Friedreich’s ataxia is caused by a genetic defect in a mitochondrial protein, frataxin, causing damage to cardiac muscle. Credit: evan_huang via Shutterstock.

Clinical-stage genetic medicine company Lexeo Therapeutics has won fast track designation from the US Food and Drug Administration (FDA) for its gene therapy candidate LX2006, designed to treat Friedreich’s ataxia (FA) cardiomyopathy. 

FA is a genetic disorder that affects the nervous system and often leads to difficulty with coordination and balance. A substantial proportion of patients with FA also develop cardiomyopathy or heart disease, which usually presents as left ventricular hypertrophy, a serious complication of the disorder. 

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LX2006 consists of an adeno-associated virus (AAV) vector carrying the frataxin gene. Mutations in the frataxin gene cause FA by reducing levels of the frataxin protein, disrupting energy production in cells, especially nerve and heart cells. 

In June 2023, Lexeo announced the completion of the first cohort and started dosing the second cohort in the Phase I/II SUNRISE-FA trial for LX2006. The therapy is to be given as a one-time intravenous (IV) infusion to patients in at least two ascending-dose cohorts with the potential for a third cohort. Long-term safety and efficacy will be observed for up to five years post-LX2006 treatment. In the first cohort, LX2006 had been well tolerated, with initial trial data anticipated later in 2024.  

There is currently no approved cure for FA. In March 2023, the FDA approved Skyclarys (omaveloxolone) developed by Reata Pharmaceuticals as the first treatment for FA. The therapy is now part of Biogen’s portfolio following the acquisition of Reata in September 2023. According to GlobalData’s Pharma Intelligence Center, Skyclarys is forecast to generate $1.66bn in 2030. 

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Apart from Skyclarys, treatments such as surgery and physical, occupational, and speech therapy, which are aimed at keeping the disease in check for as long as possible, are the standard in the FA space. 

In the announcement accompanying the fast track designation, Lexeo’s CEO Nolan Townsend said: “FA cardiomyopathy is the leading cause of death among FA patients, and there are currently no approved treatment options. The FDA’s Fast Track designation for LX2006 underscores the significant unmet need for effective treatment options to address the cardiac impact of this debilitating disease.” 

Another candidate in development for FA is Larimar Therapeutics’s nomlabofusp (CTI-1601), which was studied in a Phase II exploratory trial that had positive topline data released in February 2024. The company plans to file a biologics licence application with the FDA in H2 2025. 

Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.