Gain Therapeutics to Host Virtual KOL Event on GT-02287 for Parkinson’s Disease

Core Insights - Gain Therapeutics, Inc. will host a virtual key opinion leader event on October 14, 2025, focusing on biomarkers and clinical endpoints related to their drug candidate GT-02287 for Parkinson's disease [1][2] Company Overview - Gain Therapeutics is a clinical-stage biotechnology company specializing in the discovery and development of allosteric small-molecule therapies [12] - The lead drug candidate, GT-02287, is in clinical development for treating Parkinson's disease with or without a GBA1 mutation [6][12] Event Details - The event will feature KOLs Karl Kieburtz and Kenneth Marek, discussing recent data from a Phase 1b clinical study of GT-02287 [1][2] - A live Q&A session will follow the formal presentations [3] Clinical Study Insights - Data from the Phase 1b study indicates that GT-02287 was generally well tolerated, with no serious adverse events reported and improvements in MDS-UPDRS scores [2][10] - The primary endpoint of the ongoing Phase 1b trial is to evaluate the safety and tolerability of GT-02287 after three months of dosing [10] Drug Mechanism and Preclinical Data - GT-02287 is an allosteric enzyme modulator that restores the function of the lysosomal enzyme glucocerebrosidase (GCase), which is impaired due to GBA1 mutations [6][7] - Preclinical models show that GT-02287 can restore GCase function and has a disease-modifying effect, potentially slowing or stopping the progression of Parkinson's disease [8][9] Funding and Support - Gain Therapeutics has received funding support from The Michael J. Fox Foundation and other organizations for the development of GT-02287 [11]