Alterity Therapeutics has disclosed results from its Phase 2 ATH434-201 clinical trial in patients with Multiple System Atrophy (MSA), according to the company. The randomized, double-blind, placebo-controlled study evaluated two doses of ATH434 (50 mg and 75 mg twice daily) over 12 months in 77 adults.
The data presented at the American Neurological Association Annual Meeting showed that ATH434 produced clinically and statistically significant improvement compared to placebo on the UMSARS Part I scale, a measure of daily living disability in MSA. The company said that both doses reduced iron accumulation in affected brain regions and there were trends toward preservation of brain volume.
Secondary outcomes included improvements in motor performance (Parkinson’s Plus rating), global severity assessment, orthostatic hypotension symptoms, and increased outpatient activity via wearable sensor data.
Safety findings indicated that ATH434 was well tolerated with adverse event rates similar to placebo, and no serious adverse events were attributed to the study drug. Baseline differences in disease severity between treatment arms were noted by the company as largely explaining differences in response between dose groups.
ATH434 is an oral agent designed to restore iron balance and inhibit pathological protein aggregation. It has already received Fast Track designation from the U.S. Food and Drug Administration and Orphan Drug Designation from both the FDA and the European Commission for the treatment of MSA.
