Prosensa Comments on Drisapersen Program Update

Leiden, The Netherlands, Dec. 19, 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, com­ment­ed on a recent update by GlaxoSmithKline (GSK) to patient groups and inves­ti­ga­tors regard­ing the ongo­ing analy­ses for dris­apersen, an inves­ti­ga­tion­al anti­sense oligonu­cleotide, for the treat­ment of Duchenne Muscular Dystrophy (DMD) patients with an amenable muta­tion, which is exclu­sive­ly licensed to GSK.
On September 20, GSK and Prosensa announced that results of a Phase III study (DMD114044) of dris­apersen in boys with DMD did not meet the pri­ma­ry end­point. At that time, togeth­er with Prosensa, GSK began to eval­u­ate the results in the con­text of the over­all clin­i­cal pro­gram in addi­tion to per­form­ing addi­tion­al analy­ses to ful­ly under­stand the results of this study.
Earlier today, GSK pro­vid­ed an update to patient groups and inves­ti­ga­tors that the analy­sis of the results and assess­ment of next steps are still ongo­ing. The out­come of this eval­u­a­tion is antic­i­pat­ed in ear­ly 2014. In addi­tion, as pub­licly announced at the recent PPMD Duchenne Policy Forum, the US Food and Drug Administration is cur­rent­ly look­ing at the dris­apersen data, in part to under­stand clin­i­cal and bio­mark­er end­points.

In the mean­time, GSK has stat­ed that as per their pol­i­cy, the sum­ma­ry results of the DMD114044 study will be post­ed on GSK’s Clinical Study Register.
Prosensa’s Chief Executive Officer, Hans Schikan, com­ments, “Given the dev­as­tat­ing impact of DMD on boys and their fam­i­lies, it is of crit­i­cal impor­tance that this robust dataset is under­stood in its entire­ty and no stone is left unturned.” Schikan con­tin­ued, “The dris­apersen pro­gram rep­re­sents one of the largest datasets in this dis­ease to date, and we con­tin­ue to col­lab­o­rate with patient groups and oth­er key stake­hold­ers to help patients with DMD.”

About dris­apersen and the clin­i­cal devel­op­ment pro­gram
Drisapersen, (pre­vi­ous­ly GSK2402968/PRO051) an anti­sense oligonu­cleotide, which induces exon skip­ping of exon 51, is cur­rent­ly in late stage devel­op­ment for DMD.
GSK obtained an exclu­sive world­wide license to devel­op and com­mer­cial­ize dris­apersen from Prosensa in 2009. Drisapersen has been des­ig­nat­ed orphan drug sta­tus in the EU, US and Japan. In June 2013, dris­apersen was grant­ed Breakthrough Therapy des­ig­na­tion by the US Food and Drug Administration.
The over­all clin­i­cal pro­gram includes two open label exten­sion stud­ies, DMD114673 and DMD114349, as well as three dou­ble blind, place­bo con­trolled stud­ies, DMD114117, DMD114876 and DMD114044. For more infor­ma­tion regard­ing the clin­i­cal stud­ies involv­ing dris­apersen vis­it www.clinicaltrials.gov.

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