Gain Therapeutics (GANX) investor relations material

Study design and patient population

• Phase 1b open-label study in Parkinson's disease enrolled 21 patients, with 19 completing the first 90 days and 15 continuing into an open-label extension phase.

• All patients received GT-02287 for 90 days, with the option to continue for up to 12 months.

• The trial was conducted across seven sites in Australia, focusing on safety and tolerability after three months of dosing.

• Four patients were excluded from MDS-UPDRS analysis due to baseline or measurement criteria.

Clinical outcomes and UPDRS scores

• Average improvement of 2.2 points in combined MDS-UPDRS Part II and III scores after 90 days among 15 analyzed patients.

• Among 13 of 15 evaluable patients, a greater improvement of around four points was observed.

• Anecdotal improvements were noted in balance, gait, and sense of smell, with stabilization in MDS-UPDRS scores.

• No changes in dopamine administration were required for any subjects during the study.

Biomarker findings and mechanistic insights

• GT-02287 led to an 81% average reduction in CSF glucosylsphingosine (GluSph) after 90 days, indicating increased GCase activity in the brain.

• Reduction in GluSph is a first-ever observation for a GCase modulator in PD patients, suggesting potential to impact disease progression.

• Decrease in CSF glucosylsphingosine observed in patients with elevated baseline levels after 90 days of treatment.

• Reduction in glucosylsphingosine in CSF correlates with improvements in UPDRS scores.

• Glucosylsphingosine is a more reliable biomarker for GCase pathway engagement than glucosylceramide.