Alterity Therapeutics (ASX:ATH) surged by as high as 40% this morning before paring back to 20% –  after announcing it has received FDA approval for an Investigational New Drug (IND) to conduct clinical trials for its lead drug, ATH434.

The trial will study the use of ATH434 in the treatment of Multiple System Atrophy (MSA) in the United States.

ATH434 is the first of a new generation of small molecules designed to inhibit the aggregation of pathological proteins implicated in neurodegeneration.

Specifically, the drug aims to reduce the toxic accumulation of α-synuclein, a pathological hallmark of MSA, and preserve nerve cells by restoring normal iron balance in the brain.

The global Phase 2 clinical trials for ATH434 are already underway with sites opened in New Zealand – with first patient dosed in July – as well as the UK. The company has also received approval to expand the trial to Italy.

The study in the US will be a randomised, double-blind, placebo-controlled investigation that will explore the effect of ATH434 treatment on neuroimaging and protein biomarkers, such as excess brain iron and aggregating α-synuclein, which are important contributors to MSA pathology.

It is expected to enroll approximately 60 adult patients to receive one of two dose levels of ATH434 or placebo.

Patients will receive this treatment for 12 months which will provide an opportunity to detect changes in efficacy endpoints to optimise the design for a potential Phase 3 study.

Previous studies have shown that ATH434 has excellent potential to treat Parkinson’s disease as well as less common Parkinsonian disorders such as MSA.

MSA is a rare, degenerative neurological disease characterised by failure of the involuntary (autonomic) nervous system and impaired movement that affects up to five out of every 100,000 people globally. It is a rapidly progressive disease and causes profound disability.

Alterity’s program could be the first approved treatment for MSA that is neuroprotective, meaning it preserves nerve cells, maintains function and slows the progression of disease.

Alterity holds a strong intellectual property portfolio, with over 800 validated compounds from its chemical library.

The company was recently granted new US patents covering 230 novel compounds as next generation therapies targeting major neurodegenerative diseases.



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