After a prespecified review by an independent Data Monitoring Committee (DMC) found no safety concerns, Alterity Therapeutics will continue its Phase 2 trial of ATH434 in patients with early-stage multiple system atrophy (MSA) as planned. MSA is a rare disease that rapidly progresses, causing significant disability.
ATH434 is an oral small molecule that targets alpha-synuclein in MSA and Parkinson’s disease. Both diseases are marked by the accumulation of alpha-synuclein, however, in MSA, the misfolded protein accumulates in glial cells, which are responsible for the production of myelin. In Parkinson’s disease, alpha-synuclein accumulates in the nerve cells.
In preclinical trials, researchers demonstrated that ATH343 protected neurons from the damaging effects of alpha-synuclein, improved motor function, and restored iron levels in the brain.
The Phase 2 trial randomly assigned 77 adults with MSA to one of two doses of ATH343 or placebo. Outcome measures include iron levels in the brain and levels of accumulated alpha-synuclein. “Participants in the trial are being dosed for 12 months; therefore, [the DMC] recommendation is an important milestone as participants able to safely tolerate ATH434 as their time on study increases,” said Alterity CEO David Stamler, MD.
The study is on track to be completed in November 2024, and topline results will be available in early 2025.
