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

Pevonedistat

Small molecule targets MCL1 via inhibition

Developed for
acute myeloid leukemia · myelodysplastic syndrome · chronic myelomonocytic leukemia · high grade malignant neoplasm · acute lymphoblastic leukemia · diffuse large B-cell lymphoma · myelofibrosis · plasma cell myeloma
+21 more · therapy related acute myeloid leukemia and myelodysplastic syndrome · acute myeloid leukemia with multilineage dysplasia · B-cell prolymphocytic leukemia · cancer · cancer or benign tumor · central nervous system neoplasm · chronic lymphocytic leukemia/small lymphocytic lymphoma · follicular lymphoma · Hodgkins lymphoma · intrahepatic cholangiocarcinoma · lymphoma · lymphoplasmacytic lymphoma · mantle cell lymphoma · marginal zone lymphoma · mesothelioma · metastatic malignant neoplasm · metastatic melanoma · myeloproliferative neoplasm · non-small cell lung carcinoma · non-small cell squamous lung carcinoma · Richter transformation
Investigated by
Millennium Pharmaceuticals, Inc. · National Cancer Institute (NCI) · Takeda · M.D. Anderson Cancer Center · Children's Oncology Group · City of Hope Medical Center
+14 more · Beat AML, LLC · Dynamic Science S.L. · Justin Watts, MD · Medical College of Wisconsin · Memorial Sloan Kettering Cancer Center · Milton S. Hershey Medical Center · PETHEMA Foundation · Takeda Development Center Americas, Inc. · University of Leipzig · University of Miami · University of Michigan Rogel Cancer Center · University of Southern California · Vanderbilt-Ingram Cancer Center · Washington University School of Medicine

Trials 41 · a red edge is where a trial was stopped

PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 122 trials
Phase 1 NCT03770260 Feb 2020 → Sep 2022 plasma cell myeloma National Cancer Institute (NCI) Completed No outcome recorded
Phase 1 NCT04172844 Jan 2020 → Dec 2021 acute myeloid leukemia Medical College of Wisconsin Terminated No outcome recorded
Phase 1 NCT03814005 Jul 2019 → Mar 2021 acute myeloid leukemia, chronic myelomonocytic leukemia, impaired renal function disease, liver disorder +1 Takeda Completed No outcome recorded
Phase 1 NCT03772925 Jun 2019 → Sep 2022 acute myeloid leukemia, myelodysplastic syndrome National Cancer Institute (NCI) Terminated No outcome recorded
Phase 1 NCT03813147 May 2019 → Sep 2021 acute myeloid leukemia, myelodysplastic syndrome National Cancer Institute (NCI) Completed No outcome recorded
Phase 1 NCT03349281 Mar 2019 → Aug 2021 acute lymphoblastic leukemia University of Miami Completed No outcome recorded
Phase 1 NCT03486314 Aug 2018 → May 2019 high grade malignant neoplasm Millennium Pharmaceuticals, Inc. Completed No outcome recorded
Phase 1 NCT03459859 May 2018 → Jun 2021 acute myeloid leukemia, myelodysplastic syndrome Justin Watts, MD Completed No outcome recorded
Phase 1 NCT03479268 Apr 2018 → Nov 2026 expected B-cell prolymphocytic leukemia, Richter transformation, chronic lymphocytic leukemia/small lymphocytic lymphoma, diffuse large B-cell lymphoma +4 City of Hope Medical Center Active not recruiting No outcome recorded
Phase 1 NCT03386214 Apr 2018 → Sep 2021 myelofibrosis Washington University School of Medicine Terminated No outcome recorded
Phase 1/26 trials
Phase 1/2 NCT04800627 Mar 2021 → Nov 2022 high grade malignant neoplasm, metastatic malignant neoplasm M.D. Anderson Cancer Center Terminated No outcome recorded
Phase 1/2 NCT03862157 Feb 2019 → Jan 2027 expected acquired polycythemia vera, acute myeloid leukemia, atypical chronic myeloid leukemia, BCR-ABL1 negative, chronic eosinophilic leukemia +7 M.D. Anderson Cancer Center Active not recruiting No outcome recorded
Phase 1/2 NCT03330821 Apr 2018 → Dec 2026 expected acute myeloid leukemia with multilineage dysplasia, therapy related acute myeloid leukemia and myelodysplastic syndrome University of Southern California Active not recruiting No outcome recorded
Phase 1/2 NCT03319537 Oct 2017 → Jun 2023 mesothelioma Memorial Sloan Kettering Cancer Center Terminated 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 NCT01415765 Jul 2011 → Jan 2014 diffuse large B-cell lymphoma National Cancer Institute (NCI) Withdrawn No outcome recorded
Phase 210 trials
Phase 2 NCT04985656 Oct 2021 → Jan 2024 myelodysplastic syndrome Takeda Withdrawn No outcome recorded
Phase 2 NCT04712942 Jan 2021 → Jan 2023 acute myeloid leukemia, myelodysplastic syndrome University of Leipzig Completed No outcome recorded
Phase 2 NCT04266795 Oct 2020 → Sep 2022 acute myeloid leukemia Takeda Completed No outcome recorded
Phase 2 NCT03965689 Apr 2020 → Sep 2022 overdue non-small cell squamous lung carcinoma National Cancer Institute (NCI) Active not recruiting No outcome recorded
Phase 2 NCT04175912 Mar 2020 → Nov 2023 overdue hepatocellular carcinoma, intrahepatic cholangiocarcinoma National Cancer Institute (NCI) Active not recruiting No outcome recorded
Phase 2 NCT03745352 May 2019 → Aug 2021 acute myeloid leukemia National Cancer Institute (NCI) Withdrawn No outcome recorded
Phase 2 NCT03709576 Jul 2018 → Jul 2019 acute myeloid leukemia Milton S. Hershey Medical Center Terminated No outcome recorded
Phase 2 NCT03228186 Mar 2018 → Nov 2021 non-small cell lung carcinoma University of Michigan Rogel Cancer Center Terminated No outcome recorded
Phase 2 NCT03238248 Aug 2017 → Sep 2022 myelodysplastic syndrome, myeloproliferative neoplasm Vanderbilt-Ingram Cancer Center Completed No outcome recorded
Phase 2 NCT02610777 Apr 2016 → Sep 2019 acute myeloid leukemia, chronic myelomonocytic leukemia, myelodysplastic syndrome Millennium Pharmaceuticals, Inc. Completed No outcome recorded
Phase 32 trials
Phase 3 NCT04090736 Sep 2019 → Jun 2023 overdue acute myeloid leukemia PETHEMA Foundation Active not recruiting No outcome recorded
Phase 3 NCT03268954 Nov 2017 → May 2021 acute myeloid leukemia, chronic myelomonocytic leukemia, myelodysplastic syndrome Takeda Completed No outcome recorded
Phase not stated1 trial
NCT04484363 myelodysplastic syndrome Takeda No longer available No outcome recorded

Press releases naming this drug 3 releases

DateIssuerRelease
2021-09-01 Takeda Takeda Provides Update on Phase 3 PANTHER (Pevonedistat-3001) Trial takeda.com
2020-07-30 Takeda Takeda Announces U.S. FDA Breakthrough Therapy Designation Granted for Pevonedistat for the Treatment of Patients with Higher-Risk Myelodysplastic Syndromes (HR-MDS) takeda.com
2020-05-29 Takeda Takeda Announces Compelling Data from the Phase 2 Trial of Pevonedistat Plus Azacitidine in Patients with Higher-Risk MDS takeda.com

Evidence & citations 13 cited values

Every value below carries the sentence it was read from. 47 sources stand behind the page.

FieldValueCited text
Known as pevonedistat Pevonedistat (MLN4924), a First-in-Class NEDD8-activating enzyme inhibitor, in patients with acute myeloid leukaemia and myelodysplastic syndromes” PMID 25733005 Mar 2015
34

pevonedistat (also known as MLN4924) is the only NEDDylation inhibitor to date to enter phase III clinical trials.” PMID 41687727 Feb 2026

“Drug: PevonedistatNCT04172844

NCT03057366

NCT02782468

NCT04266795

NCT03745352

NCT03965689

NCT04712942

NCT03709576

NCT03813147

NCT03386214

NCT03323034

NCT03486314

NCT03319537

NCT03268954

NCT03330106

NCT04175912

NCT03228186

NCT04985656

NCT02610777

NCT03770260

NCT03009240

NCT03479268

NCT03349281

NCT04090736

NCT03330821

NCT03772925

NCT03238248

NCT03013998

NCT04800627

NCT03459859

NCT03814005

NCT03862157

NCT04484363

Known as MLN-4924 HYDROCHLORIDE ChEMBL registry synonym — accepted as the source's own label CHEMBL1231160
Known as MLN-4924-003 ChEMBL registry synonym — accepted as the source's own label CHEMBL1231160
Known as MLN4924 “A Phase 1/1b, Open-label Study of Pevonedistat (MLN4924, TAK-924) as Single Agent and in Combination With Azacitidine in Adult East Asian Patients With Acute Myeloid Leukemia...” NCT02782468
10

“A Phase II Study of Pevonedistat (MLN4924, TAK924) and Azacitidine as Maintenance Therapy After Allogeneic Stem Cell Transplantation for Non-Remission Acute Myelogenous Leukemia” NCT03709576

“Pevonedistat (TAK-924/MLN4924) is a novel inhibitor of NEDD8-activating enzyme (NAE) with single-agent activity in relapsed/refractory acute myeloid leukemia (AML).” PMID 29348128 Jan 2018

NCT01011530

NCT01814826

NCT00722488

NCT00677170

NCT02122770

NCT01862328

NCT01415765

NCT00911066

Known as PEVONEDISTAT (MLN4924) ChEMBL registry synonym — accepted as the source's own label CHEMBL1231160
Known as PEVONEDISTAT HYDROCHLORIDE ChEMBL registry synonym — accepted as the source's own label CHEMBL1231160
Known as TAK-924 “A Phase 1/1b, Open-label Study of Pevonedistat (MLN4924, TAK-924) as Single Agent and in Combination With Azacitidine in Adult East Asian Patients With Acute Myeloid Leukemia...” NCT02782468
2

“A Phase II Study of Pevonedistat (MLN4924, TAK924) and Azacitidine as Maintenance Therapy After Allogeneic Stem Cell Transplantation for Non-Remission Acute Myelogenous Leukemia” NCT03709576

“Pevonedistat (TAK-924/MLN4924) is a novel inhibitor of NEDD8-activating enzyme (NAE) with single-agent activity in relapsed/refractory acute myeloid leukemia (AML).” PMID 29348128 Jan 2018

Known as TAK-924/MLN4924 “The drug being tested in this study is called Pevonedistat (TAK-924/MLN4924).” NCT04985656
Known as UNII-S3AZD8D215 ChEMBL registry synonym — accepted as the source's own label CHEMBL1231160
Action Inhibit “a Novel Inhibitor of Nedd8-Activating Enzyme” NCT00677170
Modality Small molecule “Pevonedistat, the first small-molecule inhibitor of NEDD8-activating enzyme” PMID 35545778 May 2022
Route Intravenous “IV dose escalation for 5 consecutive days” NCT00677170
Target MCL1 “Pevonedistat inhibits MCL1 through activation of NOXA” PMID 38572562 Sep 2024