Capricor shares 24-month data for Duchenne therapy

Capricor Therapeutics announced positive new data from its ongoing HOPE-3 open-label extension study for its lead asset, deramiocel, for the treatment of upper limb impairment in Duchenne muscular dystrophy (DMD).

Capricor is relying on the 24-month data to support its amended BLA, which faces a November 22 FDA decision. The amendment also includes additional sensitivity and robustness analyses of the randomized HOPE-3 results.

HOPE-3 randomized 106 patients with DMD to receive intravenous deramiocel or placebo every three months for 12 months. A total of 98 patients then entered the open-label extension, where all participants received the therapy. Patients who switched from placebo to deramiocel saw their rate of upper-limb decline drop by 76% during their first year on the cell therapy compared with their prior year on placebo.

On the trial's primary measure, the Performance of the Upper Limb (PUL 2.0) scale, patients in the crossover group lost an average of 2.05 points during their year on placebo, compared with 0.49 points during their first year on deramiocel. Patients treated with deramiocel from the start of the trial declined at a similar rate across both years, losing 0.95 points in year one and 0.89 points in year two. Capricor said the similar rates suggest the slower decline was associated with starting treatment rather than simply the passage of time.

At 24 months, both groups also showed less decline than predicted by natural-history models. The placebo-to-deramiocel group declined 3.76 points compared with a predicted 5.35-point decline, while the early-treatment group declined 3.42 points compared with a predicted 5.88 points. The company cautioned that these natural-history comparisons were descriptive and were not statistically tested.

The data form the centerpiece of Capricor's regulatory strategy following a setback over the summer when FDA's Cellular, Tissue and Gene Therapies Advisory Committee voted 9-3 that the evidence did not support deramiocel's effectiveness for DMD-associated cardiomyopathy. Capricor responded by narrowing its BLA to focus on upper-limb skeletal muscle function — HOPE-3's primary endpoint — and submitting an amendment with the 24-month extension data and additional analyses. CBER classified the submission as a major amendment and pushed the PDUFA date from August 22 to November 22, 2026.

Deramiocel, an allogeneic therapy made from cardiosphere-derived cells, previously received a complete response letter from FDA in July 2025. The therapy holds orphan drug, RMAT and rare pediatric disease designations.