Lexeo Therapeutics, Inc., a company specializing in genetic medicine, has shared positive interim results for LX2006 in treating Friedreich ataxia cardiomyopathy. The findings from both the Lexeo SUNRISE-FA Phase 1/2 trial (NCT05445323) and the Weill Cornell Medicine Phase 1A trial (NCT05302271) indicate that LX2006 was well tolerated with no serious adverse events related to treatment. Significant improvements in cardiac biomarkers were observed over time.

Lexeo Therapeutics expressed optimism about LX2006's potential: "These data are encouraging for treating FA cardiomyopathy, a condition with no approved therapies. Based on the favorable safety profile and clinical benefits observed, we are eager to advance LX2006's clinical development, including potential accelerated approval."

Dr. Sandi See Tai, Chief Development Officer at Lexeo, highlighted the promising results: "The interim data demonstrate meaningful improvements in cardiac biomarkers, crucial for FA cardiomyopathy. Combined with increased frataxin protein expression in SUNRISE-FA cardiac biopsies, LX2006 shows promise in improving outcomes for FA cardiomyopathy patients. We appreciate the contributions of participants, caregivers, and investigators in achieving this milestone."

FA cardiomyopathy is a rare and progressive disorder caused by mutations in the frataxin gene, leading to left ventricular hypertrophy and eventual heart failure. Cardiac dysfunction is a major cause of mortality in up to 80% of FA patients. Participants in the SUNRISE-FA trial exhibited frataxin levels at 2% or less of normal at baseline. Lexeo's analysis also revealed elevated left ventricular mass index (LVMI) in adults with FA cardiomyopathy, which typically worsens with age and correlates with mortality in various cardiovascular conditions.

The SUNRISE-FA trial, sponsored by Lexeo, is a multicenter, open-label study evaluating LX2006's safety and efficacy in FA cardiomyopathy patients over 52 weeks across three dose levels. Weill Cornell Medicine is conducting the Phase 1A trial of AAVrh.10hFXN (LX2006), a single-site, open-label study assessing safety and efficacy in two dose cohorts of FA cardiomyopathy patients, each with five participants.

LX2006 is an AAV-based gene therapy administered intravenously for FA cardiomyopathy, the leading cause of mortality in FA patients affecting approximately 5,000 people in the United States. It aims to address FA's cardiac manifestations by delivering a functional frataxin gene to restore mitochondrial function in myocardial cells. Preclinical studies have shown LX2006's ability to reverse cardiac abnormalities, improve cardiac function, and enhance survival rates, all while maintaining a favorable safety profile. LX2006 has received Rare Pediatric Disease designation, Fast Track designation, and Orphan Drug designation from the FDA for treating FA cardiomyopathy.

 

Source: globenewswire.com

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