Sarepta Therapeutics Inc. (NASDAQ: SRPT) has announced topline data from the second part of the SRP-9001-102 study evaluating the efficacy, tolerability, and safety of a singled SRP-9001 (delandistrogene moxeparvovec) dose in Duchene muscular dystrophy.
Part 2 study evaluated 41 Duchene muscular dystrophy subjects
Part 2 of the study is an ongoing double-blind, randomized, placebo-controlled trial evaluating 41 Duchene muscular dystrophy patients with 21 in a placebo crossover group. SRP-9001 is an experimental gene transfer therapy that delivers the micro-dystrophin-encoding genes to muscle tissue for targeted micro-dystrophin protein synthesis. Results indicated that SRP-9001 treated subjects from the placebo crossover group scored statistically 2 points higher in the average North Star Ambulatory Assessment after 48 weeks.
CEO Doug Ingram said, “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 crossover 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.”
Result validate past findings on SRP-9001
Ingram continued, “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.”
Duchene is a life-threatening, brutal disease, and the company believes SRP-9001 offers a near-term alternative to address the need for a treatment that will change the disease’s trajectory. Most importantly, Sarepta will continue advancing the treatment to bring SRP-9001 to several patients across the U.S. and Globally.