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 www.clinicaltrials.gov.
About DMD
Duchenne Muscular Dystrophy (DMD) is a severely debilitating childhood neuromuscular disease that affects up to 1 in 3,500 live male births. This rare disease is caused by mutations in the dystrophin gene, resulting in the absence or defect of the dystrophin protein.
Patients suffer from progressive loss of muscle function due to the absence or defect of the dystrophin protein, often making them wheelchair bound before the age of 12. Respiratory and cardiac muscle can also be affected by the disease. Few patients survive the age of 30.
About exon skipping
The dystrophin gene is the largest gene in the body, consisting of 79 exons. Exons are small sequences of genetic code which lead to the manufacture of sections of protein. In DMD, when certain exons are mutated/deleted, the RNA cannot read past the fault. This prevents the rest of the exons being read, resulting in a non-functional dystrophin protein and the severe symptoms of DMD.
RNA-based therapeutics, specifically antisense oligonucleotides inducing exon skipping, are currently in development for DMD. This technology uses synthetic antisense oligonucleotides to skip an exon next to a deletion and thereby correct the reading frame, enabling the production of a novel dystrophin protein. Up to 13% of boys with DMD have dystrophin gene mutation/deletions amenable to an exon 51 skip.
About Prosensa Holding N.V.
Prosensa (NASDAQ: RNA) is a Dutch biotechnology company engaged in the discovery and development of RNA-modulating therapeutics for the treatment of genetic disorders. Its primary focus is on rare neuromuscular and neurodegenerative disorders with a large unmet medical need, including Duchenne Muscular Dystrophy, myotonic dystrophy and Huntington's disease. www.prosensa.com
Forward Looking Statement
This press release contains certain forward-looking statements. All statements, other than statements of historical facts, contained in this press release, including statements regarding our strategy, future operations, future financial position, future revenues, projected costs, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "estimate," "expect," "intend," "may," "plan," "predict," "project," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements around our exon -skipping drug candidates and our collaboration with GlaxoSmithKline (GSK). Actual results may differ materially from those projected or implied in such forward-looking statements. Such forward-looking information involves risks and uncertainties that could significantly affect expected results. These risks and uncertainties are discussed in the Company's SEC filings, including, but not limited to, the Company's Form 6-K containing this press release and certain sections of the Company's Registration Statement on Form F-1. In addition, any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. While we may elect to update these forward-looking statements at some point in the future, we specifically disclaim any obligation to do so, even if our views change.