Leqembi Clinical Trials

Alzheimer’s Clinical Trials News Roundup: Q4

By | February 4th, 2025

After months of delays, Alzheon gears up to present data from their pivotal Phase 3 drug trial, while Athira Pharma pays out a settlement to federal agencies related to their failed drug — and other Q4 updates from the world of Alzheimer’s research and trials. 

It’s been a quiet few quarter for Alzheimer’s drug development. The biggest news: Drugmaker Alzheon is set to share results from its Phase 3 trial of a pill for those living with two copies of the ApoE4 gene. Meanwhile, Athira Pharma’s Alzheimer’s drug was discontinued. Axsome is asking the FDA to approve its agitation drug, and the FDA did just this week (despite the Trump administration’s communications freeze) green-light a dosage change for Leqembi, making it more similar to the dosage regimen for Kisunla — at least, after the first 18 months. Last but not least, this quarter, we’ve counted up 90 different clinical trials for Alzheimer’s that are recruiting right now. 

Read on for detailed drug updates, organized by drug type. And if you’re interested in participating in one of 90 late-stage drug trials underway right now for Alzheimer’s, use this tool to learn more.

Disease-modifying drugs 

Small molecule drugs for Alzheimer’s disease

Alzheimer’s Drug Types 101: What Are Biologics?

ALZ-801/valiltromiprosate

Sponsored by: Alzheon 

Latest trial phase: Completed Phase 3 trials

Next steps: Presenting data at Alzheimer’s Disease Parkinson’s Disease Conference in April 2025. 

Alzheon’s experimental drug, ALZ-801/valiltramiprosate, is a pill that prevents good forms of beta-amyloid from turning into toxic plaques. The drug was tested in people with MCI and early Alzheimer’s who carried two copies of the ApoE4 gene. The trial was completed last year, and though the company stated they would share data and file for approval by Q3 of 2024, it was delayed without explanation. In early January 2025, the company stated that it will present data from the trial in April. The spokesperson did not mention whether there are plans to file for FDA approval. 

A Pill to Slow Down Alzheimer’s Is as Little as a Year Away

 

Blarcamesine

Sponsored by: Anavex Life Sciences

Latest trial phase: Completed Phase 2/3 trial.

Next steps: Submitted drug for approval in Europe. 

Blarcamesine is a small molecule drug that is purported to work by activating the sigma-1 protein in the brain which may have neuroprotective effects. The company completed its Phase 2/3 trial for Alzheimer’s disease in 2022. Notably, about one in three participants who received the drug dropped out of the study due to side effects. Though the company reported the drug data was successful, the efficacy of the drug has been criticized and the company is currently embroiled in shareholder lawsuits alleging it misrepresented the trial data. At the end of November 2024, the company filed an application for drug approval with European regulators who will make their decision later this year. And this month, they presented positive post-trial data on 300 participants who, after their trial concluded, continued taking the drug for three more years.  

Fosgonimenton

Sponsored by: Athira Pharma

Latest trial phase: Discontinued

Next steps: The company will focus on other drugs targeting the same pathway.

Fosgonimenton is a small molecule drug administered via injection under the skin, and is designed to switch on a cellular 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. The company’s CEO stepped down in 2021 due to a data manipulation scandal related to her research on identifying this pathway as a potential target for treating the disease. On January 6th, 2025, a few months after Athira Pharma failed to show a clinical benefit in its latest clinical trial of fosgonimeton, the U.S. Department of Justice announced that the company agreed to pay more than $4 million to settle allegations that it knowingly used studies containing falsified data to secure grant funding from the National Institutes of Health. Athira Pharma has since discontinued development of the drug.

Alzheimer’s Drugmaker Athira Shells Out $4 Million In DOJ Settlement

Disease-modifying biologics

Antibodies, vaccines, cell infusions and more

Leqembi 

Industry sponsored?: Eisai and Biogen

Latest trial phase: Approved by the FDA

Next steps: The company has filed an application to approve an injectable version of the drug.

Eisai and Biogen’s anti-amyloid drug Leqembi may become easier to access soon. The FDA has accepted the company’s application for an injectable version of the drug, which could be delivered at home through an injector pen like insulin or Ozempic. The FDA will decide whether or not to approve the new formulation by the end of August 2025.

One Year In, 5 Things We’ve Learned From Patients on Leqembi

 

Symptomatic Treatments

Small-molecule drugs for psychiatric symptoms

AXS-05

Industry sponsored?: Axsome Therapeutics

Latest trial phase: Completed two Phase 3 trials

Next steps: Filing for FDA approval later this year

AXS-05 is a small molecule drug approved in 2022 by the FDA for the treatment of  depression under the brand name Auvelity. Axsome shared data from one Phase ⅔ trial in 2020 and another in 2022. Now, after yet two more Phase 3 trials, the company thinks the drug is ready for Alzheimer’s patients as well. It targets many different proteins in the brain, and the drugmakers theorize that its effects on brain signalling could alleviate some of the agitation that develops with dementia. The company will file for FDA approval this year on the basis of two Phase 3 trials, though outside experts who weren’t involved in the trial say the data shows the drug doesn’t actually work.

Axsome Asks FDA to Approve Questionable Alzheimer’s Agitation Drug

IGC AD-1

Industry sponsored?: IGC Pharma

Latest trial phase: Ongoing Phase 2 trial

Next steps: Completion of Phase 2 trialIGC-AD1 is an oral agitation drug developed to target the brain’s endocannabinoid receptors, mimicking the biological effects of cannabis to reduce inflammation and alter brain chemistry. The drug is currently being tested in a 164-person Phase 2 trial. In late November 2024, the company announced that, so far, the participants receiving the drug show a small improvement in cognitive function over six weeks, compared to those receiving a placebo. It isn’t clear whether these effects will be sustained through the end of the study. The trial is set to finish at the end of June 2025.    

If you find our articles and interviews helpful, please consider becoming a supporting member of our community. Frustrated by the lack of an editorially independent source of information on brain health and Alzheimer’s disease, we decided to create Being Patient. We are a team of dedicated journalists covering the latest research on Alzheimer’s, bringing you access to the experts and elevating the patient perspective on what it’s like to live with dementia.

Please help support our mission.

One thought on “Alzheimer’s Clinical Trials News Roundup: Q4

Leave a Reply

We are glad you have chosen to leave a comment. Please keep in mind that comments are moderated according to our comment policy.