Capricor Therapeutics Inc (NASDAQ:CAPR) in a poster presentation released report which support the peripheral intravenous administration method the firm intends to use in HOPE-2, its intended next clinical study of its lead investigational offering, CAP-1002, for the cure of Duchenne muscular dystrophy.
CAP-1002 comprises of allogeneic cardiosphere-derived cells, whose mechanism of action is anti-fibrotic and immunomodulatory. CDCs have been demonstrated to generate new muscle cells in preclinical prototypes. In their poster presentation, Cedars-Sinai Medical Center experts and Capricor team announced that the IV administration of CDCs enhances diaphragm function and exercise capacity in a preclinical prototype of DMD, a fatal and rare genetic disorder with few treatment alternatives.
The data also demonstrate that CDCs predominantly supply to the lungs post injection and are cleared within 3 weeks. Dosing of over 2.5 times the human equivalent prescription planned for the imminent HOPE-2 study was shown to be safe in these trials.
Linda Marbán, Ph.D., the Chief Executive Officer and President of Capricor, expressed that this data lay the base for developing their clinical advancement plan of cellular therapies targeting DMD. The company recently reported promising 6-month data from HOPE-1, a clinical study in which a single dose of CAP-1002 was offered directly into the coronary circulation of young men and teens in advanced phases of DMD. The HOPE-2 clinical study will be the first to opt for IV administration of CDCs in DMD patients.
The HOPE-2 study is being intended as a double-blind, randomized, placebo-controlled Phase 2 clinical study that will assess repeat dosing of IV CAP-1002 in young men and boys with DMD. Dependent on regulatory approval, Capricor intends to start enrollment in HOPE-2 trial in Q1 2018. Capricor recently reported that the national principal investigator for the respective study will be Craig M. McDonald, M.D., a renowned thought pioneer in the clinical management of neuromuscular ailments.