Clinical TrialApr 22, 2026, 09:03 AM
Capricor presents positive Phase 3 Deramiocel data for DMD at AAN
AI Summary
Capricor Therapeutics announced the presentation of positive data from its Phase 3 HOPE-3 clinical trial of Deramiocel for Duchenne muscular dystrophy (DMD) at the American Academy of Neurology (AAN) 2026 Annual Meeting. The data highlighted a statistically and clinically meaningful difference in the upper limb functional primary endpoint (PUL v2.0) and a meaningful slowing of disease progression in self-feeding ability. The company emphasized that these findings reinforce Deramiocel's potential as a transformative therapy, with its Biologics License Application (BLA) currently under FDA review and a PDUFA target action date of August 22, 2026.
Key Highlights
- Presented positive Phase 3 HOPE-3 data for Deramiocel in Duchenne muscular dystrophy (DMD) at AAN 2026.
- Data showed statistically and clinically meaningful difference in upper limb functional primary endpoint.
- Alternative measures indicated meaningful slowing of disease progression in self-feeding ability.
- Deramiocel's Biologics License Application (BLA) is currently under FDA review.
- PDUFA target action date for Deramiocel is August 22, 2026.