Results from CAP-1002 Trial for DMD Will Be Released November 15

Results from the HOPE-1 trial of Capricor Therapeutics’ cell-based therapy CAP-1002 to treat boys and men at advanced stages of Duchenne muscular dystrophy (DMD) will be released at a late-breaking session at the American Heart Association Scientific Sessions 2017.

In a session titled “Innovative Therapies and Novel Applications,” Ronald G. Victor, M.D., associate director for clinical research at the Cedars-Sinai Heart Institute in Los Angeles and an investigator for the HOPE-1 Trial, will present the results at the Anaheim Convention Center.

The 12-month results from the HOPE-1 clinical trial of CAP-1002 in boys and men in advanced stages of Duchenne muscular dystrophy (DMD) will be presented at the American Heart Association (AHA) meeting at 9 a.m. PT on November 15, 2017.

The Phase 1/2 12-month HOPE-1 trial (NCT02485938) evaluated a single dose of CAP-1002 administered intravenously directly to blood vessels in the heart in 25 boys and men. Capricor previously reported that a six-month analysis of the HOPE-1 trial had revealed meaningful improvements in heart and upper limb function.

Results from the trial were recently presented at the 22nd Annual International Congress of the World Muscle Society, Oct. 3-7 in St. Malo, France.

The CAP-1002 treatment involves the intravenous administration of cardiosphere-derived cells (CDCs) from donor heart tissue. CDCs are special cardiac cells from donor tissue that can generate more cells. They have a modulating effect on the immune system and slow down fibrosis, as well as promote the regeneration of new cells to replace cells that have died or do not function properly.

Capricor awaits the go-ahead to proceed to the HOPE-2 trial, which will involve multiple infusions of CAP-1002 delivered to ordinary veins in the peripheral blood circulation, such as in the limbs, instead of to the coronary blood vessels (blood vessels in the heart).

“We look forward to sharing these new data at one of the world’s premier cardiovascular conferences and continuing our clinical development of CAP-1002 for the treatment of Duchenne muscular dystrophy,” Linda Marbán, Ph.D., Capricor president and CEO, said in a press release. “Subject to regulatory approval, we plan to initiate patient enrollment into the randomized, double-blind, placebo-controlled HOPE-2 clinical trial of intravenous, repeat-dose CAP-1002 in boys and young men with Duchenne muscular dystrophy in the first quarter of 2018.”

Capricor plans to host a conference call and webcast at 1:30 p.m. PT on Nov. 15^th to report on the 12-month HOPE-1 results. Capricor will issue a press release earlier that day, which will provide access information.