Intellia Therapeutics to Present Updated Data from Phase 1/2 Study of NTLA-2002 for the Treatment of Hereditary Angioedema (HAE) at the EAACI Congress 2024
April 29 2024 - 7:30AM
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage
gene editing company focused on revolutionizing medicine with
CRISPR-based therapies, today announced the acceptance of an oral
presentation from the Phase 1 portion of the ongoing NTLA-2002
Phase 1/2 study at the European Academy of Allergy and Clinical
Immunology (EAACI) Congress 2024, taking place May 31 – June 3 in
Valencia, Spain. 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. The
presentation will include updated safety and efficacy results from
the Phase 1 study across all three dose cohorts (25 mg, 50 mg and
75 mg).
Presentation Details
Title: CRISPR-based gene editing of KLKB1
resulted in long-term plasma kallikrein protein reduction and
decreased attack rate in patients with hereditary angioedema:
Updated results from a phase 1 study
Session: Breakthroughs in management of
Hereditary Angioedema
Date and Time: Sunday, June 2, 2024, from 8:30
– 10:00 a.m. CET
Presenter: Hilary Longhurst, M.D., Ph.D., FRCP,
FRCPath, Senior Medical Officer at Auckland District Health
Board and Honorary Associate Professor at University
of Auckland, New Zealand, the trial’s principal investigator
in New Zealand
Intellia Therapeutics Investor Webcast
Intellia will host a live webcast on Monday, June 3, 2024, at
8:00 a.m. ET to review the new data. To join the webcast, please
visit this link, or the Events and Presentations page of the
Investors & Media section of the company’s website at
www.intelliatx.com. A replay of the webcast will be available on
Intellia’s website for at least 30 days following the call.
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 pursuant to its clinical trial
applications and investigational new drug application, including
its ability to initiate the global, pivotal Phase 3 study in the
second half of 2024, subject to regulatory feedback and the
potential of NTLA-2002 to become the first one-time treatment for
hereditary angioedema.
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; and
uncertainties related to the authorization, initiation, enrollment
and conduct of studies and other development requirements for its
product candidates, including NTLA-2002. 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
Communicationsian.karp@intelliatx.com
Lina LiSenior Director, Investor Relations and Corporate
Communicationslina.li@intelliatx.com
Media:Matt CrensonTen Bridge
Communicationsmedia@intelliatx.com
mcrenson@tenbridgecommunications.com
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