In a lupus treatment market set for rapid transformation, Ventus Therapeutics is preparing to break new ground with its investigational oral drug for systemic lupus erythematosus (SLE). The biotech firm recently announced that its pioneering cGAS inhibitor, VENT-03, successfully cleared a Phase 1 trial, paving the way for Phase 2 trials slated for next year.
The Phase 1 study involved 72 healthy participants and evaluated VENT-03’s pharmacokinetics (PK), target engagement, safety, and tolerability. The results, published on October 31, highlighted VENT-03’s favorable safety profile, with the drug proving well-tolerated even at doses higher than those planned for the next trial phase. The company believes that VENT-03’s PK profile supports daily oral dosing—a significant advantage over existing lupus treatments, which typically require injections.
“Current lupus treatments focus on injectable options targeting limited aspects of patient symptoms via type I interferon or BAFF pathways,” stated Xavier Valencia, M.D., Head of Clinical Development at Ventus. “With a once-daily oral cGAS inhibitor, we aim to provide a more comprehensive solution that modulates both pathways, potentially offering superior efficacy and addressing a broader range of SLE symptoms.”
Founded in 2019, Ventus has attracted significant investment to fuel its innovative work, raising $60 million, $100 million, and $140 million across multiple rounds. The company also secured an additional $70 million by licensing its NLRP3 inhibitor program, VENT-01, to Novo Nordisk. Utilizing its proprietary ReSOLVE platform, Ventus develops small-molecule drugs that target diseases within immunology, inflammation, and neurology.
Besides VENT-03, Ventus’s pipeline includes NLRP3 inhibitors VENT-02 and VENT-01, both of which have completed Phase 1 trials. Under Novo Nordisk’s leadership, VENT-01 is being further developed for conditions such as metabolic dysfunction-associated steatohepatitis (MASH) and chronic kidney disease.
The landscape for lupus treatment is competitive, with multiple pharmaceutical companies seeking to capture a share of the SLE market. Notably, GSK recently invested $300 million for rights to a T-cell engager from China-based Chimagen Biosciences, complementing its existing SLE treatment, Benlysta. Other players include AstraZeneca, which gained FDA approval for its SLE treatment Saphnelo in 2021, and Biogen, with late-stage candidates dapirolizumab pegol and litifilimab in the pipeline.
A recent survey by Spherix Global Insights of 101 U.S. rheumatologists forecasts significant changes in the lupus treatment landscape. Interest is high not only in novel drugs like VENT-03 but also in potential label expansions for existing treatments such as AbbVie’s Rinvoq and Bristol Myers Squibb’s Sotyktu. With VENT-03 showing early promise, Ventus could be positioned as a formidable player in this evolving market.
