Gain Therapeutics (NASDAQ:GANX) Initiates Phase 1b Clinical Trial for GT-02287 in Australia

Gain Therapeutics, Inc., a clinical-stage biotechnology company, has recently announced the initiation of a Phase 1b clinical trial for its lead candidate, GT-02287, in Australia. The trial has received approval and will focus on assessing the safety and tolerability of GT-02287 in individuals diagnosed with Parkinson’s disease (PD) with or without a GBA1 mutation.

In a press release issued on December 23, 2024, Gain Therapeutics revealed that the Phase 1b trial follows a successful Phase 1 study conducted in healthy volunteers in the third quarter of 2024. The Phase 1 study demonstrated favorable safety and tolerability of GT-02287, as well as significant target engagement by showing a notable increase in glucocerebrosidase (GCase) activity of over 50%.

GT-02287 is a small molecule designed by Gain Therapeutics for the treatment of PD. The Phase 1b trial aims to evaluate the safety and tolerability of GT-02287 in PD patients and will be carried out at seven sites across major metropolitan areas in Australia. The trial will involve administering 13.5 mg/kg/day of GT-02287 for three months to patients with either GBA1-PD or idiopathic PD. Secondary endpoints will include assessing pharmacokinetics, GCase modulation, levels of GCase substrates, and other biomarkers in plasma and cerebrospinal fluid.

The primary objectives of the Phase 1b trial are to observe the safety and tolerability of GT-02287 and analyze its effects on key biomarkers of Parkinson’s disease. Data from the trial is expected to be available by mid-2025. Gain Therapeutics highlighted that the successful completion of this trial would pave the way for the preparation of a Phase 2 trial for GT-02287 in 2025.

Gain Therapeutics emphasized the importance of this step in advancing GT-02287 towards potentially slowing or halting the progression of Parkinson’s disease. The Company expressed eagerness for the trial’s results and highlighted the significant progress made in transitioning from preclinical to clinical stage development.

Investors and medical enthusiasts are encouraged to follow the progress of Gain Therapeutics as they navigate through the clinical trials of GT-02287 which hold promising implications for the treatment of Parkinson’s disease.

For more information, the full press release can be accessed on the official website of Gain Therapeutics or through the following clinical trials link: https://clinicaltrials.gov/study/NCT06732180.

This article was generated by an automated content engine and was reviewed by a human editor prior to publication. For additional information, read Gain Therapeutics’s 8K filing here.

About Gain Therapeutics

Gain Therapeutics, Inc, a biotechnology company, develops novel small molecule therapeutics to treat diseases across various therapeutic areas. Its drug discovery platform Magellan discovers novel allosteric binding sites in a disease; identifies proprietary small molecules that bind these sites to modulate protein function; and treats the underlying cause of the disease.