Alterity Therapeutics today announced it has commenced enrolling patients with Multiple System Atrophy (MSA) in its bioMUSE Study in the United States.
BioMUSE is a natural history study that aims to track the progression of patients with MSA, a Parkinsonian disorder without approved therapy. The study is being conducted in collaboration with Vanderbilt University Medical Center in the US under the direction of Daniel Claassen, MD, Associate Professor of Neurology and Principal Investigator. Natural history studies are important for characterizing disease progression in selected patient populations. The study will provide vital information on early stage MSA patients to optimize the design of Alterity's Phase 2 study in MSA. The study will also inform the selection of biomarkers suitable to evaluate target engagement and preliminary efficacy.
Alterity's lead compound ATH434 has already successfully completed Phase 1 clinical trial and is advancing toward a Phase 2 clinical trial.
Dr. Claassen said: 'This is an important study to expand our understanding of MSA. We are enrolling early stage patients who stand to gain the most from disease modifying treatments. I look forward to working with Alterity on this project and I hope it can provide the foundation for advancing treatments such as ATH434 into the clinic.'
MSA is a neurodegenerative disease with major sources of disability resulting from motor symptoms characteristic of Parkinson's disease and impaired ability to maintain normal blood pressure, bowel function and bladder control. Current treatment includes medications and lifestyle changes to help manage symptoms, but there is no treatment of the underlying cause and no cure.
The study is enrolling early stage MSA patients and will track changes in clinical measures and biomarkers for up to one year. Over the course of the study, patients will undergo comprehensive evaluation with detailed neurological examination and clinical rating scales of motor, autonomic and activities-of-daily-living symptoms along with specialized neuroimaging and assessment of protein biomarkers in diverse biological specimens.
Data from bioMUSE will also be used to inform patient selection in Alterity's upcoming Phase 2 clinical trial of ATH434, its lead clinical candidate for the treatment of MSA. The US FDA has encouraged Alterity to utilize data from the bioMUSE study to aid in the development of efficacy endpoints for the Phase 2 study.
Vanderbilt University Medical Centre is one of the largest academic medical centres in the southeast US managing more than 2 million patients each year. The School of Medicine's biomedical research program is among the nation's top 10 in terms of National Institutes of Health peer review funding.
Dr David Stamler, Chief Medical Officer, added: 'As we prepare for our Phase 2 study, the data from bioMUSE will provide key information to help us optimize the study design. Starting this study brings us one step closer to finding novel treatments for this devastating condition.'
Alterity Therapeutics Ltd. published this content on 27 October 2020 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 26 October 2020 22:49:03 UTC