Intellia Therapeutics Announces U.S. FDA Acceptance of Investigational New Drug Application for NTLA-5001, its CRISPR/Cas9-Engineered TCR-T Cell Candidate for Acute Myeloid Leukemia
16 September 2021 - 9:30PM
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage
genome editing company focused on developing curative therapeutics
using CRISPR/Cas9 technology both in vivo and ex
vivo, today announced that the U.S. Food and Drug Administration
(FDA) has accepted the investigational new drug (IND) application
for NTLA-5001, the company’s first wholly-owned ex vivo CRISPR
genome editing candidate for the treatment of cancer. NTLA-5001 is
an autologous T cell receptor (TCR)-T cell therapy engineered to
target the Wilms’ Tumor (WT1) antigen for the treatment of all
genetic subtypes of acute myeloid leukemia (AML). Intellia intends
to initiate patient screening by year-end in a Phase 1/2a study
evaluating NTLA-5001 in adults with persistent or recurrent AML who
have previously received first-line therapy.
“The FDA’s acceptance of our IND for NTLA-5001 is an important
milestone in our pursuit of developing advanced cell therapies
utilizing Intellia’s proprietary engineering platform to treat
patients with cancer. NTLA-5001 is our first wholly-owned ex vivo
candidate to enter the clinic, and we expect to initiate this
first-in-human study in adults with AML by year-end. Our treatment
strategy is to leverage CRISPR/Cas9 genome editing technology to
create next-generation engineered immune cells with the potential
to attack cancer cells more effectively and safely than previously
developed cell therapies,” said Intellia President and Chief
Executive Officer John Leonard, M.D. “Our study is an important
first step toward improving treatment for people living with this
aggressive form of cancer. AML is the most common type of acute
leukemia in adults, that, despite currently available treatments,
has a five-year survival rate of less than 30 percent.”
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 will contain a dose escalation and
expansion phase, with up to 54 participants. The dose-escalation
phase of the study will include two independent arms of up to three
cohorts: Arm 1 will consist of adults with AML with lower disease
burden, defined as those with less than 5% AML blasts in bone
marrow, while Arm 2 will consist of adults with AML with higher
disease burden, defined as those greater than or equal to 5% AML
blasts in bone marrow. Once a dose is identified in each arm, two
expansion cohorts will be opened for further safety assessment.
More information about the study will be available
at clinicaltrials.gov.
In addition to the U.S., Intellia has also submitted a
regulatory application to the U.K. for NTLA-5001.
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, with more than 20,000
estimated new cases in 2020. Despite currently available treatments
for AML, the five-year overall survival rate for patients remains
less than thirty percent. AML, along with other cancer types, is
often characterized by overexpression of the Wilms’ Tumor 1 (WT1)
antigen.
About NTLA-5001
NTLA-5001 is a 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 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 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 @intelliatweets.
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: acceptance of a clinical trial
application (“CTA”) or equivalent regulatory submission for
NTLA-5001 for the treatment of acute myeloid leukemia (“AML”) and
ability to initiate a clinical trial by the end of 2021; ability to
generate data to demonstrate NTLA-5001 as a potential best-in-class
engineered T cell therapy designed to treat all genetic subtypes of
AML; plans to evaluate in preclinical studies the potential use of
NTLA-5001 to treat Wilms’ Tumor 1 (“WT1”)-positive solid tumors;
plans to advance and complete preclinical studies for our research
programs; development of our modular platform to advance our
complex genome editing capabilities; further development of our
proprietary genome editing tools for research and therapeutic
development, including sequential editing; presentation of
additional data at upcoming scientific conferences, and other
preclinical data in 2021; advancement and expansion of our
CRISPR/Cas9 technology to develop human therapeutic products;
ability to maintain and expand our related intellectual property
portfolio, and avoid or acquire rights to valid intellectual
property of third parties; ability to demonstrate our 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; 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; expectations of
the potential impact of the coronavirus disease 2019 pandemic on
strategy, future operations and timing of its clinical trials or
IND submissions; ability to optimize the impact of our
collaborations on our development programs, statements regarding
the timing of regulatory filings and clinical trial execution,
including dosing of patients, regarding our development programs;
potential commercial opportunities, including value and market, for
our product candidates; our expectations regarding our use of
capital and other financial results during 2021; and our 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 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; 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 Intellia’s collaborations with Regeneron or its other
collaborations will not continue or will not be successful. 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:Lisa QuTen Bridge
Communications+1-678-662-9166media@intelliatx.comlqu@tenbridgecommunications.com
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