Full Press Release Details
Intellia Therapeutics Announces Anticipated 2025 Milestones and
Strategic Reorganization to Prioritize the Advancement of its Late-Stage Programs, NTLA-2002 and Nexiguran Ziclumeran (nex-z)
CAMBRIDGE, Mass., Jan. 9, 2025 Intellia
Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced its strategic priorities and key anticipated 2025 milestones that support the
Company s mission to transform the lives of patients and bring forth a new era in medicine.
We have made significant progress and built strong
momentum in 2024 with three actively enrolling, Phase 3, pivotal studies. Our early clinical data for both NTLA-2002 and nex-z support novel, highly differentiated product profiles that directly address the significant unmet needs of patients and
prescribers in HAE and ATTR, said John Leonard, M.D., President and Chief Executive Officer of Intellia. We understand the significant potential of our late-stage programs, and within a challenging market environment, have made a
difficult decision to focus our resources predominantly on NTLA-2002 and nex-z where we have the greatest opportunity to create significant, near-term value.
Recent Pipeline Advancement and Corporate Updates
Strategic Priorities and Anticipated 2025 Milestones
Intellia s strategic priorities reflect the Company s ongoing evolution from a late-stage development company to a commercial-ready organization by
1. Drive focused clinical execution to complete or accelerate enrollment in the pivotal studies of NTLA-2002 and nex-z
2. Advance commercial readiness by
implementing core commercialization and medical capabilities for initial launch in the U.S.
Presentation at the 43rd Annual J.P. Morgan
Healthcare Conference
John Leonard, M.D., President and Chief Executive Officer of Intellia, will present a company overview at the 43rd Annual J.P. Morgan Healthcare Conference in San Francisco on Monday, January 13, at 8:15 a.m. PT (11:15 a.m. ET). A live webcast will be available through the Events and Presentations page of
the Investors & Media section on Intellia s website, www.intelliatx.com. A replay of the webcast will be available on Intellia s website for a limited time following the conference.
About Intellia Therapeutics
Intellia Therapeutics, Inc.
(NASDAQ:NTLA) is a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies. Since its inception, Intellia has focused on leveraging gene editing technology to develop novel, first-in-class medicines that address important unmet medical needs and advance the treatment paradigm for patients. Intellia s deep scientific, technical and clinical
development experience, along with its people, is helping set the standard for a new class of medicine. To harness the full potential of gene editing, Intellia continues to expand the capabilities of its CRISPR-based platform with novel editing and
delivery technologies. Learn more at intelliatx.com and follow us @intelliatx.
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 concerning: its
ability to successfully develop and commercialize nexiguran ziclumeran ( nex-z ), formerly known as NTLA-2001, for the treatment of transthyretin ( ATTR ) amyloidosis and NTLA-2002 for the
treatment of hereditary angioedema ( HAE ) to address the significant unmet needs of patients and prescribers in HAE and ATTR; its ability to achieve near-term clinical milestones, including dosing the first patient in the Phase 3 HAELO
trial in the first quarter of 2025, completing enrollment in the the Phase 3 HAELO trial in the second half of 2025, dosing the first patient in the Phase 3 MAGNITUDE-2 trial for hereditary ATTR with
polyneuropathy ( ATTRv-PN ) in the first quarter of 2025, enroll at least 550 patients across the Phase 3 MAGNITUDE trial for ATTR with cardiomyopathy
( ATTR-CM ) by year-end, and the expected timing of data releases from its clinical trials of nex-z and NTLA-2002,
including longer-term data from the Phase 1/2 study of NTLA-2002, including data from patients that previously received the 25 mg dose or placebo and were subsequently given the 50 mg dose, and longer-term data from the Phase 1 study of nex-z, including updated measure of clinical efficacy and safety; its ability to prepare for commercial launch, including completing buildout of the commercial leadership team in the first half of 2025, expanding
the reach of medical education activities in HAE and ATTR amyloidosis in 2025, and initiating pre-approval information exchange to allow payers to begin planning for potential coverage and formulary decisions
in 2025; its interactions with regulatory authorities, including the potential submission of a biologics license application for NTLA-2002 for the treatment of HAE in the second half of 2026; its ability to optimize the impact of its collaborations
on its development programs, including its collaboration with Regeneron Pharmaceuticals, Inc. and their co-development program for ATTR amyloidosis, and to advance additional development candidates; and its
expectations regarding its uses of capital, expenses, and ability to fund operations into the first half of 2027.
Any forward-looking statements in this press release are based on management s current expectations and
beliefs of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and
uncertainties include, but are not limited to: risks related to Intellia s ability to protect and maintain its intellectual property position; risks related to Intellia s relationship with third parties, including its contract
manufacturers, licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to the authorization, initiation and conduct of preclinical and clinical
studies and other development requirements for its product candidates, including uncertainties related to regulatory approvals to conduct clinical trials; risks related to the ability to develop and commercialize any one or more of Intellia s
product candidates successfully; risks related to the results of preclinical studies or clinical studies not being predictive of future results in connection with future studies; the risk that clinical study results will not be positive; risks
related to the development and advancement of novel platform capabilities, such as DNA writing technology and gene editing in tissues outside the liver; risks related to Intellia s future financial condition and its ability to fund its
operations; and risks related to Intellia s collaborations with Regeneron Pharmaceuticals, Inc. or its other collaborations not continuing or not being successful. For a discussion of these and other risks and uncertainties, and other
important factors, any of which could cause Intellia s actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in Intellia s most recent quarterly report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in Intellia s other filings with the Securities and Exchange Commission. All information in this press
release is as of the date of the release, and Intellia undertakes no duty to update this information unless required by law.
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