Novel Maytansinoid Compounds for Stable Antibody-Drug Conjugates

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Solution Overview

Problem

There is a need for novel, potent cytotoxic molecules for use in antibody-drug conjugates (ADCs) that can be efficiently conjugated with antibodies to produce more stable and effective ADCs with reduced non-specific toxicity.

Innovation Solution

Development of novel maytansinoid compounds with improved cytotoxic activity and stability, along with a new method for their synthesis, allowing for efficient preparation and conjugation with antibodies using specific linker technologies.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If conventional cytotoxic agents are used in ADCs, then therapeutic activity is achieved, but non-specific toxicity increases and stability decreases

Engineering Contradiction:
ImproveADC stabilityVSAvoidnon-specific toxicity
Core Design Contradiction:
ReliabilityVSObject-affected harmful factors

Solution Approach 1:

The patent introduces specific structural modifications to the maytansinoid core at defined positions (R1-R6 substituents, oxidation states at specific carbon positions) to create localized improvements in stability and selectivity. Different regions of the molecule are optimized for different functions: the core maintains cytotoxic activity while specific side chains and functional groups are engineered to reduce non-specific binding and improve ADC stability.

Inventive Principle:
Principle #3Local quality

Solution Approach 2:

The patent systematically varies multiple molecular parameters including oxidation states (hydroxyl vs oxo groups), substituent types (alkyl, aryl, heteroaryl groups), and functional group positions to optimize the balance between stability and toxicity. Specific embodiments explore different oxidation states at C-4, C-6, and other positions, along with various R group configurations, to achieve the desired therapeutic window.

Inventive Principle:
Principle #35Parameter changes

2Reliability

If maytansinoid compounds are conjugated to antibodies, then targeted delivery is achieved, but conjugation complexity increases

Engineering Contradiction:
Improvetargeted delivery efficacyVSAvoidconjugation process complexity
Core Design Contradiction:
ReliabilityVSDevice complexity

Solution Approach 1:

The patent incorporates pre-installed functional groups (carboxyl, hydroxyl, amino, thiol groups) on the maytansinoid molecules before conjugation. These pre-positioned reactive groups enable direct conjugation to antibody residues without requiring complex multi-step synthesis or intermediate linkers, thereby simplifying the overall conjugation process while maintaining targeted delivery efficacy.

Inventive Principle:
Principle #10Preliminary action

Solution Approach 2:

The patent designs maytansinoid derivatives with multiple types of reactive functional groups that can conjugate to different antibody residues (lysine, cysteine, N-terminal amino groups). This multi-functional approach allows a single maytansinoid derivative to be used in various conjugation strategies, reducing the need for multiple specialized reagents and simplifying the conjugation workflow.

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

3Power

If cytotoxic potency is increased, then therapeutic efficacy is improved, but toxicity to healthy tissue increases

Engineering Contradiction:
Improvecytotoxic potencyVSAvoidtoxicity to healthy tissue
Core Design Contradiction:
PowerVSObject-affected harmful factors

Solution Approach 1:

The patent optimizes specific regions of the maytansinoid molecule to enhance binding affinity and cytotoxic potency at the target site while leaving other regions modified to reduce non-specific interactions. The core maytansinoid structure is preserved for potency, while peripheral substituents are engineered to improve selectivity and reduce off-target effects.

Inventive Principle:
Principle #3Local quality

Solution Approach 2:

The patent systematically adjusts molecular parameters including substituent electronics, steric bulk, and hydrogen bonding capacity to fine-tune the balance between potency and selectivity. By varying oxidation states, substituent types, and their positions, the patent achieves high cytotoxic activity against cancer cells while reducing toxicity to healthy tissues through improved target specificity.

Inventive Principle:
Principle #35Parameter changes

Data Source

PatentEP3512856B1Novel cytotoxic agents and conjugates thereof
Publication Date: 2021.03.24 POLYTHERICS LTD
  • EP3512856B1 patent drawingFigure 1~2
  • EP3512856B1 patent drawingFigure 3
  • EP3512856B1 patent drawingFigure 4a~4b

AI summary

Provided herein are novel maytansinoid compounds of general formula I. Also provided herein are conjugates comprising the compounds linked to a binding protein via a linker, and conjugating reagents comprising the compounds attached via a linker to at least one functional group capable of reacting with a binding protein. Also provided herein are pharmaceutical compositions comprising the compounds and conjugates, therapeutic methods and uses involving the compounds and conjugates, for example in cancer therapy, and novel synthetic processes.