Dr Stein on the Impact of Revumenib on Survival Outcomes in KMT2A-Rearranged R/R Acute Leukemia

"This is a difficult-to-treat type of leukemia and the approval of revumenib gives these patients some hope that they'll be able to take a generally well tolerated oral therapy that can put them back into remission."

Eytan M. Stein, MD, director, Program for Drug Development in Leukemia, chief, Leukemia Service, Division of Hematologic Malignancies, Memorial Sloan Kettering Cancer Center, discusses how the FDA approval of revumenib (Revuforj) could improve survival outcomes for patients with KMT2A-rearranged relapsed/ refractory acute leukemia.

The menin inhibitor received FDA approval in November 2024 for the treatment of adult and pediatric patients aged 1 year and older with relapsed/refractory acute leukemia with a KMT2A translocation. Data from the phase 1/2 AUGMENT-101 study (SNDX-5613-0700; NCT04065399), which demonstrated a 21.2% complete remission (CR) or complete remission with partial hematologic recovery (CRh) rate among those treated with revumenib (n = 104), supported this regulatory decision.

The approval of revumenib is particularly significant given the poor prognosis of patients with KMT2A rearrangements in relapsed/refractory acute leukemia, whether it be acute myeloid leukemia (AML) or acute lymphocytic leukemia (ALL), Stein begins. These patients, particularly those who have received multiple lines of prior therapy, face an extremely limited life expectancy. Retrospective data in AML, for instance, shows that the overall survival (OS) for patients with more than 2 lines of therapy after relapse is measured in mere months, Stein emphasizes. For patients who have undergone 3 or more lines of therapy, the median OS is approximately 2.4 months, he adds.

Revumenib's approval provides these patients with a new, generally well-tolerated oral therapy option that may offer a chance to achieve remission. Stein states. This is a hopeful development for a group of patients who have historically had few treatment options and poor outcomes, representing a significant advancement in the management of relapsed/refractory KMT2A-positive acute leukemia, Stein concludes.