Alterity Therapeutics Ltd. (ATHE) announced on Wednesday encouraging interim results from its ongoing ATH434-202 open-label Phase 2 clinical trial, targeting patients with multiple system atrophy (MSA).
MSA is a rare neurodegenerative disorder characterized by autonomic nervous system failure and impaired motor function.
Alterity's flagship compound, ATH434, is an oral medication designed to inhibit the aggregation of pathological proteins associated with neurodegeneration. Preclinical studies have shown that ATH434 can mitigate a-synuclein pathology and preserve neuronal functionality by restoring normal iron balance in the brain.
The trial has enrolled a total of 10 participants, all diagnosed with MSA through a comprehensive approach that includes clinical assessments, neuroimaging, and fluid biomarkers. These participants are being treated with 75 mg of oral ATH434, administered twice daily.
The interim analysis encompasses clinical and biomarker data from 7 participants treated with ATH434 for six months, as well as neuroimaging data from 3 participants treated for twelve months.
The interim findings indicate that among the 7 patients who completed six months of ATH434 treatment, the drug was well-tolerated with no significant safety issues identified.
Importantly, 43% of these participants exhibited improvements on the Unified Multiple System Atrophy Rating Scale (UMSARS) Activities of Daily Living, indicating reduced disability in daily activities. Additionally, 29% of participants experienced stable or improved neurological symptoms, as evaluated by both the treating physicians and patients.
Overall, ATH434 was well-tolerated by the study participants, with most adverse events being mild to moderate in severity.
The study conducted clinical, biomarker, and safety assessments throughout. While these preliminary results are promising, the company noted a positive trend in patient outcomes and plans to continue monitoring the participants closely.
