Alterity Therapeutics (ASX: ATH) has announced positive topline data from its Phase 2 clinical trial of ATH434, involving ten patients with advanced multiple system atrophy (MSA). Alterity Therapeutics is a pharmaceutical company dedicated to developing disease-modifying treatments for neurodegenerative diseases. The company focuses on developing therapies to treat diseases like Parkinson’s and Alzheimer’s.
The ATH434-202 trial results indicated that the oral drug provided a clinical benefit in areas affected by MSA and stabilised key neurological biomarkers associated with the disease’s pathology. The 12-month trial showed that disease progression was reduced by approximately 50%, measured by the Modified Unified MSA Rating Scale Part I (UMSARS I), compared to historical controls. Additionally, 30% of patients experienced a stabilisation of neurological symptoms, an unexpected outcome in advanced MSA cases.
Neuro-imaging data suggested that ATH434 could slow brain atrophy in advanced patients compared to placebo-treated participants from Alterity’s previous ATH434-201 study. The drug also led to reduced iron accumulation compared to placebo-treated patients, further confirming target engagement. Chief executive officer David Stamler said the results confirmed the efficacy observed in the ATH434-201 study and support advancing the ATH434 program in MSA.
MSA is a rare, progressive neurodegenerative disease characterised by autonomic nervous system failure and impaired movement due to nerve cell loss in the brain and spinal cord. While some symptoms can be managed, there are currently no approved therapies to slow or cure the disease. Alterity’s research has also been featured in a peer-reviewed journal for an innovative neuroimaging measure, highlighting the importance of advanced neuroimaging methods in evaluating MSA.
