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On August 3, 2022, DSP-5336, a small molecule inhibitor, was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with acute myeloid leukemia (AML).1
DSP-5336 is an investigational small-molecule inhibitor that blocks the binding of the scaffold nuclear protein menin to the mixed-lineage leukemia (MLL) protein, an interaction known to drive leukemic transformation. Menin is involved in biological pathways including regulation of cell growth, cell cycle control, and hematopoiesis. Preclinical studies have demonstrated that DSP-5336 has anticancer activity in human AML cell lines with MLL rearrangements or NPM1 mutations.1
A phase I/II dose-escalation and expansion study of DSP-5336 is currently underway in patients with relapsed/refractory AML with or without MLL rearrangements or NPM1 mutations (NCT04988555).
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