SAN DIEGO, Calif. — September 17, 2026
Capricor Therapeutics announced that 24-month data from the HOPE-3 Phase 3 trial and its open-label extension of Deramiocel will be presented at the 31st Annual Congress of the World Muscle Society in Hiroshima, Japan, from September 29 to October 3, 2026. The company will present late-breaking analyses covering skeletal muscle and cardiac outcomes through 24 months, building on previously reported 12-month results. Of the 106 patients randomized in HOPE-3, 82 reached the 24-month time point, including 40 originally assigned to Deramiocel and 42 to placebo. The presentations also include delayed-start and natural history analyses that were submitted as part of a recent major amendment to the Deramiocel Biologics License Application.
HOPE-3 Data Support Deramiocel Development in DMD
HOPE-3 is a Phase 3 randomized, double-blind, placebo-controlled study evaluating Deramiocel in Duchenne muscular dystrophy (DMD). The trial met its primary endpoint, with Deramiocel slowing decline in upper limb function by 54% compared with placebo at 12 months, as measured by the Performance of the Upper Limb version 2.0 assessment (p=0.03). Participants received intravenous Deramiocel or placebo every three months during the randomized treatment period, after which eligible patients could enter an open-label extension and continue treatment with Deramiocel. The upcoming WMS presentations will provide additional follow-up on both musculoskeletal and cardiac outcomes and include comparisons designed to assess treatment effects over longer periods.
WMS Program Highlights Deramiocel and StealthX Research
Capricor will deliver two clinical presentations and two preclinical posters at WMS 2026. A late-breaking poster led by Craig McDonald, M.D., will present a cross-phase delayed-start analysis of upper limb decline and a two-year comparison with natural history. An oral clinical trial update will present evidence supporting musculoskeletal and cardiac outcomes from HOPE-3. Capricor will also present preclinical research from its StealthX™ exosome platform, including engineered muscle-targeting extracellular vesicles designed to deliver micro-dystrophin for DMD and studies evaluating delivery of acid alpha-glucosidase for Pompe disease. The presentations highlight the company’s broader strategy of developing targeted exosome-based delivery technologies alongside its lead cell therapy program.
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Capricor Advances Deramiocel Toward Regulatory Decision
Deramiocel, also known as CAP-1002, is an allogeneic cardiosphere-derived cell therapy being developed for DMD, with proposed mechanisms involving immunomodulatory and anti-fibrotic effects on skeletal and cardiac muscle. The program has received Orphan Drug, Regenerative Medicine Advanced Therapy and Rare Pediatric Disease designations in the United States, as well as Orphan Drug and ATMP designations in Europe. Capricor has identified a November 22, 2026 PDUFA target action date for the Deramiocel BLA. The company’s upcoming 24-month HOPE-3 and open-label extension data are expected to add longer-term clinical evidence to the regulatory package while Capricor continues development of its StealthX platform for targeted delivery of oligonucleotides, proteins and small-molecule therapeutics.
Source: Capricor Therapeutics, press release
