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Therapeutics

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ATH434

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Phase 3-ready iron-chaperone program targeting iron dysregulation and alpha-synuclein pathology in multiple system atrophy.

Modality
oral small-molecule iron chaperone
Availability (US)
Phase 2 program completed; pivotal Phase 3 MSA trial activities planned for year-end 2026 and not yet initiated
Biomarkers
labile brain iron; quantitative susceptibility mapping MRI; alpha-synuclein aggregation; UMSARS Part I; neurofilament light
Hallmark targeted
loss of proteostasis, metal dyshomeostasis, mitochondrial dysfunction
Kind
investigational drug
Clinical trials
NCT05109091 — completed Phase 2 ATH434-201 study; NCT05864365 — completed Phase 2 biomarker study; NCT07729852 — post-trial access, Not yet recruiting