Benzinga | Oct 19, 2021 04:51AM EDT

Alterity Therapeutics Announces Expanded ATH434 Phase 2 Clinical Development Program; Clinical Trial to Enroll Patients with Early-stage Multiple Systems Atrophy

Alterity Therapeutics (NASDAQ:ATHE) ("Alterity" or "the Company") today announced an expansion of the clinical development program for the Company's lead asset, ATH434, in patients with Multiple System Atrophy (MSA), a rare and rapidly progressing Parkinsonian disorder. ATH434 has been shown to reduce abnormal accumulation of -synuclein by restoring normal iron balance in the brain with the objective of improving motor function in patients with MSA and Parkinson's Disease.

The Phase 2 clinical trial is a randomized, double-blind, placebo-controlled investigation of ATH434 in patients with early-stage MSA. The study will explore the effect of ATH434 treatment on imaging and protein biomarkers such as aggregating -synuclein and excess iron, which are important contributors to MSA pathology. Several other biomarkers and clinical endpoints will permit comprehensive assessment of ATH434 efficacy along with characterization of safety and pharmacokinetics. Based on consultation with the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA) and clinical experts in MSA, Alterity has established that patients will receive treatment for 12 months in the Phase 2 study. The longer treatment duration will provide an improved opportunity to detect changes in biomarkers and clinical endpoints to optimize design of a definitive Phase 3 study.

In addition, the Biomarkers of Progression in Multiple Systems Atrophy (bioMUSE) natural history study has reached its original enrollment goal and will be expanded to a total of 20 patients with MSA. The study has proved to be invaluable in generating data to inform and de-risk the Phase 2 trial design, and it will continue to provide longitudinal biomarker and clinical data to characterize disease progression in a patient population that mirrors those to be enrolled in the Phase 2 study.

"With our planned Phase 2 clinical trial and the expansion of bioMUSE, we have created a robust development program to advance ATH434 for the treatment of MSA," said David Stamler, M.D., Chief Executive Officer, Alterity. "By restoring normal iron balance in the brain, ATH434 has the potential to block -synuclein aggregation, preserve neurons, and treat the underlying pathology of MSA. If successful, this approach to modifying disease progression will have a profound impact on the quality of life for individuals living with MSA, a devastating disease with very few treatment options."

Dr. Stamler, continued, "Importantly, our Phase 2 clinical trial integrates regulatory feedback, expert advice, and critical learnings from bioMUSE to establish an optimal trial design with an For personal use only improved overall chance of success. There has been great interest in our program from prospective investigators and we look forward to initiating the trial in the first quarter of calendar year 2022."

The Phase 2, double-blind clinical trial is a three-arm study where early stage MSA patients will be randomized to one of two doses of ATH434 or a placebo, with twice daily dosing. Early-stage patients with a clinical diagnosis of MSA who are ambulatory, have no evidence of severe impairment, and do not have long standing motor symptoms will be enrolled in the study. The trial is expected to enroll 60 early-stage patients in approximately 30 sites in Australia, New Zealand, Europe, and the U.S. As reported last month, the initial results from the Company's bioMUSE natural history study were instrumental in guiding the design of the Phase 2 clinical trial by informing patient selection and confirming that iron content in the brain is a promising biomarker. Based on these data, Alterity believes that treating patients in the early stage of their disease will provide the best chance of improvement from ATH434