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A scaffold protein that certain leukaemias need to keep their genes switched on; the first drug against it was approved in 2024. This dossier gathers the 2 products (2 approved), 6 trials, 3 pathways and 0 resistance routes in the corpus that involve it, with external identifiers so it can be joined to UniProt, ChEMBL, Open Targets and the rest of biology.
Menin is a scaffold linking KMT2A fusion proteins to chromatin; inhibitors displace the complex and differentiate blasts.
| Cancer | Prevalence | Measure | Note | Source |
|---|---|---|---|---|
| Acute myeloid leukaemia | 25-30% | NPM1 mutation | KMT2A rearrangement ~5-10% | cBioPortal (TCGA) |
| Acute lymphoblastic leukaemia | 5-10% | KMT2A rearrangement (adult); ~70% infant ALL | Wikipedia |
Approximate, population-level figures; the measure column says what was counted. Ranges show the midpoint as a bar.
| Modality | Approved |
|---|---|
| Small molecule 2 |
| Trial | Setting | Result | Products | ||
|---|---|---|---|---|---|
| 3 | Recruiting | Phase 3 Randomized, Double-blind, Placebo-controlled Studies Assessing Ziftomenib in Combination With Either Standard of Care Nonintensive (Venetoclax+Azacitidine) or Intensive (7+3) Therapy in Patients With Untreated NPM1 Mutated or KMT2A Rearranged Acute Myeloid Leukemia | - | ||
| 3 | Recruiting | A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Revumenib in Combination With Intensive Chemotherapy in Participants With Newly Diagnosed AML With an NPM1 Mutation | - | ||
myeloMATCH NCT05564390 | platform | Recruiting | Newly diagnosed AML and MDS: genomic screening at diagnosis assigns patients to tiered, biomarker-defined sub-studies from induction through MRD-guided consolidation and maintenance | - | |
| 2 | Recruiting | A Phase 2, Multicenter, Open-Label Study of Ziftomenib Monotherapy in Japanese Patients With Relapsed or Refractory Acute Myeloid Leukemia With NPM1 Mutation | - | ||
KOMET-001 NCT04067336 | 1/2 | Positive | Relapsed/refractory NPM1-mutated AML: ziftomenib 600 mg daily monotherapy | CR 23%, ORR 33%, median OS 6.6 months. | |
AUGMENT-101 NCT04065399 | 1/2 | Positive | Relapsed/refractory KMT2A-rearranged or NPM1-mutated acute leukaemia: revumenib monotherapy | CR+CRh 22.8%, ORR 63.2% in KMT2Ar cohort. |
No recorded escape route names this target.
Cancer changes not just its genes but how they are read: chemical tags on DNA and histones silence guardians and awaken growth programmes. Unlike mutations, these changes are reversible, which is the hope behind epigenetic drugs.
Which nodes have drugs →In some leukaemias a broken chromatin protein (KMT2A, once called MLL) or a mutant NPM1 keeps embryonic growth genes (HOXA9, MEIS1) switched on, so blood cells never mature. Both need a partner called menin to stay on the DNA. Menin inhibitors pull the plug and the cells mature; the first was approved in 2024.
Which nodes have drugs →Cancer cells run a few genes (MYC, their lineage factors, their fusion oncogenes) at extreme volume from giant control regions called super-enhancers. The amplifiers, BRD4, CDK7, CDK9 and Mediator, are the same in every cell, but cancers are unusually dependent on them, and that dependence is druggable.
Which nodes have drugs →No companion diagnostic in the registry measures this target.
No model entry for this target yet; check the cancer entries on the models page.
Why unresolved. Revumenib and ziftomenib were approved on single-arm relapsed data; MEN1 resistance mutations and differentiation syndrome are described, and front-line randomised trials are only now under way.
What would answer it. Randomised front-line trials with overall survival and measurable-residual-disease negativity, plus resistance-mutation surveillance.
Query for this target: (TITLE:"Menin" OR ABSTRACT:"Menin" OR TITLE:"MEN1" OR ABSTRACT:"MEN1") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Menin, not a curated reading list.
The dossier as machine-readable JSON, at /api/v1/dossiers/menin.json: identifiers from HGNC, Ensembl, UniProt and ChEMBL, products with status, trials, pathways, hotspots, open questions and assays. The full entity record is in the open API at /api/v1/entities/menin.json. Licence CC BY-NC 4.0.