Capricor Therapeutics Presents Positive HOPE-3 Open-Label Extension Data on Upper Limb Function in Duchenne Muscular Dystrophy at 2026 World Muscle Society Congress

The extension data are part of Deramiocel’s FDA review, with a target action date of November 22, 2026.

Rhea-AI Summary

Capricor Therapeutics (NASDAQ: CAPR) presented HOPE-3 extension data showing 76% slower upper limb decline after placebo patients switched to Deramiocel.

On the Performance of Upper Limb 2.0 scale, delayed-start patients declined 2.05 points during their placebo year versus 0.49 during their first treatment year. Patients treated both years declined 0.95 and 0.89 points, respectively. At Month 24, delayed-start patients declined 3.76 points versus 5.35 predicted by natural history; early-start patients declined 3.42 versus 5.88 predicted.

The open-label analyses had no allocation for statistical significance testing, and the extension was not powered for Month 24 comparisons. Natural-history comparisons were descriptive, without matching or statistical testing. The data were included in the Deramiocel biologics license application major amendment; the FDA target action date is November 22, 2026.

Positive

• Minor pointDelayed-start patients showed 76% slower upper limb decline during their first Deramiocel year versus their placebo year.
• Minor pointDelayed-start patients’ Month 24 decline was 3.76 points versus 5.35 predicted by natural history, descriptively.
• Minor pointEarly-start patients’ Month 24 decline was 3.42 points versus 5.88 predicted by natural history, descriptively.
• Minor pointDeramiocel’s biologics license application major amendment included the extension data and randomized-trial sensitivity analyses.

Negative

• Minor pointOpen-label analyses had no allocation for statistical significance testing; the extension was not powered for Month 24 comparisons.
• Minor pointNatural-history comparisons were descriptive only, without matching or statistical testing.
• Minor pointDeramiocel’s FDA review remains pending, with a November 22, 2026 target action date.

Key Figures

Reduction in rate of decline: 76% Year-over-year PUL 2.0 difference: 1.56 points (95% CI 0.47 to 2.64) HOPE-3 population: 106 randomized; 98 entered the OLE +1 more

Reduction in rate of decline

76%

Placebo arm after starting Deramiocel; Year 2 versus its prior year on placebo

Year-over-year PUL 2.0 difference

1.56 points (95% CI 0.47 to 2.64)

Placebo arm, Year 2 versus Year 1

HOPE-3 population

106 randomized; 98 entered the OLE

HOPE-3 study

PDUFA target action date

November 22, 2026

Deramiocel BLA review

Historical Context

1 past event · Latest: Sep 17

1 event

1. Sep 17

   clinical presentation notice

   24h Move

   +10.0%

   Previewed 24-month HOPE-3 extension data and cited the trial's 12-month endpoint results.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Key Terms

pdufa, open-label extension, least-squares mean

3 terms

pdufa regulatory

"PDUFA Target Action Date November 22, 2026"

PDUFA is the Prescription Drug User Fee Act, the U.S. law under which drug companies pay fees that fund the FDA's review of new medicines. In company news the term usually appears as the PDUFA date, the target deadline by which the FDA aims to decide on a drug application; that date tells investors when to expect the approval or rejection decision for the product.

open-label extension medical

"ongoing HOPE-3 open-label extension (OLE) study"

An open-label extension is a continuation of a clinical trial where all participants and researchers know which treatment is being given, often after an initial blinded phase. It allows further study of a drug's long-term safety and effectiveness. For investors, it can indicate ongoing interest and confidence in a product's potential, influencing perceptions of its future value.

least-squares mean technical

"Least-squares mean change from a mixed model for repeated measures"

An adjusted average calculated from a statistical model that estimates what a group’s typical outcome would be after accounting for other influencing factors (like age, baseline status, or different study conditions). Investors use it to compare results more fairly—for example, to judge a drug’s effect or a strategy’s performance while removing imbalances—so it helps assess true effect sizes that can affect clinical, regulatory, and valuation decisions.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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-24-Month Crossover Analysis: Patients Starting Deramiocel After a Year on Placebo Slowed Upper Limb Decline by 76% Compared with Their Own Prior Year-

-At 24 Months, Both Groups Showed Slower Decline in Upper Limb Function Than Natural History Predicts-

-Data Included in Deramiocel BLA Major Amendment; PDUFA Target Action Date November 22, 2026-

-Webinar Scheduled for October 7, 2026, at 1:00 p.m. ET in Collaboration with PPMD to Discuss the Data-

SAN DIEGO, Oct. 05, 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 positive new data from its ongoing HOPE-3 open-label extension (OLE) study for its lead asset, Deramiocel, for the treatment of upper limb impairment in Duchenne muscular dystrophy (DMD). The data were highlighted in a late-breaking poster presentation at the 31^st Annual Congress of the World Muscle Society (WMS) in Hiroshima, Japan, supporting HOPE-3’s previously reported 12-month results, which were also presented in a separate oral session on Deramiocel's musculoskeletal and cardiac efficacy.

Key results from the late-breaking abstract are summarized below.

HOPE-3 Open-Label Extension: Upper Limb Function (PUL 2.0)

PUL 2.0 Total Score	Year 1 LS mean changea	Year 2 (OLE) LS mean changea	Difference, Year 2 vs Year 1 (95% CI)	Reduction in rate of declinec
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 historyd
	Observed at Month 24e		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 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 did Capricor’s HOPE-3 extension show after placebo patients switched to Deramiocel?

Patients who switched from placebo to Deramiocel showed a 76% reduction in the rate of upper limb decline compared with their own prior placebo year. Their adjusted mean score change was −2.05 points on placebo and −0.49 on Deramiocel. The year-to-year difference was 1.56 points, with a 95% confidence interval of 0.47 to 2.64.

When is the FDA target action date for Capricor’s Deramiocel application?

The FDA target action date for Deramiocel is November 22, 2026. The extension data were included in a major amendment to the biologics license application, together with sensitivity and robustness analyses supporting the HOPE-3 randomized clinical trial results.

How many patients contributed to Capricor’s HOPE-3 extension results?

Of 106 randomized patients, 98 entered the open-label extension, with 49 in each original treatment group. At Month 24, 42 patients from the original placebo group and 40 from the original Deramiocel group were assessed.

How were natural-history predictions calculated for Capricor’s HOPE-3 extension?

The natural-history model predicted each patient’s decline using baseline score, age and ambulatory status. During its untreated year, the placebo group had an observed decline of 2.7 points versus a predicted decline of 2.9 points at Month 12. These comparisons were descriptive, without matching or statistical testing.