LW-1017 / Lysoway Therapeutics - LARVOL DELTA

A Randomised, Double-Blind, Placebo-Controlled, First-in-Human Study of Orally Administered LW-1017 to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single and Multiple Ascending Doses of LW-1017 in Healthy Volunteers (ANZCTR) - P1 | N=80 | Recruiting | Sponsor: Lysoway Therapeutics Pty Ltd | Not yet recruiting ➔ Recruiting | Initiation date: May 2026

Enrollment open • First-in-human • Trial initiation date • CNS Disorders

Lysoway Therapeutics…announced that the first participant has been successfully dosed on May 5, 2026 in the Company’s Phase I clinical trial of LW-1017, a potent, selective, and highly brain-penetrant small-molecule TRPML1 agonist being developed for neurodegenerative diseases including Alzheimer’s disease and Parkinson’s disease (Businesswire) - "The Phase I study is being conducted in Melbourne, Australia and is designed to evaluate the safety, tolerability, and pharmacokinetics of LW-1017 in healthy volunteers through single ascending dose (SAD) and multiple ascending dose (MAD) cohorts."

Trial status • Alzheimer's Disease • Parkinson's Disease

A Randomised, Double-Blind, Placebo-Controlled, First-in-Human Study of Orally Administered LW-1017 to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses of LW-1017 in Healthy Volunteers (Part A Cohort 4) (ANZCTR) - P1 | N=8 | Not yet recruiting | Sponsor: Lysoway Therapeutics Pty Ltd

First-in-human • New P1 trial • CNS Disorders

THE TRPML1 AGONIST, LW-1017, DOSE-DEPENDENTLY AMELIORATES DISEASE PATHOLOGY AND IMPROVES BEHAVIOR IN MOUSE MODELS OF ALZHEIMER'S DISEASE AND PARKINSON'S DISEASE. (ADPD 2026) - "The TRPML1 agonist, LW-1017, potently and dose-dependently protected hippocampal neurons from Abeta accumulation, pTau spread, microgliosis, astrogliosis and neurodegeneration, leading to an improvement in cognitive performance. Similarly, LW-1017, dose-dependently protected nigrostriatal neurons from synuclein accumulation and propagation, preserved neuronal number, normalized neurotransmitter levels and restored normal motor performance."

Preclinical • Alzheimer's Disease • CNS Disorders • Movement Disorders • Parkinson's Disease

Lysoway Therapeutics Awarded Grant from The Michael J. Fox Foundation to Advance TRPML1 Agonist to Treat Parkinson’s Disease (Businesswire) - "Lysoway Therapeutics, Inc...announced that it has received a research grant from The Michael J. Fox Foundation for Parkinson's Research (MJFF). Support comes from MJFF’s Parkinson’s Disease Therapeutics Pipeline Program, which focuses on candidates with strong potential to slow or halt disease progression or alleviate burdensome symptoms for those living with Parkinson’s disease. Lysoway Therapeutics funding of $2.93 million will support the preclinical and translational development of Lysoway’s novel, highly brain-penetrant small molecule TRPML1 agonist."

Financing • Parkinson's Disease