Gain Therapeutics Announces Presentations at AD/PDTM 2026 and Provides U.S. FDA Regulatory Update

Interim analysis of Phase 1b clinical study of GT-02287, including biomarker and clinical endpoints, will be presented in oral symposium

Company remains on track to initiate Phase 2 clinical trial of GT-02287 in 3Q26 after submitting additional preclinical data requested by FDA in support of an IND

BETHESDA, Md., March 12, 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 announced presentations at the AD/PD ™ 2026 International Conference on Alzheimer’s and Parkinson’s Disease and Related Neurological Disorders, being held March 17-21, 2026 in Copenhagen, Denmark. The Company also provided an update regarding its Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) to support a Phase 2 clinical trial of GT-02287, the Company’s lead drug candidate in clinical development for the treatment of Parkinson’s disease (PD).

Details of the AD/PD oral presentation are as follows:

Presentation Title: An Open-Label Phase 1b Study of GT-02287 in Parkinson’s Disease

Presenter: Jonas Hannestad, M.D., Ph.D., Chief Medical Officer

Session Name: Modulating Neuroinflammation, ?-Synuclein, LRRK2, And Dopaminergic Repair: Early Human Data

Date: March 18, 2026

Time: 3:15-3:30pm CET

Room: Hall 180-181

Details of the AD/PD poster presentations are as follows:

Poster Title: Novel Allosteric GCase Modulators, Different From the Clinical Stage GT-02287, for the Treatment of Parkinson’s Disease

Presenting Author: Ana Maria Garcia-Collazo, Ph.D., Head of Research

Poster Number: SHIFT 02-178

Dr. Hannestad will present data on GT-02287 demonstrating CNS target engagement, beneficial effects on downstream pathway abnormalities, early signs of clinical improvement in patients, and additional data from the extension phase of the study.

Dr. Garcia-Colazzo will present data on a novel glucocerebrosidase (GCase) modulator which is chemically distinct from GT-02287 with promising pre-clinical data in Parkinson’s disease models.

Regulatory Update on GT-02287

The Company submitted an IND to conduct a placebo-controlled, dose-range-finding Phase 2 clinical trial of GT-02287 in the United States. In March 2026, the Company submitted a response to the FDA’s request for additional preclinical data. The Company has been working closely with the FDA and expects a response over the next few weeks. The Company expects to begin its Phase 2 clinical trial in 3Q 2026, as previously reported.

Gene Mack, President and CEO of Gain Therapeutics, stated, “The data from our Phase 1b study furthers our hypothesis that GT-02287 is among the first disease-modifying therapies promising to shift the treatment paradigm in PD from symptom relief to halting or slowing symptom progression, targeting the causative biology (or pathophysiology) of PD to enable a more durable and predictable treatment effect for those living with PD.”

Mr. Mack continued, “As we continue to follow patients in the Phase 1b nine-month extension study that is expected to complete in September 2026, we look forward to continuing the productive dialogue with FDA in preparation for our Phase 2 clinical trial of GT-02287, which is designed to confirm the exciting results we have observed to date from our Phase 1b study, and remains on track to begin during 3Q26.”

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 ?-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 data in these models, demonstrating a disease-modifying effect of GT-02287, suggest that the drug candidate 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 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.

Initial results from the Phase 1b clinical trial in people with Parkinson’s disease demonstrated central nervous system target engagement, a reduction to baseline levels in the prespecified endpoint glucosylsphingosine, and improvement or stabilization in MDS-UPDRS scores.

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 timing of any responses from the FDA or other regulatory bodies and agencies; the timing of the commencement of any Phase 2 clinical studies for GT-02287; and the potential therapeutic and clinical benefits of the Company’s product candidates, including GT-02287. 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. 
Apaar Jammu 
Manager, Investor Relations and Public Relations
ajammu@gaintherapeutics.com

LifeSci Advisors LLC
Chuck Padala
Managing Director
chuck@lifesciadvisors.com

Media:
Russo Partners LLC
Nic Johnson and Elio Ambrosio
nic.johnson@russopartnersllc.com
elio.ambrosio@russopartnersllc.com
(760) 846-9256