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Achieved mean reduction in left ventricular mass index (LVMI) of 11.4% at 12 months and 18.3% at 18 months in participants with elevated LVMI at baseline
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>10% reduction in LVMI at 12 months in 75% of participants with elevated LVMI at baseline
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Sustained and consistent improvements in other key measures of cardiac status, including left ventricular wall thickness and troponin I, in majority of participants at 12 months
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Increased post-treatment frataxin expression above baseline in all participants evaluated via myocardial biopsy to date
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LX2006 was well tolerated with no treatment-related serious adverse events to date; proceeding to Cohort 3 in SUNRISE-FA, with one participant dosed in this cohort to date
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Company to host webcast today at 8:00 AM ET
NEW YORK, July 15, 2024 (GLOBE NEWSWIRE) -- Lexeo Therapeutics, Inc. (Nasdaq: LXEO), a clinical stage genetic medicine company dedicated to pioneering treatments for genetically defined cardiovascular diseases and APOE4-associated Alzheimer’s disease, today 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.
“We are very encouraged by these data and the potential of LX2006 to treat FA cardiomyopathy, a devastating and fatal condition with no currently approved therapies,” said Dr. Eric Adler, Chief Medical Officer and Head of Research at Lexeo Therapeutics. “Based on the favorable safety profile and clinical benefits observed to date, we are excited to explore expedited clinical development of LX2006, including potential for accelerated approval of this possibly life-saving treatment.”
“The interim data shared today demonstrate clinically meaningful improvements across multiple cardiac biomarkers of hypertrophy, a hallmark of FA cardiomyopathy,” said Dr. Sandi See Tai, Chief Development Officer at Lexeo. “Together with the increases in frataxin protein expression observed in SUNRISE-FA cardiac biopsies to date, these results further highlight the potential of LX2006 to positively impact outcomes for people with FA cardiomyopathy. I would like to thank the participants, caregivers, and investigators participating in these trials who have helped to achieve this important milestone.”
FA cardiomyopathy is a devastating, rare, and progressive disorder caused by loss of function mutations in the frataxin gene. Thus far in participants in the SUNRISE-FA trial with cardiac biopsies, low levels of frataxin have been found in the heart at baseline, estimated to be 2% or less of normal. In terms of clinical presentation, FA cardiomyopathy is typically characterized by left ventricular hypertrophy ultimately progressing to heart failure, and cardiac dysfunction is the cause of death in up to 80% of individuals with FA. A new natural history subset analysis conducted by Lexeo showed elevated left ventricular mass index (LVMI) in adults with FA cardiomyopathy, and LVMI remained stable or increased with age without spontaneous improvement. Elevated LVMI is an indicator of left ventricular hypertrophy and correlated with mortality in multiple cardiovascular conditions including FA cardiomyopathy.
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
About the Clinical Studies
SUNRISE-FA is a Lexeo-sponsored, multicenter, 52-week, open-label trial evaluating the safety and preliminary efficacy of LX2006 in people who have FA cardiomyopathy, with three ascending-dose cohorts. Investigators at Weill Cornell Medicine are conducting a Phase 1A study of AAVrh.10hFXN, known as LX2006 at Lexeo, in a single-site, 52-week, open-label trial evaluating the safety and preliminary efficacy in people who have FA cardiomyopathy, in two ascending-dose cohorts with five participants per cohort.
About LX2006
LX2006 is an AAV-based gene therapy candidate delivered intravenously for the treatment of FA cardiomyopathy, the most common cause of mortality in individuals with FA affecting approximately 5,000 people in the United States. LX2006 is designed to target the cardiac manifestations of FA by delivering a functional frataxin gene to promote the expression of the frataxin protein and restore mitochondrial function in myocardial cells. In preclinical studies, LX2006 reversed the cardiac abnormalities in FA disease models and showed improvement in cardiac function and survival while demonstrating a favorable safety profile. The FDA has granted Rare Pediatric Disease designation, Fast Track designation, and Orphan Drug designation to LX2006 for the treatment of FA cardiomyopathy.
About Lexeo Therapeutics
Lexeo Therapeutics is a New York City-based, clinical stage genetic medicine company dedicated to transforming healthcare by applying pioneering science to fundamentally change how genetically defined cardiovascular diseases and APOE4-associated Alzheimer’s disease are treated. Using a stepwise development approach, Lexeo is leveraging early proof-of-concept functional and biomarker data to advance a pipeline of cardiovascular and APOE4-associated Alzheimer’s disease programs.