Pyrazole Triazole SOCE Modulators for STIM1 Orai1 Specificity

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Solution Overview

Problem

Current modulators for Store Operated Calcium Entry (SOCE) lack specificity and potency, particularly for targeting STIM1 and Orai1 proteins, which are crucial for treating diseases associated with abnormal SOCE, such as immunodeficiencies and muscle disorders.

Innovation Solution

Development of a class of 5-(trifluoromethyl)-1H-pyrazoles with a phenyl ring carrying a triazole in the para-position, which act as novel SOCE modulators, specifically targeting STIM1 and Orai1 to regulate calcium entry and address pathological conditions.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If current modulators are used for SOCE, then calcium entry can be modulated, but specificity and potency are insufficient

Engineering Contradiction:
ImprovespecificityVSAvoidlack of potency
Core Design Contradiction:
ReliabilityVSObject-generated harmful factors

Solution Approach 1:

The patent applies parameter changes by modifying the chemical structure of pyrazole compounds through systematic variation of substituents at specific positions (R1-R6). This structural optimization enhances both potency and specificity of SOCE modulation. The patent identifies optimal parameter ranges for substituents that maximize therapeutic effect while minimizing off-target activity.

Inventive Principle:
Principle #35Parameter changes

Solution Approach 2:

The patent applies local quality by introducing specific functional groups at defined positions on the pyrazole core structure. Different substituents (halogens, alkyl groups, aryl groups, heteroaryl groups) are placed at specific positions (R1-R6) to create localized interactions with STIM1 and Orai1 proteins, thereby achieving high specificity for CRAC channel modulation.

Inventive Principle:
Principle #3Local quality

2Reliability

If existing agents are used to modulate SOCE, then some calcium entry regulation is achieved, but selectivity over CRAC channels is poor

Engineering Contradiction:
ImproveselectivityVSAvoidpleiotropic effects on other channels
Core Design Contradiction:
ReliabilityVSObject-generated harmful factors

Solution Approach 1:

The patent applies local quality by introducing specific functional groups at defined positions on the pyrazole core structure. Different substituents (halogens, alkyl groups, aryl groups, heteroaryl groups) are placed at specific positions (R1-R6) to create localized interactions with STIM1 and Orai1 proteins, thereby achieving high specificity for CRAC channel modulation.

Inventive Principle:
Principle #3Local quality

Solution Approach 2:

The patent applies parameter changes by modifying the chemical structure of pyrazole compounds through systematic variation of substituents at specific positions (R1-R6). This structural optimization enhances both potency and specificity of SOCE modulation. The patent identifies optimal parameter ranges for substituents that maximize therapeutic effect while minimizing off-target activity.

Inventive Principle:
Principle #35Parameter changes

Data Source

PatentEP3468964B1Modulators of SOCE, compositions, and uses thereof
Publication Date: 2020.12.30 UNIV DEGLI STUDI DEL PIEMONTE ORIENTALEAMEDEO AVOGADRO
  • EP3468964B1 patent drawingFigure 1
  • EP3468964B1 patent drawingFigure 2A~2B
  • EP3468964B1 patent drawingFigure 3A~3B

AI summary

Compounds of Formula (I) able to modulate Store Operated Calcium Entry (SOCE). The disclosure also relates to the use of compounds of formula (I) for treatment of pathological conditions in which SOCE modulation might be beneficial, such as neglecting disorders linked to loss-or gain-of-function STIM1/Orai1 mutations, allergic disorders, pain, inflammatory diseases, autoimmune diseases or disorders, cancer and other proliferative diseases, neurodegenerative disorders, myelodysplastic syndromes, haematological diseases, cardiovascular diseases, degenerative diseases of the musculoskeletal system, liver diseases and disorders, kidney diseases, type I diabetes, graft rejection, graft-versus-host disease, allogeneic or xenogeneic transplantation, thyroiditis and viral infections.