Capricor Therapeutics (CAPR) Plans Expansion of CAP-1002 DMD Program

November 10, 2016 4:06 PM EST

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Capricor Therapeutics, Inc. (Nassdaq: CAPR) announced that it intends to expand its CAP-1002 clinical development program in Duchenne muscular dystrophy (DMD) to encompass the skeletal muscle aspects of the disease, in addition to the cardiac complications. Based on preclinical data in DMD models that show significant improvement in skeletal, including diaphragmatic, muscle function with CAP-1002 (allogeneic cardiosphere-derived cells), Capricor is designing a clinical trial to evaluate the potential ability of CAP-1002 to benefit skeletal muscle function in boys and young men with DMD. In the planned study, the medication will be given by systemic intra-vascular administration. This trial is expected to begin in 2017 subject to regulatory approval, and is intended to enroll people with DMD irrespective of their mutation or ambulatory status.

Linda Marbán, Ph.D., president and chief executive officer, said, "In a standard preclinical model of DMD, CAP-1002 is able to positively affect peripheral and respiratory muscles and to partially restore muscle cell energetics and function. These encouraging results indicate that CAP-1002 has the potential to not only address the heart disease in Duchenne, but to also treat the progressive failure that occurs in the peripheral and respiratory musculature. Toward this goal, we have developed a formulation of CAP-1002 that can be infused into the systemic circulation. We are hopeful that the muscle function improvements we have observed in preclinical models can be recapitulated in boys and young men with DMD."

Capricor is currently conducting the randomized, controlled Phase I/II HOPE clinical trial of CAP-1002 in DMD-associated cardiomyopathy. In HOPE, 13 patients received a single dose of CAP-1002 via intra-coronary triple-vessel infusion and 12 patients are receiving usual care, with those randomized to the control arm to be eligible to receive CAP-1002 following the completion of their 12-month follow-up. Subject to regulatory review, Capricor is exploring the possibility of re-dosing patients in the active arm who complete the 12-month trial period. It is hoped that patients who initially respond to CAP-1002 will become eligible for repeat dosing.

Barry J. Byrne, M.D., Ph.D., Director of the Powell Gene Therapy Center and Professor of Pediatrics and Molecular Genetics & Microbiology at the University of Florida, and Principal Investigator of the HOPE-Duchenne clinical trial, stated, "The prospect of sustainably preserving heart function in DMD via repeated dosing of CAP-1002 is supported by studies in preclinical models. We look forward to learning more about the treatment potential of CAP-1002 through an amendment to the HOPE trial, as well as through the anticipated trial of CAP-1002 to be given by systemic intra-vascular administration."



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