Star Therapeutics Spotlights Novel Bleeding Disorder Drug at JPM

SOUTH SAN FRANCISCO, Calif. – January 05, 2026 – All eyes in the biotechnology sector will turn to San Francisco next week for the 44th Annual J.P. Morgan Healthcare Conference, where Star Therapeutics is set to take the stage. The late clinical-stage company has secured a coveted presentation slot for its CEO and Founder, Dr. Adam Rosenthal, who will provide an update on its lead program, VGA039, a potentially transformative therapy for von Willebrand disease (VWD) and other bleeding disorders.

The presentation, scheduled for January 12, comes at a pivotal time for the company and the broader hematology field. With promising clinical data and substantial financial backing, Star Therapeutics is positioning VGA039 not just as a new treatment, but as a potential new standard of care that could address decades-old challenges in managing the world's most common inherited bleeding disorder.

A New Paradigm for Hemostasis

At the core of the excitement is the novel mechanism of VGA039. Unlike traditional treatments that focus on replacing the deficient von Willebrand factor (VWF), this first-in-class monoclonal antibody takes a different approach. It targets Protein S, a natural anticoagulant in the body. By modulating Protein S, VGA039 has a dual action: it promotes platelet attachment to the site of injury and enhances the generation of fibrin, the protein mesh that forms a stable blood clot. This rebalancing of the body's natural coagulation system is what gives VGA039 its potential as a "universal hemostatic therapy."

This innovative science is backed by increasingly robust clinical evidence. At the American Society of Hematology (ASH) Annual Meeting in December 2025, Star presented compelling interim data from its Phase 1/2 multidose study. The results showed that a once-monthly subcutaneous injection of VGA039 led to substantial bleed reductions—ranging from 73% to 87%—in patients across all types of VWD, including the most severe Type 3. The therapy proved effective in preventing a wide range of bleeds, from serious gastrointestinal events to joint and muscle bleeds, and was reported to be safe and well-tolerated.

Following this success, Star has advanced VGA039 into a pivotal Phase 3 study, known as VIVID-6. This global trial is designed to confirm the efficacy and safety of the subcutaneous therapy as a prophylactic treatment for patients with every type of VWD. The drug has already received both Fast Track and Orphan Drug designations from the U.S. Food and Drug Administration, underscoring the significant unmet need it aims to address and potentially expediting its path to market.

Addressing a Persistent Unmet Need

Von Willebrand disease affects up to 1% of the U.S. population, or over 3 million people, yet it remains widely underdiagnosed. For those who are diagnosed, the treatment burden can be immense. Current standards of care often involve frequent intravenous (IV) infusions of factor replacement concentrates, a process that is both invasive and disruptive to daily life. While effective at stopping acute bleeds, these therapies do not always prevent them, and patients live with the constant risk of severe bleeding events that can lead to long-term joint damage and other complications. The impact on quality of life is profound, with high rates of anxiety and depression reported among patients.

The global VWD treatment market, estimated to be worth hundreds of millions of dollars annually, is ripe for innovation. The demand for more convenient, less burdensome prophylactic options is a driving force in the field. A therapy like VGA039, which can be self-administered subcutaneously just once a month, represents a significant leap forward. It promises not only to improve bleed protection but also to grant patients a level of freedom and normalcy that is often out of reach with current treatments.

The Strategic Play at J.P. Morgan

The J.P. Morgan Healthcare Conference is more than just a scientific forum; it is the premier stage for biotech dealmaking and investor relations. For a private, late-stage company like Star Therapeutics, the presentation is a strategic move to capture the attention of investors, analysts, and potential pharmaceutical partners. The conference is a hotbed for M&A discussions and large financing rounds, and a strong presentation can significantly elevate a company's valuation and strategic options.

Star arrives in San Francisco from a position of financial strength. The company has raised over $300 million to date, including an oversubscribed $125 million Series D financing round announced in late 2025. The round was co-led by heavyweights Sanofi Ventures and Viking Global Investors and saw participation from a blue-chip syndicate of life sciences VCs. This robust funding provides the necessary capital to push VGA039 through its expensive Phase 3 trial and prepare for potential commercialization.

Dr. Rosenthal's presentation will be a critical opportunity to articulate the value proposition of VGA039 to an audience focused on de-risked assets and strong clinical data. In a market showing renewed optimism for M&A and a hunger for genuine innovation, Star's story of a well-funded, late-stage asset with a novel mechanism and strong data is likely to resonate powerfully.

A Legacy of Innovation in a Crowded Field

Star's credibility is bolstered by the proven track record of its leadership team. The company's claim of having invented four first-in-class antibodies is substantiated by their past successes. Dr. Rosenthal and Chief Medical Officer Dr. Gary Patou were previously leaders at True North Therapeutics, the company that developed ENJAYMO®, the first-ever approved drug for cold agglutinin disease. True North was acquired for $825 million, one of several successful exits for the team's key members. This history of identifying novel biological targets and navigating them to clinical and commercial success lends significant weight to their current efforts with VGA039.

However, Star is not operating in a vacuum. The competitive landscape for bleeding disorders is evolving rapidly. Hemab Therapeutics is also developing a subcutaneous prophylactic for VWD, HMB-002, which is currently in Phase 1/2 trials. Beyond VWD, the broader hemophilia market has been transformed by non-factor therapies like Roche's Hemlibra and Sanofi's recently approved RNAi therapy, Fitusiran, which have set a new bar for convenience and efficacy. While VGA039's unique mechanism targeting Protein S differentiates it, the company must prove its value in an increasingly innovative and competitive field. As the industry converges on San Francisco, the presentation by Dr. Rosenthal is poised to be a pivotal moment for the company and a beacon of hope for patients awaiting a new standard of care.