GANX News & Events

The Company submitted an IND to conduct a placebo-controlled, dose-range-finding Phase 2 clinical trial of GT-02287 in the United States. In March 2026, the Company submitted a response to the FDA's request for additional preclinical data. The Company has been working closely with the FDA and expects a response over the next few weeks. The Company expects to begin its Phase 2 clinical trial in 3Q 2026, as previously reported. Gene Mack, President and CEO of Gain Therapeutics, stated, "The data from our Phase 1b study furthers our hypothesis that GT-02287 is among the first disease-modifying therapies promising to shift the treatment paradigm in PD from symptom relief to halting or slowing symptom progression, targeting the causative biology of PD to enable a more durable and predictable treatment effect for those living with PD. As we continue to follow patients in the Phase 1b nine-month extension study that is expected to complete in September 2026, we look forward to continuing the productive dialogue with FDA in preparation for our Phase 2 clinical trial of GT-02287, which is designed to confirm the exciting results we have observed to date from our Phase 1b study, and remains on track to begin during 3Q26."

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.