Intellia Therapeutics, Inc. has recently announced positive three-year data from the phase 1 trial of Lonvoguran Ziclumeran (Lonvo-Z) at the European Academy of Allergy and Clinical Immunology Congress. The results indicate a promising outcome for patients with hereditary angioedema (HAE).
The data revealed that a single dose of Lonvo-Z led to a remarkable 98% mean reduction in the monthly HAE attack rate across all 10 patients with up to three years of follow-up. Impressively, all 10 patients remained attack-free and treatment-free for a median of 23 months, demonstrating the potential of Lonvo-Z to become the first one-time therapy for most HAE patients.
In terms of safety, Lonvo-Z was well tolerated, with no treatment-related adverse events observed during the period following 28 days after dosing. The most frequent adverse events were infusion-related reactions, which were mostly grade 1 and resolved with all patients receiving the full dose.
Looking ahead, Intellia's global phase 3 HAELO trial of Lonvo-Z has successfully completed screening ahead of schedule, with over half of the patients being screened in the United States. The study is no longer recruiting, and the company plans to provide an update on enrollment in the future.
Intellia also intends to present new and longer-term data from the phase 2 portion of the ongoing phase 1/2 study in the second half of 2025. The company aims to submit a biologics license application (BLA) in 2026 to support its plans for a U.S. launch in 2027.
These findings underscore the potential of Intellia's approach to gene editing therapy, offering a one-time treatment that is well tolerated and provides a highly differentiated, durable effect for patients with HAE. These results are expected to have a significant impact on the future of HAE treatment and the biotechnology industry as a whole. As a result of these announcements, the company's shares have moved -0.48% on the market, and are now trading at a price of $8.31. If you want to know more, read the company's complete 8-K report here.