On March 18, 2026, Gain Therapeutics, Inc. (Nasdaq: GANX) announced significant findings from its Phase 1b clinical study of GT-02287 during an oral presentation and poster session at the AD/PD™ 2026 International Conference on Alzheimer’s and Parkinson’s Disease and Related Neurological Disorders in Copenhagen, Denmark. The presentation highlighted new clinical and biomarker data supporting the disease-modifying potential of GT-02287 in both idiopathic and GBA1 Parkinson’s disease. The study involved 19 participants, with 16 opting to continue into a nine-month extension phase, indicating strong tolerability of the treatment. Notably, the data revealed a substantial decrease in glucosylsphingosine (GluSph) levels in cerebrospinal fluid (CSF) after 90 days of treatment, which is a promising indicator of the drug's efficacy. Furthermore, the study's preliminary results suggest that participants with elevated baseline GluSph levels experienced greater benefits compared to those with lower levels, as evidenced by a notable difference in MDS-UPDRS scores. The company also showcased preclinical data from a new series of allosteric glucocerebrosidase (GCase) modulators, led by GT-04686, which are poised for IND-enabling studies for treating Parkinson’s disease and other neurological disorders. Gene Mack, CEO of Gain Therapeutics, expressed optimism about the evolving biomarker evidence, emphasizing the potential of GT-02287 to shift the treatment paradigm towards disease modification rather than mere symptomatic relief. The ongoing study is expected to yield further data throughout the year, with the next presentation anticipated at upcoming scientific conferences.

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