Capricor Therapeutics Announces Positive 3-Year Efficacy Results from HOPE-2 Open Label Extension Study of CAP-1002 in Duchenne Muscular Dystrophy

--Results in Performance of the Upper Limb (PUL 2.0) Continue to Show Benefits in Skeletal Muscle Function After 3 Years of CAP-1002 Treatment (p< 0.001)--

--Stabilization in Left Ventricular Ejection Fraction (LVEF) Suggests Preservation of Cardiac Function--

--Results Recently Shared with FDA at Type-B Meeting Held in May 2024--

SAN DIEGO, June 04, 2024 (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 3-year results from the ongoing HOPE-2 open-label extension (OLE) study with CAP-1002 for the treatment of Duchenne muscular dystrophy (DMD). Patients treated with CAP-1002 continue to show positive benefits after 3 years of treatment in both the PUL 2.0 and LVEF measures when compared to an external comparator dataset of similar DMD patients (dataset provided by Cincinnati Children’s Hospital Medical Center (CCHMC)). Further, CAP-1002 treatment during the HOPE-2-OLE study continues to have a consistently well-tolerated safety profile throughout the study. The Company plans to present these results in more detail at the upcoming Parent Project Muscular Dystrophy (PPMD) 30th Annual Conference being held June 27-29, 2024.

3-Year HOPE-2 OLE Topline Study Results

Primary Endpoint

3-Year Timepoint
Change from Baseline*
Delta Change

Skeletal-Muscle (Upper Limb Function)
Full Performance of Upper Limb (PUL 2.0)

CAP-1002 (n=12) External Comparator (n=32)
-4.1 points -7.8 points +3.7 points
p< 0.001

*Baseline is referred to as start of HOPE-2 OLE study, changes in means are shown

“I am extremely encouraged by these positive results from our HOPE-2 OLE study which continue to support the long-term impact of CAP-1002 for the treatment of DMD,” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “Importantly, the external comparator of the disease shows a steady state of decline in both skeletal and cardiac function; however over the course of 3-years, we observed a statistically significant reduction in PUL decline as well as stabilization in LVEF in CAP-1002 treated patients suggesting sustained disease attenuation. Furthermore, at our recent Type-B meeting with the U.S. Food and Drug Administration (FDA), we shared these results with the FDA to support what we expect to see in the HOPE-3 pivotal study as we continue to work on strategies for the most expeditious pathway to potential BLA approval and commercialization. The details of our meeting will be shared later this month once the meeting minutes are final. Looking ahead, we remain on track to announce topline results from our Phase 3 HOPE-3 pivotal trial in the fourth quarter of 2024. We continue to thank the patients, families and the broader Duchenne community for continuing to work with us on this promising therapy.”

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 The Lancet. 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 entered the HOPE-2-OLE study where they receive CAP-1002 (150 million cells per infusion) every three months. The HOPE-2-OLE study previously met its primary endpoint at the one-year timepoint on the PUL 2.0 (p=0.02). The HOPE-2-OLE study remains ongoing and into its fourth year and participants continue to be monitored for safety and functional performance.

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 with mortality at a median age of approximately 30 years. It is estimated that DMD occurs in approximately one in every 3,500 male births and that the patient population is estimated to be approximately 15,000-20,000 in the United States. DMD pathophysiology is driven by the impaired production of functional dystrophin, which normally functions as a structural protein in muscle. The reduction of functional dystrophin in muscle cells leads to significant cell damage and ultimately causes muscle cell death and fibrotic replacement. Treatment options are limited and there is no cure.

About Capricor Therapeutics

Capricor Therapeutics, Inc. (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, CAP-1002, an allogeneic cardiac-derived cell therapy. Extensive preclinical and clinical studies have shown CAP-1002 to demonstrate immunomodulatory, antifibrotic, and regenerative actions specifically tailored for dystrophinopathies and heart disease. CAP-1002 is currently advancing through Phase 3 clinical development for the treatment of Duchenne muscular dystrophy (DMD). Capricor is also harnessing the power of our exosome technology, using our 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, and follow Capricor on FacebookInstagram 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 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 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, 2023, as filed with the Securities and Exchange Commission on March 11, 2024, and in our Quarterly Report on Form 10-Q for the quarter ended March 31, 2024, as filed with the Securities and Exchange Commission on May 14, 2024. 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 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: NS Pharma, Inc.), subject to regulatory approval. 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 Company Contact:
AJ Bergmann, Chief Financial Officer

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Source: Capricor Therapeutics