Capricor’s lead candidate, CAP-1002, is a cell therapy that is currently in clinical development for the treatment of Duchenne muscular dystrophy (DMD). CAP-1002 is an allogeneic product, meaning that it is manufactured from donor heart tissue and then stored until needed for use. 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.
CAP-1002 has been granted orphan drug designation by the FDA for the treatment of DMD.
We have designed a clinical trial to evaluate the potential ability of CAP-1002 to benefit skeletal muscle function in boys and young men with DMD. We expect to initiate a randomized, double-blind, placebo-controlled HOPE-2 clinical trial of intravenous (IV) CAP-1002 in the first quarter of 2018. The primary efficacy endpoint of the HOPE-2 Trial will be based on the PUL. In order to evaluate CAP-1002’s potential for sustained clinical improvements over time, the HOPE-2 Trial will test the safety and efficacy of repeat IV doses given at three-month intervals.
View a list of publications relating to CAP-1002.