Star Therapeutics announced clinical data from the VIVID 1 Phase 1 clinical study of VGA039 in healthy volunteers, revealing its potential as a subcutaneous therapy for von Willebrand disease (VWD). The data, presented at the 32nd Congress of the International Society on Thrombosis and Haemostasis (ISTH) in Bangkok, Thailand, supports VGA039's favorable safety profile and convenient subcutaneous dosing regimen. VGA039 is designed as a universal hemostatic therapy for numerous bleeding disorders.
The VIVID clinical program includes data from the first part of the study in healthy volunteers (VIVID 1), with the next phase underway in VWD patients (VIVID 2) in a multinational Phase 1 study (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.
Key Findings from VIVID 1
The oral presentation at ISTH 2024 Congress highlighted several 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, and no infusion-related or injection site reactions occurred.
- 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.
Preclinical Data
Previously reported results from 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.
Expert Commentary
"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."
Jeanette Cesta, Executive Director, VWD Connect Foundation, added, "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. We join the patient community in encouraging the advancement of new treatments that continue to bring us hope for the future."
