Drugs / Enasidenib
last change Jul 2026 re-read 2 minutes ago

Enasidenib

Small molecule targets IDH2 via inhibition

Developed for
acute myeloid leukemia · myelodysplastic syndrome · chronic myelomonocytic leukemia · chondrosarcoma · clonal hematopoiesis of indeterminate potential · refractory malignant neoplasm · therapy related acute myeloid leukemia and myelodysplastic syndrome · adenocarcinoma
+17 more · angioimmunoblastic T-cell lymphoma · childhood malignant neoplasm · glioma · hematopoietic and lymphoid cell neoplasm · intrahepatic cholangiocarcinoma · large cell neuroendocrine carcinoma · monocytic leukemia · myelodysplastic syndrome with excess blasts · myelodysplastic syndrome with excess blasts-2 · myelofibrosis · myeloid hemopathy · myeloid leukemia · myeloproliferative neoplasm · olfactory neuroblastoma · refractory plasma cell neoplasm · sinonasal undifferentiated carcinoma · T-cell non-Hodgkin lymphoma
Investigated by
Celgene Corporation · National Cancer Institute (NCI) · M.D. Anderson Cancer Center · Bristol-Myers Squibb · City of Hope Medical Center · Memorial Sloan Kettering Cancer Center
+39 more · AbbVie · Jazz Pharmaceuticals · Agios Pharmaceuticals, Inc. · Alice Mims · Astellas Pharma US, Inc. · Beat AML, LLC · Children's Oncology Group · Damon Runyon Cancer Research Foundation · Deutsch-Österreichische Studiengruppe Akute Myeloische Leukämie (AMLSG) · Eli Lilly and Company · Fight Kids Cancer · Fondation ARC · Genentech, Inc. · GlaxoSmithKline · Groupe Francophone des Myelodysplasies · Gustave Roussy, Cancer Campus, Grand Paris · HOVON Foundation · Heinrich-Heine University, Duesseldorf · Incyte Corporation · Janssen, LP · John Mascarenhas · Jonsson Comprehensive Cancer Center · Karyopharm Therapeutics Inc · Koordinierungszentrum für Klinische Studien Düsseldorf · Massachusetts General Hospital · Multiple Myeloma Research Consortium · Myeloproliferative Neoplasms Research Consortium · National Cancer Institute, France · National Institutes of Health (NIH) · OHSU Knight Cancer Institute · Oregon Health and Science University · Roswell Park Cancer Institute · Servier · Takeda · The Leukemia and Lymphoma Society · Tian Yi Zhang · University Health Network, Toronto · University of Chicago · Washington University School of Medicine

Regulatory milestones approvals, filings & regulatory actions · 2 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
Label expansion US (FDA) IDHIFA 2020-11-24 fda.gov
Approved US (FDA) IDHIFA 2017-08-01 fda.gov

Trials 43 · a red edge is where a trial was stopped

PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 116 trials
Phase 1 NCT05756777 Oct 2023 → Apr 2027 expected acute myeloid leukemia Memorial Sloan Kettering Cancer Center Recruiting No outcome recorded
Phase 1 NCT05441514 Apr 2023 → Mar 2026 acute myeloid leukemia City of Hope Medical Center Completed No outcome recorded
Phase 1 NCT04655391 Jun 2022 → Dec 2023 acute myeloid leukemia City of Hope Medical Center Withdrawn No outcome recorded
Phase 1 NCT04955938 Oct 2021 → Jun 2023 myeloproliferative neoplasm University of Chicago Withdrawn No outcome recorded
Phase 1 NCT05010772 Oct 2021 → Dec 2026 expected acute myeloid leukemia M.D. Anderson Cancer Center Recruiting No outcome recorded
Phase 1 NCT05102370 Oct 2021 → Jan 2026 clonal hematopoiesis of indeterminate potential Memorial Sloan Kettering Cancer Center Completed No outcome recorded
Phase 1 NCT04573582 Nov 2020 → Mar 2023 Celgene Corporation Completed No outcome recorded
Phase 1 NCT03878524 Apr → Dec 2020 acute myeloid leukemia, atypical chronic myeloid leukemia, BCR-ABL1 negative, blast phase chronic myelogenous leukemia, BCR-ABL1 positive, castration-resistant prostate carcinoma +14 OHSU Knight Cancer Institute Terminated No outcome recorded
Phase 1 NCT04075747 Dec 2019 → Feb 2022 acute myeloid leukemia Jazz Pharmaceuticals Completed No outcome recorded
Phase 1 NCT04310527 Oct → Nov 2019 Celgene Corporation Completed No outcome recorded
Phase 1/29 trials
Phase 1/2 NCT05282459 Jan 2022 → Dec 2024 monocytic leukemia, myeloid leukemia Tian Yi Zhang Completed No outcome recorded
Phase 1/2 NCT04774393 May 2021 → Nov 2027 expected acute myeloid leukemia M.D. Anderson Cancer Center Recruiting No outcome recorded
Phase 1/2 NCT04092179 Nov 2020 → Oct 2023 acute myeloid leukemia, refractory malignant neoplasm University Health Network, Toronto Terminated No outcome recorded
Phase 1/2 NCT03732703 Apr 2019 → Dec 2024 refractory plasma cell neoplasm Multiple Myeloma Research Consortium Completed No outcome recorded
Phase 1/2 NCT03013998 Nov 2016 → Dec 2028 expected acute myeloid leukemia Beat AML, LLC Recruiting No outcome recorded
Phase 1/2 NCT02813135 Aug 2016 → Feb 2031 expected childhood malignant neoplasm Gustave Roussy, Cancer Campus, Grand Paris Recruiting No outcome recorded
Phase 1/2 NCT02677922 Jun 2016 → Aug 2018 overdue acute myeloid leukemia Celgene Corporation Active not recruiting No outcome recorded
Phase 1/2 NCT02273739 Dec 2014 → Jun 2016 angioimmunoblastic T-cell lymphoma, chondrosarcoma, glioma, intrahepatic cholangiocarcinoma Celgene Corporation Completed No outcome recorded
Phase 1/2 NCT01915498 Aug 2013 → Jul 2019 hematopoietic and lymphoid cell neoplasm Celgene Corporation Completed No outcome recorded
Phase 216 trials
Phase 2 NCT06577441 Jun 2025 → Mar 2027 expected myelodysplastic syndrome National Cancer Institute (NCI) Recruiting No outcome recorded
Phase 2 NCT06672146 May 2025 → Mar 2027 expected acute myeloid leukemia National Cancer Institute (NCI) Recruiting No outcome recorded
Phase 2 NCT06756308 Dec 2024 → Dec 2027 expected T-cell non-Hodgkin lymphoma Memorial Sloan Kettering Cancer Center Recruiting No outcome recorded
Phase 2 NCT06240754 Oct 2024 → Jan 2028 expected clonal hematopoiesis of indeterminate potential, myeloid hemopathy Washington University School of Medicine Recruiting No outcome recorded
Phase 2 NCT05564390 Jun 2024 → May 2029 expected acute myeloid leukemia with multilineage dysplasia, myelodysplastic syndrome, therapy related acute myeloid leukemia and myelodysplastic syndrome National Cancer Institute (NCI) Recruiting No outcome recorded
Phase 2 NCT06176989 Mar 2024 → Dec 2027 expected adenocarcinoma, chondrosarcoma, large cell neuroendocrine carcinoma, olfactory neuroblastoma +1 National Cancer Institute (NCI) Recruiting No outcome recorded
Phase 2 NCT04203316 Aug 2023 → Sep 2025 overdue acute myeloid leukemia Children's Oncology Group Active not recruiting No outcome recorded
Phase 2 NCT05401097 Sep 2022 → Sep 2027 expected acute myeloid leukemia Alice Mims Recruiting No outcome recorded
Phase 2 NCT04281498 Jan 2021 → May 2023 myelofibrosis John Mascarenhas Completed No outcome recorded
Phase 2 NCT04522895 Aug 2020 → Jul 2024 acute myeloid leukemia, chronic myelomonocytic leukemia, myelodysplastic syndrome Heinrich-Heine University, Duesseldorf Completed No outcome recorded
Phase 32 trials
Phase 3 NCT03839771 Mar 2019 → Apr 2027 expected acute myeloid leukemia, myelodysplastic syndrome with excess blasts-2 HOVON Foundation Active not recruiting No outcome recorded
Phase 3 NCT02577406 Dec 2015 → Mar 2020 acute myeloid leukemia Celgene Corporation Completed No outcome recorded
Also used as a comparator or background therapy in 3 trials
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 11 trial
Phase 1 NCT02218346 Comparator Aug → Dec 2014 Agios Pharmaceuticals, Inc. Completed No outcome recorded
Phase 31 trial
Phase 3 NCT02665065 Comparator Jun 2016 → Jun 2022 overdue acute myeloid leukemia Actinium Pharmaceuticals Active not recruiting No outcome recorded
Phase not stated1 trial
NCT03723057 Background acute myeloid leukemia Celgene Corporation No longer available No outcome recorded

Evidence & citations 14 cited values

Every value below carries the sentence it was read from. 50 sources stand behind the page.

FieldValueCited text
Known as Enasidenib EnasidenibNCT02665065
35

NCT04075747

NCT05756777

NCT04203316

NCT03732703

NCT03728335

NCT03683433

NCT03515512

NCT02273739

NCT06577441

NCT01915498

NCT06176989

NCT04281498

NCT04522895

NCT05401097

NCT03720366

NCT04655391

NCT04573582

NCT05102370

NCT03383575

NCT06756308

NCT03881735

NCT03290443

NCT05010772

NCT06240754

NCT05564390

NCT04092179

NCT03825796

NCT04774393

NCT04955938

NCT06672146

NCT05282459

NCT04310527

NCT05441514

NCT03878524

NCT02813135

Known as AG-221 “Phase 2 Trial of Enasidenib (AG-221) Maintenance Post Allogeneic Hematopoietic Cell Transplantation in Patients With IDH2 Mutation” NCT03728335
10

AG-221 is a first-in-class drug targeted specifically to inhibit the IDH2 enzyme.” NCT02218346

NCT02677922

NCT03723057

NCT03013998

NCT02632708

NCT02387866

NCT03744390

NCT02577406

NCT02443168

NCT03839771

Known as AG-221 mesylate ChEMBL registry synonym — accepted as the source's own label CHEMBL3989908
Known as CC-90007 “Bioavailability of Enasidenib (CC-90007) Sprinkle Formulation Relative to the Reference Tablet Formulation and Effect of Food on the Pharmacokinetics of Sprinkle Formulation in...” NCT04310527
1

“AG-221 (CC-90007) Versus Conventional Care Regimens” NCT02577406

Known as CC-90007 Free Base ChEMBL registry synonym — accepted as the source's own label CHEMBL3989908
Known as Enasidenib mesilate ChEMBL registry synonym — accepted as the source's own label CHEMBL3989908
Known as Enasidenib mesylat Enasidenib mesylat dose escalation” NCT05282459
Known as ENASIDENIB MESYLATE ChEMBL registry synonym — accepted as the source's own label CHEMBL3989908
Known as Enasidenib methanesulfonate ChEMBL registry synonym — accepted as the source's own label CHEMBL3989908
Known as Idhifa ChEMBL registry synonym — accepted as the source's own label CHEMBL3989908
Action Inhibit “Enasidenib, an inhibitor of mutated IDH2 proteins, induces responses in patients with IDH2-mutated, relapsed or refractory acute myeloid leukaemia.” PMID 32145771 Mar 2020
Modality Small molecule “Enasidenib is an oral, small-molecule inhibitor of mIDH2 proteins.” PMID 29243965 Sep 2017
Route Oral “Enasidenib tablets administered orally every day of 28-day treatment cycles until disease progression or unacceptable toxicities.” NCT01915498
Target IDH2 “Enasidenib (AG-221) is an allosteric inhibitor that binds to the IDH2 dimer interface and blocks the production of 2HG by IDH2 mutants9,10.” PMID 29950729 Jun 2018