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Biotech Beat: Sarepta Therapeutics Inc (SRPT), Esperion Therapeutics Inc (ESPR), Valeant Pharmaceuticals Intl Inc (VRX)

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By Terry Chrisomalis

Sarepta Therapeutics Inc

On October 1, 2015 shares of Sarepta Therapeutics Inc (NASDAQ:SRPT) ended the day up approximately 22 % after the company announced additional positive phase 2b data for its Eteplirsen drug being developed to treat patients with Duchenne Muscular Dystrophy — DMD. So what exactly caused the huge spike in share price for Sarepta? Well according to the company final analysis concluded that patients in the 6 minute Walk Test — 6MWT — were able to obtain 151 meters walking distance over an external control in another study.

The fact that Etepliren helped these patients improve in walking distance was good enough but the drug showed additional positive effects. For instance the FDA wanted to see if Eteplirsen could help these patients create dystrophin proteins. That is because dystrophin proteins are needed for normal muscle movement, but these patients’ bodies lack the ability to produce these dystrophin proteins. Sarepta’s drug was able to help restore the ability for these patients to create these dystrophin proteins for muscle movement.

DMD is a very debilitating disease in which children are wheelchair bound at an early age and then have a very short life ahead because of lack of proper muscle movement. There are currently no FDA approved drugs used to treat DMD therefore Sarepta is attempting the shortest path possible to get this drug approved for these patients. One way in doing so is by working with the FDA to submit a New Drug Application — NDA — on a rolling basis. What that means is that Sarepta...