Lexeo Therapeutics Announces License Agreement to Accelerate Development of LX2006 for the Treatment of Friedreich Ataxia Cardiomyopathy

Lexeo Therapeutics gains intellectual property rights including current and future clinical data from ongoing Weill Cornell Medicine investigator-initiated trial of gene therapy candidate AAVrh.10hFXN (LX2006) to support regulatory discussions

Together with Lexeo Therapeutics’ ongoing study of LX2006, a total of 11 participants have been treated to date; includes patients at treatment durations out to 18-months with no treatment-related serious adverse events observed across either study

Interim readout of combined data set at multiple doses expected mid-year 2024

NEW YORK, April 22, 2024 (GLOBE NEWSWIRE) -- Lexeo Therapeutics, Inc. (Nasdaq: LXEO), a clinical stage genetic medicine company, today announced an in-license agreement with Cornell University to expedite development of the investigational gene therapy candidate LX2006 for the treatment of Friedreich ataxia (FA) cardiomyopathy.

Under the license agreement, Lexeo has acquired certain rights¹ including rights to current and future data generated in an ongoing investigator-initiated Phase 1A trial of AAVrh.10hFXN to treat FA cardiomyopathy (NCT05302271). The agreement will support Lexeo’s efforts to develop a potentially life-changing therapy for this unmet need.

The investigator-initiated trial is being conducted by Weill Cornell Medicine, which has pioneered groundbreaking research on the potential of gene therapy in FA, published preclinical data that supported the first ever gene therapy IND clearance for FA, and sponsored a natural history study for almost a decade to better characterize the condition and its progression. Lexeo previously licensed know-how relating to AAVrh.10hFXN from Weill Cornell Medicine and collaborated with researchers there to further study the candidate, which Lexeo refers to as LX2006. Lexeo is studying LX2006 in the company-sponsored, open label, dose-ascending, multicenter SUNRISE-FA Phase 1/2 trial (NCT05445323), in which four patients have been dosed to date across cohorts 1 & 2. Weill Cornell Medicine has dosed seven patients to date with LX2006 across dose cohorts 1 & 2 and is collecting biomarker, structural, and functional cardiac data akin to SUNRISE-FA.