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Therapeutics

229Aging Biotech
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LW-1017

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First-in-human TRPML1 agonist designed to restore lysosomal function and autophagic clearance in neurodegenerative disease.

Modality
oral brain-penetrant small-molecule TRPML1 agonist
Availability (US)
Phase 1; first healthy volunteer was dosed May 5, 2026 in a single- and multiple-ascending-dose study
Biomarkers
safety, tolerability, pharmacokinetics, TRPML1 activation, lysosomal function, autophagic flux, and aggregate biology
Hallmark targeted
disabled autophagy, loss of proteostasis, intracellular waste accumulation
Kind
investigational drug
Clinical trials
Company-reported Phase 1 SAD/MAD healthy-volunteer study in Melbourne, Australia; no ClinicalTrials.gov identifier is stated in the official release