Alterity’s ATH434 Shows Promise in MSA

Alterity Therapeutics (ASX: ATH, NASDAQ: ATHE) has announced promising new data from its Phase 2 trial of ATH434 in Multiple System Atrophy (MSA), a rare neurodegenerative disease. The data, presented at the American Academy of Neurology (AAN) 2025 Annual Meeting, revealed clinically meaningful efficacy across multiple assessments. Key findings from the ATH434-201 trial include a statistically significant 48% decrease in clinical progression at the 50 mg dose (p=0.02) and a 30% decrease at the 75 mg dose on the modified UMSARS I rating scale after 52 weeks, compared to placebo. Improvements were also noted in the Clinical Global Impression of Severity, with a nominally significant difference at the 50 mg dose (p=0.0088). The Orthostatic Hypotension Symptom Assessment also showed positive trends favoring ATH434. Wearable sensor data indicated increased activity levels in patients receiving ATH434, with improvements in step count, walking bouts, and standing time. Neuroimaging data showed that ATH434 reduced iron accumulation in MSA-affected brain regions, suggesting target engagement. These findings support the potential of ATH434 to modify disease progression in MSA, a condition with limited treatment options. The company is planning to engage with regulatory authorities to advance the development of ATH434. Additional data from the bioMUSE natural history study, presented by collaborators at Vanderbilt University Medical Center, demonstrated the utility of wearable sensors in assessing outpatient activity in MSA patients. The study found correlations between sensor-derived metrics and clinical scores, reinforcing the relevance of digital outcomes in MSA trials. Alterity’s ATH434 is an oral agent designed to inhibit the aggregation of pathological proteins implicated in neurodegeneration and to restore normal iron balance in the brain.