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First in class treatments for Parkinsonian diseases

ATH434-201 Phase 2 Clinical Trial in Early-Stage Multiple System Atrophy

The ATH434-201 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 clinical endpoints to demonstrate efficacy along with neuroimaging and protein biomarkers to demonstrate target engagement, and 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 aims 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.

ATH434 has been granted Orphan Drug designation for the treatment of MSA by the U.S. FDA and the European Commission. This designation entitles Alterity to seven years of market exclusivity in the U.S. and ten years of market exclusivity in the EU and qualifies Alterity for various development incentives, including U.S. tax credits for qualified clinical testing, reduced fees and access to EU-funded research grants. Learn more here.

Additional information on the Phase 2 trial can be found by ClinicalTrials.gov Identifier: NCT05109091.

Learn more about Multiple System Atrophy.

ATH434-202: A Biomarker Study of ATH434 in Participants with MSA

The ATH434-202 clinical trial is an open-label, single arm study in individuals with more advanced MSA than that being investigated in the randomized study. The study will assess the effect of ATH434 treatment on neuroimaging and protein biomarkers to evaluate target engagement, in addition to clinical measures, safety, and pharmacokinetics. Clinical measures important in MSA will also be assessed. ATH434-202 study participants will receive treatment with ATH434 for 12-months. The design of the Biomarker study will allow Alterity to perform interim analyses of biomarker data during conduct, with potential to provide early indications of efficacy before the randomized study reads out.

Additional information on the open label Phase 2 trial can be found at clinicaltrials.gov NCT05864365.

bioMUSE Natural History Study in MSA

Biomarkers of progression in Multiple System Atrophy (bioMUSE) is an ongoing natural history study that aims to track the progression of patients with MSA, a Parkinsonian disorder without approved therapy.  The study is being conducted in collaboration with Vanderbilt University Medical Center in the U.S. under the direction of Daniel Claassen, MD, MS, Professor of Neurology and Division Chief Behavioral and Cognitive Neurology. Natural history studies are important for characterizing disease status in selected patient populations.

The study of approximately 20 patients has provided rich data for optimizing the design of Alterity’s Phase 2 clinical trial. The study will continue to offer vital information on early stage MSA patients, inform the selection of biomarkers suitable to evaluate target engagement and preliminary efficacy, and deliver clinical data to characterize disease progression in a patient population that mirrors those to be enrolled in the Phase 2 clinical trial.

Learn more about Multiple System Atrophy.

ATH434 Nonclinical Studies in Parkinson’s Disease

In collaboration with Vanderbilt University Medical Center and the Florey Institute of Neuroscience, we are conducting several nonclinical studies with ATH434 for use in Parkinson’s disease based on imaging of brain iron, preservation of neurons, and improvements in motor performance. A study funded by the Michael J. Fox Foundation for Parkinson’s Research is evaluating the pharmacologic effect of ATH434 in a primate model that may help us determine the optimal dose of ATH434 in future Parkinson’s disease clinical trials.

Learn more about Parkinson’s disease.

Drug Discovery

We maintain an active drug discovery program to generate patentable compounds to treat a variety of neurodegenerative diseases.

Learn more about our Drug Discovery efforts.

Our Target: Parkinsonian Diseases

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

1Multiple System Atrophy | National Institute of Neurological Disorders and Stroke (nih.gov)

Parkinson’s disease

Parkinson’s disease (PD) is the second most common neurodegenerative disorder and causes unintended or uncontrollable movements of the body along with neuropsychiatric and other nonmotor features. The precise cause of PD is unknown, but some cases are hereditary while others are thought to occur from a combination of genetics and environmental factors that trigger the disease. In PD, brain cells become damaged or die in the substantia nigra, the part of the brain that produces dopamine–a chemical needed to produce smooth, purposeful movement. The cardinal symptoms of PD are tremors, rigidity, slowing of movements, and later in disease, impaired balance. Other symptoms may include difficulty swallowing, chewing, or speaking; emotional changes; urinary problems or constipation; dementia or other cognitive problems; fatigue; and problems sleeping.1 Nearly one million people in the U.S. and more than 10 million people worldwide are living with PD. Approximately 60,000 Americans are diagnosed with PD each year.2

1National Institute of Health: Neurological Disorders and Stroke, Parkinson’s Disease Information Page; 2Parkinson’s Foundation

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