Prosensa Reports Initial Findings from the Further Clinical Data Analyses of Drisapersen for the Treatment of Duchenne Muscular Dystrophy

Corporate update pre­sen­ta­tion giv­en at the 32nd Annual J.P. Morgan Healthcare Conference in San Francisco on Thursday, January 16, 2014 at 10:00 AM Pacific Time

Leiden, The Netherlands, Jan. 16, 2014 (GLOBE NEWSWIRE) — Prosensa Holding N.V. (NASDAQ: RNA), the Dutch bio­phar­ma­ceu­ti­cal com­pa­ny focus­ing on RNA-mod­u­lat­ing ther­a­peu­tics for rare dis­eases with high unmet med­ical need, today announced ini­tial find­ings from fur­ther analy­ses from the aggre­gate data from the clin­i­cal devel­op­ment pro­gram of dris­apersen for the treat­ment of Duchenne Muscular Dystrophy (DMD).
“We are encour­aged by these results that sug­gest that treat­ing ear­li­er in the dis­ease and treat­ing longer shows a delay in the pro­gres­sion of the dis­ease,” said Hans Schikan, Prosensa’s Chief Executive Officer. “These data encour­age us to engage patient groups, clin­i­cal experts and reg­u­la­tors to explore a path for­ward for dris­apersen, which includes the pos­si­bil­i­ty of re-dos­ing.”
Schikan will be pre­sent­ing details of the fur­ther data analy­ses at the 32nd Annual J.P. Morgan Healthcare Conference in San Francisco on Thursday, January 16, 2014 at 10:00 AM Pacific Time. A live web­cast of the pre­sen­ta­tion can be accessed through the Investors & Media sec­tion of the Prosensa cor­po­rate web­site at and will be archived for 90 days.
With data from more than 300 patients Prosensa has the largest clin­i­cal data set in DMD. In order to advance the gen­er­al under­stand­ing of DMD, Prosensa has also announced that it will make cer­tain data from the dris­apersen clin­i­cal pro­gram avail­able to the sci­en­tif­ic com­mu­ni­ty. In addi­tion it has ini­ti­at­ed a study to bet­ter under­stand the nat­ur­al his­to­ry of DMD and is also explor­ing poten­tial bio­mark­ers that could be of use as indi­ca­tors of effi­ca­cy in DMD clin­i­cal tri­als.
The sub­set analy­ses focus on out­comes in DMD boys sev­en years or younger and those over sev­en years old as mea­sured by the six-minute walk test (6MWT). In all stud­ies, a treat­ment dif­fer­ence was seen in the younger patient pop­u­la­tion.
Notably, the pre­lim­i­nary analy­sis of the 96 week exten­sion data from those par­tic­i­pat­ing in the phase III DMD114044 (DEMAND III) study shows a 49 meter dif­fer­ence between those on con­tin­u­al treat­ment (n=52) and those who had been on place­bo for 48 weeks fol­lowed by active drug (n=31). Those pre­vi­ous­ly par­tic­i­pat­ing in the DMD114117 study (DEMAND II) showed a 52 meter dif­fer­ence at 96 weeks (n=13, n=17, respec­tive­ly).
Key safe­ty find­ings are con­sis­tent with pre­vi­ous obser­va­tions, includ­ing injec­tion site reac­tions, pro­tein­uria and mild to severe throm­bo­cy­tope­nia.
Schikan con­cludes, “We will con­tin­ue to work close­ly with patient groups, clin­i­cal experts and reg­u­la­tors to ensure that we leave no stone unturned to bring treat­ments to boys affect­ed by DMD.”

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