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Alterity Therapeutics to Deliver an Oral Presentation of the Positive ATH434 Phase 2 Trial Results at the American Academy of Neurology Annual Meeting MELBOURNE, AUSTRALIA AND SAN FRANCISCO, USA - 3 April 2025: Alterity

Key Takeaway: Alterity Therapeutics has announced that it will deliver oral and poster presentations on the positive results of its ATH434 Phase 2 trial at the upcoming American Academy of Neurology Annual Meeting. This trial demonstrated significant improvements in patients with early-stage Multiple System Atrophy (MSA), with ATH434 shown to improve daily functioning and motor performance. The company has also utilized wearable sensor technology to further evaluate treatment outcomes. ATH434's progression has led to its Orphan Drug Designation, enhancing its potential for market success in treating neurodegenerative diseases.

Market Sentiment Analysis

POSITIVE FACTORS

  • Alterity Therapeutics presented positive topline data from the ATH434 Phase 2 trial.
  • ATH434 shows potential for treating Multiple System Atrophy (MSA), a challenging neurodegenerative condition.
  • The company has received Orphan Drug Designation for ATH434 from both the U.S. FDA and the European Commission.
  • Innovative use of wearable sensor technology may improve patient outcome assessments.

Full Press Release Details

Alterity Therapeutics to Deliver an Oral Presentation
of the Positive ATH434
Phase 2 Trial Results at the American Academy of Neurology Annual Meeting
MELBOURNE, AUSTRALIA AND SAN FRANCISCO, USA
- 3 April 2025: Alterity Therapeutics (ASX: ATH, NASDAQ: ATHE) ("Alterity" or "the Company"), a biotechnology
company dedicated to developing disease modifying treatments for neurodegenerative diseases, today announced that an oral presentation
and a poster presentation related to Alterity's clinical programs in Multiple System Atrophy (MSA) will be delivered at the American
Academy of Neurology (AAN) 2025 Annual Meeting taking place April 5 - 9, 2025 in San Diego, CA.
David Stamler, M.D., Chief Executive Officer of
Alterity, commented, "We are excited to present the positive topline data along with new analyses from our ATH434-201 clinical trial
via an oral presentation at AAN, one of the premier global neurology meetings. In addition, data will be presented on the use of wearable
sensor technology to assess patient outcomes, an important component of evaluating novel treatments for individuals with MSA."
Type: Oral Presentation
Title: Topline Data from a Randomized, Double Blind, Placebo Controlled Phase 2 Study of ATH434 in Multiple System Atrophy
Presenter: Daniel Claassen, M.D., M.S., Professor of Neurology at Vanderbilt University Medical Center
Date/Time: Wednesday, April 9, 2025 at 2:24 PM PDT (USA)
Type: Poster Presentation
Title: Association Between Wearable Sensor Data and Clinical Scores in Individuals with Early-stage Multiple System Atrophy
Presenter: Ashkan Vaziri, PhD, BioSensics LLC
Date/Time: Saturday, April 5, 2025 at 11:45 AM PDT (USA)
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 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 recently announced positive results from the randomized, double-blind, placebo-controlled
Phase 2 clinical trial in patients with early-stage MSA. A second Phase 2 open-label 2 Biomarker trial in patients with more advanced
MSA is ongoing. ATH434 has been granted Orphan Drug Designation for the treatment of MSA by the U.S. FDA and the European Commission.
About ATH434-201 Phase 2 Clinical Trial
The ATH434-201 Phase 2 clinical trial is a randomized,
double-blind, placebo-controlled investigation of 12 months treatment with ATH434 in patients with MSA. The study evaluated the efficacy,
safety and pharmacokinetics of ATH434 as well as the effect of ATH434 on neuroimaging and protein biomarkers. Wearable sensors were employed
to evaluate motor activities outside of the clinic. The study enrolled 77 adults who were randomly assigned to receive ATH434 50 mg or
75 mg twice daily or matching placebo. The topline data showed that ATH434 produced clinically and statistically significant improvement
on the modified UMSARS Part I, a functional rating scale that assesses disability on activities of daily living affected in MSA.
In addition to the robust efficacy demonstrated on the UMSARS Part I, trends in improved motor performance were observed on the Parkinson's
Plus rating scale and overall benefit was shown on the Clinical Global Impression of Severity at the 50 mg dose. Wearable sensor data
indicated that both dose levels of ATH434 led to increased activity in an outpatient setting as compared to placebo. Biomarkers were used
to evaluate potential drug effect and target engagement. Both dose levels reduced iron accumulation in MSA affected brain regions and
trends in preservation of brain volume were observed relative to placebo. Additional information on the Phase 2 trial can be found by
ClinicalTrials.gov Identifier: NCT05109091.
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 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
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 is initially focused
on developing disease modifying therapies in Parkinson's disease and related disorders. Alterity recently reported positive data
for its lead asset, ATH434, in a Phase 2 clinical trial in participants with Multiple System Atrophy (MSA), a rare and rapidly progressive
Parkinsonian disorder. ATH434 is also being evaluated in a Phase 2 clinical trial in advanced MSA. In addition, Alterity 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 website at
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, 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 the focus of Alterity Therapeutics?

Alterity Therapeutics develops disease-modifying treatments for neurodegenerative diseases.

When will the Phase 2 trial results be presented?

The results will be presented at the AAN Annual Meeting on April 9, 2025.

What is ATH434 designed to do?

ATH434 aims to inhibit pathological protein aggregation linked to neurodegeneration.

How many patients were involved in the ATH434-201 trial?

The ATH434-201 trial enrolled 77 patients with early-stage MSA.

What is Multiple System Atrophy (MSA)?

MSA is a rare, progressive neurodegenerative disease affecting movement and autonomic functions.

Last updated: Apr 3, 2025