Alterity Therapeutics Receives U.S. FDA Approval for Investigational New Drug Application for ATH434 for the Treatment of Multiple System Atrophy

Rhea-AI Summary

Alterity Therapeutics has received FDA approval for its Investigational New Drug (IND) application for ATH434, intended for the treatment of Multiple System Atrophy (MSA), a rare neurodegenerative disease. The Phase 2 clinical trial will involve approximately 60 patients and aims to evaluate the efficacy and safety of ATH434 over 12 months. ATH434 has shown promise in preclinical studies, demonstrating the ability to reduce α-synuclein pathology and restoring iron balance in the brain. This milestone opens avenues for advancing treatment options for MSA, which currently lacks disease-modifying therapies.

Positive

• FDA approval of IND for ATH434 allows Phase 2 trial in the U.S.
• ATH434 has shown preclinical efficacy in reducing α-synuclein pathology.
• The trial will involve approximately 60 adult patients, facilitating crucial data collection.
• ATH434 has received Orphan designation from the FDA and European Commission.

Negative

• None

Regulatory Authorization Granted to Proceed with ATH434 Phase 2 Clinical Trial

MELBOURNE, Australia and SAN FRANCISCO, Sept. 20, 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 approval of its Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) to allow evaluation of ATH434 in individuals with Multiple System Atrophy (MSA), a rare and highly debilitating Parkinsonian disorder. The approval of this IND authorizes Alterity to conduct its Phase 2 clinical trial in the U.S.

"The FDA's acceptance of our IND to study ATH434 in individuals with MSA is another important milestone in the development of a much-needed treatment for this devastating condition," said David Stamler, M.D., Chief Executive Officer, Alterity. "We have been working closely with leading MSA physicians in the U.S. and expect to open our first clinical trial site for enrolment in the fourth quarter of this year. This complements our ongoing progress in other regions around the world."

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 here: ClinicalTrials.gov Identifier: NCT05109091.

About ATH434

Alterity's lead candidate, ATH434, is 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]

^[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.

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SOURCE Alterity Therapeutics

FAQ

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

The Phase 2 trial aims to evaluate the efficacy and safety of ATH434 in treating Multiple System Atrophy (MSA) over 12 months.

When did Alterity receive FDA approval for ATH434?

Alterity Therapeutics received FDA approval for the ATH434 IND application on September 20, 2022.

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

The Phase 2 trial is expected to enroll approximately 60 adult patients.

What is the significance of ATH434 in treating MSA?

ATH434 is designed to inhibit the aggregation of pathological proteins implicated in neurodegeneration, offering potential as a disease-modifying treatment for MSA.

What is Multiple System Atrophy (MSA)?

MSA is a rare, neurodegenerative disease characterized by autonomic dysfunction and impaired movement, affecting approximately 15,000 individuals in the U.S.