Lexeo Therapeutics (LXEO) investor relations material

Patient impact and disease focus

• Highlighted the urgent unmet need in Friedreich's ataxia, emphasizing the devastating cardiac complications and lack of curative treatments.

• Patient stories underscored the emotional and clinical burden, motivating ongoing research and advocacy.

• Emphasized that 80% of Friedreich's ataxia patients die from cardiac disease, making cardiac intervention critical.

• Expressed hope that new therapies could transform patient outcomes and quality of life.

Platform, technology, and pipeline evolution

• Developed a leading cardiac genetic medicines platform using the AAVrh10 capsid, which offers superior heart targeting and safety at lower doses.

• Manufacturing platform supports both rare and prevalent cardiac diseases, achieving high vector yields and low empty-capsid ratios.

• Pipeline includes programs for Friedreich's ataxia, PKP2 arrhythmogenic cardiomyopathy, and preclinical efforts in desmoplakin-mediated and hypertrophic cardiomyopathies.

• Collaboration with Johnson & Johnson aims to advance localized gene therapy delivery for diseases requiring higher protein expression.

Clinical progress and data highlights

• Friedreich's ataxia program showed a 115% increase in frataxin expression and a 33% reduction in left ventricular mass index at 12 months, reversing cardiac pathology.

• Demonstrated 2-3 point improvements on the mFARS neurologic scale, matching or exceeding current treatments.

• PKP2 arrhythmogenic cardiomyopathy program achieved up to 162% increase in PKP2 expression and a 22% reduction in nonsustained ventricular tachycardia at six months, with deeper effects at nine months.

• Both programs reported clean safety profiles, with no gene therapy-related grade 3 SAEs and manageable LFT elevations.