Gain Therapeutics (GANX) investor relations material

Study overview and objectives

• Phase 1b study of GT-2287 in Parkinson's patients enrolled 21 participants, with a 90-day dosing period and a 9-month extension phase for those opting to continue.

• Main objectives are safety, tolerability, and exploratory biomarker and clinical endpoint evaluation.

• Most patients are early to mid-stage, on stable background therapy, and a subset carry GBA mutations.

• High patient retention into the extension phase suggests favorable tolerability and patient experience.

• Full data from the 90-day phase expected by year-end, with biomarker results in Q4.

Mechanism of action and preclinical insights

• GT-2287 targets glucocerebrosidase (GCase), stabilizing its conformation and trafficking in neurons.

• Preclinical models show GT-2287 improves lysosomal and mitochondrial function, reduces ER stress, and decreases alpha-synuclein aggregation.

• GBA1 mutations, present in 10–15% of Parkinson's patients, are associated with earlier onset and more aggressive disease.

• The drug aims to address underlying pathophysiology, potentially modifying disease progression.

Biomarker landscape and study endpoints

• Biomarkers such as alpha-synuclein, GCase activity, and others are being evaluated for target engagement and disease modification.

• Recent advances allow detection of synuclein pathology pre-symptomatically, aiding early diagnosis and trial design.

• The study leverages both fluid and imaging biomarkers, with ongoing efforts to develop PET tracers for synuclein.

• Genetic and biochemical markers are key to precision-targeted trials and understanding patient subtypes.