Prosensa And Partners Awarded Second European Consortium FP7 Research Grant

Leiden, The Netherlands and Newcastle, UK, Nov. 25, 2013 (GLOBE NEWSWIRE) -- Prosensa Holding N.V. (NASDAQ: RNA) the Dutchbiopharmaceutical company focusing on rare diseases with a highunmet medical need, and Newcastle University, UK, have announcedthe award of a second Framework Programme 7 (FP7) research grantfrom the European Commission. This new FP7 research grant totalsapproximately EUR 6 million to support the development of imagingbiomarkers for Duchenne muscular dystrophy (DMD).

The project - "Developing imaging technologiesfor 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-NMDproject is to deliver combined structural and molecular imagingbiomarkers with proven utility for the detection of therapeuticeffects in patients with rare neuromuscular diseases (NMD)including DMD," Professor Andrew Blamire, Professor of MagneticResonance Physics at the Newcastle Magnetic Resonance Centre atNewcastle University, said. "We hope to be able to show that usingsimultaneous MRI and magnetic resonance spectroscopic imaging(MRSI) is an effective tool to monitor biomarkers that indicatetherapeutic efficacy of drugs tested in clinical trials inneuromuscular diseases, including DMD, to ultimately speeddevelopment and bring much needed therapies to patients in a moreefficient manner."

The pan-European BIOIMAGE-NMD consortiumincludes leading expert centers in DMD already working together inanother recently awarded FP7 project called SCOPE-DMD ( as well asadditional partners with strong expertise and experience inbio-imaging. The SCOPE-DMD project, which was initiated earlierthis year, was also awarded EUR 6 million as announced previously.This FP7 project aims to evaluate Prosensa's exon 45 skipping drugcandidate, PRO045, in an innovatively designed clinicalproof-of-concept study in boys with DMD.

The academic partners in the BIOIMAGE-NMDproject are Newcastle University  as the coordinating partner,and University College London (UCL) in the United Kingdom; theInstitute of Myology in France; the Leiden University MedicalCentre 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 numberof 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 yearsit has been suggested that the development of imaging biomarkersfor clinical trials should be an integral part of the drugdevelopment pathway. The BIOIMAGE-NMD project directly addressesthis approach and is intended to provide new tools to assesstreatments in early clinical trials for exon-skippingtherapeutics."

BIOIMAGE-NMD will be conducting MRI scans in anumber of exon skipping drug candidates, including PRO053, which iscurrently in Phase I/II clinical development. The compound inducesexon 53 skipping in the dystrophin gene and could be suitable forup to 8% of all DMD patients.

Notes to editors

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.

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