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Intellia and Regeneron Present Updated Interim Data from Phase 1 Study of CRISPR-based NTLA-2001 for the Treatment of Transthyretin (ATTR) Amyloidosis Demonstrating that Deep Serum TTR Reductions Remained Durable After a Single Dose
Serum TTR reductions were sustained at all doses tested with follow-up now reaching 12 months in the 0.1 and 0.3 mg/kg and six months in the 0.7 and 1.0 mg/kg
About this update from Intellia Therapeutics, Inc.
[{"type":"text","content":"Serum TTR reductions were sustained at all doses tested with follow-up now reaching 12 months in the 0.1 and 0.3 mg/kg and six months in the 0.7 and 1.0 mg/kg cohorts\nPharmacokinetic modeling and simulation indicated that an 80 mg fixed dose provides similar exposure to the 1.0 mg/kg dose, where treatment with NTLA-2001 resulted in 93% mean and 98% maximum serum TTR reduction by day 28\nIntellia to host investor event to discuss updated data from Phase 1 study of NTLA-2001, the first-ever systemically administered in vivo CRISPR investigational therapy today, Friday, June 24, at 8:00 a.m. ET\nCAMBRIDGE, Mass. and TARRYTOWN, N.Y., June 24, 2022 /PRNewswire/ -- Intellia Therapeutics, Inc. (NASDAQ: NTLA) and Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced additional positive interim data from an ongoing Phase 1 study of their lead investigational in vivo genome editing candidate, NTLA-2001, which is being developed as a single-dose treatment for transthyretin (ATTR) amyloidosis. The data were presented in an oral presentation at the European Association for the Study of the Liver (EASL) International Liver Congress™ 2022, taking place June 22 – 26 in London.\nThe presentation today included extended follow-up data from 15 patients with hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN) treated across four single-ascending dose cohorts in Part 1 of the study. Results demonstrated sustained durability of serum transthyretin (TTR) reduction through the last measured timepoint in the ongoing observation. These data support NTLA-2001's continued development as a potential one-time treatment to permanently inactivate the TTR gene and reduce the disease-causing protein. At the highest dose evaluated, treatment with NTLA-2001 at 1.0 mg/kg resulted in a 93% mean and 98% maximum serum TTR reduction by day 28 across the six patients treated. With longer-term follow-up data now available, these deep reductions continue to be sustained through six months, with an observed mean reduction of 93%. Additionally, three patients in the 1.0 mg/kg cohort have reached nine months in the follow-up period with no evidence of a loss in TTR reduction after a single dose. In the 0.7 mg/kg dose cohort, the 86% mean serum TTR reduction observed at day 28 also remained durable through six months. Further, in the 0.1 and 0.3 mg/kg cohorts...