Business
Intellia and Regeneron Announce Updated Phase 1 Data Demonstrating a Single Dose of NTLA-2001, an Investigational CRISPR Therapy for Transthyretin (ATTR) Amyloidosis, Resulted in Rapid, Deep and Sustained Reduction in Disease-Causing Protein
Achieved 86% and 93% mean serum TTR reduction by day 28 at 0.7 mg/kg and 1.0 mg/kg doses, respectively, with dose-dependent reductions observed across all
About this update from Intellia Therapeutics, Inc.
[{"type":"text","content":"Achieved 86% and 93% mean serum TTR reduction by day 28 at 0.7 mg/kg and 1.0 mg/kg doses, respectively, with dose-dependent reductions observed across all four dose levelsDurable serum TTR reductions observed with patient follow-up ranging from 2 to 12 monthsNTLA-2001 was generally well tolerated at all dose levels On track to initiate polyneuropathy dose-expansion cohort (Part 2) in Q1 2022 Intellia to host investor event today, Monday, February 28, at 4:30 p.m. ET CAMBRIDGE, Mass. and TARRYTOWN, N.Y., Feb. 28, 2022 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA) and Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) today announced positive interim data from an ongoing Phase 1 clinical study of their lead in vivo genome editing candidate, NTLA-2001, which is being developed as a single-dose treatment for transthyretin (ATTR) amyloidosis. The interim data released today include 15 hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN) patients treated across four single-ascending dose cohorts. Single doses of 0.1 mg/kg, 0.3 mg/kg, 0.7 mg/kg, and 1.0 mg/kg of NTLA-2001 were administered via intravenous infusion and changes from baseline values of serum transthyretin (TTR) protein were measured for each patient. Treatment with NTLA-2001 led to dose-dependent reductions in serum TTR and achieved maximal reductions by day 28, with mean reductions of 52%, 87%, and 86% among the three patients each in the 0.1 mg/kg, 0.3 mg/kg, and 0.7 mg/kg dose groups, respectively, and 93% among the six patients in the 1.0 mg/kg dose group. Mean serum TTR reduction remained durable through the observation period, with patient follow-up ranging from two to 12 months. Additionally, the serum TTR reduction observed was consistent across all patients receiving doses at or greater than 0.3 mg/kg. At the 1.0 mg/kg dose level, all six patients achieved greater than 80% reduction and four of six patients achieved greater than 90% reduction by day 28. Further, the reduction in serum TTR observed at day 28 was sustained through the last measured timepoint for each of the six patients, which ranged from two to six months. “Today’s update reinforces Intellia’s progress in opening a new era of medicine. These data suggest that treatment with a one-time, systemically delivered CRISPR-based investigational therapy has the potential to substantially r...