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Intellia Therapeutics reported that its investigational therapy lonvoguran ziclumeran (lonvo-z) demonstrated a 98% mean reduction in monthly hereditary angioedema (HAE) attack rates over three years in a Phase 1/2 study involving 10 patients. All patients remained attack-free and off treatment for a median of 23 months after a single intravenous dose. The therapy showed a favorable safety profile, with only minor infusion-related reactions noted.
The Phase 3 HAELO trial concluded its screening ahead of schedule, with Intellia planning to submit a biologics license application in 2026 and targeting a U.S. market launch in 2027. The study data were presented at the European Academy of Allergy and Clinical Immunology Congress held from June 13-16, 2025, in Glasgow, United Kingdom.
Despite the promising results, concerns remain about the small sample size of 10 patients, which could affect the generalizability of the findings. The trial is no longer recruiting participants, potentially limiting future data collection.
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