Intellia Therapeutics Announces Fourth Quarter and Full-Year 2021 Financial Results and Highlights Recent Company Progress
24 Février 2022 - 1:30PM
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing
company focused on developing potentially curative therapies
leveraging CRISPR-based technologies, today reported operational
highlights and financial results for the fourth quarter and year
ended December 31, 2021.
“We began 2022 with strong momentum, executing against our
strategic priorities for the advancement of our CRISPR-based
pipeline and platform. We continued to make steady progress across
our multiple clinical programs. In addition, we nominated two new
development candidates – NTLA-2003 and NTLA-6001. Notably, we are
looking forward to sharing additional data from the landmark study
of NTLA-2001 next week,” said Intellia President and Chief
Executive Officer John Leonard, M.D. “In parallel, we continue to
propel our own scientific innovation, as well as leverage external
capabilities from across the industry to generate the next wave of
clinical candidates. As part of this strategy, we completed several
important transactions to further bolster our industry-leading
genome editing toolbox and pipeline. Intellia is well-positioned to
extend its leadership position as we aim to harness the full
potential of genomic medicines.”
Fourth Quarter 2021 and Recent Operational
Highlights
In Vivo Program Updates
- NTLA-2001 for ATTR
amyloidosis: NTLA-2001 is the first investigational
CRISPR-based therapy to be systemically delivered to edit genes
inside the human body and has the potential to be the first
single-dose treatment for transthyretin (ATTR) amyloidosis.
Delivered with the Company’s in vivo lipid nanoparticle (LNP)
technology, NTLA-2001 offers the possibility of halting and
reversing the disease by driving a deep, potentially lifelong
reduction in transthyretin (TTR) protein after a single dose.
NTLA-2001 is part of a co-development/co-promotion agreement
between Intellia, the lead party for this program, and Regeneron
Pharmaceuticals, Inc. (Regeneron).
- Intellia will be hosting a virtual
investor event on February 28, 2022, at 4:30 p.m. ET to present
additional interim clinical data from the ongoing Phase 1 study of
NTLA-2001 in patients with hereditary ATTR amyloidosis with
polyneuropathy (ATTRv-PN). Data to be presented will come from all
four ATTRv-PN dose cohorts in Part 1, the single-ascending dose
portion, and include safety and serum TTR reduction for Cohorts 3
and 4, as well as an early look at durability across all cohorts.
The Company remains on track to initiate Part 2, a single-cohort
expansion, in the polyneuropathy arm in the first quarter of
2022.
- Intellia continues to dose patients
in the cardiomyopathy arm of the Phase 1 study with NTLA-2001. In
December 2021, Intellia initiated dosing in new dose-escalation
cohorts in patients with ATTR amyloidosis with cardiomyopathy
(ATTR-CM) as part of the expanded Phase 1 study. Enrollment across
both ATTRv-PN and ATTR-CM patient populations is expected to
complete in 2022.
- NTLA-2002 for HAE:
NTLA-2002 leverages Intellia’s proprietary in vivo LNP delivery
technology to knock out the KLKB1 gene in the liver with the
potential to permanently reduce total plasma kallikrein protein and
activity, a key mediator of hereditary angioedema (HAE). This
investigational approach aims to prevent attacks for people living
with HAE by providing continuous suppression of plasma kallikrein
activity following a single dose and to eliminate the significant
treatment burden associated with currently available HAE therapies.
- In December 2021, Intellia
announced the first patient was dosed with NTLA-2002. The
first-in-human study is expected to evaluate the safety,
tolerability and activity of NTLA-2002 in adults with Type I or
Type II HAE.
- The Company anticipates presenting
interim data from the Phase 1/2 study in the second half of 2022.
The data are expected to characterize the emerging safety and
activity profile of NTLA-2002, and to potentially demonstrate
preliminary proof of concept.
- NTLA-3001 for
AATD-associated lung disease: NTLA-3001 is a wholly owned,
first-in-class, CRISPR-mediated in vivo targeted gene insertion
development candidate for the treatment of alpha-1 antitrypsin
deficiency (AATD)-associated lung disease. It is designed with the
aim to precisely insert a healthy copy of the SERPINA1 gene, which
encodes the alpha-1 antitrypsin (A1AT) protein, with the potential
to restore permanent expression of functional A1AT protein to
therapeutic levels after a single dose. This approach seeks to
eliminate the need for sub-optimal weekly IV infusions of A1AT
augmentation therapy or lung transplant in severe cases.
- Intellia continues to conduct
Investigational New Drug (IND)-enabling activities for NTLA-3001,
with plans to file an IND or IND-equivalent in 2023.
- NTLA-2003 for
AATD-associated liver disease: NTLA-2003 is a wholly owned
in vivo knockout development candidate for the treatment of
AATD-associated liver disease. It is designed to inactivate the
SERPINA1 gene responsible for the production of abnormal A1AT
protein in the liver. This approach aims to halt the progression of
liver disease and eliminate the need for liver transplant in severe
cases.
- Today, Intellia announced the
nomination of a new development candidate, NTLA-2003, for treatment
of AATD-associated liver disease. The Company is advancing towards
IND-enabling activities for this program.
Ex Vivo Program Updates
- NTLA-5001 for AML:
NTLA-5001 is an autologous T cell receptor (TCR)-T cell therapy
engineered to target the Wilms’ Tumor 1 (WT1) antigen for the
treatment of all genetic subtypes of acute myeloid leukemia (AML).
- In the fourth quarter of 2021,
Intellia initiated screening of patients in the Phase 1/2a study of
NTLA-5001 for patients with AML. The Company has begun enrolling
patients and expects to dose its first patient in the coming weeks.
Later this year, the Company plans to provide guidance around
timing of the first expected data readout, with the goal of
demonstrating clinical proof of concept for its TCR-based
platform.
- NTLA-6001 for CD30+
Lymphomas: NTLA-6001 is Intellia’s wholly owned allogeneic
CAR-T development candidate targeting CD30 for the treatment of
CD30-expressing hematologic cancers, including relapsed or
refractory classical Hodgkin’s Lymphoma (cHL).
- Today, Intellia announced the
nomination of its first ex vivo allogeneic development candidate,
NTLA-6001, for CD30-expressing hematologic cancers, including cHL.
NTLA-6001 is developed using Intellia’s proprietary allogeneic cell
engineering platform, which leverages a novel combination of
sequential gene edits. Preclinical data presented on its
differentiated allogeneic engineering platform showed allogeneic T
cells were shielded from immune rejection, both host T and natural
killer (NK) cell attack.
- Intellia is advancing towards
IND-enabling activities and plans to present preclinical data
leading to the development of NTLA-6001 at an upcoming scientific
conference in 2022.
Research and Corporate Updates
- Modular Platform and
Pipeline Expansion: Intellia is expanding its
industry-leading genome editing platform and scientific leadership
through editing, delivery and cell engineering innovations that may
enable broader in vivo and ex vivo applications.
- Following the nomination of
NTLA-2003, Intellia plans to advance at least one new in vivo
development candidate by the end of 2022.
- The Company plans to highlight
additional advances to its proprietary technology capabilities,
including both genome editing and delivery tools, at upcoming
scientific conferences in 2022.
- Collaboration
Updates
- In February, the Company executed a
licensing and collaboration agreement with ONK Therapeutics Ltd.
(ONK) for the development of allogeneic CRISPR-edited NK cell
therapies for the treatment of cancer. The agreement grants ONK a
non-exclusive license to Intellia’s proprietary ex vivo
CRISPR/Cas9-based genome editing platform and its LNP-based
delivery technologies and exclusive rights to certain guide RNAs
for development of up to five NK cell therapies. ONK will be
responsible for preclinical and clinical development for the
engineered NK cell therapies covered under the agreement. In
addition, the agreement grants Intellia options to co-develop and
co-commercialize up to two products worldwide with rights to lead
commercialization in the U.S.
- In December 2021, the Company
executed a licensing and collaboration agreement with Kyverna
Therapeutics (Kyverna) for the development of KYV-201, an
allogeneic CD19 CAR-T cell investigational candidate for the
treatment of select autoimmune diseases. This is a novel approach
aimed at targeting CD19 for inflammatory diseases as compared to
traditional oncology indications. Kyverna will lead and fund
preclinical and clinical development for KYV-201 and, as part of
the agreement, Intellia granted Kyverna rights to use its
proprietary ex vivo CRISPR/Cas9-based allogeneic platform in
exchange for an equity stake in Kyverna. Intellia will be eligible
to receive certain development and commercial milestone payments,
as well as low- to mid-single-digit royalties on potential future
sales and may choose to exercise an option to lead U.S.
commercialization for KYV-201 under a co-development and
co-commercialization agreement.
- In October 2021, the Company
executed a strategic collaboration with SparingVision to develop
novel genomic medicines utilizing Intellia’s proprietary
CRISPR/Cas9 technology for the treatment of ocular diseases. In
addition, Intellia received an equity stake in SparingVision. As
part of the collaboration, Intellia will receive an option for
exclusive U.S. commercialization rights for product candidates
arising from two of three collaboration targets, eligibility for
development and commercial milestone payments, as well as royalties
on potential future sales of products arising from the
collaboration. The companies will additionally research and develop
novel self-inactivating AAV vectors and LNP-based approaches to
address delivery of CRISPR/Cas9 genome reagents to the retina.
- Corporate Updates
- In February, Intellia completed the
acquisition of Rewrite Therapeutics, Inc. (Rewrite), a private
biotechnology company focused on advancing novel DNA writing
technologies. Rewrite’s DNA writing technology may enable a range
of precise editing strategies. These include targeted corrections,
insertions, deletions and the full range of single-nucleotide
changes, which could provide new ways to edit disease-causing genes
and broaden the therapeutic potential for genomic medicines.
- In February, the Company announced
a lease agreement to develop a 140,000-square-foot manufacturing
facility in Waltham, Massachusetts, to support the manufacturing of
key components for its CRISPR-based investigational therapies. The
new manufacturing facility will be Good Manufacturing Practice
(GMP) compliant and support both the preclinical through commercial
supply for key components of Intellia’s CRISPR-based therapies.
Additionally, this facility, in combination with existing
capabilities and partnerships, will provide capacity and
capabilities in support of Intellia’s expanding pipeline and
commercial readiness.
Upcoming Events
The Company will participate in the following events during the
first quarter of 2022:
- AAAAI Annual Meeting, February
25-28, Phoenix
- Cowen Healthcare Conference, March
7, Virtual
- Barclays Capital Global Healthcare
Conference, March 15, Miami
- Guggenheim Healthcare Talks Genomic
Medicines and Rare Disease Day, March 31, Virtual
Upcoming Milestones
The Company has set forth the following for pipeline
progression:
- NTLA-2001 for ATTR amyloidosis:
- Report additional interim data from
Phase 1 study on February 28
- Initiate Part 2, a single-dose
expansion cohort, of the Phase 1 study of NTLA-2001 in Q1 2022
- Complete enrollment of Phase 1
study for both ATTRv-PN and ATTR-CM subjects in 2022
- NTLA-2002 for HAE: Present interim
data from Phase 1/2 study in 2H 2022
- NTLA-3001 for AATD: Plan to file an
IND or IND-equivalent in 2023
- NTLA-5001 for AML: Continue to
enroll patients in Phase 1/2a study in 2022
- NTLA-6001 for CD30+ Lymphomas: Plan
to present preclinical data at an upcoming scientific conference in
2022
- Pipeline Expansion:
- Advance at least one new in vivo
development candidate by the end of 2022
- Advance additional novel platform
capabilities in 2022
Fourth Quarter and Full-Year 2021 Financial
Results
- Cash
Position: Cash, cash equivalents and
marketable securities were $1,086.0 million as of December 31,
2021, compared to $597.4 million as of December 31, 2020. The
increase was driven by net proceeds of $648.3 million from a
follow-on offering in the third quarter of 2021, $45.3 million of
net proceeds from the Company’s “At the Market” (ATM) agreement,
$43.1 million in proceeds from employee-based stock plans, and $6.3
million of funding for cost-sharing agreements received from
Regeneron. These increases were offset in part by cash used to fund
operations of approximately $254.7 million.
- Collaboration
Revenue: Collaboration revenue increased
by $6.3 million to $12.9 million during the fourth quarter of 2021,
compared to $6.6 million during the fourth quarter of 2020. This
increase was primarily driven by $5.8 million in revenue recorded
in 2021 from our joint venture with AvenCell.
- R&D
Expenses: Research and development
expenses increased by $32.9 million to $71.2 million during the
fourth quarter of 2021, compared to $38.2 million during the fourth
quarter of 2020. 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 $11.3 million to $22.1 million during the
fourth quarter of 2021, compared to $10.8 million during the fourth
quarter of 2020. This increase was primarily related to employee
related expenses, including stock-based compensation of $3.8
million.
- Net
Loss: The Company’s net loss was $81.2
million for the fourth quarter of 2021, compared to $42.2 million
during the fourth quarter of 2020.
Conference Call to Discuss Fourth Quarter and Full-Year
2021 Results
The Company will discuss these results on a conference call
today, Thursday, February 24, at 8:00 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.
- Please visit this link for a simultaneous live webcast of 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 intelliatx.com, beginning on February 24, at
12:00 p.m. ET.
About Intellia TherapeuticsIntellia
Therapeutics, a leading clinical-stage genome editing company, is
developing novel, potentially curative therapeutics leveraging
CRISPR-based technologies. To fully realize the transformative
potential of CRISPR-based technologies, Intellia is pursuing two
primary approaches. The company’s in vivo programs use
intravenously administered CRISPR as the therapy, in which
proprietary delivery technology enables highly precise editing of
disease-causing genes directly within specific target tissues.
Intellia’s ex vivo programs use CRISPR to create the therapy by
using engineered human cells to treat cancer and autoimmune
diseases. Intellia’s deep scientific, technical and clinical
development experience, along with its robust intellectual property
portfolio, have enabled the company to take a leadership role in
harnessing the full potential of genome editing to create new
classes of genetic medicine. Learn more at intelliatx.com. Follow
us on Twitter @intelliatx.
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 ability to extend its leadership successfully and
harness the full potential of genomic medicines to bolster its
genome editing capabilities and pipeline; its ability to deploy its
financial resources, including cash and cash equivalents and
revenue from operations, successfully; the safety, efficacy,
success and advancement of its clinical programs for NTLA-2001 for
the treatment of transthyretin amyloidosis, NTLA-2002 for the
treatment of hereditary angioedema, and NTLA-5001 for the treatment
of acute myeloid leukemia pursuant to its clinical trial
applications (“CTA”) and IND submissions, including the expected
timing of data releases, regulatory filings, and the initiation,
enrollment, and completion of clinical trials; the advancement of
development candidates, including NTLA-3001 for the treatment of
alpha-1 antitrypsin deficiency (AATD)-associated lung disease,
NTLA-2003 for AATD-associated liver disease, and NTLA-6001 for
CD30+ lymphomas; the ability to generate data to initiate clinical
trials and the timing of CTA and IND submissions, including for
NTLA-3001 for the AATD-associated lung disease, NTLA-2003 for
AATD-associated liver disease, and NTLA-6001 for CD30+ lymphomas;
the expansion of its CRISPR/Cas9 technology and related
technologies to advance additional development candidates and
timing expectations of advancing such development candidates; its
ability to maintain and expand its related intellectual property
portfolio; expectations of the potential impact of the coronavirus
disease 2019 pandemic, including the impact of the Delta and
Omicron variants on strategy, future operations and timing of its
clinical trials; its expectations of Rewrite’s DNA writing
technology in enabling a range of precise editing strategies and
the resulting therapeutic potential of such technology; the ability
to optimize the impact of its collaborations on its development
programs, including, but not limited to, its collaborations with
Regeneron, including its co-development program with Regeneron for
ATTR amyloidosis, with Kyverna for the development of KYV-201, with
ONK for the development of engineered NK cell therapies, and with
SparingVision for the development of ocular therapies; and the
ability to complete and utilize its planned manufacturing facility,
including to manufacture GMP-compliant clinical and commercial
supply.
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 authorization, initiation and conduct of studies and
other development requirements for its product candidates,
including uncertainties related to regulatory approvals to conduct
clinical trials; risks related to the development and/or
commercialization of any of Intellia’s or its collaborators’
product candidates, including that they may not be successfully
developed and commercialized; risks related to the results of
preclinical studies or clinical studies, including that they may
not be predictive of future results in connection with future
studies; risks related to clinical study results, including that
they may not be positive; risks related to the Rewrite acquisition,
including that it may not result in the development of a writing
technology or otherwise result in enabling precise editing
strategies; risks related to Intellia’s reliance on collaborations,
including that its collaborations with Regeneron, ONK or Kyverna or
its other collaborations will not continue or will not be
successful; and risks related to completing and maintaining a
GMP-compliant manufacturing facility. 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 report on Form 10-Q, 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, |
|
|
|
|
|
|
2021 |
|
|
|
2020 |
|
|
|
2021 |
|
|
|
2020 |
|
|
|
Collaboration revenue |
$ |
12,854 |
|
|
$ |
6,595 |
|
|
$ |
33,053 |
|
|
$ |
57,994 |
|
|
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
Research and development |
|
71,161 |
|
|
|
38,231 |
|
|
|
229,807 |
|
|
|
150,408 |
|
|
|
|
General and administrative |
|
22,108 |
|
|
|
10,763 |
|
|
|
71,096 |
|
|
|
44,169 |
|
|
|
|
|
Total
operating expenses |
|
93,269 |
|
|
|
48,994 |
|
|
|
300,903 |
|
|
|
194,577 |
|
|
|
Operating loss |
|
(80,415 |
) |
|
|
(42,399 |
) |
|
|
(267,850 |
) |
|
|
(136,583 |
) |
|
|
Other (expense) income, net: |
|
|
|
|
|
|
|
|
|
|
Loss from equity method investment |
|
(1,325 |
) |
|
|
- |
|
|
|
(1,325 |
) |
|
|
- |
|
|
|
|
Interest income |
|
503 |
|
|
|
207 |
|
|
|
1,283 |
|
|
|
2,352 |
|
|
|
|
|
Total other
(expense) income, net |
|
(822 |
) |
|
|
207 |
|
|
|
(42 |
) |
|
|
2,352 |
|
|
|
Net loss |
|
$ |
(81,237 |
) |
|
$ |
(42,192 |
) |
|
$ |
(267,892 |
) |
|
$ |
(134,231 |
) |
|
|
Net loss per share, basic and diluted |
$ |
(1.09 |
) |
|
$ |
(0.69 |
) |
|
$ |
(3.78 |
) |
|
$ |
(2.40 |
) |
|
|
Weighted average shares outstanding, basic and diluted |
|
74,427 |
|
|
|
61,306 |
|
|
|
70,894 |
|
|
|
55,987 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
INTELLIA
THERAPEUTICS, INC. |
|
CONSOLIDATED
BALANCE SHEET DATA (UNAUDITED) |
|
(Amounts in
thousands) |
|
|
|
|
|
|
|
December 31, 2021 |
|
December 31, 2020 |
|
Cash, cash equivalents and marketable securities |
$ |
1,086,049 |
|
$ |
597,371 |
|
Total
assets |
|
1,294,464 |
|
|
676,322 |
|
Total
liabilities |
|
254,220 |
|
|
149,250 |
|
Total
stockholders' equity |
|
1,040,244 |
|
|
527,072 |
|
Intellia Contacts:
Investors:Ian KarpSenior Vice President,
Investor Relations and Corporate
Communications+1-857-449-4175ian.karp@intelliatx.comLina
LiDirectorInvestor
Relations+1-857-706-1612lina.li@intelliatx.com
Media:Matt CrensonTen Bridge
Communications+1-917-640-7930media@intelliatx.com
mcrenson@tenbridgecommunications.com
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