Lysoway Therapeutics announced the publication of preclinical data on LW-1017 in Scientific Reports.
According to the company, LW-1017 is a novel, potent and highly brain-penetrant small-molecule agonist of TRPML1 that activated the channel with nanomolar potency and induced nuclear translocation of TFEB, TFE3 and MiTF and the CLEAR gene network, effects that were abolished in TRPML1-deficient cells.
The company claims oral dosing produced robust brain exposure and sustained modulation of autophagy–lysosomal biomarkers across tested doses, and that LW-1017 reduced pathological alpha-synuclein, preserved dopaminergic neurons and improved motor function in aged Parkinson’s models while reducing amyloid and tau pathology, decreasing neuroinflammation and improving cognition in aged Alzheimer’s models.
LW-1017 is currently being evaluated in a Phase I single ascending dose and multiple ascending dose trial in healthy volunteers.
The research received partial support from the Michael J. Fox Foundation and The Silverstein Foundation for Parkinson’s with GBA.
