Altality Therapeutics (ASX:ATH) announced the completion of a Phase 2 clinical trial of its lead drug candidate, ATH434, in multiple system atrophy (MSA). Although results have not been announced, Alterity expects to announce top-line results in late January or early February 2025.

“We are very pleased to announce that the final participant in our Phase 2 trial has completed all clinical evaluations,” said Dr. David Stamler, CEO of Alterity. This is the final milestone to begin.” “This is an important achievement and we would like to thank the participants, clinicians, and research teams who are working toward potential treatments for this devastating disease.”

rare disease with no cure

MSA is a neurodegenerative disease. Approximately 2 to 5 out of 100,000 people are affected. It is caused by the gradual loss of nerve cells in various areas of the brain and spinal cord, leading to movement disorders, dysfunction of the autonomic nervous system (the part of the nervous system that controls automatic and involuntary functions), and It is characterized by a progressive disorder.

Currently, there are no treatments that can change the course of the disease.

MSA is classified as “Parkinson’s disease.” It shares many of the symptoms of Parkinson’s disease, but it tends to worsen more rapidly than Parkinson’s disease, affects more body systems, and does not respond to Parkinson’s drugs.

Although the exact cause is not fully understood, one of the hallmarks of this disorder is the accumulation of a protein called alpha-synuclein within brain cells. The proteins clump together (‘aggregate’) and damage cells.

ATH434 has been granted orphan drug designation by the US FDA and the European Commission, highlighting its potential to address this unmet medical need.

ATH434 Phase 2 study

ATH434 has demonstrated preclinical potential to preserve neurological function and reduce Parkinson’s disease pathology.

This was a double-blind, placebo-controlled study in early stage “clinically possible” MSA.

Seventy-seven patients were enrolled, and the aim of the study was to investigate clinical efficacy and safety, as well as evaluate the effects of ATH434 on neuroimaging and protein biomarkers associated with MSA. Biomarkers such as brain iron levels and alpha-synuclein protein aggregation are important targets for this drug, which is designed to restore iron balance and inhibit protein aggregation.

Participants received treatment over 12 months, allowing researchers to optimize the Phase 3 trial design.

ATH434 is also being investigated in a Phase 2 open-label biomarker study in more advanced MSA cases.

Alterity’s share price soared on news of the trial completion and is currently trading at 0.45 cents, up 12.5%.