He was initially treated with azacitidine and venetoclax but demonstrated disease progression. In the setting of a KMT2A::ELL fusion, therapy was transitioned to the menin inhibitor revumenib, resulting in short-term clinical stability and tolerability under continued supportive care.

P1/2, N=420, Recruiting, Janssen Research & Development, LLC | Trial completion date: May 2028 --> Sep 2030 | Trial primary completion date: Feb 2026 --> Jun 2026

In this preclinical study, we demonstrate that the MI ziftomenib, in combination with the XPO1 inhibitor selinexor, synergistically inhibited the growth of multiple KMT2A- r and NPM1 -m AML cell lines (CI<1). In vivo , combination therapy improved survival in both MV4;11 and OCI-AML3 cell line and primary patient-derived KMT2A - r and NPM1 -m AML xenograft models in NSG mice, effective even at reduced drug doses. These preclinical findings demonstrate that simultaneous inhibition of the menin-KMT2A interaction and XPO1 can be a more effective translational strategy for treating KMT2A- r and NPM1 -m AML than MI monotherapy to deepen responses and delay/prevent relapses.

P1, N=8, Recruiting, Sumitomo Pharma America, Inc. | Not yet recruiting --> Recruiting

P3, N=875, Recruiting, Stichting Hemato-Oncologie voor Volwassenen Nederland | Not yet recruiting --> Recruiting | N=328 --> 875

The menin-lysine methyltransferase 2A acute leukemia (KMT2A) protein-protein interaction has emerged as a clinically validated epigenetic target in acute leukemia, following the approval of the reversible menin inhibitor Revumenib for KMT2A-rearranged and nucleophosmin 1 (NPM1)-mutant disease...Across all chemical classes, sustained target engagement-rather than equilibrium affinity alone-emerges as the dominant determinant of antileukemic efficacy. By integrating structure-activity relationship (SAR), resistance mechanisms, safety considerations, and translational scope across oncology and metabolic indications, this review provides a roadmap for the rational design of next-generation menin inhibitors and establishes menin as a model system for modern epigenetic drug discovery.

P2, N=60, Recruiting, Biomea Fusion Inc.

P2, N=60, Recruiting, Biomea Fusion Inc.

P1, N=29, Not yet recruiting, Children's Oncology Group

P1, N=82, Completed, Eilean Therapeutics | Recruiting --> Completed

P1, N=22, Recruiting, Richard Stone, MD | Trial completion date: Mar 2027 --> Mar 2028 | Trial primary completion date: Mar 2026 --> Mar 2027

P3, N=468, Recruiting, Syndax Pharmaceuticals