Core Viewpoint - Gain Therapeutics, Inc. has initiated dosing in a Phase 1b clinical trial for its lead drug candidate GT-02287, aimed at treating Parkinson's disease, marking a significant step in the development of a disease-modifying therapy for this condition [1][2]. Company Overview - Gain Therapeutics is a clinical-stage biotechnology company focused on discovering and developing next-generation allosteric small molecule therapies [8]. - The company’s lead drug candidate, GT-02287, is designed to treat Parkinson's disease with or without a GBA1 mutation and is an orally administered, brain-penetrant small molecule [5][8]. Clinical Trial Details - The Phase 1b trial is an open-label, multi-center study evaluating the safety and tolerability of GT-02287 in participants with GBA1-PD and idiopathic PD, with secondary endpoints including pharmacokinetics and biomarker analysis [3]. - The trial will enroll up to 20 participants who will receive GT-02287 daily for three months, with interim data expected by the end of Q2 2025 [3][4]. Previous Study Results - A prior Phase 1 study in healthy volunteers demonstrated a favorable safety and tolerability profile for GT-02287, with significant target engagement indicated by over 50% increase in glucocerebrosidase (GCase) activity [4][8]. - Preclinical data showed that GT-02287 restored GCase function, reduced neuroinflammation, and improved motor and cognitive functions in models of Parkinson's disease [5][6]. Funding and Support - Gain Therapeutics has received funding support from The Michael J. Fox Foundation for Parkinson's Research, The Silverstein Foundation, and the Eurostars-2 joint program, co-funded by the European Union Horizon 2020 research and Innosuisse [7].
Gain Therapeutics Doses First Participant in Phase 1b Clinical Trial of GT-02287 in Parkinson's Disease