AG-120 (Ivosidenib) is used in the treatment of acute myeloid leukemia (AML) with a specific genetic mutation known as IDH1. It works by inhibiting the mutated IDH1 enzyme, which helps reduce the production of an oncometabolite called 2-hydroxyglutarate (2-HG). Lowering 2-HG levels allows immature blood cells to mature properly, reducing the number of abnormal cells in the bone marrow and blood. This targeted therapy is especially beneficial for patients who are not eligible for intensive chemotherapy. It is taken orally, offering a convenient treatment option, and has shown improved outcomes in terms of remission rates and survival in clinical trials. AG-120 is also being studied in combination with other agents to expand its effectiveness in earlier lines of therapy and in other cancers with IDH1 mutations.