Capricor Therapeutics, Inc. (NASDAQ: CAPR), a clinical-stage biotechnology company developing biological therapies for cardiac and other serious medical conditions, today announced that it has been awarded a grant of up to $4.2 million from the National Institutes of Health to study CAP-2003 (cardiosphere-derived cell exosomes) for hypoplastic left heart syndrome (HLHS).
“Capricor’s successful record of competing for non-dilutive sources of capital reflects our commitment to scientific excellence as well as the importance of pursuing the therapeutic potential of our technologies. To date, we have been awarded over $30 million from government agencies to support the preclinical and clinical advancement of our cell and exosome candidates,” said Linda Marbán, Ph.D., president and chief executive officer of Capricor. “HLHS is a tragic congenital defect of the heart that often results in heart failure and premature death despite several complex surgeries. With this grant award together with a recently-awarded grant from the Department of Defense to fund the development of a commercial manufacturing process for CAP-2003, we now have two federally-funded applications that will enable us to broaden the scope of our exosome program.”
“Exosomes offer a different approach to regenerative medicine as compared to cell-based strategies, and can be handled more like traditional pharmaceuticals. CAP-2003 is independent of its cells of origin, so we believe this product may offer opportunities for clinical use that may not be ideal targets for our cellular therapeutic product (CAP-1002, allogeneic cardiosphere-derived cells). We are currently pursuing indications in which CAP-2003 can be locally delivered so as to have the best opportunity to act upon the injured cells. Both our ophthalmology program and our new HLHS program were chosen based on the direct delivery that each affords. CAP-2003 has shown compelling data in models of eye injury, and we look forward to its entry into the clinic next year for ocular graft-versus-host disease, a condition involving severe dry eye, pain and light sensitivity that is a frequent complication of bone marrow transplant,” added Dr. Marbán.
“We are excited to be part of this research effort to understand how CDC Exosomes, which are believed to be responsible for the regenerative effect of cardiosphere-derived cells, may be useful in the treatment of a serious congenital heart condition,” said Sunjay Kaushal, M.D., Ph.D., associate professor of surgery at the University of Maryland School of Medicine and director of pediatric cardiac surgery at the University of Maryland Medical Center, in Baltimore. Dr. Kaushal is a collaborator of Capricor and will be the principal investigator of a future clinical trial in HLHS. “We look forward to seeing if CAP-2003 can improve heart function, initially in pre-clinical models, and eventually in HLHS patients.”
CAP-2003 represents exosomes isolated from Capricor’s allogeneic cardiosphere-derived cells, (CAP-1002), which are in clinical development for several cardiac conditions including that associated with Duchenne muscular dystrophy.
About Hypoplastic Left Heart Syndrome
HLHS is a condition that occurs when parts of the left side of the heart such as the mitral valve, left ventricle, aortic valve, and aorta do not develop normally. The causes of HLHS among most babies are unknown, and if left untreated, HLHS is fatal. According to the Centers for Disease Control and Prevention (CDC), an estimated 960 babies are born with HLHS in the U.S. each year.