Capricor says Phase 3 HOPE-3 trial shows deramiocel slows upper limb decline 54% in Duchenne muscular dystrophy
CAPR•BLA remains under FDA review
Deramiocel’s BLA remains under FDA review, with a PDUFA target action date of Aug. 22, 2026.
Cardiac benefit also observed
Data also indicated a clinically meaningful benefit in heart function, which the company said supports a broader disease-modifying effect.
HOPE-3 trial results published in The Lancet
Capricor Therapeutics reported that The Lancet published results from its Phase 3 HOPE-3 trial of deramiocel in Duchenne muscular dystrophy.
The company said the results were already presented in the journal publication and were not flagged for a future conference readout.
Primary endpoint met in largely non-ambulatory patients
The study met its main goal, showing deramiocel slowed functional decline versus placebo in largely non-ambulatory patients.
Capricor said the treatment slowed upper limb decline by 54%.
