With elections over and third quarter earnings season drawing to a close, focus is back on pipeline and regulatory updates. This week, Acorda ACOR announced that it won’t be continuing with the development of Ampyra for post-stroke walking difficulties (PSWD). Meanwhile, Spectrum SPPI got a complete response letter (CRL) from the FDA for Qapzola.
Recap of the Week’s Most Important Stories
Acorda Pulls the Plug on Ampyra for PSWD: Acorda, which was looking to get its flagship product, Ampyra, approved for additional indications, suffered a setback with the drug failing to show sufficient efficacy in a study being conducted for improvement in PSWD. The company said that it will focus its efforts on developing other pipeline candidates including Parkinson’s disease therapies, CVT-301 and tozadenant, both in late-stage development, and earlier stage assets like CVT-427 (migraine), SYN120 (Parkinson’s disease dementia), and rHIgM22 (multiple sclerosis). (Read more: Acorda Stops Post-Stroke Development Program on Ampyra).
Spectrum Gets CRL for Qapzola: Spectrum got a CRL from the FDA for Qapzola. The news did not come as a big surprise considering the FDA’s Oncologic Drugs Advisory Committee (ODAC) had voted in September that Qapzola did not show substantial evidence of a treatment effect over placebo. Spectrum is looking to get Qapzola approved for immediate intravesical instillation post-transurethral resection of bladder tumors (post-TURBT) in patients with non-muscle invasive bladder cancer (NMIBC). The company said that it met with the FDA to discuss the next steps in the development path. Based on its discussions with the agency, Spectrum will be evaluating a new smaller study that will replace the ongoing phase III program for which enrollment has been stopped.
Celgene Acquires Marizomib Assets from Triphase: Celgene CELG, through an affiliate, has acquired all the assets to a brain-penetrant proteasome inhibitor being developed by private drug development company, Triphase Accelerator Corporation. Marizomib is currently in development for glioblastoma and relapsed and/or refractory multiple myeloma. While detailed information was not available, Celgene will be making an upfront payment apart from payments on the achievement of regulatory, approval and sales milestones. With this deal, Celgene will be fully responsible for the development of marizomib and will pay Triphase for the completion of ongoing studies. Marizomib has orphan drug status in the U.S. for glioblastoma, an area of significant unmet medical need. Celgene is a Zacks Rank #2 (Buy) stock. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Praluent Cardiovascular Outcomes Study to Continue as Planned: Regeneron REGN and partner Sanofi will be continuing with the ODYSSEY OUTCOMES study on Praluent as planned instead of stopping it early, based on the recommendation of an independent Data Monitoring Committee (DMC) following a second pre-specified interim analysis. Praluent is one of two PCSK9 inhibitors (the other being Repatha) currently available in the market. While both drugs have been touted as having blockbuster potential, sales have been disappointing so far. The cardiovascular outcomes study data, if positive, are expected to boost sales significantly (Read more: Regeneron/Sanofi's Praluent CV Outcomes Study to Continue).
Regeneron and Sanofi were also present at the annual meeting of the American College of Rheumatology (ACR) with results on sarilumab from the late-stage SARIL-RA-MONARCH study, which demonstrated sarilumab’s superiority versus AbbVie’s Humira in adults with active rheumatoid arthritis (RA). Top-line results from this study were previously announced in Mar 2016. We note that sarilumab is facing a delay in gaining FDA approval having received a CRL last month due to manufacturing deficiencies in a Sanofi plant (Read more: Sanofi/Regeneron Present Sarilumab Data at ACR Meeting).
Mixed Results on Gilead’s JAK Inhibitor: Gilead GILD had some disappointing news on the pipeline front with the company providing mixed data on its investigational JAK inhibitor, momelotinib. While the candidate met the primary endpoint (non-inferiority to Jakafi) in one of the late-stage studies, it failed to meet a key secondary endpoint. In the other phase III study, the primary endpoint was missed. Momelotinib is being investigated in patients with myelofibrosis (Read more: Gilead Reports Phase III Myelofibrosis Data on Momelotinib). However, Gilead’s disappointing data is good news for Incyte with the competitive threat for its flagship product, Jakafi, going down.
Medical – Biomedical and Genetics Industry Price Index
The NASDAQ Biotechnology Index declined 3.3% over the last five trading days. Among major biotech stocks, Celgene was up slightly (0.05%) while Vertex VRTX declined 7.4%. Over the last six months, Biogen was up 20.1% while Alexion ALXN lost 17.9% (See the last biotech stock roundup here: Biotech Stocks Soar on Election Results, Gilead HBV Drug Approved).
What's Next in the Biotech World?
Watch out for the usual pipeline and regulatory updates from biotech companies.
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