Capricor goes from HOPE-3 to promise after deramiocel’s Phase 3 success in Duchenne muscular dystrophy

Edited by Robert McTiernan··2 min read

Capricor Therapeutics trumpeted success in the pivotal Phase 3 HOPE-3 clinical trial for deramiocel as a treatment for boys and young men with Duchenne muscular dystrophy (DMD). The drug candidate met both its primary and key cardiac secondary endpoints, paving the way for a potential regulatory filing and renewed momentum for the company’s lead cell-therapy program.

Deramiocel (also known as CAP-1002) is a biologic therapy derived from allogeneic cardiosphere-derived cells (CDCs). The therapeutic mechanism, as described by Capricor and its partner Nippon Shinyaku, involves immunomodulatory and anti-fibrotic effects: CDCs release extracellular vesicles (exosomes) that alter immune cell behavior and reduce fibrosis in skeletal and cardiac muscle tissue.

Key highlights from HOPE-3

Deramiocel slowed skeletal-muscle disease progression by 54%, based on the Performance of Upper Limb (PUL v2.0) score at 12 months, the trial’s primary endpoint. In a pre-specified key secondary analysis, deramiocel also preserved cardiac function, with treated patients showing a 91% relative benefit in left ventricular ejection fraction (LVEF) compared to placebo. Meanwhile, the therapy maintained a favorable safety and tolerability profile, consistent with previous clinical experience in earlier trials.

According to Capricor, these findings support deramiocel’s potential as a first-in-class therapy designed to treat both the muscular and cardiovascular manifestations of DMD. If approved, deramiocel would mark one of the first therapies capable of addressing both muscle degeneration and cardiomyopathy in DMD, a dual benefit that could significantly affect long-term outcomes and quality of life.

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Capricor said it plans to submit a response to the FDA’s earlier Complete Response Letter (CRL) by incorporating the HOPE-3 data, consistent with prior guidance from the agency.  The company and Nippon Shinyaku are also working on “launch readiness” preparations, which suggests an approval attempt and market introduction could follow in 2026, pending full review.

Implications for DMD field

DMD is rare but severe, progressive neuromuscular disorder affecting about 1 in 3,500 live male births globally. Several disease-modifying therapies (DMTs) have recently been approved or in late-stage development, however, these therapies are limited by genotype specificity, modest efficacy, and accessibility challenges.

  • The first gene therapy – Sarepta Therapeutics’ Elevidys (delandistrogene moxeparvovec) received accelerated FDA approval in 2023 for ambulatory pediatric patients, marking a paradigm shift but with lingering questions about durability and safety.
  • Exon-skipping therapies (eteplirsen, golodirsen, viltolarsen, casimersen) are available for specific mutations, but only benefit subsets of patients and show modest clinical improvements (Mendell et al., 20162).
  • Corticosteroids remain the standard of care, delaying disease progression but associated with significant side effects.

Given DMD’s persistent unmet needs, Capricor’s positive readout for deramiocel is a major value inflection point, particularly given the biotech’s earlier regulatory setbacks. The prospect of a therapy that preserves both muscle function and heart health could transform the standard of care for DMD, especially for non-ambulatory individuals or those with advanced cardiomyopathy.