Intellia Therapeutics, Inc. (NASDAQ:NTLA) announced today
that the United Kingdom Medicines and Healthcare products
Regulatory Agency (MHRA) has approved a protocol amendment for the
Company’s ongoing Phase 1 study of NTLA-2001 to include patients
with ATTR amyloidosis with cardiomyopathy (ATTR-CM). The study of
NTLA-2001 now includes patients with ATTR-CM to be enrolled in new
dose-escalation and expansion cohorts. The inclusion of the ATTR-CM
patient population is in addition to the original Phase 1 study
population, which is currently evaluating NTLA-2001 in patients
with ATTR amyloidosis with polyneuropathy (ATTRv-PN).
The first investigational therapy of its kind, NTLA-2001 is an
in vivo CRISPR/Cas9-based genome editing candidate being developed
as a single-dose treatment for transthyretin (ATTR) amyloidosis. It
is designed to inactivate the TTR gene in liver cells to
prevent the production of transthyretin (TTR) protein and is being
developed to potentially be the first single-dose ATTR amyloidosis
treatment to not only halt but also reverse disease progression in
both ATTRv-PN and ATTR-CM patients. NTLA-2001 has received orphan
drug designation for the treatment of ATTR amyloidosis by both the
European Commission and the U.S. FDA.
“ATTR amyloidosis is a chronic, fatal disease that can impact
different organs and tissues within the body, often manifesting as
either polyneuropathy or cardiomyopathy. At Intellia, our goal is
to develop a potentially curative treatment that could benefit as
many patients living with this disease as possible,” said Intellia
President and Chief Executive Officer John Leonard, M.D. “We have
already seen promising interim data supporting the ability of
NTLA-2001 to significantly reduce serum TTR levels in ATTR patients
with polyneuropathy. We are excited to now expand our Phase 1 study
of NTLA-2001 to include ATTR patients with cardiomyopathy in order
to advance this potentially first-of-its-kind, single-dose
treatment for more patients.”
The protocol amendment to the Phase 1 study allows for
enrollment of up to 36 adults in the United Kingdom with either
hereditary ATTR-CM (ATTRv-CM) or wild-type cardiomyopathy
(ATTRwt-CM), and New York Heart Association Class I – III heart
failure, across the two-part study. The trial’s primary objectives
are to assess the safety, tolerability, pharmacokinetics and
pharmacodynamics of NTLA-2001, which will include the measurement
of serum TTR levels following a single intravenous infusion. The
secondary objectives are to evaluate the efficacy of NTLA-2001 on
clinical measures of cardiac disease in ATTR-CM patients.
In June 2021, Intellia and its collaborator Regeneron announced
positive interim clinical results from the first two cohorts of
this study. These results, which were subsequently published in the
New England Journal of Medicine, represented the first-ever
clinical data supporting in vivo CRISPR genome editing in
humans.
About NTLA-2001Based on Nobel
Prize-winning CRISPR/Cas9 technology, NTLA-2001 could potentially
be the first curative treatment for ATTR amyloidosis. NTLA-2001 is
the first investigational CRISPR therapy candidate to be
administered systemically, or intravenously, 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 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. Interim Phase 1
clinical data released in June 2021 demonstrated substantial,
dose-dependent reduction of TTR protein following a single dose of
NTLA-2001. Intellia leads development and commercialization of
NTLA-2001 as part of a multi-target discovery, development and
commercialization collaboration with Regeneron.
About the NTLA-2001 Clinical ProgramThe
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 cardiomyopathy (ATTR-CM). The
trial’s primary objectives are to assess the safety, tolerability,
pharmacokinetics and pharmacodynamics of NTLA-2001. Patients
receive a single dose of NTLA-2001 via intravenous administration.
The study will enroll up to 38 ATTRv-PN participants (ages 18-80
years) and up to 36 ATTR-CM participants (ages 18-90 years) and
consists of a single-ascending dose phase followed by a
dose-expansion phase.
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 Therapeutics
Intellia Therapeutics, a leading clinical-stage genome editing
company, is developing novel, potentially curative therapeutics
using CRISPR/Cas9 technology. To fully realize the transformative
potential of CRISPR/Cas9, 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 CRISPR/Cas9 to create new classes of genetic medicine. Learn
more at intelliatx.com. 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: being able to complete clinical studies
for NTLA-2001 for the treatment of transthyretin amyloidosis
(“ATTR”) pursuant to its clinical trial applications (“CTA”),
including submitting additional regulatory applications in other
countries; advancement and expansion of its CRISPR/Cas9 technology
to develop human therapeutic products, as well as its ability to
maintain and expand its related intellectual property portfolio;
expectations of the potential impact of the coronavirus disease
2019 pandemic on strategy, future operations and timing of its
clinical trials or CTA and IND submissions; ability to optimize the
impact of its collaborations on its development programs, including
but not limited to its collaborations with Regeneron, including its
co-development programs for ATTR amyloidosis; and statements
regarding the timing of regulatory filings regarding its
development programs.
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 and conduct of studies and
other development requirements for its product candidates; 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 will not be
predictive of future results in connection with future studies; and
the risk that Intellia’s collaborations with Regeneron or its other
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:Ian KarpSenior Vice President,
Investor Relations and Corporate
Communications+1-857-449-4175ian.karp@intelliatx.com
Lina LiDirector, Investor
Relations+1-857-706-1612lina.li@intelliatx.com
Media:Matt CrensonTen Bridge
Communications+1-917-640-7930media@intelliatx.commcrenson@tenbridgecommunications.com
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