AVEO Oncology announced the presentation of data from a multicenter, Phase 1b/2 study of FOTIVDA® (tivozanib), a potent, selective, long half-life inhibitor of all three vascular endothelial growth factor (VEGF) receptors, in patients with advanced, unresectable hepatocellular carcinoma (HCC). The data were presented during a poster session titled, “Phase 1b/2 study of tivozanib in patients with advanced inoperable hepatocellular carcinoma” (Abstract #364) at the 2018 American Society of Clinical Oncology (ASCO) Gastrointestinal Cancers Symposium held January 18-20, 2018 in San Francisco. The study, designed to evaluate the safety and efficacy of tivozanib in advanced HCC, enrolled a total of 21 patients at three study sites. In the Phase 1b portion of the trial, which used a modified 3+3 dose escalation design, 8 patients were dosed with tivozanib starting at 1.0 mg daily for 21 days followed by 7 days off drug, with inter-patient escalation to 1.5 mg daily or de-escalation to 0.5 mg daily based on cumulative dose-limiting toxicities (DLT). Upon escalation to 1.5 mg, two patients had on target dose limiting toxicities (grade 3 mucositis and hypertension), which were likely due to the high potency of tivozanib, and came off study without completing the DLT period. Tivozanib at 1.0 mg daily was selected for the Phase 2 expansion portion and was well tolerated. Of 19 evaluable patients, at a median follow up of 16.9 months, the study’s primary endpoint of median progression-free survival (PFS) and PFS at week 24 were 5.5 months and 47%, respectively. A partial response (PR) was seen in 4/19 patients (21%) and stable disease (SD) in 8/19 patients (42%), for a disease control rate (DCR) of 63%. Overall survival (OS) at 6 and 12 months was 58% and 25%, respectively, with a median OS of 7.5 months. Notably, 4 patients have maintained SD for over two years. There were no significant changes in HBV or HCV viral load during study treatment. Tivozanib was generally well tolerated at 1.0 mg daily, with adverse events consistent with those observed in previous tivozanib trials. The Phase 1b/2 study was one of several studies funded by a grant provided to the National Comprehensive Cancer Network from AVEO.