Gain Therapeutics Initiates Phase 1b Clinical Trial of Lead Candidate GT-02287 in People with GBA1 and Idiopathic Parkinson's Disease

Core Viewpoint - Gain Therapeutics has received approval to initiate a Phase 1b clinical trial for GT-02287 in Australia, aimed at assessing safety and tolerability in patients with Parkinson's disease [1][3] Group 1: Clinical Trial Details - The Phase 1b trial will evaluate the safety and tolerability of GT-02287 at a dosage of 13.5 mg/kg/day over three months [4] - Enrollment is expected to be completed by spring 2025, with data anticipated by mid-2025 [1][4] - The trial will involve seven sites across major metropolitan areas in Australia, focusing on participants with GBA1-PD or idiopathic Parkinson's disease [4] Group 2: Previous Study Results - The Phase 1 study in healthy volunteers, completed in Q3 2024, demonstrated a favorable safety profile and significant target engagement with a greater than 50% increase in glucocerebrosidase (GCase) activity [2][8] - GT-02287 showed plasma and CNS exposures within the projected therapeutic range during the Phase 1 study [2][8] Group 3: Drug Candidate Information - GT-02287 is an orally administered, brain-penetrant small molecule that acts as an allosteric protein modulator, restoring the function of the GCase enzyme [5][8] - Preclinical models indicated that GT-02287 could restore GCase function, reduce neuroinflammation, and improve motor and cognitive performance [5][6] Group 4: 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 [7]