BioCentury
ARTICLE | Clinical News

Drisapersen: Additional Phase III data

December 23, 2013 8:00 AM UTC

GlaxoSmithKline published additional data on its clinical register from the double-blind, international Phase III DEMAND 3 (DMD114044) trial evaluating continuous dosing with once-weekly subcutaneous 6 mg/kg drisapersen in 186 ambulant boys >=5 years of age with DMD. On the secondary endpoint of 10-meter walk/run test, continuous dosing with drisapersen led to a mean reduction in velocity from baseline to week 48 of 0.21 meters/second vs. a mean reduction of 0.2 meters/second for placebo. On the 4-stair climb, drisapersen led to a mean reduction in ascent velocity from baseline to week 48 of 0.14 stairs/second vs. a mean reduction of 0.12 stairs/second for placebo. Drisapersen also led to a mean reduction in NSAA score from baseline to week 48 of 7.2 points vs. a mean reduction of 6.7 points for placebo.

In September, GSK and Prosensa reported that drisapersen missed the primary endpoint of improving 6MWD from baseline to week 48 vs. placebo. Specifically, continuous dosing with drisapersen led to a mean reduction of about 43 meters in 6MWD vs. a mean reduction of about 53 meters for placebo (placebo-adjusted difference of 10.33 meters, p=0.415). Drisapersen also missed the secondary endpoints of improving 10-meter walk/run test, 4-stair climb and NSAA vs. placebo. At the time, Prosensa said data for measurements of dystrophin levels have not yet been disclosed as analysis is ongoing. Additionally, GSK said it would place a hold on all dosing of drisapersen in the ongoing Phase III DMD114349, DMD115501 and DMD114673 extension trials (see BioCentury, Sept. 23). According to Prosensa, GSK said in a Dec. 19 update to patient groups and investigators that data analysis and assessment of next steps for drisapersen are still ongoing. Prosensa said the results of the evaluation are expected early next year. ...