Capricor Therapeutics, Inc., a clinical-stage biotechnology company, announced that the randomized phase I/II HOPE-Duchenne clinical trial has completed its enrollment with 25 subjects.
HOPE (Halt cardiomyOPathy progrEssion in Duchenne) is evaluating CAP-1002, Capricor's lead investigational cardiac cell therapy, in Duchenne muscular dystrophy (DMD)-associated cardiomyopathy. Capricor expects to report top-line six-month data from this trial in the first quarter of 2017.
John Jefferies, M.D., director of the Advanced Heart Failure and Cardiomyopathy Programmes and Professor of Pediatric Cardiology and Adult Cardiovascular Diseases at Cincinnati Children's Hospital, and Principal Investigator of the HOPE-Duchenne clinical trial, stated, "It is accepted that the most common cause of death from DMD relates to the impact of this disease on heart muscle. The enthusiasm for HOPE-Duchenne has been remarkable, and we are excited to see the upcoming results of this innovative study."
"The rate of patient enrollment into HOPE-Duchenne far surpassed our expectations, signifying the need for therapeutic options as well as the desire of the DMD community to address the heart disease that is highly prevalent in this population," said Linda Marbán, Ph.D., president and chief executive officer of Capricor. "We look forward to announcing top-line six-month results from HOPE-Duchenne in the first quarter of next year, in which we will report on the safety as well as the potential efficacy of CAP-1002. In DMD, scar tissue progressively aggregates in the heart, leading to a deterioration of cardiac function for which treatment options are limited. We believe CAP-1002 is the only therapeutic candidate in development for the treatment of DMD that has been clinically shown to reduce scar tissue in the damaged heart."
The HOPE trial enrolled 25 boys with DMD who were at least 12 years of age at the time of screening and who have DMD-associated cardiomyopathy, defined as the presence of scar tissue in at least four left ventricular segments as determined by magnetic resonance imaging (MRI). The 13 subjects randomized to the active treatment arm received CAP-1002 via intracoronary infusion into each of the three main coronary arteries during a single procedure. The 12 subjects randomized to the control arm received usual care and did not receive an infusion.
The primary outcome measures of the trial will consist of a broad assessment of safety and tolerability of CAP-1002. Efficacy will be evaluated according to pre-specified secondary outcome measures, including absolute and relative changes in cardiac scar tissue and cardiac function as measured by MRI, performance on the Six-Minute Walk Test (6MWT) and the Performance of the Upper Limb (PUL), and scoring on the Pediatric Quality of Life Inventory (PedsQL).
The HOPE-Duchenne trial is being conducted at Cincinnati Children's Hospital Medical Center in Cincinnati, Ohio, Cedars-Sinai Heart Institute in Los Angeles, California, and the University of Florida in Gainesville, Florida.
HOPE-Duchenne is being funded in part through the support of the California Institute for Regenerative Medicine.
DMD afflicts approximately 20,000 boys and young men in the US. It is caused by a genetic abnormality in the dystrophin complex, a structural element which plays a critical role in muscle fiber integrity, and leads to chronic skeletal and cardiac muscle damage. Following years of progressive weakness, patients often die in their twenties. Heart disease is currently the most common cause of death among those with DMD. The US Food and Drug Administration (FDA) has not approved any treatment for DMD, and heart transplantation is not typically an option.
Capricor's lead product candidate, CAP-1002, is an investigational allogeneic cardiac cell therapy for the treatment of heart disease. CAP-1002 is manufactured from unrelated donor heart tissue through a proprietary process in which cells are expanded from cardiospheres, self-assembling, multicellular clusters of progenitor heart cells. The product is stored frozen, and stability data support "off the shelf" use for at least three years. The FDA has granted orphan drug designation to CAP-1002 for the treatment of DMD.
Capricor Therapeutics, Inc. is a clinical-stage biotechnology company focused on the discovery, development and commercialization of biological therapies for the treatment of cardiac and other serious medical conditions.