Intellia Therapeutics Announces Second Quarter 2021 Financial Results and Highlights Recent Company Progress
05 Août 2021 - 1:30PM
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage
genome editing company focused on developing curative therapeutics
using CRISPR/Cas9 technology both in vivo and ex vivo, today
reported financial results for the second quarter ended June 30,
2021, and recent operational highlights.
“This quarter marked an exciting new chapter for Intellia. We
presented the first clinical data in history supporting precision
editing of a disease-causing gene within the body following a
single, systemic dose of CRISPR/Cas9. These data open a new era of
medicine – one that holds the potential of curing genetic disease.
The interim data from our ongoing Phase 1 trial of NTLA-2001
support its potential to halt and even reverse the relentless
progression of ATTR amyloidosis with one dose. More broadly, they
offer proof of concept for our LNP-based platform for systemic in
vivo delivery. With these results, we believe we are unlocking the
treatment of diseases that originate in the liver and introducing a
rapid and reproducible development path that accelerates our in
vivo portfolio,” said Intellia President and Chief Executive
Officer John Leonard, M.D.
“In addition, together with Blackstone and Cellex, we launched a
new company combining our allogeneic cell engineering platform with
a clinically validated switchable, universal CAR-T construct. This
new venture confers numerous benefits to Intellia, extending the
reach of our technology beyond our core internal focus and giving
us a meaningful stake in the company’s success, even as we retain
our rights across a diverse ex vivo landscape. This transaction
also fortified our leading cell engineering capabilities by
securing access to high-quality donor cells and dedicated
manufacturing capacity to support the development of our
wholly-owned ex vivo programs.”
Dr. Leonard continued, “We are well-positioned to build on this
quarter’s momentum as we close in on a number of upcoming
milestones. We look forward to initiating Phase 1 trials this year
for our next two candidates, NTLA-2002 for HAE and NTLA-5001 for
AML. We will also share additional interim data from NTLA-2001
later this year. With a strong financial position and a
well-validated platform, we intend to expand and accelerate
development across our full-spectrum pipeline.”
Second Quarter 2021 and Recent Operational
Highlights
- NTLA-2001 for ATTR
Amyloidosis: NTLA-2001 is the first systemically delivered
CRISPR-based therapy dosed in a patient with the potential to be a
curative 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, lifelong reduction in transthyretin (TTR) protein
after a single dose. NTLA-2001 is part of a
co-development/co-promotion agreement between Intellia, the lead
party, and Regeneron Pharmaceuticals, Inc. (Regeneron).
- In June, Intellia and Regeneron
announced positive interim clinical data from the first two cohorts
in the ongoing dose-escalation portion of a Phase 1 trial
evaluating NTLA-2001 in adults with hereditary ATTR amyloidosis
with polyneuropathy (ATTRv-PN). A single dose of either 0.1 mg/kg
or 0.3 mg/kg of NTLA-2001 was administered systemically. Among the
three patients in the 0.1 mg/kg dose group, treatment with
NTLA-2001 led to mean serum TTR reductions of 52% by day 28. In the
second cohort of three patients, a single 0.3 mg/kg dose of
NTLA-2001 exceeded that of existing ATTRv-PN therapies with an 87%
mean reduction in serum TTR levels by day 28, including one patient
with a 96% reduction. NTLA-2001 demonstrated a dose-dependent
response and an encouraging safety profile, with no serious adverse
events in the first six patients by day 28. These results, the
first-ever clinical data supporting safety and efficacy of in vivo
CRISPR genome editing in humans, were presented at the 2021
Peripheral Nerve Society Annual Meeting and published in The New
England Journal of Medicine.
- Intellia is continuing to enroll
the study to determine if higher doses could result in a deeper
reduction in serum TTR protein levels with potential to translate
into more meaningful clinical benefit. Once the recommended dose
has been identified, Part 2 of the study, a single-dose expansion
cohort, will be initiated later this year. After completion of the
Phase 1 trial, Intellia intends to move to pivotal studies for both
polyneuropathy and cardiomyopathy manifestations of ATTR
amyloidosis.
- The Company intends to present
additional interim data from the dose-escalation portion of the
Phase 1 study at a scientific or medical meeting this year.
- NTLA-2002 for HAE:
NTLA-2002 leverages Intellia’s modular in vivo LNP delivery
technology to knock out the KLKB1 gene in the liver with the
potential to permanently reduce plasma kallikrein protein and
activity, a key mediator of HAE. This approach aims to prevent
attacks for people living with HAE by providing continuous
suppression of plasma kallikrein activity following a single dose
and to eliminate the significant treatment burden associated with
currently available HAE therapies.
- In June, Intellia announced that it
had submitted its first Clinical Trial Application (CTA) to the New
Zealand Medicines and Medical Devices Safety Authority for
NTLA-2002 to initiate a first-in-human study.
- The Company expects to enroll the
first patient by year-end and is also submitting additional
regulatory applications to enable enrollment in other countries.
The first-in-human trial is expected to evaluate safety,
tolerability and activity in patients with HAE, and will continue
to leverage insights gained from the development of NTLA-2001.
- NTLA-5001 for AML:
NTLA-5001 is an 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.
- Intellia announced today it has
submitted its first CTA to the United Kingdom Medicines and
Healthcare products Regulatory Agency for NTLA-5001 to initiate a
Phase 1 study.
- The Company is submitting
additional regulatory applications and expects to initiate patient
screening in a Phase 1 study by year-end. This first-in-human trial
is expected to evaluate the safety and activity of NTLA-5001 in
patients with persistent or recurrent AML who have previously
received first-line therapies.
- Modular Platform and
Pipeline Expansion: Intellia is advancing its modular
platform technologies to broaden the in vivo and ex
vivo applications of genome editing. This includes progressing
capabilities for innovative CRISPR/Cas9-mediated targeted transgene
insertion, in vivo editing in multiple tissue types and an
allogeneic approach for the development of “off-the-shelf” T cell
therapies. These efforts will support new therapeutic candidates
for genetic diseases requiring removal and/or restoration of a
protein, as well as next-generation engineered cell therapies for
cancers and autoimmune diseases.
- In June, Intellia announced the
launch of a new universal CAR-T cell therapy company in
collaboration with Blackstone Life Sciences and Cellex Cell
Professionals GmbH (“Cellex”), which closed on July 30. The new
company will combine clinical-stage universal CAR-T platforms with
Intellia’s differentiated allogeneic cell engineering platform to
develop therapies for immuno-oncology and autoimmune diseases. The
agreement allows Intellia to advance its ex vivo pipeline with a
key stake in the new company, with options to co-develop two
allogeneic universal CAR-T candidates.
- Through a concurrent agreement with
Cellex, Intellia also established a preferred relationship
including access to donor cells and dedicated manufacturing
capacity to support the development of Intellia’s wholly-owned ex
vivo programs.
- Intellia plans to nominate at least
one additional development candidate from across its pipeline in
2021. In addition, the Company plans to nominate its first
allogeneic development candidate by the first half of 2022.
- The Company plans to present at
upcoming scientific conferences, with in vivo and ex vivo updates,
including an allogeneic solution that enables the next generation
of engineered cell therapies.
- Corporate:
- In July, the Company closed an
underwritten public offering of 4,758,620 shares of common stock,
including the exercise in full of the underwriters’ option to
purchase an additional 620,689 shares of common stock, at the
public offering price of $145.00 per share. Intellia raised
aggregate net proceeds of $648.1 million, which were net of
estimated equity issuance costs of $41.9 million.
- In June, Intellia appointed James
Basta, J.D., as Executive Vice President, General Counsel and
Corporate Secretary. Mr. Basta has over two decades of combined
in-house corporate and law firm experience and joins Intellia from
Kura Oncology, where he served as Chief Legal Officer. Earlier in
his career, Mr. Basta held various leadership roles in the legal
department at Biogen and was a Partner at Baker McKenzie.
- In July, Intellia appointed Ian
Karp as Senior Vice President, Investor Relations and Corporate
Communications. Mr. Karp brings over two decades of pharmaceutical
and biotech industry experience across investor relations,
corporate communications, corporate development and product
commercialization. Mr. Karp joins Intellia from Karyopharm
Therapeutics, where he served as Senior Vice President, Investor
and Public Relations, and was formerly the Head of Global Investor
Relations at Shire plc.
Upcoming Events
The Company plans to participate in the following events during
the third quarter of 2021:
- Wells Fargo Securities Healthcare
Conference, September 9-10, Virtual
- H.C. Wainwright 23rd Annual Global
Investment Conference, September 15, Virtual
Upcoming Milestones
The Company has set forth the following guidance for pipeline
progression:
- ATTR:
- Report additional interim clinical
data from Phase 1 study of NTLA-2001 later this year
- Initiate Part 2 of the NTLA-2001
Phase 1 study, a single-dose expansion cohort, later this year
- HAE: Initiate enrollment in the
first-in-human study of NTLA-2002 by year-end
- AML: Initiate patient screening in
the Phase 1 study of NTLA-5001 by year-end
- Pipeline Expansion:
- Nominate at least one new development candidate in 2021,
and
- Nominate the Company’s first allogeneic development candidate
by 1H 2022
Second Quarter 2021 Financial Results
- Cash
Position: Cash, cash equivalents and
marketable securities were $551.3 million as of June 30, 2021,
compared to $597.4 million as of December 31, 2020. The decrease
was driven by cash used to fund operations of approximately $115.1
million, which was offset in part by $45.3 million of net equity
proceeds from the Company’s “At the Market” (ATM) agreement, $20.5
million in proceeds from employee-based stock plans, and $3.2
million of funding for cost-sharing agreements received from
Regeneron. The $551.3 million cash position as of June 30, 2021
does not include the proceeds from the July 2021 follow-on offering
of common stock.
- Collaboration
Revenue: Collaboration revenue decreased
by $9.7 million to $6.6 million during the second quarter of 2021,
compared to $16.3 million during the second quarter of 2020. The
decrease was primarily driven by an $8.4 million one-time
cumulative catch-up adjustment related to the modification of the
2016 Regeneron agreement recorded during Q2 2020.
- R&D
Expenses: Research and development
expenses increased by $21.1 million to $58.9 million during the
second quarter of 2021, compared to $37.8 million during the second
quarter of 2020. This increase was primarily driven by a $10.0
million one-time payment related to the third amendment to the 2014
Novartis Agreement as well as employee-related expenses due to the
continued expansion of the development organization.
- G&A
Expenses: General and administrative
expenses increased by $5.2 million to $16.7 million during the
second quarter of 2021, compared to $11.5 million during the second
quarter of 2020. This increase was primarily related to employee
related expenses, including stock-based compensation, of $2.1
million.
- Net
Loss: The Company’s net loss was $68.8
million for the second quarter of 2021, compared to $32.4 million
during the second quarter of 2020.
Financial Guidance
Intellia expects that its cash, cash equivalents and marketable
securities as of June 30, 2021, along with the proceeds from the
July 2021 public offering of common stock, will enable the Company
to fund its robust R&D plans, anticipated operating expenses
and capital expenditure requirements beyond the next 24 months.
This expectation excludes any strategic use of capital not
currently in the Company’s base-case planning assumptions.
Conference Call to Discuss Second Quarter
Earnings
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 www.intelliatx.com, beginning on August 5,
2021 at 12 p.m. ET.
About Intellia TherapeuticsIntellia
Therapeutics, a leading clinical-stage genome editing company, is
developing novel, potentially curative therapeutics using
CRISPR/Cas9 technology. To fully realize the transformative
potential of CRISPR/Cas9, 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 CRISPR/Cas9 to create new classes of genetic medicine. Learn
more at intelliatx.com. Follow us on
Twitter @intelliatweets.
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
regarding its: ability to complete clinical studies for NTLA-2001
for the treatment of transthyretin (“ATTR”) amyloidosis pursuant to
its clinical trial applications (“CTA”), including submitting
additional regulatory applications in other countries; clinical
data from the ongoing single-ascending dose portion of the Phase 1
study evaluating NTLA-2001; expectations of approvals of its CTAs
to regulatory authorities in New Zealand for NTLA-2002 for the
treatment of hereditary angioedema (“HAE”) and the United Kingdom
for NTLA-5001 for the treatment of acute myeloid leukemia (“AML”);
identification and nomination of new development candidates;
expectations for the new universal CAR-T cell company launched in
collaboration with Blackstone Life Sciences Advisors L.L.C. and
Cellex Cell Professionals GmbH; advancement and expansion of its
CRISPR/Cas9 technology to develop human therapeutic products, as
well as its ability to maintain and expand its related intellectual
property portfolio; statements regarding the timing of regulatory
filings regarding its development programs; plans to attend and
present data at scientific conferences later this year; and our
expectations regarding our use of capital and our ability to fund
operations beyond the next 24 months.
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 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; the risk
that any one or more of Intellia’s product candidates will not be
successfully developed and commercialized; the risk that the
results of preclinical studies or clinical studies will not be
predictive of future results in connection with future studies;
risks related to the COVID-19 pandemic including the effects of the
Delta variant; and the risk that Intellia’s collaborations with
Regeneron or its other collaborations will not continue or will not
be 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 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) |
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Three Months Ended June 30, |
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Six Months Ended June 30, |
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2021 |
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2020 |
|
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2021 |
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|
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2020 |
|
|
Collaboration revenue |
|
$ |
6,550 |
|
|
$ |
16,263 |
|
|
$ |
12,995 |
|
|
$ |
29,179 |
|
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Operating expenses: |
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|
|
|
|
|
|
|
|
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Research and development |
|
58,884 |
|
|
|
37,771 |
|
|
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98,160 |
|
|
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72,421 |
|
|
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General and administrative |
|
16,683 |
|
|
|
11,526 |
|
|
|
30,277 |
|
|
|
22,840 |
|
|
|
|
Total operating expenses |
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75,567 |
|
|
|
49,297 |
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|
|
128,437 |
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|
|
95,261 |
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Operating loss |
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|
|
(69,017 |
) |
|
|
(33,034 |
) |
|
|
(115,442 |
) |
|
|
(66,082 |
) |
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Interest income |
|
|
|
211 |
|
|
|
641 |
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|
|
431 |
|
|
|
1,883 |
|
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Net loss |
|
|
|
$ |
(68,806 |
) |
|
$ |
(32,393 |
) |
|
$ |
(115,011 |
) |
|
$ |
(64,199 |
) |
|
Net loss per share, basic and diluted |
$ |
(1.01 |
) |
|
$ |
(0.61 |
) |
|
$ |
(1.70 |
) |
|
$ |
(1.24 |
) |
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Weighted average shares outstanding, basic and diluted |
|
68,164 |
|
|
|
53,369 |
|
|
|
67,675 |
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|
51,938 |
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INTELLIA
THERAPEUTICS, INC. |
CONSOLIDATED
BALANCE SHEET DATA (UNAUDITED) |
(Amounts in
thousands) |
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June 30, 2021 |
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December 31, 2020 |
Cash, cash equivalents and marketable securities |
|
|
|
|
|
$ |
551,281 |
|
$ |
597,371 |
Total assets |
|
|
|
|
|
672,243 |
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676,322 |
Total liabilities |
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|
|
|
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177,430 |
|
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149,250 |
Total stockholders' equity |
|
|
|
494,813 |
|
|
527,072 |
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Intellia Contacts:
Investors:Ian KarpSenior Vice President,
Investor Relations and Corporate
Communications+1-857-449-4175ian.karp@intelliatx.com
Lina LiDirector, Investor
Relations+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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