Press release
Sarepta Therapeutics Announces Top-line Results for Part 1 of Study 102 Evaluating SRP-9001, its Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy
-- Study met the primary biological endpoint of micro-dystrophin protein expression at 12 weeks post-treatment, as measured by western blot, in
About this update from Sarepta Therapeutics, Inc.
[{"type":"text","content":"-- Study met the primary biological endpoint of micro-dystrophin protein expression at 12 weeks post-treatment, as measured by western blot, in SRP-9001-treated participants versus placebo -- -- SRP-9001-treated participants showed an increase in NSAA total score compared to placebo at 48 weeks; however, the study did not achieve statistical significance on the primary functional endpoint of improvement in NSAA total score compared to placebo at 48 weeks post-treatment -- -- In the pre-specified analysis by age-group, by which the randomization was stratified, participants aged 4-5 years at time of treatment with SRP-9001 demonstrated a statistically significant improvement in NSAA total score versus the age-matched placebo cohort, achieving a 4.3-point improvement on NSAA at 48 weeks post-treatment from baseline -- -- No new safety signals identified for SRP-9001, reinforcing the favorable safety profile observed to date -- -- Sarepta to host conference call at 4:30 p.m. Eastern time -- CAMBRIDGE, Mass., Jan. 07, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced top-line results from Part 1 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 (rAAVrh74.MHCK7.micro-dystrophin) in 41 patients with Duchenne muscular dystrophy. 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. At 12 weeks post-treatment compared to baseline, the study met its primary biological endpoint of micro-dystrophin protein expression (P","length":2200,"tagName":"div"}]