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Also known as LX2006
Low dose 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 the cardiomyopathy associated with the condition. It is designed to address the cardiac complications of Friedreich ataxia and is currently in clinical development for this rare disease.
LX2006 is a gene therapy intended to deliver a functional gene to address the underlying genetic cause of Friedreich ataxia. The therapy is designed to target the cardiac manifestations of the disease, aiming to treat the cardiomyopathy that occurs secondary to Friedreich ataxia.
LX2006 is being developed by Lexeo Therapeutics, Inc., a biopharmaceutical company traded on the stock exchange under the ticker LXEO. The company is focused on developing gene therapies for rare diseases, with LX2006 being one of its lead candidates for Friedreich ataxia.
LX2006 is currently in Phase 2 clinical development for Friedreich ataxia cardiomyopathy. The Phase 2 study is actively recruiting participants, while a Phase 1 study has been completed. The drug has not yet been approved and remains investigational.
LX2006 is being studied in two clinical trials. The Phase 2 trial, NCT07721025, is recruiting patients with Friedreich ataxia and secondary cardiomyopathy. The Phase 1 trial, NCT05445323, has completed enrollment and is no longer recruiting. Both trials are being conducted in the United States.
Yes, low dose LX2006 is the same drug as LX2006. The term "low dose" refers to a specific dosage level of the gene therapy being evaluated in clinical trials. The drug is referred to as LX2006 in trial registrations and by the developer, Lexeo Therapeutics.