(RTTNews) - Genetic medicine company Lexeo Therapeutics, Inc. (LXEO) announced Monday that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation and Orphan Drug designation to LX2020.
LX2020 is the company's AAVrh10-based gene therapy candidate designed to intravenously deliver a functional PKP2 gene to cardiac muscle for the treatment of arrhythmogenic cardiomyopathy (ACM) caused by mutations in the PKP2 gene (PKP2-ACM).
The planned Phase 1/2 trial, HEROIC-PKP2, is a first in human, 52-week open-label, dose-escalating, multicenter trial to determine the safety and tolerability of LX2020 in adult patients with PKP2-ACM.
Preliminary efficacy measures will evaluate myocardial protein expression, biomarkers measuring cardiac structure and function, and arrhythmia burden.
LX2020 will be administered as a one-time intravenous infusion to patients in two ascending-dose cohorts, evaluating the 2.0x1013 vg/kg and 6.0x1013 vg/kg dose levels with three patients in each cohort, and the potential for cohort expansion.
Long-term safety and efficacy will be evaluated for an additional four years following completion of the initial trial.
