Intellia Therapeutics Announces First Quarter 2020 Financial Results
07 May 2020 - 9:30PM
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, today reported
operational highlights and financial results for the first quarter
ended March 31, 2020.
“At Intellia, our mission is to develop curative,
CRISPR/Cas9-based treatments for severe diseases. I am grateful to
our team for remaining steadfast in our commitment to patients and
putting us in the position to deliver on our goals despite the
challenging circumstances presented by the global COVID-19
pandemic,” said Intellia President and Chief Executive Officer John
Leonard, M.D. “We look forward to advancing NTLA-2001 into clinical
testing and dosing ATTR patients with the first systemically
delivered CRISPR/Cas9-based therapy in the second half of 2020.
Additionally, we continue to advance our full-spectrum pipeline,
including IND-enabling activities for NTLA-5001, our engineered
TCR-T cell therapy candidate for AML, and the nomination of
NTLA-2002 as our development candidate for the treatment of
HAE.”
First Quarter 2020 and Recent Operational
Highlights
- ATTR Program: Intellia’s lead candidate,
NTLA-2001, is designed to apply its in vivo liver knockout approach
for the treatment of transthyretin amyloidosis (ATTR) in a single
course of treatment. The Company has manufactured clinical-scale
materials for a Phase 1 study of NTLA-2001 and remains on track to
submit an Investigational New Drug application (IND) or
IND-equivalent for this program in mid-2020. NTLA-2001 is
anticipated to be the first systemically delivered CRISPR/Cas9
therapy to enter the clinic. Subject to the impact of COVID-19, the
Company plans to dose the first patient in the second half of 2020.
NTLA-2001 is part of a co-development/co-promotion (Co/Co)
agreement between Intellia, which is the lead development and
commercialization party, and Regeneron Pharmaceuticals, Inc.
(Regeneron).
- AML Program: NTLA-5001 for the treatment of
acute myeloid leukemia (AML) is Intellia’s first T cell receptor
(TCR)-T cell development candidate, which targets the Wilms’ Tumor
1 (WT1) antigen. The Company’s approach aims to develop a broadly
applicable treatment for AML patients, regardless of mutational
subtypes of a patient’s leukemia. As presented at Keystone
Symposia’s Engineering the Genome Conference in February 2020,
Intellia demonstrated that its CRISPR/Cas9-enabled approach results
in an engineered T cell product capable of specific and potent
killing of primary AML blasts. Intellia is continuing to advance
IND-enabling activities, including process development to support
clinical T cell manufacturing. The Company is on track to submit an
IND or IND-equivalent for NTLA-5001 in the first half of 2021.
Additional efforts are underway to evaluate the potential use of
the WT1-targeted TCR construct to treat solid tumors.
- HAE Program: Intellia has nominated its third
development candidate, NTLA-2002 for the treatment of hereditary
angioedema (HAE). Building on the modular lipid nanoparticle (LNP)
delivery system developed for the ATTR program, NTLA-2002 is
designed to knock out the prekallikrein B1 (KLKB1) gene in the
liver after a single course of treatment. As part of an ongoing
durability study of its lead LNP formulation in support of
NTLA-2002, Intellia has now demonstrated six months of sustained
therapeutically relevant reduction of serum kallikrein levels and
activity following a single dose in non-human primates. Intellia
expects to submit an IND or IND-equivalent for NTLA-2002 in the
second half of 2021. NTLA-2002 is subject to an option by Regeneron
to enter into a Co/Co agreement, which must be exercised within a
limited time period after development candidate selection. Intellia
would be the lead party if the option is exercised.
- Modular Platform: Intellia continues to
advance its differentiated genome editing and delivery strategies,
including targeted insertion and consecutive editing, across its in
vivo and ex vivo efforts. These platform capabilities enable the
removal and/or restoration of a gene’s function in developing
treatments for life-threatening diseases. The in vivo research
builds on data previously shared by the Company demonstrating the
first CRISPR-mediated, targeted transgene insertion in the liver of
non-human primates, which generated circulating human FIX protein
at or above normal levels. At the upcoming American Society of Gene
and Cell Therapy (ASGCT) Annual Meeting, taking place virtually
from May 12 – 15, 2020, the Company will present preclinical data
related to certain programs and ex vivo platform capabilities. This
will include an oral presentation featuring Intellia’s enhanced
CRISPR/Cas9-based T cell engineering platform capable of highly
efficient editing of multiple genes with levels of translocations
indistinguishable from background levels, and favorable cell
product attributes including high viability and expansion
potential. This improved T cell engineering process has been
incorporated into the NTLA-5001 program.
- Novartis Collaboration: As announced in
March 2020, the U.S. Food and Drug Administration (FDA)
has accepted the IND application submitted by Novartis Institutes
for BioMedical Research (Novartis), for a CRISPR/Cas9-based
engineered cell therapy for the treatment of sickle cell disease
(SCD) developed under collaboration with Intellia. The Phase 1/2
clinical trial will begin investigating OTQ923 in adult patients
with severe complications of SCD. Novartis’ IND application
triggered a milestone payment to Intellia, and the Company is
eligible to receive additional downstream success-based milestones
and royalties.
Upcoming Events
The Company will participate in the following events during the
second quarter of 2020:
- ASGCT 23rd Annual Meeting, May 12 – 15, Virtual
- Jefferies Global Healthcare Conference, June 3, Virtual
Upcoming Milestones
The Company has set forth the following for pipeline
progression:
- ATTR:
- Submit an IND or IND-equivalent for NTLA-2001 in mid-2020
- Subject to the impact of COVID-19, plan to dose first patient
in 2H 2020
- AML:
- Submit an IND or IND-equivalent for NTLA-5001 in 1H 2021
- HAE:
- Submit an IND or IND-equivalent for NTLA-2002 in 2H 2021
- Modular Platform Advancements:
- Present preclinical data at upcoming ASGCT Annual Meeting
First Quarter 2020 Financial Results
- Cash Position: Cash, cash
equivalents and marketable securities were $250.3 million as of
March 31, 2020, compared to $284.5 million as of December 31, 2019.
The decrease was driven by cash used to fund operations of
approximately $40.1 million, which was offset in part by $4.5
million of net equity proceeds raised from the Company's “At the
Market” (ATM) agreement, $1.0 million of funding received under the
Novartis collaboration, and $0.3 million in proceeds from
employee-based stock plans.
- Collaboration Revenue:
Collaboration revenue increased by $2.5 million to $12.9 million
during the first quarter of 2020, compared to $10.4 million during
the first quarter of 2019. The increase in collaboration revenue in
2020 was mainly driven by the $5.0 million milestone payment earned
from Novartis for the IND submission of OTQ923.
- R&D Expenses: Research
and development expenses increased by approximately $10.9 million
to $34.7 million during the first quarter of 2020, compared to
$23.7 million during the first quarter of 2019. This increase was
primarily driven by the advancement of our lead 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 $0.8 million to $11.3 million
during the first quarter of 2020, compared to $10.5 million during
the first quarter of 2019. This increase was primarily driven by an
increase in intellectual property related expenses due to increased
patent-related activity.
- Net Loss: The Company’s net
loss was $31.8 million for the first quarter of 2020, compared to
$21.9 million during the first quarter of 2019.
Financial Guidance
Intellia expects that its cash, cash equivalents and marketable
securities as of March 31, 2020 will enable the Company to fund its
anticipated operating expenses and capital expenditure requirements
at least through the end of 2021. This expectation excludes any
strategic use of capital not currently in the Company’s base-case
planning assumptions.
Conference Call to Discuss First Quarter 2020
Earnings
The Company will discuss these results on a conference call
today, May 7, 2020, at 8 a.m. ET.
To join the call:
- U.S. callers should dial 1-877-317-6789 and use conference ID#
10141541, approximately five minutes before the call.
- International callers should dial 1-412-317-6789 and use
conference ID# 10141541, approximately five minutes before the
call.
A replay of the call will be available through the Events and
Presentations page of the Investors & Media section on
Intellia’s website at www.intelliatx.com, beginning on May 7, 2020
at 12 p.m. ET.
About Intellia TherapeuticsIntellia
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” 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: planned submission of an investigational new drug
(“IND”) or equivalent regulatory filing for NTLA-2001 for the
treatment of transthyretin amyloidosis (“ATTR”) in mid-2020 and its
planned dosing of first patients in the second half of 2020; plans
to complete manufacturing activities and submit an IND application
or equivalent regulatory filing for NTLA-5001, its first T cell
receptor (“TCR”)-directed engineered cell therapy development
candidate for its acute myeloid leukemia (“AML”) program in the
first half of 2021; plans to submit an IND application or
equivalent regulatory filing for NTLA-2002, a development candidate
for its hereditary angioedema (“HAE”) program in the second half of
2021; plans to advance and complete preclinical studies, including
non-human primate studies for its ATTR program, AML program, HAE
program and other in vivo and ex vivo programs; development of a
proprietary LNP/AAV hybrid delivery system, as well as its modular
platform to advance its complex genome editing capabilities, such
as gene insertion; presentation of additional data at upcoming
scientific conferences, and other preclinical data in 2020;
advancement and expansion of its CRISPR/Cas9 technology to develop
human therapeutic products, as well as its ability to maintain and
expand its related intellectual property portfolio; ability to
demonstrate its platform’s modularity and replicate or apply
results achieved in preclinical studies, including those in its
ATTR, AML and HAE programs, in any future studies, including human
clinical trials; 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; expectations of the
potential impact of the coronavirus disease 2019 pandemic on
strategy, future operations and timing of its clinical trials or
IND submissions; 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; ability to optimize
the impact of collaborations on its development programs, including
but not limited to its collaborations with Novartis or Regeneron,
and a decision by Regeneron on whether to enter into a
co-development and co-promotion agreement for the HAE program;
statements regarding the timing of regulatory filings, actions and
decisions regarding its development programs; use of capital,
expenses, future accumulated deficit and other 2020 financial
results or in the future; and ability to fund operations through
the end 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 its
intellectual property position; risks related to Intellia’s
relationship with third parties, including its licensors and
licensees; risks related to the ability of its licensors to protect
and maintain their intellectual property position; uncertainties
related to the initiation and conduct of studies and other
development requirements for its 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 or clinical 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 Mar 31, |
|
|
|
|
2020 |
|
|
|
2019 |
|
Collaboration
revenue |
$ |
12,916 |
|
|
$ |
10,433 |
|
Operating
expenses: |
|
|
|
|
Research and
development |
|
34,650 |
|
|
|
23,709 |
|
|
General and
administrative |
|
11,314 |
|
|
|
10,533 |
|
|
|
Total operating expenses |
|
45,964 |
|
|
|
34,242 |
|
Operating
loss |
|
(33,048 |
) |
|
|
(23,809 |
) |
Interest
income |
|
1,242 |
|
|
|
1,869 |
|
Net loss |
|
$ |
(31,806 |
) |
|
$ |
(21,940 |
) |
Net loss per
share, basic and diluted |
$ |
(0.63 |
) |
|
$ |
(0.49 |
) |
Weighted average
shares outstanding, basic and diluted |
|
50,491 |
|
|
|
45,234 |
|
INTELLIA THERAPEUTICS, INC. |
CONSOLIDATED STATEMENTS OF OPERATIONS
(UNAUDITED) |
(Amounts in thousands) |
|
|
|
|
|
|
|
Mar 31, 2020 |
|
December 31, 2019 |
Cash, cash equivalents and marketable securities |
$ |
250,259 |
|
|
$ |
284,472 |
|
Total
assets |
|
314,752 |
|
|
|
334,280 |
|
Total
liabilities |
|
66,993 |
|
|
|
64,399 |
|
Total
stockholders' equity |
|
247,759 |
|
|
|
269,881 |
|
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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