Gain Therapeutics announces results from SAD part of Phase 1 GT-02287 trial

Gain Therapeutics announces results from the single ascending dose part of its Phase 1 study. GT-02287 was generally well tolerated up to and including the highest planned dose level, and there were no serious adverse events. The good safety and tolerability profile and the appropriate range of plasma exposure levels achieved after oral administration further bolster GT-02287’s best and first-in-class potential. The Phase 1 clinical trial is a single center, randomized, double-blind, placebo-controlled, single- and multiple ascending dose study to evaluate the safety and tolerability of GT-02287 administered orally once daily in healthy adults. The secondary objective is to evaluate the pharmacokinetics of SAD and MAD dose levels to identify a maximum tolerated dose and identify recommended doses for further clinical development in the setting of GBA1 Parkinson’s disease. The SAD part of the Phase 1 clinical trial enrolled 40 healthy participants across five separate cohorts – all of which were completed at the planned dose levels with no premature discontinuations or safety signals. The MAD part of the Phase 1 trials was initiated in February in parallel to the last SAD cohorts after approval from the Bellberry Human Research Ethics Committee in Australia – a decision based on a review of the safety and tolerability profile observed in the SAD cohorts. GT-02287 has been shown to restore the function of the lysosomal enzyme glucocerebrosidase, which becomes misfolded and dysfunctional due to a GBA1 gene mutation, the most common genetic risk factor for the development of Parkinson’s disease. Compelling preclinical data presented at WORLDSymposium earlier in February 2024 demonstrated that treatment with GT-02287 restored motor function and substantially reduced plasma levels of the emerging neurodegeneration biomarker NfL. Based on these data, GT-02287 may have the potential to slow the progression of Parkinson’s disease.