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Capricor Therapeutics Announces First Patient Dosed in Pivotal Phase 3 Study of CAP-1002 for the Treatment of Duchenne Muscular Dystrophy
-Double-Blind, Randomized, Placebo-Controlled HOPE-3 Clinical Trial Designed to Enroll approximately 70 Patients- -HOPE-3 Builds on Positive Data Results from
About this update from Capricor Therapeutics, Inc.
[{"type":"text","content":"-Double-Blind, Randomized, Placebo-Controlled HOPE-3 Clinical Trial Designed to Enroll approximately 70 Patients- -HOPE-3 Builds on Positive Data Results from HOPE-2 Study Recently Published in The Lancet- SAN DIEGO, July 19, 2022 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of muscular and other select diseases, today announced commencement of dosing in HOPE-3, a Phase 3 clinical trial investigating CAP-1002, a cell therapy for treating late-stage Duchenne muscular dystrophy (DMD). HOPE-3 is a randomized, double-blind, placebo-controlled study designed to enroll approximately 70 patients in the United States. Capricor recently announced a partnership with Nippon Shinyaku Co., Ltd. which has commercialization and distribution rights in the U.S. This partnership provides funding for the support of HOPE-3 as well as other potential milestone-based payments to support the clinical development of CAP-1002 in DMD. “We are delighted to begin dosing patients in HOPE-3. The data from our Phase 2 clinical trial suggest that CAP-1002 can slow loss of function by as much as 70% in terms of upper limb skeletal muscle function. Since there are very limited therapeutic options for these patients and CAP-1002 has been shown to be safe and effective, we are pleased to begin this pivotal trial with the goal of achieving regulatory approval as quickly as possible,” said Linda Marbán, Ph.D., CEO of Capricor. “Beginning this clinical trial is a significant milestone, not only for Capricor, but most importantly for those boys and young men with DMD.” HOPE-3 participants will be randomized to either CAP-1002 or placebo in a 1:1 ratio. The active arm of participants in the trial will receive 150 million cardiosphere-derived cells (CAP-1002) via intravenous infusion every 3 months for a total of 4 doses. CAP-1002 is comprised of human allogeneic cardiosphere-derived cells, with a differentiated mechanism of action that is immunomodulatory and regenerative. Its broad applicability makes it suitable for patients regardless of genetic mutation. The Phase 3 study’s primary outcome measure will be the Performance of the Upper Limb (PUL) 2.0, a validated tool specifically designed for assessing high (shoulder)...