Lexeo's Gene Therapy Shows Promise for APOE4 Alzheimer's Patients in Early Trials
October 30, 2024The therapy aims to deliver a protective APOE2 allele to patients with two copies of APOE4, potentially slowing or halting the progression of Alzheimer's.
Lexeo Therapeutics has reported promising interim results from its Phase I/II study of LX1001, a gene therapy targeting Alzheimer's disease in patients with the APOE4 genetic variant.
In the study, APOE2 was detected in all patients' cerebrospinal fluid, with most participants showing stabilization of amyloid pathology and reductions in tau protein levels after 12 months.
Notably, the study observed improvements particularly in participants with moderate Alzheimer's disease.
The trial results indicated a dose- and time-dependent increase in APOE2 expression and reductions in cerebrospinal fluid tau biomarkers, suggesting a positive treatment effect.
With approximately 900,000 individuals in the U.S. estimated to be APOE4 homozygous patients with Alzheimer's, LX1001's targeted approach is particularly significant.
This development is part of a broader trend in the biotech industry, where companies are increasingly exploring gene therapies to address complex conditions like Alzheimer's.
LX1001 was well tolerated across all dose cohorts, with no reports of amyloid-related imaging abnormalities (ARIA), which is a common concern in Alzheimer's treatments.
While four serious adverse events were reported, only one was potentially related to treatment, involving mild-moderate sensorineural hearing loss.
Currently, APOE4 homozygotes lack effective treatment options, making LX1001 a hopeful prospect for these patients and their families.
Lexeo Therapeutics plans to engage with the FDA regarding the study data and anticipates updates on regulatory interactions and development plans in 2025.
The findings presented at the Clinical Trials on Alzheimer’s Disease (CTAD) conference highlight the potential of LX1001 as a new therapeutic approach for Alzheimer's.
Summary based on 5 sources
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Sources
Endpoints News • Oct 30, 2024
Lexeo shares early data on Alzheimer’s gene therapyInside Precision Medicine • Oct 30, 2024
Lexeo Therapeutics Scores Positive Early Data for APOE4-Alzheimer’s Gene Therapy