Lexeo reports interim data for heart condition gene therapy

Lexeo Therapeutics Inc. (NASDAQ: LXEO) reported interim results from its Phase I/II trial of LX2020, a gene therapy for PKP2-associated arrhythmogenic cardiomyopathy, showing the treatment was generally well tolerated across ten participants.

The company said LX2020 demonstrated dose-dependent increases in PKP2 protein expression, with a mean increase of 93% in the low-dose cohort and 162% in the high-dose cohorts, based on data from participants with at least six months of follow-up as of January 7.

According to the company, arrhythmia burden stabilized or improved in the majority of participants. Non-sustained ventricular tachycardia showed a 22% mean improvement in high-dose cohorts, while premature ventricular contractions showed a 14% mean improvement in the same group.

The trial enrolled ten participants across three cohorts, with three receiving the low dose and seven receiving the high dose. Five participants in the high-dose group experienced liver function test elevations, which were managed with medication adjustments according to the trial protocol. All elevations resolved without complications.

One participant experienced a Grade 3 serious adverse event of sustained ventricular tachycardia three months after dosing, which the company assessed as possibly treatment-related but consistent with the natural course of the disease.

"These interim data from ten participants reinforce the favorable safety profile of LX2020 and demonstrate promising trends in transduction, protein expression, and reduction in arrhythmia burden at the high dose," said CEO R. Nolan Townsend.

LX2020 targets mutations in the PKP2 gene, which account for approximately 50% of arrhythmogenic cardiomyopathy cases and affect an estimated 60,000 people in the United States. The treatment has received Orphan Drug and Fast Track designations from the FDA.

The company completed enrollment for the HEROIC-PKP2 trial in the fourth quarter of 2025 and expects 12-month data for all high-dose participants in the fourth quarter of 2026.

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