Capricor Therapeutics Announces Positive 4-Year Data from HOPE-2 Open-Label Extension Study of Deramiocel in Duchenne Muscular Dystrophy
- Treatment shows cardiac function preservation after 4 years with only -0.5 points median change from baseline
- Improved efficacy in patients with baseline LVEF >45%, supporting early intervention benefits
- Slower disease progression in year 4 (0.6 points decline) compared to year 1 (1.8 points decline)
- Favorable long-term safety profile maintained throughout the study
- BLA under priority review with multiple regulatory designations secured
- Disease progression continues despite treatment, albeit at a slower rate
- Treatment requires ongoing quarterly intravenous infusions
Insights
Capricor's 4-year data shows Deramiocel preserves cardiac function and slows DMD progression, bolstering its case during BLA priority review.
The 4-year data from Capricor's HOPE-2 Open-Label Extension study represents a significant clinical milestone for Deramiocel in Duchenne Muscular Dystrophy (DMD). The results demonstrate two critical benefits: cardiac function preservation (median change of just -0.5 points from baseline) and continued slowing of skeletal muscle deterioration (measured by PUL v2.0, with a smaller decline in year 4 compared to year 1).
What's particularly notable is the subgroup analysis showing enhanced benefits in patients with baseline LVEF >45%, suggesting early intervention may maximize therapeutic impact. This addresses a critical unmet need, as cardiomyopathy remains a leading cause of mortality in DMD patients.
The regulatory positioning is exceptionally strong. Deramiocel has secured multiple designations that accelerate its path to market: Orphan Drug status (FDA and EMA), Regenerative Medicine Advanced Therapy (RMAT) designation, Advanced Therapy Medicinal Product (ATMP) designation, and Rare Pediatric Disease Designation. The latter potentially qualifies Capricor for a valuable Priority Review Voucher upon approval.
Most importantly, the BLA is currently under priority review, and the company explicitly states their FDA dialogue remains on track without delays. This suggests the commercial pathway is advancing steadily, with the partnership with Nippon Shinyaku/NS Pharma providing established commercialization infrastructure for US and Japanese markets, subject to regulatory approval.
The consistent safety profile over four years strengthens the risk-benefit profile, which is particularly important for therapies requiring repeated administration (Deramiocel is given quarterly via IV infusion). For a devastating disease with limited treatment options and a patient population of approximately 15,000 in the US alone, Deramiocel represents a potential breakthrough addressing both cardiac and skeletal muscle aspects of DMD.
- Four-year data show preservation of cardiac function, including LVEF
- Skeletal muscle disease progression continues to slow with extended treatment (PUL v2.0)
- Deramiocel’s long-term safety profile remains favorable
- Results presented at PPMD’s 2025 Annual Conference
SAN DIEGO, June 20, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for rare diseases, today announced positive four-year safety and efficacy results from its ongoing HOPE-2 Open-Label Extension (OLE) study of Deramiocel, the Company’s lead cell therapy candidate for Duchenne Muscular Dystrophy (DMD). The data will be featured in the session titled “Therapies that Slow Progression” at the Parent Project Muscular Dystrophy (PPMD) 2025 Annual Conference, taking place June 21, 2025, in Las Vegas, Nevada.
After four years of continuous treatment, Deramiocel-treated patients showed a median change of -0.5 points compared to baseline. Further, a subgroup analysis of patients with baseline LVEF >
Additionally, treatment continued to slow skeletal muscle disease progression, as measured by Performance of the Upper Limb (PUL v2.0), with patients experiencing a smaller average decline in the fourth year (0.6 points) compared to the first year (1.8 points). Together, these findings suggest that extended treatment with Deramiocel may help attenuate the progression of DMD over time. Deramiocel continues to maintain a favorable safety profile throughout the study.
“Cardiomyopathy remains one of the leading causes of mortality in Duchenne and addressing this aspect of the disease is critical to improving outcomes,” said Pat Furlong, Founding President and CEO of PPMD. “The long-term data from the HOPE-2 OLE study are encouraging, particularly in demonstrating cardiac stabilization over four years. Deramiocel represents an important therapeutic approach and we support continued progress through the regulatory pathway to ensure that treatments targeting both heart and muscle function are available to our community as quickly as possible.”
“These four-year data reinforce the strength and durability of Deramiocel’s clinical benefit and favorable safety profile across both cardiac and skeletal muscle function,” said Linda Marbán, Ph.D., CEO of Capricor Therapeutics. “With our BLA under priority review and several key regulatory steps now completed, we are executing with focus and urgency as we move toward potential approval. Our continued dialogue with the FDA remains on track with no evidence of any delays. We thank the patients, families, and clinicians who have been instrumental in advancing this program.”
The PPMD Annual Conference is the largest international event focused on advancing research, clinical trials, and standards of care for Duchenne and Becker muscular dystrophies. Capricor’s presentation will be available following the conference in the publications section of the Company’s website.
About HOPE-2 Open Label Extension (OLE) Study
HOPE-2 was a randomized, double-blind, placebo-controlled Phase 2 study of Deramiocel in boys and young men with Duchenne Muscular Dystrophy (DMD). Patients received intravenous infusions of either Deramiocel (150 million cells) or placebo every three months. The results were published in The Lancet. Following study completion, all patients entered a treatment gap phase for approximately 392 days (range: 239–567). Eligible patients then enrolled in the HOPE-2 OLE study, receiving Deramiocel every three months. The OLE study previously met its primary endpoint at one year with statistically significant improvement on the PUL v2.0 (p=0.02). Now in its fifth year, the HOPE-2 OLE study remains ongoing, with participants continuing to be monitored for safety, cardiac function, and upper limb performance.
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, which is 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 potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in dystrophiopathies 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 a pro-inflammatory phenotype. CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials.
Deramiocel has received Orphan Drug Designation for the treatment of Duchenne Muscular Dystrophy (DMD) from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval. For Becker Muscular Dystrophy (BMD), Deramiocel has also received Orphan Drug Designation from the FDA.
About Capricor Therapeutics
Capricor Therapeutics (NASDAQ: CAPR) is a biotechnology company dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases. At the forefront of our innovation is our lead product candidate, Deramiocel, an allogeneic cardiac-derived cell therapy. Extensive preclinical and clinical studies have shown Deramiocel to exert potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in muscular dystrophies such as DMD. Deramiocel is currently in late-stage development for the treatment of Duchenne Muscular Dystrophy. Capricor is also harnessing the power of its exosome technology, using its proprietary StealthX™ platform in preclinical development focused on the areas of vaccinology, targeted delivery of oligonucleotides, proteins and small molecule therapeutics to potentially treat and prevent a diverse array of diseases. At Capricor, we stand committed to pushing the boundaries of possibility and forging a path toward transformative treatments for those in need. For more information, visit capricor.com, and follow Capricor on Facebook, Instagram and Twitter.
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 discovery efforts and clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including the ability to obtain regulatory approvals or otherwise bring products to market; manufacturing capabilities; dates for regulatory meetings; statements about our financial outlook; the potential that required regulatory inspections may be delayed or not be successful which would delay or prevent product approval; the ability to achieve product milestones and to receive milestone payments from commercial partners; plans regarding current and future collaborative activities and the ownership of commercial rights; potential future agreements; scope, duration, validity and enforceability of intellectual property rights; future revenue streams and projections; expectations with respect to the expected use of proceeds from the recently completed offerings and the anticipated effects of the offerings; 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, 2024, as filed with the Securities and Exchange Commission on March 26, 2025, and in our Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, as filed with the Securities and Exchange Commission on May 14, 2025. 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.
Capricor has entered into an agreement for the exclusive commercialization and distribution of Deramiocel for DMD in the United States and Japan with Nippon Shinyaku Co., Ltd. (U.S. subsidiary: NS Pharma, Inc.), subject to regulatory approval. Deramiocel is an Investigational New Drug (IND) and is not yet approved for any indications. Neither BMD nor any of Capricor’s exosome-based candidates have been approved for clinical use.
For more information, please contact:
Capricor Media Contact:
Raquel Cona
KCSA Strategic Communications
rcona@kcsa.com
212.896.1204
Capricor Company Contact:
AJ Bergmann, Chief Financial Officer
abergmann@capricor.com
858.727.1755
FAQ
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