• Data from HAELO Phase 3 clinical trial presented today in a late-breaking oral session at European Academy of Allergy & Clinical Immunology Annual Congress 2026
  
  
• HAELO manuscript simultaneously published in the New England Journal of Medicine
  
  

Intellia Therapeutics, Inc. (Nasdaq: NTLA), a leading biopharmaceutical company focused on revolutionizing medicine leveraging CRISPR gene editing and other core technologies, today presented additional positive results from the global Phase 3 HAELO clinical trial of lonvo-z (formerly NTLA-2002) for hereditary angioedema (HAE) in a late-breaking oral presentation at the European Academy of Allergy & Clinical Immunology (EAACI) Annual Congress 2026 in Istanbul, Türkiye. Results from the trial were simultaneously published in the New England Journal of Medicine. The presentation and publication can be accessed from the Scientific Publications and Presentations section of intelliatx.com.

As previously announced, HAELO met its primary endpoint with an 87% reduction (p<0.0001) in mean monthly attacks in the lonvo-z arm vs. the placebo arm during the efficacy evaluation period (weeks 5 to 28). In addition, 62% of patients in the lonvo-z arm were entirely attack free and therapy free for the six-month efficacy evaluation period, compared with 11% of patients in the placebo arm (p<0.0001), a key secondary endpoint. Today, Intellia reported data for the trial’s other key secondary endpoints:

^{AE-QoL: Angioedema Quality of Life score, which is a validated, angioedema-specific patient-reported outcome measure with a lower score indicating improved quality of life. A 6-point reduction is considered to be a clinically important improvement in AE-QoL.
CI: Confidence interval}

Favorable safety and tolerability data were observed for lonvo-z. The most common treatment emergent adverse events (TEAEs) during the primary observation period (infusion through week 28) that were higher in the lonvo-z group compared to placebo were infusion-related reaction, headache, fatigue, back pain, and upper respiratory tract infection. All reported TEAEs were mild or moderate and there were no serious adverse events observed in the lonvo-z arm.

“These are the first Phase 3 results to deliver on the much-heralded promise of in vivo CRISPR gene editing,” said John Leonard, M.D., Intellia President and Chief Executive Officer. “Regardless of age or prior use of long-term prophylaxis therapies, it was observed that a single lonvo-z treatment significantly reduced HAE attacks for all patients during the efficacy evaluation period, with all patients remaining LTP free as of the data cutoff. We thank the many patients, physicians and caregivers who participated in HAELO and are excited to be advancing this highly differentiated candidate toward a potential approval.”

Danny Cohn, M.D., Ph.D., Internist, Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam University Medical Center, and a HAELO principal investigator, added, “As a clinician who has witnessed patients struggle with the unpredictability and emotional toll of HAE, the prospect of offering lasting freedom from attacks and chronic medication with a one-time treatment is incredibly exciting. These results give me confidence that many patients will soon have the potential to enjoy a normal life.”

Today’s presentation and publication also included supplemental demographics, data and analyses, including: 

• A time plot showing that the mean monthly attack rate for patients receiving lonvo-z through the data cutoff (February 10, 2026) was well below the reported rate in prescreening while patients were receiving standard-of-care therapy;
  
  
• Patient-level data demonstrating that all patients in the lonvo-z arm experienced attack-rate reductions from baseline during weeks 5 to 28;
  
  
• An analysis showing that meaningful attack-rate reductions were observed for all evaluated subgroups;
  
  
• A breakdown showing that 20% of the patients who enrolled in HAELO reported having complete disease control (no attacks) as their best response to prior long-term prophylaxis therapies; and
  
  
• A plasma kallikrein time plot showing that protein levels decreased substantially by the first measurement (day 15), reached a steady state by week 5 and remained stable through the data cutoff.
  
  

A rolling biologics license application (BLA) submission for lonvo-z was initiated in April with the U.S. Food and Drug Administration (FDA). The company continues to anticipate regulatory approval and a U.S. launch in the first half of 2027.