Deramiocel (CAP-1002) - (HOPE-2)

June 26, 2026: Capricor Therapeutics (NASDAQ: CAPR) said the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) plans to hold an advisory committee meeting on July 29, 2026 to discuss the company’s Biologics License Application (BLA) seeking approval of deramiocel (CAP-1002), an investigational allogeneic cell therapy, for treatment of Duchenne muscular dystrophy (DMD). The company stated the BLA review remains on track with a PDUFA target action date of August 22, 2026.

Capricor said the BLA is supported by the Phase 2 HOPE-2 trial and long-term outcomes from the HOPE-2 open-label extension (HOPE-2-OLE), along with positive Phase 3 HOPE-3 results that achieved statistical significance on the primary endpoint (PUL v2.0), the key secondary cardiac endpoint (LVEF), and all other Type I error-controlled secondary endpoints. The AdCom meeting is expected to be live-streamed.

Deramiocel has Orphan Drug Designation for DMD from the FDA and EMA, and has RMAT (U.S.), ATMP (Europe), and FDA Rare Pediatric Disease Designation (potential Priority Review Voucher upon approval).