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KNX100 is a novel small molecule drug being developed as a transdiagnostic treatment for substance use disorders—including alcohol use disorder, opioid dependence, cocaine and methamphetamine addiction—as well as for agitation and aggression in dementia. Its mechanism of action involves modulation of the oxytocin receptor (OXTR), targeting disease-relevant pharmacological, molecular, and neural pathways to normalize dysregulated brain systems associated with these conditions. Preclinical studies have shown efficacy across multiple models of substance use disorders and behavioral symptoms in dementia. In clinical development, KNX100 has demonstrated safety and tolerability in healthy volunteers during Phase 1 trials. The drug is administered orally and is currently advancing into Phase 2 studies for agitation in dementia[1][4][5][6][7].
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