KING OF PRUSSIA, PA — Alector, Inc. announced that its experimental therapy latozinemab failed to meet primary efficacy and safety endpoints in a Phase 3 clinical trial evaluating the drug as a potential treatment for frontotemporal dementia caused by mutations in the GRN gene (FTD-GRN). The company conducted the study in collaboration with GSK.
The late-stage trial, the first of its kind targeting this rare genetic form of FTD, found that while treatment with latozinemab significantly increased plasma progranulin (PGRN) levels, it did not slow disease progression based on the trial’s co-primary clinical endpoint. The drug also failed to show an impact on biological markers of disease progression, including fluid biomarkers and volumetric MRI measures.
Despite the lack of efficacy, Alector reported that latozinemab appeared to be safe and well tolerated, with no elevated risk of adverse events among participants. Based on the results, the company said it would discontinue both the open-label extension and continuation studies associated with the program.
“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,” said Susan L.-J. Dickinson, CEO of The Association for Frontotemporal Degeneration (AFTD). “Each trial increases our knowledge of FTD and brings us closer to a future free of the disease.”
FTD-GRN is caused by inherited mutations in the GRN gene that reduce the body’s ability to produce progranulin, a protein critical for cell survival and inflammation regulation. Latozinemab was designed to inhibit a receptor that promotes progranulin degradation, theoretically restoring protein levels in patients.
Penny Dacks, Ph.D., AFTD’s Senior Director of Scientific Initiatives, described the results as disappointing but scientifically valuable. “It represents an advance in our knowledge of how to run FTD clinical trials and in which types of treatments will work or not,” Dacks said. “Our goal remains to develop safe, accessible treatments for everyone, and every study provides a new data point toward that end.”
Alector noted that the findings will inform ongoing research efforts aimed at developing next-generation therapies to raise progranulin levels or target other biological pathways involved in FTD.
Frontotemporal dementia is a group of brain disorders marked by progressive degeneration of the frontal and temporal lobes, often leading to profound changes in behavior, language, and movement. Roughly one in five cases is linked to a genetic cause, with GRN, MAPT, and C9orf72 among the most common genes involved.
Alector, headquartered in South San Francisco, is a late-stage biotechnology firm developing immuno-neurology therapies for neurodegenerative diseases, including FTD, Alzheimer’s, and Parkinson’s disease.
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