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Begbroke Science Park > News and Press > GSK and Prosensa Start DMD Phase III Study
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GSK and Prosensa Start DMD Phase III Study

GlaxoSmithKline (GSK) and Prosensa have said that the first patient has commenced treatment in the Phase III clinical study investigating GSK2402968 (`968), in ambulant boys with Duchenne Muscular Dystrophy (DMD), who have a dystrophin gene mutation amenable to an exon 51 skip (up to 13 percent of boys with DMD).

Commencement of this study confirms previously revealed plans to progress this asset into Phase III.

According to GSK, 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 GSK968 6mg/kg, once weekly, compared to placebo, for 48 weeks in ambulant boys over five years of age with DMD The primary efficacy endpoint is a measure of muscle function using the six minute walking distance test.

GSK968, an antisense oligonucleotide, which induces exon skipping of exon 51, is currently in late stage development for DMD. It has been designated orphan drug status in the EU and US, and is being developed as part of an alliance between GlaxoSmithKline and Prosensa.

DMD is a severely debilitating childhood neuromuscular disease that affects one in 3,500 live male births. This rare disease is caused by mutations in the dystrophin gene, resulting in the absence or defect of the dystrophin protein.

The dystrophin gene is the largest gene in the body, consisting of 79 exons. Exons are small sequences of genetic code which lead to the manufacture of sections of protein. In DMD, when certain exons are mutated/deleted, the RNA cannot read past the fault. This prevents the rest of the exons being read, resulting in a non-functional dystrophin protein and the severe symptoms of DMD.

Philippe Monteyne, head of development and chief medical officer for rare diseases at GSK, said: “The commencement of this Phase III study is an important milestone. 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.”

Giles Campion, chief medical officer of Prosensa, said: “We are very pleased with this achievement. It is another step forward in our joint fight against Duchenne. 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.”

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Ben Roeves