Intellia Therapeutics Announces FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to NTLA-2002 for the Treatment of Hereditary Angioedema
21 Mars 2023 - 12:30PM
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) granted
Regenerative Medicine Advanced Therapy (RMAT) designation to
NTLA-2002 for the treatment of hereditary angioedema (HAE).
NTLA-2002 is an in vivo CRISPR-based investigational therapy
designed to inactivate the target gene, kallikrein B1 (KLKB1), to
potentially prevent life-threatening swelling attacks in people
with HAE.
“The RMAT designation is important recognition for our early
clinical data. It indicates that a single dose of NTLA-2002 has the
potential to address serious unmet medical need for people living
with hereditary angioedema,” said Intellia President and Chief
Executive Officer John Leonard, M.D. “We look forward to continuing
our productive dialogue with the FDA to accelerate the development
of NTLA-2002, an investigational in vivo CRISPR-based therapy, with
the goal of bringing forth a potentially transformative treatment
to patients more quickly.”
The RMAT designation was established under the 21st Century
Cures Act to expedite the development and review of promising
therapeutic candidates, including genetic therapies, which are
intended to treat, modify, reverse or cure a serious or
life-threatening disease. RMAT designation includes benefits, such
as early interactions with the FDA, including discussions on
surrogate or intermediate endpoints that could potentially support
accelerated approval and satisfy post-approval requirements, and
potential priority review of a product’s biologics license
application (BLA).
The RMAT is the third special regulatory designation received by
Intellia for NTLA-2002. NTLA-2002 was also granted Orphan Drug
Designation by the FDA and the Innovation Passport by the U.K.
Medicines and Healthcare products Regulatory Agency.
About the NTLA-2002 Clinical
ProgramIntellia’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 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. This Phase 1/2 study will identify the dose
of NTLA-2002 for use in future studies. In 2022, Intellia reported
positive interim results from the Phase 1 study demonstrating deep,
dose-dependent reductions in plasma kallikrein and robust
reductions in patient HAE attacks. Patient screening in the Phase 2
portion of the study is ongoing. 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 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 @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 pursuant to
its clinical trial applications and investigational new drug
application, including its ability to accelerate the development of
NTLA-2002 pursuant to its Regenerative Medicine Advanced Therapy
designation; and the expected timing of regulatory filings and the
initiation, enrollment, dosing and completion of clinical trials
for NTLA-2002.
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 any one or more of
Intellia’s product candidates will not be successfully developed
and commercialized; and the risk that the results of preclinical
studies or clinical studies, such as the 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 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 LiSenior Director, Investor Relations and Corporate
Communications +1-857-706-1612lina.li@intelliatx.com
Media:Rebecca Spalding Ten Bridge
Communications+1-646-509-3831media@intelliatx.comrebecca@tenbridgecommunications.com
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