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

August 13, 2026: Capricor Therapeutics (NASDAQ: CAPR) said deramiocel’s BLA remains under active FDA review for Duchenne muscular dystrophy (DMD). On July 29, 2026, the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee voted 3-9 against whether available evidence showed substantial evidence of effectiveness for treatment of cardiomyopathy in DMD; Capricor said the vote did not address the HOPE-3 primary endpoint or overall benefit-risk, and that discussion of upper limb function was directionally supportive. The recommendation is non-binding.

Capricor also said the full HOPE-3 Phase 3 dataset was published in The Lancet in July 2026. Deramiocel showed statistically significant slowing of upper limb disease progression on PUL 2.0 (p=0.029). After reverting to the pre-unblinding statistical analysis plan during peer review, the left ventricular ejection fraction result changed to p=0.09 with a 1.8 percentage point treatment difference, versus topline p=0.04 and 2.4 points; the pre-specified cardiomyopathy subgroup remained p=0.02, and the primary endpoint was unchanged.

FDA also conducted a July 2026 BIMO inspection and issued one Form 483 observation; Capricor has responded and is awaiting feedback. The company withdrew a preliminary injunction motion in its NS Pharma dispute and expects arbitration to begin this fall.