Alnylam & Sanofi Genzyme Report Positive Results from Ongoing Phase 2 Study with Investigational RNAi Therapeutic Fitusiran in Patients with Hemophilia A and B With or Without Inhibitors

Note: The following is an edited version of a press release from Alnylam. Read the press release from Alnylam in it’s entirety here.

Alnylam Pharmaceuticals, Inc. and Sanofi Genzyme, announced today new positive results from the ongoing Phase 2 open-label extension study with fitusiran in patients with hemophilia A and B, with or without inhibitors. These results were presented today in an oral presentation at the International Society on Thrombosis and Haemostasis (ISTH) 2017 Congress, being held from July 8 – 13, 2017 in Berlin, Germany. Fitusiran is an investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of patients with hemophilia A and B, that is designed to lower levels of AT with the goal of promoting sufficient thrombin generation upon activation of the clotting cascade to restore hemostasis and prevent bleeding. The companies also announced that Phase 1 clinical trial results demonstrating an encouraging preliminary safety and tolerability profile and initial evidence that monthly subcutaneously administered fitusiran lowered AT levels and increased thrombin generation in patients with hemophilia A and B without inhibitors were published online today and will appear in the September 7, 2017, print issue of The New England Journal of Medicine (NEJM).