DMC Endorses Unaltered Continuation of Alterity's Phase 2 Trial Post Third Assessment

Alterity Therapeutics, a firm focused on neurodegenerative disease treatments, has received a positive review from a Data Monitoring Committee (DMC) for its ATH434-201 Phase 2 trial. The study is examining the effects of ATH434 on patients with early-stage multiple system atrophy (MSA), a condition lacking treatments to arrest its progression. The DMC, after a thorough review of the trial's data, found no safety concerns and advised that the trial should proceed without any changes.

The ATH434-201 trial is a randomized, double-blind, placebo-controlled study that is assessing the impact of ATH434 on neuroimaging and protein biomarkers, aiming to prove target engagement and clinical efficacy, along with safety and pharmacokinetics assessments. The biomarkers chosen for the study, such as brain iron and α-synuclein aggregation, are crucial to MSA's pathology and are thus fitting targets to validate the drug's activity. Wearable sensors are being used to monitor motor functions critical to MSA patients. The trial has enrolled 77 adults who are receiving either one of two ATH434 dosages or a placebo for a period of 12 months, which will allow for the detection of efficacy endpoint changes for a future Phase 3 study.

ATH434, Alterity's leading candidate, is an oral agent designed to prevent the aggregation of proteins associated with neurodegeneration. It has demonstrated its ability to reduce α-synuclein pathology and maintain neuronal function by restoring normal brain iron levels. As an iron chaperone, ATH434 holds promise for treating Parkinson's disease and related disorders such as MSA. It has successfully completed Phase 1 studies, showing good tolerance and achieving brain levels comparable to those in effective MSA animal models. ATH434 is currently under investigation in two clinical trials, with the ATH434-201 trial focusing on early-stage MSA patients and another open-label Phase 2 Biomarker trial for patients with more advanced MSA. The drug has received Orphan drug designation for MSA treatment from both the U.S. FDA and the European Commission.

MSA is a rare neurodegenerative condition characterized by autonomic nervous system failure and impaired movement. It is a rapidly progressive disease that leads to significant disability. MSA is marked by a combination of slowed movement or rigidity, autonomic instability affecting involuntary functions, and impaired balance or coordination. The disease is pathologically characterized by α-synuclein accumulation in glial cells and neuronal loss in various brain regions. MSA affects at least 15,000 individuals in the U.S., and while some symptoms can be managed with medication, there are currently no treatments that can slow the disease's progression or offer a cure.

Alterity Therapeutics is a clinical-stage biotechnology company with a mission to create a better future for those living with neurodegenerative diseases. The company is based in Melbourne, Australia, and San Francisco, California, USA, and is committed to developing patentable chemical compounds to intervene in disease processes.