marked.bio

menin inhibitor · small molecule

revumenib

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

Also known as SNDX-5613, Revuforj.

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
revumenib
FDA, 2025
relapsed or refractory, susceptible NPM1 mutation, adults and children >=1 year, no satisfactory alternative FDA approval 24/10/2025 (Revuforj was first approved 15/11/2024 for KMT2A-rearranged acute leukaemia). Source
Revuforj 		NPM1 mutation AML Approved 2025 Not approved

AUGMENT-101 single-arm
CR+CRh 23% (14-35)Lasting 4.5 months

Serious side effects 76%Stopped for side effects 20%Fatal 4%

Trial details and side effects
Patients
relapsed or refractory NPM1-mutated AML (NCT04065399)
Most common side effects
nausea 48%, haemorrhage 48%, infection 46%, musculoskeletal pain 37%, febrile neutropenia 37%, QT prolongation 36%, diarrhoea 29%, differentiation syndrome 25% (241 (KMT2A and NPM1 pooled) patients)
Notes
FDA approval summary (2025). Warnings: differentiation syndrome, QTc prolongation. Side effects: Revuforj US label, section 6 (differentiation syndrome 18% in NPM1-mutated AML).
Source
FDA approval summary

Biomarkers

BiomarkerActive trialsAll trialsFurthest active phase
NPM1 mutation 12 14 Phase 3
FLT3 mutation (ITD/TKD) 4 4 Phase 2

Sponsors

Industry sponsors leading active biomarker-selected trials of revumenib.

  1. Syndax Pharmaceuticals 3

Trials

12 of 14 trials shown

  1. Revumenib in Combination With Azacitidine + Venetoclax in Patients NPM1-mutated or KMT2A-rearranged AML

    Recruiting Phase 3 Stichting Hemato-Oncologie voor Volwassenen Nederland Started 2025Primary completion 2029-12 NCT06652438

    revumenib FDA

    Patient with newly diagnosed NPM1-mutated AML, consistent with NPM1c, according to the 2022 International Consensus Classification …

    NPM1 mutationAcute Myeloid Leukemia, Adult

  2. Study of Revumenib in Combination With Intensive Chemotherapy in Newly Diagnosed Acute Myeloid Leukemia (AML) With a NPM1 Mutation

    Recruiting Phase 3 Syndax Pharmaceuticals Started 2025Primary completion 2029-06 NCT07211958

    revumenib FDAIntensive Chemotherapy Regimen

    Presence of an NPM1 mutation.

    NPM1 mutationAcute Myeloid Leukemias

  3. Menin-Inhibitor Targeted Maintenance in AML

    Not yet recruiting Phase 2 Center for International Blood and Marrow Transplant Research Started 2026Primary completion 2031-06 NCT07563010

    revumenib

    NPM1-mutated (NPM1m) AML (Including FLT3-ITD or TKD co-mutation) 3.

    FLT3 mutation (ITD/TKD)NPM1 mutationRelapsed Adult AML, Stem Cell Transplant Complications, Acute Myeloid Leukemia, Transplant-Related Hematologic Malignancy

  4. A Study of Revumenib and Mezigdomide in People With Leukemia

    Recruiting Phase 1/2 Memorial Sloan Kettering Cancer Center Started 2026Primary completion 2029-01 NCT07356154

    revumenib FDAMezigdomide

    Participant has confirmed acute leukemia with detectable NPM1c, KMT2A translocation, or NUP98 translocation.

    NPM1 mutationLeukemia, Acute Leukemia, Relapse Leukemia, Refractory Leukemia

  5. A Phase I-II Study Investigating the All-Oral Combination of the Menin Inhibitor SNDX-5613 With Decitabine/Cedazuridine (ASTX727) and Venetoclax in Acute Myeloid Leukemia (SAVE)

    Recruiting Phase 1/2 M.D. Anderson Cancer Center Started 2022Primary completion 2026-12 NCT05360160

    revumenib FDAvenetoclaxdecitabine

    …d/refractory (R/R cohort) AML or myeloid phenotype MPAL with either KMT2Ar, or NUP98r, or NPM1c.

    NPM1 mutationAcute Myeloid Leukemia

  6. A Study of Revumenib in R/R Leukemias Including Those With an MLL/KMT2A Gene Rearrangement or NPM1 Mutation

    Recruiting Phase 1/2 Syndax Pharmaceuticals Started 2019Primary completion 2027-12 NCT04065399

    revumenib FDAcobicistat

    …Version 3.2020), or acute leukemia harboring KMT2A rearrangement, NUP98 rearrangement, or NPM1 mutation that have detectable disease in the bone marrow.

    NPM1 mutationAcute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Mixed Lineage Acute Leukemia, Mixed Phenotype Acute Leukemia

  7. SNDX-5613 and Gilteritinib for the Treatment of Relapsed or Refractory FLT3-Mutated Acute Myeloid Leukemia and Concurrent MLL-Rearrangement or NPM1 Mutation

    Recruiting Phase 1 Uma Borate Started 2024Primary completion 2027-01 NCT06222580

    gilteritinib FDA EUrevumenib

    …afety and Efficacy of Dual Menin and FLT3 Inhibition in Patients With Relapsed/Refractory FLT3- Mutated Acute Myeloid Leukemia Containing a Concurrent MLL-Rearrangement or NPM1 Mutation: A Phas…

    FLT3 mutation (ITD/TKD)NPM1 mutationAcute Myeloid Leukemia With FLT3/ITD Mutation, Acute Myeloid Leukemia With KMT2A Rearrangement, Acute Myeloid Leukemia With NPM1 Mutation, Recurrent Acute Myeloid Leukemia

  8. Revumenib in Combination With 7+3 + Midostaurin in AML

    Recruiting Phase 1 Richard Stone, MD Started 2024Primary completion 2027-03 NCT06313437

    revumenibmidostaurin FDA EUcytarabinedaunorubicin

    Presence of FLT3-ITD and/or TKD mutation(s) AND NPM1 mutation in bone marrow or peripheral blood

    FLT3 mutation (ITD/TKD)NPM1 mutationAcute Myeloid Leukemia, AML, Adult, AML With Gene Mutations, AML

  9. Revumenib for the Treatment of Acute Leukemia in Patients Post-Allogeneic Stem Cell Transplant

    Recruiting Phase 1 City of Hope Medical Center Started 2024Primary completion 2028-04 NCT06575296

    revumenib

    FLT3-ITD co-mutation

    FLT3 mutation (ITD/TKD)NPM1 mutationAcute Leukemia, Acute Leukemia of Ambiguous Lineage, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia

  10. A Study of Revumenib in Combination With Intensive Chemotherapy in Participants With Acute Myeloid Leukemias

    Active, not recruiting Phase 1 Syndax Pharmaceuticals Started 2024Primary completion 2027-02 NCT06226571

    revumenib FDAChemotherapy RegimenHiDAC

    KMT2Ar, NPM1m, or NUP98r mutations identified by local laboratory prior to the first dose of revumenib.

    NPM1 mutationAcute Myeloid Leukemias

  11. Study of Revumenib, Azacitidine, and Venetoclax in Pediatric and Young Adult Patients With Refractory or Relapsed Acute Myeloid Leukemia

    Recruiting Phase 1 St. Jude Children's Research Hospital Started 2024Primary completion 2027-01 NCT06177067

    revumenib FDAvenetoclaxazacitidinemethotrexatecytarabine

    Presence of KMT2A rearrangement (KMT2Ar), NUP98 rearrangement (NUP98r), NPM1 mutation or fusion, PICALM::MLLT10, DEK::NUP214, UBTF-TD, KAT6A rearrangement (KAT6Ar), or SET::NU…

    NPM1 mutationRefractory Acute Myeloid Leukemia, Relapsed Acute Myeloid Leukemia, Acute Leukemia of Ambiguous Lineage

  12. A Phase 1b Study of Menin Inhibitor SNDX- 5613 in Combination With Daunorubicin and Cytarabine in Newly Diagnosed Patients With Acute Myeloid Leukemia and NPM1 Mutated/FLT3 Wildtype or MLL/KMT2A Rearranged or NUP98 Alterations Disease

    Recruiting Phase 1 National Cancer Institute (NCI) Started 2024Primary completion 2027-12 NCT05886049

    cytarabinedaunorubicinrevumenib FDA

    Dose escalation: Patients ages 18-75 years at time of diagnosis with NPM1-mutated/FLT3-ITD wildtype and NPM1-mutated/FLT3-TKD wildtype with high-risk features (adverse ris…

    NPM1 mutationAcute Myeloid Leukemia, Acute Myeloid Leukemia With KMT2A Rearrangement, Acute Myeloid Leukemia With NPM1 Mutation, Secondary Acute Myeloid Leukemia