First Parkinson’s patient dosed in GT-02287 clinical trial
Study is recruiting adults with idiopathic disease or GBA1 gene mutation
The first patient has been dosed in Gain Therapeutics‘ clinical trial of GT-02287, an investigational, oral small molecule being developed for treating Parkinson’s disease.
With several locations open in Australia, the Phase 1b clinical trial (NCT06732180) is still recruiting up to 20 adults, ages 30-85, who’ve been diagnosed with Parkinson’s for less than seven years and have either idiopathic disease, that is, of unknown cause, or carry known mutations in the GBA1 gene. Interim data from the study is expected later this year.
“Initiation of dosing in our Phase 1b clinical trial represents an important step in the clinical development of GT-02287 and in Gain’s mission to deliver a disease-modifying therapy to people with Parkinson’s,” Gene Mack, Gain’s president and CEO, said in a company press release.
Mutations in the GBA1 gene are among the most common genetic risk factors for Parkinson’s disease. This gene codes for GCase, an enzyme that breaks down fatty molecules for recycling. When the gene is mutated, the enzyme loses activity, causing a buildup of toxic deposits, including those formed by misfolded proteins like alpha-synuclein.
GT-02287 is designed to bind to the GCase enzyme, restoring its activity. This should prevent misfolded alpha-synuclein from forming the toxic deposits that damage nerve cells in the brain that lead to symptoms of Parkinson’s. Preclinical data suggest GT-02287 may slow disease progression.
Testing safety, tolerability of GT-02287
This stage of GT-02287’s clinical development follows a Phase 1 trial in healthy volunteers where the drug reached expected levels in the cerebrospinal fluid, showing its ability to enter the brain. It was also found to be safe and well tolerated.
In the trial, which involved 72 healthy adults, GT-02287 increased GCase activity by roughly 53% over a placebo, as measured in dried blood spots, indicating the molecule engaged with the enzyme and partially restored its activity.
The ongoing Phase 1b clinical trial is testing the safety and tolerability of GT-02287 when taken daily for three months by adults with Parkinson’s. All the participants will receive GT-02287 at 13.5 mg/kg per day. The study’s secondary goals include measures of pharmacokinetics, or how GT-02287 moves into, through, and out of the body. Other goals include GT-02287’s effects on GCase activity and other biomarkers in the blood and cerebrospinal fluid over three months of treatment.
To be eligible, patients must have never taken medications for Parkinson’s or have been on a stable treatment for at least three months, with the same dose for at least four weeks before screening. They also must show no severe motor fluctuations, when symptoms aren’t well controlled, or severe uncontrolled movements, called dyskinesia.
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