Gain Therapeutics Highlights Biomarker Evidence Supporting Disease-Modifying Potential of GT-02287
Results to be discussed in KOL event today at 10 a.m. ET
BETHESDA, Md., Jan. 06, 2026 (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 provided additional biomarker and clinical data from the Phase 1b clinical study of GT-02287 in people with Parkinson’s disease (PD) with or without a GBA1 mutation that further supports the disease-modifying potential of GT-02287.
In participants with elevated baseline levels of glucosylsphingosine (GluSph) in cerebrospinal fluid (CSF) at baseline, GluSph decreased by an average of 81% after 90 days of treatment with GT-02287. Elevated GluSph, a hallmark of GCase dysfunction, has been shown to increase the aggregation of alpha synuclein and to impair mitochondrial function and other intracellular processes in neurons. The reduction in GluSph in CSF, a first-ever observation following the administration of a GCase modulator to PD patients, suggests increased GCase activity in the brain, which is expected to impact the progression of Parkinson’s disease (PD).
As of November 30, 2025, 19 patients completed Part 1 of the study, 90 days of dosing. Two of these patients resulted negative in the alpha synuclein seed amplification assay, calling into question their PD diagnosis and so were excluded from the Movement Disorder Society Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) analysis. Two additional patients were also excluded from the analysis because MDS-UPDRS Part III scores were not collected in the ‘off’ state at all time points. The remaining 15 patients experienced an average improvement of 2.20 points in the sum of MDS-UPDRS Part II and Part III scores (0.6 in Part II and 1.6 in Part III).
Gene Mack, President and CEO of Gain Therapeutics commented, “These results mark an important step forward for our program. The observed decrease in GluSph, alongside improvement or stabilization of MDS-UPDRS scores, provides encouraging evidence that our approach is engaging the underlying biology of the disease, suggesting a disease modifying action. We believe these data support continued clinical development and the potential of our therapy to meaningfully impact disease progression.”
“While this study was not designed to assess clinical efficacy, it has been encouraging to see stabilization in MDS-UPDRS scores and to observe anecdotal improvements in specific functional areas such as balance, gait, and sense of smell after 90 days of dosing with GT-02287,” said Michele DeSciscio, lead investigator of the Phase 1b clinical study of GT-02287. “We look forward to seeing longer term outcomes in the dosing extension part of the Phase 1b study of GT-02287.”
Virtual KOL Event on GT-02287
Gain Therapeutics will host a webinar “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”, featuring key opinion leaders Roy Alcalay, M.D., M.Sc., and Peter Lansbury, Ph.D., today at 10:00 a.m. ET to discuss the results announced today and host a question-and-answer session. To register, please click here.
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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