Gain Therapeutics announces preclinical data from drug candidate GT-02287

Gain Therapeutics announced preclinical data demonstrating the Company’s lead drug candidate GT-02287 significantly decreased Parkinson’s disease, or PD,-associated pathology and improved motor dysfunction in two different preclinical models of Parkinson’s disease. Of note, GT-02287 significantly reduced plasma Neurofilament Light Chain, or NfL, levels, an emerging biomarker for neurodegeneration, in a mouse model of GBA1-PD. The findings are being presented in two posters, including one that was accepted as a late breaking abstract, at the International Congress of Parkinson’s Disease and Movement Disorders being held in Copenhagen, Denmark from August 27-31. The first poster titled “Neuroprotective effect of GT-02287, a brain-penetrant structurally targeted allosteric regulator of glucocerebrosidase, leads to a significant reduction of plasma NfL levels and improvement in behavioural deficits in a mouse model of GBA1 Parkinson’s disease” will be presented by Dr. Taylor. Researchers used a mouse model of GBA1-PD, created using conduritol beta epoxide (CBE) to induce GCase deficit comparable to GBA1-PD. Oral administration of GT-02287 over 14 days significantly reduced aggregated alpha-synuclein, neuroinflammation and neuronal death in the brain. Treatment with GT-02287 also increased tyrosine hydroxylase immunostaining, a marker of dopaminergic neurons, as well as dopamine levels, and improved motor strength and motor coordination.