In single-ascending dose study in VWD patients, a subcutaneous dose of VGA039 was associated with substantial reductions in bleeds

Additional ASH posters show real-world patient data that identify over 50,000 diagnosed and treated VWD patients in the US and highlight high disease and treatment burden with current standard of care

Star Therapeutics, a clinical stage biotechnology company discovering and developing best-in-class antibodies, today announced interim clinical data from VIVID 2 (VGA039 Investigation in Von Willebrand Disease), a Phase 1 single ascending dose (SAD) study of VGA039 in Von Willebrand Disease (VWD) patients. VGA039 is an investigational monoclonal antibody that targets Protein S to restore balance in blood clotting, as a universal hemostatic therapy for numerous bleeding disorders. It is potentially the first subcutaneous therapy that addresses all types of VWD and has a convenient dosing regimen. The VGA039 clinical data is being presented at the annual meeting of the American Society of Hematology (ASH) on December 7-10, in San Diego, California.

The interim clinical data in VIVID 2 from three VWD patients with high bleeding rates showed that a subcutaneous dose of VGA039 was associated with substantial reductions in annualized bleed rate (ABR) in line with currently approved VWD prophylaxis therapies. In the interim VIVID 2 data, VGA039 sustained multi-week therapeutic concentrations following a single subcutaneous dose, in contrast to currently approved VWD prophylaxis therapies that require multiple intravenous (IV) infusions every week. These VWD patients had high bleeding rates with ABRs greater than 50 bleeds per year.

“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.”

Interim clinical data from VIVID 2 (Phase 1 SAD study in VWD patients)

The interim clinical data from VIVID 2 reported in the poster presentation at ASH 2024 describe results in VWD patients who received a 3.0 or 4.5 mg/kg dose of VGA039 administered subcutaneously and evaluated over 8 weeks, including the following highlights:

• In the seven VWD patients evaluated for safety, pharmacodynamic (PD) and pharmacokinetic (PK) activity, VGA039 was safe and well-tolerated with no injection site reactions observed. VGA039 demonstrated pharmacokinetics and pharmacodynamics that support convenient, prophylactic subcutaneous dosing.
  • Therapeutic concentrations of VGA039 were sustained for approximately 4 weeks following a single subcutaneous 4.5 mg/kg dose.
  • VGA039 showed PD activity across all types of VWD patients, including Type 1, Type 2 and Type 3.
• In three VWD patients (a Type 2M and two Type 3) with high historic bleeding rates, a single subcutaneous 4.5 mg/kg dose of VGA039 resulted in 75% to 88% reductions in annualized bleeding rate (ABR). These reductions are consistent with those observed in clinical trials of approved VWD prophylactic therapies that require multiple IV infusions every week.

“These initial results showing a significant reduction of bleeds in VWD patients highlight the exciting prospects ahead for VGA039. We are keenly aware of the unmet need of VWD patients and are working to bring the first potential subcutaneous therapy for patients with all types of VWD,” said Gary Patou, MD, Chief Medical Officer of Star Therapeutics. “Based on the positive results, we are moving ahead with our plans to begin a multi-dose study with VGA039 in early 2025.”

Additional ASH posters and session details

Star Therapeutics is also presenting three other posters at ASH 2024, including two posters with analyses of large real-world VWD patient data sets highlighting the significant disease burden and unmet treatment need in VWD.

• The posters describe data from more than 50,000 diagnosed and treated VWD patients in a large US claims dataset.
• VWD patients experienced high disease burden with bleeding comorbidities, including anemia, heavy menstrual bleeding, nosebleeds, gastrointestinal bleeds, and joint bleeds, sometimes requiring blood transfusions.
• There still remains a disparity in VWD treatment with low prophylaxis use, likely due to the high treatment burden of currently available therapies, supporting the need for less frequent treatment options for VWD patients.

The Star Therapeutics posters are presented at ASH today, December 9, at 6:00 to 8:00 p.m. PT in Hall G/H of the San Diego Convention Center, with the following details:

• 3981: A Phase 1a Study of VGA039, a Protein S-Targeting Monoclonal Antibody, in Individuals with Von Willebrand Disease Demonstrates Concentration-Dependent Increases in Thrombin Generation for Reducing Bleeding
• 3980: Diagnosis and Disease Burden of Von Willebrand Disease in a Large US Population Based-Dataset
• 3978: Disparities in Real-World Treatment Patterns Among Patients with Von Willebrand Disease in a Large US Population-Based Dataset
• 3982: Factor VIII Coagulant Activity (FVIII:C) As an Independent Predictor of Bleeding across Different Subtypes of Von Willebrand Disease (VWD)

About the VIVID clinical program

The VIVID multinational clinical program is a platform multi-phase protocol for the development of VGA039 in Von Willebrand disease (VWD). Patient bleeding is quantified by the number of bleeding events per year or annualized bleeding rate (ABR). For the Phase 1 study, VIVID 2 is the single ascending dose study in VWD patients (NCT05776069).

About von Willebrand disease

Von Willebrand disease (VWD) is the most common inherited bleeding disorder in which the blood does not clot properly, caused by absent or defective von Willebrand factor (VWF). VWD patients may experience excessive bleeding with variability in severity and frequency, negatively impacting their daily lives. Current therapies for VWD prophylaxis include factor replacement therapies requiring multiple intravenous (IV) infusions every week.

About VGA039

VGA039 is a monoclonal antibody therapy with a novel mechanism of action that targets Protein S, a key component in restoring balance to the blood clotting process. VGA039 has potential to be a universal hemostatic therapy that can treat numerous bleeding disorders, with the first disease indication of VWD. As a subcutaneously self-administered antibody therapy with a convenient dosing regimen, VGA039 has the potential to dramatically reduce treatment burden for 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/20241209479183/en/