Gain’s Phase 1b nine-month extension study of GT-02287 enrolled 16 participants, 14 of whom reached 150 days of dosing. MDS-UPDRS scores remained stable across the cohort at Day 150, while participants with elevated baseline CSF glucosylsphingosine saw an average 81% reduction after 90 days and a decrease in DOPA decarboxylase levels.

The company presented additional data at scientific conferences showcasing target engagement, downstream pathway improvements and motor function benefits, supporting disease-modifying potential. Gain’s Magellan™ platform also yielded GT-04686, a novel GCase allosteric modulator now ready for IND-enabling studies in Parkinson’s and other neurological disorders.

For the year ended December 31, 2025, R&D expenses declined by $0.6 million to $10.2 million, while G&A expenses fell by $1.1 million to $8.5 million. Net loss narrowed slightly to $20.2 million, or $0.61 per share, and cash, cash equivalents and marketable securities rose to $20.8 million as of year end.

Gain anticipates FDA clearance of its GT-02287 IND in Q2 2026, paving the way for a Phase 2 trial starting in Q3 2026 with U.S. clinical sites. Top-line results from the Phase 1b study are expected in Q4 2026, and the company will continue patient follow-up and data presentations through the year.