Lexeo Therapeutics, Inc. Common Stock

Lexeo Therapeutics announced that the FDA granted Regenerative Medicine Advanced Therapy designation to LX2020 for PKP2-associated arrhythmogenic cardiomyopathy. The designation was based on interim data from the ongoing HEROIC-PKP2 Phase 1/2 trial, which showed a dose-dependent increase in PKP2 protein expression and improvement or stabilization in arrhythmia burden while remaining generally well tolerated. LX2020 is designed to deliver a functional PKP2 gene using an AAVrh10 vector to address the underlying genetic cause of the disease. The therapy has also received Orphan Drug and Fast Track designations, and the RMAT tag may enable accelerated approval and priority review. Lexeo shares were up 1.58% at $4.48.

JPMorgan lowered its price target on Lexeo Therapeutics to $9 from $12 while maintaining an Overweight rating, citing an updated valuation model. Lexeo recently finalized the SUNRISE-FA 2 pivotal trial protocol for its gene therapy candidate LX2006 targeting Friedreich's ataxia cardiomyopathy, with topline data expected in the second half of 2027 and a potential biologics license application submission in the first half of 2028. The FDA confirmed no additional nonclinical bridging studies are required, allowing the company to proceed with patient dosing using its optimized manufacturing process.

Lexeo Therapeutics announced that key results from Phase I/II studies of its gene therapy LX2006 in Friedreich ataxia have been published in JAMA Cardiology. The published findings combine data from two independent studies totaling 17 participants, showing LX2006 was generally well tolerated with early signs of efficacy. Among participants with abnormal baseline left ventricular mass index in the mid- and high-dose cohorts, there was a 28% mean improvement at 6 months and 33% at 12 months. Cardiac biopsy data from the SUNRISE-FA trial showed all eight participants achieved increases in frataxin protein expression at three months. The pivotal SUNRISE-FA 2 study is on track to initiate in the second quarter of 2026 with topline data expected in the second half of 2027.