BETHESDA, Md., May 11, 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 quarter ended March 31, 2026, and provided a corporate update.
"The first quarter of 2026 marked another exciting quarter for Gain, as we continued to build on the important advancements achieved in 2025 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. This is underscored by the promising biomarker and early clinical evidence from our ongoing Phase 1b study supporting the potential disease modifying properties of GT-02287 that we presented at AD/PD$(TM)$ 2026 in Copenhagen this past March," said Gene Mack, President and CEO of Gain Therapeutics.
Mr. Mack added, "The analysis of biomarkers and clinical evidence of efficacy is ongoing throughout the nine-month extension of the Phase 1b study, which is expected to complete in October 2026. MDS-UPDRS scores have remained durable to date, and we look forward to sharing more details at the upcoming 3(rd) International GBA1 Meeting later this month in Phoenix, Arizona. We believe the totality of the data supports the potential of GT-02287 to treat both idiopathic and GBA1 Parkinson's disease, and we remain focused on helping to shift the treatment paradigm from managing symptoms of Parkinson's disease towards disease modification and addressing the underlying biology causing those symptoms. We expect to receive FDA clearance of our IND during Q2 2026 and remain on track to begin our Phase 2 study in Q3 2026."
First Quarter 2026 and Recent Corporate and Pipeline Highlights
Pipeline Updates
GT-02287
-- 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 five 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.
GT-04686
-- Also, at AD/PD(TM) 2026, presented new series of novel glucocerebrosidase
(GCase) allosteric modulators, represented by the advanced lead GT-04686,
identified using 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
-- Hosted a KOL event in January 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. All 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.
-- Publication of in vitro research on new small molecule allosteric
modulators of <BETA>-galactosidase, identified using the Company's
Magellan(TM) drug discovery platform, targeting GM1 gangliosidosis and
Morquio B, in the International Journal of Molecular Sciences, one of the
Multidisciplinary Digital Publishing Institute's (MDPI) flagship
publications.
Upcoming Anticipated Milestones
-- FDA clearance of our IND submission, expected in Q2 2026, 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 Parkinson's disease
expected to begin in Q3 2026.
-- Results from Phase 1b clinical study of GT-02287 expected in Q4 2026.
Q1 2026 Financial Results
Research and development (R&D) expenses increased by $0.5 million to $2.8 million for the three months ended March 31, 2026, as compared to $2.3 million for the three months ended March 31, 2025. The increases in research and development expenses were primarily related to costs associated with the Company's lead program compound GT-02287 for the treatment of Parkinson's disease, unfavorable foreign exchange currency translation as the Swiss franc and Australian dollar strengthened against the U.S. dollar, and higher research and development personnel costs.
General and administrative (G&A) expenses increased by $0.5 million to $2.6 million for the three months ended March 31, 2026, as compared to $2.1 million for the three months ended March 31, 2025. The increases in general and administrative expenses for the period were primarily attributable to higher professional fees, higher personnel costs, and unfavorable foreign exchange currency translation as the Swiss franc and Australian dollar strengthened against the U.S. dollar.
Net loss for the three months ended March 31, 2026, was $0.13 per share, basic and diluted, compared to $0.16 per share, basic and diluted, for the three months ended March 31, 2025.
Cash, cash equivalents and marketable securities were $16.5 million as of March 31, 2026, compared to $20.8 million as of December 31, 2025.
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. Additionally, participants with elevated levels of CSF GluSph also exhibited elevated levels of DOPA decarboxylase (DDC), which decreased following treatment with GT-02287.
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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