Intellia Therapeutics Receives Authorization to Initiate Phase 1/2 Clinical Trial of NTLA-2002 for the Treatment of Hereditary Angioedema
06 Octobre 2021 - 1:30PM
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage
genome editing company focused on developing curative therapeutics
using CRISPR/Cas9 technology both in vivo and ex
vivo, today announced the authorization of its Clinical Trial
Application (CTA) by the New Zealand Medicines and Medical Devices
Safety Authority (MEDSAFE) to initiate a Phase 1/2 study evaluating
NTLA-2002 for the treatment of adults with hereditary angioedema
(HAE). HAE is a 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. NTLA-2002 is a systemically administered
single-dose CRISPR/Cas9-based therapeutic candidate designed to
inactivate the target gene Kallikrein B1 (KLKB1) to permanently
reduce plasma kallikrein activity and thus prevent HAE attacks.
“We look forward to initiating this year our first-in-human
study of NTLA-2002 for people living with HAE, a debilitating
disorder that causes frequent, potentially life-threatening
attacks,” said Intellia President and Chief Executive Officer John
Leonard, M.D. “We believe NTLA-2002 has the potential to be a
curative therapy for patients with HAE by providing continuous
suppression of plasma kallikrein activity following a single dose
and eliminating the significant treatment burden associated with
currently available HAE therapies. This study of NTLA-2002
leverages early insights from our ATTR amyloidosis program, where
we established proof-of-concept for our modular in vivo genome
editing platform with interim Phase 1 data earlier this year. The
NTLA-2002 program represents the second systemic in vivo CRISPR
genome editing therapy candidate to enter human clinical
trials.”
The Phase 1/2 study will evaluate the safety, tolerability,
pharmacokinetics and pharmacodynamics of NTLA-2002 in adults with
Type I or Type II HAE. This includes the measurement of 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. More information about the study will be available at
clinicaltrials.gov.
Beyond New Zealand, Intellia is submitting additional regulatory
applications in other countries as part of its ongoing,
multi-national development approach for NTLA-2002.
About Hereditary Angioedema
Hereditary 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 NTLA-2002
Based on Nobel Prize-winning CRISPR/Cas9 technology, NTLA-2002
is potentially the first single-dose treatment 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 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, leveraging proprietary delivery
technology to enable 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 initiate clinical studies
for NTLA-2002 for the treatment of hereditary angioedema (“HAE”)
pursuant to its clinical trial applications (“CTA”), including
enrollment of a first patient by the end of 2021, 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 transthyretin
amyloidosis and HAE 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; statements
regarding the timing of regulatory filings regarding its
development programs; use of capital, expenses, future accumulated
deficit and other 2021 financial results or in the future; and
ability to fund operations beyond 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:
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:
Lisa QuTen Bridge
Communications+1-678-662-9166media@intelliatx.comlqu@tenbridgecommunications.com
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