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Amezalpat (formerly TPST-1120) is an investigational, orally available, small molecule that acts as a selective and competitive antagonist of peroxisome proliferator-activated receptor alpha (PPAR⍺). It is being developed primarily for the treatment of hepatocellular carcinoma (HCC), where it has shown efficacy in combination with atezolizumab and bevacizumab. The drug works by directly targeting tumor cells as well as modulating immune-suppressive cells and reducing angiogenesis within the tumor microenvironment. Amezalpat has received both orphan drug and fast track designations from the FDA for HCC based on positive results from phase 1b/2 clinical trials, which demonstrated improved overall survival compared to standard-of-care therapy. Additional studies are ongoing or planned in advanced solid tumors and other FAO-dependent cancers[1][3][4][5][7].
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