Capricor Therapeutics Presents Positive HOPE-3 Open-Label Extension Data On Upper Limb Function In Duchenne Muscular Dystrophy At 2026 World Muscle Society Congress
| PUL 2.0 Total Score | Year 1 LS mean change a | Year 2 (OLE) LS mean change a | Difference, Year 2 vs Year 1 (95% CI) | Reduction in rate of decline c |
| Year-by-year change | ||||
| Placebo arm (delayed start) b | −2.05 on placebo n=52 | −0.49 on Deramiocel n=49 | 1.56 (0.47 to 2.64) | 76 % |
| Deramiocel arm (early start) b | −0.95 on Deramiocel n=54 | −0.89 on Deramiocel n=49 | 0.05 (−1.03 to 1.14) | - |
| Month 24 change vs natural history d | ||||
| Observed at Month 24 e | Predicted by natural history | |||
| Placebo arm 12 months on Deramiocel | −3.76 | −5.35 | ||
| Deramiocel arm 24 months on Deramiocel | −3.42 | −5.88 |
ITT population: 106 randomized; 98 entered the OLE (49 per arm). Negative values indicate decline. 95% CIs are two-sided. No alpha was allocated to these open-label analyses, and the extension was not powered for Month 24 comparisons.
aLeast-squares mean change from a mixed model for repeated measures, with each year measured from its own starting point (Day 0 or Month 12) and adjusted for starting score, cohort, site and slow-progressor status.
bGroups are named by original randomized assignment.
cDifference as a percentage of the Year 1 decline on placebo.
dObserved mean change (unadjusted) vs a natural history model (Michaëls 2025; Niks 2026) that predicts each patient's decline from baseline score, age and ambulatory status. In its untreated year, the Placebo arm tracked the model (−2.7 observed vs −2.9 predicted at Month 12) and a published non-ambulant cohort (about −2.7 points per year; Coratti 2025). Descriptive only; no matching or statistical testing.
ePatients assessed at Month 24: n=42 (Placebo arm), n=40 (Deramiocel arm).
“Upper limb function is critical to quality of life for young men with DMD because it is directly tied to their independence,” said Craig McDonald, MD, Principal Investigator of the HOPE-3 trial at UC Davis Health.“When the young men who had been declining on placebo started Deramiocel, their rate of upper limb loss dropped by roughly 75% in their first year of treatment. Just as important, patients who were on Deramiocel both years declined at a similar rate in year 1 and year 2, which suggests that the slower decline was triggered by starting treatment, not the passage of time. Being able to follow the same young man before and after he starts treatment is a powerful way to see a treatment effect, and what we saw was consistent with the randomized, placebo-controlled period. As a clinician seeing these patients, I have had very little to offer them that could meaningfully slow the progression of upper limb functional loss once they were non-ambulant. These data suggest there could be a way to treat this aspect of the disease. Their arms are how they feed themselves, control their wheelchairs and use their phones, and how they stay independent. Time is muscle, and slowing that loss means more time doing the things that matter most to them.”
“These results add to a growing body of evidence for Deramiocel,” said Linda Marbán, Ph.D., CEO of Capricor.“What we saw in the HOPE-3 open-label extension aligns with our previous clinical studies, which together show the same pattern across multiple years of treatment. The FDA has received these data as part of the recent major amendment to the Deramiocel Biologics License Application, along with extensive sensitivity and robustness analyses we produced to support the results of the HOPE-3 randomized clinical trial. We will continue to work with the FDA through the remainder of the review, ahead of the November 22, 2026, PDUFA target action date.”
Copies of all Capricor WMS presentations will be made available in the publications section of the Capricor website. The full WMS program is available at wms2026.com/page/programme.
Webinar Information
A webinar is scheduled for Wednesday, October 7, 2026, at 1:00 p.m. ET in collaboration with Parent Project Muscular Dystrophy (PPMD) to discuss these results in more detail. To register for the webinar, please click here. For additional details, please visit Parent Project Muscular Dystrophy's website.
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. 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 (OLE).
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 the treatment of Duchenne muscular dystrophy (DMD), evaluated in clinical studies for its potential to preserve skeletal and cardiac muscle function. Capricor is also advancing its proprietary StealthXTM 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 StealthXTM exosome therapeutics are investigational and have not been approved for commercial use in any indication.
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