Intellia Therapeutics Announces Fourth Quarter and Full-Year 2022 Financial Results and Highlights Recent Company Progress
February 23 2023 - 7:30AM
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage
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, 2022.
“Intellia has hit the ground running with multiple milestones
already achieved in early 2023. In addition to recently initiating
the global Phase 2 study of NTLA-2002 outside of the U.S., we have
also submitted an IND application to enable inclusion of patients
in the U.S.,” said Intellia President and Chief Executive Officer
John Leonard, M.D. “As we look ahead, we are poised to submit an
IND application for NTLA-2001 and initiate a global pivotal trial
for the cardiomyopathy manifestation of ATTR amyloidosis.
Additionally, we look forward to presenting new and important
clinical data from the ongoing Phase 1 study, which builds on the
growing body of data that support NTLA-2001’s potential to
transform the ATTR amyloidosis treatment landscape for patients
with cardiomyopathy or polyneuropathy. In parallel, we are
advancing NTLA-3001, our first wholly owned in vivo gene insertion
candidate, which may normalize levels of the missing protein in
patients with alpha-1 antitrypsin deficiency. Together, we believe
these efforts move us closer to setting a new standard of care for
people living with serious diseases and expanding Intellia’s impact
as the leading genome editing company.”
Fourth Quarter 2022 and Recent Operational
Highlights
In Vivo Program Updates
Transthyretin (ATTR) Amyloidosis
- NTLA-2001:
NTLA-2001 is an in vivo, systemically delivered investigational
CRISPR-based therapy designed to inactivate the TTR gene in liver
cells and thereby prevent the production of transthyretin (TTR)
protein for the treatment of ATTR amyloidosis. NTLA-2001 offers the
possibility of halting and reversing the disease by driving a deep,
consistent and potentially lifelong reduction in TTR protein after
a single dose. NTLA-2001 is subject to a
co-development/co-promotion agreement between Intellia, the lead
party for this program, and Regeneron Pharmaceuticals, Inc.
- ATTR Amyloidosis with
Cardiomyopathy (ATTR-CM):
- Intellia announced in November 2022
positive interim results from the cardiomyopathy arm of the ongoing
Phase 1 clinical trial of NTLA-2001 at the American Heart
Association (AHA) Scientific Sessions 2022 held in Chicago,
Illinois. The interim data from the dose-escalation portion of the
study included 12 adult patients with ATTR-CM with New York
Heart Association (NYHA) Class I – III heart failure. Single
doses of 0.7 mg/kg and 1.0 mg/kg of NTLA-2001 led to greater than
90% mean serum TTR reductions. These deep reductions in serum TTR
were sustained through the observation period, with patient
follow-up ranging from four to six months. At both dose levels,
NTLA-2001 was generally well-tolerated. One patient in the 0.7
mg/kg dose NYHA Class III cohort experienced a Grade 3
infusion-related reaction, which resolved without clinical
sequalae. No clinically significant laboratory abnormalities were
observed at either dose level.
- In December 2022, the planned
enrollment of the dose-expansion portion of the ATTR-CM arm was
completed to support a U.S. Investigational New Drug (IND)
application submission for the pivotal study. The Company
anticipates submitting an IND application in mid-2023 and
initiating a global pivotal trial for ATTR-CM by year-end 2023,
subject to regulatory feedback.
- The Company plans to present
additional data from the ATTR-CM arm of the Phase 1 study in 2023,
including longer-term safety and durability data, as well as
emerging clinical endpoints.
- Hereditary ATTR Amyloidosis
with Polyneuropathy (ATTRv-PN):
- During the first quarter of 2023,
the planned enrollment of the dose-expansion portion of the
ATTRv-PN arm in the Phase 1 study was completed to inform a pivotal
study. The Company is preparing for a global pivotal study, which
will include discussions with regulatory authorities.
- The Company plans to present
additional clinical data from the ATTRv-PN arm of the Phase 1 study
in 2023.
Hereditary Angioedema (HAE)
- NTLA-2002:
NTLA-2002 is designed 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 HAE. This investigational
approach aims to prevent attacks for people living with HAE by
providing continuous reduction of plasma kallikrein activity,
following a single dose. It also aims to eliminate the significant
treatment burden associated with currently available HAE therapies.
NTLA-2002 is being evaluated in a Phase 1/2 study in adults with
Type I or Type II HAE.
- Intellia announced in November 2022
positive interim results from an ongoing Phase 1/2 clinical study
of NTLA-2002 at the American College of Allergy, Asthma &
Immunology (ACAAI) 2022 Annual Scientific Meeting held in
Louisville, Kentucky. The data presented were from 10 adult
patients with HAE in the Phase 1, dose-escalation portion
of the study. Single doses of 25 mg (n=3), 50 mg (n=4) and 75 mg
(n=3) of NTLA-2002 were administered via intravenous infusion,
which led to deep, dose-dependent reductions in plasma kallikrein.
All patients treated in the 25 mg and 75 mg cohorts, who completed
the pre-specified 16-week observation period, maintained an
attack-free status through the data cut-off date (patient follow-up
ranged from 2.3 to 10.6 months). Patients in the 50 mg cohort had
not completed the primary 16-week observation period. At all three
dose levels, NTLA-2002 was generally well-tolerated, and the
majority of adverse events were mild in severity. No clinically
significant laboratory abnormalities were observed.
- In January 2023, Intellia was
awarded the Innovation Passport for NTLA-2002 by the U.K. Medicines
and Healthcare products Regulatory Agency (MHRA). The Innovation
Passport is the point of entry into the U.K.’s Innovative Licensing
and Access Pathway (ILAP), which is designed to accelerate time to
market and facilitate patient access to innovative medicines.
- Intellia announced today the
initiation of patient screening in the Phase 2 portion of the Phase
1/2 of NTLA-2002 in New Zealand. The Company has selected 25 mg and
50 mg as the two, single dose levels for further evaluation in the
randomized, placebo-controlled study.
- Intellia announced today the
Company recently submitted an IND application for NTLA-2002 to the
U.S. Food and Drug Administration (FDA) to support the inclusion of
U.S. sites in the Phase 2 portion of the study.
- The Company plans to present
additional clinical data from the Phase 1 portion of the
first-in-human study in 2023, including safety, durability and
attack-rate data across all three cohorts.
Alpha-1 Antitrypsin Deficiency (AATD)
- NTLA-3001 for 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 AATD-associated lung disease. It is
designed 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 improve patient outcomes, including eliminating the need
for weekly intravenous infusions of A1AT augmentation therapy or
lung transplant in severe cases.
- Intellia is conducting IND-enabling
activities for NTLA-3001 and plans to submit an IND or
IND-equivalent filing in 2H 2023.
- NTLA-2003 for 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.
- Intellia is conducting IND-enabling
activities for NTLA-2003, with the expectation of completing these
activities by year-end 2023.
Ex Vivo Program Updates
Immuno-oncology and Autoimmune Diseases
- Intellia is advancing multiple
programs, wholly owned and in collaboration with partners,
utilizing its allogeneic platform for the treatment of
immuno-oncology and autoimmune diseases. The Company’s proprietary
allogeneic cell engineering platform is designed to avoid both T
cell- and NK cell-mediated rejection, a key unsolved challenge with
other investigational allogeneic approaches.
- NTLA-6001 for CD30+
Lymphomas: Intellia is identifying collaboration
opportunities to advance the development of a wholly owned,
allogeneic CAR-T development candidate targeting CD30.
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.
- In January 2023, Intellia achieved
a research milestone with its DNA writing technology. A $25.0
million milestone payment was made to shareholders of Rewrite
Therapeutics in February 2023.
- Sickle Cell Disease (SCD)
Program Updates:
- Intellia’s SCD Research
Efforts: Intellia is focused on developing an in vivo
editing approach for the treatment of SCD to avoid the need for
bone marrow transplantation. This is a wholly owned program
currently in preclinical development.
- Novartis’ SCD
Program: In February 2023, Novartis opted to discontinue
the development of its autologous, ex vivo, CRISPR-edited
hematopoietic stem cell (HSC) program targeting fetal hemoglobin
(HbF) for the treatment of SCD.
Upcoming Events
The Company will participate in the following events during the
first quarter of 2023:
- Cowen 43rd Annual Health Care
Conference, March 6, Boston
- Barclays Global Healthcare
Conference, March 14, Miami
Upcoming Milestones
The Company has set forth the following for pipeline
progression:
- NTLA-2001 for ATTR
amyloidosis:
- Submit an IND application in
mid-2023 to enable inclusion of U.S. sites in a pivotal study of
NTLA-2001 for patients with ATTR-CM.
- Present additional clinical data
from the ongoing Phase 1 study of NTLA-2001 in 2023.
- Initiate a global pivotal NTLA-2001
trial for ATTR-CM by year-end 2023, subject to regulatory
feedback.
- Prepare for a Phase 3 study of
NTLA-2001 for the treatment of ATTRv-PN, including discussions with
regulatory authorities.
- NTLA-2002 for HAE:
- Present additional clinical data
from the ongoing first-in-human study of NTLA-2002 in 2023.
- AATD Franchise:
- Submit an IND or IND-equivalent
application for NTLA-3001 for AATD-associated lung disease in 2H
2023.
- Complete IND-enabling activities for
NTLA-2003 for AATD-associated liver disease by year-end 2023.
Platform Innovation
- Advance novel gene editing
technologies, including DNA writing, and delivery to other tissues
outside of the liver.
Fourth Quarter and Full-Year 2022 Financial
Results
- Cash
Position: Cash, cash equivalents and
marketable securities were $1.3 billion as of December 31, 2022,
compared to $1.1 billion as of December 31, 2021. The increase was
driven by $337.9 million from a follow-on offering in the fourth
quarter of 2022, $227.9 million of net proceeds from the Company’s
“At the Market” (ATM) program and $17.2 million in proceeds from
employee-based stock plans. The increase was offset in part by cash
used to fund operations of approximately $372.8 million and the
acquisition of Rewrite for $45.0 million.
- Collaboration
Revenue: Collaboration revenue increased
by $0.7 million to $13.6 million during the fourth quarter of 2022,
compared to $12.9 million during the fourth quarter of 2021.
- R&D
Expenses: Research and development
expenses increased by approximately $28.9 million to $100.0 million
during the fourth quarter of 2022, compared to $71.2 million during
the fourth quarter of 2021. This increase was primarily driven by
the advancement of our lead programs and personnel growth to
support these programs.
- G&A
Expenses: General and administrative
expenses increased by $1.5 million to $23.6 million during the
fourth quarter of 2022, compared to $22.1 million during the fourth
quarter of 2021. This increase was primarily related to an increase
in stock-based compensation of $2.6 million, offset in part by a
decrease in legal expenses of $0.9 million.
- Net
Loss: The Company’s net loss was $113.4
million for the fourth quarter of 2022, compared to $81.2 million
during the fourth quarter of 2021.
Conference Call to Discuss Fourth Quarter and Full-Year
2022 Results
The Company will discuss these results on a conference call
today, Thursday, February 23, 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.
- 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 23, at
12 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
concerning: its ability to successfully extend its leadership
position and harness the full potential of genomic medicines to
bolster its genome editing capabilities and pipeline; the safety,
efficacy, success and advancement of its clinical programs for
NTLA-2001 for the treatment of ATTR amyloidosis and NTLA-2002 for
the treatment of hereditary angioedema (“HAE”) pursuant to its
clinical trial applications (“CTA”), including the expected timing
of data releases, regulatory filings, and the initiation,
enrollment, dosing and completion of clinical trials, such as the
completion of planned enrollment of the Phase 1 study for
NTLA-2001; its ability to select a dose to advance to a pivotal
study for NTLA-2001; the advancement of development candidates,
such as 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,
including the success of its investigational new drug
(“IND”)-enabling studies; its ability to generate data to initiate
clinical trials and the timing of CTA and IND submissions,
including the submission of an IND or IND-equivalent for NTLA-3001
in 2023; and an IND application for a global pivotal trial for
NTLA-2001 in mid-2023; its ability to advance multiple programs
utilizing an allogeneic platform for the treatment of
immuno-oncology and autoimmune diseases; the expansion of its
CRISPR/Cas9 technology and related and novel technologies to
advance additional development candidates and timing expectations
of advancing such development candidates and releasing data related
to such technologies and development candidates; its ability to
optimize the impact of its collaborations on its development
programs, including, but not limited to, its collaboration with
Regeneron Pharmaceuticals, Inc. and their co-development program
for ATTR amyloidosis; and growth as a Company and expectations
regarding its uses of capital, expenses, future accumulated deficit
and financial results.
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 contract
manufacturers, 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 preclinical and clinical studies and
other development requirements for its product candidates,
including uncertainties related to regulatory approvals to conduct
clinical trials; risks related to the ability to develop and
commercialize any one or more of Intellia’s product candidates
successfully; risks related to the results of preclinical studies
or clinical studies not being predictive of future results in
connection with future studies; the risk that clinical study
results will not be positive; risks related to the potential impact
of the coronavirus disease pandemic, including the impact of any
variants, on strategy, future operations and timing of its clinical
trials; and risks related to Intellia’s collaborations with
Regeneron Pharmaceuticals, Inc. or its other collaborations not
continuing or not being 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 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.
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INTELLIA
THERAPEUTICS, INC. |
|
CONSOLIDATED
STATEMENTS OF OPERATIONS (UNAUDITED) |
|
(Amounts in
thousands, except per share data) |
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Three Months Ended December 31, |
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Twelve Months Ended December 31, |
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2022 |
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2021 |
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2022 |
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2021 |
|
|
Collaboration revenue |
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$ |
13,573 |
|
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$ |
12,854 |
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$ |
52,121 |
|
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$ |
33,053 |
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Operating expenses: |
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|
|
|
|
|
|
|
|
Research and development |
|
100,034 |
|
|
|
71,161 |
|
|
|
419,979 |
|
|
|
229,807 |
|
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General and administrative |
|
23,626 |
|
|
|
22,108 |
|
|
|
90,306 |
|
|
|
71,096 |
|
|
Total operating expenses |
|
123,660 |
|
|
|
93,269 |
|
|
|
510,285 |
|
|
|
300,903 |
|
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Operating loss |
|
|
|
(110,087 |
) |
|
|
(80,415 |
) |
|
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(458,164 |
) |
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(267,850 |
) |
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Other (expense) income, net: |
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Interest income |
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5,354 |
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|
|
503 |
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|
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8,542 |
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|
|
1,283 |
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Loss from equity method investment |
|
(3,248 |
) |
|
|
(1,325 |
) |
|
|
(11,079 |
) |
|
|
(1,325 |
) |
|
Change in fair value of contingent consideration |
|
|
|
|
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(5,426 |
) |
|
|
- |
|
|
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(13,485 |
) |
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|
- |
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Total other (expense) income, net |
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(3,320 |
) |
|
|
(822 |
) |
|
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(16,022 |
) |
|
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(42 |
) |
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Net loss |
|
|
|
$ |
(113,407 |
) |
|
$ |
(81,237 |
) |
|
$ |
(474,186 |
) |
|
$ |
(267,892 |
) |
|
Net loss per share, basic and diluted |
$ |
(1.40 |
) |
|
$ |
(1.09 |
) |
|
$ |
(6.16 |
) |
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$ |
(3.78 |
) |
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Weighted average shares outstanding, basic and diluted |
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81,223 |
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74,427 |
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76,972 |
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70,894 |
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INTELLIA
THERAPEUTICS, INC. |
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CONSOLIDATED
BALANCE SHEET DATA (UNAUDITED) |
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(Amounts in
thousands) |
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December 31, 2022 |
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December 31, 2021 |
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Cash, cash equivalents and marketable securities |
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$ |
1,261,960 |
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$ |
1,086,049 |
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Total assets |
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|
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1,520,114 |
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1,294,464 |
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Total liabilities |
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|
|
|
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284,530 |
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|
254,220 |
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Total stockholders' equity |
|
|
|
1,235,584 |
|
|
1,040,244 |
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Intellia Contacts:
Investors:Ian KarpSenior Vice President,
Investor Relations and Corporate
Communications+1-857-449-4175ian.karp@intelliatx.comLina LiSenior
Director, Investor Relations and Corporate
Communications+1-857-706-1612lina.li@intelliatx.com
Media:Rebecca SpaldingTen Bridge
Communications+1-646-509-3831media@intelliatx.com
rebecca@tenbridgecommunications.com
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