EXCLUSIVE: Gain Therapeutics Highlights Lead Program Data Showcasing Its Impact On Cellular Death For Treating Parkinson's Disease

Gain Therapeutics Inc GANX announces the presentation of a poster at the International Conference on Alzheimer’s and Parkinson’s Diseases and related neurological disorders (AD/PD 2024) related to the mechanism of action of the company’s lead compound, GT-02287.

The poster demonstrates how GT-02287, through its interaction with glucocerebrosidase (GCase) in the endoplasmic reticulum (ER), aids in correct GCase folding, preventing ER retention, ER stress and ER-associated degradation of mutated GCase enzyme.

GCase is consequently able to travel to the lysosome, resulting in enhanced lysosomal activity and efficient processing of the GCase substrate glucosylceramide. 

An increase in GCase substrate in the lysosome was previously shown to be associated with the accumulation of aggregated alpha-synuclein, a pathological hallmark of Parkinson’s disease and related disorders. 

“These data further confirm our understanding of the mechanism of action of GT-02287, how it impacts cellular health by preventing the downstream consequences of GCase misfolding, including cellular stress and lysosomal dysfunction” commented Dr. Natalia Perez-Carmona, Senior Director of Biology at Gain Therapeutics and presenting author. 

Gain Therapeutics’ lead drug candidate, GT-02287, is in clinical development for GBA1 Parkinson’s disease (GBA1-PD). 

The orally administered, brain-penetrant small molecule is an allosteric protein modulator that restores the function of the lysosomal protein enzyme glucocerebrosidase (GCase), which becomes misfolded and impaired due to a GBA1 gene mutation, the most common genetic abnormality associated with PD. 

Last week, Gain Therapeutics initiated the Multiple Ascending Dose (MAD) part of the Phase 1 trial of GT-02287 for GBA1 Parkinson’s disease.

Last month, Gain Therapeutics announced preclinical data demonstrating that its GT-02287 provided neuroprotection and restored motor function in Parkinson’s disease models following delayed administration. 

Animals in the most challenging treatment group – those that began treatment eight days following the onset of the disease – showed motor improvement from day 14 to day 27, which suggests a progressive reversal of neuronal deficit associated with continued treatment duration.

Price Action: GANX shares are trading higher by 3.60% at $4.89 premarket on the last check Tuesday.

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