Sarepta Therapeutics, Inc. (NASDAQ: SRPT), the leader in precision genetic medicine for rare diseases, announced topline results from EMBARK (Study SRP-9001-301), a global, randomized, double-blind, placebo-controlled, Phase 3 clinical study of ELEVIDYS (delandistrogene moxeparvovec-rokl) in patients with Duchenne muscular dystrophy between the ages of 4 through 7 years.
The results of EMBARK trial support the conclusion that ELEVIDYS modifies the trajectory of Duchenne and benefits patients across age groups living with Duchenne.
While the primary endpoint was not met, participants treated with ELEVIDYS showed improvements in motor function, as measured by the North Star Ambulatory Assessment, compared to the placebo group at 52 weeks. Notably, statistically significant results were achieved in key secondary endpoints, including time to rise and the 10-meter walk test.
The study found no new safety concerns, and Sarepta plans to request an expansion of the therapy’s labeled indication based on these results, with the FDA indicating openness to review the data for such an expansion.
About EMBARK, Study 9001-301
Study SRP-9001-301, also known as EMBARK, is a multinational, phase 3, randomized, two-part crossover, placebo-controlled study of ELEVIDYS in individuals with Duchenne muscular dystrophy between the ages of 4 to 7 years. The primary endpoint is change from baseline in NSAA Total Score at Week 52 following treatment. Eligible participants received a single dose of ELEVIDYS during either Part 1 or Part 2 of the study.
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