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Intellia Therapeutics Announces Expansion of Ongoing Phase 1 Study of NTLA-2001 to Include Adults with Transthyretin Amyloidosis with Cardiomyopathy (ATTR-CM)
Approved protocol amendment enables enrollment of ATTR-CM patients in the ongoing first-in-human study of NTLA-2001, a systemically delivered
About this update from Intellia Therapeutics, Inc.
[{"type":"text","content":"Approved protocol amendment enables enrollment of ATTR-CM patients in the ongoing first-in-human study of NTLA-2001, a systemically delivered CRISPR/Cas9-based therapy\nCAMBRIDGE, Mass., Nov. 22, 2021 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA) announced today that the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) has approved a protocol amendment for the Company’s ongoing Phase 1 study of NTLA-2001 to include patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM). The study of NTLA-2001 now includes patients with ATTR-CM to be enrolled in new dose-escalation and expansion cohorts. The inclusion of the ATTR-CM patient population is in addition to the original Phase 1 study population, which is currently evaluating NTLA-2001 in patients with ATTR amyloidosis with polyneuropathy (ATTRv-PN). The first investigational therapy of its kind, NTLA-2001 is an in vivo CRISPR/Cas9-based genome editing candidate being developed as a single-dose treatment for transthyretin (ATTR) amyloidosis. It is designed to inactivate the TTR gene in liver cells to prevent the production of transthyretin (TTR) protein and is being developed to potentially be the first single-dose ATTR amyloidosis treatment to not only halt but also reverse disease progression in both ATTRv-PN and ATTR-CM patients. NTLA-2001 has received orphan drug designation for the treatment of ATTR amyloidosis by both the European Commission and the U.S. FDA. “ATTR amyloidosis is a chronic, fatal disease that can impact different organs and tissues within the body, often manifesting as either polyneuropathy or cardiomyopathy. At Intellia, our goal is to develop a potentially curative treatment that could benefit as many patients living with this disease as possible,” said Intellia President and Chief Executive Officer John Leonard, M.D. “We have already seen promising interim data supporting the ability of NTLA-2001 to significantly reduce serum TTR levels in ATTR patients with polyneuropathy. We are excited to now expand our Phase 1 study of NTLA-2001 to include ATTR patients with cardiomyopathy in order to advance this potentially first-of-its-kind, single-dose treatment for more patients.” The protocol amendment to the Phase 1 study allows for enrollment of up to 36 adults in the United Kingdom with either hereditary ATTR-CM (ATTR...