GSK and Prosensa Announce Primary Endpoint Not Met in Phase III Study of Drisapersen in Patients With Duchenne Muscular Dystrophy

LONDON and LEIDEN, Netherlands, Sept. 20, 2013 (GLOBE NEWSWIRE) — GlaxoSmithKline (GSK) and Prosensa today announced that GSK’s Phase III clin­i­cal study of dris­apersen, an inves­ti­ga­tion­al anti­sense oligonu­cleotide, for the treat­ment of Duchenne Muscular Dystrophy (DMD) patients with an amenable muta­tion, did not meet the pri­ma­ry end­point of a sta­tis­ti­cal­ly sig­nif­i­cant improve­ment in the 6 Minute Walking Distance (6MWD) test com­pared to place­bo.
A total of 186 boys were ran­domised to this dou­ble-blind, place­bo-con­trolled study (DMD114044) and received dris­apersen at a dose of 6mg/kg/week (N=125) or place­bo (N=61) via sub­cu­ta­neous injec­tion over 48 weeks. The dif­fer­ence in 6MWD (mean (CI) 10.33m (-14.65, 35.31), p=0.415) between dris­apersen and place­bo groups did not reach sta­tis­ti­cal sig­nif­i­cance. There was no treat­ment dif­fer­ence in key sec­ondary assess­ments of motor func­tion: 10-meter walk/run test, 4-stair climb and North Star Ambulatory Assessment. The most com­mon­ly report­ed adverse events includ­ed injec­tion site reac­tions (78% for dris­apersen vs 16% for place­bo) and renal adverse events (includ­ing sub­clin­i­cal pro­tein­uria; 46% for dris­apersen vs 25% for place­bo). No patients had throm­bo­cy­tope­nia.
Full eval­u­a­tion of the ben­e­fit-to-risk pro­file of dris­apersen treat­ment across all stud­ies is antic­i­pat­ed to be com­plet­ed by year end. This may include analy­ses of pooled results from var­i­ous dris­apersen stud­ies.
“We appre­ci­ate that these results will be dis­ap­point­ing for boys with DMD and their fam­i­lies. We would like to sin­cere­ly thank all those who par­tic­i­pat­ed in the study for their com­mit­ment,” com­ment­ed Carlo Russo, Senior Vice President, Head of GSK Rare Diseases Research & Development. “We are com­mit­ted to eval­u­at­ing the out­come of this study in the con­text of the over­all devel­op­ment pro­gramme with experts in the field, and we expect such eval­u­a­tion to help inform our next steps for dris­apersen. It is our hope that progress will be made in an effort to help boys with DMD.”
“While we are dis­ap­point­ed that this study did not meet its pri­ma­ry end­point, we remain com­mit­ted to the over­all pro­gramme and will con­tin­ue to work close­ly with GSK,” said Hans Schikan, Chief Executive Officer of Prosensa. “With no long term dis­ease mod­i­fy­ing ther­a­pies avail­able for DMD patients, research and devel­op­ment of pos­si­ble treat­ment options is of crit­i­cal impor­tance for boys and their fam­i­lies affect­ed by this debil­i­tat­ing dis­ease.”
Results have been sub­mit­ted for pre­sen­ta­tion at forth­com­ing sci­en­tif­ic meet­ings and will also be sub­mit­ted for pub­li­ca­tion in a sci­en­tif­ic peer-reviewed jour­nal.
Drisapersen is not approved or licensed for use any­where in the world.

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