Alterity Therapeutics announced positive topline data from its open-label Phase 2 clinical trial (ATH434-202) of ATH434 in individuals with multiple system atrophy (MSA). The trial, which enrolled patients with more advanced MSA than the prior Phase 2 study, showed ATH434 slowed disease progression and stabilized key biomarkers. The drug was found to be well-tolerated with no serious adverse events related to the treatment.
This news is particularly important because MSA is a rare and rapidly progressive neurodegenerative disease with no currently approved disease-modifying therapies. The positive data from this trial, particularly in a more advanced patient population, strengthens the potential of ATH434 to address this unmet medical need and offer hope to patients suffering from this devastating disease. The confirmation of earlier findings in a more advanced patient cohort provides greater confidence in the drug’s potential efficacy across a broader spectrum of MSA.
The trial revealed that disease progression, as measured by the Unified MSA Rating Scale Part I (UMSARS I), was reduced by approximately half compared to historical controls over 12 months. Furthermore, 30% of participants reported stable neurological symptoms throughout the study. Neuroimaging biomarkers showed slowed brain atrophy in affected areas and lower iron accumulation, further validating ATH434’s target engagement. Importantly, these results align with the findings from the earlier Phase 2 double-blind trial (ATH434-201).
The positive results from the ATH434-202 trial reinforce the potential of ATH434 as a disease-modifying treatment for MSA. The data from both Phase 2 trials support further development and suggest a promising path towards regulatory approval. This progress could eventually lead to a much-needed treatment option for individuals with MSA, significantly impacting their quality of life and potentially altering the course of this debilitating disease.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
