Drisapersen: Phase IIb data

The double-blind, international Phase IIb DMD114117 trial in 53 ambulant boys >5 years of age with DMD showed that continuous dosing with once-weekly subcutaneous 6 mg/kg drisapersen met the primary endpoint of improving 6MWD from baseline to week 24 vs. placebo, while intermittent dosing with subcutaneous 6 mg/kg drisapersen for 6 weeks of a 10-week cycle missed the endpoint. Specifically, continuous dosing with drisapersen led to a mean increase of 35.09 meters in 6MWD over placebo (p=0.014). Detailed 24-week data for the intermittent dosing regimen were not disclosed. On secondary endpoints, continuous dosing with drisapersen led to a mean increase of 35.84 meters in 6MWD at week 48 over placebo (p=0.051), while intermittent dosing with drisapersen led to a mean increase of 27.08 meters at week 48 over placebo (p=0.147). GlaxoSmithKline said there was "little change" in muscle strength at either time point for both drisapersen arms. Drisapersen was generally well tolerated with the majority of adverse events related to injection-site reactions and proteinuria. Data were presented at the RNA and Oligonucleotide Therapeutics meeting at Cold Spring Harbor. ...