Capricor Therapeutics announces FDA clearance of IND for CAP-1002
Capricor Therapeutics announced that the U.S. FDA has cleared its Investigational New Drug application to conduct a new clinical trial of CAP-1002, its lead investigational therapy, in boys and young men in advanced stages of Duchenne muscular dystrophy, a fatal genetic disorder for which there are limited treatment options. This randomized, double-blind, placebo-controlled clinical trial will be called the HOPE-2 Trial and is designed to evaluate the safety and efficacy of intravenous, repeat doses of CAP-1002 in 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. The primary efficacy endpoint will be the relative change in the mid-level dimension of the Performance of the Upper Limb test from baseline to Month 12. The HOPE-2 Trial is expected to enroll approximately 84 patients and be conducted at 10-12 U.S. sites. Capricor believes that if the primary endpoint is reached, the HOPE-2 Trial could serve as a registration trial, meaning that its results could support the submission of a Biologics License Application to obtain marketing approval of CAP-1002. Capricor expects to initiate the HOPE-2 Trial in the first quarter of 2018. Capricor plans to apply for the Regenerative Medicine Advanced Therapy (RMAT) Designation for CAP-1002 based on updated guidance recently issued by the FDA. If granted, the RMAT Designation would be expected to facilitate CAP-1002's path to potential registration.