Gain Therapeutics Begins Phase 1b Trial for GT-02287 in GBA1 and Idiopathic Parkinson’s Disease

Gain Therapeutics, Inc., a clinical-stage biotech firm, has announced that it has received approval to commence a Phase 1b clinical trial in Australia. This trial will focus on evaluating the safety and tolerability of its lead allosteric small molecule, GT-02287, in individuals with Parkinson’s disease (PD). The study will also examine biomarkers over a three-month dosing period. The company's collaboration with local PD advocacy groups is expected to facilitate complete enrollment by spring 2025, with results anticipated mid-year.

GT-02287, currently in clinical development for PD treatment with or without a GBA1 mutation, is Gain Therapeutics’ forefront allosteric small molecule. The main aim of the upcoming Phase 1b trial is to reassess the safety and tolerability of GT-02287 in patients with PD. This trial is a continuation of the successful Phase 1 study involving healthy volunteers concluded in Q3 2024, where GT-02287 exhibited favorable safety, tolerability, and engagement with its GCase target, presenting a significant increase in glucocerebrosidase (GCase) activity.

The initiation of the Phase 1b trial marks a significant milestone for Gain Therapeutics, transitioning from preclinical to clinical development. Gene Mack, CFO and Interim CEO of Gain Therapeutics, expressed optimism about GT-02287's potential in slowing or halting PD progression. The company anticipates advancing GT-02287 to Phase 2 trials in 2025 following the forthcoming trial outcomes.

Dr. Jonas Hannestad, Chief Medical Officer of Gain, noted that the Phase 1b trial will be conducted in seven major Australian cities, all of which have prior experience with Parkinson’s disease trials. This initiative is particularly significant as it involves Australian sites rarely included in PD trials, thus offering new opportunities for local investigators and participants.

The Phase 1b trial will be an open-label study assessing 13.5 mg/kg/day of GT-02287 over three months in patients diagnosed with GBA1-PD or idiopathic Parkinson’s disease. Secondary objectives include evaluating pharmacokinetics, modulation of GCase, levels of GCase substrates, and other plasma and cerebrospinal fluid biomarkers.

GT-02287 functions as an orally administered, brain-penetrant allosteric protein modulator that restores the function of the lysosomal enzyme glucocerebrosidase. This enzyme is often impaired due to GBA1 gene mutations, a common genetic factor in PD, or other age-related stressors. Preclinical models have shown that GT-02287 can restore GCase activity, reduce neuroinflammation, decrease α-synuclein aggregation, and improve both motor function and cognitive performance. The treatment also significantly lowered plasma neurofilament light chain levels, a burgeoning biomarker for neurodegeneration.

GT-02287's promising preclinical data suggest a disease-modifying potential for PD, whether genetically linked to GBA1 mutations or idiopathic. Gain Therapeutics’ leading PD program has garnered financial backing from prominent organizations such as The Michael J. Fox Foundation for Parkinson’s Research and The Silverstein Foundation for Parkinson’s with GBA, alongside support from the Eurostars-2 joint program, co-funded by the European Union Horizon 2020 research initiative and Innosuisse – the Swiss Innovation Agency.

Gain Therapeutics is at the forefront of discovering next-generation allosteric therapies. The company's innovative approach has facilitated the identification of novel small molecule modulators that can restore or inhibit protein function. Utilizing its advanced Magellan™ platform, Gain is fast-tracking drug discovery, aiming to develop breakthrough treatments for challenging disorders, including neurodegenerative diseases and rare genetic conditions.