Intellia Therapeutics Completed Global Phase 3 HAELO Trial Enrollment For Lonvoguran Ziclumeran (lonvo-z) To Treat Hereditary Angioedema

Intellia Therapeutics, Inc., a clinical-stage gene editing company developing CRISPR-based therapies, announced that it has completed enrollment in the global Phase 3 HAELO study of lonvoguran ziclumeran (lonvo-z) for the treatment of hereditary angioedema. The company expects to report topline data in the first half of 2026 and remains on track to submit a biologics license application in the second half of 2026, with plans for a U.S. launch in the first half of 2027.

John Leonard, M.D., president and chief executive officer of Intellia, said that completing enrollment within nine months of dosing the first patient represents an important milestone for the company and highlights the significant unmet need among hereditary angioedema patients. He expressed gratitude to the patients, their families, and the investigators involved in the trial and noted that the company is eager to share topline results next year as it advances toward its planned submission and potential launch.

Aleena Banerji, M.D., professor at Harvard Medical School, clinical director of the Allergy and Clinical Immunology Unit at Massachusetts General Hospital, and principal investigator for HAELO, commented that lonvo-z has shown strong potential in earlier studies to improve treatment for hereditary angioedema and said she looks forward to the Phase 3 data. The Phase 3 HAELO study began dosing in January 2025 and has now completed enrollment, with nearly half of the participants from the United States. 

It is a randomized, double-blind, placebo-controlled trial designed to evaluate the safety and efficacy of lonvo-z in at least 60 adults and adolescents aged 16 years or older with Type I or Type II hereditary angioedema. Participants are randomized in a 2:1 ratio to receive a single 50 mg infusion of lonvo-z or placebo and have the option for blinded crossover at week 28. The primary measures include the number of hereditary angioedema attacks and the number of patients who remain attack-free from week 5 through week 28.