Intellia Therapeutics Announces Third Quarter 2021 Financial Results and Highlights Recent Company Progress
04 November 2021 - 10:30PM
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 third quarter ended
September 30, 2021, and recent operational highlights.
“Intellia continues to make important progress toward advancing
its full-spectrum genome editing pipeline. Today, we are pleased to
share that we remain highly encouraged by the safety profile,
consistency of TTR reduction and ongoing effect of NTLA-2001 in the
Phase 1 study. We believe we are closing in on identifying the
recommended therapeutic dose for NTLA-2001 in patients with ATTR
amyloidosis with polyneuropathy for further evaluation in Part 2, a
single-dose expansion cohort. Additionally, while it has long been
our plan to develop NTLA-2001 for all forms of ATTR amyloidosis, we
have decided to accelerate the evaluation of NTLA-2001 in patients
with ATTR amyloidosis whose primary clinical manifestation is
cardiomyopathy. Based on the strength of our interim dataset, we
are now seeking regulatory feedback for inclusion of the ATTR-CM
patient population in our current Phase 1 study. We look forward to
sharing updates from this program in Q1 2022,” said Intellia
President and Chief Executive Officer John Leonard, M.D. “Beyond
our lead program, we remain focused on advancing NTLA-2002 for HAE
and NTLA-5001 for AML. We received regulatory clearance for both
programs to initiate first-in-human studies and we expect those
studies to begin later this year. Finally, we nominated two gene
insertion development candidates – NTLA-3001, our wholly owned AATD
program, and a Factor 9 gene insertion candidate for Hem B in
collaboration with Regeneron. Our rapidly expanding pipeline is yet
one more example of the benefits of our modular platform in
generating novel and potentially curative treatment options for the
patients we aim to serve.”
Third Quarter 2021 and Recent Operational
Highlights
In Vivo Program Updates
- NTLA-2001 for ATTR amyloidosis: NTLA-2001 is
the first systemically delivered CRISPR-based therapy to be dosed
in a patient and has the potential to be a curative treatment for
transthyretin (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 for this program, and Regeneron
Pharmaceuticals, Inc. (Regeneron).
- NTLA-2001 is completing the dose-escalation portion of the
Phase 1 study to determine the recommended dose for evaluation in
Part 2 of the study, a single-dose expansion cohort. During the
third quarter, to more fully elucidate the dose-response
relationship, Intellia began dosing of subjects in Cohort 4,
evaluating NTLA-2001 in patients with hereditary transthyretin
amyloidosis with polyneuropathy (ATTRv-PN) at the 0.7 mg/kg dose
level.
- Intellia accelerated the evaluation of NTLA-2001 for the
treatment of patients with ATTR amyloidosis with cardiomyopathy
(ATTR-CM). The Company is in discussions with regulatory
authorities on a protocol amendment to expand the Phase 1 trial
population to include patients with ATTR-CM.
- Intellia now plans to present interim data from Part 1, the
single-ascending dose portion, and to initiate Part 2, a
single-cohort expansion, in the first quarter of 2022. Data to be
presented at a company-sponsored event will be from all four
cohorts in Part 1 and include safety and serum TTR knockdown for
Cohorts 3 and 4, as well as an early look at durability across all
cohorts.
- In October, Intellia announced that NTLA-2001 received Orphan
Drug Designation (ODD) from the U.S. Food and Drug Administration
(FDA) for the treatment of ATTR amyloidosis.
- NTLA-2002 for HAE: 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 hereditary angioedema (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 October, Intellia announced the authorization of its
Clinical Trial Application (CTA) by the New Zealand Medicines and
Medical Devices Safety Authority (MEDSAFE) to initiate a Phase 1/2
study evaluating NTLA-2002. Additionally, a CTA has been
subsequently authorized by the United Kingdom’s Medicines and
Healthcare products Regulatory Agency (MHRA) for the first-in-human
study of NTLA-2002.
- Intellia intends to enroll the first patient in the
first-in-human study by year-end. The Phase 1/2 study will evaluate
safety, tolerability, pharmacokinetics and pharmacodynamics of
NTLA-2002 in adults with Type I or Type II HAE, and will leverage
insights gained from the development of NTLA-2001 enabling this
study to begin evaluating NTLA-2002 at a higher initial dose.
- NTLA-3001 for AATD-associated lung disease:
NTLA-3001 is Intellia’s first and wholly owned CRISPR/Cas9-mediated
in vivo targeted gene insertion development candidate. It is
designed with the aim to precisely insert a healthy copy of the
SERPINA1 gene, which encodes the alpha-1 antitrypsin (A1AT)
protein, with the potential to permanently restore expression of
functional A1AT protein to therapeutic levels after a single dose.
This approach seeks to address alpha-1 antitrypsin deficiency
(AATD)-associated lung disease and eliminate the need for
sub-optimal weekly IV infusions of A1AT augmentation therapy or
transplant in severe cases.
- Today, Intellia announced the nomination of a new development
candidate, NTLA-3001 for treatment of AATD. At the 29th Annual
Congress of the European Society of Gene & Cell Therapy
(ESGCT), Intellia presented data showing that insertion of a
healthy form of the SERPINA1 gene led to normal human A1AT levels
in non-human primates (NHPs) which were durable through 52 weeks in
an ongoing study. The Company is advancing towards Investigational
New Drug (IND)-enabling activities for this program and continues
to explore additional editing strategies for AATD.
Ex Vivo Program Updates
- 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 acute myeloid leukemia (AML).
- In September, Intellia announced that the U.S. FDA accepted its
IND application for NTLA-5001.
- Intellia intends to initiate patient screening by year-end for
a Phase 1/2a study evaluating NTLA-5001 in adults with persistent
or recurrent AML who have previously received first-line
therapy.
Research and Corporate Updates
- 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
support new therapeutic candidates and strategic business
development partnerships for advancing treatments for genetic
diseases, cancers and autoimmune diseases.
- ESGCT Annual Congress: In October, Intellia
presented new preclinical data demonstrating key platform
capabilities.
- Intellia shared the first data highlighting its proprietary
allogeneic cell engineering platform, demonstrating it can prevent
immune rejection of allogeneic T cells for application in TCR-T and
CAR-T cell therapy. Intellia’s proprietary approach leverages a
novel combination of sequential gene edits to shield the engineered
cell therapy from host T and NK cell attack. It does not rely on
long-term, aggressive immune suppression of patients or the
knockout of a protein required for HLA class I expression, which
are approaches currently employed by others to address the
challenge of immune rejection of the allogeneic cell product.
Intellia intends to nominate its first allogeneic cell therapy
development candidate by the first half of 2022.
- Intellia shared new data on its proprietary cell engineering
process, demonstrating LNP-based delivery of CRISPR/Cas9 ex vivo
allows for sequential editing of T cells with high efficiency,
faster expansion and minimal translocations as compared to
electroporation. The data support the ability of this platform to
be used for a variety of targeting modalities, including CAR and
TCRs, and to support both autologous and allogeneic T cell
candidates. This LNP-based approach is being used for
NTLA-5001.
- Collaboration Updates:
- In the third quarter, Intellia and Regeneron nominated a Factor
9 (F9) gene insertion development candidate for its Hemophilia B
(Hem B) program, leveraging their jointly developed targeted
transgene insertion capabilities to insert F9. This candidate is
part of a co-development/co-funding agreement between Intellia and
Regeneron, the lead party for this program. F9 is a gene that
encodes for Factor IX (FIX), a blood-clotting protein that is
missing or defective in Hem B patients. In preclinical studies, the
companies demonstrated the first CRISPR/Cas9-mediated targeted
transgene insertion in the liver of NHPs, which resulted in
circulating FIX levels at or above those found in normal human
plasma.
- In October, the Company announced a strategic collaboration
with SparingVision to develop novel genomic medicines utilizing
Intellia’s proprietary CRISPR/Cas9 technology for the treatment of
ocular diseases. As part of the collaboration, Intellia will
receive an equity stake in SparingVision, option for exclusive U.S.
commercialization rights for product candidates arising from two of
three collaboration targets, and eligibility for development and
commercial milestone payments as well as royalties on potential
future sales of products arising from the collaboration. The
companies will additionally research and develop novel
self-inactivating AAV vectors and LNP-based approaches to address
delivery of CRISPR/Cas9 genome reagents to the retina.
- In July, the Company completed the formation of a new universal
CAR-T cell therapy company, which Intellia launched in
collaboration with Blackstone Life Sciences and Cellex Cell
Professionals GmbH. 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.
Upcoming Events
The Company will participate in the following events during the
fourth quarter of 2021:
- Barclays Gene Editing & Gene
Therapy Summit, November 15, Virtual
- Evercore ISI 4th Annual HealthCONx
Conference, December 1, Virtual
- 63rd ASH Annual Meeting &
Exposition, December 11-14, Atlanta, GA and Virtual
Upcoming Milestones
The Company has set forth the following for pipeline
progression:
- ATTR:
- Report additional interim clinical
data from Phase 1 study of NTLA-2001 in Q1 2022
- Initiate Part 2, a single-dose
expansion cohort, of Phase 1 study of NTLA-2001 in Q1 2022
- HAE: Initiate enrollment in the
first-in-human study of NTLA-2002 by year-end
- AML: Initiate patient screening in
the first-in-human study of NTLA-5001 by year-end
- Pipeline Expansion: Nominate the
Company’s first allogeneic development candidate by 1H 2022
Third Quarter 2021 Financial Results
- Cash
Position: Cash, cash equivalents and
marketable securities were $1,148.7 million as of September 30,
2021, compared to $597.4 million as of December 31, 2020. The
increase was driven by net proceeds of $648.3 million from a
follow-on offering in Q3 2021, $45.3 million of net proceeds from
the Company’s “At the Market” (ATM) agreement, $40.8 million in
proceeds from employee-based stock plans, and $4.2 million of
funding for a cost-sharing agreement received from Regeneron. These
increases were offset in part by cash used to fund operations of
approximately $187.3 million.
- Collaboration
Revenue: Collaboration revenue decreased
by $15.0 million to $7.2 million during the third quarter of 2021,
compared to $22.2 million during the third quarter of 2020. The
decrease in collaboration revenue in 2021 was primarily driven by
$15.3 million related to the transfer of control of the license to
develop the Factor VIII target for hemophilia A that was recorded
during Q3 2020.
- R&D
Expenses: Research and development
expenses increased by $20.7 million to $60.5 million during the
third quarter of 2021, compared to $39.8 million during the third
quarter of 2020. 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 $8.1 million to $18.7 million during the
third quarter of 2021, compared to $10.6 million during the third
quarter of 2020. This increase was primarily related to employee
related expenses, including stock-based compensation of $3.8
million.
- Net
Loss: The Company’s net loss was $71.6
million for the third quarter of 2021, compared to $27.8 million
during the third quarter of 2020.
Conference Call to Discuss Third Quarter
Earnings
The Company will discuss these results on a conference call
today, Thursday, November 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 www.intelliatx.com, beginning on November 4
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 on regulatory approval to
expand the Phase 1 trial population to include patients with ATTR
with cardiomyopathy (“ATTR-CM”); expectations of being able to
initiate clinical trials for NTLA-2002 for the treatment of
hereditary angioedema (“HAE”) and NTLA-5001 for the treatment of
acute myeloid leukemia (“AML”); ability to enroll patients in its
Phase 1/2 study for NTLA-2002 for the treatment of hereditary
angioedema (“HAE”) in New Zealand and the United Kingdom; its
IND-enabling activities for NTLA-3001 for the treatment of alpha-1
antitrypsin deficiency; nomination and continued preclinical
development of new development candidates; expectations for a new
collaboration with Regeneron for a Factor 9 (“F9”) development
candidate for its Hemophilia B program; expectations for a new
collaboration with SparingVision for the treatment of ocular
diseases; 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; the
risk that regulatory approval to expand the Phase 1 patient
population to include patients with ATTR-CM will be denied; risks
related to the COVID-19 pandemic including the effects of the Delta
variant; and the risk that Intellia’s collaborations with
Regeneron, SparingVision 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 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. |
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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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2021 |
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2020 |
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2021 |
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2020 |
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Collaboration revenue |
|
$ |
7,204 |
|
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$ |
22,220 |
|
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$ |
20,199 |
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$ |
51,399 |
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Operating expenses: |
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Research and development |
|
60,486 |
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39,756 |
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158,646 |
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112,177 |
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General and administrative |
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18,711 |
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|
10,566 |
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48,988 |
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33,406 |
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Total operating expenses |
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79,197 |
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50,322 |
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207,634 |
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145,583 |
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Operating loss |
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(71,993 |
) |
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(28,102 |
) |
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(187,435 |
) |
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(94,184 |
) |
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Interest income |
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349 |
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|
262 |
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780 |
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2,145 |
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Net loss |
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$ |
(71,644 |
) |
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$ |
(27,840 |
) |
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$ |
(186,655 |
) |
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$ |
(92,039 |
) |
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Net loss per share, basic and diluted |
$ |
(0.97 |
) |
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$ |
(0.47 |
) |
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$ |
(2.68 |
) |
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$ |
(1.70 |
) |
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Weighted average shares outstanding, basic and diluted |
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73,706 |
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58,754 |
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69,720 |
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54,218 |
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INTELLIA THERAPEUTICS, INC. |
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CONSOLIDATED BALANCE SHEET DATA (UNAUDITED) |
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(Amounts in thousands) |
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September 30,2021 |
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December 31,2020 |
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Cash, cash equivalents and marketable securities |
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$ |
1,148,718 |
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$ |
597,371 |
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Total assets |
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1,334,961 |
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676,322 |
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Total liabilities |
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227,863 |
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149,250 |
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Total stockholders' equity |
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1,107,098 |
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527,072 |
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Intellia Contacts:
Investors:Ian KarpSenior Vice President,
Investor Relations and Corporate
Communications+1-857-449-4175ian.karp@intelliatx.comLina
LiDirector, Investor
Relations+1-857-706-1612lina.li@intelliatx.com
Media:Matt Crenson Ten Bridge
Communications+1-917-640-7930media@intelliatx.com
mcrenson@tenbridgecommunications.com
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