First group of healthy volunteers enrolled in ATH-1105 study

Phase 1 trial is recruiting up to 80 adults to test experimental therapy

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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Athira Pharma has completed enrollment of the first group of healthy volunteers who’ll receive a single dose of ATH-1105, its experimental oral therapy for amyotrophic lateral sclerosis (ALS), in a Phase 1 clinical trial.

The Phase 1 trial (NCT 06432647), now recruiting up to 80 adult healthy volunteers, will evaluate the safety, tolerability, and pharmacokinetics of ATH-1105 versus a placebo. Pharmacokinetics refers to how a medicine moves into, through, and out of the body.

The first group will receive an ascending dose of the solution or a matching placebo, given only once, while a second group will be given multiple ascending doses of ATH-1105 or a placebo. The trial should be complete by the end of the year and Athira plans to study ALS patients next year.

“We are pleased to start this Phase 1 clinical trial and we are eager to explore the potential of ATH-1105 in ALS patients, which we are targeting to initiate in 2025,” Mark Litton, PhD, Athira’s president and CEO, said in a company press release.

ATH-1105 is a small molecule candidate that’s designed to reduce inflammation and promote nerve cell growth and survival, which should slow disease progression.

“We look forward to continued development of this promising therapeutic candidate for people with ALS, who are in need of therapies that improve the course of disease,” Javier San Martin, MD, Athira chief medical officer, said.

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ATH-1105 protects against common ALS, FTD disease mechanisms

ATH-1105 in lab studies, mice

The drug works by activating a signaling pathway called the HGF/MET system, which is believed to counteract certain mechanisms that contribute to ALS.

“By modulating the neurotrophic HGF system, we believe we can prevent or slow the progressive decline of motor and nerve function, reduce inflammation, preserve body weight, and extend survival for patients suffering with this devastating neurodegenerative condition that has few treatment options,” Litton said.

In lab studies, ATH-1105 activated the HGF system and exerted neuroprotective effects in nerve cell models. Treatment with it reduced nerve cell damage induced by several toxins and stressors, along with the activity of microglia, a type of immune cell in the brain that’s overly active in ALS and drives inflammation.

In an ALS mouse model, the therapy improved motor function and prolonged the animals’ survival. It also reduced levels of toxic TDP-43, a hallmark of ALS, and of neurofilament light chain (NfL), a marker of nerve damage.

“We are particularly excited about the potential for ATH-1105 as a treatment for ALS based on our compelling preclinical data that have shown ATH-1105 can improve motor function, preserve nerve health and structure, and prolong survival in a mouse model of ALS,” Martin said.

“Our robust preclinical data to date have demonstrated ATH-1105’s neuroprotective effects including a consistent reduction in plasma neurofilament light chain levels, an established biomarker of neurodegeneration in ALS,” Litton said.