Lysoway Therapeutics Announces Phase I Dosing of LW-1017, the First TRPML1 Agonist to Enter Clinical Development

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Lysoway Therapeutics today 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.

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.

“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 plays a central role in regulating the autophagy–lysosomal pathway, a core cellular clearance system responsible for maintaining neuronal homeostasis under conditions of aging and cellular stress. Impairment of autophagy–lysosomal function has been increasingly implicated as an upstream driver of age-related neurodegenerative diseases, including Alzheimer’s disease and Parkinson’s disease.

Despite strong genetic and biological validation, development of TRPML1-targeted therapeutics has historically been challenging due to the difficulty of simultaneously achieving sufficient brain penetration, oral bioavailability, potency, and selectivity in small-molecule modulators. LW-1017 was developed using Lysoway’s proprietary structure-guided discovery platform focused on lysosomal ion channel modulators.

About Lysoway Therapeutics

Lysoway Therapeutics is a biotechnology company pioneering therapeutics targeting lysosomal ion channels for age-related neurodegenerative diseases. Leveraging structure-based drug design, high-resolution cryo-EM, and proprietary insights into lysosomal biology, Lysoway is developing highly brain-penetrant small-molecule modulators of TRPML1 and TMEM175 designed to restore autophagy–lysosomal function and cellular homeostasis.

Forward-Looking Statements

This press release contains forward-looking statements regarding the development, clinical progress, therapeutic potential, and anticipated timing of LW-1017 and other product candidates. Actual results may differ materially from those expressed or implied by these forward-looking statements due to risks and uncertainties inherent in drug development and clinical research.