Sarepta Reports Promising Results for Its Experimental Gene Therapy Targeting Duchenne Muscular Dystrophy
Sarepta Therapeutics announced positive results from a phase 2 study on SRP-9001 (delandistrogene moxeparvovec), its experimental gene therapy for Duchenne muscular dystrophy, a progressive and deadly genetic disease with no cure.
Sarepta compared trial volunteers who were switched from placebo to treatment to patients in a separate control group, matched by age and disease severity. Among the treated patients, Sarepta reported an average 1.3-point increase on a standard test of motor function, versus a projected 0.7-point decline for the control group.
The improvement was observed in children who were just over 7 years old at the start of the trial.
Results seen across 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,” said Sarepta’s CEO, Doug Ingram.