Intellia Therapeutics Highlights Strategic Priorities and Anticipated Development Milestones for 2021
- Continued advancement of global Phase 1 study of NTLA-2001, a potentially curative single-course therapy for transthyretin amyloidosis (ATTR)
- Anticipates submitting an IND or IND-equivalent in mid-2021 for NTLA-5001 for the treatment of acute myeloid leukemia (AML)
- Expects to submit an IND or IND-equivalent in 2H 2021 for NTLA-2002 for the treatment of hereditary angioedema (HAE)
- Plans to nominate at least one new development candidate in 2021 from broad research efforts and continued platform innovation
- Ended 2020 in strong financial position with
- Clinical validation: Evaluate the clinical profile of NTLA-2001 as a single-course therapy for transthyretin amyloidosis (ATTR) and Intellia’s in vivo non-viral, lipid nanoparticle (LNP)-based CRISPR/Cas9 delivery system as a platform for achieving clinically-relevant protein reduction for patients;
- Full-spectrum pipeline advancement: Rapidly progress in vivo and engineered cell therapy candidates for genetic diseases and cancers towards the clinic; and
- Platform innovation: Extend Intellia’s continued scientific leadership across genome editing, delivery and cell engineering capabilities.
“Since our founding, we set out to develop modular platform components that could serve as the engine powering an expansive portfolio of curative therapeutics. We have paved a rapid and reproducible development path for both in vivo and engineered cell therapies to address serious genetic diseases and cancers,” said Intellia President and Chief Executive Officer
Anticipated 2021 Milestones:
- NTLA-2001 for ATTR: NTLA-2001 is the first systemically delivered CRISPR-based therapy dosed in a patient, and could potentially be the first curative treatment for ATTR. By applying the Company’s in vivo LNP technology, NTLA-2001 offers the possibility of halting and reversing the disease with potent, lifelong transthyretin (TTR) protein reduction after a single course of treatment. NTLA-2001 is part of a co-development/co-promotion agreement between Intellia, the lead party, and Regeneron Pharmaceuticals, Inc. (Regeneron).
- Intellia is continuing to enroll patients in the global Phase 1 study of NTLA-2001 in adults with hereditary ATTR with polyneuropathy (hATTR-PN) in order to establish an optimal dose. 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 modular LNP delivery platform.
- Intellia intends to evaluate NTLA-2001 in a broader ATTR population of both polyneuropathy and cardiomyopathy patients following its Phase 1 safety assessment and dose optimization.
- NTLA-5001 for AML: NTLA-5001 is a potential best-in-class engineered T cell therapy designed to treat all genetic subtypes of acute myeloid leukemia (AML). This investigational candidate is a T cell receptor (TCR)-T cell therapy targeting the Wilms’ Tumor 1 (WT1) antigen utilizing Intellia’s proprietary cell engineering process.
- Intellia plans to submit an Investigational New Drug (IND) or equivalent regulatory application for NTLA-5001 in mid-2021. The first-in-human trial is expected to evaluate the safety and activity of NTLA-5001 in patients with persistent or recurrent AML who have previously received first-line therapies.
- The Company is also evaluating the potential use of NTLA-5001 to treat WT1-positive solid tumors in preclinical studies.
- NTLA-2002 for HAE: NTLA-2002 aims to prevent attacks and eliminate the current, significant treatment burden for people living with hereditary angioedema (HAE) after a single course. Intellia is applying its modular LNP delivery system to develop NTLA-2002 to knock out the KLKB1 gene in the liver to permanently reduce plasma kallikrein activity.
- Intellia plans to submit an IND or equivalent regulatory application in the second half of 2021.
- The Company is applying insights gained from NTLA-2001 to expedite clinical development of NTLA-2002.
- Pipeline Expansion: Intellia is focused on advancing its differentiated genome editing, delivery and cell engineering strategies to broaden in vivo and ex vivo applications for wholly owned and partnered programs. The Company continues to progress its robust research efforts and modular platform to develop new therapeutic candidates for genetic diseases requiring removal and/or restoration of a protein, as well as the next generation of engineered cell therapies for cancers.
- Intellia today announced plans to nominate at least one additional development candidate in 2021.
- Intellia, in partnership with lead party Regeneron, is also continuing to advance hemophilia A and B therapeutic programs toward IND-enabling studies using their jointly-developed in vivo targeted insertion technology.
- The Company plans to present at upcoming scientific conferences, with updates on multiple in vivo targets in the liver and other tissues and an allogeneic solution that enables the next generation of engineered cell therapies.
Cash Position and Financial Guidance
- Intellia ended the fourth quarter of 2020 with approximately
$597 millionin cash, cash equivalents and marketable securities. Intellia expects that its cash, cash equivalents and marketable securities as of December 31, 2020will enable the Company to fund its anticipated operating expenses and capital expenditure requirements for at least the next 24 months. This expectation excludes any strategic use of capital not currently in the Company’s base-case planning assumptions.
Intellia Therapeutics is a leading genome editing company, focused on the development of proprietary, potentially curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by both producing therapeutics that permanently edit and/or correct disease-associated genes in the human body with a single treatment course, and creating enhanced engineered cells that can treat oncological and immunological diseases. Intellia’s combination of deep scientific, technical and clinical development experience, along with its leading intellectual property portfolio, puts it in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create new classes of therapeutic products. Learn more about Intellia and CRISPR/Cas9 at intelliatx.com. Follow us on Twitter @intelliatweets.
This press release contains “forward-looking statements” of
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 our ability to protect and maintain our intellectual property position; risks related to our relationship with third parties, including our licensors and licensees; risks related to the ability of our licensors to protect and maintain their intellectual property position; uncertainties related to regulatory agencies’ evaluation of regulatory filings and other information related to our product candidates; uncertainties related to the authorization, initiation and conduct of studies and other development requirements for our product candidates; the risk that any one or more of our product candidates, including those that are co-developed, will not be successfully developed and commercialized; the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies; and the risk that our collaborations with Regeneron or our other ex vivo collaborations will not continue or will not be 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 annual report on Form 10-K as well as discussions of potential risks, uncertainties, and other important factors in Intellia’s other filings with the
Senior Vice President
Source: Intellia Therapeutics, Inc.