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ARTICLE | Clinical News

GSK reports mixed Phase IIb data for drisapersen for DMD

April 11, 2013 12:34 AM UTC

GlaxoSmithKline plc (LSE:GSK; NYSE:GSK) said continuous dosing with once-weekly subcutaneous drisapersen met the primary endpoint of improving six-minute walk distance (6MWD) from baseline to week 24 vs. placebo in the Phase IIb DMD114117 trial to treat Duchenne muscular dystrophy (DMD). However, intermittent dosing of drisapersen for six weeks of a 10-week cycle missed the endpoint. Continuous dosing with drisapersen led to a mean increase of 35.09 meters in 6MWD over placebo (p=0.014), while data for the intermittent dosing regimen were not disclosed. The double-blind, international trial enrolled 53 ambulant boys over five years of age with DMD. Data are slated to be presented on Thursday at the RNA and Oligonucleotide Therapeutics meeting in Cold Spring Harbor.

Data from a Phase III trial of drisapersen to treat DMD in 180 ambulant boys over five years of age are expected this year. The primary endpoint of the trial is muscle function as measured by the 6MWD test. GSK has exclusive, worldwide rights to develop and commercialize drisapersen, an antisense oligoribonucleotide that induces exon 51 skipping on the dystrophin gene, from Prosensa B.V. (Leiden, the Netherlands) under a 2009 deal. ...