ATH434-201

Oct. 5, 2026: Alterity Therapeutics (ASX: ATH, NASDAQ: ATHE) reported additional analyses from the randomized, double-blind, placebo-controlled Phase 2 ATH434-201 trial in multiple system atrophy (MSA) at the 2026 MDS Congress in Seoul. Using baseline cerebrospinal fluid neurofilament light chain (CSF NfL) as a disease-severity covariate, ATH434 50 mg significantly slowed functional decline versus placebo on the 11-item UMSARS Part I at Week 52 in the mITT population (N=61), with a -4.64-point difference in change from baseline (p=0.032), described as about 52% slowing. Baseline CSF NfL significantly predicted decline: each 1,000 pg/mL increase was associated with about 0.9 additional UMSARS worsening points at Week 52 (p=0.033), supporting its planned use as a covariate in Phase 3.

The 75 mg dose showed a -2.81-point difference versus placebo at Week 26 (p=0.072) and Week 52 (p=0.179). Imaging results showed the primary endpoint of change in substantia nigra iron at Week 52 was not met, though iron change was numerically lower in the putamen and globus pallidus versus placebo; dentate nucleus iron signal increased at Week 52 for 50 mg (+0.016 ppm, p=0.021). The company said it is preparing to initiate a Phase 3 pivotal MSA trial.