Australian company Alterity Therapeutics (ASX:ATH) has announced encouraging results from its Phase 2 trial of ATH434, a first-in-class therapy for Multiple System Atrophy (MSA).
The company presented the data to leading clinicians and researchers at the American Neurological Association’s 150th Annual Meeting in Baltimore.
The study, known as ATH434-201, was a 12-month randomised, double-blind, placebo-controlled trial involving 77 patients. It showed that ATH434, at both 50 mg and 75 mg doses, significantly slowed disease progression compared to placebo, with a favourable safety profile. Patients treated with the therapy demonstrated meaningful improvements on the Multiple System Atrophy Activities of Daily Living scale, alongside positive trends across motor and functional measures. Importantly, biomarker analysis revealed reductions in iron accumulation in affected brain regions, which is a key pathological hallmark of the disease.
“This data continues to demonstrate meaningful clinical impact, including reduced disease severity, improved core symptoms, and maintained function in the outpatient setting,” said Alterity CEO Dr David Stamler. “The results reinforce the therapeutic potential of ATH434 as a disease-modifying therapy and are generating optimism across the medical community.”
ATH434 is designed to inhibit the aggregation of pathological proteins by restoring normal iron balance in the brain. Preclinical studies have already demonstrated its ability to reduce α-synuclein pathology and preserve neuronal function. With Fast Track designation from the US Food and Drug Administration and Orphan Drug status in both the US and Europe, the program has attracted international attention.
The company’s second Phase 2 trial, an open-label biomarker study in patients with more advanced MSA, has also delivered positive results, reinforcing the therapeutic profile of ATH434. Wearable sensor data further supported the findings, capturing increased patient activity outside the clinic – a real-world indicator of preserved independence.
MSA remains one of the most challenging neurodegenerative diseases, affecting an estimated 15,000 people in the United States alone. It is marked by profound disability, autonomic dysfunction, and limited treatment options. Despite advances in symptom management, no drug currently slows disease progression.
By demonstrating both safety and efficacy in a controlled setting, Alterity has positioned ATH434 as one of the most advanced clinical candidates in MSA. “Advancing therapies for rare neurodegenerative disorders is central to our mission,” Dr Stamler said. “We believe ATH434 offers genuine hope for patients and families living with this devastating disease.”