공시 • Mar 19
Gain Therapeutics Inc Presents Additional Clinical and Biomarker Data from Phase 1B Clinical Study of GT-02287 and Preclinical Data from Novel Chemical Series At AD/PD 2026
Gain Therapeutics, Inc. presented an oral presentation and poster at AD/PD 2026 International Conference on Alzheimer’s and Parkinson’s Disease and Related Neurological Disorders, being held March 17-21, 2026, in Copenhagen, Denmark. The oral presentation outlined new clinical and biomarker data from the Phase 1b clinical study of GT-02287 further supporting disease-modifying potential in both idiopathic and GBA1 Parkinson’s disease. The Company also presented a poster outlining preclinical data from a structurally distinct chemical series of allosteric glucocerebrosidase (GCase) modulators, which are ready for IND-enabling studies for the treatment of Parkinson’s disease (PD) and other neurological disorders. The oral presentation, entitled, “An Open-Label Phase 1b Study of GT-02287 in Parkinson’s Disease,” was delivered on-site by the Company’s Chief Medical Officer, Jonas Hannestad, M.D., Ph.D. Data on safety, tolerability, biomarkers, and clinical scores from the completed Part 1 of the study support continued development of GT-02287 for PD. Of the 19 participants who completed dosing in Part 1, 16 chose to continue in the ongoing nine-month extension (Part 2), further supporting the tolerability of GT-02287. A Data Monitoring Committee meeting on March 5, 2026, concluded that the study should continue without any changes. As previously presented in January 2026, in all participants with elevated baseline levels of glucosylsphingosine (GluSph) in cerebrospinal fluid (CSF), GluSph decreased substantially after 90 days of treatment with GT-02287. Elevated GluSph, a result of GCase dysfunction, has been shown to increase the aggregation of alpha synuclein and to impair mitochondrial and lysosomal function in neurons. As of March 10, 2026, of the 16 participants enrolled in the ongoing nine-month extension, 14 had completed dosing for five months (Day 150). MDS-UPDRS scores remained stable over 150 days of dosing. Preliminary data (subject to quality assurance) suggests that participants with elevated baseline levels of GluSph in CSF continued to benefit more than those with low baseline levels of GluSph in CSF, with a difference of 6.7 points in the sum of MDS-UPDRS Part II and Part III scores between the two groups at Day 150. Additionally, in individuals with high levels of CSF GluSph at baseline, levels of DOPA decarboxylase (DDC) decreased following 90 days of treatment with GT-02287. DDC is responsible for converting levodopa into dopamine in the brain and peripheral nervous system and is elevated in the CSF of people with Parkinson’s – likely due to dopaminergic neuron dysfunction. The poster, titled, “Novel Allosteric GCase Modulators, Different From the Clinical Stage GT-02287, for the Treatment of Parkinson’s Disease,” was presented on-site by the Company’s Head of Research Ana Maria Garcia-Collazo, Ph.D. The new, orally available, brain penetrant chemical series – led by GT-04686 – demonstrates potential for future clinical development, as evidenced by restoration of key biological activities that are impaired in Parkinson’s disease. These findings together support advancement of this novel chemical series towards the clinic and the potential to be developed for PD and other indications where GCase deficiency is implicated. Gain Therapeutics’ lead drug candidate, GT-02287, is in clinical development for the treatment of Parkinson’s disease (PD) with or without a GBA1 mutation. The orally administered, brain-penetrant small molecule is an allosteric enzyme modulator that restores the function of the lysosomal enzyme glucocerebrosidase (GCase) which becomes misfolded and impaired due to mutations in the GBA1 gene, the most common genetic abnormality associated with PD, or other age-related stress factors. In preclinical models of PD, GT-02287 restored GCase enzymatic function, reduced ER stress, lysosomal and mitochondrial pathology, aggregated a-synuclein, neuroinflammation and neuronal death, as well as plasma neurofilament light chain (NfL) levels, a biomarker of neurodegeneration. In rodent models of both GBA1-PD and idiopathic PD, GT-02287 was shown to rescue deficits in motor function and gait and prevent the development of deficits in complex behaviors such as nesting. Compelling data in these models, demonstrating a disease-modifying effect of GT-02287, suggest that the drug candidate may have the potential to slow or stop the progression of Parkinson’s disease. Results from a Phase 1 study of GT-02287 in healthy volunteers demonstrated favorable safety and tolerability, plasma and CNS exposures in the projected therapeutic range, and target engagement with an increase in GCase activity among those receiving GT-02287 at clinically relevant doses. GT-02287 is currently 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 GT-02287 after three months of dosing in people with Parkinson’s disease. The recently commenced Phase 1b study extension allows participants to continue to be treated with GT-02287 for up to a total of 12 months. Initial results from the Phase 1b clinical trial in people with Parkinson’s disease demonstrated central nervous system target engagement, a reduction to baseline levels in the prespecified endpoint glucosylsphingosine (GluSph), and improvement or stabilization in MDS-UPDRS scores. Gain’s lead program in Parkinson’s disease has been awarded 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.