Sarepta Therapeutics (SRPT) is a Cambridge biotech company, focusing on orphan drug development. It was just announcing a positive phase 3 study results last week about the indication of Duchenne muscular dystrophy (DMD), a fatal, muscle-wasting disease. However, its shares plunged 13% on the day. The Street was apparently not happy with the results but I think it was wrong and this could be a good opportunity to buy SRPT.
DMD is a rather rare disease, occurring in about 0.03% of boys and destroying muscle function and quality of life over time. Duchenne patients will gradually lose their waling ability and eventually the respiratory muscles are destroyed. They usually could not live past their 20s. Sarepta said that the results are good enough to seek approval from the FDA later this year and target to get the drug, called eteplirsen, to be approved in 2015. If successful, it would be Sarepta's first marketed drug. I bet the company will try its best to get ready for the submission and the stock will become hot again towards the end of the year or early next year.
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