Intellia presents new interim data from first-in-human study of NTLA-2002 in HAE

Intellia Therapeutics announced updated data from an ongoing Phase 1/2 clinical study of NTLA-2002 for the treatment of hereditary angioedema, or HAE. The interim analyses were shared in a Distinguished Industry Abstract oral presentation at the American College of Allergy, Asthma & Immunology 2022 Annual Scientific Meeting. The data presented are from 10 adult patients with HAE in the Phase 1, dose-escalation portion of the study, with a data cut-off date of September 28, 2022. Single doses of 25 mg, 50 mg and 75 mg of NTLA-2002 were administered via intravenous infusion, and changes from baseline values of plasma kallikrein protein were measured for each patient. Administration of NTLA-2002 led to deep, dose-dependent reductions in plasma kallikrein, based on complete cohort biomarker data availability. For the 25 mg and 75 mg cohorts, these deep reductions in plasma kallikrein were sustained through the observation period, which ranged from week 16 to week 32. HAE attack rates are measured in the dose-escalation portion of the study, with the first analysis occurring at the end of the pre-specified 16-week primary observation period. To date, all patients in the 25 mg and 75 mg dose cohorts have reached the end of this initial observation period in ongoing follow-up as described below. Patients in the 50 mg cohort have not completed the primary 16-week observation period. At all three dose levels, NTLA-2002 was generally well-tolerated, and the majority of adverse events were mild in severity. The most frequent adverse events were infusion-related reactions, which were mostly Grade 1 and resolved within one day. There have been no dose-limiting toxicities, no serious adverse events and no adverse events of Grade 3 or higher observed to date. No clinically significant laboratory abnormalities were observed. Intellia expects to select up to two doses to further evaluate NTLA-2002 in the Phase 2, placebo-controlled, dose-expansion portion of the study, which is expected to begin in the first half of 2023. Intellia anticipates expanding country and site participation, including U.S. clinical sites, as part of the Phase 2 study.