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ML-007 is an investigational, CNS-penetrant small molecule muscarinic receptor agonist designed to selectively target the M1 and M4 muscarinic receptor subtypes, with no direct activity on dopamine receptors. It is being developed by MapLight Therapeutics as a novel therapy for neurologic and psychiatric conditions such as schizophrenia, psychotic disorders, dyskinesias, and Alzheimer’s disease psychosis. The drug acts on neural circuits in the basal ganglia that are implicated in both psychosis and cognition (via M1) and in psychosis (via M4). Clinical studies have shown that ML-007 is highly permeable, bioavailable, well-tolerated across age groups (including elderly), with dose-proportional pharmacokinetics. It has been evaluated both as a single agent and in combination with a peripherally acting anticholinergic (PAC) to mitigate peripheral side effects. The fixed-dose combination formulation of ML-007/PAC (also called ML-007C-MA) is advancing into Phase 2 trials for schizophrenia and Alzheimer’s disease psychosis[1][3][5][6][8][10].
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