Prosensa Comments On Drisapersen Program Update

Leiden, The Netherlands, Dec. 19, 2013 (GLOBE NEWSWIRE) -- Prosensa Holding N.V. (NASDAQ: RNA), the Dutch biopharmaceuticalcompany focusing on RNA-modulating therapeutics for rare diseaseswith high unmet need, commented on a recent update byGlaxoSmithKline (GSK) to patient groups and investigators regardingthe ongoing analyses for drisapersen, an investigational antisenseoligonucleotide, for the treatment of Duchenne Muscular Dystrophy(DMD) patients with an amenable mutation, which is exclusivelylicensed to GSK.

On September 20, GSK and Prosensa announced that results of aPhase III study (DMD114044) of drisapersen in boys with DMD did notmeet the primary endpoint. At that time, together with Prosensa,GSK began to evaluate the results in the context of the overallclinical program in addition to performing additional analyses tofully understand the results of this study. 

Earlier today, GSK provided an update to patient groups andinvestigators that the analysis of the results and assessment ofnext steps are still ongoing. The outcome of this evaluation isanticipated in early 2014.  In addition, as publicly announcedat the recent PPMD Duchenne Policy Forum, the US Food and DrugAdministration is currently looking at the drisapersen data, inpart to understand clinical and biomarker endpoints.In the meantime, GSK has stated that as per their policy, thesummary results of the DMD114044 study will be posted on GSK'sClinical Study Register.

Prosensa's Chief Executive Officer, Hans Schikan, comments,"Given the devastating impact of DMD on boys and their families, itis of critical importance that this robust dataset is understood inits entirety and no stone is left unturned." Schikan continued,"The drisapersen program represents one of the largest datasets inthis disease to date, and we continue to collaborate with patientgroups and other key stakeholders to help patients with DMD."

About drisapersen and the clinical developmentprogram

Drisapersen, (previously GSK2402968/PRO051) an antisenseoligonucleotide, which induces exon skipping of exon 51, iscurrently in late stage development for DMD.

GSK obtained an exclusive worldwide license to develop andcommercialize drisapersen from Prosensa in 2009. Drisapersen hasbeen designated orphan drug status in the EU, US and Japan. In June2013, drisapersen was granted Breakthrough Therapy designation bythe US Food and Drug Administration.

The overall clinical program includes two open label extensionstudies, DMD114673 and DMD114349, as well as three double blind,placebo controlled studies, DMD114117, DMD114876 and DMD114044. Formore information regarding the clinical studies involvingdrisapersen visit www.clinicaltrials.gov.

About DMD

Duchenne Muscular Dystrophy (DMD) is a severely debilitatingchildhood neuromuscular disease that affects up to 1 in 3,500 livemale births. This rare disease is caused by mutations in thedystrophin gene, resulting in the absence or defect of thedystrophin protein.

Patients suffer from progressive loss of muscle function due tothe absence or defect of the dystrophin protein, often making themwheelchair bound before the age of 12. Respiratory and cardiacmuscle can also be affected by the disease. Few patients survivethe age of 30.

About exon skipping

The dystrophin gene is the largest gene in the body, consistingof 79 exons. Exons are small sequences of genetic code which leadto the manufacture of sections of protein. In DMD, when certainexons are mutated/deleted, the RNA cannot read past the fault. Thisprevents the rest of the exons being read, resulting in anon-functional dystrophin protein and the severe symptoms ofDMD.

RNA-based therapeutics, specifically antisense oligonucleotidesinducing exon skipping, are currently in development for DMD. Thistechnology uses synthetic antisense oligonucleotides to skip anexon next to a deletion and thereby correct the reading frame,enabling the production of a novel dystrophin protein. Up to 13% ofboys with DMD have dystrophin gene mutation/deletions amenable toan exon 51 skip.

About Prosensa Holding N.V.

Prosensa (NASDAQ: RNA) is a Dutch biotechnology company engagedin the discovery and development of RNA-modulating therapeutics forthe treatment of genetic disorders. Its primary focus is on rareneuromuscular and neurodegenerative disorders with a large unmetmedical need, including Duchenne Muscular Dystrophy, myotonicdystrophy and Huntington's disease.  www.prosensa.com

Forward Looking Statement

This press release contains certain forward-lookingstatements.  All statements, other than statements ofhistorical facts, contained in this press release, includingstatements regarding our strategy, future operations, futurefinancial 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 areintended to identify forward-looking statements, although not allforward-looking statements contain these identifying words.Forward-looking statements in this press release include statementsaround our exon -skipping drug candidates and our collaborationwith GlaxoSmithKline (GSK). Actual results may differ materiallyfrom those projected or implied in such forward-lookingstatements.  Such forward-looking information involves risksand uncertainties that could significantly affect expectedresults.  These risks and uncertainties are discussed in theCompany's SEC filings, including, but not limited to, the Company'sForm 6-K containing this press release and certain sections of theCompany's Registration Statement on Form F-1. In addition, anyforward-looking statements represent our views only as of today andshould not be relied upon as representing our views as of anysubsequent date. While we may elect to update these forward-lookingstatements at some point in the future, we specifically disclaimany obligation to do so, even if our views change.
CONTACT: Prosensa Holding N.V.         Celia Economides, Director IR & Corporate Communications         Phone: +1 917 941 9059         Email: c.economides@prosensa.nl         

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