Capricor Therapeutics Reports Positive Deramiocel Data

SAN DIEGO, Oct. 05, 2026 /CourierPR/ -- Capricor Therapeutics, a biotechnology company focused on developing transformative cell and exosome-based therapeutics for rare diseases, has presented positive data from its ongoing HOPE-3 open-label extension (OLE) study for its lead asset, Deramiocel, at the 31st Annual Congress of the World Muscle Society (WMS) in Hiroshima, Japan. The data were highlighted in a late-breaking poster presentation, supporting the previously reported 12-month results of the randomized, placebo-controlled trial.

The HOPE-3 open-label extension, which analyzed patients who started Deramiocel after a year on placebo, showed significant results. Patients who started Deramiocel after a year on placebo experienced a 76% reduction in upper limb decline compared to their own prior year decline. This crossover analysis, conducted over a 24-month period, underscores the potential of Deramiocel in slowing the progression of upper limb impairment in Duchenne muscular dystrophy (DMD).

The study also revealed that both groups showed a slower decline in upper limb function than what a natural history model predicts. This model, based on studies by Michaëls (2025) and Niks (2026), predicts each patient's decline from baseline score, age, and ambulatory status. In its untreated year, the placebo arm tracked the model (−2.7 observed vs −2.9 predicted at Month 12), while a published non-ambulant cohort showed a similar decline (about −2.7 points per year; Coratti 2025).

The data, which included in Deramiocel’s Biologics License Application (BLA) as part of a major amendment, will be discussed in a webinar scheduled for October 7, 2026, at 1:00 p.m. ET, in collaboration with Parent Project Muscular Dystrophy (PPMD). The webinar will provide a detailed discussion of these results.

The FDA has received these data as part of the recent major amendment to the Deramiocel BLA, along with extensive sensitivity and robustness analyses to support the results of the HOPE-3 randomized clinical trial. Capricor Therapeutics is committed to continuing to work with the FDA through the remainder of the review, ahead of the November 22, 2026, Prescription Drug User Fee Act (PDUFA) target action date.

Deramiocel (CAP-1002) is an allogeneic cardiac-derived cell therapy in late-stage development for the treatment of DMD. The therapy consists of allogeneic cardiosphere-derived cells (CDCs) that have been shown in preclinical and clinical studies to exert immunomodulatory and anti-fibrotic actions in the preservation of skeletal and cardiac muscle function in muscular dystrophies such as DMD. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing rather than pro-inflammatory phenotype.

The 24-month crossover analysis, where patients starting Deramiocel after a year on placebo showed a 76% reduction in upper limb decline compared to their own prior year decline, is a significant finding. The webinar scheduled for October 7, 2026, at 1:00 p.m. ET, in collaboration with Parent Project Muscular Dystrophy (PPMD), will provide a detailed discussion of these results.