GSK and Prosensa announce start of Phase III study of inves­ti­ga­tion­al Duchenne Muscular Dystrophy med­ica­tion

Leiden, January 19, 2011 – 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.

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.”

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