Highlights:
- Alterity receives positive guidance from the European Medicines
Agency's Committee for Medicinal Products for Human Use on its
Phase 2 clinical trial for Multiple System Atrophy.
- Concurrence with Alterity's plan to target early stage MSA
patients.
- Endorsement on selection of biomarker endpoints to assess
pathological hallmarks of MSA.
- Agreement that ATH434 has potential as a disease modifying
treatment.
MELBOURNE, Australia and
SAN FRANCISCO, June 23, 2021 /PRNewswire/ -- Alterity
Therapeutics Limited (ASX: ATH, NASDAQ: ATHE) ("Alterity" or "the
Company") has received guidance from the European Medicines Agency
(EMA) regarding key aspects of the Company's Phase 2
clinical trial for investigational drug ATH434 in the treatment of
Multiple System Atrophy (MSA).
Alterity is actively preparing for the launch of its Phase 2
trial expected to commence in 2H of this calendar year. The
EMA is the agency of the European Union responsible for the
evaluation and supervision of medicinal products. More
specifically, EMA provides an important role in supporting the
timely and sound development of high-quality, effective, and safe
medicines, for the benefit of patients. Whilst their advice is
non-binding, it has the benefit of influencing improved trial
designs that are more likely to generate robust and complete data
to show whether a treatment works and is safe.
Given there is no approved treatment for MSA, there is currently
no regulatory precedence for defining the most suitable patient
population or clinical endpoints in efficacy studies, thus
requiring greater consideration in developing an optimal trial
design. Accordingly, Alterity has sought diverse input from
clinical experts and global regulatory authorities, and is
conducting a natural history study, called BioMUSE, to identify
biomarkers and clinical endpoints best suited to capture efficacy
signals in the Phase 2 study.
The EMA has given its support to Alterity's intention to enroll
early-stage MSA patients and to utilize biomarkers to accurately
diagnose these patients prior to enrolment. Improving
diagnostic accuracy and targeting early-stage patients will enable
Alterity to maximize the opportunity to demonstrate the efficacy of
ATH434, its potentially disease modifying therapy.
ATH434 is the first of a new generation of small molecule drug
candidates designed to block the accumulation and aggregation of
α-synuclein. Alpha-synuclein, when aggregated in the brain,
is a pathological hallmark of Parkinsonian disorders such as MSA
and is considered an important biologic target for treating these
neurodegenerative diseases. ATH434 is thought to achieve its
biological effect on α-synuclein by binding and redistributing
excess iron in areas of pathology. The EMA recognized the potential
role of iron in the pathogenesis of MSA and accordingly supported
the use of biomarker endpoints to assess iron content and
α-synuclein pathogenesis.
As a result of interactions with the EMA, and previously with
the US Food and Drug Administration (FDA), Alterity is finalizing
its Phase 2 trial design including patient selection, sample size,
treatment duration, as well as primary and secondary endpoints.
In addition, the bioMUSE Natural History Study being conducted
at Vanderbilt University Medical Center
in the US, has enrolled more than 50% of targeted patients and
continues to collect vital clinical and biomarker data to inform
the Phase 2 study design.
Alterity CEO Dr David Stamler
said: "MSA is a devastating disease with no cure and few effective
treatment options. With the valuable advice received from the EMA,
we now have a clear path forward to finalize the study design and
generate data that global regulatory authorities are seeking. This
is another important step toward bringing much needed disease
modifying treatments to individuals with MSA."
Authorisation & Additional information
This
announcement was authorized by David
Stamler, CEO of Alterity Therapeutics Limited.
About Alterity Therapeutics Limited and ATH434
Alterity's lead candidate, ATH434, is the first of a new generation of small molecules designed to inhibit
the aggregation of pathological proteins implicated in
neurodegeneration. ATH434 has been shown to
reduce abnormal accumulation of α-synuclein and tau proteins in animal models of disease by restoring
normal iron balance in the brain. In this way, it has excellent potential to treat various forms of atypical
Parkinsonism such as Multiple System Atrophy (MSA) and Progressive
Supranuclear Palsy (PSP).
ATH434 has been granted Orphan designation for the treatment of MSA by the US FDA and the European
Commission.
For further information please visit the Company's web site at
www.alteritytherapeutics.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 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, uncertainties
relating to the impact of the novel coronavirus
(COVID-19) pandemic on the company's business, operations and employees,
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 patent
protection for the Company's
intellectual property or trade secrets,
including, but not limited to, the intellectual property relating to ATH434.
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.
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SOURCE Alterity Therapeutics Limited