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Gain Therapeutics, Inc. (NASDAQ: GANX) is a pioneering biotechnology company committed to the discovery and development of novel small molecule therapeutics. Specializing in addressing rare genetic diseases, Gain Therapeutics focuses on orphan diseases caused by inborn errors of metabolism. With a strong emphasis on lysosomal storage disorders, central nervous system disorders, metabolic disorders, and oncology, the company's innovative approach aims to provide solutions where there is a high unmet medical need.
At the forefront of Gain Therapeutics' pipeline is GT-02287, a promising clinical-stage product candidate targeting GBA1 Parkinson's disease. Currently in Phase 1 clinical trials, GT-02287 is designed to be a disease-modifying treatment. It functions as an allosteric protein modulator to restore the activity of the lysosomal enzyme glucocerebrosidase (GCase), tackling the underlying cause of disease rather than just alleviating symptoms.
The company's proprietary Magellan™ platform leverages AI-supported structural biology and advanced algorithms to discover novel allosteric binding sites on disease-implicated proteins. This innovative computational technology identifies potential small molecules that can bind to these sites, offering new avenues for therapeutic development. Magellan builds upon Gain’s original SEE-Tx® platform, enhancing it with AI and virtual screening capabilities to explore vast chemical spaces.
Recent achievements highlight Gain Therapeutics' impact on the biotech landscape. Data presented at the 20th WORLDSymposium™ showcased the progressive reversal of motor deficits in preclinical models, alongside a significant reduction of neurodegeneration biomarkers. This underscores the potential of GT-02287 as a best-in-class treatment for neurodegenerative diseases.
Gain Therapeutics collaborates with notable organizations including The Michael J. Fox Foundation for Parkinson’s Research and The Silverstein Foundation for Parkinson’s with GBA. These partnerships provide essential support and validation for the company's mission to transform therapeutic landscapes with innovative treatments.
For more information, visit GainTherapeutics.com and follow their updates on LinkedIn.
Gain Therapeutics (Nasdaq: GANX), a clinical-stage biotechnology company, has announced its participation in Biotech Showcase 2025. Gene Mack, the Company's Chief Financial Officer and Interim Chief Executive Officer, will deliver a corporate presentation featuring their lead drug candidate GT-02287 and the Magellan Drug Discovery Platform.
The presentation is scheduled for Tuesday, January 14, at 9:30am PT in the Yosemite A track at the Hilton San Francisco Union Square. The Biotech Showcase, jointly produced by Demy-Colton and EBD Group, is an investor-focused conference that facilitates networking between executives and investors to promote therapeutic development and investment opportunities.
Gain Therapeutics (GANX) reported Q3 2024 financial results and provided updates on GT-02287, its lead candidate for Parkinson's disease treatment. The Phase 1 study showed a 53% increase in GCase activity with favorable safety in healthy volunteers. The company plans to initiate a Phase 1b trial in Parkinson's patients by end of 2024. Financial highlights include R&D expenses of $2.6M (up $0.3M YoY), G&A expenses of $1.8M (down $0.7M YoY), and cash position of $12M as of September 30, 2024. Net loss was $0.17 per share compared to $0.37 in Q3 2023.
Gain Therapeutics (NASDAQ: GANX) presented data at the 36th EORTC-NCI-AACR Symposium showcasing their allosteric inhibitor GT-03842, identified through their Magellan Drug Discovery Platform. The compound effectively targets and inhibits DDR2 phosphorylation, a protein associated with various cancer types and tumor progression. GT-03842 demonstrated dose-dependent inhibition in both HEK293 cells and a metastatic breast cancer model. The compound's unique mechanism, targeting outside the kinase domain, suggests potential advantages over traditional kinase inhibitors, including enhanced selectivity and reduced resistance.
Gain Therapeutics (Nasdaq: GANX) announced a poster presentation at the 36th EORTC-NCI-AACR Symposium on Molecular Targets and Cancer Therapeutics in Barcelona, Spain, from October 23-25, 2024. The poster will showcase the use of Gain's Magellan platform to discover novel allosteric discoidin domain receptor 2 (DDR2) inhibitors.
DDR2 is implicated in various cancer types and plays a important role in tumor progression. Allosteric small molecule inhibitors have the potential to overcome resistance and offer improved selectivity and safety compared to traditional kinase inhibitors. The presentation, titled 'Identification of allosteric inhibitors targeting Discoidin Domain Receptor 2 (DDR2)', will be given by Sara Cano-Crespo, Ph.D., from Gain Therapeutics during the Molecular Targeted Agents poster session on October 23, 2024, at 11am CEST.
Gain Therapeutics, Inc. (Nasdaq: GANX) has announced that its Chief Medical Officer, Jonas Hannestad, M.D., Ph.D., will present at the Michael J. Fox Foundation's 16th Annual Parkinson's Disease Therapeutics Conference on October 17, 2024, in New York City. The presentation will focus on the company's clinical stage lead drug candidate GT-02287, which is being developed for the treatment of Parkinson's disease.
Dr. Hannestad's 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 part of the conference's first session, 'Progress in the Therapeutics Pipeline.' The presentation will include Phase 1 data for GT-02287 and recent preclinical findings.
The Parkinson's Disease Therapeutics Conference is the only global event focused exclusively on Parkinson's disease drug development, bringing together 300 research and clinical development professionals from academia and industry to showcase innovative research from MJFF's portfolio.
Gain Therapeutics presented new preclinical data for GT-02287 at the Society for Neuroscience 2024 conference. The data demonstrates disease-modifying activity in both GBA-1 and idiopathic Parkinson's disease models. Key findings include:
1. Persistent rescue of motor and cognitive function after GT-02287 discontinuation
2. Improved mitochondrial function and neuroprotection in GBA1-Parkinson's disease models
3. Reduction in Tau accumulation in GBA-1 mutation and wild type cell lines
The results suggest GT-02287's potential as a disease-modifying therapy for Parkinson's disease and possibly Alzheimer's disease and other tauopathies. The compound showed positive effects on mitochondrial and lysosomal health, addressing the disease cascade resulting from GCase dysfunction.
Gain Therapeutics, a clinical-stage biotechnology company, announced participation in two upcoming investor conferences. Gene Mack, interim CEO and CFO, will represent the company at the 3rd Annual ROTH Healthcare Opportunities Conference on October 9, engaging in one-on-one meetings and a panel on 'Emerging Frontiers in Neuroscience'. He will also attend the 2024 Maxim Healthcare Virtual Summit on October 15, participating in a fireside chat with Jason McCarthy, Ph.D., from Maxim Group. Interested attendees can sign up on M-Vest for updates.
Gain Therapeutics presented Phase 1 data for GT-02287, its lead drug candidate for Parkinson's disease, at the International Congress of Parkinson's Disease and Movement Disorders. The study demonstrated that GT-02287 was safe and well-tolerated in healthy volunteers, with no serious adverse events. Notably, the drug increased GCase activity by approximately 30% in subjects who received GT-02287, indicating target engagement. The drug also showed CNS exposure, with measurable levels in cerebrospinal fluid.
The company plans to initiate a trial in Parkinson's disease patients by the end of Q4 2024, aiming to demonstrate safety, tolerability, and obtain proof of mechanism based on relevant biomarkers. Gain Therapeutics remains optimistic about GT-02287's potential as a disease-modifying treatment for Parkinson's disease.
Gain Therapeutics (Nasdaq: GANX) announced a virtual webinar on September 30, 2024, at 8:30 am ET to discuss results from the Phase 1 study of GT-02287, their novel GCase-targeting small molecule therapy for Parkinson's disease. The company will present positive results recently shared at the International Congress of Parkinson's Disease and Movement Disorders® (MDS) and outline the design of an upcoming Phase 1b trial in Parkinson's disease patients.
The webinar will include a live Q&A session following formal presentations. Interested parties can register for the event or join via the company's website. A replay will be available in the News and Events section of the Gain Therapeutics website after the event.
Gain Therapeutics (Nasdaq: GANX) announced three presentations at Neuroscience 2024, showcasing preclinical data for GT-02287, their lead drug candidate for Parkinson's disease. The event will be held in Chicago from October 5-9, 2024.
Two late-breaking posters will be presented:
- GT-02287's prevention of Tau accumulation in a cellular model
- GT-02287's disease-modifying capacity in both GBA1 and idiopathic Parkinson's disease models
A third presentation will focus on GT-02287's improvement of mitochondrial function and neuroprotective effects in GBA1-Parkinson's disease models. These studies were conducted in collaboration with researchers from Switzerland, supported by an Innosuisse-Swiss Innovation Agency grant.
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