Gain Therapeutics Begins Its GT-02287 Phase 1b Extension Study in People with Parkinson's Disease

Core Insights - Gain Therapeutics, Inc. has announced the extension of its Phase 1b study for GT-02287, allowing participants to continue treatment for an additional nine months after the initial 90-day dosing schedule [1][4] - The Phase 1b study has enrolled 21 participants, with the last participant expected to complete dosing in December 2025 [2][8] - Early data from the Phase 1b study will be presented at the International Congress of Parkinson's Disease and Movement Disorders in October 2025 [2][4] Study Details - The Phase 1b extension study will enable participants to continue dosing with GT-02287, with more than half of the participants agreeing to continue treatment [1][4] - The primary endpoint of the Phase 1b trial is to evaluate the safety and tolerability of GT-02287 after three months of dosing [8] - Additional analysis from participants enrolled as of June 30, 2025, will include functional changes and biomarker data, expected in Q4 2025 [3] Drug Candidate Information - GT-02287 is an orally administered, brain-penetrant small molecule designed to treat Parkinson's disease, targeting the lysosomal enzyme glucocerebrosidase (GCase) [5][10] - Preclinical models have shown that GT-02287 can restore GCase function and reduce various pathologies associated with Parkinson's disease [5][6] - Results from a Phase 1 study in healthy volunteers indicated favorable safety, tolerability, and significant increases in GCase activity [7] 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 [9]