Irreversible Btk Inhibitors via Michael Acceptor Covalent Bonding

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Solution Overview

Problem

Current kinase inhibitors lack specificity and efficacy in targeting Bruton's tyrosine kinase (Btk) and its homologs, leading to off-target effects and reduced therapeutic benefits in treating diseases associated with tyrosine kinase activity.

Innovation Solution

Development of irreversible kinase inhibitors that form a covalent bond with a cysteine residue on Btk and its homologs, utilizing a Michael acceptor moiety to preferentially target these kinases, thereby providing sustained inhibition and reducing off-target interactions.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If irreversible inhibitors with Michael acceptor moiety are used to target Btk, then specificity and efficacy are improved, but device complexity increases

Engineering Contradiction:
ImprovespecificityVSAvoidmolecular structure complexity
Core Design Contradiction:
ReliabilityVSDevice complexity

Solution Approach 1:

The inhibitor molecule is divided into distinct functional segments: a Michael acceptor moiety (for irreversible covalent bonding to cysteine residue), a kinase domain binding region (for specific recognition), and a linker portion. This segmentation allows each component to fulfill its specific function while maintaining overall molecular complexity at a manageable level.

Inventive Principle:
Principle #1Segmentation

Solution Approach 2:

The inhibitor employs local quality by concentrating the reactive Michael acceptor functionality at a specific location within the molecule that corresponds to the cysteine residue binding site. This localized reactive center ensures selective covalent bonding only at the intended target site on Btk, enhancing specificity without requiring complex overall molecular architecture.

Inventive Principle:
Principle #3Local quality

2Duration of action of moving object

If irreversible covalent bonding is used to achieve sustained inhibition, then duration of action is improved, but object-generated harmful factors increase

Engineering Contradiction:
Improveduration of kinase inhibitionVSAvoidoff-target effects
Core Design Contradiction:
Duration of action of moving objectVSObject-generated harmful factors

Solution Approach 1:

The inhibitor is designed with a preliminary reversible binding phase that occurs before irreversible covalent bonding. The kinase domain binding region first forms a stable reversible complex with Btk, positioning the Michael acceptor moiety precisely at the cysteine residue. Only after this preliminary recognition does covalent bonding occur, ensuring that the inhibitor is already correctly positioned and reducing the likelihood of off-target reactions.

Inventive Principle:
Principle #10Preliminary action

Solution Approach 2:

The reversible binding interaction serves as an intermediary step between initial molecular encounter and final irreversible covalent bonding. This intermediary phase acts as a selection mechanism, allowing only the correct target (Btk with matching binding pocket) to proceed to covalent inhibition, thereby preventing off-target effects while maintaining sustained duration of action.

Inventive Principle:
Principle #24Intermediary (Mediator)

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 irreversible inhibitors effectively and specifically target Btk and its homologs, offering therapeutic benefits in treating autoimmune diseases, inflammatory conditions, and cancers by providing sustained kinase inhibition with reduced side effects.

Implementation Method 1

irreversible inhibitors of Btk that form a covalent bond with a cysteine residue on Btk

Methodology Applied
Scientific EffectCovalent bonding: Chemical Bonding

Implementation Method 2

the irreversible inhibitor includes a Michael acceptor moiety

Methodology Applied
Scientific EffectMichael addition: Chemical Bonding

Data Source

PatentEP2139487B1Inhibitors of bruton's tyrosine kinase
Publication Date: 2015.11.11 PHARMACYCLICS LLC
  • EP2139487B1 patent drawingFigure 1A~1C
  • EP2139487B1 patent drawingFigure 2
  • EP2139487B1 patent drawingFigure 3

AI summary

Described herein are irreversible kinase inhibitor compounds, methods for synthesizing such irreversible inhibitors, and methods for using such irreversible inhibitors in the treatment of diseases. Further described herein are methods, assays and systems for determining an appropriate irreversible inhibitor of a protein, including a kinase.