Gain Therapeutics Reports Early Parkinson's Trial Results, Drug Cuts Disease Marker by 81% in Early Gain Therapeutics Study

Gain Therapeutics, Inc. (NASDAQ:GANX) ("Gain", or the "Company"), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, today provided additional biomarker and clinical data from the Phase 1b clinical study of GT-02287 in people with Parkinson's disease (PD) with or without a GBA1 mutation that further supports the disease-modifying potential of GT-02287.

In participants with elevated baseline levels of glucosylsphingosine (GluSph) in cerebrospinal fluid (CSF) at baseline, GluSph decreased by an average of 81% after 90 days of treatment with GT-02287. Elevated GluSph, a hallmark of GCase dysfunction, has been shown to increase the aggregation of alpha synuclein and to impair mitochondrial function and other intracellular processes in neurons. The reduction in GluSph in CSF, a first-ever observation following the administration of a GCase modulator to PD patients, suggests increased GCase activity in the brain, which is expected to impact the progression of Parkinson's disease (PD).

As of November 30, 2025, 19 patients completed Part 1 of the study, 90 days of dosing. Two of these patients resulted negative in the alpha synuclein seed amplification assay, calling into question their PD diagnosis and so were excluded from the Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) analysis. Two additional patients were also excluded from the analysis because MDS-UPDRS Part III scores were not collected in the ‘off' state at all time points. The remaining 15 patients experienced an average improvement of 2.20 points in the sum of MDS-UPDRS Part II and Part III scores (0.6 in Part II and 1.6 in Part III).