Gain Therapeutics(US:GANX) Globenewswire·2025-06-30 20:05Core Insights - Gain Therapeutics has successfully enrolled 16 participants in its Phase 1b clinical trial for GT-02287, surpassing the initial goal of 15 participants by Q3 2025 [1][2] - The analysis of 90-day biomarker activity from cerebrospinal fluid (CSF) is now expected to be available in Q4 2025, earlier than the previously planned Q1 2026 [1][2] - The independent data monitoring committee has recommended continuing the study without changes in dose level, with no serious treatment-emergent adverse events reported [1][2] Company Progress - The company plans to extend the screening window for participants until July 31, 2025, in response to clinician and patient interest [2] - Gain Therapeutics is also seeking to extend the dosing period of the Phase 1b trial beyond the current 90-day limit, with long-term toxicology studies nearing completion [2] - The company anticipates that the extension will provide valuable insights for the Phase 2 protocol design planned for early 2026 [2] Drug Candidate Overview - GT-02287 is an allosteric enzyme modulator aimed at treating Parkinson's disease, particularly in patients with or without a GBA1 mutation [4][9] - The drug has shown promise in preclinical models by restoring the function of the lysosomal enzyme glucocerebrosidase (GCase) and addressing various pathological features associated with Parkinson's disease [4][5] - Previous Phase 1 studies indicated favorable safety and tolerability, with a significant increase in GCase activity observed [6][7] Market Context - There are over 1 million Americans diagnosed with Parkinson's disease, highlighting a significant unmet medical need for effective treatments that can modify disease progression rather than just alleviate symptoms [3][5] - Gain Therapeutics has received funding support from notable organizations, including The Michael J. Fox Foundation and the European Union, to advance its research and development efforts [8][9]