Intellia Therapeutics Announces FDA Clearance of Investigational New Drug (IND) Application to Initiate a Pivotal Phase 3 Trial of NTLA-2001 for the Treatment of Transthyretin (ATTR) Amyloidosis with Cardiomyopathy
2023年10月18日 - 8:00PM
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage
genome editing company focused on developing potentially curative
therapies leveraging CRISPR-based technologies, today announced
that the U.S. Food and Drug Administration (FDA) has cleared the
company’s Investigational New Drug (IND) application for NTLA-2001
for the treatment of transthyretin (ATTR) amyloidosis with
cardiomyopathy. The global Phase 3 study of NTLA-2001, an in vivo
CRISPR-based gene editing candidate, is expected to initiate by
year-end 2023.
“The FDA clearance of the NTLA-2001 IND application allows us to
initiate a pivotal Phase 3 trial in the United States, marking the
first in vivo CRISPR-based candidate to begin late-stage clinical
development. This is another important step forward for Intellia
and our collaborator, Regeneron, as we aim to establish a new
standard of care for the treatment of ATTR amyloidosis,” said
Intellia President and Chief Executive Officer John Leonard, M.D.
“We are thrilled to further advance NTLA-2001 and our pipeline of
investigational gene editing therapies as we embark on a new era in
medicine. We look forward to sharing additional information about
the Phase 3 study at our upcoming quarterly earnings webcast, being
held on Thursday, November 9.”
About NTLA-2001Based on Nobel
Prize-winning CRISPR/Cas9 technology, NTLA-2001 could potentially
be the first single-dose treatment for ATTR amyloidosis. NTLA-2001
is the first investigational CRISPR therapy candidate to be
administered systemically, or through a vein, to edit genes inside
the human body. Intellia’s proprietary non-viral platform deploys
lipid nanoparticles to deliver to the liver a two-part genome
editing system: guide RNA specific to the disease-causing
gene and messenger RNA that encodes the Cas9 enzyme, which
carries out the precision editing. Robust preclinical and clinical
data, showing deep and long-lasting transthyretin (TTR) reduction
following in vivo inactivation of the target gene,
supports NTLA-2001’s potential as a single-administration
therapeutic. Intellia leads development and commercialization of
NTLA-2001 as part of a multi-target discovery, development and
commercialization collaboration with Regeneron. The
global Phase 1 trial is an open-label, multi-center, two-part study
of NTLA-2001 in adults with hereditary transthyretin amyloidosis
with polyneuropathy (ATTRv-PN) or transthyretin amyloidosis with
cardiomyopathy (ATTR-CM). The trial is now closed for enrollment.
Visit clinicaltrials.gov (NCT04601051) for more
details.
About Transthyretin (ATTR)
Amyloidosis Transthyretin amyloidosis, or ATTR
amyloidosis, is a rare, progressive and fatal disease. Hereditary
ATTR (ATTRv) amyloidosis occurs when a person is born with
mutations in the TTR gene, which causes the liver to
produce structurally abnormal transthyretin (TTR) protein with a
propensity to misfold. These damaged proteins build up as amyloid
in the body, causing serious complications in multiple tissues,
including the heart, nerves and digestive system. ATTRv amyloidosis
predominantly manifests as polyneuropathy (ATTRv-PN), which can
lead to nerve damage, or cardiomyopathy (ATTRv-CM), which can lead
to heart failure. Some individuals without the genetic mutation
produce non-mutated, or wild-type TTR proteins that become unstable
over time, misfolding and aggregating in disease-causing amyloid
deposits. This condition, called wild-type ATTR (ATTRwt)
amyloidosis, primarily affects the heart. There are an estimated
50,000 people worldwide living with ATTRv amyloidosis and between
200,000 and 500,000 people with ATTRwt amyloidosis.
About Intellia TherapeuticsIntellia
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 X (formerly known as Twitter) @intelliatx.
Forward-Looking StatementsThis 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, success and advancement of its
pipeline of investigational gene editing therapies, including its
clinical program for NTLA-2001 for the treatment of transthyretin
(ATTR) amyloidosis with cardiomyopathy pursuant to its clinical
trial applications and investigational new drug application; the
initiation, enrollment, dosing and completion of its clinical
trials, including its ability to initiate a global Phase 3 study of
NTLA-2001 by year-end 2023.
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, enrollment and conduct of
studies and other development requirements for its product
candidates, including NTLA-2001; 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, such as its clinical studies of
NTLA-2001, will not be predictive of future results of future
studies for the same product candidate or Intellia’s other product
candidates; and risks related to Intellia’s reliance on
collaborations, including that its collaboration with Regeneron
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
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
Communications lina.li@intelliatx.com
Media:Matt CrensonTen Bridge
Communicationsmedia@intelliatx.commcrenson@tenbridgecommunications.com
Intellia Therapeutics (NASDAQ:NTLA)
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