Pinometostat — Investigational Agent
Other · Phase 1/2 · Epizyme
Mechanism of Action
A selective small-molecule inhibitor of DOT1L, the histone H3K79 methyltransferase. In MLL-rearranged (KMT2A) leukemias, aberrant DOT1L-mediated H3K79 methylation sustains expression of leukemogenic genes such as HOXA9 and MEIS1; inhibition reverses this program and induces differentiation and apoptosis.
Investigational Indications
- MLL-rearranged (KMT2A) acute myeloid leukemia
- MLL-rearranged acute lymphoblastic leukemia
- 11q23-rearranged leukemias