Full Press Release Details
Alterity Therapeutics Phase
2 Data Monitoring Committee Recommends
Continuing Clinical Trial as Planned After Second Review
- ATH434-201 Trial on Track to Complete
- Top-Line Data Expected in
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
-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. 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:
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.