Dosing Starts in Athira’s Phase 2 ACT-AD Trial Testing ATH-1017; Enrollment Ongoing

Athira Pharma has begun dosing patients in its Phase 2 clinical trial evaluating ATH-1017, an investigational small molecule for the treatment of mild to moderate Alzheimer’s disease.

Clinical efficacy will be demonstrated by improvements in cognition and functional assessments among participants, compared with a placebo.

“The initiation of our second late-stage study for ATH-1017 represents a significant milestone for Athira as we advance our clinical program to treat this area of immense medical need,” Hans Moebius, MD, PhD, chief medical officer at Athira, said in a press release.

Called ACT-AD, the randomized, double-blind trial (NCT04491006) will evaluate the safety and efficiency of ATH-1017 (previously known as NDX-1017), compared with a placebo, in the treatment of mild to moderate Alzheimer’s.

The trial expects to recruit a total of 75 participants, ages 55 to 85, at several clinical sites across the U.S. and Australia. More information and contacts can be found here.

Similar to the company’s LIFT-AD Phase 2/3 trial, participants will be randomly assigned to receive either a low or high dose of ATH-1017 or a placebo, delivered subcutaneously (under the skin) daily for 26 weeks. Neither participants nor researchers will know which patient is given the medication and which the placebo.

The study will measure quantitative electroencephalography (qEEG), as a biomarker for brain function, and event-related potential (ERP) to assess working memory processing speed and executive function.

“The use of qEEG and ERP 300 as physiological outcome measures and indicators of improved function in brain networks could be highly valuable to the field as a whole,” said Charles Bernick, MD, co-investigator at University of Washington Alzheimer’s Disease Research Center and director of clinical trials at UW Medicine Memory & Brain Wellness Center.

ATH-1017 is a small molecule specifically designed to enhance the activity of the hepatocyte growth factor (HGF, also known as scatter factor), and its receptor protein MET, which are expressed in the central nervous system, in order to promote brain health and function.

The therapy was developed to slow or stop disease progression or even reverse it. In previous preclinical studies, ATH-1017 was shown to be able to regenerate nerve cells and improve cognitive function.

“Athira’s novel treatment approach is agnostic to the underlying disease pathology of Alzheimer’s and other dementias. It focuses on network recovery and information transmission in the brain, which has the potential to improve clinical outcomes for patients,” Moebius said.

Results from the ACT-AD trial may provide supportive information that can help optimize the LIFT-AD as a potentially pivotal trial.

“Our goal, with both this ACT-AD study and the LIFT-AD study, is to demonstrate the clinical utility of this promising agent to treat Alzheimer’s disease and preserve cognitive health,” Moebius added.