Anti-GDF8 antibody for treatment of Duchenne Muscular Dystrophy

Rare Disease Research Unit,  Pfizer Inc.

22nd January 2014

Pfizer Inc. submitted an application to TACT for review, which focused on Pfizer’s PF-06252616 experimental asset, a humanized monoclonal antibody directed against the myostatin protein (growth and differentiation factor 8, GDF8).  The asset is being considered by Pfizer for  potential future clinical trials in Duchenne muscular dystrophy (DMD). Preclinical data from Pfizer indicated that PF-06252616 effectively inhibits myostatin signalling in vitro assays and in animal models. Subsequent preclinical studies also indicated that PF-06252616 was observed to significantly increase total lean mass and muscle mass in a dose-dependent manner in wild-type mice and non-human primates. PF-06252616 has been submitted by Pfizer in an Investigational New Drug (IND) filing, inclusive of necessary pre-IND toxicity, pharmacokinetics (PK), formulation and other studies.  As part of Pfizer’s evaluation of potential indications for PF-06252616, Pfizer requested TACT review of Pfizer’s proposed Phase 1b/2 studies in DMD. Overall, TACT was enthusiastic about the maturity and breadth of the Pfizer program at this stage.

12 Apr 2017