VGA039 clinical data supports favorable safety profile in healthy volunteers and convenient subcutaneous dosing regimen

Oral presentation delivered at International Society on Thrombosis and Haemostasis (ISTH) Congress

Next stage of VIVID clinical program underway in VWD patients

Star Therapeutics, a clinical stage biotechnology company discovering and developing best-in-class antibodies, today announced the presentation of clinical data from VIVID 1 (VGA039 Investigation in Von Willebrand Disease), a Phase 1 clinical study of VGA039 in healthy volunteers. VGA039 is designed to be a universal hemostatic therapy for numerous bleeding disorders and potentially the first subcutaneous therapy for von Willebrand disease (VWD). The VGA039 clinical data was presented today in an oral presentation at the 32^nd Congress of the International Society on Thrombosis and Haemostasis (ISTH), taking place on June 22-26 in Bangkok, Thailand.

The data reported from the VIVID clinical program are from the first part of the study in healthy volunteers (VIVID 1) with the next part of the program underway in VWD patients (VIVID 2) in the multinational Phase 1 study of VGA039 (NCT05776069). The Phase 1 results in healthy volunteers demonstrated pharmacokinetics and pharmacodynamics for VGA039 that support convenient subcutaneous dosing and showed nearly 100% subcutaneous bioavailability, with a favorable safety and tolerability profile.

“We welcome the ongoing progress of VGA039 as the potential first subcutaneous treatment for VWD to reduce the substantial treatment burden that currently impacts VWD patients and families,” said Jeanette Cesta, Executive Director, VWD Connect Foundation. “We join the patient community in encouraging the advancement of new treatments that continue to bring us hope for the future.”

The oral presentation at ISTH 2024 Congress includes these key findings:

• VGA039 was safe and well-tolerated with intravenous (IV) and subcutaneous (SC) administrations of single ascending doses in healthy volunteers.
  • No serious or thromboembolic adverse events (AEs) were observed.
  • No infusion-related or injection site reactions were observed.
• Dose-proportional increases in VGA039 concentration were demonstrated in both IV and SC cohorts.
• VGA039 showed nearly 100% SC bioavailability, with a half-life (t_1/2) of 25 days.
• VGA039 demonstrated dose-dependent increases in thrombin generation and other pharmacodynamic biomarkers.

In previously reported results in an oral presentation at the American Society of Hematology (ASH) annual meeting in December 2022, VGA039 demonstrated in vivo efficacy in a VWD non-human primate model. In additional studies using patient samples, VGA039 demonstrated in vitro the ability to restore thrombin generation in numerous bleeding disorders, supporting the potential of VGA039 to be effective as a universal hemostatic agent. Star Therapeutics’ VGA039 is being developed through the company’s Vega therapeutic group.

“We are delighted to present these first clinical data for VGA039. VGA039’s high subcutaneous bioavailability and favorable pharmacokinetic profile supports convenient subcutaneous dosing for VWD prophylaxis,” said Gary Patou, M.D., Chief Medical Officer of Star Therapeutics. “We are excited to continue moving ahead with our VIVID clinical program of VGA039 in VWD patients.”

About VGA039

VGA039 is a fully human, IgG4 monoclonal antibody therapy with a novel mechanism of action that modulates Protein S, a key cofactor involved in thrombin generation. By attenuating Protein S cofactor function for tissue factor pathway inhibitor α (TFPIα) and activated protein C (aPC), VGA039 augments and restores thrombin generation during both initiation and propagation of coagulation, addressing fundamental mechanisms of clot formation. VGA039 has potential to be a universal hemostatic therapy that can treat numerous bleeding disorders, including von Willebrand disease. As a subcutaneously self-administered antibody therapy with convenient dosing, VGA039 has the potential to significantly reduce treatment burden for patients.

About von Willebrand disease

Von Willebrand disease (VWD) is a bleeding disorder in which the blood does not clot properly. In VWD, defective or low amounts of von Willebrand factor (VWF) lead to insufficient platelet adhesion and unstable clot formation. VWD causes severe bleeding that can damage organs and lead to significant impact on patients’ daily lives. Current therapies for VWD prophylaxis include factor replacement requiring frequent intravenous (IV) infusions multiples times every week.

About the VIVID Clinical Program

The VIVID (VGA039 Investigation in Von Willebrand Disease) clinical program is a platform multiphase protocol for the development of VGA039 in Von Willebrand disease. VIVID 1 is the single ascending dose study in normal healthy volunteers and VIVID 2 is the single ascending dose study in VWD patients.

About Star Therapeutics

Star Therapeutics is a clinical stage biotechnology company discovering and developing best-in-class antibodies to create life-changing therapies for patients, initially addressing unmet needs in hematology and immunology. The company applies its expertise in antibody innovation to interrogate areas of biology that have been overlooked and have the potential for addressing multiple diseases with a single therapy. Star is backed by leading life sciences investors and located in South San Francisco. For more information, please visit Star Therapeutics and follow us on LinkedIn.

View source version on businesswire.com: https://www.businesswire.com/news/home/20240626849178/en/