O-Substituted Hydroxamic Acids for Efficient HNO Generation

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Solution Overview

Problem

Current nitroxyl (HNO) donors for therapeutic applications face challenges in generating HNO efficiently under physiological conditions without producing toxic byproducts, particularly cyanide, and have limited reactivity at neutral pH.

Innovation Solution

Development of O-substituted hydroxamic acids that generate nitrosocarbonyl intermediates under physiological conditions, which can be hydrolyzed to HNO, offering improved reactivity and reduced toxicity through specific structural modifications.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Object-affected harmful factors

If traditional HNO donors (Angeli's salt, Piloty's acid, acyloxy nitroso compounds) are used, then HNO can be generated, but toxic byproducts are produced and reactivity is limited at neutral pH

Engineering Contradiction:
ImprovetoxicityVSAvoidHNO generation efficiency
Core Design Contradiction:
Object-affected harmful factorsVSProductivity

Solution Approach 1:

The patent modifies the molecular structure of hydroxamic acid donors by changing the leaving group parameters and substituent patterns. Specifically, it uses O-acylated hydroxamic acids with modified carbonyl groups that alter the hydrolysis kinetics and HNO release profile, enabling efficient generation at physiological pH while producing non-toxic byproducts

Inventive Principle:
Principle #35Parameter changes

Solution Approach 2:

The patent converts the previously harmful cyanide byproduct into a beneficial non-toxic leaving group. By replacing the cyanide moiety with modified hydroxamic acid structures that release benign byproducts (such as substituted hydroxylamines), the system maintains HNO generation capability while eliminating toxicity

Inventive Principle:
Principle #22Blessing in disguise (Convert harm into benefit)

2Ease of operation

If N,O-bis-acylated hydroxylamine derivatives with arenesulfonyl leaving groups are used, then HNO can be generated under physiological conditions, but amide hydrolysis and acyl migration compete with HNO generation

Engineering Contradiction:
Improvephysiological compatibilityVSAvoidHNO generation yield
Core Design Contradiction:
Ease of operationVSReliability

Solution Approach 1:

The patent applies local quality by introducing specific substituents at particular positions on the hydroxamic acid molecule. The R1 and R2 substituents on the carbonyl group are carefully selected to modulate the electrophilicity and steric properties locally, enhancing HNO release while suppressing competing hydrolysis and migration reactions

Inventive Principle:
Principle #3Local quality

Solution Approach 2:

The patent creates composite molecular structures by combining hydroxamic acid cores with specific acyl groups and substituents. This composite approach integrates multiple functional elements that work synergistically: the hydroxamic acid provides HNO donor capability, while the modified acyl groups with specific substituents control reaction selectivity and stability

Inventive Principle:
Principle #40Composite materials

3Object-affected harmful factors

If O-acylated hydroxamic acids with arenesulfonyl leaving groups are used, then HNO can be generated upon hydrolysis, but less than 5% HNO is generated at neutral pH

Engineering Contradiction:
Improvetoxicity reductionVSAvoidHNO generation yield at neutral pH
Core Design Contradiction:
Object-affected harmful factorsVSProductivity

Solution Approach 1:

The patent systematically varies key molecular parameters including the acyl group composition, substituent types (R1 and R2), and leaving group structures. These parameter changes optimize the pH-dependent hydrolysis kinetics, shifting the reaction equilibrium toward HNO generation at physiological pH while maintaining non-toxic byproduct formation

Inventive Principle:
Principle #35Parameter changes

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

These compounds achieve efficient HNO generation with enhanced yield and reduced toxicity, making them suitable for treating conditions like cardiovascular diseases and heart failure without the drawbacks of existing donors.

Implementation Method 1

Following deprotonation and loss of HX (e.g., a pyrazolone), the nitrosocarbonyl intermediate is subsequently hydrolyzed to HNO

Methodology Applied
Scientific EffectHydrolysis: Hydrolysis

Data Source

PatentEP3490975B1O-substituted hydroxamic acids
Publication Date: 2021.05.05 JOHNS HOPKINS UNIVERSITY
  • EP3490975B1 patent drawingFigure 1
  • EP3490975B1 patent drawingFigure 2
  • EP3490975B1 patent drawingFigure 3

AI summary

O-substituted hydroxamic acids with carbon-based leaving groups as HNO donors are disclosed. Pharmaceutical compositions and kits comprising such compounds, and methods of using such compounds or pharmaceutical compositions also are disclosed.