Capricor Shows Statistically Meaningful Upper-Limb Gains in Phase 3 DMD Trial
Capricor presented Phase 3 HOPE-3 trial data for Deramiocel at the AAN meeting showing first statistically and clinically meaningful upper-limb gains on the PUL v2.0 endpoint, with DVA measures indicating slowed self-feeding decline. BLA is under FDA review with a PDUFA date of August 22, 2026, underscoring Deramiocel’s promise in Duchenne therapy.
1. Phase 3 HOPE-3 Results
At the AAN 2026 meeting, Capricor presented data from its HOPE-3 Phase 3 trial of Deramiocel in Duchenne muscular dystrophy. The trial achieved the first statistically and clinically significant improvement in upper-limb function on the PUL v2.0 primary endpoint, supported by DVA measures showing slowed decline in self-feeding ability.
2. Regulatory Status and Timeline
Capricor has submitted a Biologics License Application (BLA) and is awaiting FDA review with a PDUFA target action date of August 22, 2026. Deramiocel also holds Orphan Drug, Regenerative Medicine Advanced Therapy, Advanced Therapy Medicinal Product and Rare Pediatric Disease designations, which may enable accelerated pathways and a priority review voucher.
3. Deramiocel Mechanism and Pipeline
Deramiocel is an allogeneic cardiosphere-derived cell therapy that leverages exosome-mediated immunomodulatory and anti-fibrotic effects to preserve skeletal and cardiac muscle function. Capricor’s proprietary exosome platform further targets oligonucleotides and proteins for potential applications in rare diseases and vaccinology.