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Gain Therapeutics Announces Positive Results in Key Exploratory Endpoint from its Phase 1b Clinical Study of GT-02287 in People with Parkinson’s Disease
Results showed CNS target engagement with reduction in GCase substrate glucosylsphingosine (GluSph) in cerebrospinal fluid (CSF), a prespecified endpoint 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) KOL event planned for early January to discuss the results; registration information herein BETHESDA, M
About this update from Gain Therapeutics, Inc.
[{"type":"image","alt":"Gain Therapeutics, Inc.","displaySize":"","headline":null,"caption":"Gain Therapeutics, Inc.","className":"","disableSlideshowImg":false,"size":{"original":{"width":150,"height":72,"url":"https://media.zenfs.com/en/globenewswire.com/71d53e58a7e975cfd00a1328d22e0699"},"resized":{"url":"https://s.yimg.com/ny/api/res/1.2/nsPBXqjXYfLs4XgIgz1iUg--/YXBwaWQ9aGlnaGxhbmRlcjt3PTIxMDtoPTEwMTtjZj13ZWJw/https://media.zenfs.com/en/globenewswire.com/71d53e58a7e975cfd00a1328d22e0699","width":150,"height":72}},"lazy":false},{"type":"text","content":"Results showed CNS target engagement with reduction in GCase substrate glucosylsphingosine (GluSph) in cerebrospinal fluid (CSF), a prespecified endpoint","length":153,"tagName":"p"},{"type":"text","content":"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)","length":242,"tagName":"p"},{"type":"text","content":"KOL event planned for early January to discuss the results; registration information herein","length":91,"tagName":"p"},{"type":"text","content":"BETHESDA, Md., Dec. 18, 2025 (GLOBE NEWSWIRE) -- 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 evidence, for the first time in Parkinson’s Disease (PD), of a reduction in glucocerebrosidase (GCase) substrate in cerebrospinal fluid (CSF).","length":413,"tagName":"p"},{"type":"text","content":"Decreased GluSph in CSF is an indication of increased GCase activity in the brain","length":81,"tagName":"p"},{"type":"text","content":"All individuals with elevated levels of glucosylsphingosine (GluSph) 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.","length":508,"tagName":"p"},{"type":"text","content":"Gene Mack, p...