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.
