Full Press Release Details
Alterity Therapeutics Completes Last Patient
Visit in ATH434-201 Phase 2
Clinical Trial in Early-Stage Multiple System Atrophy
Disease Modifying Drug Candidate Targeting Alpha-Synuclein and Iron in Parkinsonian Disorders -
Expected in Early 2025 -
MELBOURNE, AUSTRALIA AND SAN FRANCISCO, USA
- 4 December 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 the last patient
in the ATH434-201 Phase 2 trial, a randomized, double-blind, placebo-controlled investigation in early-stage multiple system atrophy (MSA),
has completed the study. With the achievement of this milestone, topline results are expected to be reported in late January or early
"We are very excited to announce that the
last participant in our Phase 2 study has completed all clinical evaluations, the final milestone that starts the clock to reporting topline
data in this rare neurodegenerative disease," said, David Stamler, M.D., Chief Executive Officer of Alterity. "The completion
of our ATH434-201 trial represents a major accomplishment for Alterity, and I would like to recognize the trial participants for their
involvement in the study. I would also like to thank our clinical sites and our study team for their hard work and dedication in conducting
the trial. Throughout the course of the trial, we have had tremendous interest from our clinical sites, doctors and patients around the
globe as we seek a treatment that could potentially slow the progression of this devastating disease. With the last patient visit behind
us, we can now focus our attention on cleaning and locking the database and reporting topline data early next year."
About ATH434-201 Phase 2 Clinical Trial
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 were also 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 received 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
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:
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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
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