Capricor Therapeutics reports promising data on CAP-1002
Capricor Therapeutics, in a poster presentation at the Alliance for Regenerative Medicine's Cell and Gene Meeting on the Mesa, provided data which support the peripheral intravenous administration method the company plans to use in HOPE-2, its planned next clinical trial of its lead investigational product, CAP-1002, for the treatment of Duchenne muscular dystrophy. CAP-1002 consists of allogeneic cardiosphere-derived cells, whose mechanism of action is immunomodulatory and anti-fibrotic. CDCs have been shown to generate new muscle cells in preclinical models. In their poster presentation, Capricor and Cedars-Sinai Medical Center researchers reported that the IV administration of CDCs increases both exercise capacity and diaphragm function in a preclinical model of DMD, a rare and fatal genetic disorder with limited treatment options. The results also show that CDCs predominantly distribute to the lungs after injection and are cleared within three weeks. Dosing of more than 2.5 times the human equivalent dose proposed for the upcoming HOPE-2 trial was observed to be safe in these studies. The HOPE-2 Trial is being planned as a randomized, double-blind, placebo-controlled Phase II clinical trial that will evaluate repeat dosing of IV CAP-1002 in boys and young men with DMD. Subject to regulatory approval, Capricor plans to commence enrollment in HOPE-2 in the first quarter of 2018.