Athira Pharma to Advance ATH-1105 for Neurodegenerative Disease Treatment

Athira Pharma, Inc., a clinical-stage biopharmaceutical company, has announced its strategic decision to concentrate efforts on the development of ATH-1105, an orally administered small molecule designed as a positive modulator of the neurotrophic hepatocyte growth factor (HGF) system. This decision follows the recent Phase 2/3 LIFT-AD clinical trial outcomes for fosgonimeton in treating Alzheimer's disease (AD). ATH-1105 is in a Phase 1 clinical trial aimed at treating neurodegenerative diseases, notably amyotrophic lateral sclerosis (ALS) and AD.

In alignment with this renewed focus, Athira Pharma is implementing significant cost containment measures. These measures include a workforce reduction of about 70%, which is expected to incur one-time costs of $2.8 million but yield annual cost savings of roughly $13.4 million. With these adjustments, the company anticipates extending its financial runway into the first quarter of 2026. Additionally, Athira plans to explore various partnering and financing options to further prolong its cash reserves, thereby supporting the continued development of ATH-1105.

Mark Litton, Ph.D., President and CEO, expressed optimism about ATH-1105's potential. He highlighted the drug candidate's enhanced ability to penetrate the blood-brain barrier and improved pharmacokinetic properties. Litton pointed out that preclinical data has shown ATH-1105 to have neuroprotective effects, evidenced by a consistent reduction in plasma neurofilament light chain (NfL) levels, a key biomarker for neurodegeneration. Javier San Martin, M.D., Chief Medical Officer, added that the NfL data from the LIFT-AD study suggests that HGF modulation could mitigate neurodegeneration, making ATH-1105 a promising candidate for treating ALS and other neurodegenerative diseases.

Dr. Litton also acknowledged the contributions of employees affected by the workforce reduction, expressing gratitude for their efforts in advancing the company's therapeutic developments.

Athira is currently conducting a Phase 1 double-blind, placebo-controlled trial (NCT 06432647) involving up to 80 healthy volunteers. This trial aims to assess the safety and tolerability of ATH-1105 through single and multiple oral ascending doses. The first cohort of healthy volunteers completed the study in June 2024, with full study completion expected by the end of 2024. The company aims to begin dosing ALS patients in 2025.

Preclinical models of ALS have shown that ATH-1105 can significantly improve survival rates, enhance motor and nerve function, reduce nerve demyelination and axon degeneration, and lessen neurodegeneration and inflammation.

Based in the Seattle, Washington area, Athira Pharma is dedicated to developing small molecules that restore neuronal health and slow neurodegeneration. The company's mission is to alter the course of neurological diseases by advancing drug candidates that modulate the neurotrophic HGF system.