BETHESDA, Md., Oct. 09, 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, “Biomarkers, Clinical Endpoints, and the Path to Disease Modification: Contextualizing the emerging data from GT-02287” featuring Key Opinion Leaders (KOLs), Karl Kieburtz, M.D., M.P.H., Professor of Neurology, University of Rochester and Kenneth Marek, M.D., President and Senior Scientist, Institute for Neurodegenerative Disorders. The event will be held on Tuesday, October 14th, 2025, at 4:00 p.m. EST. To register, please click here.
The event will discuss current thinking on biomarkers, clinical outcome scales, and other endpoints to contextualize data that was recently presented at the International Congress of Parkinson’s Disease and Movement Disorders® by Gain Therapeutics’ Chief Medical Officer Jonas Hannestad, M.D., Ph.D. To date, data from this Phase 1b clinical study in people with Parkinson’s disease (PD) with or without a GBA1 mutation has shown GT-02287 was generally well tolerated, with no treatment-emergent serious adverse events observed, as well as trending improvements observed in MDS-UPDRS (Movement Disorder Society Unified Parkinson’s Disease Rating Scale) scores.
A live question and answer session will follow the formal presentations.
About Karl Kieburtz M.D., M.P.H
Karl Kieburtz M.D., M.P.H. is a Co-Founder of Clintrex Research LLC, a Board member of BlueRidge Life Sciences, and a Professor (part time) of Neurology at the University of Rochester. In his academic career, he was the founding Director of the Center for Health & Technology, the initial Robert J. Joynt Professor in Neurology, and served as the Senior Associate Dean for Clinical Research and Director of the Clinical & Translational Science Institute in Rochester. Dr Kieburtz’s primary research interest is novel therapeutics for neurodegenerative diseases. At the University of Rochester, he led over 2 dozen international clinical trials, fully managed in his organization, including the NINDS sponsored trials of neuroprotective agents for PD (NET-PD), and served as the Chair of the Executive Committee of the Parkinson Study Group. He previously served on, and chaired, the FDA Advisory Committee on Peripheral and Central Nervous System Disorders. He has served as a scientific advisor to the US Government (Veterans Affairs, NIH), and to research Foundations (Michael J Fox, Silverstein). He has published over 450 peer-reviewed publications and has an H index >80. In 2009, he was one of the co-founders of Clintrex Research which has collaborated with over 200 organizations developing products for brain and neurological disorders. Those collaborations have led to the approval of products for Parkinson and Alzheimer disease, migraine and multiple sclerosis. Clintrex was acquired by BlueRidge Life Sciences in 2024. He received his BA in Neuroscience from Amherst College. He completed his MD and MPH degrees at the University of Rochester, as well as his Neurology training and Fellowship in Experimental Therapeutics.
About Kenneth Marek, M.D.
Kenneth Marek, M.D. is President and senior scientist at the Institute for Neurodegenerative Disorders. Dr. Marek's major research interests include identification of biomarkers for early detection, assessment of disease progression and development of new treatments for neurodegenerative disorders including Neuronal synuclein disease (Parkinson disease and Dementia with Lewy Bodies) , Alzheimer disease and related neurodegenerative disorders. His specific interest has been in in vivo neuroreceptor imaging biomarkers. He has authored numerous neurology and neuroscience publications on these topics. Dr. Marek has and continues to be the principal investigator of several ongoing multi-center international studies including the Parkinson Progression Marker Initiative (PPMI) and the Parkinson Associated Risk Syndrome (PARS) study. Dr. Marek serves as a special scientific advisor to The Michael J. Fox Foundation. He has served in leadership roles in several organizations focused on neurodegenerative disorders and has been the recipient of numerous grants to support his work in Parkinson disease, Alzheimer Disease and Huntington disease including the Robert A. Pritzker Prize for Leadership in Parkinson's Research. He also was a co-founder of Molecular NeuroImaging, LLC, and Xing imaging LLC companies providing discovery and clinical neuroimaging research services.
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 a >50% 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 7 sites in Australia, is to evaluate the safety and tolerability of GT-02287 after 3 months of dosing in people with Parkinson’s disease.
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, quality and timing of results from a Phase 1b clinical study for GT-02287; expectations regarding the timing of patient enrollment for a Phase 1b clinical study for GT-02287, including the dose extension study; 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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