Full Press Release Details
Alterity Therapeutics Announces Presentation
of bioMUSE Data at the
American Academy of Neurology (AAN) Annual Meeting
The data provides further validation of the
intended Phase 2 study design
MELBOURNE, AUSTRALIA AND SAN FRANCISCO, USA
- 7 April 2022: 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 new data are being presented
in a poster session today from the Company's ongoing Biomarkers of progression in Multiple System Atrophy (bioMUSE) study today
at the American Academy of Neurology (AAN) Annual Meeting taking place in Seattle, Washington, USA.
BioMUSE is a natural history study that is tracking
disease progression in individuals with early Multiple System Atrophy (MSA), a Parkinsonian disorder without approved therapy. The
study is being conducted in collaboration with Vanderbilt University Medical Center in the U.S. under the direction of Daniel Claassen,
MD, Associate Professor of Neurology and Principal Investigator.
The poster presented by Dr. Claassen, entitled,
"Iron Accumulation Correlates with Disease Severity in Patients with Multiple System Atrophy" assesses the relationship between
iron accumulation and symptom severity in patients with MSA. The study showed that advanced quantitative MRI methods demonstrated pathological
iron accumulation in MSA patients that relate to clinical severity. The data also support the use of quantitative susceptibility mapping
(QSM) as a biomarker of disease severity in MSA.
"BioMUSE is a groundbreaking study in the
evaluation of early stage MSA patients to track disease progression and select biomarkers for evaluating target engagement and preliminary
efficacy of clinical development compounds," said Dr. Claassen. "Through brain imaging, we have been able to measure and track
iron accumulation in various brain regions of Parkinson's disease (PD) and MSA patients that contributes to disease progression.
The study reinforces our belief that early diagnosis of MSA is vital for maximizing neuronal preservation with disease modifying therapies."
David Stamler, MD, Chief Executive Officer, Alterity,
added, "The ongoing bioMUSE trial continues to validate our approach of reducing iron accumulation to inhibit -synuclein
aggregation, rescue neurons and improve function in MSA patients. We expect to launch our global Phase 2 clinical trial of ATH434 in early-stage
MSA in the second quarter of 2022, and we are grateful to Dr. Claassen and his team for this important work to help guide our clinical
development program."
The presentation includes data from 52
individuals, 17 with MSA, 17 with PD, and 18 age-matched healthy controls with a mean age between 60 and 65 years of age. The MRI
results show that while iron accumulates in the brains of both PD and MSA patients, there are clear distinctions. In comparison to
PD, significantly increased iron concentration in MSA patients was noted in the lentiform nucleus (LFN) and dentate nucleus (DN) of
the brain. These regions play a key role in muscle coordination and control of voluntary movements. Compared to healthy controls,
MSA patients had greater iron concentration in the substantia nigra (SN) and DN. The substantia nigra is a critical brain
region for the production of dopamine and affects many functions of the central nervous system including movement control,
learning and emotion. In MSA, greater iron concentration in the SN and external globus pallidus (GPe) positively correlated with
severity as measured by the Unified Multiple System Atrophy Rating Scale (UMSARS) and Natural History and Neuroprotection in
Parkinson Plus Syndromes (NNIPPS) rating scales.
Biomarkers of progression in Multiple System Atrophy
(bioMUSE) is an ongoing, 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 U.S. 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 has provided rich data for optimizing the design of Alterity's
Phase 2 clinical trial and will be expanded to include a total of 20 patients with MSA. The ongoing study will continue to provide vital
information on early stage MSA patients, inform the selection of biomarkers suitable to evaluate target engagement and preliminary efficacy,
and deliver clinical data to characterize disease progression in a patient population that mirrors those to be enrolled in the Phase 2
About Multiple System Atrophy
Multiple System Atrophy (MSA) is a rare, neurodegenerative
disease characterized by failure of the autonomic nervous system and impaired movement. The symptoms reflect the progressive loss of function
and death of different types of nerve cells in the brain and spinal cord. It is a rapidly progressive disease and causes profound disability.
MSA is a Parkinsonian disorder characterized by a variable combination of slowed movement and/or rigidity, autonomic instability that
affects involuntary functions such as blood pressure maintenance and bladder control, and impaired balance and/or coordination that predisposes
to falls. A pathological hallmark of MSA is the accumulation of the protein -synuclein within glia, the support cells of the central
nervous system, and neuron loss in multiple brain regions. MSA affects approximately 15,000 individuals in the U.S., and while some of
the symptoms of MSA can be treated with medications, currently there are no drugs that are able to slow disease progression and there
1National Institutes of Health: Neurological Disorders and
Stroke, Multiple System Atrophy Fact Sheet
Alterity's lead candidate, ATH434, is the
first of a new generation of small molecules designed to inhibit the aggregation of pathological proteins implicated in neurodegeneration.
ATH434 has been shown preclinically to reduce -synuclein pathology and preserve nerve cells by restoring normal iron balance in
the brain. In this way, it has excellent potential to treat Parkinson's disease as well as various forms of atypical Parkinsonism
such as Multiple System Atrophy (MSA). ATH434 has successfully completed a Phase 1 clinical trial demonstrating the agent is well tolerated,
orally bioavailable, and achieved brain levels comparable to efficacious levels in animal models of MSA, with the objective of restoring
function in patients with MSA and other Parkinsonian disorders.
ATH434 has been granted Orphan designation for
the treatment of MSA by the U.S. FDA and the European Commission.
About Alterity Therapeutics Limited
Alterity Therapeutics is a clinical stage biotechnology
company dedicated to creating an alternate future for people living with neurodegenerative diseases. The Company's lead asset,
ATH434, has the potential to treat various Parkinsonian disorders. Alterity also has a broad drug discovery platform generating patentable
chemical compounds to intercede in disease processes. The Company is based in Melbourne, Australia, and San Francisco, California, USA.
For further information please visit the Company's web site at www.alteritytherapeutics.com.
Authorisation & Additional information
This announcement was authorized by David Stamler, CEO
of Alterity Therapeutics Limited.
Contact: Investor Relations
| Australia | US |
| Rebecca Wilson | Remy Bernarda |
| E: WE-AUAlterity@we-worldwide.com | remy.bernarda@iradvisory.com |
| Tp: +61 417 382 391 | Tp: +1 (415) 203-6386 |
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