Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing
company focused on developing curative therapeutics using
CRISPR/Cas9 technology both in vivo and ex vivo, announced
financial results and operational developments for the third
quarter of 2018.
“We made excellent progress across our in vivo and ex vivo
programs in the third quarter. In our ATTR program, Intellia
previously reported that our proprietary LNP delivery technology
could edit genes in the liver of non-human primates sufficiently to
reduce TTR protein levels to a potential therapeutic range. Our
continued in vivo delivery enhancement efforts have now produced
striking improvements over these liver editing and protein
knockdown levels. Data from the initial application of these
enhancements in our ATTR program suggest that we may be able to
double our previously reported liver editing results and more
significantly reduce circulating protein beyond recognized
therapeutic levels, at a lower, well-tolerated dose. We are
applying these enhancements to develop a superior ATTR development
candidate and we believe we can leverage these enhancements to
significantly improve the performance of our development candidates
for follow-on in vivo programs,” said Intellia President and Chief
Executive Officer John Leonard, M.D.
“Furthermore, in our insertion programs, we successfully
introduced functioning genes in mice using our modular LNP delivery
system of CRISPR/Cas9 in combination with our proprietary AAV
insertion templates developed in collaboration with Regeneron. In
our presentation at ESGCT, Intellia was the first company to
demonstrate technology that can insert genes in mice to produce,
and fine tune, protein levels up to and greater than those required
for normal human physiology.
“In parallel to these in vivo achievements, we are rapidly
advancing our ex vivo immuno-oncology efforts, beginning with the
development of best-in-class CRISPR-edited T cells for WT1,” added
Dr. Leonard.
Third Quarter 2018 Operational Highlights and Recent
Corporate Developments Include:
Transthyretin Amyloidosis Program
Enhancements
Intellia announced today results from its transthyretin
amyloidosis (ATTR) non-human primate (NHP) studies, conducted in
collaboration with Regeneron Pharmaceuticals, Inc., related to its
enhancements of the cargo components of its lipid nanoparticle
(LNP)-based delivery system. These novel component enhancements,
which are part of the ongoing development of its proprietary and
modular in vivo delivery platform, have produced unprecedented
results, achieving up to 78 percent (mean of 59 percent) liver
editing in our most recent NHP study. The corresponding
transthyretin (TTR) protein reduction at 21 days showed a decrease
from baseline of up to 96 percent (mean reduction of 78 percent)
after a single dose. This substantially improved level of liver
editing, achieved with a lower dose and well-tolerated safety
profile, compares with mean editing levels of 34 percent in
Intellia’s previously reported NHP studies.
Based on these new data, the Company is pursuing confirmatory
studies with the goal of integrating enhanced cargo components in
its Investigational New Drug (IND)-enabling studies and submission
of an IND (previously planned for the end of 2019, but now targeted
for 2020) for ATTR. In addition, the Company intends to apply these
technology improvements to the rest of its in vivo product
pipeline.
“We are elated to have achieved such impressive and compelling
editing and protein knockdown results in our NHP studies. We
believe that this approach, while introducing a relatively short
delay to our previous IND timeline, will ultimately yield the best
possible treatment option for patients – one that we hope will
clearly advance the standard of care well beyond approved and
potential therapies for the treatment of ATTR,” added Dr.
Leonard.
Advancing Complex Genome Editing Capabilities
Intellia advanced its complex genome editing capabilities with the
first robust demonstration of CRISPR-mediated, targeted insertion
of transgenes in the liver of mice. The Company used its modular
LNP delivery system of CRISPR/Cas9 in combination with Intellia’s
proprietary modular adeno-associated virus (AAV) to insert donor
template DNA into the albumin locus of mice. In collaboration with
Regeneron, the Company used F9 as a model gene, which encodes
Factor IX (FIX) protein, which is the clotting factor deficient in
patients with hemophilia B. Using a proprietary bi-directional
template platform technology, researchers showed that they can
detect hybrid mAlb-hF9 transcripts in over 50 percent of
hepatocytes following a single dose and measured circulating human
FIX protein levels of more than 30,000 ng/mL. These levels are
higher than those required in a clinical setting and correspond to
levels predicted to be up to 40 to 300 times higher than those
capable of preventing spontaneous bleeding episodes in hemophilia B
patients, relative to wildtype or a hyperfunctional version of F9,
respectively (sources: George, et al, NEJM, 2017; Simioni et al,
NEJM, 2009). Furthermore, the Company observed that varying either
the LNP or AAV dose modulated FIX levels. Protein levels remained
stable after dosing throughout the 12-week observation
period.
Intellia also applied the hybrid LNP-AAV delivery approach to
its wholly owned in vivo preclinical program in alpha-1 antitrypsin
deficiency (AATD). Alpha-1 antitrypsin (AAT), a protein that
protects the lungs and is mutated or absent in patients with AATD,
is produced by the SERPINA1 gene. To produce AAT protein, Intellia
combined CRISPR/Cas9 delivery by LNP with the SERPINA1 DNA template
delivered by AAV into the albumin locus. The targeted insertion
generated gene expression levels in mice that correspond to levels
that protect against the loss of pulmonary capacity in humans. This
milestone underscores the utility of Intellia’s modular LNP-based
delivery system and shows significant progress in developing an in
vivo genome editing solution for AATD.
Progressing Transgenic T Cell Receptor
TechnologyIntellia and its research collaborator, Ospedale
San Raffaele (OSR), presented data at the 26th Annual Congress of
the European Society of Gene and Cell Therapy (ESGCT) showing
progression in the Company’s lead ex vivo program for the treatment
of acute myeloid leukemia (AML), utilizing transgenic T cell
receptor (TCR) technology. In vitro data demonstrated that
CRISPR/Cas9 editing resulted in over 90 percent knockout of
endogenous TCRs and insertion of Wilms’ Tumor 1 (WT1)
epitope-specific TCRs. The resulting engineered T cells were fully
functional and killed a significant percentage of leukemic blasts.
The Company continues to advance its first ex vivo development
candidate, which is undergoing in vitro and in vivo functional
testing.
Expanded Board of Directors and Management
TeamIntellia announced today the appointment of Jesse
Goodman, M.D., to its board of directors. Dr. Goodman brings more
than three decades of expertise in medical research and public
health as a professor, practicing clinician and former director of
the U.S. Food and Drug Administration’s (FDA) Center for Biologics
Evaluation and Research (CBER).
“We are thrilled to have someone with the broad scientific,
public health, regulatory and clinical credentials of Jesse join
Intellia’s board of directors,” said Intellia Chairman Perry
Karsen. “During his time at the FDA, Jesse demonstrated exceptional
leadership in ensuring that the agency and companies could work
together to advance scientific innovation and responsibly enhance
patient access to safe and effective therapies. We expect his
contributions will be invaluable as Intellia begins transitioning
to a biotech company approaching clinical trials with
patients.”
Dr. Goodman is currently a professor of medicine at Georgetown
University, where he directs the Center on Medical Product Access,
Safety and Stewardship (COMPASS). As an attending physician in
infectious diseases, he also is an active clinician and educator.
Additionally, he serves as an independent non-executive director
for GlaxoSmithKline plc.; as president and trustee of the U.S.
Pharmacopeial Convention; and on the Regulatory Working Group of
the Coalition on Epidemic Preparedness Innovation (CEPI). Dr.
Goodman was chief scientist at the FDA from 2009 through 2014,
during which time he led preparations for and responses to major
public health threats, including emerging infectious diseases,
disasters and terrorism. He previously also served as the FDA’s
deputy commissioner for science and public health and as director
of CBER. Dr. Goodman has a bachelor of arts in biology from Harvard
University; earned a master’s in public health from the University
of Minnesota; and received his doctor of medicine from the Albert
Einstein College of Medicine. He completed his residency and
fellowship training at the Hospital of the University of
Pennsylvania and at the University of California at Los Angeles.
Dr. Goodman is board-certified in internal medicine, infectious
diseases and oncology, and has been elected to the American Society
for Clinical Investigation and to the U.S. National Academy of
Medicine.
Earlier this week, Intellia announced that Glenn Goddard had
been named its executive vice president and chief financial
officer. Mr. Goddard joins as a member of Intellia’s executive
management team, and will oversee all financial functions and
investor relations, as well as information technologies and
facilities.
Strengthened Intellectual Property Position
Yesterday, the U.S. Patent and Trademark Office (USPTO) granted
U.S. Patent No. 10,113,167 to The Regents of the University of
California, the University of Vienna and Emmanuelle Charpentier,
Ph.D., co-owners of foundational intellectual property relating to
CRISPR/Cas9 genome editing technology. The patent covers optimized
guide RNA formats (including single-guide and dual-guide formats)
that may be used in any environment, including eukaryotic cells
(such as human, animal and plant cells). The optimized formats
modify the part of a guide RNA that interacts with the CRISPR/Cas9
nuclease. The previously awarded U.S. Patent No. 10,000,772 covers
methods of using optimized guide RNAs to edit genes in certain
environments, including in eukaryotic cells. Intellia sublicenses
this intellectual property from The Regents of the University of
California and the University of Vienna under a license agreement
with Caribou Biosciences Inc.
Third Quarter 2018 Financial Results
Collaboration Revenue
Collaboration revenue was $7.4 million for the third quarter of
2018, compared to $7.3 million during the third 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 September 30, 2018, the Company has
received $122.7 million in funding from the collaborations with
Novartis Institutes for Biomedical Research, Inc. (Novartis) and
Regeneron, excluding amounts received for equity investments, and
had an accounts receivable balance of $2.8 million as of September
30, 2018.
Operating Expenses
Research and development expenses increased by $5.8 million to
$23.2 million during the third quarter of 2018, compared to $17.5
million during the third 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 $2.6 million to
$8.3 million during the third quarter of 2018, compared to $5.7
million during the third 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
and administrative obligations.
The Company’s net loss was $22.7 million for the third quarter
of 2018, compared to $15.4 million during the third quarter of
2017.
Balance Sheet
Cash and cash equivalents were $293.2 million as of September
30, 2018, compared to $340.7 million as of 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,
intellectual property related costs and general operating
costs.
As of September 30, 2018, the Company had an accumulated deficit
of $182.0 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 September 30, 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.
Conference Call to Discuss Third Quarter 2018 Earnings
and Corporate Developments
The Company will present third quarter 2018 results and
corporate developments in a conference call on Oct. 31, 2018 at 8
a.m. ET. The investor presentation may be downloaded starting at
7:30 a.m. ET from the Events and Presentations page of the Investor
Relations section of Intellia’s website at intelliatx.com
To join the call:
- U.S. callers should dial 866-548-4713 and use conference ID#
5893807, approximately five minutes before the call.
- International callers should click here to access dial-in
information and use conference ID# 5893807, approximately five
minutes before the call.
A replay of the call will be available on Intellia’s website,
beginning on Oct. 31, 2018 at 12 p.m. ET. 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 improved cell therapies that can treat cancer
and immunological diseases, or can replace 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 Therapeutics, Inc. (“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 or effective genome
editing; our ability to administer multiple doses of our
CRISPR/Cas9 product candidates; the potential timing and
advancement of our preclinical studies, including continuing
non-human primate studies for our Transthyretin Amyloidosis
(“ATTR”) program and other programs (such as alpha-1 antitrypsin
deficiency (AATD)), and clinical trials; the timing and potential
achievement of milestones to advance our pipeline including filing
INDs; our ability to replicate results achieved in our preclinical
studies, including those in our ATTR, AATD and Wilms’ Tumor 1 (WT1)
programs, in any future studies, including human clinical trials;
the potential development of our proprietary lipid nanoparticle
(LNP)- adeno-associated virus (AAV) hybrid delivery system to
advance our complex genome editing capabilities; the potential
development of other in vivo or ex vivo cell therapeutics
of all types, and those targeting WT1 in particular, using
CRISPR/Cas9 technology; our ability to conduct successful
Investigational New Drug (“IND”)-enabling studies of a lead ATTR
development candidate and subsequently submitting an IND
application 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 or thereafter; our
ability to advance a development candidate for an in vivo second
indication by late 2018 or thereafter; the intellectual property
position and strategy of Intellia’s licensors or other parties from
which it derives rights, as well as third-parties and competitors;
actions by government agencies; our growth as a company and the
anticipated contribution of the members of our board of directors
and our executives to our operations and progress; 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 and quarterly reports 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 September 30, |
|
Nine Months Ended September 30, |
|
|
|
|
|
|
2018 |
|
2017 |
|
2018 |
|
2017 |
|
Collaboration revenue |
|
|
$ |
7,408 |
|
|
$ |
7,317 |
|
|
$ |
22,554 |
|
|
$ |
19,449 |
|
|
Operating
expenses: |
|
|
|
|
|
|
|
|
|
|
|
Research
and development |
|
|
23,237 |
|
|
|
17,481 |
|
|
|
69,197 |
|
|
|
46,477 |
|
|
|
General and
administrative |
|
|
8,270 |
|
|
|
5,711 |
|
|
|
23,481 |
|
|
|
17,812 |
|
|
|
|
Total
operating expenses |
|
31,507 |
|
|
|
23,192 |
|
|
|
92,678 |
|
|
|
64,289 |
|
|
Operating
loss |
|
|
|
(24,099 |
) |
|
|
(15,875 |
) |
|
|
(70,124 |
) |
|
|
(44,840 |
) |
|
Interest
income |
|
|
|
1,397 |
|
|
|
519 |
|
|
|
3,847 |
|
|
|
1,260 |
|
|
Net loss |
|
|
|
$ |
(22,702 |
) |
|
$ |
(15,356 |
) |
|
$ |
(66,277 |
) |
|
$ |
(43,580 |
) |
|
Net loss per share, basic and diluted |
$ |
(0.53 |
) |
|
$ |
(0.44 |
) |
|
$ |
(1.55 |
) |
|
$ |
(1.25 |
) |
|
Weighted average shares outstanding, basic and diluted |
|
43,161 |
|
|
|
35,189 |
|
|
|
42,684 |
|
|
|
34,945 |
|
|
|
|
|
|
|
|
|
|
|
|
INTELLIA THERAPEUTICS, INC. |
|
CONSOLIDATED BALANCE SHEET DATA
(UNAUDITED) |
|
(Amounts in thousands) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
September 30,
2018 |
|
December 31,
2017 |
|
Cash and cash equivalents |
|
|
|
$ |
293,248 |
|
$ |
340,678 |
|
Total assets |
|
|
|
|
|
321,233 |
|
|
376,235 |
|
Total
liabilities |
|
|
|
|
|
57,963 |
|
|
75,638 |
|
Total
stockholders' equity |
|
|
|
|
263,270 |
|
|
300,597 |
|
|
|
|
|
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Intellia Contacts:
Investors:Lindsey TrickettVice
PresidentInvestor Relations+1
857-285-6211lindsey.trickett@intelliatx.com
Media:Jennifer Mound Smoter
Senior Vice
President
External Affairs
& Communications
+1
857-706-1071
jenn.smoter@intelliatx.com
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