VRN19 / Voronoi - LARVOL DELTA

VRN19: A novel USP1 Inhibitor with anti-tumor efficacy and favorable safety profile (AACR 2025) - "In the BRCA1-mutated CDX model (MDA-MB-436) and PDX with resistance to PARP inhibitors, VRN19 demonstrated synergistic effect with PARP inhibitors, particularly with olaparib, and showed efficacy comparable to KSQ-4279 without toxicity. In a 2-week toxicity study in rats, administration of 300 mg/kg VRN19 showed no significant changes in anemia markers, such as RBC, Hb, and reticulocytes, nor hepatotoxicity indicators like GGT and bilirubin. However, administration of 300 mg/kg KSQ-4279 significantly elevated anemia-related markers and GGT levels, consistent with the adverse events observed in clinical trials with KSQ-4279.In conclusion, VRN19 is a promising novel USP1 inhibitor with favorable safety and improved therapeutic outcomes."

Clinical • Late-breaking abstract • Oncology • BRCA • BRCA1 • BRCA2 • USP1