Alzheon to Present Phase 3 Data for ALZ-801, Aiming to Slow Alzheimer's Progression
Key Insights
Alzheon is set to present Phase 3 trial data for ALZ-801 in April 2025, a drug designed to prevent the formation of toxic beta-amyloid (search) plaques in early Alzheimer's patients with the ApoE4 gene.
Anavex Life Sciences has submitted blarcamesine for approval in Europe after completing Phase 2/3 trials, though its efficacy has faced criticism and legal challenges.
Athira Pharma discontinued fosgonimeton development after failing to demonstrate clinical benefit and settled with the DOJ over data manipulation allegations.
Alzheon is preparing to unveil data from its pivotal Phase 3 trial of ALZ-801, an oral drug aimed at slowing the progression of Alzheimer's disease (search) in individuals carrying two copies of the ApoE4 gene. The presentation is scheduled for the Alzheimer's Disease Parkinson's Disease Conference in April 2025.
ALZ-801: Targeting Beta-Amyloid Plaques
ALZ-801 (valiltramiprosate) is designed to prevent the transformation of beneficial beta-amyloid (search) forms into toxic plaques, a hallmark of Alzheimer's pathology. The trial focused on patients with mild cognitive impairment (MCI) and early Alzheimer's, who are carriers of two copies of the ApoE4 gene, a known risk factor for the disease. While the trial concluded last year, the company delayed sharing data, initially expected in Q3 2024, without providing a specific reason. The upcoming presentation in April is highly anticipated by the Alzheimer's research community.
Blarcamesine Seeks European Approval
Anavex Life Sciences' blarcamesine, a small molecule drug that activates the sigma-1 protein in the brain, has been submitted for approval to European regulators. The drug completed its Phase 2/3 trial in 2022. However, the trial saw a significant dropout rate of approximately one-third of participants due to side effects. Despite the company's positive reports, the drug's efficacy has been questioned, leading to shareholder lawsuits alleging data misrepresentation. Anavex presented positive post-trial data on 300 participants who continued taking the drug for three years after the trial concluded.
Athira Pharma Halts Fosgonimeton Development
Athira Pharma has discontinued the development of fosgonimeton, an injectable drug intended to activate the HGF/MET cellular signaling pathway, which has shown promise in preclinical studies for supporting brain health and protecting against neurodegeneration. This decision follows the failure to demonstrate clinical benefits in the latest clinical trial. Adding to the company's challenges, Athira Pharma reached a settlement with the U.S. Department of Justice, agreeing to pay over $4 million to resolve allegations of using falsified data in studies to secure NIH grant funding. The company's CEO had previously stepped down in 2021 due to a data manipulation scandal related to her research on the HGF/MET pathway.
Leqembi: Potential for Injectable Formulation
Eisai and Biogen's Leqembi, an anti-amyloid antibody drug already approved by the FDA, may soon be available in a more convenient injectable form. The FDA has accepted the application for this new formulation, which could be administered at home using an injector pen, similar to insulin or Ozempic. A decision on the approval of the injectable version is expected by the end of August 2025. This new formulation aims to improve patient access and ease of administration.
Axsome's AXS-05 for Agitation in Alzheimer's
Axsome Therapeutics is seeking FDA approval for AXS-05, a small molecule drug already approved for depression (Auvelity), as a treatment for agitation in Alzheimer's patients. The company is basing its application on two Phase 3 trials, although some experts have raised concerns about the drug's effectiveness for this indication. AXS-05 targets multiple proteins in the brain, and the company believes its effects on brain signaling may alleviate agitation associated with dementia.