Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing
company focused on developing curative therapeutics using
CRISPR/Cas9 technology both in vivo and ex
vivo, is presenting three oral presentations and two poster
presentations at the 23rd Annual Meeting of the American Society of
Gene and Cell Therapy (ASGCT), taking place virtually from May
12-15, 2020. Intellia researchers are presenting new data in
support of NTLA-5001, the company’s engineered cell therapy
candidate for the treatment of acute myeloid leukemia (AML).
Intellia is also providing an update on NTLA-2002, its newest
development candidate for the treatment of hereditary angioedema
(HAE).
“At Intellia, we are applying our CRISPR/Cas9 technology to
develop new processes that can produce enhanced engineered cell
therapies to treat severe cancers, such as AML, that traditional
approaches cannot address. Our proprietary platform provides a
powerful tool to generate more potent TCR-directed cells, that can
treat blood cancers initially and potentially solid tumors. The
data being presented today validate Intellia’s approach of reducing
AML tumor cell blasts, and our plans to enter the clinic with
NTLA-5001 next year,” said Intellia President and CEO John Leonard,
M.D. “We are also pleased to present data that support our recently
announced HAE development candidate, NTLA-2002, Intellia’s second
systemic therapy employing our in vivo knockout approach and
modular delivery platform.”
Data Presentations on Intellia’s First Engineered Cell
Therapy Development Candidate, NTLA-5001 for the Treatment of AML,
and Proprietary Cell Engineering Process
NTLA-5001 is Intellia’s first engineered T cell receptor (TCR) T
cell therapy development candidate, which targets the Wilms’ Tumor
1 (WT1) intracellular antigen for the treatment of AML. NTLA-5001
is being developed in collaboration with Chiara Bonini’s team at
IRCCS Ospedale San Raffaele to treat AML patients regardless of the
genetic subtype of a patient’s leukemia. AML is a cancer of the
blood and bone marrow that is rapidly fatal without immediate
treatment and is the most common type of acute leukemia in
adults (Source: NIH SEER Cancer Stat Facts: Leukemia –
AML).
Intellia’s proprietary process is a significant improvement over
standard engineering processes commonly used to introduce nucleic
acids into cells. Intellia’s process enabled multiple gene edits
using CRISPR/Cas9, while maintaining cell products with high
expansion potential and minimal undesirable chromosomal
translocations. CRISPR/Cas9 was used to insert a WT1-directed TCR
in locus, while eliminating the expression of the endogenous TCRs,
with the goal of producing homogeneous T cell therapies like
NTLA-5001.
Intellia’s novel approach with NTLA-5001 can overcome the
challenges of standard T cell therapy, including risks of reduced
specificity associated with mixed expression and mispairing of
endogenous and transgenic TCRs (tgTCRs); graph-versus-host disease
(GvHD) risks, which could lead to an attack on the patient’s
healthy cells; and reduced efficacy tied to lower tgTCR expression
per T cell. Intellia’s unprecedented process is expected to
streamline cell engineering and manufacturing, yielding a
homogenous product comprising WT1-targeted T cells with high
anti-tumor activity. Data highlights from today’s presentation
include the following:
- Developed a sequential genome editing process in primary human
T cells that lead to the knockout of three genes with up to >98%
efficiency and no detectable target-to-target translocations.
Applied sequential editing approach to achieve knockout of the
endogenous TCR with up to >99% efficiency, along with insertion
of the tgTCR targeting WT1 into 50-70% of the cells.
- Improved T cell viability post-editing that resulted in a
significant increase in T cell expansion relative to cells
engineered by standard methods, and which is expected to shorten
the time required for T cell manufacturing and to increase the
yield of therapeutic cells.
- Increased proportion of cells having a desirable early stem
cell memory phenotype with improved reactivity against
WT1-expressing tumor cell lines and higher long-term proliferative
capacity, which may be associated with better persistence in
patients.
Intellia’s cell engineering efforts are focused on its initial
clinical investigation of NLTA-5001 on AML, while continuing
preclinical studies exploring the potential for targeting WT1 in
solid tumors. The company confirmed plans last week to submit an
IND or IND-equivalent for NTLA-5001 for the treatment of AML in the
first half of 2021.
The presentation titled, “Enhanced tgTCR T Cell Product
Attributes Through Process Improvement of CRISPR/Cas9 Engineering,”
will be made today by Aaron Prodeus, Ph.D., senior scientist, Cell
Therapy, and can be found here, on the Scientific Publications
& Presentations page of Intellia’s website. These data were a
follow-on to the study presented at Keystone Symposia’s Engineering
the Genome Conference from this past February.
In Vivo Data Supports Intellia’s Novel
TCR Candidate
A second presentation on engineered cell therapy progress, in
collaboration with IRCCS Ospedale San Raffaele, showed in vivo data
demonstrating the potential of TCR-edited T cells to effectively
target WT1 tumor cells in AML. In addition to the previously
disclosed results of effective in vitro recognition of primary AML
tumor cells by edited WT1-specific cytotoxic T cells (CD8 T cells),
new data indicate that the selected TCR also enables T helper cells
(CD4 T cells) to react to WT1-expressing tumor cells, providing
cytokine support. This distinguishes Intellia’s TCR from other
therapeutic TCR candidates, which either exclusively activate toxic
CD8 T cells or require the co-transfection of CD8 into CD4 T cells
to render them functional.
Using a mouse model carrying disseminated human primary AML,
researchers observed a significant therapeutic effect, including
decreased AML tumor burden. In addition, no signs of GvHD were
observed in mice treated with the WT1-specific T cells. The data
show that tgTCR-engineered cells have targeted anti-cancer activity
in a challenging model of systemic AML, demonstrating the
therapeutic potential of Intellia’s engineered TCR T cell
approach.
The presentation titled, “Exploiting CRISPR-Genome Editing and
WT1-Specific T Cell Receptors to Redirect T Lymphocytes Against
Acute Myeloid Leukemia,” will be given today by Eliana Ruggiero,
Ph.D., Experimental Hematology Unit, Division of Immunology,
Transplantation and Infectious Diseases, IRCCS Ospedale San
Raffaele, Italy. Notably, ASGCT meeting organizers selected this
presentation as one of six to receive the ASGCT Excellence in
Research Award this year.
Continued Progress on Intellia’s Second In Vivo
Development Candidate, NTLA-2002 for the Treatment of
HAE
Intellia is presenting development data updates on its potential
HAE therapy, NTLA-2002, which utilizes the company’s systemic in
vivo knockout approach, including its proprietary lipid
nanoparticle (LNP) system. HAE is a rare genetic disorder
characterized by recurring and unpredictable severe swelling
attacks in various parts of the body, and is significantly
debilitating or even fatal in certain cases. NTLA-2002 aims to
prevent unregulated production of bradykinin by knocking out the
prekallikrein B1 (KLKB1) gene through a single course of treatment
to ameliorate the frequency and intensity of these swelling
attacks.
The KLKB1 gene knockout in an ongoing non-human primate (NHP)
study resulted in a sustained 90% reduction in kallikrein activity,
a level that translates to a therapeutically meaningful impact on
HAE attack rates (Source: Banerji et al., NEJM, 2017). This
kallikrein activity reduction was sustained for at least six
months, demonstrating the same high level of efficacy and
durability seen in earlier rodent studies.
The short talk titled, “CRISPR/Cas9-Mediated Gene Knockout of
KLKB1 to Treat Hereditary Angioedema,” will be given by Jessica
Seitzer, director, Genomics, Intellia on Fri., May 15, 2020, when
it will be made available here, on the Scientific Publications
& Presentations page of Intellia’s website. The presented data
include results from ongoing collaborations with researchers at
Regeneron, and the program is subject to an option by Regeneron to
enter into a Co/Co agreement, in which Intellia would remain the
lead party. Intellia expects to submit an IND or IND-equivalent to
initiate a Phase 1 trial for NTLA-2002 in the second half of
2021.
About Intellia Therapeutics
Intellia Therapeutics is a leading genome editing company
focused on developing proprietary, curative therapeutics using the
CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology
has the potential to transform medicine by permanently editing
disease-associated genes in the human body with a single treatment
course, and through improved cell therapies that can treat cancer
and immunological diseases, or can replace patients’ diseased
cells. The combination of deep scientific, technical and clinical
development experience, along with its leading intellectual
property portfolio, puts Intellia in a unique position to unlock
broad therapeutic applications of the CRISPR/Cas9 technology and
create a new class of therapeutic products. Learn more
about Intellia Therapeutics and CRISPR/Cas9
at intelliatx.com and 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: planned submission of an
investigational new drug (“IND”) application or similar clinical
trial application for NTLA-2001 for the treatment of transthyretin
amyloidosis (“ATTR”) in mid-2020 and its planned dosing of first
patients in the second half of 2020; plans to submit an IND
application for NTLA-5001, its first T cell receptor
(“TCR”)-directed engineered cell therapy development candidate for
its acute myeloid leukemia (“AML”) program in the first half of
2021; plans to submit an IND or similar clinical trial application
for its hereditary angioedema (“HAE”) program in the second half of
2021; plans to advance and complete preclinical studies, including
non-human primate studies for its ATTR program and HAE programs,
and other animal studies supporting other in vivo and ex vivo
programs, including its AML program; development of a proprietary
LNP/AAV hybrid delivery system, as well as its modular platform to
advance its complex genome editing capabilities, such as gene
insertion; further development of its proprietary cell engineering
process for multiple sequential editing; presentation of additional
data at upcoming scientific conferences, and other preclinical data
in 2020; advancement and expansion of its CRISPR/Cas9 technology to
develop human therapeutic products, as well as its ability to
maintain and expand its related intellectual property portfolio;
ability to demonstrate its platform’s modularity and replicate or
apply results achieved in preclinical studies, including those in
its ATTR, AML, and HAE programs, 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; ability to optimize the
impact of its collaborations on its development programs, including
but not limited to its collaborations with Novartis or Regeneron
Pharmaceuticals, Inc., and Regeneron’s ability to enter into a
co-development and co-promotion agreement for the HAE program;
statements regarding the timing of regulatory filings regarding its
development programs.
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 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 Novartis or Regeneron
or its other ex vivo 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. 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:
Media:Lynnea OlivarezDirectorExternal Affairs
& Communications+1 956-330-1917
lynnea.olivarez@intelliatx.com
Investors:Lina LiAssociate DirectorInvestor
Relations+1 857-706-1612lina.li@intelliatx.com
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