Alterity Therapeutics has dosed the first patient in the UK in a Phase II clinical study to assess ATH434 in patients with early-stage Multiple System Atrophy (MSA).
ATH434, an oral agent, will prevent the aggregation of pathological proteins implicated in neurodegeneration. It was preclinically demonstrated to lower α-synuclein pathology and protect nerve cells by restoring normal iron balance in the brain.
The effect of ATH434 treatment on neuroimaging and protein biomarkers is being assessed in the randomised, double blind and placebo controlled investigational trial to show target engagement and clinical endpoints to demonstrate efficacy.
The study will also cover safety and pharmacokinetics assessments.
According to the company, the selected biomarkers, including brain iron and aggregating α‑synuclein, are significant contributors to MSA pathology and are suitable targets to show drug activity.
To assess motor activities, which are important to MSA patients, wearable sensors will also be used in the study.
The company intends to recruit around 60 adults in the study to receive one of two dose levels of ATH434 or a placebo.
Alterity will offer treatment to participants for one year to help identify changes in efficacy endpoints, as well as optimise the design of a definitive Phase III trial.
Alterity CEO Dr David Stamler said: “MSA is a rare and highly debilitating Parkinsonian disorder, and our Phase II clinical programme is designed to evaluate the effects of ATH434 in individuals with the early-stage disease with no current therapeutic treatment options.
“With sites open in five countries, the clinical trial is steadily enrolling globally, and the enrolment of our first patient in the UK expands our reach in Europe.”
