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

Lexeo Therapeutics Inc. (NASDAQ:LXEO) is one of the best IPO stocks to buy according to Wall Street analysts. Earlier on July 7, the US FDA granted Breakthrough Therapy designation to Lexeo Therapeutics for its investigational drug, LX2006, which is a gene therapy for Friedreich 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. To date, 17 participants have been treated across both studies. The interim data showed that LX2006 was associated with clinically meaningful improvements in cardiac biomarkers and cardiac and neurologic functional measures.

A close-up of a hand holding a vial of biopharmaceutical drugs ready to be administered.

In all participants with available cardiac biopsies, an increase in frataxin expression was observed three months after treatment. This is particularly significant as cardiomyopathy is the leading cause of death in FA, with cardiac dysfunction accounting for the cause of death in approximately two-thirds of patients. The company 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.

Lexeo Therapeutics Inc. (NASDAQ:LXEO) is a clinical-stage genetic medicine company that focuses on hereditary and acquired diseases with high unmet need in the US.

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Disclosure: None. This article is originally published at Insider Monkey.