Lexeo Therapeutics (LXEO.US) announced positive results from its Phase II clinical trial of its gene therapy, LX2006.
July 16, 2021, Lexeo Therapeutics (LXEO.US) announced positive mid-term data for its investigational gene therapy treatment for Friedreich's Ataxia (FA) cardiomyopathy, LX2006. In the SUNRISE-FA Phase 1/2 trial and another Phase 1A trial, patients showed a 53.3% improvement in cardiac biomarkers with clinical significance. Based on these positive results, Lexeo plans to accelerate the development of the therapy, including discussions with regulatory agencies on the design and submission of a pivotal trial for potential accelerated approval.
LX2006 is an investigational gene therapy based on adeno-associated virus (AAV) designed to promote expression of the functional FXN gene and restore mitochondrial function in cardiomyocytes through intravenous delivery. In preclinical studies, LX2006 reversed cardiac abnormalities in the FA disease model, showing improvement in cardiac function and survival while demonstrating good safety. The FDA has granted rare pediatric disease, fast track, and orphan drug designations for LX2006 to treat FA cardiomyopathy.