Alterity Therapeutics Announces Positive ATH434 Phase 2 Trial Results in Multiple System Atrophy Led By Robust Clinical Efficacy

January 30, 2025 Alterity Therapeutics

– Clinically Meaningful Benefit Observed at Both ATH434 Doses Studied –

– Achieved Statistical Significance with Up to 48% Slowing of Clinical Progression on UMSARS Rating Scale –

–  Key MRI Biomarker Shows Iron Stabilization in MSA Affected Brain Regions –

–  ATH434 Demonstrated a Favorable Safety Profile –

Excerpt from the Press Release:

MELBOURNE, AUSTRALIA AND SAN FRANCISCO, USA – 30 January 2025:  Alterity Therapeutics (ASX: ATH, NASDAQ: ATHE) (“Alterity” or “the Company”), a biotechnology company dedicated to developing disease modifying treatments for neurodegenerative diseases, today announced positive topline results from the ATH434-201 randomized, double-blind, placebo-controlled Phase 2 clinical trial in patients with early-stage multiple system atrophy (MSA).

The topline data showed that ATH434 produced clinically and statistically significant improvement on the modified UMSARS Part I, a functional rating scale that assesses disability on activities of daily living affected in MSA¹. On this important clinical measure, ATH434 demonstrated 48% slowing of clinical progression at the 50 mg dose (p=0.03)^ and 29% slowing of clinical progression at the 75 mg dose (p=0.2) at Week 52 when compared with placebo. The 75 mg dose group showed a 62% slowing of progression (p=0.05) at Week 26. In addition to the robust efficacy demonstrated on the UMSARS I, trends of improved motor performance were observed on the Parkinson’s Plus rating scale² and overall benefit was shown on the Clinical Global Impression of Severity at the 50 mg dose (p=0.009).     

Biomarkers were used to evaluate potential drug effect and target engagement. Regarding iron content by MRI, the 50 mg dose reduced iron accumulation in MSA affected brain regions (substantia nigra, putamen, and globus pallidus) and the 75 mg dose reduced iron accumulation in the globus pallidus.

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