Pfizer enters race to develop Duchenne muscular dystrophy therapy

US pharma giant Pfizer (NYSE: PFE) is the latest company to enter the race to develop a therapy for Duchenne muscular dystrophy (DMD).

Pfizer has enrolled its first patient into a Phase II trial for PF-06252616, an anti-myostatin monoclonal antibody being developed as a potential treatment for DMD, notes a new report from research and consulting firm GlobalData. The drug blocks myostatin and aims to increase muscle content to compensate for dystrophy and muscle weakness. The trial will evaluate the efficacy, tolerability and safety profile of the drug in boys aged between six and nine years old, with DMD, regardless of genotype.

Pfizer’s approach – suppressing myostatin – differs from other drugs in the late-stage pipeline, including Translarna (ataluren), being developed by PTC Therapeutics (Nasdaq: PTCT), and Sarepta’s (Nasdaq: SRPT) eteplirsen. These aim to bypass specific mutations, such as nonsense mutations and exon deletions, and restore dystrophin production in muscles.