Gain Therapeutics Reports Positive Phase 1 Results for Parkinson’s Drug GT-02287

Gain Therapeutics, Inc., a clinical-stage biotechnology firm, has announced positive outcomes from its Phase 1 study of GT-02287, a drug candidate for Parkinson’s disease (PD) with or without a GBA1 mutation. This study, aimed at evaluating the safety, tolerability, and pharmacokinetics of GT-02287, enrolled 72 healthy volunteers, both male and female, up to 64 years old. The data revealed that single and multiple doses of GT-02287 were safe and generally well tolerated, even at the highest planned dose levels. Approximately 15% of the study participants were over the age of 50 years. Importantly, GT-02287 was present in the cerebrospinal fluid (CSF) and demonstrated peripheral target engagement.

The positive safety and tolerability profile at oral doses resulting in therapeutic plasma levels and central nervous system (CNS) exposure reinforce GT-02287’s potential as a leading treatment for Parkinson’s disease. Jonas Hannestad, M.D., Ph.D., Chief Medical Officer of Gain, indicated that the company plans to initiate a trial in patients with Parkinson’s disease by the fourth quarter of 2024. The upcoming trial aims to demonstrate safety and tolerability in PD patients and to obtain proof of mechanism based on relevant biomarkers. Data from this trial is anticipated by mid-2025.

Gene Mack, Interim CEO and current CFO of Gain, expressed enthusiasm about GT-02287’s promising profile, particularly noting its CNS exposure and target engagement. He emphasized the company's commitment to advancing GT-02287 through clinical milestones with the goal of improving the lives of PD patients.

GT-02287 is an orally administered, brain-penetrant small molecule that acts as an allosteric protein modulator. This drug works by restoring the function of the lysosomal enzyme glucocerebrosidase (GCase), which becomes misfolded and impaired due to GBA1 gene mutations or other age-related factors. Preclinical model studies of Parkinson’s disease showed that GT-02287 successfully restored GCase enzymatic function, reduced aggregated α-synuclein, neuroinflammation, and neuronal death, increased dopamine levels, and improved motor and cognitive functions. Additionally, GT-02287 significantly reduced plasma neurofilament light chain (NfL) levels, a biomarker for neurodegeneration.

Compelling preclinical data, including findings presented at the FENS Forum 2024, indicated improvements in cognitive and motor performance in mouse models of GBA1-PD after administering GT-02287. These results suggest that GT-02287 may have the potential to slow the progression of Parkinson’s disease.

The development of GT-02287 has received financial support from notable organizations including The Michael J. Fox Foundation for Parkinson’s Research (MJFF), The Silverstein Foundation for Parkinson’s with GBA, and the Eurostars-2 joint program, which includes co-funding from the European Union Horizon 2020 research and Innosuisse – Swiss Innovation Agency.

Gain Therapeutics employs advanced technologies in its drug discovery process. Leveraging AI-supported structural biology, proprietary algorithms, and supercomputer-powered physics-based models, the company’s Magellan™ drug discovery platform identifies novel allosteric binding sites on disease-implicated proteins. This platform can pinpoint pockets that are undetectable with current technologies, using AI and machine-learning tools and virtual screening capabilities to explore a vast chemical space of over five trillion compounds. Gain’s approach facilitates the discovery of novel, allosteric small molecule modulators that restore or disrupt protein function, thereby accelerating drug discovery for neurodegenerative diseases, rare genetic disorders, and oncology.

In summary, Gain Therapeutics' efforts to develop GT-02287 signify a significant advancement in Parkinson's disease treatment. The positive results from the Phase 1 study and the planned clinical trials underscore the potential of GT-02287 to be a breakthrough therapy for PD patients, promising improved clinical outcomes.

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