Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing
company focused on the development of potentially curative
therapeutics using CRISPR technology, announced financial results
for the first quarter 2017.
“The first quarter of 2017 has been highly productive for
Intellia as we continue to advance our preclinical programs toward
developing life-transforming therapies. Building on our earlier
results showing 97 percent in vivo reduction of serum transthyretin
protein levels, our durability data also continue to demonstrate
stable liver editing and support our vision that genome editing
could be effective with just a single dose,” said Nessan
Bermingham, Ph.D., chief executive officer, and founder, Intellia
Therapeutics. “We expect to advance preclinical studies
significantly this year by progressing our technology in non-human
primate studies and look toward its potential broad clinical
applications to help treat patients with unmet medical needs.”
Business Highlights
During the first quarter of 2017, the company achieved numerous
important milestones:
Significantly advanced the genome editing field with
unprecedented liver editing and lipid nanoparticle delivery data
using CRISPR/Cas9 as a potential one-time
treatment.
- In March, the company reported editing efficiency in vivo with
a significant reduction of serum transthyretin (TTR) protein
levels. More recent durability study data show stable liver editing
in mice for at least six months following a single
administration.
- Enhanced in vivo gene editing in the TTR locus, with an
efficiency of approximately 70 percent in the total mouse liver at
the target DNA site, after a single intravenous
administration;
- Decreased serum TTR protein levels of up to approximately 97
percent; and
- Achieved undetectable Cas9 messenger RNA (mRNA) and guide RNA
(gRNA) in the liver by 72 hours, post administration.
Defended and enhanced our CRISPR/Cas9 foundational and
therapeutic intellectual property position.
- The European Patent Office (EPO) decided to grant to co-owners
the Regents of the University of California, University of Vienna,
and Dr. Emmanuelle Charpentier (collectively, UC) a patent broadly
covering the CRISPR/Cas9 genome editing technology. The patent
includes claims covering compositions of the widely adopted CRISPR
single guide RNA technology for use in any non-cellular or cellular
setting, including eukaryotic cells such as human or mammalian
cells, as well as for use in human therapeutics. This European
patent, which is expected to grant on May 10, 2017, will be
nationalized in, and cover, approximately forty European countries
that are member states of the European Patent Organisation.
- In addition to the EPO decision, earlier this year, the United
Kingdom’s (U.K.) Intellectual Property Office granted to UC a
second national patent on the CRISPR/Cas9 genome editing system.
This February 7, 2017 U.K. patent covers chimeric CRISPR/Cas9
systems in which the Cas9 protein is modified to provide
alternative DNA-modulating activities while the first U.K. patent
obtained by UC covers the single guide RNA for uses in both
non-cellular and cellular settings.
- In April 2017, UC appealed to the U.S. Court of Appeals for the
Federal Circuit the decision by the Patent Trial and Appeal Board
(PTAB) to terminate the interference between certain CRISPR/Cas9
patent claims owned by UC and patents and patent applications owned
by the Broad Institute, Harvard University and the Massachusetts
Institute of Technology. UC is seeking review and reversal of the
PTAB’s February 15, 2017 decision, which concluded that, although
the claims overlap, the respective scope of UC and Broad’s claim
sets do not define the same patentable invention and, accordingly,
terminated the interference without deciding which party first
invented the use of the CRISPR/Cas9 technology in eukaryotic
cells.
First Quarter 2017 Financial
Results
Collaboration Revenue
Collaboration revenue was $6.2 million for the first quarter of
2017, compared to $1.8 million for the first 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 March 31, 2017, the company received $103.1 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 $2.4 million. Excluding the $2.6 million of the
upfront payment received from Novartis which was allocated to the
purchase of equity securities, we recognized $28.8 million in
collaboration revenue under these agreements through March 31, 2017
and had remaining deferred revenue of $74 million as of March 31,
2017.
Operating Expenses
Research and development expenses increased $8.2 million to
$13.4 million during the first quarter 2017, compared to $5.2
million during the same period of 2016. This increase was driven
primarily by increased salary and related headcount-based expenses,
including equity-based compensation expenses, as the company grew
to 88 research and development employees as of March 31, 2017, from
44 research and development employees as of March 31, 2016. This
increase supported the advancement of our early-stage research
programs including, consumption of laboratory supplies and research
materials.
General and administrative expenses increased $2.5 million to
$5.7 million during the first quarter of this year, compared to
$3.2 million in the first quarter of 2016. This increase was driven
primarily by increased salary and related headcount-based expenses,
including equity-based compensation expenses, as the company grew
to 31 general and administrative employees as of March 31, 2017,
from 17 general and administrative employees as of March 31, 2016.
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 $12.6 million for the first quarter 2017,
compared to $6.7 million for the first quarter of 2016.
Balance Sheet
Cash and cash equivalents at March 31, 2017, were $258 million,
compared to $64 million for the same quarter in 2016. The increase
in cash and cash equivalents was 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, partially offset by cash used in
operations.
Financial Guidance
Our primary uses of capital will continue to be research and
development services, compensation and related expenses, laboratory
and related supplies, 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 in connection with our ongoing
activities, particularly as research and development and
preclinical activities gather momentum, 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 March 31, 2017, the company had an accumulated deficit of
$66.2 million. We expect our losses to increase as we continue to
incur significant research and development and other expenses
related to our ongoing operations. Based on our research and
development plans and expectations related to the progress of the
company’s programs, we expect that cash and cash equivalents as of
March 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-2019, without giving
effect to any potential milestone payments or extension fees
received under our collaboration agreements with Novartis and
Regeneron.
Upcoming Events During the Second Quarter
2017
The company expects to make presentations at the following
upcoming scientific and investor conferences:
- The American Society of Gene & Cell Therapy, May 13,
Washington
- The TechConnect World Innovation Conference, May 16,
Washington
- Jefferies Healthcare Conference, June 6, New York City
- JMP Securities Life Sciences Conference, June 20, New York
City
- Goldman Sachs Third Annual Innovation Symposium, June 27, New
York City
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 non-human primate studies, and
clinical trials; the intellectual property position and strategy of
Intellia’s licensors; actions by government agencies; the impact of
our collaborations with 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 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 subsequent 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.
INTELLIA THERAPEUTICS, INC. |
CONSOLIDATED STATEMENTS OF OPERATIONS
(UNAUDITED) |
(Amounts in thousands except per share
data) |
|
|
|
Three Months Ended March 31, |
|
|
|
2017 |
|
|
|
2016 |
|
Collaboration revenue |
|
$ |
6,215 |
|
|
$ |
1,777 |
|
Operating
expenses: |
|
|
|
|
|
|
Research and development |
|
|
13,431 |
|
|
|
5,225 |
|
General and administrative |
|
|
5,732 |
|
|
|
3,246 |
|
Total operating expenses |
|
|
19,163 |
|
|
|
8,471 |
|
Operating
loss |
|
|
(12,948 |
) |
|
|
(6,694 |
) |
Interest
income |
|
|
317 |
|
|
|
5 |
|
Net
loss |
|
$ |
(12,631 |
) |
|
$ |
(6,689 |
) |
Net loss per share, basic and diluted |
|
$ |
(0.36 |
) |
|
$ |
(9.89 |
) |
Weighted average shares outstanding, basic and diluted |
|
|
34,723 |
|
|
|
676 |
|
INTELLIA THERAPEUTICS, INC. |
CONSOLIDATED BALANCE SHEET DATA
(UNAUDITED) |
(Amounts in thousands) |
|
|
|
|
|
|
|
March 31, 2017 |
|
December 31,
2016 |
Cash and cash equivalents |
|
|
|
$ |
257,551 |
|
$ |
273,064 |
Total
assets |
|
|
|
|
|
279,263 |
|
|
298,969 |
Total
liabilities |
|
|
|
|
|
79,380 |
|
|
89,132 |
Total
stockholders' equity |
|
|
|
|
199,883 |
|
|
209,837 |
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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