Gain aims to prove Parkinson's drug potential in 2024 after phase 1 success

Gain Therapeutics recently presented promising phase 1 data for its Parkinson's disease therapy, GT-02287. The trial involved 72 healthy volunteers aged up to 64 years and evaluated single and multiple oral doses of the allosteric protein modulator. Notably, the study concluded that GT-02287 was safe and generally well tolerated at all dose levels, with no discontinuations or serious adverse events reported.

GT-02287 is designed to restore the function of the glucocerebrosidase enzyme, which is often impaired by mutations in the GBA1 gene, a common genetic anomaly associated with Parkinson's disease. The phase 1 trial data revealed that GT-02287 was present in the cerebrospinal fluid and showed peripheral target engagement. This data supports the potential of GT-02287 to be a leading treatment for Parkinson’s disease, irrespective of whether patients have a GBA1 mutation.

The favorable safety and tolerability profile at therapeutic plasma levels, along with central nervous system (CNS) exposure and target engagement, bolsters GT-02287’s candidacy for further development. Gain Therapeutics plans to initiate a phase 1b trial in Parkinson's patients by the end of 2024, aiming for results by mid-2025. This upcoming trial will focus on demonstrating the drug's efficacy through key biomarkers.

Earlier preclinical studies in June added to the hopeful outlook. Mice treated with GT-02287 built nests comparable to those of healthy mice, whereas untreated mice constructed poorly formed nests. Additionally, the treatment was shown to reduce plasma neurofilament light chain levels, a known biomarker for neurodegeneration.

Gene Mack, Gain’s chief financial officer and interim CEO, expressed enthusiasm about GT-02287’s promising profile, particularly its CNS exposure and target engagement. Mack emphasized the company's commitment to achieving near-term clinical milestones and advancing GT-02287 further in clinical studies, with the ultimate goal of improving life quality for people affected by Parkinson's disease.

GT-02287 is an allosteric modulator, meaning it binds to a protein's surface and alters the configuration of the protein's binding site. This drug class, although showing potential, has faced challenges in later-stage studies for Parkinson's disease. For instance, Sage Therapeutics’ NMDA receptor positive allosteric modulator dalzanemdor did not succeed in a phase 2 trial earlier this year. Similarly, Addex Therapeutics had to halt its phase 2b/3 trial for a metabotropic glutamate receptor subtype 5 negative allosteric modulator due to COVID-related recruitment issues in 2022.

Despite these setbacks in the field, Gain Therapeutics is optimistic about advancing GT-02287 based on its current data. The biotech firm is poised to push forward, aspiring to make a significant impact on Parkinson's disease treatment through ongoing and future clinical trials.

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