Lexeo Therapeutics announces FDA Breakthrough Therapy Designation for LX2006

Lexeo Therapeutics (LXEO) announced that the U.S. Food and Drug administration has granted Breakthrough Therapy designation to LX2006 based on clinical evidence generated on both cardiac and neurologic measures of Friedreich ataxia. LX2006 has also been selected to participate in the FDA Chemistry, Manufacturing, and Controls Development and Readiness Pilot program, intended to enable earlier patient access to therapies with expedited clinical development timelines. The FDA decision was based on interim clinical data demonstrating that treatment with LX2006 was associated with clinically significant improvements in cardiac biomarkers and in cardiac and neurologic functional measures, with increased frataxin expression observed in all participants with cardiac biopsies at three months post treatment. To date, 17 participants have been treated across two trials: the Lexeo-sponsored SUNRISE-FA Phase 1/2 clinical trial and the Weill Cornell Medicine investigator-initiated Phase 1A trial. Lexeo is currently enrolling a prospective natural history study, CLARITY-FA, which will serve as a concurrent external control arm for the registrational study. The Company expects to initiate the registrational study by early 2026 and is actively working with FDA to finalize the statistical analysis plan.