Alterity Therapeutics Concludes Final Patient Visit in Phase 2 Trial for Early-Stage Multiple System Atrophy

Alterity Therapeutics (ASX: ATH, NASDAQ: ATHE), a biotechnology company focusing on neurodegenerative diseases, recently announced the completion of their ATH434-201 Phase 2 trial. The study is a randomized, double-blind, placebo-controlled trial involving early-stage multiple system atrophy (MSA) patients. With the last patient completing the study, topline results are anticipated by late January or early February 2025.

David Stamler, M.D., Chief Executive Officer of Alterity, expressed excitement over the milestone, emphasizing the significance of the trial's completion. He acknowledged the trial participants, clinical sites, and the study team for their dedication. Stamler highlighted the global interest from clinical sites, doctors, and patients in finding a treatment to potentially slow MSA progression. The focus now shifts to data cleaning and reporting topline results early next year.

The ATH434-201 Phase 2 trial aims to evaluate the impact of ATH434 treatment on neuroimaging and protein biomarkers, demonstrating target engagement and clinical efficacy. The study also assesses safety and pharmacokinetics. Selected biomarkers, such as brain iron and aggregating α-synuclein, are key contributors to MSA pathology, making them appropriate targets to demonstrate drug activity. Wearable sensors were utilized to evaluate motor activities crucial to MSA patients. The trial enrolled 77 adults who received either one of two dose levels of ATH434 or a placebo. Participants were treated for 12 months, providing an opportunity to detect changes in efficacy endpoints to optimize the design of a Phase 3 study.

ATH434, Alterity’s lead candidate, is an oral agent designed to prevent the aggregation of pathological proteins linked to neurodegeneration. Preclinical studies have shown that ATH434 can reduce α-synuclein pathology and preserve neuronal function by restoring normal brain iron balance. As an iron chaperone, it holds promise for treating Parkinson’s disease and Parkinsonian disorders, such as MSA. ATH434 successfully completed Phase 1 studies, demonstrating good tolerance and achieving brain levels comparable to effective levels in animal models of MSA. Currently, ATH434 is being tested in two clinical trials: the ATH434-201 Phase 2 trial in early-stage MSA patients and the ATH434-202 Phase 2 Biomarker trial in advanced MSA patients. The U.S. FDA and the European Commission have granted ATH434 Orphan drug designation for MSA treatment.

Multiple System Atrophy (MSA) is a rare neurodegenerative disease marked by autonomic nervous system failure and impaired movement. The symptoms reflect the progressive loss of function and death of various nerve cells in the brain and spinal cord. MSA progresses rapidly and leads to significant disability. It is characterized by a mix of slowed movement, rigidity, autonomic instability affecting involuntary functions like blood pressure and bladder control, and impaired balance and coordination, leading to falls. A pathological hallmark of MSA is the accumulation of the protein α-synuclein within glia, the central nervous system's support cells, and neuron loss in several brain regions. Approximately 15,000 individuals in the U.S. are affected by MSA. While some symptoms can be managed with medications, there are currently no drugs that can slow disease progression or provide a cure.

Alterity Therapeutics, based in Melbourne and San Francisco, is a clinical-stage biotechnology company focused on developing treatments for neurodegenerative diseases. Their lead asset, ATH434, is being evaluated for treating Parkinsonian disorders and is undergoing two Phase 2 clinical trials for Multiple System Atrophy. Additionally, Alterity has a broad drug discovery platform aimed at generating patentable compounds to address the underlying pathology of neurological diseases.