Phase 2 Trial Continues as Planned Following DMC Review

Alterity Therapeutics, a biotech firm focused on developing treatments for neurodegenerative disorders, has announced that the ATH434-201 Phase 2 clinical trial is proceeding as planned following a second review by an independent Data Monitoring Committee (DMC). The trial is assessing ATH434, a potential treatment for early-stage multiple system atrophy (MSA), a rare and currently untreatable neurodegenerative condition.

The DMC's review of clinical data from participants revealed no safety concerns, leading to a recommendation to continue the study without any changes. The DMC's role in reviewing initial safety data has been acknowledged by the U.S. Food and Drug Administration. The CEO of Alterity, David Stamler, M.D., expressed satisfaction with the DMC's findings and emphasized the significance of the study's continuation, as it indicates the safe tolerance of ATH434 by trial participants over an extended period.

The ATH434-201 trial is designed to evaluate the impact of ATH434 on neuroimaging and protein biomarkers, aiming to demonstrate the drug's effectiveness and engagement with its target. The biomarkers under investigation, such as brain iron levels and α-synuclein aggregation, are key to MSA's pathology, making them suitable indicators of the drug's potential impact. The study also employs wearable sensors to monitor motor activities critical for MSA patients. With 77 enrolled adults receiving either one of two ATH434 dosages or a placebo, the 12-month treatment period is intended to detect efficacy changes that could inform the design of a subsequent Phase 3 trial.

ATH434 is an oral agent developed to inhibit the aggregation of proteins associated with neurodegeneration. It has shown promise in preclinical studies by reducing α-synuclein pathology and preserving neuronal function through the restoration of normal brain iron balance. As an iron chaperone, ATH434 also holds potential for treating Parkinson’s disease and other Parkinsonian disorders, including MSA. The drug has successfully completed Phase 1 studies, demonstrating good tolerance and achieving brain levels comparable to those effective in 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 trial examining patients with more advanced MSA.

MSA is a rare disorder characterized by the failure of the autonomic nervous system and impaired movement. It is rapidly progressive and leads to significant disability. The disease is marked by a combination of movement issues and autonomic instability, affecting involuntary functions such as blood pressure and bladder control. The pathological hallmark of MSA is the accumulation of α-synuclein in the brain's support cells and neuron loss in various 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 slow the disease's progression or offer a cure.

Alterity Therapeutics is a clinical-stage company based in Melbourne, Australia, and San Francisco, California, committed to creating new possibilities for those living with neurodegenerative diseases. The company's lead asset, ATH434, has the potential to address a range of Parkinsonian disorders, and Alterity also has a robust drug discovery platform that generates patentable chemical compounds to intervene in disease processes.