Sarepta DMD Treatment Improves Motor Skills in Study Presented at JPM

Sarepta_Courtesy of Sarepta

Courtesy of Sarepta Therapeutics

Sarepta Therapeutics shared positive topline results from its ongoing SRP-9001-102 study (Study 102) on the effectiveness of delandistrogene moxeparvovec in treating Duchenne muscular dystrophy (DMD).

SRP-9001 is an investigation gene transfer treatment that works by delivering the micro-dystrophin-encoding gene to muscle tissues with the goal to enhance micro-dystrophin protein production. The study had 41 participants aged four to seven years and diagnosed with rare and fatal neuromuscular genetic disease. They were evaluated in a double-blind, 1:1 randomized, placebo-controlled trial at 12 and 48 weeks for micro-dystrophin expression and changes in the North Star Ambulatory Assessment (NSAA) score, respectively. 

NSAA is a 17-item rating procedure that measures functional motor abilities in ambulant patients with Duchenne. It is considered one of the primary endpoints for this trial because it helps researchers track disease progression and treatment outcomes. 

The latest results, reflecting Part 2 of the study, showed that participants from the placebo crossover group (from Part 1) scored 2 points higher on the NSAA at week 48. The patients' safety profile also remained consistent with Part 1 outcomes, with no deaths, study discontinuations, and treatment-linked serious adverse events. Common side effects were also the same as the first study, which included vomiting. Study 102 is still running as of this writing. 

DMD affects one in every 3,500 to 5,000 males worldwide, and symptoms typically emerge in the infant to toddler stage. It is caused by a mutation or change in the gene that controls dystrophin productions, which eventually leads to difficulty in walking or standing from a seated position and muscle weakness. Most patients will require using a wheelchair full-time by the time they are teenagers, which over time results in the loss of one's ability to perform activities of daily living. 

"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," commented Doug Ingram, the president and chief executive officer of Sarepta, in a statement. 

"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 III placebo-controlled global study presently underway and dosing," Ingram added. 

Sarepta said it plans to commercialize SRP-9001 in the U.S. after obtaining approval from the U.S. Food and Drug Administration. The company partnered with Roche in December 2019 to combine the latter's global reach with its gene therapy candidate for Duchenne, with the goal to expand its presence outside of the U.S. The micro-dystrophin gene therapy program used in this development effort was initially developed at the Abigail Wexner Research Institute at Nationwide Children's Hospital.  

Details of the interim results were presented at the 40th Annual J.P. Morgan Healthcare Conference.

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