Lexeo Therapeutics (LXEO) Corporate presentation summary

Strategic focus and platform strengths

• Specializes in genetic medicines for rare cardiac diseases, aiming to transform cardiovascular care through gene therapy innovations.

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

• Employs an optimized Sf9 baculovirus manufacturing process, supporting high yield, scalability, and improved product purity.

• Maintains a strong financial position with a cash runway projected into 2028, supporting ongoing and future milestones.

Clinical pipeline and programs

• LX2006 targets Friedreich Ataxia Cardiomyopathy (FA-CM), addressing a major cause of mortality in FA with no approved cardiac-specific therapies.

• LX2020 is in clinical development for PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM), a genetic heart disease with high unmet need and no disease-modifying treatments.

• Two preclinical programs, LX2021 (Desmoplakin Cardiomyopathy) and LX2022 (Hypertrophic Cardiomyopathy), are advancing toward IND-enabling studies and regulatory engagement in 2026.

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

LX2006 clinical data and impact

• Interim data show LX2006 provides sustained or deepening improvements in cardiac structure (LVMI), biomarkers, and functional outcomes in FA-CM patients.

• Statistically significant improvements in neurological function (mFARS) compared to matched controls, suggesting broader disease impact.

• LX2006 is generally well tolerated, with no Grade 3 treatment-related serious adverse events and minimal transient liver function test elevations.

• Pivotal trial in FA-CM expected to initiate in 2026, with FDA alignment on endpoints and process validation.