Intellia Therapeutics Announces Second Quarter 2022 Financial Results and Highlights Recent Company Progress
04 Août 2022 - 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 second quarter
ended June 30, 2022.
“We continue to make excellent progress in both the
cardiomyopathy and polyneuropathy arms of the landmark Phase 1
study of NTLA-2001,” said Intellia President and Chief Executive
Officer John Leonard, M.D. “In June, we presented durability data
demonstrating deep reductions in a disease-causing protein were
sustained over time following a single-dose treatment.
Additionally, we’ve completed the dose-escalation portion of the
cardiomyopathy arm and look forward to starting the dose-expansion
portion soon.”
Dr. Leonard continued, “As the leading full-spectrum genome
editing company, our ex vivo capabilities and platform are also
advancing. We believe our proprietary, differentiated cell
engineering platform can solve many of the known challenges faced
by both autologous and current allogeneic approaches. As a result,
we plan to focus exclusively on developing allogeneic cell
therapies, including an allogeneic version of NTLA-5001. Finally,
we expect several important milestones later this year, which will
include interim clinical data updates from the NTLA-2001 and
NTLA-2002 programs.”
Second 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.
- To date, over 30 patients have been
dosed across the polyneuropathy and cardiomyopathy arms. The
growing body of data, particularly at therapeutically relevant
doses, demonstrated treatment with NTLA-2001 resulted in rapid,
deep and consistent reductions of serum TTR.
- ATTR-CM arm:
Intellia announced today the completion of the dose-escalation
portion of the cardiomyopathy arm. The Company is finalizing
selection of a fixed dose, at or near the fixed-dose equivalent of
the 0.7 mg/kg dose, for evaluation in the dose-expansion portion of
the study, subject to regulatory approval. The selection is based
on clinical data from patients with ATTR-CM dosed at the 0.7 mg/kg
and 1.0 mg/kg doses, which yielded similar TTR reductions.
Additionally, both doses were generally well-tolerated. The Company
remains on track to present interim data from the cardiomyopathy
arm later this year.
- ATTRv-PN arm: The
Company announced today, subject to regulatory approval, plans to
add a second cohort to the dose-expansion portion of the
polyneuropathy arm, which will evaluate the same fixed dose
selected for the dose-expansion portion of the cardiomyopathy arm.
The decision to study a second dose is based on the following: (1)
the emerging data from the dose-escalation portion of the
cardiomyopathy arm showed similar serum TTR reduction at both the
0.7 mg/kg and 1.0 mg/kg doses, (2) the comparability of performance
at the 0.7 mg/kg and 1.0 mg/kg doses in the dose-escalation portion
of the polyneuropathy arm, which led to an 86% and 93% mean and 97%
and 98% maximum TTR reduction at day 28, respectively, and (3) a
significant elevation in liver enzymes, which normalized without
medical intervention, observed at day 28 in a patient treated in
the dose-expansion portion of the polyneuropathy arm at the 80 mg
dose (the fixed dose corresponding to 1.0 mg/kg). While the adverse
event is considered possibly related to study drug, this patient
was asymptomatic, had no increase in bilirubin and the event was
deemed nonserious by the investigator.
- Intellia plans to submit a protocol
amendment to evaluate a fixed dose corresponding to 0.7 mg/kg in
the dose-expansion portion, with enrollment across both arms
expected to be completed by the end of 2022, subject to regulatory
feedback.
- In June, Intellia presented updated
interim data from its ongoing Phase 1 study of NTLA-2001 in
patients with ATTRv-PN at the European Association for the Study of
the Liver International Liver Congress™ 2022. Extended follow-up
data from 15 ATTRv-PN patients, treated across all four
single-ascending dose cohorts, showed deep, dose-dependent
reductions in serum TTR observed with prior readouts were sustained
through the last measured timepoint of follow-up, reaching 12
months in the 0.1 mg/kg and 0.3 mg/kg cohorts and six months in the
0.7 mg/kg and 1.0 mg/kg cohorts. Both 0.7 mg/kg and 1.0 mg/kg doses
led to greater than 85% mean TTR reduction at day 28. The
durability and persistence of effect continue to support NTLA-2001
as a potential one-time treatment to permanently inactivate the TTR
gene and reduce the disease-causing protein.
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 is progressing the
single-ascending dose portion of its first-in-human study. The
Company anticipates presenting interim data in the second half of
2022, including safety, kallikrein reduction and HAE attack rate
data. These initial results are expected to characterize the
emerging safety and activity profile of NTLA-2002 and potentially
demonstrate the modularity of Intellia’s proprietary CRISPR-based,
LNP platform.
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
Acute Myeloid Leukemia (AML)
- NTLA-5001:
NTLA-5001 is an investigational autologous T cell receptor (TCR)-T
cell therapy engineered to target the Wilms’ Tumor 1 (WT1) antigen
for the treatment of all genetic subtypes of AML. In March,
Intellia announced that the first patient was dosed in the Phase
1/2a first-in-human trial of NTLA-5001.
- Intellia has decided to concentrate
its ex vivo development efforts exclusively on allogeneic cell
therapies manufactured from healthy donors. The Company’s
proprietary technologies, including its LNP-based cell engineering
platform and novel allogeneic solution, offer significant
advantages over both autologous and current investigational
allogeneic approaches. Preclinical data presented on its
differentiated allogeneic engineering platform showed allogeneic T
cells were shielded from immune rejection, both host T and natural
killer (NK) cell attack.
- Intellia announced today plans to
discontinue its first-in-human study of NTLA-5001, an
investigational autologous TCR-T cell therapy, and is pivoting to
an allogeneic version of this program currently in preclinical
development. This decision is not due to any safety or efficacy
data emerging from the trial. It is instead based on the potential
of Intellia’s allogeneic platform to consistently deliver a
high-quality, readily available and persistent cell product for
treatment of aggressive cancers.
- Preclinical data supporting the
development of a WT1-directed allogeneic TCR-T cell candidate will
be presented at a future scientific conference in 2022.
CD30+ Lymphomas
- NTLA-6001:
NTLA-6001 is a wholly owned, 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 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.
- The Company plans to highlight
additional advances to its proprietary technology capabilities,
including both genome editing and delivery tools, at upcoming
scientific conferences in 2022.
Upcoming Milestones
The Company has set forth the following for pipeline
progression:
In Vivo
- NTLA-2001 for ATTR amyloidosis:
- Present interim data from ATTR-CM
arm of Phase 1 study in 2H 2022
- Complete enrollment of Phase 1
study for both ATTRv-PN and ATTR-CM subjects by the end of
2022
- NTLA-2002 for HAE: Present interim
data from Phase 1/2 study in 2H 2022
- 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
Ex Vivo
- Present on its proprietary
allogeneic cell engineering platform at an upcoming scientific
conference in 2022
Modular Platform
- Advance additional novel platform
capabilities in 2022
Second Quarter 2022 Financial Results
- Cash
Position: Cash, cash equivalents and
marketable securities were $906.9 million as of June 30, 2022,
compared to $1.1 billion as of December 31, 2021. The decrease was
driven by cash used to fund operations of approximately $191.2
million as well as the acquisition of Rewrite for $45.0 million.
The decrease was offset in part by $38.9 million in net equity
proceeds raised from the Company’s “At the Market” (ATM) agreement
and $14.3 million in proceeds from employee-based stock plans.
- Collaboration
Revenue: Collaboration revenue increased
by approximately $7.5 million to $14.0 million during the second
quarter of 2022, compared to $6.6 million during the second quarter
of 2021. The increase was primarily driven by our collaborations
with AvenCell and Kyverna.
- R&D
Expenses: Research and development
expenses increased by $31.3 million to $90.2 million during the
second quarter of 2022, compared to $58.9 million during the second
quarter of 2021. This increase was primarily driven by the
advancement of our lead programs, research personnel growth to
support these programs and expansion of the development
organization.
- G&A
Expenses: General and administrative
expenses increased by $5.4 million to $22.1 million during the
second quarter of 2022, compared to $16.7 million during the second
quarter of 2021. This increase was primarily related to
employee-related expenses, including stock-based compensation of
$4.5 million.
- Net
Loss: The Company’s net loss was $100.7
million for the second quarter of 2022, compared to $68.8 million
during the second quarter of 2021.
Conference Call to Discuss Second Quarter 2022
Results
The Company will discuss these results on a conference call
today, Thursday, August 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 August 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; the safety,
efficacy, success and advancement of its clinical programs for
NTLA-2001 for the treatment of ATTR amyloidosis, NTLA-2002 for the
treatment of hereditary angioedema (“HAE”) and NTLA-5001 for the
treatment of acute myeloid leukemia (“AML”) pursuant to its
clinical trial applications (“CTA”) and investigational new drug
(“IND”) submissions, including the expected timing of data
releases, regulatory filings, and the initiation, enrollment, and
completion of clinical trials; its ability to successfully pivot to
an allogeneic version of its NTLA-5001 program for the treatment of
AML, including the expected timing of data releases and regulatory
filings; 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; 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 maintain and expand its related
intellectual property portfolio; expectations of 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 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.
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 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 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) |
|
|
|
|
|
|
|
|
|
Three Months Ended June 30, |
|
Six Months Ended June 30, |
|
|
2022 |
|
|
|
2021 |
|
|
|
2022 |
|
|
|
2021 |
|
Collaboration revenue |
$ |
14,030 |
|
|
$ |
6,550 |
|
|
$ |
25,282 |
|
|
$ |
12,995 |
|
Operating expenses: |
|
|
|
|
|
|
|
Research and development |
|
90,199 |
|
|
|
58,884 |
|
|
|
223,294 |
|
|
|
98,160 |
|
General and administrative |
|
22,132 |
|
|
|
16,683 |
|
|
|
44,535 |
|
|
|
30,277 |
|
Total operating expenses |
|
112,331 |
|
|
|
75,567 |
|
|
|
267,829 |
|
|
|
128,437 |
|
Operating loss |
|
(98,301 |
) |
|
|
(69,017 |
) |
|
|
(242,547 |
) |
|
|
(115,442 |
) |
Other (expense) income, net: |
|
|
|
|
|
|
|
Interest income |
|
703 |
|
|
|
211 |
|
|
|
1,243 |
|
|
|
431 |
|
Loss from equity method investment |
|
(3,252 |
) |
|
|
- |
|
|
|
(5,997 |
) |
|
|
- |
|
Change in fair value of contingent consideration |
|
172 |
|
|
|
- |
|
|
|
(249 |
) |
|
|
- |
|
Total other (expense) income, net |
|
(2,377 |
) |
|
|
211 |
|
|
|
(5,003 |
) |
|
|
431 |
|
Net loss |
$ |
(100,678 |
) |
|
$ |
(68,806 |
) |
|
$ |
(247,550 |
) |
|
$ |
(115,011 |
) |
Net loss per share, basic and diluted |
$ |
(1.33 |
) |
|
$ |
(1.01 |
) |
|
$ |
(3.29 |
) |
|
$ |
(1.70 |
) |
Weighted average shares outstanding, basic and diluted |
|
75,823 |
|
|
|
68,164 |
|
|
|
75,282 |
|
|
|
67,675 |
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INTELLIA THERAPEUTICS, INC. |
CONSOLIDATED BALANCE SHEET DATA (UNAUDITED) |
(Amounts in thousands) |
|
|
|
|
|
June 30, 2022 |
|
December 31, 2021 |
Cash, cash equivalents and marketable securities |
$ |
906,879 |
|
|
$ |
1,086,049 |
|
Total assets |
|
1,110,424 |
|
|
|
1,294,464 |
|
Total liabilities |
|
229,877 |
|
|
|
254,220 |
|
Total stockholders' equity |
|
880,547 |
|
|
|
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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