Prosensa ini­ti­ates clin­i­cal devel­op­ment of PRO044 in an open label phase I/II study – Prosensa’s sec­ond clin­i­cal can­di­date for treat­ment of Duchenne Muscular Dystrophy

Leiden, March 30, 2010 – Prosensa, the Dutch based bio­phar­ma­ceu­ti­cal com­pa­ny focus­ing on RNA mod­u­lat­ing ther­a­peu­tics announces the start of a phase I/II clin­i­cal study for PRO044 in patients with Duchenne Muscular Dystrophy.

The objec­tive of this open label, dose-esca­lat­ing study is to eval­u­ate the safe­ty and tol­er­a­bil­i­ty of the sys­temic deliv­ery of PRO044 in twelve Duchenne Muscular Dystrophy (DMD) patients receiv­ing week­ly sub­cu­ta­neous injec­tions for a peri­od of five weeks. The effect of PRO044 will be assessed at the RNA lev­el, to demon­strate spe­cif­ic exon 44 skip­ping, and at the pro­tein lev­el, to demon­strate nov­el dys­trophin expres­sion in mus­cle biop­sies.

The first patients have been enrolled at the University Hospital Leuven in Belgium by prin­ci­pal inves­ti­ga­tor Dr. Nathalie Goemans, depart­ment of Pediatric Neurology. Prosensa also received approval from the reg­u­la­to­ry author­i­ties in Sweden, where patient screen­ing has been ini­ti­at­ed, and Italy. Participating cen­ters are the Queen Silvia Children’s Hospital in Gothenburg, Sweden and the University of Ferrara in Italy.

PRO044 is Prosensa’s sec­ond RNA based ther­a­peu­tic can­di­date for the treat­ment of DMD. Prosensa’s lead DMD com­pound, PRO051/GSK2402968, is close to enter­ing phase III clin­i­cal devel­op­ment and was recent­ly part­nered with GlaxoSmithKline (GSK). GSK also has an exclu­sive option on PRO044, which will be trig­gered upon suc­cess­ful com­ple­tion of this phase I/II study.

“We are delight­ed to advance a sec­ond promis­ing can­di­date for the treat­ment of DMD into clin­i­cal devel­op­ment. With the addi­tion of PRO044 to our drug can­di­date port­fo­lio, we will be able to address a broad­er patient pop­u­la­tion upon suc­cess­ful com­ple­tion of these clin­i­cal devel­op­ment pro­grams.” com­ment­ed Dr. Giles Campion, Chief Medical Officer of Prosensa. “This impor­tant clin­i­cal devel­op­ment step con­firms Prosensa’s com­mit­ment to the treat­ment of neu­ro­mus­cu­lar dis­or­ders.”

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