Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing
company focused on developing curative therapeutics using CRISPR
technology, announced financial results and operational progress
for the fourth quarter and full year of 2017.
Using our proprietary lipid nanoparticle (LNP) delivery system,
Intellia has achieved near-complete knockout editing of the
transthyretin amyloidosis target gene (TTR) in rodents and most
recently shared data on the progress of non-human primate (NHP)
studies. In the fourth quarter, our NHP studies continued, yielding
higher levels of editing as the Company improved the LNP
formulation and dosing regimens, including repeated doses. Ongoing
efforts in the NHP studies focus on consistently achieving a
therapeutically relevant level of editing for the TTR gene as
determined by transthyretin level reductions and we continue to
observe and, as in prior animal studies, gather long-term
durability data.
In parallel with the continuous progress on ATTR, during the
fourth quarter, the Company expanded its research in the liver in
two ways. First, in collaboration with partner Regeneron
Pharmaceuticals, Inc. (Regeneron), we successfully inserted a
functional gene into a specific site in the mouse genome, achieving
therapeutically relevant levels of gene expression and
demonstrating proficiency in executing complex editing procedures.
These results provide a proof-of-principle for a large class of
genetic defects that cannot be addressed solely by gene knockout.
Second, knockout editing in livers of mice was successful for a
second therapeutic target, the SERPINA1 gene that gives rise to
liver complications in certain alpha-1 antitrypsin deficiency
patients. The Company plans to present these data at upcoming
scientific conferences and extend our evaluation in additional in
vivo studies.
Through the ongoing collaboration with Novartis Institutes for
BioMedical Research, Inc. (Novartis), the Company made further
progress on complex ex vivo editing. Data presented at the 2017
American Society of Hematology (ASH) Annual Meeting and Exposition
showed that an Intellia-qualified gRNA targeting the erythroid
specific enhancer region of the BCL11A gene in human CD34+ cells
could edit greater than 80 percent of these healthy bone
marrow-derived cells, which led to the majority of the cells
expressing fetal hemoglobin. These edited hematopoietic stem cells
maintained engraftment over a 16-week period and demonstrated
multi-lineage reconstitution, retaining their ability to complete
normal hematopoiesis (blood cell formation) after the gene
edit.
“2017 marked significant progress for Intellia with the
CRISPR/Cas9 technology and our lead ATTR program. We demonstrated
editing with our proprietary lipid nanoparticle delivery system
across multiple animal species, including NHPs, and advanced this
program towards clinical studies. After raising an additional $141
million, we are advancing the Company’s emerging pipeline of
indications in the liver, including transthyretin amyloidosis and
alpha-1 antitrypsin deficiency, expanding our in vivo editing work
beyond the liver into CNS, and accelerating our ex vivo cellular
therapy programs,” said President and Chief Executive Officer, John
Leonard, M.D., Intellia Therapeutics. “As we begin 2018, our team
remains focused on the patient and making genome editing-based
therapies a reality. We are well-positioned to deliver against our
goals and mission.”
The Company achieved several key milestones during 2017,
including:
- Produced interim, top-line NHP data demonstrating, for the
first time, liver genome editing using CRISPR/Cas9 delivered
through a LNP system;
- Confirmed re-dosing in NHPs produced increased levels of
editing, where the treatment/drug and regimen was well tolerated as
assessed by clinical signs and chemistry;
- Presented 12-month data from a long-term mouse study,
demonstrating robust and durable in vivo genome editing
following a single, systemic intravenous delivery of Intellia’s
proprietary, non-viral, LNP delivery system;
- Evaluated in vivo delivery by LNPs to a second organ, with
successful genome editing by CRISPR/Cas9 in the central nervous
system (cerebellum and striatum) in mice;
- In collaboration with Regeneron, produced positive insertion
editing data in mice, demonstrating capability to perform complex
genetic editing;
- In collaboration with Novartis, generated positive data in
sickle cell anemia in transplant mouse models; and
- Continued to enhance and defend the Company’s CRISPR/Cas9
foundational and therapeutic intellectual property position, which
included filing multiple patent applications covering our
inventions and the issuance in Europe, the United Kingdom,
Australia and China of foundational CRISPR/Cas9 patents for which
we have in-licensed rights.
Upcoming Goals
The Company has set forth the following for 2018 pipeline
progression:
- Mid-year: begin IND-enabling activities for lead ATTR
development candidate
- Mid-year: present additional editing data following in vivo
delivery by LNPs to organs beyond the liver
- Late 2018: advance lead development candidate for second
indication
- During 2018: present additional insertion/repair editing data;
and
- During 2018: present preclinical data in support of our first
proprietary ex vivo programs on immuno-oncology and
autoimmune/inflammation indications.
Fourth Quarter and Full Year 2017 Financial
Results
Collaboration Revenue
Collaboration revenue was $6.7 million for the fourth quarter of
2017, compared to $5.6 million for the fourth quarter of the prior
year. The increase in collaboration revenue in 2017 was primarily
driven by amounts recognized under Intellia’s collaboration
agreement with Regeneron which was entered in April 2016.
Since inception through December 31, 2017, the Company has
received $106.1 million in funding from the collaborations with
Novartis and Regeneron, excluding amounts received for equity
investments, and had an accounts receivable balance of $10.5
million at December 31, 2017. Excluding the $2.6 million of the
upfront payment received from Novartis, which was allocated to the
purchase of the Company’s equity securities, Intellia has
recognized $48.6 million in collaboration revenue under these
agreements from inception through December 31, 2017, and had a
remaining deferred revenue balance of $65.3 million at December 31,
2017.
Operating Expenses
Research and Development expenses increased by $9.8 million to
$21.2 million during the fourth quarter of 2017, compared to $11.3
million during the same period of 2016. This increase was driven
primarily by the advancement of Intellia’s research programs,
research personnel growth to support these programs, as well as the
expansion of the development organization, and includes laboratory
supplies, research materials and certain equipment. Additionally,
salary and related headcount-based expenses increased, as the
Company grew to 143 research and development personnel as of
December 31, 2017, from 74 research and development employees as of
December 31, 2016.
General and administrative expenses increased by $5.1 million to
$10.2 million during the fourth quarter of this year, compared to
$5.1 million in the fourth quarter of 2016. This increase was
driven primarily by increased salary and related headcount-based
expenses as the Company grew to 41 general and administrative
employees as of December 31, 2017, from 29 general and
administrative employees as of December 31, 2016, to support
Intellia’s larger research and development organization, public
company compliance and administrative obligations. The Company also
incurred increased corporate insurance, legal, and other
professional expenses related to our expanding operations since
becoming a public company in May 2016.
The Company’s net loss was $24.0 million for the fourth quarter
of 2017, compared to $10.6 million for the fourth quarter of
2016.
Balance Sheet
Cash and cash equivalents at December 31, 2017, were $340.7
million, compared to $273.1 million at December 31, 2016. The base
period cash and cash equivalents were primarily attributable to
$115.5 million in proceeds from the Company’s initial public
offering and $55.0 million in concurrent private placements in May
2016, in addition to a $75.0 million upfront payment from Regeneron
in April 2016 and a follow-on public offering of $141.0 million in
November 2017.
Financial Guidance
The Company’s primary uses of capital will continue to be
research and development programs, laboratory and related supplies,
compensation costs for current and future employees, consulting,
legal and other regulatory expenses, patent prosecution filing and
maintenance costs for Intellia’s licensed intellectual property,
and general overhead costs.
As of December 31, 2017, the Company had an accumulated deficit
of $121.1 million. The Company expects losses to increase as it
continues to incur significant research and development expenses
related to the advancement of Intellia’s therapeutic programs and
ongoing operations. Based on Intellia’s research and development
plans and expectations related to the progress with the Company’s
programs, the Company expects that the cash and cash equivalents as
of December 31, 2017, as well as technology access and research
funding from Novartis and Regeneron, will enable Intellia to fund
operating expenses and capital expenditures through
mid-2020, excluding 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 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 optimized cell therapies that can treat cancer
and immunological diseases by replacing patients’ diseased cells.
The combination of deep scientific, technical and clinical
development experience, along with our 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; Follow us
on Twitter @intelliatweets.
Forward-Looking Statements
This press release contains "forward-looking statements" of
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 our
ability to advance and expand the CRISPR/Cas9 technology to develop
into human therapeutic products, as well as our CRISPR/Cas9
intellectual property portfolio; our ability to achieve stable or
effective genome editing with a single treatment dose; the
potential timing and advancement of our preclinical studies,
including continuing non-human primate studies, and clinical
trials; our ability to replicate results achieved in our
preclinical studies in any future studies, including human clinical
trials; the potential development of ex vivo cell therapeutics of
all types using CRISPR/Cas9 technology; our ability to commence
IND-enabling activities of a lead ATTR development candidate by
mid-2018; our intent to present additional data for organs beyond
the liver, additional insertion/repair data, and preclinical data
in support of our first ex vivo programs on immuno-oncology and
autoimmune/inflammation indications during 2018; our ability to
nominate a development candidate for a second indication by late
2018; the intellectual property position and strategy of Intellia’s
licensors; actions by government agencies; 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 through
mid-2020. 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 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 be predictive
of future results in connection with future studies; and the risk
that Intellia’s collaborations with Novartis or Regeneron 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 filed with the Securities and Exchange Commission, 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 Therapeutics 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 |
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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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2017 |
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2016 |
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2017 |
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2016 |
|
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|
Collaboration revenue |
|
|
$ |
6,668 |
|
|
$ |
5,627 |
|
|
$ |
26,117 |
|
|
$ |
16,479 |
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|
Operating
expenses: |
|
|
|
|
|
|
|
|
|
|
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Research
and development |
|
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21,170 |
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|
11,331 |
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67,647 |
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|
|
31,840 |
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General and
administrative |
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10,213 |
|
|
|
5,118 |
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|
28,025 |
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|
16,798 |
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Total
operating expenses |
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31,383 |
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|
16,449 |
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|
95,672 |
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|
48,638 |
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Operating
loss |
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(24,715 |
) |
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(10,822 |
) |
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(69,555 |
) |
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(32,159 |
) |
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Interest
income |
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|
|
752 |
|
|
|
259 |
|
|
|
2,012 |
|
|
|
525 |
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Net
loss |
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|
|
$ |
(23,963 |
) |
|
$ |
(10,563 |
) |
|
$ |
(67,543 |
) |
|
$ |
(31,634 |
) |
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Net loss per share attributable to common stockholders, basic
and diluted |
$ |
(0.61 |
) |
|
$ |
(0.31 |
) |
|
$ |
(1.88 |
) |
|
$ |
(1.42 |
) |
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Weighted average shares outstanding, basic and diluted |
|
39,155 |
|
|
|
34,507 |
|
|
|
36,006 |
|
|
|
22,222 |
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INTELLIA THERAPEUTICS, INC. |
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CONSOLIDATED BALANCE SHEET DATA |
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(Amounts in thousands) |
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December 31,
2017 |
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December 31,
2016 |
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Cash and cash equivalents |
|
|
|
$ |
340,678 |
|
$ |
273,064 |
|
Total
assets |
|
|
|
|
|
376,235 |
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|
298,969 |
|
Total
liabilities |
|
|
|
|
|
75,638 |
|
|
89,132 |
|
Total
stockholders' equity |
|
|
|
|
300,597 |
|
|
209,837 |
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Intellia Contacts:
Investor Contact: Lindsey Trickett Vice
President, Investor Relations +1 857-285-6211
lindsey.trickett@intelliatx.com
Media Contacts:Jennifer Mound Smoter Senior
Vice President, External Affairs & Communications +1
857-706-1071 jenn.smoter@intelliatx.com
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