Lexeo Therapeutics Announces Progress in FDA Discussions for Accelerated Approval Pathway and Positive Interim Clinical Data for LX2006 in Friedreich Ataxia Cardiomyopathy

Core Insights - The U.S. FDA is open to pooling data from ongoing Phase I/II studies of LX2006 with pivotal data to support a Biologics License Application (BLA) for Accelerated Approval [1][4] - Interim clinical data show significant improvements in cardiac and neurologic measures for participants with Friedreich ataxia [1][6] - The company plans to initiate a pivotal study for LX2006 in the first half of 2026, pending finalization of the trial protocol [5] FDA Engagement - The FDA has indicated a willingness to accept a BLA submission that includes pooled clinical data from ongoing Phase I/II studies and new data from the planned pivotal study [4] - Lexeo Therapeutics is required to submit enhanced manufacturing comparability data and meet additional nonclinical requirements before starting the pivotal study [4] Clinical Data Highlights - Participants with abnormal left ventricular mass index (LVMI) achieved an 18% mean reduction at 6 months and a 23% mean reduction at 12 months, surpassing the FDA's target threshold of 10% [1][6] - The modified Friedreich Ataxia Rating Scale (mFARS) showed clinically meaningful improvements, indicating slowed disease progression [1][6] - Among participants with abnormal baseline LVMI, all achieved LVMI measurements within the normal range, and five out of six participants showed over 10% improvement by 12 months [7] Safety and Efficacy - LX2006 has demonstrated encouraging safety and efficacy, with no Grade 3+ serious adverse events reported to date [13] - The treatment has been well tolerated, with minimal transient liver function test elevations and no signs of frataxin over-expression in cardiac tissue [13] Company Overview - Lexeo Therapeutics is focused on developing gene therapies for cardiovascular diseases, with LX2006 targeting Friedreich ataxia cardiomyopathy, which affects approximately 5,000 individuals in the U.S. [11][12] - The company has received multiple designations from the FDA, including Breakthrough Therapy and Fast Track, to expedite the development of LX2006 [5][11]