Intellia Therapeutics Announces Fourth Quarter and Full-Year 2020 Financial Results
25 February 2021 - 11:30PM
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 fourth quarter
and year ended December 31, 2020.
“Intellia’s achievements in 2020 reflect important progress on
both our full-spectrum strategy and our mission to deliver curative
genome editing treatments for people with severe diseases. Dosing
our first patient with NTLA-2001, the first-ever systemically
delivered CRISPR-based therapy, was a major milestone for our team,
as we completed our transition to a clinical-stage company in the
fourth quarter,” said Intellia President and Chief Executive
Officer, John Leonard, M.D. “Looking ahead, we are focused on our
three core priorities for 2021: clinical validation of our approach
with NTLA-2001, advancement and expansion of our in vivo and ex
vivo pipeline, and continued platform innovation. We have made
steady progress in our global Phase 1 study of NTLA-2001 and look
forward to sharing our first clinical data this year. Additionally,
we are on track to submit first-in-human regulatory applications to
begin clinical studies of NTLA-5001 for AML and NTLA-2002 for HAE,
and we plan to nominate at least one new development candidate from
our research portfolio.”
Fourth Quarter 2020 and Recent Operational
Highlights
- NTLA-2001 for
ATTR: NTLA-2001 is the first systemically delivered
CRISPR-based therapy dosed in a patient and could potentially be
the first curative treatment for ATTR. By applying the Company’s in
vivo lipid nanoparticle (LNP) delivery technology, NTLA-2001 offers
the possibility of halting and reversing the disease with potent,
lifelong transthyretin (TTR) protein reduction after a single
course of treatment. NTLA-2001 is part of a
co-development/co-promotion agreement between Intellia, the lead
party, and Regeneron Pharmaceuticals, Inc. (Regeneron).
- The Company anticipates reporting
interim clinical data from the Phase 1 study this year. These
results are expected to characterize the emerging safety and
activity profile of NTLA-2001 at the initial dose levels.
- In November, Intellia dosed the
first patient in its global Phase 1 study evaluating NTLA-2001 in
adults with hereditary ATTR with polyneuropathy (hATTR-PN). The
Company continues to enroll patients in the study and is submitting
additional regulatory applications in other countries as part of
its ongoing, global development strategy.
- Intellia intends to evaluate
NTLA-2001 in a broader ATTR population of both polyneuropathy and
cardiomyopathy patients following its Phase 1 safety assessment and
dose optimization.
- NTLA-5001 for AML:
NTLA-5001 is a potential best-in-class engineered T cell therapy
designed to treat all genetic subtypes of AML. This investigational
candidate is an autologous T cell receptor (TCR)-T cell therapy
targeting the Wilms’ Tumor 1 (WT1) antigen utilizing Intellia’s
proprietary cell engineering process.
- Intellia plans to submit an
Investigational New Drug (IND) application or equivalent regulatory
application for NTLA-5001 in mid-2021. This first-in-human trial is
expected to evaluate the safety and activity of NTLA-5001 in
patients with persistent or recurrent AML who have previously
received first-line therapies.
- NTLA-5001 shows high anti-tumor
activity in proof-of-concept mouse models of acute leukemias. The
preclinical data presented at the American Society of Hematology
(ASH) Annual Meeting in December highlighted faster expansion and
superior function of T cells manufactured by Intellia’s proprietary
approach, compared to standard T cell engineering approaches
currently in use.
- The Company is also evaluating the
potential use of NTLA-5001 to treat WT1-positive solid tumors in
preclinical studies.
- NTLA-2002 for HAE:
NTLA-2002 aims to prevent attacks for people living with HAE after
a single course of treatment. Intellia is applying its modular LNP
delivery system to develop NTLA-2002 to knock out the KLKB1 gene in
the liver to permanently reduce plasma kallikrein activity. This
approach is expected to provide continuous suppression of
kallikrein activity and eliminate the significant treatment burden
associated with currently available therapies for HAE patients.
- Intellia commenced clinical
manufacturing activities to support the Company’s plans to submit
an IND or equivalent regulatory application in the second half of
2021.
- The Company is applying insights
gained from NTLA-2001 to expedite clinical development of
NTLA-2002. The first-in-human trial is expected to evaluate safety,
tolerability and activity in patients with HAE.
- The Company plans to present
additional preclinical results in support of NTLA-2002 at the
upcoming American Academy of Allergy, Asthma & Immunology
(AAAAI) 2021 Annual Meeting, taking place virtually from February
26 – March 1, 2021.
- Modular Platform and
Pipeline Expansion: Intellia is advancing its modular
platform technologies to broaden the in vivo and ex vivo
applications of genome editing. This includes developing
capabilities for innovative CRISPR/Cas9-mediated in vivo editing in
multiple tissue types, targeted transgene insertion and an
allogeneic approach for the development of “off-the-shelf” T cell
therapies. These efforts will support new therapeutic candidates
for genetic diseases requiring removal and/or restoration of a
protein, and next-generation engineered cell therapies for cancers
and auto-immune diseases.
- Intellia plans to nominate at least
one additional development candidate in 2021 and expects to present
preclinical data at upcoming scientific conferences highlighting
research advancements and platform innovations.
- Intellia demonstrated the
modularity of its targeted insertion approach for a second target,
in non-human primates, showing insertion of the SERPINA1 gene
produced normal levels of human alpha-1 antitrypsin (AAT) after a
single administration. In December, these results were presented at
the Alpha-1 Foundation’s 20th Gordon L. Snider Critical Issues
Workshop: The Promise of Gene-Based Interventions of Alpha-1
Antitrypsin Deficiency. Intellia is advancing multiple genome
editing strategies that may treat both lung and liver
manifestations of AAT deficiency (AATD), which occur due to
mutations in the SERPINA1 gene.
- Intellia is advancing preclinical
validation of in vivo hematopoietic stem cell (HSC) genome editing
using the Company’s proprietary non-viral delivery systems and
CRISPR/Cas9 technology to potentially cure sickle cell disease.
This research is supported by a grant from the Bill & Melinda
Gates Foundation.
- Financing: In December, Intellia closed an
underwritten public offering of 5,513,699 shares of common stock,
including the exercise in full of the underwriters’ option to
purchase additional shares, at the public offering price of $36.50
per share. Intellia received aggregate gross proceeds of
approximately $201 million, before underwriting discounts and
commissions and offering expenses.
Upcoming Events
The Company will participate in the following events during the
first quarter of 2021:
- AAAAI Annual Meeting, February
26–March 1, Virtual
- Keystone Symposium: Precision
Engineering of the Genome, Epigenome and Transcriptome, March 8-10,
Virtual
- Barclays Capital Global Healthcare
Conference, March 8, Virtual
- Oppenheimer's 31st Annual
Healthcare Conference, March 16, Virtual
- Cold Spring Harbor Laboratories meeting on Nucleic Acid
Therapies, March 24 - 26, Virtual
Upcoming Milestones
The Company has set forth the following for pipeline
progression:
- ATTR: Report initial clinical data
from Phase 1 study of NTLA-2001 in 2021
- AML: Submit an IND or
IND-equivalent for NTLA-5001 in mid-2021
- HAE: Submit an IND or
IND-equivalent for NTLA-2002 in 2H 2021
- Nominate at least one new development candidate in 2021
Fourth Quarter and Full-Year 2020 Financial
Results
- Cash
Position: Cash, cash equivalents and
marketable securities were $597.4 million as of December 31, 2020,
compared to $284.5 million as of December 31, 2019. The increase
was driven by net proceeds of $296.6 million from our follow-on
public offerings, $100.0 million upfront payment from the Regeneron
collaboration expansion, which included a $30.0 million equity
investment, $49.5 million of net equity proceeds raised from the
Company’s “At the Market” (ATM) agreement, $18.2 million from the
Regeneron and Novartis Institutes for BioMedical Research, Inc.
(Novartis) collaborations and $13.2 million in proceeds from
employee-based stock plans. These increases were offset in part by
cash used to fund operations of approximately $164.6 million.
- Collaboration
Revenue: Collaboration revenue decreased
by $4.3 million to $6.6 million during the fourth quarter of 2020,
compared to $10.9 million during the fourth quarter of 2019. The
decrease was primarily driven by a decrease in Novartis revenue as
the research portion of the collaboration ended in December
2019.
- R&D
Expenses: Research and development
expenses increased by $6.5 million to $38.2 million during the
fourth quarter of 2020, compared to $31.7 million during the fourth
quarter of 2019. This increase was primarily driven by the
advancement of our lead programs, research personnel growth to
support these programs and expansion of the development
organization.
- G&A
Expenses: General and administrative
expenses increased by $1.8 million to $10.8 million during the
fourth quarter of 2020, compared to $9.0 million during the fourth
quarter of 2019. This increase was primarily related to employee
related expenses, including stock-based compensation of $1.3
million.
- Net
Loss: The Company’s net loss was $42.2
million for the fourth quarter of 2020, compared to $28.3 million
during the fourth quarter of 2019.
Financial Guidance
Intellia expects that its cash, cash equivalents and marketable
securities as of December 31, 2020 will enable the Company to fund
its robust R&D plans, anticipated operating expenses and
capital expenditure requirements at least through the next 24
months. This expectation excludes any strategic use of capital not
currently in the Company’s base-case planning assumptions.
Conference Call to Discuss Fourth Quarter and Full-Year
2020 Earnings
The Company will discuss these results on a conference call
today, February 25, 2021, at 8 a.m. ET.
To join the call:
- U.S. callers should dial 1-833-316-0545 and international
callers should dial 1-412-317-5726, approximately five minutes
before the call.
- All participants should ask to be connected to the Intellia
Therapeutics conference 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 February 25,
2021 at 12 p.m. ET.
About Intellia TherapeuticsIntellia
Therapeutics is a leading clinical-stage genome editing
company, focused on the development of proprietary, potentially
curative therapeutics using the CRISPR/Cas9 system. Intellia
believes the CRISPR/Cas9 technology has the potential to transform
medicine by both producing therapeutics that permanently edit
and/or correct disease-associated genes in the human body with a
single treatment course, and creating enhanced engineered cells
that can treat oncological and immunological diseases. Intellia’s
combination of deep scientific, technical and clinical development
experience, along with its leading intellectual property portfolio,
puts it in a unique position to unlock broad therapeutic
applications of the CRISPR/Cas9 technology and create new classes
of therapeutic products. Learn more about Intellia and
CRISPR/Cas9 at intelliatx.com. Follow us on
Twitter @intelliatweets.
Forward-Looking StatementsThis press release
contains “forward-looking statements” of Intellia Therapeutics,
Inc. (“Intellia”, “we” or “our”) 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
our: being able to enroll and dose the necessary subjects in our
clinical studies for NTLA-2001 for the treatment of transthyretin
amyloidosis (“ATTR”), provide timing on the first expected data
readout, and successfully submit additional regulatory applications
in other countries; ability to evaluate NTLA-2001 in a broader ATTR
population; the ability to demonstrate NTLA-5001 as a potential
best-in-class engineered T cell therapy designed to treat all
genetic subtypes of acute myeloid leukemia (“AML”); plans to submit
an investigational new drug (“IND”) application or equivalent
regulatory application for NTLA-5001 in mid-2021; expectations of
evaluating the safety and activity of NTLA-5001 in patients with
persistent or recurrent AML who have previously received first-line
therapies; plans to evaluate the potential use of NTLA-5001 to
treat WT1-positive solid tumors in preclinical studies; plans to
submit an IND or equivalent regulatory application for NTLA-2002 in
the second half of 2021; expectations of evaluating safety,
tolerability and measures of activities of NTLA-2002 in patients
with hereditary angioedema (“HAE”); plans to nominate at least one
additional development candidate in 2021; plans to advance and
complete preclinical studies for our programs; development of our
modular platform to advance our complex genome editing
capabilities; further development of our proprietary genome editing
tools for research and therapeutic development sequential editing;
presentation of additional data at upcoming scientific conferences,
and other preclinical data in 2021; advancement and expansion of
our CRISPR/Cas9 technology to develop human therapeutic products,
as well as our ability to maintain and expand our related
intellectual property portfolio; ability to demonstrate our
platform’s modularity and replicate or apply results achieved in
preclinical studies, including those in our 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 Wilms’ Tumor 1 (“WT1”) in AML in
particular, using CRISPR/Cas9 technology; ability to optimize the
impact of our collaborations on our development programs, including
but not limited to our collaboration with Regeneron
Pharmaceuticals, Inc.; statements regarding the timing of
regulatory filings and clinical trial execution, including dosing
of patients, regarding our development programs; potential
commercial opportunities, including value and market, for our
product candidates; our expectations regarding our use of capital
and other financial results during 2021; and our ability to fund
operations for at least the next 24 months.
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 our ability to protect and
maintain our intellectual property position; risks related to our
relationship with third parties, including our licensors and
licensees; risks related to the ability of our licensors to protect
and maintain their intellectual property position; uncertainties
related to regulatory agencies’ evaluation of regulatory filings
and other information related to our product candidates;
uncertainties related to the authorization, initiation and conduct
of studies and other development requirements for our product
candidates; the risk that any one or more of our product
candidates, including those that are co-developed, 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 our collaborations with Regeneron or our 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 (“SEC”). 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 December 31, |
|
Twelve Months Ended December 31, |
|
|
2020 |
|
|
|
2019 |
|
|
|
2020 |
|
|
|
2019 |
|
Collaboration revenue |
|
$ |
6,595 |
|
|
$ |
10,936 |
|
|
$ |
57,994 |
|
|
$ |
43,103 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
Research and development |
|
|
38,231 |
|
|
|
31,731 |
|
|
|
150,408 |
|
|
|
108,413 |
|
General and administrative |
|
|
10,763 |
|
|
|
8,976 |
|
|
|
44,169 |
|
|
|
41,058 |
|
Total operating expenses |
|
|
48,994 |
|
|
|
40,707 |
|
|
|
194,577 |
|
|
|
149,471 |
|
Operating loss |
|
|
(42,399 |
) |
|
|
(29,771 |
) |
|
|
(136,583 |
) |
|
|
(106,368 |
) |
Interest income |
|
|
207 |
|
|
|
1,495 |
|
|
|
2,352 |
|
|
|
6,835 |
|
Net loss |
|
$ |
(42,192 |
) |
|
$ |
(28,276 |
) |
|
$ |
(134,231 |
) |
|
$ |
(99,533 |
) |
Net loss per share, basic and diluted |
|
$ |
(0.69 |
) |
|
$ |
(0.57 |
) |
|
$ |
(2.40 |
) |
|
$ |
(2.11 |
) |
Weighted average shares outstanding, basic and diluted |
|
|
61,306 |
|
|
|
49,350 |
|
|
|
55,987 |
|
|
|
47,247 |
|
|
|
|
|
|
|
|
|
|
|
INTELLIA
THERAPEUTICS, INC. |
CONSOLIDATED
BALANCE SHEET DATA (UNAUDITED) |
(Amounts in
thousands) |
|
|
|
December 31, 2020 |
|
December 31, 2019 |
Cash, cash equivalents and marketable securities |
|
$ |
597,371 |
|
$ |
284,472 |
Total
assets |
|
|
676,322 |
|
|
334,280 |
Total
liabilities |
|
|
149,250 |
|
|
64,399 |
Total
stockholders' equity |
|
|
527,072 |
|
|
269,881 |
|
|
|
|
|
Intellia Contacts:
Investors:Glenn GoddardChief Financial
Officer+1-857-706-1056glenn.goddard@intelliatx.com
Media:Julie FergusonInterim Head of External
Affairs &
Communications+1-312-385-0098julie.ferguson@intelliatx.com
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