Multicenter phase 1b-2 trial of the combination of GT90001 and nivolumab for advanced hepatocellular carcinoma

GT90001 is an anti-ALK-1 monoclonal antibody. A phase 1b-2 trial at three centers in Taiwan evaluated the combination of GT90001 and nivolumab in patients with advanced hepatocellular carcinoma (aHCC). In phase 1b, no dose-limiting toxicities were seen, and GT90001 7.0 mg/kg plus nivolumab 3.0 mg/kg every two weeks was chosen as the RP2D. Grade 3 or 4 adverse events were seen in 15% of patients; there was no grade 5 AE. The overall response rate and disease control rate were 30% (95% CI 14-6-51.9) and 40% (21.9-61.3), respectively. The median duration of response was not reached. The median progression-free survival was 2.81 months (95% CI 1.71-9.33) with PFS-rate 35% at 6 months and 25% at twelve months. The authors conclude that the combination of GT90001 and nivolumab had a manageable safety profile and promising anti-tumor activity in patients with aHCC.