# Menin inhibitors for infant KMT2A-rearranged ALL

Source: https://onco.cc/ideas/idea-menin-infant-all/  
OnCo record `idea-menin-infant-all` (Idea). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Infant leukaemia is driven almost entirely by KMT2A fusions, which menin inhibitors were built to attack. Add them to the new blinatumomab-containing backbone.

## Summary

Infant ALL EFS plateaued below 50% for two decades; blinatumomab (Interfant-21) is the first step forward. Revumenib is approved for KMT2Ar acute leukaemia including children ≥1 year and has paediatric formulation data; combining with chemotherapy and blinatumomab in the first year of life is the obvious next trial.

## Fields

- Kind: Idea
- Last checked: 2026-09-07
- Hypothesis: Adding a menin inhibitor to Interfant-21-type therapy raises 2-year EFS above 80% in KMT2A-rearranged infants without excess differentiation syndrome or hepatotoxicity.
- Rationale: KMT2A fusion is the sole driver in most infant ALL; menin inhibition produces MRD-negative remissions in relapsed KMT2Ar leukaemia.
- Proposed test: International single-arm pilot then randomised addition of revumenib or ziftomenib to Interfant-21 backbone, with pharmacokinetics and CYP3A4 interaction monitoring in infants.
- Maturity: early-clinical

## Sources

- Issa et al., The menin inhibitor revumenib in KMT2A-rearranged or NPM1-mutant leukaemia (Nature 2023): https://doi.org/10.1038/s41586-023-05812-3

## Connected records

- cancers: [Acute lymphoblastic leukaemia](https://onco.cc/cancers/all-leukemia/), [Infant acute lymphoblastic leukaemia (KMT2A-rearranged, under one year)](https://onco.cc/cancers/all-infant/)
- targets: [KMT2A (MLL) rearrangement](https://onco.cc/targets/kmt2a/), [Menin](https://onco.cc/targets/menin/)
- drugs: [Blinatumomab](https://onco.cc/drugs/blinatumomab/), [Revumenib](https://onco.cc/drugs/revumenib/), [Ziftomenib](https://onco.cc/drugs/ziftomenib/)
- trials: [AUGMENT-101](https://onco.cc/trials/augment-101/), [Interfant-06](https://onco.cc/trials/interfant-06/)
- key papers: [Blinatumomab versus Chemotherapy for Advanced Acute Lymphoblastic Leukemia](https://onco.cc/key-papers/paper-blinatumomab-all-leukemia-n-engl-j-med-2017/), [Safety and activity of blinatumomab for adult patients with relapsed or refractory B-precursor acute lymphoblastic leukaemia: a multicentre, single-arm, phase 2 study](https://onco.cc/key-papers/paper-blinatumomab-all-leukemia-lancet-oncol-2015/), [Targeted therapy with the T-cell-engaging antibody blinatumomab of chemotherapy-refractory minimal residual disease in B-lineage acute lymphoblastic leukemia patients results in high response rate and prolonged leukemia-free survival](https://onco.cc/key-papers/paper-blinatumomab-all-leukemia-j-clin-oncol-2011/)
- bottlenecks: [Rare and paediatric cancers without markets](https://onco.cc/bottlenecks/b-rare-cancers/)

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