Dr Stein on the Efficacy of Revumenib in KMT2A+ R/R Acute Leukemia

"What [the data] say to me is: [approximately] 23% of patients are clearing their blasts and having nice blood count recovery [with revumenib], but there's a large group of patients who are still able to clear their blasts. If those patients are eligible to receive [ASCT], the clearance of those blasts does allow those patients to proceed to transplant, [which will] hopefully be successful."

Eytan M. Stein, MD, director, Program for Drug Development in Leukemia, chief, Leukemia Service, Division of Hematologic Malignancies, Memorial Sloan Kettering Cancer Center, discusses key efficacy findings from the phase 1/2 AUGMENT-101 study (SNDX-5613-0700; NCT04065399), which supported the November 2024 FDA approval of revumenib (Revuforj) in KMT2A-rearranged relapsed/refractory acute leukemia.

In the efficacy portion of the study involving 104 patients with relapsed or refractory acute leukemia and a KMT2A rearrangement, 21.2% (95% CI, 13.8%-30.3%) of patients achieved a complete remission (CR) or a complete remission with partial hematologic recovery (CRh), meeting the FDA's regulatory bar for approval, Stein begins. The median duration of CR or CRh was 6.4 months (95% CI, 2.7-not estimable), with a time to response of 1.9 months (range, 0.9-5.6 months), he adds. Importantly, 14% of patients who were previously transfusion dependent achieved transfusion independence [TI]; 48% of patients at baseline remained independent. These findings highlight revumenib’s potential to improve not only remission rates but quality of life for patients by reducing transfusion dependency, Stein states.

Earlier data published in the Journal of Clinical Oncology from a subset of 57 patients showed a similar trend, with an overall response rate of 63.2% and a CR/CRh rate of 22.8%, Stein notes. This dataset further reinforces the ability of revumenib to clear blasts, providing patients an opportunity to proceed to an allogeneic stem cell transplant (ASCT), which could offer long-term disease control, he explains. Although the CR/CRh rate produced by revumenib represents a significant achievement, the ability of additional patients to clear their blasts underscores the broader potential of revumenib in combination with subsequent treatments, offering hope for better outcomes in a patient group with historically poor prognosis, Stein concludes.