Alterity presented new analyses from its ATH434-201 Phase 2 trial in Multiple System Atrophy during a Late Breaking Science oral session at the American Academy of Neurology annual meeting in Chicago on 22 April.
The analysis used the newly developed MSA Combined Outcome Assessment, MuSyCA, which integrates 11 items from UMSARS parts I and II to improve the detection of disease progression. According to the company, placebo participants worsened by about +9.7 points on MuSyCA over 52 weeks while ATH434 slowed progression with treatment effects ranging from −1.9 points at 75 mg to −4.0 points at 50 mg (p=0.034, relative treatment effect 41%) at week 52.
Using a mixed models for repeated measures approach, ATH434 also reduced decline on modified UMSARS Part I by −3.1 points at 75 mg (relative effect 35%) and −4.7 points at 50 mg (relative effect 53%, p=0.029). These results, together with biomarker and safety findings from prior trials, support ATH434 as a disease-modifying candidate and reinforce plans to engage regulators ahead of a planned Phase 3 pivotal trial.
ATH434 is an oral iron chaperone that the company claims reduces iron accumulation and abnormal protein aggregation and has received Fast Track and Orphan Drug designations.
