Gain Therapeutics Presents Additional Preclinical Data and Design of Phase 1b Clinical Study of GT-02287 at AD/PD 2025 and Provides Enrollment Update

Core Viewpoint - Gain Therapeutics, Inc. announced new evidence supporting the disease-modifying potential of its lead drug candidate GT-02287 in preclinical models of GBA1 and idiopathic Parkinson's disease, presented at the AD/PD 2025 conference [2][6][12]. Group 1: Clinical Study Updates - The ongoing Phase 1b study is assessing the safety, tolerability, pharmacokinetics, and pharmacodynamics of GT-02287 in individuals with Parkinson's disease, with biomarker analysis expected in Q2 2025 [2][3]. - Enrollment for the Phase 1b study is robust, with completion anticipated by the end of Q2 2025 [5][14]. - The primary endpoint of the Phase 1b trial is to evaluate the safety and tolerability of GT-02287 after three months of dosing [14]. Group 2: Preclinical Findings - In animal models, GT-02287 demonstrated the ability to rescue motor deficits and prevent the development of complex behavioral deficits, indicating its disease-modifying potential [6][12]. - Treatment with GT-02287 resulted in statistically significant reductions in several biomarkers of disease progression, including aggregated α-synuclein and markers of ER stress and neuroinflammation [6][11]. Group 3: Future Planning - An early biomarker analysis from the Phase 1b study participants will inform Phase 2 planning, expected to commence in the second half of 2025 [3][8]. - The results from the Phase 1b study will guide the design of a subsequent randomized, placebo-controlled, double-blind Phase 2 study to evaluate the efficacy of GT-02287 [8]. Group 4: Company Background - Gain Therapeutics is a clinical-stage biotechnology company focused on developing allosteric therapies, with GT-02287 being evaluated for Parkinson's disease and other related conditions [16][17]. - The company has received funding support from various organizations, including The Michael J. Fox Foundation and the Eurostars-2 joint program [15].