News & Events zu Alterity Therapeutics Limited (ATHE)ATHEAlterity Therapeutics Limited

Alterity's ATH434 50mg Significantly Slows Functional Decline in Multiple System Atrophy

Alterity announced additional analyses from its randomized, double-blind, placebo-controlled ATH434-201 Phase 2 trial in Multiple System Atrophy were presented at the 2026 International Congress of Parkinson's Disease and Movement Disorders in Seoul. After adjustment for baseline cerebrospinal fluid neurofilament light chain, ATH434 50 mg significantly slowed functional decline on the 11-item UMSARS Part I at Week 52 with a -4.64-point difference versus placebo in change from baseline, or about a 52% slowing of decline, roughly threefold the 1.5-point minimal clinically important difference. The 75 mg dose slowed decline with a -2.81-point difference versus placebo at Week 26 and Week 52. Higher baseline cerebrospinal fluid neurofilament light chain predicted greater decline at Week 52, with each 1,000 pg/mL increase associated with about 0.9 additional points of worsening on UMSARS I. Brain iron MRI by quantitative susceptibility mapping showed iron signal in the dentate nucleus increased versus placebo at Week 52 for the 50 mg dose by +0.016 ppm. The trial primary endpoint of change in substantia nigra iron at Week 52 was not met. "When we account for differences in baseline disease severity using CSF NfL, ATH434 50 mg significantly slowed functional decline by more than 50% compared with placebo over one year, a treatment effect well above the threshold considered clinically meaningful," said David Stamler, CEO.

Alterity Therapeutics Granted New Patent for ATH434

Alterity Therapeutics announced that the United States Patent and Trademark Office 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, 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.

Alterity Therapeutics Receives FDA Meeting Minutes Confirming ATH434 Phase 3 Trial

Alterity Therapeutics announced that it has received the official meeting minutes from its End-of Phase-2 meeting for ATH434 in Multiple System Atrophy from the FDA. The minutes confirm the elements of the registrational Phase 3 program previously announced on June 9 and the path toward a potential new drug application filing. The 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 I1 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, the Orthostatic Hypotension Symptom Assessment and the Clinical Global Impression of Severity. 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.