Gain Therapeutics, Inc. GANX shares were trading higher Wednesday after the company presented positive data on two of its lead compounds.
What Happened: Bethesda, Maryland-based Gain announced positive data from a preclinical study evaluating two of its compounds, GT-02287 and GT-02329, which are being evaluated for the treatment of Gaucher and GBA1 Parkinson's disease.
The candidates demonstrated highly statistically significant effects on all tested phenotypes, with the company terming the results as a breakthrough in the potential to treat neurodegenerative disorders characterized by misfolded proteins, including Parkinson's, Alzheimer's and Gaucher's diseases and Lewy Body dementia.
The study results were presented at "The Michael J. Fox Foundation for Parkinson's Research's Innovating from Drug Discover to the Clinic: Novel Approaches to PD Therapeutic Development" webinar.
The Gain-sponsored study is being conducted at the University of Maryland School of Medicine.
The two lead compounds identified using Gain's proprietary SEE-Tx platform, have the ability to restore enzyme function of glucocerebrosidase, or GCase, an enzyme involved in various neurodegenerative diseases.
The compounds characterized in various assays and showed the potential to halt the progression in Parkinson's patients with GBA1 gene mutations as well as patients whose GCase protein is misfolded due to ageing cellular processes, the company said.
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Why It's Important: Given that STARs are small molecule therapies, they have significant advantages over the current standard of care for disorders associated with protein misfolding, including most enzyme replacement therapies and gene therapies.
They can be administered as a simple oral pill and can reach organs and tissues not typically accessible through current therapeutic options, including the brain, bone and cartilage. In addition, the company believes that STARs are relatively easy and cost-effective to manufacture.
GANX Price Action: At last check, Gain shares were soaring 13.53% to $8.10.
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