Alterity Therapeutics Presents ATH434-201 Phase 2 Data at the American Neurological Association Annual Meeting

Core Insights - Alterity Therapeutics announced positive results from the ATH434-201 Phase 2 clinical trial for Multiple System Atrophy (MSA), demonstrating clinically meaningful efficacy in modifying disease progression at both 50 mg and 75 mg doses [1][2][3] Company Overview - Alterity Therapeutics is a biotechnology company focused on developing disease-modifying treatments for neurodegenerative diseases, particularly MSA and Parkinson's disease [4][8] - The lead candidate, ATH434, is an oral agent designed to inhibit the aggregation of pathological proteins implicated in neurodegeneration, showing preclinical efficacy in reducing α-synuclein pathology and restoring normal iron balance in the brain [4][8] Clinical Trial Details - The ATH434-201 Phase 2 clinical trial was a randomized, double-blind, placebo-controlled study involving 77 adults, assessing the efficacy, safety, and pharmacokinetics of ATH434 over 12 months [5][6] - Results indicated that ATH434 produced clinically and statistically significant improvements on the modified Unified Multiple System Atrophy Rating Scale (UMSARS) Part I, with additional positive trends in motor performance and patient-reported outcomes [5][6] Efficacy and Safety - ATH434 demonstrated target engagement by reducing iron accumulation in MSA-affected brain regions, with both dose levels showing a favorable safety profile comparable to placebo [2][5] - The study reported no serious adverse events attributed to ATH434, reinforcing its tolerability [2][6] Regulatory Status - ATH434 has received Fast Track Designation and Orphan Drug Designation from the U.S. FDA and the European Commission for the treatment of MSA, highlighting its potential as a significant therapeutic option in a market with currently no approved disease-modifying treatments [4][8]