GANX News & Events

Gain Therapeutics 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 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.

Gain Therapeutics provided evidence, for the first time in Parkinson's Disease, of a reduction in glucocerebrosidase substrate in cerebrospinal fluid. All individuals with elevated levels of glucosylsphingosine in the CSF displayed large decreases back towards levels observed in healthy individuals after 90 days of treatment with GT-02287. Importantly, the change observed in GluSph in CSF was a prespecified exploratory endpoint of this Phase 1b study. 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. Part 1 of the ongoing Phase 1b study has concluded. The Phase 1b study enrolled 21 participants; 19 completed the 90-day dosing period, and 15 chose to continue in the nine-month extension portion of the study that is anticipated to conclude in September 2026.