Prosensa Initiates First Patient Dosing in Phase I/II tri­al of PRO053

Leiden, The Netherlands, Sept. 6, 2013 (GLOBE NEWSWIRE) — Prosensa Holding N.V. (NASDAQ: RNA), the Dutch bio­phar­ma­ceu­ti­cal com­pa­ny focus­ing on RNA-mod­u­lat­ing ther­a­peu­tics for rare dis­eases with high unmet need, today announced the dos­ing of the first patient in its Phase I/II clin­i­cal tri­al of PRO053, an exon-skip­ping com­pound for the treat­ment of Duchenne mus­cu­lar dys­tro­phy (DMD).
“We are pleased that our fourth exon-skip­ping com­pound, PRO053, has advanced into clin­i­cal tri­als.” Giles Campion, Prosensa’s Chief Medical Officer, said. “Bringing new drug can­di­dates, such as PRO053, into the clin­ic exem­pli­fies how we can lever­age our sci­en­tif­ic and reg­u­la­to­ry exper­tise gleaned from the expe­ri­ence in advanc­ing the inves­ti­ga­tion­al devel­op­ment com­pound, dris­apersen, through the clin­ic. Most impor­tant­ly, a broad­er clin­i­cal port­fo­lio may offer addi­tion­al patients with sub­types of DMD a much-need­ed poten­tial treat­ment option.”
PRO053, the company’s fourth drug devel­op­ment can­di­date, induces exon 53 skip­ping and may be applic­a­ble to approx­i­mate­ly 8% of all DMD patients.
This is an open-label study designed to assess the safe­ty, effi­ca­cy, tol­er­a­bil­i­ty and phar­ma­co­ki­net­ics of mul­ti­ple dos­es of PRO053 in a sub­set of patients with DMD. The pri­ma­ry out­come mea­sure of the study will be change from base­line in the six-minute walk test (6MWT) after 48 weeks of treat­ment. A num­ber of sec­ondary out­come mea­sures will also be assessed, includ­ing safe­ty, mus­cle func­tion and strength, and func­tion­al out­come based on a ques­tion­naire. The ini­tial dose-find­ing por­tion of the study will be con­duct­ed at sev­er­al clin­i­cal tri­al sites in Europe. The study may then be extend­ed to addi­tion­al ter­ri­to­ries in and out­side of Europe.
Prosensa’s cur­rent port­fo­lio includes six com­pounds for the treat­ment of DMD, all of which have received orphan drug sta­tus in the United States and the European Union. The com­pounds use an inno­v­a­tive tech­nique called exon-skip­ping to pro­vide a per­son­al­ized med­i­cine approach to treat dif­fer­ent pop­u­la­tions of DMD patients. The company’s lead inves­ti­ga­tion­al drug can­di­date, dris­apersen, which was exclu­sive­ly licensed to GlaxoSmithKline, is in Phase III clin­i­cal tri­als.

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