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Gain Therapeutics Announces Positive Topline Results from the Phase 1 Clinical Trial of GT-02287, a Novel GCase-Targeting Small Molecule Therapy for Parkinson’s Disease
Gain Therapeutics (Nasdaq: GANX) has announced positive topline results from its Phase 1 clinical trial of GT-02287, a novel GCase-targeting small molecule therapy for Parkinson's disease.
The study, which enrolled 72 healthy volunteers, demonstrated that GT-02287 was safe and well-tolerated at all dose levels, with no discontinuations or serious adverse events reported. Importantly, the drug showed presence in the cerebrospinal fluid and achieved peripheral target engagement.
Based on these promising results, Gain plans to initiate a Phase 1b trial in Parkinson's disease patients by Q4 2024, with data expected by mid-2025. The company aims to demonstrate safety, tolerability, and proof of mechanism through relevant biomarkers in this upcoming trial.
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