Alterity Therapeutics Initiates ATH434 Phase 2 Clinical Trial for the Treatment of Patients With Multiple System Atrophy

Multiple system atrophy is a rare, rapidly progressive neurodegenerative disease that causes profound disability


Melbourne, Australia, June 2, 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 enrollment in New Zealand.



SMA is a rare neurodegenerative disease. Although it resembles Parkinson’s disease, MSA progresses faster and leads to profound disability. In addition to the characteristic motor symptoms of Parkinson’s disease, SMA is manifested by more severe impairment of the autonomic nervous system resulting in bladder dysfunction and the inability to maintain normal blood pressure, as well as uncoordinated movements or awkwardness that contribute to the fall. Currently available treatments only address certain symptoms of SMA. There are no therapies that slow the progression of the disease and there is no cure.


ATH434 is designed to reduce toxic accumulation of α-synuclein, a pathological hallmark of MSA, and preserve nerve cells by restoring normal iron balance in the brain. Therefore, ATH434 has the potential to address underlying disease pathology and preserve function in people with MSA.


“The advancement of our ATH434 program underscores our commitment to providing much-needed treatment for people with MSA,” said david stamler, MD, Chairman and CEO, Alterity. “The opening of registrations for our Phase 2 in New Zealand is an important first step for this clinical program, and I am grateful to our team and our investigators who supported the launch of the trial. During this year, we will extend the trial to other regions of the world. »


About the ATH434 Phase 2 Clinical Trial


The Phase 2 clinical trial is a randomized, double-blind, placebo-controlled study of TH434 in patients with early-stage SMA. The study will explore the effect of ATH434 treatment on imaging and protein biomarkers, such as α-synuclein aggregation and excess iron, which are important contributors to MSA pathology. Clinical endpoints and biomarkers, including the use of wearable sensors, will allow a comprehensive evaluation of ATH434 efficacy as well as characterization of safety and pharmacokinetics. The use of wearable sensors will allow the 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 doses of ATH434 or a placebo.


Patients will receive treatment for 12 months, giving them the opportunity to detect changes in efficacy parameters to optimize the design of a definitive phase 3 study. Additional information about the phase trial 2 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 preclinically shown to reduce α-synuclein pathology and preserve nerve cells by restoring normal iron balance in the brain. In this way, it has excellent potential for treating Parkinson’s disease as well as various forms of atypical parkinsonism such as multiple system atrophy (MSA). ATH434 successfully completed a Phase 1 clinical trial demonstrating that the agent is well tolerated, orally bioavailable, and reaches brain levels comparable to effective levels in animal models of MSA, with the goal of restoring function in patients with MSA and other parkinsonian disorders.


ATH434 has been granted orphan designation for the treatment of MSA by the US FDA and European Commission


About Multiple System Atrophy


Multiple system atrophy (MSA) is a rare neurodegenerative disease characterized by autonomic nervous system failure and impaired movement. Symptoms reflect the progressive loss of function and death of different types of nerve cells in the brain and spinal cord. It is a disease that progresses rapidly and leads to profound disability. SMA is a parkinsonian disorder characterized by a variable combination of slowed movements and/or rigidity, autonomic instability that affects involuntary functions such as maintenance of blood pressure and bladder control, and poor balance. and/or impaired coordination that predispose to falls. A pathological hallmark of MSA is the accumulation of the protein α-synuclein in the glia, central nervous system support cells, and the loss of neurons in several regions of the brain. MSA affects approximately 15,000 people in the United States, and although some of the symptoms of MSA can be treated with medication, there is currently no medication that can slow the progression of the disease and there is no cure. .1


1National Institute of Health: Neurological Disorders and Strokes, Multiple System Atrophy Fact Sheet


About Alterity Therapeutics Limited


Alterity Therapeutics is a clinical-stage biotechnology company dedicated to creating an alternative future for people 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 intervene in disease processes. The Company is based in Melbourne, Australiaand San Francisco, California, UNITED STATES. For more information, please visit the Company’s website at www.alteritytherapeutics.com.


Authorization and additional information


This announcement has been authorized by david stamlerCEO 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 attempted to identify these forward-looking statements by using terms such as that “expects”, “intends”, “hopes”, “anticipates”, “believes”, “could”, “can”, “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 these forward-looking statements are described in the sections entitled “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: statements relating to the Company’s drug development program, including but not limited to , the initiation, progress and results 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. These statements involve risks and uncertainties, including, but not limited to, risks and uncertainties relating to difficulties or delays in the financing, development, testing, regulatory approval, production and commercialization of Company’s drug components, including but not limited to ATH434, uncertainties related to the impact of the novel coronavirus (COVID-19) pandemic on the company’s business, operations and employees, the company’s ability to secure additional future sources of funding, unexpected adverse side effects, or inadequate therapeutic efficacy of drug compounds, including but not limited to ATH434, which could slow or prevent the bringing products to market, the uncertainty of obtaining patent protection for the Company’s intellectual property or trade secrets, the uncertainty of enforcing c success of the Company’s patent rights and the uncertainty of the Company’s freedom to operate.


Any forward-looking statement we make in this press release is based solely on information currently available to us and speaks only as of the date on which it is made. We undertake no obligation to update publicly any forward-looking statements, 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

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