Press Release: Lexeo Therapeutics Reports Fourth Quarter and Full Year 2025 Financial Results and Operational Highlights

SUNRISE-FA 2 open-label, pivotal trial protocol and SAP for LX2006 submitted to FDA in Q1 2026 following Type B meeting; study initiation on track for 1H 2026

First CMC Development and Readiness Program (CDRP) meeting for LX2006 in Q1 2026; aligned with FDA on flexible approach to process validation such as reduced PPQ manufacturing batches

Late-breaking oral presentations at the American College of Cardiology (ACC) Annual Meeting demonstrate continued, encouraging efficacy and safety data across LX2006 and LX2020 programs

Established research collaboration with Johnson & Johnson to explore targeted cardiac delivery of AAV gene therapy using novel, localized routes of administration

Strengthened leadership team with addition of new Chief Medical Officer and senior appointments bolstering cardiac expertise

Cash, cash equivalents and investments of $246.6 million expected to provide operational runway into 2028

NEW YORK, March 30, 2026 (GLOBE NEWSWIRE) -- Lexeo Therapeutics, Inc. (Nasdaq: LXEO), a clinical stage genetic medicine company dedicated to pioneering novel treatments for cardiovascular diseases, today provided business updates across its portfolio and reported financial results for the fourth quarter and full year 2025.

"Over the past year we have made meaningful progress across our cardiac pipeline, including significant advancements for both LX2006 and LX2020," said R. Nolan Townsend, Chief Executive Officer of Lexeo Therapeutics. "Most recently, we were pleased to be selected for two Late-Breaker presentations at the American College of Cardiology and appreciate the opportunity to engage the cardiology community around the future of precision medicine in cardiovascular care. Looking ahead, we remain focused on disciplined execution as we continue to advance our pipeline toward key milestones. With additional resources from our October financing, the expansion of our cardiac leadership team, and multiple announced preclinical research collaborations, we believe Lexeo is well positioned to drive significant long-term value for patients and shareholders."

Program Updates and Recent Progress

LX2006 in Friedreich Ataxia $(FA)$

   -- In October 2025, Lexeo announced progress in FDA discussions and reported 
      positive interim clinical data from both ongoing Phase I/II studies of 
      LX2006 demonstrating an encouraging safety profile and evidence of 
      meaningful cardiac and functional benefit. LX2006 was generally well 
      tolerated and associated with improvements in cardiac structure, 
      biomarkers, and functional outcomes, including reduced left ventricular 
      mass index (LVMI) and improved modified Friedreich Ataxia Rating Scale 
      (mFARS) scores across participants with more than six months of 
      follow--up. 
 
   -- In November 2025, FDA approved the analytical comparability report 
      establishing comparability between LX2006 HEK293 and Sf9 manufacturing 
      processes, endorsing use of the optimized, Sf9 final commercial 
      manufacturing process for LX2006 in the planned pivotal study and 
      clearing comparability requirements to begin dosing patients. 
 
   -- In February 2026, Lexeo submitted the final registrational trial design 
      and statistical analysis plan $(SAP)$ for the SUNRISE--FA 2 pivotal study 
      to the FDA following a Type B meeting. The proposed open-label study is 
      generally consistent with previously communicated guidance on key design 
      elements, such as study size, duration, patient population, and the LVMI 
      primary endpoint, and incorporates FDA feedback on minimizing bias, 
      including physician selection bias of study participants. Lexeo expects 
      to receive final feedback from the FDA in the second quarter of 2026 and 
      plans to provide a more detailed update at that time. 
 
   -- In March 2026, Lexeo conducted an initial Chemistry, Manufacturing and 
      Controls $(CMC)$ Development and Readiness Program (CDRP) meeting with the 
      FDA for LX2006, during which the agency reiterated its support for a 
      flexible validation approach including potential for reduced process 
      performance qualification (PPQ) runs and concurrent process validation. 
      Lexeo is proceeding with validation and commercial manufacturing 
      readiness activities to support the Biologic License Application (BLA) 
      submission for LX2006. 
 
   -- In March 2026, Lexeo shared updated interim clinical data for LX2006 at 
      the ACC Annual Meeting. 
 
          -- Phase I/II interim clinical data continue to show sustained or 
             deepening improvements across both cardiac and neurologic measures 
             of FA, including statistically significant improvement in mean 
             mFARS scores for LX2006-treated participants compared to a 
             propensity-matched control cohort from the UNIFAI natural history 
             study. 
 
          -- LX2006 remains generally well tolerated with no Grade 3+ SAEs to 
             date. 
 
   -- Anticipated milestones for the remainder of 2026 include: 
 
          -- FDA feedback on protocol submission expected in Q2 2026 
 
          -- Initiate SUNRISE-FA 2 pivotal trial in the first half of 2026 

LX2020 in PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)

   -- In January 2026, Lexeo reported positive interim clinical data from the 
      HEROIC PKP2 Phase I/II clinical trial evaluating LX2020. Clinical data 
      from the program were presented at the ACC Annual Meeting in March 2026. 
 
          -- Cardiac biopsy assessments demonstrated dose dependent increases 
             in PKP2 protein expression, vector copy number, and transgene 
             mRNA. 
 
          -- In high-dose cohorts, mean arrythmia burden was reduced at the 
             latest visit and the majority of participants reported symptom 
             improvement. 
 
          -- LX2020 remains generally well tolerated across ten participants 
             dosed with no clinically significant complement activation to 
             date. 
 
   -- Anticipated milestones for the remainder of 2026 include: 
 
          -- 12-month data update for all high dose participants in Q4 2026 
 
          -- Regulatory engagement with the FDA expected in 2026 

Corporate Updates

   -- Strengthened cardiovascular and commercial expertise through key 
      leadership appointments, including the appointment of Narinder Bhalla, MD, 
      as Chief Medical Officer. Dr. Bhalla brings deep experience building and 
      scaling global medical and clinical development organizations and leading 
      successful product launches, most recently at Bristol Myers Squibb and at 
      AstraZeneca. Additional senior leadership appointments further underscore 
      Lexeo's expanding capabilities in cardiovascular medicine and late-stage 
      clinical development. 
 
   -- Established research collaboration with Johnson & Johnson to explore 
      targeted cardiac delivery of AAV gene therapy. Collaboration combines 
      Lexeo's expertise in cardiac genetic medicine with Johnson & Johnson's 
      expertise in cardiovascular therapeutics and circulatory technologies, 
      including Impella$(TM)$ heart pumps, to enable the accelerated development 
      of a preclinical cardiac target using novel, localized routes of viral 
      gene therapy administration. 
 
   -- Executed $154 million equity financing in October 2025 to further advance 
      development of the company's cardiac pipeline and to support 
      registrational readiness activities for LX2006. 

Fourth Quarter and Full Year 2025 Financial Results

   -- Cash Position: As of December 31, 2025, cash, cash equivalents, and 
      investments in marketable securities were $246.6 million, which Lexeo 
      believes will be sufficient to fund operations into 2028. 
 
   -- Research & Development Expenses: Research and Development expenses were 
      $16.2 million for the three months ended December 31, 2025, compared to 
      $18.4 million for the three months ended December 31, 2024. Research and 
      Development expenses were $63.8 million for the year ended December 31, 
      2025, compared to $74.1 million for the year ended December 31, 2024. 
 
   -- General & Administrative Expenses: General and Administrative expenses 
      were $6.9 million for the three months ended December 31, 2025, compared 
      to $9.0 million for the three months ended December 31, 2024. General and 
      Administrative expenses were $45.5 million for the year ended December 
      31, 2025, compared to $31.7 million for the year ended December 31, 2024. 
 
   -- Net Loss: Net loss was $20.9 million or $0.27 per share (basic and 
      diluted) for the three months ended December 31, 2025, compared to $25.9 
      million or $0.78 per share (basic and diluted) for the three months ended 
      December 31, 2024. Net loss was $100.0 million or $1.86 per share (basic 
      and diluted) for the year ended December 31, 2025, compared to $98.3 
      million or $3.09 per share (basic and diluted) for the year ended 
      December 31, 2024. 

About Lexeo Therapeutics

Lexeo Therapeutics is a New York City-based, clinical stage genetic medicine company dedicated to reshaping heart health by applying pioneering science to fundamentally change how cardiovascular diseases are treated. The Company is advancing a portfolio of therapeutic candidates that take aim at the underlying genetic causes of conditions, including LX2006 in Friedreich ataxia (FA), LX2020 in plakophilin-2 (PKP2) arrhythmogenic cardiomyopathy, and others in devastating diseases with high unmet need.

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