Neurology Publishes Study Reporting Significant Improvements in Duchenne Muscular Dystrophy Patients Treated with Capricor’s Novel Cell Therapy

Sustained Cardiac and Skeletal Muscle Function Improvements Shown after a Single Dose of CAP-1002

Beverly Hills, California, UNITED STATES

LOS ANGELES, Jan. 24, 2019 (GLOBE NEWSWIRE) -- Neurology®, an international peer-reviewed journal, on Wednesday published the results of the HOPE-Duchenne clinical trial that evaluated the safety and efficacy of a single intracoronary dose of Capricor Therapeutics’ (NASDAQ: CAPR) novel cell therapy candidate, CAP-1002, in boys and young men in advanced stages of Duchenne muscular dystrophy.

The Phase I/II, randomized, controlled, open-label trial found that CAP-1002 demonstrated improvement in cardiac muscle function and reduction in cardiac scarring that were statistically-significant and sustained improvement of skeletal muscle functions in patients with Duchenne muscular dystrophy, a fatal genetic disease with limited treatment options. The HOPE-Duchenne trial also found no serious safety issues, according to the study published in the January 23, 2019, online issue of Neurology, the medical journal of the American Academy of Neurology.

“CAP-1002 is the only therapy so far that has shown reduction in myocardium scar and improvement of localized cardiac function in late stage Duchenne associated cardiomyopathy. Interestingly, it also showed improvement in upper limb strength,” said Michael Taylor, M.D., Ph.D., the study’s corresponding author and Director of Cardiac MR research at Children's Healthcare of Atlanta. “These findings suggest that CAP-1002 could be an option for older patients who have scarring in the heart as a result of the underlying disease. As many of these patients are in later stages of the disease, the improvement in upper limb strength is important in order to maintain independence.”

The HOPE-Duchenne clinical trial enrolled 25 patients and was conducted at three centers in the U.S. All participants had significant cardiac scarring and approximately two-thirds were wheelchair-dependent at the time they began the trial. During the 12-month course of the trial, all patients received the standard-of-care for Duchenne muscular dystrophy, including oral steroids, and 13 also received one dose of intracoronary CAP-1002 (75 million cells) upon randomization.

Using the Performance of the Upper Limb (PUL) test, which is a validated functional assessment of upper limb activities of daily living (ADL), the researchers found sustained improvement in eight of the nine CAP-1002 treated patients in the mid-distal PUL, which was manifested by improvements in the patients’ abilities to use their arms and hands. The control patients had no improvement in the mid-distal PUL tests.

Researchers also reported that magnetic resonance imaging (MRI) found that the participants who received CAP-1002 had significant myocardial scar reduction and improvement in heart inferior wall systolic thickening compared to the usual care group. Progressive cardiac scarring eventually impairs the heart’s pumping ability and is the leading cause of death in Duchenne muscular dystrophy. Usual care patients had no relevant changes in cardiac scar or function over the course of the trial, while treated patients had less scar in their hearts at both the 6 month and 12 month follow-up after treatment, which suggests a beneficial effect of CAP-1002.

“Capricor’s pre-clinical studies and the HOPE-Duchenne trial provide evidence that CAP-1002 can help preserve skeletal and cardiac muscle function in patients in the advanced stages of Duchenne muscular dystrophy,” said Linda Marbán, Ph.D., Capricor CEO. “Most importantly, we have shown that there is an opportunity for stabilization or even improvement of disease in patients with later stage Duchenne muscular dystrophy, which represents a large proportion of those living with the disease.”

Capricor is investigating CAP-1002 in the HOPE-2 clinical trial, a Phase II, randomized, placebo-controlled clinical trial testing repeat intravenous doses of CAP-1002. Earlier this week, Capricor announced positive outcomes from its Comprehensive Multidisciplinary meeting with the FDA regarding that trial.

The HOPE-Duchenne trial was funded by Capricor and in part by the California Institute for Regenerative Medicine (CIRM).

A more detailed presentation on the data is available on Capricor’s website at

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is a devastating genetic disorder that causes muscle degeneration and leads to death, generally before the age of 30, most commonly from heart failure. It occurs in one in every 3,600 live male births across all races, cultures and countries. Duchenne muscular dystrophy 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, or CDCs, a type of progenitor cell that has been shown in pre-clinical and clinical studies to exert potent immuno-modulatory activity, and is being investigated for its potential to modify the immune system’s activity to encourage cellular regeneration. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to approximately 140 human subjects across several clinical trials.

About Capricor Therapeutics

Capricor Therapeutics, Inc. (NASDAQ:CAPR) is a clinical-stage biotechnology company focused on the discovery, development and commercialization of first-in-class biological therapeutics for the treatment of rare disorders. Capricor’s lead candidate, CAP-1002, is an allogeneic cell therapy that is currently in clinical development for the treatment of Duchenne muscular dystrophy. Capricor has also established itself as one of the leading companies investigating the field of extracellular vesicles and is exploring the potential of CAP-2003, a cell-free, exosome-based candidate, to treat a variety of disorders. For more information, visit

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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; 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, 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, 2017 as filed with the Securities and Exchange Commission on March 22, 2018, in its Registration Statement on Form S-3, as filed with the Securities and Exchange Commission on September 28, 2015, together with the prospectus included therein and prospectus supplements thereto and in its Quarterly Report on Form 10-Q for the quarter ended September 30, 2018, as filed with the Securities and Exchange Commission on November 14, 2018. 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. CAP-2003 has not yet been approved for clinical investigation.


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