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Highlights
- Last patient in ATH434-202 Phase 2 trial for advanced MSA has completed study.
- Trial evaluates the safety, efficacy, and target engagement of ATH434 in MSA patients.
- Topline data from the study is expected in mid-2025.
Alterity Therapeutics (ASX: ATH, NASDAQ: ATHE), a biotechnology company focused on developing disease-modifying treatments for neurodegenerative diseases, has announced the completion of clinical evaluations for the last patient in its ATH434-202 Phase 2 trial. This open-label study is designed to assess the safety, efficacy, and target engagement of the company’s drug candidate, ATH434, in patients with advanced multiple system atrophy (MSA).
The ATH434-202 trial involved 10 participants with advanced MSA, a progressive neurodegenerative disorder that severely impacts motor and autonomic functions. Participants received 75 mg doses of ATH434 for 12 months. The study’s primary objective is to evaluate the impact of treatment on brain volume, a key biomarker for MSA, and to assess other factors such as neuroimaging, protein biomarkers, and safety. These biomarkers, which include brain volume, iron levels, and aggregating α-synuclein, are central to the pathology of MSA and are critical for determining drug activity.
David Stamler, M.D., Chief Executive Officer of Alterity, expressed the company’s satisfaction with completing this important milestone in the study. He emphasized the significance of the data that will be generated from this open-label study, especially given its differences from the previously conducted randomized Phase 2 trial. Stamler also highlighted that the findings from the ATH434-202 trial will be crucial in guiding the next steps in the company’s development program for ATH434.
Alterity expects to report topline data from the ATH434-202 Phase 2 trial in mid-2025, which will provide valuable insights into the drug’s potential for treating advanced MSA. This trial marks a key step in Alterity's ongoing efforts to develop therapies for neurodegenerative diseases, particularly those with limited treatment options like MSA.






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