Analyses and assessment of next steps are ongoing
PPMD just received the following update from GSK regarding the current status of their drisapersen study:
Dear Patient Group Representative,
On the 20th of September this year, we shared the results about our Phase III study (DMD114044) of drisapersen in boys with Duchenne Muscular Dystrophy – we did not see a statistically significant difference in 6 Minute Walk Distance, the primary endpoint, between the boys who received drisapersen and those who received placebo. At that time, the dosing of drisapersen within the ongoing clinical studies was placed on hold and we set about evaluating the results in the context of the overall clinical programme together with performing additional analyses to fully understand the results of the Phase III study.
It was anticipated that these additional analyses were to be completed by year end, but together with Prosensa, we are still analysing the results and assessing our next steps. The outcome of the evaluation, which will include integrated results across the overall drisapersen clinical programme, including data from the long term extension studies, is anticipated in January 2014. In addition, as publicly announced at the recent PPMD Duchenne Policy Forum, the FDA is currently looking at the drisapersen data, in part to understand clinical and biomarker endpoints.
We are taking additional time to ensure that we have addressed the key questions to support our assessment on the overall benefit-to-risk profile of drisapersen in boys with DMD. Until then, dosing of drisapersen will continue to be held within the ongoing studies. Study investigators will also be informed. In the meantime, per GSK’s policy, we will be posting a summary of the DMD114044 results on GSK Clinical Study Register (www.gsk-clinicalstudyregister.com).
We appreciate that this is a difficult time for the boys and their families and thank them for their patience as we thoroughly explore the data from the drisapersen clinical development programme. We want to ensure that we are making informed decisions regarding this program for the best interest of the boys with DMD. We currently plan to communicate the final assessment of our next steps during the early part of 2014.
Please note that our offices will be closed from 24 Dec 2013 to 1 Jan 2014 and we will respond to any questions that you may have regarding this communication as soon as we can. We would like to take this opportunity to thank you once again for the efforts and support you provide to help the boys with DMD.
With best wishes for the holidays.
GSK Global Patient Relations Team
Comments from Prosensa regarding the drisapersen program update (read the full release):
Prosensa Holding N.V. (NASDAQ: RNA), the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, commented on a recent update by GlaxoSmithKline (GSK) to patient groups and investigators regarding the ongoing analyses for drisapersen, an investigational antisense oligonucleotide, for the treatment of Duchenne Muscular Dystrophy (DMD) patients with an amenable mutation, which is exclusively licensed to GSK.
On September 20, GSK and Prosensa announced that results of a Phase III study (DMD114044) of drisapersen in boys with DMD did not meet the primary endpoint. At that time, together with Prosensa, GSK began to evaluate the results in the context of the overall clinical program in addition to performing additional analyses to fully understand the results of this study.
Earlier today, GSK provided an update to patient groups and investigators that the analysis of the results and assessment of next steps are still ongoing. The outcome of this evaluation is anticipated in early 2014. In addition, as publicly announced at the recent PPMD Duchenne Policy Forum, the US Food and Drug Administration is currently looking at the drisapersen data, in part to understand clinical and biomarker endpoints.
In the meantime, GSK has stated that as per their policy, the summary results of the DMD114044 study will be posted on GSK's Clinical Study Register.
Prosensa's Chief Executive Officer, Hans Schikan, comments, "Given the devastating impact of DMD on boys and their families, it is of critical importance that this robust dataset is understood in its entirety and no stone is left unturned." Schikan continued, "The drisapersen program represents one of the largest datasets in this disease to date, and we continue to collaborate with patient groups and other key stakeholders to help patients with DMD."
About drisapersen and the clinical development program
Drisapersen, (previously GSK2402968/PRO051) an antisense oligonucleotide, which induces exon skipping of exon 51, is currently in late stage development for DMD.
GSK obtained an exclusive worldwide license to develop and commercialize drisapersen from Prosensa in 2009. Drisapersen has been designated orphan drug status in the EU, US and Japan. In June 2013, drisapersen was granted Breakthrough Therapy designation by the US Food and Drug Administration.
The overall clinical program includes two open label extension studies, DMD114673 and DMD114349, as well as three double blind, placebo controlled studies, DMD114117, DMD114876 and DMD114044. For more information regarding the clinical studies involving drisapersen visit www.clinicaltrials.gov.