On 3 September, Athira Pharma, a late clinical-stage biopharmaceutical company focused on developing small molecules to restore neuronal health and slow neurodegeneration, announced top-line results from its Phase 2/3 LIFT-AD clinical trial of fosgonimeton in people with mild-to-moderate Alzheimer’s disease (AD). The LIFT-AD trial was a randomised, placebo-controlled and double-blind study that evaluated the efficacy and safety of fosgonimeton. 312 people with mild-to-moderate AD received once-daily subcutaneous injections of either fosgonimeton or placebo during 26 weeks. The company reported that the study failed to meet its primary endpoint, which was the change from baseline after 26 weeks of treatment using the Global Statistical Test (GST), a combination of results from measures of cognition (ADAS-Cog11) and function (ADCS-ADL23).
The topline results also showed that the trial did not meet its key secondary endpoints of cognition (ADAS-Cog11) and function (ADCS-ADL23). In a prespecified subgroup analyses of participants with moderate AD or who were APOE4 carriers, fosgonimeton showed a numerically greater treatment effect in clinical outcomes compared to placebo. In addition, data across biomarkers of protein pathology (Aβ42/40, p-Tau181, and p-Tau217), inflammation (GFAP) and neurodegeneration (NfL) showed directional improvements with fosgonimeton treatment versus placebo after 26 weeks. The drug was generally well tolerated, with a favourable safety profile. Full analysis of the results is planned to be presented at the 17th Annual Clinical Trials on Alzheimer’s Disease (CTAD) taking place on October 29 - November 1, 2024, in Madrid, Spain.
