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New Published Study Reports Cardiosphere-Derived Cells Improved Skeletal and Cardiac Muscle Function in Mouse Model of Duchenne Muscular Dystrophy

February 23, 2018

Cedars-Sinai Researchers Say Findings Lay Groundwork for Testing of CAP-1002 in Patients with Duchenne Muscular Dystrophy

LOS ANGELES, Feb. 22, 2018 (GLOBE NEWSWIRE) — Capricor Therapeutics (NASDAQ:CAPR) today announced the online publication in Stem Cell Reports of a new study by researchers at the Smidt Heart Institute at Cedars-Sinai Medical Center, who found that cardiosphere-derived cells (CDCs) improved cardiac muscle function, walking abilities and survival in a mouse model of Duchenne muscular dystrophy. The CDCs used in the study are the research grade version of CAP-1002, Capricor’s cell therapy product.

In the study reported today, the Cedars-Sinai researchers tested the effectiveness of CDCs in a mouse model of Duchenne muscular dystrophy, a progressive and fatal genetic disease. The researchers showed for the first time that the skeletal and cardiac improvements seen in Capricor’s HOPE-Duchenne study could be directly attributed to treatment with CDCs.

“What is most notable about this study is that the researchers further elucidated the mechanism of action of the cells to the exosomes they release,” said Linda Marbán, Ph.D., Capricor president and chief executive officer.

Exosomes are the nano-sized vesicles secreted by the CDCs that serve as cellular messengers carrying out the instructions of the CDCs to treat inflammation and fibrosis. The findings reported today also provide further evidence of the potential of CAP-1002 to stimulate tissue repair and regeneration with a powerful one-two punch: first reducing inflammation, which then enables new healthy muscle to form, as was shown in the mouse model of Duchenne muscular dystrophy.

“Capricor identified the importance of the CDCs to treat Duchenne muscular dystrophy several years ago, and we are delighted to see this extensive body of stellar scientific work published so that others can be aware of its implications as a potential strategy to treat Duchenne muscular dystrophy,” said Dr. Marbán. “This study’s publication helps explain why we are hopeful that our research will continue to show the cells work in people as robustly as they appear to work in mice.”

The HOPE-Duchenne trial found that a single intracoronary dose of CAP-1002 produced significant and sustained improvement in cardiac and skeletal muscle functions in boys and young men in advanced stages of Duchenne muscular dystrophy.

The upcoming HOPE-2 clinical trial will evaluate the safety and efficacy of four intravenous doses of CAP-1002 delivered every three months over a one-year period. It will enroll approximately 84 boys and young men whose ability to walk has been seriously impaired by the loss of muscle function that occurs as Duchenne muscular dystrophy progresses.

“We look forward to initiating HOPE-2 so we can see in a double-blind, randomized, placebo-controlled trial whether the effects seen in the mice and in the first human study are validated,” said Dr. Marbán.

In their published study, the Cedars-Sinai researchers reported that they “had not aspired to restore skeletal muscle function, but merely to offset the pathophysiological consequences of dystrophin deletion in the heart. We now report that CDCs and their secreted exosomes potently improve not only cardiac but also skeletal muscle structure and function…An unanticipated, minor restoration of dystrophin expression was also observed, but this cannot explain all of the observed benefits.”

“CAP-1002 can potentially be very important in the toolbox of therapies which are evolving to treat Duchenne muscular dystrophy,” Dr. Marbán said. “While gene therapy offers tremendous potential in restoring dystrophin expression and sustaining muscle function, there will still be significant inflammation and fibrosis which can offset the restorative effects. We believe that CAP-1002 can work synergistically with the emerging disease modifying therapies to control those pathological aspects of Duchenne muscular dystrophy.”

The researchers also concluded that their findings “further motivate the testing of CDCs in Duchenne patients, while identifying exosomes as next-generation therapeutic candidates.”

The CDCs used in the study published in Stem Cell Reports were manufactured at Cedars-Sinai. The study was funded in part by a grant from Coalition Duchenne.

For more information about the HOPE-2 Trial, please visit:

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 unique population of cells that contains cardiac progenitor cells. CAP-1002 has been shown to exert potent immunomodulatory activity and stimulate 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. The HOPE-Duchenne trial was funded in part by the California Institute for Regenerative Medicine. For more information, visit

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, 2016 as filed with the Securities and Exchange Commission on March 16, 2017, 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, 2017, as filed with the Securities and Exchange Commission on November 14, 2017. 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.

For more information, please contact:

AJ Bergmann, Chief Financial Officer