1. GT-02287 reduces GCase substrate in PD patients' cerebrospinal fluid. 2. Reduction of glucosylsphingosine suggests increased GCase activity in the brain. 3. 79% of participants continued treatment in the Phase 1b study extension. 4. The Phase 1b study shows GT-02287 is generally well-tolerated. 5. Upcoming KOL event to discuss emerging biomarker data on January 6, 2026.
FAQ
Why Bullish?
The reduction in GluSph indicates positive therapeutic efficacy for GT-02287. Historical data on biopharmaceutical pipeline advancements suggest similar results have led to stock price gains.
How important is it?
Positive results in CNS engagements and potential market differentiation of GT-02287 drive investor sentiment.
Why Long Term?
The long-term positive data from ongoing studies can create sustained bullish pressure on GANX stock.
Gain Therapeutics Announces Promising Phase 1b Clinical Study Results for GT-02287 in Parkinson's Disease
BETHESDA, Md., December 18, 2025 (GLOBE NEWSWIRE) - Gain Therapeutics, Inc. (Nasdaq: GANX), a clinical-stage biotechnology company focused on allosteric small molecule therapies, has announced significant results from its Phase 1b clinical study of GT-02287. This marks a breakthrough in the exploration of treatments for Parkinson's disease (PD), providing the first evidence of reduced glucocerebrosidase (GCase) substrate glucosylsphingosine (GluSph) in cerebrospinal fluid (CSF), a prespecified exploratory endpoint.
Key Findings from the Clinical Study
The study's findings reveal that participants displayed substantial decreases in GCase substrate GluSph levels in CSF after 90 days of treatment with GT-02287. This is particularly noteworthy as it suggests enhanced GCase activity in the brain, which could have implications for the progression of PD.
- Reduction in CSF GluSph levels indicates increased GCase activity.
- 90 days of treatment resulted in significant decreases towards levels typical in healthy individuals.
- The change in GluSph levels was a prespecified exploratory endpoint of the Phase 1b study.
CEO Remarks on Results
Gene Mack, president and CEO of Gain Therapeutics, expressed enthusiasm about the results. He stated, “We are excited by the unfolding biomarker evidence of GT-02287 activity and CNS target engagement. To our knowledge, GT-02287 is the first GCase modulator to demonstrate a reduction of GluSph in CSF in people with PD, providing downstream evidence of GCase enhancement in the brain.”
Mack further noted the potential impact of reduced GluSph levels on neuronal health, suggesting these changes may lead to clinically observable improvements in PD patients.
Details of the Phase 1b Study
The initial 90-day dosing part of the Phase 1b study enrolled 21 participants, with 19 completing the period. Out of these, 15 (79%) opted to extend their participation for an additional nine months, which is anticipated to conclude in September 2026. This ongoing study aligns with earlier findings presented at the International Congress of Parkinson’s Disease and Movement Disorders in October 2025, highlighting that GT-02287 remains well-tolerated within the projected therapeutic range.
Upcoming Discussion and Future Directions
Gain Therapeutics plans to host a virtual Key Opinion Leader (KOL) event on January 6, 2026, where experts will delve deeper into the biomarkers and implications of GT-02287 in PD treatment.
This meeting will feature insights from renowned figures in the field, including Roy Alcalay, M.D., M.Sc., and Peter Lansbury, Ph.D., providing additional context to the findings from the Phase 1b study.
About GT-02287
GT-02287, Gain Therapeutics' lead drug candidate, is currently in clinical development for PD treatment, specifically targeting patients with or without a GBA1 mutation. This orally administered small molecule functions as an allosteric enzyme modulator that aims to restore GCase function, addressing the misfolding and impairment associated with genetic mutations.
In preclinical models, GT-02287 has demonstrated promising outcomes, including:
- Restoration of GCase enzymatic function.
- Reduction of neuroinflammation and neuronal death.
- Improvement in motor function and behavioral deficits in rodent models.
Financial Outlook
Gain Therapeutics is well-positioned financially, with sufficient capital to fund operations through the ongoing Phase 1b extension and into 2026. The company's innovative approach to drug discovery aims to pave the way for new therapies for difficult-to-treat disorders, including neurodegenerative diseases and other genetic conditions.
About Gain Therapeutics, Inc.
Gain Therapeutics, Inc. is dedicated to the discovery and development of next-generation allosteric therapies. The lead candidate, GT-02287, reflects Gain's commitment to addressing unmet medical needs in neurological disorders and beyond.