The biotech sector was in focus over the past week with pipeline and regulatory updates from quite a few biotech companies. While the development of antibodies and vaccines for coronavirus is still in news from this sector as the pandemic registers a spike again, other pipeline updates also grabbed the spotlight.
Recap of the Week’s Most Important Stories:
Bristol Myers’ CAR T-Cell Therapy BLA Review Delayed: Bristol Myers Squibb BMY announced that the FDA has informed about a delay in its review of the Biologics License Application (BLA) for lisocabtagene maraleucel (liso-cel). The BLA is seeking an approval for the therapy to treat adults with relapsed or refractory (R/R) large B-cell lymphoma after at least two prior therapies. The FDA notified the company that the review was not completed by the Prescription Drug User Fee Act (PDUFA) action date of Nov 16, 2020. Liso-cel is an investigational chimeric antigen receptor (CAR) T-cell therapy. The candidate was added to the company’s portfolio with the acquisition of Celgene Corporation.
The regulatory agency was unable to conduct an inspection of a third-party manufacturing facility in Texas during the current review cycle due to travel restrictions imposed amid the COVID-19 pandemic. Hence, the FDA is deferring actions on the application until the inspection is done. The application remains under review and the authoritative body did not provide a new action date. Meanwhile, the FDA nod to liso-cel by Dec 31 is one of the required remaining milestones for the Contingent Value Rights issued upon the close of the Celgene acquisition in the fourth quarter of 2019. The other one remaining is the FDA approval of Idecabtagene Vicleucel (ide-cel) by Mar 31, 2021.
Bristol-Myers currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Alkermes Gets Complete Response Letter for Schizophrenia Drug: Alkermes plc ALKS suffered a setback after it received a Complete Response Letter (CRL) from the FDA regarding its New Drug Application (NDA) for ALKS 3831 (olanzapine/samidorphan) to address adults with schizophrenia and those with bipolar I disorder. Following a remote review of the manufacturing records, the agency stated that resolution of certain conditions related to the tablet coating process at the company’s Wilmington, OH facility is required before its approval. The CRL did not identify or raise any concerns about the clinical or non-clinical data in the NDA. Further, the agency did not direct Alkermes to complete any additional clinical studies to support the approval of the application.
Moderna’s Updates on mRNA Vaccine : Moderna MRNA announced that the European Medicines Agency’s (EMA) human medicines committee (CHMP) started a rolling review of mRNA-1273, its vaccine candidate against COVID-19, following the confirmation of eligibility of mRNA-1273 for submission on Oct 14, 2020.
Moderna management announced that the phase III COVE study, which is evaluating mRNA-1273, met the primary efficacy endpoint at the first interim analysis, demonstrating a vaccine efficacy of 94.5%. The interim efficacy analysis was conducted by the Data Safety Monitoring Board (DSMB), appointed by the National Institutes of Health (NIH). The COVE study enrolled 30,000 participants with diverse backgrounds in the United States. The patient population of the study includes high-risk groups as well as individuals from communities of color.
Based on the interim safety and efficacy data, the company plans to submit an emergency use authorization (EUA) to the FDA in the coming weeks.
Adamis Plunges on CRL for Zimhi: Adamis Pharmaceuticals Corporation ADMP plummeted after it announced the receipt of a Complete Response Letter (CRL) from the FDA regarding its New Drug Application (NDA) for Zimhi high dose naloxone injection product for the treatment of opioid overdose. Per the CRL, the agency cannot approve the NDA in its present form and provided recommendations needed for resubmission. The questions raised by the FDA related generally to new Chemistry, Manufacturing and Controls (CMC) issues. The company will request a type A meeting or consider other options to resolve the issues. As a matter of fact, it had received a CRL earlier too.
Gilead Announces Results From NASH Combination Trial: Gilead Sciences, Inc. GILD and partner Novo Nordisk A/S announced positive results from a phase II proof-of-concept study at The Liver Meeting Digital Experience.
The five-arm study evaluated combinations of Novo Nordisk’s semaglutide, a GLP-1 receptor agonist, with Gilead’s investigational FXR agonist cilofexor and/or Gilead’s investigational ACC inhibitor firsocostat over 24 weeks in 108 patients with non-alcoholic steatohepatitis (NASH). Both companies had earlier collaborated in 2019 for evaluating combination treatments of NASH.
The study showed that all regimens were well tolerated in NASH patients with mild to moderate fibrosis. Consequently, the study met its primary endpoint. Both companies also presented preclinical data supporting the development of combination approaches to address NASH. Semaglutide alone and in combination with cilofexor and/or GS-834356 (an analog of firsocostat) were administered daily for 12 weeks to a murine model of diet-induced NASH (n=15-16/group). Results from this preclinical study demonstrated that while semaglutide significantly improved NASH and fibrosis-related endpoints, the addition of either cilofexor or the firsocostat analog further improved liver fat reduction.
The Nasdaq Biotechnology Index gained 1.95% in the last five trading sessions. Among the biotech giants, Biogen gained 5.28% during this period. Over the past six months, shares of Alexion have rallied 24.31%. (See the last biotech stock roundup here: Biotech Stock Roundup: Gilead, Amgen, Vertex and Alexion Report Q3 Earnings & More)
What’s Next in Biotech?
Stay tuned for more pipeline and regulatory updates.
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