SEATTLE, Feb. 24 2017 /PRNewswire/ -- DMD Therapeutics, Inc., a start-up biotech company, is developing proprietary therapeutics for Duchenne muscular dystrophy (DMD), an orphan disease associated with serious morbidity and early mortality that has a major unmet need. The Company's first drug candidate, DMD-813, has been shown to reduce damage and inflammation in muscle leading to markedly increased muscle strength in the standard mouse model of DMD (mdx mice). Further studies have documented significant increases in the ability of treated animals to ambulate long distances, which is important given that DMD boys become wheelchair bound due to loss of ambulation. With $400,000 in initial funding committed from three foundations, Ryan's Quest, Michael's Cause, and Pietro's Fight, DMD Therapeutics is now moving DMD-813 into drug development.
"The preclinical results with DMD-813 therapy are striking, and its primary mechanism of action affects one of the molecular mechanisms responsible for muscle weakness in DMD. There are few if any other drug candidates that demonstrate the same level of efficacy in preventing muscle damage and restoring muscle strength in mdx mice," said Carlo Rago, PhD, Chief Scientific Officer and Co-Founder of DMD Therapeutics.
"DMD-813 brings together many of the attributes of an ideal treatment for DMD. It has demonstrated its therapeutic effects in skeletal muscle, including the diaphragm, a major cause of pulmonary failure that often leads to death in DMD," said Ron Berenson, MD, Co-Founder of DMD Therapeutics.
Drs. Berenson and Rago are joined by a team of experts in biotechnology, who have extensive experience in all aspects of drug development, including animal studies, manufacturing, toxicology, clinical trials, and regulatory affairs. Ryan's Quest, Michael's Cause, and Pietro's Fight have provided the initial seed funding to facilitate R&D and early manufacturing steps.
"We are confident that this management team will drive this promising agent through development and clinical trials. If the preclinical results hold up in patients, we should end up with a highly effective therapy for DMD," said Dave Schultz, Founder of Ryan's Quest.
About DMD Therapeutics Inc.
DMD Therapeutics Inc. is a privately held corporation registered in Delaware. The Company was founded by Carlo Rago, PhD and Ron Berenson, MD in 2016 to develop novel therapeutics for DMD and other muscular dystrophies.
About the Duchenne Foundations
Ryan's Quest, Michael's Cause, and Pietro's Fight are 501(c)3 non-profit foundations that work closely together to evaluate and fund the most promising candidate therapies for DMD. They focus on funding preclinical, IND-enabling, and clinical studies. Follow and support these foundations at www.ryansquest.org, www.pietrosfight.org, and www.michaelscause.org.
About Duchenne Muscular Dystrophy
DMD is an X-linked genetic disorder, which occurs in approximately 1 in 3,800 male births with an incidence rate that does not significantly vary around the world. In rare cases, females are affected. There are approximately 15,000 and 32,000 individuals living with this disease in the US and Europe, respectively. It is a severe disorder characterized by progressive deterioration of muscle function, which becomes evident in children at approximately 3-5 years old. Patients become wheelchair bound in the early second decade of life. In their late teens and early twenties, most patients develop cardiac and pulmonary failure leading to death in their late twenties to early thirties.
SOURCE DMD Therapeutics Inc.