Drugs / Enasidenib
Enasidenib
also known as
AG-221 · CC-90007 · AG-221 mesylate · CC-90007 Free Base · Enasidenib mesilate · ENASIDENIB MESYLATE · Enasidenib methanesulfonate · Idhifa
Small molecule targets IDH2 via inhibition
Regulatory milestones approvals, filings & regulatory actions · 3 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed acute megakaryoblastic leukemia in adult 1 milestone | |||||
| Filed | US (FDA) | IDHIFA | acute megakaryoblastic leukemia in adult | — | “A New Drug Application (NDA) was filed with the U.S. Food and Drug Administration ( FDA ) for IDHIFA ® (enasidenib)” agios.com ↗ “Supported Celgene’s submission of an NDA for enasidenib in IDH2m positive R/R AML.” agios.com ↗ “Supported Celgene’s submission of an NDA for enasidenib in IDH2m positive R/R AML.” agios.com ↗ |
| Approved Indication not stated 2 milestones | |||||
| Label expansion | US (FDA) | IDHIFA | — | 2020-11-24 | fda.gov ↗ |
| Approved | US (FDA) | IDHIFA | — | 2017-08-01 | fda.gov ↗ |
Trials 43
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| 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| Phase 1 | NCT04655391 | Jun 2022 → Dec 2023 | acute myeloid leukemia | City of Hope Medical Center | Withdrawn | No outcome recorded |
| Phase 1 | NCT04955938 | Oct 2021 → Jun 2023 | myeloproliferative neoplasm | University of Chicago | Withdrawn | No outcome recorded Stop: Enrollment |
| 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 Stop: Enrollment |
| 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 | NCT03720366 | Oct 2018 → Sep 2023 | acute myeloid leukemia | Celgene Corporation | Completed | No outcome recorded |
| Phase 1 | NCT03515512 | Jul 2018 → Dec 2021 | acute myeloid leukemia, chronic myelomonocytic leukemia | Massachusetts General Hospital | Completed | No outcome recorded |
| Phase 1 | NCT03290443 | Sep 2017 → Oct 2018 | — | Celgene Corporation | Completed | No outcome recorded |
| Phase 1 | NCT02632708 | Dec 2015 → Dec 2018 overdue | therapy related acute myeloid leukemia and myelodysplastic syndrome | Servier | Active not recruiting | No outcome recorded |
| Phase 1 | NCT02443168 | May → Jul 2015 | — | Celgene Corporation | Completed | No outcome recorded |
| Phase 1 | NCT02387866 | Mar → May 2015 | — | Celgene Corporation | Completed | No outcome recorded |
| Phase 1/28 trials · 1 met primary | ||||||
| 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 Stop: Business |
| Phase 1/2 | NCT03732703 | Apr 2019 → Dec 2024 | refractory plasma cell neoplasm | Multiple Myeloma Research Consortium | Completed | 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 | Met primary |
| 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 | 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 2 | NCT03881735 | Dec 2019 → Nov 2021 | acute myeloid leukemia | Roswell Park Cancer Institute | Withdrawn | No outcome recorded Stop: Enrollment |
| Phase 2 | NCT03728335 | Jul 2019 → Jan 2027 expected | acute myeloid leukemia | City of Hope Medical Center | Recruiting | No outcome recorded |
| Phase 2 | NCT03825796 | Apr 2019 → Sep 2024 overdue | acute myeloid leukemia | Jonsson Comprehensive Cancer Center | Active not recruiting | No outcome recorded |
| Phase 2 | NCT03744390 | Apr 2019 → Feb 2023 overdue | acute myeloid leukemia, myelodysplastic syndrome | Groupe Francophone des Myelodysplasies | Active not recruiting | No outcome recorded |
| Phase 2 | NCT03683433 | Sep 2018 → Sep 2027 expected | acute biphenotypic leukemia, bilineal acute myeloid leukemia, chronic myelomonocytic leukemia, myelodysplastic syndrome | M.D. Anderson Cancer Center | Recruiting | No outcome recorded |
| Phase 2 | NCT03383575 | Jan 2018 → Feb 2027 expected | acute myeloid leukemia, chronic myelomonocytic leukemia, myelodysplastic syndrome with excess blasts | M.D. Anderson Cancer Center | Recruiting | No outcome recorded |
| Phase 2/31 trial | ||||||
| Phase 2/3 | NCT03013998 | Nov 2016 → Dec 2032 expected | acute myeloid leukemia | Beat AML, LLC | Recruiting | No outcome recorded |
| Phase 32 trials · 1 missed | ||||||
| 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 | Missed primary |
| Comparator or background therapy3 trials | ||||||
| Phase 1 | NCT02218346 Comparator | Aug → Dec 2014 | — | Agios Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 3 | NCT02665065 Comparator | Jun 2016 → Jun 2022 overdue | acute myeloid leukemia | Actinium Pharmaceuticals | Active not recruiting | Met primary |
| — | NCT03723057 Background | — | acute myeloid leukemia | Celgene Corporation | No longer available | No outcome recorded |
News releases announcing trial results or a regulatory action · 23
| Date | Issuer | Release |
|---|---|---|
| 2020-11-11 | Bristol-Myers Squibb | Results Bristol Myers Squibb Showcases Research Advancing Outcomes Addressing Hard-to-Treat Blood Cancers and Diseases Across Small Molecule, Biologic and Cell Therapies at ASH 2020 bms.com ↗
Real-World Use of Enasidenib in Relapsed or Refractory (RR) Acute Myeloid Leukemia (AML) Is Associated with Reduced Risk of Disease Progression and Death |
| 2020-08-25 | Bristol-Myers Squibb | Results Bristol Myers Squibb Provides Update on Phase 3 IDHENTIFY Trial in Patients with Relapsed or Refractory Acute Myeloid Leukemia bms.com ↗
did not meet the primary endpoint of overall survival (OS) |
| 2020-05-11 | Bristol-Myers Squibb | Results Bristol Myers Squibb Research at ASCO Demonstrates Diverse Approaches in Treating Cancer to Improve Outcomes for Patients bms.com ↗
Enasidenib plus azacitidine significantly improves complete remission and OR vs azacitidine alone in patients with newly diagnosed AML with isocitrate dehydrogenase 2 (IDH2) mutations: Results of a randomized Phase 2 study |
| 2019-11-06 | Agios Pharmaceuticals, Inc. | Results Agios to Present Broad Set of Clinical and Translational Data for Oncology and Rare Genetic Disease Programs at the 2019 ASH Annual Meeting agios.com ↗ |
| 2018-12-03 | Agios Pharmaceuticals, Inc. | Results Agios Announces Updated Data from Phase 1 Study of Ivosidenib or Enasidenib in Combination with Standard Induction and Consolidation Chemotherapy in Newly Diagnosed AML Patients With an IDH Mutation agios.com ↗ |
| 2018-11-20 | AbbVie | Results AbbVie to Highlight Breadth of Innovative Science and Ongoing Commitment to Blood Cancer Patients with New Data From Studies of Ibrutinib and Venetoclax at 2018 American Society of Hematology Annual Meeting & Exposition abbvie.com ↗
Combination of Enasidenib and Venetoclax Shows Superior Anti-Leukemic Activity Against IDH2 Mutated AML in Patient-Derived Xenograft Models |
| 2018-11-01 | Agios Pharmaceuticals, Inc. | Results Agios to Present Updated Clinical Data at the 2018 ASH Annual Meeting agios.com ↗
Ivosidenib or Enasidenib Combined with Induction and Consolidation Chemotherapy in Patients with Newly Diagnosed AML with an IDH1 or IDH2 Mutation is Safe, Effective, and Leads to MRD-Negative Complete Remissions |
| 2018-06-04 | Agios Pharmaceuticals, Inc. | Results New Data from Phase 1 Study of Ivosidenib or Enasidenib in Combination with Azacitidine Demonstrate Robust Responses and a Well Tolerated Safety Profile in Newly Diagnosed IDHm AML Patients agios.com ↗
Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, today presented encouraging new data from a Phase 1 study evaluating ivosidenib (AG-120) or enasidenib (IDHIFA®; AG-221) in combination with azacitidine in newly diagnosed isocitrate dehydrogenase (IDH) mutant acute myeloid leukemia (AML) patients. |
| 2017-12-11 | Agios Pharmaceuticals, Inc. | Results Data from Phase 1 Studies of Ivosidenib or Enasidenib in Combination with Full Doses of Standard of Care Chemotherapy Demonstrate Tolerability and Preliminary Clinical Activity in Newly Diagnosed AML Patients With an IDH Mutation agios.com ↗
The Phase 1 frontline combination trials showed that ivosidenib and enasidenib are well tolerated when combined with standard induction chemotherapy or azacitadine and both trials demonstrated early encouraging signs of efficacy. |
| 2017-08-01 | Agios Pharmaceuticals, Inc. | Regulatory FDA Grants Approval of IDHIFA®, the First Oral Targeted Therapy for Adult Patients with Relapsed/Refractory Acute Myeloid Leukemia and an IDH2 Mutation agios.com ↗
IDHIFA® (enasidenib) was granted approval from the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with relapsed or refractory AML (R/R AML) with an isocitrate dehydrogenase-2 (IDH2) mutation as detected by an FDA approved test. |
| 2017-06-24 | Agios Pharmaceuticals, Inc. | Results New Data from Phase 1/2 Trial of Oral IDHIFA® (enasidenib) Demonstrate Durable Complete Responses in Patients with IDH2 Mutant Relapsed or Refractory AML agios.com ↗ |
| 2017-06-06 | Agios Pharmaceuticals, Inc. | Results Updated Data from Phase 1 Trial of Oral IDHIFA® (enasidenib) Demonstrate Complete Responses and Duration of Response in Patients with Relapsed or Refractory AML and an IDH2 Mutation agios.com ↗
IDHIFA is an investigational first-in-class, oral, targeted inhibitor of the mutant IDH2 enzyme, which demonstrated an overall response rate of 40.3 percent, including a complete response rate of 19.3 percent in the study. |
| 2017-03-01 | Agios Pharmaceuticals, Inc. | Regulatory FDA Accepts New Drug Application and Grants Priority Review for Enasidenib in Relapsed or Refractory AML with an IDH2 Mutation agios.com ↗
The NDA was granted Priority Review and has been given a Prescription Drug User Fee Act (PDUFA) action date of Aug. 30, 2017. |
| 2016-11-03 | Agios Pharmaceuticals, Inc. | Results Agios to Present New Data from PKR and IDH Programs at the 2016 ASH Annual Meeting agios.com ↗
Enasidenib (AG-221), a Potent Oral Inhibitor of Mutant Isocitrate Dehydrogenase 2 (IDH2) Enzyme, Induces Hematologic Responses in Patients with Myelodysplastic Syndromes (MDS) |
| 2015-12-06 | Agios Pharmaceuticals, Inc. | Results Agios Announces Data from Ongoing Phase 1/2 Trial of AG-221 Showing Durable Responses in Patients with Advanced Hematologic Malignancies agios.com ↗
In patients with relapsed or refractory AML, the study had an overall response rate of 37 percent (59 of 159 response-evaluable patients) and a complete remission rate of 18 percent (29 of 159 response-evaluable patients). |
| 2015-06-12 | Agios Pharmaceuticals, Inc. | Results Agios Announces New Data from Ongoing Phase 1 Dose Escalation and Expansion Trial of AG-221 Showing Durable Clinical Activity in Patients with Advanced Hematologic Malignancies agios.com ↗
The study had an overall response rate of 40 percent (63 of 158 response-evaluable patients, using the criteria below) and a complete remission rate of 16 percent (26 of 158 response-evaluable patients). |
| 2014-12-07 | Agios Pharmaceuticals, Inc. | Results Agios Announces New Data from Ongoing Phase 1 Trial of AG-221 Showing Robust Clinical Activity in Patients with Advanced Hematologic Malignancies agios.com ↗ |
| 2014-11-06 | Agios Pharmaceuticals, Inc. | Results Agios Pharmaceuticals to Present Clinical and Preclinical Data at the 2014 American Society of Hematology Annual Meeting agios.com ↗
Data included in the abstract from our ongoing Phase 1 study of AG-221 in patients with advanced IDH2 mutant-positive hematologic malignancies show further evidence of safety and durable clinical activity among patients who desperately need new and better treatment options. |
| 2014-08-13 | Agios Pharmaceuticals, Inc. | Regulatory Agios Pharmaceuticals Announces the U.S. FDA Grants Fast Track Designation to AG-221 for Treatment of Patients With Acute Myelogenous Leukemia That Harbor an IDH2 Mutation agios.com ↗
the U.S. Food and Drug Administration ( FDA ) has granted Fast Track designation to AG-221 for the treatment of patients with acute myelogenous leukemia (AML) that harbor an isocitrate dehydrogenase-2 (IDH2) mutation. |
| 2014-06-16 | Agios Pharmaceuticals, Inc. | Regulatory Agios Pharmaceuticals Announces Orphan Drug Designation of AG-221 for Treatment of Acute Myelogenous Leukemia agios.com ↗
today announced that the U.S. Food and Drug Administration ( FDA ) has granted the company orphan drug designation for AG-221 for treatment of patients with acute myelogenous leukemia (AML). |
| 2014-06-14 | Agios Pharmaceuticals, Inc. | Results New Agios Clinical Data from Ongoing Phase 1 Trial of AG-221 Continue to Show Complete and Durable Remissions in Patients with Difficult to Treat Hematologic Malignancies agios.com ↗
The new data show objective responses in 14 out of 25 evaluable patients on AG-221 and an additional five patients with stable disease. |
| 2014-04-06 | Agios Pharmaceuticals, Inc. | Results Agios Announces Initial Phase 1 Data Demonstrating Clinical Activity of AG-221, First-in-Class Inhibitor of IDH2 Mutations, in Patients with Advanced Blood Cancers agios.com ↗
These preliminary data demonstrate the clinical activity, tolerability and unique mechanism of action of AG-221 in patients with advanced hematologic malignancies with an isocitrate dehydrogenase-2 (IDH2) mutation. |
| 2013-12-09 | Agios Pharmaceuticals, Inc. | Results Agios Presents Preclinical Data from Lead Programs at American Society of Hematology Annual Meeting agios.com ↗
“AG-221 offers a survival advantage in a primary human IDH2 mutant AML xenograft model,” an oral presentation, provides strong preclinical in vivo evidence of AG-221’s potential clinical benefit for patients with tumors that harbor an IDH2 mutation. |
All press releases naming this drug 32 releases
Evidence & citations 13 cited values
Every value below carries the sentence it was read from. 50 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Enasidenib | “Enasidenib” NCT02665065 ↗ |
| 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 ↗ |
| 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 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 |