GAIN THERAPEUTICS, INC. Gain Therapeutics, Inc. announced preclinical data demonstrating the Company's lead drug candidate GT-02287 significantly decreased Parkinson's disease (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 (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(R) being held in Copenhagen, Denmark from August 27-31, 2023.
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
Prominently, GT-02287 significantly reduced plasma NfL levels in this GBA1-PD model. The emerging neurodegeneration biomarker was previously accepted by the U.S. Food and Drug Administration ("FDA") for the accelerated approval of tofersen for the treatment of amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene (SOD1-ALS) and was recommended by the FDA as an exploratory endpoint for neuronopathic mucopolysaccharidosis II (MPS II) clinical trials. Data from the poster describes the effect of GT-02287 on rotenone-induced neurotoxic effects in a PD animal model.
GT-02287 restored GCase levels in the brain and reduced the accumulation of toxic GCase substrates in the cerebrospinal fluid. Additionally, GT-02287 reduced aggregated
