Company: ATHE
Filing Date: 2025-08-29
Form Type: 20-F
Source: 0001213900-25-082027
Chunk: 53

Company: ALTERITY THERAPEUTICS LTD
Filing Date: 2025-08-29
Form: 20-F
Item: Item 4
Chunk 53
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434-201. These outcomes are potentially promising as stabilization of MSA symptoms is unexpected in this patient population.

bioMUSE natural history study for individuals with MSA 

Biomarkers of progression in Multiple system atrophy (bioMUSE) is a natural history study to track the progression of individuals with early MSA. The study was conducted in collaboration with Vanderbilt University Medical Center in the US under the direction of Daniel Claassen, MD, Professor of Neurology and Principal Investigator. Natural history studies are important for characterizing disease progression in target patient populations. The goal of the bioMUSE observational study was to optimize patient selection and choose endpoints in our Phase 2 clinical trials, and the data generated was invaluable in informing and reducing risk in these trials.

In May 2024, we hosted a webinar to discuss data from the bioMUSE Natural History Study. The study enrolled 21 individuals who were observed for 12 months to characterize early-stage MSA in terms of various biomarkers. In particular, the focus is on brain iron, brain volume, and the pathology in glial support cells. Utilizing novel MRI technology, Alterity’s partners at Vanderbilt have optimized specialized MRI methods, including machine learning (a form of artificial intelligence), to establish standardized methods to analyze brain iron and brain volumes with precision. Importantly, they developed a new, novel imaging biomarker to assess brain volume in MSA affected regions. The bioMUSE data showed a statistically significant increase in iron over 12 months in the substantia nigra, and statistically significant decreases in brain volume observed in affected regions at 12 months.

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Key data from bioMUSE have been presented at major medical and scientific meetings listed below. The study continues to generate important scientific data validating our state-of-the-art approach to utilizing various biomarkers to improve the accuracy of diagnosing MSA. Advanced MRI methods employed in the study, referred to as quantitative susceptibility mapping (QSM), have allowed us to measure iron accumulation in multiple areas of the brain affected in MSA patients. Similarly, standardized methods have been established to analyze brain volumes with precision.

Findings to date indicate that advanced MRI methods for measuring iron may improve patient selection in clinical trials of disease modifying therapy and have potential to serve as a biomarker for assessing treatment induced changes. Analysis also demonstrated that wearable sensors can quantify motor impairment in individuals with MSA that is not captured by neurological examination. This means that wearable sensors can be used to assess disease progression in clinical trials.

Multiple publications and presentations have been delivered on