Prosensa announces the start of an international multi-center phase I/II clinical study with ‘smart drug’ PRO051 in patients with Duchenne Muscular Dystrophy

Leiden, The Netherlands, May 5, 2008 – Prosensa announces that it has started a phase I/II study to explore the effect, safety and tolerability of systemic injections of PRO051 in Duchenne Muscular Dystrophy (DMD) patients. This trial is performed in collaboration with UZ Leuven (Belgium), the Queen Silvia Children’s hospital (Sweden) and the LUMC (the Netherlands). The UZ Leuven and the Queen Silvia Children’s hospital have already started to enroll patients. In this study, an important parameter will be the presence of dystrophin in muscle biopsies, the protein missing in patients with Duchenne Muscular Dystrophy (DMD). This clinical trial in patients with DMD uses an antisense oligoribonucleotide, a ‘smart drug’ removing an unwanted segment of the faulty DMD gene product, and represents a novel approach to combat genetic diseases like DMD.

Dr. Nathalie Goemans, the coordinating and principal investigator in Leuven says: “I am excited that, based on the encouraging proof of concept data from the previous clinical study, we can now proceed with this next important step in the investigations that are required to determine whether this highly promising approach can be developed into an effective and safe treatment for patients with this devastating disease. “

"We are proud that quickly after completion and publication of the first trial we are able to conduct this study. We expect to provide further information on the developments later this year. In this study, we extend our recent success proving the concept of local dystrophin production to a study with systemic application to achieve widespread dystrophin expression in muscles. ", says Gerard Platenburg, Prosensa's CEO.

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