Intellia Therapeutics Highlights Strategic Priorities and Anticipated 2023 Key Milestones
05 Janvier 2023 - 1:30PM
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage
genome editing company focused on developing potentially curative
therapies leveraging CRISPR-based technologies, today announced its
strategic priorities for the upcoming two years as the Company
enters its next phase of pipeline execution and platform
innovation.
2023 – 2024 Strategic
Priorities
- Initiate global pivotal
trials for Intellia’s first two investigational in vivo
CRISPR-based therapies, NTLA-2001 for transthyretin (ATTR)
amyloidosis and NTLA-2002 for hereditary angioedema (HAE);
- Advance new platform
capabilities to the clinic, including CRISPR-based in vivo
targeted gene insertion and a first-of-its-kind allogeneic cell
engineering solution designed to avoid NK cell-mediated rejection;
and
- Lead the development of new
gene editing and delivery modalities, compatible with its
modular platform, which will extend Intellia’s position of
technological leadership and drive future pipeline growth.
“2022 proved to be another outstanding year for Intellia, with
several significant clinical milestones achieved across our
pipeline, further reinforcing the ability of our modular CRISPR
genome editing platform to target a broad range of diseases,” said
Intellia President and Chief Executive Officer John Leonard, M.D.
“These accomplishments reflect steady execution against our core
strategy: to harness the immense power of genome editing, both for
in vivo and ex vivo applications. As we look ahead, our highest
priority will be to prepare for the initiation of global pivotal
trials for our first two investigational in vivo CRISPR-based
therapies, NTLA-2001 for ATTR amyloidosis and NTLA-2002 for
hereditary angioedema. As these programs continue to progress, we
believe we are moving closer to setting a new standard of care for
people living with these and other serious diseases. In addition,
we are advancing the next wave of platform capabilities, such as in
vivo gene insertion and our proprietary allogeneic solution.
Importantly, while the possibilities to apply our industry-leading
genome editing technology are expansive, we are taking a
disciplined approach with our portfolio by deploying resources on
high-impact opportunities and collaborating with a network of other
scientific leaders to expand the applications of our innovative
technologies.”
Based on these strategic priorities, which will be the Company’s
focus over the next two years, Intellia anticipates reaching the
following key program milestones in 2023:
In Vivo Programs
- NTLA-2001 for ATTR
amyloidosis:
- Submit an IND application in
mid-2023 to enable inclusion of U.S. sites in a pivotal study of
NTLA-2001 for patients with ATTR amyloidosis with cardiomyopathy
(ATTR-CM).
- Present additional clinical data
from the ongoing Phase 1 study of NTLA-2001 in 2023.
- Initiate a global pivotal NTLA-2001
trial for ATTR-CM by year-end 2023, subject to regulatory
feedback.
- Prepare for a Phase 3 study of
NTLA-2001 for the treatment of ATTR amyloidosis with polyneuropathy
(ATTRv-PN), including discussions with regulatory authorities.
- NTLA-2002 for HAE:
- Initiate Phase 2 portion of the
ongoing NTLA-2002 Phase 1/2 study in 1H 2023.
- Submit an IND in 1H 2023 to support
inclusion of U.S. sites in the Phase 2 study of NTLA-2002.
- Present additional clinical data
from the ongoing first-in-human study of NTLA-2002 in 2023.
- Alpha-1 antitrypsin
deficiency (AATD) franchise:
- Submit an IND or IND-equivalent
application for NTLA-3001, Intellia’s wholly owned insertion
candidate in development for AATD-associated lung disease, in 2H
2023.
- Complete IND-enabling activities for
NTLA-2003, a wholly owned knockout candidate for AATD-associated
liver disease, by year-end 2023.
- Prevalent diseases:
- Progress one new in vivo development
candidate, nominated in 2022, for the treatment of an undisclosed
prevalent condition.
Ex Vivo Programs
- NTLA-6001 for CD30+
Lymphomas:
- Identify collaboration opportunities
to advance development of NTLA-6001.
- Additional
ex vivo candidates:
- Advance multiple programs, wholly
owned or in collaboration with partners, utilizing allogeneic
platform.
Platform Innovation
- Advance novel gene editing
technologies, including DNA writing, and delivery to other tissues
outside of the liver.
Cash Position
- Intellia ended the fourth quarter of
2022 with approximately $1.3 billion in cash, cash equivalents and
marketable securities.
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 @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
expand and validate its industry-leading genome editing platform,
including the advancement of novel gene editing technologies, such
as DNA writing, and delivery to other tissues outside of the liver;
the safety, efficacy, success and advancement of its clinical
programs for NTLA-2001 for the treatment of transthyretin (“ATTR”)
amyloidosis and NTLA-2002 for the treatment of hereditary
angioedema (“HAE”), including the expected timing of data releases,
regulatory filings, and the initiation, dosing, and completion of
clinical trials, such as the submission of an investigational new
drug (“IND”) for NTLA-2001 in mid-2023 and for NTLA-2002 in 1H
2023, the initiation of a global pivotal NTLA-2001 trial for
ATTR-CM in 2023, and the initiation of the Phase 2 portion of the
ongoing NTLA-2002 Phase 1/2 study in 1H 2023; the advancement of
development candidates, such as NTLA-3001 for the treatment of
alpha-1 antitrypsin deficiency (“AATD”)-associated lung disease,
NTLA-2003 for AATD-associated liver disease and NTLA-6001 for CD30+
lymphomas, including the timing and success of its IND-enabling
activities and its IND or IND-equivalent submissions, such as the
submission of an IND or IND-equivalent for NTLA-3001 in 2023; its
ability to advance additional development candidates and timing
expectations of advancing such development candidates and releasing
data related to such technologies and development candidates,
including candidates utilizing its allogeneic platform; 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 ability to identify new collaboration opportunities.
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, 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.
Intellia Contacts:
Investors:Ian KarpSenior Vice President,
Investor Relations and Corporate
Communications+1-857-449-4175ian.karp@intelliatx.com
Lina LiSenior Director, Investor Relations and Corporate
Communications+1-857-706-1612lina.li@intelliatx.com
Media:Matt CrensonTen Bridge
Communications+1-917-640-7930media@intelliatx.com
mcrenson@tenbridgecommunications.com
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