On January 6, 2022 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapies leveraging CRISPR-based technologies, reported its expected milestones and the following strategic priorities for 2022 (Press release, Intellia Therapeutics, JAN 6, 2022, View Source [SID1234598317]):
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Accelerating clinical validation of in vivo pipeline: Further characterize the safety and efficacy of NTLA-2001, including in patients with cardiomyopathy and complete enrollment of the Phase 1 study; establish the initial clinical profile of NTLA-2002 as a single-dose therapy for the treatment of hereditary angioedema (HAE).
Strategic pipeline expansion: Significantly progress in vivo and ex vivo pipeline, including determining the initial safety profile of NTLA-5001 for acute myeloid leukemia (AML), advancing in vivo insertion candidates and nominating multiple new development candidates.
Platform innovation: Broaden the Company’s industry-leading platform through expansion of Intellia’s genome editing, delivery and cell engineering capabilities.
"Unequivocally, 2021 was a landmark year for Intellia. We demonstrated that our proprietary CRISPR-based platform and LNP technology can turn revolutionary science into potentially transformational medicines. Our platform enables us to advance genome editing approaches, which maximizes our ability to target a multitude of life-threatening diseases," said Intellia President and Chief Executive Officer John Leonard, M.D. "As we begin 2022 with great momentum, we are poised to significantly expand our full-spectrum pipeline of potentially curative therapies with the nomination of at least two new in vivo candidates and our first allogeneic development candidate during the year. Importantly, we look forward to sharing additional data from the ongoing study of NTLA-2001 and interim results from the Phase 1/2 study of NTLA-2002, which we expect will further demonstrate the modularity of our genome editing platform."
Anticipated 2022 Milestones:
In Vivo Programs
NTLA-2001 for ATTR amyloidosis: NTLA-2001 is the first investigational CRISPR-based therapy to be systemically delivered to edit genes inside the human body, and has the potential to be the first single-dose treatment for transthyretin (ATTR) amyloidosis. Delivered with the Company’s in vivo lipid nanoparticle (LNP) technology, NTLA-2001 offers the possibility of halting and reversing the disease by driving a deep, lifelong reduction in transthyretin (TTR) protein after a single dose. NTLA-2001 is part of a co-development/co-promotion agreement between Intellia, the lead party for this program, and Regeneron Pharmaceuticals, Inc. (Regeneron).
Today, Intellia announced that the first patient in the cardiomyopathy arm of the Phase 1 study has been dosed with NTLA-2001. This follows the Company’s recent announcement that the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) 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 now includes patients with ATTR-CM 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, which continues to evaluate NTLA-2001 in patients with hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN). Intellia expects to complete enrollment of the Phase 1 study for both ATTRv-PN and ATTR-CM subjects in 2022.
Intellia intends to present additional interim clinical data of NTLA-2001 in ATTRv-PN patients from Part 1, the single-ascending dose portion, and to initiate Part 2, a single-cohort expansion, in the first quarter of 2022. Data to be presented at a company-sponsored event will be from all four ATTRv-PN dose cohorts in Part 1 and include safety and serum TTR knockdown for Cohorts 3 and 4, as well as an early look at durability across all cohorts.
NTLA-2002 for HAE: NTLA-2002 leverages Intellia’s proprietary in vivo LNP delivery technology to knock out the KLKB1 gene in the liver with the potential to permanently reduce total plasma kallikrein protein and activity, a key mediator of HAE. This investigational approach aims to prevent attacks for people living with HAE by providing continuous suppression of plasma kallikrein activity following a single dose and to eliminate the significant treatment burden associated with currently available HAE therapies.
In December 2021, Intellia dosed the first patient in its second clinical study of a CRISPR-based therapeutic candidate evaluating NTLA-2002 for HAE. The first-in-human Phase 1/2 trial is expected to evaluate the safety, tolerability and activity of NTLA-2002 in adults with Type I or Type II HAE, and will continue to leverage insights gained from the development of NTLA-2001.
The Company expects to present interim data from the Phase 1/2 study in the second half of 2022. These results are expected to characterize the emerging safety and activity profile of NTLA-2002 and demonstrate preliminary proof-of-concept.
NTLA-3001 for AATD-associated lung disease: NTLA-3001 is Intellia’s wholly owned CRISPR-mediated in vivo targeted gene insertion development candidate. It is designed with the aim to precisely insert a healthy copy of the SERPINA1 gene, which encodes the alpha-1 antitrypsin (A1AT) protein, with the potential to restore permanent expression of functional A1AT protein to therapeutic levels after a single dose. This approach seeks to address alpha-1 antitrypsin deficiency (AATD)-associated lung disease and eliminate the need for sub-optimal weekly IV infusions of A1AT augmentation therapy or lung transplant in severe cases.
Intellia is conducting Investigational New Drug (IND)-enabling activities for NTLA-3001 with plans to file an IND or IND-equivalent in 2023. The Company also continues to explore additional editing strategies for AATD.
Ex Vivo Programs
NTLA-5001 for AML: NTLA-5001 is an investigational autologous T cell receptor (TCR)-T cell therapy engineered to target the Wilms’ Tumor 1 (WT1) antigen for the treatment of all genetic subtypes of acute myeloid leukemia (AML).
In the fourth quarter of 2021, Intellia initiated screening of patients in the Phase 1/2a study of NTLA-5001 for patients with AML. The Company expects to dose its first patient in the coming weeks and enroll patients throughout the year. Later this year, the Company plans to provide guidance around timing of the first expected data readout, with the goal of demonstrating clinical proof-of-concept for its TCR-based platform.
Modular Platform and Pipeline Expansion
Platform Innovation: Intellia is expanding its industry-leading genome editing platform and scientific leadership through editing, delivery and cell engineering innovations that will enable broader in vivo and ex vivo applications.
Intellia plans to advance at least two new in vivo development candidates by the end of 2022.
The Company expects to nominate its first allogeneic ex vivo development candidate by the first half of 2022.
The Company plans to highlight additional advances to its proprietary technology capabilities, including both genome editing and delivery tools, at upcoming scientific conferences in 2022.
Corporate Updates:
In January, Intellia and Kyverna Therapeutics announced a licensing and collaboration agreement for the development of KYV-201, an allogeneic CD19 CART-cell therapy for the treatment of a variety of B cell-mediated autoimmune diseases. Intellia may exercise an option to lead U.S. commercialization for KYV-201 under a co-development and co-commercialization agreement.
In December 2021, Intellia appointed Derek Hicks as Executive Vice President, Chief Business Officer. Mr. Hicks joins Intellia with more than 25 years of combined business, leadership and biotechnology experience, having most recently served as Head of Business Development at Spark Therapeutics.
Cash Position
Intellia ended the fourth quarter of 2021 with approximately $1.1 billion in cash, cash equivalents and marketable securities.
Intellia’s Presentation at the 40th Annual J.P. Morgan Healthcare Conference
Intellia is scheduled to present virtually at the 40th Annual J.P. Morgan Healthcare Conference on Wednesday, January 12, at 2:15 p.m. ET. A live audio webcast of Intellia’s presentation can be accessed under the Events and Presentations page of the Investors & Media section on the company’s website at www.intelliatx.com. A replay of the webcast will be available on Intellia’s website for at least two weeks following the presentation.