What's Happening?
Star Therapeutics has presented interim data from its Phase 1/2 multidose study of VGA039, a monoclonal antibody therapy for von Willebrand disease (VWD), at the American Society of Hematology Annual Meeting.
The study demonstrated substantial reductions in bleeding rates across all types of VWD and bleeds, including in patients switching from prior intravenous prophylaxis. VGA039, administered subcutaneously once monthly, showed a favorable safety profile and significant efficacy, with bleed reductions ranging from 73% to 100%. The therapy targets Protein S, promoting platelet attachment and enhancing fibrin deposition to restore hemostasis. The promising results support the ongoing pivotal Phase 3 trial of VGA039.
Why It's Important?
The interim results for VGA039 are significant as they offer a potential new treatment option for VWD, a common inherited bleeding disorder affecting over 130,000 people in the U.S. Current treatments require frequent intravenous infusions, which can be burdensome for patients. VGA039's once-monthly subcutaneous administration could dramatically reduce treatment burden and improve quality of life for VWD patients. The therapy's ability to significantly reduce bleeding rates and its favorable safety profile suggest it could become a new standard of care, providing a more convenient and effective treatment option for patients.
What's Next?
Star Therapeutics is enrolling patients in the pivotal Phase 3 trial, VIVID-6, to further investigate the safety and efficacy of VGA039. The trial's success could lead to regulatory approval and wider clinical use of VGA039, potentially transforming the treatment landscape for VWD. The company plans to continue presenting data and engaging with regulatory authorities to advance VGA039's development. If successful, VGA039 could become a universal hemostatic therapy for multiple bleeding disorders, starting with VWD, offering hope to patients with limited treatment options.
