Capricor Therapeutics Announces Positive 18-Month Results from Ongoing HOPE-2 Open Label Extension Study of CAP-1002 in Duchenne Muscular Dystrophy Patients

-Statistically Significant Results in Performance of the Upper Limb PUL 2.0 (p=0.02) Extended to 18 Months, Demonstrating Long-Term Benefit in Skeletal Muscle Function-

-Results Continue to Suggest Potential Disease Modification in Duchenne Muscular Dystrophy (DMD)-

-Capricor to Host Webinar in Conjunction with Parent Project Muscular Dystrophy (PPMD) Today at 1:00 p.m. ET-

SAN DIEGO, Jan. 25, 2023 (GLOBE NEWSWIRE) -- Capricor Therapeutics (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 18-month results from its ongoing HOPE-2 open label extension (OLE) study in patients with later-stage Duchenne muscular dystrophy (DMD). Data from the OLE study continues to show evidence for disease modification with statistically significant differences in the Performance of the Upper Limb (PUL version 2.0) scale in the CAP-1002 original treatment group when compared to the original placebo group from HOPE-2 (p=0.02). In addition, disease progression is attenuated equally in both groups once patients begin treatment in the OLE.

“DMD is a progressive disease associated with loss of function over time and there is a critical need for treatment options to slow the rate of decline and preserve upper limb function to enable key everyday activities,” said Dr. Craig McDonald, national Principal Investigator and University of California, Davis, Professor and Department of Physical Medicine and Rehabilitation Chair. “We are encouraged by the improvements in upper limb function in the treatment group at 18-months and will continue to treat and follow these patients to further evaluate the disease modifying potential of CAP-1002. Taken together, these data build on the impressive results from HOPE-2 and suggest that patients accumulate benefit over time with steady preservation of skeletal muscle functions, which underscore the potential long-term benefit of CAP-1002.”

The study met its primary endpoint at the one-year timepoint and continues to show statistically significant improvements (p=0.02) on the Performance of the Upper Limb scale for patients treated with CAP-1002 at 18-months. In the study, CAP-1002 was made available to the original 20 patients enrolled in the HOPE-2 study. Of those, 13 entered and 12 completed 18-months of study follow-up. The breakdown of patients included seven from the original placebo group and six from the original CAP-1002 treatment group. All patients were off CAP-1002 or placebo for a mean of approximately one-year before resumption or initiation of therapy under the HOPE-2 OLE program. As in HOPE-2, CAP-1002 was administered quarterly and current results are from the 18-month analysis.

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

Linda Marbán, Ph.D., CEO of Capricor, commented, “In the OLE phase of the HOPE-2 study, we observed statistically significant slowing of the progression of disease across patients treated with CAP-1002. We are extremely pleased by the robust and consistent results observed to date, which, together with the favorable safety/tolerability profile, suggest that CAP-1002 holds promise as a potential anchor therapy for DMD patients. Further, the long-term efficacy and potential disease modification effect will augment our clinical package as we continue on our regulatory pathway towards potential approval of CAP-1002 for treatment of patients with DMD. We thank the patients, their families, caregivers, and the broader Duchenne community for working with us on this promising therapy.”

Capricor is currently conducting a pivotal Phase 3 trial, HOPE-3, designed as a randomized, double-blind, placebo-controlled study with approximately 70 patients and enrollment criteria similar to HOPE-2. The Phase 3 study is currently enrolling subjects (NCT05126758). Furthermore, in 2022, Capricor entered into a partnership for the exclusive commercialization and distribution of CAP-1002 for DMD in the United States with Nippon Shinyaku Co., Ltd. (U.S. subsidiary: NS Pharma, Inc.), subject to regulatory approval.

Webinar Details

Capricor will host a webinar in conjunction with PPMD today, January 25, at 1:00 p.m. ET to discuss the data findings. To listen to the webinar, please click the following link:

For additional details, please visit A replay of the webinar will be made available on PPMD’s website.

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 and are non-ambulant, the later stage of the disease process. The study was conducted at nine sites across the United States. 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. The study met its primary efficacy endpoint, PUL 1.2 mid-level dimension, showing a mean 12-month change from baseline in mid-level PUL 1.2, favoring CAP-1002 over placebo (2.6 point difference; (p=0.014). Cardiac MRI assessments showed improvements in heart function and structure with CAP-1002 treatment. Left ventricular ejection fraction (LVEF), a global measure of cardiac pump function, decreased in the placebo group over time, but improved in the CAP-1002 group, showing a 107% slowing of progression of cardiac disease (p=0.002).

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 18 months. Patients continued through the gap phase (off treatment for both groups) and the OLE Phase (on treatment for both groups).

Patients in the study were evaluated using the Performance of the Upper Limb (PUL 2.0), a validated tool specifically designed for assessing high (shoulder), mid (elbow) and distal (wrist and hand) function, with a conceptual framework reflecting the progression of weakness in upper limb function.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy 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 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 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. Capricor is also developing its exosome technology as a next-generation therapeutic platform. Capricor’s current focus is on developing exosomes capable of delivering nucleic acids, including mRNA, as well as proteins to treat or prevent a variety of diseases. 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; 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, 2021, as filed with the Securities and Exchange Commission on March 11, 2022 and in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2022 as filed with the Securities and Exchange Commission on November 10, 2022. 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

Capricor Investor Contact:
Joyce Allaire
LifeSci Advisors, LLC

Capricor Company Contact:
AJ Bergmann, Chief Financial Officer