Lexeo Therapeutics reveals promising heart gene therapy results

Lexeo Therapeutics has recently revealed encouraging interim results from its Phase 1 and 2 clinical trials for LX2006, a gene therapy developed to treat cardiomyopathy associated with Friedreich ataxia (FA). FA is a rare genetic disorder caused by a deficiency in the frataxin protein, which leads to various cardiac complications, including left ventricular hypertrophy (abnormal thickening of the left ventricle walls), heart failure, arrhythmias, and myocardial fibrosis. Patients with FA cardiomyopathy often experience symptoms such as shortness of breath, fatigue, and palpitations. While current management strategies rely on medications to alleviate symptoms, no treatments address the underlying cause of the disorder.

The trials for LX2006 have demonstrated promising outcomes, showing significant improvements in cardiac health markers. Notably, there was an 11.4% mean reduction in left ventricular mass index (LVMI) at 12 months and an 18.3% reduction at 18 months for participants who had elevated LVMI at the start of the trials. Furthermore, 75% of these participants exhibited over a 10% reduction in LVMI at the 12-month mark.

In addition to the decrease in LVMI, the trials also reported sustained enhancements in other cardiac status markers. Participants experienced a reduction in left ventricular wall thickness as well as decreased levels of troponin I, a protein that signifies heart muscle damage. Another significant finding was the increased expression of the frataxin protein in all participants who underwent myocardial biopsy evaluations.

So far, a total of 13 participants have been dosed across the two trials, providing a substantial amount of data to support the efficacy of LX2006. The results indicate that LX2006 not only addresses the symptoms of FA cardiomyopathy but also targets its root cause by increasing frataxin protein expression, thereby potentially altering the disease's progression.

These findings bring hope for a new and effective treatment option for individuals suffering from FA cardiomyopathy, offering a much-needed approach that goes beyond symptom management to tackling the disorder at its genetic foundation.