With new FDA green light for developer, Parkinson’s clinical trial to launch soon

The U.S. Food and Drug Administration (FDA) has given Gain Therapeutics a green light to launch a clinical trial — to run across three continents — testing GT-02287, an oral therapy designed to prevent the formation of toxic protein clumps in Parkinson’s disease.

The agency clearance follows positive early results from studies involving both healthy volunteers and people with Parkinson’s. The data showed biomarker and clinical evidence of GT-02287’s activity, with a favorable safety profile.

The Phase 2 trial is planned to start between July and September, at sites in the U.S., Australia, and Europe, Gain stated in a company press release announcing the FDA’s authorization of an Investigational New Drug (IND) application for GT-02287 that clears the study’s start.

“The FDA’s decision is a significant milestone for Gain and we believe it validates the extensive preclinical and clinical work supporting further development of GT-02287,” said Gene Mack, Gain’s president and CEO, noting that, “in clinical studies to date, GT-02287 demonstrated target engagement with favorable safety and tolerability.”

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Toxic protein clumps damage brain blood vessels in Parkinson’s: Study

Mutations in the GBA1 gene are among the most common genetic risk factors for Parkinson’s. These mutations reduce the activity of glucocerebrosidase (GCase), an enzyme that helps break down cellular waste. As a result, harmful substances and misfolded proteins, such as alpha-synuclein and tau, accumulate in the brain, contributing to the development and progression of Parkinson’s.

In earlier trial, GT-02287 improved daily living for Parkinson’s patients

An oral small molecule, GT-02287 is designed to bind to GCase and restore its activity, thereby preventing the formation of toxic protein clumps and slowing the progression of Parkinson’s.

Preclinical studies demonstrated that the treatment slowed Parkinson’s progression and improved motor function and coordination in mouse models of Parkinson’s with or without GBA1 mutations. In the animals, improvements were seen in cognition and activities of daily living.

Treatment also reduced disease biomarkers, including alpha-synuclein clumping and blood levels of neurofilament light chain, a marker of neurodegeneration.

In a Phase 1 clinical trial that enrolled 73 healthy volunteers, the treatment was found to be generally safe and well tolerated. At a daily dose of 7.7 mg/kg and higher, it reached therapeutic blood levels and was detected in the cerebrospinal fluid (CSF), the liquid that surrounds the brain and spinal cord. That showed the therapy could reach the brain.

Additional results from an open-label Phase 1b trial (NCT06732180) showed that GT-02287 improved motor function and daily living activities over three months in people with Parkinson’s, with or without GBA1 mutations.

We believe GT-02287 represents the next generation of differentiated therapeutics designed to address the underlying biology of Parkinson’s disease and move beyond symptomatic relief to create a new backbone of treatment that can slow or stop disease progression.

During the ongoing extension study, the treatment was well tolerated and associated with improvements in motor and nonmotor symptoms, including gait, sleep, and smell. Use of the therapy also reduced levels of glucosylsphingosine (GluSph), a fatty molecule that accumulates when GCase is not functioning properly. Drops of 81% on average were seen in patients with elevated levels at the beginning of the study.

“We believe GT-02287 represents the next generation of differentiated therapeutics designed to address the underlying biology of Parkinson’s disease and move beyond symptomatic relief to create a new backbone of treatment that can slow or stop disease progression,” Mack said.