Core Insights - Alterity Therapeutics presented significant findings on ATH434, demonstrating clinically meaningful efficacy in treating Multiple System Atrophy (MSA) during the 2025 International MSA Congress [1][2][3] Group 1: ATH434 Clinical Trial Results - The Phase 2 double-blind trial of ATH434 involved 71 patients, showing a 48% relative treatment effect at the 50 mg dose and a 30% effect at the 75 mg dose on the modified UMSARS I activities of daily living scale at 52 weeks [3][4] - ATH434 improved key symptoms of MSA, with statistically significant results on the Clinical Global Impression of Severity Scale (p=0.0088) and positive trends in the Orthostatic Hypotension Symptom Assessment [3][4][9] - Increased outpatient activity was observed with wearable sensors, indicating improvements in step count and total walking time compared to placebo [4][9] Group 2: MSA Atrophy Index (MSAai) - The MSA Atrophy Index (MSAai) was introduced as a novel imaging biomarker to enhance MSA diagnosis and monitoring, correlating with clinical presentation and tracking disease progression [5][6] - The bioMUSE study indicated that MSA patients had significantly lower MSAai values compared to healthy controls and other movement disorders, with strong correlations to UMSARS scores [5][6] Group 3: α-Synuclein Research Findings - The bioMUSE study revealed that higher concentrations of α-synuclein in skin biopsies were associated with greater burden of orthostatic symptoms, providing insights into disease progression [2][6] - Cutaneous α-synuclein deposition increased significantly over 12 months, with a mean increase from 6.59 to 7.71 (p=0.042) [6] Group 4: Company Overview and Future Directions - Alterity Therapeutics is focused on developing disease-modifying therapies for neurodegenerative diseases, with ATH434 being a lead candidate showing robust clinical efficacy and a favorable safety profile [7][12] - The company has received Fast Track Designation and Orphan Drug Designation from the U.S. FDA for ATH434, indicating its potential in treating MSA [7][12]