TGR5 Modulators via Cholic Acid Structural Modification

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Solution Overview

Problem

There is a need for more potent and selective TGR5 modulators to further understand the physiological and pharmacological actions of the TGR5 receptor for the treatment and prevention of diseases such as obesity, diabetes, and metabolic syndrome, as existing TGR5 agonists have limitations in selectivity and potency, and high doses of cholic acid require further investigation for therapeutic relevance and safety.

Innovation Solution

Development of compounds according to the formula IIIA, which include specific chemical moieties that modulate TGR5, such as R1, R2, R3, R4, R5, R6, R7, R8, and R9, to create TGR5 modulators that can be used in compositions for treating or preventing metabolic, inflammatory, liver, autoimmune, cardiac, kidney, cancer, and gastrointestinal diseases.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If high doses of cholic acid are used to activate TGR5, then therapeutic effects are achieved, but toxicity concerns arise

Engineering Contradiction:
Improvetherapeutic effectVSAvoidtoxicity
Core Design Contradiction:
ReliabilityVSObject-affected harmful factors

Solution Approach 1:

The patent modifies the chemical structure of cholic acid by introducing specific substituents at defined positions (R1-R10 groups) to create analogs with altered pharmacological properties. These structural parameter changes enable the compounds to maintain TGR5 activation efficacy while reducing toxicity, as evidenced by the selective agonist activity described in the background section.

Inventive Principle:
Principle #35Parameter changes

Solution Approach 2:

The patent applies local quality modification by introducing specific functional groups (hydroxyl, alkyl, halogen, etc.) at particular positions on the cholic acid core structure. This localized modification allows the molecule to maintain its TGR5-binding capability while altering its metabolic profile and reducing harmful effects associated with high-dose cholic acid administration.

Inventive Principle:
Principle #3Local quality

2Reliability

If existing TGR5 agonists are used, then some therapeutic effects are observed, but selectivity and potency are limited

Engineering Contradiction:
Improvetherapeutic effectVSAvoidselectivity and potency
Core Design Contradiction:
ReliabilityVSMeasurement precision

Solution Approach 1:

The patent systematically varies multiple parameters of the cholic acid structure (substituents at positions 1, 2, 3, 4, 6, 7, 12, 23, and conjugation types) to optimize both potency and selectivity. The defined chemical formulas with specific R-group variations enable precise tuning of receptor interaction properties, achieving superior selectivity for TGR5 over other bile acid receptors.

Inventive Principle:
Principle #35Parameter changes

Solution Approach 2:

The patent creates composite molecular structures by combining the cholic acid core with various functional groups and substitution patterns. These composite structures integrate multiple pharmacophoric elements that work synergistically to enhance TGR5 binding affinity and selectivity, resulting in compounds with improved therapeutic profiles compared to natural bile acids.

Inventive Principle:
Principle #40Composite materials

Applied Scientific Principles

This section explains which scientific principles are used to turn an abstract innovation direction into a practical engineering solution.

Function Achieved in This Case

The described compounds effectively modulate TGR5, demonstrating therapeutic potential in treating and preventing various diseases by improving metabolic profiles, weight management, and enhancing energy expenditure without the toxicity concerns associated with high doses of cholic acid.

Implementation Method 1

TGR5 receptor is a G-protein-coupled receptor that has been identified as a cell-surface receptor that is responsive to bile acids

Methodology Applied
Scientific EffectG-protein-coupled receptor activation:

Implementation Method 2

Bile acids have been shown to induce internalization of the TGR5 fusion protein from the cell membrane to the cytoplasm

Methodology Applied
Scientific EffectReceptor-mediated endocytosis:

Implementation Method 3

TGR5 is associated with the intracellular accumulation of cAMP, that is widely expressed in diverse cell types

Methodology Applied
Scientific EffectcAMP accumulation:

Implementation Method 4

the cAMP-dependent induction of type 2 iodothyronine deiodinase (D2), which by, locally converting T4 into T3, gives rise to increased thyroid hormone activity

Methodology Applied
Scientific EffectDeiodination:

Data Source

PatentEP2698375B1TGR5 modulators and method of use thereof
Publication Date: 2018.02.21 INTERCEPT PHARMACEUTICALS INC
  • EP2698375B1 patent drawingFigure 1
  • EP2698375B1 patent drawingFigure 2~2B
  • EP2698375B1 patent drawingFigure 2C~2D

AI summary

The invention relates to compounds of Formula A: (A) or a salt, solvate, hydrate, or prodrug thereof. The compounds of Formula A are TGR5 modulators useful for the treatment of various diseases, including metabolic disease, inflammatory disease, liver disease, autoimmune disease, cardiac disease, kidney disease, cancer and gastrointestinal disease.