Waltham, Mass.– Ventus Therapeutics has announced it will present Phase 1 clinical trial results for its investigational drug VENT-03 at the upcoming 16th International Congress on Systemic Lupus Erythematosus (LUPUS 2025), being held May 21–24 in Toronto. VENT-03 is the first oral small-molecule inhibitor of cyclic GMP-AMP synthase (cGAS) to reach this stage of development, showing early promise as a potential breakthrough treatment for lupus and other autoimmune diseases.
According to Ventus, the Phase 1 study demonstrated that VENT-03 was safe and well tolerated across all tested doses in healthy volunteers. The drug also exhibited a favorable pharmacokinetic (PK) profile that supports once-daily oral dosing, along with pharmacodynamic (PD) data indicating strong engagement with its intended biological target.
“This is the first reported Phase 1 data for a cGAS inhibitor, and the results underscore VENT-03’s potential as a first- and best-in-class therapy,” said Marcelo Bigal, M.D., Ph.D., President and CEO of Ventus Therapeutics. “We believe cGAS inhibition could address significant unmet needs in lupus and other autoimmune conditions, and we are excited to begin Phase 2 trials later this year.”
The Phase 1 trial enrolled 72 healthy adult participants in both single and multiple ascending dose cohorts. Participants received up to 2,000 mg of VENT-03 in a single dose and 900 mg daily for 10 days. The adverse event profile was comparable between the VENT-03 and placebo groups, with most side effects classified as mild and unrelated to the study drug.
The drug candidate was discovered through Ventus’ proprietary ReSOLVE® platform, which leverages structural biology and computational chemistry to identify and optimize small molecules. With its once-daily oral dosing and favorable safety data, VENT-03 may offer a more convenient and tolerable treatment option for lupus patients compared to current therapies.
Mona Kotecha, M.D., Chief Medical Officer at Ventus, highlighted the importance of innovation in lupus care. “Over five million people worldwide are affected by lupus, which often impacts multiple organs and significantly reduces quality of life,” she said. “VENT-03 has the potential to provide a new, more effective, and safer treatment option. We’re excited to engage with the lupus community at LUPUS 2025 and share these encouraging results.”
The company expects to launch a Phase 2 trial of VENT-03 in lupus patients by the end of 2025.
