Table 2.

Key clinical outcomes of Menin inhibitors in ASH 2023

Inhibitor	Study Name	Patient Population	Key Efficacy Outcomes	Safety Profile
Revumenib	AUGMENT-101	Relapsed/refractory KMT2Ar AML	CR + CRh: 22.8% (21/94), CRc: 43.9% (41/94), ORR: 63.2% (59/94)	Manageable; differentiation syndrome (16.0%), febrile neutropenia (13.8%), and QTc prolongation (13.8%);
Revumenib	Post-HSCT Study	Post-HSCT patients	CR: 6/9, MRD negativity: 5/9	Manageable; no new safety signals, common AEs included cytopenias (20%) and infections (10%)
Ziftomenib	Komet-008	Relapsed/refractory AML	Not available	Manageable; common AEs included anemia (25%), pneumonia (20%), thrombocytopenia (15%), neutropenia (15%)
DSP-5336	N/A	MLLr and NPM1 mutated AML	CR with incomplete recovery: 1/6, morphologic leukemia-free state: 1/6	Not available
BMF-219	COVALENT-101	Relapsed/refractory acute leukemia	CR: 2/5	Vomiting 13% (3) and Differentiation Syndrome (DS) 13% (3). No Grade 5 TRAEs were reported.
JNJ-75,276,617	Phase 1 Study	Relapsed/refractory AML	CR + CRh: 22.8% (21/94), CRc: 43.9% (41/94), ORR: 63.2% (59/94)	Differentiation syndrome (8 [14%]), neutropenia (6 [10%]), anemia and thrombocytopenia (4 [7%] each).

Abbreviations AE: Adverse Event AML: Acute Myeloid Leukemia CR: Complete Remission CRh: Complete Remission with Partial Hematologic Recovery CRc: Composite Complete Remission HSCT: Hematopoietic Stem Cell Transplant KMT2Ar: KMT2A rearrangements MLL: Mixed Lineage Leukemia MRD: Measurable Residual Disease NPM1: Nucleophosmin 1 ORR: Overall Response Rate QTc: Corrected QT Interval TEAE: Treatment-Emergent Adverse Event