Note: The below is an edited version of an article originally published on StreetInsider.com. The original release can be read in it’s entirety here.
Alnylam Pharmaceuticals, Inc. announced today new positive results from its ongoing Phase 1 study with Fitusiran, an investigational RNAi therapeutic targeting antithrombin for the treatment of hemophilia A and B and rare bleeding disorders. Fitusiran is designed to lower levels of antithrombin with the goal of promoting sufficient thrombin generation to prevent bleeding in patients with hemophilia and other rare bleeding disorders. New clinical data showed that once-monthly subcutaneous administration of Fitusiran achieved dose-dependent lowering of antithrombin and increases in thrombin generation, resulting in a median estimated annualized bleeding rate of zero in evaluable patients with hemophilia A or B without inhibitors.