Gain Therapeutics Begins Its GT-02287 Phase 1b Extension Study in People with Parkinson’s Disease
Extension of the Phase 1b study will allow participants to continue dosing with GT-02287 for an additional nine months after completing the first 90-day dosing schedule
The Phase 1b study enrolled 21 subjects and the last participants will complete 90 days of dosing in December 2025
Early results from the Phase 1b study to be presented at the International Congress of Parkinson’s Disease (PD) and Movement Disorders in October
BETHESDA, Md., Sept. 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 the start of the Phase 1b extension study allowing participants to continue treatment for an additional nine months. More than half of the participants have agreed to continue treatment.
The Phase 1b study enrolled 21 participants and is expected to complete in December 2025. Early data from the Phase 1b study will be presented on October 7, 2025, at the International Congress of Parkinson’s Disease and Movement Disorders® in Honolulu, HI.
Additional 90-day analysis from participants enrolled as of June 30, 2025, including functional changes scored according to the Movement Disorder Society Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) as well as biomarker data from cerebrospinal fluid and blood, will be available as expected in Q4 2025.
“We are grateful to the individuals participating in the study and to the dedicated team of clinical investigators for their continued support,” said Gene Mack, President and Chief Executive Officer of Gain Therapeutics. “We look forward to presenting early data from our Phase 1b study at the annual MDS meeting in October, additional data will be forthcoming throughout the fourth quarter of 2025. Early observations related to safety, tolerability, pharmacokinetics, and functional impact are encouraging. We are also pleased by the commitment of participants to continue treatment with GT-02287, allowing us to further evaluate long-term safety and efficacy.”
Information on the poster to be presented at International Congress of Parkinson’s Disease and Movement Disorders® is below:
Poster Title: GT-02287 in Parkinson’s Disease: Interim Data from a Phase 1b study
E-Poster Number: 891
E-Poster Session: Tuesday, October 7, 12:00 – 2:30pm HST
The International Parkinson and Movement Disorder Society (MDS) gathers thousands of the field’s clinicians, researchers, trainees and industry supporters with an interest in the current research and approaches for the diagnosis and treatment of movement disorders on an annual basis at its International Congress of Parkinson’s Disease and Movement Disorders®.
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.
Investors:
Gain Therapeutics, Inc.
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