Completed Mental Health Brain & Nervous System

Clinical Development of AUT00206 for the treatment of schizophrenia

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A new drug called AUT00206 is about to be tested in humans for the first time, targeting brain circuits that go awry in schizophrenia. Schizophrenia affects how people think, feel, and perceive reality, but existing drugs often have limited effectiveness and harsh side effects. This drug works on Kv3 channels—proteins that help brain cells fire rapidly and synchronise—which are thought to be faulty in the disorder. The trial will first check the drug’s safety in healthy volunteers, then test it in patients with schizophrenia. Crucially, it includes a ketamine challenge, which mimics some schizophrenia-like symptoms in healthy people, to see if the drug counteracts them—translating earlier lab findings directly into human evidence. If the trial succeeds, it could lead to a fundamentally different class of antipsychotic medication, one that targets the underlying circuit dysfunction rather than just dampening dopamine. That could mean better symptom control with fewer side effects for millions of people worldwide. The data will also help Autifony secure the partnerships or funding needed to move into larger Phase II trials, and academic collaborators at the Institute of Psychiatry, Manchester, and Newcastle will continue studying how the drug works at a cellular level.

View original technical description
AUT00206 is a new drug that has been characterised in preclinical studies under an Early Stage Innovate UK award (Ref. 101443) due to complete in 2Q15. '206 acts on Kv3 channels that are implicated in brain circuits believed to be dysfunctional in schizophrenia. This pioneering proposal builds on the preclinical work to take ‘206 into the clinic, and conduct a first-in-human trial to explore safety as well as bio-markers of efficacy in patients with schizophrenia. The clinical project will also include an experimental ketamine-challenge, translating Early Stage preclinical work into humans. The programme will provide crucial data to aid progression of ‘206 into Phase II and enable Autifony to secure a high value partnering deal or further funding. Continued academic collaboration the Institute of Psychiatry, and Universities of Manchester and Newcastle will follow on from previous work on the ‘206 mechanism of action, critical to understanding the therapeutic potential of this new medicine.

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