Recent Updates
Recently added Catalysts
ATHE Positive Sentiment Score: 80/100

Alterity Therapeutics Announces Multiple Oral and Poster Presentations to be Featured at the International MSA Congress -ATH434 Phase 2 Data to be Presented at Opening Plenary Session - MELBOURNE, AUSTRALIA AND SAN FRANC

Key Takeaway: Alterity Therapeutics has announced the presentation of its clinical programs related to Multiple System Atrophy (MSA) at the upcoming International MSA Congress from May 9-11, 2025, in Boston. CEO David Stamler will present data from the Phase 2 trial of ATH434, a therapy designed to slow disease progression in MSA, which has demonstrated robust clinical efficacy in preliminary results. Additionally, the company is sponsored at the congress, emphasizing its commitment to improving the quality of life for those affected by MSA.

Market Sentiment Analysis

POSITIVE FACTORS

  • Alterity Therapeutics will present multiple promising clinical data on MSA at an international congress.
  • ATH434 has received Fast Track Designation from the U.S. FDA, enhancing its development potential.
  • Preliminary Phase 2 trial results show significant improvement in MSA patients compared to placebo.

Full Press Release Details

Alterity Therapeutics Announces Multiple Oral
and Poster Presentations to be Featured at the International MSA Congress
2 Data to be Presented at Opening Plenary Session -
MELBOURNE, AUSTRALIA AND SAN FRANCISCO, USA
- 7 May 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 multiple oral and poster
presentations related to Alterity's clinical programs in Multiple System Atrophy (MSA) will be featured at the 2025 International
MSA Congress taking place May 9 - 11, 2025 in Boston, MA, USA. The Congress is presented by Mission MSA, a non-profit organization dedicated
to improving the quality of life and building hope for people affected by MSA through support services, educational resources, research
funding, and community engagement.
David Stamler, M.D., Chief Executive Officer of
Alterity, commented, "We are excited to play a prominent role at the MSA Congress which brings together a noteworthy group of physicians
and scientists as well as patients and care partners, all of whom are focused on advancing MSA research and care. Mission MSA is one of
the primary patient education and advocacy organizations for the disease in the U.S., and we are also proud to support the event as a
sponsor. We look forward to presenting our double-blind Phase 2 data for ATH434 as well as our bioMUSE natural history study with our
colleagues at Vanderbilt University Medical Center. Our clinical advancements in MSA continue to generate significant interest throughout
the community, bolstered by the recent Fast Track Designation for ATH434 that was granted by the US FDA."
Type: Oral Presentation
Session: Opening Plenary: Toward Disease Modifying Therapies of MSA
Title: ATH434 Slowed Disease Progression in a Phase 2 Study in Multiple System Atrophy
Presenter: David Stamler, M.D., Chief Executive Officer of Alterity
Date/Time: Friday, May 9, 2025 at 9:10 AM EDT (USA)
Type: Oral Presentation
Session: Biomarkers in MSA: Imaging
Title: MSA Atrophy Index (MSAai): A Quantitative Imaging Marker for Diagnosis and Monitoring of Multiple System Atrophy
Presenter: Amy Brown, M.D., M.S., Assistant Professor, Movement Disorders Division, Department of Neurology, Vanderbilt University Medical Center
Date/Time: Saturday, May 10, 2025 at 2:55 PM EDT
Type: Poster Session
Title: ATH434 Slowed Disease Progression in a Phase 2 Study in Multiple System Atrophy
Presenter: David Stamler, M.D., Chief Executive Officer of Alterity
Date/Time: Saturday, May 10, 2025 at 12:00 PM EDT
Poster: #85
Type: Poster Session
Title: Cutaneous Phosphorylated Alpha-Synuclein Deposition Informs Autonomic Function in Individuals with Early-Stage Multiple System Atrophy
Presenter: Leah Mann, PhD, Postdoctoral Research Fellow, Vanderbilt University Medical Center
Date/Time: Saturday, May 10, 2025 at 12:00 PM EDT
Poster: #86
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 in preclinical models.
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). Phase 1 studies have demonstrated the agent is well tolerated and achieved brain levels comparable to efficacious
levels in animal models of MSA. Positive results from the randomized, double-blind, placebo-controlled Phase 2 clinical trial in patients
with MSA demonstrated robust clinical efficacy, target engagement on key biomarkers, and a favorable safety profile. A second Phase 2
open-label biomarker trial in patients with more advanced MSA is ongoing. ATH434 has been granted Fast Track Designation by the U.S. FDA,
and Orphan Drug Designation by the U.S. FDA and the European Commission for the treatment of MSA.
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 data showed that, compared to placebo, ATH434 produced clinically and statistically significant
improvement on the modified Unified Multiple System Atrophy Rating Scale (UMSARS) Part I, a functional rating scale that assesses
disability on activities of daily living affected in MSA. Additional efficacy assessments demonstrated improvement consistent with the
positive UMSARS Part I findings including trends in improved motor performance on the Parkinson's Plus rating scale, the Clinical
Global Impression of Severity Scale, and the Orthostatic Hypotension Symptom Assessment (a patient reported outcome). Wearable sensor
data indicated that ATH434 also led to increased activity in an outpatient setting. Biomarkers were used to evaluate potential drug effect
and target engagement relative to placebo. Both dose levels stabilized or reduced iron accumulation in MSA affected brain regions with
trends in preservation of brain volume. ATH434 was well tolerated with similar adverse event rates compared to placebo and no serious
adverse events attributed to ATH434. Additional information on the Phase 2 trial can be found by ClinicalTrials.gov Identifier: NCT05109091.
Biomarkers of progression in Multiple System Atrophy
(bioMUSE) is a natural history study that aims to track the progression of individuals with MSA, a parkinsonian disorder without approved
therapy. The study is being conducted in collaboration with Vanderbilt University Medical Center in the U.S. under the direction
of Daniel Claassen, M.D., M.S., Professor of Neurology and Principal Investigator. Natural history studies are important for characterizing
disease progression in selected patient populations. The study has provided rich data for optimizing the design of Alterity's randomized
ATH434-201 Phase 2 clinical trial and enrolled approximately 20 individuals with clinically probable or clinically established MSA. BioMUSE
continues to provide vital information on early stage MSA patients, informs the selection of biomarkers suitable to evaluate target engagement
and preliminary efficacy, and delivers clinical data to characterize disease progression in a patient population that mirrors those currently
enrolling in the Phase 2 clinical trial.
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
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:
Head of Investor Relations and Business Development
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 is dedicated to developing disease-modifying treatments for neurodegenerative diseases.

When is the International MSA Congress in 2025?

The 2025 International MSA Congress will be held from May 9 to 11 in Boston, MA.

What does ATH434 aim to treat?

ATH434 is designed to inhibit pathological protein aggregation, targeting neurodegenerative diseases.

What designation has the FDA granted ATH434?

ATH434 has received Fast Track and Orphan Drug Designations from the U.S. FDA.

Who is leading the bioMUSE study?

The bioMUSE study is led by Daniel Claassen, M.D., M.S., at Vanderbilt University Medical Center.

Last updated: May 9, 2025