Prosensa and Partners Awarded Second European Consortium FP7 Research Grant

BIOIMAGE-NMD project receives EUR 6M to devel­op bio­mark­ers to mon­i­tor effec­tive­ness of DMD treat­ments

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.

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