Lexeo Therapeutics Secures FDA Breakthrough Therapy Designation for LX2006, Shares Rise 8% in Pre-market Trade.

Lexeo Therapeutics (LXEO) has been granted FDA Breakthrough Therapy designation for LX2006, a treatment for Friedreich ataxia. The designation was based on interim clinical data showing significant improvements in cardiac biomarkers and functional measures. LX2006 has also been selected for the FDA's Chemistry, Manufacturing, and Controls Development and Readiness Pilot program. Shares of Lexeo are up 8% in pre-market trade on Monday.

Title: Lexeo Therapeutics Secures FDA Breakthrough Therapy Designation for LX2006

Lexeo Therapeutics, Inc. (Nasdaq: LXEO) has received a significant boost from the U.S. Food and Drug Administration (FDA), which granted Breakthrough Therapy designation to LX2006, a treatment for Friedreich ataxia (FA). The designation, based on interim clinical data from Phase I/II trials, highlights substantial improvements in cardiac biomarkers and functional measures. Additionally, LX2006 has been selected for the FDA's Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) program, aimed at expediting patient access to promising therapies.

The FDA's decision was influenced by interim clinical data showing clinically significant improvements in cardiac biomarkers and functional measures of both cardiac and neurologic aspects of FA. Notably, increased expression of frataxin was observed in all participants with cardiac biopsies at three months post-treatment. To date, 17 participants have been treated across two trials: the Lexeo-sponsored SUNRISE-FA Phase 1/2 clinical trial (NCT05445323) and the Weill Cornell Medicine investigator-initiated Phase 1A trial (NCT05302271). Lexeo is currently enrolling a prospective natural history study, CLARITY-FA, which will serve as a concurrent external control arm for the registrational study. The company expects to initiate the registrational study by early 2026 and is actively working with the FDA to finalize the statistical analysis plan (SAP).

Breakthrough Therapy designation is designed to expedite the development and review of investigational therapies for serious or life-threatening diseases where preliminary evidence suggests substantial improvement over existing treatments. This designation is in addition to the Regenerative Medicine Advanced Therapy (RMAT) designation, Orphan Drug designation, and Fast Track designation previously granted to LX2006 by the FDA. The CDRP program aims to facilitate the expedited CMC development of investigational therapies, enhancing communication between the FDA and sponsors to support faster patient access.

Lexeo Therapeutics, a New York City-based clinical stage genetic medicine company, is dedicated to pioneering novel treatments for cardiovascular diseases. The company's portfolio includes LX2006 for Friedreich ataxia, LX2020 for plakophilin-2 (PKP2) arrhythmogenic cardiomyopathy, and other treatments for high unmet need diseases. Shares of Lexeo are up 8% in pre-market trade on Monday, reflecting investor optimism about the potential of LX2006 and the company's progress in advancing its pipeline.

References:
[1] https://www.morningstar.com/news/globe-newswire/9489843/lexeo-therapeutics-announces-fda-breakthrough-therapy-designation-for-lx2006-in-friedreich-ataxia
[2] https://finance.yahoo.com/news/lexeo-therapeutics-announces-fda-breakthrough-110000430.html