Announcement • Jul 07

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.

Alterity Therapeutics Limited Stock Price

ATH Share Price Performance

ATH Community Narratives

Trending Discussion

Recently Updated Narratives

Snowflake Analysis

Alterity Therapeutics Limited Key Details

About ATH

Recent ATH News & Updates

Price target increased by 44% to AU$3.28

Alterity Therapeutics Announces Granting Of New U.S. Composition Of Matter Patent For ATH434

Alterity Therapeutics Limited Receives FDA End-Of-Phase 2 Meeting Minutes Confirming Registrational Pathway For ATH434 In Multiple System Atrophy

Recent updates

Alterity Therapeutics Limited Competitors

Racura Oncology

Clinuvel Pharmaceuticals

Tetratherix

Starpharma Holdings