Drisapersen fails Phase III for Duchenne
GlaxoSmithKline (GSK) and Prosensa announced that the 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.
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