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Q-134R is a brain-penetrant, orally available small molecule hydroxyquinoline derivative developed by Avidin Biotech in partnership with Aperus Pharma. It is being investigated for the treatment of Alzheimer's disease, where it acts as a neuroprotective, anti-inflammatory, and nootropic agent. The drug's primary mechanism of action is the selective inhibition of Nuclear Factor of Activated T-cells (NFAT) signaling. Notably, Q-134R achieves this suppression without inhibiting calcineurin activity, thereby avoiding the immunosuppressive side effects typically associated with calcineurin inhibitors. Preclinical studies have shown the compound to be metabolically stable with a favorable toxicity profile, demonstrating cognitive enhancement in various models. It has successfully completed a Phase 1a safety trial and is currently in Phase 2 clinical development.
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