Intellia Therapeutics Posts Back To Back Positive Data From Gene Editing Therapies

  • Intellia Therapeutics Inc NTLA and Regeneron Pharmaceuticals Inc REGN announced positive interim results from an ongoing Phase 1 trial of NTLA-2001, a CRISPR/Cas9 genome editing therapy for transthyretin (ATTR) amyloidosis. 
  • The interim data include 12 ATTR amyloidosis adult patients with cardiomyopathy (ATTR-CM) with heart failure. 
  • Interim data from the cardiomyopathy arm of the Phase 1 study of NTLA-2001 showed deep and sustained mean serum transthyretin (TTR) reductions of 93% and 92% at 0.7 mg/kg and 1.0 mg/kg doses, respectively.
  • Related: Intellia Therapeutics Shares Are Gaining After Positive Data From Second CRISPR Candidate.
  • At both dose levels, NTLA-2001 was generally well tolerated. Two of 12 patients reported transient infusion reactions.
  • One patient in the 0.7 mg/kg dose experienced a Grade 3 infusion-related reaction, which resolved without clinical consequence. No clinically significant liver findings were observed at either dose level.
  • A protocol amendment has been submitted to evaluate a fixed dose corresponding to 0.7 mg/kg in the dose-expansion portion, with enrollment across both arms expected to be completed by the end of 2022, subject to regulatory feedback.
  • Price Action: NTLA shares are down 4.13% at $65.75 during the premarket session on the last check Friday. REGN stock closed at $705.85 on Thursday.
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