Wednesday, April 24, 2013

GSK's Drisapersen reports positive Phase 2data in Duchenne muscular dystrophy

Drisapersen, designed to target exon 51 of the dystrophin gene in boys with Duchenne muscular who have certain dystrophin mutations, demonstrated improvement in walking ability when compared to placebo in a Phase 2 b study. The 53-person Phase 2b trial compared two drisapersen dosing regimens — "continuous" and "intermittent" — to placebo regimens. It was conducted in Australia, Belgium, Germany, Israel, Spain, Turkey and the United Kingdom between September 2010 and September 2012.

Participants were required to be boys with DMD, to be at least 5 years old, to be able to walk and to rise from the floor in seven seconds or less, and to have a dystrophin gene mutation that could potentially be helped by skipping exon 51, as well as meeting many other study criteria.
Boys with Duchenne MD who received weekly ("continuous") drisapersen treatment walked significantly farther in six minutes than boys in the placebo group after 24 and 48 weeks of treatment. At 24 weeks, the continuous treatment group showed a clinically meaningful and statistically significant difference of 35 meters (115 feet) from the placebo group on the 6MWT. In other words, the drisapersen-treated boys walked an average of 115 feet farther in six minutes than the boys given a placebo. The significant difference on the 6MWT between the continuous treatment group and the placebo group was maintained at 48 weeks, with the continuous treatment group walking an average of 35.8 meters (117 feet) farther than the placebo group at this second time point.

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