Deramiocel treatment yielded a mean PUL 2.0 decline under 5 points over five years versus a projected 12-point drop, while LVEF remained stable versus a modeled 3.2% annual decrease. The HOPE-3 Phase 3 trial (n=106) achieved primary PUL 2.0 (p=0.03) and cardiac LVEF (p=0.04) endpoints ahead of an August 22, 2026 PDUFA.
Deramiocel maintained durable muscle benefit over five years in the HOPE-2 open-label extension, with mean PUL 2.0 decline under 5 points versus an expected ~12-point drop in a matched standard-of-care cohort. Cardiac MRI showed stable LVEF compared to a modeled 3.2% annual decrease, and no new safety signals emerged after over 800 intravenous infusions.
The multicenter, randomized, double-blind, placebo-controlled HOPE-3 trial enrolled 106 Duchenne muscular dystrophy patients and met its primary PUL 2.0 endpoint (p=0.03) and key cardiac secondary LVEF endpoint (p=0.04), along with all Type I error-controlled secondary endpoints, reinforcing the durable skeletal and cardiac muscle benefits observed in the OLE study.
Capricor’s Biologics License Application for Deramiocel is under FDA review with a PDUFA target action date of August 22, 2026. The company is preparing to present the full clinical data package to regulators and the patient community to support potential approval and future commercialization.
