Gain Therapeutics to Host Virtual KOL Event on GT-02287 for Parkinson's Disease
BETHESDA, Md., Dec. 18, 2025 (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 it will host a virtual key opinion leader (KOL) event, "Understanding GCase Substrates in Parkinson's Disease: Perspectives on Biomarkers and Disease Modification, Contextualizing emerging biomarker data from the Phase 1b clinical study of GT-02287". Featured Key Opinion Leaders (KOLs) will be Roy Alcalay, M.D., M.Sc., Chief of Movement Disorders Division, Tel Aviv Sourasky Medical Center, Professor of Neurology, Tel Aviv University, Associate Professor of Clinical Neurology, Columbia University, and Peter Lansbury, Ph.D., Professor of Neurology, Harvard. The event will be held on Tuesday, January 6, 2026 at 10:00 a.m. EST. To register, please click here.
The event will also review biomarker results from the Phase 1b clinical study of GT-02287, demonstrating disease modifying potential in Parkinson's disease patients with or without a GBA1 mutation.
A live question and answer session will follow the formal presentations.
About Roy Alcalay, M.D., M.Sc.
Roy Alcalay, M.D., M.Sc. is chief of the Movement Disorders Division at Tel Aviv Sourasky Medical Center and the Norma and Alan Aufzien Professor of Neurology at the Gray Faculty of Medical & Health Sciences, Tel Aviv University, Israel. He moved from Columbia University where he continues to hold a part-time position focusing on research and education. He obtained his medical degree from Tel Aviv University, his neurology training from the Harvard University residency program at Massachusetts General Hospital and Brigham and Women's Hospital, and his training in movement disorders at Columbia University. He earned a master's degree in biostatistics (patient-oriented research track) from Columbia University. His research focuses on biomarkers and genetics in Parkinson's disease. He is a member of the executive steering committee of The Michael J. Fox Foundation-funded PPMI study and the principal investigator of the Parkinson's Foundation-funded study PD GENEration. He serves on the scientific advisory boards of the Parkinson's Foundation and the Silverstein Foundation for Parkinson's with GBA.
About Peter Lansbury, Ph.D.
Peter Lansbury, Ph.D. is a Professor of Neurology at Harvard University. For the past thirty years, he has studied the biochemical processes underlying Alzheimer's disease, Parkinson's disease, prion disease, and ALS, with the goal of translating this knowledge into novel medicines. Peter and his research group have authored numerous papers in the area of protein aggregation as it relates to neurodegeneration. His laboratory was the first to over express alpha-synuclein and to characterize its structural properties. He was awarded a Presidential Young investigator Award by the National Science Foundation and a Zenith Fellow award from the Alzheimer's Association. Dr. Lansbury also founded and directed several key research initiatives, including the Laboratory for Drug Discovery in Neurodegeneration and the Morris K. Udall NIH Parkinson's Disease Research Center of Excellence at Brigham and Women's Hospital, where he served as director for a decade. Peter was previously Founder and Chief Science Officer of Link Medicine through its sale to AstraZeneca and Chief Science Officer at Lysosomal Therapeutics, Inc. through its sale to Bial.
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 enzyme modulator that restores the function of the lysosomal 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 ER stress, lysosomal and mitochondrial pathology, aggregated α-synuclein, neuroinflammation and neuronal death, as well as plasma neurofilament light chain (NfL) levels, a biomarker of neurodegeneration. In rodent models of both GBA1-PD and idiopathic PD, GT-02287 was shown to rescue deficits in motor function and gait and prevent the development of deficits in complex behaviors such as nesting.
Compelling preclinical data in models of both GBA1-PD and idiopathic PD, demonstrating a disease-modifying effect after administration of GT-02287, suggest that GT-02287 may have the potential to slow or stop the progression of Parkinson's disease.
Results from a Phase 1 study of GT-02287 in healthy volunteers demonstrated favorable safety and tolerability, plasma and CNS exposures in the projected therapeutic range, and target engagement with an increase in glucocerebrosidase (GCase) activity among those receiving GT-02287 at clinically relevant doses.
GT-02287 is currently being evaluated in a Phase 1b clinical trial for the treatment of Parkinson's disease with or without a GBA1 mutation. The primary endpoint of the trial, which enrolled participants across seven sites in Australia, is to evaluate the safety and tolerability of GT-02287 after three months of dosing in people with Parkinson's disease. The recently commenced Phase 1b study extension allows participants to continue to be treated with GT-02287 for up to a total of 12 months.
Gain's lead program in Parkinson's disease has been awarded funding support early in its development 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 in a Phase 1b clinical trial. GT-02287 has further potential in Gaucher's disease, dementia with Lewy bodies, and Alzheimer's disease. Gain has multiple undisclosed preclinical assets targeting lysosomal storage disorders, metabolic diseases, and solid tumors.
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.
Forward-Looking Statements
This release contains "forward-looking statements" made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements are typically preceded by words such as "believes," "expects," "anticipates," "intends," "will," "may," "should," or similar expressions. These forward-looking statements reflect management's current knowledge, assumptions, judgment and expectations regarding future performance or events. Although management believes that the expectations reflected in such statements are reasonable, they give no assurance that such expectations will prove to be correct or that those goals will be achieved, and you should be aware that actual results could differ materially from those contained in the forward-looking statements. Forward-looking statements are subject to a number of risks and uncertainties, including, but not limited to, statements regarding: the development of the Company's current or future product candidates including GT-02287; expectations regarding the completion and timing of results from a Phase 1b clinical study for GT-02287, including any extension studies; expectations regarding the timing of patient enrollment for a Phase 1b clinical study for GT-02287, including any extension studies; the timing of any submissions to the FDA or other regulatory bodies and agencies; and the potential therapeutic and clinical benefits of the Company's product candidates. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the Company's business in general, please refer to the Company's Form 10-K for the year ended December 31, 2024. All forward-looking statements are expressly qualified in their entirety by this cautionary notice. You are cautioned not to place undue reliance on any forward-looking statements, which speak only as of the date of this release. We have no obligation, and expressly disclaim any obligation, to update, revise or correct any of the forward-looking statements, whether because of new information, future events or otherwise.
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Gain Therapeutics, Inc.
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