Alterity Therapeutics is proceeding with its Phase II trial of ATH434 in Multiple System Atrophy (MSA) after it received acceptance from the UK Medicines & Healthcare products Regulatory Agency (MHRA) of its clinical trial authorization (CTA) request.
The randomised, double-blind, placebo-controlled study will analyse the company’s lead candidate, ATH434, in patients with early-stage MSA, a rare, neurodegenerative disease.
The study will explore the candidate’s treatment effect on imaging and protein biomarkers such as aggregating α-synuclein and excess iron, the key contributors to MSA pathology.
Clinical endpoints and other biomarkers will enable assessment of the therapy’s efficacy, along with safety and pharmacokinetics’ characterisation.
In this trial, subjects will be given treatment for 12 months, which will help detect changes in efficacy endpoints.
This will enable to optimise design of a definitive Phase III clinical trial.
Alterity Therapeutics CEO David Stamler said: “Approval by the MHRA in the UK is another important step forward for our ATH434 clinical development programme.
“We expect to open our first Phase II clinical trial site in New Zealand this quarter and then expand the trial globally in the UK, other European countries, Australia and the United States.
“We look forward to bringing this potential therapy to individuals with MSA who currently have no treatments to address the underlying pathology of their disease.”
ATH434, which has been designed to prevent aggregation of pathological proteins implicated in neurodegeneration, received Orphan designation from the US Food and Drug Administration and the European Commission to treat MSA.
Autonomic nervous system failure and impaired movement cause MSA and its symptoms reflect the progressive loss of function and various nerve cells death in the brain and spinal cord.