Vega Therapeutics launches and unveils its first-in-class antibody therapy for von Willebrand disease at ASH Annual Meeting

  • With $40 million in financing to date, company has advanced lead drug candidate, VGA039, to clinical stage to treat von Willebrand disease (VWD)

  • Oral presentation at ASH will feature preclinical data of VGA039, a monoclonal antibody with potential to be a universal hemostatic therapy for bleeding disorders

  • Company has received approval of Clinical Trial Application for Phase 1 study of VGA039

SOUTH SAN FRANCISCO, Calif., December 06, 2022--(BUSINESS WIRE)--Vega Therapeutics, Inc., a clinical stage biotechnology company developing novel therapies for rare blood disorders, today announced its launch and oral presentation of its first-in-class antibody therapy, VGA039, at the 64th American Society of Hematology (ASH) Annual Meeting. Vega was spun out of its parent company, Star Therapeutics, to advance the discovery and development of antibody therapies for patients with blood disorders, starting with von Willebrand disease (VWD).

Vega will disclose the first public information about VGA039 in a podium presentation on December 12th at the ASH Annual Meeting in New Orleans. The presentation will describe preclinical data that demonstrate the potential of VGA039 as a universal hemostatic therapy for bleeding disorders and, in particular, addressing a fundamental mechanism of clot formation in VWD. The company also announced today that the Austrian Competent Authority has approved the Clinical Trial Application for a Phase 1 study of VGA039.

"While other bleeding disorders, such as hemophilia, have benefited from new treatment options that include effective antibody therapies that reduce treatment burden for patients, drug innovation for VWD has lagged. At Vega, we see a future where we can better meet the needs of patients by advancing VGA039 as the first purpose-built antibody therapy for VWD," said Adam Rosenthal, PhD, CEO and Co-Founder of Vega Therapeutics. "We are excited to introduce VGA039 to clinicians and the VWD community in our presentation at ASH and anticipate initiating the first clinical study with VGA039 in the coming months."

Vega has been funded with $40 million to date by Star Therapeutics and its investors, including Westlake Village BioPartners, OrbiMed, Redmile Group, RA Capital, Cowen Healthcare Investments, Cormorant Asset Management, and New Leaf Venture Partners. Proceeds from this funding have enabled Vega to discover VGA039 as a wholly owned drug candidate and advance it to the clinic, including interrogating the novel biology of Protein S in bleeding disorders and conducting antibody development.

"We are pleased with the tremendous progress the team has made to rapidly and efficiently advance a novel antibody therapy for bleeding disorders into the clinic. The data for VGA039 are compelling, and we look forward to supporting Vega as it works to bring this innovative therapy to patients with VWD," said Beth Seidenberg, MD, Founding Managing Director at Westlake Village BioPartners.

Details of the oral presentation at ASH on VGA039 are as follows:

  • Title: Pre-Clinical Characterization of VGA039, an Anti-Protein S Monoclonal Antibody Being Developed as a Universal Hemostatic Agent for Various Bleeding Disorders

  • Presenter: Sandip Panicker, PhD, Chief Scientific Officer

  • Abstract: 691

  • Session: 321 – Coagulation and Fibrinolysis: Basic and Translational

  • Oral presentation session: Monday, December 12, 2022, 10:30 a.m. CT

Vega will describe VGA039’s novel mechanism of action as a monoclonal antibody that modulates Protein S – a key cofactor involved in thrombin generation during both initiation and propagation of coagulation. The presentation will also cover in vitro data demonstrating efficacy of VGA039 in numerous congenital bleeding disorders, including VWD, and the pharmacokinetics of VGA039 following intravenous (IV) and subcutaneous (SC) administration. By promoting thrombin generation through targeting Protein S, VGA039 is mechanistically well suited to overcome VWD pathophysiology, and as a subcutaneously self-administered antibody therapy, has potential to transform VWD treatment. The abstract for the ASH oral presentation includes this overview and can be found here.

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) leads 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 treatments are limited, and include factor replacement therapies that require frequent intravenous (IV) infusions.

About Vega Therapeutics
Vega Therapeutics is a clinical stage biotechnology company developing novel, first-in-class therapies for rare blood disorders with overlooked patient needs, starting with von Willebrand disease (VWD). The company’s lead product candidate, VGA039, is the first purpose-built antibody therapy for VWD with a novel mechanism of action targeting Protein S. 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 a fundamental mechanism of clot formation in VWD. VGA039 has potential to be a universal hemostatic therapy that can treat various bleeding disorders. For more information, please visit our website and follow us on LinkedIn.

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