Around one-third of patients with acute myeloid leukemia (AML) harbor FLT3 gene mutations which are associated with poor prognosis and high risk of relapse. Several compounds targeting FLT3 internal tandem duplication (ITD) have been developed in the past decades, but none has overcome myelosuppressive toxicity caused by the simultaneous inhibition of FLT3 and c-Kit. Therefore, there is a need for new treatment options.
Shanghaitech University has identified thiazolidinedione compounds acting as DNA methyltransferase 3A (DNMT3A) inhibitors reported to be useful for the treatment of acute myeloid leukemia and overgrowth syndrome.
Medexus Pharmaceuticals Inc. looks set for its U.S. launch of bifunctional alkylating agent treosulfan in the first half of 2025 following a long-awaited FDA approval of the drug, branded Grafapex, for use in combination with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation (allo-HSCT) in adults and pediatric patients, 1 and older, with acute myeloid leukemia or myelodysplastic syndrome.
Researchers from Shenyang Pharmaceutical University and affiliated organizations have detailed the discovery and preclinical characterization of novel reversible lysine-specific demethylase 1 (LSD1) inhibitors as candidates for acute myeloid leukemia (AML) therapy.
Researchers from Oxford Biomedica (UK) Ltd. have published findings from their work aiming to identify antigens that could represent novel targets for CAR T-cell therapies against acute myeloid leukemia (AML).
Kymera Therapeutics Inc. has published the characterization of KT-253, a novel compound designed to degrade the murine double minute 2 (MDM2) protein, offering a promising approach for cancers retaining wild-type p53.
The curtain is coming down on one of Europe’s longest-established biopharmas, with Novartis AG announcing it is to shut Morphosys AG’s facilities, following its 2024 acquisition of the one-time antibody pioneer for $2.9 billion. The closure of sites in the U.S. and Germany by the end of 2025 will affect 330 employees.
KAT2A is a histone acetyltransferase that functions as a transcriptional activator which, together with its paralogue KAT2B, is markedly overexpressed in acute myeloid leukemia (AML) compared to in hematopoietic progenitor cells.
