My name is Hartmut Döhner. I’m a professor of hematology at the University of Ulm in Germany.
I’d like to briefly summarize data presented at the ASH meeting in Orlando. These are the first results from a phase 1b study combining intensive chemotherapy with a new class of agents called menin inhibitors. Specifically, we studied bleximenib, a compound developed by Johnson & Johnson.
We combined standard intensive chemotherapy with bleximenib. Menin inhibitors are among the most promising new agents in development for patients with acute myeloid leukemia (AML).
Our study included 25 patients who received bleximenib at a dose of 100 mg twice daily in combination with the “3+7” intensive chemotherapy regimen. The outcome data are very encouraging: the overall response rate exceeded 90%, and the composite complete remission rate was 87%.
Safety and feasibility are crucial in early studies. We observed no additional adverse events beyond those expected from standard intensive chemotherapy. Specific risks related to menin inhibitors, such as differentiation syndrome—which can present with symptoms like dyspnea, fever, and weight gain—were not observed in this study. Cardiac adverse events, such as QTc prolongation, occurred in three patients but were mild (grade 1–2) and did not require stopping treatment.
Overall, these data are very promising and support the launch of a global phase 3 study. This study will compare intensive chemotherapy plus bleximenib versus intensive chemotherapy plus placebo.
Menin inhibitors are being developed for genetically defined AML subtypes, including AML with mutated NPM1 and AML with KMT2A rearrangements. These subtypes share a deregulated HOX gene expression signature, caused by recruitment of chromatin complexes that include menin. Blocking these interactions induces differentiation of leukemic cells.
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HealthTree • December 29, 2025