Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage
gene editing company focused on revolutionizing medicine with
CRISPR-based therapies, today reported operational highlights and
financial results for the first quarter ended March 31, 2024.
“Intellia continues to make outstanding progress across our
pipeline of in vivo and ex vivo single-dose CRISPR-based therapies.
With one ongoing and two soon-to-be-initiated pivotal Phase 3
trials, Intellia is undoubtedly at the forefront of a new era in
medicine,” said Intellia President and Chief Executive Officer John
Leonard, M.D. “We have been extremely pleased with the speed of
enrollment in the Phase 3 MAGNITUDE trial for patients with ATTR
amyloidosis with cardiomyopathy, which is tracking ahead of our
initial projections. In addition, we now expect to start a pivotal
Phase 3 trial of NTLA-2001 for patients with polyneuropathy by
year-end, based on productive discussions with the FDA. Moving to
NTLA-2002, we expect to report key data readouts from the Phase 1/2
study this year. These data will support the dose selection for the
Phase 3 trial and highlight what we believe is the potential for
NTLA-2002 to dramatically change the HAE treatment paradigm.
Building on our success with in vivo gene inactivation, we are
excited to initiate the first-in-human study of NTLA-3001 for AATD
this year, positioning us to be the first to clinically validate
CRISPR-based gene insertion. We look forward to continuing our
strong execution, with many notable milestones to mark the progress
against our strategic priorities.”
First Quarter 2024 and Recent Operational
Highlights
Transthyretin (ATTR) Amyloidosis
- NTLA-2001:
NTLA-2001 is an investigational in vivo CRISPR-based therapy
designed to inactivate the TTR gene in the liver and thereby
prevent the production of transthyretin (TTR) protein for the
treatment of ATTR amyloidosis. NTLA-2001 offers the possibility of
halting and reversing the disease by driving a deep, consistent and
potentially lifelong reduction in TTR protein after a single dose.
Intellia leads development and commercialization of NTLA-2001 in
collaboration with Regeneron.
- ATTR Amyloidosis with
Cardiomyopathy (ATTR-CM):
- The pivotal Phase 3 MAGNITUDE trial
is rapidly enrolling. In March, the first patients in the U.S. and
globally were dosed. Enrollment is currently tracking well ahead of
the Company’s initial projections, with over 30 patients dosed to
date and more than 40 additional patients in screening. Many
additional sites are expected to open in the weeks and months ahead
to further accelerate enrollment.
- Hereditary ATTR Amyloidosis
with Polyneuropathy (ATTRv-PN):
- Intellia announced today alignment
with the U.S. Food and Drug Administration (FDA) on a pivotal Phase
3 trial design to support a biologics license application (BLA)
filing for NTLA-2001 as a single-dose treatment for people living
with ATTRv-PN, subject to review of its investigational new drug
(IND) application. The study is expected to be a small,
placebo-controlled trial conducted at ex-U.S. sites with
approximately 50 ATTRv-PN patients. The Company plans to initiate
the study by year-end.
- The Company plans to present
updated data from the ongoing Phase 1 study in the second half of
2024.
Hereditary Angioedema (HAE)
- NTLA-2002:
NTLA-2002 is a wholly owned, investigational in vivo CRISPR-based
therapy designed to knock out the KLKB1 gene in the liver, with the
goal of lifelong control of HAE attacks after a single dose.
- Intellia plans to initiate the
global pivotal Phase 3 study, including U.S. patients, in the
second half of 2024, subject to regulatory feedback.
- The Company will present updated
data from the Phase 1 study at the European Academy of Allergy and
Clinical Immunology (EAACI) Congress 2024, taking place May 31 –
June 3 in Valencia, Spain. Long-term data from the Phase 1 portion
of the Phase 1/2 study will include safety, kallikrein reduction
and attack rate data, including number of patients who continue to
be completely attack free through the latest follow-up.
Additionally, Intellia plans to report topline results from the
Phase 2 portion in mid-2024 and present full results at a medical
meeting in the second half of 2024.
- In January, landmark findings from
the Phase 1 portion of the Phase 1/2 study of NTLA-2002 were
published in the New England Journal of Medicine (NEJM).
Alpha-1 Antitrypsin Deficiency (AATD)-Associated Lung
Disease
- NTLA-3001:
NTLA-3001 is a 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
the wild-type SERPINA1 gene, which encodes the alpha-1 antitrypsin
(AAT) protein, with the potential to restore permanent expression
of fully functional AAT protein to normal levels after a single
dose. This is Intellia’s first wholly owned gene insertion program.
- Intellia expects to dose the first
patient in a Phase 1 study of NTLA-3001 in 2024.
In Vivo Platform Expansion
- Intellia is expanding the range of
diseases that can be targeted with its CRISPR-based technologies by
deploying new editing and delivery innovations. This includes
advancing gene editing programs in five different tissues outside
the liver, either independently or in collaboration with partners.
These research and preclinical programs are targeting diseases that
originate in the bone marrow, brain, muscle, lung and eye, which,
if successful, could dramatically expand the opportunities for
CRISPR-based treatments.
- In February, Intellia and ReCode
announced a strategic collaboration to develop novel genomic
medicines for the treatment of cystic fibrosis (CF). The
collaboration will leverage Intellia’s proprietary CRISPR-based
gene editing platform, including its DNA writing technology, and
ReCode’s proprietary Selective Organ Targeting (SORT) lipid
nanoparticle delivery platform to precisely correct one or more CF
disease-causing gene mutations.
Ex Vivo Program Updates
- Intellia is advancing multiple
programs, wholly owned and in collaboration with partners,
utilizing its allogeneic platform for the treatment of
immuno-oncology and autoimmune diseases. The Company’s proprietary
allogeneic cell engineering platform avoids both T cell- and NK
cell-mediated rejection in preclinical models, a key unsolved
challenge with other investigational allogeneic approaches. Cell
therapies engineered with Intellia’s allogeneic platform, combined
with edits to enhance cell function, offer a new approach to target
solid tumors.
Corporate Updates
- Corporate Responsibility
Report: In April, Intellia published its 2024 Corporate
Responsibility Report. The report highlights the Company’s
Environmental, Social and Governance (ESG) principles and practices
as part of its objective to build a sustainable company, while
delivering on its commitments to patients, employees and
shareholders.
Upcoming Events
The Company will participate in the following events during the
second quarter of 2024:
- Bank of America Health Care
Conference, May 14, Las Vegas
- RBC Capital Markets Global
Healthcare Conference, May 14, New York
- EAACI Congress 2024, May 31 – June
3, Valencia, Spain
- Goldman Sachs 45th Annual Global
Healthcare Conference, June 10, Miami
First Quarter 2024 Financial Results
- Cash
Position: Cash, cash equivalents and
marketable securities were $953.4 million as of March 31, 2024,
compared to $1.0 billion as of December 31, 2023. The decrease was
driven by cash used to fund operations of $137.2 million. The
decrease was offset in part by $58.0 million of net equity proceeds
from the Company’s “At the Market” (ATM) program, $12.6 million of
interest income, $5.9 million of reimbursement from its
collaborators, and $2.0 million in proceeds from employee-based
stock plans. The cash position is expected to fund operations into
late 2026.
- Collaboration
Revenue: Collaboration revenue was $28.9
million during the first quarter of 2024, compared to $12.6 million
during the first quarter of 2023. The $16.3 million increase was
mainly driven by a $21.0 million non-cash revenue recognition
adjustment related to the AvenCell collaboration.
- R&D
Expenses: Research and development
expenses were $111.8 million during the first quarter of 2024,
compared to $97.1 million during the first quarter of 2023. The
$14.7 million increase was primarily driven by the advancement of
our lead programs. Stock-based compensation expense included in
research and development expenses was $20.2 million for the first
quarter of 2024.
- G&A
Expenses: General and administrative
expenses were $31.1 million during the first quarter of 2024,
compared to $27.4 million during the first quarter of 2023. The
$3.7 million increase was primarily related to stock-based
compensation. Stock-based compensation expense included in general
and administrative expenses was $14.0 million for the first quarter
of 2024.
- Net
Loss: Net loss was $107.4 million for the
first quarter of 2024, compared to $103.1 million during the first
quarter of 2023.
Conference Call to Discuss First Quarter 2024
Results
The Company will discuss these results on a conference call
today, Thursday, May 9, 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 May 9 at 12 p.m.
ET.
About Intellia TherapeuticsIntellia
Therapeutics, Inc. (NASDAQ:NTLA) is a leading clinical-stage gene
editing company focused on revolutionizing medicine with
CRISPR-based therapies. The company’s in vivo programs
use CRISPR to enable precise editing of disease-causing genes
directly inside the human body. Intellia’s ex
vivo programs use CRISPR to engineer human cells outside the
body for the treatment of cancer and autoimmune diseases.
Intellia’s deep scientific, technical and clinical development
experience, along with its people, is helping set the standard for
a new class of medicine. To harness the full potential of gene
editing, Intellia continues to expand the capabilities of its
CRISPR-based platform with novel editing and delivery technologies.
Learn more at intelliatx.com and follow us @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: the safety, efficacy, success and advancement of its
clinical programs for NTLA-2001 for transthyretin (“ATTR”)
amyloidosis, NTLA-2002 for the treatment of hereditary angioedema
(“HAE”), and NTLA-3001 for the treatment of alpha-1 antitrypsin
deficiency (“AATD”)-associated lung disease, pursuant to its
clinical trial applications (“CTA”) and investigational new drug
(“IND”) submissions, including the expected timing of data
releases, regulatory feedback, regulatory filings, and the
initiation, enrollment, dosing and completion of clinical trials,
such as the presentation of additional data from the NTLA-2001 and
NTLA-2002 clinical trials in 2024, its ability to rapidly enroll
the Phase 3 MAGNITUDE study, the planned initiation of its Phase 3
trial of NTLA-2001 for the treatment of hereditary ATTR amyloidosis
with polyneuropathy by year-end, the planned initiation of a global
pivotal Phase 3 study of NTLA-2002 in 2H 2024, its expectations
that data will inform the dose selection for the Phase 3 trial of
NTLA-2002, its ability to dose the first patient in its NTLA-3001
Phase 1 study in 2024, the potential of NTLA-2001 to halt and
reverse disease by driving a deep, consistent and potentially
lifelong reduction in TTR protein after a single dose, the
potential of NTLA-2002 to lead to lifelong control of HAE attacks
after a single dose, and the potential of NTLA-3001 to restore
permanent expression of functional alpha-1 antitrypsin protein to
therapeutic levels after a single dose; the expansion of its
CRISPR/Cas9 technology and related novel technologies, including
DNA writing and related research milestones and delivery to other
tissues outside of the liver, such as bone marrow, brain, muscle,
lung and eye; its ability to advance multiple ex vivo programs
utilizing an allogeneic platform, which is designed to avoid both T
cell- and NK cell-mediated rejection, for the treatment of
immuno-oncology and autoimmune diseases; its ability to advance
additional in vivo and ex vivo 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. (“Regeneron”)
and their co-development programs for ATTR amyloidosis, as well as
their expanded research collaboration to develop additional in vivo
CRISPR-based gene editing therapies focused on neurological and
muscular diseases and its collaboration with ReCode Therapeutics,
Inc. (“ReCode”) to develop novel genomic medicines for the
treatment of cystic fibrosis utilizing CRISPR-based gene editing,
including DNA writing, and Selective Organ Targeting lipid
nanoparticle delivery technologies; and its 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, collaborators, 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 delay
of planned clinical trials due to regulatory feedback or other
developments; and risks related to Intellia’s collaborations with
Regeneron, ReCode, 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 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, including its quarterly report on Form
10-Q. 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 March 31, |
|
|
|
|
|
|
2024 |
|
|
|
2023 |
|
Collaboration revenue |
|
$ |
28,935 |
|
|
$ |
12,606 |
|
Operating expenses: |
|
|
|
|
Research and development |
|
111,847 |
|
|
|
97,116 |
|
General and administrative |
|
31,091 |
|
|
|
27,448 |
|
Total operating expenses |
|
142,938 |
|
|
|
124,564 |
|
Operating loss |
|
|
|
(114,003 |
) |
|
|
(111,958 |
) |
Other income (expense), net: |
|
|
|
Interest income |
|
|
12,632 |
|
|
|
11,980 |
|
Change in fair value of investments, net |
|
(6,065 |
) |
|
|
- |
|
Loss from equity method investment |
|
- |
|
|
|
(3,048 |
) |
Change in fair value of contingent consideration |
|
- |
|
|
|
(100 |
) |
Total other income (expense), net |
|
6,567 |
|
|
|
8,832 |
|
Net loss |
|
|
|
$ |
(107,436 |
) |
|
$ |
(103,126 |
) |
Net loss per share, basic and diluted |
$ |
(1.12 |
) |
|
$ |
(1.17 |
) |
Weighted average shares outstanding, basic and diluted |
|
95,502 |
|
|
|
87,772 |
|
INTELLIA THERAPEUTICS, INC. |
|
CONSOLIDATED BALANCE SHEET DATA (UNAUDITED) |
|
(Amounts in thousands) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
March 31, 2024 |
|
December 31, 2023 |
|
Cash, cash equivalents and marketable securities |
|
|
|
|
$ |
953,384 |
|
$ |
1,012,087 |
|
Total assets |
|
|
|
|
1,259,589 |
|
|
1,300,977 |
|
Total liabilities |
|
|
|
|
223,452 |
|
|
250,808 |
|
Total stockholders' equity |
|
|
1,036,137 |
|
|
1,050,169 |
|
|
|
|
|
|
|
|
|
|
Intellia Contacts:
Investors:Ian KarpSenior Vice President,
Investor Relations and Corporate
Communicationsian.karp@intelliatx.com
Lina LiSenior Director, Investor Relations and Corporate
Communicationslina.li@intelliatx.com
Media:Matt CrensonTen Bridge
Communicationsmedia@intelliatx.com
mcrenson@tenbridgecommunications.com
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