Lexeo Therapeutics Receives FDA Breakthrough Therapy Designation for Friedreich Ataxia Gene Therapy

Lexeo Therapeutics has received FDA Breakthrough Therapy designation for its gene therapy, LX2006, to treat Friedreich's ataxia. The designation was based on interim clinical data showing clinically meaningful improvements in cardiac biomarkers and functional measures. The therapy has shown increased frataxin expression in cardiac biopsies, which is significant as cardiomyopathy is the leading cause of death in FA. A registrational study is expected to begin by early 2026.

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 the company's investigational gene therapy, LX2006, for the treatment of Friedreich's ataxia (FA). The designation was based on interim clinical data from two ongoing trials, the Lexeo-sponsored SUNRISE-FA Phase 1/2 trial and the Weill Cornell Medicine investigator-initiated Phase 1A trial [1].

The interim data showed that LX2006 was associated with clinically meaningful improvements in cardiac biomarkers and functional measures. Notably, an increase in frataxin expression was observed in cardiac biopsies three months after treatment, which is particularly significant as cardiomyopathy is the leading cause of death in FA, accounting for approximately two-thirds of patient deaths [1].

Lexeo expects to begin a registrational study by early 2026 and is currently enrolling patients in a natural history study, CLARITY-FA, to serve as an external control arm for the planned registrational study [1]. The company's Chief Executive Officer, R. Nolan Townsend, stated that the FDA Breakthrough Therapy designation underscores the potential of LX2006 and that Lexeo is moving quickly to initiate the registrational study in close partnership with patient advocates, clinicians, and the FA community [3].

The company's most advanced cardiovascular product candidate, LX2020 for the treatment of arrhythmogenic cardiomyopathy, also received IND clearance from the FDA and is being evaluated in an ongoing Phase I/II clinical trial [2]. Additionally, Lexeo has announced a strategic partnership with Perceptive Xontogeny Venture Funds and venBio Partners to advance non-viral, RNA-based therapeutics for genetic cardiac diseases [3].

Lexeo's financial results for the second quarter of 2025 show a robust cash position, with $152.5 million in cash, cash equivalents, and investments in marketable securities [3]. The company anticipates that this funding will support its operating and capital expenditures into 2028.

References:
[1] https://finance.yahoo.com/news/lexeo-therapeutics-friedreich-ataxia-gene-041352828.html
[2] https://www.marketscreener.com/news/chardan-cuts-price-target-on-lexeo-therapeutics-to-15-from-20-keeps-buy-rating-ce7c51dfd08df323
[3] https://ir.lexeotx.com/news-releases/news-release-details/lexeo-therapeutics-reports-second-quarter-2025-financial-results