Bicyclic Derivatives as GABAA α5 Receptor Modulators

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Solution Overview

Problem

There is an unmet need for compounds that can selectively modulate the GABA A α5 receptor to treat or prevent diseases related to this receptor without causing undesirable CNS side effects.

Innovation Solution

Development of bicyclic derivatives with high affinity and selectivity for the GABA A α5 receptor, acting as negative allosteric modulators, which can be used alone or in combination with other active ingredients to treat or prevent diseases associated with the GABA A α5 receptor.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Adaptability or versatility

If non-selective GABA A receptor modulators are used, then broad therapeutic effects are achieved, but CNS side effects such as sedation, amnesia, and anxiety occur

Engineering Contradiction:
Improvebroad therapeutic effectsVSAvoidCNS side effects
Core Design Contradiction:
Adaptability or versatilityVSObject-affected harmful factors

Solution Approach 1:

The patent applies local quality by designing compounds with specific molecular features (cyclopropyl, cyclobutyl, or cyclopentyl rings at positions R1 and R2, and specific substituent patterns) that confer selectivity for the GABA A α5 receptor subtype. This structural specificity ensures the drug acts locally on the target receptor subtype rather than broadly on all GABA A receptors, thereby achieving therapeutic effects while minimizing off-target CNS side effects

Inventive Principle:
Principle #3Local quality

2Object-affected harmful factors

If selective GABA A α5 receptor modulators are developed, then CNS side effects are reduced, but compound selectivity and affinity must be precisely optimized

Engineering Contradiction:
ImproveCNS side effectsVSAvoidcompound selectivity and affinity
Core Design Contradiction:
Object-affected harmful factorsVSManufacturing precision

Solution Approach 1:

The patent employs parameter changes by systematically varying molecular parameters including the type of cyclic group (cyclopropyl, cyclobutyl, cyclopentyl), the nature of substituents R3-R10, and their positions to optimize both selectivity for and affinity to the GABA A α5 receptor. This structured parameter optimization enables precise tuning of drug-receptor interaction while maintaining selectivity

Inventive Principle:
Principle #35Parameter changes

3Reliability

If early GABA A receptor modulators were used, then cognitive enhancing effects were observed, but undesired CNS side effects like sedation and amnesia occurred

Engineering Contradiction:
Improvecognitive enhancing effectsVSAvoidsedation and amnesia
Core Design Contradiction:
ReliabilityVSObject-generated harmful factors

Solution Approach 1:

The patent applies inversion by developing negative allosteric modulators (NAMs) that reduce excessive GABA A α5 receptor activity rather than enhancing it. This inverted approach addresses the problem by dampening overactive α5-containing receptors that contribute to cognitive impairment, thereby achieving cognitive enhancement without the sedative and amnestic side effects associated with positive modulators

Inventive Principle:
Principle #13The other way round (Inversion)

Data Source

PatentEP3856342B1Bicyclic derivatives as gabaa a5 receptor modulators
Publication Date: 2025.12.10 RICHTER GEDEON NYRT
  • EP3856342B1 patent drawing
  • EP3856342B1 patent drawing
  • EP3856342B1 patent drawing

AI summary

The present invention provides compounds of formula (I) and/or salt thereof and/or geometric isomer thereof and/or stereoisomer thereof and/or enantiomer thereof and/or racemate thereof and/or diastereomer thereof and/or biologically active metabolite thereof and/or prodrug thereof and/or solvate thereof and/or hydrate thereof and/or polymorph thereof having affinity and selectivity for the gamma-aminobutyric acid A receptor subunit alpha 5 and act as GABAA α5 negative allosteric modulators, thereby useful in the treatment or prevention of diseases related to the GABAA α5 receptor, process for the preparation thereof, pharmaceutical compositions comprising them alone or in combination with one or more other active ingredients and their use as medicaments.