Gain Therapeutics Reports Positive Interim Phase 1b Data for GT-02287 in Parkinson’s Disease

On May 26, 2026, Gain Therapeutics, Inc. reported positive interim clinical and biomarker data from Part 2 of its Phase 1b open-label study evaluating GT-02287 in patients with Parkinson’s disease (PD). The data, presented at the 3rd International GBA1 Meeting, covered a nine-month extension study. Key findings showed that in participants with elevated baseline levels of glucosylsphingosine (GluSph) in cerebrospinal fluid (CSF), treatment with GT-02287 led to an average 81% reduction in GluSph after 90 days. Additionally, levels of DOPA decarboxylase (DDC) decreased, suggesting improved dopaminergic neuron function. Clinical outcomes at Day 150 demonstrated that MDS-UPDRS scores remained stable across the overall population, with participants having high baseline GluSph showing a 4.8-point improvement over the low-baseline group. Patients also reported unsolicited benefits such as improved smell, taste, balance, and sleep. The drug has been well-tolerated, with 16 of 19 original participants continuing into the extension. Gain Therapeutics plans to initiate a Phase 2 clinical trial incorporating objective assessments like UPSIT and wearable sensors to further validate these findings.