Posted: 27 July 2023
Alterity Therapeutics (ASX: ATH, NASDAQ: ATHE) (“Alterity” or “the Company”), a biotechnology company dedicated to developing disease modifying treatments for neurodegenerative diseases, today announced that an independent Data Monitoring Committee (DMC) recommended the ATH434-201 Phase 2 study continue as planned. The ATH434-201 clinical trial is a randomized, double-blind, placebocontrolled investigation of ATH434 in patients with early-stage multiple system atrophy (MSA), a rare neurodegenerative disease with no approved treatments to slow or stop its progression.
The DMC conducted a prespecified review of unblinded clinical data from an initial cohort of study participants. The DMC expressed no concerns about safety and recommended that the study continue without modification. The plan for the DMC to review initial safety data was cleared with the U.S. Food and Drug Administration. “The recommendation to continue the Phase 2 trial as planned marks another significant advance in the development of ATH434 for this devastating condition,” said David Stamler, M.D., Chief Executive Officer of Alterity. “Enrollment is continuing globally, and this milestone brings us one step closer to creating a novel treatment for individuals with MSA. We remain on track to complete enrollment in the third quarter of this year and look forward to top-line data by the end of next year.”
The ATH434-201 Phase 2 clinical trial is evaluating the effect of ATH434 treatment on neuroimaging and protein biomarkers to demonstrate target engagement and clinical endpoints to demonstrate efficacy, in addition to assessments of safety and pharmacokinetics. The selected biomarkers, including brain iron and aggregating α-synuclein, are important contributors to MSA pathology and are therefore appropriate targets to demonstrate drug activity. Wearable sensors will also be employed to evaluate motor activities that are important to patients with MSA. The study is expected to enroll approximately 60 adults to receive one of two dose levels of ATH434 or placebo. Participants will receive treatment for 12 months which will provide an opportunity to detect changes in efficacy endpoints to optimize design of a definitive Phase 3 study. Additional information on the Phase 2 trial can be found by ClinicalTrials.gov Identifier: NCT05109091.
