Lexeo Therapeutics shares promising results for heart gene therapy

Today, Lexeo Therapeutics announced positive interim phase 1 and 2 clinical data for LX2006, a gene therapy targeting Friedreich ataxia (FA) cardiomyopathy.

FA cardiomyopathy is a heart condition linked to Friedreich ataxia, a rare genetic disorder caused by frataxin protein deficiency. This leads to abnormal thickening of the heart's left ventricle walls (left ventricular hypertrophy), heart failure, arrhythmias, and myocardial fibrosis. Symptoms include shortness of breath, fatigue, and palpitations, and while management typically involves medications, there are no treatments targeting the root cause.

In trials, LX2006 showed promising results, including an 11.4% mean reduction in left ventricular mass index (LVMI) at 12 months and an 18.3% reduction at 18 months for participants with elevated LVMI at baseline. Additionally, 75% of these participants experienced over a 10% reduction in LVMI at 12 months.

The trials also reported sustained improvements in cardiac status markers, such as reduced left ventricular wall thickness and decreased troponin I levels. Increased frataxin protein expression was observed in all participants evaluated via myocardial biopsy. As of now, 13 participants have been dosed across two trials.