Capricor Therapeutics (NASDAQ:CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for rare diseases, today announced that the U.S. Food and Drug Administration's Cellular, Tissue and Gene Therapies Advisory Committee voted that available evidence did not support the effectiveness of Deramiocel for the treatment of cardiomyopathy in patients with Duchenne muscular dystrophy (DMD), a rare and life-threatening disease for which no approved cardiomyopathy treatment currently exists (3 for, 9 against, 0 abstain). The Committee's vote is non-binding.

The Committee's voting question addressed a narrower indication than Capricor had proposed and did not include a vote on Deramiocel's overall benefit-risk profile. In a separate discussion on upper limb function, the Committee's feedback was directionally supportive of the clinical evidence from the Phase 3 HOPE-3 trial, including results on its primary endpoint, PUL 2.0.