The announcement of preliminary data from a Phase III clinical trial evaluating casimersen and golodirsen in Duchenne muscular dystrophy (DMD) patients amenable to skipping exons 45 and 53, respectively saw shares of Sarepta Therapeutics (Nasdaq: SRPT) gain 3% pre-market on Thursday, and still closed the day up 1.8% at $118.68.
The ESSENCE study is a global, randomized double-blind, placebo-controlled Phase III study evaluating the efficacy and safety of casimersen and golodirsen in patients amenable to skipping exons 45 or 53, respectively.
After soliciting feedback from the US Food and Drug Administration, Sarepta conducted an interim analysis for levels of dystrophin protein expression in those patients who are amenable to exon 45 skipping to determine the potential for a New Drug Application (NDA) filing based on dystrophin as a surrogate endpoint. With these results, Sarepta intends to work toward submission of an NDA for casimersen in the middle of 2019.
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