Gain Therapeutics, Inc.

We are a biotechnology company developing new small molecule drugs to treat diseases in several areas, including central nervous system (CNS) disorders, lysosomal storage disorders (LSDs), metabolic disorders, and other diseases like oncology that can be targeted through protein degradation. Our main product candidate, GT-02287, is for treating Parkinson's disease and is currently in a Phase 1b study involving people with Parkinson's disease, with or without a GBA1 mutation. We have compiled extensive preclinical data demonstrating how GT-02287 works and its safety. In preclinical models of GBA1 and idiopathic Parkinson's disease, GT-02287 has been shown to restore glucocerebrosidase (GCase) function in the lysosome, improve mitochondrial health, reduce toxic lipid substrates and toxic forms of alpha-synuclein, reduce neuroinflammation, improve survival of dopaminergic neurons, increase dopamine levels, completely restore locomotor function, improve cognition, and reduce the plasma-based neurodegeneration marker, neurofilament light chain (NfL), back to the level of healthy animals. In September 2024, we announced results from a first-in-human Phase 1 clinical trial in Australia. This trial assessed the safety, tolerability, pharmacokinetics, and food effect of GT-02287 in 72 healthy volunteers. The study included a single ascending dose part with 40 participants receiving one dose at different levels, and a multiple ascending dose part with 32 participants receiving one daily dose for 14 days at different levels. Unblinded data confirmed that single and multiple doses of GT-02287 were safe and generally well tolerated up to and including the highest planned dose levels across all age groups (approximately 15% of which were over the age of 50 years). No serious adverse events or Grade 3 (severe) adverse events were observed, and no other safety signals were detected. The pharmacokinetic profile of GT-02287 was linear across the tested dose ranges, and plasma exposures at daily doses of 7.7 mg/kg and above were within the projected therapeutic range.