Gain Therapeutics, Inc. (GANX) has reported positive results from its Phase 1 clinical trial evaluating the safety and efficacy of its drug candidate, GT-02287, for the treatment of Parkinson’s disease. The company previously announced that no serious adverse events were observed during the trial.
The Phase 1 study enrolled 72 healthy volunteers and assessed the safety, tolerability, and pharmacokinetics of GT-02287. A review of the unblinded data after the database was locked confirmed that single and multiple doses of GT-02287 were safe and well-tolerated across all age groups, even at the highest planned dose levels. Importantly, the study demonstrated that GT-02287 reached the cerebrospinal fluid (CSF), suggesting it can effectively reach the brain, where it targets its intended mechanism of action.
The favorable safety and tolerability profile at oral dose levels that resulted in therapeutic plasma levels, CNS exposure, and target engagement, make GT-02287 a promising candidate for further development.
“On the heels of this data, we expect to initiate a trial in people with Parkinson’s disease by Q4 2024 with the goal of demonstrating safety and tolerability in patients with Parkinson’s disease and to obtain proof of mechanism based on relevant biomarkers. We anticipate having data from Parkinson’s disease patients by mid-point 2025,” commented Jonas Hannestad, Chief Medical Officer of Gain.
This news follows the release of preclinical data in February, which demonstrated that GT-02287 provided neuroprotection and restored motor function in Parkinson’s disease models.
The positive results from the Phase 1 trial are a significant step forward for Gain Therapeutics and their efforts to develop a new treatment for Parkinson’s disease. The company is now focused on initiating a Phase 2 trial in patients with Parkinson’s disease, with the goal of demonstrating safety, tolerability, and efficacy in this patient population. If successful, GT-02287 could offer a new hope for the millions of people living with Parkinson’s disease worldwide.
