US-based biotechnology firm Capricor Therapeutics Inc (NASDAQ:CAPR) announced that the American Heart Association (AHA) Scientific Sessions 2017 has accepted for presentation at a late-breaking session the 12-month results from the company’s HOPE-1 clinical trial of its lead investigational product, CAP-1002, in boys and young men in advanced stages of Duchenne muscular dystrophy.
The news prompted a wave of positive reactions from industry watchers, and as a result, on Wednesday shares are up 8%.
Ronald G. Victor, M.D., associate director for clinical research at the Cedars-Sinai Heart Institute and an investigator for the HOPE-1 Trial, will present the results during a session entitled “Innovative Therapies and Novel Applications,” which will begin at 9 a.m. on Nov. 15 at the Anaheim Convention Center.
HOPE-1 is a randomized Phase I/II 12-month clinical trial that evaluated a single dose of CAP-1002, a cell-based therapeutic candidate, in 25 boys and young men in advanced stages of Duchenne muscular dystrophy, a rare, life-threatening genetic disorder.
“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,” said Linda Marbán, Ph.D., Capricor president and CEO. “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 previously reported that meaningful improvements in cardiac and skeletal muscle function were observed at a pre-specified six-month analysis of the HOPE-1 Trial. CAP-1002 is the company’s lead investigational product and consists of allogeneic cardiosphere-derived cells, which improved muscle function and increased new muscle cell generation in preclinical models of Duchenne muscular dystrophy.
Duchenne muscular dystrophy is a devastating genetic disorder that causes muscle degeneration and leads to death, generally before the age of 30, most commonly from heart failure. It occurs in one in every 3,600 live male births across all races, cultures and countries. Duchenne muscular dystrophy afflicts approximately 15,000 to 20,000 boys and young men in the U.S. Treatment options are limited, and there is no cure.
Capricor is planning to host a conference call and webcast at 1:30 p.m. PT on Nov. 15th to review the 12-month HOPE-1 results. Access information will be provided on a press release that Capricor plans to issue earlier that day.
The HOPE-1 trial was funded in part by the California Institute for Regenerative Medicine.
On the ratings front, H.C. Wainwright analyst Joseph Pantginis reiterated a Buy rating on CAPR, with a price target of $6.50, in a report issued on September 15. The current price target implies an upside of 177% from current levels. According to TipRanks.com, Pantginis has a yearly average loss of 18.7%, a 28% success rate, and is ranked #4663 out of 4703 analysts.
Capricor Therapeutics, Inc. engages in the discovery, development and commercialization of first-in-class biological therapies for the treatment of cardiac and other serious medical conditions. Its product candidate consists of CAP-1002, Cenderitide, Exosomes, CAP-1001, CU-NP and CSps.