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 second quarter ended June 30, 2024.
“We are delighted to report the Phase 2 study of NTLA-2002 met
its primary efficacy and all secondary endpoints at both dose
levels and, importantly, provided clear support for advancing the
50 mg dose into the pivotal Phase 3 trial in patients with
hereditary angioedema,” said Intellia President and Chief Executive
Officer John Leonard, M.D. “Based on these positive results and our
recent successful end-of-Phase 2 meeting with the FDA, we see a
clear path to initiating the Phase 3 trial in the coming months. We
look forward to presenting the detailed Phase 2 results at a
medical meeting in the fourth quarter as we continue to advance
what we believe could be a functional cure for hereditary
angioedema. With three pivotal Phase 3 trials and our first gene
insertion trial expected to be active by year-end, Intellia is
closer than ever to transforming the future of medicine with our
one-time, in vivo gene editing therapies.”
Second Quarter 2024 and Recent Operational
Highlights
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 announced today positive
topline results from the Phase 2, randomized, double-blind,
placebo-controlled study of NTLA-2002 in patients with HAE. The
clinical trial met its primary efficacy and all secondary endpoints
in the 16-week primary observation period, with a single 25 mg or
50 mg dose leading to deep reductions in attacks. No new safety
findings were observed. Intellia has selected the 50 mg dose for
further evaluation in the global pivotal Phase 3 trial based upon
the greater number of patients with complete attack elimination and
greater kallikrein protein reduction compared to the 25 mg dose
observed in the Phase 2 study, which is consistent with the
previously reported Phase 1 results. The Company plans to present
the detailed Phase 2 data at an upcoming medical meeting in the
fourth quarter of this year.
- The Company announced today the
successful completion of an end-of-Phase 2 meeting with the U.S.
Food and Drug Administration (FDA) supporting its Phase 3 plans for
NTLA-2002. The Phase 3 study is on track to initiate in the second
half of 2024, subject to regulatory feedback.
- In June, Intellia presented positive long-term data from the
ongoing Phase 1 study. Eight of 10 patients remained completely
attack-free following the 16-week primary observation period. These
patients have experienced ongoing attack-free durations of greater
than 18 months after a single-dose treatment, with the longest
ongoing individual attack-free duration reaching over 26 months.
Across all patients, NTLA-2002 led to a 98% mean reduction in
monthly HAE attack rate. Consistent with previously reported
results, NTLA-2002 was well-tolerated, with the majority of adverse
events being mild in severity through the latest follow-up. These
interim data were presented at the European Academy of Allergy and
Clinical Immunology (EAACI) Congress 2024 in Valencia, Spain.
Transthyretin (ATTR) Amyloidosis
- NTLA-2001:
NTLA-2001, now known as nexiguran ziclumeran (nex-z), is an
investigational in vivo CRISPR-based therapy designed to inactivate
the TTR gene in liver cells, thereby preventing 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 enrolling rapidly and continues to track ahead of the Company’s
initial projections. During the second quarter, the Company
received approval for its application under the new European Union
Clinical Trials Regulation, which enables the Phase 3 trial to
proceed in Denmark, Germany, France, Italy, Spain and Sweden. The
MAGNITUDE trial of NTLA-2001 is now cleared by regulatory agencies
in over 12 countries and actively enrolling at over 35 sites
globally.
- Hereditary ATTR Amyloidosis
with Polyneuropathy (ATTRv-PN):
- The Company plans to initiate a
pivotal Phase 3 trial of NTLA-2001 as a single-dose treatment for
ATTRv-PN by year-end. As previously announced, the study is
expected to be a small, placebo-controlled trial conducted at
ex-U.S. sites with approximately 50 ATTRv-PN patients.
- Intellia plans to present updated
data from the ongoing Phase 1 study in the second half of
2024.
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.
- In July, Intellia announced the
authorization of its Clinical Trial Application by the United
Kingdom’s Medicines and Healthcare products Regulatory Agency
(MHRA) to initiate a first-in-human study of NTLA-3001. Intellia
expects to dose the first patient in the Phase 1/2 study of
NTLA-3001 in the second half of 2024.
In Vivo Platform Expansion
- In June, Intellia presented
positive clinical proof-of-concept data that redosing with CRISPR,
utilizing the Company’s proprietary non-viral, LNP-based delivery
platform, enabled an additive pharmacodynamic effect. In the three
patients who previously received the lowest dose of 0.1 mg/kg in
the Phase 1 dose-escalation study of NTLA-2001, follow-on dosing
with a 55 mg dose of NTLA-2001 led to a deeper protein reduction.
Median reduction in serum TTR was 90% at day 28 after redosing. The
corresponding reduction from original baseline levels was a 95%
median reduction in serum TTR. NTLA-2001 was generally well
tolerated across all patients after receiving the follow-on dose.
While redosing is not planned for the NTLA-2001 program in ATTR
amyloidosis, a redosing option could be an important advantage of
Intellia’s LNP-based delivery platform for future investigational
therapies where a target additive effect is desired. These data
were presented at the Peripheral Nerve Society (PNS) Annual Meeting
in Montreal, Canada.
- 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.
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
both hematological and solid tumors.
Corporate Updates
- In June, Intellia announced the
appointment of Brian Goff to its board of directors. Mr. Goff joins
the board of directors with over three decades of
commercialization, operations and sales and marketing experience at
leading biopharmaceutical companies.
- In June, Intellia announced the
appointment of Edward Dulac as Executive Vice President, Chief
Financial Officer, and Treasurer, effective July 22, 2024. Mr.
Dulac succeeds Glenn Goddard, who stepped down from his role
effective June 30, 2024. Mr. Dulac joins Intellia with more than 20
years of combined finance, business development and corporate
strategy experience.
Upcoming Events
The Company will participate in the following events during the
third quarter of 2024:
- Morgan Stanley 22nd Annual Global
Healthcare Conference, September 4, New York
- Wells Fargo Healthcare Conference,
September 4, Boston
- Cantor Global Healthcare
Conference, September 17, New York
Second Quarter 2024 Financial Results
- Cash
Position: Cash, cash equivalents and
marketable securities were $939.9 million as of June 30, 2024,
compared to $1.0 billion as of December 31, 2023. The decrease was
driven by cash used to fund operations of $234.4 million. The
Company’s investments were offset in part by $96.4 million of net
equity proceeds from the Company’s “At the Market” (ATM) program,
$35.9 million of reimbursement from collaborators, including a
one-time $30.0 million payment received in April 2024 related to
the Company’s technology collaboration with Regeneron, $25.1
million of interest income and $4.8 million in proceeds from
employee-based stock plans. The cash position is expected to fund
operations into late 2026.
- Collaboration
Revenue: Collaboration revenue was $7.0
million during the second quarter of 2024, compared to $13.6
million during the second quarter of 2023. The $6.6 million
decrease was mainly driven by a reduction in revenue related to the
AvenCell license and collaboration agreement.
- R&D
Expenses: Research and development
(R&D) expenses were $114.2 million during the second quarter of
2024, compared to $115.3 million during the second quarter of 2023.
The $1.1 million decrease was primarily driven by a decrease in
employee-related expenses. Stock-based compensation expense
included in R&D expenses was $25.4 million for the second
quarter of 2024.
- G&A
Expenses: General and administrative
(G&A) expenses were $31.8 million during the second quarter of
2024, compared to $30.7 million during the second quarter of 2023.
The $1.1 million increase was primarily related to stock-based
compensation. Stock-based compensation expense included in G&A
expenses was $15.4 million for the second quarter of 2024.
- Net
Loss: Net loss was $147.0 million for the
second quarter of 2024, compared to $123.7 million during the
second quarter of 2023.
Conference Call to Discuss Second Quarter 2024
Results
The Company will discuss these results on a conference call
today, Thursday, August 8 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 8 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, now known as nexiguran ziclumeran
or “nex-z”, 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 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 be a functional cure for patients with HAE and to
demonstrate 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 normal levels after a
single dose; the expansion of its CRISPR/Cas9 technology and
related novel technologies, including advancing gene editing
programs in tissues outside of the liver, such as bone marrow,
brain, muscle, lung and eye, and the potential of those programs to
dramatically expand the opportunities for CRISPR-based treatments;
its ability to advance multiple ex vivo programs utilizing its
allogeneic platform, which is designed to avoid both T cell- and NK
cell-mediated rejection, for the treatment of immuno-oncology and
autoimmune diseases, and to combine its allogeneic platform with
edits to enhance cell function and offer a new approach to target
both hematological and solid tumors; 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; 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, 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.
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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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2024 |
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2023 |
|
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|
2024 |
|
|
|
2023 |
|
|
|
Collaboration revenue |
|
$ |
6,957 |
|
|
$ |
13,594 |
|
|
$ |
35,892 |
|
|
$ |
26,200 |
|
|
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
Research and development |
|
114,207 |
|
|
|
115,276 |
|
|
|
226,054 |
|
|
|
212,392 |
|
|
|
General and administrative |
|
31,793 |
|
|
|
30,652 |
|
|
|
62,884 |
|
|
|
58,100 |
|
|
|
Total operating expenses |
|
146,000 |
|
|
|
145,928 |
|
|
|
288,938 |
|
|
|
270,492 |
|
|
|
Operating loss |
|
|
|
(139,043 |
) |
|
|
(132,334 |
) |
|
|
(253,046 |
) |
|
|
(244,292 |
) |
|
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Other (expense) income, net: |
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|
|
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Interest income |
|
|
12,422 |
|
|
|
12,653 |
|
|
|
25,054 |
|
|
|
24,633 |
|
|
|
Change in fair value of investments, net |
|
(20,354 |
) |
|
|
- |
|
|
|
(26,419 |
) |
|
|
- |
|
|
|
Loss from equity method investment |
|
- |
|
|
|
(4,000 |
) |
|
|
- |
|
|
|
(7,048 |
) |
|
|
Change in fair value of contingent consideration |
|
- |
|
|
|
- |
|
|
|
- |
|
|
|
(100 |
) |
|
|
Total other (expense) income, net |
|
(7,932 |
) |
|
|
8,653 |
|
|
|
(1,365 |
) |
|
|
17,485 |
|
|
|
Net loss |
$ |
(146,975 |
) |
|
$ |
(123,681 |
) |
|
$ |
(254,411 |
) |
|
$ |
(226,807 |
) |
|
|
Net loss per share, basic and diluted |
$ |
(1.52 |
) |
|
$ |
(1.40 |
) |
|
$ |
(2.64 |
) |
|
$ |
(2.58 |
) |
|
|
Weighted average shares outstanding, basic and diluted |
|
96,975 |
|
|
|
88,185 |
|
|
|
96,238 |
|
|
|
87,979 |
|
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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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June 30, 2024 |
|
December 31, 2023 |
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Cash, cash equivalents and marketable securities |
|
|
|
|
|
$ |
939,873 |
|
$ |
1,012,087 |
|
Total assets |
|
|
|
|
|
1,191,536 |
|
|
1,300,977 |
|
Total liabilities |
|
|
|
|
|
220,474 |
|
|
250,808 |
|
Total stockholders’ equity |
|
|
|
971,062 |
|
|
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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