Lexeo Down Despite Upbeat Rare Disease Gene Therapy Data

Shares of Lexeo Therapeutics (NASDAQ: LXEO) lost 26% on Monday after it reported positive interim data from two cohorts of the phase I/II SUNRISE-FA study evaluating LX2006, an investigational gene therapy for treating Friedreich ataxia (FA) cardiomyopathy.

Data from the SUNRISE-FA study showed that treatment with LX2006 achieved 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. LVMI is an estimator of cardiac morbidity and mortality. Treatment with the therapy was also well-tolerated across all study participants, with no treatment-related serious adverse events.

The interim data also showed that treatment with LX2006 also showed improvement in other key measures of cardiac status, including left ventricular wall thickness (an early indicator of an inefficient heart) and troponin I (a biomarker of myocardial injury), in the majority of participants at 12 months.

Based on the above results, management sees a path for seeking the FDA’s approval for LX2006 under the accelerated pathway.

Lexeo intends to report additional data at a future …

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