Star Therapeutics Unveils Clinical Data for VGA039, a Subcutaneous Von Willebrand Disease Therapy

Star Therapeutics, a biotechnology company specializing in developing advanced antibody treatments, recently shared promising clinical data from its VGA039 study at the 32nd Congress of the International Society on Thrombosis and Haemostasis (ISTH) in Bangkok, Thailand. The VGA039 study, known as VIVID 1, is a Phase 1 clinical trial focusing on healthy volunteers. This novel antibody therapy aims to serve as a universal treatment for multiple bleeding disorders and potentially become the first subcutaneous therapy for von Willebrand disease (VWD).

VGA039 demonstrated favorable pharmacokinetics and pharmacodynamics in healthy volunteers, supporting its potential as a convenient subcutaneous therapy. The study showed nearly 100% bioavailability when administered subcutaneously, along with a favorable safety and tolerability profile. The trial's success in healthy individuals paves the way for the next phase of the VIVID program, which will involve VWD patients.

Jeanette Cesta, Executive Director of the VWD Connect Foundation, emphasized the significant impact of VGA039 on reducing the treatment burden for VWD patients and expressed hope for future advancements in VWD treatments. The oral presentation at the ISTH Congress highlighted several key findings, including VGA039's safety and tolerability across both intravenous (IV) and subcutaneous (SC) administrations. No serious adverse events were reported, and no reactions were observed at injection sites. The study also showed dose-proportional increases in VGA039 concentration in both IV and SC groups, with a half-life of 25 days, and dose-dependent increases in thrombin generation and other pharmacodynamic markers.

In earlier presentations, VGA039 demonstrated efficacy in a VWD non-human primate model and the ability to restore thrombin generation in various bleeding disorders using patient samples. These results support VGA039's potential as a universal hemostatic agent. Star Therapeutics' Chief Medical Officer, Gary Patou, M.D., expressed enthusiasm about the promising clinical data and the ongoing development of VGA039, which aims to provide a more convenient subcutaneous dosing regimen for VWD patients.

VGA039 is a fully human, IgG4 monoclonal antibody with a unique mechanism that modulates Protein S, a crucial cofactor in thrombin generation. By attenuating Protein S function for tissue factor pathway inhibitor α (TFPIα) and activated protein C (aPC), VGA039 enhances and restores thrombin generation during coagulation, addressing key aspects of clot formation. This mechanism positions VGA039 as a potential universal therapy for various bleeding disorders, including VWD. Its subcutaneous administration and convenient dosing could significantly ease the treatment burden for patients.

Von Willebrand disease is a bleeding disorder where blood fails to clot properly due to defective or insufficient von Willebrand factor (VWF), leading to severe bleeding and significant impacts on patients' daily lives. Current VWD treatments often require frequent IV infusions multiple times a week, highlighting the need for more convenient therapeutic options.

The VIVID clinical program is designed to develop VGA039 for VWD through a multi-phase approach. VIVID 1 focuses on single ascending doses in healthy volunteers, while VIVID 2 will extend this investigation to VWD patients.

Star Therapeutics is dedicated to discovering and developing best-in-class antibody therapies for unmet needs in hematology and immunology. The company's expertise in antibody innovation aims to address multiple diseases with single therapies, backed by leading life sciences investors. Located in South San Francisco, Star Therapeutics continues to advance its mission to create life-changing treatments for patients.