Alterity Therapeutics Ltd (ASX:ATH) has reported positive interim results from its Phase 2 clinical trial assessing the treatment of patients with neurodegenerative condition multiple system atrophy (or MSA) with drug ATH434.
The trial involves 10 participants, and the interim results relate to 7 who have been treated with ATH434 over a six-month period, with 3 of these also completing 12 months of treatment.
Reduced disability related to activities of daily living was observed among 43% of participants, assessed in this way through the unified MSA rating scale (or UMSARS) after six months of treatment.
During the same period, 29% were assessed by both the treating physician and patient as having stable or improved neurological symptoms, and were noted as ‘clinical responders’.
Importantly, the clinical responders on average had reduced accumulation of iron in the substantia nigra, putamen and globus pallidus – as seen on the MRI – as well as stable levels of NFL, a marker of axonal injury, when compared to participants who declined.
This suggests ATH434’s efficacy in reducing α-synuclein pathology and preserving neuronal function by restoring normal iron balance in the brain.
Alterity CEO Dr David Stamler said he was encouraged by the positive data.
“As MSA is a rapidly progressive and unremitting disease, we expected to see decline in all participants,” he said.
“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.”
The price of Alterity shares rose on the news. At 11:37 AEDT, they were trading at 0.6 cents, a rise of 20% since the market opened.