Alterity Therapeutics Launches ATH434 Phase 2 Clinical Trial for the Treatment of Patients with Multiple System Atrophy in Europe

Rhea-AI Summary

Alterity Therapeutics (ASX: ATH, NASDAQ: ATHE) has announced the opening of enrollment for its Phase 2 clinical trial of ATH434 for Multiple System Atrophy (MSA) in the UK. This trial aims to assess the treatment's impact on neuroimaging and protein biomarkers, targeting around 60 adult patients. ATH434 has shown promise in preclinical studies, demonstrating the ability to inhibit pathogenic protein aggregation and restore normal iron balance in the brain. MSA affects approximately 15,000 individuals in the U.S., with no current market-approved drugs to slow its progression.

Positive

• Enrollment in Phase 2 clinical trial for ATH434 is now open in the UK.
• Positive preclinical results showing ATH434's efficacy in reducing α-synuclein pathology.
• ATH434 received Orphan designation from the U.S. FDA and the European Commission.

Negative

• None.

Second region now open for enrolment for rare, rapidly progressive, neurodegenerative disease

MELBOURNE, Australia and SAN FRANCISCO, Aug. 25, 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 the Company's Phase 2 clinical trial of ATH434 for the treatment of patients with Multiple System Atrophy (MSA) is now open for enrolment in the United Kingdom. 

"Our Phase 2 clinical trial continues to progress as planned with the launch of the study in the UK for patients with MSA," said David Stamler, M.D., Chief Executive Officer, Alterity. "In a short period of time we have now commenced enrolment in our second region, and we look forward to broadening our reach over the next several months. Because MSA is a rare disease, we are committed to providing access to the trial to eligible patients globally."

About ATH434 Phase 2 Clinical Trial

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 neuroimaging and protein biomarkers, such as excess brain iron and aggregating α-synuclein, which are important contributors to MSA pathology. Clinical endpoints will permit comprehensive assessment of ATH434 efficacy along with characterization of safety and pharmacokinetics. The use of wearable sensors will allow evaluation of motor parameters that are important in patients with MSA. The study is expected to enroll approximately 60 adult patients to receive one of two dose levels of ATH434 or placebo. 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. Additional information on the Phase 2 trial can be found by ClinicalTrials.gov Identifier: NCT05109091.

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 successfully completed Phase 1 studies demonstrating the agent is well tolerated, orally bioavailable, and achieved brain levels comparable to efficacious levels in animal models of 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.

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SOURCE Alterity Therapeutics

FAQ

What is the purpose of the ATH434 Phase 2 clinical trial?

The ATH434 Phase 2 clinical trial aims to evaluate the drug's effect on neuroimaging and protein biomarkers in patients with early-stage Multiple System Atrophy (MSA).

How many patients will be enrolled in the ATH434 trial?

Approximately 60 adult patients will be enrolled in the Phase 2 clinical trial of ATH434.

What is Multiple System Atrophy (MSA)?

Multiple System Atrophy (MSA) is a rare, neurodegenerative disease characterized by the failure of the autonomic nervous system and impaired movement.

When did Alterity Therapeutics open enrollment for the ATH434 trial?

Enrollment for the ATH434 Phase 2 clinical trial in the UK was opened as of August 25, 2022.

What are the expected outcomes of the ATH434 trial?

The trial will assess ATH434's efficacy and safety over 12 months, providing valuable data for a potential Phase 3 study.