Discovery of VU6016235: A Highly Selective, Orally Bioavailable, and Structurally Distinct Tricyclic M(4) Muscarinic Acetylcholine Receptor Positive Allosteric Modulator (PAM)

VU6016235 的发现:一种高选择性、口服生物利用度高且结构独特的三环 M(4) 毒蕈碱乙酰胆碱受体正向变构调节剂 (PAM)

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Abstract

Herein, we report structure-activity relationship (SAR) studies to develop novel tricyclic M(4) PAM scaffolds with improved pharmacological properties. This endeavor involved a "tie-back" strategy to replace a 5-amino-2,4-dimethylthieno[2,3-d]pyrimidine-6-carboxamide core, which led to the discovery of two novel tricyclic cores. While both tricyclic cores displayed low nanomolar potency against both human and rat M(4) and were highly brain-penetrant, the 2,4-dimethylpyrido[4',3':4,5]thieno[2,3-d]pyrimidine tricycle core provided lead compound, VU6016235, with an overall superior pharmacological and drug metabolism and pharmacokinetics (DMPK) profile, as well as efficacy in a preclinical antipsychotic animal model.

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