Prosensa Regains Rights to Drisapersen From GSK and Re.

Posted: Published on January 13th, 2014

This post was added by Dr Simmons

(Nasdaq:RNA) and GlaxoSmithKline (GSK) today announcedthat Prosensa has regained all rights from GSK to drisapersen and will retainrights to all other programs for the treatment of Duchenne Muscular Dystrophy(DMD). This transfer of rights represents the termination of the collaborationagreement between GSK and Prosensa executed in 2009. Prosensa will now have the full, unencumbered rights to continue thedevelopment of drisapersen as well as each of its DMD programs. The parties have agreed that Prosensa is well suited to continue thedevelopment of all of the DMD programs. Prosensa and GSK have also agreed tomake certain data from the drisapersen studies available in due course to thescientific community for the purpose of furthering the general understanding ofDMD. 'We are fully committed to our mission of developing innovative, RNA-basedtherapeutics to address unmet medical needs for patients with rare geneticdisorders,' said Hans Schikan, Prosensa's Chief Executive Officer. 'Prosensa isnow in a favorable strategic position to advance the DMD portfolio, whichincludes drisapersen and five additional compounds, three of which arecurrently in clinical development. We will continue to work closely withpatient groups, investigators, academia and regulators to ensure that we doeverything we can to bring treatments to boys affected by DMD.' 'We have completed our review and believe that further analysis of drisapersendata may benefit from being viewed in the context of the body of informationwhich has been developed by Prosensa through their work on additionalexon-skipping programs. We greatly valued the scientific expertise thatProsensa provided since our collaboration started in 2009.' said Dr CarloRusso, Senior Vice President, Head of GSK Rare Diseases Research & Development.'We worked closely with Prosensa with the aim of developing new medicines tohelp boys with DMD and thanks to the dedication of the boys and their families,we have gathered the largest clinical dataset related this condition. The valueof this data to the scientific community's understanding of DMD should not beunderestimated, and we would like to thank everyone who participated in thestudies for their contributions and patience as we worked through ouranalyses.' Prosensa's Schikan, added 'GSK has been a valuable development partner, and weare grateful for the commitment made by the company over the last four years todevelop a disease modifying therapy for this devastating disease. Together wehave been able to progress the largest global clinical program in DMD, and wehave made tremendous progress in understanding the natural history andepidemiology of this rare and devastating disease. We look forward to updatingthe community with our plans as soon we are in a position to do so.' About drisapersen and the clinical development programDrisapersen, (previously GSK2402968/PRO051), an antisense oligonucleotide whichinduces exon skipping of exon 51, is currently in-late stage development forDMD. In 2009 GSK obtained an exclusive worldwide license from Prosensa todevelop and commercialize drisapersen. Drisapersen has orphan drug status inthe EU, US, Japan and Australia. In June 2013, drisapersen was grantedBreakthrough Therapy designation by the US Food and Drug Administration. The overall drisapersen clinical program comprises three double-blind,placebo-controlled studies (DMD114117, DMD114876 and DMD114044) and two longterm open-label extension studies (DMD114673 and DMD114349). For more information regarding the ongoing clinical studies involvingdrisapersen visit http://www.clinicaltrials.gov. About DMDDuchenne Muscular Dystrophy (DMD) is a severely debilitating childhoodneuromuscular disease that affects up to 1 in 3,500 live male births. This raredisease is caused by mutations in the dystrophin gene, resulting in the absenceor defect of the dystrophin protein. Patients suffer from progressive loss ofmuscle function, often making them wheelchair bound before the age of 12.Respiratory and cardiac muscle can also be affected by the disease. Fewpatients survive the age of 30. About exon skippingThe 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 ofsections of protein. In DMD, when certain exons are mutated/deleted, the RNAcannot read the genetic code past the fault. This prevents the rest of theexons being read, resulting in a non-functional dystrophin protein and thesevere symptoms of DMD. RNA-based therapeutics, specifically antisense oligonucleotides inducing exonskipping, are currently in development for DMD. This technology uses syntheticantisense oligonucleotides to skip an exon next to a deletion and therebycorrect the reading frame, enabling the production of a novel dystrophinprotein. Up to 13% of boys with DMD have dystrophin gene mutation/deletionsamenable to an exon 51 skip. About Prosensa Holding N.V.Prosensa (Nasdaq:RNA) is a Dutch biotechnology company engaged in the discoveryand development of RNA-modulating therapeutics for the treatment of geneticdisorders. Its primary focus is on rare neuromuscular and neurodegenerativedisorders with a large unmet medical need, including Duchenne musculardystrophy, myotonic dystrophy and Huntington's disease. http://www.prosensa.comGSK - one of the world's leading research-based pharmaceutical and healthcarecompanies - is committed to improving the quality of human life by enablingpeople to do more, feel better and live longer. For further information pleasevisit http://www.gsk.com. Prosensa Contact: Prosensa Holding N.V. Celia Economides, Senior Director IR & Corporate CommunicationsPhone: +1 917 941 9059 Email: c.economides@prosensa.nl GSK enquiries: UK Media enquiries: David Mawdsley +44 (0) 20 8047 (London) 5502 Simon Steel +44 (0) 20 8047 (London) 5502 David Daley +44 (0) 20 8047 (London) 5502 Catherine Hartley +44 (0) 20 8047 (London) 5502 US Media enquiries: Stephen Rea +1 215 751 4394 (Philadelphia) Melinda Stubbee +1 919 483 2510 (North Carolina) Mary Anne Rhyne +1 919 483 0492 (North Carolina) Emily Beamer +1 215 751 6622 (Philadelphia) Jennifer Armstrong +1 215 751 5664 (Philadelphia) Analyst/Investor Sally Jackson +44 (0) 20 8047 (London) enquiries: 5543 Kirsty Collins (SRI & +44 (0) 20 8047 (London) CG) 5534 Tom Curry + 1 215 751 5419 (Philadelphia) Gary Davies +44 (0) 20 8047 (London) 5503 James Dodwell +44 (0) 20 8047 (London) 2406 Jeff McLaughlin +1 215 751 7002 (Philadelphia) Ziba Shamsi +44 (0) 20 8047 (London) 3289 Lucy Singah +44 (0) 20 8047 (London) 2248 Prosensa Forward Looking StatementsThis 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, futurefinancial position, future revenues, projected costs, prospects, plans andobjectives 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 identifyforward-looking statements, although not all forward-looking statements containthese identifying words. Forward-looking statements in this press releaseinclude statements around our exon -skipping drug candidates and ourcollaboration with GlaxoSmithKline (GSK). Actual results may differ materiallyfrom those projected or implied in such forward-looking statements. Suchforward-looking information involves risks and uncertainties that couldsignificantly affect expected results. These risks and uncertainties arediscussed in the Company's SEC filings, including, but not limited to, theCompany's Form 6-K containing this press release and certain sections of theCompany's Registration Statement on Form F-1. In addition, any forward-lookingstatements represent our views only as of today and should not be relied uponas representing our views as of any subsequent date. While we may elect toupdate these forward-looking statements at some point in the future, wespecifically disclaim any obligation to do so, even if our views change. GSK Cautionary statement regarding forward-looking statementsGSK cautions investors that any forward-looking statements or projections madeby GSK, including those made in this announcement, are subject to risks anduncertainties that may cause actual results to differ materially from thoseprojected. Factors that may affect GSK' s operations are described under Item3.D 'Risk factors' in the company's Annual Report on Form 20-F for 2012. GSK Registered in England & Wales:No. 3888792 GSK Registered Office: 980 Great West Road Brentford, Middlesex TW8 9GSNews Source: NASDAQ OMXEnd of Corporate News---------------------------------13.01.2014 Dissemination of a Corporate News, transmitted by DGAP - acompany of EQS Group AG.The issuer is solely responsible for the content of this announcement.DGAP's Distribution Services include Regulatory Announcements,Financial/Corporate News and Press Releases.Media archive at http://www.dgap-medientreff.de and http://www.dgap.de---------------------------------Language: English Company: Prosensa Holding N.V. Netherlands ISIN: NL0010524443 End of News DGAP News-Service --------------------------------- 247766 13.01.2014

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Prosensa Regains Rights to Drisapersen From GSK and Re.

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