Prosensa Awarded EUR 6 mil­lion EU FP7 Research Grant

LEIDEN, The Netherlands – August 1, 2013 – Prosensa Holding N.V. (NASDAQ: RNA), the Dutch bio­phar­ma­ceu­ti­cal com­pa­ny focus­ing on RNA-mod­u­lat­ing ther­a­peu­tics for rare dis­eases with high unmet need, today announced that it has been award­ed a pres­ti­gious Framework Programme 7 (FP7) research grant from the European Union to sup­port the ongo­ing clin­i­cal study of the Company’s third nov­el devel­op­ment can­di­date, PRO045, for the treat­ment of Duchenne mus­cu­lar dys­tro­phy (DMD). The project, titled “Consortium for Products Across Europe in Duchenne Muscular Dystrophy” (SCOPE-DMD) is expect­ed to run for three years. Prosensa is part of a high-qual­i­ty, pan-European con­sor­tium that has been award­ed the grant of approx­i­mate­ly EUR 6 mil­lion to eval­u­ate its exon 45-skip­ping drug can­di­date, PRO045, in an inno­v­a­tive­ly designed clin­i­cal proof-of-con­cept study in boys with DMD.
The con­sor­tium includes lead­ing expert cen­ters in DMD such as Newcastle University in the United Kingdom, the coor­di­nat­ing part­ner with­in the project, the Institute of Myology in France and Leiden University Medical Center in the Netherlands, as well as an indus­tri­al part­ner, BioSpring in Germany. The com­bined expe­ri­ence and exper­tise of this con­sor­tium will help advance the devel­op­ment of PRO045 which entered clin­i­cal tri­als ear­li­er this year.
“We are delight­ed with this fund­ing, which will allow us to fur­ther advance and accel­er­ate the devel­op­ment of anoth­er exon-skip­ping com­pound. The Duchenne com­mu­ni­ty is eager­ly await­ing a swift devel­op­ment of prod­ucts which tar­get addi­tion­al exons after the progress with exon 51 skip­ping,” com­ment­ed Prof. Volker Straub, M.D., Professor of Neuromuscular Genetics and Harold Macmillan Chair of Medicine at Newcastle University’s Institute of Genetic Medicine. Professor Straub is also the for­mer coor­di­na­tor of TREAT-NMD, a key ini­tia­tive to cre­ate infra­struc­ture to ensure that the most promis­ing new ther­a­pies for neu­ro­mus­cu­lar dis­eases reach patients as quick­ly as pos­si­ble.
“FP7 grants are award­ed on the basis of a high­ly com­pet­i­tive, two-stage, peer-review process, there­fore this award serves as val­i­da­tion of our exon-skip­ping tech­nol­o­gy plat­form,” said Hans Schikan, CEO of Prosensa. “We very much look for­ward to work­ing with this esteemed con­sor­tium on PRO045 and are grate­ful to the EU for its sup­port of impor­tant clin­i­cal research ini­tia­tives that could have a sig­nif­i­cant impact on the health and lives of young boys diag­nosed with this rare and dev­as­tat­ing genet­ic dis­ease,” he added.
PRO045 is cur­rent­ly in a Phase I/IIa dose-esca­lat­ing safe­ty study to assess its safe­ty and effi­ca­cy. The com­pound induces exon 45 skip­ping in the dys­trophin gene and could be suit­able for approx­i­mate­ly 8% of all DMD patients. PRO045 has been grant­ed orphan drug sta­tus in the European Union and the United States.
FP7 is the EU’s main instru­ment for sup­port­ing inno­v­a­tive research in Europe, and has two main strate­gic objec­tives: to strength­en the sci­en­tif­ic and tech­no­log­i­cal base of European indus­try and to encour­age its inter­na­tion­al com­pet­i­tive­ness, while pro­mot­ing research that sup­ports EU poli­cies.

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