Intellia Therapeutics Highlights Recent Progress and Anticipated 2020 Milestones
09 Janvier 2020 - 1:30PM
Intellia Therapeutics, Inc. (NASDAQ: NTLA), a leading genome
editing company focused on the development of curative therapeutics
using CRISPR/Cas9 technology both in vivo and ex vivo, today
provided an update on recent progress and the Company’s 2020
priorities and expected milestones.
“2020 will be a significant year for Intellia, as we execute on
our full-spectrum strategy. With milestones anticipated across our
pipeline, we are making important progress towards the development
of curative treatments for severe diseases. In particular, we
expect to dose ATTR patients with the first-ever systemically
delivered CRISPR/Cas9-based therapy this year, and we are beginning
IND-enabling activities for our newly announced development
candidate, NTLA-5001, a WT1-TCR-directed engineered cell therapy,
for treatment of AML,” said Intellia President and Chief Executive
Officer, John Leonard, M.D. “We are focused on developing a robust
platform with modular genome editing capabilities that enable a
fast and reproducible path to development. Today’s update reflects
this strategy, and it also features the announcement of our third
development program, an in vivo knockout approach for HAE.
Importantly, this program leverages the infrastructure and insights
from NTLA-2001 and underscores our ability to produce a rapid
succession of new clinical candidates. We are excited by the strong
momentum across our diverse pipeline and look forward to providing
updates on our development programs in the upcoming
year.”
Program Updates and Anticipated 2020
Milestones:
- ATTR Program: Intellia remains on track to
submit an investigational new drug (IND) application in mid-2020
for its lead in vivo candidate, NTLA-2001, for treatment of
transthyretin amyloidosis (ATTR). NTLA-2001 is anticipated to be
the first systemically delivered CRISPR/Cas9 therapy to enter the
clinic, and Intellia anticipates dosing the first patients in the
second half of 2020. In addition, Intellia completed a 12-month
durability study of its lead lipid nanoparticle (LNP) formulation
in support of NTLA-2001, maintaining an average reduction of
>95% of serum transthyretin (TTR) protein and sustained liver
genome editing after a single dose in non-human primates (NHPs).
NTLA-2001 is part of a co-development/co-promotion (Co/Co)
agreement between Intellia, which is the lead development and
commercialization party, and Regeneron Pharmaceuticals, Inc.
(Regeneron). Intellia and Regeneron have a 75% and 25% share of
worldwide development costs and profits, respectively.
- AML Program: Intellia today announced
NTLA-5001 as its first engineered T cell therapy development
candidate, utilizing its T cell receptor (TCR)-directed approach to
target the Wilms’ Tumor 1 (WT1) intracellular antigen for the
treatment of acute myeloid leukemia (AML). Intellia’s
WT1-TCR-directed approach aims to develop a broadly applicable
treatment for AML patients, regardless of mutational background of
a patient’s leukemia. This approach employs CRISPR/Cas9 to
efficiently knock out and replace the endogenous TCR with a
natural, high affinity therapeutic TCR. The resulting cells are
capable of specific and potent killing of AML blasts, and have no
detectable bone marrow cell toxicity. The Company expects to
present preclinical data in support of NTLA-5001 at an upcoming
scientific meeting in the first quarter of 2020 and plans to submit
an IND application in the first half of 2021. Additional efforts
are underway to evaluate the potential use of the WT1-TCR construct
to treat other tumor types, including solid tumors.
- HAE Program: Today, Intellia announced that
the Company is committed to developing a CRISPR/Cas9-based therapy
for hereditary angioedema (HAE) as its third development program.
HAE is a rare genetic disorder characterized by recurring and
unpredictable severe swelling attacks in various parts of the body,
and is significantly debilitating or even fatal in certain cases.
The disease is caused by increased levels of bradykinin, a protein
which leads to swelling. Most patients with HAE have a C1 esterase
inhibitor (C1-INH) protein deficiency, which normally prevents the
unregulated release and buildup of bradykinin. Using its modular
LNP-based CRISPR/Cas9 delivery system, Intellia aims to knock out
the kallikrein B1 (KLKB1) gene, which is part of a biological
pathway that results in release of bradykinin. Knocking out this
gene should reduce the undesired bradykinin activity in HAE
patients. The Company plans to present preclinical data at an
upcoming scientific meeting in the first quarter of 2020. In
addition, Intellia is evaluating several potential guide RNAs and
expects to nominate a development candidate in the first half of
2020. Intellia’s KLKB1 HAE program is subject to an option by
Regeneron to enter into a Co/Co agreement, in which Intellia would
remain the lead party.
Cash Position and Financial Guidance:
- Intellia ended the fourth quarter of 2019 with approximately
$284.5 million in cash, cash equivalents and marketable securities.
Intellia expects that its cash, cash equivalents and marketable
securities as of December 31, 2019 will enable the Company to fund
its anticipated operating expenses and capital expenditure
requirements at least through the end of 2021. This expectation
excludes any strategic use of capital not currently in the
Company’s base-case planning assumptions.
About Intellia Therapeutics
Intellia Therapeutics is a leading genome editing company
focused on developing proprietary, curative therapeutics using the
CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology
has the potential to transform medicine by permanently editing
disease-associated genes in the human body with a single treatment
course, and through improved cell therapies that can treat cancer
and immunological diseases, or can replace patients’ diseased
cells. The combination of deep scientific, technical and clinical
development experience, along with its leading intellectual
property portfolio, puts Intellia in a unique position to unlock
broad therapeutic applications of the CRISPR/Cas9 technology and
create a new class of therapeutic products. Learn more about
Intellia Therapeutics and CRISPR/Cas9
at intelliatx.com and 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 its planned submission of an investigational
new drug (“IND”) application for NTLA-2001 for the treatment of
transthyretin amyloidosis (“ATTR”) in mid-2020; its plans to submit
an IND application for NTLA-5001, its first T cell receptor
(“TCR”)-directed engineered cell therapy development candidate for
its acute myeloid leukemia (“AML”) program in the first half of
2021; its plans to nominate a development candidate for its
hereditary angioedema (“HAE”) program in the first half of 2020;
its plans to advance and complete preclinical studies, including
non-human primate studies for its ATTR program, AML program, HAE
program and other in vivo and ex vivo programs; its presentation of
additional data at upcoming scientific conferences, and other
preclinical data in 2020; the advancement and expansion of its
CRISPR/Cas9 technology to develop human therapeutic products, as
well as maintain and expand its related intellectual property
portfolio; the ability to demonstrate its platform’s modularity and
replicate or apply results achieved in preclinical studies,
including those in its ATTR, AML and HAE programs, in any future
studies, including human clinical trials; its ability to develop
other in vivo or ex vivo cell therapeutics of all types, and those
targeting WT1 in AML in particular, using CRISPR/Cas9 technology;
its business plans and objectives for its preclinical studies and
clinical trials, including the therapeutic potential and clinical
benefits thereof, as well as the potential patient populations that
may be addressed by its ATTR program, AML program, HAE program and
other in vivo and ex vivo programs; the impact of its
collaborations on its development programs, including but not
limited to its collaboration with Regeneron Pharmaceuticals, Inc.
(“Regeneron”) and Regeneron’s ability to enter into a Co/Co
agreement for the HAE program; statements regarding the timing of
regulatory filings for its development programs; its use of
capital, including expenses, future accumulated deficit and other
financial results during 2019 or in the future; and the ability to
fund operations through the end of 2021.
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 our
intellectual property position; risks related to Intellia’s
relationship with third parties, including our licensors; risks
related to the ability of our licensors to protect and maintain
their intellectual property position; uncertainties related to the
initiation and conduct of studies and other development
requirements for our product candidates; the risk that any one or
more of Intellia’s product candidates will not be successfully
developed and commercialized; and the risk that the results of
preclinical studies or clinical studies will not be predictive of
future results in connection with future studies. 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 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: Lina Li Associate Director Investor
Relations +1 857-706-1612 lina.li@intelliatx.com
Media: Jennifer Mound Smoter Senior Vice
President External Affairs & Communications +1 857-706-1071
jenn.smoter@intelliatx.com
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