LX2020’s preclinical studies in a relevant murine model of PKP2-ACM demonstrated promising outcomes, with restored desmosomal integrity, reduced arrhythmias, and extended survival. The therapy delivers a functional PKP2 gene intravenously to the cardiac muscle, elevating PKP2 protein levels in the critical cardiac desmosome—a structure vital for cell adhesion.
The upcoming Phase 1/2 trial, HEROIC-PKP2, will be a first-in-human, 52-week open-label, dose-escalating, multicenter study evaluating LX2020’s safety and tolerability in adult PKP2-ACM patients. Additionally, efficacy measures will assess myocardial protein expression, cardiac structure and function biomarkers, and arrhythmia burden. LX2020 will be administered as a one-time intravenous infusion in two ascending-dose cohorts, with potential expansion, followed by long-term safety and efficacy monitoring for four years.
PKP2-ACM is characterized by myocardial cell loss, replaced by fibrotic tissue and fatty deposits, primarily caused by PKP2 gene variants, leading to heart dysfunction, rhythm abnormalities, and sudden death. Current treatments only manage symptoms and lack targeted disease intervention.
The clearance of LX2020’s IND represents a significant step toward offering a potential one-time treatment for PKP2-ACM patients, who currently lack effective therapeutic options.
LEXEO Therapeutics aims to develop effective treatments for genetically defined cardiovascular diseases and a subgroup of genetically defined Alzheimer’s disease through a diverse pipeline of AAV-based gene therapies.
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