Updates provided on lonzo-z (also known as NTLA-2002), a potential HAE medicine currently being trialed in patients

A series of updates on lonzo-z, which is currently undergoing testing amongst HAE patients, were provided by the pharmaceutical company Intellia. The key updates shared were:

• Recruitment ended earlier than expected during the second quarter, and the Company now expects to complete randomization in the global Phase 3 HAELO study during the third quarter of 2025.
• Intellia presented three-year follow-up data from the Phase 1 portion of the ongoing Phase 1/2 study after receiving a single dose of lonvo-z. Results were shared in an oral presentation at the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2025 on June 15 in Glasgow, United Kingdom. At the time of the 12 February 2025 data cutoff, all 10 patients were attack-free and treatment-free for a median of nearly two years. With up to three years of follow-up, a single dose of lonvo-z led to a mean reduction in monthly HAE attack rate of 98% over the study period, compared to the pre-treatment baseline. Across all three dose levels, lonvo-z was generally well tolerated and showed a safety profile consistent with earlier data presented.
• Intellia expects to present additional data from the ongoing Phase 1/2 study in the second half of 2025.
• The Company is on track to submit a Biologics License Application (BLA) in the second half of 2026

John Leonard MD, Intellia President and Chief Executive Officer, said: “We are exceeding many of our internal expectations. The enthusiasm from both patients and physicians for Intellia’s late-stage programs has resulted in strong enrollment numbers that allow us to plan to accelerate completion of the Phase 3 HAELO study in HAE ahead of our original plans. We are full steam ahead in achieving our mission of getting one-time therapies to more patients.”

(Source: Intellia)