Endlyz Therapeutics emerges with pipeline for Parkinson’s disease

Research targets lysosomal function restoration for neurodegeneration

Endlyz Therapeutics has launched from stealth, unveiling its pipeline of small molecule modulators designed to restore lysosomal function in Parkinson’s disease (PD) and other neurodegenerative conditions.

The company also announced a $2.2 million grant from the Michael J. Fox Foundation for Parkinson’s Research and a collaboration with Prof. Peter Vangheluwe at KU Leuven.

Endlyz focuses on ATP13A2 and ATP10B, polyamine and lipid transporter enzymes critical for neuronal health. Its lead small molecule potentiators of ATP13A2, linked to the PARK9 gene, aim to combat endo-lysosomal dysfunction, a key driver of neurodegeneration.

Dr. Joanna Wolak, CEO and co-founder of Endlyz, said: “Our small-molecule ATP13A2-targeting series represents a ground-breaking approach to treating Parkinson’s disease. By restoring lysosomal function, we aim to slow or halt disease progression rather than merely address symptoms.”

The collaboration with Prof. Vangheluwe, a world expert in lysosomal transporters, will leverage his research and CD3 at KU Leuven’s drug discovery expertise. Supported by Quantum AI, the partnership aims to accelerate candidate drug discovery for activating ATP10B.

Endlyz is supported by leading investors, including SV Health Investors’ Dementia Discovery Fund, Oxford Science Enterprises, AbbVie Ventures, and Parkinson’s UK. The company plans to secure additional funding in 2025 to advance its lead programme through IND-enabling studies, expand its pipeline, and explore new indications.

The Michael J. Fox Foundation funding will enable biomarker discovery to bridge preclinical and clinical development, enhancing translational research and therapeutic innovation for neurodegeneration.