Revumenib for patients with relapsed or refractory (R/R) Nucleophosmin 1–Mutated (NPM1m) Acute Myeloid Leukemia (AML): Outcomes by prior treatment in the phase 2 AUGMENT-101 study
Abstract
NPM1m AML accounts for ~30% of newly diagnosed adult AML and 12% of R/R AML cases. Approximately 50% of adult patients with NPM1m AML experience progressive disease or death after frontline treatment. Historical data suggest that only 48% and 10% of patients achieve complete remission (CR) after first salvage with high- or low-intensity treatments, respectively; CR rates decrease with each subsequent line (CR with second salvage, 30% and 8%; CR with subsequent salvage, 11% and 2%). Current standard of care varies depending on patient and disease characteristics and may include intensive chemotherapy, venetoclax (ven) ± hypomethylating agents, targeted therapies (such as FLT3 inhibitors [i], IDH1i, or IDH2i if a corresponding co-mutation is present), and allogeneic hematopoietic stem cell transplant (HSCT). Despite these available options, the prognosis for patients with R/R NPM1m AML remains poor, with a 1-year survival rate of only 16%. Novel treatments are needed.
The menin–lysine methyltransferase 2A (KMT2A) interaction is a critical driver of leukemogenesis in NPM1m AML. Revumenib is a first-in-class, oral, selective inhibitor of the menin-KMT2A interaction. Safety (safety population, N=84) and clinical outcomes (efficacy-evaluable population, n=77) from the phase 2 AUGMENT-101 study in patients with R/R NPM1m AML have been previously reported and demonstrated clinically meaningful responses across a range of prior treatments, including ven, FLT3i, IDH1i, IDH2i, and HSCT…
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