Methods for treating patients with hematologic malignancies

A compound of Formula (1) effectively treats AML patients with mutant forms of NPM1, DNMT3A, RAS, or FLT3 by addressing drug resistance and targeting AML stem/progenitor cells, achieving significant clinical responses and improving treatment outcomes.

WO2024124199A9 Publication Date: 2025-06-12APTOSE BIOSCIENCES INC +2
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Patent Information

Application Number
PCT/US2023/083227
Authority / Receiving Office
WO · WO
Patent Type
Applications
Current Assignee / Owner
Priority Date
2022-12-10
Filing Date
2023-12-08
Publication Date
2025-06-12

AI Technical Summary

Technical Problem

Current treatments for acute myeloid leukemia (AML), particularly those with mutations such as NPM1, DNMT3A, RAS, or FLT3, face challenges including drug resistance and inability to target AML stem/progenitor cells, leading to disease recurrence and limited long-term efficacy.

Method used

The use of a compound of Formula (1) or its pharmaceutically acceptable salts, solvates, stereoisomers, tautomers, or combinations thereof, which is administered to subjects with mutant forms of NPM1, DNMT3A, RAS, or a combination thereof, to treat cancer, including AML.

Benefits of technology

The compound demonstrates clinical benefits in treating AML patients with adverse mutations, including those resistant to conventional therapeutic agents, by achieving significant clinical responses and potentially overcoming treatment resistance.

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Abstract

The present disclosure relates to compounds of Formula 1 and pharmaceutical compositions thereof for the treatment of cancer in a subject having a mutant form of NPM1, DNMT3A, RAS, or a combination thereof, or for specific treatments of subjects with relapsed or treatment-refractory (R / R) acute myeloid leukemia (AML).
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