Progress of clinical trials of NTLA-2002 outlined during Intellia Therapeutics’ second quarter 2023 financial results presentation

Intellia announced that due to the substantial interest from physicians and patients to participate in the NTLA-2002 clinical program, all patients have been identified for the global Phase 2 portion of the study, with enrollment expected to be completed in the second half of 2023.

Based on the strong momentum of the program, the company plans to complete enrollment at ex-U.S. sites in the ongoing Phase 2 study. Further, following the March 2023 IND clearance, the FDA requested supplemental preclinical data related to the inclusion of female patients of child-bearing potential. Intellia expects to submit these data in advance of the planned Phase 3 trial, which will complement the clinical data collected from female patients of child-bearing potential dosed in the ongoing Phase 1/2 study.

Intellia plans to initiate the global pivotal Phase 3 study, including U.S. patients, as early as the third quarter of 2024, subject to regulatory feedback.

In June, Intellia announced additional positive interim results from the Phase 1 portion of the ongoing Phase 1/2 study of NTLA-2002. The company report that:

Across all 10 patients, a 95% mean reduction in monthly attack rate was observed after a single dose of NTLA-2002 through the latest follow-up.
The median duration of follow-up was 9.0 months (range of 5.6 – 14.1 months).
At all three dose levels evaluated, NTLA-2002 has been well tolerated, and the majority of adverse events were mild in severity.
These interim data were presented at the European Academy of Allergy and Clinical Immunology (EAACI) Hybrid Congress 2023, held in Hamburg, Germany, and virtually.

Intellia President and Chief Executive Officer John Leonard said: “For NTLA-2002, we are thrilled to see the excitement for this investigational therapy from the HAE community. In just a handful of months, we were able to identify all patients required to fully enroll in the ongoing Phase 2 study and now expect to initiate the pivotal Phase 3 program next year.”

NTLA-2002 is designed to knock out the KLKB1 gene in the liver, with the potential to permanently reduce total plasma kallikrein protein and activity, a key mediator of HAE. This investigational approach aims to prevent attacks for people living with HAE by providing continuous reduction of plasma kallikrein activity following a single dose. NTLA-2002 is being evaluated in a Phase 1/2 study in adults with Type I or Type II HAE.

(Source: Intellia Therapeutics)