marked.bio

IDH2 inhibitor · small molecule

enasidenib

13 active trials across 2 of our biomarkers, targeting IDH2. 1 active approval for a biomarker on marked.bio.

Also known as AG-221, CC-90007, Idhifa.

Counts only trials that select patients by one of the biomarkers on marked.bio, not every trial of this drug.

Approvals and pivotal results

Biomarker-selected approvals in the United States and the European Union, with the pivotal trial's results and side effects where we have them. Results from different trials are not directly comparable.

TherapyBiomarkerTumourUnited StatesEuropean Union
enasidenib
FDA, 2017
relapsed or refractory, IDH2 mutation FDA approval 01/08/2017; companion test Abbott RealTime IDH2 Assay. Boxed warning: differentiation syndrome. Source
Idhifa 		IDH1/2 mutation AML Approved 2017 Not approved

AG221-C-001 single-arm, 199 patients
CR+CRh 23%Lasting 8.2 months

Trial details and side effects
Patients
relapsed or refractory IDH2-mutant AML (NCT01915498)
Most common side effects
nausea, vomiting, diarrhoea, raised bilirubin, decreased appetite
Notes
FDA approval as reported by ESMO (2017); differentiation syndrome in 14%.
Source
US approval report

Biomarkers

BiomarkerActive trialsAll trialsFurthest active phase
IDH1/2 mutation 13 25 Phase 3
FLT3 mutation (ITD/TKD) 1 2 Phase 1

Trials

13 of 25 trials shown

  1. A Study of Ivosidenib or Enasidenib in Combination With Induction Therapy and Consolidation Therapy, Followed by Maintenance Therapy in Patients With Newly Diagnosed Acute Myeloid Leukemia or Myedysplastic Syndrome EB2, With an IDH1 or IDH2 Mutation, Respectively, Eligible for Intensive Chemotherapy

    Active, not recruiting Phase 3 Stichting Hemato-Oncologie voor Volwassenen Nederland Started 2019Primary completion 2027-04 NCT03839771

    ivosidenib FDA EUenasidenib FDA

    Newly diagnosed AML or MDS-EB2 defined according to WHO criteria, with a documented IDH1 or IDH2 gene mutation (as determined by the clinical trial assay) at a specific site (IDH1 R132, IDH2 R140, IDH…

    IDH1/2 mutationAcute Myeloid Leukemia, Myelodysplastic Syndrome With Excess Blasts-2

  2. Comparing New Treatments for People With Newly Diagnosed Acute Myeloid Leukemia That Has an IDH2 Gene Change (A MyeloMATCH Treatment Trial)

    Recruiting Phase 2 National Cancer Institute (NCI) Started 2025Primary completion 2027-03 NCT06672146

    decitabineenasidenib FDAvenetoclax

    …x Compared With ASTX727, Venetoclax, and Enasidenib for Newly Diagnosed Older Adults With IDH2 Mutant Acute Myeloid Leukemia: A MyeloMATCH Substudy

    IDH1/2 mutationAcute Myeloid Leukemia

  3. Testing the Addition of an IDH2 Inhibitor, Enasidenib, to Usual Treatment (Cedazuridine-Decitabine) for Higher-Risk Myelodysplastic Syndrome (MDS) With IDH2 Mutation (A MyeloMATCH Treatment Trial)

    Recruiting Phase 2 National Cancer Institute (NCI) Started 2025Primary completion 2027-03 NCT06577441

    decitabineenasidenib FDA

    REGISTRATION ELIGIBILITY CRITERIA (STEP 1): Patients must have a detectable pathogenic IDH2 mutation based on the National Cancer Institute (NCI) Myeloid Panel.

    IDH1/2 mutationMyelodysplastic Syndrome

  4. Enasidenib in IDH2-Mutated Malignant Sinonasal and Skull Base Tumors

    Recruiting Phase 2 National Cancer Institute (NCI) Started 2024Primary completion 2027-12 NCT06176989

    enasidenib FDA

    …lly advanced or metastatic SNUC, ONB, LCNEC, SNAC, and CS with documented somatic (tumor) IDH2 mutations R140 or R172.

    IDH1/2 mutationMetastatic Chondrosarcoma, Locally Advanced Chondrosarcoma, Metastatic Sinonasal Adenocarcinoma, Locally Advanced Sinonasal Adenocarcinoma

  5. A Study of Enasidenib in People With T-Cell Lymphoma

    Active, not recruiting Phase 2 Memorial Sloan Kettering Cancer Center Started 2024Primary completion 2027-12 NCT06756308

    enasidenib FDArituximab

    Pathologically-confirmed AITL at the enrolling institution, with confirmed IDH2 mutation (by MSK ddPCR).

    IDH1/2 mutationT-cell Lymphoma

  6. Enasidenib for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia Patients With an IDH2 Mutation

    Active, not recruiting Phase 2 Children's Oncology Group Started 2023Primary completion 2025-09 NCT04203316

    enasidenib FDA

    Patient must have AML with an IDH2 mutation identified from a peripheral blood or bone marrow sample at the time of diagnosis and/or …

    IDH1/2 mutationRecurrent Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia

  7. IDH Targeted/Non- Targeted vs Non-targeted/IDH-targeted Approaches in the Treatment of Newly Diagnosed IDH Mutated AML Patients Not Candidates for Intensive Induction Therapy (I- DATA Study)

    Recruiting Phase 2 Alice Mims Started 2022Primary completion 2027-09 NCT05401097

    azacitidineenasidenib FDAivosidenib FDA EUvenetoclax

    Patients with newly diagnosed IDH1 or IDH2 mutated AML

    IDH1/2 mutationAcute Myeloid Leukemia

  8. Decitabine/Cedazuridine and Venetoclax in Combination With Ivosidenib or Enasidenib for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia

    Recruiting Phase 1/2 M.D. Anderson Cancer Center Started 2021Primary completion 2027-11 NCT04774393

    decitabineenasidenib FDAivosidenib FDA EUvenetoclax

    Subjects must have documented IDH1 or IDH2 gene mutation

    IDH1/2 mutationAcute Myeloid Leukemia, Recurrent Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia

  9. IDH2 (AG 221) Inhibitor in Patients With IDH2 Mutated Myelodysplastic Syndrome

    Active, not recruiting Phase 2 Groupe Francophone des Myelodysplasies Started 2019Primary completion 2023-02 NCT03744390

    enasidenib FDA

    Presence of IDH2 mutation in either blood or marrow prior to start of therapy

    IDH1/2 mutationMyelodysplastic Syndromes, Leukemia Acute Myeloid

  10. Azacitidine and Enasidenib in Treating Patients With IDH2-Mutant Myelodysplastic Syndrome

    Recruiting Phase 2 M.D. Anderson Cancer Center Started 2018Primary completion 2027-02 NCT03383575

    azacitidineenasidenib FDA

    Subjects must have an IDH2 gene mutation (IDH2-R140 or R172) as determined by local laboratory result

    IDH1/2 mutationAcute Myeloid Leukemia, Blasts 20-30 Percent of Bone Marrow Nucleated Cells, Chronic Myelomonocytic Leukemia, IDH2 Gene Mutation

  11. Enasidenib and Azacitidine in Treating Patients With Recurrent or Refractory Acute Myeloid Leukemia and IDH2 Gene Mutation

    Recruiting Phase 2 M.D. Anderson Cancer Center Started 2018Primary completion 2027-09 NCT03683433

    azacitidineenasidenib FDA

    Subjects must have documented IDH2 gene mutation

    IDH1/2 mutationAcute Bilineal Leukemia, Acute Biphenotypic Leukemia, Chronic Myelomonocytic Leukemia, IDH2 Gene Mutation

  12. A Study of Gilteritinib in Combination With Ivosidenib or Enasidenib in People With Acute Myeloid Leukemia (AML)

    Active, not recruiting Phase 1 Memorial Sloan Kettering Cancer Center Started 2023Primary completion 2027-04 NCT05756777

    gilteritinibivosidenib FDA EUenasidenib FDA

    Patient has relapsed or refractory AML with dually mutant IDH2/FLT3, IDH1/FLT3 (ITD or TKD) , or other FLT3 mutation sensitive to gilteritinib.

    IDH1/2 mutationFLT3 mutation (ITD/TKD)Acute Myeloid Leukemia (AML)

  13. Safety Study of AG-120 or AG-221 in Combination With Induction and Consolidation Therapy in Participants With Newly Diagnosed Acute Myeloid Leukemia (AML) With an IDH1 and/or IDH2 Mutation

    Active, not recruiting Phase 1 Institut de Recherches Internationales Servier Started 2015Primary completion 2018-12 NCT02632708

    ivosidenib FDA EUenasidenib FDAcytarabinedaunorubicinidarubicinmitoxantroneetoposide

    …e promyelocytic leukemia (APL) \[AML with t(15;17)\], with locally documented IDH1 and/or IDH2 gene mutation scheduled for induction therapy followed by consolidation therapy.

    IDH1/2 mutationNewly Diagnosed Acute Myeloid Leukemia (AML), Untreated AML, AML Arising From Myelodysplastic Syndrome (MDS), AML Arising From Antecedent Hematologic Disorder (AHD)