Back to discover
Drug

Pevonedistat

Late-stage clinical development
ClassNEDD8 activating enzyme inhibitor
RxNorm875767ChEMBLCHEMBL1231160UNIIS3AZD8D215
7
Active clinical trials
2
Protein targets
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

A Phase 2 Study of MLN4924 (Pevonedistat) in Combination With Carboplatin and Paclitaxel in Advanced NSCLC Previously Treated With Immunotherapy

Clinical trial2026-09-22Results posted · ClinicalTrials.gov

A Master Protocol for Biomarker-Based Treatment of AML (The Beat AML Trial)

Clinical trial2026-09-03Entered late-stage (PHASE2/PHASE3) · ClinicalTrials.gov

A Phase 2 Study of Pevonedistat in Combination With Carboplatin and Paclitaxel in Advanced Intrahepatic Cholangiocarcinoma

Clinical trial2026-07-31Results posted · ClinicalTrials.gov

A Phase I/II Study of Azacitidine, Venetoclax and Pevonedistat in Adults With Newly Diagnosed Secondary or Therapy-Related AML

Clinical trial2026-07-16Results expected Q1 2027 · ClinicalTrials.gov

Phase 1B/II Study of Escalating Doses of Pevonedistat (TAK-924, Formerly MLN4924) Administered in Combination With Standard Induction Chemotherapy (Cytarabine and Idarubicin) in Newly Diagnosed High Risk Acute Myelogenous Leukemia (AML)

Clinical trial2026-06-01Results expected Q4 2026 · ClinicalTrials.gov

A Phase I Study of Pevonedistat (MLN4924, TAK924) in Combination With Ibrutinib in Patients With Relapsed/Refractory Chronic Lymphocytic Leukemia and Non-Hodgkin Lymphoma

Clinical trial2026-04-27Results expected Q4 2026 · ClinicalTrials.gov

A Phase I Study of Pevonedistat (TAK-924) and Decitabine Combination Therapy in High Risk Acute Myeloid Leukemia

Clinical trial2026-03-19Completed · ClinicalTrials.gov

A Phase 3, Randomized, Controlled, Open-label, Clinical Study of Pevonedistat Plus Azacitidine Versus Single-Agent Azacitidine as First-Line Treatment for Patients With Higher-Risk Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, or Low-Blast Acute Myelogenous Leukemia

Clinical trial2025-10-29Results posted · ClinicalTrials.gov

A Randomized, Open-label, Controlled, Phase 2 Study of Pevonedistat, Venetoclax, and Azacitidine Versus Venetoclax Plus Azacitidine in Adults With Newly Diagnosed Acute Myeloid Leukemia Who Are Unfit for Intensive Chemotherapy

Clinical trial2025-10-21Results posted · ClinicalTrials.gov

MLN9708 (Ixazomib) and MLN4924 (Pevonedistat) in Relapsed/Refractory Multiple Myeloma Patients: A Phase 1b Trial

Clinical trial2025-06-19Results posted · ClinicalTrials.gov

A Phase 1 Study of MLN4924 (Pevonedistat) and Belinostat in Relapsed/Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome

Clinical trial2025-05-08Terminated · ClinicalTrials.gov

A Feasibility Trial of MLN4924 (Pevonedistat, TAK 924) Given in Combination With Azacitidine, Fludarabine, and Cytarabine, in Children, Adolescents, and Young Adults With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome

Clinical trial2025-04-13Results posted · ClinicalTrials.gov

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Pevonedistat
RxNorm CUI
875767
ChEMBL ID
CHEMBL1231160
UNII
S3AZD8D215
Primary mechanism
NEDD8 activating enzyme inhibitor

Pharmacology & targets

2 targets

Known molecular targets and mechanisms supported by curated pharmacology databases.

NEDD8-activating enzyme E1 catalytic subunit
UBA3 · Q8TBC4
INHIBITORNEDD8 activating enzyme inhibitor
NEDD8-activating enzyme E1 regulatory subunit
NAE1 · Q13564
INHIBITORNEDD8 activating enzyme inhibitor

Clinical trials

31 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
31
registered trials across all phases
LATEST COMPLETION 2026
7
Active studies
1
Recruiting
3
Late-stage (III+)
15
Completed
9
Discontinued
PHASE DISTRIBUTIONn = 31
Phase 114Phase 1 / 24Phase 210Phase 2 / 31Phase 32

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

A Master Protocol for Biomarker-Based Treatment of AML (The Beat AML Trial)

RecruitingPH II/IIIKey trialBeat AML, LLCn=3,0002032

A Randomized Phase III, Multicentre, Open Label Clinical Trial Comparing Azacitidine Plus Pevonedistat Versus Azacitidine in Older/Unfit Patients With Newly Diagnosed Acute Myeloid Leukemia Who Are Ineligible for Standard Induction Chemotherapy

Active, not recruitingPH IIIPETHEMA Foundationn=3022026

A Phase 3, Randomized, Controlled, Open-label, Clinical Study of Pevonedistat Plus Azacitidine Versus Single-Agent Azacitidine as First-Line Treatment for Patients With Higher-Risk Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, or Low-Blast Acute Myelogenous Leukemia

CompletedPH IIITakedan=4542024

Recent completions

Trials that read out recently, adding to the completed evidence base.

A Phase I Study of Pevonedistat (TAK-924) and Decitabine Combination Therapy in High Risk Acute Myeloid Leukemia

CompletedPH ICity of Hope Medical Centern=122026

A Randomized, Open-label, Controlled, Phase 2 Study of Pevonedistat, Venetoclax, and Azacitidine Versus Venetoclax Plus Azacitidine in Adults With Newly Diagnosed Acute Myeloid Leukemia Who Are Unfit for Intensive Chemotherapy

CompletedPH IITakedan=1642025

A Phase 3, Randomized, Controlled, Open-label, Clinical Study of Pevonedistat Plus Azacitidine Versus Single-Agent Azacitidine as First-Line Treatment for Patients With Higher-Risk Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, or Low-Blast Acute Myelogenous Leukemia

CompletedPH IIITakedan=4542024

A Phase 1 Study of Pevonedistat (MLN4924), a NEDD8 Activating Enzyme (NAE) Inhibitor, in Combination With Temozolomide and Irinotecan in Pediatric Patients With Recurrent or Refractory Solid Tumors

CompletedPH IChildren's Oncology Groupn=302024

A Feasibility Trial of MLN4924 (Pevonedistat, TAK 924) Given in Combination With Azacitidine, Fludarabine, and Cytarabine, in Children, Adolescents, and Young Adults With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome

CompletedPH INational Cancer Institute (NCI)n=122023

A Phase II Trial of Pevonedistat and Azacitidine in MDS or MDS/MPN Patients Who Fail Primary Therapy With DNA Methyl Transferase Inhibitors

CompletedPH IIVanderbilt-Ingram Cancer Centern=712023
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
  • ClinicalTrials.gov — clinical trials