Background: GlaxoSmithKline (GSK) and Prosensa Therapeutics today announced the results of Phase III clinical data for drisapersen, an exon-51 skipping compound for the treatment of Duchenne muscular dystrophy. The data did not meet its primary endpoint of a statistically significant improvement in the 6 Minute Walking Distance (6MWD) test compared to placebo. GSK has indicated it would evaluate the outcome to help inform its next steps for drisapersen. Prosensa said it remains committed to the drug development program and will continue to work closely with GSK.
September 20, 2013
“While we are disappointed by the Phase III trial results for drisapersen, we hold out hope that they will provide critical information to the research community that can be applied in future studies, including those by GlaxoSmithKline and Prosensa,” said Debra Miller, CEO and Founder, CureDuchenne. “We are fully confident in the exon skipping technology as a viable platform to develop a treatment for Duchenne, and Duchenne families should not give up hope.”
In addition to its support of Glaxo and Prosensa, CureDuchenne is working with multiple companies to develop a robust pipeline of potential drugs to treat Duchenne. CureDuchenne also provided early support to Sarepta, another company in trials for its exon skipping drug, eteplirsen. Currently, there is no treatment for Duchenne, a progressive muscle-wasting disease and the most common and lethal form of muscular dystrophy. Duchenne impacts one in every 3,500 boys; nearly 20,000 boys are living with the disease in the United States. Boys with Duchenne are usually diagnosed before the age of 5 and are in a wheelchair by age 12; most don’t survive their mid-20s.
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. With the help of CureDuchenne’s distinguished international panel of Scientific Advisors, the organization has raised more than $11 million towards promising research aimed at treating and curing Duchenne. In fact, 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. For more information on how to help raise awareness and funds needed for research, visit www.cureduchenne.org.