Gain Therapeutics (GANX) investor relations material

Clinical development highlights

• Lead asset GT-02287, developed via an AI-driven platform, targets GCase for Parkinson's disease and has shown safety, tolerability, and target engagement in first-in-human studies in 2024.

• Phase I-B study began in March 2025, enrolling both idiopathic and GBA1-mutant Parkinson's patients, with 19 of 21 completing 90 days and 16 entering open-label extension.

• IND clearance by FDA is expected this quarter, with phase II clinical testing planned for next quarter and final phase I-B results, including open-label extension, anticipated in Q4 2025.

• All intellectual property for GT-02287 and backup compounds is owned, with unique patent protection.

Biomarker and clinical efficacy data

• GT-02287 reduced elevated glucosylsphingosine in CSF to normal levels after 90 days, indicating target engagement and potential disease modification.

• Patients with high baseline glucosylsphingosine showed greater improvement in MDS-UPDRS scores, suggesting a biomarker-driven response.

• Anecdotal improvements included return of taste and smell, improved gait, and concentration, prompting inclusion of secondary endpoints in phase II.

• Stabilization of disease observed, with a three-point benefit on MDS-UPDRS maintained over 150 days, considered clinically meaningful.

Competitive landscape and strategic outlook

• GT-02287 is positioned as a potential first disease-modifying therapy for Parkinson's, with a differentiated mechanism compared to other GCase modulators like VQ-101 and Bial's discontinued compound.

• Phase II will focus on MDS-UPDRS improvement and biomarker validation, with stratification by glucosylsphingosine and GBA1 mutation.

• Current cash position of $16 million is sufficient to fund operations into Q2 next year, with ongoing evaluation of financing and partnership opportunities.

• Strategic partnering may occur after phase II top-line data or earlier, depending on market and diligence processes.