Announcement • Aug 13
Alterity Therapeutics Announces Granting Of New U.S. Composition Of Matter Patent For ATH434 Alterity Therapeutics announced that the United States Patent and Trademark Office (USPTO) has granted a new composition of matter patent for ATH434, the Company’s lead clinical asset. ATH434 is an oral agent designed to treat the underlying pathology of neurodegenerative diseases such as Multiple System Atrophy (MSA), Parkinson’s disease and related disorders. The newly granted patent represents a significant intellectual property milestone for Alterity and strengthens ATH434’s long-term commercial potential as the Company prepares to initiate Phase 3 trial activities in MSA by year-end 2026. The new patent provides composition-of-matter protection for a crystalline structure of the mesylate salt form of ATH434 as well as protection for methods for treating neurological conditions using it. This form of ATH434 was used in the Company’s Phase 2 clinical trials in MSA and will be utilized in the planned Phase 3 study. The patent extends protection for ATH434 to at least 2045, and together with existing intellectual property (IP) and regulatory designations, provides multi-layered market protection. In addition to reinforcing Alterity’s position in MSA, the new patent enables future development opportunities for ATH434 in Parkinson’s disease and other neurodegenerative diseases where iron dysregulation and protein aggregation are implicated. The USPTO granted the patent entitled "Crystalline Form, and Process for its Production" which covers the composition of matter of a crystalline form of ATH434 mesylate as well as methods for treating neurological conditions using it. The allowed claims protect the novel solid-state crystalline form of ATH434 mesylate and treatment methods, providing another layer of protection as a commercial asset for the treatment of MSA and additional neurodegenerative diseases. The patent has an estimated expiration date of at least 2045 and is expected to be listed in the FDA’s Approved Drug Products with Therapeutic Equivalence Evaluations publication (the “Orange Book”), upon regulatory approval of ATH434 for commercialization. Composition of matter claims are widely recognized as one of the strongest forms of pharmaceutical patent protection. ATH434 is an oral agent designed to reduce iron accumulation and inhibit abnormal protein aggregation associated with neurodegeneration. ATH434 has been shown to reduce a-synuclein pathology and preserve neuronal function by restoring normal iron balance in the brain in preclinical models. With properties shared by endogenous iron chaperones, it has the 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 clinically meaningful 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. 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. 
