Lexeo Therapeutics (LXEO) Oppenheimer 36th Annual Healthcare Life Sciences Conference summary

Strategic positioning and platform differentiation

• Focus on precision medicine for inherited cardiac diseases using AAVrh.10 vector, which shows superior heart biodistribution compared to other vectors.

• Clinical validation of cardiotropism and safety profile in both Friedreich's ataxia (FA) and PKP2-associated arrhythmogenic cardiomyopathy (PKP2-ACM) programs.

• Utilization of robust trial design tools, including non-invasive biomarkers and hard clinical endpoints.

Friedreich's ataxia (FA) program highlights

• FA affects about 15,000 patients globally, with 70% mortality due to cardiac disease.

• LX2006 gene therapy shows significant reductions in left ventricular mass index (LVMI) and troponin, reversing cardiac hypertrophy.

• Therapy demonstrates benefit in both cardiac and neurologic endpoints, potentially suitable for all FA patients, including pediatric cohorts.

• Dual primary endpoints for pivotal study: increase in frataxin and ≥10% improvement in LVMI.

• Natural history study accelerates enrollment and provides comparative data on untreated disease progression.

Regulatory and clinical development plans

• Finalizing pivotal study design for FA with FDA, focusing on LVMI as the key endpoint for accelerated approval.

• Natural history study supports trial design and regulatory discussions, ensuring robust data on disease progression.

• Expectation to report pivotal study results in early 2026, with study start before end of first half of the year.