Full Press Release Details
Alterity Therapeutics Completes
Enrolment in ATH434-201 Phase 2 Clinical Trial in Multiple System Atrophy
- ATH434-201 is a randomized,
double-blind, placebo-controlled study in early-stage MSA -
- Lead clinical development
program enrolled 77 participants globally -
MELBOURNE, AUSTRALIA AND SAN FRANCISCO, USA
- 08 November 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 it has successfully completed enrolment in its ATH434-201 Phase 2 clinical trial. ATH434-201 is a randomized, double-blind,
placebo-controlled study in patients with early-stage multiple system atrophy (MSA) conducted across the U.S., Europe, Australia and
"Completing enrolment in our
ATH434-201 clinical trial in MSA is a significant milestone for Alterity as we look to bring a new oral therapy to people living with
this devastating disease," said David Stamler, M.D., Chief Executive Officer of Alterity. "Due to the great clinical need
and physician interest in our novel approach to address the underlying pathology of MSA, we exceeded our enrolment target. With our trial
now fully enrolled, study treatment will conclude in the fourth quarter of 2024 and the results from the trial will clarify the path forward
for potential approval of ATH434."
"We sincerely thank the clinical
trial participants, investigators, and all study staff for their dedication and ongoing support of the study as we pursue the common goal
of improving treatment of MSA," concluded Dr. Stamler.
About ATH434-201 Phase 2 Clinical
The ATH434-201 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. Selected biomarkers, such as brain iron and aggregating -synuclein,
are important contributors to MSA pathology and are therefore appropriate targets to demonstrate drug activity. Wearable sensors have
also been employed to evaluate motor activities that are important to patients with MSA. The study enrolled 77 adults who were randomly
assigned 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 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 is currently being studied in two clinical trials: Study ATH434-201
is a randomized, double-blind, placebo-controlled Phase 2 clinical trial in patients with early-stage MSA and Study ATH434-202 is an open-label
Phase 2 Biomarker trial in patients with more advanced MSA. ATH434 has been granted Orphan drug 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 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 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 and is currently being evaluated in two Phase 2 clinical
trials in Multiple System Atrophy. Alterity also 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 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:
Remy Bernarda remy.bernarda@iradvisory.com
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
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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
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