NEW YORK – Gene therapy developer Lexeo Therapeutics on Tuesday said the US Food and Drug Administration has cleared its investigational new drug application for LX2020.
New York-based Lexeo's LX2020 is an adeno-associated virus rh10 vector-based gene therapy candidate to treat patients with mutations in the PKP2 gene that cause arrhythmogenic cardiomyopathy, a genetic cardiac disease. LX2020 is designed to intravenously deliver a functional copy of PKP2 to cardiac muscle, with a goal of increasing PKP2 protein levels.
The company's planned Phase I/II trial, dubbed HEROIC-PKP2, is a first-in-human, open-label, dose-escalating trial to assess the safety and tolerability of LX2020 in adult patients. Investigators will evaluate myocardial protein expression, biomarkers measuring cardiac structure and function, and arrhythmia burden for participants in the trial.
The initial trial will run for 52 weeks, with patients evaluated for an additional four years after completing the study.
"This IND clearance marks an important step in advancing a potential one-time treatment for individuals with PKP2-ACM," Lexeo CSO Eric Adler said in a statement. "Current clinical management strategies are only marginally effective and primarily focus on symptom management."
The LX2020 development program is based on research led by Farah Sheikh at the University of California, San Diego School of Medicine.