Early Results Show Promise for Lexeo's Gene Therapy in Rare Heart Disease

Lexeo Therapeutics has reported encouraging interim data from its Phase I/II clinical trial of LX2006, a gene therapy aimed at treating Friedreich Ataxia Cardiomyopathy. The therapy showed a significant reduction in left ventricular mass index (LVMI), with a mean reduction of 11.4% at 12 months and 18.3% at 18 months among participants with elevated LVMI at baseline. Other key cardiac measures, such as left ventricular wall thickness and troponin I levels, also improved consistently. LX2006 was well tolerated with no treatment-related serious adverse events and demonstrated clinically meaningful improvements in cardiac biomarkers.

The company has dosed 11 patients so far, including four with abnormally high LVMI. The therapy not only showed reductions in hypertrophy but also an increase in the frataxin protein, which is crucial for heart muscle function. Lexeo Therapeutics is exploring the potential for accelerated approval of LX2006, driven by the positive interim results. However, challenges remain in assessing the therapy's impact on cardiorespiratory fitness, as evidenced by gaps in V02 max data. The company plans to continue evaluating these measures in future studies and aims to present more detailed findings at upcoming medical congresses.