Sarepta Therapeutics, Inc. announced topline results from Part 2 of Study SRP-9001-102 (Study 102), an ongoing, randomized, double-blind, placebo-controlled clinical trial to evaluate the safety, efficacy and tolerability of a single dose of SRP-9001 (delandistrogene moxeparvovec) in 41 patients with Duchenne muscular dystrophy, 21 of whom were in the placebo crossover cohort. SRP-9001 is an investigational gene transfer therapy intended to deliver its micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. SRP-9001-treated participants from the placebo crossover group (n=20, aged 5-8 at time of dosing SRP-9001) scored a statistically significant 2.0 points higher on the mean North Star Ambulatory Assessment at 48 weeks compared to propensity-score weighted external controls (p value=0.0009).

Mean NSAA scores from these Part 2 participants improved 1.3 points from baseline for the SRP-9001 treated group and the NSAA scores in the external control group (n=103) declined 0.7 points from baseline. Additional results will be shared at a future medical congress. Study 102 remains ongoing and all participants continue to be monitored for safety in addition to longer-term assessments of functional outcomes.