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Patients with acute myeloid leukemia (AML) and myelodysplastic syndromes have very limited curative treatment options; therefore, the development of novel therapeutics remains of high importance for the future. On May 17, 2021, an announcement was made about the first patient dosed in a first-in-human and first-in-class clinical study of FHD-286 for the treatment of relapsed/refractory AML. The dose-escalation trial is evaluating the safety, pharmacokinetics, pharmacodynamics, and clinical activity of FHD-286 administered orally as a monotherapy.
FHD-286 is the first clinical candidate of a new class of inhibitors of the BAF chromatin remodeling complex ATPases BRG1 and BRM proteins, directly targeting the genetically determined dependencies of hematologic malignancies within the chromatin regulatory system. FHD-286 is characterized as a highly potent, allosteric, and selective small-molecule enzymatic inhibitor. In preclinical studies, FHD-286 has shown antitumor activity across a broad range of cancers.
Editorial theme | Immune dysregulation and genetic mutations associated with MDS pathogenesis
The pathogenesis of myelodysplastic syndromes (MDS) is heterogeneous, involving inflammation, cytokines, growth factors, the accumulation of genetic damage,...
CA-4948, an IRAK4 inhibitor, attained orphan drug designation by the FDA for the treatment of relapsed/refractory MDS and AML
CA-4948 is a first-in-class, small-molecule inhibitor of interleukin-1 receptor-associated kinase 4 (IRAK4), which plays a...
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