Data demonstrates improvement in cognitive function in GBA1 Parkinson’s disease model in addition to reversal of motor function deficits
BETHESDA, Md., June 27, 2024 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, today announces the presentation of a poster at the Federation of European Neuroscience Societies (FENS) Forum 2024. The poster describes improvement in cognitive performance and activities of daily living associated with administration of GT-02287 the Company’s lead novel allosteric therapeutic candidate in a mouse model of GBA1-Parkinson's disease (GBA1-PD). FENS Forum 2024 is being held June 25–29, 2024 in Vienna, Austria.
“These data further confirm our conviction that GT-02287 can slow or stop progression of Parkinson’s disease and given the cognitive decline observed in GBA1 Parkinson’s patients, we hope that we can one day deliver this drug to those that need it and help them improve their everyday life. As we are completing our Phase 1 clinical study, we look forward to evaluating this drug in patients suffering from this chronic and debilitating disease with no adequate treatment options,” commented Dr. Joanne Taylor, Senior Vice President of Research at Gain Therapeutics. “FENS Forum 2024 is an important opportunity for us to continue discussing our differentiated approach to addressing GBA1 Parkinson’s disease and further discourse and collaboration with the neuroscience community.”
The poster, titled, “GT-02287, a Clinical-Stage GCase Enhancer, Improves Activities of Daily Living and Cognitive Performance in a Preclinical Model of GBA1 Parkinson’s disease,” which will be presented on-site on June 27th by Dr. Beatriz Calvo-Flores Guzman and available online the same day demonstrates how GT-02287 improves nest-building performance and motor performance in a mouse model of GBA1-PD. Nest building in rodents is a natural and spontaneous behavior that closely resembles the tasks of daily living and cognitive performance in humans. GT-02287 was administrated orally once daily from day eight in a mouse model of GBA1-PD, which utilizes a combination of a-synuclein pre-formed fibril (PFF) administration and glucocerebrosidase (GCase) activity lowering with conduritol beta epoxide (CBE). Nest-building performance was assessed by scoring the quality of the nest and motor function was tested in the wire hang test at day 28. Plasma NfL, and brain aggregated alpha-synuclein, Iba-1, and GFAP levels were also measured. The results suggest that GT-02287 can rescue deficits in complex behaviors in which cognitive function is involved in a GBA1 Parkinson’s disease model in addition to improving motor function deficits in this model as reported previously and this is reflected in appropriate biomarker changes.
A PDF of the poster presented at the FENS Forum 2024 conference is available on the Science & Technology section of the Company’s website at https://www.gaintherapeutics.com/science-and-technology/posters.
About FENS Forum
Founded in 1998 at the first Forum of European Neuroscience, the Federation of European Neuroscience Societies (FENS) is the main organization for neuroscience in Europe. FENS currently represents 40 national and single-discipline neuroscience societies across 31 European countries and more than 22,000 member scientists.
FENS promotes neuroscience research to policymakers, funding bodies and the general public, both regionally and internationally. FENS promotes excellence in neuroscience research and facilitates exchanges and networking between neuroscientists within the European Research Area and beyond.
About GT-02287
Gain Therapeutics’ lead drug candidate, GT-02287, is in clinical development for the treatment of GBA1 Parkinson’s disease (GBA1-PD). The orally administered, brain-penetrant small molecule is an allosteric protein modulator that restores the function of the lysosomal protein enzyme glucocerebrosidase (GCase) which becomes misfolded and impaired due to a GBA1 gene mutation, the most common genetic abnormality associated with PD. In preclinical models of PD, GT-02287 restored GCase enzymatic function, reduced aggregated a-synuclein, neuroinflammation and neuronal death, increased dopamine levels and improved motor function and cognitive performance. Additionally, GT-02287 significantly reduced plasma neurofilament light chain (NfL) levels, an emerging biomarker for neurodegeneration.
Gain’s lead program in Parkinson’s disease has been awarded funding support from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) and The Silverstein Foundation for Parkinson’s with GBA, as well as from the Eurostars-2 joint program with co-funding from the European Union Horizon 2020 research and Innosuisse – Swiss Innovation Agency.
About Gain Therapeutics, Inc.
Gain Therapeutics, Inc. is a clinical-stage biotechnology company leading the discovery and development of next generation allosteric therapies. Gain’s lead drug candidate GT-02287 for the treatment of GBA1 Parkinson’s disease, is currently being evaluated in a Phase 1 clinical trial.
Leveraging AI-supported structural biology, proprietary algorithms, and supercomputer-powered physics-based models, the company’s Magellan™ drug discovery platform can identify novel allosteric binding sites on disease-implicated proteins, pinpointing pockets that cannot be found or drugged with current technologies. Its AI and machine-learning tools and virtual screening capabilities leverage the emerging on-demand compound libraries covering vast chemical spaces of trillions of compounds to identify and select suitable small molecule hits for experimental validation.
Gain’s unique approach enables the discovery of novel, allosteric small molecule modulators that can restore or disrupt protein function. Deploying its highly advanced platform, Gain is accelerating drug discovery and unlocking novel disease-modifying treatments for untreatable or difficult-to-treat disorders including neurodegenerative diseases, rare genetic disorders and oncology.
Forward Looking Statements
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