FDA Grants Breakthrough Therapy Status to Lexeo's LX2006 for Friedreich Ataxia

Lexeo Therapeutics, Inc., a clinical-stage genetic medicine enterprise, has announced that the U.S. Food and Drug Administration (FDA) awarded Breakthrough Therapy designation to its investigational treatment LX2006. The designation is based on promising clinical data from Phase I/II trials that illustrate significant enhancements in cardiac biomarkers and functional measures for Friedreich ataxia (FA). Additionally, LX2006 has been chosen for inclusion in the FDA Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) program. This initiative is designed to advance the readiness of treatments for registration and facilitate quicker patient access.

The company’s Chief Development Officer, Dr. Sandi See Tai, emphasized the importance of receiving the Breakthrough Therapy status, citing its potential influence on both cardiac health and neurological functionality. Dr. Tai expressed optimism for LX2006, particularly given the current lack of therapies for FA cardiomyopathy, a leading cause of mortality among FA patients. She anticipates ongoing collaboration with the FDA to bring this therapy to patients efficiently.

The FDA’s decision relied on interim data showing LX2006 significantly improved cardiac health markers and functional abilities related to FA, coupled with observed increases in frataxin expression among participants who underwent cardiac biopsies three months after treatment. So far, 17 individuals have been treated in trials: the Lexeo-sponsored SUNRISE-FA Phase 1/2 study and an investigator-led Phase 1A trial at Weill Cornell Medicine. Lexeo is also enrolling participants for a natural history study named CLARITY-FA, which will act as an external control for forthcoming registrational studies. The company plans to commence these registrational studies by early 2026 and is collaborating with the FDA to finalize the statistical analysis plan.

The Breakthrough Therapy designation aims to speed up the development and assessment of treatments targeting serious or life-threatening conditions, particularly where preliminary evidence suggests substantial improvements over existing therapies. LX2006’s designation adds to its previous accolades, including Regenerative Medicine Advanced Therapy, Orphan Drug, and Fast Track designations granted by the FDA. The CDRP program is an FDA initiative to hasten the Chemistry, Manufacturing, and Controls development process, fostering more direct communication between the FDA and sponsors to quicken patient access to promising therapies in areas with significant unmet needs.

Lexeo Therapeutics, headquartered in New York City, focuses on transforming heart health by leveraging advanced scientific methods to address cardiovascular diseases. The company is developing a range of therapeutic candidates targeting genetic causes of health conditions, including LX2006 for Friedreich ataxia and LX2020 for plakophilin-2 arrhythmogenic cardiomyopathy, among others addressing severe diseases with high unmet needs.