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LX2020 · 1 trial · 3 indications
Evaluation of Safety and Tolerability of LX2020
| Arm | Type | Description |
|---|---|---|
| LX2020 | EXPERIMENTAL | Single ascending dose of LX2020, with a starting dose of 2.0 x10\^13 gc/kg, in multiple cohorts |
| Name | Type | Description |
|---|---|---|
| LX2020 | GENETIC | LX2020 is an adeno-associated viral vector encoding the human Plakophilin-2 (PKP2) gene (AAVrh.10hPKP2) |
Selected Inclusion Criteria: * Adults with a clinical diagnosis of ACM meeting the 2010 revised Task Force Criteria (TFC) * Genetic testing documenting a pathogenic or likely pathogenic variant in PKP2 * Frequent premature ventricular complexes (PVCs) * Implantable cardioverter-defibrillator (ICD) ...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Lexeo Therapeutics, Inc. | LXEO | 3 | PHASE1 | LX2020 |
LX2020 is an investigational gene therapy being developed for arrhythmogenic cardiomyopathy, a rare heart condition. It is designed to address the underlying genetic cause of the disease in patients with a PKP2 pathogenic variant. The therapy is currently in Phase 1 clinical development and has not yet been approved by regulatory authorities.
LX2020 targets the PKP2 gene, which encodes plakophilin-2, a protein essential for heart muscle cell adhesion. Mutations in PKP2 are a leading cause of arrhythmogenic cardiomyopathy. By delivering a functional copy of the gene, the therapy aims to restore normal protein function and potentially halt disease progression.
LX2020 is being developed by Lexeo Therapeutics, Inc., a biopharmaceutical company traded on the Nasdaq under the ticker symbol LXEO. The company specializes in genetic medicines for rare diseases, with a focus on cardiovascular and central nervous system conditions. Lexeo is conducting the ongoing Phase 1 clinical trial for LX2020.
LX2020 is currently in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by the FDA or any other regulatory agency. The therapy has received Fast Track, Orphan Drug, and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA, which are intended to expedite its development and review.
LX2020 is being evaluated in a single Phase 1 clinical trial with the identifier NCT06109181. The study, titled 'Gene Therapy for ACM Due to a PKP2 Pathogenic Variant,' is active but not recruiting participants. It is enrolling approximately 10 adults aged 18 years and older in the United States with arrhythmogenic cardiomyopathy caused by a PKP2 mutation.
LX2020 is a gene therapy specifically designed for patients with arrhythmogenic cardiomyopathy caused by a PKP2 pathogenic variant, a condition also referred to as PKP2-ACM or PKP2-ARVC. While the drug is not formally named after the condition, it is intended to treat this specific genetic form of the disease.