Star Therapeutics Secures $125M for Von Willebrand Disease Drug Development

Star Therapeutics has raised $125 million in a Series D funding round to advance its pipeline of bleeding disorder treatments, with a primary focus on its lead candidate for von Willebrand disease (VWD). The financing, co-led by Sanofi Ventures and Viking Global Investors, will support the company's ongoing Phase 3 trial of VGA039, a novel antibody therapy aimed at transforming VWD treatment.

VGA039: A Potential Game-Changer for VWD Patients

VGA039, Star Therapeutics' flagship product, is designed to target Protein S, promoting proper blood clotting in patients with VWD. The drug has received FDA fast track designation and is currently undergoing a pivotal Phase 3 trial. CEO and founder Adam Rosenthal highlighted the drug's potential to be a "transformative" option for patients, offering a self-administered, once-monthly subcutaneous injection that could provide a significant convenience advantage over standard factor replacement therapies.

"We can treat all types of patients, and all types of bleeds," Rosenthal stated, emphasizing the drug's potential broad applicability across VWD subtypes. This universal approach could position VGA039 as a leading therapy in the VWD market, which affects up to 1% of the U.S. population, according to CDC estimates.

Expanding Horizons in Hematology

While VWD remains the primary focus, Star Therapeutics is exploring VGA039's potential in other blood disorders. The company's approach of targeting multiple diseases with each antibody therapy aligns with its "constellation" strategy, potentially broadening the impact of its research efforts.

Jason Hafler, Managing Director at Sanofi Ventures, who will join Star's board of directors, commented on the investment: "VWD represents a compelling opportunity, with a sizable patient population and significant unmet need. With VGA039 advancing into Phase 3 for VWD, Star is well-positioned to deliver both substantial patient impact and long-term value."

Financial Strength and Future Plans

The latest funding round brings Star Therapeutics' total raised capital to over $300 million since its inception in 2018. The company's financial strategy appears to be paying off, with Rosenthal noting the unique position Star finds itself in: "It's one of those rare circumstances where the market potential is so big, but the spend to get to market is actually quite small. It's something that an independent biotech can very easily do on their own."

While Star remains open to potential partnerships for commercialization, Rosenthal didn't rule out the possibility of an initial public offering in the future. The substantial investor interest, including participation from both new and existing backers such as Sofinnova, Blue Owl Capital, and RA Capital Management, underscores the confidence in Star's approach and the potential of its pipeline.