Merck’s Keytruda Fails in Late-Stage HCC Trial

Merck’s vaunted PD-1 inhibitor Keytruda took a stumble in a late-stage trial as a treatment of patients with advanced hepatocellular carcinoma (HCC) who were previously treated with systemic therapy. In the pivotal Phase, III KEYNOTE-240 trial, Keytruda plus best supportive care failed to hit endpoints in both overall survival and progression-free survival in comparison to placebo and best supportive care. Merck said in the final analysis of the KEYNOTE-240 data there was some improvement in overall survival, however, that did not meet statistical significance according to trial design. Also, the company said that when it came to progression-free survival, there were favorable results in the Keytruda arm, but again, it did not meet statistical significance.
The key secondary endpoint of objective response rate (ORR) was not formally tested since superiority was not reached for overall survival or progression-free survival. Keytruda continues to show a consistent safety profile. The failure of KEYNOTE-240 was a blow for Merck in this indication, particularly after Keytruda was approved by the U.S. Food and Drug Administration (FDA) in November 2018 for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with the chemotherapy drug sorafenib.