ASX biotech Alterity edges closer to first treatment for rare neurodegenerative disease

An ASX-listed biotech is pushing the boundaries of neurodegenerative research with the development of an AI-powered neuroimaging tool designed to measure disease progression in patients with Multiple System Atrophy (MSA) – a highly debilitating and rapidly progressive rare disease.

MSA patients typically survive around 7.5 years after symptom onset, with more than half requiring a wheelchair within five years. There are currently no approved disease-modifying treatments for this neurodegenerative disease.

Alterity Therapeutics’ lead candidate, ATH434, has shown early promise in Phase 2 trials, and this new imaging development adds fuel to the company’s momentum ahead of a pivotal Phase 3 program.

“This research used state-of-the-art technology employed in the bioMUSE study that goes above and beyond traditional MRI methods for assessing brain volume in patients with MSA,” said Dr David Stamler, CEO of Alterity.

“Importantly, we observed that statistically significant reductions in brain volume over 12 months correlated with clinical worsening of the disease. The results underscore the importance of utilising advanced neuroimaging methods and analytical tools in evaluating MSA, which we implemented in our Phase 2 clinical program.”

Patients with MSA recorded significantly lower MSA-Atrophy Index (AI) scores compared to other diagnostic groups (p < 0.001). The MSA-AI differentiated MSA from related synucleinopathies such as Parkinson’s disease and dementia with Lewy bodies (p < 0.001), correlated strongly with disease severity (p < 0.001), and predicted future progression (p = 0.035). Most notably, reductions in MSA-AI scores over one year were closely linked with worsening clinical scores (p = 0.01).

“While previous MRI studies have reported brain volume reductions in MSA affected brain regions, tracking these changes reliably has been challenging,” Dr Stamler explained. “Development of the MSA Atrophy Index can enhance the understanding of MSA progression and provide support for using brain atrophy markers for the evaluation of disease-modifying therapies. These tools offer potential applications in diagnosis, staging, and monitoring of disease severity, contributing to more personalised care in MSA. We look forward to leveraging this invaluable technology for patient selection and disease progression in our Phase 3 clinical trial.”

The company’s AI neuroimaging measure complements promising data from its Phase 2 ATH434-201 study, presented recently at the American Academy of Neurology (AAN) meeting.

At the 50 mg dose, ATH434 reduced clinical progression by 48% versus placebo on the modified UMSARS I scale (p = 0.02), while the 75 mg dose saw a 30% reduction over 52 weeks. On the Clinical Global Impression of Severity Scale, the 50 mg dose yielded a nominally significant improvement (p = 0.0088). Wearable sensor data revealed increased patient activity at both dose levels, with step count gains at 50 mg reaching nominal significance (p < 0.05).

Alterity is preparing for its end-of-Phase 2 meeting with the U.S. FDA, an inflection point in defining the regulatory path forward for a Phase 3 trial of ATH434.

In relation to the next steps locally, the company told Health Industry Hub, “We’re excited to re-engage with our extensive network of trial sites worldwide, including our established partners in Australia. With the final Phase 3 trial design soon to be confirmed, we look forward to discussions with the TGA regarding the optimal regulatory pathways for approval.”

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