Lexeo Therapeutics (LXEO) investor relations material

Strategic focus and platform strengths

• Focused on genetic medicines for rare cardiac diseases, aiming to reshape heart health through pioneering science and gene therapy innovations.

• Platform leverages a differentiated AAVrh.10 capsid with proven cardiac tropism, enabling lower doses and improved therapeutic index.

• Optimized Sf9 baculovirus manufacturing platform supports high-yield, commercial-scale vector production with attractive economics.

• Strong financial position with a cash runway into 2028, supporting multiple value-creating milestones.

Pipeline and clinical programs

• Pipeline includes two clinical-stage programs: LX2006 for Friedreich Ataxia Cardiomyopathy (FA-CM) and LX2020 for PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM), plus two preclinical programs targeting Desmoplakin and Hypertrophic Cardiomyopathies.

• LX2006 is the only clinical-stage gene therapy for FA-CM, with data showing sustained improvements in cardiac and neurologic measures and a favorable safety profile.

• LX2020 targets PKP2-ACM, a disease with no disease-modifying treatments; interim data show encouraging efficacy and safety signals.

• Preclinical programs address significant unmet needs in desmoplakin and hypertrophic cardiomyopathies, with ongoing IND-enabling studies.

Clinical data and outcomes

• LX2006 demonstrated durable reductions in left ventricular mass index (LVMi) and improvements in cardiac biomarkers, with most participants reaching normal LVMi and reduced troponin I levels.

• Statistically significant improvements in neurological function (mFARS scores) compared to matched controls, suggesting benefit beyond cardiac endpoints.

• LX2020 showed increased PKP2 expression and reductions in arrhythmia burden (PVCs and NSVT) in high-dose cohorts, with most participants reporting improved patient-reported outcomes.

• Both LX2006 and LX2020 have been generally well tolerated, with no clinically significant complement activation and manageable adverse events.