marked.bio

menin inhibitor · small molecule

ziftomenib

9 active trials across 2 of our biomarkers, targeting menin. 1 active approval for a biomarker on marked.bio.

Also known as KO-539, Komzifti.

Counts only trials that select patients by one of the biomarkers on marked.bio, not every trial of this drug.

Approvals and pivotal results

Biomarker-selected approvals in the United States and the European Union, with the pivotal trial's results and side effects where we have them. Results from different trials are not directly comparable.

TherapyBiomarkerTumourUnited StatesEuropean Union
ziftomenib
FDA, 2025
relapsed or refractory, susceptible NPM1 mutation, adults, no satisfactory alternative FDA approval 13/11/2025. Source
Komzifti 		NPM1 mutation AML Approved 2025 Not approved

KOMET-001 (KO-MEN-001) single-arm, 112 patients
CR+CRh 21% (14-30)Lasting 5 months

Serious side effects 79%Stopped for side effects 21%Fatal 4%

Trial details and side effects
Patients
relapsed or refractory NPM1-mutated AML (NCT04067336)
Most common side effects
infection 52%, haemorrhage 38%, diarrhoea 36%, nausea 35%, fatigue 34%, oedema 30%, musculoskeletal pain 28%, differentiation syndrome 26% (112 patients)
Notes
FDA approval summary (2025). Warnings: differentiation syndrome, QTc prolongation. Side effects: Komzifti US label, section 6.
Source
FDA approval summary

Biomarkers

BiomarkerActive trialsAll trialsFurthest active phase
NPM1 mutation 9 9 Phase 3
FLT3 mutation (ITD/TKD) 2 2 Phase 1

Sponsors

Industry sponsors leading active biomarker-selected trials of ziftomenib.

  1. Kura Oncology 4
  2. Kyowa Kirin 1

Trials

9 of 9 trials shown

  1. Studies to Assess Ziftomenib in Combination With Ven+Aza or 7+3 in Patients With Untreated NPM1-m or KMT2A-r AML

    Recruiting Phase 3 Kura Oncology Started 2025Primary completion 2031-11 NCT07007312

    ziftomenib FDAvenetoclaxazacitidinedaunorubicincytarabine

    …nintensive (Venetoclax+Azacitidine) or Intensive (7+3) Therapy in Patients With Untreated NPM1 Mutated or KMT2A Rearranged Acute Myeloid Leukemia

    NPM1 mutationAcute Myeloid Leukemia (AML)

  2. Ziftomenib for the Treatment of Patients With NPM1 Mutated or KMT2A Rearranged Acute Myeloid Leukemia Not Eligible for Standard Therapy

    Recruiting Phase 2 Uma Borate Started 2026Primary completion 2027-12 NCT06930352

    cytarabineHydroxyureaziftomenib FDA

    KMT2A rearrangement (defined as KMT2A translocations) OR NPM1 mutation (defined as NPM1 mutation resulting in cytoplasmic localization, or NPM1c) OR other mutat…

    NPM1 mutationAcute Myeloid Leukemia

  3. A Phase 2 Clinical Study of Ziftomenib in Patients With Relapsed or Refractory NPM1-Mutated Acute Myeloid Leukemia

    Recruiting Phase 2 Kyowa Kirin Started 2026Primary completion 2027-03 NCT07623616

    ziftomenib FDA

    A Phase 2 Clinical Study of Ziftomenib in Patients With Relapsed or Refractory NPM1-Mutated Acute Myeloid Leukemia

    NPM1 mutationAcute Myeloid Leukemia (AML)

  4. First in Human Study of Ziftomenib in Relapsed or Refractory Acute Myeloid Leukemia

    Active, not recruiting Phase 1/2 Kura Oncology Started 2019Primary completion 2028-10 NCT04067336

    ziftomenib FDAmidazolamitraconazole

    Patients with a documented nucleophosmin 1 mutation (NPM1-m)

    NPM1 mutationAdvanced Malignant Neoplasm, Acute Myeloid Leukemia, Mixed Lineage Leukemia, Mixed Lineage Acute Leukemia

  5. Ziftomenib + Mezigdomide in Adolesc. and Adults w/ R/R AML

    Recruiting Phase 1 Massachusetts General Hospital Started 2026Primary completion 2027-07 NCT07355335

    ziftomenib FDAMezigdomide

    …mphoid Tumors (5th Edition) with documented KMT2A rearrangement or NPM1 cytoplasmic-type (NPM1c) mutation.

    NPM1 mutationKMT2A-rearranged, NPM1-mutant Refractory or Relapsed AML

  6. A Phase I Study Investigating the Combination of the Menin Inhibitor Ziftomenib With Venetoclax and Gemtuzumab in Pediatric Patients With Acute Myeloid Leukemia

    Recruiting Phase 1 M.D. Anderson Cancer Center Started 2025Primary completion 2028-12 NCT06448013

    venetoclaxgemtuzumab ozogamicinziftomenib FDA

    Presence of KMT2Ar, NUP98r, NPM1c, UBTF-ITD or other HOX pathway mutation.

    NPM1 mutationAcute Myeloid Leukemia

  7. Safety and Tolerability of Ziftomenib Combinations in Patients With Relapsed/Refractory Acute Myeloid Leukemia

    Recruiting Phase 1 Kura Oncology Started 2024Primary completion 2026-12 NCT06001788

    ziftomenibfludarabineidarubicincytarabinegilteritinib FDA EUGranulocyte colony-stimulating factor

    Has a documented FLT3 mutation (cA-3 only).

    FLT3 mutation (ITD/TKD)NPM1 mutationAML, AML With Mutated NPM1, Hematologic Malignancy, KMT2Ar

  8. Ziftomenib Maintenance Post Allo-HCT

    Recruiting Phase 1 Massachusetts General Hospital Started 2024Primary completion 2027-04 NCT06440135

    ziftomenib FDA

    NPM1 mutation

    NPM1 mutationAcute Myeloid Leukemia, Acute Myeloid Leukemia in Remission, NPM1 Mutation, KMT2A Rearrangement

  9. A Study to Investigate the Safety and Tolerability of Ziftomenib in Combination With Venetoclax/Azacitidine, Venetoclax, 7+3, or 7+3+Quizartinib in Patients With AML

    Recruiting Phase 1 Kura Oncology Started 2023Primary completion 2030-04 NCT05735184

    ziftomenibvenetoclaxazacitidinedaunorubicincytarabinequizartinib FDA EU

    Those intending treatment with intensive chemotherapy in Arm C should be NPM1-m and FLT3-ITD+ with an allelic ratio ≥0.05 and eligible for FLT3-targeted treatment

    FLT3 mutation (ITD/TKD)NPM1 mutationAcute Myeloid Leukemia, Mixed Lineage Leukemia Gene Mutation, Refractory AML, AML With Mutated NPM1