ATH434 Shows 48% Reduction in Disease Progression: Key Data From Alterity Therapeutics’ Phase 2 Trial

Slowing Multiple System Atrophy Progression by Nearly Half

Alterity Therapeutics (NASDAQ: ATHE, ASX: ATH) presented standout data from its ATH434-201 Phase 2 trial at the 2025 International Congress of Parkinson's Disease and Movement Disorders. In patients with Multiple System Atrophy (MSA), the investigational therapy ATH434 slowed disease progression by a striking 48% at the 50 mg dose compared to placebo, based on the Unified Multiple System Atrophy Rating Scale Part I (UMSARS I) over 52 weeks. Even at the 75 mg dose, ATH434 demonstrated a substantial 30% reduction, and when accounting for baseline differences, this strengthened to 35%. These results represent some of the most robust clinical effects reported to date for a disease that currently has no treatment to slow its advance.

Stabilizing Orthostatic Hypotension: Addressing a Debilitating Symptom

One of MSA’s most challenging symptoms is orthostatic hypotension (OH), or severe drops in blood pressure upon standing. In the trial, patients receiving ATH434 at both the 50 mg and 75 mg doses experienced stability in their OH symptoms over 52 weeks, while placebo recipients saw their condition worsen by about 6 points on the OH Symptom Assessment. These findings not only demonstrate clinical efficacy but also provide real-world relief in one of MSA’s most disabling features.

Advanced Biomarker Analysis Supports Efficacy and Diagnostic Progress

ATH434’s impact extended beyond clinical symptoms. Biomarker data showed a reduction in iron accumulation within critical brain regions like the globus pallidus and substantia nigra at both dose levels, as measured by quantitative MRI techniques. Furthermore, trends towards reduced brain atrophy and improvements in wearable sensor data (such as step count and total walking time) painted a comprehensive picture of clinical and biological benefit.

Enhanced Diagnostic Confidence Through Multimodal Approaches

Researchers also highlighted how combining advanced neuroimaging with alpha-synuclein aggregation profiles significantly increased diagnostic accuracy for MSA. While cerebrospinal fluid (CSF) aggregation markers were supportive in roughly 79% of subjects, neuroimaging matched clinical expectations in over 96%—and was particularly valuable when clinical subtypes or biomarkers alone were ambiguous. Notably, MRI provided objective confirmation for clinical diagnoses in 90% of cases and offered new insight where clinical judgment fell short.

Favorable Safety and FDA Support Fuel Forward Momentum

ATH434 maintained a safety profile similar to placebo, with no serious or severe adverse events attributed to the therapy across both doses. This strengthens its standing as a viable candidate for regulatory approval, and it’s already granted Fast Track and Orphan Drug Designations by both the FDA and European Commission.

What’s Next for Alterity and the MSA Community?

Alterity’s robust Phase 2 results not only bolster hopes for a much-needed disease-modifying therapy in MSA, but also push the field forward with advanced biomarker and neuroimaging-driven patient stratification. Investors, researchers, and patients alike will want to follow ATH434’s continued progress as the company moves toward pivotal late-stage studies. With no cure and few treatments that even address MSA symptoms, this breakthrough has the potential to redefine the treatment paradigm.

Key Takeaway

The positive clinical, biomarker, and safety data for ATH434 significantly elevate confidence in its ability to slow progression of Multiple System Atrophy—marking a promising leap for a community in need of better answers.

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