Athira’s Alzheimer’s Drug Fails in Later-stage Clinical Trial

By Simon Spichak, MSc | September 17th, 2024

Drugmaker Athira was trying a fresh approach to Alzheimer's treatment. But their experimental drug, designed to protect brain cells for people with moderate Alzheimer's, has failed out of trials.

Athira Pharma’s experimental Alzheimer’s drug fosgonimeton hit a speedbump on its way toward FDA approval: The treatment failed to show a clinical benefit in its latest clinical trial. 

The small molecule drug, which is injected under the skin, turns on a signaling pathway called HGF/MET that has been shown to help support healthy brain function and protect brain cells from inflammation and neurodegenerative disease in mouse models and cells in a Petri dish. 

Alzheimer’s Drug Types 101: What Are Small Molecule Drugs?

The company’s CEO stepped down in 2021 due to a data manipulation scandal related to her research identifying this pathway as a potential target for treating the disease. 

Athira recruited 312 participants with mild to moderate Alzheimer’s disease for the Phase 2/3 trial, which lasted 26 weeks. Participants were split randomly into two groups: One half received the drug, while the other half received a placebo. Ultimately, however, the results show that the participants who took the drug did not show slower cognitive decline than the placebo group. 

“These are not the results we hoped for,” Dr. Javier San Martin, chief medical officer of Athira, said in a press release. The participants in the placebo group didn’t experience cognitive decline over the 26 weeks, as would be expected in Alzheimer’s disease. The company thinks this might have made it harder to show that the drug works to slow cognitive decline. They also believe that the study might be too short to see an effective change, and the trial may have been too short to see an effective change.

The company did not announce whether they will run another drug trial, but there are many other drugs making their way through clinical trials to treat Alzheimer’s.

Updates on Alzheimer’s Drug Research from AAIC 2024

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3 thoughts on “Athira’s Alzheimer’s Drug Fails in Later-stage Clinical Trial

  1. I think the article title, while true in a scientifically semantically accurate sense, is still misleading. This trial didn’t end up saying anything about whether Fosgonimeton would be effective in protecting brain cells or not. Since there was no decline in the placebo group, this trail didn’t say anything at all about the drug other than that 26 weeks is not long enough to test any Alzheimer’s drug (or, alternatively, that their inclusion criteria was bad). What I don’t understand was why they wouldn’t either extend the trial length (perhaps it had ended too long before analysis was complete to continue using the same cohorts…or perhaps, again, the selection criteria was bad) or run a new trial. If they believe in the drug, they should absolutely run a new longer trial.

    1. I don’t agree with the content of this article. The study was much longer in its duration than 26 weeks, and as a caregiver who was providing injections on a daily basis, I am convinced there benefits.

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