Intellia Therapeutics Announces Second Quarter 2019 Financial Results and Company Update
01 Août 2019 - 1:30PM
Intellia Therapeutics, Inc. (NASDAQ:NTLA), reported operational
highlights and financial results for the second quarter ended June
30, 2019.
“We continue to build our in vivo and engineered cell therapy
pipeline in support of our mission to develop curative genome
editing therapies for patients,” said Intellia President and Chief
Executive Officer John Leonard, M.D. “With IND-enabling toxicology
studies underway for NTLA-2001, we are well-positioned to submit an
IND in mid-2020 for the first systemically delivered
CRISPR/Cas9-based therapy for transthyretin amyloidosis.
Additionally, by the end of this year, we plan to select our first
engineered cell therapy development candidate to treat acute
myeloid leukemia.”
Second Quarter 2019 and More Recent Operational
Highlights
- ATTR Program: Intellia’s
first in vivo development candidate, NTLA-2001, is for the
treatment of transthyretin amyloidosis (ATTR). As part of an
ongoing durability study of the Company’s lead lipid nanoparticle
(LNP) formulation in support of NTLA-2001, we have demonstrated six
months of durable liver editing with sustained reduction of
circulating transthyretin (TTR) protein (average reduction >95%)
following a single dose in non-human primates (NHPs). The Company
announced today it has conducted its pre-investigational new drug
(IND) meeting with the U.S. Food and Drug Administration (FDA), has
initiated IND-enabling toxicology studies and expects to submit an
IND application for NTLA-2001 in mid-2020. Additionally, the
Company’s supply chain operations are in place to support
manufacturing of Phase 1 materials. NTLA-2001 is being co-developed
with Regeneron Pharmaceuticals, Inc. (Regeneron), with Intellia as
the lead development and commercialization party.
- AML Program: Intellia’s lead
T cell receptor (TCR)-based therapy uses engineered T cells to
target Wilms’ Tumor 1 (WT1) for the treatment of acute myeloid
leukemia (AML). During the second quarter of 2019, the Company and
its research collaborators at IRCCS Ospedale San Raffaele initiated
functional testing of multiple lead TCRs in patient-derived
xenograft models and remain on track to nominate a development
candidate by the end of 2019. In parallel, Intellia has begun
establishing manufacturing capabilities to support clinical
evaluation.
- In Vivo Insertion in NHPs: As
previously disclosed, Intellia demonstrated the first
CRISPR/Cas9-mediated, targeted transgene insertion in the liver of
NHPs, using Factor 9 (F9) as a model gene. F9 is a gene that
encodes for the Factor IX (FIX) protein, a blood-clotting protein
that is missing or defective in hemophilia B patients. The study is
a collaborative effort between Intellia and Regeneron, using
Intellia’s LNP delivery system of CRISPR/Cas9 with an
adeno-associated virus (AAV) containing a proprietary
bi-directional insertion template. Following a single dose of the
hybrid LNP-AAV delivery system containing an F9 DNA template, the
Company demonstrated that the circulating human FIX protein levels
achieved at day 14 were durable through the two months of completed
observation and were within the normal human range (source: Amiral
et al, Clin. Chem., 1984). Additionally, the NHP data expands on
the durability of clinically relevant human FIX protein levels
achieved in mice for over 12 months. Intellia is currently working
closely with Regeneron on moving the program forward and is also
independently evaluating the hybrid LNP-AAV delivery system for
targeted insertion across several other transgenes of interest in
an in vivo setting.
- Expanded Management Team: In May, Intellia
announced the appointment of Laura Sepp-Lorenzino, Ph.D., as
executive vice president and chief scientific officer. Dr.
Sepp-Lorenzino brings decades of leadership and research and
development experience and leads Intellia’s drug research
organization.
Upcoming Milestones
The Company has set forth the following for pipeline
progression:
- ATTR: ° Commence manufacturing of NTLA-2001 Phase 1
materials by the end of 2019 ° Submit IND
application for NTLA-2001 in mid-2020
- AML: ° Nominate first engineered cell therapy
development candidate by the end of 2019
- Platform: ° Present additional in vivo and
engineered cell therapy data at upcoming scientific conferences by
the end of 2019
Second Quarter 2019 Financial Results
- Cash Position: Cash, cash
equivalents and marketable securities were $275.8 million as of
June 30, 2019, compared to $314.1 million as of December 31, 2018.
The decrease was driven by cash used to fund operations of $59.8
million, which was offset in part by $7.9 million of net equity
proceeds raised from the Company's At The Market (“ATM”) offerings,
$7.0 million of funding received under the Novartis collaboration,
$4.1 million of ATTR cost reimbursements made by Regeneron, and
$2.6 million in proceeds from employee-based stock plans.
- ATM Proceeds Due: The Company
has $27.1 million in current assets on the balance sheet as of June
30, 2019 related to proceeds due from the ATM offerings that were
received in the first week of July 2019.
- Collaboration Revenue:
Collaboration revenue increased by $3.4 million to $11.1 million
during the second quarter of 2019, compared to $7.7 million during
the second quarter of 2018. The increase in collaboration revenue
in 2019 was primarily driven by amounts recognized from the
expansion of the existing collaboration with Novartis, as well as
by amounts recognized under the Company’s ATTR Co/Co agreement with
Regeneron. As previously disclosed, Regeneron is obligated to fund
approximately 50% of the development costs for the ATTR
program.
- R&D Expenses: Research
and development expenses increased by $2.0 million to $25.5 million
during the second quarter of 2019, compared to $23.5 million during
the second quarter of 2018. This increase was primarily driven by
the advancement of Intellia’s pipeline and the expansion of the
research and development organization.
- G&A Expenses: General and
administrative expenses increased by $5.3 million to $13.1 million
during the second quarter of 2019, compared to $7.8 million during
the second quarter of 2018. This increase was primarily driven by
personnel-related costs to support Intellia’s larger research and
development organization, and an increase in legal and intellectual
property (IP) costs.
- Net Loss: The Company’s net
loss was $25.7 million for the second quarter of 2019, compared to
$22.2 million during the second quarter of 2018.
Financial Guidance
Intellia expects that its cash, cash equivalents, marketable
securities and proceeds due from the ATM offerings as of June 30,
2019, as well as technology access and funding from Novartis and
Regeneron, will enable Intellia to fund its anticipated operating
expenses and capital expenditure requirements into the second half
of 2021. This expectation excludes 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 Company’s
base-case planning assumptions.
Conference Call to Discuss Second Quarter 2019
Earnings
The Company will discuss these results on a conference call
today, August 1, 2019, at 8 a.m. ET.
To join the call:
- U.S. callers should dial 866-575-6539 and use conference ID#
6223506, approximately five minutes before the call.
- International callers should dial +1 856-344-9299 and use
conference ID# 6223506, approximately five minutes before the
call.
A replay of the call will be available through the Events and
Presentations page of the Investor Relations section of the
Company’s website at www.intelliatx.com, beginning on August 1,
2019 at 12 p.m. ET.
About Intellia Therapeutics
Intellia Therapeutics is a leading genome editing company
focused on developing 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 StatementsThis press release
contains “forward-looking statements” of Intellia Therapeutics,
Inc. (“Intellia” or the “Company”) 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 beliefs and expectations
regarding its ability to conduct successful investigational new
drug application (“IND”)-enabling toxicology studies of NTLA-2001;
its plans to commence related manufacturing efforts of Phase 1
materials for NTLA-2001; its planned submission of an IND
application for NTLA-2001 in mid-2020; its plans to generate
preclinical and other data necessary to nominate a first engineered
cell therapy development candidate for its AML program by the end
of 2019; its plans to advance and complete preclinical studies,
including non-human primate studies for its ATTR program, AML
program and other in vivo and ex vivo programs; develop our
proprietary LNP/AAV hybrid delivery system to advance our complex
genome editing capabilities, such as gene insertion; its
presentation of additional data at upcoming scientific conferences
regarding CRISPR-mediated, targeted transgene insertion in the
liver of NHPs, using F9 as a model gene, via the Company’s
proprietary LNP-AAV delivery technology, and other preclinical data
by the end of 2019; the advancement and expansion of its
CRISPR/Cas9 technology to develop human therapeutic products, as
well as maintain and expand its related intellectual property
portfolio; the ability to demonstrate its platform’s modularity and
replicate or apply results achieved in preclinical studies,
including those in its ATTR and AML programs, in any future
studies, including human clinical trials; its ability to develop
other in vivo or ex vivo cell therapeutics of all types,
and those targeting WT1 in AML in particular, using CRISPR/Cas9
technology; the ability to continue its growth and realize the
anticipated contribution of the members of its board of directors
and executives to its operations and progress; the impact of its
collaborations on its development programs, including but not
limited to its collaboration with Regeneron Pharmaceuticals, Inc.;
statements regarding the timing of regulatory filings regarding its
development programs; its use of capital, including ATM
receivables, expenses, future accumulated deficit and other
financial results during the second quarter of 2019; and the
ability to fund operations into the second half of 2021.
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, including through our arbitration
proceedings against Caribou; risks related to Intellia’s
relationship with third parties, including our licensors; 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 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 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 June 30, |
|
Six Months Ended June 30, |
|
|
2019 |
|
|
|
2018 |
|
|
|
2019 |
|
|
|
2018 |
|
Collaboration
revenue |
$ |
11,118 |
|
|
$ |
7,677 |
|
|
$ |
21,551 |
|
|
$ |
15,146 |
|
Operating
expenses: |
|
|
|
|
|
|
|
|
Research and
development |
|
25,460 |
|
|
|
23,467 |
|
|
|
49,169 |
|
|
|
45,960 |
|
|
General and
administrative |
|
13,118 |
|
|
|
7,805 |
|
|
|
23,651 |
|
|
|
15,211 |
|
|
|
Total operating expenses |
|
38,578 |
|
|
|
31,272 |
|
|
|
72,820 |
|
|
|
61,171 |
|
Operating
loss |
|
(27,460 |
) |
|
|
(23,595 |
) |
|
|
(51,269 |
) |
|
|
(46,025 |
) |
Interest
income |
|
1,777 |
|
|
|
1,376 |
|
|
|
3,646 |
|
|
|
2,450 |
|
Net loss |
$ |
(25,683 |
) |
|
$ |
(22,219 |
) |
|
$ |
(47,623 |
) |
|
$ |
(43,575 |
) |
Net loss per
share, basic and diluted |
$ |
(0.56 |
) |
|
$ |
(0.52 |
) |
|
$ |
(1.05 |
) |
|
$ |
(1.03 |
) |
Weighted average shares outstanding, basic and diluted |
|
45,814 |
|
|
|
42,836 |
|
|
|
45,526 |
|
|
|
42,441 |
|
|
|
|
|
|
|
|
|
|
|
|
|
INTELLIA THERAPEUTICS, INC. |
CONSOLIDATED STATEMENTS OF
OPERATIONS
(UNAUDITED) |
(Amounts in thousands) |
|
|
|
|
|
June 30, 2019 |
|
December 31, 2018 |
Cash, cash equivalents and marketable securities |
$ |
275,838 |
|
$ |
314,059 |
Total assets |
|
352,441 |
|
|
347,315 |
Total liabilities |
|
75,575 |
|
|
69,395 |
Total stockholders'
equity |
|
276,866 |
|
|
277,920 |
|
|
|
|
|
|
|
|
Intellia Contacts:
Investors:Lina LiAssociate DirectorInvestor
Relations+1 857-706-1612lina.li@intelliatx.com
Media:Jennifer Mound SmoterSenior Vice
PresidentExternal Affairs & Communications+1
857-706-1071jenn.smoter@intelliatx.com
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