LX2006 (FA Cardiomyopathy) - LX2006 is the only clinical program targeting Friedreich Ataxia (FA) cardiomyopathy, which causes death in 60-80% of individuals with FA[5, 21, 24] - Interim clinical data shows robust cardiac FXN expression in all participants and clinically meaningful reductions in multiple cardiomyopathy markers[5] - FDA alignment on key elements of accelerated approval pathway based on LVMI reduction and protein expression, with a registrational study expected to start by early 2026 and potential efficacy readout in 2027[5] - Natural history study showed a 19% higher risk of death per 10g/m2 increase in LVMI in Friedreich Ataxia (FA)[32] - Interim Phase 1/2 results show participants with abnormal LVMI at baseline achieved a mean reduction of 25% in LVMI at 12 months or sooner[42] - All participants in the LX2006 trials showed an increase in frataxin expression versus baseline, with Cohort 3 averaging a 115% increase[42, 45] - 5 out of 6 participants with abnormal LVMI at baseline achieved >10% reduction in LVMI by 12-month or sooner visit[48, 50] LX2020 (PKP2-ACM) - LX2020 is a potential best-in-class treatment for PKP2-ACM, affecting approximately 60,000 people in the US with no disease-modifying treatment available[5, 63] - Observed increased protein expression levels in two post-treatment cardiac biopsies and a 67% reduction in PVCs from baseline in one participant that reached 6-months[5, 91, 92] General - AAVrh10 demonstrates approximately 1.5x to 2x greater biodistribution to the heart compared to AAV9 in large animal models[12, 16] - Lexeo Therapeutics has approximately $181 million in pro forma cash and marketable securities, projecting a runway into 2028 with 54 million pro forma shares of common stock[95]
Lexeo Therapeutics (LXEO) Earnings Call Presentation