Intellia Therapeutics Announces Fourth Quarter and Full-Year 2018 Financial Results
February 27 2019 - 5:30AM
Intellia Therapeutics, Inc.® (NASDAQ:NTLA), a leading genome
editing company focused on developing curative therapeutics using
CRISPR/Cas9 technology in both in vivo and ex vivo applications,
today reported operational highlights and financial results for the
fourth quarter and year ended December 31, 2018. In addition,
Intellia highlighted select corporate milestones for 2019 and
upcoming events for the first quarter of 2019.
“We are delivering on our full-spectrum strategy, and we expect
to have two candidates in development in 2019. Our in vivo
transthyretin amyloidosis program is on track for IND filing in
2020, and by the end of this year we anticipate having our first
engineered cell therapy development candidate targeting acute
myeloid leukemia,” said Intellia President and Chief Executive
Officer John Leonard, M.D. “We have shown that a single lipid
nanoparticle administration can produce very substantial
transthyretin protein reduction in non-human primates, reaching
levels that we believe hold great therapeutic promise for patients.
We also look forward to sharing additional data on our acute
myeloid leukemia program this year. We believe our TCR-based,
CRISPR-engineered cell therapy will provide a much-needed option
for patients.”
Recent Operational Highlights
- ATTR Program: During the
fourth quarter, Intellia completed confirmatory non-human
primate (NHP) studies, previewed in October, using its
lead candidate for the treatment of transthyretin amyloidosis
(ATTR). Fifty-one days post infusion, these NHP studies verified a
favorable tolerability profile across various dose levels, and a
near-complete (average of >95 percent) reduction in
circulating transthyretin (TTR) protein in the liver. The
improvements in TTR protein reduction were the result of certain
modifications made to the lipid nanoparticle (LNP) cargo components
of the therapy, and these modifications have been incorporated into
the ongoing, dose-range finding studies and scale-up activities.
These modifications will also have application in subsequent
programs. The ATTR program is being co-developed with Regeneron
Pharmaceuticals, Inc. (Regeneron) with Intellia being the lead
party. Intellia confirmed that it is on track for submitting an
Investigational New Drug (IND) application in 2020 for ATTR.
- In Vivo
Insertion: In October, Intellia
demonstrated in vivo CRISPR-mediated, targeted transgene
insertion in mouse liver. Data shared at the Annual
Congress of the European Society of Gene and Cell Therapy
highlighted the use of Intellia’s bi-directional DNA
template, delivered in a proprietary hybrid
LNP/adeno-associated viral (AAV) template delivery system.
Insertion of the human F9 gene, the gene encoding the protein
deficient in hemophilia B, yielded Factor IX protein levels in mice
at or above therapeutic targets in patients. This work was done in
collaboration with Regeneron. Additionally, the Company
demonstrated the versatility of the hybrid LNP/AAV approach by
successfully inserting a functional human SERPINA1 gene (encoding
alpha-1 antitrypsin) into the same locus. These experiments in mice
yielded human protein expression levels consistent with those of
normal individuals without alpha-1 antitrypsin deficiency.
- Engineered Cell Therapies: As
part of the broad engineered cell therapy platform that the Company
is developing, Intellia and its partner, Ospedale San Raffaele,
isolated novel active T cell receptors (TCRs) recognizing an
epitope of the Wilms’ Tumor 1 (WT1) protein. This protein is
overexpressed in many blood cancers, as well as in solid tumors. T
cells modified with these TCRs successfully killed acute
myeloid leukemia (AML) blasts in an in vitro model. This work will
be the foundation for the Company’s first wholly owned ex vivo
development candidate for the treatment of AML.
- Novartis: In December,
Intellia announced an expansion of the existing cell
therapy collaboration with Novartis Institutes for Biomedical
Research, Inc. (Novartis) to include ocular stem cells.
The Company received a $10 million payment from Novartis in
relation to the inclusion of this cell type. Regarding its
proprietary LNP delivery system and improvements, Intellia also
obtained expanded access to Novartis’ LNP library, including the
rights to use these lipids for in vivo or ex vivo applications in
any genome editing technology.
- Board of Directors: In
January of 2019, Intellia
appointed Fred Cohen, M.D., D.Phil,
F.A.C.P., to its board of directors, adding both
biotechnology drug development experience and extensive medical
expertise to the current knowledge base of the Company’s
board.
Upcoming Milestones
The Company has set forth the following for 2019 pipeline
progression:
- ATTR: Complete dose-range finding studies, initiate
IND-enabling toxicology studies and commence manufacturing of
lipid and CRISPR/Cas9 cargo
- Engineered Cell Therapy: Nominate first engineered cell therapy
development candidate for acute myeloid leukemia by the end of
2019
- Present additional in vivo NHP insertion data and ATTR
formulation improvement data at upcoming scientific
conferences
Upcoming Events
The Company will participate in the following investor
events:
- Leerink Healthcare Conference, February 28, New York City
- Barclays Global Healthcare Conference, March 12, Miami
Fourth Quarter and Full Year 2018 Financial
Results
- Cash Position: Cash, cash
equivalents and marketable securities were $314.1 million as of
December 31, 2018, compared to $340.7 million as of December 31,
2017. The decrease was driven by cash used to fund operations of
approximately $96 million, which was offset in part by $28.5
million of net equity proceeds raised from the Company's "At the
Market" (ATM) agreement, $11.7 million in proceeds from
employee-based stock plans, $10.4 million of ATTR cost
reimbursements made by Regeneron, and $19.0 million of funding
received under the Novartis collaboration. The Novartis funding
received included a $10.0 million upfront payment related to the
expansion of the existing collaboration in the fourth quarter of
2018.
- Collaboration Revenue:
Collaboration revenue increased by $1.2 million to $7.9 million
during the fourth quarter of 2018, compared to $6.7 million during
the fourth quarter of 2017. The increase in collaboration revenue
in 2018 was primarily driven by amounts recognized from the
expansion of the existing collaboration with Novartis, as well as
by amounts recognized under the Company’s ATTR Co/Co agreement with
Regeneron. As previously disclosed, Regeneron is obligated to fund
approximately 50 percent of the development costs for the ATTR
program.
- R&D Expenses: Research
and development expenses decreased by $1.3 million to $19.9 million
during the fourth quarter of 2018, compared to $21.2 million during
the fourth quarter of 2017. This decrease was driven primarily by
lower consumable costs, as well as the timing of general R&D
expenses.
- G&A Expenses: General and
administrative expenses decreased by $1.5 million to $8.7 million
during the fourth quarter of 2018, compared to $10.2 million during
the fourth quarter of 2017. This decrease was driven primarily by a
decrease in stock-based compensation.
- Net Loss: The Company’s net
loss was $19.1 million for the fourth quarter of 2018, compared to
$24.0 million during the fourth quarter of 2017.
Financial
Guidance
Intellia expects that its cash, cash equivalents and marketable
securities as of December 31, 2018, as well as technology access
and funding from Novartis and Regeneron, will enable Intellia to
fund its anticipated operating expenses and capital expenditure
requirements into the first half of 2021. This expectation excludes
any potential milestone payments or extension fees that could be
earned and distributed under the collaboration agreements
with Novartis and Regeneron or any strategic use of
capital not currently in the base-case planning assumptions.
About Intellia Therapeutics
Intellia Therapeutics® is a leading genome editing company
focused on developing 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”) 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 ability to advance and
expand the CRISPR/Cas9 technology to develop into human therapeutic
products, as well as our intellectual property portfolio; our
ability to achieve stable or effective genome editing; our ability
to administer our CRISPR/Cas9 product candidates; the potential
timing and advancement of our preclinical studies, including
continuing non-human primate studies for our ATTR program and other
programs (such as alpha-1 antitrypsin deficiency (AATD) and AML),
and clinical trials; the timing and potential achievement of
milestones to advance our pipeline including nominating development
candidates and filing INDs; our ability to replicate or apply
results achieved in our preclinical studies, including those in our
ATTR, AATD and AML programs, in any future studies, including human
clinical trials; the potential development of our proprietary
LNP/AAV hybrid delivery system to advance our complex genome
editing capabilities; the potential development of other in vivo
or ex vivo cell therapeutics of all types, and those
targeting WT1 in AML in particular, using CRISPR/Cas9 technology;
our ability to conduct successful IND-enabling studies of a lead
ATTR development candidate and subsequently submitting an IND
application in 2020 that will be accepted by the regulatory
agencies; our intent to present additional ATTR data, additional
insertion/repair data, and other preclinical data during 2019 or
thereafter; our ability to advance a development candidate for a
second indication by late 2019 or thereafter; our plans to commence
manufacturing efforts in 2019; the intellectual property position
and strategy of Intellia’s licensors, or other parties from which
it derives rights, as well as third-parties and competitors;
actions by government agencies; our growth as a company and the
anticipated contribution of the members of our board of directors
and our executives to our operations and progress; the impact of
our collaborations on our development programs; the potential
timing of regulatory filings regarding our development programs;
the potential commercialization opportunities, including value and
market, for product candidates; our expectations regarding our uses
of capital, expenses, future accumulated deficit and other 2018
financial results; and our ability to fund operations into the
first half 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 our
intellectual property position; risks related to Intellia’s
relationship with third parties, including our licensors; risks
related to the ability of our licensors to protect and maintain
their intellectual property position; uncertainties related to the
initiation and conduct of studies and other development
requirements for our 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 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. |
CONSOLIDATED STATEMENTS OF OPERATIONS
(UNAUDITED) |
(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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2018 |
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2017 |
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2018 |
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2017 |
Collaboration revenue |
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$ |
7,880 |
|
$ |
6,668 |
|
$ |
30,434 |
|
$ |
26,117 |
Operating
expenses: |
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Research
and development |
|
19,918 |
|
21,170 |
|
89,115 |
|
67,647 |
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General and
administrative |
|
8,708 |
|
10,213 |
|
32,189 |
|
28,025 |
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Total
operating expenses |
28,626 |
|
31,383 |
|
121,304 |
|
95,672 |
Operating
loss |
|
|
(20,746) |
|
(24,715) |
|
(90,870) |
|
(69,555) |
Interest
income |
|
|
1,680 |
|
752 |
|
5,527 |
|
2,012 |
Net
loss |
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$ |
(19,066) |
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$ |
(23,963) |
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$ |
(85,343) |
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$ |
(67,543) |
Net loss
per share, basic and diluted |
$ |
(0.43) |
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$ |
(0.61) |
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$ |
(1.98) |
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$ |
(1.88) |
Weighted
average shares outstanding, basic and diluted |
44,215 |
|
39,155 |
|
43,069 |
|
36,006 |
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INTELLIA THERAPEUTICS, INC. |
CONSOLIDATED BALANCE SHEET DATA
(UNAUDITED) |
(Amounts in thousands) |
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December 31, 2018 |
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December 31, 2017 |
Cash, cash
equivalents and marketable securities |
|
$ |
314,059 |
|
$ |
340,678 |
Total
assets |
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347,315 |
|
376,235 |
Total
liabilities |
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69,395 |
|
75,638 |
Total
stockholders' equity |
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277,920 |
|
300,597 |
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Intellia Contacts:
Investors:Lindsey TrickettVice
PresidentInvestor Relations+1
857-285-6211lindsey.trickett@intelliatx.com
Media:Jennifer Mound Smoter Senior Vice
President External Affairs & Communications +1
857-706-1071 jenn.smoter@intelliatx.com
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