Gain Therapeutics Announces Completion of Target Enrollment in Phase 1b Clinical Study Evaluating GT-02287 in People With Parkinson's Disease With or Without a GBA1 Mutation

Core Insights - Gain Therapeutics has successfully enrolled 16 participants in its Phase 1b clinical trial for GT-02287, surpassing the initial goal of 15 participants by Q3 2025 [1][2] - The analysis of 90-day biomarker activity from cerebrospinal fluid (CSF) is now expected to be available in Q4 2025, earlier than the previously planned Q1 2026 [1][2] - The independent data monitoring committee has recommended continuing the study without changes in dose level, with no serious treatment-emergent adverse events reported [1][2] Company Overview - Gain Therapeutics is a clinical-stage biotechnology company focused on developing allosteric small molecule therapies, with GT-02287 as its lead candidate for treating Parkinson's disease [4][9] - The drug is designed to restore the function of the lysosomal enzyme glucocerebrosidase (GCase), which is impaired due to mutations in the GBA1 gene, a common genetic abnormality associated with Parkinson's disease [4][5] Clinical Trial Details - The Phase 1b trial aims to evaluate the safety and tolerability of GT-02287 over a 90-day dosing period in participants with Parkinson's disease, with enrollment ongoing across seven sites in Australia [7][9] - The company plans to extend the screening window for participants until July 31, 2025, and is seeking to extend the dosing period beyond the current 90-day limit [2][3] Preclinical and Clinical Evidence - Preclinical studies have shown that GT-02287 can restore GCase enzymatic function and reduce various pathologies associated with Parkinson's disease, suggesting a potential disease-modifying effect [5][6] - Results from a Phase 1 study in healthy volunteers indicated favorable safety and tolerability, with a greater than 50% increase in GCase activity observed [6][9] Funding and Support - Gain Therapeutics has received funding support from organizations such as The Michael J. Fox Foundation for Parkinson's Research and the Eurostars-2 joint program, which is co-funded by the European Union [8][9]