Biotech Stock Roundup: Celgene Submits MS Drug to FDA, INSY & CBAY Crash

The biotech sector was back in focus this week with quite a few regulatory updates. While Celgene’s CELG multiple sclerosis (MS) drug was submitted for approval, Vertex expanded its collaboration with CRISPR. Meanwhile, shares of both Insys INSY and CymaBay Therapeutics CBAY plunged on disappointing news.

Recap of the Week’s Top Stories:

Celgene's MS Drug Accepted for Review in US/EU: Celgene announced that the FDA has accepted for review the new drug application (NDA) for ozanimod for the treatment of people with relapsing forms of multiple sclerosis (RMS) in the United States. The European Medicines Agency (EMA) also accepted for review the Marketing Authorization Application for ozanimod for the treatment of adults with relapsing-remitting multiple sclerosis  in the European Union. 

The FDA has set an action date of Mar 25, 2020. A regulatory decision from the EMA is expected in the first half of 2020. The FDA and EMA applications are based primarily on ozanimod data from the SUNBEAM and RADIANCE Part B phase III studies. In February 2018, the company received a Refusal to File letter from the FDA regarding its NDA for ozanimod for the treatment of people with RMS. Upon its preliminary review, the FDA determined that the nonclinical and clinical pharmacology sections in the NDA were insufficient to permit a complete review. The company then submitted a NDA again in March 2019 to the FDA based on data from the SUNBEAM and RADIANCE studies.

Celgene currently carries a Zacks Rank #2 (Buy). You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here. 

Insys Crashes on Bankruptcy News: Insys Therapeutics plummeted 51.4% after it filed for bankruptcy protection under Chapter 11 of the U.S. Bankruptcy Code in the U.S. Bankruptcy Court in the District of Delaware. The protection will enable the company to facilitate the sale of substantially all its assets and address its legacy legal liabilities. Insys has been embroiled in various litigations in recent times amid allegations of fueling the ongoing opioid endemic in the United States. The company has also been accused of bribing doctors to prescribe its lead drug, Subsys, to patients. This month, Insys signed a settlement with the United States, formalizing its previously announced settlement relating to certain civil statutory claims. As part of this agreement, the company agreed to pay $195 million.

Biogen Acquires Nightstar: Biogen BIIB announced that it has acquired clinical-stage gene therapy company Nightstar Therapeutics for $800 million. The acquisition will add NST’s lead asset, NSR-REP1, to the company’s pipeline. The candidate is being evaluated for the treatment of choroideremia (CHM), a rare, degenerative, X-linked inherited retinal disorder, which leads to blindness and has no approved treatments. NSR-RPGR is NST’s second clinical program for the treatment of X-linked retinitis pigmentosa (XLRP), which is also a rare inherited retinal disease, primarily affecting males with no approved treatments. Biogen now has added two mid- to late-stage clinical assets, as well as preclinical programs, in ophthalmology.

Vertex Expands Collaboration With CRISPR: Vertex Pharmaceuticals VRTX announced that it is expanding its collaboration with CRISPR Therapeutics CRSP for an exclusive licensing agreement to discover and develop gene editing therapies targeting Duchenne muscular dystrophy (“DMD”) and Myotonic dystrophy type 1 (DM1).

Per the expanded collaboration terms, Vertex will pay CRISPR $1 billion, including $175 million in upfront payment and potential research, development, regulatory, and commercial milestone payments for the DMD and DM1 programs. Vertex will get exclusive worldwide rights to CRISPR Therapeutics’ existing and future intellectual property including foundational CRISPR/Cas9 technology.

Moreover, Vertex will be responsible for all research, development, manufacturing, and commercialization activities and related costs. However, research costs for a specified guide RNA research related to DM1 program will be shared by both the companies. Vertex and CRISPR had signed a four-year research collaboration back to October 2015. Per this agreement, Vertex and CRISPR are developing gene editing therapies for cystic fibrosis and sickle cell disease. In December last year, the companies selected gene therapy, CTX001, to move into clinical development for sickle cell disease and beta thalassemia.