It’s a very rewarding trading day for investors in Capricor Therapeutics Inc (NASDAQ:CAPR) with shares up nearly 130%, making the stock Wall Street’s bull of the day. The reason? Capricor announced this morning that the FDA has granted Rare Pediatric Disease Designation to CAP-1002, Capricor’s development candidate for the treatment of Duchenne muscular dystrophy
The Rare Pediatric Disease Designation, as well as the Orphan Drug Designation previously granted to CAP-1002 by the FDA, covers the broad treatment of DMD. Upon receiving market approval for CAP-1002 by the FDA, Capricor would be eligible to receive a Priority Review Voucher.
“The Rare Pediatric Disease Designation adds to our previous Orphan Drug Designation for CAP-1002 for the treatment of Duchenne muscular dystrophy, which together underscore the urgent need for treatment options for this devastating rare disease as well as provide Capricor with certain incentives for their development,” said Linda Marbán, Ph.D., Capricor’s president and chief executive officer. “We believe CAP-1002 has the potential to provide meaningful clinical benefit to patients. We look forward to commencing our next clinical trial in DMD, for which design will be relevant to the overall registration plan for this candidate.”
In April 2017, Capricor announced positive results from its Phase I/II HOPE clinical trial of CAP-1002 in DMD patients with advanced disease. The company recently met with the FDA to discuss potential product registration strategies for this indication, and Capricor plans to commence a randomized, double-blind, placebo-controlled clinical trial of intravenous, repeat-dose CAP-1002 in boys and young men with Duchenne in the second half of this year, subject to regulatory approval.
Capricor expects to report 12-month results from the HOPE Trial in the fourth quarter of 2017.
The HOPE Trial is 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 $2.15, in a report issued on July 10. The current price target implies an upside of 216% from current levels. According to TipRanks.com, Pantginis has a yearly average loss of 18.8%, a 30% success rate, and is ranked #4554 out of 4599 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.