Alterity Therapeutics Receives Regulatory Authorization in France and Austria to Proceed with ATH434 Phase 2 Clinical Trial

Rhea-AI Summary

Alterity Therapeutics (NASDAQ: ATHE) has received regulatory approval to commence its Phase 2 clinical trial for ATH434, targeting Multiple System Atrophy (MSA) in Austria and France. This trial aims to assess the drug's impact on neuroimaging, protein biomarkers, and clinical endpoints over 12 months with approximately 60 participants. ATH434, an oral treatment designed to inhibit protein aggregation involved in neurodegeneration, has shown promising results in preclinical studies. With this approval, the company enhances its European presence, focusing on delivering access to this critical trial for patients suffering from MSA.

Positive

• Received regulatory approval for Phase 2 trial of ATH434 in Austria and France.
• Trial to assess efficacy through neuroimaging and protein biomarkers.
• ATH434 has shown preclinical success in reducing α-synuclein pathology.
• Expanded footprint in Europe with trials open in five countries.

Negative

• None.

MELBOURNE, Australia and SAN FRANCISCO, Feb. 16, 2023 (GLOBE NEWSWIRE) -- 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 Company has been granted authorization to conduct its Phase 2 clinical trial for ATH434 in Multiple System Atrophy (MSA) by the Austrian Federal Office for Safety in Healthcare and the French National Agency for the Safety of Medicine and Health Products. MSA is a rare and highly debilitating Parkinsonian disorder.

“The regulatory approvals in France and Austria will allow us to expand our footprint in Europe and are another important step forward for our ATH434 development program,” said David Stamler, M.D., Chief Executive Officer, Alterity. “With the trial now open in five countries we are focused on providing access to our Phase 2 clinical trial for individuals living with MSA. We expect to open trial sites in France and Austria over the next few months.”

The 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. The selected biomarkers, including brain iron and aggregating α-synuclein, are important contributors to MSA pathology and are therefore appropriate targets to demonstrate drug activity. Wearable sensors will also be employed to evaluate motor activities that are important to patients with MSA. The study is expected to enroll approximately 60 adults 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 nerve cells 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 a randomized, double-blind, placebo-controlled Phase 2 clinical trial in patients with early-stage MSA. 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

¹National 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.

Investor and Media Contacts:

Australia
Ana Luiza Harrop
we-aualteritytherapeutics@we-worldwide.com
+61 452 510 255

U.S.
Remy Bernarda
remy.bernarda@iradvisory.com
+1 (415) 203-6386

FAQ

What is the goal of Alterity Therapeutics' Phase 2 clinical trial for ATH434?

The goal is to evaluate the efficacy of ATH434 in patients with early-stage Multiple System Atrophy through neuroimaging and biomarker assessments.

When will the trial sites open in France and Austria for ATH434?

Trial sites in France and Austria are expected to open over the next few months.

How many participants will be enrolled in the Phase 2 trial of ATH434?

Approximately 60 adults will participate in the Phase 2 trial of ATH434.

What are the expected outcomes of the Phase 2 trial for ATH434?

The trial aims to demonstrate target engagement, clinical efficacy, and safety over a treatment period of 12 months.

What is the significance of ATH434 for patients with Multiple System Atrophy?

ATH434 may offer a disease-modifying treatment option for MSA, a currently untreatable and progressive neurodegenerative disease.