Sarepta: Second Patient Doing Well On Muscular Dystophy Drug Trial

BOTHELL, Wash. (TheStreet) --Another kid with muscular dystrophy is showing vast improvement after treatment with Sarepta Therapeutics' (SRPT) experimental drug eteplirsen, pushing the company shares higher in Thursday trading.

The latest anecdotal report from Sarepta's small phase II study of the Duchenne muscular dystrophy drug comes courtesy of KFVS, a local TV station in southeast Missouri. The station broadcast a news report Wednesday night on Justin Trovillion, 12, a DMD patient who appears to be responding well to treatment with the higher, 50 mg dose of eteplirsen.

Sarepta shares rose $1.73, or 14%, to $14.29 as a link to the KFVS story was passed along by investors.

The improvements observed in Trovillion's muscle strength and endurance seem to echo those of Max Leclaire, 10, of Vermont and another patient treated with the 50 mg dose of eteplirsen in Sarepta's phase II trial.

Sarepta only enrolled four boys into the 50 mg dose arm of the eteplirsen study, so it now appears as if at least 50% are demonstrating significant improvements. Sarepta announced positive, interim results from the study in July and is expected to present important follow-up data in October.

If the final eteplirsen results confirm the benefit seen at the interim analysis, Sarepta is expected to request accelerated approval for the drug from the U.S. Food and Drug Administration. Under new rules, FDA has been encouraged to accelerate the approval of life-saving drugs for diseases that have no cure. DMD certainly fits that category. The challenge for both Sarepta and FDA will be to review eteplirsen based on a small study that enrolled just 12 patients.

Sarepta needs additional cash to ramp up manufacturing of eteplirsen, which is very complicated to produce. With the stock ramping higher, speculation has centered around if and when the company will announce a new financing.

Dystrophin is a protein that plays key role in muscle function and repair. The genetic inability to make dystrophin is what causes muscular dystrophy. Eteplirsen is designed to "skip over" the section of damaged gene in DMD patients and therefore restore the gene's ability to produce partially functioning dystrophin.

GlaxoSmithKline (GSK) is also developing a similar drug for DMD.

--Written by Adam Feuerstein in Boston.



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