Alterity Therapeutics Launches ATH434 Phase 2 Clinical Trial in the United States for the Treatment of Individuals with Multiple System Atrophy

U.S. now open for enrollment of rare, rapidly progressive Parkinsonian Disorder

MELBOURNE, Australia and SAN FRANCISCO, Jan. 9, 2023 /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 enrollment at Vanderbilt University Medical Center in Nashville, Tennessee.

"Vanderbilt University has been a tremendous partner for our ATH434 clinical development program, and we are very excited to utilize their expertise as our first U.S. trial site," said David Stamler, M.D., Chief Executive Officer, Alterity. "ATH434 is a potential disease modifying treatment for individuals living with MSA, a rapidly progressive Parkinsonian disorder with no approved treatment. The opening of enrollment in the U.S. is a major milestone for this trial and we are now actively recruiting patients in three regions: Europe, Asia-Pacific, and the U.S. We expect to add additional sites in the U.S. and Europe over the next few months to increase access to the study."

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^[1].

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 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^[2]

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.^[3]

Authorisation & Additional information

This announcement was authorized by David Stamler, CEO of Alterity Therapeutics Limited.

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References

1. ^{^} ClinicalTrials.gov Identifier: NCT05109091 (clinicaltrials.gov)
2. ^{^} Multiple System Atrophy Fact Sheet (www.ninds.nih.gov)
3. ^{^} www.alteritytherapeutics.com. (www.alteritytherapeutics.com)
4. ^{^} https://www.prnewswire.com/news-releases/alterity-therapeutics-launches-ath434-phase-2-clinical-trial-in-the-united-states-for-the-treatment-of-individuals-with-multiple-system-atrophy-301716116.html (www.prnewswire.com)