Lexeo Therapeutics Announces Promising Interim Results in Friedreich Ataxia Cardiomyopathy Trials

Encouraging Progress

Dr. Eric Adler, Chief Medical Officer and Head of Research at Lexeo Therapeutics, expressed optimism regarding the interim data. “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,” he said. The favorable safety profile and clinical benefits observed so far have prompted the company to explore expedited clinical development, including potential accelerated approval for LX2006.

Dr. Sandi See Tai, Chief Development Officer at Lexeo, added, “The interim data shared today demonstrate clinically meaningful improvements across multiple cardiac biomarkers of hypertrophy, a hallmark of FA cardiomyopathy.” Increased frataxin protein expression in cardiac biopsies further underscores the therapeutic potential of LX2006. Dr. See Tai also expressed gratitude to trial participants, caregivers, and investigators for their contributions to achieving this milestone.

About Friedreich Ataxia Cardiomyopathy

FA cardiomyopathy is a rare, progressive disorder caused by loss-of-function mutations in the frataxin gene, characterized by left ventricular hypertrophy and eventual heart failure. In the SUNRISE-FA trial, participants exhibited significantly low frataxin levels in the heart at baseline. Lexeo’s new natural history subset analysis revealed elevated left ventricular mass index (LVMI) in adults with FA cardiomyopathy, correlating with increased mortality risk.

Interim Safety and Clinical Results

The interim safety results indicated that LX2006 was well-tolerated, with no signs of complement activation, immunogenicity, or cardiac/hepatic safety signals. All adverse events were transient and resolved, with no participants discontinuing the studies.

Key clinical outcomes included:

• Left ventricular mass index (LVMI): 75% of participants with elevated LVMI at baseline saw >10% reduction at 12 months. Overall, 50% of participants achieved this reduction.
• Left ventricular (LV) lateral wall thickness: Average reduction of 13.6% at 12 months.
• High-sensitivity Troponin I (hsTnI): 53.3% average reduction at 12 months.
• Frataxin protein expression: Increases observed in all evaluated participants.

Dosing and Future Plans

As of July 15, 2024, 13 participants have been dosed across three cohorts, with the highest dose level cohort recently beginning enrollment. Lexeo plans to share further details, including additional cardiac biopsy results, at a scientific conference in Fall 2024.

About LX2006

LX2006, an AAV-based gene therapy, targets the cardiac manifestations of FA by delivering a functional frataxin gene to restore mitochondrial function in myocardial cells. Preclinical studies have shown significant improvements in cardiac function and survival.

About Lexeo Therapeutics

Lexeo Therapeutics, based in New York City, is focused on pioneering treatments for genetically defined cardiovascular diseases and APOE4-associated Alzheimer’s disease. The company leverages early proof-of-concept functional and biomarker data to advance its therapeutic pipeline.

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