Alterity Therapeutics to Present New Data on ATH434 at the World Orphan Drug Congress USA 2024

Core Viewpoint - Alterity Therapeutics is advancing its lead candidate ATH434, an oral agent targeting neurodegenerative diseases, with new data to be presented at upcoming medical conferences, highlighting its potential in treating conditions like Multiple System Atrophy (MSA) and Parkinson's disease [1][2][3]. Company Overview - Alterity Therapeutics is a clinical-stage biotechnology company focused on developing treatments for neurodegenerative diseases, particularly through its lead asset ATH434 [9]. - The company is based in Melbourne, Australia, and San Francisco, California, and is dedicated to creating innovative therapies for patients suffering from these conditions [9]. Product Information - ATH434 is designed to inhibit the aggregation of pathological proteins associated with neurodegeneration, showing preclinical efficacy in reducing α-synuclein pathology and restoring normal iron balance in the brain [3]. - The drug has completed Phase 1 studies, demonstrating good tolerance and achieving brain levels comparable to effective doses in animal models of MSA [3]. Clinical Trials - ATH434 is currently being evaluated in two Phase 2 clinical trials: ATH434-201, a randomized, double-blind, placebo-controlled trial for early-stage MSA, and ATH434-202, an open-label biomarker trial for more advanced MSA [3][4]. - The ATH434-201 trial involves 77 adults receiving either ATH434 or placebo over 12 months, focusing on neuroimaging and protein biomarkers to assess efficacy and safety [5]. Research and Development - The bioMUSE study, in collaboration with Vanderbilt University Medical Center, tracks MSA progression and informs the design of the ATH434-201 trial, providing critical data on early-stage MSA patients [6]. - The study has enrolled approximately 20 individuals with clinically probable or established MSA, contributing to the understanding of disease progression and biomarker selection [6]. Disease Context - Multiple System Atrophy (MSA) is a rare neurodegenerative disease affecting at least 15,000 individuals in the U.S., characterized by autonomic dysfunction and impaired movement, with no approved therapies to slow its progression [7]. - Parkinson's disease, another focus area for ATH434, affects nearly one million people in the U.S. and is marked by motor and non-motor symptoms due to the loss of dopamine-producing brain cells [8].