Alterity Therapeutics Presents Positive Data from ATH434 Phase 2 Trial at the 2025 International Congress of Parkinson’s Disease and Movement Disorders

Core Insights - The data from the ATH434-201 Phase 2 clinical trial indicates that ATH434 slows disease progression and stabilizes orthostatic hypotension in patients with Multiple System Atrophy (MSA) [1][2] - New analyses presented at the MDS conference enhance confidence in the trial results, particularly regarding the efficacy of the 75 mg dose of ATH434 [2][3] - Advanced neuroimaging and biomarker analysis improve understanding of MSA diagnosis and disease tracking [1][7] Company Overview - Alterity Therapeutics is focused on developing disease-modifying treatments for neurodegenerative diseases, particularly MSA [1][14] - The company has demonstrated clinically meaningful efficacy for ATH434 in a randomized, double-blind, placebo-controlled Phase 2 clinical trial [14] - ATH434 has received Fast Track Designation and Orphan Drug Designation from the FDA for the treatment of MSA [10][14] Clinical Trial Details - The ATH434-201 trial involved 77 adults, randomized to receive either 50 mg or 75 mg of ATH434 or a placebo, administered twice daily for 12 months [12] - Results showed a 48% relative treatment effect at the 50 mg dose and a 30% relative treatment effect at the 75 mg dose on the modified UMSARS I scale at 52 weeks [3][12] - The trial also indicated that ATH434 was well tolerated, with similar adverse event rates compared to placebo [6][12] Efficacy and Safety Findings - ATH434 demonstrated a clinically significant reduction in disease severity on the UMSARS I scale, with improvements in daily living activities [3][6] - The treatment showed beneficial effects on orthostatic hypotension symptoms, with patients in the placebo group worsening while those on ATH434 remained stable [5][6] - Neuroimaging data indicated reduced iron accumulation in brain regions affected by MSA, suggesting target engagement [6][10] Diagnostic Advancements - The trial utilized state-of-the-art neuroimaging and biomarkers to refine MSA diagnosis and track disease evolution across its clinical phenotypes [2][7] - A multimodal approach combining α-synuclein aggregation profiles with clinical and imaging data may enhance diagnostic accuracy in MSA [7][8] - The study found a 90% concordance between clinical classification and quantitative MRI assessments, highlighting the value of imaging in diagnosis [8]