• Alias: AG-120
    • An orally available inhibitor of IDH1 that specifically inhibits a mutated form of IDH1 in the cytoplasm, which inhibits the formation of the oncometabolite, 2HG. This may lead to both an induction of cellular differentiation and an inhibition of cellular proliferation in IDH1-expressing tumor cells.
    • Phase 3 in IDH1 gene–mutated cholangiocarcinoma; phase 1b/2 in combination with SubQ azacitidine in newly diagnosed AML with IDH1 or IDH2 mutation.
    • Recommended phase 3 dose: 500 mg PO daily until disease progression
    • Half-life: 38.4 to 86.3 hours
    • Common side effects: Fatigue, diarrhea, nausea, appetite, peripheral edema, prolonged QT, dyspnea, anemia, hypertension, transaminitis
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