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Investing.com -- Lexeo Therapeutics Inc (NASDAQ:LXEO) stock rose 2.3% after the company announced that the U.S. Food and Drug Administration granted breakthrough therapy designation for its Friedreich ataxia treatment.
The FDA designated LX2006, Lexeo’s gene therapy for Friedreich ataxia, as a breakthrough therapy based on interim clinical data showing improvements in cardiac biomarkers and functional measures. Additionally, the FDA selected LX2006 for its Chemistry, Manufacturing, and Controls Development and Readiness Pilot program, which aims to accelerate patient access to therapies.
The breakthrough designation was supported by data from 17 participants across two clinical trials: the company-sponsored SUNRISE-FA Phase 1/2 trial and a Weill Cornell Medicine investigator-initiated Phase 1A trial. Results demonstrated clinically significant improvements in cardiac biomarkers and cardiac and neurologic functional measures, with increased frataxin expression observed in all participants with cardiac biopsies three months after treatment.
Friedreich ataxia is a genetic disorder affecting the nervous system and causing movement problems. The condition typically leads to cardiomyopathy, which is the primary cause of death in patients with the disease.
"We are highly encouraged by the impact of LX2006 on key measures of cardiac health, especially given the lack of treatments for FA cardiomyopathy today, which is the leading cause of death in FA," said Dr. Sandi See Tai, Chief Development Officer of Lexeo Therapeutics.
Stifel analyst Paul Matteis reiterated a Buy rating and $21.00 price target on Lexeo Therapeutics. The analyst commented, "We view receipt of BTD here after the FDA has reviewed clinical data to be a bullish sign for their regulatory prospects--of note this also occurred for SLNO in PWS--and continuing to show that for rare/GTx, FDA may continue to remain flexible/accommodating."
Lexeo is currently enrolling patients in a prospective natural history study called CLARITY-FA, which will serve as a concurrent external control arm for the registrational study expected to begin by early 2026.
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