FDA IND Clearance for GT-02287
Gain Therapeutics Inc (NASDAQ:GANX) saw its shares rise 21.2% in after‑hours trading on Monday following the U.S. Food and Drug Administration’s clearance of its Investigational New Drug (IND) application for GT-02287, an oral, brain‑penetrant small‑molecule designed to restore lysosomal glucocerebrosidase activity for the treatment of Parkinson’s disease, irrespective of GBA1 mutation status.
The IND authorization permits Gain to commence a Phase 2a clinical trial in the United States, with additional sites in Australia and Europe, scheduled to start in the third quarter of 2026. The Phase 2a study will enroll participants across these regions, building on Phase 1a and Phase 1b studies that were conducted in Australia.
In the Phase 1b extension, all 16 participants remained on the trial at Day 150. Biomarker analysis revealed an average 81% reduction in cerebrospinal fluid glucosylsphingosine after 90 days among participants with elevated baseline levels. Those participants also demonstrated greater improvement in MDS‑UPDRS Part II and Part III scores at Day 150 compared with subjects who had low baseline glucosylsphingosine.
GT‑02287 represents the first allosteric modulator emerging from Gain’s Magellan AI drug‑discovery platform to achieve IND clearance. Parkinson’s disease currently affects more than 1.1 million individuals in the United States, with approximately 90,000 new diagnoses each year, and no approved therapies exist that halt or reverse disease progression.