Leiden, The Netherlands, Aug. 1, 2013 (GLOBE NEWSWIRE) -- Prosensa Holding N.V. (NASDAQ: RNA), the Dutch biopharmaceuticalcompany focusing on RNA-modulating therapeutics for rare diseaseswith high unmet need, today announced that it has been awarded aprestigious Framework Programme 7 (FP7) research grant from theEuropean Union to support the ongoing clinical study of theCompany's third novel development candidate, PRO045, for thetreatment of Duchenne muscular dystrophy (DMD). The project, titled "Consortium for Products Across Europe inDuchenne Muscular Dystrophy" (SCOPE-DMD) is expected to run forthree years. Prosensa is part of a high-quality, pan-European consortium thathas been awarded the grant of approximately EUR 6 million toevaluate its exon 45-skipping drug candidate, PRO045, in aninnovatively designed clinical proof-of-concept study in boys withDMD. The consortium includes leading expert centers in DMD such asNewcastle University in the United Kingdom, the coordinatingpartner within the project, the Institute of Myology in France andLeiden University Medical Center in the Netherlands, as well as anindustrial partner, BioSpring in Germany. The combined experienceand expertise of this consortium will help advance the developmentof PRO045 which entered clinical trials earlier this year. "We are delighted with this funding, which will allow us tofurther advance and accelerate the development of anotherexon-skipping compound. The Duchenne community is eagerly awaitinga swift development of products which target additional exons afterthe progress with exon 51 skipping," commented Prof. Volker Straub,M.D., Professor of Neuromuscular Genetics and Harold MacmillanChair of Medicine at Newcastle University's Institute of GeneticMedicine. Professor Straub is also the former coordinator ofTREAT-NMD, a key initiative to create infrastructure to ensure thatthe most promising new therapies for neuromuscular diseases reachpatients as quickly as possible. "FP7 grants are awarded on the basis of a highly competitive,two-stage, peer-review process, therefore this award serves asvalidation of our exon-skipping technology platform," said HansSchikan, CEO of Prosensa. "We very much look forward to workingwith this esteemed consortium on PRO045 and are grateful to the EUfor its support of important clinical research initiatives thatcould have a significant impact on the health and lives of youngboys diagnosed with this rare and devastating genetic disease," headded.
PRO045 is currently in a Phase I/IIa dose-escalating safetystudy to assess its safety and efficacy. The compound induces exon45 skipping in the dystrophin gene and could be suitable forapproximately 8% of all DMD patients. PRO045 has been grantedorphan drug status in the European Union and the United States.FP7 is the EU's main instrument for supporting innovativeresearch in Europe, and has two main strategic objectives: tostrengthen the scientific and technological base of Europeanindustry and to encourage its international competitiveness, whilepromoting research that supports EU policies. 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 About DMD Duchenne muscular dystrophy (DMD) is a severely debilitatingchildhood neuromuscular disease that affects 1 in 3,500 live malebirths. This rare disease is caused by mutations in the dystrophingene, resulting in the absence or defect of the dystrophin protein.As a result, patients suffer from progressive loss of musclestrength, often rendering them wheelchair-bound before the age of12. Respiratory and cardiac muscle can also be affected by thedisease and most patients die in early adulthood due to respiratoryand cardiac failure. About exon skipping and RNA modulation The dystrophin gene is the largest gene in the body, consistingof 79 exons. Exons are small sequences of genetic code which, viaan intermediate step involving RNA, lead to the assembly ofsections of protein. In DMD, when certain exons aremutated/deleted, the RNA cannot read past the fault. This preventsthe remainder of the exons from being read, resulting in anon-functional dystrophin protein and the severe symptoms of DMD.RNA-based therapeutics, specifically antisense oligonucleotidesinducing exon skipping, are currently in development for DMD. Theseantisense oligonucleotides skip an exon next to a defective exonand thereby correct the reading frame, enabling the production of anovel, largely functional dystrophin protein. Prosensa's exonskipping technology was licensed from Leiden University MedicalCenter.
Forward Looking StatementThis 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 45-skipping drug candidate, PRO045, and grantfunding around this program. 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.
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