Subcutaneous, every-four-week maintenance dosing of a novel protein S antibody is well-tolerated and substantially reduces bleeding rates: Results from A phase 1/2 multidose study of VGA039 in patients with von Willebrand disease
A Phase 1/2 study reveals VGA039 significantly improves hemostasis in adolescents and adults with von Willebrand Disease, indicating a favorable safety profile.
Key Points
To evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of subcutaneous VGA039 in patients with von Willebrand Disease.
Open-label phase 1/2 study conducted in adolescents and adults with von Willebrand Disease.
Patients received subcutaneous VGA039 with a single loading dose followed by maintenance doses every four weeks.
Safety, pharmacokinetics, and pharmacodynamics were measured throughout the treatment period.
VGA039 resulted in a substantial 74% reduction in bleeding rates over 148 days compared to prior prophylaxis.
All patients reported no significant drug-related adverse events except for mild headache in one patient.
Maintained target VGA039 concentrations were associated with effective hemostatic outcomes.