Our blood has different clotting factors, or protein, that helps clot and stop bleeding after injuries. But when you don’t have enough of these proteins, the blood can’t clot properly, which can cause multiple issues. For example, people with von Willebrand disease (VWD) don’t have enough von Willebrand factor (VWF) or their VWF doesn’t function like it should. This causes abnormal bleeding after dental work, surgery, injuries, or other similar experiences. Vega Therapeutics (“Vega”) is currently working to develop novel treatment solutions for individuals with rare bleeding disorders like VWD. According to an April 2023 news release, the company recently initiated a Phase 1 clinical study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VGA039.

Both healthy participants and people with VWD will enroll in the study. However, the study will initially begin using only healthy participants. The first participant is located in the European Union, though participants from the United States will also enroll as the FDA approved the IND.

VGA039 and its Applicability for von Willebrand Disease (VWD)

First described in 1925, von Willebrand disease is an underserved bleeding disorder caused by more than 300 VWF mutations. It is considered underserved as an estimated 90% of people with von Willebrand disease are not diagnosed; improved diagnostic processes can help patients while increasing our understanding of this disorder. Typically, people are born with this condition. VWD may be acquired in extremely rare cases. Because people with VWD lack enough VWF, they experience prolonged bleeding after injuries, surgery, or similar experiences. VWD is considered mild if people have lower VWF levels than normal, moderate if the VWF levels are normal but defective, and severe if VWF is absent or extremely low. Symptoms of VWD include easy bruising, bloody stool or urine, excessive and long-lasting bleeding following even minor injuries, abnormal menstruation, heavy nosebleeds, and bleeding from the gums.

Earlier this year, Patient Worthy reported on VGA039, which was unveiled during the 65th American Society of Hematology (ASH) Annual Meeting. VGA039 is a first-in-class monoclonal antibody that modulates Protein S. Protein S plays a role in generating thrombin, a blood plasma enzyme that contributes to blood clotting. Preclinical data has shown extreme efficacy in treating not just VWD, but other bleeding disorders. It can be administered subcutaneously by the patient, offering a more efficient and less invasive treatment option than existing therapies which may be painful and require frequent visits to the doctor.