Intellia Therapeutics Receives Authorization to Initiate Phase 1/2 Clinical Trial of NTLA-3001 for the Treatment of Alpha-1 Antitrypsin Deficiency
30 July 2024 - 9:30PM
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage
gene editing company focused on revolutionizing medicine with
CRISPR-based therapies, today announced the authorization of its
Clinical Trial Application (CTA) by the United Kingdom’s Medicine
and Healthcare products Regulatory Agency (MHRA) to initiate a
Phase 1/2 study evaluating NTLA-3001 for the treatment of alpha-1
antitrypsin deficiency (AATD)-associated lung disease. AATD is a
rare, genetic disease that most commonly manifests in lung
dysfunction due to insufficient levels of alpha-1 antitrypsin (AAT)
protein. NTLA-3001 is a systemically administered in vivo
CRISPR/Cas9-based targeted gene insertion candidate. It is designed
to precisely insert a healthy copy of the SERPINA1 gene, which
encodes the AAT protein, with the potential to restore permanent
expression of functional AAT protein to therapeutic levels after a
single dose. This approach seeks to improve patient outcomes,
including eliminating the need for weekly intravenous infusions of
AAT augmentation therapy or lung transplant in severe cases.
“NTLA-3001 is a groundbreaking in vivo CRISPR-based gene
insertion candidate designed to durably produce functional AAT
protein at normal levels after a one-time treatment. We are excited
to receive regulatory authorization to begin this important
first-in-human study of NTLA-3001 for people living with AATD,”
said Intellia President and Chief Executive Officer John Leonard,
M.D. “In addition, this study serves to validate our modular gene
insertion platform, which we plan to leverage to address numerous
diseases caused by a missing or defective protein.”
The Phase 1/2 study will be an international, multicenter,
single-arm, open-label study of NTLA-3001 in adults with
AATD-associated lung disease. The study will enroll up to 30
patients and consist of a dose-escalation phase, followed by a
dose-expansion phase to confirm the recommended dose. The study
will evaluate the safety, tolerability, pharmacokinetics and
pharmacodynamics of NTLA-3001. More information about the study may
be found on clinicaltrials.gov when available.
Beyond its first application in the United Kingdom, Intellia is
submitting additional regulatory applications in other countries as
part of its ongoing, multi-national development strategy for
NTLA-3001.
About NTLA-3001
NTLA-3001 is a wholly owned, first-in-class CRISPR-mediated in
vivo targeted gene insertion development candidate for the
treatment of AATD-associated lung disease. It is designed to
precisely insert a copy of the SERPINA1 gene, which encodes the
alpha-1 antitrypsin (AAT) protein, with the potential to restore
permanent expression of functional AAT protein to therapeutic
levels after a single dose. This approach seeks to improve patient
outcomes, including eliminating the need for weekly intravenous
infusions of AAT augmentation therapy or lung transplant in severe
cases.
About Alpha-1 Antitrypsin Deficiency
Alpha-1 antitrypsin deficiency (AATD) is an inherited condition
that increases the risk of liver and lung disease. AATD is caused
by changes in the SERPINA1 gene that normally provides instructions
for making alpha-1 antitrypsin (AAT) protein in the liver that is
then secreted to protect the lungs. Mutations to the SERPINA1 gene
lead to the production of abnormal AAT protein that then
accumulates in the liver. As a result, AAT protein levels in the
blood and lungs are very low. The shortage of AAT in the blood and
lungs places the lungs at risk for emphysema, a type of chronic
obstructive pulmonary disease (COPD). AATD occurs in greater than
60,000 people in the U.S. and around 250,000 worldwide.
About Intellia Therapeutics
Intellia 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 the safety, efficacy, success and advancement of
NTLA-3001 for the treatment of alpha-1 antitrypsin deficiency
(“AATD”)-associated lung disease pursuant to its clinical trial
applications (“CTA”), including its ability to dose the first
patient in its Phase 1/2 study in the second half of 2024 and the
submission of regulatory applications in other countries; the
ability of NTLA-3001 to durably normalize AAT protein levels after
a single dose, halt the progression of lung disease associated with
AATD, and improve patient outcomes; and the modularity of its gene
insertion platform, including its ability to address numerous
diseases caused by a missing or defective protein.
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,
including uncertainties related to regulatory approvals to conduct
clinical trials; 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 clinical study
results will not be positive. 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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