Lexeo Therapeutics Inc (NASDAQ:LXEO) saw its stock rise 2.3% after the U.S. Food and Drug Administration granted breakthrough therapy designation to its gene therapy candidate for Friedreich ataxia, LX2006.
This designation was awarded based on interim clinical results that demonstrated significant improvements in cardiac biomarkers and functional outcomes. LX2006 was also chosen for the FDA’s Chemistry, Manufacturing, and Controls Development and Readiness Pilot program, which aims to speed up patient access to promising treatments.
The breakthrough status is supported by data from 17 patients enrolled in two studies: the company-led SUNRISE-FA Phase 1/2 trial and a Phase 1A investigator-initiated trial at Weill Cornell Medicine. These studies showed meaningful cardiac and neurological improvements, alongside increased frataxin levels in all participants who underwent cardiac biopsies three months post-treatment.
Friedreich ataxia is a hereditary disorder that affects movement and the nervous system, with cardiomyopathy being the leading cause of mortality among patients.
“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 maintained a Buy rating with a $21.00 price target, commenting, “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 actively recruiting patients for the CLARITY-FA natural history study, designed to serve as an external control for a pivotal registrational trial expected to launch by early 2026.