Lexeo Therapeutics Reports Positive Interim Phase 1/2 Data for LX2006 in Friedreich Ataxia Cardiomyopathy

Lexeo Therapeutics, Inc. has announced promising interim results for LX2006, a treatment for Friedreich ataxia (FA) cardiomyopathy. This comes from both the Lexeo SUNRISE-FA Phase 1/2 clinical trial and the Weill Cornell Medicine Phase 1A trial. Notably, LX2006 has been well tolerated without any serious treatment-related adverse events, and it has shown significant improvements in cardiac biomarkers over time.

Dr. Eric Adler, Chief Medical Officer at Lexeo Therapeutics, expressed optimism about the efficacy of LX2006 in treating FA cardiomyopathy, a severe and currently untreatable condition. The favorable safety profile and clinical benefits observed thus far have spurred the company to consider expedited clinical development and potential accelerated approval of the treatment.

Dr. Sandi See Tai, Chief Development Officer at Lexeo, highlighted the interim results, which show substantial improvements in multiple cardiac biomarkers of hypertrophy—a key feature of FA cardiomyopathy. Additionally, increases in frataxin protein expression were observed in cardiac biopsies from the SUNRISE-FA trial. These results underscore the potential of LX2006 to improve outcomes for individuals suffering from this debilitating condition. Dr. See Tai also extended gratitude to the participants, caregivers, and investigators involved in these trials.

FA cardiomyopathy is a rare and progressive disorder caused by mutations in the frataxin gene. In the SUNRISE-FA trial, participants exhibited critically low levels of frataxin in their heart tissue at baseline. Clinically, FA cardiomyopathy is often marked by left ventricular hypertrophy, which can progress to heart failure. Cardiac dysfunction is a leading cause of mortality, accounting for up to 80% of deaths in individuals with FA. A recent analysis by Lexeo indicated that elevated left ventricular mass index (LVMI) in adults with FA cardiomyopathy remains stable or increases with age, with no natural improvement.

The interim safety results from these trials are promising. LX2006 has been well tolerated, with no serious treatment-related adverse events reported. This has allowed the progression to Cohort 3 in the SUNRISE-FA trial, with one participant already dosed in this cohort.

Clinical results from eight participants with more than six months of follow-up have shown significant improvements. Specifically, there was a mean reduction in LVMI of 11.4% at 12 months and 18.3% at 18 months among participants with elevated LVMI at baseline. Additionally, 75% of these participants experienced a reduction in LVMI of more than 10% at 12 months. Consistent improvements were also noted in other key cardiac measures like left ventricular wall thickness and troponin I levels. Furthermore, myocardial biopsies showed increased frataxin expression above baseline in all evaluated participants.

The SUNRISE-FA trial, sponsored by Lexeo, is a multicenter, 52-week, open-label study assessing the safety and preliminary efficacy of LX2006 in FA cardiomyopathy patients across three ascending-dose cohorts. Concurrently, Weill Cornell Medicine is conducting a 52-week, single-site, open-label Phase 1A study evaluating the same treatment in two ascending-dose cohorts, each with five participants.

LX2006 is an AAV-based gene therapy candidate designed to treat FA cardiomyopathy by delivering a functional frataxin gene to heart cells, thus promoting frataxin protein expression and restoring mitochondrial function. Preclinical studies have shown that LX2006 can reverse cardiac abnormalities in FA models, improve cardiac function and survival, and demonstrate a favorable safety profile. The FDA has recognized LX2006 with Rare Pediatric Disease designation, Fast Track designation, and Orphan Drug designation for the treatment of FA cardiomyopathy.

Lexeo Therapeutics, headquartered in New York City, is a clinical stage genetic medicine company aiming to revolutionize treatment for genetically defined cardiovascular diseases and APOE4-associated Alzheimer’s disease through innovative scientific approaches.

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