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U.S. FDA grants Fast Track designation to DSP-5336 for the treatment of R/R AML

By Jennifer Reilly

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Jul 16, 2024

Learning objective: After reading this article, learners will be able to cite a new clinical development in AML


DSP-5336, an oral inhibitor of the menin and mixed-lineage leukemia (MLL) interaction, for the treatment of patients with relapsed/refractory acute myeloid leukemia (AML) with a KMT2A rearrangement.1

DSP-5336 is currently under investigation as part of a phase I/II study in patients with relapsed/refractory AML.1 The latest data from this study were presented at the European Hematology Association (EHA) 2024 Hybrid Congress. Data presented highlighted an objective response rate of 57%, which included responses in patients with both nucleophosmin 1 (NPM1) mutation and KMT2A (MLL) rearrangement.1 A complete remission or complete with partial hematologic recovery was observed in 24% of patients.1 There were no dose-limiting toxicities observed, nor cardiac signals or treatment-related discontinuations/deaths.1

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Approximately what proportion of your patients with FLT3-mutations also have NPM1 and DNMT3A co-mutations?