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LW-1017
- Development stage
- Phase 1
- Lead developer
- Lysoway Therapeutics
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
LW-1017 is a brain-penetrant small-molecule agonist of the lysosomal calcium channel TRPML1 (Mucolipin-1), developed by Lysoway Therapeutics for the treatment of age-related neurodegenerative disorders. By activating TRPML1, the drug aims to restore the function of the autophagy-lysosomal pathway, which is often impaired in diseases like Parkinson's and Alzheimer's. Preclinical data indicates that LW-1017 effectively crosses the blood-brain barrier, reduces the accumulation of alpha-synuclein, amyloid-beta, and phosphorylated tau, and provides neuroprotection. The candidate has completed IND-enabling studies and is scheduled for first-in-human Phase I clinical trials in May 2026.
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