BETHESDA, Md., March 26, 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 reported financial results for the fourth quarter and year ended December 31, 2025, and provided a corporate update.
"We are encouraged by the progress made in 2025, as we made important advancements related to both the scientific understanding and clinical development of our lead candidate GT-02287, in development for the treatment of Parkinson's disease with or without a GBA1 mutation. The promising impact GT-02287 has on the causative biology of Parkinson's disease has been further elucidated from the analysis of functional and biomarker changes from the Phase 1b study," commented Gene Mack, President and CEO of Gain Therapeutics.
Mr. Mack continued, "We will continue to follow patients in the Phase 1b nine-month extension study that is expected to complete in September 2026 and look forward to presenting longer-term follow up at additional scientific conferences throughout the balance of the year. To date, MDS-UPDRS scores remained stable and durable across the overall study population after 150 days of treatment with GT-02287 and are becoming more encouraging with the passage of time. We believe the totality of the data continues to support the potential of GT-02287 in both idiopathic and GBA1 Parkinson's disease and we hope to one day shift the treatment paradigm away from symptomatic relief and to disease modification."
Fourth Quarter 2025 and Recent Corporate and Pipeline Highlights
Pipeline Updates
-- Presented initial data from the Phase 1b clinical study of GT-02287
demonstrating the disease-modifying potential of GT-02287 in people with
Parkinson's disease $(PD)$ at the International Congress of Parkinson's
Disease and Movement Disorders$(R)$ in October 2025 in Honolulu, HI.
-- Presented preclinical data demonstrating evidence that GT-02287
beneficially impacts mitochondrial health in addition to lysosomal health
and endoplasmic reticulum stress at Neuroscience 2025 in November 2025 in
San Diego, CA.
-- Presented additional data from the Phase 1b clinical study of GT-02287
demonstrating central target engagement, beneficial effects on downstream
pathway abnormalities, and clinical improvement in participants, that
supported the disease-modifying potential of GT-02287 at AD/PD$(TM)$ 2026
International Conference on Alzheimer's and Parkinson's Disease and
Related Neurological Disorders in March 2026 in Copenhagen, Denmark.
-- As of March 2026, 14 of 16 participants enrolled in Phase 1b
nine-month extension had completed 5 months of dosing (Day 150).
-- MDS-UPDRS scores remained stable over 150 days of dosing.
-- In participants with elevated baseline levels of
glucosylsphingosine (GluSph) in cerebrospinal fluid $(CSF)$, GluSph
decreased by an average of 81% after 90 days of treatment with
GT-02287.
-- In participants with elevated baseline levels of GluSph in CSF,
levels of DOPA decarboxylase $(DDC)$ decreased following 90 days of
treatment with GT-02287.
-- Also at AD/PD(TM) 2026, presented new series of novel glucocerebrosidase
(GCase) allosteric modulators, represented by the advanced lead GT-04686,
identified by the Company's proprietary Magellan(TM) drug discovery
platform.
-- Novel chemical series, led by GT-04686, demonstrated activity in
both in vitro and in vivo models, including increase in GCase
activity and lipid substrate depletion in patient fibroblasts
harboring both mutated and wildtype GBA1, as well as restoration
of motor and non-motor function in an animal model of Parkinson's
disease.
-- GT-04686 is ready for IND-enabling studies for the treatment of
Parkinson's disease and other neurological disorders.
Corporate Updates
-- Publication of Phase 1 clinical study data detailing GCase target
engagement and therapeutic plasma and cerebrospinal fluid levels after
GT-02287 administration in healthy volunteers in Movement Disorders, an
official journal of the International Parkinson and Movement Disorder
Society.
-- Hosted a KOL event highlighting current thinking on Parkinson's disease
clinical outcome scales and biomarkers as well as the Company's initial
Phase 1b data. A replay of the event can be found here.
-- Hosted a KOL event highlighting biomarker evidence supporting
disease-modifying potential of GT-02287 and current thinking on GCase
substrates and biomarkers in PD. A replay of the event can be found here.
-- Completed enrollment in the Phase 1b nine-month extension for GT-02287 in
people with PD. 16 participants have been enrolled as of January 2026.
-- Continued Investigational New Drug $(IND)$ engagement with the U.S. Food
and Drug Administration (FDA) in preparation for GT-02287 Phase 2
clinical development in the United States.
Upcoming Anticipated Milestones
-- Clearance of IND submission to FDA, expected in 2Q26, facilitating Phase
2 clinical development of GT-02287 to include clinical sites in the
United States.
-- Phase 2 clinical trial of GT-02287 in people with PD expected to begin in
3Q26.
-- Results from Phase 1b clinical study of GT-02287 expected in 4Q26.
Year-End 2025 Financial Results
Research and development (R&D) expenses decreased by $0.6 million to $10.2 million for the year ended December 31, 2025, as compared to $10.8 million for the year ended December 31, 2024. The decrease in research and development expenses was primarily related to optimization of our pipeline costs. The decrease was partially offset by unfavorable foreign exchange currency translation as the Swiss franc strengthened against the U.S. dollar.
General and administrative (G&A) expenses decreased by $1.1 million to $8.5 million for the year ended December 31, 2025, from $9.6 million for the year ended December 31, 2024. The decrease in general and administrative expenses was primarily attributable to lower stock-based compensation and lower legal fees. The decrease was partially offset by higher personnel costs and unfavorable foreign exchange currency translation as the Swiss franc strengthened against the U.S. dollar.
Net loss for the year ended December 31, 2025, was $20.2 million, or $0.61 per share, basic and diluted, compared to $20.4 million, or $0.89 per share, basic and diluted, for the year ended December 31, 2024.
Cash, cash equivalent and marketable securities were $20.8 million as of December 31, 2025, compared to $10.4 million as of December 31, 2024.
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 <ALPHA>-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 (GluSph), 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.
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