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Intellia Therapeutics Announces Positive Interim Clinical Data for its Second Systemically Delivered Investigational CRISPR Candidate, NTLA-2002 for the Treatment of Hereditary Angioedema (HAE)
Positive interim clinical data further validate the modularity of Intellia’s industry-leading genome editing platform and its potential to target a multitude
About this update from Intellia Therapeutics, Inc.
[{"type":"text","content":"Positive interim clinical data further validate the modularity of Intellia’s industry-leading genome editing platform and its potential to target a multitude of genetic diseases A single dose of NTLA-2002 led to a 65% and 92% mean plasma kallikrein reduction at 25 mg and 75 mg doses, respectively, at week eightHAE attacks were reduced by 91% in the 25 mg dose cohort through week 16; two of three patients remain attack free since treatment with third patient attack free since week 10 through latest follow-up NTLA-2002 was generally well-tolerated at both dose levelsIntellia plans to initiate the Phase 2 dose-expansion portion of the study in 1H 2023Intellia to host investor event today, Friday, September 16, at 8:00 a.m. ET CAMBRIDGE, Mass., Sept. 16, 2022 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing potentially curative therapeutics leveraging CRISPR-based technologies, today announced positive interim results from an ongoing Phase 1/2 clinical study of NTLA-2002, its second in vivo genome editing candidate. NTLA-2002 is a systemically administered CRISPR candidate being developed for hereditary angioedema (HAE) and is designed to knock out the KLKB1 gene in liver cells, thereby reducing the production of kallikrein protein. Uncontrolled activity of kallikrein is responsible for the overproduction of bradykinin, which leads to the recurring, debilitating and potentially fatal swelling attacks that occur in people living with HAE. The interim data were shared today in an oral presentation at the 2022 Bradykinin Symposium held in Berlin, Germany. The data presented are from the initial six adult patients with HAE in the ongoing dose-escalation study with a data cut-off date of July 27, 2022. Single doses of 25 mg (n=3) and 75 mg (n=3) 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 dose-dependent reductions in plasma kallikrein and achieved maximal reductions by week eight, with mean reductions of 65% and 92% in the 25 mg and 75 mg dose cohorts, respectively. Furthermore, these reductions were sustained through at least 16 weeks in the 25 mg cohort and eight weeks in the 75 mg cohort for which complete cohort...