LW-1017 (TRPML1 agonist)
Cognitive · TRPML1 agonist (autophagy)
Tier D+
Bottom line
Read Off Label grades LW-1017 (TRPML1 agonist) as D+ (3.7/10) based on preclinical evidence, med benefit magnitude, and a med-risk safety profile.
Lysoway Therapeutics.
Typical use: Oral; Phase 1 single/multiple ascending dose — Investigational.
What this is
Lysoway Therapeutics. First TRPML1 agonist in the clinic, first participant dosed May 2026 in a Phase 1 SAD/MAD study (Melbourne) in healthy volunteers, developed for Alzheimer's/Parkinson's. A clean safety and target-engagement readout would be early human proof that pharmacologically boosting autophagy works. No published human data yet. Frontier. Company-sponsored.
Mechanism
Brain-penetrant small-molecule agonist of TRPML1, a lysosomal calcium channel gating autophagy-lysosomal clearance, the proteostasis pathway underlying rapamycin, spermidine and fasting; aimed at neurodegenerative protein aggregation.
Dose & route
Oral; Phase 1 single/multiple ascending dose
Citations
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Common questions
- Does LW-1017 (TRPML1 agonist) work?
- Read Off Label rates the evidence for LW-1017 (TRPML1 agonist) as Preclinical and the benefit magnitude as med, producing an overall grade of D+ (3.7/10). Lysoway Therapeutics.
- Is LW-1017 (TRPML1 agonist) safe?
- LW-1017 (TRPML1 agonist) has a med risk profile in published human data. Legal status: Investigational (Phase 1 SAD/MAD). This is not medical advice — see the disclaimer.
- What is the typical dose for LW-1017 (TRPML1 agonist)?
- Oral; Phase 1 single/multiple ascending dose
- How does LW-1017 (TRPML1 agonist) work?
- Brain-penetrant small-molecule agonist of TRPML1, a lysosomal calcium channel gating autophagy-lysosomal clearance, the proteostasis pathway underlying rapamycin, spermidine and fasting; aimed at neurodegenerative protein aggregation.
This is an independent synthesis of published research by a non-clinician. Scores are opinions supported by citations, not prescriptions. See the full disclaimer and methodology for how this score was produced and what it does and doesn't mean.