Full Press Release Details
Therapeutics Doses First Patient in ATH434 Phase 2 Clinical Trial in Multiple System Atrophy
AUSTRALIA AND SAN FRANCISCO, USA - 6 July 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
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.
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.
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."
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
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 -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.
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 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.
has been granted Orphan designation for the treatment of MSA by the U.S. FDA and the European Commission.
Multiple System Atrophy
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
Institute of Health: Neurological Disorders and Stroke, Multiple System Atrophy Fact Sheet
Alterity Therapeutics Limited
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.
& Additional information
announcement was authorized by David Stamler, CEO of Alterity Therapeutics Limited.
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
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 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 uncertainty of the Company freedom to operate.
forward-looking statement made by us in this press release is based only on information currently available to us and speaks only as
of the date on which it is made. We undertake no obligation to publicly update any forward-looking statement, whether written or oral,
that may be made from time to time, whether as a result of new information, future developments or otherwise.