Full Press Release Details
Therapeutics Announces Presentation of Wearable Sensor Data from
the bioMUSE Natural History Study at the International Congress of Parkinson's
Disease and Movement Disorders
- Wearable sensors strongly correlated with
clinical scales on motor impairment -
- bioMUSE Study Validates Use of Wearable Sensors
for Alterity's Ongoing Phase 2 Clinical Trial -
AUSTRALIA AND SAN FRANCISCO, USA - 26 September 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 a poster from the ongoing Biomarkers of Progression in Multiple System Atrophy (bioMUSE) natural history study was presented
at the International Congress of Parkinson's Disease and Movement Disorders. The presentation correlates data from wearable sensors
with clinical assessments of motor function in individuals with Multiple System Atrophy (MSA).
MSA is a rapidly progressive parkinsonian
disorder that results in slowed movements, lack of coordination and difficulty with walking (gait) and balance that predisposes to falls.
The poster, entitled Wearable Sensors for Quantitative Motor Assessments in Multiple System Atrophy, describes 12 participants
from the ongoing bioMUSE study who wore sensors for up to one year to characterize their motor disability in an outpatient setting. In
the study, sensor parameters strongly correlated with clinical scales of motor impairment that were largely driven by changes in gait
stability. Investigators concluded that step count and walking time are sensitive measures of disease progression in early MSA.
"In this study, we were able
to determine that wearable sensors provide a quantitative measurement of MSA progression that is not captured by neurological examination,"
said Daniel Claassen, MD, Associate Professor of Neurology at Vanderbilt University Medical Center and Principal Investigator in the bioMUSE
study. "The level of detail captured by the wearable sensors clearly showed a meaningful slowdown in activity, particularly in patients
with rapidly progressive disease. These results provide a tool for physicians to assess the gait and activity levels of their patients,
and will inform future trials in MSA as potential outcome measures for disease modifying therapies."
David Stamler, M.D.,
Chief Executive Officer, Alterity, added, "We are very encouraged by these findings as they validate the use of wearable
sensors in our ongoing ATH434 Phase 2 clinical trial in MSA. We are using these sensors in study participants to evaluate motor performance
in a real-world setting. Progressive decline in motor function represents an important source of disability in MSA so it is important
to have tools to measure it with precision. The data presented demonstrate that we can quantify activity levels of participants to support
the outcome of the study."
The poster presentation includes
data from n=12 participants who wore a PAMSys device, a validated wearable sensor for continuous monitoring of gait and activity parameters.
PAMSys actigraphy sensors were worn continuously for up to 12-months, allowing assessment of gait parameters (step count, bouts of walking,
steps per bout, cadence/variability), postures (minutes of sitting, lying, standing, or walking) and postural transitions (sit-to-stand).
Clinical assessments were obtained at baseline and months 3, 6, 9 and 12. At each time point, sensor parameters were obtained by averaging
data over 14-day periods.
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.
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 successfully completed Phase 1 studies demonstrating the agent is well tolerated, orally
bioavailable, and achieved brain levels comparable to efficacious levels in animal models of MSA. ATH434 has been granted Orphan designation
for the treatment of MSA by the U.S. FDA and the European Commission.
About ATH434 Phase 2 Clinical Trial
The Phase 2 clinical trial is a randomized,
double-blind, placebo-controlled investigation of ATH434 in patients with early-stage MSA. The study will explore the effect of ATH434
treatment on neuroimaging and protein biomarkers, such as excess brain iron and aggregating -synuclein, which are important contributors
to MSA pathology. Clinical endpoints will permit comprehensive assessment of ATH434 efficacy along with characterization of safety and
pharmacokinetics. The use of wearable sensors will allow evaluation of motor parameters that are important in patients with MSA. The study
is expected to enroll approximately 60 adult patients to receive one of two dose levels of ATH434 or placebo. Patients will receive treatment
for 12 months which will provide an opportunity to detect changes in efficacy endpoints to optimize design of a definitive Phase 3 study.
Additional information on the Phase 2 trial can be found here: ClinicalTrials.gov Identifier: NCT05109091.
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 is no cure.1
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.
Investor and Media Contacts:
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.
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 Report on
Form 20-F as well as reports on Form 6-K, including, but 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 uncertaintyof
obtaining patent protection for the Company's intellectualproperty or trade secrets, the uncertainty of successfully enforcing the Company's