Sarepta Reports Significant Clinical Benefit With Eteplirsen In Phase IIb Trial

(RTTNews.com) - Sarepta Therapeutics (SRPT) announced that treatment with its exon-skipping compound, eteplirsen, achieved a significant clinical benefit on the primary clinical outcome, the 6-minute walk test or 6MWT, over a placebo/delayed treatment cohort in a Phase IIb study in Duchenne muscular dystrophy or DMD patients. Eteplirsen administered once weekly at 50mg/kg over 36 weeks led to a 69.4 meter benefit compared to patients who received placebo for 24 weeks followed by 12 weeks of treatment with eteplirsen in the open-label extension.

In the predefined prospective analysis of the study's intent-to-treat population on the primary clinical outcome measure, the change in 6MWT distance from baseline, eteplirsen-treated patients who received 50mg/kg of the drug weekly showed a decline of 8.7 meters in distance walked from baseline, while patients who received placebo/delayed-eteplirsen treatment for 36 weeks showed a decline of 78.0 meters from baseline, for a statistically significant treatment benefit of 69.4 meters over 36 weeks.

There was no statistically significant difference between the cohort of patients who received 30mg/kg weekly of eteplirsen and the placebo/delayed treatment cohort.

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Sarepta Reports Significant Clinical Benefit With Eteplirsen In Phase IIb Trial