Capricor Reports Positive Phase 3 HOPE-3 Results: Deramiocel Demonstrates Skeletal and Cardiac Benefits in Duchenne Muscular Dystrophy

December 3, 2025 — Capricor Therapeutics announced that its investigational cell therapy Deramiocel achieved both primary and key secondary endpoints in the pivotal Phase 3 HOPE-3 trial for Duchenne Muscular Dystrophy (DMD). The trial enrolled approximately 106 adolescent and young adult male patients (mean age ~15) who received either Deramiocel or placebo alongside standard corticosteroid treatment. Over the 12-month study period, Deramiocel demonstrated a 54% improvement in upper-limb function (primary endpoint) and showed statistically significant preservation or improvement of cardiac function, as measured by left ventricular ejection fraction (LVEF), compared to placebo. The treatment was well tolerated with no new serious safety concerns.

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Earlier in 2025, the FDA had rejected Deramiocel’s Biologic License Application (BLA) citing insufficient evidence of effectiveness. The new HOPE-3 data, however, provide robust randomized, placebo-controlled evidence supporting both skeletal and cardiac benefits. Capricor has announced its intention to submit a complete response to the FDA based on these data, seeking regulatory reconsideration.

If approved, Deramiocel could become the first therapy indicated for cardiomyopathy associated with DMD, meeting a critical unmet need. Company executives emphasize that the results reinforce Deramiocel’s clinical potential and position it as a leading candidate in the DMD treatment landscape.

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Source: Capricor Therapeutics press release, Capricor Announces Positive Topline Results from Pivotal Phase 3 HOPE-3 Study of Deramiocel in Duchenne Muscular Dystrophy, December 3, 2025.