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AG-120
AG-120 is an isocitrate dehydrogenase 1 (IDH1) inhibitor that blocks the production of the oncometabolite 2-hydroxyglutarate in IDH1-mutant cancers.
AG-120 is an isocitrate dehydrogenase 1 (IDH1) inhibitor that blocks the production of the oncometabolite 2-hydroxyglutarate in IDH1-mutant cancers. Used for Acute myeloid leukemia (AML) with IDH1 mutation, Myelodysplastic syndrome (MDS) with IDH1 mutation.
At a glance
| Generic name | AG-120 |
|---|---|
| Also known as | Ivosidenib |
| Sponsor | Institut de Recherches Internationales Servier |
| Drug class | IDH1 inhibitor |
| Target | IDH1 (isocitrate dehydrogenase 1) |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | Phase 3 |
Mechanism of action
AG-120 selectively inhibits mutant IDH1, preventing the accumulation of 2-hydroxyglutarate (2-HG), which drives leukemic transformation and blocks cellular differentiation. By restoring normal metabolism and differentiation pathways, the drug allows myeloid maturation and reduces leukemic burden in IDH1-mutant hematologic malignancies.
Approved indications
- Acute myeloid leukemia (AML) with IDH1 mutation
- Myelodysplastic syndrome (MDS) with IDH1 mutation
Common side effects
- Differentiation syndrome
- Nausea
- Fatigue
- Diarrhea
- Elevated transaminases
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
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- AG-120 CI brief — competitive landscape report
- AG-120 updates RSS · CI watch RSS
- Institut de Recherches Internationales Servier portfolio CI