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.
Disclaimer: Product candidates described in the Pipeline are under development, subject to ongoing testing and clinical evaluation, and have not been approved by any regulatory authority for any commercial use in humans.
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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 previously evaluated in the completed exploratory Phase 2 ACT-AD clinical trial [NCT04491006] in people with mild-to-moderate Alzheimer’s disease. Topline results for this trial were announced in June 2022, and did not meet the primary endpoint, change in Event-Related-Potential (ERP) P300 latency, or secondary endpoints based on the protocoled analysis. However, a post-hoc analysis of results in a pre-specified subgroup suggested positive effects on measures of cognition, function and neurodegeneration in participants taking fosgonimeton alone without concomitant acetylcholinesterase inhibitors. Access the topline data summary of this trial here.
Fosgonimeton is currently being evaluated in a late-stage randomized, double-blind, placebo-controlled, parallel-group clinical trial, LIFT-AD [NCT04488419]. The trial is designed to evaluate the safety and efficacy of fosgonimeton in individuals with mild-to-moderate AD not on concomitant acetylcholinesterase inhibitors. In January 2024, Athira completed enrollment of the LIFT-AD clinical trial.. The company expects to report topline data in the second half of 2024.
To date, over 85% of participants completing the ACT-AD and LIFT-AD clinical trials have elected to enroll in the ongoing open label extension trial [NCT04886063]. Fosgonimeton has shown a favorable safety profile and to be consistently well tolerated.
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. Topline results for this trial were announced in November 2023, and did not meet the primary endpoint, a composite score of the change in ERP P300 latency and cognitive assessment (ADAS-Cog13). Once daily treatment with fosgonimeton 40 mg (n=5), however, showed positive effects in cognitive measures compared to placebo (n=7) over the 6-month double-blind treatment period. Access the topline data summary of this trial here.
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.
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 expects to initiate a first-in-human study of ATH-1105 in the second quarter of 2024.
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.