Intellia Shares Shoot Higher On Promising Biomarker Data From CRISPR/Cas9 Genome Editing Candidate

  • Intellia Therapeutics Inc NTLA and Regeneron Pharmaceuticals Inc REGN have announced positive interim data from an ongoing Phase 1 study of NTLA-2001, which is being developed as a single-dose treatment for transthyretin (ATTR) amyloidosis.
  • Interim results come from the first six patients for the in vivo CRISPR/Cas9 genome editing candidate that reduced serum levels of transthyretin, a key biomarker for the disease, by 87% in patients who received a higher dose. 
  • Mean reductions of 52% among the three patients in the 0.1 mg/kg dose group and 87% among the three patients in the 0.3 mg/kg dose group, including one patient with a 96% reduction.
  • The results bested the standard of care therapy, which typically reduces transthyretin by 80%.
  • NTLA-2001 was generally well-tolerated at both dose levels, with no serious adverse events and no liver findings by day 28. 
  • The candidate is continuing to be evaluated in the dose-escalation portion of the study.
  • After identifying a recommended dose, Intellia expects to begin a single-dose expansion cohort in Part 2 of the Phase 1 trial later this year. 
  • After completing the Phase 1 trial, the company plans to move to pivotal studies for both polyneuropathy and cardiomyopathy manifestations of ATTR amyloidosis.
  • Intellia will host an investor event today at 8:00 a.m. E.T.
  • Price Action: NTLA shares are up 31.1% at $116.49 during the premarket session on the last check Monday, while REGN stock closed at $550.55 on Friday.
Posted In: BiotechNewsHealth CareContractsFDAGeneralBriefsGene EditingTransthyretin Amyloidosis