Discovery of VU6024578/BI02982816: An mGlu1 Positive Allosteric Modulator with Efficacy in Preclinical Antipsychotic and Cognition Models

J Med Chem. 2024 Dec 26;67(24):22291-22312. doi: 10.1021/acs.jmedchem.4c02554. Epub 2024 Dec 12.

Abstract

Herein, we report progress toward a metabotropic glutamate receptor subtype 1 (mGlu1) positive allosteric modulator (PAM) clinical candidate and the discovery of VU6024578/BI02982816. From a weak high-throughput screening hit (VU0538160, EC50 > 10 μM, 71% Glumax), optimization efforts improved functional potency over 185-fold to deliver the selective (inactive on mGlu2-5,7,8) and CNS penetrant (rat Kp = 0.99, Kp,uu = 0.82; MDCK-MDR1 ER = 1.7, Papp = 73 × 10-6 cm/s) mGlu1 PAM (VU6024578/BI02982816, EC50 = 54 nM, 83% Glumax). An excellent rat pharmacokinetic profile allowed the evaluation of VU6024578/BI02982816 in both amphetamine-induced hyperlocomotion (minimum effective dose (MED) = 3 mg/kg, p.o.) and MK-801 induced disruptions of novel object recognition (MED = 10 mg/kg p.o.), thus providing efficacy in preclinical models of psychosis and cognition. However, unanticipated AEs in dog prevented further consideration as a candidate. Thus, VU6024578/BI02982816 can serve as a best-in-class in vivo rodent tool to study selective mGlu1 activation.

MeSH terms

  • Allosteric Regulation / drug effects
  • Animals
  • Antipsychotic Agents* / chemical synthesis
  • Antipsychotic Agents* / chemistry
  • Antipsychotic Agents* / pharmacokinetics
  • Antipsychotic Agents* / pharmacology
  • Antipsychotic Agents* / therapeutic use
  • Cognition / drug effects
  • Dogs
  • Drug Discovery
  • Humans
  • Male
  • Rats
  • Rats, Sprague-Dawley
  • Receptors, Metabotropic Glutamate* / agonists
  • Receptors, Metabotropic Glutamate* / metabolism
  • Structure-Activity Relationship

Substances

  • Receptors, Metabotropic Glutamate
  • metabotropic glutamate receptor type 1
  • Antipsychotic Agents