Hereditary/Acquired Bleeding Disorders
First-in-Human Results Show Promise With BT200, a New Anti-von Willebrand Factor Agent
At the European Hematology Association (EHA) 2021 Virtual Congress in June, researchers reported phase 1 efficacy and safety findings on BT200 (abstract S302), an aptamer that binds to the A1 domain of VWF, inhibiting its binding to platelet glycoprotein Ib. (Aptamers are single-stranded RNA or DNA molecules that bind to extracellular targets.) Pharmacodynamic analysis suggested a dose-dependent inhibition of VWF activity with BT200, which was administered intravenously or subcutaneously. Combination of BT200 with desmopressin had an apparently additive effect on factor VIII levels. The BT200 half-life was 7 to 12 days with SC administration, “supporting once-weekly treatment,” said study presenter Katerina Kovecevic, PhD, who noted that the increased factor VIII levels observed led to a phase 2 trial of BT200 now underway in patients with hemophilia A and von Willebrand disease type IIb.
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