Companies like Vital Therapies and Amgen (AMGN - Free Report) provided pipeline and regulatory updates over the last few days. Meanwhile, BioMarin (BMRN - Free Report) struck a deal with Medivation for its experimental cancer treatment.
Recap of the Week’s Most Important Stories
1. Acorda’s (ACOR - Free Report) shares were up with the company announcing that the United States Patent and Trademark Office (USPTO) Patent Trials and Appeal Board (PTAB) will not institute the inter partes review (IPR) of a couple of patents covering the company’s flagship product, Ampyra (Read more: Acorda Gets Favorable Outcome in Ampyra IPR Issue).
2. Vital Therapies’ shares crashed 79.4% on news that its investigational cell-based therapy for the treatment of liver failure failed to meet the primary endpoint of overall survival in a late-stage study (Read more: Vital Therapies Hit By Disappointing Data on Liver Drug).
Another company that suffered a setback recently is Israel-based Macrocure . The company announced that its lead pipeline candidate CureXcell is not likely to meet the primary endpoint in a late-stage study being conducted for venous leg ulcers. Shares plunged 75.3% on the update.
3. BioMarin has been in the news quite a bit last week – firstly related to its experimental Duchenne muscular dystrophy treatment (DMD) and also for its $570 million deal with Medivation for its phase III PARP inhibitor, talazoparib. The Medivation deal will allow BioMarin to focus on its core area of expertise - rare and ultra-rare genetic diseases (Read more: Medivation to Gain Global Rights to BioMarin's Talazoparib).
Meanwhile, BioMarin got rare pediatric disease designation for its experimental DMD treatment, drisapersen, from the FDA (Read more: BioMarin's Drisapersen Gets Rare Pediatric Disease Status). Under the FDA's Rare Pediatric Disease Priority Review Voucher program, when a drug for a rare pediatric disease is approved, the company may get a voucher which can be redeemed to obtain priority review for a subsequent marketing application for a different product. Importantly, the Priority Review Voucher can be sold or transferred an unlimited number of times.
What is interesting is that another company, Sarepta, involved in the development of a DMD treatment also received rare pediatric disease designation from the FDA. Sarepta also got priority review status for eteplirsen with a response from the FDA expected by Feb 26, 2016. Priority review vouchers are much sought after - just last week, AbbVie said that it will be shelling out $350 million for a priority review voucher from United Therapeutics (Read more: United Therapeutics to Sell Priority Review Voucher to AbbVie).
4. Amgen has submitted a regulatory application in the U.S. for etelcalcetide, previously known as AMG 416. The company is looking to get etelcalcetide approved for the treatment of secondary hyperparathyroidism in chronic kidney disease patients on hemodialysis. Approval would make etelcalcetide the first calcimimetic agent that can be administered intravenously at the end of a dialysis session.
5. Alkermes (ALKS - Free Report) is facing a delay in the potential approval of its experimental schizophrenia treatment, Aristada. The FDA told the company that it would not be able to provide a response by the action date of Aug 22. While the agency did not provide any details about the new action date, it indicated that the delay would be brief (Read more: Alkermes' Aristada Review Date Postponed by FDA).
Over the last five trading days, Regeneron recorded a decline of 16.37%. Biogen (BIIB - Free Report) lost 31.1% over the last six months with Regeneron being the highest gainer (17.96%) during this period.
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The NASDAQ Biotechnology Index was down 11.6% over the last five trading days (See the last biotech stock roundup here: Kite Addresses Patient Death Concerns, Omeros Soars on Data).
What's Next in the Biotech World?
Amgen has an important regulatory event coming up with the FDA expected to decide on the approval status of Repatha, the company’s PCSK9 inhibitor, this week.
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