Sarepta Therapeutics, Inc (NASDAQ:SRPT) stock has been in the news recently for its eteplirsen drug which is designed for treatment of Duchenne muscular dystrophy (DMD) which is a muscle wasting disease caused by the lack of dystrophin production. The FDA advisory on April 26 voted that the drug is not effective for the treatment of DMD. The decision was taken after hearing pleas of dozens of parents of effected children who told that the drug made substantial difference to them in the clinical trials. The final decision would be taken on May 26th. So we should be expecting volatility in the next few days.
Dr. Janet Woodcock, director of FDA’s Center for Drug Evaluation and Research told that “Its possible to reach differenet conclusions based on the data presented today,” she said during brief remarks. “Failing to approve a drug that actually works in devastating diseases – these consequences are extreme.”
FDA was not happy with the way the studies were conducted for the trial as well the sample size of 12 was also considered small.
The company’s interim chief executive officer and chief medical officer Edward Kaye M.D. during the announcement of first quarter results said “The Peripheral and Central Nervous System Advisory Committee met last week to review eteplirsen for the treatment of Duchenne muscular dystrophy amenable to exon 51 skipping, and we await our May 26 PDUFA date.” “Following the FDA’s decision, we plan to provide a clinical and corporate update.”
FDA had rejected two other drugs for DMD recently. In January 2016 it rejected BioMarin Pharmaceutical’s drug drisapersen which is branded as Kyndrisa. The clinical trials showed that the drug did not successfully increase dystrophin levels. On Feb 23,2016 FDA rejected PTC Therapeutics (NASDAQ:PTCT) drug Translarna’s New Drug application (NDA) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). Translarna has got conditional approval in the European Union (EU) in 2014. The global revenues for 2015 from Translarna were $ 33.7 million.
Sarepta Therapeutics Pipeline:
Review of Financial Position:
The company released its First quarter 2016results on May 05, 2016. The company did not report any revenues for the first quarter of 2016 as well as 2015. Non-GAAP net loss was $ 52.5 million or $ (1.15) per share compared to $ 47.4 million or $ (1.15) per share. The loss increased this year due to rise in operating expenses. Non-GAAP Research and development expenses were $35.9 million for the first quarter of 2016 compared to $ 36.7 million for the first quarter of 2015. Non-GAAP general and administrative expenses were $ 16.6 million for this year compared to $ 11 million for first quarter of 2015. The company had $ 140.6 million cash and cash equivalents as of March 31, 2016 compared to $ 204 million as December 31, 2015. The decrease in cash was due to funding the company’s ongoing operations.
About Sarepta Therapeutics:
Sarepta Therapeutics is a biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare, infectious and other diseases. The Company is primarily focused on rapidly advancing the development of its potentially disease-modifying DMD drug candidates, including its lead DMD product candidate, eteplirsen, designed to skip exon 51. Sarepta is also developing therapeutics for the treatment of rare, infectious and other diseases.
(Please note that biotech stocks discussed might have huge volatility. Article is based on public information which I believe to be reliable but I cannot guarantee that the information is accurate. Please consult your financial advisors before investing.)