Alterity Therapeutics: Treating Neurodegenerative Diseases?

When a small firm’s stock price soars, and more than doubles, curiosity pushes us to investigate.   

Alterity Therapeutics

Yesterday the stock soared for a firm called Alterity Therapeutics (ATHE) closing at $3.43 UP $2.08. We found that this firm’s market cap is $3.72 billion which boosted our curiosity to learn more about the nature of the news that rallied the stock and the firm’s business.        

Alterity Therapeutics’ soaring stock price has come from its lead product, ATH434, which happens to be the first of a new generation of small molecules designed to inhibit the aggregation of pathological proteins implicated in neurodegeneration.

Neurodegenerative diseases are many and most of them lack effective diagnosis and treatments.

Mode of Action

Alterity’s lead drug, ATH434, has shown to reduce abnormal accumulation of α-synuclein and tau proteins in animal models of disease by restoring normal iron balance in the brain. The product has been described as having excellent potential to treat various forms of atypical Parkinsonism.

Parkinsonian disorders are a group of diseases in which individuals experience slowness of movement, stiffness and tremor; a constellation of symptoms referred to as Parkinsonism. Although the most common neurodegenerative cause of these symptoms is Parkinson’s disease there are less common, atypical forms in which patients experience parkinsonism as well as other debilitating symptoms.

Atypical Parkinsonism

Neurodegenerative causes of atypical parkinsonism include Multiple System Atrophy (MSA), Progressive Supranuclear Palsy and Dementia with Lewy Bodies, among others. The atypical forms of Parkinsonism have various symptoms which keeps them from responding well to drugs that treat the motor symptoms of Parkinson’s disease.

The company selected the MSA condition to become the first target for its lead product.   

MSA is highly debilitating and has yet to find an approved treatment.

Alterity Therapeutics in the NEWS

  • New data might have independently confirmed and extended laboratory findings and the safety profile of ATH434.
  • Alterity Therapeutics announced that new clinical and experimental pharmacology data for its lead drug candidate, ATH434, has been selected for presentation at the 2020 International Congress of Parkinson’s Disease and Movement Disorders (MDS 2020) and the American Neurological Association’s 2020 Annual Meeting (ANA 2020).
  • New animal data from the laboratory of Dr. Nadia Stefanova, Professor of Translational Neurodegeneration Research at the Medical University of Innsbruck, will be presented at ANA 2020.
  • The new data from an experiment testing ATH434 in an animal model of MSA independently confirm and extend previous findings demonstrating that ATH434 reduces α-synuclein pathology, preserves neurons and improves motor performance. 
  • ATH434 – an orally bioavailable, brain penetrant, small molecule designed to inhibit α-synuclein aggregation, is being developed for the treatment of MSA, a Parkinsonian disorder. Alpha-synuclein aggregation is implicated in the pathology of MSA and of Parkinson’s disease.

Professor Gregor Wenning, Chair of the Division of Neurobiology at Medical University of Innsbruck and Co-Founding Director of the European MSA Study Group, said, “There is a great need for new treatments of this devastating condition. The exceptional work from Dr. Stefanova’s team demonstrates the effectiveness of ATH434 in a disease-predictive animal model. I look forward to the continued progress of ATH434 into patient studies.”

Alterity will also present cardiac safety data from its Phase 1 Study of ATH434, marking it the first time such information is shared with an international group of clinicians and researchers in the field of neurological disorders.

The new safety data, which focuses on evaluating electrical activity in the heart as measured by the QT interval, reinforces previous safety findings from the Phase 1 clinical study that ATH434 was generally well tolerated at all doses and had an adverse event profile comparable to placebo in adult and older adult volunteers.

The data to be presented indicates that there is no evidence of cardiac liability at clinically tested doses. 

Alterity’s Chief Medical Officer, Dr. David Stamler, said, “Drug-induced QT prolongation can pose a significant risk for patients, so a clean bill of health on this safety measure is important as we advance to Phase 2.  In searching for new treatments to modify disease progression, we need to develop agents that are as safe as possible.”   

When paired with favorable pharmacokinetic and safety data previously reported, the new animal and clinical data support the continued development of ATH434 for MSA. The company announced last month that following discussion with the US Food and Drug Administration it had established a development pathway for ATH434 in MSA and is intending to pursue a global development strategy.

Due to global restrictions in the wake of COVID-19 this year both conferences will be held in a virtual format with MDS 2020 being held in September and ANA 2020 to be held in October. 

Prohost Observations 

The news about ATH434 is promising. The product is orally administered, safe, promising and first of a new generation of small molecules designed to inhibit the aggregation of pathological proteins implicated in neurodegeneration.

If approved, ATH434 would represent a great moment in the history for the treatment of neurodegenerative diseases.

Today, in early hours, the stock was trading at $2.98 DOWN $0.45 as a result of profit-taking.       

Prohost Biotech is looking forward to hearing the firm’s presentations at both conferences in September and October 2020.

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