Prosensa and Partners Awarded Second European Consortium FP7 Research Grant

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Leiden, The Netherlands and Newcastle, UK, Nov. 25, 2013 (GLOBE NEWSWIRE) -- Prosensa Holding N.V. (NASDAQ: RNA) the Dutch biopharmaceutical company focusing on rare diseases with a high unmet medical need, and Newcastle University, UK, have announced the award of a second Framework Programme 7 (FP7) research grant from the European Commission. This new FP7 research grant totals approximately EUR 6 million to support the development of imaging biomarkers for Duchenne muscular dystrophy (DMD).

The project - "Developing imaging technologies for therapeutic interventions in rare diseases" - will be known as "BIOIMAGE-NMD" and is expected to run three and a half years.

"The overarching objective of the BIOIMAGE-NMD project is to deliver combined structural and molecular imaging biomarkers with proven utility for the detection of therapeutic effects in patients with rare neuromuscular diseases (NMD) including DMD," Professor Andrew Blamire, Professor of Magnetic Resonance Physics at the Newcastle Magnetic Resonance Centre at Newcastle University, said. "We hope to be able to show that using simultaneous MRI and magnetic resonance spectroscopic imaging (MRSI) is an effective tool to monitor biomarkers that indicate therapeutic efficacy of drugs tested in clinical trials in neuromuscular diseases, including DMD, to ultimately speed development and bring much needed therapies to patients in a more efficient manner."

The pan-European BIOIMAGE-NMD consortium includes leading expert centers in DMD already working together in another recently awarded FP7 project called SCOPE-DMD ( as well as additional partners with strong expertise and experience in bio-imaging. The SCOPE-DMD project, which was initiated earlier this year, was also awarded EUR 6 million as announced previously. This FP7 project aims to evaluate Prosensa's exon 45 skipping drug candidate, PRO045, in an innovatively designed clinical proof-of-concept study in boys with DMD.

The academic partners in the BIOIMAGE-NMD project are Newcastle University as the coordinating partner, and University College London (UCL) in the United Kingdom; the Institute of Myology in France; the Leiden University Medical Centre in The Netherlands; Universita Cattolica del Sacro Cuore (UCSC) in Italy; and the Katholieke Universiteit Leuven (KULeuven) in Belgium. These academic centers are working alongside a number of industrial partners including Prosensa, SCITO, based in France, and Consultants for Research in Imaging and Spectroscopy (CRIS).

Dr. Giles Campion, Chief Medical Officer & Senior Vice-President R&D at Prosensa, said, "In recent years it has been suggested that the development of imaging biomarkers for clinical trials should be an integral part of the drug development pathway. The BIOIMAGE-NMD project directly addresses this approach and is intended to provide new tools to assess treatments in early clinical trials for exon-skipping therapeutics."

BIOIMAGE-NMD will be conducting MRI scans in a number of exon skipping drug candidates, including PRO053, which is currently in Phase I/II clinical development. The compound induces exon 53 skipping in the dystrophin gene and could be suitable for up to 8% of all DMD patients.

Notes to editors

About Prosensa Holding N.V.

Prosensa (NASDAQ: RNA) is a Dutch biotechnology company engaged in the discovery and development of RNA-modulating therapeutics for the treatment of genetic disorders. Its primary focus is on rare neuromuscular and neurodegenerative disorders with a large unmet medical need, including Duchenne muscular dystrophy, myotonic dystrophy and Huntington's disease.

Prosensa's current portfolio includes six compounds for the treatment of DMD, all of which have received orphan drug status in the United States and the European Union. The compounds use an innovative technique called exon-skipping to provide a personalized medicine approach to treat different populations of DMD patients.

CONTACT: Celia Economides, Director IR & Corporate Communications Phone: +1 917 941 9059 Email:

Source:Prosensa Holding N.V.