Lexeo Therapeutics Unveils Promising Interim Data for LX1001 Gene Therapy in APOE4-Associated Alzheimer’s Disease

Positive Interim Data:
Lexeo Therapeutics has announced positive interim data from its ongoing Phase 1/2 clinical trial of LX1001, an AAV-based gene therapy candidate designed to deliver the protective APOE2 gene into the central nervous system of APOE4 homozygous Alzheimer’s disease patients.

Safety and Efficacy:
The data show an emerging favorable tolerability profile with no serious adverse events reported. Additionally, there is evidence of APOE2 expression in the cerebrospinal fluid and declines in tau and amyloid biomarkers, indicating potential therapeutic efficacy.

Clinical Trial Details:
The Phase 1/2 trial is an open-label, dose-ranging study evaluating the safety and tolerability of LX1001 in 15 Alzheimer’s disease patients who are homozygous for the APOE4 allele. The trial includes four dose cohorts, with 12-month data presented for cohorts 1-3 and 6-month data for cohort 4.

Presentation at CTAD 2024:
The interim data were selected for a late-breaking oral presentation at the Clinical Trials on Alzheimer’s Disease (CTAD) 2024 conference, highlighting the safety and efficacy of LX1001 across four dose cohorts.

Fast Track Designation:
LX1001 has been granted Fast Track designation by the U.S. Food and Drug Administration, expediting its review and development to meet an unmet medical need.

Future Plans:
Lexeo Therapeutics plans to continue advancing LX1001 and other preclinical stage programs targeting APOE4-associated Alzheimer’s disease, leveraging early proof-of-concept functional and biomarker data.