Alterity Therapeutics presented data at the 2025 International MSA Congress, highlighting positive Phase 2 results for their drug ATH434 in treating Multiple System Atrophy (MSA). The company also unveiled the MSA Atrophy Index (MSAai), a new imaging biomarker, and presented research linking skin α-synuclein levels to orthostatic symptoms in MSA. This research focused on improving diagnosis, monitoring, and treatment of this debilitating neurodegenerative disease.
These findings are crucial for the MSA patient community currently lacking effective treatment options. The ATH434 data offers a potential breakthrough, demonstrating clinically meaningful improvements in daily living activities and key MSA symptoms. The MSAai provides clinicians with a more precise tool for diagnosing and tracking disease progression, aiding earlier intervention and potentially improving patient outcomes. The α-synuclein research enhances understanding of MSA progression and could pave the way for developing more targeted therapies and improved symptom management strategies.
The Phase 2 trial of ATH434 showed a statistically significant reduction in disease severity compared to placebo. Patients receiving the 50mg dose experienced a 48% relative treatment effect, demonstrating improvements in daily activities, symptom severity, and outpatient activity levels. The MSAai effectively differentiated MSA from other movement disorders and correlated with disease severity and progression. The study also confirmed that skin α-synuclein levels increase over time in MSA patients and correlate with the severity of orthostatic symptoms.
The positive Phase 2 data for ATH434 strongly positions Alterity to advance this potential treatment for MSA. The development of the MSAai as a reliable biomarker may lead to more efficient clinical trials and improved diagnostic accuracy in clinical practice. The ongoing research on α-synuclein could lead to a deeper understanding of MSA pathophysiology and potentially inform the development of new therapeutic targets.
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.
