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

Data Lay Groundwork for HOPE-2 Clinical Trial of Company's Lead Investigational Product

LA JOLLA, CA, USA I October 6, 2017 I 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 4th, Capricor presented the first six months of follow-up data from this randomized 12-month trial at the 22nd 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

SOURCE: Capricor Therapeutics

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