Alterity Therapeutics Completes Last Patient Visit in ATH434-202 Open-Label Phase 2 Trial in Multiple System Atrophy

Core Insights - Alterity Therapeutics is advancing its lead drug candidate, ATH434, aimed at treating neurodegenerative diseases, specifically targeting Multiple System Atrophy (MSA) [1][4] - The last patient in the ATH434-202 Phase 2 trial has completed the study, with topline data expected to be reported in mid-2025 [1][2] Company Overview - Alterity Therapeutics is a clinical-stage biotechnology company focused on developing disease-modifying therapies for neurodegenerative diseases, particularly Parkinson's disease and related disorders [7] - The company is based in Melbourne, Australia, and San Francisco, California, and has a drug discovery platform aimed at creating treatments for neurological diseases [7] ATH434-202 Phase 2 Clinical Trial - The ATH434-202 trial is an open-label study involving 10 participants with advanced MSA, where participants received a 75 mg dose of ATH434 for 12 months [3] - The study aims to evaluate the effects of ATH434 on neuroimaging and protein biomarkers, alongside clinical measures, safety, and pharmacokinetics [3] - The primary objective is to assess the impact of ATH434 on brain volume in a more advanced patient population compared to previous trials [3] Drug Mechanism and Efficacy - ATH434 is designed to inhibit the aggregation of pathological proteins associated with neurodegeneration, specifically targeting α-synuclein pathology [4] - Preclinical studies have shown that ATH434 can reduce α-synuclein pathology and preserve neuronal function by restoring normal iron balance in the brain [4] - The drug has received Orphan Drug Designation for MSA treatment from the U.S. FDA and the European Commission [4] Disease Context - Multiple System Atrophy (MSA) is a rare, rapidly progressive neurodegenerative disease affecting at least 15,000 individuals in the U.S., characterized by autonomic dysfunction and impaired movement [5] - Currently, there are no approved drugs that can slow the progression of MSA, highlighting the potential significance of ATH434 in the treatment landscape [5]