Acute myeloid leukemia (AML) is a deadly blood cancer with poor long-term survival, and relapse/refractory AML remains a major clinical challenge, with overall survival rates of less than 30% at one year. Early clinical data from NCT04891757 demonstrate evidence of antileukemic activity and differentiation in heavily pretreated relapse/refractory AML and MDS patients. In parallel, we have built an integrated proteomics and drug-response deep learning–based map to identify biologically distinct patient subpopulations, with protein selection criteria that have the potential to guide the clinical positioning of FHD-286.

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