Intellia Therapeutics Announces Third Quarter 2020 Financial Results
05 November 2020 - 11:30PM
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing
company focused on developing curative therapeutics using
CRISPR/Cas9 technology both in vivo and ex vivo, today reported
operational highlights and financial results for the third quarter
ended September 30, 2020.
“We are very pleased with the recent regulatory authorization to
begin our Phase 1 study of NTLA-2001, which keeps us on track to
dose our first patient by year-end. This is an important step
toward improving the lives of ATTR patients with a potentially
curative treatment, and marks our transition into a clinical-stage
company. Further, advancing NTLA-2001 is a major milestone for the
field of genome editing, as this is the first clinical trial of a
systemically delivered CRISPR/Cas9-based therapy,” said Intellia
President and Chief Executive Officer John Leonard, M.D. “In
parallel, we are progressing NTLA-5001 and NTLA-2002 for the
treatment of AML and HAE, respectively, each to an IND or
equivalent regulatory submission next year. We also continue to
develop innovative capabilities across our platform, based on the
Nobel Prize-winning CRISPR/Cas9 technology, to generate our next
wave of therapeutic candidates.”
Third Quarter 2020 and Recent
Operational Highlights
- ATTR Program:
Intellia recently announced the authorization of its Clinical Trial
Application (CTA) by the United Kingdom’s Medicines and Healthcare
products Regulatory Agency (MHRA) to initiate a first-in-human
clinical trial of NTLA-2001, an investigational therapy in
development for the treatment of all clinical manifestations of
ATTR. By applying the Company’s in vivo liver gene knockout
technology, NTLA-2001 allows for the possibility of lifelong
transthyretin (TTR) protein reduction after a single course of
treatment. Intellia’s first-in-human study will evaluate NTLA-2001
in adults with hereditary ATTR with polyneuropathy (hATTR-PN). The
Phase 1 study will be a two-part, open label, multi-center study to
assess the safety, tolerability, pharmacokinetics and
pharmacodynamics of NTLA-2001, which will include the measurement
of serum TTR levels following a single intravenous infusion.
Intellia is on track to dose its first patient by the end of 2020,
subject to the impact of the COVID-19 pandemic, and is submitting
additional regulatory applications in other countries as part of
its ongoing, global development strategy. Once safety and an
optimal dose have been determined in the first-in-human study,
Intellia intends to further evaluate NTLA-2001 in a broader ATTR
patient population of both polyneuropathy and cardiomyopathy
patients. NTLA-2001 is part of a co-development/co-promotion
agreement between Intellia, the lead development and
commercialization party, and Regeneron Pharmaceuticals, Inc.
(Regeneron).
- AML Program:
NTLA-5001 is a wholly owned, T cell receptor (TCR)-T cell therapy
development candidate targeting the Wilms’ Tumor 1 (WT1) antigen
for the treatment of AML. The Company seeks to develop NTLA-5001 as
a broadly applicable treatment for AML patients, regardless of the
mutational subtypes of the cancer. Intellia continues to advance
Investigational New Drug application (IND)-enabling activities and
remains on track to submit an IND or IND-equivalent for NTLA-5001
in the first half of 2021. At the upcoming 62nd American Society of
Hematology (ASH) Annual Meeting, taking place virtually from
December 5 – 8, 2020, the Company will present new preclinical
results in support of NTLA-5001, showing high anti-tumor activity
of its lead WT1-directed TCR-T therapy in a mouse model of AML. The
preclinical data will also highlight the advantages of its
proprietary T cell engineering process to produce multiple, highly
efficient sequential edits in T cells that have superior function
and minimal translocations compared to results from standard T cell
engineering approaches. Additional efforts are underway to evaluate
the potential use of NTLA-5001 to treat WT1-positive solid
tumors.
- HAE Program:
NTLA-2002 is a wholly owned, in vivo development candidate for the
treatment of HAE. Today, Intellia announced results from its
completed non-human primate (NHP) study of its lead lipid
nanoparticle (LNP) formulation for NTLA-2002. Following a single
dose, the knockout of the prekallikrein B1 (KLKB1) gene resulted in
a year-long therapeutically relevant reduction of serum kallikrein
protein levels and activity. Building on Intellia’s modular LNP
delivery system, NTLA-2002 is designed to knock out the KLKB1 gene
in the liver after a single course of treatment. This approach is
expected to prevent improperly regulated bradykinin production and
therefore reduce HAE attacks. During the third quarter, the Company
initiated Good Laboratory Practices (GLP) toxicology studies in
preparation for an IND or IND-equivalent submission for NTLA-2002,
which remains on track for the second half of 2021.
- Modular Platform:
Intellia continues to make significant progress across its platform
technologies, broadening the in vivo and ex vivo application of
genome editing. This includes developing innovative
CRISPR/Cas9-mediated targeted transgene insertion and allogeneic
cell solutions. At the 16th Annual Meeting of the Oligonucleotide
Therapeutics Society, held September 27-30, 2020, Intellia
presented new data highlighting the potential to develop
single-course therapies that may have a lifelong effect for a
variety of genetic diseases. The data showed the persistence of
both in vivo knockout and insertion CRISPR/Cas9 edits and
corresponding durability of effect following a partial hepatectomy
(PHx) and liver regrowth in a murine model. Unlike traditional gene
therapy, for which a significant loss (over 80%) in transgene
expression was observed in the insertion PHx model, Intellia’s
targeted gene insertion approach yielded durable edits, with no
significant loss in expression in the same model. Intellia and
Regeneron are co-developing potential hemophilia A and B
CRISPR/Cas9-based treatments using their jointly developed
insertion capabilities. Intellia is also continuing to develop its
proprietary platform to advance its wholly-owned programs.
- Board of
Directors: In October 2020, Intellia
appointed John F. Crowley to its Board of Directors. Mr. Crowley,
Chairman and Chief Executive Officer of Amicus Therapeutics, is a
well-established leader in biotech and pharmaceuticals and a
visionary advocate for the advancement of treatments for people
living with rare diseases.
- Scientific
Co-Founder Awarded Nobel
Prize: Jennifer Doudna, Ph.D.,
one of Intellia’s scientific co-founders, was awarded the 2020
Nobel Prize in Chemistry for inventing the revolutionary
CRISPR/Cas9 genome editing technology. Dr. Doudna shares the award
with her research collaborator, Dr. Emmanuelle Charpentier.
Upcoming Events
The Company will participate in the following events during the
fourth quarter of 2020:
- Credit Suisse Healthcare
Conference, November 11, Virtual
- Barclays Gene Editing and Therapy
Summit, November 16, Virtual
- 62nd ASH Annual Meeting, December
5-8, Virtual
Upcoming Milestones
The Company has set forth the following for pipeline
progression:
- ATTR: Dose first patient in Phase 1
study by year-end
- AML: Submit an IND or
IND-equivalent for NTLA-5001 in 1H 2021
- HAE: Submit an IND or
IND-equivalent for NTLA-2002 in 2H 2021
Third Quarter 2020
Financial Results
- Cash
Position: Cash, cash equivalents and
marketable securities were $407.9 million as of September 30, 2020,
compared to $284.5 million as of December 31, 2019. The increase
was driven by net proceeds of $107.7 million from the June
follow-on public offering, $100.0 million upfront payment from the
Regeneron collaboration expansion, which included a $30.0 million
equity investment, $14.7 million of net equity proceeds raised from
the Company’s “At the Market” (ATM) agreement, $18.2 million of
funding received under the Regeneron and Novartis collaborations
and $2.7 million in proceeds from employee-based stock plans. These
increases were offset in part by cash used to fund operations of
approximately $119.8 million.
- Collaboration
Revenue: Collaboration revenue increased
by $11.6 million to $22.2 million during the third quarter of 2020,
compared to $10.6 million during the third quarter of 2019. The
increase was mainly driven by a $15.3 million amount recognized for
the transfer of control of the license to develop the Factor VIII
target for hemophilia A associated with the extension of the
Regeneron collaboration.
- R&D
Expenses: Research and development
expenses increased by approximately $12.2 million to $39.8 million
during the third quarter of 2020, compared to $27.5 million during
the third quarter of 2019. This increase was primarily driven by
the advancement of our lead programs, research personnel growth to
support these programs, and the expansion of the development
organization.
- G&A
Expenses: General and administrative
expenses increased by approximately $2.1 million to $10.6 million
during the third quarter of 2020, compared to $8.4 million during
the third quarter of 2019. This increase was primarily related to
employee related expenses, including stock-based compensation, of
$2.0 million.
- Net
Loss: The Company’s net loss was $27.8
million for the third quarter of 2020, compared to $23.6 million
during the third quarter of 2019.
Financial Guidance
Intellia expects that its cash, cash equivalents and marketable
securities as of September 30, 2020 will enable the Company to fund
its anticipated operating expenses and capital expenditure
requirements for at least 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
Third Quarter
2020 Earnings
The Company will discuss these results on a conference call
today, November 5, 2020, at 8 a.m. ET.
To join the call:
- U.S. callers should dial 1-877-317-6789 and international
callers should dial 1-412-317-6789, approximately five minutes
before the call.
- All participants should ask to be connected to the Intellia
Therapeutics conference 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 November 5,
2020 at 12 p.m. ET.
About Intellia TherapeuticsIntellia
Therapeutics is a leading genome editing company, focused on the
development of proprietary, potentially curative therapeutics using
the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9
technology has the potential to transform medicine by both
producing therapeutics that permanently edit and/or correct
disease-associated genes in the human body with a single treatment
course, and creating enhanced engineered cells that can treat
oncological and immunological diseases. Intellia’s combination of
deep scientific, technical and clinical development experience,
along with its leading intellectual property portfolio, puts it in
a unique position to unlock broad therapeutic applications of the
CRISPR/Cas9 technology and create new classes of therapeutic
products. Learn more about Intellia Therapeutics and CRISPR/Cas9 at
intelliatx.com and follow us on Twitter @intelliatweets.
Forward-Looking Statements This press release
contains “forward-looking statements” of Intellia Therapeutics,
Inc. (“Intellia”, “we” or “our”) 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: being able to initiate clinical studies for NTLA-2001 for the
treatment of transthyretin amyloidosis (“ATTR”) pursuant to its
clinical trial applications (“CTA”), including dosing of a first
patient by the end of 2020, and submitting similar regulatory
applications in other countries; plans to submit an investigational
new drug (“IND”) application or similar clinical trial application
for NTLA-5001, its first T cell receptor (“TCR”)-directed
engineered cell therapy development candidate for its acute myeloid
leukemia (“AML”) program in the first half of 2021; plans to submit
an IND or similar clinical trial application for its hereditary
angioedema (“HAE”) program in the second half of 2021; plans to
advance and complete preclinical studies, including non-human
primate studies for its HAE and other programs, and other animal
studies supporting other in vivo and ex vivo programs, including
its AML program; development of a proprietary LNP/AAV hybrid
delivery system, as well as its modular platform to advance its
complex genome editing capabilities, such as gene insertion;
further development of its proprietary cell engineering process for
multiple sequential editing; presentation of additional data at
upcoming scientific conferences, and other preclinical data in
2020; 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;
ability to demonstrate its platform’s modularity and replicate or
apply results achieved in preclinical studies, including those in
its ATTR, AML, and HAE programs, in any future studies, including
human clinical trials; expectations of the potential impact of the
coronavirus disease 2019 pandemic on strategy, future operations
and timing of its clinical trials or IND submissions; ability to
develop other in vivo or ex vivo cell therapeutics of all types,
and those targeting WT1 in AML in particular, using CRISPR/Cas9
technology; ability to optimize the impact of its collaborations on
its development programs, including but not limited to its
collaborations with Novartis Institutes for BioMedical Research,
Inc. (“Novartis”) or Regeneron Pharmaceuticals, Inc. (“Regeneron”),
including its co-development programs for hemophilia A and
hemophilia B; statements regarding the timing of regulatory filings
and clinical trial execution, including dosing of patients,
regarding its development programs; the potential commercial
opportunities, including value and market, for our product
candidates; our expectations regarding our use of capital and other
financial results during 2020; and our ability to fund operations
for at least 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 regulatory agencies’ evaluation of regulatory filings
and other information related to its product candidates;
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, including those that are co-developed, 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; and
the risk that Intellia’s collaborations with Novartis or Regeneron
or its other ex vivo 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. |
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CONSOLIDATED
STATEMENTS OF OPERATIONS (UNAUDITED) |
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(Amounts in
thousands, except per share data) |
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Three Months Ended September 30, |
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Nine Months Ended September 30, |
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2020 |
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2019 |
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2020 |
|
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2019 |
|
|
|
Collaboration revenue |
|
$ |
22,220 |
|
|
$ |
10,616 |
|
|
$ |
51,399 |
|
|
$ |
32,167 |
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Operating expenses: |
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Research and development |
|
39,756 |
|
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|
27,513 |
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|
112,177 |
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|
|
76,682 |
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|
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General and administrative |
|
10,566 |
|
|
|
8,431 |
|
|
|
33,406 |
|
|
|
32,082 |
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|
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Total operating expenses |
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50,322 |
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|
|
35,944 |
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|
145,583 |
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|
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108,764 |
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Operating loss |
|
|
|
(28,102 |
) |
|
|
(25,328 |
) |
|
|
(94,184 |
) |
|
|
(76,597 |
) |
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Interest income |
|
|
|
262 |
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|
|
1,694 |
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|
|
2,145 |
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|
|
5,340 |
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Net loss |
|
|
|
$ |
(27,840 |
) |
|
$ |
(23,634 |
) |
|
$ |
(92,039 |
) |
|
$ |
(71,257 |
) |
|
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Net loss per share, basic and diluted |
$ |
(0.47 |
) |
|
$ |
(0.49 |
) |
|
$ |
(1.70 |
) |
|
$ |
(1.53 |
) |
|
|
Weighted average shares outstanding, basic and diluted |
|
58,754 |
|
|
|
48,554 |
|
|
|
54,218 |
|
|
|
46,547 |
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INTELLIA
THERAPEUTICS, INC. |
CONSOLIDATED
BALANCE SHEET DATA (UNAUDITED) |
(Amounts in
thousands) |
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September 30, 2020 |
|
December 31, 2019 |
Cash, cash
equivalents and marketable securities |
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$ |
407,947 |
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$ |
284,472 |
Total assets |
|
458,555 |
|
|
334,280 |
Total liabilities |
|
128,935 |
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|
64,399 |
Total stockholders' equity |
|
329,620 |
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|
269,881 |
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Intellia Contacts:
Investors:Lina LiAssociate DirectorInvestor
Relations+1 857-706-1612lina.li@intelliatx.com
Media:Jennifer Mound SmoterSenior Vice
PresidentExternal Affairs & Communications+1
857-706-1071jenn.smoter@intelliatx.com
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