Gain Therapeutics has dosed the first Parkinson’s disease patient in a Phase 1b clinical trial of its lead candidate, GT-02287. The trial will assess the drug’s safety and tolerability in patients with Parkinson’s disease, both with and without a GBA1 mutation. Interim analysis is expected by the end of Q2 2025.
This trial initiation is a critical step for Gain Therapeutics and potentially for Parkinson’s disease treatment. A successful outcome could validate GT-02287’s mechanism of action, specifically its ability to restore glucocerebrosidase (GCase) function. This is particularly relevant because GBA1 mutations, which impair GCase, are the most common genetic risk factor for Parkinson’s disease. A therapy addressing this underlying mechanism holds the promise of disease modification, a significant advancement over current symptomatic treatments.
The Phase 1b trial is an open-label, multi-center study involving up to 20 participants who will receive daily doses of GT-02287 for three months. Secondary endpoints include pharmacokinetics, GCase modulation, substrate levels, and other biomarkers in plasma and cerebrospinal fluid. This follows a successful Phase 1 study in healthy volunteers, where GT-02287 demonstrated a favorable safety profile, target engagement, and significant increases in GCase activity. Preclinical studies have shown the drug’s potential to reduce alpha-synuclein aggregation, neuroinflammation, and neuronal death, along with improvements in motor and cognitive function. Additionally, GT-02287 reduced plasma neurofilament light chain levels, a promising biomarker for neurodegeneration.
Positive interim results from the Phase 1b trial could significantly boost Gain Therapeutics’ position in the Parkinson’s disease therapeutic landscape. It would pave the way for larger, later-stage trials, potentially establishing GT-02287 as a much-needed disease-modifying therapy for a large patient population. This success could also spur further research into GCase modulation as a therapeutic strategy for Parkinson’s disease, opening new avenues for drug development.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
