Multicenter phase 2 trial of first-line regorafenib plus nivolumab for unresectable hepatocellular carcinoma

The antiangiogenic multikinase inhibitor regorafenib has anti-tumor activity in patients with unresectable hepatocellular carcinoma (uHCC), with potential immunomodulatory effects, suggesting that its combination with immune checkpoint inhibitor may have clinically meaningful benefits in patients with uHCC. The multicenter phase 2 RENOBATE trial in South Korea evaluated the combination of regorafenib and nivolumab as first-line treatment for uHCC patients. The figure shows clinical outcomes of the trial. The ORR was 31.0%, meeting the primary end point. The median progression-free survival was 7.38 months with a one-year PFS rate of 37.8%, and the median overall survival was not reached with a one-year OS rate of 80.5%. The most common adverse events were palmar-plantar erythrodysesthesia syndrome (38.1% of patients), alopecia (26.2%), and skin rash (23.8%). The authors conclude that these results support further clinical research of the regorafenib-nivolumab combination as front-line treatment for uHCC.