Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing
company focused on the development of curative therapeutics using
CRISPR/Cas9 technology, announced financial results and operational
progress for the first quarter of 2018.
John Leonard, M.D., was appointed Intellia’s President and Chief
Executive Officer in the first quarter of 2018, and one of his
first initiatives was to broaden the Company’s strategy. “We are
building the premier CRISPR-based genome editing company with
leading in vivo and ex vivo capabilities,” said Dr. Leonard. “We
are very pleased with the scientific data generated from our in
vivo non-human primate (NHP) studies, and the progress with our
modular, scalable lipid nanoparticle (LNP) delivery system has
allowed us to target a timeframe for our first Investigational New
Drug (IND) submission. As we continue to execute on our full
spectrum of in vivo and ex vivo genome editing platforms, we
will share progress on our differentiated, wholly owned ex vivo
approach, starting this month at the American Society of Gene and
Cell Therapy Annual Meeting.”
The Company announced today that it anticipates submitting an
IND application for its lead indication, transthyretin amyloidosis
(ATTR), by the end of 2019 and confirms plans to initiate
IND-enabling studies in mid-2018. Over the past six months, ongoing
NHP studies have demonstrated well-tolerated editing to
therapeutically relevant levels of transthyretin (TTR) protein
reduction (60 to 80 percent) after a single systemic administration
via LNP delivery to NHP hepatocytes. Rates of editing were durable
over the six-month period without re-dosing the animals. In support
of the proposed IND submission, Intellia has narrowed the field of
potential guides to its current development candidate for early
human trials. The guide-optimization process used high-throughput
screening to evaluate the entire TTR gene for those guides with
high levels of activity and undetectable off-target cutting. The
Company has completed studies to understand potential dosing
regimens and is continuing studies on durability of the effect,
both of which may expedite Phase I clinical trials. Intellia has
also developed an enhanced LNP formulation through optimization
campaigns that is currently being tested for multiple follow-on
liver indications, and anticipates that this modular approach may
minimize development timelines for each additional and subsequent
liver-targeted product candidate.
Intellia has also demonstrated continued progression of its
modular liver platform capability to knockout various targets of
interest in the livers of mice, including SERPINA1 for alpha-1
antitrypsin deficiency (AATD) and HAO1 for primary hyperoxaluria
type 1 (PH1), each of which has resulted in protein expression
reductions believed to be therapeutically relevant. This initial
knockout edit in AATD lays the groundwork for developing an
approach that restores production of the missing protein in AATD,
required for the amelioration of the disease.
The table below shows editing rates and corresponding protein
reductions in the livers of mice for ATTR, AATD and PH1. ATTR and
AATD both produce aberrant proteins hence treatment of these
conditions requires reductions in the level of the disease-causing
proteins. PH1 results from the low level activity of a particular
protein for which treatment requires reducing the levels of
substrate for that defective protein to metabolize, achieved by
knocking out the gene that encodes HAO1. In each of these three
cases, Intellia’s modular LNP delivery system achieved high levels
of reduction of the targeted protein. These initial editing rates
and corresponding protein reductions are evidence of Intellia’s
ability to successfully target monogenic liver diseases by knocking
out harmful genetic mutations.
Target |
Indication |
% editing in mouse model |
% protein reduction |
TTR |
Transthyretin amyloidosis |
70 |
% |
97 |
% |
SERPINA1 |
Alpha-1 antitrypsin deficiency |
85 |
% |
95 |
% |
HAO1 |
Primary hyperoxaluria type 1 |
74 |
% |
90 |
% |
Beyond the liver, the Company continues to advance its
application of CRISPR/Cas9 technology to the central nervous system
(CNS), including through its collaboration with Beverly Davidson,
Ph.D., of the Children’s Hospital of Philadelphia, who will share
updated LNP delivery data in a presentation at the American Society
of Gene and Cell Therapy Annual Meeting later this month.
In ex vivo applications, Intellia seeks to develop allogeneic
cellular therapies, which are cells derived from unmatched tissue
donors, which are modified outside of the human body to allow them
to be administered to an unrelated patient. This endeavor is
supported through multiple efforts, including recently acquired
access to intellectual property from researchers at the Karolinska
Institutet and Intellia’s collaboration with Ospedale San Raffaele,
announced in June of 2017.
In February of 2018, Cell Reports published Intellia’s first
peer-reviewed paper entitled “A single administration of
CRISPR/Cas9 lipid nanoparticles achieves robust and
persistent in vivo genome editing.” This landmark paper
documented Intellia’s delivery of Cas9 mRNA and single guide RNA
using its proprietary LNPs to achieve a 97 percent reduction in
mouse TTR protein levels in the liver, which was sustained for at
least 12 months.
During the course of 2018, Intellia plans to share additional
preclinical data on its TTR genome editing program, including the
achievement of a near ten-fold reduction in the required dose,
derived via improvements in potency, as well as other knockout
targets and data on delivery via LNPs to the CNS of NHPs.
Additionally, Intellia plans to share preclinical data on both
immuno-oncology and autoimmune disease targets in 2018.
First Quarter 2018 Financial Results
Collaboration Revenue
Collaboration revenue was $7.5 million for the first quarter of
2018, compared to $6.2 million during the first quarter of 2017.
The increase in collaboration revenue in 2018 was primarily driven
by amounts recognized under Intellia’s collaboration agreement with
Regeneron.
Since inception through March 31, 2018, the Company has received
$112.1 million in funding from the collaborations with Novartis and
Regeneron, excluding amounts received for equity investments, and
had an accounts receivable balance of $7.5 million at March 31,
2018.
Operating Expenses
Research and development expenses increased by $9.1 million to
$22.5 million during the first quarter of 2018, compared to $13.4
million during the first quarter of 2017. 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 and research materials such as reagents.
General and administrative expenses increased by $1.7 million to
$7.4 million during the first quarter of 2018, compared to $5.7
million during the first quarter of 2017. This increase was driven
primarily by increased salary and related headcount-based expenses
to support Intellia’s larger research and development organization,
public company compliance, and administrative obligations.
The Company’s net loss was $21.4 million for the first quarter
of 2018, compared to $12.6 million during the first quarter of
2017.
Balance Sheet
Cash and cash equivalents at March 31, 2018, were $327.8
million, compared to $340.7 million at December 31, 2017.
Financial Guidance
The Company’s primary uses of capital will continue to be for
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 March 31, 2018, the Company had an accumulated deficit of
$137.0 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 of the Company’s
programs, the Company expects that the cash and cash equivalents as
of March 31, 2018, 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.
Upcoming Events During the Second Quarter
2018
The Company expects to make presentations at the following
upcoming scientific and investor conferences:
- The American Society of Gene and Cell Therapy Annual Meeting,
May 16, Chicago
- Jefferies Global Health Care Conference, June 5, New York
City
- JMP Securities Life Sciences Conference, June 20, New York
City
About Intellia Therapeutics
Intellia Therapeutics is a leading genome editing company
focused on the development of 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 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 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 conduct
successful IND-enabling studies of a lead ATTR development
candidate and subsequently submitting an IND application by the end
of 2019 that will be accepted by the regulatory agencies; 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 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 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.
|
|
|
|
|
|
INTELLIA THERAPEUTICS, INC. |
CONSOLIDATED STATEMENTS OF OPERATIONS
(UNAUDITED) |
(Amounts in thousands, except per share
data) |
|
|
|
|
|
|
|
|
|
Three Months Ended March 31, |
|
|
|
|
2018 |
|
|
|
2017 |
|
Collaboration revenue |
$ |
7,469 |
|
|
$ |
6,215 |
|
Operating
expenses: |
|
|
|
|
Research
and development |
|
22,493 |
|
|
|
13,431 |
|
|
General and
administrative |
|
7,406 |
|
|
|
5,732 |
|
|
|
Total operating
expenses |
|
29,899 |
|
|
|
19,163 |
|
Operating
loss |
|
(22,430 |
) |
|
|
(12,948 |
) |
Interest
income |
|
1,074 |
|
|
|
317 |
|
Net
loss |
|
$ |
(21,356 |
) |
|
$ |
(12,631 |
) |
Net loss per share attributable to common stockholders, basic
and diluted |
$ |
(0.51 |
) |
|
$ |
(0.36 |
) |
Weighted average shares outstanding, basic and diluted |
|
42,043 |
|
|
|
34,723 |
|
|
|
|
|
|
|
|
|
|
|
|
|
INTELLIA THERAPEUTICS, INC. |
CONSOLIDATED BALANCE SHEET DATA
(UNAUDITED) |
(Amounts in thousands) |
|
|
|
|
|
|
|
|
|
|
|
|
|
March 31, 2018 |
|
December 31,
2017 |
Cash and
cash equivalents |
$ |
327,778 |
|
|
$ |
340,678 |
|
Total
assets |
|
359,545 |
|
|
|
376,235 |
|
Total
liabilities |
|
64,046 |
|
|
|
75,638 |
|
Total
stockholders' equity |
|
295,499 |
|
|
|
300,597 |
|
|
|
|
|
|
|
|
|
Intellia Contacts: |
|
Investor
Contact: |
Media
Contact: |
Lindsey Trickett |
Jennifer Mound
Smoter |
Vice President,
Investor Relations |
Senior Vice President,
External Affairs & Communications |
+1 857-285-6211 |
+1 857-706-1071 |
lindsey.trickett@intelliatx.com |
jenn.smoter@intelliatx.com |
|
|
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