Australian company Alterity Therapeutics has announced positive interim data from the Phase 2 clinical trial of ATH434 in patients with multiple system atrophy.
Multiple System Atrophy (MSA) is a rare, neurodegenerative disease characterised by failure of the autonomic nervous system and impaired movement. The symptoms reflect the progressive loss of function and death of different types of nerve cells in the brain and spinal cord. It is a rapidly progressive disease and causes profound disability.
In preclinical studies, ATH434 has been shown to reduce α-synuclein pathology and preserve neuronal function by restoring normal iron balance in the brain.
The interim data analysis from the phase 2 trial included clinical and biomarker data on seven participants treated with ATH434 for six months and neuroimaging data on three participants treated for 12 months.
The company said that after six months of treatment, 43 per cent of participants showed improvement on the UMSARS Activities of Daily Living Scale, indicating reduced disability. Over the same period, 29 per cent of participants had stable or improved neurological symptoms as assessed by the treating physician and the patient.
"I am very encouraged by these positive interim data in advanced MSA patients," said Alterity CEO Dr David Stamler.
"As MSA is a rapidly progressive and unremitting disease, we expected to see decline in all participants. Instead, we saw favorable clinical and biomarker outcomes in some patients suggesting that ATH434 has the potential to modify the course of this devastating condition. We were also very pleased to see that the clinical responders had biomarker evidence of stable disease as this provides an objective indication of potential efficacy."
Dr Stamler continued, "In the ATH434-202 trial, the participants who stabilised or improved with ATH434 treatment had less advanced disease than those who progressed. This is noteworthy as we have enrolled earlier-stage MSA patients in our randomised, double-blind clinical trial ATH434-201.
"Although the number of patients studied thus far is small, the new data reinforces that we have taken the right approach in our randomized trial and increases my overall confidence in the ATH434 development program."
Final, 12-month data from the ATH434-202 trial are expected in the first half of 2025.
