Last reviewed 2026-04-23 · How we verify

AG120

Inhibits mutant IDH1 enzyme

Inhibits mutant IDH1 enzyme Used for Acute myeloid leukemia (AML) with isocitrate dehydrogenase 1 (IDH1) mutation.

At a glance

Mechanism of action

AG-120 is an inhibitor of the mutant isocitrate dehydrogenase 1 (IDH1) enzyme, which is a key driver of cancer cell growth and survival in certain types of cancer.

Approved indications

• Acute myeloid leukemia (AML) with isocitrate dehydrogenase 1 (IDH1) mutation

Common side effects

• Fatigue
• Nausea
• Diarrhea

Key clinical trials

• Ivosidenib in Treating Patients With Advanced Solid Tumors, Lymphoma, or Histiocytic Disorders With IDH1 Mutations (A Pediatric MATCH Treatment Trial) (PHASE2)
• A Study of Gilteritinib in Combination With Ivosidenib or Enasidenib in People With Acute Myeloid Leukemia (AML) (PHASE1)
• Phase II Study Evaluating Ivosidenib Maintenance After SOC Adjuvant Chemotherapy in Curative mIDH1 Cholangiocarcinoma (PHASE2)
• Ivosidenib and Venetoclax With or Without Azacitidine in Treating Patients With IDH1 Mutated Hematologic Malignancies (PHASE1, PHASE2)
• Study Evaluating the Efficacy and Safety of the Addition of Ivosidenib to Oral Azacitidine (Onureg®) in Patients Over 55 With Acute Myeloid Leukemia (AML) and IDH1 Mutation, in Complete Remission After Intensive Chemotherapy. (PHASE2)
• IMPACT-AML: A Randomized Pragmatic Clinical Trial for Relapsed or Refractory Acute Myeloid Leukemia. (PHASE3)
• Ivosidenib as Post-HSCT Maintenance for AML (PHASE2)
• A Study of the Combination of Ivosidenib, Azacitidine, and Venetoclax Followed by Ivosidenib Alone in People With Acute Myeloid Leukemia (PHASE2)

Primary sources

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