Benzinga | Jan 10, 2022 10:51AM EST

Sarepta Therapeutics Reports Its SRP-9001 Showed Statistically Significant Improvements vs Pre-Specified Matched External Control In Part 2 Of Study

* Participants from the placebo crossover group in Part 2 of Study SRP-9001-102 , scored 2.0 points higher on the mean North Star Ambulatory Assessment (NSAA) 48 weeks after treatment with SRP-9001 compared to pre-specified matched external control cohort ( p value=0.0009)

* Results continue to reinforce the tolerability profile of SRP-9001 with no new safety signals identified

CAMBRIDGE, Mass., Jan. 10, 2022 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today 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. Results were presented today at the 40th Annual J.P. Morgan Healthcare Conference.

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.

"We are delighted to report positive results for Part 2 of our blinded, placebo-controlled Study 102 in Duchenne, where the 48-week functional benefits of SRP-9001 in patients dosed at cross-over were statistically significant when compared to pre-specified matched external controls. Furthermore, the safety profile of SRP-9001 remains consistent with the wealth of previous clinical data," said Doug Ingram, president and chief executive officer, Sarepta. "Study 102, Part 2 results add to the totality of evidence for SRP-9001 generated thus far with promising results across multiple clinical trials and more than 80 patients dosed, encompassing a wide range of phenotypes as well as the oldest and heaviest Duchenne patients to be dosed with a full body AAV gene therapy infusion to date. The totality of results that we have seen across our multiple trials bolsters our confidence in the potential disease-modifying benefits of this therapy and reinforces our conviction in the probability of success of EMBARK, our large, phase 3 placebo-controlled global study presently underway and dosing. We are reminded daily that Duchenne is a brutal, life-ending disease and SRP-9001 is the greatest near-term hope we all have to address the need for a therapy that changes the trajectory of this disease. We will continue to move as quickly as possible to bring SRP-9001 to patients in the United States and around the world."

The safety profile of patients treated in Part 2 of Study 102 is consistent with that seen in Part 1. There were no treatment-related serious adverse events, no deaths and no study discontinuations due to an adverse event. The most common treatment-related adverse event in patients treated in Part 2 was vomiting, similar to Part 1. For patients treated in Part 1, no new safety signals emerged after two years of follow up.

Study 102 remains ongoing and all participants continue to be monitored for safety in addition to longer-term assessments of functional outcomes.

*The NSAA is a 17-item rating scale that is used to measure functional motor abilities in ambulant individuals with Duchenne. It is used to monitor the progression of the disease and treatment effects which makes it suitable as an endpoint in clinical trials for Duchenne.

**The external control used a prospectively defined consolidated comparison group of Duchenne patients, matched for variables including age, steroid usage, baseline NSAA and timed function tests with the participants in Study 102. The prospectively defined propensity score analysis allows for a robust balancing of the multiple variables.