Intellia Therapeutics Highlights Strategic Priorities and Anticipated Development Milestones for 2022
06 Janvier 2022 - 1:30PM
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing
company focused on developing curative therapies leveraging
CRISPR-based technologies, today outlined its expected milestones
and the following strategic priorities for 2022:
- 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.
About Intellia Therapeutics
Intellia Therapeutics, a leading clinical-stage genome editing
company, is developing novel, potentially curative therapeutics
leveraging CRISPR-based technologies. To fully realize the
transformative potential of CRISPR-based technologies, Intellia is
pursuing two primary approaches. The company’s in vivo programs use
intravenously administered CRISPR as the therapy, in which
proprietary delivery technology enables highly precise editing of
disease-causing genes directly within specific target tissues.
Intellia’s ex vivo programs use CRISPR to create the therapy by
using engineered human cells to treat cancer and autoimmune
diseases. Intellia’s deep scientific, technical and clinical
development experience, along with its robust intellectual property
portfolio, have enabled the company to take a leadership role in
harnessing the full potential of genome editing to create new
classes of genetic medicine. Learn more at intelliatx.com. Follow
us on Twitter @intelliatweets.
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: the safety, efficacy and advancement of its
clinical programs for NTLA-2001 for the treatment of transthyretin
amyloidosis, NTLA-2002 for the treatment of hereditary angioedema,
and NTLA-5001 for the treatment of acute myeloid leukemia pursuant
to its clinical trial applications (“CTA”) and IND submissions,
including the expected timing of data releases, regulatory filings,
and the initiation and completion of clinical trials; the
advancement of development candidates including NTLA-3001 for the
treatment of alpha-1 antitrypsin deficiency (AATD)-associated lung
disease; the ability to generate data to initiate clinical trials
and the timing of CTA and IND submissions; the expansion of its
CRISPR/Cas9 technology and related technologies to advance
additional development candidates, as well as its ability to
maintain and expand its related intellectual property portfolio;
expectations of the potential impact of the coronavirus disease
2019 pandemic on strategy, future operations and timing of its
clinical trials; and the 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 program for ATTR amyloidosis, and with Kyverna for
the development of KYV-201.
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 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 studies and
other development requirements for its product candidates,
including uncertainties related to regulatory approvals to conduct
clinical trials; the risk that any one or more of Intellia’s
product candidates 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; the risk that clinical study
results will not be positive; and the risk that Intellia’s
collaborations with Regeneron or Kyverna or its other
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 and 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 (“SEC”). 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.
Intellia Contacts:
Investors:Ian KarpSenior Vice President,
Investor Relations and Corporate
Communications+1-857-449-4175ian.karp@intelliatx.com
Lina LiDirector, Investor
Relations+1-857-706-1612lina.li@intelliatx.com
Media:Matt CrensonTen Bridge
Communications+1-917-640-7930media@intelliatx.commcrenson@tenbridgecommunications.com
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