Phase 2 DMC Recommends Continuing Clinical Trial as Planned
Released
Released
Announcement PDF
Alterity Therapeutics Phase 2 Data Monitoring Committee Recommends
Continuing Clinical Trial as Planned After Second Review
- ATH434-201 Trial on Track to Complete in November 2024 –
- Top-Line Data Expected in January 2025 MELBOURNE, AUSTRALIA AND SAN FRANCISCO, USA – 6 February 2024: 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 independent Data Monitoring Committee (DMC) has completed its second review of trial
data and recommended the ATH434-201 Phase 2 study continue as planned. The ATH434-201
clinical trial is a randomized, double-blind, placebo-controlled investigation of ATH434 in patients
with early-stage multiple system atrophy (MSA), a rare neurodegenerative disease with no
approved treatments to slow or stop its progression.
The DMC conducted its second prespecified review of unblinded clinical data from study
participants. The DMC expressed no concerns about safety and recommended that the study
continue without modification. The plan for the DMC to review initial safety data has been
cleared with the U.S. Food and Drug Administration.
“With accumulated data from our Phase 2 study, we are pleased to report that the Data
Monitoring Committee has determined that there are no safety concerns and that the study can
continue as planned,” said David Stamler, M.D., Chief Executive Officer of Alterity. “Participants
in the trial are being dosed for 12 months; therefore, this recommendation is an important
milestone as participants able to safely tolerate ATH434 as their time on study increases. We
remain on track to complete the study in November 2024 and report top-line data in January
2025.”
The ATH434-201 Phase 2 clinical trial is evaluating 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 α-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.
About ATH434
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 α-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 α-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
1Multiple System Atrophy | National Institute of Neurological Disorders and Stroke (nih.gov)
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:
Australia
Hannah Howlett
we-aualteritytherapeutics@we-worldwide.com
+61 450 648 064
U.S.
Remy Bernarda
remy.bernarda@iradvisory.com
+1 (415) 203-6386
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.
Share your feedback
Discover