Gain Therapeutics, Inc. announced dosing of the first two subjects in a Phase 1 clinical trial of GT-02287, Gain?s lead drug candidate for the treatment of GBA1 Parkinson?s disease. The Company expects to complete this trial in the first half of 2024. Compelling preclinical data demonstrated that GT-02287 can restore the function of the lysosomal enzyme glucocerebrosidase (GCase), which becomes misfolded and dysfunctional due to a GBA1 gene mutation, the most common genetic risk factor for the development of Parkinson?s disease.

Restoring GCase function with GT-02287 was shown to have profound effect in animal models of Parkinson?s disease on the entire disease cascade, including a neuroprotective effect on dopaminergic neurons and improvement of motor deficiencies. Based on these data, GT-02287 has the potential to slow or even stop the progression of Parkinson?s disease. The Phase 1 clinical trial is a single center, randomized, double-blind, placebo-controlled, single- and multiple-ascending dose (SAD/MAD) 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 (MTD) and identify recommended doses for further clinical development in the setting of GBA1 Parkinson?s disease. In addition, as an exploratory endpoint, this study will look at GCase target engagement and activity in blood, which may provide an early clinical validation of the effect of GT-02287 on GCase. Gain?s broad pipeline of novel allosteric therapies, including GT-02287, was discovered via the Company?s SEE-Tx® drug discovery platform.

Designed to leverage AI-supported 3D structural biology and supercomputer-powered proprietary physics-based models, Gain is exploiting the untapped opportunities of allosteric binding sites and allosteric modulators to treat disease.