Positive data from trial of potential HAE gene therapy presented at key European congress
All ten HAE patients who were trialling a potential gene therapy were attack-free and treatment-free after 23 months of follow-up, according to new data presented the recent 2025 European Academy of Allergy and Clinical Immunology (EAACI) Congress. Originally known as NTLA-2002, the potential therapy is now known as lonvoguran ziclumeran or lonvo-z.
The data, shared with HAEi by the pharmaceutical company Intellia Therapeutics, showed that:
- With up to three years of follow-up, a single dose of lonvo-z led to a 98% mean reduction in monthly HAE attack rate in all 10 patients
- All 10 patients were attack-free and treatment-free for a median of 23 months through the latest follow-up, demonstrating the potential of lonvo-z to become the first one-time therapy for most HAE patients
- Lonvo-z was well tolerated and continues to demonstrate a favorable safety profile
Intellia President and Chief Executive Officer John Leonard MD said: “Seeing all 10 patients in the Phase 1 portion of this study free from both HAE attacks and chronic therapy at nearly two years of median follow-up is incredibly encouraging. These data fuel our optimism for the outcomes of our ongoing Phase 3 HAELO study, which we expect to report in the first half of 2026.”
Dr Joshua Jacobs, Medical Director, Allergy and Asthma Clinical Research, Inc, said: “People living with HAE often report a reduced quality of life because they worry about the likelihood of their next attack, either because they still experience attacks or are reminded of it by their use of chronic therapy. Based on the data, it is reasonable to expect lonvo-z could offer patients the potential to be free from both physical HAE attacks and the burden of managing chronic HAE treatment.”
(Source: Intellia)