Athira to Begin Open-label Extension Studies of ATH-1017
Both clinical trials — the Phase 2/3 LIFT-AD (NCT04488419) and Phase 2 ACT-AD (NCT04491006) — are investigating ATH-1017, a small molecule designed to enhance the effect of hepatocyte growth factor (HGF), and its receptor, MET. Both are expressed in the central nervous system.
HGF works on specific cell types via its receptor to promote cell growth, cell migration, and new blood vessel formation. The hope is that by enhancing the activity of this receptor pair, brain health and function will be improved.
In previous preclinical studies, ATH-1017 was well-tolerated in all tested doses and was able to regenerate nerve cells and improve cognitive function.
These two studies are evaluating the safety and efficacy of ATH-1017 in patients with mild-to-moderate Alzheimer’s disease.
Individuals were assigned randomly into three groups to receive either low-dose ATH-1017 (40mg/day), high-dose ATH-1017 (70mg/day), or placebo.
Improvement in cognition, global and functional assessments will be evaluated in both studies comparing the high- and low-dose treatments to the placebo, with data expected by 2022.
The goal of the new open-label extension study (NCT04886063) is to determine the safety and tolerability of ATH-1017 in patients who completed 26 weeks of treatment in both of the studies. All patients, even those who had previously received a placebo, now will be given ATH-1017. Researchers are hopeful this trial can provide additional, longer-term safety and tolerability information regarding ATH-1017 administration up to one year in this patient population.
“This treatment extension allows us to meet investigator and patient interest in continuing treatment with ATH-1017. We can now collect up to 1 year of safety and efficacy data on ATH-1017, and patients who received placebo during the randomized portion of the trial will now be able to receive up to 26 weeks of therapy,” Hans Moebius, MD, PhD, chief medical officer at Athira, said in a press release.
“It’s rewarding to have our first patients completing six months of study now continue their treatment. Our novel treatment approach is agnostic to the underlying disease pathology and is designed to focus on neuronal regeneration, which has the potential to improve clinical outcomes for patients,” Moebius said.
Michael Mega, MD, PhD, director of the Center for Cognitive Health in Portland, Oregon, and a principal investigator in the ACT-AD trial added: “There remains an urgent need for therapies that improve cognition for patients who are living with mild-to-moderate Alzheimer’s disease. … The data we will continue to collect in this open label extension could help us to better understand the long-term safety and efficacy profile of ATH-1017 and could help Athira best design future clinical trials of ATH-1017.”