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BTX-10504 is an oral aqueous suspension formulation of ciclopirox, a synthetic hydroxypyridone antifungal agent that has been repurposed by BioTheryX for the treatment of hematologic malignancies, including acute myeloid leukemia (AML), multiple myeloma, and myelodysplastic syndromes. Preclinical and early clinical development focused on exploiting ciclopirox’s ability to disrupt key survival pathways in malignant cells—such as iron-dependent and cell-cycle–related processes—leading to growth inhibition and apoptosis in leukemic and myeloma cells. BioTheryX advanced BTX-10504 into a phase 1 clinical trial in blood cancers with support from The Leukemia & Lymphoma Society, but no active development has been reported in recent years, and the program appears to have stalled at early clinical stages.[3][5][7]
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