UPDATE AS OF 10/4/13: We received the following Q&A from GSK today, addressing many of the community's questions. Click here.
Today we learned that GSK’s phase III study of the exon 51 skipping drug drisapersen failed to show a statistically significant improvement in the six-minute walk test compared to placebo (Read the official press release and GSK's statement, below). We know that all of those families around the world who participated in this study are particularly devastated—it’s not at all trivial to participate in a trial like this. I think it is also pretty fair to say that the GSK staff involved in this study are devastated as well.
GSK will address the Duchenne community via Webinar on October 15th in collaboration with PPMD, CureDuchenne, Action Duchenne, and UPPMD. Details will be forthcoming and you will have the opportunity to submit your questions.
This is not the first time this community has experienced a heart-wrenching set back and, unfortunately, it probably won’t be the last. Currently 42% of all phase III trials fail, largely due to lack of efficacy. Given the resources and time required to conduct a phase III study, this rate of failure is not sustainable in rare disease. PPMD is committed to improving the quality of drug candidates for Duchenne to the extent that it is possible to do so. Ultimately, though, a phase III trial is an experiment and we won’t know the answer until we do the experiment.
For all of those families participating in any type of clinical research you should know that you are heroes in this fight against Duchenne. We lost a battle, not the war.
Dear Patient Group Representative,
I hope this message finds you well.
I am writing to share some disappointing news about our Phase III study of drisapersen in patients with Duchenne Muscular Dystrophy – we recently completed analysis of the data and did not see a statistically significant difference in 6 Minute Walk Distance between patients who received drisapersen and those who received placebo. The study also included key assessments of motor function and unfortunately there was no treatment difference in these endpoints: 10-meter walk/run test, 4-stair climb and North Star Ambulatory Assessment. The safety profile in this study was generally consistent with what was seen in earlier trials and the most commonly reported adverse events included injection site reactions and proteinuria. No patients had thrombocytopenia in this study. We will be closely working with the patient advocacy community to arrange a webinar of the results to provide an opportunity for boys and their families to ask questions.
We believe there is value in evaluating these results in the context of the overall development programme and additional analyses are undergoing and planned to fully understand the results of this Ph III study. We will also be consulting with experts in the field before deciding on next steps for drisapersen. Until this work is complete, anticipated by year end, we are holding all dosing with drisapersen within ongoing studies. Although treatment dosing will be held, safety monitoring and other relevant assessments will continue. We have notified study investigators and are recommending that patients currently participating in ongoing drisapersen clinical studies contact their local clinical study team. You can find information regarding the ongoing clinical studies involving drisapersen, by visiting www.clinicaltrials.gov.
GSK and Prosensa issued a press release to announce the results and I have attached the release for reference. We have submitted the study results for presentation at a forthcoming scientific meeting and will also be submitting a paper for publication in a scientific peer-review journal.
I appreciate that these results are not the outcome that any of us had hoped for and I would like to sincerely thank all those who participated in the study for their commitment. I have provided my contact information below, should you have any questions.