Capricor Therapeutics to Present HOPE-3 and HOPE-3 Open-Label Extension Data at 2026 World Muscle Society Congress
Capricor links new 24‑month HOPE-3 data and StealthX preclinical work to the ongoing BLA review for Deramiocel in Duchenne muscular dystrophy.
Rhea-AI Summary
Capricor Therapeutics (CAPR) will present 24‑month data from the Phase 3 HOPE-3 trial and its open-label extension of Deramiocel in Duchenne muscular dystrophy at the 2026 World Muscle Society Congress in Hiroshima, Japan.
The late-breaking presentations will include skeletal muscle and cardiac outcomes, a delayed-start analysis and natural history comparisons that were included in a recent major amendment to the Deramiocel BLA, which has a PDUFA target action date of November 22, 2026. HOPE-3 randomized 106 patients; 82 reached 24 months (40 originally on Deramiocel, 42 on placebo). The trial met its primary endpoint, with Deramiocel slowing decline in upper limb function by 54% versus placebo on PUL 2.0 (p=0.03). Capricor will also present two preclinical posters from its StealthX exosome platform.
Positive
- PDUFA date set: Deramiocel BLA has a PDUFA target action date of November 22, 2026
- HOPE-3 primary endpoint met: Deramiocel slowed upper limb decline by 54% vs placebo on PUL 2.0 (p=0.03)
- Longer-term follow-up: 82 of 106 randomized HOPE-3 patients reached the 24‑month time point in the study/OLE
Negative
- None.
Details
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Key Figures
- 24-month outcomes
- 24 months
- HOPE-3 and open-label extension presentations
- Patients randomized
- 106 patients
- Phase 3 HOPE-3 study
- Patients at 24 months
- 82 patients
- 40 Deramiocel and 42 placebo patients
- PDUFA target action date
- November 22, 2026
- Deramiocel BLA
- Upper limb decline reduction
- 54 percent
- Deramiocel versus placebo on PUL version 2.0
- Primary endpoint p-value
- p=0.03
- HOPE-3 upper limb function endpoint
Key Terms
pdufa regulatory
open-label extension medical
phase 3 medical
extracellular vesicles medical
AI-generated analysis. How Rhea-AI works. Not financial advice.
– 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 –
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.
About Capricor Therapeutics
Capricor Therapeutics (NASDAQ: CAPR) is a biotechnology company dedicated to advancing cell and exosome-based therapeutics for the treatment of rare diseases. Our lead product candidate, Deramiocel, is an allogeneic cardiac-derived cell therapy in late-stage development for Duchenne muscular dystrophy (DMD), evaluated in clinical studies for its potential to preserve skeletal and cardiac muscle function. Capricor is also advancing its proprietary StealthX™ exosome platform for the targeted delivery of oligonucleotides, proteins, and small-molecule therapeutics across a range of diseases. At Capricor, we are committed to delivering new therapies for patients with rare diseases. For more information, visit capricor.com and follow Capricor on Facebook, Instagram and X.
Cautionary Note Regarding Forward-Looking Statements
Statements in this press release regarding the efficacy, safety, and intended utilization of Capricor’s product candidates; the initiation, conduct, size, timing and results of clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including future interactions with regulatory authorities and the ability to obtain regulatory approvals or otherwise bring products to market; manufacturing capabilities; dates for regulatory meetings; the potential that required regulatory inspections may be delayed or not be successful which would delay or prevent product approval, revenue and reimbursement estimates, projected terms of definitive agreements, our financial position, our possible uses of existing cash and investment resources; results of securities litigation; and statements regarding our litigation with Nippon Shinyaku Co., Ltd. and NS Pharma, Inc., including the nature of the dispute, our expectations regarding any legal proceedings, and our ability to commercialize Deramiocel independent of our existing distribution agreement and any other statements about Capricor’s management team’s future expectations, beliefs, goals, plans or prospects constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words “believes,” “plans,” “could,” “anticipates,” “expects,” “estimates,” “should,” “target,” “will,” “would” and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements. More information about these and other risks that may impact Capricor’s business is set forth in Capricor’s Annual Report on Form 10-K for the year ended December 31, 2025, as filed with the Securities and Exchange Commission on March 17, 2026 and in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2026, as filed with the Securities and Exchange Commission on August 14, 2026. All forward-looking statements in this press release are based on information available to Capricor as of the date hereof, and Capricor assumes no obligation to update these forward-looking statements.
Deramiocel and Capricor’s StealthX™ exosome therapeutics are investigational and have not been approved for commercial use in any indication.
For more information, please contact:
Capricor Media Contact:
Caitlin Kasunich / Raquel Cona
KCSA Strategic Communications
ckasunich@kcsa.com / rcona@kcsa.com
212.896.1241 / 516.779.2630
Capricor Company Contact:
AJ Bergmann, Chief Financial Officer
abergmann@capricor.com
858.727.1755
FAQ
AI-generated questions and answers. How Rhea-AI works. Not financial advice.
What is the design of the HOPE-3 trial for Deramiocel in Duchenne muscular dystrophy?
HOPE-3 is a Phase 3, randomized, double-blind, placebo-controlled trial in patients with Duchenne muscular dystrophy. It enrolled 106 patients who were randomized to receive Deramiocel or placebo, administered intravenously every three months over a 12‑month treatment period. Patients who completed the randomized portion were eligible to continue receiving Deramiocel in an open-label extension.
What is Deramiocel and how is it characterized in this announcement?
Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells, a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert immunomodulatory and anti-fibrotic actions that preserve skeletal and cardiac muscle function in muscular dystrophies such as Duchenne muscular dystrophy. The cells secrete extracellular vesicles (exosomes) that target macrophages and shift them toward a healing phenotype.
What regulatory designations does Deramiocel currently hold for Duchenne muscular dystrophy?
For the treatment of Duchenne muscular dystrophy, Deramiocel holds Orphan Drug, Regenerative Medicine Advanced Therapy (RMAT) and Rare Pediatric Disease designations in the United States, and Orphan Drug and Advanced Therapy Medicinal Product (ATMP) designations in Europe. The Rare Pediatric Disease designation may qualify Capricor for a Priority Review Voucher upon approval.
Which additional Capricor programs will be presented at the 2026 World Muscle Society Congress?
Beyond Deramiocel, Capricor will present two preclinical posters from its StealthX exosome platform: one on engineered muscle-targeting extracellular vesicles for systemic delivery of micro-dystrophin as a redosable strategy for Duchenne muscular dystrophy (Poster session 2, DMD treatments, September 30, 2026) and another on delivery of acid α-glucosidase by muscle-targeting extracellular vesicles as a potential new approach for Pompe disease treatment (Poster session 3, Glycogenoses, October 2, 2026).
Where and when will the HOPE-3 and HOPE-3 OLE data be presented at WMS 2026?
The HOPE-3 24‑month and open-label extension data will be presented in a late-breaking poster titled “Deramiocel slows upper limb decline in the HOPE-3 OLE: cross-phase delayed-start analysis and 2-year comparison with natural history” and in an oral presentation titled “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.” Both will be part of the 31st Annual Congress of the World Muscle Society, held September 29 – October 3, 2026, in Hiroshima, Japan, with the oral presentation scheduled in the clinical trial updates session on October 3, 2026.