Capricor Reports Five-Year Data on Deramiocel

Capricor Therapeutics announced five-year data from its ongoing HOPE-2 open-label extension study of Deramiocel, the company's lead cell therapy candidate for Duchenne Muscular Dystrophy, or DMD. The data will be presented at the Parent Project Muscular Dystrophy annual conference, taking place June 25-27 in Orlando, Florida, alongside previously reported results from the company's HOPE-3 Phase 3 trial. Among the patients who remain enrolled in the HOPE-2 OLE study, treatment with Deramiocel continued to attenuate disease progression over five years, as measured by Performance of the Upper Limb. Patients experienced a mean total-score decline of less than five points over five years. By comparison, a cohort-matched external comparator of standard-of-care DMD patients showed a modeled decline of roughly 2.4 points per year, which if continued would project to approximately 12 points over five years. A separately published natural-history analysis reported a decline in non-ambulant patients of approximately 8.1 points over three years, consistent with a meaningful treatment benefit. Cardiac function, as measured by left ventricular ejection fraction on cardiac MRI, remained stable over the full five-year period, in contrast to the modeled decline of approximately 3.2% per year observed in a propensity-matched external cardiac comparator. Deramiocel continued to demonstrate a favorable safety profile throughout the study, consistent with over 800 intravenous infusions administered to date across the Deramiocel clinical development program.