Intellia Reports New Lonvo-z Data: 81% Lower Attack Rate Versus Prior Prophylaxis in HAE, All 50 mg Patients Off Long-Term Prophylaxis
Intellia Therapeutics, Inc. (Nasdaq: NTLA), a gene-editing company based in Cambridge, Massachusetts, on November 12 announced new data for lonvo-z (lonvoguran ziclumeran), its one-time CRISPR gene-editing candidate for hereditary angioedema (HAE), drawn from seven presentations at the American College of Allergy, Asthma & Immunology (ACAAI) 2026 Annual Scientific Meeting in Phoenix, Arizona.
In the Phase 3 HAELO trial, among 35 patients who were taking long-term prophylaxis (LTP) at study entry and were randomized to lonvo-z, the mean HAE attack rate fell by 81% during the efficacy observation period compared with the rate on prior LTP. Following treatment, 63% of these patients achieved complete control, more than tripling the attack-free rate seen on prior prophylaxis.
In the Phase 1/2 study, patients who received a one-time 50 mg dose saw deep, consistent and durable reductions in plasma kallikrein, with a mean reduction of 84% at four weeks maintained through up to four years of follow-up. The proportion of patients attack-free after one year was at least 90% in each month, and all patients receiving 50 mg were LTP-free as of the data cutoff.
Across an integrated analysis of 93 patients who received a single 50 mg dose in the Phase 1/2 and Phase 3 HAELO studies, the overall attack rate fell by 89% from baseline through up to four years of follow-up, and 97% of patients never initiated LTP after treatment. The most common treatment-emergent adverse events were infusion-related reactions, headache, fatigue and nasopharyngitis, and no new long-term risks were identified.
In patient-reported outcomes, interviews with 23 Phase 1/2 patients at a median follow-up of 2.4 years found all of them described their HAE as well controlled after lonvo-z, compared with 48% before treatment, and 22 of the 23 (96%) described lonvo-z as an optimal therapy. In the HAELO trial, lonvo-z produced a 23.5-point mean reduction in Angioedema Quality of Life total score by week 28, versus 6.5 points for placebo, against a 6-point threshold for a clinically important difference.
Lonvo-z is designed to permanently lower kallikrein by inactivating the KLKB1 gene with a single outpatient dose, and could become the first one-time treatment for HAE. It holds Orphan Drug and Regenerative Medicine Advanced Therapy designations from the U.S. Food and Drug Administration (FDA). In September 2026, the FDA accepted the Biologics License Application for lonvo-z and granted Priority Review, with a PDUFA target action date of March 10, 2027. HAE is a rare genetic disease estimated to affect one in 50,000 people, with attacks that can be painful, debilitating and life-threatening. (Source: Intellia Therapeutics)
Source: Intellia Therapeutics
Published by GSM · USA (original Chinese edition)
Photo: 图：CRISPR 基因编辑示意图。美国国家人类基因组研究所供图，Wikimedia Commons，公共领域。 / Image: Illustration of CRISPR gene editing. By the National Human Genome Research Institute, via Wikimedia Commons, public domain.
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