CureDuchenne Ventures Launched to Accelerate Drug Discovery and Development for Duchenne Muscular Dystrophy

NEWPORT BEACH, Calif., September 26, 2013— CureDuchenne announced today the launch of  CureDuchenne Ventures, a new initiative to identify and develop a robust pipeline of therapies to treat Duchenne, the most common and lethal form of muscular dystrophy. This drug discovery and development initiative is based on CureDuchenne’s successful venture philanthropy model, in which the organization provides early stage funding to biotechnology, pharmaceutical companies and academic investigators to help develop breakthrough therapies to treat Duchenne.

“We’re launching CureDuchenne Ventures at an exciting time in Duchenne research,” said Debra Miller, CEO and Founder of CureDuchenne. “Duchenne is a progressive disease and currently there is no treatment or cure, so our goal is to accelerate research that could benefit this generation of those impacted with Duchenne.”

CureDuchenne Ventures will focus on venture philanthropists, foundations, and donors with an interest in scientific research for rare diseases, such as Duchenne. The first research project to be funded through CureDuchenne Ventures is a collaboration with biopharmaceutical company, Lexicon Pharmaceuticals (Woodlands, TX). Lexicon’s experimental drug, LX2931, which is in clinical development as a potential treatment of rheumatoid arthritis and autoimmune disease, is currently being evaluated for its potential to initiate muscle regeneration in studies with the mdx mouse model of Duchenne. CureDuchenne plans to raise $5 million to support clinical development of LX2931 as a potential treatment for Duchenne, with clinical studies slated to begin as early as 2014.

“Multiple technologies exist to repair, restore or replace the missing protein in Duchenne; however, the limiting factor in getting treatments out of the laboratory and into the clinic is funding,” said Michael G. Kelly, Chief Scientific Officer, CureDuchenne. “CureDuchenne Ventures will power our ability moving forward to support the most promising therapies and ensure that multiple avenues of research are explored. Multiple therapeutic strategies, working together, will be needed to effectively treat the whole disease, and the purpose of our drug discovery and development initiative is to identify and support those programs that would critically impact the various components of the disease.”

CureDuchenne is aided by an in-house Chief Scientific Officer with significant drug development experience and a Scientific Advisory Board that includes the world’s foremost scientists, innovators and thought leaders working on Duchenne. The organization has adopted a broad research funding strategy that takes a comprehensive pathophysiological approach to treating the disease. This includes protein replacement strategies including exon skipping; treatments targeted at reducing the severity of the disease and slowing its progression; drug repositioning; and strategies to address cardiac issues that accompany the disease.

Currently, there is no treatment for Duchenne. Duchenne is a progressive muscle-wasting disease that impacts approximately 1 in 3,500 boys. Boys with Duchenne are usually diagnosed before the age of five and are in a wheelchair by age 12; most don’t survive their mid-20s.

Through its venture philanthropy approach, CureDuchenne already has raised more than $11 million toward promising research aimed at treating and curing Duchenne. CureDuchenne has funded seven research projects that have advanced to human clinical trials. CureDuchenne’s research investments have leveraged more than $100 million from biotech and pharmaceutical companies, and other foundations to fund research leading to a cure.

CureDuchenne was a very early supporter of exon skipping research, and funded development of eteplirsen (Sarepta Therapeutics) and drisapersen (GlaxoSmithKline/Prosensa Therapeutics).

About CureDuchenne

CureDuchenne is a national nonprofit organization 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 through venture philanthropy. For more information on how to help raise awareness and funds needed for research, visit www.cureduchenne.org.