MELBOURNE, Australia and SAN FRANCISCO, July 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 the first patient has been dosed in
the Company's Phase 2 clinical trial of ATH434 in Multiple System
Atrophy (MSA), a rare and highly debilitating Parkinsonian
disorder.
While it is similar to Parkinson's disease, MSA progresses more
rapidly and causes profound disability. In addition to the motor
symptoms characteristic of Parkinson's disease, MSA manifests with
more severe autonomic nervous system impairment resulting in
bladder dysfunction and the inability to maintain normal blood
pressure, as well as uncoordinated or clumsy movements that
contribute to falling. There are no known causes or specific risk
factors associated with the disease.
"Dosing of our first patient is a significant milestone for
Alterity as we look to bring a potential new treatment option to
individuals living with MSA," said David
Stamler, M.D., Chief Executive Officer, Alterity. "This is
just the first step as we expect to expand enrolment in multiple
regions over the second half of this year. We are grateful to the
entire clinical team at the New Zealand Brain Research Institute
whose efficiency and dedication to their patients supported this
accomplishment."
"As a clinician looking after people with MSA, I'm very pleased
that Alterity has chosen to bring this clinical trial of a novel
prospective therapy to New
Zealand. There is presently no available treatment to slow
down or prevent progression of this distressing brain disorder so
trials such as this are very welcome," added Professor Tim Anderson, Lead Investigator of the trial at
the New Zealand Brain Research Institute.
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 imaging and protein biomarkers, such as aggregating
α-synuclein and excess iron, which are important contributors to
MSA pathology. Clinical and biomarker endpoints will permit
comprehensive assessment of ATH434 efficacy along with
characterization of safety and pharmacokinetics. The study is
expected to enroll approximately 60 adult patients to receive one
of two doses 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 by
ClinicalTrials.gov Identifier: NCT05109091.
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 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
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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