The FDA granted orphan drug designation to ezurpimtrostat (GNS561) for hepatocellular carcinoma (HCC) treatment. Genoscience Pharma's phase 2b trial combines ezurpimtrostat with atezolizumab/bevacizumab, aiming to improve HCC outcomes. Ezurpimtrostat, a PPT-1 inhibitor, shows promise in liver cancer treatment, with phase 1b trial data indicating safety and reduced PPT1 expression in liver cancer tissues.