24 May 2018 - - Waltham, Massachusetts-based blood disorders therapeutics developer Bioverativ Inc. (NASDAQ: BIVV), a Sanofi company, has presented initial clinical data for BIVV001 (rFVIIIFc-VWF-XTEN), a von Willebrand factor -independent factor VIII therapy for people with hemophilia A, the company said.
Preliminary safety and pharmacokinetic data from the ongoing EXTEN-A Phase 1/2a study showed that a single low dose of BIVV001 extended the half-life of factor VIII to 37 hours with high factor activity levels, and was generally well tolerated.
BIVV001 is an investigational factor VIII therapy that has the potential to provide comprehensive protection in all treatment scenarios including management of acute bleeds, perioperative care, emergency situations, and prophylactic use. The role of factor also goes beyond the coagulation cascade and may have a role to play in joint and bone health of people with hemophilia.
EXTEN-A is an ongoing Phase 1/2a, open-label, multicenter study to evaluate the safety and pharmacokinetic of BIVV001 in both a low-dose and high-dose cohort of subjects aged 18-65 years with severe hemophilia A. Primary endpoints include occurrence of adverse events and development of inhibitors. Secondary endpoints related to pharmacokinetic parameters were also presented.
Bioverativ is a global biotechnology company dedicated to research, development, and commercialization of innovative therapies for hemophilia and other rare blood disorders.