Alterity Therapeutics Phase 2 Trial to Continue After Third Review

Alterity Therapeutics, a biotechnology company focused on developing treatments for neurodegenerative diseases, announced a significant update regarding its ATH434-201 clinical trial. The company's independent Data Monitoring Committee (DMC) has conducted its third review of the trial data. Following a detailed examination of unblinded clinical data from participants, the DMC has recommended that the Phase 2 study continue as planned, emphasizing that there are no safety concerns.

The ATH434-201 trial is a randomized, double-blind, placebo-controlled study targeting patients with early-stage multiple system atrophy (MSA), a rare neurodegenerative disease with no approved treatments to halt its advance. The trial aims to assess the effects of ATH434 on neuroimaging and protein biomarkers, specifically focusing on target engagement and clinical efficacy markers. Additionally, safety and pharmacokinetic assessments are being conducted. The study includes a cohort of 77 adults who have been randomly assigned to receive one of two doses of ATH434 or a placebo. These participants will receive treatment for 12 months, which will provide an opportunity to observe changes in efficacy endpoints and inform the design of a definitive Phase 3 study.

David Stamler, M.D., CEO of Alterity, expressed satisfaction with the DMC's recommendation, highlighting the continued favorable safety profile of ATH434. Stamler noted that the trial is on track to conclude in November 2024, with top-line data expected in January 2025. The DMC's review process and the continuation of the trial have been approved by the U.S. Food and Drug Administration.

ATH434 is an oral agent developed to inhibit the aggregation of pathological proteins linked to neurodegeneration. Preclinical studies have demonstrated its ability to reduce α-synuclein pathology and preserve neuronal function by restoring normal iron balance in the brain. This mechanism of action positions ATH434 as a potential treatment for Parkinson’s disease and other Parkinsonian disorders, including MSA. In Phase 1 studies, ATH434 was well tolerated and achieved brain levels comparable to those effective in animal models. Currently, there are two ongoing clinical trials for ATH434: the ATH434-201 Phase 2 trial for early-stage MSA patients and the ATH434-202 open-label Phase 2 Biomarker trial for patients with more advanced MSA. The U.S. FDA and the European Commission have granted ATH434 Orphan drug designation for the treatment of MSA.

Multiple System Atrophy is a progressive neurodegenerative disease marked by the degeneration of different types of nerve cells in the brain and spinal cord. Symptoms include impaired movement, autonomic instability, and balance issues, leading to severe disability. The disease is characterized by the accumulation of α-synuclein protein within glial cells and neuron loss across various brain regions. Affecting approximately 15,000 individuals in the U.S., MSA currently has no treatments that slow its progression or offer a cure.

Alterity Therapeutics, headquartered in Melbourne, Australia, and San Francisco, California, is dedicated to developing innovative treatments for neurodegenerative diseases. The company's lead asset, ATH434, shows promise in treating various Parkinsonian disorders. Alterity also has a comprehensive drug discovery platform aimed at generating new chemical compounds to intervene in disease processes.