Gain Therapeutics Announces Positive Results in Key Exploratory Endpoint from its Phase 1b Clinical Study of GT-02287 in People with Parkinson’s Disease

Core Insights - Gain Therapeutics, Inc. has reported a significant reduction in glucosylsphingosine (GluSph) levels in cerebrospinal fluid (CSF) of Parkinson's disease (PD) patients after treatment with their drug candidate GT-02287, indicating increased GCase activity in the brain, which may influence PD progression [1][2][3] Group 1: Study Results - The Phase 1b study of GT-02287 showed that all participants with elevated GluSph levels experienced substantial decreases towards levels seen in healthy individuals after 90 days of treatment [2] - The study enrolled 21 participants, with 19 completing the 90-day dosing period, and 15 (79%) opting to continue into a nine-month extension [4] - The data monitoring committee has recommended the continuation of the Phase 1b study with no changes, confirming the treatment's tolerability [5] Group 2: Future Plans and Events - A KOL event is scheduled for January 6, 2026, to discuss the results of the study and emerging biomarker data [6] - The company plans to present longer follow-up data at the AD/PD™ conference in March 2026, focusing on the effects of GT-02287 on MDS-UPDRS scores [3] Group 3: Drug Candidate Overview - GT-02287 is an orally administered, brain-penetrant small molecule that acts as an allosteric enzyme modulator, aimed at restoring the function of the lysosomal enzyme glucocerebrosidase (GCase) [7][8] - Preclinical studies have shown that GT-02287 can restore GCase function, reduce ER stress, and improve motor function in models of both GBA1-PD and idiopathic PD [9][10] 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 [12]