Enzomenib — Investigational Agent
Menin Inhibitor · Phase 1/2 · Sumitomo Pharma
Mechanism of Action
Oral small-molecule inhibitor of the protein-protein interaction between menin and the histone methyltransferase KMT2A (MLL1). In leukemias driven by KMT2A rearrangements (KMT2A-r) or NPM1 mutations, the menin-KMT2A complex is required to sustain aberrant expression of pro-leukemic target genes such as HOXA9 and MEIS1, which block myeloid differentiation and maintain the leukemic stem cell program. By disrupting the menin-KMT2A interaction, enzomenib releases the transcriptional block, downregulates HOX/MEIS1 gene expression, and induces terminal differentiation and apoptosis of leukemic…
Investigational Indications
- Relapsed/refractory KMT2A-rearranged (MLL-rearranged) acute myeloid leukemia (AML)
- Relapsed/refractory NPM1-mutant AML
- KMT2A-rearranged acute lymphoblastic leukemia (ALL)
- Acute leukemias of ambiguous lineage with KMT2A rearrangement