Full Press Release Details
Alterity Therapeutics Presents ATH434-201 Phase
2 Clinical Trial Results at European MSA Symposium
MELBOURNE, AUSTRALIA AND SAN FRANCISCO, USA
- 28 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 David Stamler, M.D.,
Chief Executive Officer presented the ATH434-201 Phase 2 clinical trial results at the annual MSA Research Symposium hosted by University
College London, Institute of Neurology in partnership with the MSA Trust of the U.K.
"We were honoured to be selected to present
the recent data from our double-blind Phase 2 trial," said, Dr. Stamler. "The Symposium brought together prominent clinicians
and researchers from both Europe and the US along with industry scientists, all of whom are focused on increasing their understanding
of MSA and advancing new therapies for this aggressive disorder. The strong clinical efficacy data and novel mechanism of ATH434 was well
received by this esteemed group of clinicians and academics, as we collectively seek solutions to improve the lives of individuals living
Presentation: A Randomized, Double Blind, Placebo
Controlled Study of ATH434 in MSA
The oral presentation included data from Alterity's
ATH434-201 Phase 2 clinical trial. The clinical analysis included 71 patients who had at least one post-baseline assessment of the key
clinical endpoint, the modified UMSARS1 I activities of daily living scale. On this endpoint, ATH434 demonstrated a clinically
significant reduction in disease severity versus placebo, with a 48% relative treatment effect at the 50 mg dose (p=0.02)^
and a 30% relative treatment effect at the 75 mg dose (p=0.16) at 52 weeks. Additional efficacy assessments showed improvement consistent
with the UMSARS I findings: the Clinical Global Impression of Severity Scale2 demonstrated improvement compared to placebo
at both dose levels, with difference at 50 mg achieving nominal statistical significance (p=0.0088). On the Orthostatic Hypotension Symptom
Assessment (a patient reported outcome), on average placebo patients worsened by approximately 6 points over 52 weeks whereas both ATH434
treatment groups improved over the same period (p=0.08 at 50 mg, p=0.14 at 75 mg). Increased activity in the outpatient setting was observed
at both dose levels as compared to placebo with wearable sensors, with clinically meaningful improvements in step count, bouts of walking,
total walking time, and total standing time. ATH434 was well tolerated with similar adverse event rates compared to placebo and no serious
adverse events attributed to ATH434. Regarding neuroimaging, ATH434 demonstrated target engagement by stabilizing or reducing iron at
both dose levels compared to placebo in MSA affected brain regions. In addition, ATH434 demonstrated trends in reducing brain atrophy
at both dose levels compared to placebo. Overall, the study results support continued advancement of ATH434 for the treatment of MSA.
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
1 UMSARS: Unified Multiple System Atrophy Rating Scale
^ All p-values are uncorrected
2 Clinical Global Impression of Severity: a clinician assessment
of the total picture of the subject including the impact of the illness on function and level of distress
System Atrophy | National Institute of Neurological Disorders and Stroke (nih.gov)
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.
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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