Capricor's HOPE-3 cardiac results for Deramiocel win AHA award

Capricor Therapeutics said in a press release that an abstract on cardiac data from its HOPE-3 Phase 3 study of Deramiocel in Duchenne muscular dystrophy, which met the primary endpoint of upper limb function, won the American Heart Association’s Outstanding Research Award in Pediatric Cardiology during Scientific Sessions 2026 in Chicago on November 7. The report contained fresh cardiac MRI findings on myocardial fibrosis, assessed with late gadolinium enhancement, and matched patients from a natural history database who did not receive Deramiocel.

The study randomly assigned 106 patients with Duchenne muscular dystrophy to one of two arms, Deramiocel or placebo, and followed them for 12 months. The primary endpoint, upper limb function, was met. For left ventricular ejection fraction, the key secondary endpoint across all randomized participants, the comparison favored Deramiocel by 1.8 percentage points over placebo but missed significance (p=0.09). In a prespecified subgroup of patients with cardiomyopathy, the difference was 2.8 percentage points (p=0.02). On cardiac MRI, patients treated with Deramiocel had 3.0 fewer newly scarred heart segments than placebo patients (p=0.02). Capricor said those subgroup and scar-imaging p-values were nominal and not adjusted for multiple comparisons.

In a post hoc, non-randomized comparison against matched patients in the Duchenne Muscular Dystrophy Cardiac Care Consortium's natural history dataset, Deramiocel was associated with a 2.3 percentage point advantage in overall left ventricular ejection fraction (p=0.02). The cardiomyopathy subgroup difference was 2.8 percentage points (p=0.01), and scar imaging showed 2.9 fewer new segments (p=0.01). The scar-imaging analyses included only 9 Deramiocel-treated patients, compared with 13 placebo patients in the randomized comparison and 62 matched patients in the natural history comparison.

“Cardiomyopathy is the leading cause of death in patients with Duchenne muscular dystrophy,” said Jonathan Soslow, professor of pediatrics at Vanderbilt University Medical Center, who led the abstract. He noted that the cardiomyopathy subgroup had the largest gains in ventricular function, and those on treatment had slower accumulation of heart scar. Duchenne muscular dystrophy affects about 15,000 people in the U.S., primarily boys.

Capricor’s chief executive, Linda Marbán, said, “Our Biologics License Application remains under FDA review, with a PDUFA target action date of November 22, 2026.” She added that the 12-month HOPE-3 findings appeared in The Lancet and these cardiac results have now earned the AHA award. Deramiocel is an allogeneic cardiosphere-derived cell therapy given intravenously every three months. The product is investigational and has not been approved for any indication.