Intellia Therapeutics Announces Third Quarter 2023 Financial Results and Highlights Recent Company Progress
09 Novembro 2023 - 9:30AM
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 third quarter
ended September 30, 2023.
“2023 has been a year of remarkable progress in which Intellia
received two IND clearances for investigational in vivo CRISPR
therapies. With the imminent start of the NTLA-2001 MAGNITUDE Phase
3 trial, Intellia has now become a late-stage drug development
company,” said Intellia President and Chief Executive Officer John
Leonard, M.D. “Looking ahead, we expect to achieve several notable
milestones in the coming weeks and months, including completing
enrollment of the NTLA-2002 Phase 2 study in people with hereditary
angioedema and submitting a regulatory filing to begin clinical
development for NTLA-3001, our in vivo gene insertion program for
people living with alpha-1 antitrypsin deficiency. We look forward
to advancing our pipeline and platform as we move closer to
realizing the potential of CRISPR-based medicines.”
Third 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.
- ATTR Amyloidosis with
Cardiomyopathy (ATTR-CM):
- The Company announced in October
that the U.S. Food and Drug Administration (FDA) cleared the
NTLA-2001 Phase 3 Investigational New Drug (IND) application for
the treatment of ATTR-CM. The MAGNITUDE pivotal Phase 3 trial is a
randomized, double-blind, placebo-controlled study to evaluate the
efficacy and safety of NTLA-2001 in approximately 765 patients with
ATTR-CM. The primary endpoint of the study is a composite endpoint
of cardiovascular (CV)-related mortality and CV-related events.
Patients will be randomized 2:1 NTLA-2001:placebo, with a single 55
mg infusion of NTLA-2001 administered. The Company expects to
initiate the study by year-end with patient dosing to commence
early 2024.
- Hereditary ATTR Amyloidosis
with Polyneuropathy (ATTRv-PN):
- The Company is actively preparing
for a global pivotal Phase 3 study of NTLA-2001 for the treatment
of ATTRv-PN, including discussions with regulatory
authorities.
- Updated Data from NTLA-2001
Phase 1 Study
- In November, Intellia announced new
positive interim results from the Phase 1 study of NTLA-2001.
Updated data from over 60 patients showed consistent, deep and
durable serum TTR reduction achieved with a single dose of
NTLA-2001, including 29 patients who reached 12 months or more of
follow-up as of the data cut-off date of May 11, 2023. Across all
patients who received a dose of 0.3 mg/kg or higher (n=62), the
median serum TTR reduction was 91% and the median absolute residual
serum TTR concentration was 17 µg/mL at day 28. Across all patients
and at all dose levels tested, NTLA-2001 was generally well
tolerated, and the majority of adverse events were mild in
severity. These interim data were presented at the 4th
International ATTR Amyloidosis Meeting, held in Madrid, Spain.
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.
- In October, Intellia announced that
the European Medicines Agency (EMA) granted Priority Medicine
(PRIME) designation to NTLA-2002 for the treatment of HAE. PRIME
designation is granted by the EMA to drug candidates that may offer
a major therapeutic advantage over existing treatments or that
benefit patients without treatment options.
- Following the identification of all
patients, Intellia is on track to complete enrollment in the Phase
2 portion of the Phase 1/2 study in Q4 2023.
- Intellia plans to initiate the
global pivotal Phase 3 study, including U.S. patients, as early as
the third quarter of 2024, subject to regulatory feedback.
Alpha-1 Antitrypsin Deficiency (AATD)
- 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 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 plans to submit a Clinical
Trial Application (CTA) in Q1 2024 to initiate a first-in-human,
Phase 1 study of NTLA-3001.
- 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.
- Based on a prioritization of
resources, the Company is making a strategic shift to halt further
IND-enabling activities for NTLA-2003 to advance an AATD
research-stage program leveraging the Company’s DNA writing
technology.
Ex Vivo Program Updates
- 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.
Platform and Pipeline Expansion
- As Intellia’s DNA writing
technology has met key internal research milestones, the Company
will now prioritize the advancement of a research-stage program for
the treatment of AATD.
- In October, Intellia and Regeneron
announced an expanded research collaboration to develop additional
in vivo CRISPR-based gene editing therapies focused on neurological
and muscular diseases. The collaboration will leverage Regeneron’s
proprietary antibody-targeted adeno-associated virus (AAV) vectors
and delivery systems and Intellia’s proprietary Nme2 CRISPR/Cas9
(Nme2Cas9) systems adapted for viral vector delivery and designed
to precisely modify a target gene.
- Additionally, Regeneron has
exercised its option to extend the existing technology
collaboration term with Intellia for two years. The technology
collaboration term now extends to April 2026, and Intellia will
receive a $30 million payment in the first half of 2024.
- In September, SparingVision
announced that it had selected a second target as part of its
strategic collaboration with Intellia to develop novel genomic
medicines, utilizing CRISPR-based gene editing technologies for the
treatment of ocular diseases.
Upcoming Event
The Company will participate in the Jefferies London Healthcare
Conference, taking place November 14-16 in London.
Upcoming Milestones
The Company has set forth the following expected milestones for
pipeline progression:
- NTLA-2001 for ATTR
amyloidosis:
- Initiate a global pivotal study for
NTLA-2001 for ATTR-CM by year-end 2023.
- Prepare for a Phase 3 study of
NTLA-2001 for the treatment of ATTRv-PN, including discussions with
regulatory authorities.
- NTLA-2002 for HAE:
- Complete enrollment in the Phase 2
portion of the Phase 1/2 study in Q4 2023.
- NTLA-3001 for AATD:
- Submit a CTA application for
NTLA-3001 for AATD-associated lung disease in Q1 2024.
- Platform
Innovation:
- Advance novel gene editing
technologies, including DNA writing and delivery to other tissues
outside of the
liver.
Third Quarter 2023 Financial Results
- Cash
Position: Cash, cash equivalents and
marketable securities were $992.5 million as of September 30, 2023,
compared to $1.3 billion as of December 31, 2022. The decrease was
driven by cash used to fund operations of $343.2 million. The
decrease was offset in part by $37.4 million of interest income,
$12.6 million of reimbursement from its collaborators, $16.2
million of net equity proceeds from the Company’s “At the Market”
(ATM) program and $7.7 million in proceeds from employee-based
stock plans.
- Collaboration
Revenue: Collaboration revenue decreased
by $1.3 million to $12.0 million during the third quarter of 2023,
compared to $13.3 million during the third quarter of 2022.
- R&D
Expenses: Research and development
expenses increased by $17.0 million to $113.7 million during the
third quarter of 2023, compared to $96.7 million during the third
quarter of 2022. This increase was primarily driven by the
advancement of our lead programs and personnel growth to support
these programs. Stock-based compensation expense included in
research and development expenses was $21.2 million for the third
quarter of 2023.
- G&A
Expenses: General and administrative
expenses increased by $7.3 million to $29.4 million during the
third quarter of 2023, compared to $22.1 million during the third
quarter of 2022. This increase was primarily related to an increase
in stock-based compensation of $5.3 million. Stock-based
compensation expense included in general and administrative
expenses was $14.1 million for the third quarter of 2023.
- Net
Loss: The Company’s net loss was $122.2
million for the third quarter of 2023, compared to $113.2 million
during the third quarter of 2022.
Conference Call to Discuss Third Quarter 2023
Results
The Company will discuss these results on a conference call
today, Thursday, November 9 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 November 9 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 X (formerly known as 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 transthyretin (“ATTR”) amyloidosis and NTLA-2002 for
the treatment of hereditary angioedema (“HAE”) pursuant to its
clinical trial application (“CTA”), including the expected timing
of data releases, regulatory feedback, 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 planned completion of enrollment of the NTLA-2002 Phase
2 study in patients with HAE; the planned initiation of a global
pivotal Phase 3 MAGNITUDE trial for NTLA-2001 by the end of 2023;
the planned initiation of a global pivotal Phase 3 study of
NTLA-2002 as early as Q3 2024, subject to regulatory feedback; the
advancement of development candidates, such as NTLA-3001 for the
treatment of alpha-1 antitrypsin deficiency (“AATD”)-associated
lung disease; 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 planned CTA submission for
NTLA-3001 for AATD in Q1 2024; 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 related research milestones and delivery to other
tissues outside of the liver; its ability to advance additional in
vivo and ex vivo 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 expanded research collaboration to develop
additional in vivo CRISPR-based gene editing therapies focused on
neurological and muscular diseases and its collaboration with
SparingVision to develop novel genomic medicines utilizing
CRISPR-based gene editing technologies for the treatment of ocular
diseases; and its 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 delay
of planned clinical trials due to regulatory feedback or other
developments and risks related to Intellia’s collaborations with
Regeneron Pharmaceuticals, Inc., SparingVision, 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. 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 September 30, |
|
Nine Months Ended September 30, |
|
|
|
|
|
2023 |
|
|
|
2022 |
|
|
|
2023 |
|
|
|
2022 |
|
Collaboration revenue |
$ |
11,992 |
|
|
$ |
13,266 |
|
|
$ |
38,192 |
|
|
$ |
38,548 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
Research and development |
|
113,696 |
|
|
|
96,651 |
|
|
|
326,088 |
|
|
|
319,945 |
|
|
General and administrative |
|
29,403 |
|
|
|
22,145 |
|
|
|
87,503 |
|
|
|
66,680 |
|
|
|
Total operating expenses |
|
143,099 |
|
|
|
118,796 |
|
|
|
413,591 |
|
|
|
386,625 |
|
Operating loss |
|
|
(131,107 |
) |
|
|
(105,530 |
) |
|
|
(375,399 |
) |
|
|
(348,077 |
) |
Other income (expense), net: |
|
|
|
|
|
|
|
Interest income |
|
12,740 |
|
|
|
1,945 |
|
|
|
37,373 |
|
|
|
3,188 |
|
Loss from equity method investment |
|
(3,857 |
) |
|
|
(1,834 |
) |
|
|
(10,905 |
) |
|
|
(7,831 |
) |
Change in fair value of contingent consideration |
|
- |
|
|
|
(7,810 |
) |
|
|
(100 |
) |
|
|
(8,059 |
) |
|
|
Total other income (expense), net |
|
8,883 |
|
|
|
(7,699 |
) |
|
|
26,368 |
|
|
|
(12,702 |
) |
Net loss |
|
$ |
(122,224 |
) |
|
$ |
(113,229 |
) |
|
$ |
(349,031 |
) |
|
$ |
(360,779 |
) |
Net loss per share, basic and diluted |
$ |
(1.38 |
) |
|
$ |
(1.49 |
) |
|
$ |
(3.96 |
) |
|
$ |
(4.78 |
) |
Weighted average shares outstanding, basic and diluted |
|
88,645 |
|
|
|
76,047 |
|
|
|
88,204 |
|
|
|
75,543 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
INTELLIA THERAPEUTICS, INC. |
CONSOLIDATED BALANCE SHEET DATA (UNAUDITED) |
(Amounts in thousands) |
|
|
|
|
|
|
|
September 30, 2023 |
|
December 31, 2022 |
Cash, cash equivalents and marketable securities |
|
$ |
992,540 |
|
|
$ |
1,261,960 |
|
Total assets |
|
|
1,243,349 |
|
|
|
1,520,114 |
|
Total liabilities |
|
|
205,935 |
|
|
|
284,530 |
|
Total stockholders' equity |
|
|
1,037,414 |
|
|
|
1,235,584 |
|
|
|
|
|
|
Intellia Contacts:
Investors:Ian KarpSenior Vice President,
Investor Relations and Corporate
Communicationsian.karp@intelliatx.comLina LiSenior Director,
Investor Relations and Corporate
Communicationslina.li@intelliatx.com
Media:Matt CrensonTen Bridge
Communicationsmedia@intelliatx.com
mcrenson@tenbridgecommunications.com
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