Prosensa Comments on Drisapersen Program Update

Leiden, The Netherlands, Dec. 19, 2013 (GLOBE NEWSWIRE) — 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

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