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, 2022.
“During the most recent quarter, Intellia achieved significant
clinical milestones across two landmark first-in-human studies,”
said Intellia President and Chief Executive Officer John Leonard,
M.D. “We believe the latest data from our lead in vivo program,
NTLA-2001, indicate it has the potential to set a new standard for
how people living with ATTR amyloidosis are treated for this
life-threatening disease. We are rapidly completing the
dose-expansion portion of the study and are engaging with
regulatory agencies, including in the U.S., as we move closer to a
global, pivotal trial. Interim data from our second in vivo
program, NTLA-2002, demonstrated robust reductions in both plasma
kallikrein levels and the rate of swelling attacks in patients with
hereditary angioedema. These initial data support the potential for
a single dose of NTLA-2002 to permanently prevent the debilitating
and potentially fatal HAE attacks that characterize this lifelong
genetic disease. We look forward to presenting additional data from
both NTLA-2001 and NTLA-2002 later this month.”
Dr. Leonard continued, “Together, these interim datasets
demonstrate the modularity of our CRISPR-based platform, providing
powerful evidence of our ability to apply this technology broadly
to address a wide range of diseases. Beyond gene knockout programs,
we are advancing our first in vivo insertion candidate, NTLA-3001
for the treatment of alpha-1 antitrypsin deficiency, with plans to
submit a regulatory filing next year.”
Third Quarter 2022 and Recent Operational
Highlights
In Vivo Program Updates
Transthyretin (ATTR) Amyloidosis
- NTLA-2001:
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 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 being evaluated in a Phase 1, two-part,
open-label study in adults with hereditary transthyretin
amyloidosis with polyneuropathy (ATTRv-PN) or transthyretin
amyloidosis with cardiomyopathy (ATTR-CM). NTLA-2001 is subject to
a co-development/co-promotion agreement between Intellia, the lead
party for this program, and Regeneron Pharmaceuticals, Inc.
- ATTR-CM arm:
Intellia announced in September positive interim results from the
cardiomyopathy arm of the ongoing Phase 1 clinical trial of
NTLA-2001. The interim data were from 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 were
administered via intravenous infusion, and the change from baseline
in serum TTR protein concentration was measured for each patient.
These data showed deep and sustained mean serum TTR reductions of
93% and 92% at the 0.7 mg/kg and 1.0 mg/kg doses, respectively, at
day 28. At both dose levels, NTLA-2001 was generally
well-tolerated. Two of 12 patients reported transient infusion
reactions, which was the only observed treatment-related adverse
event. One patient in the 0.7 mg/kg dose NYHA Class III cohort
experienced a Grade 3 infusion-related reaction, which resolved
without clinical consequence. No clinically significant laboratory
abnormalities were observed at either dose level. These data
support NTLA-2001’s potential as a one-time treatment to
permanently inactivate the TTR gene and reduce the disease-causing
protein in people with ATTR-CM.
- Intellia initiated dosing at a 55
mg dose, the fixed dose corresponding to 0.7 mg/kg, in Part 2, the
dose-expansion portion of the study. The Company remains on track
to complete, by the end of this year, planned enrollment of both
arms of the Phase 1 study that will inform the dose selection for
subsequent pivotal studies.
- Intellia will be presenting interim
clinical data from the cardiomyopathy arm in a Late-Breaking
Science session on November 5, 2022, at this year’s American Heart
Association (AHA) Scientific Sessions, taking place in Chicago,
Illinois.
Hereditary Angioedema (HAE)
- NTLA-2002:
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 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, and 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 September
positive interim results from an ongoing Phase 1/2 clinical study
of NTLA-2002, its second in vivo genome editing candidate, at the
2022 Bradykinin Symposium held in Berlin, Germany. Administration
of single doses of NTLA-2002 led to dose-dependent reductions in
plasma kallikrein, with mean reductions of 65% and 92% in the 25 mg
(n=3) and 75 mg (n=3) dose cohorts, respectively, by week eight. In
addition to plasma kallikrein levels, HAE attack rates are also
being measured in the study, with the first analysis occurring at
the end of the pre-specified 16-week primary observation period. A
single dose of 25 mg of NTLA-2002 resulted in a 91% mean reduction
in HAE attacks through the 16-week observation period.
Additionally, two of the three patients have not had a single HAE
attack since treatment, and all three patients have been
attack-free since week 10 (based on follow-up through weeks 24 –
32). Patients in the 75 mg cohort had not completed the primary
16-week observation period. At both dose levels, NTLA-2002 was
generally well-tolerated, and the majority of adverse events were
mild in severity.
- Intellia will be presenting
additional data at the American College of Allergy, Asthma &
Immunology (ACAAI) Annual Scientific Meeting, taking place November
10 – 14 in Louisville, Kentucky. The new presentation is expected
to include interim safety and kallikrein reduction data from the 50
mg dose cohort, and additional safety, kallikrein reduction and
attack rate data from the 25 mg and 75 mg dose cohorts from the
dose-escalation portion of the study.
- Intellia expects to select up to
two doses to further evaluate in the Phase 2, placebo-controlled,
dose-expansion portion of the study, slated to begin in the first
half of 2023. Intellia anticipates expanding country and site
participation, including U.S. clinical sites, as part of the Phase
2 study.
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 IV infusions of A1AT augmentation therapy or lung
transplant in severe cases.
- Intellia is conducting
Investigational New Drug (IND)-enabling activities for NTLA-3001,
with plans to file an IND or IND-equivalent in 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.
Ex Vivo Program Updates
CD30+ Lymphomas
- NTLA-6001:
NTLA-6001 is an allogeneic CAR-T development candidate targeting
CD30 for the treatment of CD30-expressing hematologic cancers,
including relapsed or refractory classical Hodgkin lymphoma (cHL).
NTLA-6001 is the first wholly owned candidate developed using
Intellia’s proprietary allogeneic cell engineering platform.
- Intellia is conducting IND-enabling
activities for NTLA-6001.
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.
- Intellia plans to advance at least
one additional new in vivo development candidate by the end of
2022.
- Allogeneic
Platform: In October, at the European Society of Gene
& Cell Therapy 29th Congress, Intellia highlighted its
proprietary allogeneic solution to create engineered T cells with
high anti-tumor activity, which have the potential to be uniquely
capable of persisting in the patient to maintain durable responses.
Notably, a novel combination of gene edits, including knockout of
HLA Class II and HLA-A while retaining HLA-B and HLA-C proteins,
yielded T cells capable of avoiding rejection by host T and natural
killer (NK) cells in preclinical models. By only matching for HLA-B
and HLA-C with homozygous donors for a 2/2 match, Intellia’s
approach allows for a simplified HLA matching strategy between
healthy donor T cells and recipient patients. Development of such
an “off-the-shelf” therapy aims to address the majority of the
patient population with only a small set of donors. Intellia’s
allogeneic platform is being deployed for investigational TCR-T and
CAR-T cell therapies.
Upcoming Events
The Company will participate in the following events during the
fourth quarter of 2022:
- American Heart Association
Scientific Sessions 2022, November 5, Chicago
- American College of Allergy, Asthma
& Immunology 2022 Annual Scientific Meeting, November 12,
Louisville
- Piper Sandler 34th Annual
Healthcare Conference, November 29, New York
Upcoming Milestones
The Company has set forth the following for pipeline
progression:
In Vivo
- NTLA-2001 for ATTR amyloidosis:
- Present additional interim data
from ATTR-CM arm of Phase 1 study at AHA
- Complete planned enrollment of
Phase 1 study for both ATTRv-PN and ATTR-CM subjects by the end of
this year
- NTLA-2002 for HAE:
- Present additional interim data
from Phase 1/2 study at ACAAI
- Initiate Phase 2 portion of the
study in 1H 2023
- NTLA-3001 for AATD: File an IND or
IND-equivalent in 2023
- Advance at least one additional new
in vivo development candidate by the end of 2022
Third Quarter 2022 Financial Results
- Cash
Position: Cash, cash equivalents and
marketable securities were $848.7 million as of September 30, 2022,
compared to $1.1 billion as of December 31, 2021. The decrease was
driven by cash used to fund operations of approximately $276.2
million as well as the acquisition of Rewrite for $45.0 million.
The decrease was offset in part by $62.1 million in net equity
proceeds raised from the Company’s “At the Market” (ATM) agreement
and $15.1 million in proceeds from employee-based stock plans.
Subsequent to the end of the third quarter, through October 27,
2022, the Company increased its cash position by approximately $115
million. This included an additional $107.2 million in net equity
proceeds raised from its ATM agreement and an additional $8.3
million received for ATM transactions with trade dates in September
that were settled in October.
- Collaboration
Revenue: Collaboration revenue increased
by approximately $6.1 million to $13.3 million during the third
quarter of 2022, compared to $7.2 million during the third quarter
of 2021. The increase was primarily driven by our collaborations
with AvenCell and Kyverna.
- R&D
Expenses: Research and development
expenses increased by $36.2 million to $96.7 million during the
third quarter of 2022, compared to $60.5 million during the third
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 $3.4 million to $22.1 million during the
third quarter of 2022, compared to $18.7 million during the third
quarter of 2021. This increase was primarily related to
employee-related expenses, including stock-based compensation of
$2.4 million.
- Net
Loss: The Company’s net loss was $113.2
million for the third quarter of 2022, compared to $71.6 million
during the third quarter of 2021.
Conference Call to Discuss Third Quarter 2022
Results
The Company will discuss these results on a conference call
today, Thursday, November 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 November 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; 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 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 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; the expansion of its CRISPR/Cas9 technology
and related 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 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 September 30, |
|
Nine Months Ended September 30, |
|
|
2022 |
|
|
|
2021 |
|
|
|
2022 |
|
|
|
2021 |
|
Collaboration revenue |
$ |
13,266 |
|
|
$ |
7,204 |
|
|
$ |
38,548 |
|
|
$ |
20,199 |
|
Operating
expenses: |
|
|
|
|
|
|
|
Research and development |
|
96,651 |
|
|
|
60,486 |
|
|
|
319,945 |
|
|
|
158,646 |
|
General and administrative |
|
22,145 |
|
|
|
18,711 |
|
|
|
66,680 |
|
|
|
48,988 |
|
Total operating expenses |
|
118,796 |
|
|
|
79,197 |
|
|
|
386,625 |
|
|
|
207,634 |
|
Operating
loss |
|
(105,530 |
) |
|
|
(71,993 |
) |
|
|
(348,077 |
) |
|
|
(187,435 |
) |
Other
(expense) income, net: |
|
|
|
|
|
|
|
Interest income |
|
1,945 |
|
|
|
349 |
|
|
|
3,188 |
|
|
|
780 |
|
Loss from equity method investment |
|
(1,834 |
) |
|
|
- |
|
|
|
(7,831 |
) |
|
|
- |
|
Change in fair value of contingent consideration |
|
(7,810 |
) |
|
|
- |
|
|
|
(8,059 |
) |
|
|
- |
|
Total other (expense) income, net |
|
(7,699 |
) |
|
|
349 |
|
|
|
(12,702 |
) |
|
|
780 |
|
Net
loss |
$ |
(113,229 |
) |
|
$ |
(71,644 |
) |
|
$ |
(360,779 |
) |
|
$ |
(186,655 |
) |
Net loss per
share, basic and diluted |
$ |
(1.49 |
) |
|
$ |
(0.97 |
) |
|
$ |
(4.78 |
) |
|
$ |
(2.68 |
) |
Weighted
average shares outstanding, basic and diluted |
|
76,047 |
|
|
|
73,706 |
|
|
|
75,543 |
|
|
|
69,720 |
|
INTELLIA
THERAPEUTICS, INC. |
CONSOLIDATED
BALANCE SHEET DATA (UNAUDITED) |
(Amounts in
thousands) |
|
|
|
|
|
September 30, 2022 |
|
December 31, 2021 |
Cash, cash equivalents and marketable securities |
$ |
848,727 |
|
$ |
1,086,049 |
Total
assets |
|
1,084,458 |
|
|
1,294,464 |
Total
liabilities |
|
259,507 |
|
|
254,220 |
Total
stockholders' equity |
|
824,951 |
|
|
1,040,244 |
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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