SOHO 2025 | The activity of revumenib in AML with NUP98 rearrangements

So the background to using menin inhibitors in subtypes of leukemia outside of KMT2A-rearranged and NPM1-mutant AML, is that menin seems to be a protein that’s very important, not only in those subtypes of leukemia, but really in any leukemia where the leukemia is being propagated by the upregulation of HOX genes. AML with a NUP98 rearrangement is one of those subtypes of acute myeloid leukemia where HOX upregulation seems to be the mode of pathogenesis. So we hypothesized that by giving a menin inhibitor to those groups of patients with NUP98 rearrangements, you’d be able to see some remission. So we reported a small series that was actually part of AUGMENT-101 of patients who received menin inhibitors. Those patients, there are only a handful of patients, but a number of those patients achieved a complete remission, and I think that that’s really proof of concept. You know, it’s not enough to say what the actual rate of remission is, because the numbers are too small, but it’s certainly proof of concept that the hypothesis that menin is important in this subtype of acute leukemia, I think, has been proved. It’ll have to be given to a larger number of patients to see what the true response rate is and the duration of response and all of that. But I do think that this is an important first step.

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