New clinical data confirms efficacy of Alterity Therapeutics’ lead drug for treatment of multiple system atrophy

Neurodegenerative disease-focused biotech Alterity Therapeutics (ASX: ATH) has announced that new clinical and experimental pharmacology data for its lead candidate ATH434 has confirmed the drug can reduce alpha-synuclein pathology, preserve neurons and improve motor performance in patients with multiple system atrophy (MSA).

The new data was generated from an experiment testing ATH434 in an animal model of multiple system atrophy and has been selected for presentation at the 2020 International Congress of Parkinson’s Disease and Movement Disorders and the American Neurological Association’s 2020 annual meeting.

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

The cardiac data – which focuses on evaluating electrical activity in the heart as measured by the QT interval on electrocardiograms – reinforces previous findings from a Phase 1 clinical study which showed the drug 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 there is no evidence of cardiac liability at clinically-tested doses.

Unmet medical need

ATH434 is an orally bio-available, brain penetrant, small molecule inhibitor of alpha-synuclein aggregation and is being developed for the treatment of Parkinson’s-related MSA.

MSA is a rare and rapidly-progressive neurological disorder affecting adults, and has no known cause.

In addition to presenting with motor symptoms like those in Parkinson’s disease, individuals with MSA may also experience loss of ability to co-ordinate voluntary movements and impaired regulation of involuntary bodily functions such as blood pressure, bowel and bladder control.

As the condition progresses, daily activities become increasingly difficult and complications such as increased difficulty swallowing, vocal cord paralysis, progressive immobility and poor balance become more prominent.

Symptoms tend to appear after age 50 and rapidly advance, leading to profound disability.

There is currently an unmet medical need relating to treatments for the condition, with most symptoms incapable of being adequately addressed by available drugs for patients with Parkinson’s disease.

Alterity announced last month that following discussions 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.