Intellia Therapeutics Announces First Patient Dosed in Phase 1/2a Clinical Trial of NTLA-5001 for the Treatment of Acute Myeloid Leukemia
01 Mars 2022 - 1:30PM
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage
genome editing company focused on developing potentially curative
therapeutics leveraging CRISPR-based technologies, today announced
that the first patient has been dosed with NTLA-5001, the company’s
ex vivo CRISPR/Cas9 genome editing candidate for the treatment of
acute myeloid leukemia (AML). NTLA-5001 is an autologous T cell
receptor (TCR)-T cell therapy designed to target the Wilms’ Tumor
(WT1) antigen, which is found in AML and many other hematologic and
solid tumors. It is the company’s first ex vivo candidate developed
using Intellia’s advanced lipid nanoparticle cell engineering
platform, designed to improve cell performance as compared to
traditional ex vivo delivery technologies.
“As Intellia’s first wholly-owned ex vivo candidate to be dosed
in a patient, this NTLA-5001 milestone represents a significant
step forward in our full-spectrum approach to genome editing,” said
Intellia President and Chief Executive Officer John Leonard, M.D.
“AML is the most common type of acute leukemia in adults, where
despite recent advancements, a significant therapeutic need still
exists. We look forward to advancing this investigational
engineered cell therapy as a treatment for people living with this
aggressive cancer of the blood and bone marrow.”
About the NTLA-5001 Clinical Program
The Phase 1/2a study will evaluate the safety, tolerability,
cell kinetics and anti-tumor activity of a single dose of NTLA-5001
in adults who have detectable AML after having received standard
first-line therapy. The study includes a dose escalation and
expansion phase, with up to 54 total participants. The
dose-escalation phase of the study includes two independent arms of
up to three cohorts each: Arm 1 consists of adults with AML with
lower disease burden, defined as those with less than 5% blasts in
bone marrow, while Arm 2 consists of adults with AML with higher
disease burden, defined as those with greater than or equal to 5%
blasts in bone marrow. Once a dose is identified in each arm, two
expansion cohorts will be opened for further safety assessment.
Visit clinicaltrials.gov (NCT05066165) for more details.
About NTLA-5001
NTLA-5001 is an investigational CRISPR/Cas9-engineered T cell
receptor (TCR)-T cell therapy in development for the treatment of
all genetic subtypes of acute myeloid leukemia (AML). This
autologous cell therapy candidate is designed for AML patients with
the HLA-A*02:01 allele and whose tumors carry the Wilms’ Tumor 1
(WT1) antigen, which is widely overexpressed in AML and other
cancers. NTLA-5001 is Intellia’s first wholly owned ex vivo
therapeutic candidate, developed using its proprietary cell
engineering platform for the treatment of cancer. Based on
preclinical results, Intellia believes its proprietary cell
engineering platform will result in a pipeline of more efficacious
and safer cell-based cancer therapies.
About Acute Myeloid Leukemia
Acute myeloid leukemia (AML) is a cancer of the blood and bone
marrow that is rapidly fatal without immediate treatment. It is the
most common type of acute leukemia in adults in the U.S., with more
than 20,000 estimated new cases in 2021. Despite currently
available treatments for AML, the five-year overall survival rate
for patients remains less than 30%. AML, along with other cancer
types, is often characterized by overexpression of the Wilms’ Tumor
1 (WT1) antigen.
About Intellia Therapeutics
Intellia 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 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: its ability to
conduct and complete clinical studies for NTLA-5001 for the
treatment of AML, including manufacturing of NTLA-5001 to support
such clinical trials; the safety, efficacy and advancement of our
clinical program for NTLA-5001 for the treatment of AML; its
ability to generate data to demonstrate NTLA-5001 as a potential
engineered T cell therapy designed to treat all genetic subtypes of
AML; the ability to enroll patients in both arms of the Phase 1/2a
study; the ability to enroll patients in expansion cohorts upon the
identification of a preferred dosage in each arm of the Phase 1/2a
study; its ability to develop its modular platform and
full-spectrum approach to advance its complex genome editing
capabilities, including to apply its proprietary cell engineering
platform to additional product candidates; the advancement and
expansion of its CRISPR/Cas9 technology to develop human
therapeutic products; its ability to maintain and expand its
related intellectual property portfolio, and avoid or acquire
rights to valid intellectual property of third parties; its ability
to demonstrate its platform’s modularity and replicate or apply
results achieved in preclinical studies, including those in our AML
program, in any future studies, including human clinical trials;
its ability to develop other in vivo or ex
vivo cell therapeutics of all types, and those targeting WT1
in AML in particular, using CRISPR/Cas9 technology; its
expectations of the potential impact of the coronavirus disease
2019 pandemic on strategy, future operations, manufacturing and
timing of its clinical trials or IND submissions; the timing of
regulatory filings and clinical trial execution, including dosing
of patients; and potential commercial opportunities, including
value and market, for its product candidates, including NTLA-5001
for AML; and its ability to fund operations beyond the next 24
months.
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 the successful enrollment of patients in the Phase 1/2a
study for NTLA-5001 for the treatment of AML; risks related to the
successful manufacturing of NTLA-5001 to support the Phase 1/2a
study for NTLA-5001 for the treatment of AML; 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, including manufacturing, for its in vivo
and ex vivo product candidates, including NTLA-5001; the risk that
any one or more of Intellia’s product candidates, including
NTLA-5001, will not be successfully developed and commercialized;
the risk that the results of preclinical studies or clinical
studies, including for NTLA-5001, will not be predictive of future
results in connection with future studies; and the risk that
Intellia’s will not be able to demonstrate its platform’s
modularity and replicate or apply results achieved in preclinical
studies to develop additional product candidates, including to
apply its proprietary cell engineering platform successfully to
additional 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 (SEC). 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 LiDirector, Investor
Relations+1-857-706-1612lina.li@intelliatx.com
Media:Matt CrensonTen Bridge
Communications+1-917-640-7930media@intelliatx.commcrenson@tenbridgecommunications.com
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