Clinical Read2026-06-17

LX2006 - (SUNRISE-FA)

Lexeo Therapeutics, Inc.LXEO
Indication
Cardiac Friedreich’s Ataxia
Stage
phase1
Event
Topline Data
Details

June 17, 2026: Lexeo Therapeutics, Inc. (NASDAQ: LXEO) announced publication in JAMA Cardiology of combined Phase I/II results for LX2006 gene therapy in Friedreich ataxia (FA) cardiomyopathy, pooling nine participants from a Weill Cornell Medicine study and eight from Lexeo’s SUNRISE-FA study (17 total). Patients received a one-hour IV infusion and were followed 6–36 months across three dose cohorts. Reported findings included LVMI improvement or stabilization in most participants; among those with abnormal baseline LVMI in mid/high-dose cohorts (n=3), mean LVMI improved 28% at 6 months and 33% at 12 months, with some maintaining improvement up to three years. Secondary cardiac biomarkers (high-sensitivity troponin I, lateral wall thickness) generally improved or stabilized, and SUNRISE-FA cardiac biopsies (n=8) showed increased frataxin protein expression at 3 months in all participants. mFARS stabilization was also reported. Safety: generally well tolerated with no Grade 3+ SAEs to date; minimal transient LFT elevations; one possibly treatment-related Grade 2 asymptomatic myocarditis at one year.

Upcoming catalyst: SUNRISE-FA 2 pivotal/registrational study is on track to initiate by end of June (also noted as Q2 2026), with topline data expected in 2H 2027.