PLA2g15 inhibitors

PLA2G15 inhibitors represented by formula (I) address the lack of potent inhibitors for treating lysosomal storage diseases and neurodegenerative disorders by inhibiting PLA2G15 activity, restoring BMP balance and improving disease outcomes.

WO2025153720A1 Publication Date: 2025-07-24SCENIC BIOTECH BV
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Patent Information

Application Number
PCT/EP2025/051217
Authority / Receiving Office
WO · WO
Patent Type
Applications
Current Assignee / Owner
Priority Date
2024-06-10
Filing Date
2025-01-17
Publication Date
2025-07-24

AI Technical Summary

Technical Problem

There is a need for potent and selective inhibitors of PLA2G15 proteins, which are clinically relevant but few have been identified, particularly for treating lysosomal storage diseases, Alzheimer's disease, and Parkinson's disease.

Method used

Development of PLA2G15 inhibitors represented by formula (I) or their salts and solvates, which can inhibit the activity of PLA2G15 proteins, thereby addressing lysosomal dysregulation and associated diseases.

Benefits of technology

The inhibitors effectively decrease PLA2G15 activity, leading to restored balance of bis(monoacylglycerol)-phosphate (BMP) levels, alleviating lysosomal dysfunction and providing therapeutic benefits for conditions like Niemann Pick type C disease, Alzheimer's disease, and Parkinson's disease.

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Abstract

The current invention relates to PLA2G15 inhibitors represented by formula (I), and corresponding compositions and uses. Preferably, the inhibitors and compositions are for use in the treatment of lysosomal storage diseases, Alzheimer's disease and Parkinson's disease; in particular for use in the treatment of Niemann Pick type C or a neuronal ceroid lipofuscinosis such as CLN3 disease or Batten disease, CLN5 disease, or GRN frontotemporal dementia.
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