Clinical Data and Outcomes - Participants with abnormal LVMI at baseline achieved a mean LVMI reduction of 18% at 6 months and 23% at 12 months[5, 47] - In participants with abnormal LVMI at baseline, Cohorts 2 and 3 showed a mean LVMI reduction of 28% at 6 months and 33% at 12 months[25] - 11 out of 16 participants showed improvement or stabilization in mFARS relative to baseline at the latest visit, indicating neurological functional improvement[39, 40] - All participants evaluated showed increases in frataxin expression vs baseline[45] Regulatory and Study Design - The FDA is open to a BLA submission that includes pooled clinical data from ongoing Phase I/II studies of LX2006 with new clinical data from the planned pivotal study[5] - The FDA agreed to evaluate the LVMI co-primary endpoint at a time point earlier than 12 months, potentially reducing the size and length of the pivotal study[5, 17] - Lexeo plans to initiate the pivotal study in the first half of 2026[47] Safety and Tolerability - LX2006 has been generally well-tolerated, with no clinically significant complement activation and minimal, transient LFT elevations[3, 47] - No participants discontinued from either study[20] Disease Context - Cardiac complications account for up to 80% of deaths in individuals with Friedreich Ataxia (FA)[9, 10] - Up to 40% of adults with FA have left ventricular hypertrophy as defined by abnormal LVMI[10]
Lexeo Therapeutics (NasdaqGM:LXEO) Earnings Call Presentation