Cationic Steroidal Antimicrobial Salt Synthesis

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Solution Overview

Problem

Clinical use of antimicrobial peptides is hindered by high production costs, susceptibility to proteases, and deactivation by lung mucosa components, necessitating the development of non-peptide mimics that maintain broad-spectrum antibacterial activity while being more stable and cost-effective.

Innovation Solution

Development of 1,5-naphthalenedisulfonic acid addition salts of cationic steroidal antimicrobials (CSAs), which are synthesized into solid, crystalline, or flowable forms, offering improved stability and pharmaceutical administerability through specific processes involving solvent dilution, acid addition, and temperature cycling.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If antimicrobial peptides are used for clinical treatment, then broad-spectrum antibacterial activity is achieved, but production cost increases and susceptibility to proteases occurs

Engineering Contradiction:
Improveantibacterial activityVSAvoidproduction cost
Core Design Contradiction:
ReliabilityVSEase of manufacture

Solution Approach 1:

The patent creates non-peptide mimic compounds (ceragenins) that copy the essential amphiphilic morphology and cationic charge distribution of natural antimicrobial peptides. By using small molecule steroidal or non-steroidal structures with hydrophobic faces and cationic faces, the invention reproduces the antibacterial mechanism without using peptide bonds, thereby eliminating protease susceptibility and reducing production costs through simpler synthesis routes.

Inventive Principle:
Principle #26Copying

Solution Approach 2:

The patent modifies the chemical structure parameters by replacing peptide backbones with small molecule structures containing hydrophobic and cationic regions. This structural parameter change maintains the amphiphilic morphology necessary for membrane disruption while improving stability against enzymatic degradation and simplifying manufacturing processes.

Inventive Principle:
Principle #35Parameter changes

2Reliability

If antimicrobial peptides are used, then antibacterial activity is achieved, but susceptibility to proteolytic degradation occurs

Engineering Contradiction:
Improveantibacterial activityVSAvoidproteolytic stability
Core Design Contradiction:
ReliabilityVSStability of the object's composition

Solution Approach 1:

The patent creates non-peptide mimic compounds (ceragenins) that copy the essential amphiphilic morphology and cationic charge distribution of natural antimicrobial peptides. By using small molecule steroidal or non-steroidal structures with hydrophobic faces and cationic faces, the invention reproduces the antibacterial mechanism without using peptide bonds, thereby eliminating protease susceptibility and reducing production costs through simpler synthesis routes.

Inventive Principle:
Principle #26Copying

Solution Approach 2:

The patent employs small molecule structures that are inherently more stable than peptides against enzymatic degradation. These simplified molecular structures do not contain peptide bonds that proteases can cleave, effectively making them resistant to biological degradation without requiring complex stabilization strategies.

Inventive Principle:
Principle #27Cheap short-living objects (Disposable)

3Ease of manufacture

If non-peptide mimics are developed, then production cost decreases and protease stability increases, but water solubility and charge density control becomes challenging

Engineering Contradiction:
Improveproduction costVSAvoidsolubility control
Core Design Contradiction:
Ease of manufactureVSAdaptability or versatility

Solution Approach 1:

The patent modifies the chemical structure parameters by replacing peptide backbones with small molecule structures containing hydrophobic and cationic regions. This structural parameter change maintains the amphiphilic morphology necessary for membrane disruption while improving stability against enzymatic degradation and simplifying manufacturing processes.

Inventive Principle:
Principle #35Parameter changes

Solution Approach 2:

The patent applies local quality by creating distinct hydrophobic faces and cationic faces on the molecular structure. This spatial separation of properties allows the molecule to interact with bacterial membranes through hydrophobic insertion while the cationic face provides electrostatic attraction to negatively charged membrane components, enabling controlled solubility and membrane targeting.

Inventive Principle:
Principle #3Local quality

4Reliability

If CSA salts are synthesized through traditional methods, then antimicrobial activity is achieved, but manufacturing complexity and storage stability issues occur

Engineering Contradiction:
Improveantimicrobial activityVSAvoidsynthesis complexity
Core Design Contradiction:
ReliabilityVSDevice complexity

Solution Approach 1:

The patent employs preliminary action by pre-forming the free base form of the ceragenin compound through streamlined synthesis, then performing a simple salt formation step with appropriate acids to generate the final stable product. This two-stage approach with predetermined intermediates simplifies manufacturing and ensures consistent product quality.

Inventive Principle:
Principle #10Preliminary action

Solution Approach 2:

The patent utilizes phase transitions by controlling the formation of crystalline salt forms from solution. By selecting appropriate acids and solvents, the ceragenin compounds form stable crystalline salts with improved storage stability and defined physical properties, facilitating standardized manufacturing and quality control.

Inventive Principle:
Principle #36Phase transitions

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 CSA salts exhibit comparable or enhanced antimicrobial activity, stability, and pharmaceutical properties, such as solubility and storage stability, simplifying synthesis and manufacturing compared to existing methods.

Implementation Method 1

diluting the free base of a CSA with a solvent; adding at least one equivalent of an acid to the diluted CSA in solvent

Methodology Applied
Scientific EffectSolvation: Solvation

Implementation Method 2

precipitating or temperature cycling the reaction mixture; isolating a CSA salt

Methodology Applied
Scientific EffectPrecipitation: Precipitation

Implementation Method 3

temperature cycling the reaction mixture; The temperature cycling may be conducted for at least about 48 hours

Methodology Applied
Scientific EffectTemperature cycling: Phase Change

Data Source

PatentEP3285751B1Cationic steroidal antimicrobial salts
Publication Date: 2020.10.07 BRIGHAM YOUNG UNIV
  • EP3285751B1 patent drawingFigure 1~2
  • EP3285751B1 patent drawingFigure 3~4
  • EP3285751B1 patent drawingFigure 5

AI summary

Disclosed herein are acid addition salts of cationic steroidal antimicrobials ("CSAs" or "ceragenins") and methods of making the same. Particularly advantageous salt forms are identified, such as 1,5-naphthalenedisulfonic acid addition salts and sulfate addition salts. The acid addition salts may be formulated for treating subjects with ailments responsive to CSAs, including but not limited to treating bacterial infections. Embodiments include formulations and methods of administering acid addition salts of CSAs.