Intellia Therapeutics Receives European Union Orphan Drug Designation for NTLA-2002, an Investigational In Vivo CRISPR Genome Editing Treatment for Hereditary Angioedema
14 Novembro 2023 - 9:30AM
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage
genome editing company focused on developing potentially curative
single-dose therapeutics leveraging CRISPR-based technologies,
today announced that the European Commission (EC) has granted
orphan drug designation to NTLA-2002 for the treatment of
hereditary angioedema (HAE). NTLA-2002 is an in vivo CRISPR-based
investigational therapy designed to prevent potentially
life-threatening swelling attacks in people with HAE.
“The European Union orphan drug designation for NTLA-2002
represents another important milestone for Intellia as we continue
to make rapid progress in the development of a novel, potential
one-time treatment for people with hereditary angioedema,” said
Intellia President and Chief Executive Officer John Leonard,
M.D. “We are on track to complete enrollment of the Phase 2 portion
of the study in the coming weeks, which will bring us one step
closer to our goal of delivering a potentially life-changing
treatment for people who suffer from this serious and debilitating
disease.”
Orphan drug designation in the European Union (EU) is granted by
the EC based on a positive opinion issued by the European Medicines
Agency (EMA) Committee for Orphan Medicinal Products. To qualify
for orphan drug designation, a candidate therapy must be intended
for the treatment, prevention or diagnosis of a life-threatening or
chronically debilitating disease that occurs in not more than five
in 10,000 people in the EU. The designation provides regulatory,
financial and commercial incentives to develop therapies for rare
diseases where there are either no satisfactory treatment options
or significant benefit to those affected by the disease.
Intellia has received five special regulatory designations for
NTLA-2002. NTLA-2002 was also granted Orphan Drug Designation and
RMAT Designation by the U.S. Food and Drug Administration (FDA),
the Innovation Passport by the U.K. Medicines and Healthcare
products Regulatory Agency (MHRA), as well as PRIME designation by
the EMA.
About the NTLA-2002 Clinical ProgramIntellia’s
global Phase 1/2 study is evaluating the safety, tolerability,
activity, 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 well as HAE attack rate. The Phase 1 portion of the
study is an open-label, single-ascending dose design used to
identify two dose levels of NTLA-2002 for further evaluation in the
Phase 2, randomized, placebo-controlled portion of the study. The
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-2002Based 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 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 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 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: the safety, efficacy, success and
advancement of its clinical program for NTLA-2002 for the treatment
of hereditary angioedema (“HAE”) pursuant to its clinical trial
applications and investigational new drug application, including
its ability to complete enrollment of the Phase 2 portion of the
study in the coming weeks, and its ability to deliver a potentially
life-changing treatment for people with HAE.
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-2002; the risk that NTLA-2002 will not
be successfully developed and commercialized; and the risk that the
results of preclinical studies or clinical studies, such as the
Phase 1/2 clinical study of NTLA-2002, will not be predictive of
future results in connection with future studies for the same
product candidate or Intellia’s other 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 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
Communicationslina.li@intelliatx.com
Media:Matt CrensonTen Bridge
Communicationsmedia@intelliatx.com
TBCIntellia@tenbridgecommunications.com
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