Alterity Therapeutics (ASXATH) has unveiled a new commercial assessment of its lead candidate ATH434, positioning the therapy as a potential first-in-class treatment for Multiple System Atrophy (MSA), a rare and debilitating Parkinsonian disorder with no approved therapies.
The company recently reported positive data from two Phase 2 clinical trials, including a double-blind study showing that ATH434 slowed disease progression and stabilised orthostatic hypotension, one of MSA’s most difficult-to-manage symptoms. A companion open-label trial in advanced patients reinforced these results, demonstrating consistent efficacy and a favourable safety profile.
Alterity said an independent market research and forecasting firm has estimated that ATH434 could achieve global peak sales of US$2.4 billion if approved. More than 70 per cent of neurologists surveyed said they would be “extremely likely” or “very likely” to prescribe the drug based on the Phase 2 data, highlighting the urgent need for a disease-modifying therapy in MSA.
ATH434 is an orally administered small molecule designed to redistribute excess iron in the brain, thereby reducing α-synuclein aggregation, which is a pathological hallmark of MSA. The therapy has already been granted Fast Track designation by the US Food and Drug Administration and Orphan Drug designation in both the US and Europe.
Alterity’s Chief Executive Officer, Dr David Stamler, said the assessment confirms both the scientific and commercial potential of ATH434. “We are very pleased with the outcome of the new commercial assessment for ATH434 that results in a potential worldwide peak sales opportunity in multiple system atrophy of USD $2.4 billion, if approved,” he said.