their cancer.
It is not known if BRUKINSA is safe and effective in children.
Please see full Prescribing Information including Patient Information.
ALPINE is BeiGene’s second Phase 3 head-to-head trial comparing BRUKINSA to ibrutinib.
About ALPINE
ALPINE is a randomized, global Phase 3 trial (NCT03734016) comparing BRUKINSA against ibrutinib in previously treated patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).
In the trial, a total of 652 patients were randomized into two arms with the first receiving BRUKINSA (160 mg orally twice daily) and the second receiving ibrutinib (420 mg orally once daily) until disease progression or unacceptable toxicity. The primary analysis of objective response rate (ORR), defined by pre-specified non-inferiority of BRUKINSA versus ibrutinib, was assessed by investigator and independent review committee (IRC) using the modified 2008 iwCLL guidelines with modification for treatment-related lymphocytosis for patients with CLL and per Lugano Classification for non-Hodgkin’s lymphoma for patients with SLL. There was hierarchical testing of non-inferiority followed by superiority in ORR as assessed by investigator and IRC. Key secondary endpoints include progression-free survival (PFS), duration of response, overall survival, and incidence of adverse events. The study is ongoing, with pre-specified endpoints of ORR and PFS to be evaluated at the planned final analysis expected in 2022.
About BRUKINSA
BRUKINSA is a small molecule inhibitor of Bruton’s tyrosine kinase (BTK) discovered by BeiGene scientists that is currently being evaluated globally in a broad clinical program as a monotherapy and in combination with other therapies to treat various B-cell malignancies. Because new BTK is continuously synthesized, BRUKINSA was specifically designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity. With differentiated pharmacokinetics compared to approved BTK inhibitors, BRUKINSA has been demonstrated to inhibit the proliferation of malignant B cells within a number of disease relevant tissues.
BRUKINSA is approved in the following indications and regions:
- For the treatment of mantle cell lymphoma (MCL) in adult patients who have received at least one prior therapy (United States, November 2019)*;
- For the treatment of MCL in adult patients who have received at least one prior therapy (China, June 2020)**;
- For the treatment of chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) in adult patients who have received at least one prior therapy (China, June 2020)**;
- For the treatment of relapsed or refractory MCL (United Arab Emirates, February 2021); and
- For the treatment of Waldenström’s macroglobulinemia (WM) in adult patients (Canada, March 2021).
To-date, more than 30 marketing authorization applications in multiple indications have been submitted outside of the United States and China, covering the EU and more than 20 other countries.
* This indication was approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
** This indication was approved under conditional approval. Complete approval for this indication may be contingent upon results from ongoing randomized, controlled confirmatory clinical trials.
Please see the full U.S. Prescribing Information for BRUKINSA.
To learn more about BeiGene, please visit www.beigene.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20210428005360/en/
BeiGene posts positive data from interim analysis of late-stage brukinsa leukemia study
Apr. 28, 2021 9:24 AM ET BeiGene, Ltd. (BGNE) By: Aakash Babu, SA News Editor
- BeiGene (NASDAQ:BGNE) announces positive results from a planned interim analysis of the Phase 3 ALPINE trial comparing brukinsa (zanubrutinib) against ibrutinib in adults with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).
- https://seekingalpha.com/news/3686879-beigene-posts-positive-data-from-interim-analysis-of-late-stage-brukinsa-leukemia-study
- https://seekingalpha.com/symbol/BGNE
- https://www.beigene.com/?loc=us
- https://www.brukinsa.com/
Opdivo (nivolumab)
U.S. Food and Drug Administration Accepts for Priority Review Bristol Myers Squibb’s Application for Opdivo (nivolumab) as Adjuvant Treatment for Patients with Muscle-Invasive Urothelial Carcinoma
04/30/2021CATEGORY:
Application based on Phase 3 CheckMate -274 trial, in which Opdivo nearly doubled disease-free survival compared to placebo
U.S. Food and Drug Administration assigned a target action date of September 3, 2021; if approved,Opdivo would be the first adjuvant immunotherapy option for patients with muscle-invasive urothelial carcinoma in the U.S.
CheckMate -274 is one of four positive Phase 3 trials for Opdivo-based treatments in earlier stages of cancers
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) has accepted the supplemental Biologics License Application (sBLA) for Opdivo (nivolumab) for the adjuvant treatment of patients with surgically resected, high-risk muscle-invasive urothelial carcinoma, based on results from the CheckMate -274 trial. The FDA granted the application Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) goal date of September 3, 2021.
“After patients undergo surgery for muscle-invasive urothelial carcinoma, they continue to face uncertainties given the high rate of disease recurrence and the lack of safe and effective treatment options,” said Dana Walker, M.D., M.S.C.E., vice president, development program lead, genitourinary cancers, Bristol Myers Squibb. “Based on the ground-breaking disease-free survival results from CheckMate -274, we believe Opdivo has the potential to change the future of treatment for muscle-invasive urothelial carcinoma. We look forward to working with the FDA towards the goal of bringing the first adjuvant immunotherapy option to these patients in the U.S.”
Opdivo (nivolumab)
About CheckMate -274
CheckMate -274 is a Phase 3 randomized, double-blind, multi-center study evaluating Opdivo compared to placebo in patients with muscle-invasive urothelial cancer at a high risk of recurrence after radical surgery. A total of 709 patients were randomized 1:1 to receive Opdivo 240 mg or placebo every two weeks for up to one year. The primary endpoints of the trial are DFS in all randomized patients (i.e., the intention-to-treat population) and in the subset of patients whose tumors express PD-L1 ≥1%. Key secondary endpoints include overall survival, non-urothelial tract recurrence free survival and disease-specific survival.
About Opdivo
Opdivo is a programmed death-1 (PD-1) immune checkpoint inhibitor that is designed to uniquely harness the body’s own immune system to help restore anti-tumor immune response. By harnessing the body’s own immune system to fight cancer, Opdivo has become an important treatment option across multiple cancers.
Opdivo’s leading global development program is based on Bristol Myers Squibb’s scientific expertise in the field of Immuno-Oncology and includes a broad range of clinical trials across all phases, including Phase 3, in a variety of tumor types. To date, the Opdivo clinical development program has treated more than 35,000 patients. The Opdivo trials have contributed to gaining a deeper understanding of the potential role of biomarkers in patient care, particularly regarding how patients may benefit from Opdivo across the continuum of PD-L1 expression.
In July 2014, Opdivo was the first PD-1 immune checkpoint inhibitor to receive regulatory approval anywhere in the world. Opdivo is currently approved in more than 65 countries, including the United States, the European Union, Japan and China. In October 2015, the Company’s Opdivo and Yervoy combination regimen was the first Immuno-Oncology combination to receive regulatory approval for the treatment of metastatic melanoma and is currently approved in more than 50 countries, including the United States and the European Union.
Indications
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express PD-L1 (≥1%) as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab) and 2 cycles of platinum-doublet chemotherapy, is indicated for the first-line treatment of adult patients with metastatic or recurrent non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab) is indicated for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving OPDIVO.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with unresectable malignant pleural mesothelioma (MPM).
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of patients with intermediate or poor risk advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab), in combination with cabozantinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab) is indicated for the treatment of patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy.
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with classical Hodgkin lymphoma (cHL) that has relapsed or progressed after autologous hematopoietic stem cell transplantation (HSCT) and brentuximab vedotin or after 3 or more lines of systemic therapy that includes autologous HSCT. This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab) is indicated for the treatment of patients with recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN) with disease progression on or after platinum-based therapy.
OPDIVO® (nivolumab) is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of adult and pediatric (12 years and older) patients with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab) is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection.
OPDIVO® (nivolumab) is indicated for the treatment of patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) after prior fluoropyrimidine- and platinum-based chemotherapy.
OPDIVO® (nivolumab), in combination with fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the treatment of patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma.
Please see US Full Prescribing Information for OPDIVO and YERVOY.
For more information about Bristol Myers Squibb, visit us at BMS.com
FDA accepts Bristol Myers' Opdivo sBLA for priority review
Apr. 30, 2021 7:44 AM ET Bristol-Myers Squibb Company (BMY) By: Aakash Babu, SA News Editor
- Bristol Myers Squibb (NYSE:BMY) announces that the U.S. FDA has accepted its supplemental Biologics License Application ((sBLA)) for Opdivo to treat a form of carcinoma, uhttps://seekingalpha.com/news/3688578-fda-accepts-bristol-myers-opdivo-sbla-for-priority-reviewnder priority review.
- https://seekingalpha.com/symbol/BMY
- https://www.bms.com/
biotecMAX™ April 2021 Insight
Opdivo (nivolumab) Plus Yervoy (ipilimumab)
Bristol Myers Squibb Receives Positive CHMP Opinion Recommending Approval for Opdivo (nivolumab) Plus Yervoy (ipilimumab) as First-Line Treatment for Unresectable Malignant Pleural Mesothelioma
04/23/2021CATEGORY:
Recommendation based on positive results from the Phase 3 CheckMate -743 trial, in which Opdivo plus Yervoy demonstrated significantly superior overall survival vs. standard-of-care chemotherapy
CheckMate -743 represents the first Phase 3 trial to show benefit with immunotherapy in previously untreated malignant pleural mesothelioma
If approved, Opdivo plus Yervoy will be the first new treatment option authorized for European patients that has demonstrated improved survival in more than 15 years
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 Opdivo (nivolumab) plus Yervoy (ipilimumab) for the first-line treatment of adults with unresectable malignant pleural mesothelioma (MPM). The European Commission (EC), which has the authority to approve medicines for the European Union (EU), will now review the CHMP recommendation.
“For more than 15 years, no new treatment options that can improve survival have been approved for malignant pleural mesothelioma, and today most patients only live for just over a year from the time of their diagnosis,” said Abderrahim Oukessou, M.D., vice president, thoracic cancers development lead, Bristol Myers Squibb. “Now, with the positive CHMP opinion for Opdivo plus Yervoy, we are one step closer to helping address the pressing unmet need for effective, proven therapies for this aggressive cancer. We look forward to potentially bringing the first immunotherapy combination that may offer a chance for a longer life to patients in the EU.”
Opdivo (nivolumab) Plus Yervoy (ipilimumab)
About CheckMate -743
CheckMate -743 is an open-label, multi-center, randomized Phase 3 trial evaluating Opdivo plus Yervoy compared to chemotherapy (pemetrexed and cisplatin or carboplatin) in patients with previously untreated malignant pleural mesothelioma (n=605). Patients with interstitial lung disease, active autoimmune disease, medical conditions requiring systemic immunosuppression, or active brain metastasis were excluded from the trial. In the trial, 303 patients were randomized to receive Opdivo at 3 mg/kg every two weeks and Yervoy at 1 mg/kg every six weeks; 302 patients were randomized to receive cisplatin 75 mg/m2 or carboplatin AUC 5 plus pemetrexed 500 mg/m2 in 21-day cycles for six cycles. Treatment in both arms continued until disease progression or unacceptable toxicity or, in the Opdivo plus Yervoy arm, up to 24 months. The primary endpoint of the trial was OS in all randomized patients. Additional efficacy outcome measures included progression-free survival (PFS), objective response rate (ORR) and duration of response (DOR), as assessed by blinded independent central review (BICR) utilizing modified RECIST criteria. Exploratory endpoints included safety, pharmacokinetics, immunogenicity and patient reported outcomes.
About Opdivo
Opdivo is a programmed death-1 (PD-1) immune checkpoint inhibitor that is designed to uniquely harness the body’s own immune system to help restore anti-tumor immune response. By harnessing the body’s own immune system to fight cancer, Opdivo has become an important treatment option across multiple cancers.
Opdivo’s leading global development program is based on Bristol Myers Squibb’s scientific expertise in the field of Immuno-Oncology, and includes a broad range of clinical trials across all phases, including Phase 3, in a variety of tumor types. To date, the Opdivo clinical development program has treated more than 35,000 patients. The Opdivo trials have contributed to gaining a deeper understanding of the potential role of biomarkers in patient care, particularly regarding how patients may benefit from Opdivo across the continuum of PD-L1 expression.
In July 2014, Opdivo was the first PD-1 immune checkpoint inhibitor to receive regulatory approval anywhere in the world. Opdivo is currently approved in more than 65 countries, including the United States, the European Union, Japan and China. In October 2015, the Company’s Opdivo and Yervoy combination regimen was the first Immuno-Oncology combination to receive regulatory approval for the treatment of metastatic melanoma and is currently approved in more than 50 countries, including the United States and the European Union.
About Yervoy
Yervoy is a recombinant, human monoclonal antibody that binds to the cytotoxic T-lymphocyte-associated antigen-4 (CTLA-4). CTLA-4 is a negative regulator of T-cell activity. Yervoy binds to CTLA-4 and blocks the interaction of CTLA-4 with its ligands, CD80/CD86. Blockade of CTLA-4 has been shown to augment T-cell activation and proliferation, including the activation and proliferation of tumor infiltrating T-effector cells. Inhibition of CTLA-4 signaling can also reduce T-regulatory cell function, which may contribute to a general increase in T-cell responsiveness, including the anti-tumor immune response. On March 25, 2011, the U.S. Food and Drug Administration (FDA) approved Yervoy 3 mg/kg monotherapy for patients with unresectable or metastatic melanoma. Yervoy is approved for unresectable or metastatic melanoma in more than 50 countries. There is a broad, ongoing development program in place for Yervoy spanning multiple tumor types.
U.S. FDA-Approved Indications
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express PD-L1 (≥1%) as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab) and 2 cycles of platinum-doublet chemotherapy, is indicated for the first-line treatment of adult patients with metastatic or recurrent non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab) is indicated for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving OPDIVO.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with unresectable malignant pleural mesothelioma (MPM).
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of patients with intermediate or poor risk advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab), in combination with cabozantinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab) is indicated for the treatment of patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy.
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with classical Hodgkin lymphoma (cHL) that has relapsed or progressed after autologous hematopoietic stem cell transplantation (HSCT) and brentuximab vedotin or after 3 or more lines of systemic therapy that includes autologous HSCT. This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab) is indicated for the treatment of patients with recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN) with disease progression on or after platinum-based therapy.
OPDIVO® (nivolumab) is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of adult and pediatric (12 years and older) patients with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab) is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection.
OPDIVO® (nivolumab) is indicated for the treatment of patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) after prior fluoropyrimidine- and platinum-based chemotherapy.
OPDIVO® (nivolumab), in combination with fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the treatment of patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma.
For more information about Bristol Myers Squibb, visit us at BMS.com
Bristol Myers' Opdivo + Yervoy gets positive CHMP opinion for malignant pleural mesothelioma
Apr. 23, 2021 7:03 AM ET Bristol-Myers Squibb Company (BMY) By: Mamta Mayani, SA News Editor
- The European Medicines Agency's advisory group CHMP has recommended approval of Bristol Myers Squibb' (NYSE:BMY) Opdivo (nivolumab) plus Yervoy (ipilimumab) for the first-line treatment of adults with unresectable malignant pleural mesothelioma (MPM).
- https://seekingalpha.com/news/3685006-bristol-myers-opdivo-yervoy-gets-positive-chmp-opinion-for-malignant-pleural-mesothelioma
- https://seekingalpha.com/symbol/BMY
- https://www.bms.com/
- https://www.opdivo.com/about-opdivo/how-the-combination-works-combinationtherapy
Mobocertinib (TAK-788)
Takeda Announces U.S. FDA Grants Priority Review for New Drug Application for Mobocertinib (TAK-788) as a Treatment for EGFR Exon20 Insertion+ Metastatic Non-Small Cell Lung Cancer
− Prescription Drug User Fee Act (PDUFA) Target Action Date Set for October 26, 2021
April 27, 2021 05:30 PM Eastern Daylight Time
CAMBRIDGE, Mass. & OSAKA, Japan--(BUSINESS WIRE)--Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) (“Takeda”) today announced that the U.S. Food and Drug Administration (FDA) has granted priority review for the company’s New Drug Application (NDA) for mobocertinib (TAK-788) for the treatment of adult patients with epidermal growth factor receptor (EGFR) Exon20 insertion mutation-positive (insertion+) metastatic non-small cell lung cancer (mNSCLC), as detected by an FDA-approved test, who have received prior platinum-based chemotherapy. Mobocertinib is the first oral therapy specifically designed to selectively target EGFR Exon20 insertion mutations.
Takeda has established an Expanded Access Program (EAP) (NCT04535557) for patients in the U.S. who may be eligible to receive access to mobocertinib during the review of the NDA. Additional information about Takeda’s EAP is available here.
About Mobocertinib (TAK-788)
Mobocertinib is an investigational, first-in-class, oral tyrosine kinase inhibitor (TKI) specifically designed to selectively target epidermal growth factor receptor (EGFR) Exon20 insertion mutations. In 2019, the U.S. FDA granted mobocertinib Orphan Drug Designation for the treatment of lung cancer with HER2 mutations or EGFR mutations including Exon20 insertion mutations. In April 2020, mobocertinib received Breakthrough Therapy Designation from the FDA for patients with EGFR Exon20 insertion+ metastatic non-small cell lung cancer (mNSCLC) whose disease has progressed on or after platinum-based chemotherapy. In October 2020, mobocertinib was designated as a Breakthrough Therapy in China by the Center for Drug Evaluation (CDE) for locally advanced or metastatic NSCLC patients with EGFR Exon20 insertion mutations who have been previously treated with at least one prior systemic chemotherapy.
About the Phase 1/2 Trial
The Phase 1/2 trial aims to evaluate the safety, pharmacokinetics and anti-tumor activity of oral mobocertinib in patients with non-small cell lung cancer (NSCLC). The trial is comprised of a Phase 1 dose-escalation, evaluating mobocertinib as a monotherapy and in combination with chemotherapy, several expansion cohorts and an extension cohort in patients with epidermal growth factor receptor (EGFR) Exon20 insertion+ metastatic NSCLC (mNSCLC).
The platinum-pretreated population efficacy analysis investigated 114 patients with EGFR Exon20 insertion+ mNSCLC who received prior platinum-based therapy in the Phase 1/2 trial and were treated with mobocertinib at the 160 mg once daily dose.
For more information, visit www.takedaoncology.com.
For more information, visit https://www.takeda.com.
FDA grants priority review for Takeda's mobocertinib application in lung cancer
Apr. 28, 2021 12:29 AM ET
Takeda Pharmaceutical Company Limited (TAK)
By: Mamta Mayani, SA News Editor
- The FDA has granted priority review to Takeda Pharmaceutical's (NYSE:TAK) New Drug Application (NDA) for mobocertinib (TAK-788) for the treatment of adult patients with epidermal growth factor receptor (EGFR) Exon20 insertion mutation-positive metastatic non-small cell lung cancer (mNSCLC), who have received prior platinum-based chemotherapy.
- https://seekingalpha.com/news/3686626-fda-grants-priority-review-for-takedas-mobocertinib-application-in-lung-cancer
- https://seekingalpha.com/symbol/TAK
- Takeda Pharmaceutical Company Limited (TSE: 4502/NYSE: TAK)
Nirsevimab MELODY Phase III trial met primary endpoint of reducing RSV lower respiratory tract infections in healthy infants
PUBLISHED26 April 2021
26 April 2021 07:00 BST
First potential passive immunisation to show protection against RSV in the general infant population
The MELODY Phase III trial for nirsevimab met its primary endpoint of a statistically significant reduction in the incidence of medically attended lower respiratory tract infections (LRTI) caused by respiratory syncytial virus (RSV) compared to placebo in healthy late preterm and term infants (35 weeks or more) during their first RSV season.1
Nirsevimab is a long-acting antibody, using AstraZeneca’s proprietary YTE technology, and being developed by AstraZeneca and Sanofi, with the potential to provide immunity directly to infants and offer immediate protection against RSV. It is the first potential immunisation to show protection against RSV in the general infant population in a Phase III trial.
Preliminary analysis of the safety profile for nirsevimab was consistent with previous trial data. No clinically meaningful differences in safety results between the nirsevimab and placebo groups have been seen.1
Nirsevimab is also being evaluated in the MEDLEY Phase II/III trial which will assess the safety and tolerability of nirsevimab compared to Synagis (palivizumab) among preterm infants and children with chronic lung disease (CLD) and congenital heart disease (CHD) entering their first and second RSV seasons.5 The MEDLEY trial is also expected to read out earlier with first data anticipated in the second half of 2021. MELODY, MEDLEY and the Phase IIb trial will form the basis of AstraZeneca’s regulatory submissions planned for 2022.
Nirsevimab has been granted breakthrough designation by three major regulatory agencies around the world. These include Breakthrough Therapy Designation by The China Center for Drug Evaluation under the National Medical Products Administration; Breakthrough Therapy Designation from the US Food and Drug Administration; and access granted to the European Medicines Agency PRIority MEdicines (PRIME) scheme.
MELODY
MELODY is a randomised, placebo-controlled Phase III trial conducted across at least 21 countries designed to determine the incidence of medically attended LRTI due to RSV confirmed by reverse transcriptase polymerase chain reaction (RT-PCR) testing through 150 days after dosing, versus placebo, in healthy infants entering their first RSV season. Healthy late preterm and term infants born at 35 weeks 0 days or greater gestational age were randomised (2:1) to receive a single 50mg (in infants weighing <5kg) or 100mg (in infants weighing >5kg) intramuscular injection of nirsevimab or placebo. Between July 2019 and February 2021 approximately 1,500 infants were dosed with either nirsevimab or placebo at the RSV season start.1 An additional 1,500 infants will be enrolled in the Northern and Southern Hemispheres to complete the safety evaluation.
Nirsevimab
Nirsevimab is a long-acting antibody being developed as a passive immunisation for the prevention of LRTI caused by RSV. It is being developed for use in a broader infant population than the current standard of care, Synagis, including for use in all infants experiencing their first RSV season and for infants with CHD or CLD entering their first and second RSV season.13-15 Due to its extended half-life technology nirsevimab may only require one dose during a typical five-month RSV season.14 The current anti-RSV antibody, AstraZeneca’s Synagis, is limited to high-risk infants and provides one-month protection, requiring five injections to cover an RSV season.15
Nirsevimab is a passive immunisation, designed to provide RSV protection to all infants, whereby an antibody is given directly to an infant to help prevent RSV, unlike active immunisation, where a person’s immune system is activated to prevent or fight infection through a vaccine.16 Passive immunisation could offer immediate protection unlike active immunisation, which can take weeks to develop protection.16
In March 2017, AstraZeneca and Sanofi announced an agreement to develop and commercialise nirsevimab. Under the terms of the agreement, AstraZeneca leads all development activity through initial approvals and retains manufacturing activities and Sanofi will lead commercialisation activities and record revenues. Under the terms of the global agreement, Sanofi made an upfront payment of €120m, has paid a development milestone of €30m and will pay up to a further €465m upon achievement of certain development and sales-related milestones. The two companies share all costs and profits. Revenue from the agreement is reported as Collaboration Revenue in the Company’s financial statements.
Related, in November 2018, AstraZeneca divested US commercial rights for Synagis to Swedish Orphan Biovitrum AB (publ) (Sobi) in addition to the right to participate in payments that may be received by AstraZeneca from the US profits or losses for nirsevimab. Under the agreement, AstraZeneca received upfront consideration of $1.5bn, consisting of $1.0bn in cash and $500m in ordinary shares of Sobi upon completion, and will have received a total of $60m in non-contingent payments for nirsevimab during 2019-2021. AstraZeneca will also receive up to $470m in sales-related payments for Synagis, a $175m milestone following the submission of the Biologics License Application for nirsevimab and potential net payments of approximately $110m on achievement of other nirsevimab profit and development-related milestones.
Please visit astrazeneca.com
Sanofi/AstraZeneca's Phase 3 nirsevimab study meets primary endpoint
Apr. 26, 2021 5:21 AM ET Sanofi (SNY)
By: Mamta Mayani, SA News Editor1 Comment
- Sanofi (NASDAQ:SNY) announces positive topline results from Phase 3 MELODY trial showing nirsevimab reduced lower respiratory tract infections (LRTI) requiring medical attention due to respiratory syncytial virus (RSV) in healthy preterm and term infants.
- https://seekingalpha.com/news/3685329-sanofiastrazenecas-late-sage-nirsevimab-study-meets-primary-endpoint
- Nirsevimab, being developed in partnership with AstraZeneca (NASDAQ:AZN), is an extended half-life monoclonal antibody (mAb) aiming to protect all infants from RSV.
- https://seekingalpha.com/symbol/SNY
- https://www.sanofi.com/en
- https://seekingalpha.com/symbol/AZN
- https://www.astrazeneca.com/
ENSPRYNG® (satralizumab)
CHMP recommends EU approval of Roche’s ENSPRYNG (satralizumab) for adults and adolescents with neuromyelitis optica spectrum disorder (NMOSD)
- If approved, ENSPRYNG will be the first and only treatment available to both adults and adolescents from 12 years of age with anti-aquaporin-4 antibody (AQP4-IgG) seropositive NMOSD in the EU
- ENSPRYNG is the only subcutaneous treatment option for NMOSD that can be administered at home every four weeks
- Recommendation is based on results from the two pivotal Phase III SAkuraStar and SAkuraSky studies, in which ENSPRYNG demonstrated robust and sustained efficacy in reducing the risk of relapse and a favourable safety profile
- NMOSD is a rare, lifelong and debilitating autoimmune disorder of the central nervous system that can cause blindness, muscle weakness and paralysis
Basel, 23 April 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of ENSPRYNG® (satralizumab) as the first subcutaneous treatment option for adults and adolescents from 12 years of age living with anti-aquaporin-4 antibody (AQP4-IgG) seropositive neuromyelitis optica spectrum disorder (NMOSD), as a monotherapy or in combination with immunosuppressive therapy (IST). AQP4-IgG are present in around 70-80% of people with NMOSD, who tend to experience a more severe disease course.
ENSPRYNG is a humanised monoclonal antibody designed to target and inhibit interleukin-6 (IL-6) receptor activity, believed to play a key role in the inflammation associated with NMOSD. The treatment was designed by Chugai, a member of the Roche Group, using novel recycling antibody technology which, compared to conventional technology, allows for longer duration of antibody circulation and subcutaneous dosing every four weeks after an initial loading dose.
The CHMP recommendation is based on the results of the Phase III SAkuraStar and SAkuraSky studies in which ENSPRYNG showed robust and sustained efficacy results in reducing the risk of relapse and a favourable safety profile in people with AQP4-IgG seropositive NMOSD. A final decision regarding the approval is expected from the European Commission in the near future.
About SAkuraStar and SAkuraSky in NMOSD
SAkuraStar was a pivotal Phase III study evaluating the efficacy and safety of ENSPRYNG monotherapy administered to adults with neuromyelitis optica spectrum disorder (NMOSD). In the anti-aquaporin-4 antibody (AQP4-IgG) seropositive subgroup, 83% treated with ENSPRYNG remained relapse free at 48 weeks, compared with 55% of those treated with placebo. At 96 weeks, 77% of those treated with ENSPRYNG remained relapse free, compared with 41% with placebo.
SAkuraSky was a pivotal Phase III study evaluating the efficacy and safety of ENSPRYNG in combination with baseline immunosuppressive therapy in adults and adolescents with NMOSD. Overall, 92% of AQP4-seropositive participants receiving ENSPRYNG in combination with immunosuppressants remained relapse free at 48 and 96 weeks, compared with 60% and 53% with placebo at 48 and 96 weeks.
The primary endpoint of both the SAkuraStar and SAkuraSky studies was time to first protocol-defined relapse (PDR) adjudicated by an independent review committee in the double-blind period.
ENSPRYNG demonstrated a favourable safety and tolerability profile in the Phase III studies.
The most common adverse reactions observed in the safety population were: headache, arthralgia, white blood cell count decrease, hyperlipidaemia and injection-related reactions.
About ENSPRYNG® (satralizumab)
ENSPRYNG, which was designed by Chugai, a member of the Roche Group, is a humanised monoclonal antibody that targets interleukin-6 (IL-6) receptor activity. The cytokine IL-6 is believed to be a key driver in NMOSD, triggering the inflammation cascade and leading to damage and disability. ENSPRYNG was designed using novel recycling antibody technology which, compared to conventional technology, allows for longer duration of the antibody and subcutaneous dosing every four weeks.
Positive Phase III results for ENSPRYNG, as both monotherapy and in combination with baseline immunosuppressive therapy, suggest that IL-6 inhibition is an effective therapeutic approach for NMOSD. The Phase III clinical development programme for ENSPRYNG includes two studies: SAkuraStar and SAkuraSky.
ENSPRYNG is currently approved in 20 countries, including the United States, Canada, Japan and Switzerland. Applications are under review with numerous regulators.
ENSPRYNG has been designated as an orphan drug in the U.S., Europe and Japan. In addition, it was granted Breakthrough Therapy Designation for the treatment of NMOSD by the FDA in December 2018.
For more information, please visit www.roche.com.
European advisory group backs approval Roche's Enspryng for NMOSD
Apr. 26, 2021 12:22 AM ETRoche Holding AG (RHHBY)By: Mamta Mayani, SA News Editor
- The EMA's Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of Roche (OTCQX:RHHBY) unit Chugai Pharmaceutical's (OTCPK:CHGCF) Enspryng (satralizumab), as the treatment for adults and adolescents from 12 years of age living with anti-aquaporin-4 antibody (AQP4-IgG) seropositive neuromyelitis optica spectrum disorder (NMOSD), as a monotherapy or in combination with immunosuppressive therapy (IST).
- https://seekingalpha.com/news/3685317-european-advisory-group-backs-approval-roches-enspryng-for-nmosd
- https://seekingalpha.com/symbol/RHHBY
- https://seekingalpha.com/symbol/RHHBY
- https://seekingalpha.com/symbol/CHGCF
- https://www.chugai-pharm.co.jp/english/
Selumetinib
Selumetinib recommended for approval in the EU by CHMP as the first medicine for paediatric patients with neurofibromatosis type 1 and plexiform neurofibromas
PUBLISHED26 April 2021
26 April 2021 07:10 BST
Recommendation based on the SPRINT Phase II trial, which showed selumetinib reduced tumour volume in children
AstraZeneca and MSD’s selumetinib has been recommended for conditional marketing authorisation in the European Union (EU) for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in paediatric patients with neurofibromatosis type 1 (NF1) aged three years and above.1
NF1 is a debilitating genetic condition affecting 1 in 3,000 individuals worldwide.2,3 In 30-50% of people with NF1, tumours develop on the nerve sheaths (plexiform neurofibromas) and can cause clinical issues such as disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment, and bladder/bowel dysfunction.4,5-8
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency based its positive opinion on results from the National Cancer Institute (NCI) Cancer Therapy Evaluation Program (CTEP)-sponsored SPRINT Stratum 1 Phase II trial. Results were published in The New England Journal of Medicine.8 Safety and efficacy data from the SPRINT trial with longer follow up will be provided to the CHMP as a condition of the recommendation for approval.
The trial showed selumetinib demonstrated an objective response rate (ORR) of 66% (33 of 50 patients, confirmed partial response) in paediatric patients with NF1 PN when treated with selumetinib as twice-daily oral monotherapy.1 ORR is defined as the percentage of patients with confirmed complete or partial response of at least 20% reduction in tumour volume.1
SPRINT
The SPRINT Stratum 1 Phase I/II trial was designed to evaluate the objective response rate and impact on patient-reported and functional outcomes in paediatric patients with NF1-related inoperable PNs treated with selumetinib monotherapy.11 This trial sponsored by NCI CTEP was conducted under a Cooperative Research and Development Agreement between NCI and AstraZeneca with additional support from Neurofibromatosis Therapeutic Acceleration Program (NTAP).
Selumetinib
Selumetinib is an inhibitor of mitogen-activated protein kinase kinases 1 and 2 (MEK1/2).1 MEK1/2 proteins are upstream regulators of the extracellular signal-related kinase (ERK) pathway. Both MEK and ERK are critical components of the RAS-regulated RAF-MEK-ERK pathway, which is often activated in different types of cancers.11
Selumetinib received US FDA Breakthrough Therapy Designation in April 2019, Rare Pediatric Disease Designation in December 2019 and US Orphan Drug Designation in February 2018. Further orphan drug designations have been granted in the EU, Japan, Russia, Switzerland, South Korea, Taiwan and Australia.
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 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. Independently, the companies will develop Lynparza and Koselugo in combination with their respective PD-L1 and PD-1 medicines.
Please visit astrazeneca.com
European advisory group backs AstraZeneca/Merck's selumetinib for neurofibromatosis in children
Apr. 26, 2021 6:19 AM ET AstraZeneca PLC (AZN)
By: Mamta Mayani, SA News Editor1 Comment
- AstraZeneca (NASDAQ:AZN) and Merck's (NYSE:MRK) selumetinib has been recommended for conditional marketing authorization in the European Union (EU) for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in paediatric patients with neurofibromatosis type 1 (NF1) aged three years and above.
- https://seekingalpha.com/news/3685341-european-advisory-group-backs-astrazenecamercks-selumetinib-for-neurofibromatosis-in-children
- https://seekingalpha.com/symbol/AZN
- https://seekingalpha.com/symbol/MRK
- https://www.astrazeneca.com/
Ruxolitinib Cream
Incyte Announces New Findings from a Randomized Phase 2 Study of Ruxolitinib Cream in Patients with Vitiligo
April 23, 2021 DownloadPDF Format (opens in new window)
- Presentations will be available on demand as part of the American Academy of Dermatology Virtual Meeting Experience 2021 (AAD VMX)
WILMINGTON, Del.--(BUSINESS WIRE)-- Incyte (Nasdaq:INCY) today announced findings from two analyses of its randomized, dose-ranging, vehicle-controlled Phase 2 study evaluating ruxolitinib cream, an investigational nonsteroidal, anti-inflammatory, JAK inhibitor therapy, in adult patients with vitiligo. These presentations (Poster #27535 and #27568) will be available on demand as part of the American Academy of Dermatology Virtual Meeting Experience 2021 (AAD VMX), held virtually from April 23–25, 2021.
As previously announced, the Phase 2 study met its primary endpoint, demonstrating that significantly more patients treated with ruxolitinib cream for 24 weeks achieved a ≥50 percent improvement from baseline in the facial vitiligo area scoring index (F-VASI50) score compared to patients treated with a vehicle control (non-medicated cream). Fifty-two-week results were also previously reported.
Updated results at Week 104 (Poster #27535) continue to show improvements in facial repigmentation, measured by F-VASI50 score, and total body repigmentation, measured by the proportion of patients achieving a ≥50 percent improvement from baseline in the total vitiligo area scoring index (T-VASI50), a key secondary endpoint, with 1.5% ruxolitinib cream administered twice daily. The results also add to previous findings indicating that a longer duration of therapy was associated with greater repigmentation, as assessed using the F-VASI and T-VASI responses at Weeks 24, 52 and 104.
These presentations are available on demand on the AAD VMX website at https://eposters.aad.org/categories, and can be accessed until July 12, 2021.
About the Phase 2 Study (NCT03099304)
This randomized, dose-ranging, vehicle-controlled, Phase 2 study is evaluating the safety and efficacy of ruxolitinib cream for adult patients with vitiligo.
The first part of the study spanned 24 weeks and enrolled 157 adults (aged 18-75 years) diagnosed with vitiligo and with depigmented areas of at least 0.5 percent of the body surface area (BSA) on the face and at least 3 percent of the total BSA on non-facial areas. Patients were equally randomized across five treatment arms, including: ruxolitinib cream 1.5 percent, 0.5 percent or 0.15 percent administered QD; ruxolitinib cream 1.5 percent administered BID; or vehicle control for 24 weeks.
The second part of the study spanned an additional 28 weeks (52 weeks total) and included patients enrolled in the first part of the study. Patients receiving vehicle control or those patients who achieved <25 percent improvement in F-VASI at Week 24 on ruxolitinib cream 0.15 percent were rerandomized to receive ruxolitinib cream 1.5 percent BID, 1.5 percent QD or 0.5 percent QD. After Week 52, patients who completed baseline, Week 24 and Week 52 assessments could continue to receive open-label treatment with 1.5% ruxolitinib cream BID for an additional 104 weeks.
The primary efficacy endpoint was the percentage of patients treated with ruxolitinib cream who achieved a F-VASI50 score at Week 24, compared to patients treated with vehicle control. Key secondary endpoints included the proportion of patients who achieved a F-PhGVA score of 0 or 1 at Week 24, the proportion of patients who achieved T-VASI50 at Week 52 and the safety and tolerability of ruxolitinib cream.
For more information about the study, please visit: https://clinicaltrials.gov/ct2/show/NCT03099304
About Ruxolitinib Cream
Ruxolitinib cream is a proprietary formulation of Incyte’s selective JAK1/JAK2 inhibitor ruxolitinib that has been designed for topical application. Ruxolitinib cream is currently in Phase 3 development for the treatment of atopic dermatitis (TRuE-AD) and for the treatment of adolescents and adults with vitiligo (TRuE-V). Incyte has worldwide rights for the development and commercialization of ruxolitinib cream.
About Incyte Dermatology
Incyte’s science-first approach and expertise in immunology has formed the foundation of the company. In Dermatology, the Company’s research and development efforts are focused on leveraging our knowledge of the JAK-STAT pathway to identify and develop topical and oral therapies with the potential to modulate immune pathways driving uncontrolled inflammation and help restore normal immune function.
Currently, Incyte is exploring the potential of JAK inhibition for a number of immune-mediated dermatologic conditions with a high unmet medical need, including atopic dermatitis, vitiligo and hidradenitis suppurativa. To learn more, visit the Dermatology section of Incyte.com.
For additional information on Incyte, please visit Incyte.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20210423005324/en/
Incyte presents more data from positive late-stage studies for ruxolitinib in atopic dermatitis
Apr. 23, 2021 11:52 AM ET Incyte Corporation (INCY)
By: Dulan Lokuwithana, SA News Editor
- Incyte (INCY +1.3%) has announced additional findings from two Phase 3 studies evaluating ruxolitinib cream, an investigational JAK1/JAK2 inhibitor for patients with atopic dermatitis ("AD").
- https://seekingalpha.com/news/3685158-incyte-presents-more-data-from-late-stage-studies-for-ruxolitinib-in-atopic-dermatitis
- https://seekingalpha.com/symbol/INCY
- https://www.incyte.com/
- https://www.jakafi.com/
ZYNLONTA™ (loncastuximab tesirine-lpyl)
ADC Therapeutics Announces FDA Approval of ZYNLONTA™ (loncastuximab tesirine-lpyl) in Relapsed or Refractory Diffuse Large B-Cell Lymphoma
APRIL, 23, 2021
First and only CD19-targeted antibody drug conjugate (ADC) as a single-agent treatment for adult patients with relapsed or refractory (r/r) diffuse large B-cell lymphoma (DLBCL)
ZYNLONTA addresses an unmet need across a broad population of third-line (3L)+ r/r patients, including patients with DLBCL not otherwise specified, DLBCL arising from low grade lymphoma and high-grade B-cell lymphoma
ZYNLONTA demonstrated 48.3% overall response rate, 24.1% complete response rate and durable responses in heavily pretreated patients in pivotal LOTIS-2 trial
Investor conference call and webcast to be held Friday, April 23rd at 4 p.m. ET
LAUSANNE, Switzerland--(BUSINESS WIRE)-- ADC Therapeutics SA (NYSE: ADCT) today announced that the U.S. Food and Drug Administration (FDA) has approved ZYNLONTA™ (loncastuximab tesirine-lpyl) for the treatment of adult patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified (NOS), DLBCL arising from low grade lymphoma and high-grade B-cell lymphoma.1 ZYNLONTA, a CD19-targeted antibody drug conjugate (ADC), has been granted accelerated approval by the FDA based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
“There is a significant unmet need for treatment options for patients with r/r DLBCL, including those who have been heavily pretreated and have difficult-to-treat disease,” said Paolo F. Caimi, MD, University Hospitals Cleveland Medical Center and Case Comprehensive Cancer Center, Case Western Reserve University. “Single-agent ZYNLONTA demonstrated clinically important outcomes in the pivotal LOTIS-2 study across several disease subtypes. Notably, this included transplant eligible and ineligible patients and patients who previously received stem cell transplant or CAR-T cell therapy.”
ZYNLONTA™ (loncastuximab tesirine-lpyl)
“The FDA approval of ZYNLONTA is an exciting advancement for patients with r/r DLBCL and a transformational event for ADC Therapeutics,” said Chris Martin, Chief Executive Officer of ADC Therapeutics. “We extend our deepest gratitude to the patients who participated in our LOTIS-1 and LOTIS-2 clinical trials, their families, the study investigators and our employees, as their commitment made this important milestone possible.”
DLBCL, the most common type of non-Hodgkin lymphoma in the United States, is a rapidly progressing, aggressive disease that is heterogeneous with multiple subtypes.2 More than 40% of first-line DLBCL treatments fail.3 For patients who fail first-line therapy, prognoses are poor, worsening with each line of therapy as the chance for cure or long-term disease-free survival diminishes.4,5
ZYNLONTA will be commercially available in the United States shortly. ADC Therapeutics has launched the Advancing Patient Support Program, a comprehensive patient support program offering financial assistance, ongoing education and other resources to eligible patients who are prescribed ZYNLONTA.
Please see full Prescribing Information at www.adctherapeutics.com for ZYNLONTA.
ADC Therapeutics is based in Lausanne (Biopôle), Switzerland and has operations in London, the San Francisco Bay Area and New Jersey. For more information, please visit https://adctherapeutics.com/
View source version on businesswire.com: https://www.businesswire.com/news/home/20210423005537/en/
https://adctherapeutics.com/our-pipeline/
FDA approves ADC's biologic Zynlonta for B-cell lymphoma
Apr. 23, 2021 2:11 PM ET ADC Therapeutics SA (ADCT)
By: Jonathan M Block, SA News Editor
- The FDA has approved Zynlonta (loncastuximab tesirine-lpyl) from ADC Therapeutics (NYSE:ADCT) for diffuse large B-cell lymphoma.
- https://seekingalpha.com/news/3685211-fda-approves-adcs-biologic-zynlonta-for-b-cell-lymphoma
- https://seekingalpha.com/symbol/ADCT
- https://adctherapeutics.com/
- https://adctherapeutics.com/our-pipeline/
- https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/761196s000lbl.pdf
Tagrisso (osimertinib)
Tagrisso recommended for approval in the EU by CHMP for the adjuvant treatment of patients with early-stage EGFR-mutated lung cancer
PUBLISHED26 April 2021
26 April 2021 07:05 BST
Opinion based on unprecedented results from the ADAURA Phase III trial where Tagrisso reduced the risk of disease recurrence or death by 80%
AstraZeneca’s Tagrisso (osimertinib) has been recommended for marketing authorisation in the European Union for the adjuvant treatment of adult patients with early-stage (IB, II and IIIA) epidermal growth factor receptor-mutated (EGFRm) non-small cell lung cancer (NSCLC) after complete tumour resection with curative intent. If approved, Tagrisso will be indicated for EGFRm patients whose tumours have exon 19 deletions or exon 21 (L858R) mutations.
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency based its positive opinion on results from the ADAURA Phase III trial. In the trial, Tagrisso demonstrated a statistically significant and clinically meaningful improvement in disease-free survival (DFS) in the primary analysis population of patients with Stage II and IIIA EGFRm NSCLC, and in the overall trial population of patients with Stage IB-IIIA disease. These results were published in The New England Journal of Medicine.
ADAURA
ADAURA is a randomised, double-blind, global, placebo-controlled Phase III trial in the adjuvant treatment of 682 patients with Stage IB, II and IIIA EGFRm NSCLC following complete tumour resection and adjuvant chemotherapy as indicated. Patients were treated with Tagrisso 80mg once-daily oral tablets or placebo for three years or until disease recurrence.
The trial enrolled patients in more than 200 centres across more than 20 countries, including the US, in Europe, South America, Asia and the Middle East. The primary endpoint was DFS in Stage II and IIIA patients and a key secondary endpoint was DFS in Stage IB, II and IIIA patients.
The data readout was originally anticipated in 2022. In April 2020, an Independent Data Monitoring Committee recommended for the trial to be unblinded two years early based on a determination of overwhelming efficacy. Investigators and patients continue to participate and remain blinded to treatment. The trial will continue to assess overall survival.
Tagrisso
Tagrisso (osimertinib) is a third-generation, irreversible EGFR-TKI with clinical activity against central nervous system metastases. Tagrisso (40mg and 80mg once-daily oral tablets) has been used to treat approximately 250,000 patients across indications worldwide and AstraZeneca continues to explore Tagrisso as a treatment for patients across multiple stages of EGFRm NSCLC.
In Phase III trials, Tagrisso is being tested in the Stage III locally advanced unresectable setting (LAURA), in the neoadjuvant resectable setting (NeoADAURA) and in combination with chemotherapy (FLAURA2). AstraZeneca is also researching ways to address tumour mechanisms of resistance through the SAVANNAH and ORCHARD Phase II trials, which test Tagrisso given concomitantly with savolitinib, an oral, potent and highly selective MET TKI, as well as other potential new medicines.
What is TAGRISSO?
TAGRISSO is a prescription medicine used to treat adults with non-small cell lung cancer (NSCLC) that has certain abnormal epidermal growth factor receptor (EGFR) gene(s):
- to help prevent your lung cancer from coming back after your tumor(s) has been removed by surgery, or
- as your first treatment when your lung cancer has spread to other parts of the body (metastatic), or
- when your lung cancer has spread to other parts of the body (metastatic) and you have had previous treatment with an EGFR tyrosine kinase inhibitor (TKI) medicine that did not work or is no longer working
Your healthcare provider will perform a test to make sure that TAGRISSO is right for you.
It is not known if TAGRISSO is safe and effective in children.
Please see complete Prescribing Information, including Patient Information.
You may report side effects related to AstraZeneca products by clicking here.
Please visit astrazeneca.com
European advisory group backs approval of AstraZeneca's Tagrisso in EGFR-mutated lung cancer
Apr. 26, 2021 5:42 AM ET AstraZeneca PLC (AZN)
By: Mamta Mayani, SA News Editor
- AstraZeneca’s (NASDAQ:AZN) Tagrisso (osimertinib) has been recommended for marketing authorization in the European Union for the adjuvant treatment of adult patients with early-stage epidermal growth factor receptor-mutated (EGFRm) non-small cell lung cancer (NSCLC) after complete tumour resection with curative intent.
- https://seekingalpha.com/news/3685332-european-advisory-group-backs-approval-of-astrazenecas-tagrisso-in-egfr-mutated-lung-cancer
- https://seekingalpha.com/symbol/AZN
- https://www.astrazeneca.com/
- https://www.tagrisso.com/
OTEZLA® (apremilast)
OTEZLA® (apremilast) Significantly Improved Measures Of Disease Severity In Adults With Mild-To-Moderate Plaque Psoriasis
Clinically Meaningful Improvements in Physician-Reported Outcomes Maintained Through Week 32
Consistent Efficacy Was Observed at Week 16 in Subgroups of Patients with BSA <5% and BSA >5% in the Phase 3 ADVANCE Trial
Supplemental New Drug Application for Otezla to Treat Adults with Mild-to-Moderate Plaque Psoriasis Currently Under Review by the U.S. Food and Drug Administration
THOUSAND OAKS, Calif., April 23, 2021 /PRNewswire/ -- Amgen (NASDAQ:AMGN) today announced Otezla® (apremilast) improved measures of disease severity in adults with mild-to-moderate plaque psoriasis regardless of their Body Surface Area (BSA) affected by the disease, according to findings from the placebo-controlled, Phase 3 ADVANCE trial. Results were presented at the American Academy of Dermatology Virtual Meeting Experience 2021.
"Many people with mild-to-moderate plaque psoriasis still report challenges in managing their symptoms, which can have a significant impact on their daily lives, despite the availability of existing topical treatment options," said Linda Stein Gold, M.D., director of dermatology clinical research at Henry Ford Health System, Detroit, and lead investigator for the ADVANCE study. "The ADVANCE findings demonstrated that oral apremilast significantly improved clinical measures of disease severity, such as Body Surface Area and scalp involvement."
In ADVANCE, oral Otezla 30 mg twice daily achieved a statistically significant improvement of the primary endpoint of static Physician's Global Assessment (sPGA) response at week 16 compared to placebo (21.6% vs. 4.1%, p<0.0001). These clinical improvements were maintained through week 32.
Otezla also demonstrated improvements in all secondary endpoints at week 16. A greater proportion of adults with BSA ≤5% treated with Otezla compared to placebo achieved a BSA ≤3% (respectively 71.7% vs. 35.8%), at least a 75% improvement in BSA (29.0% vs. 6.1%) and a Scalp PGA (ScPGA) response score of clear or almost clear (35.6% vs. 12.9%)
About ADVANCE (PSOR-022)
ADVANCE (NCT03721172) is a Phase 3, multicenter, randomized, placebo-controlled, double-blind study evaluating the efficacy and safety of Otezla in patients with mild-to-moderate plaque psoriasis (defined as BSA involvement of 2% to 15%, Psoriasis Area and Severity Index (PASI) score of 2 to 15, sPGA score of 2 to 3). The study randomized 595 patients 1:1 to receive Otezla (n=297) 30 mg twice daily or placebo (n=298) for the first 16 weeks. All patients then received Otezla during an open-label extension phase through week 32.
The primary endpoint was the percentage of patients with sPGA response [defined as a sPGA score of clear (0) or almost clear (1) with at least a 2-point reduction from baseline] at week 16.
About Otezla® (apremilast)
OTEZLA® (apremilast) is an oral small-molecule inhibitor of phosphodiesterase 4 (PDE4) specific for cyclic adenosine monophosphate (cAMP). PDE4 inhibition results in increased intracellular cAMP levels, which is thought to indirectly modulate the production of inflammatory mediators. The specific mechanism(s) by which Otezla exerts its therapeutic action in patients is not well defined.
Otezla® (apremilast) U.S. INDICATIONS
Otezla® (apremilast) is indicated for the treatment of adult patients with moderate to severe plaque psoriasis who are candidates for phototherapy or systemic therapy.
Otezla is indicated for the treatment of adult patients with active psoriatic arthritis.
Otezla is indicated for the treatment of adult patients with oral ulcers associated with Behçet's Disease.
Otezla® (apremilast) U.S. IMPORTANT SAFETY INFORMATION
Contraindications
- Otezla® (apremilast) is contraindicated in patients with a known hypersensitivity to apremilast or to any of the excipients in the formulation
Please click here for Otezla® Full Prescribing Information.
View original content to download multimedia:http://www.prnewswire.com/news-releases/otezla-apremilast-significantly-improved-measures-of-disease-severity-in-adults-with-mild-to-moderate-plaque-psoriasis-301276001.html
SOURCE Amgen
Amgen announces detailed results from positive Phase 3 trial for Otezla in plaque psoriasis
Apr. 23, 2021 4:29 PM ET Amgen Inc. (AMGN)
By: Dulan Lokuwithana, SA News Editor
- Amgen (NASDAQ:AMGN) has announced detailed positive results from the Phase 3 ADVANCE trial that evaluated its PDE4 inhibitor Otezla® (apremilast) in patients with mild-to-moderate plaque psoriasis.
- https://seekingalpha.com/news/3685264-amgen-announces-detailed-results-from-positive-phase-3-trial-for-otezla-in-plaque-psoriasis
- https://seekingalpha.com/symbol/AMGN
- https://www.amgen.com/
- https://www.otezla.com/
VENCLYXTO® (venetoclax)
April 23, 2021
AbbVie Receives Positive CHMP Opinion for VENCLYXTO® (venetoclax) as a Combination Regimen for Adult Patients with Newly Diagnosed Acute Myeloid Leukemia Who Are Ineligible for Intensive Chemotherapy
- Positive opinion based on data from the VIALE-A and M14-358 trials, which evaluated the safety, efficacy and pharmacokinetics of VENCLYXTO in combination with hypomethylating agents in adult patients with AML
- Represents the third positive CHMP opinion for an extension of indications for VENCLYXTO; supports growing utility of this therapy across multiple hematologic malignancies
- Acute myeloid leukemia is a difficult-to-treat blood cancer, and the most common type of acute leukemia in the world
NORTH CHICAGO, Ill., April 23, 2021 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion for VENCLYXTO® (venetoclax) in combination with hypomethylating agents for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) who are ineligible for intensive chemotherapy. The positive CHMP opinion is a scientific recommendation for marketing authorization to the European Commission (EC), which is expected to deliver its final decision on VENCLYXTO combination therapy for use in AML in the first half of 2021.
The positive CHMP opinion represents the third for an extension of indications for VENCLYXTO. The opinion is based on results from the double-blind, placebo-controlled VIALE-A (M15-656) and the Phase 1b open-label, nonrandomized, multicenter M14-358 trial.
"This positive CHMP opinion for VENCLYXTO in acute myeloid leukemia is a critical step to providing new therapeutic options in the European Union for patients with this devastating disease," said Mohamed Zaki, M.D., Ph.D., vice president and global head of oncology development at AbbVie. "Enabling improved outcomes including potentially prolonging the lives of patients with malignant diseases such as acute myeloid leukemia is part of our mission and an objective we pursue relentlessly every day."
About the VENCLYXTO AML Clinical Trial Program
AbbVie's clinical trial program to evaluate VENCLYXTO combination therapy in patients with newly diagnosed acute myeloid leukemia (AML) who were ineligible for intensive chemotherapy included two studies conducted around the world.
VIALE-A (M15-656) Phase 3 Trial
The randomized, double-blind, placebo-controlled VIALE-A (M15-656) trial evaluated the efficacy and safety of VENCLYXTO in combination with azacitidine in patients with newly diagnosed AML who were ineligible for intensive chemotherapy. The study met its primary endpoints of statistically significant improvement of overall survival (OS) and complete remission rate and complete remission with incomplete hematologic recovery (CR + CRi). OS was 14.7 months for the VENCLYXTO plus azacitidine arm versus 9.6 months in the placebo plus azacitidine arm. The study also met secondary endpoints, with the VENCLYXTO plus azacitidine arm resulting in a CR rate of 36.7 percent vs. 17.9 percent in the placebo plus azacitidine arm and a composite complete remission rate (CR + CRi) of 66.4 percent versus 28.3 percent. The safety profile of VENCLYXTO plus azacitidine was consistent with the known side-effect profiles of both agents, and adverse events (AEs) were consistent with expectations for an older AML population; no differences between the two treatment arms with respect to quality-of-life measures were seen. The most frequently reported serious AEs in the VENCLYXTO plus azacitidine arm and placebo plus azacitidine arm were febrile neutropenia (in 30 percent and 10 percent), pneumonia (in 17 percent and 22 percent), sepsis (in 6 percent and 8 percent), and haemorrhage (in 9 percent and 6 percent).5
M14-358 Phase 1b Trial
The non-randomized, open-label M14-358 trial evaluated VENCLYXTO in combination with azacitidine or decitabine in patients with newly diagnosed AML. The trial showed patients treated with VENCLYXTO in combination with azacitidine achieved a CR rate of 44 percent and a CR+CRi rate of 71 percent. The median duration of response for patients treated with VENCLYXTO in combination with azacitidine who achieve a CR or CRi was 21.9 months. Median time to first CR or CRi was 1.2 months (range: 0.7 to 7.7 months). Patients who received VENCLYXTO in combination with decitabine achieved a CR rate of 55 percent and a CR+CRi rate of 74 percent. The median duration of response for patients treated with VENCLYXTO in combination with decitabine who achieved a CR or CRi was 15 months. Median time to first CR or CRi was 1.9 months (range: 0.9 to 5.4 months). The most frequently reported serious AEs in patients receiving VENCLYXTO in combination with azacitidine were febrile neutropenia (31 percent) and pneumonia (26 percent). The most frequently reported serious AEs in patients receiving VENCLYXTO in combination with decitabine were febrile neutropenia (42 percent), pneumonia (29 percent), bacteraemia (16 percent) and sepsis (6 percent).6
About VENCLYXTO® (venetoclax)
VENCLYXTO® (venetoclax) is a first-in-class medicine that selectively binds and inhibits the B-cell lymphoma-2 (BCL-2) protein. In some blood cancers, BCL-2 prevents cancer cells from undergoing their natural death or self-destruction process, called apoptosis. VENCLYXTO targets the BCL-2 protein and works to help restore the process of apoptosis.
VENCLYXTO is being developed by AbbVie and Roche. It is jointly commercialized by AbbVie and Genentech, a member of the Roche Group, in the U.S. and by AbbVie outside of the U.S. Together, the companies are committed to BCL-2 research and to studying venetoclax in clinical trials across several blood and other cancers. Venetoclax is approved in more than 80 countries, including the U.S.
Indication and Important VENCLYXTO (venetoclax) EU Safety Information7
Indication
Venclyxto in combination with obinutuzumab is indicated for the treatment of adult patients with previously untreated chronic lymphocytic leukaemia (CLL).
Venclyxto in combination with rituximab is indicated for the treatment of adult patients with CLL who have received at least one prior therapy.
Venclyxto monotherapy is indicated for the treatment of CLL:
- In the presence of 17p deletion or TP53 mutation in adult patients who are unsuitable for or have failed a B-cell receptor pathway inhibitor, or
- In the absence of 17p deletion or TP53 mutation in adult patients who have failed both chemoimmunotherapy and a B-cell receptor pathway inhibitor.
For more information, please visit http://www.abbvie.com/oncology.
For more information about AbbVie, please visit us at www.abbvie.com.
European advisory group backs AbbVie's Venclyxto combo for acute myeloid leukemia
Apr. 23, 2021 8:41 AM ET AbbVie Inc. (ABBV) By: Mamta Mayani, SA News Editor
- AbbVie (NYSE:ABBV) announces that the Committee for Medicinal Products for Human Use (CHMP) of the EMA has adopted a positive opinion for Venclyxto (venetoclax) in combination with hypomethylating agents for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) who are ineligible for intensive chemotherapy.
- https://seekingalpha.com/news/3685072-european-advisory-group-backs-abbvies-venclyxto-combo-for-acute-myeloid-leukemia
- https://seekingalpha.com/symbol/ABBV
SOURCE AbbVie
VAZKEPA (icosapent ethyl)
AMARIN RECEIVES GREAT BRITAIN MARKETING AUTHORIZATION FOR VAZKEPA FROM THE MEDICINES AND HEALTHCARE PRODUCTS REGULATORY AGENCY (MHRA) 1,2
BackPDF VersionApr 22, 2021
VAZKEPA (icosapent ethyl) is the first and only authorized treatment for its cardiovascular risk reduction indication1,2,3
VAZKEPA authorization for Great Britain follows recent VAZKEPA authorization for European Union
DUBLIN, Ireland and BRIDGEWATER, N.J., April 22, 2021 (GLOBE NEWSWIRE) -- Amarin Corporation plc (NASDAQ: AMRN) today announced that the Medicines and Healthcare Products Regulatory Agency (MHRA) has granted a Marketing Authorization for VAZKEPA (icosapent ethyl) as a treatment to reduce the risk of cardiovascular events in high cardiovascular risk statin-treated adult patients who have elevated triglycerides (≥150 mg/dL) and either established cardiovascular disease or diabetes, and at least one additional cardiovascular risk factor. The Great Britain Marketing Authorization for VAZKEPA applies to England, Scotland and Wales. Under the Brexit Northern Ireland agreement, the European centralized marketing authorization for the European Union covers Northern Ireland.
The MHRA’s license swiftly followed the European Commission (EC) marketing authorization of icosapent ethyl for the European Union as announced on March 30, 2021.3 Amarin’s understanding is that icosapent ethyl is among the first products to be submitted and licensed through the MHRA’s new ‘reliance’ route following the end of the Brexit transition period. Icosapent ethyl has been identified as a new active substance with likely multi-factorial mechanisms of action.1
VAZKEPA (icosapent ethyl)
About REDUCE-IT®
REDUCE-IT was a global cardiovascular outcomes study designed to evaluate the effect of VASCEPA in adult patients with LDL-C controlled to between 41-100 mg/dL (median baseline 75 mg/dL) by statin therapy and various cardiovascular risk factors including persistent elevated triglycerides between 135-499 mg/dL (median baseline 216 mg/dL) and either established cardiovascular disease (secondary prevention cohort) or diabetes mellitus and at least one other cardiovascular risk factor (primary prevention cohort).
REDUCE-IT, conducted over seven years and completed in 2018, followed 8,179 patients at over 400 clinical sites in 11 countries with the largest number of sites located within the United States. REDUCE-IT was conducted based on a special protocol assessment agreement with FDA. The design of the REDUCE-IT study was published in March 2017 in Clinical Cardiology.7 The primary results of REDUCE-IT were published in The New England Journal of Medicine in November 2018.2 The total events results of REDUCE-IT were published in the Journal of the American College of Cardiology in March 2019.8 These and other publications can be found in the R&D section on the company’s website at www.amarincorp.com.
About VASCEPA® (icosapent ethyl) Capsules
VASCEPA (icosapent ethyl) capsules are the first-and-only prescription treatment approved by the U.S. Food and Drug Administration (FDA) comprised solely of the active ingredient, icosapent ethyl (IPE), a unique form of eicosapentaenoic acid. VASCEPA was launched in the United States in January 2020 as the first and only drug approved by the U.S. FDA for treatment of the studied high-risk patients with persistent cardiovascular risk after statin therapy. VASCEPA was initially launched in the United States in 2013 based on the drug’s initial FDA approved indication for use as an adjunct therapy to diet to reduce triglyceride levels in adult patients with severe (≥500 mg/dL) hypertriglyceridemia. Since launch, VASCEPA has been prescribed over ten million times. VASCEPA is covered by most major medical insurance plans. In addition to the United States, VASCEPA is approved and sold in Canada, Lebanon and the United Arab Emirates. In Europe, in March 2021 marketing authorization was granted to icosapent ethyl in the European Union for the reduction of risk of cardiovascular events in patients at high cardiovascular risk, under the brand name VAZKEPA.
Indications and Limitation of Use (in the United States)
VASCEPA is indicated:
- As an adjunct to maximally tolerated statin therapy to reduce the risk of myocardial infarction, stroke, coronary revascularization and unstable angina requiring hospitalization in adult patients with elevated triglyceride (TG) levels (≥ 150 mg/dL) and
- established cardiovascular disease or
- diabetes mellitus and two or more additional risk factors for cardiovascular disease.
- As an adjunct to diet to reduce TG levels in adult patients with severe (≥ 500 mg/dL) hypertriglyceridemia.
The effect of VASCEPA on the risk for pancreatitis in patients with severe hypertriglyceridemia has not been determined.
Information regarding Amarin’s plans for commercialization and securing market access in Europe and the United Kingdom can be found in the FAQ section under investor relations at www.amarincorp.com.
UK approves Amarin's Vazkepa for cardiovascular risk reduction
Apr. 22, 2021 11:22 AM ETAmarin Corporation plc (AMRN)By: Jonathan M Block, SA News Editor12 Comments
- The UK's Medicines and Healthcare Products Regulatory Agency ("MHRA") has granted marketing approval to Amarin (NASDAQ:AMRN) for Vazkepa (icosapent ethyl) as a treatment to reduce the risk of cardiovascular events in statin-treated adult patients with elevated triglycerides.
- https://seekingalpha.com/news/3684653-uk-approves-amarin-vazkepa-for-cardiovascular-risk-reduction
- https://seekingalpha.com/symbol/AMRN
- https://amarincorp.com/default.aspx
Onureg® (azacitidine tablets; CC-486)
Bristol Myers Squibb Receives Positive CHMP Opinion for Onureg® (azacitidine tablets; CC-486) as Frontline Oral Maintenance Therapy for Adults with Acute Myeloid Leukemia in First Remission
04/23/2021CATEGORY:
If approved, Onureg will become the first and only once daily oral frontline maintenance therapy in Europe for patients with a broad range of acute myeloid leukemia (AML) subtypes
In the pivotal QUAZAR® AML-001 study, Onureg demonstrated significant overall survival and showed a relapse-free survival benefit in patients with AML in first remission
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended approval of Onureg® (azacitidine tablets; CC-486) as a maintenance therapy in adult patients with acute myeloid leukemia (AML) who achieved complete remission (CR) or complete remission with incomplete blood count recovery (CRi) following induction therapy with or without consolidation treatment and who are not candidates for, including those who choose not to proceed to, hematopoietic stem cell transplantation (HSCT).
The CHMP recommendation will now be reviewed by the European Commission (EC), which has the authority to approve medicines for the European Union (EU). If approved, Onureg will be the first and only once daily frontline oral maintenance therapy to demonstrate significant overall survival in patients with a broad range of AML subtypes in first remission.
The CHMP adopted a positive opinion based on results from the QUAZAR® AML-001 study, a Phase 3, international, randomized, double-blind trial. Eligible patients were ages 55 years or older, had newly diagnosed AML, intermediate or poor cytogenetics, had achieved first CR or CRi following intensive induction chemotherapy with or without consolidation treatment (per investigator preference prior to study entry), and were not candidates for HSCT at the time of screening.1
About QUAZAR® AML-001
QUAZAR® AML-001, is a Phase 3, international, randomized, double-blind study. Eligible patients were ages 55 years or older, had newly diagnosed AML, intermediate or poor cytogenetics, had achieved first CR or CRi following intensive induction chemotherapy with or without consolidation treatment (per investigator preference prior to study entry) within four months before randomization, and were not candidates for HSCT at the time of screening. The study enrolled 472 patients, randomized 1:1 to receive either Onureg 300 mg (N=238) or placebo (N=234) orally, once daily, for 14 days of a 28-day cycle, plus best supportive care.1
Median OS, the primary endpoint, from time of randomization was greater than two years (24.7 months; 95% CI: 18.7 to 30.5) in the Onureg arm compared to 14.8 months for placebo (HR: 0.69, 95% CI: 0.55 to 0.86; p=0.0009). A subgroup analysis showed consistency in the OS benefit for patients in either CR or CRi. The median duration of treatment was 12 cycles (1 to 82) for Onureg1 and 6 cycles with placebo (1 to 76).4
Serious adverse reactions occurred in 15% of patients who received Onureg. Serious adverse reactions in ≥2% of patients who received Onureg included pneumonia (8%) and febrile neutropenia (7%). One fatal adverse reaction (sepsis) occurred in a patient who received Onureg. The most common adverse reactions with Onureg versus placebo were nausea (65%, 24%), vomiting (60%, 10%), diarrhea (50%, 21%), fatigue/asthenia (44%, 25%), constipation (39%, 24%), pneumonia (27%, 17%), abdominal pain (22%, 13%) arthralgia (14%, 10%), decreased appetite (13%, 6%), febrile neutropenia (12%, 8%), dizziness (11%,
About Onureg ®
Onureg, the first and only FDA-approved continued AML treatment for patients in first remission, is a once daily oral hypomethylating agent that incorporates into DNA and RNA. The main mechanism of action is thought to be hypomethylation of DNA, as well as direct cytotoxicity to abnormal hematopoietic cells in the bone marrow. Hypomethylation may restore normal function to genes that are critical for cell differentiation and proliferation.9,10 Onureg is approved in the U.S. for continued treatment of adult patients with AML who achieved first CR or CRi following intensive induction chemotherapy and are not able to complete intensive curative therapy. Onureg is also approved in Canada as maintenance therapy for adult patients with AML who achieved CR or CRi following induction therapy with or without consolidation treatment, and who are not eligible for HSCT.
Please see full Prescribing Information for ONUREG.
For more information about Bristol Myers Squibb, visit us at BMS.com
Onureg® (azacitidine tablets; CC-486)
ONUREG® is the first FDA-approved continued treatment for people with AML who are in first remission
ONUREG® is a prescription medicine used for continued treatment of adults with acute myeloid leukemia (AML) who:
- had a first complete remission (CR) following intensive induction chemotherapy with or without recovery of your blood cell counts, and
- who are not able to complete intensive curative therapy.
It is not known if ONUREG® is safe and effective in children under 18 years of age.
EMA advisory committee backs Bristol Myers' Onureg in Acute Myeloid Leukemia
Apr. 23, 2021 7:11 AM ET Bristol-Myers Squibb Company (BMY)
By: Mamta Mayani, SA News Editor1 Comment
- The Committee for Medicinal Products for Human Use (CHMP) of the EMA has recommended approval of Bristol Myers Squibb's Onureg (azacitidine tablets; CC-486) as a maintenance therapy in adult patients with acute myeloid leukemia (AML).
- https://seekingalpha.com/news/3685010-ema-advisory-committee-backs-bristol-myers-onureg-in-acute-myeloid-leukemia
- https://seekingalpha.com/symbol/BMY
- https://www.bms.com/
- https://www.onureg.com/
biotecMAX™ April 2021 Insight
PADCEV® (enfortumab vedotin-ejfv)
Seagen and Astellas Announce U.S. FDA Acceptance of Two Supplemental Biologics License Applications for PADCEV® (enfortumab vedotin-ejfv) in Locally Advanced or Metastatic Urothelial Cancer
04/19/2021
- Priority Review Granted with Action Date of August 17 -
- Australia and Canada Regulators Will Review Applications as Part of FDA’s Project Orbis -
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 the U.S. Food and Drug Administration (FDA) filed two supplemental Biologics License Application (sBLA) submissions for PADCEV® (enfortumab vedotin-ejfv) for review as part of the Real-Time Oncology Review (RTOR) pilot program. The applications were granted Priority Review, with a target action date of August 17, 2021. The review of both applications will also be conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence.
The FDA’s RTOR program aims to explore a more efficient review process to ensure that safe and effective treatments are available to patients as early as possible. Project Orbis provides a framework for concurrent submission and review of oncology drugs among participating international partners. The first sBLA is based on the phase 3 EV-301 trial and seeks to convert PADCEV’s accelerated approval to regular approval. The second sBLA, based on the pivotal trial EV-201’s cohort 2, requests an expansion of the current indication to include patients with locally advanced or metastatic urothelial cancer who have been previously treated with a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor and are ineligible for cisplatin. Results from EV-301 were published in the New England Journal of Medicine.Results from EV-301 and EV-201 cohort 2 were presented at the 2021 American Society of Clinical Oncology Genitourinary Cancers Symposium.
“With our recent regulatory submissions, we intend to provide the highest level of clinical evidence supporting PADCEV use – overall survival data from a randomized phase 3 trial – and expand availability in multiple countries where there is unmet medical need,” said Andrew Krivoshik, M.D., Ph.D., Senior Vice President and Oncology Therapeutic Area Head, Astellas.
About the EV-301 Trial
The EV-301 trial (NCT03474107) is a global, multicenter, open-label, randomized phase 3 trial designed to evaluate enfortumab vedotin versus physician's choice of chemotherapy (docetaxel, paclitaxel or vinflunine) in approximately 600 patients with locally advanced or metastatic urothelial cancer who were previously treated with a PD-1/L1 inhibitor and a platinum-based therapy.3 The primary endpoint is overall survival and secondary endpoints include progression-free survival, overall response rate, duration of response and disease control rate, as well as assessment of safety/tolerability and quality-of-life parameters.
About the EV-201 Trial
The EV-201 trial (NCT03219333) is a single-arm, dual-cohort, pivotal phase 2 clinical trial of enfortumab vedotin for patients with locally advanced or metastatic urothelial cancer who have been previously treated with a PD-1 or PD-L1 inhibitor, including those who have also been treated with a platinum-containing chemotherapy (cohort 1) and those who have not received a platinum-containing chemotherapy in this setting and who are ineligible for cisplatin (cohort 2). The trial enrolled 128 patients in cohort 1 and 91 patients in cohort 2 at multiple centers internationally.4 The primary endpoint is confirmed objective response rate per blinded independent central review. Secondary endpoints include assessments of duration of response, disease control rate, progression-free survival, overall survival, safety and tolerability.
For more information, please visit our website at https://www.astellas.com/en.
For more information on our marketed products and robust pipeline, visit www.seagen.com
About the Astellas and Seagen Collaboration
Astellas and Seagen are co-developing enfortumab vedotin under a collaboration that was entered into in 2007 and expanded in 2009.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210419005204/en/
FDA accepts Seagen, Astellas' enfortumab vedotin applications in urothelial cancer
Apr. 19, 2021 8:37 AM ET Seagen Inc. (SGEN) By: Mamta Mayani, SA News Editor
- Under Real-Time Oncology Review (RTOR) pilot program, FDA has accepted for review Seagen (NASDAQ:SGEN) and Astellas Pharma's (OTCPK:ALPMF) two supplemental Biologics License Applications (sBLA) for PADCEV (enfortumab vedotin-ejfv) in locally advanced or metastatic urothelial cancer.
- The applications were granted Priority Review, with a target action date of August 17, 2021.
- https://seekingalpha.com/news/3682887-fda-accepts-seagen-astellas-enfortumab-vedotin-applications-in-urothelial-cancer
- https://seekingalpha.com/symbol/ALPMF
- https://seekingalpha.com/symbol/SGEN
- www.seagen.com
- https://www.padcev.com/
Filgotinib
Gilead Sciences submits new drug application in Japan for filgotinib for the treatment of ulcerative colitis with an inadequate response to conventional therapies
Application is based on Phase 2b/3 SELECTION study data with patients with moderately to severely active ulcerative colitis
Mechelen, Belgium; 23 April 2021; 06.01 CET; – Galapagos NV (Euronext & Nasdaq: GLPG) today report that their collaboration partner Gilead Sciences K.K. (“Gilead”) and Eisai Co., Ltd. (“Eisai”) today announced that Gilead submitted an application to Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) for approval of filgotinib for an additional indication to treat patients with moderately to severely active ulcerative colitis (UC). Filgotinib is a new oral Janus kinase (JAK) inhibitor approved in Japan in September 2020 for the treatment of rheumatoid arthritis.
This latest regulatory submission is based on data from the randomized, double-blind, placebo-controlled Phase 2b/3 SELECTION study evaluating the efficacy and safety of filgotinib for the induction and maintenance of remission in patients with moderately to severely active ulcerative colitis who have had an inadequate response with, lost response to, or were intolerant to either conventional therapy or a biologic agent. This study showed a statistically significant higher proportion of patients treated with filgotinib 200mg once daily achieved clinical remission at week 10 and maintained remission at week 58 compared with placebo. No new safety risks were identified.
About filgotinib
Filgotinib is approved and marketed as Jyseleca (200mg and 100mg tablets) in the Europe Union, Great Britain and Japan for the treatment of adults with moderately to severely active rheumatoid arthritis (RA) who have responded inadequately or are intolerant to one or more disease modifying anti-rheumatic drugs (DMARDs). Filgotinib may be used as monotherapy or in combination with methotrexate (MTX). This definition from the European Summary of Product Characteristics for filgotinib, which includes contraindications and special warnings and precautions, is available at www.ema.europa.eu. The interview form from the Japanese Ministry of Health, Labour and Welfare is available at www.info.pmda.go.jp. The Great Britain Summary of Product Characteristics is available at www.medicines.org.uk/emc. Applications have been submitted to the EMA, the MHRA and the PMDA for the treatment of adults with moderately to severely active ulcerative colitis who have had an inadequate response with, lost response to, or were intolerant to either conventional therapy or a biologic agent and are currently under review. Filgotinib is not approved in any other countries.
About the filgotinib collaboration
Gilead and Galapagos NV are collaborative partners in the global development and commercialization of filgotinib. Galapagos will be responsible for the commercialization of filgotinib in Europe (transition anticipated to be completed by end of 2021), while Gilead will remain responsible for filgotinib outside of Europe, including in Japan, where filgotinib is co-marketed with Eisai. Filgotinib in UC has been filed in Europe and Japan and a global Phase 3 program is ongoing in Crohn’s Disease. More information about clinical trials can be accessed at www.clinicaltrials.gov.
More information at www.glpg.com.
https://www.glpg.com/filgotinib
Gilead Sciences submits filgotinib application in Japan for ulcerative colitis
Apr. 23, 2021 2:54 AM ET
By: Mamta Mayani, SA News Editor3 Comments
- Galapagos' (NASDAQ:GLPG) collaboration partner Gilead Sciences (NASDAQ:GILD) has submitted an application to Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) for approval of filgotinib for an additional indication to treat moderately to severely active ulcerative colitis (UC).
- https://seekingalpha.com/news/3684954-gilead-sciences-submits-filgotinib-application-in-japan-for-ulcerative-colitis
- https://seekingalpha.com/symbol/GLPG
- https://seekingalpha.com/symbol/GILD
- https://www.gilead.com/
Jemperli (dostarlimab)
APRIL 22, 20211:34 PMUPDATED 39 MINUTES AGO
BRIEF-FDA Granted Accelerated Approval Of Jemperli (Dostarlimab) To GlaxoSmithKline
By Reuters Staff April 22 (Reuters) - FDA:
* FDA APPROVES IMMUNOTHERAPY FOR ENDOMETRIAL CANCER WITH SPECIFIC BIOMARKER
AnaptysBio is trading higher after FDA approval for Jemperli in endometrial cancer
Apr. 22, 2021 1:20 PM ET AnaptysBio, Inc. (ANAB) By: Dulan Lokuwithana, SA News Editor
- The FDA has approved Jemperli (dostarlimab) for the treatment of adults with recurrent or advanced endometrial cancer in a second-line setting, according to the regulator’s website.
- AnaptysBio (ANAB +2.6%) and GlaxoSmithKline (GSK -1.4%) have a collaboration agreement for commercialization of dostarlimab.
- https://seekingalpha.com/news/3684724-anaptysbio-is-trading-higher-after-fda-approval-for-jemperli-in-endometrial-cancer
- https://www.gsk.com/en-gb/
- https://seekingalpha.com/symbol/GSK
- https://www.anaptysbio.com/
- https://seekingalpha.com/symbol/ANAB
- https://www.anaptysbio.com/pipeline/overview/
- https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/761174s000lbl.pdf
23 April 2021
European Commission approves GSK’s JEMPERLI (dostarlimab), the first anti-PD-1 therapy approved for recurrent or advanced endometrial cancer
For media and investors only
Issued: London UK
- European Commission approves GSK’s JEMPERLI (dostarlimab), the first anti-PD-1 therapy approved for recurrent or advanced endometrial cancer
GlaxoSmithKline (LSE/NYSE: GSK) plc today announced the European Commission has granted conditional marketing authorisation for JEMPERLI (dostarlimab), a programmed death receptor-1 (PD-1)-blocking antibody, for use in women with mismatch repair-deficient (dMMR)/microsatellite instability-high (MSI-H) recurrent or advanced endometrial cancer who have progressed on or following prior treatment with a platinum containing regimen. The approval makes dostarlimab the first anti-PD-1 therapy available for endometrial cancer in Europe.
About JEMPERLI (dostarlimab)
Dostarlimab is a programmed death receptor-1 (PD-1)-blocking antibody that binds with high affinity to the PD-1 receptor and blocks its interaction with the ligands PD-L1 and PD-L2.[iii] In addition to GARNET, dostarlimab is being investigated in other registrational-enabling studies, as monotherapy and as part of combination regimens for women with recurrent or primary advanced endometrial cancer stage III or IV non-mucinous epithelial ovarian cancer for patients with advanced solid tumours or metastatic cancer.
Dostarlimab was discovered by AnaptysBio and licensed to TESARO, Inc., under a Collaboration and Exclusive License Agreement signed in March 2014. The collaboration has resulted in three monospecific antibody drugs that have progressed into the clinic. These are: dostarlimab (GSK4057190), a PD-1 antagonist; cobolimab, (GSK4069889), a TIM-3 antagonist; and GSK4074386, a LAG-3 antagonist. GSK is responsible for the ongoing research, development, commercialisation, and manufacture of each of these products under the Agreement.
Important Information for JEMPERLI in the EU
Indication
JEMPERLI is indicated as monotherapy for the treatment of adult patients with mismatch repair deficient (dMMR)/microsatellite instability‑high (MSI‑H) recurrent or advanced endometrial cancer (EC) that has progressed on or following prior treatment with a platinum‑containing regimen.
GlaxoSmithKline wins EU approval for Jemperli in endometrial cancer
Apr. 23, 2021 10:49 AM ET
By: Dulan Lokuwithana, SA News Editor
- Only a day after its regulatory approval in the U.S., GlaxoSmithKline (GSK -0.1%) announces that the European Commission has granted the conditional marketing authorization for Jemperli (dostarlimab) for endometrial cancer in a second-line setting.
https://seekingalpha.com/symbol/GSK
Oral semaglutide (7 mg and 14 mg)
09:52
21 April 2021
Download
Download pdf
Novo Nordisk to initiate phase 3a development in obesity with oral semaglutide
Bagsværd , De n mark, 21 April 2021 – Novo Nordisk today announced the decision to enter phase 3a development in obesity with oral semaglutide 50 mg. The decision follows the completion of the STEP phase 3a clinical programme with once-weekly subcutaneous (sc) semaglutide 2.4 mg.
Novo Nordisk intends to initiate a pivotal phase 3a programme with approximately 1,000 people with obesity or overweight with comorbidities. The global 68-week trial is planned for initiation in the second half of 2021 and will investigate the efficacy and safety of oral semaglutide compared to placebo.
“There is a significant unmet medical need within obesity treatment today. With oral semaglutide we aim to introduce a convenient and effective treatment option for people with obesity and healthcare providers enabling broader use of anti-obesity medication,” said Martin Holst Lange, executive vice president, Development at Novo Nordisk. “As a complement to our injectable anti-obesity medications, oral semaglutide has the potential to help more people living with obesity achieve weight loss goals and improve their health.”
About semaglutide 2.4 mg for weight management and STEP
Once-weekly sc semaglutide 2.4 mg is currently under regulatory review in the US and the EU as a treatment for adults with obesity. Semaglutide is an analogue of the human glucagon-like peptide-1 (GLP-1) hormone. It induces weight loss by reducing hunger, increasing feeling of fullness and thereby helping people eat less and reduce their calorie intake.
The submissions for regulatory review are based on the results from the STEP (Semaglutide Treatment Effect in People with obesity) phase 3 clinical development programme. The global clinical phase 3a programme consisted of four trials and enrolled approximately 4,500 adults with overweight or obesity.
About oral semaglutide
Oral semaglutide (7 mg and 14 mg) is approved as an adjunct to diet and exercise to improve glycaemic control in adults with type 2 diabetes in the US, EU and Japan under the trade name, Rybelsus®. The approval of Rybelsus® is based on the results from 10 clinical trials which included 9,543 adults with type 2 diabetes. Rybelsus® demonstrated a safe and well-tolerated profile across the clinical trials, with the most common adverse event being mild to moderate nausea which diminished over time.
For more information, visit novonordisk.com
https://www.novonordisk-us.com/
Novo Nordisk to begin late-stage trial for oral semaglutide in obesity
Apr. 21, 2021 11:55 AM ET Novo Nordisk A/S (NVO)
By: Dulan Lokuwithana, SA News Editor
- Novo Nordisk (NVO +0.8%) announced its plans to initiate a global Phase 3 trial in H2 2021 for oral semaglutide 50 mg in obesity.
- The pivotal Phase 3a program is set to enroll ~1K people with obesity or overweight with comorbidities.
- https://seekingalpha.com/news/3684032-novo-nordisk-to-begin-late-stage-trial-for-oral-semaglutide-in-obesity
- https://seekingalpha.com/symbol/NVO
- https://www.ozempic.com/
FINTEPLA® (Fenfluramine) Oral Solution
Zogenix Presents New Data at Virtual AAN 2021 Showing Improved Executive Function in Lennox-Gastaut Syndrome (LGS) Patients Treated with FINTEPLA® (Fenfluramine) Oral Solution
04.22.21PDF Version
- LGS is one of the most challenging developmental and epileptic encephalopathies to manage because of high seizure frequency and significant neurodevelopmental impairment.
- More LGS study patients treated with FINTEPLA (fenfluramine) showed improvement in each of the BRIEF 2 indexes that have been used to assess behavior, emotion, and cognitive function in intractable epilepsy and other developmental conditions.
- Improvement was statistically significant for Cognition (27% vs 13% placebo, p=0.046) and the Global Executive Composite score (25% vs 11% placebo, p=0.034), where approximately twice as many patients achieved clinically meaningful improvement.
EMERYVILLE, Calif., April 22, 2021 (GLOBE NEWSWIRE) -- Zogenix (NASDAQ: ZGNX), a global biopharmaceutical company developing rare disease therapies, today announced new data showing the positive impact of treatment with FINTEPLA® (fenfluramine) oral solution on everyday executive function for children and young adults with Lennox-Gastaut syndrome (LGS). FINTEPLA is being investigated for the treatment of seizures associated with LGS, a rare, severe epilepsy that is one of the most challenging developmental and epileptic encephalopathies to manage due to the high seizure frequency and significant neurodevelopmental impairment patients experience.
The data, presented in an oral presentation and fireside chat at the virtual American Academy of Neurology (AAN) Annual Meeting this week, was the first presentation of FINTEPLA’s impact on LGS patients’ abilities to self-regulate behavior, emotions, and cognition. These new data complement earlier Phase 3 trial data demonstrating FINTEPLA’s ability to significantly reduce drop seizure frequency and, especially, generalized tonic-clonic seizure frequency (a primary risk factor for sudden unexpected death in epilepsy, or SUDEP) for many LGS patients.
FINTEPLA® (Fenfluramine) Oral Solution
Results
Mean age of patients in the FINTEPLA (n=92) vs. placebo (n=45) groups was 12 ± 3.6, with 44% of the FINTEPLA patients being female vs. 53% in the placebo group. The frequency of clinically elevated T-scores at baseline, defined as a T-score ≥65, was substantial, suggesting frequent impairment in executive function, as would be expected for children with LGS. Subjects with greater improvement in seizure control were more likely to show clinically meaningful improvements in executive functioning after the 14-week study duration.
More patients on FINTEPLA (fenfluramine) than placebo showed improvement in each of the four BRIEF 2 indexes (Behavior, Emotions, Cognition, and Global Executive Composite). Improvement was significantly greater for the CRI (Cognition) and GEC (Global Executive Composite) scores, indicating clinically meaningful improvement in the FINTEPLA (fenfluramine)-treated patients.
Presentation
Dr. Kim Bishop presented and discussed the data during Virtual AAN 2021; the slides from her presentation are archived on the Zogenix Newsroom.
INDICATIONS AND USAGE
FINTEPLA is indicated for the treatment of seizures associated with Dravet syndrome in patients 2 years of age and older.
Zogenix's Fintepla shows improved executive function in rare, severe epilepsy
Apr. 22, 2021 8:17 AM ET Zogenix, Inc. (ZGNX) By: Mamta Mayani, SA News Editor
- Zogenix (NASDAQ:ZGNX) announces new data showing the positive impact of treatment with Fintepla (fenfluramine) oral solution on everyday executive function for children and young adults with Lennox-Gastaut syndrome (LGS), a rare, severe epilepsy.
- https://seekingalpha.com/news/3684506-zogenixs-fintepla-shows-improved-executive-function-in-rare-severe-epilepsy
- https://seekingalpha.com/symbol/ZGNX
- https://www.zogenix.com/
- https://www.zogenix.com/pipeline/
ENHERTU® ▼ (TRASTUZUMAB DERUXTECAN)
ENHERTU® ▼ (TRASTUZUMAB DERUXTECAN) NOW AVAILABLE VIA THE CANCER DRUGS FUND FOR THE TREATMENT OF HER2 POSITIVE METASTATIC BREAST CANCER
For UK media ONLY
- NICE decision based on results from the DESTINY-Breast01 trial
- Reimbursement decisions in Scotland, Wales, and Northern Ireland to be announced later this year
April 20, 2021 04:01 AM Eastern Daylight Time
UXBRIDGE, England & CAMBRIDGE, England--(BUSINESS WIRE)--Daiichi Sankyo UK, Limited (hereafter, Daiichi Sankyo) and AstraZeneca UK today welcome the news that the National Institute for Health and Care Excellence (NICE) has recommended Enhertu® (trastuzumab deruxtecan) for use within the Cancer Drugs Fund (CDF) as an option for treating HER2 positive unresectable or metastatic breast cancer in adults who have received two or more prior anti-HER2 based therapies.
ENHERTU® ▼ (TRASTUZUMAB DERUXTECAN)
About DESTINY-Breast01
DESTINY-Breast01 was a phase 2, single-arm, open-label, global, multicentre, two-part trial evaluating the safety and efficacy of trastuzumab deruxtecan in patients with HER2 positive unresectable and/or metastatic breast cancer who had received two or more prior anti-HER2-based regimens, including trastuzumab emtansine (100%), trastuzumab (100%), and pertuzumab (65.8%). The primary endpoint of the trial was confirmed ORR, as determined by independent central review. Key secondary objectives were disease control rate, clinical-benefit rate, duration of response, progression-free survival, and safety.
About trastuzumab deruxtecan
Trastuzumab deruxtecan is a HER2 directed antibody drug conjugate (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 programme in AstraZeneca’s ADC scientific platform.
ADCs are targeted cancer medicines that deliver cytotoxic chemotherapy (‘payload’) to cancer cells via a linker attached to a monoclonal antibody that binds to a specific target expressed on cancer cells. Trastuzumab deruxtecan is comprised of a humanised anti-HER2 IgG1 monoclonal antibody with the same amino acid sequence as trastuzumab attached to a topoisomerase I inhibitor payload, an exatecan derivative, via a tetrapeptide-based cleavable linker.
About the collaboration between Daiichi Sankyo and AstraZeneca
Daiichi Sankyo and AstraZeneca entered into a global collaboration to jointly develop and commercialise trastuzumab deruxtecan in March 2019, except in Japan where Daiichi Sankyo maintains exclusive rights. Daiichi Sankyo is responsible for the manufacturing and supply of trastuzumab deruxtecan.
For more information, please visit www.daiichi-sankyo.co.uk
For more information, please visit www.astrazeneca.co.uk
AstraZeneca's Enhertu now available in U.K. for breast cancer
Apr. 20, 2021 4:52 AM ET AstraZeneca PLC (AZN) By: Mamta Mayani, SA News Editor
- AstraZeneca (NASDAQ:AZN) and Daiichi Sankyo (OTCPK:DSKYF) announce that the National Institute for Health and Care Excellence (NICE) has recommended Enhertu (trastuzumab deruxtecan) for use within the Cancer Drugs Fund (CDF) as an option for treating HER2 positive unresectable or metastatic breast cancer in adults who have received two or more prior anti-HER2 based therapies.
- https://seekingalpha.com/news/3683228-astrazenecas-enhertu-now-available-in-uk-for-breast-cancer
- https://seekingalpha.com/symbol/DSKYF
- https://seekingalpha.com/symbol/AZN
What is ENHERTU?
ENHERTU (en-HER-too) is a prescription medicine used in adults to treat human epidermal growth factor receptor 2 (HER2)-positive:
- Breast cancer that cannot be removed by surgery or that has spread to other parts of your body (metastatic), and who have received two or more prior anti-HER2 breast cancer treatments.
ENHERTU was FDA approved for this use based on a clinical study that measured how many patients responded and how long they responded. ENHERTU is still being studied to confirm these results. - Stomach cancer called gastric or gastroesophageal junction (GEJ) adenocarcinoma that has spread to areas near your stomach (locally advanced) or that has spread to other parts of your body (metastatic), and who have received a prior trastuzumab-based regimen.
It is not known if ENHERTU is safe and effective in children.
Amgen's Investigational Targeted Treatment Bemarituzumab Granted Breakthrough Therapy Designation
Potential First-in-Class Therapy for a Subset of Gastric and Gastroesophageal Cancers That Overexpress Fibroblast Growth Factor Receptor 2 (FGFR2b)
Designation is Supported by Results From the Phase 2 FIGHT Trial
THOUSAND OAKS, Calif., April 19, 2021 /PRNewswire/ -- Amgen (NASDAQ:AMGN) today announced that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for investigational bemarituzumab as first-line treatment for patients with fibroblast growth factor receptor 2b (FGFR2b) overexpressing and human epidermal growth factor receptor 2 (HER2)-negative metastatic and locally advanced gastric and gastroesophageal (GEJ) adenocarcinoma in combination with modified FOLFOX6 (fluoropyrimidine, leucovorin, and oxaliplatin), based on an FDA-approved companion diagnostic assay showing at least 10% of tumor cells overexpressing FGFR2b.
"The FIGHT trial is the first study to evaluate targeting the overexpression of FGFR2b in cancer. Bemarituzumab demonstrated clinically meaningful outcomes in key endpoints for patients with advanced gastric or gastroesophageal cancer as a frontline therapy," said David M. Reese, M.D., executive vice president of Research and Development at Amgen. "Amgen looks forward to further investigating the role of FGFR2b and will continue to work with regulatory agencies on next steps to bring this potential first-in-class, frontline therapy to patients."
More than one million new gastric cancer cases are diagnosed annually, and gastric cancer is particularly prevalent in Asia.1,2 Approximately 80 to 85% of patients with advanced gastric and GEJ cancers are HER2-negative, and approximately 30% of these patients present with FGFR2b overexpression.3,4
Amgen acquired Five Prime Therapeutics on April 16, 2021. In addition to bemarituzumab, Five Prime's pipeline complements Amgen's efforts to bring innovative therapies to oncology patients.
About Bemarituzumab
Bemarituzumab (anti-FGFR2b) is a potential first-in-class investigational targeted antibody that is designed to block fibroblast growth factors (FGFs) from binding and activating FGFR2b, inhibiting several downstream pro-tumor signaling pathways and potentially slowing cancer progression. Bemarituzumab is being developed in gastric and GEJ cancer as a targeted therapy for tumors that overexpress FGFR2b. The company is also evaluating the potential for bemarituzumab in other cancers that overexpress FGFR2b.
Zai Lab (Shanghai) Co. Ltd. was granted an exclusive license to develop and commercialize bemarituzumab in Greater China, and Zai Lab collaborated with Five Prime on the Phase 2 FIGHT trial in Greater China.
please visit AmgenOncology.com
For more information, visit www.amgen.com
View original content to download multimedia:http://www.prnewswire.com/news-releases/amgens-investigational-targeted-treatment-bemarituzumab-granted-breakthrough-therapy-designation-301271859.html
SOURCE Amgen
FDA grants Amgen's bemarituzumab Breakthrough Therapy designation
Apr. 19, 2021 4:25 PM ET Amgen Inc. (AMGN) By: Jonathan M Block, SA News Editor
- The FDA has granted Breakthrough Therapy designation to Amgen's (NASDAQ:AMGN) bemarituzumab for certain types of gastric and gastroesophageal ("GEJ") adenocarcinomas.
- https://seekingalpha.com/news/3683144-fda-grants-amgens-bemarituzumab-breakthrough-therapy-designation
- https://seekingalpha.com/symbol/AMGN
- https://www.amgen.com/
A Study of Bemarituzumab (FPA144) Combined With Modified FOLFOX6 (mFOLFOX6) in Gastric/Gastroesophageal Junction Cancer (FIGHT) (FIGHT)
https://clinicaltrials.gov/ct2/show/NCT03694522
Jazz Pharmaceuticals Presents Phase 3 Study Results of Xywav™ (calcium, magnesium, potassium, and sodium oxybates) Oral Solution in Adult Patients with Idiopathic Hypersomnia at 2021 American Academy of Neurology Annual Meeting
April 20, 2021Download PDFXywav demonstrated statistically significant differences in change in Epworth Sleepiness Scale score (p-value <0.0001), Patient Global Impression of Change (p-value <0.0001) and the Idiopathic Hypersomnia Severity Scale (p-value <0.0001)
The safety profile in this study was consistent with the known safety profile of Xywav with no new safety signals observed in this population
Company to host investor webcast today at 12:30 p.m. ET
DUBLIN, April 20, 2021 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced positive results from the Phase 3 study of XywavTM (calcium, magnesium, potassium, and sodium oxybates) oral solution in adult patients with idiopathic hypersomnia, which will be presented during the Clinical Trials Plenary Session of the 2021 American Academy of Neurology (AAN) Annual Meeting between 10:00 a.m. and 12:30 p.m. ET. Jazz will host an investor and analyst presentation via webcast today at 12:30 p.m. ET.
Today's presentation will further quantify the previously reported Phase 3 top-line results. These additional data were submitted to the U.S. Food and Drug Administration (FDA) in the supplemental New Drug Application that was recently accepted for filing and granted Priority Review.
About the Phase 3 Study in Idiopathic Hypersomnia
The Phase 3 study of Xywav was a multi-national, double-blind, multicenter, placebo-controlled, randomized withdrawal study evaluating the efficacy and safety of an investigational use of Xywav in adult patients with idiopathic hypersomnia. Patients entering the study had excessive daytime sleepiness typical of the idiopathic hypersomnia population. The primary endpoint was the change in the ESS score from Xywav and placebo over the randomized-withdrawal period. The key secondary endpoints were PGIc and IHSS. IHSS is a recently developed and validated scale, and is a self-report measure of hypersomnolence symptoms, consequences, and responsiveness to treatment.8
The study design included a titration and optimization period of up to 14 weeks, a Xywav stable-dose period of two weeks, followed by a 1:1 randomization to either Xywav or placebo for 2 weeks. More information about the study design is available at www.clinicaltrials.gov (identifier: NCT03533114).
More information about Xywav, including Full Prescribing Information and Medication Guide, is available here. <http://pp.jazzpharma.com/pi/xywav.en.USPI.pdf>
About Xywav™ (calcium, magnesium, potassium, and sodium oxybates) oral solution
Xywav, also known as JZP-258, is a lower-sodium oxybate approved by the U.S. Food and Drug Administration (FDA) for the treatment of cataplexy or excessive daytime sleepiness in patients 7 years of age and older with narcolepsy. It is an investigational product being evaluated for the treatment of idiopathic hypersomnia in adult patients. While the exact mechanism of action of Xywav is unknown, it is hypothesized that the therapeutic effects of Xywav on cataplexy and excessive daytime sleepiness are mediated through GABAB actions during sleep at noradrenergic and dopaminergic neurons, as well as at thalamocortical neurons.16 Xywav received Fast Track designation by the FDA in September 2020 for the treatment of idiopathic hypersomnia.
For more information, please visit www.jazzpharmaceuticals.com
View original content to download multimedia:http://www.prnewswire.com/news-releases/jazz-pharmaceuticals-presents-phase-3-study-results-of-xywav-calcium-magnesium-potassium-and-sodium-oxybates-oral-solution-in-adult-patients-with-idiopathic-hypersomnia-at-2021-american-academy-of-neurology-annual-meeting-301272417.html
SOURCE Jazz Pharmaceuticals plc
Jazz Pharma reports positive Xywav data in neurologic sleep disorder
Apr. 20, 2021 8:16 AM ETJazz Pharmaceuticals plc (JAZZ)By: Mamta Mayani, SA News Editor
- Jazz Pharmaceuticals (NASDAQ:JAZZ) announces positive results from Phase 3 study of Xywav (calcium, magnesium, potassium, and sodium oxybates) oral solution in adult patients with idiopathic hypersomnia.
- https://seekingalpha.com/news/3683341-jazz-pharma-reports-positive-xywav-data-in-neurologic-sleep-disorder
- https://seekingalpha.com/symbol/JAZZ
- https://www.jazzpharma.com/
TYSABRI® (natalizumab) / VUMERITY® (diroximel fumarate)
NEW DATA AT AAN 2021 FROM ACROSS BIOGEN’S MS PORTFOLIO DEMONSTRATE POSITIVE IMPACT OF TREATMENT ON PEOPLE LIVING WITH RELAPSING MULTIPLE SCLEROSIS
April 16, 2021 at 7:30 AM EDT
- New findings from MS PATHS show that treatment with TYSABRI® (natalizumab) can lead to meaningful improvements in mental and social health compared to Ocrevus® (ocrelizumab)
- Real-world data from VUMERITY® (diroximel fumarate) reinforce the treatment’s positive gastrointestinal tolerability profile
- Biogen advances leading research to help inform future patient management including new information on the clinical profile of extended interval dosing with natalizumab
CAMBRIDGE, Mass., April 16, 2021 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) today announced new data from its industry-leading portfolio of multiple sclerosis (MS) therapies to be presented at the American Academy of Neurology (AAN) 2021 Virtual Annual Meeting, April 17-22. The presentations include data on quality of life benefits and analyses of extended interval dosing (EID) with TYSABRI® (natalizumab) as well as new real-world experience data from VUMERITY® (diroximel fumarate). The research adds to the vast clinical knowledge Biogen continues to advance as part of its commitment to the care of people living with MS.
“With chronic conditions like MS, where every patient has a different experience with the disease, it is critically important to understand how treatment impacts their daily living and quality of life,” said Maha Radhakrishnan, M.D., Chief Medical Officer at Biogen. “These data show that the benefits TYSABRI provides in terms of a patient’s quality of life are substantial and that the positive gastrointestinal tolerability profile of VUMERITY can help people with relapsing MS continue with treatment, which is essential to delay its progression.”
Analyses Demonstrate Improved Quality of Life Outcomes with TYSABRI and Further Evaluate Extended Interval Dosing
To better understand clinically meaningful quality of life benefits following treatment with TYSABRI, MS PATHS (Partners Advancing Technology and Health Solutions) researchers analyzed patient reported data on 12 different domains on the Neuro-QoL (Quality of Life in Neurological Disorders) questionnaire such as sleep disturbance, anxiety, fatigue, depression and participation in daily activities. Results included:
- In people treated with TYSABRI or Ocrevus® (ocrelizumab) with baseline impairment, statistically significant improvements were seen in 10 of 12 and 8 of 12 Neuro-QoL domains, respectively. In 11 of 12 domains on the Neuro-QoL questionnaire, the adjusted annualized rate of improvement was greater with TYSABRI as compared to Ocrevus.
- The difference between the two therapies was statistically significant in favor of TYSABRI in three of the domains: satisfaction with social roles and activities (p=0.02), participation in social roles and activities (p=0.0001) and emotional and behavioral dyscontrol (p=0.01).
About TYSABRI® (natalizumab)
TYSABRI is a well-established relapsing multiple sclerosis (RMS) treatment indicated for relapsing forms of MS in adults that has been proven in clinical trials to slow physical disability progression, reduce the formation of new brain lesions and cut relapses. TYSABRI is approved in 80 countries, and over 220,000 people worldwide have been treated with TYSABRI, with over 880,000 patient-years of experience, based on clinical trials and prescription data.1
TYSABRI increases the risk of progressive multifocal leukoencephalopathy (PML), a rare opportunistic viral infection of the brain which has been associated with death or severe disability. Risk factors that increase the risk of PML are the presence of anti-JC virus antibodies, prior immunosuppressant use and longer TYSABRI treatment duration. Patients who have all three risk factors have the highest risk of developing PML. When initiating and continuing treatment with TYSABRI, physicians should consider whether the expected benefit of TYSABRI is sufficient to offset this risk.
TYSABRI also increases the risk of developing encephalitis and meningitis caused by herpes simplex and varicella zoster viruses, and serious, life-threatening and sometimes fatal cases have been reported in the post-marketing setting in MS patients receiving TYSABRI. Clinically significant liver injury, including acute liver failure requiring transplant, has also been reported in the post-marketing setting. Other serious adverse events that have occurred in TYSABRI-treated patients include hypersensitivity reactions (e.g., anaphylaxis), a decrease in lymphocyte counts and infections, including opportunistic and other atypical infections.
Please click here for Important Safety Information, including Boxed Warning, and full Prescribing Information, including Medication Guide for TYSABRI in the U.S., or visit your respective country’s product website.
About VUMERITY® (diroximel fumarate)
VUMERITY is an oral fumarate with a distinct chemical structure from TECFIDERA® (dimethyl fumarate), approved in the U.S. for the treatment of relapsing forms of multiple sclerosis in adults, to include clinically isolated syndrome, relapsing-remitting disease and active secondary progressive disease. Once in the body, VUMERITY rapidly converts to monomethyl fumarate, the same active metabolite of dimethyl fumarate.
VUMERITY is contraindicated in patients with known hypersensitivity to diroximel fumarate, dimethyl fumarate or to any of the excipients of VUMERITY; and in patients taking dimethyl fumarate. Serious side effects for VUMERITY are based on data from dimethyl fumarate (which has the same active metabolite as VUMERITY) and include anaphylaxis and angioedema, progressive multifocal leukoencephalopathy, which is a rare opportunistic viral infection of the brain that has been associated with death or severe disability, a decrease in mean lymphocyte counts during the first year of treatment, herpes zoster and other serious infections, liver injury and flushing. The most common adverse events, obtained using data from dimethyl fumarate (which has the same active metabolite as VUMERITY), were flushing, abdominal pain, diarrhea and nausea.
Please click here for Important Safety Information and full Prescribing Information, including Patient Information for VUMERITY in the U.S.
About TECFIDERA® (dimethyl fumarate)
TECFIDERA, a treatment for relapsing forms of multiple sclerosis (MS) in adults, is the most prescribed oral medication for relapsing MS in the world and has been shown to reduce the rate of MS relapses, slow the progression of disability and impact the number of MS brain lesions, while demonstrating a well-characterized safety profile in people with relapsing forms of MS. TECFIDERA is approved in 69 countries, and more than 500,000 patients have been treated with it, representing more than 950,000 patient-years of exposure across clinical trial use and patients prescribed TECFIDERA. Of these, 6,335 patients (14,241 patient-years) were from clinical trials.2
TECFIDERA is contraindicated in patients with a known hypersensitivity to dimethyl fumarate or any of the excipients of TECFIDERA. Serious side effects include anaphylaxis and angioedema, and cases of progressive multifocal leukoencephalopathy, a rare opportunistic viral infection of the brain which has been associated with death or severe disability, have been seen with TECFIDERA patients in the setting of prolonged lymphopenia although the role of lymphopenia in these cases is uncertain. Other serious side effects include a decrease in mean lymphocyte counts during the first year of treatment, herpes zoster and other serious infections, liver injury and flushing. In clinical trials, the most common adverse events associated with TECFIDERA were flushing, abdominal pain, diarrhea and nausea.
Please click here for Important Safety Information and full Prescribing Information, including Patient Information for TECFIDERA in the U.S., or visit your respective country’s product website.
About PLEGRIDY® (peginterferon beta-1a)
PLEGRIDY is a pegylated interferon dosed once every two weeks for relapsing forms of multiple sclerosis (MS) in adults, the most common form of MS. PLEGRIDY is currently approved in over 60 countries including the U.S., Canada, Australia and Switzerland and across the European Union. Over 61,000 people worldwide have been treated with PLEGRIDY, with over 120,000 patient-years of experience, based on prescription data.3 Biogen continues to work toward making PLEGRIDY available in additional countries across the globe.
The efficacy and safety of PLEGRIDY are supported by one of the largest pivotal studies with interferons conducted in people living with relapsing-remitting MS. In clinical studies, PLEGRIDY has been proven to significantly reduce the rate of MS relapses, slow the progression of disability and reduce the number of MS brain lesions while demonstrating a well-characterized safety profile for patients with relapsing forms of MS. Side effects reported include liver problems, including liver failure and increases in liver enzymes; depression or suicidal thoughts; serious allergic reactions; injection site reactions, cardiac problems, including congestive heart failure; blood problems, such as decreases in white blood cell or platelet counts; autoimmune disorders; and seizures. In clinical trials, the most common adverse events associated with PLEGRIDY were injection site reactions and flu-like symptoms. A list of adverse events can be found in the full PLEGRIDY product labeling for each country where it is approved. PLEGRIDY can be considered for use in relapsing MS patients throughout the full course of pregnancy and during breast-feeding, if clinically needed.
Please click here for Important Safety Information and full Prescribing Information, including Medication Guide for PLEGRIDY in the U.S., or visit your respective country’s product website.
We routinely post information that may be important to investors on our website at www.biogen.com
Biogen’s Tysabri and Vumerity show positive impact in multiple sclerosis
Apr. 16, 2021 7:59 AM ET Biogen Inc. (BIIB) By: Mamta Mayani, SA News Editor
- Biogen (NASDAQ:BIIB) announces new data on quality of life benefits and analyses of extended interval dosing (EID) with TYSABRI (natalizumab) as well as from VUMERITY (diroximel fumarate) in multiple sclerosis (MS).
- In people treated with TYSABRI or Ocrevus (ocrelizumab), statistically significant improvements were seen in 10 of 12 and 8 of 12 Neuro-QoL domains, respectively.
- https://seekingalpha.com/news/3682536-biogens-tysabri-and-vumerity-show-positive-impact-in-multiple-sclerosis
- https://seekingalpha.com/symbol/BIIB
https://www.biogenoptions.com/en_us/home/treatment-overview/tecfidera.html
Opdivo® (nivolumab) in Combination with Chemotherapy
U.S. Food and Drug Administration Approves Opdivo® (nivolumab) in Combination with Chemotherapy for Patients with Advanced or Metastatic Gastric Cancer, Gastroesophageal Junction Cancer, and Esophageal Adenocarcinoma, Regardless of PD-L1 Expression Status
04/16/2021CATEGORY:
Opdivo is the first and only immunotherapy in combination with chemotherapy to deliver superior overall survival versus chemotherapy alone in a trial of this patient population1
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced that Opdivo (nivolumab) (injection for intravenous use), in combination with fluoropyrimidine- and platinum-containing chemotherapy, was approved by the U.S. Food and Drug Administration (FDA) for the treatment of patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma, regardless of PD-L1 expression status.1 The approval is based on the Phase 3 CheckMate -649 trial evaluating Opdivo in combination with mFOLFOX6 (fluorouracil, leucovorin, and oxaliplatin) or CapeOX (capecitabine and oxaliplatin), compared to chemotherapy (mFOLFOX6 or CapeOX) alone.1,2
In the trial of this patient population, Opdivo plus chemotherapy demonstrated superior overall survival (OS) compared to chemotherapy alone, both in all randomized patients (OS HR 0.80; 95% CI: 0.71 to 0.90; P=0.0002), as well as in patients with PD-L1 combined positive score (CPS) ≥ 5 (OS HR 0.71; 95% CI: 0.61 to 0.83; P<0.0001).1 In an exploratory analysis of all patients, 55% of patients on Opdivo in combination with chemotherapy were alive at one year versus 48% of patients on chemotherapy alone.2 The combination also significantly reduced the risk of disease progression or death compared to chemotherapy alone (PD-L1 CPS ≥ 5: progression-free survival (PFS) HR 0.68; 95% CI: 0.58 to 0.79; P<0.0001).1
Opdivo® (nivolumab)
About CheckMate -649
CheckMate -649 is a randomized, multicenter, open-label Phase 3 trial in patients with previously untreated advanced or metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma.1,2 The trial excluded patients who were known human epidermal growth factor receptor 2 (HER2) positive, or had untreated CNS metastases.1 In the trial, patients were randomized to receive Opdivo in combination with chemotherapy (patients with PD-L1 CPS ≥ 5: n=473; all randomized patients: n=789) or chemotherapy alone (patients with PD-L1 CPS ≥ 5: n=482; all randomized patients: n=792).1 Patients received one of the following treatments: Opdivo 240 mg in combination with mFOLFOX6 (fluorouracil, leucovorin and oxaliplatin) every two weeks or mFOLFOX6 every two weeks; or Opdivo 360 mg in combination with CapeOX (capecitabine and oxaliplatin) every three weeks or CapeOX every three weeks.1 Patients were treated until disease progression, unacceptable toxicity, or up to two years.1 The primary endpoints, assessed in patients with PD-L1 CPS ≥ 5, were PFS assessed by Blinded Independent Central Review (BICR) and OS.1 Secondary endpoints included OS and PFS in patients with PD-L1 CPS ≥1 and in all randomized patients, and overall response rate (ORR) as assessed by BICR in patients with PD-L1 CPS ≥ 1 and ≥ 5, and in all randomized patients.1,2
The FDA-approved dosing for Opdivo (injection for intravenous use) for patients with gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma is 360 mg every three weeks (30-minute intravenous infusion) with fluoropyrimidine- and platinum-containing chemotherapy every three weeks or 240 mg every two weeks (30-minute intravenous infusion) with fluoropyrimidine- and platinum-containing chemotherapy every two weeks until disease progression, unacceptable toxicity, or up to two years.1
INDICATION
OPDIVO® (nivolumab), in combination with fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the treatment of patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma.
Please see U.S. Full Prescribing Information for OPDIVO.
FDA approves Bristol's Opdivo as first-line gastric cancer treatment
Apr. 16, 2021 1:09 PM ET Bristol-Myers Squibb Company (BMY) By: Jonathan M Block, SA News Editor12 Comments
- The FDA has approved Bristol-Myers Squibb's (NYSE:BMY) Opdivo (nivolumab)as a first-line therapy for advanced or metastatic gastric cancer.
- https://seekingalpha.com/news/3682658-fda-approves-bristol-myers-squibb-opdivo-as-first-line-gastric-cancer-treatment
- https://seekingalpha.com/symbol/BMY
Sarclisa® (isatuximab)
European Commission approves second indication of Sarclisa® (isatuximab) for relapsed multiple myeloma
European Commission approves second indication of Sarclisa® (isatuximab) for relapsed multiple myeloma
- Approval based on Phase 3 IKEMA study demonstrating Sarclisa added to standard of care carfilzomib and dexamethasone reduced risk of disease progression or death by 47% in patients who had relapsed after one to three prior therapies
- Sarclisa combination therapy was associated with undetectable levels of multiple myeloma (MM) in nearly 30% of patients with relapsed MM
- Second EU approval in less than 12 months for Sarclisa in combination with a standard of care regimen for the treatment of relapsed or refractory MM
PARIS – April 19, 2021 – Today, the European Commission (EC) approved Sarclisa® (isatuximab) in combination with carfilzomib and dexamethasone (Kd) for the treatment of adult patients with relapsed multiple myeloma who have received at least one prior therapy. This marks the second EC approval of Sarclisa in combination with a standard of care regimen in less than 12 months.
“As there is no cure for multiple myeloma and patients often experience disease relapse, we must persist in our pursuit for additional treatment options. Nearly 30% of patients treated with the Sarclisa regimen had a profound response with undetectable levels of multiple myeloma,” said Philippe Moreau, M.D., Department of Hematology, University Hospital of Nantes, France. “This new therapeutic regimen has the potential to become a standard of care for patients with relapsed multiple myeloma, who now have another treatment option earlier in the progression of their disease.”
Sarclisa® (isatuximab)
About Sarclisa
Sarclisa is a monoclonal antibody that binds to a specific epitope on the CD38 receptor on MM cells. It is designed to work through multiple mechanisms of action including programmed tumor cell death (apoptosis) and immunomodulatory activity. CD38 is highly and uniformly expressed on the surface of MM cells, making it a potential target for antibody-based therapeutics such as Sarclisa.
Sarclisa is approved in the EU, U.S., Switzerland, UK, Canada, Australia, Japan, Russia, the UAE, South Korea, Taiwan and Qatar in combination with pom-dex for the treatment of certain adults with relapsed refractory MM. It is also approved in the U.S. in combination with carfilzomib and dexamethasone for the treatment of adult patients with relapsed or refractory MM who have received one to three prior lines of therapy. In the U.S., the generic name for Sarclisa is isatuximab-irfc, with irfc as the suffix designated in accordance with Nonproprietary Naming of Biological Products Guidance for Industry issued by the FDA.
Sarclisa continues to be evaluated in multiple ongoing Phase 3 clinical trials in combination with current standard treatments across the MM treatment continuum. It is also under investigation for the treatment of other hematologic malignancies and solid tumors. The safety and efficacy of these additional uses have not been reviewed by any regulatory authority worldwide.
For more information on Sarclisa clinical trials please visit www.clinicaltrials.gov.
Sanofi's Sarclisa OK'd in European as second indication for multiple myeloma
Apr. 19, 2021 1:50 AM ET Sanofi (SNY) By: Mamta Mayani, SA News Editor
- The European Commission has approved Sanofi's (NASDAQ:SNY) Sarclisa (isatuximab) in combination with carfilzomib and dexamethasone (Kd) for the treatment of adult patients with relapsed multiple myeloma (MM) who have received at least one prior therapy.
- https://seekingalpha.com/news/3682782-sanofis-sarclisa-okd-in-european-as-second-indication-for-multiple-myeloma
- https://seekingalpha.com/symbol/SNY
- https://www.sanofi.com/en
- https://www.sarclisa.com/
DARZALEX® SC (daratumumab injection)
DARZALEX® SC Becomes the First and Only Health Canada-Approved Treatment for Patients with Newly Diagnosed Light Chain (AL) Amyloidosis, A Rare Disease
Mon April 19, 2021 7:30 AM|Canada Newswire|About: JNJ
DARZALEX® SC combination regimen is supported by the Phase 3 ANDROMEDA study demonstrating a significantly higher hematologic complete response rate in this rare and serious blood cell disorder
TORONTO, April 19, 2021 /CNW/ - The Janssen Pharmaceutical Companies of Johnson & Johnson (JNJ) announced today Health Canada approved DARZALEX® SC (daratumumab injection), a subcutaneous (SC) formulation of daratumumab, in combination with bortezomib, cyclophosphamide and dexamethasone (D-VCd, also known as DCyBorD) for the treatment of adult patients with newly diagnosed light chain (AL) amyloidosis.1 The DARZALEX® SC combination regimen is the first and only Health Canada approved treatment for patients with AL amyloidosis,2 a rare blood cell disorder with no cure.3 AL amyloidosis occurs when an abnormal protein produced in the bone marrow is deposited and builds up in vital organs like the heart and kidneys, impacting their ability to function.4
DARZALEX® SC (daratumumab injection)
About DARZALEX® SC
DARZALEX® is the first CD38-directed monoclonal antibody (mAb) approved to treat multiple myeloma.23 In 2020, DARZALEX® SC became the only subcutaneously administered CD38-directed antibody approved to treat patients with multiple myeloma.24 And now, DARZALEX® SC in combination with bortezomib, cyclophosphamide and dexamethasone is the first and only Health Canada approved treatment for patients with AL amyloidosis. 25 The safety and efficacy of DARZALEX® SC have not been established in AL amyloidosis patients with advanced cardiac disease (Mayo Stage IIIB or NYHA Class IIIB or IV).26
Daratumumab binds to CD38, a surface protein highly expressed across clonal plasma cells that cause serious conditions such as multiple myeloma and AL amyloidosis.27 Daratumumab induces tumor cell death through cell lysis via multiple immune-mediated mechanisms of action, including complement-dependent cytotoxicity (CDC), antibody-dependent cellular cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP).28 Daratumumab has also demonstrated immunomodulatory effects such as increasing CD4+ and CD8+ T-cells counts, which may contribute to clinical response.29
In August 2012, Janssen Biotech, Inc. and Genmab A/S entered a worldwide agreement, which granted Janssen an exclusive license to develop, manufacture and commercialize DARZALEX®. Janssen Inc. commercializes DARZALEX® and DARZALEX® SC in Canada. For full Prescribing Information and more information about DARZALEX® SC, please visit www.janssen.com/canada.
Learn more at www.janssen.com/canada.
JNJ's Darzalex combo regimen OK'd in Canada for rare blood cell disorder
Apr. 19, 2021 7:58 AM ETJohnson & Johnson (JNJ)By: Mamta Mayani, SA News Editor
- Health Canada approves Johnson & Johnson (NYSE:JNJ) unit, Janssen Pharmaceutical's DARZALEX SC (daratumumab injection), in combination with bortezomib, cyclophosphamide and dexamethasone for the treatment of adult patients with newly diagnosed light chain (AL) amyloidosis, a rare blood cell disorder with no cure.
- https://seekingalpha.com/news/3682852-jnjs-darzalex-combo-regimen-okd-in-canada-for-rare-blood-cell-disorder
- https://seekingalpha.com/symbol/JNJ
- https://www.darzalex.com/
- https://www.janssen.com/canada/
biotecMAX™ April 2021 Insight
Medtronic Extended infusion set
Medtronic Announces European Launch of World's First Infusion Set with Wear up to Seven Days to Help Reduce Burden for People with Diabetes
DUBLIN, April 14, 2021 /PRNewswire/ -- Medtronic plc (NYSE: MDT), the global leader in medical technology, today introduced in select European countries the Medtronic Extended infusion set — the first and only infusion set that can be worn for up to 7 days. An infusion set is tubing that delivers insulin from the pump to the body and typically requires a set change every few days.
This innovation doubles the length of time an infusion set can be worn so users can safely stay on insulin pump therapy with fewer interruptions and insertions1 while introducing enhanced convenience and comfort2 to their diabetes management routine. The Medtronic Extended infusion set recently received positive feedback about the experience from more than 100 insulin pump users in Finland who had a chance to use this product as part of a patient evaluation program. The set is compatible with all MiniMed™ 600 and 700 series insulin pumps.
"It is currently recommended that insulin infusion sets be changed every three days," said Bruce Buckingham, M.D., emeritus professor of pediatrics and endocrinology at Stanford. "The new extended wear infusion set decreased the number of times an infusion set needed to be changed by 50% and the number of infusion set failures associated with high glucose levels was low. Participants in the study commented on the new infusion set being more comfortable to wear compared to their previous infusion sets and were happy with the extended wear feature. Overall, they saw this as a meaningful improvement that reduced the overall burden of insulin pump therapy."
About Unomedical
Unomedical is a subsidiary of ConvaTec plc. ConvaTec is a FTSE-listed global medical products and technologies company focused on therapies for the management of chronic conditions, with leading market positions in advanced wound care, ostomy care, continence care and infusion care.
About the Diabetes Business at Medtronic (www.medtronicdiabetes.com)
Medtronic is working together with the global community to change the way people manage diabetes. The company aims to transform diabetes care by expanding access, integrating care and improving outcomes, so people living with diabetes can enjoy greater freedom and better health.
View original content to download multimedia:http://www.prnewswire.com/news-releases/medtronic-announces-european-launch-of-worlds-first-infusion-set-with-wear-up-to-seven-days-to-help-reduce-burden-for-people-with-diabetes-301268185.html
SOURCE Medtronic plc
Medtronic launches its insulin delivery infusion set in Europe
Apr. 14, 2021 9:20 AM ET
By: Aakash Babu, SA News Editor
- Medtronic (NYSE:MDT) announces the introduction of the Medtronic Extended infusion set, the first and only infusion set that can be worn for up to 7 days, in select European countries to help reduce burden for people with diabetes.
- https://seekingalpha.com/news/3681726-medtronic-launches-its-insulin-delivery-infusion-set-in-europe
- https://seekingalpha.com/symbol/MDT
- https://www.medtronicdiabetes.com/home
- www.medtronic.com
- https://www.medtronicdiabetes.com/products/infusion-sets
TECFIDERA® (DIMETHYL FUMARATE)
BIOGEN GROWS PRESENCE IN CHINA WITH THE APPROVAL OF TECFIDERA® (DIMETHYL FUMARATE) FOR THE TREATMENT OF RELAPSING MULTIPLE SCLEROSIS
April 15, 2021 at 4:30 PM EDT
- TECFIDERA was approved under the National Medical Products Administration priority review process evaluating therapies with urgent clinical needs
- More than 500,000 individuals worldwide living with multiple sclerosis (MS) have been treated with TECFIDERA, which has a well-established safety and efficacy profile
- Biogen’s expansion in China now includes treatment options approved for relapsing MS and spinal muscular atrophy
CAMBRIDGE, Mass., April 15, 2021 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) today announced that China’s National Medical Products Administration (NMPA) has approved TECFIDERA® (dimethyl fumarate) for the treatment of relapsing multiple sclerosis (MS). First introduced in 2013, TECFIDERA has demonstrated a well-established safety and efficacy profile with more than 10 years of data from clinical trials and real-world experience.1 It has been used to treat more than 500,000 individuals with MS worldwide.1 The approval brings a new treatment option to people in China living with relapsing MS and also continues to expand the company’s presence in the country.
“It is truly a milestone to bring this well-established treatment to China and be able to help people living with relapsing multiple sclerosis,” said Rachid Izzar, President, Intercontinental Region at Biogen. “We thank the NMPA for undertaking priority review to approve TECFIDERA. We are committed to expanding our presence in China and working with the MS community to address unmet medical needs through innovative therapies and solutions.”
TECFIDERA® (DIMETHYL FUMARATE)
About TECFIDERA® (dimethyl fumarate)
TECFIDERA, a treatment for relapsing forms of multiple sclerosis (MS) in adults, is the most prescribed oral medication for relapsing MS in the world and has been shown to reduce the rate of MS relapses, slow the progression of disability and impact the number of MS brain lesions, while demonstrating a well-characterized safety profile in people with relapsing forms of MS including clinically isolated syndrome, relapsing-remitting disease and active secondary disease. TECFIDERA is approved in 69 countries, and more than 500,000 patients have been treated with it, representing more than 950,000 patient-years of exposure across clinical trial use and patients prescribed TECFIDERA. Of these, 6,335 patients (14,241 patient-years) were from clinical trials.3
TECFIDERA is contraindicated in patients with a known hypersensitivity to dimethyl fumarate or any of the excipients of TECFIDERA. Serious side effects include anaphylaxis and angioedema, and cases of progressive multifocal leukoencephalopathy, a rare opportunistic viral infection of the brain which has been associated with death or severe disability, have been seen with TECFIDERA patients in the setting of prolonged lymphopenia although the role of lymphopenia in these cases is uncertain. Other serious side effects include a decrease in mean lymphocyte counts during the first year of treatment, herpes zoster and other serious infections, liver injury and flushing. In clinical trials, the most common adverse events associated with TECFIDERA were flushing, abdominal pain, diarrhea and nausea.
Please click here for Important Safety Information and full Prescribing Information, including Patient Information for TECFIDERA in the U.S., or visit your respective country’s product website.
We routinely post information that may be important to investors on our website at www.biogen.com.
Biogen's TECFIDERA wins approval in China to treat multiple sclerosis
Apr. 15, 2021 4:40 PM ET
By: Aakash Babu, SA News Editor2 Comments
- Biogen (NASDAQ:BIIB) announces that China’s National Medical Products Administration (NMPA) has approved TECFIDERA (dimethyl fumarate) for the treatment of relapsing multiple sclerosis (MS).
- TECFIDERA is approved in 69 countries, and more than 500K patients have been treated with it worldwide.
- https://seekingalpha.com/news/3682388-biogens-tecfidera-wins-approval-in-china-to-treat-multiple-sclerosis
- https://seekingalpha.com/symbol/BIIB
- https://www.biogen.com/
Evrysdi™ (risdiplam)
Wednesday, Apr 14, 2021
Genentech’s Evrysdi Continues to Improve Motor Function and Survival in Babies With Type 1 Spinal Muscular Atrophy (SMA)
More than twice as many babies (61% vs. 29%) were able to sit without support for at least five seconds after 24 months compared to 12 months of treatment
Evrysdi increased survival and reduced need for permanent ventilation
Evrysdi has proven efficacy across adults, children and babies 2 months and older
South San Francisco, CA -- April 14, 2021 --
Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY) today announced new 2-year data from Part 2 of FIREFISH, a Phase 2/3 global study evaluating Evrysdi™ (risdiplam) in infants aged 1-7 months at enrollment with symptomatic Type 1 spinal muscular atrophy (SMA). The data showed Evrysdi continued to improve motor function between months 12 and 24, including the ability to sit without support. The study also showed Evrysdi continued to improve survival, improve ability to feed orally and reduce the need for permanent ventilation*. Exploratory data suggested Evrysdi continued to improve the ability to swallow and reduce hospitalizations compared to the natural course of Type 1 SMA. Safety for Evrysdi was consistent with its established safety profile. These longer-term data build upon one-year pivotal findings from FIREFISH Part 2 and will be presented at the 73rd American Academy of Neurology (AAN) Annual Meeting being held virtually April 17-22, 2021.
Evrysdi™ (risdiplam)
About Evrysdi™ (risdiplam)
Evrysdi is a survival of motor neuron 2 (SMN2) splicing modifier designed to treat SMA by increasing and sustaining production of the survival of motor neuron (SMN) protein. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and movement. Evrysdi is administered daily at home in liquid form by mouth or by feeding tube.
The U.S. Food and Drug Administration (FDA) approved Evrysdi for the treatment of SMA in adults and children 2 months of age and older in August of 2020. In March 2021, the European Commission (EC) approved Evrysdi for the treatment of 5q SMA in patients 2 months of age and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies. Evrysdi has been approved in 39 countries and submitted in a further 33 countries.
Evrysdi is currently being evaluated in four multicenter trials in people with SMA:
- FIREFISH (NCT02913482) – an open-label, two-part pivotal clinical trial in infants with Type 1 SMA. Part 1 was a dose-escalation study in 21 infants with the primary objective of assessing the safety profile of Evrysdi in infants and determining the dose for Part 2. Part 2 is a pivotal, single-arm study of Evrysdi in 41 infants with Type 1 SMA treated for 2 years. Patients enrolled in Part 2 received the final dose of Evrysdi defined in Part 1. Enrollment for Part 2 was completed in November 2018. The primary objective of Part 2 was to assess efficacy as measured by the proportion of infants sitting without support after 12 months of treatment, as assessed in the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development – Third Edition (BSID-III) (defined as sitting without support for 5 seconds). The study met its primary endpoint. Patients continued to be evaluated through 24 months of treatment for additional safety and efficacy findings. After 24 months, patients will enter a 3-year open-label extension phase and continue to receive Evrysdi at the same dose.
- SUNFISH (NCT02908685) – SUNFISH is a two-part, double-blind, placebo controlled pivotal study in people aged 2-25 years with Types 2 or 3 SMA. Part 1 (n=51) determined the dose for the confirmatory Part 2. Part 2 (n=180) evaluated motor function using total score of Motor Function Measure 32 (MFM-32) at 12 months. MFM-32 is a validated scale used to evaluate fine and gross motor function in people with neurological disorders, including SMA. Patients in the placebo arm of Part 2 received placebo for 12 months followed by Evrysdi treatment for 12 months. The study met its primary endpoint.
- JEWELFISH (NCT03032172) – an open-label exploratory trial designed to assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) in people with SMA aged 6 months to 60 years who received other investigational or approved SMA therapies for at least 90 days prior to receiving Evrysdi. The study has completed recruitment (n=174).
- RAINBOWFISH (NCT03779334) – an open-label, single-arm, multicenter study, investigating the efficacy, safety, pharmacokinetics and pharmacodynamics of Evrysdi in babies (~n=25), from birth to 6 weeks of age (at first dose) with genetically diagnosed SMA who are not yet presenting with symptoms. The study is currently recruiting.
What is Evrysdi?
Evrysdi is a prescription medicine used to treat spinal muscular atrophy (SMA) in adults and children 2 months of age and older.
It is not known if Evrysdi is safe and effective in children under 2 months of age.
Please see the full Prescribing Information for additional Important Safety Information.
Roche’s Evrysdi continues to improve motor function, survival in babies with spinal muscular atrophy
Apr. 15, 2021 3:11 AM ET Roche Holding AG (RHHBY)
By: Mamta Mayani, SA News Editor
- Roche (OTCQX:RHHBY) unit, Genentech announces new 2-year data from Part 2 of FIREFISH, a Phase 2/3 study evaluating Evrysdi (risdiplam) in infants aged 1-7 months with symptomatic Type 1 spinal muscular atrophy (SMA).
- https://seekingalpha.com/news/3681967-roches-evrysdi-improves-motor-function-survival-in-babies-with-spinal-muscular-atrophy
- https://seekingalpha.com/symbol/RHHBY
- https://www.evrysdi.com/
casirivimab and imdevimab REGN-COV 2069
Phase III prevention trial showed subcutaneous administration of investigational antibody cocktail casirivimab and imdevimab reduced risk of symptomatic COVID-19 infections by 81%
- Among individuals who still experienced symptomatic infections, those who received casirivimab and imdevimab were able to clear the virus faster and had much shorter symptom duration
- In a cohort of recently-infected asymptomatic patients, casirivimab and imdevimab reduced the overall risk of progressing to symptomatic COVID-19 by 31%
- Detailed results will be shared with regulatory authorities including the EMA and the FDA
Basel, 12 April 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today confirmed positive results from the phase III REGN-COV 2069 trial assessing the ability of the investigational antibody cocktail casirivimab and imdevimab to reduce the risk and burden of COVID-19 infection among household contacts of SARS-CoV-2 infected individuals. The trial, which was jointly run with the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), met its primary and key secondary endpoints. It showed that the subcutaneous administration of casirivimab and imdevimab reduced the risk of symptomatic infections by 81% in those who were not infected when they entered the trial. In addition, individuals treated with casirivimab and imdevimab who still experienced a symptomatic infection resolved their symptoms on average within one week, compared to three weeks with placebo. No new or serious safety signals were observed.
“Today’s data confirm the potential dual value of casirivimab and imdevimab to reduce household COVID-19 infections and to decrease the disease burden in those who do become infected, when given as a subcutaneous option,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. “Although vaccinations are increasing globally, there remains a critical unmet need worldwide to prevent infections and provide immediate protection from COVID-19 between close contacts. This is why we are excited to bring this data to health authorities with the goal of making the combination available to more people as soon as possible.”
The phase III, double-blind, placebo-controlled trial assessed the effect of casirivimab and imdevimab on individuals without SARS-CoV-2 antibodies or any COVID-19 symptoms, who lived in the same household as an individual who tested positive to SARS-CoV-2 within the prior four days. It included 1,505 people who were not infected with SARS-CoV-2 at baseline and received either one dose of casirivimab with imdevimab (1,200 mg) or placebo, administered as subcutaneous injections.
casirivimab and imdevimab REGN-COV 2069
About REGN-COV 2069
REGN-COV 2069 is a phase III, randomised, double-blind, placebo-controlled multi-part study assessing the efficacy and safety of casirivimab and imdevimab in preventing symptomatic infection in household contacts of individuals infected with COVID-19.
In January 2021, initial data from the trial showed that there was a reduction in overall infections seen with casirivimab and imdevimab within the first week, with approximately 50% lower overall rates of infection (symptomatic and asymptomatic) and 100% prevention of symptomatic infections. Casirivimab and imdevimab showed efficacy when administered via a low-dose (1,200 mg) subcutaneous route.
In the safety assessment of these patients, adverse events occurred more frequently in participants on placebo during the efficacy analysis period (12% in the casirivimab and imdevimab group and 18% in the placebo group) and during the follow-up period (11% in the casirivimab and imdevimab group and 20% in the placebo group).
About casirivimab and imdevimab
Casirivimab and imdevimab is a cocktail of two monoclonal antibodies (also known as REGN10933 and REGN10987, respectively) and was designed by Regeneron scientists to block infectivity of SARS-CoV-2, the virus that causes COVID-19. They evaluated thousands of fully-human antibodies produced by the company's proprietary VelocImmune® mice, which have been genetically modified to have a human immune system, as well as antibodies identified from humans who have recovered from COVID-19.
The two potent, virus-neutralising antibodies casirivimab and imdevimab are believed to bind non-competitively to the critical receptor binding domain of the virus's spike protein, which is hypothesised to diminish the ability of mutant viruses to escape treatment and to protect against spike variants that may arise in the human population, as detailed in Science publications.
The cocktail of casirivimab and imdevimab has not been granted a marketing authorisation by any health authority. In November 2020, the antibody cocktail was authorised by the United States (U.S.) Food and Drug Administration (FDA) under an Emergency Use Authorization (EUA) for the treatment of mild to moderate COVID-19 in adults and paediatric patients (12 years of age and older weighing at least 40 kg) with positive results of direct SARS-CoV-2 viral testing, and who are at high risk for progressing to severe COVID-19 and/or hospitalisation. The US EUA is temporary and does not take the place of the formal biologics license application (BLA) submission, review and approval process.
In February 2021, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) issued a scientific opinion under Article 5(3) of Regulation 726/2004 supporting the use of casirivimab and imdevimab as a treatment option for patients with confirmed COVID-19 who do not require oxygen supplementation and who are at high risk of progressing to severe COVID-19. The scientific opinion can be considered by EU member states when making decisions on the use of medicines at a national level before a formal authorisation is issued. The review under Article 5(3) was separate, but ran in parallel to the rolling review of casirivimab and imdevimab, which is currently ongoing by the EMA.
Casirivimab and imdevimab’s development, manufacturing and clinical trials have been funded in part by the Biomedical Advanced Research and Development Authority (BARDA), part of the Office of the Assistant Secretary for Preparedness and Response at the US Department of Health and Human Services under OT number: HHSO100201700020C.
For more information, please visit www.roche.com.
Regeneron/Roche's antibody cocktail reduces risk of COVID-19 infections
Apr. 12, 2021 2:50 AM ETRoche Holding AG (RHHBY)By: Mamta Mayani, SA News Editor4 Comments
- Roche (OTCQX:RHHBY) and Regeneron Pharmacceuticals (NASDAQ:REGN) Phase III REGN-COV 2069 trial assessing antibody cocktail casirivimab and imdevimab in COVID-19 met primary and key secondary endpoints.
- https://seekingalpha.com/news/3680865-regeneronroches-antibody-cocktail-reduces-risk-of-covid-19-infections
- https://seekingalpha.com/symbol/RHHBY
- https://seekingalpha.com/symbol/REGN
Tagrisso (osimertinib)
Tagrisso approved in China in early lung cancer
PUBLISHED14 April 2021
14 April 2021 07:00 BST
Tagrisso is the only targeted medicine to show efficacy in the treatment of early-stage lung cancer in a global trial and the first such medicine approved in China
Approval based on unprecedented results from the ADAURA Phase III trial where Tagrisso reduced the risk of disease recurrence or death by 80%
AstraZeneca’s Tagrisso (osimertinib) has been approved in China for the adjuvant treatment of patients with early-stage (IB, II and IIIA) epidermal growth factor receptor-mutated (EGFRm) non-small cell lung cancer (NSCLC) after tumour resection with curative intent, with or without adjuvant chemotherapy as recommended by the patient’s physician. Tagrisso is indicated for EGFRm patients whose tumours have exon 19 deletions or exon 21 (L858R) mutations.
The approval by China’s National Medical Products Administration (NMPA) was based on positive results from the ADAURA Phase III trial. In the trial, Tagrisso demonstrated a statistically significant and clinically meaningful improvement in disease-free survival (DFS) in the primary analysis population of patients with Stage II and IIIA EGFRm NSCLC. The ADAURA trial also showed a statistically significant and clinically meaningful improvement in DFS in the overall trial population of patients with Stage IB-IIIA disease, a key secondary endpoint.
Consistent DFS results were seen regardless of prior adjuvant chemotherapy use and across all prespecified subgroups, including in Asian and non-Asian patients. The safety and tolerability of Tagrisso in this trial was consistent with previous trials in the metastatic setting. The ADAURA results were published in The New England Journal of Medicine.
ADAURA
ADAURA is a randomised, double-blind, global, placebo-controlled Phase III trial in the adjuvant treatment of 682 patients with Stage IB, II and IIIA EGFRm NSCLC following complete tumour resection and adjuvant chemotherapy as indicated. Patients were treated with Tagrisso 80mg once-daily oral tablets or placebo for three years or until disease recurrence.
The trial enrolled patients in more than 200 centres across more than 20 countries, including the US, in Europe, South America, Asia and the Middle East. The primary endpoint was DFS in Stage II and IIIA patients and a key secondary endpoint was DFS in Stage IB, II and IIIA patients.
The data readout was originally anticipated in 2022. In April 2020, an Independent Data Monitoring Committee recommended for the trial to be unblinded two years early based on a determination of overwhelming efficacy. Investigators and patients continue to participate and remain blinded to treatment. The trial will continue to assess overall survival.
Tagrisso
Tagrisso (osimertinib) is a third-generation, irreversible EGFR-TKI with clinical activity against central nervous system metastases. Tagrisso (40mg and 80mg once-daily oral tablets) has been used to treat approximately 250,000 patients across indications worldwide and AstraZeneca continues to explore Tagrisso as a treatment for patients across multiple stages of EGFRm NSCLC.
In Phase III trials, Tagrisso is being tested in the Stage III locally advanced unresectable setting (LAURA), in the neoadjuvant resectable setting (NeoADAURA) and in combination with chemotherapy (FLAURA2). AstraZeneca is also researching ways to address tumour mechanisms of resistance through the SAVANNAH and ORCHARD Phase II trials, which test Tagrisso given concomitantly with savolitinib, an oral, potent and highly selective MET TKI, as well as other potential new medicines.
What is TAGRISSO?
TAGRISSO is a prescription medicine used to treat adults with non-small cell lung cancer (NSCLC) that has certain abnormal epidermal growth factor receptor (EGFR) gene(s):
- to help prevent your lung cancer from coming back after your tumor(s) has been removed by surgery, or
- as your first treatment when your lung cancer has spread to other parts of the body (metastatic), or
- when your lung cancer has spread to other parts of the body (metastatic) and you have had previous treatment with an EGFR tyrosine kinase inhibitor (TKI) medicine that did not work or is no longer working
Your healthcare provider will perform a test to make sure that TAGRISSO is right for you.
It is not known if TAGRISSO is safe and effective in children.
Please see complete Prescribing Information, including Patient Information.
You may report side effects related to AstraZeneca products by clicking here.
Please visit astrazeneca.com
AstraZeneca wins regulatory approval for Tagrisso in China
Apr. 14, 2021 7:02 AM ETAstraZeneca PLC (AZN)By: Dulan Lokuwithana, SA News Editor
- AstraZeneca (NASDAQ:AZN) announced that Tagrisso (osimertinib) was authorized in China as an adjuvant treatment for a certain group of patients with non-small cell lung cancer (NSCLC) after tumor resection.
- https://seekingalpha.com/news/3681616-astrazeneca-wins-regulatory-approval-for-tagrisso-in-china
- hhttps://www.tagrisso.com/ttps://seekingalpha.com/symbol/AZN
- https://www.astrazeneca.com/
ORLADEYO™ (berotralstat)
BioCryst Announces Approval of Japanese NHI Price Listing of ORLADEYO™ (berotralstat) for Prophylactic Treatment of Hereditary Angioedema
—ORLADEYO is the first and only approved prophylactic therapy for HAE in Japan—
—NHI price listing triggers $15 million milestone payment to BioCryst—
RESEARCH TRIANGLE PARK, N.C., April 14, 2021 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the Japanese National Health Insurance System (NHI) has approved the addition of oral, once-daily ORLADEYO™ (berotralstat) to the NHI drug price list on April 21, 2021.
Oral, once-daily ORLADEYO was approved in Japan in January 2021 for prophylactic treatment of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older. ORLADEYO is the first and only prophylactic HAE medication approved in Japan.
ORLADEYO will be commercialized in Japan by BioCryst’s partner, Torii Pharmaceutical Co., Ltd. Torii plans to launch ORLADEYO following the NHI drug price listing.
“Our goal is to bring ORLADEYO to HAE patients around the world who want a new oral, once-daily option to prevent their attacks. In Japan, ORLADEYO is the first approved prophylactic HAE medication, which has the potential to significantly impact the lives of HAE patients,” said Jon Stonehouse, president and chief executive officer of BioCryst.
ORLADEYO™ (berotralstat)
About ORLADEYO™ (berotralstat)
ORLADEYO™ (berotralstat) is the first and only oral therapy designed specifically to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older. One capsule of ORLADEYO per day works to prevent HAE attacks by decreasing the activity of plasma kallikrein.
U.S. Indication and Important Safety Information
INDICATION
ORLADEYO™ (berotralstat) is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older.
Please see full Prescribing Information.
For more information, please visit the company’s website at www.biocryst.com.
BioCryst gets approval of Japanese NHI price listing of ORLADEYO
Apr. 14, 2021 9:03 AM ETBioCryst Pharmaceuticals, Inc. (BCRX)By: Aakash Babu, SA News Editor2 Comments
- BioCryst Pharmaceuticals (NASDAQ:BCRX) announces that the Japanese National Health Insurance System (NHI) has approved the addition of oral, once-daily ORLADEYO (berotralstat) to the NHI drug price list on April 21, 2021.
- https://seekingalpha.com/news/3681716-biocryst-gets-approval-of-japanese-nhi-price-listing-of-orladeyo
- https://seekingalpha.com/symbol/BCRX
- https://ir.biocryst.com/
Belapectin (GR-MD-02)
Journal for ImmunoTherapy of Cancer Publishes Phase 1 Clinical Research Showing Belapectin, Galectin Therapeutics’ Galectin-3 Inhibitor, Enhances Tumor Response in Combination with Anti-PD-1 Therapy
Combination therapy with belapectin suggests a better objective response rate with fewer adverse events than pembrolizumab (KEYTRUDA®) alone
NORCROSS, Ga., April 13, 2021 (GLOBE NEWSWIRE) -- Galectin Therapeutics Inc. (NASDAQ:GALT), the leading developer of therapeutics that target galectin proteins, today announced that a paper published in the peer-reviewed Journal for ImmunoTherapy of Cancer (JITC), the highest ranked fully open access immunology journal, provides further clinical evidence that using belapectin, a potent galectin-3 inhibitor, in combination with pembrolizumab (KEYTRUDA®), a PD-1 inhibitor, significantly enhances tumor response to immunotherapy in patients with advanced metastatic melanoma (MM) and head and neck squamous cell carcinoma (HNSCC).
The paper, titled “Enhancing Clinical and Immunological Effects of anti-PD-1 with Belapectin, a Galectin-3 Inhibitor” (doi:10.1136/jitc-2021-002371) describes results from an ongoing Phase 1 clinical study, a collaboration between Galectin Therapeutics and Providence Cancer Institute in Portland, Oregon.
Following the recent publication of positive preclinical results that showed the inhibition of galectin-3 in combination with an agonist anti-OX40 monoclonal antibody reprograms the tumor microenvironment to favor anti-tumor activity, the current study tests the clinical hypothesis that galectin-3 blockade with belapectin in combination with pembrolizumab enhances tumor response for patients with advanced MM or HNSCC.
About Belapectin (GR-MD-02)
Belapectin (GR-MD-02) is a complex carbohydrate drug that targets galectin-3, a critical protein in the pathogenesis of NASH and fibrosis. Galectin-3 plays a major role in diseases that involve scarring of organs including fibrotic disorders of the liver, lung, kidney, heart and vascular system. Belapectin binds to galectin-3 and disrupts its function. Preclinical data in animals have shown that belapectin has robust treatment effects in reversing liver fibrosis and cirrhosis. A Phase 2 study showed belapectin may prevent the development of esophageal varices in NASH cirrhosis; these results provide the basis for the conduct of the NAVIGATE trial. The NAVIGATE trial (NAVIGATEnash.com), entitled “A Seamless Adaptive Phase 2b/3, Double-Blind, Randomized, Placebo-controlled Multicenter, International Study Evaluating the Efficacy and Safety of Belapectin (GR-MD-02) for the Prevention of Esophageal Varices in NASH Cirrhosis” began enrolling patients in June 2020 and is posted on www.clinicaltrials.gov (NCT04365868). Galectin-3 also has a significant role in cancer, and the Company is supporting a Phase 1 study in combined immunotherapy of belapectin and KEYTRUDA in treatment of advanced melanoma and in head and neck cancer.
Additional information is available at www.galectintherapeutics.com.
Galectin's belapectin + Keytruda enhances tumor response in melanoma, head/neck cancer
Apr. 14, 2021 12:29 AM ET Galectin Therapeutics Inc. (GALT) By: Mamta Mayani, SA News Editor
- Galectin Therapeutics (NASDAQ:GALT) announces that a paper published in the peer-reviewed Journal for ImmunoTherapy of Cancer (JITC), provides further clinical evidence that using belapectin in combination with pembrolizumab (Keytruda), significantly enhances tumor response to immunotherapy in patients with advanced metastatic melanoma (MM) and head and neck squamous cell carcinoma (HNSCC).
- https://seekingalpha.com/news/3681585-galectins-belapectin-keytruda-enhances-tumor-response-in-melanoma-headneck-cancer
- https://seekingalpha.com/symbol/GALT
- www.galectintherapeutics.com.
TYVYT® (Sintilimab Injection)
Innovent and Lilly Release Phase 3 Results of TYVYT® (Sintilimab Injection) as a Second-Line Treatment for Squamous Non-Small Cell Lung Cancer at AACR Annual Meeting 2021
Publish at：Apr 13 2021
SAN FRANCISCO, U.S. and SUZHOU, China, April 13, 2021 — Innovent Biologics, Inc. (“Innovent”, HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high-quality medicines for the treatment of cancer, metabolic, autoimmune and other major diseases, announced with Eli Lilly and Company (“Lilly”, NYSE: LLY) that the results of the Phase 3 ORIENT-3 study were released today in an oral presentation at the American Association for Cancer Research (AACR) Annual Meeting 2021.
ORIENT-3 is a randomized, open-label, Phase 3 clinical trial evaluating TYVYT® (sintilimab injection) versus docetaxel as a second-line treatment for advanced or metastatic squamous non-small cell lung cancer (sqNSCLC). A total of 290 patients whose cancer had progressed following first-line treatment with platinum-based chemotherapy were enrolled. Based on the primary analysis population (280 patients, excluding patients on the docetaxel arm who received immunotherapy prior to disease progression), TYVYT® (sintilimab injection) demonstrated a statistically significant improvement in overall survival (OS) compared to docetaxel, meeting the pre-specified primary endpoint. The median OS was 11.79 months for patients on the TYVYT® (sintilimab injection) arm and 8.25 months for those on the docetaxel arm (HR=0.74, 95% CI: 0.56-0.96, P=0.02489). The median progression-free survival (PFS) as assessed by investigators was 4.30 months versus 2.79 months (HR=0.52, 95% CI: 0.39-0.68, P<0.00001), and the confirmed objective response rate (ORR) was 25.5 percent versus 2.2 percent (P<0.00001), respectively. Safety was consistent with previous studies of TYVYT® (sintilimab injection), and no new safety signals were identified.
TYVYT® (Sintilimab Injection)
"TYVYT® (sintilimab injection) was the first anti-PD-1 inhibitor included in the New Catalogue of the National Reimbursement Drug List in 2019,” said Dr. Hui Zhou, Vice President of Medical Science and Strategy Oncology of Innovent. “In August 2020, the NMPA accepted a new indication application for TYVYT® (sintilimab injection) in combination with chemotherapy for first-line treatment of squamous NSCLC. In the ORIENT-3 study, sintilimab as second-line monotherapy demonstrated a significantly improved survival benefit for patients with advanced squamous non-small cell lung cancer, and we look forward to the potential approval of this indication, to help more patients with this type of lung cancer.”
About the ORIENT-3 Trial
ORIENT-3 is a randomized, open-label, multi-center, Phase 3 clinical trial to evaluate the efficacy and safety of TYVYT® (sintilimab injection) as second-line therapy for advanced or metastatic sqNSCLC (ClinicalTrials.gov, NCT 03150875). The primary endpoint is overall survival (OS). The secondary endpoints include progression-free survival (PFS) as assessed by investigators based on RECIST v1.1, objective response rate (ORR) and safety profile.
A total of 290 patients were enrolled in ORIENT-3 and randomized in a 1:1 ratio to receive either TYVYT® (sintilimab injection) 200mg or docetaxel every three weeks. The patients received treatment until radiographic disease progression, unacceptable toxicity or any other conditions that require treatment discontinuation.
About TYVYT® (Sintilimab Injection)
TYVYT® (sintilimab injection), an innovative drug with global quality standards jointly developed in China by Innovent and Lilly, has been granted marketing approval by the NMPA (National Medical Products Administration) for the treatment of relapsed or refractory classic Hodgkin's lymphoma after two lines or later of systemic chemotherapy, and is included in the 2019 Guidelines of Chinese Society of Clinical Oncology for Lymphoid Malignancies. In February 2021, TYVYT® (sintilimab injection) was approved by the China NMPA in combination with pemetrexed and platinum chemotherapy as first-line therapy for the treatment of nonsquamous non-small cell lung cancer. TYVYT® (sintilimab injection) was included in the National Reimbursement Drug List (NRDL) in November 2019 as the first PD-1 inhibitor and the only PD-1 included in the list in that year.
Currently TYVYT® (sintilimab injection) has three supplemental New Drug Applications (“sNDA") under review by the NMPA. In August 2020, the NMPA accepted the sNDA for TYVYT® (sintilimab injection) in combination with gemcitabine and platinum chemotherapy as first-line therapy in squamous NSCLC. In January 2021, the NMPA accepted the sNDA for TYVYT® (sintilimab injection) in combination with BYVASDA® (bevacizumab injection) as first-line therapy in Hepatocellular Carcinoma (HCC) and the sNDA for TYVYT® (sintilimab injection) as second-line therapy in squamous NSCLC. Besides, in May 2020, TYVYT® (sintilimab injection) monotherapy met the primary endpoint of overall survival in the Phase 2 ORIENT-2 study as second-line therapy in patients with advanced or metastatic esophageal squamous cell carcinoma.
TYVYT® (sintilimab injection), 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 with TYVYT® (sintilimab injection) to evaluate its safety and efficacy in a wide variety of cancer indications, including more than 10 registrational or pivotal clinical trials. Meanwhile, Innovent is conducting clinical research studies on TYVYT® (sintilimab injection) worldwide.
Lilly's Tyvyt 'significantly improves' overall survival as a second-line treatment for lung cancer
Apr. 13, 2021 2:53 AM ETEli Lilly and Company (LLY)By: Mamta Mayani, SA News Editor
- Eli Lilly (NYSE:LLY) and Innovent Biologics (OTCPK:IVBIY) announce that the results of Phase 3 ORIENT-3 study evaluating Tyvyt (sintilimab injection) vs. docetaxel as a second-line treatment for advanced or metastatic squamous non-small cell lung cancer (sqNSCLC) were released at AACR Meeting 2021.
- https://seekingalpha.com/news/3681219-lillys-tyvyt-significantly-improves-overall-survival-in-lung-cancer
- https://seekingalpha.com/symbol/IVBIY
- https://seekingalpha.com/symbol/LLY
For more information, please visit: www.innoventbio.com.
To learn more about Lilly, please visit us at lilly.com
and lilly.com/newsroom.
About Innovent Biologics’ strategic collaboration with Eli Lilly and Company
Innovent entered into a strategic collaboration with Lilly focused on biological medicine in March 2015 – a groundbreaking partnership between a Chinese pharmaceutical company and a multinational pharmaceutical company. Under the agreement, Innovent and Lilly are co-developing and commercializing oncology medicines, including TYVYT® (sintilimab injection), in China. In October 2015, the two companies announced the extension of their existing collaboration to include co-development of three additional oncology antibodies targeting oncology indications. In August 2019, Innovent further entered a licensing agreement with Lilly to develop and commercialize a potentially global best-in-class diabetes medicine in China. Its collaboration with Lilly indicates that Innovent has established a comprehensive level of cooperation between China’s innovative pharmaceuticals sector and the international pharmaceuticals sector in fields such as R&D, CMC, clinical development and commercialization. In August 2020，Lilly and Innovent announced a global expansion of their strategic alliance for TYVYT® (sintilimab injection), whereby Lilly obtained an exclusive
license for TYVYT® (sintilimab injection) for geographies outside of China and plans to pursue registration of TYVYT® (sintilimab injection) in the U.S. and other markets.
Note:
TYVYT® (sintilimab injection) is not an approved product in the United States.
Retevmo® (selpercatinib)
Lilly Presents New Data on Retevmo® (selpercatinib) in Advanced RET Fusion-Positive Gastrointestinal and Other Cancers at 2021 American Association for Cancer Research (AACR) Annual Meeting
April 11, 2021Download PDFConfirmed Objective Response Rate of 47 Percent, with Responses Observed in Nine Unique Cancer Types
Median Duration of Response Not Reached at 13 Months Median Follow-Up, with 11 of 15 Responses Ongoing
Safety Consistent with Known Profile of Retevmo
New Data Expands on Retevmo's Established Data in Lung and Thyroid Cancers
INDIANAPOLIS, April 11, 2021 /PRNewswire/ -- Eli Lilly and Company (NYSE: LLY) today announced for the first time data from the Phase 1/2 LIBRETTO-001 trial showing treatment with Retevmo® (selpercatinib) demonstrated encouraging antitumor activity and safety across RET fusion-positive advanced solid tumors beyond lung and thyroid cancers, including multiple treatment-refractory gastrointestinal (GI) malignancies. The data were presented at the 2021 American Association for Cancer Research (AACR) Annual Meeting, held virtually April 10-15, 2021.
"We are excited to broaden the body of evidence for Retevmo in RET fusion-positive cancers beyond lung and thyroid tumors," said David Hyman, M.D., chief medical officer, oncology at Lilly. "These encouraging outcomes, including in difficult-to-treat GI malignancies, support a growing body of evidence that RET fusions are potentially actionable in a wide range of tumor types. These findings further demonstrate the importance of broad tumor profiling in advanced cancers. We look forward to discussing these new data with regulatory authorities this year."
About LIBRETTO-001
The Phase 1/2 LIBRETTO-001 trial is the largest clinical trial of patients with RET-driven cancers treated with a RET inhibitor. The trial, which spans 16 countries and 89 sites, included a dose escalation phase (Phase 1) and a dose expansion phase (Phase 2). The Phase 2 portion of the trial had major efficacy outcomes of ORR and DoR, and prespecified secondary endpoints of central nervous system (CNS) ORR and CNS DoR, as determined by an independent review committee according to Response Evaluation Criteria in Solid Tumors (RECIST) v1.1.
LIBRETTO-001 continues to enroll patients with RET-altered tumors beyond lung cancer.
About Retevmo® (selpercatinib)
Retevmo (selpercatinib, formerly known as LOXO-292) (pronounced reh-TEHV-moh) is a selective and potent RET kinase inhibitor. Retevmo may affect both tumor cells and healthy cells, which can result in side effects. RET-driver alterations are predominantly mutually exclusive from other oncogenic drivers. Retevmo is an U.S. FDA-approved oral prescription medicine, 120 mg or 160 mg dependent on weight (<50 kg or ≥50 kg, respectively), taken twice daily until disease progression or unacceptable toxicity.i Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials.
Please see full Prescribing Information for Retevmo.
To learn more about Lilly's commitment to people with cancer, please visit www.LillyOncology.com.
To learn more about Lilly, please visit us at lilly.com
View original content to download multimedia:http://www.prnewswire.com/news-releases/lilly-presents-new-data-on-retevmo-selpercatinib-in-advanced-ret-fusion-positive-gastrointestinal-and-other-cancers-at-2021-american-association-for-cancer-research-aacr-annual-meeting-301266274.html
SOURCE Eli Lilly and Company
Lilly's retevmo shows antitumor activity in solid tumors
Apr. 12, 2021 5:06 AM ETEli Lilly and Company (LLY)By: Mamta Mayani, SA News Editor
- Eli Lilly (NYSE:LLY) announces data from the Phase 1/2 LIBRETTO-001 trial showing treatment with Retevmo (selpercatinib) demonstrated encouraging antitumor activity and safety across RET fusion-positive advanced solid tumors beyond lung and thyroid cancers, including multiple treatment-refractory gastrointestinal (GI) malignancies.
- https://seekingalpha.com/news/3680871-lillys-retevmo-shows-antitumor-activity-in-solid-tumors
- https://seekingalpha.com/symbol/LLY
- https://www.retevmo.com/
Trodelvy (sacituzumab govitecan-hziy)
April 13, 2021
U.S. FDA Grants Accelerated Approval to Trodelvy® for the Treatment of Metastatic Urothelial Cancer
– Accelerated Approval Granted for Locally Advanced or Metastatic Urothelial Cancer Following a Platinum-Containing Chemotherapy and a PD-1/PD-L1 Inhibitor –
– New Indication Marks Second FDA Approval for Trodelvy in 2021 –
FOSTER CITY, Calif.--(BUSINESS WIRE)-- Gilead Sciences, Inc. (Nasdaq: GILD) today announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval of Trodelvy® (sacituzumab govitecan-hziy) for use in adult patients with locally advanced or metastatic urothelial cancer (UC) who have previously received a platinum-containing chemotherapy and either a programmed death receptor-1 (PD-1) or a programmed death-ligand 1 (PD-L1) inhibitor. The accelerated approval was based on data from the international Phase 2, single-arm TROPHY study. Of the 112 patients who were evaluable for efficacy, 27.7% of those treated with Trodelvy responded to treatment, with 5.4% experiencing a complete response and 22.3% experiencing a partial response. The median duration of response was 7.2 months (95% CI: 4.7-8.6). The Trodelvy U.S. Prescribing Information has a BOXED WARNING for severe or life-threatening neutropenia and severe diarrhea; see below for Important Safety Information.
The FDA’s accelerated approval mechanism enables drugs that treat serious diseases with unmet medical need to be approved based on a surrogate or intermediate clinical endpoint. Continued approval is contingent upon verification and description of clinical benefit in a confirmatory trial.
A global, randomized Phase 3 confirmatory clinical trial TROPiCS-04 (NCT04527991) is underway and is also intended to support global registrations. More information on TROPiCS-04 is available at https://clinicaltrials.gov/ct2/show/NCT04527991.
About Trodelvy
Trodelvy (sacituzumab govitecan-hziy) is a first-in-class antibody and topoisomerase inhibitor conjugate directed to the Trop-2 receptor, a protein frequently expressed in multiple types of epithelial tumors, including metastatic triple-negative breast cancer (TNBC) and metastatic UC, where high expression is associated with poor survival and relapse.
In addition to the accelerated approval of Trodelvy for the treatment of locally advanced or metastatic UC, Trodelvy is approved in the U.S. to treat adult patients with unresectable locally advanced or metastatic TNBC who have received two or more prior systemic therapies, at least one of them for metastatic disease.
Beyond the regulatory approvals of Trodelvy in the U.S., regulatory reviews for Trodelvy in metastatic TNBC are currently underway in the EU,U.K., Canada, Switzerland and Australia, as well as in Singapore through our partner Everest Medicines. Trodelvy is also being developed as an investigational treatment for hormone receptor-positive/human epidermal growth factor receptor 2-negative (HR+/HER 2-) metastatic breast cancer and metastatic non-small cell lung cancer. Additional evaluation across multiple solid tumors is also underway.
About the TROPHY U-01 Study
The Phase 2 TROPHY-U01 (also known as IMMU-132-06) trial is an ongoing, international, multi-center, open-label, multi-cohort, single-arm study evaluating Trodelvy monotherapy or combination therapy in patients with metastatic UC after progression on a platinum-based regimen and anti-PD-1/PD-L1-based immunotherapy. In Cohorts 1 and 2, patients received Trodelvy 10 mg/kg administered intravenously on Days 1 and 8 of a 21-day cycle to be continued until disease progression or loss of clinical benefit. Trodelvy is approved under accelerated approval based on the objective response rate (ORR) and duration of response (DoR) established in Cohort 1.
Cohorts 2, 3, 4 and 5 of the study are ongoing. Cohort 2 is assessing the safety and efficacy of Trodelvy monotherapy in platinum-ineligible patients after progression on anti-PD-1/PD-L1-based immunotherapy. Cohort 3 is assessing the safety and efficacy of Trodelvy on Days 1 and 8 of a 21-day cycle followed by pembrolizumab at the standard approved dose (200 mg) only on Day 1 of a 21-day cycle in patients with metastatic UC who have progressed after prior platinum therapy. Cohorts 4 and 5 are assessing the safety and efficacy of Trodelvy combination therapy in patients with treatment naive metastatic UC, with those in Cohort 4 receiving cisplatin and those in Cohort 5 receiving cisplatin and avelumab, respectively, in addition to Trodelvy.
The primary endpoint is ORR based on RECIST 1.1 criteria evaluated by independent central review in all five cohorts. In Cohorts 1 and 2, secondary endpoints are DoR and progression-free survival (PFS) based on central review and overall survival (OS). Secondary endpoints in Cohorts 3, 4 and 5 include DoR, clinical benefit rate (CBR) and PFS based on central review by RECIST 1.1 criteria; DoR, CBR and PFS based on investigator review by RECIST 1.1 and iRECIST criteria, OS and safety and tolerability of Trodelvy in combination with pembrolizumab, cisplatin, or cisplatin and avelumab, depending on the Cohort. More information about TROPHY is available at https://clinicaltrials.gov/ct2/show/NCT03547973.
Please see full Prescribing Information, including BOXED WARNING.
U.S. Prescribing Information for Trodelvy, including BOXED WARNING, is available at www.gilead.com.
Trodelvy, Gilead and the Gilead logo are trademarks of Gilead Sciences, Inc., or its related companies.
For more information about Gilead, please visit the company’s website at www.gilead.com, follow Gilead on Twitter (@Gilead Sciences) or call Gilead Public Affairs at 1-800-GILEAD-5 or 1-650-574-3000.
i Dr. Tagawa is a paid consultant for Gilead Sciences, Inc.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210413006168/en/
Jacquie Ross, Investors
Gilead wins FDA accelerated approval for Trodelvy in urothelial cancer
Apr. 13, 2021 3:16 PM ET Gilead Sciences, Inc. (GILD) By: Jonathan M Block, SA News Editor7 Comments
- The FDA has granted accelerated approval to Gilead Sciences' (NASDAQ:GILD) Trodelvy (sacituzumab govitecan) for patients with advanced urothelial cancer.
- https://seekingalpha.com/news/3681484-gilead-wins-fda-accelerated-approval-for-trodelvy-in-urothelial-cancer
- https://seekingalpha.com/symbol/GILD
- https://www.gilead.com/
Aliqopa (copanlisib) in combination with Rituxan (rituximab)
Bayer releases positive phase 3 data for Aliqopa in non-Hodgkin's lymphoma
Apr. 10, 2021 10:25 PM ET Bayer Aktiengesellschaft (BAYZF) By: Jonathan M Block, SA News Editor4 Comments
- Data from a late-stage trial showed that Bayer's (OTCPK:BAYZF) Aliqopa (copanlisib) in combination with Rituxan (rituximab) led to an increase in progression-free survival ("PFS") in non-Hodgkin's lymphoma patients/.
- https://seekingalpha.com/news/3680849-bayer-releases-positive-phase-3-data-for-aliqopa-in-non-hodgkins-lymphoma
- https://seekingalpha.com/symbol/BAYRY
- https://www.aliqopa-us.com/
Aliqopa (copanlisib) in combination with Rituxan (rituximab)
April 10, 2021
Not intended for U.S. and UK Media – Plenary Session at AACR Annual Meeting 2021:
Combination of copanlisib and rituximab significantly increases progression-free survival in patients with relapsed indolent non-Hodgkin's Lymphoma
https://www.bayer.com/en/news-stories
Sitravatinib in Combination with Tislelizumab
BeiGene Presents Clinical Data on Sitravatinib in Combination with Tislelizumab at the AACR Annual Meeting 2021
April 11, 2021 at 4:00 PM EDT
- The combination showed preliminary antitumor activity in patients with unresectable or metastatic melanoma refractory or resistant to PD-1/L1 inhibitors and platinum-resistant ovarian cancer in an ongoing Phase 1b clinical trial, with a generally well-tolerated safety profile.
CAMBRIDGE, Mass. & BEIJING--(BUSINESS WIRE)--Apr. 11, 2021-- BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a commercial-stage biotechnology company focused on developing and commercializing innovative medicines worldwide, today announced that clinical data on its anti-PD-1 antibody tislelizumab, in combination with the investigational spectrum-selective kinase inhibitor sitravatinib being jointly developed with Mirati Therapeutics, Inc. (Mirati), were presented in two oral presentations at the American Association for Cancer Research (AACR) Annual Meeting 2021. Data presented at the meeting were from two cohorts of a Phase 1b trial (NCT03666143), in patients with unresectable or metastatic melanoma who were refractory or resistant to PD-1/L1 inhibitors and in patients with advanced platinum-resistant ovarian cancer (PROC).
BeiGene has an exclusive collaboration and license agreement with Mirati for the development, manufacturing and commercialization of sitravatinib in Asia (excluding Japan), Australia, and New Zealand.
About Sitravatinib
Sitravatinib is an investigational, spectrum-selective receptor tyrosine kinase (RTK) inhibitor that can potentially stimulate the body’s immune response to fight cancer. Sitravatinib targets the VEGFR and TAM (TYRO3, AXL, MERTK) receptor families, which are implicated in orchestrating an immunosuppressive tumor microenvironment (TME). Inhibiting these receptors has been shown to stimulate an anti-tumor immune response and potentially re-sensitize patients to checkpoint inhibitor (CPI) therapy in patients who previously developed resistance to CPI therapy. By targeting specific RTKs with sitravatinib, the immunosuppressive TME is converted to an immune-supportive TME, and combining with CPI therapy may help regain an immune response potentially overcoming resistance to CPI therapy. Sitravatinib is being evaluated in multiple clinical trials, including the Phase 3 SAPPHIRE study, in patients with advanced non-small cell lung cancer who are resistant to CPI therapy, and certain patients who are naïve to CPI therapy.
For more information visit www.mirati.com/science.
About Tislelizumab
Tislelizumab (BGB-A317) is a humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to FcγR on macrophages. In pre-clinical studies, binding to FcγR on macrophages has been shown to compromise the anti-tumor activity of PD-1 antibodies through activation of antibody-dependent macrophage-mediated killing of T effector cells. Tislelizumab is the first drug from BeiGene’s immuno-oncology biologics program and is being developed internationally as a monotherapy and in combination with other therapies for the treatment of a broad array of both solid tumor and hematologic cancers.
The China National Medical Products Administration (NMPA) has granted tislelizumab full approval for first-line treatment of patients with advanced squamous non-small cell lung cancer (NSCLC) in combination with chemotherapy. Tislelizumab has also received conditional approval from the NMPA for the treatment of patients with classical Hodgkin’s lymphoma (cHL) who received at least two prior therapies, and for the treatment of patients with locally advanced or metastatic urothelial carcinoma (UC) with PD-L1 high expression whose disease progressed during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy. Full approval for these indications is contingent upon results from ongoing randomized, controlled confirmatory clinical trials.
In addition, three supplemental Biologics License Applications for tislelizumab have been accepted by the Center for Drug Evaluation (CDE) of the NMPA and are under review for first-line treatment of patients with advanced non-squamous NSCLC in combination with chemotherapy, for the second- or third-line treatment of patients with locally advanced or metastatic NSCLC who progressed on prior platinum-based chemotherapy, and for previously treated unresectable hepatocellular carcinoma.
Currently, 16 potentially registration-enabling clinical trials are being conducted in China and globally, including 13 Phase 3 trials and three pivotal Phase 2 trials.
In January 2021, BeiGene and Novartis entered into a collaboration and license agreement granting Novartis rights to develop, manufacture, and commercialize tislelizumab in North America, Europe, and Japan.
Tislelizumab is not approved for use outside of China.
To learn more about BeiGene, please visit www.beigene.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20210411005024/en/
BeiGene's tislelizumab combo study shows antitumor activity in solid tumors
Apr. 12, 2021 12:58 AM ET BeiGene, Ltd. (BGNE) By: Mamta Mayani, SA News Editor
- BeiGene (NASDAQ:BGNE) presents clinical data on tislelizumab, in combination with sitravatinib being jointly developed with Mirati Therapeutics (NASDAQ:MRTX) at the American Association for Cancer Research (AACR) Annual Meeting 2021.
- https://seekingalpha.com/news/3680862-beigenes-tislelizumab-combo-study-shows-antitumor-activity-in-solid-tumors
- https://seekingalpha.com/symbol/BGNE