Intellia Therapeutics, Inc. (NASDAQ:NTLA) and Regeneron
Pharmaceuticals, Inc. (NASDAQ:REGN) today announced positive
interim results from an ongoing Phase 1 clinical trial of
NTLA-2001, an investigational, in vivo CRISPR/Cas9 genome editing
therapy in development as a single-dose treatment for transthyretin
(ATTR) amyloidosis. The interim data include 12 adult patients with
ATTR amyloidosis with cardiomyopathy (ATTR-CM) with New York Heart
Association (NYHA) Class I – III heart failure. Single doses of 0.7
mg/kg and 1.0 mg/kg of NTLA-2001 were administered via intravenous
infusion, and the change from baseline in serum transthyretin (TTR)
protein concentration was measured for each patient.
Administration of NTLA-2001 led to rapid and deep reductions in
serum TTR by day 28 as follows:
Cohort |
Mean (min, max) % serum TTR reduction by
day 28 |
0.7 mg/kg, NYHA Class I/II (n=3)* |
92% (91%, 95%) |
0.7 mg/kg, NYHA Class III (n=6)* |
94% (91%, 97%) |
1.0 mg/kg, NYHA Class I/II (n=3) |
92% (90%, 95%) |
*Mean (min, max) % serum TTR reduction by day 28 for 0.7 mg/kg
cohort (n=9) was 93% (91%, 97%).
These profound reductions in serum TTR were sustained throughout
the observation period, with patient follow-up ranging from two to
six months as of the data cut-off date of July 1, 2022. These data
support NTLA-2001’s potential as a one-time treatment to
permanently inactivate the TTR gene and reduce the disease-causing
protein in people with ATTR-CM.
“ATTR amyloidosis is a multifaceted disease in need of
additional treatment options. These new interim results demonstrate
that NTLA-2001 can profoundly reduce serum TTR levels in patients
whose condition results in cardiomyopathy,” said Intellia President
and Chief Executive Officer John Leonard, M.D. “Together with the
previously reported data from the polyneuropathy arm of this
landmark study, these results strongly suggest that NTLA-2001 could
serve as a single-dose treatment regardless of disease
manifestation. At these deep and consistent levels of protein
reduction, we believe NTLA-2001 has the potential to halt and even
reverse the underlying cause of ATTR amyloidosis. Given the
similarly robust TTR reductions observed at the two doses tested,
we have selected a fixed dose comparable to the 0.7 mg/kg level for
evaluation across both arms in the ongoing dose-expansion portion
of the study. We look forward to completing the Phase 1 study as we
advance closer to a potential pivotal trial, which we expect will
include patients in the U.S.”
“We’re encouraged to see profound and sustained serum TTR
reductions in people with cardiomyopathy manifestations of this
rare and fatal disease, further bolstering the prospects for a
one-time, in vivo treatment for multiple ATTR patient groups,” said
George D. Yancopoulos, M.D., Ph.D., President and Chief Scientific
Officer of Regeneron. “Intellia and Regeneron are working together
diligently to advance this potentially groundbreaking application
of CRISPR technology, which could one day be used for many
different genetic diseases.”
At both dose levels, NTLA-2001 was generally well tolerated. Two
of 12 patients reported transient infusion reactions, which was the
only observed treatment-related adverse event. One patient in the
0.7 mg/kg dose NYHA Class III cohort experienced a Grade 3
infusion-related reaction, which resolved without clinical
consequence. Per the study protocol, this group was subsequently
expanded from three to six patients to further characterize safety
at this dose level. No additional patients in the 0.7 mg/kg dose
NYHA Class III cohort reported a treatment-related adverse event.
No clinically significant liver findings were observed at either
dose level.
The Phase 1 study, run by Intellia as the program’s development
and commercialization lead as part of a multi-target collaboration
with Regeneron, is evaluating NTLA-2001 in patients with either
ATTR-CM or hereditary ATTR amyloidosis with polyneuropathy
(ATTRv-PN). A protocol amendment has been submitted to evaluate a
fixed dose corresponding to 0.7 mg/kg in the dose-expansion
portion, with enrollment across both arms expected to be completed
by the end of 2022, subject to regulatory feedback.
NTLA-2002 Interim Clinical ResultsIn a separate
press release issued earlier today, Intellia announced positive
interim clinical data from an ongoing Phase 1/2 clinical study of
NTLA-2002, its second in vivo genome editing candidate, for the
treatment of hereditary angioedema (HAE). Please visit this link,
or the Press Releases section of the company’s website at
www.intelliatx.com.
Intellia Therapeutics Investor Event and Webcast
InformationIntellia will host a live webcast today,
Friday, September 16, 2022, at 8:00 a.m. ET, to provide a clinical
update from its in vivo portfolio, during which the company will
review these results from NTLA-2001 alongside interim data from
NTLA-2002. To join the webcast, please visit this link, or the
Events and Presentations page of the Investors & Media section
of the company’s website at www.intelliatx.com. A replay of the
webcast will be available on Intellia’s website for at least 30
days following the call.
About NTLA-2001Based on Nobel
Prize-winning CRISPR/Cas9 technology, NTLA-2001 could potentially
be the first single-dose treatment for ATTR amyloidosis. NTLA-2001
is the first investigational CRISPR therapy candidate to be
administered systemically, or through a vein, to edit genes inside
the human body. Intellia’s proprietary non-viral platform deploys
lipid nanoparticles to deliver to the liver a two-part genome
editing system: guide RNA specific to the disease-causing
gene and messenger RNA that encodes the Cas9 enzyme, which
carries out the precision editing. Robust preclinical data, showing
deep and long-lasting transthyretin (TTR) reduction following in
vivo inactivation of the target gene, supports NTLA-2001’s
potential as a single-administration therapeutic. Intellia leads
development and commercialization of NTLA-2001 as part of a
multi-target discovery, development and commercialization
collaboration with Regeneron. The global Phase 1 trial is an
open-label, multi-center, two-part study of NTLA-2001 in adults
with hereditary transthyretin amyloidosis with polyneuropathy
(ATTRv-PN) or transthyretin amyloidosis with cardiomyopathy
(ATTR-CM). Visit clinicaltrials.gov (NCT04601051) for more
details.
About Transthyretin (ATTR) Amyloidosis
Transthyretin amyloidosis, or ATTR amyloidosis, is a rare,
progressive and fatal disease. Hereditary ATTR (ATTRv) amyloidosis
occurs when a person is born with mutations in
the TTR gene, which causes the liver to produce
structurally abnormal transthyretin (TTR) protein with a propensity
to misfold. These damaged proteins build up as amyloid in the body,
causing serious complications in multiple tissues, including the
heart, nerves and digestive system. ATTRv amyloidosis predominantly
manifests as polyneuropathy (ATTRv-PN), which can lead to nerve
damage, or cardiomyopathy (ATTRv-CM), which can lead to heart
failure. Some individuals without the genetic mutation produce
non-mutated, or wild-type TTR proteins that become unstable over
time, misfolding and aggregating in disease-causing amyloid
deposits. This condition, called wild-type ATTR (ATTRwt)
amyloidosis, primarily affects the heart. There are an estimated
50,000 people worldwide living with ATTRv amyloidosis and between
200,000 and 500,000 people with ATTRwt amyloidosis.
About Intellia TherapeuticsIntellia
Therapeutics, a leading clinical-stage genome editing company, is
developing novel, potentially curative therapeutics leveraging
CRISPR-based technologies. To fully realize the transformative
potential of CRISPR-based technologies, 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 genome editing to create
new classes of genetic medicine. Learn more at intelliatx.com.
Follow us on Twitter @intelliatx.
About RegeneronRegeneron (NASDAQ: REGN) is a
leading biotechnology company that invents life-transforming
medicines for people with serious diseases. Founded and led for
nearly 35 years by physician-scientists, our unique ability to
repeatedly and consistently translate science into medicine has led
to nine FDA-approved treatments and numerous product candidates in
development, almost all of which were homegrown in our
laboratories. Our medicines and pipeline are designed to help
patients with eye diseases, allergic and inflammatory diseases,
cancer, cardiovascular and metabolic diseases, pain, hematologic
conditions, infectious diseases and rare diseases.
Regeneron is accelerating and improving the traditional drug
development process through our proprietary VelociSuite®
technologies, such as VelocImmune®, which uses unique genetically
humanized mice to produce optimized fully human antibodies and
bispecific antibodies, and through ambitious research initiatives
such as the Regeneron Genetics Center, which is conducting one of
the largest genetics sequencing efforts in the world.
For additional information about the company, please
visit www.regeneron.com or follow @Regeneron on
Twitter.
Intellia 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 conduct and complete
clinical studies for NTLA-2001 for the treatment of transtherytin
amyloidosis (ATTR); its ability to generate data to demonstrate
NTLA-2001 as a potential single-dose treatment for ATTR; the belief
that NTLA-2001 can halt and potentially even reverse the underlying
cause of ATTR; its ability to develop its modular platform and
full-spectrum approach to advance its complex genome editing
capabilities, including to apply its proprietary CRISPR/Cas9
technology platform to additional product candidates; the
advancement and expansion of its CRISPR/Cas9 technology to develop
human therapeutic products; its ability to maintain and expand its
related intellectual property portfolio, and avoid or acquire
rights to valid intellectual property of third parties; its ability
to demonstrate its platform’s modularity and replicate or apply
results achieved in preclinical studies, including those in its
NTLA-2001 program, in any future studies, including human clinical
trials; its ability to develop other in vivo or ex vivo cell
therapeutics of all types, and NTLA-2001 in particular, using
CRISPR/Cas9 technology; and the timing of regulatory filings and
clinical trial execution, including enrollment and dosing of
patients.
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 the successful enrollment of patients in the Phase 1
study for NTLA-2001 for the treatment of ATTRv-PN or ATTR-CM; risks
related to Intellia’s ability to protect and maintain its
intellectual property position; risks related to the authorization,
initiation and conduct of studies and other development
requirements, including manufacturing, for its in vivo and ex vivo
product candidates, including NTLA-2001; the risk that any one or
more of Intellia’s product candidates, including NTLA-2001, will
not be successfully developed and commercialized; the risk that the
results of preclinical studies or clinical studies, including for
NTLA-2001, will not be predictive of future results in connection
with future studies; and the risk that Intellia’s will not be able
to demonstrate its platform’s modularity and replicate or apply
results achieved in preclinical studies to develop additional
product candidates, including to apply its proprietary CRISPR/Cas9
technology platform successfully to additional product candidates.
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 and quarterly report of 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.
Regeneron Forward-Looking Statements and Use of Digital
Media
This press release includes forward-looking statements that
involve risks and uncertainties relating to future events and
the future performance of Regeneron Pharmaceuticals,
Inc. ("Regeneron" or the "Company"), and actual events or
results may differ materially from these forward-looking
statements. Words such as "anticipate," "expect," "intend," "plan,"
"believe," "seek," "estimate," variations of such words, and
similar expressions are intended to identify such forward-looking
statements, although not all forward-looking statements contain
these identifying words. These statements concern, and these risks
and uncertainties include, among others, the impact of SARS-CoV-2
(the virus that has caused the COVID-19 pandemic) on Regeneron's
business and its employees, collaborators, and suppliers and other
third parties on which Regeneron relies, Regeneron's and its
collaborators' ability to continue to conduct research and clinical
programs, Regeneron's ability to manage its supply chain, net
product sales of products marketed or otherwise commercialized by
Regeneron and/or its collaborators or licensees (collectively,
"Regeneron's Products"), and the global economy; the nature,
timing, and possible success and therapeutic applications of
Regeneron's Products and product candidates being developed by
Regeneron and/or its collaborators or licensees (collectively,
"Regeneron's Product Candidates") and research and clinical
programs now underway or planned, such as NTLA-2001 (a product
candidate being developed for transthyretin (ATTR) amyloidosis
under a multi-target discovery, development, and commercialization
collaboration between Regeneron and Intellia
Therapeutics, Inc.); the extent to which the results from the
research and development programs conducted by Regeneron and/or its
collaborators or licensees (including the Phase 1 clinical study
evaluating NTLA-2001 discussed in this press release) may be
replicated in other studies and/or lead to advancement of product
candidates to clinical trials, therapeutic applications, or
regulatory approval; the potential of the CRISPR/Cas9 genome
editing technology discussed in this press release for in vivo
therapeutic development; uncertainty of the utilization, market
acceptance, and commercial success of Regeneron's Products and
Regeneron's Product Candidates and the impact of studies (whether
conducted by Regeneron or others and whether mandated or
voluntary), including the studies discussed or referenced in this
press release, on any of the foregoing or any potential regulatory
approval of Regeneron's Products and Regeneron's Product Candidates
(such as NTLA-2001); the likelihood, timing, and scope of possible
regulatory approval and commercial launch of Regeneron's Product
Candidates (such as NTLA-2001) and new indications for Regeneron's
Products; the ability of Regeneron's collaborators, licensees,
suppliers, or other third parties (as applicable) to perform
manufacturing, filling, finishing, packaging, labeling,
distribution, and other steps related to Regeneron's Products and
Regeneron's Product Candidates; the ability of Regeneron and/or its
collaborators to manufacture and manage supply chains for multiple
products and product candidates; safety issues resulting from the
administration of Regeneron's Products and Regeneron's Product
Candidates in patients, including serious complications or side
effects in connection with the use of Regeneron's Products and
Regeneron's Product Candidates (such as NTLA-2001) in clinical
trials; determinations by regulatory and administrative
governmental authorities which may delay or restrict Regeneron's
ability to continue to develop or commercialize Regeneron's
Products and Regeneron's Product Candidates; ongoing regulatory
obligations and oversight impacting Regeneron's Products, research
and clinical programs, and business, including those relating to
patient privacy; the availability and extent of reimbursement of
Regeneron's Products from third-party payers, including private
payer healthcare and insurance programs, health maintenance
organizations, pharmacy benefit management companies, and
government programs such as Medicare and Medicaid; coverage and
reimbursement determinations by such payers and new policies and
procedures adopted by such payers; competing drugs and product
candidates that may be superior to, or more cost effective than,
Regeneron's Products and Regeneron's Product Candidates;
unanticipated expenses; the costs of developing, producing, and
selling products; the ability of Regeneron to meet any of its
financial projections or guidance and changes to the assumptions
underlying those projections or guidance; the potential for any
license, collaboration, or supply agreement, including Regeneron's
agreements with Sanofi, Bayer, and Teva Pharmaceutical Industries
Ltd. (or their respective affiliated companies, as applicable), as
well as Regeneron's collaboration with Intellia Therapeutics,
Inc. discussed in this press release, to be cancelled or
terminated; and risks associated with intellectual property of
other parties and pending or future litigation relating thereto
(including without limitation the patent litigation and other
related proceedings relating to EYLEA® (aflibercept)
Injection, Dupixent® (dupilumab), Praluent® (alirocumab),
and REGEN-COV® (casirivimab and imdevimab)), other litigation
and other proceedings and government investigations relating to the
Company and/or its operations, the ultimate outcome of any such
proceedings and investigations, and the impact any of the foregoing
may have on Regeneron's business, prospects, operating results, and
financial condition. A more complete description of these and other
material risks can be found in Regeneron's filings with
the U.S. Securities and Exchange Commission, including its
Form 10-K for the year ended December 31, 2021 and its
Form 10-Q for the quarterly period ended June 30, 2022. Any
forward-looking statements are made based on management's current
beliefs and judgment, and the reader is cautioned not to rely on
any forward-looking statements made by Regeneron. Regeneron does
not undertake any obligation to update (publicly or otherwise) any
forward-looking statement, including without limitation any
financial projection or guidance, whether as a result of new
information, future events, or otherwise.
Regeneron uses its media and investor relations website and
social media outlets to publish important information about the
Company, including information that may be deemed material to
investors. Financial and other information about Regeneron is
routinely posted and is accessible on Regeneron's media and
investor relations website (https://newsroom.regeneron.com/) and
its Twitter feed (https://twitter.com/regeneron).
Intellia Contacts:
Investors:Ian KarpSenior Vice President,
Investor Relations and Corporate
Communications+1-857-449-4175ian.karp@intelliatx.com
Lina LiSenior Director, Investor Relations and Corporate
Communications+1-857-706-1612lina.li@intelliatx.com
Media:Rebecca SpaldingTen Bridge
Communications+1-646-509-3831media@intelliatx.com
rebecca@tenbridgecommunications.com
Regeneron Contacts:
Investors: Vesna
Tosic+1-914-847-5443vesna.tosic@regeneron.com
Media: Alexandra
Bowie+1-914-847-3407alexandra.bowie@regeneron.com
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