Sarepta Therapeutics Inc.’s ( '>SRPT ) shares are slowly climbing following the announcement of encouraging additional data from a phase IIb open label extension study (Study 202) on eteplirsen for the treatment of patients suffering from Duchenne muscular dystrophy (DMD).
The study had met its primary endpoint of increased novel dystrophin at week 48 – these results were presented earlier. 96 week data showed continued stability of walking ability in eteplirsen-treated patients.
Post 96 weeks of treatment, patients taking 30 mg/kg and 50 mg/kg doses of eteplirsen experienced less than a 5% decline from baseline in walking ability as measured by the 6-minute walk test (6MWT). A statistically significant treatment benefit of 70.8 meters was observed as compared to placebo.
According to information provided by Sarepta, roughly one in every 3,500 boys globally is affected by DMD.
Sarepta intends to file a New Drug Application (:NDA) for eteplirsen in the first half of 2014 with the U.S. Food and Drug Administration (:FDA).The decision to file the NDA was based on productive interactions with the FDA including a meeting with the agency in July.
We note that several other companies including Prosensa (RNA) are developing therapies targeting the DMD market. However, a few days back, a phase III study being conducted by Prosensa failed to meet the primary endpoint as Prosensa’s drisapersen did not show statistically significant improvement in the 6MWD test versus placebo. Sarepta shares climbed 18% on the drisapersen setback news.