Alterity Therapeutics (ASX: ATH, NASDAQ: ATHE), a biotechnology company dedicated to developing disease modifying treatments for neurodegenerative diseases, has announced positive topline data from its open-label Phase 2 clinical trial of ATH434 in patients with multiple system atrophy (MSA). The ATH434-202 trial focused on individuals with more advanced disease compared to the company’s previous double-blind Phase 2 trial. ATH434 is designed to reduce α-synuclein pathology and preserve neuronal function by redistributing iron in the central nervous system.
The topline data revealed that ATH434 demonstrated a clinical benefit in key areas of impairment associated with MSA, as well as stabilizing critical biomarkers related to the disease’s pathology. Specifically, the trial showed clinical improvement based on the Modified Unified MSA Rating Scale Part I (UMSARS I). Disease progression was reduced by approximately half compared to historical controls over the 12-month treatment period, with 30% of participants reporting stable neurological symptoms. Additionally, ATH434 stabilized low blood pressure symptoms related to orthostatic hypotension in study participants.
Neuroimaging outcomes indicated that ATH434 slowed brain atrophy in MSA-affected areas, as measured by the MSA Atrophy Index (MSA-AI), compared to placebo-treated participants in a previous study. The effects on brain volume were comparable to those observed in a 75 mg dose group from the earlier study. Furthermore, ATH434 led to lower iron accumulation in the putamen and globus pallidus, providing further evidence of the drug’s target engagement. No serious adverse events related to ATH434 were reported, and the treatment was well-tolerated overall.
“I am very encouraged by the positive results from the ATH434-202 trial, as they reinforce the robust efficacy we observed in our double-blind study,” said David Stamler, M.D., Chief Executive Officer of Alterity. The company believes that ATH434 has the potential to slow the progression of this devastating disease and is committed to bringing this new therapy to patients as soon as possible. Additional information on the open-label Phase 2 trial can be found at clinicaltrials.gov (NCT05864365).
