Intellia Therapeutics Presents Positive Pooled Phase 1/2 Data of Lonvoguran Ziclumeran (lonvo-z) in Patients with Hereditary Angioedema

• Deep, stable and durable reductions in kallikrein observed 
• Among 32 patients who received a 50 mg dose of lonvo-z as of data cutoff:
  • 31 (97%) were attack-free and long-term prophylaxis (LTP)-free
  • 24 (75%) were attack-free and LTP-free for at least seven months (up to 32 months)
  • Among the 11 patients who originally received a 50 mg dose in Phase 2, 10 were attack-free and LTP-free
• Continue to observe a well-tolerated safety profile with up to three years of patient follow-up and no new long-term risks identified

CAMBRIDGE, Mass., Nov. 08, 2025 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ: NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today presented positive clinical data from a pooled analysis of all patients who received a 50 milligram (mg) dose of lonvo-z in the company’s ongoing Phase 1/2 clinical trial in patients with hereditary angioedema (HAE). These results were shared in an oral presentation today at the American College of Allergy, Asthma & Immunology (ACAAI) 2025 Annual Scientific Meeting in Orlando, Florida.

“Today’s data further support our belief that lonvo-z could completely redefine the HAE treatment landscape,” said Intellia President and Chief Executive Officer John Leonard, M.D. “With up to three years of follow-up, the vast majority of patients who received a one-time 50 mg dose of lonvo-z – including 10 of our original 11 patients who received this dose in Phase 2 – were both attack-free and LTP-free as of the data cutoff. We are looking forward to our approaching topline readout from our Phase 3 HAELO clinical trial by mid-2026.”

“Hereditary angioedema is a condition with significant burden that is marked by the unpredictable nature of when the next attack could occur, the painful and often prolonged swellings themselves as well as the need for lifelong treatment,” said Dr. Danny Cohn, M.D., Ph.D., Internist, Department of Vascular Medicine, Amsterdam University Medical Center. “The data presented today offer hope that lonvo-z could significantly reduce or remove those burdens for many patients via a one-time treatment. I am eagerly awaiting results from the ongoing HAELO Phase 3 clinical trial.”