TAK-418

TAK‑418 is a selective, orally active small molecule inhibitor of lysine-specific demethylase 1 (LSD1/KDM1A), developed by Takeda as a potential therapeutic agent for central nervous system disorders, particularly Kabuki syndrome. TAK‑418 acts by inhibiting LSD1 enzyme activity with high potency (IC50 of 2.9 nM), thereby modulating epigenetic regulation and normalizing dysregulated gene expression in the brain. Preclinical studies have shown that TAK‑418 increases histone methylation at specific gene loci and improves symptoms in neurodevelopmental disorder models. It is brain penetrant and has demonstrated favorable pharmacokinetics and safety profiles in phase I clinical trials[1][2][3][4][5].

Other names

5-((1R,2R)-2-((Cyclopropylmethyl)amino)cyclopropyl)-N-(tetrahydro-2H-pyran-4-yl)thiophene-3-carboxamide