Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing
company focused on the development of potentially curative
therapeutics using CRISPR technology, announced financial and
operational results for the second quarter of 2017.
The company has made significant progress using our proprietary
lipid nanoparticle (LNP) delivery system throughout the quarter. We
advanced into non-human primate studies, while first time data from
rat studies, the second species we tested in vivo, confirmed the
durable mouse data demonstrating high levels of sustained genome
editing and serum transthyretin (TTR) reduction post-single-dose
intravenous administration.
“We are very excited that our initial non-human primate data in
vivo continue to validate our mRNA delivery technology. These
preclinical data accelerate our momentum as we advance the
development of potential therapies to treat patients with high
unmet medical needs,” said Chief Executive Officer and founder
Nessan Bermingham, Ph.D., Intellia Therapeutics. “During the
quarter, we have further strengthened our company by advancing
partnerships, including a research agreement with Ospedale San
Raffaele in Milan for our ex vivo technology focused on novel
engineered cell therapies using CRISPR, expanded our global
intellectual property portfolio, and appointed two new members to
our Board of Directors.”
Second Quarter 2017 Operational Highlights
The company achieved several key operational milestones during
the second quarter of 2017, including:
- Commenced non-human primate studies in support of our
preclinical work relating to our lead development program in
TTR. We demonstrated robust green fluorescent protein expression
throughout non-human primate liver 24 hours after a single
systemically delivered administration dose in vivo, using our
proprietary lipid nanoparticle delivery system.
- Extended our data set beyond the mouse and delivered
CRISPR/Cas9 in the rat. Specifically, initial data from rat models
post-single-dose intravenous administration in vivo using our
proprietary lipid nanoparticle delivery system showed up to 91
percent reduction in serum TTR protein levels and up to 66 percent
editing at the target DNA site, demonstrating robust and
dose-responsive gene editing of TTR. We presented these results at
the American Society of Gene & Cell Therapy Annual Meeting in
Washington D.C. in May 2017.
- Provided an update on our ongoing durability study,
demonstrating durable liver editing through nine-months post a
single intravenous dose administration in mice using our
proprietary lipid nanoparticle delivery system. Throughout the
liver, the data continues to demonstrate durability and high
editing efficiency of LNP-mediated editing of the TTR gene, showing
97 percent reduction in serum TTR protein levels and approximately
70 percent editing at the target DNA site. The intended transient
nature of LNP delivery has been confirmed in rodents with
undetectable Cas9 mRNA and gRNA levels in the liver by 72 hours
post administration.
- Entered into a three-year research collaboration, option, and
license agreement to engineer optimized T cell cancer therapies
with a leading scientific research institution, Ospedale San
Raffaele SRL of Milan, Italy. • The
collaboration aims to discover innovative targets against
tough-to-treat cancers, leveraging Intellia’s proprietary
CRISPR/Cas9 platform to generate next-generation T cell therapies
to address unmet needs in hematological and solid tumors.
• The collaboration is the first partnership of
Intellia’s eXtellia division, focused on advancing next generations
of engineered cell therapies through unique and proprietary
applications of CRISPR genome editing in areas including
immuno-oncology and auto-immunity.
- Continued to defend and enhance our CRISPR/Cas9 foundational
and therapeutic intellectual property position through filing and
prosecution of patent applications covering our internal,
collaboration and in-licensed inventions. In relation to the
foundational CRISPR/Cas9 genome editing intellectual property
portfolio to which we have rights for human therapeutics and
companion diagnostics, and which is co-owned by the Regents of the
University of California, the University of Vienna, and Dr.
Emmanuelle Charpentier (collectively “UC”), the following was
achieved: • China’s State Intellectual
Property Office granted to UC a patent broadly covering CRISPR/Cas9
single-guide genome editing methods and compositions. The patent
includes claims that cover methods for editing DNA in non-cellular
and cellular settings, including in eukaryotic cells such as human
and mammalian cells. It also includes CRISPR/Cas9 composition of
matter and system claims for use in any setting, including claims
covering the use of CRISPR/Cas9 in producing medicines for treating
disease. • UC submitted its opening brief
July 25, 2017 to the U.S. Court of Appeals for the Federal Circuit
in their appeal from a February 15, 2017 decision by the U.S.
Patent and Trademark Office’s Patent Trial and Appeal Board
(“PTAB”) in an interference proceeding relating to the CRISPR/Cas9
genome editing technology. In the appeal, UC requests the reversal
of the PTAB’s decision to terminate the interference between
certain CRISPR/Cas9 patent claims owned by UC and patents and
patent applications owned by the Broad Institute, Massachusetts
Institute of Technology, the President and Fellows of Harvard
College and the Rockefeller University.
- Named Moncef Slaoui, Ph.D. and Frank Verwiel, M.D. to its Board
of Directors. In addition the Board established a Science &
Technology Committee: • Moncef Slaoui, Ph.D.
and Frank Verwiel, M.D. joined the Board of Directors. Drs. Slaoui
and Verwiel began Board responsibilities on July 25, 2017.
Intellia’s Board of Directors also established a Science and
Technology Committee, which will be chaired by Dr. Slaoui.
Additionally, Carl L. Gordon, Ph.D., CFA, General Partner, OrbiMed
Advisors, LLC, informed the company of his resignation from the
Intellia Board of Directors effective at the close of business on
July 25, 2017.
Second Quarter 2017 Financial
Results
Collaboration Revenue
Collaboration revenue was $5.9 million for the second quarter of
2017, compared to $4.2 million for the second quarter of the prior
year. The increase in collaboration revenue in 2017 was primarily
driven by amounts recognized under our collaboration agreement with
Regeneron Pharmaceuticals, Inc. (Regeneron), which was entered into
in April 2016.
Through June 30, 2017, the company received $104.1 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 $2.8 million. Excluding the $2.6 million of the
upfront payment received from Novartis, which was allocated to the
purchase of equity securities, we recognized $34.7 million in
collaboration revenue under these agreements through June 30, 2017,
and had remaining deferred revenue of $69.6 million as of June 30,
2017.
Operating Expenses
Research and development expenses increased $8.2 million to
$15.6 million during the second quarter 2017, compared to $7.4
million during the same period of 2016. This increase was driven
primarily by greater support for the advancement of our early-stage
research programs and includes laboratory supplies and research
materials. Additionally, salary and related headcount-based
expenses increased as the company grew to 110 research and
development employees as of June 30, 2017, from 56 research and
development employees as of June 30, 2016.
General and administrative expenses increased $2.7 million to
$6.4 million during the second quarter of this year, compared to
$3.7 million in the second quarter of 2016. This increase was
driven primarily by increased salary and related headcount-based
expenses as the company grew to 33 general and administrative
employees as of June 30, 2017, from 19 general and administrative
employees as of June 30, 2016, to support our 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.
Our net loss was $15.6 million for the second quarter 2017,
compared to $6.9 million for the second quarter of 2016.
Balance Sheet
Cash and cash equivalents at June 30, 2017, were $241 million,
compared to $301 million for the same quarter in 2016. The base
period cash and cash equivalents were primarily attributable to
$115.5 million in proceeds from our initial public offering, $55
million in concurrent private placements, and a $75 million upfront
payment from Regeneron in April 2016. The year-over-year change is
attributed to cash used in ongoing operations.
Financial Guidance
Our 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 our licensed
intellectual property, and general overhead costs.
During 2017, the company expects expenses to continue to
increase compared to prior periods relating to our ongoing
activities, particularly as research and development and
preclinical activities gather further momentum toward human
clinical trials, and we spend a full year occupying our new office
and laboratory facility, which we began to occupy in the fourth
quarter of 2016.
As of June 30, 2017, the company had an accumulated deficit of
$81.8 million. We expect our losses to increase as we continue to
incur significant research and development and other expenses
related to the advancement of our therapeutic programs and our
ongoing operations. Based on our research and development plans and
expectations related to the progress of the company’s programs, we
expect that the cash and cash equivalents as of June 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 our
collaboration agreements with Novartis and Regeneron.
About Intellia Therapeutics
Intellia Therapeutics is a leading genome editing company
focused on the development of 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. Our combination of deep scientific,
technical and clinical development experience, along with our
leading intellectual property portfolio, puts us 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 liver 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; 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. |
|
CONSOLIDATED STATEMENTS OF OPERATIONS
(UNAUDITED) |
|
(Amounts in thousands except per share
data) |
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Three Months Ended June 30, |
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Six Months Ended June 30, |
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2017 |
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2016 |
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2017 |
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2016 |
|
Collaboration revenue |
|
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$ 5,917 |
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$ 4,206 |
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$ 12,132 |
|
$ 5,983 |
|
Operating
expenses: |
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Research
and development |
|
15,565 |
|
7,423 |
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28,996 |
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12,648 |
|
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General and
administrative |
|
6,369 |
|
3,729 |
|
12,101 |
|
6,975 |
|
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Total
operating expenses |
|
21,934 |
|
11,152 |
|
41,097 |
|
19,623 |
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Operating
loss |
|
|
(16,017) |
|
(6,946) |
|
(28,965) |
|
(13,640) |
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Interest
income |
|
|
424 |
|
46 |
|
741 |
|
51 |
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Net
loss |
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$ (15,593) |
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$ (6,900) |
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$ (28,224) |
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$ (13,589) |
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Net loss per share attributable to common stockholders, basic
and diluted |
$ (0.45) |
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$ (0.36) |
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$ (0.81) |
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$ (1.37) |
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Weighted average shares outstanding, basic and diluted |
34,916 |
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19,121 |
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34,820 |
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9,899 |
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INTELLIA THERAPEUTICS, INC. |
CONSOLIDATED BALANCE SHEET DATA
(UNAUDITED) |
(Amounts in thousands) |
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June 30, 2017 |
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December 31, 2016 |
Cash and cash equivalents |
|
|
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$ 241,047 |
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$ 273,064 |
Total
assets |
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264,975 |
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298,969 |
Total
liabilities |
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77,390 |
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89,132 |
Total
stockholders' equity |
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187,585 |
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209,837 |
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Intellia Contacts:
Media Contact:
Jennifer Mound Smoter
Senior Vice President, External Affairs & Communications
+1 857-706-1071
jenn.smoter@intelliatx.com
Investor Contact:
Graeme Bell
Executive Vice President, Chief Financial Officer
+1 857-706-1081
graeme.bell@intelliatx.com
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