Posted on: October 4th, 2017 | 0 comments
Capricor presented at the 22nd Annual International Congress of the World Muscle Society, and reported that teens and young men in the advanced stages of Duchenne muscular dystrophy experienced meaningful improvements in cardiac and upper limb function after a single dose of Capricor’s lead investigational product, CAP-1002.
The late breaking abstract and results presented at the late breaking poster session describe the first six months of follow-up data from the randomized 12-month Phase I/II HOPE clinical trial of CAP-1002. CAP-1002 is a cell-based therapeutic candidate and consists of allogeneic cardiosphere-derived cells, whose mechanism of action is immunomodulatory and anti-fibrotic, and which have been shown to generate new muscle cells in preclinical models.
“These findings are especially significant because the patients in the HOPE trial were preteens or young men who were in advanced stages of Duchenne muscle disease,” said Ron Victor, M.D., professor of medicine and Burns and Allen Chair in Cardiology Research at Cedars-Sinai Heart Institute and a principal investigator for the HOPE Trial. “Most other studies in DMD have focused on pediatric patients in earlier stages of the disease. To see such positive results in a clinical trial with just one dose of CAP-1002 sets the stage for the next step of evaluating multiple doses of this innovative cellular therapy in a larger trial.”
The HOPE Trial was a randomized, open label trial of 25 males living with Duchenne, of ages 12 to 25 years (mean 17.8). For 17 of them, the disease had progressed to the point of wheelchair dependence for mobility. Cardiomyopathy, or heart disease, secondary to Duchenne was an eligibility criterion and was evidenced by scar in four or more left ventricular segments. All participants had been receiving chronic corticosteroid therapy at entry. Thirteen received a single dose of CAP-1002, while the others received the standard of care, and all participants were to be followed for 12 months. CAP-1002 was administered by infusion into each of the three main coronary arteries for a total dose of 75 million cells.
“[The] exciting findings propel us into our next phase of development of CAP-1002 for the treatment of DMD,” said Linda Marbán, Ph.D., Capricor’s president and chief executive officer. “Subject to regulatory approvals, we expect to initiate the randomized, double-blind, placebo-controlled HOPE-2 clinical trial of intravenous (IV), repeat-dose CAP-1002 in the first quarter of 2018. The primary efficacy endpoint will be based on the PUL, and the HOPE-2 Trial may potentially serve as a registration study. We also look forward to presenting 12-month follow-up results from the HOPE Trial at a major medical conference later this quarter.”