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2026-07-30 04:54 UTC → 2026-07-30 05:12 UTC ·
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In late July 2026 the The FDA announced that advisory committee voted 9‑to‑3 against recommending approval of Capricor Therapeutics’ experimental stem‑cell therapy deramiocel failed to meet the primary and secondary endpoints of its Phase 3 HOPE‑3 trial for Duchenne muscular dystrophy (DMD), citing a lack of statistically significant improvement in muscle function, cardiac imaging, dystrophy‑related cardiomyopathy, reinforcing earlier staff concerns about insufficient efficacy evidence and safety concerns. Two days later detailed results showed that deramiocel slowed the decline of arm post‑hoc changes to key outcome analyses. The Phase III HOPE‑3 trial, which enrolled 106 boys and elbow movement by young men (54 receiving deramiocel, 52 receiving placebo), showed a 54 % and 65 % respectively slower decline in arm function and appeared up to preserve heart function in participants with pre‑existing cardiomyopathy, with a reported 91 % slower progression of heart‑function loss in that subgroup. participants with pre‑existing cardiomyopathy. Allergic‑type reactions were more common frequent (42 % vs 15 % placebo) but remained mild to moderate moderate, and no deaths occurred. The FDA advisory committee voted 9‑to‑3 against recommending approval, reaffirming concerns about insufficient efficacy evidence and the company’s alteration of key outcome analyses after the trial. The panel also questioned the baseline cardiac function of trial participants. The recommendation vote means a final FDA decision is still expected by 22 August, and Capricor’s shares fell more than 66 % after the staff briefing. Capricor CEO Linda Marbán called the panel’s vote outcome a setback, setback while emphasizing that deramiocel could become deramiocel’s potential as the first therapy to directly target heart disease in Duchenne patients.