Pfizer begins Phase II trial of PF-06252616 to treat duchenne muscular dystrophy

The 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 genetic disorder characterized by progressive muscle degeneration and weakness.

Myostatin is a naturally occurring protein that helps control muscle growth and it 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 worldwidei 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."

Based on the proposed mechanism of action of PF-06252616, the company is exploring whether there is the potential to increase muscle mass and function in boys with DMD who are weak and have lost muscle.

Muscular Dystrophy Association Research for Parent Project Muscular Dystrophy (PPMD) vice-president Sharon Hesterlee said: "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) granted orphan drug designation for PF-06252616 in July 2012 and fast track designation in November 2012.

Image: Pfizer world headquarters. Photo: courtesy of Jim.henderson.