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Lexeo Therapeutics Inc, a New York City-based clinical stage genetic medicine company dedicated to pioneering novel treatments for cardiovascular diseases, announced updates to key components of an accelerated approval pathway for LX2006 in Friedreich ataxia (FA) cardiomyopathy, alongside new interim clinical data from ongoing phase I/II studies.
“We are encouraged by our recent dialogue with the US FDA on LX2006, and we appreciate the Agency’s collaborative spirit as we work to deliver a potentially life-changing therapy to the FA community as efficiently as possible,” said R. Nolan Townsend, chief executive officer of Lexeo Therapeutics. “Given the highly compelling data to date that demonstrate clinically meaningful improvements across both cardiac and neurologic measures of FA, we are now pursuing a development strategy that could enable a smaller pivotal study, given the potential to pool data with the ongoing phase I/II trials, as well as potentially assessing the co-primary endpoint of LVMI earlier than 12 months. This approach could accelerate our overall timeline toward a BLA submission for LX2006 under the Accelerated Approval pathway.”
In response to questions from the company regarding the possibility of a faster path to a BLA, the US FDA has indicated openness to a BLA submission for accelerated approval that includes clinical data from the ongoing phase I/II studies of LX2006 pooled with new clinical data to be generated in the planned pivotal study. To enable pooling of these data to support licensure, Lexeo will submit enhanced manufacturing comparability data and meet an additional nonclinical requirement prior to the initiation of the planned pivotal study, given the company’s intention to leverage its optimized, high-yield Sf9-baculovirus manufacturing platform for future clinical and commercial drug supply, compared to the adherent HEK293 process used for phase I/II clinical supply. The FDA also previously agreed to evaluate the co-primary endpoint of LVMI at a time point earlier than 12 months. Lexeo continues to engage with the FDA on the pivotal protocol and comparability. In discussions to date, there have been no changes to the previously disclosed alignment with the FDA on key parameters related to the LX2006 planned registrational study.
Collectively, Lexeo believes this regulatory feedback has the potential to reduce the size and length of the planned pivotal study, possibly accelerating the overall timeline to BLA submission. Lexeo plans to initiate the LX2006 pivotal study as soon as possible in the first half of 2026, pending finalization of the trial protocol. FDA has previously granted Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug and Fast Track designations to LX2006, and admitted LX2006 into the CMC Development and Readiness Pilot (CDRP) programme.
LX2006 is an AAV-based gene therapy candidate for the treatment of FA cardiomyopathy, the leading cause of death in individuals with FA which affects approximately 5,000 people in the United States. LX2006 is designed to systemically deliver a functional frataxin gene to promote the expression of the frataxin protein and restore mitochondrial function in myocardial cells. LX2006 is being evaluated in two clinical trials, the Lexeo-sponsored SUNRISE-FA phase 1/2 clinical trial (NCT05445323) and the Weill Cornell Medicine investigator-initiated phase 1A trial (NCT05302271). LX2006 has been granted Breakthrough Therapy, Regenerative Medicine Advanced Therapy, Orphan Drug, Rare Pediatric Disease and Fast Track designations by the FDA, admitted into the US FDA CMC Development and Readiness Pilot (CDRP) programme, and granted orphan medicinal product designation by the European Commission.
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