Intellia Therapeutics, Inc. (Nasdaq:NTLA), a leading genome editing
company focused on developing curative therapeutics using CRISPR
technology, announced financial and operational results for the
third quarter of 2017.
The Company made considerable progress in its Transthyretin
Amyloidosis (TTR) program using its proprietary lipid nanoparticle
(LNP) delivery system. Based on interim, top-line data from
Intellia’s first exploratory non-human primate (NHP) studies, the
Company has demonstrated successful editing in the livers of
individual animals with NHP guide RNAs (gRNAs) as well as with
exploratory human cross-reactive gRNAs. These interim results,
coupled with on-going studies and further optimization efforts, are
anticipated to allow for the selection of a human guide development
candidate as early as the first quarter of 2018.
In addition, Intellia completed a 12-month mouse durability study,
maintaining approximately 97 percent reduction in serum TTR protein
levels, accompanied by sustained editing at the target DNA site in
the liver after a single intravenous administration of its
CRISPR/Cas9 system.
“We accomplished some significant milestones during the third
quarter, demonstrating the first CRISPR/Cas9 liver-targeted genome
editing with systemic delivery of LNPs in non-human primates, the
third animal species in which we have achieved editing. Our
delivery system was safe and well tolerated in the studies,
enabling us to redose without adverse events observed. In our
52-week mouse study, we demonstrated editing durability after a
single dose of CRISPR/Cas9 using our delivery system. Moving beyond
the liver, we also demonstrated the potential adaptability of our
approach by showing delivery and editing in the central nervous
system in mice. These data further advance Intellia’s mission to
develop curative genome editing therapies for current unmet medical
needs,” said Chief Executive Officer and Founder, Nessan
Bermingham, Ph.D., Intellia Therapeutics. “Financially, the company
continued to have a strong cash position at the end of the
quarter.”
Third Quarter 2017 Highlights
The Company achieved several key milestones during the third
quarter of 2017, including:
- Produced interim, top-line NHP data demonstrating, for the
first time, liver genome editing using CRISPR/Cas9 delivered
through a LNP system, including observing over 20 percent editing
in certain animals:
- Observed editing ranges of 0.10 percent up to 32.0 percent
after a single dose, with multiple animals showing editing greater
than 20 percent after a second dose. These results were observed in
experiments with various exploratory gRNA, LNP formulations and
dosing regimens, similar to prior results from Intellia’s initial
rodent studies;
- Confirmed re-dosing in NHPs produced increased levels of
editing, with no adverse events observed;
- Well-tolerated safety profile observed to date with both
NHP-specific gRNA and exploratory human cross-reactive gRNAs. This
was assessed by gross observation, as well as by clinical
chemistry, hematology, and cytokine and complement levels;
- Animals with the highest levels of editing showed a higher
reduction in TTR serum protein levels; and
- Initiated further studies to optimize editing efficiency and to
explore dosing parameters, which will lead toward the selection of
a development candidate.
- Presented 12-month data from the completed long-term mouse
study, demonstrating robust and durable in vivo genome
editing following a single, systemic intravenous administration:
- Achieved and maintained approximately a 97 percent reduction in
serum TTR protein levels through one year, corresponding with
approximately 70 percent editing at the target DNA site in the
liver;
- Confirmed the transient nature of Intellia’s LNP delivery
system with 99 percent clearance of mRNA within 10 hours and
clearance of sgRNA within 72 hours in the liver; and
- The treatment was observed to be safe and well tolerated during
the 52-week study.
- Made progress in evaluating 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.
- Continued to defend and enhance the Company’s CRISPR/Cas9
foundational and therapeutic intellectual property position through
filing and prosecution of patent applications covering its
internal, collaboration and in-licensed inventions.
Third Quarter 2017 Financial Results
Collaboration Revenue
Collaboration revenue was $7.3 million for the third quarter of
2017, compared to $4.9 million for the third 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 Pharmaceuticals, Inc. (Regeneron), which
was entered into in April 2016.
Since its inception through September 30, 2017, the Company has
received $105.1 million in funding under its collaborations with
Novartis Institutes for BioMedical Research, Inc. (Novartis) and
Regeneron, excluding amounts received for equity investments and
recorded accounts receivable of $3.5 million. Excluding the $2.6
million of the upfront payment received from Novartis, which was
allocated to its purchase of the Company’s equity securities,
Intellia recognized $42.0 million in collaboration revenue under
these agreements through September 30, 2017, and had remaining
deferred revenue of $65.0 million as of September 30, 2017.
Operating Expenses
Research and development expenses increased by $9.6 million to
$17.5 million during the third quarter 2017, compared to $7.9
million during the same period of 2016. This increase was driven
primarily by personnel growth and the advancement of Intellia’s
research programs, as well as the initial build-out of the
development organization, and includes laboratory supplies,
research materials and bench-top equipment. Additionally, salary
and related headcount-based expenses increased, as the Company grew
to 135 research and development personnel as of September 30, 2017,
from 66 research and development employees as of September 30,
2016.
General and administrative expenses increased by $1.0 million to
$5.7 million during the third quarter of this year, compared to
$4.7 million in the third quarter of 2016. This increase was driven
primarily by increased salary and related headcount-based expenses
as the Company grew to 34 general and administrative employees as
of September 30, 2017, from 23 general and administrative employees
as of September 30, 2016, to support Intellia’s public company
compliance and administration obligations. The Company also
incurred increased corporate insurance, legal, and other
professional expenses related to its expanding operations since
becoming a public company in May 2016.
The Company’s net loss was $15.4 million for the third quarter
2017, compared to $7.5 million for the third quarter 2016.
Balance Sheet
Cash and cash equivalents at September 30, 2017, were $222
million, compared to $291 million for the same quarter in 2016. The
base period cash and cash equivalents were primarily attributable
to $115.5 million in proceeds from the Company’s initial public
offering, $55 million in concurrent private placements, and a $75
million upfront payment from Regeneron in April 2016.
Financial Guidance
The Company’s primary uses of capital will continue to be
research and development programs, laboratory and related supplies,
compensation and related expenses, legal and other regulatory
expenses, patent prosecution, filing and maintenance costs for
Intellia’s licensed intellectual property, and general overhead
costs.
During the remainder of 2017, the Company expects expenses to
continue to increase compared to prior periods relating to its
ongoing activities, particularly as certain research and
development activities progress toward human clinical trials, and
as the Company spends a full year in its new office and laboratory
facility, which it has occupied since the fourth quarter of
2016.
As of September 30, 2017, the Company had an accumulated deficit
of $97.2 million. The Company expects its losses to increase as it
continues to incur significant research and development and other
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 September 30, 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-2019, excluding any potential milestone payments or
extension fees received under its collaboration agreements with
Novartis and Regeneron.
Conference Call to Discuss Third Quarter 2017
Earnings
The Company will present third quarter 2017
results to investors and analysts in a conference call on October
31, 2017 at 8:30am EDT, hosted by Nessan Bermingham, Ph.D., Chief
Executive Officer and Founder, John Leonard, M.D.,Executive Vice
President, R&D, and Graeme Bell, Chief Financial Officer. The
analyst and investor presentation can be downloaded starting at
8:00am EDT from the Investor Relations section of the company’s
website at www.intelliatx.com.
To participate on the day, U.S. callers should
dial 888-752-0423 and use Conference ID# 9089048, approximately
five minutes before the call. International callers should dial +1
918-398-4936 and use Conference ID# 9089048, approximately five
minutes before the call.
About Intellia Therapeutics
Intellia Therapeutics is a leading genome editing company
focused on developing proprietary, potentially 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. 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; 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
Intellia’s 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 editing; 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 the ex vivo cell therapeutics
through our eXtellia division, including the development of
next-generation T cell therapies that address unmet needs in
hematological and solid tumors, immuno-oncology and auto-immunity;
the intellectual property position and strategy of Intellia’s
licensors; actions by government agencies; the impact of our
collaborations with Ospedale San Raffaele, Novartis and Regeneron
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 2017 financial
results; and our ability to fund operations through mid-2019. 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 quarterly report on Form 10-Q
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
(UNAUDITED) |
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(Amounts in thousands except per share
data) |
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Three Months Ended September 30, |
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Nine Months Ended September 30, |
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2017 |
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2016 |
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2017 |
|
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2016 |
|
|
|
Collaboration revenue |
|
$ |
7,317 |
|
|
$ |
4,869 |
|
|
$ |
19,449 |
|
|
$ |
10,852 |
|
|
|
Operating
expenses: |
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|
|
|
|
|
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Research
and development |
|
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17,481 |
|
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|
7,861 |
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46,477 |
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|
|
20,509 |
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General and
administrative |
|
|
5,711 |
|
|
|
4,705 |
|
|
|
17,812 |
|
|
|
11,680 |
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Total
operating expenses |
|
|
23,192 |
|
|
|
12,566 |
|
|
|
64,289 |
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|
32,189 |
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Operating
loss |
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|
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(15,875 |
) |
|
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(7,697 |
) |
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(44,840 |
) |
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(21,337 |
) |
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Interest
income |
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|
|
519 |
|
|
|
215 |
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|
|
1,260 |
|
|
|
266 |
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Net
loss |
|
|
|
$ |
(15,356 |
) |
|
$ |
(7,482 |
) |
|
$ |
(43,580 |
) |
|
$ |
(21,071 |
) |
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Net loss per share attributable to common stockholders, basic
and diluted |
$ |
(0.44 |
) |
|
$ |
(0.22 |
) |
|
$ |
(1.25 |
) |
|
$ |
(1.16 |
) |
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Weighted average shares outstanding, basic and diluted |
|
35,189 |
|
|
|
34,316 |
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|
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34,945 |
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|
18,098 |
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INTELLIA THERAPEUTICS, INC. |
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CONSOLIDATED BALANCE SHEET DATA
(UNAUDITED) |
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(Amounts in thousands) |
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September 30,
2017 |
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December 31,
2016 |
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Cash and cash equivalents |
|
|
|
$ |
222,264 |
|
$ |
273,064 |
|
Total
assets |
|
|
|
|
|
249,170 |
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|
298,969 |
|
Total
liabilities |
|
|
|
|
|
73,323 |
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|
89,132 |
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Total
stockholders' equity (deficit) |
|
|
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175,847 |
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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 Contact:Jennifer Mound Smoter Senior Vice
President, External Affairs & Communications +1 857-706-1071
jenn.smoter@intelliatx.com
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