Intellia Therapeutics Announces First Quarter 2023 Financial Results and Highlights Recent Company Progress
04 Mai 2023 - 1:30PM
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 first quarter
ended March 31, 2023.
“We’ve had a productive start to the year with the successful
clearance of our first IND application for an investigational in
vivo CRISPR-based therapy alongside broad pipeline and platform
progress,” said Intellia President and Chief Executive Officer John
Leonard, M.D. “Today, we are pleased to announce dosing has begun
in the Phase 2 study of NTLA-2002, and based on strong interest
from investigators and patients, we expect to complete enrollment
in the second half of this year. Our rapid progression of the
clinical development of NTLA-2002 supports our goal to bring forth
a potential functional cure for the treatment of hereditary
angioedema. Simultaneously, we are working toward submitting our
second in vivo IND application and initiating a global pivotal
trial for NTLA-2001. We look forward to sharing new interim data
from both the NTLA-2001 and NTLA-2002 first-in-human studies in the
months to come.”
First Quarter 2023 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 plans to submit a U.S.
Investigational New Drug (IND) application in mid-2023. Subject to
regulatory feedback, the Company anticipates initiating a global
pivotal trial for ATTR-CM by year-end 2023.
- The Company expects 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):
- As previously announced, 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 actively preparing for a
global pivotal study, which includes discussions with regulatory
authorities.
- Intellia recently began redosing
patients in the 0.1 mg/kg cohort (n=3), the initial cohort and
lowest dose tested in the dose-escalation portion of the Phase 1
study, with the 55 mg dose selected for the dose-expansion
cohort.
- 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 a wholly owned, in vivo, systemically delivered
investigational CRISPR-based therapy. 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 today that the
first patient has been dosed in the global Phase 2 portion of its
Phase 1/2 clinical trial of NTLA-2002. Based on encouraging study
interest from both investigators and patients, the Company expects
to complete enrollment (n=25) in 2H 2023.
- The Company announced in March that
the U.S. Food and Drug Administration (FDA) cleared the
NTLA-2002 Phase 2 IND application. Additionally, the FDA granted
Regenerative Medicine Advanced Therapy (RMAT) designation to
NTLA-2002 for the treatment of HAE.
- 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.
- The Company plans to present
additional clinical data from the Phase 1 portion of the
first-in-human study in 2023. Data expected to be presented include
updated safety, durability of pharmacodynamic effect and
attack-rate measures from all three patient 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
preclinical 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.
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.
- Board of Directors
Update: In April, Intellia announced the appointment of
Bill Chase to its board of directors. Mr. Chase will be a member of
the audit committee and will succeed Caroline Dorsa as chair of the
audit committee upon her retirement from the board on June 15,
2023.
- Corporate Responsibility
Report: In May, Intellia published its 2023 Corporate
Responsibility Report. The report highlights the Company’s
Environmental, Social and Governance (ESG) principles and practices
as part of its objective to build a sustainable company while
delivering on its commitments to patients, employees and
shareholders.
Upcoming Events
The Company will participate in the following events during the
second quarter of 2023:
- Bank of America Securities 2023
Health Care Conference, May 9, Las Vegas
- 2023 RBC Capital Markets Global
Healthcare Conference, May 16, New York City
- Barclays Gene Editing & Therapy
Summit, May 24, New York City
- Stifel Genetic Medicines Day, May
30, virtual
Upcoming Milestones
The Company has set forth the following expected milestones 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.
- Complete enrollment in the Phase 2
portion of the Phase 1/2 study in 2H 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.
First Quarter 2023 Financial Results
- Cash
Position: Cash, cash equivalents and
marketable securities were $1.2 billion as of March 31, 2023,
compared to $1.3 billion as of December 31, 2022. The decrease was
driven by cash used to fund operations of approximately $120.9
million. The decrease was offset in part by $12.0 million of
interest income, $3.4 million of reimbursement from its
collaborators, $1.5 million of net equity proceeds from the
Company’s “At the Market” (ATM) program and $0.8 million in
proceeds from employee-based stock plans.
- Collaboration
Revenue: Collaboration revenue increased
by $1.3 million to $12.6 million during the first quarter of 2023,
compared to $11.3 million during the first quarter of 2022.
- R&D
Expenses: Research and development
expenses decreased by approximately $36.0 million to $97.1 million
during the first quarter of 2023, compared to $133.1 million during
the first quarter of 2022. This decrease was primarily driven by
$56.0 million of expense related to the acquisition of Rewrite
Therapeutics, Inc. during the first quarter of 2022. The decrease
related to the acquisition of Rewrite Therapeutics, Inc. was offset
by an increase in expenses of $20.0 million primarily driven by the
advancement of its lead programs and personnel growth to support
these programs. Stock-based compensation expense included in
research and development expenses was $16.9 million for the first
quarter of 2023.
- G&A
Expenses: General and administrative
expenses increased by $5.0 million to $27.4 million during the
first quarter of 2023, compared to $22.4 million during the first
quarter of 2022. This increase was primarily related to an increase
in stock-based compensation of $2.1 million. Stock-based
compensation expense included in general and administrative
expenses was $10.3 million for the first quarter of 2023.
- Net
Loss: The Company’s net loss was $103.1
million for the first quarter of 2023, compared to $146.9 million
during the first quarter of 2022.
Conference Call to Discuss First Quarter 2023
Results
The Company will discuss these results on a conference call
today, Thursday, May 4, 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 May 4, 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, including to
enable broader in vivo and ex vivo applications; 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
presentation of additional data, including emerging clinical
endpoints, related to the NTLA-2001 and NTLA-2002 clinical trials;
the completion of planned enrollment of the Phase 2 study for
NTLA-2002 in the second half of 2023; the advancement of
development candidates, such as NTLA-3001 for the treatment of
alpha-1 antitrypsin deficiency (“AATD”)-associated lung disease and
NTLA-2003 for AATD-associated liver disease, including the success
of its investigational new drug (“IND”)-enabling studies and
completion of IND-enabling activities for NTLA-2003 by the end of
2023; its ability to generate data to initiate clinical trials and
the timing of CTA and IND submissions and initiation of related
clinical trials, including the submission of an IND or
IND-equivalent for NTLA-3001 in the second half of 2023, submission
of an IND application for NTLA-2001 in mid-2023 and initiating a
global pivotal trial for NTLA-2001 by the end of 2023; its ability
to leverage the designation of NTLA-2002 under the U.S. Food and
Drug Administration’s Regenerative Medicine Advanced Therapy and
the U.K. Medicines and Healthcare products Regulatory Agency's
Innovation Passport to accelerate time to market and facilitate
patient access; 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 novel technologies, including DNA writing and delivery
to other tissues outside of the liver, 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; 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.
INTELLIA
THERAPEUTICS, INC. |
CONSOLIDATED
STATEMENTS OF OPERATIONS (UNAUDITED) |
(Amounts in
thousands, except per share data) |
|
|
|
|
|
Three Months Ended March 31, |
|
|
2023 |
|
|
|
2022 |
|
Collaboration revenue |
$ |
12,606 |
|
|
$ |
11,252 |
|
Operating
expenses: |
|
|
|
Research and development |
|
97,116 |
|
|
|
133,095 |
|
General and administrative |
|
27,448 |
|
|
|
22,403 |
|
Total operating expenses |
|
124,564 |
|
|
|
155,498 |
|
Operating
loss |
|
(111,958 |
) |
|
|
(144,246 |
) |
Other income
(expense), net: |
|
|
|
Interest income |
|
11,980 |
|
|
|
540 |
|
Loss from equity method investment |
|
(3,048 |
) |
|
|
(2,745 |
) |
Change in fair value of contingent consideration |
|
(100 |
) |
|
|
(421 |
) |
Total other income (expense), net |
|
8,832 |
|
|
|
(2,626 |
) |
Net
loss |
$ |
(103,126 |
) |
|
$ |
(146,872 |
) |
Net loss per
share, basic and diluted |
$ |
(1.17 |
) |
|
$ |
(1.96 |
) |
Weighted
average shares outstanding, basic and diluted |
|
87,772 |
|
|
|
74,751 |
|
INTELLIA
THERAPEUTICS, INC. |
CONSOLIDATED
BALANCE SHEET DATA (UNAUDITED) |
(Amounts in
thousands) |
|
|
|
|
|
March 31, 2023 |
|
December 31, 2022 |
Cash, cash equivalents and marketable securities |
$ |
1,158,580 |
|
|
$ |
1,261,960 |
|
Total
assets |
|
1,417,889 |
|
|
|
1,520,114 |
|
Total
liabilities |
|
227,046 |
|
|
|
284,530 |
|
Total
stockholders' equity |
|
1,190,843 |
|
|
|
1,235,584 |
|
|
|
|
|
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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