Athira Pharma Presents Preclinical Data Supporting Therapeutic Potential of ATH-1105 in ALS


Athira Pharma, Inc. recently presented data highlighting the potential therapeutic benefits of ATH-1105, a small molecule positive modulator of the HGF/MET neurotrophic system, in a preclinical mouse model of amyotrophic lateral sclerosis (ALS). The findings were presented at the Motor Neurone Disease Association’s 33rd International Symposium on ALS/MND.

“These exciting data support the potential for ATH-1105 as a therapeutic candidate in ALS, demonstrating improvements in measures of motor and nerve function, biomarkers of neurodegeneration and inflammation as well as nerve morphology,” said Kevin Church, PhD, Executive Vice President, Research of Athira. “We continue to report evidence supporting the potential benefits of enhancing the HGF/MET neurotrophic system with our small molecules to improve neuronal function and provide neuroprotection, and these new data build on that body of evidence.”

Data presented highlight the neuroprotective effects of daily ATH-1105 treatment in the TDP-43 mouse model of ALS. The study results show that compared to untreated TDP-43 controls:

  • ATH-1105-treated mice were protected against loss of body weight and had significant improvement in motor function and coordination, as assessed by balance beam cross times, rotarod latency to fall, grip strength and latency to fall in the Kondziela screen test.
  • ATH-1105-treated mice had significant improvements in nerve function, as measured by compound muscle action potential (CMAP) and nerve conduction velocity (NCV) throughout the course of the study.
  • Biomarker assessments of ATH-1105-treated mice had significantly reduced plasma levels of inflammatory cytokines (TNF-alpha and IL-6) and neurofilament light (NfL), suggesting a reduction in systemic inflammation and neurodegeneration, respectively.
  • Assessment of sciatic nerve histology showed that ATH-1105-treated mice had a significant increase in the number of axons, larger axonal diameters, and normal myelin thickness levels, suggestive of neuroprotection.

“Importantly, these positive findings in this ALS model are consistent with data we have reported in preclinical models of other neurodegenerative diseases, which also showed that enhancing the HGF/MET neurotrophic system has a positive impact on neurodegeneration and neuroinflammation, and could be disease modifying,” said Mark Litton, PhD, President and Chief Executive Officer of Athira Pharma. “We look forward to advancing ATH-1105 as a potential new therapy for debilitating neurodegenerative disorders such as ALS.”

Athira Pharma, Inc., headquartered in the Seattle, WA, area, is a late clinical-stage biopharmaceutical company focused on developing small molecules to restore neuronal health and slow neurodegeneration. Athira aims to provide rapid cognitive improvement and alter the course of neurological diseases with its novel mechanism of action. Athira is currently advancing its pipeline of therapeutic candidates targeting the HGF/MET neurotrophic system for Alzheimer’s and Parkinson’s disease dementia, Dementia with Lewy bodies and neuropsychiatric indications. For more information, visit www.athira.com.