Alterity’s ATH434 Yields UMSARS Gains in 77-Patient Phase 2 Trial, Secures FDA Fast Track
Alterity Therapeutics’ ATH434 demonstrated clinically significant improvement on the UMSARS Part I scale in a 77-patient Phase 2 trial with 50 mg and 75 mg twice-daily dosing. The oral iron-chaperone therapy showed favorable safety, reduced brain iron accumulation by biomarker, and holds FDA Fast Track and Orphan designations for MSA.
1. Phase 2 Efficacy Results
In the ATH434-201 Phase 2 trial, 77 adults with Multiple System Atrophy received 50 mg or 75 mg twice daily or placebo for 12 months. ATH434 produced clinically and statistically significant improvements on the modified Unified Multiple System Atrophy Rating Scale Part I, which measures activities of daily living affected by MSA.
2. Safety and Biomarker Findings
ATH434 was well tolerated, with adverse event rates comparable to placebo and no serious events attributed to the drug. Biomarker assessments and advanced neuroimaging showed reduced iron accumulation in affected brain regions and trends toward preserved brain volume.
3. Regulatory Designations and Next Steps
ATH434 has received FDA Fast Track and Orphan Drug designations for MSA, highlighting accelerated development and market exclusivity benefits. Alterity plans to leverage these designations to advance ATH434 toward late-stage trials and potential approval.