Shares Of Sarepta Declined On New Phase 2 Data, Is The Drop Justified?

Shares of Sarepta Therapeutics (SRPT) tumbled around 14% on Monday Jan. 12, 2015 when the company announced mid-stage data from a phase IIb study, treating patients with Duchenne Muscular Dystrophy. The company reported more results in the trial known as the 6-minute walk test -- 6MWT. The 6MWT is a test used to determine the distance a patient is able to walk in 6 minutes on a hard flat surface. 

The reason for the share price drop was because the 6MWT, in 168 weeks showed a decline in the amount of the distance that patients were able to walk as compared to the previous 6MWT in 144 weeks. One thing to note before looking further into the data is that Duchenne Muscular Dystrophy is a very difficult disease to treat and has no treatment options for these patients. The only treatment that can be conducted in order to reduce the symptoms of muscle loss is giving the patient steroids, but as many know prolonged use of this drug is not safe. 

One thing the company displayed in the results is that over a three-year period, patients lost less than 80 meters of walking distance. Now as you can see losing 80 meters is a lot, but still the fact that these DMD patients are even able to walk any type of distance is a gift in itself. This is because by the age of 12 DMD patients lose the ability to walk and are forced into a wheelchair to get around. The fact that Sarepta's Exon skipping drug Eteplirsen is able to allow the ability for these patients to walk is great in itself. 

How does that translate to Eteplirsen being approved by the FDA? Well the fact that patients saw a reduction in their walking distance is unfortunate, but in the end Sarepta still met its primary endpoint set out by the FDA. That primary endpoint is the production of the muscle protein Dystrophin. With the primary endpoint met, the company will still be able to file an NDA for Eteplirsen, and expects the submission to occur in the middle of 2015. While the share price of Sarepta has been beaten down we believe that the data shown does not translate to a negative effect for the future of the company. As mentioned in this article Sarepta is still on track to submit the NDA for Eteplirsen for FDA approval which still presents investors with a nice catalyst to look forward too. 

 

Disclosure: No position in any stocks mentioned.

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