Gain Therapeutics Achieves Key Milestones in Phase 1 Study of GT-02287, a Promising Small Molecule Therapy for Parkinson’s Disease
Bethesda, Md.’ - Gain Therapeutics, Inc. (Nasdaq: GANX), a clinical-stage biotechnology company pioneering the discovery and development of next-generation allosteric small molecule therapies, has announced the completion of dosing in the Multiple Ascending Dose (MAD) part of its Phase 1 clinical study for GT-02287. This novel small molecule therapy is designed to target the GCase enzyme, a key player in the pathology of GBA1 Parkinson’s disease.
GT-02287 has shown encouraging safety and tolerability across both the Single Ascending Dose (SAD) and MAD components of the Phase 1 study. In the MAD segment, completed recently, GT-02287 was well-tolerated, with no serious adverse events or other significant safety concerns reported. This outcome mirrors the positive results from the SAD segment, where GT-02287 also demonstrated a robust safety profile at the highest planned dose level, exhibiting no serious adverse events and featuring an appropriate range of plasma exposure levels post-oral administration. These milestones significantly bolster GT-02287’s potential to become a best-in-class therapy for Parkinson’s disease linked to GBA1 mutations.
Adding to this stream of positive developments, Gain Therapeutics has announced the acceptance of a late-breaking abstract for a poster presentation at the prestigious Federation of European Neuroscience Societies (FENS) Forum 2024. The new data to be presented highlights GT-02287’s ability to improve cognitive performance in a preclinical model of GBA1 Parkinson’s disease. This preclinical evidence showcases the broader therapeutic potential of GT-02287 beyond its direct effects on motor symptoms.
We are thrilled to report the successful completion of the MAD portion of our Phase 1 study with GT-02287, stated Eric Richman, CEO of Gain Therapeutics. These findings, combined with our earlier SAD data and the promising cognitive improvements observed in preclinical models, greatly reinforce our confidence in the therapeutic potential of GT-02287 for patients suffering from GBA1 Parkinson’s disease. We look forward to sharing further detailed results at the FENS Forum 2024.
GT-02287 is designed to specifically address the enzymatic dysfunction caused by GBA1 mutations, which are implicated in both sporadic and genetic forms of Parkinson’s disease. By targeting and modulating the GCase enzyme, GT-02287 aims to mitigate disease progression and improve cognitive and motor functions, which are severely compromised in affected individuals.
The positive outcomes from the Phase 1 study pave the way for subsequent clinical trials that will further explore the efficacy and safety of GT-02287 in a broader population of Parkinson’s disease patients. Gain Therapeutics is committed to advancing GT-02287 through its clinical development pipeline with the goal of providing a novel, effective treatment option for the Parkinson’s community.
About Gain Therapeutics, Inc.’
Gain Therapeutics, Inc. is a biopharmaceutical company focused on discovering and developing novel therapeutics using its proprietary See-Tx platform. Gain is at the forefront of designing allosteric small molecule candidates that aim to address unmet medical needs across a spectrum of diseases, including neurological disorders like Parkinson’s disease.
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