Alterity Therapeutics has launched the Phase II clinical trial of its lead candidate, ATH434, to treat patients with multiple system atrophy (MSA), a rare neurodegenerative disease, in New Zealand. 

The trial is currently open for subject enrolment, the company noted. 

A small molecule, ATH434 is intended to lower the toxic build-up of α-synuclein, a pathological hallmark of MSA and protect nerve cells by reinstating normal iron balance in the brain. 

The double-blind, placebo-controlled, randomised trial will analyse ATH434 in subjects with early-stage MSA. 

It will enrol nearly 60 adult subjects, who will be given one of two doses of ATH434 or a placebo. 

The trial will analyse the impact of the treatment with ATH434 on imaging and protein biomarkers, such as aggregating α-synuclein and excess iron, which are key contributors to MSA pathology. 

Clinical and biomarker endpoints will aid in a thorough analysis of ATH434’s efficacy and characterisation of safety and pharmacokinetics. 

Trial subjects will be treated for a year, offering a chance to identify changes in efficacy endpoints to streamline the design of a definitive Phase III trial. 

Alterity Therapeutics CEO David Stamler said: “The advancement of our ATH434 programme underscores our commitment to bring a much-needed treatment to individuals with MSA. 

“The opening of enrolment for our Phase II in New Zealand is an important first step for this clinical programme and I am grateful to our team and our investigators who supported the launch of the trial. 

“Over the course of this year, we will expand the trial into other regions globally.”

In a Phase I trial, ATH434 was found to be well-tolerated, orally bioavailable and attained brain levels in line with efficacious levels in animal models of MSA, with the goal of resonating function in people with MSA and other Parkinsonian disorders.