Capricor Therapeutics to Present HOPE-3 and HOPE-3 Open-Label Extension Data at 2026 World Muscle Society Congress
SAN DIEGO, Sept. 17, 2026 (GLOBE NEWSWIRE) — Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced that data from HOPE-3 and its open-label extension (OLE) of Deramiocel, the Company’s lead asset for the treatment of Duchenne muscular dystrophy (DMD), will be presented in a late-breaking poster and an oral presentation at the 31st Annual Congress of the World Muscle Society (WMS 2026), taking place September 29 – October 3, 2026, in Hiroshima, Japan. The presentations will report skeletal muscle and cardiac outcomes through 24 months, building on the previously reported 12-month HOPE-3 results. Of the 106 patients randomized in HOPE-3, 82 reached the 24-month time point, comprising 40 originally assigned to Deramiocel and 42 to placebo.
“These HOPE-3 24-month data, along with additional analyses supporting Deramiocel’s potential safety and efficacy, were part of our recent major amendment to the Deramiocel BLA,” said Linda Marbán, Ph.D., CEO of Capricor. “We look forward to presenting these results at the World Muscle Society Congress.”
Beyond the Deramiocel program, Capricor will also present two preclinical posters from its StealthX™ exosome platform.
WMS Presentations
Title: Deramiocel slows upper limb decline in the HOPE-3 OLE: cross-phase delayed-start analysis and 2-year comparison with natural history
Presenting author: Dr. Craig McDonald, University of California, Davis
Details: Late-breaking poster session
Title: 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
Presenting author: Dr. Craig McDonald
Details: Oral presentation, clinical trial updates session, October 3, 2026
Title: Engineered muscle-targeting extracellular vesicles for systemic delivery of micro-dystrophin: novel redosable strategy for Duchenne muscular dystrophy
Presenting author: Mafalda Cacciottolo, Ph.D., Capricor Therapeutics
Details: Poster session 2, DMD treatments, September 30, 2026
Title: Delivery of acid α-glucosidase by muscle-targeting extracellular vesicles: a new road for Pompe disease treatment
Presenting author: Mafalda Cacciottolo, Ph.D.
Details: Poster session 3, Glycogenoses, October 2, 2026
Copies of the presentations and posters will be added to the publications section of the Capricor website following each presentation. The full WMS 2026 program is available at https://www.wms2026.com/page/programme.
About the HOPE-3 Study
HOPE-3 is a Phase 3, randomized, double-blind, placebo-controlled trial evaluating Deramiocel in patients with Duchenne muscular dystrophy. The study enrolled 106 patients, randomized to receive Deramiocel or placebo administered intravenously every three months over a 12-month treatment period. HOPE-3 met its primary endpoint, with Deramiocel slowing decline in upper limb function by 54 percent versus placebo as measured by Performance of the Upper Limb (PUL) version 2.0 (p=0.03). One-year results were published in The Lancet in July 2026. Patients who completed the randomized portion of the study were eligible to continue receiving Deramiocel in an open-label extension.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a severe, X-linked genetic disorder characterized by progressive muscle degeneration affecting the skeletal, respiratory, and cardiac muscles. It is caused by the absence of functional dystrophin, a key structural protein in muscle cells. DMD affects approximately 15,000 individuals in the United States and primarily impacts boys. Over time, deterioration of the heart muscle leads to cardiomyopathy and heart failure, the leading cause of death in DMD. There is no cure, and treatment options remain limited.
About Deramiocel
Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert immunomodulatory and anti-fibrotic actions in the preservation of skeletal and cardiac muscle function in muscular dystrophies such as DMD. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing rather than pro-inflammatory phenotype. For the treatment of DMD, 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.
September 17, 2026 8:30 am EDT
– Late-Breaking Data to Include HOPE-3 Delayed-Start and Natural History Analyses Submitted in the Recent Major Amendment to the Deramiocel BLA –
– PDUFA Target Action Date of November 22, 2026 –
