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Ezurpimtrostat

Generic Name
Ezurpimtrostat
Brand Names
-
Drug Type
Small Molecule
Chemical Formula
C25H31ClN4
CAS Number
1914148-72-3
Unique Ingredient Identifier
MS6LGW5JFK
Associated Conditions
-
Associated Therapies
-
onclive.com
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FDA Grants Orphan Drug Designation to Ezurpimtrostat for Hepatocellular Carcinoma Treatment

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.
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