Pfizer Begins Duchenne Muscular Dystrophy Drug Phase 2 Trial

Pfizer announced that its Phase 2 clinical trial for the investigational compound PF-06252616 in boys with Duchenne muscular dystrophy (DMD) has kicked off with the enrollment of the first patient.

PF-06252616 is an investigational, infused, monoclonal antibody that targets myostatin — a protein that naturally occurs in muscles to help control muscle growth. Blocking the activity of the protein is considered to have potential therapeutic effect in treating DMD and other muscle wasting diseases. The drug was granted both Orphan Drug Designation and Fast Track status by the U.S. Food and Drug Administration (FDA) in 2012. PF-06252616 has also received Orphan Medical Product designation from the European Medicines Agency (EMA) in February last year.

The mid-stage clinical trial will investigate the safety, efficacy, and tolerability of PF-06252616 male patients aged 6 through 10 years old diagnosed with DMD, regardless of their disease’s genotype. The company said it is exploring the possibility of the drug increasing muscle mass and improving function in boys with DMD who have lost some degree of mass and function.

“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,” said Dr. Kevin Lee, SVP and CSO of Pfizer’s Rare Disease Research Unit.

“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,” commented Dr. Sharon Hesterlee, VP of research for Parent Project Muscular Dystrophy (PPMD).

Other drug makers have made progress with their investigational treatments for Duchenne muscular dystrophy this year. Biotech firm Prosensa filed its New Drug Application (NDA) for drisapersen with the FDA in October, while Sarepta Therapeutics initiated a clinical study last month for patients who have advanced DMD.