Gain Therapeutics Presents Initial Data from Phase 1b Clinical Study of GT-02287 in Parkinson's Patients at International Congress of Parkinson's Disease and Movement Disorders®
Improvement observed in MDS-UPDRS scores in first 9 participants enrolled; 90-day follow-up continuing in remaining participants
Plasma exposures in patients within projected therapeutic range and comparable to exposures observed in Phase 1 healthy volunteer study
GT-02287 was generally well-tolerated with no treatment emergent serious adverse events (TESAEs); no changes recommended from two independent DMC reviews
Gain to host GT-02287 KOL event on October 14th at 4 p.m. EST
BETHESDA, Md., Oct. 06, 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 presented a poster at the International Congress of Parkinson's Disease and Movement Disorders® summarizing early safety and tolerability findings, the primary endpoint, and observations from key exploratory endpoints from the Phase 1b clinical study of GT-02287 in people with Parkinson's disease (PD) with or without a GBA1 mutation.
"We are pleased to be able to present encouraging early takeaways from our Phase 1b study," said Gene Mack, President and Chief Executive Officer of Gain Therapeutics. "The stability and trending improvements we have observed in MDS-UPDRS scores was not something we were expecting to see with 90 days of dosing. We look forward to further contextualizing our observations in an upcoming webinar."
The poster, titled, "GT-02287 in Parkinson's Disease: Interim data from a Phase 1b study," was presented on-site by Chief Medical Officer Jonas Hannestad, M.D., Ph.D. GT-02287 was generally well tolerated, with no treatment-emergent serious adverse events observed. A transient increase in alkaline phosphatase and other liver enzymes has been observed in some participants and normalized despite ongoing dosing. At two different meetings, the data monitoring committee (DMC) has recommended continuation of the study and more recently, Australian health authorities have approved the Phase 1b study extension for patients who can now be treated for up to 12 months.
Of the 21 participants, two are treatment-naïve, two are on deep brain stimulation, and 18 are on levodopa and/or dopamine agonists or other PD drugs.
Mean MDS-UPDRS scores at baseline were 5.8, 7.4, and 24.7 for Parts I, II, and III, respectively. Importantly, several participants experienced an improvement in their UPDRS Part II and III scores after 90 days of dosing with GT-02287 while mean Part I scores remained unchanged. The mean improvement in Parts II and III by Day 90, which was not observed by Day 30, suggests that GT-02287 has a disease-slowing effect, consistent with the preclinical models in vivo and the proposed mechanism of action of GT-02287, supporting continued development.
The plasma pharmacokinetics (PK) profile was consistent across all 14 participants sampled, was within the projected therapeutics range, and comparable to exposures observed in healthy volunteers in the Phase 1 study.
A PDF of the poster presented at the International Congress of Parkinson's Disease and Movement Disorders is available on the Science and Technology section of the Company's website at https://gaintherapeutics.com/science-and-technology/posters.
Upcoming Virtual KOL Event Registration
Gain Therapeutics will host a webinar on Tuesday, October 14 at 4:00pm ET to review results presented at the International Congress of Parkinson's Disease and Movement Disorders. Further details about the event will be issued at a later date in a separate press release. To register, please click here: https://lifescievents.com/event/gst492thwp/
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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