Prosensa and TREAT-NMD enter into strategic collaboration for PRO-051 clinical trial planning

Leiden, The Netherlands, August 31, 2009 – Prosensa, the Dutch based biopharmaceutical company focusing on RNA modulating therapeutics, announces the successful completion of a feasibility enquiry using the TREAT-NMD Global Database for DMD and the TREAT-NMD Care and Trial Sites Registry for the planning of the phase II/III study for its lead compound PRO-051.

Prosensa recently completed a phase I/II clinical trial for PRO-051, its lead compound for the treatment of Duchenne Muscular Dystrophy (DMD), and the company anticipates starting a phase II/III clinical study early next year. PRO-051 is directed to a specific mutation in the dystrophin gene that occurs in approximately 13% of the DMD patient population. In order to set up the pivotal study, patients with a genetic mutation amenable to exon skipping by PRO-051 need to be selected.

TREAT-NMD has developed a global database that contains precise genetic and clinical information from patients with DMD, including age, ambulation status and medication use. Using the TREAT-NMD Global Database for DMD, Prosensa has identified around 300 patients from 21 countries who meet the inclusion criteria for the upcoming trial. The TREAT-NMD database holds up-to-date information about all these patients that will allow them to be contacted for trial recruitment purposes through the national registries. These patients were matched to 50 potential trial sites and selected patients and sites will be contacted for participation in the upcoming clinical trial.

“Patient recruitment for clinical studies is a very expensive and time-consuming process, in particular for rare diseases such as DMD” comments Dr. Giles Campion, CMO and VP R&D of Prosensa. “Therefore, this successful collaboration with TREATNMD is extremely valuable for us as it facilitates the acceleration of the recruitment process and hopefully allows us to bring this treatment faster to patients.”

“We are delighted that our global patient database is providing such useful results for pharmaceutical companies like Prosensa,” said Professor Hanns Lochmüller, leader of the TREAT-NMD patient registries initiative. “Our approach is unique because the data we are giving companies isn’t just generic statistical information. It is up-to-date information about real patients who can be recruited into clinical trials through the registry or contacted when a treatment is available. At the same time, it’s safe for patients, since we don’t give out identifiable information to companies but act as a trusted intermediary and all our registries have full ethical approval and comply with data protection laws.”

Prosensa and TREAT-NMD will both be attending and presenting at the World Muscle Society Conference in Geneva from September 9th till 12th.

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