Alterity Therapeutics (ASX: ATH, NASDAQ: ATHE), a biotechnology company dedicated to developing disease modifying treatments for neurodegenerative diseases, has today announced it has commenced a new Phase 2 clinical trial of ATH434 in patients with Multiple System Atrophy (MSA) and the first patient has been enrolled. This open label study, entitled “A Biomarker Study of ATH434 in Participants with MSA” (ATH434-202) is in addition to the ongoing, randomized, double-blind, placebo-controlled Phase 2 trial in early-stage MSA (ATH434-201).
The Biomarker trial will enroll approximately 15 individuals with more advanced MSA than those participating in the randomized trial. A key aim of the study is to assess the efficacy of ATH434 on objective biomarkers that measure target engagement and are relevant to the underlying pathology of disease. Clinical measures important in MSA will also be assessed.
“This Phase 2 Biomarker study complements our ongoing randomized Phase 2 trial, allowing us to evaluate the effect of ATH434 on two MSA populations of differing severity,” said David Stamler, M.D., Chief Executive Officer, Alterity. “Individuals with more advanced MSA may also benefit from ATH434 and measuring key biomarkers will permit us to evaluate drug activity in this population. The data derived from these two trials will help us optimize and potentially accelerate future development.”
“Importantly, the randomized Phase 2 trial is proceeding as planned, but the design of this new study will allow us to perform interim analyses of biomarker data during conduct, which could give us early indications of efficacy. We expect to have preliminary data from an initial cohort of enrolled participants before the randomized study reads out,” added Dr. Stamler.
ATH434-202 study participants will receive treatment with ATH434 for 12-months. The study will assess the effect of ATH434 treatment on neuroimaging and protein biomarkers to evaluate target engagement, in addition to clinical measures, safety, and pharmacokinetics. The selected biomarkers, including brain iron and aggregating α-synuclein, are important contributors to MSA pathology and are appropriate targets to demonstrate drug activity. The primary objective of this study is to evaluate the impact of 12 months treatment with ATH434 on brain iron by MRI (QSM/R2*) in a more advanced patient population than is being studied in Alterity’s randomized Phase 2 trial. Additional information on the open label Phase 2 trial can be found at clinicaltrials.gov NCT05864365.
About ATH434
Alterity’s lead candidate, ATH434, is an oral agent designed to inhibit the aggregation of pathological proteins implicated in neurodegeneration. ATH434 has been shown preclinically to reduce α-synuclein pathology and preserve nerve cells by restoring normal iron balance in the brain. As an iron chaperone, it has excellent potential to treat Parkinson’s disease as well as various Parkinsonian disorders such as Multiple System Atrophy (MSA). ATH434 successfully completed Phase 1 studies demonstrating the agent is well tolerated and achieved brain levels comparable to efficacious levels in animal models of MSA. ATH434 is currently being studied in two clinical trials: Study ATH434-201 is a randomized, double-blind, placebo-controlled Phase 2 clinical trial in patients with early-stage MSA and Study ATH434-202 is an open-label Phase 2 Biomarker trial in patients with MSA. ATH434 has been granted Orphan drug designation for the treatment of MSA by the U.S. FDA and the European Commission.
Alterity Therapeutics is a clinical stage biotechnology company dedicated to creating an alternate future for people living with neurodegenerative diseases. The Company’s lead asset, ATH434, has the potential to treat various Parkinsonian disorders. Alterity also has a broad drug discovery platform generating patentable chemical compounds to intercede in disease processes. The Company is based in Melbourne, Australia, and San Francisco, California, USA.
