Alterity Therapeutics announces expansion of its ATH434 Phase 2 clinical development program

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– Clinical trial to recruit patients with early stage multisystem atrophy (MSA) –

– Extend bioMUSE’s natural history study –

Melbourne, Australia and SAN FRANCISCO, Oct. 19, 2021 / PRNewswire / – Alterity Therapeutics (ASX: ATH, NASDAQ: ATHE) (“Alterity” or “the Company”), a biotechnology company dedicated to the development of disease-modifying therapies for neurodegenerative diseases, has today announced an expansion of the clinical development program of the Company’s core asset, ATH434, in patients with multisystem atrophy (MSA), a rare and rapidly progressive parkinsonian disease. ATH434 has been shown to reduce the abnormal accumulation of -synuclein by restoring normal iron balance in the brain with the aim of improving motor function in patients with ASM and Parkinson’s disease.

Alterity Therapeutics Limited Logo (PRNewsfoto / Alterity Therapeutics Limited)

The Phase 2 clinical trial is a randomized, double-blind, placebo-controlled study of ATH434 in patients with early-stage MSA. The study will explore the effect of ATH434 treatment on imaging and protein biomarkers such as -synuclein aggregation and excess iron, which are important contributors to MSA pathology. Several other biomarkers and clinical parameters will allow a complete evaluation of the efficacy of ATH434 as well as a characterization of the safety and pharmacokinetics. Based on consultations with the U.S. Food and Drug Administration (FDA), European Medicines Agency (EMA), and clinical experts in MSA, Alterity has established that patients will receive treatment for 12 months within the phase 2 study. The longer treatment duration will provide a better opportunity to detect changes in biomarkers and clinical parameters in order to optimize the design of a definitive phase 3 study.

In addition, the natural history study Biomarkers of Progression in Multiple Systems Atrophy (bioMUSE) has met its initial recruitment target and will be expanded to a total of 20 patients with ASM. The study has proven invaluable in generating data to inform and reduce the risks of the Phase 2 trial design, and it will continue to provide longitudinal biomarkers and clinical data to characterize disease progression in a patient population that reflects those who will be recruited in the phase 2 study.

“With our planned Phase 2 clinical trial and the expansion of bioMUSE, we have created a robust development program to advance ATH434 for the treatment of MSA,” said David stamler, MD, CEO, Alterity. “By restoring normal iron balance in the brain, ATH434 has the potential to block -synuclein aggregation, preserve neurons, and treat the underlying pathology of AMS. this approach to alter the progression of the disease will have a profound impact on the quality of life for people living with MSA, a devastating disease with very few treatment options. “

Dr Stamler continued, “It is important to note that our Phase 2 clinical trial incorporates regulatory commentary, expert advice and essential learnings from bioMUSE to establish an optimal trial design with a better overall chance of success. . investigators and look forward to starting the trial in the first quarter of calendar year 2022. “

The double-blind phase 2 clinical trial is a three-arm study where patients with early stage MSA will be randomized to receive one of two doses of ATH434 or a placebo, given twice daily . Early-stage patients with a clinical diagnosis of ASM who are ambulatory, have no signs of severe impairment, and do not have long-standing motor symptoms will be included in the study. The trial is expected to enroll 60 early-stage patients at approximately 30 sites in Australia, New Zealand, Europe, and the United States As reported last month, initial results from the company’s bioMUSE natural history study helped guide the design of the Phase 2 clinical trial by informing patient selection and confirming that iron content in the brain is a promising biomarker. Based on this data, Alterity believes that treating patients at the early stage of their disease will offer the best chance of improvement over ATH434.

About ATH434

Alterity’s lead candidate, ATH434, is the first in a new generation of small molecules designed to inhibit the aggregation of pathological proteins involved 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 multisystem atrophy (MSA). ATH434 has successfully completed a phase 1 clinical trial demonstrating that the agent is well tolerated, orally bioavailable, and achieves 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 received orphan designation for the treatment of MSA by the US FDA and the European Commission.

About bioMUSE

Biomarkers of progression in Multiple Systems Atrophy (bioMUSE) is an ongoing natural history study that aims to track the progression of patients with MSA, a parkinsonian disease without approved treatment. The study is carried out in collaboration with Vanderbilt University Medical Center in the United States under the direction of Daniel Claassen, MD, associate professor of neurology and principal investigator. Natural history studies are important in characterizing disease progression in selected patient populations. The study provided rich data to optimize the design of Alterity’s Phase 2 clinical trial and will be expanded to include a total of 20 patients with ASM. The ongoing study will continue to provide vital information about patients with early-stage MSA, inform the selection of appropriate biomarkers to assess target engagement and preliminary efficacy, and provide clinical data to characterize the progression of the disease in a patient population that reflects those who will be recruited into the phase 2 clinical trial.

About multisystem atrophy

Multisystem atrophy (MSA) is a rare neurodegenerative disease characterized by a combination of symptoms that affect both the autonomic nervous system and movement. Symptoms reflect the gradual loss of function and death of different types of nerve cells in the brain and spinal cord. It is a rapidly progressive disease that causes profound disability. MSA is a parkinsonian disorder characterized by motor impairment, autonomic instability that affects involuntary functions such as maintaining blood pressure and bladder control, and impaired balance and / or coordination that predisposes at the falls. A pathological feature of MSA is the accumulation of the α-synuclein protein in the supporting cells of the central nervous system 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 drug that can slow the progression of the disease and there is no cure.1

About Alterity Therapeutics Limited

Alterity Therapeutics is a clinical-stage biotechnology company dedicated to creating an alternative future for people living with neurodegenerative diseases. The Company’s main asset, ATH434, has the potential to treat various forms of parkinsonian disorders. Alterity also has an extensive drug discovery platform generating patentable chemicals to intervene in disease processes. The Company is based in Melbourne, Australia, and San Francisco, California, UNITED STATES. For more information, please visit the Company’s website at www.alteritytherapeutics.com.

1National Institute of Health: Neurological Disorders and Stroke, Multiple Systems Atrophy Fact Sheet

Authorization & additional information

This ad has been authorized by David stamler, CEO of Alterity Therapeutics Limited.

Contact: Investor Relations

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 such forward-looking statements by using such words. that “expects”, “intends”, “hopes”, “anticipates”, “believes”, “could”, “could”, “evidence” and “estimates” and other expressions similar, but these words are not the exclusive means of identifying such statements.

Significant factors that could cause actual results to differ materially from those indicated in these forward-looking statements are described in the sections headed “Risk Factors” in the Company’s filings with the SEC, including its annual report on most recent on Form 20-F as well as 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 y limit, 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 which 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 marketing of the products. drug components of the Company, including, but not limited to ATH434, uncertainties regarding the impact of the novel coronavirus (COVID-19) pandemic on the business, operations and employees of the Company, the company’s ability to secure additional future sources of funding, unexpected adverse side effects or inadequate therapeutic efficacy of the Company’s drug compounds, including, but not limited to ATH434, which could slow down or prevent the marketing of products, the uncertainty of obtaining patent protection for the Company’s intellectual property or trade secrets, the uncertainty of successfully enforce the Company’s patent rights and the uncertainty of the Company’s freedom to operate.

Any forward-looking statements we make in this press release are based solely on information currently available to us and speak only as of the date on which they are made. We assume no obligation to publicly update 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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