Therapy Detail

Therapy Name Enasidenib
Therapy Description

Idhifa (enasidenib) inhibits IDH2 to prevent 2HG formation, which may promote differentiation and inhibit proliferation of cancer cells (PMID: 28280273). Idhifa (enasidenib) is FDA-approved for use in patients with relapsed or refractory acute myeloid leukemia harboring an IDH2 mutation (FDA.gov).

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Drug Name Trade Name Synonyms Drug Classes Drug Description
Enasidenib Idhifa AG-221 IDH2 Inhibitor 2 Idhifa (enasidenib) inhibits IDH2 to prevent 2HG formation, which may promote differentiation and inhibit proliferation of cancer cells (PMID: 28280273). Idhifa (enasidenib) is FDA-approved for use in patients with relapsed or refractory acute myeloid leukemia harboring an IDH2 mutation (FDA.gov).
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
IDH1 R132C IDH1 S280F acute myeloid leukemia resistant Enasidenib Clinical Study Actionable In a clinical study, IDH1 S280F was identified as an acquired mutation in cis with the original IDH1 R132C in a patient with acute myeloid leukemia (AML) who developed resistance to Idhifa (enasidenib) after initial response (PMID: 29950729). 29950729
IDH2 R140Q acute myeloid leukemia sensitive Enasidenib Preclinical - Pdx & cell culture Actionable In a preclinical study, Enasidenib (AG-221) treatment reduced 2-hydroxyglutarate (2HG) levels and induced differentiation of leukemic blasts from acute myeloid leukemia (AML) patients harboring IDH2 R140Q in culture, and decreased 2HG levels and blast percentage and improved survival relative to Cytosar-U (cytarabine) treatment in IDH2 R140Q-mutant primary AML xenograft models (PMID: 28193778). 28193778
IDH2 R172K colorectal cancer predicted - sensitive Enasidenib Preclinical - Cell culture Actionable In a preclinical study, Enasidenib (AG-221) decreased 2-hydroxyglutarate (2HG) levels in a colorectal carcinoma cell line expressing IDH2 R172K in culture (PMID: 28193778). 28193778
IDH2 R172K acute myeloid leukemia sensitive Enasidenib Preclinical - Patient cell culture Actionable In a preclinical study, Enasidenib (AG-221) decreased 2-hydroxyglutarate (2HG) levels and induced differentiation of leukemic blasts from acute myeloid leukemia (AML) patients harboring IDH2 R172K in culture (PMID: 28193778). 28193778
IDH2 R140Q IDH2 Q316E acute myeloid leukemia resistant Enasidenib Clinical Study Actionable In a clinical study, IDH2 Q316E was identified as an acquired mutation in trans with the original IDH2 R140Q in a patient with acute myeloid leukemia (AML) who developed resistance to Idhifa (enasidenib) after initial response, coexpression of IDH2 Q316E in trans or in cis with IDH2 R140Q conferred resistance to Idhifa (enasidenib) in culture and in animal models of AML, supporting a mechanism of variants cooperating to confer resistance (PMID: 29950729). 29950729
IDH2 mutant hematologic cancer sensitive Enasidenib Phase I Actionable In a Phase I study, Enasidenib (AG-221) demonstrated safety and efficacy in patients with hematological cancer harboring IDH2 mutations and included 8 CR, 1 CRp, 3 CRi, and 8 PR (ASH Meeting, Dec 2014, abstract #115). detail...
IDH2 mutant acute myeloid leukemia sensitive Enasidenib Guideline Actionable Idhifa (enasidenib) is included in guidelines for patients with relapsed or refractory acute myeloid leukemia harboring an IDH2 mutation (NCCN.org). detail...
IDH2 mutant acute myeloid leukemia sensitive Enasidenib FDA approved Actionable In a Phase I/II trial that supported FDA approval, Idhifa (enasidenib) treatment resulted in an overall response rate of 40.3% (71/176) with a median response duration of 5.8 months, complete remission in 19.3% (34/176), and stable disease in 48.3% (85/176) of acute myeloid leukemia patients harboring IDH2 mutations (PMID: 28588020; NCT01915498). 28588020
IDH2 R140Q glioblastoma multiforme predicted - sensitive Enasidenib Preclinical - Cell line xenograft Actionable In a preclinical study, Enasidenib (AG-221) decreased 2-hydroxyglutarate (2HG) levels in a glioblastoma cell line expressing IDH2 R140Q in culture and in xenograft models (PMID: 28193778). 28193778
IDH2 R172K glioblastoma multiforme predicted - sensitive Enasidenib Preclinical - Cell culture Actionable In a preclinical study, Enasidenib (AG-221) decreased 2-hydroxyglutarate (2HG) levels in a glioblastoma cell line expressing IDH2 R172K in culture (PMID: 28193778). 28193778
IDH2 R140Q IDH2 I139M acute myeloid leukemia resistant Enasidenib Clinical Study Actionable In a clinical study, IDH2 I319M was identified as an acquired mutation in trans with the original IDH2 R140Q in a patient with acute myeloid leukemia (AML) who developed resistance to Idhifa (enasidenib) after initial response, coexpression of IDH2 I319M in trans or in cis with IDH2 R140Q conferred resistance to Idhifa (enasidenib) in culture, supporting a mechanism of variants cooperating to confer resistance (PMID: 29950729). 29950729
Clinical Trial Phase Therapies Title Recruitment Status
NCT02632708 Phase I Ivosidenib Enasidenib Cytarabine + Daunorubicin + Etoposide + Mitoxantrone Safety Study of AG-120 or AG-221 in Combination With Induction and Consolidation Therapy in Patients With Newly Diagnosed Acute Myeloid Leukemia With an IDH1 and/or IDH2 Mutation Active, not recruiting
NCT02577406 Phase III Azacitidine Cytarabine Enasidenib An Efficacy and Safety Study of AG-221 (CC-90007) Versus Conventional Care Regimens in Older Subjects With Late Stage Acute Myeloid Leukemia Harboring an Isocitrate Dehydrogenase 2 Mutation (IDHENTIFY) Recruiting
NCT03728335 Phase I Enasidenib Enasidenib as Maintenance Therapy in Treating Patients With Acute Myeloid Leukemia With IDH2 Mutation After Donor Stem Cell Transplant Not yet recruiting
NCT02273739 Phase Ib/II Enasidenib Study of Orally Administered AG-221 in Subjects With Advanced Solid Tumors, Including Glioma, and With Angioimmunoblastic T-cell Lymphoma, With an IDH2 Mutation Completed
NCT01915498 Phase I Enasidenib Phase 1 Study of AG-221 in Subjects With Advanced Hematologic Malignancies With an IDH2 Mutation Active, not recruiting
NCT03383575 Phase II Azacitidine + Enasidenib Enasidenib Targeted Therapy With the IDH2-Inhibitor Enasidenib (AG221) for High-Risk IDH2-Mutant Myelodysplastic Syndrome Recruiting