Bioverativ Data Presented at ASH Underscore Potential for Once Weekly Dosing with Sustained High Factor Levels in Hemophilia A

The following is an excerpt from a press release from Bioverativ. Read the press release in its entirety here.

Bioverativ Inc., a Sanofi company dedicated to transforming the lives of people with rare blood disorders, presented new data from the EXTEN-A Phase 1/2a trial of BIVV001 showing that a single 65 IU/kg dose of BIVV001 extended the half-life of factor VIII to an unprecedented 44 hours with high factor activity levels and was generally well tolerated. The data were presented in an oral session at the 60th Annual Meeting of the American Society of Hematology.
BIVV001 is a novel and investigational von Willebrand factor-independent factor VIII therapy for people with hemophilia A that has the potential to provide protection in all treatment scenarios. Factor replacement therapy is the cornerstone of effective treatment of hemophilia A, as it naturally provides what is missing in the body (clotting factor VIII), and it has a consistent and well-characterized safety and efficacy profile.
“Longer prophylactic dosing intervals that offer maximum overall protection from bleeds are still an unmet need for people with severe hemophilia A,” said Barbara A. Konkle, M.D., Associate Chief Scientific Officer, Bloodworks Northwest and Professor of Medicine/Hematology, University of Washington, who presented the data at ASH today. “In these initial results, a single 65 IU/kg dose of BIVV001 resulted in a much greater half-life than traditional recombinant factor therapy, achieving an average factor activity level of 18.5 percent at seven days post infusion.”
Factor activity levels refer to the amount of factor VIII in a person’s blood, and they determine a person’s symptoms. Participants in the EXTEN-A trial have severe hemophilia A (<1 percent). Moderate hemophilia A is characterized by factor levels of 1-5 percent, and mild hemophilia A is from 6-49 percent.ⁱ
“We are very encouraged by these results, which suggest that BIVV001 has the potential to markedly improve the treatment paradigm for patients and physicians, and we look forward to learning more as the trial continues,” said Tim Harris, PhD., D.Sc., Executive Vice President of Research and Development at Bioverativ.
Read the press release in its entirety here.