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Ocinaplon is an investigational anxiolytic drug belonging to the pyrazolopyrimidine class. It was developed as a selective modulator of GABA-A receptors and exhibits a pharmacological profile similar to benzodiazepines but with greater selectivity for anxiolytic effects and reduced sedative or amnestic side effects[1][3][4]. Ocinaplon acts as a positive allosteric modulator at GABA-A receptors, with modest selectivity for subtypes containing the α2 subunit over those containing α1[1][7]. It was evaluated primarily for the treatment of generalized anxiety disorder (GAD), where it demonstrated efficacy in reducing anxiety symptoms without typical benzodiazepine-associated adverse effects such as sedation or dependence[5][6]. However, development was discontinued after cases of liver toxicity were observed during Phase III clinical trials[1][3].
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