Intellia Therapeutics Announces Fourth Quarter and Full-Year 2019 Financial Results
27 Février 2020 - 1:30PM
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, today reported
operational highlights and financial results for the fourth quarter
and year ended December 31, 2019.
“In 2019, we advanced our full-spectrum strategy, guiding both
our in vivo and ex vivo lead programs toward the clinic. We also
continued to build on our genome editing and delivery capabilities
to enable a rapid succession of candidates,” said Intellia
President and Chief Executive Officer, John Leonard, M.D. “We are
off to a productive start in 2020. We announced the nomination of
NTLA-5001, a WT1-directed TCR-T cell therapy for the treatment of
AML, and plan to select our third development candidate in the
first half of this year, which will be for the treatment of HAE. In
addition, in the second half of the year, we expect to begin dosing
ATTR patients with NTLA-2001, a potential single-course treatment
for ATTR patients. This is anticipated to be the first-ever
systemically delivered CRISPR/Cas9-based therapy to enter the
clinic, representing an important milestone in our mission to
deliver potentially curative therapies from our proprietary modular
platform.”
2019 and Recent Operational Highlights
- ATTR Program: Intellia remains on track to
submit an investigational new drug (IND) application in mid-2020
for its lead in vivo candidate, NTLA-2001, for the treatment of
transthyretin amyloidosis (ATTR). In December 2019, Intellia
completed a 12-month durability study of its lead lipid
nanoparticle (LNP) formulation in support of NTLA-2001, maintaining
an average reduction of >95% of serum transthyretin (TTR)
protein and sustained liver genome editing after a single dose in
non-human primates (NHPs). NTLA-2001 is anticipated to be the first
systemically delivered CRISPR/Cas9 therapy to enter the clinic, and
Intellia expects to dose the first patients in the second half of
2020. NTLA-2001 is part of a co-development/co-promotion (Co/Co)
agreement between Intellia, which is the lead development and
commercialization party, and Regeneron Pharmaceuticals, Inc.
(Regeneron).
- AML Program: In January 2020, Intellia
announced NTLA-5001 as its first engineered T cell therapy
development candidate for the treatment of acute myeloid leukemia
(AML). NTLA-5001 utilizes a T cell receptor (TCR)-directed approach
to target the Wilms’ Tumor 1 (WT1) intracellular antigen to develop
a broadly applicable treatment for AML patients, regardless of
mutational background of a patient’s leukemia. At the recent
Keystone Symposia’s Engineering the Genome Conference, the Company
presented data in support of NTLA-5001, demonstrating that the
selection of a naturally-occurring, high-affinity TCR, in
combination with Intellia’s CRISPR-enabled engineering and targeted
insertion, results in an engineered T cell capable of specific and
potent killing of primary AML blasts. Importantly, data presented
showed that CRISPR-enabled engineering overcomes certain key
challenges of traditional TCR approaches, such as mispairing
between therapeutic and endogenous TCR, therefore creating a more
homogenous T cell product. The engineered T cell carrying the
therapeutic TCR also exhibited no detectable off-target reactivity
to bone marrow cells. The Company plans to submit an IND
application for NTLA-5001 in the first half of 2021. Additional
efforts are underway to evaluate the potential use of the
WT1-directed TCR construct to treat other tumor types, including
solid tumors.
- HAE Program: In January 2020, Intellia
announced hereditary angioedema (HAE) as its third development
program. Building on the modular LNP delivery platform developed in
the ATTR program, the Company aims to knock out the kallikrein B1
(KLKB1) gene with a single course of treatment to reduce the
spontaneous activation of biological pathways responsible for
generating bradykinin and thereby ameliorate the frequency and
intensity of HAE attacks. At the recent Keystone Conference, the
Company reported results from an ongoing NHP study, which
demonstrated a 90% reduction in kallikrein activity sustained for
at least five months following a single dose. The reduction of
kallikrein activity observed in this study corresponds to the
reduced enzymatic levels in patients that meaningfully impact HAE
attack rates (Source: Banerji et al., NEJM, 2017). Intellia expects
to select its development candidate in the first half of 2020.
Intellia’s KLKB1 HAE program is subject to an option by Regeneron
to enter into a Co/Co agreement prior to the initiation of
IND-enabling studies, with Intellia as the lead party.
- Modular Platform: Intellia continued to
progress differentiated genome editing and delivery strategies,
including targeted insertion and consecutive editing, across its in
vivo and ex vivo efforts. These platform capabilities enable the
removal and/or restoration of a gene’s function in developing
treatments for life-threatening diseases. In 2019, the Company
demonstrated the first CRISPR-mediated, targeted transgene
insertion in the liver of NHPs. The targeted insertion of the
Factor 9 gene generated circulating human Factor IX protein at or
above normal levels. Additionally, Intellia presented the first
demonstration of a consecutive in vivo gene knockout followed by a
targeted insertion in an alpha-1 antitrypsin deficiency (AATD)
mouse model. The consecutive edits led to >98% reduction of the
disease-causing protein and sustained restoration of the normal
protein to therapeutically relevant circulating levels throughout
the study. The Company continues to advance these platform
capabilities and leverage them to develop the next wave of in vivo
and ex vivo clinical candidates.
- Board of Directors Update: Dr. Frank Verwiel
was elected Chairman of the Board of Directors in February 2020,
succeeding Perry Karsen, who will remain a member of the
Board.
Upcoming Milestones
The Company has set forth the following for pipeline
progression:
- ATTR:
- Submit IND application for NTLA-2001 in mid-2020
- Dose first patients in 2H 2020
- AML:
- Submit IND application for NTLA-5001 in 1H 2021
- HAE:
- Nominate a development candidate in 1H 2020
- R&D Advancements:
- Present preclinical data at upcoming scientific conferences in
2020
Upcoming Event
The Company will participate in the following investor event
during the first quarter of 2020:
- Barclays Capital Global Healthcare Conference, March 10,
Miami
Fourth Quarter and Full Year 2019 Financial
Results
- Cash Position: Cash, cash
equivalents and marketable securities were $284.5 million as of
December 31, 2019, compared to $314.1 million as of December 31,
2018. The decrease was driven by cash used to fund operations of
approximately $124.9 million, which was offset in part by $72.3
million of net equity proceeds raised from the Company's "At the
Market" (ATM) offerings, $9.0 million of funding received under the
Novartis collaboration, $9.9 million of ATTR cost reimbursements
made by Regeneron, and $4.2 million in proceeds from employee-based
stock plans.
- Collaboration Revenue:
Collaboration revenue increased by approximately $3.1 million to
$10.9 million during the fourth quarter of 2019, compared to $7.9
million during the fourth quarter of 2018. The increase in
collaboration revenue in 2019 was primarily driven by amounts
recognized under the Company’s ATTR Co/Co agreement with Regeneron.
As previously disclosed, Regeneron funded approximately 50% of the
development costs for the ATTR program throughout 2019. Starting in
June 2020, Regeneron will share approximately 25% of worldwide
development costs and commercial profits for the ATTR program.
- R&D Expenses: Research
and development expenses increased by $11.8 million to $31.7
million during the fourth quarter of 2019, compared to $19.9
million during the fourth quarter of 2018. This increase was mainly
due to IND-enabling activities for NTLA-2001, research efforts
supporting the selection of NTLA-5001 and the expansion of the
Company’s research and development team.
- G&A Expenses: General and
administrative expenses increased by $0.3 million to $9.0 million
during the fourth quarter of 2019, compared to $8.7 million during
the fourth quarter of 2018. This increase was driven primarily by
employee-related expenses.
- Net Loss: The Company’s net
loss was $28.3 million for the fourth quarter of 2019, compared to
$19.1 million during the fourth quarter of 2018.
Financial Guidance
Intellia expects that its cash, cash equivalents and marketable
securities as of December 31, 2019 will enable the Company to fund
its anticipated operating expenses and capital expenditure
requirements at least through the end of 2021. This expectation
excludes any strategic use of capital not currently in the
Company’s base-case planning assumptions.
Conference Call to Discuss Fourth Quarter and Full Year
2019 Earnings
The Company will discuss these results on a conference call
today, February 27, 2020, at 8 a.m. ET.
To join the call:
- U.S. callers should dial 1-877-317-6789 and use conference ID#
10138773, approximately five minutes before the call.
- International callers should dial 1-412-317-6789 and use
conference ID# 10138773, approximately five minutes before the
call.
A replay of the call will be available through the Events and
Presentations page of the Investor Relations section on Intellia’s
website, beginning on February 27, 2020 at 12 p.m. ET.
About Intellia TherapeuticsIntellia
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 nominate a
development candidate for its hereditary angioedema (“HAE”) program
in the first half of 2020; plans to advance and complete
preclinical studies, including non-human primate studies for its
ATTR program, AML program, HAE program and other in vivo and ex
vivo programs; 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; 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 continue its growth and realize the
anticipated contribution of the members of its board of directors
and executives to its operations and progress; ability to optmize
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; use of capital, expenses,
future accumulated deficit and other 2019 financial results or in
the future; and ability to fund operations through the end of
2021.
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.
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INTELLIA
THERAPEUTICS, INC. |
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CONSOLIDATED
STATEMENTS OF OPERATIONS (UNAUDITED) |
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(Amounts in
thousands, except per share data) |
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Three Months Ended December 31, |
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Twelve Months Ended December 31, |
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2019 |
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2018 |
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2019 |
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2018 |
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Collaboration revenue |
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$ |
10,936 |
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$ |
7,880 |
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$ |
43,103 |
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$ |
30,434 |
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Operating expenses: |
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Research and development |
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31,731 |
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19,918 |
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108,413 |
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89,115 |
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General and administrative |
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8,976 |
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8,708 |
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41,058 |
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32,189 |
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Total operating expenses |
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40,707 |
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28,626 |
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149,471 |
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121,304 |
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Operating loss |
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(29,771 |
) |
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(20,746 |
) |
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(106,368 |
) |
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(90,870 |
) |
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Interest income |
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1,495 |
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1,680 |
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6,835 |
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5,527 |
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Net loss |
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$ |
(28,276 |
) |
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$ |
(19,066 |
) |
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$ |
(99,533 |
) |
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$ |
(85,343 |
) |
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Net loss per share, basic and diluted |
$ |
(0.57 |
) |
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$ |
(0.43 |
) |
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$ |
(2.11 |
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$ |
(1.98 |
) |
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Weighted average shares outstanding, basic and diluted |
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49,350 |
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44,215 |
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47,247 |
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43,069 |
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INTELLIA
THERAPEUTICS, INC. |
CONSOLIDATED
STATEMENTS OF OPERATIONS (UNAUDITED) |
(Amounts in
thousands) |
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December 31, 2019 |
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December 31, 2018 |
Cash, cash
equivalents and marketable securities |
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$ |
284,472 |
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$ |
314,059 |
Total assets |
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334,280 |
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347,315 |
Total liabilities |
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64,399 |
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69,395 |
Total stockholders' equity |
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269,881 |
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277,920 |
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Intellia Contacts:
Investors:Lina LiAssociate DirectorInvestor
Relations+1 857-706-1612lina.li@intelliatx.com
Media:Jennifer Mound SmoterSenior Vice
PresidentExternal Affairs & Communications+1
857-706-1071jenn.smoter@intelliatx.com
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