Alterity Therapeutics Parkinson’s Disease and Multiple System Atrophy Data Featured at the American Academy of Neurology (AAN) 2024 Annual Meeting - Seite 2

Dr. Daniel Claassen, Professor of Neurology at Vanderbilt University Medical Center and coordinating investigator for the ATH434-201 Phase 2 study, commented, “The specialized neuroimaging and biomarker assessments evaluated and refined in the bioMUSE study were used to select and track patients in the Phase 2 study, making this program unique among current MSA clinical studies. It is vital to select study patients with a high degree of accuracy. The biomarkers being tested in the Alterity program hold promise for assessing the potential disease modifying benefits of ATH434.”

Presentation Summaries:

Title: A Phase 2 Study of ATH434, a Novel Inhibitor of α-Synuclein Aggregation, for the Treatment of Multiple System Atrophy
Lead Author: David Stamler, M.D., Chief Executive Officer of Alterity Therapeutics
Results: The poster describes the baseline characteristics for the 65 evaluable participants from Alterity’s ATH434-201 randomized, double-blind Phase 2 clinical trial, with a focus on baseline fluid biomarkers, neuroimaging and clinical data. The participants met the strict criteria designed to confirm that participants were diagnosed with early-stage MSA and had a mean of two years of motor symptoms. ATH434 is a potential disease modifying therapy based on its ability to redistribute excess labile iron without impairing normal iron storage, inhibit α‐synuclein aggregation and reduce oxidative stress. Importantly, the increased iron levels in the trial participants were evident in multiple subcortical brain regions with two distinct patterns of iron accumulations observed. In addition, MSA participants with less than four years of motor symptoms have elevated plasma Neurofilament Light Chain (NfL) levels at baseline which correlate significantly with disease severity.

Title: Neurofilament Light Chain and Clinical Progression in Early Multiple System Atrophy
Lead Author: Daniel O. Claassen, M.D., M.S., Professor of Neurology, Vanderbilt University Medical Center
Results: The poster describes results from the bioMUSE Natural History Study in which changes in clinical severity of 15 patients across a span of 12 months were compared with plasma biomarkers with a goal of establishing meaningful correlations. The advancement of MSA is profoundly aggressive, highlighting the critical need for biomarkers to delineate its progression over time. Emerging interest surrounds the use of the fluid biomarker NfL, found in both cerebrospinal fluid (CSF) and plasma, as an indicator of axonal damage in MSA. This fluid biomarker holds promise for measuring the extent of disease, tracking its progression, and forecasting the onset of clinical manifestations associated with MSA. In this observational study, the plasma NfL and CSF NfL were highly correlated, indicating that the more easily obtained plasma values have a meaningful relationship with brain pathology. Plasma NfL significantly increased over 12 months, and both plasma and CSF NfL were associated with disease progression in MSA. These data suggest that NfL may be a marker of disease modification in studies of MSA.