Alterity Therapeutics has announced promising topline results from its Phase 2 ATH434-202 clinical trial for multiple system atrophy (MSA). The study targeted advanced disease stages and found that ATH434 reduced disease progression and stabilized biomarkers associated with MSA pathology. The safety evaluation concluded that ATH434 was well-tolerated, with most adverse effects being mild to moderate. Analysts forecast an average target price of $11.00, indicating a potential upside of 67.94% from the current price.
Melbourne, Australia and San Francisco, USA – Alterity Therapeutics (ASX: ATH, NASDAQ: ATHE) announced promising topline results from its Phase 2 ATH434-202 clinical trial for multiple system atrophy (MSA). The open-label study, conducted on patients with advanced MSA, demonstrated significant clinical benefits and target engagement, as well as a favorable safety profile.
The trial evaluated ATH434, an oral agent designed to inhibit the aggregation of pathological proteins implicated in neurodegeneration. The study found that ATH434 reduced disease progression and stabilized key biomarkers associated with MSA pathology. Over the 12-month treatment period, the Modified Unified MSA Rating Scale Part I (UMSARS I) scores increased by 3.5 points, compared to a historical increase of 6.5 points in a similar MSA population. Additionally, 43% of participants had stable UMSARS scores, and 30% reported stable or improved neurological symptoms as assessed by the Clinical Global Impression of Change (CGIC) and Patient Global Impression of Change (PGIC) scales.
Neuroimaging biomarkers indicated that ATH434 slowed brain atrophy in MSA affected areas, as measured by the MSA Atrophy Index (MSA-AI), and reduced iron accumulation in the putamen and globus pallidus. These outcomes suggest that ATH434 engages its target effectively and supports the clinical efficacy observed in the study.
Safety evaluations showed that ATH434 was well-tolerated, with most adverse events being mild to moderate. No serious adverse events related to ATH434 were reported, and only one participant discontinued the study due to an adverse event unrelated to treatment.
Analysts forecast an average target price of $11.00, indicating a potential upside of 67.94% from the current price.
References:
[1] https://alteritytherapeutics.com/investor-centre/news/2025/07/28/alterity-therapeutics-reports-positive-topline-data-from-open-label-phase-2-clinical-trial-of-ath434-in-multiple-system-atrophy/
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