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Peony

(Pevonedistat)

Phase 3Small molecule

Pevonedistat is used to treat certain types of blood cancers, particularly myelodysplastic syndromes. It works by helping the body's immune system destroy cancer cells that have become resistant to other treatments.[1] Peony is currently being investigated across 7 indications[3], with its most advanced program in Phase 3.[1]

Peony is one of 198 small molecule programs targeting Acute Myeloid Leukemia. It is currently in Phase 3.

7

Indications

32

Total Trials

8

Active Trials

197

Competitors

Pipeline

Status by Indication[3][1]

Indication

Acute Myeloid LeukemiaWithdrawn

Non-Small Cell Lung CancerActive, not recruiting

Nasopharyngeal CarcinomaActive, not recruiting

Leukemia, Myeloid, AcuteActive, not recruiting

Metastatic CholangiocarcinomaActive, not recruiting

Refractory Acute Myeloid LeukemiaRecruiting

LymphomaCompleted

Acute Myeloid LeukemiaPhase 3

Withdrawn

Non-Small Cell Lung CancerPhase 2

Active, not recruiting

Nasopharyngeal CarcinomaPreclinical

Active, not recruiting

Leukemia, Myeloid, AcutePhase 3

Active, not recruiting

Metastatic CholangiocarcinomaPhase 2

Active, not recruiting

Refractory Acute Myeloid LeukemiaPreclinical

Recruiting

LymphomaPhase 1

Completed

Competition

Competitive Landscape

449 competitors in Non-Small Cell Lung Cancer

#DrugCompanyPhaseTrials

Peony

Apatinib

LLaNova Medicines Zhejiang Co., Ltd.

HLX43

Shanghai Henlius Biotech

Nanocrystalline Megestrol Acetate

Changchun GeneScience Pharmaceutical Co., Ltd.

Remeron

OOrganon and Co

Valemetostat Tosylate

Aqupla

Jiangsu Simcere Pharmaceutical Co., Ltd.

Ftorafur

TThe First Affiliated Hospital with Nanjing Medical University

Vibramycin

PPfizer's Upjohn has merged with Mylan to form Viatris Inc.

Treatment

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q4 2026

Phase 2Active, not recruiting

Metastatic Cholangiocarcinoma

NCT04175912

Phase 1Active, not recruiting

Lymphoma

NCT03479268

Phase 1/2Active, not recruiting

Acute Myeloid Leukemia

NCT03330821

Q1 2027

PreclinicalActive, not recruiting

Acute Myeloid Leukemia

NCT03862157

Q4 2032

PreclinicalRecruiting

Refractory Acute Myeloid Leukemia

NCT03013998

Clinical

Trials by Indication[1]

Acute Myeloid Leukemia5 trials

Pevonedistat and Decitabine in Treating Patients With High Risk Acute Myeloid Leukemia

Active, not recruiting·—·Phase 1·NCT03009240·30 enrolled

Pevonedistat With Azacitidine Versus Azacitidine Alone in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia

Withdrawn·NNational Cancer Institute (NCI)·Phase 2·NCT03745352

Pevonedistat, Cytarabine, and Idarubicin in Treating Patients With Acute Myeloid Leukemia

Active, not recruiting·UUniversity of Southern California·Phase 1/2·NCT03330821·53 enrolled

Show 2 more trials

A Study of Pevonedistat and Venetoclax Combined With Azacitidine to Treat Acute Myeloid Leukemia (AML) in Adults Unable to Receive Intensive Chemotherapy

Completed· Takeda logo

Takeda·Phase 2·NCT04266795·164 enrolled

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

Active, not recruiting·MM.D. Anderson Cancer Center·Preclinical·NCT03862157·40 enrolled

Advanced Solid Tumors3 trials

A Study of [14 C]-Pevonedistat in Participants With Advanced Solid Tumors

Completed· Takeda logo

Takeda·Phase 1·NCT03057366·8 enrolled

A Study to Evaluate the Effects of Pevonedistat on the Corrected QT (QTc) Interval in Participants With Advanced Solid Tumors

Completed· Takeda logo

Takeda·Phase 1·NCT03330106·68 enrolled

A Study to Evaluate the Effects of Rifampin on Pharmacokinetics (PK) of Pevonedistat in Participants With Advanced Solid Tumors

Completed· Takeda logo

Takeda·Phase 1·NCT03486314·20 enrolled

Myelodysplastic Syndromes2 trials

Expanded Access Program With Pevonedistat (Given With Azacitidine) for Adults With Higher-risk Myelodysplastic Syndromes

No longer available· Takeda logo

Takeda··NCT04484363

A Study of Pevonedistat Combined With Decitabine and Cedazuridine in Adults With Higher-risk Myelodysplastic Syndromes

Withdrawn· Takeda logo

Takeda·Phase 2·NCT04985656

Non-Small Cell Lung Cancer2 trials

Testing the Combination of MLN4924 (Pevonedistat), Carboplatin, and Paclitaxel in Patients With Advanced Non-small Cell Lung Cancer (NSCLC) Who Have Previously Been Treated With Immunotherapy

Active, not recruiting·NNational Cancer Institute (NCI)·Phase 2·NCT03965689·27 enrolled

Trial of Pevonedistat Plus Docetaxel in Patients With Previously Treated Advanced Non-Small Cell Lung Cancer

Terminated·UUniversity of Michigan Rogel Cancer Center·Phase 2·NCT03228186·40 enrolled

Chronic Myelomonocytic Leukemia2 trials

An Efficacy and Safety Study of Pevonedistat Plus Azacitidine Versus Single-Agent Azacitidine in Participants With Higher-Risk Myelodysplastic Syndromes (HR MDS), Chronic Myelomonocytic Leukemia (CMML) and Low-Blast Acute Myelogenous Leukemia (AML)

Completed· Takeda logo

Takeda·Phase 2·NCT02610777·120 enrolled

Pevonedistat Plus Azacitidine Versus Single-Agent Azacitidine as First-Line Treatment for Participants With Higher-Risk Myelodysplastic Syndromes (HR MDS), Chronic Myelomonocytic Leukemia (CMML), or Low-Blast Acute Myelogenous Leukemia (AML)

Completed· Takeda logo

Takeda·Phase 3·NCT03268954·454 enrolled

16 more indications

Lymphoma2 trials

Pevonedistat, Irinotecan, and Temozolomide in Treating Patients With Recurrent or Refractory Solid Tumors or Lymphoma

Completed·CChildren's Oncology Group·Phase 1·NCT03323034·30 enrolled

Pevonedistat and Ibrutinib in Treating Participants With Relapsed or Refractory CLL or Non-Hodgkin Lymphoma

Active, not recruiting·—·Phase 1·NCT03479268·18 enrolled

Myelodysplastic Syndrome2 trials

Pevonedistat and Belinostat in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome

Terminated·NNational Cancer Institute (NCI)·Phase 1·NCT03772925·18 enrolled

Pevonedistat, Azacitidine, Fludarabine Phosphate, and Cytarabine in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome

Completed·NNational Cancer Institute (NCI)·Phase 1·NCT03813147·12 enrolled

Treatment1 trial

Treatment of MDS/AML Patients With an Impending Hematological Relapse With AZA or ATA and Pevonedistat

Completed·UUniversity of Leipzig·Phase 2·NCT04712942·14 enrolled

Unresectable Solid Tumor1 trial

Pevonedistat and Pembrolizumab for the Treatment of dMMR/MSI-H Metastatic or Locally Advanced Unresectable Solid Tumor

Terminated·MM.D. Anderson Cancer Center·Phase 1/2·NCT04800627·2 enrolled

Myelofibrosis1 trial

Pevonedistat in Combination With Ruxolitinib for Treatment of Patients With Myelofibrosis

Terminated·WWashington University School of Medicine·Phase 1·NCT03386214·8 enrolled

Solid Tumors1 trial

A Study of Pevonedistat in People With Blood Cancers or Solid Tumors With Kidney or Liver Problems

Completed· Takeda logo

Takeda·Phase 1·NCT03814005·17 enrolled

Leukemia1 trial

Pevonedistat, Azacitidine (or Decitabine), and Venetoclax for the Treatment of Patients With Acute Myelogenous Leukemia

Terminated·MMedical College of Wisconsin·Phase 1·NCT04172844·16 enrolled

Allogeneic Stem Cell Transplantation1 trial

Pevonedistat and Azacitidine as Maintenance Therapy After Allogeneic Stem Cell Transplantation for Non-Remission AML

Terminated·MMilton S. Hershey Medical Center·Phase 2·NCT03709576·3 enrolled

Relapsed/Refractory1 trial

Pevonedistat With VXLD Chemotherapy for Adolescent/Young Adults With Relapsed/Refractory ALL or Lymphoblastic NHL

Completed·—·Phase 1·NCT03349281·6 enrolled

Adult1 trial

Pevonedistat and Low Dose Cytarabine in Adult Patients With AML and MDS

Completed·—·Phase 1·NCT03459859·12 enrolled

Mesothelioma1 trial

Pevonedistat Alone and in Combination With Chemotherapy in Patients With Mesothelioma

Terminated·MMemorial Sloan Kettering Cancer Center·Phase 1/2·NCT03319537·9 enrolled

MDS/MPN1 trial

Pevonedistat and Azacitidine in MDS or MDS/MPN Patients Who Fail Primary Therapy With DNA Methyl Transferase Inhibitors

Completed·VVanderbilt-Ingram Cancer Center·Phase 2·NCT03238248·71 enrolled

Multiple Myeloma1 trial

Ixazomib and Pevonedistat in Treating Patients With Multiple Myeloma That Has Come Back or Does Not Respond to Treatment

Completed·NNational Cancer Institute (NCI)·Phase 1·NCT03770260·8 enrolled

Refractory Acute Myeloid Leukemia1 trial

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

Recruiting·BBeat AML, LLC·Preclinical·NCT03013998·3,000 enrolled

Metastatic Cholangiocarcinoma1 trial

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

Active, not recruiting·NNational Cancer Institute (NCI)·Phase 2·NCT04175912·40 enrolled

Leukemia, Myeloid, Acute1 trial

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 recruiting·PPETHEMA Foundation·Phase 3·NCT04090736·302 enrolled

Sources

References

[1]
ClinicalTrials.gov — 32 trial records for Pevonedistat. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Pevonedistat compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Pevonedistat. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Takeda company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: NEDD8 Activating Enzyme Inhibitor[2]
Modality: Small molecule[2]
Highest Phase: Phase 3[1]
Company: Takeda

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