BETHESDA, Md., Oct. 09, 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 announced that Jonas Hannestad, M.D., Ph.D., Chief Medical Officer of Gain, will give an oral presentation on the Company’s clinical stage lead drug candidate GT-02287 at the Michael J. Fox Foundation’s (MJFF) 16th Annual Parkinson’s Disease Therapeutics Conference on October 17, 2024, in New York City.
The oral presentation titled, “Brain-penetrant small molecule GT-02287 is well-tolerated in healthy volunteers, shows GCase target engagement, and achieves therapeutic exposures shown to modulate PD pathobiology in preclinical models,” will be presented as part of the conference’s first session, “Progress in the Therapeutics Pipeline”.
“We look forward to presenting Phase 1 data for GT-02287, in development for the treatment of Parkinson’s disease, at the upcoming MJFF conference and appreciate the opportunity to contextualize these data with exciting preclinical data that have been generated recently for GT-02287. The Michael J. Fox Foundation shares our mission of bringing potentially life-changing treatments to people living with Parkinson’s disease,” commented Jonas Hannestad.
The Parkinson's Disease Therapeutics Conference (PDTC) is The Michael J. Fox Foundation's annual scientific conference and the only one in the world focused exclusively on Parkinson's disease drug development. Each year, the PD Therapeutics Conference brings together 300 research and clinical development professionals from both academia and industry toshowcase the most exciting and innovative research from MJFF's research portfolio. Novel advances in basic, translational, and clinical research from both academic and industry labs are highlighted through speaker presentations and a poster session. The event is a platform for field leaders to share new and unpublished results and for fostering new relationships and collaborations.
About GT-02287 Gain Therapeutics’ lead drug candidate, GT-02287, is in clinical development for the treatment of Parkinson’s disease (PD) with or without a GBA1 mutation. 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 mutations in the GBA1 gene, the most common genetic abnormality associated with PD, or other age-related stress factors. In preclinical models of PD, GT-02287 restored GCase enzymatic function, reduced aggregated α-synuclein, neuroinflammation and neuronal death, and improved motor function and cognitive performance. Additionally, GT-02287 significantly reduced plasma neurofilament light chain (NfL) levels, an emerging biomarker for neurodegeneration.
Compelling preclinical data in models of both GBA1-PD and idiopathic PD, demonstrating a disease-modifying effect after administration of GT-02287, suggests that GT-02287 may have the potential to slow or stop the progression of Parkinson’s disease.
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 is currently being evaluated for the treatment of Parkinson’s disease with or without a GBA1 mutation. Results from a Phase 1 study of GT-02287 in healthy volunteers demonstrated favorable safety and tolerability, plasma exposure in the projected therapeutic range, CNS exposure, and target engagement and modulation of GCase enzyme.
Gain’s unique approach enables the discovery of novel, allosteric small molecule modulators that can restore or disrupt protein function. Deploying its highly advanced Magellan? 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.
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