This morning, Capricor Therapeutics, Inc. announced that the HOPE-2 clinical trial has been initiated at UC Davis Medical Center in Sacramento, CA.
The trial will evaluate the efficacy and safety of Capricor’s novel cellular treatment, CAP-1002, in boys and young men with Duchenne muscular dystrophy (DMD), a devastating and life-threatening genetic disorder with few therapeutic options and no approved cure.
CAP-1002 consists primarily of a unique population of cells that are made of cardiac progenitor cells, or allogeneic cardiosphere-derived cells (CDCs). It has demonstrated the potential to exert potent immunomodulatory activity and stimulate cellular regeneration.
Earlier this month, Capricor announced interim results from the Phase 1/2 HOPE (Halt cardiomyopathy progression in Duchenne) trial, in which it was discovered that a single intracoronary dose of CAP-1002 was generally safe, well tolerated and exhibited significant and continued signals of improvement in cardiac and skeletal muscle function in boys and young men in the advanced stages of DMD.
After presenting the results, the company was granted Rare Pediatric Disease designation for the potential treatment option in July, giving it the potential to receive a priority review voucher and fast-track a potential future therapy, should CAP-1002 be approved. Shortly thereafter, the company held a successful meeting with the FDA, in which the regulatory authority approved of plans to continue developing the drug. Additionally, Capricor received orphan drug designation for CAP-1002, giving the company 7-year market exclusivity upon approval.
Up to 84 boys and young men with DMD will be enrolled in the Phase 2, randomized, double-blind, placebo-controlled trial. UC Davis Medical Center is the first site in the United States to initiate enrolling and treating participants, but an estimated 12-15 investigative sites are expected to eventually participate in the trial.
“We are very pleased to begin this important clinical trial of CAP-1002,” said Craig McDonald, M.D., the national principal investigator for the HOPE-2 clinical trial and UC Davis professor and chair of its Department of Physical Medicine and Rehabilitation in a press release. “The HOPE-2 trial will study whether CAP-1002 can maintain or improve cardiac and skeletal muscle function. Because many of the participants are non-ambulatory, the study will focus primarily on the impact on arm mobility.”
Linda Marbán, Ph.D., Capricor president and chief executive officer stressed the importance of the continued development of CAP-1002 as a potential therapeutic option for the devastating disease: “While gene and other therapies have the potential to restore dystrophin expression and sustain muscle function, there will still be significant inflammation and fibrosis which can offset the restorative effects,” she said. “CAP-1002 may work synergistically with the emerging disease-modifying therapies to control those additional pathological aspects of Duchenne muscular dystrophy because CAP-1002’s primary mechanism of action is immunomodulatory, meaning it can help balance inflammation in this chronic inflammatory disease.”
Participants in the HOPE-2 trial will be randomized to receive either placebo or CAP-1002, delivered intravenously every 3 months for a total of 4 administrations. Participants will be followed for a yearlong period following randomization, and an open label extension (OLE) study is planned should the trial evidence suggest an appropriate risk/benefit profile of CAP-1002 in the indication.