Alterity Therapeutics (ASX:ATH) said topline data from the ATH434-202 drug candidate open-label phase two clinical trial in individuals with multiple system atrophy showed that the candidate clinical benefit on the modified unified MSA rating scale part I, and stabilized overall neurological symptoms, according to a Monday Australian bourse filing.
The key biomarker endpoint was defined as the change in brain volume from baseline to 12 months, as measured by the MSA Atrophy Index. A secondary biomarker endpoint was the change in iron content in areas affected by the disease from baseline to 12 months.
Over a 12-month treatment period, disease progression as assessed with the rating scale was reduced by around half as compared to historical controls. Further, 30% of participants reported stable neurological symptoms throughout the study. The candidate, on average, stabilized low blood pressure symptoms in study participants.
The trial evaluated a patient population with more advanced disease than was studied in the firm's double-blind phase two trial.
Neuroimaging outcomes indicated that ATH434 slowed brain atrophy in multiple system atrophy-affected areas, as measured by the MSA Atrophy Index, when compared to placebo-treated participants in Study 201.
Multiple system atrophy is a neurodegenerative disease characterized by failure of the autonomic nervous system and impaired movement.
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