Intellia Therapeutics Receives U.S. FDA Orphan Drug Designation for NTLA-5001 for the Treatment of Acute Myeloid Leukemia
10 March 2022 - 8:01AM
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 U.S. Food and Drug Administration (FDA) has granted orphan
drug designation for Intellia’s ex vivo investigational T cell
receptor (TCR)-T cell therapy, NTLA-5001, for the treatment of
acute myeloid leukemia (AML).
NTLA-5001 is an autologous TCR-T cell therapy designed to target
the Wilms’ Tumor (WT1) antigen, which is highly expressed in AML
and many other hematologic and solid tumors. NTLA-5001 is currently
being evaluated in a Phase 1/2a study in adults with persistent or
recurrent AML who have previously received first-line therapy.
“The FDA’s decision to grant orphan drug designation for
NTLA-5001 reflects the serious need for novel treatment options for
people living with AML, a disease with notably poor long-term
survival,” said Intellia President and Chief Executive Officer John
Leonard, M.D. “As part of our full-spectrum genome editing
strategy, we seek to leverage our proprietary CRISPR/Cas9-based
platform to engineer differentiated cell therapies targeting
cancers for which there are currently limited or no treatment
options. We look forward to advancing our investigational TCR-T
cell therapy, NTLA-5001, through the clinic in hopes of improving
future treatment options for patients in need.”
The FDA's Orphan Drug Designation program provides orphan status
to drugs defined as those intended for the treatment, diagnosis or
prevention of rare diseases that affect fewer than 200,000 people
in the United States. Orphan drug designation qualifies the sponsor
of the drug for certain development incentives, including tax
credits for qualified clinical testing, prescription drug user-fee
exemptions and seven-year marketing exclusivity upon FDA
approval.
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. NTLA-5001
utilizes a WT1-targeting TCR identified in collaboration with IRCCS
Ospedale San Raffaele. 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: the safety, efficacy and advancement of its
clinical programs for NTLA-5001 for the treatment of acute myeloid
leukemia pursuant to its clinical trial applications (“CTA”) and
IND submissions, including the expected timing of data releases,
regulatory filings, and the initiation and completion of clinical
trials; the expansion of its CRISPR/Cas9 technology and related
technologies to advance additional development candidates,
including to apply such technologies to engineer differentiated
cell therapies, as well as its ability to maintain and expand its
related intellectual property portfolio; and expectations of the
potential impact of the coronavirus disease 2019 pandemic on
strategy, and future operations and timing of its clinical
trials.
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; risks related to
the authorization, initiation and conduct of studies and other
development requirements for its product candidates, such as
NTLA-5001, including risks related to regulatory approvals to
conduct clinical trials; the risk that any one or more of
Intellia’s product candidates, such as NTLA-5001, will not be
successfully developed and commercialized; and the risk that the
results of preclinical studies or clinical studies will not be
predictive of future results in connection with future studies for
Intellia’s current and future product candidates, including current
and future engineered cell therapy 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 (“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 Crenson Ten Bridge
Communications+1-917-640-7930media@intelliatx.commcrenson@tenbridgecommunications.com
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