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Alterity Therapeutics Enrolls First Patient in the U.S. in ATH434 Phase 2 Clinical Trial in Multiple System Atrophy MELBOURNE, AUSTRALIA AND SAN FRANCISCO, USA - 16 March 2023: Alterity Therapeutics (ASX: ATH, NASDAQ: AT

Key Takeaway: Alterity Therapeutics has initiated its Phase 2 clinical trial for ATH434 in Multiple System Atrophy (MSA) with the dosing of the first patient in the U.S. at Vanderbilt University Medical Center. The trial aims to assess the efficacy and safety of ATH434, an oral treatment designed to inhibit the aggregation of pathological proteins involved in neurodegeneration. With participation from five countries and expected enrollment of about 60 adults, the study seeks to evaluate treatment effects on biomarkers and clinical endpoints over a 12-month period. Previous Phase 1 studies indicated that ATH434 is well tolerated, providing an optimistic outlook for its potential to treat MSA and related disorders.

Market Sentiment Analysis

POSITIVE FACTORS

  • First patient has been dosed in the U.S. for ATH434 Phase 2 trial.
  • The trial is gaining momentum with active recruitment across five countries.
  • ATH434 has shown potential in preclinical studies to reduce neurodegenerative pathology.

Full Press Release Details

Alterity Therapeutics Enrolls
First Patient in the U.S. in ATH434 Phase 2 Clinical Trial in Multiple System Atrophy
MELBOURNE, AUSTRALIA AND SAN FRANCISCO, USA
- 16 March 2023: 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 participant has
been dosed in the U.S. in the Company's Phase 2 clinical trial of ATH434 in Multiple System Atrophy (MSA), a rare and highly debilitating
Parkinsonian disorder.
"The U.S. is an important
region for our ATH434 Phase 2 clinical trial, and we are thrilled to have dosed our first patient at Vanderbilt University Medical Center,"
said David Stamler, M.D., Chief Executive Officer, Alterity. "Our colleagues at Vanderbilt are long-time supporters of the ATH434
development program and we are grateful for their continued efforts. The Phase 2 trial is gaining momentum with active recruitment in
five countries, as we look to bring a potential new treatment option to individuals living with MSA."
The Phase 2 clinical trial is a randomized,
double-blind, placebo-controlled investigation of ATH434 in patients with early-stage MSA. The study will evaluate the effect of ATH434
treatment on neuroimaging and protein biomarkers to demonstrate target engagement, and clinical endpoints to demonstrate efficacy, in
addition to assessments of safety and pharmacokinetics. The selected biomarkers, including brain iron and aggregating -synuclein,
are important contributors to MSA pathology and are therefore appropriate targets to demonstrate drug activity. Wearable sensors will
also be employed to evaluate motor activities that are important to patients with MSA. The study is expected to enroll approximately 60
adults to receive one of two dose levels of ATH434 or placebo. Participants 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.
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 -synuclein pathology and preserve nerve cells 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 a randomized, double-blind, placebo-controlled
Phase 2 clinical trial in patients with early-stage 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 -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. Important 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.
Any 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.

Frequently Asked Questions

What is ATH434 being tested for?

ATH434 is being tested as a treatment for Multiple System Atrophy (MSA).

Where was the first patient for the trial dosed?

The first patient was dosed at Vanderbilt University Medical Center.

How many participants will enroll in the Phase 2 trial?

Approximately 60 adults will participate in the Phase 2 trial.

What is the duration of treatment in the trial?

Participants will receive treatment for 12 months.

What designation has ATH434 received from the FDA?

ATH434 has received Orphan designation for treating MSA from the FDA.

Last updated: Mar 16, 2023