Gain Therapeutics(US:GANX)2025-10-14 21:00Summary of Gain Therapeutics Conference Call on October 14, 2025 Company Overview - Company: Gain Therapeutics (NasdaqGM: GANX) - Focus: Development of GT-022287 for Parkinson's disease Key Industry Insights - Parkinson's Disease Prevalence: Second most prevalent neurodegenerative disease in the U.S. with approximately 1 million patients [7] - Genetic Mutation: 10-15% of Parkinson's patients have a mutation in the GBA1 gene, which is the target for GT-022287 [7] - Mechanism of Action: GT-022287 stabilizes the enzyme glucocerebrosidase (GCase), improving its function in lysosomes and mitochondria, which is crucial for cellular health [9][10] Clinical Study Updates - Phase 1b Study: Ongoing in Australia, focusing on safety and tolerability of GT-022287 in Parkinson's patients [3][11] - Dosing Information: Patients treated for 90 days at a dose of 13.5 mg/kg, with an extension phase for an additional 9 months [11][12] - Enrollment: 21 participants enrolled, with 16 completing the initial 90-day study [12][45] - Adverse Events: 93 total adverse events reported, with 85% classified as mild [50] - Biomarker Engagement: Preliminary data shows a 53% increase in GCase activity [12] Biomarkers and Clinical Endpoints - Biomarker Importance: Biomarkers are critical for understanding disease progression and treatment efficacy [15][20] - Alpha-Synuclein: A key biomarker for Parkinson's disease, with ongoing efforts to quantify its presence [21][28] - MDS-UPDRS: The Unified Parkinson's Disease Rating Scale is used to assess clinical outcomes, with a focus on both self-reported and clinician-rated measures [33][36] Future Directions - Data Availability: Full data set from the 90-day study expected by the end of the year, including safety, tolerability, and biomarker data [58] - Phase 2 Study: Planned for Q2 of the following year, aiming to confirm the efficacy of GT-022287 with a placebo-controlled design [59][60] Additional Insights - GBA1 Mutation Impact: Patients with GBA1 mutations may experience a more aggressive form of Parkinson's disease [7][70] - Regulatory Considerations: There is a growing emphasis on the clinical meaningfulness of self-reported outcomes in regulatory assessments [38][39] - Long-Term Efficacy: The drug's potential to slow disease progression rather than provide immediate symptomatic relief is a key focus [75] Conclusion Gain Therapeutics is making significant strides in the development of GT-022287 for Parkinson's disease, with ongoing studies aimed at understanding its safety, tolerability, and biological impact on disease progression. The integration of biomarkers and clinical endpoints will be crucial in evaluating the drug's effectiveness in future trials.