[Comment] Drug development for hepatocellular carcinoma

In The Lancet Oncology, Josep M Llovet and colleagues1 report the eagerly awaited results of the LEAP-002 study comparing lenvatinib plus pembrolizumab with lenvatinib plus placebo in advanced hepatocellular carcinoma.1 Despite promising results from the previous phase 1b study of lenvatinib plus pembrolizumab,2 among 794 patients randomly assigned to lenvatinib plus pembrolizumab (n=395; median age 66·0 years IQR 57–72) or lenvatinib plus placebo (n=399; 66·0 years 57–73; 644 [81%] were male, 150 [19%] were female, 345 [43%] were Asian, 345 [43%] were White, 22 [3%] were multiple races, 21 [3%] were American Indian or Alaska Native, 21 [3%] were Native Hawaiian or other Pacific Islander, and 13 [2%] were Black or African American) the LEAP-002 trial did not meet the prespecified significance for superiority for either of the primary endpoints of progression-free survival and overall survival.