Intellia Therapeutics Announces Publication of Positive Interim Phase 1 Data for NTLA-2002 in Patients with Hereditary Angioedema in the New England Journal of Medicine
31 Janeiro 2024 - 7:00PM
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage
gene editing company focused on revolutionizing medicine with
CRISPR-based therapies, today announced that interim results from
the Phase 1 portion of the Phase 1/2 study of NTLA-2002 were
published online in the New England Journal of
Medicine (NEJM). NTLA-2002 is an investigational in vivo
CRISPR-based gene editing therapy in development as a single-dose
treatment for hereditary angioedema (HAE), a rare genetic condition
that leads to potentially life-threatening swelling attacks.
“Despite currently available treatments, people living with
hereditary angioedema continue to face frequent anxiety about their
next swelling attack. The interim NTLA-2002 clinical data published
suggest that a single dose of NTLA-2002 may eliminate angioedema
attacks for people suffering from hereditary angioedema,” said
Intellia President and Chief Executive Officer John Leonard,
M.D. “We are highly encouraged by these data and look forward to
presenting extended follow-up from the Phase 1 and results from the
Phase 2 portion later this year. Additionally, we remain on track
to initiate a global pivotal study for NTLA-2002 in the second half
of 2024, subject to regulatory feedback. This marks the second
consecutive Intellia in vivo CRISPR-based program to have its
initial clinical data published in the New England Journal of
Medicine, further supporting the immense potential impact our
proprietary gene editing platform could have on the future of human
health.”
The reported data showed that a single dose of NTLA-2002 led to
a 95% mean reduction in monthly HAE attack rate across all 10
patients in the Phase 1 portion. Nine out of 10 patients remained
completely attack-free following the 16-week primary observation
period through the latest follow-up. Further, all patients who
discontinued concomitant long-term HAE prophylaxis treatment after
NTLA-2002 administration (n=6) have reported no HAE attacks since
discontinuation. NTLA-2002 has been well tolerated at all dose
levels. The most frequent adverse events reported were mild,
transient infusion-related reactions and fatigue. The data were
previously shared in a late-breaking presentation at the 2023
European Academy of Allergy and Clinical Immunology Hybrid
Congress.
About the NTLA-2002 Clinical ProgramIntellia’s
ongoing Phase 1/2 study is evaluating the safety and activity of
NTLA-2002 in adults with Type I or Type II hereditary angioedema
(HAE). The Phase 1/2 is an international, open-label study designed
to identify a dose level of NTLA-2002 for further evaluation in a
Phase 3 study. Enrollment of the Phase 1/2 is complete. Intellia
plans to initiate the global, pivotal Phase 3 study in the second
half of 2024, subject to regulatory feedback.
Visit clinicaltrials.gov (NCT05120830) for more
details.
About NTLA-2002Based on Nobel-prize winning
CRISPR/Cas9 technology, NTLA-2002 has the potential to become the
first one-time treatment for hereditary angioedema (HAE). NTLA-2002
is designed to prevent HAE attacks by inactivating the kallikrein
B1 (KLKB1) gene, which encodes for prekallikrein, the kallikrein
precursor protein. Interim Phase 1 clinical data showed dramatic
reductions in attack rate, as well as consistent, deep and durable
reductions in kallikrein levels. NTLA-2002 has received five
notable regulatory designations, including Orphan Drug and RMAT
Designation by the U.S. Food and Drug Administration, the
Innovation Passport by the U.K. Medicines and Healthcare products
Regulatory Agency (MHRA), Priority Medicines (PRIME) Designation by
the European Medicines Agency, as well as Orphan Drug Designation
by the European Commission.
About Hereditary Angioedema Hereditary
angioedema (HAE) is a rare, genetic disease 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. Although there is no known cure for HAE, there are
preventative and on-demand treatment options to help manage the
condition, including long- and short-term prophylaxis used to
prevent swelling attacks. 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, Inc. (NASDAQ:NTLA) is a leading clinical-stage gene
editing company focused on revolutionizing medicine with
CRISPR-based therapies. The company’s in vivo programs
use CRISPR to enable precise editing of disease-causing genes
directly inside the human body. Intellia’s ex
vivo programs use CRISPR to engineer human cells outside the
body for the treatment of cancer and autoimmune diseases.
Intellia’s deep scientific, technical and clinical development
experience, along with its people, is helping set the standard for
a new class of medicine. To harness the full potential of gene
editing, Intellia continues to expand the capabilities of its
CRISPR-based platform with novel editing and delivery technologies.
Learn more at intelliatx.com and follow us @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
the initiation of a global pivotal study for NTLA-2002 in the
second half of 2024 subject to regulatory feedback, the potential
for NTLA-2002 to eliminate angioedema attacks after a single dose
in people living with HAE, and the expected timing of future data
releases such as the presentation of extended follow-up data from
the Phase 1 portion and results from the Phase 2 portion of the
Phase 1/2 study later this year; and the potential impact its
proprietary gene editing platform could have on the future of human
health.
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
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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