Gain Therapeutics, Inc. (Nasdaq: GANX) announced that its Chief Medical Officer, Jonas Hannestad, M.D., Ph.D., will present crucial Phase 1 data for their lead drug candidate GT-02287 at the upcoming Michael J. Fox Foundation's (MJFF) 16th Annual Parkinson's Disease Therapeutics Conference in New York City.
Clinical Development Progress
The presentation, scheduled for October 17, 2024, will showcase results from GT-02287's Phase 1 trial in healthy volunteers. Initial data indicate that the drug candidate is well-tolerated and achieves therapeutic exposure levels previously shown to affect Parkinson's disease pathobiology in preclinical models. Importantly, the compound demonstrated successful target engagement with glucocerebrosidase (GCase), a key enzyme implicated in Parkinson's disease pathology.
Innovative Therapeutic Approach
GT-02287 represents a novel approach to treating Parkinson's disease, functioning as an orally administered, brain-penetrant small molecule that acts as an allosteric protein modulator. The drug's mechanism focuses on restoring the function of GCase, which becomes impaired due to GBA1 gene mutations or age-related stress factors.
Promising Preclinical Evidence
Preclinical studies have yielded encouraging results across multiple disease markers. The compound has shown ability to:
- Restore GCase enzymatic function
- Reduce aggregated α-synuclein
- Decrease neuroinflammation and neuronal death
- Improve motor function and cognitive performance
- Lower plasma neurofilament light chain (NfL) levels, a key neurodegeneration biomarker
Multi-Institutional Support
The development program has garnered significant support from leading organizations in Parkinson's research, including funding from:
- The Michael J. Fox Foundation for Parkinson's Research
- The Silverstein Foundation for Parkinson's with GBA
- The Eurostars-2 joint program with EU Horizon 2020 research
- Innosuisse – Swiss Innovation Agency
Therapeutic Potential
GT-02287's demonstrated effects in both GBA1-mutation carriers and idiopathic Parkinson's disease models suggest broader therapeutic potential. The drug candidate shows promise as a disease-modifying treatment, potentially capable of slowing or halting disease progression rather than merely managing symptoms.
"We look forward to presenting Phase 1 data for GT-02287 at the upcoming MJFF conference and appreciate the opportunity to contextualize these data with exciting preclinical results," stated Dr. Hannestad, highlighting the significance of this milestone in the drug's development journey.
