Capricor Therapeutics Inc (NASDAQ: CAPR) shares rose Wednesday after the company reported the FDA clearance of Investigational New Drug application for CAP-1002.
The Los Angeles-based company said 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 (BLA) to obtain marketing approval of CAP-1002. Capricor expects to initiate the HOPE-2 Trial in the first quarter of 2018.
CEO Linda Marban said, “While there are many clinical initiatives in Duchenne muscular dystrophy, this is one of the very few to focus on non-ambulant patients. These boys and young men are looking to maintain what function they have in their arms and hands and, based on our previous study, we think CAP-1002 may be able to do exactly that.”
CAPR shares soared 27 cents, or 14.1%, approaching noon Wednesday, to $2.19.
