About 30% of patients with acute myeloid leukemia (AML) harbor mutations in the gene encoding receptor-type tyrosine-protein kinase FLT3 in the form of internal tandem duplication (ITD) or mutations in the tyrosine kinase domain.

Researchers from Epics Therapeutics SA presented preclinical data for EP-102, a novel small-molecule inhibitor of N6-adenosine-methyltransferase catalytic subunit (METTL3), being developed for the treatment of cancer.

Researchers from Hibercell Inc. recently presented preclinical data for HC-7366, a first-in-class direct activator of GCN2 (general control nonderepressible 2) currently in phase I development for the treatment of solid tumors (NCT05121948).

Senti Biosciences Inc. has received clearance of its IND application from the FDA for SENTI-202, an off-the-shelf chimeric antigen receptor natural killer (CAR-NK) cell therapy product candidate designed to selectively target and eliminate CD33- and/or FLT3-expressing hematologic malignancies, such as acute myeloid leukemia (AML) and myelodysplastic syndrome, while sparing healthy bone marrow cells.

MYB is a transcription factor essential for cell proliferation and differentiation, and is regarded as a target for cancer therapy because it is overexpressed in many different tumor types such as adenoid cystic carcinoma (ACC), acute myeloid leukemia (AML) or T-cell acute lymphoblastic leukemia, among others.

Researchers from Zhejiang University and affiliated organizations have reported the discovery of novel orally bioavailable bromodomain-containing protein 9 (BRD9) proteolysis-targeting chimera (PROTAC) candidates for the treatment of acute myelocytic leukemia (AML). Synthesis and optimization led to the discovery of a novel series of BRD9 PROTACs, with compound [I] selected as the lead candidate with the best degradation and proliferation inhibition activities in vitro.

CD33 is known to be highly expressed in myeloid cells and a good therapeutic target for treating acute myeloid leukemia (AML). In the search for more potent compounds, researchers from Dragonfly Therapeutics Inc. and Bristol Myers Squibb Inc. investigated the therapeutic potential of BMS-986357, also known as CC-96191.

High expression of CD123, the IL-3 receptor α chain (IL-3α), is observed on both leukemic blasts and leukemic stem cells, thus suggesting it can be considered an attractive therapeutic target in AML.