Prosensa Awarded up to €5m Innovation Credit from Dutch Government for research in Myotonic Dystrophy

Leiden, The Netherlands, 25 October 2011 – Prosensa, the Dutch company focusing on RNA modulating therapeutics for rare diseases with unmet medical needs, announced today that it has received an innovation credit of up to €5m from AgentschapNL, a subsidiary of the Dutch Ministry of Economic Affairs, Agriculture and Innovation, to support further development of a series of preclinical compounds for the treatment of Myotonic Dystrophy type 1 (DM1).

According to the terms of the agreement, Prosensa will receive €940,000 to support the initial stage of the project, with the potential to receive additional funding of up to €5m after successful completion of specific milestones and an initial review in 2012.

The genetic mutation underlying DM1 is characterized by expansion of so-called triplet repeats in the DMPK gene leading to transcripts with toxic effects. Prosensa applies its RNA modulation platform to reduce the number of these expanded repeat, toxic transcripts. One compound, PRO135, reversed the transcript toxic effects after in vivo intramuscular administration in preclinical DM1 models. Prosensa works in close collaboration with the department of Cell Biology, Nijmegen Centre for Molecular Life Sciences, Radboud University Nijmegen Medical Centre, on this project, one of the leading centres in DM1 research in the world.

Judith van Deutekom, Vice President of Discovery at Prosensa commented: “We are very pleased with this grant by the Dutch government, which allows us to accelerate our research in this important therapeutic area and helps us grow into a specialist rare disease company. The innovation credit from the Dutch government is intended to stimulate development of technologically innovative products. In addition to Duchenne muscular dystrophy, we are also working on a potential therapy for patients affected by Myotonic Dystrophy, yet another rare disease for which no treatment currently exists.”

Notes to editors:

About Myotonic Dystrophy

Myotonic Dystrophy is a rare, progressive, degenerative disorder that affects many organs in the body, but primarily skeletal muscle, heart and brain. It is recognized as one of the most common forms of muscular dystrophy in adults and affects approximately 1:8,000 individuals worldwide. Typical symptoms are myotonia, gastro-intestinal problems, respiratory insufficiency and several cognitive problems.

There is currently no effective treatment available for DM1 patients and the congenital form (1:20,000 newborns) leads to a life-long severe disability and premature death.

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 current focus is on developing treatments for Duchenne muscular dystrophy (DMD), Myotonic Dystrophy (DM1) and Huntington’s disease. In 2009 Prosensa entered into a strategic alliance for part of its DMD exon skipping program with GlaxoSmithKline. Prosensa’s lead compound (GSK2402968/PRO051), being developed by GSK, entered phase III clinical trials in January 2011.

Prosensa is a privately held biopharmaceutical company, backed by a consortium of Abingworth, AGF Private Equity, GIMV, LSP and MedSciences Capital. For more information about Prosensa, please visit

 Prosensa recently was shortlisted for the Biotech Company of the Year category at the 2011 Scrip Awards, as well as for the 2011 Most Innovative SME Award by EuropaBio.

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