Lysoway Therapeutics Announces Phase I Readiness of Brain-Penetrant TRPML1 Agonist LW-1017 and Nomination of a TMEM175 Development Candidate

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Lysoway Therapeutics, Inc., a biotechnology company advancing structure-guided small-molecule modulators of lysosomal ion channels to treat age-related neurodegeneration, today announced that its lead TRPML1 agonist, LW-1017, is Phase I ready. A first-in-human (FIH) study is planned in Australia, with first patient first dose (FPFD) targeted for May 2026. The Company also announced the formal nomination of a development candidate for its TMEM175 agonist program, reflecting the reproducibility of its discovery platform.

All IND-enabling studies for LW-1017 have been completed with wide safety margins, supporting the initiation of human dosing. The planned Phase I study will assess safety, tolerability, and human pharmacokinetics to inform subsequent global development.

TRPML1 is a lysosomal calcium channel that coordinates autophagy–lysosomal responses to cellular stress by promoting autophagy–lysosomal flux, lysosomal exocytosis, and activation of the transcription factors TFEB and TFE3, thereby enhancing lysosomal biogenesis and cellular clearance. Genetic, cellular, and disease-model evidence implicates impaired TRPML1 signaling in age-associated neurodegenerative processes, positioning TRPML1 as an upstream regulator of neuronal homeostasis.

In preclinical studies, LW-1017 demonstrated robust pharmacodynamic activity consistent with restoration of age-impaired autophagy–lysosomal pathway function. Oral administration achieved sustained central nervous system exposure and modulation of lysosomal and autophagy biomarkers, supporting effective target engagement in aged systems. Therapeutic relevance was demonstrated across two mechanistically related aged models of neurodegeneration. In aged Parkinson’s disease models, LW-1017 reduced pathological alpha-synuclein, preserved nigrostriatal dopaminergic integrity, and improved motor performance. In aged Alzheimer’s disease models, LW-1017 reduced amyloid and phosphorylated tau pathology, preserved neuronal integrity, and improved cognitive performance. Efficacy was observed at low oral doses, consistent with efficient brain penetration.

In parallel, Lysoway has formally nominated a development candidate for its TMEM175 agonist program. TMEM175 is supported by human genetics linking the target to Parkinson’s disease risk and lysosomal dysfunction. Nomination of a TMEM175 development candidate underscores Lysoway’s ability to reproducibly translate genetically validated, technically challenging lysosomal ion-channel targets into development-ready, brain-penetrant small molecules.

For ubiquitously expressed lysosomal ion channels, high brain exposure is essential for both efficacy and safety. TRPML1 and TMEM175 are broadly expressed in peripheral tissues, creating a narrow therapeutic window for compounds lacking sufficient central nervous system penetration. Accordingly, high brain penetration is not an advantage but a necessity. Using its structure-guided approach, Lysoway engineered agonists with brain-to-plasma AUC ratios greater than 1.5 for LW-1017 and greater than 1.0 for TMEM175 agonists in preclinical studies, supporting robust central target engagement while preserving systemic safety margins.

“Age-related decline of the autophagy–lysosomal pathway is a convergent driver of neurodegeneration, leading to toxic protein accumulation, neuroinflammation, and neuronal dysfunction across multiple diseases,” said Yongchang Qiu, PhD, Founder and Chief Executive Officer of Lysoway Therapeutics. “Lysosomal ion channels sit at key regulatory nodes of this system yet have historically been extremely difficult drug targets, particularly for CNS indications. We are proud to have overcome these challenges through a structure-guided approach that enables highly brain-penetrant agonists. LW-1017’s Phase I readiness, together with the formal nomination of a TMEM175 development candidate, demonstrates both the clinical momentum of our lead program and the reproducibility of our platform in drugging lysosomal ion channels for CNS indications.”

About Lysoway Therapeutics

Lysoway Therapeutics is a biotechnology company pioneering therapeutics targeting lysosomal ion channels to counteract age-related neurodegeneration. Leveraging structure-based drug design, high-resolution cryo-EM, and proprietary insights into lysosomal biology, Lysoway is developing highly brain-penetrant small-molecule agonists of TRPML1 and TMEM175 to restore coordinated autophagy–lysosomal function and cellular homeostasis. For more information, visit www.lysoway.com.

Forward-Looking Statements

This press release contains forward-looking statements related to planned development activities and timelines, which are subject to risks and uncertainties.