NEWPORT BEACH, Calif.--CureDuchenne Ventures LLC, a company that funds research to find a cure for Duchenne muscular dystrophy that was formed by the nonprofit CureDuchenne, announced today that they are investing $1 million in Capricor Therapeutics, a biotechnology company focused on developing novel therapeutics for the treatment of cardiovascular diseases, to advance promising research to treat heart disease associated with Duchenne muscular dystrophy. Capricor has shown positive pre-clinical data for Cardiosphere-derived cells (CDCs) on Duchenne muscular dystrophy cardiomyopathy. CureDuchenne is investing in Capricor to support the conduct of a first clinical trial (Phase I) in patients with Duchenne.
The planned trial would use CAP-1002 cardiosphere-derived cells delivered to the heart. The goal is to improve global cardiac function, decrease fibrosis, improve exercise capacity, and reverse abnormalities in mitochondrial abundance, structure and function.
"We are delighted to partner with Capricor on this important research to address heart issues with patients living with Duchenne," said Debra Miller, President of CureDuchenne Ventures. "This research has the potential to improve heart function and extend the lives of boys with Duchenne."
Duchenne is a progressive muscle-wasting disease that impacts approximately 1 in 3,500 boys. Boys with Duchenne are usually diagnosed by age 5, in a wheelchair by 12 and most don't survive their mid-20s. Heart failure is the leading cause of death in patients with Duchenne.
"Our research intends to halt cardiomyopathy progression in Duchenne which is a devastating complication that affects all most all those affected by the disease," said Dr. Linda Marbán, Chief Executive Officer of Capricor. "We appreciate working with an innovative and forward-thinking organization like CureDuchenne that continues to fund impactful research to find treatments for Duchenne. We look forward to submitting a clinical development plan to the FDA in order to move this study forward."
"Funding projects like Capricor is part of CureDuchenne Venture's comprehensive research strategy to discover and vet therapies that treat the whole disease, from replacing the missing protein, to drug repositioning to slow the progression of the disease, and addressing cardiac issues," said Miller. "Capricor's therapy can work hand-in-hand with other skeletal muscle potential treatments such as exon skipping."
CureDuchenne and Capricor will be hosting a webinar on January 21 at 3:00 p.m. ET/noon PT to discuss the program. The call in number is (844) 337-8088 and the conference id number is: 65477372
CureDuchenne Ventures LLC was formed by CureDuchenne, a national non-profit that has funded seven research projects that have advanced to human clinical trials. CureDuchenne has leveraged $100 million in pharma and biotech research and development investments. Now three of the projects CureDuchenne supported with funding, Prosensa Holding N.V., Sarepta Therapeutics and PTC Therapeutics, are the closest to becoming the first drugs to be approved for the treatment of the disorder.
About CureDuchenne Ventures and CureDuchenne
CureDuchenne Ventures LLC collaborates with pharmaceutical and biotechnology companies to facilitate the development of drugs to treat Duchenne muscular dystrophy. CureDuchenne Ventures LLC was formed by CureDuchenne, a national nonprofit that has a successful track record of supporting research and raising awareness of the disease.
CureDuchenne is a national nonprofit organization located in Newport Beach, Calif., dedicated to finding a cure for Duchenne, the most common and most lethal form of muscular dystrophy. As the leading genetic killer of young boys, Duchenne affects more than 300,000 boys worldwide. CureDuchenne has garnered international attention for its efforts to raise funds and awareness for Duchenne. With the help of CureDuchenne's distinguished international panel of Scientific Advisors, funds raised by CureDuchenne support the most promising research aimed at treating and curing Duchenne. To date, seven CureDuchenne research projects have made their way into human clinical trials – a unique accomplishment as few health-related nonprofits have been successful in being a catalyst for human clinical trials.
About Capricor Therapeutics
Capricor Therapeutics, Inc. (CAPR), a publicly traded biotechnology company, is focused on the development of novel therapeutics to prevent and treat heart disease. The Company has two leading product candidates; CAP-1002 and Cenderitide. The Company was formed through the November 2013 merger between Capricor, Inc., a privately held company whose mission is to improve the treatment of heart disease by commercializing cardiac stem cell therapies for patients, and Nile Therapeutics, Inc., a clinical-state biopharmaceutical company developing innovative products for the treatment of cardiovascular disease. For additional information visit www.capricor.com.