Lexeo Therapeutics, Inc. (NASDAQ: LXEO) has announced positive interim data of LX2006 for the treatment of Friedreich ataxia (FA) cardiomyopathy. The company reported a mean reduction in left ventricular mass index (LVMI) of 11.4% at 12 months and 18.3% at 18 months in participants with elevated LVMI at baseline. Additionally, more than 75% of participants with elevated LVMI at baseline achieved a greater than 10% reduction in LVMI at 12 months.
Furthermore, sustained and consistent improvements in other key measures of cardiac status were observed, including left ventricular wall thickness and troponin I in the majority of participants at 12 months. Post-treatment frataxin expression increased above baseline in all participants evaluated via myocardial biopsy to date.
The interim safety results indicated that LX2006 was well tolerated with no treatment-related serious adverse events to date in either study. No signs of complement activation or other immunogenicity were observed, and there were no cardiac or hepatic safety signals detected. All adverse events were transient and resolved, and no participants discontinued from either study.
As of July 15, 2024, 13 participants had been dosed across two trials, with the Sunrise-FA independent data and safety monitoring board endorsing proceeding to cohort 3 dose level. The company also expects to share further details of these interim results, including an additional cardiac biopsy from cohort 2, at a scientific conference in fall 2024.
LX2006 is an AAV-based gene therapy candidate delivered intravenously for the treatment of FA cardiomyopathy, which is designed to target the cardiac manifestations of FA by delivering a functional frataxin gene to promote the expression of the frataxin protein and restore mitochondrial function in myocardial cells.
Today the company's shares have moved -26.0% to a price of $13.03. For the full picture, make sure to review Lexeo Therapeutics's 8-K report.
