Athira Pharma, Inc. 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. 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.