Clinical Trials

Date: 2022-03-02

Type of information: Interim results

phase: 1-2

Announcement: interim results

Company: Lexeo Therapeutics (USA-NY)

Product: LX1001

Action mechanism: gene therapy. AAV-based investigational gene therapy designed to deliver the protective apolipoprotein E2 (APOE2) gene into the CNS of APOE4 homozygous Alzheimer’s disease patients to halt or slow disease progression.

Disease: Alzheimer's disease

Therapeutic area: Neurodegenerative diseases

Country: USA

Trial details:

The Phase 1/2 clinical trial is an open-label, dose-ranging study evaluating the safety and tolerability of LX1001 in approximately 15 Alzheimer’s disease patients 50 years of age or older who each have two copies of the APOE4 allele (APOE4 homozygous patients). The trial consists of three dose-ascending cohorts. Secondary endpoints include evaluation of the conversion of the cerebrospinal fluid (CSF) from the APOE4 homozygous profile to an APOE4/E2 profile and CSF biomarkers. This trial is enrolling patients with clinical diagnoses ranging from mild cognitive impairment to mild or moderate dementia. Patients must also have evidence of amyloid plaques and CSF biomarkers consistent with Alzheimer’s disease to be included in the trial. (NCT03634007)

Latest news: • On March 2, 2022, Lexeo Therapeutics, a clinical-stage gene therapy company advancing a deep and diverse pipeline of adeno-associated virus (AAV)-based gene therapy candidates for genetically defined cardiovascular and central nervous system (CNS) diseases, announced positive initial expression and biomarker data from the low-dose cohort (cohort 1) of its ongoing Phase 1/2 clinical trial of LX1001. Key initial results from four evaluated patients in cohort 1 of the Phase 1/2 clinical trial showed :

Expression of the protective APOE2 protein in the CSF in all evaluated patients with follow-up data at three months or longer (two patients with data through the month-3 visit, and two patients with data through the month-12 visit)
- Increases in CSF APOE2 levels from baseline, relative to their respective CSF APOE4 levels in all evaluated patients with follow-up data at the month-3 visit
- Increases in CSF APOE2 levels from baseline, relative to their respective CSF APOE4 levels in the two patients with follow-up data at the month-12 visit
Declines in CSF core biomarkers total tau (T-tau) and phosphorylated tau (P-tau) protein levels relative to baseline in the two patients with data through the month-12 visit
- T-tau and P-tau protein levels are widely accepted biomarkers that reflect key pathological changes in the brain of Alzheimer’s disease patients, such as accumulation of tau proteins that occur during the neurodegenerative process
Generally well-tolerated with no serious adverse events to date

Initial data from the mid-dose cohort (cohort 2) and additional 12-month follow-up data from cohort 1 are expected in the second half of 2022 and will be presented at a future medical conference.

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