Core Insights - Gain Therapeutics has received approval to initiate a Phase 1b clinical trial for GT-02287 in Australia, targeting individuals diagnosed with Parkinson's disease [1][9] - The trial aims to assess the safety and tolerability of GT-02287 over three months, with enrollment expected to complete by spring 2025 and data anticipated by mid-2025 [1][2][10] - GT-02287 is designed to treat Parkinson's disease with or without a GBA1 mutation and has shown promising preclinical data indicating a potential to slow or stop disease progression [4][11] Company Developments - The transition from preclinical to clinical stage development was marked by the successful completion of healthy volunteer studies in 2024 [2] - The Phase 1 study demonstrated a favorable safety profile and significant target engagement, with over 50% increase in glucocerebrosidase (GCase) activity [9][13] - Gain Therapeutics is preparing for a Phase 2 trial in 2025, contingent on the results of the Phase 1b trial [2][10] Clinical Trial Details - The Phase 1b trial will be conducted at seven sites across major metropolitan areas in Australia, focusing on patients with GBA1-PD or idiopathic Parkinson's disease [10] - The trial will administer 13.5 mg/kg/day of GT-02287 and will evaluate secondary endpoints including pharmacokinetics and biomarker levels in plasma and cerebrospinal fluid [10][11] - The study is supported by funding from The Michael J. Fox Foundation and other organizations [12]
Gain Therapeutics Initiates Phase 1b Clinical Trial of Lead Candidate GT-02287 in People with GBA1 and Idiopathic Parkinson's Disease