Gain Therapeutics, Inc. (GANX), a clinical-stage biotechnology company, Friday announced that it has dosed the first participant in its Phase 1b clinical trial of GT-02287 for the treatment of Parkinson's disease or PD with or without a GBA1 mutation.
This trial is designed to evaluate the safety, tolerability, and pharmacokinetics of GT-02287 in individuals with GBA1-PD and idiopathic PD.
The trial will enroll up to 20 participants who will receive the drug daily for three months. Interim data from this trial are expected by the end of the second quarter 2025.
The Phase 1b trial follows the successful completion of a Phase 1 study in healthy volunteers, which demonstrated favorable safety and tolerability, as well as significant target engagement of GT-02287. The drug showed a >50 percent increase in glucocerebrosidase or GCase activity, a key enzyme linked to Parkinson's disease.
GT-02287 is designed to restore the function of the GCase enzyme, which is impaired in people with GBA1 mutations and age-related stress factors.
Preclinical data suggests that GT-02287 may slow or stop the progression of Parkinson's disease, making it a promising disease-modifying therapy.
Currently, GANX is trading at $2.19, down by 0.45 percent on the Nasdaq.
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