BCAT2 inhibitors

WO2024238820A3PCT designated stage expired Publication Date: 2024-12-26AGIOS PHARMACEUTICALS INC
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Patent Information

Application Number
PCT/US2024/029738
Authority / Receiving Office
WO · WO
Patent Type
Applications
Current Assignee / Owner
Priority Date
2023-05-17
Filing Date
2024-05-16
Publication Date
2024-12-26

AI Technical Summary

Technical Problem

Current therapies are inadequate for treating branched chain amino acid (BCAA)-catabolism-related diseases such as propionic acidemia and methylmalonic acidemia, which result in toxic metabolite accumulation leading to severe clinical complications, including metabolic decompensations, neurological deficits, and cardiac issues, with no approved market therapy available for these conditions.

Method used

Development of BCAT2 inhibitors, specifically compounds of Formula I and Formula II, which are used to inhibit branched-chain amino acid aminotransferase 2 (BCAT2), thereby reducing the flux through the BCAA catabolic pathway and lowering toxic metabolite levels, presented in pharmaceutical compositions for treating BCAA-catabolism-related diseases.

Benefits of technology

The BCAT2 inhibitors effectively reduce toxic metabolite levels, potentially alleviating the severe clinical complications associated with BCAA-catabolism-related diseases, providing a therapeutic benefit for conditions like propionic acidemia and methylmalonic acidemia.

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Abstract

The disclosure is directed to compounds, and pharmaceutically acceptable salts thereof, that inhibit branched-chain amino acid aminotransferase 2 (BCAT2), pharmaceutical compositions comprising those compounds, and methods of using those compounds for treating branched chain amino acid (BCAA)-catabolism-related diseases. The disclosure is also directed to in vivo models for the study of disorders characterized by defects in the metabolism of BCAAs.
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Citation Information

Patent Citations

  • BCAT modulation

    WO2021007350A1