Capricor DMD cell therapy data show 76% upper limb decline slowdown
Capricor Therapeutics has presented 24-month data from the open-label extension of its HOPE-3 trial, showing that patients with Duchenne muscular dystrophy (DMD) who crossed over from placebo to Deramiocel slowed their rate of upper limb decline by 76% in the year after starting treatment. The results were shown as a late-breaking poster at the 31st World Muscle Society Congress in Hiroshima.
The crossover analysis is the headline figure. Patients who spent their first trial year on placebo declined by a least-squares mean of 2.05 points on the Performance of the Upper Limb 2.0 (PUL 2.0) scale. After switching to Deramiocel in the open-label extension, the same group declined by only 0.49 points, a difference of 1.56 points (95% CI: 0.47 to 2.64). Patients who had received Deramiocel from the outset showed a consistent rate of decline across both years, at 0.95 and 0.89 points respectively, suggesting the benefit does not erode over time. Comparison against a natural history model by Michaëls and Niks indicated that both groups were faring better than would be predicted for untreated patients by month 24.
Craig McDonald, principal investigator at UC Davis Health, said the within-patient design lent the findings particular weight. "Being able to follow the same young man before and after he starts treatment is a powerful way to see a treatment effect, and what we saw was consistent with the randomised, placebo-controlled period," he said, adding that upper limb function underpins the practical independence of non-ambulant patients.
Regulatory path
The extension data have been submitted to the US Food and Drug Administration as part of a major amendment to Capricor's Biologics License Application for Deramiocel. The agency has set a PDUFA target action date of 22 November 2026. Chief executive Linda Marbán noted that the company has also filed extensive sensitivity and robustness analyses to support the primary randomised trial results, which were published in The Lancet in July 2026.
Deramiocel holds Orphan Drug, Rare Paediatric Disease and Regenerative Medicine Advanced Therapy designations in the United States and Orphan Drug and Advanced Therapy Medicinal Product designations in Europe. The Rare Paediatric Disease designation could qualify Capricor for a Priority Review Voucher on approval, a transferable asset that has recently traded at between 100 million and 150 million US dollars in secondary markets.
Market context
The DMD treatment landscape has become materially more crowded since the conditional approvals of exon-skipping antisense oligonucleotides and the accelerated approval of Sarepta Therapeutics' SRP-9001 gene therapy. Deramiocel's mechanism is distinct: it uses allogeneic cardiosphere-derived cells that act via secreted exosomes to shift macrophage phenotype toward an anti-inflammatory, pro-repair profile, targeting both skeletal and cardiac muscle. That dual action is increasingly relevant as DMD patients live longer and cardiomyopathy becomes a more pressing clinical concern.
Capricor is also advancing its StealthX exosome delivery platform for oligonucleotides and small molecules, though that programme remains at an earlier stage and does not affect the near-term regulatory narrative for Deramiocel. With a PDUFA date now seven weeks away, investors will be focused on whether the FDA requires an advisory committee meeting and how the agency interprets the open-label, non-randomised nature of the extension data presented at WMS.