Drisapersen, Etiplersen, GSK, Prosensa and Sarepta, a Positive Week for Duchenne

Thanks to GSK, Prosensa and Sarepta and, the landscape is changing for the patients and families that are suffering from Duchenne, a severe muscle disease that shortens the life expectancy and quality of life for approximately 300,000 boys and young men worldwide. Even though these potential treatments that are currently in the news have not yet been approved, and there is still much work to do, I’d like to take a moment and say thank you to just a few of the key players that are giving us a chance for hope for our sons.

Today at Cold Springs Harbor, John Kraus, GSK, gave more details from this week’s news that drisapersen, did indeed show positive results in the Phase ll trial. The GSK team has brought a new level of resources and experience to Duchenne research that only a large pharmaceutical company can bring. The GSK team has been diligent in pushing trials forward around the world, having big pharma pay attention to Duchenne is a positive force for this disease. http://www.fiercebiotech.com/story/glaxosmithkline-measures-38-meter-leap-over-placebo-duchenne-walking-test/2013-04-11

Hans Schikan, CEO of Prosensa, and team developed their lead compound, to the point that they were able to partner with GSK to take this drug, drisapersen into several clinical trials, which includes over 300 patients. The results from the pivotal Phase lll should be available late this year. We have known the Prosensa team since 2004 when we became the first non-profit organization to contribute to their drug development program. They have two other exon skipping projects in clinical trials right now and we appreciate their vision and dedication. I’d also like to thank the CureDuchenne donors who contributed significant personal funds so that CureDuchenne could develop this partnership at a time when exon skipping was a big unknown.

Chris Garabedian and his team at Sarepta have done a phenomenal job in executing a strategy that has resulted in positive results in their Phase ll trial for their lead drug, etiplersen. Just today, Sarepta announced that they have licensed exon skipping molecules for mutations that are not as common as the current patients who are participating in the trial. CureDuchenne has been working with Dr. Kevin Flanigan at Nationwide Children’s Hospital to screen and investigate the possibility of using exon skipping for rare Duchenne mutations such as duplications, so we are very pleased to see this collaboration between Dr. Steve Wilton, University of Western Australia and Sarepta. We can’t stop until all Duchenne boys and young men have a treatment. Dr. Wilton has been working on antisense for many, many years. http://investorrelations.sareptatherapeutics.com/phoenix.zhtml?c=64231&p=RssLanding&cat=news&id=1805787

CureDuchenne was an early contributor to both Sarepta (formerly AVI) and Prosensa, but there have been numerous donors and investors that have enabled these companies to get this far. Notably, Dr. Eric Hoffman at Children’s National Medical Center and Foundation to Eradicate Duchenne were protagonists in funding Sarepta several years ago.

These and many more individuals have dedicated their careers, donors have given their personal wealth and investors have poured millions into these projects.  Given how little government funding is allocated to Duchenne research, the contributions of these people and companies have and will continue to be important to the Duchenne community.

As parents, we all want a treatment and cure for our kids. I think it’s important to take a moment and acknowledge these individuals and companies and many, many others who have gotten us this far, and give them a big thank you. Please know that we are counting on you and we want to help any way we can.