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ACT-541478 is an investigational small molecule agonist of the G protein-coupled receptor 139 (GPR139), developed by Idorsia Pharmaceuticals for the treatment of central nervous system (CNS) disorders. GPR139 is an orphan receptor primarily expressed in the habenula and the medial septum of the brain, regions associated with the regulation of mood, motivation, and cognitive functions. By targeting this receptor, ACT-541478 aims to modulate neuronal activity in these specific circuits. The compound has undergone Phase 1 clinical testing in healthy adult and elderly volunteers to evaluate its safety, tolerability, pharmacokinetics, and pharmacodynamics following oral administration.
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