LX2020 / Lexeo Therap - LARVOL DELTA

LX2020-01: Gene Therapy for ACM Due to a PKP2 Pathogenic Variant (clinicaltrials.gov) - P1/2 | N=10 | Active, not recruiting | Sponsor: Lexeo Therapeutics | Recruiting ➔ Active, not recruiting | Trial primary completion date: Feb 2026 ➔ Dec 2026

Enrollment closed • Trial primary completion date • Cardiomyopathy • Cardiovascular • Gene Therapies

GRIT-PKP2: Long-term Follow-up Study of Gene Therapy for Arrhythmogenic Cardiomyopathy Due to a Plakophilin-2 Pathogenic Variant (clinicaltrials.gov) - P=N/A | N=10 | Enrolling by invitation | Sponsor: Lexeo Therapeutics | Not yet recruiting ➔ Enrolling by invitation

Enrollment open • Cardiomyopathy • Cardiovascular • Gene Therapies

GRIT-PKP2: Long-term Follow-up Study of Gene Therapy for Arrhythmogenic Cardiomyopathy Due to a Plakophilin-2 Pathogenic Variant (clinicaltrials.gov) - P=N/A | N=10 | Not yet recruiting | Sponsor: Lexeo Therapeutics

New trial • Cardiomyopathy • Cardiovascular • Gene Therapies

An AAV GMP Manufacturing Solution for Large Clinical Demand Indications (ASGCT 2025) - "A 200L GMP batch can yield approximately thirty (30) 6E13 vg/kg doses for Lexeo's LX2020 PKP2 program...The universal problem of high COGS and low yield has not allowed gene therapy to have the deep impact on healthcare that it has the potential to do. Lexeo's manufacturing process and overall CMC strategy can dramatically expand patient access and transform the gene therapy field."

Clinical • Gene Therapies

Preclinical efficacy and safety of AAVrh10-based plakophilin-2 gene therapy (LX2020) as a treatment for arrhythmogenic cardiomyopathy. (PubMed, NPJ Regen Med) - "Safety assessments in non-human primates revealed no adverse events. These data support LX2020 as a viable treatment for PKP2 ACM patients."

Journal • Preclinical • Cardiomyopathy • Cardiovascular • Gene Therapies • Heart Failure

Study design and rationale of heroic-pkp2: a phase 1/2 study of the safety and efficacy of lx2020 gene therapy in patients with arrhythmogenic cardiomyopathy due to a plakophilin-2 pathogenic variant (CVCT USA 2024) - No abstract available

Clinical • Gene therapy • P1/2 data • Cardiomyopathy • Cardiovascular • Gene Therapies

A Phase 1/2 Study Of The Safety And Efficacy Of LX2020 Gene Therapy In Patients With Arrhythmogenic Cardiomyopathy Due To A Plakophilin-2 Pathogenic Variant (HFSA 2024) - "HEROIC-PKP2 will evaluate the safety and preliminary efficacy of gene therapy for PKP2-ACM. Successful outcomes from this trial will lead to the development of gene therapies targeting the primary cause of PKP2-ACM. This will lead the way to treat other genetic causes of ACM and open new avenues for precision medicine in the management of cardiovascular diseases."

Clinical • Gene therapy • P1/2 data • Cardiomyopathy • Cardiovascular • Gene Therapies

Lexeo Therapeutics Reports Second Quarter 2024 Financial Results and Operational Highlights (GlobeNewswire) - "Expected Upcoming Milestones: LX2006 for the treatment of Friedreich ataxia cardiomyopathy: Previously disclosed data, and one additional cardiac biopsy from Cohort 2, will be shared at a scientific conference in Fall 2024; Update on ongoing regulatory engagements expected by end of 2024; LX2020 for the treatment of PKP2-ACM: Interim data readout (Cohort 1) in 2H 2024; LX1001 for the treatment of APOE4-associated Alzheimer’s disease: Interim Phase 1/2 data readout (all cohorts) in 2H 2024; LX2021 for the treatment of DSP cardiomyopathy: Initiate IND-enabling studies in 2024."

P1/2 data • Preclinical • Regulatory • Alzheimer's Disease • Cardiomyopathy • CNS Disorders • Friedreich ataxia

Gene Therapy for ACM Due to a PKP2 Pathogenic Variant (clinicaltrials.gov) - P1/2 | N=10 | Recruiting | Sponsor: Lexeo Therapeutics | Not yet recruiting ➔ Recruiting

Enrollment open • Gene therapy • Cardiomyopathy • Cardiovascular • Gene Therapies

Gene Therapy for ACM Due to a PKP2 Pathogenic Variant (clinicaltrials.gov) - P1/2 | N=10 | Not yet recruiting | Sponsor: Lexeo Therapeutics

Gene therapy • New P1/2 trial • Cardiomyopathy • Cardiovascular • Gene Therapies

Study Design And Rationale Of HEROIC-PKP2,A PHAse 1/2 Study Of The Safety And EFficacy Of Lx2020 Gene TheRApy In Patients With ArrhythmOGenIC CArdiomyopathy Due To APLaKOPHilin-2 Pathogenic Variant (HFSA 2023) - "HEROIC-PKP2 will evaluate the safety and preliminary efficacy of gene therapy for PKP2-ACM. Successful outcomes from This trial may pave the way for the development of gene therapies targeting other genetic causes of ACM and open new avenues for precision medicine in the management of cardiovascular diseases."

Clinical • Gene therapy • P1/2 data • Atrial Fibrillation • Cardiomyopathy • Cardiovascular • Gene Therapies

LX2020-An AAV Based Gene Therapy Improves the Arrhythmogenic Right Ventricular Cardiomyopathy Phenotype in a Severe Mouse Model Harboring Human PKP2 Mutation (ASGCT 2023) - "Collectively, these data indicate that LX2020 overcomes cardiac PKP2 deficits in ARVC settings by driving human PKP2 expression and that this is sufficient to prevent and stabilize the progression of a rapid ARVC disease in young and adult PKP2 Hom mice, respectively. LX2020 thus represents a potentially effective clinically relevant gene therapy approach to treat patients with ARVC disease."

Gene therapy • Preclinical • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Fibrosis • Gene Therapies • Heart Failure • Immunology