GT-02287

June 29, 2026: Gain Therapeutics, Inc. (NASDAQ: GANX) announced the FDA has authorized its IND for GT-02287, enabling initiation of Phase 2 clinical development in Parkinson’s disease (with or without a GBA1 mutation) in the U.S. The company expects to start the Phase 2a trial in 3Q26, with sites planned across the U.S., Australia (where Phase 1a/1b were conducted), and select European centers.

The update includes Phase 1b extension findings through five months of treatment: all 16 participants who entered the ongoing extension remained on study at Day 150, and an independent Data Monitoring Committee recommended continuing without modification. Biomarker analyses showed sustained target engagement, including an average 81% reduction in CSF glucosylsphingosine (GluSph) after 90 days in participants with elevated baseline levels, along with reductions in DOPA decarboxylase (DDC). Clinically, participants with elevated baseline CSF GluSph showed greater improvement in MDS-UPDRS Part II and III at Day 150 versus those with low baseline GluSph, while overall scores were stable; participants also reported improvements in motor and non-motor symptoms (e.g., smell, gait, sleep).