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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.
Menin (encoded by MEN1) is a scaffold protein that links KMT2A fusion proteins to chromatin; small-molecule inhibitors displace the complex, switch off the leukaemia gene programme and let blasts differentiate. Revumenib (Revuforj) is approved for KMT2A-rearranged and NPM1-mutant acute leukaemia, and ziftomenib followed. NPM1 mutation is found in roughly 25 to 30 percent of AML and KMT2A rearrangement in about 5 to 10 percent, while KMT2A rearrangement drives around 70 percent of infant ALL. Menin inhibitors are the first transcription-complex disruptors in routine haematology, and combinations with venetoclax and azacitidine are in phase 3. Differentiation syndrome, QT prolongation and acquired MEN1 mutations that restore binding are the recognised open problems. It is a scaffold certain leukaemias need to keep their genes on, and the first drug against it was approved in 2024.
In plain words · A scaffold protein that certain leukaemias need to keep their genes switched on; the first drug against it was approved in 2024.
A scaffold protein that certain leukaemias need to keep their genes switched on; the first drug against it was approved in 2024.
Menin is a scaffold linking KMT2A fusion proteins to chromatin; inhibitors displace the complex and differentiate blasts.
2 products aim at Menin: small molecules. Transcription factors have no pocket to plug, so drugs either degrade them or block the partner protein they need to dock on DNA.
Broadly expressed or essential: HPA finds the RNA at low tissue specificity; the 2 medicines aimed at it (Revumenib, Ziftomenib) act on the wild-type protein, so normal tissue is exposed and the therapeutic window comes from the tumour's faster division or its dependence on the protein. HPA MEN1: RNA low tissue specificity; no normal tissue stained high; highest cancer staining liver cancer (6 of 12 high). Distribution: 1 cancer family in the corpus carries a prevalence row, label threshold or catalogue link for it (Leukaemia); Open Targets associates it with 8 specific cancer types at or above 0.5 (multiple endocrine neoplasia type 1, parathyroid gland adenoma, multiple endocrine neoplasia, Angiofibroma, lung carcinoid tumor, hereditary neoplastic syndrome and more); the corpus evidence decides and the Open Targets list is quoted for comparison. (Rule 7 of scripts/fetch-target-specificity.ts.)
Sources: Human Protein Atlas MEN1 tissue; Open Targets ENSG00000133895 associations
First described 1997. Earliest sequence paper UniProt cites for the protein: Chandrasekharappa S.C. et al, Science, 1997, "Positional cloning of the gene for multiple endocrine neoplasia-type 1". Source.
Menin is a scaffold linking KMT2A fusion proteins to chromatin; inhibitors displace the complex and differentiate blasts.
RNA: low tissue specificity, detected in all normal tissues.
No normal tissue stained high.
Medium only: carcinoid, endometrial cancer, glioma, lung cancer.
Human Protein Atlas version 25.1, antibody staining at reliability approved, enhanced or supported; used under CC BY-SA 3.0. Staining counts are patients per level in the atlas cohort, not population prevalence.
| 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.
Revumenib (Revuforj) is the first menin inhibitor (2024), for acute leukaemias with KMT2A rearrangements or NPM1 mutations.
Ziftomenib is the second menin inhibitor for leukaemia, approved in November 2025 as a once-daily pill for relapsed NPM1-mutated AML.
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.
Shares Hallmark (2022): unlocking phenotypic plasticity, Cancer stem cell theory and phenotypic plasticity, Cancer stem cells & phenotypic plasticity, Epigenetic progenitor theory: cancer without a first mutation and the tag epigenetic.
Shares Epigenetic therapy roadmap: loosening silenced genes → mutation-specific enzymes → editing the epigenome and the tag epigenetic.
Shares Eytan M. Stein, Differentiation syndrome, Epigenetic progenitor theory: cancer without a first mutation, Epigenetic therapy roadmap: loosening silenced genes → mutation-specific enzymes → editing the epigenome and the tag epigenetic.
Shares MEIS1, HOXA9, Menin / KMT2A (HOXA9-MEIS1 axis), Revumenib.
Shares KOMET-001, AUGMENT-101: revumenib, the first menin inhibitor, in relapsed leukaemias driven by KMT2A rearrangement or NPM1 mutation, Ziftomenib, Acute myeloid leukaemia.
Shares NPM1 mutation, Ziftomenib, Revumenib, NPM1-mutated and KMT2A-rearranged acute myeloid leukaemia.
Shares AUGMENT-101, AUGMENT-101: revumenib, the first menin inhibitor, in relapsed leukaemias driven by KMT2A rearrangement or NPM1 mutation, Revumenib, Acute myeloid leukaemia.
Shares Menin inhibitors for infant KMT2A-rearranged ALL, KMT2A (MLL) rearrangement, Infant acute lymphoblastic leukaemia (KMT2A-rearranged, under one year), Acute lymphoblastic leukaemia.