Intellia Therapeutics Shares Are Gaining After Positive Data From Second CRISPR Candidate

  • Intellia Therapeutics Inc NTLA announced interim results from an ongoing Phase 1/2 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. 
  • The uncontrolled activity of kallikrein is responsible for the overproduction of bradykinin, which leads to fatal swelling attacks in HAE patients.
  • 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 biomarker data were available.
  • Treatment with a single dose of 25 mg of NTLA-2002 resulted in a mean reduction in HAE attacks of 91% throughout the 16-week observation period. Additionally, two of the three patients have not had a single HAE attack since treatment, and all three patients have been attack-free since week 10.
  • Patients in the 75 mg cohort have not completed the primary 16-week observation period.
  • NTLA-2002 was generally well-tolerated at both dose levels, and the majority of adverse events were mild in severity. The most frequent adverse events were infusion-related reactions, primarily mild and resolved within one day. 
  • Price Action: NTLA shares are up 7.20% at $73.52 during the premarket session on the last check Friday.
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