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 for high-yield, high-quality vector production, supporting scalability and commercial readiness.

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

Clinical pipeline and key programs

• LX2006 targets Friedreich Ataxia Cardiomyopathy (FA-CM), addressing a major cause of mortality in FA patients; pivotal trial initiation expected in Q2 2026.

• LX2020 is in phase 1/2 for PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM), with interim data showing promising efficacy and safety; regulatory engagement anticipated in 2026.

• Two preclinical programs: LX2021 for Desmoplakin Cardiomyopathy and LX2022 for Hypertrophic Cardiomyopathy, both addressing significant unmet needs.

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

LX2006 clinical data and impact

• Demonstrates sustained or deepening improvements in cardiac structure (LVMI), biomarkers, and functional outcomes in FA-CM patients.

• Statistically significant improvement in neurological function (mFARS) compared to matched controls.

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

• Durable cardiac improvements observed, including in patients with advanced disease.