Wolfgang Miesbach Reports Promising Results for New Subcutaneous von Willebrand Disease Therapy VGA039
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared a post on LinkedIn:
“Promising results in new subcutaneous von Willebrand disease treatment presented at BIC 2025!
Carolyn Miller presented Phase I results for VGA039:
This monoclonal antibody targets Protein S to restore balance in blood clotting by inhibiting both tissue factor pathway inhibitor α (TFPIα) and activated protein C (aPC), enhancing thrombin generation during both initiation and propagation phases of coagulation.
The VIVID 2 study enrolled 10 VWD patients (ages 21-52) with diverse VWD subtypes, including those with mild hemophilia A. Results showed increased thrombin generation lasting 4+ weeks without dose-limiting toxicities:
Safe and well-tolerated across all VWD types (1, 2A, 2M, 2N, 3)
- Single subcutaneous injection maintained therapeutic concentrations for ~4 weeks
- Significant bleed reduction: 75-88% reduction in annualized bleeding rates in high-bleed patients
- Universal approach: Works across all VWD types by targeting Protein S
Unlike current VWD treatments requiring multiple weekly IV infusions, VGA039 may offer convenient subcutaneous dosing with sustained multi-week protection.”
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