Alterity Therapeutics (ASX:ATH) has announced the completion of the Phase 2 clinical trial in multiple system atrophy (MSA) for its lead drug candidate, ATH434. No results were released, but Alterity plans to release topline results in late January or early February 2025.
“We are very excited to announce that the last participant in our Phase 2 study has completed all clinical evaluations, the final milestone that starts the clock to reporting topline data,” said Alterity CEO Dr David Stamler. “This is a significant achievement, and we extend our gratitude to participants, clinicians, and our study team as we work towards a potential treatment for this devastating disease.”
A rare disease with no cure
MSA is a neurodegenerative disorder. It affects around 2-5 in 100,000 people. It’s caused by the gradual loss of nerve cells in different areas of the brain and spinal cord, and is characterised by impaired movement, dysfunction in the autonomic nervous system (the part of the nervous system that controls automatic, involuntary functions) and progressive disability.
There are no current therapies able to change the course of the disease.
MSA is categorised as “Parkinsonian”. It shares many of the symptoms of Parkinson’s disease, but it tends to worsen more quickly than Parkinson’s, it affects more bodily systems, and it doesn’t respond to Parkinson’s medication.
The exact cause isn’t fully understood, but one of the disorder’s hallmarks is the build-up of a protein called alpha-synuclein in brain cells. This protein clumps together (“aggregates”) and damages the cells.
ATH434 has been granted orphan drug designation by the US FDA and European Commission, highlighting its potential to address this unmet medical need.
The ATH434 Phase 2 trial
ATH434 has demonstrated preclinical potential to preserve neuronal function and reduce pathology in Parkinsonian disorders.
This was a double-blind, placebo-controlled study in early-stage “clinically probable” MSA.
77 patients were enrolled, and the purpose of the trial was to evaluate ATH434’s effects on neuroimaging and protein biomarkers associated with MSA, alongside investigating clinical efficacy and safety. Biomarkers such as brain iron levels and alpha-synuclein protein aggregation are key targets of the drug, which is designed to restore iron balance and inhibit protein aggregation.
Participants received treatment over 12 months, allowing researchers to optimise Phase 3 trial design.
ATH434 is also under investigation in a second Phase 2 open-label biomarker study for more advanced MSA cases.
Shares in Alterity jumped on news of trial completion, and are currently trading 12.5% higher at 0.45 cents.