Intellia Therapeutics Receives U.S. FDA Orphan Drug Designation for NTLA-2002, an Investigational CRISPR Therapy for the Treatment of Hereditary Angioedema
01 Septembre 2022 - 10:01PM
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 granted orphan
drug designation for Intellia’s in vivo CRISPR/Cas9 genome editing
candidate, NTLA-2002, for the treatment of hereditary angioedema
(HAE).
NTLA-2002 is a systemically administered investigational therapy
designed to knockout the target gene kallikrein B1 (KLKB1) to
reduce plasma kallikrein activity and thus prevent HAE attacks.
NTLA-2002 is currently being evaluated in a Phase 1/2 study in
adults with Type I or Type II HAE.
“Orphan drug designation represents an important milestone in
the development of NTLA-2002 and underscores the importance of
developing innovative, new treatment options for people living with
HAE,” said Intellia President and Chief Executive Officer John
Leonard, M.D. “We hope to demonstrate in our ongoing clinical trial
that NTLA-2002 can result in deep and sustained kallikrein activity
reduction following a single dose, and potentially prevent the
unpredictable swelling attacks caused by this genetic disease. We
look forward to presenting interim data on September 16 at the 2022
Bradykinin Symposium, including safety, kallikrein reduction and
HAE attack rate data.”
The FDA’s Orphan Drug Designation Program provides orphan status
to drugs intended for the treatment, diagnosis or prevention of
rare diseases that affect fewer than 200,000 people in the United
States. Orphan drug designation qualifies the sponsor of the drug
for certain development incentives, including tax credits for
qualified clinical testing, prescription drug user-fee exemptions
and seven-year marketing exclusivity upon FDA approval.
About the NTLA-2002 Clinical Program
Intellia’s multi-national Phase 1/2 study is evaluating the
safety, tolerability, pharmacokinetics and pharmacodynamics of
NTLA-2002 in adults with Type I or Type II hereditary angioedema
(HAE). This includes the measurement of plasma kallikrein protein
levels and activity as determined by HAE attack rate measures. The
Phase 1 portion of the study is an open-label, single-ascending
dose design used to identify up to two dose levels of NTLA-2002
that will be further evaluated in the randomized,
placebo-controlled Phase 2 portion of the study. This Phase 1/2
study will identify the dose of NTLA-2002 for use in future
studies. Visit clinicaltrials.gov (NCT05120830) for more
details.
About NTLA-2002
Based on Nobel Prize-winning CRISPR/Cas9 technology, NTLA-2002
is the first single-dose investigational treatment being explored
in clinical trials for the potential to continuously reduce
kallikrein activity and prevent attacks in people living with
hereditary angioedema (HAE). NTLA-2002 is a wholly owned
investigational CRISPR therapeutic candidate designed to inactivate
the kallikrein B1 (KLKB1) gene, which encodes for
prekallikrein, the kallikrein precursor protein. NTLA-2002 is
Intellia’s second investigational CRISPR therapeutic candidate
to be administered systemically, by intravenous infusion, to edit
disease-causing genes inside the human body with a single dose of
treatment. 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
together carry out the precision editing.
About Hereditary AngioedemaHereditary
angioedema (HAE) is a rare, genetic disorder characterized by
severe, recurring and unpredictable inflammatory attacks in various
organs and tissues of the body, which can be painful, debilitating
and life-threatening. It is estimated that one in 50,000 people are
affected by HAE, and current treatment options often include
life-long therapies, which may require chronic intravenous (IV) or
subcutaneous (SC) administration as often as twice per week, or
daily oral administration to ensure constant pathway suppression
for disease control. Despite chronic administration, breakthrough
attacks still occur. Kallikrein inhibition is a clinically
validated strategy for the preventive treatment of HAE attacks.
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 Twitter @intelliatx.
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 ability to conduct and complete clinical studies for
NTLA-2002 for the treatment of hereditary angioedema (HAE); its
ability to generate data to demonstrate NTLA-2002 as a potential
single-dose treatment for HAE, including safety, kallikrein
reduction and attack rate data; its ability to develop its modular
platform and full-spectrum approach to advance its complex genome
editing capabilities, including to apply its proprietary
CRISPR/Cas9 technology platform to additional product candidates;
the advancement and expansion of its CRISPR/Cas9 technology to
develop human therapeutic products; its ability to maintain and
expand its related intellectual property portfolio, and avoid or
acquire rights to valid intellectual property of third parties; its
ability to demonstrate its platform’s modularity and replicate or
apply results achieved in preclinical studies, including those in
its NTLA-2002 program, in any future studies, including human
clinical trials; its ability to develop other in vivo or ex vivo
cell therapeutics of all types, and NTLA-2002 in particular, using
CRISPR/Cas9 technology; and the timing of regulatory filings and
clinical trial execution, including dosing of patients.
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 the successful enrollment of patients in the Phase 1/2
study for NTLA-2002 for the treatment of HAE; risks related to
Intellia’s ability to protect and maintain its intellectual
property position; risks related to the authorization, initiation
and conduct of studies and other development requirements,
including manufacturing, for its in vivo and ex vivo product
candidates, including NTLA-2002; the risk that any one or more of
Intellia’s product candidates, including NTLA-2002, will not be
successfully developed and commercialized; the risk that the
results of preclinical studies or clinical studies, including for
NTLA-2002, will not be predictive of future results in connection
with future studies; and the risk that Intellia’s will not be able
to demonstrate its platform’s modularity and replicate or apply
results achieved in preclinical studies to develop additional
product candidates, including to apply its proprietary CRISPR/Cas9
technology platform successfully to additional product candidates.
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 and quarterly report of 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 (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.comLina LiSenior
Director, Investor Relations and Corporate
Communications+1-857-706-1612lina.li@intelliatx.com
Media:Rebecca SpaldingTen Bridge
Communications+1-646-509-3831media@intelliatx.com
rebecca@tenbridgecommunications.com
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