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Also known as Low dose LX2006, Mid Dose LX2006
LX2006 · 2 trials · 2 indications
| Arm | Type | Description |
|---|---|---|
| LX2006 | EXPERIMENTAL | Cohort 1: Participants ≥16 years of age with FA-CM Cohort 2: Participants ≥6 to \<16 years of age with FA-CM. As Cohort 2 will not be randomized, all participants will receive LX2006 upon enrollment. Participants in Cohort 2 will be enrolled after safety is assessed in a group of participants in Cohort 1. |
| Usual Care | OTHER | Cohort 1: Participants ≥16 years of age with FA-CM Participants will receive usual care for 26 weeks before receiving treatment with LX2006 (single crossover). |
| Cohort 1/ Cohort 2/ Cohort 3 | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| LX2006 | GENETIC | Adeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN) |
| Usual Care | OTHER | Cohort 1: Participants ≥16 years of age with FA-CM Participants will receive usual care for 26 weeks before receiving treatment with LX2006 (single crossover). |
| Low dose LX2006 | GENETIC | Adeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN) |
| Mid Dose LX2006 | GENETIC | Adeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN) |
| High Dose LX2006 | GENETIC | Adeno-associated viral vector encoding the FXN gene (AAVrh.10hFXN) |
Inclusion Criteria: * Male or female, age at least 6 years at the time of signing the informed consent (and assent, if applicable). * Diagnosis of FA, based on clinical phenotype and genotype (GAA expansion on the frataxin gene) * Onset of FA on or before 25 years of age * Confirmed left ventricula...
Low dose LX2006 is an investigational gene therapy being developed for Friedreich Ataxia, specifically for cardiomyopathy associated with the condition. It is designed to address the cardiac complications of Friedreich Ataxia and is currently in clinical development, with a Phase 2 trial recruiting patients.
LX2006 targets the FXN gene, which encodes the frataxin protein. In Friedreich Ataxia, mutations in FXN lead to frataxin deficiency, and LX2006 is designed to deliver a functional copy of the gene to address the underlying cause of the disease, particularly its cardiac manifestations.
LX2006 is developed by Lexeo Therapeutics, Inc., a biopharmaceutical company traded on Nasdaq under the ticker LXEO. The company is advancing LX2006 as a gene therapy for Friedreich Ataxia cardiomyopathy.
LX2006 is in Phase 2 clinical development for Friedreich Ataxia cardiomyopathy. A Phase 2 trial (NCT07721025) is currently recruiting, and a Phase 1 trial (NCT05445323) is active but not recruiting. The therapy has received FDA designations including Breakthrough Therapy, RMAT, Orphan Drug, Fast Track, Rare Pediatric Disease, and Accelerated Approval.
LX2006 is being studied in two clinical trials. NCT07721025 is a Phase 2 study in Friedreich Ataxia cardiomyopathy, recruiting 26 participants aged 6 years and older in the United States. NCT05445323 is a Phase 1 study in the same condition, with 8 participants aged 18 and older, and is active but not recruiting.
Yes, low dose LX2006 is the same drug as LX2006. The term low dose refers to a specific dosage level being evaluated in clinical trials, but the drug asset itself is LX2006, developed by Lexeo Therapeutics for Friedreich Ataxia cardiomyopathy.