Sarepta reports topline results from EMBARK study

Sarepta Therapeutics announced topline results from EMBARK (Study SRP-9001-301), a global, randomized, double-blind, placebo-controlled, Phase 3 clinical study of ELEVIDYS (delandistrogene moxeparvovec-rokl) in patients with Duchenne muscular dystrophy between the ages of 4 through 7 years. “The results of EMBARK, our double-blind, placebo-controlled trial, support the conclusion that ELEVIDYS modifies the trajectory of Duchenne and benefits patients across age groups living with this ferociously degenerative disease. The results favored ELEVIDYS across all endpoints in the study, including achieving statistical significance on all pre-specified key secondary endpoints and in each age subgroup of the key secondary endpoints. Indeed, passing 5 seconds on time to rise is the strongest predictor of early loss of ambulation and in EMBARK, ELEVIDYS reduced those odds over 52 weeks by greater than 90 percent,” said Doug Ingram, president and chief executive officer, Sarepta. “Based on the EMBARK results, we intend to move swiftly to request an update to expand the labeled indication to treat all patients. Importantly, we have shared the EMBARK topline results with FDA leadership and they have confirmed that, based on the totality of the evidence, they are open to such label expansion if supported by review of the data, and that they intend to proceed rapidly with consideration of the submission.” In the study, ELEVIDYS-treated patients improved 2.6 points on their North Star Ambulatory Assessment (NSAA) total score 52 weeks after treatment compared to 1.9 points in placebo-treated patients. The difference of 0.65-points between treated and placebo groups did not reach statistical significance (n=125; p=0.24). All key pre-specified functional secondary endpoints demonstrated robust evidence for a clinically meaningful treatment benefit that was consistent across age groups in ELEVIDYS-treated patients compared to placebo at 52 weeks.