Target enrollment complete in Phase 1b trial of GT-02287
Recruiting continues in Australia as original goal reached ahead of plan
Target enrollment for a Phase 1b clinical study assessing the safety and tolerability of GT-02287 in people with Parkinson’s disease is complete, three months ahead of the original goal, the therapy’s developer said.
After enrolling the first 16 participants, the Phase 1b clinical trial (NCT06732180) is recruiting up to 20 adults, ages 30-85, at several sites in Australia until the end of July. Enrolled participants must have been diagnosed with Parkinson’s within the past seven years, regardless of whether their condition is associated with mutations in the GBA1 gene.
Gain Therapeutics said it’s also planning to ask health authorities in Australia to extend the dosing period, which started in March, beyond the 90 days currently allowed, to better inform the design of a Phase 2 trial.
“The faster-than-anticipated enrollment means all those participants will reach their 90-day visit in time to facilitate biomarker analysis of all CSF [cerebrospinal fluid] samples taken by fourth quarter of this year instead of the first quarter of 2026,” Gene Mack, president and CEO of Gain Therapeutics, said in a company press release. The CSF is the liquid that surrounds and protects the brain and spinal cord.
Mutations in the GBA1 gene, which encodes for the GCase enzyme needed to break down fatty molecules and recycle cellular components, are known to increase the risk of Parkinson’s disease.  When the gene is mutated, the enzyme loses activity, setting the stage for the toxic accumulation of misfolded proteins like alpha-synuclein, a hallmark of Parkinson’s.
Potential to slow, stop disease progression
GT-02287 is an oral small-molecule allosteric enzyme activator, meaning it is designed to bind to GCase and restore its activity. This is expected to prevent the formation of misfolded alpha-synuclein clumps that damage nerve cells and contribute to Parkinson’s symptoms.
Preclinical data have shown that GT-02287 restored GCase activity and reduced the aggregation of alpha-synuclein, nerve cell death, and the blood levels of neurofilament light chain, a biomarker of neurodegeneration. In a mouse model of Parkinson’s disease associated with GBA1 mutations, GT-02287 rescued motor and cognitive performance, indicating it may have the potential to slow or stop disease progression.
In a Phase 1 trial involving healthy volunteers, GT-02287 was found to be safe, well tolerated, and capable of entering the brain, a critical requirement for treating neurological symptoms of Parkinson’s. The treatment also increased GCase activity by approximately 53% compared with a placebo.
The Phase 1b trial is an open-label study in which all participants receive GT-02287 orally, once daily, at a dose of 13.5 mg/kg, for 90 days (about three months). While the trial’s main goal is to assess the treatment’s safety and tolerability, outcome measures also include GT-02287’s pharmacological properties and its effects on GCase activity and other biomarkers in the blood and CSF over three months of treatment.
Participants must have either never taken medications for Parkinson’s or have been on a stable treatment regimen for at least three months. They must also show no severe motor fluctuations (when symptoms are not well controlled) or dyskinesia (uncontrolled movements).
“We are grateful to the patients and clinicians whose strong interest in novel, potentially disease-modifying treatments for Parkinson’s Disease, together with previous evidence of GT-02287 target engagement from our Phase 1 healthy volunteer study, led to completing enrollment of the study before the end of the summer as originally planned,” Mack said.
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