San Diego – October 19, 2026 -- Capricor Therapeutics (NASDAQ: CAPR) will present 24-month skeletal muscle and cardiac data from its Phase 3 HOPE-3 trial of Deramiocel in Duchenne muscular dystrophy (DMD) at the World Muscle Society Congress, weeks ahead of an FDA PDUFA target action date of November 22, 2026.
The 31st Annual Congress (WMS 2026) runs September 29 to October 3, 2026, in Hiroshima, Japan. Capricor will deliver one late-breaking poster and one oral presentation on Deramiocel, plus two preclinical posters from its StealthX exosome platform.
HOPE-3 24-month cohort reaches 82 of 106 randomized patients
Of the 106 patients randomized in HOPE-3, 82 reached the 24-month time point: 40 originally assigned to Deramiocel and 42 to placebo. The data build on the 12-month results reported previously.
The late-breaking poster covers a cross-phase delayed-start analysis in the open-label extension (OLE) and a two-year comparison with natural history. Both analyses were submitted in the recent major amendment to the Deramiocel Biologics License Application (BLA). Dr. Craig McDonald of the University of California, Davis, will present.
McDonald will also give the oral presentation on October 3, 2026, in the clinical trial updates session. It addresses evidence for both musculoskeletal and cardiac efficacy.
Primary endpoint showed 54% slower upper limb decline
HOPE-3 is a randomized, double-blind, placebo-controlled study. Patients received Deramiocel or placebo intravenously every three months over a 12-month treatment period. Deramiocel slowed decline in upper limb function by 54 percent versus placebo on the Performance of the Upper Limb (PUL) version 2.0 scale (p=0.03). The one-year results were published in The Lancet in July 2026.
CEO Linda Marbán said the 24-month data and additional supporting analyses formed part of the amended BLA.
Two StealthX exosome posters target DMD and Pompe disease
Mafalda Cacciottolo, Ph.D., of Capricor will present both preclinical posters. The first, on September 30, 2026, covers engineered muscle-targeting extracellular vesicles for systemic delivery of micro-dystrophin as a redosable DMD strategy. The second, on October 2, 2026, covers delivery of acid α-glucosidase by muscle-targeting extracellular vesicles for Pompe disease.
Regulatory designations and litigation backdrop
Deramiocel holds Orphan Drug, RMAT and Rare Pediatric Disease designations in the U.S., and Orphan Drug and ATMP designations in Europe. The Rare Pediatric Disease designation may qualify Capricor for a Priority Review Voucher upon approval. Deramiocel remains investigational and unapproved for commercial use.
Capricor's forward-looking statements also reference ongoing litigation with Nippon Shinyaku Co., Ltd. and NS Pharma, Inc., including its ability to commercialize Deramiocel independent of its existing distribution agreement.