NEW YORK, July 15, 2024 (GLOBE NEWSWIRE) -- Lexeo Therapeutics, Inc. (Nasdaq: LXEO), announced positive interim data of LX2006 for the treatment of Friedreich ataxia (FA) cardiomyopathy. Across both the Lexeo SUNRISE-FA Phase 1/2 clinical trial (NCT05445323) and the Weill Cornell Medicine investigator-initiated Phase 1A trial (NCT05302271), LX2006 was well tolerated with no treatment-related serious adverse events, and clinically meaningful improvements in cardiac biomarkers were observed with increasing improvement over time.
Interim Safety Results
- LX2006 was well tolerated with no treatment-related serious adverse events to date in either study
- No signs of complement activation or other immunogenicity observed
- No cardiac or hepatic safety signals observed
- All adverse events were transient and resolved
- No participants discontinued from either study
Interim Clinical Results (from 8 participants with > 6-months of follow-up)
- Left ventricular mass index (LVMI): Of participants with elevated LVMI at baseline, 75% achieved >10% reduction at 12 months (n=4). Of all participants, 50% achieved >10% reduction in LVMI at 12 months (n=6).
- Among the participants with elevated LVMI at baseline, mean reduction in LVMI was 11.4% at 12 months (n=4) and 18.3% at 18 months (n=2).
- Left ventricular (LV) lateral wall thickness: wall thickening, an early indicator of left ventricular hypertrophy, was reduced by 13.6% on average in all participants at 12 months (n=6).
- High-sensitivity Troponin I (hsTnI): troponin I, a biomarker of myocardial injury, was reduced by 53.3% on average in all participants at 12 months (n=5).
- Frataxin protein expression evaluated via myocardial biopsy: observed increased frataxin levels compared to baseline following treatment with LX2006 in all participants evaluated to date utilizing two distinct measurements:
- LCMS: frataxin increase observed in 3 of 3 evaluable participants.
- IHC: frataxin increase observed in 2 of 2 evaluable participants.
Dosing Update and Next Steps
- As of July 15, 2024, 13 participants dosed to date across two trials:
- Cohort 1 (1.8x1011vg/kg): n=6
- Cohort 2 (5.6x1011 vg/kg): n=6
- Cohort 3 (1.2x1012 vg/kg): n=1
- SUNRISE-FA independent Data and Safety Monitoring Board endorsed proceeding to Cohort 3 dose level (1.2x1012vg/kg). This cohort has started enrollment with 1 participant dosed to date and will include at least 3 participants.
- The Weill Cornell investigator-initiated trial is currently enrolling in Cohort 2.
- Lexeo expects to share further details of these interim results, including an additional cardiac biopsy from Cohort 2, at a scientific conference in Fall 2024.
