OX-201

OX-201 is a potent, highly selective, and brain-penetrant orexin receptor 2 (OX2R) agonist, developed primarily by Takeda Pharmaceutical. It demonstrates an EC50 of 8.0 nM for OX2R with over 1,000-fold selectivity versus orexin receptor 1 (OX1R), showing minimal off-target activity at high concentrations. Preclinical studies demonstrate that OX-201 promotes wakefulness and suppresses cataplexy via OX2R-expressing brain regions. It is under evaluation for conditions such as narcolepsy and tauopathies, including Alzheimer’s disease, due to potential benefits in improving sleep/wake regulation and facilitating neural protein clearance. The main route of administration in preclinical models is oral. OX-201 is currently in preclinical development and not approved for any indication[1][2][3][5][7].