Capricor to present 24-month Deramiocel DMD data at WMS 2026
Capricor Therapeutics will present 24-month data from its HOPE-3 Phase 3 trial and open-label extension (OLE) of Deramiocel at the 31st Annual World Muscle Society Congress, running 29 September to 3 October 2026 in Hiroshima, Japan. The presentations cover skeletal muscle and cardiac outcomes for patients with advanced Duchenne muscular dystrophy (DMD) and arrive at a critical moment: the FDA has set a PDUFA target action date of 22 November 2026 for the Deramiocel biologics licence application.
Of the 106 patients randomised in HOPE-3, 82 reached the 24-month timepoint, comprising 40 originally assigned to Deramiocel and 42 from the placebo arm. The extended dataset formed part of a major amendment to the BLA submitted ahead of the November review deadline. Previously published 12-month results, reported in The Lancet in July 2026, showed Deramiocel slowing decline in upper limb function by 54 per cent versus placebo on the Performance of the Upper Limb 2.0 scale (p=0.03).
Clinical programme at WMS 2026
Capricor has four presentations scheduled across the congress. The two Deramiocel sessions are headlined by an oral presentation on 3 October, in which Dr Craig McDonald of the University of California Davis will summarise musculoskeletal and cardiac evidence from HOPE-3. A late-breaking poster from the same investigator will present a cross-phase delayed-start analysis and a two-year comparison with natural history data, both submitted in the BLA amendment.
Beyond Deramiocel, Capricor will show two preclinical posters from its StealthX exosome platform. One describes muscle-targeting extracellular vesicles carrying micro-dystrophin as a potentially redosable approach to DMD; the second explores the same delivery mechanism for acid alpha-glucosidase in Pompe disease. These presentations signal that Capricor is building a broader pipeline behind Deramiocel, though both programmes remain at preclinical stage.
Regulatory and competitive context
Deramiocel holds Orphan Drug, RMAT and Rare Pediatric Disease designations in the United States, and Orphan Drug and ATMP designations in Europe. The Rare Pediatric Disease designation could entitle Capricor to a priority review voucher on approval, an asset that has traded at significant premiums in recent years and would provide meaningful non-dilutive capital for a company of Capricor's size.
The DMD treatment landscape has become considerably more active in recent years. Sarepta Therapeutics received accelerated approval for its Elevidys gene therapy in 2023 and obtained broader approval in 2024, establishing a high bar for new entrants. Other modalities under investigation include exon-skipping antisense oligonucleotides, stop-codon readthrough agents and additional gene therapy platforms. Deramiocel's cell-based, immunomodulatory mechanism differentiates it from these approaches, targeting macrophage polarisation and anti-fibrotic activity rather than dystrophin restoration directly. Whether that distinction translates into a complementary or competitive positioning relative to Elevidys will be a key commercial question for Capricor's commercial planning team.
The WMS congress presentations will be publicly available on the Capricor website following each session. Investors and clinicians will pay particular attention to the delayed-start and natural history comparisons, which are designed to strengthen the causal interpretation of the 12-month primary endpoint result. FDA reviewers will have seen the same data in the BLA amendment; how those analyses hold up in scientific debate at WMS may shape the pre-approval narrative ahead of the November decision.