Gain Therapeutics has announced positive topline results from the Phase I clinical trial of GT-02287, a drug candidate targeting Parkinson’s disease.
The trial assessed the safety, tolerability, and pharmacokinetics of GT-02287 for the potential treatment of Parkinson’s.
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It included 72 healthy male and female volunteers aged up to 64 years.
The company previously reported that no discontinuations or serious adverse events were observed at the conclusion of the trial.
The unblinded data review post-database lock confirmed that GT-02287 was found to be safe and well-tolerated at all dose levels, including the highest planned doses.
GT-02287’s presence in the cerebrospinal fluid (CSF) indicated central nervous system (CNS) exposure and peripheral target engagement.
The favourable safety and tolerability profile of GT-02287 at oral doses that achieved therapeutic plasma levels, CNS exposure, and target engagement enhances its potential as a leading treatment for Parkinson’s disease, whether or not patients have a GBA1 mutation.
A brain-penetrant small molecule, the drug acts as an allosteric protein modulator that restores the function of the lysosomal enzyme glucocerebrosidase (GCase), which is commonly impaired in Parkinson’s disease due to mutations in the GBA1 gene or age-related stress factors.
In preclinical models of Parkinson’s disease, GT-02287 has shown potential by restoring GCase enzymatic function, reducing α-synuclein aggregation, neuroinflammation, and neuronal death.
Gain Therapeutics chief medical officer Jonas Hannestad said: “On the heels of this data, we expect to initiate a trial in people with Parkinson’s disease by Q4 2024 with the goal of demonstrating safety and tolerability in patients with Parkinson disease and to obtain proof of mechanism based on relevant biomarkers.
“We anticipate having data from Parkinson’s disease patients by mid-point 2025.”
In October last year, the company dosed the first two subjects in a Phase I clinical trial of GT-02287 to treat GBA1 Parkinson’s disease.
