GT-02287 shows biomarker, symptom stability in Gain (GANX) Parkinson’s study
Rhea-AI Filing Summary
Gain Therapeutics reported interim results from Part 2, the nine‑month extension of its Phase 1b open‑label study of GT‑02287 in Parkinson’s disease. Sixteen of 19 patients from Part 1 chose to continue and all 16 have completed five months of dosing.
In patients with elevated baseline levels of the biomarker GluSph in cerebrospinal fluid, GluSph fell by an average of 81% after 90 days of treatment. At Day 150, these patients showed a 4.8‑point better combined MDS‑UPDRS Part II and III score than those with low baseline GluSph, and overall scores remained stable.
The study’s Data Monitoring Committee recommended continuation without changes, and patients reported perceived benefits such as improved smell, taste, balance, gait, and sleep. A planned Phase 2 trial is expected to add formal smell and gait assessments using UPSIT and Opal wearable sensors.
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Insights
Early GT-02287 data show strong biomarker shifts and stable symptoms but remain preliminary.
The interim Phase 1b extension data for GT‑02287 in Parkinson’s disease highlight an average 81% reduction in CSF GluSph after 90 days in patients who started with elevated levels, a mechanistically relevant biomarker tied to GCase dysfunction and alpha‑synuclein aggregation.
Clinically, MDS‑UPDRS Part II and III scores remained stable over 150 days across the study, with a 4.8‑point advantage for patients with high baseline GluSph, and 16 of 19 Part 1 participants continued into Part 2. A Data Monitoring Committee recommended the trial continue unchanged, supporting the current safety and tolerability profile.
The company plans a Phase 2 trial with structured endpoints such as UPSIT smell testing and Opal wearable gait assessments, which may better quantify patient‑reported benefits like improved smell, balance, and sleep. Actual impact will depend on future controlled data and confirmation of these trends.
8-K Event Classification
Key Figures
Key Terms
Phase 1b open-label clinical study medical
glucosylsphingosine (GluSph) medical
MDS-UPDRS medical
Data Monitoring Committee regulatory
DOPA decarboxylase (DDC) medical
University of Pennsylvania Smell Identification Test (UPSIT) medical
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Item 8.01. Other Events.
On May 26, 2026, Gain Therapeutics, Inc. (the “Company”) disclosed interim clinical and biomarker data from Part 2 (the nine-month extension) of its Phase 1b open-label clinical study of GT-02287 in patients with Parkinson’s disease with or without a GBA1 mutation. The data was released via an oral presentation at the 3rd International GBA1 Meeting 2026, held May 22–23, 2026, in Phoenix, AZ.
The data presented, which includes safety, tolerability, biomarkers, and clinical scores from the Phase 1b nine-month study extension support continued development of GT-02287 for PD. As previously reported, 16 of 19 participants who completed dosing in Part 1 of the Phase 1b chose to continue in the ongoing nine-month extension (Part 2), further supporting the tolerability of GT-02287. A Data Monitoring Committee meeting on March 5, 2026, concluded that the study should continue without any changes.
To date, all 16 participants remain on study and have completed five months of dosing (Day 150).
As previously reported, in all 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. Elevated GluSph, a result of glucocerebrosidase (GCase) dysfunction, has been shown to increase the aggregation of alpha synuclein and to impair mitochondrial and lysosomal function in neurons. Furthermore, in individuals with high levels of CSF GluSph at baseline, levels of DOPA decarboxylase (DDC) decreased following 90 days of treatment with GT-02287. DDC is responsible for converting levodopa into dopamine in the brain and is elevated in the CSF of people with Parkinson’s – likely due to dopaminergic neuron dysfunction
Figure 1: Change in MDS-UPDRS scores from Baseline at Day 150 in all patients for whom CSF GluSph and MDS-UPDRS scores were available, patients with low baseline GluSph, and patients with high baseline GluSph.
Participants with elevated baseline levels of GluSph in CSF continued to benefit more than those with low baseline levels of GluSph in CSF after 150 days of dosing with GT-02287, with a difference of 4.8 points in the sum of MDS-UPDRS Part II and Part III scores between the two groups at Day 150. MDS-UPDRS scores remained stable and durable across overall study population after 150 days of treatment with GT-02287.
Additionally, participants in the Phase 1b study provided unsolicited descriptions of perceived benefit after 90 days of dosing with GT-02287. Perceived benefits most commonly described included four instances of improved sense of smell and taste, four instances of improved balance or gait, and three instances of improved sleep. The planned Phase 2 clinical trial is anticipated to include endpoints to further assess treatment effects beyond benefits perceived and informally reported, including administration of the University of Pennsylvania Smell Identification Test (UPSIT) at baseline and at the end of the study, as well as the use of Opal wearable sensors for in-clinic gait assessments to be conducted at multiple timepoints throughout the study.
Signature
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| GAIN THERAPEUTICS, INC. | ||
| Dated: May 26, 2026 | By: | /s/ Gene Mack |
| Gene Mack | ||
| Chief Executive Officer | ||
FAQ
What did Gain Therapeutics (GANX) report about its GT-02287 Phase 1b study?
Gain Therapeutics reported interim data from the nine-month extension of its Phase 1b GT-02287 study in Parkinson’s disease. Sixteen patients continued treatment, with biomarker improvements, stable motor scores, and supportive safety oversight guiding plans for a more rigorous Phase 2 trial.
How many Parkinson’s patients remained on GT-02287 treatment in the Gain Therapeutics study?
Sixteen of 19 patients who completed Part 1 of the Phase 1b study chose to continue into the nine-month extension. All 16 remained in the trial and completed five months (Day 150) of dosing with GT-02287, supporting continued tolerability and engagement with therapy.
What biomarker changes did Gain Therapeutics (GANX) observe with GT-02287?
In participants with elevated baseline GluSph in cerebrospinal fluid, levels decreased by an average of 81% after 90 days of GT-02287. Elevated GluSph is linked to GCase dysfunction, alpha‑synuclein aggregation, and impaired neuronal function in Parkinson’s disease.
How did GT-02287 affect MDS-UPDRS scores in the Gain Therapeutics Phase 1b study?
MDS‑UPDRS scores remained stable across the overall study population after 150 days of GT‑02287 treatment. Patients with high baseline GluSph showed a 4.8‑point better combined MDS‑UPDRS Part II and III score at Day 150 compared with those starting with low GluSph.
What patient-reported benefits were noted with GT-02287 in the Gain Therapeutics trial?
Participants provided unsolicited reports after 90 days of GT‑02287, describing perceived improvements in smell and taste, balance or gait, and sleep. These observations were qualitative but are helping shape Phase 2 endpoints to more formally measure such potential benefits.
What are Gain Therapeutics’ next steps for GT-02287 in Parkinson’s disease?
Gain Therapeutics plans a Phase 2 clinical trial of GT‑02287 with added endpoints, including the University of Pennsylvania Smell Identification Test and Opal wearable sensor-based gait assessments, to more precisely measure treatment effects observed during the Phase 1b study.