- The data provides further validation of the intended Phase 2
study design
MELBOURNE,
Australia and SAN
FRANCISCO, April 6,
2022 /PRNewswire/ -- 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 α–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.
About bioMUSE
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
clinical trial.
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 α-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 is no cure.[1]
[1]National Institutes of Health: Neurological
Disorders and Stroke, Multiple System Atrophy Fact Sheet
About ATH434
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 α-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.
Forward Looking Statements
This press release contains "forward-looking statements"
within the meaning of section 27A of the Securities Act of 1933 and
section 21E of the Securities Exchange Act of 1934. The Company has
tried to identify such forward-looking statements by use of such
words as "expects," "intends," "hopes," "anticipates," "believes,"
"could," "may," "evidences" and "estimates," and other similar
expressions, but these words are not the exclusive means of
identifying such statements.
Important factors that could cause actual results to differ
materially from those indicated by such forward-looking statements
are described in the sections titled "Risk Factors" in the
Company's filings with the SEC, including its most recent Annual
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not limited to the following: statements relating to the Company's
drug development program, including, but not limited to the
initiation, progress and outcomes of clinical trials of the
Company's drug development program, including, but not limited to,
ATH434, and any other statements that are not historical facts.
Such statements involve risks and uncertainties, including, but not
limited to, those risks and uncertainties relating to the
difficulties or delays in financing, development, testing,
regulatory approval, production and marketing of the Company's drug
components, including, but not limited to, ATH434, uncertainties
relating to the impact of the novel coronavirus (COVID-19) pandemic
on the company's business, operations and employees, the ability of
the Company to procure additional future sources of financing,
unexpected adverse side effects or inadequate therapeutic efficacy
of the Company's drug compounds, including, but not limited to,
ATH434, that could slow or prevent products coming to market, the
uncertainty of obtaining patent protection for the Company's
intellectual property or trade secrets, the uncertainty of
successfully enforcing the Company's patent rights and the
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whether written or oral, that may be made from time to time,
whether as a result of new information, future developments or
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SOURCE Alterity Therapeutics Limited