Regulatory Authorization Granted to Proceed with ATH434 Phase
2 Clinical Trial
MELBOURNE, Australia and SAN FRANCISCO, Sept. 20,
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 the approval of
its Investigational New Drug (IND) application by the U.S. Food and
Drug Administration (FDA) to allow evaluation of ATH434 in
individuals with Multiple System Atrophy (MSA), a rare and highly
debilitating Parkinsonian disorder. The approval of this IND
authorizes Alterity to conduct its Phase 2 clinical trial in the
U.S.
"The FDA's acceptance of our IND to study ATH434 in individuals
with MSA is another important milestone in the development of a
much-needed treatment for this devastating condition," said
David Stamler, M.D., Chief Executive
Officer, Alterity. "We have been working closely with leading MSA
physicians in the U.S. and expect to open our first clinical trial
site for enrolment in the fourth quarter of this year. This
complements our ongoing progress in other regions around the
world."
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 α-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 ATH434
Alterity's lead candidate, ATH434, is 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
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 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 Institute
of Health: Neurological Disorders and Stroke, Multiple System
Atrophy Fact Sheet
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.
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SOURCE Alterity Therapeutics