Gain Therapeutics Unveils Promising Biomarker Evidence for GT-02287’s Disease-Modifying Potential
BETHESDA, Md., January 6, 2026 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX), a clinical-stage biotechnology company specializing in allosteric small molecule therapies, has announced significant findings from its Phase 1b clinical study of GT-02287. This investigational therapy is designed for individuals with Parkinson’s disease (PD), both with and without a GBA1 mutation, revealing compelling biomarker data that underscores its potential as a disease modifier.
Key Findings from the GT-02287 Clinical Study
The latest data highlights a remarkable average reduction of 81% in glucosylsphingosine (GluSph) levels in cerebrospinal fluid (CSF) after a 90-day treatment with GT-02287. Elevated GluSph is indicative of GCase dysfunction, which contributes to the aggregation of alpha-synuclein and deteriorates mitochondrial function in neurons.
- 81% average reduction in GluSph after treatment.
- Significant performance improvement in Movement Disorder Society Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) scores by an average of 2.20 points among 15 evaluated patients.
These findings represent a landmark in the treatment of PD as they suggest enhanced GCase activity in the brain, which may positively affect disease progression.
Patient Outcomes and Expert Insights
As of November 30, 2025, 19 patients completed the first part of the study. Some patients were excluded from the MDS-UPDRS analysis due to inconclusive PD diagnoses. However, the remaining group demonstrated notable improvements in their symptoms.
Gene Mack, President and CEO of Gain Therapeutics, stated, “These results mark an important step forward for our program. The observed decrease in GluSph, alongside improvement or stabilization of MDS-UPDRS scores, provides encouraging evidence that our approach is engaging the underlying biology of the disease, suggesting a disease-modifying action.”
Michele DeSciscio, lead investigator, adds, “...while this study was not designed to assess clinical efficacy, it has been encouraging to see stabilization in MDS-UPDRS scores and anecdotal improvements in areas such as balance and gait.”
Upcoming Virtual KOL Event
To further discuss these ground-breaking results, Gain Therapeutics is hosting a virtual event titled “Understanding GCase Substrates in Parkinson’s Disease,” featuring key opinion leaders Dr. Roy Alcalay and Dr. Peter Lansbury at 10:00 a.m. ET today. This event will delve into the emerging biomarker data from the Phase 1b study of GT-02287 and include a Q&A session.
Interested participants can register for the event here.
About GT-02287 and Gain Therapeutics
GT-02287 is Gain Therapeutics' lead therapeutic candidate, targeting both idiopathic PD and GBA1-PD. It functions as an allosteric enzyme modulator to restore glucocerebrosidase (GCase) activity, critically impaired by genetic mutations and age-related factors.
Preclinical studies have shown GT-02287’s ability to mitigate significant symptoms associated with PD, including motor function, neuroinflammation, and neurodegeneration, promising a disease-altering impact. The company is funding this initiative, supported by organizations such as The Michael J. Fox Foundation for Parkinson’s Research.
Forward-Looking Statements
This press release includes forward-looking statements in compliance with the Private Securities Litigation Reform Act of 1995. These statements, which are often accompanied by terms such as “believes,” “expects,” or “anticipates,” reflect the company’s current views and expectations. Actual results may differ materially due to various risks and uncertainties.