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 presentation from the Company’s Biomarkers of
progression in Multiple System Atrophy (bioMUSE) Natural History
Study at the recent 34th International Symposium on the Autonomic
Nervous System (AAS).
The poster entitled, “Relationship between
N-acetylaspartate and neurofilament light chain in multiple system
atrophy” was presented by Paula Trujillo Diaz, PhD, Research
Assistant Professor, Department of Neurology, Vanderbilt University
Medical Center. Because MSA is pathologically characterized by
degeneration and loss of neurons in the brain, identifying
biomarkers to assess disease severity is critical.
N-acetylaspartate (NAA) is a novel biomarker of neuronal integrity
with potential for assessing disease severity, monitoring the
course of disease, and evaluating the efficacy of disease modifying
therapies in MSA. In the study, the data provided evidence that NAA
correlates with levels of neurofilament light chain (NfL) in
patients with early MSA. NfL is a widely used biomarker that is a
measure of neuronal damage. The results suggest that NAA
concentration may reflect the degree of neuronal integrity in these
subjects.
“These valuable data produced by our partners at
Vanderbilt continue to demonstrate that we are leading the way in
the biomarker evaluation of MSA,” said David Stamler, M.D., Chief
Executive Officer of Alterity. “The data presented at AAS reveals
another potentially important biomarker for the evaluation of this
rapidly progressive disease with no approved treatment. The field
is seeking non-invasive biomarkers to assess disease severity and
this novel biomarker represents another potential shot on goal for
demonstrating the efficacy of ATH434, our lead drug candidate in
Phase 2 for the treatment of MSA. The findings suggest that the NAA
metabolite may be a useful biomarker for assessing disease severity
and treatment response in MSA.”
The study assessed 13 early-stage MSA patients
(motor symptom onset ≤ 4 yrs) with diagnosis supported by a
multimodal approach that utilizes neuroimaging and fluid
biomarkers1. Participants completed neurologic examination and
clinical assessment with the Unified Multiple System Atrophy Rating
Scale (UMSARS) and the Natural History and Neuroprotection in
Parkinson Plus Syndromes scale (NNIPPS). All participants had
α-synuclein seed amplification assay results consistent with MSA,
CSF NfL > 2000, and plasma NfL > 20. The investigators
utilized a non-invasive MRI technique known as magnetic resonance
spectroscopy (MRS) that allows quantification of metabolites such
as NAA in the brain. NAA is a a marker of neuronal integrity given
its role in cellular energetics and myelin synthesis. In this
study, the investigators tested the hypothesis that the quantity of
NAA measured using MRS is correlated with NfL levels.
About ATH434
Alterity’s lead candidate, ATH434, is an oral
agent designed to inhibit the aggregation of pathological proteins
implicated in neurodegeneration. ATH434 has been shown
preclinically to reduce α-synuclein pathology and preserve neuronal
function by restoring normal iron balance in the brain. As an iron
chaperone, it has excellent potential to treat Parkinson’s disease
as well as various Parkinsonian disorders such as Multiple System
Atrophy (MSA). ATH434 successfully completed Phase 1 studies
demonstrating the agent is well tolerated and achieved brain levels
comparable to efficacious levels in animal models of MSA. ATH434 is
currently being studied in two clinical trials: Study ATH434-201 is
a randomized, double-blind, placebo-controlled Phase 2 clinical
trial in patients with early-stage MSA and Study ATH434-202 is an
open-label Phase 2 Biomarker trial in patients with more advanced
MSA. ATH434 has been granted Orphan drug designation for the
treatment of MSA by the U.S. FDA and the European Commission.
About bioMUSE
Biomarkers of progression in Multiple System
Atrophy (bioMUSE) is a natural history study that aims to track the
progression of individuals 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, M.D., M.S., 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 randomized ATH434-201 Phase 2
clinical trial and enrolled approximately 20 individuals with
clinically probable or clinically established MSA. BioMUSE
continues to provide vital information on early stage MSA patients,
informs the selection of biomarkers suitable to evaluate target
engagement and preliminary efficacy, and delivers clinical data to
characterize disease progression in a patient population that
mirrors those currently enrolling 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 at
least 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.2
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 and is currently being evaluated in two Phase 2 clinical
trials in Multiple System Atrophy. Alterity also has a broad drug
discovery platform generating patentable chemical compounds to
treat the underlying pathology of neurological diseases. 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.
1Claasen, et al, “A multimodal approach for diagnosis of early
Multiple System Atrophy”, MDS 20232Multiple System Atrophy |
National Institute of Neurological Disorders and Stroke
(nih.gov)
Authorisation & Additional informationThis
announcement was authorized by David Stamler, CEO of Alterity
Therapeutics Limited.
Investor and Media Contacts:
AustraliaHannah
Howlettwe-aualteritytherapeutics@we-worldwide.com+61 450 648
064
U.S.Remy Bernardaremy.bernarda@iradvisory.com
+1 (415) 203-6386
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