BOTHELL, Wash— Athira Pharma, Inc. (NASDAQ: ATHA), a late clinical-stage biopharmaceutical company focused on developing small molecules to restore neuronal health and slow neurodegeneration, today announced publication of research highlighting the neuroprotective and anti-inflammatory effects of ATH-1105 in preclinical models of amyotrophic lateral sclerosis (ALS). The original research article, “ATH-1105, a small-molecule positive modulator of the neurotrophic HGF system, is neuroprotective, preserves neuromotor function, and extends survival in preclinical models of ALS,” authored by Berthiaume, A., and Reda, S., et al., was published in the peer-reviewed journal, Frontiers in Neuroscience. ATH-1105 is a next-generation, orally administered small molecule drug candidate designed to enhance the neurotrophic hepatocyte growth factor (HGF) system.
“These data demonstrate that ATH-1105 treatment results in significant, consistent beneficial effects both in cell culture and in vivo models of ALS. Through enhancement of the neurotrophic HGF system, ATH-1105 protects spinal motor neurons from ALS-relevant insults in vitro and in animal models of ALS, prevents the progressive decline of motor and nerve function, reduces inflammation, preserves body weight and extends survival. Also, the significant reduction in plasma neurofilament light chain (NfL) levels, an established biomarker of neurodegeneration in ALS, is highly encouraging,” said Kevin Church, Ph.D., Chief Scientific Officer of Athira. “These studies further support the therapeutic potential and continued development of ATH-1105, which is targeted to advance into first-in-human studies this year.”
Key findings highlighted in the publication include:
- ATH-1105 enhances neurotrophic HGF system signaling and protects primary neuron cultures from various insults relevant to ALS.
- ATH-1105 treatment reduced astrocyte reactivity in spinal motor neuron-astrocyte co-cultures and preserved neuromuscular junction integrity in spinal motor neuron-muscle co-cultures following exposure to toxic levels of glutamate, believed to be a key driver of ALS pathology.
- In a TDP-43 mouse model of ALS, ATH-1105 treatment significantly preserved body weight, reduced motor and nerve function decline over time, decreased plasma biomarkers of inflammation and neurodegeneration, prevented axonal degeneration and TDP-43 pathology in peripheral nerves, and ultimately extended survival.
“There is an urgent need for new ALS treatment options, particularly those aimed at slowing or stopping neurodegeneration,” said Mark Litton, Ph.D., President and Chief Executive Officer of Athira. “The results reported in this peer-reviewed publication suggest that ATH-1105 demonstrated consistent translation of neuroprotective and anti-inflammatory effects from in vitro to in vivo models, which led to improved motor function and survival in an ALS animal model. These findings further support our plans to progress ATH-1105 into first-in-human studies in the first half of 2024.”
About ATH-1105
ATH-1105 is an orally administered small molecule designed to positively modulate the neurotrophic hepatocyte growth factor (HGF) system, which plays a critical role in nervous system maintenance and repair, including stimulation of cell survival, increase in neuronal outgrowth and modulation of neuronal network repair. In preclinical models of amyotrophic lateral sclerosis (ALS), ATH-1105 has been shown to significantly increase survival, enhance motor and nerve function, reduce peripheral nerve demyelination and axon degeneration, and improve neurodegeneration and inflammation.
About Athira Pharma, Inc.
Athira Pharma, Inc., headquartered in the Seattle, Washington area, is a late clinical-stage biopharmaceutical company focused on developing small molecules to restore neuronal health and slow neurodegeneration. Athira aims to alter the course of neurological diseases by advancing its pipeline of therapeutic candidates that modulate the neurotrophic HGF system, including fosgonimeton (ATH-1017), which is being evaluated for the potential treatment of mild-to-moderate Alzheimer’s disease in the Phase 2/3 LIFT-AD trial that is targeted to report topline data in the second half of 2024.
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