Gain Therapeutics to Present GT-02287 Phase 1b Data, Targets Q3 Phase 2 Start
Gain Therapeutics will present interim Phase 1b GT-02287 data showing CNS target engagement, glucosylsphingosine reduction and early clinical improvements at the AD/PD 2026 conference on March 18. The company has responded to FDA’s request for additional preclinical data and remains on track to initiate a placebo-controlled Phase 2 trial in 3Q 2026.
1. AD/PD 2026 Presentations
Gain Therapeutics will present interim results from its Phase 1b trial of GT-02287 at the AD/PD 2026 conference in Copenhagen on March 18. Chief Medical Officer Jonas Hannestad will deliver an oral presentation on CNS target engagement and clinical endpoints, while Head of Research Ana Maria Garcia-Collazo will present a poster on a novel allosteric GCase modulator.
2. Phase 2 IND Regulatory Update
In March 2026, the company submitted a response to the FDA’s request for additional preclinical data to support its IND application. Gain expects a decision in the coming weeks and remains on track to initiate a placebo-controlled, dose-range-finding Phase 2 trial in the United States during 3Q 2026.
3. Phase 1b Interim Analysis Findings
Interim data from the Phase 1b study demonstrate target engagement in the central nervous system, reduction of glucosylsphingosine to baseline levels and early signs of clinical improvement in MDS-UPDRS scores. The study enrolled participants across seven sites in Australia and includes a nine-month extension phase.
4. Future Outlook and Extension
The ongoing nine-month extension is slated to complete in September 2026, allowing continued assessment of safety, tolerability and durability of GT-02287’s effects. These developments set the stage for the Phase 2 trial designed to confirm the promising therapeutic profile observed to date.