Lexeo Therapeutics (LXEO) investor relations material

Program highlights and clinical data

• Lead program uses AAV gene therapy for Friedreich's ataxia, showing normalization of heart mass and neurologic improvement comparable to approved therapies.

• Registrational study for Friedreich's ataxia to begin this year, with updates on statistical plan and study design expected in early 2026.

• Arrhythmogenic cardiomyopathy program targets PKP2 mutation, with early data showing significant reduction in ventricular tachycardia and improvement in ejection fraction.

• Preclinical pipeline focuses on genetic cardiomyopathies, leveraging AAV vectors for precision cardiac treatment.

Regulatory and study design updates

• FDA open to pooling phase I/II data with future registrational study; final statistical plan update expected early 2026.

• Efforts underway to minimize bias and clarify confirmatory endpoints, with cardiac endpoints likely for future studies.

• LVMI (left ventricular mass index) agreed as a key clinical endpoint, with a 10% improvement considered meaningful and linked to mortality reduction.

• FDA open to primary endpoints at time points shorter than 12 months; trade-offs between effect size and study duration under consideration.

Commercial and operational considerations

• Early adopters expected to be cardiologists, but neurologists also likely to engage due to neurologic benefits.

• Therapy uses a manageable immune suppression regimen and has shown a strong safety profile, with plans to include adolescent and pediatric cohorts.

• Manufacturing process transitioned to high-yield Sf9 suspension, with FDA-approved comparability protocol and CDRP designation to expedite BLA pathway.

• Prospective natural history study running in parallel, serving as a feeder for the treatment study and supporting BLA submission.