Gain Therapeutics Presents Additional Clinical and Biomarker Data from Phase 1b Clinical Study of GT-02287 and Preclinical Data from Novel Chemical Series at AD/PD 2026

Core Insights - Gain Therapeutics, Inc. presented new clinical and biomarker data for GT-02287 at the AD/PD 2026 conference, supporting its potential as a disease-modifying treatment for Parkinson's disease [1][2][6] Group 1: Clinical Study Results - The Phase 1b study of GT-02287 showed a reduction in cerebrospinal fluid (CSF) glucosylsphingosine (GluSph) levels after 90 days of treatment, indicating a positive response in participants with elevated baseline GluSph [3][4] - MDS-UPDRS scores remained stable over 150 days of dosing, with participants having high baseline GluSph showing a 6.7-point improvement compared to those with low baseline levels [4][6] - The study's Data Monitoring Committee recommended continuing the trial without changes, reflecting the treatment's safety and tolerability [2] Group 2: Biomarker Analysis - Higher levels of DOPA decarboxylase (DDC) were observed at baseline, which decreased after 90 days of GT-02287 treatment, suggesting a positive impact on dopaminergic neuron function [5][6] - The correlation between decreased GluSph, reduced DDC levels, and improved MDS-UPDRS scores supports the drug's mechanism of action targeting the underlying biology of Parkinson's disease [6] Group 3: Future Development - Gain Therapeutics is advancing a novel chemical series of allosteric glucocerebrosidase (GCase) modulators, led by GT-04686, which is ready for IND-enabling studies for Parkinson's disease and other neurological disorders [1][7][8] - The company aims to shift the treatment paradigm from symptomatic relief to disease modification, with ongoing studies expected to yield further data [6][14]