High-Surface-Area Lyophilized Arsenic Trioxide for Oral Delivery

Resolve Bottlenecks,
Find Innovative Solutions
Generate Solutions

Solution Overview

Problem

Conventional arsenic trioxide treatment for hematological malignancies requires daily intravenous administration, which is inconvenient, risky, and expensive, and there is no FDA-approved oral formulation, due to its insolubility and low bioavailability.

Innovation Solution

A lyophilized composition comprising arsenic trioxide is developed, which is solubilized and lyophilized to enhance bioavailability, allowing oral administration in the form of capsules or tablets, with a method involving alkalization, acidification, and freeze-drying processes to create a stable and effective oral formulation.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If intravenous administration of arsenic trioxide is used, then therapeutic efficacy is achieved, but treatment convenience and patient quality of life deteriorate

Engineering Contradiction:
Improvetherapeutic efficacyVSAvoidtreatment convenience
Core Design Contradiction:
ReliabilityVSEase of operation

Solution Approach 1:

The patent changes the physical and chemical parameters of arsenic trioxide by converting it from an insoluble crystalline form to a soluble lyophilized formulation. This involves dissolving arsenic trioxide in alkaline solution, adjusting pH, and performing freeze-drying to create a highly soluble powder that can be orally administered, thereby improving treatment convenience while maintaining therapeutic efficacy

Inventive Principle:
Principle #35Parameter changes

Solution Approach 2:

The patent creates a composite pharmaceutical formulation by combining arsenic trioxide with alkaline agents (such as sodium bicarbonate or citrate buffers) and lyoprotectants (such as mannitol or sucrose). This composite approach enhances solubility and stability, enabling oral administration while preserving the therapeutic activity of arsenic trioxide

Inventive Principle:
Principle #40Composite materials

2Reliability

If intravenous administration of arsenic trioxide is used, then therapeutic effect is achieved, but treatment cost and risk increase

Engineering Contradiction:
Improvetherapeutic effectVSAvoidtreatment complexity
Core Design Contradiction:
ReliabilityVSDevice complexity

Solution Approach 1:

The patent extracts the active therapeutic component (arsenic trioxide) from its conventional intravenous formulation and isolates it in a purified, lyophilized form. This extraction allows the drug to be delivered through a simpler oral route, eliminating the need for complex intravenous administration infrastructure and reducing treatment costs while maintaining therapeutic effectiveness

Inventive Principle:
Principle #2Taking out (Extraction)

3Ease of operation

If oral formulation of arsenic trioxide is developed, then treatment convenience improves, but bioavailability and solubility worsen

Engineering Contradiction:
Improvetreatment convenienceVSAvoidbioavailability
Core Design Contradiction:
Ease of operationVSReliability

Solution Approach 1:

The patent dramatically changes the solubility parameter of arsenic trioxide by dissolving it in alkaline solution (pH 10-12) followed by pH adjustment and freeze-drying. This process creates a lyophilized powder with enhanced solubility and bioavailability, ensuring that oral administration achieves sufficient drug absorption while maintaining treatment convenience

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

The lyophilized arsenic trioxide formulation provides systemic bioavailability comparable to intravenous administration, offering a convenient, less risky, and cost-effective treatment option for hematological malignancies with improved patient compliance and quality of life.

Implementation Method 1

A lyophilized composition comprising arsenic trioxide is developed, which is solubilized and lyophilized to enhance bioavailability

Methodology Applied
Scientific EffectSolubilization: Solvation

Implementation Method 2

lyophilized composition comprising arsenic trioxide is developed, which is solubilized and lyophilized

Methodology Applied
Scientific EffectFreeze-drying: Freeze Drying

Data Source

PatentEP4599823A1High surface-area lyophilized compositions comprising arsenic for oral administration in patients
Publication Date: 2025.08.13 QUETZAL THERAPEUTICS LLC
  • EP4599823A1 patent drawingFigure 1
  • EP4599823A1 patent drawingFigure 2
  • EP4599823A1 patent drawingFigure 3

AI summary

The present invention relates to treating malignancies such as tumors or cancers by orally administering lyophilized compositions comprising arsenic to a subject in such need. Malignancies include various hematological malignancies, such as acute myeloid leukemia (AML) including acute promyelocytic leukemia (APL), myelodysplastic syndrome (MDS), multiple myeloma (MM) and lymphomas and solid tumors including glioblastoma multiforme and breast cancer. Arsenic treatment has shown great promise in the treatment of several cancers but requires daily intravenous (IV) administration. This invention relates to a novel formulation comprising a lyophilized compositions comprising arsenic. As a result, the formulation facilitates a systemic bioavailability comparable to that of intravenous (IV) administration of arsenic trioxide currently practiced. The present invention also relates to a method for lyophilizing the arsenic trioxide, preparing the oral formulation comprising lyophilized compositions comprising arsenic, and a method for treating a subject with malignancies using the oral formulation.