Lexeo Therapeutics (LXEO) Corporate presentation summary

Strategic focus and platform

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

• Platform leverages deep expertise in genetic cardiac disease, with two clinical and two preclinical programs.

• Utilizes a differentiated AAVrh10 capsid with proven cardiac tropism, enabling lower doses and improved therapeutic index.

• Innovative Sf9 baculovirus manufacturing platform supports high yield, scalability, and commercial readiness.

• Strong financial position with cash runway into 2028, supporting multiple milestones.

Clinical pipeline and programs

• LX2006 targets Friedreich Ataxia Cardiomyopathy (FA-CM), addressing a major cause of mortality in FA; pivotal SUNRISE-FA2 study to initiate in Q2 2026, with topline data expected in 2H 2027 and BLA filing in 1H 2028.

• LX2020 addresses PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM), a rare genetic disease with no disease-modifying treatments; 12-month high-dose data expected Q4 2026.

• Preclinical programs include LX2021 for Desmoplakin Cardiomyopathy and LX2022 for Hypertrophic Cardiomyopathy, both targeting high unmet needs.

• Research collaboration with Johnson & Johnson explores novel cardiac AAV gene therapy delivery routes.

Key clinical data and outcomes

• LX2006 shows sustained or deepening improvements in cardiac measures (LVMI, troponin I) and neurological function (mFARS), with most participants reaching normal LVMI and improved or stabilized mFARS scores.

• LX2006 generally well tolerated, with no Grade 3 treatment-related SAEs and minimal transient LFT elevations.

• LX2020 interim data show increased PKP2 expression, reduced or stabilized PVCs and NSVT, and improved patient-reported outcomes in high-dose cohort.

• LX2020 generally well tolerated; one Grade 3 SAE (sustained VT) resolved with standard care.