Ventus Therapeutics Doses First Patient in Phase 2 Trial of VENT-03, an Oral cGAS Inhibitor, in Lupus
Ventus Therapeutics announced that the first patient has been dosed in a Phase 2 clinical trial of VENT-03 in patients with lupus.12
VENT-03 is described as the most advanced oral small‑molecule cGAS (cyclic GMP‑AMP synthase) inhibitor currently in clinical development and the first oral cGAS inhibitor to complete Phase 1 and enter Phase 2 in patients.12
The Phase 2 study is a multicenter, randomized, double‑blind, placebo‑controlled trial with an open‑label extension in patients with lupus who have active skin disease.12
Key objectives include evaluating the safety and efficacy of VENT-03 on interferon‑stimulated and inflammatory gene signatures in blood and skin, as well as its effect on clinical disease severity.12
Additional objectives are to assess VENT‑03’s impact on systemic disease activity using standard lupus scales and on a robust panel of biomarkers, including cardiovascular and senescence markers relevant to broader diseases beyond lupus.12
The placebo‑controlled portion of the trial is 28 days, followed by a 2‑month open‑label extension.12
Top‑line data from the 28‑day placebo‑controlled phase are expected in the second half of 2026, with open‑label extension data to follow.12
VENT‑03 was discovered using Ventus’ proprietary ReSOLVE structural immunology platform.12
Ventus positions VENT‑03 as a potential once‑daily oral treatment that offers a differentiated approach for autoimmune disease, with the goal of being a more convenient option for patients with limited current therapies.12
The company highlights the potential for cGAS inhibition, and VENT‑03 specifically, to be applied beyond lupus to other interferon‑driven immunology and inflammation (I&I) diseases, cardiometabolic conditions, and inflammaging.12
Sources:
1. https://financialpost.com/pmn/business-wire-news-releases-pmn/ventus-therapeutics-announces-first-patient-dosed-in-phase-2-clinical-trial-of-vent-03-a-novel-small-molecule-cgas-inhibitor-in-patients-with-lupus
2. https://www.businesswire.com/news/home/20251209988664/en/Ventus-Therapeutics-Announces-First-Patient-Dosed-in-Phase-2-Clinical-Trial-of-VENT-03-a-Novel-Small-Molecule-cGAS-Inhibitor-in-Patients-with-Lupus