Gain Therapeutics to Present Long-Term Data on Rexaceract at Parkinson’s Congress
News related to:Gain Therapeutics, Inc · 2 min read
BETHESDA, Md., Sept. 29, 2026 /CourierPR/ -- Gain Therapeutics, Inc., a clinical-stage biotechnology company, will present long-term open-label data on its lead drug candidate, rexaceract, at the International Congress of Parkinson’s Disease and Movement Disorders in Seoul, Korea, from October 4 to 8, 2026.
The presentation will take place at E-Poster Station 12, located on the third floor of Hall D.
Rexaceract, formerly known as GT-02287, is an orally administered, brain-penetrant small molecule that acts as a beta-glucocerebrosidase (GCase) positive allosteric modulator. It aims to restore the function of the lysosomal enzyme GCase, which is often misfolded or impaired due to mutations in the GBA1 gene or other age-related stress factors. The modulator stabilizes and chaperones GCase to the lysosomes and mitochondria, thereby enhancing its function.
Preclinical studies have shown that rexaceract can restore GCase enzymatic function, reduce endoplasmic reticulum stress, lysosomal and mitochondrial pathology, and prevent the aggregation of α-synuclein. Additionally, it has been observed to reduce neuroinflammation and neuronal death, as well as lower plasma neurofilament light chain (NfL) levels, a biomarker of neurodegeneration. In rodent models of both GBA1-PD and idiopathic PD, rexaceract has been shown to rescue motor function and gait, and prevent the development of complex behavioral deficits.
The Phase 1 study of rexaceract in healthy volunteers demonstrated favorable safety and tolerability, with plasma and central nervous system (CNS) exposures within the projected therapeutic range. The drug also showed target engagement, as evidenced by an increase in GCase activity among those receiving clinically relevant doses.
Currently, rexaceract is being evaluated in a Phase 1b clinical trial for the treatment of Parkinson’s disease with or without a GBA1 mutation. The primary endpoint of the trial, which enrolled participants across seven sites in Australia, is to evaluate the safety and tolerability of rexaceract after three months of dosing in people with Parkinson’s disease. The Phase 1b study extension allows participants to continue to be treated with rexaceract for up to a total of 12 months.
Gain Therapeutics has received funding support for its lead program in Parkinson’s disease from The Michael J. Fox Foundation for Parkinson’s Research, The Silverstein Foundation for Parkinson’s with GBA, and the Eurostars-2 joint program with co-funding from the European Union Horizon 2020 research and Innosuisse, Swiss Innovation Agency.
The company’s unique approach involves the discovery of novel, allosteric small molecule modulators that can restore or disrupt protein function. Deploying its advanced Magellan™ platform, Gain is accelerating drug discovery and unlocking novel disease-modifying treatments for untreatable or difficult-to-treat disorders, including neurodegenerative diseases, rare genetic disorders, and oncology.