Star Therapeutics announced interim Phase 1 clinical trial data for VGA039, a novel subcutaneous monoclonal antibody for Von Willebrand Disease (VWD). The therapy targets Protein S to rebalance blood clotting, potentially offering the first subcutaneous treatment option for all VWD types. Early data from three patients with high bleeding rates showed significant reductions in annualized bleed rate (ABR) after a single dose, comparable to existing intravenous prophylaxis therapies, but with a more convenient administration route.
This development is potentially groundbreaking for VWD patients, who currently face a significant treatment burden with frequent intravenous infusions. A convenient, subcutaneous option like VGA039 could improve adherence, quality of life, and potentially long-term outcomes by simplifying prophylaxis. The ability of VGA039 to address all VWD types with a single therapy also simplifies treatment decisions and could improve access to effective preventative care. This advancement also represents significant progress in a field where treatment innovation has lagged behind other bleeding disorders like hemophilia.
The interim data from the Phase 1 trial showed substantial ABR reductions in patients who received a single 3.0 or 4.5 mg/kg subcutaneous dose of VGA039. Importantly, therapeutic concentrations were sustained for several weeks following the single dose, contrasting with the weekly intravenous infusions required by current treatments. The patients included in this interim analysis had high baseline bleeding rates, exceeding 50 bleeds per year, demonstrating the drug’s potential efficacy in severe cases. Separate analyses presented at the same conference highlighted the substantial unmet need in VWD, with data identifying over 50,000 diagnosed and treated patients in the US and emphasizing the high disease and treatment burden associated with the current standard of care.
These early results are promising for VGA039’s potential to transform VWD treatment. Developing a convenient, subcutaneous, and universally applicable therapy could significantly improve patient care and outcomes. Further research, including planned multi-dose studies, will be critical to fully characterize VGA039’s efficacy and safety profile. Positive results from these future studies could pave the way for a new standard of care in VWD management, offering patients a more manageable and effective treatment option.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
