Clinical Trial of GT-02287 by Gain Therapeutics Extended for Parkinson's Disease Patients
In a significant development for the treatment of Parkinson's disease (PD), Gain Therapeutics, Inc., a clinical-stage biotechnology company, has announced the start of a Phase 1b extension study for its lead drug candidate, GT-02287.
GT-02287, an orally administered, brain-penetrant small molecule, acts as an allosteric enzyme modulator, restoring the function of the lysosomal enzyme glucocerebrosidase (GCase). In preclinical models of PD, GT-02287 has shown to reduce ER stress, lysosomal and mitochondrial pathology, aggregated α-synuclein, neuroinflammation and neuronal death, as well as plasma neurofilament light chain (NfL) levels, a biomarker of neurodegeneration.
The Phase 1b study, which enrolled 21 participants, is expected to complete in December 2025. Early data from the study will be presented on October 7, 2025, at the International Congress of Parkinson's Disease and Movement Disorders in Honolulu, HI. The poster presentation will be titled "GT-02287 in Parkinson's Disease: Interim Data from a Phase 1b study" and will have E-Poster Number 891.
Results from a Phase 1 study of GT-02287 in healthy volunteers showed favorable safety and tolerability, plasma and CNS exposures in the projected therapeutic range, and target engagement with a >50% increase in GCase activity among those receiving GT-02287 at clinically relevant doses.
The Phase 1b study's primary endpoint is to evaluate safety and tolerability after 3 months of dosing in people with Parkinson's disease. Encouragingly, more than half of the participants have agreed to continue treatment.
Gain Therapeutics' unique approach enables the discovery of novel, allosteric small molecule modulators that can restore or disrupt protein function, accelerating drug discovery and unlocking novel disease-modifying treatments for untreatable or difficult-to-treat disorders including neurodegenerative diseases, rare genetic disorders, and oncology.
The company's lead program in Parkinson's disease has received funding support early in 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 Eurostars-2 joint program with co-funding from the European Union Horizon 2020 research and Innosuisse - Swiss Innovation Agency.
Additional 90-day analysis from participants enrolled as of June 30, 2025, including functional changes scored according to the Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) and biomarker data from cerebrospinal fluid and blood, will be available in Q4 2025.
The clinic researchers involved in the study include Dr. Andrew Siderowf and Dr. Tanya Simuni, and the presentation of the early results will take place at the American Academy of Neurology (AAN) Annual Meeting in Boston.
The International Parkinson and Movement Disorder Society (MDS) holds its International Congress of Parkinson's Disease and Movement Disorders annually, providing a platform for the latest research and advancements in the field.
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