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ANAVEX 3-71 is an oral, CNS-penetrant small molecule drug candidate developed by Anavex Life Sciences for the treatment of neurodegenerative and neuropsychiatric disorders. It acts as a dual Sigma non-opioid intracellular receptor 1 (SIGMAR1) agonist and M1 muscarinic acetylcholine receptor positive allosteric modulator with agonistic effects. This differentiated mechanism is designed to address disruptions in neuronal homeostasis and offers the potential to treat all symptom domains of schizophrenia—including positive symptoms (hallucinations and delusions), negative symptoms (social withdrawal, lack of pleasure), and cognitive impairment—without the side effects associated with standard antipsychotics. Preclinical studies have demonstrated disease-modifying activity against hallmarks of Alzheimer’s disease such as cognitive deficits, amyloid pathology, tau pathology, mitochondrial dysfunction, and neuroinflammation. The drug has shown robust safety signals in healthy volunteers at single doses ranging from 5 to 200 mg once daily[1][2][4][5][6].
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