Clinical Guidelines

AUGMENT-101 Trial: Menin Inhibitor Revumenib Shows Activity in <em>KMT2A</em>-Rearranged Acute Leukemia

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Dr. Ibrahim Aldoss presented efficacy and safety results of the phase II AUGMENT-101 trial at the 2023 ASH Annual Meeting & Exposition. The trial evaluated the menin inhibitor revumenib in heavily pretreated patients with relapsed or refractory KMT2A-rearranged acute leukemia. The drug demonstrated clinically meaningful activity, with high rates of response and measurable residual disease negativity. KMT2A-rearranged leukemia, representing about 10% of all acute leukemias, has a poor prognosis, and there is currently no approved treatment targeting the menin-KMT2A interaction. Revumenib holds promise in this area and in NPM1-mutated leukemia. The safety profile of revumenib was encouraging, with manageable toxicities. The study was significant as it provided insights into a novel treatment approach for a challenging type of leukemia.

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