Pfizer has enrolled the first patient in its multicentre Phase II clinical trial of PF-06252616, an experimental, infused, anti-myostatin monoclonal antibody, to treat boys with Duchenne muscular dystrophy (DMD).
The Phase II trial is designed to evaluate the safety, tolerability and efficacy of PF-06252616 in boys aged six to less than ten years old diagnosed with DMD regardless of genotype.
DMD is a genetic disorder characterised by progressive muscle degeneration and weakness.
Myostatin is a naturally occurring protein that helps control muscle growth and is believed that blocking its activity may have potential therapeutic application in treating muscle wasting diseases such as DMD.
Pfizer Rare Disease Research Unit senior vice-president and chief scientific officer Kevin Lee said: "DMD is a devastating and debilitating disease impacting approximately 1 in 3,500 male births worldwide with no current treatment options.
"We are pleased to be taking this important next step in the development of PF-06252616 as an investigational therapy for DMD in the hopes of potentially bringing a much-needed therapy to individuals and families with this devastating disease."
The company said that based on the proposed mechanism of action of PF-06252616, it is exploring whether there is potential to increase muscle mass and function in boys with DMD who are weak and have lost muscle.
Muscular Dystrophy Association vice-president of Research for Parent Project Muscular Dystrophy (PPMD) Sharon Hesterlee said: "We are enthusiastic about the potential for myostatin inhibitors to stimulate increases in muscle mass and strength for people living with Duchenne muscular dystrophy.
"This approach could potentially add an important angle in our fight against this disease and we are pleased to see the time and great care that Pfizer has expended on its development.
"Our Duchenne community is proud to have the extraordinary passion and expertise of the Pfizer team fighting with us."
The US Food and Drug Administration (FDA) has granted orphan drug designation for PF-06252616 in July 2012 and fast track designation in November the same year.