Focus will be on Milestones for Development and Commercialization CAP-1002 Therapy for Duchenne Muscular Dystrophy
LOS ANGELES, Nov. 12, 2018 (GLOBE NEWSWIRE) — Capricor Therapeutics (NASDAQ: CAPR) announced today that it will meet with the U.S. Food and Drug Administration (FDA) in December to discuss clinical trial design, surrogate or intermediate endpoints and manufacturing processes for CAP-1002, Capricor’s novel cell therapy. Currently, patients are being enrolled in the HOPE-2 clinical trial, which is investigating CAP-1002 as a therapy for the treatment of Duchenne muscular dystrophy.
The in-person meeting is part of the expedited review process afforded to Capricor for its CAP-1002 product candidate after being granted the Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA in February 2018. The FDA grants the RMAT designation to regenerative medicine therapies intended to treat a serious condition and for which preliminary clinical evidence indicates a potential to address unmet medical needs for that condition.
“We are very pleased to have this opportunity to discuss CAP-1002 and the HOPE-2 clinical trial with the FDA, and we appreciate the key opinion leaders in the field of Duchenne muscular dystrophy who will be joining us for the meeting,” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “HOPE-2 is a randomized, placebo-controlled clinical trial testing repeat intravenous doses of CAP-1002. The study is currently enrolling up to 84 boys and young men at approximately 10-15 centers across the U.S. The participants are in the advanced stages of Duchenne muscular dystrophy and are typically not eligible for many of the other clinical trials being conducted for Duchenne. We believe that CAP-1002 could fill a currently unmet medical need for these patients.”
Pre-clinical and clinical studies have shown that CAP-1002 is generally safe, well-tolerated and demonstrated significant and sustained signals of improvement in cardiac and skeletal muscle function in patients with Duchenne muscular dystrophy, a fatal genetic disease with limited treatment options.
The RMAT designation allows eligible therapies similar access to expedited development and review of a marketing application that are available to drugs that receive breakthrough therapy designation – including increased opportunities to meet with the FDA, early interactions with the FDA to discuss any potential surrogate or intermediate endpoints and the potential to support accelerated approval of drugs receiving the RMAT designation.
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 alters 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 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. For more information, visit www.capricor.com.