Alterity Therapeutics (ASX:ATH, NASDAQ:ATHE) has announced the completion of its last patient visit in the ATH434-202 open-label Phase 2 clinical trial for multiple system atrophy (MSA), a rare and rapidly progressive Parkinsonian disorder.
The Melbourne- and San Francisco-based biotech is developing ATH434, an oral disease-modifying drug candidate aimed at treating MSA and related neurodegenerative diseases. The ATH434-202 study follows a previously reported Phase 2 double-blind, placebo-controlled trial, which delivered positive results in patients with early-stage MSA.
In this follow-on study, 10 individuals with advanced MSA received a 75mg daily dose of ATH434 for 12 months. The trial aims to evaluate safety, target engagement, and the impact of the treatment on biomarkers such as brain volume, iron deposition, and aggregating a-synuclein—key contributors to MSA pathology.
CEO Dr David Stamler said the data would help guide future development of ATH434: “The 202 study gives us the opportunity to evaluate the effects of ATH434 treatment in a population that faces severe challenges due to the stage of their illness.”
Topline data from the trial is expected mid-2025.
ATH434 has Orphan Drug Designation for MSA in both the US and Europe and has shown promise preclinically in reducing a-synuclein aggregation and restoring brain iron balance. Alterity is continuing to advance a broader drug discovery platform targeting neurodegenerative diseases.
