Intellia Therapeutics Highlights Strategic Priorities and Anticipated Development Milestones for 2021
07 Janvier 2021 - 1:30PM
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing
company focused on developing curative therapeutics using
CRISPR/Cas9 technology both in vivo and ex vivo, today outlined its
expected 2021 milestones and the following strategic priorities:
- 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 John Leonard, M.D.
“Over the next 12 months, we will evaluate the clinical profile of
NTLA-2001, both as a one-time treatment option for ATTR patients
and as a validation of our non-viral approach to in vivo delivery.
In addition, we anticipate first-in-human regulatory submissions
for NTLA-5001 and NTLA-2002, at least one new development candidate
and new platform innovations to create the next wave of genomic
medicines. These priorities for 2021 reflect our long-term vision
for Intellia: to unlock genome editing’s full therapeutic
potential.”
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 million in cash, cash equivalents
and marketable securities. Intellia expects that its cash, cash
equivalents and marketable securities as of December 31, 2020 will
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.
About Intellia TherapeuticsIntellia
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.
Forward-Looking Statements This press release
contains “forward-looking statements” of Intellia Therapeutics,
Inc. (“Intellia”, “we” or “our”) 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
our: being able to enroll and dose the necessary subjects in our
clinical studies for NTLA-2001 for the treatment of transthyretin
amyloidosis (“ATTR”), provide timing on the first expected data
readout, and successfully secure additional clinical studies
authorizations, such as clinical trial applications (“CTA”), in
other countries; ability to evaluate NTLA-2001 in a broader ATTR
population; plans to submit an investigational new drug (“IND”)
application or similar clinical trial application for NTLA-5001,
our first T cell receptor (“TCR”)-directed engineered cell therapy
development candidate in our acute myeloid leukemia (“AML”) program
in mid-2021; expectations of evaluating the safety and activity of
NTLA-5001 in patients with persistent or recurrent AML who have
previously received first-line therapies; plans to submit an IND or
similar clinical trial application for our hereditary angioedema
(“HAE”) program in the second half of 2021; plans to nominate at
least one additional development candidate in 2021; plans to
advance and complete preclinical studies for our programs;
development of our modular platform to advance our complex genome
editing capabilities, such as gene insertion; further development
of our proprietary cell engineering process for multiple sequential
editing; presentation of additional data at upcoming scientific
conferences, and other preclinical data in 2021; advancement and
expansion of our CRISPR/Cas9 technology to develop human
therapeutic products, as well as our ability to maintain and expand
our related intellectual property portfolio; ability to demonstrate
our platform’s modularity and replicate or apply results achieved
in preclinical studies, including those in our ATTR, AML, and HAE
programs, in any future studies, including human clinical trials;
ability to develop other in vivo or ex vivo cell therapeutics of
all types, and those targeting Wilms’ Tumor 1 (WT1) in AML in
particular, using CRISPR/Cas9 technology; ability to optimize the
impact of our collaborations on our development programs, including
but not limited to our collaboration with Regeneron
Pharmaceuticals, Inc. (“Regeneron”), including our co-development
programs for hemophilia A and hemophilia B; Regeneron’s ability to
successfully co-develop products in the hemophilia A and B
programs, and the potential timing and receipt of future milestones
and royalties, or profits, as applicable, based on our license,
collaboration and, if applicable, co-development agreements with
Regeneron and Novartis; statements regarding the timing of
regulatory filings and clinical trial execution, including dosing
of patients, regarding our development programs; potential
commercial opportunities, including value and market, for our
product candidates; our expectations regarding our use of capital
and other financial results during 2021; and our ability to fund
operations for at least the next 24 months.
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 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:Lina LiAssociate DirectorInvestor
Relations+1 857-706-1612lina.li@intelliatx.com
Media:Jennifer Mound SmoterSenior Vice
PresidentExternal Affairs & Communications+1
857-706-1071jenn.smoter@intelliatx.com
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