Capricor's HOPE-3 DMD Data Published in The Lancet; FDA 483 Issued
Capricor Therapeutics announced the publication of final one-year results from its HOPE-3 Phase 3 clinical trial for Deramiocel in Duchenne muscular dystrophy (DMD) in The Lancet. The trial met its primary endpoint, showing Deramiocel slowed upper limb function decline by 54% (p=0.03). An update to the statistical model for the key secondary endpoint, LVEF, resulted in a p=0.09 (1.8 percentage point difference), compared to p=0.04 previously reported. Additionally, the company received a Form FDA 483 with one observation following a Bioresearch Monitoring (BIMO) inspection related to its Biologics License Application (BLA) for Deramiocel, which remains under active FDA review with a PDUFA target action date of August 22, 2026.
Key Highlights
- Capricor's HOPE-3 Phase 3 DMD data published in The Lancet.
- Deramiocel slowed upper limb function decline by 54% (p=0.03).
- Updated LVEF statistical model yields p=0.09 (1.8 percentage point difference).
- Cardiomyopathy subgroup LVEF result is nominally significant at p=0.02.
- FDA BIMO inspection concluded July 20, 2026, with one Form 483 observation.
- Observation relates to SOPs, documentation, vendor oversight, and audit report timeliness.
- Deramiocel BLA remains under active FDA review with PDUFA date of August 22, 2026.
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