Capricor Therapeutics to Present HOPE-3 and HOPE-3 Open-Label Extension Data at 2026 World Muscle Society Congress
Capricor Therapeutics (NASDAQ: CAPR) will present new data on Deramiocel, its lead investigational therapy for Duchenne muscular dystrophy (DMD), at the 31st Annual Congress of the World Muscle Society (WMS 2026). The congress takes place in Hiroshima, Japan, from 29 September to 3 October 2026.
The San Diego-based biotechnology company, which develops cell- and exosome-based therapeutics for rare diseases, will share results from the Phase 3 HOPE-3 trial and its open-label extension (OLE) in a late-breaking poster and an oral presentation. The data cover skeletal muscle and cardiac outcomes through 24 months, building on the 12-month HOPE-3 results reported previously.
Of the 106 patients randomised in HOPE-3, 82 reached the 24-month mark: 40 originally assigned to Deramiocel and 42 to placebo.
Linda Marbán, PhD, CEO of Capricor, said the 24-month data, together with additional analyses supporting Deramiocel’s potential safety and efficacy, formed part of the company’s recent major amendment to the Deramiocel Biologics License Application (BLA). “We look forward to presenting these results at the World Muscle Society Congress,” she added.
Capricor will also present two preclinical posters from its StealthX™ exosome platform.
Presentations at WMS 2026
Deramiocel slows upper limb decline in the HOPE-3 OLE: cross-phase delayed-start analysis and 2-year comparison with natural history
Presented by Dr Craig McDonald, University of California, Davis. Late-breaking poster session.
HOPE-3, a phase 3 study of deramiocel, an allogeneic cell therapy, in advanced Duchenne muscular dystrophy: evidence to support both musculoskeletal and cardiac efficacy
Presented by Dr Craig McDonald. Oral presentation, clinical trial updates session, 3 October 2026.
Engineered muscle-targeting extracellular vesicles for systemic delivery of micro-dystrophin: novel redosable strategy for Duchenne muscular dystrophy
Presented by Mafalda Cacciottolo, PhD, Capricor Therapeutics. Poster session 2 (DMD treatments), 30 September 2026.
Delivery of acid α-glucosidase by muscle-targeting extracellular vesicles: a new road for Pompe disease treatment
Presented by Mafalda Cacciottolo, PhD. Poster session 3 (Glycogenoses), 2 October 2026.
Copies of each presentation and poster will be added to the publications section of Capricor’s website after they are presented. The full WMS 2026 programme is available at https://www.wms2026.com/page/programme.
About the HOPE-3 study
HOPE-3 is a Phase 3, randomised, double-blind, placebo-controlled trial of Deramiocel in patients with DMD. It enrolled 106 patients, who received either Deramiocel or placebo intravenously every three months over a 12-month treatment period.
The trial met its primary endpoint. Deramiocel slowed the decline in upper limb function by 54% compared with placebo, measured by 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. Patients who completed the randomised portion of the study could continue receiving Deramiocel in the open-label extension.
About Duchenne muscular dystrophy
DMD is a severe, X-linked genetic disorder that causes progressive degeneration of the skeletal, respiratory and cardiac muscles. It results from a lack of functional dystrophin, a key structural protein in muscle cells. The condition affects around 15,000 people in the US, mostly boys. Over time, damage to the heart muscle leads to cardiomyopathy and heart failure, which is the leading cause of death in DMD. There is no cure, and treatment options remain limited.
About Deramiocel
Deramiocel (CAP-1002) is made up of allogeneic cardiosphere-derived cells (CDCs), a rare population of heart cells. In preclinical and clinical studies, CDCs have shown immunomodulatory and anti-fibrotic effects that help preserve skeletal and cardiac muscle function in muscular dystrophies such as DMD.
CDCs work by releasing extracellular vesicles called exosomes. These target macrophages and shift them away from a pro-inflammatory state towards a healing one.
For DMD, Deramiocel holds Orphan Drug, Regenerative Medicine Advanced Therapy (RMAT) and Rare Pediatric Disease designations in the US, and Orphan Drug and Advanced Therapy Medicinal Product (ATMP) designations in Europe. The Rare Pediatric Disease designation may make Capricor eligible for a Priority Review Voucher if the therapy is approved.
