Capricor Announces Initiation of HOPE-2 Clinical Trial of CAP-1002 for Duchenne Muscular Dystrophy

UC Davis Medical Center First Site in Nation to Enroll and Treat Participants

LOS ANGELES, April 30, 2018 (GLOBE NEWSWIRE) -- Capricor Therapeutics, Inc. (NASDAQ:CAPR) today announced it has initiated the HOPE-2 clinical trial at UC Davis Medical Center. The trial will test the safety and efficacy of Capricor’s novel cellular therapy, CAP-1002, in boys and young men with Duchenne muscular dystrophy, a devastating and fatal genetic disorder with limited treatment options and no cure.

Up to 84 boys and young men with Duchenne muscular dystrophy will be enrolled in HOPE-2, a Phase 2, randomized, double-blind, placebo-controlled trial that will test CAP-1002 in participants with advanced stages of Duchenne muscular dystrophy. The medical center in Sacramento is the first site in the nation to begin enrolling and treating participants. Approximately 12-15 investigative sites are expected to participate in the trial.

“We are very pleased to begin this important clinical trial of CAP-1002,” said Craig McDonald, M.D., the national principal investigator for the HOPE-2 clinical trial and UC Davis professor and chair of its Department of Physical Medicine and Rehabilitation. “The HOPE-2 trial will study whether CAP-1002 can maintain or improve cardiac and skeletal muscle function. Because many of the participants are non-ambulatory, the study will focus primarily on the impact on arm mobility.”

Linda Marbán, Ph.D., Capricor president and chief executive officer, said CAP-1002 is one of the very few clinical initiatives to focus on helping 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 are thrilled to begin enrolling participants in HOPE-2 because we have seen the potential for improvements in muscle function in both pre-clinical studies and in our earlier HOPE-Duchenne trial,” she said. “We have also been granted the RMAT and orphan disease designations by the U.S. Food and Drug Administration (FDA). These designations will enable us to work closely with the FDA in finalizing the regulatory approval pathway for CAP-1002 and to receive expedited FDA reviews. We are hopeful that HOPE-2 may potentially be a registration trial.”

Dr. Marbán said CAP-1002 could be an important tool in the toolbox to treat Duchenne muscular dystrophy.

“While gene and other therapies have the potential to restore dystrophin expression and sustain muscle function, there will still be significant inflammation and fibrosis which can offset the restorative effects,” she said. “CAP-1002 may work synergistically with the emerging disease-modifying therapies to control those additional pathological aspects of Duchenne muscular dystrophy because CAP-1002’s primary mechanism of action is immunomodulatory, meaning it can help balance inflammation in this chronic inflammatory disease.”

Capricor’s previous clinical trial, the HOPE-Duchenne trial, evaluated the safety and efficacy of a single dose of CAP-1002 in boys and young men with heart disease related to Duchenne muscular dystrophy. It found CAP-1002 was generally safe, well tolerated and demonstrated significant and sustained signals of improvement in cardiac and skeletal muscle function.

Participants in the HOPE-2 trial will be randomized to receive either placebo or CAP-1002, delivered intravenously every three months for a total of four administrations. Participants will be followed for a one-year period following randomization. An open label extension is planned if trial evidence suggests an appropriate risk/benefit profile of CAP-1002. For more information, please visit www.HOPE2Trial.com.

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, please visit www.capricor.com.

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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. 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
+1-310-358-3200
abergmann@capricor.com

Released April 30, 2018