LOS ANGELESApril 4, 2016 /PRNewswire/ -- Capricor Therapeutics, Inc., a biotechnology company focused on the discovery, development and commercialization of first-in-class therapeutics, today announced that the independent Data Safety Monitoring Board (DSMB) of the Phase I/II HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne) clinical trial has completed its pre-specified review of the first patient cohort. Based on the DSMB's recommendation, enrollment in the trial will continue as planned. The HOPE-Duchenne trial is evaluating CAP-1002, Capricor's lead product candidate, in boys with Duchenne muscular dystrophy (DMD)-associated cardiomyopathy.
"The DSMB's recommendation to proceed with enrollment in the HOPE-Duchenne clinical trial is encouraging, and confirms our initial understanding of the safety of CAP-1002 in the DMD patient population, which is an important step toward establishing a potential new treatment to help these boys. In addition, the DSMB's recommendation reassures us with respect to the safety of the catheter procedure for the administration of CAP-1002 for these potential trial participants," said Linda Marbán, Ph.D., president and chief executive officer of Capricor Therapeutics. "With heart disease being one of the primary drivers of death and disability in older DMD patients, each step we take toward better managing DMD is an important one. We look forward to sharing additional milestones from the HOPE-Duchenne trial in the coming months, including its top-line results, which are expected in the first quarter of 2017."
CAP-1002 is an allogeneic, intracoronary-delivered, cardiosphere-derived cell treatment. This investigational "off-the-shelf" cell therapy is derived from donor heart tissue and is infused directly into the coronary arteries by catheter. CAP-1002 has been granted orphan drug designation by the U.S. Food and Drug Administration for the treatment of DMD.
In February 2016, Capricor announced that the first patient had been treated in the HOPE-Duchenne clinical trial. This multi-center, randomized, open-label, usual care-controlled study is designed to evaluate the safety and preliminary efficacy of CAP-1002 in 24 male patients with DMD who have significant cardiac involvement. The first patient cohort consisted of six patients.
About Duchenne Muscular Dystrophy (DMD)  DMD is a rare, genetic disorder of muscle and afflicts approximately 20,000 boys and young men in the U.S. It results from an inherited abnormality in the dystrophin complex, leading to progressive damage to skeletal and cardiac muscle. No treatment has been approved for the treatment of DMD, and nearly all of the candidates in development are directed toward improving skeletal muscle. However, heart disease is the leading cause of death in DMD patients despite optimal medical therapy, and virtually all DMD patients 15 years of age and older develop heart failure. Heart transplantation is not generally an option for those with DMD.