First Patient Dosed in Phase 3 of Study in Patients with Severe Hemophilia A

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

Sanofi and Sobi^TM announced the first patient has been dosed in the Phase 3, open-label, interventional study of BIVV001 (rFVIIIFc-VWF-XTEN), in patients with severe hemophilia A (XTEND-1 study; NCT04161495). Sobi and Sanofi are development partners for BIVV001 and the XTEND-1 study is designed to investigate the efficacy, safety and tolerability of prophylactic once-weekly dosing.
Factor VIII (FVIII) replacement therapy is a single therapy that can be used across treatment scenarios including, prophylaxis, acute bleed control and perioperative hemostasis. FVIII replacement products that offer extended bleed protection with once-weekly dosing intervals remain an unmet need for people living with severe hemophilia A.
Final results from the Phase 1 repeat dosing study of BIVV001 were recently presented at the 61st Annual Meeting of the American Society of Hematology in December in Orlando. The study evaluated the safety and pharmacokinetics of repeat dosing of BIVV001 and showed the potential to provide high sustained levels of factor VIII (FVIII) activity with once-weekly dosing. The study also indicated that BIVV001 was safe and well tolerated.
“Based on the final data of the Phase 1 repeat dose study, we are pleased to initiate Phase 3 development. BIVV001 has the potential to further advance the treatment of people with hemophilia A also by normalizing FVIII levels for part of the treatment interval, and we look forward to moving this important development program forward,” says Milan Zdravkovic, Head of R&D and Chief Medical Officer at Sobi.
Read the press release in its entirety here.