Clinical TrialJun 26, 2026, 09:06 AM
Capricor Deramiocel shows durable 5-year muscle benefit in DMD
AI Summary
Capricor Therapeutics announced positive five-year data from its ongoing HOPE-2 Open-Label Extension (OLE) study of Deramiocel for Duchenne Muscular Dystrophy (DMD). The data showed durable skeletal and cardiac muscle benefit, with attenuated disease progression and stable cardiac function, maintaining a favorable safety profile. These findings will be presented alongside previously reported positive results from the HOPE-3 Phase 3 trial, which met its primary and key secondary endpoints. The company's Biologics License Application (BLA) for Deramiocel is currently under FDA review, with a PDUFA target action date of August 22, 2026.
Key Highlights
- Deramiocel demonstrated durable skeletal and cardiac muscle benefit over five years in HOPE-2 OLE study.
- PUL 2.0 year-over-year decline attenuated to approximately 1 point in HOPE-2 OLE.
- Patients experienced a mean total-score decline of less than 5 points over five years in HOPE-2 OLE.
- Cardiac function (LVEF) remained stable over five years in HOPE-2 OLE, contrasting modeled decline of 3.2% per year.
- HOPE-3 Phase 3 trial met its primary endpoint (PUL 2.0, p=0.03) and key secondary cardiac endpoint (LVEF, p=0.04).
- Deramiocel maintained a favorable safety profile across both studies, with no new safety signals.
- Biologics License Application (BLA) for Deramiocel is under FDA review with an August 22, 2026, PDUFA target action date.
- Over 800 intravenous infusions administered to date across the Deramiocel clinical development program.
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