Intellia Therapeutics, Inc. (NASDAQ:NTLA), announced that the first
patient has been treated with NTLA-2001, which the company is
developing as a single-course, potentially curative therapy for
transthyretin amyloidosis (ATTR). Intellia’s global Phase 1 study
is to evaluate NTLA-2001 for hereditary ATTR with polyneuropathy
(hATTR-PN). Following safety assessment and dose optimization,
Intellia intends to further evaluate NTLA-2001 in a broader ATTR
patient population of both polyneuropathy and cardiomyopathy
patients.
“With today’s news, we’re entering a new era of potential genome
editing cures for patients,” said Intellia’s President and Chief
Executive Officer John Leonard, M.D. “Once we’ve assessed safety
and established an optimal dose, we intend to rapidly initiate
trials for the clinical manifestations of ATTR. NTLA-2001 may halt
and reverse ATTR progression by producing a deeper, permanent TTR
protein reduction for all patients – regardless of disease type –
than the chronically administered treatments currently
available.”
“Only a few short years ago, there were no treatments available
for this devastating disease,” said Carlos Heras-Palou, M.D.,
founder and president of the United Kingdom ATTR Amyloidosis
Patients Association (UKATPA), who also has hATTR. “Now, a cure for
ATTR utilizing the groundbreaking CRISPR/Cas9 gene editing
technology may be within reach.”
About the NTLA-2001 Clinical
ProgramIntellia’s global Phase 1 trial is an open-label,
multi-center, two-part study of NTLA-2001 in adults with hereditary
transthyretin amyloidosis with polyneuropathy (hATTR-PN). 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 participants (ages 18-80 years) and
consist of a single-ascending dose phase in Part 1 and, following
the identification of an optimal dose, an expansion cohort in Part
2. Visit clinicaltrials.gov (NCT04601051) for more details.
Enrollment is ongoing at a clinical trial site in the U.K.
Intellia is submitting additional regulatory applications in other
countries as part of its ongoing, global development strategy. The
company is planning to rapidly move to pivotal studies enrolling
both polyneuropathy and cardiomyopathy patients.
About NTLA-2001Based on Nobel
Prize-winning CRISPR/Cas9 technology, NTLA-2001 could potentially
be the first curative treatment for ATTR. NTLA-2001 is the first
experimental CRISPR therapy to be administered systemically, or
through a vein, to edit genes inside the human body. Intellia’s
proprietary non-viral platform utilizes lipid nanoparticles
designed to deliver to the liver a simple, two-part genome
editing system: guide RNA specific to the disease-causing
gene and messenger RNA that encodes the Cas9 protein. Our
robust preclinical data showing deep and long-lasting transthyretin
(TTR) reduction following knockout of the target gene in vivo
support NTLA-2001’s potential as a single course of treatment.
NTLA-2001 is part of a co-development/co-promotion agreement
between Intellia, the lead development and commercialization party,
and Regeneron Pharmaceuticals, Inc.
About Transthyretin Amyloidosis
(ATTR)Transthyretin amyloidosis, or ATTR, is a rare,
progressive and fatal disease. Hereditary ATTR (hATTR) occurs when
a person is born with DNA mutations in the TTR gene, which causes
the liver to produce a protein called transthyretin (TTR) in a
misfolded form and build up in the body. hATTR can predominantly
manifest as polyneuropathy (hATTR-PN), which can lead to nerve
damage, and cardiomyopathy (hATTR-CM), which involves heart muscle
disease that can lead to heart failure. In addition, non-mutated,
or wild-type TTR protein, can also accumulate in the body, leading
to wild-type ATTR (wtATTR). There are an estimated 50,000 hATTR
patients worldwide and between 200,000 and 500,000 people with
wtATTR.
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 Therapeutics 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” 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”) and submitting similar 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; ability to demonstrate its platform’s modularity and
replicate or apply results achieved in preclinical studies,
including those in its ATTR, HAE, hemophilia A and hemophilia B
programs, in any future studies, including human clinical trials;
expectations of the potential impact of the coronavirus disease
2019 pandemic on strategy, future operations and timing of its
clinical trials or 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, hemophilia A and hemophilia B;
statements regarding the timing of regulatory filings regarding its
development programs; use of capital, expenses, future accumulated
deficit and other 2020 financial results or in the future; and
ability to fund operations at least through 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
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. 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:
Media:Jennifer Mound SmoterSenior Vice
President
External Affairs &
Communications
+1 857-706-1071
jenn.smoter@intelliatx.com
Lynnea OlivarezDirectorExternal Affairs & Communications+1
956-330-1917lynnea.olivarez@intelliatx.com
Investors:Lina LiAssociate DirectorInvestor
Relations+1 857-706-1612lina.li@intelliatx.com
Photos accompanying this announcement are available
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Videos accompanying this announcement are available
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