Capricor announces new pre-clinical data on CAP-1002
Capricor Therapeutics announced that researchers found that repeat dosing of the company's proprietary cellular therapy yields an increase in exercise performance in a disease model of Duchenne muscular dystrophy, the mdx mouse. The company has previously presented data showing that single doses lead to significant improvement in treadmill run times. In an abstract presentation at the 11th Annual Neuromuscular Translational Research Conference in Cambridge, England, researchers reported that while the therapy, CAP-1002, is recognized by the immune system, its low immunogenic profile and immunomodulatory capabilities allowed it to be administered multiple times without significant safety issues. They also reported that repeat dosing was shown to sustain the effect of the therapeutic. A previous clinical trial, the HOPE-Duchenne trial, found a single intracoronary dose of CAP-1002 was generally safe, well tolerated and demonstrated significant and sustained signals of improvement in cardiac and skeletal muscle function in boys and young men in advanced stages of Duchenne muscular dystrophy. The HOPE-2 clinical trial will test the safety and efficacy of repeat doses of CAP-1002 in boys and young men with Duchenne muscular dystrophy, a devastating and fatal genetic disorder with limited treatment options and no cure. Up to 84 boys and young men with Duchenne muscular dystrophy will be enrolled in the Phase 2, randomized, double-blind, placebo-controlled trial. HOPE-2 will test repeat doses of CAP-1002 in participants with advanced stages of Duchenne muscular dystrophy. Capricor has been granted the RMAT and orphan disease designations by the U.S. Food and Drug Administration. These designations will enable the company to work closely with the FDA in finalizing the regulatory approval pathway for CAP-1002 and to receive expedited FDA reviews.