Alterity Therapeutics Announces Regulatory Authorization to Proceed with ATH434 Phase 2 Clinical Trial in the United Kingdom

Rhea-AI Summary

Alterity Therapeutics (ASX: ATH, NASDAQ: ATHE) has received approval from the UK's Medicines & Healthcare products Regulatory Agency (MHRA) for its Phase 2 clinical trial of ATH434, aimed at treating Multiple System Atrophy (MSA), a debilitating neurodegenerative condition. The trial will begin in New Zealand and expand to other regions, including the UK and the U.S. ATH434 targets protein aggregation and aims to improve patient outcomes. This follows a successful Phase 1 trial, highlighting ATH434's potential as a novel treatment for patients with no current options.

Positive

• MHRA approval enables progression to Phase 2 trial for ATH434.
• Successful Phase 1 trial showed ATH434 is well-tolerated and bioavailable.
• ATH434 targets underlying pathology in MSA, addressing a significant unmet medical need.

Negative

• None.

MELBOURNE, Australia, April 27, 2022 /PRNewswire/ -- 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 United Kingdom Medicines & Healthcare products Regulatory Agency (MHRA) has accepted Alterity's clinical trial authorisation (CTA) request to conduct its Phase 2 clinical trial for ATH434 in Multiple System Atrophy (MSA), a rare and highly debilitating Parkinsonian disorder.

"Approval by the MHRA in the UK is another important step forward for our ATH434 clinical development program," said David Stamler, M.D., Chief Executive Officer, Alterity. "We expect to open our first Phase 2 clinical trial site in New Zealand this quarter and then expand the trial globally in the UK, other European countries, Australia and the United States. We look forward to bringing this potential therapy to individuals with MSA who currently have no treatments to address the underlying pathology of their disease."

The Phase 2 clinical trial is a randomized, double-blind, placebo-controlled investigation of ATH434 in patients with early-stage MSA. The study will explore the effect of ATH434 treatment on imaging and protein biomarkers such as aggregating α-synuclein and excess iron, which are important contributors to MSA pathology. Clinical endpoints and other biomarkers will permit comprehensive assessment of ATH434 efficacy along with characterization of safety and pharmacokinetics. Patients 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.

About 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 preclinically to reduce α-synuclein pathology and preserve nerve cells by restoring normal iron balance in the brain. In this way, it has excellent potential to treat Parkinson's disease as well as various forms of atypical Parkinsonism such as Multiple System Atrophy (MSA). ATH434 has successfully completed a Phase 1 clinical trial demonstrating the agent is well tolerated, orally bioavailable, and achieved brain levels comparable to efficacious levels in animal models of MSA, with the objective of restoring function in patients with MSA and other Parkinsonian disorders.

ATH434 has been granted Orphan 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 approximately 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]

^[1]National Institute of Health: Neurological Disorders and Stroke, Multiple System Atrophy Fact Sheet

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.

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 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.

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SOURCE Alterity Therapeutics Limited

FAQ

What is ATH434 and its significance in treating Multiple System Atrophy?

ATH434 is a novel treatment aiming to inhibit protein aggregation linked to neurodegeneration, specifically targeting MSA, which currently has no effective therapies.

What are the details of the Phase 2 trial for ATH434?

The Phase 2 trial is a randomized, double-blind, placebo-controlled study that will assess the efficacy and safety of ATH434 in MSA patients over 12 months.

When will the Phase 2 trial for ATH434 commence?

The first trial site is expected to open in New Zealand this quarter, with plans to expand globally thereafter.

What is the role of the MHRA in the approval of clinical trials?

The MHRA regulates medicines and clinical trials in the UK, ensuring that trials are safe, ethical, and scientifically valid.

How might ATH434 impact the market for neurodegenerative disease treatments?

If successful, ATH434 could offer a new treatment option for MSA patients, potentially capturing market share in a segment with currently no disease-modifying therapies.