CAMBRIDGE, Mass. — Lysoway Therapeutics has dosed the first participant in a Phase 1 clinical trial of LW-1017, a small-molecule therapy being developed for neurodegenerative diseases including Alzheimer’s disease and Parkinson’s disease.
The Cambridge-based company said the first participant was dosed May 5. Lysoway said LW-1017 is a potent, selective and highly brain-penetrant TRPML1 agonist designed to restore autophagy-lysosomal function in neurodegenerative diseases.
The Phase 1 study is being conducted in Melbourne, Australia, and is designed to evaluate the safety, tolerability and pharmacokinetics of LW-1017 in healthy volunteers. The trial includes single ascending dose and multiple ascending dose cohorts.
“Dosing the first participant in our Phase I study marks a major milestone for Lysoway and for the TRPML1 field,” said Dr. Yongchang Qiu, Founder and Chief Executive Officer of Lysoway Therapeutics. “We believe TRPML1 activation represents a differentiated therapeutic approach that harnesses endogenous lysosomal biology, targets upstream disease biology, and may broadly impact aggregate biology and cellular homeostasis. We are excited to advance the first TRPML1 agonist into the clinic.”
TRPML1 is a lysosomal ion channel that helps regulate the autophagy-lysosomal pathway, a cellular clearance system involved in maintaining neuronal health during aging and cellular stress. Impaired autophagy-lysosomal function has been linked to age-related neurodegenerative diseases, including Alzheimer’s and Parkinson’s.
Lysoway said development of TRPML1-targeted therapies has been difficult because small-molecule modulators must achieve brain penetration, oral bioavailability, potency and selectivity at the same time.
LW-1017 was developed using Lysoway’s proprietary structure-guided discovery platform focused on lysosomal ion channel modulators.
