Prosensa and GlaxoSmithKline Initiate Development of Four Additional Products
`under Existing Alliance in Duchenne Muscular Dystrophy; Broadened program
`marks key inflexion in Prosensa's progress to a fully integrated specialty-pharma
`company
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`Business Wire
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`June 23, 2010 Wednesday 7:05 AM GMT
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`Copyright 2010 Business Wire, Inc.
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`Distribution: Pharmaceutical Writers; Biotech Writers
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`Length: 810 words
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`Dateline: LEIDEN, Netherlands
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`Body
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`Prosensa, the Dutch biopharmaceutical company focusing on RNA modulating therapeutics, announces the initiation of two
`further programmes under its existing alliance with GSK covering novel RNA-based treatments for Duchenne Muscular
`Dystrophy (DMD). The initiation of these additional programs under the terms of the existing alliance agreement is a validation
`of both the potential of Prosensa's "exon skipping" platform and the ongoing relationship.
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`The two new programs are included under the existing alliance agreement between both parties and will address the
`development of four compounds which target different subpopulations of patients suffering from DMD. To access these new
`programs, GSK has made two initiation payments to Prosensa and Prosensa becomes eligible for further pre-option milestone
`payments based on research progress.
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`Within these new programs, Prosensa and GSK will focus on the skipping of four exons (i.e. exon 45, 52, 53, and 55), in
`addition to their existing programs which target skipping of exon 51 and 44 (PRO051/GSK2402968 and PRO044). The
`initiation of these programs confirms the joint commitment of both companies to find treatments for DMD.
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`Under the terms of the collaboration, GSK has an option to select two of these additional four compounds for later-stage
`development and commercialization. Prosensa will retain certain limited European commercialization rights alongside GSK for
`the two compounds selected by GSK. For the two compounds not selected by GSK, Prosensa will retain full commercialization
`rights.
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`"We are very pleased with this news. Prosensa and GSK's commitment to progress further developments of additional products
`that can provide for a solution in DMD is encouraging and welcomed by all of us" said Elizabeth Vroom, Chair of the United
`Parent Project Muscular Dystrophy, which unites different parent project organizations set up by parents of children with DMD
`in many countries all over the world.
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`"The expansion of our DMD portfolio with four additional compounds is a great step forward in our efforts to develop safe and
`effective treatments for this severely debilitating disease and to help as many patients as possible," said Hans Schikan, Chief
`Executive Officer of Prosensa, adding, "Thanks to the successful progress in our existing program with GSK, we are now able
`to extend our productive collaboration with these four further compounds. With Prosensa retaining full commercialization
`rights to two of these exon skipping compounds and any other products which are not part of the alliance with GSK, this set-up
`enables us to develop into a fully integrated specialty-pharma company."
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`Notes to editors:
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`Prosensa and GlaxoSmithKline Initiate Development of Four Additional Products under Existing Alliance in Duchenne
`Muscular Dystrophy; Broadened program marks ke....
`
`About Prosensa Prosensa is an innovative Dutch biopharmaceutical company focused on the discovery, development and
`commercialization of RNA modulating therapeutics correcting gene expression in diseases with large unmet medical needs, in
`particular neuromuscular disorders. Prosensa's focus is on developing a treatment for DMD. In 2009 Prosensa entered into a
`strategic alliance for part of its DMD exon skipping program with GlaxoSmithKline in a licensing deal that could yield up to
`GBP 437 million plus double-digit royalty payments.
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`Prosensa is a privately held biopharmaceutical company, backed by a consortium of highly regarded investors such as
`Abingworth, AGF Private Equity, GIMV, LSP and MedSciences Capital.For more information about Prosensa, please visit
`www.prosensa.com .
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`About DMD and exon skipping Duchenne Muscular Dystrophy is a severely debilitating childhood neuromuscular disease that
`affects 1 in 3,500 newborn boys. The young patients suffer from progressive loss of muscle strength due to the absence of the
`protein dystrophin, often making them wheelchair bound before the age of 12. Most patients die in early adulthood due to
`respiratory and cardiac failure. Today, there is no treatment to prevent the eventual fatal outcome. The disease is caused by
`mutations in the DMD gene, resulting in the absence of the dystrophin protein, which is crucial for the integrity of muscle fiber
`membranes.
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`RNA-based therapeutics, specifically antisense oligonucleotides inducing exon skipping, are currently amongst the most
`promising therapies for DMD. More specifically, antisense oligonucleotides have the capacity to skip an exon and thereby
`correct the reading frame of DMD transcripts aiming at the synthesis of a largely functional dystrophin protein (for a graphical
`explanation of exon skipping, click here ). Different mutations in the gene require different oligonucleotide drugs.
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`CONTACT: Prosensa Therapeutics BV
`Hans Schikan, Chief Executive Officer
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`h.schikan@prosensa.nl
`or
`Luc Dochez, Chief Business Officer
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`l.dochez@prosensa.nl
`T: +31 71 3322100
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`www.prosensa.com or
`College Hill Life Sciences
`Adam Michael/Anastasios Koutsos
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`prosensa@collegehill.com
`T: +44 20 7457 2020
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`http://www.businesswire.com
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`Load-Date: June 24, 2010
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`End of Document
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