Intellia Therapeutics Highlights its Three-Year Strategic Priorities and Anticipated 2024 Key Milestones
04 Januar 2024 - 10:01PM
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
through 2026 and key anticipated 2024 milestones that support the
company’s mission to transform the lives of patients and bring
forth a new era in medicine.
“2023 was a momentous year for Intellia as our lead
investigational therapy, NTLA-2001, advanced into the final stage
of clinical development for the treatment of transthyretin
amyloidosis with cardiomyopathy,” said Intellia President and Chief
Executive Officer John Leonard, M.D. “Looking ahead, we will
continue to turn groundbreaking science into real-world medicines
by capitalizing on our extensive experience and capabilities to
edit disease-causing genes. We are rapidly expanding the potential
to treat an even larger range of diseases, including those that
originate outside of the liver, by deploying our novel delivery and
editing technologies. With our comprehensive gene editing toolbox,
we are well-positioned to harness the full potential of
CRISPR-based medicines for patients.”
Recent Pipeline Advancement and Corporate
Updates
- NTLA-2001 for Transthyretin
(ATTR) Amyloidosis: Initiated and actively enrolling the
pivotal Phase 3 MAGNITUDE trial in adults with ATTR amyloidosis
with cardiomyopathy (ATTR-CM).
- NTLA-2002 for Hereditary
Angioedema (HAE): Completed enrollment and dosing in the
Phase 2 portion of the Phase 1/2 study in adults with HAE.
- NTLA-3001 for Alpha-1
Antitrypsin Deficiency (AATD)-Associated Lung Disease:
Submitted a clinical trial application to initiate a first-in-human
Phase 1 study of NTLA-3001 ahead of previous Q1 2024
expectation.
- Organizational
Streamlining: Following an internal strategic review,
Intellia will be streamlining company-wide operations to further
focus resources on key strategic priorities and programs. These
changes will result in a pause of select exploratory research-stage
programs and a workforce reduction of approximately 15%.
- Cash Runway:
Intellia ended the fourth quarter of 2023 with approximately $1.0
billion in cash, cash equivalents and marketable securities. The
cash position is expected to fund operations into mid-2026.
- Board Update: In
December, Intellia announced the departure of John Crowley from its
board of directors following his appointment as Chief Executive
Officer of the Biotechnology Innovation Organization (BIO), the
life sciences industry’s leading trade and advocacy
association.
2024 – 2026 Strategic
Priorities
- Complete patient enrollment
for pivotal studies of NTLA-2001 and NTLA-2002, including
planned biologics license application (BLA) submission for
NTLA-2002 in 2026;
- Launch clinical trials for
next wave of in vivo and
ex vivo programs, validating
CRISPR-based in vivo targeted gene insertion and an allogeneic cell
engineering solution designed to avoid NK cell-mediated rejection;
and
- Expand the range of diseases
that can be targeted by deploying new editing and delivery
innovations, including advancing gene editing in tissues
outside the liver and DNA writing technology.
Anticipated 2024 Milestones
- NTLA-2001 for ATTR
Amyloidosis:
- Dose the first patient in MAGNITUDE
trial of NTLA-2001 for the treatment of ATTR-CM in Q1 2024.
- Continue to open new sites and
enroll patients, globally, in the MAGNITUDE study.
- Prepare for the Phase 3 study of
NTLA-2001 for the treatment of ATTR amyloidosis with polyneuropathy
(ATTRv-PN) in 2024.
- Present updated clinical data from
the ongoing Phase 1 study of NTLA-2001 in 2024.
- NTLA-2002 for HAE:
- Initiate the global pivotal Phase 3
trial of NTLA-2002 in 2H 2024, subject to regulatory feedback.
- Present updated data from Phase 1
and new data from Phase 2 portion in 2024.
- NTLA-3001 for
AATD-Associated Lung Disease:
- Dose first patient in Phase 1 study
of NTLA-3001 in 2024.
Presentation at the 42nd
Annual J.P. Morgan Healthcare Conference
John Leonard, M.D., President and Chief Executive Officer of
Intellia, will present a company overview at the 42nd Annual J.P.
Morgan Healthcare Conference in San Francisco on Tuesday, January
9, at 5:15 p.m. PT (8:15 p.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. The company’s in
vivo programs use CRISPR to enable precise editing of
disease-causing genes directly inside the human body.
Intellia’s ex vivo programs use CRISPR to engineer human
cells outside the body for the treatment of cancer and autoimmune
diseases. 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
expand and validate its industry-leading gene 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, NTLA-2002 for the treatment of hereditary angioedema
(“HAE”), and NTLA-3001 for the treatment of alpha-1 antitrypsin
deficiency (“AATD”)-associated lung disease, including the expected
timing of data releases, regulatory filings, and the initiation,
dosing, and completion of clinical trials, such as the dosing of
the first patient in the pivotal Phase 3 MAGNITUDE trial of
NTLA-2001 for ATTR amyloidosis with cardiomyopathy (“ATTR-CM”) in
Q1 2024, the preparation for a Phase 3 study of NTLA-2001 for the
treatment of ATTR amyloidosis with polyneuropathy (“ATTRv-PN”) in
2024, the presentation of updated clinical data from the ongoing
Phase 1 study of NTLA-2001 in 2024, the initiation of a global
pivotal Phase 3 trial of NTLA-2002 in 2H 2024, the presentation of
additional data from the Phase 1/2 trial of NTLA-2002 in 2024, the
submission of a biologics license application for NTLA-2002 in
2026, and the dosing of the first patient in the Phase 1 study of
NTLA-3001 for the treatment of AATD-associated lung disease in
2024; the advancement of additional development candidates and
expectations of advancing such development candidates and releasing
data related to such technologies and development candidates,
including its ability to launch clinical trials for its next wave
of in vivo and ex vivo programs and to validate CRISPR-based in
vivo targeted gene insertion and an allogeneic cell engineering
solution designed to avoid NK cell-mediated rejection; 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 fund operations into mid-2026.
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.
Intellia Contacts:
Investors:Ian KarpSenior Vice President,
Investor Relations and Corporate
Communicationsian.karp@intelliatx.com
Lina LiSenior Director, Investor Relations and Corporate
Communicationslina.li@intelliatx.com
Media:Matt CrensonTen Bridge
Communicationsmedia@intelliatx.com
mcrenson@tenbridgecommunications.com
Intellia Therapeutics (NASDAQ:NTLA)
Historical Stock Chart
Von Apr 2024 bis Mai 2024
Intellia Therapeutics (NASDAQ:NTLA)
Historical Stock Chart
Von Mai 2023 bis Mai 2024