Capricor Therapeutics Reports Intravenous Administration of CAP-1002 Shown to be Effective in Preclinical Models of Duchenne Muscular Dystrophy

LA JOLLA, Calif., Oct. 6, 2017 /PRNewswire/ -- Capricor Therapeutics, Inc. (NASDAQ: CAPR), in a poster presentation at the Alliance for Regenerative Medicine's Cell and Gene Meeting on the Mesa, provided data which support the peripheral intravenous (IV) administration method the company plans to use in HOPE-2, its planned next clinical trial of its lead investigational product, CAP-1002, for the treatment of Duchenne muscular dystrophy (DMD).

CAP-1002 consists of allogeneic cardiosphere-derived cells (CDCs), whose mechanism of action is immunomodulatory and anti-fibrotic. CDCs have been shown to generate new muscle cells in preclinical models.

In their poster presentation, Capricor and Cedars-Sinai Medical Center researchers reported that the IV administration of CDCs increases both exercise capacity and diaphragm function in a preclinical model of DMD, a rare and fatal genetic disorder with limited treatment options.

The results also show that CDCs predominantly distribute to the lungs after injection and are cleared within three weeks. Dosing of more than 2.5 times the human equivalent dose proposed for the upcoming HOPE-2 trial was observed to be safe in these studies.

"These findings lay the groundwork for advancing our clinical development program of cellular therapies for the treatment of DMD," said Linda Marbán, Ph.D., Capricor's president and chief executive officer. "Capricor recently announced positive 6-month results from HOPE-1, a clinical trial in which a single dose of CAP-1002 was delivered directly into the coronary circulation of teens and young men in advanced stages of DMD. The HOPE-2 clinical trial will be the first to use IV administration of CDCs in people with DMD."

The HOPE-2 Trial is being planned as a randomized, double-blind, placebo-controlled Phase II clinical trial that will evaluate repeat dosing of IV CAP-1002 in boys and young men with DMD. Subject to regulatory approval, Capricor plans to commence enrollment in HOPE-2 in the first quarter of 2018.

Capricor recently announced that the national principal investigator for the HOPE-2 Trial will be Craig M. McDonald, M.D., a distinguished thought leader in the clinical management of neuromuscular diseases, including muscular dystrophies, and the development of novel outcome measures for DMD clinical trials.

HOPE-2 will build on the findings from HOPE-1, the Phase I/II clinical trial of CAP-1002. On Wednesday, October 4^th, Capricor presented the first six months of follow-up data from this randomized 12-month trial at the 22^nd Annual International Congress of the World Muscle Society. The company reported that teens and young men in the advanced stages of DMD experienced meaningful improvements in cardiac and upper limb function after a single dose of CAP-1002. CAP-1002 was administered by infusion into each of the three main coronary arteries for a total dose of 75 million cells.

DMD is a devastating genetic disorder that causes muscle degeneration and leads to death generally before the age of 30, most commonly from heart failure. DMD occurs in one in every 3,600 live male births across all races, cultures and countries. DMD afflicts approximately 15,000 to 20,000 boys and young men in the U.S.

The HOPE-1 trial was funded in part by the California Institute for Regenerative Medicine.

The poster presented in La Jolla is available at the Events & Presentations section of Capricor's website.

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 alter 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 developing biological therapies for Duchenne muscular dystrophy (DMD) and other rare diseases. Capricor's lead candidate, CAP-1002, is a cell-based candidate currently in clinical development for the treatment of DMD. Capricor is also exploring the potential of CAP-2003, a cell-free, extracellular vesicle-based candidate, to treat a variety of disorders. For more information, visit www.capricor.com.

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 timing of regulatory approvals; 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 June 30, 2017, as filed with the Securities and Exchange Commission on August 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. Capricor's exosomes technology, including CAP-2003, has not yet been approved for clinical investigation.

For more information, please contact:

AJ Bergmann, Vice President of Finance
+1-310-358-3200
abergmann@capricor.com

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SOURCE Capricor Therapeutics, Inc.