Sarepta Therapeutics, Inc. (NASDAQ:SRPT) is a biopharmaceutical company. The Company is focused on the discovery and development of unique ribonucleic acid for the treatment of rare, infectious and other diseases. The company has an Advisory Committee with the FDA scheduled April 25, after which the FDA with make a decision regarding the drug’s approval under the Prescription Drug User Fee Act (PDUFA) on May 26.
The committee will discuss the new drug application (NDA) for Sarepta’s product candidate, the eteplirsen injection for intravenous infusion, intended to treat Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping.
DMD is rare inherited disorder of progressive muscular weakness, typically in boys. There fewer than 200,000 US cases per year. Many different types of mutations can lead to DMD, some of which block dystrophin production, which is a protein that plays a role in stabilizing the membrane of muscle fibers. Symptoms of the disorder include frequent falls, trouble getting up or running, waddling gait, big calves, and learning disabilities.
Eteplirsen is designed to skip an exon (a part of a gene that will become a part of the final mature RNA) for purposes of treatment. Exon skipping is a potential treatment approach meant restore production of dystrophin protein.
On Janurary 15th, shares of Sarepta crashed more than 55%, after the FDA posted negative briefing documents on eteplirsen. The briefing documents focused on evidence of efficacy in two key areas, dystrophin data and clinical data vs. controls/natural history. The FDA appeared to be unconvinced of the eteplirsen's efficacy, claiming their is a lack of a clear-cut increase in dystrophin levels. The advisory committee was supposed to take place shortly after the briefing documents were released, on Janurary 22nd, but the meeting was canceled due to a heavy snow storm in the Wahington DC area. Sarepta hopes to convince the committee to reccomend eteplirsen on April 25, the new date that was assigned.
Sarepta shares increased by more than 55% since March, and yet the stock is still trading about 47% below its 52 week high. The biotech has about $23.5 million in debt, and about $193 in cash. With an average quarterly cash burn of about $60 million, and considering the company recently announced plans to cut expenses by closing its Oregon facility and moving some of its employees to the sites in Massachusetts, the company should have enough cash to continue operations with no further delusion by end of year. Last time the company raised debt was in second quarter of 2015.