ASH 2024 | Phase I results of the menin inhibitor enzomenib in patients with R/R acute leukemia

Yeah, so I presented a Phase I dose escalation study of enzomenib, which is a novel menin inhibitor. And there’s several menin inhibitors that are in development. Revumenib is actually the first FDA approved menin inhibitor for relapsed/refractory acute leukemia patients with a KMT2A rearrangement. Several other menin inhibitors in development. Enzomenib is a specific and potent menin inhibitor that we’re developing for NPM1-mutated acute leukemia and KMT2A-rearranged relapsed/refractory acute leukemia patients. 

Enzomenib is unique in that this has a favorable chemical formula and chemical properties that kind of differentiate this from some of the other menin inhibitors. There’s no drug accumulation. There’s very low volume of distribution, and these are things that perhaps make enzomenib a little bit more favorable pharmacokinetically when compared with some of the other menin inhibitors. 

So we enrolled 84 patients on a dose escalation, dose optimized study design. And we reported results in patients treated with 200 or 300 milligrams twice daily of enzomenib. There are 84 patients overall that have been enrolled. We haven’t seen any dose-limiting toxicities. Most of the safety profile is what you would expect to see in a patient population of this magnitude. But I think what was most notable is when we looked at the two dose levels, 200 and 300 milligrams twice daily, there are 40 patients with either KMT2A rearrangements or NPM1 mutations, 23 with a KMT2A, 17 with NPM1 mutations, and we’re seeing very high response rates. Overall response rate in the 73% range for the 300 milligram dose level in the KMT2A rearranged subgroup and in the 59% range overall in NPM1-mutated patients. Some of these responses are durable so we’re really excited about the activity of enzomenib for further development.

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