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Gene Mack, President and CEO of Gain Therapeutics. Image / Gain Therapeutics
 28.03.2025

Gain Therapeutics announces the administration of its drug GT-02287 to first patient in phase 1b clinical trial for Parkinson’s disease

The clinical-stage biotechnology company, Gain Therapeutics, has announced the administration of the first dose to a participant with Parkinson’s disease in its phase 1b clinical trial of GT-02287, its leading allosteric small molecule in development for the treatment of this disease. The Swiss company, a leader in the discovery and development of the next generation of small molecule allosteric therapies, has its R&D department at the Barcelona Science Park.

It is the leading drug candidate from Gain Therapeutics, currently in clinical development for the treatment of Parkinson’s disease, with or without a mutation in the GBA1 gene, the main genetic risk factor for developing the disease. GT-02287 is a small molecule that is administered orally and can penetrate the brain. It acts as an allosteric modulator of the enzyme glucocerebrosidase (GCase), restoring its function when it is disrupted by mutations in the GBA1 gene or by age-related stress factors.

In preclinical studies, GT-02287 helped restore the GCase enzyme, reduced inflammation and the accumulation of damaged proteins in the brain, and improved movement and memory. Additionally, it also decreased NfL levels in plasma, a biomarker of neurodegeneration. These results suggest that GT-02287 could help slow down or halt the progression of Parkinson’s disease, both in cases with a genetic mutation and in forms with no known cause.

“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 disease. We look forward to continued enrollment and anticipate an interim analysis from the towards the end of 2Q 2025,” says Gene Mack, President and CEO of the company.

The phase 1b clinical trial, open and conducted at multiple centers, aims to evaluate the safety and tolerability of GT-02287 in individuals with GBA1-associated Parkinson’s disease and idiopathic Parkinson’s disease, also analyzing pharmacokinetics, GCase modulation, and biomarkers in plasma and cerebrospinal fluid. The study will include up to 20 participants who will receive the treatment for three months, with interim data expected by the end of the second quarter of 2025.

The company’s main program has received financial support since the early stages of its development from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) and The Silverstein Foundation for Parkinson’s with GBA, as well as from the joint Eurostars-2 program, with co-financing from the European Union through the Horizon 2020 program and from Innosuisse – Swiss Innovation Agency.

» Link to the news: GAIN Therapeutics website [+]