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Lexeo Therapeutics to Dose First Human in Arrhythmogenic Cardiomyopathy Gene Therapy Trial

NEW YORK – Lexeo Therapeutics plans to dose the first patient in a first-in-human trial of its gene therapy candidate LX2020 for arrhythmogenic cardiomyopathy in the first half of 2024 with a readout of interim data from the first cohort of patients expected in the second half of the year, Lexeo CEO R. Nolan Townsend said Wednesday morning at the JP Morgan Healthcare Conference.

The US Food and Drug Administration last year cleared the company's investigational new drug (IND) application, granting New York-based Lexeo permission to begin a Phase I/II trial, called HEROIC-PKP2.

LX2020 is an adeno-associated virus rh10 vector-based gene therapy designed to treat patients with mutations in the PKP2 gene that cause the genetic cardiac disease. The gene therapy aims to increase PKP2 protein levels by delivering a functional copy of PKP2 to cardiac muscle administered through an intravenous infusion.

"We're moving this program into clinical studies shortly," Townsend said. Investigators will evaluate arrhythmias, as well as myocardial protein expression and other biomarkers of cardiac structure and function.

In mid-2024, Lexeo expects to have an interim data readout from a Phase I/II trial of another cardiovascular gene therapy, LX2006, being tested as a treatment for Friedreich's ataxia cardiomyopathy, an inherited neuromuscular disorder caused by deficiency in the frataxin protein. LX2006 delivers a functional FXN gene to promote frataxin expression.

Lexeo is monitoring multiple biomarkers from cardiac biopsies and imaging "that we think will support a potential pathway toward an accelerated approval for this medicine," Townsend said of LX2006. He said that left ventricular mass index, levels of troponin I, and protein expression are among endpoints the company is considering for late-stage clinical trials.

By the end of 2024, Lexeo expects to have a data readout from a Phase I/II trial of a gene therapy candidate for Alzheimer's disease patients with two copies of the APOE4 allele, which increases the likelihood of developing the neurodegenerative disorder. LX1001 is designed to express the APOE2 allele, which is thought to have a protective effect. Lexeo also said it plans to initiate pre-IND meetings with the FDA this year for another Alzheimer's candidate, LX1021, which delivers a specific mutation-modified APOE2 allele for patients in that same patient population.

"You can think of this as us correcting the genetics of the disease, rather than a specific pathogenic mechanism of the disease," Townsend said.

As of the end of September, the company had $136.4 million in cash and marketable securities, with a projected cash runway into Q4 2025.