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Intellia Therapeutics Receives Authorization to Initiate Phase 1 Clinical Trial of NTLA-2001 for Transthyretin Amyloidosis (ATTR)NTLA-2001: First single-course therapy that potentially halts and reverses ATTR On track to dose first patient by year-end with a systemically delivered CRISPR/Cas9-based therapy CAMBRIDGE, Mass., Oct. 19, 2020 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), announced the authorization of its Clinical Trial Application (CTA) by the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) to initiate its Phase 1 study, which will evaluate NTLA-2001 for the treatment of hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN). Intellia’s lead candidate, NTLA-2001 could be the first curative treatment for ATTR. By applying the company’s in vivo liver knockout technology, NTLA-2001 allows for the possibility of lifelong transthyretin (TTR) protein reduction after a single course of treatment. The investigational therapy is delivered via Intellia’s proprietary non-viral lipid nanoparticle platform, which the company is also using to develop in vivo treatments for other diseases. “Starting our global NTLA-2001 Phase 1 trial for ATTR patients is a major milestone in Intellia’s mission to develop medicines to cure severe and life-threatening diseases,” said Intellia’s President and Chief Executive Officer John Leonard, M.D. “Our trial is the first step toward demonstrating that our therapeutic approach could have a permanent effect, potentially halting and reversing all forms of ATTR. Once we have established safety and the optimal dose, our goal is to expand this study and rapidly move to pivotal studies, in which we aim to enroll both polyneuropathy and cardiomyopathy patients.” Goals of the NTLA-2001 Global Phase 1 Clinical Trial The company expects to dose the first patient in this Phase 1 trial by the end of 2020, subject to the impact of the COVID-19 pandemic. Intellia’s global first-in-human trial will be an open-label, multi-center, two-part study of NTLA-2001 in adults with hATTR-PN, the hereditary form of amyloidosis with peripheral nerve damage. The study will enroll up to 38 patients and consist of a single-ascending dose phase in Part 1 and, following the identification of an optimal dose, a single-cohort expansion in Part 2. The trial’s primary objectives are to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of NTLA-2001, which will include the measurement of serum TTR levels following a single intravenous infusion. The secondary objectives are to evaluate the efficacy of NTLA-2001 on clinical measures of neurologic function in hATTR-PN patients. “I am pleased to be leading the Phase 1 clinical trial in the U.K. for NTLA-2001, which I believe to be a breakthrough, single-course, genome editing therapy with the potential to transform the lives of ATTR patients around the world,” said Julian Gillmore, M.D, Ph.D., FRCP, Professor of Medicine, National Amyloidosis Centre, UCL Division of Medicine, Royal Free Hospital, U.K., the trial’s national coordinating investigator and an expert in the treatment of ATTR. “The U.K. is a pioneer and leading authority in genetic diagnosis, research and medicine, which is critical when supporting patients with rare diseases like ATTR.” Beyond its first application in the United Kingdom, Intellia is submitting additional regulatory applications in other countries as part of its ongoing, global development strategy. NTLA-2001 is part of a co-development/co-promotion agreement between Intellia, the lead development and commercialization party, and Regeneron Pharmaceuticals, Inc. More information about the NTLA-2001 Phase 1 trial may be found on clinicaltrials.gov when available.
About Intellia Therapeutics Forward-Looking Statements This press release contains “forward-looking statements” of Intellia Therapeutics, Inc. (“Intellia” or the “Company”) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellia’s beliefs and expectations regarding its: being able to initiate clinical studies for NTLA-2001 for the treatment of transthyretin amyloidosis (“ATTR”) pursuant to its clinical trial applications (“CTA”), including dosing of a first patient in the United Kingdom by the end of 2020, and submitting similar regulatory applications in other countries; plans to complete manufacturing activities and submit an IND or equivalent regulatory filing for other in vivo product candidates, such as NTLA-2002, a development candidate for its hereditary angioedema (“HAE”) program in the second half of 2021; plans to advance and complete preclinical studies, including any necessary non-human primate studies, for its HAE program, hemophilia A, hemophilia B, and other in vivo and ex vivo programs; development of a proprietary LNP/AAV hybrid delivery system and additional editing capabilities; advancement and expansion of its CRISPR/Cas9 technology to develop human therapeutic products, as well as its ability to maintain and expand its related intellectual property portfolio; ability to demonstrate its platform’s modularity and replicate or apply results achieved in preclinical studies, including those in its ATTR, HAE, hemophilia A and hemophilia B programs, in any future studies, including human clinical trials; expectations of the potential impact of the coronavirus disease 2019 pandemic on strategy, future operations and timing of its clinical trials or IND submissions; ability to optimize the impact of its collaborations on its development programs, including but not limited to its collaborations with Regeneron, including its co-development programs for ATTR, hemophilia A and hemophilia B; statements regarding the timing of regulatory filings regarding its development programs; use of capital, expenses, future accumulated deficit and other 2020 financial results or in the future; and ability to fund operations at least through the next 24 months. Intellia Contacts: Media: Lynnea Olivarez Investors: |