Intellia Therapeutics Announces Second Quarter 2023 Financial Results and Highlights Recent Company Progress
August 03 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 second quarter
ended June 30, 2023.
“We continue to make significant and rapid progress in the
development of our first two in vivo, CRISPR-based therapeutic
candidates,” said Intellia President and Chief Executive Officer
John Leonard, M.D. “For NTLA-2002, we are thrilled to see the
excitement for this investigational therapy from the HAE community.
In just a handful of months, we were able to identify all patients
required to fully enroll the ongoing Phase 2 study and now expect
to initiate the pivotal Phase 3 program next year. For NTLA-2001,
our team has been working diligently to prepare for an IND
submission in September and to begin the Phase 3 study for the
cardiomyopathy manifestation of ATTR amyloidosis before the end of
this year. Overall, we continue to believe that NTLA-2001 and
NTLA-2002 represent only the beginning for our pipeline and
platform as additional in vivo and ex vivo candidates advance
toward the clinic.”
Second 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 an
Investigational New Drug (IND) application to the U.S. Food and
Drug Administration (FDA) in September. Subject to regulatory
feedback, the Company anticipates initiating a global pivotal study
for ATTR-CM by year-end 2023.
- The Company expects to present
additional data from the ATTR-CM arm of the Phase 1 study by
year-end 2023, including longer-term safety and durability data, as
well as emerging clinical endpoints.
- Hereditary ATTR Amyloidosis
with Polyneuropathy (ATTRv-PN):
- The Company is actively preparing
for a global pivotal Phase 3 study, including discussions with
regulatory authorities.
- The Company plans to present
additional data from the ATTRv-PN arm of the Phase 1 study by
year-end 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 due
to the substantial interest from physicians and patients to
participate in the NTLA-2002 clinical program, all patients have
been identified for the global Phase 2 portion of the study with
enrollment expected to be completed in the second half of
2023.
- Based on the strong momentum of the
program, the Company plans to complete enrollment at ex-U.S. sites
in the ongoing Phase 2 study. Further, following the March 2023 IND
clearance, the FDA requested supplemental preclinical data related
to the inclusion of female patients of child-bearing potential.
Intellia expects to submit these data in advance of the planned
Phase 3 trial, which will complement the clinical data collected
from female patients of child-bearing potential dosed in the
ongoing Phase 1/2 study.
- 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.
- In June, Intellia announced
additional positive interim results from the Phase 1 portion of the
ongoing Phase 1/2 study of NTLA-2002. Across all 10 patients, a 95%
mean reduction in monthly attack rate was observed after a single
dose of NTLA-2002 through the latest follow-up. The median duration
of follow-up was 9.0 months (range of 5.6 - 14.1 months). At all
three dose levels evaluated, NTLA-2002 has been well tolerated, and
the majority of adverse events were mild in severity. These interim
data were presented at the European Academy of Allergy and Clinical
Immunology (EAACI) Hybrid Congress 2023, held in Hamburg, Germany,
and virtually.
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 a Clinical Trial
Application (CTA) by year-end 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 became chair of the audit committee
on June 15, 2023. In June, Intellia announced the retirement
of Jean-François Formela, M.D., from its board of directors,
effective June 15, 2023.
Upcoming Events
The Company will participate in the following events during the
third quarter of 2023:
- Wells Fargo Healthcare Conference,
September 6, Everett, Massachusetts
- BMO Gene Editing Event, September
26, virtual
- Cantor Global Healthcare
Conference, September 27, New York City
Upcoming Milestones
The Company has set forth the following expected milestones for
pipeline progression:
- NTLA-2001 for ATTR
amyloidosis:
- Submit an IND application in
September 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 by year-end 2023.
- Initiate a global pivotal study for
NTLA-2001 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:
- Complete enrollment in the Phase 2
portion of the Phase 1/2 study in 2H 2023.
- AATD Franchise:
- Submit a CTA application for
NTLA-3001 for AATD-associated lung disease by year-end 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.
Second Quarter 2023 Financial Results
- Cash
Position: Cash, cash equivalents and
marketable securities were $1.1 billion as of June 30, 2023,
compared to $1.3 billion as of December 31, 2022. The decrease was
driven by cash used to fund operations of approximately $227.3
million. The decrease was offset in part by $24.6 million of
interest income, $8.0 million of reimbursement from its
collaborators, $1.5 million of net equity proceeds from the
Company’s “At the Market” (ATM) program and $3.3 million in
proceeds from employee-based stock plans.
- Collaboration
Revenue: Collaboration revenue decreased
by $0.4 million to $13.6 million during the second quarter of 2023,
compared to $14.0 million during the second quarter of 2022.
- R&D
Expenses: Research and development
expenses increased by approximately $25.1 million to $115.3 million
during the second quarter of 2023, compared to $90.2 million during
the second 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 $22.4 million for the
second quarter of 2023.
- G&A
Expenses: General and administrative
expenses increased by $8.5 million to $30.7 million during the
second quarter of 2023, compared to $22.1 million during the second
quarter of 2022. This increase was primarily related to an increase
in stock-based compensation of $5.1 million. Stock-based
compensation expense included in general and administrative
expenses was $14.0 million for the second quarter of 2023.
- Net
Loss: The Company’s net loss was $123.7
million for the second quarter of 2023, compared to $100.7 million
during the second quarter of 2022.
Conference Call to Discuss Second Quarter 2023
Results
The Company will discuss these results on a conference call
today, Thursday, August 3 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 August 3 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 application (“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 planned submission of an IND application for NTLA-2001 in
September 2023 and planned initiation of a global pivotal 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 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 planned CTA submission for NTLA-3001 by the
end of 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 novel technologies, including DNA writing 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 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. 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 June 30, |
|
Six Months Ended June 30, |
|
2023 |
|
2022 |
|
2023 |
|
2022 |
Collaboration revenue |
$ |
13,594 |
|
|
$ |
14,030 |
|
|
$ |
26,200 |
|
|
$ |
25,282 |
|
Operating
expenses: |
|
|
|
|
|
|
|
|
|
|
|
Research and development |
115,276 |
|
|
90,199 |
|
|
212,392 |
|
|
223,294 |
|
General and administrative |
30,652 |
|
|
22,132 |
|
|
58,100 |
|
|
44,535 |
|
Total operating expenses |
145,928 |
|
|
112,331 |
|
|
270,492 |
|
|
267,829 |
|
Operating
loss |
(132,334 |
) |
|
(98,301 |
) |
|
(244,292 |
) |
|
(242,547 |
) |
Other income
(expense), net: |
|
|
|
|
|
|
|
|
|
|
|
Interest income |
12,653 |
|
|
703 |
|
|
24,633 |
|
|
1,243 |
|
Loss from equity method investment |
(4,000 |
) |
|
(3,252 |
) |
|
(7,048 |
) |
|
(5,997 |
) |
Change in fair value of contingent consideration |
- |
|
|
172 |
|
|
(100 |
) |
|
(249 |
) |
Total other income (expense), net |
8,653 |
|
|
(2,377 |
) |
|
17,485 |
|
|
(5,003 |
) |
Net loss |
$ |
(123,681 |
) |
|
$ |
(100,678 |
) |
|
$ |
(226,807 |
) |
|
$ |
(247,550 |
) |
Net loss per
share, basic and diluted |
$ |
(1.40 |
) |
|
$ |
(1.33 |
) |
|
$ |
(2.58 |
) |
|
$ |
(3.29 |
) |
Weighted average
shares outstanding, basic and diluted |
88,185 |
|
|
75,823 |
|
|
87,979 |
|
|
75,282 |
|
|
|
|
|
|
|
|
|
|
|
|
|
INTELLIA
THERAPEUTICS, INC. |
CONSOLIDATED
BALANCE SHEET DATA (UNAUDITED) |
(Amounts in
thousands) |
|
|
|
|
|
|
|
|
|
June 30, 2023 |
|
December 31, 2022 |
Cash, cash
equivalents and marketable securities |
$ |
1,072,087 |
|
|
$ |
1,261,960 |
|
Total
assets |
|
1,323,240 |
|
|
|
1,520,114 |
|
Total
liabilities |
|
218,352 |
|
|
|
284,530 |
|
Total
stockholders' equity |
|
1,104,888 |
|
|
|
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:Matt CrensonTen Bridge
Communications+1-917-640-7930media@intelliatx.com
mcrenson@tenbridgecommunications.com
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