The Celgene Corporation CELG – Juno Therapeutics’ JUNO immunotherapy deal worth at least a billion dollars was the main story in the biotech sector this week. While concerns are being raised about the high price tag, the deal just goes to show the growing interest in the field of immunotherapy. Meanwhile, quite a few companies provided updates on the regulatory front.
Recap of the Week’s Most Important Stories
1. Juno, which is focused on the development of immunotherapies, saw its shares surging 15.2% on a deal worth more than $1 billion with Celgene. The collaboration will see the companies leveraging T-cell therapeutic strategies to develop treatments for patients with cancer and autoimmune diseases. The initial focus will be on chimeric antigen receptor technology (CAR-T) and T-cell receptor (TCR) technologies (Read more: Juno-Celgene in Immunotherapy Deal, Initial Deal Value $1B).
2. Intercept ICPT reported some progress on the regulatory front with its lead pipeline candidate – obeticholic acid (OCA). The company said that it has finished submitting the NDA for OCA for primary biliary cirrhosis (PBC) in the U.S. where it is seeking accelerated approval. Moreover, the company said that its marketing application in the EU has been accepted for review.
Intercept has orphan drug status for OCA for PBC in the U.S. as well as Europe and fast track designation for PBC in the U.S. There is significant need for new PBC treatments. Although ursodeoxycholic acid (UDCA) is currently the only approved medication for this indication and is the standard of care, most patients continue to experience persistent elevations above the upper limit of normal in the serum marker alkaline phosphatase (ALP), corresponding with increased risk of liver failure, need for liver transplant and death.
OCA is being developed for other indications as well including nonalcoholic steatohepatitis (NASH) for which the FDA granted breakthrough designation (Read more: Intercept Pharmaceuticals Files for OCA in U.S. and Europe).
3. Just a few days after the EMA accepted BioMarin’s BMRN regulatory application for its experimental Duchenne muscular dystrophy (DMD) treatment, drisapersen, the company announced that the FDA has granted priority review status to the drug in the U.S. This means a response regarding the approval status of drisapersen in the U.S. should be out by Dec 27 (Read more: BioMarin’s NDA for DMD Drug Drisapersen Accepted by FDA).
Meanwhile, Sarepta SRPT, another company focused on bringing a DMD treatment to market, finished submitting its NDA for its experimental DMD treatment, eteplirsen. While the FDA informed BioMarin that drisapersen will come up before an advisory committee, we believe eteplirsen will also be reviewed by an FDA advisory committee before a final decision regarding approval status is taken (Read more: Sarepta Files Eteplirsen NDA, Secures $40M Debt Financing).
4. Alexion ALXN received good news with the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopting positive opinions for two of the company’s drugs which target genetic, progressive, ultra-rare metabolic diseases. While the proposed indication for Strensiq is long-term enzyme replacement therapy in patients with pediatric-onset hypophosphatasia (HPP) to treat the bone manifestations of the disease, that for Kanuma is long-term enzyme replacement therapy in patients of all ages with lysosomal acid lipase deficiency (LAL-d). A final response should be out in the third quarter of 2015 (Read more: Alexion’s Strensiq and Kanuma Get Positive CHMP Opinions).
5. Biogen BIIB and partner Swedish Orphan Biovitrum said that their regulatory application for hemophilia B treatment, Alprolix, has been accepted for review in the EU. Alprolix, which is already approved in the U.S., brought in sales of $76 million in 2014 (Read more: Biogen/Sobi’s Hemophilia B Drug Alprolix under EU Review).
Over the last five trading days, all the major biotechs recorded a decline in their share price with Vertex recording the highest decline (6.18%) among these stocks. Meanwhile, Regeneron was the highest gainer (24.93%) over the last six months.
The NASDAQ Biotechnology Index declined 3.7% over the last five trading days (See the last biotech stock roundup here: BioMarin Jumps on Dwarfism Data, Vical Plunges on Vaccine Data).