Gene Therapy Candidate Fast Tracked for Friedreich Ataxia Cardiomyopathy

The Food and Drug Administration (FDA) has granted Fast Track designation to LX2006 for the treatment of Friedreich ataxia cardiomyopathy.

LX2006 (AAVrh.10hFXN) is an adeno-associated virus gene therapy candidate designed to deliver a functional frataxin gene to cardiac cells to improve mitochondrial function. The designation was supported by preclinical data that showed LX2006 reversed cardiac abnormalities in disease models and improved cardiac function and survival.

The Company is currently evaluating LX2006 in patients with Friedreich ataxia cardiomyopathy in the 52-week, open-label, phase 1/2 SUNRISE-FA trial (ClinicalTrials.gov Identifier: NCT05445323). The dose escalation study is assessing the safety and tolerability of 3 different doses of LX2006. Cardiac function and other preliminary efficacy endpoints will also be investigated.

“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,” said R. Nolan Townsend, CEO of Lexeo Therapeutics. “We believe today’s Fast Track designation, along with the previously announced Rare Pediatric Disease and Orphan Drug designations granted to LX2006, will allow for enhanced regulatory interactions and the potential for this life-improving therapy to reach FA patients more quickly.”