Intellia Therapeutics, Inc. (NTLA), announced a new three-year follow-up data from its ongoing Phase 1/2 study of lonvoguran ziclumeran or lonvo-z, formerly NTLA-2002 for the treatment of hereditary angioedema or HAE.
Results were presented at the 2025 European Academy of Allergy and Clinical Immunology or EAACI Congress.
A single dose of lonvo-z led to a 98% average reduction in monthly HAE attack rates across all 10 patients in the Phase 1 study, with a median of 23 months attack- and treatment-free.
The therapy, designed using CRISPR/Cas9 technology, showed durable, dose-dependent reductions in plasma kallikrein, a key protein involved in HAE symptoms.
Lonvo-z was well tolerated at all three tested doses (25 mg, 50 mg, 75 mg), with the most common side effects being mild, transient infusion-related reactions.
No serious treatment-emergent or treatment-related adverse events were observed, including in long-term follow-up.
Based on these encouraging results, Intellia confirmed that its global Phase 3 HAELO trial has completed patient screening ahead of schedule, with over half of participants screened from U.S. sites.
An update on enrollment will be provided later.
Top-line data from the HAELO trial are expected in the first half of 2026, and a Biologics License Application or BLA submission is planned for 2026 with a potential U.S. launch in 2027.
Lonvo-z is being developed as a one-time intravenous gene editing treatment targeting the KLKB1 gene to prevent HAE attacks by permanently reducing kallikrein activity.
It has received multiple global regulatory designations, including FDA Orphan Drug and RMAT, EMA PRIME, and UK MHRA Innovation Passport.
Currently, NTLA, is trading at $8.78, up by 5.48 percent on the Nasdaq.
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