Pfizer Initiates Phase 2 Study of PF-06252616 in Duchenne Muscular Dystrophy http://www.pfizer.com/news/press-re...of_pf_06252616_in_duchenne_muscular_dystrophy Pfizer Inc. (NYSEFE) announced today enrollment of the first patient in a multicenter Phase II clinical trial of the investigational compound PF-06252616 in boys with Duchenne muscular dystrophy (DMD), a genetic disorder characterized by progressive muscle degeneration and weakness. PF-06252616 is an experimental, infused, anti-myostatin monoclonal antibody. Myostatin is a naturally occurring protein in muscles that helps control muscle growth; it is believed that blocking the activity of myostatin may have potential therapeutic application in treating muscle wasting diseases such as DMD. The phase 2 clinical trial will evaluate the safety, tolerability and efficacy of PF-06252616 in boys aged 6 to <10 years old diagnosed with DMD regardless of genotype. Based on the proposed mechanism of action of PF-06252616, Pfizer is exploring whether there is the potential to increase muscle mass and function in boys with DMD who are weak and have lost muscle. PF-06252616 was granted Orphan Drug designation in July 2012 and Fast Track Designation in November 2012 by the U.S. Food and Drug Administration (FDA). The FDA’s Fast Track Designation is a process designed to facilitate the development and expedite the review of new drugs and biologics intended to treat or prevent serious conditions and that address an unmet medical need.ii The European Medicines Agency (EMA) granted the investigational candidate Orphan Medical Product designation in February 2013.