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C4X3256 is an investigational, oral, small-molecule, highly-selective antagonist of the orexin 1 receptor (OX1R). It was originally developed by C4X Discovery and is now licensed and clinically developed by Indivior. Its primary mechanism is selective blockade of OX1R in the central nervous system, a pathway shown to mediate craving and relapse behaviors associated with substance use disorders, notably opioid use disorder (OUD). Preclinical data demonstrated efficacy in animal models of opioid, nicotine, and cocaine addiction, reducing drug-seeking and relapse behaviors. Unlike opioid-based therapies, C4X3256 is non-opioid and does not act on opioid receptors. It is currently in phase 2 clinical development for opioid use disorder, supported by a U.S. National Institutes of Health (NIH) HEAL grant.
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