Dr Issa on the Efficacy and Safety of Revumenib in R/R KMT2A+ Leukemias

We have the largest data set on KMT2A-rearranged leukemia, showing consistently the efficacy of revumenib, regardless of age…and this supports the label as a standard of care.”

Ghayas C. Issa, MD, assistant professor, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, discusses key findings and clinical implications from the phase 2 AUGMENT-101 trial (NCT04065399) evaluating revumenib (Revuforj) in relapsed or refractory KMT2A-rearranged leukemias.

The AUGMENT-101 trial highlights the efficacy and safety of revumenib, a menin inhibitor, in patients with KMT2A-rearranged acute myeloid leukemia and acute lymphoblastic leukemia, Issa begins. This subset represents a high-risk population with limited treatment options, underscoring the importance of novel therapeutic agents, Issa explains.

Data from an updated analysis, presented at the 2024 ASH Annual Meeting demonstrated consistent efficacy outcomes, supporting revumenib’s use as a standard of care treatment for patients with relapsed or refractory KMT2A-rearranged leukemias, Issa explains.

Specifically, revumenib achieved a complete remission (CR) or CR with partial hematologic recovery rate of 22.7%, with an overall response rate of 63.9%. These results were observed regardless of patient age, supporting the use of revumenib in both younger and older patients. Importantly, the durability of responses and minimal residual disease negativity observed in most responders further validate the drug’s clinical utility, he adds.

No new safety signals were reported, and no patients discontinued treatment due to cytopenias, differentiation syndrome, or QTc prolongation. Grade 3 or higher treatment-emergent or treatment-related adverse effects occurred in 91.4% and 54.3% of patients, respectively, of which 77.6% and 36.2% were serious.

The favorable safety and efficacy profile of revumenib positions it as a new standard of care for relapsed or refractory KMT2A-rearranged leukemias, Issa concludes. This approval provides a new treatment option for patients with historically poor outcomes, addressing an unmet need in this population, Issa notes.