Alterity Therapeutics has disclosed positive findings from its ATH434 Phase 2 clinical trial, presented at the 2025 International Congress of Parkinson’s Disease and Movement Disorders. According to the company, the open-label study, dubbed ATH434-202, involved 10 participants with advanced multiple system atrophy (MSA) treated over 12 months with 75 mg twice daily.
The company said that the data demonstrated reductions in disease progression relative to historical controls and showed consistent changes across clinical and neuroimaging endpoints. In particular, Alterity claims that the modified Unified MSA Rating Scale (UMSARS) scores improved or stabilized in some participants and that biomarkers and imaging indicated slowed brain atrophy and reduced iron accumulation in affected brain regions.
Alterity noted that no new safety signals emerged and that ATH434 was generally well tolerated over the study period. The firm said that these results support the continued development of ATH434 for MSA and potential Parkinsonian disorders more broadly.
The company also highlighted that ATH434 has already shown efficacy in an earlier randomized, double-blind Phase 2 trial in less advanced MSA (ATH434-201), where it met key functional and biomarker endpoints. Alterity holds that the consistency between the two trials strengthens the case for advancing ATH434 toward regulatory engagement and possible future pivotal trials.
The firm said it plans to engage with authorities, including the U.S. Food and Drug Administration, to determine the path forward for ATH434’s development in rare neurodegenerative disorders.
