Capricor Therapeutics Announces Positive 24-Month Results from Ongoing HOPE-2 Open Label Extension Study of CAP-1002 in Duchenne Muscular Dystrophy

-Improvements in Left Ventricular Ejection Fraction (LVEF) for the Majority of Patients Suggest Preservation of Cardiac Function-

-Results in Performance of the Upper Limb PUL v2.0 Continue to Show Long-Term Benefit in Skeletal Muscle Function (p=0.02)-

-Safety and Efficacy Results Continue to Suggest Potential Disease Attenuation in Duchenne Muscular Dystrophy (DMD)-

-Results to be Presented at the PPMD Annual Conference Being Held Today-

SAN DIEGO, June 30, 2023 (GLOBE NEWSWIRE) -- (NASDAQ: CAPR), a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of muscular and other select diseases, today announced positive 24-month safety and efficacy results from its ongoing HOPE-2 open label extension (OLE) study with its lead asset, CAP-1002, for the treatment of Duchenne muscular dystrophy (DMD). Data from the OLE study demonstrated that the majority of patients had an improvement in left ventricular ejection fraction (LVEF), after two years of CAP-1002 treatment, which suggests preservation of cardiac function. Additionally, patients continue to show statistically significant benefit (p=0.021) after two years of treatment in the Performance of the Upper Limb (PUL v2.0) scale when compared to the original rate of decline of the placebo group from HOPE-2 after one year. Furthermore, the OLE study continues to show a favorable safety profile for long-term treatment of CAP-1002. These data will be featured in an oral presentation being webcast today at this year’s .

“The results from this two-year open label study are tremendously impactful for DMD patients showing cardiac and skeletal functional benefits, which underscores the potential long-term benefits of CAP-1002 treatment in DMD,” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “Importantly, the natural history of DMD cardiomyopathy suggests a steady decline in cardiac function as measured by ejection fraction, however, in HOPE-2-OLE, we observed improvements in heart function in six of nine patients. Furthermore, as the HOPE-2-OLE data highlights the disease modifying potential of CAP-1002, we believe it is imperative to start treatment as early as possible to prevent the irreversible loss of muscle. Taken together with the favorable safety/tolerability profile, these data position CAP-1002 as a potential anchor therapy for DMD patients. We thank the patients, their families, caregivers, and the broader Duchenne community for continuing to work with us on this promising therapy.”

The HOPE-2-OLE study previously met its primary endpoint at the one-year timepoint on the PUL v2.0 scale (p=0.02). At the 24-month timepoint, data showed statistically significant differences in the PUL v2.0 in the OLE treatment group when compared to the original rate of decline of the placebo group from HOPE-2 after one-year (p=0.021). LVEF was measured using cardiac magnetic resonance imaging (cMRI) and six of nine patients showed improvements in heart function with CAP-1002 treatment compared to their final assessment at the end of the HOPE-2 study. Over time, there was an increasing correlation with PUL v2.0 and ejection fraction results (24-month OLE results r=0.75, p=0.02).

Dr. Marbán continued, “We are encouraged by the robust and consistent results observed to date and will continue to work closely with the U.S. Food and Drug Administration (FDA) to bring CAP-1002 to patients as quickly as possible. We plan to discuss these results as well as the key features of our ongoing Phase 3 HOPE-3 trial and outline our proposed path towards submission of a potential Biologics License Application (BLA) during our upcoming meeting with the FDA. In parallel, HOPE-3 continues to enroll patients and we remain on track to complete enrollment in the second half of 2023 and report the interim analysis in the fourth quarter of 2023.”

CAP-1002 treatment during the HOPE-2-OLE study continues to yield a consistent safety profile and has been well-tolerated throughout the study. The HOPE-2-OLE study remains ongoing, and participants continue to be monitored for safety and functional performance.

About HOPE-2 Open Label Extension (OLE) Study

HOPE-2 was a randomized, double-blind, placebo-controlled, Phase 2 clinical study of Capricor’s lead investigational therapy, CAP-1002, in boys and young men who have DMD. Study patients were treated via intravenous delivery with either CAP-1002 (150 million cells per infusion) or placebo every 3 months. Data from a total of 20 patients was analyzed (12 placebo and 8 treated) at the 12-month time-point and the results were published in . After the completion of the HOPE-2 study, all patients stopped treatment for approximately 392 days (mean, range [239, 567]), which is referred to as the gap phase. Then all eligible patients who wished to remain on treatment re-entered the OLE study where they received CAP-1002 (150 million cells per infusion) every three months over the course of 24 months. Patients continued through the gap phase (off treatment for both groups) and the OLE phase (on treatment for both groups).

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a devastating genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles. Patients suffering from DMD typically lose their ability to walk in their teenage years and generally die of cardiac or respiratory complications by age 30. It occurs in approximately one in every 3,600 live male births across all races, cultures and countries. DMD afflicts approximately 200,000 boys and young men around the world. Treatment options are limited and there is no cure.

About CAP-1002

CAP-1002 consists of allogeneic cardiosphere-derived cells (CDCs), a population of stromal cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory, antifibrotic and regenerative actions in dystrophinopathy and heart failure. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile so that they adopt a healing, rather than a pro-inflammatory, phenotype. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to over 200 human subjects across several clinical trials.

About Capricor Therapeutics

Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of muscular and other select diseases. Capricor’s lead candidate, CAP-1002, is an allogeneic cardiac-derived cell therapy that is currently in late-stage clinical development for treating Duchenne muscular dystrophy. Further, Capricor has entered into a partnership for the exclusive commercialization and distribution of CAP-1002 for DMD in the United States and Japan with Nippon Shinyaku Co., Ltd. (U.S. subsidiary: , Inc.), subject to regulatory approval. Capricor is also developing its exosome technology as a next-generation therapeutic platform. Capricor’s focus is on developing exosomes capable of delivering nucleic acids as well as proteins to treat or prevent a variety of diseases. For more information, visit , and follow Capricor on ,  and .

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; 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; scope, duration, validity and enforceability of intellectual property rights; future royalty streams and revenue 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, 2022, as filed with the Securities and Exchange Commission on March 17, 2023 and in our Quarterly Report on Form 10-Q for the quarter ended March 31, 2023, as filed with the Securities and Exchange Commission on May 12, 2023. 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.

CAP-1002 is an Investigational New Drug and is not approved for any indications. None of Capricor’s exosome-based candidates have been approved for clinical investigation.

For more information, please contact:

Capricor Media Contact:

Raquel Cona

KCSA Strategic Communications



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LifeSci Advisors, LLC



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Capricor Company Contact:

AJ Bergmann, Chief Financial Officer



858.727.1755