Brace yourself. Capricor Therapeutics Inc (NASDAQ:CAPR) is on the move. The drug maker’s stock price spiked nearly 8% today, following the news that the company received U.S. FDA clearance for the Investigational New Drug application (IND) for CAP-1002, its lead investigational therapy, in boys and young men in advanced stages of Duchenne muscular dystrophy.
This randomized, double-blind, placebo-controlled clinical trial will be called the HOPE-2 Trial and is designed to evaluate the safety and efficacy of intravenous, repeat doses of CAP-1002 in boys and young men whose ability to walk has been seriously impaired by the loss of muscle function that occurs as Duchenne muscular dystrophy progresses. The primary efficacy endpoint will be the relative change in the mid-level dimension of the Performance of the Upper Limb test from baseline to Month 12. The HOPE-2 Trial is expected to enroll approximately 84 patients and be conducted at 10-12 U.S. sites.
Capricor believes that if the primary endpoint is reached, the HOPE-2 Trial could serve as a registration trial, meaning that its results could support the submission of a Biologics License Application (BLA) to obtain marketing approval of CAP-1002. Capricor expects to initiate the HOPE-2 Trial in the first quarter of 2018.
Capricor plans to apply for the Regenerative Medicine Advanced Therapy (RMAT) Designation for CAP-1002 based on updated guidance recently issued by the FDA. If granted, the RMAT Designation would be expected to facilitate CAP-1002’s path to potential registration.
“The FDA’s clearance of this IND upon its initial submission is a significant step forward in our development of CAP-1002,” said Linda Marbán, Ph.D., president and chief executive officer. “While there are many clinical initiatives in Duchenne muscular dystrophy, this is one of the very few to focus on non-ambulant patients. These boys and young men are looking to maintain what function they have in their arms and hands and, based on our previous study, we think CAP-1002 may be able to do exactly that.”
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 210% from current levels. According to TipRanks.com, Pantginis has a yearly average loss of 19.1%, a 29% success rate, and is ranked #4684 out of 4735 analysts.
Capricor 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.