Press release
Sarepta Therapeutics Announces Results from Part 2 of the EMBARK Study Demonstrating Sustained Benefits and Disease Stabilization in Ambulatory Individuals with Duchenne Muscular Dystrophy Following Treatment with ELEVIDYS
– Treatment with ELEVIDYS corresponded with increases on the North Star Ambulatory Assessment (NSAA) at one year in crossover patients, while the study
About this update from Sarepta Therapeutics, Inc.
[{"type":"text","content":"\n– Treatment with ELEVIDYS corresponded with increases on the North Star Ambulatory Assessment (NSAA) at one year in crossover patients, while the study remained blinded\n\n\n– MRI results at two years in patients treated in Part 1 show minimal muscle pathology progression, aligning closely with observed functional benefits\n\n\n– Crossover-treated patients show statistically significant benefits of ELEVIDYS treatment on NSAA, Time to Rise (TTR), and 10-meter walk/run (10MWR), when compared to a pre-specified, well-matched external control (EC)\n\n\n– Part 1-treated patients show sustained expression at week 64, and functional improvements on NSAA, TTR and 10MWR were sustained two years after treatment and show a widening divergence compared to EC\n\n\n– Safety remained consistent with the profile of ELEVIDYS already established across a broad Duchenne population\n\n\n– Investor webcast to be held today at 8:30 a.m. ET\n\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nSarepta Therapeutics, Inc. (NASDAQ: SRPT), the leader in precision genetic medicine for rare diseases, today announced positive topline results from Part 2 of EMBARK (Study SRP-9001-301), a global, randomized, double-blind, placebo-controlled, Phase 3 clinical study of ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy in patients with Duchenne muscular dystrophy.\n\n\nCrossover-treated patients, those who received a placebo in Part 1 and crossed over at 52 weeks and were treated with ELEVIDYS in Part 2, improved 2.34 points from baseline compared to matched external controls on the North Star Ambulatory Assessment (NSAA) 52 weeks after treatment (P","length":1874,"tagName":"div"}]