GlaxoSmithKline (GSK) and Prosensa today announced that the first patient has commenced treatment in the Phase III clinical study investigating GSK2402968, in ambulant boys with Duchenne Muscular Dystrophy (DMD), who have a dystrophin gene mutation amenable to an exon 51 skip (up to 13% of boys with DMD). Commencement of this study confirms previously announced plans to progress this asset into Phase III.
“Currently, there is no approved treatment to alter the course of DMD - a disease that puts boys in wheelchairs and often leads to death in early adulthood.”
This randomised, placebo controlled study will enrol 180 patients, from up to 18 countries, and is currently the most advanced ongoing study for this rare, severely debilitating, neuromuscular disease.
The study is designed to assess the efficacy and safety of GSK2402968 6mg/kg, once weekly, for 48 weeks in ambulant boys over 5 years of age with DMD, compared to placebo. The primary efficacy endpoint is a measure of muscle function using the six minute walking distance (6MWD) test.
"The commencement of this Phase III study is an important milestone," said Dr Philippe Monteyne, Head of Development and Chief Medical Officer for GSK Rare Diseases. "Currently, there is no approved treatment to alter the course of DMD - a disease that puts boys in wheelchairs and often leads to death in early adulthood."
"We are very pleased with this achievement. It is another step forward in our joint fight against Duchenne," said Dr Giles Campion, Chief Medical Officer of Prosensa. "If the results of this study are positive, we hope it will lead to an approved treatment option for the thousands of young people worldwide living with this devastating disease."