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.
