RARE Daily

Lexeo Enters Data Collaboration with Cornell to Accelerate Development of Gene Therapy for FA Cardiomyopathy

April 22, 2024

Rare Daily Staff

Lexeo Therapeutics entered an in-license agreement with Cornell University to expedite development of the investigational gene therapy candidate LX2006 for the treatment of Friedreich ataxia cardiomyopathy.

LX2006 is an AAV-based gene therapy candidate delivered intravenously for the treatment of FA cardiomyopathy, the most common cause of mortality in patients with Friedreich’s ataxia (FA) affecting approximately 5,000 patients in the United States. LX2006 is designed to target the cardiac manifestations of FA by delivering a functional frataxin gene to promote the expression of the frataxin protein and restore mitochondrial function in myocardial cells. In preclinical studies, LX2006 reversed the cardiac abnormalities in FA disease models and showed improvement in cardiac function and survival while demonstrating a favorable safety profile.

Under the license agreement, Lexeo has acquired certain rights including rights to current and future data generated in an ongoing investigator-initiated phase 1A trial of AAVrh.10hFXN to treat FA cardiomyopathy. 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, in which four patients have been dosed to date across cohorts 1 and 2. Weill Cornell Medicine has dosed seven patients to date with LX2006 across dose cohorts 1 and 2 and is collecting biomarker, structural, and functional cardiac data akin to SUNRISE-FA.

“The larger aggregate data set, combined with Orphan Drug, Rare Pediatric Disease, and Fast Track designations from FDA, is anticipated to facilitate an accelerated path to regulatory engagements for LX2006,” said R. Nolan Townsend, CEO of Lexeo Therapeutics. “We are excited about the opportunity to advance research in FA cardiomyopathy, which is the leading cause of death in FA and has no approved treatment options today.”

The interim clinical data readout of LX2006 is expected mid-year 2024. With the newly-licensed data, the readout will now include participants across the two studies, approximately doubling the number of evaluable patients and including patients with a treatment duration out to 18-months. Lexeo also expects to provide an analysis of natural history data and baseline characteristics for study participants from both studies to characterize the cardiovascular disease phenotype seen in FA cardiomyopathy ahead of the interim readout.

“This agreement with Lexeo Therapeutics builds upon years of collaboration between Weill Cornell Medicine and Lexeo to benefit patients with FA cardiomyopathy,” said Lisa Placanica, senior managing director, Center for Technology Licensing at Weill Cornell Medicine. “It is our intention that this license agreement will accelerate the clinical investigation and development of LX2006 as a potential life-saving therapy for patients with FA.”

Photo: R. Nolan Townsend, CEO of Lexeo Therapeutics

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