Pfizer has kicked off a Phase II study assessing its investigational compound PF-06252616 in Duchenne muscular dystrophy (DMD).
The trial is looking at the safety, tolerability and efficacy of the drug in boys aged six up to 10 years old with DMD (any genotype), to gage whether it could potentially boost muscle mass and function.
DMD is a genetic disorder characterised by progressive muscle degeneration and weakness. The disease affects around one in 3,500 male births worldwide, but as yet there are no treatment options for patients.
Pfizer’s drug is an anti-myostatin monoclonal antibody. It is hoped that by blocking myostatin - a naturally occurring protein in muscles that helps control muscle growth - this class of medicines could be used to treat muscle wasting diseases such as DMD, the firm noted.
“We are enthusiastic about the potential for myostatin inhibitors to stimulate increases in muscle mass and strength for people living with Duchenne muscular dystrophy,” said Sharon Hesterlee, research lead for Parent Project Muscular Dystrophy (PPMD), noting that the approach “could potentially add an important angle in our fight against this disease”.
PF-06252616 was granted Orphan Drug designation in July 2012 and Fast Track Designation in November 2012 in the US. The European Medicines Agency assigned the drug with an Orphan Medical Product designation in February 2013.