Gain Therapeutics' Late-Breaking Abstract Accepted for Poster at FENS Forum 2024

Gain Therapeutics, Inc. (Nasdaq: GANX), a biotechnology company focused on discovering and developing next-generation allosteric small molecule therapies, has revealed promising preclinical data on its lead drug candidate, GT-02287. The new data, highlighting improvements in cognitive performance in a preclinical model of GBA1 Parkinson’s disease (GBA1-PD), will be presented at the FENS Forum 2024 in Vienna, Austria. This forum is the largest neuroscience congress in Europe, encompassing a wide array of neuroscience research.

GT-02287, an orally administered, brain-penetrant small molecule, is designed to enhance the activity of the enzyme glucocerebrosidase (GCase). This enzyme is often impaired due to mutations in the GBA1 gene, which are the most common genetic mutations associated with Parkinson's disease. By restoring GCase function, GT-02287 has shown potential in reducing neuroinflammation, α-synuclein aggregation, and neuronal death, while simultaneously improving dopamine levels and motor and cognitive functions. Additionally, it has demonstrated efficacy in lowering plasma neurofilament light chain (NfL) levels, a biomarker for neurodegeneration.

The poster presentation, scheduled for June 27, 2024, will be delivered by Dr. Beatriz Calvo-Flores Guzman of Gain Therapeutics. This presentation is part of the late-breaking abstracts session, emphasizing the significance of the findings.

The promising results have garnered support from notable organizations, including The Michael J. Fox Foundation for Parkinson’s Research, The Silverstein Foundation for Parkinson’s with GBA, and the Eurostars-2 program, which receives co-funding from the European Union and Innosuisse – the Swiss Innovation Agency.

Gain Therapeutics leverages advanced technologies like AI-supported structural biology and proprietary algorithms through its Magellan™ drug discovery platform. This platform helps identify novel allosteric binding sites on disease-implicated proteins that traditional methods may not detect. Magellan™ is an evolution of the SEE-Tx® platform, now enhanced with AI and machine learning tools to explore vast chemical spaces and identify potential compounds.

Gain Therapeutics' innovative approach focuses on discovering allosteric small molecule modulators that can restore or disrupt protein functions. This accelerates drug discovery and aims to develop disease-modifying treatments for challenging and untreatable conditions, including neurodegenerative diseases, rare genetic disorders, and cancers.

GT-02287 is currently in Phase 1 clinical trials, and the company is optimistic about its potential therapeutic benefits. However, as with all forward-looking statements, there are inherent risks and uncertainties. These include the impact of global conditions like the post-COVID-19 environment, unexpected safety or efficacy data in clinical trials, regulatory hurdles, and competition.

Gain Therapeutics remains committed to updating stakeholders as required by law but emphasizes the uncertain nature of drug development. The company continues to harness its advanced platforms to push the boundaries of therapeutic discovery and deliver innovative solutions to patients with unmet medical needs.