Alterity Therapeutics Presents New Data on Multiple System Atrophy, a Rare Parkinsonian Disorder

Posted: 4 September 2023

Alterity Therapeutics (ASX: ATH, NASDAQ: ATHE) (“Alterity” or “the Company”), a biotechnology company dedicated to developing disease modifying treatments for neurodegenerative diseases, today announced that presentations from its bioMUSE natural history study of Multiple System Atrophy (MSA) were delivered at the International Congress of Parkinson’s Disease and Movement Disorders (MDS) taking place August 27 – 31, 2023 in Copenhagen, Denmark.

The posters presented from Alterity’s bioMUSE study address the need for incorporating biomarkers as a critical component for diagnosis of MSA. The diagnosis of early MSA can be challenging as individuals often present similarly to Parkinson’s disease (PD). In contrast to PD, MSA is rapidly progressive and, therefore, it is vital to accurately diagnose patients enrolling in clinical trials.

“The approach of using a diverse set of biomarkers to augment clinical criteria for MSA will greatly improve the diagnosis of this devastating disease,” said David Stamler, M.D., Chief Executive Officer of Alterity. “Based on our collaboration with the clinical and neuroimaging experts from Vanderbilt, we are in a unique position to implement this strategy in our ATH434-201 Phase 2 clinical trial. Our unique protocol design is helping ensure we are enrolling the right patient population thus giving ATH434 the best chance at success.”

Daniel O. Claassen, M.D., M.S., Professor of Neurology, Vanderbilt University Medical Center, added, “As with any disease, accurate diagnosis is critical to provide the best treatment options for patients, and because MSA is a rare, rapidly progressing disease, timing is of the essence. Diagnosis of early-stage MSA is vital for maximizing neuronal preservation with disease modifying therapies, and thus identifying biomarkers for early pathology is critical. Our findings presented this week support the use of specialized MRI techniques and fluid biomarkers to improve the specificity of MSA diagnosis as well as assess clinical measures of disease severity and treatment response in MSA.”

Two poster presentations were given at the MDS Congress.

The poster entitled, “A multimodal approach for diagnosis of early Multiple System Atrophy” was presented by Dr. Claassen. The analysis describes three clinically probable MSA patients with divergent MRI and fluid biomarker data, supporting the use of biomarkers to improve diagnostic accuracy in early MSA. The presented cases demonstrate that no single biomarker can be relied upon to aid in the diagnosis of early MSA. In addition, divergent clinical and biomarker findings in this case series suggests a multimodal clinical-biomarker approach is required for accurate diagnosis of clinically probable or early MSA. These examples support application of clinical and quantitative biomarkers in clinical trials evaluating disease-modifying treatments for early MSA.

The poster entitled, “Preliminary evidence for evolution of myoinositol and N-acetylaspartate as biomarkers of disease severity in early-stage Multiple System Atrophy” was presented by Paula Trujillo Diaz, PhD, Research Assistant Professor, Department of Neurology, Vanderbilt University Medical Center. The study assessed 13 early-stage MSA patients (motor symptoms ≤ 3 yrs) with diagnosis based on clinical parameters, fluid biomarkers, and quantitative MRI for iron deposition. The investigators then applied a non-invasive MRI technique known as magnetic resonance spectroscopy (MRS) that allows metabolite quantification in the brain, including myoinositol (mI; a marker of gliosis) and N-acetylaspartate (NAA; a marker of neuronal integrity). The results suggest that an increase in mI/water and decrease in NAA/water decrease over one-year in patients with MSA is consistent with MSA pathology. The findings suggest that metabolite concentration by MRS may be useful biomarkers for assessing clinical measures of disease severity and treatment response in MSA.

The poster presentations can be accessed on the Published Scientific Research section of the Alterity website here.

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