Gain Therapeutics (NASDAQ:GANX) has announced positive results from its Phase 1 study evaluating the safety, tolerability, and pharmacokinetics of GT-02287, a novel GCase-targeting small molecule therapy for Parkinson’s disease.
The company reports that the review of unblinded data following database lock confirmed that single and multiple doses of GT-002287 were safe and generally well-tolerated up to and including the highest planned dose levels across all age levels; approximately 50% of participants were aged 50 and over.
Moreover, Gain suggests the results demonstrated a favorable safety and tolerability profile at oral dose levels, resulting in therapeutic plasma levels, CNS exposure, and target engagement, further strengthens GT-02287’s potential as a lead treatment for Parkinson’s disease in patients with or without a GBA1 mutation.
“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 disease and to obtain proof of mechanism based on relevant biomarkers. We anticipate having data from Parkinson’s disease patients by mid-point 2025,” said Jonas Hannestad, M.D., Ph.D., chief medical officer of Gain.
In a statement, Gene Mack, Interim CEO and current CFO of Gain, said, “We are enthusiastic about the promising profile of GT-02287, particularly with the added observation of CNS exposure and target engagement. We’re proud to be executing on our near-term clinical milestones and look forward to presenting the full data and advancing GT-02287 further in the clinic with the goal of improving the lives of people with Parkinson’s disease.”
