Iadademstat granted Orphan Drug designation in the US for the treatment of acute myeloid leukaemia

This is a first-in-class small molecule inhibitor of the epigenetic enzyme LSD1. It is being evaluated in an ongoing Phase II study (ALICE), in which a response rate of 85% has been demonstrated. It has already been designated an orphan drug by the European Medicines Agency.

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Biospace Inc.