molecules of the month

The Takeda lysine-specific demethylase 1 (LSD1) inhibitor (TAK-418) is an oral CNS-penetrant clinical candidate for neurodevelopmental disorders such as autism spectrum disorder. We previously highlighted irreversible and reversible LSD1 inhibitors intended to treat cancer. This tranylcypromine-derived clinical candidate is interesting because it is a relatively rare example of a mechanism-based CNS-acting drug. Oral administration of TAK-418 at only 1 mg/kg normalized mRNA expression in neurodevelopmental disorder model rodents and improved sociability. The proposed mechanism of action for neurodevelopmental disorders (restoring gene expression homeostasis by inhibiting a stabilizer of faulty epigenetic machinery) will be interesting to watch.