Posted: 12 February 2024
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) has completed its second review of trial data and recommended the ATH434-201 Phase 2 study continue as planned. The ATH434-201 clinical trial is a randomized, double-blind, placebo-controlled 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 its second prespecified review of unblinded clinical data from 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 has been cleared with the U.S. Food and Drug Administration.
“With accumulated data from our Phase 2 study, we are pleased to report that the Data Monitoring Committee has determined that there are no safety concerns and that the study can continue as planned,” said David Stamler, M.D., Chief Executive Officer of Alterity. “Participants in the trial are being dosed for 12 months; therefore, this recommendation is an important milestone as participants able to safely tolerate ATH434 as their time on study increases. We remain on track to complete the study in November 2024 and report top-line data in January 2025.”
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. Selected biomarkers, such as brain iron and aggregating α-synuclein, are important contributors to MSA pathology and are therefore appropriate targets to demonstrate drug activity. Wearable sensors have also been employed to evaluate motor activities that are important to patients with MSA. The study enrolled 77 adults who were randomly assigned 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.
