Gain Therapeutics Highlights Biomarker Evidence Supporting Disease-Modifying Potential of GT-02287

Core Insights - Gain Therapeutics, Inc. has provided additional biomarker and clinical data from the Phase 1b clinical study of GT-02287, indicating its potential as a disease-modifying therapy for Parkinson's disease [1] Group 1: Clinical Data and Results - In participants with elevated baseline levels of glucosylsphingosine (GluSph) in cerebrospinal fluid (CSF), GluSph decreased by an average of 81% after 90 days of treatment with GT-02287, suggesting increased GCase activity in the brain [2] - Out of 19 patients who completed Part 1 of the study, 15 patients showed an average improvement of 2.20 points in the sum of MDS-UPDRS Part II and Part III scores after treatment [3] - The study observed stabilization in MDS-UPDRS scores and anecdotal improvements in specific functional areas such as balance, gait, and sense of smell after 90 days of dosing with GT-02287 [4] Group 2: Drug Candidate Information - GT-02287 is an orally administered, brain-penetrant small molecule that acts as an allosteric enzyme modulator, restoring the function of the lysosomal enzyme glucocerebrosidase (GCase) [6] - Preclinical models have shown that GT-02287 can restore GCase enzymatic function and reduce various pathologies associated with Parkinson's disease [6][7] - The drug is currently being evaluated in a Phase 1b clinical trial, with the primary endpoint focused on safety and tolerability after three months of dosing [9] Group 3: Future Developments and Support - Gain Therapeutics has received funding support from The Michael J. Fox Foundation for Parkinson's Research and other organizations to advance the development of GT-02287 [10] - The company is hosting a virtual event to discuss the results of the Phase 1b clinical study and engage with key opinion leaders in the field [5]