Gene Therapy for Friedreich Ataxia Cardiomyopathy Set to Move Forward


The FDA has approved LEXEO Therapeutics’ investigational new drug application.

LEXEO Therapeutics has announced that the FDA has approved its investigational new drug application for its gene therapy candidate, LX2006, for the potential treatment of Friedreich ataxia cardiomyopathy (FA cardiomyopathy).1

The regulatory milestone marks the first clinical-stage cardiovascular program for the company, and its third clinical-stage gene therapy candidate overall.

“Representing our third clinical-stage program overall, LX2006 is also the first disease-modifying, clinical-stage gene therapy treatment for Friedreich ataxia and the first clinical-stage program from our cardiovascular gene therapy pipeline,” R. Nolan Townsend, chief executive officer, LEXEO, said in a statement.

The intravenous, AAV-based gene therapy is designed to target the cardiac manifestations of FA, which are the leading cause of death in this population, by delivering a functional frataxin gene to promote frataxin protein expression and restore mitochondrial functional in myocardial cells.

“The potential of LX2006 is supported by our robust preclinical studies showing that LX2006 was able to significantly reverse the cardiac manifestations of the disease,” added Jay A. Barth, MD, executive vice president and chief medical officer, LEXEO. Improvement in cardiac function and survival, as well as a favorable safety profile, were also observed in the preclinical analyses.

LEXEO plans to initiate a phase 1/2 clinical trial in mid-2022, which will be a 52-week, dose-ascending, open-label trial in patients with FA cardiomyopathy, who will receive a single intravenous infusion of LX2006 as part of 2 dose cohorts with 5 patients assigned to each.

Notably, the FDA previously granted rare pediatric disease designation and orphan drug designation to the gene therapy for the treatment of FA in June 2021.2

LEXEO’s pipeline also includes 3 other cardiac-targeted gene therapy candidates that are in the discovery phase, as well as 4 additional gene therapy candidates targeted towards central nervous system disorders, 2 of which are in clinical-stage development: LX1001 for the treatment of APOE2+ Alzheimer disease, which has received fast track designation,3 and LX1004 for the treatment of CLN2 Batten disease, which has received rare pediatric disease and orphan drug designations from the FDA.4

1. LEXEO Announces FDA Clearance of IND Application for LX2006 for Friedreich’s Ataxia Cardiomyopathy. News release. LEXEO Therapeutics. February 16, 2022.
2. LEXEO Therapeutics Receives Rare Pediatric Disease & Orphan Drug Designations for LX2006. News release. LEXEO Therapeutics. June 30, 2021.
3. LEXEO Therapeutics Announces FDA Fast Track Designation Granted to LX1001. News release. LEXEO Therapeutics. April 20, 2021.
4. LEXEO Therapeutics Receives Rare Pediatric Disease & Orphan Drug Designations for LX1004. News release. LEXEO Therapeutics. July 28, 2021.
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