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Intellia and Regeneron Announce Initial Data from the Cardiomyopathy Arm of Ongoing Phase 1 Study of NTLA-2001, an Investigational CRISPR Therapy for the Treatment of Transthyretin (ATTR) Amyloidosis
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%
About this update from Intellia Therapeutics, Inc.
[{"type":"text","content":"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, at day 28 \nNTLA-2001 was generally well-tolerated at both dose levels\nIntellia to discuss data at investor event today, Friday, September 16, at 8:00 a.m. ET\nCAMBRIDGE, Mass. and TARRYTOWN, N.Y., Sept. 16, 2022 /PRNewswire/ -- Intellia Therapeutics, Inc. (NASDAQ: NTLA) and Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) today announced positive interim results from an ongoing Phase 1 clinical trial of NTLA-2001, an investigational in vivo CRISPR/Cas9 genome editing therapy in development as a single-dose treatment for transthyretin (ATTR) amyloidosis. The interim data include 12 adult patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM) with New York Heart Association (NYHA) Class I – III heart failure. Single doses of 0.7 mg/kg and 1.0 mg/kg of NTLA-2001 were administered via intravenous infusion, and the change from baseline in serum transthyretin (TTR) protein concentration was measured for each patient.\nAdministration of NTLA-2001 led to rapid and deep reductions in serum TTR by day 28 as follows:\nCohort\nMean (min, max) % serum TTR reduction by day 28\n0.7 mg/kg, NYHA Class I/II (n=3)*\n92% (91%, 95%)\n0.7 mg/kg, NYHA Class III (n=6)*\n94% (91%, 97%)\n1.0 mg/kg, NYHA Class I/II (n=3)\n92% (90%, 95%)\n*Mean (min, max) % serum TTR reduction by day 28 for 0.7 mg/kg cohort (n=9) was 93% (91%, 97%).\nThese profound reductions in serum TTR were sustained throughout the observation period, with patient follow-up ranging from two to six months as of the data cut-off date of July 1, 2022. These data support NTLA-2001's potential as a one-time treatment to permanently inactivate the TTR gene and reduce the disease-causing protein in people with ATTR-CM.\n\"ATTR amyloidosis is a multifaceted disease in need of additional treatment options. These new interim results demonstrate that NTLA-2001 can profoundly reduce serum TTR levels in patients whose condition results in cardiomyopathy,\" said Intellia President and Chief Executive Officer John Leonard, M.D. \"Together with the previously reported data from the polyneuropathy arm of this landmark study, these results strongly suggest that NTLA-2001 could serve as a single-do...