Vaderis Therapeutics Secures $152M Series B, Launches Phase 3 HEROIC Trial of Engasertib for HHT
核心洞察
Vaderis Therapeutics (搜索) raised $152 million in Series B financing to advance engasertib (搜索), a first-in-class AKT1/2 inhibitor for hereditary hemorrhagic telangiectasia (搜索) (HHT).
The global Phase 3 HEROIC study has been initiated following positive proof-of-concept data published in The New England Journal of Medicine demonstrating clinically meaningful improvements.
HHT affects approximately 1 in 3,800 people in the U.S. and currently has no approved therapies, leaving patients reliant on off-label cancer drugs and symptom management.
Vaderis Therapeutics (搜索), a Swiss clinical-stage biotechnology company, has secured $152 million in Series B financing and simultaneously launched the global Phase 3 HEROIC study of its lead candidate engasertib (搜索) for hereditary hemorrhagic telangiectasia (搜索) (HHT), a rare inherited blood vessel disorder with no approved therapies. The financing was co-led by Life Sciences at Goldman Sachs Alternatives (搜索) and TCGX (搜索), with participation from Omega Funds, EQT Life Sciences (搜索), Perceptive Advisors, Kalehua Capital, and existing investors Medicxi and Droia.
"We'll look at all strategic options as we go forward, but now we're in a position to fully execute everything we intend to between now and 2029," said Azmi Nabulsi, Vaderis' CEO, in an interview with BioPharma Dive. The company now has sufficient capital to potentially bring engasertib (搜索) to market independently should the pivotal trial succeed.
The Unmet Need in HHT
HHT is a genetic vascular disease characterized by the formation of abnormal blood vessels, leading to recurrent and severe nosebleeds (epistaxis), chronic anemia, and potentially life-threatening vascular abnormalities. The disease also results in the appearance of small, visible blood vessels on the skin called telangiectasias. Vaderis estimates that HHT affects approximately 1 in every 3,800 people in the United States.
No curative therapies exist for HHT. Current treatment approaches are limited to symptom management and procedures that either destroy or block blood flow into malformed arteries. Multiple cancer drugs, including Roche's Avastin, are used off-label to treat complications, but no therapy has been specifically developed and approved for HHT.
"HHT remains a serious, lifelong disease with no approved treatment anywhere in the world, despite affecting tens of thousands of patients," said Christoph Broja, CFA, Partner at EQT Life Sciences (搜索), who will join the Vaderis board as an observer.
Engasertib (搜索): A Targeted AKT (搜索) Inhibitor
Engasertib (搜索) is designed as a targeted therapy for HHT, inhibiting an enzyme called AKT (搜索) that is involved in cell survival and growth but becomes hyperactive in the disease. By specifically targeting the AKT1 and AKT2 isoforms, Vaderis aims to normalize blood vessel production while avoiding some of the tolerability issues associated with broader AKT inhibition, such as hyperglycemia.
Pierre Saint-Mezard, Vaderis' chief scientific officer, hypothesizes that this selective approach should be potent and safe enough to serve as a "chronic therapy that can put this disease at bay." While AKT (搜索) inhibitors are an established drug class — AstraZeneca's Truqap is approved for breast cancer — Vaderis is among at least a few companies testing these agents specifically for HHT.
"We're giving the patients back their lives, not just controlling their symptoms," Nabulsi said.
Phase 3 HEROIC Trial
The HEROIC study represents the first Phase 3 trial utilizing a molecule specifically developed for HHT. The decision to advance into pivotal development follows the publication of positive proof-of-concept and long-term extension data in The New England Journal of Medicine, which demonstrated clinically meaningful and sustained improvements across multiple measures of disease in patients with HHT.
According to a federal database, the Phase 3 trial could produce initial results in 2028. EQT Life Sciences (搜索), which invested $17.5 million as part of the round, will draw on its experience supporting biotechnology companies through late-stage clinical development to work alongside Vaderis' management team.
"Today represents a defining moment for HHT patients. Closing this financing and initiating HEROIC as the first Phase 3 study utilizing a molecule specifically developed for HHT marks an exciting new chapter," Nabulsi said. "This milestone reflects the dedication of our patients, investigators, study teams, and advocacy organizations, to whom we extend our deepest gratitude."
Colin Walsh, managing director at Life Sciences at Goldman Sachs Alternatives (搜索), added: "The company's strong scientific foundation, disciplined execution and clear focus on addressing a significant unmet medical need gave us conviction in both the financing and the Phase 3 program."