Lexeo's Gene Therapy Shows Promise for APOE4 Alzheimer's Patients in Early Trials

October 30, 2024
Lexeo's Gene Therapy Shows Promise for APOE4 Alzheimer's Patients in Early Trials
  • The 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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