Spirocyclic M1/M4 Agonists for Selective CNS Targeting

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Solution Overview

Problem

Current muscarinic receptor agonists for treating Alzheimer's disease and schizophrenia exhibit significant side effects due to non-selectivity for M1 and M4 receptors, leading to peripheral cholinergic effects such as nausea, gastrointestinal issues, and bradycardia, while existing treatments for pain management have limitations in targeting central and peripheral pain pathways effectively.

Innovation Solution

Development of novel spirocyclic compounds that act as selective agonists for M1 and/or M4 muscarinic receptors, reducing activity at M2 and M3 receptor subtypes to minimize peripheral side effects and enhance therapeutic efficacy in cognitive disorders and pain management.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If non-selective muscarinic receptor agonists are used to treat cognitive disorders, then cognitive function improves, but peripheral side effects such as nausea, gastrointestinal issues, and bradycardia increase

Engineering Contradiction:
Improvecognitive function improvementVSAvoidperipheral side effects
Core Design Contradiction:
ReliabilityVSObject-affected harmful factors

Solution Approach 1:

The patent applies local quality by designing compounds with specific molecular structures (spirocyclic frameworks with particular substituent patterns) that confer selective affinity for M1 and M4 receptors over M2 and M3 receptors. This structural differentiation enables the drug to exert its cognitive-enhancing effects in the central nervous system while minimizing activation of peripheral muscarinic receptors that mediate unwanted side effects.

Inventive Principle:
Principle #3Local quality

Solution Approach 2:

The patent segments the muscarinic receptor subtype responses by creating compounds that differentially activate specific receptor subtypes. The spirocyclic compounds are designed to selectively engage M1 and M4 receptors in the brain while having reduced activity at M2 and M3 receptors in peripheral tissues, effectively separating the therapeutic effects from the side effects.

Inventive Principle:
Principle #1Segmentation

2Object-affected harmful factors

If selective M1 and M4 receptor agonists are developed, then peripheral side effects are reduced, but therapeutic efficacy in targeting pain pathways may be limited

Engineering Contradiction:
Improveperipheral side effectsVSAvoidpain management efficacy
Core Design Contradiction:
Object-affected harmful factorsVSReliability

Solution Approach 1:

The patent applies universality by designing spirocyclic compounds that simultaneously target multiple receptor subtypes (M1 and M4) with a single molecular entity. This multi-functional approach ensures that the compound can address both cognitive disorders and pain management through activation of different muscarinic receptor subtypes involved in these distinct pathological processes, while maintaining selectiveivity to reduce peripheral side effects.

Inventive Principle:
Principle #6Universality (Multi-functionality)

Data Source

PatentEP3271358B1Spirocyclic compounds as agonists of the muscarinic m1 receptor and/or m4 receptor
Publication Date: 2020.11.18 NXERA PHARMA UK LTD
  • EP3271358B1 patent drawingFigure 1
  • EP3271358B1 patent drawingFigure 2
  • EP3271358B1 patent drawing

AI summary

This invention relates to compounds that are agonists of the muscarinic M1 and/or M4 receptor and which are useful in the treatment of muscarinic M1/M4 receptor mediated diseases. Also provided are pharmaceutical compositions containing the compounds and the therapeutic uses of the compounds. Compounds provided are of formula. (I) where p, q, r, s, X, Z, Y, R1, R2, R3and R4 are as defined herein.