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

July 30, 2026: Capricor Therapeutics (NASDAQ: CAPR) announced the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee voted that the available evidence did not support the effectiveness of deramiocel (CAP-1002) for treating cardiomyopathy in patients with Duchenne muscular dystrophy (DMD) (3 for, 9 against, 0 abstain). The vote is non-binding.

Capricor said the committee’s voting question covered a narrower indication than the company proposed and did not include a vote on deramiocel’s overall benefit-risk profile. In a separate discussion focused on upper limb function, the committee’s feedback was described as directionally supportive of the Phase 3 HOPE-3 trial evidence, including results on the primary endpoint (PUL 2.0).

Capricor stated it remains focused on working with FDA toward potential approval ahead of the August 22, 2026 PDUFA target action date. The release also notes deramiocel has FDA and EMA Orphan Drug Designation for DMD, FDA RMAT designation, EU ATMP designation, and FDA Rare Pediatric Disease Designation (which may qualify for a Priority Review Voucher upon approval).