Gain Therapeutics Launches MAD Phase in GT-02287 Clinical Trial for GBA1 Parkinson's

Gain Therapeutics, a biotech firm listed on Nasdaq, has commenced the Multiple Ascending Dose (MAD) phase of its Phase 1 clinical trial for GT-02287, a potential treatment for GBA1 Parkinson's disease. The decision to proceed was endorsed by the Bellberry Human Research Ethics Committee in Australia, following satisfactory safety and tolerability outcomes from the Single Ascending Dose (SAD) phase. The company's CEO, Matthias Alder, confirmed that the trial is progressing as scheduled and is expected to conclude in the second quarter of the year.
GT-02287 is designed to rectify the malfunction of the lysosomal enzyme glucocerebrosidase (GCase), which is linked to the GBA1 gene mutation, a prevalent genetic risk factor for Parkinson's. Promising preclinical results from February 2024 indicated that GT-02287 could fully restore motor functions and normalize levels of the neurodegeneration biomarker NfL. This suggests the drug may slow or halt the progression of the disease.
The drug candidate, GT-02287, is an orally administered, brain-penetrating small molecule that modulates the function of misfolded GCase. It has demonstrated the ability to reduce α-synuclein aggregation, neuroinflammation, and neuronal death, while also increasing dopamine levels and improving motor function. The drug has also shown to decrease NfL levels, a biomarker for neurodegeneration.
GBA1 Parkinson's disease, which affects up to 15% of Parkinson's patients, is characterized by early onset and rapid symptom progression. The current standard of care addresses only the symptoms without impacting the disease's progression, highlighting the need for innovative treatments like GT-02287.
Gain Therapeutics utilizes an AI-driven platform, Magellan™, to identify new allosteric binding sites on proteins involved in diseases, leading to the discovery of novel small molecule modulators. The company is focused on developing next-generation allosteric therapies for a range of disorders, including neurodegenerative diseases and rare genetic disorders.

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