Deramiocel (CAP-1002) - (HOPE-3)

July 29, 2026: Capricor Therapeutics (NASDAQ: CAPR) announced publication in The Lancet of results from its pivotal Phase 3 HOPE-3 trial of deramiocel (CAP-1002), an investigational allogeneic cardiosphere-derived cell therapy for Duchenne muscular dystrophy (DMD). The randomized, double-blind, placebo-controlled trial (n=106) met its primary endpoint, with deramiocel slowing upper-limb function decline by 54% versus placebo as measured by PUL 2.0 (p=0.03). The release also states the study showed clinically meaningful cardiac benefit.

Regulatory: Capricor said deramiocel’s BLA remains under active FDA review, with a PDUFA target action date of August 22, 2026, and noted the evidence will be discussed at an upcoming FDA Advisory Committee meeting (date not provided). Deramiocel has Orphan Drug designation (FDA/EMA) and FDA RMAT and Rare Pediatric Disease designations, and ATMP designation in Europe.