Alterity Therapeutics (ATH) NWR Virtual Healthcare Conference summary

Program overview and disease focus

• Lead candidate ATH434 targets multiple system atrophy (MSA), aiming to slow disease progression by addressing underlying pathology, with potential for Parkinson's and other orphan diseases.

• MSA is a rapidly progressing neurodegenerative disorder with high unmet need; patients often require a wheelchair within 5 years and have a 7-8 year survival post-symptom onset.

• ATH434 works by redistributing excess iron in the brain, reducing alpha-synuclein aggregation and oxidative injury, aiming to preserve neuronal function.

• The drug is orally administered, crosses the blood-brain barrier, and has orphan drug and fast track designations in the US and EU.

• The development team has extensive experience with FDA approvals in neurology.

Clinical trial results and efficacy

• Phase II trial showed up to 48% reduction in functional decline versus placebo on the MSA rating scale, with both 50 mg and 75 mg doses effective.

• Active treatment groups showed stabilization in orthostatic hypotension symptoms and less decline in step count and walking time compared to placebo.

• Neuroimaging confirmed target engagement with reduced iron accumulation in key brain regions, especially at the 50 mg dose.

• No serious or severe adverse events related to the drug; safety profile similar to placebo.

• Trends toward less brain atrophy in treated groups, though not statistically significant due to sample size.

Commercial potential and market assessment

• Independent assessment found over 70% of surveyed US neurologists likely to prescribe ATH434 due to unmet need.

• Estimated peak global sales for MSA indication alone are $2.4 billion.

• Success in MSA could drive expansion into Parkinson's disease and other neurodegenerative disorders.