Athira abandons Alzheimer's drug, shifts focus to ALS

Athira Pharma has halted the development of fosgonimeton, a daily injectable drug for Alzheimer's disease, following underwhelming results in a mid-stage clinical trial. The Seattle-based biopharmaceutical company announced in its recent earnings release that it has decided to "pause further development" of fosgonimeton. This drug, designed to bind to hepatocyte growth factor (HGF) receptors to promote neuronal growth and survival, did not meet its primary or secondary endpoints in a Phase 2/3 trial targeting patients with mild-to-moderate dementia. Despite these setbacks, Athira had initially indicated its intention to continue investigating the HGF target due to promising "biomarker and subgroup data" which pointed to a "broad neuroprotective mechanism of action."

In light of these developments, Athira has shifted its focus to ATH-1105, an oral drug also targeting HGF receptors, but aimed at treating amyotrophic lateral sclerosis (ALS). This strategic pivot was accompanied by a significant reduction in workforce, with the company cutting 70% of its staff to manage costs effectively. Currently, ATH-1105 is undergoing a Phase 1 dose-escalation study in healthy volunteers, expected to conclude by the end of the year. Preclinical research has shown that ATH-1105 enhances motor and nerve function, supports neuronal survival, and lowers levels of plasma neurofilament light chain, a biomarker indicative of ALS progression. Athira plans to begin dosing ALS patients with ATH-1105 in 2025.

Athira's current financial position includes $68.9 million in cash, equivalents, and investments as of the end of September. The company believes these resources will sustain its operations until the first quarter of 2026. Meanwhile, Athira is exploring various "strategic alternatives," which may involve new partnerships and additional funding options to support its ongoing projects.

The past few years have been turbulent for Athira. In 2021, former CEO Leen Kawas was implicated in a controversy involving altered images in her doctoral thesis, leading to a significant conflict within the company's leadership and a major investor. This led to a change in the company's leadership structure. In 2022, fosgonimeton encountered another setback when it failed in a separate mid-stage trial for Alzheimer's disease, which focused on a different primary endpoint related to memory processing speed.

Despite these challenges, Athira remains committed to advancing its pipeline, particularly focusing on the potential of HGF receptor-targeting drugs like ATH-1105 in treating neurodegenerative diseases such as ALS. The company's strategic shift and focus on innovative oral therapies could pave the way for future breakthroughs in this field.