Alector Announces Results from its Phase 3 Clinical Trial of Potential FTD-GRN Therapeutic

King of Prussia, Pa., Oct. 21, 2025 (GLOBE NEWSWIRE) -- Biotechnology company Alector announced today that the drug, latozinemab, failed to meet the criteria for safety and efficacy in a Phase 3 clinical trial. The study investigated the drug as an intervention for cases of FTD caused by a variant of the gene GRN. Three genes, GRN, MAPT, and C9orf72, cause the majority of genetic FTD—itself roughly 20% of FTD cases. “Even though this trial shows that latozinemab is not the treatment FTD-GRN families had hoped for, it represents a major step forward as the first trial of its kind for the FTD field. We must—and will—continue to support clinical research, knowing that each trial increases our knowledge of FTD and inspires the hope that a future free of FTD is within sight,” said Susan L-J Dickinson, AFTD CEO. In some families, FTD is caused by inherited variations in the GRN gene that impedes the ability of the body to produce progranulin (PRGN), a protein that aids in cell survival and helps regulate inflammation. Lowered levels of PRGN are associated with neurodegeneration. Latozinemab works by inhibiting a receptor that causes the natural degradation of PRGN. In the clinical trial, treatment with latozinemab did result in a significant increase in the levels of plasma PGRN. However, the treatment did not slow progression of disease as measured by the clinical co-primary endpoint, a common clinical assessment referenced by the acronym CDR plus NACC FTLD-SB. Biological markers of disease progression were also unaffected, such as fluid biomarkers and volumetric magnetic resonance imaging (vMRI). Treatment appeared to be safe, with no increased risk of adverse events during the trial. Alector was developing the drug in collaboration with the biopharmaceuticals firm GSK. Phase 3 is the largest and most critical phase in evaluating new treatments before they can be approved for public use. With the results announced today, Alector has concluded the study. Based on these results, the open-label extension portion of the trial and the continuation study will both be discontinued.Participants can expect to hear soon from their study site team. “While the results are certainly disappointing,” said AFTD Senior Director of Scientific Initiatives Penny Dacks, PhD, “it represents an advance in our knowledge of how to run FTD clinical trials and in which types of treatments will work or not. We extend our thanks to the team at Alector and to the many families that participated in this pivotal research study.” Other avenues of research are being aggressively pursued with alternative methods of raising PGRN levels and other treatments unrelated to PGRN. “Our goal is safe, accessible treatments for everyone,” said Dacks. “Our strategy as a field should be multiple shots on goal, where we learn from every clinical trial.” To encourage drug development for all types of FTD, AFTD encourages everyone to enroll in the FTD Disorders Registry (ftdregistry.org). The more people in that secure, non-profit database, the more that we can show drug developers that the FTD community is all-in to develop effective treatments, and the more shots-on-goal we will have to find effective treatments. About FTD: FTD is an umbrella term for clinical diseases with progressive degeneration of the frontal and temporal lobes of the brain. Roughly 20% of people diagnosed with FTD have an underlying genetic cause. Pathogenic variants in more than a dozen genes can cause FTD; however, the most common genes are C9orf72, GRN, and MAPT. Unlike Alzheimer's, which is characterized by a decline in memory, FTD causes irreversible changes in personality, behavior, language, and/or movement, while leaving memory relatively preserved. About AlectorAlector is a late-stage clinical biotechnology company focused on developing therapies to counteract the devastating progression of neurodegenerative diseases. Leveraging the principles of genetics, immunology, and neuroscience, the company is advancing a portfolio of genetically validated programs that aim to remove toxic proteins, replace deficient proteins, and restore immune and nerve cell function. Supported by biomarkers, Alector’s product candidates seek to treat a range of indications, such as frontotemporal dementia, Alzheimer’s disease, and Parkinson's disease. The company is also developing Alector Brain Carrier (ABC), a proprietary blood-brain barrier platform, which is being selectively applied to its next-generation product candidates and research pipeline. ABC aims to enhance the delivery of therapeutics, achieve deeper brain penetration and efficacy at lower doses, and ultimately improve patient outcomes while reducing costs. Alector is headquartered in South San Francisco, California. For more information, please visit www.alector.com. About AFTD: Online at theaftd.org, AFTD is the largest national nonprofit devoted to providing resources to help families affected by FTD today, and advancing research to foster accurate diagnosis, treatments, and a cure. Our volunteer-founded organization – driven by thousands of volunteers and donors – reflects a community's profound determination to #endFTD. SOURCE: The Association for Frontotemporal Degeneration

Alector Announces Results from its Phase 3 Clinical Trial of Potential FTD-GRN Therapeutic

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