Lexeo Therapeutics Finalizes Pivotal Trial Design for LX2006 in Friedreich Ataxia Cardiomyopathy

Lexeo Therapeutics has finalized the protocol and statistical analysis plan (SAP) for SUNRISE-FA 2, a pivotal trial of its gene therapy candidate LX2006 for Friedreich ataxia (FA) cardiomyopathy. The open-label study will enroll 26 participants, with 13 receiving a single high-dose IV administration of LX2006 and 13 serving as an untreated control group. The primary endpoint is the change in left ventricular mass index (LVMI) at six months, assessed via cardiac MRI. The trial design incorporates FDA feedback, including the removal of a protein expression co-primary endpoint. Lexeo expects to initiate the trial and enroll the first patient by the end of June 2026. The company is pursuing an accelerated approval pathway, with topline data expected in the second half of 2027 and a potential Biologics License Application (BLA) submission in the first half of 2028. Additionally, Lexeo reported clinical data from 17 participants in its Phase I/II study, showing sustained improvements in LVMI and mFARS scores, with the therapy being generally well tolerated.