Date: 2022-02-16
Type of information: Clinical trial authorisation
phase: 1-2
Announcement: clinical trial authorization
Company: Lexeo Therapeutics (USA-NY)
Product: LX2006
Action mechanism: gene therapy. AAV-based gene therapy candidate designed to intravenously deliver a functional frataxin gene and promote the expression of the frataxin protein and restore mitochondrial function in myocardial cells. In preclinical studies, LX2006 reversed the cardiac abnormalities in Friedreich's ataxia disease models and showed improvement in cardiac function and survival while demonstrating a favorable safety profile. The FDA has granted Rare Pediatric Disease designation and Orphan Drug designation to LX2006 for the treatment of Friedreich’s ataxia.
Disease: Friedreich's ataxia cardiomyopathy
Therapeutic area: Rare diseases - Genetic diseases -Neurodegenerative diseases - Cardiovascular diseases
Country: USA
Trial details: The Phase 1/2 study is a 52-week, dose-ascending, open-label trial of LX2006 in patients who have FA cardiomyopathy. LX2006 will be administered as a one-time intravenous infusion to patients in two ascending-dose cohorts of five patients each.
Latest news: • On February 16, 2022, Lexeo Therapeutics announced that the FDA has cleared its Investigational New Drug (IND) application for LX2006. LX2006 is an AAV-based gene therapy candidate designed to intravenously deliver a functional frataxin gene, for the treatment of Friedreich’s ataxia cardiomyopathy (FA cardiomyopathy), the most common cause of mortality in patients with Friedreich’s ataxia.. Lexeo plans to initiate this open-label, dose-escalation Phase 1/2 clinical trial of LX2006 in patients with FA cardiomyopathy in the middle of 2022.
