Our Pipeline
We have designed our proprietary drug discovery platform to develop novel therapeutic candidates that offer the potential to impact a wide range of neurological diseases and conditions. It includes small molecules designed to act on a naturally occurring neurotrophic system, to promote regeneration, provide neuroprotection, and potentially modify neurodegenerative diseases.
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ATH-1020 is a novel, orally available, next-generation small molecule drug candidate designed to positively modulate the neurotrophic HGF system. In preclinical models, ATH-1020 was shown to reduce measures of pain in models of diabetic neuropathy.
Athira completed a Phase 1 clinical trial [NCT05169671] of ATH-1020 in healthy volunteers. ATH-1020 showed a favorable safety profile and was well-tolerated. Athira plans to evaluate options with this compound and will consider its advancement in relation to other opportunities and resources.
Fosgonimeton is a small molecule drug candidate designed to enhance the activity of the neurotrophic hepatocyte growth factor (HGF) system. By targeting the protection and repair of neuronal networks, fosgonimeton offers disease-modifying potential for a broad range of neurodegenerative diseases, including Alzheimer’s disease (AD) and Parkinson’s disease (PD).
Fosgonimeton is currently delivered via a simple at-home injection just under the skin.
For AD, fosgonimeton was evaluated in the completed exploratory Phase 2 ACT-AD clinical trial [NCT04491006] in people with mild-to-moderate Alzheimer’s disease and in the late-stage randomized, double-blind, placebo-controlled, parallel-group clinical trial, LIFT-AD [NCT04488419]. Access the topline data summary of this trial here.
For PD, Fosgonimeton was previously evaluated in an exploratory Phase 2 SHAPE clinical trial [NCT04831281] in people with Parkinson’s disease dementia or dementia with Lewy bodies. Access the topline data summary of this trial here.
ATH-1105 is a novel, orally available, next-generation, small molecule drug candidate designed to positively modulate the neurotrophic HGF system.
In preclinical models of amyotrophic lateral sclerosis (ALS), ATH-1105 was shown to improve motor and nerve function as well as biomarkers of neurodegeneration and inflammation.
Athira is currently conducting a first-in-human, dose escalation Phase 1 clinical trial evaluating safety, tolerability and pharmacokinetics of ATH-1105 in up to 80 healthy volunteers. Athira expects to commence dosing of ALS patients in 2025.
Several new compounds are currently in preclinical discovery and development for neurodegenerative diseases and other indications for which Athira believes positive modulation of the neurotrophic HGF system may have therapeutic potential.