The efficacy and safety of Surufatinib monotherapy as a third-line treatment for advanced hepatocellular carcinoma: A single-arm, open-label, multi-center phase II study.

e16209 Background: Hepatocellular carcinoma (HCC) is an aggressive liver cancer with limited treatment options, particularly in advanced stages. Surufatinib is a small-molecule tyrosine kinase inhibitor that targets VEGFR-1/2/3, FGFR-1, and CSF-1R, has dual anti-angiogenesis and immune-modulating effects. It may provide a new treatment option for patients with advanced HCC. Methods: This single-arm, open-label, multi-center Phase II trial (NCT05282433) evaluated Surufatinib monotherapy (300 mg qd, po, q3w) as a third-line treatment for advanced HCC. Eligible patients had received ≤2 prior systemic treatments, ECOG PS 0-1, BCLC stage B or C, Child-Pugh A or B, and measurable tumors. The primary endpoint was PFS, the secondary endpoints included DCR, ORR, OS, and safety. Results: As of December 12, 2024, 14 patients were enrolled (median age 60 years, 92.86% male, 78.57% BCLC stage C, 85.71% Child-Pugh A, 64.29% AFP < 400 μg/L, 85.71% CA199 < 35 U/L, 21.43% with vascular invasion, 42.86% with lung metastases, and 100% had received anti-angiogenic therapy). The median PFS was 4.3 months (95% CI: 3.0-NA) with an ORR of 7.14% and DCR of 78.57%. The median OS has not been reached. The 6-month, 12-month and 18-month OS rates were 76.61%, 67.03% and 67.03%, respectively. Grade ≥3 TRAE were thrombocytopenia (10.81%) and leukopenia (8.11%), with no unexpected adverse events or deaths to TRAE reported. Conclusions: Surufatinib demonstrates promising efficacy and manageable safety in the third-line treatment of advanced hepatocellular carcinoma. Larger sample sizes are needed to validate the results of this study. Patient enrollment is ongoing, and we will continue to analyze and report findings to provide new treatment options for patients with advanced hepatocellular carcinoma. Clinical trial information: NCT05282433 .