Clinical EfficacyPhase 2 data showed ATH434 produced a clinically meaningful reduction in disease severity on the UMSARS scale and stabilized orthostatic hypotension versus decline on placebo, supporting potential symptomatic benefit in multiple system atrophy.
Safety And Development RiskATH434 was well tolerated in the study with no treatment-related serious adverse events reported, which may reduce safety concerns ahead of later-stage trials.
Target Engagement (neuroimaging)Neuroimaging analyses demonstrated reduced iron accumulation in the globus pallidus and other affected brain regions, providing mechanistic evidence that ATH434 engages its intended target.