Intellia Therapeutics Presents New Interim Data from First-in-Human…

Intellia Therapeutics Presents New Interim Data from First-in-Human…

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  • A marked reduction in plasma kallikrein levels and HAE onset rates was observed at all doses tested
  • All patients treated in the 25 mg and 75 mg cohorts have sustained seizure-free interval at latest
    follow up
  • The first three patients treated have a sustained seizure-free interval of 5.5 10.6 months after a single dose of NTLA-2002
  • NTLA-2002 was generally well tolerated at all doses
  • Intellia is hosting an investor event to discuss updated data from the Phase 1/2 study of NTLA-2002, its second systemically administered study in vivo CRISPR Candidate, Monday, November 14 at 8:00 p.m. ET

CAMBRIDGE, Mass., Nov. 12, 2022 (GLOBE NEWSWIRE) — Intellia Therapeutics, Inc. NTLA, a leading clinical-stage genome-editing company focused on developing potentially curative therapeutics utilizing CRISPR-based technologies, today announced updated data from an ongoing Phase 1/2 clinical trial of NTLA-2002 in the treatment of hereditary angioedema (HAE) known. The interim analyzes were presented today in a Distinguished Industry Abstract at the American College of Allergy, Asthma & Immunology (ACAAI) 2022 Annual Scientific Meeting, being held November 10-14 in Louisville, Kentucky.

Data presented are from 10 adult patients with HAE in Phase 1, dose-escalation portion of the study, with data cut-off September 28, 2022. Single doses of 25 mg (n=3), 50 mg (n=4), and 75 mg (n=3) NTLA-2002 were administered via intravenous infusion and changes from baseline in plasma kallikrein protein were measured for each patient.

Plasma kallikrein reduction
NTLA-2002 administration resulted in a profound, dose-dependent reduction in plasma kallikrein levels based on the full availability of cohort biomarker data, as described below. In the 25 mg and 75 mg cohorts, these large reductions in plasma kallikrein levels persisted throughout the observation period, which ran from week 16 to week 32.

cohort Mean reduction in plasma kallikrein
At the latest follow-up
25 mg (n=3) 64% (Week 32)
50mg…

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