Gain Therapeutics provides additional data from Phase 1b study of GT-02287

Gain Therapeutics (GANX) 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.