Intellia Therapeutics Announces First Quarter 2019 Financial Results and Company Update
May 02 2019 - 6:30AM
Intellia Therapeutics, Inc. (NASDAQ:NTLA), reported operational
highlights and financial results for the first quarter ended March
31, 2019. Additionally, the Company highlighted important corporate
milestones for 2019.
“2019 is off to a productive start in support of our mission to
advance genome editing to treat a range of severe and
life-threatening diseases. We are excited by our achievements in
gene knockout and insertion across both our in vivo and engineered
cell therapy efforts. In particular, we presented data at the 22nd
Annual Meeting of the American Society of Gene and Cell Therapy
showing unprecedented CRISPR-mediated, targeted gene insertion in
the liver of non-human primates, achieving normal circulating human
levels of protein production,” said Intellia President and Chief
Executive Officer John Leonard, M.D. “These achievements highlight
Intellia’s leadership in CRISPR/Cas9 genome editing as we advance
our pipeline towards the clinic. Looking ahead, we remain on track
to file an IND application next year for NTLA-2001, our lead in
vivo candidate for the treatment of transthyretin amyloidosis, and
expect to nominate a development candidate in our first engineered
cell therapy program for acute myeloid leukemia by year-end.”
First Quarter 2019 and More Recent Operational
Highlights
- ATTR Program: Intellia’s lead
candidate for the treatment of transthyretin amyloidosis (ATTR),
which demonstrated an average of >95% reduction in circulating
transthyretin (TTR) protein in non-human primates (NHPs), has been
nominated as the Company’s first in vivo development candidate to
advance into Investigational New Drug (IND)-enabling toxicology
studies. Preliminary results from substantially completed
dose-range finding (DRF) studies showed a favorable tolerability
profile; and data from multiple studies in NHPs demonstrated
durable liver editing with sustained reduction of circulating TTR
through 10 months of observation following a single dose.Today,
Intellia announced plans to begin IND-enabling toxicology studies
of NTLA-2001 in mid-2019 and that it remains on track to submit an
IND application in 2020. NTLA-2001 is being co-developed with
Regeneron Pharmaceuticals, Inc. (Regeneron), with Intellia as the
lead development and commercialization party.
- AML Program: Intellia and its
research collaborators at IRCCS Ospedale San Raffaele presented new
in vitro data at the 22nd Annual Meeting of the American Society of
Gene and Cell Therapy (ASGCT), showing that CRISPR/Cas9 editing
resulted in >98% knockout of endogenous T cell receptors (TCRs)
followed by insertion of Wilms’ Tumor 1 (WT1)-specific TCRs into
>95% of isolated T cells. In addition, the engineered T cells
were functional and capable of specifically killing high levels of
a panel of leukemic blasts from patients that expressed the WT1
epitope. Based on these results, Intellia has identified multiple
lead TCRs restricted to the HLA-A*02:01 allele to move into
functional testing in patient-derived xenograft models for an
autologous TCR-based therapy targeting WT1 for the treatment of
acute myeloid leukemia (AML). These studies are expected to begin
in mid-2019 and will inform the nomination of the Company’s first
engineered cell therapy development candidate by the end of
2019.
- In Vivo Insertion in
NHPs: At the 2019 ASGCT Meeting, Intellia
presented data demonstrating the first CRISPR-mediated, targeted
transgene insertion in the liver of NHPs, using Factor 9 (F9) as a
model gene. F9 is a gene that encodes for Factor IX (FIX) protein,
a blood-clotting protein that is missing or defective in hemophilia
B patients. In a collaboration between Intellia and Regeneron,
researchers combined Intellia’s lipid nanoparticle (LNP) delivery
system of CRISPR/Cas9 with an adeno-associated virus (AAV)
containing a proprietary bi-directional insertion template. NHP
data showed that a single administration achieved ~3-4 μg/mL of
circulating human FIX protein at day 14 and was sustained through
28 days (~3-5 μg/mL) of completed observation in an ongoing study.
The levels of circulating human FIX protein demonstrated in NHPs
correspond with the normal 3-5 μg/mL range of human FIX protein
levels (source: Amiral et al, Clin. Chem., 1984). The NHP data
shared also incorporates the improved CRISPR/Cas9 LNP identified
from the ATTR program and demonstrates the modularity of Intellia’s
platform to apply learnings to other programs. This data expands on
the clinically relevant human FIX protein levels achieved in mice,
first reported in October, which have remained stable through 10
months of observation.
- Modular In Vivo Knockout
Update: Today, at the 2019 ASGCT Meeting,
Intellia will present new data demonstrating that independent
CRISPR-mediated knockout of each of two targets of interest, either
lactate dehydrogenase A (Ldha) or hydroxyacid oxidase 1 (Hao1), via
the Company’s proprietary LNP delivery technology, results in a
durable, therapeutically relevant reduction of oxalate excretion in
a disease mouse model of primary hyperoxaluria type 1 (PH1).LDHA
and HAO1 are enzymes involved in oxalate production. In people with
PH1, mutations in a specific liver enzyme cause the production of a
surplus of oxalate, which can combine with calcium to form
insoluble deposits in the kidney and throughout the body, leading
to damage of the kidneys, heart, eyes and skeletal system. An
approximate 30% reduction in urinary oxalate in patients with PH1
is considered to be therapeutically relevant (source: Nephrology
Dialysis Transplantation 1999; 14:2556-2558). In collaboration with
the University of Alabama at Birmingham, Intellia researchers found
that a CRISPR-mediated knockout of the Ldha gene in a PH1 mouse
model disrupts LDHA protein production and reduces urinary oxalate
levels by 63%. Researchers also observed that a CRISPR-mediated
knockout of the Hao1 gene disrupts glycolate-to-glyoxylate
conversion, resulting in a urinary oxalate level reduction of 57%
in a PH1 mouse model. In each individual knockout approach, these
reduced levels of urinary oxalate were sustained for at least 15
weeks.Today’s presentation, titled “CRISPR/Cas9-Mediated Gene
Knockout to Address Primary Hyperoxaluria,” is accessible through
the Events and Presentations page of the Investor Relations section
of Intellia’s website.
Upcoming Milestones
The Company has set forth the following for 2019 pipeline
progression:
- ATTR:
- Initiate IND-enabling toxicology studies in mid-2019
- Commence manufacturing of NTLA-2001 Phase 1 materials
- AML:
- Initiate functional testing in patient-derived xenograft models
of multiple lead TCRs in mid-2019
- Nominate first engineered cell therapy development candidate by
the end of 2019
First Quarter 2019 Financial Results
- Cash Position: Cash, cash
equivalents and marketable securities were $296.6 million as of
March 31, 2019, compared to $314.1 million as of December 31, 2018.
The decrease was driven by cash used to fund operations of
approximately $29 million, which was offset in part by $6.0 million
of funding received under the Novartis collaboration, $3.6 million
of net equity proceeds raised from the Company's "At the Market"
(ATM) agreement, $1.5 million of ATTR cost reimbursements made by
Regeneron, and $0.4 million in proceeds from employee-based stock
plans.
- Collaboration Revenue:
Collaboration revenue increased by approximately $3.0 million to
$10.4 million during the first quarter of 2019, compared to $7.5
million during the first 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 $1.2 million to $23.7 million
during the first quarter of 2019, compared to $22.5 million during
the first quarter of 2018. 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.
- G&A Expenses: General and
administrative expenses increased by $3.1 million to $10.5 million
during the first quarter of 2019, compared to $7.4 million during
the first quarter of 2018. This increase was driven primarily by
employee and intellectual property (IP)-related expenses to support
Intellia’s growing research and development efforts.
- Net Loss: The Company’s net
loss was $21.9 million for the first quarter of 2019, compared to
$21.4 million during the first quarter of 2018.
Financial Guidance
Intellia expects that its cash, cash equivalents and marketable
securities as of March 31, 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 first 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 First Quarter 2019
Earnings
The Company will discuss these results on a conference call
today, May 2, 2019, 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 800-458-4148 and use conference
ID# 7725705, approximately five minutes before the call.
- International callers should click here to access dial-in
information and use conference ID# 7725705, approximately five
minutes before the call.
A replay of the call will be available on Intellia’s website,
beginning on May 2, 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 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 intellectual property portfolio; our
ability to achieve stable or effective genome editing; our ability
to administer our CRISPR/Cas9 product candidates; the potential
timing and advancement of our preclinical studies, including
continuing non-human primate studies for our ATTR program and other
programs, animal studies for our AML program, and clinical trials;
the timing and potential achievement of milestones to advance our
pipeline including nominating development candidates and filing
INDs; the modularity of our platform and our ability to replicate
or apply results achieved in our preclinical studies, including
those in our ATTR and AML programs, in any future studies,
including human clinical trials; the potential development of our
proprietary LNP/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 AML in particular, using CRISPR/Cas9 technology;
our ability to initiate and conduct successful IND-enabling
toxicology studies of NTLA-2001, our lead ATTR development
candidate, and subsequently submitting an IND application in 2020
that will be accepted by the regulatory agencies; our intent to
present additional data regarding CRISPR-mediated knockout of each
of two targets of interest, Ldha and Hao1, via the Company’s
proprietary LNP delivery technology, resulting in the reduction of
oxalate excretion, and other preclinical data during 2019 or
thereafter our plans to commence manufacturing efforts in 2019;
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;
our expectations regarding our uses of capital, expenses, future
accumulated deficit and other financial results during the first
quarter of 2019; and our ability to fund operations into the first
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; 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 March 31, |
|
|
|
|
|
|
|
|
|
2019 |
|
|
|
2018 |
|
|
|
|
Collaboration revenue |
|
|
$ |
10,433 |
|
|
$ |
7,469 |
|
|
|
|
Operating
expenses: |
|
|
|
|
|
|
|
|
|
Research
and development |
|
|
23,709 |
|
|
|
22,493 |
|
|
|
|
|
General and
administrative |
|
|
10,533 |
|
|
|
7,406 |
|
|
|
|
|
|
Total
operating expenses |
|
34,242 |
|
|
|
29,899 |
|
|
|
|
Operating
loss |
|
|
|
(23,809 |
) |
|
|
(22,430 |
) |
|
|
|
Interest
income |
|
|
|
1,869 |
|
|
|
1,074 |
|
|
|
|
Net
loss |
|
|
|
$ |
(21,940 |
) |
|
$ |
(21,356 |
) |
|
|
|
Net loss per share, basic and diluted |
$ |
(0.49 |
) |
|
$ |
(0.51 |
) |
|
|
|
Weighted average shares outstanding, basic and diluted |
|
45,234 |
|
|
|
42,043 |
|
|
|
|
INTELLIA THERAPEUTICS, INC. |
|
CONSOLIDATED BALANCE SHEET DATA
(UNAUDITED) |
|
(Amounts in thousands) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
March 31, 2019 |
|
December 31,
2018 |
|
Cash, cash equivalents and marketable securities |
|
$ |
296,582 |
|
$ |
314,059 |
|
Total
assets |
|
|
|
|
|
344,363 |
|
|
347,315 |
|
Total
liabilities |
|
|
|
|
|
80,025 |
|
|
69,395 |
|
Total
stockholders' equity |
|
|
|
|
264,338 |
|
|
277,920 |
|
|
|
|
|
|
|
|
|
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Intellia Contacts:
Investors:Lina LiSenior ManagerInvestor
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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