Athira Pharma, Inc. a late clinical-stage biopharmaceutical company focused on developing small molecules to restore neuronal health and slow neurodegeneration, today announced that following an unblinded interim efficacy and futility analysis, an independent data monitoring committee recommended continuation of the LIFT-AD study of fosgonimeton in patients with mild-to-moderate Alzheimer’s disease. The committee also determined that, with the additional enrollment of fewer than 150 patients for a total enrollment of less than 300 patients without background therapy the study will be well powered for the primary endpoint given the preliminary effect size observed. The primary endpoint of LIFT-AD is the Global Statistical Test, an unweighted composite score comprising measures of cognition and function.
Results from the completed exploratory ACT-AD Phase 2 study showed a favorable safety profile and suggested positive effects on measures of cognition function and neurodegeneration in patients taking fosgonimeton without background therapy. Guided by these results, the Company proactively amended LIFT-AD to focus on patients not on background therapy. The unblinded interim analysis was then conducted in approximately 100 patients not on background therapy to corroborate observations from ACT-AD and ensure LIFT-AD is well powered to determine the effect of fosgonimeton on clinically meaningful and commercially relevant endpoints.
“The results from the data monitoring committee’s unblinded analysis give us confidence in a potentially positive outcome for LIFT-AD, as stringent evaluation criteria were applied based on validated and clinically meaningful cognitive and functional outcomes. This analysis supports the potential clinical benefits of fosgonimeton treatment and underscores the rationale for continued development of this promising new therapy.”
Hans Moebius, M.D., Ph.D., Chief Medical Officer of Athira
“We are very excited by the results of this independent review as we believe they mitigate the risk of the fosgonimeton development plan, support the potential clinical benefit of fosgonimeton and inform the sample size needed to achieve success with LIFT-AD,” said Mark Litton, Ph.D., President and Chief Executive Officer of Athira. “We are now targeting to complete enrollment in mid-2023 and report topline data in early 2024. Importantly, we have a strong balance sheet to execute our plans through key data readouts and beyond. Moving forward, we remain keenly focused on advancing this novel investigational therapy with the hope of positively impacting the lives of millions of Alzheimer’s patients.
“Our goal with fosgonimeton is to demonstrate its ability to improve cognition and function and to ultimately provide neuroprotection. The ACT-AD study suggested these benefits, and the results of the LIFT-AD interim analysis corroborate those findings,” added Dr. Litton. “We believe any drug that can demonstrate neuroprotection could become a treatment of choice for mild-to-moderate Alzheimer’s patients.”
About the LIFT-AD Clinical Study
LIFT-AD is a randomized, double-blind, placebo-controlled, parallel-group study of fosgonimeton for patients with mild-to-moderate Alzheimer’s disease. Patients are randomized across two dose groups and one placebo group on a 1:1:1 basis to receive a subcutaneous injection of fosgonimeton or placebo once daily over a treatment course of 26 weeks. The primary endpoint for LIFT-AD is the Global Statistical Test, an unweighted composite score comprising measures of cognition (Alzheimer's Disease Assessment Scale-Cognitive Subscale and function. Additional information on the LIFT-AD study can be found at: NCT04488419.
About Fosgonimeton
Fosgonimeton is a small molecule designed to enhance the activity of hepatocyte growth factor (HGF) and its receptor, MET, to impact neurodegeneration and regenerate brain tissue. The function of the HGF/MET receptor system may be impaired in the brain under conditions of neurodegeneration. In addition to Alzheimer’s disease, fosgonimeton has the potential to address the broader dementia population, including Parkinson’s disease dementia and Dementia with Lewy bodies, as the mode of action focuses on network recovery and synaptic signal transmission in the brain.
The ACT-AD trial was supported by a grant from the National Institute on Aging of the National Institutes of Health under Award Number R01AG06268. The information presented in this press release is solely the responsibility of Athira and does not necessarily represent the official views of the National Institutes of Health.
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 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.