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Lexeo, Weill Cornell Data Rights Pact to Speed Development of FA Cardiomyopathy Gene Therapy

NEW YORK – Lexeo Therapeutics has in-licensed rights to data from Cornell University that will allow it to expedite development of the investigational gene therapy LX2006 for treating Friedreich ataxia (FA) cardiomyopathy.

Under the license agreement, New York-based Lexeo has gained a package of intellectual property rights, including to current and future data generated within a Phase Ia trial of the gene therapy that investigators at Weill Cornell Medicine are conducting.

Researchers at the academic medical center published preclinical research on this gene therapy candidate and conducted a decadelong natural history study to better understand cardiomyopathy caused by FA, an inherited neuromuscular disorder. FA is caused by a genetic defect in the mitochondrial protein frataxin, and the condition negatively impacts skeletal and cardiac muscles. A large proportion of FA patients develop cardiomyopathy and around 60 percent die of cardiac causes. 

LX2006 uses an adeno-associated virus to intravenously deliver a functional frataxin gene to promote regular protein expression and restore mitochondrial function in myocardial cells. In preclinical studies, cardiac abnormalities resolved with LX2006, and the gene therapy appeared to have a favorable safety profile. The treatment has rare pediatric disease designation, fast-track status, and orphan drug designation from the US Food and Drug Administration.

The preclinical research conducted by Weill Cornell Medicine supported an investigational new drug application, which the FDA cleared, setting the stage for clinical trials.

Lexeo previously collaborated with Weill Cornell researchers to study LX2006 and is conducting its own open-label dose-ascending Phase I/II trial, dubbed SUNRISE-FA, in which four patients have received the gene therapy. Weill Cornell Medicine has dosed seven patients to date in its Phase IA trial and is collecting biomarker, structural, and functional cardiac data, similar to what Lexeo is collecting within SUNRISE-FA. Researchers in both trials will follow patients for five years after they receive LX2006, which is also known as AAVrh.10hFXN. 

The latest in-licensing agreement will allow Lexeo to combine the data from these two studies and double the number of evaluable patients, including some who have 18 months of data. The company expects an interim data readout from the combined cohort analysis in mid-2024. Lexeo will also conduct an analysis of natural history data and evaluate study participants' baseline characteristics to provide a more detailed picture of the type of cardiovascular disease experienced by FA patients.