LX2020 - (HEROIC-PKP2)

August 5, 2026: Lexeo Therapeutics (NASDAQ: LXEO) said the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to LX2020, an investigational AAV-based gene therapy for plakophilin-2-associated arrhythmogenic cardiomyopathy (PKP2-ACM). The designation was based on recent interim clinical data from the ongoing HEROIC-PKP2 Phase I/II trial (NCT06109181).

LX2020 now has FDA RMAT, Orphan Drug, and Fast Track designations. The company said RMAT status provides enhanced FDA interaction on clinical development, manufacturing, and potential regulatory pathways, and may enable eligibility for accelerated approval, priority review, and rolling review.

LX2020 is designed to systemically deliver a functional full-length PKP2 gene using an AAVrh10 capsid to cardiomyocytes to restore the desmosomal complex and cell-to-cell adhesion. The study is described as single-arm, open-label, and multi-center.

The company said there are no approved disease-modifying treatments for PKP2-ACM and stated it expects to share additional clinical and regulatory updates before the end of 2026.