Star Therapeutics, a clinical stage biotechnology company headquartered in San Francisco, CA, recently announced new interim clinical trial data for their investigational von Willebrand disease therapy VGA039. The data comes from VIVID 2, a phase 1 single ascending dose study that is being conducted at multiple sites in the U.S. and abroad.
VGA039 is a subcutaneously-delivered monoclonal antibody therapy designed to target Protein S as a means of restoring proper blood clotting in people with all types of von Willebrand disease (VWD). Protein S functions with other proteins in the body to prevent excessive blood clotting.
According to a press release by Star Therapeutics, their interim clinical data is based on just three VWD patients, one with type 2M and two with type 3. The data showed that a subcutaneous dose of VGA039 was associated with substantial (75% to 88%) reductions in annualized bleed rates in the three patients. These reductions were in line with currently FDA-approved VWD prophylaxis therapies. Investigators also reported that VGA039 sustained “multi-week therapeutic concentrations” in response to a single subcutaneous dose. This stands in contrast to other currently-approved VWD treatments, which necessitate multiple intravenous infusions of von Willebrand factor-replacement therapies.
“Drug innovation for VWD has lagged behind advancements in treatments for other bleeding disorders, like hemophilia. VGA039 offers a promising breakthrough as a subcutaneous therapy, potentially transforming care for VWD patients by reducing their high treatment burden,” said Dr. Nicholetta Machin of the Hemophilia Center of Western Pennsylvania and University of Pittsburgh. “The interim clinical data is particularly encouraging, showing significant bleed reduction in VWD patients with high baseline bleeding rates.”
The interim clinical data from VIVID 2 was reported in a poster presentation during the American Society of Hematology (ASH) meeting, which was held December 7-10, 2024, in San Diego, California. Read the full press release to learn more about this and related posters presented at ASH.
Source: Star Therapeutics press release dated December 9, 2024
Writer: Jonathan Angarola, Senior Manager of Medical Programs & Information
