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Syndax Pharmaceuticals’ treatment for a genetically defined form of advanced leukemia met its goal in a pivotal study, leading to complete remission for about a quarter of patients in the trial.

The results, disclosed Monday, will set in motion an application for Food and Drug Administration approval, which the company expects to complete by the end of the year.


The study enrolled adults and children with treatment-resistant forms of acute myeloid leukemia or acute lymphoid leukemia who have either a genetic “rearrangement” called KMT2A or a mutation affecting a protein called NPM1. Syndax’s drug, called revumenib, led to complete remission for 13 of the 57 patients, a 23% rate that met the trial’s primary goal and led the study’s independent data monitors to recommend stopping it early.

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