Announcement • 21h
Alterity Therapeutics Limited Receives FDA End-Of-Phase 2 Meeting Minutes Confirming Registrational Pathway For ATH434 In Multiple System Atrophy Alterity Therapeutics Limited has received the official meeting minutes from its End-of-Phase-2 (EOP2) meeting for ATH434 in Multiple System Atrophy (MSA) from the U.S. Food and Drug Administration (FDA). The minutes confirm the key elements of the registrational Phase 3 program previously announced on 9 June 2026 and the path toward a potential New Drug Application (NDA) filing. The EOP2 minutes confirmed that the FDA agreed with the proposed Phase 3 trial design, including the study population, treatment regimen, and efficacy endpoints. Alignment was reached on the selection and analysis of the primary endpoint the 11-item UMSARS Part I rating scale, a functional measure of activities of daily living affected in MSA. Agreement was also reached on selection of key secondary endpoints, including the Swallowing Disturbance Questionnaire (SDQ), the Orthostatic Hypotension Symptom Assessment (OHSA), and the Clinical Global Impression of Severity (CGI-S). The Phase 3 study is expected to enroll approximately 200 patients who will be randomized in a 1:1 ratio and treated with ATH434 50 mg or matching placebo twice daily for 12 months. The FDA further indicated that a single pivotal trial plus confirmatory evidence could provide the necessary data to support an approval of ATH434 for the treatment of MSA. Alterity expects that the data from its ATH434-201 Phase 2 clinical trial will provide the required confirmatory evidence. The FDA also indicated that the anticipated size of Alterity's safety database at the conclusion for the Phase 3 was reasonable. A single pivotal trial provides an efficient route to completion of the clinical development program and potential filing of an NDA, both in time and resources required. The Company also plans to offer an open label extension to participants who complete the Phase 3 trial to continue their treatment and enhance the safety database for ATH434. Alterity's lead candidate, ATH434, is an oral agent designed to reduce iron accumulation and inhibit abnormal protein aggregation associated with neurodegeneration. ATH434 has been shown to reduce -synuclein pathology and preserve neuronal function by restoring normal iron balance in the brain in preclinical models. As an iron chaperone, it has excellent potential to treat Parkinson's disease as well as various Parkinsonian disorders such as Multiple System Atrophy (MSA). Positive results from the randomized, double-blind, placebo-controlled Phase 2 clinical trial in patients with MSA demonstrated robust clinical efficacy, target engagement as indicated by key biomarkers, and a favorable safety profile. Positive data from a second Phase 2 open-label biomarker trial in patients with more advanced MSA reinforced these results. ATH434 has been granted Fast Track Designation by the U.S. Food and Drug Administration (FDA), and Orphan Drug Designation by the FDA and the European Commission for the treatment of MSA. 11-item UMSARS Part I (previously described as modified UMSARS I): Unified Multiple System Atrophy Rating Scale, 11-Items include: Orthostatic symptoms, Swallowing, Speech, Handwriting, Cutting food, Dressing, Hygiene, Walking, Falling, Urinary and Bowel function. New Risk • Jun 03
New major risk - Market cap size The company's market capitalization is less than US$10m. Market cap: AU$1.96m (US$1.40m) This is considered a major risk. Companies with a small market capitalization are most likely businesses that have not yet released a product to market or are simply a very small company without a wide reach. Either way, risk is elevated with these companies because there is a chance the product may not come to fruition or the company's addressable market or demand may not be as large as expected. In addition, if the company's size is the main factor, it is less likely to have many investors and analysts following it and scrutinizing its performance and outlook. Currently, the following risks have been identified for the company: Major Risks Share price has been highly volatile over the past 3 months (19% average weekly change). Earnings are forecast to decline by an average of 3.7% per year for the foreseeable future. Market cap is less than US$10m (AU$1.96m market cap, or US$1.40m). Minor Risks Currently unprofitable and not forecast to become profitable over next 3 years (AU$41m net loss in 3 years). Shareholders have been diluted in the past year (19% increase in shares outstanding). Revenue is less than US$5m (AU$6.6m revenue, or US$4.7m). 
